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One-Shot gene therapy aims to free patients from frequent bleeding treatments

NCT ID NCT05203679

Summary

This study is testing a single injection of an experimental gene therapy called BBM-H901 for adults with severe Hemophilia B. The goal is to see if the therapy is safe and if it can help the body produce its own missing clotting factor, which could reduce or eliminate the need for regular factor IX infusions. Researchers will monitor participants for a year to check for side effects and measure changes in bleeding rates and factor levels.

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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

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