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
Phase of Trial: Phase III
Latest Information Update: 06 Feb 2019
At a glance
- Drugs Valoctocogene-roxaparvovec (Primary)
- Indications Haemophilia A
- Focus Registrational; Therapeutic Use
- Sponsors BioMarin Pharmaceutical
- 15 Oct 2018 Planned End Date changed from 1 Sep 2023 to 1 Mar 2024.
- 31 Aug 2018 Biomarkers information updated
- 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.