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A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401) — GENEr8-LTE  

Hemophilia A Clinical Trial

Official title:
A Long-Term Follow-Up Study in Subjects With Severe Hemophilia A Who Received BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in a Prior BioMarin Clinical Trial

Clinical Trial Summary

The BMN 270 clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of BMN 270 for at least 5 years post-infusion. This long-term follow-up study is needed to help further understand the long-term safety of BMN 270 beyond 5 years and to assess the durability of efficacy.

Clinical Trial Description

Study 270-401 will collect additional follow-up data in a single study for approximately 10 years among all subjects who consent to participate and have completed their primary treatment study (ie, for any study in which they received BMN 270). ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05768386
Study type Observational
Source BioMarin Pharmaceutical
Contact
Status Enrolling by invitation
Phase
Start date January 1, 2023
Completion date January 2040
View Details
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