acronym: InPedILD®

lead_sponsor: Boehringer Ingelheim

summary: The main objective of the study is to evaluate dose-exposure and safety of nintedanib in children and adolescents with fibrosing Interstitial Lung Disease (ILD).

ReferenceCitation: Deterding R, Griese M, Deutsch G, Warburton D, DeBoer EM, Cunningham S, Clement A, Schwerk N, Flaherty KR, Brown KK, Voss F, Schmid U, Schlenker-Herceg R, Verri D, Dumistracel M, Schiwek M, Stowasser S, Tetzlaff K, Clerisme-Beaty E, Young LR. Study design of a randomised, placebo-controlled trial of nintedanib in children and adolescents with fibrosing interstitial lung disease. ERJ Open Res. 2021 Jun 21;7(2):00805-2020. doi: 10.1183/23120541.00805-2020. eCollection 2021 Apr.

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Inserm

repository_normalized: HAL

repository_institution: University of Edinburgh - Edinburgh Research Explorer

repositories:
HAL
PubMed Central
pure.ed.ac.uk

repositories_concat: HAL;PubMed Central;pure.ed.ac.uk

publisher_dissemination: European Respiratory Society

location_facility: Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center

location_facility: Institute of Phthisiology and Pulmonology n. a. F.G.Yanovsky

location_facility: State Novosibirsk Regional Clinical Hospital

location_facility: Teaching Hospital Motol, Oncology Clinic

location_facility: Oslo Universitetssykehus HF, Rikshospitalet

location_facility: Riley Hospital for Children at Indiana University Health

location_facility: Independent Public Teaching Children's Hospital

location_facility: Hospital de Pediatría " Prof. Dr. Juan P. Garrahan"

location_facility: Azienda Ospedaliera Universitaria di Padova

location_facility: Serviços Medicos Respirar Sul Fluminense

location_facility: Centro Hospitalar Universitário de Lisboa Norte, EPE - Hospital Santa Maria

location_facility: Centro de Pesquisa Clinica do Instituto da Crianca - HCFMUSP

location_facility: General Hospital of Thessaloniki "Ippokrateio"

location_facility: St. Petersburg State Pediatric University

location_facility: Children's Hematology,Oncology&Immunology na Rogachev,Moscow

location_facility: Regional Children Clinical Hospital, Pulmonology, Kharkiv

location_facility: Regional clinical children hospital, Zaporizhya

title: A Double Blind, Randomised, Placebo-controlled Trial to Evaluate the Dose-exposure and Safety of Nintedanib Per os on Top of Standard of Care for 24 Weeks, Followed by Open Label Treatment With Nintedanib of Variable Duration, in Children and Adolescents (6 to 17 Year-old) With Clinically Significant Fibrosing Interstitial Lung Disease

ipd_sharing_description:
Clinical studies sponsored by Boehringer Ingelheim, phases I to IV, interventional and non-interventional, are in scope for sharing of the raw clinical study data and clinical study documents, except for the following exclusions:

studies in products where Boehringer Ingelheim is not the license holder;
studies regarding pharmaceutical formulations and associated analytical methods, and studies pertinent to pharmacokinetics using human biomaterials;
studies conducted in a single center or targeting rare diseases (because of limitations with anonymization).

For more details refer to: https://www.mystudywindow.com/msw/datasharing

location_country:
Finland
Poland
Argentina
Australia
Brazil
Czechia
Canada
France
Portugal
Hungary
Denmark
United States
Ukraine
Belgium
Italy
Norway
United Kingdom
Mexico
Spain
Greece
Russian Federation


title: Optimize PRO Transcatheter Aortic Valve Replacement Post Market Study

summary:
The purpose of this study is to collect clinical evidence on valve performance and procedural outcomes associated with an "optimized" TAVR care pathway and post-TAVR conduction disturbance pathway while using the Evolut™ PRO and Evolut™ PRO+ devices.

The purpose of the addendum is to collect post-market clinical evidence on valve performance and procedural outcomes associated with the Evolut FX Device.

study_start_date_type: Actual

status: Active, not recruiting

study_completion_date_type: Anticipated

study_type: Observational

time_perspective: Prospective

enrollment_type: Anticipated

ipd_sharing: No

lead_sponsor: Medtronic Cardiovascular

location_country:
Germany
Ireland
Italy
Israel
United Kingdom
Australia
Sweden
Belgium
United States
Spain
Canada
France

location_facility: University of Alabama at Birmingham Hospital

location_facility: Hôpitaux Universitaires - Hôpital Henri Mondor

location_facility: New York Presbyterian - Columbia University Medical Center

location_facility: Royal Victoria Hospital - Belfast Health and Social Care Trust

location_facility: Hospital Universitario Central de Asturas

location_facility: Laval University Institute of Cardiology and Respirology of Quebec

location_facility: Fondazione Policlinico Universitario Agostino Gemelli

location_facility: California Pacific Medical Center - Sutter Health

location_facility: St. John's Hospital/Prarie Education and Research Cooperative

location_facility: Galway University Hospitals - University Hospital Galway (UHG)

location_facility: Abbott Northwestern Hospital (Minneapolis Heart)

location_facility: Dixie Regional Medical Center Intermountain

location_facility: St. Vincent's Hospital / Cardiology Associates of Fairfield County, P.C.

intervention_type: Device

lead_sponsor_type: industriel

status_simplified: Ongoing


acronym: DEVOTE

intervention_type: Drug

lead_sponsor: Biogen

summary:
The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital of Philadelphia-Infant Test of Neuromuscular Disorders (CHOP-INTEND) total score (Part B); to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A and C).

The secondary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A, B and C); to examine the effect of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A and C); to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA, to examine the effect of nusinersen administered intrathecally at higher doses compared to the currently approved dose in participants with SMA (Part B).

ReferenceType: derived

ReferenceCitation: Finkel RS, Ryan MM, Pascual Pascual SI, Day JW, Mercuri E, De Vivo DC, Foster R, Montes J, Gurgel-Giannetti J, MacCannell D, Berger Z. Scientific rationale for a higher dose of nusinersen. Ann Clin Transl Neurol. 2022 Jun;9(6):819-829. doi: 10.1002/acn3.51562. Epub 2022 May 13.

oa_host_type: repository

evidence: oa repository (via pmcid lookup)

host_type: repository

license_normalized: no license

repository_normalized: PubMed Central

version: publishedVersion

repositories_concat: PubMed Central

unpaywall_oa_status: green

publisher_dissemination: Wiley

location_facility: National Guard Health Affairs: King Abdulaziz Medical City

location_facility: Hospital Universitari i Politecnic La Fe

location_facility: Ann & Robert H. Lurie Children's Hospital of Chicago

location_facility: Magyarorszagi Reformatus Egyhaz Bethesda Gyermekkorhaz

location_facility: Fondazione Serena Onlus - Centro Clinico Nemo

location_facility: General Hospital of Thessaloniki "Hippokration"

location_facility: Guangzhou Woman and Children's Medical Center

location_facility: Hospital das Clínicas da Faculdade de Medicina da USP

location_facility: Kaohsiung Medical University Chung-Ho Memorial Hospital

location_facility: Instytut Pomnik - Centrum Zdrowia Dziecka

location_facility: Children's Hospital Chongqing University of Medical Science

location_facility: Fundacion Hospitalaria San Vicente de Paul

location_facility: McGill University Health Centre/Glen Site/Montreal Children's Hospital

location_facility: HC-UFMG - Hospital das Clinicas da Universidade Federal de Minas Gerais

location_facility: Great Ormond Street Hospital for Children

location_facility: The University of Texas Southwestern Medical Center

location_facility: Universitaetsklinikum Giessen und Marburg GmbH

location_facility: King Faisal Specialist Hospital & Research Center

location_facility: Fondazione Policlinico Universitario Agostino Gemelli IRCCS

location_facility: Xiangya Hospital, Central South University

location_facility: The Second Hospital affiliated to West China Medical University

location_facility: Russian Children Neuromuscular Center of Veltischev

location_facility: Hospital Infantil de Mexico Federico Gomez

location_facility: Kyungpook National University Chilgok Hospital

location_facility: London Health Sciences Centre (LHSC) - Children's Hospital

location_facility: Antiguo Hospital Civil de Guadalajara Fray Antonio Alcalde

location_facility: Saint George University Hospital Medical Center

location_facility: Tokyo Women's Medical University Hospital

title: Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

primary_purpose: Treatment

type: EudraCT Number

source: euctr

enrollment_type: Actual

ipd_sharing_description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

design_allocation: Randomized

location_country:
Germany
Ireland
Poland
Australia
Brazil
Japan
Turkey
Chile
Canada
France
Colombia
Hungary
United States
Latvia
Lebanon
China
Estonia
Italy
United Kingdom
Netherlands
Mexico
Korea, Republic of
Saudi Arabia
Spain
Greece
Taiwan
Russian Federation
Israel

study_type: Interventional

ipd_sharing: Yes


type: U.S. NIH Grant/Contract

title: Sickle Cell Disease and CardiovAscular Risk - Red Cell Exchange Trial (SCD-CARRE)

acronym: SCD-CARRE

summary: The SCD-CARRE trial is a Phase 3, prospective, randomized, multicenter, controlled, parallel two-arm study aimed to determine if automated exchange blood transfusion and standard of care administered to high mortality risk adult SCD patients reduces the total number of episodes of clinical worsening of SCD requiring acute health care encounters (non-elective infusion center/ER/hospital visits) or resulting in death over 12 months as compared with standard of care.

status: Recruiting

ipd_sharing_description: Results from research conducted under this project will be shared in several ways. Manuscripts will be submitted for publication in high-quality peer-reviewed journals, following the NIH Public Access Policy guidelines. Findings will be presented at relevant national conferences, public lectures, scientific institutions and meetings. The timeline for participant recruitment, data collection and analysis will foster publication, facilitated by the investigators' Publications Committee, of critically important and clinically relevant data throughout the trial. The study datasets will be archived and made available to qualified individuals after a period of exclusive use by the SCD-CARRE trial research team and after publication of the primary manuscripts, following NIH guidelines.

lead_sponsor: Gladwin, Mark, MD

collaborators: National Heart, Lung, and Blood Institute (NHLBI)

location_country:
United States
Brazil
France

location_facility: University Hospitals Cleveland Medical Center

location_facility: UCSF Benioff Children's Hospital Oakland

location_facility: Howard University Center for Sickle Cell Disease

location_facility: University of North Carolina at Chapel Hill

location_facility: University of Texas Health Science Center at Houston

intervention_type: Other


title: A Phase 1/2 Open-label, Multicenter Study Evaluating the Safety and Pharmacokinetics of Imvotamab (IGM-2323) as a Single Agent and in Combination in Subjects With Relapsed/Refractory Non-Hodgkin Lymphomas

summary:
This is a Phase 1/2 study of imvotamab in adult subjects with relapsed or refractory B-cell Non-Hodgkin Lymphoma. This study will consist of a dose-escalation stage, a combination stage, and a randomized dose-expansion stage where subjects will be enrolled into indication-specific expansion cohorts. imvotamab will be administered intravenously (IV).

Additional CD20-positive NHL histologies (e.g. MZL and MCL), may be allowed with Medical Monitor approval during the Dose-Escalation Phase of the study.

lead_sponsor: IGM Biosciences, Inc.

sponsor_collaborators:
IGM Biosciences, Inc.
ADC Therapeutics S.A.

location_country:
Italy
Australia
United States
Korea, Republic of
Spain
Czechia
France

location_facility: Azienda Ospedaliero Universitaria di Bologna-Ematologia Bologna

location_facility: Fondazione Policlinico Universitario Agostino Gemelli

location_facility: START-Madrid: Centro Integral Oncologico Clara Campal


title: Antiplatelet Removal and HemocompatIbility EventS With the HeartMate 3 Pump IDE Study

summary: Prospective, randomized, double-blinded, placebo-controlled clinical investigation of advanced heart failure patients treated with the HM3 with two different antithrombotic regimens: vitamin K antagonist with aspirin versus vitamin K antagonist with placebo

status: Completed

study_completion_date_type: Actual

primary_purpose: Supportive Care

ReferenceCitation: Mehra MR, Crandall DL, Gustafsson F, Jorde UP, Katz JN, Netuka I, Uriel N, Connors JM, Sood P, Heatley G, Pagani FD. Aspirin and left ventricular assist devices: rationale and design for the international randomized, placebo-controlled, non-inferiority ARIES HM3 trial. Eur J Heart Fail. 2021 Jul;23(7):1226-1237. doi: 10.1002/ejhf.2275. Epub 2021 Jul 1.

oa_colors: green hybrid

oa_host_type: publisher;repository

evidence: open (via crossref license)

host_type: publisher

license: cc-by-nc

license_normalized: cc-by-nc

evidence: oa repository (via OAI-PMH doi match)

repository_institution: University of Michigan - Deep Blue

repositories_concat: PubMed Central;deepblue.lib.umich.edu

unpaywall_oa_status: hybrid

ipd_sharing_description: To be determined

lead_sponsor: Abbott Medical Devices

location_country:
Italy
United Kingdom
Australia
Austria
United States
Kazakhstan
Czechia
Canada
France

location_facility: California Pacific Medical Center - Van Ness Campus

location_facility: University of Minnesota Medical Center Fairview

location_facility: Miami Transplant Institute - Jackson Memorial

location_facility: National Research Center for Cardiac Surgery

location_facility: Hospital of the University of Pennsylvania

location_facility: New York-Presbyterian/Columbia University Medical Center

location_facility: Montefiore Medical Center - Moses Division

status_simplified: Completed


title: A Phase Ib/II, Open-label, Multicenter Study of Novel Oncology Therapies in Combination With Chemotherapy and Bevacizumab as First-line Therapy in Metastatic Microsatellite-stable Colorectal Cancer (COLUMBIA-1)

summary: COLUMBIA-1 is a Phase 1b/2 platform study to evaluate the safety and efficacy of standard of care (FOLFOX plus bevacizumab) alone and in combination with novel oncology therapies in first-line metastatic microsatellite-stable colorectal cancer (MSS-CRC).

status: Terminated

ipd_sharing_description: Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All request will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

lead_sponsor: MedImmune LLC

location_country:
Australia
United States
Spain
Canada
France

status_simplified: Unknown


type: Other Identifier

title: Extravascular ICD Pivotal Study (EV ICD)

acronym: EV ICD

summary:
Pivotal Summary:

The study is designed to demonstrate safety and efficacy of the Extravascular Implantable Cardioverter Defibrillator (EV ICD) System.

Continued Access Summary:

This study is designed to provide continued access to the Extravascular Implantable Cardioverter Defibrillator (EV ICD) System.

primary_purpose: Other

ReferenceCitation: Friedman P, Murgatroyd F, Boersma LVA, Manlucu J, O'Donnell D, Knight BP, Clementy N, Leclercq C, Amin A, Merkely BP, Birgersdotter-Green UM, Chan JYS, Biffi M, Knops RE, Engel G, Munoz Carvajal I, Epstein LM, Sagi V, Johansen JB, Sterlinski M, Steinwender C, Hounshell T, Abben R, Thompson AE, Wiggenhorn C, Willey S, Crozier I; Extravascular ICD Pivotal Study Investigators. Efficacy and Safety of an Extravascular Implantable Cardioverter-Defibrillator. N Engl J Med. 2022 Oct 6;387(14):1292-1302. doi: 10.1056/NEJMoa2206485. Epub 2022 Aug 28.

oa_colors: green other

evidence: open (via free article)

repository_institution: University of Southern Denmark - University of Southern Denmark Research Portal

unpaywall_oa_status: bronze

publisher_dissemination: Massachusetts Medical Society

ReferenceCitation: Crozier I, O'Donnell D, Boersma L, Murgatroyd F, Manlucu J, Knight BP, Birgersdotter-Green UM, Leclercq C, Thompson A, Sawchuk R, Willey S, Wiggenhorn C, Friedman P. The extravascular implantable cardioverter-defibrillator: The pivotal study plan. J Cardiovasc Electrophysiol. 2021 Sep;32(9):2371-2378. doi: 10.1111/jce.15190. Epub 2021 Aug 5.

license: cc-by

license_normalized: cc-by

evidence: oa repository (via OAI-PMH title and first author match)

repository_institution: Authorea - Authorea

repository_normalized: Authorea - Authorea

version: submittedVersion

repositories:
Authorea - Authorea
PubMed Central

repositories_concat: Authorea - Authorea;PubMed Central

lead_sponsor: Medtronic Cardiac Rhythm and Heart Failure

location_country:
Italy
Poland
New Zealand
Hungary
Hong Kong
Denmark
Australia
Norway
Netherlands
Austria
Switzerland
United Kingdom
United States
Saudi Arabia
Spain
Canada
France

location_facility: London Health Sciences Centre - University Campus

location_facility: AdventHealth Cardiovascular Research Institute

location_facility: Institut Universitaire de Cardiologie et de Pneumologie de Québec (IUCPQ)

location_facility: Continued Access: University of Minnesota Medical Center Fairview

location_facility: Continued Access: North Shore University Hospital

location_facility: Kepler Universitätsklinikum Med Campus III.

location_facility: University of California San Diego (UCSD)

location_facility: University of Washington (UW) Medical Center

location_facility: Narodowy Instytut Kardiologii - Stefana kardynała Wyszyńskiego

location_facility: Continued Access: OhioHealth Research and Innovation Institute (OHRI)

location_facility: Continued Access: Lourdes Cardiology Services

location_facility: Continued Access: Medstar Washington Hospital Center

location_facility: Continued Access: Saint Luke's Mid America Heart Institute

location_facility: The College of Medicine & King Khalid University Hospital, King Saud University

location_facility: Continued Access: University Hospitals Cleveland Clinic Medical Center

location_facility: Prairie Education & Research Cooperative

location_facility: Continued Access: New York-Presbyterian Hospital/Weill Cornell Medical Center

location_facility: HonorHealth Cardiac Arrhythmia Group - Osborn


acronym: FREESIA

lead_sponsor: Janssen Sciences Ireland UC

summary: The purpose of this study is to evaluate the effect of JNJ-53718678 on the development of respiratory syncytial virus (RSV) lower respiratory tract infection (LRTIs) in adult hematopoietic stem cell transplant (HSCT) recipients with RSV upper RTI.

location_facility: Hospital Privado-Universitario de Cordoba

location_facility: Centro de Pesquisa e Ensino em Oncologia de Santa Catarina - CEPEN

location_facility: University Hospitals Cleveland Medical Center

location_facility: Sociedade Beneficente de Senhoras - Hospital Sírio Libanês

location_facility: Ematologia Fondazione Univ. Policlinico Gemelli Università Cattolica del Sacro Cuore

location_facility: Real e Benemérita Associação Portuguesa de Beneficência

location_facility: Multiprofile Hospital for Active Treatment 'Sveta Marina' EAD

location_facility: Universidade Federal De Minas Gerais - Hospital das Clínicas

location_facility: Henry Ford Hospital - Hematology/oncology

location_facility: Universidade Federal do Ceara - Hospital Universitario Walter Cantidio

location_facility: Ospedale San Raffaele HSR Istituto Scientifico Universitario San Raffaele

location_facility: Fundação Antônio Prudente - A.C. Camargo Cancer Center

location_facility: Specialized Hospital for Active Treatment of Haematologic Diseases

location_facility: The Catholic University of Korea, Seoul St. Mary's Hospital

location_facility: Institut Universitaire du Cancer Toulouse - Oncopole

location_facility: Japanese Red Cross Society Nagano Hospital

location_facility: Fundação Faculdade Regional de Medicina de São José do Rio Preto - Hospital de Base

location_facility: MD Anderson Cancer Center - University of Texas

title: A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Clinical Outcomes, Antiviral Activity, Safety, Tolerability, Pharmacokinetics, and Pharmacokinetics/Pharmacodynamics of JNJ-53718678 in Adult and Adolescent Hematopoietic Stem Cell Transplant Recipients With Respiratory Syncytial Virus Infection of the Upper Respiratory Tract

ipd_sharing_description: The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale open Data Access (YODA) Project site at yoda.yale.edu

location_country:
Italy
Israel
Argentina
United Kingdom
Australia
Brazil
Japan
Netherlands
Belgium
Sweden
United States
Korea, Republic of
Spain
Malaysia
Taiwan
Bulgaria
France


acronym: STEADFAST

lead_sponsor: Novartis Pharmaceuticals

summary: The goal of the study is to evaluate descriptively the effect of crizanlizumab + standard of care and standard of care alone on renal function in sickle cell disease patients ≥ 16 years with chronic kidney disease due to sickle cell nephropathy.

location_facility: East Carolina University BrodySchool of Med. (3)

location_facility: University of Texas Health Science Center at Houston

location_facility: Our Lady of the Lake Regional Medical Center

location_facility: University of Illinois Hospital and Health Sciences System

title: A Phase II, Multicenter, Randomized, Open Label Two Arm Study Evaluating the Effect of Crizanlizumab + Standard of Care and Standard of Care Alone on Renal Function in Sickle Cell Disease Patients ≥ 16 Years With Chronic Kidney Disease Due to Sickle Cell Nephropathy (STEADFAST)

ipd_sharing_description:
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.

location_country:
Ireland
United Kingdom
Brazil
Netherlands
Lebanon
United States
Spain
Greece
Panama
Turkey
France


type: Registry Identifier

title: A Multi-center, Open-label, Phase 1b Study to Assess the Pharmacokinetics, Safety, and Efficacy of Subcutaneous and Intravenous Isatuximab (SAR650984) in Combination With Pomalidomide and Dexamethasone, in Patients With Relapsed/Refractory Multiple Myeloma (RRMM)

summary:
Primary Objectives:

To evaluate the safety and tolerability of isatuximab administered subcutaneously (SC) versus intravenously (IV)
To assess the safety and tolerability (including local injection site tolerability) of isatuximab using the (investigational) isatuximab injector device
To evaluate the pharmacokinetics (PK) of SC and IV isatuximab

Secondary Objectives:

To estimate absolute bioavailability of SC and IV isatuximab
To measure receptor occupancy (RO) after isatuximab SC versus IV administration
To assess efficacy of isatuximab after SC and IV administration
To assess patient expectations prior to and patient experience and satisfaction after SC administration
To evaluate potential immunogenicity of SC or IV isatuximab

ipd_sharing_description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

lead_sponsor: Sanofi

location_country:
Australia
Japan
Belgium
United States
Spain
France

location_facility:
Investigational Site Number :2500002
Investigational Site Number :0360001
Investigational Site Number :0360004
Investigational Site Number :3920001
Investigational Site Number :7240001
Investigational Site Number :8400005
Investigational Site Number :0560001
Investigational Site Number :8400002
Investigational Site Number :2500001
Investigational Site Number :0360002
Investigational Site Number :7240003
Investigational Site Number :8400001
Investigational Site Number :3920002
Investigational Site Number :0360003
Investigational Site Number :7240002


title: An Open-Label Roll-Over Study to Evaluate the Long-Term Safety and Efficacy of DCR-PHXC Solution for Injection (Subcutaneous Use) in Patients With Primary Hyperoxaluria

summary: The proposed study is designed to provide patients previously enrolled in Phase 1 and 2 studies of DCR-PHXC and their siblings (<18 years old) long-term access to DCR-PHXC, and to evaluate the long-term safety and efficacy of DCR-PHXC in patients with PH.

status: Enrolling by invitation

lead_sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

location_country:
Germany
Italy
Norway
United Kingdom
Australia
Japan
Netherlands
Lebanon
United States
Spain
Turkey
Canada
France

location_facility:
Clinical Research Site
Clinical Trial Site


acronym: UNITE

lead_sponsor: Teva Branded Pharmaceutical Products R&D, Inc.

summary:
The primary objective is to evaluate the efficacy of monthly 225 mg sc fremanezumab in adult participants with migraine and major depressive disorder (MDD)

The secondary objectives are to evaluate the efficacy of monthly 225 mg sc of fremanezumab in adult participants with migraine and MDD on the reduction of MDD symptoms, responder rates in monthly migraine days, improving quality of life, improving disability, and the safety and tolerability of monthly 225 mg sc and quarterly 675 mg sc fremanezumab in adult participants with migraine and MDD.

The total duration of participant participation in the study is planned to be approximately 28 weeks.

location_facility:
Teva Investigational Site 63075
Teva Investigational Site 30234
Teva Investigational Site 40056
Teva Investigational Site 14331
Teva Investigational Site 14333
Teva Investigational Site 80175
Teva Investigational Site 40058
Teva Investigational Site 40055
Teva Investigational Site 50482
Teva Investigational Site 14332
Teva Investigational Site 50481
Teva Investigational Site 40057
Teva Investigational Site 53448
Teva Investigational Site 14336
Teva Investigational Site 50480
Teva Investigational Site 31272
Teva Investigational Site 32737
Teva Investigational Site 30237
Teva Investigational Site 53447
Teva Investigational Site 35265
Teva Investigational Site 30236
Teva Investigational Site 35267
Teva Investigational Site 14340
Teva Investigational Site 14343
Teva Investigational Site 14339
Teva Investigational Site 14329
Teva Investigational Site 58321
Teva Investigational Site 80172
Teva Investigational Site 53445
Teva Investigational Site 32732
Teva Investigational Site 54184
Teva Investigational Site 63076
Teva Investigational Site 54183
Teva Investigational Site 50483
Teva Investigational Site 54186
Teva Investigational Site 30242
Teva Investigational Site 58319
Teva Investigational Site 30235
Teva Investigational Site 14411
Teva Investigational Site 34254
Teva Investigational Site 14345
Teva Investigational Site 14330
Teva Investigational Site 80173
Teva Investigational Site 54190
Teva Investigational Site 30232
Teva Investigational Site 63077
Teva Investigational Site 31273
Teva Investigational Site 14342
Teva Investigational Site 31274
Teva Investigational Site 32733
Teva Investigational Site 14337
Teva Investigational Site 54185
Teva Investigational Site 14335
Teva Investigational Site 14341
Teva Investigational Site 32736
Teva Investigational Site 32734
Teva Investigational Site 14338
Teva Investigational Site 53446
Teva Investigational Site 58320
Teva Investigational Site 32731
Teva Investigational Site 31276
Teva Investigational Site 80178
Teva Investigational Site 80177
Teva Investigational Site 14334

title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study Followed by an Open-Label Extension to Evaluate the Efficacy and Safety of Fremanezumab for Preventive Treatment of Migraine in Patients With Major Depressive Disorder

primary_purpose: Prevention

ipd_sharing_description: Qualified researchers may request access to patient level data and related study documents including the study protocol and the statistical analysis plan. Requests will be reviewed for scientific merit, product approval status, and conflicts of interest. Patient level data will be de-identified and study documents will be redacted to protect the privacy of trial participants and to protect commercially confidential information. Please email USMedInfo@tevapharm.com to make your request.

location_country:
Germany
Finland
Italy
Poland
Israel
United Kingdom
United States
Spain
Greece
Czechia
Ukraine
Russian Federation
France


title: A Phase 3, Multicenter, Double-blind, Randomized, Placebo-controlled Clinical Study to Evaluate the Safety and Efficacy of Ertugliflozin (MK-8835/PF-04971729) in Pediatric Participants (Ages 10 to 17 Years, Inclusive) With Type 2 Diabetes Mellitus

summary: This study will evaluate the safety and efficacy of ertugliflozin (MK-8835) in pediatric participants with T2DM on metformin with/without insulin. The primary hypothesis of the study is that the addition of ertugliflozin reduces hemoglobin A1C (HbA1C) more than the addition of placebo after 24 weeks of treatment.

lead_sponsor: Merck Sharp & Dohme LLC

sponsor_collaborators:
Merck Sharp & Dohme LLC
Pfizer

location_country:
Poland
Costa Rica
Turkey
Mauritius
Canada
France
Colombia
Hungary
United States
Dominican Republic
Ukraine
Belgium
Malaysia
Philippines
Italy
Guatemala
United Kingdom
Mexico
United Arab Emirates
Saudi Arabia
Russian Federation
Israel

location_facility: Centro de Atencion e Investigacion Clinica SC ( Site 1009)

location_facility: Centro De Diabetes Cardiovascular IPS Ltda ( Site 0101)

location_facility: Chelsea and Westminster Hospital ( Site 2103)

location_facility: Southern Endocrinology and Associates PA ( Site 2218)

location_facility: Private Practice - Dr. Flor de Maria Ranchos Monterroso ( Site 0502)

location_facility: The Children's Hospital of Philadelphia ( Site 2205)

location_facility: MHI Regional Childrens Clinical Hospital ( Site 1908)

location_facility: Consultorio Medico de Endocrinologia Pediatrica ( Site 1002)

location_facility: Ankara Bilkent Şehir Hastanesi-Çocuk Hastanesi, Çocuk Endokrinoloji ( Site 2407)

location_facility: Memorial Regional Hospital-Joe DiMaggio Children's Hospital Division of Pediatric Endocrinology ( Si

location_facility: Rostov Scientific Research Institution of Obstetrics and Pediatry ( Site 1606)

location_facility: MedPlus Medicina Prepagada S.A. ( Site 0102)

location_facility: Vinnitsa Regional Endocrinology Dispensary, VNMU n.a. M.I.Pyrogov ( Site 1901)

location_facility: Semmelweis Egyetem II. sz. Gyermekgyogyaszati Klinika ( Site 0703)

location_facility: King Salman bin Abdulaziz hospital Al Riyadh ( Site 1710)

location_facility: Kazan State Medical University ( Site 1601)

location_facility: Hospital Infantil Dr. Robert Reid Cabral ( Site 0300)

location_facility: Center of Excellence in Diabetes and Endocrinology ( Site 2203)

location_facility: Royal London Hospital (Whitechapel) ( Site 2100)

location_facility: King Abdulaziz Medical City - Al Ahsa ( Site 1730)

location_facility: Samara City Pediatric Clinical Hospital n.a. N.N. Ivanova ( Site 1610)

location_facility: Unidad de Investigacion Clinica Cardiometabolica de Occidente ( Site 1007)

location_facility: Coastal Children''s Services ( Site 2202)

location_facility: U.O. di Diabetologia dell'Eta Evolutiva - AUSL 2 ( Site 0904)

location_facility: Pecsi Tudomanyegyetem Klinikai Kozpont Gyermekgyogyaszati Klinika ( Site 0708)

location_facility: London Health Sciences Centre ( Site 0002)

location_facility: Instytut Diabetologii Sp z o o ( Site 1512)

location_facility: Marmara Üniversitesi Prof. Dr. Asaf Ataseven Hospital ( Site 2400)

location_facility: Siberian State Medical University ( Site 1602)

location_facility: Al Jalila Children s Specialty Hospital ( Site 2004)

location_facility: Heim Pal Orszagos Gyermekgyogyaszati Intezet ( Site 0702)

location_facility: Békés Megyei Központi Kórház Dr. Réthy Pál Tagkórház-Gyermekosztály ( Site 0705)

location_facility: Hospital Universiti Sains Malaysia ( Site 1102)

location_facility: Centro de Estudios de Investigacion Metabolicos y Cardiovasculares ( Site 1003)

location_facility: West Visayas State University Medical Center ( Site 1401)

location_facility: St.Petersburg State Pediatric Medical University ( Site 1600)

location_facility: King Abdul Aziz Medical City - AlRiyadh ( Site 1700)

location_facility: Rashid Center For Diabetes and Research ( Site 2006)

location_facility: Petz Aladar Megyei Oktato Korhaz ( Site 0709)

location_facility: Poradnia Chorob Metabolicznych. Centrum Zdrowia Tuchow ( Site 1500)

location_facility: Hopital Maisonneuve-Rosemont CIUSSS de l Est de L Ile de Montreal ( Site 0001)

location_facility: Cliniques Universitaires Saint-Luc ( Site 2300)

location_facility: Mustafa Al Qaysi Medical Centre ( Site 2010)

location_facility: IRCCS Ospedale Pediatrico Bambino Gesu ( Site 0903)

location_facility: Thumbay University Hospital ( Site 2001)

location_facility: Institute of Children and Adolescents Health Care of the Academy of Medical Sciences ( Site 1915)

location_facility: Institute for Studies on Diabetes Foundation Inc. ( Site 1402)

location_facility: Children's City Clinical Hospital #1 ( Site 1604)

location_facility: I. U. Cerrahpasa Tip Fakultesi ( Site 2406)

location_facility: Bashkir State Medical University Hospital ( Site 1603)

location_facility: Clinical Medical Research Sp. z o.o. ( Site 1511)

location_facility: Borsod-Abauj-Zemplen Megyei Korhaz es Egyetemi OktatoKorhaz ( Site 0701)

location_facility: Bio Investigación AMARC, S.C. ( Site 1006)

location_facility: Centro de Investigacion Medica Aguascalientes ( Site 1000)

location_facility: Federal State Budget Institution Endocrinological Research Center ( Site 1611)

location_facility: University Malaya Medical Centre ( Site 1100)

location_facility: The University of Alabama at Birmingham ( Site 2207)

location_facility: Chernivtsi Regional Children Clinical Hospital No. 1-Department of Pediatrics and Medical Genetics (

location_facility: King Abdul Aziz Medical City. National Guard Health Affairs ( Site 1715)

location_facility: Vita Verum Medical Egeszsegugyi Szolgaltato Bt ( Site 0706)

location_facility: Consultorio Privado Dr. Geraldine Utrilla ( Site 0501)

location_facility: Unidad Biomedica Avanzada Monterrey S. A. ( Site 1005)

location_facility: Ukr Center of Endocrine Surgery and Transplatation MOH Ukraine ( Site 1903)

location_facility: Voronezh State Medical University named after N.N.Burdenko ( Site 1608)

location_facility: Unidad de Medicina Especializada SMA ( Site 1004)

location_facility: Odessa Regional Children Clinical Hospital ( Site 1912)

location_facility: West Middlesex University Hospital ( Site 2104)

location_facility: King Abdul Aziz Medical City - AlRiyadh ( Site 1705)

location_facility: SI Dnipropetrovsk Regional Children Clinical Hospital DOR ( Site 1914)

location_facility: Centro de Investigacion Medica de Occidente S.C. ( Site 1001)

location_facility: CAIMED Investigación en Salud S.A de C.V ( Site 1008)

location_facility: The Edmond and Lily Safra Children s Hospital ( Site 0804)

location_facility: Cukurova Uni. Tip Fakultesi ( Site 2403)

location_facility: Institute of Endocrinology and Metabolism n.a. Komissarenko ( Site 1905)

location_facility: ICCT Research International, Inc. ( Site 2211)

location_facility: King Salman bin Abdulaziz hospital - Al Riyadh ( Site 1720)

location_facility: Szabolcs Szatmar Bereg Megyei Korhazak es Egyetemi Oktato korhaz ( Site 0704)

location_facility: Soroka University Medical Center ( Site 0802)

location_facility: Children's Hospital - Los Angeles ( Site 2201)


title: A Phase 2 Study of TAS-120 in Metastatic Breast Cancers Harboring Fibroblast Growth Factor Receptor (FGFR) Amplifications

summary: The purpose of the trial is to evaluate a patient's response to a Fibroblast Growth Factor Receptor (FGFR) inhibitor, futibatinib (TAS-120), used either alone or in combination with the hormonal therapy, fulvestrant. This study will be conducted in patients with metastatic breast cancer who have specific Fibroblast Growth Factor Receptor gene abnormalities and who have previously received conventional therapies to treat their breast cancer, or who are not able to tolerate certain cancer therapies. This study will also evaluate the safety of taking futibatinib, or futibatinib and fulvestrant, by learning about the potential side effects.

design_allocation: Non-Randomized

lead_sponsor: Taiho Oncology, Inc.

location_country:
Italy
Portugal
United Kingdom
United States
Spain
Canada
France

location_facility: Instituto Portugues de Oncologia do Porto

location_facility: Centro Hospitalar Universitario Lisboa Norte

location_facility: Fondazione Policlinico Universitario Agostino Gemelli IRCCS

location_facility: Azienda Ospedaliero Universitaria Pisana


title: A Multicenter, Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Bimekizumab in the Treatment of Subjects With Active Psoriatic Arthritis

acronym: BE VITAL

summary: This is a study to assess the long-term safety, long-term efficacy and tolerability of bimekizumab administered subcutaneously (sc) in adult subjects with psoriatic arthritis (PsA).

ipd_sharing_description: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.

lead_sponsor: UCB Biopharma SRL

location_country:
Germany
Italy
Poland
Hungary
United Kingdom
Australia
Japan
Belgium
United States
Spain
Czechia
Canada
Russian Federation
France


title: Master Protocol to Assess the Safety and Antitumor Activity of Genetically Engineered NY-ESO-1-Specific (c259) T Cells, Alone or in Combination With Other Agents, in HLA-A2+ Participants With NY-ESO-1 and/or LAGE-1a Positive Solid Tumors (IGNYTE-ESO)

acronym: IGNYTE-ESO

summary: This trial will evaluate safety and efficacy of human engineered T-cell therapies, in participants with advanced tumors.

ipd_sharing_description: IPD for this study will be made available via the Clinical Study Data Request site.

lead_sponsor: GlaxoSmithKline

location_country:
Italy
United Kingdom
Netherlands
United States
Spain
Canada
France


title: Multicenter, Open-label, Safety and Tolerability Study of Ascending Doses of HepaStem in Patients With Cirrhotic and Pre-cirrhotic Non-alcoholic Steato-hepatitis (NASH)

acronym: PANASH

summary: Multicenter, open-label, safety and tolerability study of ascending doses of HepaStem in patients with cirrhotic and pre-cirrhotic non-alcoholic steato-hepatitis (NASH) to determine the safety and tolerability of ascending single and repeated doses of HepaStem administered to patients with cirrhotic and pre-cirrhotic non-alcoholic steato-hepatitis (NASH)

lead_sponsor: Cellaion SA

location_country: Spain Bulgaria Belgium France

location_facility: Hospital General Universitario Gregorio Marañon

location_facility: Multiprofile hospital for active treatment (MHAT) Sofia Military Medical Academy

location_facility: University Multiprofile Hospital for Active Treatment "Tsaritsa Yoana - ISUL"


summary: This is an open-label extension study to evaluate the long-term efficacy, safety and tolerability of LNP023 in subjects with C3 glomerulopathy

title: An Open-label, Non-randomized Extension Study to Evaluate the Long-term Efficacy, Safety and Tolerability of LNP023 in Subjects With C3 Glomerulopathy

ipd_sharing_description: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.

location_country:
Germany
Italy
Israel
Argentina
United Kingdom
Japan
Brazil
Netherlands
Switzerland
United States
China
Spain
Greece
France


acronym: BE MOBILE 2

summary: The purpose of the study is to demonstrate the efficacy, safety and tolerability of bimekizumab administered subcutaneously (sc) compared to placebo in the treatment of subjects with active ankylosing spondylitis (AS).

ReferenceType: result

ReferenceCitation: van der Heijde D, Deodhar A, Baraliakos X, Brown MA, Dobashi H, Dougados M, Elewaut D, Ellis AM, Fleurinck C, Gaffney K, Gensler LS, Haroon N, Magrey M, Maksymowych WP, Marten A, Massow U, Oortgiesen M, Poddubnyy D, Rudwaleit M, Shepherd-Smith J, Tomita T, Van den Bosch F, Vaux T, Xu H. Efficacy and safety of bimekizumab in axial spondyloarthritis: results of two parallel phase 3 randomised controlled trials. Ann Rheum Dis. 2023 Apr;82(4):515-526. doi: 10.1136/ard-2022-223595. Epub 2023 Jan 17. Erratum In: Ann Rheum Dis. 2023 Sep;82(9):e213.

evidence: open (via page says license)

repository_institution: Bielefeld University - PUB – Publications at Bielefeld University

repository_institution: DANS - Data Archiving and Networked Services - NARCIS - National Academic Research and Collaborations Information System

repository_institution: University of East Anglia - UEA Digital Repository

version: acceptedVersion

publisher_dissemination: British Medical Journal

title: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Efficacy and Safety of Bimekizumab in Subjects With Active Ankylosing Spondylitis

location_country:
Germany
Poland
Hungary
United Kingdom
Japan
Netherlands
Belgium
United States
China
Spain
Turkey
Czechia
Bulgaria
France


acronym: BE MOBILE 1

summary: The purpose of the study is to demonstrate the efficacy, safety and tolerability of bimekizumab administered subcutaneously (sc) compared to placebo in the treatment of subjects with active nonradiographic axial spondyloarthritis (nr-axSpA).

repositories_institution: Bielefeld University - PUB – Publications at Bielefeld University

repositories_institution: DANS - Data Archiving and Networked Services - NARCIS - National Academic Research and Collaborations Information System

repositories_institution: University of East Anglia - UEA Digital Repository

title: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Efficacy and Safety of Bimekizumab in Subjects With Active Nonradiographic Axial Spondyloarthritis

location_country:
Germany
Poland
Hungary
United Kingdom
Japan
Belgium
United States
China
Spain
Turkey
Czechia
Bulgaria
France


title: PEACE (Pegzilarginase Effect on Arginase 1 Deficiency Clinical Endpoints): A Randomized, Double-blind, Placebo-controlled Phase 3 Study of the Efficacy and Safety of Pegzilarginase in Children and Adults With Arginase 1 Deficiency

summary: CAEB1102-300A is a multi-center randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of pegzilarginase in patients with ARG1-D. This study will consist of a screening period; a randomized, double-blind treatment period; a long-term extension; and a follow up visit for final safety assessments.

lead_sponsor: Aeglea Biotherapeutics

location_country:
Germany
Italy
United Kingdom
Austria
United States
Canada
France

location_facility: Ann & Robert H. Lurie Children's Hospital of Chicago

location_facility: Azienda Ospedaliera Città della Salute e della Scienza di Torino

location_facility: University of Florida College of Medicine

location_facility: Great Ormond Street Hospital for Children

location_facility: University of Texas Health Science Center Medical School at Houston

location_facility: Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz

location_facility: Cohen Children's Medical Center (Northwell Health)


acronym: ILLUMINATE

lead_sponsor: ProQR Therapeutics

summary: The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects with Leber's Congenital Amaurosis (LCA) due to the CEP290 p.Cys998X mutation after 24 months of treatment

location_facility: Amsterdam University Medica Center - Locatie AMC

location_facility: McGill University Health Centre - Centre for Innovative Medicine

location_facility: INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte

location_facility: Moorfields Eye Hospital - NHS Foundation Trust

location_facility: Justus-Liebig Universität - Department of Ophthalmology

location_facility: Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP)

location_facility: The Hospital for Sick Children - SickKids

location_facility: University of Tuebingen - Inst. for Ophthalmic Research

location_facility: Centre de maladies rares CHNO des Quinze Vingt

location_facility: Eye Clinic University of Campania Luigi Vanvitelli

title: Double-masked, Randomized, Controlled, Multiple-dose Study to Evaluate Efficacy, Safety, Tolerability and Syst. Exposure of QR-110 in Leber's Congenital Amaurosis (LCA) Due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

location_country:
Germany
Italy
United Kingdom
Brazil
Netherlands
Belgium
United States
Canada
France


title: Expanded Access for the Treatment of Cancers With Rearranged During Transfection (RET) Activation

summary:
Expanded access for participants with cancer with RET activation who are ineligible for an ongoing selpercatinib (also known as LOXO-292) clinical trial or have other considerations that prevent access to selpercatinib through an existing clinical trial.

The treating physician/investigator contacts Lilly when, based on their medical opinion, a patient meets the criteria for inclusion in the expanded access program.

status: Available

study_type: Expanded Access

lead_sponsor: Loxo Oncology, Inc.

sponsor_collaborators:
Loxo Oncology, Inc.
Eli Lilly and Company

location_country:
Germany
France
Italy
Poland
New Zealand
Singapore
Australia
Japan
Switzerland
United States
Spain
Israel

location_facility: Queensland Health - Metro North Hospital and Health Service

location_facility: Azienda Ospedaliero Universitaria Pisana

location_facility: ASST Grande Ospedale Metropolitano Niguarda Comitato Etico Milano Area C

location_facility: University of Wisconsin-Madison Hospital and Health Clinic

location_facility: University of Chicago Medicine-Comprehensive Cancer Center

location_facility: NSW Health - Sydney Local Health District

location_facility: Soroka Medical Center - Pediatric Outpatient Clinic

location_facility: Centrum Onkologii Instytut im Marii Curie w Warszawie

location_facility: Institut Bergonié - Centre Régional de Lutte Contre Le Cancer de Bordeaux et Sud Ouest


acronym: BE OPTIMAL

summary: This is a study to demonstrate the clinical efficacy, safety and tolerability of bimekizumab administered subcutaneously (sc) compared with placebo in the treatment of subjects with active Psoriatic Arthritis (PsA).

ReferenceCitation: Ritchlin CT, Coates LC, McInnes IB, Mease PJ, Merola JF, Tanaka Y, Asahina A, Gossec L, Gottlieb AB, Warren RB, Ink B, Bajracharya R, Shende V, Coarse J, Landewe RB. Bimekizumab treatment in biologic DMARD-naive patients with active psoriatic arthritis: 52-week efficacy and safety results from the phase III, randomised, placebo-controlled, active reference BE OPTIMAL study. Ann Rheum Dis. 2023 Nov;82(11):1404-1414. doi: 10.1136/ard-2023-224431. Epub 2023 Sep 11.

title: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Active Reference (Adalimumab) Study Evaluating the Efficacy and Safety of Bimekizumab in the Treatment of Subjects With Active Psoriatic Arthritis

ipd_sharing_description: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.


title: An Extension Trial to Evaluate the Long-term Safety and Efficacy of Bimatoprost SR in Patients With Open Angle Glaucoma or Ocular Hypertension

acronym: Maia

summary: This study will evaluate the long-term safety and efficacy of Bimatoprost Sustained Release (SR) in patients with open-angle glaucoma or ocular hypertension who completed 1 of the 4 Phase 3 Bimatoprost SR studies (192024-091, -092, -093, or -095) and received Bimatoprost SR or who received commercial DURYSTA (Bimatoprost SR) in the open-label Phase 4 ARGOS study (MED-MA-EYE-0648) and completed (or exited early from) the study.

ipd_sharing_description: AbbVie is committed to responsible data sharing regarding the clinical trials we sponsor. This includes access to anonymized, individual and trial-level data (analysis data sets), as well as other information (e.g., protocols, analyses plans, clinical study reports), as long as the trials are not part of an ongoing or planned regulatory submission. This includes requests for clinical trial data for unlicensed products and indications.

lead_sponsor: AbbVie

location_country:
Germany
Poland
New Zealand
Argentina
Brazil
Thailand
Turkey
Czechia
Canada
France
Colombia
Hong Kong
Denmark
United States
Belgium
Peru
Philippines
Egypt
Italy
United Kingdom
Singapore
Korea, Republic of
Spain
South Africa
Russian Federation
Israel

location_facility: The Chinese University of Hong Kong /ID# 240283

location_facility: Azienda Ospedaliera di Perugia /ID# 240310

location_facility: Wolstan & Goldberg Eye Associates /ID# 240221

location_facility: Azienda Ospedaliero Universitaria Pisana-Stabilimento di Santa Chiara /ID# 240301

location_facility: Bostan Diagnostic Eye Center /ID# 240268

location_facility: Klinika Diagnostyki i Mikrochirurgii Jaskry /ID# 240258

location_facility: Marmara Universitesi Pendik Egitim ve Arastirma Hastanesi /ID# 240296

location_facility: Albemarle Clinical Trials LLC /ID# 240219

location_facility: Medical Center Ophthalmology Associates /ID# 240208

location_facility: Tel Aviv Sourasky Medical Center /ID# 240311

location_facility: Hospital Universitario Reina Sofia /ID# 240322

location_facility: Hospital Universitario Austral /ID# 240331

location_facility: Eye Associates of New Mexico /ID# 240216

location_facility: Ophthalmic Consultants Long Island /ID# 240211

location_facility: Ophthalmic Consultant Centres /ID# 240205

location_facility: Eye Associates of North Jersey /ID# 250445

location_facility: Midwest Vision Research Foundation at Pepose Vision Institute /ID# 250402

location_facility: Clinica de Oftalmologia Sandiego /ID# 240233

location_facility: Rochester Ophthalmological Group PC /ID# 240290

location_facility: A.O.U. Policlinico G. Rodolico S.Marco- Presidio G.Rodolico /ID# 240305

location_facility: Hospital Clinico Universitario San Carlos /ID# 240303

location_facility: Pretoria Eye Institute - Private Practice /ID# 240223

location_facility: Scott and Christie and Associates /ID# 240189

location_facility: Logan Ophthalmic Research Inc. /ID# 240280

location_facility: Arizona Glaucoma Specialists /ID# 240212

location_facility: Thammasat University Hospital /ID# 240231

location_facility: Hospital Universitario Virgen Macarena /ID# 240325

location_facility: International Research Center /ID# 240346

location_facility: Universitaetsklinikum Magdeburg /ID# 240314

location_facility: North Bay Eye Associates Inc. /ID# 248912

location_facility: Ain Shams University Hospital /ID# 240338

location_facility: Eye Associates of Fort Meyers /ID# 240218

location_facility: S. Fyodorov Eye Microsurgery Federal State Institution /ID# 240251

location_facility: The Clinical Trials Network (CTNx) /ID# 240284

location_facility: Levenson Eye Associates Inc. /ID# 240209

location_facility: CHU Bordeaux - Hopital Pellegrin /ID# 240316

location_facility: Peregrine Eye and Laser Institute /ID# 240260

location_facility: Eskisehir Osmangazi University Medical School /ID# 240225

location_facility: Fundacion Oftalmologica Nacional /ID# 240226

location_facility: Clinical ophthalmology hospital n.a. V.P. Vihodtsev. /ID# 240253

location_facility: Clinical Eye Research of Boston LLC /ID# 240203

location_facility: Nature Coast Clinical Research - Crystal River /ID# 240334

location_facility: Centrum Diagnostyki i Mikrochirurgii Oka - LENS sp. z o.o /ID# 240248

location_facility: Centre Ophtalmologique Pôle Vision /ID# 240256

location_facility: Charlotte Eye,Ear,Nose Throat /ID# 240339

location_facility: The Lady Davis Carmel Medical Center /ID# 240293

location_facility: Clinica De Oftalmologia Nova Campinas /ID# 240271

location_facility: Emerson Clinical Research Institute. LLC /ID# 240274

location_facility: Maharaj Nakorn Chiang Mai Hospital /ID# 240194

location_facility: Centro Oftalmologico Macula Diagnostico and Tratamiento /ID# 240269

location_facility: Fundacion Oftalmologica de Santander - FOSCAL /ID# 240634

location_facility: Universitair Ziekenhuis Leuven /ID# 240312

location_facility: Singapore National Eye Centre /ID# 240224

location_facility: Hospital de Olhos de Mato Grosso Do Sul /ID# 240276

location_facility: Hospital Oftalmologico Medicina dos Olhos /ID# 240277

location_facility: Glaucoma associates/consultants of the capital region /ID# 240195

location_facility: University of Illinois at Chicago Illinois Eye and Ear Infirmary /ID# 250981

location_facility: Hamilton Glaucoma Center Shiley Eye Center UCSD /ID# 240196

location_facility: Rutgers New Jersey Medical School Campus, Doctors Office Center /ID# 240181

location_facility: Azienda Sanitaria Locale (ASL) Chieti /ID# 240217

location_facility: DCT Shah Eye Research Institut /ID# 240306

location_facility: Seoul National University Hospital /ID# 240262

location_facility: Consultorio Oftalmologico Dr. Fabian Lerner /ID# 240247

location_facility: Internationale Innovative Ophthalmochirurgie /ID# 240232

location_facility: Cambridge University Hospitals NHS Foundation Trust /ID# 240351

location_facility: Optimum Profesorskie Centrum Okulistyki Sp. z o.o. /ID# 240307


title: A Phase Ib, Multicenter, Open-Label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Cibisatamab in Combination With Atezolizumab After Pretreatment With Obinutuzumab in Patients With Previously Treated Metastatic, Microsatellite-Stable Colorectal Adenocarcinoma With High CEACAM5 Expression

summary: CO40939 is a Phase Ib, open-label, multicenter, single-arm study designed to evaluate the safety, efficacy, pharmacokinetics, and immunogenicity of cibisatamab in combination with atezolizumab administered after pretreatment with obinutuzumab in patients with Stage IV microsatellite stable (MSS) metastatic colorectal cancer (mCRC) whose tumors have high carcinoembryonic antigen-related cell adhesion molecule 5 (CEACAM5) expression and who have progressed on two or more chemotherapy regimens. The study is composed of a safety run-in and an exploratory part.

ipd_sharing_description: Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).

lead_sponsor: Hoffmann-La Roche

location_country:
United States
Denmark
Spain
France

location_facility: Clinica Universitaria de Navarra; Servicio de Oncologia

location_facility: Centre Leon Berard; Departement Oncologie Medicale

location_facility: Hospital Univ Vall d'Hebron; Servicio de Oncologia

location_facility: START Madrid-FJD, Hospital Fundacion Jimenez Diaz


intervention_type: Biological

lead_sponsor: Pfizer

summary: This study will evaluate the efficacy and safety of PF-06838435 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circulating activity of 2% or less). The gene therapy is designed to introduce genetic material into cells to compensate for missing or non-functioning Factor IX. Eligible study participants will have completed a minimum 6 months of routine Factor IX prophylaxis therapy during the lead in study (C0371004). Participants will be dosed once (intravenously) and will be evaluated over the course of 6 years. The main objective of the study will evaluate the annualized bleeding rate [ABR] for participants treated with gene therapy versus standard of care (SOC) therapy (FIX prophylaxis replacement regimen).

location_facility: Juravinski Hospital - Hamilton Health Sciences

location_facility: Indiana Hemophilia and Thrombosis Center, Inc

location_facility: Kaohsiung Medical University Chung-Ho Memorial Hospital

location_facility: Mississippi Center for Advanced Medicine

location_facility: Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti - HEMORIO

location_facility: St. Vincent Hospital & Health Care Center, Inc.

location_facility: National Center for Child Health and Development

location_facility: University of California, San Francisco - Clinical Research Center

location_facility: Skåne University Hospital, Department of Hematology, Oncology and Radiation Physics

location_facility: Newcastle upon Tyne Hospitals NHS Foundation Trust

location_facility: McMaster University Medical Centre - Hamilton Health Sciences

location_facility: Centro de Hematologia e Hemoterapia - Hemocentro de Campinas - UNICAMP

location_facility: Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus

location_facility: King Faisal Specialist Hospital and Research Centre

location_facility: Gaziantep University Sahinbey Training and Research Hospital

location_facility: Hopital Cardiologique Louis Pradel - CRTH

location_facility: Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti- HEMORIO

location_facility: Indiana Hemophilia & Thrombosis Center, Inc.

location_facility: General Hospital of Athens "LAIKO", 2nd Regional Blood Transfusion Center

location_facility: Universitätsklinikum Bonn, Anstalt des öffentlichen Rechts

location_facility: University of California, San Francisco - Outpatient Hematology Clinic

location_facility: Kyung Hee University Hospital at Gangdong

title: Phase 3, Open-label, Single-arm Study to Evaluate Efficacy and Safety of FIX Gene Transfer With PF-06838435 (rAAV-Spark100-hFIX-R338L) in Adult Male Participants With Moderately Severe to Severe Hemophilia B (FIX:C ≤2%) (BeneGene-2)

ipd_sharing_description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

location_country:
Germany
United Kingdom
Australia
Brazil
Japan
Sweden
United States
Korea, Republic of
Saudi Arabia
Greece
Turkey
Taiwan
Canada
France


summary: The purpose of this study is to evaluate the efficacy and safety of DCR-PHXC in Children and Adults with Primary Hyperoxaluria Type 1 (PH1) and Primary Hyperoxaluria Type 2 (PH2)

sponsor_collaborators: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

title: A Phase 2 Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of DCR-PHXC Solution for Injection (Subcutaneous Use) in Patients With Primary Hyperoxaluria

location_country:
Germany
Italy
Poland
New Zealand
Israel
United Kingdom
Romania
Australia
Japan
Netherlands
Lebanon
United States
Spain
Canada
France


title: An Extension Study of Venetoclax for Subjects Who Have Completed a Prior Venetoclax Clinical Trial

summary: The purpose of this extension study is to provide venetoclax and obtain long-term safety data for subjects who continue to tolerate and derive benefit from receiving venetoclax in ongoing studies.

location_country:
Ireland
Poland
New Zealand
Australia
Japan
Turkey
Canada
France
Portugal
Hong Kong
Denmark
United States
Ukraine
Belgium
United Kingdom
Austria
Mexico
Korea, Republic of
Spain
Greece
Taiwan
Russian Federation
Sweden

location_facility: Royal Prince Alfred Hospital /ID# 239557

location_facility: National Cancer Center Hospital /ID# 241516

location_facility: CHU Grenoble - Hopital Michallon /ID# 240497

location_facility: SP ZOZ Zespol Szpitali Miejskich w Chorzowie /ID# 225474

location_facility: King's College Hospital NHS Foundation Trust /ID# 218735

location_facility: Dartmouth-Hitchcock Medical Center /ID# 210555

location_facility: Vehbi Koc vakfi Amerikan Hasta /ID# 225046

location_facility: National Hospital Organization Mito Medical Center /ID# 241986

location_facility: The Clatterbridge Cancer Centre NHS Foundation Trust /ID# 218736

location_facility: Municipal Non-Profit Enterprise City Clinical Hospital 4 of Dnipro City Council /ID# 240575

location_facility: Regional Oncological Dispensary /ID# 225195

location_facility: Skane University Hospital Lund /ID# 224747

location_facility: Instytut Hematologii i Transfuzjologii /ID# 225478

location_facility: National Taiwan University Hospital /ID# 224946

location_facility: Kobe City Medical Center General Hospital /ID# 241518

location_facility: UCLA Santa Monica Hematology Oncology /ID# 210551

location_facility: Medizinische Universitaet Graz /ID# 223817

location_facility: Dana-Farber Cancer Institute /ID# 215360

location_facility: Hospital Universitario Fundacion Jimenez Diaz /ID# 225476

location_facility: University Hospital Southampton NHS Foundation Trust /ID# 225161

location_facility: University Hospital Plymouth NHS Trust /ID# 218731

location_facility: Hospital Universitario Dr. Jose Eleuterio Gonzalez /ID# 227017

location_facility: The Royal Wolverhampton NHS Trust /ID# 225164

location_facility: CHUQ- Hôpital de l'Enfant-Jesus /ID# 224616

location_facility: Istanbul University Istanbul Medical Faculty /ID# 225045

location_facility: University of Michigan Comprehensive Cancer Center Michigan Medicine /ID# 231132

location_facility: Wellington Regional Hospital /ID# 225593

location_facility: Federal State Budgetary Scientific Institution N.N. Blokhin /ID# 225194

location_facility: Centro de Investigacion Clínica Chapultepec SA de CV /ID# 227018

location_facility: Blackpool Teaching Hospitals NHS Foundation Trust /ID# 225163

location_facility: General Hospital of Athens Laiko /ID# 224087

location_facility: China Medical University Hospital /ID# 224945

location_facility: University College London Hospitals NHS Foundation Trust /ID# 240467

location_facility: General Hospital of Thessaloniki George Papanikolaou /ID# 224088

location_facility: University of Arizona Cancer Center - Tucson /ID# 210548


title: Open Label, Non-randomized Extension Trial to Assess Long Term Safety and Efficacy of Arimoclomol in Subjects With Amyotropic Lateral Sclerosis Who Have Completed the ORARIALS-01 Trial

summary: A multicenter, non-randomized, open label trial, to assess long term safety and efficacy of Arimoclomol in subjects with Amyotrophic Lateral Sclerosis (ALS) who have completed the ORARIALS-01 trial.

lead_sponsor: ZevraDenmark

location_country:
Germany
Italy
Poland
United Kingdom
Sweden
Netherlands
Belgium
United States
Spain
Canada
France

location_facility: Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK) - Ambulanz fuer ALS und andere Motoneuronenerkrankungen

location_facility: Centre Hospitalier Regional Universitaire (CHRU) Montpellier - Hopital Gui De Chauliac

location_facility: Azienda Ospedaliero Universitaria (AUO) di Torino - Citta'della Salute e della Scienza di Torino

location_facility: St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Center

location_facility: Hospital Universitario Vall d'Hebron ALS Unit. Consultas Externas; Office: 9-10-11

location_facility: Hospital Carlos III - Hospital Universitario La Paz, ALS Unit

location_facility: Medizinische Hochschule Hannover (MHH) - Klinik fuer Neurologie

location_facility: UC Irvine Health ALS and Neuromuscular Center

location_facility: Universitaetsklinikum Ulm - Klinik fuer Neurologie

location_facility: Leonard Wolfson Experimental Neurology Centre

location_facility: University of Pensylvania, Perelman Center for Advanced Medicine - Penn Neuroscience Center

location_facility: Groupe Hospitalier Pitie-Salpetriere - Centre d'Investigation Clinique Neurosciences 1422


acronym: NeoCOAST

lead_sponsor: MedImmune LLC

summary: Study D9108C00002 (NeoCOAST) is a platform study assessing the effectiveness and safety of neoadjuvant durvalumab alone or in combination with novel agents in participants with resectable, early-stage (Stage I [>2cm] to IIIA) non-small cell lung cancer (NSCLC).

title: A Phase 2 Open-label, Multicenter, Randomized, Multidrug Platform Study of Neoadjuvant Durvalumab Alone or in Combination With Novel Agents in Subjects With Resectable, Early-stage (I [> 2 cm] to IIIA) Non-small Cell Lung Cancer (NeoCOAST)

ipd_sharing_description:
Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All request will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure

Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

location_country:
Italy
Portugal
Switzerland
United States
Spain
Canada
France


lead_sponsor: Pharmacyclics LLC.

summary: Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)

sponsor_collaborators:
Pharmacyclics LLC.
Janssen Research & Development, LLC

location_facility: Hospital Universitario Universitario La Paz

location_facility: Hospital Infantil Universitario Nino Jesus

location_facility: Federal State Budgetary Institution "Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology

location_facility: children's and Women's Health Centre of British Columbia

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: The Edmond and Lily Safra Children's Hospital

location_facility: Cancer Center for Children. The Children's Hospital at Westmead

location_facility: Federal State Budgetary Educational Institution of Higher Professional Education "LP.Pavlov First Saint Petersburg State Medical

location_facility: Ann & Robert H Lurie Children's Hospital of Chicago

location_facility: Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica

location_facility: Schneider Children's Medical Center in Israel

location_facility: Safari Day Care, Great Ormond Street Hospital

location_facility: U.O.C. Ematologia Oncoematologia Pediatrica

location_facility: S.C. Farmacia Pediatrica - Presidio Ospedaliero Infantile Regina Margherita

location_facility: University of Minnesota Masonic Children's Hospital

title: Phase 1/2 Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects With Chronic Graft Versus Host Disease (cGVHD)

location_country:
Germany
Italy
Israel
United Kingdom
Australia
Netherlands
Austria
United States
Korea, Republic of
Spain
Canada
Russian Federation
France


acronym: RUBATO OL

lead_sponsor: Actelion

summary: The aim of this open-label (OL) trial is to study the long-term use of macitentan for up to 2 years in Fontan-palliated adult and adolescent patients beyond the 52 weeks of treatment in the parent RUBATO double-blind (DB) study (AC-055H301, NCT03153137). This OL trial studies the long-term effect of macitentan in Fontan-palliated patients as it is not known if the effect of macitentan is sustained beyond 52 weeks (end of the parent RUBATO DB study). In addition, the trial also studies the long-term safety of macitentan as this is also unknown. Furthermore, the opportunity will be given to patients who were on placebo in the parent RUBATO DB study to receive macitentan 10 mg and benefit from a potentially active treatment.

sponsor_collaborators:
Actelion
Covance
Henry Ford Health System
Almac Clinical Technologies
ActiGraph LLC
Medidata Solutions

location_facility: Wojewodzki Szpital Specjalistyczny We Wroclawiu

location_facility: The Prince Charles Hospital, Adult Congenital Heart Disease Unit

location_facility: Massachusetts General Hospital Heart Center

location_facility: Providence Medical Research Providence Health Care

location_facility: Krakowski Szpital Specjalityczny im. Jana Pawla II, Oddzial Kliniczny Chorob Serca i Naczyn

title: Prospective, Multi-center, Single-arm, Open-label Long-term Study Assessing the Safety, Tolerability, and Effectiveness of Macitentan in Fontan-palliated Adult and Adolescent Subjects

ipd_sharing_description: The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials\transparency. As noted on this site, requests for access to the study data can be submitted through Yale open Access (YODA) Project site at yoda.yale.edu

location_country:
Poland
New Zealand
United Kingdom
Denmark
Australia
United States
China
Taiwan
Czechia
Canada
France


acronym: EMBOLDEN

lead_sponsor: Shire

summary:
The main aim of the study is to determine if SHP611 given by injection into the spinal fluid that surrounds the brain and spinal cord (intrathecal; IT) prolongs the time for children with Metachromatic Leukodystrophy (MLD) to retain the ability to move from place to place. Other aims of the study are to determine the effects of intrathecal administration of SHP611 on movement and speech functions and to learn how well SHP611 injected in the spinal fluid that surrounds the brain and spinal cord is tolerated.

Study participants will receive SHP611 for about 2 years with the possibility of an extended treatment period.

sponsor_collaborators: Takeda Development Center Americas, Inc.

location_facility: IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN

location_facility: Ann & Robert H. Lurie Children's Hospital of Chicago

location_facility: University of Iowa Stead Family Children's Hospital

location_facility: New York University Langone Medical Center

location_facility: Los Angeles Biomedical Research Institute at Harbor-UCLA

location_facility: Birmingham Children's Hospital NHS Foundation Trust

location_facility: Stollery Children's Hospital University of Alberta

title: A Global, Multicenter, Single-arm, Matched External Control Study of Intrathecal SHP611 in Subjects With Late Infantile Metachromatic Leukodystrophy

ipd_sharing_description: De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants).

location_country:
Germany
Italy
Israel
Argentina
United Kingdom
Japan
Brazil
Netherlands
Belgium
United States
Spain
Greece
Canada
France


title: A Long-Term, Single-Arm, Open-Label, Multicenter, Phase 3 Follow-on Trial of ARGX-113-1704 to Evaluate the Safety and Tolerability of ARGX-113 in Patients With Myasthenia Gravis Having Generalized Muscle Weakness

acronym: ADAPT+

summary: This is a Long-Term, Single-Arm, Open-Label, Multicenter Phase 3 follow-on trial of the ARGX-113-1704 study to evaluate the safety and tolerability of ARGX-113 in patients with gMG. Patients who have completed at least 1 cycle of treatment and at least 1 year of trial ARGX-113-1705 and have started Part B are eligible to enroll in the open-label trial ARGX-113-2002 to receive efgartigimod by SC administration.

ReferenceCitation: Howard JF Jr, Bril V, Vu T, Karam C, Peric S, Margania T, Murai H, Bilinska M, Shakarishvili R, Smilowski M, Guglietta A, Ulrichts P, Vangeneugden T, Utsugisawa K, Verschuuren J, Mantegazza R; ADAPT Investigator Study Group. Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial. Lancet Neurol. 2021 Jul;20(7):526-536. doi: 10.1016/S1474-4422(21)00159-9. Erratum In: Lancet Neurol. 2021 Aug;20(8):e5.

repository_institution: University of Antwerp - Institutional Repository University of Antwerp

repository_normalized: irua

repositories_concat: irua

publisher_dissemination: Elsevier

lead_sponsor: argenx

location_country:
Germany
Italy
Poland
Hungary
Denmark
Japan
Netherlands
Belgium
United States
Czechia
Canada
Georgia
Serbia
Russian Federation
France

location_facility:
Investigator Site 6
Investigator Site 28
Investigator Site 13
Investigator Site 3
Investigator Site 30
Investigator Site 51
Investigator Site 36
Investigator Site 7
Investigator Site 22
Investigator Site 2
Investigator Site 8
Investigator Site 45
Investigator Site 34
Investigator Site 1
Investigator Site 52
Investigator Site 10
Investigator Site 33
Investigator Site 15
Investigator Site 38
Investigator Site 44
Investigator Site 24
Investigator Site 49
Investigator Site 16
Investigator Site 47
Investigator Site 5
Investigator Site 18
Investigator Site 23
Investigator Site 39
Investigator Site 46
Investigator Site 27
Investigator Site 35
Investigator Site 14
Investigator Site 11
Investigator Site 25
Investigator Site 41
Investigator Site 32
Investigator Site 50
Investigator Site 29
Investigator Site 26
Investigator Site 37
Investigator Site 17
Investigator Site 48
Investigator Site 43
Investigator Site 40
Investigator Site 19
Investigator Site 21
Investigator Site 31
Investigator Site 9
Investigator Site 20
Investigator Site 42
Investigator Site 4
Investigator Site 12


acronym: ATLAS-OLE

lead_sponsor: Genzyme, a Sanofi Company

summary:
Primary Objective:

To characterize the long-term safety and tolerability of fitusiran

Secondary Objectives:

To characterize the efficacy and long-term efficacy of fitusiran as assessed by the frequency of:

Bleeding episodes
Spontaneous bleeding episodes
Joint bleeding episodes
To characterize the effects of fitusiran on health-related quality of life (HRQOL) measures in participants ≥17 years of age

location_facility:
Investigational Site Number :0360001
Investigational Site Number :3720001
Investigational Site Number :3720002
Investigational Site Number :8400009
Investigational Site Number :8400011
Investigational Site Number :8400006
Investigational Site Number :7920002
Investigational Site Number :4100003
Investigational Site Number :4100002
Investigational Site Number :4580001
Investigational Site Number :8400016
Investigational Site Number :3920004
Investigational Site Number :1580001
Investigational Site Number :3560001
Investigational Site Number :3800002
Investigational Site Number :8040004
Investigational Site Number :1560014
Investigational Site Number :3560007
Investigational Site Number :2080001
Investigational Site Number :2760003
Investigational Site Number :1580008
Investigational Site Number :1560007
Investigational Site Number :1580004
Investigational Site Number :8400007
Investigational Site Number :0360002
Investigational Site Number :1560005
Investigational Site Number :8260001
Investigational Site Number :4580002
Investigational Site Number :4580003
Investigational Site Number :7920006
Investigational Site Number :3480002
Investigational Site Number :1240001
Investigational Site Number :1560006
Investigational Site Number :1560004
Investigational Site Number :1560013
Investigational Site Number :1560011
Investigational Site Number :8040003
Investigational Site Number :7920005
Investigational Site Number :0360003
Investigational Site Number :3560010
Investigational Site Number :3560004
Investigational Site Number :3920008
Investigational Site Number :2500003
Investigational Site Number :1560009
Investigational Site Number :1580007
Investigational Site Number :1580002
Investigational Site Number :7100002
Investigational Site Number :8400019
Investigational Site Number :3760001
Investigational Site Number :3560005
Investigational Site Number :3920002
Investigational Site Number :1560010
Investigational Site Number :8040002
Investigational Site Number :2760001
Investigational Site Number :8040001
Investigational Site Number :8400012
Investigational Site Number :3920003
Investigational Site Number :3800004
Investigational Site Number :7920003
Investigational Site Number :4100001
Investigational Site Number :8400002
Investigational Site Number :7920004
Investigational Site Number :8400008
Investigational Site Number :3560002
Investigational Site Number :4100004
Investigational Site Number :7920008
Investigational Site Number :8400001
Investigational Site Number :2500002
Investigational Site Number :3920001
Investigational Site Number :3920005
Investigational Site Number :1580003
Investigational Site Number :1560008
Investigational Site Number :8260004
Investigational Site Number :1580005
Investigational Site Number :1560002
Investigational Site Number :1560012
Investigational Site Number :7920007
Investigational Site Number :1560003
Investigational Site Number :7920012

title: An Open-label, Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX

location_country:
Germany
Ireland
Australia
Japan
Turkey
Canada
France
Hungary
Denmark
United States
Ukraine
India
China
Malaysia
Italy
United Kingdom
Korea, Republic of
South Africa
Taiwan
Israel


title: An Extended Access Program to Assess Long Term Safety of Bardoxolone Methyl in Patients With Chronic Kidney Disease

acronym: EAGLE

summary: This extended access study will assess the long-term safety and tolerability of bardoxolone methyl in qualified patients with chronic kidney disease (CKD) who previously participated in one of the qualifying clinical studies with bardoxolone methyl. Patients will remain in the study until bardoxolone methyl is available through commercial channels or until patient withdrawal, whichever is sooner.

ipd_sharing_description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

lead_sponsor: Biogen

location_country:
Australia
Japan
United States
Spain
Puerto Rico
France

location_facility: Nephrology Research NorthShore University Health System

location_facility: Japanese Red Cross Nagoya Daini Hospital

location_facility: Tokyo Metropolitan Children's Medical Center

location_facility: Arizona Kidney Disease and Hypertension Research Services, PLLC

location_facility: Tokyo Women's Medical University Hospital

location_facility: Centricity Research Phoenix Multispecialty

location_facility: South Carolina Nephrology & Hypertension Center, Inc

location_facility: Nephrology Associates of Northern Virginia, Inc.

location_facility: Mountain Kidney & Hypertension Associates

location_facility: Washington University School of Medicine

location_facility: Hopital Necker, Universite Paris Descartes

location_facility: Local Incorporated Administrative Agency Osaka City Hospital Organization Osaka City General Hospital

location_facility: Tufts Medical Center - Division of Nephrology

location_facility: Niigata University Medical and Dental Hospital

location_facility: University of Colorado Anschutz Medical Center

location_facility: Puerto Rico Clinical and Translational Research Consortium (PRCTRC)

location_facility: University of California San Francisco - Children's Renal Center


lead_sponsor: Rhythm Pharmaceuticals, Inc.

summary: This pivotal, phase 3 study is designed to confirm the efficacy and safety of setmelanotide, a potent melanocortin receptor type 4 (MC4R) agonist, for the treatment of obesity and hyperphagia in participants with Bardet Biedl syndrome (BBS) or Alström syndrome (AS). The study's primary efficacy endpoint is to evaluate the proportion of participants (≥ 12 years of age at baseline) who lose ≥ 10% of their baseline body weight following approximately (~) 52 weeks of treatment with setmelanotide compared to a historical control rate.

ReferenceCitation: Haqq AM, Chung WK, Dollfus H, Haws RM, Martos-Moreno GA, Poitou C, Yanovski JA, Mittleman RS, Yuan G, Forsythe E, Clement K, Argente J. Efficacy and safety of setmelanotide, a melanocortin-4 receptor agonist, in patients with Bardet-Biedl syndrome and Alstrom syndrome: a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial with an open-label period. Lancet Diabetes Endocrinol. 2022 Dec;10(12):859-868. doi: 10.1016/S2213-8587(22)00277-7. Epub 2022 Nov 7. Erratum In: Lancet Diabetes Endocrinol. 2023 Feb;11(2):e2.

oa_host_type: closed

repositories_concat: closed

ReferenceCitation: Haws RM, Gordon G, Han JC, Yanovski JA, Yuan G, Stewart MW. The efficacy and safety of setmelanotide in individuals with Bardet-Biedl syndrome or Alstrom syndrome: Phase 3 trial design. Contemp Clin Trials Commun. 2021 May 3;22:100780. doi: 10.1016/j.conctc.2021.100780. eCollection 2021 Jun.

oa_colors: green gold

evidence: oa journal (via doaj)

unpaywall_oa_status: gold

location_facility: Sorbonne University, Hôpital de la Pitié-Salpêtrière

location_facility: Universidad Autónoma de Madrid University Hospital Niño

location_facility: UMMS Baystate Health; BAYSTATE MEDICAL CENTER; Baystate Children's Specialty Center

location_facility: Centre Hospitalier Universitaire de Strasbourg

location_facility: University of Tennessee Health Science Center

title: A Phase 3 Trial of Setmelanotide (RM-493), a Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Patients With Moderate to Severe Obesity

location_country:
United Kingdom
United States
Spain
Canada
Puerto Rico
France


lead_sponsor: Idorsia Pharmaceuticals Ltd.

summary: The purpose of the study is to assess the efficacy and safety of 4 doses of cenerimod versus placebo in adult subjects with systemic lupus erythematosus (SLE).

location_facility: Municipal Institution "Zaporizhzhya Regional Clinical Hospital"

location_facility: Advanced Research Institute Inc Allergy & Rheumatology

location_facility: Unidad de Investigaciones Reumatológicas A.C.

location_facility: Private Small Scale Medical Center "Pulse"

location_facility: Hospital Clinic i Provincial de Barcelona

location_facility: Joint Muscle Medical Care and Research Institute - Lilington Office

location_facility: Hospital Universitario Regional de Málaga

location_facility: Clinical Rheumatological Hospital Number 25

location_facility: Poltava Regional Clinical Hospital named after M.V. Sklifossovsky

location_facility: New York Presbyterian Columbia University Medical Center

location_facility: Fondazione Policlinico Universitario A. Gemelli

location_facility: Consultorio Privado de Especialidad del Dr. José Javier Orozco Alcalá

location_facility: Municipal Nonprofit Institution "Vinnytsia City Clinical Hospital №1"

location_facility: Unidad de Atención Médica e Investigación en Salud

location_facility: Scientific and Research Institute of Invalid Rehabilitation (EST Complex) of Vinnytsia M.I.Pyrogov NMU MOHU,

location_facility: Szpital Uniwersytecki Number 2 im. dr. Jana Biziela w Bydgoszczy

location_facility: Azienda Ospedaliera San Camillo Forlanini

location_facility: Istanbul Universitesi Istanbul Tip Fakultesi

location_facility: North Georgia Rheumatology Group - Duluth

location_facility: Centro de Investigación Clínica GRAMEL, S.C.

location_facility: Ternopil University Clinic - Rheumatology department

location_facility: Investigacion Biomedica para el Desarrollo de Farmacos S.A. de C.V.

location_facility: University of the Philippines Manila - Philippine General Hospital

location_facility: Municipal Nonprofit Institution of Lviv Regional Council "Lviv Regional Clinical Hospital"

location_facility: Diagnostic and Consulting Center "Aleksandrovska" EOOD

location_facility: Municipal institution "Regional Clinical Hospital n.a. O.F. Herbachevskoho"

location_facility: Centro Integral en Reumatología S.A. de C.V.

location_facility: Consultorio Médico del Dr. Federico Galván Villegas

location_facility: Center for Rheumatology Immunology and Arthritis

location_facility: Integral Rheumatology & Immunology Specialists

location_facility: UBAM Unidad Biomédica Avanzada Monterrey

location_facility: Zakarpattya Regional Clinical Hospital n.a. A.Novak - Rheumatology Department

location_facility: Vinnytsya Regional Clinical Hospital - Rheumatology Department

location_facility: University of Colorado School of Medicine

location_facility: Valerius Medical Group and Research Center

location_facility: Twoja Przychodnia Poznańskie Centrum Medyczne

location_facility: Non-governmental private healthcare organization, Scientific Clinical Centre of JSC Russian Railways

location_facility: Multiprofile Hospital For Active Treatment Trimontium

location_facility: State Institution of Health Protection "Clinical Hospital #8"

location_facility: Municipal Institution Kyiv Regional Council "Kyiv Regional Clinical Hospital"

location_facility: Clinic of State Institution "National Scientific Centre "Acad. Strazhesko Institute of Cardiology"

location_facility: Cambridge University Hospitals NHS Foundation Trust

location_facility: Morales Vargas Centro de Investigación S.C.

location_facility: Szpital Specjalistyczny im. J. Dietla w Krakowie

location_facility: Far Eastern University - Nicanor Reyes Medical Foundation

location_facility: Federal State Budget Scientific Research Institution "Scientific Research Institute of Rheumatology n.a. V.A. Nasonova"

location_facility: Śląskie Centrum Reumatologii, Rehabilitacji i Zapobiegania Niepełnosprawności im gen. Jerzego Zi

location_facility: Egyesitett Szent Istvan es Szent Laszlo Korhaz Rendelointezet

location_facility: Guy's and Saint Thomas' NHS Foundation Trust

location_facility: Spitalul Clinic Judetean de Urgenta Cluj-Napoca

location_facility: Angeles University Foundation Medical Center

location_facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

location_facility: Hospital Universitario "Dr. Gonzalo Valdés Valdés"

location_facility: State University of New York Upstate Medical University

location_facility: City Clinical Hospital #15 named after O.M. Filatova

location_facility: University Multiprofile Hospital for Active Treatment Pulmed

location_facility: Centro de Atención e Investigación Cardiovascular del Potosí, S.C.

location_facility: Centrul Medical de Diagnostic si Tratament Ambulatoriu Neomed

location_facility: Polyclinic of Private Security and Detectives

location_facility: Paramount Medical Research and Consulting

location_facility: Centre Hospitalier Regional Universitaire Brest Hôpital de la Cavale Blanche à Brest

location_facility: Consultorio Particular Dr. Miguel Cortés Hernández

location_facility: Necmettin Erbakan Üniversitesi Meram Tıp Fakültesi

title: A Phase 2b, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy, Safety, and Tolerability of Cenerimod in Subjects With Moderate to Severe Systemic Lupus Erythematosus (SLE)

location_country:
Germany
Poland
Japan
Thailand
Turkey
Chile
Czechia
France
Hungary
United States
Ukraine
Romania
Puerto Rico
Philippines
Italy
United Kingdom
Mexico
Spain
Greece
Taiwan
Bulgaria
Georgia
Russian Federation
Israel


title: A Randomized, Multicenter, Phase Ib/III Study to Investigate the Pharmacokinetics, Efficacy, and Safety of Atezolizumab Subcutaneous Compared With Atezolizumab Intravenous in Patients With Previously Treated Locally Advanced or Metastatic Non-Small Cell Lung Cancer

summary: This study will evaluate the pharmacokinetics, safety, and efficacy of atezolizumab subcutaneous (SC) compared with atezolizumab intravenous (IV) in participants with locally advanced or metastatic Non-Small Cell Lung Cancer (NSCLC) who have not been exposed to cancer immunotherapy (CIT) and for whom prior platinum-based therapy has failed. The study is comprised of two parts, as follows: A dose-finding part (Part 1, Phase Ib) will aim to identify the dose of atezolizumab SC to be tested in Part 2. A dose-confirmation part (Part 2, Phase III, randomized) will aim to confirm that the dose moved forward from Part 1 yields drug exposure that is comparable to that of atezolizumab IV.

ReferenceCitation: Felip E, Burotto M, Zvirbule Z, Herraez-Baranda LA, Chanu P, Kshirsagar S, Maiya V, Chan P, Pozzi E, Marchand M, Monchalin M, Tanaka K, Tosti N, Wang B, Restuccia E. Results of a Dose-Finding Phase 1b Study of Subcutaneous Atezolizumab in Patients With Locally Advanced or Metastatic Non-Small Cell Lung Cancer. Clin Pharmacol Drug Dev. 2021 Oct;10(10):1142-1155. doi: 10.1002/cpdd.936. Epub 2021 Mar 31.

location_country:
Poland
New Zealand
Argentina
Brazil
Costa Rica
Thailand
Turkey
Chile
France
Hungary
Latvia
Ukraine
China
Peru
Italy
Guatemala
United Kingdom
Mexico
Korea, Republic of
Spain
Greece
South Africa
Russian Federation

location_facility: SBIH Kaluga Region Clinical Oncology Dispensary

location_facility: Auckland City Hospital, Cancer and Blood Research

location_facility: Cancer Center of Guangzhou Medical University

location_facility: Fejer Megyei Szent Gyorgy Egyetemi Oktato Korhaz

location_facility: Groote Schuur Hospital ( Uni of Capetown ); Oncology Dept

location_facility: ICIMED Instituto de Investigación en Ciencias Médicas

location_facility: James Lind Centro de Investigación Del Cáncer

location_facility: Hospital Universitario La Paz; Servicio de Oncologia

location_facility: Oncosite - Centro de Pesquisa Clinica Em Oncologia Ltda

location_facility: Hacettepe Uni Medical Faculty Hospital; Oncology Dept

location_facility: Murmansk Regional Clinical Hospital named after P.A. Bayandin

location_facility: Asst Papa Giovanni XXIII; Oncologia Medica

location_facility: Municipal Institution City Clinical Hospital #4 of Dnipro City Council

location_facility: Instituto do Cancer do Estado de Sao Paulo - ICESP

location_facility: St James Hospital; Dept of Oncology/Hematology

location_facility: Chulalongkorn Hospital; Medical Oncology

location_facility: IRCCS Istituto Clinico Humanitas; Oncologia

location_facility: Communal Non profit Enterprise Regional Center of Oncology; Oncosurgical dept of thoracic organs

location_facility: RCI Sumy Regional Clinical Oncological Dispensary

location_facility: Ege Uni Medical Faculty Hospital; Oncology Dept

location_facility: Chelyabinsk Regional Clinical Oncology Dispensary

location_facility: Bradford Hill Centro de Investigaciones Clinicas

location_facility: Zhejiang Cancer Hospital; Zhejiang Cancer Hospital cancer department

location_facility: Clínica San Gabriel; Unidad de Investigación Oncológica de la Clínica San Gabriel

location_facility: Union Hospital of Tongji Medical College, Dept. of Cancer Center; Cancer Center

location_facility: Tauranga Hospital, Clinical Trials Unit; BOP Clinical School

location_facility: Rajavithi Hospital; Division of Medical Oncology

location_facility: Riga East Clinical University Hospital Latvian Oncology Centre

location_facility: Fundación CENIT para la Investigación en Neurociencias

location_facility: Samsung Medical Centre; Medical Oncology

location_facility: Centro Oncologico Riojano Integral (CORI)

location_facility: Kartal Dr Lutfi Kirdar Sehir Hastanesi; Medical Oncology Department

location_facility: FSBI Russian Oncology Research Center n.a. Blokhin of MOH RF

location_facility: Maharaj Nakorn Chiang Mai Hospital; Department of Medicine

location_facility: West China Hospital - Sichuan University

location_facility: MEDSI Clinical Hospital on Pyatnitsky Highway; Department of antitumor drug therapy

location_facility: Harbin Medical University Cancer Hospital

location_facility: Clinica Internacional, Sede San Borja; Unidad de Investigacion de Clínica Internacional

location_facility: General Hospital "G.Papanikolaou"; Pulmonogy Clinic

location_facility: Filial #1 Regional Oncology Dispensary of Nizhniy Novgorod

location_facility: Sir Run Run Shaw Hospital Zhejiang University

location_facility: Prince of Songkla University; Division of Pulmonary Disease, Department of Medicine

location_facility: Narodowy Instytut Onkologii im. M. Sklodowskiej-Curie; Oddzial Badan Wczesnych Faz

location_facility: Waikato Hospital - Cancer and Blood Research Trials Unit; Regional Cancer Centre

location_facility: Regionalny Szpital Specjalistyczny im. W. Bieganskiego; Oddzial Onkologii Klinicznej

location_facility: Mazowieckie Centrum Leczenia Chorob Pluc i Gruzlicy; Dept of Pulmonology & subdiv. of oncology

location_facility: Ivano-Frankivsk Regional Oncology Center

location_facility: Medipol University Medical Faculty; Oncology Department

location_facility: Faculty of Med. Siriraj Hosp.; Med.-Div. of Med. Oncology