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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

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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

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 14 Apr 2025

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At a glance

  • Drugs Valoctocogene-roxaparvovec (Primary)
  • Indications Haemophilia A
  • Focus Adverse reactions; First in man; Pharmacodynamics; Registrational
  • Acronyms GENEr8-INH
  • Sponsors BioMarin Pharmaceutical

Most Recent Events

  • 06 Aug 2024 Status changed from active, no longer recruiting to completed.
  • 07 Jun 2024 According to a BioMarin Pharmaceutical Media Release, additional data to be presented at International Society on Thrombosis and Haemostasis (ISTH)
  • 01 Mar 2024 Results assessing safety and efficacy of valoctocogene roxaparvovec 5-6 years post-treatment published in the Haemophilia.

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