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A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
Sponsor: BioMarin Pharmaceutical
Summary
The BMN 270 clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of BMN 270 for at least 5 years post-infusion. This long-term follow-up study is needed to help further understand the long-term safety of BMN 270 beyond 5 years and to assess the durability of efficacy.
Official title: A Long-Term Follow-Up Study in Subjects With Severe Hemophilia A Who Received BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in a Prior BioMarin Clinical Trial
Key Details
Gender
MALE
Age Range
18 Years - Any
Study Type
OBSERVATIONAL
Enrollment
172
Start Date
2023-01-01
Completion Date
2040-01
Last Updated
2026-01-12
Healthy Volunteers
No
Conditions
Locations (41)
Orthopedic Institute for Children, Orthopedic Hemophilia Treatment Center
Los Angeles, California, United States
UC Davis Hemophilia Treatment Center
Sacramento, California, United States
University of California San Diego, Hemophilia & Thrombosis Treatment Center
San Diego, California, United States
University of California San Francisco - Moffit Hospital, Outpatient Hematology Clinic
San Francisco, California, United States
UCN Hemophilia and Thrombosis Center
Shandon, California, United States
Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus
Aurora, Colorado, United States
St Joseph's Children's Hospital, Center for Bleeding and Clotting Disorders
Tampa, Florida, United States
University Medical Center, Inc,; DBA University of Louisville Hospital, James Graham Brown Cancer Center
Louisville, Kentucky, United States
University of Michigan, Pediatric Hematology and Oncology
Ann Arbor, Michigan, United States
Children's Hospital of Michigan, Hemophilia and Thrombosis Center
Detroit, Michigan, United States
Washington University School of Medicine, Department of Pediatrics, Division of Hematology/Oncology
St Louis, Missouri, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, United States
Royal Prince Alfred Hospital, Institute of Haematology
Sydney, New South Wales, Australia
Royal Brisbane and Women's Hospital
Herston, Queensland, Australia
Royal Adelaide Hospital
Adelaide, South Australia, Australia
The Alfred Hospital
Melbourne, Victoria, Australia
Fiona Stanley Hospital
Murdoch, Western Australia, Australia
University Hospitals Leuven, Campus Gasthuisberg, Department of Heart and Vascular Disease
Leuven, Belgium
Holy Spirit Hematology and Hemotherapy Center
Vitória, Espírito Santo, Brazil
Arthur De Siqueira Cavalcanti Hematology State Institute / Coordinating Hemocenter
Rio de Janeiro, Rio de Janeiro, Brazil
Campinas University Clinical Hospital, Hematology and Hemotherapy Center (Hemocentro UNICAMP)
Campinas, São Paulo, Brazil
Sao Paulo University Clinical Hospital
São Paulo, São Paulo, Brazil
CHRU Lille Hospital Center Heart-Lung Institute
Lille, France
CHU La Timone - La Timone Children's Hospital
Marseille, France
University Hospital Bonn, Institute of Experimental Hematology and Transfusion Medicine, Hemophilia Care Center
Bonn, North Rhine-Westphalia, Germany
Vivantes Hospital in Friedrichshain
Berlin, State of Berlin, Germany
Chaim Sheba Medical Center, National Institute of Hemophilia and Coagulation
Ramat Gan, Israel
Maggiore Polyclinic Hospital, Foundation IRCCS Ca' Granda, Center for Hemophilia and Thrombosis "Angelo Bianchi Bonomi"
Milan, Italy
Charlotte Maxeke Johannesburg Academic Hospital, Hemophilia Comprehensive Care Center
Johannesburg, South Africa
Kyung Hee University Hospital at Gangdong
Seoul, South Korea
University Hospital Virgen del Rocio (HUVR), Hematology Pediatrics
Seville, Andalusia, Spain
Hospital Teresa Herrera, Department of Hematology and Hemotherapy, Unit of Hemostasis and Thrombosis
A Coruña, Galicia, Spain
Addenbrooke's Hospital, Hemophilia and Thrombophilia Center
Cambridge, United Kingdom
Glasgow Royal Infirmary, Department of Hematology
Glasgow, United Kingdom
Hammersmith Hospital, Clinical Research Facility, Centre for Translational and Experimental Medicine
London, United Kingdom
Queen Elizabeth Hospital Birmingham. Adult Hemophilia Centre, West Midlands Adult Comprehensive Care Hemophilia Centre
London, United Kingdom
Royal London Hospital, Barts and the London Hemophilia Center
London, United Kingdom
St Thomas' Hospital, Centre for Hemophilia, Hemostasis and Thrombosis
London, United Kingdom
Nuffield Orthopaedic Centre, Hemophilia & Thrombosis Center
Oxford, United Kingdom
University Hospital Southampton NHS Foundation Trust
Southampton, United Kingdom