NCT06224907

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

GENEr8-JPN: A Phase 3 Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Japanese Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 6 (estimated)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Accepted

Summary

This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in Japanese patients with severe hemophilia A.

Detailed description

This is a Phase 3, single-arm, open-label study in Japanese hemophilia A (HA) participants with endogenous coagulation factor VIII (FVIII) activity levels \<1 IU/dL treated continuously with prophylactic exogenous FVIII for a minimum of 1 year prior to enrollment. Participants will be enrolled at approximately 10 sites in Japan. Participants must have high-quality, well-documented historical data available concerning previous bleeding episodes and exogenous FVIII usage over the previous 12 months in order to be eligible to enroll in the study. Approximately 6 Japanese adult participants with severe HA will receive a 6E13 vg/kg dose of BMN 270 as a single intravenous infusion.

Conditions

• Hemophilia A

Interventions

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

Timeline

• Start date: 2023-12-25
• Primary completion: 2025-04-16
• Completion: 2029-03-01
• First posted: 2024-01-25
• Last updated: 2026-03-19

Locations

4 sites across 1 country: Japan

Regulatory

• FDA-regulated drug study