title: Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

acronym: UNIFAI

summary: This project is a global, multicenter, prospective, observational natural history study that can be used to understand the disease progression and support the development of safe and effective drugs and biological products for Friedreich ataxia.

lead_sponsor: Friedreich's Ataxia Research Alliance

location_country:
Germany
Ireland
Italy
India
New Zealand
United Kingdom
Australia
Brazil
Austria
Belgium
United States
Spain
Greece
Czechia
Canada
France

location_facility: University of Iowa, Stead Family Children's Hospital

location_facility: Tallaght University Hospital, Department of Neurology

location_facility: University Hospital Aachen, Dept. of Neurology

location_facility: Hospital Sant Joan de Déu, Servicio de Neurología

location_facility: University Hospital Bonn, Dept. of Neurology

location_facility: University of Tübingen, Dept. of Neurodegenerative Diseases, Hertie-Institute for Clinical Brain Research

location_facility: All India Institute of Medical Sciences (AIIMS)

location_facility: University College of London, Ataxia Centre, National Hospital for Neurology and Neurosurgery

location_facility: National and Kapodistrian University of Athens, Neurogenetics Unit

location_facility: Bambino Gesù Children's Hospital, Department of Neurosciences

location_facility: Motol University Hospital, Centre for Hereditary Ataxias

location_facility: Université Libre de Bruxelles, Hôpital Erasme, Dpt of Neurology

location_facility: Referente Clinico-Scientifico di Polo IRCCS "E. Medea"

location_facility: University of Munich, Dept. of Neurology, Friedrich-Baur-Institut

location_facility: Medical University Innsbruck, Department of Neurology

location_facility: Fondazione IRCCS Istituto Neurologico Carlo Besta

location_facility: Hôpital de Hautepierre, Service de Neurologie

location_facility: Hospital Universitario La Paz, Servicio de Neurologia


title: A Randomized Study of Andexanet Alfa Compared to Usual Care in Patients Receiving a Factor Xa Inhibitor Who Require Urgent Surgery or Procedure (ANNEXA-RS)

acronym: ANNEXA-RS

summary: The study will aim to find out if the drug andexanet alfa is safe and effective in preventing major bleeding during urgent surgery or invasive procedures. The study will compare the use of andexanet alfa to the usual care given at the study center.

study_start_date_type: Anticipated

status: Not yet recruiting

study_completion_date_type: Anticipated

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Anticipated

ipd_sharing: Yes

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 requests 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.

lead_sponsor: AstraZeneca

location_country:
Finland
Germany
Poland
New Zealand
Argentina
Australia
Brazil
Japan
Czechia
Canada
Serbia
France
Slovenia
Portugal
Slovakia
Hungary
Denmark
Switzerland
United States
Romania
Belgium
China
Malaysia
Estonia
Italy
United Kingdom
Singapore
Austria
Mexico
Korea, Republic of
Spain
Greece
Taiwan
Bulgaria
Georgia
Israel

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Ongoing


type: Other Identifier

type: EudraCT Number

title: A Phase 1/2 Randomized, Umbrella Study to Evaluate the Safety and Efficacy of Pembrolizumab Plus Enfortumab Vedotin (EV) in Combination With Investigational Agents Versus Pembrolizumab Plus EV, as First-Line Treatment for Participants With Advanced Urothelial Carcinoma (KEYMAKER-U04): Substudy 04B

summary: This study is a substudy being conducted under one pembrolizumab umbrella master study KEYMAKER-U04. The substudy will consist of 2 parts. Part 1 will evaluate the efficacy and safety of coformulated favezelimab/pembrolizumab plus EV and coformulated vibostolimab/pembrolizumab plus EV relative to pembrolizumab plus EV. There will be no comparison of coformulated favezelimab/pembrolizumab plus EV versus coformulated vibostolimab/pembrolizumab plus EV. If ORR and/or DRR are substantially better on coformulated favezelimab/pembrolizumab plus EV and/or coformulated vibostolimab/pembrolizumab plus EV compared with pembrolizumab plus EV, after evaluation of the totality of data, the sponsor might consider Part 2 (expansion) to further characterize the efficacy and safety of the treatment arms under study.

study_start_date_type: Actual

status: Recruiting

lead_sponsor: Merck Sharp & Dohme LLC

location_country:
Israel
United Kingdom
Australia
Netherlands
United States
Korea, Republic of
Spain
Chile
Taiwan
Canada
France

location_facility: Cleveland Clinic-Taussig Cancer Center ( Site 3036)

location_facility: Sunnybrook Research Institute - Odette Cancer Centre ( Site 3108)

location_facility: University of California, Irvine (UCI) Health - UC Irvine Medical Center ( Site 3045)

location_facility: Sheba Medical Center-ONCOLOGY ( Site 3503)

location_facility: National Cheng Kung University Hospital-Clinical Trial Center ( Site 3803)

location_facility: Huntsman Cancer Institute-HCI Clinical Trials Office ( Site 3041)

location_facility: Royal Brisbane and Women's Hospital-Medical Oncology Clinical Trials Unit, Cancer Care Services ( Si

location_facility: Icahn School of Medicine at Mount Sinai ( Site 3018)

location_facility: Severance Hospital, Yonsei University Health System-Medical oncology ( Site 3903)

location_facility: Chang Gung Memorial Hospital at Kaohsiung-Oncology and Hematology ( Site 3802)

location_facility: National Taiwan University Hospital-Oncology ( Site 3801)

location_facility: Indiana University Melvin and Bren Simon Cancer Center ( Site 3011)

location_facility: The Ottawa Hospital - General Campus-The Ottawa Hospital Cancer Centre ( Site 3105)

location_facility: centre hospitalier lyon sud ( Site 3606)

location_facility: Rabin Medical Center-Oncology ( Site 3504)

location_facility: Asan Medical Center-Department of Oncology ( Site 3901)

location_facility: Erasmus Medisch Centrum-Medical Oncology ( Site 3303)

location_facility: Rambam Health Care Campus-Oncology Division ( Site 3501)

location_facility: Princess Margaret Cancer Centre ( Site 3106)

location_facility: CHU de Bordeaux Hop St ANDRE ( Site 3607)

location_facility: Hospital Clinico San Carlos ( Site 3765)

location_facility: Washington University School of Medicine ( Site 3038)

location_facility: Austin Health-Cancer Clinical Trials Centre ( Site 3950)

location_facility: ROYAL MARSDEN HOSPITAL (CHELSEA) ( Site 3201)

location_facility: Hospital Universitari Vall d'Hebron ( Site 3767)

location_facility: Memorial Sloan Kettering Cancer Center ( Site 3031)

location_facility: Nederlands Kanker Instituut - Antoni van Leeuwenhoek (NKI-AVL)-medical oncology ( Site 3302)

intervention_type: Biological


title: A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Year of Age and Older

summary: The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) in participants with cystic fibrosis (CF).

status: Enrolling by invitation

ipd_sharing: No

ipd_sharing_description: Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing

lead_sponsor: Vertex Pharmaceuticals Incorporated

location_country:
Germany
United Kingdom
Australia
Sweden
Netherlands
Switzerland
United States
France

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

location_facility: The Emory Clinic / Children's Healthcare of Atlanta at Egleston

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: Great Ormond Street Hospital for Children

location_facility: Erasmus Medical Center / Sophia Children's Hospital

location_facility: Riley Hospital for Children at Indiana University Health

location_facility: Washington University School of Medicine / St. Louis Children's Hospital

location_facility: Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota

location_facility: Charite Paediatric Pulmonology Department

location_facility: Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center

location_facility: University of Wisconsin Hospital and Clinics

status_simplified: Unknown


title: A Global, Open-label, Adaptive Design Study to Investigate the Efficacy and Safety of SerpinPC in Subjects With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

summary:
The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of prophylactic SerpinPC administered subcutaneously (SC) to participants with severe hemophilia A (HemA) (with or without inhibitors) or moderately severe to severe hemophilia B (HemB) (without inhibitors) as part of the SerpinPC registrational program.

This study consists of 3 parts: Part 1: dose-justification phase, Part 2: dose-confirmatory phase, Part 3: extension phase for participants who complete either Part 1 or Part 2.

This adaptive design study has a randomized dose-justification component to investigate the efficacy and safety of SerpinPC as a therapeutic option, principally for participants with HemB without inhibitors. SerpinPC has a novel mechanism of action compared with marketed treatments and those that are in development.

lead_sponsor: ApcinteX Ltd

sponsor_collaborators:
ApcinteX Ltd
Centessa Pharmaceuticals plc

location_country:
Italy
Poland
United Kingdom
Australia
Belgium
United States
Spain
Armenia
South Africa
Taiwan
Canada
France

location_facility: Hospital Regional Universitario de Malaga Hospital Carlos Haya - Hospital Materno-Infantil

location_facility: A.O.U Citt della Salute e della Scienza di Torino

location_facility: Centre of Haematology named after prof. R. O. Yeolian

location_facility: Hospital Clínico Universitario Virgen de la Arrixaca

location_facility: University of Colorado School of Medicine

location_facility: Presidio Ospedaliero Universitario S. Maria della Misericordia - ASUFC

location_facility: Barts and London School of Medicine and Dentistry


title: A Phase 1/2, Multi-Center, Open-Label Study to Evaluate the Safety, Tolerability, and Preliminary Anti-tumor Activity of TNG462 in Patients With MTAP-deleted Advanced or Metastatic Solid Tumors

summary: This is a first in human study in patients with advanced or metastatic solid tumors known to have an MTAP deletion. The first part of the study is an open-label, dose escalation and the second part is an open label dose expansion in specific MTAP-deleted tumor types. The study drug, TNG462, is a selective PRMT5 inhibitor administered orally. The study is planned to treat up to 159 participants.

design_allocation: Non-Randomized

lead_sponsor: Tango Therapeutics, Inc.

location_country:
United States
Spain
France

location_facility: Huntsman Cancer Institute, University of Utah

location_facility: The University of Texas MD Anderson Cancer Center

location_facility: Hospital Universitario Fundacion Jimenez Diaz


title: A Phase 1/2 Open-Label Multicenter Trial to Characterize the Safety, Tolerability, and Preliminary Efficacy of CFT1946 as Monotherapy and in Combination With Trametinib in Subjects With BRAF V600 Mutant Solid Tumors

summary: The purpose of this study is to evaluate the safety and tolerability of CFT1946 as well as to determine the maximum tolerated dose (MTD) and/or the recommended Phase 2 dose (RP2D) of CFT1946 as monotherapy (Arm A) and in combination with trametinib (CFT1946 + trametinib; Arm B).

lead_sponsor: C4 Therapeutics, Inc.

location_facility: Washington University School of Medicine

location_facility: Hospital Clinico Universitario de Valencia

location_facility: South Texas Accelerated Research Therapeutics (START) Madrid - Hospital Fundacion Jiminez Diaz

location_facility: David H. Koch Center for Cancer Care at Memorial Sloan Kettering Cancer Center

location_facility: Virginia Cancer Specialists (NEXT Oncology Virginia)


title: A Phase 1 Open-Label, Multi-Center, Safety and Efficacy Study of PRT2527 as Monotherapy and in Combination With Zanubrutinib in Participants With Relapsed/Refractory Hematologic Malignancies

summary: This is a Phase 1 dose-escalation study of PRT2527, a potent and highly selective cyclin-dependent kinase (CDK) 9 inhibitor, in participants with select relapsed or refractory (R/R) hematologic malignancies. The purpose of this study is to evaluate the safety, tolerability, recommended phase 2 dose (PR2D), and preliminary efficacy of PRT2527 as a monotherapy and in combination with zanubrutinib.

lead_sponsor: Prelude Therapeutics

sponsor_collaborators:
Prelude Therapeutics
BeiGene

location_country:
Italy
Poland
Australia
United States
Canada
France

location_facility: University of Virginia Comprehensive Cancer Center

location_facility: Laura and Isaac Perlmutter Cancer Center at NYU Langone Health

location_facility: American Oncology Partners of Maryland, PA

location_facility: lstituto Romagnolo per lo Studio dei Tumori "Dino Amadori" IRST

location_facility: Ospedale Santa Maria delle Croci - AUSL della Romagna


title: A Phase 1 Study of KTX-1001, an Oral, First-In-Class, Selective, and Potent MMSET Catalytic Inhibitor That Suppresses H3K36me2 in Patients With Relapsed and Refractory Multiple Myeloma

summary: A Phase I study to evaluate the safety of a novel, orally available, selective, and potent small molecule inhibitor of the histone lysine methyl transferase MMSET (also known as NSD2/WHSC1) to prevent the dimethylation of H3K36 in adult patients with relapsed or refractory multiple myeloma (RRMM).

lead_sponsor: K36 Therapeutics, Inc.

location_country:
United States
Spain
Canada
France

location_facility: Mayo Clinic - Transplant Center - Rochester

location_facility: University of Texas Southwestern Harold C. Simmons Comprehensive Cancer Center

location_facility: Instituto de Investigacion Biomedica de Salamanca (IBSAL)

location_facility: UCSF Medical Center - Hematology and Blood and Marrow Transplant Clinic

location_facility: The Winship Cancer Institute of Emory University

location_facility: University of Kansas Cancer Center - Fairway

location_facility: University Health Network (UHN) - Princess Margaret Cancer Centre (Princess Margaret Hospital)

location_facility: Centre Hospitalier Universitaire de Nantes (CHU de Nantes) - Hotel-Dieu

location_facility: Institut Universitaire du Cancer de Toulouse - Oncopole


title: A Phase I, Multicenter, Open-label, First-in-Human, Dose Escalation and Expansion Study of AZD9592 as Monotherapy and in Combination With Anti-cancer Agents in Patients With Advanced Solid Tumors

acronym: EGRET

summary: This is a first-in-human (FIH) Phase I, multi-center, open-label, study of AZD9592, in patients with advanced solid tumors. The study consists of several study modules, each evaluating the safety, tolerability, preliminary efficacy, pharmacokinetics (PK), pharmacodynamics, anti-tumor activity, and immunogenicity of AZD9592, as monotherapy or in combination with anti-cancer agents.

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
Australia
Japan
United States
China
Korea, Republic of
Spain
Malaysia
Taiwan
Canada
France


title: A Multicenter, Open-label Extension Trial to Evaluate the Efficacy, Safety and Tolerability of HZN-825 in Patients With Diffuse Cutaneous Systemic Sclerosis

summary:
Primary Objectives:

The primary efficacy objective is to assess the efficacy of 52 weeks of open-label treatment with HZN-825 in participants with diffuse cutaneous systemic sclerosis, as measured by change from both baselines in forced vital capacity percent (FVC %) predicted.
The primary safety objective is to examine the safety and tolerability of 52 weeks of open-label treatment with HZN-825, inclusive of, but not limited to, adverse events (AEs), serious AEs (SAEs) and the adverse event of special interest (AESI), from Day 1 to 4 weeks after last dose.

lead_sponsor: Horizon Pharma Ireland, Ltd., Dublin Ireland

location_country:
France
Argentina
Mexico
United States
Korea, Republic of
Greece
Israel

location_facility: Medical University of South Carolina - PPDS

location_facility: Centro de Investigaciones Reumatologicas

location_facility: Centre Hospitalier Universitaire de Bordeaux, Hopital Pellegrin

location_facility: Centro de Alta Especialidad En Reumatologia E Investigacion Del Potosi SC

location_facility: Clinica de Investigacion en Reumatologia y Obesidad

location_facility: Centro de Investigación y Tratamiento Reumatológico S.C

location_facility: Michigan Medicine University of Michigan

location_facility: Unidad de Atencion Medica e Investigacion en Salud

location_facility: Centro de Investigaciones Médicas Tucumán

location_facility: Centro de Estudios de Investigacion Basica Y Clinica SC

location_facility: Aprillus Asistencia e Investigacion de Arcis Salud SRL


title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of GS-5245 for the Treatment of COVID-19 in Participants With High-Risk for Disease Progression

acronym: BIRCH

summary: The goal of this clinical study is to test how well the study drug, obeldesivir (formerly GS-5245), works and how safe it is in treating coronavirus disease 2019 (COVID-19) in participants that have a higher risk of getting a serious illness.

status: Terminated

study_completion_date_type: Actual

enrollment_type: Actual

lead_sponsor: Gilead Sciences

location_country:
Italy
Poland
Portugal
Hungary
Singapore
Romania
Japan
Brazil
United Kingdom
Mexico
Korea, Republic of
Spain
South Africa
Turkey
Taiwan
Bulgaria
Canada
France

location_facility: Multiprofile Hospital for Active Treatment - Samokov EOOD, Department of Internal Diseases

location_facility: Dr. Anil K. Gupta Medicine Professional Corporation

location_facility: Vancouver ID Research and Care Centre Society

location_facility: Faculdade de Medicina da Universidade Federal de Minas Gerais

location_facility: Specialized Hospital for Active Treatment of Pneumophthisiatric Diseases-Vratsa EOOD, Department of Pneumology

location_facility: PanAmerican Clinical Research Mexico S.A de C.V.

location_facility: Diagnostic Consultative Center 1 - Lon EOOD

location_facility: Hospital General Universitario Reina Sofia

location_facility: Diagnostic and Consulting Center Aleksandrovska EOOD

location_facility: Hualien Tzu Chi Hospital, Buddhist Tzu Chi Medical Foundation

location_facility: Unita Operativa Di Malattie Infettive Ospedale San Raffaele S.r.l.

location_facility: Panamerican Clinical Research Mexico S.A. de C.V.

location_facility: MHAT Sveta Karidad EAD, First Department of Anesthesiology and Intensive Care

location_facility: Diagnostic Consultative Center XX - Sofia EOOD

location_facility: National Centre for Infectious Diseases, Tan Tock Seng Hospital

location_facility: Centro de Pesquisa Clinica da Universidade Municipal de São Caetano do Sul (USCS)

location_facility: Specialìzed Hosp¡tal for Active Trealment of Pneumophthisiatric Diseases Haskovo EOOD, Department of pneumology and phthisiatry

location_facility: Multiprofile Hospital for Active Treatment Dr. Stamen Iliev AD, Department of Pneumology Phthisiatrics

location_facility: Diagnostic Consultative Center Sveti Georgi EOOD

location_facility: Hospital Universitari Germans Trias i Pujol

location_facility: Oaxaca Site Management Organization S.C.

location_facility: Chonnam National University Bitgoeul Hospital

location_facility: Multiprofile Hospital for Active Treatment Sveti Panteleymon EOOD, First Department of Internal Diseases

location_facility: Sibiu Emergency Clinical County Hospital

location_facility: CHU de Montpellier-Hopital La Colombiere

location_facility: Hospital General Universitario Dr. Balmis

location_facility: Acibadem University School of Medicine Atakent Hospital

location_facility: Hospital Universitario Puerta de Hierro - Majadahonda

location_facility: Hospital Universitario Virgen de Las Nieves.

location_facility: Multiprofile Hospital for Active Treatment Puls AD, Department of Internal Diseases

location_facility: Hospital Universitaro y Politecnico La Fe

location_facility: Centro Hospitalar de Entre Douro e Vouga - Hospital de Sao Sebaststiao

location_facility: "Prof. Dr. Matei Bals" National Institute for Infectious Diseases


title: A Long-Term Extension, Multi-Center Safety Study of AV-101 in Subjects With Pulmonary Arterial Hypertension (PAH) Who Have Completed Study AV-101-002 (IMPAHCT-FUL)

acronym: IMPAHCT-FUL

summary: IMPAHCT-FUL: Inhaled iMatinib Pulmonary Arterial Hypertension Clinical Trial - Follow Up Long Term Extension (LTE) Trial is a follow up study to establish the long-term safety of AV-101. The long-term effects of AV-101 on efficacy measures will also be assessed. Subjects who successfully complete the 24-week placebo-controlled parent trial (AV-101-002) will be offered the opportunity to continue into this LTE study. Subjects who enroll in the study will receive one of three active AV-101 doses until such time as the optimal dose has been selected in the parent study.

lead_sponsor: Aerovate Therapeutics

location_country:
Italy
Poland
Israel
Argentina
Singapore
Australia
United Kingdom
Mexico
Belgium
United States
Spain
South Africa
Chile
Canada
France

location_facility: Groupement Hospitalier Sud - Hôpital Bicêtre

location_facility: Hadassah University Hospital - Ein Kerem

location_facility: Kaiser Permanente - Los Angeles Medical Center

location_facility: Cliniques Universitaires de Bruxelles Hopital Erasme

location_facility: Azienda Ospedaliero Universitaria Ospedali Riuniti - Foggia

location_facility: Unidad de Investigacion Clinica en Medicina S.C.

location_facility: Istituto Mediterraneo per i Trapianti e Terapie ad Alta Specializzazione

location_facility: Centro de Investigaciones Clinicas de la Universidad Catolica

location_facility: Krakowski Szpital Specjalistyczny im. Jana Pawla II

location_facility: Les Hôpitaux Universitaires de Strasbourg

location_facility: Azienda Ospedaliera Universitaria Policlinico Umberto I


type: Registry Identifier

title: A Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel Group, Phase 2 Study to Evaluate Clinical Efficacy and Safety of Deucravacitinib (BMS-986165) in Participants With Alopecia Areata

summary: The purpose of this study is to evaluate the efficacy of Deucravacitinib versus placebo at Week 24 and safety and tolerability of Deucravacitinib versus placebo in adults with Alopecia Areata.

status: Active, not recruiting

ipd_sharing_description: BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria. Additional information regarding Bristol Myers Squibb's data sharing policy and process can be found at: https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosurecommitment.html

lead_sponsor: Bristol-Myers Squibb

location_country:
Poland
Australia
Japan
United States
Canada
France

location_facility: Mount Sinai Medical Center - Faculty Practice Associates (FPA) - Dermatology Associates

location_facility: University of Pittsburgh Medical Center (UPMC) - Arthritis and Autoimmunity Center - Falk Clinic


title: A Phase 2, Open-Label, Multicenter, Basket Study Evaluating the Efficacy of Brexucabtagene Autoleucel in Adults With Rare B-cell Malignancies (ZUMA-25)

summary: Master protocol: The goal of this master clinical study is to test how well the study drug, brexucabtagene autoleucel, works in participants with rare B-cell malignancies: relapsed/refractory Waldenstrom macroglobulinemia (r/r WM) (Substudy A - no longer recruiting), relapsed/refractory Richter transformation (r/r RT) (Substudy B), relapsed/refractory Burkitt lymphoma (r/r BL) (Substudy C and relapsed/refractory hairy cell leukemia (r/r HCL) (Substudy D - no longer recruiting).

lead_sponsor: Kite, A Gilead Company

location_country:
Germany
Italy
Netherlands
Austria
Switzerland
United States
Spain
France

location_facility: IRCCS Azienda Ospedaliero - Universitaria di Bologna

location_facility: City of Hope (City of Hope National Medical Center)

location_facility: Medical University of Vienna, Department of Internal Medicine I, Div. of Hematology

location_facility: Istituto Oncologico Della Svizzera Italiana (IOSI)

location_facility: ASST Grande Ospedale Metropolitano Niguarda

location_facility: Azienda Ospedale di Perugia - Ospedale S. Maria della Misericordia

location_facility: Washington University School of Medicine

location_facility: Centre hospitalier de Toulouse - Hematology department

location_facility: Radboud University Nijmegen Medical Centre


title: A Phase I/II, Open-Label, Single-Arm, Two-Part Trial to Evaluate Safety, Tolerability, Pharmacokinetics, and Anti-Tumor Activity of Glofitamab in Monotherapy and in Combination With Chemoimmunotherapy in Pediatric and Young Adult Participants With Relapsed/Refractory Mature B-Cell Non-Hodgkin Lymphoma

acronym: iMATRIX GLO

summary: The purpose of this study is to evaluate the safety and efficacy of glofitamab, as monotherapy and in combination with a standard chemoimmunotherapy regimen: rituximab, ifosfamide, carboplatin, and etoposide (R-ICE) in pediatric and young adult participants with relapsed and refractory (R/R) mature B-cell non-Hodgkin lymphoma (B-NHL).

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:
Germany
Italy
Denmark
Australia
United States
Korea, Republic of
Spain
France

location_facility: Hôpital Pellegrin; Service d'oncologie pédiatrique

location_facility: UCSF Benioff Children's Hospital Oakland

location_facility: Hospital Infantil Universitario Niño Jesus; Servicio de Onco-hematologia

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: Kaiser Permanente Oakland Medical Center

location_facility: Universitaetsklinikum Muenster; Paedriatrische Haematologie und Onkologie

location_facility: IRCCS Ospedale Pediatrico Bambino Gesù; Clinical trial center - Pad. Salviati 1 floor

location_facility: Ospedaliera Ospedale Infantile Regina Margherita; Oncoematologia Pediatrica-Centro Trapianti Cellule

location_facility: Seoul National University Hospital- Pediatric Site

location_facility: Rigshospitalet; Ny Medicin til Børn med Kræft


title: ORACLE: A Long-term Follow-up Study to Evaluate the Safety of GT005 in Participants With Geographic Atrophy Secondary to Age-related Macular Degeneration Treated in a Gyroscope-sponsored Antecedent Study

acronym: ORACLE

summary: The purpose of this study is to evaluate the long-term safety of GT005 in participants with Geographic Atrophy (GA) secondary to AMD who have been treated in an antecedent study.

primary_purpose: Other

lead_sponsor: Gyroscope Therapeutics Limited

sponsor_collaborators:
Gyroscope Therapeutics Limited
Novartis Pharmaceuticals

location_country:
Germany
United Kingdom
Australia
United States
Spain
France

location_facility: University Retina and Macula Associates, P.C. - Lemont Office

location_facility: Clinica Universidad de Navarra - Pamplona

location_facility: Retina Consultants of Texas (Retina Consultants of Houston) - Texas Medical Center

location_facility: Vitreo Retinal Associates Pa - the Millennium Center Location

location_facility: South Tyneside and Sunderland Nhs Foundation Trust - Sunderland Eye Infirmary

location_facility: The University of Melbourne - The Centre for Eye Research Australia (CERA)

location_facility: Mid Atlantic Retina - Wills Eye Hospital

intervention_type: Genetic


title: Re-Ablation Using a Tailored Approach Targeting EGM-Dispersion

acronym: RESTART

summary: Multi-center, international, non-randomized clinical trial evaluating the use of Volta's VX1 algorithm as used in combination with repeat catheter ablation after AF recurrence after previous catheter ablation.

lead_sponsor: Volta Medical

location_country:
United States
Belgium
France

location_facility: Kansas City Cardiac Arrhythmia Research LLC

intervention_type: Device


title: Efficacy and Safety of NNC6019-0001 at Two Dose Levels in Participants With Transthyretin Amyloid Cardiomyopathy (ATTR CM)

summary: This study is testing a potential new medicine, NNC6019-0001, for people who have a heart disease due to TTR amyloidosis.The study will look at if this medicine can reduce the symptoms of a heart disease due to TTR amyloidosis, such as heart failure. Participants will either get NNC6019-0001 (apotential new medicine) or placebo (a medicine which has no effect on the body). Which treatment participants get is decided by chance. The chance of getting NNC6019-0001 is two times higher than getting placebo. NNC6019-0001 is not yet approved in any country or region in the world. It is a new medicine that doctors cannot prescribe yet. Participants will get an infusion of the study medicine 13 times, once every 4 weeks. The study will last for about 64 weeks after the first dose of medicine. Participants cannot participate in this study if they have a heart disease other than a heart disease due to TTR amyloidosis.

ipd_sharing_description: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com

lead_sponsor: Novo Nordisk A/S

location_country:
Germany
Italy
Portugal
Japan
Netherlands
United States
Spain
Czechia
Canada
France


title: A Phase 1, First-in-human Study of JNJ-80948543, a T-cell Redirecting Antibody, in Participants With NHL and CLL

summary: The purpose of this study is to characterize safety and to determine the putative recommended Phase 2 dose(s) (RP2D[s]) and optimal dosing schedule(s) of JNJ-80948543 in Part A (Dose Escalation) and to further characterize the safety of JNJ-80948543 at the putative RP2D(s) in Part B (Cohort Expansion).

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

lead_sponsor: Janssen Research & Development, LLC

location_country:
France
Poland
Denmark
Australia
United States
Israel

location_facility: The University of Texas MD Anderson Cancer Center

location_facility: Uniwersyteckie Centrum Kliniczne, Osrodek Badan Klinicznych Wczesnych Faz


title: XIENCE Skypoint Large Vessel Post Approval Study

acronym: SPIRIT XLV PAS

summary: SPIRIT XLV PAS is a prospective, single arm, multi-center, US and OUS post-approval observational study to evaluate the continued safety and effectiveness of the XIENCE Skypoint Large Vessel Everolimus Eluting Coronary Stent System (EECSS) Large Vessel (LV) sizes (diameter 4.5 mm and 5.0 mm) during commercial use in a real-world setting.

study_type: Observational

time_perspective: Prospective

ipd_sharing: Undecided

lead_sponsor: Abbott Medical Devices

location_facility: Via Christi Regional Medical Center - St. Francis Campus

location_facility: Hospital Universitario Marqués de Valdecilla

location_facility: Hopital Cardiovasculaire et Pneumologique Louis Pradel


lead_sponsor: Vertex Pharmaceuticals Incorporated

summary: The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

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

location_facility: The Emory Clinic / Children's Healthcare of Atlanta at Egleston

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: Great Ormond Street Hospital for Children

location_facility: Erasmus Medical Center / Sophia Children's Hospital

location_facility: Riley Hospital for Children at Indiana University Health

location_facility: Washington University School of Medicine / St. Louis Children's Hospital

location_facility: Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota

location_facility: Charite Paediatric Pulmonology Department

location_facility: Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center

location_facility: University of Wisconsin Hospital and Clinics

location_facility: Texas Children's Hospital - Baylor College of Medicine

title: A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-121/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Through 11 Years of Age

source: euctr


title: A Multicentre Trial Evaluating the Efficacy and Safety of Oral Decitabine Tetrahydrouridine (NDec) in Patients With Sickle Cell Disease

summary: This study examines how well a new, potential medicine called NDec works and is tolerated in people with sickle cell disease. NDec is a combination of two medicines (decitabine-tetrahydrouridine). Both medicines are new for the treatment of sickle cell disease. Participants who are not taking Hydroxyurea (HU) will get NDec, NDec and placebo, or placebo. Participants who are on HU treatment before joining the study will get NDec, NDec and placebo, or continue on HU. Which treatment participants get is decided by chance. Participants getting NDec and/or Placebo will get capsules to take twice weekly. The study will last for about a year.

ipd_sharing_description: "According to the Novo Nordisk disclosure commitment on novonordisk-trials.com"

location_country:
Italy
India
United Kingdom
Lebanon
United States
Spain
Greece
South Africa
Turkey
Canada
France


title: A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH)

summary: This is a study to evaluate the safety and efficacy of losmapimod in treating participants with Facioscapulohumeral Muscular Dystrophy (FSHD). Participants diagnosed with Facioscapulohumeral muscular dystrophy type 1 (FSHD1) or Facioscapulohumeral muscular dystrophy type 2 (FSHD2) will participate in Part A (Placebo-controlled treatment period) and will be randomized in a 1:1 ratio to receive losmapimod 15 milligrams (mg) or placebo orally twice daily (BID). Upon completion of Part A, participants will have the option to rollover into Part B (open-label extension) to evaluate the long-term safety, tolerability, and efficacy of losmapimod and will receive losmapimod 15 mg orally BID.

ReferenceType: background

ReferenceCitation: Barbour AM, Sarov-Blat L, Cai G, Fossler MJ, Sprecher DL, Graggaber J, McGeoch AT, Maison J, Cheriyan J. Safety, tolerability, pharmacokinetics and pharmacodynamics of losmapimod following a single intravenous or oral dose in healthy volunteers. Br J Clin Pharmacol. 2013 Jul;76(1):99-106. doi: 10.1111/bcp.12063.

ReferenceCitation: Han JJ, Kurillo G, Abresch RT, de Bie E, Nicorici A, Bajcsy R. Reachable workspace in facioscapulohumeral muscular dystrophy (FSHD) by Kinect. Muscle Nerve. 2015 Feb;51(2):168-75. doi: 10.1002/mus.24287. Epub 2014 Nov 19.

ReferenceCitation: Mellion ML, Ronco L, Berends CL, Pagan L, Brooks S, van Esdonk MJ, van Brummelen EMJ, Odueyungbo A, Thompson LA, Hage M, Badrising UA, Raines S, Tracewell WG, van Engelen B, Cadavid D, Groeneveld GJ. Phase 1 clinical trial of losmapimod in facioscapulohumeral dystrophy: Safety, tolerability, pharmacokinetics, and target engagement. Br J Clin Pharmacol. 2021 Dec;87(12):4658-4669. doi: 10.1111/bcp.14884. Epub 2021 May 14.

lead_sponsor: Fulcrum Therapeutics

location_country:
Germany
Italy
United Kingdom
Denmark
Netherlands
United States
Spain
Canada
France

location_facility: University of Colorado Anschutz Medical Campus

location_facility: Hospital Universitari i Politecnic La Fe

location_facility: Newcastle upon Tyne NHS Foundation Trust

location_facility: Montreal Neurological Institute and Hospital

location_facility: Fondazione Serena Onlus- Centro Clinico NEMO

location_facility: University of California Los Angeles (UCLA)

location_facility: Washington University School of Medicine

location_facility: LMU Klinikum Ludwig-Maximilians-Universität München

location_facility: Institute de Myologie, Groupe Hospitalier Pitié-Salpêtrière

location_facility: University of Massachusetts Memorial Medical Center

location_facility: Fondazione IRCCS Istituto Neurologico Carlo Besta


title: A Phase II/III, Extension Study of Orally Administered PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema Due to C1-Inhibitor Deficiency (Type I or Type II)

summary: This study evaluates the safety and efficacy of long-term on-demand treatment with orally administered deucrictibant for acute hereditary angioedema (HAE) attacks, including laryngeal attacks, in patients with HAE due to C1-esterase inhibitor (C1-INH) deficiency (type I/II). The study will enroll patients from Study PHA022121-C201 (NCT04618211) who elect to participate in this extension study and meet the eligibility requirements.

lead_sponsor: Pharvaris Netherlands B.V.

location_country:
Germany
Poland
Hungary
Israel
United States
Spain
Czechia
Bulgaria
Canada
France


title: Phase 3 Randomized, Placebo-Controlled Study to Assess Safety, Tolerability, and Efficacy of Garetosmab in Patients With Fibrodysplasia Ossificans Progressiva

acronym: OPTIMA

summary:
This study is researching an experimental drug called garetosmab. The study is focused on adult patients with fibrodysplasia ossificans progressiva (FOP). The aim of the study is to see how safe and effective the study drug is in patients with FOP.

The study is looking at several other research questions, including:

What side effects may happen from receiving the study drug
How much study drug is in your blood at different times
Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)

ipd_sharing_description: All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing

lead_sponsor: Regeneron Pharmaceuticals

location_country:
Finland
Italy
Poland
United Kingdom
Hong Kong
Australia
Brazil
Japan
United States
Korea, Republic of
Spain
Malaysia
South Africa
Chile
France

location_facility: Royal National Orthropaedic Hospital NHS Trust

location_facility: HUS Children and Adolescents Park Hospital Clinical Trial Unit

location_facility: University of California Los Angeles (UCLA) Medical Center


lead_sponsor: Ionis Pharmaceuticals, Inc.

summary: The purpose of this study is to evaluate the long-term safety and efficacy of donidalorsen in people with HAE and the effects of donidalorsen on the number of HAE attacks and their impact on quality of life (QoL).

title: An Open-Label, Long Term Safety and Efficacy Study of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

location_country:
Germany
Italy
Poland
Israel
United Kingdom
Netherlands
Belgium
United States
Spain
Turkey
Bulgaria
Canada
Puerto Rico
France


title: Characteristics and Management of Acute Kidney Injury in Hospitalized Patients With Cirrhosis: a Multicenter Intercontinental Observational Prospective Study: The International Club of Ascites GLOBAL AKI Project

acronym: GLOBAL-AKI

summary:
The aims of this study will be to identify the clinical characteristics, the management and the outcomes of acute kidney injury in patients with cirrhosis worldwide.

Specific aims:

To establish the severity of AKI across different regions
To identify precipitants of AKI across different centers
To identify the phenotypes of AKI across different centers
To evaluate differences in the management of AKI across different centers and their impact on clinical outcomes
To assess outcomes of acute kidney injury (resolution of AKI, in-hospital mortality, 28-day mortality, 90-day mortality)

ReferenceType: result

ReferenceCitation: Angeli P, Gines P, Wong F, Bernardi M, Boyer TD, Gerbes A, Moreau R, Jalan R, Sarin SK, Piano S, Moore K, Lee SS, Durand F, Salerno F, Caraceni P, Kim WR, Arroyo V, Garcia-Tsao G. Diagnosis and management of acute kidney injury in patients with cirrhosis: revised consensus recommendations of the International Club of Ascites. J Hepatol. 2015 Apr;62(4):968-74. doi: 10.1016/j.jhep.2014.12.029. Epub 2015 Jan 28. No abstract available. Erratum In: J Hepatol. 2015 Jul;63(1):290.

oa_colors: green hybrid

oa_host_type: publisher;repository

evidence: open (via crossref license)

host_type: publisher

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

version: publishedVersion

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

host_type: repository

license_normalized: no license

unpaywall_oa_status: hybrid

publisher_dissemination: Elsevier

lead_sponsor: Azienda Ospedaliera di Padova

location_country:
Germany
Poland
Argentina
Brazil
Chile
France
Hungary
Denmark
United States
Ethiopia
India
China
Peru
Egypt
Italy
Paraguay
Netherlands
Mexico
Korea, Republic of
Spain
Russian Federation

location_facility: Hospital Nacional Prof. Alejandro Posadas

location_facility: Hospital de Gastroenterología "Dr. Carlos Bonorino Udaondo"

location_facility: IRCCS Azienda Ospedaliera-Universitaria di Bologna

location_facility: Hospital de Clínicas Facultad de Ciencias Médicas U.N.A.

location_facility: Shanghai Jiao Tong University School of Medicine

location_facility: Azienda Ospedaliero Universitaria Città della Salute e della Scienza di Torino

location_facility: Instituto Nacional de Ciencias Médicas y Nutrición "Salvador Zubirán"

intervention_type: Combination Product


title: Phase IIIb, Open-label, Single-arm, Multi-center Study to Evaluate the Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally (1.2 x 10^14 Vector Genomes) to Participants 2 to < 18 Years of Age With Spinal Muscular Atrophy (SMA) Who Have Discontinued Treatment With Nusinersen (Spinraza®) or Risdiplam (Evrysdi®)

acronym: STRENGTH

summary: This is an open-label, single arm, multi-center study. Approximately 28 participants aged 2 to <18 years will be enrolled stratified as 2 to 5 years and 6 to < 18 years. The study is comprised of 3 periods, Screening (up to 45 days), Treatment (1 day), and Follow-up (52 weeks).

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 https://www.clinicalstudydatarequest.com/.

lead_sponsor: Novartis Pharmaceuticals

location_country:
Italy
Australia
Japan
Netherlands
Belgium
United States
Spain
Canada
France


title: Endurant Stent Graft System vs Excluder Endoprothesis: A Global, Prospective, Randomized Clinical Trial in Sac Regression

acronym: ADVANCE

summary: The purpose of this trial is to generate clinical evidence related to key performance outcomes of Endurant II/IIs Stent Graft Systems verses Gore Excluder / Excluder Conformable AAA Endoprosthesis in subjects with Abdominal Aortic Aneurysms. Subjects are randomized and imaging collected at all follow-up time points to assess the primary endpoint.

lead_sponsor: Medtronic Cardiovascular

location_country:
Germany
Italy
Finland
United Kingdom
Japan
Netherlands
Switzerland
United States
Spain
Taiwan
France

location_facility: Niigata University Medical and Dental Hospital

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: University of California Irvine Medical Center

location_facility: Oxford University Hospitals NHS Trust - John Radcliffe Hospital

location_facility: University of California Davis Medical Center

location_facility: Hospital of the University of Pennsylvania

location_facility: University of Texas Health Science Center at Houston

location_facility: Azienda Ospedaliera Universitaria Federico II

location_facility: Assistance Publique - Hopitaux de Marseille Hopital de la Timone

location_facility: University Hospitals, Cleveland Medical Center


title: A Phase 3, Long-Term Extension Study to Evaluate the Safety and Efficacy of Imsidolimab (ANB019) in the Treatment of Adult Subjects With Generalized Pustular Psoriasis

summary: This is a Phase 3, long term extension study to evaluate the safety and efficacy of imsidolimab compared with placebo in adult subjects with generalized pustular psoriasis (GPP).

lead_sponsor: AnaptysBio, Inc.

location_country:
Germany
Poland
Romania
Australia
Tunisia
United States
Korea, Republic of
Spain
Thailand
Malaysia
Turkey
Morocco
Taiwan
Georgia
France

intervention_type: Other


title: A Phase II Randomized, Double-blind, Placebo-controlled, Proof-of-Concept Study to Evaluate the Safety and Efficacy of Cotadutide in Participants With Non-cirrhotic Non-alcoholic Steatohepatitis With Fibrosis

acronym: PROXYMO-ADV

summary: The purpose of this study is to evaluate the safety and efficacy of cotadutide in participants with non-cirrhotic NASH with fibrosis.

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: AstraZeneca

location_country:
Germany
New Zealand
Argentina
Australia
Japan
Thailand
Turkey
Canada
France
United States
Malaysia
Italy
United Kingdom
Austria
Korea, Republic of
Spain
Greece
South Africa
Taiwan
Israel


title: A Phase 1b Dose Escalation and Dose Expansion Study Evaluating the Safety, Pharmacokinetics, and Antitumor Activity of Furmonertinib in Patients With Advanced or Metastatic Non-Small Cell Lung Cancer (NSCLC) With Activating Epidermal Growth Factor Receptor (EGFR) or Human Epidermal Growth Factor Receptor 2 (HER2) Mutations

summary: This is a Phase 1b, open-label, multi-center, dose-escalation and dose expansion study designed to evaluate the safety, pharmacokinetics (PK), and preliminary antitumor activity of furmonertinib in patients with advanced or metastatic non-small cell lung cancer (NSCLC) with activating, including uncommon, Epidermal Growth Factor Receptor (EGFR) or Human Epidermal Growth Factor Receptor 2 (HER2) mutations. Patients will be enrolled into one of 2 stages: Stage 1 (Dose Escalation and Backfill Cohorts) and Stage 2 (Dose Expansion).

lead_sponsor: ArriVent BioPharma, Inc.

location_country:
Italy
United Kingdom
Australia
Japan
Netherlands
United States
China
Korea, Republic of
Spain
Canada
France

location_facility:
ArriVent Investigative Site
Allist Investigative Site
Arrivent Investigative Site


lead_sponsor: Ultragenyx Pharmaceutical Inc

summary: The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels with removal of dietary protein restriction and alternative pathway medication.

location_facility: University Hospitals Cleveland Medical Center

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

location_facility: University of Florida College of Medicine

location_facility: Hospital Clinico Universitario de Santiago

location_facility: Erasmus Universitair Medisch Centrum Rotterrdam

location_facility: Fundacio Hospital Universitari Vall D'Hebron-Institute de Recerca

location_facility: University of Arkansas for Medical Sciences

location_facility: Centro Hospitalar Universitario de Sao Joao

title: A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients With Late-onset OTC Deficiency

location_country:
Germany
Italy
Portugal
United Kingdom
Argentina
Australia
Brazil
Japan
Netherlands
United States
Spain
Canada
France


title: First-in-Human Study of the BCL-2 Inhibitor ABBV-453 in Biomarker-Selected Subjects With Relapsed or Refractory Multiple Myeloma

summary:
Multiple myeloma (MM) is a plasma cell disease characterized by the growth of clonal plasma cells in the bone marrow. The purpose of this study is to assess the safety and toxicity of ABBV-453 in adult participants with relapsed/refractory (R/R) MM. Adverse events and change in disease activity will be assessed.

ABBV-453 is an investigational drug being developed for the treatment of R/R MM. Part 1 will be a monotherapy dose escalation phase to determine the best dose of ABBV-453. In Part 2, participants are placed in 1 of 3 groups called treatment arms. Each group receives a different treatment. Approximately 28 to 48 adult participants in Part 1 and 150 to 312 adult participants in Part 2 with R/R MM will be enrolled in the study in approximately 70 sites worldwide.

In Part 1 and the Japan Cohort, Participants will receive oral ABBV-453 tablets once daily (QD) in 28-day cycles. In Part 2, Arm 1, participants will receive continuous doses of oral ABBV-453 tablets QD in combination with oral dexamethasone tablets once weekly in 28-day cycles. In Part 2, Arm 2, participants will receive continuous doses of oral ABBV-453 tablets QD in combination with subcutaneous injections of daratumumab every 1 to 4 weeks and oral dexamethasone tablets once weekly in, 28-day cycles. In Part 2, Arm 3, participants will receive continuous doses of oral ABBV-453 tablets QD in combination with subcutaneous injections of daratumumab every 1 to 4 weeks, oral lenalidomide capsules QD on Days 1-21, and oral dexamethasone tablets once weekly, in 28-day cycles.

There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at an approved institution (hospital or clinic). The effect of the treatment will be frequently checked by medical assessments, blood tests, and side effects.

lead_sponsor: AbbVie

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

location_facility: Vanderbilt Ingram Cancer Center /ID# 242810

location_facility: CHU de Nantes, Hotel Dieu -HME /ID# 248029

location_facility: Hadassah Medical Center-Hebrew University /ID# 250484

location_facility: Sylvester Comprehensive Cancer Center /ID# 243417

location_facility: LMU - Klinikum Campus Innenstadt /ID# 248746

location_facility: Universitaetsklinik Heidelberg /ID# 249742

location_facility: Hadassah University Hospital /ID# 250484

location_facility: Princess Margaret Cancer Centre /ID# 249121

location_facility: Dana-Farber Cancer Institute /ID# 242752

location_facility: Tel Aviv Sourasky Medical Center /ID# 250483

location_facility: The Chaim Sheba Medical Center /ID# 250482

location_facility: Stanford University School of Med /ID# 242809

location_facility: University of Michigan Comprehensive Cancer Center Michigan Medicine /ID# 242754

location_facility: CLINICA UNIVERSIDAD DE NAVARRA-Madrid /ID# 248030

location_facility: Memorial Sloan Kettering Cancer Center /ID# 243503

location_facility: Hospital of the University of Pennsylvania /ID# 242842

location_facility: St Vincent's Hospital Melbourne /ID# 244827

location_facility: American Oncology Partners of Maryland, PA /ID# 244858


title: Randomised, Open-label and Parallel Group Trial to Investigate the Effects of Oral BI 685509 Alone or in Combination With Empagliflozin on Portal Hypertension After 8 Weeks Treatment in Patients With Clinically Significant Portal Hypertension (CSPH) in Compensated Cirrhosis

summary:
This study is open to adults with liver cirrhosis caused by hepatitis B, hepatitis C or nonalcoholic steatohepatitis (NASH). People can join this study if they have high blood pressure in the portal vein (main vessel going to the liver).

The purpose of this study is to find out whether a medicine called BI 685509 taken alone or in combination with a medicine called empagliflozin helps people with this condition.

Participants take BI 685509 as tablets twice a day for 8 weeks. Half of the participants with NASH who also have type 2 diabetes take empagliflozin as tablets once a day in addition to BI 685509.

Participants are in the study for about 3 months. During this time, they visit the study site about 10 times. At 2 of the visits, the doctors check the pressure in a liver vein to see whether the treatment works. This is done with a catheter (a long thin tube) and gives information about the pressure in the portal vein. The doctors also regularly check participants' health and take note of any unwanted effects.

ipd_sharing_description:
Once the criteria in section "Time frame" are fulfilled, researchers can use the following link https://www.mystudywindow.com/msw/datasharing to request access to the clinical study documents regarding this study, and upon a signed "Document Sharing Agreement".

Furthermore, researchers can request access to the clinical study data, for this and other listed studies, after the submission of a research proposal and according to the terms outlined in the website.

lead_sponsor: Boehringer Ingelheim

location_country:
Germany
Italy
Israel
Argentina
Denmark
Japan
Romania
Netherlands
Austria
Belgium
Singapore
United States
China
Spain
Switzerland
United Kingdom
Canada
France

location_facility: Centre Hospitalier de l'Universite de Montreal (CHUM)

location_facility: ASST Grande Ospedale Metropolitano Niguarda

location_facility: Universitätsmedizin der Johannes Gutenberg-Universität Mainz

location_facility: Azienda Ospedaliera Policlinico di Modena

location_facility: National Hospital Organization Yokohama Medical Center

location_facility: Regional Institute of Gastroenterology Hepatology "Prof. Dr. O. Fodor"

location_facility: The Affiliated Hospital of Hangzhou Normal University


title: A Phase 1 First-Time-in-Human, Open-Label Study of GSK4381562 Administered as Monotherapy and in Combination With Anticancer Agents in Participants With Selected Advanced Solid Tumors

summary: This is a first time in-human (FTIH) study designed to investigate the safety, tolerability, pharmacokinetics (PK), and immunogenicity of GSK4381562 in participants with select loco-regionally recurrent solid tumors or metastatic solid tumors where curative or standard treatment options have been exhausted

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

lead_sponsor: GlaxoSmithKline

location_country:
United Kingdom
Australia
Japan
United States
China
Korea, Republic of
Spain
Canada
France


title: A Phase 1/2, Multi-Center, Open-Label Study to Evaluate the Safety, Tolerability, and Preliminary Anti-tumor Activity of TNG908 in Patients With MTAP-deleted Advanced or Metastatic Solid Tumors

summary: This is a first in human study in patients with advanced or metastatic solid tumors known to have an MTAP deletion. The first part of the study is an open-label, dose escalation and the second part is an open label dose expansion in specific MTAP-deleted tumor types. The study drug, TNG908, is a selective PRMT5 inhibitor administered orally. The study is planned to treat up to 192 participants.

lead_sponsor: Tango Therapeutics, Inc.

location_country:
United States
France

location_facility: The University of Texas MD Anderson Cancer Center


title: A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid

summary:
ARGX-113-2009 is an operationally seamless 2-part, phase 2/3, prospective, global, multicenter, randomized, double-blinded, placebo-controlled study to investigate the efficacy, safety, tolerability, immunogenicity, participant-reported outcome measures (including those assessing participant QoL), PK, and PD of efgartigimod PH20 SC administered via subcutaneous (SC) injection in adult participants with moderate to severe BP. This study intends to demonstrate that efgartigimod is an effective and safe treatment for BP, providing participants with control of disease activity (CDA) and eventually remission while reducing their cumulative exposure to OCS.

study will consist of 2 parts:

Part A of the study is a phase 2 evaluation that intends to provide proof of concept for the therapeutic activity of efgartigimod PH20 SC in participants with BP.
Part B of the study is a phase 3 evaluation that intends to confirm the results obtained from part A in a separate, larger group of participants with BP.

An interim analysis will be performed during part A (on data obtained through week 26 for all Part A participants) to assess the primary endpoint and several secondary endpoints, confirm the appropriate sample size for part B of the study, and determine whether the efficacy results observed through week 26 of part A warrant continued study of efgartigimod PH20 SC for the treatment of participants with BP (futility analysis).

Other than differences in main goals, endpoints, and statistical analyses, parts A and B are identical in schedule, structure, assessments, and conduct.

lead_sponsor: argenx

location_country:
Germany
Poland
Australia
Japan
Croatia
Czechia
Serbia
France
Slovakia
Hungary
United States
Latvia
Romania
China
Italy
United Kingdom
Netherlands
Spain
Greece
Bulgaria
Israel

location_facility:
Investigator site 14 - BG3590010
Investigator site 116 - CN0860027
Investigator site 29 - FR0330029
Investigator site 115 - US0010157
Investigator site 72 - US0010186
Investigator site 41 - IL9720001
Investigator site 82 - LV3710003
Investigator site 92 - US0010150
Investigator site 2 - US0010087
Investigator site 23 - IT0390006
Investigator site 86 - IT0390067
Investigator site 49 - ES0340058
Investigator site 8 - ES0340053
Investigator site 100 - JP0810046
Investigator site 90 - RO0400014
Investigator site 114 - SK4210003
Investigator site 35 - US0010156
Investigator site 63 - IL9720018
Investigator site 5 - US0010088
Investigator site 50 - US0010149
Investigator site 45 - DE0490001
Investigator site 59 - GR0300005
Investigator site 11 - HU0360003
Investigator site 27 - AU0610006
Investigator site 67 - RS3810012
Investigator site 91 - CN0860017
Investigator site 71 - UK0440036
Investigator site 106 - JP0810068
Investigator site 10 - US0010153
Investigator site 122 - CN0860065
Investigator site 56 - DE0490028
Investigator site 98 - JP0810043
Investigator site 88 - LV3710004
Investigator site 117 - CZ4200013
Investigator site 119 - IT0390066
Investigator site 113 - SK4210002
Investigator site 110 - CN0860053
Investigator site 37 - HR3850001
Investigator site 107 - CN0860021
Investigator site 4 - US0010137
Investigator site 58 - GR0300001
Investigator 24 - ES0340051
Investigator site 51 - DE0490008
Investigator site 77 - IT0390062
Investigator site 95 - CN0860018
Investigator site 52 - DE0490024
Investigator site 74 - US0010178
Investigator site 81 - IT0390030
Investigator site 103 - JP0810070
Investigator site 6 - US0010138
Investigator site 47 - IT0390031
Investigator site 9 - ES0340052
Investigator site 99 - JP0810050
Investigator site 105 - JP0810067
Investigator site 28 - BG3590018
Investigator site 84 - RS3810010
Investigator site 46 - DE0490039
Investigator site 123 - CN0860095
Investigator site 48 - ES0340059
Investigator 68 - RS381011
Investigator site 12 - ES0340025
Investigator site 79 - PL0480025
Investigator site 17 - PL0480047
Investigator site 80 - DE0490041
Investigator site 34 - US0010182
Investigator site 120 - SK4210004
Investigator site 21 - US0010152
Investigator 20 - ES0340029
Investigator site 60 - GE0300004
Investigator site 22 - HR3850003
Investigator site 38 - FR0330040
Investigator site 97 - CZ4200015
Investigator site 93 - US0010169
Investigator site 87 - LV3710005
Investigator site 19 - PL0480046
Investigator site 57 - DE0490046
Investigator site 126 - CN0860026
Investigator site 121 - US0010092
Investigator site 111 - CN0860064
Investigator site 33 - UK0440022
Investigator site 55 - DE0490026
Investigator site 43 - IT0390060
Investigator site 25 - US0010158
Investigator site 7 - HU0360008
Investigator site 101 - JP0810049
Investigator site 70 - ES0340061
Investigator site 16 - HR3850002
Investigator site 42 - IT0390005
Investigator site 78 - IT0390039
Investigator site 31 - ES0340057
Investigator site 3 - US0010151
Investigator site 112 - JP0810045
Investigator site 83 - PL0480050
Investigator site 73 - US0010098
Investigator site 118 - CZ4200016
Investigator site 127 - CN860020
Investigator site 75 - DE0490047
Investigator site 89 - RO0400015
Investigator site 15 - BG3590020
Investigator site 32 - ES0340050
Investigator site 108 - CN0860023
Investigator site 39 - IL9720003
Investigator site 69 - RS3810009
Investigator site 125 - AU0610019
Investigator site 54 - DE0490030
Investigator site 1 - US0010017
Investigator site 30 - IT0390040
Investigator site 18 - PL0480048
Investigator site 62 - GE0300006
Investigator site 53 - DE0490023
Investigator site 104 - JP0810071
Investigator site 66 - NL0310015
Investigator site 44 - UK0440037
Investigator site 76 - GR030003
Investigator site 94 - CN0860066
Investigator site 64 - IT0390061
Investigator site 109 - CN0860025
Investigator site 61 - GE0300002
Investigator site 124 - CN0860098
Investigator site 102 - JP0810069
Investigator site 26 - HU0360023
Investigator site 13 - US0010155
Investigator site 40 - IL9720002
Investigator site 96 - CZ4200012
Investigator site 85 - US0010159
Investigator site 65 - IT0390055
Investigator site 36 - AU0610013


title: A Phase II, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in AS Patients With Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients With Primary Steroid-Resistant FSGC

summary: This is a Phase 2, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in Alport Syndrome Patients with Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients with Primary Steroid-Resistant Focal Segmental Glomerulosclerosis

lead_sponsor: River 3 Renal Corp.

location_country:
Germany
United Kingdom
Netherlands
Belgium
United States
France

location_facility:
Investigative Site
Investigative site


title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study With an Open-Label Extension Assessing the Efficacy, Safety, and Pharmacokinetics/Pharmacodynamics of Tirzepatide in Pediatric and Adolescent Participants With Type 2 Diabetes Mellitus Inadequately Controlled With Metformin, or Basal Insulin, or Both

acronym: SURPASS-PEDS

summary:
The purpose of this study is to learn more about the safety and efficacy of tirzepatide compared to placebo in children or teenagers with type 2 diabetes taking metformin, or basal insulin, or both.

The overall study will last about 60 weeks with up to 14 clinic visits and 6 phone visits. Clinic visits will include blood sample collection, physical exam and questionnaire.

ipd_sharing_description: Anonymized individual patient level data will be provided in a secure access environment upon approval of a research proposal and a signed data sharing agreement.

lead_sponsor: Eli Lilly and Company

location_country:
Italy
India
Israel
United Kingdom
Australia
Brazil
Mexico
United States
France

location_facility: Postgraduate Institute of Medical Education & Research

location_facility: Instituto de Pesquisa clinica de Campinas

location_facility: Kovai Diabetes Speciality Center and Hospital

location_facility: Consultorio Médico de Endocrinología y Pediatría

location_facility: Bhakti Vedanta Hospital and Research Institute

location_facility: SUNY Downstate Health Sciences University

location_facility: Universita degli Studi della Campania Luigi Vanvitelli

location_facility: Indiana University Health University Hospital

location_facility: Children's Hospital of Philadelphia (CHOP)

location_facility: Azienda Ospedaliera Universitaria Integrata Verona - Ospedale Borgo Trento

location_facility: M S Ramaiah Medical College and Hospitals

location_facility: Center Of Excellence in Diabetes and Endocrinology

location_facility: Instituto da Crianca do Hospital das Clinicas da FMUSP

location_facility: Assistance Publique - Hopitaux de Paris (AP-HP) - Hopital Robert Debre - Centre Hospitalo Universitaire (CHU)

location_facility: Centre Hospitalier Universitaire d'Angers


title: A Phase 2, Open-Label, Single-Arm, Multicenter Study to Evaluate the Efficacy and Safety of Pemigatinib in Participants With Advanced Non-Small Cell Lung Cancer With an FGFR Alteration Who Progressed on Previous Therapy (FIGHT 210)

summary: This is an open-label, single arm study to study the safety, efficacy and tolerability of Pemigatinib when used on participants with squamous or nonsquamous NSCLC with a documented FGFR1-3 mutations or fusions/rearrangement who have progressed on prior therapies and have no available standard treatment options

status: Completed

ipd_sharing_description: Incyte shares data with qualified external researchers after a research proposal is submitted. These requests are reviewed and approved by a 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. The trial data availability is according to the criteria and process described on https://www.incyte.com/our-company/compliance-and-transparency

lead_sponsor: Incyte Corporation

location_country:
Germany
Italy
United States
Spain
France

location_facility: Florida Cancer Specialists & Research Institute

location_facility: Hospital Regional Universitario de Malaga

location_facility: Azienda Ospedaliera Di Perugia - Ospedale Santa Maria Della Misericordia

location_facility: Istituto Tumori Giovanni Paolo Ii Irccs Ospedale Oncologico Bari

location_facility: Chu de Toulouse Hopital Larrey Centre de Reference Des Maladies Rares de La Peau Service de Dermatol

location_facility: Ico Girona Hospital Universitari de Girona Dr Josep Trueta

location_facility: Azienda Ospedaliera Universitaria San Luigi Gonzaga Orbassano

location_facility: Hospital General Universitario Gregorio Maranon

location_facility: Istituto Romagnolo Per Lo Studio Dei Tumori Dino Amadori

location_facility: Complejo Hospitalario Universitario A Coruna

location_facility: Hospital General Universitario Vall D Hebron

location_facility: Azienda Ospedaliero Universitaria Pisana

location_facility: Istituto Nazionale Tumori Regina Elena Irccs

status_simplified: Completed


title: Aveir Dual-Chamber Leadless i2i IDE Study

summary: Prospective, non-randomized, multi-center, international study designed to evaluate the safety and effectiveness of the Aveir™ Dual-Chamber (DR) Leadless Pacemaker system.

ipd_sharing_description: Any de-identified individual participant data collected will be shared with investigators or other study committee members whose proposed use of the data has been approved by the Aveir DR i2i Study Steering Committee.

lead_sponsor: Abbott Medical Devices

location_country:
Italy
United Kingdom
Hong Kong
Japan
Netherlands
Austria
Belgium
United States
Spain
Taiwan
Czechia
Canada
France

location_facility: New York Presbyterian Hospital/Cornell University

location_facility: Kaiser Permanente Los Angeles Medical Center

location_facility: Prairie Education & Research Cooperative

location_facility: Wake Forest University Medical Center Clinical Sciences

location_facility: New York-Presbyterian/Columbia University Medical Center

location_facility: Institut de Cardiologie de Montreal (Montreal Heart Inst.)

location_facility: University of California at San Diego (UCSD) Medical Center

location_facility: Donald Guthrie Foundation for Education & Research

location_facility: Kansas City Cardiac Arrhythmia Research Foundation

location_facility: National Cerebral & Cardiovascular Center Hospital


title: A Phase 1b, Single Ascending Dose, Randomized, Double-blind Study to Evaluate the Safety, Tolerability, and Activity of SAR439459 in Adults With Osteogenesis Imperfecta

summary:
SAR439459 is a human anti-Transforming growth factor β (TGFβ) monoclonal antibody. This phase 1 clinical study investigates the safety, tolerability, and activity of a single dose of SAR439459 in adult participants with OI.

Participants will receive a single IV dose of SAR439459 with safety, pharmacokinetic (PK), and pharmacodynamic (PD) assessments over 24 weeks.

There will be up to 3 dose cohorts. In addition to safety, tolerability, and PK assessments, bone mineral density (BMD) will be evaluated by dual-energy Xray absorptimetry (DXA) scan and a series of blood biomarkers will be monitored to document pharmacodynamic effects of the single dose of SAR439459.

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:
United States
Australia
Canada
France

location_facility: Department of Medicine/ School of Clinical Sciences at Monash Health Monash University_246 Clayton Road_Site Number :0360002

location_facility: Bone Research and Education Centre_Site Number :1240003

location_facility: Hopital Lariboisiere_Site Number :2500001

location_facility: Toronto general Hospital_Site Number :1240002

location_facility: Baylor College of Medicine - Site Number:8400003

location_facility: Hopital Edouard Herriot _Site Number :2500002

location_facility: Vanderbilt University Site Number : 8400011

location_facility: Kennedy Krieger Institute_Site number 8400004

location_facility: Indiana University School of Medicine_Site Number: 8400002


summary: This is a parallel arm, Phase 3, double-blind, double-dummy, active-comparator, 2 arm study to evaluate the efficacy and safety of daily oral venglustat versus intravenous Cerezyme infusions every two weeks for improvement or stabilization of the neurological manifestations and maintenance of systemic disease stability in participants aged ≥12 and <18 years and adult patients with Gaucher disease Type 3 (GD3) who have been treated with Enzyme Replacement Therapy (ERT) for at least 3 years.

location_facility: Hopital Necker - Investigational Site Number: 2500001

location_facility: Lysosomal and Rare Disorders Research and Treatment Center, Inc - Investigational Site Number: 84000

location_facility: University of Iowa - Investigational Site Number: 8400002

location_facility: Children's Hospital Research Institute of Manitoba - Investigational Site Number: 1240001

location_facility: Yale University School of Medicine - Investigational Site Number: 8400003

location_facility: Peking Union Medical College Hospital - Investigational Site Number: 1560001

location_facility: 47-87, boulevard de l'hôpital - Investigational Site Number: 2500003

location_facility: Debreceni Egyetem, Klinikai Központ, Reumatológiai Klinika - Investigational Site Number: 3480001

location_facility: Hospital de Ninos - Investigational Site Number: 320001

location_facility: Xinhua Hospital - Investigational Site Number: 1560004

location_facility: The First Affiliated Hospital - Investigational Site Number: 1560002

location_facility: SphinCS GmbH - Investigational Site Number: 2760001

location_facility: Azienda Ospedaliera Universitaria (AOU) "Federico II" - Investigational Site Number: 3800002

location_facility: Tohoku University School of Medicine - Investigational Site Number: 3920001

location_facility: National Taiwan University Hospital-Investigational Site Number: 1580001

title: A Phase 3, Multicenter, Multinational, Randomized, Double-blind, Double-dummy, Active-comparator Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 (GD3) Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ERT)

location_country:
Germany
Italy
Hungary
Argentina
Japan
United States
China
Turkey
Canada
France


title: A Phase I/II Open-label, Multi-center Study to Assess Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of AZD7789, an Anti-PD-1 and Anti-TIM-3 Bispecific Antibody, in Patients With Relapsed or Refractory Classical Hodgkin Lymphoma.

summary: The study is intended to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of AZD7789 in patients with relapsed/refractory classical Hodgkin Lymphoma (r/r cHL).

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 requests 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.

sponsor_collaborators: AstraZeneca Parexel

location_country:
Germany
Italy
United Kingdom
Denmark
United States
China
Spain
Canada
France


title: ENCORAFENIB/BINIMETINIB MASTER PROTOCOL: AN OPEN-LABEL CONTINUATION STUDY FOR PARTICIPANTS CONTINUING FROM ENCORAFENIB/BINIMETINIB CLINICAL STUDIES

summary:
The purpose of this clinical trial (called the FLOTILLA study) is to give continued access to the study medicines, as well as safety follow-up, for participants in prior clinical trials of encorafenib and/or binimetinib.

All participants who took part in earlier encorafenib and/or binimetinib studies may participate the FLOTILLA study if they are still benefiting from the use of the study medicines. This will be determined by the study doctor. People may not participate in the FLOTILLA study if they have not enrolled in a prior study of encorafenib or binimetinib.

Participants that had enrolled but had stopped receiving the study treatment in a prior study cannot enrolled in this study. Participants in the FLOTILLA study will receive encorafenib and/or binimetinib at the same dose and frequency as in their prior study, for up to about 5 years.

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.

lead_sponsor: Pfizer

location_country:
Germany
Italy
Portugal
Slovakia
Hungary
Israel
United Kingdom
Australia
Brazil
Japan
Netherlands
United States
Korea, Republic of
Spain
Czechia
Canada
France

location_facility: Severance Hospital, Yonsei University Health System

location_facility: Hospital General Universitario Gregorio Marañon

location_facility: Hospital Universitario Puerta de Hierro Majadahonda

location_facility: BP - A Beneficencia Portuguesa de São Paulo

location_facility: Rosemere Cancer Centre - Royal Preston Hospital

location_facility: Charité Universitaetsmedizin Berlin - Campus Mitte

location_facility: Instituto Português de Oncologia de Lisboa Francisco Gentil

location_facility: Institut Català d'Oncologia (ICO) - Badalona

location_facility: Rockefeller Outpatient Pavilion (53rd Street)

location_facility: Real e Benemerita Associacao Portuguesa de Beneficencia

location_facility: Fundação Pio XII - Hospital de Câncer de Barretos

location_facility: St. Joseph's Hospital and Medical Center

location_facility: Hospital Universitario Central de Asturias

location_facility: Istituto Nazionale Tumori IRCCS Fondazione Pascale

location_facility: ONCOSITE - Centro de Pesquisa Clinica em Oncologia

location_facility: Instituto Português de Oncologia do Porto Francisco Gentil, EPE

location_facility: Hospital Universitario Arnau de Vilanova de Lleida


title: A Multi-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Clinical Study of The Safety and Efficacy of CGT9486 in Subjects With Nonadvanced Systemic Mastocytosis

summary: This is a multi-part, randomized, double-blind, placebo-controlled Phase 2 clinical study comparing the safety and efficacy of bezuclastinib (CGT9486) plus best supportive care (BSC) with placebo plus BSC in patients with nonadvanced systemic mastocytosis (NonAdvSM), including indolent systemic mastocytosis and smoldering systemic mastocytosis, whose symptoms are not adequately controlled by BSC. This study will be conducted in three parts. Patients in Parts 1a, 1b and 2 will receive bezuclastinib or placebo, and may roll over onto Part 3 to receive treatment with bezuclastinib.

lead_sponsor: Cogent Biosciences, Inc.

location_country:
Germany
Italy
Norway
Australia
Netherlands
United States
Spain
Canada
France

location_facility: Mid Florida Hematology and Oncology Center

location_facility: Policlinico Universitario Agostino Gemelli

location_facility: IRCCS Azienda Ospedaliero-Universitaria di Bologna - Policlinico di Sant'Orsola

location_facility: Walter Reed National Military Medical Center

location_facility: The University of Texas MD Anderson Cancer Center


title: A Dose-Escalation and Expansion Study of the Safety and Efficacy of XL092 in Combination With Immuno-Oncology Agents in Subjects With Unresectable Advanced or Metastatic Solid Tumors

summary:
This is a multicenter Phase 1b, open label, dose-escalation and cohort-expansion study, evaluating the safety, tolerability, PK, preliminary antitumor activity, and effect of biomarkers of XL092 administered alone, and in combination with nivolumab (doublet), nivolumab + ipilimumab (triplet) and nivolumab + relatlimab (triplet) in subjects with advanced solid tumors.

In the Expansion Stage, the safety and efficacy of XL092 as monotherapy and in combination therapy will be further evaluated in tumor-specific Expansion Cohorts.

lead_sponsor: Exelixis

location_country:
Italy
Poland
New Zealand
Israel
Australia
Switzerland
Austria
Belgium
United States
Spain
France

location_facility:
Exelixis Clinical Site #40
Exelixis Clinical Site #34
Exelixis Clinical Site #11
Exelixis Clinical Site #3
Exelixis Clinical Site #29
Exelixis Clinical Site #54
Exelixis Clinical Site #19
Exelixis Clinical Site #21
Exelixis Clinical Site #70
Exelixis Clinical Site #38
Exelixis Clinical Site #26
Exelixis Clinical Site #8
Exelixis Clinical Site #1
Exelixis Clinical Site #16
Exelixis Clinical Site #69
Exelixis Clinical #15
Exelixis Clinical Site #62
Exelixis Clinical Site #17
Exelixis Clinical Site #39
Exelixis Clinical Site #58
Exelixis Clinical Site #28
Exelixis Clinical Site #18
Exelixis Clinical Site #13
Exelixis Clinical Site #43
Exelixis Clinical Site #48
Exelixis Clinical Site #12
Exelixis Clinical Site #14
Exelixis Clinical Site #35
Exelixis Clinical Site #63
Exelixis Clinical Site #45
Exelixis Clinical Site #60
Exelixis Clinical Site #32
Exelixis Clinical Site #42
Exelixis Clinical Site #25
Exelixis Clinical Site #68
Exelixis Clinical Site #41
Exelixis Clinical Site #44
Exelixis Clinical Site #55
Exelixis Clinical Site #57
Exelixis Clinical Site #49
Exelixis Clinical Site #10
Exelixis Clinical Site #23
Exelixis Clinical Site #53
Exelixis Clinical Site #64
Exelixis Clinical Site #59
Exelixis Clinical Site #22
Exelixis Clinical Site #20
Exelixis Clinical Site #36
Exelixis Clinical Site #6
Exelixis Clinical Site #37
Exelixis Clinical Site #72
Exelixis Clinical Site #56
Exelixis Clinical Site #33
Exelixis Clinical Site #9
Exelixis Clinical Site #66
Exelixis Clinical Site #24
Exelixis Clinical Site #67
Exelixis Clinical Site #4
Exelixis Clinical Site #65
Exelixis Clinical Site #52
Exelixis Clinical Site #31
Exelixis Clinical Site #7
Exelixis Clinical Site #5
Exelixis Clinical Site #51
Exelixis Clinical Site #71
Exelixis Clinical Site #50
Exelixis Clinical Site #61
Exelixis Clinical Site #30
Exelixis Clinical Site #46
Exelixis Clinical Site #2
Exelixis Clinical Site #27
Exelixis Clinical Site #47


title: Single Blind Randomized Controlled Trial to Assess the Safety and Efficacy of High Dose Pulse Intravenous Corticosteroid Therapy to Treat Patients With Complicated/Fulminant Acute Myocarditis

acronym: MYTHS

summary: This is a phase III, multi-center international, single blind randomized controlled trial to test the efficacy of pulsed intravenous (IV) methylprednisolone versus standard therapy on top of maximal support in patients with Acute myocarditis (AM).

ReferenceCitation: Ammirati E, Frigerio M, Adler ED, Basso C, Birnie DH, Brambatti M, Friedrich MG, Klingel K, Lehtonen J, Moslehi JJ, Pedrotti P, Rimoldi OE, Schultheiss HP, Tschope C, Cooper LT Jr, Camici PG. Management of Acute Myocarditis and Chronic Inflammatory Cardiomyopathy: An Expert Consensus Document. Circ Heart Fail. 2020 Nov;13(11):e007405. doi: 10.1161/CIRCHEARTFAILURE.120.007405. Epub 2020 Nov 12.

ReferenceCitation: Kociol RD, Cooper LT, Fang JC, Moslehi JJ, Pang PS, Sabe MA, Shah RV, Sims DB, Thiene G, Vardeny O; American Heart Association Heart Failure and Transplantation Committee of the Council on Clinical Cardiology. Recognition and Initial Management of Fulminant Myocarditis: A Scientific Statement From the American Heart Association. Circulation. 2020 Feb 11;141(6):e69-e92. doi: 10.1161/CIR.0000000000000745. Epub 2020 Jan 6.

ReferenceCitation: Ammirati E, Cipriani M, Moro C, Raineri C, Pini D, Sormani P, Mantovani R, Varrenti M, Pedrotti P, Conca C, Mafrici A, Grosu A, Briguglia D, Guglielmetto S, Perego GB, Colombo S, Caico SI, Giannattasio C, Maestroni A, Carubelli V, Metra M, Lombardi C, Campodonico J, Agostoni P, Peretto G, Scelsi L, Turco A, Di Tano G, Campana C, Belloni A, Morandi F, Mortara A, Ciro A, Senni M, Gavazzi A, Frigerio M, Oliva F, Camici PG; Registro Lombardo delle Miocarditi. Clinical Presentation and Outcome in a Contemporary Cohort of Patients With Acute Myocarditis: Multicenter Lombardy Registry. Circulation. 2018 Sep 11;138(11):1088-1099. doi: 10.1161/CIRCULATIONAHA.118.035319.

ReferenceCitation: Ammirati E, Veronese G, Brambatti M, Merlo M, Cipriani M, Potena L, Sormani P, Aoki T, Sugimura K, Sawamura A, Okumura T, Pinney S, Hong K, Shah P, Braun O, Van de Heyning CM, Montero S, Petrella D, Huang F, Schmidt M, Raineri C, Lala A, Varrenti M, Foa A, Leone O, Gentile P, Artico J, Agostini V, Patel R, Garascia A, Van Craenenbroeck EM, Hirose K, Isotani A, Murohara T, Arita Y, Sionis A, Fabris E, Hashem S, Garcia-Hernando V, Oliva F, Greenberg B, Shimokawa H, Sinagra G, Adler ED, Frigerio M, Camici PG. Fulminant Versus Acute Nonfulminant Myocarditis in Patients With Left Ventricular Systolic Dysfunction. J Am Coll Cardiol. 2019 Jul 23;74(3):299-311. doi: 10.1016/j.jacc.2019.04.063.

ReferenceCitation: Chen H, Liu J, Yang M. Corticosteroids for viral myocarditis. Cochrane Database Syst Rev. 2006 Oct 18;(4):CD004471. doi: 10.1002/14651858.CD004471.pub2.

ReferenceCitation: Mason JW, O'Connell JB, Herskowitz A, Rose NR, McManus BM, Billingham ME, Moon TE. A clinical trial of immunosuppressive therapy for myocarditis. The Myocarditis Treatment Trial Investigators. N Engl J Med. 1995 Aug 3;333(5):269-75. doi: 10.1056/NEJM199508033330501.

ReferenceCitation: Ammirati E, Cipriani M, Lilliu M, Sormani P, Varrenti M, Raineri C, Petrella D, Garascia A, Pedrotti P, Roghi A, Bonacina E, Moreo A, Bottiroli M, Gagliardone MP, Mondino M, Ghio S, Totaro R, Turazza FM, Russo CF, Oliva F, Camici PG, Frigerio M. Survival and Left Ventricular Function Changes in Fulminant Versus Nonfulminant Acute Myocarditis. Circulation. 2017 Aug 8;136(6):529-545. doi: 10.1161/CIRCULATIONAHA.117.026386. Epub 2017 Jun 2.

ReferenceCitation: Ammirati E, Moslehi JJ. Diagnosis and Treatment of Acute Myocarditis: A Review. JAMA. 2023 Apr 4;329(13):1098-1113. doi: 10.1001/jama.2023.3371.

lead_sponsor: Niguarda Hospital

collaborators: Istituto Di Ricerche Farmacologiche Mario Negri

location_country:
Finland
Germany
Italy
Slovenia
Denmark
Sweden
Belgium
United States
Spain
Greece
Czechia
France

location_facility: Hospital Universitario Virgen de la Victoria

location_facility: Hospital Universitario Puerta de Hierro Majadahonda

location_facility: Institute for Clinical and Experimental Medicine - IKEM

location_facility: Hospices Civils de Lyon, Lyon 08, Rhône-Alpes

location_facility: Charité - University Medicine Berlin, Campus Virchow Klinikum

location_facility: Presidio Ospedaliero Universitario "Santa Maria della Misericordia"

location_facility: Rigshospitalet and University of Copenhagen

location_facility: Charles University in Prague and General University Hospital

location_facility: ASST Grande Ospedale Metropolitano Niguarda

location_facility: Hospital General Universitario Gregorio Marañón in Madrid

location_facility: Université Nantes, CHU Nantes, CNRS, INSERM, l'institut du thorax

location_facility: Azienda Ospedaliera Universitaria Senese, Policlinico Santa Maria alle Scotte

location_facility: Azienda Ospedaliero Universitaria di Parma

location_facility: Complexo Hospitalario Universitario A Coruña (CHUAC)

location_facility: Masaryk University and St. Anne's University Hospital

location_facility: Azienda Ospedaliera Specialistica dei Colli - Ospedale Monaldi

location_facility: Azienda Ospedaliera San Camillo Forlanini di Roma

location_facility: AOU Ospedali Riuniti Umberto I°-Lancisi-Salesi di Ancona

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

location_facility: Fondazione Policlinico Universitario Agostino Gemelli IRCCS

location_facility: Hospital Universitario Virgen de la Arrixaca

location_facility: Lund University and Skåne University Hospital

location_facility: Heart and Lung Center, Helsinki University Hospital

lead_sponsor_type: academique


title: A Phase 2 Open-label Clinical Study to Evaluate the Efficacy and Safety of Zilovertamab Vedotin (MK-2140) in Participants With Relapsed or Refractory Diffuse Large B-Cell Lymphoma (waveLINE-004)

summary: The purpose of this study is to evaluate zilovertamab vedotin with respect to objective response rate and duration of response per Lugano Response Criteria as assessed by blinded independent central review (BICR). Safety and tolerability will also be evaluated in this Phase 2, single arm, interventional study.

lead_sponsor: Merck Sharp & Dohme LLC

location_country:
Italy
Poland
Israel
Norway
Sweden
United States
China
Korea, Republic of
Spain
Greece
Thailand
Chile
Turkey
Czechia
Canada
Estonia
France

location_facility: Haukeland Universitetssjukehus ( Site 1601)

location_facility: University of Chicago Medical Center ( Site 0207)

location_facility: Innovative Clinical Research Institute ( Site 0202)

location_facility: Hacettepe Universitesi-Department of Hematology ( Site 2302)

location_facility: Fondazione IRCCS Istituto Nazionale dei Tumori-Struttura Complessa di Ematologia ( Site 1501)

location_facility: Vseobecna fakultni nemocnice v Praze-I. Interní klinika - klinika hematologie ( Site 0801)

location_facility: Skånes Universitetssjukhus Lund ( Site 2100)

location_facility: Princess Margaret Cancer Centre-Division of Medical Oncology and Hematology ( Site 0100)

location_facility: Soroka Medical Center-Hematology Department ( Site 1403)

location_facility: Karolinska Universitetssjukhuset Solna ( Site 2102)

location_facility: Clínica Alemana de Santiago ( Site 2704)

location_facility: Maharaj Nakorn Chiang Mai Hospital ( Site 0702)

location_facility: General Hospital of Athens "Laiko"-Hematology Department ( Site 1213)

location_facility: Massachusetts General Hospital-Cancer Center Protocol Office ( Site 0203)

location_facility: Dokuz Eylül Üniversitesi-Hematology ( Site 2304)

location_facility: Pitie Salpetriere University Hospital-Clinical haematology ( Site 1000)

location_facility: Shaare Zedek Medical Center ( Site 1404)

location_facility: Franciscan St. Francis Health ( Site 0225)

location_facility: Atlantic Health System Morristown Medical Center ( Site 0213)

location_facility: The James Cancer Hospital and Solove Research Institute at The Ohio State University Comprehensive C

location_facility: Instituto Catalan de Oncologia - Hospital Duran i Reynals-Haematology Department ( Site 2002)

location_facility: Fudan University Shanghai Cancer Center ( Site 2908)

location_facility: MEDICAL COLLEGE OF WISCONSIN ( Site 0234)

location_facility: Georgetown University Medical Center ( Site 0204)

location_facility: Avera Cancer Institute- Research ( Site 0233)

location_facility: James Lind Centro de Investigación del Cáncer ( Site 2705)

location_facility: University of Maryland-Greenebaum Comprehensive Cancer Center ( Site 0211)

location_facility: Ankara University Hospital Cebeci-hematology ( Site 2300)

location_facility: Evangelismos General Hospital of Athens ( Site 1214)

location_facility: Hospital Universitario de Salamanca - Complejo Asistencial Universitario de Salamanca ( Site 2003)

location_facility: Oslo universitetssykehus, Radiumhospitalet ( Site 1600)

location_facility: Severance Hospital, Yonsei University Health System-Medical oncology ( Site 0601)

location_facility: Saint Louis University Cancer Center ( Site 0209)

location_facility: The First Hospital of Jilin University-Hematology ( Site 2910)

location_facility: Hospital Universitari Vall d'Hebron ( Site 2005)

location_facility: University of Cincinnati Medical Center-University of Cincinnati Cancer Center ( Site 0217)

location_facility: Fakultní nemocnice Brno Bohunice-Interni hematologicka a onkologicka klinika ( Site 0800)

location_facility: North Estonia Medical Centre Foundation ( Site 0900)

location_facility: Henan Cancer Hospital-hematology department ( Site 2903)

location_facility: Istituto Nazionale Tumori IRCCS Fondazione Pascale ( Site 1502)

location_facility: The First Affiliated Hospital, Zhejiang University-Bone marrow transplant centre ( Site 2912)

location_facility: St. Joseph Hospital-The Center for Cancer Prevention and Treatment ( Site 0229)

location_facility: Karadeniz Teknik Universitesi Tip Fakultesi-Hematology ( Site 2307)

location_facility: Uniwersytecki Szpital Kliniczny-Klinika Hematologii, Nowotworow Krwi i Transplantacji Szpiku ( Site

location_facility: University Hospitals Cleveland Medical Center ( Site 0222)

location_facility: Humanitas-U.O di Oncologia medica ed Ematologia ( Site 1503)

location_facility: West China Hospital of Sichuan University-Head and Neck Oncology ( Site 2911)

location_facility: IRCCS - AOU di Bologna-Istituto di Ematologia "L. e A. Seragnoli" ( Site 1500)

location_facility: Szpitale Pomorskie Sp. z o. o.-Hematology and Bone Marrow Transplantation Department ( Site 1702)

location_facility: Hospital Universitario Fundación Jiménez Díaz-Oncology & Hematology ( Site 2000)

location_facility: Tianjin Medical University Cancer Institute and Hospital-lymphoma ( Site 2901)

location_facility: SUN YAT-SEN UNIVERSITY CANCER CENTRE-Internal Medicine ( Site 2907)

location_facility: Centre Hospitalier de la Côte Basque ( Site 1002)

location_facility: Narodowy Instytut Onkologii im. Marii Sklodowskiej-Curie - P-Klinika Nowotworów Układu Chłonnego ( S

location_facility: Faculty of Medicine Siriraj Hospital ( Site 0701)

location_facility: Szpital Specjalistyczny im. Ludwika Rydygiera w Krakowie-Oncology Department ( Site 1707)


title: A Phase 2/3 Multicenter, Open-label, Randomized, Active-Control Study of Zilovertamab Vedotin (MK-2140) in Combination With Standard of Care in Participants With Relapsed or Refractory Diffuse Large B-Cell Lymphoma (waveLINE-003)

summary: The purpose of this Phase 2/3, randomized, multisite, open-label, dose confirmation, and expansion study is to evaluate the safety, and efficacy of zilovertamab vedotin (ZV) in combination with standard of care options for the treatment of rrDLBCL. This study will be divided into 2 parts: Dose Confirmation (Part 1) and Efficacy Expansion (Part 2) and will enroll participants who are at least 18 years of age with rrDLBCL. The hypotheses are: ZV in combination with rituximab, gemcitabine, and oxaliplatin (R-GemOx) is superior to R-GemOx with respect to progression-free survival (PFS) per Lugano response criteria by blinded independent review committee (BICR); and that ZV in combination with bendamustine rituximab (BR) is superior to BR with respect to PFS per Lugano response criteria by BICR.

location_country:
France
Italy
Poland
United Kingdom
United States
China
Korea, Republic of
Thailand
Turkey
Canada
Israel

location_facility: Ankara University Hospital Cebeci-hematology ( Site 1901)

location_facility: Humanitas-U.O di Oncologia medica ed Ematologia ( Site 1203)

location_facility: Chongqing University Cancer Hospital-Medical Oncology ( Site 3025)

location_facility: Istituto Nazionale Tumori IRCCS Fondazione Pascale ( Site 1202)

location_facility: Maharaj Nakorn Chiang Mai Hospital ( Site 0401)

location_facility: St. Vincent Frontier Cancer Center-Research ( Site 0108)

location_facility: centre hospitalier lyon sud-Service Hématologie ( Site 0702)

location_facility: Vanderbilt University Medical Center-Vanderbilt-Ingram Cancer Center ( Site 0156)

location_facility: Emek Medical Center-Hematology Unit ( Site 1102)

location_facility: University of Massachusetts Medical School ( Site 0119)

location_facility: Princess Margaret Cancer Centre-Division of Medical Oncology and Hematology ( Site 0200)

location_facility: Union Hospital Tongji Medical College Huazhong University of Science and Technology ( Site 3017)

location_facility: Ege University Medicine of Faculty ( Site 1902)

location_facility: Dokuz Eylül Üniversitesi-Hematology ( Site 1905)

location_facility: Faculty of Medicine Siriraj Hospital-Division of Hematology, Department of Medicine ( Site 0400)

location_facility: Szpitale Pomorskie Sp. z o. o.-Hematology and Bone Marrow Transplantation Department ( Site 1302)

location_facility: Dana-Farber Cancer Institute-Lymphoma ( Site 0111)

location_facility: Pitie Salpetriere University Hospital-Clinical haematology ( Site 0700)

location_facility: Louisiana State University Health Sciences Center New Orleans ( Site 0134)

location_facility: Innovative Clinical Research Institute ( Site 0122)

location_facility: University College London Hospital-Cancer Clinical Trials Unit ( Site 2100)

location_facility: Centrum Onkologii Ziemi Lubelskiej-Oddzial Hematologiczny ( Site 1304)

location_facility: Seoul National University Hospital-Oncology ( Site 0302)

location_facility: Norton Women's and Children's Hospital-Norton Cancer Institute - St. Matthews ( Site 0133)

location_facility: IRCCS - AOU di Bologna-Istituto di Ematologia "L. e A. Seragnoli" ( Site 1200)


title: A Phase 1/2 Study of the Highly Selective ROS1 Inhibitor NVL-520 in Patients With Advanced NSCLC and Other Solid Tumors (ARROS-1)

summary:
Phase 1/2, dose escalation and expansion study designed to evaluate the safety and tolerability of NVL-520, determine the recommended phase 2 dose (RP2D), and evaluate the antitumor activity in patients with advanced ROS1-positive (ROS1+) NSCLC and other advanced ROS1-positive solid tumors.

Phase 1 will determine the RP2D and, if applicable, the maximum tolerated dose (MTD) of NVL-520 in patients with advanced ROS1-positive solid tumors.

Phase 2 will determine the objective response rate (ORR) as assessed by Blinded Independent Central Review (BICR) of NVL-520 at the RP2D. Secondary objectives will include the duration of response (DOR), time to response (TTR), progression-free survival (PFS), overall survival (OS), and clinical benefit rate (CBR) of NVL-520 in patients with advanced ROS1-positive NSCLC and other solid tumors.

lead_sponsor: Nuvalent Inc.

location_country:
Singapore
Australia
Netherlands
Belgium
United States
Spain
Taiwan
Canada
France

location_facility: University of Washington / Fred Hutchinson Cancer Center

location_facility: NEXT Oncology - Virginia Cancer Specialists

location_facility: Washington University School of Medicine

location_facility: UOMI Cancer Center - Clinica Tres Torres