A Long-Term Follow-Up Study in Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial
Research type
Research Study
Full title
A Long-Term Follow-Up Study in Subjects with Severe Hemophilia A Who Received BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in a Prior BioMarin Clinical Trial
IRAS ID
1005791
Contact name
Paul Pisacane
Contact email
Sponsor organisation
BioMarin Pharmaceutical Inc.
Eudract number
2022-001246-38
Research summary
This is a non-dosing long-term follow-up study in subjects with severe haemophilia A who received BMN 270 in a previous clinical study. Baseline information will be collected from the participants' data entered in their primary treatment study. Data collection via phone call, mobile nursing/remote visits (where available), and/or site visits will be conducted quarterly for approximately 10 years. This long-term follow-up study is needed to help further understand the long-term safety of BMN 270 beyond 5-7 years (length of the participant's previous study) and to assess the drug's clinical benefit over time.
Approximately 175-300 participants are anticipated to be enrolled in this study globally, with approximately 44 being enrolled in the UK.
The study is sponsored by BioMarin Pharmaceutical Inc.REC name
London - Fulham Research Ethics Committee
REC reference
22/LO/0504
Date of REC Opinion
22 Aug 2022
REC opinion
Further Information Favourable Opinion