A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)

Enrolling by invitation

Conditions studied: Hemophilia A

In brief

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.

Key facts

Study ID

NCT05768386

Run by

BioMarin Pharmaceutical

People needed

172

Starts

2023-01-01

Expected to finish

2040-01-01

Last updated by the study team

2026-01-12

Who can join

Age: 18 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Participants must have completed their primary treatment study or be currently enrolled in the primary treatment study at the time of closure by the Sponsor. Participants may enroll in 270-401 even if they have restarted FVIII prophylaxis or other hemophilia A treatment.
• 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.

You may not qualify if…

• Participants who do not directly enroll in 270-401 at the time of the study completion visit in their primary treatment study should enroll in 270-401 within 4 months of the date of that study completion visit. If a participant wishes to enroll in 270-401 after 4 months, they must receive prior approval from the Medical Monitor.
• 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.

Where it is running

• 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

Full record on ClinicalTrials.gov

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