Phase 3 Study To Evaluate Efficacy/Safety of Valoctocogene Roxaparvovec an AAV Vector-Mediated Gene Transfer of hFVIII at a Dose of 4E13vg/kg in Hemophilia A Patients With Residual FVIII Levels ≤1IU/dL Receiving Prophylactic FVIII Infusions

Trial Profile

Phase 3 Study To Evaluate Efficacy/Safety of Valoctocogene Roxaparvovec an AAV Vector-Mediated Gene Transfer of hFVIII at a Dose of 4E13vg/kg in Hemophilia A Patients With Residual FVIII Levels ≤1IU/dL Receiving Prophylactic FVIII Infusions

Recruiting
Phase of Trial: Phase III

Latest Information Update: 20 Jul 2018

At a glance

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

    • 22 May 2018 According to a BioMarin Pharmaceutical media release, this study is expected to complete enrollment in one to two quarters after GENEr8-1 in 2019.
    • 10 Apr 2018 Status changed from not yet recruiting to recruiting.
    • 09 Dec 2017 According to a BioMarin Pharmaceutical media release, this trial will enroll its first patient at the start of 2018.
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