Phase 3 Open-Label Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec an AAV Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Residual FVIII Levels ≤1 IU/dL Receiving Prophylactic FVIII Infusions
Phase of Trial: Phase III
Latest Information Update: 22 Dec 2017
At a glance
- Drugs Valoctocogene-roxaparvovec (Primary)
- Indications Haemophilia A
- Focus Registrational; Therapeutic Use
- Sponsors BioMarin Pharmaceutical
- 19 Dec 2017 According to a BioMarin Pharmaceutical media release, the first patient has been dosed in this trial.
- 09 Dec 2017 According to a BioMarin Pharmaceutical media release, this trial will enroll its first patient this month.
- 07 Dec 2017 Status changed from planning to recruiting.