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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)

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Trial Status Enrolling By Invitation

Trial Identifier

NCT05768386

Condition

Hemophilia A, Hemophilia a

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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.

Eligibility Criteria

Birth Sex

Male

Age

From 18 Years

Healthy Volunteers

No

Study Type:

Observational

Number of Participants:

172

Study Started:

2023-01-01

Study Updated:

2024-04-24

Trial Locations

  • Orthopedic Institute for Children, Orthopedic Hemophilia Treatment Center

    Los Angeles, California, United States

  • UC Davis Hemophilia Treatment Center

    Sacramento, 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 of Michigan, Pediatric Hematology and Oncology

    Ann Arbor, Michigan, United States

  • Hemophilia Center of Western Pennsylvania

    Pittsburgh, Pennsylvania, United States

  • Addenbrooke's Hospital, Hemophilia and Thrombophilia Center

    Cambridge, , 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

  • University Hospital Southampton NHS Foundation Trust

    Southampton, , United Kingdom

Inclusion Criteria

  • 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.

Exclusion Criteria

  • 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

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