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: 11 Jul 2017

At a glance

  • Drugs BMN 270 (Primary)
  • Indications Haemophilia A
  • Focus Adverse reactions; First in man; Therapeutic Use
  • Sponsors BioMarin Pharmaceutical
  • Most Recent Events

    • 11 Jul 2017 Interim results (n=15) published in a BioMarin Pharmaceutical media release.
    • 16 Jun 2017 According to a BioMarin Pharmaceutical media release, the company will report interim results from this trial at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress
    • 09 Jun 2017 Status changed from recruiting to active, no longer recruiting.
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