clinical trial · NCT04684940
Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
Hemophilia A With InhibitorHemophilia A With Anti Factor VIIIValoctocogene roxaparvovec
brief summary
This Phase I/II clinical study will evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe haemophilia A and inhibitors to FVIII. Part A of the study will involve subjects who have active inhibitors to FVIII, and Part B involving subjects with a prior history of inhibitors.
started
Dec 10, 2020
primary completion
Apr 1, 2029
completion
Apr 1, 2029
last updated
Jul 14, 2025
official title
A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Active or Prior Inhibitors
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol