NCT02576795

Gene Therapy Study in Severe Haemophilia A Patients (270-201)

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

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 15 (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, 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.

Conditions

• Severe Haemophilia A

Interventions

Type	Name	Description
BIOLOGICAL	valoctocogene roxaparvovec	Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Severe Hemophilia A

Timeline

• Start date: 2015-09-28
• Primary completion: 2024-02-14
• Completion: 2024-02-14
• First posted: 2015-10-15
• Last updated: 2025-04-10
• Results posted: 2025-04-10

Locations

5 sites across 1 country: United Kingdom

Regulatory

• FDA-regulated drug study