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

    paul.pisacane@bmrn.com

  • 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