NCT04684940

Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors

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

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Hemophilia A With Inhibitor
• Hemophilia A With Anti Factor VIII

Interventions

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

Timeline

• Start date: 2020-12-10
• Primary completion: 2029-04-01
• Completion: 2029-04-01
• First posted: 2020-12-28
• Last updated: 2025-07-14

Locations

9 sites across 6 countries: United States, Brazil, Israel, South Korea, Taiwan, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study