A Phase 1/2 Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL and Pre-existing Antibodies Against AAV5
Phase of Trial: Phase I/II
Latest Information Update: 24 Jan 2019
At a glance
- Drugs Valoctocogene-roxaparvovec (Primary)
- Indications Haemophilia A
- Focus Adverse reactions
- Sponsors BioMarin Pharmaceutical
- 15 May 2018 According to a BioMarin Pharmaceuticals media release, first patient has been dosed in this study.
- 27 Apr 2018 Status changed from planning to recruiting.
- 25 Apr 2018 According to a BioMarin Pharmaceuticals media release, company expects to start in second quarter of 2018.