Clinical Trial

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A

Study acronym: GENEr8-JPN
Active, Not Recruiting Phase 3
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Summary
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in Japanese patients with severe hemophilia A.
Protocol Amendment History 3 amendments
This ClinicalTrials.gov record has been amended 3 times since 2024-01-24; most recent amendment 2026-05-26.
Status change: Recruiting → Active, Not Recruiting 2024-05-06
Trial Details
NCT Number NCT06224907
Lead Sponsor BioMarin Pharmaceutical
Conditions Hemophilia A
Enrollment 6 participants
Start Date 2023-12-25
Primary Completion 2025-04-16 (estimated)
Study Completion 2029-03 (estimated)
Updated on ClinicalTrials.gov 2026-03-19