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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: 20 Dec 2023

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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

    • 12 Dec 2023 Results (n-134) assessing protective effect of valoctocogene roxaparvovec presented at the 65th American Society of Hematology Annual Meeting and Exposition
    • 05 Sep 2023 Results of HRQOL in adult men with severe HA without inhibitors after valoctocogene roxaparvovec gene transfer , published in the Journal of Thrombosis and Haemostasis
    • 01 Jul 2023 Results (NCT03370913 and NCT02847637) Comparing bleeding outcomes of valoctocogene roxaparvovec gene therapy, emicizumab prophylaxis, and FVIII replacement prophylaxis in severe haemophilia A published in the Haemophilia
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