A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
- Conditions
- Hemophilia A
- Registration Number
- NCT05768386
- Lead Sponsor
- BioMarin Pharmaceutical
- Brief 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.
- Detailed Description
Study 270-401 will collect additional follow-up data in a single study for approximately 10 years among all subjects who consent to participate and have completed their primary treatment study (ie, for any study in which they received BMN 270).
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- Male
- Target Recruitment
- 172
- 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.
- 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.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Evaluate the long-term safety of BMN 270 Duration of study (10 years) Occurrence of adverse drug reactions, serious adverse events, and events of special interest.
- Secondary Outcome Measures
Name Time Method Evaluate the long-term impact of BMN 270 on HRQoL Duration of study (10 years) Changes in Haemo-QOL-A
Evaluate the long-term effects of BMN 270 in subjects with hemophilia A previously treated in a BioMarin clinical trial Duration of study (10 years) Changes in annualized bleeding rate (ABR) (treated bleeds and all bleeds) and FVIII activity measured over time (CSA and OSA)
Evaluate the use of hemostatic agents (ie, emicizumab) Duration of study (10 years) Annualized use of concomitant hemostatic medications (annualized FVIII utilization and annualized FVIII infusion rate)
Trial Locations
- Locations (13)
UCN Hemophilia and Thrombosis Center
🇺🇸Shandon, California, 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
Royal London Hospital, Barts and the London Hemophilia Center
🇬🇧London, United Kingdom
St Thomas' Hospital, Centre for Hemophilia, Hemostasis and Thrombosis
🇬🇧London, United Kingdom
Queen Elizabeth Hospital Birmingham. Adult Hemophilia Centre, West Midlands Adult Comprehensive Care Hemophilia Centre
🇬🇧London, United Kingdom
University Hospital Southampton NHS Foundation Trust
🇬🇧Southampton, United Kingdom
Hemophilia Center of Western Pennsylvania
🇺🇸Pittsburgh, Pennsylvania, United States
Orthopedic Institute for Children, Orthopedic Hemophilia Treatment Center
🇺🇸Los Angeles, 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
UC Davis Hemophilia Treatment Center
🇺🇸Sacramento, California, United States
University of Michigan, Pediatric Hematology and Oncology
🇺🇸Ann Arbor, Michigan, United States