NCT05768386

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

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

• Status: Enrolling By Invitation
• Phase: —
• Study type: Observational
• Enrollment: 172 (estimated)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed 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).

Conditions

• Hemophilia A

Timeline

• Start date: 2023-01-01
• Primary completion: 2040-01-01
• Completion: 2040-01-01
• First posted: 2023-03-14
• Last updated: 2026-01-12

Locations

41 sites across 12 countries: United States, Australia, Belgium, Brazil, France, Germany, Israel, Italy, South Africa, South Korea, Spain, United Kingdom