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A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions

Trial Profile

A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions

Recruiting
Phase of Trial: Phase III

Latest Information Update: 07 Jan 2019

At a glance

  • Drugs Valoctocogene-roxaparvovec (Primary)
  • Indications Haemophilia A
  • Focus Registrational; Therapeutic Use
  • Sponsors BioMarin Pharmaceutical
  • Most Recent Events

    • 07 Jan 2019 According to a BioMarin Pharmaceutical media release, the company has completed enrollment of the initial cohort of patients intended to support a BLA submission through the accelerated approval pathway. The study is targeting enrollment of 130 patients by mid-year 2019.
    • 25 Oct 2018 According to a BioMarin Pharmaceutical media release, enrollment completion is expected during second quarter of 2019.
    • 22 May 2018 According to a BioMarin Pharmaceutical media release, enrollment completion in this newly amended study is expected in the first quarter of 2019.
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