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## Protocol Section ### Identification Module **NCT ID:** NCT05013879 **Brief Title:** Kinesiotape for Edema After Bilateral Total Knee Arthroplasty **Official Title:** Effect of Kinesiotaping on Edema Management, Pain and Function on Patients With Bilateral Total Knee Arthroplasty #### Organization Study ID Info **ID:** 2021-13203 #### Organization **Class:** OTHER **Full Name:** Montefiore Medical Center ### Status Module #### Completion Date **Date:** 2023-11-24 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2024-02-23 **Type:** ACTUAL **Last Update Submit Date:** 2024-02-21 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2023-11-24 **Type:** ACTUAL #### Start Date **Date:** 2021-10-18 **Type:** ACTUAL **Status Verified Date:** 2024-02 #### Study First Post Date **Date:** 2021-08-19 **Type:** ACTUAL **Study First Submit Date:** 2021-08-02 **Study First Submit QC Date:** 2021-08-18 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Burke Rehabilitation Hospital #### Lead Sponsor **Class:** OTHER **Name:** Montefiore Medical Center #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to determine if kinesiotaping for edema management will decrease post-operative edema in patients with bilateral total knee arthroplasty. The leg receiving kinesiotaping during inpatient rehabilitation may have decreased edema and pain and improved movement and function when compared to the leg not receiving kinesiotape. **Detailed Description:** After being informed about the study and potential risk, all patients undergoing inpatient rehabilitation after bilateral total knee arthroplasty will have Kinesio(R)Tape applied to one randomly selected leg while the other leg serves as a control. Measurement of bilateral leg circumference, knee range of motion, numerical rating scale for pain, and selected questions from the Knee Injury and Osteoarthritis Outcome Score will occur at regular intervals throughout the rehabilitation stay. Patients will receive standard rehabilitation. ### Conditions Module **Conditions:** - Arthroplasty Complications - Arthroplasty, Replacement, Knee **Keywords:** - edema - arthroplasty, knee, bilateral - kinesiotaping ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** SINGLE_GROUP **Intervention Model Description:** Repeated measures with two within-subjects factors: time and taped/untaped leg ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 65 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Kinesio(R)Tape for edema management applied to a randomly selected lower extremity plus standard inpatient rehabilitation after bilateral total knee arthroplasty **Intervention Names:** - Device: Kinesio(R)Tape for edema control **Label:** Kinesiotape leg plus standard rehabilitation **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Control leg receiving standard inpatient rehabilitation alone. **Label:** Control leg with standard rehabilitation alone **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Kinesiotape leg plus standard rehabilitation **Description:** Kinesio(R)Tape is an elastic, cotton tape with an adhesive backing. When applied for edema management, strips of Kinesio(R)Tape are applied to the lower leg in a criss-cross fashion by a physical therapist who is a Certified Kinesiotape Practitioner. **Name:** Kinesio(R)Tape for edema control **Other Names:** - kinesiotaping or kinesiological taping **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Bilateral circumferences, in centimeters, at the following points: 10 cm above the superior pole of the patella; middle of the knee joint; calf circumference at the broadest part of the calf and at 3 inches below the fibular head landmark; figure of eight method for foot and ankle circumference - a measurement from the lateral malleolus to the navicular tuberosity, under the plantar aspect of the foot towards the tuberosity of the fifth metatarsal, around to the medial malleolus, and posterior to the leg to return to the lateral malleolus. **Measure:** Change from baseline and during 1-2-day time intervals of circumferences of both knees and lower extremities **Time Frame:** During inpatient rehabilitation stay for each subject: at baseline (day 0), day 1, day 2, and every other day until day 8 #### Secondary Outcomes **Description:** Patient self-report: Pain rating for each leg on a integer scale of 0 (no pain) to 10 (worst pain imaginable) **Measure:** Change from baseline and day-to-day changes of bilateral knee pain on numerical pain rating scale **Time Frame:** During inpatient rehabilitation stay for each subject: at baseline (day 0), day 1, day 2, and every other day until day 8 **Description:** Physical therapist's measurement of active and active assistive knee range of motion (degrees) for flexion and extension using a standard goniometer **Measure:** Change from baseline and during 1-2-day time intervals for bilateral knee range of motion **Time Frame:** During inpatient rehabilitation stay for each subject: at baseline (day 0), day 1, day 2, and every other day until day 8 **Description:** Patient self-report using the KOOS sections relating to pain, stiffness, activities of daily living **Measure:** Change from baseline to Day 4 to Discharge Day for selected parts of the Knee Injury and Osteoarthritis Outcome Score (KOOS) self-report **Time Frame:** At start of study, 4 days after start of study, and day 8 **Description:** Time (sec) to rise from a seated position, walk 10 m, turn, walk back to seat, and sit down. Patient will use appropriate assistive device and have appropriate guarding by a physical therapist. **Measure:** Change from baseline and during 1-2-day time intervals for Timed Up-and-Go Test **Time Frame:** During inpatient rehabilitation stay for each subject: at baseline (day 0), day 1, day 2, and every other day until day 8 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * admitted to Burke Rehabilitation Hospital for inpatient rehabilitation within 5 days after same-day or staged bilateral total knee arthroplasty; * 50-85 years of age; * able to read and understand English or a hospital-provided translator when consenting for the study; * free from contraindications for kinesiotaping (see below); and, * able to tolerate an active rehabilitation program. Exclusion Criteria: * stage III or IV heart failure, stage III or IV renal failure; * fragile, very hairy or sensitive skin; * anesthesia or paraesthesia of any area of the lower extremity, except the surgical sites * active skin rashes or infections or skin lesions in the lower extremity; * prior history of allergic reactions to skin taping, bandaids, surgical tape; athletic tape or other skin-adhering electrode adhesives; * prior history of lower extremity lymphedema;3 * prior history of lower extremity venous or arterial disease; * post-operative complications in the surgical sites;4 * partial joint arthroplasty or revision arthroplasty of one or both knees;1,5 * inability to give informed consent offered in English or through a hospital-provided translator * age less than 50 years or over 85 years; * inability to tolerate an active rehabilitation program. **Maximum Age:** 85 Years **Minimum Age:** 50 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** White Plains **Country:** United States **Facility:** Burke Rehabilitation Hospital **State:** New York **Zip:** 10605 #### Overall Officials **Official 1:** **Affiliation:** Burke Rehabilitation Hospital **Name:** Suzanne Babyar, PT, PhD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Tornatore L, De Luca ML, Ciccarello M, Benedetti MG. Effects of combining manual lymphatic drainage and Kinesiotaping on pain, edema, and range of motion in patients with total knee replacement: a randomized clinical trial. Int J Rehabil Res. 2020 Sep;43(3):240-246. doi: 10.1097/MRR.0000000000000417. **PMID:** 32459670 **Citation:** Guney Deniz H, Kinikli GI, Onal S, Sevinc C, Caglar O, Yuksei I. Comparison of Kinesio Tape application and manual lymphatic drainage on lower extremity oedema and functions after total knee arthroplasty. [Abstract]. Ann Rheum Dis. 2018; 77: 1791. **Citation:** Donec V, Krisciunas A. The effectiveness of Kinesio Taping(R) after total knee replacement in early postoperative rehabilitation period. A randomized controlled trial. Eur J Phys Rehabil Med. 2014 Aug;50(4):363-71. Epub 2014 May 13. **PMID:** 24819349 **Citation:** Sulman M, Riaz S, Khan RR, Faisal Z, Rajput R, Noor M. Effectiveness of Kinesio Taping on pain and function after total knee arthroplasty. Pak J Med Health Sci. 2020;14:1267-1270. **Citation:** Oktas B, Vergili O. The effect of intensive exercise program and kinesiotaping following total knee arthroplasty on functional recovery of patients. J Orthop Surg Res. 2018 Sep 12;13(1):233. doi: 10.1186/s13018-018-0924-9. **PMID:** 30208939 **Citation:** Alghadir A, Anwer S, Brismee JM. The reliability and minimal detectable change of Timed Up and Go test in individuals with grade 1-3 knee osteoarthritis. BMC Musculoskelet Disord. 2015 Jul 30;16:174. doi: 10.1186/s12891-015-0637-8. **PMID:** 26223312 **Citation:** Hancock GE, Hepworth T, Wembridge K. Accuracy and reliability of knee goniometry methods. J Exp Orthop. 2018 Oct 19;5(1):46. doi: 10.1186/s40634-018-0161-5. **PMID:** 30341552 **Citation:** Unver B, Ertekin O, Karatosun V. Pain, fear of falling and stair climbing ability in patients with knee osteoarthritis before and after knee replacement: 6 month follow-up study. J Back Musculoskelet Rehabil. 2014;27(1):77-84. doi: 10.3233/BMR-130422. **PMID:** 23948839 **Citation:** Bakar Y, Ozdemir OC, Sevim S, Duygu E, Tugral A, Surmeli M. Intra-observer and inter-observer reliability of leg circumference measurement among six observers: a single blinded randomized trial. J Med Life. 2017 Jul-Sep;10(3):176-181. **PMID:** 29075347 **Citation:** Collins NJ, Roos EM. Patient-reported outcomes for total hip and knee arthroplasty: commonly used instruments and attributes of a "good" measure. Clin Geriatr Med. 2012 Aug;28(3):367-94. doi: 10.1016/j.cger.2012.05.007. Epub 2012 Jun 22. **PMID:** 22840304 ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M13066 - Name: Pain - Relevance: LOW - As Found: Unknown - ID: M7657 - Name: Edema - Relevance: HIGH - As Found: Edema ### Condition Browse Module - Meshes - ID: D000004487 - Term: Edema ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01402479 **Brief Title:** An Open-labeled Trial of Ramipril in Patients With Migraine **Official Title:** An Open-labeled Trial of Ramipril in Patients With Migraine #### Organization Study ID Info **ID:** 0408-131-005 #### Organization **Class:** OTHER **Full Name:** Seoul National University Hospital ### Status Module #### Completion Date **Date:** 2005-07 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2011-08-08 **Type:** ESTIMATED **Last Update Submit Date:** 2011-08-05 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2005-07 **Type:** ACTUAL #### Start Date **Date:** 2004-10 **Status Verified Date:** 2011-07 #### Study First Post Date **Date:** 2011-07-26 **Type:** ESTIMATED **Study First Submit Date:** 2011-07-24 **Study First Submit QC Date:** 2011-07-24 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Seoul National University Hospital #### Responsible Party **Old Name Title:** Manho Kim, MD, PhD **Old Organization:** Department of Neurology, Seoul National University Hospital ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Physiology of migraine involving renin-angiotensin systems (RAS) has been implicated. Ramipril is a broadly-used angiotensin-converting enzyme inhibitor. The investigators attempt to test the efficacy of ramipril on the prophylaxis of migraine attacks. ### Conditions Module **Conditions:** - Migraine With Hypertension **Keywords:** - migraine, - ramipril, - hypertension ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 50 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** open label single arm trial **Intervention Names:** - Drug: Ramipril **Label:** ramipril **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - ramipril **Description:** ramipril 2.5mg twice a day **Name:** Ramipril **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** headache days **Measure:** headache frequency **Time Frame:** 12 week ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patients with chronic migraine are included in this study. Migraineurs should be aged 20 to 70 years old with the ability to read and understand the self-report scales, including the headache diary, used in this study. Exclusion Criteria: 1. Medication overuse headache are excluded in this study. 2. Treatment with other ACEI or medication that may affect ARS 3. Treatment with migraine prophylactic medications or anti-hypertensive agents including β adrenergic receptor or calcium channel blockers 4. Past history of hepatic or renal dysfunction; an abnormal electrocardiography; a psychiatric disorder; a history of substance abuse; pregnancy or lactation; use of anti-psychotics, antidepressants, or anti-anxiety drugs. **Maximum Age:** 70 Years **Minimum Age:** 20 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Seoul **Country:** Korea, Republic of **Facility:** Seoul National University Hospital **Zip:** 110-744 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014652 - Term: Vascular Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000051270 - Term: Headache Disorders, Primary - ID: D000020773 - Term: Headache Disorders - ID: D000001927 - Term: Brain Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M10024 - Name: Hypertension - Relevance: HIGH - As Found: Hypertension - ID: M11852 - Name: Migraine Disorders - Relevance: HIGH - As Found: Migraine - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown - ID: M9351 - Name: Headache - Relevance: LOW - As Found: Unknown - ID: M22529 - Name: Headache Disorders - Relevance: LOW - As Found: Unknown - ID: M26657 - Name: Headache Disorders, Primary - Relevance: LOW - As Found: Unknown - ID: M5204 - Name: Brain Diseases - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000008881 - Term: Migraine Disorders - ID: D000006973 - Term: Hypertension ### Intervention Browse Module - Ancestors - ID: D000000806 - Term: Angiotensin-Converting Enzyme Inhibitors - ID: D000011480 - Term: Protease Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000000959 - Term: Antihypertensive Agents ### Intervention Browse Module - Browse Branches - Abbrev: AnAg - Name: Antihypertensive Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Infe - Name: Anti-Infective Agents ### Intervention Browse Module - Browse Leaves - ID: M19553 - Name: Ramipril - Relevance: HIGH - As Found: Behavioral intervention - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown - ID: M4134 - Name: Angiotensin-Converting Enzyme Inhibitors - Relevance: LOW - As Found: Unknown - ID: M19609 - Name: HIV Protease Inhibitors - Relevance: LOW - As Found: Unknown - ID: M14343 - Name: Protease Inhibitors - Relevance: LOW - As Found: Unknown - ID: M4277 - Name: Antihypertensive Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000017257 - Term: Ramipril ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05600179 **Brief Title:** OCTA in Epivascular Glia After Dex Implant **Official Title:** Evaluation of Changes in Epivascular Glia Before and After Intravitreal Dexamethasone Implant : an OCT Pilot Study #### Organization Study ID Info **ID:** 0110/2022 #### Organization **Class:** OTHER **Full Name:** Federico II University ### Status Module #### Completion Date **Date:** 2022-09-30 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2022-12-08 **Type:** ACTUAL **Last Update Submit Date:** 2022-12-06 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2022-09-30 **Type:** ACTUAL #### Start Date **Date:** 2021-01-01 **Type:** ACTUAL **Status Verified Date:** 2022-12 #### Study First Post Date **Date:** 2022-10-31 **Type:** ACTUAL **Study First Submit Date:** 2022-10-26 **Study First Submit QC Date:** 2022-10-28 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Federico II University #### Responsible Party **Investigator Affiliation:** Federico II University **Investigator Full Name:** Gilda Cennamo **Investigator Title:** professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The aim of this prospective study was for the first time, to analyze specific morphologic features in diabetic eyes with macular oedema, such as changes of the foveal avascular zone and the presence of epivascular glia, and see how they would change after dexamethasone intravitreal implant ### Conditions Module **Conditions:** - Diabetic Retinopathy ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 38 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Drug: Dexamethasone intravitreal implant **Label:** patients with diabetic macular edema #### Arm Group 2 **Label:** healthy patients ### Interventions #### Intervention 1 **Arm Group Labels:** - patients with diabetic macular edema **Description:** Dexamethasone intravitreal implant **Name:** Dexamethasone intravitreal implant **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Changes in epivascular glia after Dexamethasone implant in patients with diabetic retinopathy and inflammatory macular edema **Time Frame:** up to 6 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * diabetic retinopathy complicated by macular edema Exclusion Criteria: * congenital eye disorders, * previous diagnosis of other ocular diseases * history of vitreous hemorrhage **Healthy Volunteers:** True **Maximum Age:** 80 Years **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** The participans were older than 18 years with diagnosis of inflammatory diabetic macular edema glaucoma.. ### Contacts Locations Module #### Locations **Location 1:** **City:** Naples **Country:** Italy **Facility:** University of Naples "Federico II" **Zip:** 80100 ### IPD Sharing Statement Module **IPD Sharing:** YES ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012164 - Term: Retinal Diseases - ID: D000005128 - Term: Eye Diseases - ID: D000003925 - Term: Diabetic Angiopathies - ID: D000014652 - Term: Vascular Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000048909 - Term: Diabetes Complications - ID: D000003920 - Term: Diabetes Mellitus - ID: D000004700 - Term: Endocrine System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC11 - Name: Eye Diseases - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: BC19 - Name: Gland and Hormone Related Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases ### Condition Browse Module - Browse Leaves - ID: M7125 - Name: Diabetic Retinopathy - Relevance: HIGH - As Found: Diabetic Retinopathy - ID: M14999 - Name: Retinal Diseases - Relevance: LOW - As Found: Unknown - ID: M8271 - Name: Eye Diseases - Relevance: LOW - As Found: Unknown - ID: M7120 - Name: Diabetic Angiopathies - Relevance: LOW - As Found: Unknown - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown - ID: M7115 - Name: Diabetes Mellitus - Relevance: LOW - As Found: Unknown - ID: M26004 - Name: Diabetes Complications - Relevance: LOW - As Found: Unknown - ID: M7862 - Name: Endocrine System Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000003930 - Term: Diabetic Retinopathy ### Intervention Browse Module - Ancestors - ID: D000000893 - Term: Anti-Inflammatory Agents - ID: D000000932 - Term: Antiemetics - ID: D000001337 - Term: Autonomic Agents - ID: D000018373 - Term: Peripheral Nervous System Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000005765 - Term: Gastrointestinal Agents - ID: D000005938 - Term: Glucocorticoids - ID: D000006728 - Term: Hormones - ID: D000006730 - Term: Hormones, Hormone Substitutes, and Hormone Antagonists - ID: D000018931 - Term: Antineoplastic Agents, Hormonal - ID: D000000970 - Term: Antineoplastic Agents ### Intervention Browse Module - Browse Branches - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: AnEm - Name: Antiemetics - Abbrev: Gast - Name: Gastrointestinal Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M7102 - Name: Dexamethasone - Relevance: HIGH - As Found: Children - ID: M235549 - Name: Dexamethasone acetate - Relevance: LOW - As Found: Unknown - ID: M4217 - Name: Anti-Inflammatory Agents - Relevance: LOW - As Found: Unknown - ID: M4251 - Name: Antiemetics - Relevance: LOW - As Found: Unknown - ID: M8881 - Name: Gastrointestinal Agents - Relevance: LOW - As Found: Unknown - ID: M9047 - Name: Glucocorticoids - Relevance: LOW - As Found: Unknown - ID: M9789 - Name: Hormones - Relevance: LOW - As Found: Unknown - ID: M9788 - Name: Hormone Antagonists - Relevance: LOW - As Found: Unknown - ID: M20966 - Name: Antineoplastic Agents, Hormonal - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000003907 - Term: Dexamethasone ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03878979 **Brief Title:** Preoperative Immune Checkpoint Inhibitor for Patients With Primary Untreated or Recurrent/Metastatic SCCHN **Official Title:** Preoperative Immune Checkpoint Inhibitor Therapy for Patients With Primary Untreated or Recurrent/Metastatic Squamous Cell Carcinoma of the Head and Neck (RM-SCCHN) #### Organization Study ID Info **ID:** J1923 #### Organization **Class:** OTHER **Full Name:** Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins #### Secondary ID Infos **Domain:** JHM IRB **ID:** IRB00207577 **Type:** OTHER **Domain:** Bristol-Myers Squibb **ID:** CA209-9H7 **Type:** OTHER ### Status Module #### Completion Date **Date:** 2023-10-17 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2024-02-20 **Type:** ACTUAL **Last Update Submit Date:** 2024-02-15 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2023-10-17 **Type:** ACTUAL #### Start Date **Date:** 2019-07-08 **Type:** ACTUAL **Status Verified Date:** 2024-02 #### Study First Post Date **Date:** 2019-03-18 **Type:** ACTUAL **Study First Submit Date:** 2019-03-15 **Study First Submit QC Date:** 2019-03-15 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Bristol-Myers Squibb #### Lead Sponsor **Class:** OTHER **Name:** Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Oversight Has DMC:** True ### Description Module **Brief Summary:** Nivolumab (also known as BMS-936558) before surgery to people with newly diagnosed or recurrent squamous cell carcinoma of head and neck (SCCHN). **Detailed Description:** This research is being done to see if it is safe and feasible to give the investigational drugs, nivolumab (also known as BMS-936558) before surgery to people with newly diagnosed or recurrent squamous cell carcinoma of head and neck (SCCHN). Patients with recurrent disease may have a limited number of sites of metastatic (spread) squamous cell carcinoma of head and neck. Another goal of this study is to learn how nivolumab impacts the immune system's ability to treat the cancer. While nivolumab is approved by the U. S. Food and Drug Administration (FDA) for the treatment of patients with metastatic SCCHN with progression on or after platinum-based chemotherapy, the word "investigational" in this context means that the study drugs are not approved by the FDA for the treatment of head and neck cancers prior to surgery and thus is still being tested in research studies. However, the FDA is allowing the use of nivolumab in this study. This study will have two arms. Cohort (arm) 1 will examine one dose of nivolumab given about 4 weeks before surgical resection (removal) of a newly diagnosed SCCHN. Twelve patients will be enrolled to this arm. Cohort 2 will examine one dose of nivolumab given about 4 weeks before surgical resection (removal) of SCCHN which has recurred and possibly spread to distant sites, but still can be resected with one surgery. Twelve patients will be enrolled to this arm. ### Conditions Module **Conditions:** - Head and Neck Squamous Cell Carcinoma - Head and Neck Cancer - Head and Neck Cancer Metastatic ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** The study will accrue to 2 cohorts, each of which consists of about 12 patients. If enrollment on 1 cohort is significantly faster, the lesser enrolling cohort number of patients may be decreased in favor of the better enrolling cohort to keep the total number of evaluable patients at 24 patients.Total of 24 patients will be accrued to the study. ##### Masking Info **Masking:** SINGLE **Who Masked:** - PARTICIPANT **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 26 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** One dose of nivolumab given about 4 weeks before surgical resection (removal) of a newly diagnosed SCCHN. **Intervention Names:** - Drug: Nivolumab 480mg and surgical resection **Label:** Newly diagnosed SCCHN **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** One dose of nivolumab given about 4 weeks before surgical resection (removal) of SCCHN which has recurred. **Intervention Names:** - Drug: Nivolumab 480mg and surgical resection **Label:** Reccurence of SCCHN **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Newly diagnosed SCCHN - Reccurence of SCCHN **Description:** One dose of Nivolumab 480mg given four weeks prior to surgical resection. **Name:** Nivolumab 480mg and surgical resection **Other Names:** - Opdivo - ONO-4538 - BMS-936558 - MDX 1106 **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Safety of neoadjuvant nivolumab administration in patients with newly diagnosed head and neck cancer and those with locoregional recurrence or oligometastatic disease undergoing surgical resection measured by number of participants with drug related adverse events as defined by CTCAE v5.0, occurring up to 100 days after the last dose of nivolumab or 30 days after surgery (whichever is longer) **Measure:** Safety as measured by number of participants with drug-related adverse events **Time Frame:** Up to 100 days after the last dose of nivolumab or 30 days after surgery (whichever is longer) **Description:** Feasibility of neoadjuvant nivolumab administration in patients with newly diagnosed head and neck cancer and those with locoregional recurrence or oligometastatic disease undergoing surgical resection, measured by number of participants with successful completion of preoperative treatment and proceeding to surgery without any extended treatment related delays more than \> 28 days from pre-planned day 0. **Measure:** Feasibility as measured by number of participants with successful completion of preoperative treatment and no extended treatment-related delays **Time Frame:** Up to 100 days after the last dose of nivolumab or 30 days after surgery (whichever is longer) #### Secondary Outcomes **Description:** Number of participants with \< 10% residual tumor in the resection specimen. **Measure:** Major pathologic response rate **Time Frame:** Day 0 (after surgery) **Description:** Number of months until radiologic or clinical progression or death, whichever occurs first. **Measure:** Progression free survival (PFS) **Time Frame:** up to 3 years **Description:** Number of participants with response as determined by RECIST version 1.1 and immune-related response criteria (irRC). Per RECIST criteria, Complete response (CR) is a disappearance of all target lesions, Partial response (PR) is \>= 30% decrease in the sum of the largest diameter (LD) of target lesions, Progressive disease (PD) is \>= 20% increase in the sum of the LD of target lesions. Per irRC, immune-related Complete Response (irCR) is the disappearance of all lesions, measured or unmeasured, and no new lesions; an immune-related Partial Response (irPR) is a 50% drop in tumour burden from baseline as defined by the irRC; and immune-related Progressive Disease (irPD) is a 25% increase in tumour burden from the lowest level recorded. **Measure:** Radiographic response rate **Time Frame:** up to 4 weeks post-intervention ### Eligibility Module **Eligibility Criteria:** Inclusion: Cohort 1: Subjects must have histologically confirmed previously untreated squamous cell carcinoma of the head and neck which is amenable to surgical resection as part of standard of care. Cohort 2: Subjects must have histologically confirmed recurrent squamous cell carcinoma of head and neck, which is amenable for salvage surgery. Sites of recurrence may either be locoregional or distant if resection can be done ideally in one surgical field. * The primary site should be a head and neck squamous cell carcinoma (including, but not limited to oral cavity, oropharynx, hypopharynx, or larynx, paranasal sinuses, nasal cavity). Squamous cell carcinoma of unknown primary, diagnosed in lymph nodes in neck, can be included but should be tested for p16 and confirmed specific assay. * Subjects with oropharyngeal primary tumors must have confirmation of human papillomavirus (HPV) tumor status per clinical standards, although not necessary at enrollment. * Subjects must have been determined to be candidates for surgical resection by a multidisciplinary team including a surgeon, a medical oncologist and a radiation oncologist. * Subjects must have at least one lesion that can be biopsied at baseline. * Eastern Cooperative Oncology Group (ECOG) performance status 0 to 1. * Age \>18 years. * Life expectancy of greater than 6 months. * Patients must have normal organ and marrow function as defined below: * leukocytes ≥ 1,500/ microliter (mcL) * absolute neutrophil count ≥ 1,000/mcL * platelets ≥ 100,000/mcL * total bilirubin ≤ 1.5 X institutional upper limit of normal (except subjects with Gilbert syndrome, who can have total bilirubin \< 3.0 mg/dL) * AST(SGOT)/ALT(SGPT) ≤ 3 X institutional upper limit of normal * Creatinine OR creatinine clearance within normal institutional limits OR ≥ 40 mL/min (using modified Cockcroft-Gault formula) for patients with creatinine levels above institutional normal. * Resting and walking O2 saturation must remain above 90% at the time of screening * Women of childbearing potential (WOCBP) must have a negative urine or serum pregnancy test (minimum sensitivity 25 IU/L or equivalent units of HCG) within 24 hours prior to the start of study treatment. * Women must not be breastfeeding * Women of childbearing potential (WOCBP) must agree to follow instructions for method(s) of contraception for the duration of treatment with study treatment and for 5 months post-treatment completion. Women should use an adequate method(s) of contraception (Refer to nivolumab IB for WOCBP and methods of contraception to be provided) * Males who are sexually active with WOCBP must agree to follow instructions for method(s) of contraception (Refer to protocol appendix E) for the duration of treatment with study treatment(s) and 7 months post-treatment completion. In addition, male participants must be willing to refrain from sperm donation during this time. Men who are sexually active with WOCBP must agree to follow instructions for method(s) of contraception * Patient understands the study regimen, its requirements, risks and discomforts and is able and willing to sign the informed consent form. Voluntary signed and dated IRB/IEC approved written informed consent form in accordance with regulatory and institutional guidelines must be obtained before the performance of any protocol related procedures that are not part of normal patient care. Subjects must be competent to report adverse events (AEs), understand the drug dosing schedule and use of medications to control AEs. * Measurable disease - either radiologically (per RECIST) or clinically measurable on exam in order to assess treatment response. Exclusion Criteria * Any active history of a known autoimmune disease. Subjects with vitiligo, type 1 diabetes mellitus, residual hypothyroidism requiring hormone replacement, or conditions not expected to recur in the absence of an external trigger are permitted to enroll. * Patients who have had prior chemotherapy for newly diagnosed (cohort 1) or recurrent (cohort 2) head and neck cancer. In cohort 2 only, previous perioperative chemotherapy or chemoradiation for the management of localized or locally advanced disease permitted. * Patients who had prior surgical resection of distant metastasis (metastatectomy) within 3 months of enrollment. * Patients who received prior therapy with anti-PD-1, anti-PD-L1, anti-PD-L2, anti CD137, anti-CTLA-4 antibody therapies, any other antibody or drug specifically targeting T-cell co-stimulation or checkpoint pathways. Any live / attenuated vaccine (eg varicella, zoster, yellow fever, rotavirus, oral polio and measles, mumps, rubella (MMR)) during treatment and until 100 days post last dose. * Patients with uncontrolled brain metastases * Patients with brain metastases must have stable neurologic status following local therapy (surgery or radiation) for at least 2 weeks without the use of steroids or on stable or decreasing dose of \< 10mg daily prednisone (or equivalent), and must be without neurologic dysfunction that would confound the evaluation of neurologic and other adverse events. Patients with a history of carcinomatous meningitis are not eligible. * Patients who have an active concurrent malignancy that is not controlled/cured and could impact life expectancy within the next 3 years. E.g. patients with localized cutaneous squamous cell carcinoma or basal cell carcinoma or treated prostate cancer with no evidence of disease progression may be allowed to enroll after review by the study team. * Uncontrolled inter-current illness including, but not limited to, ongoing or active infection, administration of live vaccination in the prior 3 months, symptomatic congestive heart failure, unstable angina pectoris, uncontrolled cardiac arrhythmia, myocardial infarction or new onset angina within six months of enrollment, or psychiatric illness/social situations that would limit compliance with study requirements. * Women who are pregnant or nursing. * Men with female partners who are not willing to use contraception. * Active infection with hepatitis B or hepatitis C. * Patients with a condition requiring systemic treatment with either corticosteroids (\>10 mg daily prednisone equivalent) or other immunosuppressive medications within 14 days of randomization. Inhaled or topical steroids and adrenal replacement steroid doses \< 10 mg daily prednisone equivalent, are permitted in the absence of active autoimmune disease. * Patients Epstein-Barr virus + (EBV+) with nasopharynx carcinoma * Patient with HIV are excluded given the unknown risk of interaction with HAART and the unknown benefit of immunotherapy in this population. * Participants who have received a live / attenuated vaccine within 30 days of first treatment. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Baltimore **Country:** United States **Facility:** Johns Hopkins Sidney Kimmel Comprehensive Cancer Center **State:** Maryland **Zip:** 21287 #### Overall Officials **Official 1:** **Affiliation:** Johns Hopkins University **Name:** Tanguy Lim-Seiwert, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000009369 - Term: Neoplasms - ID: D000018307 - Term: Neoplasms, Squamous Cell - ID: D000009371 - Term: Neoplasms by Site ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M9348 - Name: Head and Neck Neoplasms - Relevance: HIGH - As Found: Head and Neck Cancer - ID: M5534 - Name: Carcinoma - Relevance: HIGH - As Found: Carcinoma - ID: M5550 - Name: Carcinoma, Squamous Cell - Relevance: HIGH - As Found: Squamous Cell Carcinoma - ID: M1689 - Name: Squamous Cell Carcinoma of Head and Neck - Relevance: HIGH - As Found: Head and Neck Squamous Cell Carcinoma - ID: M14850 - Name: Recurrence - Relevance: LOW - As Found: Unknown - ID: M12320 - Name: Neoplasms, Glandular and Epithelial - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: M20451 - Name: Neoplasms, Squamous Cell - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000002277 - Term: Carcinoma - ID: D000002294 - Term: Carcinoma, Squamous Cell - ID: D000006258 - Term: Head and Neck Neoplasms - ID: D000077195 - Term: Squamous Cell Carcinoma of Head and Neck ### Intervention Browse Module - Ancestors - ID: D000074322 - Term: Antineoplastic Agents, Immunological - ID: D000000970 - Term: Antineoplastic Agents - ID: D000082082 - Term: Immune Checkpoint Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M1854 - Name: Nivolumab - Relevance: HIGH - As Found: Prospective - ID: M2342 - Name: Immune Checkpoint Inhibitors - Relevance: LOW - As Found: Unknown - ID: M1346 - Name: Antineoplastic Agents, Immunological - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000077594 - Term: Nivolumab ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04175379 **Brief Title:** The Effect of Permissive Hypercapnia on Oxygenation and Post-operative Pulmonary Complication During One-lung Ventilation **Official Title:** The Effect of Permissive Hypercapnia on Oxygenation and Post-operative Pulmonary Complication During One-lung Ventilation : Prospective, Randomized Controlled Study #### Organization Study ID Info **ID:** 4-2019-0904 #### Organization **Class:** OTHER **Full Name:** Yonsei University ### Status Module #### Completion Date **Date:** 2021-10 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** ENROLLING_BY_INVITATION #### Last Update Post Date **Date:** 2020-01-02 **Type:** ACTUAL **Last Update Submit Date:** 2019-12-30 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2021-08 **Type:** ESTIMATED #### Start Date **Date:** 2019-11-25 **Type:** ACTUAL **Status Verified Date:** 2019-12 #### Study First Post Date **Date:** 2019-11-25 **Type:** ACTUAL **Study First Submit Date:** 2019-11-15 **Study First Submit QC Date:** 2019-11-21 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Yonsei University #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Permissive hypercapnia increased the survival rate in patients with acute respiratory distress syndrome (ARDS) who required mechanical ventilation in critical care medicine. This has been explained by its association with ventilator induced lung injury. Since then, a protective lung ventilation strategy has been very important, with a low tidal volume of 4-6 ml/kg. Patients undergoing surgery will inevitably require mechanical ventilation. In particular, patients undergoing one lung ventilation for thoracic surgery may have increased airway pressure and a greater chance of ventilator induced lung injury. Recently, protective lung ventilation has been applied to patients undergoing one ung ventilation during thoracic surgery. The purpose of this study is to evaluate the difference in the degree of pulmonary oxygenation and the incidence of postoperative pulmonary complications in hypercapnia induced by controlling the respiratory rate with a constant tidal volume. ### Conditions Module **Conditions:** - Thoracic Surgery ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** Random sampling using random numbers is divided into three groups, and the ratio of each group is 1: 1: 1. ##### Masking Info **Masking:** TRIPLE **Masking Description:** Patients, care givers and outcomes assessors are blinded. The investigator should not be included in the blind because they need to adjust the ventilator settings. **Who Masked:** - PARTICIPANT - CARE_PROVIDER - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 279 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** In group 40, target PaCO2 is 40 during surgery **Intervention Names:** - Other: group 40 **Label:** group 40 **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** In group 50, target PaCO2 is 50 during surgery **Intervention Names:** - Other: group 50 **Label:** group 50 **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** In group 60, target PaCO2 is 60 during surgery **Intervention Names:** - Other: group 60 **Label:** group 60 **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - group 40 **Description:** During surgery, the TV(tidal volume) should maintain 6ml/kg (ideal body weight). After position change and OLV(one lung ventilation) for operation, each patient adjusts RR(respiratory rate) to reach target PaCO2 40 ± 5mmHg. Hemodynamic records and arterial blood tests are performed at the following times: After tracheal intubation, 15 minutes after in two lung ventilatory state at the supine position (T0), after 30 minutes reaching to the target PaCO2 by adjusting RR at the lateral position starting one lung ventilation (T1), and after 60 minutes while maintaining target PaCO2 (T2). **Name:** group 40 **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - group 50 **Description:** During surgery, the TV(tidal volume) should maintain 6ml/kg (ideal body weight). After position change and OLV(one lung ventilation) for operation, each patient adjusts RR(respiratory rate) to reach target PaCO2 50 ± 5mmHg. Hemodynamic records and arterial blood tests are performed at the following times: After tracheal intubation, 15 minutes after in two lung ventilatory state at the supine position (T0), after 30 minutes reaching to the target PaCO2 by adjusting RR at the lateral position starting one lung ventilation (T1), and after 60 minutes while maintaining target PaCO2 (T2). **Name:** group 50 **Type:** OTHER #### Intervention 3 **Arm Group Labels:** - group 60 **Description:** During surgery, the TV(tidal volume) should maintain 6ml/kg (ideal body weight). After position change and OLV(one lung ventilation) for operation, each patient adjusts RR(respiratory rate) to reach target PaCO2 60 ± 5mmHg. Hemodynamic records and arterial blood tests are performed at the following times: After tracheal intubation, 15 minutes after in two lung ventilatory state at the supine position (T0), after 30 minutes reaching to the target PaCO2 by adjusting RR at the lateral position starting one lung ventilation (T1), and after 60 minutes while maintaining target PaCO2 (T2). **Name:** group 60 **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** (arterial oxygen partial pressure / fractional inspired oxygen) at the time of T2 (PaO2 of ABGA/FiO2) T2 **Measure:** PaO2/FiO2 ratio **Time Frame:** about 60 minutes after reaching to the target PaCO2 (T2) #### Secondary Outcomes **Description:** desaturation event (\<90%) the first 3 days after surgery **Measure:** Post-op complication: desaturation event **Time Frame:** first 3 days after surgery **Description:** necessity of oxygen therapy within the first 2\~7 days after surgery hospitalized days, ICU days, expire **Measure:** Post-op complication: oxygen therapy **Time Frame:** first 2~7 days after surgery **Description:** The presence or absence of post operative complication like pneumonia, acute lung injury, re-intubation, ICU admission, ventilator care, empyema, broncho-pleura fistula, air-leakage, pleural effusion, pulmonary embolism, tracheostomy, wound infection, AKI, MI, etc. **Measure:** Post-op complication **Time Frame:** 30 days after surgery **Description:** length of hospitalized stays CU days, expire **Measure:** Post-op complication: hospitalized days **Time Frame:** 30 days after surgery **Description:** length of ICU stays **Measure:** Post-op complication: ICU days **Time Frame:** 30 days after surgery **Description:** patient has been dead or not **Measure:** Dead **Time Frame:** 30 days after surgery ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Adult patients aged 40-80 years who are planning to have thoracoscopic single lobectomy or segmentectomy with one lung ventilation during surgery. 2. American Society of Anesthesiologists (ASA) classification 1\~3 Exclusion Criteria: 1. patients with heart failure (NYHA class III\~IV) 2. patients who are having moderate obstructive lung disease or restrictive lung disease 3. Low DLCO (\< 75%) 4. patients with brain disease history or increased ICP 5. patients with pulmonary hypertension (mean PAP\>25mmHg) 6. patients with liver disease (AST level ≥100 IU/mL or ALT ≥ level 50 IU/L) or kidney disease (Creatine level ≥ 1.5 mg/dL) 7. patients with pre-existing hypercapnia or metabolic acidosis 8. body mass index (BMI) \> 30 kg/m2 9. patients who have had contralateral lung surgery 10. patients who cannot read explanation and consent form 11. patients who are pregnant **Healthy Volunteers:** True **Maximum Age:** 80 Years **Minimum Age:** 40 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Seoul **Country:** Korea, Republic of **Facility:** Department of Anaesthesiology and Pain Medicine, Anaesthesia and Pain Research Institute, Yonsei University College of Medicine **Zip:** 03722 ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012818 - Term: Signs and Symptoms, Respiratory ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M9986 - Name: Hypercapnia - Relevance: HIGH - As Found: Hypercapnia - ID: M15623 - Name: Signs and Symptoms, Respiratory - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006935 - Term: Hypercapnia ### Intervention Browse Module - Browse Branches - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Ot - Name: Other Dietary Supplements ### Intervention Browse Module - Browse Leaves - ID: M4854 - Name: Benzocaine - Relevance: LOW - As Found: Unknown - ID: T433 - Name: Tannic Acid - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03058679 **Acronym:** DINE-CD **Brief Title:** Trial of Specific Carbohydrate and Mediterranean Diets to Induce Remission of Crohn's Disease **Official Title:** Open Label, Randomized, Multicenter, Comparative Effectiveness Trial of Specific Carbohydrate and Mediterranean Diets to Induce Remission in Patients With Crohn's Disease #### Organization Study ID Info **ID:** 825907 #### Organization **Class:** OTHER **Full Name:** University of Pennsylvania ### Status Module #### Completion Date **Date:** 2020-03-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-07-21 **Type:** ACTUAL **Last Update Submit Date:** 2021-06-30 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2020-03-01 **Type:** ACTUAL #### Results First Post Date **Date:** 2021-07-21 **Type:** ACTUAL **Results First Submit Date:** 2021-03-26 **Results First Submit QC Date:** 2021-06-30 #### Start Date **Date:** 2017-09-29 **Type:** ACTUAL **Status Verified Date:** 2021-06 #### Study First Post Date **Date:** 2017-02-23 **Type:** ACTUAL **Study First Submit Date:** 2017-02-16 **Study First Submit QC Date:** 2017-02-16 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Patient-Centered Outcomes Research Institute **Class:** OTHER **Name:** Crohn's and Colitis Foundation **Class:** OTHER **Name:** University of North Carolina, Chapel Hill #### Lead Sponsor **Class:** OTHER **Name:** University of Pennsylvania #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** This protocol is designed to compare the effectiveness of two dietary interventions for patients with Crohn's disease (CD): the Specific Carbohydrate Diet (SCD) and a Mediterranean style diet (MSD) that has been demonstrated to have numerous other health benefits. The two diets will be compared in terms of their ability to resolve both the symptoms and bowel inflammation that characterize this debilitating disease. **Detailed Description:** This study tested whether the SCD is superior to a MSD for managing symptoms and reducing inflammatory markers in patients with CD. The study was designed to include 194 patients with CD who have 1) active symptoms defined by a short Crohn's Disease Activity Index (sCDAI) score \>175. Although the initial plan was to also require that all patients have active inflammation documented by a fecal calprotectin (FCP) concentration \>250mcg/g or high sensitivity C-reactive protein (CRP) \>7 mg/L or ulceration of the small bowel and/or colon consistent with an SES-CD score \>4 as documented in routine clinical practice within 4 weeks of screening, a decision was made early in the recruitment period to enroll all patients with CD with sCDAI\>175 regardless of the results of the tests for inflammatory markers and to perform subgroup analyses of those with and without evidence of inflammation. Eligible participants were randomly assigned to follow one of the diets in a 1:1 ratio. Participants were provided with 3 meals and 2 snacks each day for a period of 6 weeks. The meals and snacks were prepared by the food vendor, Healthy Chef Creations, and were delivered directly to the participant's home once per week. Participants in both groups were also provided with instructions on how to follow the diets on their own. Participants reported their symptoms through an electronic diary and provided stool samples for FCP measurement and blood for CRP measurement at weeks 0, 6 and 12. The primary and secondary outcomes were assessed at week 6. Following week 6, participants were able to pay out of pocket to purchase food from Healthy Chef Creations and/or could attempt to follow their assigned diet completely on their own. At week 12, in addition to the primary and secondary outcomes assessed again ### Conditions Module **Conditions:** - Crohn Disease **Keywords:** - diet - specific carbohydrate diet - Mediterranean style diet - randomized controlled trial ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 197 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** For the first six 6 weeks of the trial, participants received a weekly delivery of prepared meals compliant with the SCD (breakfast, lunch, dinner, and two 2 snacks per day). Meals were prepared by Healthy Chef Creations (Orlando, FL) based on menus developed by the food vendor in consultation with study dietitians. Participants assigned to the SCD received a three3-day starter diet as recommended in Breaking the Vicious Cycle. Meals were designed to be heated in an oven or microwave. No other preparation was required. **Intervention Names:** - Other: Diet **Label:** Specific Carbohydrate Diet **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** For the first six 6 weeks of the trial, participants received a weekly delivery of prepared meals compliant with the MD (breakfast, lunch, dinner, and two 2 snacks per day). Meals were prepared by Healthy Chef Creations (Orlando, FL) based on menus developed by the food vendor in consultation with study dietitians. Meals were designed to be heated in an oven or microwave. No other preparation was required. **Intervention Names:** - Other: Diet **Label:** Mediterranean Style Diet **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Mediterranean Style Diet - Specific Carbohydrate Diet **Description:** food for the diet will be provided to the participants for 6 weeks and participants will follow the diet on their own for the remaining 6 weeks **Name:** Diet **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Assessed by Short Crohn's Disease Activity Index (sCDAI) - diarrhea, abdominal pain and general well being; sCDAI \<150 in the absence of initiation or increase of any CD medications **Measure:** Percentage of Participants That Achieved Symptomatic Remission at Week 6 **Time Frame:** 6 weeks **Description:** reduction of calprotectin to less than 250 μg/g and by greater than 50% from screening among those with screening FC \>250 μg/g **Measure:** Reduction in Bowel Inflammation Among Those Whose Screening Fecal Calprotectin (FC) Was Greater Than 250μg/g at Baseline and Who Had an FC Results at Both Baseline and Week 6 **Time Frame:** 6 weeks #### Secondary Outcomes **Description:** Assessed by the CDAI - CDAI \<150 **Measure:** Percentage of Participants That Reached Clinical Remission at Week 6 **Time Frame:** 6 weeks **Description:** reduction in high-sensitivity CRP (hsCRP) to \<5 mg/L and \>50% reduction from screening among those with screening hsCRP \>5mg/L **Measure:** Percentage of Participants With a Reduction in Systemic Inflammation at Week 6 **Time Frame:** 6 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria 1. Age ≥18 2. Documented diagnosis of Crohn's disease 3. sCDAI score \>175 4. Documentation of receipt of a baseline stool sample by the data coordinating center and hsCRP. 5. Access to a computer with internet and the ability to complete daily online surveys 6. Capable of providing consent to participate 7. Able to receive weekly food shipments delivered every Friday for 6 weeks Exclusion Criteria 1. Pregnancy 2. sCDAI \>400 3. Hospitalized patients 4. Anticipated need for surgery within 6 weeks of randomization 5. Use of the Specific Carbohydrate Diet within 4 weeks of screening 6. Start or change\*\*\* dose of thiopurines (azathioprine and 6-MP), methotrexate, natalizumab, or vedolizumab within 12 weeks prior to screening 7. Start or change\*\*\* dose of anti-tumor necrosis factor (TNF) agents (including infliximab (Remicade), adalimumab (Humira), certolizumab pegol (Cimzia), golimumab (Simponi) or ustekinumab within 8 weeks prior to screening. 8. Start or change in dose of any 5-aminosalicylic acid (5-ASA) medications within 2 weeks of screening. 9. Start or change dose of corticosteroids within 1 week of screening or a dose \>20mg/day prednisone or equivalent\* 10. Use of antibiotics (other than topical formulations) for any reason within 2 weeks prior to screening 11. Known symptomatic intestinal stricture. 12. Presence of an ostomy 13. Baseline stool frequency \>4 bowel movements/day when well 14. BMI \<16 15. BMI ≥40 16. Celiac disease 17. Documented C difficile colitis within four weeks of screening 18. Diabetes Mellitus requiring medication 19. Albumin\<2.0mg/dl, within 4 weeks of screening (if tested as part of routine clinical care) 20. Known allergy to tree nuts or peanuts 21. Other conditions that would be a contraindication to any of the study diets or preclude the participant from completing the study. 22. Currently participating in another clinical trial of a drug to treat Inflammatory Bowel Disease (IBD) or a dietary therapy for any indication. * Patients may continue these medications at stable dose for the first six weeks and budesonide may be used at any dose. After the 6th week in the study, patients may taper their steroid dose. The study will provide a recommended taper schedule. * Loading/induction doses of biologic type medication will be considered a stable doses. \*\*\*Exception for treatment failures: if a subject is determined to fail on any of the following standard lines of treatment at the treating investigator's discretion, subjects may screen for study intervention based upon the following wash out periods: 4 weeks for thiopurine and methotrexate and 8 weeks for natalizumab, vedolizumab, anti-TNF, or ustekinumab. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Tucson **Country:** United States **Facility:** University of Arizona **State:** Arizona **Zip:** 85724 **Location 2:** **City:** San Francisco **Country:** United States **Facility:** UCSF Colitis and Crohn's Disease Center **State:** California **Zip:** 94115 **Location 3:** **City:** Denver **Country:** United States **Facility:** University of Colorado Denver **State:** Colorado **Zip:** 80204 **Location 4:** **City:** Atlanta **Country:** United States **Facility:** Emory University **State:** Georgia **Zip:** 30322 **Location 5:** **City:** Atlanta **Country:** United States **Facility:** Atlanta Gastroenterology **State:** Georgia **Zip:** 30342 **Location 6:** **City:** Chicago **Country:** United States **Facility:** University of Chicago **State:** Illinois **Zip:** 60614 **Location 7:** **City:** Evanston **Country:** United States **Facility:** NorthShore University HealthSystem **State:** Illinois **Zip:** 60201 **Location 8:** **City:** Evanston **Country:** United States **Facility:** Northwestern University **State:** Illinois **Zip:** 60208 **Location 9:** **City:** Indianapolis **Country:** United States **Facility:** Indiana University Health University Hospital **State:** Indiana **Zip:** 46202 **Location 10:** **City:** Louisville **Country:** United States **Facility:** The University of Louisville **State:** Kentucky **Zip:** 40202 **Location 11:** **City:** Baltimore **Country:** United States **Facility:** University of Maryland Baltimore **State:** Maryland **Zip:** 21201 **Location 12:** **City:** Boston **Country:** United States **Facility:** Boston Children's Hospital **State:** Massachusetts **Zip:** 02115 **Location 13:** **City:** Chesterfield **Country:** United States **Facility:** Clinical Research Institute of Michigan **State:** Michigan **Zip:** 48047 **Location 14:** **City:** Troy **Country:** United States **Facility:** Troy Gastroenterology **State:** Michigan **Zip:** 48092 **Location 15:** **City:** Minneapolis **Country:** United States **Facility:** The University of Minnesota **State:** Minnesota **Zip:** 55455 **Location 16:** **City:** Plymouth **Country:** United States **Facility:** Minnesota Gastroenterology, P.A **State:** Minnesota **Zip:** 55446 **Location 17:** **City:** Rochester **Country:** United States **Facility:** Mayo Clinic - Rochester **State:** Minnesota **Zip:** 55905 **Location 18:** **City:** Lebanon **Country:** United States **Facility:** Dartmouth-Hitchcock Medical Center **State:** New Hampshire **Zip:** 03756 **Location 19:** **City:** New York **Country:** United States **Facility:** NYU Langone Medical Center **State:** New York **Zip:** 10016 **Location 20:** **City:** New York **Country:** United States **Facility:** Weill Cornell - NewYork Presbyterian **State:** New York **Zip:** 10021 **Location 21:** **City:** New York **Country:** United States **Facility:** Icahn School of Medicine at Mount Sinai **State:** New York **Zip:** 10029 **Location 22:** **City:** New York **Country:** United States **Facility:** Lenox Hill Hospital **State:** New York **Zip:** 10075 **Location 23:** **City:** Chapel Hill **Country:** United States **Facility:** The University of North Carolina **State:** North Carolina **Zip:** 27599 **Location 24:** **City:** Charlotte **Country:** United States **Facility:** Atrium Health (formerly Carolinas HealthCare System) **State:** North Carolina **Zip:** 28203 **Location 25:** **City:** Winston-Salem **Country:** United States **Facility:** Wake Forest Baptist Medical Center **State:** North Carolina **Zip:** 27157 **Location 26:** **City:** Cincinnati **Country:** United States **Facility:** University of Cincinnati **State:** Ohio **Zip:** 45267 **Location 27:** **City:** Cleveland **Country:** United States **Facility:** University Hospitals Cleveland Medical Center **State:** Ohio **Zip:** 44106 **Location 28:** **City:** Columbus **Country:** United States **Facility:** Ohio State University - Wexner Medical Center **State:** Ohio **Zip:** 43210 **Location 29:** **City:** Philadelphia **Country:** United States **Facility:** University of Pennsylvania **State:** Pennsylvania **Zip:** 19104 **Location 30:** **City:** Pittsburgh **Country:** United States **Facility:** University of Pittsburgh Medical Center **State:** Pennsylvania **Zip:** 15213 **Location 31:** **City:** Providence **Country:** United States **Facility:** Lifespan Health System **State:** Rhode Island **Zip:** 02903 **Location 32:** **City:** Nashville **Country:** United States **Facility:** Vanderbilt University Medical Center **State:** Tennessee **Zip:** 37232 **Location 33:** **City:** Salt Lake City **Country:** United States **Facility:** University of Utah **State:** Utah **Zip:** 84112 **Location 34:** **City:** Seattle **Country:** United States **Facility:** Virginia Mason Medical Center **State:** Washington **Zip:** 98101 **Location 35:** **City:** Madison **Country:** United States **Facility:** University of Wisconsin-Madison **State:** Wisconsin **Zip:** 53706 #### Overall Officials **Official 1:** **Affiliation:** University of Pennsylvania **Name:** James D Lewis, MD, MSCE **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Lewis JD, Sandler RS, Brotherton C, Brensinger C, Li H, Kappelman MD, Daniel SG, Bittinger K, Albenberg L, Valentine JF, Hanson JS, Suskind DL, Meyer A, Compher CW, Bewtra M, Saxena A, Dobes A, Cohen BL, Flynn AD, Fischer M, Saha S, Swaminath A, Yacyshyn B, Scherl E, Horst S, Curtis JR, Braly K, Nessel L, McCauley M, McKeever L, Herfarth H; DINE-CD Study Group. A Randomized Trial Comparing the Specific Carbohydrate Diet to a Mediterranean Diet in Adults With Crohn's Disease. Gastroenterology. 2021 Sep;161(3):837-852.e9. doi: 10.1053/j.gastro.2021.05.047. Epub 2021 May 27. Erratum In: Gastroenterology. 2022 Nov;163(5):1473. **PMID:** 34052278 ## Document Section ### Large Document Module #### Large Docs - Date: 2019-04-10 - Filename: Prot_SAP_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: True - Label: Study Protocol and Statistical Analysis Plan - Size: 1727953 - Type Abbrev: Prot_SAP - Upload Date: 2020-12-23T10:06 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000015212 - Term: Inflammatory Bowel Diseases - ID: D000005759 - Term: Gastroenteritis - ID: D000005767 - Term: Gastrointestinal Diseases - ID: D000004066 - Term: Digestive System Diseases - ID: D000007410 - Term: Intestinal Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC06 - Name: Digestive System Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M6638 - Name: Crohn Disease - Relevance: HIGH - As Found: Crohn's Disease - ID: M10444 - Name: Intestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M17917 - Name: Inflammatory Bowel Diseases - Relevance: LOW - As Found: Unknown - ID: M8875 - Name: Gastroenteritis - Relevance: LOW - As Found: Unknown - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000003424 - Term: Crohn Disease ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** As per the DINE-CD protocol, allergic reaction to a component of the food, intolerance of the food other than allergic reaction, worsening of CD and worsening of extraintestinal manifestations of CD was considered expected. AEs were included for all randomized participants who started the diet with the exception of 3 participant who were randomized in error with an sCDAI \<150 (as noted in the participant flow). #### Event Groups **Group ID:** EG000 **Title:** Mediterranean Style Diet **Deaths Num At Risk:** 92 **Description:** Diet: food for the diet will be provided to the participants for 6 weeks and participants will follow the diet on their own for the remaining 6 weeks **ID:** EG000 **Other Num Affected:** 18 **Other Num at Risk:** 92 **Serious Number Affected:** 5 **Serious Number At Risk:** 92 **Title:** Mediterranean Style Diet **Group ID:** EG001 **Title:** Specific Carbohydrate Diet **Deaths Num At Risk:** 99 **Description:** Diet: food for the diet will be provided to the participants for 6 weeks and participants will follow the diet on their own for the remaining 6 weeks **ID:** EG001 **Other Num Affected:** 19 **Other Num at Risk:** 99 **Serious Number Affected:** 3 **Serious Number At Risk:** 99 **Title:** Specific Carbohydrate Diet **Frequency Threshold:** 5 #### Other Events **Term:** Abdominal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Diarrhea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Gastrointestingal disorders other - Worsening of Crohn's disease symptons **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (4.0) #### Serious Events **Term:** Small intestinal obstruction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (4.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 92 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 99 **Num Events:** 1 **Term:** Abdominal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (4.0) ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 92 **Num Events:** 3 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 99 **Num Events:** 3 **Term:** Worsening of Crohn's disease **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (4.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 92 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 99 **Num Events:** 2 **Term:** Rectal hemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (4.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 92 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 99 **Term:** Diarrhea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (4.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 92 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 99 **Term:** Nausea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (4.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 92 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 99 **Term:** Vomiting **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (4.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 92 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 99 **Time Frame:** AEs were recorded at each study contact from screening visit to a participant's end of study visit. For participants that completed the study, AEs were assessed at screening, baseline, wk 6 and week 12. Among participants who withdrew early from the study, AEs were assessed at the above named timepoints until the time of withdraw. The clinical course of each AE was followed until resolution, stabilization, or until it was determined that study intervention or participation was not the cause. ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 92 **Group ID:** BG001 **Value:** 99 **Group ID:** BG002 **Value:** 191 **Units:** Participants ### Group **ID:** BG000 **Title:** Mediterranean Style Diet **Description:** Diet: food for the diet will be provided to the participants for 6 weeks and participants will follow the diet on their own for the remaining 6 weeks ### Group **ID:** BG001 **Title:** Specific Carbohydrate Diet **Description:** Diet: food for the diet will be provided to the participants for 6 weeks and participants will follow the diet on their own for the remaining 6 weeks ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 29.5 **Upper Limit:** 53.0 **Value:** 37.0 #### Measurement **Group ID:** BG001 **Lower Limit:** 27.0 **Upper Limit:** 46.0 **Value:** 36.0 #### Measurement **Group ID:** BG002 **Lower Limit:** 28.0 **Upper Limit:** 50.0 **Value:** 37 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 63 #### Measurement **Group ID:** BG001 **Value:** 58 #### Measurement **Group ID:** BG002 **Value:** 121 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 29 #### Measurement **Group ID:** BG001 **Value:** 41 #### Measurement **Group ID:** BG002 **Value:** 70 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 4 #### Measurement **Group ID:** BG001 **Value:** 4 #### Measurement **Group ID:** BG002 **Value:** 8 **Category Title:** Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 88 #### Measurement **Group ID:** BG001 **Value:** 92 #### Measurement **Group ID:** BG002 **Value:** 180 **Category Title:** Not Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 3 #### Measurement **Group ID:** BG002 **Value:** 3 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 2 #### Measurement **Group ID:** BG002 **Value:** 2 **Category Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 2 #### Measurement **Group ID:** BG001 **Value:** 5 #### Measurement **Group ID:** BG002 **Value:** 7 **Category Title:** Black #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 2 **Category Title:** Multiracial #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 4 **Category Title:** Other #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 2 #### Measurement **Group ID:** BG002 **Value:** 2 **Category Title:** Unknown #### Measurement **Group ID:** BG000 **Value:** 86 #### Measurement **Group ID:** BG001 **Value:** 88 #### Measurement **Group ID:** BG002 **Value:** 174 **Category Title:** White **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 21.3 **Upper Limit:** 29.9 **Value:** 25.1 #### Measurement **Group ID:** BG001 **Lower Limit:** 22.5 **Upper Limit:** 29.0 **Value:** 25.6 #### Measurement **Group ID:** BG002 **Lower Limit:** 21.8 **Upper Limit:** 29.5 **Value:** 25.3 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 68 #### Measurement **Group ID:** BG001 **Value:** 79 #### Measurement **Group ID:** BG002 **Value:** 147 **Category Title:** Never #### Measurement **Group ID:** BG000 **Value:** 21 #### Measurement **Group ID:** BG001 **Value:** 20 #### Measurement **Group ID:** BG002 **Value:** 41 **Category Title:** Past #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 3 **Category Title:** Current **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 5 **Upper Limit:** 21 **Value:** 10 #### Measurement **Group ID:** BG001 **Lower Limit:** 3 **Upper Limit:** 16 **Value:** 10 #### Measurement **Group ID:** BG002 **Lower Limit:** 4 **Upper Limit:** 17 **Value:** 10 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 56 #### Measurement **Group ID:** BG001 **Value:** 60 #### Measurement **Group ID:** BG002 **Value:** 116 **Category Title:** Non-stricturing, nonpenetrating #### Measurement **Group ID:** BG000 **Value:** 19 #### Measurement **Group ID:** BG001 **Value:** 17 #### Measurement **Group ID:** BG002 **Value:** 36 **Category Title:** Stricturing #### Measurement **Group ID:** BG000 **Value:** 9 #### Measurement **Group ID:** BG001 **Value:** 13 #### Measurement **Group ID:** BG002 **Value:** 22 **Category Title:** Penetrating #### Measurement **Group ID:** BG000 **Value:** 8 #### Measurement **Group ID:** BG001 **Value:** 9 #### Measurement **Group ID:** BG002 **Value:** 17 **Category Title:** Stricturing and penetrating **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 22 #### Measurement **Group ID:** BG001 **Value:** 30 #### Measurement **Group ID:** BG002 **Value:** 52 **Category Title:** Ileum alone #### Measurement **Group ID:** BG000 **Value:** 17 #### Measurement **Group ID:** BG001 **Value:** 13 #### Measurement **Group ID:** BG002 **Value:** 30 **Category Title:** Colon alone #### Measurement **Group ID:** BG000 **Value:** 53 #### Measurement **Group ID:** BG001 **Value:** 55 #### Measurement **Group ID:** BG002 **Value:** 108 **Category Title:** Ileum and Colon #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 1 **Category Title:** Missing **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 24 #### Measurement **Group ID:** BG001 **Value:** 20 #### Measurement **Group ID:** BG002 **Value:** 44 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 34 #### Measurement **Group ID:** BG001 **Value:** 29 #### Measurement **Group ID:** BG002 **Value:** 63 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 55 #### Measurement **Group ID:** BG001 **Value:** 53 #### Measurement **Group ID:** BG002 **Value:** 108 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 11 #### Measurement **Group ID:** BG001 **Value:** 11 #### Measurement **Group ID:** BG002 **Value:** 22 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 6 #### Measurement **Group ID:** BG001 **Value:** 15 #### Measurement **Group ID:** BG002 **Value:** 21 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 3 #### Measurement **Group ID:** BG002 **Value:** 3 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 11 #### Measurement **Group ID:** BG002 **Value:** 14 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 2 #### Measurement **Group ID:** BG002 **Value:** 3 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 28 #### Measurement **Group ID:** BG001 **Value:** 34 #### Measurement **Group ID:** BG002 **Value:** 62 **Category Title:** 0 #### Measurement **Group ID:** BG000 **Value:** 22 #### Measurement **Group ID:** BG001 **Value:** 29 #### Measurement **Group ID:** BG002 **Value:** 51 **Category Title:** 1 #### Measurement **Group ID:** BG000 **Value:** 32 #### Measurement **Group ID:** BG001 **Value:** 26 #### Measurement **Group ID:** BG002 **Value:** 58 **Category Title:** 2 #### Measurement **Group ID:** BG000 **Value:** 9 #### Measurement **Group ID:** BG001 **Value:** 9 #### Measurement **Group ID:** BG002 **Value:** 18 **Category Title:** 3 #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 2 **Category Title:** 4 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 22 #### Measurement **Group ID:** BG001 **Value:** 23 #### Measurement **Group ID:** BG002 **Value:** 45 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 27 #### Measurement **Group ID:** BG001 **Value:** 22 #### Measurement **Group ID:** BG002 **Value:** 49 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 1.2 **Upper Limit:** 6.2 **Value:** 2.5 #### Measurement **Group ID:** BG001 **Lower Limit:** 1.4 **Upper Limit:** 8.1 **Value:** 3.2 #### Measurement **Group ID:** BG002 **Lower Limit:** 1.4 **Upper Limit:** 7.5 **Value:** 2.9 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 28 #### Measurement **Group ID:** BG001 **Value:** 37 #### Measurement **Group ID:** BG002 **Value:** 65 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 16.0 **Upper Limit:** 185.0 **Value:** 40.0 #### Measurement **Group ID:** BG001 **Lower Limit:** 16.0 **Upper Limit:** 223.0 **Value:** 107.5 #### Measurement **Group ID:** BG002 **Lower Limit:** 16.0 **Upper Limit:** 194.0 **Value:** 70 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 13 #### Measurement **Group ID:** BG001 **Value:** 23 #### Measurement **Group ID:** BG002 **Value:** 36 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 80 #### Measurement **Group ID:** BG001 **Value:** 89 #### Measurement **Group ID:** BG002 **Value:** 169 **Category Title:** Not performed #### Measurement **Group ID:** BG000 **Value:** 8 #### Measurement **Group ID:** BG001 **Value:** 8 #### Measurement **Group ID:** BG002 **Value:** 16 **Category Title:** Yes #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 1 **Category Title:** Probably #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 2 **Category Title:** Probably not #### Measurement **Group ID:** BG000 **Value:** 2 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 3 **Category Title:** No **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 38 #### Measurement **Group ID:** BG001 **Value:** 50 #### Measurement **Group ID:** BG002 **Value:** 88 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 195.7 **Upper Limit:** 247.0 **Value:** 217.7 #### Measurement **Group ID:** BG001 **Lower Limit:** 197.0 **Upper Limit:** 263.8 **Value:** 226.0 #### Measurement **Group ID:** BG002 **Lower Limit:** 195.7 **Upper Limit:** 259.6 **Value:** 223.0 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 179.3 **Upper Limit:** 247.0 **Value:** 206.8 #### Measurement **Group ID:** BG001 **Lower Limit:** 169.8 **Upper Limit:** 246.2 **Value:** 210.0 #### Measurement **Group ID:** BG002 **Lower Limit:** 171.5 **Upper Limit:** 246.2 **Value:** 209.5 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 33.0 **Upper Limit:** 44.0 **Value:** 38.0 #### Measurement **Group ID:** BG001 **Lower Limit:** 33.0 **Upper Limit:** 45.0 **Value:** 40.0 #### Measurement **Group ID:** BG002 **Lower Limit:** 33.0 **Upper Limit:** 45.0 **Value:** 39.0 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 55.9 **Upper Limit:** 65.9 **Value:** 60.4 #### Measurement **Group ID:** BG001 **Lower Limit:** 54.8 **Upper Limit:** 65.6 **Value:** 58.9 #### Measurement **Group ID:** BG002 **Lower Limit:** 55.0 **Upper Limit:** 65.8 **Value:** 59.6 **Class Title:** Fatigue #### Measurement **Group ID:** BG000 **Lower Limit:** 55.7 **Upper Limit:** 63.8 **Value:** 60.1 #### Measurement **Group ID:** BG001 **Lower Limit:** 55.7 **Upper Limit:** 63.6 **Value:** 61.3 #### Measurement **Group ID:** BG002 **Lower Limit:** 55.7 **Upper Limit:** 63.7 **Value:** 61.2 **Class Title:** Pain interference #### Measurement **Group ID:** BG000 **Lower Limit:** 51.4 **Upper Limit:** 62.1 **Value:** 57.3 #### Measurement **Group ID:** BG001 **Lower Limit:** 49.4 **Upper Limit:** 60.2 **Value:** 54.5 #### Measurement **Group ID:** BG002 **Lower Limit:** 50.9 **Upper Limit:** 61.4 **Value:** 56.0 **Class Title:** Sleep disturbance #### Measurement **Group ID:** BG000 **Lower Limit:** 41.0 **Upper Limit:** 56.1 **Value:** 48.4 #### Measurement **Group ID:** BG001 **Lower Limit:** 34.8 **Upper Limit:** 53.8 **Value:** 47.2 #### Measurement **Group ID:** BG002 **Lower Limit:** 34.8 **Upper Limit:** 54.5 **Value:** 47.9 **Class Title:** Social isolation ### Measure #### Measurement **Group ID:** BG000 **Value:** 30 #### Measurement **Group ID:** BG001 **Value:** 40 #### Measurement **Group ID:** BG002 **Value:** 70 **Category Title:** 0 #### Measurement **Group ID:** BG000 **Value:** 39 #### Measurement **Group ID:** BG001 **Value:** 40 #### Measurement **Group ID:** BG002 **Value:** 79 **Category Title:** 1-3 #### Measurement **Group ID:** BG000 **Value:** 22 #### Measurement **Group ID:** BG001 **Value:** 18 #### Measurement **Group ID:** BG002 **Value:** 40 **Category Title:** >3 #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 2 **Category Title:** Missing **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 1.0 **Upper Limit:** 4.0 **Value:** 3.0 #### Measurement **Group ID:** BG001 **Lower Limit:** 1.0 **Upper Limit:** 3.0 **Value:** 2.0 #### Measurement **Group ID:** BG002 **Lower Limit:** 2.0 **Upper Limit:** 4.0 **Value:** 3.0 **Class Title:** **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** INTER_QUARTILE_RANGE **Parameter Type:** MEDIAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Ethnicity (NIH/OMB) **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Race/Ethnicity, Customized **Unit of Measure:** Participants ### Measure 5 **Dispersion Type:** INTER_QUARTILE_RANGE **Parameter Type:** MEDIAN **Title:** Body Mass Index (BMI) **Unit of Measure:** kg/m2 ### Measure 6 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Smoking status **Unit of Measure:** Participants ### Measure 7 **Dispersion Type:** INTER_QUARTILE_RANGE **Parameter Type:** MEDIAN **Title:** Years since CD diagnosis **Unit of Measure:** years ### Measure 8 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** CD behavior **Unit of Measure:** Participants ### Measure 9 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Crohn's disease distribution **Unit of Measure:** Participants ### Measure 10 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** History of perianal fistula **Unit of Measure:** Participants ### Measure 11 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** History of intestinal surgery **Unit of Measure:** Participants ### Measure 12 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Current use of any biologic **Unit of Measure:** Participants ### Measure 13 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Current use of immunomodulators **Unit of Measure:** Participants ### Measure 14 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Current use of oral 5-ASA **Unit of Measure:** Participants ### Measure 15 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Current use of rectal 5 ASA **Unit of Measure:** Participants ### Measure 16 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Current use of oral steroids **Unit of Measure:** Participants ### Measure 17 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Current use of rectal steroids **Unit of Measure:** Participants ### Measure 18 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Number of prior anti-TNF medications **Unit of Measure:** Participants ### Measure 19 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Prior ustekinumab **Unit of Measure:** Participants ### Measure 20 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Prior vedolizumab **Unit of Measure:** Participants ### Measure 21 **Dispersion Type:** INTER_QUARTILE_RANGE **Parameter Type:** MEDIAN **Title:** hsCRP mg/L **Unit of Measure:** mg/L ### Measure 22 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** hsCRP >5 mg/L **Unit of Measure:** Participants ### Measure 23 **Dispersion Type:** INTER_QUARTILE_RANGE **Parameter Type:** MEDIAN **Title:** fecal calprotectin (FC) ug/g **Unit of Measure:** ug/g ### Measure 24 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** fecal calprotectin (FC) >250 ug/g **Unit of Measure:** Participants ### Measure 25 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Inflammation on colonoscopy **Unit of Measure:** Participants ### Measure 26 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** FC>250 ug/g or hsCRP>5 mg/L or definite inflammation on colonoscopy **Unit of Measure:** Participants ### Measure 27 **Description:** Short Crohn's Disease Activity Index. This is a method to measure Crohn's disease patient's symptoms using only a questionnaire,which can be completed without an office visit or lab work. The variables included in the short CDAI are abdominal pain, diarrhea frequency, and general well-being. The higher the score, the worse the disease activity. The sCDAI uses the same scale as the full CDAI, such that scores,\<150 define remission, 150 to 219 mild activity, 220 to450 moderate activity, and.450 severe activity. The total score is reported. **Dispersion Type:** INTER_QUARTILE_RANGE **Parameter Type:** MEDIAN **Title:** sCDAI (median, Q1-Q3) **Unit of Measure:** units on a scale ### Measure 28 **Description:** The Crohn's Disease Activity Index - CDAI scores range from 0 to 600. A score of less than 150 denotes relative disease quiescence (remission); 150 to 219, mildly active disease; 220 to 450, moderately active disease; and greater than 450, severe disease. The higher the score, the worse the disease activity. This index uses the following variables to determine a score: number of stools, abdominal pain, general well-being, extraintestinal complications, antidiarrheal agents used in the previous 7 days, abdominal mass felt on palpation, hematocrit, and body weight **Dispersion Type:** INTER_QUARTILE_RANGE **Parameter Type:** MEDIAN **Title:** CDAI (median, Q1-Q3) **Unit of Measure:** units on a scale ### Measure 29 **Description:** The SIBDQ is a 10-item health-related quality of life (HRQOL) questionnaire validated for use in CD patients. It assesses physical, social, and emotional status and is scored on a 7-point Likert scale from 1 (severe problem) to 7 (no problems at all). Scores for each question are summed to provide a total score. The total score is reported. The absolute score ranges from 10 (poor HRQOL) to 70 (optimum HRQOL). **Dispersion Type:** INTER_QUARTILE_RANGE **Parameter Type:** MEDIAN **Title:** Short IBDQ (median, Q1-Q3) **Unit of Measure:** units on a scale ### Measure 30 **Description:** Patient-Reported Outcomes Measurement Information System (PROMIS)- set of person-centered measures that evaluate and monitor health status. All assessments are for the last 7 days. To calculate a total raw score, each item is scored on a scale of 1-5. If the form has 7 items, the lowest possible raw score is 7 and the highest possible raw score is 35. Raw scores are then translated into T scores. The T-score rescales the raw score into a standardized score with a mean of 50 and a standard deviation (SD) of 10. T-scores are reported. Higher T-scores denote worse outcomes. **Dispersion Type:** INTER_QUARTILE_RANGE **Parameter Type:** MEDIAN **Title:** PROMIS measures (median, Q1-Q3) **Unit of Measure:** units on a scale ### Measure 31 **Description:** This self-reported survey assessed five criteria defining possible inflammatory back pain:(1) improvement with exercise (2) pain at night (3) insidious onset (4) age at onset \<40 years and (5) no improvement with rest. The higher the score the more likely the person had inflammatory back pain. **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Inflammatory back pain screen **Unit of Measure:** Participants ### Measure 32 **Description:** Alternate Mediterranean Diet Score - Participants completed a 24-hour dietary recall at baseline, during week 6 and during week 12. These data were used to compute the Alternate Mediterranean Diet Score (AMeD) where a higher score implies greater consistency with a Mediterranean diet. AMeD scores range from 0 (minimal adherence) to 9 (maximal adherence). **Dispersion Type:** INTER_QUARTILE_RANGE **Parameter Type:** MEDIAN **Title:** AMeD Score **Unit of Measure:** units on a scale ## Results Section - More Information Module ### Certain Agreement ### Point of Contact **Email:** [email protected] **Organization:** University of Pennsylvania **Phone:** 215-573-5137 **Title:** Dr. James D. Lewis ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### Outcome Measure 4 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 40 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 46 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 8 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 44 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 48 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Assessed by Short Crohn's Disease Activity Index (sCDAI) - diarrhea, abdominal pain and general well being; sCDAI \<150 in the absence of initiation or increase of any CD medications **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** percentage of participants who achieved symptomatic remission at week 6 **Reporting Status:** POSTED **Time Frame:** 6 weeks **Title:** Percentage of Participants That Achieved Symptomatic Remission at Week 6 **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Diet: food for the diet will be provided to the participants for 6 weeks and participants will follow the diet on their own for the remaining 6 weeks **ID:** OG000 **Title:** Mediterranean Style Diet ##### Group **Description:** Diet: food for the diet will be provided to the participants for 6 weeks and participants will follow the diet on their own for the remaining 6 weeks **ID:** OG001 **Title:** Specific Carbohydrate Diet #### Outcome Measure 2 **Description:** reduction of calprotectin to less than 250 μg/g and by greater than 50% from screening among those with screening FC \>250 μg/g **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** This measure includes only those whose screening fecal calprotectin (FC) was greater than 250μg/g at baseline and who had an FC results at both baseline and week 6 **Reporting Status:** POSTED **Time Frame:** 6 weeks **Title:** Reduction in Bowel Inflammation Among Those Whose Screening Fecal Calprotectin (FC) Was Greater Than 250μg/g at Baseline and Who Had an FC Results at Both Baseline and Week 6 **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Diet: food for the diet will be provided to the participants for 6 weeks and participants will follow the diet on their own for the remaining 6 weeks **ID:** OG000 **Title:** Mediterranean Style Diet ##### Group **Description:** Diet: food for the diet will be provided to the participants for 6 weeks and participants will follow the diet on their own for the remaining 6 weeks **ID:** OG001 **Title:** Specific Carbohydrate Diet #### Outcome Measure 3 **Description:** Assessed by the CDAI - CDAI \<150 **Parameter Type:** COUNT_OF_PARTICIPANTS **Reporting Status:** POSTED **Time Frame:** 6 weeks **Title:** Percentage of Participants That Reached Clinical Remission at Week 6 **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Diet: food for the diet will be provided to the participants for 6 weeks and participants will follow the diet on their own for the remaining 6 weeks **ID:** OG000 **Title:** Mediterranean Style Diet ##### Group **Description:** Diet: food for the diet will be provided to the participants for 6 weeks and participants will follow the diet on their own for the remaining 6 weeks **ID:** OG001 **Title:** Specific Carbohydrate Diet #### Outcome Measure 4 **Description:** reduction in high-sensitivity CRP (hsCRP) to \<5 mg/L and \>50% reduction from screening among those with screening hsCRP \>5mg/L **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** This measure includes only those that had a screening hsCRP \> 5mg/L and had hsCRP measured at both screening and at week 6 **Reporting Status:** POSTED **Time Frame:** 6 weeks **Title:** Percentage of Participants With a Reduction in Systemic Inflammation at Week 6 **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Diet: food for the diet will be provided to the participants for 6 weeks and participants will follow the diet on their own for the remaining 6 weeks **ID:** OG000 **Title:** Mediterranean Style Diet ##### Group **Description:** Diet: food for the diet will be provided to the participants for 6 weeks and participants will follow the diet on their own for the remaining 6 weeks **ID:** OG001 **Title:** Specific Carbohydrate Diet ### Participant Flow Module #### Group **Description:** Diet: food for the diet will be provided to the participants for 6 weeks **ID:** FG000 **Title:** Specific Carbohydrate Diet #### Group **Description:** Diet: food for the diet will be provided to the participants for 6 weeks **ID:** FG001 **Title:** Mediterranean Style Diet #### Period **Title:** Baseline to Week 6 ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 3 ###### Reason **Group ID:** FG001 **Number of Subjects:** 2 ##### Withdraw **Type:** Change in Crohn's disease medication ###### Reason **Group ID:** FG000 **Number of Subjects:** 4 ###### Reason **Group ID:** FG001 **Number of Subjects:** 2 ##### Withdraw **Type:** Adverse Event ###### Reason **Group ID:** FG000 **Number of Subjects:** 6 ###### Reason **Group ID:** FG001 **Number of Subjects:** 6 ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 4 ###### Reason **Group ID:** FG001 **Number of Subjects:** 3 ##### Withdraw **Type:** Difficulty following the diet ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 2 ##### Withdraw **Type:** Physician Decision ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 99 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 92 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 81 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 77 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 18 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 15 #### Period **Title:** Week 6 to Week 12 ##### Withdraw **Type:** Change in medication ###### Reason **Group ID:** FG000 **Number of Subjects:** 5 ###### Reason **Group ID:** FG001 **Number of Subjects:** 7 ##### Withdraw **Type:** Adverse Event ###### Reason **Group ID:** FG000 **Number of Subjects:** 4 ###### Reason **Group ID:** FG001 **Number of Subjects:** 3 ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 9 ###### Reason **Group ID:** FG001 **Number of Subjects:** 4 ##### Withdraw **Type:** Difficulty following the assigned diet ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ###### Reason **Group ID:** FG001 **Number of Subjects:** 3 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 81 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 77 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 61 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 60 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 20 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 17 **Pre-Assignment Details:** There were 3 main reasons that participants who consented to the study were not randomized. The majority did not meet eligibility criteria. A small number withdrew or were lost to follow-up **Recruitment Details:** The study was conducted in 33 clinical sites across the United States. Enrollment in the trial occurred between September 29, 2017 and October 8, 2019. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT03791879 **Brief Title:** Caudal Dexmedetomidine Analgesia in Pediatrics . **Official Title:** The Analgesic Effectiveness and Safety of Upgraded Caudal Dexmedetomidine Doses in Pediatric Hypospadias Surgery. #### Organization Study ID Info **ID:** MFM IR.18.11.322 - 2018/11/11 #### Organization **Class:** OTHER **Full Name:** Mansoura University ### Status Module #### Completion Date **Date:** 2020-10-15 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-03-01 **Type:** ACTUAL **Last Update Submit Date:** 2021-02-26 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2020-10-01 **Type:** ACTUAL #### Start Date **Date:** 2019-01-01 **Type:** ACTUAL **Status Verified Date:** 2021-02 #### Study First Post Date **Date:** 2019-01-03 **Type:** ACTUAL **Study First Submit Date:** 2018-12-29 **Study First Submit QC Date:** 2018-12-31 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Mansoura University #### Responsible Party **Investigator Affiliation:** Mansoura University **Investigator Full Name:** Mohamed Abd Latif Ghanim **Investigator Title:** Associate Professor os anesthesia ICU & Pain medicine. **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** Dexmedetomidine (DEXM) is a highly selective α2-adrenoceptor agonist that has been used increasingly in pediatric anesthesia. This prospective double blinded randomized comparative study is designed to evaluate the analgesic effect of caudal increasing doses of DEXM 0.5 , 1 , 1.5 , 2µg/kg combined with Levobupivacaine (Levob) 0.125% (ED95% =125%=least effective concentration) in providing pain relief over a 24-h period and lowest surgical stress peak. Study hypothesis: Levobupivacaine 0.125 %( ED95) combined with different increasing doses of dexamedatomedine \>1 µg/kg could not add more analgesic \& stress response obtundation outcome, but increase side effects (sedation and hemodynamic depression). The peak cortisol level during urology surgery was at the end of the 1st postoperative (PO) hour. Aim of the Study: To detect the optimal analgesic and safe caudal adjuvant DEXM dose associated with the least side effects\& stress response modulation, guided by PO Cortisol peak difference in between the study groups during pediatric hypospadias surgery. **Detailed Description:** Background: Dexmedetomidine (DEXM) is a highly selective α2-adrenoceptor agonist that has been used increasingly in pediatric anesthesia. This prospective double blinded randomized comparative study is designed to evaluate the analgesic effect of caudal increasing doses of DEXM 0.5 , 1 , 1.5 , 2µg/kg combined with Levobupivacaine (Levob) 0.125% (ED95% =125%=least effective concentration) in providing pain relief over a 24-h period and lowest surgical stress peak. Study hypothesis: Levobupivacaine 0.125 %( ED95) combined with different increasing doses of dexamedatomedine \>1 µg/kg could not add more analgesic \& stress response obtundation outcome, but increase side effects (sedation and hemodynamic depression).The peak cortisol level during urology surgery was at the end of the 1st postoperative (PO) hour. Aim of the Study: To detect the optimal analgesic and safe caudal adjuvant DEXM dose associated with the least side effects\& stress response modulation, guided by PO Cortisol peak difference in between the study groups during pediatric hypospadias surgery. Optimal effective dose defined as; the least caudal DEXM dose which produce the best analgesic outcome \[least time to 1st analgesic request and lowest rescue analgesic dose (24Hs)\] associated with the least stress response (cortisol PO. first One hour peak), least PO sedation, and hemodynamic stability IO. \&PO. HR \& MAP. The peak cortisol level during urology surgery was at the end of the 1st postoperative (PO) hour. . Material \& Methods A prospective randomized double blinded (drug injector and PO assessor) comparative study will be conducted in Mansoura university hospital after obtaining ethics committee approval and written informed parental consent 164 boys (age 1-6 years, ASA I) scheduled for hypospadias surgery. Exclusion criteria; (Operative time exceeding 3 hours, bodyweight \>25kg, bleeding diathesis, infection at the site of block, pre-existing neurological or spinal disease or abnormalities of the sacrum, inability to palpate the sacral hiatus by anatomic landmark palpation technique) or those with a history of allergic reactions to local anesthetics were excluded from the study. Patients will fast for solids 6h and water and 2 hours before surgery. After Patient Preoperative preparation, General anesthesia induction, caudal block and caudal drug injection (Levob plus DEXM) bolus will be injected according to each group as follow; * Group A will take Caudal Levob 0. 125%+ DEXM 0.5µg/kg. * Group B will take Caudal Levob 0.125%+ DEXM 1µg/kg. * Group C will take Caudal Levob 0. 125%+ DEXM 1.5 µg/kg. * Group D will take Caudal Levob 0. 125%+ DEXM 2µg/kg. The study outcomes recording: 1ry outcome: Time to (1st analgesic request OPS \>4). Secondary outcome: Total 24hours PO. Rescue analgesic dose IM pethidine 0.5mg/kg \[2,3\] based on local policy and protocol, Patients demographic data \[age, weight, end tidal Sevoflurane (volume %) prior starting skin closure, intraoperative fentanyl total dose, recovery time (time from discontinuation of anesthesia to spontaneous eye opening)\], Serum Cortisol level at basal before anesthesia and 1hour postoperative in between groups \[1\] , Apnea incidence (NO\&%), and desaturation (NO\&%), Objective pain score, Sedation score, Behavioral score for Agitation assessment, and Modified bromage score (residual Lower limb muscle weakness) recorded every 15 minutes for 1st hour then every 30 minutes for next 3 PO hours then at next 6th, 12th, 18th, 24th PO hours, hemodynamics, HR\&SBP the incidence of bradycardia and hypotension per 50% percentile. Recorded every 10 minutes IO for maximum operative time 3 hours and every 30 min PO for next 2h in the recovery room until the child was discharged to the ward. ### Conditions Module **Conditions:** - 164 Boys for Hypospadias Surgery Under General Anesthesia With Caudal Block **Keywords:** - Caudal - Dexmedetomidine - Levobupivacaine - hypospadias ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** TRIPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - OUTCOMES_ASSESSOR **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Count:** 164 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Drug: Caudal dexamedatomidine analgesia **Label:** • Group A will take Caudal Levob 0. 125%+ DEXM 0.5µg/k **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Intervention Names:** - Drug: Caudal dexamedatomidine analgesia **Label:** • Group B will take Caudal Levob 0.125%+ DEXM 1µg/kg. **Type:** ACTIVE_COMPARATOR #### Arm Group 3 **Intervention Names:** - Drug: Caudal dexamedatomidine analgesia **Label:** • Group C will take Caudal Levob 0. 125%+ DEXM 1.5 µg/ **Type:** ACTIVE_COMPARATOR #### Arm Group 4 **Intervention Names:** - Drug: Caudal dexamedatomidine analgesia **Label:** • Group D will take Caudal Levob 0. 125%+ DEXM 2µg/kg. **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - • Group A will take Caudal Levob 0. 125%+ DEXM 0.5µg/k - • Group B will take Caudal Levob 0.125%+ DEXM 1µg/kg. - • Group C will take Caudal Levob 0. 125%+ DEXM 1.5 µg/ - • Group D will take Caudal Levob 0. 125%+ DEXM 2µg/kg. **Description:** Technique performance (Caudal block): classical technique; palpation, identification and puncture with the patients in the lateral position (permits easy access to the airway under general anesthesia) using a 22-G short-beveled needle under sterile conditions advancing needle pierces the sacrococcygeal ligament. After needle insertion, pre-calculated volume of Levob plus DEXM bolus was injected as follow; Group A Caudal Levob 0.125%+ DEXM 0.5µg/kg. Group B Caudal Levob 0.125%+ DEXM 1µg/kg. Group C Caudal Levob 0.125%+ DEXM 1. 5 µg/kg. Group D Caudal Levob 0.125%+ DEXM 2µg/kg. **Name:** Caudal dexamedatomidine analgesia **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** time from full recovery till child express moderate pain OPS 4 and ask for the first analgesic dose **Measure:** Time to (1st analgesic request objective pain score (OPS) ≥4) **Time Frame:** Basal (0) till 24 hours. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * 164 boys (age 1-8 years, ASA I) scheduled for hypospadias surgery Exclusion Criteria: * Operative time exceeding 3 hours, bodyweight \>25kg, bleeding diathesis, infection at the site of block, pre-existing neurological or spinal disease or abnormalities of the sacrum, inability to palpate the sacral hiatus by anatomic landmark palpation technique) or those with a history of allergic reactions to local anesthetics were excluded from the study **Gender Based:** True **Maximum Age:** 8 Years **Minimum Age:** 1 Year **Sex:** MALE **Standard Ages:** - CHILD ### Contacts Locations Module #### Locations **Location 1:** **City:** Mansoura **Country:** Egypt **Facility:** Anesthesia department,Faculty of medicine, Mansoura univerisety #### Overall Officials **Official 1:** **Affiliation:** Mansoura Univeristy **Name:** mohamed A Ghanem, A professor **Role:** STUDY_CHAIR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014564 - Term: Urogenital Abnormalities - ID: D000052776 - Term: Female Urogenital Diseases - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications - ID: D000091642 - Term: Urogenital Diseases - ID: D000010409 - Term: Penile Diseases - ID: D000005832 - Term: Genital Diseases, Male - ID: D000091662 - Term: Genital Diseases - ID: D000052801 - Term: Male Urogenital Diseases - ID: D000000013 - Term: Congenital Abnormalities ### Condition Browse Module - Browse Branches - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M10071 - Name: Hypospadias - Relevance: HIGH - As Found: Hypospadias - ID: M12 - Name: Congenital Abnormalities - Relevance: LOW - As Found: Unknown - ID: M17314 - Name: Urogenital Abnormalities - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M13320 - Name: Penile Diseases - Relevance: LOW - As Found: Unknown - ID: M2876 - Name: Genital Diseases - Relevance: LOW - As Found: Unknown - ID: M8944 - Name: Genital Diseases, Male - Relevance: LOW - As Found: Unknown - ID: M27095 - Name: Male Urogenital Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000007021 - Term: Hypospadias ### Intervention Browse Module - Browse Branches - Abbrev: Analg - Name: Analgesics - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M22662 - Name: Dexmedetomidine - Relevance: LOW - As Found: Unknown - ID: M4107 - Name: Anesthetics - Relevance: LOW - As Found: Unknown - ID: M4032 - Name: Analgesics - Relevance: LOW - As Found: Unknown - ID: M1832 - Name: Levobupivacaine - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05531279 **Brief Title:** A Study of PEG-rhG-CSF and rhG-CSF Used for Aplastic Anemia Granulocyte Deficiency **Official Title:** A Randomized,Open-label Dose-discovery Study of PEG-rhG-CSF and rhG-CSF in the Adjuvant Therapy of Aplastic Anemia Granulocyte Deficiency #### Organization Study ID Info **ID:** IIT2021022-EC-1 #### Organization **Class:** OTHER **Full Name:** Institute of Hematology & Blood Diseases Hospital, China ### Status Module #### Completion Date **Date:** 2026-01-05 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2022-09-26 **Type:** ACTUAL **Last Update Submit Date:** 2022-09-22 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2025-06-05 **Type:** ESTIMATED #### Start Date **Date:** 2022-06-05 **Type:** ACTUAL **Status Verified Date:** 2022-09 #### Study First Post Date **Date:** 2022-09-07 **Type:** ACTUAL **Study First Submit Date:** 2022-09-02 **Study First Submit QC Date:** 2022-09-02 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Institute of Hematology & Blood Diseases Hospital, China #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** This study was a single-center,open-label,randomized,dose-exploring prospective study.Patients with granulocytotic aplastic anemia who received cytokine treatment with PEG-rhG-CSF or rhG-CSF were enrolled.Clinical demographic data,disease characteristics of aplastic anemia,clinical diagnosis and treatment,laboratory data and adverse events were collected to explore the dose and safety of PEG-rhG-CSF and rhG-CSF in patients with severe aplastic anemia. **Detailed Description:** This study was a single-center,open-label,randomized,dose-exploring prospective study.Patients with granulocytotic aplastic anemia who received cytokine treatment with PEG-rhG-CSF or rhG-CSF were enrolled.Clinical demographic data,disease characteristics of aplastic anemia,clinical diagnosis and treatment,laboratory data and adverse events were collected to explore the dose and safety of PEG-rhG-CSF and rhG-CSF in patients with severe aplastic anemia.Research objectives: To explore the reasonable injection frequency of long-acting PEG-rhG-CSF in the adjuvant treatment of aplastic anemia patients with granulocytosis through a single center prospective clinical study.Disease classification of aplastic anemia: a total of 45 cases of SAA/VSAA with ANC\<0.5×109/L were stratified and randomized into three groups according to the radio of 1:1:1(15 cases in each group).PEG-rhG-CSF group A(q7d):6mg d1,8,subcutaneously injected;PEG-rhG-CSF group B(q10d):6mg d1,11,subcutaneously injected;RhG-CSF group(short-acting ): 480ug d1-14(daily for 14days),subcutaneously injected.Dose/protocol adjustment: after monitoring ANC\>0.5×109/L,the drug was stopped,and then ANC\<0.5×109/L was temporarily supplemented with one dose of the original group of drugs. ### Conditions Module **Conditions:** - Severe Aplastic Anemia **Keywords:** - G-CSF ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 45 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** PEG-rhG-CSF group A(q7d):6mg d1,8,subcutaneously injected,after monitoring ANC\>0.5×109/L,the drug was stopped,and then ANC\<0.5×109/L was temporarily supplemented with one dose of the same drug. **Intervention Names:** - Drug: PEG-rhG-CSF **Label:** PEG-rhG-CSF group A **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** PEG-rhG-CSF group B(q10d):6mg d1,11,subcutaneously injected,after monitoring ANC\>0.5×109/L,the drug was stopped,and then ANC\<0.5×109/L was temporarily supplemented with one dose of the same drug. **Intervention Names:** - Drug: PEG-rhG-CSF **Label:** PEG-rhG-CSF group B **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** rhG-CSF group(short-acting ): 480ug d1-14(daily for 14days),subcutaneously injected.After monitoring ANC\>0.5×109/L,the drug was stopped,and then ANC\<0.5×109/L was temporarily supplemented with one dose of the same drug. **Intervention Names:** - Drug: PEG-rhG-CSF **Label:** rhG-CSF group(short-acting ) **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - PEG-rhG-CSF group A - PEG-rhG-CSF group B - rhG-CSF group(short-acting ) **Description:** PEG-rhG-CSF group A(q7d):6mg d1,8,subcutaneously injected; PEG-rhG-CSF group B(q10d):6mg d1,11,subcutaneously injected; RhG-CSF group(short-acting ): 480ug d1-14(daily for 14days),subcutaneously injected. **Name:** PEG-rhG-CSF **Other Names:** - rhG-CSF **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** total number of effective (ANC\>0.5×109/L) days during 2 weeks of treatment(cases×days). **Measure:** total number of effective (ANC>0.5×109/L) days **Time Frame:** 2 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Age 18-70 years old, male or female, or weight≥50kg. 2. Patients with severe or very severe aplastic anemia of absolute neutrophil value\< 0.5×109/L 3. ECOG score ≤ 2 points. 4. Normal renal function. Exclusion Criteria: 1. Patients with clonal chromosomal abnormalities. 2. Patients with previous malignant tumors. 3. Patients with severe or uncontrolled infectious diseases and /or bleeding. 4. Patients with AIDS or syphilis positive. 5. Severe organ dysfunction. 6. Patients used GM/G-CSF,PEG-rhG-CSF,interleukin-11 within 2 weeks before admission. 7. Allergic to G-CSF or PEG-rhG-CSF related components. 8. Participated in other clinical trials within 6 months. **Maximum Age:** 70 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Fengkui Zhang, Doctor **Phone:** +862223909229 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Huihui Fan, Doctor **Phone:** +862223909223 **Role:** CONTACT #### Locations **Location 1:** **City:** Tianjin **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Fengkui Zhang, Doctor - **Phone:** 8602223909229 - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Huihui Fan, Doctor - **Phone:** 8602223909223 - **Role:** CONTACT **Country:** China **Facility:** Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences **State:** Tianjin **Status:** RECRUITING **Zip:** 300020 #### Overall Officials **Official 1:** **Affiliation:** Institute of Hematology ＆ Blood Diseases Hospital，Chinese Academy of Medical Sciences **Name:** Liping Jing, Doctor **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **Access Criteria:** After the paper is written and published **Description:** We can share the plan after completing the experiment **Info Types:** - STUDY_PROTOCOL - ICF - CSR **IPD Sharing:** YES **Time Frame:** Follow-up and publication of the paper are planned for December 2025 ### References Module #### References **Citation:** Tichelli A, Schrezenmeier H, Socie G, Marsh J, Bacigalupo A, Duhrsen U, Franzke A, Hallek M, Thiel E, Wilhelm M, Hochsmann B, Barrois A, Champion K, Passweg JR. A randomized controlled study in patients with newly diagnosed severe aplastic anemia receiving antithymocyte globulin (ATG), cyclosporine, with or without G-CSF: a study of the SAA Working Party of the European Group for Blood and Marrow Transplantation. Blood. 2011 Apr 28;117(17):4434-41. doi: 10.1182/blood-2010-08-304071. Epub 2011 Jan 13. **PMID:** 21233311 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000006402 - Term: Hematologic Diseases - ID: D000080983 - Term: Bone Marrow Failure Disorders - ID: D000001855 - Term: Bone Marrow Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC15 - Name: Blood and Lymph Conditions - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M4071 - Name: Anemia, Aplastic - Relevance: HIGH - As Found: Aplastic Anemia - ID: M4070 - Name: Anemia - Relevance: HIGH - As Found: Anemia - ID: M9490 - Name: Hematologic Diseases - Relevance: LOW - As Found: Unknown - ID: M2241 - Name: Bone Marrow Failure Disorders - Relevance: LOW - As Found: Unknown - ID: M13118 - Name: Pancytopenia - Relevance: LOW - As Found: Unknown - ID: M5134 - Name: Bone Marrow Diseases - Relevance: LOW - As Found: Unknown - ID: T460 - Name: Aplastic Anemia - Relevance: HIGH - As Found: Aplastic Anemia ### Condition Browse Module - Meshes - ID: D000000740 - Term: Anemia - ID: D000000741 - Term: Anemia, Aplastic ### Intervention Browse Module - Browse Branches - Abbrev: Micro - Name: Micronutrients - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Ot - Name: Other Dietary Supplements ### Intervention Browse Module - Browse Leaves - ID: M271049 - Name: Coenzyme Q10 - Relevance: LOW - As Found: Unknown - ID: M17201 - Name: Ubiquinone - Relevance: LOW - As Found: Unknown - ID: T383 - Name: Coenzyme Q10 - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00684879 **Brief Title:** Screening Behavior in Adults With Hereditary Hemorrhagic Telangiectasia **Official Title:** Illness Perceptions and the Health Belief Model: Screening Behavior in Adults With Hereditary Hemorrhagic Telangiectasia #### Organization Study ID Info **ID:** 999908143 #### Organization **Class:** NIH **Full Name:** National Institutes of Health Clinical Center (CC) #### Secondary ID Infos **ID:** 08-HG-N143 ### Status Module #### Completion Date **Date:** 2016-01-07 #### Expanded Access Info #### Last Update Post Date **Date:** 2019-11-25 **Type:** ACTUAL **Last Update Submit Date:** 2019-11-22 **Overall Status:** COMPLETED #### Start Date **Date:** 2008-05-21 **Status Verified Date:** 2016-01-07 #### Study First Post Date **Date:** 2008-05-28 **Type:** ESTIMATED **Study First Submit Date:** 2008-05-24 **Study First Submit QC Date:** 2008-05-24 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** NIH **Name:** National Human Genome Research Institute (NHGRI) #### Responsible Party **Type:** SPONSOR ### Description Module **Brief Summary:** This study will explore the factors that influence screening behaviors of adults diagnosed with hereditary hemorrhagic telangiectasia (HHT), an inherited condition in which blood vessel defects called arteriovenous malformations (AVMs) result in direct connections between arteries and veins. Patients most commonly have small AVMs called telangiectases on the tongue, face, hands, mouth, and throat and the mucosal linings of the nose and gastrointestinal tract. Recurrent nosebleeds are a hallmark of the disease. Large AVMs can also occur in various organs, causing sudden and life-threatening complications. The study will examine how patients think and feel about their condition and what actions they take to screen for internal symptoms of the disease. Men and women 18 years of age and older who have HHT may be eligible for this study. Participants fill out a 30-minute questionnaire, available in print or online, that includes questions about the participant s * beliefs about HHT * actions taken to screen for internal symptoms of HHT * experience with HHT * current health status, family history and demographic information **Detailed Description:** The proposed study aims to understand the factors that influence screening behaviors of adults with hereditary hemorrhagic telangiectasia (HHT). HHT is a chronic condition, but with early diagnosis followed by adherence to recommended screening guidelines, the major complications of this disorder can be avoided and disability or even death can be prevented. Yet, it has come to the attention of healthcare professionals that the recommended screening is not commonly followed by individuals with HHT, even when the risk of serious complications is known. Nonadherence to screening recommendations is not unique to HHT. It is rather common across chronic conditions, and genetic diseases, such as HHT, are no exception. However, HHT may have an added barrier to screening and treatment adherence in that it is a rare and underdiagnosed condition. Inadequate knowledge of healthcare providers may be a serious barrier to prevention, diagnosis, and treatment. The Health Belief Model (HBM) can be used to frame this study of HHT screening. The HBM posits that preventive health behaviors will be acted upon if individuals regard themselves as susceptible to the threat, they believe the consequences to be severe, and the perceived benefits outweigh the perceived barriers. In addition to the HBM constructs, this study will also consider the role of illness representations which provide a more personal view of the lived experience of individuals with HHT. A cosss-sectional design will be used to investigate the relationships among the domains of illness representations, HBM constructs (perceived susceptibility, perceived benefits, barriers, self-efficacy, response efficacy, cues to action), and HHT-specific screening guidelines. Participants will be recruited from the HHT Foundation International, Inc., an outline HHT Awareness social group, and HHT Clinics. Participants will be asked to complete either a web-based or a paper survey. The main outcome measure is screening behaviors and intentions to screen for complications associated with HHT. ### Conditions Module **Conditions:** - Osler-Rendu-Weber Disease - Osler-Rendu Disease - Telangiectasia, Hereditary Hemorrhagic **Keywords:** - Hereditary Hemorrhagic Telangiectasia - Health Belief Model - Illness Perceptions - Screening - HHT ### Design Module #### Design Info **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 320 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Eligibility Module **Eligibility Criteria:** * INCLUSION CRITERIA: Men and women who self-report having a diagnosis of HHT. To read and write English. EXCLUSION CRITERIA: Individuals younger than 18. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Bethesda **Country:** United States **Facility:** National Human Genome Research Institute (NHGRI), 9000 Rockville Pike **State:** Maryland **Zip:** 20892 #### Overall Officials **Official 1:** **Affiliation:** National Human Genome Research Institute (NHGRI) **Name:** Barbara B Biesecker **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Bayrak-Toydemir P, Mao R, Lewin S, McDonald J. Hereditary hemorrhagic telangiectasia: an overview of diagnosis and management in the molecular era for clinicians. Genet Med. 2004 Jul-Aug;6(4):175-91. doi: 10.1097/01.gim.0000132689.25644.7c. **PMID:** 15266205 **Citation:** Guttmacher AE, Marchuk DA, White RI Jr. Hereditary hemorrhagic telangiectasia. N Engl J Med. 1995 Oct 5;333(14):918-24. doi: 10.1056/NEJM199510053331407. No abstract available. **PMID:** 7666879 **Citation:** Rand CS. Measuring adherence with therapy for chronic diseases: implications for the treatment of heterozygous familial hypercholesterolemia. Am J Cardiol. 1993 Sep 30;72(10):68D-74D. doi: 10.1016/0002-9149(93)90014-4. **PMID:** 8213501 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014652 - Term: Vascular Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000020141 - Term: Hemostatic Disorders - ID: D000006474 - Term: Hemorrhagic Disorders - ID: D000006402 - Term: Hematologic Diseases - ID: D000054079 - Term: Vascular Malformations - ID: D000018376 - Term: Cardiovascular Abnormalities - ID: D000000013 - Term: Congenital Abnormalities ### Condition Browse Module - Browse Branches - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC15 - Name: Blood and Lymph Conditions - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M16456 - Name: Telangiectasis - Relevance: HIGH - As Found: Telangiectasia - ID: M16455 - Name: Telangiectasia, Hereditary Hemorrhagic - Relevance: HIGH - As Found: Telangiectasia, Hereditary Hemorrhagic - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown - ID: M21977 - Name: Hemostatic Disorders - Relevance: LOW - As Found: Unknown - ID: M5059 - Name: Blood Coagulation Disorders - Relevance: LOW - As Found: Unknown - ID: M9560 - Name: Hemorrhagic Disorders - Relevance: LOW - As Found: Unknown - ID: M9490 - Name: Hematologic Diseases - Relevance: LOW - As Found: Unknown - ID: M12 - Name: Congenital Abnormalities - Relevance: LOW - As Found: Unknown - ID: M27558 - Name: Vascular Malformations - Relevance: LOW - As Found: Unknown - ID: M20503 - Name: Cardiovascular Abnormalities - Relevance: LOW - As Found: Unknown - ID: T2750 - Name: Hereditary Hemorrhagic Telangiectasia - Relevance: HIGH - As Found: Hereditary Hemorrhagic Telangiectasia ### Condition Browse Module - Meshes - ID: D000013684 - Term: Telangiectasis - ID: D000013683 - Term: Telangiectasia, Hereditary Hemorrhagic ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02385279 **Acronym:** DESSOLVE III **Brief Title:** Study Comparing the MiStent SES Versus the XIENCE EES Stent **Official Title:** Multicenter Randomized Study of the MiStent Sirolimus Eluting Absorbable Polymer Stent System (MiStent SES) for Revascularization of Coronary Arteries #### Organization Study ID Info **ID:** ECRI-005 #### Organization **Class:** INDUSTRY **Full Name:** ECRI bv ### Status Module #### Completion Date **Date:** 2021-02-04 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-05-08 **Type:** ACTUAL **Last Update Submit Date:** 2022-07-12 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2017-01-31 **Type:** ACTUAL #### Results First Post Date **Date:** 2023-05-08 **Type:** ACTUAL **Results First Submit Date:** 2021-12-08 **Results First Submit QC Date:** 2022-07-12 #### Start Date **Date:** 2015-03-20 **Type:** ACTUAL **Status Verified Date:** 2022-07 #### Study First Post Date **Date:** 2015-03-11 **Type:** ESTIMATED **Study First Submit Date:** 2015-02-17 **Study First Submit QC Date:** 2015-03-05 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Micell Technologies **Class:** INDUSTRY **Name:** Stentys #### Lead Sponsor **Class:** INDUSTRY **Name:** ECRI bv #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The primary objective of this study is to compare the performance of MISTENT to that of XIENCE in an all-comers patient population with symptomatic ischemic heart disease. The patients will be followed through 3 years for major clinical events. ### Conditions Module **Conditions:** - Coronary Stenosis **Keywords:** - CAD - ACS - All comers - PCI ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - PARTICIPANT **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 1398 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Percutaneous Coronary Intervention with the MiStent Sirolimus Eluting Absorbable Polymer Coronary Stent. It is a balloon expandable sirolimus eluting stent with an absorbable polymer coating. **Intervention Names:** - Device: MiStent **Label:** MiStent® **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Percutaneous Coronary Intervention with the XIENCE EES (Everolimus Eluting) Coronary Stent System. The stents are balloon expandable drug eluting stents using everolimus with a non-erodible or durable polymer coating. **Intervention Names:** - Device: XIENCE EES **Label:** XIENCE EES **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - MiStent® **Description:** Percutaneous Coronary Intervention **Name:** MiStent **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - XIENCE EES **Description:** Percutaneous Coronary Intervention **Name:** XIENCE EES **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** DOCE is a composite of clinical endpoint of cardiac death, myocardial infarction not clearly attributable to a non-target vessel and clinically-indicated target lesion revascularization. **Measure:** Number of Participants With Occurrence of a Device Oriented Composite Endpoint (DOCE) **Time Frame:** 12 months postprocedure #### Secondary Outcomes **Description:** POCE defined as all-cause death, any Myocardial Infarction (MI), or any revascularization **Measure:** POCE **Time Frame:** At 12 months **Description:** MACE defined as all-cause death, any MI, or any Target Vessel Revascularization (TVR) **Measure:** MACE **Time Frame:** At 12 months **Description:** Target Vessel Failure (TVF) defined as cardiac death, TV MI, or clinically indicated TVR **Measure:** Target Vessel Failure (TVF) **Time Frame:** At 12 months **Description:** All-cause death **Measure:** All-cause Death **Time Frame:** At 12 months **Description:** Any Myocardial infarction **Measure:** Myocardial Infarction **Time Frame:** At 12 months **Description:** Any revascularization **Measure:** Any Revascularization **Time Frame:** At 12 months **Description:** Definite or probably stent thrombosis according to ARC **Measure:** Stent Thrombosis **Time Frame:** At 12 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: All comers" patients: * Male or female patients 18 years or older; * Presence of one or more coronary artery stenoses of 50% or more in a native coronary artery or in a saphenous venous or arterial bypass conduit suitable for coronary stent implantation. * The vessel should have a reference vessel diameter ranging from 2.5 mm to 3.75 mm (no limitation on the number of treated lesions, vessels, or lesion length); All lesions of the patient must comply with the angiographic inclusion criteria. * The patient is judged to be capable of providing voluntary informed consent and has been fully informed of the nature of the study, is willing to comply with all study requirements and will provide written informed consent as approved by the Ethics Committee of the respective clinical site. Exclusion Criteria: * Known pregnancy or breastfeeding at time of randomization; * Known contraindication or hypersensitivity to sirolimus, everolimus, cobalt-chromium, or to medications such as aspirin, heparin, bivalirudin, and all of the following four medications: clopidogrel bisulfate, ticlopidine, prasugrel, ticagrelor; * Concurrent medical condition with a life expectancy of less than 12 months. * The patient is unwilling/ not able to return for outpatient clinic at 1 month and 12 months follow-up. * Currently participating in another trial and not yet at its primary endpoint. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Corbeil **Country:** France **Facility:** Research Center Corbeil **Location 2:** **City:** Nimes **Country:** France **Facility:** Research Center Nimes **Location 3:** **City:** Poitiers **Country:** France **Facility:** Research Center Poitiers **Location 4:** **City:** Jena **Country:** Germany **Facility:** Research Center Jena **Location 5:** **City:** Leipzig **Country:** Germany **Facility:** Research Center Leipzig **Location 6:** **City:** Munster **Country:** Germany **Facility:** Research Center Munster **Location 7:** **City:** Ulm **Country:** Germany **Facility:** Research Center Ulm **Location 8:** **City:** Wiesbaden **Country:** Germany **Facility:** Research Center Wiesbaden **Location 9:** **City:** Amersfoort **Country:** Netherlands **Facility:** Research Center Amersfoort **Location 10:** **City:** Amsterdam **Country:** Netherlands **Facility:** Research Center Amsterdam **Location 11:** **City:** Blaricum **Country:** Netherlands **Facility:** Tergooi **Location 12:** **City:** Emmen **Country:** Netherlands **Facility:** Research Center Emmen **Location 13:** **City:** Leeuwarden **Country:** Netherlands **Facility:** Research Center Leeuwarden **Location 14:** **City:** Nijmegen **Country:** Netherlands **Facility:** Research Center Nijmegen **Location 15:** **City:** Venlo **Country:** Netherlands **Facility:** Research Center Venlo **Location 16:** **City:** Belchatow **Country:** Poland **Facility:** Research Center Belchatow **Location 17:** **City:** Bielsko-Biala **Country:** Poland **Facility:** Research Center Bielsko-Biala **Location 18:** **City:** Chrzanow **Country:** Poland **Facility:** Research center Chrzanow **Location 19:** **City:** Tychy **Country:** Poland **Facility:** Research Center Tychy **Location 20:** **City:** Zgierz **Country:** Poland **Facility:** Research Center Zgierz #### Overall Officials **Official 1:** **Affiliation:** European Cardiovascular Research Institute **Name:** Ernest Spitzer, MD **Role:** STUDY_DIRECTOR ### References Module #### References **Citation:** de Winter RJ, Katagiri Y, Asano T, Milewski KP, Lurz P, Buszman P, Jessurun GAJ, Koch KT, Troquay RPT, Hamer BJB, Ophuis TO, Wohrle J, Wyderka R, Cayla G, Hofma SH, Levesque S, Zurakowski A, Fischer D, Kosmider M, Goube P, Arkenbout EK, Noutsias M, Ferrari MW, Onuma Y, Wijns W, Serruys PW. A sirolimus-eluting bioabsorbable polymer-coated stent (MiStent) versus an everolimus-eluting durable polymer stent (Xience) after percutaneous coronary intervention (DESSOLVE III): a randomised, single-blind, multicentre, non-inferiority, phase 3 trial. Lancet. 2018 Feb 3;391(10119):431-440. doi: 10.1016/S0140-6736(17)33103-3. Epub 2017 Dec 5. **PMID:** 29203070 **Citation:** Wang R, Kawashima H, Hara H, Gao C, Ono M, Takahashi K, Tu S, Soliman O, Garg S, van Geuns RJ, Tao L, Wijns W, Onuma Y, Serruys PW. Comparison of Clinically Adjudicated Versus Flow-Based Adjudication of Revascularization Events in Randomized Controlled Trials. Circ Cardiovasc Qual Outcomes. 2021 Nov;14(11):e008055. doi: 10.1161/CIRCOUTCOMES.121.008055. Epub 2021 Oct 20. **PMID:** 34666500 **Citation:** Katagiri Y, Andreini D, Miyazaki Y, Takahashi K, Komiyama H, Mushtaq S, Sonck J, Schoors D, Maisano F, Kaufman PA, Leal I, Lindeboom W, Piek JJ, Wykrzykowska JJ, Morel MA, Bartorelli AL, Onuma Y, Serruys PW; SYNTAX III REVOLUTION Investigators. Site vs. core laboratory variability in computed tomographic angiography-derived SYNTAX scores in the SYNTAX III trial. Eur Heart J Cardiovasc Imaging. 2021 Aug 14;22(9):1063-1071. doi: 10.1093/ehjci/jeaa172. **PMID:** 32888011 **Citation:** Takahashi K, Serruys PW, Kogame N, Buszman P, Lurz P, Jessurun GAJ, Koch KT, Troquay RPT, Hamer BJB, Oude Ophuis T, Milewski KP, Hofma SH, Wykrzykowska JJ, Onuma Y, de Winter RJ, Wijns W. Final 3-Year Outcomes of MiStent Biodegradable Polymer Crystalline Sirolimus-Eluting Stent Versus Xience Permanent Polymer Everolimus-Eluting Stent: Insights From the DESSOLVE III All-Comers Randomized Trial. Circ Cardiovasc Interv. 2020 Jun;13(6):e008737. doi: 10.1161/CIRCINTERVENTIONS.119.008737. Epub 2020 May 29. **PMID:** 32466676 ## Document Section ### Large Document Module #### Large Docs - Date: 2018-03-07 - Filename: Prot_SAP_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: True - Label: Study Protocol and Statistical Analysis Plan - Size: 568483 - Type Abbrev: Prot_SAP - Upload Date: 2021-12-08T05:05 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000003327 - Term: Coronary Disease - ID: D000017202 - Term: Myocardial Ischemia - ID: D000006331 - Term: Heart Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000014652 - Term: Vascular Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC14 - Name: Heart and Blood Diseases ### Condition Browse Module - Browse Leaves - ID: M6475 - Name: Constriction, Pathologic - Relevance: LOW - As Found: Unknown - ID: M22913 - Name: Coronary Stenosis - Relevance: HIGH - As Found: Coronary Stenosis - ID: M6549 - Name: Coronary Disease - Relevance: LOW - As Found: Unknown - ID: M6546 - Name: Coronary Artery Disease - Relevance: LOW - As Found: Unknown - ID: M10543 - Name: Ischemia - Relevance: LOW - As Found: Unknown - ID: M19506 - Name: Myocardial Ischemia - Relevance: LOW - As Found: Unknown - ID: M9419 - Name: Heart Diseases - Relevance: LOW - As Found: Unknown - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000023921 - Term: Coronary Stenosis ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Infe - Name: Anti-Infective Agents ### Intervention Browse Module - Browse Leaves - ID: M255 - Name: Everolimus - Relevance: LOW - As Found: Unknown - ID: M21960 - Name: Sirolimus - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** Adverse event reporting in the context of a post-marketing trial. #### Event Groups **Group ID:** EG000 **Title:** MiStent® **Deaths Num Affected:** 85 **Deaths Num At Risk:** 703 **Description:** Percutaneous Coronary Intervention with the MiStent Sirolimus Eluting Absorbable Polymer Coronary Stent. It is a balloon expandable sirolimus eluting stent with an absorbable polymer coating. MiStent: Percutaneous Coronary Intervention **ID:** EG000 **Other Num Affected:** 76 **Other Num at Risk:** 703 **Serious Number Affected:** 434 **Serious Number At Risk:** 703 **Title:** MiStent® **Group ID:** EG001 **Title:** XIENCE EES **Deaths Num Affected:** 78 **Deaths Num At Risk:** 695 **Description:** Percutaneous Coronary Intervention with the XIENCE EES (Everolimus Eluting) Coronary Stent System. The stents are balloon expandable drug eluting stents using everolimus with a non-erodible or durable polymer coating. XIENCE EES: Percutaneous Coronary Intervention **ID:** EG001 **Other Num Affected:** 71 **Other Num at Risk:** 695 **Serious Number Affected:** 426 **Serious Number At Risk:** 695 **Title:** XIENCE EES **Frequency Threshold:** 0 #### Other Events **Term:** Non Serious AE **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MEDDEV 2.7/3 #### Serious Events **Term:** Myocardial infarction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MEDDEV 2.7/3 ##### Stats **Group ID:** EG000 **Num Affected:** 17 **Num At Risk:** 703 **Group ID:** EG001 **Num Affected:** 15 **Num At Risk:** 695 **Term:** Definite stent thrombosis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MEDDEV 2.7/3 ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 703 **Group ID:** EG001 **Num Affected:** 5 **Num At Risk:** 695 **Term:** Other **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MEDDEV 2.7/3 ##### Stats **Group ID:** EG000 **Num Affected:** 414 **Num At Risk:** 703 **Group ID:** EG001 **Num Affected:** 406 **Num At Risk:** 695 **Time Frame:** 12 months (primary reporting), and yearly up to 5 years. ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 703 **Group ID:** BG001 **Value:** 695 **Group ID:** BG002 **Value:** 1398 **Units:** Participants ### Group **ID:** BG000 **Title:** MiStent® **Description:** Percutaneous Coronary Intervention with the MiStent Sirolimus Eluting Absorbable Polymer Coronary Stent. It is a balloon expandable sirolimus eluting stent with an absorbable polymer coating. MiStent: Percutaneous Coronary Intervention ### Group **ID:** BG001 **Title:** XIENCE EES **Description:** Percutaneous Coronary Intervention with the XIENCE EES (Everolimus Eluting) Coronary Stent System. The stents are balloon expandable drug eluting stents using everolimus with a non-erodible or durable polymer coating. XIENCE EES: Percutaneous Coronary Intervention ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 257 #### Measurement **Group ID:** BG001 **Value:** 271 #### Measurement **Group ID:** BG002 **Value:** 528 **Category Title:** Between 18 and 65 years #### Measurement **Group ID:** BG000 **Value:** 446 #### Measurement **Group ID:** BG001 **Value:** 424 #### Measurement **Group ID:** BG002 **Value:** 870 **Category Title:** >=65 years #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 703 **Group ID:** BG001 **Value:** 695 **Group ID:** BG002 **Value:** 1398 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 10.7 **Value:** 66.4 #### Measurement **Group ID:** BG001 **Spread:** 10.7 **Value:** 66.3 #### Measurement **Group ID:** BG002 **Spread:** 10.7 **Value:** 66.4 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 703 **Group ID:** BG001 **Value:** 695 **Group ID:** BG002 **Value:** 1398 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 209 #### Measurement **Group ID:** BG001 **Value:** 182 #### Measurement **Group ID:** BG002 **Value:** 391 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 494 #### Measurement **Group ID:** BG001 **Value:** 513 #### Measurement **Group ID:** BG002 **Value:** 1007 **Category Title:** Male #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 703 **Group ID:** BG001 **Value:** 695 **Group ID:** BG002 **Value:** 1398 **Class Title:** ### Measure #### Measurement **Group ID:** BG002 **Value:** 0 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 0 **Group ID:** BG001 **Value:** 0 **Group ID:** BG002 **Value:** 0 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 297 #### Measurement **Group ID:** BG001 **Value:** 296 #### Measurement **Group ID:** BG002 **Value:** 593 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 703 **Group ID:** BG001 **Value:** 695 **Group ID:** BG002 **Value:** 1398 **Class Title:** Netherlands #### Measurement **Group ID:** BG000 **Value:** 187 #### Measurement **Group ID:** BG001 **Value:** 184 #### Measurement **Group ID:** BG002 **Value:** 371 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 703 **Group ID:** BG001 **Value:** 695 **Group ID:** BG002 **Value:** 1398 **Class Title:** Poland #### Measurement **Group ID:** BG000 **Value:** 56 #### Measurement **Group ID:** BG001 **Value:** 56 #### Measurement **Group ID:** BG002 **Value:** 112 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 703 **Group ID:** BG001 **Value:** 695 **Group ID:** BG002 **Value:** 1398 **Class Title:** France #### Measurement **Group ID:** BG000 **Value:** 163 #### Measurement **Group ID:** BG001 **Value:** 159 #### Measurement **Group ID:** BG002 **Value:** 322 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 703 **Group ID:** BG001 **Value:** 695 **Group ID:** BG002 **Value:** 1398 **Class Title:** Germany **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Age, Categorical **Unit of Measure:** Participants ### Measure 2 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Race and Ethnicity were not collected from any participant. **Title:** Race and Ethnicity Not Collected **Unit of Measure:** Participants ### Measure 5 **Parameter Type:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** participants ## Results Section - More Information Module ### Certain Agreement **Restrictive Agreement:** True ### Point of Contact **Email:** [email protected] **Organization:** European Cardiovascular Research Institute **Phone:** +31102062828 **Title:** Dr. E. Spitzer ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### Outcome Measure 4 ### Outcome Measure 5 ### Outcome Measure 6 ### Outcome Measure 7 ### Outcome Measure 8 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 40 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 45 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 93 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 101 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 65 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 65 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 45 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 53 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 25 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 18 **Title:** #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 17 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 15 **Title:** #### Outcome Measure 7 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 61 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 78 **Title:** #### Outcome Measure 8 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 6 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** DOCE is a composite of clinical endpoint of cardiac death, myocardial infarction not clearly attributable to a non-target vessel and clinically-indicated target lesion revascularization. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Intention-to-treat **Reporting Status:** POSTED **Time Frame:** 12 months postprocedure **Title:** Number of Participants With Occurrence of a Device Oriented Composite Endpoint (DOCE) **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Percutaneous Coronary Intervention with the MiStent Sirolimus Eluting Absorbable Polymer Coronary Stent. It is a balloon expandable sirolimus eluting stent with an absorbable polymer coating. MiStent: Percutaneous Coronary Intervention **ID:** OG000 **Title:** MiStent® ##### Group **Description:** Percutaneous Coronary Intervention with the XIENCE EES (Everolimus Eluting) Coronary Stent System. The stents are balloon expandable drug eluting stents using everolimus with a non-erodible or durable polymer coating. XIENCE EES: Percutaneous Coronary Intervention **ID:** OG001 **Title:** XIENCE EES #### Outcome Measure 2 **Description:** POCE defined as all-cause death, any Myocardial Infarction (MI), or any revascularization **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** ITT **Reporting Status:** POSTED **Time Frame:** At 12 months **Title:** POCE **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Percutaneous Coronary Intervention with the MiStent Sirolimus Eluting Absorbable Polymer Coronary Stent. It is a balloon expandable sirolimus eluting stent with an absorbable polymer coating. MiStent: Percutaneous Coronary Intervention **ID:** OG000 **Title:** MiStent® ##### Group **Description:** Percutaneous Coronary Intervention with the XIENCE EES (Everolimus Eluting) Coronary Stent System. The stents are balloon expandable drug eluting stents using everolimus with a non-erodible or durable polymer coating. XIENCE EES: Percutaneous Coronary Intervention **ID:** OG001 **Title:** XIENCE EES #### Outcome Measure 3 **Description:** MACE defined as all-cause death, any MI, or any Target Vessel Revascularization (TVR) **Parameter Type:** COUNT_OF_PARTICIPANTS **Reporting Status:** POSTED **Time Frame:** At 12 months **Title:** MACE **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Percutaneous Coronary Intervention with the MiStent Sirolimus Eluting Absorbable Polymer Coronary Stent. It is a balloon expandable sirolimus eluting stent with an absorbable polymer coating. MiStent: Percutaneous Coronary Intervention **ID:** OG000 **Title:** MiStent® ##### Group **Description:** Percutaneous Coronary Intervention with the XIENCE EES (Everolimus Eluting) Coronary Stent System. The stents are balloon expandable drug eluting stents using everolimus with a non-erodible or durable polymer coating. XIENCE EES: Percutaneous Coronary Intervention **ID:** OG001 **Title:** XIENCE EES #### Outcome Measure 4 **Description:** Target Vessel Failure (TVF) defined as cardiac death, TV MI, or clinically indicated TVR **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** ITT **Reporting Status:** POSTED **Time Frame:** At 12 months **Title:** Target Vessel Failure (TVF) **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Percutaneous Coronary Intervention with the MiStent Sirolimus Eluting Absorbable Polymer Coronary Stent. It is a balloon expandable sirolimus eluting stent with an absorbable polymer coating. MiStent: Percutaneous Coronary Intervention **ID:** OG000 **Title:** MiStent® ##### Group **Description:** Percutaneous Coronary Intervention with the XIENCE EES (Everolimus Eluting) Coronary Stent System. The stents are balloon expandable drug eluting stents using everolimus with a non-erodible or durable polymer coating. XIENCE EES: Percutaneous Coronary Intervention **ID:** OG001 **Title:** XIENCE EES #### Outcome Measure 5 **Description:** All-cause death **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** ITT **Reporting Status:** POSTED **Time Frame:** At 12 months **Title:** All-cause Death **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Percutaneous Coronary Intervention with the MiStent Sirolimus Eluting Absorbable Polymer Coronary Stent. It is a balloon expandable sirolimus eluting stent with an absorbable polymer coating. MiStent: Percutaneous Coronary Intervention **ID:** OG000 **Title:** MiStent® ##### Group **Description:** Percutaneous Coronary Intervention with the XIENCE EES (Everolimus Eluting) Coronary Stent System. The stents are balloon expandable drug eluting stents using everolimus with a non-erodible or durable polymer coating. XIENCE EES: Percutaneous Coronary Intervention **ID:** OG001 **Title:** XIENCE EES #### Outcome Measure 6 **Description:** Any Myocardial infarction **Parameter Type:** COUNT_OF_PARTICIPANTS **Reporting Status:** POSTED **Time Frame:** At 12 months **Title:** Myocardial Infarction **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Percutaneous Coronary Intervention with the MiStent Sirolimus Eluting Absorbable Polymer Coronary Stent. It is a balloon expandable sirolimus eluting stent with an absorbable polymer coating. MiStent: Percutaneous Coronary Intervention **ID:** OG000 **Title:** MiStent® ##### Group **Description:** Percutaneous Coronary Intervention with the XIENCE EES (Everolimus Eluting) Coronary Stent System. The stents are balloon expandable drug eluting stents using everolimus with a non-erodible or durable polymer coating. XIENCE EES: Percutaneous Coronary Intervention **ID:** OG001 **Title:** XIENCE EES #### Outcome Measure 7 **Description:** Any revascularization **Parameter Type:** COUNT_OF_PARTICIPANTS **Reporting Status:** POSTED **Time Frame:** At 12 months **Title:** Any Revascularization **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Percutaneous Coronary Intervention with the MiStent Sirolimus Eluting Absorbable Polymer Coronary Stent. It is a balloon expandable sirolimus eluting stent with an absorbable polymer coating. MiStent: Percutaneous Coronary Intervention **ID:** OG000 **Title:** MiStent® ##### Group **Description:** Percutaneous Coronary Intervention with the XIENCE EES (Everolimus Eluting) Coronary Stent System. The stents are balloon expandable drug eluting stents using everolimus with a non-erodible or durable polymer coating. XIENCE EES: Percutaneous Coronary Intervention **ID:** OG001 **Title:** XIENCE EES #### Outcome Measure 8 **Description:** Definite or probably stent thrombosis according to ARC **Parameter Type:** COUNT_OF_PARTICIPANTS **Reporting Status:** POSTED **Time Frame:** At 12 months **Title:** Stent Thrombosis **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Percutaneous Coronary Intervention with the MiStent Sirolimus Eluting Absorbable Polymer Coronary Stent. It is a balloon expandable sirolimus eluting stent with an absorbable polymer coating. MiStent: Percutaneous Coronary Intervention **ID:** OG000 **Title:** MiStent® ##### Group **Description:** Percutaneous Coronary Intervention with the XIENCE EES (Everolimus Eluting) Coronary Stent System. The stents are balloon expandable drug eluting stents using everolimus with a non-erodible or durable polymer coating. XIENCE EES: Percutaneous Coronary Intervention **ID:** OG001 **Title:** XIENCE EES ### Participant Flow Module #### Group **Description:** Percutaneous Coronary Intervention with the MiStent Sirolimus Eluting Absorbable Polymer Coronary Stent. It is a balloon expandable sirolimus eluting stent with an absorbable polymer coating. MiStent: Percutaneous Coronary Intervention **ID:** FG000 **Title:** MiStent® #### Group **Description:** Percutaneous Coronary Intervention with the XIENCE EES (Everolimus Eluting) Coronary Stent System. The stents are balloon expandable drug eluting stents using everolimus with a non-erodible or durable polymer coating. XIENCE EES: Percutaneous Coronary Intervention **ID:** FG001 **Title:** XIENCE EES #### Period **Title:** Overall Study ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 703 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 695 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 676 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 677 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 27 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 18 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT04128579 **Acronym:** EQUALISE **Brief Title:** Study of EQ001 (Itolizumab) in Systemic Lupus Erythematosus With or Without Active Proliferative Nephritis **Official Title:** A Phase 1b Multiple Ascending-dose Study of EQ001 in Subjects With Systemic Lupus Erythematosus With or Without Active Proliferative Lupus Nephritis #### Organization Study ID Info **ID:** EQ001-19-002 #### Organization **Class:** INDUSTRY **Full Name:** Equillium ### Status Module #### Completion Date **Date:** 2024-01-18 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2024-02-28 **Type:** ACTUAL **Last Update Submit Date:** 2024-02-26 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2023-11-16 **Type:** ACTUAL #### Start Date **Date:** 2019-10-01 **Type:** ACTUAL **Status Verified Date:** 2024-02 #### Study First Post Date **Date:** 2019-10-16 **Type:** ACTUAL **Study First Submit Date:** 2019-09-30 **Study First Submit QC Date:** 2019-10-14 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Biocon Limited #### Lead Sponsor **Class:** INDUSTRY **Name:** Equillium #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Oversight Has DMC:** True ### Description Module **Brief Summary:** This is a multi-center study to evaluate the safety, tolerability, PK, PD, and clinical activity of itolizumab (EQ001) in subjects with Systemic Lupus Erythematosus with or without Active Proliferative Lupus Nephritis **Detailed Description:** The study will enroll approximately 55 subjects, with up to 5 dose escalating cohorts of 6 open-label subjects enrolled for Type A-SLE and a single dose cohort of approximately 20 open-label subjects enrolled for Type B-Lupus Nephritis. Subjects will receive itolizumab administered subcutaneously every two weeks for a total of either 2 (Type A) or 13 (Type B) doses with 4 or 12 weeks of follow-up after the last dose of investigational product. ### Conditions Module **Conditions:** - Lupus Erythematosus - Lupus Nephritis **Keywords:** - Systemic Lupus Erythematosus - Active Proliferative Lupus Nephritis ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** SEQUENTIAL **Intervention Model Description:** cohort based escalation of 6 subjects (Type A) single dose cohort of 20 subjects (Type B) ##### Masking Info **Masking:** NONE **Masking Description:** Type A and Type B are open-label. **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 55 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** EQ001 administered in an unblinded dose escalating cohort fashion by subcutaneous injection every two weeks for a total of 2 doses (up to 5 cohorts with dosing to be determined in the range of 0.4 -- 3.2 mg/kg). **Intervention Names:** - Drug: Itolizumab [Bmab 600] **Label:** EQ001 Type A cohort **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** EQ001 administered in an unblinded single dose cohort by subcutaneous injection every two weeks for a total of 13 doses (1.6 mg/kg). **Intervention Names:** - Drug: Itolizumab [Bmab 600] **Label:** EQ001 for Type B cohort **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - EQ001 Type A cohort - EQ001 for Type B cohort **Description:** EQ001 **Name:** Itolizumab [Bmab 600] **Other Names:** - Bmab600 - Itolizumab **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Number of participants with treatment-related adverse events as assessed by the Common Terminology Criteria for Adverse Events (CTCAE) v5.0. **Measure:** Incidence of Treatment Emergent Adverse Events **Time Frame:** Type A up to Day 57 or Type B up to Day 253 #### Secondary Outcomes **Description:** To characterize the pharmacokinetics of itolizumab **Measure:** To characterize the PK of itolizumab **Time Frame:** Type A up to Day 57 or Type B up to Day 253 **Description:** the % levels of free versus EQ001-bound CD6 receptor on T cells **Measure:** CD6 receptor occupancy **Time Frame:** Type A up to Day 57 or Type B up to Day 253 ### Eligibility Module **Eligibility Criteria:** Type A Cohort Key Inclusion Criteria: 1. Is male or female, age ≥ 18 and ≤ 75 years 2. Has previously been documented to have met or currently meets Systemic Lupus International Collaborating Clinics (SLICC) and/or American College of Rheumatology (ACR) criteria for SLE 3. Received at least 1 immunosuppressive or immunomodulatory treatment for SLE at any time in the past or currently 4. Has documented elevation of antinuclear antibodies (ANA) in the past or during Screening 5. Restricted SLE treatments are stable and/or washed out 6. During Screening, has adequate hematologic function Type B Cohort Key Inclusion Criteria: 1. Is male or female, age ≥ 18 and ≤ 75 years 2. Has a diagnosis of SLE 3. Kidney biopsy with a histologic diagnosis of LN Classes III or IV (+/- V) 4. Has a urine protein to creatinine ratio of \> 1000 mg/g 5. Requires induction treatment due to newly diagnosed LN or relapsing/flaring disease or has an incomplete response to current treatment 6. Has adequate hematologic function 7. Restricted SLE treatments are stable and/or washed out 8. Most recent eGFR ≥ 40 mL/min/1.73m2 9. Has evidence of serologic activity Key Exclusion Criteria: 1. Acute or chronic infections requiring systemic antibacterial, antifungal, or antiviral therapy 2. Positive for hepatitis B virus (HBV), hepatitis C virus (HCV), or HIV 3. Active TB or a positive TB test **Maximum Age:** 75 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Sun City **Country:** United States **Facility:** AKDHC Medical Research Services, LLC **State:** Arizona **Zip:** 85351 **Location 2:** **City:** Chula Vista **Country:** United States **Facility:** California Institute of Renal Research **State:** California **Zip:** 91910 **Location 3:** **City:** La Jolla **Country:** United States **Facility:** University of California San Diego Perlman Ambulatory Clinic **State:** California **Zip:** 92037 **Location 4:** **City:** Clearwater **Country:** United States **Facility:** Clinical Research of West Florida - Clearwater **State:** Florida **Zip:** 33765-2616 **Location 5:** **City:** Fort Lauderdale **Country:** United States **Facility:** Centre for Rheumatology, Immunology and Arthritis **State:** Florida **Zip:** 33309 **Location 6:** **City:** Gainesville **Country:** United States **Facility:** University of Florida, Division of Rheumatology **State:** Florida **Zip:** 32610 **Location 7:** **City:** Leesburg **Country:** United States **Facility:** Clinical Site Partners Leesburg, LLC **State:** Florida **Zip:** 34748 **Location 8:** **City:** Miami **Country:** United States **Facility:** SouthCoast Research Center Inc **State:** Florida **Zip:** 33136 **Location 9:** **City:** Miami **Country:** United States **Facility:** Hope Clinical Trials **State:** Florida **Zip:** 33165 **Location 10:** **City:** Orlando **Country:** United States **Facility:** Omega Research Maitland, LLC **State:** Florida **Zip:** 32810 **Location 11:** **City:** Tampa **Country:** United States **Facility:** Clinical Research of West Florida - Tampa **State:** Florida **Zip:** 33603 **Location 12:** **City:** Tampa **Country:** United States **Facility:** University of South Florida **State:** Florida **Zip:** 33606 **Location 13:** **City:** Lawrenceville **Country:** United States **Facility:** Georgia Nephrology **State:** Georgia **Zip:** 30046 **Location 14:** **City:** Bronx **Country:** United States **Facility:** Albert Einstein College of Medicine, Montefiore Medical Center **State:** New York **Zip:** 10461 **Location 15:** **City:** Great Neck **Country:** United States **Facility:** Northwell Health / Division of Rheumatology **State:** New York **Zip:** 11021 **Location 16:** **City:** New York **Country:** United States **Facility:** Columbia University Medical Center, Div of Nephrology **State:** New York **Zip:** 10032 **Location 17:** **City:** Bethlehem **Country:** United States **Facility:** Northeast Clinical Research Center, LLC **State:** Pennsylvania **Zip:** 18017 **Location 18:** **City:** Dallas **Country:** United States **Facility:** Dallas Renal Group **State:** Texas **Zip:** 75230 **Location 19:** **City:** Houston **Country:** United States **Facility:** Prolato Clinical Research Center (PCRC) **State:** Texas **Zip:** 77054 **Location 20:** **City:** Chandigarh **Country:** India **Facility:** Post Graduate Institute of Medical Education and Research (PGIMER) **Location 21:** **City:** Gurugramam **Country:** India **Facility:** Medanta - The Medicity Hospital **Location 22:** **City:** New Delhi **Country:** India **Facility:** MAX Super Specialty Hospital **Location 23:** **City:** Puducherry **Country:** India **Facility:** Jawaharlal Nehru Institute of Postgraduate Medical Education and Research (JIPMER) **Location 24:** **City:** Warszawa **Country:** Poland **Facility:** Miedzyleski Szpital Specjalistyczny w Warszawie, Oddzial Nefrologiczny i Stacja Dializ **Zip:** 04-749 **Location 25:** **City:** Łódź **Country:** Poland **Facility:** SP ZOZ Centralny Szpital Kliniczny Uniwersytetu Medycznego w Lodzi, Klinika Nefrologii, Hipertensjologii **Zip:** 92-213 #### Overall Officials **Official 1:** **Affiliation:** UCSD **Name:** Kenneth Kalunian, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### See Also Links **Label:** company website **URL:** https://equilliumbio.com/ ## Derived Section ### Condition Browse Module - Ancestors - ID: D000003240 - Term: Connective Tissue Diseases - ID: D000001327 - Term: Autoimmune Diseases - ID: D000007154 - Term: Immune System Diseases - ID: D000007674 - Term: Kidney Diseases - ID: D000014570 - Term: Urologic Diseases - ID: D000052776 - Term: Female Urogenital Diseases - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications - ID: D000091642 - Term: Urogenital Diseases - ID: D000052801 - Term: Male Urogenital Diseases - ID: D000005921 - Term: Glomerulonephritis ### Condition Browse Module - Browse Branches - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: All - Name: All Conditions - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: BC20 - Name: Immune System Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M12338 - Name: Nephritis - Relevance: HIGH - As Found: Nephritis - ID: M11178 - Name: Lupus Nephritis - Relevance: HIGH - As Found: Lupus Nephritis - ID: M11177 - Name: Lupus Erythematosus, Systemic - Relevance: HIGH - As Found: Systemic Lupus Erythematosus - ID: M6464 - Name: Connective Tissue Diseases - Relevance: LOW - As Found: Unknown - ID: M4629 - Name: Autoimmune Diseases - Relevance: LOW - As Found: Unknown - ID: M10200 - Name: Immune System Diseases - Relevance: LOW - As Found: Unknown - ID: M10698 - Name: Kidney Diseases - Relevance: LOW - As Found: Unknown - ID: M17319 - Name: Urologic Diseases - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M27095 - Name: Male Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M9031 - Name: Glomerulonephritis - Relevance: LOW - As Found: Unknown - ID: T3523 - Name: Lupus Nephritis - Relevance: HIGH - As Found: Lupus Nephritis - ID: T2525 - Name: Glomerulonephritis - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000009393 - Term: Nephritis - ID: D000008181 - Term: Lupus Nephritis - ID: D000008180 - Term: Lupus Erythematosus, Systemic ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05646979 **Brief Title:** The Effect of Emotional Freedom Technique Applied Before Cesarean Section **Official Title:** The Effect of Emotional Freedom Technique Applied Before Cesarean Section on Perception of Anxiety, Fear of Surgery and Traumatic Birth #### Organization Study ID Info **ID:** Inonu Universty, Malatya #### Organization **Class:** OTHER **Full Name:** Inonu University ### Status Module #### Completion Date **Date:** 2023-06-30 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-03-02 **Type:** ACTUAL **Last Update Submit Date:** 2023-02-28 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2023-06-30 **Type:** ESTIMATED #### Start Date **Date:** 2022-12-31 **Type:** ACTUAL **Status Verified Date:** 2023-02 #### Study First Post Date **Date:** 2022-12-12 **Type:** ACTUAL **Study First Submit Date:** 2022-12-04 **Study First Submit QC Date:** 2022-12-09 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Inonu University #### Responsible Party **Investigator Affiliation:** Inonu University **Investigator Full Name:** Sinem GUVEN Santur **Investigator Title:** Pirincipal İnvestigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Since all the follow-up and care of the pregnant during the prenatal period are carried out by the midwives, the care provided by the midwives to the women during the prenatal period plays a key role for the woman to have a comfortable and healthy pregnancy. Midwives should take psychological approaches in order to reduce the negative feelings of women before cesarean section. Considering all these, it is thought that Emotional Freedom Technique, which does not require any invasive intervention, is inexpensive and easy to apply, will contribute to women's feeling better by reducing pre-cesarean anxiety, surgical fear and traumatic birth perception. **Detailed Description:** Although childbirth is a normal physiological event for the female body, it causes stress for the woman and brings with it many risks. Cesarean section, which is one of the birth types; It is a surgical procedure that enables delivery to occur through an incision made on the abdominal wall and uterus of the mother. Although the cesarean rate recommended by the World Health Organization (WHO) is 15%, the rate of cesarean section in our country is 52.1% according to the Turkey Demographic and Health Survey. Every surgical procedure to be performed, the decision of surgery and the waiting period may cause anxiety in individuals. Anxiety and fear experienced before surgery; It can cause individuals to experience uneasiness, restlessness and dissatisfaction due to the uncertainty they are in. Preoperative pain, fear of death, the thought that the body will be harmed and suffering, fear of losing consciousness and control due to anesthesia, and the uncertainty associated with anesthesia may cause fear of surgery. Anxiety before cesarean section is multifaceted, and it has been reported that the pregnancy process increases anxiety. In studies, it was determined that the anxiety level of mothers before cesarean section was high. The extreme degree of anxiety and fear plays a very important role in the formation of traumatic perception towards birth. Many methods are used to reduce negative emotions in the prenatal period. Emotional Freedom Technique (EFT), which is one of these methods, is a method used to resolve emotional blockages in the energy body of the person. EFT aims to reveal all the points where there is a possibility of negative emotion experienced in the past and stuck in a part of the body. By focusing on the emotions that negatively affect the person in EFT, the energy flow is regulated with stimulations often made by touch. When administered to pregnant women, it may contribute to a decrease in post-traumatic stress disorder and to reduce the level of anxiety and fear. Since all the follow-up and care of the pregnant during the prenatal period are carried out by the midwives, the care provided by the midwives to the women during the prenatal period plays a key role for the woman to have a comfortable and healthy pregnancy. Midwives should take psychological approaches in order to reduce the negative feelings of women before cesarean section. Considering all these, it is thought that EFT, which does not require any invasive intervention, is inexpensive and easy to apply, will contribute to women's feeling better by reducing pre-cesarean anxiety, surgical fear and traumatic birth perception. ### Conditions Module **Conditions:** - Cesarean Section - Emotional Regulation - Surgery - Traumatic Birth **Keywords:** - Cesarean section - Emotional freedom technique - Surgery fear - Traumatic birth perception ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** The randomized experiment was designed as a control study. ##### Masking Info **Masking:** SINGLE **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 53 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** EFT application will be applied to the women in the experimental group who will have a cesarean section at 32-38 weeks of gestation. **Intervention Names:** - Behavioral: EFT group **Label:** Experimental group **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Women in the control group who will have a cesarean section at 32-38 weeks of gestation will not be interfered with. **Label:** Control group **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Experimental group **Description:** EFT protocol will be applied to the women in the experimental group who will have a cesarean section at 32-38 weeks of gestation. **Name:** EFT group **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** The negative 10 feel the greatest pain, disappointment, fear, stress, sadness, or discomfort imaginable. When it reaches Positive 10, the client feels very different and wonderful. The meanings of the ratings in this range change gradually. The scale has no cutoff score. It is interpreted according to the mean of the scores and its relevance to other variables. **Measure:** Subjective Units of Experience- (SUE) **Time Frame:** Each participant will be evaluated for 1 week. #### Secondary Outcomes **Description:** The scale consists of a total of 13 questions covering thoughts and feelings such as anxiety, fear and anxiety that the woman feels when she thinks about the concept of birth. For each problem, a score is given from 0 to 10, whether I'm afraid or afraid. The lowest and highest scores that can be obtained from the scale are 0 and 130. According to the total mean score of the scale, the 0-26 score range is "very low", the 27-52 score range is "low", the 53-78 score range is "moderate", the 79-104 score range is "high", and the 105-130 score range is "very high" indicates the perception of traumatic birth. **Measure:** Traumatic Birth Perception Scale **Time Frame:** It will be applied as a post-test 1 week after it is applied as a pre-test to the pregnants between 32-38 weeks included in the study. **Description:** The scale is in 11-point Likert type, consists of 8 questions and scores between 0 and 10. The scale, each of which consists of 4 items, consists of two sub-dimensions measuring short-term fears and long-term fears related to the source of fear. Items 1 to 4 in the scale measure the fear of short-term results of surgery, while items 5 to 8 measure the fear of long-term results. The items in the scale are evaluated as "0: I am not afraid at all" and "10: I am very afraid". **Measure:** Surgical Fear Scale **Time Frame:** It will be applied as a post-test 1 week after it is applied as a pre-test to the pregnants between 32-38 weeks included in the study. **Description:** The State Anxiety Scale is an individual's at a certain moment and under certain conditions; It requires him to describe how he feels. The emotions and behaviors expressed in the inventory items are indicated by choosing one of the options "(1) Not at all, (2) A little, (3) A lot and (4) Completely" according to the severity of such experiences. The total score obtained can vary between 20 and 80. **Measure:** State anxiety scale **Time Frame:** It will be applied as a post-test 1 week after it is applied as a pre-test to the pregnants between 32-38 weeks included in the study. **Description:** Pregnant information form prepared by the researcher in line with the literature, socio-demographic (pregnant woman's age, education and employment status, income status, family type and place of residence), obstetric (current gestational week and pregnancy history), psycho-social and medical history (desire for pregnancy) status, whether there is a chronic disease) consists of questions. **Measure:** Demographic Descriptive Data **Time Frame:** It will be obtained in 1 month at the beginning of the research. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * 18 years or older, * at 32-38 weeks of gestation, * Cesarean delivery planned, * Not carrying any risk factors (preeclampsia, IUGG, premature rupture of membranes, getational diabetes, etc.) during pregnancy, * Single pregnancy, * Not having any problems (such as fetal anomaly, intrauterine growth retardation) related to the health of the fetus, * Pregnant women who do not have conditions such as infection, wound, scar in the tapping areas. Exclusion Criteria: * Diagnosing a risky pregnancy during the research process, * Participant's unwillingness to continue with the EFT protocol **Healthy Volunteers:** True **Maximum Age:** 49 Years **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Sinem Güven Santur **Phone:** 05424640068 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Zeliha Ozşahin **Phone:** 4223410220 **Phone Ext:** 3792 **Role:** CONTACT #### Locations **Location 1:** **City:** Malatya **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Sinem GüvenSantur, phd - **Phone:** 05424640068 - **Role:** CONTACT **Country:** Turkey **Facility:** Malatya Training and Research Hospital **State:** Kayseri **Status:** RECRUITING **Zip:** 44000 #### Overall Officials **Official 1:** **Affiliation:** https://akademik.ksu.edu.tr/Default.aspx?kod=CfTMEAi69foG7wFizm48ztSNEmgmVjdANJTT1Xcj9gA= **Name:** Esra Karataş Okyay **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** https://avesis.inonu.edu.tr/zeliha.ozsahin/bilimselfaaliyetler **Name:** Zeliha Özşahin **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **Description:** In the research, data collection forms will be filled by face-to-face interview method. Data will be collected with Personal Information Form, State Anxiety Scale (STAI-I), Surgical Fear Scale, Traumatic Birth Perception Scale (TDAS) and Subjective Units of Experience (SUE) Scale. **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007232 - Term: Infant, Newborn, Diseases - ID: D000014947 - Term: Wounds and Injuries ### Condition Browse Module - Browse Branches - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - Abbrev: BC26 - Name: Wounds and Injuries ### Condition Browse Module - Browse Leaves - ID: M4324 - Name: Anxiety Disorders - Relevance: LOW - As Found: Unknown - ID: M5002 - Name: Birth Injuries - Relevance: HIGH - As Found: Traumatic Birth - ID: M10276 - Name: Infant, Newborn, Diseases - Relevance: LOW - As Found: Unknown - ID: M17685 - Name: Wounds and Injuries - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001720 - Term: Birth Injuries ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01216579 **Brief Title:** Steroid Titration Against Mannitol IN Asthma **Official Title:** Titrating Inhaled Steroid Dose Against Mannitol Hyper-responsiveness or BTS Outcomes: Comparative Effects on Asthma Exacerbations Over 1 Year #### Organization Study ID Info **ID:** FARD002 #### Organization **Class:** OTHER **Full Name:** University of Dundee ### Status Module #### Expanded Access Info #### Last Update Post Date **Date:** 2010-10-07 **Type:** ESTIMATED **Last Update Submit Date:** 2010-10-06 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2010-02 **Type:** ACTUAL #### Start Date **Date:** 2004-03 **Status Verified Date:** 2008-03 #### Study First Post Date **Date:** 2010-10-07 **Type:** ESTIMATED **Study First Submit Date:** 2010-10-06 **Study First Submit QC Date:** 2010-10-06 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Dundee #### Responsible Party **Old Name Title:** Professor Brian Jonathon Lipworth **Old Organization:** University of Dundee ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The investigators hypothesise that titration of asthma medication against mannitol challenge results will reduce the number of mild asthma exacerbations, in one year, when compared with titration against BTS guidelines. To test this hypothesis the investigators propose a primary care, parallel treatment, patient blinded study in which matched groups of asthmatic patients will be treated in accordance either with BTS guidelines or with our treatment algorithm dependent on mannitol challenge result. Purpose of the study is to evaluate the efficacy of a treatment algorithm based on the measurement of airway hyperresponsiveness to mannitol challenge, a surrogate marker of airway inflammation, in the long term treatment of asthma in comparison to BTS guidelines. ### Conditions Module **Conditions:** - Asthma ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** BASIC_SCIENCE #### Enrollment Info **Count:** 164 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Asthmatic patients managed as per British Thoracic Society guidelines by symptoms and lung function. **Intervention Names:** - Other: mannitol (an airway challenge agent) **Label:** Reference arm **Type:** PLACEBO_COMPARATOR #### Arm Group 2 **Description:** Group of asthmatic patients managed according to their mannitol challenge. **Intervention Names:** - Other: mannitol (an airway challenge agent) **Label:** Mannitol managed arm **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Mannitol managed arm - Reference arm **Name:** mannitol (an airway challenge agent) **Type:** OTHER ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Male or female asthmatics aged \>/= 16 years * Females must be non pregnant and non lactating * FEV1 \>/= 50% predicted * Mannitol PD10 \</= 635 mg at end of step down period * No recent exacerbations of asthma requiring oral prednisolone in the previous 3 months * Able to perform all the techniques necessary to carry out the challenge testing and lung function and compliant with taking the study medication * Good inhaler technique which will be reinforced at each study visit Exclusion Criteria: * Male or female patients aged 15 or below * FEV1 \</= 50% predicted * Patients who are currently taking a pulse of oral corticosteroids * Patients with the following concomitant illnesses:bronchiectasis, allergic bronchopulmonary aspergillosis, COPD, heart failure, pulmonary fibrosis, rhino-sinusitis with polyps * Immunocompromised patients * Patients with recurrent LRTI * Patients with documented aspirin induced asthma on LRTAs * Pregnancy * Known or suspected hypersensitivity to ICS or other excipients of the MDIs * HIV/Hepatitis B or C positive **Maximum Age:** 65 Years **Minimum Age:** 16 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT ### Contacts Locations Module #### Overall Officials **Official 1:** **Affiliation:** University of Dundee **Name:** Brian J Lipworth, MD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Anderson WJ, Short PM, Jabbal S, Lipworth BJ. Inhaled corticosteroid dose response in asthma: Should we measure inflammation? Ann Allergy Asthma Immunol. 2017 Feb;118(2):179-185. doi: 10.1016/j.anai.2016.11.018. Epub 2017 Jan 3. **PMID:** 28065396 **Citation:** Anderson WJ, McFarlane LC, Lipworth BJ. Prospective follow-up of novel markers of bone turnover in persistent asthmatics exposed to low and high doses of inhaled ciclesonide over 12 months. J Clin Endocrinol Metab. 2012 Jun;97(6):1929-36. doi: 10.1210/jc.2011-3410. Epub 2012 Mar 21. **PMID:** 22438232 **Citation:** Lipworth BJ, Short PM, Williamson PA, Clearie KL, Fardon TC, Jackson CM. A randomized primary care trial of steroid titration against mannitol in persistent asthma: STAMINA trial. Chest. 2012 Mar;141(3):607-615. doi: 10.1378/chest.11-1748. Epub 2011 Oct 13. **PMID:** 21998259 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000001982 - Term: Bronchial Diseases - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000008173 - Term: Lung Diseases, Obstructive - ID: D000008171 - Term: Lung Diseases - ID: D000012130 - Term: Respiratory Hypersensitivity - ID: D000006969 - Term: Hypersensitivity, Immediate - ID: D000006967 - Term: Hypersensitivity - ID: D000007154 - Term: Immune System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: BC20 - Name: Immune System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M4556 - Name: Asthma - Relevance: HIGH - As Found: Asthma - ID: M27137 - Name: Respiratory Aspiration - Relevance: LOW - As Found: Unknown - ID: M5258 - Name: Bronchial Diseases - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown - ID: M11168 - Name: Lung Diseases - Relevance: LOW - As Found: Unknown - ID: M11170 - Name: Lung Diseases, Obstructive - Relevance: LOW - As Found: Unknown - ID: M10018 - Name: Hypersensitivity - Relevance: LOW - As Found: Unknown - ID: M14967 - Name: Respiratory Hypersensitivity - Relevance: LOW - As Found: Unknown - ID: M10020 - Name: Hypersensitivity, Immediate - Relevance: LOW - As Found: Unknown - ID: M10200 - Name: Immune System Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001249 - Term: Asthma ### Intervention Browse Module - Ancestors - ID: D000004234 - Term: Diuretics, Osmotic - ID: D000004232 - Term: Diuretics - ID: D000045283 - Term: Natriuretic Agents - ID: D000045505 - Term: Physiological Effects of Drugs ### Intervention Browse Module - Browse Branches - Abbrev: NaAg - Name: Natriuretic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M11342 - Name: Mannitol - Relevance: HIGH - As Found: Abuse - ID: M7411 - Name: Diuretics - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000008353 - Term: Mannitol ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01584479 **Brief Title:** Periodontal Disease Prevention Study **Official Title:** Periodontal Disease Prevention Study:A Retrospective Cohort Study to Assess the Effect of Genetics and Dental Preventive Care on Periodontal Disease #### Organization Study ID Info **ID:** HUM00037624 #### Organization **Class:** OTHER **Full Name:** University of Michigan ### Status Module #### Completion Date **Date:** 2012-03 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2015-07-09 **Type:** ESTIMATED **Last Update Submit Date:** 2015-06-17 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2012-03 **Type:** ACTUAL #### Results First Post Date **Date:** 2015-07-09 **Type:** ESTIMATED **Results First Submit Date:** 2014-06-13 **Results First Submit QC Date:** 2015-06-17 #### Start Date **Date:** 2010-12 **Status Verified Date:** 2015-06 #### Study First Post Date **Date:** 2012-04-25 **Type:** ESTIMATED **Study First Submit Date:** 2012-04-12 **Study First Submit QC Date:** 2012-04-24 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Michigan #### Responsible Party **Investigator Affiliation:** University of Michigan **Investigator Full Name:** William Giannobile **Investigator Title:** Principal Investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** This study is one component of a program to improve the maintenance of good dental health and the prevention of disease by use of risk stratification methods to efficiently guide increased preventive services to adult dental patients who are at increased risk for the major dental diseases, caries and adult periodontitis. The investigators will use a retrospective cohort model to analyze a large dental claims database to determine if the frequency of preventive services influenced the periodontal disease outcomes and to determine if periodontal risk assessment information can be used to stratify dental patients into "high risk" and "low risk" categories that influence the effect of preventive services on periodontitis outcomes. Primary Objective To evaluate whether dental patients who are classified as "low risk" for periodontal disease progression, based on history of periodontitis (claims history), smoking, diabetes, and IL-1 genetic variations, have different primary and secondary endpoints if they had two dental cleanings per year compared to one cleaning per year. Secondary Objectives To evaluate whether dental patients who are classified as "high risk" for periodontal disease progression, based on a history of periodontitis, smoking, diabetes, and IL-1 genetic variations, have different primary and secondary endpoints if they had two dental cleanings per year compared to one cleaning per year. To evaluate whether dental patients who have had one dental cleaning per year have different primary and secondary endpoints if they are classified as "low risk" for periodontal disease compared to patients who are classified as "high risk." To evaluate whether dental patients who have had two dental cleaning per year have different primary and secondary endpoints if they are classified as "low risk" for periodontal disease compared to patients who are classified as "high risk." ### Conditions Module **Conditions:** - Periodontitis **Keywords:** - periodontitis ### Design Module #### Bio Spec **Description:** buccal swabs **Retention:** SAMPLES_WITH_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** RETROSPECTIVE #### Enrollment Info **Count:** 5117 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 1 visit - Low Risk \~1200 subjects - Low Risk Experimental Group = 1 visit intervention, no history of periodontitis, non-smoker, non-diabetic, IL-1 genotype (-) **Label:** Low Risk Experimental Group #### Arm Group 2 **Description:** 2 visits - Low Risk \~1200 subjects - Low risk Control Group = 2 visit intervention, no history of periodontitis, non-smoker, non-diabetic, IL-1 genotype (-) **Label:** Low risk Control Group #### Arm Group 3 **Description:** 1 visit - High Risk \~800 subjects High Risk Experimental Group = 1 visit intervention, one or more of the following risk factors: history of periodontitis, smoker or diabetic, IL-1 genotype (+) **Label:** High Risk Experimental Group #### Arm Group 4 **Description:** 2 visits - High Risk \~800 subjects High Risk Control Group = 2 visit intervention, one or more of the following risk factors: history of periodontitis, smoker or diabetic, IL-1 genotype (+) **Label:** High Risk Control Group ### Outcomes Module #### Primary Outcomes **Description:** Tooth loss rate over 16 years was calculated as the cumulative percentage of participants with tooth loss over 16 years. **Measure:** Percentage of Participants With Tooth Loss Over 16 Year Period **Time Frame:** 16 years #### Secondary Outcomes **Measure:** Change in Total Dental Claims During Monitoring Period **Time Frame:** 10 and 15 years **Measure:** Change in Total Periodontal Claims During the Monitoring Period **Time Frame:** 10 and 15 years **Measure:** Change in Periodontal Surgery Claims **Time Frame:** 10 and 15 years **Measure:** Change in Relationship of Risk Category & Tooth Loss Rate With Systemic Disease History on Questionnaire. **Time Frame:** 10 and 15 years **Measure:** Change in Evaluation for Dental Caries and Oral Cancer Within the Risk Categories **Time Frame:** 10 and 15 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Study may include all participants who are 34-55 years old in 1992 (48-68 years old in 2010) and who have at least a 15-year dental claim history with Delta Dental. Exclusion Criteria: * Organ transplant recipients are excluded. **Healthy Volunteers:** True **Maximum Age:** 55 Years **Minimum Age:** 34 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT **Study Population:** Study may include all participants who are 34-55 years old in 1992 (48-68 years old in 2010) and who have at least a 15-year dental claim history with Delta Dental. ### Contacts Locations Module #### Locations **Location 1:** **City:** Ann Arbor **Country:** United States **Facility:** Michigan Center for Oral Health Research **State:** Michigan **Zip:** 48106 #### Overall Officials **Official 1:** **Affiliation:** University of Michigan **Name:** William V Giannobile, DDS, DMedSc **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Giannobile WV, Braun TM, Caplis AK, Doucette-Stamm L, Duff GW, Kornman KS. Patient stratification for preventive care in dentistry. J Dent Res. 2013 Aug;92(8):694-701. doi: 10.1177/0022034513492336. Epub 2013 Jun 10. **PMID:** 23752171 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009059 - Term: Mouth Diseases - ID: D000009057 - Term: Stomatognathic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC07 - Name: Mouth and Tooth Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M13427 - Name: Periodontitis - Relevance: HIGH - As Found: Periodontitis - ID: M13419 - Name: Periodontal Diseases - Relevance: HIGH - As Found: Periodontal Diseases - ID: M12019 - Name: Mouth Diseases - Relevance: LOW - As Found: Unknown - ID: M12017 - Name: Stomatognathic Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000010518 - Term: Periodontitis - ID: D000010510 - Term: Periodontal Diseases ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** This is an survey research study with the patient collection of their own biological specimen and the filling out of a survey to release their payer data (non-interventional). #### Event Groups **Group ID:** EG000 **Title:** Low Risk Experimental Group **Description:** 1 visit - Low Risk \~1200 subjects - Low Risk Experimental Group = 1 visit intervention, no history of periodontitis, non-smoker, non-diabetic, IL-1 genotype (-) **ID:** EG000 **Other Num at Risk:** 732 **Serious Number At Risk:** 732 **Title:** Low Risk Experimental Group **Group ID:** EG001 **Title:** Low Risk Control Group **Description:** 2 visits - Low Risk \~1200 subjects - Low risk Control Group = 2 visit intervention, no history of periodontitis, non-smoker, non-diabetic, IL-1 genotype (-) **ID:** EG001 **Other Num at Risk:** 1686 **Serious Number At Risk:** 1686 **Title:** Low Risk Control Group **Group ID:** EG002 **Title:** High Risk Experimental Group **Description:** 1 visit - High Risk \~800 subjects High Risk Experimental Group = 1 visit intervention, one or more of the following risk factors: history of periodontitis, smoker or diabetic, IL-1 genotype (+) **ID:** EG002 **Other Num at Risk:** 852 **Serious Number At Risk:** 852 **Title:** High Risk Experimental Group **Group ID:** EG003 **Title:** High Risk Control Group **Description:** 2 visits - High Risk \~800 subjects High Risk Control Group = 2 visit intervention, one or more of the following risk factors: history of periodontitis, smoker or diabetic, IL-1 genotype (+) **ID:** EG003 **Other Num at Risk:** 1847 **Serious Number At Risk:** 1847 **Title:** High Risk Control Group **Frequency Threshold:** 0 ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 732 **Group ID:** BG001 **Value:** 1686 **Group ID:** BG002 **Value:** 852 **Group ID:** BG003 **Value:** 1847 **Group ID:** BG004 **Value:** 5117 **Units:** Participants ### Group **ID:** BG000 **Title:** Low Risk Experimental Group **Description:** 1 visit - Low Risk \~1200 subjects - Low Risk Experimental Group = 1 visit intervention, no history of periodontitis, non-smoker, non-diabetic, IL-1 genotype (-) ### Group **ID:** BG001 **Title:** Low Risk Control Group **Description:** 2 visits - Low Risk \~1200 subjects - Low risk Control Group = 2 visit intervention, no history of periodontitis, non-smoker, non-diabetic, IL-1 genotype (-) ### Group **ID:** BG002 **Title:** High Risk Experimental Group **Description:** 1 visit - High Risk \~800 subjects High Risk Experimental Group = 1 visit intervention, one or more of the following risk factors: history of periodontitis, smoker or diabetic, IL-1 genotype (+) ### Group **ID:** BG003 **Title:** High Risk Control Group **Description:** 2 visits - High Risk \~800 subjects High Risk Control Group = 2 visit intervention, one or more of the following risk factors: history of periodontitis, smoker or diabetic, IL-1 genotype (+) ### Group **ID:** BG004 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Value:** 732 #### Measurement **Group ID:** BG001 **Value:** 1686 #### Measurement **Group ID:** BG002 **Value:** 852 #### Measurement **Group ID:** BG003 **Value:** 1847 #### Measurement **Group ID:** BG004 **Value:** 5117 **Class Title:** >34 and < 55 years ### Measure #### Measurement **Group ID:** BG000 **Value:** 461 #### Measurement **Group ID:** BG001 **Value:** 1130 #### Measurement **Group ID:** BG002 **Value:** 537 #### Measurement **Group ID:** BG003 **Value:** 1238 #### Measurement **Group ID:** BG004 **Value:** 3366 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 271 #### Measurement **Group ID:** BG001 **Value:** 556 #### Measurement **Group ID:** BG002 **Value:** 315 #### Measurement **Group ID:** BG003 **Value:** 609 #### Measurement **Group ID:** BG004 **Value:** 1751 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 732 #### Measurement **Group ID:** BG001 **Value:** 1686 #### Measurement **Group ID:** BG002 **Value:** 852 #### Measurement **Group ID:** BG003 **Value:** 1847 #### Measurement **Group ID:** BG004 **Value:** 5117 **Class Title:** United States **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Description:** The participants analyzed had to be within this age range during the year 2010. **Parameter Type:** NUMBER **Title:** Age, Customized **Unit of Measure:** participants ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** participants **Population Description:** See Giannobile et al 2013 manuscript included ## Results Section - More Information Module ### Certain Agreement ### Point of Contact **Email:** [email protected] **Organization:** University of Michigan **Phone:** 734-764-1562 **Title:** William Giannobile, Principal Investigator ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### Outcome Measure 4 ### Outcome Measure 5 ### Outcome Measure 6 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 16.4 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 13.8 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 22.1 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 16.9 **Title:** #### Outcome Measure 2 #### Outcome Measure 3 #### Outcome Measure 4 #### Outcome Measure 5 #### Outcome Measure 6 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Tooth loss rate over 16 years was calculated as the cumulative percentage of participants with tooth loss over 16 years. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** 16 years **Title:** Percentage of Participants With Tooth Loss Over 16 Year Period **Type:** PRIMARY **Unit of Measure:** percentage of participants ##### Group **Description:** 1 visit - Low Risk \~1200 subjects - Low Risk Experimental Group = 1 visit intervention, no history of periodontitis, non-smoker, non-diabetic, IL-1 genotype (-) **ID:** OG000 **Title:** Low Risk Experimental Group ##### Group **Description:** 2 visits - Low Risk \~1200 subjects - Low risk Control Group = 2 visit intervention, no history of periodontitis, non-smoker, non-diabetic, IL-1 genotype (-) **ID:** OG001 **Title:** Low Risk Control Group ##### Group **Description:** 1 visit - High Risk \~800 subjects High Risk Experimental Group = 1 visit intervention, one or more of the following risk factors: history of periodontitis, smoker or diabetic, IL-1 genotype (+) **ID:** OG002 **Title:** High Risk Experimental Group ##### Group **Description:** 2 visits - High Risk \~800 subjects High Risk Control Group = 2 visit intervention, one or more of the following risk factors: history of periodontitis, smoker or diabetic, IL-1 genotype (+) **ID:** OG003 **Title:** High Risk Control Group #### Outcome Measure 2 **Reporting Status:** NOT_POSTED **Time Frame:** 10 and 15 years **Title:** Change in Total Dental Claims During Monitoring Period **Type:** SECONDARY #### Outcome Measure 3 **Reporting Status:** NOT_POSTED **Time Frame:** 10 and 15 years **Title:** Change in Total Periodontal Claims During the Monitoring Period **Type:** SECONDARY #### Outcome Measure 4 **Reporting Status:** NOT_POSTED **Time Frame:** 10 and 15 years **Title:** Change in Periodontal Surgery Claims **Type:** SECONDARY #### Outcome Measure 5 **Reporting Status:** NOT_POSTED **Time Frame:** 10 and 15 years **Title:** Change in Relationship of Risk Category & Tooth Loss Rate With Systemic Disease History on Questionnaire. **Type:** SECONDARY #### Outcome Measure 6 **Reporting Status:** NOT_POSTED **Time Frame:** 10 and 15 years **Title:** Change in Evaluation for Dental Caries and Oral Cancer Within the Risk Categories **Type:** SECONDARY ### Participant Flow Module #### Group **Description:** 1 visit - Low Risk 732 evaluable subjects - Low Risk Experimental Group = 1 visit intervention, no history of periodontitis, non-smoker, non-diabetic, IL-1 genotype (-) **ID:** FG000 **Title:** Low Risk Experimental Group #### Group **Description:** 2 visits - Low Risk 1,668 evaluable subjects - Low risk Control Group = 2 visit intervention, no history of periodontitis, non-smoker, non-diabetic, IL-1 genotype (-) **ID:** FG001 **Title:** Low Risk Control Group #### Group **Description:** 1 visit - High Risk 852 evaluable subjects High Risk Experimental Group = 1 visit intervention, one or more of the following risk factors: history of periodontitis, smoker or diabetic, IL-1 genotype (+) **ID:** FG002 **Title:** High Risk Experimental Group #### Group **Description:** 2 visits - High Risk 1,847 evaluable subjects High Risk Control Group = 2 visit intervention, one or more of the following risk factors: history of periodontitis, smoker or diabetic, IL-1 genotype (+) **ID:** FG003 **Title:** High Risk Control Group #### Period **Title:** Overall Study ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 732 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 1686 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 852 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 1847 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 732 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 1686 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 852 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 1847 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 0 **Recruitment Details:** From 25,452 individuals meeting inclusion criteria, 9,927 (0.39 of eligible) consented to participate, and 5,117(0.515 of consented) returned completed questionnaires and were successfully genotyped. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT01602679 **Brief Title:** Acute Effects of Progesterone on LH Pulses During the Follicular Phase (CRM006) **Official Title:** Acute Effects of Progesterone on LH Pulses During the Follicular Phase #### Organization Study ID Info **ID:** 16085 #### Organization **Class:** OTHER **Full Name:** University of Virginia #### Secondary ID Infos **ID:** R01HD058671 **Link:** https://reporter.nih.gov/quickSearch/R01HD058671 **Type:** NIH ### Status Module #### Completion Date **Date:** 2015-07-31 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-10-16 **Type:** ACTUAL **Last Update Submit Date:** 2018-10-15 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2015-07-31 **Type:** ACTUAL #### Results First Post Date **Date:** 2018-10-16 **Type:** ACTUAL **Results First Submit Date:** 2018-07-31 **Results First Submit QC Date:** 2018-10-15 #### Start Date **Date:** 2012-05 **Status Verified Date:** 2018-10 #### Study First Post Date **Date:** 2012-05-21 **Type:** ESTIMATED **Study First Submit Date:** 2012-05-17 **Study First Submit QC Date:** 2012-05-18 **Why Stopped:** Interim analysis suggested futility. ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) #### Lead Sponsor **Class:** OTHER **Name:** University of Virginia #### Responsible Party **Investigator Affiliation:** University of Virginia **Investigator Full Name:** Chris McCartney **Investigator Title:** Associate Professor of Medicine **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Oversight Has DMC:** True ### Description Module **Brief Summary:** The rapidity with which progesterone slows LH (and by inference GnRH) pulse frequency in women is unclear. The investigators hypothesize that progesterone slows LH pulse frequency within 10 hours. The investigators propose to assess this further with a randomized, cross-over, placebo-controlled study. Regularly cycling women without hyperandrogenism will be admitted to the Clinical Research Unit on cycle day 5-9 (mid-follicular phase) for a 10 hour frequent sampling study to observe LH, FSH, estradiol, progesterone, and testosterone. Either oral micronized progesterone suspension or placebo will be administered at 0900 h. During a subsequent menstrual cycle, subjects will undergo another study identical to the first except that oral progesterone will be exchanged for placebo or vice versa in accordance with a crossover design. **Detailed Description:** The rapidity with which progesterone slows LH (and by inference GnRH) pulse frequency in women is unclear. The investigators hypothesize that progesterone slows LH pulse frequency within 10 hours. The investigators propose to assess this further with a randomized, cross-over, placebo-controlled study. Regularly cycling women without hyperandrogenism will be admitted to the Clinical Research Unit on cycle day 5-9 (mid-follicular phase) for a frequent sampling study. Beginning at 0900 h, blood for LH, FSH, estradiol, progesterone, and testosterone will be obtained over a 10-hour period. Either oral micronized progesterone (100 mg p.o.) suspension or placebo will be administered at 0900 h. During a subsequent menstrual cycle, subjects will undergo another study identical to the first except that oral progesterone will be exchanged for placebo or vice versa in accordance with a crossover design. The primary endpoint of interest is LH pulse frequency; the investigators will compare LH pulse frequency after progesterone administration to LH pulse frequency after placebo administration. ### Conditions Module **Conditions:** - Female Reproductive Physiology ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER **Intervention Model Description:** Randomized, placebo-controlled, crossover study ##### Masking Info **Masking:** QUADRUPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** BASIC_SCIENCE #### Enrollment Info **Count:** 12 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants first received oral micronized progesterone (100 mg p.o.) suspension. After a washout period of approximately 20 days, they then received placebo (matching oral micronized progesterone suspension). **Intervention Names:** - Drug: oral micronized progesterone suspension - Drug: Placebo **Label:** micronized progesterone, then placebo **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants first received placebo. After a washout period of approximately 20 days, they then received oral micronized progesterone syrup (100 mg p.o.)Placebo contains only inert ingredients and is not expected to exert any direct physiological effects **Intervention Names:** - Drug: oral micronized progesterone suspension - Drug: Placebo **Label:** Placebo, then micronized progesterone **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Placebo, then micronized progesterone - micronized progesterone, then placebo **Description:** oral micronized progesterone (100 mg p.o.) suspension **Name:** oral micronized progesterone suspension **Other Names:** - Progesterone **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Placebo, then micronized progesterone - micronized progesterone, then placebo **Description:** Placebo contains only inert ingredients and is not expected to exert any direct physiological effects **Name:** Placebo **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** The primary endpoint is the change in the number of LH pulses per hour (over 10 h), comparing (a) number of LH pulses at baseline to (b) number of LH pulses immediately after progesterone or placebo administration **Measure:** Change in Number of LH Pulses Per Hour **Time Frame:** 10 hours following administration of micronized progesterone or placebo #### Secondary Outcomes **Description:** This secondary endpoint is the change of the mean LH (over 10 h), comparing (a) mean LH at baseline to (b) mean LH immediately after progesterone or placebo administration **Measure:** Change in Mean LH **Time Frame:** 10 hours following administration of micronized progesterone or placebo **Description:** This secondary endpoint is the change in the mean LH amplitude (over 10 h), comparing (a) mean LH amplitude at baseline to (b) mean LH amplitude immediately after progesterone or placebo administration **Measure:** Change in Mean LH Amplitude **Time Frame:** 10 hours following administration of micronized progesterone or placebo ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Subjects will be healthy women with regular menstrual cycles and no evidence of hyperandrogenism. * Subjects will be 18-30 years old; the investigators use a cutoff age of 30 years because age-related alterations in the hypothalamic-pituitary-ovarian axis is uncommon before age 30 years. * Subjects will be willing to strictly avoid pregnancy (using non-hormonal methods) during the time of study and must be willing and able to provide informed consent. Exclusion Criteria: * Pregnancy * Lactation * History of allergy to progesterone * BMI \< 18 kg/m2 or \> 30 kg/m2 (underweight and obesity can affect hypothalamic-pituitary-ovarian function) * Excessive exercise, defined as routine and current engagement in either (a) moderate exercise (e.g., brisk walking) exceeding 14 hours per week or (a) vigorous exercise exceeding 7 hours a week. * Clinical hyperandrogenism (primarily hirsutism) * Abnormally elevated free testosterone or DHEAS concentration * A previous diagnosis of diabetes, a fasting glucose ≥ 126 mg/dl * Abnormal TSH (subjects with adequately treated hypothyroidism, reflected by normal TSH values, will not be excluded; or, for a new diagnosis of hypothyroidism, further study will at the least be delayed pending appropriate treatment) (confirmed on repeat) * Abnormal prolactin (confirmed on repeat) * Evidence of Cushing's syndrome by history or physical exam * History of venous thromboembolism, breast/ovarian/endometrial cancer * The investigators will exclude women with any other cancer diagnosis and/or treatment (with the exception of basal cell or squamous skin carcinoma) unless they have remained clinically disease free (based on appropriate surveillance) for five years. * Women with anemia (hematocrit \< 36% and hemoglobin level \< 12 g/dl) will be treated with iron for a maximum of 2 sequential months before the 1st admission and/or before the 2nd admission. If they remain anemic after 2 sequential months of ferrous gluconate (325 mg bid), they will then be excluded from further participation in the study. * Women with a significant history of cardiac or pulmonary dysfunction (e.g., known or suspected congestive heart failure; known or suspected coronary atherosclerosis; asthma requiring systemic intermittent corticosteroids; etc.) will be excluded. * Women with liver enzymes, alkaline phosphatase, or bilirubin \> 1.5 times upper limit of normal (confirmed on repeat) will be excluded, with the exception that mild bilirubin elevations will be accepted in the setting of known Gilbert's syndrome. * Abnormal sodium or potassium concentrations (confirmed on repeat); bicarbonate concentrations \<20 or \>30 (confirmed on repeat) * Women with abnormal renal function (i.e., serum creatinine \> 1.4) will be excluded (confirmed on repeat) * Due to the amount of blood being drawn in the study, subjects with body weight \< 110 pounds will be excluded from the study * Being a study of the acute effects of progesterone on the hypothalamic-pituitary unit, subjects must not take hormonal medications (e.g., oral contraceptives) or other medications known to affect the reproductive axis for 60 days prior to the study and during the study. **Healthy Volunteers:** True **Maximum Age:** 30 Years **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Charlottesville **Country:** United States **Facility:** Center for Research in Reproduction **State:** Virginia **Zip:** 22908 #### Overall Officials **Official 1:** **Affiliation:** University of Virginia **Name:** Christopher R. McCartney, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Description:** We do not have current plans to share IPD **IPD Sharing:** UNDECIDED ## Document Section ### Large Document Module #### Large Docs - Date: 2014-09-24 - Filename: Prot_SAP_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: True - Label: Study Protocol and Statistical Analysis Plan - Size: 1004760 - Type Abbrev: Prot_SAP - Upload Date: 2018-07-03T15:29 ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: Rare - Name: Rare Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Ancestors - ID: D000011372 - Term: Progestins - ID: D000006728 - Term: Hormones - ID: D000006730 - Term: Hormones, Hormone Substitutes, and Hormone Antagonists - ID: D000045505 - Term: Physiological Effects of Drugs ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M14245 - Name: Progesterone - Relevance: HIGH - As Found: Chinese - ID: M14244 - Name: Progestins - Relevance: LOW - As Found: Unknown - ID: M9789 - Name: Hormones - Relevance: LOW - As Found: Unknown - ID: M9788 - Name: Hormone Antagonists - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000011374 - Term: Progesterone ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Oral Micronized Progesterone Suspension **Deaths Num At Risk:** 4 **Description:** Oral micronized progesterone (100 mg) suspension **ID:** EG000 **Other Num Affected:** 1 **Other Num at Risk:** 4 **Serious Number At Risk:** 4 **Title:** Oral Micronized Progesterone Suspension **Group ID:** EG001 **Title:** Placebo **Deaths Num At Risk:** 3 **Description:** Placebo contains only inert ingredients and is not expected to exert any direct physiological effects **ID:** EG001 **Other Num at Risk:** 3 **Serious Number At Risk:** 3 **Title:** Placebo **Frequency Threshold:** 0 #### Other Events **Term:** Irregular Menstrual Bleeding **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Notes:** Temporary spotting **Organ System:** Reproductive system and breast disorders **Source Vocabulary:** **Time Frame:** From start of study procedures until end of study participation, up to 6 months ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 4 **Group ID:** BG001 **Value:** 3 **Group ID:** BG002 **Value:** 7 **Units:** Participants ### Group **ID:** BG000 **Title:** Oral Micronized Progesterone Suspension, Then Placebo **Description:** Participants first received oral micronized progesterone (100 mg p.o.) suspension. After a washout period of approximately 20 days, they then received Placebo. oral micronized progesterone suspension: oral micronized progesterone (100 mg p.o.) suspension Placebo: Placebo contains only inert ingredients and is not expected to exert any direct physiological effects ### Group **ID:** BG001 **Title:** Placebo, Then Oral Micronized Progesterone Suspension **Description:** Participants first received Placebo. After a washout period of approximately 20 days, they then received oral micronized progesterone suspension. Placebo contains only inert ingredients and is not expected to exert any direct physiological effects Placebo: Placebo contains only inert ingredients and is not expected to exert any direct physiological effects oral micronized progesterone suspension: oral micronized progesterone (100 mg p.o.) suspension ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 4 #### Measurement **Group ID:** BG001 **Value:** 3 #### Measurement **Group ID:** BG002 **Value:** 7 **Category Title:** Between 18 and 65 years #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** >=65 years **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 4 #### Measurement **Group ID:** BG001 **Value:** 3 #### Measurement **Group ID:** BG002 **Value:** 7 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** American Indian or Alaska Native #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Native Hawaiian or Other Pacific Islander #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** White #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** More than one race #### Measurement **Group ID:** BG000 **Value:** 4 #### Measurement **Group ID:** BG001 **Value:** 3 #### Measurement **Group ID:** BG002 **Value:** 7 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 4 #### Measurement **Group ID:** BG001 **Value:** 3 #### Measurement **Group ID:** BG002 **Value:** 7 **Class Title:** United States **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Age, Categorical **Unit of Measure:** Participants ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Race (NIH/OMB) **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** participants ## Results Section - More Information Module ### Certain Agreement ### Point of Contact **Email:** [email protected] **Organization:** University of Virginia Center for Research in Reproduction **Phone:** 4342430329 **Title:** Dr. Christopher R. McCartney ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.28 - **Upper Limit:** - **Value:** 0.77 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.35 - **Upper Limit:** - **Value:** 0.79 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2.2 - **Upper Limit:** - **Value:** 5.8 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 2.3 - **Upper Limit:** - **Value:** 4.6 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2.8 - **Upper Limit:** - **Value:** 4.3 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 2.8 - **Upper Limit:** - **Value:** 3.6 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** The primary endpoint is the change in the number of LH pulses per hour (over 10 h), comparing (a) number of LH pulses at baseline to (b) number of LH pulses immediately after progesterone or placebo administration **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 10 hours following administration of micronized progesterone or placebo **Title:** Change in Number of LH Pulses Per Hour **Type:** PRIMARY **Unit of Measure:** pulses/h ##### Group **Description:** oral micronized progesterone (100 mg p.o.) suspension oral micronized progesterone suspension: oral micronized progesterone (100 mg p.o.) suspension **ID:** OG000 **Title:** Oral Micronized Progesterone Suspension ##### Group **Description:** Placebo contains only inert ingredients and is not expected to exert any direct physiological effects Placebo: Placebo contains only inert ingredients and is not expected to exert any direct physiological effects **ID:** OG001 **Title:** Placebo #### Outcome Measure 2 **Description:** This secondary endpoint is the change of the mean LH (over 10 h), comparing (a) mean LH at baseline to (b) mean LH immediately after progesterone or placebo administration **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 10 hours following administration of micronized progesterone or placebo **Title:** Change in Mean LH **Type:** SECONDARY **Unit of Measure:** IU/L ##### Group **Description:** oral micronized progesterone (100 mg p.o.) suspension oral micronized progesterone suspension: oral micronized progesterone (100 mg p.o.) suspension **ID:** OG000 **Title:** Oral Micronized Progesterone Suspension ##### Group **Description:** Placebo contains only inert ingredients and is not expected to exert any direct physiological effects Placebo: Placebo contains only inert ingredients and is not expected to exert any direct physiological effects **ID:** OG001 **Title:** Placebo #### Outcome Measure 3 **Description:** This secondary endpoint is the change in the mean LH amplitude (over 10 h), comparing (a) mean LH amplitude at baseline to (b) mean LH amplitude immediately after progesterone or placebo administration **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 10 hours following administration of micronized progesterone or placebo **Title:** Change in Mean LH Amplitude **Type:** SECONDARY **Unit of Measure:** IU/L ##### Group **Description:** oral micronized progesterone (100 mg p.o.) suspension oral micronized progesterone suspension: oral micronized progesterone (100 mg p.o.) suspension **ID:** OG000 **Title:** Oral Micronized Progesterone Suspension ##### Group **Description:** Placebo contains only inert ingredients and is not expected to exert any direct physiological effects Placebo: Placebo contains only inert ingredients and is not expected to exert any direct physiological effects **ID:** OG001 **Title:** Placebo ### Participant Flow Module #### Group **Description:** Participants first received oral micronized progesterone (100 mg p.o.) suspension. After a washout period of approximately 20 days, they then received placebo. oral micronized progesterone suspension: oral micronized progesterone (100 mg p.o.) suspension **ID:** FG000 **Title:** Oral Micronized Progesterone Suspension, Then Placebo #### Group **Description:** Participants first received Placebo. After a washout period of approximately 20 days, they then received oral micronized progesterone suspension. Placebo contains only inert ingredients and is not expected to exert any direct physiological effects Placebo: Placebo contains only inert ingredients and is not expected to exert any direct physiological effects **ID:** FG001 **Title:** Placebo, Then Oral Micronized Progesterone Suspension #### Period **Title:** Overall Study ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 4 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 3 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 4 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 3 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 0 **Pre-Assignment Details:** 12 subjects enrolled in this study. 5 subjects completed screening procedures only (these 5 subjects were withdrawn from study prior to being assigned to an arm of intervention). **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT02562079 **Acronym:** VISS **Brief Title:** Vasculopathy, Inflammation and Systemic Sclerosis **Official Title:** Vasculopathy, Inflammation and Systemic Sclerosis: The Role of Endothelial Cell Activation and OX40/OX40L in Modulation of T Lymphocyte Activation #### Organization Study ID Info **ID:** CHUBX 2011/37 #### Organization **Class:** OTHER **Full Name:** University Hospital, Bordeaux ### Status Module #### Completion Date **Date:** 2016-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-04-07 **Type:** ACTUAL **Last Update Submit Date:** 2017-04-05 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2016-12 **Type:** ACTUAL #### Start Date **Date:** 2012-03 **Type:** ACTUAL **Status Verified Date:** 2017-04 #### Study First Post Date **Date:** 2015-09-29 **Type:** ESTIMATED **Study First Submit Date:** 2014-12-29 **Study First Submit QC Date:** 2015-09-25 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Societe Francaise de Rhumatologie #### Lead Sponsor **Class:** OTHER **Name:** University Hospital, Bordeaux #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** It is a study of basic research with mechanistically objectives and including clinical biological samples. **Detailed Description:** Systemic sclerosis (SSc) is a rare and severe disease characterised by a fibrotic process and an incompletely elucidate physiopathology. Several shared featured have been identified between SSc and another autoimmune disease, the systemic lupus erythematous (SLE) as an interferon-alpha signature, the role of platelets and the polymorphism of OX40 ligand (OX40L). In SLE, OX40L has been shown highly linked to the active form of the disease, was increased by the CD40L of platelets and induced the CD8 cytotoxicity while inhibiting the suppressive functions of regulator T lymphocytes. The third main factor of the SSc physiopathology apart from autoimmunity and fibrosis is the vasculopathy with an important role of endothelial cells (EC). They turned out to be half-professional antigen presenting cells and can modulate the adaptive immunity. ### Conditions Module **Conditions:** - Systemic Sclerosis **Keywords:** - T lymphocyte - OX40/OX40L ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** OTHER #### Enrollment Info **Count:** 350 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Biological: Blood samples - Biological: Biopsy **Label:** subjects SSc diagnosed **Type:** EXPERIMENTAL #### Arm Group 2 **Intervention Names:** - Biological: Blood samples - Biological: Biopsy **Label:** subjects Localised sclerosis diagnosed **Type:** EXPERIMENTAL #### Arm Group 3 **Intervention Names:** - Biological: Blood samples **Label:** subjects Sc **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - subjects Localised sclerosis diagnosed - subjects SSc diagnosed - subjects Sc **Description:** * biological features of the standard follow-up * 2 more blood tube for the biological collection (serum and PBMC) **Name:** Blood samples **Type:** BIOLOGICAL #### Intervention 2 **Arm Group Labels:** - subjects Localised sclerosis diagnosed - subjects SSc diagnosed **Description:** Skin biopsies **Name:** Biopsy **Type:** BIOLOGICAL ### Outcomes Module #### Primary Outcomes **Measure:** Assessment of OX40L expression in endothelial cells and skin biopsies. **Time Frame:** Day 1 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Age from 18 to 75. * SSc diagnosed according to the American College of Rheumatology (ACR) criteria. * With skin manifestations since less than 10 years. * Localised sclerosis (LSc) diagnosed, morphea type. Exclusion Criteria: * Age inferior to 18 or upper than 75. * Skin manifestations since more than 10 years. * Haemostasis diseases (independent from treatments). * Stem cell transplant. * Immunosuppressive treatments in the last 6 months. * Associate autoimmune disease. **Maximum Age:** 75 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Bordeaux **Country:** France **Facility:** Service de Rhumatologie - Tripode - Hôpital Pellegrin **Zip:** 33076 #### Overall Officials **Official 1:** **Affiliation:** University Hospital Bordeaux, France **Name:** Marie-Elise TRUCHETET, MD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010335 - Term: Pathologic Processes - ID: D000003240 - Term: Connective Tissue Diseases - ID: D000012871 - Term: Skin Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M10293 - Name: Inflammation - Relevance: HIGH - As Found: Inflammation - ID: M15415 - Name: Sclerosis - Relevance: HIGH - As Found: Sclerosis - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown - ID: M15412 - Name: Scleroderma, Systemic - Relevance: HIGH - As Found: Systemic Sclerosis - ID: M25560 - Name: Scleroderma, Diffuse - Relevance: HIGH - As Found: Systemic Sclerosis - ID: M6464 - Name: Connective Tissue Diseases - Relevance: LOW - As Found: Unknown - ID: M15674 - Name: Skin Diseases - Relevance: LOW - As Found: Unknown - ID: T5565 - Name: Systemic Scleroderma - Relevance: HIGH - As Found: Systemic Sclerosis ### Condition Browse Module - Meshes - ID: D000012595 - Term: Scleroderma, Systemic - ID: D000045743 - Term: Scleroderma, Diffuse - ID: D000012598 - Term: Sclerosis - ID: D000007249 - Term: Inflammation ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01497379 **Brief Title:** Safety & Efficacy of Subretinal Implants for Partial Restoration of Vision in Blind Patients **Official Title:** Safety & Efficacy of Subretinal Implants for Partial Restoration of Vision in Blind Patients: A Prospective Mono- & Multicenter Clinical Study Based on Randomized Intra-individual Implant Activation in Degenerative Retinal Disease Patients. #### Organization Study ID Info **ID:** HKCTR-1198 #### Organization **Class:** INDUSTRY **Full Name:** Retina Implant AG #### Secondary ID Infos **Domain:** Clinicaltrials.gov **ID:** RI-MC-CT-2009 **Type:** REGISTRY ### Status Module #### Completion Date **Date:** 2015-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-03-29 **Type:** ACTUAL **Last Update Submit Date:** 2017-03-28 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2014-01 **Type:** ACTUAL #### Start Date **Date:** 2011-10 **Status Verified Date:** 2017-03 #### Study First Post Date **Date:** 2011-12-22 **Type:** ESTIMATED **Study First Submit Date:** 2011-12-15 **Study First Submit QC Date:** 2011-12-19 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Retina Implant AG #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** Patients who are legally blind, caused by retinal degeneration of photoreceptor rods \& cones (e.g. Retinitis pigmentosa), receive a subretinal implant to restore vision partially. ### Conditions Module **Conditions:** - Retinal Degeneration - Retinitis Pigmentosa **Keywords:** - Hereditary retinal - degeneration outer - retinal layers - photoreceptor rods cones - Retinitis pigmentosa - blindness - reading ability - retina implant - subretinal ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** SINGLE **Who Masked:** - PARTICIPANT **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 2 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** intra-individual implant activation **Intervention Names:** - Procedure: surgical implantation of subretinal device **Label:** intra-individual implant ON **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** intra-individual implant deactivation **Intervention Names:** - Procedure: surgical implantation of subretinal device **Label:** intra-individual implant OFF **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - intra-individual implant ON **Description:** surgical implantation of subretinal device **Name:** surgical implantation of subretinal device **Other Names:** - Retinal implant, subretinal implant **Type:** PROCEDURE #### Intervention 2 **Arm Group Labels:** - intra-individual implant OFF **Description:** intra-individual implant OFF **Name:** surgical implantation of subretinal device **Other Names:** - Retinal implant, subretinal implant **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Description:** treatment shows no permanent damage of function and structures that have been functional before surgery and no permanent damage to health and/or well being of patients **Measure:** Safety **Time Frame:** 1 year **Description:** Activities of Daily Living \& Mobility are significantly improved with implant-ON versus OFF, as shown via tests: * Activities of Daily Living tasks or * Recognition tasks or * Mobility or * a combination of the above **Measure:** Efficacy **Time Frame:** 1 year #### Secondary Outcomes **Description:** Patient long term safety: * stability of implant function * stability of body structure \& function related to implant system **Measure:** Safety **Time Frame:** 1 year **Description:** Visual Acuity / Light-perception and/or Object-recognition are significantly improved with implant-ON versus OFF as shown via: * FrACT or * BaLM or * Grating test (e.g. BaGA) and/or Quality of life * Quality of life (questionnaire) or * a combination of the above **Measure:** Efficacy **Time Frame:** 1 year ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Hereditary retinal degeneration of the outer retinal layers i.e. photoreceptor rods \& cones. * Pseudophakia * Angiography shows retinal vessels adequately perfused, despite pathological RP condition. * Age between 18 and 78 years. * Blindness (at least monocular) i.e. visual functions not appropriate for localization of objects, self sustained navigation and orientation (impaired light localization or worse). * Ability to read normal print in earlier life, optically corrected without magnifying glass. * Willing and able to give written informed consent in accordance to EN ISO 14155 (section 6.7) and local legislation prior to participation in the study. Able to perform the study during the full time period of one year Exclusion Criteria: * Period of appropriate visual functions \< 12 years / lifetime. * Optical Coherence Tomography (OCT) shows significant retina edema \&/or scar tissue within target region for implant. * Retina detected as too thin to expect required rest-functionality of inner retina as shown via Optical Coherence Tomography (OCT). * Lack of inner-retinal function, as determined by Electrically Evoked Phosphenes (EEP). * Heavy clumped pigmentation at posterior pole * Any other ophthalmologic disease with relevant effect upon visual function (e.g. glaucoma, optic neuropathies, trauma, diabetic retinopathy, retinal detachment). * Amblyopia reported earlier in life on eye to be implanted * Systemic diseases that might imply considerable risks with regard to the surgical interventions and anaesthesia (e.g. cardiovascular/ pulmonary diseases, severe metabolic diseases). * Neurological and/or psychiatric diseases (e.g. M. Parkinson, epilepsy, depression). * Hyperthyroidism or hypersensitivity to iodine * Women who are pregnant or nursing, or women of childbearing potential who are not willing to use a medically acceptable means of birth control for the duration of the study, or women unwilling to perform a pregnancy test before entering the study. * Participation in another interventional clinical trial within the past 30 days. **Maximum Age:** 78 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Hong Kong **Country:** Hong Kong **Facility:** Ophthalmology Eye Institute, University of Hong Kong **State:** Cyberport **Zip:** 100 #### Overall Officials **Official 1:** **Affiliation:** Chair Professor in Ophthalmology Eye Institute **Name:** David Wong, Prof., MD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Stingl K, Bartz-Schmidt KU, Besch D, Chee CK, Cottriall CL, Gekeler F, Groppe M, Jackson TL, MacLaren RE, Koitschev A, Kusnyerik A, Neffendorf J, Nemeth J, Naeem MA, Peters T, Ramsden JD, Sachs H, Simpson A, Singh MS, Wilhelm B, Wong D, Zrenner E. Subretinal Visual Implant Alpha IMS--Clinical trial interim report. Vision Res. 2015 Jun;111(Pt B):149-60. doi: 10.1016/j.visres.2015.03.001. Epub 2015 Mar 23. **PMID:** 25812924 #### See Also Links **Label:** Hong Kong clinical trials registry **URL:** http://www.hkclinicaltrials.com/ ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012164 - Term: Retinal Diseases - ID: D000005128 - Term: Eye Diseases - ID: D000015785 - Term: Eye Diseases, Hereditary - ID: D000058499 - Term: Retinal Dystrophies - ID: D000030342 - Term: Genetic Diseases, Inborn ### Condition Browse Module - Browse Branches - Abbrev: BC11 - Name: Eye Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M14999 - Name: Retinal Diseases - Relevance: LOW - As Found: Unknown - ID: M5047 - Name: Blindness - Relevance: LOW - As Found: Unknown - ID: M15008 - Name: Retinitis - Relevance: HIGH - As Found: Retinitis - ID: M15009 - Name: Retinitis Pigmentosa - Relevance: HIGH - As Found: Retinitis Pigmentosa - ID: M14997 - Name: Retinal Degeneration - Relevance: HIGH - As Found: Retinal Degeneration - ID: M8271 - Name: Eye Diseases - Relevance: LOW - As Found: Unknown - ID: M18339 - Name: Eye Diseases, Hereditary - Relevance: LOW - As Found: Unknown - ID: M29107 - Name: Retinal Dystrophies - Relevance: LOW - As Found: Unknown - ID: M23686 - Name: Genetic Diseases, Inborn - Relevance: LOW - As Found: Unknown - ID: T4945 - Name: Retinitis Pigmentosa - Relevance: HIGH - As Found: Retinitis Pigmentosa ### Condition Browse Module - Meshes - ID: D000012173 - Term: Retinitis - ID: D000012174 - Term: Retinitis Pigmentosa - ID: D000012162 - Term: Retinal Degeneration ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04756479 **Acronym:** COVID-AGEBRU **Brief Title:** Mortality Due to COVID-19 in the COVID-AGEBRU Study **Official Title:** Predictors of Intrahospital Mortality in Older Patients Admitted Due to COVID-19 in the COVID-19 AGEBRU Study #### Organization Study ID Info **ID:** COVID-19 AGE-BRUGMANN 2020/111 #### Organization **Class:** OTHER **Full Name:** Brugmann University Hospital ### Status Module #### Completion Date **Date:** 2020-06-30 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2022-04-06 **Type:** ACTUAL **Last Update Submit Date:** 2022-03-28 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2020-06-30 **Type:** ACTUAL #### Start Date **Date:** 2020-03-01 **Type:** ACTUAL **Status Verified Date:** 2022-03 #### Study First Post Date **Date:** 2021-02-16 **Type:** ACTUAL **Study First Submit Date:** 2021-02-08 **Study First Submit QC Date:** 2021-02-15 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Brugmann University Hospital #### Responsible Party **Investigator Affiliation:** Brugmann University Hospital **Investigator Full Name:** Murielle Surquin **Investigator Title:** Geriatrics Department **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The COVID-Age Brugmann study aims to identify the clinical predictors of mortality risk in older patients admitted to an acute care unit due to COVID-19 **Detailed Description:** The COVID-19 disease has been shown to be associated to higher mortality risk and health adverse consequences. Despite of the fact that the SAR-COV2 virus has been shown that affects every populations, the negative impact is higher in older people, pointing this population as the most severely affected. The COVID-Age Brugmann study aims to identify if comorbidity, geriatric syndromes, and health issues at admission are associated to mortality risk in older patients admitted to an acute care unit due to COVID-19 ### Conditions Module **Conditions:** - Covid19 - Old Age; Debility - Morbidity, Multiple ### Design Module #### Design Info **Observational Model:** CASE_ONLY **Time Perspective:** RETROSPECTIVE #### Enrollment Info **Count:** 160 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Outcomes Module #### Primary Outcomes **Description:** Vital status was obtained from medical records **Measure:** All-cause mortality during hospital admission (admission up to discharge time) **Time Frame:** Through study completion, an average of one month. #### Secondary Outcomes **Description:** Clinical and demographical conditions, and geriatric syndromes **Measure:** Prevalence of medical diseases and geriatric syndromes **Time Frame:** Through study completion, an average of one month. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patients aged 70 and older * Patients admitted to an acute care geriatric unit due to COVID-19 * Patients with COVID-19 by PCR, serology or CT scan suggestive of this disease Exclusion Criteria: * No other demographic or clinical exclusion criteria will be applied **Minimum Age:** 70 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - OLDER_ADULT **Study Population:** Consecutive patients aged 70 and over hospitalized in an acute care unit in Brugmann university hospital, in Belgium, due to COVID-19 in the period Mars to June 2020. ### Contacts Locations Module #### Locations **Location 1:** **City:** Brussels **Country:** Belgium **Facility:** CHU Brugmann **Zip:** 1020 #### Overall Officials **Official 1:** **Affiliation:** CHU Brugmann, Director of the Geriatrics Department **Name:** Murielle MS Surquin, MD PhD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Mendes A, Serratrice C, Herrmann FR, Genton L, Perivier S, Scheffler M, Fassier T, Huber P, Jacques MC, Prendki V, Roux X, Di Silvestro K, Trombert V, Harbarth S, Gold G, Graf CE, Zekry D. Predictors of In-Hospital Mortality in Older Patients With COVID-19: The COVIDAge Study. J Am Med Dir Assoc. 2020 Nov;21(11):1546-1554.e3. doi: 10.1016/j.jamda.2020.09.014. Epub 2020 Sep 15. **PMID:** 33138936 **Citation:** Menager P, Briere O, Gautier J, Riou J, Sacco G, Brangier A, Annweiler C, Geria-Covid Study Group OBOT. Regular Use of VKA Prior to COVID-19 Associated with Lower 7-Day Survival in Hospitalized Frail Elderly COVID-19 Patients: The GERIA-COVID Cohort Study. Nutrients. 2020 Dec 24;13(1):39. doi: 10.3390/nu13010039. **PMID:** 33374341 **Citation:** Miralles O, Sanchez-Rodriguez D, Marco E, Annweiler C, Baztan A, Betancor E, Cambra A, Cesari M, Fontecha BJ, Gasowski J, Gillain S, Hope S, Phillips K, Piotrowicz K, Piro N, Sacco G, Saporiti E, Surquin M, Vall-Llosera E. Unmet needs, health policies, and actions during the COVID-19 pandemic: a report from six European countries. Eur Geriatr Med. 2021 Feb;12(1):193-204. doi: 10.1007/s41999-020-00415-x. Epub 2020 Oct 15. Erratum In: Eur Geriatr Med. 2021 Jun;12(3):667. **PMID:** 33057981 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000011024 - Term: Pneumonia, Viral - ID: D000011014 - Term: Pneumonia - ID: D000012141 - Term: Respiratory Tract Infections - ID: D000007239 - Term: Infections - ID: D000014777 - Term: Virus Diseases - ID: D000018352 - Term: Coronavirus Infections - ID: D000003333 - Term: Coronaviridae Infections - ID: D000030341 - Term: Nidovirales Infections - ID: D000012327 - Term: RNA Virus Infections - ID: D000008171 - Term: Lung Diseases - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC01 - Name: Infections - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M2561 - Name: COVID-19 - Relevance: HIGH - As Found: COVID-19 - ID: M1175 - Name: Frailty - Relevance: HIGH - As Found: Debility - ID: M13904 - Name: Pneumonia - Relevance: LOW - As Found: Unknown - ID: M13914 - Name: Pneumonia, Viral - Relevance: LOW - As Found: Unknown - ID: M10283 - Name: Infections - Relevance: LOW - As Found: Unknown - ID: M6368 - Name: Communicable Diseases - Relevance: LOW - As Found: Unknown - ID: M14978 - Name: Respiratory Tract Infections - Relevance: LOW - As Found: Unknown - ID: M17522 - Name: Virus Diseases - Relevance: LOW - As Found: Unknown - ID: M20490 - Name: Coronavirus Infections - Relevance: LOW - As Found: Unknown - ID: M6555 - Name: Coronaviridae Infections - Relevance: LOW - As Found: Unknown - ID: M23685 - Name: Nidovirales Infections - Relevance: LOW - As Found: Unknown - ID: M15149 - Name: RNA Virus Infections - Relevance: LOW - As Found: Unknown - ID: M11168 - Name: Lung Diseases - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000086382 - Term: COVID-19 - ID: D000073496 - Term: Frailty ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02312479 **Brief Title:** Safety and Performance Study of the Nyxoah SAT System for Treating OSA **Official Title:** Safety and Performance Study of the Use of the Nyxoah SAT System for the Treatment of Obstructive Sleep Apnea #### Organization Study ID Info **ID:** SAT2014A #### Organization **Class:** INDUSTRY **Full Name:** Nyxoah S.A. ### Status Module #### Completion Date **Date:** 2016-04 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2016-09-27 **Type:** ESTIMATED **Last Update Submit Date:** 2016-09-26 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2015-11 **Type:** ACTUAL #### Start Date **Date:** 2014-12 **Status Verified Date:** 2016-09 #### Study First Post Date **Date:** 2014-12-09 **Type:** ESTIMATED **Study First Submit Date:** 2014-12-04 **Study First Submit QC Date:** 2014-12-04 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Nyxoah S.A. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** A prospective open-label, single treatment study to assess the safety and the performance of the Nyxoah SAT system for the treatment of Obstructive Sleep Apnea ### Conditions Module **Conditions:** - Obstructive Sleep Apnea ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 6 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Device: Nyxoah SAT system **Label:** Nyxoah SAT therapy **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Nyxoah SAT therapy **Description:** The Nyxoah SAT system is comprised of an implantable nerve stimulator implanted over one of the tongue muscles via a minimally invasive procedure. Stimulation of the Hypoglossal nerves causes the tongue muscles to contract, thus maintaining an open airway during sleep. **Name:** Nyxoah SAT system **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Measure:** Incidence of serious device related adverse events **Time Frame:** 6-months post-implantation **Measure:** Mean change of AHI (Apnea-Hypopnea Index) measured by in-lab polysomnography (PSG) from baseline measurement to 6-months post-implantation **Time Frame:** 6-months post-implantation ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Diagnosed with moderate to severe Obstructive Sleep Apnea * Have failed or have not tolerated CPAP treatment * Willing and capable of providing informed consent * Willing and capable of returning to all follow-up visits and sleep studies, including evaluation procedures and filling out questionnaires Exclusion Criteria: * BMI limits * Subjects with complete concentric collapse at the soft palate level per endoscopy **Maximum Age:** 70 Years **Minimum Age:** 21 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Edegem **Country:** Belgium **Facility:** Antwerp University Hospital **Location 2:** **City:** Mannheim **Country:** Germany **Facility:** Universitäts-HNO-Klinik Mannheim #### Overall Officials **Official 1:** **Affiliation:** Universitäts-HNO-Klinik Mannheim **Name:** Joachim T Maurer, OA Dr. med. **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** Universiteit Antwerpen **Name:** Evert Hamans, PhD Dr. **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000001049 - Term: Apnea - ID: D000012120 - Term: Respiration Disorders - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000020919 - Term: Sleep Disorders, Intrinsic - ID: D000020920 - Term: Dyssomnias - ID: D000012893 - Term: Sleep Wake Disorders - ID: D000009422 - Term: Nervous System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BXM - Name: Behaviors and Mental Disorders ### Condition Browse Module - Browse Leaves - ID: M4361 - Name: Apnea - Relevance: LOW - As Found: Unknown - ID: M15694 - Name: Sleep Apnea Syndromes - Relevance: HIGH - As Found: Sleep Apnea - ID: M22010 - Name: Sleep Apnea, Obstructive - Relevance: HIGH - As Found: Obstructive Sleep Apnea - ID: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M14957 - Name: Respiration Disorders - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown - ID: M22242 - Name: Parasomnias - Relevance: LOW - As Found: Unknown - ID: M22654 - Name: Sleep Disorders, Intrinsic - Relevance: LOW - As Found: Unknown - ID: M22655 - Name: Dyssomnias - Relevance: LOW - As Found: Unknown - ID: M15696 - Name: Sleep Wake Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000012891 - Term: Sleep Apnea Syndromes - ID: D000020181 - Term: Sleep Apnea, Obstructive ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03786679 **Acronym:** NOSWEPH **Brief Title:** Non-operative Treatment in Sweden of Proximal Humeral Fractures **Official Title:** Non-operative Treatment in Sweden of Proximal Humeral Fratures, a Randomised Multicenter Trial. #### Organization Study ID Info **ID:** 06000836 #### Organization **Class:** OTHER_GOV **Full Name:** Linkoeping University ### Status Module #### Completion Date **Date:** 2024-02-25 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-03-08 **Type:** ACTUAL **Last Update Submit Date:** 2023-03-07 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-02-25 **Type:** ESTIMATED #### Start Date **Date:** 2019-02-25 **Type:** ACTUAL **Status Verified Date:** 2023-03 #### Study First Post Date **Date:** 2018-12-26 **Type:** ACTUAL **Study First Submit Date:** 2018-11-05 **Study First Submit QC Date:** 2018-12-23 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER_GOV **Name:** Lars Adolfsson #### Responsible Party **Investigator Affiliation:** Linkoeping University **Investigator Full Name:** Lars Adolfsson **Investigator Title:** Professor **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Proximal humeral fractures are common especially in the elderly population. The majority of these fractures are minimally displaced and may be treated non-operatively. There is however a controversy about which fractures that need surgery and randomised trials have not been able to show a clinically important advantage in patient reported outcome measures for those operated. The trend is therefore that also displaced and comminute fractures are treated non-operatively. There is however very little scientific support for how the non-operative treatment should be designed and performed. Therefore this prospective multicenter study is aiming at investigating the benefit of a four week immobilisation orthosis as compared to early range of motion exercises for those patients not assigned for surgery one week after the trauma. ### Conditions Module **Conditions:** - Proximal Humeral Fracture **Keywords:** - Non-operative treatment, orthosis, healing, rehabilitation ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** Parallel assignment ##### Masking Info **Masking:** SINGLE **Masking Description:** The assessors are not involved in the treatment and the radiological assessment of images will be assessed with anonymyized images. **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 400 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** An orthosis with the broken arm in neutral position fixed for four weeks. After these four weeks the patient is instructed to start rehabilitation. **Intervention Names:** - Device: Ultrasling ER III orthosis **Label:** Orthosis group **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** The patient is instructed to start early rehabilitation about one week after the trauma. **Intervention Names:** - Device: Ultrasling ER III orthosis **Label:** Early rehabilitation group **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Early rehabilitation group - Orthosis group **Description:** Application of orthosis and start of rehabilitation after four weeks. **Name:** Ultrasling ER III orthosis **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** recording of fracture union on radiological images **Measure:** Union of fracture **Time Frame:** Followed 12 months #### Secondary Outcomes **Description:** Shoulder specific patient reported outcome measure, PROM, with a maximum score of 48 points **Measure:** Oxford shoulder score **Time Frame:** 12 months **Description:** Patient reported outcome measure of pain at rest, at night and during activity in scales with 10 steps grading subjective assessment of pain **Measure:** Numerical pain reporting scale **Time Frame:** 12 months **Description:** Patient reported outcome measure of shoulder function in a 11-item PROM **Measure:** Quick DASH **Time Frame:** 12 months **Description:** Patient rated assessment of global improvement in a numeric scale with 7 steps **Measure:** Global assessment of improvement **Time Frame:** 12 months **Description:** The elevation, abduction, internal and external rotation of the injured shoulder **Measure:** Shoulder range of motion **Time Frame:** 12 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * A proximal humeral fracture verified on radiology no older than 7-10 days. Exclusion Criteria: * Surgically treated proximal humeral fracture * Fracture only involving the greater tuberosity * Previous surgery in the fractured shoulder * Ongoing malignancy in the fractured shoulder * Neurologic disease * Radiating pain from the neck in the affected arm * Associated vascular or nerve injuries * Dementia * Alcohol abuse * Unwilling to participate in the trial **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Hanna C Björnsson Hallgren, MD, PhD **Phone:** 0046709473276 **Role:** CONTACT #### Locations **Location 1:** **City:** Linköping **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Hanna C Björnsson Hallgren, MD, PhD - **Phone:** +46101031000 - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** lars e adolfsson, MD, PhD - **Phone:** +46101031000 - **Role:** CONTACT **Country:** Sweden **Facility:** Lars Adolfsson **Status:** RECRUITING **Zip:** 58185 #### Overall Officials **Official 1:** **Affiliation:** Linkoeping University **Name:** Lars E Adolfsson, Professor **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** Linkoeping University **Name:** Hanna C Björnsson Hallgren, MD, PhD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Handoll HH, Elliott J, Thillemann TM, Aluko P, Brorson S. Interventions for treating proximal humeral fractures in adults. Cochrane Database Syst Rev. 2022 Jun 21;6(6):CD000434. doi: 10.1002/14651858.CD000434.pub5. **PMID:** 35727196 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000050723 - Term: Fractures, Bone - ID: D000014947 - Term: Wounds and Injuries - ID: D000001134 - Term: Arm Injuries - ID: D000070599 - Term: Shoulder Injuries ### Condition Browse Module - Browse Branches - Abbrev: BC26 - Name: Wounds and Injuries - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M26370 - Name: Fractures, Bone - Relevance: LOW - As Found: Unknown - ID: M9869 - Name: Humeral Fractures - Relevance: HIGH - As Found: Humeral Fractures - ID: M15592 - Name: Shoulder Fractures - Relevance: HIGH - As Found: Proximal Humeral Fracture - ID: M17685 - Name: Wounds and Injuries - Relevance: LOW - As Found: Unknown - ID: M4444 - Name: Arm Injuries - Relevance: LOW - As Found: Unknown - ID: M602 - Name: Shoulder Injuries - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006810 - Term: Humeral Fractures - ID: D000012784 - Term: Shoulder Fractures ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04236479 **Brief Title:** Mesenchymal Stromal Cells for Infants With Congenital Heart Disease (MedCaP) **Official Title:** Mesenchymal Stromal Cells Delivery Through Cardiopulmonary Bypass in Pediatric Cardiac Surgery #### Organization Study ID Info **ID:** Pro00011914 #### Organization **Class:** OTHER **Full Name:** Children's National Research Institute ### Status Module #### Completion Date **Date:** 2025-04 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-09-21 **Type:** ACTUAL **Last Update Submit Date:** 2023-09-19 **Overall Status:** ACTIVE_NOT_RECRUITING #### Primary Completion Date **Date:** 2024-09 **Type:** ESTIMATED #### Start Date **Date:** 2020-07-29 **Type:** ACTUAL **Status Verified Date:** 2023-09 #### Study First Post Date **Date:** 2020-01-22 **Type:** ACTUAL **Study First Submit Date:** 2020-01-15 **Study First Submit QC Date:** 2020-01-17 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Catherine Bollard #### Responsible Party **Investigator Affiliation:** Children's National Research Institute **Investigator Full Name:** Catherine Bollard **Investigator Title:** Director, Center for Cancer and Immunology/ Center for Emerging Technologies in Immune Cell Therapies (CETI) **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Oversight Has DMC:** True ### Description Module **Brief Summary:** The proposed study will be a prospective, open-label, single-center, safety and feasibility phase 1 trial of allogeneic bone marrow-derived mesenchymal stromal cell (BM-MSC) delivery though cardiopulmonary bypass (CPB) using a homogeneous population of infants with congenital heart disease (CHD) who will be undergoing a two-ventricle repair within the first six months of life **Detailed Description:** This study is a prospective, open-label, single-center, safety and feasibility phase 1 trial of allogeneic bone marrow-derived mesenchymal stromal cell (BM-MSC) delivery though cardiopulmonary bypass (CPB) using a homogeneous population of infants with congenital heart disease (CHD) who will be undergoing a two-ventricle repair within the first six months of life. The dose-escalation methods with a modified continual reassessment at the five dose levels (1x10\^6, 10x10\^6, 20x10\^6, 40x10\^6, 80x10\^6, cells/kg) will be performed to determine safety and feasibility of allogeneic BM-MSC infusion during pediatric cardiac surgery and the maximum tolerated dose in infants with CHD. In addition to the primary objective of assessing the safety and feasibility of BM-MSC delivery through CPB, our secondary objectives are designed to develop biological signature measures and clinical outcome measures feasible for use in larger efficacy and effectiveness trials with a particular focus on neurodevelopmental outcome and early postoperative course after BM-MSC treatment. We will determine actual magnitude of differences in neuroimaging and neurodevelopmental variables and postoperative inflammatory and pathophysiological variables after BM-MSC delivery in infants with CHD. Enrollment, follow-up, and analysis are planned to occur over 36 months for the treatment and initial follow-up portions of the study. Long-term follow-up until 18 months of age will be subsequently reported. ### Conditions Module **Conditions:** - Congenital Heart Disease (CHD) ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 17 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** The dose-escalation methods with a modified continual reassessment at the five dose levels (1x10\^6, 10x10\^6, 20x10\^6, 40x10\^6, 80x10\^6 cells/kg) will be performed to determine safety and feasibility of allogeneic BM-MSC infusion during pediatric cardiac surgery and the maximum tolerated dose in infants with CHD. **Intervention Names:** - Biological: BM-MSC **Label:** Bone marrow-derived mesenchymal stromal cell (BM-MSC) **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Bone marrow-derived mesenchymal stromal cell (BM-MSC) **Description:** Allogeneic bone marrow-derived mesenchymal stromal cell (BM-MSC) delivery through cardiopulmonary bypass (CPB) using a homogeneous population of infants with congenital heart disease (CHD) who will be undergoing a two-ventricle repair within the first six months of life. **Name:** BM-MSC **Type:** BIOLOGICAL ### Outcomes Module #### Primary Outcomes **Description:** Dose Limiting Toxicity is attributable to the MSC administration. **Measure:** Number of subjects who experience serious adverse events, adverse events, and/or early treatment discontinuations. **Time Frame:** 45 days following the MSC administration #### Secondary Outcomes **Description:** Secondary objective will be measured by using the Pediatric Cardiac Critical Care Consortium (PC4) registry system. **Measure:** Actual magnitude of differences in neuroimaging and neurodevelopmental variables will be measured after MSC delivery. **Time Frame:** 18 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Neonatal and young infantile patients who are ≤ 3 months of age * Scheduled to undergo reparative two-ventricle repair for congenital heart defects without aortic arch reconstruction, including the following: a. D-Transposition of the Great Arteries (d-TGA) Group: i. d-TGA with intact ventricular septum (d-TGA, IVS) ii. d-TGA with ventricular septal defect (d-TGA, VSD) b. Ventricular Septal Defect (VSD) Group: i. VSD without aortic arch obstruction (AAO) ii. Complete common atrioventricular canal defect (CAVC) c. Tetralogy of Fallot (TOF) Group: i. Tetralogy of Fallot (TOF) ii. Tetralogy of Fallot with Pulmonary Atresia (TOF,PA) iii. Truncus arteriosus (TA) iv. Double outlet right ventricle (DORV) * Scheduled surgery at or before three months of age. * Parent/guardian capable of providing informed consent. Exclusion Criteria: * Birth weight less than 2.0 kg * Recognizable phenotypic syndrome * Associated extracardiac anomalies of greater than minor severity * Previous cardiac surgery * Associated cardiovascular anomalies requiring aortic arch reconstruction and/or additional open cardiac surgical procedures in infancy * Prior severe hypoxic event * Significant screening test values that place subjects at increased risk of complications from participation in the study **Maximum Age:** 6 Months **Sex:** ALL **Standard Ages:** - CHILD ### Contacts Locations Module #### Locations **Location 1:** **City:** Washington **Country:** United States **Facility:** Children's National Health System **State:** District of Columbia **Zip:** 20010 #### Overall Officials **Official 1:** **Affiliation:** CNMC **Name:** Richard Jonas, MD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000018376 - Term: Cardiovascular Abnormalities - ID: D000000013 - Term: Congenital Abnormalities ### Condition Browse Module - Browse Branches - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth ### Condition Browse Module - Browse Leaves - ID: M9419 - Name: Heart Diseases - Relevance: HIGH - As Found: Heart Disease - ID: M9418 - Name: Heart Defects, Congenital - Relevance: HIGH - As Found: Congenital Heart Disease - ID: M12 - Name: Congenital Abnormalities - Relevance: LOW - As Found: Unknown - ID: M20503 - Name: Cardiovascular Abnormalities - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006331 - Term: Heart Diseases - ID: D000006330 - Term: Heart Defects, Congenital ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03235479 **Brief Title:** Safety and Efficacy Study in Adult Subjects With Acute Migraines **Official Title:** BHV3000-301: Phase 3: Double-Blind, Randomized, Placebo-Controlled, Safety and Efficacy Trial of BHV-3000 (Rimegepant) for the Acute Treatment of Migraine #### Organization Study ID Info **ID:** BHV-3000-301 #### Organization **Class:** INDUSTRY **Full Name:** Pfizer ### Status Module #### Completion Date **Date:** 2018-01-26 **Type:** ACTUAL #### Disp First Post Date **Date:** 2019-01-11 **Type:** ACTUAL **Disp First Submit Date:** 2019-01-09 **Disp First Submit QC Date:** 2019-01-09 #### Expanded Access Info #### Last Update Post Date **Date:** 2023-02-16 **Type:** ACTUAL **Last Update Submit Date:** 2023-02-14 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2018-01-21 **Type:** ACTUAL #### Results First Post Date **Date:** 2020-12-23 **Type:** ACTUAL **Results First Submit Date:** 2020-11-30 **Results First Submit QC Date:** 2020-11-30 #### Start Date **Date:** 2017-07-18 **Type:** ACTUAL **Status Verified Date:** 2023-02 #### Study First Post Date **Date:** 2017-08-01 **Type:** ACTUAL **Study First Submit Date:** 2017-07-14 **Study First Submit QC Date:** 2017-07-27 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Pfizer #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to compare the efficacy of BHV-3000 (rimegepant) versus placebo in subjects with Acute Migraines ### Conditions Module **Conditions:** - Migraine, With or Without Aura ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** Randomized Controlled Trial ##### Masking Info **Masking:** TRIPLE **Masking Description:** Double-blind to Sponsor, Investigator and Participant **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 1485 **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants were administered a single oral dose of 75 mg of rimegepant tablet on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **Intervention Names:** - Drug: Rimegepant **Label:** Rimegepant 75 mg **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants were administered a single oral dose of matching placebo tablet for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **Intervention Names:** - Drug: Placebo **Label:** Placebo **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Rimegepant 75 mg **Description:** 75 mg tablet QD **Name:** Rimegepant **Other Names:** - BHV-3000 **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Placebo **Description:** Placebo tablet to match rimegepant dose QD **Name:** Placebo **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the electronic diary (eDiary). Pain freedom was defined as pain level of none. **Measure:** Percentage of Participants With Freedom From Pain at 2 Hours Post-dose **Time Frame:** 2 hours post-dose **Description:** MBS was reported as nausea, photophobia, or phonophobia at migraine onset using the eDiary. Symptom status (absent, present) was assessed post-dose using the eDiary separately for nausea, photophobia, and phonophobia. Freedom from MBS was defined as MBS reported at onset that was absent post-dose. **Measure:** Percentage of Participants With Freedom From Most Bothersome Symptom (MBS) at 2 Hours Post-dose **Time Frame:** 2 hours post-dose #### Secondary Outcomes **Description:** Photophobia (sensitivity to light) status was measured as absent or present in the eDiary. Freedom from photophobia was defined as photophobia absent. **Measure:** Percentage of Participants With Freedom From Photophobia at 2 Hours Post-dose **Time Frame:** 2 hours post-dose **Description:** Phonophobia (sensitivity to sound) status was measured as absent or present in the eDiary. Freedom from phonophobia was defined as phonophobia absent. **Measure:** Percentage of Participants With Freedom From Phonophobia at 2 Hours Post-dose **Time Frame:** 2 hours post-dose **Description:** Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the eDiary. Pain relief was defined as pain level of none or mild. **Measure:** Percentage of Participants With Pain Relief at 2 Hours Post-dose **Time Frame:** 2 hours post-dose **Description:** Nausea status was measured as absent or present in the eDiary. Freedom from nausea was defined as nausea absent. **Measure:** Percentage of Participants With Freedom From Nausea at 2 Hours Post-dose **Time Frame:** 2 hours post-dose **Description:** Participants who did not experience relief of their migraine headache at the end of 2 hours after dosing with study medication (and after the 2-hour assessments had been completed on the eDiary) were permitted to use the following rescue medications: aspirin, ibuprofen, acetaminophen up to 1000 mg/day (this includes Excedrin Migraine), naproxen (or any other type of nonsteroidal anti-inflammatory drug), antiemetics (e.g., metoclopramide or promethazine), or baclofen. The participant's use of rescue medication was recorded by the participant in a paper diary. **Measure:** Percentage of Participants With Rescue Medication Use Within 24 Hours Post-dose **Time Frame:** 24 hours post-dose **Description:** Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the eDiary. Sustained pain freedom was defined as pain level of none at 2 hours up to 24 hours post-dose with no rescue medication use through 24 hours post-dose. **Measure:** Percentage of Participants With Sustained Pain Freedom From 2 to 24 Hours Post-dose **Time Frame:** From 2 hours up to 24 hours post-dose **Description:** Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the eDiary. Sustained pain relief was defined as pain level of none or mild at 2 hours up to 24 hours post-dose with no rescue medication use through 24 hours post-dose. **Measure:** Percentage of Participants With Sustained Pain Relief From 2 to 24 Hours Post-dose **Time Frame:** From 2 hours up to 24 hours post-dose **Description:** Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the eDiary. Sustained pain freedom was defined as pain level of none at 2 hours up to 48 hours post-dose with no rescue medication use through 48 hours post-dose. **Measure:** Percentage of Participants With Sustained Pain Freedom From 2 to 48 Hours Post-dose **Time Frame:** From 2 hours up to 48 hours post-dose **Description:** Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the eDiary. Sustained pain relief was defined as pain level of none or mild at 2 hours up to 48 hours post-dose with no rescue medication use through 48 hours post-dose. **Measure:** Percentage of Participants With Sustained Pain Relief From 2 to 48 Hours Post-dose **Time Frame:** From 2 hours up to 48 hours post-dose **Description:** Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the eDiary. Pain relapse was defined as pain level of mild, moderate, or severe after 2 hours up to 48 hours for the participants who were pain-free at 2 hours post-dose. **Measure:** Percentage of Participants With Pain Relapse From 2 to 48 Hours Post-dose **Time Frame:** From 2 hours up to 48 hours post-dose **Description:** Functional disability level was assessed in the eDiary on a 4-point scale: normal function, mild impairment, severe impairment, required bed rest. Freedom from functional disability was defined as normal function. **Measure:** Percentage of Participants With Freedom From Functional Disability at 2 Hours Post-dose **Time Frame:** 2 hours post-dose ### Eligibility Module **Eligibility Criteria:** Key Inclusion Criteria: 1. Patient has at least 1 year history of migraines (with or without aura), consistent with a diagnosis according to the International Classification of Headache Disorder, 3rd Edition, Beta version\[1\] including the following: * Not more than 8 attacks of moderate or severe intensity per month within last 3 months * Consistent migraine headaches of at least 2 migraine headache attacks of moderate or severe intensity in each of the 3 months prior to the Screening Visit and maintains this requirement during the Screening Period 2. Less than 15 days with headache (migraine or non-migraine) per month in each of the 3 months prior to the Screening Visit and maintains this requirement during the Screening Period 3. Patients on prophylactic migraine medication are permitted to remain on therapy provided they have been on a stable dose for at least 3 months prior to study entry. 4. Patients with contraindications for use of triptans may be included provided they meet all other study entry criteria. Key Exclusion Criteria: 1. Patient history of HIV disease 2. Patient history with current evidence of uncontrolled, unstable or recently diagnosed cardiovascular disease, such as ischemic heart disease, coronary artery vasospasm, and cerebral ischemia. Patients with Myocardial Infarction (MI), Acute Coronary Syndrome (ACS),Percutaneous Coronary Intervention (PCI), cardiac surgery, stroke or transient ischemic attack (TIA) during the 6 months prior to screening. 3. Uncontrolled hypertension (high blood pressure), or uncontrolled diabetes (however patients can be included who have stable hypertension and/or diabetes for 3 months prior to being enrolled) 4. Patient has a current diagnosis of major depression, other pain syndromes, psychiatric conditions (eg, schizophrenia), dementia, or significant neurological disorders (other than migraine) that, in the Investigator's opinion, might interfere with study assessments 5. Patient has a history of gastric, or small intestinal surgery, or has a disease that causes mal-absorption 6. The patient has a history or current evidence of any significant and/or unstable medical conditions (eg, history of congenital heart disease or arrhythmia, known suspected infection, hepatitis B or C, or cancer) that, in the investigator's opinion, would expose them to undue risk of a significant adverse event (AE) or interfere with assessments of safety or efficacy during the course of the trial 7. History of, treatment for, or evidence of, alcohol or drug abuse within the past 12 months or patients who have met DSM-V criteria for any significant substance use disorder within the past 12 months from the date of the screening visit. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Birmingham **Country:** United States **Facility:** Central Research Associates, Inc **State:** Alabama **Zip:** 35205 **Location 2:** **City:** Gilbert **Country:** United States **Facility:** Neurological Physicians of Arizona/Radiant Research Inc **State:** Arizona **Zip:** 85282 **Location 3:** **City:** Tempe **Country:** United States **Facility:** Clinical Research Consortium Arizona **State:** Arizona **Zip:** 85283 **Location 4:** **City:** Tucson **Country:** United States **Facility:** Radiant Research, Inc. **State:** Arizona **Zip:** 85712 **Location 5:** **City:** Little Rock **Country:** United States **Facility:** Woodland International Research Group, LLC **State:** Arkansas **Zip:** 72211 **Location 6:** **City:** Encino **Country:** United States **Facility:** Pharmacology Research Institute **State:** California **Zip:** 91316 **Location 7:** **City:** San Francisco **Country:** United States **Facility:** Optimus Medical Group **State:** California **Zip:** 94102 **Location 8:** **City:** Santa Monica **Country:** United States **Facility:** California Medical Clinic for Headache **State:** California **Zip:** 90404 **Location 9:** **City:** Walnut Creek **Country:** United States **Facility:** Diablo Clinical Research, Inc **State:** California **Zip:** 94598 **Location 10:** **City:** Hollywood **Country:** United States **Facility:** Qps Mra, Llc **State:** Florida **Zip:** 33024 **Location 11:** **City:** Lake City **Country:** United States **Facility:** Multi-Specialty Research Associates, Inc **State:** Florida **Zip:** 32055 **Location 12:** **City:** Miami **Country:** United States **Facility:** Qps Mra, Llc **State:** Florida **Zip:** 33143 **Location 13:** **City:** Miami **Country:** United States **Facility:** Advanced Pharma CR, LLC **State:** Florida **Zip:** 33147 **Location 14:** **City:** Orlando **Country:** United States **Facility:** Compass Research, LLC **State:** Florida **Zip:** 32806 **Location 15:** **City:** Ormond Beach **Country:** United States **Facility:** Ormond Medical Arts Pharmaceutical Research **State:** Florida **Zip:** 32174 **Location 16:** **City:** Savannah **Country:** United States **Facility:** Meridian Clinical Research **State:** Georgia **Zip:** 31406 **Location 17:** **City:** New Orleans **Country:** United States **Facility:** New Orleans Center for Clinical Research **State:** Louisiana **Zip:** 70119 **Location 18:** **City:** Boston **Country:** United States **Facility:** Boston Clinical Trials, Inc **State:** Massachusetts **Zip:** 02131 **Location 19:** **City:** Marlborough **Country:** United States **Facility:** Milford Emergency Associates, Inc. **State:** Massachusetts **Zip:** 01752 **Location 20:** **City:** Ann Arbor **Country:** United States **Facility:** Michigan Head Pain & Neurological Institute **State:** Michigan **Zip:** 48104 **Location 21:** **City:** Minneapolis **Country:** United States **Facility:** Clinical Research Institute, Inc **State:** Minnesota **Zip:** 55402 **Location 22:** **City:** Minneapolis **Country:** United States **Facility:** Clinical Research Institute **State:** Minnesota **Zip:** 55402 **Location 23:** **City:** Kansas City **Country:** United States **Facility:** The Center for Pharmaceutical Research **State:** Missouri **Zip:** 64114 **Location 24:** **City:** Saint Louis **Country:** United States **Facility:** Sundance Clinical Research, LLC **State:** Missouri **Zip:** 63141 **Location 25:** **City:** Norfolk **Country:** United States **Facility:** Meridian Clinical Research -Norfolk **State:** Nebraska **Zip:** 68701 **Location 26:** **City:** Omaha **Country:** United States **Facility:** Meridian Clinical Research, LLC **State:** Nebraska **Zip:** 68134 **Location 27:** **City:** Las Vegas **Country:** United States **Facility:** Clinical Research Consortium- Las Vegas **State:** Nevada **Zip:** 89119 **Location 28:** **City:** Berlin **Country:** United States **Facility:** Hassman Research Institute, LLC **State:** New Jersey **Zip:** 08009 **Location 29:** **City:** Brooklyn **Country:** United States **Facility:** SPRI Clinical Trials, LLC **State:** New York **Zip:** 11235 **Location 30:** **City:** Endwell **Country:** United States **Facility:** Regional Clinical Research, Inc. **State:** New York **Zip:** 13760 **Location 31:** **City:** Manlius **Country:** United States **Facility:** Central New York Clinical Research **State:** New York **Zip:** 13104 **Location 32:** **City:** New York **Country:** United States **Facility:** Fieve Clinical Research **State:** New York **Zip:** 10168 **Location 33:** **City:** Rochester **Country:** United States **Facility:** Rochester Clinical Research, Inc **State:** New York **Zip:** 14609 **Location 34:** **City:** Greensboro **Country:** United States **Facility:** PharmQuest, LLC **State:** North Carolina **Zip:** 27408 **Location 35:** **City:** Cincinnati **Country:** United States **Facility:** CTI Clinical Research Center **State:** Ohio **Zip:** 45227 **Location 36:** **City:** Portland **Country:** United States **Facility:** Oregon Center for Clinical Investigations, Inc **State:** Oregon **Zip:** 97214 **Location 37:** **City:** Philadelphia **Country:** United States **Facility:** Clinical Research of Philadelphia, LLC **State:** Pennsylvania **Zip:** 19114-1029 **Location 38:** **City:** Uniontown **Country:** United States **Facility:** Preferred Primary Care Physicians **State:** Pennsylvania **Zip:** 15401 **Location 39:** **City:** Warwick **Country:** United States **Facility:** Omega Medical Research **State:** Rhode Island **Zip:** 02886 **Location 40:** **City:** Mount Pleasant **Country:** United States **Facility:** Coastal Carolina Research Center **State:** South Carolina **Zip:** 29464 **Location 41:** **City:** Dakota Dunes **Country:** United States **Facility:** Meridian Clinical Research **State:** South Dakota **Zip:** 57409 **Location 42:** **City:** Austin **Country:** United States **Facility:** FutureSearch Trials of Neurology, LP **State:** Texas **Zip:** 78731 **Location 43:** **City:** Dallas **Country:** United States **Facility:** FutureSearch Trials of Neurology, LP **State:** Texas **Zip:** 75231 **Location 44:** **City:** Houston **Country:** United States **Facility:** Texas Center for Drug Development **State:** Texas **Zip:** 77081 **Location 45:** **City:** Salt Lake City **Country:** United States **Facility:** J.Lewis Research Inc / Foothill Family Clinic South **State:** Utah **Zip:** 84121 **Location 46:** **City:** South Jordan **Country:** United States **Facility:** J.Lewis Research Inc. / Jordan River Family Med **State:** Utah **Zip:** 84095 **Location 47:** **City:** Norfolk **Country:** United States **Facility:** Clinical Research Associates of Tidewater, Inc. **State:** Virginia **Zip:** 23507 **Location 48:** **City:** Virginia Beach **Country:** United States **Facility:** Tidewater Integrated Medical Research **State:** Virginia **Zip:** 23454 **Location 49:** **City:** Bellevue **Country:** United States **Facility:** Northwest Clinical Research Center **State:** Washington **Zip:** 98007 **Location 50:** **City:** Seattle **Country:** United States **Facility:** Seattle Women's:Health, Research & Gynecology **State:** Washington **Zip:** 98105 ## Document Section ### Large Document Module #### Large Docs - Date: 2018-01-23 - Filename: Prot_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: False - Label: Study Protocol - Size: 2402887 - Type Abbrev: Prot - Upload Date: 2020-11-25T01:19 - Date: 2018-06-20 - Filename: SAP_001.pdf - Has ICF: False - Has Protocol: False - Has SAP: True - Label: Statistical Analysis Plan - Size: 6275241 - Type Abbrev: SAP - Upload Date: 2020-11-25T01:20 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000051270 - Term: Headache Disorders, Primary - ID: D000020773 - Term: Headache Disorders - ID: D000001927 - Term: Brain Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M11852 - Name: Migraine Disorders - Relevance: HIGH - As Found: Migraine - ID: M9351 - Name: Headache - Relevance: LOW - As Found: Unknown - ID: M22529 - Name: Headache Disorders - Relevance: LOW - As Found: Unknown - ID: M26657 - Name: Headache Disorders, Primary - Relevance: LOW - As Found: Unknown - ID: M5204 - Name: Brain Diseases - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000008881 - Term: Migraine Disorders ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** The safety population, all enrolled participants who received at least 1 dose of rimegepant or placebo, was used to determine the number of participants at risk for SAEs and other AEs. #### Event Groups **Group ID:** EG000 **Title:** Rimegepant 75 mg **Deaths Num At Risk:** 546 **Description:** Participants were administered a single oral dose of 75 mg of rimegepant on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** EG000 **Other Num at Risk:** 546 **Serious Number Affected:** 2 **Serious Number At Risk:** 546 **Title:** Rimegepant 75 mg **Group ID:** EG001 **Title:** Placebo **Deaths Num At Risk:** 549 **Description:** Participants were administered a single oral dose of matching placebo for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** EG001 **Other Num at Risk:** 549 **Serious Number Affected:** 1 **Serious Number At Risk:** 549 **Title:** Placebo **Frequency Threshold:** 5 #### Serious Events **Term:** Acute Respiratory Failure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 20.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 546 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 549 **Term:** Pulmonary Embolism **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 20.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 546 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 549 **Term:** Colitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 20.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 546 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 549 **Num Events:** 1 **Time Frame:** Serious adverse events (SAEs) were collected from informed consent up to the end of study, and adverse events (AEs) were collected from randomization up to the end of the study (up to 52 days). ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 543 **Group ID:** BG001 **Value:** 541 **Group ID:** BG002 **Value:** 1084 **Units:** Participants ### Group **ID:** BG000 **Title:** Rimegepant 75 mg **Description:** Participants were administered a single oral dose of 75 mg of rimegepant on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. ### Group **ID:** BG001 **Title:** Placebo **Description:** Participants were administered a single oral dose of matching placebo for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 12.3286 **Value:** 41.945 #### Measurement **Group ID:** BG001 **Spread:** 12.1381 **Value:** 41.326 #### Measurement **Group ID:** BG002 **Spread:** 12.2322 **Value:** 41.636 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 464 #### Measurement **Group ID:** BG001 **Value:** 463 #### Measurement **Group ID:** BG002 **Value:** 927 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 79 #### Measurement **Group ID:** BG001 **Value:** 78 #### Measurement **Group ID:** BG002 **Value:** 157 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 58 #### Measurement **Group ID:** BG001 **Value:** 68 #### Measurement **Group ID:** BG002 **Value:** 126 **Category Title:** Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 485 #### Measurement **Group ID:** BG001 **Value:** 473 #### Measurement **Group ID:** BG002 **Value:** 958 **Category Title:** Not Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 3 #### Measurement **Group ID:** BG002 **Value:** 4 **Category Title:** American Indian or Alaska Native #### Measurement **Group ID:** BG000 **Value:** 6 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 13 **Category Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 2 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 2 **Category Title:** Native Hawaiian or Other Pacific Islander #### Measurement **Group ID:** BG000 **Value:** 107 #### Measurement **Group ID:** BG001 **Value:** 80 #### Measurement **Group ID:** BG002 **Value:** 187 **Category Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 417 #### Measurement **Group ID:** BG001 **Value:** 444 #### Measurement **Group ID:** BG002 **Value:** 861 **Category Title:** White #### Measurement **Group ID:** BG000 **Value:** 10 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 17 **Category Title:** More than one race #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 353 #### Measurement **Group ID:** BG001 **Value:** 358 #### Measurement **Group ID:** BG002 **Value:** 711 **Class Title:** Migraine without Aura #### Measurement **Group ID:** BG000 **Value:** 190 #### Measurement **Group ID:** BG001 **Value:** 183 #### Measurement **Group ID:** BG002 **Value:** 373 **Class Title:** Migraine with Aura ### Measure #### Measurement **Group ID:** BG000 **Value:** 90 #### Measurement **Group ID:** BG001 **Value:** 92 #### Measurement **Group ID:** BG002 **Value:** 182 **Category Title:** Yes #### Measurement **Group ID:** BG000 **Value:** 453 #### Measurement **Group ID:** BG001 **Value:** 449 #### Measurement **Group ID:** BG002 **Value:** 902 **Category Title:** No **Class Title:** **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Ethnicity (NIH/OMB) **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Race (NIH/OMB) **Unit of Measure:** Participants ### Measure 5 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Primary Migraine Type **Unit of Measure:** Participants ### Measure 6 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Randomization Strata, Prophylactic Migraine Medication Use **Unit of Measure:** Participants **Population Description:** The analysis was performed on modified intent to treat (mITT) participants, defined as those who were randomized only once, took study medication, had moderate to severe pain level at migraine onset, and who provided at least 1 post-dose efficacy measurement (i.e., after taking study medication). ## Results Section - More Information Module ### Certain Agreement **Restriction Type:** LTE60 **Restrictive Agreement:** True ### Point of Contact **Email:** [email protected] **Organization:** Biohaven Pharmaceuticals, Inc. **Phone:** 203-404-0410 **Title:** Chief Medical Officer ## Results Section - Outcome Measures Module ### Outcome Measure 1 #### Analysis **CI Lower Limit:** 0.5 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 9.3 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY **Other Analysis Description:** **P-Value:** 0.0298 **P-Value Comment:** **Parameter Type:** Risk Difference (RD) **Parameter Value:** 4.9 **Statistical Comment:** **Statistical Method:** Cochran-Mantel-Haenszel **Tested Non-Inferiority:** ### Outcome Measure 2 #### Analysis **CI Lower Limit:** 3.4 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 14.4 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY **Other Analysis Description:** **P-Value:** 0.0016 **P-Value Comment:** **Parameter Type:** Risk Difference (RD) **Parameter Value:** 8.9 **Statistical Comment:** **Statistical Method:** Cochran-Mantel-Haenszel **Tested Non-Inferiority:** ### Outcome Measure 3 #### Analysis **CI Lower Limit:** 4.4 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 15.9 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY **Other Analysis Description:** **P-Value:** 0.0005 **P-Value Comment:** **Parameter Type:** Risk Difference (RD) **Parameter Value:** 10.2 **Statistical Comment:** **Statistical Method:** Cochran-Mantel-Haenszel **Tested Non-Inferiority:** ### Outcome Measure 4 #### Analysis **CI Lower Limit:** 0.8 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 14.6 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY **Other Analysis Description:** **P-Value:** 0.0299 **P-Value Comment:** **Parameter Type:** Risk Difference (RD) **Parameter Value:** 7.7 **Statistical Comment:** **Statistical Method:** Cochran-Mantel-Haenszel **Tested Non-Inferiority:** ### Outcome Measure 5 #### Analysis **CI Lower Limit:** 4.4 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 16.2 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY **Other Analysis Description:** **P-Value:** 0.0006 **P-Value Comment:** **Parameter Type:** Risk Difference (RD) **Parameter Value:** 10.3 **Statistical Comment:** **Statistical Method:** Cochran-Mantel-Haenszel **Tested Non-Inferiority:** ### Outcome Measure 6 #### Analysis **CI Lower Limit:** -2.4 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 12.9 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY **Other Analysis Description:** **P-Value:** 0.1815 **P-Value Comment:** P-value ≥ 0.05; therefore, all secondary outcome measures listed after this outcome measure in the hierarchy were not tested. **Parameter Type:** Risk Difference (RD) **Parameter Value:** 5.2 **Statistical Comment:** **Statistical Method:** Cochran-Mantel-Haenszel **Tested Non-Inferiority:** ### Outcome Measure 7 ### Outcome Measure 8 ### Outcome Measure 9 ### Outcome Measure 10 ### Outcome Measure 11 ### Outcome Measure 12 ### Outcome Measure 13 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 15.8 - **Spread:** - **Upper Limit:** 22.5 - **Value:** 19.2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 11.3 - **Spread:** - **Upper Limit:** 17.2 - **Value:** 14.2 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 32.6 - **Spread:** - **Upper Limit:** 40.7 - **Value:** 36.6 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 24.0 - **Spread:** - **Upper Limit:** 31.5 - **Value:** 27.7 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 30.6 - **Spread:** - **Upper Limit:** 39.2 - **Value:** 34.9 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 20.9 - **Spread:** - **Upper Limit:** 28.6 - **Value:** 24.8 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 33.4 - **Spread:** - **Upper Limit:** 43.7 - **Value:** 38.6 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 26.2 - **Spread:** - **Upper Limit:** 35.5 - **Value:** 30.9 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 51.8 - **Spread:** - **Upper Limit:** 60.2 - **Value:** 56.0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 41.5 - **Spread:** - **Upper Limit:** 49.9 - **Value:** 45.7 **Title:** #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 41.4 - **Spread:** - **Upper Limit:** 52.3 - **Value:** 46.9 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 36.2 - **Spread:** - **Upper Limit:** 47.0 - **Value:** 41.6 **Title:** #### Outcome Measure 7 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 17.1 - **Spread:** - **Upper Limit:** 23.8 - **Value:** 20.4 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 27.9 - **Spread:** - **Upper Limit:** 35.6 - **Value:** 31.8 **Title:** #### Outcome Measure 8 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 11.1 - **Spread:** - **Upper Limit:** 16.9 - **Value:** 14.0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 5.8 - **Spread:** - **Upper Limit:** 10.4 - **Value:** 8.1 **Title:** #### Outcome Measure 9 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 34.8 - **Spread:** - **Upper Limit:** 43.0 - **Value:** 38.9 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 24.2 - **Spread:** - **Upper Limit:** 31.7 - **Value:** 27.9 **Title:** #### Outcome Measure 10 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 8.9 - **Spread:** - **Upper Limit:** 14.3 - **Value:** 11.6 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 5.0 - **Spread:** - **Upper Limit:** 9.4 - **Value:** 7.2 **Title:** #### Outcome Measure 11 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 29.7 - **Spread:** - **Upper Limit:** 37.7 - **Value:** 33.7 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 20.3 - **Spread:** - **Upper Limit:** 27.4 - **Value:** 23.9 **Title:** #### Outcome Measure 12 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 30.6 - **Spread:** - **Upper Limit:** 49.6 - **Value:** 40.1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 39.0 - **Spread:** - **Upper Limit:** 61.0 - **Value:** 50.0 **Title:** #### Outcome Measure 13 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 29.4 - **Spread:** - **Upper Limit:** 37.3 - **Value:** 33.3 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 18.4 - **Spread:** - **Upper Limit:** 25.3 - **Value:** 21.8 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the electronic diary (eDiary). Pain freedom was defined as pain level of none. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** The analysis was performed on modified intent to treat (mITT) participants. **Reporting Status:** POSTED **Time Frame:** 2 hours post-dose **Title:** Percentage of Participants With Freedom From Pain at 2 Hours Post-dose **Type:** PRIMARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants were administered a single oral dose of 75 mg of rimegepant on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG000 **Title:** Rimegepant 75 mg ##### Group **Description:** Participants were administered a single oral dose of matching placebo for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG001 **Title:** Placebo #### Outcome Measure 2 **Description:** MBS was reported as nausea, photophobia, or phonophobia at migraine onset using the eDiary. Symptom status (absent, present) was assessed post-dose using the eDiary separately for nausea, photophobia, and phonophobia. Freedom from MBS was defined as MBS reported at onset that was absent post-dose. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** The analysis was performed on mITT participants. **Reporting Status:** POSTED **Time Frame:** 2 hours post-dose **Title:** Percentage of Participants With Freedom From Most Bothersome Symptom (MBS) at 2 Hours Post-dose **Type:** PRIMARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants were administered a single oral dose of 75 mg of rimegepant on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG000 **Title:** Rimegepant 75 mg ##### Group **Description:** Participants were administered a single oral dose of matching placebo for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG001 **Title:** Placebo #### Outcome Measure 3 **Description:** Photophobia (sensitivity to light) status was measured as absent or present in the eDiary. Freedom from photophobia was defined as photophobia absent. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** The analysis was performed on mITT participants with photophobia present at migraine onset. **Reporting Status:** POSTED **Time Frame:** 2 hours post-dose **Title:** Percentage of Participants With Freedom From Photophobia at 2 Hours Post-dose **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants were administered a single oral dose of 75 mg of rimegepant on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG000 **Title:** Rimegepant 75 mg ##### Group **Description:** Participants were administered a single oral dose of matching placebo for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG001 **Title:** Placebo #### Outcome Measure 4 **Description:** Phonophobia (sensitivity to sound) status was measured as absent or present in the eDiary. Freedom from phonophobia was defined as phonophobia absent. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** The analysis was performed on mITT participants with phonophobia present at migraine onset. **Reporting Status:** POSTED **Time Frame:** 2 hours post-dose **Title:** Percentage of Participants With Freedom From Phonophobia at 2 Hours Post-dose **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants were administered a single oral dose of 75 mg of rimegepant on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG000 **Title:** Rimegepant 75 mg ##### Group **Description:** Participants were administered a single oral dose of matching placebo for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG001 **Title:** Placebo #### Outcome Measure 5 **Description:** Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the eDiary. Pain relief was defined as pain level of none or mild. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** The analysis was performed on mITT participants. **Reporting Status:** POSTED **Time Frame:** 2 hours post-dose **Title:** Percentage of Participants With Pain Relief at 2 Hours Post-dose **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants were administered a single oral dose of 75 mg of rimegepant on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG000 **Title:** Rimegepant 75 mg ##### Group **Description:** Participants were administered a single oral dose of matching placebo for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG001 **Title:** Placebo #### Outcome Measure 6 **Description:** Nausea status was measured as absent or present in the eDiary. Freedom from nausea was defined as nausea absent. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** The analysis was performed on mITT participants with nausea present at migraine onset. **Reporting Status:** POSTED **Time Frame:** 2 hours post-dose **Title:** Percentage of Participants With Freedom From Nausea at 2 Hours Post-dose **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants were administered a single oral dose of 75 mg of rimegepant on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG000 **Title:** Rimegepant 75 mg ##### Group **Description:** Participants were administered a single oral dose of matching placebo for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG001 **Title:** Placebo #### Outcome Measure 7 **Description:** Participants who did not experience relief of their migraine headache at the end of 2 hours after dosing with study medication (and after the 2-hour assessments had been completed on the eDiary) were permitted to use the following rescue medications: aspirin, ibuprofen, acetaminophen up to 1000 mg/day (this includes Excedrin Migraine), naproxen (or any other type of nonsteroidal anti-inflammatory drug), antiemetics (e.g., metoclopramide or promethazine), or baclofen. The participant's use of rescue medication was recorded by the participant in a paper diary. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** The analysis was performed on mITT participants. **Reporting Status:** POSTED **Time Frame:** 24 hours post-dose **Title:** Percentage of Participants With Rescue Medication Use Within 24 Hours Post-dose **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants were administered a single oral dose of 75 mg of rimegepant on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG000 **Title:** Rimegepant 75 mg ##### Group **Description:** Participants were administered a single oral dose of matching placebo for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG001 **Title:** Placebo #### Outcome Measure 8 **Description:** Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the eDiary. Sustained pain freedom was defined as pain level of none at 2 hours up to 24 hours post-dose with no rescue medication use through 24 hours post-dose. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** The analysis was performed on mITT participants. **Reporting Status:** POSTED **Time Frame:** From 2 hours up to 24 hours post-dose **Title:** Percentage of Participants With Sustained Pain Freedom From 2 to 24 Hours Post-dose **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants were administered a single oral dose of 75 mg of rimegepant on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG000 **Title:** Rimegepant 75 mg ##### Group **Description:** Participants were administered a single oral dose of matching placebo for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG001 **Title:** Placebo #### Outcome Measure 9 **Description:** Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the eDiary. Sustained pain relief was defined as pain level of none or mild at 2 hours up to 24 hours post-dose with no rescue medication use through 24 hours post-dose. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** The analysis was performed on mITT participants. **Reporting Status:** POSTED **Time Frame:** From 2 hours up to 24 hours post-dose **Title:** Percentage of Participants With Sustained Pain Relief From 2 to 24 Hours Post-dose **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants were administered a single oral dose of 75 mg of rimegepant on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG000 **Title:** Rimegepant 75 mg ##### Group **Description:** Participants were administered a single oral dose of matching placebo for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG001 **Title:** Placebo #### Outcome Measure 10 **Description:** Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the eDiary. Sustained pain freedom was defined as pain level of none at 2 hours up to 48 hours post-dose with no rescue medication use through 48 hours post-dose. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** The analysis was performed on mITT participants. **Reporting Status:** POSTED **Time Frame:** From 2 hours up to 48 hours post-dose **Title:** Percentage of Participants With Sustained Pain Freedom From 2 to 48 Hours Post-dose **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants were administered a single oral dose of 75 mg of rimegepant on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG000 **Title:** Rimegepant 75 mg ##### Group **Description:** Participants were administered a single oral dose of matching placebo for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG001 **Title:** Placebo #### Outcome Measure 11 **Description:** Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the eDiary. Sustained pain relief was defined as pain level of none or mild at 2 hours up to 48 hours post-dose with no rescue medication use through 48 hours post-dose. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** The analysis was performed on mITT participants. **Reporting Status:** POSTED **Time Frame:** From 2 hours up to 48 hours post-dose **Title:** Percentage of Participants With Sustained Pain Relief From 2 to 48 Hours Post-dose **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants were administered a single oral dose of 75 mg of rimegepant on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG000 **Title:** Rimegepant 75 mg ##### Group **Description:** Participants were administered a single oral dose of matching placebo for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG001 **Title:** Placebo #### Outcome Measure 12 **Description:** Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the eDiary. Pain relapse was defined as pain level of mild, moderate, or severe after 2 hours up to 48 hours for the participants who were pain-free at 2 hours post-dose. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** The analysis population was performed on mITT participants with pain freedom at 2 hours post-dose. **Reporting Status:** POSTED **Time Frame:** From 2 hours up to 48 hours post-dose **Title:** Percentage of Participants With Pain Relapse From 2 to 48 Hours Post-dose **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants were administered a single oral dose of 75 mg of rimegepant on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG000 **Title:** Rimegepant 75 mg ##### Group **Description:** Participants were administered a single oral dose of matching placebo for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG001 **Title:** Placebo #### Outcome Measure 13 **Description:** Functional disability level was assessed in the eDiary on a 4-point scale: normal function, mild impairment, severe impairment, required bed rest. Freedom from functional disability was defined as normal function. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** The analysis was performed on mITT participants. **Reporting Status:** POSTED **Time Frame:** 2 hours post-dose **Title:** Percentage of Participants With Freedom From Functional Disability at 2 Hours Post-dose **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants were administered a single oral dose of 75 mg of rimegepant on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG000 **Title:** Rimegepant 75 mg ##### Group **Description:** Participants were administered a single oral dose of matching placebo for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** OG001 **Title:** Placebo ### Participant Flow Module #### Group **Description:** Participants were administered a single oral dose of 75 mg of rimegepant on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** FG000 **Title:** Rimegepant 75 mg #### Group **Description:** Participants were administered a single oral dose of matching placebo for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization. **ID:** FG001 **Title:** Placebo #### Period **Title:** Overall Study ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 8 ###### Reason **Group ID:** FG001 **Number of Subjects:** 11 ##### Withdraw **Type:** Not Experienced Moderate/Severe Migraine ###### Reason **Group ID:** FG000 **Number of Subjects:** 23 ###### Reason **Group ID:** FG001 **Number of Subjects:** 17 ##### Withdraw **Type:** Pregnancy ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ##### Withdraw **Type:** Protocol Deviation ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 3 ##### Withdraw **Type:** Technical Problems ###### Reason **Group ID:** FG000 **Number of Subjects:** 3 ###### Reason **Group ID:** FG001 **Number of Subjects:** 2 ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ###### Reason **Group ID:** FG001 **Number of Subjects:** 2 ##### Withdraw **Type:** Other Reasons ###### Reason **Group ID:** FG000 **Number of Subjects:** 3 ###### Reason **Group ID:** FG001 **Number of Subjects:** 5 ##### Milestone **Type:** STARTED **Comment:** Randomized ###### Achievement **Group ID:** FG000 **Number of Subjects:** 582 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 580 ##### Milestone **Type:** Treated ###### Achievement **Group ID:** FG000 **Number of Subjects:** 546 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 549 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 541 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 540 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 41 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 40 **Pre-Assignment Details:** A total of 1485 participants were enrolled, of which 1162 participants were randomized to BHV-3000 (rimegepant) 75 milligram (mg) or placebo. A total of 323 participants failed screening mainly due to failure to meet eligibility criteria.The randomization was stratified in a 1:1 ratio based on use of prophylactic migraine medications (yes or no). **Recruitment Details:** The study was conducted at 50 centers in the United States. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT02328079 **Brief Title:** Steroid-Antiviral Treatment in Rehabilitation of Facial Palsy **Official Title:** Antiviral Treatment in Facial Palsy. Randomized Control Trial #### Organization Study ID Info **ID:** Antiviral in facial palsy #### Organization **Class:** OTHER **Full Name:** Assiut University ### Status Module #### Completion Date **Date:** 2014-11 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-09-16 **Type:** ACTUAL **Last Update Submit Date:** 2020-09-12 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2014-11 **Type:** ACTUAL #### Start Date **Date:** 2013-04 **Status Verified Date:** 2020-09 #### Study First Post Date **Date:** 2014-12-31 **Type:** ESTIMATED **Study First Submit Date:** 2014-12-10 **Study First Submit QC Date:** 2014-12-26 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Assiut University #### Responsible Party **Investigator Affiliation:** Assiut University **Investigator Full Name:** Eman M. Khedr **Investigator Title:** Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this study is to assess the efficacy of antiviral medicine (acyclovir) in recovery of complete facial Palsy. Fifty patients (Males and females) with acute Facial Palsy within the first 3 days of onset with age ranged from 15-60 years old. Each patient was submitted to the following clinical evaluation using House and Brackmann 6 facial function scoring system and Synnybrook grading system. Neurophysiological assessment of facial nerve and muscles was done before and after the end of treatment, then after the end of first and second month of treatment. EMG was done for facial muscles of both sides beside measuring facial nerve excitability to determine the excitation threshold by recording the minimum electrical stimulus required to produce visible muscle contraction. A difference greater than 3.5 mA between the affected and unaffected side is considered significant in terms of poor prognosis. Nerve conduction study of facial nerves of both sides using concentric needle electrode. Trigeminal Blink reflex for both sides of the face. Facial functional recovery was defined as "good" or "complete" using the same criteria used in the 2001 practice guideline. An outcome of grade I or II was considered a good recovery using the House and Brackmann 6 facial function scoring system **Detailed Description:** The purpose of this study is to assess the efficacy of antiviral medicine (acyclovir) in recovery of complete facial Palsy. Fifty patients (Males and females) with acute Facial Palsy within the first 3 days of onset with age ranged from 15-60 years old. Each patient was submitted to the following clinical evaluation using House and Brackmann 6 facial function scoring system and Synnybrook grading system. Neurophysiological assessment of facial nerve and muscle was done before and after the end of treatment, then after the end of first and second month of treatment. EMG was done for facial muscles of both sides beside measuring facial nerve excitability to determine the excitation threshold by recording the minimum electrical stimulus required to produce visible muscle contraction. A difference greater than 3.5 mA between the affected and unaffected side is considered significant in terms of poor prognosis. Nerve conduction study of facial nerves of both sides using concentric needle electrode. Trigeminal Blink reflex for both sides of the face. Facial functional recovery was defined as "good" or "complete" using the same criteria used in the 2001 practice guideline. An outcome of grade I or II was considered a good recovery using the House and Brackmann 6 facial function scoring system ### Conditions Module **Conditions:** - Facial Palsy **Keywords:** - Facial Palsy (acute), Acyclovir, Prednisolone ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 50 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** prednisolone 60 mg /day IM /IV for 6 consecutive days then reduced by 10 mg /day (for a total treatment time for 12 days) **Intervention Names:** - Drug: Steroids (Prednisolone) and Steroids plus Antiviral (Prednisolone + acyclovir) **Label:** Steroid Group **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Prednisolone IM/ IV 60 mg /day + IV acyclovir 500 mg three time /day for 6 consecutive days) then reduced by 10 mg /day (for a total treatment time for 12 days) **Intervention Names:** - Drug: Steroids (Prednisolone) and Steroids plus Antiviral (Prednisolone + acyclovir) **Label:** Steroid + Antiviral Group **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Steroid + Antiviral Group - Steroid Group **Description:** Group allocations: Steroid group (prednisolone 60 mg /day IM /IV), Steroid plus Antiviral group (Prednisolone IM/ IV 60 mg /day + IV acyclovir 500 mg three time /day for 6 consecutive days) then reduced by 10 mg /day (for a total treatment time for 12 days) were placed in serially-numbered opaque closed envelopes. Each patient was placed in the appropriate group after opening the corresponding sealed envelope. **Name:** Steroids (Prednisolone) and Steroids plus Antiviral (Prednisolone + acyclovir) **Other Names:** - Steroid group (Prednisolone), Steroid plus Antiviral group (Prednisolone + acyclovir) **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Evaluations of facial muscle function using clinical scale were performed blindly by a neurologist who was unaware of the type of treatment of which the patient had received **Measure:** Facial muscle function using clinical scale **Time Frame:** 2 months #### Secondary Outcomes **Description:** Measurment of facial n. coduction **Measure:** Nerve conduction study of facial nerve. **Time Frame:** 2 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Acute onset facial palsy within the first three days of onset. Age ranged from 15-60 years old Exclusion Criteria: * Patients with brittle diabetes mellitus, Morbid obesity, renal or liver impairment, osteopenia, prior history of steroid intolerance. **Maximum Age:** 60 Years **Minimum Age:** 15 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT ### References Module #### References **Citation:** Khedr EM, Badry R, Ali AM, Abo El-Fetoh N, El-Hammady DH, Ghandour AM, Abdel-Haleem A. Steroid/Antiviral for the treatment of Bell's palsy: Double blind randomized clinical trial. Restor Neurol Neurosci. 2016 Nov 22;34(6):897-905. doi: 10.3233/RNN-150605. **PMID:** 27689547 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009461 - Term: Neurologic Manifestations - ID: D000009422 - Term: Nervous System Diseases - ID: D000020969 - Term: Disease Attributes - ID: D000010335 - Term: Pathologic Processes - ID: D000009059 - Term: Mouth Diseases - ID: D000009057 - Term: Stomatognathic Diseases - ID: D000006566 - Term: Herpesviridae Infections - ID: D000004266 - Term: DNA Virus Infections - ID: D000014777 - Term: Virus Diseases - ID: D000007239 - Term: Infections - ID: D000005155 - Term: Facial Nerve Diseases - ID: D000003389 - Term: Cranial Nerve Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC01 - Name: Infections - Abbrev: BC07 - Name: Mouth and Tooth Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M13157 - Name: Paralysis - Relevance: HIGH - As Found: Palsy - ID: M22138 - Name: Bell Palsy - Relevance: HIGH - As Found: Facial Palsy - ID: M8301 - Name: Facial Paralysis - Relevance: HIGH - As Found: Facial Palsy - ID: M21089 - Name: Facies - Relevance: HIGH - As Found: Facial - ID: M12404 - Name: Neurologic Manifestations - Relevance: LOW - As Found: Unknown - ID: M22700 - Name: Disease Attributes - Relevance: LOW - As Found: Unknown - ID: M12019 - Name: Mouth Diseases - Relevance: LOW - As Found: Unknown - ID: M12017 - Name: Stomatognathic Diseases - Relevance: LOW - As Found: Unknown - ID: M10283 - Name: Infections - Relevance: LOW - As Found: Unknown - ID: M6368 - Name: Communicable Diseases - Relevance: LOW - As Found: Unknown - ID: M9643 - Name: Herpesviridae Infections - Relevance: LOW - As Found: Unknown - ID: M17522 - Name: Virus Diseases - Relevance: LOW - As Found: Unknown - ID: M7442 - Name: DNA Virus Infections - Relevance: LOW - As Found: Unknown - ID: M8298 - Name: Facial Nerve Diseases - Relevance: LOW - As Found: Unknown - ID: M6605 - Name: Cranial Nerve Diseases - Relevance: LOW - As Found: Unknown - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown - ID: T707 - Name: Bell's Palsy - Relevance: HIGH - As Found: Facial Palsy ### Condition Browse Module - Meshes - ID: D000020330 - Term: Bell Palsy - ID: D000005158 - Term: Facial Paralysis - ID: D000010243 - Term: Paralysis - ID: D000019066 - Term: Facies ### Intervention Browse Module - Ancestors - ID: D000000893 - Term: Anti-Inflammatory Agents - ID: D000005938 - Term: Glucocorticoids - ID: D000006728 - Term: Hormones - ID: D000006730 - Term: Hormones, Hormone Substitutes, and Hormone Antagonists - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018931 - Term: Antineoplastic Agents, Hormonal - ID: D000000970 - Term: Antineoplastic Agents - ID: D000000932 - Term: Antiemetics - ID: D000001337 - Term: Autonomic Agents - ID: D000018373 - Term: Peripheral Nervous System Agents - ID: D000005765 - Term: Gastrointestinal Agents - ID: D000018696 - Term: Neuroprotective Agents - ID: D000020011 - Term: Protective Agents - ID: D000000890 - Term: Anti-Infective Agents ### Intervention Browse Module - Browse Branches - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: AnEm - Name: Antiemetics - Abbrev: NeuroAg - Name: Neuroprotective Agents - Abbrev: Gast - Name: Gastrointestinal Agents - Abbrev: Infe - Name: Anti-Infective Agents ### Intervention Browse Module - Browse Leaves - ID: M14120 - Name: Prednisolone - Relevance: HIGH - As Found: Vein - ID: M11749 - Name: Methylprednisolone - Relevance: HIGH - As Found: Vein - ID: M3567 - Name: Acyclovir - Relevance: HIGH - As Found: Aqueous - ID: M1833 - Name: Methylprednisolone Acetate - Relevance: HIGH - As Found: Vein - ID: M11750 - Name: Methylprednisolone Hemisuccinate - Relevance: HIGH - As Found: Vein - ID: M229449 - Name: Prednisolone acetate - Relevance: HIGH - As Found: Vein - ID: M211887 - Name: Prednisolone hemisuccinate - Relevance: HIGH - As Found: Vein - ID: M248881 - Name: Prednisolone phosphate - Relevance: HIGH - As Found: Vein - ID: M4314 - Name: Antiviral Agents - Relevance: HIGH - As Found: Intravesical - ID: M4217 - Name: Anti-Inflammatory Agents - Relevance: LOW - As Found: Unknown - ID: M9047 - Name: Glucocorticoids - Relevance: LOW - As Found: Unknown - ID: M9789 - Name: Hormones - Relevance: LOW - As Found: Unknown - ID: M9788 - Name: Hormone Antagonists - Relevance: LOW - As Found: Unknown - ID: M20966 - Name: Antineoplastic Agents, Hormonal - Relevance: LOW - As Found: Unknown - ID: M4251 - Name: Antiemetics - Relevance: LOW - As Found: Unknown - ID: M8881 - Name: Gastrointestinal Agents - Relevance: LOW - As Found: Unknown - ID: M20773 - Name: Neuroprotective Agents - Relevance: LOW - As Found: Unknown - ID: M21869 - Name: Protective Agents - Relevance: LOW - As Found: Unknown - ID: M4214 - Name: Anti-Infective Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000000212 - Term: Acyclovir - ID: D000000998 - Term: Antiviral Agents - ID: D000011239 - Term: Prednisolone - ID: D000077555 - Term: Methylprednisolone Acetate - ID: D000008775 - Term: Methylprednisolone - ID: D000008776 - Term: Methylprednisolone Hemisuccinate - ID: C000009935 - Term: Prednisolone acetate - ID: C000021322 - Term: Prednisolone hemisuccinate - ID: C000009022 - Term: Prednisolone phosphate ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02898779 **Brief Title:** Metabolism and Pharmacokinetics of Primaquine Enantiomers in Human Volunteers, Study 1 **Official Title:** Development of Safer Drugs for Malaria in U.S. Troops, Civilian Personnel, and Travelers: Clinical Evaluation of Primaquine Enantiomer #### Organization Study ID Info **ID:** PQ Study 1 #### Organization **Class:** OTHER **Full Name:** University of Mississippi, Oxford ### Status Module #### Completion Date **Date:** 2018-03-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-05-15 **Type:** ACTUAL **Last Update Submit Date:** 2018-05-14 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2018-03-01 **Type:** ACTUAL #### Start Date **Date:** 2017-05-01 **Type:** ACTUAL **Status Verified Date:** 2018-05 #### Study First Post Date **Date:** 2016-09-13 **Type:** ESTIMATED **Study First Submit Date:** 2016-07-21 **Study First Submit QC Date:** 2016-09-07 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Mississippi, Oxford #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Oversight Has DMC:** False ### Description Module **Brief Summary:** To investigate the comparative tolerability, metabolism and pharmacokinetics of individual enantiomers of PQ in healthy human volunteers. The specific aim is the comparative evaluation of the metabolism, pharmacokinetic behavior, and tolerability of the isomers of PQ (RPQ and SPQ and the racemic mixture RSPQ) in normal healthy human volunteers. **Detailed Description:** The primary objective of this project is to investigate the comparative tolerability, metabolism and pharmacokinetics of individual enantiomers of PQ in healthy human volunteers. The overall approach is as follows: in 36 healthy volunteers with documented normal G6PD activity, we will administer a single oral dose of RPQ, SPQ, or RSPQ. At various times after dosing, we will draw blood samples, in which we will record the plasma levels of the parent drugs, along with plasma and urinary metabolites. The comparative pharmacokinetics, tolerability and hematological effects of these two enantiomers and the racemate will be assessed. ### Conditions Module **Conditions:** - Malaria ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER ##### Masking Info **Masking:** SINGLE **Who Masked:** - PARTICIPANT **Primary Purpose:** BASIC_SCIENCE #### Enrollment Info **Count:** 36 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Interventions: Primaquine, R-Primaquine, S-Primaquine, SR Primaquine A single center, prospective, cross-over, randomized phase 1 trial. Thirty-six participants, enrolled into a two cohort pharmacokinetic study evaluating two dose levels of primaquine isomers. Cohort 1 (Low Dose Level)- single dose of 15 mg of S-Primaquine and 15 mg of R-Primaquine compared to 30 mg RS-Primaquine over 24 hours. Participants will cross-over after a one week wash-out period. **Intervention Names:** - Drug: Primaquine, R-Primaquine, S-Primaquine, SR Primaquine **Label:** Cohort 1 ( Primaquine Low Dose) **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Interventions: Primaquine, R-Primaquine, S-Primaquine, SR Primaquine A single center, prospective, cross-over, randomized phase 1 trial. Thirty-six participants, enrolled into a two cohort pharmacokinetic study evaluating two dose levels of primaquine isomers. Cohort 2 (High Dose Level)-single dose of 22.5 mg of S-Primaquine and 22.5 mg of R-Primaquine compared to 45 mg RS-Primaquine over 24 hours. Participants will cross-over among the treatment arms following a one week wash-out period between each. **Intervention Names:** - Drug: Primaquine, R-Primaquine, S-Primaquine, SR Primaquine **Label:** Cohort 2 (Primaquine High Dose) **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Cohort 1 ( Primaquine Low Dose) - Cohort 2 (Primaquine High Dose) **Description:** Cohort 1: Eighteen individuals (6 per group) Cohort 2: Eighteen individuals (6 per group) Group 1-15 mg of S-Primaquine followed by one-week washout, 15 mg of R-Primaquine followed by one week washout, and 30 mg of RS-Primaquine. Group 2- 15 mg of R-Primaquine followed by one-week washout, 30 mg of RSPQ followed by one week washout, and 15 mg of SPQ. Group 3- 30 mg of RS-Primaquine followed by one week washout,15 mg of SPQ followed by a one week washout, and 15 mg of RPQ. **Name:** Primaquine, R-Primaquine, S-Primaquine, SR Primaquine **Other Names:** - Primaquine **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** This study would provide information on differential pharmacokinetics and metabolism of enantiomers of primaquine in normal human volunteers **Measure:** Primary outcome: Plasma concentration of parent primaquine and carboyprimaquine following a single dose treatment with primaquine (racemate or enantiomers) not to exceed 45 mg **Time Frame:** between 0-24 Hours #### Secondary Outcomes **Description:** This study would provide information on differential pharmacokinetics and metabolism of enantiomers of primaquine in normal human volunteers **Measure:** Area Under Curve (AUC) for primaquine up to 24 hours after the primaquine administration **Time Frame:** between 0-24 hours **Description:** This study would provide information on differential pharmacokinetics and metabolism of enantiomers of primaquine in normal human volunteers **Measure:** Maximum concentration (Cmax) for primaquine up to 24 hours after the primaquine administration **Time Frame:** between 0-24 hours **Description:** This study would provide information on differential pharmacokinetics and metabolism of enantiomers of primaquine in normal human volunteers **Measure:** Area Under Curve (AUC) for carboxyprimaquine, the major plasma metabolite of primaquine, up to 24 hours after primaquine administration **Time Frame:** between 0-24 hours **Description:** This study would provide information on differential pharmacokinetics and metabolism of enantiomers of primaquine in normal human volunteers **Measure:** Maximum concentration of carboxyprimaquine, the major plasma metabolite of primaquine, up to 24 hours after primaquine administration **Time Frame:** between 0-24 hours **Description:** This study would provide information on differential pharmacokinetics and metabolism of enantiomers of primaquine in normal human volunteers. The exact nature of these metabolites will be determined from previous animal and human studies **Measure:** Maximum concentration of selected metabolites primaquine (other than carboxyprimaquine) up to 24 hours after primaquine administration **Time Frame:** between 0-24 hours **Description:** To monitor hemoglobin and methemoglobin levels in normal human volunteers treated with single dose of primaquine not to exceed 45 mg **Measure:** hemoglobin and methemoglobin levels in the blood after administration of primaquine **Time Frame:** 0-72 hours **Description:** To determine correlation between metabolism of primaquine and CYP 2D6 genotype **Measure:** Genotyping of Cytochrome P-450 (CYP) **Time Frame:** day 0 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Adults (18-60 years of age) * Informed consent * Healthy Exclusion Criteria: * Known history of liver, kidney or hematological disease; * known history of cardiac disease, arrhythmia, QT prolongation; * Autoimmune disorder; * Report of an active infection; * Evidence of G6PD deficiency **Healthy Volunteers:** True **Maximum Age:** 60 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Oxford **Country:** United States **Facility:** University of Mississippi **State:** Mississippi **Zip:** 38677 #### Overall Officials **Official 1:** **Affiliation:** University of Mississippi Medical Center **Name:** Babu L Tekwani, Ph.D **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Avula B, Tekwani BL, Chaurasiya ND, Nanayakkara NP, Wang YH, Khan SI, Adelli VR, Sahu R, Elsohly MA, McChesney JD, Khan IA, Walker LA. Profiling primaquine metabolites in primary human hepatocytes using UHPLC-QTOF-MS with 13C stable isotope labeling. J Mass Spectrom. 2013 Feb;48(2):276-85. doi: 10.1002/jms.3122. **PMID:** 23378100 **Citation:** Avula B, Khan SI, Tekwani BL, Nanayakkara NP, McChesney JD, Walker LA, Khan IA. Analysis of primaquine and its metabolite carboxyprimaquine in biological samples: enantiomeric separation, method validation and quantification. Biomed Chromatogr. 2011 Sep;25(9):1010-7. doi: 10.1002/bmc.1557. Epub 2010 Nov 5. **PMID:** 21058417 **Citation:** Baker JK, McChesney JD. Differential metabolism of the enantiomers of primaquine. J Pharm Sci. 1988 May;77(5):380-2. doi: 10.1002/jps.2600770503. **PMID:** 3411455 **Citation:** Lu H. Stereoselectivity in drug metabolism. Expert Opin Drug Metab Toxicol. 2007 Apr;3(2):149-58. doi: 10.1517/17425255.3.2.149. **PMID:** 17428147 **Citation:** Fasinu PS, Tekwani BL, Nanayakkara NP, Avula B, Herath HM, Wang YH, Adelli VR, Elsohly MA, Khan SI, Khan IA, Pybus BS, Marcsisin SR, Reichard GA, McChesney JD, Walker LA. Enantioselective metabolism of primaquine by human CYP2D6. Malar J. 2014 Dec 17;13:507. doi: 10.1186/1475-2875-13-507. **PMID:** 25518709 **Citation:** Fasinu PS, Avula B, Tekwani BL, Nanayakkara NP, Wang YH, Bandara Herath HM, McChesney JD, Reichard GA, Marcsisin SR, Elsohly MA, Khan SI, Khan IA, Walker LA. Differential kinetic profiles and metabolism of primaquine enantiomers by human hepatocytes. Malar J. 2016 Apr 19;15:224. doi: 10.1186/s12936-016-1270-1. **PMID:** 27093859 **Citation:** Graves PM, Gelband H, Garner P. Primaquine or other 8-aminoquinoline for reducing Plasmodium falciparum transmission. Cochrane Database Syst Rev. 2015 Feb 19;(2):CD008152. doi: 10.1002/14651858.CD008152.pub4. **PMID:** 25693791 **Citation:** Jin X, Pybus BS, Marcsisin R, Logan T, Luong TL, Sousa J, Matlock N, Collazo V, Asher C, Carroll D, Olmeda R, Walker LA, Kozar MP, Melendez V. An LC-MS based study of the metabolic profile of primaquine, an 8-aminoquinoline antiparasitic drug, with an in vitro primary human hepatocyte culture model. Eur J Drug Metab Pharmacokinet. 2014 Jun;39(2):139-46. doi: 10.1007/s13318-013-0139-8. **PMID:** 23797843 **Citation:** Mihaly GW, Ward SA, Edwards G, Orme ML, Breckenridge AM. Pharmacokinetics of primaquine in man: identification of the carboxylic acid derivative as a major plasma metabolite. Br J Clin Pharmacol. 1984 Apr;17(4):441-6. doi: 10.1111/j.1365-2125.1984.tb02369.x. **PMID:** 6721990 **Citation:** Nanayakkara NP, Ager AL Jr, Bartlett MS, Yardley V, Croft SL, Khan IA, McChesney JD, Walker LA. Antiparasitic activities and toxicities of individual enantiomers of the 8-aminoquinoline 8-[(4-amino-1-methylbutyl)amino]-6-methoxy-4-methyl-5-[3,4-dichlorophenoxy]quinoline succinate. Antimicrob Agents Chemother. 2008 Jun;52(6):2130-7. doi: 10.1128/AAC.00645-07. Epub 2008 Mar 31. **PMID:** 18378716 **Citation:** Nanayakkara NP, Tekwani BL, Herath HM, Sahu R, Gettayacamin M, Tungtaeng A, van Gessel Y, Baresel P, Wickham KS, Bartlett MS, Fronczek FR, Melendez V, Ohrt C, Reichard GA, McChesney JD, Rochford R, Walker LA. Scalable preparation and differential pharmacologic and toxicologic profiles of primaquine enantiomers. Antimicrob Agents Chemother. 2014 Aug;58(8):4737-44. doi: 10.1128/AAC.02674-13. Epub 2014 Jun 9. **PMID:** 24913163 **Citation:** Potter BM, Xie LH, Vuong C, Zhang J, Zhang P, Duan D, Luong TL, Bandara Herath HM, Dhammika Nanayakkara NP, Tekwani BL, Walker LA, Nolan CK, Sciotti RJ, Zottig VE, Smith PL, Paris RM, Read LT, Li Q, Pybus BS, Sousa JC, Reichard GA, Marcsisin SR. Differential CYP 2D6 metabolism alters primaquine pharmacokinetics. Antimicrob Agents Chemother. 2015 Apr;59(4):2380-7. doi: 10.1128/AAC.00015-15. Epub 2015 Feb 2. **PMID:** 25645856 **Citation:** Pybus BS, Marcsisin SR, Jin X, Deye G, Sousa JC, Li Q, Caridha D, Zeng Q, Reichard GA, Ockenhouse C, Bennett J, Walker LA, Ohrt C, Melendez V. The metabolism of primaquine to its active metabolite is dependent on CYP 2D6. Malar J. 2013 Jun 20;12:212. doi: 10.1186/1475-2875-12-212. **PMID:** 23782898 **Citation:** Saunders D, Vanachayangkul P, Imerbsin R, Khemawoot P, Siripokasupkul R, Tekwani BL, Sampath A, Nanayakkara NP, Ohrt C, Lanteri C, Gettyacamin M, Teja-Isavadharm P, Walker L. Pharmacokinetics and pharmacodynamics of (+)-primaquine and (-)-primaquine enantiomers in rhesus macaques (Macaca mulatta). Antimicrob Agents Chemother. 2014 Dec;58(12):7283-91. doi: 10.1128/AAC.02576-13. Epub 2014 Sep 29. **PMID:** 25267666 **Citation:** Schmidt LH, Alexander S, Allen L, Rasco J. Comparison of the curative antimalarial activities and toxicities of primaquine and its d and l isomers. Antimicrob Agents Chemother. 1977 Jul;12(1):51-60. doi: 10.1128/AAC.12.1.51. **PMID:** 407841 **Citation:** Tekwani BL, Walker LA. 8-Aminoquinolines: future role as antiprotozoal drugs. Curr Opin Infect Dis. 2006 Dec;19(6):623-31. doi: 10.1097/QCO.0b013e328010b848. **PMID:** 17075340 **Citation:** Tekwani BL, Avula B, Sahu R, Chaurasiya ND, Khan SI, Jain S, Fasinu PS, Herath HM, Stanford D, Nanayakkara NP, McChesney JD, Yates TW, ElSohly MA, Khan IA, Walker LA. Enantioselective pharmacokinetics of primaquine in healthy human volunteers. Drug Metab Dispos. 2015 Apr;43(4):571-7. doi: 10.1124/dmd.114.061127. Epub 2015 Jan 30. **PMID:** 25637634 **Citation:** Vale N, Moreira R, Gomes P. Primaquine revisited six decades after its discovery. Eur J Med Chem. 2009 Mar;44(3):937-53. doi: 10.1016/j.ejmech.2008.08.011. Epub 2008 Sep 11. **PMID:** 18930565 **Citation:** Vasquez-Vivar J, Augusto O. Hydroxylated metabolites of the antimalarial drug primaquine. Oxidation and redox cycling. J Biol Chem. 1992 Apr 5;267(10):6848-54. **PMID:** 1313024 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000011528 - Term: Protozoan Infections - ID: D000010272 - Term: Parasitic Diseases - ID: D000007239 - Term: Infections - ID: D000096724 - Term: Mosquito-Borne Diseases - ID: D000079426 - Term: Vector Borne Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC01 - Name: Infections - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M11280 - Name: Malaria - Relevance: HIGH - As Found: Malaria - ID: M10283 - Name: Infections - Relevance: LOW - As Found: Unknown - ID: M6368 - Name: Communicable Diseases - Relevance: LOW - As Found: Unknown - ID: M14388 - Name: Protozoan Infections - Relevance: LOW - As Found: Unknown - ID: M13185 - Name: Parasitic Diseases - Relevance: LOW - As Found: Unknown - ID: M3255 - Name: Mosquito-Borne Diseases - Relevance: LOW - As Found: Unknown - ID: M2054 - Name: Vector Borne Diseases - Relevance: LOW - As Found: Unknown - ID: T3571 - Name: Malaria - Relevance: HIGH - As Found: Malaria ### Condition Browse Module - Meshes - ID: D000008288 - Term: Malaria ### Intervention Browse Module - Ancestors - ID: D000000962 - Term: Antimalarials - ID: D000000981 - Term: Antiprotozoal Agents - ID: D000000977 - Term: Antiparasitic Agents - ID: D000000890 - Term: Anti-Infective Agents ### Intervention Browse Module - Browse Branches - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M14192 - Name: Primaquine - Relevance: HIGH - As Found: Adequate - ID: M4280 - Name: Antimalarials - Relevance: LOW - As Found: Unknown - ID: M4298 - Name: Antiprotozoal Agents - Relevance: LOW - As Found: Unknown - ID: M4294 - Name: Antiparasitic Agents - Relevance: LOW - As Found: Unknown - ID: M4214 - Name: Anti-Infective Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000011319 - Term: Primaquine ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02515279 **Brief Title:** An Observational Study of Peginterferon Alfa-2a Plus Ribavirin for Hepatitis C Virus (HCV) Infection in Austria **Official Title:** A Non-Interventional Trial of Pegasys/Copegus in HCV Patients for 12 Months #### Organization Study ID Info **ID:** ML22273 #### Organization **Class:** INDUSTRY **Full Name:** Hoffmann-La Roche ### Status Module #### Completion Date **Date:** 2014-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-04-10 **Type:** ACTUAL **Last Update Submit Date:** 2017-03-10 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2014-12 **Type:** ACTUAL #### Results First Post Date **Date:** 2016-02-10 **Type:** ESTIMATED **Results First Submit Date:** 2016-01-12 **Results First Submit QC Date:** 2016-01-12 #### Start Date **Date:** 2008-11 **Status Verified Date:** 2017-03 #### Study First Post Date **Date:** 2015-08-04 **Type:** ESTIMATED **Study First Submit Date:** 2015-07-29 **Study First Submit QC Date:** 2015-07-31 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Hoffmann-La Roche #### Responsible Party **Type:** SPONSOR ### Description Module **Brief Summary:** This noninterventional, open-label study will observe the safety and tolerability of peginterferon alfa-2a in combination with ribavirin among Austrian participants treated for HCV infection according to routine practice. ### Conditions Module **Conditions:** - Hepatitis C ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 463 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** All participants were treated with Peginterferon alfa-2a+Ribavirin (Pegasys/Copegus) according to the summary of product characteristics and to the investigator's discretion. The daily recommended dose for Pegasys, for the treatment of chronic Hepatitis C, was 180 micrograms once weekly by subcutaneous administration. Copegus was administered orally in doses according to the physician's decision (depending on the participant's weight and genotype). All participants were observed for 12 months. **Intervention Names:** - Drug: Peginterferon alfa-2a - Drug: Ribavirin **Label:** Participants With Hepatitis C ### Interventions #### Intervention 1 **Arm Group Labels:** - Participants With Hepatitis C **Description:** 180 micrograms subcutaneous weekly for 48 weeks. **Name:** Peginterferon alfa-2a **Other Names:** - Pegasys **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Participants With Hepatitis C **Description:** 1000-1600 mg day orally for 48 weeks. **Name:** Ribavirin **Other Names:** - Copegus **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability or incapacity; and congenital anomaly. Percentage of participants with AEs included participants affected with both SAEs and non-SAEs. **Measure:** Percentage of Participants With Serious Adverse Events (SAEs) and Adverse Events (AEs) **Time Frame:** Up to 6 years **Description:** Clinical response to the treatment was measured by qualitative negative polymerase chain reaction (PCR). A participant was considered to have and end of treatment response if there was undetectable Hepatitis C Virus (HCV) ribonucleic acid (RNA) after completing treatment. Participants with available PCR results were reported. **Measure:** Percentage of Participants With End of Treatment Response **Time Frame:** 12 months **Description:** Clinical response to the treatment was measured by qualitative negative polymerase chain reaction (PCR). SVR24 is defined as the percentage of participants with undetectable HCV RNA 24 weeks after completing treatment. **Measure:** Percentage of Participants With Sustained Virologic Response 24 (SVR24) **Time Frame:** 18 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Ongoing treatment with peginterferon alfa-2a and ribavirin at the discretion of the prescribing physician * HCV infection Exclusion Criteria: * None specified **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT **Study Population:** The study will enroll Austrian participants receiving peginterferon alfa-2a and ribavirin for HCV infection according to routine practice. ### Contacts Locations Module #### Locations **Location 1:** **City:** Ried-innkreis **Country:** Austria **Facility:** A.Ö. Krankenhaus Der Barmherzigen Schwestern Ried; Interne Abtl. **Zip:** 4910 #### Overall Officials **Official 1:** **Affiliation:** Hoffmann-La Roche **Name:** Clinical Trials **Role:** STUDY_DIRECTOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000008107 - Term: Liver Diseases - ID: D000004066 - Term: Digestive System Diseases - ID: D000006525 - Term: Hepatitis, Viral, Human - ID: D000014777 - Term: Virus Diseases - ID: D000007239 - Term: Infections - ID: D000004769 - Term: Enterovirus Infections - ID: D000010850 - Term: Picornaviridae Infections - ID: D000012327 - Term: RNA Virus Infections - ID: D000086982 - Term: Blood-Borne Infections - ID: D000003141 - Term: Communicable Diseases - ID: D000018178 - Term: Flaviviridae Infections ### Condition Browse Module - Browse Branches - Abbrev: BC01 - Name: Infections - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC06 - Name: Digestive System Diseases ### Condition Browse Module - Browse Leaves - ID: M10283 - Name: Infections - Relevance: LOW - As Found: Unknown - ID: M6368 - Name: Communicable Diseases - Relevance: LOW - As Found: Unknown - ID: M9592 - Name: Hepatitis A - Relevance: HIGH - As Found: Hepatitis - ID: M9591 - Name: Hepatitis - Relevance: HIGH - As Found: Hepatitis - ID: M17522 - Name: Virus Diseases - Relevance: LOW - As Found: Unknown - ID: M9611 - Name: Hepatitis C - Relevance: HIGH - As Found: Hepatitis C - ID: M11107 - Name: Liver Diseases - Relevance: LOW - As Found: Unknown - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M9610 - Name: Hepatitis, Viral, Human - Relevance: LOW - As Found: Unknown - ID: M7930 - Name: Enterovirus Infections - Relevance: LOW - As Found: Unknown - ID: M13745 - Name: Picornaviridae Infections - Relevance: LOW - As Found: Unknown - ID: M15149 - Name: RNA Virus Infections - Relevance: LOW - As Found: Unknown - ID: M2593 - Name: Blood-Borne Infections - Relevance: LOW - As Found: Unknown - ID: M20324 - Name: Flaviviridae Infections - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006506 - Term: Hepatitis A - ID: D000006526 - Term: Hepatitis C - ID: D000006505 - Term: Hepatitis ### Intervention Browse Module - Ancestors - ID: D000000963 - Term: Antimetabolites - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000000998 - Term: Antiviral Agents - ID: D000000890 - Term: Anti-Infective Agents ### Intervention Browse Module - Browse Branches - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M15083 - Name: Ribavirin - Relevance: HIGH - As Found: Bevacizumab - ID: M247369 - Name: Peginterferon alfa-2a - Relevance: HIGH - As Found: Longitudinal - ID: M4281 - Name: Antimetabolites - Relevance: LOW - As Found: Unknown - ID: M4314 - Name: Antiviral Agents - Relevance: LOW - As Found: Unknown - ID: M4214 - Name: Anti-Infective Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000012254 - Term: Ribavirin - ID: C000100416 - Term: Peginterferon alfa-2a ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** All participant who were enrolled in the study. #### Event Groups **Group ID:** EG000 **Title:** Participants With Hepatitis C **Description:** All participants were treated with Peginterferon alfa-2a+Ribavirin (Pegasys/Copegus) according to the summary of product characteristics and to the investigator's discretion. The daily recommended dose for Pegasys, for the treatment of chronic Hepatitis C, was 180 micrograms once weekly by subcutaneous administration. Copegus was administered orally in doses according to the physician's decision (depending on the participant's weight and genotype). All participants were observed for 12 months. **ID:** EG000 **Other Num Affected:** 148 **Other Num at Risk:** 463 **Serious Number Affected:** 18 **Serious Number At Risk:** 463 **Title:** Participants With Hepatitis C **Frequency Threshold:** 5 #### Other Events **Term:** Leukopenia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 18.1 **Term:** Anaemia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 18.1 **Term:** Thrombocytopenia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 18.1 **Term:** Fatigue **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 18.1 **Term:** Depression **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 18.1 **Term:** Pruritus **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 18.1 #### Serious Events **Term:** Anaemia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 18.1 ##### Stats **Group ID:** EG000 **Num Affected:** 8 **Num At Risk:** 463 **Term:** Leukopenia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 18.1 ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 463 **Term:** Thrombocytopenia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 18.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 463 **Term:** Influenza like illness **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 18.1 ##### Stats **Group ID:** EG000 **Num Affected:** 4 **Num At Risk:** 463 **Term:** Fatigue **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 18.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 463 **Term:** Headache **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 18.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 463 **Term:** Diarrhoea **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 18.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 463 **Term:** Arthralgia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 18.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 463 **Term:** Depression **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 18.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 463 **Term:** Pneumonia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 18.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 463 **Term:** Drug eruption **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 18.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 463 **Term:** Pruritus **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 18.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 463 **Term:** Syncope **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 18.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 463 **Time Frame:** up to 6 years ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 463 **Units:** Participants ### Group **ID:** BG000 **Title:** Participants With Hepatitis C **Description:** All participants were treated with Peginterferon alfa-2a+Ribavirin (Pegasys/Copegus) according to the summary of product characteristics and to the investigator's discretion. The daily recommended dose for Pegasys, for the treatment of chronic Hepatitis C, was 180 micrograms once weekly by subcutaneous administration. Copegus was administered orally in doses according to the physician's decision (depending on the participant's weight and genotype). All participants were observed for 12 months. ### Measure #### Measurement **Group ID:** BG000 **Spread:** 13.3 **Value:** 41.0 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 151 **Class Title:** Female #### Measurement **Group ID:** BG000 **Value:** 310 **Class Title:** Male #### Measurement **Group ID:** BG000 **Value:** 2 **Class Title:** Missing **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 2 **Parameter Type:** NUMBER **Title:** Sex/Gender, Customized **Unit of Measure:** participants **Population Description:** All participants who were enrolled in the study. ## Results Section - More Information Module ### Certain Agreement **Other Details:** The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights. **Restriction Type:** OTHER **Restrictive Agreement:** True ### Limitations and Caveats **Description:** The sustained viral response that had to be filled out 6 months (or later) after the end of treatment was documented poorly due to reasons such as participants that were lost to follow-up or the study closure was before the visit date for the SVR. ### Point of Contact **Email:** [email protected] **Organization:** Hoffmann-LaRoche **Phone:** 800-821-8590 **Title:** Medical Communications ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 44.28 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3.9 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 83.8 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 9.7 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 26.6 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability or incapacity; and congenital anomaly. Percentage of participants with AEs included participants affected with both SAEs and non-SAEs. **Parameter Type:** NUMBER **Population Description:** All participants who were enrolled in the study. **Reporting Status:** POSTED **Time Frame:** Up to 6 years **Title:** Percentage of Participants With Serious Adverse Events (SAEs) and Adverse Events (AEs) **Type:** PRIMARY **Unit of Measure:** percentage of participants ##### Group **Description:** All participants were treated with Peginterferon alfa-2a+Ribavirin (Pegasys/Copegus) according to the summary of product characteristics and to the investigator's discretion. The daily recommended dose for Pegasys, for the treatment of chronic Hepatitis C, was 180 micrograms once weekly by subcutaneous administration. Copegus was administered orally in doses according to the physician's decision (depending on the participant's weight and genotype). All participants were observed for 12 months. **ID:** OG000 **Title:** Participants With Hepatitis C #### Outcome Measure 2 **Description:** Clinical response to the treatment was measured by qualitative negative polymerase chain reaction (PCR). A participant was considered to have and end of treatment response if there was undetectable Hepatitis C Virus (HCV) ribonucleic acid (RNA) after completing treatment. Participants with available PCR results were reported. **Parameter Type:** NUMBER **Population Description:** All participants who were enrolled in the study. N (Number of participants analysed)=participants who were evaluable for this measure. **Reporting Status:** POSTED **Time Frame:** 12 months **Title:** Percentage of Participants With End of Treatment Response **Type:** PRIMARY **Unit of Measure:** percentage of participants ##### Group **Description:** All participants were treated with Peginterferon alfa-2a+Ribavirin (Pegasys/Copegus) according to the summary of product characteristics and to the investigator's discretion. The daily recommended dose for Pegasys, for the treatment of chronic Hepatitis C, was 180 micrograms once weekly by subcutaneous administration. Copegus was administered orally in doses according to the physician's decision (depending on the participant's weight and genotype). All participants were observed for 12 months. **ID:** OG000 **Title:** Participants With Hepatitis C #### Outcome Measure 3 **Description:** Clinical response to the treatment was measured by qualitative negative polymerase chain reaction (PCR). SVR24 is defined as the percentage of participants with undetectable HCV RNA 24 weeks after completing treatment. **Parameter Type:** NUMBER **Population Description:** All participant who were enrolled in the study. N=number of participants evaluable for this measure. **Reporting Status:** POSTED **Time Frame:** 18 months **Title:** Percentage of Participants With Sustained Virologic Response 24 (SVR24) **Type:** PRIMARY **Unit of Measure:** percentage of participants ##### Group **Description:** All participants were treated with Peginterferon alfa-2a+Ribavirin (Pegasys/Copegus) according to the summary of product characteristics and to the investigator's discretion. The daily recommended dose for Pegasys, for the treatment of chronic Hepatitis C, was 180 micrograms once weekly by subcutaneous administration. Copegus was administered orally in doses according to the physician's decision (depending on the participant's weight and genotype). All participants were observed for 12 months. **ID:** OG000 **Title:** Participants With Hepatitis C ### Participant Flow Module #### Group **Description:** All participants were treated with Peginterferon alfa-2a+Ribavirin (Pegasys/Copegus) according to the summary of product characteristics and to the investigator's discretion. The daily recommended dose for Pegasys, for the treatment of chronic Hepatitis C, was 180 micrograms once weekly by subcutaneous administration. Copegus was administered orally in doses according to the physician's decision (depending on the participant's weight and genotype). All participants were observed for 12 months. **ID:** FG000 **Title:** Participants With Hepatitis C #### Period **Title:** Overall Study ##### Withdraw **Type:** Adverse Event ###### Reason **Group ID:** FG000 **Number of Subjects:** 7 ##### Withdraw **Type:** Lack of Efficacy ###### Reason **Group ID:** FG000 **Number of Subjects:** 31 ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 9 ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 18 ##### Withdraw **Type:** End-of-treatment visit not attended ###### Reason **Group ID:** FG000 **Number of Subjects:** 17 ##### Withdraw **Type:** Other ###### Reason **Group ID:** FG000 **Number of Subjects:** 22 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 463 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 359 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 104 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT05042479 **Acronym:** VIRTUOSO **Brief Title:** Using of Virtual Reality to Relieve Procedural Pain in Pediatric Oncology. **Official Title:** Using of Virtual Reality to Relieve Procedural Pain in Pediatric Oncology. #### Organization Study ID Info **ID:** RC21_0008 #### Organization **Class:** OTHER **Full Name:** Nantes University Hospital ### Status Module #### Completion Date **Date:** 2025-02-11 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-12-07 **Type:** ACTUAL **Last Update Submit Date:** 2023-12-06 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2025-02-11 **Type:** ESTIMATED #### Start Date **Date:** 2022-06-30 **Type:** ACTUAL **Status Verified Date:** 2023-12 #### Study First Post Date **Date:** 2021-09-13 **Type:** ACTUAL **Study First Submit Date:** 2021-08-25 **Study First Submit QC Date:** 2021-09-06 ### Sponsor Collaborators Module #### Collaborators **Class:** UNKNOWN **Name:** For Ever Fabien #### Lead Sponsor **Class:** OTHER **Name:** Nantes University Hospital #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** Medical procedures can be a very frightening experience for children. It is known that children who received painful medical procedures can develop a higher sensitivity of pain during their following experiences. During their treatments for malignant diseases, children are exposed to a lot of painful procedures (eg. needle insertion, lumbar punction, myelogram, etc...) Therefore, medical societies propose the use of interventions like distraction techniques for pain management in complement of pharmacological treatment. In addition, the repetitions of painful procedures and ineffective prevention of pain can create care phobia. Within this context, immersive and participative virtual reality (VR) could be an innovative distraction technique for pain management among children undergoing medical procedures. Attention Pain Theory can explain how virtual reality can reduce the perception of pain. Attention is required to feel pain. When the patient is focused on another subject like an immersive virtual environment, his brain is less available to treat information like painful stimulus from care procedures. The investigators hypothesis is that VR can reduce procedural-related pain and can decrease fear during the following procedures. Results of previous studies are varied : some showed a non-significant reduction of patient's procedural pain despite the use of VR, whereas others concluded to a decrease of pain. The question of the benefit of VR for the patients who are exposed to repeated painful procedures remains still unclear, especially with patients who are likely to feel chronic pain or many pain-related exposures. The aim of this study is to evaluate the non-inferiority of virtual reality as a distraction technique for pain management in children and adolescents with onco-hematological diseases, undergoing painful procedures, compared to standard of care. **Detailed Description:** VIRTUOSO is a study that aims to evaluate the impact of using a virtual reality headset to prevent care-induced pain in pediatric oncology. This study plans to include 34 patients. It is a single-center, non-inferiority, controlled, with cross-over study. Efficiency of VR will be evaluated for each patient across 3 potentially painful care-procedures : For the first treatment, the child will benefit usual distraction and pain prevention techniques. For the 2nd treatment, the child will use a VR headset as well as pain prevention techniques (excluding oxygen-nitrous oxide mixtures). The child will choose the application he/she wishes to use according to his/her age. For the 3rd treatment, the child will choose his/her favorite technique. The virtual reality headset is an Occulus Quest headset, composed of 2 integrated earphones. It is adapted to the environment and can be used sitting or standing. Usual pharmacological techniques for pain prevention will be maintained as per local practice. All concomitant treatments are authorized except the use of oxygen-nitrous oxide mixtures. The duration of this study is approximately 20 months. Inclusion will be for 10 months, patient follow-up for 6 months. Data analysis will be performed during 4 months. During each treatment, if the child wishes to stop VR, the helmet will be removed and the usual techniques will be offered. After each procedure, a self-assessment of the pain will be done immediately, using VAS. At the end of the third treatment, a short questionnaire will be proposed to the child in order to collect the reasons for his choice. Another questionnaire will also be proposed to the parent present during the care. A qualitative component will explore the social representations of the parents. A range of semi-directive interviews will be conducted with 6 families by a trained professional under the supervision of the methodologist. ### Conditions Module **Conditions:** - Hematologic Malignancy - Hematologic Non Malignancy - Solid Tumor, Childhood - Child, Only **Keywords:** - Pediatric - Pediatric oncology - Pain - Virtual reality - Nurse ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Count:** 34 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Efficiency of VR will be evaluated for each patient across 3 potentially painful care-procedures : For the first treatment, the child will benefit usual distraction and pain prevention techniques. For the 2nd, the child will use a VR headset as well as pain prevention techniques (excluding oxygen-nitrous oxide mixtures). The child will choose the application he/she wishes to use according to his/her age and parental agreement. For the 3rd treatment, the child will choose his/her favorite technique. **Intervention Names:** - Other: Usual distraction and pain prevention techniques. - Other: Virtual reality - Other: Choice between usual distraction or virtual reality **Label:** Standard treatment then virtual reality then choice between the two **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Standard treatment then virtual reality then choice between the two **Description:** For the first painful procedure, the child will benefit usual distraction and pain prevention techniques.Pain prevention and management techniques commonly used in the conduct of care: * Application of a patch or anaesthetic cream 1 hour before the treatment * Vigilant sedation using a premedication for lumbar punctures: Midazolam /Nalbuphine/Hydroxyzine/Morphine. * Apart from care using VR, MEOPA can be given to the child for all assessed care on prescription. * Apart from care using VR, the usual distraction techniques are left to the patient's choice (electronic tablet, smartphone, games, songs...) * The presence of the parents **Name:** Usual distraction and pain prevention techniques. **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - Standard treatment then virtual reality then choice between the two **Description:** For the 2nd painful procedure, the child will use a VR (virtual reality) headset as well as pain prevention techniques (excluding oxygen-nitrous oxide mixtures). The child will choose the application he/she wishes to use according to his/her age. **Name:** Virtual reality **Type:** OTHER #### Intervention 3 **Arm Group Labels:** - Standard treatment then virtual reality then choice between the two **Description:** For the 3rd painful procedure, the child will choose his/her favorite technique between : Usual distraction and pain prevention techniques or virtual reality **Name:** Choice between usual distraction or virtual reality **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Pain levels measured on the VAS. VAS : Visual Analogue Scale . Minimum value : 0. Maximum value : 10. The maximum value is the worse outcome. **Measure:** Pain level measured by VAS (self-evaluation) during the first care using standard treatment. **Time Frame:** Immediately after the first care. **Description:** Pain levels measured on the VAS. VAS : Visual Analogue Scale . Minimum value : 0. Maximum value : 10. The maximum value is the worse outcome. **Measure:** Pain level measured by VAS (self-evaluation) during the second care using virtual reality **Time Frame:** Immediately after the second care . **Description:** Pain levels measured on the VAS. VAS : Visual Analogue Scale . Minimum value : 0. Maximum value : 10. The maximum value is the worse outcome. **Measure:** Pain level measured by VAS (self-evaluation) during the third care using virtual reality or standard treatment **Time Frame:** Immediately after the third care . #### Secondary Outcomes **Description:** Patient preference between VR and usual pain prevention technique for the same care. The difference between the pain level measured on a VAS with the use of VR and the pain level with the usual technique. VAS : Visual Analogue Scale . Minimum value : 0. Maximum value : 10. The maximum value is the worse outcome. **Measure:** Percentage of children who choose VR in the 3rd care **Time Frame:** Immediately after the 3rd intervention. **Description:** Analysis of the answers to the patient questionnaire after treatment n°3. Description of the reasons for selecting the preferred technique. **Measure:** Patient questionnaire **Time Frame:** Immediately after the 3rd intervention. **Description:** Record of side effects reported in the e-CRF. Description of the potential side effects to the use of VR in care. **Measure:** Side effects to the use of VR **Time Frame:** During each intervention with virtual reality (care 2 and care 3 if VR is chosen). Up to 6 months. **Description:** Direct observation of the care unit. Understand the obstacles and levers for professionals to implement a new pain prevention device during care. **Measure:** Ethnographic approach to caregivers' experiences after using the VR headset. **Time Frame:** Through study completion - up to 20 months. **Description:** Analysis of the responses to the parent questionnaire. Understand parents' experience of care when implementing a new pain prevention device during care. **Measure:** parent questionnaire. **Time Frame:** Immediately after the 3rd intervention. **Description:** Understand the social representations of parents and children regarding the use of a virtual reality device. **Measure:** Thematic analysis of the verbatim collected during the semi-directive interviews with the parents after Care 3 **Time Frame:** Immediately after the 3rd intervention. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: Quantitative component: * Child aged 7 to 18 years * Child followed in pediatric onco-hematology in the context of hematological malignancy or non malignant hematology or solid tumor management. * Child who has already experienced a potentially painful care procedure before entry the study * Child who will have the same potentially painful care procedure (Hüber needle placement or lumbar puncture) on three occasions during the course of their care. * Child with consent. * Child whose parental authority holders have given their consent to participate to the study. Qualitative component: * Professional who used both care-induced pain prevention techniques and agreed to be interviewed. * A parent or guardian. Exclusion Criteria: * Child who refuse to use this distraction technique (VR). * Child who refuse to not use MEOPA during VR. * Child who hasn't experienced the potentially painful care procedure that will be done during the study at least once before inclusion * Child who does not speak French and whose parents do not speak French **Maximum Age:** 18 Years **Minimum Age:** 7 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Thomas JEZEQUEL **Phone:** (0)2.76.64.38.63 **Phone Ext:** +33 **Role:** CONTACT #### Locations **Location 1:** **City:** Nantes **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Thomas Jezequel - **Role:** CONTACT **Country:** France **Facility:** Nantes CHU **Status:** RECRUITING **Zip:** 44000 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009369 - Term: Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000006402 - Term: Hematologic Diseases - ID: D000010146 - Term: Pain - ID: D000009461 - Term: Neurologic Manifestations ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC15 - Name: Blood and Lymph Conditions - Abbrev: BC10 - Name: Nervous System Diseases ### Condition Browse Module - Browse Leaves - ID: M13066 - Name: Pain - Relevance: LOW - As Found: Unknown - ID: M1219 - Name: Pain, Procedural - Relevance: HIGH - As Found: Procedural Pain - ID: M21314 - Name: Hematologic Neoplasms - Relevance: HIGH - As Found: Hematologic Malignancies - ID: M9490 - Name: Hematologic Diseases - Relevance: LOW - As Found: Unknown - ID: M12404 - Name: Neurologic Manifestations - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000019337 - Term: Hematologic Neoplasms - ID: D000073818 - Term: Pain, Procedural ### Intervention Browse Module - Browse Branches - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Analg - Name: Analgesics - Abbrev: AdjAn - Name: Adjuvants, Anesthesia - Abbrev: PsychDr - Name: Psychotropic Drugs - Abbrev: Derm - Name: Dermatologic Agents ### Intervention Browse Module - Browse Leaves - ID: M4107 - Name: Anesthetics - Relevance: LOW - As Found: Unknown - ID: M11982 - Name: Morphine - Relevance: LOW - As Found: Unknown - ID: M11845 - Name: Midazolam - Relevance: LOW - As Found: Unknown - ID: M12546 - Name: Nitrous Oxide - Relevance: LOW - As Found: Unknown - ID: M12217 - Name: Nalbuphine - Relevance: LOW - As Found: Unknown - ID: M9970 - Name: Hydroxyzine - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03789279 **Acronym:** RATATOUILLE **Brief Title:** Observational Study of Hand Function After Distal Transradial Access for Angiography **Official Title:** RATATOUILLE Study (inteRnATional hAnd funcTiOn stUdy Following dIstaL radiaL accEss) #### Organization Study ID Info **ID:** 18-WS-0182 #### Organization **Class:** OTHER **Full Name:** NHS National Waiting Times Centre Board #### Secondary ID Infos **Domain:** IRAS **ID:** 245250 **Type:** OTHER ### Status Module #### Completion Date **Date:** 2020-12-31 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-03-09 **Type:** ACTUAL **Last Update Submit Date:** 2021-03-07 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2020-12-31 **Type:** ACTUAL #### Start Date **Date:** 2019-01-01 **Type:** ACTUAL **Status Verified Date:** 2021-03 #### Study First Post Date **Date:** 2018-12-28 **Type:** ACTUAL **Study First Submit Date:** 2018-12-26 **Study First Submit QC Date:** 2018-12-27 ### Sponsor Collaborators Module #### Collaborators **Class:** UNKNOWN **Name:** MC Zuiderzee #### Lead Sponsor **Class:** OTHER **Name:** NHS National Waiting Times Centre Board #### Responsible Party **Investigator Affiliation:** University of Glasgow **Investigator Full Name:** Dr Thomas J Ford **Investigator Title:** Honorary Clinical Lecturer **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Traditionally, coronary angiograms are performed through the radial artery which is accessed above the palm of the 'right' hand. In recent years, some cardiologists are performing this procedure from the back of the wrist in as the radial artery courses through the anatomical snuffbox (distal radial access). The aim of this study is to determine the prevalence of hand dysfunction following coronary angiography via the distal radial artery. **Detailed Description:** In this multi centre observational registry, we will recruit patients undergoing planned invasive coronary angiography. Inclusion criteria include: 1. Age ≥ 18 years. 2. The distal radial artery must be palpable and non-occlusive flow must be confirmed by Doppler ultrasound. 3. Patient should be able to comply with the protocol. 4. Provide written informed consent before study participation. The primary endpoint is the overall prevalence of hand dysfunction defined as any significant reduction from baseline score in any of the following five domains: DASH score Levine Katz score VAS score Sensory function Pinch grip strength. Specifically hand function will be assessed at 4 time points using simple tests of hand strength, sensation and ultrasound to assess the arterial latency. ### Conditions Module **Conditions:** - Radial Artery Occlusion - Nerve Injury **Keywords:** - Invasive coronary angiography - Distal transradial arterial access - Anatomical snuffbox - dTRA - dRA - Distal radial - Radial artery occlusion ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 40 **Type:** ACTUAL **Patient Registry:** True **Study Type:** OBSERVATIONAL **Target Duration:** 12 Months ### Arms Interventions Module #### Arm Group 1 **Description:** Patients undergoing invasive coronary angiography via the distal radial approach (anatomical snuffbox) **Label:** Distal transradial arterial access ### Outcomes Module #### Primary Outcomes **Description:** Any significant deterioration from baseline in hand function according to the 5 studied domains. **Measure:** Prevalence of hand dysfunction **Time Frame:** 1 month #### Secondary Outcomes **Description:** Successful introduction of sheath **Measure:** Success of distal radial access **Time Frame:** Day 0 **Description:** Surgical complications or clinically important vascular access complications **Measure:** Vascular access complications (other than occlusion and bleeding) **Time Frame:** Day 0 **Description:** Time from skin puncture to successful placement of wire into the artery **Measure:** Puncture time **Time Frame:** Day 0 **Description:** USS guide **Measure:** Radial artery occlusion - including level of occlusion (prox/distal) **Time Frame:** 0-12 months **Description:** Minutes **Measure:** Fluoroscopy time **Time Frame:** Day 0 **Description:** Minutes **Measure:** Hemostasis duration **Time Frame:** Day 0 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Age ≥ 18 years. 2. The distal radial artery must be palpable and non-occlusive flow must be confirmed by Doppler ultrasound. 3. Patient should be able to comply with the protocol. 4. Provide written informed consent before study participation. Exclusion Criteria: 1. Obligatory femoral or forearm radial access 2. Previous ipsilateral forearm radial artery occlusion. 3. Undergoing another procedure involving the ipsilateral radial artery, performed between the index procedure and the follow-up date. 4. Enrolment in another study that competes or interferes with this study. 5. Poor clinical condition like cardiogenic shock, that prohibits pre- and postprocedural function tests. 6. Any other condition which, in the opinion of the investigator or operator, may pose a significant hazard to the subject if he or she is enrolled in the study. 7. Co-morbidity that excludes patient follow-up. **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Eligible patients for inclusion are those by whom coronary angiography or angioplasty is indicated as part of clinical decision-making or therapy. Operators with adequate experience at the discretion of the principal investigator in distal radial access are eligible to participate. ### Contacts Locations Module #### Locations **Location 1:** **City:** Glasgow **Country:** United Kingdom **Facility:** University of Glasgow/Golden Jubilee Research Foundation **Zip:** G12 8QQ #### Overall Officials **Official 1:** **Affiliation:** MC Zuiderzee **Name:** Ferdinand Kiemeneij, MD **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** MC Zuiderzee **Name:** Ahmed Hassan, MD **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014652 - Term: Vascular Diseases - ID: D000002318 - Term: Cardiovascular Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M4465 - Name: Arterial Occlusive Diseases - Relevance: HIGH - As Found: Artery Occlusion - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001157 - Term: Arterial Occlusive Diseases ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00739479 **Brief Title:** CCRC: Effects of Partially Hydrolyzed Whey Peptides (PHWP) On Weight Loss In Individuals With The Metabolic Syndrome (METS) **Official Title:** CCRC: Effects of Partially Hydrolyzed Whey Peptides (PHWP) On Weight Loss In Individuals With The Metabolic Syndrome (METS) #### Organization Study ID Info **ID:** 200816190-1 #### Organization **Class:** OTHER **Full Name:** University of California, Davis ### Status Module #### Completion Date **Date:** 2010-08 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2012-10-29 **Type:** ESTIMATED **Last Update Submit Date:** 2012-10-25 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2010-08 **Type:** ACTUAL #### Start Date **Date:** 2008-08 **Status Verified Date:** 2008-08 #### Study First Post Date **Date:** 2008-08-21 **Type:** ESTIMATED **Study First Submit Date:** 2008-08-19 **Study First Submit QC Date:** 2008-08-19 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** California Dairy Research Foundation #### Lead Sponsor **Class:** OTHER **Name:** Sidika E. Karakas, MD #### Responsible Party **Investigator Affiliation:** University of California, Davis **Investigator Full Name:** Sidika E. Karakas, MD **Investigator Title:** Principal Investigator **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The aim of this study is to compare the effects of two different protein supplements (partially hydrolyzed whey protein, PHWP vs. partially hydrolyzed gelatin, PHG) on weight loss in obse individuals with metabolic syndrome (METS). These two supplements will contain equal amounts of protein but differ considerably in their amino acid contents. Whey protein is rich in essential amino acids whereas gelatin is rich in proline. In obese individuals with METS, the hypotheses are: * PHWP will augment fat-mass loss and increase lean-mass to fat-mass ration more than PHG. * PHWP will improve insulin action more than PHG. * PHWP will decrease cardiovascular disease risk more than PHG. **Detailed Description:** The Metabolic Syndrome (METS) is a clinical disorder characterized by the following problems: Obesity, especially located in the waist area, elevated blood fats (lipids), high blood pressure and insulin resistance. The METS affects one third of the adult population in the USA and increases the risks for both diabetes and hardening of the arteries, leading to heart attacks and strokes. The best treatment for improving the symptoms of METS is weight loss. In previous studies, it has been demonstrated that whey protein (WP) supplementation increased weight loss and especially fat-mass loss in obese, insulin resistant women when compared to carbohydrates. We now propose to compare WP to another protein source in patients with the METS during weight loss. In addition, we will use a special preparation called partially hydrolyzed whey protein, which may have specific properties that increase fat mass loss. ### Conditions Module **Conditions:** - Metabolic Syndrome - Overweight **Keywords:** - Metabolic Syndrome - METS - Obesity - Partially Hydrolyzed Whey Protein - Partially Hydrolyzed Gelatin - PHWP - PHG - Weight Loss ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - INVESTIGATOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 35 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients will be randomized to receive PHWP. Since sex and baseline weight can influence the response, randomization will be stratified according to these variables. **Intervention Names:** - Dietary Supplement: Weight Loss **Label:** 1 **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Patients will be randomized to receive PHG. Since sex and baseline weight can influence the response, randomization will be stratified according to these variables. **Intervention Names:** - Dietary Supplement: Weight Loss **Label:** 2 **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - 1 - 2 **Description:** Participants will reduce their total dietary intake by \~800 kcal/day. \~100 kcal will be replaced by either the PHWP of PHG protein supplement, resulting in a total energy restriction of \~700 kcal/day. **Name:** Weight Loss **Type:** DIETARY_SUPPLEMENT ### Outcomes Module #### Primary Outcomes **Measure:** The investigators hope to determine the effects of incorporating PHWP into the diet of a person diagnosed with METS. **Time Frame:** 2 Years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Clinical Diagnoses of Metabolic Syndrome (at least 3/5 of the following) * Waist Circumference: men: \>40 in women: \>35 in * Blood Pressure: \>135/\>85 mm Hg * Triglycerides: \>150 mg/dl * HDL-cholesterol: men: \<40 mg/dl women: \<50 mg/dl * Fasting Glucose: \>100 mg/dl * Ages 18 to 65 Years * BMI range of 27 to 42 kg/m\^2 * Body weight \<300 lbs * Weight Stable for 3 Months Exclusion Criteria: * Subjects who habitually consume protein supplements or have eating disorders * Recent delivery (within 12 months), lactation, pregnancy or intention to become pregnant * Type 2 diabetes, kidney disease, liver disease, anemia, gout, cancer, untreated thyroid disease, gastrointestinal disease, other metabolic diseases or malabsorption syndromes * Triglyceride \>500 mg/dl, Cholesterol \>260 mg/dl * Use of insulin sensitizers, lipid lowering medication or ACE inhibitors * Use of anti-obesity medications or supplements for at lease 6 months prior to start of study * Known allergy or adverse reaction to protein and dairy products (including lactose) **Maximum Age:** 65 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Sacramento **Country:** United States **Facility:** UC Davis CCRC **State:** California **Zip:** 95616 #### Overall Officials **Official 1:** **Affiliation:** University of California, Davis **Name:** Sidika E Kasim-Karakas, MD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Piccolo BD, Comerford KB, Karakas SE, Knotts TA, Fiehn O, Adams SH. Whey protein supplementation does not alter plasma branched-chained amino acid profiles but results in unique metabolomics patterns in obese women enrolled in an 8-week weight loss trial. J Nutr. 2015 Apr;145(4):691-700. doi: 10.3945/jn.114.203943. Epub 2015 Feb 4. **PMID:** 25833773 **Citation:** Comerford KB, Buchan W, Karakas SE. The effects of weight loss on FABP4 and RBP4 in obese women with metabolic syndrome. Horm Metab Res. 2014 Mar;46(3):224-31. doi: 10.1055/s-0033-1353204. Epub 2013 Aug 26. **PMID:** 23979787 #### See Also Links **Label:** UC Davis Website - Featured Clinical Trials **URL:** http://www.ucdmc.ucdavis.edu/clinicaltrials/ ## Derived Section ### Condition Browse Module - Ancestors - ID: D000004194 - Term: Disease - ID: D000010335 - Term: Pathologic Processes - ID: D000044343 - Term: Overnutrition - ID: D000009748 - Term: Nutrition Disorders - ID: D000001835 - Term: Body Weight - ID: D000001836 - Term: Body Weight Changes - ID: D000007333 - Term: Insulin Resistance - ID: D000006946 - Term: Hyperinsulinism - ID: D000044882 - Term: Glucose Metabolism Disorders - ID: D000008659 - Term: Metabolic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases ### Condition Browse Module - Browse Leaves - ID: M5114 - Name: Body Weight - Relevance: LOW - As Found: Unknown - ID: M18102 - Name: Weight Loss - Relevance: HIGH - As Found: Weight Loss - ID: M16355 - Name: Syndrome - Relevance: HIGH - As Found: Syndrome - ID: M12701 - Name: Obesity - Relevance: LOW - As Found: Unknown - ID: M26186 - Name: Overweight - Relevance: HIGH - As Found: Overweight - ID: M23005 - Name: Metabolic Syndrome - Relevance: HIGH - As Found: Metabolic Syndrome - ID: M25307 - Name: Overnutrition - Relevance: LOW - As Found: Unknown - ID: M12684 - Name: Nutrition Disorders - Relevance: LOW - As Found: Unknown - ID: M5115 - Name: Body Weight Changes - Relevance: LOW - As Found: Unknown - ID: M10370 - Name: Insulin Resistance - Relevance: LOW - As Found: Unknown - ID: M9997 - Name: Hyperinsulinism - Relevance: LOW - As Found: Unknown - ID: M11639 - Name: Metabolic Diseases - Relevance: LOW - As Found: Unknown - ID: M25403 - Name: Glucose Metabolism Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000050177 - Term: Overweight - ID: D000024821 - Term: Metabolic Syndrome - ID: D000013577 - Term: Syndrome - ID: D000015431 - Term: Weight Loss ### Intervention Browse Module - Browse Branches - Abbrev: Ot - Name: Other Dietary Supplements - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: T435 - Name: Whey Protein - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04591379 **Brief Title:** Intratumoral Influenza Vaccine for Early Colorectal Cancer **Official Title:** Intratumoral Influenza Vaccine for Early Colorectal Cancer #### Organization Study ID Info **ID:** REG-083-2020 #### Organization **Class:** OTHER **Full Name:** Zealand University Hospital ### Status Module #### Completion Date **Date:** 2021-11-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2022-12-13 **Type:** ACTUAL **Last Update Submit Date:** 2022-12-12 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2021-09-22 **Type:** ACTUAL #### Start Date **Date:** 2021-02-26 **Type:** ACTUAL **Status Verified Date:** 2022-12 #### Study First Post Date **Date:** 2020-10-19 **Type:** ACTUAL **Study First Submit Date:** 2020-09-07 **Study First Submit QC Date:** 2020-10-13 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** University of Copenhagen #### Lead Sponsor **Class:** OTHER **Name:** Zealand University Hospital #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Is US Export:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** The aim of this explorative phase II clinical trial is to establish the safety and efficacy of intratumoral influenza vaccine in patients with colorectal cancer, as an additive treatment prior to intended curative surgery. **Detailed Description:** This is an explorative phase 2 clinical trial which will be conducted in two phases. The aim of this study is to establish the safety and efficacy of treating patients with early colorectal cancer with intratumoral influenza vaccine as a down staging and immune response enhancing treatment prior to intended curative surgery. The first part of the study will be conducted as a pilot study. Six patients with histologically verified or clinically suspicious sigmoid colon cancer who are planned to undergo curative surgery will be included. Patients will be recruited from the Department of Surgery, Zealand University Hospital after their case has been reviewed by the multidisciplinary team (MDT). Standard treatment involves intended curative surgery within two weeks after the diagnosis. The treatment will be performed within a few days and it will be ensured that the experimental treatment will not lead to a significant delay of intended curative surgery. If the pilot study finishes without violating any stop rules and without any serious adverse events the second part of the study will be initiated. This will be conducted as a phase 2 study where 24 patients with histologically verified or clinically suspicious sigmoid colon cancer and rectal cancer will be included. Patients will be recruited from the Department of Surgery, Zealand University Hospital after their case has been reviewed by the multidisciplinary team (MDT). Standard treatment involves intended curative surgery within two weeks after the diagnosis. The treatment will be performed within a few days and it will be ensured that the experimental treatment will not lead to a significant delay of intended curative surgery. ### Conditions Module **Conditions:** - Colorectal Cancer ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 10 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Drug: Influenza Vaccines **Label:** Intervention arm **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Intervention arm **Description:** Intratumoral application of an unattenuated influenza vaccine **Name:** Influenza Vaccines **Type:** DRUG ### Outcomes Module #### Other Outcomes **Description:** To investigate if the treatment will induce a systemic immunologic response. **Measure:** Efficacy - systemic immunological changes **Time Frame:** Blood samples from the day of treatment (day 0), before surgery (day 7-14 after treatment), after surgery (postOP day 1-2) and postoperative follow-up (postOP day 12-16) **Description:** To assess quality of recovery for patients recruited into this trial. A quality of recovery questionnaire (QoR-15) will be given to patients pre- and post-treatment. **Measure:** Quality of recovery **Time Frame:** Assessed before treatment (day 0), before surgery (day 7-14 after treatment), after surgery (postOP day 1) and postoperative follow-up (postOP day 12-16) #### Primary Outcomes **Description:** To investigate if intratumoral influenza vaccine is a safe treatment modality for tumor down staging prior to intended curative surgery in patients undergoing treatment for colorectal cancer. Adverse events / reactions are recorded from day of treatment (Day 0) until the surgery, as it will be difficult to differ between adverse events/reactions to the experimental treatment or surgery. All adverse events / reactions should be described in medical terminology in the patient's file and recorded in case report forms (CRF). The following information must be recorded: start date/date when observed, severity, any initiated treatment, assessment of the AE if it meets the criteria for SAE, end date, and relationship to study drug. For AEs that meet the criteria for SAE, the outcome must be recorded. **Measure:** Safety - Adverse reactions are classified according to CTCAE version 4.0 **Time Frame:** Day of surgery (day 7-14 after treatment) #### Secondary Outcomes **Description:** To investigate if intratumoral influenza vaccine will induce immunologic invasion of the primary tumor This will be analyzed by immunhistochemistry and NanoString **Measure:** Efficacy - local immunological changes **Time Frame:** Pathological specimens from day of treatment (day 0) and surgery (day 7-14 after treatment) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patients must be mentally capable of understanding the information given. * Patients must give written informed consent. * Clinically suspected or histologically verified malignant tumor of the rectum or sigmoid colon. * Tumor described as passable at index endoscopy. * Men or women aged at least 18 years. * Case reviewed by MDT (surgery, radiology, oncology). Case considered curable with standard surgical resection. Exclusion Criteria: * Highly inflamed gastrointestinal tissue which is ulcerated and bleeding * Ongoing immunosuppressive treatment. * Concurrent treatment with an investigational medicinal product. * Patients with any other clinical condition or prior therapy that, in the opinion of the investigator, would make the patient unsuitable for the study or unable to comply with the study recruitments. * Advanced tumor stages, clinical UICC stage IV. * Indication for neoadjuvant chemoradiation or chemotherapy prior to surgery * Acute surgical resection. * Pregnancy * Any previous allergic reaction to influenza vaccine or constituents, egg and chicken proteins, neomycin, formaldehyde or octoxinol-9 * Acute febrile illness * Acute infectious disease * Influenza vaccine administered within 30 days before study inclusion **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Køge **Country:** Denmark **Facility:** Zealand University Hospital **State:** Zealand **Zip:** 4600 #### Overall Officials **Official 1:** **Affiliation:** Zealand University Hospital **Name:** Ismail Gögenur, Professor **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Info Types:** - STUDY_PROTOCOL - ICF **IPD Sharing:** YES **Time Frame:** Study protocol and informed consent form will be available upon start of recruitment of patients ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007414 - Term: Intestinal Neoplasms - ID: D000005770 - Term: Gastrointestinal Neoplasms - ID: D000004067 - Term: Digestive System Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000004066 - Term: Digestive System Diseases - ID: D000005767 - Term: Gastrointestinal Diseases - ID: D000003108 - Term: Colonic Diseases - ID: D000007410 - Term: Intestinal Diseases - ID: D000012002 - Term: Rectal Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC01 - Name: Infections - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC06 - Name: Digestive System Diseases ### Condition Browse Module - Browse Leaves - ID: M10295 - Name: Influenza, Human - Relevance: LOW - As Found: Unknown - ID: M17890 - Name: Colorectal Neoplasms - Relevance: HIGH - As Found: Colorectal Cancer - ID: M10448 - Name: Intestinal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M8886 - Name: Gastrointestinal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7256 - Name: Digestive System Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M6336 - Name: Colonic Diseases - Relevance: LOW - As Found: Unknown - ID: M10444 - Name: Intestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M14844 - Name: Rectal Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000015179 - Term: Colorectal Neoplasms ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M17360 - Name: Vaccines - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00717379 **Brief Title:** Study of Tacrolimus Immunosuppressive Therapy After Kidney Transplantation **Official Title:** Open-Label, Randomized, Multicenter, Parallel-Group Efficacy and Safety Study of Tacrolimus Immunosuppressive Therapy After Kidney Transplantation #### Organization Study ID Info **ID:** PRG-EC-2R01 #### Organization **Class:** INDUSTRY **Full Name:** Astellas Pharma Inc ### Status Module #### Completion Date **Date:** 2008-10 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2009-04-15 **Type:** ESTIMATED **Last Update Submit Date:** 2009-04-13 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2008-10 **Type:** ACTUAL #### Start Date **Date:** 2007-05 **Status Verified Date:** 2009-04 #### Study First Post Date **Date:** 2008-07-17 **Type:** ESTIMATED **Study First Submit Date:** 2008-07-16 **Study First Submit QC Date:** 2008-07-16 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Astellas Pharma Inc #### Responsible Party **Old Name Title:** Disclosure Office Europe **Old Organization:** Astellas Pharma Europe BV ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** To compare the efficacy and safety of Tacrolimus in combination with MMF and Steroids in two regimens of steroid in an adult kidney transplanted population. ### Conditions Module **Conditions:** - Kidney Transplantation - Kidney Failure, Chronic - Renal Insufficiency, Chronic **Keywords:** - Organ Transplantation - Tacrolimus - Prograf - Kidney - Immunosuppression ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 50 **Type:** ACTUAL **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** steroid regimen 1 **Intervention Names:** - Drug: Tacrolimus - Drug: Mycophenolate Mofetil - Drug: Methylprednisolone or equivalent - Drug: Prednisone **Label:** 1 **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** steroid regimen 2 **Intervention Names:** - Drug: Tacrolimus - Drug: Mycophenolate Mofetil - Drug: Methylprednisolone or equivalent - Drug: Prednisone **Label:** 2 **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - 1 - 2 **Description:** oral **Name:** Tacrolimus **Other Names:** - Prograf - FK506 **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - 1 - 2 **Description:** oral **Name:** Mycophenolate Mofetil **Other Names:** - MMF **Type:** DRUG #### Intervention 3 **Arm Group Labels:** - 1 - 2 **Description:** oral **Name:** Methylprednisolone or equivalent **Type:** DRUG #### Intervention 4 **Arm Group Labels:** - 1 - 2 **Description:** oral **Name:** Prednisone **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Incidence and time to first biopsy-proven acute rejection **Time Frame:** 6 months #### Secondary Outcomes **Measure:** Overall frequency of acute rejection episodes within 6 months post transplantation **Time Frame:** 6 months **Measure:** Severity of biopsy proven acute rejections (BANFF criteria) within 6 months post transplantation **Time Frame:** 6 months **Measure:** Incidence of and time to first corticosteroid-resistant acute rejection **Time Frame:** 6 months **Measure:** Subject and graft survival **Time Frame:** 6 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Female subject of childbearing potential must have a negative serum pregnancy test at enrolment and must agree to maintain effective birth control during the study * Has an end stage kidney disease and is a suitable candidate for primary renal transplantation or retransplantation * Subject is receiving a kidney transplant, from a cadaveric or living donor between 5 and 65 years of age with compatible AB0 blood type Exclusion Criteria: * Pregnant woman or breast-feeding mother * Has an immunological high risk, defined as having a most recently measured PRA grade of \> 50% within the previous six months * Known allergy to the study drug or any of its components * Requires ongoing dosing with a systemic immunosuppressive drug at study entry for any reason other than kidney transplantation * Requires initial sequential or parallel therapy with immunosuppressive antibody preparation(s) * Subject or donor is known to be HIV positive * Has significant liver disease, defined as having during the past 28 days continuously elevated ASAT (SGOT) and/or ALAT (SGPT) levels greater than 3 times the upper value of the normal range of the investigational site * Diagnosis of malignancy or history of malignancy, except non metastatic basal or squamous cell carcinoma of the skin that has been treated successfully * Has significant, uncontrolled concomitant infections and/or severe diarrhoea, vomiting, or active peptic ulcer * Previously received or is receiving an organ transplant other than kidney * Receiving a graft from a non-heart-beating donor * Cold ischemia time of the donor kidney \>30 hours * Any form of substance abuse, psychiatric disorder or condition which, in the opinion of the investigator, may complicate communication with the investigator **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Moscow **Country:** Russian Federation **Zip:** 115446 **Location 2:** **City:** Moscow **Country:** Russian Federation **Zip:** 119992 **Location 3:** **City:** Moscow **Country:** Russian Federation **Zip:** 123182 **Location 4:** **City:** Omsk **Country:** Russian Federation **Zip:** 644112 **Location 5:** **City:** St. Petersburg **Country:** Russian Federation **Zip:** 197110 **Location 6:** **City:** Volzskii **Country:** Russian Federation **Zip:** 404120 #### Overall Officials **Official 1:** **Affiliation:** ZAO Astellas Pharma Russia **Name:** Central Contact **Role:** STUDY_DIRECTOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007674 - Term: Kidney Diseases - ID: D000014570 - Term: Urologic Diseases - ID: D000052776 - Term: Female Urogenital Diseases - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications - ID: D000091642 - Term: Urogenital Diseases - ID: D000052801 - Term: Male Urogenital Diseases - ID: D000002908 - Term: Chronic Disease - ID: D000020969 - Term: Disease Attributes - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M26717 - Name: Renal Insufficiency, Chronic - Relevance: HIGH - As Found: Renal Insufficiency, Chronic - ID: M10699 - Name: Kidney Failure, Chronic - Relevance: HIGH - As Found: Kidney Failure, Chronic - ID: M26718 - Name: Renal Insufficiency - Relevance: HIGH - As Found: Renal Insufficiency - ID: M10698 - Name: Kidney Diseases - Relevance: LOW - As Found: Unknown - ID: M17319 - Name: Urologic Diseases - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M27095 - Name: Male Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M6147 - Name: Chronic Disease - Relevance: LOW - As Found: Unknown - ID: M22700 - Name: Disease Attributes - Relevance: LOW - As Found: Unknown - ID: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: HIGH - As Found: Chronic ### Condition Browse Module - Meshes - ID: D000051437 - Term: Renal Insufficiency - ID: D000007676 - Term: Kidney Failure, Chronic - ID: D000051436 - Term: Renal Insufficiency, Chronic ### Intervention Browse Module - Ancestors - ID: D000000893 - Term: Anti-Inflammatory Agents - ID: D000005938 - Term: Glucocorticoids - ID: D000006728 - Term: Hormones - ID: D000006730 - Term: Hormones, Hormone Substitutes, and Hormone Antagonists - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018931 - Term: Antineoplastic Agents, Hormonal - ID: D000000970 - Term: Antineoplastic Agents - ID: D000007166 - Term: Immunosuppressive Agents - ID: D000007155 - Term: Immunologic Factors - ID: D000065095 - Term: Calcineurin Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000000903 - Term: Antibiotics, Antineoplastic - ID: D000000904 - Term: Antibiotics, Antitubercular - ID: D000000995 - Term: Antitubercular Agents - ID: D000000900 - Term: Anti-Bacterial Agents - ID: D000000890 - Term: Anti-Infective Agents - ID: D000000932 - Term: Antiemetics - ID: D000001337 - Term: Autonomic Agents - ID: D000018373 - Term: Peripheral Nervous System Agents - ID: D000005765 - Term: Gastrointestinal Agents - ID: D000018696 - Term: Neuroprotective Agents - ID: D000020011 - Term: Protective Agents ### Intervention Browse Module - Browse Branches - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: AnEm - Name: Antiemetics - Abbrev: NeuroAg - Name: Neuroprotective Agents - Abbrev: Gast - Name: Gastrointestinal Agents ### Intervention Browse Module - Browse Leaves - ID: M14121 - Name: Prednisone - Relevance: HIGH - As Found: Min - ID: M14120 - Name: Prednisolone - Relevance: LOW - As Found: Unknown - ID: M12128 - Name: Mycophenolic Acid - Relevance: HIGH - As Found: Mindfulness - ID: M18950 - Name: Tacrolimus - Relevance: HIGH - As Found: 3 weeks - ID: M11749 - Name: Methylprednisolone - Relevance: HIGH - As Found: Smoking - ID: M1833 - Name: Methylprednisolone Acetate - Relevance: LOW - As Found: Unknown - ID: M11750 - Name: Methylprednisolone Hemisuccinate - Relevance: LOW - As Found: Unknown - ID: M229449 - Name: Prednisolone acetate - Relevance: LOW - As Found: Unknown - ID: M211887 - Name: Prednisolone hemisuccinate - Relevance: LOW - As Found: Unknown - ID: M248881 - Name: Prednisolone phosphate - Relevance: LOW - As Found: Unknown - ID: M10212 - Name: Immunosuppressive Agents - Relevance: LOW - As Found: Unknown - ID: M4217 - Name: Anti-Inflammatory Agents - Relevance: LOW - As Found: Unknown - ID: M9047 - Name: Glucocorticoids - Relevance: LOW - As Found: Unknown - ID: M9789 - Name: Hormones - Relevance: LOW - As Found: Unknown - ID: M9788 - Name: Hormone Antagonists - Relevance: LOW - As Found: Unknown - ID: M20966 - Name: Antineoplastic Agents, Hormonal - Relevance: LOW - As Found: Unknown - ID: M10201 - Name: Immunologic Factors - Relevance: LOW - As Found: Unknown - ID: M30452 - Name: Calcineurin Inhibitors - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown - ID: M4222 - Name: Anti-Bacterial Agents - Relevance: LOW - As Found: Unknown - ID: M4224 - Name: Antibiotics, Antitubercular - Relevance: LOW - As Found: Unknown - ID: M4311 - Name: Antitubercular Agents - Relevance: LOW - As Found: Unknown - ID: M4214 - Name: Anti-Infective Agents - Relevance: LOW - As Found: Unknown - ID: M4251 - Name: Antiemetics - Relevance: LOW - As Found: Unknown - ID: M8881 - Name: Gastrointestinal Agents - Relevance: LOW - As Found: Unknown - ID: M20773 - Name: Neuroprotective Agents - Relevance: LOW - As Found: Unknown - ID: M21869 - Name: Protective Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000009173 - Term: Mycophenolic Acid - ID: D000011241 - Term: Prednisone - ID: D000008775 - Term: Methylprednisolone - ID: D000016559 - Term: Tacrolimus ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01294579 **Brief Title:** Ofatumumab and Bendamustine Followed by Maintenance Ofatumumab for Rituximab Relapsed Indolent B-cell Non-Hodgkin's Lymphoma (B-NHL) **Official Title:** A Phase II Open-Label Study of Ofatumumab and Bendamustine Followed by Maintenance Ofatumumab for Indolent B-cell Non-Hodgkin's Lymphoma (B-NHL) Which Has Relapsed After Rituximab or a Rituximab Containing Therapy #### Organization Study ID Info **ID:** 114612 #### Organization **Class:** INDUSTRY **Full Name:** Novartis ### Status Module #### Completion Date **Date:** 2016-12-20 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-08-09 **Type:** ACTUAL **Last Update Submit Date:** 2018-08-06 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2016-12-20 **Type:** ACTUAL #### Results First Post Date **Date:** 2018-08-09 **Type:** ACTUAL **Results First Submit Date:** 2017-12-15 **Results First Submit QC Date:** 2018-08-06 #### Start Date **Date:** 2011-05-17 **Type:** ACTUAL **Status Verified Date:** 2018-08 #### Study First Post Date **Date:** 2011-02-11 **Type:** ESTIMATED **Study First Submit Date:** 2011-02-03 **Study First Submit QC Date:** 2011-02-10 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Novartis Pharmaceuticals #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this phase II open label study was is to evaluate the safety and efficacy of ofatumumab and bendamustine followed by maintenance ofatumumab in subjects with indolent B-NHL who had relapsed after Rituximab treatment. A maximum of 53 subjects at least 18 years old with Small lymphocytic, lymphoplasmacytic, marginal zone lymphoma, or follicular lymphoma; Grades 1, 2 and 3a, would have been enrolled (34 in Stage 1 and 19 in Stage 2). Subjects should have had Rituximab-sensitive disease, defined as a Partial Remission (PR) or Complete Remission (CR) to the last rituximab-containing therapy lasting at least 6 months following completion of therapy or subjects should have relapsed or have had disease progression following response to prior rituximab-based therapy a Eastern Cooperative Oncology Group (ECOG) Performance status of 0 1 or 2. During the induction phase, ofatumumab 1000 mg IV on day 1 of each cycle (cycles 1-6) were followed by Bendamustine 90 mg/m2 IV on days 1, 2 of each cycle (cycles 1-6).During the maintenance phase, subjects with a PR or CR after the induction phase received ofatumumab 1000 mg IV every 2 months for 2 years. ### Conditions Module **Conditions:** - Lymphoma, Non-Hodgkin **Keywords:** - Non-Hodgkin's Lymphoma - Ofatumumab - Relapsed - Rituximab - Bendamustine ### Design Module #### Design Info ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 49 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 1000 mg intravenous (IV) on day 1 of each cycle (cycles 1-6) for induction phase and 1000 mg IV every 2 months for 2 years. Bendamustine 90 mg/m2 was given on day 1 (after the ofatumumab infusion) and day 2 of each cycle (cycles 1-6) **Intervention Names:** - Biological: Ofatumumab - Drug: Bendamustine **Label:** ofatumumab and bendamustine **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - ofatumumab and bendamustine **Description:** 1000 mg intravenous (IV) on day 1 of each cycle (cycles 1-6) for induction phase 1000 mg IV every 2 months for 2 years **Name:** Ofatumumab **Type:** BIOLOGICAL #### Intervention 2 **Arm Group Labels:** - ofatumumab and bendamustine **Description:** 90 mg/m2 on day 1 (after the ofatumumab infusion) and day 2 of each cycle (cycles 1-6) **Name:** Bendamustine **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Complete response (CR) included all of the following: complete disappearance of all detectable clinical evidence of disease and disease-related symptoms if present before therapy. All target nodes had to have regressed to ≤ 1.5cm in the longest diameter. Non-measureable nodes 1.1 to 1.5cm in the longest diameter and \>1cm in the short axis at baseline had to regress to ≤ 1cm in the short axis by visual estimation; enlarged spleen or liver (with nodules) must have returned to normal size and nodules disappeared and if bone marrow was involved, infiltrate had to have cleared on repeat biopsy sample. CR was not valid without imaging data. The corresponding 2-sided 95% exact confidence interval (CI) of the response rate was estimated by the Clopper-Pearson method. **Measure:** Complete Remission (CR) Rate of Induction Therapy After Cycle 6 (28 Days) (FAS) **Time Frame:** Baseline up to 24 weeks #### Secondary Outcomes **Description:** The overall response = CR (defined in Primary Outcome) + Partial Response (PR) which required all of the following: \> or = to 50% decrease from baseline in target nodules; \> or = to 50% decrease in hepatic/splenic nodules and no increase in liver or spleen size; no unequivocal progression in non-target lestions; no new sites of disease. **Measure:** Overall Response Rate (ORR) During Induction Phase After Cycle 6 (FAS) **Time Frame:** Baseline up to 24 weeks **Description:** Rate of conversion from PR in the Induction phase, to CR with maintenance ofatumumab in subjects who have a PR with induction therapy with ofatumumab and bendamustine **Measure:** Conversion Rate of Partial Response in Induction Phase to Complete Response With Maintenance Ofatumumab (FAS) **Time Frame:** Partial response in induction phase up to 24 weeks **Description:** Progression free survival (PFS) is defined as the interval between first treatment and disease progression or death due to any cause. PFS criteria: A previously normal node (≤ 1.5 x ≤ 1.0cm), including nodes that were not previously visible, must increase to \>2.0 x ≥ 1.5cm; ≥ 50% increase from nadir in the PPD of any target node. The long axis must increase by at least 5 mm and to \>2.0cm.; ≥ 50% increase from nadir in the long axis of any target node. The long axis must increase by at least 5 mm and to \>2.0 cm.; ≥ 50% increase from nadir in the SPD of target nodes and at least one node should have a long axis \>1.5 cm. **Measure:** Percentage of Participants With Progression Free Survival (PFS) up to 30 Months (FAS) **Time Frame:** Baseline up to approximately 30 months **Description:** Progression Free Survival (PFS) is defined as the interval between first treatment and disease progression or death due to any cause. PFS events: progression documented between scheduled visits, death before first PD assessment (or death at baseline or prior to any adequate assessments), death between adequate assessment visits. For the PFS analysis, the survival function was estimated using Kaplan-Meier estimates. **Measure:** Kaplan-Meier Estimates of Progression Free Survival up to 30 Months (FAS) **Time Frame:** Baseline up to approximately 30 months **Description:** Due to recruitment issues, this data analysis was not done per changes in planned analysis. This data was only presented as patient listings. The statistical analysis plan was modified to indicate that Pharmacokinetic/Pharmacodynamic exploratory analyses were not done. **Measure:** Pharmacokinetic Profile Which Includes Measuring Blood Levels of Ofatumumab and Bendamustine in Combination and Ofatumumab Alone During Maintenance Treatment and Measuring Blood Levels of Circulating B Cell **Time Frame:** up to 30 months **Description:** Deaths were collected and were considered to be an on treatment death up to 60 days post treatment. **Measure:** All Deaths by Preferred Term (Safety Set) up to Approximately 30 Months **Time Frame:** Baseline up to approximately 30 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Signed written informed consent * Small lymphocytic, lymphoplasmacytic, marginal zone lymphoma, and follicular lymphoma; Grades 1, 2 and 3a, defined according to World Health organization (WHO) guidelines. \[Tefferi, 2008\] * Tumor was verified to be CD20+ (based on local evaluation), from a current or previous tissue biopsy. Tissue biopsy should be repeated if no report or specimen is available, CD20 staining was not previously performed, or there is clinical suspicion that the indolent lymphoma has transformed to aggressive lymphoma/higher malignancy grade. * Rituximab-sensitive disease, defined as a Partial Remission (PR) or Complete Remission (CR) to the last rituximab-containing therapy lasting at least 6 months following completion of therapy. Last rituximab-containing therapy is defined as the last therapy regimen containing at least one full dose of rituximab. * Relapse or disease progression following response to prior rituximab-based therapy, that requiried treatment by 2007 Revised Response Criteria for Malignant Lymphoma (RRCML) guidelines. * CT imaging in screening (based on local evaluation) showing 2 or more clearly demarcated lesions with a largest diameter \> 1.5 cm, or 1 clearly demarcated lesion with a largest diameter \> 2.0 cm. * ECOG Performance Status of 0, 1, or 2. * Age ≥ 18 years. * Life expectancy of at least 6 months in the opinion of the investigator. * Women of childbearing potential must have had a negative serum pregnancy test within 14 days of first dose of study treatment and agree to use effective contraception during the study and for one year following the last dose of study drug. * Men with a female partner of childbearing potential must have had either had a prior vasectomy or agree to use effective contraception from 2 weeks prior to administration of the first dose of study treatment until one year after the last dose of study treatment. * Must not have been on any prohibited medications. * Subjects who had received prior bendamustine were eligible if they had achieved a response (CR/PR) which lasted \> 6 months after the end of bendamustine containing treatment. Exclusion Criteria: * Lactating women * CLL, Grade 3b follicular lymphoma or evidence that the indolent lymphoma had transformed to aggressive lymphoma. Subjects suspicious for transformation should have undergone a biopsy to exclude the possibility of transformation. Subjects with a previous diagnosis of small lymphocytic leukemia (SLL) and a screening monoclonal B-lymphocyte count of ≥ 5000/µl are defined by 2008 International Workshop on Chronic Lymphocytic Leukemia (IWCLL) criteria to have CLL; such patients were NOT eligible for this study. * Rituximab-refractory disease, defined as failure to have responded to or progression within 6 months of completing rituximab or rituximab-containing combination therapy. * Previous treatment with ofatumumab. * Previous radioimmunotherapy (RIT) within 6 months of study entry. Subjects who have received RIT must have attained a PR or CR lasting at least 6 months, and must have recovered from any hematologic or other toxicity. * Previous allogeneic stem cell transplantation at any time OR autologous stem cell transplantation within 6 months of study entry. * Prior use of monoclonal antibody (other than anti CD20) within 3 months prior to randomization. Chemotherapy or other systemic lymphoma therapy within 4 weeks of study entry. * Received treatment with an investigational agent within 4 weeks of study entry, or was actively participating in another interventional clinical study. * Known Central Nervous System (CNS) involvement by lymphoma. * Current or previous other malignancy within 2 years of study entry. Exception: Subjects who have been disease-free for 2 years or more, or subjects with a history of completely resected non-melanoma skin cancer or successfully treated in situ carcinoma are eligible. * Chronic or currently active infectious disease requiring systemic antibiotics, antifungal, or antiviral treatment including, but not limited to: chronic renal infection, chronic chest infection with bronchiectasis, tuberculosis, active Hepatitis C, and known HIV disease. All Human Immunodeficient virus (HIV)-positive subjects are excluded from this study, regardless of whether they have an Acquired Immunodeficiency Syndrome (AIDS) defining disease and/or are on antiviral therapy. * Clinically significant cardiac disease including unstable angina, acute myocardial infarction within 6 months of study entry, uncontrolled congestive heart failure, and uncontrolled arrhythmia. Subjects with congestive heart disease or arrhythmia such as atrial fibrillation whose cardiac disease is well controlled on a stable medical regimen are eligible. * Other significant concurrent, uncontrolled medical conditions including, but not limited to, renal, hepatic, autoimmune, hematological, gastrointestinal, endocrine, pulmonary, neurological, cerebral or psychiatric disease which, in the Investigator's opinion, will impact study participation. * Positive serology for Hepatitis B (HB) defined as a positive test for Hepatitis B surface antigen (HBsAg). In addition, if negative for HBsAg but (Hepatitis B core antibody (HBcAb) positive (regardless of Hepatitis B surface antibody \[HBsAb\] status), a HB DNA test had to have been performed and if positive the subject will be excluded. If HBV DNA is negative, subject may be included but must have undergone HBV DNA monitoring. Prophylactic antiviral therapy may have been initiated at the discretion of the investigator. * Current active liver or biliary disease. Exception: Subjects with Gilbert's syndrome or asymptomatic gallstones, liver metastases related to indolent NHL or otherwise stable chronic liver disease per investigator assessment, are eligible. * Screening laboratory values: Neutrophils \< 1.5 x 109/L (unless due to NHL involvement of the bone marrow). Platelets \< 100 x 109/L (unless due to NHL involvement of the bone marrow). Serum creatinine ≥2.0 mg/dL; subjects with serum creatinine ≥2.0 mg/dL were are eligible if the creatinine clearance (Cockcroft Gault equation \[Cockcroft, 1976\]) is ≥40 mL/min. Total bilirubin \> 1.5 times ULN \[upper normal limit\] (unless due to liver involvement by NHL or Gilbert's disease). Transaminases \> 3 times ULN (unless due to NHL involvement). * Known or suspected inability to fully comply with study protocol. * Known or suspected hypersensitivity to ofatumumab, bendamustine or mannitol. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Chandler **Country:** United States **Facility:** Novartis Investigative Site **State:** Arizona **Zip:** 85224 **Location 2:** **City:** Burbank **Country:** United States **Facility:** Novartis Investigative Site **State:** California **Zip:** 91505 **Location 3:** **City:** Fresno **Country:** United States **Facility:** Novartis Investigative Site **State:** California **Zip:** 93720 **Location 4:** **City:** Oxnard **Country:** United States **Facility:** Novartis Investigative Site **State:** California **Zip:** 93030 **Location 5:** **City:** Aurora **Country:** United States **Facility:** Novartis Investigative Site **State:** Colorado **Zip:** 80045 **Location 6:** **City:** Orange Park **Country:** United States **Facility:** Novartis Investigative Site **State:** Florida **Zip:** 32073 **Location 7:** **City:** Burlington **Country:** United States **Facility:** Novartis Investigative Site **State:** Massachusetts **Zip:** 01805 **Location 8:** **City:** Omaha **Country:** United States **Facility:** Novartis Investigative Site **State:** Nebraska **Zip:** 68198-9200 **Location 9:** **City:** Las Vegas **Country:** United States **Facility:** Novartis Investigative Site **State:** Nevada **Zip:** 89169 **Location 10:** **City:** Cary **Country:** United States **Facility:** Novartis Investigative Site **State:** North Carolina **Zip:** 27518 **Location 11:** **City:** Charlotte **Country:** United States **Facility:** Novartis Investigative Site **State:** North Carolina **Zip:** 28204 **Location 12:** **City:** Raleigh **Country:** United States **Facility:** Novartis Investigative Site **State:** North Carolina **Zip:** 27607 **Location 13:** **City:** Kettering **Country:** United States **Facility:** Novartis Investigative Site **State:** Ohio **Zip:** 45429 **Location 14:** **City:** Eugene **Country:** United States **Facility:** Novartis Investigative Site **State:** Oregon **Zip:** 97401 **Location 15:** **City:** Charleston **Country:** United States **Facility:** Novartis Investigative Site **State:** South Carolina **Zip:** 29414 **Location 16:** **City:** Greenville **Country:** United States **Facility:** Novartis Investigative Site **State:** South Carolina **Zip:** 29601 **Location 17:** **City:** San Antonio **Country:** United States **Facility:** Novartis Investigative Site **State:** Texas **Zip:** 78229 **Location 18:** **City:** Sherman **Country:** United States **Facility:** Novartis Investigative Site **State:** Texas **Zip:** 75090 **Location 19:** **City:** Tyler **Country:** United States **Facility:** Novartis Investigative Site **State:** Texas **Zip:** 75702 **Location 20:** **City:** Waco **Country:** United States **Facility:** Novartis Investigative Site **State:** Texas **Zip:** 76712 **Location 21:** **City:** Vancouver **Country:** United States **Facility:** Novartis Investigative Site **State:** Washington **Zip:** 98684 **Location 22:** **City:** Yakima **Country:** United States **Facility:** Novartis Investigative Site **State:** Washington **Zip:** 98902 #### Overall Officials **Official 1:** **Affiliation:** Novartis Pharmaceuticals **Name:** Novartis Pharmaceuticals **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ### References Module #### References **Citation:** Lyons RM, Shtivelband M, Kingsley E, Moezi M, Richards D, Sharman J, Feng X, Cannan M, Fellague-Chebra R, Boyd TE. Efficacy and safety of ofatumumab and bendamustine followed by ofatumumab maintenance in patients with relapsed indolent non-Hodgkin lymphoma after prior rituximab. Leuk Lymphoma. 2021 Jun;62(6):1353-1360. doi: 10.1080/10428194.2020.1869957. Epub 2021 Jan 15. **PMID:** 33448893 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000009369 - Term: Neoplasms - ID: D000008232 - Term: Lymphoproliferative Disorders - ID: D000008206 - Term: Lymphatic Diseases - ID: D000007160 - Term: Immunoproliferative Disorders - ID: D000007154 - Term: Immune System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC15 - Name: Blood and Lymph Conditions - Abbrev: BC20 - Name: Immune System Diseases - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M11220 - Name: Lymphoma - Relevance: HIGH - As Found: Lymphoma - ID: M11222 - Name: Lymphoma, Non-Hodgkin - Relevance: HIGH - As Found: Lymphoma, Non-Hodgkin - ID: M18828 - Name: Lymphoma, B-Cell - Relevance: HIGH - As Found: B-Cell Non-Hodgkin Lymphoma - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: M11225 - Name: Lymphoproliferative Disorders - Relevance: LOW - As Found: Unknown - ID: M11203 - Name: Lymphatic Diseases - Relevance: LOW - As Found: Unknown - ID: M10206 - Name: Immunoproliferative Disorders - Relevance: LOW - As Found: Unknown - ID: M10200 - Name: Immune System Diseases - Relevance: LOW - As Found: Unknown - ID: T3543 - Name: Lymphosarcoma - Relevance: HIGH - As Found: Lymphoma - ID: T640 - Name: B-cell Lymphoma - Relevance: HIGH - As Found: B-Cell Non-Hodgkin Lymphoma ### Condition Browse Module - Meshes - ID: D000008223 - Term: Lymphoma - ID: D000008228 - Term: Lymphoma, Non-Hodgkin - ID: D000016393 - Term: Lymphoma, B-Cell ### Intervention Browse Module - Ancestors - ID: D000018906 - Term: Antineoplastic Agents, Alkylating - ID: D000000477 - Term: Alkylating Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000000970 - Term: Antineoplastic Agents ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: ARhu - Name: Antirheumatic Agents ### Intervention Browse Module - Browse Leaves - ID: M430 - Name: Bendamustine Hydrochloride - Relevance: HIGH - As Found: Action - ID: M373 - Name: Rituximab - Relevance: LOW - As Found: Unknown - ID: M347498 - Name: Ofatumumab - Relevance: HIGH - As Found: Lunch - ID: M20942 - Name: Antineoplastic Agents, Alkylating - Relevance: LOW - As Found: Unknown - ID: M3820 - Name: Alkylating Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000069461 - Term: Bendamustine Hydrochloride - ID: C000527517 - Term: Ofatumumab ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** Deaths are reported as a secondary outcome #### Event Groups **Group ID:** EG000 **Title:** Ofatumumab + Bendamustine **Deaths Num Affected:** 7 **Deaths Num At Risk:** 49 **Description:** Ofatumumab + Bendamustine **ID:** EG000 **Other Num Affected:** 47 **Other Num at Risk:** 49 **Serious Number Affected:** 18 **Serious Number At Risk:** 49 **Title:** Ofatumumab + Bendamustine **Frequency Threshold:** 5 #### Other Events **Term:** Anaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Leukopenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Lymphopenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Neutropenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Thrombocytopenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA (19.0) **Term:** External ear pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Ear and labyrinth disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Abdominal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Constipation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Diarrhoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Nausea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Vomiting **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Asthenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Chest discomfort **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Chills **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Fatigue **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Mucosal inflammation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Oedema peripheral **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Pyrexia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Sinusitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) **Term:** Upper respiratory tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) **Term:** Urinary tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) **Term:** Contusion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (19.0) **Term:** Infusion related reaction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (19.0) **Term:** Blood creatinine increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (19.0) **Term:** Lymphocyte count decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (19.0) **Term:** Neutrophil count decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (19.0) **Term:** Weight decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (19.0) **Term:** White blood cell count decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (19.0) **Term:** Decreased appetite **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Dehydration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Hyperuricaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Vitamin D deficiency **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Arthralgia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Back pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Bone pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Muscle spasms **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Muscular weakness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Myalgia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Pain in extremity **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Dizziness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Dysgeusia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Neuropathy peripheral **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Insomnia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Pollakiuria **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Cough **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Dyspnoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Oropharyngeal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Throat irritation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Night sweats **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Pruritus **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Rash **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Urticaria **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Flushing **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Hypertension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Hypotension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA (19.0) #### Serious Events **Term:** Febrile neutropenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 49 **Term:** Pancytopenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Term:** Gastric ulcer **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Term:** Fatigue **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 49 **Term:** Pyrexia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 49 **Term:** Anaphylactic reaction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Immune system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Term:** Diverticulitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Term:** Infected skin ulcer **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Term:** Neutropenic sepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Term:** Pneumonia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 49 **Term:** Upper respiratory tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Term:** Urinary tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Term:** Electrocardiogram QT prolonged **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Term:** Failure to thrive **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Term:** Rhabdomyolysis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Term:** Myelodysplastic syndrome **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 49 **Term:** Prostate cancer **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Term:** Squamous cell carcinoma of lung **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Term:** Squamous cell carcinoma of skin **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Term:** Syncope **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Term:** Chronic obstructive pulmonary disease **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Term:** Pneumonitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Time Frame:** Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to approximately 30 months ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 49 **Units:** Participants ### Group **ID:** BG000 **Title:** Ofatumumab and Bendamustine **Description:** 1000 mg intravenous (IV) on day 1 of each cycle (cycles 1-6) for induction phase and 1000 mg IV every 2 months for 2 years. Bendamustine 90 mg/m2 was given on day 1 (after the ofatumumab infusion) and day 2 of each cycle (cycles 1-6) ### Measure #### Measurement **Group ID:** BG000 **Spread:** 11.61 **Value:** 66.3 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 14 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 35 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 3 **Class Title:** African American/African heritage #### Measurement **Group ID:** BG000 **Value:** 45 **Class Title:** White #### Measurement **Group ID:** BG000 **Value:** 1 **Class Title:** Mixed race **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Race/Ethnicity, Customized **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement **Other Details:** The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial. **Restriction Type:** OTHER **Restrictive Agreement:** True ### Limitations and Caveats **Description:** The study was terminated early based on the evolution of the current therapeutic treatment landscape in Non Hodgkin Indolent Lymphomas and low enrollment rates for this trial. ### Point of Contact **Email:** [email protected] **Organization:** Novartis Pharmaceuticals **Phone:** 862-778-8300 **Title:** Study Director ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### Outcome Measure 4 ### Outcome Measure 5 ### Outcome Measure 6 ### Outcome Measure 7 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 13.3 - **Spread:** - **Upper Limit:** 38.9 - **Value:** 24.5 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 52.5 - **Spread:** - **Upper Limit:** 80.1 - **Value:** 67.3 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 15.2 - **Spread:** - **Upper Limit:** 64.6 - **Value:** 37.5 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 42.9 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 32.7 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 10.2 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 57.1 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** Upper limit of CI was not estimable - **Group ID:** OG000 - **Lower Limit:** 17.3 - **Spread:** - **Upper Limit:** NA - **Value:** 29.7 **Title:** #### Outcome Measure 6 #### Outcome Measure 7 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Complete response (CR) included all of the following: complete disappearance of all detectable clinical evidence of disease and disease-related symptoms if present before therapy. All target nodes had to have regressed to ≤ 1.5cm in the longest diameter. Non-measureable nodes 1.1 to 1.5cm in the longest diameter and \>1cm in the short axis at baseline had to regress to ≤ 1cm in the short axis by visual estimation; enlarged spleen or liver (with nodules) must have returned to normal size and nodules disappeared and if bone marrow was involved, infiltrate had to have cleared on repeat biopsy sample. CR was not valid without imaging data. The corresponding 2-sided 95% exact confidence interval (CI) of the response rate was estimated by the Clopper-Pearson method. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** Baseline up to 24 weeks **Title:** Complete Remission (CR) Rate of Induction Therapy After Cycle 6 (28 Days) (FAS) **Type:** PRIMARY **Unit of Measure:** percentage of participants ##### Group **Description:** 1000 mg intravenous (IV) on day 1 of each cycle (cycles 1-6) for induction phase and 1000 mg IV every 2 months for 2 years. Bendamustine 90 mg/m2 was given on day 1 (after the ofatumumab infusion) and day 2 of each cycle (cycles 1-6) **ID:** OG000 **Title:** Ofatumumab and Bendamustine #### Outcome Measure 2 **Description:** The overall response = CR (defined in Primary Outcome) + Partial Response (PR) which required all of the following: \> or = to 50% decrease from baseline in target nodules; \> or = to 50% decrease in hepatic/splenic nodules and no increase in liver or spleen size; no unequivocal progression in non-target lestions; no new sites of disease. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** Baseline up to 24 weeks **Title:** Overall Response Rate (ORR) During Induction Phase After Cycle 6 (FAS) **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** 1000 mg intravenous (IV) on day 1 of each cycle (cycles 1-6) for induction phase and 1000 mg IV every 2 months for 2 years. Bendamustine 90 mg/m2 was given on day 1 (after the ofatumumab infusion) and day 2 of each cycle (cycles 1-6) **ID:** OG000 **Title:** Ofatumumab and Bendamustine #### Outcome Measure 3 **Description:** Rate of conversion from PR in the Induction phase, to CR with maintenance ofatumumab in subjects who have a PR with induction therapy with ofatumumab and bendamustine **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** Partial response in induction phase up to 24 weeks **Title:** Conversion Rate of Partial Response in Induction Phase to Complete Response With Maintenance Ofatumumab (FAS) **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** 1000 mg intravenous (IV) on day 1 of each cycle (cycles 1-6) for induction phase and 1000 mg IV every 2 months for 2 years. Bendamustine 90 mg/m2 was given on day 1 (after the ofatumumab infusion) and day 2 of each cycle (cycles 1-6) **ID:** OG000 **Title:** Ofatumumab and Bendamustine #### Outcome Measure 4 **Description:** Progression free survival (PFS) is defined as the interval between first treatment and disease progression or death due to any cause. PFS criteria: A previously normal node (≤ 1.5 x ≤ 1.0cm), including nodes that were not previously visible, must increase to \>2.0 x ≥ 1.5cm; ≥ 50% increase from nadir in the PPD of any target node. The long axis must increase by at least 5 mm and to \>2.0cm.; ≥ 50% increase from nadir in the long axis of any target node. The long axis must increase by at least 5 mm and to \>2.0 cm.; ≥ 50% increase from nadir in the SPD of target nodes and at least one node should have a long axis \>1.5 cm. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** Baseline up to approximately 30 months **Title:** Percentage of Participants With Progression Free Survival (PFS) up to 30 Months (FAS) **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** 1000 mg intravenous (IV) on day 1 of each cycle (cycles 1-6) for induction phase and 1000 mg IV every 2 months for 2 years. Bendamustine 90 mg/m2 was given on day 1 (after the ofatumumab infusion) and day 2 of each cycle (cycles 1-6) **ID:** OG000 **Title:** Ofatumumab and Bendamustine #### Outcome Measure 5 **Description:** Progression Free Survival (PFS) is defined as the interval between first treatment and disease progression or death due to any cause. PFS events: progression documented between scheduled visits, death before first PD assessment (or death at baseline or prior to any adequate assessments), death between adequate assessment visits. For the PFS analysis, the survival function was estimated using Kaplan-Meier estimates. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEDIAN **Reporting Status:** POSTED **Time Frame:** Baseline up to approximately 30 months **Title:** Kaplan-Meier Estimates of Progression Free Survival up to 30 Months (FAS) **Type:** SECONDARY **Unit of Measure:** months ##### Group **Description:** 1000 mg intravenous (IV) on day 1 of each cycle (cycles 1-6) for induction phase and 1000 mg IV every 2 months for 2 years. Bendamustine 90 mg/m2 was given on day 1 (after the ofatumumab infusion) and day 2 of each cycle (cycles 1-6) **ID:** OG000 **Title:** Ofatumumab and Bendamustine #### Outcome Measure 6 **Description:** Due to recruitment issues, this data analysis was not done per changes in planned analysis. This data was only presented as patient listings. The statistical analysis plan was modified to indicate that Pharmacokinetic/Pharmacodynamic exploratory analyses were not done. **Reporting Status:** POSTED **Time Frame:** up to 30 months **Title:** Pharmacokinetic Profile Which Includes Measuring Blood Levels of Ofatumumab and Bendamustine in Combination and Ofatumumab Alone During Maintenance Treatment and Measuring Blood Levels of Circulating B Cell **Type:** SECONDARY ##### Group **Description:** 1000 mg intravenous (IV) on day 1 of each cycle (cycles 1-6) for induction phase and 1000 mg IV every 2 months for 2 years. Bendamustine 90 mg/m2 was given on day 1 (after the ofatumumab infusion) and day 2 of each cycle (cycles 1-6) **ID:** OG000 **Title:** Ofatumumab and Bendamustine #### Outcome Measure 7 **Description:** Deaths were collected and were considered to be an on treatment death up to 60 days post treatment. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** Baseline up to approximately 30 months **Title:** All Deaths by Preferred Term (Safety Set) up to Approximately 30 Months **Type:** SECONDARY **Unit of Measure:** participants ##### Group **Description:** 1000 mg intravenous (IV) on day 1 of each cycle (cycles 1-6) for induction phase and 1000 mg IV every 2 months for 2 years. Bendamustine 90 mg/m2 was given on day 1 (after the ofatumumab infusion) and day 2 of each cycle (cycles 1-6) **ID:** OG000 **Title:** Ofatumumab and Bendamustine ### Participant Flow Module #### Group **Description:** 1000 mg intravenous (IV) on day 1 of each cycle (cycles 1-6) for induction phase and 1000 mg IV every 2 months for 2 years. Bendamustine 90 mg/m2 was given on day 1 (after the ofatumumab infusion) and day 2 of each cycle (cycles 1-6) **ID:** FG000 **Title:** Ofatumumab and Bendamustine #### Period **Title:** Overall Study ##### Withdraw **Type:** Disease progression ###### Reason **Group ID:** FG000 **Number of Subjects:** 12 ##### Withdraw **Type:** Adverse Event ###### Reason **Group ID:** FG000 **Number of Subjects:** 11 ##### Withdraw **Type:** Study closed/terminated ###### Reason **Group ID:** FG000 **Number of Subjects:** 3 ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ##### Withdraw **Type:** Physician Decision ###### Reason **Group ID:** FG000 **Number of Subjects:** 3 ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 5 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 49 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 14 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 35 **Pre-Assignment Details:** Subjects completed represents subjects who completed treatment. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT02949479 **Acronym:** DISSO **Brief Title:** Dissociation Investigation Study in Sex Offenders **Official Title:** Dissociative Experience During the Offence in Sex Offenders : Clinical and Prognosis Correlates #### Organization Study ID Info **ID:** 2016-A01019-42 #### Organization **Class:** OTHER **Full Name:** Hôpital le Vinatier ### Status Module #### Completion Date **Date:** 2021-12-10 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2022-01-27 **Type:** ACTUAL **Last Update Submit Date:** 2022-01-26 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2021-09-21 **Type:** ACTUAL #### Start Date **Date:** 2017-03-29 **Type:** ACTUAL **Status Verified Date:** 2021-12 #### Study First Post Date **Date:** 2016-10-31 **Type:** ESTIMATED **Study First Submit Date:** 2016-10-05 **Study First Submit QC Date:** 2016-10-27 **Why Stopped:** STUDY INVESTIGATORS HAVE LEFT THE INSTITUTION, THE DEPARTMENT NO LONGER HAS TIME TO INCLUDE ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Hôpital le Vinatier #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Background: Adverse childhood experience have been described in sexual offenders but the link with the offence need to be further investigated. Investigators postulate that one of the clinical moderating factors could be dissociative experience, a consequence of these early adverse experiences reactivated during the offence. The purpose of the study is to estimate the prevalence of clinical dissociation during the offence in a male adult population referred to our center for a sexual offence and to explore its correlations with epidemiological and clinical data (personal, legal history, psychiatric comorbidities), clinical trauma and dissociation, prognosis estimates. **Detailed Description:** The study will be proposed to the subjects after their usual clinical evaluation in the center for sexual offence and to extend this evaluation by a specific focus on childhood abuse and neglect trauma and dissociative history. After receiving complete information, the participants will sign the consent form and be referred to a unique on-site visit of approximately 2 hours long. During this visit, the participants will benefit from a psychiatric examination in search for clinical features of dissociation during the offence, lifetime dissociative experience, lifetime post-traumatic stress disorder; they will have to complete bioevaluation forms for dissociation (Dissociative Experience Scale) and childhood abuse and neglect (Childhood Experience of Care and Abuse). After this completion, they will benefit from a second part psychiatric examination, so as to complete the assessment and answer their questions if any. ### Conditions Module **Conditions:** - Sex Offenses **Keywords:** - sex offenses ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** HEALTH_SERVICES_RESEARCH #### Enrollment Info **Count:** 30 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** The study will be proposed to the subjects after their usual clinical evaluation in the center for sexual offence, and to extend this evaluation by a specific focus on childhood abuse and neglect, trauma and dissociative history **Intervention Names:** - Behavioral: Dissociation Investigation in Sex Offenders **Label:** Dissociation Investigation in Sex Offenders **Type:** OTHER ### Interventions #### Intervention 1 **Arm Group Labels:** - Dissociation Investigation in Sex Offenders **Description:** prevalence of a clinical dissociative state during the offence; secondary : correlations of dissociation with childhood abuse or neglect, with significant lifetime dissociative experience, post traumatic stress disorder or dissociative disorder (DSM-5), violence and sexual estimated risk using actuarial and professional structured judgement tools **Name:** Dissociation Investigation in Sex Offenders **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** the presence of a clinical dissociative state will be assessed retrospectively with the use of clinical examination. **Measure:** point prevalence of clinical dissociation during the offence, assessed with the use of clinical examination (narrative and DSM-5 criteria) **Time Frame:** 3 years #### Secondary Outcomes **Description:** lifetime dissociation measured by the Dissociative Experience Scale **Measure:** lifetime dissociation **Time Frame:** 3 years **Description:** Childhood abuse ant Neglect assessed by the Childhood Experience of Care and Abuse (CECA) **Measure:** childhood abuse or neglect **Time Frame:** 3 years **Description:** psychiatric comorbidities **Measure:** psychiatric comorbidities **Time Frame:** 3 years **Description:** risk assessment tools scoring **Measure:** prognosis issues **Time Frame:** 3 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * male subject referred to the center for having committed a sexual offense; social insurance affiliation Exclusion Criteria: * schizophrenia, mental retardation (IQ\<70) **Minimum Age:** 18 Years **Sex:** MALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Bron **Country:** France **Facility:** Centre Hospitalier Le Vinatier **State:** Rhône Alpes **Zip:** 69678 #### Overall Officials **Official 1:** **Affiliation:** Centre Hospitalier le Vinatier **Name:** Mouchet-Mages Sabine, PH **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02589379 **Acronym:** PMPPOPF **Brief Title:** Ultrasound Elastography for Prediction of Postoperative Pancreatic Fistula **Official Title:** The Value of Endoscopic Ultrasound Elastography for Prediction of Pancreatic Fistula in Patients Undergoing Pancreatic Resection for Benign or Malignant Disease #### Organization Study ID Info **ID:** POPF-0134 #### Organization **Class:** OTHER **Full Name:** University of Zurich ### Status Module #### Completion Date **Date:** 2019-05 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2016-01-20 **Type:** ESTIMATED **Last Update Submit Date:** 2016-01-16 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2019-05 **Type:** ESTIMATED #### Start Date **Date:** 2015-07 **Status Verified Date:** 2016-01 #### Study First Post Date **Date:** 2015-10-28 **Type:** ESTIMATED **Study First Submit Date:** 2015-10-09 **Study First Submit QC Date:** 2015-10-26 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Zurich #### Responsible Party **Investigator Affiliation:** University of Zurich **Investigator Full Name:** Mickael Lesurtel **Investigator Title:** Professor, MD, PhD **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this study is to assess the value of preoperative objective modalities such as endoscopic ultrasound elastography and magnet resonance imaging in predicting development of postoperative pancreatic fistula in patients undergoing pancreatic surgery for benign or malignant disease. **Detailed Description:** One of the most generally accepted causes of postoperative pancreatic fistula (POPF) is soft texture of the pancreas. The stiffness of organs depends on histologic features such as content of fat, fibrotic tissue and micro vascular structures. Yet, pancreatic stiffness was evaluated only subjectively. Endoscopic ultrasonography (EUS) and magnetic resonance imaging (MRI) may allow objective quantification of pancreatic stiffness prior to surgery. Endoscopic ultrasound elastography (EUE) is based on real-time Doppler technology. Image colors are derived from vibration patterns which depend on the stiffness of a specific region of the investigated organ. The retrieved images could be converted into numeric matrix using a specially designed program. Magnetic resonance imaging (MRI) is a non-invasive method for quantitatively assessing the mechanical properties of tissues based on fat tissue content. In this trial the investigators will assess at first the value of EUE and MRI in predicting development of postoperative pancreatic fistula and in a next step the value EUE and MRI as an objective method in prediction of pancreatic stiffness. ### Conditions Module **Conditions:** - Pancreatic Fistula **Keywords:** - postoperative pancreatic fistula - soft and hard pancreas ### Design Module #### Bio Spec **Description:** Non-tumorous pancreatic tissue **Retention:** SAMPLES_WITHOUT_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 90 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** All consecutive patients undergoing pancreatic resection for benign or malignant disease and meet inclusion criteria. **Intervention Names:** - Procedure: Pancreatic Resection **Label:** Pancreatic Resection ### Interventions #### Intervention 1 **Arm Group Labels:** - Pancreatic Resection **Description:** Endoscopic ultrasound elastography, intraoperative ultrasound elastography and magnet resonance imaging to asses pancreatic stiffness prior to pancreatic resection **Name:** Pancreatic Resection **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Measure:** POPF rate (in %) as defined by the International Study Group on Pancreatic Fistula (ISGPF) in the soft versus hard pancreas group based on the median EUE stiffness measurements generated by values derived from hue histograms. **Time Frame:** within the 30 days after surgery #### Secondary Outcomes **Measure:** Pearson correlation coefficient between preoperative EUE measurements of pancreatic stiffness generated by values derived from hue histograms with the fat content (in %) of the resected specimen on histology. **Time Frame:** within the 30 days after surgery **Measure:** Pearson correlation coefficient between preoperative EUE measurements of pancreatic stiffness generated by values derived from hue histograms with intra-operative ultrasound elastography measurements generated by values derived from hue histograms. **Time Frame:** within the 30 days after surgery **Measure:** Postoperative pancreatic fistula rate (in %) as defined by the ISGPF in the soft versus hard pancreas group based on the median MRI fat measurement (in %) on histology. **Time Frame:** within the 30 days after surgery **Measure:** Pearson correlation coefficient between preoperative MRI measurements of pancreatic fat (in %) with the fat content (in %) of the resected specimen on histology. **Time Frame:** within the 30 days after surgery **Measure:** Postoperative pancreatic fistula rate (in %) as defined by the ISGPF in the soft versus hard pancreas group based on the surgeons assessment of stiffness on a visual analogue scale from soft (0) to hard (10) intraoperatively. **Time Frame:** within the 30 days after surgery **Measure:** Pearson correlation coefficient between the two surgeons assessing pancreatic stiffness intraoperatively. **Time Frame:** within the 30 days after surgery **Description:** within the 30 days after surgery **Measure:** Intraclass correlation coefficient of the all measurements derived from EUE, IUE, MRI, surgeons assessment and histology. **Time Frame:** within the 30 days after surgery ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Adult (more than 18 years) with resectable pancreatic lesion(s) Exclusion Criteria: * Total pancreatectomy * Unresectable pancreatic lesion(s) * Pregnancy **Minimum Age:** 18 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Patients undergoing pancreatic resection for benign or malignant disease ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Henrik Petrowsky, Prof, MD **Phone:** +41 44 255 30 41 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Dilmurodjon Eshmuminov, MD **Phone:** +41 44 255 11 11 **Role:** CONTACT #### Locations **Location 1:** **City:** Zurich **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Henrik Petrowsky, Prof, MD - **Phone:** +41 44 255 30 41 - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Dilmurodjon Eshmuminov, MD - **Phone:** +41 44 255 11 11 - **Role:** CONTACT **Country:** Switzerland **Facility:** University Hospital Zurich **Status:** RECRUITING **Zip:** 8091 #### Overall Officials **Official 1:** **Affiliation:** University of Zurich **Name:** Henrik Petrowsky, Prof, MD **Role:** STUDY_CHAIR ### References Module #### References **Citation:** Bassi C, Dervenis C, Butturini G, Fingerhut A, Yeo C, Izbicki J, Neoptolemos J, Sarr M, Traverso W, Buchler M; International Study Group on Pancreatic Fistula Definition. Postoperative pancreatic fistula: an international study group (ISGPF) definition. Surgery. 2005 Jul;138(1):8-13. doi: 10.1016/j.surg.2005.05.001. **PMID:** 16003309 **Citation:** Lin JW, Cameron JL, Yeo CJ, Riall TS, Lillemoe KD. Risk factors and outcomes in postpancreaticoduodenectomy pancreaticocutaneous fistula. J Gastrointest Surg. 2004 Dec;8(8):951-9. doi: 10.1016/j.gassur.2004.09.044. **PMID:** 15585382 **Citation:** Mathur A, Pitt HA, Marine M, Saxena R, Schmidt CM, Howard TJ, Nakeeb A, Zyromski NJ, Lillemoe KD. Fatty pancreas: a factor in postoperative pancreatic fistula. Ann Surg. 2007 Dec;246(6):1058-64. doi: 10.1097/SLA.0b013e31814a6906. **PMID:** 18043111 **Citation:** Nathan H, Cameron JL, Goodwin CR, Seth AK, Edil BH, Wolfgang CL, Pawlik TM, Schulick RD, Choti MA. Risk factors for pancreatic leak after distal pancreatectomy. Ann Surg. 2009 Aug;250(2):277-81. doi: 10.1097/SLA.0b013e3181ae34be. **PMID:** 19638926 **Citation:** Saftoiu A, Vilmann P, Gorunescu F, Janssen J, Hocke M, Larsen M, Iglesias-Garcia J, Arcidiacono P, Will U, Giovannini M, Dietrich CF, Havre R, Gheorghe C, McKay C, Gheonea DI, Ciurea T; European EUS Elastography Multicentric Study Group. Efficacy of an artificial neural network-based approach to endoscopic ultrasound elastography in diagnosis of focal pancreatic masses. Clin Gastroenterol Hepatol. 2012 Jan;10(1):84-90.e1. doi: 10.1016/j.cgh.2011.09.014. Epub 2011 Oct 1. **PMID:** 21963957 **Citation:** Sato N, Yamaguchi K, Chijiiwa K, Tanaka M. Risk analysis of pancreatic fistula after pancreatic head resection. Arch Surg. 1998 Oct;133(10):1094-8. doi: 10.1001/archsurg.133.10.1094. **PMID:** 9790207 **Citation:** Schafer M, Mullhaupt B, Clavien PA. Evidence-based pancreatic head resection for pancreatic cancer and chronic pancreatitis. Ann Surg. 2002 Aug;236(2):137-48. doi: 10.1097/00000658-200208000-00001. **PMID:** 12170018 **Citation:** Watanabe H, Kanematsu M, Tanaka K, Osada S, Tomita H, Hara A, Goshima S, Kondo H, Kawada H, Noda Y, Tanahashi Y, Kawai N, Yoshida K, Moriyama N. Fibrosis and postoperative fistula of the pancreas: correlation with MR imaging findings--preliminary results. Radiology. 2014 Mar;270(3):791-9. doi: 10.1148/radiol.13131194. Epub 2013 Nov 8. **PMID:** 24475834 **Citation:** Yeh TS, Jan YY, Jeng LB, Hwang TL, Wang CS, Chen SC, Chao TC, Chen MF. Pancreaticojejunal anastomotic leak after pancreaticoduodenectomy--multivariate analysis of perioperative risk factors. J Surg Res. 1997 Feb 1;67(2):119-25. doi: 10.1006/jsre.1996.4974. **PMID:** 9073557 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020763 - Term: Pathological Conditions, Anatomical - ID: D000016154 - Term: Digestive System Fistula - ID: D000004066 - Term: Digestive System Diseases - ID: D000010182 - Term: Pancreatic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC06 - Name: Digestive System Diseases ### Condition Browse Module - Browse Leaves - ID: M8532 - Name: Fistula - Relevance: HIGH - As Found: Fistula - ID: M13105 - Name: Pancreatic Fistula - Relevance: HIGH - As Found: Pancreatic Fistula - ID: M22519 - Name: Pathological Conditions, Anatomical - Relevance: LOW - As Found: Unknown - ID: M18616 - Name: Digestive System Fistula - Relevance: LOW - As Found: Unknown - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M13102 - Name: Pancreatic Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000010185 - Term: Pancreatic Fistula - ID: D000005402 - Term: Fistula ### Intervention Browse Module - Browse Branches - Abbrev: Gast - Name: Gastrointestinal Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M22554 - Name: Pancrelipase - Relevance: LOW - As Found: Unknown - ID: M13114 - Name: Pancreatin - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04614779 **Brief Title:** Long-term Clinical Study of CN128 in Thalassemia Patients **Official Title:** Phase IIa Clinical Study to Assess the Safety and Efficacy of CN128 Tablets in the Treatment of Iron Overload in Transfusion Dependent Thalassemia Patients Aged 16 and Above #### Organization Study ID Info **ID:** A160605 #### Organization **Class:** OTHER **Full Name:** Hangzhou Zede Pharma-Tech Co., Ltd. ### Status Module #### Completion Date **Date:** 2022-08-18 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-11-22 **Type:** ACTUAL **Last Update Submit Date:** 2023-11-20 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2022-08-18 **Type:** ACTUAL #### Start Date **Date:** 2020-09-30 **Type:** ACTUAL **Status Verified Date:** 2023-11 #### Study First Post Date **Date:** 2020-11-04 **Type:** ACTUAL **Study First Submit Date:** 2020-09-23 **Study First Submit QC Date:** 2020-10-30 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Hangzhou Zede Pharma-Tech Co., Ltd. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** 1. Primary objectives: • To evaluate the safety and efficacy of long-term orally administration of CN128 in thalassaemia patients with blood transfusion dependent and aged 16 and above. 2. Design: * The study is designed as a single arm and opened phase IIa clinical trial, so as to investigate the safety and efficacy of CN128. * A total of 50 eligible subjects are planned to be enrolled, and orally administration of CN128 for 24 weeks or 48 weeks according to the administration plan. The treatment period is from day 0 to 24 weeks, and the extended treatment period was from 25 weeks to 96 weeks. * Subjects' medication status, uncomfortable symptoms, concomitant medication or non-drug therapy were recorded daily. 3. Subject inclusion criteria: * Thalassemia patients. * The number of blood transfusion per month ≥1. Or hemoglobin can not be maintained at 90g/L above, if blood transfusions is less than once per month. * Serum ferritin ≥ 500 µg/L * Patients aged 16 and above * Volunteer for the trial and sign the informed consent. 4. Subject exclusion criteria: * Active hepatitis B (HBsAg positive, HBsAb negative) or hepatitis C (HCV antibody positive, detectable HCV RNA, and alanine transaminase (ALT) beyond normal range) * Active gastrointestinal disease history (including: gastric ulcer, duodenal ulcer, stomach or esophageal varices, ulcerative colitis, Crohn's disease, gastrointestinal cancer, familial genetic multiple intestinal polyps), and History of gastrointestinal perforation, gastrointestinal surgery that influence drug absorption, and other potential intestinal complications considered by researchers; * ALT or Aspartate transaminase (AST) \> 2.5 × Upper limit of normal (ULN), or serum creatinine \> 1.5 × ULN; * Neutropenia patient (neutrophil count \< 1.5 × 109 / L); * Active infection uncontrolled; * The patients who are currently taking CYP3A strong inducer or strong inhibitor drugs, or the drug that may extend the QT interval, or the drug that may decrease neutrophil count, but can not temporarily interrupt the use of such drugs; * Congenital long QT syndrome or known family history of long QT syndrome; QTc \> 480 ms; clinically significant ventricular or atrial fast arrhythmia; * The patients who can not accept MRI as detection means, such as claustrophobic for MRI, pacemaker, and those using ferromagnetic metal implants. * Birth planner (including male subjects) within or within 3 months after the end of the trial; * Participated in other clinical trials in the three months before taking the test preparation, except for non-interventional studies; * Pregnant or lactating women; * Unsuitable to participate in the trial considered by the researchers. 5. Usage: * All subjects will be given the lower (10 mg/kg bw, bid) or higher dose (15 mg/kg bw, bid) for 24 or 48 weeks, according to the administration plan. * All subjects will be given the lower (15 mg/kg bw, bid) or higher dose (20 mg/kg bw, bid) for 49 or 96 weeks, according to the administration plan. 6. Safety assessments: Safety evaluations include adverse events, adverse reactions, severe adverse events, and severe adverse reactions; growth (weight, height); total and free testosterone in men, follicle-generating hormone and luteinizing hormone in women; vital signs and electrocardiogram; hearing, laboratory tests (blood routine analytes, blood biochemistry, coagulation function, thyroid and para-thyroid function, urine routine analytes.), urine pregnancy test (women of childbearing age),Levels of drug exposure during the study. 7. Efficacy assessments: Efficacy evaluations include serum ferritin, liver iron content (MRI R2) and cardiac iron content (MRI T2\*). 8. Statistics: * Subject characteristic distribution Demographic characteristics, general conditions, and baseline conditions (pre-treatment) of enrolled subjects were analyzed.The measurement data are described by means, standard deviation, minimum value and maximum value, while the qualitative data list frequency and percentage. * Safety analysis Descriptive statistical analysis was used for safety endpoints. * Effectiveness analysis Mean, standard deviation, median, minimum and maximum values were described and 95% confidence intervals were calculated. Paired T-test was used to compare each time point with the baseline if necessary. The 95% confidence interval was calculated by using Clopper-Pearson method for the proportion of patients. **Detailed Description:** Clinical Trial - IIa - Study Description - Detailed Description 1. Primary objectives: * To evaluate the adverse events, adverse reactions, severe adverse events and severe adverse reactions during the study period, so as to investigate the safety; * To evaluate the changes of serum ferritin over time after orally administration of CN128 in thalassaemia patients with blood transfusion dependent and aged 16 and above, so as to investigate the efficacy; * To evaluate the effect on iron excretion in liver after orally administration of CN128 for 12, 24 weeks in thalassaemia patients with blood transfusion dependent and aged 16 and above, so as to investigate the efficacy; * To evaluate the effect on iron excretion in heart after orally administration of CN128 for 12, 24 weeks in thalassaemia patients with blood transfusion dependent and aged 16 and above, so as to investigate the efficacy; * To evaluate the proportion of patients with decreased or unchanged liver iron content after orally administration of CN128 for 12, 24 weeks in thalassaemia patients with blood transfusion dependent and aged 16 and above, so as to investigate the efficacy; * To evaluate the safety and efficacy of long-term orally administration of CN128 in thalassaemia patients with blood transfusion dependent and aged 16 and above. 2. Design: * The study is designed as a single arm and opened phase IIa clinical trial, so as to investigate the safety and efficacy of CN128. * A total of 50 eligible subjects are planned to be enrolled, and orally administration of CN128 for 24 weeks or 48 weeks according to the administration plan. The treatment period is from day 0 to 24 weeks, and the extended treatment period was from 25 weeks to 96 weeks. * Administration plan: (1)0 day\~48 weeks： The trial will start with the lower dose of CN128 (10 mg/kg body weight \[bw\], bid) for two weeks, then the subjects will return to the study center. If no unacceptable toxicity associated with CN128 is found, the subjects will be given the higher dose (15 mg/kg body weight \[bw\], bid). If unacceptable toxicity associated with CN128 is found, the subjects will be suspended or stopped administration. If the adverse event turns to normal or abnormal but no clinical significance after suspension administration, the subjects will be given the lower dose of CN128 (10 mg/kg body weight \[bw\], bid). After taking CN128 at 15 mg/kg, if unacceptable toxicity associated with CN128 is found, the dosage will be reduce to 10 mg/kg. If no symptoms appear, the dose can be increased to 15 mg/kg. If the unacceptable toxicity related to CN128 still occurs, the subjects will be suspended or stopped administration. If the adverse event turns to normal or abnormal but no clinical significance after suspension administration, the subjects will be given the lower dose of CN128 (10 mg/kg body weight \[bw\], bid). The dose will be assessed once every two or four weeks. Subjects' medication status, uncomfortable symptoms, concomitant medication or non-drug therapy were recorded daily. (2)49 weeks\~96 weeks： Subjects who have completed 48 weeks of treatment may continue dosing at the original dose until the end of the study or withdraw early if they do not experience intolerable toxicity associated with CN128 tablets and have fair efficacy (≥20% elevation on MRI T2\*) and if, in the judgment of the investigator, the benefits outweigh the risks. Subjects who have completed 48 weeks of treatment, who have not experienced intolerable toxicity associated with CN128 tablets, but who have been assessed by the investigator as having poor efficacy (\<20% elevation of MRI T2\*) and in the judgment of the investigator the benefit outweighs the risk, may, with the subject's consent, have the dosage increased at the additional visit or the established most recent visit to 15 mg/kg in the morning and 20 mg/kg in the evening, with an assessment made at 2 weeks of dosing： If no relevant intolerable toxicity occurs, the dose may continue to be increased to 20 mg/kg bid and assessed after 2 weeks of dosing: 1. If no relevant intolerable toxicity occurs, the 20 mg/kg bid dose may be administered until the end of the study or early withdrawal (daily dose 40 mg/kg/d); 2. If relevant intolerable toxicity occurs, the dose may be reduced to 15 mg/kg in the morning and 20 mg/kg bid in the evening until the end of the study or early withdrawal (daily dose 35 mg/kg/d). If relevant intolerable toxicity occurs, the dose may be reduced to 15 mg/kg bid until the end of the study or early withdrawal (daily dose 30 mg/kg/d). 3. Subject inclusion criteria: * Thalassemia patients. * The number of blood transfusion per month ≥1. Or hemoglobin can not be maintained at 90g/L above, if blood transfusions is less than once per month. * Serum ferritin ≥ 500 µg/L * Patients aged 16 and above * Volunteer for the trial and sign the informed consent. 4. Subject exclusion criteria: * Active hepatitis B (HBsAg positive, HBsAb negative) or hepatitis C (HCV antibody positive, detectable HCV RNA, and ALT beyond normal range) * Active gastrointestinal disease history (including: gastric ulcer, duodenal ulcer, stomach or esophageal varices, ulcerative colitis, Crohn's disease, gastrointestinal cancer, familial genetic multiple intestinal polyps), and History of gastrointestinal perforation, gastrointestinal surgery that influence drug absorption, and other potential intestinal complications considered by researchers; * ALT or AST \> 2.5 × ULN, or serum creatinine \> 1.5 × ULN; * Neutropenia patient (neutrophil count \< 1.5 × 109 / L); * Active infection uncontrolled; * The patients who are currently taking CYP3A strong inducer or strong inhibitor drugs, or the drug that may extend the QT interval, or the drug that may decrease neutrophil count, but can not temporarily interrupt the use of such drugs; * The patients who are allergic or contraindicated to the main ingredients or excipients of CN128 tablets; * Congenital long QT syndrome or known family history of long QT syndrome; QTc \> 480 ms; clinically significant ventricular or atrial fast arrhythmia; * The patients who can not accept MRI as detection means, such as claustrophobic for MRI, pacemaker, and those using ferromagnetic metal implants; * Birth planner (including male subjects) within or within 3 months after the end of the trial; * Participated in other clinical trials in the three months before taking the test preparation, except for non-interventional studies; * Pregnant or lactating women; * Unsuitable to participate in the trial considered by the researchers. 5. Usage: All subjects will be given the lower (10 mg/kg bw, bid) or higher dose (15 mg/kg bw, bid) for 24 or 48 weeks, according to the administration plan. All subjects will be given the lower (15 mg/kg bw, bid) or higher dose (20 mg/kg bw, bid) for 49 or 96 weeks, according to the administration plan. 6. Safety assessments: Safety evaluations include adverse events, adverse reactions, severe adverse events, and severe adverse reactions; growth (weight, height); total and free testosterone in men, follicle-generating hormone and luteinizing hormone in women; vital signs and electrocardiogram; hearing, laboratory tests (blood routine analytes, blood biochemistry, coagulation function, thyroid and para-thyroid function, urine routine analytes.), urine pregnancy test (women of childbearing age),Levels of drug exposure during the study. 7. Efficacy assessments: Efficacy evaluations include serum ferritin, liver iron content (MRI R2) and cardiac iron content (MRI T2\*). 8. Statistics: * Subject characteristic distribution Demographic characteristics, general conditions, and baseline conditions (pre-treatment) of enrolled subjects were analyzed.The measurement data are described by means, standard deviation, minimum value and maximum value, while the qualitative data list frequency and percentage. * Safety analysis Descriptive statistical analysis was used for safety endpoints. Summarize the incidence of adverse events, adverse reactions, adverse events leading to withdrawal from the trial, adverse events leading to death, severe adverse events, and severe adverse reactions. The incidence is calculated by subsystem, symptom/sign. Severity of adverse events and adverse reactions: if multiple adverse events occur in the same subject, the most serious one is included in the analysis; if different adverse events occurred in the same subject, the most severe adverse events were counted in the analysis. Drug exposure during the study: describe medication compliance during the study, actual dose, administration adjustments during the study, whether the study was discontinued, and reasons for the suspension. * Effectiveness analysis Mean, standard deviation, median, minimum and maximum values were described and 95% confidence intervals were calculated. Paired T-test was used to compare each time point with the baseline if necessary. The 95% confidence interval was calculated by using Clopper-Pearson method for the proportion of patients. ### Conditions Module **Conditions:** - Thalassemia - Iron Overload ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 18 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** All subjects will be given the lower (10 mg/kg bw, bid) or higher dose (15 mg/kg bw, bid) for 24 or 48 weeks, according to the administration plan. All subjects will be given the lower (15 mg/kg bw, bid) or higher dose (20 mg/kg bw, bid) for 49 or 96 weeks, according to the administration plan. The dosage form is tablets. **Intervention Names:** - Drug: CN128 Tablets **Label:** CN128 Group **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - CN128 Group **Description:** Iron chelator, oral tablets **Name:** CN128 Tablets **Other Names:** - CN128 **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** To determine the incidence, type and severity of adverse events, adverse reactions, severe adverse events and severe adverse reactions in patients up to 96 weeks. **Measure:** Number of participants with adverse events, adverse reactions, severe adverse events and severe adverse reactions as a measure of safety and tolerability during the study period **Time Frame:** up to 96 weeks **Description:** The patient's weight will be determined, and it's one kind of Physical examination. **Measure:** Absolute Change in Weight (Unit: kg) From Baseline Over Time **Time Frame:** Baseline, 24, 48, 72 and 96 weeks. **Description:** The patient's height will be determined, and it's one kind of Physical examination. **Measure:** Absolute Change in Height (Unit: m) From Baseline Over Time **Time Frame:** Baseline,24, 48, 72 and 96 weeks. **Description:** Hormon will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Hormon (total and free testosterone in men, follicle-generating hormone and luteinizing hormon in women) From Baseline Over Time **Time Frame:** Baseline, 24, 48, 72 and 96 weeks. **Description:** The patient's temperature will be determined, and it's one kind of vital signs checks. **Measure:** Absolute Change in Temperature (Unit: ℃）From Baseline Over Time **Time Frame:** Baseline, 2, 4, 8, 12, 16, 20, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** Both patient's systolic and diastolic blood pressure will be measured, and it's one kind of vital signs checks. **Measure:** Absolute Change in Blood pressure (Unit: mmHg ) From Baseline Over Time **Time Frame:** Baseline, 2, 4, 8, 12, 16, 20, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's heart rate will be determined, and it's one kind of vital signs checks. **Measure:** Absolute Change in Heart rate (Unit: bpm) From Baseline Over Time **Time Frame:** Baseline, 2, 4, 8, 12, 16, 20, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's respiration will be determined, and it's one kind of vital signs checks. **Measure:** Absolute Change in Respiration (Unit: bpm) From Baseline Over Time **Time Frame:** Baseline, 2, 4, 8, 12, 16, 20, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's electrocardiogram will be measured, and it's one kind of laboratory test. **Measure:** Absolute Change in Electrocardiogram (P-R (Unit: ms), QRS (Unit: ms), QTc (Unit: ms), etc) From Baseline Over Time **Time Frame:** Baseline, 2, 4, 8, 12, 16, 20, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's auditory function will be determined by otorhinolaryngology. **Measure:** Change in Auditory Function From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's white blood count will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in White Blood Count (Unit: 10E9/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's neutrophil count will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Neutrophil Count (Unit: 10E9/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's lymphocyte count will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Lymphocyte Count (Unit: 10E9/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's monocyte count will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Monocyte Count (Unit: 10E9/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's eosinophilic count will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Eosinophilic Count (Unit: 10E9/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's basophilic count will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Basophilic Count (Unit: 10E9/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's red blood count will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Red Blood Count (Unit: 10E9/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's hemoglobin will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Hemoglobin (Unit: g/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's hematocrit will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Hematocrit (Unit:%) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's blood platelet count will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Blood Platelet Count (Unit: 10E9/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's thrombocytocrit will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Thrombocytocrit (Unit: %) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's alanine aminotransferase will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Alanine Aminotransferase (Unit: U/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's total bilirubin will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Total Bilirubin (Unit:µmol/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's total protein will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Total Protein (Unit:g/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's albumin will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Albumin (Unit: g/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's globulin will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Globulin (Unit: g/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's alkaline phosphatase will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Alkaline Phosphatase (Unit: U/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's lactate dehydrogenase will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Lactate Dehydrogenase (Unit: IU/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's urea will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Urea (Unit: mmol/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's creatinine will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Creatinine (Unit:µmol/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's glucose will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Glucose (Unit: mmol/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's potassium will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Potassium (Unit: mmol/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's sodium will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Sodium (Unit: mmol/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's chlorine will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Chlorine (Unit: mmol/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's calcium will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Calcium (Unit: mmol/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's aspartate aminotransferase will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Aspartate Aminotransferase (Unit: U/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's fibrinogen will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Fibrinogen (Unit: g/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's prothrombin time will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Prothrombin time (Unit: s) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's APTT will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Activated Partial Thromboplastin Time (APTT, Unit: s) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's thyroid and para-gland function will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Thyroid and para-gland function (Serum total thyroxine, parathyroid hormone, total triiodothyronine and thyrotropin) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's urine glucose will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Urine Glucose (Unit: mmol/L) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The patient's urine protein will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Urine Protein (Unit: g/L)) From Baseline Over Time **Time Frame:** Baseline, 2, 12, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** The dosages will be investigated up to 96 weeks. **Measure:** Absolute Change in Dosage（mg/kg）during the study period **Time Frame:** Up to 96 weeks **Description:** The duration of administration will be investigated up to 96 weeks. **Measure:** Duration of Administration during the study period **Time Frame:** Up to 96 weeks **Description:** The number of participants will be investigated up to 96 weeks. **Measure:** Number of Participants during the study period **Time Frame:** Up to 96 weeks **Description:** Serum ferritin will be determined, and it's one kind of laboratory test. **Measure:** Absolute Change in Serum ferritin (Unit: µg/L) From Baseline Over Time **Time Frame:** Baseline, 2, 4, 8, 12, 16, 20, 24, 32, 40, 48, 60, 72, 84 and 96 weeks. **Description:** Liver iron content will be determined by liver magnetic resonance. **Measure:** Absolute Change in Liver iron content (MRI R2) From Baseline Over Time **Time Frame:** Baseline, 12, 24, 48, 72 and 96 weeks. **Description:** Heart iron content will be determined by cardio magnetic resonance. **Measure:** Absolute Change in Heart iron content (MRI T2*) From Baseline Over Time **Time Frame:** Baseline, 12, 24, 48, 72 and 96 weeks. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Thalassemia patients. * The number of blood transfusion per month ≥1. Or hemoglobin can not be maintained at 90g/L above, if blood transfusions is less than once per month. * Serum ferritin ≥ 500 µg/L * Patients aged 16 and above * Volunteer for the trial and sign the informed consent. Exclusion Criteria: * Active hepatitis B (HBsAg positive, HBsAb negative) or hepatitis C (HCV antibody positive, detectable HCV RNA, and ALT beyond normal range) * Active gastrointestinal disease history (including: gastric ulcer, duodenal ulcer, stomach or esophageal varices, ulcerative colitis, Crohn's disease, gastrointestinal cancer, familial genetic multiple intestinal polyps), and History of gastrointestinal perforation, gastrointestinal surgery that influence drug absorption, and other potential intestinal complications considered by researchers; * ALT or AST \> 2.5 × ULN, or serum creatinine \> 1.5 × ULN; * Neutropenia patient (neutrophil count \< 1.5 × 109 / L); * Active infection uncontrolled; * The patients who are currently taking CYP3A strong inducer or strong inhibitor drugs, or the drug that may extend the QT interval, or the drug that may decrease neutrophil count, but can not temporarily interrupt the use of such drugs; * The patients who are allergic or contraindicated to the main ingredients or excipients of CN128 tablets; * Congenital long QT syndrome or known family history of long QT syndrome; QTc \> 480 ms; clinically significant ventricular or atrial fast arrhythmia; * The patients who can not accept MRI as detection means, such as claustrophobic for MRI, pacemaker, and those using ferromagnetic metal implants; * Birth planner (including male subjects) within or within 3 months after the end of the trial; * Participated in other clinical trials in the three months before taking the test preparation, except for non-interventional studies; * Pregnant or lactating women; * Unsuitable to participate in the trial considered by the researchers. **Maximum Age:** 60 Years **Minimum Age:** 16 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Nanning **Country:** China **Facility:** The First Affiliated Hospital Of Guangxi Medical University **State:** Guangxi **Zip:** 530021 #### Overall Officials **Official 1:** **Affiliation:** First Affiliated Hospital of Guangxi Medical University **Name:** Jianmin Jianmin, PhD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000000745 - Term: Anemia, Hemolytic, Congenital - ID: D000000743 - Term: Anemia, Hemolytic - ID: D000000740 - Term: Anemia - ID: D000006402 - Term: Hematologic Diseases - ID: D000006453 - Term: Hemoglobinopathies - ID: D000030342 - Term: Genetic Diseases, Inborn - ID: D000019189 - Term: Iron Metabolism Disorders - ID: D000008659 - Term: Metabolic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC15 - Name: Blood and Lymph Conditions - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - Abbrev: All - Name: All Conditions - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M16557 - Name: Thalassemia - Relevance: HIGH - As Found: Thalassemia - ID: M21178 - Name: Iron Overload - Relevance: HIGH - As Found: Iron Overload - ID: M4070 - Name: Anemia - Relevance: LOW - As Found: Unknown - ID: M9547 - Name: Hemolysis - Relevance: LOW - As Found: Unknown - ID: M4073 - Name: Anemia, Hemolytic - Relevance: LOW - As Found: Unknown - ID: M4075 - Name: Anemia, Hemolytic, Congenital - Relevance: LOW - As Found: Unknown - ID: M9490 - Name: Hematologic Diseases - Relevance: LOW - As Found: Unknown - ID: M9539 - Name: Hemoglobinopathies - Relevance: LOW - As Found: Unknown - ID: M23686 - Name: Genetic Diseases, Inborn - Relevance: LOW - As Found: Unknown - ID: M11639 - Name: Metabolic Diseases - Relevance: LOW - As Found: Unknown - ID: M21177 - Name: Iron Metabolism Disorders - Relevance: LOW - As Found: Unknown - ID: T5622 - Name: Thalassemia - Relevance: HIGH - As Found: Thalassemia ### Condition Browse Module - Meshes - ID: D000013789 - Term: Thalassemia - ID: D000019190 - Term: Iron Overload ### Intervention Browse Module - Browse Branches - Abbrev: Micro - Name: Micronutrients - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M10533 - Name: Iron - Relevance: LOW - As Found: Unknown - ID: M5860 - Name: Chelating Agents - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01990079 **Acronym:** QUIT4EVER **Brief Title:** Use of Technological Advances to Prevent Smoking Relapse Among Smokers With PTSD **Official Title:** Use of Technological Advances to Prevent Smoking Relapse Among Smokers With PTSD #### Organization Study ID Info **ID:** Pro00048990 #### Organization **Class:** OTHER **Full Name:** Duke University ### Status Module #### Completion Date **Date:** 2015-08 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2015-09-17 **Type:** ESTIMATED **Last Update Submit Date:** 2015-09-15 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2015-08 **Type:** ACTUAL #### Start Date **Date:** 2013-12 **Status Verified Date:** 2015-09 #### Study First Post Date **Date:** 2013-11-21 **Type:** ESTIMATED **Study First Submit Date:** 2013-11-15 **Study First Submit QC Date:** 2013-11-15 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Duke University #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The primary goal of the study is to evaluate the use of a new smart phone application in preventing relapse to smoking among people with PTSD. The technology intervention will combine a mobile system to reward non-smoking, smoking cessation counseling, smoking cessation medications, and use of the smart phone app. The primary aim is to evaluate how effective this intervention is in preventing smoking relapse compared to another intervention that does not include the app. **Detailed Description:** The primary goal of the current study will be to evaluate the use of a new smart phone application (app; Stay Quit Coach) in preventing relapse to smoking among individuals with PTSD. The enhanced technology intervention will combine mobile contingency management (mCM), guideline-based smoking cessation counseling, bupropion and nicotine replacement therapy (NRT), and use of the Stay Quit Coach. For this study, we will propose a clinical trial with a two-group design in which 20 smokers with PTSD will be randomized to either: QUIT4EVER, an intervention that combines guideline-based smoking cessation counseling, bupropion and NRT, mCM and Stay Quit Coach. COMBINED CONTACT CONTROL (CCC) an intervention that is identical to QUIT4EVER except Stay Quit Coach will not be included. The CCC controls for compensation, monitoring, time and attention effects. Specific aims are to: AIM 1: evaluate the efficacy of QUIT4EVER on rates of abstinence from cigarettes (assessed with multiple measures including bioverified abstinence) during short and long term abstinence (measured at 3 and 6 months). Hypothesis 1: QUIT4EVER will be associated with increased long term abstinence (self-reported and bio-verified prolonged abstinence at the 3 and 6 month follow-up). AIM 2: assess the impact of QUIT4EVER on counseling treatment completion and medication adherence. Hypothesis 2. Increased abstinence associated with QUIT4EVER will be partially mediated by increased telephone counseling treatment completion and greater medication adherence. AIM 3: calculate the relative cost-effectiveness of the QUIT4EVER intervention in quality adjusted life years (QALY). Hypothesis 3: QUIT4EVER based treatment will result in greater cost-effectiveness compared to the control condition as measured by the incremental cost-effectiveness ratio. Overall, results of this study could lead to a highly efficient, effective, and easily disseminated treatment method for reducing smoking among smokers with PTSD and other psychiatric disorders. ### Conditions Module **Conditions:** - PTSD - Smoking **Keywords:** - PTSD - smoking ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 15 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** QUIT4EVER is an intervention that combines 4 guideline-based smoking cessation counseling sessions, bupropion and nicotine replacement therapy, mobile contingency management, and the smart-phone application Stay Quit Coach. **Intervention Names:** - Drug: Bupropion - Drug: nicotine replacement therapy - Other: Smoking cessation counseling - Behavioral: mobile contingency management - Other: Stay Quit Coach **Label:** QUIT4Ever **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** This intervention combines 4 guideline-based smoking cessation counseling sessions, bupropion and nicotine replacement therapy, and mobile contingency management. **Intervention Names:** - Drug: Bupropion - Drug: nicotine replacement therapy - Other: Smoking cessation counseling - Behavioral: mobile contingency management **Label:** Control Contact Condition **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Control Contact Condition - QUIT4Ever **Description:** All participants who are medically eligible will be prescribed bupropion, which they will start two weeks prior to their quit day. Dosage will be 150 mg/daily for days 1-7 and 300 mg/daily (administered in two daily doses) until the 3-month follow-up. **Name:** Bupropion **Other Names:** - Zyban, Wellbutrin **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Control Contact Condition - QUIT4Ever **Description:** Participants will be prescribed NRT patch and one nicotine rescue method (e.g., nicotine gum, lozenge, inhaler) for use during the post-quit phase of the study. Participants will be given the choice between nicotine gum, nicotine inhaler, or nicotine nasal spray, and will be instructed to use the rescue method as needed to reduce cigarette cravings **Name:** nicotine replacement therapy **Other Names:** - nicotine gum, patch, inhaler, or lozenge **Type:** DRUG #### Intervention 3 **Arm Group Labels:** - Control Contact Condition - QUIT4Ever **Description:** Participants will receive four 20-minute smoking cessation counseling sessions and a participant manual. The four sessions are based on standard cognitive-behavioral therapy techniques shown to be efficacious for smoking cessation. **Name:** Smoking cessation counseling **Type:** OTHER #### Intervention 4 **Arm Group Labels:** - Control Contact Condition - QUIT4Ever **Description:** Participants will be asked to provide video recordings of themselves taking carbon monoxide readings in order to confirm smoking abstinence. Participants are asked to upload these videos to the study's secured server, and are provided monetary reward for videos that suggest smoking abstinence. **Name:** mobile contingency management **Other Names:** - mCM **Type:** BEHAVIORAL #### Intervention 5 **Arm Group Labels:** - QUIT4Ever **Description:** Stay Quit Coach is a smart phone application that serves as a source of readily available support and information for adults who are already in treatment to quit smoking and to help them stay quit after treatment. The app guides user in creating tailored plans that include their personal reasons for quitting, interactive tools to help users cope with urges to smoke, motivational messages, support contacts to help users stay smoke free and how to address lapses. Participants assigned to this condition will be asked to use Stay Quit Coach from Session 2 through the 6-month follow-up. **Name:** Stay Quit Coach **Other Names:** - smart phone app **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Participants' self-report of smoking in the past seven days will be measured at the end of the treatment intervention, and at 3 and 6-month follow-up contacts. **Measure:** smoking, self-report **Time Frame:** 6 months follow-up #### Secondary Outcomes **Description:** For participants reporting smoking abstinence at 3 and 6-months post treatment follow-ups, we will bio-verify smoking abstinence by collecting saliva samples that will be used to determine salivary cotinine levels. **Measure:** saliva cotinine **Time Frame:** 6 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Meets criteria for current PTSD; 2. Has current smoking status of at least 10 cigarettes per day (verified with breath carbon monoxide measurement); 3. Has been smoking for at least 1 year; 4. Is aged 18 to 70; 5. Can speak and write current fluent conversation English; and 6. Is willing to make a smoking cessation attempt. Exclusion Criteria: 1. Is pregnant; 2. Has diagnosis, based on DSM-IV criteria, of schizophrenia, schizophreniform disorder, schizoaffective disorder, current psychotic symptoms, delusional disorder, current (not in remission) substance use disorder, and/or current manic episode; 3. Will not be stable on medications for the study period; 4. Has history of myocardial infarction in past 6 months; 5. Uses any other forms of nicotine such as cigars, pipes, or chewing tobacco with unwillingness to stop use during study period; or 6. Is currently imprisoned. 7. Note: Participants may be excluded or asked to refrain from taking certain study medications if they have a seizure disorder, uncontrolled diabetes, an eating disorder, or current or past cirrhosis or hepatitis. **Maximum Age:** 70 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Durham **Country:** United States **Facility:** Duke University Medical Center **State:** North Carolina **Zip:** 27706 #### Overall Officials **Official 1:** **Affiliation:** Duke University **Name:** Jean C. Beckham, Ph.D. **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Hicks TA Bs, Thomas SP, Wilson SM, Calhoun PS, Kuhn ER, Beckham JC. A Preliminary Investigation of a Relapse Prevention Mobile Application to Maintain Smoking Abstinence Among Individuals With Posttraumatic Stress Disorder. J Dual Diagn. 2017 Jan-Mar;13(1):15-20. doi: 10.1080/15504263.2016.1267828. Epub 2016 Dec 5. **PMID:** 27918881 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000040921 - Term: Stress Disorders, Traumatic - ID: D000068099 - Term: Trauma and Stressor Related Disorders - ID: D000001523 - Term: Mental Disorders - ID: D000020969 - Term: Disease Attributes - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: BC26 - Name: Wounds and Injuries ### Condition Browse Module - Browse Leaves - ID: M14850 - Name: Recurrence - Relevance: HIGH - As Found: Relapse - ID: M16103 - Name: Stress Disorders, Post-Traumatic - Relevance: HIGH - As Found: PTSD - ID: M24916 - Name: Stress Disorders, Traumatic - Relevance: LOW - As Found: Unknown - ID: M17685 - Name: Wounds and Injuries - Relevance: LOW - As Found: Unknown - ID: M222 - Name: Trauma and Stressor Related Disorders - Relevance: LOW - As Found: Unknown - ID: M4815 - Name: Mental Disorders - Relevance: LOW - As Found: Unknown - ID: M14473 - Name: Psychotic Disorders - Relevance: LOW - As Found: Unknown - ID: M22700 - Name: Disease Attributes - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000012008 - Term: Recurrence - ID: D000013313 - Term: Stress Disorders, Post-Traumatic ### Intervention Browse Module - Ancestors - ID: D000005731 - Term: Ganglionic Stimulants - ID: D000001337 - Term: Autonomic Agents - ID: D000018373 - Term: Peripheral Nervous System Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018722 - Term: Nicotinic Agonists - ID: D000018679 - Term: Cholinergic Agonists - ID: D000018678 - Term: Cholinergic Agents - ID: D000018377 - Term: Neurotransmitter Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000018687 - Term: Antidepressive Agents, Second-Generation - ID: D000000928 - Term: Antidepressive Agents - ID: D000011619 - Term: Psychotropic Drugs - ID: D000018765 - Term: Dopamine Uptake Inhibitors - ID: D000014179 - Term: Neurotransmitter Uptake Inhibitors - ID: D000049990 - Term: Membrane Transport Modulators - ID: D000015259 - Term: Dopamine Agents - ID: D000065690 - Term: Cytochrome P-450 CYP2D6 Inhibitors - ID: D000065607 - Term: Cytochrome P-450 Enzyme Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors ### Intervention Browse Module - Browse Branches - Abbrev: PsychDr - Name: Psychotropic Drugs - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: CNSSti - Name: Central Nervous System Stimulants - Abbrev: CaAg - Name: Cardiotonic Agents ### Intervention Browse Module - Browse Leaves - ID: M19013 - Name: Bupropion - Relevance: HIGH - As Found: 10 days - ID: M12478 - Name: Nicotine - Relevance: HIGH - As Found: Prior to - ID: M5506 - Name: Carbon Monoxide - Relevance: LOW - As Found: Unknown - ID: M4029 - Name: Central Nervous System Stimulants - Relevance: LOW - As Found: Unknown - ID: M20796 - Name: Nicotinic Agonists - Relevance: LOW - As Found: Unknown - ID: M20758 - Name: Cholinergic Agents - Relevance: LOW - As Found: Unknown - ID: M20504 - Name: Neurotransmitter Agents - Relevance: LOW - As Found: Unknown - ID: M4247 - Name: Antidepressive Agents - Relevance: LOW - As Found: Unknown - ID: M14474 - Name: Psychotropic Drugs - Relevance: LOW - As Found: Unknown - ID: M7473 - Name: Dopamine - Relevance: LOW - As Found: Unknown - ID: M20832 - Name: Dopamine Uptake Inhibitors - Relevance: LOW - As Found: Unknown - ID: M17962 - Name: Dopamine Agents - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown - ID: M30537 - Name: Cytochrome P-450 Enzyme Inhibitors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000016642 - Term: Bupropion - ID: D000009538 - Term: Nicotine ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01180179 **Brief Title:** PPI vs H2RA in Patients With Helicobacter Pylori-Negative Idiopathic Bleeding Ulcers **Official Title:** Prevention of Recurrent Idiopathic Gastroduodenal Ulcer Bleeding: a Double-blind Randomized Trial #### Organization Study ID Info **ID:** NNH_RCT #### Organization **Class:** OTHER **Full Name:** Chinese University of Hong Kong ### Status Module #### Completion Date **Date:** 2019-05 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2019-08-01 **Type:** ACTUAL **Last Update Submit Date:** 2019-07-31 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2019-05 **Type:** ACTUAL #### Start Date **Date:** 2010-06 **Type:** ACTUAL **Status Verified Date:** 2019-07 #### Study First Post Date **Date:** 2010-08-12 **Type:** ESTIMATED **Study First Submit Date:** 2010-08-03 **Study First Submit QC Date:** 2010-08-11 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Chinese University of Hong Kong #### Responsible Party **Investigator Affiliation:** Chinese University of Hong Kong **Investigator Full Name:** Francis KL Chan **Investigator Title:** Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The aim of this study is to compare the efficacy of a proton pump inhibitor (lansoprazole) and a histamine-2 receptor antagonist (famotidine) in preventing recurrent ulcer bleeding in patients with a history of H. pylori-negative idiopathic peptic ulcers. **Detailed Description:** Peptic ulcer disease used to be caused by a bacterial infection (Helicobacter pylori) in the stomach or the use of certain painkillers (nonsteroidal anti-inflammatory drugs or NSAIDs). However, there has been an increasing trend of peptic ulcer disease with unknown cause (idiopathic ulcer) worldwide since the last decade. Studies in North America found that idiopathic ulcers accounted for 11% and 44% of all peptic ulcers. A meta-analysis of 7 US trials found that 20% of patients with H. pylori-associated ulcers had recurrent ulcers within 6 months, despite successful cure of H. pylori infection and no reported use of NSAIDs. In a pooled analysis of 6 clinical trials with a total of 2900 patients, 27% of duodenal ulcers were not associated with NSAID use or H. pylori infection. The emerging problem of H. pylori-negative idiopathic peptic ulcers is not only limited to western countries. Previously, H. pylori-negative idiopathic peptic ulcers accounted for less than 5% of peptic ulcers in Asia. A recent Korean study reported that the proportion of peptic ulcers not associated with H. pylori infection or NSAID use was over 20%. ### Conditions Module **Conditions:** - Peptic Ulcer **Keywords:** - H. pylori-negative idiopathic peptic ulcers - Recurrent ulcer bleeding ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** QUADRUPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 228 **Type:** ACTUAL **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Lansoprazole 30mg once daily **Intervention Names:** - Drug: Lansoprazole **Label:** Lansoprazole 30mg once daily **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Famotidine 40mg once daily **Intervention Names:** - Drug: Famotidine **Label:** Famotidine 40mg once daily **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Lansoprazole 30mg once daily **Description:** 30mg once daily **Name:** Lansoprazole **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Famotidine 40mg once daily **Description:** 40mg once daily **Name:** Famotidine **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** According to prespecified criteria - hematemesis or melena documented by the admitting physician, or a decrease in the hemoglobin level of at least 2 g/dL, with ulcers or bleeding erosions confirmed on endoscopy. A prespecified interim-analysis is performed on the primary endpoint when all patients have been randomised and have completed the 12 months follow-up. The interim-analysis is performed by an independent statistician, blinded for the treatment allocation. The statistician will report to the independent data and safety monitoring committee (DSMC). The DSMC will have unblinded access to all data and will discuss the results of the interim-analysis with the steering committee in a joint meeting. The steering committee decides on the continuation of the trial and will report to the central ethics committee. The Peto approach is used: the trial will be ended using symmetric stopping boundaries at P \< 0.001. **Measure:** Recurrent ulcer bleeding **Time Frame:** 24 months #### Secondary Outcomes **Description:** Recurrent ulcer detected by endoscopy at 24-month, with or without clinical symptoms. **Measure:** Recurrent ulcer detected by endoscopy at 24-month **Time Frame:** at the 24th month of follow-up ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. A history of H. pylori-negative idiopathic peptic ulcers, defined as 1. No exposure to aspirin, NSAIDs or drugs of unknown nature including traditional Chinese medicine within the 4 weeks before hospitalization; 2. Biopsies taken during endoscopy must be negative for both the urease test and histology for H. pylori in the absence of acid suppressive therapy; and 3. No other causes of ulceration identified. 2. Endoscopically confirmed ulcer healing 3. Age \>18 years old 4. Informed consent Exclusion Criteria: 1. Concomitant steroid or anticoagulant 2. Concomitant use of NSAIDs, aspirin or COX2 inhibitors 3. Previous gastric surgery 4. Requirement of maintenance PPI (e.g. reflux oesophagitis) 5. Advanced comorbidity (defined as ASA 4 or above) or active malignancy 6. Subjects who are pregnant or lactating, or is intending to become pregnant before, during, or within 1 month after participating in this study 7. Subjects who have known hypersensitivity or allergies to any component of lansoprazole or famotidine. 8. Subject who has current or historical evidence of Zollinger-Ellison syndrome or other hypersecretory condition **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Hong Kong **Country:** China **Facility:** Endoscopy Center, Prince of Wales Hospital, Shatin #### Overall Officials **Official 1:** **Affiliation:** Chinese University of Hong Kong **Name:** Grace L Wong, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Wong GLH, Lau LHS, Ching JYL, Tse YK, Ling RHY, Wong VWS, Chiu PWY, Lau JYW, Chan FKL. Prevention of recurrent idiopathic gastroduodenal ulcer bleeding: a double-blind, randomised trial. Gut. 2020 Apr;69(4):652-657. doi: 10.1136/gutjnl-2019-318715. Epub 2019 Jun 22. **PMID:** 31229990 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010335 - Term: Pathologic Processes - ID: D000004378 - Term: Duodenal Diseases - ID: D000007410 - Term: Intestinal Diseases - ID: D000005767 - Term: Gastrointestinal Diseases - ID: D000004066 - Term: Digestive System Diseases - ID: D000013272 - Term: Stomach Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC06 - Name: Digestive System Diseases ### Condition Browse Module - Browse Leaves - ID: M9556 - Name: Hemorrhage - Relevance: HIGH - As Found: Bleeding - ID: M17206 - Name: Ulcer - Relevance: HIGH - As Found: Ulcer - ID: M14850 - Name: Recurrence - Relevance: LOW - As Found: Unknown - ID: M13348 - Name: Peptic Ulcer - Relevance: HIGH - As Found: Peptic Ulcer - ID: M7552 - Name: Duodenal Diseases - Relevance: LOW - As Found: Unknown - ID: M10444 - Name: Intestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M16062 - Name: Stomach Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000010437 - Term: Peptic Ulcer - ID: D000014456 - Term: Ulcer - ID: D000006470 - Term: Hemorrhage ### Intervention Browse Module - Ancestors - ID: D000000897 - Term: Anti-Ulcer Agents - ID: D000005765 - Term: Gastrointestinal Agents - ID: D000054328 - Term: Proton Pump Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000006635 - Term: Histamine H2 Antagonists - ID: D000006633 - Term: Histamine Antagonists - ID: D000018494 - Term: Histamine Agents - ID: D000018377 - Term: Neurotransmitter Agents - ID: D000045505 - Term: Physiological Effects of Drugs ### Intervention Browse Module - Browse Branches - Abbrev: Gast - Name: Gastrointestinal Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M18303 - Name: Famotidine - Relevance: HIGH - As Found: Mobile Health - ID: M30379 - Name: Lansoprazole - Relevance: HIGH - As Found: Marginal - ID: M30380 - Name: Dexlansoprazole - Relevance: HIGH - As Found: Marginal - ID: M4188 - Name: Antacids - Relevance: LOW - As Found: Unknown - ID: M4219 - Name: Anti-Ulcer Agents - Relevance: LOW - As Found: Unknown - ID: M8881 - Name: Gastrointestinal Agents - Relevance: LOW - As Found: Unknown - ID: M27630 - Name: Proton Pump Inhibitors - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown - ID: M9708 - Name: Histamine - Relevance: LOW - As Found: Unknown - ID: M9711 - Name: Histamine H2 Antagonists - Relevance: LOW - As Found: Unknown - ID: M212144 - Name: Histamine phosphate - Relevance: LOW - As Found: Unknown - ID: M9709 - Name: Histamine Antagonists - Relevance: LOW - As Found: Unknown - ID: M20504 - Name: Neurotransmitter Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000064747 - Term: Lansoprazole - ID: D000064748 - Term: Dexlansoprazole - ID: D000015738 - Term: Famotidine ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02351479 **Brief Title:** Hula, a Physical Activity Intervention for Female-Cancer Survivors **Official Title:** Hula, a Physical Activity Intervention for Female-Cancer Survivors #### Organization Study ID Info **ID:** Bantum-2013-2 #### Organization **Class:** OTHER **Full Name:** University of Hawaii ### Status Module #### Completion Date **Date:** 2017-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2019-04-18 **Type:** ACTUAL **Last Update Submit Date:** 2019-04-16 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2017-12 **Type:** ACTUAL #### Start Date **Date:** 2013-09 **Status Verified Date:** 2019-04 #### Study First Post Date **Date:** 2015-01-30 **Type:** ESTIMATED **Study First Submit Date:** 2015-01-20 **Study First Submit QC Date:** 2015-01-29 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Hawaii #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** A six-month interventional program to determine the biological and psychosocial effects of hula as a form of physical activity for female breast, cervical, endometrial or ovarian cancer survivors living on Oahu. ### Conditions Module **Conditions:** - Physical Activity - Breast Cancer - Cervical Cancer - Ovarian Cancer - Endometrial Cancer **Keywords:** - hula - breast cancer - female - hawaii - physical activity - cervical cancer - ovarian cancer - endometrial cancer ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Count:** 73 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Hula is an ancient, Native Hawaiian dance form of cultural expression and physical activity. Participants will attend one-hour hula classes twice a week for six months. **Intervention Names:** - Behavioral: Hula **Label:** Hula **Type:** OTHER ### Interventions #### Intervention 1 **Arm Group Labels:** - Hula **Name:** Hula **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** Compliance as measured by the proportion of total sessions attended and a questionnaire regarding satisfaction with the program at the end of the intervention **Measure:** Hula Program Feasibility (Compliance as measured by the proportion of total sessions attended and a questionnaire regarding satisfaction with the program at the end of the intervention) **Time Frame:** 6 months after the start of the intervention program #### Secondary Outcomes **Description:** Circulating levels of sex hormones measured in serum samples. **Measure:** Biomarkers - Sex Hormones (Circulating levels of sex hormones measured in serum samples) **Time Frame:** Baseline, 6 months and 12 months after the start of the intervention program **Description:** Circulating levels of cytokines measured in serum samples. **Measure:** Biomarkers - Cytokines (Circulating levels of cytokines measured in serum samples.) **Time Frame:** Baseline, 6 months and 12 months after the start of the intervention program **Description:** Circulating levels of leptin, CRP, IGF-1, and IGFBP3 measured in serum samples collectively. **Measure:** Biomarkers - Inflammatory Markers (Circulating levels of leptin, CRP, IGF-1, and IGFBP3 measured in serum samples collectively.) **Time Frame:** Baseline, 6 months and 12 months after the start of the intervention program **Description:** DNA methylation patterns as detected with the Illumina Assay containing methylation sites in RefSeq genes and CpG islands collectively. **Measure:** DNA Methylation Patterns (DNA methylation patterns as detected with the Illumina Assay containing methylation sites in RefSeq genes and CpG islands collectively.) **Time Frame:** Baseline, 6 months and 12 months after the start of the intervention program **Description:** Collectively looking at demographic items as measured on the baseline questionnaire, including ethnicity, education, family, history of cancer, stage of cancer, treatment type, and comorbidities. **Measure:** Self-Report Outcomes - Demographics (Collectively looking at demographic items as measured on the baseline questionnaire, including ethnicity, education, family, history of cancer, stage of cancer, treatment type, and comorbidities.) **Time Frame:** Baseline, 6 months and 12 months after the start of the intervention program **Description:** Minutes per week of exercise assessed using The Women's Health Initiative Physical Activity Questionnaire **Measure:** Self-Report Outcomes - Physical Activity (Minutes per week of exercise assessed using The Women's Health Initiative Physical Activity Questionnaire) **Time Frame:** Baseline, 6 months and 12 months after the start of the intervention program **Measure:** Self-Report Outcomes - Health-Related Quality of Life (Quality of life of participants assessed using The European Organization for Research and Treatment of Cancer QLQ-C30.) **Time Frame:** Baseline, 6 months and 12 months after the start of the intervention program **Description:** The severity and impact of fatigue on daily functioning assessed using The Brief Fatigue Inventory (BFI). **Measure:** Self-Report Outcomes - Fatigue (assessed using The Brief Fatigue Inventory (BFI) **Time Frame:** Baseline, 6 months and 12 months after the start of the intervention program **Description:** The presence of depression as indicated using The Centers for Epidemiological Studies Depression Scale (CES-ED). **Measure:** Self-Report Outcomes - Depression (indicated using The Centers for Epidemiological Studies Depression Scale (CES-ED) **Time Frame:** Baseline, 6 months and 12 months after the start of the intervention program **Description:** Affect of participants assessed using The Profile of Mood States short form (POMS-SF) questionnaire. **Measure:** Self-Report Outcomes - Affective States (assessed using The Profile of Mood States short form (POMS-SF) questionnaire.) **Time Frame:** Baseline, 6 months and 12 months after the start of the intervention program **Description:** How people adjust and cope with a cancer diagnosis assessed using The 10-item Social Constraints Scale. **Measure:** Self-Report Outcomes - Social Constraints (assessed using The 10-item Social Constraints Scale.) **Time Frame:** Baseline, 6 months and 12 months after the start of the intervention program **Description:** 2 subscales assess cognitive deficiency and cognitive capability **Measure:** Self-Report Outcomes - Cognitive Functioning (assessed using the 42-item FACTcog **Time Frame:** Baseline, 6 months and 12 months after the start of the intervention program ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Lives on Oahu * Diagnosed with primary breast,cervical, endometrial or ovarian cancer (stage I-III) * Completed initial regional and systemic treatment for breast cancer 2 months ago * Physically capable of doing the hula-based physical activity * Receives approval by attending physician to participate in trial Exclusion Criteria: * Currently undergoing chemotherapy or radiation therapy **Minimum Age:** 21 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Honolulu **Country:** United States **Facility:** University of Hawaii Cancer Center **State:** Hawaii **Zip:** 96813 #### Overall Officials **Official 1:** **Affiliation:** University of Hawaii Cancer Research Center **Name:** Erin Bantum, MA, PhD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000004701 - Term: Endocrine Gland Neoplasms - ID: D000010049 - Term: Ovarian Diseases - ID: D000000291 - Term: Adnexal Diseases - ID: D000005831 - Term: Genital Diseases, Female - ID: D000052776 - Term: Female Urogenital Diseases - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications - ID: D000091642 - Term: Urogenital Diseases - ID: D000005833 - Term: Genital Neoplasms, Female - ID: D000014565 - Term: Urogenital Neoplasms - ID: D000091662 - Term: Genital Diseases - ID: D000004700 - Term: Endocrine System Diseases - ID: D000006058 - Term: Gonadal Disorders - ID: D000014594 - Term: Uterine Neoplasms - ID: D000002577 - Term: Uterine Cervical Diseases - ID: D000014591 - Term: Uterine Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: All - Name: All Conditions - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: BC19 - Name: Gland and Hormone Related Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M5220 - Name: Breast Neoplasms - Relevance: LOW - As Found: Unknown - ID: M5830 - Name: Uterine Cervical Neoplasms - Relevance: HIGH - As Found: Cervical Cancer - ID: M12974 - Name: Ovarian Neoplasms - Relevance: HIGH - As Found: Ovarian Cancer - ID: M1704 - Name: Carcinoma, Ovarian Epithelial - Relevance: LOW - As Found: Unknown - ID: M19235 - Name: Endometrial Neoplasms - Relevance: HIGH - As Found: Endometrial Cancer - ID: M7863 - Name: Endocrine Gland Neoplasms - Relevance: LOW - As Found: Unknown - ID: M12972 - Name: Ovarian Diseases - Relevance: LOW - As Found: Unknown - ID: M3643 - Name: Adnexal Diseases - Relevance: LOW - As Found: Unknown - ID: M8943 - Name: Genital Diseases, Female - Relevance: LOW - As Found: Unknown - ID: M2876 - Name: Genital Diseases - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M8945 - Name: Genital Neoplasms, Female - Relevance: LOW - As Found: Unknown - ID: M17315 - Name: Urogenital Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7862 - Name: Endocrine System Diseases - Relevance: LOW - As Found: Unknown - ID: M9163 - Name: Gonadal Disorders - Relevance: LOW - As Found: Unknown - ID: M17342 - Name: Uterine Neoplasms - Relevance: LOW - As Found: Unknown - ID: M5825 - Name: Uterine Cervical Diseases - Relevance: LOW - As Found: Unknown - ID: M17339 - Name: Uterine Diseases - Relevance: LOW - As Found: Unknown - ID: T4352 - Name: Ovarian Cancer - Relevance: HIGH - As Found: Ovarian Cancer - ID: T4354 - Name: Ovarian Epithelial Cancer - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000010051 - Term: Ovarian Neoplasms - ID: D000002583 - Term: Uterine Cervical Neoplasms - ID: D000016889 - Term: Endometrial Neoplasms ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01757379 **Brief Title:** Determination of the Bioavailability of Short Chain Fatty Acids in Healthy Humans **Official Title:** Evaluation of the Bioavailability and Production of Short Chain Fatty Acids in the Colon. A Stable Isotope Study in Healthy Humans. #### Organization Study ID Info **ID:** ML5768 #### Organization **Class:** OTHER **Full Name:** KU Leuven ### Status Module #### Expanded Access Info #### Last Update Post Date **Date:** 2015-01-14 **Type:** ESTIMATED **Last Update Submit Date:** 2015-01-13 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2012-04 **Type:** ACTUAL #### Start Date **Date:** 2011-04 **Status Verified Date:** 2015-01 #### Study First Post Date **Date:** 2012-12-28 **Type:** ESTIMATED **Study First Submit Date:** 2012-12-21 **Study First Submit QC Date:** 2012-12-21 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** KU Leuven #### Responsible Party **Investigator Affiliation:** KU Leuven **Investigator Full Name:** Kristin Verbeke **Investigator Title:** Professor K. Verbeke **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of the study is to evaluate the bioavailability of acetate, propionate and butyrate in healthy humans using a stable isotope technology. In addition the level of acetate, propionate and butyrate production from inulin will be determined using the principle of isotope dilution. ### Conditions Module **Conditions:** - Short Chain Fatty Acids Bioavailability **Keywords:** - Short chain fatty acids - Acetate - Propionate - Butyrate - Bioavailability ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER ##### Masking Info **Masking:** SINGLE **Who Masked:** - PARTICIPANT **Primary Purpose:** BASIC_SCIENCE #### Enrollment Info **Count:** 12 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Dietary Supplement: 13C-labeled Acetate **Label:** 13C-labeled acetate **Type:** EXPERIMENTAL #### Arm Group 2 **Intervention Names:** - Dietary Supplement: 13C-labeled propionate **Label:** 13C-labeled propionate **Type:** EXPERIMENTAL #### Arm Group 3 **Intervention Names:** - Dietary Supplement: 13C-labeled butyrate **Label:** 13C-labeled butyrate **Type:** EXPERIMENTAL #### Arm Group 4 **Intervention Names:** - Dietary Supplement: Inulin **Label:** Inulin **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - 13C-labeled acetate **Description:** 400 mg of sodium acetate 1-13C (2 colon delivery capsules with 200 mg) **Name:** 13C-labeled Acetate **Type:** DIETARY_SUPPLEMENT #### Intervention 2 **Arm Group Labels:** - 13C-labeled propionate **Description:** 340 mg of sodium propionate 1-13C (2 colon delivery capsules with 170 mg) **Name:** 13C-labeled propionate **Type:** DIETARY_SUPPLEMENT #### Intervention 3 **Arm Group Labels:** - 13C-labeled butyrate **Description:** 990 mg sodium butyrate 1-13C (2 colon delivery capsules with 495 mg) **Name:** 13C-labeled butyrate **Type:** DIETARY_SUPPLEMENT #### Intervention 4 **Arm Group Labels:** - Inulin **Description:** 15 g of inulin dissolved in 200 ml of water **Name:** Inulin **Type:** DIETARY_SUPPLEMENT ### Outcomes Module #### Primary Outcomes **Description:** Participants performed each 4 test days and they were followed each test day for 12 hours, with sample collections at regular time points. Blood samples were collected every hour during the first 4 hours and afterwards every 20 minutes during 8 hours. Urine samples were collected during 24h in different fractions: 0-4h, 4-8h, 8-12h, 12-24h. **Measure:** Concentrations of the short chain fatty acids (acetate, propionate and butyrate) in plasma and urine samples **Time Frame:** 12 hours each test day and 4 test days/volunteer ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Healthy volunteers * Regular dietary pattern (3 meals/day) * Age: 18-65y * BMI: 18,5-27 kg/m2 Exclusion Criteria: * Intake of antibiotics 1 month prior to the study * Abdominal surgery in the past, with the exception of appendectomy * Intake of medication influencing the gastro-intestinal system 14 days prior to the study * In treatment at a dietician * Intake of pre- and/or probiotics * Exposure to radioactivity 1 year prior to the study * Serious chronic disease of the gastrointestinal tract * Use of medication that affects the gastro-intestinal tract during the last 2 weeks prior to the study * Pregnancy, pregnancy desire or lactation * Blood donation during the last 3 months prior to the study * Diabetes (type 1 or 2) * Aberrant hemoglobin level (Hb) in blood. Normal between 14.0 and 18.0 g/dL for men and between 12.0 and 16.0 g/dL for women **Healthy Volunteers:** True **Maximum Age:** 65 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Leuven **Country:** Belgium **Facility:** KU Leuven/ UZ Leuven **State:** Vlaams-Brabant **Zip:** 3000 #### Overall Officials **Official 1:** **Affiliation:** KU Leuven **Name:** Kristin Verbeke, Professor **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Intervention Browse Module - Browse Branches - Abbrev: Ot - Name: Other Dietary Supplements - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: HB - Name: Herbal and Botanical ### Intervention Browse Module - Browse Leaves - ID: T401 - Name: Inulin - Relevance: HIGH - As Found: Sildenafil - ID: T120 - Name: Cola - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00575679 **Acronym:** BADS **Brief Title:** Behavior and Driving Safety Study **Official Title:** Behavior and Driving Safety Study #### Organization Study ID Info **ID:** 243197 #### Organization **Class:** OTHER **Full Name:** University of California, Davis #### Secondary ID Infos **ID:** UCDIRB-200614967 **Domain:** UC Davis **ID:** OTS-AL0757 **Type:** OTHER ### Status Module #### Completion Date **Date:** 2014-02-20 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2019-01-04 **Type:** ACTUAL **Last Update Submit Date:** 2019-01-02 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2014-02-20 **Type:** ACTUAL #### Start Date **Date:** 2008-01 **Type:** ACTUAL **Status Verified Date:** 2019-01 #### Study First Post Date **Date:** 2007-12-18 **Type:** ESTIMATED **Study First Submit Date:** 2007-12-14 **Study First Submit QC Date:** 2007-12-14 ### Sponsor Collaborators Module #### Collaborators **Class:** UNKNOWN **Name:** Sacramento Police Department **Class:** OTHER_GOV **Name:** California Office of Traffic Safety **Class:** OTHER **Name:** University of Michigan **Class:** OTHER **Name:** Robert Wood Johnson Foundation **Class:** UNKNOWN **Name:** Teachable Moment Foundation #### Lead Sponsor **Class:** OTHER **Name:** University of California, Davis #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to determine whether brief motivational interviews reduce the likelihood of driving under the influence of alcohol (DUI). **Detailed Description:** Despite extensive legislative, law enforcement, and public awareness efforts, driving under the influence of alcohol (DUI) remains a major cause of mortality and loss of years of productive life. A brief motivational interview (BI) is a non-confrontational, patient-centered discussion during which the individual's motivation and confidence to change health-related behaviors are explored. Previous studies of BIs in health care settings suggest that BIs administered after hospitalization for alcohol-related injury reduce the likelihood of repeat injury hospitalization and arrest for DUI. ### Conditions Module **Conditions:** - Alcoholic Intoxication **Keywords:** - Automobile driving - Driving under the influence - Interview, psychological - Brief intervention - Brief motivational interview ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - OUTCOMES_ASSESSOR **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 864 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Brief motivational interview **Intervention Names:** - Behavioral: Brief motivational interview **Label:** 1 **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** No discussion **Label:** 2 **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - 1 **Description:** The brief motivational interview is a non-confrontational, patient-centered discussion between a counselor (e.g., social worker, nurse, physician, or psychologist) and an at-risk individual during which the individual's motivation and confidence to change health-related behaviors are explored. **Name:** Brief motivational interview **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Measure:** Time to repeat arrest for driving under the influence of alcohol **Time Frame:** Up until one year after the conclusion of enrollment #### Secondary Outcomes **Measure:** Alcohol Use Disorders Identification Test score **Time Frame:** 6 and 12 months **Measure:** Time to alcohol-related injury hospitalization **Time Frame:** Up until one year after the conclusion of enrollment ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Second or greater arrest for driving under the influence of alcohol * Arrest involved operation of a motor vehicle * At least 18 years of age * English-speaking Exclusion Criteria: * Arrest involved injury to another person * Previous participation in this study **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Sacramento **Country:** United States **Facility:** Sacramento County Main Jail **State:** California **Zip:** 95814 #### Overall Officials **Official 1:** **Affiliation:** University of California, Davis **Name:** Garth H Utter, MD MSc **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** University of California, Davis **Name:** Leon J Owens, MD **Role:** STUDY_DIRECTOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000019973 - Term: Alcohol-Related Disorders - ID: D000019966 - Term: Substance-Related Disorders - ID: D000064419 - Term: Chemically-Induced Disorders - ID: D000001523 - Term: Mental Disorders ### Condition Browse Module - Browse Branches - Abbrev: BC25 - Name: Substance Related Disorders - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M3781 - Name: Alcoholic Intoxication - Relevance: HIGH - As Found: Alcoholic Intoxication - ID: M21842 - Name: Alcohol-Related Disorders - Relevance: LOW - As Found: Unknown - ID: M21837 - Name: Substance-Related Disorders - Relevance: LOW - As Found: Unknown - ID: M30302 - Name: Chemically-Induced Disorders - Relevance: LOW - As Found: Unknown - ID: M4815 - Name: Mental Disorders - Relevance: LOW - As Found: Unknown - ID: M14473 - Name: Psychotic Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000000435 - Term: Alcoholic Intoxication ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01355679 **Brief Title:** Molecular Guided Therapy for Refractory or Recurrent Neuroblastoma **Official Title:** A Feasibility Trial Using Molecular-Guided Therapy for the Treatment of Patients With Refractory or Recurrent Neuroblastoma **NCT ID Aliases:** - NCT01375517 #### Organization Study ID Info **ID:** NMTRC 001 #### Organization **Class:** OTHER **Full Name:** Milton S. Hershey Medical Center ### Status Module #### Completion Date **Date:** 2015-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2024-04-03 **Type:** ACTUAL **Last Update Submit Date:** 2024-04-01 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2013-09 **Type:** ACTUAL #### Results First Post Date **Date:** 2016-07-28 **Type:** ESTIMATED **Results First Submit Date:** 2015-05-18 **Results First Submit QC Date:** 2016-06-16 #### Start Date **Date:** 2011-05 **Status Verified Date:** 2024-04 #### Study First Post Date **Date:** 2011-05-18 **Type:** ESTIMATED **Study First Submit Date:** 2011-05-13 **Study First Submit QC Date:** 2011-05-17 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Giselle Sholler #### Responsible Party **Investigator Affiliation:** Milton S. Hershey Medical Center **Investigator Full Name:** Giselle Sholler **Investigator Title:** Study Chair **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this study is to test the feasibility (ability to be done) of an experimental test to help plan your cancer treatment. This study plan is not studying the effectiveness of the proposed combinations of therapy for your cancer that you may receive after the experimental testing. This study will look at an experimental technology to determine a tumor's molecular makeup (gene expression profile). This technology (called "OncInsights") is being used to discover new ways to understand cancers and potentially predict the best treatments for patients with cancer in the future. The experimental technology has not been approved by the U.S. Food and Drug Administration. ### Conditions Module **Conditions:** - Neuroblastoma ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** DIAGNOSTIC #### Enrollment Info **Count:** 16 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** A total of 14 eligible neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles). **Intervention Names:** - Device: Guided Therapy **Label:** Guided therapy **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Guided therapy **Description:** A total of 14 eligible neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles). **Name:** Guided Therapy **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Feasibility parameter defined as: Enrollment onto study, quality mRNA obtained, gene chip completed, tumor board held, medical monitor review and approval, start of treatment by 21 days post biopsy/surgical resection date, and then completion of 1 cycle of therapy." **Measure:** Percentage of Participants That Are Able to Meet Feasibility Parameters. **Time Frame:** 1 year #### Secondary Outcomes **Description:** To determine the safety of allowing a molecular tumor board to determine individualized treatment plans **Measure:** Number of Participants With Adverse Events as a Measure of Safety **Time Frame:** 1 year **Description:** Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR. **Measure:** Overall Response Rate (ORR) of Participants Using RECIST Criteria **Time Frame:** 1 year **Description:** Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions **Measure:** Activity of Treatments Chosen Based on Progression Free Survival (PFS) **Time Frame:** 1 year ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patients must have histologically proven neuroblastoma and confirmation of refractory or recurrent disease with histologic confirmation at diagnosis or at the time of recurrence/progression * Patients must be age \> 12 months and ≤ 21 at initial diagnosis. * Life expectancy must be more than 3 months * If measurable disease, this must be demonstrated by residual abnormal tissue at a primary or metastatic site measuring more than 1 cm in any dimension by standardized imaging (CT or MRI); tumor must be accessible for biopsy. Patients with bone marrow only disease expected to be \> 75% are eligible to enroll. * Current disease state must be one for which there is currently no known curative therapy * Lansky or KarnofskyScore must be more than 50 * Patients without bone marrow metastases must have an ANC \> 750/μl and platelet count \> 50,000/μl * Adequate liver function must be demonstrated, defined as: * Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND * SGPT (ALT) \< 10 x upper limit of normal (ULN) for age * No other significant organ toxicity defined as \> Grade 2 by National Cancer Institute Common Toxicity Criteria for Adverse Events NCI-CTCAE V4.0 * A negative serum pregnancy test is required for female participants of child bearing potential (≥ 13 years of age or after onset of menses) * Both male and female post-pubertal study subjects need to agree to use one of the more effective birth control methods during treatment and for six months after treatment is stopped. These methods include total abstinence (no sex), oral contraceptives ("the pill"), an intrauterine device (IUD), levonorgestrol implants (Norplant), or medroxyprogesterone acetate injections (Depo-provera shots). If one of these cannot be used, contraceptive foam with a condom is recommended. * Informed Consent: All patients and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines. Voluntary consent for optional biology studies will be included. Exclusion Criteria: * Patients who have received any chemotherapy within the last 7 days prior to enrollment and 14 days prior to study treatment start date. * Patients who have received any radiotherapy within the last 30 days must have another site of disease to follow. * Patients receiving anti-tumor therapy for their disease or any investigational drug concurrently * Patients with serious infection or a life-threatening illness (unrelated to tumor) that is \> Grade 2 (NCI CTCAE V4.0), or active, serious infections requiring parenteral antibiotic therapy. * Patients with any other medical condition, including malabsorption syndromes, mental illness or substance abuse, deemed by the Investigator to be likely to interfere with the interpretation of the results or which would interfere with a patient's ability to sign or the legal guardian's ability to sign the informed consent, and patient's ability to cooperate and participate in the study **Minimum Age:** 1 Year **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Hartford **Country:** United States **Facility:** Connecticut Children's Hospital **State:** Connecticut **Zip:** 06106 **Location 2:** **City:** Orlando **Country:** United States **Facility:** Arnold Palmer Hospital for Children- MD Anderson **State:** Florida **Zip:** 32806 **Location 3:** **City:** Bethesda **Country:** United States **Facility:** National Cancer Institute **State:** Maryland **Zip:** 20877 **Location 4:** **City:** Grand Rapids **Country:** United States **Facility:** Helen DeVos Children's Hospital **State:** Michigan **Zip:** 49503 **Location 5:** **City:** Kansas City **Country:** United States **Facility:** Children's Mercy Hospitals and Clinics **State:** Missouri **Zip:** 64108 **Location 6:** **City:** Saint Louis **Country:** United States **Facility:** Cardinal Glennon Children's Medical Center **State:** Missouri **Zip:** 63104 **Location 7:** **City:** Charlotte **Country:** United States **Facility:** Levine Children's Hospital **State:** North Carolina **Zip:** 28204 #### Overall Officials **Official 1:** **Affiliation:** Beat Childhood Cancer at Atrium Health **Name:** Giselle Sholler, MD **Role:** STUDY_CHAIR ### References Module #### See Also Links **Label:** Beat Childhood Cancer **URL:** https://beatcc.org/ ## Derived Section ### Condition Browse Module - Ancestors - ID: D000018241 - Term: Neuroectodermal Tumors, Primitive, Peripheral - ID: D000018242 - Term: Neuroectodermal Tumors, Primitive - ID: D000018302 - Term: Neoplasms, Neuroepithelial - ID: D000017599 - Term: Neuroectodermal Tumors - ID: D000009373 - Term: Neoplasms, Germ Cell and Embryonal - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000009369 - Term: Neoplasms - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009380 - Term: Neoplasms, Nerve Tissue ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC04 - Name: Neoplasms - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M14850 - Name: Recurrence - Relevance: LOW - As Found: Unknown - ID: M12391 - Name: Neuroblastoma - Relevance: HIGH - As Found: Neuroblastoma - ID: M20387 - Name: Neuroectodermal Tumors, Primitive, Peripheral - Relevance: LOW - As Found: Unknown - ID: M19845 - Name: Neuroectodermal Tumors - Relevance: LOW - As Found: Unknown - ID: M20388 - Name: Neuroectodermal Tumors, Primitive - Relevance: LOW - As Found: Unknown - ID: M20446 - Name: Neoplasms, Neuroepithelial - Relevance: LOW - As Found: Unknown - ID: M12318 - Name: Neoplasms, Germ Cell and Embryonal - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: M12320 - Name: Neoplasms, Glandular and Epithelial - Relevance: LOW - As Found: Unknown - ID: M12325 - Name: Neoplasms, Nerve Tissue - Relevance: LOW - As Found: Unknown - ID: T4085 - Name: Neuroblastoma - Relevance: HIGH - As Found: Neuroblastoma - ID: T4092 - Name: Neuroepithelioma - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000009447 - Term: Neuroblastoma ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Guided Therapy **Description:** A total of 14 eligible neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles). **ID:** EG000 **Other Num Affected:** 12 **Other Num at Risk:** 14 **Serious Number At Risk:** 14 **Title:** Guided Therapy **Frequency Threshold:** 0 #### Other Events **Term:** Abdominal Pain **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (3.0) **Term:** ALT elevation **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** CTCAE (3.0) **Term:** AST Elevation **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Anemia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Elevated Bilirubin **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Constipation **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Dehydration **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Fatigue **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Fever **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** CTCAE (3.0) **Term:** Hypoalbunemia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Hypocalcemia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Hypophosphatemia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Infection **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** CTCAE (3.0) **Term:** Leukopenia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Lymphocytopenia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Mucositis **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Myalgia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Nausea **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Neutropenia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Pain **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Rash **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Tachycardia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Thrombocytopenia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Vomiting **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (3.0) **Term:** Weight Loss **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE (3.0) **Time Frame:** Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year. ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 16 **Units:** Participants ### Group **ID:** BG000 **Title:** Guided Therapy **Description:** All subjects receive guided therapy in therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. ### Measure #### Measurement **Group ID:** BG000 **Value:** 15 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 1 **Category Title:** Between 18 and 65 years #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** >=65 years **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 7 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 9 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** American Indian or Alaska Native #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Native Hawaiian or Other Pacific Islander #### Measurement **Group ID:** BG000 **Value:** 1 **Category Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 11 **Category Title:** White #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** More than one race #### Measurement **Group ID:** BG000 **Value:** 4 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 16 **Class Title:** United States **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Age, Categorical **Unit of Measure:** Participants ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Race (NIH/OMB) **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** participants **Population Description:** Multiply relapsed or refractory neuroblastoma. ## Results Section - More Information Module ### Certain Agreement ### Point of Contact **Email:** [email protected] **Organization:** NMTRC at Spectrum Health **Phone:** 616-267-0334 **Title:** Giselle Sholler, MD ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### Outcome Measure 4 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 100 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 12 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 7 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 43 - **Spread:** - **Upper Limit:** 59 - **Value:** 59 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Feasibility parameter defined as: Enrollment onto study, quality mRNA obtained, gene chip completed, tumor board held, medical monitor review and approval, start of treatment by 21 days post biopsy/surgical resection date, and then completion of 1 cycle of therapy." **Parameter Type:** NUMBER **Population Description:** All subjects had soft tissue disease in which biopsy was possible. Two subjects were deemed ineligible due to benign tumor type after biopsy. **Reporting Status:** POSTED **Time Frame:** 1 year **Title:** Percentage of Participants That Are Able to Meet Feasibility Parameters. **Type:** PRIMARY **Unit of Measure:** percentage of participants ##### Group **Description:** All subjects receive guided therapy in therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. **ID:** OG000 **Title:** Guided Therapy #### Outcome Measure 2 **Description:** To determine the safety of allowing a molecular tumor board to determine individualized treatment plans **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** 1 year **Title:** Number of Participants With Adverse Events as a Measure of Safety **Type:** SECONDARY **Unit of Measure:** participants ##### Group **Description:** A total of 14 eligible neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles). **ID:** OG000 **Title:** Guided Therapy #### Outcome Measure 3 **Description:** Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** 1 year **Title:** Overall Response Rate (ORR) of Participants Using RECIST Criteria **Type:** SECONDARY **Unit of Measure:** percentage of participants with PR or CR ##### Group **Description:** A total of 14 eligible neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles). **ID:** OG000 **Title:** Guided Therapy #### Outcome Measure 4 **Description:** Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 1 year **Title:** Activity of Treatments Chosen Based on Progression Free Survival (PFS) **Type:** SECONDARY **Unit of Measure:** Days ##### Group **Description:** A total of 14 eligible neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles). **ID:** OG000 **Title:** Guided Therapy ### Participant Flow Module #### Group **Description:** A total of 14 neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles). Guided Therapy: A total of 14 neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for disease response, progression and safety. All patients will be **ID:** FG000 **Title:** Guided Therapy #### Period **Title:** Overall Study ##### Withdraw **Type:** benign tumor types found at biopsy ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 16 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 14 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 2 **Recruitment Details:** This was an open label, multi-center prospective feasibility study in patients with refractory or recurrent neuroblastoma that enrolled at NMTRC centers across the country between 8/11/2011 and 11/26/2012. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT00652379 **Brief Title:** Co-treatment With Pegvisomant and a Somatostatin Analogue (SA) in SA-responsive Acromegalic Patients **Official Title:** Co-treatment With Pegvisomant and a Somatostatin Analogue (SA) in SA-responsive Acromegalic Patients: Impact on Insulin Sensitivity, Glucose Tolerance, and Pharmacoeconomics #### Organization Study ID Info **ID:** GH-2007-228 #### Organization **Class:** OTHER **Full Name:** University of Aarhus #### Secondary ID Infos **ID:** 2007-005244-25 **Type:** EUDRACT_NUMBER ### Status Module #### Completion Date **Date:** 2011-05 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2012-01-26 **Type:** ESTIMATED **Last Update Submit Date:** 2012-01-25 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2009-11 **Type:** ACTUAL #### Start Date **Date:** 2008-06 **Status Verified Date:** 2012-01 #### Study First Post Date **Date:** 2008-04-03 **Type:** ESTIMATED **Study First Submit Date:** 2008-03-26 **Study First Submit QC Date:** 2008-03-31 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Aarhus University Hospital Skejby **Class:** OTHER **Name:** Aarhus University Hospital **Class:** OTHER **Name:** The Research Council for Health and Disease, Denmark #### Lead Sponsor **Class:** OTHER **Name:** University of Aarhus #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this study is to investigate if co-treatment of acromegalic patients, who beforehand are considered well-controlled on SA monotherapy, with pegvisomant and SA will improve insulin sensitivity and glucose tolerance, and if these effects of co-treatment can be obtained at a neutral cost as compared to SA mono therapy. Second to investigate body composition, substrate metabolism, symptoms, intrahepatic and intramyocellular fat. ### Conditions Module **Conditions:** - Acromegaly - Insulin Resistance - Impaired Glucose Tolerance **Keywords:** - Acromegaly - Insulin sensitivity - Glucose tolerance - Body composition - Growth Hormone ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 18 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Co-treatment with Pegvisomant (15-30 mg twice a week) and a 50 percent reduced somatostatin-analog dose **Intervention Names:** - Drug: Pegvisomant - Drug: Somatostatin analog (lanreotide or octreotide) **Label:** 1 **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Somatostatin analog, unaltered dosage **Intervention Names:** - Drug: Somatostatin analog (lanreotide or octreotide) **Label:** 2 **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - 1 **Description:** Pegvisomant s.c 15-30 mg 2 times a week **Name:** Pegvisomant **Other Names:** - Somavert **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - 1 - 2 **Description:** Study arm 2: usual dosage of a somatostatin analog Study arm 1: half dosage of somatostatin analog **Name:** Somatostatin analog (lanreotide or octreotide) **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Insulin sensitivity **Time Frame:** 0 and after 24 weeks #### Secondary Outcomes **Measure:** Glucose tolerance **Time Frame:** 0 and after 24 weeks **Measure:** Symptoms, QoL questionaire **Time Frame:** 0, 12 and 24 weeks **Measure:** Intrahepatic and intramyocellular fat **Time Frame:** 0 and 24 weeks **Measure:** Substrate metabolism **Time Frame:** 0 and 24 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Age \> 18 * Diagnosed with acromegaly * Safe anticonceptive for fertile women * Well controlled on somatostatin analog (a serum IGF-I within normal range a nadir GH \< 0.5 µg/l.) Exclusion Criteria: * Pregnancy * Liver disease * Diabetes mellitus type I * Magnetic or electronic implants **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Aarhus C **Country:** Denmark **Facility:** Department of Endocrinology, Aarhus University Hospital **State:** Aarhus **Zip:** 8000 #### Overall Officials **Official 1:** **Affiliation:** Aarhus University Hospital **Name:** Jens Otto L. Jørgensen, MD Professor **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000006946 - Term: Hyperinsulinism - ID: D000044882 - Term: Glucose Metabolism Disorders - ID: D000008659 - Term: Metabolic Diseases - ID: D000001849 - Term: Bone Diseases, Endocrine - ID: D000001847 - Term: Bone Diseases - ID: D000009140 - Term: Musculoskeletal Diseases - ID: D000006964 - Term: Hyperpituitarism - ID: D000010900 - Term: Pituitary Diseases - ID: D000007027 - Term: Hypothalamic Diseases - ID: D000001927 - Term: Brain Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000004700 - Term: Endocrine System Diseases - ID: D000006943 - Term: Hyperglycemia ### Condition Browse Module - Browse Branches - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC20 - Name: Immune System Diseases - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC19 - Name: Gland and Hormone Related Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M10370 - Name: Insulin Resistance - Relevance: HIGH - As Found: Insulin Resistance - ID: M10018 - Name: Hypersensitivity - Relevance: LOW - As Found: Unknown - ID: M20295 - Name: Glucose Intolerance - Relevance: HIGH - As Found: Impaired Glucose Tolerance - ID: M3531 - Name: Acromegaly - Relevance: HIGH - As Found: Acromegaly - ID: M9997 - Name: Hyperinsulinism - Relevance: LOW - As Found: Unknown - ID: M11639 - Name: Metabolic Diseases - Relevance: LOW - As Found: Unknown - ID: M25403 - Name: Glucose Metabolism Disorders - Relevance: LOW - As Found: Unknown - ID: M5126 - Name: Bone Diseases - Relevance: LOW - As Found: Unknown - ID: M5128 - Name: Bone Diseases, Endocrine - Relevance: LOW - As Found: Unknown - ID: M12097 - Name: Musculoskeletal Diseases - Relevance: LOW - As Found: Unknown - ID: M13791 - Name: Pituitary Diseases - Relevance: LOW - As Found: Unknown - ID: M10077 - Name: Hypothalamic Diseases - Relevance: LOW - As Found: Unknown - ID: M5204 - Name: Brain Diseases - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M7862 - Name: Endocrine System Diseases - Relevance: LOW - As Found: Unknown - ID: M9994 - Name: Hyperglycemia - Relevance: LOW - As Found: Unknown - ID: T139 - Name: Acromegaly - Relevance: HIGH - As Found: Acromegaly ### Condition Browse Module - Meshes - ID: D000000172 - Term: Acromegaly - ID: D000007333 - Term: Insulin Resistance - ID: D000018149 - Term: Glucose Intolerance ### Intervention Browse Module - Ancestors - ID: D000005765 - Term: Gastrointestinal Agents - ID: D000018931 - Term: Antineoplastic Agents, Hormonal - ID: D000000970 - Term: Antineoplastic Agents - ID: D000006728 - Term: Hormones - ID: D000006730 - Term: Hormones, Hormone Substitutes, and Hormone Antagonists - ID: D000045505 - Term: Physiological Effects of Drugs ### Intervention Browse Module - Browse Branches - Abbrev: Hypo - Name: Hypoglycemic Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: Gast - Name: Gastrointestinal Agents ### Intervention Browse Module - Browse Leaves - ID: M10365 - Name: Insulin - Relevance: LOW - As Found: Unknown - ID: M9789 - Name: Hormones - Relevance: LOW - As Found: Unknown - ID: M173166 - Name: Insulin, Globin Zinc - Relevance: LOW - As Found: Unknown - ID: M15806 - Name: Somatostatin - Relevance: HIGH - As Found: Consequences of - ID: M17982 - Name: Octreotide - Relevance: HIGH - As Found: Neuropathy - ID: M146123 - Name: Lanreotide - Relevance: HIGH - As Found: Search - ID: M8881 - Name: Gastrointestinal Agents - Relevance: LOW - As Found: Unknown - ID: M20966 - Name: Antineoplastic Agents, Hormonal - Relevance: LOW - As Found: Unknown - ID: M9788 - Name: Hormone Antagonists - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000015282 - Term: Octreotide - ID: C000060347 - Term: Lanreotide - ID: D000013004 - Term: Somatostatin ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03743779 **Brief Title:** Mastering Diabetes Pilot Study **Official Title:** Mastering Diabetes Pilot Study #### Organization Study ID Info **ID:** 18-X-272 #### Organization **Class:** OTHER **Full Name:** Ohio University ### Status Module #### Completion Date **Date:** 2019-07-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2019-11-15 **Type:** ACTUAL **Last Update Submit Date:** 2019-11-13 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2018-12-01 **Type:** ACTUAL #### Start Date **Date:** 2018-09-09 **Type:** ACTUAL **Status Verified Date:** 2019-11 #### Study First Post Date **Date:** 2018-11-16 **Type:** ACTUAL **Study First Submit Date:** 2018-11-08 **Study First Submit QC Date:** 2018-11-14 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Ohio University #### Responsible Party **Investigator Affiliation:** Ohio University **Investigator Full Name:** David Drozek **Investigator Title:** Associate Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** This is a pilot study looking at past results of the Mastering Diabetes program utilizing a survey. **Detailed Description:** Adult past and present participants of Mastering Diabetes will be sent an email asking for their participation in the survey. The email will contain a hot link to a REDCap survey which will begin with a description of the study followed by a consent form. Once the consent is agreed, the survey will ask for participants' demographics and information about the variables described below No personal identifiable information is being collected. The data from this study will help develop a prospective study to better evaluate the effectiveness of Mastering Diabetes in controlling diabetes. Future comparative studies between programs are anticipated, which will help identify the strengths and weaknesses of different programs, as well as identify subpopulations that might benefit from one program as opposed to another. Data will be shared via posters and papers among the lifestyle medicine community to help contribute to better lifestyle interventions. ### Conditions Module **Conditions:** - Diabetes Mellitus **Keywords:** - diabetes - lifestyle - plant-based, whole food - vegetarian - vegan - exercise ### Design Module #### Design Info **Observational Model:** OTHER **Time Perspective:** RETROSPECTIVE #### Enrollment Info **Count:** 253 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants enrolled in Mastering Diabetes. **Intervention Names:** - Behavioral: Mastering Diabetes **Label:** Intervention ### Interventions #### Intervention 1 **Arm Group Labels:** - Intervention **Description:** The Mastering Diabetes provides a step-by-step program to transition to a low-fat, plant-based, whole-food lifestyle and reduce overwhelm. The program also provides direct access to expert coaches in the online community, which contains others living with all forms of diabetes. I includes access to live Q\&A videoconferences twice per month to interact with coaches and personalize the experience. The program is designed with seven core modules. Many participants continue in the program after completion of the modules for ongoing support. The cost was $29/month or $249/year, but as of July 2018, only the $249/year option is offered. **Name:** Mastering Diabetes **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** change in glycosylated hemoglobin during program **Measure:** glycosylated hemoglobin **Time Frame:** 1 year **Description:** change in weight during the program **Measure:** weight **Time Frame:** 1 year #### Secondary Outcomes **Description:** Survey question is: Since completing Mastering Diabetes: 1. My health has continued to improve 2. My health has neither improved or declined 3. My health has declined 4. I don't know if my health has improved or declined **Measure:** perception of health change **Time Frame:** 1 year **Description:** Survey question is: I feel that Mastering Diabetes was: 1. Very helpful 2. Somewhat helpful 3. Neither helpful or unhelpful 4. Somewhat unhelpful 5. Very unhelpful **Measure:** perception of benefit **Time Frame:** 1 year **Description:** Survey question: Concerning the changes I made during participation in Mastering Diabetes: 1. I am still doing all of them 2. I am still doing most of them 3. I am still doing about half of them 4. I am still doing some of them 5. I am not doing any of them **Measure:** adherence **Time Frame:** 1 year ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Adult past and present participants of Mastering Diabetes Exclusion Criteria: * None **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** People with diabetes who enroll in the Mastering Diabetes program ### Contacts Locations Module #### Locations **Location 1:** **City:** Athens **Country:** United States **Facility:** Ohio University **State:** Ohio **Zip:** 45701 #### Overall Officials **Official 1:** **Affiliation:** Ohio University **Name:** David Drozek, DO **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ## Derived Section ### Condition Browse Module - Ancestors - ID: D000044882 - Term: Glucose Metabolism Disorders - ID: D000008659 - Term: Metabolic Diseases - ID: D000004700 - Term: Endocrine System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: BC19 - Name: Gland and Hormone Related Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M7115 - Name: Diabetes Mellitus - Relevance: HIGH - As Found: Diabetes - ID: M11639 - Name: Metabolic Diseases - Relevance: LOW - As Found: Unknown - ID: M25403 - Name: Glucose Metabolism Disorders - Relevance: LOW - As Found: Unknown - ID: M7862 - Name: Endocrine System Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000003920 - Term: Diabetes Mellitus ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02334579 **Brief Title:** Stereotactic Body Radiotherapy for Stage I-III Prostate Cancer **Official Title:** Function-Preserving Stereotactic Body Radiotherapy for Clinical State I-III Prostate Cancer #### Organization Study ID Info **ID:** IR 5642 #### Organization **Class:** OTHER **Full Name:** Swedish Medical Center ### Status Module #### Completion Date **Date:** 2024-12 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2019-07-12 **Type:** ACTUAL **Last Update Submit Date:** 2019-07-11 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2023-12 **Type:** ESTIMATED #### Start Date **Date:** 2014-12 **Status Verified Date:** 2019-07 #### Study First Post Date **Date:** 2015-01-08 **Type:** ESTIMATED **Study First Submit Date:** 2014-12-04 **Study First Submit QC Date:** 2015-01-06 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Swedish Medical Center #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to find out the effects (good and bad) of highly focused radiation on you and your prostate cancer. The purpose of this evaluation is to see if this treatment causes fewer side effects that other standard treatment approaches, and to evaluate the effect of this treatment on your prostate tumor and your quality of life over time. ### Conditions Module **Conditions:** - Prostate Cancer - Prostatic Cancer - Prostate Neoplasms - Prostatic Neoplasms - Cancer of the Prostate **Keywords:** - Radiation - Stereotactic Radiosurgery - Radiotherapy - Prostate Tumor - Prostate Surgery - CyberKnife ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 146 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** This treatment concentrates large doses of radiation onto the tumor so that injury from radiation to the nearby normal tissue will be minimal. CyberKnife Stereotactic Radiosurgery is not investigational and is considered standard of care. **Intervention Names:** - Radiation: CyberKnife Stereotactic Radiosurgery **Label:** CyberKnife Stereotactic Radiosurgery **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - CyberKnife Stereotactic Radiosurgery **Description:** Five treatments given over about one week. **Name:** CyberKnife Stereotactic Radiosurgery **Type:** RADIATION ### Outcomes Module #### Other Outcomes **Description:** Measure quality of life after five years following cyberknife stereotactic radiosurgery. **Measure:** Quality of life **Time Frame:** 5 years #### Primary Outcomes **Description:** In the low- and intermediate-risk prostate cancer groups, to determine whether study treatment improves patient-reported quality of life outcomes compared to the approach used in a previous multi-center cyberknife stereotactic radiosurgery trial. **Measure:** Quality of life outcomes (low- and intermediate-risk prostate cancer groups) **Time Frame:** 8 years **Description:** In the high-risk group, to estimate rates of acute and late grade 3-5 gastrointestinal and genitourinary toxicities following cyberknife stereotactic radiosurgery. **Measure:** Rates of acute and late grade 3-5 gastrointestinal and genitourinary toxicities (high-risk group) **Time Frame:** 8 years #### Secondary Outcomes **Description:** After five years following cyberknife stereotactic radiosurgery, estimate rates of toxicities related to treatment. **Measure:** Rates of toxicities related to treatment **Time Frame:** 5 years **Description:** Measure disease free survival after five years following cyberknife stereotactic radiosurgery. **Measure:** Disease free survival **Time Frame:** 5 years **Description:** Measure overall survival after five years following cyberknife stereotactic radiosurgery. **Measure:** Overall survival **Time Frame:** 5 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Histologically proven prostate adenocarcinoma * Clinical stage T1a-T3, N0-Nx, M0-Mx * Patients belonging in one of the following risk groups: (1) LOW RISK: CS T1a-T2a, Gleason 2-6, PSA\<10, Nx-0, Mx-0; (2) INTERMEDIATE RISK: CS T2b, Gleason ≤7, PSA\<20, Nx-0, Mx-0, or CS T1a-T2b, Gleason 2-6, PSA≥10 \& \<20, Nx-0, Mx-0, or CS T1a-T2b, Gleason 7, PSA≤20, Nx-0, Mx-0; or (3) HIGH RISK: CS T2c-T3, any Gleason, any PSA, or CS T1-3, Gleason ≥8 and/or PSA≥20 * Karnofsky performance status 70-100 * Hormone therapy: includes LHRH agonists (e.g. leuprolide, goserelin, triptorelin), antagonists (e.g. degarelix), peripheral blockers (e.g. flutamide, bicalutamide, nilutamide), estrogens (e.g. DES) and bilateral orchiectomy * Low and Intermediate risk groups: no hormone ablation for two months prior to enrollment, or during treatment * High risk group: three hormone therapy regimens are allowed * 5-alpha reductase inhibitors (e.g., finasteride or dutasteride) are allowed Exclusion Criteria: * Prior prostatectomy or cryotherapy of the prostate * Prior high-dose radiotherapy to the prostate or lower pelvis * Implanted hardware or other material that would prohibit appropriate treatment planning or treatment delivery, in the investigator's opinion **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Mary Monahan **Phone:** (206) 320-7129 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Robert Meier, MD **Phone:** (206) 320-7130 **Role:** CONTACT #### Locations **Location 1:** **City:** Seattle **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Mary Monahan - **Phone:** 206-320-7029 - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Robert Meier, MD - **Phone:** (206) 320-7130 - **Role:** CONTACT ***Contact 3:*** - **Name:** Robert Meier, MD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Swedish Medical Center Radiosurgery Center **State:** Washington **Status:** RECRUITING **Zip:** 98122 #### Overall Officials **Official 1:** **Affiliation:** Swedish Medical Center Radiosurgery Center **Name:** Robert Meier, MD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### See Also Links **Label:** Swedish Medical Center Radiosurgery Center **URL:** http://www.swedish.org/services/radiosurgery-center ## Derived Section ### Condition Browse Module - Ancestors - ID: D000005834 - Term: Genital Neoplasms, Male - ID: D000014565 - Term: Urogenital Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000005832 - Term: Genital Diseases, Male - ID: D000091662 - Term: Genital Diseases - ID: D000091642 - Term: Urogenital Diseases - ID: D000011469 - Term: Prostatic Diseases - ID: D000052801 - Term: Male Urogenital Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M14335 - Name: Prostatic Neoplasms - Relevance: HIGH - As Found: Prostate Cancer - ID: M8946 - Name: Genital Neoplasms, Male - Relevance: LOW - As Found: Unknown - ID: M17315 - Name: Urogenital Neoplasms - Relevance: LOW - As Found: Unknown - ID: M2876 - Name: Genital Diseases - Relevance: LOW - As Found: Unknown - ID: M8944 - Name: Genital Diseases, Male - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M14333 - Name: Prostatic Diseases - Relevance: LOW - As Found: Unknown - ID: M27095 - Name: Male Urogenital Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000011471 - Term: Prostatic Neoplasms - ID: D000009369 - Term: Neoplasms ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02166879 **Brief Title:** Undetected Sleep Apnea in the Postanesthesia Acute Care Unit (PACU) **Official Title:** Undetected Sleep Apnea in the Postanesthesia Acute Care Unit #### Organization Study ID Info **ID:** PA11-0591 #### Organization **Class:** OTHER **Full Name:** M.D. Anderson Cancer Center ### Status Module #### Completion Date **Date:** 2020-08 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2020-01-14 **Type:** ACTUAL **Last Update Submit Date:** 2020-01-13 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2020-08 **Type:** ESTIMATED #### Start Date **Date:** 2011-09-02 **Type:** ACTUAL **Status Verified Date:** 2020-01 #### Study First Post Date **Date:** 2014-06-18 **Type:** ESTIMATED **Study First Submit Date:** 2014-06-13 **Study First Submit QC Date:** 2014-06-16 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** M.D. Anderson Cancer Center #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The primary aim is to determine whether patients with suspected OSAHS as predicted by the STOP-BANG questionnaire will have an increased length of stay (LOS) in the postanesthesia acute care unit (PACU) compared with those without suspected OSAHS. The second aim will be to determine the LOS in patients with known sleep apnea by history. This length of stay will be compared with LOS in patient with an affirmative response to the STOP-BANG questionnaire to determine if prior knowledge of diagnosed sleep apnea will be associated with a lower LOS than in patients with suspected OSA. The third aim will be to characterize the adverse clinical outcomes (respiratory, cardiovascular, and neurological) associated with suspected OSAHS in patients who respond affirmatively to the STOP-BANG questionnaire and in those patients with known OSA. These data (including number of desaturations, bradypnea, brady- or tachycardia, and use of reversal agents) will be recorded by the PACU nursing staff. Unexpected admissions to the hospital and transfers to the intensive care units will also be measured. These data will help identify the most critical determinants of length of stay. **Detailed Description:** The study will retrospectively review the anesthetic record, including both the preoperative assessment records and the PACU flow sheets. The study will be conducted in the Anesthesiology Assessment Center. PACU. The STOP-BANG questionnaire is administered as part of the standard of care during the preoperative assessment. Anesthesia Assessment Center clinics assess approximately 500 patients a week, of which approximately 25% are scheduled for ambulatory surgical procedures in the Mays clinic. Patients are administered the STOP-BANG questionnaire as part of the standard of care while in the perioperative center. This will be recorded on the Initial Anesthesia Assessment Center Patient Record. This form is filled out by hand by the patients themselves and then scanned into Clinic Station. The neck circumference portion of the Bang questionnaire is assessed and recorded by the medical assistant with the use of a single use disposable paper tape measure. The data are recorded along with basic demographic, historical, and anthropometric data (including BMI) in a preoperative assessment record. In order to satisfy the first specific aim, length of stay in minutes as determined by the PACU nursing record will be documented. Nurses charting the LOS will not be specifically made aware of the STOP-BANG status of the subjects. Length of stay is charted in the PACU nursing forms electronically in PICIS and relayed to Clinic Station as part of the standard of care for all patients admitted to the PACU. In order to satisfy the third specific aim, patients enrolled in the study will be monitored for secondary outcomes via chart review of the PACU nursing record and electronic medical record. These outcomes are charted in the PACU nursing forms electronically in PCIS and relayed to Clinic Station as standard of care in all patients in the PACU. ### Conditions Module **Conditions:** - Sleep Apnea, Obstructive **Keywords:** - Obstructive Sleep Apnea-Hypopnea Syndrome - Obstructive sleep apnea - OSA - Chart review - Questionnaire - Survey ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** RETROSPECTIVE #### Enrollment Info **Count:** 900 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Chart review from patients undergoing elective surgery. **Intervention Names:** - Behavioral: Questionnaire **Label:** Obstructive Sleep Apnea Hypopnea Syndrome (OSAHS) ### Interventions #### Intervention 1 **Arm Group Labels:** - Obstructive Sleep Apnea Hypopnea Syndrome (OSAHS) **Description:** Participants complete anesthesia questionnaire before elective surgical procedure. **Name:** Questionnaire **Other Names:** - Survey **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** Length of stay in minutes as determined by the PACU nursing record documented. A difference in LOS of 15 minutes is considered to be clinically important. A 2-sample t-test used to compare OSAHS risk groups with respect to LOS. **Measure:** Length of Stay (LOS) in the Postanesthesia Acute Care Unit (PACU) **Time Frame:** 1 day ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Patients assessed in the Anesthesia Assessment Center who are determined to require same day surgical procedures in the Ambulatory Care Center in the Mays Clinic. 2. Age ≥18 Exclusion Criteria: 1) Pregnancy **Healthy Volunteers:** True **Minimum Age:** 18 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Participants undergoing elective surgical procedures at UT MD Anderson Cancer Center in Houston, Texas ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Dave Balachandran, MD **Phone:** 713-792-6238 **Role:** CONTACT #### Locations **Location 1:** **City:** Houston **Country:** United States **Facility:** University of Texas MD Anderson Cancer Center **State:** Texas **Status:** RECRUITING **Zip:** 77030 #### Overall Officials **Official 1:** **Affiliation:** M.D. Anderson Cancer Center **Name:** Dave Balachandran, MD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### See Also Links **Label:** University of Texas MD Anderson Cancer Center Website **URL:** http://www.mdanderson.org ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012120 - Term: Respiration Disorders - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000012818 - Term: Signs and Symptoms, Respiratory - ID: D000020919 - Term: Sleep Disorders, Intrinsic - ID: D000020920 - Term: Dyssomnias - ID: D000012893 - Term: Sleep Wake Disorders - ID: D000009422 - Term: Nervous System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M4361 - Name: Apnea - Relevance: HIGH - As Found: Apnea - ID: M15694 - Name: Sleep Apnea Syndromes - Relevance: HIGH - As Found: Sleep Apnea - ID: M22010 - Name: Sleep Apnea, Obstructive - Relevance: HIGH - As Found: Sleep Apnea, Obstructive - ID: M14957 - Name: Respiration Disorders - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown - ID: M15623 - Name: Signs and Symptoms, Respiratory - Relevance: LOW - As Found: Unknown - ID: M22242 - Name: Parasomnias - Relevance: LOW - As Found: Unknown - ID: M22654 - Name: Sleep Disorders, Intrinsic - Relevance: LOW - As Found: Unknown - ID: M22655 - Name: Dyssomnias - Relevance: LOW - As Found: Unknown - ID: M15696 - Name: Sleep Wake Disorders - Relevance: LOW - As Found: Unknown - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001049 - Term: Apnea - ID: D000012891 - Term: Sleep Apnea Syndromes - ID: D000020181 - Term: Sleep Apnea, Obstructive ### Intervention Browse Module - Browse Branches - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M4107 - Name: Anesthetics - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04138979 **Brief Title:** Intestinal Microbiota of Breast Cancer Patients Undergoing Chemotherapy **Official Title:** Intestinal Microbiota of Breast Cancer Patients Undergoing Chemotherapy #### Organization Study ID Info **ID:** Yunwei Wei 2019-09-13 #### Organization **Class:** OTHER **Full Name:** First Affiliated Hospital of Harbin Medical University ### Status Module #### Completion Date **Date:** 2020-10-15 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2019-10-25 **Type:** ACTUAL **Last Update Submit Date:** 2019-10-23 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2020-10-15 **Type:** ESTIMATED #### Start Date **Date:** 2019-09-12 **Type:** ACTUAL **Status Verified Date:** 2019-09 #### Study First Post Date **Date:** 2019-10-25 **Type:** ACTUAL **Study First Submit Date:** 2019-10-23 **Study First Submit QC Date:** 2019-10-23 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** First Affiliated Hospital of Harbin Medical University #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Is US Export:** False ### Description Module **Brief Summary:** To date, few studies have addressed the link between gut microbiota and breast cancer chemotherapy, and previous studies have only provided a link between the gut and breast cancer. **Detailed Description:** To date, few studies have addressed the link between gut microbiota and breast cancer chemotherapy, and previous studies have only provided a link between the gut and breast cancer.At present, studies on the interaction mechanism between intestinal flora and breast cancer mainly focus on two aspects: first, intestinal flora affects the occurrence and progress of breast cancer by affecting estrogen metabolism;Second, intestinal flora can influence the occurrence and treatment of breast cancer by affecting the regulation of the immune system.However, the specific types (or groups) of bacteria that promote the occurrence, development and mechanism of breast cancer still need to be further studied.In addition, since most of the current clinical studies on microorganisms and breast cancer are small sample studies, the accuracy of their conclusions remains to be studied. ### Conditions Module **Conditions:** - Microbiota **Keywords:** - gut microbiota - 16S rRNA gene - breast cancer chemotherapy ### Design Module #### Design Info **Observational Model:** CASE_CONTROL **Time Perspective:** CROSS_SECTIONAL #### Enrollment Info **Count:** 80 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 20 healthy volunteers were included in the healthy control group **Label:** Control group #### Arm Group 2 **Description:** First chemotherapy for breast cancer **Intervention Names:** - Drug: cyclophosphamide **Label:** Disease group ### Interventions #### Intervention 1 **Arm Group Labels:** - Disease group **Description:** First use of chemotherapy after breast cancer **Name:** cyclophosphamide **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** The microbiota measured by 16S rRNA gene **Measure:** Transcriptional changes in gut microbiota **Time Frame:** baseline,1 day,7 day,14 days,22 days,29 days,36 days,44days,51days,58days,66days,73days,80days ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Aged 18 to 65 years * First use of chemotherapy after breast cancer Exclusion Criteria: * Pregnancy * Lactation * Cigarette smoking * Alcohol addiction * Hypertension * Diabetes mellitus * Lipid dysregulation * BMI \> 27 * Recent (\< 3 months prior) use of antibiotics, probiotics, prebiotics, symbiotics, hormonal medication, laxatives, proton pump inhibitors, insulin sensitizers or Chinese herbal medicine * History of disease with an autoimmune component, such as MS, rheumatoid arthritis, IBS, or IBD * History of malignancy or any gastrointestinal tract surgery **Healthy Volunteers:** True **Maximum Age:** 65 Years **Minimum Age:** 18 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** All patients were included according to the inclusion and exclusion criteria ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Yunwei Wei, doctor **Phone:** +86-0451-85553099 **Role:** CONTACT #### Locations **Location 1:** **City:** Harbin **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Yunwei Wei - **Phone:** +86-0451-85553099 - **Role:** CONTACT **Country:** China **Facility:** First affiliated hospital of Harbin medical university **State:** Heilongjiang **Status:** RECRUITING **Zip:** 150001 ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Luu TH, Michel C, Bard JM, Dravet F, Nazih H, Bobin-Dubigeon C. Intestinal Proportion of Blautia sp. is Associated with Clinical Stage and Histoprognostic Grade in Patients with Early-Stage Breast Cancer. Nutr Cancer. 2017 Feb-Mar;69(2):267-275. doi: 10.1080/01635581.2017.1263750. Epub 2017 Jan 17. **PMID:** 28094541 **Citation:** Qin J, Li R, Raes J, Arumugam M, Burgdorf KS, Manichanh C, Nielsen T, Pons N, Levenez F, Yamada T, Mende DR, Li J, Xu J, Li S, Li D, Cao J, Wang B, Liang H, Zheng H, Xie Y, Tap J, Lepage P, Bertalan M, Batto JM, Hansen T, Le Paslier D, Linneberg A, Nielsen HB, Pelletier E, Renault P, Sicheritz-Ponten T, Turner K, Zhu H, Yu C, Li S, Jian M, Zhou Y, Li Y, Zhang X, Li S, Qin N, Yang H, Wang J, Brunak S, Dore J, Guarner F, Kristiansen K, Pedersen O, Parkhill J, Weissenbach J; MetaHIT Consortium; Bork P, Ehrlich SD, Wang J. A human gut microbial gene catalogue established by metagenomic sequencing. Nature. 2010 Mar 4;464(7285):59-65. doi: 10.1038/nature08821. **PMID:** 20203603 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000001941 - Term: Breast Diseases - ID: D000012871 - Term: Skin Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M5220 - Name: Breast Neoplasms - Relevance: HIGH - As Found: Breast Cancer - ID: M5218 - Name: Breast Diseases - Relevance: LOW - As Found: Unknown - ID: M15674 - Name: Skin Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001943 - Term: Breast Neoplasms ### Intervention Browse Module - Ancestors - ID: D000007166 - Term: Immunosuppressive Agents - ID: D000007155 - Term: Immunologic Factors - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018501 - Term: Antirheumatic Agents - ID: D000018906 - Term: Antineoplastic Agents, Alkylating - ID: D000000477 - Term: Alkylating Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000000970 - Term: Antineoplastic Agents - ID: D000019653 - Term: Myeloablative Agonists ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: ARhu - Name: Antirheumatic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M6727 - Name: Cyclophosphamide - Relevance: HIGH - As Found: Cycle - ID: M10212 - Name: Immunosuppressive Agents - Relevance: LOW - As Found: Unknown - ID: M10201 - Name: Immunologic Factors - Relevance: LOW - As Found: Unknown - ID: M20604 - Name: Antirheumatic Agents - Relevance: LOW - As Found: Unknown - ID: M20942 - Name: Antineoplastic Agents, Alkylating - Relevance: LOW - As Found: Unknown - ID: M3820 - Name: Alkylating Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000003520 - Term: Cyclophosphamide ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03460379 **Brief Title:** Bariatric Surgery and Pharmacokinetics of Escitalopram **Official Title:** Bariatric Surgery and Pharmacokinetics Escitalopram: BAR-MEDS Escitalopram #### Organization Study ID Info **ID:** 2016/1145k #### Organization **Class:** OTHER **Full Name:** Norwegian University of Science and Technology ### Status Module #### Completion Date **Date:** 2026-10 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-03-27 **Type:** ACTUAL **Last Update Submit Date:** 2023-03-24 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2026-10 **Type:** ESTIMATED #### Start Date **Date:** 2016-11-02 **Type:** ACTUAL **Status Verified Date:** 2023-03 #### Study First Post Date **Date:** 2018-03-09 **Type:** ACTUAL **Study First Submit Date:** 2018-03-04 **Study First Submit QC Date:** 2018-03-04 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** St. Olavs Hospital **Class:** OTHER **Name:** Volvat Medisinsk Senter Stokkan **Class:** OTHER **Name:** Namsos Hospital **Class:** OTHER **Name:** Alesund Hospital #### Lead Sponsor **Class:** OTHER **Name:** Norwegian University of Science and Technology #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Changes to gastric pH, gastric emptying time, gastrointestinal transit-time or the pre-systemic metabolizing effect of enzymes secreted in the mucosa may all alter the pharmacokinetics of medicines. These factors are potentially influenced by bariatric surgery. Little is so far known about how gastric bypass and sleeve gastrectomy impacts the biological availability of medication. In this study the pharmacokinetic effects of bariatric surgery on escitalopram are investigated. ### Conditions Module **Conditions:** - Obesity, Morbid **Keywords:** - Bariatric surgery - Gastrointestinal tract - Biological availability - Pharmacokinetics - Citalopram ### Design Module #### Bio Spec **Description:** Blood **Retention:** SAMPLES_WITH_DNA #### Design Info **Observational Model:** CASE_ONLY **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 12 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Description:** Patients are tested for their normal prescription escitalopram medication **Name:** Escitalopram **Other Names:** - Citalopram - Cytalopram **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Escitalopram concentration in blood serum (area under curve (AUC)) **Time Frame:** From baseline to 1 year postoperatively ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Preparing to undergo gastric bypass or sleeve gastrectomy in Central Norway * Being a Norwegian citizen Exclusion Criteria: * Having previously undergone resections in the GI-tract **Maximum Age:** 70 Years **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Patients referred for bariatric surgery (gastric bypass or sleeve gastrectomy) ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Magnus Strømmen, MSc **Phone:** 0047 72829970 **Role:** CONTACT #### Locations **Location 1:** **City:** Trondheim **Contacts:** ***Contact 1:*** - **Name:** Magnus Strømmen, MSc - **Role:** CONTACT **Country:** Norway **Facility:** St. Olavs University Hospital **Status:** RECRUITING #### Overall Officials **Official 1:** **Affiliation:** St. Olavs University Hospital **Name:** Magnus Strømmen, MSc **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009765 - Term: Obesity - ID: D000050177 - Term: Overweight - ID: D000044343 - Term: Overnutrition - ID: D000009748 - Term: Nutrition Disorders - ID: D000001835 - Term: Body Weight ### Condition Browse Module - Browse Branches - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M12701 - Name: Obesity - Relevance: LOW - As Found: Unknown - ID: M12702 - Name: Obesity, Morbid - Relevance: HIGH - As Found: Obesity, Morbid - ID: M26186 - Name: Overweight - Relevance: LOW - As Found: Unknown - ID: M25307 - Name: Overnutrition - Relevance: LOW - As Found: Unknown - ID: M12684 - Name: Nutrition Disorders - Relevance: LOW - As Found: Unknown - ID: M5114 - Name: Body Weight - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000009767 - Term: Obesity, Morbid ### Intervention Browse Module - Ancestors - ID: D000017367 - Term: Selective Serotonin Reuptake Inhibitors - ID: D000014179 - Term: Neurotransmitter Uptake Inhibitors - ID: D000049990 - Term: Membrane Transport Modulators - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000018377 - Term: Neurotransmitter Agents - ID: D000018490 - Term: Serotonin Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018687 - Term: Antidepressive Agents, Second-Generation - ID: D000000928 - Term: Antidepressive Agents - ID: D000011619 - Term: Psychotropic Drugs ### Intervention Browse Module - Browse Branches - Abbrev: PsychDr - Name: Psychotropic Drugs - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: AnDyAg - Name: Anti-Dyskinesia Agents ### Intervention Browse Module - Browse Leaves - ID: M17983 - Name: Citalopram - Relevance: HIGH - As Found: Valsartan - ID: M2732 - Name: Escitalopram - Relevance: HIGH - As Found: Enrollment - ID: M7104 - Name: Dexetimide - Relevance: LOW - As Found: Unknown - ID: M19649 - Name: Selective Serotonin Reuptake Inhibitors - Relevance: LOW - As Found: Unknown - ID: M15512 - Name: Serotonin - Relevance: LOW - As Found: Unknown - ID: M20504 - Name: Neurotransmitter Agents - Relevance: LOW - As Found: Unknown - ID: M4247 - Name: Antidepressive Agents - Relevance: LOW - As Found: Unknown - ID: M14474 - Name: Psychotropic Drugs - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000015283 - Term: Citalopram - ID: D000089983 - Term: Escitalopram ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00372879 **Brief Title:** Clinical Trial of Vitamin E to Treat Muscular Cramps in Patients With ALS **Official Title:** Randomized Crossover Design Trial of Vitamin E vs Placebo for Treatment of Cramps in Amyotrophic Lateral Sclerosis. #### Organization Study ID Info **ID:** R-06-451 #### Organization **Class:** OTHER **Full Name:** Lawson Health Research Institute #### Secondary ID Infos **ID:** ALSClin-001 ### Status Module #### Completion Date **Date:** 2010-08 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2016-03-03 **Type:** ESTIMATED **Last Update Submit Date:** 2016-03-01 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2010-08 **Type:** ACTUAL #### Start Date **Date:** 2006-12 **Status Verified Date:** 2016-03 #### Study First Post Date **Date:** 2006-09-07 **Type:** ESTIMATED **Study First Submit Date:** 2006-09-05 **Study First Submit QC Date:** 2006-09-05 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Lawson Health Research Institute #### Responsible Party **Investigator Affiliation:** Lawson Health Research Institute **Investigator Full Name:** Michael Strong **Investigator Title:** Professor Department of Clinical Neurological Sciences **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Muscular cramps are a common and uncomfortable symptom of amyotrophic lateral sclerosis (ALS). This clinical trial will compare the response of high dose vitamin E supplementation to placebo for treatment of muscular cramps in patients with ALS. We hypothesize that vitamin E will be more effective than placebo in treating cramps. **Detailed Description:** This will be a single centre randomized placebo controlled crossover design trial. Participants will be randomized at study entry to protocol A (vitamin E first) or protocol B (placebo first). The first 2 weeks of the study will be a baseline assessment of the frequency, severity and duration of cramps. Patients will be blinded for the remainder of the duration of the study. For weeks 3-6 of the study, group A will receive vitamin E 800 IU bid and group B will receive placebo. For weeks 7-10 of the study, group A will receive placebo and group B will receive vitamin E. Participants will record cramp frequency and characteristics via a daily journal for the duration of the study, however the data analysis will focus on cramp frequency only during weeks 5-6 and 9-10. Weeks 3-4 and 7-8 will be used as time periods to allow the new drug to come to steady state and allow for washout of the previous drug. Data analysis will focus on the difference in cramp frequency in individual patients in each treatment period. ### Conditions Module **Conditions:** - Amyotrophic Lateral Sclerosis **Keywords:** - amyotrophic lateral sclerosis - vitamin E - muscle cramp ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER ##### Masking Info **Masking:** QUADRUPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 32 **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Vitamin E first and placebo second **Intervention Names:** - Dietary Supplement: Vitamin E **Label:** Crossover group 1 **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Placebo first then vitamin E **Intervention Names:** - Dietary Supplement: Vitamin E **Label:** Crossover group 2 **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Crossover group 1 - Crossover group 2 **Description:** Vitamin E 800IU bid **Name:** Vitamin E **Type:** DIETARY_SUPPLEMENT ### Outcomes Module #### Primary Outcomes **Measure:** Reduction in number of muscle cramps experienced in a two week period. **Time Frame:** 4 weeks #### Secondary Outcomes **Measure:** Reduction in the duration of cramps and reduction in the severity of cramps **Time Frame:** 4 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Adults (\> age 18 years) * Probable or definite ALS by El Escorial Revised criteria * At least 2 painful muscle cramps in one or more of the limbs per week. * May have tried other medications for cramping in the past. * If participants are currently on treatment for cramps and are continuing to have at least 2 cramps per week, they can be included in the trial. In this situation, the individual's previous cramp medication can be continued during the trial. * Ideally, patients should not have any medication alterations during the duration of the trial. * Willing to discontinue supplementary vitamin E and multivitamins containing \> 400 IU of vitamin E during the trial. Exclusion Criteria: * Patients who are unable to safely consume the trial capsules. Individuals with significant dysphagia can be included into the study if they have a functioning PEG tube or GJ tube, through which the medication can be given. * Patients who are unable to fill out the daily diary, either personally or via a proxy. * Patients who have had medication changes within the last 4 weeks prior to the onset of the trial will be excluded. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** London **Country:** Canada **Facility:** London Health Sciences Centre **State:** Ontario **Zip:** N6A 5A5 #### Overall Officials **Official 1:** **Affiliation:** Clinical Neurological Sciences, London Health Sciences Centre **Name:** Michael J Strong, MD, FRCPC **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** Clinical Neurological Sciences, London Health Sciences Centre **Name:** Christen L Shoesmith, MD, FRCPC **Role:** STUDY_DIRECTOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010335 - Term: Pathologic Processes - ID: D000019636 - Term: Neurodegenerative Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000009468 - Term: Neuromuscular Diseases - ID: D000013118 - Term: Spinal Cord Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000057177 - Term: TDP-43 Proteinopathies - ID: D000057165 - Term: Proteostasis Deficiencies - ID: D000008659 - Term: Metabolic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M15415 - Name: Sclerosis - Relevance: HIGH - As Found: Sclerosis - ID: M18879 - Name: Motor Neuron Disease - Relevance: HIGH - As Found: Lateral Sclerosis - ID: M4024 - Name: Amyotrophic Lateral Sclerosis - Relevance: HIGH - As Found: Amyotrophic Lateral Sclerosis - ID: M15837 - Name: Spasm - Relevance: LOW - As Found: Unknown - ID: M12077 - Name: Muscle Cramp - Relevance: LOW - As Found: Unknown - ID: M21558 - Name: Neurodegenerative Diseases - Relevance: LOW - As Found: Unknown - ID: M12411 - Name: Neuromuscular Diseases - Relevance: LOW - As Found: Unknown - ID: M15915 - Name: Spinal Cord Diseases - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M28759 - Name: TDP-43 Proteinopathies - Relevance: LOW - As Found: Unknown - ID: M28747 - Name: Proteostasis Deficiencies - Relevance: LOW - As Found: Unknown - ID: M11639 - Name: Metabolic Diseases - Relevance: LOW - As Found: Unknown - ID: T349 - Name: Amyotrophic Lateral Sclerosis - Relevance: HIGH - As Found: Amyotrophic Lateral Sclerosis - ID: T4699 - Name: Primary Lateral Sclerosis - Relevance: HIGH - As Found: Lateral Sclerosis ### Condition Browse Module - Meshes - ID: D000016472 - Term: Motor Neuron Disease - ID: D000000690 - Term: Amyotrophic Lateral Sclerosis - ID: D000012598 - Term: Sclerosis ### Intervention Browse Module - Ancestors - ID: D000014815 - Term: Vitamins - ID: D000018977 - Term: Micronutrients - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000000975 - Term: Antioxidants - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000020011 - Term: Protective Agents ### Intervention Browse Module - Browse Branches - Abbrev: Micro - Name: Micronutrients - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Vi - Name: Vitamins ### Intervention Browse Module - Browse Leaves - ID: M22972 - Name: Tocopherols - Relevance: LOW - As Found: Unknown - ID: M17553 - Name: Vitamin E - Relevance: HIGH - As Found: Untreated - ID: M22975 - Name: Tocotrienols - Relevance: LOW - As Found: Unknown - ID: M22969 - Name: alpha-Tocopherol - Relevance: LOW - As Found: Unknown - ID: M17558 - Name: Vitamins - Relevance: LOW - As Found: Unknown - ID: M21009 - Name: Micronutrients - Relevance: LOW - As Found: Unknown - ID: M16885 - Name: Trace Elements - Relevance: LOW - As Found: Unknown - ID: M4292 - Name: Antioxidants - Relevance: LOW - As Found: Unknown - ID: M21869 - Name: Protective Agents - Relevance: LOW - As Found: Unknown - ID: T466 - Name: Tocopherol - Relevance: LOW - As Found: Unknown - ID: T467 - Name: Tocotrienol - Relevance: LOW - As Found: Unknown - ID: T480 - Name: Vitamin E - Relevance: HIGH - As Found: Untreated ### Intervention Browse Module - Meshes - ID: D000014810 - Term: Vitamin E ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03284879 **Brief Title:** Post-Marketing Surveillance Study of OTEZLA **Official Title:** OTEZLA® Tablets Drug Use-Results Survey #### Organization Study ID Info **ID:** CC-10004-PSOR-018 #### Organization **Class:** INDUSTRY **Full Name:** Amgen ### Status Module #### Completion Date **Date:** 2022-03-20 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2022-04-29 **Type:** ACTUAL **Last Update Submit Date:** 2022-04-28 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2021-10-31 **Type:** ACTUAL #### Start Date **Date:** 2017-09-05 **Type:** ACTUAL **Status Verified Date:** 2022-04 #### Study First Post Date **Date:** 2017-09-15 **Type:** ACTUAL **Study First Submit Date:** 2017-08-31 **Study First Submit QC Date:** 2017-09-13 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Amgen #### Responsible Party **Type:** SPONSOR ### Description Module **Brief Summary:** To evaluate the safety and efficacy of OTEZLA in actual clinical settings of use in patients with Psoriasis vulgaris that is with an inadequate response to topical therapies and Psoriasis arthropathica 1. Planned registration period 2 years 2. Planned surveillance period for 4 years from 6 months after launch ### Conditions Module **Conditions:** - Psoriasis **Keywords:** - Psoriasis vulgaris - Psoriasis arthropathica - Serious infections - Gastrointestinal Disorders - Serious Hypersensitivity - Weight Decrease - Vasculitis - Malignancies - Depression and Suicidal Ideation ### Design Module #### Design Info **Observational Model:** CASE_ONLY **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 1086 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients with psoriasis vulgaris and patients with psoriatic arthritis who are treated with OTEZLA Tablets **Label:** Patients with PsV and PsA treated with OTEZLA Tablets ### Outcomes Module #### Primary Outcomes **Description:** Number of participants with adverse events **Measure:** Adverse Events (AEs) **Time Frame:** Up to approximately 12 months **Description:** General Improvement Rating will be assessed by physician's observation **Measure:** General Improvement Rating **Time Frame:** Approximately 1 year from administration **Description:** Visual Analog Scale (VAS) pain will be used for psoriatic arthritis patients' assessment. **Measure:** General health assessment on VAS **Time Frame:** Approximately 1 year from administration **Description:** PGA: assessment for psoriatic vulgaris by physician to classify disease activity in a consistent manner **Measure:** Changes in physician general assessment **Time Frame:** Approximately 1 year from administration **Description:** The Dermatology Life Quality Index or DLQI is a dermatology-specific Quality of Life instrument. It is a simple 10-question validated questionnaire **Measure:** Percentage of patients with Dermatology Life Quality Index (DLQI) **Time Frame:** Approximately 1 year from administration **Description:** Activity for arthritis Baseline is calculated by Disease Activity Score-DAS28 method **Measure:** Change from baseline activity for arthritis **Time Frame:** Approximately 1 year from administration ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: - Patients who have received a diagnosis of either of the following diseases and have received OTEZLA for the first time will be included in this survey. * Psoriasis vulgaris that is with an inadequate response to topical therapies * Psoriasis arthropathica Exclusion Criteria: * N/A **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT **Study Population:** Patients who have received a diagnosis of either of the following diseases and have received OTEZLA for the first time will be included in this survey. * Psoriasis vulgaris that is with an inadequate response to topical therapies * Psoriasis arthropathica ### Contacts Locations Module #### Locations **Location 1:** **City:** Kitakyushu **Country:** Japan **Facility:** Tugi dermatology clinic **State:** Fukuoka **Zip:** 804-0081 #### Overall Officials **Official 1:** **Affiliation:** Amgen **Name:** MD **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **Access Criteria:** Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors. If not approved, a Data Sharing Independent Review Panel will arbitrate and make the final decision. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the URL below. **Description:** De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request **Info Types:** - STUDY_PROTOCOL - SAP - ICF - CSR **IPD Sharing:** YES **Time Frame:** Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study. **URL:** http://www.amgen.com/datasharing ### References Module #### See Also Links **Label:** AmgenTrials clinical trials website **URL:** http://www.amgentrials.com **Label:** Expanded Access for CC-10004 **URL:** https://clinicaltrials.gov/ct2/show/NCT03284879?term=CC-10004-PSOR-018&rank=1 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000017444 - Term: Skin Diseases, Papulosquamous - ID: D000012871 - Term: Skin Diseases ### Condition Browse Module - Browse Branches - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions - Abbrev: BC01 - Name: Infections - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC20 - Name: Immune System Diseases - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: BC06 - Name: Digestive System Diseases - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: BC05 - Name: Musculoskeletal Diseases ### Condition Browse Module - Browse Leaves - ID: M7058 - Name: Depression - Relevance: LOW - As Found: Unknown - ID: M10283 - Name: Infections - Relevance: LOW - As Found: Unknown - ID: M6368 - Name: Communicable Diseases - Relevance: LOW - As Found: Unknown - ID: M5114 - Name: Body Weight - Relevance: LOW - As Found: Unknown - ID: M18102 - Name: Weight Loss - Relevance: LOW - As Found: Unknown - ID: M10018 - Name: Hypersensitivity - Relevance: LOW - As Found: Unknown - ID: M7061 - Name: Depressive Disorder - Relevance: LOW - As Found: Unknown - ID: M14422 - Name: Psoriasis - Relevance: HIGH - As Found: Psoriasis - ID: M29364 - Name: Suicidal Ideation - Relevance: LOW - As Found: Unknown - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M17405 - Name: Vasculitis - Relevance: LOW - As Found: Unknown - ID: M18178 - Name: Arthritis, Psoriatic - Relevance: LOW - As Found: Unknown - ID: M15674 - Name: Skin Diseases - Relevance: LOW - As Found: Unknown - ID: M19713 - Name: Skin Diseases, Papulosquamous - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000011565 - Term: Psoriasis ### Intervention Browse Module - Browse Branches - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: ARhu - Name: Antirheumatic Agents - Abbrev: Analg - Name: Analgesics - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M341074 - Name: Apremilast - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00189579 **Brief Title:** Addition of Herceptin to a Carboplatin-Paclitaxel Regimen in Patients With Ovarian Cancer **Official Title:** A Phase II Study Designed to Evaluate the Safety and Efficacy of the Addition of Herceptin to a Carboplatin-Paclitaxel Regimen in Early Relapse Patients (< 6 Months) With Ovarian Cancer, Overexpressing HER2, Previously Treated With Carboplatin-Paclitaxel #### Organization Study ID Info **ID:** TCHERCEPTIN1 #### Organization **Class:** OTHER **Full Name:** ARCAGY/ GINECO GROUP ### Status Module #### Completion Date **Date:** 2007-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2007-08-17 **Type:** ESTIMATED **Last Update Submit Date:** 2007-08-16 **Overall Status:** TERMINATED **Status Verified Date:** 2007-08 #### Study First Post Date **Date:** 2005-09-19 **Type:** ESTIMATED **Study First Submit Date:** 2005-09-12 **Study First Submit QC Date:** 2005-09-12 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Hoffmann-La Roche #### Lead Sponsor **Class:** OTHER **Name:** ARCAGY/ GINECO GROUP ### Description Module **Brief Summary:** The purpose of this study is to evaluate whether or not the addition of Herceptin may be of benefit to a standard regimen of carboplatin-paclitaxel. ### Conditions Module **Conditions:** - Ovarian Cancer **Keywords:** - HERCEPTIN - Carboplatin - Paclitaxel - Relapse ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 45 **Type:** ESTIMATED **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Name:** Herceptin **Type:** DRUG ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * 18 years or older; patients with histologically proven diagnosis of ovarian cancer. * Patients with metastatic disease and an overexpression of HER2 (as determined by immunochemistry) * Patients who have progressed while receiving treatment, or within 6 months after completion of treatment. Patients must have received carboplatin and paclitaxel. * Patients who have received at minimum one line of chemotherapy * 3 weeks minimum since last treatment with chemotherapy must have elapsed * Patients must have at least 1 measurable lesion per Response Evaluation Criteria in Solid Tumors (RECIST) or an assessable cancer antigen (CA) 125 \> 1.25 x upper limit of normal (ULN) * Patients must have ECOG of 2 or less * Left ventricular ejection fraction (LVEF) of 50% or better * Patients have given their signed and verbal consent Exclusion Criteria: * Previous treatment with Herceptin or similar products affected growth factors (eg: Iressa) * Another experimental treatment in the previous 30 days * No overexpression of HER2 receptors * Patients having received high-dose chemotherapy or stem-cell interventions * Other cancers within the last 5 years * Patients with dyspnea at rest or requiring oxygen therapy **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Paris **Country:** France **Facility:** Hopital Hotel-Dieu **Zip:** 75004 #### Overall Officials **Official 1:** **Affiliation:** Hopital Hotel-Dieu **Name:** Eric Pujade-Lauraine, MD, PhD **Role:** STUDY_CHAIR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000004701 - Term: Endocrine Gland Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000010049 - Term: Ovarian Diseases - ID: D000000291 - Term: Adnexal Diseases - ID: D000005831 - Term: Genital Diseases, Female - ID: D000052776 - Term: Female Urogenital Diseases - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications - ID: D000091642 - Term: Urogenital Diseases - ID: D000005833 - Term: Genital Neoplasms, Female - ID: D000014565 - Term: Urogenital Neoplasms - ID: D000091662 - Term: Genital Diseases - ID: D000004700 - Term: Endocrine System Diseases - ID: D000006058 - Term: Gonadal Disorders - ID: D000002277 - Term: Carcinoma - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009370 - Term: Neoplasms by Histologic Type ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: BC19 - Name: Gland and Hormone Related Diseases - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M12974 - Name: Ovarian Neoplasms - Relevance: HIGH - As Found: Ovarian Cancer - ID: M1704 - Name: Carcinoma, Ovarian Epithelial - Relevance: HIGH - As Found: Ovarian Cancer - ID: M5534 - Name: Carcinoma - Relevance: LOW - As Found: Unknown - ID: M7863 - Name: Endocrine Gland Neoplasms - Relevance: LOW - As Found: Unknown - ID: M12972 - Name: Ovarian Diseases - Relevance: LOW - As Found: Unknown - ID: M3643 - Name: Adnexal Diseases - Relevance: LOW - As Found: Unknown - ID: M8943 - Name: Genital Diseases, Female - Relevance: LOW - As Found: Unknown - ID: M2876 - Name: Genital Diseases - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M8945 - Name: Genital Neoplasms, Female - Relevance: LOW - As Found: Unknown - ID: M17315 - Name: Urogenital Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7862 - Name: Endocrine System Diseases - Relevance: LOW - As Found: Unknown - ID: M9163 - Name: Gonadal Disorders - Relevance: LOW - As Found: Unknown - ID: M12320 - Name: Neoplasms, Glandular and Epithelial - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: T4352 - Name: Ovarian Cancer - Relevance: HIGH - As Found: Ovarian Cancer - ID: T4354 - Name: Ovarian Epithelial Cancer - Relevance: HIGH - As Found: Ovarian Cancer ### Condition Browse Module - Meshes - ID: D000010051 - Term: Ovarian Neoplasms - ID: D000077216 - Term: Carcinoma, Ovarian Epithelial ### Intervention Browse Module - Ancestors - ID: D000074322 - Term: Antineoplastic Agents, Immunological - ID: D000000970 - Term: Antineoplastic Agents ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M19537 - Name: Paclitaxel - Relevance: LOW - As Found: Unknown - ID: M231 - Name: Albumin-Bound Paclitaxel - Relevance: LOW - As Found: Unknown - ID: M18650 - Name: Carboplatin - Relevance: LOW - As Found: Unknown - ID: M325 - Name: Trastuzumab - Relevance: HIGH - As Found: Shoulder - ID: M1346 - Name: Antineoplastic Agents, Immunological - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000068878 - Term: Trastuzumab ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04746079 **Brief Title:** Positive Imagery Therapy and the Incidence of Emergence Reactions With the Use of Ketamine **Official Title:** Positive Imagery Therapy and the Incidence of Emergence Reactions With the Use of Ketamine #### Organization Study ID Info **ID:** 20-025 #### Organization **Class:** OTHER **Full Name:** Mercy Health Ohio ### Status Module #### Completion Date **Date:** 2023-02 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2022-09-23 **Type:** ACTUAL **Last Update Submit Date:** 2022-09-22 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2023-02 **Type:** ESTIMATED #### Start Date **Date:** 2021-02-05 **Type:** ACTUAL **Status Verified Date:** 2022-05 #### Study First Post Date **Date:** 2021-02-09 **Type:** ACTUAL **Study First Submit Date:** 2021-02-08 **Study First Submit QC Date:** 2021-02-08 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Lake Erie College of Osteopathic Medicine #### Lead Sponsor **Class:** OTHER **Name:** Mercy Health Ohio #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this study is to determine if positive imagery therapy while using ketamine in procedural sedation reduces the number of emergence reactions and impacts pre and post-procedural anxiety. **Detailed Description:** This is a multi-center, randomized controlled trial looking at if positive imagery therapy while using ketamine during procedural sedation will reduce the number of emergence reactions and impacts pre and post procedural anxiety. After informed consent has been established and the subject is determined to meet eligibility criteria, participants will be randomized into 2 groups. The interventional group will undergo positive imagery therapy during sedation and the control will not. The duration of subject participation will be from the onset of sedation beginning until the patient is recovered. ### Conditions Module **Conditions:** - Procedural Sedation - Emergence Delirium **Keywords:** - ketamine ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** The sealed envelope method. ##### Masking Info **Masking:** NONE **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 180 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Perform procedural sedation with slow push of ketamine, 1.5mg/kg, over thirty seconds while reading the Positive Imagery Therapy below: "Relax and close your eyes. Take deep breaths in through your nose and out through your mouth as you listen to the sound of my voice. (Three second pause.) I want you to picture yourself lying on a towel on a soft sandy beach. (Three second pause.) You can see a palm trees swaying in the wind beneath a bright blue sky with a few white puffy clouds. (Three second pause.) You can feel the sand between your toes, the warm sunlight on your skin and a cool breeze. (Three second pause) You can smell coconut lotion in the breeze. (Three second pause.) You can hear the sound of waves gently crashing on the beach and seagulls crying in the distance. \*\*End of vignette\*\* **Intervention Names:** - Behavioral: Positive Imagery Therapy **Label:** Positive Imagery Therapy **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Perform procedural sedation with slow push of ketamine, 1.5mg/kg, over thirty seconds with no positive imagery therapy. **Label:** Control **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Positive Imagery Therapy **Description:** Perform procedural sedation with slow push of ketamine, 1.5mg/kg, over thirty seconds while reading the Positive Imagery Therapy. **Name:** Positive Imagery Therapy **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** RASS score measured is -5 to +4. A lower score indicates someone is more alert, calm, drowsy indicating the patient has no emergence reaction. Higher score indicates an emergence reaction occurred. **Measure:** Richmond Agitation Sedation Score (RASS) for those receiving procedural sedation with and without ketamine **Time Frame:** From beginning of ketamine administration until patient returns to baseline. Estimated less than 1 hour. **Description:** PAS is measures from 0-16. A lower score indicated no agitation or emergence reaction while a higher score does. **Measure:** Pittsburgh Agitation Score for those receiving procedural sedation with and without ketamine **Time Frame:** From beginning of ketamine administration until patient returns to baseline. Estimated less than 1 hour. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * at least 18 years of age * requires procedural sedation in the emergency department for whom ketamine is selected by the provider as the medication for procedural sedation Exclusion Criteria: * age below 18 years of age * any patient with a contraindication to the use of ketamine for the procedural sedation * any prisoners * pregnant females **Healthy Volunteers:** True **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Todd Bolotin, MD **Phone:** (330) 219-3838 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Kayla Prokopakis, DO **Phone:** (740) 512-8498 **Role:** CONTACT #### Locations **Location 1:** **City:** Youngstown **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Todd Bolotin, MD - **Phone:** 330-219-3838 - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Chad Donley, MD - **Phone:** 740-607-6318 - **Role:** CONTACT **Country:** United States **Facility:** Mercy Health St Elizabeth Youngstown **State:** Ohio **Status:** RECRUITING **Zip:** 44504 #### Overall Officials **Official 1:** **Affiliation:** Bon Secours Mercy Health **Name:** Todd Bolotin, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Sener S, Eken C, Schultz CH, Serinken M, Ozsarac M. Ketamine with and without midazolam for emergency department sedation in adults: a randomized controlled trial. Ann Emerg Med. 2011 Feb;57(2):109-114.e2. doi: 10.1016/j.annemergmed.2010.09.010. **PMID:** 20970888 **Citation:** Akhlaghi N, Payandemehr P, Yaseri M, Akhlaghi AA, Abdolrazaghnejad A. Premedication With Midazolam or Haloperidol to Prevent Recovery Agitation in Adults Undergoing Procedural Sedation With Ketamine: A Randomized Double-Blind Clinical Trial. Ann Emerg Med. 2019 May;73(5):462-469. doi: 10.1016/j.annemergmed.2018.11.016. Epub 2019 Jan 3. **PMID:** 30611640 **Citation:** Stoker AD, Rosenfeld DM, Buras MR, Alvord JM, Gorlin AW. Evaluation of Clinical Factors Associated with Adverse Drug Events in Patients Receiving Sub-Anesthetic Ketamine Infusions. J Pain Res. 2019 Dec 23;12:3413-3421. doi: 10.2147/JPR.S217005. eCollection 2019. **PMID:** 31920366 **Citation:** Schwenk ES, Goldberg SF, Patel RD, Zhou J, Adams DR, Baratta JL, Viscusi ER, Epstein RH. Adverse Drug Effects and Preoperative Medication Factors Related to Perioperative Low-Dose Ketamine Infusions. Reg Anesth Pain Med. 2016 Jul-Aug;41(4):482-7. doi: 10.1097/AAP.0000000000000416. **PMID:** 27281730 **Citation:** Sherwin TS, Green SM, Khan A, Chapman DS, Dannenberg B. Does adjunctive midazolam reduce recovery agitation after ketamine sedation for pediatric procedures? A randomized, double-blind, placebo-controlled trial. Ann Emerg Med. 2000 Mar;35(3):229-38. doi: 10.1016/s0196-0644(00)70073-4. **PMID:** 10692189 **Citation:** Vardy JM, Dignon N, Mukherjee N, Sami DM, Balachandran G, Taylor S. Audit of the safety and effectiveness of ketamine for procedural sedation in the emergency department. Emerg Med J. 2008 Sep;25(9):579-82. doi: 10.1136/emj.2007.056200. **PMID:** 18723707 **Citation:** Newton A, Fitton L. Intravenous ketamine for adult procedural sedation in the emergency department: a prospective cohort study. Emerg Med J. 2008 Aug;25(8):498-501. doi: 10.1136/emj.2007.053421. **PMID:** 18660398 **Citation:** Strayer RJ, Nelson LS. Adverse events associated with ketamine for procedural sedation in adults. Am J Emerg Med. 2008 Nov;26(9):985-1028. doi: 10.1016/j.ajem.2007.12.005. Erratum In: Am J Emerg Med. 2009 May;27(4):512. **PMID:** 19091264 **Citation:** Green SM, Roback MG, Krauss B, Brown L, McGlone RG, Agrawal D, McKee M, Weiss M, Pitetti RD, Hostetler MA, Wathen JE, Treston G, Garcia Pena BM, Gerber AC, Losek JD; Emergency Department Ketamine Meta-Analysis Study Group. Predictors of emesis and recovery agitation with emergency department ketamine sedation: an individual-patient data meta-analysis of 8,282 children. Ann Emerg Med. 2009 Aug;54(2):171-80.e1-4. doi: 10.1016/j.annemergmed.2009.04.004. Epub 2009 Jun 6. **PMID:** 19501426 **Citation:** Treston G, Bell A, Cardwell R, Fincher G, Chand D, Cashion G. What is the nature of the emergence phenomenon when using intravenous or intramuscular ketamine for paediatric procedural sedation? Emerg Med Australas. 2009 Aug;21(4):315-22. doi: 10.1111/j.1742-6723.2009.01203.x. **PMID:** 19682018 **Citation:** Somashekara SC, Govindadas D, Devashankaraiah G, Mahato R, Deepalaxmi S, Srinivas V, Murugesh JV, Devanand. Midazolam premedication in attenuating ketamine psychic sequelae. J Basic Clin Pharm. 2010 Sep;1(4):209-13. Epub 2010 Nov 15. **PMID:** 24825990 **Citation:** Trivedi S, Kumar R, Tripathi AK, Mehta RK. A Comparative Study of Dexmedetomidine and Midazolam in Reducing Delirium Caused by Ketamine. J Clin Diagn Res. 2016 Aug;10(8):UC01-4. doi: 10.7860/JCDR/2016/18397.8225. Epub 2016 Aug 1. **PMID:** 27656531 **Citation:** Perumal DK, Adhimoolam M, Selvaraj N, Lazarus SP, Mohammed MA. Midazolam premedication for Ketamine-induced emergence phenomenon: A prospective observational study. J Res Pharm Pract. 2015 Apr-Jun;4(2):89-93. doi: 10.4103/2279-042X.155758. **PMID:** 25984547 **Citation:** Bergman SA. Ketamine: review of its pharmacology and its use in pediatric anesthesia. Anesth Prog. 1999 Winter;46(1):10-20. **PMID:** 10551055 **Citation:** Roback MG, Wathen JE, Bajaj L, Bothner JP. Adverse events associated with procedural sedation and analgesia in a pediatric emergency department: a comparison of common parenteral drugs. Acad Emerg Med. 2005 Jun;12(6):508-13. doi: 10.1197/j.aem.2004.12.009. **PMID:** 15930401 **Citation:** Cheong SH, Lee KM, Lim SH, Cho KR, Kim MH, Ko MJ, Shim JC, Oh MK, Kim YH, Lee SE. Brief report: the effect of suggestion on unpleasant dreams induced by ketamine administration. Anesth Analg. 2011 May;112(5):1082-5. doi: 10.1213/ANE.0b013e31820eeb0e. Epub 2011 Feb 23. **PMID:** 21346162 **Citation:** Asl Aminabadi N, Erfanparast L, Sohrabi A, Ghertasi Oskouei S, Naghili A. The Impact of Virtual Reality Distraction on Pain and Anxiety during Dental Treatment in 4-6 Year-Old Children: a Randomized Controlled Clinical Trial. J Dent Res Dent Clin Dent Prospects. 2012 Fall;6(4):117-24. doi: 10.5681/joddd.2012.025. Epub 2012 Nov 12. **PMID:** 23277857 **Citation:** Huet A, Lucas-Polomeni MM, Robert JC, Sixou JL, Wodey E. Hypnosis and dental anesthesia in children: a prospective controlled study. Int J Clin Exp Hypn. 2011 Oct-Dec;59(4):424-40. doi: 10.1080/00207144.2011.594740. **PMID:** 21867378 **Citation:** Heilbrunn BR, Wittern RE, Lee JB, Pham PK, Hamilton AH, Nager AL. Reducing anxiety in the pediatric emergency department: a comparative trial. J Emerg Med. 2014 Dec;47(6):623-31. doi: 10.1016/j.jemermed.2014.06.052. Epub 2014 Sep 27. **PMID:** 25271180 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000003693 - Term: Delirium - ID: D000003221 - Term: Confusion - ID: D000019954 - Term: Neurobehavioral Manifestations - ID: D000009461 - Term: Neurologic Manifestations - ID: D000009422 - Term: Nervous System Diseases - ID: D000019965 - Term: Neurocognitive Disorders - ID: D000001523 - Term: Mental Disorders - ID: D000011183 - Term: Postoperative Complications - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M772 - Name: Emergence Delirium - Relevance: HIGH - As Found: Emergence Delirium - ID: M6894 - Name: Delirium - Relevance: LOW - As Found: Unknown - ID: M6446 - Name: Confusion - Relevance: LOW - As Found: Unknown - ID: M21826 - Name: Neurobehavioral Manifestations - Relevance: LOW - As Found: Unknown - ID: M12404 - Name: Neurologic Manifestations - Relevance: LOW - As Found: Unknown - ID: M21836 - Name: Neurocognitive Disorders - Relevance: LOW - As Found: Unknown - ID: M4815 - Name: Mental Disorders - Relevance: LOW - As Found: Unknown - ID: M14473 - Name: Psychotic Disorders - Relevance: LOW - As Found: Unknown - ID: M14065 - Name: Postoperative Complications - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000071257 - Term: Emergence Delirium ### Intervention Browse Module - Browse Branches - Abbrev: Analg - Name: Analgesics - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: HB - Name: Herbal and Botanical ### Intervention Browse Module - Browse Leaves - ID: M10674 - Name: Ketamine - Relevance: LOW - As Found: Unknown - ID: T119 - Name: Coconut - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03885479 **Brief Title:** Impact of Different Dietary IgGs on the Pathogenesis of IBD **Official Title:** Impact of Different Dietary IgGs on the Pathogenesis of Inflammatory Bowel Disease #### Organization Study ID Info **ID:** IBDIgG #### Organization **Class:** OTHER **Full Name:** Assiut University ### Status Module #### Completion Date **Date:** 2020-03-31 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** NOT_YET_RECRUITING #### Last Update Post Date **Date:** 2019-03-21 **Type:** ACTUAL **Last Update Submit Date:** 2019-03-20 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2019-12-31 **Type:** ESTIMATED #### Start Date **Date:** 2019-04-01 **Type:** ESTIMATED **Status Verified Date:** 2019-03 #### Study First Post Date **Date:** 2019-03-21 **Type:** ACTUAL **Study First Submit Date:** 2019-03-18 **Study First Submit QC Date:** 2019-03-20 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Assiut University #### Responsible Party **Investigator Affiliation:** Assiut University **Investigator Full Name:** Hebatallah Mohammed Abdelwahab Mohammed Abdelrahman **Investigator Title:** Principal investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** Identify the association between certain food IgGs (Wheat, rice, broad beans, cow milk, eggs, chicken and beef) and the immunological response in patients with IBD **Detailed Description:** Inflammatory bowel disease (IBD) is comprised of two major disorders: ulcerative colitis and Crohn disease. Ulcerative colitis and Crohn disease have distinct pathologic and clinical characteristics but their pathogenesis remains poorly understood. In 2015, an estimated 1.3% of US adults (3 million) reported being diagnosed with IBD (either Crohn's disease or ulcerative colitis). This was a large increase from 1999 (0.9% or 2 million adults).The incidence and prevalence of Crohn disease and ulcerative colitis (UC) appear to be lower in Asia and the Middle East , however, in some newly industrialized countries in Africa, Asia, and South America, the incidence of IBD has been rising. Ulcerative colitis is a chronic inflammatory condition characterized by relapsing and remitting episodes of inflammation limited to the mucosal layer of the colon. It almost invariably involves the rectum and typically extends in a proximal and continuous fashion to involve other portions of the colon. Crohn disease is characterized by transmural inflammation and by skip lesions. The transmural inflammatory nature of Crohn disease may lead to fibrosis and strictures, and to obstructive clinical presentations that are not typically seen in ulcerative colitis. The transmural inflammation more commonly results in sinus tracts, giving rise to microperforations and fistulae. Food antigens are thought to trigger an immunologic response resulting in the development of IBD. However, specific pathogenic antigens have not been identified. While studies attempting to associate specific diets with the development of IBD have had inconsistent results, the data suggest that a "Western" style diet (processed, fried, and sugary foods) is associated with an increased risk of developing Crohn disease, and possibly ulcerative colitis. To date, studies concerning food intolerance in IBD have largely focused on classic food allergies based on IgE mediated antibody responses. The levels of total or food-specific IgEs have been observed to be increased in the sera of IBD patients, and IgE-mediated food allergies are more frequent in IBD patients than in those without IBDs. Nevertheless, reactions mediated by food specific IgGs, featuring a more delayed response following exposure to a particular antigen, are also expected to contribute to adverse reactions in IBD, and food-specific IgGs help physicians identify the candidate food for elimination in IBD patients. Furthermore, IgG-mediated adverse reactions have also been reported to be involved in some cases of food hypersensitivity. Elimination diet can help in the remission of the disease. An elimination diet involves removing a food from the diet for a period of time and seeing whether symptoms resolve during that time. In patients receiving enteral nutrition, it involves introducing one new food at a time to identify foods that precipitate IBD symptoms. Many patients can identify foods that they believe may precipitate or worsen their disease and it is reasonable for them to avoid such foods. Using an elimination diet to identify at-risk foods may decrease the possibility of a "flare" of IBD. ### Conditions Module **Conditions:** - Inflammatory Bowel Diseases - Food Intolerance **Keywords:** - Inflammatory bowel disease - Food specific IgGs ### Design Module #### Design Info **Observational Model:** CASE_CONTROL **Time Perspective:** CROSS_SECTIONAL #### Enrollment Info **Count:** 150 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 1. Full history taking and examination 2. Colonoscopy, biopsy and histopathology to determine the extent of the lesion 3. An enzyme-linked immunosorbent assay (ELISA) for the semi-quantitative analysis of serum food-specific IgGs against 7 food-derived antigens (Wheat, rice, beans, cow milk, eggs, chicken and beef) 4. Patients will be categorized into 3 groups (UC patients, Crohn disease patients and controls) 5. All of the following factors will be taken into consideration; type and duration of the treatment, age of diagnosis, BMI, smoking status and activity of the disease at the time of the study **Intervention Names:** - Diagnostic Test: ELISA for the semi-quantitative analysis of serum food-specific IgGs against food-derived antigens **Label:** IBD patients #### Arm Group 2 **Description:** An enzyme-linked immunosorbent assay (ELISA) for the semi-quantitative analysis of serum food-specific IgGs against 7 food-derived antigens (Wheat, rice, beans, cow milk, eggs, chicken and beef) **Intervention Names:** - Diagnostic Test: ELISA for the semi-quantitative analysis of serum food-specific IgGs against food-derived antigens **Label:** Controls ### Interventions #### Intervention 1 **Arm Group Labels:** - Controls - IBD patients **Description:** An enzyme-linked immunosorbent assay (ELISA) for the semi-quantitative analysis of serum food-specific IgGs against 7 food-derived antigens (Wheat, rice, beans, cow milk, eggs, chicken and beef) **Name:** ELISA for the semi-quantitative analysis of serum food-specific IgGs against food-derived antigens **Type:** DIAGNOSTIC_TEST ### Outcomes Module #### Primary Outcomes **Description:** Level of serum food specific IgGs in patients with Inflammatory bowel diseasea against 7 food-derived antigens (Wheat, rice, beans, cow milk, eggs, chicken and beef) **Measure:** Level of serum food specific IgGs in patients with IBD **Time Frame:** baseline ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Inflammatory bowel disease patients (Ulcerative colitis and Crohn's disease) * Patients aged ≥ 18 years old Exclusion Criteria: • Patients who started TNF-α inhibitor (Infliximab) **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Case group: IBD patients Control group: Normal individuals ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Hebatallah Mohammed Abdelwahab Mohammed Abdelrahman, Doctor **Phone:** 00201068231787 **Role:** CONTACT **Contact 2:** **Name:** Lobna Abdelwahed Ahmed, Professor **Phone:** 00201093337630 **Role:** CONTACT #### Overall Officials **Official 1:** **Affiliation:** Assiut University **Name:** Mohamed Elyamany, Professor **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ### References Module #### References **Citation:** Silverberg MS, Satsangi J, Ahmad T, Arnott ID, Bernstein CN, Brant SR, Caprilli R, Colombel JF, Gasche C, Geboes K, Jewell DP, Karban A, Loftus EV Jr, Pena AS, Riddell RH, Sachar DB, Schreiber S, Steinhart AH, Targan SR, Vermeire S, Warren BF. Toward an integrated clinical, molecular and serological classification of inflammatory bowel disease: report of a Working Party of the 2005 Montreal World Congress of Gastroenterology. Can J Gastroenterol. 2005 Sep;19 Suppl A:5A-36A. doi: 10.1155/2005/269076. **PMID:** 16151544 **Citation:** Dahlhamer JM, Zammitti EP, Ward BW, Wheaton AG, Croft JB. Prevalence of Inflammatory Bowel Disease Among Adults Aged >/=18 Years - United States, 2015. MMWR Morb Mortal Wkly Rep. 2016 Oct 28;65(42):1166-1169. doi: 10.15585/mmwr.mm6542a3. **PMID:** 27787492 **Citation:** Nguyen GC, Chong CA, Chong RY. National estimates of the burden of inflammatory bowel disease among racial and ethnic groups in the United States. J Crohns Colitis. 2014 Apr;8(4):288-95. doi: 10.1016/j.crohns.2013.09.001. Epub 2013 Sep 24. **PMID:** 24074875 **Citation:** Ananthakrishnan AN, Khalili H, Konijeti GG, Higuchi LM, de Silva P, Korzenik JR, Fuchs CS, Willett WC, Richter JM, Chan AT. A prospective study of long-term intake of dietary fiber and risk of Crohn's disease and ulcerative colitis. Gastroenterology. 2013 Nov;145(5):970-7. doi: 10.1053/j.gastro.2013.07.050. Epub 2013 Aug 2. **PMID:** 23912083 **Citation:** Mekkel G, Barta Z, Ress Z, Gyimesi E, Sipka S, Zeher M. [Increased IgE-type antibody response to food allergens in irritable bowel syndrome and inflammatory bowel diseases]. Orv Hetil. 2005 Apr 24;146(17):797-802. Hungarian. **PMID:** 17918636 **Citation:** Bentz S, Hausmann M, Piberger H, Kellermeier S, Paul S, Held L, Falk W, Obermeier F, Fried M, Scholmerich J, Rogler G. Clinical relevance of IgG antibodies against food antigens in Crohn's disease: a double-blind cross-over diet intervention study. Digestion. 2010;81(4):252-64. doi: 10.1159/000264649. Epub 2010 Jan 30. **PMID:** 20130407 **Citation:** Koretz RL, Avenell A, Lipman TO, Braunschweig CL, Milne AC. Does enteral nutrition affect clinical outcome? A systematic review of the randomized trials. Am J Gastroenterol. 2007 Feb;102(2):412-29; quiz 468. doi: 10.1111/j.1572-0241.2006.01024.x. **PMID:** 17311654 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000005767 - Term: Gastrointestinal Diseases - ID: D000004066 - Term: Digestive System Diseases - ID: D000005759 - Term: Gastroenteritis - ID: D000012817 - Term: Signs and Symptoms, Digestive ### Condition Browse Module - Browse Branches - Abbrev: BC06 - Name: Digestive System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M10444 - Name: Intestinal Diseases - Relevance: HIGH - As Found: Bowel Disease - ID: M17917 - Name: Inflammatory Bowel Diseases - Relevance: HIGH - As Found: Inflammatory Bowel Disease - ID: M1256 - Name: Food Intolerance - Relevance: HIGH - As Found: Food Intolerance - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M8875 - Name: Gastroenteritis - Relevance: LOW - As Found: Unknown - ID: M15622 - Name: Signs and Symptoms, Digestive - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000007410 - Term: Intestinal Diseases - ID: D000015212 - Term: Inflammatory Bowel Diseases - ID: D000073923 - Term: Food Intolerance ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00865579 **Brief Title:** Open-Label Trial to Determine the Long-Term Safety of Safinamide in Parkinson's Disease Patients **Official Title:** Open-Label Trial to Determine the Long-Term Safety of Safinamide in Parkinson's Disease Patients #### Organization Study ID Info **ID:** 28850 #### Organization **Class:** INDUSTRY **Full Name:** Newron Pharmaceuticals SPA #### Secondary ID Infos **ID:** 63,901 **ID:** EudraCT-Number: 2008-005492-94 ### Status Module #### Completion Date **Date:** 2012-06 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-09-18 **Type:** ACTUAL **Last Update Submit Date:** 2017-09-15 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2012-06 **Type:** ACTUAL #### Start Date **Date:** 2009-04 **Type:** ACTUAL **Status Verified Date:** 2017-09 #### Study First Post Date **Date:** 2009-03-19 **Type:** ESTIMATED **Study First Submit Date:** 2009-03-10 **Study First Submit QC Date:** 2009-03-18 **Why Stopped:** study terminated early due to change in Sponsorship ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Newron Pharmaceuticals SPA #### Responsible Party **Type:** SPONSOR ### Description Module **Brief Summary:** Parkinson's Disease (PD) is a major neurodegenerative disorder in which there is a progressive loss of dopamine-containing neurons. The understanding that PD is a syndrome of dopamine (DA) deficiency led to the introduction in the clinical practice of L-dopa, a precursor of DA that crosses the blood brain barrier, and also to the use of selective inhibitors of MAO-B, the major DA metabolising enzyme in humans. Safinamide is an inhibitor of MAO-B. This study is to evaluate the long term safety and tolerability of safinamide in PD patients, that have already completed a previous clinical study with Safinamide. The physical and neurological conditions as well as other safety parameters will get compared from baseline to subsequent visits. ### Conditions Module **Conditions:** - Parkinson's Disease **Keywords:** - MAO inhibitors - motor fluctuations - dyskinesia - Parkinson's Disease ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 964 **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** All subjects to receive first 50mg/d Safinamide with an increase of target dose of 100mg/d after 14 days of taper period until end of treatment visit. In case of any intolerance the daily dose of 100mg might be decreased to 50mg/d. Patients permanently discontinuing treatment will enter a 7day taper phase before treatment discontinuation at a dose of 50mg/day. Subjects already taking 50mg/d may stop Safinamide immediately. **Intervention Names:** - Drug: Safinamide **Label:** 1 **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - 1 **Description:** The Investigational Medicinal Product will be provided by the Sponsor in the form of tablets at dosage strengths of safinamide 50 mg (small - 7 mm) or safinamide 100 mg (large - 9 mm). Trial Medication is to be taken once daily, in the morning. **Name:** Safinamide **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Change from baseline in Physical Exams **Time Frame:** Anticipated time frame up to 3 years **Measure:** Change from baseline in Neurologic Exams **Time Frame:** Anticipated time frame up to 3 years **Measure:** Change from baseline in Vital Signs **Time Frame:** Anticipated time frame up to 3 years **Measure:** Change from baseline in Laboratory Evaluations **Time Frame:** Anticipated time frame up to 3 years **Measure:** Change from baseline in Electrocardiograms **Time Frame:** Anticipated time frame up to 3 years **Measure:** Summary of Participants who had Adverse Experiences **Time Frame:** Anticipated time frame up to 3 years **Measure:** Change from baseline in Unified Parkinson's Disease Rating Scale (UPDRS) **Time Frame:** Anticipated time frame up to 3 years **Measure:** Change from baseline in Dermatologic Exams **Time Frame:** Anticipated time frame up to 3 years **Measure:** Change from baseline in Ophthalmologic Exams **Time Frame:** Anticipated time frame up to 3 years #### Secondary Outcomes **Measure:** Change from baseline in Health Resource Utilisation **Time Frame:** Anticipated time frame up to 3 years **Measure:** Change from baseline in EuroQol Group EQ-5D™ Quality of Life Scale **Time Frame:** Anticipated time frame up to 3 years **Measure:** Change from baseline in Parkinson Disease Questionnaire 39 (PDQ-39) **Time Frame:** Anticipated time frame up to 3 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. the subject has completed a previous clinical study with Safinamide in PD 2. the subject successfully completed all trial requirements of the antecedent trial 3. if female, they must be either post-menopausal for at least 2 years, surgically sterilized or have undergone hysterectomy or, if of child bearing potential, they must be willing to avoid pregnancy by using an adequate method of contraception for four weeks prior to, during and four weeks after the last dose of study medication. For the purpose of this trial women of child bearing potential are defined of all female subjects after puberty unless they are postmenopausal for at least two years, are surgically sterile or are sexually inactive 4. subjects must be willing and able to participate in the trial and provide written informed consent Exclusion Criteria: 1. the subject experienced a clinically significant adverse effect to attributable to Investigational Medicinal Product (IMP) during a previous trial that could put the subject at risk for further treatment with Safinamide 2. if female, the subject is pregnant or lactating 3. any medical issues, which have emerged since the initial clinical trial, that in the opinion of the investigator precludes a subject's ability to participate in this open-label trial **Minimum Age:** 30 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Timis **Country:** Romania **Facility:** Research Site #### Overall Officials **Official 1:** **Affiliation:** EMD Serono Inc., an Affiliate of Merck KGaA, Darmstadt, Germany **Name:** Jonathan Willmer, MD **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **Description:** No efficacy endpoints were analyzed in this trial. **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020734 - Term: Parkinsonian Disorders - ID: D000001480 - Term: Basal Ganglia Diseases - ID: D000001927 - Term: Brain Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000009069 - Term: Movement Disorders - ID: D000080874 - Term: Synucleinopathies - ID: D000019636 - Term: Neurodegenerative Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases ### Condition Browse Module - Browse Leaves - ID: M13213 - Name: Parkinson Disease - Relevance: HIGH - As Found: Parkinson's Disease - ID: M22574 - Name: Dyskinesias - Relevance: LOW - As Found: Unknown - ID: M22494 - Name: Parkinsonian Disorders - Relevance: LOW - As Found: Unknown - ID: M25603 - Name: Ganglion Cysts - Relevance: LOW - As Found: Unknown - ID: M16358 - Name: Synovial Cyst - Relevance: LOW - As Found: Unknown - ID: M4774 - Name: Basal Ganglia Diseases - Relevance: LOW - As Found: Unknown - ID: M5204 - Name: Brain Diseases - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M12029 - Name: Movement Disorders - Relevance: LOW - As Found: Unknown - ID: M2217 - Name: Synucleinopathies - Relevance: LOW - As Found: Unknown - ID: M21558 - Name: Neurodegenerative Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000010300 - Term: Parkinson Disease ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M11960 - Name: Monoamine Oxidase Inhibitors - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00294879 **Brief Title:** Effects of Inspiration Rise Time on Work of Breathing and Comfort of Conscious Patients on Mechanical Ventilation #### Organization Study ID Info **ID:** LBCH 0601 #### Organization **Class:** OTHER **Full Name:** Radboud University Medical Center ### Status Module #### Completion Date **Date:** 2008-03 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2008-06-02 **Type:** ESTIMATED **Last Update Submit Date:** 2008-05-30 **Overall Status:** COMPLETED #### Start Date **Date:** 2006-02 **Status Verified Date:** 2008-05 #### Study First Post Date **Date:** 2006-02-22 **Type:** ESTIMATED **Study First Submit Date:** 2006-02-21 **Study First Submit QC Date:** 2006-02-21 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Radboud University Medical Center #### Responsible Party **Old Name Title:** Radboud University Nijmegen Medical Centre **Old Organization:** Radboud University Nijmegen Medical Centre ### Description Module **Brief Summary:** Introduction Pressure Support Ventilation is widely used in patients in the ICU. Matching the patient's respiratory needs with adequate ventilator settings is necessary to ensure a low work of breathing (WOB) and maximal patient comfort. The inspiratory rise time (IRT) determines the time to reach the selected airway pressure. A short IRT results in a high peak inspiratory flow and a short time to reach that peak, but is also associated with the development of turbulent flow, resulting in increased WOB. Aim of this study is to investigate the effects of different IRT settings on WOB and patient comfort during pressure support ventilation. Methods We will performed a prospective, single blind cohort study in patients on Pressure Support Ventilation. 10 healthy adult patients admitted to the ICU after elective facial or neck surgery will be included. All patients will be ventilated in pressure support mode using a Servo 300 ventilator (Siemens. Elema, Solna, Sweden), with a positive end expiratory pressure of 5 cm H2O, pressure support level of 12 cm H2O above PEEP and an inspiratory oxygen fraction of 0.40. Patients have to be awake and cooperative (Ramsay 2). WOB will be measured with an esophageal balloon, and miniature flowmeter (Bicore system). Breathing comfort will be evaluated using a visual analogue scale (VAS) ranging from 1 to 10. WOB and patient comfort will be measured (in random order) at 0, 5, and 10% IRT. For statistical analysis the two-way analysis of variance will used. A p value of \< 0.05 will be considered statistically significant. **Detailed Description:** Introduction Pressure Support Ventilation is widely used in patients in the ICU. Matching the patient's respiratory needs with adequate ventilator settings is necessary to ensure a low work of breathing (WOB) and maximal patient comfort. The inspiratory rise time (IRT) determines the time to reach the selected airway pressure. A short IRT results in a high peak inspiratory flow and a short time to reach that peak, but is also associated with the development of turbulent flow, resulting in increased WOB. Aim of this study is to investigate the effects of different IRT settings on WOB and patient comfort during pressure support ventilation. Methods We will performed a prospective, single blind cohort study in patients on Pressure Support Ventilation. 10 healthy adult patients admitted to the ICU after elective facial or neck surgery will be included. All patients will be ventilated in pressure support mode using a Servo 300 ventilator (Siemens. Elema, Solna, Sweden), with a positive end expiratory pressure of 5 cm H2O, pressure support level of 12 cm H2O above PEEP and an inspiratory oxygen fraction of 0.40. Patients have to be awake and cooperative (Ramsay 2). WOB will be measured with an esophageal balloon, and miniature flowmeter (Bicore system). Breathing comfort will be evaluated using a visual analogue scale (VAS) ranging from 1 to 10. WOB and patient comfort will be measured (in random order) at 0, 5, and 10% IRT. For statistical analysis the two-way analysis of variance will used. A p value of \< 0.05 will be considered statistically significant. ### Conditions Module **Conditions:** - Mechanical Ventilation ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER ##### Masking Info **Masking:** SINGLE #### Enrollment Info **Count:** 10 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Name:** pressure support ventilation at 0, 5, and 10% IRT **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Measure:** Work of breathing #### Secondary Outcomes **Measure:** patient comfort ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * awake and cooperative patient * ICU admission following surgery of head or neck Exclusion Criteria: * coagulopathy * inability to sit * hypoxemia and/or hypercapnia and /or hypocapnia * Pain score \> 5 (on scale 1-10) * esophageal abnormalities * Pregnancy * COPD **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Nijmegen **Country:** Netherlands **Facility:** Radboud University Nijmegen Medical Centre **Zip:** 6500 HB #### Overall Officials **Official 1:** **Affiliation:** Radboud University Medical Center **Name:** Cornelia WE Hoedemaekers, MD PhD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M27137 - Name: Respiratory Aspiration - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03630679 **Brief Title:** A Study Evaluating Sleep, Stress and Infant Nutrition Using a Chatbot **Official Title:** A Pilot Study Evaluating Sleep, Stress, and Infant Nutrition Using a Chatbot With Parents of Preterm and Full-term Infants #### Organization Study ID Info **ID:** OBVIO-DAN-003 #### Organization **Class:** INDUSTRY **Full Name:** Danone Asia Pacific Holdings Pte, Ltd. ### Status Module #### Completion Date **Date:** 2019-05-09 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-08-13 **Type:** ACTUAL **Last Update Submit Date:** 2020-08-02 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2019-03-01 **Type:** ACTUAL #### Results First Post Date **Date:** 2020-08-03 **Type:** ACTUAL **Results First Submit Date:** 2020-06-24 **Results First Submit QC Date:** 2020-07-16 #### Start Date **Date:** 2018-11-07 **Type:** ACTUAL **Status Verified Date:** 2020-08 #### Study First Post Date **Date:** 2018-08-15 **Type:** ACTUAL **Study First Submit Date:** 2018-07-17 **Study First Submit QC Date:** 2018-08-10 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER_GOV **Name:** KK Women's and Children's Hospital #### Lead Sponsor **Class:** INDUSTRY **Name:** Danone Asia Pacific Holdings Pte, Ltd. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** 20 parents with healthy preterm infants (born at \<37 weeks of gestation), age 0-6 months and discharged from the hospital at time of enrollment and 20 parents with healthy full-term infants (born at ≥37 weeks of gestation), age 0-6 months and discharged from the hospital at time of enrollment will be enrolled to obtain records of sleep, stress, and infant nutrition from parents of infants (preterm and full-term) through interaction with the study chatbot. **Detailed Description:** 20 parents with healthy preterm infants (born at \<37 weeks of gestation), age 0-6 months and discharged from the hospital at time of enrollment and 20 parents with healthy full-term infants (born at ≥37 weeks of gestation), age 0-6 months and discharged from the hospital at time of enrollment will be enrolled to obtain records of sleep, stress, and infant nutrition from parents of infants (preterm and full-term) through interaction with the study chatbot. Data obtained from parents of preterm infants and parents of full-term infants on sleep, stress, and infant nutrition will be compared. An evaluation of the usability of the mobile app hosting the chatbot, the study chatbot and its functionality in general among this population will also be conducted ### Conditions Module **Conditions:** - Preterm Birth - Healthy ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 45 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** born at \<37 weeks of gestation **Label:** Preterm #### Arm Group 2 **Description:** born at \>/= 37 weeks of gestation **Label:** Full term Term ### Outcomes Module #### Other Outcomes **Description:** Satisfaction scale scores for Chatbot and ClaimIt based on a scale score of 1 (very dissatisfied) to 5 (very satisfied) on the Usability of Chatbot and ClaimIt eQuestionnaire **Measure:** Satisfaction Scale Scores for Chatbot and ClaimIt **Time Frame:** 8 weeks #### Primary Outcomes **Description:** Obtaining number of chats for sleep, stress, and infant nutrition from parents of infants (preterm and full-term) through interaction with the study chatbot **Measure:** Number of Chats on Sleep, Stress and Infant Nutrition **Time Frame:** 8 weeks #### Secondary Outcomes **Description:** Comparison on ratings for quality of their (parent) sleep from parents of preterm infants and parents of full-term infants **Measure:** Ratings by Parents of Quality of Their Sleep **Time Frame:** 8 weeks **Description:** Comparison of particular reasons for stress caused by the baby from parents of preterm infants and parents of full-term infants **Measure:** Particular Reasons for Stress Caused by the Baby **Time Frame:** 8 weeks **Description:** Comparison of number of chats on the feeding from parents of preterm infants and parents of full-term infants who give their infants formula **Measure:** Number of Chats on Feedings Among Parents Who Give Their Infants Formula **Time Frame:** 8 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: Subjects (parent and infant) must meet the following criteria: * Healthy infants (preterm and full-term) must be 0-6 months of age at time of enrollment * Infants must be at home (discharged from the hospital) at time of enrollment * Informed consent from parent whose age is ≥21 years * Parent must be proficient in the English language * Parent must be able to comply with the required study tasks, as per PI's judgment * In-home access to reliable internet connections; a mobile device suitable for electronic communication Exclusion Criteria: Infant must not meet any of the following criteria: * Known to have current or previous illnesses/conditions which could interfere with the study outcome (per PI's clinical judgment) * Must not be currently participating in any other clinical study Parent must not meet any of the following criteria: * Must not be known to have a significant medical condition that might interfere with the study (per PI's clinical judgment) that meets one of the following criteria: * Presence of current mental illness or history of mental illness * Any acute or chronic illness that makes the parent unsuitable for the study based on the PI's judgment * Must not be a single parent * Inability of the parent to comply with the study protocol or PI's uncertainty about the willingness or ability of the parent to comply with the protocol requirements **Healthy Volunteers:** True **Maximum Age:** 6 Months **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - CHILD **Study Population:** * 20 parents with healthy preterm infants (born at \<37 weeks of gestation), age 0-6 months and discharged from the hospital at time of enrollment * 20 parents with healthy full-term infants (born at ≥37 weeks of gestation), age 0-6 months and discharged from the hospital at time of enrollment ### Contacts Locations Module #### Locations **Location 1:** **City:** Singapore **Country:** Singapore **Facility:** KK Women's and Children's Hospital #### Overall Officials **Official 1:** **Affiliation:** KK Women's and Children's Hospital **Name:** Mei Chien Chua, MBBS **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Wong J, Foussat AC, Ting S, Acerbi E, van Elburg RM, Mei Chien C. A Chatbot to Engage Parents of Preterm and Term Infants on Parental Stress, Parental Sleep, and Infant Feeding: Usability and Feasibility Study. JMIR Pediatr Parent. 2021 Oct 26;4(4):e30169. doi: 10.2196/30169. **PMID:** 34544679 ## Document Section ### Large Document Module #### Large Docs - Date: 2018-08-31 - Filename: Prot_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: False - Label: Study Protocol - Size: 647266 - Type Abbrev: Prot - Upload Date: 2020-03-13T03:21 - Date: 2019-04-11 - Filename: SAP_001.pdf - Has ICF: False - Has Protocol: False - Has SAP: True - Label: Statistical Analysis Plan - Size: 3020813 - Type Abbrev: SAP - Upload Date: 2020-03-19T02:23 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007752 - Term: Obstetric Labor, Premature - ID: D000007744 - Term: Obstetric Labor Complications - ID: D000011248 - Term: Pregnancy Complications - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications - ID: D000091642 - Term: Urogenital Diseases ### Condition Browse Module - Browse Branches - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M25869 - Name: Premature Birth - Relevance: HIGH - As Found: Preterm Birth - ID: M10772 - Name: Obstetric Labor, Premature - Relevance: LOW - As Found: Unknown - ID: M10764 - Name: Obstetric Labor Complications - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000047928 - Term: Premature Birth ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Preterm **Deaths Num At Risk:** 20 **Description:** born at \<37 weeks of gestation **ID:** EG000 **Other Num at Risk:** 20 **Serious Number At Risk:** 20 **Title:** Preterm **Group ID:** EG001 **Title:** Full Term Term **Deaths Num At Risk:** 25 **Description:** born at \>/= 37 weeks of gestation **ID:** EG001 **Other Num at Risk:** 25 **Serious Number At Risk:** 25 **Title:** Full Term Term **Frequency Threshold:** 0 **Time Frame:** duration of the study - 4 months ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 20 **Group ID:** BG001 **Value:** 25 **Group ID:** BG002 **Value:** 45 **Units:** Participants ### Group **ID:** BG000 **Title:** Preterm **Description:** born at \<37 weeks of gestation ### Group **ID:** BG001 **Title:** Full Term Term **Description:** born at \>/= 37 weeks of gestation ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Value:** 2 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 3 **Category Title:** 21-25 years old #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 10 #### Measurement **Group ID:** BG002 **Value:** 13 **Category Title:** 26-30 years old #### Measurement **Group ID:** BG000 **Value:** 10 #### Measurement **Group ID:** BG001 **Value:** 8 #### Measurement **Group ID:** BG002 **Value:** 18 **Category Title:** 31-35 years old #### Measurement **Group ID:** BG000 **Value:** 4 #### Measurement **Group ID:** BG001 **Value:** 6 #### Measurement **Group ID:** BG002 **Value:** 10 **Category Title:** 36-40 years old #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 1 **Category Title:** 41-45 years old #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 20 **Group ID:** BG001 **Value:** 25 **Group ID:** BG002 **Value:** 45 **Class Title:** Parent's age ### Measure #### Measurement **Group ID:** BG000 **Value:** 10 #### Measurement **Group ID:** BG001 **Value:** 13 #### Measurement **Group ID:** BG002 **Value:** 23 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 10 #### Measurement **Group ID:** BG001 **Value:** 12 #### Measurement **Group ID:** BG002 **Value:** 22 **Category Title:** Male #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 20 **Group ID:** BG001 **Value:** 25 **Group ID:** BG002 **Value:** 45 **Class Title:** ### Measure #### Measurement **Group ID:** BG002 **Value:** 0 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 0 **Group ID:** BG001 **Value:** 0 **Group ID:** BG002 **Value:** 0 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 20 #### Measurement **Group ID:** BG001 **Value:** 25 #### Measurement **Group ID:** BG002 **Value:** 45 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 20 **Group ID:** BG001 **Value:** 25 **Group ID:** BG002 **Value:** 45 **Class Title:** Singapore **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Age, Customized **Unit of Measure:** Participants ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Race and Ethnicity were not collected from any participant. **Title:** Race and Ethnicity Not Collected **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** participants ## Results Section - More Information Module ### Certain Agreement ### Point of Contact **Email:** [email protected] **Organization:** Danone Asia Pacific Holdings **Phone:** 82281172 **Title:** Digital Project Manager ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### Outcome Measure 4 ### Outcome Measure 5 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 131 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 125 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 20 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 13 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 42 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 48 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 55 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 38 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 7 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 9 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 19 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 13 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 30 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 19 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 22 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 13 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.96 - **Upper Limit:** - **Value:** 3.62 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.82 - **Upper Limit:** - **Value:** 4.0 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.85 - **Upper Limit:** - **Value:** 3.69 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.76 - **Upper Limit:** - **Value:** 3.92 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Obtaining number of chats for sleep, stress, and infant nutrition from parents of infants (preterm and full-term) through interaction with the study chatbot **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** 8 weeks **Title:** Number of Chats on Sleep, Stress and Infant Nutrition **Type:** PRIMARY **Unit of Measure:** Chats ##### Group **Description:** born at \<37 weeks of gestation **ID:** OG000 **Title:** Preterm ##### Group **Description:** born at \>/= 37 weeks of gestation **ID:** OG001 **Title:** Full Term Term #### Outcome Measure 2 **Description:** Comparison on ratings for quality of their (parent) sleep from parents of preterm infants and parents of full-term infants **Parameter Type:** NUMBER **Population Description:** Per Protocol **Reporting Status:** POSTED **Time Frame:** 8 weeks **Title:** Ratings by Parents of Quality of Their Sleep **Type:** SECONDARY **Unit of Measure:** chat responses ##### Group **Description:** born at \<37 weeks of gestation **ID:** OG000 **Title:** Preterm ##### Group **Description:** born at \>/= 37 weeks of gestation **ID:** OG001 **Title:** Full Term Term #### Outcome Measure 3 **Description:** Comparison of particular reasons for stress caused by the baby from parents of preterm infants and parents of full-term infants **Parameter Type:** NUMBER **Population Description:** Per Protocol **Reporting Status:** POSTED **Time Frame:** 8 weeks **Title:** Particular Reasons for Stress Caused by the Baby **Type:** SECONDARY **Unit of Measure:** chat responses ##### Group **Description:** born at \<37 weeks of gestation **ID:** OG000 **Title:** Preterm ##### Group **Description:** born at \>/= 37 weeks of gestation **ID:** OG001 **Title:** Full Term Term #### Outcome Measure 4 **Description:** Comparison of number of chats on the feeding from parents of preterm infants and parents of full-term infants who give their infants formula **Parameter Type:** NUMBER **Population Description:** Per Protocol **Reporting Status:** POSTED **Time Frame:** 8 weeks **Title:** Number of Chats on Feedings Among Parents Who Give Their Infants Formula **Type:** SECONDARY **Unit of Measure:** chat responses ##### Group **Description:** born at \<37 weeks of gestation **ID:** OG000 **Title:** Preterm ##### Group **Description:** born at \>/= 37 weeks of gestation **ID:** OG001 **Title:** Full Term Term #### Outcome Measure 5 **Description:** Satisfaction scale scores for Chatbot and ClaimIt based on a scale score of 1 (very dissatisfied) to 5 (very satisfied) on the Usability of Chatbot and ClaimIt eQuestionnaire **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Per Protocol **Reporting Status:** POSTED **Time Frame:** 8 weeks **Title:** Satisfaction Scale Scores for Chatbot and ClaimIt **Type:** OTHER_PRE_SPECIFIED **Unit of Measure:** score on a scale ##### Group **Description:** born at \<37 weeks of gestation **ID:** OG000 **Title:** Preterm ##### Group **Description:** born at \>/= 37 weeks of gestation **ID:** OG001 **Title:** Full Term Term ### Participant Flow Module #### Group **Description:** born at \<37 weeks of gestation **ID:** FG000 **Title:** Preterm #### Group **Description:** born at \>/= 37 weeks of gestation **ID:** FG001 **Title:** Full Term Term #### Period **Title:** Overall Study ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 20 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 25 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 13 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 13 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 7 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 12 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT04092179 **Brief Title:** Study of Enasidenib and Venetoclax in IDH2-Mutated Blood Cancers **Official Title:** Phase Ib/II Study of IDH2 Inhibitor Enasidenib in Combination With BCL2 Inhibitor Venetoclax in Patients With IDH2-Mutated Myeloid Malignancies (ENAVEN-AML) #### Organization Study ID Info **ID:** 19-5939 #### Organization **Class:** OTHER **Full Name:** University Health Network, Toronto #### Secondary ID Infos **Domain:** Princess Margaret Cancer Centre **ID:** ENAVEN-AML **Type:** OTHER ### Status Module #### Completion Date **Date:** 2023-10-26 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2024-01-24 **Type:** ACTUAL **Last Update Submit Date:** 2024-01-23 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2023-10-26 **Type:** ACTUAL #### Start Date **Date:** 2020-11-05 **Type:** ACTUAL **Status Verified Date:** 2024-01 #### Study First Post Date **Date:** 2019-09-17 **Type:** ACTUAL **Study First Submit Date:** 2019-09-13 **Study First Submit QC Date:** 2019-09-13 **Why Stopped:** Insufficient Funding ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Celgene **Class:** INDUSTRY **Name:** AbbVie #### Lead Sponsor **Class:** OTHER **Name:** University Health Network, Toronto #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this research study is to see how safe and tolerable, and to find the highest or best dose, of an investigational combination of drugs called enasidenib and venetoclax, in patients with relapsed (the cancer has come back) or refractory (the cancer does not respond or have stopped responding to treatment) acute myeloid leukemia (AML, a type of blood cancer). This study will also see how useful the combination of enasidenib and venetoclax is in the treatment of patients with relapsed or refractory AML. **Detailed Description:** This study will have two parts: Phase 1b and Phase 2. The part that patients may participate in will depend on when they join the study. In the phase 1b portion of the study, small groups participants will receive increasing doses of venetoclax in combination with a flat dose of enadisenib until the highest dose or best dose of venetoclax that is safe and tolerable in combination with enadisenib is found. In the phase 2 portion of the study, an additional group of participants will receive the highest or best dose of venetoclax found in the Phase 1b portion of the study with a flat dose of enadisenib to see how useful the combination is in treating relapsed or refractory acute myeloid leukemia (AML). ### Conditions Module **Conditions:** - Acute Myeloid Leukemia - Relapsed Cancer - Refractory Cancer - IDH2 Gene Mutation ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SEQUENTIAL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 27 **Type:** ACTUAL **Phases:** - PHASE1 - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Enasidenib and venetoclax will be taken by mouth (orally), once a day, every day, continuously. Every 28-day period will be called a cycle. Participants will start venetoclax alone on Cycle 1 Day 1 and continue the study drug alone until Day 15. On Day 15, participants will take enasidenib and venetoclax together and will continue to take the combination of study drugs until intolerable side effects or disease worsening. **Intervention Names:** - Drug: Enasidenib - Drug: Venetoclax **Label:** Venetoclax and Enadisenib **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Venetoclax and Enadisenib **Description:** Enasidenib is a drug that blocks a protein called isocitrate dehydrogenase (IDH) 2 from working. The family of IDH proteins have been indicated in the development of leukemia. By blocking IDH2, enasidenib may help stop cancer cells from growing. It is believed that the drug may be more useful in patients with a change (mutation) in their IDH 2 protein. The IDH2 gene (substances in the body that contain instructions for the proper development and function of cells) makes IDH2 proteins. As such, only patients with IDH 2 mutated gene are eligible for this study. Enasidenib is currently approved for the treatment of IDH2 mutated AML. **Name:** Enasidenib **Other Names:** - IDHIFA **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Venetoclax and Enadisenib **Description:** Venetoclax is a drug that blocks a protein called B-cell lymphoma (BCL2) protein from working. BCL2 is a protein that helps control whether a cell lives or dies and is thought to help cancer cells to live. Blocking BCL2 is believed to help kill cancer cells. Venetoclax is currently approved for the treatment of type of blood cancer called chronic lymphocytic leukemia (CLL) who have received prior treatment. **Name:** Venetoclax **Other Names:** - VENCLEXTA **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Overall response rate (ORR) **Time Frame:** 3 years **Measure:** Dose Limiting Toxicity **Time Frame:** 28 days **Description:** Dose indicated by the mTPI decision **Measure:** Maximum tolerated dose or Recommended Phase 2 Dose **Time Frame:** 3 years **Description:** The time from the date of first response until progression, relapse, death, or last follow-up. **Measure:** Duration of Response **Time Frame:** 3 years #### Secondary Outcomes **Description:** The first day of treatment until death or last contact. **Measure:** Overall Survival **Time Frame:** 3 years **Description:** The number of days from the first day of treatment to the date of earliest evidence of relapse or progression, subsequent treatment other than stem cell transplant while in response, or death, or date of last disease assessment. **Measure:** Event Free Survival **Time Frame:** 3 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Age ≥ 18 years * Eastern Cooperative Oncology Group (ECOG) performance score of ≤2 * IDH2 (R140 or IDH R172) mutated AML disease status as determined by local laboratory * Relapsed and/or refractory acute myeloid leukemia (AML). Treatment-naïve patients who are not eligible for standard induction chemotherapy or high-risk myelodysplastic syndromes (MDS) or myeloproliferative neoplasms (MPN) may also be eligible. * Adequate hepatic function * Adequate renal function * Willing and able to provide informed consent * In the absence of rapidly proliferative disease, the interval from prior treatment to time of initiation will be at least 7 days for cytotoxic and non-cytotoxic (immunotherapy) agents Exclusion Criteria: * Known allergy or hypersensitivity to enasidenib or venetoclax * Previously received either an IDH2 inhibitor or BCL2 inhibitor * With any uncontrolled clinically significant medical conditions * The use of other chemotherapeutic agents or anti-leukemic agents, radiotherapy or other investigational therapy is not permitted during study with exceptions * Receiving concomitant treatment with strong cytochrome P450 2A (CYP3A4) inhibitors within 3 days of start of study therapy * Receiving concomitant strong CYP3A inducers (avasimibe, carbamazepine, phenytoin, rifampin, rifabutin, St. John's Wort) within 3 days of start of study therapy. * Taking the following sensitive CYP substrate medications that have a narrow therapeutic range are excluded from the study unless the subject can be transferred to other medications at least 5 half-lives prior to the start of study treatment: paclitaxel and docetaxel (CYP2C8), phenytoin (CYP2C9), S-mephenytoin (CYP2C19), thioridazine (CYP2D6), theophylline, and tizanidine (CYP1A2) * Active graft-versus-host-disease (GVHD) status post stem cell transplant * Severe gastrointestinal or metabolic condition which could interfere with the absorption of oral study medications * Concurrent active malignancy under treatment * Administration or consumption of any of the following within 3 days prior to first dose of study drug: grapefruit or grapefruits products, Seville oranges (including marmalade containing Seville oranges) and start fruit * Heart-rate corrected QT (QTc) interval ≥480 msec (Fridericia's formula) except for underlying right-bundle branch block (RBBB). * Positive for HIV * Subject has an unacceptable white blood cell count * Positive urine pregnancy test, * Participants who not willing to maintain adequate contraception **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Edmonton **Country:** Canada **Facility:** University of Alberta Hospital **State:** Alberta **Zip:** T6G 2B7 **Location 2:** **City:** Toronto **Country:** Canada **Facility:** Princess Margaret Cancer Centre **State:** Ontario **Zip:** M5G 1Z5 #### Overall Officials **Official 1:** **Affiliation:** Princess Margaret Cancer Centre **Name:** Steven Chan, M.D. **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC15 - Name: Blood and Lymph Conditions - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M10945 - Name: Leukemia - Relevance: LOW - As Found: Unknown - ID: M10955 - Name: Leukemia, Myeloid - Relevance: LOW - As Found: Unknown - ID: M18127 - Name: Leukemia, Myeloid, Acute - Relevance: LOW - As Found: Unknown - ID: M21314 - Name: Hematologic Neoplasms - Relevance: LOW - As Found: Unknown - ID: T3995 - Name: Myeloid Leukemia - Relevance: HIGH - As Found: Myeloid Leukemia - ID: T182 - Name: Acute Myeloid Leukemia - Relevance: HIGH - As Found: Acute Myeloid Leukemia - ID: T188 - Name: Acute Non Lymphoblastic Leukemia - Relevance: LOW - As Found: Unknown - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Ancestors - ID: D000000970 - Term: Antineoplastic Agents ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M249656 - Name: Venetoclax - Relevance: HIGH - As Found: Third ### Intervention Browse Module - Meshes - ID: C000579720 - Term: Venetoclax ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06368479 **Brief Title:** Diagnostic Sensitivity and Specificity of iStatis HBsAg Test at the Point-Of-Care Site Settings **Official Title:** A Prospective Cross-Sectional Study to Evaluate Diagnostic Sensitivity and Specificity of iStatis HBsAg Test at the Point-Of-Care Site Settings #### Organization Study ID Info **ID:** CLS-016B #### Organization **Class:** INDUSTRY **Full Name:** bioLytical Laboratories ### Status Module #### Completion Date **Date:** 2024-07-01 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-05-06 **Type:** ACTUAL **Last Update Submit Date:** 2024-05-02 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-07-01 **Type:** ESTIMATED #### Start Date **Date:** 2024-03-20 **Type:** ACTUAL **Status Verified Date:** 2024-05 #### Study First Post Date **Date:** 2024-04-16 **Type:** ACTUAL **Study First Submit Date:** 2024-04-10 **Study First Submit QC Date:** 2024-04-11 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** bioLytical Laboratories #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** A prospective cross-sectional study in which surgically non-invasive sample-taking is done only for the purpose of testing the samples on iStatis HBsAg Test at the point of care. **Detailed Description:** Participants are prospctively recruited and enrolled to participate in the study. The Capillary (fingerstick) whole blood, and venous whole blood samples (in serum separating tube (SST) and Ethylenediaminetetraacetic acid (EDTA) tube) are collected by a healthcare professional. Serum and plasma samples will be extracted through laboratory processing of the collected serum separating tube and Ethylenediaminetetraacetic acid VWB, respectively. The collected samples of capillary will be tested on iStatis HBsAg Test. The collected Ethylenediaminetetraacetic acid and serum separating tube VWB will be shipped to the central laboratory to appropriately process and extract serum and plasma samples. Ethylenediaminetetraacetic acid VWB, plasma and serum will be tested on iStatis HBsAg Test in the laboratory. An aliquot of the obtained plasma sample will be further tested for reference and confirmatory testing. The results from iStatis HBsAg Test results will not be used for participant management decisions. ### Conditions Module **Conditions:** - Hepatitis B - Hepatitis B Infection **Keywords:** - Hepatitis B rapid test - Hepatitis B infection - Hepatitis B iStatis test - Hepatitis B point of care test ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER **Primary Purpose:** SCREENING #### Enrollment Info **Count:** 4400 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Capillary (fingerstick) blood samples are tested using iStatis HBsAg Test onsite. **Intervention Names:** - Device: iStatis HBsAg Test **Label:** iStatis Testing Onsite **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Venous whole blood collected in Ethylenediaminetetraacetic acid tube, plasma and serum samples are tested using iStatis HBsAg Test **Intervention Names:** - Device: iStatis HBsAg Test **Label:** iStatis Testing In Lab **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Either serum or plasma sample will be tested on Abbott Architect HBsAg Qualitative Confirmatory Assay and ADVIA Centaur (depending on the test result outcome). **Label:** Reference Test **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - iStatis Testing In Lab - iStatis Testing Onsite **Description:** Capillary (fingerstick) blood, EDTA venous whole blood, serum and plasma samples to be tested on the iStatis HBsAg Test at the point of care setting. **Name:** iStatis HBsAg Test **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** To evaluate the device performance i.e. diagnostic sensitivity and diagnostic specificity of the iStatis Hepatitis B surface Antigen (HBsAg) Test compared to comparator assay. **Measure:** iStatis Performance **Time Frame:** 3 Months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Participants/subjects (males, females, and pregnant women) getting tested for Hepatitis B virus for one or more of the following reasons: * at risk for Hepatitis B virus * having signs and symptoms indicative for Hepatitis B virus * Routine testing Unknown or other reasons will be captured * Participants/subjects of 18 years or older and, who are able to give/sign the informed consent. Exclusion Criteria: * Participant younger than 18 years old * Participants unable to provide written informed consent **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Ana Subramanian **Phone:** 16042046784 **Role:** CONTACT #### Locations **Location 1:** **City:** Hillcrest **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Cherie Cawood - **Phone:** +27828202955 - **Role:** CONTACT **Country:** South Africa **Facility:** Epicentre Health Research **State:** Kwazulu Natal **Status:** RECRUITING **Zip:** 3650 ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007239 - Term: Infections - ID: D000008107 - Term: Liver Diseases - ID: D000004066 - Term: Digestive System Diseases - ID: D000006525 - Term: Hepatitis, Viral, Human - ID: D000014777 - Term: Virus Diseases - ID: D000004769 - Term: Enterovirus Infections - ID: D000010850 - Term: Picornaviridae Infections - ID: D000012327 - Term: RNA Virus Infections - ID: D000086982 - Term: Blood-Borne Infections - ID: D000003141 - Term: Communicable Diseases - ID: D000018347 - Term: Hepadnaviridae Infections - ID: D000004266 - Term: DNA Virus Infections ### Condition Browse Module - Browse Branches - Abbrev: BC01 - Name: Infections - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC20 - Name: Immune System Diseases - Abbrev: BC06 - Name: Digestive System Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M10283 - Name: Infections - Relevance: LOW - As Found: Unknown - ID: M6368 - Name: Communicable Diseases - Relevance: LOW - As Found: Unknown - ID: M10018 - Name: Hypersensitivity - Relevance: LOW - As Found: Unknown - ID: M9592 - Name: Hepatitis A - Relevance: HIGH - As Found: Hepatitis - ID: M9595 - Name: Hepatitis B - Relevance: HIGH - As Found: Hepatitis B - ID: M9591 - Name: Hepatitis - Relevance: HIGH - As Found: Hepatitis - ID: M11107 - Name: Liver Diseases - Relevance: LOW - As Found: Unknown - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M9610 - Name: Hepatitis, Viral, Human - Relevance: LOW - As Found: Unknown - ID: M17522 - Name: Virus Diseases - Relevance: LOW - As Found: Unknown - ID: M7930 - Name: Enterovirus Infections - Relevance: LOW - As Found: Unknown - ID: M13745 - Name: Picornaviridae Infections - Relevance: LOW - As Found: Unknown - ID: M15149 - Name: RNA Virus Infections - Relevance: LOW - As Found: Unknown - ID: M2593 - Name: Blood-Borne Infections - Relevance: LOW - As Found: Unknown - ID: M20487 - Name: Hepadnaviridae Infections - Relevance: LOW - As Found: Unknown - ID: M7442 - Name: DNA Virus Infections - Relevance: LOW - As Found: Unknown - ID: T4202 - Name: Oculocerebral Syndrome With Hypopigmentation - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006506 - Term: Hepatitis A - ID: D000006509 - Term: Hepatitis B - ID: D000006505 - Term: Hepatitis ### Intervention Browse Module - Browse Branches - Abbrev: AnCoag - Name: Anticoagulants - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M7662 - Name: Edetic Acid - Relevance: LOW - As Found: Unknown - ID: M7543 - Name: Pentetic Acid - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01323179 **Brief Title:** Preopioid Versus Nonopioid in Total Knee Arthroplasty (TKA) **Official Title:** Acute Pain After Fast-track Total Knee Arthroplasty: Preoperative Opioid Versus Non-opioid #### Organization Study ID Info **ID:** 11123111 #### Organization **Class:** OTHER **Full Name:** Hvidovre University Hospital ### Status Module #### Completion Date **Date:** 2013-02 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2013-06-05 **Type:** ESTIMATED **Last Update Submit Date:** 2013-06-04 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2013-02 **Type:** ACTUAL #### Start Date **Date:** 2011-03 **Status Verified Date:** 2013-06 #### Study First Post Date **Date:** 2011-03-25 **Type:** ESTIMATED **Study First Submit Date:** 2011-03-24 **Study First Submit QC Date:** 2011-03-24 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Hvidovre University Hospital #### Responsible Party **Investigator Affiliation:** Hvidovre University Hospital **Investigator Full Name:** Troels Haxholdt Lunn **Investigator Title:** MD **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Observational study evaluating pain response after total knee arthroplasty (TKA) in patients taking strong, weak or no opioids preoperatively. ### Conditions Module **Conditions:** - Postoperative Pain **Keywords:** - Postoperative pain - Postoperative opioid requirements ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 140 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients taking strong opioids preoperatively **Label:** Strong opioids #### Arm Group 2 **Description:** Patients taking weak opioids preoperatively **Label:** Weak opioids #### Arm Group 3 **Description:** Patients not taking opioids preoperatively **Label:** Opioid native ### Outcomes Module #### Primary Outcomes **Description:** Pain during ambulation and at rest **Measure:** Pain **Time Frame:** 24 hours #### Secondary Outcomes **Description:** Pain during ambulation and at rest **Measure:** Pain **Time Frame:** 7 days **Measure:** Opioid consumption **Time Frame:** 7 days ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: Patients operated with elective, primary, unilateral total knee arthroplasty (TKA) (taking strong or weak opioid daily for minimum 4 weeks or opioid naive patients) Exclusion Criteria: * Bilateral / revision arthroplasty * Disease affection central or peripheral nerve function * Alcohol and medical abuse * Daily use of glucocorticoids * Malignancy * BMI \> 40 * Dementia or other cognitive dysfunction * Treatment of anxiety or depression **Minimum Age:** 18 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Patients operated with elective, primary, unilateral total knee artroplasty (TKA). ### Contacts Locations Module #### Locations **Location 1:** **City:** Copenhagen **Country:** Denmark **Facility:** Dep. of Anesthesiology, Hvidovre University Hospital **State:** Hvidovre **Zip:** 2650 **Location 2:** **City:** Hellerup **Country:** Denmark **Facility:** Dep. of ortopedic surgery, Gentofte Hospital **Zip:** 2900 **Location 3:** **City:** Holstebro **Country:** Denmark **Facility:** Dep. of ortopedic surgery, Regionshospitalet Holstebro **Zip:** 7500 **Location 4:** **City:** Vejle **Country:** Denmark **Facility:** Dep. of ortopedic surgery, Vejle Sygehus **Zip:** 7100 #### Overall Officials **Official 1:** **Affiliation:** Dep. of Anesthesiology, Hvidovre University Hospital **Name:** Troels H Lunn, MD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000011183 - Term: Postoperative Complications - ID: D000010335 - Term: Pathologic Processes - ID: D000010146 - Term: Pain - ID: D000009461 - Term: Neurologic Manifestations ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M13069 - Name: Pain, Postoperative - Relevance: HIGH - As Found: Postoperative Pain - ID: M29525 - Name: Acute Pain - Relevance: LOW - As Found: Unknown - ID: M13066 - Name: Pain - Relevance: LOW - As Found: Unknown - ID: M14065 - Name: Postoperative Complications - Relevance: LOW - As Found: Unknown - ID: M12404 - Name: Neurologic Manifestations - Relevance: LOW - As Found: Unknown - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000010149 - Term: Pain, Postoperative ### Intervention Browse Module - Browse Branches - Abbrev: Analg - Name: Analgesics - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M4033 - Name: Analgesics, Opioid - Relevance: LOW - As Found: Unknown - ID: M20786 - Name: Analgesics, Non-Narcotic - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01499979 **Brief Title:** Hypothermic Perfusion During Hemihepatectomy **Official Title:** In Situ Hypothermic Perfusion During Right Hemihepatectomy #### Organization Study ID Info **ID:** 2011_214 #### Organization **Class:** OTHER **Full Name:** Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) #### Secondary ID Infos **Domain:** Centrale Commissie Mensgebonden Onderzoek (CCMO) **ID:** NL37241.018.11 **Type:** OTHER ### Status Module #### Completion Date **Date:** 2015-08 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2016-01-26 **Type:** ESTIMATED **Last Update Submit Date:** 2016-01-25 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2015-05 **Type:** ACTUAL #### Start Date **Date:** 2012-02 **Status Verified Date:** 2016-01 #### Study First Post Date **Date:** 2011-12-26 **Type:** ESTIMATED **Study First Submit Date:** 2011-12-20 **Study First Submit QC Date:** 2011-12-23 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) #### Responsible Party **Investigator Affiliation:** Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) **Investigator Full Name:** Megan J. Reiniers **Investigator Title:** PhD student **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Rationale Currently, hepatic resection is often the only curative treatment for primary or secondary hepatic malignancies and is also frequently performed in patients with benign liver tumors to prevent malignant transformation and/or alleviate symptoms. Liver resections are nowadays associated with low mortality and acceptable morbidity. As result of that, an increasing number of patients is currently under consideration for resection of more complex or large tumors, thus requiring extensive resection procedures. Application of vascular exclusion (i.e., clamping of the portal vein and hepatic artery) during such procedures reduces blood loss, which is one of the most important factors affecting peri-operative outcomes. However, vascular exclusion leads to ischemia-reperfusion (I/R) injury as an inevitable side-effect, which adversely impacts postoperative liver function and regeneration. Additional cooling of the liver by means of hypothermic perfusion is expected to further reduce intraoperative blood loss, as well as to protect the liver from I/R injury. Therefore, the aim of this pilot study is to cool the future remnant liver (FRL) in situ during right hemihepatectomy under vascular exclusion. Consequently, an overall improvement in postoperative outcomes is expected due to a decrease in intraoperative blood loss, reduced parenchymal damage, and a better ability of the liver remnant to regenerate. Objective To reduce intraoperative blood loss and enhance tolerance of the FRL to I/R injury during right hemihepatectomy under vascular exclusion by means of in situ hypothermic perfusion with retrograde outflow (R-IHP) of the FRL. Study design The study is designed as a prospective randomized pilot study in 18 patients (9 interventions and 9 controls) to assess the effects of the proposed intervention. Additionally, 4 patients will be included separately for assessment of the intervention's feasibility prior to randomized inclusion. Study population Eligible patients for participation in this study are those planned to undergo right hemihepatectomy under vascular inflow occlusion because of a malignant or benign liver tumor, and who do not suffer from any hepatic co-morbidity that might influence postoperative outcomes (i.e., severe steatosis, cholestasis, cirrhosis, or hepatitis B/C infection). Intervention During right hemihepatectomy, the FRL of patients allocated to the intervention group will be perfused with a chilled perfusion solution (i.e., lactated Ringer's solution). ### Conditions Module **Conditions:** - Hepatic Ischemia-reperfusion Injury **Keywords:** - Ischemia-reperfusion injury - In situ hypothermic perfusion - In situ hypothermic preservation ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - PARTICIPANT **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 22 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients that will receive intermittent vascular inflow occlusion, the standard method for vascular occlusion at our institution, during liver resection. **Label:** Vascular inflow occlusion **Type:** NO_INTERVENTION #### Arm Group 2 **Description:** Patients will receive in situ hypothermic perfusion of the future remnant liver during liver resection. **Intervention Names:** - Procedure: In situ hypothermic perfusion **Label:** Hypothermic perfusion **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Hypothermic perfusion **Description:** In situ perfusion of the future remnant liver with chilled lactated Ringer's solution during liver resection. **Name:** In situ hypothermic perfusion **Other Names:** - In situ hypothermic preservation **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Description:** Hepatocellular damage expressed as an postoperative increase in transaminases (i.e., AST and ALT). **Measure:** Postoperative hepatocellular damage **Time Frame:** 5 days postoperatively #### Secondary Outcomes **Description:** Blood loss during surgery **Measure:** Intraoperative blood loss **Time Frame:** 2-3 hours **Description:** Incidence of surgery-related complications **Measure:** Postoperative complications **Time Frame:** 5 days postoperatively **Description:** Regeneration of liver function (measured via hepatobiliary scintigraphy) and -volume (measured via CT volumetry). **Measure:** Regeneration of liver function and volume **Time Frame:** 3 days ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patients scheduled for right hemihepatectomy under vascular inflow occlusion for a malignant or benign hepatic tumor * Diagnostic exclusion of hepatic co-morbidity, that is: * Cirrhosis, * Severe steatosis (≥ 30%), * Cholestasis, and * Hepatitis B/C infection * Age ≥ 18 years * Signed informed consent obtained prior to any study-specific procedure * ASA classification I-III Exclusion Criteria: * Patients diagnosed with any of the hepatic co-morbidities listed under point 2 of the inclusion criteria * Age \< 18 years * BMI \> 35 kg/m2 * ASA classification IV/V * Patient is scheduled for a combined surgical procedure (e.g., bile duct resection, gastrointestinal procedures) * Patient underwent liver resection ≤ 1 year prior to scheduled surgery * Emergency operations * Pregnancy or breast feeding **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Amsterdam **Country:** Netherlands **Facility:** Academic Medical Center (AMC) **State:** Noord-Holland **Zip:** 1105 AZ #### Overall Officials **Official 1:** **Affiliation:** Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) **Name:** Prof. Thomas M. van Gulik, MD, PhD **Role:** STUDY_CHAIR **Official 2:** **Affiliation:** Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) **Name:** Megan J. Reiniers, MSc **Role:** PRINCIPAL_INVESTIGATOR **Official 3:** **Affiliation:** Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) **Name:** Rowan F. van Golen, MSc **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ### References Module #### References **Citation:** Azoulay D, Eshkenazy R, Andreani P, Castaing D, Adam R, Ichai P, Naili S, Vinet E, Saliba F, Lemoine A, Gillon MC, Bismuth H. In situ hypothermic perfusion of the liver versus standard total vascular exclusion for complex liver resection. Ann Surg. 2005 Feb;241(2):277-85. doi: 10.1097/01.sla.0000152017.62778.2f. **PMID:** 15650638 **Citation:** Dinant S, van Veen SQ, Roseboom HJ, van Vliet AK, van Gulik TM. Liver protection by hypothermic perfusion at different temperatures during total vascular exclusion. Liver Int. 2006 May;26(4):486-93. doi: 10.1111/j.1478-3231.2006.01248.x. **PMID:** 16629653 **Citation:** Verhoef C, de Wilt JH, Brunstein F, Marinelli AW, van Etten B, Vermaas M, Guetens G, de Boeck G, de Bruijn EA, Eggermont AM. Isolated hypoxic hepatic perfusion with retrograde outflow in patients with irresectable liver metastases; a new simplified technique in isolated hepatic perfusion. Ann Surg Oncol. 2008 May;15(5):1367-74. doi: 10.1245/s10434-007-9714-z. Epub 2008 Feb 1. **PMID:** 18239976 **Citation:** Reiniers MJ, van Golen RF, Heger M, Mearadji B, Bennink RJ, Verheij J, van Gulik TM. In situ hypothermic perfusion with retrograde outflow during right hemihepatectomy: first experiences with a new technique. J Am Coll Surg. 2014 Jan;218(1):e7-16. doi: 10.1016/j.jamcollsurg.2013.09.013. Epub 2013 Nov 6. No abstract available. **PMID:** 24210146 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010335 - Term: Pathologic Processes - ID: D000014652 - Term: Vascular Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000011183 - Term: Postoperative Complications - ID: D000001832 - Term: Body Temperature Changes ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC14 - Name: Heart and Blood Diseases ### Condition Browse Module - Browse Leaves - ID: M10085 - Name: Hypothermia - Relevance: HIGH - As Found: Hypothermic - ID: M10543 - Name: Ischemia - Relevance: HIGH - As Found: Ischemia - ID: M18099 - Name: Reperfusion Injury - Relevance: HIGH - As Found: Reperfusion Injury - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown - ID: M14065 - Name: Postoperative Complications - Relevance: LOW - As Found: Unknown - ID: M5111 - Name: Body Temperature Changes - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000015427 - Term: Reperfusion Injury - ID: D000007511 - Term: Ischemia - ID: D000007035 - Term: Hypothermia ### Intervention Browse Module - Browse Branches - Abbrev: PhSol - Name: Pharmaceutical Solutions - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Hemat - Name: Hematinics ### Intervention Browse Module - Browse Leaves - ID: M21860 - Name: Pharmaceutical Solutions - Relevance: LOW - As Found: Unknown - ID: M11110 - Name: Liver Extracts - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04090879 **Brief Title:** Low Nicotine Content Cigarettes in Vulnerable Populations: Affective Disorders **Official Title:** Low Nicotine Content Cigarettes in Vulnerable Populations: Affective Disorders #### Organization Study ID Info **ID:** CHRMS19-0129 #### Organization **Class:** OTHER **Full Name:** Brown University #### Secondary ID Infos **ID:** U54DA036114-06 **Link:** https://reporter.nih.gov/quickSearch/U54DA036114-06 **Type:** NIH ### Status Module #### Completion Date **Date:** 2024-01 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-08-18 **Type:** ACTUAL **Last Update Submit Date:** 2023-08-16 **Overall Status:** ACTIVE_NOT_RECRUITING #### Primary Completion Date **Date:** 2024-01 **Type:** ESTIMATED #### Start Date **Date:** 2019-09-18 **Type:** ACTUAL **Status Verified Date:** 2023-08 #### Study First Post Date **Date:** 2019-09-16 **Type:** ACTUAL **Study First Submit Date:** 2019-09-13 **Study First Submit QC Date:** 2019-09-13 ### Sponsor Collaborators Module #### Collaborators **Class:** FED **Name:** Food and Drug Administration (FDA) **Class:** NIH **Name:** National Institute on Drug Abuse (NIDA) **Class:** OTHER **Name:** University of Vermont #### Lead Sponsor **Class:** OTHER **Name:** Brown University #### Responsible Party **Investigator Affiliation:** Brown University **Investigator Full Name:** Jennifer Tidey **Investigator Title:** Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** While the prevalence of smoking in the United States general population has declined over the past 50 years, there has been little to no decline among people with mental health conditions. Affective Disorders (ADs) are the most common mental health conditions in the US, and over 40% of people with ADs are current smokers. A national policy of reducing the nicotine content of cigarettes has the potential to reduce tobacco use, dependence, and related adverse health outcomes. Controlled trials in psychiatrically-stable smokers have shown that reducing the nicotine content in cigarettes can reduce cigarettes per day (CPD), dependence and tobacco toxicant exposure, with few adverse consequences. The goal of the proposed trial is to experimentally model whether increasing the availability and appeal of an alternative, non-combusted source of nicotine (e-cigarettes) moderates the effect of altering the nicotine in cigarettes in smokers with ADs. Additionally, investigators will test whether allowing participants to personalize the flavor of the e-liquid alters any moderating effects their availability may have on tobacco cigarette smoking. Daily smokers with current ADs will be recruited at Brown University and the University of Vermont. Investigators will study two research cigarettes referred to here as Research Cigarette 1 (RC1) and Research Cigarette 2 (RC2). One of these cigarettes will be a normal nicotine content cigarette and the other will be a reduced nicotine content cigarette. Investigators will study two e-cigarette conditions referred to here as E-Cigarette Condition 1 (EC1) and E-Cigarette Condition 2 (EC2). Both e-cigarette conditions will involve the same commercially available devices and same nicotine-containing e-liquid, but in one condition that e-liquid will be available only in tobacco flavor while in the other condition that e-liquid will be available in multiple flavors from which participants can choose based on personal taste preference. Participants will be assigned to one of the following four study conditions: (1) RC1 only; (2) RC2 only; (3) RC2 + EC1; (4) RC2 + EC2. Participants will be asked to use only their assigned study products for 16 weeks. Outcome measures include total CPD, cigarette demand assessed by behavioral economics-based purchase tasks, craving, withdrawal, psychiatric symptoms, breath carbon monoxide (CO), biomarkers of tobacco toxicant exposure, brain function and structure, and airway inflammation (fractional nitric oxide concentration in exhaled breath \[FeNO\]). ### Conditions Module **Conditions:** - Tobacco Use Disorder **Keywords:** - Biomarkers of Exposure - Compensatory Smoking - Nicotine Dependence - Reduced Nicotine Cigarettes - Affective Disorders - Tobacco Withdrawal - Vulnerable Populations - E-Cigarettes ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** Randomized parallel groups research design ##### Masking Info **Masking:** TRIPLE **Masking Description:** Double blind dosing of tobacco cigarette nicotine levels. **Who Masked:** - PARTICIPANT - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** BASIC_SCIENCE #### Enrollment Info **Count:** 232 **Type:** ESTIMATED **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Research Cigarettes #1 **Intervention Names:** - Other: Either normal nicotine content cigarettes (15.8mg/g) or reduced nicotine content cigarettes (0.4mg/g) **Label:** RC 1 only **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Research Cigarettes #2 **Intervention Names:** - Other: Either normal nicotine content cigarettes (15.8mg/g) or reduced nicotine content cigarettes (0.4mg/g) **Label:** RC 2 only **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Research Cigarettes #2 plus E-cigarettes #1 (participants receive tobacco flavor only) **Intervention Names:** - Other: Either normal nicotine content cigarettes (15.8mg/g) or reduced nicotine content cigarettes (0.4mg/g) - Other: E-Cigarettes **Label:** RC 2 + EC 1 **Type:** EXPERIMENTAL #### Arm Group 4 **Description:** Research Cigarettes #2 plus E-cigarettes #2 (participants can choose among varying flavors) **Intervention Names:** - Other: Either normal nicotine content cigarettes (15.8mg/g) or reduced nicotine content cigarettes (0.4mg/g) - Other: E-Cigarettes **Label:** RC 2 + EC 2 **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - RC 1 only - RC 2 + EC 1 - RC 2 + EC 2 - RC 2 only **Description:** 1) Altering the nicotine content of the tobacco research cigarette **Name:** Either normal nicotine content cigarettes (15.8mg/g) or reduced nicotine content cigarettes (0.4mg/g) **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - RC 2 + EC 1 - RC 2 + EC 2 **Description:** 1) Altering the availability of e-cigarettes; 2) Altering option to personalize the e-liquid in the e-cig condition **Name:** E-Cigarettes **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Measure:** Cigarettes Per Day **Time Frame:** 16 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria * Between 21 years and 70 years old * Must have current diagnosis of an Affective Disorder. Exclusion Criteria * Being without an Affective Disorder * Younger than 21 years old * Older than 70 years old **Healthy Volunteers:** True **Maximum Age:** 70 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Providence **Country:** United States **Facility:** Brown University **State:** Rhode Island **Zip:** 02912 **Location 2:** **City:** Burlington **Country:** United States **Facility:** University of Vermont **State:** Vermont **Zip:** 05401 #### Overall Officials **Official 1:** **Affiliation:** Brown University **Name:** Jennifer W Tidey, Ph.D. **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** YES ## Derived Section ### Condition Browse Module - Ancestors - ID: D000019966 - Term: Substance-Related Disorders - ID: D000064419 - Term: Chemically-Induced Disorders - ID: D000001523 - Term: Mental Disorders ### Condition Browse Module - Browse Branches - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions - Abbrev: BC25 - Name: Substance Related Disorders ### Condition Browse Module - Browse Leaves - ID: M21835 - Name: Mood Disorders - Relevance: HIGH - As Found: Affective Disorder - ID: M16785 - Name: Tobacco Use Disorder - Relevance: HIGH - As Found: Tobacco Use Disorder - ID: M21837 - Name: Substance-Related Disorders - Relevance: LOW - As Found: Unknown - ID: M30302 - Name: Chemically-Induced Disorders - Relevance: LOW - As Found: Unknown - ID: M4815 - Name: Mental Disorders - Relevance: LOW - As Found: Unknown - ID: M14473 - Name: Psychotic Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000014029 - Term: Tobacco Use Disorder - ID: D000019964 - Term: Mood Disorders ### Intervention Browse Module - Ancestors - ID: D000005731 - Term: Ganglionic Stimulants - ID: D000001337 - Term: Autonomic Agents - ID: D000018373 - Term: Peripheral Nervous System Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018722 - Term: Nicotinic Agonists - ID: D000018679 - Term: Cholinergic Agonists - ID: D000018678 - Term: Cholinergic Agents - ID: D000018377 - Term: Neurotransmitter Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: CNSSti - Name: Central Nervous System Stimulants ### Intervention Browse Module - Browse Leaves - ID: M12478 - Name: Nicotine - Relevance: HIGH - As Found: Prior to - ID: M4029 - Name: Central Nervous System Stimulants - Relevance: LOW - As Found: Unknown - ID: M20796 - Name: Nicotinic Agonists - Relevance: LOW - As Found: Unknown - ID: M20758 - Name: Cholinergic Agents - Relevance: LOW - As Found: Unknown - ID: M20504 - Name: Neurotransmitter Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000009538 - Term: Nicotine ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03470779 **Brief Title:** Impact of Combined Psychotherapy and Physiotherapy Group Treatment Program for Survivors of Torture **Official Title:** A Randomized Controlled Wait-list Pilot Study Examining the Feasibility and Impact of a Combined Psychotherapy and Physiotherapy Group Treatment Program for Survivors of Torture Incarcerated in an Adult Prison in Kurdistan, Iraq #### Organization Study ID Info **ID:** STU00206726 #### Organization **Class:** OTHER **Full Name:** Northwestern University ### Status Module #### Completion Date **Date:** 2019-01-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2019-03-13 **Type:** ACTUAL **Last Update Submit Date:** 2019-03-11 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2018-11-15 **Type:** ACTUAL #### Start Date **Date:** 2018-04-19 **Type:** ACTUAL **Status Verified Date:** 2019-03 #### Study First Post Date **Date:** 2018-03-20 **Type:** ACTUAL **Study First Submit Date:** 2018-03-13 **Study First Submit QC Date:** 2018-03-13 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Northwestern University #### Responsible Party **Investigator Affiliation:** Northwestern University **Investigator Full Name:** Jeff Hartman **Investigator Title:** Assistant professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The aim of this study is to assess the impact and feasibility of an interdisciplinary group treatment approach, involving psychotherapy and physiotherapy, with survivors of torture that are incarcerated in a prison in Kurdistan, Iraq. The primary aim is to develop initial estimates of treatment effects on symptoms and poor functioning consistent with centralized pain and post-traumatic stress disorder, anxiety, and/or depression. The secondary aim is to assess the feasibility of studying this interdisciplinary treatment program in which local Kurdish psychotherapists and physiotherapists provide a 10-week intervention in a prison, in the Kurdish Sorani language, and to Kurdish participants that present with mental health symptoms, physical complaints, and poor functioning **Detailed Description:** The aim of this randomized wait-list controlled pilot study is to assess the impact and feasibility of an interdisciplinary group treatment approach, involving psychotherapy and physiotherapy, with survivors of torture that are incarcerated in a prison in Kurdistan, Iraq. The primary aim is to develop initial estimates of treatment effects on symptoms and poor functioning consistent with centralized pain and post-traumatic stress disorder, anxiety, and/or depression. It is hypothesized that participants in the study will present with statistically significant improvements in some to all measures of symptoms and function. The secondary aim is to assess the feasibility of studying this interdisciplinary treatment program in which local Kurdish psychotherapists and physiotherapists provide a 10-week intervention in a prison, in the Kurdish Sorani language, and to Kurdish participants that present with mental health symptoms, physical complaints, and poor functioning ### Conditions Module **Conditions:** - Pain Syndrome - Disability Physical ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** The study design is a randomized, wait-list controlled pilot study. A parallel group study design will be used to compare a treatment group and a wait-list control group. This study design will be effective for addressing the gap in literature by examining both the initial treatment estimates related to health and functioning and the feasibility of performing a similar study on a larger scale. ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 30 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants randomly assigned to the treatment group will participate in an interdisciplinary treatment program in which local Kurdish psychotherapists and physiotherapists provide a 10-week intervention program. Treatment will consist of group physiotherapy and group psychotherapy **Intervention Names:** - Other: Physiotherapy - Other: Psychotherapy **Label:** Treatment group **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants randomly assigned to the wait-list group will not receive treatment but will participate in outcome data collection for comparison purposes. **Label:** Wait-list group **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Treatment group **Description:** Treatment group participants will participate in weekly group physiotherapy and psychotherapy sessions as part of the standard program procedure. **Name:** Physiotherapy **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - Treatment group **Description:** Treatment group participants will participate in weekly group physiotherapy and psychotherapy sessions as part of the standard program procedure. **Name:** Psychotherapy **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** The HSCL-25 is a 25-question symptom inventory which measures symptoms of anxiety and depression. **Measure:** Hopkins Symptom Checklist-25 **Time Frame:** 10 weeks #### Secondary Outcomes **Description:** This useful questionnaire can be used to quantify activity limitation and measure functional outcome for patients with any orthopaedic condition. **Measure:** Patient Specific Functional Scale **Time Frame:** 10-weeks **Description:** The Pittsburgh Sleep Quality Index (PSQI) is an effective instrument used to measure the quality and patterns of sleep in adults. **Measure:** Pittsburgh Sleep Quality Index **Time Frame:** 10-weeks **Description:** The self-report measure Patient Global Impression of Change (PGIC) reflects a patient's belief about the efficacy of treatment **Measure:** Patient's Global Impression of Change Scale **Time Frame:** 10-weeks **Description:** The General Self-Efficacy Scale is a 10-item psychometric scale that is designed to assess optimistic self-beliefs to cope with a variety of difficult demands in life. **Measure:** General Self Efficacy Scale **Time Frame:** 10-weeks **Description:** This tool was published with the purpose of providing a single self-report instrument that identified symptoms associated with central sensitization and the quantified the degree of those symptoms. **Measure:** Central Sensitization Inventory **Time Frame:** 10-weeks **Description:** The Harvard Trauma Questionnaire (HTQ) is a checklist that inquires about a variety of trauma events, as well as the emotional symptoms considered to be uniquely associated with trauma. **Measure:** Harvard Trauma Questionnaire Part 4 **Time Frame:** 10 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Age equal to or greater than 18 years * Incarcerated in the reformatory where research is conducted * Native speaker of Sorani, a Kurdish language * History of torture is reported and documented during the psychotherapy intake * Presents with symptoms consistent with post-traumatic stress disorder, anxiety, and/or depression evident by a total score of greater than or equal to 1.75 on Hopkins Symptoms Checklist-25 and/or by a total score greater than or equal to 2.5 on the Harvard Trauma Questionnaire Part 4 * Presents with symptoms consistent with centralized pain evident by a score of greater than or equal to 40 on the Central Sensitization Inventory Part A. Exclusion Criteria: * Reports he will not remain in the current reformatory for at least 6 months from the onset of the study * Is unable to make the time commitment required to participate * Presents with symptoms consistent with a psychiatric condition which makes participation in the study unsafe for himself or others, based on the evaluation by the treating psychotherapist * Presents with a high risk to self or others which makes participation in the study unsafe for himself or others, based on evaluation by the treating psychotherapist * Participation is deemed unsafe due to a severe medical condition previously diagnosed by a medical doctor and/or signs/symptoms consistent with a possible medical condition based on the evaluation by the treating physiotherapist, 6.) reports previously receiving treatment by Wchan * Current substance abuse reported by participant or identified by treating psychotherapist * Currently receiving mental health services and/or physiotherapy services from other organization * Reports unresolvable conflict with participant/s enrolled in the study. **Gender Based:** True **Gender Description:** This program and study are being administered in an all-male reformatory. Thus, only men are being studied. **Maximum Age:** 100 Years **Minimum Age:** 18 Years **Sex:** MALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Sulaymaniyah **Country:** Iraq **Facility:** Sulaymaniyah Adult Male Reformatory **State:** Kurdistan #### Overall Officials **Official 1:** **Affiliation:** Northwestern University **Name:** Jeff A Hartman, DPT, MPH **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Description:** Still in deliberation **IPD Sharing:** UNDECIDED ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M13066 - Name: Pain - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06091579 **Brief Title:** A Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Treprostinil Palmitil Inhalation Powder (TPIP) in Healthy Participants **Official Title:** A Phase 1, Randomized, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, And Pharmacokinetics of Treprostinil Palmitil Inhalation Powder in Healthy Volunteers #### Organization Study ID Info **ID:** INS1009-102 #### Organization **Class:** INDUSTRY **Full Name:** Insmed Incorporated ### Status Module #### Completion Date **Date:** 2021-01-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-10-19 **Type:** ACTUAL **Last Update Submit Date:** 2023-10-16 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2021-01-12 **Type:** ACTUAL #### Start Date **Date:** 2020-09-17 **Type:** ACTUAL **Status Verified Date:** 2023-10 #### Study First Post Date **Date:** 2023-10-19 **Type:** ACTUAL **Study First Submit Date:** 2023-10-16 **Study First Submit QC Date:** 2023-10-16 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Insmed Incorporated #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Oversight Has DMC:** False ### Description Module **Brief Summary:** The primary purpose of this study is to evaluate the safety and tolerability of single and multiple doses of treprostinil palmitil inhalation powder in healthy participants. ### Conditions Module **Conditions:** - Healthy Volunteers ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** SEQUENTIAL ##### Masking Info **Masking:** QUADRUPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 42 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants in the single ascending dose (SAD) Cohort 1 received a single dose of TPIP at Dose A, Dose B, or Dose C by oral inhalation on Day 1. **Intervention Names:** - Drug: Treprostinil Palmitil Inhalation Powder **Label:** Part A (SAD Cohort 1): TPIP **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants in SAD Cohort 2 received a single dose of TPIP at Dose D or matching placebo by oral inhalation on Day 1. **Intervention Names:** - Drug: Treprostinil Palmitil Inhalation Powder - Drug: Placebo **Label:** Part A (SAD Cohort 2): TPIP or Placebo **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Participants in SAD Cohort 3 received a single dose of TPIP at Dose E or matching placebo by oral inhalation on Day 1. **Intervention Names:** - Drug: Treprostinil Palmitil Inhalation Powder - Drug: Placebo **Label:** Part A (SAD Cohort 3): TPIP or Placebo **Type:** EXPERIMENTAL #### Arm Group 4 **Description:** Participants in the multiple ascending dose (MAD) Cohort 1 received TPIP at Dose B, Dose C or matching placebo, once daily (QD) by oral inhalation on Days 1 through 7. **Intervention Names:** - Drug: Treprostinil Palmitil Inhalation Powder - Drug: Placebo **Label:** Part B (MAD Cohort 1): TPIP or Placebo **Type:** EXPERIMENTAL #### Arm Group 5 **Description:** Participants in MAD Cohort 2 received TPIP up to Dose D or matching placebo, QD by oral inhalation on Days 1 through 7. **Intervention Names:** - Drug: Treprostinil Palmitil Inhalation Powder - Drug: Placebo **Label:** Part B (MAD Cohort 2): TPIP or Placebo **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Part A (SAD Cohort 1): TPIP - Part A (SAD Cohort 2): TPIP or Placebo - Part A (SAD Cohort 3): TPIP or Placebo - Part B (MAD Cohort 1): TPIP or Placebo - Part B (MAD Cohort 2): TPIP or Placebo **Description:** Oral inhalation using a Plastiape capsule-based dry powder inhaler. **Name:** Treprostinil Palmitil Inhalation Powder **Other Names:** - INS1009 **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Part A (SAD Cohort 2): TPIP or Placebo - Part A (SAD Cohort 3): TPIP or Placebo - Part B (MAD Cohort 1): TPIP or Placebo - Part B (MAD Cohort 2): TPIP or Placebo **Description:** Oral placebo inhalation using a Plastiape capsule-based dry powder inhaler. **Name:** Placebo **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Safety and tolerability of single and multiple doses of treprostinil inhalation powder will be determined in healthy participants. **Measure:** Parts A and B: Number of Participants who Experienced an Adverse Event (AE) **Time Frame:** Up to Day 31 in Part A and Day 37 in Part B #### Secondary Outcomes **Description:** Pharmacokinetics of treprostinil following a single and multiple doses will be assessed in healthy participants. **Measure:** Parts A and B: Area Under the Plasma Concentration Versus Time Curve (AUC) of Treprostinil **Time Frame:** Part A: Predose and at multiple time points postdose up to Day 4; Part B: Predose and at multiple time points postdose up to Day 10 **Description:** Pharmacokinetics of treprostinil palmitil following a single and multiple doses will be assessed in healthy participants. **Measure:** Parts A and B: Area Under the Plasma Concentration Versus Time Curve (AUC) of Treprostinil Palmitil **Time Frame:** Part A: Predose and at multiple time points postdose up to Day 4; Part B: Predose and at multiple time points postdose up to Day 10 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * The participant is considered by the investigator to be in good general health as determined by medical history, physical examination findings, vital sign measurements, 12-lead electrocardiogram (ECG) results, and clinical laboratory test results within normal limits or considered not clinically significant by the investigator, at screening. Exclusion Criteria: * The participant has an allergy, documented hypersensitivity, or contraindication to the ingredients or to any of the excipients of treprostinil palmitil inhalation powder or treprostinil. * The participant has used any prescription (excluding hormonal birth control) or over-the-counter medications, including herbal or nutritional supplements, within 14 days before the first dose of study drug and throughout the study. * The participant has a history of anaphylaxis, previously documented hypersensitivity reaction to any drug. * The participant has had a surgical procedure that required general anesthesia (or equivalent) within 90 days prior to screening. * The participant has a body mass index \<19.0 or \>32.0 kilograms per square meter (kg/m\^2) at screening. * The participant has a history of syncope not due to dehydration or vasovagal syncope (eg, congenital cardiac arrhythmias such as Wolff-Parkinson-White syndrome, nodal tachycardia, ventricular tachycardia, etc). * The participant has active liver disease or hepatic dysfunction at screening or check-in visits. * The participant has a history of human immunodeficiency virus (HIV) infection. * The participant has a history of abnormal bleeding or bruising. * The participant has a history of malignancy in the past 5 years, with exception of nonmelanoma skin cancer. * The participant has a current history (within the past 12 months) of substance and/or alcohol abuse. * The participant is a current user of cigarettes (average of ≥1 cigarette/day) or e-cigarettes within 30 days prior to screening. * The participant has a positive test result for drugs of abuse, alcohol, or cotinine (indicating active current smoking) at screening or before the first dose of study drug or throughout the study. Note: Other inclusion/exclusion criteria may apply. **Healthy Volunteers:** True **Maximum Age:** 45 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Austin **Country:** United States **Facility:** USA001 **State:** Texas **Zip:** 78744 ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Ismat FA, Usansky HH, Villa R, Zou J, Teper A. Safety, Tolerability, and Pharmacokinetics of Treprostinil Palmitil Inhalation Powder for Pulmonary Hypertension: A Phase 1, Randomized, Double-Blind, Single- and Multiple-Dose Study. Adv Ther. 2022 Nov;39(11):5144-5157. doi: 10.1007/s12325-022-02296-x. Epub 2022 Sep 7. **PMID:** 36070132 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012120 - Term: Respiration Disorders - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M27137 - Name: Respiratory Aspiration - Relevance: HIGH - As Found: Inhalation - ID: M14957 - Name: Respiration Disorders - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000053120 - Term: Respiratory Aspiration ### Intervention Browse Module - Ancestors - ID: D000000959 - Term: Antihypertensive Agents ### Intervention Browse Module - Browse Branches - Abbrev: AnAg - Name: Antihypertensive Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M255601 - Name: Treprostinil - Relevance: HIGH - As Found: Push - ID: M4277 - Name: Antihypertensive Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: C000427248 - Term: Treprostinil ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00441779 **Brief Title:** Transfusion-Associated Microchimerism in Previously Injured Individuals Who Received a Blood Transfusion **Official Title:** Retrospective Study of the Prevalence of Transfusion-Associated Microchimerism Following Traumatic Injury, Burns, and Elective Orthopedic Procedures #### Organization Study ID Info **ID:** 1378 #### Organization **Class:** NIH **Full Name:** National Heart, Lung, and Blood Institute (NHLBI) #### Secondary ID Infos **ID:** 1R01HL083388 **Link:** https://reporter.nih.gov/quickSearch/1R01HL083388 **Type:** NIH ### Status Module #### Completion Date **Date:** 2011-10 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2016-07-12 **Type:** ESTIMATED **Last Update Submit Date:** 2016-07-11 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2011-10 **Type:** ACTUAL #### Start Date **Date:** 2008-08 **Status Verified Date:** 2014-10 #### Study First Post Date **Date:** 2007-03-01 **Type:** ESTIMATED **Study First Submit Date:** 2007-02-28 **Study First Submit QC Date:** 2007-02-28 **Why Stopped:** A separate study (similar subjects) identified transfusion-associated microchimerism only rarely, making this observational study impractical to conduct. ### Sponsor Collaborators Module #### Lead Sponsor **Class:** NIH **Name:** National Heart, Lung, and Blood Institute (NHLBI) #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Blood transfusions are frequently necessary in situations in which there is a large amount of blood loss. In some individuals who receive a blood transfusion, white blood cells from the donor's blood may remain in the body for years, a condition known as microchimerism. This study will evaluate the occurrence of microchimerism among the following three groups of individuals who previously received transfusions: 1) individuals with traumatic injuries; 2) individuals with burn injuries; and 3) individuals who underwent elective orthopedic operations. **Detailed Description:** Approximately 10% to 15% of injured patients who receive blood transfusions experience a condition known as transfusion-associated microchimerism. This occurs when white blood cells, or leukocytes, from the donor's blood persist in the recipient long after the transfusion occurs. The genetically distinct donor cells can remain in the individual for decades, and may account for as many as 4% of the white blood cells in the recipient's body. This suggests that the donor cells are tolerated by the recipient's immune system. The purpose of this study is to compare the incidence of microchimerism among individuals with three different types of injuries: 1) traumatic injuries; 2) thermal, or burn, injuries; and 3) injuries resulting from elective orthopedic surgical procedures. In this study, blood samples will be collected from individuals who were treated for traumatic injury, thermal injury, or elective orthopedic surgical procedures at the University of California at Davis Medical Center. Individuals who were treated from 2000 to 2003 and received a blood transfusion, as well as a control group of individuals who did not receive a transfusion, will be approached to enroll in the study. Individuals who agree to participate will have their blood analyzed for evidence of microchimerism. Information on health status, injury characteristics, hospital care, blood transfusion details, and blood donor characteristics will be collected from all participants. ### Conditions Module **Conditions:** - Chimerism - Blood Transfusion - Wounds and Injuries **Keywords:** - Microchimerism - Injuries - Burns - Orthopedic Surgery - Leukocytes ### Design Module #### Bio Spec **Description:** Whole blood, plasma, peripheral blood mononuclear cells **Retention:** SAMPLES_WITH_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** RETROSPECTIVE #### Enrollment Info **Count:** 59 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Traumatic injury **Label:** 1 #### Arm Group 2 **Description:** Elective orthopedic surgery **Label:** 2 #### Arm Group 3 **Description:** Burn injury **Label:** 3 ### Outcomes Module #### Primary Outcomes **Measure:** Microchimerism **Time Frame:** 5-11 years after transfusion ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Hospitalized for traumatic injury, thermal injury, or an elective orthopedic surgical procedure from 2000 to 2003 at the University of California at Davis Medical Center * Received at least 1 unit of transfused red blood cells Exclusion Criteria: * Currently incarcerated * Inadequate decision-making capacity of the participant and no available surrogate decision-maker * Prior bone marrow or solid organ transplantation * Prior blood transfusion other than at the time of hospitalization * Any history of an autoimmune disorder prior to hospitalization **Minimum Age:** 8 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT **Study Population:** Previously hospitalized patients ### Contacts Locations Module #### Locations **Location 1:** **City:** Sacramento **Country:** United States **Facility:** University of California, Davis, Medical Center **State:** California **Zip:** 95817 #### Overall Officials **Official 1:** **Affiliation:** Vitalant Research Institute **Name:** Michael P. Busch, MD, PhD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Utter GH, Owings JT, Lee TH, Paglieroni TG, Reed WF, Gosselin RC, Holland PV, Busch MP. Blood transfusion is associated with donor leukocyte microchimerism in trauma patients. J Trauma. 2004 Oct;57(4):702-7; discussion 707-8. doi: 10.1097/01.ta.0000140666.15972.37. **PMID:** 15514522 **Citation:** Utter GH, Owings JT, Lee TH, Paglieroni TG, Reed WF, Gosselin RC, Holland PV, Busch MP. Microchimerism in transfused trauma patients is associated with diminished donor-specific lymphocyte response. J Trauma. 2005 May;58(5):925-31; discussion 931-2. doi: 10.1097/01.ta.0000162142.72817.5c. **PMID:** 15920405 **Citation:** Lee TH, Paglieroni T, Utter GH, Chafets D, Gosselin RC, Reed W, Owings JT, Holland PV, Busch MP. High-level long-term white blood cell microchimerism after transfusion of leukoreduced blood components to patients resuscitated after severe traumatic injury. Transfusion. 2005 Aug;45(8):1280-90. doi: 10.1111/j.1537-2995.2005.00201.x. **PMID:** 16078913 ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC26 - Name: Wounds and Injuries - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M17685 - Name: Wounds and Injuries - Relevance: HIGH - As Found: Wounds and Injuries - ID: M5326 - Name: Burns - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000014947 - Term: Wounds and Injuries ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03964779 **Brief Title:** Prevalence of Thyroid Dysfunction and Anti-thyroid Antibodies in Infertile Women **Official Title:** Prevalence of Thyroid Dysfunction and Anti-thyroid Antibodies in Infertile Women #### Organization Study ID Info **ID:** 57 #### Organization **Class:** OTHER **Full Name:** Cairo University ### Status Module #### Completion Date **Date:** 2020-01 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** NOT_YET_RECRUITING #### Last Update Post Date **Date:** 2019-05-28 **Type:** ACTUAL **Last Update Submit Date:** 2019-05-24 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2019-12 **Type:** ESTIMATED #### Start Date **Date:** 2019-05 **Type:** ESTIMATED **Status Verified Date:** 2019-05 #### Study First Post Date **Date:** 2019-05-28 **Type:** ACTUAL **Study First Submit Date:** 2019-05-23 **Study First Submit QC Date:** 2019-05-24 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Cairo University #### Responsible Party **Investigator Affiliation:** Cairo University **Investigator Full Name:** Ahmed M Maged, MD **Investigator Title:** professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** The study population will be divided into three groups: * Group (A) consisting of 40 infertile women with either unexplained or anovulatory infertility with/without associated male factor of infertility, * Group (B) consisting of 40 infertile women with tubal (mechanical) factor of infertility with/without associated male factor of infertility, and * Group (C) consisting of 40 women with exclusive male factor of infertility and will be used as a control group. . All women will be subjected to: * Informed consent * Full history taking, including age, duration of infertility and whether primary or secondary * General and pelvix examination * Trans-vaginal ultrasonography * Determination of hormonal profile (FSH, LH, Estradiol, Prolactin) * Determination of ovulatory status * Determination of of tubal patency * Determination of presence of male factor * Determination of TSH and antithyroid antibodies (antithyroglobulin and antithyroid peroxidase) blood levels **Detailed Description:** The study population will be divided into three groups: * Group (A) consisting of 40 infertile women with either unexplained or anovulatory infertility with/without associated male factor of infertility, * Group (B) consisting of 40 infertile women with tubal (mechanical) factor of infertility with/without associated male factor of infertility, and * Group (C) consisting of 40 women with exclusive male factor of infertility and will be used as a control group. . All women will be subjected to: * Informed consent * Full history taking, including age, duration of infertility and whether primary or secondary * General and pelvix examination * Trans-vaginal ultrasonography * Determination of hormonal profile (FSH, LH, Estradiol, Prolactin) * Determination of ovulatory status (by previous documented history of induction of ovulation OR previous ultrasonography OR previous estimation midluteal serum progesterone, etc ...) * Determination of of tubal patency (by previous hysterosalpingiography or laparoscopy) * Determination of presence of male factor (by seminal fluid analysis) * Determination of TSH and antithyroid antibodies (antithyroglobulin and antithyroid peroxidase) blood levels ### Conditions Module **Conditions:** - Infertility, Female ### Design Module #### Design Info **Observational Model:** CASE_CONTROL **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 120 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 40 infertile women with either unexplained or anovulatory infertility with/without associated male factor of infertility **Intervention Names:** - Diagnostic Test: Hormonal assay - Diagnostic Test: Antibody assay **Label:** Anovulatory women #### Arm Group 2 **Description:** 40 infertile women with tubal (mechanical) factor of infertility with/without associated male factor of infertility **Intervention Names:** - Diagnostic Test: Hormonal assay - Diagnostic Test: Antibody assay **Label:** Tubal factor women #### Arm Group 3 **Description:** 40 women with exclusive male factor of infertility and will be used as a control group **Intervention Names:** - Diagnostic Test: Hormonal assay - Diagnostic Test: Antibody assay **Label:** male factor couple ### Interventions #### Intervention 1 **Arm Group Labels:** - Anovulatory women - Tubal factor women - male factor couple **Description:** Measurement of Thyroid stimulating hormone **Name:** Hormonal assay **Other Names:** - TSH assay **Type:** DIAGNOSTIC_TEST #### Intervention 2 **Arm Group Labels:** - Anovulatory women - Tubal factor women - male factor couple **Description:** antithyroglobulin and antithyroid peroxidase **Name:** Antibody assay **Other Names:** - antithyroid antibodies **Type:** DIAGNOSTIC_TEST ### Outcomes Module #### Primary Outcomes **Description:** prevalence of thyroid dysfunction (clinical or subclinical, hypothyroidism or hyperthyroidism) among infertile women **Measure:** prevalence of thyroid dysfunction among infertile women **Time Frame:** through study completion, an average of 6 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * - Age: 18 - 38 years * Infertile women whether due to functional, anatomical, male factors OR unexplained OR combined factors Exclusion Criteria: * - Age: below 18 and above 38 years * Patients with autoimmune diseases (such as lupus and rheumatoid arthritis) * Acute illness that require hospitalization * Patients on the following medications: steroids, dopamine, iodine, amiodarone, lithium, donperidone, thyroid hormone, and phenytoin. * Patients who work at night. **Gender Based:** True **Maximum Age:** 40 Years **Minimum Age:** 20 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** FEMALE **Standard Ages:** - ADULT **Study Population:** 120 women with infertility will be divided into three groups: * Group (A) consisting of 40 infertile women with either unexplained or anovulatory infertility with/without associated male factor of infertility, * Group (B) consisting of 40 infertile women with tubal (mechanical) factor of infertility with/without associated male factor of infertility, and * Group (C) consisting of 40 women with exclusive male factor of infertility and will be used as a control group. ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Ahmed Maged **Phone:** +201005227404 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Ahmed wali **Phone:** +201001735088 **Role:** CONTACT #### Locations **Location 1:** **City:** Cairo **Country:** Egypt **Facility:** Kasr Alainy medical school **Zip:** 12151 #### Overall Officials **Official 1:** **Affiliation:** Professor **Name:** Ahmed Maged **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ## Derived Section ### Condition Browse Module - Ancestors - ID: D000091662 - Term: Genital Diseases - ID: D000091642 - Term: Urogenital Diseases - ID: D000005831 - Term: Genital Diseases, Female - ID: D000052776 - Term: Female Urogenital Diseases - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications ### Condition Browse Module - Browse Branches - Abbrev: BC19 - Name: Gland and Hormone Related Diseases - Abbrev: All - Name: All Conditions - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions ### Condition Browse Module - Browse Leaves - ID: M16718 - Name: Thyroid Diseases - Relevance: LOW - As Found: Unknown - ID: M10290 - Name: Infertility - Relevance: HIGH - As Found: Infertility - ID: M10291 - Name: Infertility, Female - Relevance: HIGH - As Found: Infertility, Female - ID: M2876 - Name: Genital Diseases - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M8943 - Name: Genital Diseases, Female - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000007246 - Term: Infertility - ID: D000007247 - Term: Infertility, Female ### Intervention Browse Module - Ancestors - ID: D000007155 - Term: Immunologic Factors - ID: D000045505 - Term: Physiological Effects of Drugs ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M9789 - Name: Hormones - Relevance: LOW - As Found: Unknown - ID: M4225 - Name: Antibodies - Relevance: HIGH - As Found: Program - ID: M10184 - Name: Immunoglobulins - Relevance: HIGH - As Found: Program - ID: M19117 - Name: Immunoglobulins, Intravenous - Relevance: LOW - As Found: Unknown - ID: M10201 - Name: Immunologic Factors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000000906 - Term: Antibodies - ID: D000007136 - Term: Immunoglobulins ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03126279 **Brief Title:** fMRI and Central Sensitization in Chronic Knee Osteoarthritis. A Pre and Post TKR Study **Official Title:** Predicting Chronic Post TKR Pain by Assessing Central Sensitisation Using Functional Brain MRI #### Organization Study ID Info **ID:** 10093 #### Organization **Class:** OTHER **Full Name:** University of Nottingham ### Status Module #### Completion Date **Date:** 2017-08 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2017-04-24 **Type:** ACTUAL **Last Update Submit Date:** 2017-04-21 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2017-06 **Type:** ESTIMATED #### Start Date **Date:** 2015-08 **Status Verified Date:** 2017-04 #### Study First Post Date **Date:** 2017-04-24 **Type:** ACTUAL **Study First Submit Date:** 2017-04-11 **Study First Submit QC Date:** 2017-04-21 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Aalborg University **Class:** OTHER_GOV **Name:** Medical Research Council **Class:** OTHER **Name:** Arthritis Research UK **Class:** OTHER **Name:** Royal College of Surgeons of Edinburgh #### Lead Sponsor **Class:** OTHER **Name:** University of Nottingham #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Painful osteoarthritis (OA) is the 4th largest cause of disability in the UK. Preoperative temporal summation, a measure of central pain facilitation, has been shown to predict postoperative pain after total knee replacement surgery (TKR). The assessment of the brain's response to noxious stimuli using non-invasive functional MRI (fMRI) may be key in identifying imaging biomarkers within the brain that map central sensitization changes seen in OA. fMRI may help explain why up to 20% of patients undergoing TKR surgery develop persistent post-operative pain. To test these concepts the study aims to functionally characterise the brain activity related to temporal summation of pain in healthy individuals and OA patients using a novel fMRI cuff algometer. Assessment of outcomes in terms of pain and function will be performed 6 months post TKR surgery ### Conditions Module **Conditions:** - Osteoarthritis - Pain - Postoperative Pain - Surgery **Keywords:** - Osteoarthritis - Pain - Postoperative Pain ### Design Module #### Design Info **Observational Model:** CASE_CONTROL **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 50 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients will be recruited from orthopaedic preoperative clinics from those awaiting total knee replacement surgery. **Intervention Names:** - Procedure: Total Knee Replacement Surgery **Label:** Preoperative Chronic Knee OA Patients #### Arm Group 2 **Description:** Healthy volunteers will be recruited that are aged match to our OA patient cohort so meaningful comparisons regarding brain activity and pain sensitisation characteristics can be assessed. **Label:** Healthy Volunteers ### Interventions #### Intervention 1 **Arm Group Labels:** - Preoperative Chronic Knee OA Patients **Description:** All chronic knee osteoarthritis patients will be recruited as they are on the NHS waiting list for a total knee replacement. Only this group will undergo the procedure **Name:** Total Knee Replacement Surgery **Other Names:** - Total **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Description:** Using BOLD fMRI we aim to assess the difference in neural brain activity between patients with chronic knee OA pain and a group of healthy volunteers and to assess the reversal of this activity pattern 6 months post TKR surgery **Measure:** Brain activity related to temporal summation of pain in healthy individuals and OA patients using a novel fMRI cuff algometer. **Time Frame:** 6 months post total knee replacement surgery #### Secondary Outcomes **Description:** Pain using visual analogue scale **Measure:** Assessment of knee pain using the Visual Analogue Scale for Pain (0-10) **Time Frame:** 6 months post total knee replacement surgery **Description:** pain and function outcome measure **Measure:** Assessment of improvement in the Oxford Knee Score **Time Frame:** 6 months post total knee replacement surgery **Description:** quality of life index measure **Measure:** Assessment of change in healthy related quality of life measure using the EQ5D-5L questionnaire **Time Frame:** 6 months post total knee replacement ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * OA Group Unilateral Knee OA No previous knee surgery Able to give informed consent Age \> 40 years - Healthy Volunteers Group No OA or knee pain Able to give informed consent Age \> 40 years Exclusion Criteria: -OA Group Major medical, psychiatric, neurological Cx Other chronic pain condition (fibromyalgia) Contraindications to MRI Active Cancer Neuropathic drug treatment -Healthy Volunteers Group Pregnancy Lower limb pain or previous knee surgery Contraindications to MRI Active Cancer Major medical, psychiatric, neurological condition **Healthy Volunteers:** True **Minimum Age:** 40 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Patients with predominelaty unilateral knee osteoarthritis awaiting for total knee replacement surgery for pain will be included. Patients will recruited from Nottingham University Hospitals NHS Trust. Healthy volunteers will also be recruited as part of this study and they will be recruited from posters and advertisement within the university. ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Thomas Kurien, BMBS **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Brigitte E Scammell, FRCS **Role:** CONTACT #### Locations **Location 1:** **City:** Nottingham **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Thomas Kurien, MRCSEd - **Role:** CONTACT ***Contact 2:*** - **Name:** Thomas Kurien, MRCSEd - **Role:** SUB_INVESTIGATOR ***Contact 3:*** - **Name:** Brigitte E Scammell, FRCS - **Role:** SUB_INVESTIGATOR ***Contact 4:*** - **Name:** Dorothee P Auer, PhD - **Role:** PRINCIPAL_INVESTIGATOR ***Contact 5:*** - **Name:** Kristian K Petersen, PhD - **Role:** SUB_INVESTIGATOR ***Contact 6:*** - **Name:** Lars Ardent-Nielsen, PhD - **Role:** SUB_INVESTIGATOR ***Contact 7:*** - **Name:** Thomas Graven-Nieslen, PhD - **Role:** SUB_INVESTIGATOR ***Contact 8:*** - **Name:** David A Walsh, PhD - **Role:** SUB_INVESTIGATOR ***Contact 9:*** - **Name:** Robert W Kerslake, PhD - **Role:** SUB_INVESTIGATOR ***Contact 10:*** - **Name:** Diane Reckziegel, PhD - **Role:** SUB_INVESTIGATOR ***Contact 11:*** - **Name:** Sarina Iwabuchi, PhD - **Role:** SUB_INVESTIGATOR ***Contact 12:*** - **Name:** William J Cottam, MSc - **Role:** SUB_INVESTIGATOR **Country:** United Kingdom **Facility:** Academic Division of Trauma, Orthopaedics and Sports Medicine **State:** Nottinghamshire **Status:** RECRUITING **Zip:** NG7 2UH #### Overall Officials **Official 1:** **Affiliation:** The University of Nottingham **Name:** Dorothee P Auer, PhD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000001168 - Term: Arthritis - ID: D000007592 - Term: Joint Diseases - ID: D000009140 - Term: Musculoskeletal Diseases - ID: D000012216 - Term: Rheumatic Diseases - ID: D000011183 - Term: Postoperative Complications - ID: D000010335 - Term: Pathologic Processes - ID: D000010146 - Term: Pain - ID: D000009461 - Term: Neurologic Manifestations ### Condition Browse Module - Browse Branches - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M12926 - Name: Osteoarthritis - Relevance: HIGH - As Found: Osteoarthritis - ID: M22168 - Name: Osteoarthritis, Knee - Relevance: LOW - As Found: Unknown - ID: M13069 - Name: Pain, Postoperative - Relevance: HIGH - As Found: Postoperative Pain - ID: M13066 - Name: Pain - Relevance: LOW - As Found: Unknown - ID: M4476 - Name: Arthritis - Relevance: LOW - As Found: Unknown - ID: M10621 - Name: Joint Diseases - Relevance: LOW - As Found: Unknown - ID: M12097 - Name: Musculoskeletal Diseases - Relevance: LOW - As Found: Unknown - ID: M15045 - Name: Rheumatic Diseases - Relevance: LOW - As Found: Unknown - ID: M6323 - Name: Collagen Diseases - Relevance: LOW - As Found: Unknown - ID: M14065 - Name: Postoperative Complications - Relevance: LOW - As Found: Unknown - ID: M12404 - Name: Neurologic Manifestations - Relevance: LOW - As Found: Unknown - ID: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000010003 - Term: Osteoarthritis - ID: D000010149 - Term: Pain, Postoperative ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02079779 **Brief Title:** Efficacy Study of an Interactive Robot for the Rehabilitation of the Upper Limb in Acute Stroke Patients **Official Title:** Study of the Effectiveness of an Interactive Robot for the Rehabilitation of the Upper Limb in Acute Stroke Patients by Evaluating the 3 Fields of the ICF: a Prospective, Randomized, Controlled, Simple Blind Study #### Organization Study ID Info **ID:** IONS-Gilliaux-02 #### Organization **Class:** OTHER **Full Name:** Cliniques universitaires Saint-Luc- Université Catholique de Louvain ### Status Module #### Completion Date **Date:** 2017-10-02 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-08-24 **Type:** ACTUAL **Last Update Submit Date:** 2018-08-23 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2015-01-09 **Type:** ACTUAL #### Start Date **Date:** 2015-01-09 **Type:** ACTUAL **Status Verified Date:** 2018-08 #### Study First Post Date **Date:** 2014-03-06 **Type:** ESTIMATED **Study First Submit Date:** 2014-03-04 **Study First Submit QC Date:** 2014-03-05 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Cliniques universitaires Saint-Luc- Université Catholique de Louvain #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** Stroke is the principal cause of permanent disability within the investigators population. This incapacity justifies an intensive and prolonged multidisciplinary rehabilitation, which can be optimized by robotics. The investigators team has developed a robot designed to rehabilitate the upper limb. This robot allows the patient to perform active, passive, or assisted exercises. The system is also able to assess movement quality and to provide a feedback to the patient and the therapist via a graphical interface. This therapy is designed to improve functional recovery of patients, and then their quality of life. Few quality studies have evaluated the efficacy of robotic assisted therapy in patients at the acute stage of rehabilitation (\< 3 months post stroke) when most improvements are observed. Thus, the aim of this study was to objectify the effectiveness of robotic-assisted rehabilitation in the acute stage after stroke by evaluating the 3 fields of the ICF (International Classification of Functioning, Disability and Health) and performing a prospective multicenter randomized controlled single blind trial. In this study, 60 stroke patients will be recruited and randomized into two groups. All patients will receive a similar classical rehabilitation as a basis. Patients of the control and experimental groups will receive a supplement of classical rehabilitation and robotic-assisted therapy, respectively. **Detailed Description:** INTRODUCTION The cerebral vascular accident affects two people per thousand each year (Duncan et al. 2005). This injury is the leading cause of permanent disability in our population. The brain damage is expressed by different neurological impairments and functional disabilities. These disabilities justify intensive and sustained multidisciplinary rehabilitation to reduce neurological impairments, to improve the activities and participation of patients, and, ultimately, their quality of life (Duncan et al., 2005). Recently appeared in rehabilitation to stimulate maximum brain plasticity robotic devices meet the actual recommendations existing in stroke's rehabilitation (Langhorne et al., 2011). Indeed, the robots allow the execution of a large number of movements whose quality is controlled. A visual interface gives the patient a feedback of its movements, and offers exercises oriented functional tasks that have meaning for him and the possibly dive into a virtual reality. All these elements justify the clinical development of robots to assist the therapists (Pignolo et al. 2009). The therapeutic efficacy of these robots for the rehabilitation of the upper limb was evaluated especially in chronic stroke patients (\> 6 months after stroke) (Mehrholz et al., 2012). Unfortunately, few quality studies was conducted in these patients at the acute stage of rehabilitation, during which the potential of brain plasticity is the most important and the largest functional progress are observed (Stinear et al. 2012). In addition, few studies have evaluated the effect of these therapies robotic on the three fields of the ICF (Mehrholz et al., 2012). Many studies focus on impairments (e.g. muscle strength) without assessing the functional ability of the patient in activities of daily living. OBJECTIVES To perform a multicenter, single blind, randomized, controlled trial to assess the efficacy of of robotic-assisted therapy in acute stroke patients by evaluating the three fields of the ICF. METHODS Patients will be included in the Cliniques Universitaires Saint-Luc (Brussels, Belgique) and the center of William Lennox (Ottignies, Belgique). The patients will be randomised into two groups (control and experimental), using a stratified randomization method to ensure the equivalence of the two groups for motor neurological impairments (score of the Fugl-Meyer). This randomization will be organized independently in each centre participating in the study. The study of statistical power shows that 27 patients per group are necessary. This study of power has taken into account a statistical power desired to 99%, a minimum detectable change to 0.09 and a standard deviation of 0.08 on the upper limb kinematics of the patient (Gilliaux et al. 2014). In estimating a risk of out drop to 10%, the investigators plan to recruit 60 patients. In each centre, in the acute phase, the patients receive daily a multidisciplinary rehabilitation (physiotherapy, occupational therapy, speech therapy,...) more or less intensive (60 to 300 min). In the experimental group, one of these daily therapies will be devoted to the rehabilitation robot, 4 times a week for 9 weeks. The control group will benefit from conventional treatments. As such, the duration of support will be similar between the two groups. All treatments will be administered by therapists specialized in neurological rehabilitation. Patients will be evaluated three times in the study: before the start of treatments, at the end of treatments, and 6 months after the stroke event. All assessments will be carried out by a therapist which will be not informed the group that the patient is assigned (single-blind). PERSPECTIVES From this study, the investigators hope to demonstrate the efficacy of robotic-assisted therapy in acute stroke patients by evaluating the three fields of the ICF. These results could prove that this tool can be a significant complement for the stroke rehabilitation. ### Conditions Module **Conditions:** - Acute Stroke **Keywords:** - Robotic-assisted therapy - Acute Stroke - International Classification of Functioning, Disability and Health ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 45 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** All patients will receive a similar classical rehabilitation as a basis. The 30 patients of this group will receive a supplement of robotic-assisted therapy. **Intervention Names:** - Other: Robotic-assisted therapy - Other: Classical therapy **Label:** Robotic-assisted therapy **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** All patients will receive a similar classical rehabilitation as a basis. The 30 patients of this group will receive a supplement of classical rehabilitation. **Intervention Names:** - Other: Classical therapy **Label:** Classical therapy **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Robotic-assisted therapy **Description:** The robotic device is designed to intensively rehabilitate the upper limb. Indeed, this robot allows the patient to perform a lot of active, passive, or assisted exercises. The level of assistance is determined and provided by the robot in function of the patient performance (i.e. quality of movements). **Name:** Robotic-assisted therapy **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - Classical therapy - Robotic-assisted therapy **Description:** Conventional therapy will be performed by therapists specialized in neuro-rehabilitation **Name:** Classical therapy **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Measure:** Kinematic **Time Frame:** Change from Baseline in Kinematic at an expected average of 2 months (after the treamtment) and 6 months post stroke #### Secondary Outcomes **Measure:** Fugl-Meyer upper limb assessment **Time Frame:** Change from Baseline in upper limb motor control at an expected average of 2 months (after the treamtment) and 6 months post stroke **Measure:** Stroke Impairment Assessment Set (sensitive and Pain items) **Time Frame:** Change from Baseline in sensitivity and pain at an expected average of 2 months (after the treamtment) and 6 months post stroke **Measure:** Box and Block test **Time Frame:** Change from Baseline in manual ability at an expected average of 2 months (after the treamtment) and 6 months post stroke **Measure:** Strenght test of the Medical Research council **Time Frame:** Change from Baseline in upper limb strenght at an expected average of 2 months (after the treamtment) and 6 months post stroke **Measure:** Ashworth test **Time Frame:** Change from Baseline in upper limb spasticity at an expected average of 2 months (after the treamtment) and 6 months post stroke **Measure:** Bell's test **Time Frame:** Change from Baseline in Hemineglect at an expected average of 2 months (after the treamtment) and 6 months post stroke **Measure:** Wolf Motor Function Test **Time Frame:** Change from Baseline in activity of daily living at an expected average of 2 months (after the treamtment) and 6 months post stroke **Measure:** Abilhand **Time Frame:** Change from Baseline in activity of daily living at an expected average of 2 months (after the treamtment) and 6 months post stroke **Measure:** Activlim **Time Frame:** Change from Baseline in activity of daily living at an expected average of 2 months (after the treamtment) and 6 months post stroke **Measure:** Stroke Impact Scale (Participation item) **Time Frame:** Change from Baseline in participation in social activities at an expected average of 2 months (after the treamtment) and 6 months post stroke ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * first stroke * acute stroke (less than 1 month) * unilateral localisation of the stroke * moderate to severe upper limb impairments (7\<Fugl Meyer score\<50/66) Exclusion Criteria: * brainstem or cerebellum stroke * an unstable clinical condition contraindicating the upper limb rehabilitation treatments * cognitive disorders preventing the understanding of the instructions * other neurological or orthopedic pathology affecting the upper limb **Maximum Age:** 90 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Brussel **Country:** Belgium **Facility:** Cliniques Universitaires Saint Luc **Zip:** 1200 **Location 2:** **City:** Bruxelles **Country:** Belgium **Facility:** Centre Hospitalier Valida **Location 3:** **City:** Ottignies **Country:** Belgium **Facility:** Centre Neurologique William Lennox **Zip:** 1340 #### Overall Officials **Official 1:** **Affiliation:** Université Catholique de Louvain **Name:** Maxime Gilliaux, PhD student **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** Université Catholique de Louvain **Name:** Gaetan Stoquart, Professor **Role:** PRINCIPAL_INVESTIGATOR **Official 3:** **Affiliation:** Université Catholique de Louvain **Name:** Christine Detrembleur, Professor **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Duncan PW, Zorowitz R, Bates B, Choi JY, Glasberg JJ, Graham GD, Katz RC, Lamberty K, Reker D. Management of Adult Stroke Rehabilitation Care: a clinical practice guideline. Stroke. 2005 Sep;36(9):e100-43. doi: 10.1161/01.STR.0000180861.54180.FF. No abstract available. **PMID:** 16120836 **Citation:** Langhorne P, Bernhardt J, Kwakkel G. Stroke rehabilitation. Lancet. 2011 May 14;377(9778):1693-702. doi: 10.1016/S0140-6736(11)60325-5. **PMID:** 21571152 **Citation:** Mehrholz J, Hadrich A, Platz T, Kugler J, Pohl M. Electromechanical and robot-assisted arm training for improving generic activities of daily living, arm function, and arm muscle strength after stroke. Cochrane Database Syst Rev. 2012 Jun 13;(6):CD006876. doi: 10.1002/14651858.CD006876.pub3. **PMID:** 22696362 **Citation:** Pignolo L. Robotics in neuro-rehabilitation. J Rehabil Med. 2009 Nov;41(12):955-60. doi: 10.2340/16501977-0434. **PMID:** 19841823 **Citation:** Stinear CM, Barber PA, Petoe M, Anwar S, Byblow WD. The PREP algorithm predicts potential for upper limb recovery after stroke. Brain. 2012 Aug;135(Pt 8):2527-35. doi: 10.1093/brain/aws146. Epub 2012 Jun 10. **PMID:** 22689909 **Citation:** Gilliaux M, Lejeune TM, Detrembleur C, Sapin J, Dehez B, Selves C, Stoquart G. Using the robotic device REAplan as a valid, reliable, and sensitive tool to quantify upper limb impairments in stroke patients. J Rehabil Med. 2014 Feb;46(2):117-25. doi: 10.2340/16501977-1245. **PMID:** 24213596 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000002561 - Term: Cerebrovascular Disorders - ID: D000001927 - Term: Brain Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000014652 - Term: Vascular Diseases - ID: D000002318 - Term: Cardiovascular Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M22306 - Name: Stroke - Relevance: HIGH - As Found: Acute Stroke - ID: M5810 - Name: Cerebrovascular Disorders - Relevance: LOW - As Found: Unknown - ID: M5204 - Name: Brain Diseases - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000020521 - Term: Stroke ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05849779 **Acronym:** IPA **Brief Title:** Inhaled Sevoflurane for ARDS Prevention **Official Title:** Randomized Clinical Trial of Inhaled Sedation With Sevoflurane in Critically Ill Patients at Risk of Developing the Acute Respiratory Distress Syndrome #### Organization Study ID Info **ID:** IPA trial (AOI 2019 JABAUDON) #### Organization **Class:** OTHER **Full Name:** University Hospital, Clermont-Ferrand ### Status Module #### Completion Date **Date:** 2025-07-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-05-20 **Type:** ACTUAL **Last Update Submit Date:** 2024-05-17 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2025-05-31 **Type:** ESTIMATED #### Start Date **Date:** 2023-07-24 **Type:** ACTUAL **Status Verified Date:** 2024-05 #### Study First Post Date **Date:** 2023-05-09 **Type:** ACTUAL **Study First Submit Date:** 2023-04-27 **Study First Submit QC Date:** 2023-05-05 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University Hospital, Clermont-Ferrand #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** This study focuses on patients who are at risk of developing a serious, life-threatening respiratory disease called Acute Respiratory Distress Syndrome (ARDS), which severely disrupts the function of their lungs. Preclinical studies have shown that the use of a volatile anesthetic agent such as Sevoflurane could be beneficial in the treatment and prevention of this respiratory condition. By improving gas exchange and attenuating pulmonary inflammation in particular, this agent would make it possible to prevent deterioration or to restore pulmonary function more rapidly. Half of the patients will receive inhaled sedation with sevoflurane and the other half will receive intravenous sedation already routinely used in participating ICUs (typically propofol, dexmedetomidine or a benzodiazepine, i.e. drugs approved for sedation). The aim of this study is to assess whether the use of Sevoflurane could be beneficial in the prevention of ARDS. **Detailed Description:** MAIN OBJECTIVE To assess the efficacy of inhaled sevoflurane, compared to current intravenous sedation practice, for improving PaO2/FiO2 in ICU patients at high risk for ARDS. HYPOTHESIS The investigators hypothesized that a strategy of inhaled sedation with sevoflurane could be more effective than current intravenous sedation practice at improving pulmonary function during the early days of ICU admission, in patients at risk of ARDS. ### Conditions Module **Conditions:** - Acute Respiratory Distress Syndrome **Keywords:** - ARDS - Sedation - Inhaled sevoflurane ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** Investigator-initiated, single-center, prospective, randomized, stratified, parallel-group clinical trial with blinded outcome assessment and concealed allocation of patients at risk of developing the ARDS to a strategy of inhaled sedation with sevoflurane or to a strategy of current intravenous sedation practice. ##### Masking Info **Masking:** SINGLE **Masking Description:** Patients will be followed up for primary and secondary endpoints by members of the research staff who will be unaware of the trial group allocation. Information on whether the primary and secondary outcomes occur will be collected and entered into the electronic web-based case report form (eCRF) by trial or clinical trained personal (clinical research associate), blinded to the allocation group, under the supervision of the local principal investigator (PI) or designee who will also be unaware of the trial group allocation. Finally, the independent trial statistician and the members of the data monitoring and safety committee (DMSC) will also remain blinded for the allocation during analysis. However, the observation of differences in serious adverse events between the two groups will allow, for safety reasons may the DMSC deem necessary, to unblind allocation groups. **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 80 **Type:** ESTIMATED **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Sevoflurane as vaporized via the Anesthesia Conserving Device (Sedaconda-ACD-S, Sedana Medical, Danderyd, Sweden). **Intervention Names:** - Drug: Inhaled sedation with sevoflurane **Label:** Inhaled sedation with sevoflurane **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** The investigators will not mandate the sedative type, but rather encourage the use of sedatives that are already routinely used in participating ICUs (typically a benzodiazepine, propofol, or dexmedetomidine, i.e. drugs approved for sedation). **Intervention Names:** - Drug: Intravenous sedation (current practice) **Label:** Intravenous sedation **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Inhaled sedation with sevoflurane **Description:** In both arms, the management of sedation will be conducted in the broader picture of ICU patient care, as per current standard practice in participating ICUs, and following the current guidelines for Pain, Agitation, Delirium, Immobility, and Sleep Disruption (PADIS) published in 2018 by the Society of Critical Care Medicine. Among many recommendations, this will include the monitoring and titration of both sedation and analgesia using validated scores such as the Richmond Agitation-Sedation Scale (RASS) for sedation. As a result of the current recommendations, the level, dose, and duration of sedation will vary among patients and will be decided by the treating clinicians. The choice of the analgesic drug(s) will be as per the treating clinicians. Other aspects of critical care will adhere to standard care, including the use of the "Checklist for Lung Injury Prevention" (CLIP). **Name:** Inhaled sedation with sevoflurane **Other Names:** - Experimental **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Intravenous sedation **Description:** In both arms, the management of sedation will be conducted in the broader picture of ICU patient care, as per current standard practice in participating ICUs, and following the current guidelines for Pain, Agitation, Delirium, Immobility, and Sleep Disruption (PADIS) published in 2018 by the Society of Critical Care Medicine. Among many recommendations, this will include the monitoring and titration of both sedation and analgesia using validated scores such as the Richmond Agitation-Sedation Scale (RASS) for sedation. As a result of the current recommendations, the level, dose, and duration of sedation will vary among patients and will be decided by the treating clinicians. The choice of the analgesic drug(s) will be as per the treating clinicians. Other aspects of critical care will adhere to standard care, including the use of the "Checklist for Lung Injury Prevention" (CLIP). **Name:** Intravenous sedation (current practice) **Other Names:** - Control **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** longitudinal evolution in the PaO2/FiO2 ratio **Measure:** PaO2/FiO2 ratio **Time Frame:** within 5 days from randomization #### Secondary Outcomes **Description:** Progression to ARDS will be assessed according to the Berlin criteria, including chest radiographs **Measure:** Progression to ARDS **Time Frame:** within 5 days from randomization **Description:** Pneumonia will be defined according to the 3 following criteria: * Two chest radiographs showing signs of pneumonia, or one in absence of cardiomyopathy or underlying pulmonary condition. * One item among: body temperature ≥38.3°C without evident cause, leukocytes \<4000/mm3 or ≥12000/mm3 * Two items among: purulent secretions, cough or dyspnea, increased need for oxygen supplementation or ventilatory assistance. **Measure:** Rate of pneumonia **Time Frame:** Presence of pneumonia will be assessed daily until Day 5, and at Day 28 or ICU discharge, whichever comes first. **Description:** Ventilator free days to day 28 are defined as the number of days from the time of initiating unassisted breathing to day 28 after randomization, assuming survival for at least two consecutive calendar days after initiating unassisted breathing and continued unassisted breathing to day 28. If a patient returns to assisted breathing and subsequently achieves unassisted breathing to day 28, VFDs will be counted from the end of the last period of assisted breathing to day 28. A period of assisted breathing lasting less than 24 hours and for the purpose of a surgical procedure will not count against the VFD calculation. If a patient was receiving assisted breathing at day 27 or dies prior to day 28, VFDs will be zero. **Measure:** Ventilator-free days to day 28 **Time Frame:** 28 days after randomization **Description:** Organ failure is defined as present when the most abnormal vital signs or clinically available lab value meets the definition of clinically significant organ failure according to SOFA scores. Patients will be followed daily from randomization to day 5 for development of organ failures. **Measure:** Organ failure to day 5 **Time Frame:** 5 days after randomization **Description:** The occurrence of death in the ICU will be recorded until day 28. **Measure:** Mortality at day 28 **Time Frame:** 28 days after randomization **Description:** The total number of days from admission to ICU discharge will be recorded until day 28 **Measure:** Length of ICU-stay up to 28 days **Time Frame:** 28 days after randomization **Description:** - Oxygenation Index on study days 1-5 **Measure:** Physiological measures: Oxygenation **Time Frame:** 28 days after randomization **Description:** - PaCO2 on study days 1-5 **Measure:** Physiological measures: PaCO2 **Time Frame:** 28 days after randomization **Description:** - Arterial pH on study days 1-5 **Measure:** Physiological measures: pH **Time Frame:** 28 days after randomization **Description:** - Level of PEEP (and static auto-PEEP in patients under controlled ventilation) on study days 1-5 **Measure:** Physiological measures: PEEP **Time Frame:** 28 days after randomization **Description:** - Plateau pressure, static compliance of the respiratory system on study day 1-5 **Measure:** Physiological measures: Plateau pressure **Time Frame:** 28 days after randomization **Description:** - Development of pneumothorax through day 28 **Measure:** Physiological measures: Pneumothorax **Time Frame:** 28 days after randomization **Description:** - Time to switching from controlled to pressure-support ventilation through day 5 **Measure:** Physiological measures: Switch from controlled to pressure-support ventilation **Time Frame:** 28 days after randomization **Description:** - Airway occlusion pressure at 0.1 s (P0.1), an index of respiratory drive, on the day the patient is switched to pressure-support ventilation if within 5 days since randomization **Measure:** Physiological measures: Airway occlusion pressure **Time Frame:** 28 days after randomization **Description:** - Hemodynamic measures (mean arterial pressure, dose of infused norepinephrine or other vasopressor, serum lactate level) on study days 1-5 **Measure:** Hemodynamic measures **Time Frame:** 28 days after randomization **Description:** - KDIGO criteria for acute kidney injury 24 through day 5 **Measure:** Physiological measures: Acute kidney injury **Time Frame:** 28 days after randomization **Description:** - Supraventricular tachycardia (SVT) or new onset atrial fibrillation through day 5 **Measure:** Physiological measures: Supraventricular tachycardia **Time Frame:** 28 days after randomization **Description:** The Confusion Assessment Method for the ICU (CAM-ICU, Appendix C )97 will be assessed daily from study entry to study day 28, death or ICU discharge, whichever comes first. **Measure:** ICU-acquired delirium **Time Frame:** 28 days after randomization **Description:** Plasma samples will be collected from indwelling catheters (when available) at study entry and on days 1, 2, 3, 4, 5 in order to assemble a biological collection aimed at further investigating the effects of inhaled sedation with sevoflurane in patients with ARDS. The investigators will also collect whole blood samples at study entry and on day 2 for future studies of macrophage activation profiles and RNA and DNA studies. **Measure:** Biomarker measurements **Time Frame:** from inclusion to 5 days ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Age ≥ 18 years 2. Admitted to participating ICUs with at least one known risk factor for ARDS and a LIPS equals to, or greater than, 4 (Appendix D)105 3. Patient under invasive mechanical ventilation 4. With expected duration of sedation superior or equal to 4 hours 5. Affiliation to the French Sécurité Sociale Exclusion Criteria: * Patient under judicial protection, guardianship or supervision, as defined by art L1121-8 of the Public Health Code * Patient under psychiatric care as defined by art. L1121-6 of the Public Health Code * Patient deprived of their freedom by judiciary or administrative order * Known pregnancy * Presence of ARDS prior to randomization * Endotracheal ventilation for greater than 24 hours prior to randomization * Home mechanical ventilation (non-invasive ventilation or via tracheotomy) except for CPAP/BIPAP used solely for sleep-disordered breathing * Tidal volume of 6 mL/kg predicted body weight (PBW) below 200 mL (i.e. height inferior to 134cm for a man and 139cm for a woman) * Moribund patient, i.e. not expected to survive 24 hours despite intensive care * Previous hypersensitivity or anaphylactic reaction to sevoflurane or to the intravenous sedation agent routinely used in the participating ICU (such as midazolam, propofol, or dexmedetomidine) * Absolute contra-indications to the intravenous sedation agent routinely used in the participating ICU (such as midazolam, propofol, or dexmedetomidine) * Medical history of malignant hyperthermia * Long QT syndrome at risk of arrhythmic events * Medical history of liver disease attributed to previous exposure to a halogenated agent (including sevoflurane) * Suspected or proven intracranial hypertension * Enrollment in another interventional trial with direct impact on oxygenation **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Lise Laclautre **Phone:** +33473754963 **Role:** CONTACT #### Locations **Location 1:** **City:** Clermont-ferrand **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Dominique Morand - **Phone:** +33684894678 - **Role:** CONTACT **Country:** France **Facility:** CHU Clermont-Ferrand **State:** Not Required For This Country **Status:** RECRUITING **Zip:** 63000 #### Overall Officials **Official 1:** **Affiliation:** University Hospital, Clermont-Ferrand **Name:** Matthieu JABAUDON **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000004194 - Term: Disease - ID: D000010335 - Term: Pathologic Processes - ID: D000008171 - Term: Lung Diseases - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000012120 - Term: Respiration Disorders - ID: D000007235 - Term: Infant, Premature, Diseases - ID: D000007232 - Term: Infant, Newborn, Diseases - ID: D000055370 - Term: Lung Injury ### Condition Browse Module - Browse Branches - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC26 - Name: Wounds and Injuries - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M14965 - Name: Respiratory Distress Syndrome - Relevance: HIGH - As Found: Respiratory Distress Syndrome - ID: M14964 - Name: Respiratory Distress Syndrome, Newborn - Relevance: HIGH - As Found: Respiratory Distress Syndrome - ID: M16355 - Name: Syndrome - Relevance: HIGH - As Found: Syndrome - ID: M19010 - Name: Critical Illness - Relevance: LOW - As Found: Unknown - ID: M27137 - Name: Respiratory Aspiration - Relevance: LOW - As Found: Unknown - ID: M28144 - Name: Acute Lung Injury - Relevance: HIGH - As Found: Acute Respiratory Distress Syndrome - ID: M28143 - Name: Lung Injury - Relevance: LOW - As Found: Unknown - ID: M11168 - Name: Lung Diseases - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown - ID: M14957 - Name: Respiration Disorders - Relevance: LOW - As Found: Unknown - ID: M25869 - Name: Premature Birth - Relevance: LOW - As Found: Unknown - ID: M10279 - Name: Infant, Premature, Diseases - Relevance: LOW - As Found: Unknown - ID: M10276 - Name: Infant, Newborn, Diseases - Relevance: LOW - As Found: Unknown - ID: T4927 - Name: Respiratory Distress Syndrome, Infant - Relevance: HIGH - As Found: Respiratory Distress Syndrome - ID: T192 - Name: Acute Respiratory Distress Syndrome - Relevance: HIGH - As Found: Acute Respiratory Distress Syndrome - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000012128 - Term: Respiratory Distress Syndrome - ID: D000012127 - Term: Respiratory Distress Syndrome, Newborn - ID: D000055371 - Term: Acute Lung Injury - ID: D000013577 - Term: Syndrome ### Intervention Browse Module - Ancestors - ID: D000010975 - Term: Platelet Aggregation Inhibitors - ID: D000018685 - Term: Anesthetics, Inhalation - ID: D000018681 - Term: Anesthetics, General - ID: D000000777 - Term: Anesthetics - ID: D000002492 - Term: Central Nervous System Depressants - ID: D000045505 - Term: Physiological Effects of Drugs ### Intervention Browse Module - Browse Branches - Abbrev: Analg - Name: Analgesics - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: PlAggInh - Name: Platelet Aggregation Inhibitors ### Intervention Browse Module - Browse Leaves - ID: M22662 - Name: Dexmedetomidine - Relevance: LOW - As Found: Unknown - ID: M18307 - Name: Propofol - Relevance: LOW - As Found: Unknown - ID: M4107 - Name: Anesthetics - Relevance: LOW - As Found: Unknown - ID: M1673 - Name: Sevoflurane - Relevance: HIGH - As Found: Metabolic - ID: M4032 - Name: Analgesics - Relevance: LOW - As Found: Unknown - ID: M10043 - Name: Hypnotics and Sedatives - Relevance: LOW - As Found: Unknown - ID: M13865 - Name: Platelet Aggregation Inhibitors - Relevance: LOW - As Found: Unknown - ID: M20765 - Name: Anesthetics, Inhalation - Relevance: LOW - As Found: Unknown - ID: M20761 - Name: Anesthetics, General - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000077149 - Term: Sevoflurane ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01070979 **Brief Title:** Hormone Replacement Therapy for Use in Postmenopausal Women for Relief of Hot Flushes and Urogenital Symptoms. **Official Title:** A Multicenter, Double-Blind, Controlled, Randomized Study to Compare the Efficacy in Relief of Hot Flushes in Women Receiving Oral Estradiol Acetate Tablets, Oral Estradiol Tablets or Oral Conjugated Equine Estrogens #### Organization Study ID Info **ID:** PR-03602.1 #### Organization **Class:** INDUSTRY **Full Name:** Warner Chilcott ### Status Module #### Completion Date **Date:** 2003-09 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2013-04-22 **Type:** ESTIMATED **Last Update Submit Date:** 2013-04-15 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2003-09 **Type:** ACTUAL #### Results First Post Date **Date:** 2011-04-08 **Type:** ESTIMATED **Results First Submit Date:** 2011-02-14 **Results First Submit QC Date:** 2011-03-09 #### Start Date **Date:** 2003-02 **Status Verified Date:** 2013-04 #### Study First Post Date **Date:** 2010-02-18 **Type:** ESTIMATED **Study First Submit Date:** 2010-02-17 **Study First Submit QC Date:** 2010-02-17 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Warner Chilcott #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Multicenter, double-blind, controlled, parallel group, randomized study to compare the clinical benefit of Estradiol acetate tablets, estradiol tablets and conjugated equine estrogen tablets, each administered orally, once daily, to postmenopausal women. ### Conditions Module **Conditions:** - Hormone Replacement Therapy ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** QUADRUPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 249 **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Drug: Estradiol acetate **Label:** Estradiol acetate (E3A) **Type:** EXPERIMENTAL #### Arm Group 2 **Intervention Names:** - Drug: Estradiol **Label:** Estradiol **Type:** ACTIVE_COMPARATOR #### Arm Group 3 **Intervention Names:** - Drug: Conjugated equine estrogens **Label:** Conjugated equine estrogens (CEE): **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Estradiol acetate (E3A) **Description:** Tablet containing 0.9 mg E3A, daily oral administration. **Name:** Estradiol acetate **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Estradiol **Description:** Tablet containing 1 mg estradiol, daily oral administration. **Name:** Estradiol **Other Names:** - Estrace **Type:** DRUG #### Intervention 3 **Arm Group Labels:** - Conjugated equine estrogens (CEE): **Description:** Tablet containing 0.625 mg CEE, daily oral administration. **Name:** Conjugated equine estrogens **Other Names:** - Premarin **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Severity of hot flush definitions: mild - sensation of heat without perspiration, moderate - sensation of heat with perspiration, able to continue activity, severe - sensation of heat with perspiration, causing the subject to stop activity or awaken from sleep **Measure:** Mean Change From Baseline in the Number of Moderate to Severe Hot Flushes, Week 4, ITT (Intention to Treat) Population **Time Frame:** Baseline to Week 4 **Description:** Severity of hot flush definitions: mild - sensation of heat without perspiration, moderate - sensation of heat with perspiration, able to continue activity, severe - sensation of heat with perspiration, causing the subject to stop activity or awaken from sleep **Measure:** Mean Change From Baseline in the Number of Moderate to Severe Hot Flushes, Week 12, ITT Population **Time Frame:** Baseline to Week 12 #### Secondary Outcomes **Description:** Patient self-reported outcome. Severity of hot flush definitions: mild (1) - sensation of heat without perspiration, moderate (2) - sensation of heat with perspiration, able to continue activity, severe (3) - sensation of heat with perspiration, causing the subject to stop activity or awaken from sleep. Minimum 0/no hot flushes, Maximum 3/all severe hot flushes. Lower the score the greater the improvement in reducing hot flushes. **Measure:** Mean Change From Baseline in the Severity of Moderate to Severe Hot Flushes, Week 4, ITT Population **Time Frame:** Baseline to Week 4 **Description:** Patient self-reported outcome. Severity of hot flush definitions: mild (1) - sensation of heat without perspiration, moderate (2) - sensation of heat with perspiration, able to continue activity, severe (3) - sensation of heat with perspiration, causing the subject to stop activity or awaken from sleep. Minimum 0/no hot flushes, Maximum 3/all severe hot flushes. Lower the score the greater the improvement in reducing hot flushes. **Measure:** Mean Change From Baseline in the Severity of Moderate to Severe Hot Flushes, Week 12, ITT Population **Time Frame:** Baseline to Week 12 **Description:** Urogenital Symptom Severity scored none=0, mild=1, moderate=2, severe=3. **Measure:** Mean Change From Baseline in Total Urogenital Symptom Score, Week 4, ITT Population **Time Frame:** Baseline to Week 4 **Description:** Urogenital Symptom Severity scored none=0, mild=1, moderate=2, severe=3. **Measure:** Change From Baseline in Total Urogenital Symptom Score, Week 8, ITT Population **Time Frame:** Baseline to Week 8 **Description:** Urogenital Symptom Severity scored none=0, mild=1, moderate=2, severe=3. **Measure:** Change From Baseline in Total Urogenital Symptom Score, Week 12, ITT Population **Time Frame:** Baseline to Week 12 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Age ≥ 40 years of age; bilateral oophorectomy ≥ 35 years of age. 2. Non-hysterectomized women: * Amenorrhea for ≥ 12 months or * Amenorrhea for ≤ 12 months, but longer than 6 months, and serum FSH (follicle stimulating hormone) levels \> 40 units/L and serum estradiol levels \< 20 pg /mL, Hysterectomized women: * Bilateral oophorectomy - subjects may enter the study 6 weeks after surgery or * History of removal of ovaries may be confirmed by - serum FSH levels \> 40 units/L and serum estradiol levels \< 20 pg/mL or via surgical report / ultrasound. 3. Seven or more moderate or severe hot flushes daily for 1 week or 60 or more moderate or severe flushes in 1 week during the 2 week screening period prior to study entry. Exclusion Criteria: 1. Hormone therapy administered via the following routes and during the specified timeframes: oral within 8 weeks, vaginal (rings, creams, gels) within 1 week, transdermal within 4 weeks, intramuscular within 6 weeks, progestational implants, estrogen or estrogen/progestational injectable drug therapy within 3 months, estrogen pellet or progestational injectable within 6 months. 2. Abnormal Pap smear suggestive of low grade squamous intraepithelial lesion (LGSIL) or worse. Enrollment of subjects with an ASCUS (atypical squamous cells of undetermined significance) interpretation must be discussed with the sponsor prior to randomization. 3. Urinary tract infection 4. Congestive heart failure 5. Uncontrolled hypertension; sitting systolic BP ≥ 160 mmHg or diastolic ≥ 95 mmHg 6. History of stroke or transient ischemic attacks 7. Treatment with anticoagulants (heparin or warfarin). 8. Uncontrolled thyroid disorders. 9. Insulin-dependent diabetes mellitus. 10. Increase frequency or severity of headaches including migraines during previous estrogen therapy. **Minimum Age:** 35 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Phoenix **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Arizona **Zip:** 85031 **Location 2:** **City:** Carmichael **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** California **Zip:** 95608 **Location 3:** **City:** San Diego **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** California **Zip:** 92108 **Location 4:** **City:** San Diego **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** California **Zip:** 92123 **Location 5:** **City:** Aventura **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Florida **Zip:** 33180 **Location 6:** **City:** Boynton Beach **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Florida **Zip:** 33437 **Location 7:** **City:** Clearwater **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Florida **Zip:** 33765 **Location 8:** **City:** Daytona Beach **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Florida **Zip:** 32114 **Location 9:** **City:** Gainesville **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Florida **Zip:** 32605 **Location 10:** **City:** Longwood **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Florida **Zip:** 32779 **Location 11:** **City:** Melbourne **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Florida **Zip:** 32935 **Location 12:** **City:** Miami **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Florida **Zip:** 33143 **Location 13:** **City:** Palm Springs **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Florida **Zip:** 33461 **Location 14:** **City:** Pinellas Park **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Florida **Zip:** 33781 **Location 15:** **City:** Sarasota **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Florida **Zip:** 34232 **Location 16:** **City:** Venice **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Florida **Zip:** 34285 **Location 17:** **City:** Roswell **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Georgia **Zip:** 30075 **Location 18:** **City:** Chicago **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Illinois **Zip:** 60612 **Location 19:** **City:** Peoria **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Illinois **Zip:** 61615 **Location 20:** **City:** Laurel **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Maryland **Zip:** 20707 **Location 21:** **City:** Lincoln **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Nebraska **Zip:** 68510 **Location 22:** **City:** Raleigh **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** North Carolina **Zip:** 27612 **Location 23:** **City:** Winston Salem **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** North Carolina **Zip:** 27103 **Location 24:** **City:** Cleveland **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Ohio **Zip:** 44122 **Location 25:** **City:** Columbus **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Ohio **Zip:** 43212 **Location 26:** **City:** Mogadore **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Ohio **Zip:** 44260 **Location 27:** **City:** Portland **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Oregon **Zip:** 97201 **Location 28:** **City:** Pittsburgh **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Pennsylvania **Zip:** 15206 **Location 29:** **City:** Nashville **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Tennessee **Zip:** 37203 **Location 30:** **City:** Salt Lake City **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Utah **Zip:** 84124 **Location 31:** **City:** Spokane **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Washington **Zip:** 99201 **Location 32:** **City:** Tacoma **Country:** United States **Facility:** Warner Chilcott Investigational Site **State:** Washington **Zip:** 98405 #### Overall Officials **Official 1:** **Affiliation:** Warner Chilcott **Name:** Herman Ellman, MD **Role:** STUDY_DIRECTOR ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M8610 - Name: Flushing - Relevance: LOW - As Found: Unknown - ID: M21519 - Name: Hot Flashes - Relevance: HIGH - As Found: Hot Flushes ### Condition Browse Module - Meshes - ID: D000019584 - Term: Hot Flashes ### Intervention Browse Module - Ancestors - ID: D000006728 - Term: Hormones - ID: D000006730 - Term: Hormones, Hormone Substitutes, and Hormone Antagonists - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000080066 - Term: Contraceptive Agents, Hormonal - ID: D000003270 - Term: Contraceptive Agents - ID: D000012102 - Term: Reproductive Control Agents - ID: D000003271 - Term: Contraceptive Agents, Female ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Repr - Name: Reproductive Control Agents ### Intervention Browse Module - Browse Leaves - ID: M9789 - Name: Hormones - Relevance: LOW - As Found: Unknown - ID: M8116 - Name: Estrogens - Relevance: HIGH - As Found: Interviewing - ID: M266279 - Name: Estradiol 17 beta-cypionate - Relevance: HIGH - As Found: Cell lymphoma - ID: M266280 - Name: Estradiol 3-benzoate - Relevance: HIGH - As Found: Cell lymphoma - ID: M8108 - Name: Estradiol - Relevance: HIGH - As Found: Cell lymphoma - ID: M234941 - Name: Polyestradiol phosphate - Relevance: HIGH - As Found: Cell lymphoma - ID: M8115 - Name: Estrogens, Conjugated (USP) - Relevance: HIGH - As Found: Golytely - ID: M9788 - Name: Hormone Antagonists - Relevance: LOW - As Found: Unknown - ID: M6494 - Name: Contraceptive Agents - Relevance: LOW - As Found: Unknown - ID: M2116 - Name: Contraceptive Agents, Hormonal - Relevance: LOW - As Found: Unknown - ID: M6495 - Name: Contraceptive Agents, Female - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: C000007630 - Term: Estradiol 17 beta-cypionate - ID: C000074283 - Term: Estradiol 3-benzoate - ID: D000004958 - Term: Estradiol - ID: C000008958 - Term: Polyestradiol phosphate - ID: D000004967 - Term: Estrogens - ID: D000004966 - Term: Estrogens, Conjugated (USP) ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** February 2003 thru September 2003, One subject randomized to estradiol group never took any study drug therefore not included in AE assessment. #### Event Groups **Group ID:** EG000 **Title:** Estradiol Acetate (E3A) **Description:** 1 tablet daily containing 0.9 mg estradiol acetate **ID:** EG000 **Other Num Affected:** 19 **Other Num at Risk:** 79 **Serious Number At Risk:** 79 **Title:** Estradiol Acetate (E3A) **Group ID:** EG001 **Title:** Estradiol **Description:** 1 tablet daily containing 1 mg estradiol **ID:** EG001 **Other Num Affected:** 25 **Other Num at Risk:** 84 **Serious Number Affected:** 1 **Serious Number At Risk:** 84 **Title:** Estradiol **Group ID:** EG002 **Title:** Conjugated Equine Estrogens (CEE) **Description:** 1 tablet daily containing 0.625 mg conjugated equine estrogen **ID:** EG002 **Other Num Affected:** 30 **Other Num at Risk:** 85 **Serious Number At Risk:** 85 **Title:** Conjugated Equine Estrogens (CEE) **Frequency Threshold:** 3 #### Other Events **Term:** Breast Tenderness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Reproductive system and breast disorders **Source Vocabulary:** MedDRA (6.0) **Term:** Nausea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (6.0) **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (6.0) **Term:** Metrorrhagia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Reproductive system and breast disorders **Source Vocabulary:** MedDRA (6.0) **Term:** Abdominal Distention **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (6.0) **Term:** Vaginosis Fungal **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (6.0) **Term:** Weight Increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (6.0) **Term:** Arthralgia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (6.0) **Term:** Nasopharyngitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (6.0) **Term:** Anxiety **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA (6.0) #### Serious Events **Term:** metrorrhagia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Reproductive system and breast disorders **Source Vocabulary:** MedDRA (6.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 79 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 84 **Num Events:** 1 **Group ID:** EG002 **Num At Risk:** 85 **Time Frame:** 7 months ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 79 **Group ID:** BG001 **Value:** 85 **Group ID:** BG002 **Value:** 85 **Group ID:** BG003 **Value:** 249 **Units:** Participants ### Group **ID:** BG000 **Title:** Estradiol Acetate (E3A) **Description:** 1 tablet daily containing 0.9 mg estradiol acetate ### Group **ID:** BG001 **Title:** Estradiol **Description:** 1 tablet daily containing 1 mg estradiol ### Group **ID:** BG002 **Title:** Conjugated Equine Estrogens (CEE) **Description:** 1 tablet daily containing 0.625 mg conjugated equine estrogen ### Group **ID:** BG003 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 6.6 **Value:** 52.2 #### Measurement **Group ID:** BG001 **Spread:** 7.0 **Value:** 52.3 #### Measurement **Group ID:** BG002 **Spread:** 6.3 **Value:** 53.8 #### Measurement **Group ID:** BG003 **Spread:** 6.7 **Value:** 52.8 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 79 #### Measurement **Group ID:** BG001 **Value:** 85 #### Measurement **Group ID:** BG002 **Value:** 85 #### Measurement **Group ID:** BG003 **Value:** 249 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 0 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 79 #### Measurement **Group ID:** BG001 **Value:** 85 #### Measurement **Group ID:** BG002 **Value:** 85 #### Measurement **Group ID:** BG003 **Value:** 249 **Class Title:** United States **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age Continuous **Unit of Measure:** years ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** participants ## Results Section - More Information Module ### Certain Agreement **Restriction Type:** LTE60 **Restrictive Agreement:** True ### Point of Contact **Email:** [email protected] **Organization:** Warner Chilcott **Phone:** 973-442-3376 **Title:** Grexan Wulff, Manager Regulatory Affairs ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### Outcome Measure 4 ### Outcome Measure 5 ### Outcome Measure 6 ### Outcome Measure 7 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 4.5 - **Upper Limit:** - **Value:** -54.1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0.721 - **Spread:** 4.5 - **Upper Limit:** 1.048 - **Value:** -62.0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** 0.833 - **Spread:** 4.3 - **Upper Limit:** 1.178 - **Value:** -54.5 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.106 - **Upper Limit:** - **Value:** -0.53 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.106 - **Upper Limit:** - **Value:** -0.51 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** 0.102 - **Upper Limit:** - **Value:** -0.59 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 4.8 - **Upper Limit:** - **Value:** -63.6 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 4.7 - **Upper Limit:** - **Value:** -72.2 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** 4.6 - **Upper Limit:** - **Value:** -67.2 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.127 - **Upper Limit:** - **Value:** -1.05 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.125 - **Upper Limit:** - **Value:** -1.34 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** 0.122 - **Upper Limit:** - **Value:** -1.17 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.413 - **Upper Limit:** - **Value:** -1.89 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.407 - **Upper Limit:** - **Value:** -2.26 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** 0.398 - **Upper Limit:** - **Value:** -1.96 **Title:** #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.432 - **Upper Limit:** - **Value:** -1.96 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.425 - **Upper Limit:** - **Value:** -2.58 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** 0.416 - **Upper Limit:** - **Value:** -2.42 **Title:** #### Outcome Measure 7 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.437 - **Upper Limit:** - **Value:** -2.49 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.431 - **Upper Limit:** - **Value:** -2.59 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** 0.422 - **Upper Limit:** - **Value:** -2.52 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Severity of hot flush definitions: mild - sensation of heat without perspiration, moderate - sensation of heat with perspiration, able to continue activity, severe - sensation of heat with perspiration, causing the subject to stop activity or awaken from sleep **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Intention to Treat (ITT) with Last Observation Carried Forward (LOCF) **Reporting Status:** POSTED **Time Frame:** Baseline to Week 4 **Title:** Mean Change From Baseline in the Number of Moderate to Severe Hot Flushes, Week 4, ITT (Intention to Treat) Population **Type:** PRIMARY **Unit of Measure:** Change in Hot Flush Count ##### Group **Description:** 1 tablet daily containing 0.9 mg estradiol acetate **ID:** OG000 **Title:** Estradiol Acetate (E3A) ##### Group **Description:** 1 tablet daily containing 1 mg estradiol **ID:** OG001 **Title:** Estradiol ##### Group **Description:** 1 tablet daily containing 0.625 mg conjugated equine estrogen **ID:** OG002 **Title:** Conjugated Equine Estrogens (CEE) #### Outcome Measure 2 **Description:** Patient self-reported outcome. Severity of hot flush definitions: mild (1) - sensation of heat without perspiration, moderate (2) - sensation of heat with perspiration, able to continue activity, severe (3) - sensation of heat with perspiration, causing the subject to stop activity or awaken from sleep. Minimum 0/no hot flushes, Maximum 3/all severe hot flushes. Lower the score the greater the improvement in reducing hot flushes. **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Intention to Treat (ITT) with Last Observation Carried Forward (LOCF) **Reporting Status:** POSTED **Time Frame:** Baseline to Week 4 **Title:** Mean Change From Baseline in the Severity of Moderate to Severe Hot Flushes, Week 4, ITT Population **Type:** SECONDARY **Unit of Measure:** Change in Score ##### Group **Description:** 1 tablet daily containing 0.9 mg estradiol acetate **ID:** OG000 **Title:** Estradiol Acetate (E3A) ##### Group **Description:** 1 tablet daily containing 1 mg estradiol **ID:** OG001 **Title:** Estradiol ##### Group **Description:** 1 tablet daily containing 0.625 mg conjugated equine estrogen **ID:** OG002 **Title:** Conjugated Equine Estrogens (CEE) #### Outcome Measure 3 **Description:** Severity of hot flush definitions: mild - sensation of heat without perspiration, moderate - sensation of heat with perspiration, able to continue activity, severe - sensation of heat with perspiration, causing the subject to stop activity or awaken from sleep **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Intention to Treat (ITT) with Last Observation Carried Forward (LOCF) **Reporting Status:** POSTED **Time Frame:** Baseline to Week 12 **Title:** Mean Change From Baseline in the Number of Moderate to Severe Hot Flushes, Week 12, ITT Population **Type:** PRIMARY **Unit of Measure:** Change in Hot Flush Count ##### Group **Description:** 1 tablet daily containing 0.9 mg estradiol acetate **ID:** OG000 **Title:** Estradiol Acetate (E3A) ##### Group **Description:** 1 tablet daily containing 1 mg estradiol **ID:** OG001 **Title:** Estradiol ##### Group **Description:** 1 tablet daily containing 0.625 mg conjugated equine estrogen **ID:** OG002 **Title:** Conjugated Equine Estrogens (CEE) #### Outcome Measure 4 **Description:** Patient self-reported outcome. Severity of hot flush definitions: mild (1) - sensation of heat without perspiration, moderate (2) - sensation of heat with perspiration, able to continue activity, severe (3) - sensation of heat with perspiration, causing the subject to stop activity or awaken from sleep. Minimum 0/no hot flushes, Maximum 3/all severe hot flushes. Lower the score the greater the improvement in reducing hot flushes. **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Intention to Treat (ITT) with Last Observation Carried Forward (LOCF) **Reporting Status:** POSTED **Time Frame:** Baseline to Week 12 **Title:** Mean Change From Baseline in the Severity of Moderate to Severe Hot Flushes, Week 12, ITT Population **Type:** SECONDARY **Unit of Measure:** Change in Score ##### Group **Description:** 1 tablet daily containing 0.9 mg estradiol acetate **ID:** OG000 **Title:** Estradiol Acetate (E3A) ##### Group **Description:** 1 tablet daily containing 1 mg estradiol **ID:** OG001 **Title:** Estradiol ##### Group **Description:** 1 tablet daily containing 0.625 mg conjugated equine estrogen **ID:** OG002 **Title:** Conjugated Equine Estrogens (CEE) #### Outcome Measure 5 **Description:** Urogenital Symptom Severity scored none=0, mild=1, moderate=2, severe=3. **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Intention to Treat (ITT) with Last Observation Carried Forward (LOCF) **Reporting Status:** POSTED **Time Frame:** Baseline to Week 4 **Title:** Mean Change From Baseline in Total Urogenital Symptom Score, Week 4, ITT Population **Type:** SECONDARY **Unit of Measure:** Change in Score ##### Group **Description:** 1 tablet daily containing 0.9 mg estradiol acetate **ID:** OG000 **Title:** Estradiol Acetate (E3A) ##### Group **Description:** 1 tablet daily containing 1 mg estradiol **ID:** OG001 **Title:** Estradiol ##### Group **Description:** 1 tablet daily containing 0.625 mg conjugated equine estrogen **ID:** OG002 **Title:** Conjugated Equine Estrogens (CEE) #### Outcome Measure 6 **Description:** Urogenital Symptom Severity scored none=0, mild=1, moderate=2, severe=3. **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Intention to Treat (ITT) with Last Observation Carried Forward (LOCF) **Reporting Status:** POSTED **Time Frame:** Baseline to Week 8 **Title:** Change From Baseline in Total Urogenital Symptom Score, Week 8, ITT Population **Type:** SECONDARY **Unit of Measure:** Change in Score ##### Group **Description:** 1 tablet daily containing 0.9 mg estradiol acetate **ID:** OG000 **Title:** Estradiol Acetate (E3A) ##### Group **Description:** 1 tablet daily containing 1 mg estradiol **ID:** OG001 **Title:** Estradiol ##### Group **Description:** 1 tablet daily containing 0.625 mg conjugated equine estrogen **ID:** OG002 **Title:** Conjugated Equine Estrogens (CEE) #### Outcome Measure 7 **Description:** Urogenital Symptom Severity scored none=0, mild=1, moderate=2, severe=3. **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Intention to Treat (ITT) with Last Observation Carried Forward (LOCF) **Reporting Status:** POSTED **Time Frame:** Baseline to Week 12 **Title:** Change From Baseline in Total Urogenital Symptom Score, Week 12, ITT Population **Type:** SECONDARY **Unit of Measure:** Change in Score ##### Group **Description:** 1 tablet daily containing 0.9 mg estradiol acetate **ID:** OG000 **Title:** Estradiol Acetate (E3A) ##### Group **Description:** 1 tablet daily containing 1 mg estradiol **ID:** OG001 **Title:** Estradiol ##### Group **Description:** 1 tablet daily containing 0.625 mg conjugated equine estrogen **ID:** OG002 **Title:** Conjugated Equine Estrogens (CEE) ### Participant Flow Module #### Group **Description:** 1 tablet daily containing 0.9 mg estradiol acetate **ID:** FG000 **Title:** Estradiol Acetate (E3A) #### Group **Description:** 1 tablet daily containing 1 mg estradiol **ID:** FG001 **Title:** Estradiol #### Group **Description:** 1 tablet daily containing 0.625 mg conjugated equine estrogen **ID:** FG002 **Title:** Conjugated Equine Estrogens (CEE) #### Period **Title:** Overall Study ##### Withdraw **Type:** Adverse Event ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ###### Reason **Group ID:** FG001 **Number of Subjects:** 3 ###### Reason **Group ID:** FG002 **Number of Subjects:** 3 ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ###### Reason **Group ID:** FG002 **Number of Subjects:** 1 ##### Withdraw **Type:** Protocol Violation ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 2 ###### Reason **Group ID:** FG002 **Number of Subjects:** 6 ##### Withdraw **Type:** Surgery, moving, personal, abn. labs ###### Reason **Group ID:** FG000 **Number of Subjects:** 4 ###### Reason **Group ID:** FG001 **Number of Subjects:** 4 ###### Reason **Group ID:** FG002 **Number of Subjects:** 2 ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 3 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ###### Reason **Group ID:** FG002 **Number of Subjects:** 1 ##### Withdraw **Type:** No study drug taken ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ###### Reason **Group ID:** FG002 **Number of Subjects:** 0 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 79 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 85 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 85 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 68 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 73 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 72 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 11 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 12 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 13 **Pre-Assignment Details:** Discontinue estrogen/hormone therapy prior to enrollment **Recruitment Details:** Enrollment of postmenopausal women for relief of hot flushes and urogenital symptoms beginning Feb '03 at 33 sites in the US. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT02948179 **Acronym:** ESPERANCE **Brief Title:** Using Preimplantation Genetic Diagnosis in Autosomal Dominant Polycystic Kidney Disease Patients: a Multicenter Clinical Trial **Official Title:** Efficacy and Safety of Preimplantation Genetic Diagnosis in Blocking Pathogenic Gene Inheritance for Autosomal Dominant Polycystic Kidney Disease: a Multicenter Clinical Trial #### Organization Study ID Info **ID:** CZKIPLA-ADPKD-003 #### Organization **Class:** OTHER **Full Name:** Shanghai Changzheng Hospital ### Status Module #### Completion Date **Date:** 2020-12-31 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-01-26 **Type:** ACTUAL **Last Update Submit Date:** 2021-01-25 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2020-11-30 **Type:** ACTUAL #### Start Date **Date:** 2016-09-02 **Type:** ACTUAL **Status Verified Date:** 2021-01 #### Study First Post Date **Date:** 2016-10-28 **Type:** ESTIMATED **Study First Submit Date:** 2016-10-20 **Study First Submit QC Date:** 2016-10-26 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** The First Affiliated Hospital of Anhui Medical University **Class:** OTHER **Name:** The First Affiliated Hospital with Nanjing Medical University **Class:** OTHER_GOV **Name:** Shandong Provincial Hospital **Class:** OTHER **Name:** Tang-Du Hospital **Class:** OTHER **Name:** Shengjing Hospital **Class:** OTHER **Name:** Second Xiangya Hospital of Central South University **Class:** OTHER **Name:** Reproductive & Genetic Hospital of CITIC-Xiangya **Class:** OTHER **Name:** Peking University Third Hospital **Class:** OTHER **Name:** Navy General Hospital, Beijing **Class:** OTHER **Name:** The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School **Class:** OTHER **Name:** The First Affiliated Hospital of Soochow University **Class:** OTHER **Name:** Sir Run Run Shaw Hospital **Class:** OTHER **Name:** Fuzhou General Hospital **Class:** OTHER **Name:** The First Affiliated Hospital of Zhengzhou University **Class:** OTHER **Name:** Wuhan TongJi Hospital **Class:** OTHER **Name:** Wuhan Union Hospital, China **Class:** OTHER **Name:** Renmin Hospital of Wuhan University **Class:** OTHER **Name:** Xiangya Hospital of Central South University **Class:** OTHER **Name:** First Affiliated Hospital, Sun Yat-Sen University **Class:** OTHER **Name:** Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University **Class:** OTHER **Name:** Southwest Hospital, China **Class:** OTHER **Name:** LanZhou University **Class:** OTHER **Name:** Shaanxi Provincial People's Hospital **Class:** OTHER **Name:** West China Hospital **Class:** OTHER **Name:** West China Second University Hospital **Class:** OTHER **Name:** Sichuan Provincial People's Hospital **Class:** OTHER **Name:** Hebei Medical University Third Hospital **Class:** UNKNOWN **Name:** Hebei Province Center for Reproductive Medicine **Class:** OTHER **Name:** The Second Hospital of Hebei Medical University **Class:** UNKNOWN **Name:** Hospital for Reproductive Medicine Affiliated to Shandong University #### Lead Sponsor **Class:** OTHER **Name:** Changlin Mei #### Responsible Party **Investigator Affiliation:** Shanghai Changzheng Hospital **Investigator Full Name:** Changlin Mei **Investigator Title:** Professor, Director of kidney Institute **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** Autosomal dominant polycystic kidney disease (ADPKD) is the most common monogenic hereditary kidney disease in humans. ADPKD may affect all the generations of the ADPKD family and the probability of ADPKD is 50% in the second generation for each gender. It has been confirmed that PKD1 and PKD2 are two pathogenic genes of ADPKD. Nowadays, the investigators have established an effective gene detection technology platform for PKD1/2 gene with long fragment PCR and next generation sequencing. First, the investigators performed genetic testing in patients with clinically diagnosed ADPKD and strong fertility desire, but afraid of hereditary risk. Using Preimplantation genetic diagnosis, including multiple annealing and looping-based amplification cycles amplification technique, the investigators successfully screened out healthy embryos by In Vitro Fertilization. Then the investigators transplanted embryos returned to the parent. When the baby is born, using umbilical cord blood gene detection, the investigators confirmed that the neonates do not inherit genetic defects form parents. The investigators have succeeded in one couple. The investigators design a multicenter clinical trial to confirm those procedures efficacy and safety. ### Conditions Module **Conditions:** - Polycystic Kidney, Type 1 Autosomal Dominant Disease ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 459 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** ADPKD patients will complete the whole process of preimplantation genetic diagnosis with healthy baby without pathogenic gene inheritance. **Intervention Names:** - Procedure: Preimplantation Genetic Diagnosis **Label:** Preimplantation genetic diagnosis group **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** ADPKD patients, pathogenic mutations in PKD1, have natural pregnancy without preimplantation genetic diagnosis.The investigators will perform genetic tests on the blood or umbilical cord blood of infants born between January 2014 and June 2020. **Label:** Natural pregnancy group **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Preimplantation genetic diagnosis group **Description:** Using Preimplantation genetic diagnosis, including multiple annealing and looping-based amplification cycles amplification technique, the investigators have had screened out healthy embryos by In Vitro Fertilization. Then the investigators transplanted embryos returned to the parent. Finally, participants will have healthy baby without pathogenic gene inheritance. **Name:** Preimplantation Genetic Diagnosis **Type:** PROCEDURE ### Outcomes Module #### Other Outcomes **Description:** the incidence of ovarian hyperstimulation syndrome **Measure:** The incidence of ovarian hyperstimulation syndrome **Time Frame:** through study completion, an average of 2 years **Description:** the incidence of organ injuries **Measure:** The incidence of organ injuries **Time Frame:** through study completion, an average of 2 years **Description:** the incidence of infection **Measure:** The incidence of infection **Time Frame:** through study completion, an average of 2 years **Description:** the incidence of abortion rate **Measure:** The incidence of abortion rate **Time Frame:** through study completion, an average of 2 years #### Primary Outcomes **Description:** The investigators will do umbilical cord blood gene detection for the baby to confirm with or without pathogenic gene inheritance. The investigators will compare two groups of healthy newborns rate. **Measure:** Healthy baby Rate without pathogenic gene inheritance **Time Frame:** through study completion, an average of 2 year #### Secondary Outcomes **Description:** Using couples blood to do pretest of preimplantation genetic diagnosis in Peripheral blood mononuclear lymphocyte. **Measure:** Success rate in pretest of preimplantation genetic diagnosis **Time Frame:** through study completion, an average of 2 year **Description:** The rate of amniotic fluid puncture test or umbilical cord blood gene detection confirm the offspring containing pathogenic gene mutation. **Measure:** Technical failure rate of preimplantation genetic diagnosis. **Time Frame:** Amniotic fluid puncture test (pregnancy 16 to 19 weeks ) and Birth day **Description:** The proportion of good eggs obtained after ovulation induction **Measure:** Oocyte retrieval rate **Time Frame:** through study completion, an average of 2 years **Description:** The well-developed blastocyst ratio obtained after intracytoplasmic sperm injection **Measure:** Good quality embryo rate **Time Frame:** through study completion, an average of 2 years **Description:** Successful pregnancy rate of transplanted embryo **Measure:** Pregnancy rate **Time Frame:** Four weeks after embryo transplantation **Description:** Healthy newborn birth rate in preimplantation genetic diagnosis group **Measure:** Take home baby rate **Time Frame:** Two week after neonatus birth day **Description:** The investigators do twice kidney MRI scan for calculating total kidney volume change rate between enroll and postpartum 6 months. **Measure:** The total kidney volume change rate **Time Frame:** From enroll to postpartum 6 months **Description:** The investigators do twice serum creatinine test between enroll and postpartum 6 months, then using CKD-EPI formula to calculate eGFR. **Measure:** The estimated glomerular filtration rate change **Time Frame:** From enroll to postpartum 6 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Only one ADPKD patient in one couple without gender limitation * Wife has age limitation from 20 years to 35 years * ADPKD ADPKD diagnosis with or without family history * Find out specific pathogenic mutations in the PKD1 gene with at least one of the following: one of family patients done kidney transplantation or renal replacement therapy before 58 years old; one of family patients died of complications before 55 years old; the patient with total kidney volume more than 650ml; the patient with total kidney volume increase rate more than 6% every year; the patient's PKD1 mutation belongs to truncated gene mutation. * Both husband and wife have assisted reproductive conditions and will * Pregnancy compliance with Chinese laws * Signed informed consent Exclusion Criteria: * Active pathogenic microorganism infection, such as hepatitis B or C, HIV, pulmonary tuberculosis, giant cell virus, fungi or other contraindications for preimplantation genetic diagnosis and so on * Any one of the couple has used any drugs which may lead to abnormal reproductive system function, reproductive cell abnormalities, pregnancy risk increases in the past 3 months, or has history of drug abuse * Any one of the couple has malignancy * The wife has uncontrolled hypertension or refractory hypertension * The wife has diabetes mellitus * The wife has albuminuria * The wife has autoimmune disease * The wife has other disorders or functional abnormalities, such as liver or renal dysfunction, which may be aggravated by pregnancy or assisted reproduction * Allergy to drugs or related products which cannot avoid in our study * Participating in other clinical studies in last 3 months * Participants cannot follow the study program * Other conditions that the researchers considered unsuitable for participation **Maximum Age:** 35 Years **Minimum Age:** 20 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Hefei **Country:** China **Facility:** The First Affiliated Hospital of Anhui Medical University **State:** Anhui **Zip:** 230000 **Location 2:** **City:** Beijing **Country:** China **Facility:** Navy General Hospital **State:** Beijing **Zip:** 100000 **Location 3:** **City:** Beijing **Country:** China **Facility:** Peking University Third Hospital **State:** Beijing **Zip:** 100000 **Location 4:** **City:** Chongqing **Country:** China **Facility:** Southwest Hospital **State:** Chongqing **Zip:** 400000 **Location 5:** **City:** Fuzhou **Country:** China **Facility:** Fuzhou General Hospital **State:** Fujian **Zip:** 350000 **Location 6:** **City:** Lanzhou **Country:** China **Facility:** The First Hospital of Lanzhou University **State:** Gansu **Zip:** 730000 **Location 7:** **City:** Guangzhou **Country:** China **Facility:** Sun Yat-sen Memorial Hospital of Sun Yat-sen University **State:** Guangdong **Zip:** 510000 **Location 8:** **City:** Guangzhou **Country:** China **Facility:** The First Affiliated Hospital of Sun Yat-sen University **State:** Guangdong **Zip:** 510000 **Location 9:** **City:** Shijiazhuang **Country:** China **Facility:** Hebei Province Center for Reproductive Medicine **State:** Hebei **Zip:** 050000 **Location 10:** **City:** Shijiazhuang **Country:** China **Facility:** The Second Hospital of Hebei Medical University **State:** Hebei **Zip:** 050000 **Location 11:** **City:** Shijiazhuang **Country:** China **Facility:** The Third Hospital of Hebei Medical University **State:** Hebei **Zip:** 050000 **Location 12:** **City:** Zhengzhou **Country:** China **Facility:** The First Affiliated Hospital of Zhengzhou University **State:** Henan **Zip:** 450000 **Location 13:** **City:** Wuhan **Country:** China **Facility:** Renmin Hospital of Wuhan University **State:** Hubei **Zip:** 430000 **Location 14:** **City:** Wuhan **Country:** China **Facility:** Wuhan Tongji Hospital **State:** Hubei **Zip:** 430000 **Location 15:** **City:** Wuhan **Country:** China **Facility:** Wuhan Union Hospital **State:** Hubei **Zip:** 430000 **Location 16:** **City:** Changsha **Country:** China **Facility:** Reproductive & Genetic Hospital of CITIC-Xiangya **State:** Hunan **Zip:** 410000 **Location 17:** **City:** Changsha **Country:** China **Facility:** Second Xiangya Hospital of Central South University **State:** Hunan **Zip:** 410000 **Location 18:** **City:** Changsha **Country:** China **Facility:** Xiangya Hospital of Central South University **State:** Hunan **Zip:** 410000 **Location 19:** **City:** Nanjing **Country:** China **Facility:** The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School **State:** Jiangsu **Zip:** 210000 **Location 20:** **City:** Nanjing **Country:** China **Facility:** The First Affiliated Hospital with Nanjing Medical University **State:** Jiangsu **Zip:** 210000 **Location 21:** **City:** Suzhou **Country:** China **Facility:** The First Affiliated Hospital of Soochow University **State:** Jiangsu **Zip:** 215000 **Location 22:** **City:** Shenyang **Country:** China **Facility:** Shengjing Hospital **State:** Liaoning **Zip:** 110000 **Location 23:** **City:** Jinan **Country:** China **Facility:** Hospital for Reproductive Medicine Affiliated to Shandong University **State:** Shandong **Zip:** 250000 **Location 24:** **City:** Jinan **Country:** China **Facility:** Shandong Provincial Hospital **State:** Shandong **Zip:** 250000 **Location 25:** **City:** Shanghai **Country:** China **Facility:** Shanghai Changzheng Hospital **State:** Shanghai **Zip:** 200000 **Location 26:** **City:** Taiyuan **Country:** China **Facility:** Shanxi Provincial People's Hospital **State:** Shanxi **Zip:** 030000 **Location 27:** **City:** Xi'an **Country:** China **Facility:** Tang-Du Hospital **State:** Shanxi **Zip:** 710000 **Location 28:** **City:** Chengdu **Country:** China **Facility:** Shanxi Provincial People's Hospital **State:** Sichuan **Zip:** 610000 **Location 29:** **City:** Chengdu **Country:** China **Facility:** West China Hospital of Sichuan University **State:** Sichuan **Zip:** 610000 **Location 30:** **City:** Chengdu **Country:** China **Facility:** West China Second University Hospital **State:** Sichuan **Zip:** 610000 **Location 31:** **City:** Hangzhou **Country:** China **Facility:** Sir Run Run Shaw Hospital of Zhejiang University **State:** Zhejiang **Zip:** 310000 #### Overall Officials **Official 1:** **Affiliation:** Institute of Nephrology, Changzheng Hospital **Name:** changlin Mei, Master **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** Center of Reproductive Medicine, Changzheng Hospital **Name:** wen Li, doctor **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014570 - Term: Urologic Diseases - ID: D000052776 - Term: Female Urogenital Diseases - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications - ID: D000091642 - Term: Urogenital Diseases - ID: D000052801 - Term: Male Urogenital Diseases - ID: D000007592 - Term: Joint Diseases - ID: D000009140 - Term: Musculoskeletal Diseases - ID: D000009135 - Term: Muscular Diseases - ID: D000009139 - Term: Musculoskeletal Abnormalities - ID: D000000013 - Term: Congenital Abnormalities - ID: D000052177 - Term: Kidney Diseases, Cystic - ID: D000000015 - Term: Abnormalities, Multiple - ID: D000072661 - Term: Ciliopathies - ID: D000030342 - Term: Genetic Diseases, Inborn ### Condition Browse Module - Browse Branches - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: All - Name: All Conditions - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M10698 - Name: Kidney Diseases - Relevance: HIGH - As Found: Kidney Disease - ID: M10712 - Name: Polycystic Kidney Diseases - Relevance: HIGH - As Found: Polycystic Kidney - ID: M19237 - Name: Polycystic Kidney, Autosomal Dominant - Relevance: HIGH - As Found: Polycystic Kidney, Type 1 Autosomal Dominant Disease - ID: M4484 - Name: Arthrogryposis - Relevance: HIGH - As Found: Autosomal Dominant - ID: M17319 - Name: Urologic Diseases - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M27095 - Name: Male Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M10621 - Name: Joint Diseases - Relevance: LOW - As Found: Unknown - ID: M12097 - Name: Musculoskeletal Diseases - Relevance: LOW - As Found: Unknown - ID: M12092 - Name: Muscular Diseases - Relevance: LOW - As Found: Unknown - ID: M12 - Name: Congenital Abnormalities - Relevance: LOW - As Found: Unknown - ID: M12096 - Name: Musculoskeletal Abnormalities - Relevance: LOW - As Found: Unknown - ID: M26983 - Name: Kidney Diseases, Cystic - Relevance: LOW - As Found: Unknown - ID: M14 - Name: Abnormalities, Multiple - Relevance: LOW - As Found: Unknown - ID: M1076 - Name: Ciliopathies - Relevance: LOW - As Found: Unknown - ID: M23686 - Name: Genetic Diseases, Inborn - Relevance: LOW - As Found: Unknown - ID: T591 - Name: Autosomal Dominant Multiple Pterygium Syndrome - Relevance: HIGH - As Found: Autosomal Dominant - ID: T493 - Name: Arthrogryposis Multiplex Congenita - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001176 - Term: Arthrogryposis - ID: D000007674 - Term: Kidney Diseases - ID: D000007690 - Term: Polycystic Kidney Diseases - ID: D000016891 - Term: Polycystic Kidney, Autosomal Dominant ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06326879 **Acronym:** EOrOS **Brief Title:** A Comparative Study Between Early Onset Colorectal Cancer and Late Onset Colorectal Cancer Patients **Official Title:** Early-Onset Colorectal Cancer Versus Late-Onset Colorectal Cancer: a Prospective Observational Cohort Study to Explore the Clinical, Sociodemographic, Genetic, and Molecular Characteristics Associated With Adverse Oncological Outcomes ( #### Organization Study ID Info **ID:** 3527 #### Organization **Class:** OTHER **Full Name:** Istituto Clinico Humanitas ### Status Module #### Completion Date **Date:** 2027-10-30 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-03-25 **Type:** ACTUAL **Last Update Submit Date:** 2024-03-22 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2026-12-31 **Type:** ESTIMATED #### Start Date **Date:** 2023-11-30 **Type:** ACTUAL **Status Verified Date:** 2024-03 #### Study First Post Date **Date:** 2024-03-22 **Type:** ACTUAL **Study First Submit Date:** 2024-03-18 **Study First Submit QC Date:** 2024-03-18 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Istituto Clinico Humanitas #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** This study aims to investigate the clinical, socioeconomic, behavioral, genetic, and molecular factors characterizing Early Onset Colorectal Cancer (EOCRC) patients compared with Late Onset Colorectal Cancer (LOCRC) patients **Detailed Description:** Early Onset Colorectal Cancer (EOCRC), defined as a Colorectal Cancer (CRC) arising before the age of 50, is increasing and displays more aggressive features compared with Late Onset Colorectal Cancer (LOCRC, with a diagnosis after the age of 50). EOCRC patients have indeed a higher incidence rate of CRC recurrence after surgery compared with LOCRC, even at early CRC stages (stage I or II). The reasons underlying the more aggressive patterns are almost unknown but may span and can be represented by a combination of socioeconomic factors (including low economic income and access to screening programs, diagnosis, or therapy), behavioral and lifestyle factors (including diet, sedentary behaviors, or increased stress and anxiety), and molecular or genetic factors. The aim of this study is to prospectively validate our previous retrospective findings, demonstrating a higher incidence rate of CRC recurrence in early stage EOCRC, and explore the possible association between lifestyle, dietary, socioeconomics, molecular and genetic factors and postoperative CRC survival. ### Conditions Module **Conditions:** - Colorectal Cancer **Keywords:** - Early Onset Colorectal Cancer - Cancer survival - Genetic and molecular characterization ### Design Module #### Bio Spec **Description:** Serum samples collected before the surgical intervention, and six, twelve, and twenty-four months after surgery; fresh tumor sample collected at surgery; Formalin-Fixed Paraffin-Embedded (FFPE) tumor collected at surgery **Retention:** SAMPLES_WITH_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 340 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients aged less or equal to 49 years old at the time of colorectal cancer diagnosis **Intervention Names:** - Other: No intervention **Label:** Early Onset Colorectal Cancer (EOCRC) #### Arm Group 2 **Description:** Patients aged more or equal to 50 years old at the time of colorectal cancer diagnosis **Intervention Names:** - Other: No intervention **Label:** Late Onset Colorectal Cancer (LOCRC) ### Interventions #### Intervention 1 **Arm Group Labels:** - Early Onset Colorectal Cancer (EOCRC) - Late Onset Colorectal Cancer (LOCRC) **Description:** Questionnaires compilation and sample collection **Name:** No intervention **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Incidence rate ratio of 24-month colorectal cancer recurrence- defined as any radiological or endoscopic evidence of local recurrence or distal metastasis after the index surgery in stage I-III patients- between the study cohorts **Measure:** Incidence rate ratio of colorectal cancer recurrence **Time Frame:** 24 months after surgery #### Secondary Outcomes **Description:** Compare the association between physical activity, dietary, and lifestyle behaviors and 24-month colorectal cancer recurrence in the study cohorts. **Measure:** Correlation between lifestyle and incidence rate of colorectal cancer recurrence **Time Frame:** 24 months after surgery **Description:** Compare the association between the genetic profile of the surgical specimens, as identified by whole exome sequencing of the fresh and paraffin-embedded tumor specimen, and the 24-month CRC recurrence in the study cohorts **Measure:** Correlation between genetic profile and incidence rate of colorectal cancer recurrence **Time Frame:** 24 months after surgery **Description:** Compare the association between the molecular profile of the serum samples and surgical specimens, as identified by RNA sequencing and spatial transcriptomic analyses at different time points during the follow-up, and 24-month CRC recurrence between the study cohorts **Measure:** Correlation between molecular profile and incidence rate of colorectal cancer recurrence **Time Frame:** 24 months after surgery **Description:** Compare the association between socioeconomic characteristics, including access to health resources and inclusion in screening programs or experimental therapies, and 24-month CRC recurrence in the study cohorts. **Measure:** Correlation between socioeconomic characteristics and incidence rate of colorectal cancer recurrence **Time Frame:** 24 months after surgery ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patients aged more than 18 years old at the time of inclusion. * Patients with a proven diagnosis of colorectal adenocarcinoma, as reported by the staging histological biopsies. * Patients with a preoperative staging I-III. * Patients scheduled for elective curative colorectal resection. * Patients with rectal cancer who underwent neoadjuvant therapy can be included in the study. * Patients able and willing to comply with the protocol requirements (samples' collection and questionnaires' compilation). Exclusion Criteria: * Patients with metastatic disease at diagnosis. * Patients requiring an emergent procedure. * Patients undergoing palliative surgery (for example, fecal diversion). * Patients with a surgical indication for benign lesions (for example, adenoma or dysplasia). * Patients with an intraoperative finding of a lesion other than adenocarcinoma will be withdrawn from the study. * Patients with an intraoperative finding of distal metastasis or peritoneal carcinosis will be withdrawn from the study. * Patients with a concomitant diagnosis of Inflammatory Bowel Disease. * Patients with a known genetic syndrome (for example, Lynch syndrome or Familial Adenomatous Polyposis). **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** The study will include adult patients with a proven diagnosis of stage I-III colorectal adenocarcinoma, scheduled for elective curative cancer resection. ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Annalisa Maroli, PhD **Phone:** 02 8224 7776 **Phone Ext:** 0039 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Stefano De Zanet, MS **Phone:** 02 8224 4623 **Phone Ext:** 0039 **Role:** CONTACT #### Locations **Location 1:** **City:** Rozzano **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Annalisa Maroli, PhD - **Phone:** 02 8224 7776 - **Phone Ext:** 0039 - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Stefano De Zanet, MS - **Phone:** 02 8224 4623 - **Phone Ext:** 0039 - **Role:** CONTACT **Country:** Italy **Facility:** IRCCS Humanitas Research Hospital **State:** MI **Status:** RECRUITING **Zip:** 20089 #### Overall Officials **Official 1:** **Affiliation:** IRCCS Huamanitas Research Hospital **Name:** Antonino Spinelli, MD, PhD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007414 - Term: Intestinal Neoplasms - ID: D000005770 - Term: Gastrointestinal Neoplasms - ID: D000004067 - Term: Digestive System Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000004066 - Term: Digestive System Diseases - ID: D000005767 - Term: Gastrointestinal Diseases - ID: D000003108 - Term: Colonic Diseases - ID: D000007410 - Term: Intestinal Diseases - ID: D000012002 - Term: Rectal Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC06 - Name: Digestive System Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M17890 - Name: Colorectal Neoplasms - Relevance: HIGH - As Found: Colorectal Cancer - ID: M10448 - Name: Intestinal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M8886 - Name: Gastrointestinal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7256 - Name: Digestive System Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M6336 - Name: Colonic Diseases - Relevance: LOW - As Found: Unknown - ID: M10444 - Name: Intestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M14844 - Name: Rectal Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000015179 - Term: Colorectal Neoplasms ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06355479 **Brief Title:** A Study to Evaluate the Efficacy and Safety of OsrHSA in Patients of Hepatic Cirrhosis With Hypoalbuminemia **Official Title:** A Phase 3 Randomized, Double-blind, Active-controlled Multi-center Study to Evaluate the Efficacy, Safety of OsrHSA in Patients of Hepatic Cirrhosis With Hypoalbuminemia #### Organization Study ID Info **ID:** HY1001-2022-P3 #### Organization **Class:** INDUSTRY **Full Name:** Healthgen Biotechnology Corp. ### Status Module #### Completion Date **Date:** 2023-12-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2024-04-09 **Type:** ACTUAL **Last Update Submit Date:** 2024-04-04 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2023-11-22 **Type:** ACTUAL #### Start Date **Date:** 2023-04-10 **Type:** ACTUAL **Status Verified Date:** 2024-04 #### Study First Post Date **Date:** 2024-04-09 **Type:** ACTUAL **Study First Submit Date:** 2024-03-21 **Study First Submit QC Date:** 2024-04-04 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Healthgen Biotechnology Corp. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Is US Export:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** The goal of this clinical trial is to evaluate the efficacy of OsrHSA works to treat hypoalbuminemia in hepatic cirrhosis patients. It will also learn about the safety and immunogenicity of OsrHSA. The main question it aims to answer is whether OsrHSA is effective in elevating the serum albumin level of cirrhotic patients with hypoalbuminemia. Researchers will compare OsrHSA to the positive comparator, plasma-derived HSA (pHSA) to see if OsrHSA presents as non-inferior to pHSA in the indication of hypoalbuminemia in hepatic cirrhosis patients. Participants will be randomized in a 1:1 ratio to receive OsrHSA or HpHSA (20g IV qd) for up to 14 days, following an EOT visit. Follow-up visits will be taken on EOT+7d, EOT+14d, and EOT+30d, respectively. ### Conditions Module **Conditions:** - Hypoalbuminemia - Hepatic Cirrhosis ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** QUADRUPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 328 **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** OsrHSA 20g, IV qd, Day1 up to Day 14 **Intervention Names:** - Drug: OsrHSA **Label:** OsrHSA **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** pHSA 20g, IV qd, Day1 up to Day 14 **Intervention Names:** - Drug: Plasbumin®-20 **Label:** pHSA **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - OsrHSA **Description:** Recombinant Human Serum Albumin from Oryza Sativa **Name:** OsrHSA **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - pHSA **Description:** Albumin (Human) 20%, USP **Name:** Plasbumin®-20 **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Percentage of participants whose albumin level reaches 35 g/L or above at any time up to 14 days of the study intervention. **Measure:** To evaluate the efficacy of OsrHSA on elevating the serum albumin level. **Time Frame:** Day 1 to Day 14 #### Secondary Outcomes **Description:** Time to reach serum albumin 35 g/L or more by the end of the treatment (EOT) **Measure:** To estimate the time to reach 35 g/L or more in serum albumin **Time Frame:** Day 1 to Day 14 **Description:** To estimate the change from baseline to the end of treatment (EOT) in serum albumin **Measure:** To estimate the change from baseline in serum albumin **Time Frame:** Day 1 to Day 14 **Description:** The change from baseline to the end of treatment (EOT) will be measured in Colloid osmotic pressure **Measure:** To estimate the change from baseline in colloid osmotic pressure **Time Frame:** Day 1 to Day 14 **Description:** The change in body weight will be measured from baseline to the end of treatment (EOT) **Measure:** To estimate the change from baseline in body weight **Time Frame:** Day 1 to Day 14 **Description:** The change in abdominal circumferences will be measured from baseline to the end of treatment (EOT) **Measure:** To estimate the change from baseline in abdominal circumference, and ascites severity **Time Frame:** Day 1 to Day 14 **Description:** The change in ascites severity will be measured from baseline to the end of treatment (EOT) **Measure:** To estimate the change from baseline in ascites severity **Time Frame:** Day 1 to Day 14 ### Eligibility Module **Eligibility Criteria:** Main Inclusion Criteria: 1. Diagnosis of hepatic cirrhosis 2. Adult males or females, aged 18-75 years (both inclusive) at the time of consent 3. Serum albumin level ≤ 30 g/L 4. Capable of giving informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and this protocol Main Exclusion Criteria: 1. History of allergy to rice; a history of allergy to any component of the HpHSA product 2. Therapeutic/ Large-volume paracentesis (\> 5L each time) during the treatment period 3. Ascites resulted from non-hepatic cirrhosis, Budd-Chiari syndrome 4. Participants with Grade III or Grade IV hepatic encephalopathy using West Heaven Criteria 5. A transjugular intrahepatic peritoneal shunt (TIPS) is performed within 1 month prior to the first dosing 6. Evidence of upper gastrointestinal hemorrhage 6 months prior to the first dosing 7. Participants with stage C and stage D hepatocellular carcinoma according to China Liver Cancer Staging (CNLC) Classification 8. Evidence of extrahepatic neoplastic disorders 9. Transplantation 10. HIV positive 11. Participants with pleural effusion and need therapeutic thoracentesis during the treatment period 12. Uncontrolled infection with body temperature ≥ 38.5 degrees Celsius (101.3 degrees Fahrenheit) or ≤ 35 degrees Celsius (95 degrees Fahrenheit) and white blood cells \> 12.0×10\^9/L. i.e. severe intraabdominal infections, sepsis, respiratory tract infections, urine tract infections. 13. Other serious underlying diseases, including but not limited to: hepatopulmonary syndrome, heart failure grade III-IV (NYHA scale of heart function), severe structural heart disease, symptomatic ischemic heart disease, severe chest and lung disease, hemodialysis, active biliary obstructive disease, etc. 14. With the following abnormal laboratory test values: Hematology: white blood cell count \< 2.0×10\^9/L, absolute neutrophil count \< 1.0×10\^9/L, platelets \< 30×10\^9/L, or hemoglobin \< 75 g/L; Chemistry: ALT and/or AST \> 5× upper limit of normal (ULN), total bilirubin \> 3× ULN; Coagulation: INR\>2.0; Renal function: Cr \> 2×ULN, urine protein \>2+; Echocardiography: LVEF \< 50% 15. Pregnant or breastfeeding or plan to get pregnant in 6 months 16. Potentially fertile participants (other than women who have undergone hysterectomy, bilateral salpingectomy, bilateral tubal ligation, or postmenopause for more than 1 year, and men who have undergone bilateral vasectomy) who do not consent to or are unable to use effective contraception throughout the study and 120 days after the end of the study (or early discontinuation of the study); 17. Enrolled in any clinical trials within 3 months prior to the first dose of study intervention 18. Any other condition that the investigator considers would make the participant unsuitable for the clinical trial **Maximum Age:** 75 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Beijing **Country:** China **Facility:** Beijing Friendship Hospital, Capital Medical University **State:** Beijing **Location 2:** **City:** Beijing **Country:** China **Facility:** Beijing Youan Hospital, Capital Medical University **State:** Beijing **Location 3:** **City:** Chongqing **Country:** China **Facility:** Chongqing University Three Gorges Hospital **State:** Chongqing **Location 4:** **City:** Chongqing **Country:** China **Facility:** The Second Affiliated Hospital of Chongqing Medical University **State:** Chongqing **Location 5:** **City:** Fuzhou **Country:** China **Facility:** Mengchao Hepatobiliary Hospital of Fujian Medical University **State:** Fujian **Location 6:** **City:** Fuzhou **Country:** China **Facility:** The First Affiliated Hospital of Fujian Medical University **State:** Fujian **Location 7:** **City:** Guangzhou **Country:** China **Facility:** Nanfang Hospital **State:** Guangdong **Location 8:** **City:** Guangzhou **Country:** China **Facility:** The Third Affiliated Hospital, Sun Yat-Sen University **State:** Guangdong **Location 9:** **City:** Huizhou **Country:** China **Facility:** Huizhou Central People's Hospital **State:** Guangdong **Location 10:** **City:** Guilin **Country:** China **Facility:** Affiliated Hospital of Guilin Medical University **State:** Guangxi **Location 11:** **City:** Liuzhou **Country:** China **Facility:** Liuzhou Worker's Hospital **State:** Guangxi **Location 12:** **City:** Nanning **Country:** China **Facility:** The First Affiliated Hospital of Guangxi Medical University **State:** Guangxi **Location 13:** **City:** Zunyi **Country:** China **Facility:** Affiliated Hospital Of Zunyi Medical University **State:** Guizhou **Location 14:** **City:** Harbin **Country:** China **Facility:** The Fourth Affiliated Hospital, Harbin Medical University **State:** Heilongjiang **Location 15:** **City:** Luoyang **Country:** China **Facility:** Luoyang Central Hospital **State:** Henan **Location 16:** **City:** Zhengzhou **Country:** China **Facility:** The First Affiliated Hospital of Zhengzhou University **State:** Henan **Location 17:** **City:** Zhengzhou **Country:** China **Facility:** The Sixth People's Hospital of Zhengzhou **State:** Henan **Location 18:** **City:** Jingzhou **Country:** China **Facility:** Jingzhou Central Hospital **State:** Hubei **Location 19:** **City:** Shiyan **Country:** China **Facility:** TaiHe Hospital **State:** Hubei **Location 20:** **City:** Wuhan **Country:** China **Facility:** The Central Hospital of Wuhan **State:** Hubei **Location 21:** **City:** Wuhan **Country:** China **Facility:** Wuhan Jinyintan Hospital **State:** Hubei **Location 22:** **City:** Yichang **Country:** China **Facility:** Yichang Central People's Hospital **State:** Hubei **Location 23:** **City:** Yueyang **Country:** China **Facility:** Yueyang Central Hospital **State:** Hunan **Location 24:** **City:** Zhenjiang **Country:** China **Facility:** The Third People's Hospital of Zhenjiang **State:** Jiangsu **Location 25:** **City:** Ganzhou **Country:** China **Facility:** First Affiliated Hospital of Gannan Medical University **State:** Jiangxi **Location 26:** **City:** Nanchang **Country:** China **Facility:** The First Affiliated Hospital of Nanchang University **State:** Jiangxi **Location 27:** **City:** Pingxiang **Country:** China **Facility:** Pingxiang People's Hospital **State:** Jiangxi **Location 28:** **City:** Yichun **Country:** China **Facility:** Yichun People's Hospital **State:** Jiangxi **Location 29:** **City:** Chang chun **Country:** China **Facility:** Hepatobiliary Hospital Of Jilin **State:** Jilin **Location 30:** **City:** Chang chun **Country:** China **Facility:** The First Hospital of Jilin University **State:** Jilin **Location 31:** **City:** Shenyang **Country:** China **Facility:** The Sixth People's Hospital of Shenyang **State:** Liaoning **Location 32:** **City:** Xining **Country:** China **Facility:** The People's Hospital of Qinghai **State:** Qinghai **Location 33:** **City:** Jinan **Country:** China **Facility:** Shandong Public Health Clinical Center **State:** Shandong **Location 34:** **City:** Chengdu **Country:** China **Facility:** West China Hospital, Sichuan University **State:** Sichuan **Location 35:** **City:** Kunming **Country:** China **Facility:** The Second Affiliated Hospital of Kunming Medical University **State:** Yunnan **Location 36:** **City:** Rui'an **Country:** China **Facility:** Ruian People's Hospital **State:** Zhejiang **Location 37:** **City:** Wenzhou **Country:** China **Facility:** The First School of Medicine, School of Information and Engineering, The First Affiliated Hospital of Wenzhou Medical University **State:** Zhejiang ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010335 - Term: Pathologic Processes - ID: D000008107 - Term: Liver Diseases - ID: D000004066 - Term: Digestive System Diseases - ID: D000007019 - Term: Hypoproteinemia - ID: D000001796 - Term: Blood Protein Disorders - ID: D000006402 - Term: Hematologic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC06 - Name: Digestive System Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC15 - Name: Blood and Lymph Conditions ### Condition Browse Module - Browse Leaves - ID: M11103 - Name: Liver Cirrhosis - Relevance: HIGH - As Found: Hepatic Cirrhosis - ID: M24397 - Name: Hypoalbuminemia - Relevance: HIGH - As Found: Hypoalbuminemia - ID: M8485 - Name: Fibrosis - Relevance: HIGH - As Found: Cirrhosis - ID: M11107 - Name: Liver Diseases - Relevance: LOW - As Found: Unknown - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M10069 - Name: Hypoproteinemia - Relevance: LOW - As Found: Unknown - ID: M5077 - Name: Blood Protein Disorders - Relevance: LOW - As Found: Unknown - ID: M9490 - Name: Hematologic Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000008103 - Term: Liver Cirrhosis - ID: D000034141 - Term: Hypoalbuminemia - ID: D000005355 - Term: Fibrosis ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02591979 **Brief Title:** PASHOS Project: Advanced Platform for Sleep Apnea Syndrome Assessment **Official Title:** PASHOS Project: Advanced Platform for Sleep Apnea Syndrome Assessment. A Multi-Centre Study to Validate a Sleep Apnea Screening Model in Primary Health Care #### Organization Study ID Info **ID:** IIBSP-PAS-2013-151 #### Organization **Class:** OTHER **Full Name:** Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau ### Status Module #### Completion Date **Date:** 2018-10 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2019-03-05 **Type:** ACTUAL **Last Update Submit Date:** 2019-03-03 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2018-10 **Type:** ACTUAL #### Start Date **Date:** 2015-03 **Status Verified Date:** 2019-03 #### Study First Post Date **Date:** 2015-10-30 **Type:** ESTIMATED **Study First Submit Date:** 2015-10-27 **Study First Submit QC Date:** 2015-10-29 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to develop and validate a work-model in Primary Health Care for identifying patients with Sleep Apnea Syndrome, based on clinical variables and an ambulatory monitoring study. ### Conditions Module **Conditions:** - Sleep Apnea Syndrome **Keywords:** - Sleep Apnea Syndrome - Primary Health Care ### Design Module #### Design Info **Observational Model:** OTHER **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 198 **Type:** ACTUAL **Patient Registry:** True **Study Type:** OBSERVATIONAL **Target Duration:** 3 Months ### Outcomes Module #### Primary Outcomes **Description:** At primary healthcare **Measure:** Apnea/Hypopnea Index **Time Frame:** At baseline **Description:** At Sleep Lab Unit **Measure:** Apnea/Hypopnea Index **Time Frame:** At 3 months #### Secondary Outcomes **Measure:** Cardiovascular risk factors **Time Frame:** At baseline **Measure:** History of cardiovascular or cerebrovascular disease **Time Frame:** At baseline **Measure:** Lung function tests **Time Frame:** At baseline **Description:** At primary healthcare **Measure:** Self-perceived sleepiness. Epworth Test **Time Frame:** At baseline **Description:** At Sleep lab unit **Measure:** Self-perceived sleepiness. Epworth Test **Time Frame:** At 3 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patients between 18 to 75 years. Exclusion Criteria: * Cognitive impairment or psycho-physical deterioration that impede to perform ambulatory monitoring study. * Worsened or unstable cardiovascular or cerebrovascular disease. * Chronic insomnia (\<5h sleep / day). * Relevant respiratory comorbidity that could interfere with arterial saturation measurements, as moderate to severe chronic obstructive pulmonary disease. * Neuromuscular disease. * Refusal to participate in the study. **Maximum Age:** 75 Years **Minimum Age:** 18 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Patients visited at Primary Care consultations will be included according to a randomization schedule to reach the necessary sample. ### Contacts Locations Module #### Locations **Location 1:** **City:** Barcelona **Country:** Spain **Facility:** Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau **Zip:** 08025 ### References Module #### References **Citation:** Penacoba P, Llauger MA, Fortuna AM, Flor X, Sampol G, Pedro-Pijoan AM, Grau N, Santiveri C, Juvanteny J, Aoiz JI, Bayo J, Lloberes P, Mayos M; PASHOS Working Group. A new tool to screen patients with severe obstructive sleep apnea in the primary care setting: a prospective multicenter study. BMC Pulm Med. 2022 Jan 15;22(1):38. doi: 10.1186/s12890-022-01827-0. **PMID:** 35033055 **Citation:** Penacoba P, Llauger MA, Fortuna AM, Flor X, Sampol G, Pedro Pijoan AM, Grau N, Santiveri C, Juvanteny J, Aoiz JI, Bayo J, Lloberes P, Mayos M; PASHOS Working Group. Primary care and sleep unit agreement in management decisions for sleep apnea: a prospective study in Spain. J Clin Sleep Med. 2020 Sep 15;16(9):1579-1589. doi: 10.5664/jcsm.8492. **PMID:** 32279702 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000004194 - Term: Disease - ID: D000010335 - Term: Pathologic Processes - ID: D000012120 - Term: Respiration Disorders - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000012818 - Term: Signs and Symptoms, Respiratory - ID: D000020919 - Term: Sleep Disorders, Intrinsic - ID: D000020920 - Term: Dyssomnias - ID: D000012893 - Term: Sleep Wake Disorders - ID: D000009422 - Term: Nervous System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BXM - Name: Behaviors and Mental Disorders ### Condition Browse Module - Browse Leaves - ID: M16355 - Name: Syndrome - Relevance: HIGH - As Found: Syndrome - ID: M4361 - Name: Apnea - Relevance: HIGH - As Found: Apnea - ID: M15694 - Name: Sleep Apnea Syndromes - Relevance: HIGH - As Found: Sleep Apnea Syndrome - ID: M14957 - Name: Respiration Disorders - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown - ID: M15623 - Name: Signs and Symptoms, Respiratory - Relevance: LOW - As Found: Unknown - ID: M22242 - Name: Parasomnias - Relevance: LOW - As Found: Unknown - ID: M22654 - Name: Sleep Disorders, Intrinsic - Relevance: LOW - As Found: Unknown - ID: M22655 - Name: Dyssomnias - Relevance: LOW - As Found: Unknown - ID: M15696 - Name: Sleep Wake Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001049 - Term: Apnea - ID: D000012891 - Term: Sleep Apnea Syndromes - ID: D000013577 - Term: Syndrome ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03777579 **Brief Title:** A Study of First-line JS001 and Nab-paclitaxel Versus Palcelbo and Nab-Paclitaxel in Participants With Advanced Recurrent or Metastatic TNBC **Official Title:** A Phase III, Multicenter, Randomized, Placebo-Controlled Study of JS001 (Anti-PD-1 Antibody) in Combination With Nab-Paclitaxel Compared With Placebo With Nab-Paclitaxel as First-line Therapy for Patients With Primarily Diagnose or Recurrent and Metastatic Triple-Negative Breast Cancer #### Organization Study ID Info **ID:** NABP201801 #### Organization **Class:** INDUSTRY **Full Name:** CSPC ZhongQi Pharmaceutical Technology Co., Ltd. ### Status Module #### Completion Date **Date:** 2020-07-30 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2019-08-08 **Type:** ACTUAL **Last Update Submit Date:** 2019-08-06 **Overall Status:** SUSPENDED #### Primary Completion Date **Date:** 2019-12-30 **Type:** ESTIMATED #### Start Date **Date:** 2018-12-21 **Type:** ACTUAL **Status Verified Date:** 2018-12 #### Study First Post Date **Date:** 2018-12-17 **Type:** ACTUAL **Study First Submit Date:** 2018-12-12 **Study First Submit QC Date:** 2018-12-13 **Why Stopped:** trial handovered to another sponser. ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** CSPC ZhongQi Pharmaceutical Technology Co., Ltd. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** This multicenter, randomized, double-blind study will evaluate the efficacy, safety of JS001 administered with nab-paclitaxel compared with placebo in combination with nab-paclitaxel as first-line therapy in participants with primarily diagnosed stage IV and recurrent or metastatic triple-negative breast cancer (TNBC) who have not received prior systemic therapy for metastatic breast cancer (mBC). ### Conditions Module **Conditions:** - Triple Negative Breast Cancer ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - INVESTIGATOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 375 **Type:** ESTIMATED **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants assigned to JS001 plus nab-paclitaxel will receive both agents until disease progression or unacceptable toxicity. **Intervention Names:** - Drug: JS001，an engineered anti-PD-1 antibody - Drug: Nab-Paclitaxel **Label:** JS001 Plus Nab-Paclitaxel **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants assigned to placebo plus nab-paclitaxel will receive both agents until disease progression or unacceptable toxicity. **Intervention Names:** - Drug: Nab-Paclitaxel - Drug: Placebo **Label:** Placebo Plus Nab-Paclitaxel **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - JS001 Plus Nab-Paclitaxel **Description:** JS001 at a fixed dose of 240 milligrams via intravenous (IV) infusion on Days 1 of each 21-day cycle until disease progression or unacceptable toxicity. **Name:** JS001，an engineered anti-PD-1 antibody **Other Names:** - Terepril monoclonal antibody **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - JS001 Plus Nab-Paclitaxel - Placebo Plus Nab-Paclitaxel **Description:** Nab-Paclitaxel at a starting dose of 125mg per square meter via IV infusion on Days 1, 8 of each 21-day cycle. Nab-Paclitaxel will be administered until disease progression or unacceptable toxicity. **Name:** Nab-Paclitaxel **Other Names:** - Paclitaxel For Injection（Albumin Bound） **Type:** DRUG #### Intervention 3 **Arm Group Labels:** - Placebo Plus Nab-Paclitaxel **Description:** Placebo administered via intravenous (IV) infusion on Days 1 of each 21-day cycle until disease progression or unacceptable toxicity. **Name:** Placebo **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** PFS is defined as the time from randomization to the first occurrence of PD, as determined by the investigator using RECIST v1.1, or death from any cause during the study, whichever occurs first. PD is defined as greater than or equal to (\>/=) 20 percent (%) relative increase and \>/=5 millimeter (mm) of absolute increase in the sum of diameters (SD) of target lesions (TLs), taking as reference the smallest SD recorded since treatment started, or appearance of 1 or more new lesions. **Measure:** Progression-Free Survival (PFS) Assessed Using Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1)by Independent Review Committee (IRC) **Time Frame:** From Day 1 to disease progression (PD) or death from any cause, assessed up to end of study (up to approximately 30 months) #### Secondary Outcomes **Description:** PFS is defined as the time from randomization to the first occurrence of PD, as determined by the investigator using RECIST v1.1, or death from any cause during the study, whichever occurs first. PD is defined as greater than or equal to (\>/=) 20 percent (%) relative increase and \>/=5 millimeter (mm) of absolute increase in the sum of diameters (SD) of target lesions (TLs), taking as reference the smallest SD recorded since treatment started, or appearance of 1 or more new lesions. **Measure:** Progression-Free Survival (PFS) Assessed Using RECIST v1.1 by investigator **Time Frame:** From Day 1 to PD or death from any cause, assessed up to end of study (up to approximately 30 months) **Description:** ORR is defined as the rate of CR or PR, as determined by IRC using RECIST v1.1 criteria. **Measure:** Objective response rate (ORR) Assessed Using Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1)by IRC **Time Frame:** From Day 1 to PD or death from any cause, assessed up to end of study (up to approximately 30 months) **Description:** DoR is defined as the time period from the date of initial CR or PR until the date of PD or death from any cause, whichever occurs first. **Measure:** Duration of response (DoR) Assessed Using Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1)by IRC **Time Frame:** From Day 1 to PD or death from any cause, assessed up to end of study (up to approximately 30 months) **Description:** DCR is defined as the rate of of CR, PR, or stable disease according to RECIST v1.1. **Measure:** Disease control rate (DCR) Assessed Using Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1)by IRC **Time Frame:** From Day 1 to PD or death from any cause, assessed up to end of study (up to approximately 30 months) **Description:** OS is defined as the time from randomization to death from any cause. **Measure:** Overall Survival (OS) **Time Frame:** From Day 1 to death from any cause, assessed up to end of study (up to approximately 30 months) **Description:** OS is defined as the time from randomization to death from any cause. **Measure:** OS rate at 12 months **Time Frame:** the percent of participants that are alive at 12months from Day 1. **Description:** OS is defined as the time from randomization to death from any cause. **Measure:** OS rate at 24 months **Time Frame:** the percent of participants that are alive at 24 months from Day 1. **Description:** Progression is confirmed in the target lesion category if the next imaging assessment after iUPD (4-8 weeks later) confirms a further increase in sum of measures of target disease from iUPD, with an increase of at least 5mm. **Measure:** PFS Assessed Using immune-related Response Evaluation Criteria in Solid Tumors (iRECIST) by investigator **Time Frame:** From Day 1 to death from any cause, assessed up to end of study (up to approximately 30 months) **Description:** ORR is defined as the rate of iCR or iPR, as determined by investigator using iRECIST criteria. **Measure:** ORR Assessed Using immune-related Response Evaluation Criteria in Solid Tumors (iRECIST) by investigator **Time Frame:** From Day 1 to death from any cause, assessed up to end of study (up to approximately 30 months) **Description:** DoR is defined as the time period from the date of initial iCR or iPR until the date of iCPD or death from any cause, whichever occurs first. **Measure:** DoR Assessed Using immune-related Response Evaluation Criteria in Solid Tumors (iRECIST) by investigator **Time Frame:** From Day 1 to death from any cause, assessed up to end of study (up to approximately 30 months) **Description:** DCR is defined as the rate of of iCR, iPR, or stable disease according to iRECIST. **Measure:** DCR Assessed Using immune-related Response Evaluation Criteria in Solid Tumors (iRECIST) by investigator **Time Frame:** From Day 1 to death from any cause, assessed up to end of study (up to approximately 30 months) **Description:** percentage and CTC AE(v5.0) of AEs **Measure:** Percentage and severity of Participants With Adverse Events (AEs) **Time Frame:** From Day 1 to 60 days after last dose of study drug, assessed up to end of study (up to approximately 30 months) **Measure:** Percentage of Participants With Anti-Drug Antibodies (ATAs) **Time Frame:** Pre-dose (60minutes±10minutes) on Day 1 of Cycles 1, 3, 5, 7, 9 and at every 6 cycles thereafter until disease progression, at disease progression. (maximum up to 30 months) (1 Cycle = 21 days) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Primarily diagnosed stage IV or recurrent and metastatic, histologically documented TNBC characterized by absence of human epidermal growth factor 2 (HER2), estrogen receptor (ER), and progesterone receptor (PR) expression; 2. No prior chemotherapy or targeted systemic therapy for inoperable stage IV or metastatic TNBC； 3. Eligible for taxane monotherapy； 4. Eastern Cooperative Oncology Group performance status of 0 or 1； 5. Measurable disease as defined by RECIST v1.1； 6. Adequate hematologic and end-organ function。 Exclusion Criteria: 1. Known central nervous system (CNS) disease with active syndrome or untreated disease, except for treated asymptomatic CNS metastases； 2. History of autoimmune disease； 3. History of Anaphylaxis to PD-(L)1 antibody or CTLA-4 antibody or paclitaxel; 4. Prior allogeneic stem cell or solid organ transplantation; 5. Active hepatitis B or hepatitis C; 6. Positive of HIV antibody. **Maximum Age:** 70 Years **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Beijing **Country:** China **Facility:** The fifth medical center of PLA general hospital #### Overall Officials **Official 1:** **Affiliation:** Beijing 302 Hospital **Name:** JIANG ZE FEI, PHD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000001941 - Term: Breast Diseases - ID: D000012871 - Term: Skin Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M5220 - Name: Breast Neoplasms - Relevance: HIGH - As Found: Breast Cancer - ID: M14850 - Name: Recurrence - Relevance: LOW - As Found: Unknown - ID: M30373 - Name: Triple Negative Breast Neoplasms - Relevance: HIGH - As Found: Triple Negative Breast Cancer - ID: M5218 - Name: Breast Diseases - Relevance: LOW - As Found: Unknown - ID: M15674 - Name: Skin Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001943 - Term: Breast Neoplasms - ID: D000064726 - Term: Triple Negative Breast Neoplasms ### Intervention Browse Module - Ancestors - ID: D000000972 - Term: Antineoplastic Agents, Phytogenic - ID: D000000970 - Term: Antineoplastic Agents - ID: D000050257 - Term: Tubulin Modulators - ID: D000050256 - Term: Antimitotic Agents - ID: D000050258 - Term: Mitosis Modulators - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000007155 - Term: Immunologic Factors - ID: D000045505 - Term: Physiological Effects of Drugs ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M19537 - Name: Paclitaxel - Relevance: HIGH - As Found: Surgery - ID: M231 - Name: Albumin-Bound Paclitaxel - Relevance: HIGH - As Found: Surgery - ID: M4225 - Name: Antibodies - Relevance: HIGH - As Found: Program - ID: M4230 - Name: Antibodies, Monoclonal - Relevance: LOW - As Found: Unknown - ID: M10184 - Name: Immunoglobulins - Relevance: HIGH - As Found: Program - ID: M19117 - Name: Immunoglobulins, Intravenous - Relevance: LOW - As Found: Unknown - ID: M26197 - Name: Tubulin Modulators - Relevance: LOW - As Found: Unknown - ID: M26196 - Name: Antimitotic Agents - Relevance: LOW - As Found: Unknown - ID: M10201 - Name: Immunologic Factors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000017239 - Term: Paclitaxel - ID: D000068196 - Term: Albumin-Bound Paclitaxel - ID: D000000906 - Term: Antibodies - ID: D000007136 - Term: Immunoglobulins ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05017779 **Brief Title:** A Hybrid Effectiveness-implementation Trial of a High School-based Executive Function Treatment for Autistic Youth **Official Title:** A Hybrid Effectiveness-implementation Trial of a School Based Executive Function Treatment for Transition Age Autistic Youth #### Organization Study ID Info **ID:** R01MH124772 **Link:** https://reporter.nih.gov/quickSearch/R01MH124772 **Type:** NIH #### Organization **Class:** OTHER **Full Name:** Children's National Research Institute ### Status Module #### Completion Date **Date:** 2024-11-30 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-10-17 **Type:** ACTUAL **Last Update Submit Date:** 2023-10-13 **Overall Status:** ENROLLING_BY_INVITATION #### Primary Completion Date **Date:** 2024-10-30 **Type:** ESTIMATED #### Start Date **Date:** 2021-09-07 **Type:** ACTUAL **Status Verified Date:** 2023-10 #### Study First Post Date **Date:** 2021-08-24 **Type:** ACTUAL **Study First Submit Date:** 2021-07-27 **Study First Submit QC Date:** 2021-08-18 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Children's National Research Institute #### Responsible Party **Investigator Affiliation:** Children's National Research Institute **Investigator Full Name:** Cara Pugliese **Investigator Title:** Clinical Psychologist **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** This study will test the effectiveness of a school-based cognitive behavioral executive function (EF) intervention, Unstuck \& On Target High School (UOT:HS), for transition-age youth with autism spectrum disorder (ASD). UOT:HS was designed to be embedded in high schools and delivered by school staff to improve generalization of skills, increase access to mental health care, and fill a gap in evidence-based approaches to support postsecondary transition. UOT:HS targets flexibility and planning skills and focuses on key functions needed for adult success across 25, 1-hour lessons. School staff will be trained to deliver UOT:HS, study staff will provide ongoing check-ins, and parents will be offered home extensions for each lesson and two trainings to generalize skills to the home environment. Behavioral and parent-report data will be collected prior to intervention, post-intervention, and at 4-to-6-month follow-up. ### Conditions Module **Conditions:** - Executive Dysfunction - Autism Spectrum Disorder - Adolescent Behavior **Keywords:** - Autism Spectrum Disorder - Executive Functioning - Intervention - High school ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 224 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** School staff will receive training on the Unstuck and On Target: High School (UOT:HS) curriculum, and deliver lessons to students during the school day. Interventionists will have the option to participate in ongoing check-ins with study staff. Parents are provided home extensions for each lesson and have the option to participate in trainings delivered by study staff to support generalization of skills to the home environment. **Intervention Names:** - Behavioral: Unstuck & On Target: High School **Label:** Intervention (Unstuck & On Target: High School) **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants in schools assigned to the TAU condition will continue to receive the standard school-based Individualized Education Plan (IEP) accommodations and school supports that would typically be provided. **Intervention Names:** - Other: Treatment as Usual **Label:** Usual Care **Type:** OTHER ### Interventions #### Intervention 1 **Arm Group Labels:** - Intervention (Unstuck & On Target: High School) **Description:** Unstuck \& On Target: High School (UOT:HS) is a group-based curriculum for high school students that targets executive function skills using Cognitive Behavior Therapy (CBT) techniques. UOT:HS focuses on key functions needed for adult success, such as: self-advocacy, flexibility, time management, motivation, goal setting, developing plans, monitoring progress. Guided practice begins with concrete interventionist support and moves to interventionist cueing, self-cueing, and finally automatic use of the skills without support. Lessons are delivered by school personnel within the school setting. Home extension activities are provided to parents. **Name:** Unstuck & On Target: High School **Other Names:** - Formerly Flexible Futures **Type:** BEHAVIORAL #### Intervention 2 **Arm Group Labels:** - Usual Care **Description:** Participants in schools assigned to the Treatment as Usual (TAU) condition will continue to receive the standard school-based IEP accommodations and school supports that would typically be provided. Data will be collected on the types of supports TAU schools offer, and what supports TAU students receive. **Name:** Treatment as Usual **Type:** OTHER ### Outcomes Module #### Other Outcomes **Description:** The "Executive Function Challenge Task" (EFCT) is an objective and ecologically valid task developed by our research team to assess flexibility and planning skills in a social context conducted by a trained research staff member masked to treatment condition. The EFCT uses a standardized, semi-structured protocol which does not provide explicit rules for completing the tasks to mimic the implicit, unspoken, unstructured expectations in everyday life. The EFCT consists of challenges across several activities and responses are scored on a 3-point scale for each task. The scale (0-good, 1-intermediate, 2-poor performance) has task-specific behavioral markers to guide scoring. **Measure:** Executive Function Challenge Task (EFCT) **Time Frame:** Baseline to End of Intervention (up to end of academic year, approx 9 months) **Description:** The "Behavior Rating Inventory of Executive Function, Second Edition" (BRIEF-2) is a well-established parent-report measure of real-world EF skills, with the Shift subscale measuring cognitive flexibility. **Measure:** Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2) **Time Frame:** Baseline, End of Intervention, Follow-up (approx. 6 months after end of intervention) **Description:** The Dimensional Change Card Sort (DCCS) (NIH Toolbox) is a 4-minute, standard lab-based task for assessing cognitive flexibility in terms of set-shifting. Participants are required to sort a series of bivalent cards first according to one dimension (e.g., color) and then according to another (e.g., shape). It is normed from ages 4-85. **Measure:** Dimensional Change Card Sort - NIH Toolbox **Time Frame:** Baseline to End of Intervention (up to end of academic year, approx 9 months) #### Primary Outcomes **Description:** Change in Classroom Behavior will serve as the primary outcome at end of intervention. Classroom behavior will be assessed through 15-minute classroom observations conducted by a trained research staff member masked to treatment condition. Observations will occur during the school day in an academic (non-intervention) class. Raters use a standardized form to detect the presence or absence of seven observable behaviors: social appropriateness, on task behavior, initiation, transitions, organization, getting stuck/preservation, expression of overwhelm/negativity. **Measure:** Classroom Behavior **Time Frame:** Baseline to End of Intervention (up to end of academic year, approx 9 months) **Description:** Change in adaptive behavior will serve as the primary outcome at follow-up (e.g., approximately six month after end of intervention). Adaptive behavior will be measured via parent-report on the Adaptive Behavior Assessment System, Third Edition (ABAS-3). The ABAS-3 is a well-validated parent report measure that assesses practical, everyday skills needed to effectively and independently take care of oneself and interact with others across the lifespan. Performance is represented as standard scores, with higher scores indicating better adaptive skills. **Measure:** Adaptive Behavior (at follow-up) **Time Frame:** Baseline, End of Intervention, Follow-up (approx. 6 months after end of intervention) #### Secondary Outcomes **Description:** Change in adaptive behavior will serve as the secondary outcome at the end of intervention. Adaptive behavior will be measured via parent-report on the Adaptive Behavior Assessment System, Third Edition (ABAS-3). The ABAS-3 is a well-validated parent report measure that assesses practical, everyday skills needed to effectively and independently take care of oneself and interact with others across the lifespan. Performance is represented as standard scores (mean=100; SD=15), with higher scores indicating better adaptive skills. **Measure:** Adaptive Behavior (end of intervention) **Time Frame:** Baseline, End of Intervention (up to end of academic year, approx 9 months) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. full scale IQ \> 80 on a standardized IQ test, either confirmed through educational testing within the last two years or confirmed by the WASI-2 administered by research personnel. Students with a verbal IQ above 75 will be considered for inclusion based on a discussion with teacher guided by probes related to pragmatic language that align with verbal demands of the curriculum. 2. Two of the following: * eligible and/or receiving school-based services or supports for autism * a prior clinical diagnosis of autism from a qualified health professional (as indicated by parent report) * a score of \> 7 on the Social Communication Questionnaire via parent report and/or a score of \> 11 via teacher report * a score of \> 6 on the Autism Spectrum Quotient-10 via self-report 3. capacity to benefit and understand unstuck material as determined by teacher Exclusion Criteria: 1. Students must have a level of proficiency in English to complete questionnaires and study procedures in English. Parents must have a level of proficiency in English or Spanish to complete questionnaires and study procedures in English or Spanish. 2. Student is not able to participate in UOT:HS due to their schedule or ability to benefit from curriculum material as determined by teacher. **Maximum Age:** 22 Years **Minimum Age:** 14 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Rockville **Country:** United States **Facility:** Center for Autism Spectrum Disorders, Children's National Hospital **State:** Maryland **Zip:** 20850 #### Overall Officials **Official 1:** **Affiliation:** Children's Research Institute, Children's National Hospital **Name:** Cara Pugliese, PhD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Access Criteria:** Request login access to NDA data **Description:** De-identified individual data that supports the results will be shared via the National Institute of Mental Health's National Data Archive (NDA). **IPD Sharing:** YES **Time Frame:** Descriptive/raw de-identified data will be reported semi-annually through NDA. ## Derived Section ### Condition Browse Module - Ancestors - ID: D000002659 - Term: Child Development Disorders, Pervasive - ID: D000065886 - Term: Neurodevelopmental Disorders - ID: D000001523 - Term: Mental Disorders ### Condition Browse Module - Browse Branches - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M4623 - Name: Autistic Disorder - Relevance: LOW - As Found: Unknown - ID: M206 - Name: Autism Spectrum Disorder - Relevance: HIGH - As Found: Autism Spectrum Disorder - ID: M5903 - Name: Child Development Disorders, Pervasive - Relevance: LOW - As Found: Unknown - ID: M5902 - Name: Developmental Disabilities - Relevance: LOW - As Found: Unknown - ID: M30644 - Name: Neurodevelopmental Disorders - Relevance: LOW - As Found: Unknown - ID: M4815 - Name: Mental Disorders - Relevance: LOW - As Found: Unknown - ID: M14473 - Name: Psychotic Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000067877 - Term: Autism Spectrum Disorder ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06351579 **Acronym:** ALTO **Brief Title:** Data Collection Post Radical Prostatectomy **Official Title:** A Look at Outcomes After Post Radical Prostatectomy #### Organization Study ID Info **ID:** 1010159 #### Organization **Class:** INDUSTRY **Full Name:** Levee Medical, Inc. ### Status Module #### Completion Date **Date:** 2026-03-15 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-04-08 **Type:** ACTUAL **Last Update Submit Date:** 2024-04-02 **Overall Status:** NOT_YET_RECRUITING #### Primary Completion Date **Date:** 2025-12-30 **Type:** ESTIMATED #### Start Date **Date:** 2024-06-15 **Type:** ESTIMATED **Status Verified Date:** 2024-04 #### Study First Post Date **Date:** 2024-04-08 **Type:** ACTUAL **Study First Submit Date:** 2024-04-02 **Study First Submit QC Date:** 2024-04-02 ### Sponsor Collaborators Module #### Collaborators **Class:** UNKNOWN **Name:** RQMplus #### Lead Sponsor **Class:** INDUSTRY **Name:** Levee Medical, Inc. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The study is to collect information on patients undergoing radical prostatectomy (RP), with a primary focus on the occurrence, duration, and severity of post-prostatectomy incontinence. Data will be collected at multiple time points, allowing for a dynamic understanding of urinary incontinence patterns at post RP. **Detailed Description:** Urinary incontinence is predominantly iatrogenic following radical prostatectomy (RP). Surgical removal of the prostate leads to a shortened urethra, widened bladder neck, and diminished urethral support. This results in increased demand on the urinary sphincter to maintain urinary continence. Most men will experience acute urinary incontinence following RP. Continence progressively improves over time; however, it is estimated that between 5-15% of men will experience chronic urinary incontinence. Current treatments for post-prostatectomy urinary incontinence include conservative therapies such as pelvic floor muscle training and surgical treatments. This study is to systematically collect detailed information on patients undergoing radical prostatectomy, with a primary focus on the occurrence, duration, and severity of post-prostatectomy incontinence. Data will be collected at multiple time points, allowing for a dynamic understanding of urinary incontinence patterns at post RP. ### Conditions Module **Conditions:** - Stress Urinary Incontinence - Radical Prostatectomy - BPH **Keywords:** - SUI - BPH - Prostatectomy ### Design Module #### Design Info **Observational Model:** OTHER **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 30 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Description:** This is an observational (registry) study for data collection only. No device or intervention will be used in this study. **Name:** Control **Type:** OTHER ### Outcomes Module #### Other Outcomes **Description:** The Expanded Prostate Cancer Index Composite is a validated, comprehensive questionnaire designed to evaluate patient function and bother after localized prostate cancer treatment. The questionnaire contains 13 items across 5 domains (i.e., urinary incontinence, urinary irritative/obstructive, bowel, sexual and hormonal). Response options for each item form a Likert scale, and multi-item scale scores are transformed linearly to a 0-100 scale, with higher scores representing better quality of life. **Measure:** Expanded Prostate Index Composite (EPIC-26) score. **Time Frame:** Baseline, post-foley, 6 weeks, 3 months, 6 months, and 12 months post-prostatectomy procedure. #### Primary Outcomes **Description:** The 24-hour (at-home) pad weight test - The pads are pre-weighed prior to providing them to subjects. The test should be started with an empty bladder. The subject will wear pads and perform their normal daily activities for 24 hours. The subject will be asked to change the pads approximately once every 6 hours during waking hours, or more frequently as needed. The pads will be stored in an air tight bag or container after removal, stored in a refrigerator, and brought to the site at the required follow-up visit for weighing. **Measure:** The change in average 24-hour pad weight over time **Time Frame:** Baseline, post-foley, 6 weeks, 3 months, 6 months, and 12 months post-prostatectomy procedure. #### Secondary Outcomes **Description:** The 1-hour pad test involves the following: * Subject puts on a pre-weighed pad and is asked to avoid voiding; * Subject drinks 500 ml of sodium-free liquid in \<15 minutes, then sits or rests; * Subjects walks for 30 minutes, including climbing one flight of stairs (up and down) * Subject performs the following activities: standing up from sitting (10x), coughing vigorously (10x), running on the spot for 1 minute, bending to pick up an object from the floor (5x), and washing hands in running water for 1 minute; * The total amount of urine leaked is determined by weighing the pad. If voiding is urgent and unavoidable, the test is terminated. Collect voiding volume and repeat the test after re-hydration. **Measure:** Change in pad weight during a 1-hour provocative pad weight test **Time Frame:** Baseline, post-foley, 6 weeks, 3 months, 6 months, and 12 months post-prostatectomy procedure. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Male 45-70 years of age 2. Diagnosed with prostate cancer and scheduled for radical prostatectomy 3. Gleason Grade Group 3 or lower 4. Prostate size less than 80 grams 5. Able and willing to provide written consent to participate in the study 6. Able and willing to comply with study follow-up visits and procedures Exclusion Criteria: 1. Malignant tumors outside of the prostate capsule confirmed via baseline assessments (e.g., mpMRI, bone scan) 2. History of urinary incontinence, including stress or urge urinary incontinence 3. On medications to treat overactive bladder (OAB) 4. Presence of urethral stricture or bladder neck contracture 5. Current or chronic urinary tract infection 6. Prior urologic outlet surgical or minimally invasive procedure (e.g., TURP, HoLEP, Rezum, etc.). 7. Prior pelvic radiation or anticipated need for radiation after radical prostatectomy 8. Presence of stones in the bladder 9. History of neurogenic bladder, sphincter abnormalities, or poor detrusor muscle function 10. Body mass index \>35 11. History of cancer (excluding prostate cancer meeting the inclusion criteria) which is not considered in complete remission - a potential participant is considered cured if there has been no evidence of cancer within five years of enrollment 12. Diagnosed or suspected primary neurologic conditions such as multiple sclerosis or Parkinson's disease or other neurological diseases known to affect bladder function, sphincter function or poor detrusor muscle function 13. History of clinically significant congestive heart failure (i.e., New York Heart Association (NYHA) Class III and IV) 14. Insulin-dependent diabetes mellitus 15. Current uncontrolled diabetes (i.e., hemoglobin A1c ≥7.5%) 16. Intravesical prostatic protrusion (IPP) \>5mm 17. History of immunosuppressive conditions (e.g., AIDS, post-transplant) 18. Any significant medical history that would pose an unreasonable risk or make the subject unsuitable for the study per investigator discretion 19. Any cognitive or psychiatric condition that interferes with or precludes direct and accurate communication with the study investigator regarding the study or affect the ability to complete the study quality of life questionnaires 20. Subject currently participating in other investigational studies unless approved by the Sponsor in writing **Maximum Age:** 70 Years **Minimum Age:** 45 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** MALE **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** The study will enroll male subjects diagnosed with prostate cancer who are scheduled for radical prostatectomy. Subjects must meet all the inclusion criteria and none of the exclusion criteria to participate in the study. ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Karen Cornett **Phone:** 7046576278 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Yiming Deng **Phone:** 4083966040 **Role:** CONTACT #### Overall Officials **Official 1:** **Affiliation:** Founder and CTO, Levee Medical, Inc **Name:** Bruce Choi **Role:** STUDY_CHAIR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014555 - Term: Urination Disorders - ID: D000014570 - Term: Urologic Diseases - ID: D000052776 - Term: Female Urogenital Diseases - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications - ID: D000091642 - Term: Urogenital Diseases - ID: D000052801 - Term: Male Urogenital Diseases - ID: D000059411 - Term: Lower Urinary Tract Symptoms - ID: D000020924 - Term: Urological Manifestations ### Condition Browse Module - Browse Branches - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BXM - Name: Behaviors and Mental Disorders ### Condition Browse Module - Browse Leaves - ID: M17299 - Name: Urinary Incontinence - Relevance: HIGH - As Found: Urinary Incontinence - ID: M7936 - Name: Enuresis - Relevance: LOW - As Found: Unknown - ID: M17300 - Name: Urinary Incontinence, Stress - Relevance: HIGH - As Found: Stress Urinary Incontinence - ID: M17305 - Name: Urination Disorders - Relevance: LOW - As Found: Unknown - ID: M17319 - Name: Urologic Diseases - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M27095 - Name: Male Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M29464 - Name: Lower Urinary Tract Symptoms - Relevance: LOW - As Found: Unknown - ID: M22659 - Name: Urological Manifestations - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000014549 - Term: Urinary Incontinence - ID: D000014550 - Term: Urinary Incontinence, Stress ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03615079 **Acronym:** iSAD **Brief Title:** Internet-based CBT After Stroke Pilot **Official Title:** Internet-based Cognitive Behavioral Therapy to Reduce Depressive Symptoms After Stroke Pilot/Feasibility Study #### Organization Study ID Info **ID:** 829426 #### Organization **Class:** OTHER **Full Name:** University of Pennsylvania ### Status Module #### Completion Date **Date:** 2021-07-30 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-11-11 **Type:** ACTUAL **Last Update Submit Date:** 2021-11-10 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2021-07-30 **Type:** ACTUAL #### Results First Post Date **Date:** 2021-11-10 **Type:** ACTUAL **Results First Submit Date:** 2021-07-29 **Results First Submit QC Date:** 2021-10-13 #### Start Date **Date:** 2018-06-11 **Type:** ACTUAL **Status Verified Date:** 2021-11 #### Study First Post Date **Date:** 2018-08-03 **Type:** ACTUAL **Study First Submit Date:** 2018-06-18 **Study First Submit QC Date:** 2018-07-30 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Pennsylvania #### Responsible Party **Investigator Affiliation:** University of Pennsylvania **Investigator Full Name:** Michael Mullen **Investigator Title:** Assistant Professor of Neurology **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Mood disorders occur in 25-30% of stroke patients and are associated with lower quality of life, higher mortality, increased healthcare utilization, and higher costs. Cognitive behavioral therapy (CBT) interventions have been shown to both treat and prevent post-stroke mood disorders, thus having the ability to improve quality of life and reduce costs. This study aims to test the feasibility of internet-based CBT combined with a telephone/email based coaching service after stroke. ### Conditions Module **Conditions:** - Stroke - Depression ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Count:** 28 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Behavioral: Cognitive behavioral therapy **Label:** Cognitive Behavioral Therapy (CBT) program **Type:** OTHER ### Interventions #### Intervention 1 **Arm Group Labels:** - Cognitive Behavioral Therapy (CBT) program **Description:** An 8-week, internet-based cognitive behavioral therapy (CBT) program for depression **Name:** Cognitive behavioral therapy **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** Subject's depressive symptoms will be quantified using the Patient Health Questionnaire 9. This assessment includes 9 close-ended questions with a scoring system ranging from 0 to 27. Scores greater than 10 are considered "positive" for depressive symptoms. We will compare the average PHQ-9 score at study enrollment to the PHQ-9 at 90 days to determine the reduction in depressive symptoms over time. **Measure:** Change in Depressive Symptoms Over Time **Time Frame:** Baseline and 90 days #### Secondary Outcomes **Description:** EuroQOL-5D questionnaire that measures health related quality of life. The scale ranges from 1 to 100, with higher scores indicating better outcomes. **Measure:** Quality of Life Assessed by the EuroQOL-5D **Time Frame:** 90 days ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Age ≥18 years of age * Acute ischemic stroke within the past 3 months * Regular access to the internet, sufficient to allow a minimum of interactions with the internet daily, either through a personal smartphone or web-based internet browser. * Subject is willing and able to participate in internet-based cognitive behavioral therapy * Can participate in the program in English * Willingness and ability to sign informed consent by the patient * Symptoms of mild to moderately depressed mood, defined as a score of 5-19 on the Patient Health Questionnaire-9 at the time of study enrollment. Exclusion Criteria: * Severely depressed patients, defined by a score of 20+ on the Patient Health Questionnare-9 are excluded * Patients with an active bipolar disorder diagnosis are excluded * Patients with personality disorder diagnoses are excluded * Patients with active suicidality or past suicide attempts are excluded * History of schizophrenia or schizoaffective disorder * Active participation in face-to-face psychotherapy prior to stroke * Patients with a history of dementia are excluded * Patients with aphasia, defined as a score of 1 or greater on NIH Stroke Scale Item 9 are excluded. * Patients without regular internet access through a computer, tablet or smartphone are excluded. * Subjects requiring long-term inpatient nursing care are excluded. For patients enrolled as inpatients, individuals being discharged to both home and acute rehab are eligible. Individuals being discharged to a skilled nursing facility or hospice are excluded. * Expected life expectancy less than 6 months or other inability to comply with study follow-up. * Pregnant women and prisoners are excluded **Maximum Age:** 99 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Philadelphia **Country:** United States **Facility:** University of Pennsylvania **State:** Pennsylvania **Zip:** 19104 #### Overall Officials **Official 1:** **Affiliation:** University of Pennsylvania **Name:** Michael Mullen, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Document Section ### Large Document Module #### Large Docs - Date: 2018-12-13 - Filename: Prot_SAP_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: True - Label: Study Protocol and Statistical Analysis Plan - Size: 1261154 - Type Abbrev: Prot_SAP - Upload Date: 2021-07-29T10:11 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000002561 - Term: Cerebrovascular Disorders - ID: D000001927 - Term: Brain Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000014652 - Term: Vascular Diseases - ID: D000002318 - Term: Cardiovascular Diseases ### Condition Browse Module - Browse Branches - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC14 - Name: Heart and Blood Diseases ### Condition Browse Module - Browse Leaves - ID: M7058 - Name: Depression - Relevance: LOW - As Found: Unknown - ID: M7061 - Name: Depressive Disorder - Relevance: LOW - As Found: Unknown - ID: M22306 - Name: Stroke - Relevance: HIGH - As Found: Stroke - ID: M5810 - Name: Cerebrovascular Disorders - Relevance: LOW - As Found: Unknown - ID: M5204 - Name: Brain Diseases - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000020521 - Term: Stroke ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** Definition did not differ #### Event Groups **Group ID:** EG000 **Title:** Cognitive Behavioral Therapy (CBT) Program **Deaths Num At Risk:** 28 **Description:** Cognitive behavioral therapy: An 8-week, internet-based cognitive behavioral therapy (CBT) program for depression **ID:** EG000 **Other Num at Risk:** 28 **Serious Number At Risk:** 28 **Title:** Cognitive Behavioral Therapy (CBT) Program **Frequency Threshold:** 0 **Time Frame:** 90 days ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 11 **Units:** Participants ### Group **ID:** BG000 **Title:** Cognitive Behavioral Therapy (CBT) Program **Description:** Cognitive behavioral therapy: An 8-week, internet-based cognitive behavioral therapy (CBT) program for depression ### Measure #### Measurement **Group ID:** BG000 **Spread:** 14.7 **Value:** 55.3 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 7 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 4 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 1 **Category Title:** Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 10 **Category Title:** Not Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** American Indian or Alaska Native #### Measurement **Group ID:** BG000 **Value:** 1 **Category Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Native Hawaiian or Other Pacific Islander #### Measurement **Group ID:** BG000 **Value:** 3 **Category Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 6 **Category Title:** White #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** More than one race #### Measurement **Group ID:** BG000 **Value:** 1 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 11 **Class Title:** United States **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Ethnicity (NIH/OMB) **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Race (NIH/OMB) **Unit of Measure:** Participants ### Measure 5 **Parameter Type:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** participants **Population Description:** Subjects who engaged with the CBT application at least once ## Results Section - More Information Module ### Certain Agreement ### Limitations and Caveats **Description:** The aim of this study was to enroll 20 subjects into the computer based cognitive-behavioral therapy intervention. Unfortunately enrollment was slower than expected and the proportion of enrolled subjects who utilized the application was low, limiting the analysis and preventing meaningful conclusions. ### Point of Contact **Email:** [email protected] **Organization:** University of Pennsylvania **Phone:** 215-662-3339 **Title:** Michael Mullen ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 5 - **Spread:** - **Upper Limit:** 7 - **Value:** 6 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 16 - **Upper Limit:** - **Value:** 73 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Subject's depressive symptoms will be quantified using the Patient Health Questionnaire 9. This assessment includes 9 close-ended questions with a scoring system ranging from 0 to 27. Scores greater than 10 are considered "positive" for depressive symptoms. We will compare the average PHQ-9 score at study enrollment to the PHQ-9 at 90 days to determine the reduction in depressive symptoms over time. **Dispersion Type:** Inter-Quartile Range **Parameter Type:** MEDIAN **Population Description:** 1 subject missing day 90 PHQ-9 **Reporting Status:** POSTED **Time Frame:** Baseline and 90 days **Title:** Change in Depressive Symptoms Over Time **Type:** PRIMARY **Unit of Measure:** scores on scale ##### Group **Description:** Cognitive behavioral therapy: An 8-week, internet-based cognitive behavioral therapy (CBT) program for depression **ID:** OG000 **Title:** Cognitive Behavioral Therapy (CBT) Program #### Outcome Measure 2 **Description:** EuroQOL-5D questionnaire that measures health related quality of life. The scale ranges from 1 to 100, with higher scores indicating better outcomes. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 90 days **Title:** Quality of Life Assessed by the EuroQOL-5D **Type:** SECONDARY **Unit of Measure:** scores on a scale ##### Group **Description:** Cognitive behavioral therapy: An 8-week, internet-based cognitive behavioral therapy (CBT) program for depression **ID:** OG000 **Title:** Cognitive Behavioral Therapy (CBT) Program ### Participant Flow Module #### Group **Description:** Cognitive behavioral therapy: An 8-week, internet-based cognitive behavioral therapy (CBT) program for depression **ID:** FG000 **Title:** Cognitive Behavioral Therapy (CBT) Program #### Period **Title:** Overall Study ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 28 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 11 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 17 **Pre-Assignment Details:** The proportion of subjects who engaged with the CBT application was not what was expected and as such enrollment was expanded with the goal of getting 20 subjects to use the application. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT02575079 **Brief Title:** Parafilm to Prevent CLABSI in Pediatric Patients Undergoing HCT **Official Title:** Parafilm Application to Prevent Central Line Associated Bloodstream Infections in Pediatric Patients Undergoing Hematopoietic Cell Transplantation #### Organization Study ID Info **ID:** IRB00080344 #### Organization **Class:** OTHER **Full Name:** Emory University ### Status Module #### Completion Date **Date:** 2018-03-29 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2019-06-25 **Type:** ACTUAL **Last Update Submit Date:** 2019-06-06 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2018-03-29 **Type:** ACTUAL #### Results First Post Date **Date:** 2019-06-25 **Type:** ACTUAL **Results First Submit Date:** 2019-03-29 **Results First Submit QC Date:** 2019-06-06 #### Start Date **Date:** 2015-03 **Status Verified Date:** 2019-06 #### Study First Post Date **Date:** 2015-10-14 **Type:** ESTIMATED **Study First Submit Date:** 2015-10-12 **Study First Submit QC Date:** 2015-10-12 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Emory University #### Responsible Party **Investigator Affiliation:** Emory University **Investigator Full Name:** Lakshmanan Krishnamurti **Investigator Title:** Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this study is to see if applying parafilm as an external barrier on the central line in children having a bone marrow transplant helps to prevent central line associated bloodstream infection(s) and also to assess the ease of use of parafilm. **Detailed Description:** Central line associated bloodstream infections (CLABSI) result in significant morbidity and mortality, particularly in patients undergoing hematopoietic cell transplantation (HCT). HCT patients are at high risk for CLABSI due to multiple factors, including prolonged immune suppression and the need for long-term central venous access. The investigators want to assess the feasibility of the use of a parafilm central line barrier (parafilm) as standard of care in pediatric HCT patients. The investigators will also assess the effectiveness of the parafilm to reduce the incidence of CLABSI in these patients. ### Conditions Module **Conditions:** - Central Line Associated Bloodstream Infections (CLABSI) - Bone Marrow Transplant ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** Arm 1 is comprised of study participants receiving parafilm applied over the CVC hub or around the CVC hub connection. Arm 2 is a retrospective historical cohort of pediatric patients receiving HCT for the purpose of comparing CLABSI rates. ##### Masking Info **Masking:** NONE **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 119 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Pediatric patients undergoing hematopoietic stem cell transplant (HCT) with central venous catheters (CVCs) will have pre-cut, single-use sections of parafilm applied over the CVC hub (if not connected) or around the CVC hub connection (if connected). Parafilm will be maintained on the CVC and routine CVC care will be continued, per institutional standard of practice, until the CVC is removed. **Intervention Names:** - Device: Parafilm **Label:** Parafilm **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Pediatric patients receiving hematopoietic cell transplantation in the 16 months preceding the study intervention. Data were obtained retrospectively through medical records for the purpose of comparing CLABSI rates among recipients of parafilm to a control group. **Label:** Historical Cohort **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Parafilm **Description:** Single-use, pre-cut sections of parafilm over the central venous catheter (CVC) hub (if not connected) or around the CVC hub connection (if connected). Parafilm will be maintained on the CVC until it is removed. **Name:** Parafilm **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Central line associated bloodstream infections (CLABSI) is the identification of a bacterial infection from a blood culture drawn from a central venous catheter (CVC) in the absence of a primary source of infection from another body site. The CLABSI rate (total number of CLABSI/1000 catheter-days) in parafilm study participants will be calculated and compared to the CLABSI rate in a historical cohort of pediatric patients who are serving as a control group. **Measure:** Rate of Central Line Associated Bloodstream Infections (CLABSI) **Time Frame:** Baseline (at the time of placement of the CVC or admission for HCT, whichever occurs later) to end of study (CVC removal or transfer back to primary service, whichever occurs sooner, average of 3 months) **Description:** In the inpatient setting, compliance was measured by once daily recording of parafilm change by nursing in the electronic medical record. These data were significantly limited by electronic medical record charting on parafilm change being required only once per day. Thus, once daily data collection failed to capture daily parafilm change, given multiple nursing shifts on a single calendar day. Nonetheless, the percentage of catheter-days with intact correctly applied parafilm, as reported by nursing staff, was calculated. **Measure:** Reported Inpatient Percentage of Central-line Days With Correctly Applied Parafilm **Time Frame:** At discharge (an expected average of 6 weeks from admission) **Description:** Observed compliance occurred during weekly line rounds. Rounding providers indicated whether a patient's parafilm is intact and correctly applied, not applied, or incorrectly applied or not intact. The percentage of "observed catheter-days" where patients had correct use of intact parafilm were to be calculated. To track observed inpatient compliance with applying parafilm, data fields were added to the entered to the nursing charts of the participant's electronic medical record (EMR). The primary field added was a no/question about parafilm applied. Per study protocol, the intent was to document nursing changing of the parafilm, at the time it was changed. However, on reviewing the data, it appears that nursing just charted once per day whether they had changed parafilm or not, while the parafilm may have been changed earlier by a different nurse. **Measure:** Observed Inpatient Percentage of Catheter-days With Correctly Applied Parafilm **Time Frame:** Time between initial bone marrow transplant (BMT) discharge and either central venous line (CVL) removal or transfer back to primary MD (an expected average of 2-3 months) **Description:** For outpatient reported compliance, parents of study participants provided their logs to outpatient staff who documented the number of days parafilm was correctly in use/intact vs not in use or incorrectly in use/not intact. The cumulative percentage of catheter-days where parafilm was intact was calculated. **Measure:** Reported Outpatient Percentage of Catheter-days With Correctly Applied Parafilm **Time Frame:** Baseline (at the time of placement of the CVC or admission for HCT, whichever occurs later) to end of study (CVC removal or transfer back to primary service, whichever occurs sooner, average of 3 months) **Description:** For observed compliance, the outpatient nurse recorded whether the patient's parafilm was correctly applied/intact (yes), not applied (no), or incorrectly applied/not intact (yes-inc) at the beginning of the visit. The percentage of outpatient "observed catheter-days" where parafilm was correctly in use/intact was calculated. **Measure:** Observed Outpatient Percentage of Catheter-days With Correctly Applied Parafilm **Time Frame:** Time between initial BMT discharge and either CVL removal or transfer back to primary MD (an expected average of 2-3 months) #### Secondary Outcomes **Description:** The number of antibiotic treatment courses per participant in the parafilm study will be recorded. **Measure:** Number of Antibiotic Treatment Courses Per Participant **Time Frame:** Baseline (at the time of placement of the CVC or admission for HCT, whichever occurs later) to end of study (CVC removal or transfer back to primary service, whichever occurs sooner, average of 3 months) **Description:** Total duration of antibiotic treatment exposure (excluding prophylactic antibiotics) among participants in the parafilm study will be reported. **Measure:** Duration of Antibiotic Treatment Exposure **Time Frame:** Baseline (at the time of placement of the CVC or admission for HCT, whichever occurs later) to end of study (CVC removal or transfer back to primary service, whichever occurs sooner, average of 3 months) **Description:** The cumulative number of ICU admissions secondary to sepsis among participants in the parafilm study is reported. **Measure:** Number of ICU Admissions Secondary to Sepsis **Time Frame:** Baseline (at the time of placement of the CVC or admission for HCT, whichever occurs later) to end of study (CVC removal or transfer back to primary service, whichever occurs sooner, average of 3 months) **Description:** Deaths from infection and death from any cause among participants in the parafilm study are reported. **Measure:** Death **Time Frame:** Baseline (at the time of placement of the CVC or admission for HCT, whichever occurs later) to end of study (CVC removal or transfer back to primary service, whichever occurs sooner, average of 3 months) **Description:** The perception of parafilm from provider and caregiver surveys regarding parafilm ease of use, perceived benefit, and other parameters will be reported **Measure:** Perception of Parafilm **Time Frame:** Month 3 (average time till removal of CVC) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Must be between the age of 0 and 21 years at the time of enrollment * Must be undergoing allogeneic or autologous HCT for a malignant or non-malignant disorder * Must have or be scheduled to have a tunneled CVC Exclusion Criteria: * Patients undergoing other interventions to prevent CLABSI (e.g. Children's Oncology Group (COG) study ACCL1034 with Chlorhexidine Gluconate (CHG); antimicrobial lock therapy, etc.) * Patients who only have a port **Maximum Age:** 21 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Atlanta **Country:** United States **Facility:** Children's Healthcare of Atlanta **State:** Georgia **Zip:** 30322 #### Overall Officials **Official 1:** **Affiliation:** Emory University **Name:** Lakshmanan Krishnamurti, MD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Stenger EO, Newton JG, Leong T, Kendrick L, McManus L, Rooke C, Krishnamurti L. Application of parafilm as a physical barrier on CVC connections is feasible and may reduce CLABSI among pediatric HCT patients. Biology of Blood Marrow Transplantation 25 (Suppl 3): S131, 2019. ## Document Section ### Large Document Module #### Large Docs - Date: 2018-10-24 - Filename: Prot_SAP_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: True - Label: Study Protocol and Statistical Analysis Plan - Size: 271677 - Type Abbrev: Prot_SAP - Upload Date: 2019-03-18T09:44 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000018746 - Term: Systemic Inflammatory Response Syndrome - ID: D000007249 - Term: Inflammation - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC01 - Name: Infections - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M10283 - Name: Infections - Relevance: HIGH - As Found: Infection - ID: M6368 - Name: Communicable Diseases - Relevance: LOW - As Found: Unknown - ID: M20864 - Name: Sepsis - Relevance: HIGH - As Found: Bloodstream Infection - ID: M16869 - Name: Toxemia - Relevance: LOW - As Found: Unknown - ID: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M20818 - Name: Systemic Inflammatory Response Syndrome - Relevance: LOW - As Found: Unknown - ID: M10293 - Name: Inflammation - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000007239 - Term: Infections - ID: D000018805 - Term: Sepsis ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** Adverse events will be described using event terms and severity grading from the National Cancer Institute (NCI) CTCAE version 4.0. Adverse events will be characterized by severity grading, attribution (relationship to study treatment), and expectedness (based upon prior experience). Only unexpected serious adverse events and serious adverse events related to the study intervention were collected. Non-serious adverse events were not collected. #### Event Groups **Group ID:** EG000 **Title:** Parafilm **Deaths Num Affected:** 3 **Deaths Num At Risk:** 48 **Description:** Pediatric patients undergoing hematopoietic stem cell transplant (HCT) with central venous catheters (CVCs) who had pre-cut, single-use sections of parafilm applied over the CVC hub (if not connected) or around the CVC hub connection (if connected). **ID:** EG000 **Serious Number Affected:** 3 **Serious Number At Risk:** 48 **Title:** Parafilm **Frequency Threshold:** 0 #### Serious Events **Term:** Acute hypoxemic respiratory failure **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 48 **Num Events:** 1 **Term:** Graft versus host disease (GVHD) **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Immune system disorders ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 48 **Num Events:** 2 **Time Frame:** Adverse events were collected from the time a participant entered the study until CVC removal (3 months on average). ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 48 **Group ID:** BG001 **Value:** 71 **Group ID:** BG002 **Value:** 119 **Units:** Participants ### Group **ID:** BG000 **Title:** Parafilm **Description:** Pediatric patients undergoing hematopoietic stem cell transplant (HCT) with central venous catheters (CVCs) who had pre-cut, single-use sections of parafilm applied over the CVC hub (if not connected) or around the CVC hub connection (if connected). ### Group **ID:** BG001 **Title:** Historical Cohort **Description:** Pediatric patients receiving hematopoietic cell transplantation in the 16 months preceding the study intervention. Data were obtained retrospectively through medical records for the purpose of comparing CLABSI rates among recipients of parafilm to a control group. ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Value:** 7 #### Measurement **Group ID:** BG001 **Value:** 21 #### Measurement **Group ID:** BG002 **Value:** 28 **Category Title:** Less than or equal to 2 years #### Measurement **Group ID:** BG000 **Value:** 25 #### Measurement **Group ID:** BG001 **Value:** 22 #### Measurement **Group ID:** BG002 **Value:** 47 **Category Title:** Between 3 and 9 years #### Measurement **Group ID:** BG000 **Value:** 16 #### Measurement **Group ID:** BG001 **Value:** 28 #### Measurement **Group ID:** BG002 **Value:** 44 **Category Title:** Between 10 and 21 years **Class Title:** Age in years ### Measure #### Measurement **Group ID:** BG000 **Value:** 17 #### Measurement **Group ID:** BG001 **Value:** 25 #### Measurement **Group ID:** BG002 **Value:** 42 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 31 #### Measurement **Group ID:** BG001 **Value:** 46 #### Measurement **Group ID:** BG002 **Value:** 77 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 8 #### Measurement **Group ID:** BG001 **Value:** 6 #### Measurement **Group ID:** BG002 **Value:** 14 **Category Title:** Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 40 #### Measurement **Group ID:** BG001 **Value:** 65 #### Measurement **Group ID:** BG002 **Value:** 105 **Category Title:** Not Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 4 #### Measurement **Group ID:** BG002 **Value:** 4 **Category Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 21 #### Measurement **Group ID:** BG001 **Value:** 32 #### Measurement **Group ID:** BG002 **Value:** 53 **Category Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 25 #### Measurement **Group ID:** BG001 **Value:** 34 #### Measurement **Group ID:** BG002 **Value:** 59 **Category Title:** White #### Measurement **Group ID:** BG000 **Value:** 2 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 3 **Category Title:** Other **Class Title:** Ethnicity ### Measure #### Measurement **Group ID:** BG000 **Value:** 48 #### Measurement **Group ID:** BG001 **Value:** 71 #### Measurement **Group ID:** BG002 **Value:** 119 **Class Title:** United States ### Measure #### Measurement **Group ID:** BG000 **Value:** 35 #### Measurement **Group ID:** BG001 **Value:** 49 #### Measurement **Group ID:** BG002 **Value:** 84 **Category Title:** Allogeneic #### Measurement **Group ID:** BG000 **Value:** 13 #### Measurement **Group ID:** BG001 **Value:** 22 #### Measurement **Group ID:** BG002 **Value:** 35 **Category Title:** Autologous **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 17 #### Measurement **Group ID:** BG001 **Value:** 24 #### Measurement **Group ID:** BG002 **Value:** 41 **Category Title:** Malignant, hematologic #### Measurement **Group ID:** BG000 **Value:** 12 #### Measurement **Group ID:** BG001 **Value:** 21 #### Measurement **Group ID:** BG002 **Value:** 33 **Category Title:** Malignant, solid tumor #### Measurement **Group ID:** BG000 **Value:** 19 #### Measurement **Group ID:** BG001 **Value:** 26 #### Measurement **Group ID:** BG002 **Value:** 45 **Category Title:** Non-malignant **Class Title:** **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Age, Customized **Unit of Measure:** Participants ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Ethnicity (NIH/OMB) **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Race/Ethnicity, Customized **Unit of Measure:** Participants ### Measure 5 **Parameter Type:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** participants ### Measure 6 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Type of HCT **Unit of Measure:** Participants ### Measure 7 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Underlying disease **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement **PI Sponsor Employee:** True ### Limitations and Caveats **Description:** Assessing feasibility of outpatient parafilm use was limited by difficulty collecting parafilm compliance calendars from caretakers and observed variability of compliance in the outpatient setting. ### Point of Contact **Email:** elizabeth.o'[email protected] **Organization:** Emory University **Phone:** 404-785-1272 **Title:** Elizabeth Stenger ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### Outcome Measure 4 ### Outcome Measure 5 ### Outcome Measure 6 ### Outcome Measure 7 ### Outcome Measure 8 ### Outcome Measure 9 ### Outcome Measure 10 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 2.19 - **Spread:** - **Upper Limit:** 5.83 - **Value:** 3.69 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 2.86 - **Spread:** - **Upper Limit:** 6.40 - **Value:** 4.37 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 65.2 **Title:** #### Outcome Measure 3 #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 64.7 **Title:** #### Outcome Measure 5 #### Outcome Measure 6 #### Outcome Measure 7 #### Outcome Measure 8 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 8 **Title:** #### Outcome Measure 9 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 **Title:** #### Outcome Measure 10 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Central line associated bloodstream infections (CLABSI) is the identification of a bacterial infection from a blood culture drawn from a central venous catheter (CVC) in the absence of a primary source of infection from another body site. The CLABSI rate (total number of CLABSI/1000 catheter-days) in parafilm study participants will be calculated and compared to the CLABSI rate in a historical cohort of pediatric patients who are serving as a control group. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** Baseline (at the time of placement of the CVC or admission for HCT, whichever occurs later) to end of study (CVC removal or transfer back to primary service, whichever occurs sooner, average of 3 months) **Title:** Rate of Central Line Associated Bloodstream Infections (CLABSI) **Type:** PRIMARY **Unit of Measure:** CLABSI/1000 catheter-days ##### Group **Description:** Pediatric patients undergoing hematopoietic stem cell transplant (HCT) with central venous catheters (CVCs) who had pre-cut, single-use sections of parafilm applied over the CVC hub (if not connected) or around the CVC hub connection (if connected). **ID:** OG000 **Title:** Parafilm ##### Group **Description:** Pediatric patients receiving hematopoietic cell transplantation in the 16 months preceding the study intervention. Data were obtained retrospectively through medical records for the purpose of comparing CLABSI rates among recipients of parafilm to a control group. **ID:** OG001 **Title:** Historical Cohort #### Outcome Measure 2 **Description:** In the inpatient setting, compliance was measured by once daily recording of parafilm change by nursing in the electronic medical record. These data were significantly limited by electronic medical record charting on parafilm change being required only once per day. Thus, once daily data collection failed to capture daily parafilm change, given multiple nursing shifts on a single calendar day. Nonetheless, the percentage of catheter-days with intact correctly applied parafilm, as reported by nursing staff, was calculated. **Parameter Type:** NUMBER **Population Description:** Participants in the parafilm study are included in this analysis. Percentage of inpatient days where nursing charted parafilm change in the electronic medical record is reported for the entire parafilm cohort. **Reporting Status:** POSTED **Time Frame:** At discharge (an expected average of 6 weeks from admission) **Title:** Reported Inpatient Percentage of Central-line Days With Correctly Applied Parafilm **Type:** PRIMARY **Unit of Measure:** percentage of central-line days ##### Group **Description:** Pediatric patients undergoing hematopoietic stem cell transplant (HCT) with central venous catheters (CVCs) who had pre-cut, single-use sections of parafilm applied over the CVC hub (if not connected) or around the CVC hub connection (if connected). **ID:** OG000 **Title:** Parafilm #### Outcome Measure 3 **Description:** Observed compliance occurred during weekly line rounds. Rounding providers indicated whether a patient's parafilm is intact and correctly applied, not applied, or incorrectly applied or not intact. The percentage of "observed catheter-days" where patients had correct use of intact parafilm were to be calculated. To track observed inpatient compliance with applying parafilm, data fields were added to the entered to the nursing charts of the participant's electronic medical record (EMR). The primary field added was a no/question about parafilm applied. Per study protocol, the intent was to document nursing changing of the parafilm, at the time it was changed. However, on reviewing the data, it appears that nursing just charted once per day whether they had changed parafilm or not, while the parafilm may have been changed earlier by a different nurse. **Population Description:** The data collected indicated that nursing staff did not understand how the parafilm monitoring needed to be charted for the purposes of data analysis. Although a data field was created in the EMR, in practice, the data collected was not an accurate assessment of parafilm use, thus no data are available for analysis of this outcome measure. **Reporting Status:** POSTED **Time Frame:** Time between initial bone marrow transplant (BMT) discharge and either central venous line (CVL) removal or transfer back to primary MD (an expected average of 2-3 months) **Title:** Observed Inpatient Percentage of Catheter-days With Correctly Applied Parafilm **Type:** PRIMARY ##### Group **Description:** Pediatric patients undergoing hematopoietic stem cell transplant (HCT) with central venous catheters (CVCs) who had pre-cut, single-use sections of parafilm applied over the CVC hub (if not connected) or around the CVC hub connection (if connected). **ID:** OG000 **Title:** Parafilm #### Outcome Measure 4 **Description:** For outpatient reported compliance, parents of study participants provided their logs to outpatient staff who documented the number of days parafilm was correctly in use/intact vs not in use or incorrectly in use/not intact. The cumulative percentage of catheter-days where parafilm was intact was calculated. **Parameter Type:** NUMBER **Population Description:** The population used for this analysis consists of caretakers who returned outpatient logs of parafilm use at the end of the study. Forty-one participants used parafilm in the outpatient setting and of these 34 returned the outpatient logs. **Reporting Status:** POSTED **Time Frame:** Baseline (at the time of placement of the CVC or admission for HCT, whichever occurs later) to end of study (CVC removal or transfer back to primary service, whichever occurs sooner, average of 3 months) **Title:** Reported Outpatient Percentage of Catheter-days With Correctly Applied Parafilm **Type:** PRIMARY **Unit of Measure:** percent of catheter-days ##### Group **Description:** Pediatric patients undergoing hematopoietic stem cell transplant (HCT) with central venous catheters (CVCs) who had pre-cut, single-use sections of parafilm applied over the CVC hub (if not connected) or around the CVC hub connection (if connected). **ID:** OG000 **Title:** Parafilm #### Outcome Measure 5 **Description:** For observed compliance, the outpatient nurse recorded whether the patient's parafilm was correctly applied/intact (yes), not applied (no), or incorrectly applied/not intact (yes-inc) at the beginning of the visit. The percentage of outpatient "observed catheter-days" where parafilm was correctly in use/intact was calculated. **Population Description:** Observed outpatient compliance was not performed as it was difficult to implement this in a busy clinic setting. **Reporting Status:** POSTED **Time Frame:** Time between initial BMT discharge and either CVL removal or transfer back to primary MD (an expected average of 2-3 months) **Title:** Observed Outpatient Percentage of Catheter-days With Correctly Applied Parafilm **Type:** PRIMARY ##### Group **Description:** Pediatric patients undergoing hematopoietic stem cell transplant (HCT) with central venous catheters (CVCs) who had pre-cut, single-use sections of parafilm applied over the CVC hub (if not connected) or around the CVC hub connection (if connected). **ID:** OG000 **Title:** Parafilm #### Outcome Measure 6 **Description:** The number of antibiotic treatment courses per participant in the parafilm study will be recorded. **Population Description:** Data on antibiotic treatment courses were not collected. **Reporting Status:** POSTED **Time Frame:** Baseline (at the time of placement of the CVC or admission for HCT, whichever occurs later) to end of study (CVC removal or transfer back to primary service, whichever occurs sooner, average of 3 months) **Title:** Number of Antibiotic Treatment Courses Per Participant **Type:** SECONDARY ##### Group **Description:** Pediatric patients undergoing hematopoietic stem cell transplant (HCT) with central venous catheters (CVCs) who had pre-cut, single-use sections of parafilm applied over the CVC hub (if not connected) or around the CVC hub connection (if connected). **ID:** OG000 **Title:** Parafilm #### Outcome Measure 7 **Description:** Total duration of antibiotic treatment exposure (excluding prophylactic antibiotics) among participants in the parafilm study will be reported. **Population Description:** Data on duration of antibiotic treatments were not collected. **Reporting Status:** POSTED **Time Frame:** Baseline (at the time of placement of the CVC or admission for HCT, whichever occurs later) to end of study (CVC removal or transfer back to primary service, whichever occurs sooner, average of 3 months) **Title:** Duration of Antibiotic Treatment Exposure **Type:** SECONDARY ##### Group **Description:** Pediatric patients undergoing hematopoietic stem cell transplant (HCT) with central venous catheters (CVCs) who had pre-cut, single-use sections of parafilm applied over the CVC hub (if not connected) or around the CVC hub connection (if connected). **ID:** OG000 **Title:** Parafilm #### Outcome Measure 8 **Description:** The cumulative number of ICU admissions secondary to sepsis among participants in the parafilm study is reported. **Parameter Type:** NUMBER **Population Description:** Participants in the parafilm study are included in this analysis. **Reporting Status:** POSTED **Time Frame:** Baseline (at the time of placement of the CVC or admission for HCT, whichever occurs later) to end of study (CVC removal or transfer back to primary service, whichever occurs sooner, average of 3 months) **Title:** Number of ICU Admissions Secondary to Sepsis **Type:** SECONDARY **Unit of Measure:** ICU admissions ##### Group **Description:** Pediatric patients undergoing hematopoietic stem cell transplant (HCT) with central venous catheters (CVCs) who had pre-cut, single-use sections of parafilm applied over the CVC hub (if not connected) or around the CVC hub connection (if connected). **ID:** OG000 **Title:** Parafilm #### Outcome Measure 9 **Description:** Deaths from infection and death from any cause among participants in the parafilm study are reported. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Participants in the parafilm study are included in this analysis. **Reporting Status:** POSTED **Time Frame:** Baseline (at the time of placement of the CVC or admission for HCT, whichever occurs later) to end of study (CVC removal or transfer back to primary service, whichever occurs sooner, average of 3 months) **Title:** Death **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Pediatric patients undergoing hematopoietic stem cell transplant (HCT) with central venous catheters (CVCs) who had pre-cut, single-use sections of parafilm applied over the CVC hub (if not connected) or around the CVC hub connection (if connected). **ID:** OG000 **Title:** Parafilm #### Outcome Measure 10 **Description:** The perception of parafilm from provider and caregiver surveys regarding parafilm ease of use, perceived benefit, and other parameters will be reported **Population Description:** A survey was initially planned for in the protocol but was removed from the study procedures prior to any participants completing the survey. **Reporting Status:** POSTED **Time Frame:** Month 3 (average time till removal of CVC) **Title:** Perception of Parafilm **Type:** SECONDARY ##### Group **Description:** Pediatric patients undergoing hematopoietic stem cell transplant (HCT) with central venous catheters (CVCs) who had pre-cut, single-use sections of parafilm applied over the CVC hub (if not connected) or around the CVC hub connection (if connected). **ID:** OG000 **Title:** Parafilm ### Participant Flow Module #### Group **Description:** Pediatric patients undergoing hematopoietic stem cell transplant (HCT) with central venous catheters (CVCs) who had pre-cut, single-use sections of parafilm applied over the CVC hub (if not connected) or around the CVC hub connection (if connected). **ID:** FG000 **Title:** Parafilm #### Group **Description:** Pediatric patients receiving hematopoietic cell transplantation in the 16 months preceding the study intervention. Data were obtained retrospectively through medical records for the purpose of comparing CLABSI rates among recipients of parafilm to a control group. **ID:** FG001 **Title:** Historical Cohort #### Period **Title:** Overall Study ##### Withdraw **Type:** Death ###### Reason **Group ID:** FG000 **Number of Subjects:** 3 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 3 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 48 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 71 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 42 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 71 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 6 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 0 **Pre-Assignment Details:** Fifty-one individuals consented to participate in the study. Prior to beginning any study interventions one was lost to follow up, one withdrew, and one did not begin the prep regimen, resulting in 48 participants who began the study intervention. **Recruitment Details:** Participants were enrolled in the parafilm study between March 2015 and June 2017. All study procedures and follow up was completed on March 29, 2018. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT03499379 **Brief Title:** Stress Hormones and IUDs **Official Title:** Effect of Initiation of Intrauterine Contraception on Hair Cortisol Concentration #### Organization Study ID Info **ID:** OHSU IRB 18244 #### Organization **Class:** OTHER **Full Name:** Oregon Health and Science University ### Status Module #### Completion Date **Date:** 2019-12-31 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-01-07 **Type:** ACTUAL **Last Update Submit Date:** 2020-01-03 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2019-12-31 **Type:** ACTUAL #### Start Date **Date:** 2018-04-16 **Type:** ACTUAL **Status Verified Date:** 2020-01 #### Study First Post Date **Date:** 2018-04-17 **Type:** ACTUAL **Study First Submit Date:** 2018-04-09 **Study First Submit QC Date:** 2018-04-13 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Society of Family Planning #### Lead Sponsor **Class:** OTHER **Name:** Oregon Health and Science University #### Responsible Party **Investigator Affiliation:** Oregon Health and Science University **Investigator Full Name:** Nora Doty, MD **Investigator Title:** Instructor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Determine what kind of side effects women experience in the first year after they start using an intrauterine device. **Detailed Description:** The purpose of this study is to find out if there is a difference in levels of stress hormones in women who use copper or hormonal intrauterine devices (IUDs). This study will provide more information on the potential effect of levonorgestrel intrauterine system (LNG-IUS) use on an individual's stress response. Ultimately, this study hopes to provide data to better counsel women on potential mood effects of the LNG-IUS. From a research and clinical management perspective, hair cortisol may provide a tool to evaluate women at risk for discontinuation of hormonal contraception due to concern of mood symptoms. ### Conditions Module **Conditions:** - Contraception - Mood Change **Keywords:** - Intrauterine device (IUD) - Depression - Stress hormones - Mood changes ### Design Module #### Bio Spec **Description:** A hair sample of approximately 10 (up to 20) hairs cut close to the scalp of the posterior vertex. Whole blood samples collected at each study visit (approximately 10mL). The serum from those samples will tested for hormone levels. **Retention:** SAMPLES_WITH_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 39 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Women obtaining a copper or hormonal intrauterine device for the purpose of contraception. A hair sample of approximately 10 (up to 20) hairs cut close to the scalp of the posterior vertex will be taken at the time of IUD insertion, 6 months post-insertion, and 12 months post-insertion. **Intervention Names:** - Device: Mirena - Device: Paraguard **Label:** Women initiating use of an intrauterine device ### Interventions #### Intervention 1 **Arm Group Labels:** - Women initiating use of an intrauterine device **Description:** A radio-opaque T-shaped polyethylene device containing 52mg of levonorgestrel dispersed in polydimethylsiloxane on its stem. The progestin is released at a rate of 15 mcg per day. **Name:** Mirena **Other Names:** - levonorgestrel intrauterine system **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - Women initiating use of an intrauterine device **Description:** A T-shaped polyethylene device with 380 mm2 of exposed surface area of fine copper wire wound around its arms and stem. Barium sulfate has been added to the polyethylene frame to make the device radio-opaque. A 3-mm plastic ball is located at the base of the IUD, through which the polyethylene monofilament string passes. **Name:** Paraguard **Other Names:** - Copper T380A IUD **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** A hair sample of approximately 10 (up to 20) hairs cut close to the scalp 6 months after start of study participation (baseline - IUD insertion) to assess change from baseline to 6 months. **Measure:** Mean change in hair cortisol concentration - 6 months **Time Frame:** Baseline & 6 months post-insertion #### Secondary Outcomes **Description:** A hair sample of approximately 10 (up to 20) hairs cut close to the scalp 12 months after start of study participation (baseline - IUD insertion) to assess change from baseline to 12 months. **Measure:** Mean change in hair cortisol concentration - 12 months **Time Frame:** Baseline & 12 months post-insertion ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Generally healthy * Age 18-39 years * Regular menstrual cycles * Getting an IUD for the purpose of contraception Exclusion Criteria: * History of mood disorders * BMI less than 18.5 or greater than 35 * Chronic medical conditions * Recently pregnant or lactating **Healthy Volunteers:** True **Maximum Age:** 39 Years **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** FEMALE **Standard Ages:** - ADULT **Study Population:** Study participants will be selected from women's health care clinics in the community. ### Contacts Locations Module #### Locations **Location 1:** **City:** Portland **Country:** United States **Facility:** Oregon Health & Science University **State:** Oregon **Zip:** 97239 #### Overall Officials **Official 1:** **Affiliation:** Oregon Health and Science University **Name:** Nora Doty, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M7058 - Name: Depression - Relevance: LOW - As Found: Unknown - ID: M7061 - Name: Depressive Disorder - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Ancestors - ID: D000080066 - Term: Contraceptive Agents, Hormonal - ID: D000003270 - Term: Contraceptive Agents - ID: D000012102 - Term: Reproductive Control Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000003271 - Term: Contraceptive Agents, Female - ID: D000003280 - Term: Contraceptives, Oral, Synthetic - ID: D000003276 - Term: Contraceptives, Oral ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: Repr - Name: Reproductive Control Agents - Abbrev: Micro - Name: Micronutrients - Abbrev: Mi - Name: Mineral ### Intervention Browse Module - Browse Leaves - ID: M9789 - Name: Hormones - Relevance: LOW - As Found: Unknown - ID: M14244 - Name: Progestins - Relevance: LOW - As Found: Unknown - ID: M9912 - Name: Hydrocortisone - Relevance: LOW - As Found: Unknown - ID: M155245 - Name: Hydrocortisone 17-butyrate 21-propionate - Relevance: LOW - As Found: Unknown - ID: M228609 - Name: Hydrocortisone acetate - Relevance: LOW - As Found: Unknown - ID: M263259 - Name: Hydrocortisone hemisuccinate - Relevance: LOW - As Found: Unknown - ID: M19256 - Name: Levonorgestrel - Relevance: HIGH - As Found: SBRT - ID: M6523 - Name: Copper - Relevance: LOW - As Found: Unknown - ID: M6494 - Name: Contraceptive Agents - Relevance: LOW - As Found: Unknown - ID: M2116 - Name: Contraceptive Agents, Hormonal - Relevance: LOW - As Found: Unknown - ID: M6495 - Name: Contraceptive Agents, Female - Relevance: LOW - As Found: Unknown - ID: M6500 - Name: Contraceptives, Oral - Relevance: LOW - As Found: Unknown - ID: T338 - Name: Copper Supplement - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000016912 - Term: Levonorgestrel ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04669379 **Brief Title:** Is an Online-tool Capable of Improving Patients' Outcomes After Surgery? **Official Title:** Preoperative Optimization of Patient's Expectations by the Use of an Online-Tool - a Randomized Controlled Trial #### Organization Study ID Info **ID:** Online-Tool Erwartungen prä-OP #### Organization **Class:** OTHER **Full Name:** Philipps University Marburg Medical Center ### Status Module #### Completion Date **Date:** 2022-09-30 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2022-01-31 **Type:** ACTUAL **Last Update Submit Date:** 2022-01-28 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2022-06-30 **Type:** ESTIMATED #### Start Date **Date:** 2021-06-15 **Type:** ACTUAL **Status Verified Date:** 2022-01 #### Study First Post Date **Date:** 2020-12-16 **Type:** ACTUAL **Study First Submit Date:** 2020-12-09 **Study First Submit QC Date:** 2020-12-09 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Philipps University Marburg Medical Center #### Responsible Party **Investigator Affiliation:** Philipps University Marburg Medical Center **Investigator Full Name:** Winfried Rief **Investigator Title:** Professor, Dr. **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Recovery after the surgery depends on psychological factors such as preoperative information, expectations, and surgery-associated anxiety. Prior studies have shown that even short preoperative psychological interventions can improve postoperative outcomes. However, the content of these interventions needs further examination. This study aims to examine if the developed preoperative intervention (i) reduces preoperative anxiety, (ii) increases positive expectations, and (iii) improves the long-term outcome and postoperative recovery. Therefore an online-tool has been developed. Using an online-approach makes it possible to reach many patients taking as little time and cost as possible. Patients who undergo surgery in the next two weeks and want to participate in the study will be asked for the planned surgical procedure. There will be a filter (stratum) for surgery. Patients with the same kind of surgery are randomized into two groups (Control vs. intervention group) after they've filled some questionnaires at the baseline assessment. Following this, the intervention group will participate in the psychological online intervention (around 30 minutes). The intervention will focus on the treatment outcome expectations and personal control expectations to increase patients' positive expectations. The control group will receive no psychological intervention. Both groups will fill out questionnaires again in the evening, two days before the surgery, around one week after the surgery, and three months after the surgery. ### Conditions Module **Conditions:** - Patients Undergoing Surgery **Keywords:** - Expectations - Preoperative intervention - placebo effect - surgery - clinical trial - recovery - online intervention ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** The patients will be randomly distributed to the intervention group (IG/EXPECT) or the control group (CG). The intervention group will receive a psychological preoperative online-intervention to optimize expectations. The control group will receive no intervention at all. ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 293 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Behavioral: EXPECT: Preoperative optimization of patient's expectations **Label:** Intervention group (IG/EXPECT) **Type:** EXPERIMENTAL #### Arm Group 2 **Label:** Standard of Care (SOC) **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Intervention group (IG/EXPECT) **Description:** Patients work through an online-tool (around 30 minutes). This preoperative intervention aims to optimize patients' expectations by focusing on positive and realistic expectations regarding the surgery-benefits and recovery process, giving information, and giving advice for coping resources. **Name:** EXPECT: Preoperative optimization of patient's expectations **Type:** BEHAVIORAL ### Outcomes Module #### Other Outcomes **Description:** The B-IPQ surveys the cognitive and emotional representations of illness. Item 1-5 measure cognitive illness representations (consequences, timeline, personal control, treatment control, and identity). Item 6 and 8 quantify emotional representations (concern \& emotions). Item 7 assesses illness comprehensibility. Item 9 is an open question(three most important causal factors in their illness). Items range from 0-10: item 1: no disability at all to very strong disability, item 2: really short to forever, item 3: no control at all zo extreme control, item 4: not at all to extremely helpful, item 5: no complaints at all to very much and strong complaints, item 6: no worries at all to extreme worries,item 7: not at all to very clear, item 8: emotionally not included at all to emotional extremely included. **Measure:** Change in Patients' Illness Perception (Brief Illness Perception Questionnaire, B-IPQ) from Baseline to two days pre-surgery to one week post-surgery to three months after surgery **Time Frame:** Baseline, two days pre-surgery, up to one week post-surgery, up to three months post-surgery **Description:** Items range from 1 (absolutely wrong) - 5 (absolutely right). Higher scores mean a better outcome. **Measure:** Change in patients' expectations (Expected Illness Perception Questionnaire, IPQ-E) from Baseline to one week post-surgery to three months post-surgery **Time Frame:** Baseline, up to one week post-surgery, up to three months post-surgery **Description:** Items range from 1 (absolutely) - 5 (absolutely not). Higher scores mean a worse outcome. **Measure:** Change in patients optimism (Life-Orientation-Test Revised, LOT-R) from Baseline to one week post-surgery to three months post-surgery **Time Frame:** Baseline, up to one week post-surgery, up to three months post-surgery **Description:** Items range from 1 (disagree strongly) - 5 (agree strongly). The questionnaire includes the scales "openness to experience", "conscientiousness", "extraversion", agreeableness and neuroticism **Measure:** Personality (Big Five Inventory, BFI-10) **Time Frame:** Baseline **Description:** Rating of experience with own prior surgeries. Rating of experience with surgeries of close others. First patients are asked if they or close others had a prior surgery before (yes/no). If they answer yes, they are asked to rate their or their close others experience (item ranges from 1 (very bad) - 5 (very good) **Measure:** Patients' experience with prior surgeries **Time Frame:** Baseline #### Primary Outcomes **Description:** Items range from 0 (no disability at all) - 10 (total disability). Consequently higher scores mean a worse outcome. **Measure:** Change in Pain Disability Index (PDI) from Baseline to two days pre-surgery to one week post-surgery to three months post-surgery **Time Frame:** Baseline, up to one week post-surgery, up to three months post-surgery #### Secondary Outcomes **Description:** Items range from 1 (not at all) - 5 (extremely). Higher scores mean patients are more interested in information and do have greater worries **Measure:** Change in The Amsterdam Preoperative Anxiety and Information Scale (APAIS) from Baseline to two days pre-surgery **Time Frame:** Baseline, two days pre-surgery **Description:** There are different item scales. The item scale of the first item (General health perceptions) ranges from 1 (excellent)to 5 (bad). The item scales of the second and third item (limitations in physical activities because of health problems)range from 1 (yes, absolutely restricted) to 2 (yes, a bit restricted) to 3 (no, not restricted at all). The item scales of the items 4-7 (limitations in usual role activities because of physical health or emotional problems) are just "yes" and"no". The item scale of the eighth item (bodily pain) ranges from 1 (Absolutely not) to 5 (a lot). The item scales of the items 9-12 (general mental health, vitality and limitations in social activities because of physical or emotional problems)range from 1 (always) to 5 (never). **Measure:** Change in Health Related Quality of Life (Short Form 12, SF-12) from Baseline to one week post-surgery to three months post-surgery **Time Frame:** Baseline, up to one week post-surgery, up to three months post-surgery **Description:** Items range from 1 (not at all) - 4 (nearly everyday). Consequently higher scores mean a worse outcome. **Measure:** Change in Patient health questionnaire screener (PHQ) from Baseline to one week post-surgery to three months post-surgery **Time Frame:** Baseline, two days pre-surgery, up to one week post-surgery, up to three months post-surgery **Description:** Items range from 0 (absolutely wrong) - 6 (absolutely right). Higher scores mean a better outcome. **Measure:** Change in The Primary Appraisal Secondary Appraisal questionnaire (PASA) from Baseline to two days pre-surgery to one week post-surgery **Time Frame:** Baseline, two days pre-surgery, up to one week post-surgery, up to three months post-surgery **Description:** Items range from 0 (not at all) - 8 (absolutely right). Higher scores mean a better outcome (after reversion of the inverse items 5 and 8). **Measure:** Change in The Control Attitudes Scale-Revised (CAS-R) from Baseline to one week post-surgery **Time Frame:** Baseline, up to one week post-surgery ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * In-patient operation under general anesthetic * Age 18 or above * Fluency in German * Informed consent Exclusion Criteria: * Participation in other research programs: in agreement with Coordinating Investigator patient can participate substudies, if this do not interfere with the main study * Emergency surgery **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Nicole Horn **Phone:** +496421-2823341 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Stefan Salzmann, Dr. **Phone:** +496421-2823350 **Role:** CONTACT #### Locations **Location 1:** **City:** Marburg **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Winfried Rief, PhD - **Phone:** +49 6421 28-23657 - **Role:** CONTACT ***Contact 2:*** - **Name:** Nicole Horn - **Role:** SUB_INVESTIGATOR ***Contact 3:*** - **Name:** Stefan Salzmann - **Role:** SUB_INVESTIGATOR **Country:** Germany **Facility:** Department of Clinical Psychology and Psychotherapy, Philipps-University Marburg **Status:** RECRUITING **Zip:** 35032 #### Overall Officials **Official 1:** **Affiliation:** Clinical Psychology and Psychotherapy Dept. of Psychology, Philipps-University Marburg **Name:** Winfried Rief, Prof. Dr. **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ### References Module #### References **Citation:** Auer CJ, Glombiewski JA, Doering BK, Winkler A, Laferton JA, Broadbent E, Rief W. Patients' Expectations Predict Surgery Outcomes: A Meta-Analysis. Int J Behav Med. 2016 Feb;23(1):49-62. doi: 10.1007/s12529-015-9500-4. **PMID:** 26223485 **Citation:** Gaab, J. (2009). PASA - Primary Appraisal Secondary Appraisal - Ein Fragebogen zur Erfassung von situationsbezogenen kognitiven Bewertungen, Verhaltenstherapie,19:114-115 **Citation:** Juergens MC, Seekatz B, Moosdorf RG, Petrie KJ, Rief W. Illness beliefs before cardiac surgery predict disability, quality of life, and depression 3 months later. J Psychosom Res. 2010 Jun;68(6):553-60. doi: 10.1016/j.jpsychores.2009.10.004. Epub 2009 Dec 5. **PMID:** 20488272 **Citation:** Laferton JA, Kube T, Salzmann S, Auer CJ, Shedden-Mora MC. Patients' Expectations Regarding Medical Treatment: A Critical Review of Concepts and Their Assessment. Front Psychol. 2017 Feb 21;8:233. doi: 10.3389/fpsyg.2017.00233. eCollection 2017. **PMID:** 28270786 **Citation:** Löwe, B., Spitzer, R. L., Zipfel, S., & Herzog, W. (2002). PHQ-D Gesundheitsfragebogen für Patienten (German Version of the Patient Health Questionnaire). Karlsruhe: Pfizer. **Citation:** Moerman N, van Dam FS, Muller MJ, Oosting H. The Amsterdam Preoperative Anxiety and Information Scale (APAIS). Anesth Analg. 1996 Mar;82(3):445-51. doi: 10.1097/00000539-199603000-00002. **PMID:** 8623940 **Citation:** Moser DK, Riegel B, McKinley S, Doering LV, Meischke H, Heo S, Lennie TA, Dracup K. The Control Attitudes Scale-Revised: psychometric evaluation in three groups of patients with cardiac illness. Nurs Res. 2009 Jan-Feb;58(1):42-51. doi: 10.1097/NNR.0b013e3181900ca0. **PMID:** 19092554 **Citation:** Rief, W., & Glombiewski, J. A. (2016). Erwartungsfokussierte Psychotherapeutische Interventionen (EFPI). Verhaltenstherapie, 26(1), 47-54. **Citation:** Rief W, Shedden-Mora MC, Laferton JA, Auer C, Petrie KJ, Salzmann S, Schedlowski M, Moosdorf R. Preoperative optimization of patient expectations improves long-term outcome in heart surgery patients: results of the randomized controlled PSY-HEART trial. BMC Med. 2017 Jan 10;15(1):4. doi: 10.1186/s12916-016-0767-3. **PMID:** 28069021 **Citation:** Salzmann, S., Laferton, J., Auer, C., Shedden-Mora, M., Wambach, K., & Rief, W. (2018). Patientenerwartungen optimieren: Beschreibung einer präoperativen Kurzintervention am Beispiel von Patienten vor einer Bypass-Operation. Verhaltenstherapie, 28(3), 157-165. **Citation:** Spitzer RL, Kroenke K, Williams JB, Lowe B. A brief measure for assessing generalized anxiety disorder: the GAD-7. Arch Intern Med. 2006 May 22;166(10):1092-7. doi: 10.1001/archinte.166.10.1092. **PMID:** 16717171 **Citation:** Ware J Jr, Kosinski M, Keller SD. A 12-Item Short-Form Health Survey: construction of scales and preliminary tests of reliability and validity. Med Care. 1996 Mar;34(3):220-33. doi: 10.1097/00005650-199603000-00003. **PMID:** 8628042 **Citation:** Broadbent E, Petrie KJ, Main J, Weinman J. The brief illness perception questionnaire. J Psychosom Res. 2006 Jun;60(6):631-7. doi: 10.1016/j.jpsychores.2005.10.020. **PMID:** 16731240 **Citation:** Scheier MF, Carver CS, Bridges MW. Distinguishing optimism from neuroticism (and trait anxiety, self-mastery, and self-esteem): a reevaluation of the Life Orientation Test. J Pers Soc Psychol. 1994 Dec;67(6):1063-78. doi: 10.1037//0022-3514.67.6.1063. **PMID:** 7815302 **Citation:** Rammstedt, B. und O. P. John, 2007: Measuring personality in one minute orless: A 10-item short version of the Big Five Inventory in English and German.Journal of Research in Personality 41: 203-212 ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05360979 **Brief Title:** A Clinical Study of Immunotherapy Combined With Chemotherapy and Anti-angiogenic Therapy in Operable NSCLC **Official Title:** A Prospective, Single-arm Clinical Study of Envafolimab Combined With Chemotherapy and Recombinant Human Endostatin in Patients With Operable Stage II, IIIA, and IIIB (T3N2) Non-small Cell Lung Cancer #### Organization Study ID Info **ID:** NSCLC-LXL002 #### Organization **Class:** OTHER **Full Name:** Second Xiangya Hospital of Central South University ### Status Module #### Completion Date **Date:** 2025-05-15 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2022-05-04 **Type:** ACTUAL **Last Update Submit Date:** 2022-05-01 **Overall Status:** ENROLLING_BY_INVITATION #### Primary Completion Date **Date:** 2023-05-15 **Type:** ESTIMATED #### Start Date **Date:** 2022-05-15 **Type:** ESTIMATED **Status Verified Date:** 2022-05 #### Study First Post Date **Date:** 2022-05-04 **Type:** ACTUAL **Study First Submit Date:** 2021-12-26 **Study First Submit QC Date:** 2022-05-01 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Second Xiangya Hospital of Central South University #### Responsible Party **Investigator Affiliation:** Second Xiangya Hospital of Central South University **Investigator Full Name:** xianling liu **Investigator Title:** Director of the oncology department of the Second Xiangya Hospital of Central South University **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The objective of this prospective, single-arm, single-center clinical study is to evaluate the efficacy and safety of envafolimab combined with platinum-containing dual-drug chemotherapy and recombinant human endostatin regimens for treating patients with operable II, IIIA, and IIIB (T3N2) stage NSCLC. **Detailed Description:** Patients with stage II, IIIA, and IIIB (T3N2) NSCLC that did not previously receive systemic treatment and can be treated with surgery were recruited. After signing the informed consent, eligible subjects who meet the inclusion criteria will receive neoadjuvant therapy comprising envafolimab combined with platinum-containing chemotherapy and recombinant human endostatin, as well as postoperative envafolimab single-agent adjuvant therapy. During the preoperative neoadjuvant therapy period, 3 cycles of envafolimab (300 mg fixed-dose Q3W) with recombinant human endostatin (210 mg, CIV \[continuous intravenous pump injection\], 72 h, Q3W), and platinum-containing dual-drug chemotherapy (Q3W) will be performed, with a dosing cycle performed every 3 weeks. The study drug will be administered on the first day of each cycle. All subjects will undergo preoperative imaging evaluation and surgical indication evaluation at 3-5 weeks after the third cycle of preoperative medication. After completing the first 3 cycles of neoadjuvant therapy, all subjects persistently presenting indications for surgery will undergo radical surgery for NSCLC within 4-6 weeks according to the standards defined by the World Association for the Study of Lung Cancer. The pathological staging will be performed according to the AJCC Cancer Staging Manual (8th edition). Local pathologists will evaluate the surgical margins of all specimens removed during the operation. Tumor tissue samples collected from subjects during the research process will be submitted to the designated central laboratory for pathological remission assessment and translational research. Postoperative envafolimab monotherapy (300 mg, Q3W) will be initiated at 4-6 weeks post-surgery and maintained for 1 year Adverse events (AE) will be monitored throughout the study period, and the severity of AEs will be assessed according to the guidelines listed in the National Cancer Institute (NCI) Commonly Used Terminology Criteria for Adverse Events (CTCAE) version 5.0 or above. Safety follow-ups will be conducted for all patients receiving treatment and those who warrant early discontinuation. All subjects will be followed up for OS until death, withdrawal of informed consent, or the end of the study. ### Conditions Module **Conditions:** - Non-small Cell Lung Cancer ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 42 **Type:** ESTIMATED **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** After signing the informed consent, eligible subjects who meet the inclusion criteria will receive neoadjuvant therapy comprising envafolimab combined with platinum-containing chemotherapy and recombinant human endostatin, as well as postoperative envafolimab single-agent adjuvant therapy. **Intervention Names:** - Drug: envafolimab **Label:** Neoadjuvant and adjuvant therapy **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Neoadjuvant and adjuvant therapy **Description:** Envafolimab: 300 mg, D1, Q3W, subcutaneously administered **Name:** envafolimab **Other Names:** - recombinant human endostatin - chemotherapy **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Major pathological reaction (MPR), defined as remaining viable tumor cells ≤ 10% at surgical resection of the primary tumor.MPR rate, defined as the proportion of intention-to-treat (ITT) population reaching MPR. **Measure:** MPR rate **Time Frame:** 0-36months #### Secondary Outcomes **Description:** Event-free lifetime (EFS), defined as the time from the beginning of group entry to the occurrence of any of the following events **Measure:** the event-free survival (EFS) **Time Frame:** 0-36months **Description:** pCR, defined as the absence of any surviving primary tumor cells on surgical removal of the primary tumor. **Measure:** the complete pathological remission rate (pCR) **Time Frame:** 0-36months **Description:** DFS, defined as the time from enrollment to local or distant recurrence (including the occurrence of new primary NSCLC) or death from any cause, whichever occurs first. **Measure:** the disease-free survival (DFS) **Time Frame:** 0-36months **Description:** OS, defined as the time from treatment to the death of the subject due to any cause. **Measure:** the 3-year survival rate and overall survival (OS) **Time Frame:** 0-36months **Description:** The incidence and severity of all AEs, treatment-emergent AEs (TEAEs), serious AEs (SAEs), and immune-related AEs (irAEs), and their correlation with study drugs. **Measure:** Number of participants with treatment-related adverse events as assessed by CTCAE v4.0, AEs, TEAEs, SAEs,irAEs **Time Frame:** 0-36months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Subjects must meet all following inclusion criteria to be eligible for study entry: 1. The subjects fully understand the research and voluntarily sign the informed consent form (ICF); 2. Subjects are aged between 18 to 70 years, irrespective of gender; 3. Patients with resectable stage II, stage IIIA, and stage IIIB (T3N2) (AJCC staging 8th edition) NSCLC with no prior treatment and confirmed by histology; TNM staging can be confirmed by positron emission tomography (PET)-computed tomography (CT) or pathological biopsy; 4. The presence of measurable lesions according to version 1.1 of the evaluation standard for the efficacy of solid tumors; 5. Tumor tissue specimens can be submitted for pathological diagnosis, programmed death-ligand 1 (PD-L1) expression, and biomarker detection before enrollment (tumor tissues must be fresh specimens or archived samples obtained within three months before enrollment; tumor tissue specimen must be histological samples, including, but not limited to, puncture tissues obtained by thick and hollow needles, tissues obtained by bronchoscope clamps, or surgical removal samples. Puncture tissues obtained by fine needles and samples obtained by bronchial brushing are unacceptable); 6. Eastern Cooperative Oncology Group (ECOG) score 0-1; 7. Good organ function; 8. Hematology: Absolute neutrophil count (ANC) ≥ 1500/μL; platelets ≥ 100000/μL; hemoglobin ≥ 9.0 g/dL or ≥ 5.6 mmol/L; 9. Kidney: Serum creatinine ≤ 1.5× upper limit of normal (ULN) or calculated creatinine clearance rate (CrCl) ≥ 60 mL/min (using Cock-Gault formula); 10. Liver: total bilirubin ≤ 1.5 × ULN or for subjects with total bilirubin level \> 1.5 × ULN, direct bilirubin within normal limits; Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase \[SGOT\]) and alanine aminotransferase (ALT) (serum glutamic-pyruvic transaminase \[SGPT\]) ≤ 2.5 × ULN; 11. Endocrine system: Thyroid-stimulating hormone (TSH) is within normal limits. Note: If TSH is not within the normal range at baseline, and if T3 and free T4 are within the normal range, then the subject can be considered to meet inclusion criteria; Coagulation function: international normalized ratio (INR) or prothrombin time (PT), activation partial thromboplastin time (aPTT) ≤ 1.5 × ULN, except for cases: subjects who are receiving anticoagulant therapy, if the PT or aPTT is within the intended use range of anticoagulant drugs; 12. Patients are willing and able to comply with the research plan's visits, treatment plans, laboratory examinations, and other research procedures; 13. According to the assessment of the surgeon, the total lung function can withstand the planned lung resection; 14. Women of childbearing age must undergo a serum pregnancy test within 3 days before the first treatment, and the result should be negative. Female subjects of childbearing age and male subjects whose partners are women of childbearing age must agree to use high-efficiency methods of contraception during the study and within 180 days after the last administration of the study drug. Exclusion Criteria: - 1. Presence of unresectable or metastatic disease; 2. Subjects with NSCLC, large cell neuroendocrine carcinoma (LCNEC), sarcomatoid tumors involving the upper sulcus; subjects with non-squamous NSCLC with known EGFR (epidermal growth factor receptor) mutations or ALK translocations; 3. Subjects with early-stage NSCLC who previously received systemic anticancer treatment, including experimental drug treatment; subjects with a history of (non-infectious) pneumonia/interstitial lung disease that requires steroid treatment, or currently present pneumonia/interstitial disease that requires steroid treatment pulmonary disease; 4. Subjects with a history of active tuberculosis; 5. Subjects with active infections requiring systemic treatment; 6. Subjects with known or suspected autoimmune diseases or immunodeficiency, except for patients with a history of hypothyroidism who do not need hormone therapy or are receiving physiological dose hormone replacement therapy; subjects with stable type I diabetes with controlled blood sugar levels; 7. Subjects with uncontrolled active hepatitis B (defined as a positive test result for hepatitis B virus surface antigen \[HBsAg\] during the screening period, and the detection value of HBV-DNA exceeds the ULN value of the laboratory department of the research center); (Subjects whose HBV-DNA content \<500 IU/mL tested within 28 days before enrollment, and have received at least 14 days of local standard antiviral therapy and are willing to continue to receive antiviral therapy during the study period can be included); Subjects with active hepatitis C (defined as a positive test result of hepatitis C virus surface antibody \[HCsAb\] and HCV-RNA positive during the screening period); 8. Known human immunodeficiency virus (HIV) infection (known HIV antibody positive); 9. Subjects vaccinated with a live vaccine within 30 days before the first administration, including, but not limited to, the following: mumps, rubella, measles, chickenpox/shingles (chickenpox), yellow fever, rabies, Bacille Calmette-Guerin (BCG) and typhoid vaccine (inactivated virus vaccine allowed); 10. Subjects who previously received PD-1/PD-L1 drug treatment or treatment of another drug targeting T cell receptors (such as CTLA-4 and OX-40); 11. Subjects who have had a severe allergic reaction to other monoclonal antibodies; 12. Subjects who have a history of severe allergies to pemetrexed, paclitaxel or albumin paclitaxel or docetaxel, cisplatin, carboplatin, recombinant human endostatin active ingredients, or preventive medications; 13. Subjects who are known to have serious or uncontrolled underlying diseases; 14. Subjects presenting malignant tumors other than NSCLC within 5 years before the first administration. Malignant tumors with negligible risk of metastasis or death (e.g., expected DFS\> 5 years) and expected curative results after treatment (e.g., fully treated cervical carcinoma in situ, basal or squamous cell skin cancer, radical surgery treated ductal carcinoma in situ) can be excluded. 15. Subjects with grade III-IV congestive heart failure (New York Heart Association classification) and poorly controlled and clinically significant arrhythmia; 16. Subjects who have experienced any arterial thrombosis, embolism or ischemia, such as myocardial infarction, unstable angina, cerebrovascular accident, or transient ischemic attack, within 6 months before selection for treatment. **Gender Based:** True **Maximum Age:** 70 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Changsha **Country:** China **Facility:** the second Xiangya hospital **State:** Hunan **Zip:** 410000 ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012142 - Term: Respiratory Tract Neoplasms - ID: D000013899 - Term: Thoracic Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000008171 - Term: Lung Diseases - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000002283 - Term: Carcinoma, Bronchogenic - ID: D000001984 - Term: Bronchial Neoplasms ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M11172 - Name: Lung Neoplasms - Relevance: HIGH - As Found: Lung Cancer - ID: M5546 - Name: Carcinoma, Non-Small-Cell Lung - Relevance: HIGH - As Found: Non-Small Cell Lung Cancer - ID: M5534 - Name: Carcinoma - Relevance: LOW - As Found: Unknown - ID: M14979 - Name: Respiratory Tract Neoplasms - Relevance: LOW - As Found: Unknown - ID: M16658 - Name: Thoracic Neoplasms - Relevance: LOW - As Found: Unknown - ID: M11168 - Name: Lung Diseases - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown - ID: M5540 - Name: Carcinoma, Bronchogenic - Relevance: LOW - As Found: Unknown - ID: M5260 - Name: Bronchial Neoplasms - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000008175 - Term: Lung Neoplasms - ID: D000002289 - Term: Carcinoma, Non-Small-Cell Lung ### Intervention Browse Module - Ancestors - ID: D000000970 - Term: Antineoplastic Agents - ID: D000020533 - Term: Angiogenesis Inhibitors - ID: D000043924 - Term: Angiogenesis Modulating Agents - ID: D000006133 - Term: Growth Substances - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000006131 - Term: Growth Inhibitors ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M22318 - Name: Angiogenesis Inhibitors - Relevance: LOW - As Found: Unknown - ID: M2853 - Name: Immunomodulating Agents - Relevance: LOW - As Found: Unknown - ID: M25114 - Name: Endostatins - Relevance: HIGH - As Found: Intravenous bolus - ID: M9231 - Name: Growth Inhibitors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000043169 - Term: Endostatins ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05566379 **Brief Title:** Mindfulness in Post Acute Sequelae of SARS-CoV-2 Infection (PASC) Dysautonomia **Official Title:** Effects of a Mindfulness Intervention on Physical and Psychological Well-Being and Quality of Life in Patients With Post Acute Sequelae of SARS-CoV-2 Infection (PASC) Dysautonomia #### Organization Study ID Info **ID:** 22-001020 #### Organization **Class:** OTHER **Full Name:** University of California, Los Angeles ### Status Module #### Completion Date **Date:** 2023-06 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-05-10 **Type:** ACTUAL **Last Update Submit Date:** 2023-05-09 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2023-06 **Type:** ESTIMATED #### Start Date **Date:** 2023-02-28 **Type:** ACTUAL **Status Verified Date:** 2023-05 #### Study First Post Date **Date:** 2022-10-04 **Type:** ACTUAL **Study First Submit Date:** 2022-10-02 **Study First Submit QC Date:** 2022-10-02 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of California, Los Angeles #### Responsible Party **Investigator Affiliation:** University of California, Los Angeles **Investigator Full Name:** Jeffrey J. Hsu **Investigator Title:** Principal Investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The current pilot study will recruit participants experiencing new, returning, or ongoing symptoms related to COVID-19 illness for at least four weeks after being first infected with SARS-CoV-2. All participants will attend a virtual 6-week course entitled Mindful Awareness Practices (MAPs) created, hosted and led by expert facilitators from the Mindful Awareness Research Center (MARC) at University of California Los Angeles (UCLA). This intervention will consist of a mix of lecture, practice, group feedback, and discussion regarding mindfulness. Mindfulness is the mental state achieved by focusing one's awareness on the present while acknowledging and accepting any feelings, thoughts, or bodily sensations. The research team will collect self-reported measures of mental health symptoms, physical health symptoms, and demographic information before and after participants attend MAPs. Objective health measures will also be collected by the research team including an active stand test, a 6-minute walk, and a blood sample. **Detailed Description:** The purpose of the current pilot project is to assess the feasibility and effects of a 6-week group mindfulness intervention in patients with Post Acute Sequelae of SARS-CoV-2 Infection (PASC)-related dysautonomia. This single-arm study uses a one group pretest/posttest design. A convenience sample of patients will be recruited from the Ahmanson-University of California Los Angeles (UCLA) COVID-19 Cardiology Specialty Clinic and UCLA Long COVID Program. Outcomes Measures: Once consented, eligible participants will complete a baseline demographics survey, preintervention assessments including the composite autonomic symptom scoring (COMPASS-31), outcome measures questionnaires for perceived stress, anxiety, depression, fatigue, resilience, sleep, event specific distress, well-being and quality of life. A peripheral blood collection device for evaluation of inflammatory gene expression will be performed along with an active stand test and 6- minute walk prior to the mindfulness intervention. The same measures will be collected following completion of the intervention. Additionally, a logbook of patient self-reported practice frequency will be reviewed, and a focused 3 question audio recorded interview will be conducted at the post intervention visit. At 4-week follow-up post intervention, the final set of assessment questionnaires, and a 3-question mindfulness experience survey will be collected. Intervention: Participants will complete a virtual group based 6-week mindfulness meditation-based intervention, Mindful Awareness Practices (MAPs), developed by Diana Winston and colleagues at the Mindful Awareness Research Center (MARC) at UCLA. In response to COVID-19 precautions participants will meet virtually for six weekly, 2-hour group sessions. The group size is estimated at 15 participants. MAPs is a standardized intervention that has been used in several previous studies. MAPS includes presentation of theoretical framework and materials on mindfulness, relaxation, and the mind-body connection, and experiential practice of meditation. Lecture, discussion, and group process focus on solving problems concerning barriers to effective practice, working with difficult thoughts and emotions, managing pain, and cultivation of loving kindness. Written materials are provided with a summary of information covered each week. Additionally, participants are instructed to practice mindfulness exercises at home on a daily basis (5-20 minutes/day) and advised in the informal use of mindfulness in daily life. Participants will be asked to keep a self-reported log of their daily practice. Classes will be led by experienced mindfulness instructors who received specialized training at the UCLA MARC Center. Overview: Baseline Visit (in person): collect within 1-2 week(s) prior to the intervention. Consent Baseline demographics survey COMPASS-31: the composite autonomic symptom score Questionnaires: perceived stress (PSS), anxiety (GAD-7), depression (PHQ 8), event specific distress (IES-R), fatigue (FSI), sleep (ISI), well-being (MHC-SF), resilience (CD-RISC10), QOL (SF-20). Tasso device blood collection for evaluation of inflammatory gene expression. Perform an active stand test Perform 6-minute walk Post Intervention Visit (in person): collect within 1-2 weeks post intervention completion COMPASS-31: the composite autonomic symptom score Questionnaires: perceived stress (PSS), anxiety (GAD-7), depression (PHQ 8), event specific distress (IES-R), fatigue (FSI), sleep (ISI), well-being (MHC-SF), resilience (CD-RISC10), QOL (SF-20). Tasso device blood collection for evaluation of inflammatory gene expression Perform an active stand test Perform Six-minute walk Three question mindfulness experience focused interview Frequency of Practice Logbook review 4-Week Post Intervention Visit (remote link / in person NOT required): collect 4 weeks (+2) post intervention Questionnaires: perceived stress (PSS), anxiety (GAD-7), depression (PHQ 8), event specific distress (IES-R), fatigue (FSI), sleep (ISI), well-being (MHC-SF), resilience (CD-RISC10), QOL (SF-20). 3-question Likert mindfulness experience survey ### Conditions Module **Conditions:** - Long COVID - Dysautonomia **Keywords:** - Mindfulness , Long COVID, Dysautonomia ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Count:** 30 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** All participants will attend a virtual 6-week course entitled Mindful Awareness Practices (MAPs) created, hosted and led by expert facilitators from the Mindful Awareness Research Center at UCLA. Participants will learn Mindful concepts and a variety of Mindfulness practices. This intervention will consist of a mix of lecture, practice, group feedback, and discussion regarding mindfulness. Mindfulness is the mental state achieved by focusing one's awareness on the present while acknowledging and accepting any feelings, thoughts, or bodily sensations. MAPs classes meets weekly for two hours per week for six weeks. Participants are encouraged to complete some daily meditation practice starting at five minutes a day and working up to 20 minutes daily by the end of the course. **Intervention Names:** - Other: Mindfulness - Mindful Awareness Practices ( MAPs) **Label:** Mindfulness Intervention **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Mindfulness Intervention **Description:** All participants will attend a virtual 6-week course entitled Mindful Awareness Practices (MAPs) created, hosted and led by expert facilitators from the Mindful Awareness Research Center at UCLA. Participants will learn Mindful concepts and a variety of Mindfulness practices. This intervention will consist of a mix of lecture, practice, group feedback, and discussion regarding mindfulness. Mindfulness is the mental state achieved by focusing one's awareness on the present while acknowledging and accepting any feelings, thoughts, or bodily sensations. MAPs classes meets weekly for two hours per week for six weeks. Participants are encouraged to complete some daily meditation practice starting at five minutes a day and working up to 20 minutes daily by the end of the course. **Name:** Mindfulness - Mindful Awareness Practices ( MAPs) **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** COMPASS-31 (the composite autonomic symptom) score is a self-rating questionnaire evaluating six domains of autonomic function: orthostatic intolerance, vasomotor, secretomotor, gastrointestinal, bladder, and pupillomotor domains. The six domain scores sum to a total score of 0 to 100, and a higher score indicates more severe autonomic symptoms. It is based on the original Autonomic Symptom Profile (ASP) and COMPASS, is internally consistent and applies a much-simplified scoring algorithm suitable for widespread use in autonomic research and practice. **Measure:** Change from baseline in the composite autonomic symptom score COMPASS-31 for autonomic symptoms **Time Frame:** Baseline / Pre Intervention(1-2 weeks prior to intervention), Post Intervention ( 1-2 weeks post intervention completion) **Description:** Active Stand Test. Clinically, an active stand test can be used to assess short-term neural and cardiovascular function and identify the hemodynamic correlates of patient symptoms and attributable causes of (pre-)syncope, and to detect autonomic dysfunction, variants of orthostatic hypotension, postural orthostatic tachycardia syndrome and orthostatic hypertension. During a standardized active stand test procedure, heart rate and blood pressure are measured after resting lying down, then immediately upon standing and after 2, 5 and 10 minutes. **Measure:** Change from baseline in Active Stand Test of hemodynamic symptom parameters, exercise tolerance. **Time Frame:** Baseline / Pre Intervention(1-2 weeks prior to intervention), Post Intervention ( 1-2 weeks post intervention completion) **Description:** Six Minute Walk. The six-minute walk (6MWT) was developed in 1963 by Balke to evaluate functional capacity and endurance during physical activity. The standardized six-minute walk test (6MWT) measures the maximum distance an individual is able to walk over a total of six minutes. The individual is allowed to self-pace and rest as needed as they traverse back and forth along a flat marked walkway. Baseline heart rate and oxygen saturation are measured then continuously monitored to identify the lowest oxygen saturation, which may occur before the end of the test. The patient's baseline and post-test perceived dyspnea and fatigue are rated using the Borg scale 0-10 (0 none- 10 maximum). **Measure:** Change from baseline of Six Minute Walk in hemodynamic parameters, exercise tolerance **Time Frame:** Baseline / Pre Intervention(1-2 weeks prior to intervention), Post Intervention ( 1-2 weeks post intervention completion) **Description:** Self-reported QOL SF-20. A 20-item questionnaire. The survey measures health across 6 domains: physical functioning (6 questions), role functioning (2 questions), social functioning (1 question), mental health (5 questions), health perceptions (5 questions), and pain (1 question). Scores across each of these domains are reported on a 0% to 100% scale, with 0% representing the worst possible score in that domain and 100% the best possible score. Raw scores are transformed to fit the 0% to 100% interval as described in the original publication (note that for question #1 on general health, an initial transformation is performed as follows: 1 = 5, 2 = 4.36, 3 = 3.43, 4 = 1.99, 5 = 1). Reversal of scoring is completed as necessary such that the highest score always represents the best possible score. The exception to this scoring pattern is the pain score, for which 0% represents the best possible score and 100% the worst possible score. **Measure:** Change from baseline in mean score in Health-Related Quality of Life (QOL) SF-20 **Time Frame:** Baseline / Pre Intervention(1-2 weeks prior to intervention), Post Intervention (1-2 weeks post intervention completion); 4 weeks post intervention #### Secondary Outcomes **Description:** Perceived Stress Scale (PSS) appraises thoughts and feelings in perception of stress. It is a 10-item scale (score 0-40) with upper scores signifying increased perceived stress. While the aim is to capture stress level, the PSS is used to obtain a patients' perceptions of their own stress. **Measure:** Change from baseline in mean scores of PSS- Perceived Stress Scale. **Time Frame:** Baseline / Pre Intervention(1-2 weeks prior to intervention), Post Intervention (1-2 weeks post intervention completion); 4 weeks post intervention **Description:** Generalized Anxiety Disorder (GAD7) It is a 7-item scale (score 0-21), with acuity distinguished as mild (5-9), moderate (10-14) or severe (15-21) distress (Spitzer et al. 2006). The higher score indicates greater anxiety. **Measure:** Change from baseline in mean scores of GAD7- Generalized Anxiety Disorder **Time Frame:** Baseline / Pre Intervention(1-2 weeks prior to intervention), Post Intervention (1-2 weeks post intervention completion); 4 weeks post intervention **Description:** Depression (PHQ8) adapted from the PHQ-9, is established as a valid diagnostic and severity measure for depressive disorders in large clinical studies. A PHQ-8 score of 0 to 4 indicates no depression, of 5 to 9 indicates mild depression, of 10 to 14 indicates moderate depression, of 15 to 19 indicates moderately severe depression, and of 20 or higher indicates severe depression. **Measure:** Change from baseline in mean scores of PHQ-8 - Depressive Symptoms **Time Frame:** Baseline / Pre Intervention(1-2 weeks prior to intervention), Post Intervention (1-2 weeks post intervention completion); 4 weeks post intervention **Description:** Impact of Event Scale -Revised (IES-R). is a 22-item self-report measure that assesses subjective distress caused by traumatic events. The IES-R contains seven additional items related to the hyperarousal symptoms of PTSD, which were not included in the original IES. Items correspond directly to 14 of the 17 The Diagnostic and Statistical Manual of Mental Disorders (DSM-IV) symptoms of PTSD. Respondents are asked to identify a specific stressful life event and then indicate how much they were distressed or bothered during the past seven days by each "difficulty" listed. Items are rated on a 5-point scale ranging from 0 ("not at all") to 4 ("extremely"). The IES-R yields a total score (ranging from 0 to 88) and subscale scores can also be calculated for the Intrusion, Avoidance, and Hyperarousal subscales. **Measure:** Change from baseline in mean scores of IES-R - event-related distress scale **Time Frame:** Baseline / Pre Intervention(1-2 weeks prior to intervention), Post Intervention (1-2 weeks post intervention completion); 4 weeks post intervention **Description:** The Fatigue Symptom Inventory (FSI) is a 14-item self-report measure designed to assess the severity, frequency, and daily pattern of fatigue as well as its perceived interference with quality of life in the past week. Severity is measured on point scales (0=not at all fatigued; 10=as fatigued as I could be) that assess most, least, and average fatigue in the past week as well as current fatigue. Frequency is measured by the number of days (0-7) that respondents felt fatigued and the extent of each day they felt fatigued (0=none of the day; 10=the entire day). Perceived interference is measured on point scales (0=no interference; 10=extreme interference) that assess the degree to which fatigue was felt to interfere with with general level of activity, enjoyment, and mood. **Measure:** Change from baseline in mean scores of FSI - The Fatigue Symptom Inventory. **Time Frame:** Baseline / Pre Intervention(1-2 weeks prior to intervention), Post Intervention (1-2 weeks post intervention completion); 4 weeks post intervention **Description:** The Insomnia Severity Index (ISI) is a seven-item self-report questionnaire, which asks respondents to rate the nature and symptoms of their sleep problems. Dimensions evaluated are severity of sleep onset, sleep maintenance, and early morning awakening problems, sleep dissatisfaction, interference of sleep difficulties with daytime functioning, noticeability of sleep problems by others, and distress caused by the sleep difficulties. A 5-point Likert scale is used to rate each item (e.g., 0 = no problem; 4 = very severe problem), yielding a total score ranging from 0 to 28. The total score is interpreted as follows: absence of insomnia (0-7); sub-threshold insomnia (8-14); moderate insomnia (15-21); and severe insomnia (22-28). **Measure:** Change from baseline in mean scores of the ISI - The Insomnia Severity Index **Time Frame:** Baseline / Pre Intervention(1-2 weeks prior to intervention), Post Intervention (1-2 weeks post intervention completion); 4 weeks post intervention **Description:** The Mental Health Continuum Short Form (MHC-SF) is a 14- item scale containing 3 items for emotional (hedonic) well-being, 5 items for social well-being, and 6 items for psychological well-being (eudaimonic). Each of the items can be scored between 0 and 5, with the total score on the scale can range from 0 to 70 points. Higher scores indicate a higher level of emotional wellbeing. This scale also provides a flourishing and languishing mental health indicator based on these three subscales. **Measure:** Change from baseline in mean scores of well-being from MHC-SF-The Mental Health Continuum Short Form **Time Frame:** Baseline / Pre Intervention(1-2 weeks prior to intervention), Post Intervention (1-2 weeks post intervention completion); 4 weeks post intervention **Description:** Connor Davidson Resilience Scale (CD-RISC 10) is a 10-item scale (score 0-40) that measures components of adaptation, coping and recovery in response to stressful events, trauma, or tragedy. The higher scores reflect greater resilience. **Measure:** Change from baseline in mean scores of CD-RISC 10 - Connor Davidson Resilience Scale. **Time Frame:** Baseline / Pre Intervention(1-2 weeks prior to intervention), Post Intervention (1-2 weeks post intervention completion); 4 weeks post intervention ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: Adult females (18 years - 54 years of age) * Previous SARS-CoV-2 infection confirmed by Polymerase chain reaction (PCR) testing and diagnoses of PASC and dysautonomia confirmed by objective testing (e.g., autonomic reflex screen, active stand test) * Ability to comprehend English and complete assessments and patient-reported surveys * Availability of a smartphone, tablet, or computer with Internet access Exclusion Criteria: * Inability to participate in the virtual intervention or complete outcomes surveys * Current participation regular mindfulness practice * Current enrollment in another COVID-19 related study. **Maximum Age:** 54 Years **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Elizabeth Vandenbogaart, DNP MSN ACNP **Phone:** 310 825 8816 **Phone Ext:** 2 **Role:** CONTACT #### Locations **Location 1:** **City:** Los Angeles **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Elizabeth Vandenbogaart, DNP MSN ACNP - **Phone:** 310-825-8816 - **Phone Ext:** 2 - **Role:** CONTACT **Country:** United States **Facility:** UCLA Health **State:** California **Status:** RECRUITING **Zip:** 90095 #### Overall Officials **Official 1:** **Affiliation:** University of California, Los Angeles **Name:** Jeffrey J. Hsu, MD, PhD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Barizien N, Le Guen M, Russel S, Touche P, Huang F, Vallee A. Clinical characterization of dysautonomia in long COVID-19 patients. Sci Rep. 2021 Jul 7;11(1):14042. doi: 10.1038/s41598-021-93546-5. **PMID:** 34234251 **Citation:** Bisaccia G, Ricci F, Recce V, Serio A, Iannetti G, Chahal AA, Stahlberg M, Khanji MY, Fedorowski A, Gallina S. Post-Acute Sequelae of COVID-19 and Cardiovascular Autonomic Dysfunction: What Do We Know? J Cardiovasc Dev Dis. 2021 Nov 15;8(11):156. doi: 10.3390/jcdd8110156. **PMID:** 34821709 **Citation:** Raj SR, Arnold AC, Barboi A, Claydon VE, Limberg JK, Lucci VM, Numan M, Peltier A, Snapper H, Vernino S; American Autonomic Society. Long-COVID postural tachycardia syndrome: an American Autonomic Society statement. Clin Auton Res. 2021 Jun;31(3):365-368. doi: 10.1007/s10286-021-00798-2. 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Clin Psychol Rev. 2016 Apr;45:102-14. doi: 10.1016/j.cpr.2016.03.009. Epub 2016 Apr 1. **PMID:** 27111302 **Citation:** Simonelli C, Paneroni M, Vitacca M, Ambrosino N. Measures of physical performance in COVID-19 patients: a mapping review. Pulmonology. 2021 Nov-Dec;27(6):518-528. doi: 10.1016/j.pulmoe.2021.06.005. Epub 2021 Jun 24. **PMID:** 34284976 **Citation:** Zhang D, Lee EKP, Mak ECW, Ho CY, Wong SYS. Mindfulness-based interventions: an overall review. Br Med Bull. 2021 Jun 10;138(1):41-57. doi: 10.1093/bmb/ldab005. **PMID:** 33884400 **Citation:** Zou L, Sasaki JE, Zeng N, Wang C, Sun L. A Systematic Review With Meta-Analysis of Mindful Exercises on Rehabilitative Outcomes Among Poststroke Patients. Arch Phys Med Rehabil. 2018 Nov;99(11):2355-2364. doi: 10.1016/j.apmr.2018.04.010. Epub 2018 May 5. **PMID:** 29738744 **Citation:** Bower JE, Crosswell AD, Stanton AL, Crespi CM, Winston D, Arevalo J, Ma J, Cole SW, Ganz PA. Mindfulness meditation for younger breast cancer survivors: a randomized controlled trial. Cancer. 2015 Apr 15;121(8):1231-40. doi: 10.1002/cncr.29194. Epub 2014 Dec 23. Erratum In: Cancer. 2015 Jun 1;121(11):1910. **PMID:** 25537522 **Citation:** Boyle CC, Cole SW, Dutcher JM, Eisenberger NI, Bower JE. Changes in eudaimonic well-being and the conserved transcriptional response to adversity in younger breast cancer survivors. Psychoneuroendocrinology. 2019 May;103:173-179. doi: 10.1016/j.psyneuen.2019.01.024. Epub 2019 Jan 23. **PMID:** 30703712 **Citation:** Tawakol A, Ishai A, Takx RA, Figueroa AL, Ali A, Kaiser Y, Truong QA, Solomon CJ, Calcagno C, Mani V, Tang CY, Mulder WJ, Murrough JW, Hoffmann U, Nahrendorf M, Shin LM, Fayad ZA, Pitman RK. Relation between resting amygdalar activity and cardiovascular events: a longitudinal and cohort study. Lancet. 2017 Feb 25;389(10071):834-845. doi: 10.1016/S0140-6736(16)31714-7. Epub 2017 Jan 12. Erratum In: Lancet. 2017 Feb 25;389(10071):804. Lancet. 2017 Feb 25;389(10071):804. **PMID:** 28088338 **Citation:** Scott-Sheldon LAJ, Gathright EC, Donahue ML, Balletto B, Feulner MM, DeCosta J, Cruess DG, Wing RR, Carey MP, Salmoirago-Blotcher E. Mindfulness-Based Interventions for Adults with Cardiovascular Disease: A Systematic Review and Meta-Analysis. Ann Behav Med. 2020 Jan 1;54(1):67-73. doi: 10.1093/abm/kaz020. **PMID:** 31167026 **Citation:** Shaygan M, Yazdani Z, Valibeygi A. The effect of online multimedia psychoeducational interventions on the resilience and perceived stress of hospitalized patients with COVID-19: a pilot cluster randomized parallel-controlled trial. BMC Psychiatry. 2021 Feb 11;21(1):93. doi: 10.1186/s12888-021-03085-6. **PMID:** 33573631 **Citation:** O'Donnell KT, Dunbar M, Speelman DL. Effectiveness of using a meditation app in reducing anxiety and improving well-being during the COVID-19 pandemic: A structured summary of a study protocol for a randomized controlled trial. Trials. 2020 Dec 9;21(1):1006. doi: 10.1186/s13063-020-04935-6. **PMID:** 33298117 **Citation:** Kubo A, Kurtovich E, McGinnis M, Aghaee S, Altschuler A, Quesenberry C Jr, Kolevska T, Liu R, Greyz-Yusupov N, Avins A. Pilot pragmatic randomized trial of mHealth mindfulness-based intervention for advanced cancer patients and their informal caregivers. Psychooncology. 2024 Feb;33(2):e5557. doi: 10.1002/pon.5557. Epub 2020 Oct 5. **PMID:** 32979294 **Citation:** Levine GN, Lange RA, Bairey-Merz CN, Davidson RJ, Jamerson K, Mehta PK, Michos ED, Norris K, Ray IB, Saban KL, Shah T, Stein R, Smith SC Jr; American Heart Association Council on Clinical Cardiology; Council on Cardiovascular and Stroke Nursing; and Council on Hypertension. Meditation and Cardiovascular Risk Reduction: A Scientific Statement From the American Heart Association. J Am Heart Assoc. 2017 Sep 28;6(10):e002218. doi: 10.1161/JAHA.117.002218. **PMID:** 28963100 **Citation:** Campbell-Sills L, Stein MB. Psychometric analysis and refinement of the Connor-davidson Resilience Scale (CD-RISC): Validation of a 10-item measure of resilience. J Trauma Stress. 2007 Dec;20(6):1019-28. doi: 10.1002/jts.20271. **PMID:** 18157881 **Citation:** Cohen S, Kamarck T, Mermelstein R. A global measure of perceived stress. J Health Soc Behav. 1983 Dec;24(4):385-96. No abstract available. **PMID:** 6668417 **Citation:** Finucane C, van Wijnen VK, Fan CW, Soraghan C, Byrne L, Westerhof BE, Freeman R, Fedorowski A, Harms MPM, Wieling W, Kenny R. A practical guide to active stand testing and analysis using continuous beat-to-beat non-invasive blood pressure monitoring. Clin Auton Res. 2019 Aug;29(4):427-441. doi: 10.1007/s10286-019-00606-y. Epub 2019 May 10. **PMID:** 31076939 **Citation:** Keyes CL, Wissing M, Potgieter JP, Temane M, Kruger A, van Rooy S. Evaluation of the mental health continuum-short form (MHC-SF) in setswana-speaking South Africans. Clin Psychol Psychother. 2008 May-Jun;15(3):181-92. doi: 10.1002/cpp.572. **PMID:** 19115439 **Citation:** Kroenke K, Strine TW, Spitzer RL, Williams JB, Berry JT, Mokdad AH. The PHQ-8 as a measure of current depression in the general population. J Affect Disord. 2009 Apr;114(1-3):163-73. doi: 10.1016/j.jad.2008.06.026. Epub 2008 Aug 27. **PMID:** 18752852 **Citation:** Sletten DM, Suarez GA, Low PA, Mandrekar J, Singer W. COMPASS 31: a refined and abbreviated Composite Autonomic Symptom Score. Mayo Clin Proc. 2012 Dec;87(12):1196-201. doi: 10.1016/j.mayocp.2012.10.013. **PMID:** 23218087 **Citation:** Spitzer RL, Kroenke K, Williams JB, Lowe B. A brief measure for assessing generalized anxiety disorder: the GAD-7. Arch Intern Med. 2006 May 22;166(10):1092-7. doi: 10.1001/archinte.166.10.1092. **PMID:** 16717171 **Citation:** Weiss, D. S., & Marmar, C. R. (1996). The Impact of Event Scale - Revised. In J. Wilson & T. M. Keane (Eds.), Assessing psychological trauma and PTSD (pp. 399-411). New York: Guilford. **Citation:** BALKE B. A SIMPLE FIELD TEST FOR THE ASSESSMENT OF PHYSICAL FITNESS. REP 63-6. Rep Civ Aeromed Res Inst US. 1963 Apr:1-8. No abstract available. **PMID:** 14131272 **Citation:** Hann DM, Jacobsen PB, Azzarello LM, Martin SC, Curran SL, Fields KK, Greenberg H, Lyman G. Measurement of fatigue in cancer patients: development and validation of the Fatigue Symptom Inventory. Qual Life Res. 1998 May;7(4):301-10. doi: 10.1023/a:1024929829627. **PMID:** 9610214 **Citation:** Bastien CH, Vallieres A, Morin CM. Validation of the Insomnia Severity Index as an outcome measure for insomnia research. 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No abstract available. **PMID:** 3393032 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007239 - Term: Infections - ID: D000011024 - Term: Pneumonia, Viral - ID: D000011014 - Term: Pneumonia - ID: D000012141 - Term: Respiratory Tract Infections - ID: D000014777 - Term: Virus Diseases - ID: D000018352 - Term: Coronavirus Infections - ID: D000003333 - Term: Coronaviridae Infections - ID: D000030341 - Term: Nidovirales Infections - ID: D000012327 - Term: RNA Virus Infections - ID: D000008171 - Term: Lung Diseases - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000094025 - Term: Post-Infectious Disorders - ID: D000002908 - Term: Chronic Disease - ID: D000020969 - Term: Disease Attributes - ID: D000010335 - Term: Pathologic Processes - ID: D000009422 - Term: Nervous System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC01 - Name: Infections - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M2561 - Name: COVID-19 - Relevance: HIGH - As Found: SARS-CoV-2 Infection - ID: M10283 - Name: Infections - Relevance: LOW - As Found: Unknown - ID: M6368 - Name: Communicable Diseases - Relevance: LOW - As Found: Unknown - ID: M3013 - Name: Post-Acute COVID-19 Syndrome - Relevance: HIGH - As Found: Post-Acute Sequelae of SARS-CoV-2 Infection - ID: M4643 - Name: Autonomic Nervous System Diseases - Relevance: HIGH - As Found: Dysautonomia - ID: M27979 - Name: Primary Dysautonomias - Relevance: HIGH - As Found: Dysautonomia - ID: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M13904 - Name: Pneumonia - Relevance: LOW - As Found: Unknown - ID: M13914 - Name: Pneumonia, Viral - Relevance: LOW - As Found: Unknown - ID: M14978 - Name: Respiratory Tract Infections - Relevance: LOW - As Found: Unknown - ID: M17522 - Name: Virus Diseases - Relevance: LOW - As Found: Unknown - ID: M20490 - Name: Coronavirus Infections - Relevance: LOW - As Found: Unknown - ID: M6555 - Name: Coronaviridae Infections - Relevance: LOW - As Found: Unknown - ID: M23685 - Name: Nidovirales Infections - Relevance: LOW - As Found: Unknown - ID: M15149 - Name: RNA Virus Infections - Relevance: LOW - As Found: Unknown - ID: M11168 - Name: Lung Diseases - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown - ID: M3014 - Name: Post-Infectious Disorders - Relevance: LOW - As Found: Unknown - ID: M6147 - Name: Chronic Disease - Relevance: LOW - As Found: Unknown - ID: M22700 - Name: Disease Attributes - Relevance: LOW - As Found: Unknown - ID: T6034 - Name: Quality of Life - Relevance: LOW - As Found: Unknown - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown - ID: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000086382 - Term: COVID-19 - ID: D000094024 - Term: Post-Acute COVID-19 Syndrome - ID: D000001342 - Term: Autonomic Nervous System Diseases - ID: D000054969 - Term: Primary Dysautonomias ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05505279 **Brief Title:** Ventilatory Effects of THRIVE During EBUS **Official Title:** Does High Flow Nasal Canula (HFNC) Prevent Hypercapnia During EBUS Under Procedural Sedation? #### Organization Study ID Info **ID:** 2022-2031 #### Organization **Class:** OTHER **Full Name:** Rijnstate Hospital ### Status Module #### Completion Date **Date:** 2024-04 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-11-18 **Type:** ACTUAL **Last Update Submit Date:** 2023-11-15 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-04 **Type:** ESTIMATED #### Start Date **Date:** 2022-10-05 **Type:** ACTUAL **Status Verified Date:** 2023-11 #### Study First Post Date **Date:** 2022-08-17 **Type:** ACTUAL **Study First Submit Date:** 2022-08-10 **Study First Submit QC Date:** 2022-08-15 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Fisher and Paykel Healthcare #### Lead Sponsor **Class:** OTHER **Name:** Rijnstate Hospital #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Is US Export:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** High flow nasal cannula (HFNC) is used in interventional procedures to prevent hypoxia during sedation. In patients with a patent airway, HFNC reduces dead space ventilation as well. It is unknown if dead space ventilation is also reduced by HFNC in an EndoBroncheal UltraSound procedure, in which the airway is partially blocked by the endoscope. Especially in patients with Chronic Obstructive Pulmonary Disease (COPD) the partial blocking of the airway may reduce ventilation. If HFNC is able to reduce dead space during an EBUS-procedure, it may facilitate CO2 clearance, which may lead to a reduction in work of breathing. This study aims to investigate if HFNC reduces dead space ventilation in patients undergoing an EBUS-procedure and if this is flow-dependent. A randomized, double-blinded, cross-over study is designed. ### Conditions Module **Conditions:** - Hypercapnia - Sedation Complication - High Flow Nasal Cannula **Keywords:** - High Flow Nasal Cannula - Sedation - Dead space ventilation ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER ##### Masking Info **Masking:** TRIPLE **Masking Description:** The patient is sedated and the investigators are blinded for the randomization. Only the care provider is aware of the randomisation. **Who Masked:** - PARTICIPANT - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Count:** 20 **Type:** ESTIMATED **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Initially, all patients will receive 3L/min of oxygen for 10 minutes. Than, patients in this arm will receive 30L/min of High Flow Nasal Oxygen for 15 minutes and subsequently 70 L/min for another 15 minutes. **Intervention Names:** - Device: THRIVE (High Flow Nasal Cannula) **Label:** 30 L - 70 L **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Initially, all patients will receive 3L/min of oxygen for 10 minutes. Than, patients in this arm will receive 70L/min of High Flow Nasal Oxygen for 15 minutes and subsequently 30 L/min for another 15 minutes. **Intervention Names:** - Device: THRIVE (High Flow Nasal Cannula) **Label:** 70 L - 30 L **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - 30 L - 70 L - 70 L - 30 L **Description:** 2 different rates of nasal flow compared to each other and a baseline of level of nasal oxygen **Name:** THRIVE (High Flow Nasal Cannula) **Other Names:** - THRIVE - Optiflow - High Flow Nasal Cannula - High Flow Nasal Oxygen **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Inspiratory CO2, measured at the carinal level **Measure:** Inspiratory CO2 at 10 minutes (baseline) **Time Frame:** at t=10 minutes **Description:** Inspiratory CO2, measured at the carinal level **Measure:** Inspiratory CO2 at 25 minutes **Time Frame:** at=25 minutes **Description:** Inspiratory CO2, measured at the carinal level **Measure:** Inspiratory CO2 at 45 minutes **Time Frame:** at t=45 minutes **Description:** Expiratory CO2, measured at the carinal level **Measure:** expiratory CO2 at 10 minutes (baseline) **Time Frame:** at t=10 minutes **Description:** Expiratory CO2, measured at the carinal level **Measure:** expiratory CO2 at 25 minutes **Time Frame:** at t=25 minutes **Description:** Expiratory CO2, measured at the carinal level **Measure:** expiratory CO2 at 45 minutes **Time Frame:** t=45 minutes **Description:** Angle of the D-E segment (inspiratory slope) **Measure:** Slope of capnography at 10 minutes (baseline) **Time Frame:** At t=10 minutes **Description:** Angle of the D-E segment (inspiratory slope) **Measure:** Slope of capnography at 25 minutes **Time Frame:** At t=25 minutes **Description:** Angle of the D-E segment (inspiratory slope) **Measure:** Slope of capnography at 45 minutes **Time Frame:** At t=45 minutes #### Secondary Outcomes **Description:** Respiratory rate, measured by capnography **Measure:** Respiratory rate **Time Frame:** At t=10 minutes **Description:** Respiratory rate, measured by capnography **Measure:** Respiratory rate **Time Frame:** At t=25 minutes **Description:** Respiratory rate, measured by capnography **Measure:** Respiratory rate **Time Frame:** At t=45 minutes **Description:** Levels of inspiratory and expiratory CO2 measured at the level of the left main bronchus, level of carina and the subglottic level. **Measure:** The level of HFNC dead space washout **Time Frame:** At t=10 minutes **Description:** Levels of inspiratory and expiratory CO2 measured at the level of the left main bronchus, level of carina and the subglottic level. **Measure:** The level of HFNC dead space washout **Time Frame:** At t=25 minutes **Description:** Levels of inspiratory and expiratory CO2 measured at the level of the left main bronchus, level of carina and the subglottic level. **Measure:** The level of HFNC dead space washout **Time Frame:** At t=45 minutes ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Adult patients with COPD Gold classification 3 or 4 * Scheduled EBUS with sedation Exclusion Criteria: * Known neurodegenerative diseases, such as Amyotrophic Lateral Sclerosis, dementia, Multiple Sclerosis or Guillain-Barré. * Allergy or intolerance for propofol or esketamine * Severe pulmonary hypertension (PAPsyst \> 60 mmHg) * Pregnancy * upper airway obstruction, such as subglottic stenosis or obstructing tumors. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Jeffrey Miechels **Phone:** 0031-88-0058888 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Mark V Koning, MD, PhD **Phone:** 0031-88-0058888 **Role:** CONTACT #### Locations **Location 1:** **City:** Arnhem **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Jeffrey Miechels - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Mark V Koning, MD, PhD - **Role:** CONTACT ***Contact 3:*** - **Name:** Niels Claessens, MD, PhD - **Role:** SUB_INVESTIGATOR **Country:** Netherlands **Facility:** Rijnstate Hospital **Status:** RECRUITING **Zip:** 6815 AD #### Overall Officials **Official 1:** **Affiliation:** Dept. of Anesthesiology and Critical Care, Rijnstate Hospital, Arnhem **Name:** Jeffrey Miechels **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** Dept. of Anesthesiology and Critical Care, Rijnstate Hospital, Arnhem **Name:** Mark V Koning, MD, PhD **Role:** STUDY_CHAIR **Official 3:** **Affiliation:** Dept. of Pulmonology, Rijnstate Hospital, Arnhem **Name:** Niels Claessens, MD, PhD **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **Description:** Upon reasonable request and within the limits of the law on patient data distribution. **IPD Sharing:** YES ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012818 - Term: Signs and Symptoms, Respiratory ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M6845 - Name: Death - Relevance: LOW - As Found: Unknown - ID: M9986 - Name: Hypercapnia - Relevance: HIGH - As Found: Hypercapnia - ID: M15623 - Name: Signs and Symptoms, Respiratory - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006935 - Term: Hypercapnia ### Intervention Browse Module - Ancestors - ID: D000005731 - Term: Ganglionic Stimulants - ID: D000001337 - Term: Autonomic Agents - ID: D000018373 - Term: Peripheral Nervous System Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018722 - Term: Nicotinic Agonists - ID: D000018679 - Term: Cholinergic Agonists - ID: D000018678 - Term: Cholinergic Agents - ID: D000018377 - Term: Neurotransmitter Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: CNSSti - Name: Central Nervous System Stimulants ### Intervention Browse Module - Browse Leaves - ID: M12478 - Name: Nicotine - Relevance: HIGH - As Found: >50 - ID: M4029 - Name: Central Nervous System Stimulants - Relevance: LOW - As Found: Unknown - ID: M20796 - Name: Nicotinic Agonists - Relevance: LOW - As Found: Unknown - ID: M20758 - Name: Cholinergic Agents - Relevance: LOW - As Found: Unknown - ID: M20504 - Name: Neurotransmitter Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000009538 - Term: Nicotine ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02820779 **Acronym:** COVER **Brief Title:** New Covered Stent (Willis) for the Endovascular Reconstruction of Intracranial Vessel Wall Defects Registry **Official Title:** New Covered Stent (Willis) for the Endovascular Reconstruction of Intracranial Vessel Wall Defects Registry #### Organization Study ID Info **ID:** COVER 001 #### Organization **Class:** OTHER **Full Name:** Xuanwu Hospital, Beijing ### Status Module #### Completion Date **Date:** 2019-05 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** NOT_YET_RECRUITING #### Last Update Post Date **Date:** 2016-07-01 **Type:** ESTIMATED **Last Update Submit Date:** 2016-06-28 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2019-05 **Type:** ESTIMATED #### Start Date **Date:** 2016-06 **Status Verified Date:** 2016-06 #### Study First Post Date **Date:** 2016-07-01 **Type:** ESTIMATED **Study First Submit Date:** 2016-06-23 **Study First Submit QC Date:** 2016-06-28 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** First Affiliated Hospital of Harbin Medical University **Class:** OTHER **Name:** The Second Affiliated Hospital of Harbin Medical University **Class:** OTHER **Name:** Qilu Hospital of Shandong University **Class:** OTHER **Name:** Shanghai Tongji Hospital, Tongji University School of Medicine **Class:** OTHER **Name:** Nanfang Hospital, Southern Medical University **Class:** OTHER **Name:** Tang-Du Hospital **Class:** OTHER **Name:** West China Hospital **Class:** OTHER **Name:** The First Affiliated Hospital of Zhengzhou University **Class:** OTHER **Name:** Shaanxi Provincial People's Hospital **Class:** OTHER_GOV **Name:** Shandong Provincial Hospital **Class:** OTHER **Name:** First Affiliated Hospital of Xinjiang Medical University #### Lead Sponsor **Class:** OTHER **Name:** Xuanwu Hospital, Beijing #### Responsible Party **Investigator Affiliation:** Xuanwu Hospital, Beijing **Investigator Full Name:** Hongqi Zhang, MD **Investigator Title:** Cerebrovascular Expert Committee of Chinese Medical Doctor Association **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to evaluate the effectiveness, long-term safety and explore the safety and efficacy factors WILLIS™ intracranial stent graft system in clinical applications. **Detailed Description:** Intracranial aneurysm is a cerebrovascular disorder in which weakness in the wall of a cerebral artery or vein causes a localized dilation or ballooning of the blood vessel.WILLIS® intracranial stent graft system is indicated for the treatment of intracranial aneurysms. This study is a multi-center, prospective registration study. Recruiting 100 specific patients to undergo a treatment with the WILLIS intracranial stent graft system, while giving the anti-platelet aggregation drugs and other medical therapy. Patients enrolled will undergo a one-year follow-up. ### Conditions Module **Conditions:** - Aneurysm, Intracranial - Carotid-Cavernous Sinus Fistula **Keywords:** - Willis - Intracranial aneurysms ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 100 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients undergo WILLIS intracranial covered stent interventional treatment **Intervention Names:** - Device: WILLIS **Label:** WILLIS **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - WILLIS **Description:** WILLIS® intracranial stent graft system is composed of the stent, delivery system, cobalt-based alloy stent and PTFE graft. It can effectively shunt the blood flow and keep it off of the aneurysm wall. **Name:** WILLIS **Other Names:** - WILLIS® intracranial stent graft system **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Treatment success is defined as target lesion such as aneurysms, cavernous fistula shows no shadow and target vessel stenosis of no more than 50%. **Measure:** 1-year treatment success rate **Time Frame:** 12 months #### Secondary Outcomes **Description:** Technically success is defined as stent reach the target lesion and successfully released. **Measure:** Technically success rate of surgery **Time Frame:** immediately after surgery **Measure:** Target lesion treatment success rate **Time Frame:** Immediately after surgery **Measure:** X-ray exposure time **Time Frame:** 24 hours **Measure:** Operative time **Time Frame:** through surgery completion **Measure:** Surgery-related complications or death **Time Frame:** 12 months **Measure:** Various causes of death **Time Frame:** the perioperative period to 12 months **Measure:** Recurrence of target lesion **Time Frame:** 12 months **Measure:** Target lesion was treated by interventional or surgical therapy once again **Time Frame:** 12 months **Description:** Ipsilateral symptomatic stroke is defined as the target lesion is conformed occurs ipsilateral stroke and the increase of NIHSS score point ≥4. **Measure:** Postoperative ipsilateral symptomatic stroke **Time Frame:** 30 days; 6 months; 12 months **Measure:** Occurs intracranial hemorrhagic stroke that caused by aneurysm rupture **Time Frame:** 12 months **Measure:** Occurs intracranial ischemic stroke that caused by thrombus **Time Frame:** 12 months **Measure:** All adverse events **Time Frame:** 12 months **Measure:** Device-related serious adverse events **Time Frame:** 12 months **Description:** Stenosis is defined as target stenosis ≥50% **Measure:** Target lesion appears stenosis 12 months after surgery **Time Frame:** 12 months **Measure:** Modified Rankin Scale **Time Frame:** 30 days; 6 months; 12 months **Measure:** NIHSS **Time Frame:** 30 days; 6 months; 12 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Age over 18; * Arterial wall defects conformed by imaging examination due to internal carotid artery, vertebral artery aneurysms or various causes; * Target lesion vessel reference diameter 3.5-4.5mm; * Investigators believe that the patient is suitable for the interventional therapy of WILLIS intracranial covered stent; * Participate in the study voluntarily, accept follow up study and signed Informed Consent Form. Exclusion Criteria: * Existence of branch artery (eg anterior choroidal artery, posterior communicating artery, posterior inferior cerebellar artery, etc.) of target lesion which may cause severe neurological dysfunction after occlusion; * No suitable vessel entrance, or diseased artery extremely tortuous; * Coagulation disorders or serious heart, liver, kidney dysfunction or systemic infection, which not suitable for interventional treatment; * Life expectancy \<1 year; * Contraindications to heparin, aspirin, clopidogrel, anesthesia, X-ray or contrast agents; * Mental disorder **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Hongqi Zhang, MD **Role:** CONTACT #### Locations **Location 1:** **City:** Guangzhou **Country:** China **Facility:** Nanfang hospital, Southern Medical university **State:** Guangdong **Zip:** 510515 **Location 2:** **City:** Harbin **Country:** China **Facility:** The 2nd Affiliated Hospital of Harbin Medical university **State:** Heilongjiang **Zip:** 150001 **Location 3:** **City:** Harbin **Country:** China **Facility:** The First Affiliated Hospital of Harbin Medical university **State:** Heilongjiang **Zip:** 150001 **Location 4:** **City:** Zhengzhou **Country:** China **Facility:** First Affiliated Hospital of Zhengzhou University **State:** Henan **Zip:** 450052 **Location 5:** **City:** Jinan **Country:** China **Facility:** Qilu Hospital Of Shang Dong University **State:** Shandong **Zip:** 250012 **Location 6:** **City:** Jinan **Country:** China **Facility:** Shandong Provincial Hospital **State:** Shandong **Zip:** 250021 **Location 7:** **City:** Taiyuan **Country:** China **Facility:** ShanXi Provincial People's Hospital **State:** Shanxi **Zip:** 030012 **Location 8:** **City:** Xi'an **Country:** China **Facility:** Tangdu Hospital Fourth Military Medical University **State:** Shanxi **Zip:** 710038 **Location 9:** **City:** Chengdu **Country:** China **Facility:** West China Hospital Sichuan University **State:** Sichuan **Zip:** 610041 **Location 10:** **City:** Urumqi **Country:** China **Facility:** The First Affiliated Hospital Of Xinjiang Medical University **State:** Xinjiang **Zip:** 830054 **Location 11:** **City:** Shanghai **Country:** China **Facility:** Tongji Hospital **Zip:** 200333 #### Overall Officials **Official 1:** **Affiliation:** [email protected] **Name:** Hongqi Zhang, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014652 - Term: Vascular Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000020763 - Term: Pathological Conditions, Anatomical - ID: D000020765 - Term: Intracranial Arterial Diseases - ID: D000002561 - Term: Cerebrovascular Disorders - ID: D000001927 - Term: Brain Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000096826 - Term: Cavernous Sinus Syndromes - ID: D000002340 - Term: Carotid Artery Diseases - ID: D000020212 - Term: Carotid Artery Injuries - ID: D000020214 - Term: Cerebrovascular Trauma - ID: D000020196 - Term: Trauma, Nervous System - ID: D000001164 - Term: Arteriovenous Fistula - ID: D000001165 - Term: Arteriovenous Malformations - ID: D000054079 - Term: Vascular Malformations - ID: D000018376 - Term: Cardiovascular Abnormalities - ID: D000016157 - Term: Vascular Fistula - ID: D000000013 - Term: Congenital Abnormalities - ID: D000014947 - Term: Wounds and Injuries ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - Abbrev: BC26 - Name: Wounds and Injuries ### Condition Browse Module - Browse Leaves - ID: M8532 - Name: Fistula - Relevance: HIGH - As Found: Fistula - ID: M5781 - Name: Intracranial Aneurysm - Relevance: HIGH - As Found: Aneurysm, Intracranial - ID: M4113 - Name: Aneurysm - Relevance: HIGH - As Found: Aneurysm - ID: M22042 - Name: Carotid-Cavernous Sinus Fistula - Relevance: HIGH - As Found: Carotid-Cavernous Sinus Fistula - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown - ID: M22519 - Name: Pathological Conditions, Anatomical - Relevance: LOW - As Found: Unknown - ID: M22521 - Name: Intracranial Arterial Diseases - Relevance: LOW - As Found: Unknown - ID: M5810 - Name: Cerebrovascular Disorders - Relevance: LOW - As Found: Unknown - ID: M5204 - Name: Brain Diseases - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M5594 - Name: Carotid Artery Diseases - Relevance: LOW - As Found: Unknown - ID: M22039 - Name: Carotid Artery Injuries - Relevance: LOW - As Found: Unknown - ID: M17685 - Name: Wounds and Injuries - Relevance: LOW - As Found: Unknown - ID: M22040 - Name: Cerebrovascular Trauma - Relevance: LOW - As Found: Unknown - ID: M22023 - Name: Trauma, Nervous System - Relevance: LOW - As Found: Unknown - ID: M4472 - Name: Arteriovenous Fistula - Relevance: LOW - As Found: Unknown - ID: M12 - Name: Congenital Abnormalities - Relevance: LOW - As Found: Unknown - ID: M4473 - Name: Arteriovenous Malformations - Relevance: LOW - As Found: Unknown - ID: M27558 - Name: Vascular Malformations - Relevance: LOW - As Found: Unknown - ID: M20503 - Name: Cardiovascular Abnormalities - Relevance: LOW - As Found: Unknown - ID: M18619 - Name: Vascular Fistula - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000002532 - Term: Intracranial Aneurysm - ID: D000020216 - Term: Carotid-Cavernous Sinus Fistula - ID: D000000783 - Term: Aneurysm - ID: D000005402 - Term: Fistula ### Intervention Browse Module - Browse Branches - Abbrev: Micro - Name: Micronutrients - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M6265 - Name: Cobalt - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01433679 **Brief Title:** The Effect of a Web-Based Behavioral Intervention on Physical Activity Levels in Adolescents **Official Title:** The Effect of a Web-Based Behavioral Intervention on Physical Activity Levels in Adolescents #### Organization Study ID Info **ID:** HLZZ-001 #### Organization **Class:** OTHER **Full Name:** HopeLab Foundation ### Status Module #### Completion Date **Date:** 2012-07 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2012-07-16 **Type:** ESTIMATED **Last Update Submit Date:** 2012-07-12 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2012-07 **Type:** ACTUAL #### Start Date **Date:** 2011-09 **Status Verified Date:** 2012-07 #### Study First Post Date **Date:** 2011-09-14 **Type:** ESTIMATED **Study First Submit Date:** 2011-09-12 **Study First Submit QC Date:** 2011-09-12 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** West Virginia University #### Lead Sponsor **Class:** OTHER **Name:** HopeLab Foundation #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The primary purpose of this study is to test whether rewarding physical activity with a motivational website will increase physical activity levels in middle school-aged children over six months. As a secondary outcome, the study also tests the intervention's impact on biological measures of inflammation and metabolic function in a sub-set of study participants who agree to provide blood samples. **Detailed Description:** Physical activity is associated with a variety of positive health outcomes, as well as improved metabolic profiles and reduced inflammation. However, levels of moderate to vigorous physical activity (MVPA) diminish dramatically as children move into the middle school years. To address this problem, this study tests a behavioral intervention, called "Zamzee," designed to motivate middle school-aged children to increase their levels of MVPA. The Zamzee intervention consists of a 3-axis accelerometer that tracks individual physical activity rates over time and a website that displays individual physical activity rates and provides rewards for maintaining or improving physical activity rates. The primary aim of this randomized, controlled trial is to test whether middle school-aged children randomly assigned to the Zamzee intervention will show significantly greater levels of physical activity levels over six months, compared with control group participants who wear the accelerometer but have no access to the rewards website. A secondary aim is to test the intervention's impact on biological parameters that may contribute to the long-term health effects of inactivity (including C-reactive protein as a measure of inflammation, and hemoglobin-A1C as a measure of metabolic status) in a sub-set of study participants who agree to provide blood samples. ### Conditions Module **Conditions:** - Health Behavior - Adolescent Behavior - Motivation **Keywords:** - Health Behavior - Exercise - Adolescent Behavior - Motivation - Biological Markers ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 448 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants randomly assigned to the Website Intervention arm receive access to the motivational rewards website. The website displays the individual's physical activity data and allocates reward points based on the amount and intensity of physical activity. The website also allows reward points to be redeemed for various rewards such as gift cards to retail outlets, donations to charities, small tangible goods, and customization of participants' cartoon-like avatars on the website. **Intervention Names:** - Behavioral: Zamzee Intervention **Label:** Website intervention **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants in the control group will not have access to the motivational website. No other product or intervention will be introduced to the control group. **Label:** Control **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Website intervention **Description:** The Zamzee intervention is designed to motivate middle school-aged children to increase their rates of moderate to vigorous physical activity (MVPA) by providing rewards based on amount and duration of physical activity. Rewards include gift cards to retail stores, donations to charity, small tangible goods, and customization of their cartoon-like avatars on the website. **Name:** Zamzee Intervention **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** The amount and intensity of physical activity is recorded continuously every study day that participants wear their accelerometer for the duration of the six month study. The primary outcome reported will be rates of "moderate to vigorous physical activity" as defined by the CDC. **Measure:** Physical activity **Time Frame:** Six months #### Secondary Outcomes **Description:** In the subset of study participants who agree to provide blood samples, blood samples collected at study baseline and at the six month study completion will be assayed for measures of inflammation (e.g., C-reactive protein) and metabolic function (e.g.,hemoglobin-A1C). **Measure:** Metabolic and inflammatory biomarkers **Time Frame:** Six months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Middle-school-aged students aged 11 to 14 Exclusion Criteria: * Previously participation in a Zamzee pilot study * Existing medical conditions or health complications that will interfere with the ability to be physically active * Inability to read and write English **Healthy Volunteers:** True **Maximum Age:** 14 Years **Minimum Age:** 11 Years **Sex:** ALL **Standard Ages:** - CHILD ### Contacts Locations Module #### Locations **Location 1:** **City:** Los Osos **Country:** United States **Facility:** Los Osos Middle School **State:** California **Zip:** 93402 **Location 2:** **City:** Oakland **Country:** United States **Facility:** Berkley Maynard Academy **State:** California **Zip:** 94605 **Location 3:** **City:** Oakland **Country:** United States **Facility:** E.C. Reems Academy **State:** California **Zip:** 94605 **Location 4:** **City:** Pismo Beach **Country:** United States **Facility:** Judkins Middle School **State:** California **Zip:** 93449 **Location 5:** **City:** Vista **Country:** United States **Facility:** Vista Academy of Visual and Performing Arts **State:** California **Zip:** 92083 **Location 6:** **City:** Morgantown **Country:** United States **Facility:** West Virginia University **State:** West Virginia **Zip:** 26506-6845 #### Overall Officials **Official 1:** **Affiliation:** HopeLab Foundation **Name:** Jana Haritatos, PhD **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** HopeLab Foundation **Name:** Steve Cole, PhD **Role:** STUDY_DIRECTOR ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02573779 **Brief Title:** Growth and Microbiome Development in Very Low Birth Weight Infants Fed Primarily Mother's Own Milk vs. Donor Human Milk **Official Title:** Intestinal Microbiota and Short Term Outcomes in Very Low Birth Weight Infants Fed Primarily Donor Human Milk Compared to Infants Fed Primarily Mother's Own Milk #### Organization Study ID Info **ID:** H-36828 #### Organization **Class:** OTHER **Full Name:** Baylor College of Medicine ### Status Module #### Completion Date **Date:** 2018-10-25 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2024-02-21 **Type:** ACTUAL **Last Update Submit Date:** 2024-02-19 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2016-09-30 **Type:** ACTUAL #### Results First Post Date **Date:** 2020-04-29 **Type:** ACTUAL **Results First Submit Date:** 2019-12-20 **Results First Submit QC Date:** 2020-04-20 #### Start Date **Date:** 2015-07-10 **Type:** ACTUAL **Status Verified Date:** 2024-02 #### Study First Post Date **Date:** 2015-10-12 **Type:** ESTIMATED **Study First Submit Date:** 2015-10-08 **Study First Submit QC Date:** 2015-10-09 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Baylor College of Medicine #### Responsible Party **Investigator Affiliation:** Baylor College of Medicine **Investigator Full Name:** Amy Hair **Investigator Title:** Assistant Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** A study to compare growth, development of the intestinal bacterial environment, and other short term outcomes in groups of babies fed primarily their own mother's milk compared to those who receive primarily donor human milk. The investigators hypothesize that infants who receive primarily their own mother's milk will have better growth, a more diverse intestinal bacterial environment, and possibly some improved short term outcomes such as better feeding tolerance and lower rates of infection. **Detailed Description:** Background: Human milk feeding provides numerous benefits to preterm infants due to improvements in gastrointestinal maturation, host defense, infection rates, and improved long-term outcomes in neurodevelopment as well as cardiovascular and metabolic disease. There is accumulating evidence that an exclusive human milk-based diet decreases the rates of necrotizing enterocolitis (NEC) and death, and is associated with better feeding tolerance in very low birth weight (VLBW) infants than a diet of bovine milk-based products. In order to provide VLBW infants the benefits afforded by human milk feeding, the use of donor milk (DM) in neonatal intensive care units (NICU) has increased as many mothers are unable to provide sufficient milk needed for their premature infants. While there have been numerous studies that have favorably compared feeding of mother's own milk (MOM) to formula as well as studies that compare DM to formula, there are relatively few that compare maternal milk to donor milk. In regard to feeding tolerance and infection prevention, it has been proposed that DM may be less beneficial than MOM due to reduction in biologically active components during pasteurization, including human milk oligosaccharides (HMOs) and other immunological factors, growth factors, and hormones. Finally, alterations in the intestinal microbiota of preterm infants are suspected to contribute to disease states such as NEC, specifically within infants who have decreased microbial diversity. To the investigators knowledge, no studies comparing the intestinal microbiota among infants fed primarily MOM versus those fed primarily DM have been published. Purpose: To compare growth velocities, time to reach full enteral feeding volume, intestinal microbiota, and short term outcomes (NEC, late-onset sepsis, white matter injury) between infants fed primarily mother's own milk versus pasteurized donor human milk. Hypothesis: Infants fed primarily (50% or greater) mother's own milk will have increased intestinal microbiome diversity compared to infants fed primarily pasteurized donor human milk. Design: This prospective cohort study will be conducted in the Level III NICU at Texas Children's Hospital - Pavilion for Women and the Level II NICU at Texas Children's Hospital -- West Tower. Infants less than 1500 g birth weight will be fed exclusively human milk (mother's milk and or donor breast milk) fortified with donor human milk-derived fortifier per the investigators hospital guidelines. Once enteral feeding is established, infants will be categorized into cohorts based on percentage of feeding volume consisting of mother's own milk, including broad categorization of greater than 50% maternal milk versus less than 50%, and possibly tiered analysis of infants who receive less 25% maternal milk, 25-75% maternal milk, and greater than 75% maternal milk. An enrollment goal of greater than 125 infants including twins and multiples will be targeted for adequate sample size. Procedure: Infants will be enrolled within 72 hours of birth and started on parenteral nutrition and enteral human milk feedings per standardized feeding protocols and discretion of the attending neonatologist on service. Decisions to decrease or discontinue enteral feedings due to medical instability will be made by the attending neonatologist. Infants will be preferentially fed their own mothers' milk when available. For mothers who are unable to express adequate milk volume for their infants, pooled, pasteurized donor human milk will be offered per established NICU protocol. Feeds will be supplemented with human-milk based fortifier per protocol. At time of initial consent for the study, mothers will also be asked for consent to obtain a small sample (0.2-0.5 mL) of colostrum or expressed milk produced in the first week of life, as well as weekly milk samples thereafter, in order to analyze bacterial content of milk as it compares to the developing infant microbiome. However, consent for milk collection is not required for the infant's participation in the study. For infants who receive primarily donor milk, weekly samples of the milk they receive may similarly be analyzed for bacterial content. Infant stool samples will be collected during the first week of life and then at weekly intervals for six weeks for research purposes. The samples will be analyzed via 16S rRNA sequencing to determine diversity of intestinal microbiota. Additional analysis for metabolomics will be considered if lab availability and cost allows. Once weekly, a research nurse or physician will document growth measurements, including weight, length, and head circumference. Outcome data from the infants' medical records will be recorded, including time to regain birth weight, feeding tolerance as indicated by time required to reach full enteral feeding volumes of 100 ml/kg/day (for hydration) and 130-160 ml/kg/day (final goal volume for nutrition), and rates of NEC, spontaneous intestinal perforation (SIP), late-onset sepsis, and bronchopulmonary dysplasia (BPD). As all preterm infants less than 1000 g birth weight born at TCH-PFW have routinely performed brain MRIs at term gestation or hospital discharge, enrolled infants who fall into this subgroup will have their MRI results reviewed for outcome analysis. Additional medical record data for collection will include mechanism of delivery (cesarean vs. vaginal), antibiotics received by mother during 2nd and 3rd trimester as well as at time of delivery and while nursing/expressing milk, antibiotics received by baby during hospitalization, length of hospitalization, postmenstrual age at discharge, days NPO, days of parenteral nutrition, percentage of patients with patent ductus arteriosus, and rates of intraventricular hemorrhage, retinopathy of prematurity, and death. Although there is no long-term follow up currently designed for this study, at time of initial enrollment there will be an optional consent to allow study personnel to access medical records for patients who go on to have neurodevelopmental follow up visits at clinics within the investigators institution. There will also be optional consent for families to be contacted when the infant is approximately 6, 12, 18, and 24 months for developmental follow up. This will allow for possible comparison of neurodevelopmental outcomes at approximately 18-24 months in the subgroup of patients whose mothers consent to these aspects of the study. No labs will be requested for research purposes. No interventions are part of this protocol. ### Conditions Module **Conditions:** - Infant, Very Low Birth Weight **Keywords:** - human milk - enteral nutrition - microbiome - microbiota ### Design Module #### Bio Spec **Description:** At time of initial consent for the study, mothers will also be asked for consent to obtain a small sample (0.2-0.5 mL) of colostrum or expressed milk produced in the first week of life, as well as weekly milk samples thereafter, in order to analyze bacterial content of milk as it compares to the developing infant microbiome. For infants who receive primarily donor milk, weekly samples of the milk they receive may similarly be analyzed for bacterial content. Infant stool samples will be collected during the first week of life and then at weekly intervals for six weeks. The samples will be analyzed via 16S rRNA sequencing to determine diversity of intestinal microbiota. Additional analysis for metabolomics will be considered if lab availability and cost allows. **Retention:** SAMPLES_WITH_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 125 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Infants fed \>50% mother's own milk with enteral feeding. **Intervention Names:** - Other: Observational - no intervention **Label:** Infants fed mother's own milk #### Arm Group 2 **Description:** Infants fed \<50% mother's own milk (and thus \>50% donor human milk) with enteral feeding. **Intervention Names:** - Other: Observational - no intervention **Label:** Donor milk fed infants ### Interventions #### Intervention 1 **Arm Group Labels:** - Donor milk fed infants - Infants fed mother's own milk **Description:** This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **Name:** Observational - no intervention **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Stool samples during the first 6 weeks of life will be analyzed to compare development of microbial diversity **Measure:** Intestinal Microbiome Diversity **Time Frame:** 6 weeks **Description:** Hospital length of stay will be calculated from birth to discharge of infant. **Measure:** Hospital Length of Stay **Time Frame:** Birth to discharge **Description:** Weight gain will be evaluated weekly throughout the study (defined as g/kg/day) **Measure:** Weight Gain **Time Frame:** 6-10 weeks **Description:** Linear growth will be measured weekly (defined as cm/week) **Measure:** Linear Growth **Time Frame:** 6-10 weeks **Description:** Growth in head circumference will be measured weekly (defined as cm/wk) **Measure:** Head Circumference Growth **Time Frame:** 6-10 weeks #### Secondary Outcomes **Description:** Number of days for infant to reach full enteral feeds. **Measure:** Days to Final Enteral Feed Volume **Time Frame:** 6-10 weeks **Description:** the number of patients with a diagnosis of NEC (Stage ≥ IIA) will be collected **Measure:** Rates of Necrotizing Enterocolitis (NEC) **Time Frame:** 6-10 weeks **Description:** the number of patients who develop a spontaneous intestinal perforation will be collected **Measure:** Rates of Spontaneous Intestinal Perforation (SIP) **Time Frame:** 6-10 weeks **Description:** the number of patients with late-onset sepsis will be collected **Measure:** Rates of Late-onset Sepsis **Time Frame:** 6-10 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Infants less than 72 hours old and less than 1500 g birth weight, who have reasonable expectation of survival and can adhere to a feeding protocol involving mother's own milk and/or donor milk that will include fortification using Prolacta and potentially human cream. Exclusion Criteria: * Exclusion criteria will include birth weight greater than 1500 g, age \> 72 hours old, major congenital anomalies, clinically significant heart disease, abdominal wall defects and/or intestinal atresias, severe perinatal hypoxia, or otherwise less than reasonable expectation of survival through the study period. **Maximum Age:** 3 Days **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - CHILD **Study Population:** Newborn infants less than 1500 g birth weight who are fed primarily either mother's own milk or donor human milk. ### Contacts Locations Module #### Locations **Location 1:** **City:** Houston **Country:** United States **Facility:** Baylor College of Medicine / Texas Children's Hospital **State:** Texas **Zip:** 77030 **Location 2:** **City:** Houston **Country:** United States **Facility:** Texas Children's Hospital **State:** Texas **Zip:** 77030 #### Overall Officials **Official 1:** **Affiliation:** Baylor College of Medicine - Texas Children's Hospital **Name:** Amy Hair, MD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Schanler RJ. Outcomes of human milk-fed premature infants. Semin Perinatol. 2011 Feb;35(1):29-33. doi: 10.1053/j.semperi.2010.10.005. **PMID:** 21255704 **Citation:** Sullivan S, Schanler RJ, Kim JH, Patel AL, Trawoger R, Kiechl-Kohlendorfer U, Chan GM, Blanco CL, Abrams S, Cotten CM, Laroia N, Ehrenkranz RA, Dudell G, Cristofalo EA, Meier P, Lee ML, Rechtman DJ, Lucas A. An exclusively human milk-based diet is associated with a lower rate of necrotizing enterocolitis than a diet of human milk and bovine milk-based products. J Pediatr. 2010 Apr;156(4):562-7.e1. doi: 10.1016/j.jpeds.2009.10.040. Epub 2009 Dec 29. **PMID:** 20036378 **Citation:** Cristofalo EA, Schanler RJ, Blanco CL, Sullivan S, Trawoeger R, Kiechl-Kohlendorfer U, Dudell G, Rechtman DJ, Lee ML, Lucas A, Abrams S. Randomized trial of exclusive human milk versus preterm formula diets in extremely premature infants. J Pediatr. 2013 Dec;163(6):1592-1595.e1. doi: 10.1016/j.jpeds.2013.07.011. Epub 2013 Aug 20. **PMID:** 23968744 **Citation:** Abrams SA, Schanler RJ, Lee ML, Rechtman DJ. Greater mortality and morbidity in extremely preterm infants fed a diet containing cow milk protein products. Breastfeed Med. 2014 Jul-Aug;9(6):281-5. doi: 10.1089/bfm.2014.0024. Epub 2014 May 27. **PMID:** 24867268 **Citation:** Sisk PM, Lovelady CA, Gruber KJ, Dillard RG, O'Shea TM. Human milk consumption and full enteral feeding among infants who weigh </= 1250 grams. Pediatrics. 2008 Jun;121(6):e1528-33. doi: 10.1542/peds.2007-2110. Erratum In: Pediatrics. 2008 Nov;122(5):1162-3. **PMID:** 18519456 **Citation:** Schanler RJ, Shulman RJ, Lau C. Feeding strategies for premature infants: beneficial outcomes of feeding fortified human milk versus preterm formula. Pediatrics. 1999 Jun;103(6 Pt 1):1150-7. doi: 10.1542/peds.103.6.1150. **PMID:** 10353922 **Citation:** Section on Breastfeeding. Breastfeeding and the use of human milk. Pediatrics. 2012 Mar;129(3):e827-41. doi: 10.1542/peds.2011-3552. Epub 2012 Feb 27. **PMID:** 22371471 **Citation:** Schanler RJ. Mother's own milk, donor human milk, and preterm formulas in the feeding of extremely premature infants. J Pediatr Gastroenterol Nutr. 2007 Dec;45 Suppl 3:S175-7. doi: 10.1097/01.mpg.0000302967.83244.36. Erratum In: J Pediatr Gastroenterol Nutr. 2009 Jan;48(1):121-2. **PMID:** 18185087 **Citation:** Hair AB, Hawthorne KM, Chetta KE, Abrams SA. Human milk feeding supports adequate growth in infants </= 1250 grams birth weight. BMC Res Notes. 2013 Nov 13;6:459. doi: 10.1186/1756-0500-6-459. **PMID:** 24220185 **Citation:** Hair AB, Blanco CL, Moreira AG, Hawthorne KM, Lee ML, Rechtman DJ, Abrams SA. Randomized trial of human milk cream as a supplement to standard fortification of an exclusive human milk-based diet in infants 750-1250 g birth weight. J Pediatr. 2014 Nov;165(5):915-20. doi: 10.1016/j.jpeds.2014.07.005. Epub 2014 Aug 15. **PMID:** 25130571 **Citation:** Dewey KG, Heinig MJ, Nommsen-Rivers LA. Differences in morbidity between breast-fed and formula-fed infants. J Pediatr. 1995 May;126(5 Pt 1):696-702. doi: 10.1016/s0022-3476(95)70395-0. **PMID:** 7751991 **Citation:** Le Huerou-Luron I, Blat S, Boudry G. Breast- v. formula-feeding: impacts on the digestive tract and immediate and long-term health effects. Nutr Res Rev. 2010 Jun;23(1):23-36. doi: 10.1017/S0954422410000065. Epub 2010 May 10. **PMID:** 20450531 **Citation:** Duijts L, Ramadhani MK, Moll HA. Breastfeeding protects against infectious diseases during infancy in industrialized countries. A systematic review. Matern Child Nutr. 2009 Jul;5(3):199-210. doi: 10.1111/j.1740-8709.2008.00176.x. **PMID:** 19531047 **Citation:** ESPGHAN Committee on Nutrition; Arslanoglu S, Corpeleijn W, Moro G, Braegger C, Campoy C, Colomb V, Decsi T, Domellof M, Fewtrell M, Hojsak I, Mihatsch W, Molgaard C, Shamir R, Turck D, van Goudoever J. Donor human milk for preterm infants: current evidence and research directions. J Pediatr Gastroenterol Nutr. 2013 Oct;57(4):535-42. doi: 10.1097/MPG.0b013e3182a3af0a. **PMID:** 24084373 **Citation:** Bertino E, Giuliani F, Baricco M, Di Nicola P, Peila C, Vassia C, Chiale F, Pirra A, Cresi F, Martano C, Coscia A. Benefits of donor milk in the feeding of preterm infants. Early Hum Dev. 2013 Oct;89 Suppl 2:S3-6. doi: 10.1016/j.earlhumdev.2013.07.008. Epub 2013 Aug 6. **PMID:** 23932110 **Citation:** Quigley M, McGuire W. Formula versus donor breast milk for feeding preterm or low birth weight infants. Cochrane Database Syst Rev. 2014 Apr 22;(4):CD002971. doi: 10.1002/14651858.CD002971.pub3. **PMID:** 24752468 **Citation:** Schanler RJ, Lau C, Hurst NM, Smith EO. Randomized trial of donor human milk versus preterm formula as substitutes for mothers' own milk in the feeding of extremely premature infants. Pediatrics. 2005 Aug;116(2):400-6. doi: 10.1542/peds.2004-1974. **PMID:** 16061595 **Citation:** Montjaux-Regis N, Cristini C, Arnaud C, Glorieux I, Vanpee M, Casper C. Improved growth of preterm infants receiving mother's own raw milk compared with pasteurized donor milk. Acta Paediatr. 2011 Dec;100(12):1548-54. doi: 10.1111/j.1651-2227.2011.02389.x. Epub 2011 Jul 14. **PMID:** 21707744 **Citation:** Garcia-Lara NR, Vieco DE, De la Cruz-Bertolo J, Lora-Pablos D, Velasco NU, Pallas-Alonso CR. Effect of Holder pasteurization and frozen storage on macronutrients and energy content of breast milk. J Pediatr Gastroenterol Nutr. 2013 Sep;57(3):377-82. doi: 10.1097/MPG.0b013e31829d4f82. **PMID:** 23752081 **Citation:** Marx C, Bridge R, Wolf AK, Rich W, Kim JH, Bode L. Human milk oligosaccharide composition differs between donor milk and mother's own milk in the NICU. J Hum Lact. 2014 Feb;30(1):54-61. doi: 10.1177/0890334413513923. Epub 2013 Nov 26. **PMID:** 24282194 **Citation:** Evans TJ, Ryley HC, Neale LM, Dodge JA, Lewarne VM. Effect of storage and heat on antimicrobial proteins in human milk. Arch Dis Child. 1978 Mar;53(3):239-41. doi: 10.1136/adc.53.3.239. **PMID:** 306224 **Citation:** Torrazza RM, Neu J. The altered gut microbiome and necrotizing enterocolitis. Clin Perinatol. 2013 Mar;40(1):93-108. doi: 10.1016/j.clp.2012.12.009. **PMID:** 23415266 **Citation:** Wang Y, Hoenig JD, Malin KJ, Qamar S, Petrof EO, Sun J, Antonopoulos DA, Chang EB, Claud EC. 16S rRNA gene-based analysis of fecal microbiota from preterm infants with and without necrotizing enterocolitis. ISME J. 2009 Aug;3(8):944-54. doi: 10.1038/ismej.2009.37. Epub 2009 Apr 16. **PMID:** 19369970 **Citation:** Mai V, Young CM, Ukhanova M, Wang X, Sun Y, Casella G, Theriaque D, Li N, Sharma R, Hudak M, Neu J. Fecal microbiota in premature infants prior to necrotizing enterocolitis. PLoS One. 2011;6(6):e20647. doi: 10.1371/journal.pone.0020647. Epub 2011 Jun 6. **PMID:** 21674011 **Citation:** Harmsen HJ, Wildeboer-Veloo AC, Raangs GC, Wagendorp AA, Klijn N, Bindels JG, Welling GW. Analysis of intestinal flora development in breast-fed and formula-fed infants by using molecular identification and detection methods. J Pediatr Gastroenterol Nutr. 2000 Jan;30(1):61-7. doi: 10.1097/00005176-200001000-00019. **PMID:** 10630441 **Citation:** Hanson LA, Korotkova M, Telemo E. Breast-feeding, infant formulas, and the immune system. Ann Allergy Asthma Immunol. 2003 Jun;90(6 Suppl 3):59-63. doi: 10.1016/s1081-1206(10)61662-6. **PMID:** 12839115 **Citation:** Ward RE, Ninonuevo M, Mills DA, Lebrilla CB, German JB. In vitro fermentation of breast milk oligosaccharides by Bifidobacterium infantis and Lactobacillus gasseri. Appl Environ Microbiol. 2006 Jun;72(6):4497-9. doi: 10.1128/AEM.02515-05. **PMID:** 16751577 **Citation:** Bjorkstrom MV, Hall L, Soderlund S, Hakansson EG, Hakansson S, Domellof M. Intestinal flora in very low-birth weight infants. Acta Paediatr. 2009 Nov;98(11):1762-7. doi: 10.1111/j.1651-2227.2009.01471.x. Epub 2009 Aug 8. **PMID:** 19673724 **Citation:** Ford SL, Lohmann P, Preidis GA, Gordon PS, O'Donnell A, Hagan J, Venkatachalam A, Balderas M, Luna RA, Hair AB. Improved feeding tolerance and growth are linked to increased gut microbial community diversity in very-low-birth-weight infants fed mother's own milk compared with donor breast milk. Am J Clin Nutr. 2019 Apr 1;109(4):1088-1097. doi: 10.1093/ajcn/nqz006. **PMID:** 30982856 ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M5114 - Name: Body Weight - Relevance: HIGH - As Found: Weight - ID: M5006 - Name: Birth Weight - Relevance: HIGH - As Found: Birth Weight ### Condition Browse Module - Meshes - ID: D000001835 - Term: Body Weight - ID: D000001724 - Term: Birth Weight ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Infants Fed Mother's Own Milk **Deaths Num Affected:** 2 **Deaths Num At Risk:** 74 **Description:** Infants fed \>50% mother's own milk with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** EG000 **Other Num Affected:** 26 **Other Num at Risk:** 74 **Serious Number Affected:** 14 **Serious Number At Risk:** 74 **Title:** Infants Fed Mother's Own Milk **Group ID:** EG001 **Title:** Donor Milk Fed Infants **Deaths Num Affected:** 2 **Deaths Num At Risk:** 43 **Description:** Infants fed \<50% mother's own milk (and thus \>50% donor human milk) with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** EG001 **Other Num Affected:** 17 **Other Num at Risk:** 43 **Serious Number Affected:** 20 **Serious Number At Risk:** 43 **Title:** Donor Milk Fed Infants **Frequency Threshold:** 0 #### Other Events **Term:** BPD **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** FiO2 \> 21% for ≥28 d **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** #### Serious Events **Term:** Necrotizing Enterocolitis (NEC)- Stage ≥ IIA **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Overall necrotizing enterocolitis (NEC) Stage ≥ IIA among study infants (all exclusively human milk-fed): 2.4% (1.6% surgical NEC). **Organ System:** Gastrointestinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 74 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 43 **Term:** Spontaneous Intestinal Perforation (SIP) **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 74 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 43 **Term:** Late-onset sepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations ##### Stats **Group ID:** EG000 **Num Affected:** 4 **Num At Risk:** 74 **Group ID:** EG001 **Num Affected:** 6 **Num At Risk:** 43 **Term:** Severe bronchopulmonary dysplasia (BPD) **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Positive pressure or FiO2 \> 30% at 36 wk PMA **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 8 **Num At Risk:** 74 **Group ID:** EG001 **Num Affected:** 11 **Num At Risk:** 43 **Term:** death **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 74 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 43 **Time Frame:** Through study completion (1 year). ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 74 **Group ID:** BG001 **Value:** 43 **Group ID:** BG002 **Value:** 117 **Units:** Participants ### Group **ID:** BG000 **Title:** Infants Fed Mother's Own Milk **Description:** Infants fed \>50% mother's own milk with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. ### Group **ID:** BG001 **Title:** Donor Milk Fed Infants **Description:** Infants fed \<50% mother's own milk (and thus \>50% donor human milk) with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 2.0 **Value:** 28.7 #### Measurement **Group ID:** BG001 **Spread:** 2.5 **Value:** 28.4 #### Measurement **Group ID:** BG002 **Spread:** 2.2 **Value:** 28.6 **Class Title:** Gestational age ### Measure #### Measurement **Group ID:** BG000 **Value:** 35 #### Measurement **Group ID:** BG001 **Value:** 18 #### Measurement **Group ID:** BG002 **Value:** 53 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 39 #### Measurement **Group ID:** BG001 **Value:** 25 #### Measurement **Group ID:** BG002 **Value:** 64 **Category Title:** Male **Class Title:** **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Customized **Unit of Measure:** weeks ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement **PI Sponsor Employee:** True ### Point of Contact **Email:** [email protected] **Organization:** Baylor College of Medicine/Texas Children's Hospital **Phone:** 832-826-1398 **Title:** Dr. Amy B. Hair ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### Outcome Measure 4 ### Outcome Measure 5 ### Outcome Measure 6 ### Outcome Measure 7 ### Outcome Measure 8 ### Outcome Measure 9 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 3.6 - **Upper Limit:** - **Value:** 55.7 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 4.1 - **Upper Limit:** - **Value:** 46.3 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 33.2 - **Upper Limit:** - **Value:** 72.5 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 62.2 - **Upper Limit:** - **Value:** 88.2 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2.2 - **Upper Limit:** - **Value:** 13.6 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1.5 - **Upper Limit:** - **Value:** 12.5 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.23 - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.22 - **Upper Limit:** - **Value:** 1 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.19 - **Upper Limit:** - **Value:** 0.8 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.17 - **Upper Limit:** - **Value:** 0.8 **Title:** #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 4.5 - **Upper Limit:** - **Value:** 12.4 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 7.0 - **Upper Limit:** - **Value:** 13.9 **Title:** #### Outcome Measure 7 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 **Title:** #### Outcome Measure 8 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 **Title:** #### Outcome Measure 9 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 6 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Stool samples during the first 6 weeks of life will be analyzed to compare development of microbial diversity **Dispersion Type:** Standard Error **Parameter Type:** MEAN **Population Description:** Stool samples were selected for microbiota analysis from infants who had samples collected at every time point over the first 6 weeks of life (n=90). **Reporting Status:** POSTED **Time Frame:** 6 weeks **Title:** Intestinal Microbiome Diversity **Type:** PRIMARY **Unit of Measure:** taxa represented ##### Group **Description:** Infants fed \>50% mother's own milk with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** OG000 **Title:** Infants Fed Mother's Own Milk ##### Group **Description:** Infants fed \<50% mother's own milk (and thus \>50% donor human milk) with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** OG001 **Title:** Donor Milk Fed Infants #### Outcome Measure 2 **Description:** Hospital length of stay will be calculated from birth to discharge of infant. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** Birth to discharge **Title:** Hospital Length of Stay **Type:** PRIMARY **Unit of Measure:** days ##### Group **Description:** Infants fed \>50% mother's own milk with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** OG000 **Title:** Infants Fed Mother's Own Milk ##### Group **Description:** Infants fed \<50% mother's own milk (and thus \>50% donor human milk) with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** OG001 **Title:** Donor Milk Fed Infants #### Outcome Measure 3 **Description:** Weight gain will be evaluated weekly throughout the study (defined as g/kg/day) **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 6-10 weeks **Title:** Weight Gain **Type:** PRIMARY **Unit of Measure:** g/kg/day ##### Group **Description:** Infants fed \>50% mother's own milk with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** OG000 **Title:** Infants Fed Mother's Own Milk ##### Group **Description:** Infants fed \<50% mother's own milk (and thus \>50% donor human milk) with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** OG001 **Title:** Donor Milk Fed Infants #### Outcome Measure 4 **Description:** Linear growth will be measured weekly (defined as cm/week) **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 6-10 weeks **Title:** Linear Growth **Type:** PRIMARY **Unit of Measure:** cm/week ##### Group **Description:** Infants fed \>50% mother's own milk with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** OG000 **Title:** Infants Fed Mother's Own Milk ##### Group **Description:** Infants fed \<50% mother's own milk (and thus \>50% donor human milk) with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** OG001 **Title:** Donor Milk Fed Infants #### Outcome Measure 5 **Description:** Growth in head circumference will be measured weekly (defined as cm/wk) **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 6-10 weeks **Title:** Head Circumference Growth **Type:** PRIMARY **Unit of Measure:** cm/week ##### Group **Description:** Infants fed \>50% mother's own milk with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** OG000 **Title:** Infants Fed Mother's Own Milk ##### Group **Description:** Infants fed \<50% mother's own milk (and thus \>50% donor human milk) with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** OG001 **Title:** Donor Milk Fed Infants #### Outcome Measure 6 **Description:** Number of days for infant to reach full enteral feeds. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Infants in both groups **Reporting Status:** POSTED **Time Frame:** 6-10 weeks **Title:** Days to Final Enteral Feed Volume **Type:** SECONDARY **Unit of Measure:** days ##### Group **Description:** Infants fed \>50% mother's own milk with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** OG000 **Title:** Infants Fed Mother's Own Milk ##### Group **Description:** Infants fed \<50% mother's own milk (and thus \>50% donor human milk) with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** OG001 **Title:** Donor Milk Fed Infants #### Outcome Measure 7 **Description:** the number of patients with a diagnosis of NEC (Stage ≥ IIA) will be collected **Parameter Type:** COUNT_OF_PARTICIPANTS **Reporting Status:** POSTED **Time Frame:** 6-10 weeks **Title:** Rates of Necrotizing Enterocolitis (NEC) **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Infants fed \>50% mother's own milk with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** OG000 **Title:** Infants Fed Mother's Own Milk ##### Group **Description:** Infants fed \<50% mother's own milk (and thus \>50% donor human milk) with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** OG001 **Title:** Donor Milk Fed Infants #### Outcome Measure 8 **Description:** the number of patients who develop a spontaneous intestinal perforation will be collected **Parameter Type:** COUNT_OF_PARTICIPANTS **Reporting Status:** POSTED **Time Frame:** 6-10 weeks **Title:** Rates of Spontaneous Intestinal Perforation (SIP) **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Infants fed \>50% mother's own milk with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** OG000 **Title:** Infants Fed Mother's Own Milk ##### Group **Description:** Infants fed \<50% mother's own milk (and thus \>50% donor human milk) with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** OG001 **Title:** Donor Milk Fed Infants #### Outcome Measure 9 **Description:** the number of patients with late-onset sepsis will be collected **Parameter Type:** COUNT_OF_PARTICIPANTS **Reporting Status:** POSTED **Time Frame:** 6-10 weeks **Title:** Rates of Late-onset Sepsis **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Infants fed \>50% mother's own milk with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** OG000 **Title:** Infants Fed Mother's Own Milk ##### Group **Description:** Infants fed \<50% mother's own milk (and thus \>50% donor human milk) with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** OG001 **Title:** Donor Milk Fed Infants ### Participant Flow Module #### Group **Description:** Infants fed \>50% mother's own milk with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** FG000 **Title:** Infants Fed Mother's Own Milk #### Group **Description:** Infants fed \<50% mother's own milk (and thus \>50% donor human milk) with enteral feeding. Observational - no intervention: This study will observe cohorts of infants who are fed primarily either their own mother's milk or donor milk as part of their routine care. No direct intervention is performed as part of the study. **ID:** FG001 **Title:** Donor Milk Fed Infants #### Period **Title:** Overall Study ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 74 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 43 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 74 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 43 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 0 **Pre-Assignment Details:** Of 223 inborn infants \<1500 g BW between September 2015 to August 2016, 33 infants were excluded, 59 could not be enrolled within 72 h, and 6 were not enrolled because their parents declined. Of the 125 enrolled infants, 8 were removed from the study owing to GI defects or complications unknown at time of enrollment that precluded enteral feeding. **Recruitment Details:** Eligible study participants included premature inborn infants at \<1500 g BW with no barriers to enteral milk feeding. Infants were approached and enrolled within the first 72 hr of life. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT05433779 **Acronym:** IMPROVE-2 **Brief Title:** Evaluation of Intravascular Monitoring of Partial PRessure of Oxygen for Neonatal intensiVE Care Patients -2 **Official Title:** Evaluation of Intravascular Monitoring of Partial PRessure of Oxygen for Neonatal intensiVE Care Patients -2 #### Organization Study ID Info **ID:** IMPROVE-2 #### Organization **Class:** INDUSTRY **Full Name:** Neosense Technologies ### Status Module #### Completion Date **Date:** 2023-06-16 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-09-21 **Type:** ACTUAL **Last Update Submit Date:** 2023-09-15 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2023-06-16 **Type:** ACTUAL #### Start Date **Date:** 2022-12-15 **Type:** ACTUAL **Status Verified Date:** 2023-09 #### Study First Post Date **Date:** 2022-06-27 **Type:** ACTUAL **Study First Submit Date:** 2022-06-14 **Study First Submit QC Date:** 2022-06-22 **Why Stopped:** The clinical investigation IMPROVE-2 has been closed after a decision by the Swedish Medical Products Agency. ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Neosense Technologies #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The study is a non-blinded trial with one single intervention in sick newborn infants. The Neosense umbilical catheter will be used instead of the routinely used umbilical catheter on infants where the treating physician has ordered use of an umbilical catheter. The catheter will be connected to the Neosense monitor/measuring unit. Blood samples for blood gas analysis are collected from the patients according to the clinical routine (every 4th to 6th hour). Oxygen tension data from the Neosense measuring system, from the time points when a blood gas sample was collected, will be recorded. This data will be compared to the oxygen tension value from the blood gas sample collected at the same time point. The blood gas samples will be analysed according to clinical and laboratory routine. The infants will remain in the study as long as the Neosense measuring system is used ### Conditions Module **Conditions:** - Catheter - Newborn, Infant, Disease - Lung Diseases **Keywords:** - Neonatal - Umbilical Arteries - Oxygen Partial Pressure ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** DIAGNOSTIC #### Enrollment Info **Count:** 8 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Device: Neosense Umbilical Catheter **Label:** Neosense Umbilical Catheter **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Neosense Umbilical Catheter **Description:** The Neosense umbilical catheter is used instead of the routinely used umbilical catheter **Name:** Neosense Umbilical Catheter **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** The oxygen tension measured by the Neosense system, compared to the oxygen tension measured by a blood gas analyser. **Measure:** Measurement performance (continuously) of an intravascular oxygen sensor in the Neosense system **Time Frame:** Measurement comparisons done at every blood gas sample, i.e. every 4th - 6th hour during the study (up to 8 days) #### Secondary Outcomes **Description:** The number of occasions when the Neosense catheter had to be removed and the underlying reason. This includes all anticipated device effects as well as placing the catheter in the wrong vessel, impossible to measure blood pressure, collect blood samples and/or administer fluids. **Measure:** Clinical complications with the Neosense catheter. **Time Frame:** Time Frame: During study, up to 8 days **Description:** Number of attempts to place the catheter for each patient. **Measure:** Any deviations from normal procedures when inserting an umbilical catheter **Time Frame:** During day 1 **Description:** Timepoint for monitor lock up or battery depletion, in case of occurrence **Measure:** Number of monitor lock up or battery depletions **Time Frame:** During study, in average 4-5 days **Description:** Occurence of anticipated adverse device effects for the monitor. **Measure:** Adverse device effects of the Neosense monitor. **Time Frame:** During study, in average 4-5 days **Description:** Any accessories needed to introduce the Neosense catheter. **Measure:** Any deviations from normal procedures when inserting an umbilical catheter. **Time Frame:** During day 1 **Description:** Any accessories needed to handle the Neosense catheter during insertion. **Measure:** Any deviations from normal procedures when inserting an umbilical catheter. **Time Frame:** During day 1 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: Sick newborn infants where the clinical routine indicates use of an umbilical catheter. At least one of the criteria below should be fulfilled: * The infant needs invasive measurement of blood pressure. * The infant needs repetitive sampling of blood. * The infant needs prolonged infusion(s) \> 2 days. * The infant needs infusion of vessel irritating and potentially vessel harming solutions. * The infant is born extremely preterm (before 28+0 weeks gestational age). * A newborn infant with severe respiratory disorder, requiring oxygen treatment (more than 40% Fraction of inspired oxygen (FiO2)). * The infant is undergoing therapeutic hypothermia following asphyxia ("oxygen deficiency at birth"). * The infant has a severe infection/sepsis. Also: • Signed informed consent form by both parents (or guardians), (if the infant has only one parent (or guardian) one informed consent is required). Exclusion Criteria: * Gastroschisis * Omphalocele * Peritonitis * Necrotizing enterocolitis * Omphalitis * The infant has a severe infection/sepsis * Expected MRI investigation within the 7 first days after birth * The infants birth weight is below 750 g * The infant is born before week 25+0 * Local vascular compromise in lower limbs or buttocks area, or portal venous hypertension **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Danderyd **Country:** Sweden **Facility:** Danderyd Hospital **Location 2:** **City:** Huddinge **Country:** Sweden **Facility:** Karolinska University Hospital **Location 3:** **City:** Solna **Country:** Sweden **Facility:** Karolinska University Hospital **Location 4:** **City:** Stockholm **Country:** Sweden **Facility:** Södersjukhuset **Location 5:** **City:** Uppsala **Country:** Sweden **Facility:** Uppsala University Children's Hospital #### Overall Officials **Official 1:** **Affiliation:** Uppsala University Children's Hospital **Name:** Richard Sindelar, MD PhD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012140 - Term: Respiratory Tract Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M11168 - Name: Lung Diseases - Relevance: HIGH - As Found: Lung Disease - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000008171 - Term: Lung Diseases ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03031379 **Brief Title:** Shortened Preoperative Fasts in ICU **Official Title:** Feasibility of Shortened Preoperative Fasts in Intubated ICU Patients Undergoing Tracheotomy #### Organization Study ID Info **ID:** MMC-12-06-219 #### Organization **Class:** OTHER **Full Name:** Montefiore Medical Center ### Status Module #### Completion Date **Date:** 2014-04 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-04-20 **Type:** ACTUAL **Last Update Submit Date:** 2018-04-18 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2014-04 **Type:** ACTUAL #### Start Date **Date:** 2012-04 **Status Verified Date:** 2012-02 #### Study First Post Date **Date:** 2017-01-25 **Type:** ESTIMATED **Study First Submit Date:** 2017-01-22 **Study First Submit QC Date:** 2017-01-22 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Montefiore Medical Center #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** Randomized controlled trial assessing the safety and benefits of shortening preoperative fasts on intubated ICU patients undergoing tracheotomy. **Detailed Description:** Patients undergoing bedside tracheotomy in an ICU are enrolled and randomized into two groups. Arm A receive a standard fast of at least 6 hours. Arm B receive a shortened fast of only 45 minutes, just before the surgery begins. Patients will be monitored for aspiration, pneumonia and our ability to delivery better nutrition as a result of the intervention. ### Conditions Module **Conditions:** - Intubated ICU Patients ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** TRIPLE **Who Masked:** - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 24 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** standard fast, at least 6 hours prior to tracheotomy **Label:** A - control **Type:** NO_INTERVENTION #### Arm Group 2 **Description:** fast of only 45 minutes prior to incision **Intervention Names:** - Other: shortened fast **Label:** B - shortened fast **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - B - shortened fast **Description:** fast of 45 minutes prior to surgery as opposed to standard 6 hour fast **Name:** shortened fast **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Calories delivered before and after surgery **Measure:** Calories delivered **Time Frame:** 72 hours #### Secondary Outcomes **Description:** intraoperative or postoperative aspiration **Measure:** Aspiration **Time Frame:** 5 days ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * intubated ICU patient undergoing tracheotomy, receiving enteral nutrition, adult, proxy available for consent Exclusion Criteria: * pregnant, \<18yo, cannot tolerate enteral feeding **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Bronx **Country:** United States **Facility:** Montefiore Medical Center **State:** New York **Zip:** 10467 ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04470479 **Brief Title:** Oral Pilocarpine in the Treatment of the Dry Eye of Patients With Sjogrens Syndrome **Official Title:** Oral Pilocarpine in the Treatment of the Dry Eye of Patients With Sjogrens Syndrome #### Organization Study ID Info **ID:** 0910/03 #### Organization **Class:** OTHER **Full Name:** Federal University of São Paulo ### Status Module #### Completion Date **Date:** 2006-04-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-07-14 **Type:** ACTUAL **Last Update Submit Date:** 2020-07-13 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2006-04-01 **Type:** ACTUAL #### Start Date **Date:** 2005-03-01 **Type:** ACTUAL **Status Verified Date:** 2020-07 #### Study First Post Date **Date:** 2020-07-14 **Type:** ACTUAL **Study First Submit Date:** 2020-07-07 **Study First Submit QC Date:** 2020-07-13 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Federal University of São Paulo #### Responsible Party **Investigator Affiliation:** Federal University of São Paulo **Investigator Full Name:** Sergio Felberg **Investigator Title:** Posgraduate Medical Doctor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Is US Export:** True **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this study was to access the possible beneficial effects of oral use of pilocarpine in relieving signs and symptoms of patients with Sjogren's syndrome **Detailed Description:** After being informed about the study, its potential risks and having signed the informed consent form, this placebo-controlled, crossover study involved patients with Sjögren's syndrome to use oral pilocarpine or placebo for ten weeks and after two weeks of medication withdrawal, to invert the treatment for the same period of time. The assessments applied were the Ocular Surface Disease Index, NEI-VFQ-25 questionnaire, non-invasive break-up time, traditional break-up time, evaluation of the cornea and ocular surface with fluorescein and rose bengal dyes, Schirmer test, and tear ferning test. Side effects observed during the treatment period were also assessed. ### Conditions Module **Conditions:** - Dry Eye **Keywords:** - dry eye - Sjogren - pilocarpine hydrochloride ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER **Intervention Model Description:** This was a placebo-controlled, crossover study that involved patients with Sjögren's syndrome to use 20mg/day of oral pilocarpine or placebo for ten weeks and after two weeks of medication withdrawal, to invert the treatment for the same period of time. The assessments applied were the Ocular Surface Disease Index, NEI-VFQ-25 questionnaire, non-invasive break-up time, traditional break-up time, evaluation of the cornea and ocular surface with fluorescein and rose bengal dyes, Schirmer test, tear ferning test, osmolarity and the activity of lysozyme. R ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - INVESTIGATOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 32 **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients with Sjögren's syndrome were allocated to receive oral pilocarpine 20mg per day, (5mg every 6 hours, for ten weeks. **Intervention Names:** - Drug: Pilocarpine Hydrochloride **Label:** Pilocarpine Hydrochloride **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Patients with Sjögren's syndrome were allocated to receive placebo administered in the same way (1 tablet every 6 hours), for ten weeks **Intervention Names:** - Drug: Placebo **Label:** placebo **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Pilocarpine Hydrochloride **Description:** Oral pilocarpine hydrochloride 5mg tablets were administered four times a day for 10 weeks to half the group of selected patients. The other half ingested placebo in the same way. At the end of this period and after two weeks of washing out the medications, the patients had to invert the treatments. **Name:** Pilocarpine Hydrochloride **Other Names:** - Salagen **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - placebo **Description:** Placebo **Name:** Placebo **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** The Ocular Surface Disease Index is a valid and reliable instrument for measuring the severity of dry eye disease, and it possesses the necessary psychometric properties to be used as an end point in clinical trials. The answers to the questions generate an index ranging from 0 to 100, where 0 means no complaints related to dry eye and 100 indicates complaints with maximum intensity. (Change = week 10 score - baseline score) **Measure:** Change from baseline in the Ocular Surface Disease Index questionnaire at week 10 **Time Frame:** Baseline and week 10 **Description:** The Ocular Surface Disease is a valid and reliable instrument for measuring the severity of dry eye disease, and it possesses the necessary psychometric properties to be used as an end point in clinical trials. The answers to the questions generate an index ranging from 0 to 100, where 0 means no complaints related to dry eye and 100 indicates complaints with maximum intensity. (Change = week 22 score - baseline score) **Measure:** Change from baseline Ocular Surface Disease Index questionnaire at week 22 **Time Frame:** Baseline and week 22 **Description:** The VFQ-25 is a reliable and validated 25-item version of the 51-item National Eye Institute Visual Function Questionnaire (NEI-VFQ). It is especially useful in settings such as clinical trials, where interview length is a critical consideration. The set of responses provided scores between 0 and 100, where 0 means the maximum negative impact of the disease on the quality of life of patients and 100 means no negative impact of the disease on quality of life. (Change = week 10 score - baseline score) **Measure:** Change from baseline in the NEI-VFQ-25 questionnaire at week 10 **Time Frame:** Baseline and week 10 **Description:** The VFQ-25 is a reliable and validated 25-item version of the 51-item National Eye Institute Visual Function Questionnaire (NEI-VFQ). It is especially useful in settings such as clinical trials, where interview length is a critical consideration. The set of responses provided scores between 0 and 100, where 0 means the maximum negative impact of the disease on the quality of life of patients and 100 means no negative impact of the disease on quality of life. (Change = week 22 score - baseline score) **Measure:** Change from baseline in the NEI-VFQ-25 questionnaire at week 22 **Time Frame:** Baseline and week 22 **Description:** Tear breakup time is a clinical test used to assess evaporative disease of dry eye. To measure tear breakup time, fluorescein is instilled into the patient's tear film and the patient is asked not to blink while the tear film is viewed under a cobalt blue lighting. The time of appearance of the first tear film break point, recorded in seconds, indicates the tear film breakup time. Times greater than 10 seconds are considered normal. (Change = week 10 measure - baseline measure) **Measure:** Change from baseline in tear breakup time test at week 10 **Time Frame:** Baseline and week 10 **Description:** Tear breakup time is a clinical test used to assess evaporative disease of dry eye. To measure tear breakup time, fluorescein is instilled into the patient's tear film and the patient is asked not to blink while the tear film is viewed under a cobalt blue lighting. The time of appearance of the first tear film break point, recorded in seconds, indicates the tear film breakup time. Times greater than 10 seconds are considered normal. (Change = week 22 measure - baseline measure) **Measure:** Change from baseline in tear breakup time test at week 22 **Time Frame:** Baseline and week 22 **Description:** When instilled in the ocular surface, the rose bengal dye will stain dead cells and spaces not covered by the tear film, being a method used to evaluate the damage to the ocular surface caused by the dry eye. For quantification, the van bijesterveld scale was used, which gives a score from 0 to 9 points according to the intensity of the color distributed onto the ocular surface. The van bijesterveld scale is described as follows: The surface of the eye should be separated into three regions: the nasal bulbar conjunctiva, the cornea and the medial bulbar conjunctiva. Each of these regions may receive a score ranging from 0 to 3 points (0 = not colored, 1 = colored with distant points, 2 = colored with close points and 3 = colored with confluent points). The scores are then added up, generating a global score ranging from 0 to 9 points. Score 0 means no impairment of the ocular surface and score 9 means maximum impairment of the ocular surface). (Change = week 10 - baseline measure) **Measure:** Change from baseline in score with rose bengal staining at week 10 **Time Frame:** Baseline and week 10 **Description:** When instilled in the ocular surface, the rose bengal dye will stain dead cells and spaces not covered by the tear film, being a method used to evaluate the damage to the ocular surface caused by the dry eye. For quantification, the van bijesterveld scale was used, which gives a score from 0 to 9 points according to the intensity of the color distributed onto the ocular surface. The van bijesterveld scale is described as follows: The surface of the eye should be separated into three regions: the nasal bulbar conjunctiva, the cornea and the medial bulbar conjunctiva. Each of these regions may receive a score ranging from 0 to 3 points (0 = not colored, 1 = colored with distant points, 2 = colored with close points and 3 = colored with confluent points). The scores are then added up, generating a global score ranging from 0 to 9 points. Score 0 means no impairment of the ocular surface and score 9 means maximum impairment of the ocular surface). (Change = week 22 - baseline measure) **Measure:** Change from baseline in score with rose bengal staining at week 22 **Time Frame:** Baseline and week 22 **Description:** After dripping fluorescein dye on the ocular surface, the cornea of the patients was analyzed with the patient accommodated in the slit lamp and under cobalt blue light. Fluorescein will stain spaces without cells, which represents spaces in distress caused by dry eyes. The score used ranges from 0 to 3 points (0 = not colored, 1 = blush with distant points, 2 = blush with close points and 3 = blush with confluent points). Score 0 means no corneal involvement (absence of keratitis) and score 3 means maximum involvement of the ocular surface) (Change = week 10 - baseline score) **Measure:** Change from baseline in corneal keratitis score with fluorescein dye at week 10 **Time Frame:** Baseline and week 10 **Description:** After dripping fluorescein dye on the ocular surface, the cornea of the patients was analyzed with the patient accommodated in the slit lamp and under cobalt blue light. Fluorescein will stain spaces without cells, which represents spaces in distress caused by dry eyes. The score used ranges from 0 to 3 points (0 = not colored, 1 = blush with distant points, 2 = blush with close points and 3 = blush with confluent points). Score 0 means no corneal involvement (absence of keratitis) and score 3 means maximum involvement of the ocular surface) (Change = week 22 - baseline score) **Measure:** Change from baseline in corneal keratitis score with fluorescein dye at week 22 **Time Frame:** Baseline and week 22 **Description:** The Schirmer test measures, in millimeters, the production of the tear film for five minutes. A strip of standardized paper is placed on the lateral third of the lower eyelid of both eyes and after 5 minutes. The tear moistens the paper and the reading of the moistening is done. The test can vary from 0 to 35 millimeters, being considered normal (normal tear flow) values greater than 10 millimeters. Values below 10 millimeters indicate dry eye and the lower the value, the more dry the eye. (Change = week 10 - baseline measure) **Measure:** Change from baseline in The Schirmer test at week 10 **Time Frame:** Baseline and week 10 **Description:** The Schirmer test measures, in millimeters, the production of the tear film for five minutes. A strip of standardized paper is placed on the lateral third of the lower eyelid of both eyes and after 5 minutes. The tear moistens the paper and the reading of the moistening is done. The test can vary from 0 to 35 millimeters, being considered normal (normal tear flow) values greater than 10 millimeters. Values below 10 millimeters indicate dry eye and the lower the value, the more dry the eye. (Change = week 22 - baseline measure) **Measure:** Change from baseline in The Schirmer test at week 22 **Time Frame:** Baseline and week 22 **Description:** The tear ferning test is a laboratory test but it has the potential to be applied in the clinic setting to investigate the tear film in a simple way. Drying a small sample of tear fluid onto a clean, glass microscope slide produces a characteristic crystallisation pattern, described as a 'tear ferning". The scale proposed by Rolando was used, which classifies the findings into 4 types (type 1, 2, 3 and 4), according to the teardrop crystallization pattern. Patterns 1 and 2 are considered normal and patterns 3 and 4 are considered abnormal and indicate that the tear has an inadequate composition and quality (Change = week 10 - baseline score) **Measure:** Change from baseline in Tear ferning test (Rolando'score) at week 10 **Time Frame:** Baseline and week 10 **Description:** The tear ferning test is a laboratory test but it has the potential to be applied in the clinic setting to investigate the tear film in a simple way. Drying a small sample of tear fluid onto a clean, glass microscope slide produces a characteristic crystallisation pattern, described as a 'tear ferning". The scale proposed by Rolando was used, which classifies the findings into 4 types (type 1, 2, 3 and 4), according to the teardrop crystallization pattern. Patterns 1 and 2 are considered normal and patterns 3 and 4 are considered abnormal and indicate that the tear has an inadequate composition and quality (Change = week 22 - baseline score) **Measure:** Change from baseline in Tear ferning test (Rolando'score) at week 22 **Time Frame:** Baseline and week 22 #### Secondary Outcomes **Description:** A list of the systemic side effects most frequently described with the oral use of pilocarpine was presented to patients, where they could indicate the presence or absence of this effect: the side effects on the list are: sweating, chills, nausea, salivation, abdominal pain, dizziness, facial flushing, rhinitis, weakness, visual blurring, tremor, tachycardia, headache and diarrhea. **Measure:** The frequency of systemic side effects most frequently reported by patients at week 10 **Time Frame:** week 10 **Description:** A list of the systemic side effects most frequently described with the oral use of pilocarpine was presented to patients, where they could indicate the presence or absence of this effect: the side effects on the list are: sweating, chills, nausea, salivation, abdominal pain, dizziness, facial flushing, rhinitis, weakness, visual blurring, tremor, tachycardia, headache and diarrhea. **Measure:** The frequency of systemic side effects most frequently reported by patients at week 22 **Time Frame:** week 22 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patients with Sjögren's syndrome, both in primary and secondary forms, whose diagnoses were established according to the criteria defined by the - American-European Consensus for the diagnosis of Sjögren's syndrome. * Patients with the secondary form of the syndrome, collagen disease considered controlled by a rheumatologist, before the start of the trial and stable until the end of the study. * Systemic therapy instituted up to two months before the beginning of the protocol. * Literate patients. * Signature of the informed consent form Exclusion Criteria: * Eye or eyelid surface disease not attributed to Sjogren's syndrome. * Temporary or permanent occlusion of tear points. * Use of contact lenses. * Use of systemic medication that is known to influence tear flow. * Need to modify the systemic treatment of the underlying disease during the trial. * Pregnancy or breastfeeding. * Known hypersensitivity reaction to pilocarpine hydrochloride. * Severe cardio-pulmonary disease. **Maximum Age:** 65 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** São Paulo **Country:** Brazil **Facility:** Irmandade Santa casa de Misericórdia de São Paulo **Zip:** 01221010 #### Overall Officials **Official 1:** **Affiliation:** Federal University of São Paulo **Name:** Sergio Felberg, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Access Criteria:** IPD may be requested from the principal investigator upon request by email or letter addressed to registered contacts **Description:** Detailed description of the methods may be made available after publication **Info Types:** - STUDY_PROTOCOL - ICF **IPD Sharing:** YES **Time Frame:** 6 months after publication, for 3 months ### References Module #### References **Citation:** Kawakita T, Shimmura S, Tsubota K. Effect of Oral Pilocarpine in Treating Severe Dry Eye in Patients With Sjogren Syndrome. Asia Pac J Ophthalmol (Phila). 2015 Mar-Apr;4(2):101-5. doi: 10.1097/APO.0000000000000040. **PMID:** 26065354 **Citation:** Cifuentes M, Del Barrio-Diaz P, Vera-Kellet C. Pilocarpine and artificial saliva for the treatment of xerostomia and xerophthalmia in Sjogren syndrome: a double-blind randomized controlled trial. Br J Dermatol. 2018 Nov;179(5):1056-1061. doi: 10.1111/bjd.16442. Epub 2018 May 29. **PMID:** 29432648 **Citation:** Papas AS, Sherrer YS, Charney M, Golden HE, Medsger TA Jr, Walsh BT, Trivedi M, Goldlust B, Gallagher SC. Successful Treatment of Dry Mouth and Dry Eye Symptoms in Sjogren's Syndrome Patients With Oral Pilocarpine: A Randomized, Placebo-Controlled, Dose-Adjustment Study. J Clin Rheumatol. 2004 Aug;10(4):169-77. doi: 10.1097/01.rhu.0000135553.08057.21. **PMID:** 17043506 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007766 - Term: Lacrimal Apparatus Diseases - ID: D000005128 - Term: Eye Diseases - ID: D000007637 - Term: Keratoconjunctivitis - ID: D000003231 - Term: Conjunctivitis - ID: D000003229 - Term: Conjunctival Diseases - ID: D000007634 - Term: Keratitis - ID: D000003316 - Term: Corneal Diseases - ID: D000001172 - Term: Arthritis, Rheumatoid - ID: D000001168 - Term: Arthritis - ID: D000007592 - Term: Joint Diseases - ID: D000009140 - Term: Musculoskeletal Diseases - ID: D000012216 - Term: Rheumatic Diseases - ID: D000014987 - Term: Xerostomia - ID: D000012466 - Term: Salivary Gland Diseases - ID: D000009059 - Term: Mouth Diseases - ID: D000009057 - Term: Stomatognathic Diseases - ID: D000003240 - Term: Connective Tissue Diseases - ID: D000001327 - Term: Autoimmune Diseases - ID: D000007154 - Term: Immune System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: BC07 - Name: Mouth and Tooth Diseases - Abbrev: BC11 - Name: Eye Diseases - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: BC20 - Name: Immune System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M15664 - Name: Sjogren's Syndrome - Relevance: HIGH - As Found: Sjogren's Syndrome - ID: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M18040 - Name: Dry Eye Syndromes - Relevance: HIGH - As Found: Dry Eye - ID: M10664 - Name: Keratoconjunctivitis Sicca - Relevance: HIGH - As Found: Dry Eye - ID: M10663 - Name: Keratoconjunctivitis - Relevance: LOW - As Found: Unknown - ID: M10786 - Name: Lacrimal Apparatus Diseases - Relevance: LOW - As Found: Unknown - ID: M8271 - Name: Eye Diseases - Relevance: LOW - As Found: Unknown - ID: M6455 - Name: Conjunctivitis - Relevance: LOW - As Found: Unknown - ID: M6453 - Name: Conjunctival Diseases - Relevance: LOW - As Found: Unknown - ID: M10660 - Name: Keratitis - Relevance: LOW - As Found: Unknown - ID: M6539 - Name: Corneal Diseases - Relevance: LOW - As Found: Unknown - ID: M4476 - Name: Arthritis - Relevance: LOW - As Found: Unknown - ID: M4480 - Name: Arthritis, Rheumatoid - Relevance: LOW - As Found: Unknown - ID: M10621 - Name: Joint Diseases - Relevance: LOW - As Found: Unknown - ID: M12097 - Name: Musculoskeletal Diseases - Relevance: LOW - As Found: Unknown - ID: M15045 - Name: Rheumatic Diseases - Relevance: LOW - As Found: Unknown - ID: M6323 - Name: Collagen Diseases - Relevance: LOW - As Found: Unknown - ID: M17724 - Name: Xerostomia - Relevance: LOW - As Found: Unknown - ID: M15285 - Name: Salivary Gland Diseases - Relevance: LOW - As Found: Unknown - ID: M12019 - Name: Mouth Diseases - Relevance: LOW - As Found: Unknown - ID: M12017 - Name: Stomatognathic Diseases - Relevance: LOW - As Found: Unknown - ID: M6464 - Name: Connective Tissue Diseases - Relevance: LOW - As Found: Unknown - ID: M4629 - Name: Autoimmune Diseases - Relevance: LOW - As Found: Unknown - ID: M10200 - Name: Immune System Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000012859 - Term: Sjogren's Syndrome - ID: D000015352 - Term: Dry Eye Syndromes - ID: D000007638 - Term: Keratoconjunctivitis Sicca ### Intervention Browse Module - Ancestors - ID: D000008916 - Term: Miotics - ID: D000001337 - Term: Autonomic Agents - ID: D000018373 - Term: Peripheral Nervous System Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018721 - Term: Muscarinic Agonists - ID: D000018679 - Term: Cholinergic Agonists - ID: D000018678 - Term: Cholinergic Agents - ID: D000018377 - Term: Neurotransmitter Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M13757 - Name: Pilocarpine - Relevance: HIGH - As Found: Motivational interview - ID: M20795 - Name: Muscarinic Agonists - Relevance: LOW - As Found: Unknown - ID: M20758 - Name: Cholinergic Agents - Relevance: LOW - As Found: Unknown - ID: M20504 - Name: Neurotransmitter Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000010862 - Term: Pilocarpine ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02266979 **Brief Title:** Study to Determine Appropriate Nurse: Patient Ratios in Peritoneal Dialysis Programs **Official Title:** Study to Determine Appropriate Nurse: Patient Ratios in Peritoneal Dialysis Programs #### Organization Study ID Info **ID:** AEFIIRPDstaff2014 #### Organization **Class:** OTHER **Full Name:** Pontificia Universidade Católica do Rio Grande do Sul ### Status Module #### Completion Date **Date:** 2016-09 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2016-11-15 **Type:** ESTIMATED **Last Update Submit Date:** 2016-11-13 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2016-09 **Type:** ACTUAL #### Start Date **Date:** 2014-06 **Status Verified Date:** 2016-11 #### Study First Post Date **Date:** 2014-10-17 **Type:** ESTIMATED **Study First Submit Date:** 2014-09-28 **Study First Submit QC Date:** 2014-10-13 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Ana Elizabeth Prado Lima Figueiredo #### Responsible Party **Investigator Affiliation:** Pontificia Universidade Católica do Rio Grande do Sul **Investigator Full Name:** Ana Elizabeth Prado Lima Figueiredo **Investigator Title:** Professor **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** As the resolution of the Brazilian Federal Council of Nursing (COFEN) sizing the framework of nursing professionals is essential to ensure the safety and quality of patient care. To achieve such goals actions are needed to evaluate the variety of tasks and performance specific to each area in nursing profession. The National Agency for Health Surveillance (ANVISA), through Board Resolution (RDC-154) determines that the nurse to patient ratio is 1 to 30 in hemodialysis and 1 to 50 on peritoneal dialysis, however these targets are not based on any evidence, and same apply to other Latin-American countries. Objective: To determine the appropriate nurse: patient ratio for peritoneal dialysis unit. Method: This research will be developed in two phases. The first with an observational design with descriptive and qualitative approach. There will be a focus group with experts in the field, to defined the activities and responsibilities of the peritoneal dialysis nurse, and produce a tool with a list of activities. A time-orientated score will be created. In the second stage a cross-sectional quantitative approach will be used. To apply the instrument from by direct observation of activities performed by nurses during their workday. With the instrument activities will be timed and the overall time of each nursing activity will be determined by multiplying the time spent by the total number of patients. It is expected tat this strategy will increase time on treatment and/or decrease dropout rates, by developing an easy test/ formula application to determine the correct ratio according to the peritoneal dialysis program. This tool will provide better outcomes for patients as well as allow for growth of peritoneal dialysis programs with qualified assistance and patient safety. **Detailed Description:** Research question: Can a time-oriented score system and expert consensus be applied to PD to develop an easy and repeatable system to evaluate work load for peritoneal dialysis nurses to achieve the best outcomes and allow for planned growth of the unit? ### Conditions Module **Conditions:** - Chronic Renal Failure **Keywords:** - peritoneal dialysis - nursing staff - nursing activities - Personnel Downsizing ### Design Module #### Design Info **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 10 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Outcomes Module #### Primary Outcomes **Description:** after focal groups a list we all activities performed by PD nurses will be prepared **Measure:** number of activities ( direct or indirect care) performed by peritoneal dialysis nurse **Time Frame:** 6 months #### Secondary Outcomes **Description:** the time spend to perform each activities will be measured to calculate time spent with patients **Measure:** time to perform activities **Time Frame:** 6 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * To have at least one year of experience with peritoneal dialysis Exclusion Criteria: * Not applicable * Consent withdraw **Healthy Volunteers:** True **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT **Study Population:** Renal nurses, master and PhD nurse experts in peritoneal dialysis to develop a list of activities to be timed ### Contacts Locations Module #### Locations **Location 1:** **City:** Porto Alegre **Country:** Brazil **Facility:** Pontificia Universidade Católica Rio Grande do Sul **State:** Rio Grande do Sul **Zip:** 90.619-900 #### Overall Officials **Official 1:** **Affiliation:** Pontificia Universidade Católica do Rio Grande do Sul **Name:** Ana E Figueiredo, RN, PhD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000051437 - Term: Renal Insufficiency - ID: D000007674 - Term: Kidney Diseases - ID: D000014570 - Term: Urologic Diseases - ID: D000052776 - Term: Female Urogenital Diseases - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications - ID: D000091642 - Term: Urogenital Diseases - ID: D000052801 - Term: Male Urogenital Diseases - ID: D000051436 - Term: Renal Insufficiency, Chronic - ID: D000002908 - Term: Chronic Disease - ID: D000020969 - Term: Disease Attributes - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M10699 - Name: Kidney Failure, Chronic - Relevance: HIGH - As Found: Chronic Renal Failure - ID: M26718 - Name: Renal Insufficiency - Relevance: LOW - As Found: Unknown - ID: M10698 - Name: Kidney Diseases - Relevance: LOW - As Found: Unknown - ID: M17319 - Name: Urologic Diseases - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M27095 - Name: Male Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M26717 - Name: Renal Insufficiency, Chronic - Relevance: LOW - As Found: Unknown - ID: M6147 - Name: Chronic Disease - Relevance: LOW - As Found: Unknown - ID: M22700 - Name: Disease Attributes - Relevance: LOW - As Found: Unknown - ID: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000007676 - Term: Kidney Failure, Chronic ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06248879 **Brief Title:** Intermittent Oro-esophageal Tube Feeding in Late Dysphagia for Nasopharyngeal Carcinoma **Official Title:** A Randomly Controlled Study to Explore the Effect Intermittent Oro-esophageal Tube Feeding in Late Dysphagia for Nasopharyngeal Carcinoma #### Organization Study ID Info **ID:** IOE-Biyanai-Xin #### Organization **Class:** OTHER **Full Name:** People's Hospital of Zhengzhou University ### Status Module #### Completion Date **Date:** 2024-04 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-03-06 **Type:** ACTUAL **Last Update Submit Date:** 2024-03-02 **Overall Status:** WITHDRAWN #### Primary Completion Date **Date:** 2024-04 **Type:** ESTIMATED #### Start Date **Date:** 2024-02-28 **Type:** ACTUAL **Status Verified Date:** 2024-03 #### Study First Post Date **Date:** 2024-02-08 **Type:** ACTUAL **Study First Submit Date:** 2024-01-31 **Study First Submit QC Date:** 2024-01-31 **Why Stopped:** ethical issue ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** People's Hospital of Zhengzhou University #### Responsible Party **Investigator Affiliation:** People's Hospital of Zhengzhou University **Investigator Full Name:** Zeng Changhao **Investigator Title:** Principle Investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** This is a prospective multicenter study with patients with delayed dysphagia after radiotherapy. Patients enrolled are randomly divided equally into the observation group and the control group. All patients receive conventional care, and the observation group received Intermittent Oro-esophageal Tube Feeding while the control group received Nasogastric Tube Feeding for enteral nutrition support. Baseline information (demographics, medical history, etc.), nutritional status at admission and after treatment, depression, dysphagia, and quality of life after treatment as well as adverse events are compared. **Detailed Description:** Palliation to delayed dysphagia after radiotherapy for nasopharyngeal carcinoma continues to be a challenge. This is a prospective multicenter study with patients with delayed dysphagia after radiotherapy. Patients enrolled are randomly divided equally into the observation group and the control group. All patients receive conventional care, and the observation group received Intermittent Oro-esophageal Tube Feeding while the control group received Nasogastric Tube Feeding for enteral nutrition support. Baseline information (demographics, medical history, etc.), nutritional status at admission and after treatment, depression, dysphagia, and quality of life after treatment as well as adverse events are compared. ### Conditions Module **Conditions:** - Dysphagia ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** During the 15-day treatment, both groups of patients are hospitalized, while conventional care and enteral nutrition support are provided to the two groups. Specifically, conventional care includes health education, dietary adjustments, nasopharyngeal hygiene, management of risk factors (blood pressure and lipid control, etc.), exercise rehabilitation, and psychological support. The frequency and content of these interventions are arranged based on the patients; health condition. The observation group receives Intermittent Oro-esophageal Tube Feeding for enteral nutrition support **Intervention Names:** - Device: Intermittent Oral-esophageal Tube Feeding - Behavioral: comprehensive rehabilitation therapy **Label:** The observation group **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** During the 15-day treatment, both groups of patients are hospitalized, while conventional care and enteral nutrition support are provided to the two groups. Specifically, conventional care includes health education, dietary adjustments, nasopharyngeal hygiene, management of risk factors (blood pressure and lipid control, etc.), exercise rehabilitation, and psychological support. The frequency and content of these interventions are arranged based on the patients; health condition.The control group receives nasogastric tube for enteral nutrition support **Intervention Names:** - Device: Nasogastric Tube Feeding - Behavioral: comprehensive rehabilitation therapy **Label:** The control group **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - The observation group **Description:** The specific procedure was as follows: the infant was placed in a semi-recumbent or sitting position with the head fixed. Before each feeding, the infant's oral and nasal secretions were to be cleared. An intermittent oro-esophageal tube was appropriately lubricated with water on the head part. The professional medical staff held the tube and slowly inserted it through one side of the mouth into the upper part of the esophagus. The depth of insertion depended on the patient's age and height. After each feeding, the tube was immediately removed, and the patient was held upright for at least 30 minutes in case of reflux. **Name:** Intermittent Oral-esophageal Tube Feeding **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - The control group **Description:** Nasogastric Tube Feeding were used for feeding to provide nutritional support. Each feeding was administered by a nurse using the infant's mother's breast milk through the tube. The amount of each feeding varied from 20 to 100 ml depending on the age of the infant, with feedings given every 2 to 3 hours, approximately 10 times per day. The duration of each feeding procedure ranged from 10 to 20 minutes. The total daily intake ranged from 200 to 1000 ml. Each tube was kept indwelling for 5 to 7 days. When the tube needed to be replaced, it was removed after the last feeding of a day and a new tube was to be inserted through the other nostril on the following morning to continue the nutritional support. **Name:** Nasogastric Tube Feeding **Type:** DEVICE #### Intervention 3 **Arm Group Labels:** - The control group - The observation group **Description:** Both groups were given comprehensive rehabilitation therapy. The main intervention measures included: 1) non-invasive ventilator treatment, generally at least once every night and typically not exceeding continuous daily usage.; 2) attention to feeding and sleeping positions, with a recommended sleeping position of lateral recumbent and the head of the bed raised by 20-30°; 3) swallowing function training, such as tongue muscle stretching training, assisted anterior jaw protrusion training, lemon ice stimulation to the soft palate, pharyngeal wall, etc., generally 5 days per week, twice per day, 5-20 minutes each time; 4) pulmonary ultrashort wave therapy, generally at least 2-3 times a week, and not more than once a day; 5) physical therapy, such as intensive training for gross motor functions including lifting the head, turning over, sitting, crawling, standing, etc., generally 3-5 days per week, 1-2 times per day, 5-20 min each time. **Name:** comprehensive rehabilitation therapy **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** Hemoglobin was recorded through the blood routine test. (Hb, g/L) **Measure:** concentration of Hemoglobin **Time Frame:** day 1 and day 15 **Description:** Serum albumin was recorded through the blood routine test. (ALB, g/L) **Measure:** concentration of Serum albumin **Time Frame:** day 1 and day 15 **Description:** Serum prealbumin was recorded through the blood routine test.(PA, g/L) **Measure:** concentration of Serum prealbumin **Time Frame:** day 1 and day 15 **Description:** Body Mass Index was assessed with the combination of body weight and height: weight (kg)/ \[height (m)\] \^2 **Measure:** Body Mass Index **Time Frame:** day 1 and day 15 #### Secondary Outcomes **Description:** The PATIENT HEALTH QUESTIONNAIRE scale is utilized to evaluate the depression of patients at admission and after treatment. Options representing varying degrees of severity, ranging from 0 to 3 were provided in each aspect. The total scores between 0 and 4 were classified as negative, indicating the absence of depressive symptoms, while scores above 4 were classified as positive, indicating the presence of potential depressive symptoms. **Measure:** Depression **Time Frame:** day 1 and day 15 **Description:** The Functional Oral Intake Scale (FOIS) was used to evaluate function of oral intake. In the FOIS assessment, two professional rehabilitation therapists communicated with the patients, observed and recorded to assess their swallowing function. The assessment scale consists of seven levels, with a higher level indicating progressively better swallowing function (less dysphagia). **Measure:** Functional Oral Intake Scale **Time Frame:** day 1 and day 15 **Description:** In this study, the Penetration-Aspiration Scale (PAS) was recruited, which was a commonly used to evaluate the occurrence of penetration or aspiration during swallowing. It categorizes dysphagia into eight levels, with Level 0 indicating no penetration or aspiration and Level 8 indicating severe aspiration. A higher level indicates more severe dysphagia. **Measure:** Penetration-Aspiration Scale **Time Frame:** day 1 and day 15 **Description:** The Chinese version of the Swallowing-Quality of Life questionnaire (SWAL-QOL) was used to assess the quality of life of patients.he Likert scale ranging from 1 to 5 was utilized for scoring, with a total of 44 items, including difficulties in swallowing, dietary restrictions, oral health, social communication, and others. The total score was converted to a standard percentage scale ranging from 0 to 100, with positively correlated with quality of life. **Measure:** Swallowing-Quality of Life questionnaire **Time Frame:** day 1 and day 15 **Description:** The total amount of nutrients consumed by the patient on the day was recorded, excluding fresh water, units: milliliters **Measure:** Feeding amount **Time Frame:** day 1 and day 15 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Age between 18 and 65 years. * With the history of Nasopharyngeal Carcinoma and radiation therapy. * With dysphagia occurred at least three years after radiotherapy (confirmed by videofluoroscopic swallowing study), in need of and feasible for enteral nutrition support. * Conscious and with stable vital signs; * Willing to participate and sign the written informed consent form either personally or by a family member. Exclusion Criteria: * Presence of other diseases that might cause dysphagia. * With distant metastasis of tumors, or complicated with severe systemic disorders or malignancies. * Concurrent participation in other treatments that could interfere with the trial. * Inability to cooperate with treatment due to aphasia, mental health issues, etc. * Received tube feeding for enteral nutrition support within the past three years. **Maximum Age:** 65 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Zhengzhou **Country:** China **Facility:** Affiliated Hospital of Zhengzhou University **State:** Henan **Zip:** 450001 #### Overall Officials **Official 1:** **Affiliation:** Site Coordinator of United Medical Group located in Miami **Name:** Nieto Luis, Master **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000002277 - Term: Carcinoma - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000009369 - Term: Neoplasms - ID: D000009303 - Term: Nasopharyngeal Neoplasms - ID: D000010610 - Term: Pharyngeal Neoplasms - ID: D000010039 - Term: Otorhinolaryngologic Neoplasms - ID: D000006258 - Term: Head and Neck Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000009302 - Term: Nasopharyngeal Diseases - ID: D000010608 - Term: Pharyngeal Diseases - ID: D000009057 - Term: Stomatognathic Diseases - ID: D000010038 - Term: Otorhinolaryngologic Diseases - ID: D000004935 - Term: Esophageal Diseases - ID: D000005767 - Term: Gastrointestinal Diseases - ID: D000004066 - Term: Digestive System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: All - Name: All Conditions - Abbrev: BC07 - Name: Mouth and Tooth Diseases - Abbrev: BC09 - Name: Ear, Nose, and Throat Diseases - Abbrev: BC06 - Name: Digestive System Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M5534 - Name: Carcinoma - Relevance: LOW - As Found: Unknown - ID: M1730 - Name: Nasopharyngeal Carcinoma - Relevance: HIGH - As Found: Nasopharyngeal Carcinoma - ID: M6882 - Name: Deglutition Disorders - Relevance: HIGH - As Found: Dysphagia - ID: M12320 - Name: Neoplasms, Glandular and Epithelial - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: M12254 - Name: Nasopharyngeal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M13517 - Name: Pharyngeal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M12962 - Name: Otorhinolaryngologic Neoplasms - Relevance: LOW - As Found: Unknown - ID: M9348 - Name: Head and Neck Neoplasms - Relevance: LOW - As Found: Unknown - ID: M12253 - Name: Nasopharyngeal Diseases - Relevance: LOW - As Found: Unknown - ID: M13515 - Name: Pharyngeal Diseases - Relevance: LOW - As Found: Unknown - ID: M12017 - Name: Stomatognathic Diseases - Relevance: LOW - As Found: Unknown - ID: M12961 - Name: Otorhinolaryngologic Diseases - Relevance: LOW - As Found: Unknown - ID: M8085 - Name: Esophageal Diseases - Relevance: LOW - As Found: Unknown - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown - ID: T4047 - Name: Nasopharyngeal Carcinoma - Relevance: HIGH - As Found: Nasopharyngeal Carcinoma ### Condition Browse Module - Meshes - ID: D000077274 - Term: Nasopharyngeal Carcinoma - ID: D000003680 - Term: Deglutition Disorders ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01609179 **Brief Title:** IPI-926 Extension Protocol for Continuation of Treatment With IPI-926 **Official Title:** IPI-926 Extension Protocol for Continuation of IPI-926 Treatment in Patients Experiencing Clinical Benefit While Enrolled in an IPI-926 Protocol #### Organization Study ID Info **ID:** IPI-926-09 #### Organization **Class:** INDUSTRY **Full Name:** Infinity Pharmaceuticals, Inc. ### Status Module #### Completion Date **Date:** 2012-11 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2012-11-14 **Type:** ESTIMATED **Last Update Submit Date:** 2012-11-13 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2012-10 **Type:** ACTUAL #### Start Date **Date:** 2012-03 **Status Verified Date:** 2012-11 #### Study First Post Date **Date:** 2012-05-31 **Type:** ESTIMATED **Study First Submit Date:** 2012-05-29 **Study First Submit QC Date:** 2012-05-29 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Infinity Pharmaceuticals, Inc. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** A treatment protocol that enables patients to have continued access to IPI-926. **Detailed Description:** The extension protocol is a continuation of treatment with IPI-926, as administered to each individual patient during participation in their original IPI-926 protocol. Patients who are completing their participation in the original IPI-926 protocol in which they enrolled, as defined in the original protocol, and, have stable disease or confirmed complete or partial response as defined by the original protocol may continue to receive treatment with IPI-926 in the extension protocol. ### Conditions Module **Conditions:** - Basal Cell Carcinoma - Chondrosarcoma ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 9 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Drug: IPI-926 **Label:** IPI-926 **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - IPI-926 **Description:** IPI-926 is administered orally as a capsule formulation, as a fixed dose in mg/day. Patients will be administered the same dose with same cycle length of IPI-926 and combination therapy that they were receiving in the original protocol at the time of transition into the extension study. The following are potential once-daily doses that patients are receiving in original protocols: 60 mg, 90 mg, 100 mg, 110 mg, 130 mg, or 160 mg. **Name:** IPI-926 **Other Names:** - saridegib **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Monitoring of AEs, including SAEs as well as review of laboratory sample test data to assess safety and tolerability of IPI-926 **Measure:** Incidence of adverse events (AEs) and serious adverse events (SAEs), and laboratory test results **Time Frame:** Up to 30 days after the last patient study visit ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Voluntarily sign the informed consent form 2. Currently receiving IPI-926 while participating in an Infinity-sponsored IPI-926 study. Note: For blinded studies, patient's treatment assignment must be unblinded according to the instructions in the original protocol to confirm they are receiving IPI-926. 3. Eastern Cooperative Oncology Group (ECOG) performance status: 0 or 1. 4. Documented response or stable disease, as defined in the original protocol, at the time of entry to the extension study. 5. Willingness and ability to continue IPI-926 dispensation and follow-up procedures at the current investigational site. 6. Willingness and ability to comply with scheduled visits, treatment plans, and laboratory tests and other study procedures. 7. Women of child-bearing potential (WCBP), defined as a sexually mature woman who has not undergone a hysterectomy or tubal ligation or who has not been naturally postmenopausal for at least 24 consecutive months, must have a negative serum or urine pregnancy test prior to treatment. All WCBP, all sexually active male patients, and all partners of patients must agree to use adequate methods of birth control throughout the study. Exclusion Criteria: 1. Discontinued IPI-926 or withdrew informed consent to participate in original Infinity-sponsored IPI-926 study. 2. Require addition of or change to a new concomitant therapy to adequately treat the malignancy under study. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Aurora **Country:** United States **Facility:** University of Colorado Denver **State:** Colorado **Zip:** 80045 **Location 2:** **City:** Baltimore **Country:** United States **Facility:** Johns Hopkins **State:** Maryland **Zip:** 21205 #### Overall Officials **Official 1:** **Affiliation:** Infinity Pharmaceuticals, Inc. **Name:** Tess Schmalbach, MD **Role:** STUDY_DIRECTOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000002277 - Term: Carcinoma - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000009369 - Term: Neoplasms - ID: D000018295 - Term: Neoplasms, Basal Cell - ID: D000009372 - Term: Neoplasms, Connective Tissue - ID: D000018204 - Term: Neoplasms, Connective and Soft Tissue - ID: D000012509 - Term: Sarcoma ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: All - Name: All Conditions - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M5534 - Name: Carcinoma - Relevance: LOW - As Found: Unknown - ID: M5537 - Name: Carcinoma, Basal Cell - Relevance: HIGH - As Found: Basal Cell Carcinoma - ID: M6053 - Name: Chondrosarcoma - Relevance: HIGH - As Found: Chondrosarcoma - ID: M12320 - Name: Neoplasms, Glandular and Epithelial - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: M20439 - Name: Neoplasms, Basal Cell - Relevance: LOW - As Found: Unknown - ID: M12317 - Name: Neoplasms, Connective Tissue - Relevance: LOW - As Found: Unknown - ID: M20350 - Name: Neoplasms, Connective and Soft Tissue - Relevance: LOW - As Found: Unknown - ID: M15327 - Name: Sarcoma - Relevance: LOW - As Found: Unknown - ID: T1172 - Name: Chondrosarcoma - Relevance: HIGH - As Found: Chondrosarcoma - ID: T5284 - Name: Soft Tissue Sarcoma - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000002280 - Term: Carcinoma, Basal Cell - ID: D000002813 - Term: Chondrosarcoma ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02625779 **Brief Title:** Combined Cytidine and Creatine-containing Drug in the Treatment of the Bipolar Depression **Official Title:** A Double-Blind, Placebo-Controlled Trial of Combined Cytidine- and Creatine-containing Drug and Dietary Supplement in the Treatment of the Bipolar Depression #### Organization Study ID Info **ID:** NARSAD_Bipolar #### Organization **Class:** OTHER **Full Name:** Ewha Womans University Mokdong Hospital ### Status Module #### Completion Date **Date:** 2017-04-30 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2018-02-09 **Type:** ACTUAL **Last Update Submit Date:** 2018-02-08 **Overall Status:** WITHDRAWN #### Primary Completion Date **Date:** 2017-04-30 **Type:** ESTIMATED #### Start Date **Date:** 2016-03-01 **Type:** ESTIMATED **Status Verified Date:** 2018-02 #### Study First Post Date **Date:** 2015-12-09 **Type:** ESTIMATED **Study First Submit Date:** 2015-12-07 **Study First Submit QC Date:** 2015-12-07 **Why Stopped:** The research project has been cancelled before any participants were enrolled. ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Ewha Womans University Mokdong Hospital #### Responsible Party **Investigator Affiliation:** Ewha Womans University Mokdong Hospital **Investigator Full Name:** In Kyoon Lyoo **Investigator Title:** Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** This research is aimed to investigate the efficacy and safety of the creatine and cytidine augmentation in treating bipolar depression and to evaluate changes in relevant brain biochemical metabolism using proton and phosphorous magnetic resonance spectroscopy. ### Conditions Module **Conditions:** - Depression, Bipolar **Keywords:** - Bipolar depression - Cytidine - Creatine - Magnetic resonance spectroscopy ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** QUADRUPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Valproate: 300mg/day for 8 weeks Placebo: for 8 weeks **Intervention Names:** - Drug: Valproate and Placebo **Label:** Valproate and Placebo **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Valproate: 300mg/day for 8 weeks Cytidine-containing Drug: 2g/day for 8 weeks **Intervention Names:** - Drug: Valproate and Cytidine-containing Drug **Label:** Valproate and Cytidine-containing Drug **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Valproate: 300mg/day for 8 weeks Creatine-containing Drug: 3g/day for the first week 5g/day for week 2-8 **Intervention Names:** - Drug: Valproate and Creatine-containing Drug **Label:** Valproate and Creatine-containing Drug **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Valproate and Placebo **Name:** Valproate and Placebo **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Valproate and Cytidine-containing Drug **Name:** Valproate and Cytidine-containing Drug **Type:** DRUG #### Intervention 3 **Arm Group Labels:** - Valproate and Creatine-containing Drug **Name:** Valproate and Creatine-containing Drug **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** assessed with structured clinical interview **Measure:** Change in symptoms of bipolar depression **Time Frame:** Baseline and 8 weeks **Description:** assessed with structured clinical interview **Measure:** Change in depressive symptoms **Time Frame:** Baseline and 8 weeks #### Secondary Outcomes **Description:** assessed with proton magnetic resonance spectroscopy **Measure:** Change in brain Glx (glutamate+glutamine) level **Time Frame:** Baseline and 8 weeks **Description:** assessed with phosphorous magnetic resonance spectroscopy **Measure:** Change in brain phosphocreatine level **Time Frame:** Baseline and 8 weeks **Measure:** Number of participants with adverse events **Time Frame:** Baseline through 8 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Aged 19-65 years * Bipolar disorder I or II (DSM-IV-TR) with current depressive episode * Informed consent Exclusion Criteria: * Use of medication for bipolar depression or other psychotropic drugs * Current Axis I mental disorders other than bipolar depression based on structured clinical interview * Current borderline or antisocial personality disorder based on structured clinical interview * Major medical or neurological illnesses (epilepsy, multiple sclerosis, brain tumor, cerebrovascular disease, etc) * Hypersensitivity to divalproate or valpromide * Diagnosis of porphyria * Current or past liver diseases * Severe dysfunction in liver or pancreas * Use of mefloquine * Alcohol or substance abuse/dependence * Intelligence quotient of 80 or below * Contraindications to magnetic resonance imaging * Pregnancy or breastfeeding * Allergy or intolerance to the study drugs **Maximum Age:** 65 Years **Minimum Age:** 19 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Seoul **Country:** Korea, Republic of **Facility:** Ewha Womans University Mokdong Hospital ## Derived Section ### Condition Browse Module - Ancestors - ID: D000001526 - Term: Behavioral Symptoms - ID: D000019964 - Term: Mood Disorders - ID: D000001523 - Term: Mental Disorders - ID: D000068105 - Term: Bipolar and Related Disorders ### Condition Browse Module - Browse Branches - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M7058 - Name: Depression - Relevance: HIGH - As Found: Depression - ID: M7061 - Name: Depressive Disorder - Relevance: HIGH - As Found: Depression - ID: M4996 - Name: Bipolar Disorder - Relevance: HIGH - As Found: Bipolar Depression - ID: M4818 - Name: Behavioral Symptoms - Relevance: LOW - As Found: Unknown - ID: M21835 - Name: Mood Disorders - Relevance: LOW - As Found: Unknown - ID: M4815 - Name: Mental Disorders - Relevance: LOW - As Found: Unknown - ID: M14473 - Name: Psychotic Disorders - Relevance: LOW - As Found: Unknown - ID: M226 - Name: Bipolar and Related Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000003863 - Term: Depression - ID: D000003866 - Term: Depressive Disorder - ID: D000001714 - Term: Bipolar Disorder ### Intervention Browse Module - Ancestors - ID: D000000927 - Term: Anticonvulsants - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000018682 - Term: GABA Agents - ID: D000018377 - Term: Neurotransmitter Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018692 - Term: Antimanic Agents - ID: D000014149 - Term: Tranquilizing Agents - ID: D000002492 - Term: Central Nervous System Depressants - ID: D000011619 - Term: Psychotropic Drugs ### Intervention Browse Module - Browse Branches - Abbrev: AntiConv - Name: Anticonvulsants - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: PsychDr - Name: Psychotropic Drugs - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Ot - Name: Other Dietary Supplements ### Intervention Browse Module - Browse Leaves - ID: M17383 - Name: Valproic Acid - Relevance: HIGH - As Found: Immediate release - ID: M4246 - Name: Anticonvulsants - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown - ID: M20504 - Name: Neurotransmitter Agents - Relevance: LOW - As Found: Unknown - ID: M14474 - Name: Psychotropic Drugs - Relevance: LOW - As Found: Unknown - ID: T385 - Name: Creatine - Relevance: HIGH - As Found: Elbow ### Intervention Browse Module - Meshes - ID: D000014635 - Term: Valproic Acid ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03087279 **Acronym:** ICAN **Brief Title:** Assessing Impact of Active Learning on Student Outcomes: Texas Initiatives for Children's Activity and Nutrition (ICAN) **Official Title:** A Cluster Randomized Control Trial to Assess the Impact of Active Learning on Child Activity, Attention Control, and Academic Outcomes: The Texas I-CAN Trial #### Organization Study ID Info **ID:** 1R01HD070741 **Link:** https://reporter.nih.gov/quickSearch/1R01HD070741 **Type:** NIH #### Organization **Class:** OTHER **Full Name:** University of Texas at Austin ### Status Module #### Completion Date **Date:** 2015-05-31 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-06-26 **Type:** ACTUAL **Last Update Submit Date:** 2017-06-22 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2015-05-31 **Type:** ACTUAL #### Start Date **Date:** 2012-08-15 **Type:** ACTUAL **Status Verified Date:** 2017-06 #### Study First Post Date **Date:** 2017-03-22 **Type:** ACTUAL **Study First Submit Date:** 2017-03-09 **Study First Submit QC Date:** 2017-03-15 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Texas at Austin #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** Background: Active learning is designed to pair physical activity with the teaching of academic content. This has been shown to be a successful strategy to increase physical activity and improve academic performance. The existing designs have confounded academic lessons with physical activity. As a result, it is impossible to determine if the subsequent improvement in academic performance is due to: (1) physical activity, (2) the academic content of the active learning, or (3) the combination of academic material taught through physical activity. Methods / Design: The Texas I-CAN project is a 3-arm, cluster randomized control trial in which 28 elementary schools were assigned to either control, math intervention, or spelling intervention. As a result, each intervention condition serves as an unrelated content control for the other arm of the trial, allowing the impact of physical activity to be separated from the content. That is, schools that perform only active math lessons provide a content control for the spelling schools on spelling outcomes. This also calculated direct observations of attention and behavior control following periods of active learning. Discussion: This design is unique in its ability to separate the impact of physical activity, in general, from the combination of physical activity and specific academic content. This, in combination with the ability to examine both proximal and distal outcomes along with measures of time on task will do much to guide the design of future, school-based interventions. ### Conditions Module **Conditions:** - Physical Activity ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** OTHER #### Enrollment Info **Count:** 2716 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Texas I-CAN Active math lesson in academic classroom **Intervention Names:** - Behavioral: Texas I-CAN! **Label:** Texas I-CAN Active Math Lessons **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Texas I-CAN Active language arts lessons in academic classroom **Intervention Names:** - Behavioral: Texas I-CAN! **Label:** Texas I-CAN Active Language Arts Lessons **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Regular, Sedentary academic lessons in math and language arts **Label:** Control **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Texas I-CAN Active Language Arts Lessons - Texas I-CAN Active Math Lessons **Description:** physically active, academic lessons in the elementary school classroom **Name:** Texas I-CAN! **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** time in moderate to vigorous physical activity **Measure:** Physical activity level **Time Frame:** one school week **Description:** student attentional focus in the classroom 15 minutes prior to and 15 minutes post active lesson implemented in the classroom on one school day **Measure:** Time on task **Time Frame:** change in time on task from baseline to 15 minutes post academic lesson **Description:** acute academic scores on math and language arts test developed by fourth grade teacher **Measure:** Acute academic achievement **Time Frame:** change in math and language arts scores from Baseline to 2 weeks **Description:** Academic scores on math sub tests (Calculation, Fluency) of the Woodcock Johnson III **Measure:** Long-term math achievement **Time Frame:** Change in math achievement scores from Baseline to 7 months **Description:** Academic scores on spelling sub test of the Woodcock Johnson III and comprehension section of Gates MacGinitie test **Measure:** Long-term language arts achievement **Time Frame:** Change in language arts achievement from Baseline to 7 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * students in 4th grade in a participating elementary school and their 4th grade teachers Exclusion Criteria: * none **Healthy Volunteers:** True **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT ### Contacts Locations Module #### Overall Officials **Official 1:** **Affiliation:** UT Austin **Name:** John B Bartholomew, PHD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Bartholomew JB, Jowers EM, Errisuriz VL, Vaughn S, Roberts G. A cluster randomized control trial to assess the impact of active learning on child activity, attention control, and academic outcomes: The Texas I-CAN trial. Contemp Clin Trials. 2017 Oct;61:81-86. doi: 10.1016/j.cct.2017.07.023. Epub 2017 Jul 22. **PMID:** 28739542 ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02987179 **Brief Title:** Identification of Desaturation Episodes by Means of Continuous Measurement of Oxygen Saturation During Hemodialysis **Official Title:** Identification of Desaturation Episodes by Means of Continuous Measurement of Oxygen Saturation During Hemodialysis #### Organization Study ID Info **ID:** RRI-16-002 #### Organization **Class:** OTHER **Full Name:** Renal Research Institute ### Status Module #### Completion Date **Date:** 2017-12 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** ENROLLING_BY_INVITATION #### Last Update Post Date **Date:** 2016-12-08 **Type:** ESTIMATED **Last Update Submit Date:** 2016-12-06 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2017-03 **Type:** ESTIMATED #### Start Date **Date:** 2016-12 **Status Verified Date:** 2016-12 #### Study First Post Date **Date:** 2016-12-08 **Type:** ESTIMATED **Study First Submit Date:** 2016-12-06 **Study First Submit QC Date:** 2016-12-06 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Renal Research Institute #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The clinical diagnosis of sleep apnea is difficult among ESRD patients since the characteristic clinical features of sleep apnea may be absent and since sleep-related symptoms, such as fatigue and sleepiness, may be attributed to kidney failure. However, the evaluation of patients with possible sleep apnea is the same among ESRD patients as in the general population. ### Conditions Module **Conditions:** - ESRD - Sleep Apnea - Oxygen Deficiency ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 30 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** The following inclusion criteria must be met for each subject. * Age ≥18 years and able to give written informed consent to the study * On chronic hemodialysis for ≥ 90 days at time of enrollment * Ability to read * Consent to have video recording taken during study visit **Intervention Names:** - Other: Observational **Label:** ESRD Patients ### Interventions #### Intervention 1 **Arm Group Labels:** - ESRD Patients **Description:** Collect anthropomorphic, WatchPAT, Crit-line Monitor measurements as well as sleep questionnaires and video records **Name:** Observational **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** • To determine the sensitivity and specificity of the CLM for identifying intradialytic desaturation episodes, using the WatchPAT device as the reference method. **Measure:** Identifying Intradialytis desaturation episodes **Time Frame:** 1 week ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Age ≥18 years and able to give written informed consent to the study * On chronic hemodialysis for ≥ 90 days at time of enrollment * Ability to read * Consent to have video recording taken during study visit Exclusion Criteria: * Subjects with cognitive impairment to consent * Sickle cell anemia * Prescription of nasal oxygen * Simultaneous participation in another clinical trial that may affect breathing frequency/patterns, blood oxygenation, sleep/wake cycle or other parameters related to the outcomes of interest to this study * Use of a breathing device, e.g. CPAP * Central venous catheter used as vascular access for hemodialysis * Artificial fingernails (may disturb the measurement of the WatchPAT) * Use of one of the following medications: alpha blockers, short acting nitrates (less than 3 hours before the study). * Permanent pacemaker * Sustained non-sinus cardiac arrhythmias. **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Chronic hemodialysis patients ### Contacts Locations Module #### Locations **Location 1:** **City:** New York **Country:** United States **Facility:** Renal Research Institute **State:** New York **Zip:** 10065 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012818 - Term: Signs and Symptoms, Respiratory ### Condition Browse Module - Browse Branches - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases ### Condition Browse Module - Browse Leaves - ID: M4361 - Name: Apnea - Relevance: LOW - As Found: Unknown - ID: M15694 - Name: Sleep Apnea Syndromes - Relevance: LOW - As Found: Unknown - ID: M4185 - Name: Hypoxia - Relevance: HIGH - As Found: Oxygen Deficiency - ID: M15623 - Name: Signs and Symptoms, Respiratory - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000000860 - Term: Hypoxia ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01359579 **Brief Title:** A Study of the Pharmacokinetics and Safety of Varespladib in Subjects With Normal or Impaired Renal Function **Official Title:** A Phase 1, Open-label, Pharmacokinetic, Safety, and Tolerability Study of a Single Oral Dose of Varespladib Methyl in Subjects With Normal Renal Function, and Subjects With Mild, Moderate, or Severe Renal Impairment #### Organization Study ID Info **ID:** AN-CVD2213 #### Organization **Class:** INDUSTRY **Full Name:** Anthera Pharmaceuticals ### Status Module #### Completion Date **Date:** 2012-06 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2012-03-20 **Type:** ESTIMATED **Last Update Submit Date:** 2012-03-19 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2012-04 **Type:** ESTIMATED #### Start Date **Date:** 2011-06 **Status Verified Date:** 2012-03 #### Study First Post Date **Date:** 2011-05-25 **Type:** ESTIMATED **Study First Submit Date:** 2011-05-19 **Study First Submit QC Date:** 2011-05-23 **Why Stopped:** Lack of efficacy ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Anthera Pharmaceuticals #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to compare and evaluate the pharmacokinetic characteristics and the safety of varespladib methyl in mild or moderate renal impairment patients and healthy volunteers. ### Conditions Module **Conditions:** - Renal Impairment **Keywords:** - renal impairment - phase 1 - healthy volunteers ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** BASIC_SCIENCE #### Enrollment Info **Count:** 28 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Drug: varespladib methyl **Label:** Subjects with mild renal impairment **Type:** EXPERIMENTAL #### Arm Group 2 **Intervention Names:** - Drug: varespladib methyl **Label:** Subjects with moderate renal impairment **Type:** EXPERIMENTAL #### Arm Group 3 **Intervention Names:** - Drug: varespladib methyl **Label:** Subjects with normal renal function **Type:** EXPERIMENTAL #### Arm Group 4 **Intervention Names:** - Drug: varespladib methyl **Label:** Subjects with severe renal impairment **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Subjects with mild renal impairment - Subjects with moderate renal impairment - Subjects with normal renal function - Subjects with severe renal impairment **Description:** Single oral 500 mg dose **Name:** varespladib methyl **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Measure of blood and urine levels of varespladib in subjects with renal impairment in comparison to subjects with normal renal function **Time Frame:** PK samples will be collected predose and 0.5, 1, 1.5, 2, 3, 4, 6, 9, 12, 24, 48, and 72 hours postdose #### Secondary Outcomes **Measure:** Safety measures to include adverse events and changes in clinical laboratory results **Time Frame:** From admistration of study drug through follow-up on Day 8 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Males and non-pregnant, non-lactating females 18 years or older with a BMI of 18-40 kg/m2 inclusive. * Regarding renal function, subjects will be classified as either normal or as suffering from mild,moderate or severe renal impairment. Classification of renal impairment will be estimated by the MDRD and Cockcroft-Gault formulae Exclusion Criteria: * Any disease, condition and/or chronic medications which might compromise the hematologic, cardiovascular, pulmonary renal, gastrointestinal, hepatic, or central nervous system; or other conditions that might interfere with the distribution, metabolism or excretion of study drug, or would place the subject at increased risk * Evidence of significant respiratory, gastrointestinal or hepatic disease at screening * Positive screen for hepatitis B surface antigen, or HIV * Positive test in drugs of abuse screens or alcohol on admission to the clinic **Healthy Volunteers:** True **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Orlando **Country:** United States **Facility:** Investigator Site 101 **State:** Florida **Zip:** 32809 **Location 2:** **City:** Minneapolis **Country:** United States **Facility:** Investigator Site 102 **State:** Minnesota **Zip:** 55404 **Location 3:** **City:** Knoxville **Country:** United States **Facility:** Investigator Site 103 **State:** Tennessee **Zip:** 37920 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007674 - Term: Kidney Diseases - ID: D000014570 - Term: Urologic Diseases - ID: D000052776 - Term: Female Urogenital Diseases - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications - ID: D000091642 - Term: Urogenital Diseases - ID: D000052801 - Term: Male Urogenital Diseases ### Condition Browse Module - Browse Branches - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M26718 - Name: Renal Insufficiency - Relevance: HIGH - As Found: Renal Impairment - ID: M10698 - Name: Kidney Diseases - Relevance: LOW - As Found: Unknown - ID: M17319 - Name: Urologic Diseases - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M27095 - Name: Male Urogenital Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000051437 - Term: Renal Insufficiency ### Intervention Browse Module - Ancestors - ID: D000064801 - Term: Phospholipase A2 Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M346839 - Name: Varespladib methyl - Relevance: HIGH - As Found: DC1 - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: C000545088 - Term: Varespladib methyl ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04361279 **Brief Title:** A Study Comparing SIBP-02 and Rituximab Combination With CHOP in Previously Untreated Subjects With CD20+ DLBCL **Official Title:** A Phase III, Multicenter, Randomized, Double-blind Study Comparing the Efficacy and Safety of SIBP-02 (Rituximab Biosimilar) and Rituximab Combination With CHOP in Previously Untreated Subjects With CD20+ DLBCL #### Organization Study ID Info **ID:** SIBP-02-03 #### Organization **Class:** INDUSTRY **Full Name:** Shanghai Institute Of Biological Products ### Status Module #### Completion Date **Date:** 2022-05-20 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-12-27 **Type:** ACTUAL **Last Update Submit Date:** 2023-12-24 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2022-05-20 **Type:** ACTUAL #### Start Date **Date:** 2019-07-25 **Type:** ACTUAL **Status Verified Date:** 2023-12 #### Study First Post Date **Date:** 2020-04-24 **Type:** ACTUAL **Study First Submit Date:** 2020-04-22 **Study First Submit QC Date:** 2020-04-23 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Shanghai Institute Of Biological Products #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** This is a randomised, double-blind, positive drug parallel controlled equivalence clinical trial initiated at about 30 sites in China. In the trial, it is planned to enroll 414 subjects, randomized to two treatment groups in a ratio of 1:1 to receive the test drug and the positive control. ### Conditions Module **Conditions:** - Diffuse Large B-Cell Lymphoma ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** TRIPLE **Who Masked:** - PARTICIPANT - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 421 **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Biological: SIBP-02, 375 mg/m2, intravenous infusion, administered on day1. Drug: Cyclophosphamide, 750 mg/m2, intravenous infusion, administered on day2. Drug: Doxorubicin Hydrochloride, 50 mg/m2, intravenous infusion, administered on day2. Drug: Vincristine Sulfate, 1.4 mg/m2, intravenous infusion, administered on day2. Drug: Prednisone Acetate Tablets, 100 mg, orally administered on day2-6, one time per day. Treatment cycle: 6 cycles, each cycle is 3 weeks. **Intervention Names:** - Drug: SIBP-02 **Label:** SIBP-02 **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Biological: Rituximab, 375 mg/m2, intravenous infusion, administered on day1. Drug: Cyclophosphamide, 750 mg/m2, intravenous infusion, administered on day2. Drug: Doxorubicin Hydrochloride, 50 mg/m2, intravenous infusion, administered on day2. Drug: Vincristine Sulfate, 1.4 mg/m2, intravenous infusion, administered on day2. Drug: Prednisone Acetate Tablets, 100 mg, orally administered on day2-6, one time per day. Treatment cycle: 6 cycles, each cycle is 3 weeks. **Intervention Names:** - Drug: Rituximab **Label:** Rituximab **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - SIBP-02 **Description:** Injection，100mg/10ml **Name:** SIBP-02 **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Rituximab **Description:** Injection，100mg/10ml **Name:** Rituximab **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** defined as the proportion of patients with Complete Response (CR) and Partial Response (PR) in accordance with the evaluation criteria of the International Working Group (IWG) Lymphoma Efficacy Evaluation System (2007) **Measure:** Overall Response Rate (ORR) **Time Frame:** After 6 cycles of treatment, each cycle is 3 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * 18-75 years old, male or female; * Patients diagnosed as CD20-positive diffuse large B-cell lymphoma after histopathological or cytological examination and untreated; * ECOG score ≤ 2 when enrolled; * Echocardiography measured LVEF ≥ 50%; * The laboratory indicators during the screening period shall meet the following criteria: White blood cell count (WBC) ≥ 4.0 × 109/L or the lower limit of normal of the local laboratory; patients with bone marrow invasion, WBC ≥ 3.0 × 109/L; Absolute neutrophil count (ANC) ≥ 2.0 × 109/L or the lower limit of normal of the local laboratory; in the patients with bone marrow invasion, ANC ≥ 1.5 × 109/L; Hemoglobin (HB) ≥ 90 g/L; in the patients with bone marrow invasion, HB ≥ 80 g/L; Platelets (PLT) ≥ 100 × 109/L; in the patients with bone marrow invasion, PLT ≥ 75 × 109/L; Total bilirubin ≤ 1.5 times upper limit of normal (ULN); Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 × ULN; Alkaline phosphatase ≤ 1.5 × ULN in the patients without bone marrow invasion; Serum creatinine level ≤ 1.5 × ULN; * Patients having at least one two-dimensional measurable lesion as the basis for evaluation: for intra-nodal lesions, ≥1.5 cm in the long diameter and ≥1.0 cm in the short diameter; for extra-nodal lesions, ≥1.0 cm in the long diameter; * Patients with lymphoma International Prognostic Index (IPI) score of 0-2, with stage I to IV; * Female at the childbearing age who show negative in the pregnancy test, and agree to take effective contraceptive measures during the study period and within 12 months after the last dose; male patients who agree to take effective contraceptive measures during the study period and within 3 months after the last dose; * Patients with the expected survival period of greater than 6 months; * Patients voluntary to sign the Informed Consent Form. Exclusion Criteria: * Patients with primary central nervous system lymphoma and secondary central nervous system invasion, gray zone lymphoma between Burkitt and DLBCL, gray zone lymphoma between DLBCL and HL, primary mediastinal DLBCL, primary exudative lymphoma, plasmablastic lymphoma, primary skin DLBCL, ALK-positive DLBCL or transformed lymphoma; * Patients with double hit (BCL-2 and c-MYC gene rearrangement) or triple hit (BCL-2, BCL-6 and c-MYC gene rearrangement) diffuse large B-cell lymphoma diagnosed by the FISH test method; or patients with the pathological immunohistochemical test results as follows: BCL-2 ≥70% positive and c-MYC≥40% positive and tumor cells judged to be the source of germinal center according to Han's evaluation criteria but without exact FISH test result; * Patients with history of malignant tumors other than cutaneous squamous cell carcinoma, cutaneous basal cell carcinoma and cervical carcinoma in situ within 5 years prior to enrollment; * Patients with major surgery (excluding diagnostic surgery) within 2 months prior to enrollment; * Patients treated for non-Hodgkin's lymphoma: Chemotherapy and immunotherapy; Radiotherapy or local radiotherapy for DLBCL; Monoclonal antibody therapy (including Rituxan® and biosimilars of Rituxan®) Surgery (except biopsy); * Patients previously receiving cytotoxic drugs or anti-CD20 antibodies to treat other diseases (e.g. rheumatoid arthritis); * Patients receiving any monoclonal antibody within 3 months prior to enrollment; * Patients who participated in other clinical trials and used other trial-related drugs within 3 months prior to enrollment; * Those vaccinated within 1 month prior to enrollment; * Those receiving hematopoietic cytokines, e.g. granulocyte colony-stimulating factor (G-CSF) within 2 weeks prior to enrollment; * Those with the maximum dose of \>100mg Prednisone Acetate Tablets or equivalent cortisols for more than 5 days for the purpose of controlling lymphoma, or with the daily dose of \>30mg Prednisone Acetate Tablets or equivalent cortisols for more than 10 days for the other purposes. For the patients with daily dose of ≤30mg Prednisone Acetate Tablets or equivalent cortisols, there shall be written record on stable use of pre-randomization dose for at least 4 weeks; * Patients with peripheral nervous system or central nervous system disorder; * Patients with suspected active or latent tuberculosis; * Patients with known uncontrolled active bacterial, viral, fungal, mycobacterial, parasitic or other infections (excluding nail bed fungal infection) or with any significant systemic infection event that requires intravenous antibiotic treatment or hospitalization (except for neoplastic fever) within 4 weeks prior to enrollment; * HIV antibody positive; * HCV antibody positive; * HBV infection: (1) HBsAg positive, or (2) HBsAg negative, HBcAb positive and HBV DNA \> 1 × 103 * Patients with complicated other diseases that might restrict the patients from participation in the clinical trial in the opinion of the investigator, including but not limited to: Cardiovascular disease: congestive heart failure (NYHA Class III-IV), unstable angina, poorly controlled arrhythmias, poorly controlled hypertension or hypotension or myocardial infarction in the past 6 months; Severe mental illness; Gastric ulcer, intestinal infarction or gastrointestinal perforation (except for ones caused by primary disease); Pulmonary diseases (asthma, interstitial lung disease or severe obstructive pulmonary disease); Poorly controlled diabetes. * Patients with contraindication to any drug in the CHOP chemotherapy regime; * Patients with allergy constitution or known to be allergic to the active ingredient, excipient or rodent product or xenogeneic protein of any drug contained in this clinical trial (including the CHOP regime); * Patients unsuitable for enrollment due to alcohol or drug abuse in the opinion of the investigator; * Female in the pregnancy or breast-feeding period; * Those unsuitable to participate in the clinical trial in the opinion of the investigator. **Maximum Age:** 75 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Hangzhou **Country:** China **Facility:** The First Affiliated Hospital of College of Medicine, Zhejiang University #### Overall Officials **Official 1:** **Affiliation:** SINOPHARM **Name:** Shanghai Institute Of Biological Products Co., Ltd **Role:** STUDY_DIRECTOR **Official 2:** **Affiliation:** Zhejiang University **Name:** The First Affiliated Hospital of College of Medicine **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ## Derived Section ### Condition Browse Module - Ancestors - ID: D000016393 - Term: Lymphoma, B-Cell - ID: D000008228 - Term: Lymphoma, Non-Hodgkin - ID: D000008223 - Term: Lymphoma - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000009369 - Term: Neoplasms - ID: D000008232 - Term: Lymphoproliferative Disorders - ID: D000008206 - Term: Lymphatic Diseases - ID: D000007160 - Term: Immunoproliferative Disorders - ID: D000007154 - Term: Immune System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC15 - Name: Blood and Lymph Conditions - Abbrev: BC20 - Name: Immune System Diseases - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M11220 - Name: Lymphoma - Relevance: LOW - As Found: Unknown - ID: M18831 - Name: Lymphoma, Large B-Cell, Diffuse - Relevance: HIGH - As Found: Diffuse Large B-Cell Lymphoma - ID: M18828 - Name: Lymphoma, B-Cell - Relevance: LOW - As Found: Unknown - ID: M11222 - Name: Lymphoma, Non-Hodgkin - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: M11225 - Name: Lymphoproliferative Disorders - Relevance: LOW - As Found: Unknown - ID: M11203 - Name: Lymphatic Diseases - Relevance: LOW - As Found: Unknown - ID: M10206 - Name: Immunoproliferative Disorders - Relevance: LOW - As Found: Unknown - ID: M10200 - Name: Immune System Diseases - Relevance: LOW - As Found: Unknown - ID: T3543 - Name: Lymphosarcoma - Relevance: LOW - As Found: Unknown - ID: T640 - Name: B-cell Lymphoma - Relevance: HIGH - As Found: B-cell Lymphoma - ID: T1866 - Name: Diffuse Large B-Cell Lymphoma - Relevance: HIGH - As Found: Diffuse Large B-Cell Lymphoma ### Condition Browse Module - Meshes - ID: D000016403 - Term: Lymphoma, Large B-Cell, Diffuse ### Intervention Browse Module - Ancestors - ID: D000074322 - Term: Antineoplastic Agents, Immunological - ID: D000000970 - Term: Antineoplastic Agents - ID: D000007155 - Term: Immunologic Factors - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018501 - Term: Antirheumatic Agents ### Intervention Browse Module - Browse Branches - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: ARhu - Name: Antirheumatic Agents ### Intervention Browse Module - Browse Leaves - ID: M14121 - Name: Prednisone - Relevance: LOW - As Found: Unknown - ID: M6727 - Name: Cyclophosphamide - Relevance: LOW - As Found: Unknown - ID: M373 - Name: Rituximab - Relevance: HIGH - As Found: Healthy - ID: M7492 - Name: Doxorubicin - Relevance: LOW - As Found: Unknown - ID: M227339 - Name: Liposomal doxorubicin - Relevance: LOW - As Found: Unknown - ID: M17495 - Name: Vincristine - Relevance: LOW - As Found: Unknown - ID: M1346 - Name: Antineoplastic Agents, Immunological - Relevance: LOW - As Found: Unknown - ID: M10201 - Name: Immunologic Factors - Relevance: LOW - As Found: Unknown - ID: M20604 - Name: Antirheumatic Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000069283 - Term: Rituximab ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03966079 **Acronym:** STEP **Brief Title:** Thrombolysis Endovascular Treatment of Pulmonary Embolism **Official Title:** Single-dose rtPA Thrombolysis Endovascular Treatment of Pulmonary Embolism With Right Ventricular Dysfunction:a Pilot Study #### Organization Study ID Info **ID:** BeijingTCGH #### Organization **Class:** OTHER **Full Name:** Beijing Tsinghua Chang Gung Hospital ### Status Module #### Completion Date **Date:** 2020-01-31 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-11-30 **Type:** ACTUAL **Last Update Submit Date:** 2020-11-26 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2020-01-31 **Type:** ACTUAL #### Start Date **Date:** 2017-01-01 **Type:** ACTUAL **Status Verified Date:** 2020-11 #### Study First Post Date **Date:** 2019-05-29 **Type:** ACTUAL **Study First Submit Date:** 2019-05-20 **Study First Submit QC Date:** 2019-05-27 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Beijing Tsinghua Chang Gung Hospital #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Is US Export:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** To assess the efficacy and safety of endovascular thrombolysis of pulmonary embolism with right ventricular dysfunction with single-dose rtPA **Detailed Description:** The patients with right ventricular dysfunction who are diagnosed by CT pulmonary angiography will receive 20 mg of single-dose recombinant tissue plasminogen activator delivered through the in this study. The Change of RV to LV Diameter Ratio,Rate of death,rate of major bleeding,pulmonary embolism recurrence at 1month will be reviewed to assess the efficacy and safety. ### Conditions Module **Conditions:** - Pulmonary Embolism - Acute Pulmonary Embolism **Keywords:** - Acute Pulmonary Embolism ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP **Intervention Model Description:** Prospective Single Group Assignment ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 50 **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients receive 20 mg of single-dose recombinant tissue plasminogen activator delivered through the catheter **Intervention Names:** - Drug: recombinant tissue plasminogen activator **Label:** Intervention arm **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Intervention arm **Description:** Patients receive 20 mg of single-dose recombinant tissue plasminogen activator delivered through the catheter **Name:** recombinant tissue plasminogen activator **Other Names:** - rtPA **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Determine whether single-dose rtpa catheter-directed thrombolysis decrease the ratio of right ventricular (RV)-to-left ventricular (LV) diameter within 1month in patients with right ventricular dysfunction **Measure:** Change of RV to LV Diameter Ratio **Time Frame:** baseline and 30days after baseline #### Secondary Outcomes **Measure:** Rate of death **Time Frame:** 30days **Measure:** Rate of major Bleeding **Time Frame:** 30days **Measure:** Pulmonary embolism recurrence **Time Frame:** 30days ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * CT evidence of proximal PE * Age ≥ 18 years * Pulmonary embolism symptom duration ≤14 days * Massive PE (syncope, systemic arterial hypotension, cardiogenic shock, or resuscitated cardiac arrest) OR Submassive PE (RV diameter-to-LV diameter ≥ 0.9 on contrast-enhanced chest CT) Exclusion Criteria: * Age \<18 years * PE symptom duration \>14 days * Stroke or transient ischemic attack (TIA), head trauma, or other active intracranial or intraspinal disease within one year * Recent (within one month) or active bleeding from a major organ * Pregnancy * Chronic pulmonary hypertension or severe chronic obstructive pulmonary disease * Administration of thrombolytic agents within the previous 3 days * Life expectancy \< 30 days * Any other condition that the investigator feels would place the patient at increased risk if the investigational therapy is initiated * Inability to follow protocol requirements **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Beijing **Country:** China **Facility:** Beijing Tsinghua Chang Gung Hospital **State:** Beijing **Zip:** 102218 #### Overall Officials **Official 1:** **Affiliation:** Director of department **Name:** Wu weiwei, doctor **Role:** STUDY_DIRECTOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000016769 - Term: Embolism and Thrombosis - ID: D000014652 - Term: Vascular Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000008171 - Term: Lung Diseases - ID: D000012140 - Term: Respiratory Tract Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M20824 - Name: Ventricular Dysfunction - Relevance: LOW - As Found: Unknown - ID: M20601 - Name: Ventricular Dysfunction, Right - Relevance: LOW - As Found: Unknown - ID: M14509 - Name: Pulmonary Embolism - Relevance: HIGH - As Found: Pulmonary Embolism - ID: M7784 - Name: Embolism - Relevance: HIGH - As Found: Embolism - ID: M16686 - Name: Thrombosis - Relevance: LOW - As Found: Unknown - ID: M19128 - Name: Embolism and Thrombosis - Relevance: LOW - As Found: Unknown - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown - ID: M11168 - Name: Lung Diseases - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000011655 - Term: Pulmonary Embolism - ID: D000004617 - Term: Embolism ### Intervention Browse Module - Ancestors - ID: D000005343 - Term: Fibrinolytic Agents - ID: D000050299 - Term: Fibrin Modulating Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### Intervention Browse Module - Browse Branches - Abbrev: FiAg - Name: Fibrinolytic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M13848 - Name: Plasminogen - Relevance: HIGH - As Found: Defibrillator - ID: M13849 - Name: Tissue Plasminogen Activator - Relevance: HIGH - As Found: Respiration - ID: M8473 - Name: Fibrinolytic Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000010958 - Term: Plasminogen - ID: D000010959 - Term: Tissue Plasminogen Activator ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01186679 **Acronym:** ABMST-SCI **Brief Title:** Safety and Efficacy of Autologous Bone Marrow Stem Cells in Treating Spinal Cord Injury **Official Title:** Surgical Transplantation of Autologous Bone Marrow Stem Cells With Glial Scar Resection for Patients of Chronic Spinal Cord Injury and Intra-thecal Injection for Acute and Subacute Injury - A Preliminary Study #### Organization Study ID Info **ID:** ISSL-AuBM-SCI #### Organization **Class:** INDUSTRY **Full Name:** International Stemcell Services Limited ### Status Module #### Completion Date **Date:** 2010-08 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2010-08-23 **Type:** ESTIMATED **Last Update Submit Date:** 2010-08-20 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2010-02 **Type:** ACTUAL #### Start Date **Date:** 2008-01 **Status Verified Date:** 2010-08 #### Study First Post Date **Date:** 2010-08-23 **Type:** ESTIMATED **Study First Submit Date:** 2010-08-20 **Study First Submit QC Date:** 2010-08-20 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** International Stemcell Services Limited #### Responsible Party **Old Name Title:** Dr.SGA.Rao, Chairman **Old Organization:** International Stemcell Services Ltd. ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The projected data related to the burden of spinal cord injuries induced limb paralysis in India is quite alarming. This is attributed to the rapid industrialization and economical development in the country. Increase in vehicular traffic has caused numerous road traffic accidents. Rapid increase in populations, development in the computer technology and real estate business lead to construction of huge buildings which indirectly adds to the injuries due to fall. Spinal cord injuries could not be treated adequately with the prevailing treatment modalities. In view of this, there is definitely an urgent need for finding different methods of treatment for these patients who cannot undergo established modalities of treatment or these have been tried unsuccessfully. Since a large number of these patients will loose their productive life and at the prime of their lives, one such alternate therapy, which seems to offer some promise, is "stem cell" therapy, which has been well studied and published in prestigious journals. In our present study, we want to evaluate the safety and efficacy of autologous bone marrow derived stem cells surgically transplanted directly into the lesion site with glial scar resection for 8 indian patients of chronic spinal cord injury and intra-thecal injection for 4 indian patients of acute and subacute injury. ### Conditions Module **Conditions:** - Spinal Cord Injuries ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 12 **Type:** ACTUAL **Phases:** - PHASE1 - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 1. Surgical transplantation into the lesion site in chronic patients 2. Direct intrathecal implantation in acute and subacute patients **Intervention Names:** - Procedure: laminectomy **Label:** Intralesional **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** direct into the CSF through lumbar puncture **Intervention Names:** - Procedure: Intrathecal **Label:** intrathecal **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Intralesional **Description:** surgical laminectomy with glial scar resection **Name:** laminectomy **Type:** PROCEDURE #### Intervention 2 **Arm Group Labels:** - intrathecal **Description:** direct into the CSF through lumbar puncture **Name:** Intrathecal **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Description:** American Spinal Injury Assessment scale of A,B,C,D or E **Measure:** Number of Participants with adverse events as a measure of safety and tolerability. Significant clinical improvement in ASIA impairment scale and general condition **Time Frame:** 18 months #### Secondary Outcomes **Description:** MRI findings of the lesion, Nerve conduction studies of the region and somatosensory evoked potentials of the same region **Measure:** Changes in the MRI, Neurological improvement (cranial/spinal reflexes) and evoked potentials study **Time Frame:** 18 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Must be able to give voluntary (patients may not be able to write) consent. 2. Must be able to understand study information provided to him. 3. Patients with complete spinal cord trans-section: at least post 6 months after spinal cord Injury (in chronic patients), \< 2 weeks in acute category and 2-8 weeks in subacute patients. 4. The level of spinal cord injury must be between C4 and T12(neurological level) 5. Spinal cord injury categorized in terms of ASIA Impairment scale. 6. Age should be between 20-55 years Exclusion Criteria: * Mechanical ventilation due to neurological impairment * Multiple level trauma * Undetermined size and location of Spinal Cord injury * Gunshot or other penetrating trauma to the spinal cord * Longitudinal dimension of injury by MRI is greater than 3spinal segments * Associated severe head injury * More than 9cms long bone fracture * Women who are pregnant or lactating * Serious pre-existing medical conditions * Disease or impairment that precludes adequate neurological examination. * Should not have co-morbidities like Diabetes, Systemic Hypertension etc. * Severe co-morbidities/bed sores Tests positive for infectious diseases Deranged Coagulation profile and Hb \< 8mg/dl **Maximum Age:** 55 Years **Minimum Age:** 20 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Bangalore **Country:** India **Facility:** Sita Bhateja Speciality Hospital **State:** Karnataka **Zip:** 560025 #### Overall Officials **Official 1:** **Affiliation:** Sita Bhateja Speciality Hospital **Name:** Dr.Arvind Bhateja, MCh.Neurosurgery **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000013118 - Term: Spinal Cord Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000020196 - Term: Trauma, Nervous System ### Condition Browse Module - Browse Branches - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC26 - Name: Wounds and Injuries - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M15916 - Name: Spinal Cord Injuries - Relevance: HIGH - As Found: Spinal Cord Injury - ID: M6160 - Name: Cicatrix - Relevance: LOW - As Found: Unknown - ID: M17685 - Name: Wounds and Injuries - Relevance: HIGH - As Found: Injury - ID: M15915 - Name: Spinal Cord Diseases - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M22023 - Name: Trauma, Nervous System - Relevance: LOW - As Found: Unknown - ID: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: LOW - As Found: Unknown - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000013119 - Term: Spinal Cord Injuries - ID: D000014947 - Term: Wounds and Injuries ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01298479 **Brief Title:** Sayana-Uniject Volumetric Delivery **Official Title:** Quantitative Determination of the Volume of Suspension Delivered Via the Uniject Delivery System Following Simulated Subcutaneous Injection by Healthcare Professionals Into a Synthetic Subcutaneous Injection Training Device #### Organization Study ID Info **ID:** A6791034 #### Organization **Class:** INDUSTRY **Full Name:** Pfizer ### Status Module #### Completion Date **Date:** 2010-06 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-12-07 **Type:** ACTUAL **Last Update Submit Date:** 2018-12-05 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2010-06 **Type:** ACTUAL #### Start Date **Date:** 2010-05 **Status Verified Date:** 2018-12 #### Study First Post Date **Date:** 2011-02-17 **Type:** ESTIMATED **Study First Submit Date:** 2010-12-16 **Study First Submit QC Date:** 2011-02-16 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Pfizer #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to determine the volumetric delivery of the Uniject. **Detailed Description:** Observe subjects deliver the drug None used ### Conditions Module **Conditions:** - Volume Delivery **Keywords:** - volume delivery ### Design Module #### Design Info **Observational Model:** OTHER **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 25 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** All subjects **Intervention Names:** - Other: Uniject **Label:** Group 1 ### Interventions #### Intervention 1 **Arm Group Labels:** - Group 1 **Description:** single use container **Name:** Uniject **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Measure:** Weight of drug suspension delivered (estimated by change in mass of the Uniject(TM) delivery system). **Time Frame:** Visit 1 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Nurses Exclusion Criteria: * Non-nurses **Healthy Volunteers:** True **Maximum Age:** 65 Years **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** nurses ### Contacts Locations Module #### Locations **Location 1:** **City:** Bruxelles **Country:** Belgium **Facility:** Pfizer Investigational Site **Zip:** B-1070 #### Overall Officials **Official 1:** **Affiliation:** Pfizer **Name:** Pfizer CT.gov Call Center **Role:** STUDY_DIRECTOR ### References Module #### See Also Links **Label:** To obtain contact information for a study center near you, click here. **URL:** https://trialinfoemail.pfizer.com/pages/landing.aspx?StudyID=A6791034&StudyName=Sayana-Uniject%20Volumetric%20Delivery ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01533779 **Brief Title:** Neutrophil Extracellular Traps (NETs) Formation Following Chemotherapy and Their Role in Antitumor Activity **Official Title:** Neutrophil Extracellular Traps (NETs) Formation Following Chemotherapy for Pediatric Hematological and Solid Tumors, and Its Relation to Other Neutrophil Functions and the Role of NETs in Antitumor Activity #### Organization Study ID Info **ID:** 0592-11-TLV #### Organization **Class:** OTHER_GOV **Full Name:** Tel-Aviv Sourasky Medical Center ### Status Module #### Completion Date **Date:** 2015-02 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2012-02-15 **Type:** ESTIMATED **Last Update Submit Date:** 2012-02-14 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2015-02 **Type:** ESTIMATED #### Start Date **Date:** 2012-02 **Status Verified Date:** 2012-02 #### Study First Post Date **Date:** 2012-02-15 **Type:** ESTIMATED **Study First Submit Date:** 2012-02-12 **Study First Submit QC Date:** 2012-02-14 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Max Planck Institute for Infection Biology **Class:** OTHER **Name:** Meir Medical Center #### Lead Sponsor **Class:** OTHER_GOV **Name:** Tel-Aviv Sourasky Medical Center #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Examine neutrophil extracellular traps (NETs) formation, in relation to other neutrophil functions like chemotaxis, superoxide production, hydrogen peroxide production, and the presence of myeloperoxidase, in pediatric patients undergoing chemotherpy for solid and hematological malignancies. This data could shed new light on the mechanism responsible for the increased susceptibility to infection among these patients and aid in improving their prophylactic antimicrobial treatment. NETs formation against tumor cell lines and their ability to kill tumor cells will also be examined. The finding of NETs activity against tumor cells could have a major contribution to the investigators understanding of the function of the immune system against cancer. **Detailed Description:** Neutrophil function, including NETs formation, chemotaxis, superoxide production, hydrogen peroxide production, and the presence of myeloperoxidase, will be examined in 50 pediatric patientsundergoing chemotherapy for solid and hematological malignancies. Children with the following malignancies will be examined: acute lymphoblastic leukemia,acute myelogenous leukemia,Hodgkin's lymphoma,non-Hodgkin's lymphoma, primary bone sarcoma,rhabdomyosarcoma,non-rhabdomyosarcoma,neuroblastoma,Wilms' tumor,hepatoblastoma or hepatocellular carcinoma,germ cell tumors, and hystiocytosis. Also those with brain tumors such as medulloblastom,low grade glioma,high grade glioma,ependymoma,and embryonal and pineal region tumors. Data gathered on the patients will include background data (age, gender, ethnicity) and background diseases, data on current illness (histologic type, grade, stage, response treatment,infectious episodes), and on the use of ranulocyte-colony stimulating factor (G-CSF). The following time points will be examined: 1. At diagnosis, before initiation of chemotherapy. 2. immediately before the 2nd course. 3. After the middle course. 4. A month after the last course. 5. Three months after the last course. 6. In acute myelogenous leukemia and acute lymphoblastic leukemia,time points also include the middle of the maintenance course and 3 months after the end of maintenance. An additional blood examination will be used to examine NETs formation against tumor cell lines and their ability to kill tumor cells. ### Conditions Module **Conditions:** - Pediatric Solid Malignancies - Pediatric Hematological Malignancies **Keywords:** - children - malignancy - neutrophil - neutrophil extracellular traps - superoxide production - hydrogen peroxide production - chemotaxis - myeloperoxidase - immunosurveillance - cancer cell lines ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 50 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: Included in the study were patients with: * Acute lymphoblastic leukemia or acute myelogenous leukemia. * Hodgkin's lymphoma or non-Hodgkin's lymphoma. * Primary bone sarcoma, rhabdomyosarcoma, non-rhabdomyosarcoma, neuroblastoma, Wilms' tumor, hepatoblastoma or hepatocellular carcinoma, hystiocytosis, and germ cell tumors. * Medulloblastoma, low grade glioma, high grade glioma, ependymoma, and embryonal and pineal region tumors. Exclusion Criteria: * A severe background disease that may affect Neutrophil function (e.g., diabetes, lupus). **Healthy Volunteers:** True **Maximum Age:** 21 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - CHILD - ADULT **Study Population:** All pediatric patients from birth to 21 years, male and female, undergoing chemotherapy treatment for solid and hematological malignancies. ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Sivan Achituv, MD **Phone:** 972-527360718 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Ronit Elhasid, MD **Phone:** 972-3-6974252 **Role:** CONTACT #### Locations **Location 1:** **City:** Tel-Aviv **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Sivan Achituv, MD - **Phone:** 972-527360718 - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Ronit Elhasid, MD - **Phone:** 972-3-6974252 - **Role:** CONTACT **Country:** Israel **Facility:** Department of pediatric hemato-oncology, Tel-Aviv Sourasky Medical Center **Status:** RECRUITING **Zip:** 64239 #### Overall Officials **Official 1:** **Affiliation:** The department of pediatric hemato-oncology, Dana Childrens' Hospital, Tel-Aviv Sourasky Medical Center, Tel-Aviv, Israel **Name:** Sivan Achituv, MD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009371 - Term: Neoplasms by Site - ID: D000006402 - Term: Hematologic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC15 - Name: Blood and Lymph Conditions - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M21314 - Name: Hematologic Neoplasms - Relevance: HIGH - As Found: Hematological Malignancies - ID: M9490 - Name: Hematologic Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000009369 - Term: Neoplasms - ID: D000019337 - Term: Hematologic Neoplasms ### Intervention Browse Module - Browse Branches - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M9918 - Name: Hydrogen Peroxide - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01704079 **Brief Title:** Safety and Efficacy of CTAP101 to Treat Secondary Hyperparathyroidism in Stage 3 or 4 CKD and Vitamin D Insufficiency **Official Title:** A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy and Safety of CTAP101 Capsules to Treat Secondary Hyperparathyroidism in Subjects With Stage 3 or 4 Chronic Kidney Disease and Vitamin D Insufficiency #### Organization Study ID Info **ID:** CTAP101-CL-3002 #### Organization **Class:** INDUSTRY **Full Name:** OPKO Health, Inc. ### Status Module #### Completion Date **Date:** 2014-09 **Type:** ACTUAL #### Disp First Post Date **Date:** 2015-11-16 **Type:** ESTIMATED **Disp First Submit Date:** 2015-10-22 **Disp First Submit QC Date:** 2015-10-22 #### Expanded Access Info #### Last Update Post Date **Date:** 2019-12-18 **Type:** ACTUAL **Last Update Submit Date:** 2019-12-17 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2014-08 **Type:** ACTUAL #### Results First Post Date **Date:** 2016-08-17 **Type:** ESTIMATED **Results First Submit Date:** 2016-07-08 **Results First Submit QC Date:** 2016-07-08 #### Start Date **Date:** 2012-11 **Status Verified Date:** 2019-12 #### Study First Post Date **Date:** 2012-10-11 **Type:** ESTIMATED **Study First Submit Date:** 2012-10-06 **Study First Submit QC Date:** 2012-10-09 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** OPKO IP Holdings II, Inc. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** This study will evaluate the efficacy of CTAP101 Capsules versus placebo in reducing intact parathyroid hormone (iPTH) by at least 30% from pretreatment baseline; safety and tolerability of CTAP101 will also be evaluated **Detailed Description:** This is a phase 3, multi-center, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of CTAP101 Capsules administered for 26 weeks to treat SHPT in subjects ≥18 years of age with stage 3 or 4 CKD and vitamin D insufficiency. The study will be conducted at approximately 40 sites within the United States (US). Approximately 550 subjects will be screened to randomize approximately 210 eligible subjects, stratified by CKD stage (approximately 105 subjects in each stage) in a 2:1 ratio to receive either CTAP101 Capsules or matching placebo. An Interactive Voice Response System (IVRS) will provide study treatment group assignments using the computer-generated randomization code provided by the IVRS vendor. Subjects will receive an initial daily dose of 1 capsule (CTAP101 Capsules, 30 µg, or matching placebo) for the first 12 weeks. After l2 weeks (visit 8) and per pre-defined criteria, the dose may be increased in a blinded fashion by the IVRS with oversight by an independent medical monitor. ### Conditions Module **Conditions:** - Chronic Kidney Disease - Hyperparathyroidism, Secondary - Vitamin D Deficiency **Keywords:** - Parathyroid Diseases - Renal Insufficiency - Kidney Failure, Chronic - Hyperparathyroidism, Secondary - Vitamin D - Hyperparathyroidism - Kidney Diseases - Kidney Failure - Renal Insufficiency, Chronic ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - INVESTIGATOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 216 **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 1 CTAP101 30 μg capsule daily at bedtime for 12 weeks, with a possible increase to 2 CTAP101 30 μg capsules daily at bedtime for study duration, if needed (26 weeks total). Subjects not requiring a dose increase after 12 weeks would receive 1 CTAP101 30 μg capsule and one sugar pill (to CTAP101 30 μg capsule) for weeks 13-26. **Intervention Names:** - Drug: CTAP101 30 μg capsules - Other: Sugar pill to CTAP101 30 μg capsules **Label:** CTAP101 30 μg capsules **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** 1 sugar pill to CTAP101 30 μg capsule daily at bedtime for 12 weeks, with an increase to 2 sugar pills to CTAP101 30 μg capsules daily at bedtime for weeks 13-26. **Intervention Names:** - Other: Sugar pill to CTAP101 30 μg capsules **Label:** Sugar pill to CTAP101 30 μg **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - CTAP101 30 μg capsules **Description:** CTAP101 30 μg capsule taken daily at bedtime **Name:** CTAP101 30 μg capsules **Other Names:** - Calcifediol **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - CTAP101 30 μg capsules - Sugar pill to CTAP101 30 μg **Description:** Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime. **Name:** Sugar pill to CTAP101 30 μg capsules **Other Names:** - Placebo **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Number of subjects in the intent to treat population attaining mean decrease in plasma intact Parathyroid Hormone (iPTH) of ≥30% from P\\pre-treatment baseline in the efficacy assessment phase (EAP) referred to as responders **Measure:** Number of Participants in the Intent to Treat Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline **Time Frame:** Approximately 6 months #### Secondary Outcomes **Description:** Number of subjects in the per protocol population attaining a mean decrease in plasma intact parathyroid hormone (iPTH) of ≥30% from pre-treatment baseline in the efficacy assessment phase (EAP), referred to as responders **Measure:** Number of Participants in the Per Protocol Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline **Time Frame:** Approximately 6 months **Description:** Subjects in the intent to treat population with normal serum total 25-hydroxyvitamin D (\>/= 30 ng/dL) **Measure:** Subjects in the Intent to Treat Population With Normal Serum Total 25-hydroxyvitamin D **Time Frame:** Approximately 6 months **Description:** Subjects in the per protocol population with normal serum total 25-hydroxyvitamin D (\>/= 30 ng/mL) **Measure:** Subjects in the Per Protocol Population With Normal Serum Total 25-hydroxyvitamin D **Time Frame:** Approximately 6 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Urinary albumin excretion ≤ 3000 mcg/mg of creatinine 2. Stage 3 or 4 CKD 3. Plasma iPTH: ≥ 85 pg/mL and \< 500 pg/mL 4. Serum Ca: ≥ 8.4 mg/dL and \< 9.8 mg/dL 5. Serum P: ≥ 2.0 mg/dL and \< 5.0 mg/dL 6. Serum 25-hydroxyvitamin D: ≥ 10 ng/mL and \< 30 ng/mL. 7. Stable dose of Vitamin D therapy ≤ 1600 IU/day and receiving same dose for at least 2 months Exclusion Criteria: 1. History of kidney transplant or parathyroidectomy 2. Spot urine calcium:creatinine ratio \> 0.2 (\>200 mg/g Cr) 3. Current serious illness, such as malignancy, HIV, liver disease, cardiovascular event or hepatitis 4. Currently on dialysis 5. Use of pharmacological dose of ergocalciferol or cholecalciferol (≥ 50,000 IU mcg per month) during the study **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Bannockburn **Country:** United States **Facility:** OPKO Renal **State:** Illinois **Zip:** 60015 #### Overall Officials **Official 1:** **Affiliation:** OPKO Renal **Name:** Joel Melnick, MD **Role:** STUDY_DIRECTOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014570 - Term: Urologic Diseases - ID: D000052776 - Term: Female Urogenital Diseases - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications - ID: D000091642 - Term: Urogenital Diseases - ID: D000052801 - Term: Male Urogenital Diseases - ID: D000051437 - Term: Renal Insufficiency - ID: D000002908 - Term: Chronic Disease - ID: D000020969 - Term: Disease Attributes - ID: D000010335 - Term: Pathologic Processes - ID: D000009385 - Term: Neoplastic Processes - ID: D000009369 - Term: Neoplasms - ID: D000001361 - Term: Avitaminosis - ID: D000003677 - Term: Deficiency Diseases - ID: D000044342 - Term: Malnutrition - ID: D000009748 - Term: Nutrition Disorders - ID: D000010279 - Term: Parathyroid Diseases - ID: D000004700 - Term: Endocrine System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: BC19 - Name: Gland and Hormone Related Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M10698 - Name: Kidney Diseases - Relevance: HIGH - As Found: Kidney Disease - ID: M26717 - Name: Renal Insufficiency, Chronic - Relevance: HIGH - As Found: Chronic Kidney Disease - ID: M12307 - Name: Neoplasm Metastasis - Relevance: HIGH - As Found: Secondary - ID: M10699 - Name: Kidney Failure, Chronic - Relevance: LOW - As Found: Unknown - ID: M17551 - Name: Vitamin D Deficiency - Relevance: HIGH - As Found: Vitamin D Deficiency - ID: M26718 - Name: Renal Insufficiency - Relevance: LOW - As Found: Unknown - ID: M10012 - Name: Hyperparathyroidism - Relevance: HIGH - As Found: Hyperparathyroidism - ID: M10013 - Name: Hyperparathyroidism, Secondary - Relevance: HIGH - As Found: Hyperparathyroidism, Secondary - ID: M13192 - Name: Parathyroid Diseases - Relevance: LOW - As Found: Unknown - ID: M17319 - Name: Urologic Diseases - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M27095 - Name: Male Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M6147 - Name: Chronic Disease - Relevance: LOW - As Found: Unknown - ID: M22700 - Name: Disease Attributes - Relevance: LOW - As Found: Unknown - ID: M12330 - Name: Neoplastic Processes - Relevance: LOW - As Found: Unknown - ID: M4660 - Name: Avitaminosis - Relevance: LOW - As Found: Unknown - ID: M6879 - Name: Deficiency Diseases - Relevance: LOW - As Found: Unknown - ID: M25306 - Name: Malnutrition - Relevance: LOW - As Found: Unknown - ID: M12684 - Name: Nutrition Disorders - Relevance: LOW - As Found: Unknown - ID: M7862 - Name: Endocrine System Diseases - Relevance: LOW - As Found: Unknown - ID: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000009362 - Term: Neoplasm Metastasis - ID: D000007674 - Term: Kidney Diseases - ID: D000051436 - Term: Renal Insufficiency, Chronic - ID: D000014808 - Term: Vitamin D Deficiency - ID: D000006961 - Term: Hyperparathyroidism - ID: D000006962 - Term: Hyperparathyroidism, Secondary ### Intervention Browse Module - Ancestors - ID: D000014815 - Term: Vitamins - ID: D000018977 - Term: Micronutrients - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000050071 - Term: Bone Density Conservation Agents ### Intervention Browse Module - Browse Branches - Abbrev: BDCA - Name: Bone Density Conservation Agents - Abbrev: Micro - Name: Micronutrients - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Vi - Name: Vitamins ### Intervention Browse Module - Browse Leaves - ID: M17550 - Name: Vitamin D - Relevance: LOW - As Found: Unknown - ID: M6003 - Name: Cholecalciferol - Relevance: LOW - As Found: Unknown - ID: M17558 - Name: Vitamins - Relevance: LOW - As Found: Unknown - ID: M5375 - Name: Calcifediol - Relevance: HIGH - As Found: Risk Population - ID: M13194 - Name: Parathyroid Hormone - Relevance: LOW - As Found: Unknown - ID: M21009 - Name: Micronutrients - Relevance: LOW - As Found: Unknown - ID: M16885 - Name: Trace Elements - Relevance: LOW - As Found: Unknown - ID: T442 - Name: Cholecalciferol - Relevance: LOW - As Found: Unknown - ID: T479 - Name: Vitamin D3 - Relevance: LOW - As Found: Unknown - ID: T440 - Name: Calciferol - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000002112 - Term: Calcifediol ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Sugar Pill to CTAP101 30 μg **Description:** 1 sugar pill to CTAP101 30 μg capsule daily at bedtime for 12 weeks, with an increase to 2 sugar pills to CTAP101 30 μg capsules daily at bedtime for weeks 13-26. Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime. **ID:** EG000 **Other Num Affected:** 18 **Other Num at Risk:** 72 **Serious Number Affected:** 11 **Serious Number At Risk:** 72 **Title:** Sugar Pill to CTAP101 30 μg **Group ID:** EG001 **Title:** CTAP101 30 μg Capsules **Description:** 1 CTAP101 30 μg capsule daily at bedtime for 12 weeks, with a possible increase to 2 CTAP101 30 μg capsules daily at bedtime for study duration, if needed (26 weeks total). Subjects not requiring a dose increase after 12 weeks would receive 1 CTAP101 30 μg capsule and one sugar pill (to CTAP101 30 μg capsule) for weeks 13-26. CTAP101 30 μg capsules: CTAP101 30 μg capsule taken daily at bedtime Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime. **ID:** EG001 **Other Num Affected:** 32 **Other Num at Risk:** 144 **Serious Number Affected:** 22 **Serious Number At Risk:** 144 **Title:** CTAP101 30 μg Capsules **Frequency Threshold:** 5 #### Other Events **Term:** Constipation **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Oedema peripheral **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Nasopharyngitis **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 **Term:** Gout **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Metabolic acidosis **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Pain in extremity **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Hypertension **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 15.0 #### Serious Events **Term:** Anaemia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 144 **Num Events:** 2 **Term:** Acute myocardial infarction **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 72 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Cardiac arrest **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Cardiac disorder **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Cardiac failure **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 72 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Cardiac failure acute **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Cardiac failure congestive **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 144 **Num Events:** 3 **Term:** Myocardial infarction **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Asthenia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 72 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 144 **Term:** Chest pain **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 72 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Device malfunction **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Cholelitiasis **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Cellulitis **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 72 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 144 **Term:** Localized infection **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Pneumonia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 72 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 144 **Num Events:** 3 **Term:** Pneumonia bacterial **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 72 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 144 **Term:** Sepsis **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 72 **Num Events:** 2 **Group ID:** EG001 **Num At Risk:** 144 **Term:** Urinary tract infection **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Fall **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Blood creatinine increased **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 144 **Num Events:** 3 **Term:** Dehydration **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 72 **Num Events:** 2 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Fluid overload **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Hyperkalaemia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 72 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 144 **Num Events:** 2 **Term:** Hypoglycaemia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 144 **Num Events:** 2 **Term:** Musculoskeletal chest pain **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 72 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 144 **Term:** Rhabdomyolysis **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Cerebrovascular accident **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 72 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Dizziness **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Encephalopathy **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Myasthenia gravis crisis **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Status epilepticus **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 72 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 144 **Term:** Syncope **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 144 **Num Events:** 2 **Term:** Thalamic infarction **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 72 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 144 **Term:** Mental status changes **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 72 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 144 **Term:** Urinary tract obstruction **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Acute pulmonary oedema **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 72 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 144 **Term:** Acute respiratory failure **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Respiratory failure **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 72 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 144 **Term:** Angioedema **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Deep vein thrombosis **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Term:** Hypertensive emergency **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 72 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 144 **Num Events:** 1 **Time Frame:** 6 months from randomization to end of therapy ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 72 **Group ID:** BG001 **Value:** 144 **Group ID:** BG002 **Value:** 216 **Units:** Participants ### Group **ID:** BG000 **Title:** Sugar Pill to CTAP101 30 μg **Description:** 1 sugar pill to CTAP101 30 μg capsule daily at bedtime for 12 weeks, with an increase to 2 sugar pills to CTAP101 30 μg capsules daily at bedtime for weeks 13-26. Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime. ### Group **ID:** BG001 **Title:** CTAP101 30 μg Capsules **Description:** 1 CTAP101 30 μg capsule daily at bedtime for 12 weeks, with a possible increase to 2 CTAP101 30 μg capsules daily at bedtime for study duration, if needed (26 weeks total). Subjects not requiring a dose increase after 12 weeks would receive 1 CTAP101 30 μg capsule and one sugar pill (to CTAP101 30 μg capsule) for weeks 13-26. CTAP101 30 μg capsules: CTAP101 30 μg capsule taken daily at bedtime Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime. ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 10.06 **Value:** 65.3 #### Measurement **Group ID:** BG001 **Spread:** 10.90 **Value:** 66.8 #### Measurement **Group ID:** BG002 **Spread:** 10.63 **Value:** 66.3 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 39 #### Measurement **Group ID:** BG001 **Value:** 71 #### Measurement **Group ID:** BG002 **Value:** 110 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 33 #### Measurement **Group ID:** BG001 **Value:** 73 #### Measurement **Group ID:** BG002 **Value:** 106 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 72 #### Measurement **Group ID:** BG001 **Value:** 144 #### Measurement **Group ID:** BG002 **Value:** 216 **Class Title:** United States **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** participants ## Results Section - More Information Module ### Certain Agreement **Restriction Type:** LTE60 **Restrictive Agreement:** True ### Point of Contact **Email:** [email protected] **Organization:** OPKO Health, Inc. **Phone:** 305-575-4172 **Title:** Douglass Laidlaw, PhD, Vice President, Medical Affairs ## Results Section - Outcome Measures Module ### Outcome Measure 1 #### Analysis **CI Lower Limit:** **CI Number of Sides:** **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** <0.0001 **P-Value Comment:** **Parameter Type:** **Parameter Value:** **Statistical Comment:** **Statistical Method:** Cochran-Mantel-Haenszel **Tested Non-Inferiority:** ### Outcome Measure 2 #### Analysis **CI Lower Limit:** **CI Number of Sides:** **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** <0.0001 **P-Value Comment:** **Parameter Type:** **Parameter Value:** **Statistical Comment:** **Statistical Method:** Cochran-Mantel-Haenszel **Tested Non-Inferiority:** ### Outcome Measure 3 #### Analysis **CI Lower Limit:** **CI Number of Sides:** **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** <0.0001 **P-Value Comment:** **Parameter Type:** **Parameter Value:** **Statistical Comment:** **Statistical Method:** Cochran-Mantel-Haenszel **Tested Non-Inferiority:** ### Outcome Measure 4 #### Analysis **CI Lower Limit:** **CI Number of Sides:** **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** <0.0001 **P-Value Comment:** **Parameter Type:** **Parameter Value:** **Statistical Comment:** **Statistical Method:** Cochran-Mantel-Haenszel **Tested Non-Inferiority:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 49 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 47 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 120 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 116 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Number of subjects in the intent to treat population attaining mean decrease in plasma intact Parathyroid Hormone (iPTH) of ≥30% from P\\pre-treatment baseline in the efficacy assessment phase (EAP) referred to as responders **Parameter Type:** NUMBER **Population Description:** Intent to treat **Reporting Status:** POSTED **Time Frame:** Approximately 6 months **Title:** Number of Participants in the Intent to Treat Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline **Type:** PRIMARY **Unit of Measure:** participants ##### Group **Description:** 1 sugar pill to CTAP101 30 μg capsule daily at bedtime for 12 weeks, with an increase to 2 sugar pills to CTAP101 30 μg capsules daily at bedtime for weeks 13-26. Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime. **ID:** OG000 **Title:** Sugar Pill to CTAP101 30 μg ##### Group **Description:** 1 CTAP101 30 μg capsule daily at bedtime for 12 weeks, with a possible increase to 2 CTAP101 30 μg capsules daily at bedtime for study duration, if needed (26 weeks total). Subjects not requiring a dose increase after 12 weeks would receive 1 CTAP101 30 μg capsule and one sugar pill (to CTAP101 30 μg capsule) for weeks 13-26. CTAP101 30 μg capsules: CTAP101 30 μg capsule taken daily at bedtime Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime. **ID:** OG001 **Title:** CTAP101 30 μg Capsules #### Outcome Measure 2 **Description:** Number of subjects in the per protocol population attaining a mean decrease in plasma intact parathyroid hormone (iPTH) of ≥30% from pre-treatment baseline in the efficacy assessment phase (EAP), referred to as responders **Parameter Type:** NUMBER **Population Description:** Per protocol **Reporting Status:** POSTED **Time Frame:** Approximately 6 months **Title:** Number of Participants in the Per Protocol Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline **Type:** SECONDARY **Unit of Measure:** participants ##### Group **Description:** 1 sugar pill to CTAP101 30 μg capsule daily at bedtime for 12 weeks, with an increase to 2 sugar pills to CTAP101 30 μg capsules daily at bedtime for weeks 13-26. Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime. **ID:** OG000 **Title:** Sugar Pill to CTAP101 30 μg ##### Group **Description:** 1 CTAP101 30 μg capsule daily at bedtime for 12 weeks, with a possible increase to 2 CTAP101 30 μg capsules daily at bedtime for study duration, if needed (26 weeks total). Subjects not requiring a dose increase after 12 weeks would receive 1 CTAP101 30 μg capsule and one sugar pill (to CTAP101 30 μg capsule) for weeks 13-26. CTAP101 30 μg capsules: CTAP101 30 μg capsule taken daily at bedtime Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime. **ID:** OG001 **Title:** CTAP101 30 μg Capsules #### Outcome Measure 3 **Description:** Subjects in the intent to treat population with normal serum total 25-hydroxyvitamin D (\>/= 30 ng/dL) **Parameter Type:** NUMBER **Population Description:** Intent to treat **Reporting Status:** POSTED **Time Frame:** Approximately 6 months **Title:** Subjects in the Intent to Treat Population With Normal Serum Total 25-hydroxyvitamin D **Type:** SECONDARY **Unit of Measure:** participants ##### Group **Description:** 1 sugar pill to CTAP101 30 μg capsule daily at bedtime for 12 weeks, with an increase to 2 sugar pills to CTAP101 30 μg capsules daily at bedtime for weeks 13-26. Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime. **ID:** OG000 **Title:** Sugar Pill to CTAP101 30 μg ##### Group **Description:** 1 CTAP101 30 μg capsule daily at bedtime for 12 weeks, with a possible increase to 2 CTAP101 30 μg capsules daily at bedtime for study duration, if needed (26 weeks total). Subjects not requiring a dose increase after 12 weeks would receive 1 CTAP101 30 μg capsule and one sugar pill (to CTAP101 30 μg capsule) for weeks 13-26. CTAP101 30 μg capsules: CTAP101 30 μg capsule taken daily at bedtime Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime. **ID:** OG001 **Title:** CTAP101 30 μg Capsules #### Outcome Measure 4 **Description:** Subjects in the per protocol population with normal serum total 25-hydroxyvitamin D (\>/= 30 ng/mL) **Parameter Type:** NUMBER **Population Description:** Per protocol **Reporting Status:** POSTED **Time Frame:** Approximately 6 months **Title:** Subjects in the Per Protocol Population With Normal Serum Total 25-hydroxyvitamin D **Type:** SECONDARY **Unit of Measure:** participants ##### Group **Description:** 1 sugar pill to CTAP101 30 μg capsule daily at bedtime for 12 weeks, with an increase to 2 sugar pills to CTAP101 30 μg capsules daily at bedtime for weeks 13-26. Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime. **ID:** OG000 **Title:** Sugar Pill to CTAP101 30 μg ##### Group **Description:** 1 CTAP101 30 μg capsule daily at bedtime for 12 weeks, with a possible increase to 2 CTAP101 30 μg capsules daily at bedtime for study duration, if needed (26 weeks total). Subjects not requiring a dose increase after 12 weeks would receive 1 CTAP101 30 μg capsule and one sugar pill (to CTAP101 30 μg capsule) for weeks 13-26. CTAP101 30 μg capsules: CTAP101 30 μg capsule taken daily at bedtime Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime. **ID:** OG001 **Title:** CTAP101 30 μg Capsules ### Participant Flow Module #### Group **Description:** 1 sugar pill to CTAP101 30 μg capsule daily at bedtime for 12 weeks, with an increase to 2 sugar pills to CTAP101 30 μg capsules daily at bedtime for weeks 13-26. Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime. **ID:** FG000 **Title:** Sugar Pill to CTAP101 30 μg #### Group **Description:** 1 CTAP101 30 μg capsule daily at bedtime for 12 weeks, with a possible increase to 2 CTAP101 30 μg capsules daily at bedtime for study duration, if needed (26 weeks total). Subjects not requiring a dose increase after 12 weeks would receive 1 CTAP101 30 μg capsule and one sugar pill (to CTAP101 30 μg capsule) for weeks 13-26. CTAP101 30 μg capsules: CTAP101 30 μg capsule taken daily at bedtime Sugar pill to CTAP101 30 μg capsules: Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime. **ID:** FG001 **Title:** CTAP101 30 μg Capsules #### Period **Title:** Overall Study ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 5 ###### Reason **Group ID:** FG001 **Number of Subjects:** 7 ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ###### Reason **Group ID:** FG001 **Number of Subjects:** 5 ##### Withdraw **Type:** Adverse Event ###### Reason **Group ID:** FG000 **Number of Subjects:** 4 ###### Reason **Group ID:** FG001 **Number of Subjects:** 7 ##### Withdraw **Type:** Protocol Violation ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ##### Withdraw **Type:** Subject noncompliance ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ###### Reason **Group ID:** FG001 **Number of Subjects:** 3 ##### Withdraw **Type:** Serum Calcium >/= 11.0 mg/dL, repeat con ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 72 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 144 ##### Milestone **Type:** Entered Extension Study ###### Achievement **Group ID:** FG000 **Number of Subjects:** 53 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 102 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 58 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 121 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 14 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 23 **Pre-Assignment Details:** Subjects were stratified by CKD stage and were randomized in a 2:1 ratio to receive a daily 30 μg oral dose of CTAP101 capsules (or matching placebo) for 12 weeks at bedtime. Subjects presenting on a regimen of bone metabolism therapy were to discontinue their prior treatment for at least 28 days' washout (except for bisphosphonates). **Recruitment Details:** This multicenter study was conducted at 45 investigational sites within the US. Subjects were enrolled and treated from 04 February 2013 through 13 August 2014. **Has Results:** True