Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)

PHASE3CompletedINTERVENTIONAL
Enrollment

144

Participants

Timeline

Start Date

December 19, 2017

Primary Completion Date

November 16, 2020

Study Completion Date

November 20, 2024

Conditions
Hemophilia A
Interventions
BIOLOGICAL

valoctocogene roxaparvovec

Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A

Trial Locations (49)

27517

UNC Hemophilia and Thrombosis Center, Chapel Hill

33607

St. Joseph's Children's Hospital, Center for Bleeding and Clotting Disorders, Tampa

40202

James Graham Brown Cancer Center, Louisville

43205

Nationwide Children's Hospital, Columbus

48201

Wayne State University, Detroit Medical Center, Detroit

55455

University of Minnesota, Minneapolis

80045

University of Colorado, Aurora

92122

University of California San Diego, Hematology and Oncology, Hemophilia &Thrombosis Treatment Center, San Diego

95817

UC Davis Hemophilia Treatment Center, Sacramento

90007-2664

Los Angeles Orthopedic Hospital, Orthopedic Hemophilia Treatment Center, Los Angeles

94143-0106

UCSF Medical Center, San Francisco

60611-2605

Ann & Robert H. Lurie Children's Hospital of Chicago, Department of Hematology, Chicago

70112-2699

Tulane University Hematology & Medical Oncology, New Orleans

48109-5718

University of Michigan, Pediatric Hematology and Oncology, Ann Arbor

63110-1093

Washington University School of Medicine, Department of Pediatrics, Division of Hematology/Oncology, St Louis

Unknown

The Royal Adelaide Hospital (RAH), Adelaide

Royal Brisbane and Women's Hospital, Brisbane

Alfred Hospital, Melbourne

Fiona Stanley Hospital, Perth

Royal Prince Alfred Hospital, Sydney

University Hospital Leuven, Leuven

Campinas Estadual University (UNICAMP) / Campinas Hemocentro / Hematologia E Hemoterapia Center, Campinas

Parana's Hematology And Hemotherapy Center (HEMEPAR), Curitiba

Arthur De Siqueira Cavalcanti Hematology State Institute, Rio de Janeiro

Sao Paulo University Clinical Hospital, São Paulo

Holy Spirit Hematology and Hemotherapy Center, Vitória

Regional University Hospital of Lille (CHRU de Lille), Lille

Hopital de la Timone Marseille - Assistance Publique des Hopitaux de Marseille, Marseille

Vivantes Clinic im Friedrichshain- Landsberger Allee, Berlin

University Clinic Bonn, Bonn

Chaim Sheba Medical Center, Ramat Gan

Maggiore Polyclinic Hospital, IRCCS Ca' Granda, Center for Hemophilia and Thrombosis Angelo Bianchi Bonomi, Milan

Charlotte Maxeke Johannesburg Academic Hospital, Hemophilia Comprehensive Care Center, Johannesburg

Department of Pediatrics, Kyung Hee University Hospital at Gangdong, Seoul

Hospital Teresa Herrera, A Coruña

University Hospital Virgen del Rocio (HUVR), Seville

Changhua Christian Hospital, Changhua

Kaohsiung Medical University Chung-Ho Memorial Hospital, Kaohsiung City

Taichung Veterans General Hospital, Taichung

National Taiwan University Hospital, Taipei

Tri-Service General Hospital, Taipei

Queen Elizabeth Hospital, Birmingham

Addenbrookes Hospital, Cambridge

Glasgow Royal Infirmary, Department of Hematology, Glasgow

Barts and The London School of Medicine and Dentistry, Haemophilia Centre, London

Hammersmith, London

St Thomas' Hospital, London

Churchill Hospital, Oxford Hemophilia and Thrombosis Center, Oxford

University Hospital Southampton NHS Foundation Trust, Southampton

Sponsors
All Listed Sponsors
lead

BioMarin Pharmaceutical

INDUSTRY