clinical trial · NCT02576795
Gene Therapy Study in Severe Haemophilia A Patients (270-201)
Severe Haemophilia Avaloctocogene roxaparvovec
brief summary
This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, dose escalation study in order to determine the safety and efficacy of valoctocogene roxaparvovec (an Adenovirus-Associated Virus based gene therapy vector in participants with severe haemophilia A.
started
Sep 28, 2015
primary completion
Feb 14, 2024
completion
Feb 14, 2024
last updated
Apr 10, 2025
official title
A Phase 1/2, Dose-Escalation, Safety, Tolerability and Efficacy Study of Valoctocogene Roxaparvovec, an Adenovirus-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Patients With Severe Haemophilia A
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol