A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
NCT ID: NCT05768386
Last Updated: 2026-01-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ENROLLING_BY_INVITATION
172 participants
OBSERVATIONAL
2023-01-01
2040-01-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
PROSPECTIVE
Eligibility Criteria
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Inclusion Criteria
* Subjects must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. If the subject is unable to provide consent, a legally authorized representative may provide written informed consent.
Exclusion Criteria
* Participants must be overtly healthy and not have any condition that, in the opinion of the Investigator or Medical Monitor, would prevent the participant from fully complying with the requirements of the study and/or would impact or interfere with evaluation and interpretation of the study data (including, if applicable, advanced HIV disease).
* Where applicable, per country regulation, the participant must not currently be committed to an institution by virtue of an order issued either by judicial or administrative authorities.
18 Years
MALE
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director, MD
Role: STUDY_DIRECTOR
BioMarin Pharmaceutical
Locations
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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
Countries
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Other Identifiers
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2022-001246-38
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
270-401
Identifier Type: -
Identifier Source: org_study_id
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