Gene therapy could help hemophilia b patients stop bleeding

NCT ID NCT03961243

First seen Apr 24, 2026 · Last updated Apr 24, 2026

Summary

This early-stage trial tests a gene therapy for hemophilia B, a bleeding disorder. Doctors take a patient's own stem cells, add a working gene for clotting factor IX, and put them back. The goal is to see if it's safe and helps the body make its own clotting factor, reducing the need for regular factor infusions. The study involves 10 males aged 2 and older with severe hemophilia B.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Shenzhen Geno-immune Medical Institute

    RECRUITING

    Shenzhen, Guangdong, 518000, China

    Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.