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

Trial Profile

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
  • Most Recent Events

    • 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.
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top