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A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Active or Prior Inhibitors

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Trial Profile

A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Active or Prior Inhibitors

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

Latest Information Update: 16 Jul 2024

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At a glance

  • Drugs Valoctocogene-roxaparvovec (Primary)
  • Indications Haemophilia A
  • Focus Adverse reactions
  • Acronyms GENEr8-INH
  • Sponsors BioMarin Pharmaceutical

    • Most Recent Events

    • 05 Jul 2024 Planned End Date changed from 1 Feb 2029 to 1 Apr 2029.
    • 05 Jul 2024 Planned primary completion date changed from 1 Feb 2029 to 1 Apr 2029.
    • 05 Jul 2024 Status changed from recruiting to active, no longer recruiting.

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