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NCT06224907

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

Study Summary

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

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Interventions

Valoctocogene roxaparvovecBIOLOGICAL
Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Severe Hemophilia A

Study Locations

FacilityCityStateCountry
Asahikawa Medical University HospitalHokkaidoAsahikawaJapan
Saitama Medical University HospitalSaitamaIruma-gunJapan
Nagoya University HospitalAichiNagoyaJapan
Tokyo Medical University HospitalTokyoShinjuku-kuJapan

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026