One-Time gene shot could free hemophilia b patients from frequent infusions

NCT ID NCT05203679

First seen Apr 03, 2026 · Last updated Apr 25, 2026 · Updated 2 times

Summary

This study tests a one-time gene therapy (BBM-H901) for adults with severe hemophilia B, a bleeding disorder caused by low clotting factor IX. The treatment uses a harmless virus to deliver a working gene, helping the body produce its own clotting factor. The goal is to reduce or prevent bleeding episodes and the need for regular factor infusions. The trial involves 32 participants and is in Phase 2/3.

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Contacts and locations

Locations

  • Anhui Provincial Hospital

    Hefei, Anhui, 230022, China

  • Henan Cancer Hospital

    Zhengzhou, Henan, 450003, China

  • Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College

    Tianjin, Tianjin Municipality, 300020, China

  • Nanfang Hospital Southern Medical University

    Guangzhou, Guangdong, 510515, China

  • North China University of Science and Technology Affiliated Hospital

    Tangshan, Hebei, 063000, China

  • Peking Union Medical College Hospital

    Beijing, Beijing Municipality, 100005, China

  • The Second Hospital of Shanxi Medical University

    Taiyuan, Shanxi, 030001, China

  • The Second People's Hospital of Shenzhen

    Shenzhen, Guangdong, 518025, China

  • The second Affiliated Hospital of Kunming Medical University

    Kunming, Yunnan, 650032, China

Conditions

Explore the condition pages connected to this study.