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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

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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

Status: Active, no longer recruiting
Phase of Trial: Phase III

Latest Information Update: 19 Jul 2024

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At a glance

  • Drugs Valoctocogene-roxaparvovec (Primary)
  • Indications Haemophilia A
  • Focus Registrational; Therapeutic Use
  • Acronyms BMN 270-301; GENEr8-1
  • Sponsors BioMarin Pharmaceutical

    • Most Recent Events

    • 03 Jul 2024 Results assessing Clinical immunogenicity outcomes of valoctocogene roxaparvovec, an AAV5-vectored gene therapy for hemophilia A published in the Molecular Therapy
    • 01 Jul 2024 Results utilizing pharmacokinetic simulations to provide guidance on best practices for maintaining haemostatic control while transitioning from emicizumab prophylaxis to valoctocogene roxaparvovec published in the Haemophilia.
    • 07 Jun 2024 According to a BioMarin Pharmaceutical media release, data from the study will be presented at the 32nd Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Bangkok, Thailand, June 22-26, 2024.

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