Trial Profile
A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions
Status:
Active, no longer recruiting
Phase of Trial:
Phase III
Latest Information Update: 20 Dec 2023
Price :
$35
*
At a glance
- Drugs Valoctocogene-roxaparvovec (Primary)
- Indications Haemophilia A
- Focus Registrational; Therapeutic Use
- Acronyms BMN 270-301; GENEr8-1
- Sponsors BioMarin Pharmaceutical
- 12 Dec 2023 Results (n-134) assessing protective effect of valoctocogene roxaparvovec presented at the 65th American Society of Hematology Annual Meeting and Exposition
- 05 Sep 2023 Results of HRQOL in adult men with severe HA without inhibitors after valoctocogene roxaparvovec gene transfer , published in the Journal of Thrombosis and Haemostasis
- 01 Jul 2023 Results (NCT03370913 and NCT02847637) Comparing bleeding outcomes of valoctocogene roxaparvovec gene therapy, emicizumab prophylaxis, and FVIII replacement prophylaxis in severe haemophilia A published in the Haemophilia