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A Phase 3b, Single Arm, Open-Label Study to Evaluate the Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII, With Prophylactic Corticosteroids in Hemophilia A Patients

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Trial Profile

A Phase 3b, Single Arm, Open-Label Study to Evaluate the Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII, With Prophylactic Corticosteroids in Hemophilia A Patients

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

Latest Information Update: 11 Apr 2024

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At a glance

  • Drugs Valoctocogene-roxaparvovec (Primary) ; Corticosteroids
  • Indications Haemophilia A
  • Focus Registrational; Therapeutic Use
  • Acronyms GENEr8-3
  • Sponsors BioMarin Pharmaceutical

Most Recent Events

  • 06 Feb 2024 According to a BioMarin Pharmaceutical media release, 1-year results from this study will be presented at the 2024 European Association for Haemophilia and Allied Disorders (EAHAD) Congress, Feb. 6-9, 2024.
  • 26 Oct 2022 According to BioMarin media release, the company has completed the enrollment for this study.
  • 03 Aug 2022 According to a BioMarin Pharmaceutical media release, results are expected in early 2023.

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