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Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors — GENEr8-INH  

Hemophilia A With Inhibitor Clinical Trial

Official title:
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

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04684940
Study type Interventional
Source BioMarin Pharmaceutical
Contact Trial Specialist
Phone 1-800-983-4587
Email medinfo@bmrn.com
Status Recruiting
Phase Phase 1/Phase 2
Start date December 10, 2020
Completion date February 2029
View Details
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