One-Time gene therapy could free hemophilia patients from frequent infusions

NCT ID NCT06224907

First seen Mar 26, 2026 · Last updated May 13, 2026 · Updated 7 times

Summary

This study tests a one-time gene therapy (valoctocogene roxaparvovec) in 6 Japanese men with severe hemophilia A. The goal is to see if it can raise their clotting factor levels and reduce or stop the need for regular factor VIII infusions. Participants receive the therapy via IV and are monitored for safety and effectiveness over time.

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Contacts and locations

Locations

  • Asahikawa Medical University Hospital

    Hokkaido, Asahikawa, 078-8510, Japan

  • Nagoya University Hospital

    Aichi, Nagoya, 466-8560, Japan

  • Saitama Medical University Hospital

    Saitama, Iruma-gun, 350-0495, Japan

  • Tokyo Medical University Hospital

    Tokyo, Shinjuku-ku, 160-0023, Japan

Conditions

Explore the condition pages connected to this study.