Decade-Long watch: tracking a One-Time gene therapy for a lifelong bleeding disorder
NCT ID NCT05768386
Summary
This study aims to track the long-term safety and durability of a one-time gene therapy (BMN 270) for severe Hemophilia A over approximately 10 years. It will follow about 172 patients who received the therapy in earlier BioMarin trials. The main goal is to see if the treatment's benefits last and to monitor for any long-term side effects.
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Contacts and locations
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Locations
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Addenbrooke's Hospital, Hemophilia and Thrombophilia Center
Cambridge, United Kingdom
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Arthur De Siqueira Cavalcanti Hematology State Institute / Coordinating Hemocenter
Rio de Janeiro, Rio de Janeiro, 24000-000, Brazil
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CHRU Lille Hospital Center Heart-Lung Institute
Lille, 59000, France
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CHU La Timone - La Timone Children's Hospital
Marseille, 13005, France
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Campinas University Clinical Hospital, Hematology and Hemotherapy Center (Hemocentro UNICAMP)
Campinas, São Paulo, 13083-878, Brazil
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Chaim Sheba Medical Center, National Institute of Hemophilia and Coagulation
Ramat Gan, 5266202, Israel
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Charlotte Maxeke Johannesburg Academic Hospital, Hemophilia Comprehensive Care Center
Johannesburg, 2193, South Africa
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Children's Hospital of Michigan, Hemophilia and Thrombosis Center
Detroit, Michigan, 48201, United States
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Fiona Stanley Hospital
Murdoch, Western Australia, 6150, Australia
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Glasgow Royal Infirmary, Department of Hematology
Glasgow, G4 0SF, United Kingdom
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Hammersmith Hospital, Clinical Research Facility, Centre for Translational and Experimental Medicine
London, United Kingdom
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Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, 15213, United States
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Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus
Aurora, Colorado, 80045, United States
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Holy Spirit Hematology and Hemotherapy Center
Vitória, Espírito Santo, 29040-090, Brazil
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Hospital Teresa Herrera, Department of Hematology and Hemotherapy, Unit of Hemostasis and Thrombosis
A Coruña, Galicia, 15006, Spain
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Kyung Hee University Hospital at Gangdong
Seoul, 05278, South Korea
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Maggiore Polyclinic Hospital, Foundation IRCCS Ca' Granda, Center for Hemophilia and Thrombosis "Angelo Bianchi Bonomi"
Milan, 20122, Italy
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Nuffield Orthopaedic Centre, Hemophilia & Thrombosis Center
Oxford, OX3 7LD, United Kingdom
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Orthopedic Institute for Children, Orthopedic Hemophilia Treatment Center
Los Angeles, California, 90007, United States
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Queen Elizabeth Hospital Birmingham. Adult Hemophilia Centre, West Midlands Adult Comprehensive Care Hemophilia Centre
London, United Kingdom
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Royal Adelaide Hospital
Adelaide, South Australia, 5000, Australia
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Royal Brisbane and Women's Hospital
Herston, Queensland, 4029, Australia
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Royal London Hospital, Barts and the London Hemophilia Center
London, United Kingdom
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Royal Prince Alfred Hospital, Institute of Haematology
Sydney, New South Wales, 2050, Australia
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Sao Paulo University Clinical Hospital
São Paulo, São Paulo, 05403 000, Brazil
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St Joseph's Children's Hospital, Center for Bleeding and Clotting Disorders
Tampa, Florida, 33614, United States
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St Thomas' Hospital, Centre for Hemophilia, Hemostasis and Thrombosis
London, United Kingdom
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The Alfred Hospital
Melbourne, Victoria, 3004, Australia
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UC Davis Hemophilia Treatment Center
Sacramento, California, 95817, United States
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UCN Hemophilia and Thrombosis Center
Shandon, California, 27517, United States
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University Hospital Bonn, Institute of Experimental Hematology and Transfusion Medicine, Hemophilia Care Center
Bonn, North Rhine-Westphalia, 53127, Germany
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University Hospital Southampton NHS Foundation Trust
Southampton, United Kingdom
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University Hospital Virgen del Rocio (HUVR), Hematology Pediatrics
Seville, Andalusia, 41013, Spain
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University Hospitals Leuven, Campus Gasthuisberg, Department of Heart and Vascular Disease
Leuven, 3000, Belgium
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University Medical Center, Inc,; DBA University of Louisville Hospital, James Graham Brown Cancer Center
Louisville, Kentucky, 40202-3229, United States
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University of California San Diego, Hemophilia & Thrombosis Treatment Center
San Diego, California, 92121, United States
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University of California San Francisco - Moffit Hospital, Outpatient Hematology Clinic
San Francisco, California, 94143, United States
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University of Michigan, Pediatric Hematology and Oncology
Ann Arbor, Michigan, 48109-5718, United States
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Vivantes Hospital in Friedrichshain
Berlin, State of Berlin, 10249, Germany
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Washington University School of Medicine, Department of Pediatrics, Division of Hematology/Oncology
St Louis, Missouri, 63110-1093, United States
Conditions
Explore the condition pages connected to this study.