clinical trial · NCT03520712
Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
Hemophilia AGene TherapyClotting DisordersBlood DisorderValoctocogene Roxaparvovec
brief summary
This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, single dose study to determine the safety of valoctocogene roxaparvovec (an Adenovirus-Associated Virus (AAV) based gene therapy vector) in severe Hemophilia A patients with pre-existing antibodies against AAV5.
started
Apr 24, 2018
primary completion
Aug 7, 2024
completion
Aug 7, 2024
last updated
Aug 22, 2025
official title
A Phase 1/2 Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL and Pre-existing Antibodies Against AAV5
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol