A Phase 1/2, Dose-Escalation Safety, Tolerability and Efficacy Study of BMN 270, an Adenovirus-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Patients with Severe Haemophilia A
Active, no longer recruiting
Phase of Trial: Phase I/II
Latest Information Update: 26 Oct 2017
At a glance
- Drugs Valoctocogene-roxaparvovec (Primary)
- Indications Haemophilia A
- Focus Adverse reactions; First in man; Therapeutic Use
- Sponsors BioMarin Pharmaceutical
- 26 Oct 2017 According to a BioMarin Pharmaceutical media release, the U.S. Food and Drug Administration (FDA) granted valoctocogene roxaparvovec (formerly BMN 270) Breakthrough Therapy Designation based on data from this trial.
- 18 Oct 2017 An updated data of the 4e13 vg/kg dose at up to 36 weeks of observation at the September 14, 2017 data cut, has been presented in a BioMarin Pharmaceutical media release.
- 11 Jul 2017 Interim results (n=15) published in a BioMarin Pharmaceutical media release.
Most Recent Events
Table of Contents
- At a glance
- Trial Overview
- Trial Details
- Trial Centres
- Trial History