NCT03520712

Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5

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

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Hemophilia A
• Gene Therapy
• Clotting Disorders
• Blood Disorder

Interventions

Type	Name	Description
BIOLOGICAL	Valoctocogene Roxaparvovec	Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A

Timeline

• Start date: 2018-04-24
• Primary completion: 2024-08-07
• Completion: 2024-08-07
• First posted: 2018-05-11
• Last updated: 2025-08-22
• Results posted: 2025-08-22

Locations

9 sites across 4 countries: South Africa, South Korea, Taiwan, United Kingdom

Regulatory

• FDA-regulated drug study