One-Time gene shot could tame hemophilia b bleeding

NCT ID NCT01687608

First seen Mar 26, 2026 · Last updated May 01, 2026 · Updated 5 times

Summary

This study tests a single intravenous dose of gene therapy (AskBio009) in up to 30 adults with hemophilia B, a bleeding disorder. The therapy delivers a working copy of the factor IX gene to help the body produce its own clotting factor. The goal is to reduce the frequency and severity of bleeding episodes. Participants must have severe hemophilia B and a history of frequent bleeds.

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Contacts and locations

Locations

  • BloodCenter of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Bloodworks Northwest

    Seattle, Washington, 98104, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital of Boston

    Boston, Massachusetts, 022105, United States

  • Emory University

    Atlanta, Georgia, 30322, United States

  • Gulf States Hemophilia and Thrombosis Center

    Houston, Texas, 77030, United States

  • Medical University of South Carolina

    Charleston, South Carolina, 29425, United States

  • Mount Sinai Medical Center

    New York, New York, 10029, United States

  • Orthopaedic Hemophilia Treatment Center

    Los Angeles, California, 90007, United States

  • Rush University Medical Center

    Chicago, Illinois, 60612, United States

  • The Hemophilia Center, Oregon Health and Science University

    Portland, Oregon, 97239, United States

  • U of Colorado School of Medicine, Hemophilia & Thrombosis Treatment Center

    Aurora, Colorado, 80045, United States

  • University of California Davis Medical Center

    Sacramento, California, 95817, United States

  • University of California at San Diego Medical Center

    San Diego, California, 92103-8651, United States

  • University of Minnesota, Masonic Clinical Research Unit, Clinical and Translational Science Institute

    Minneapolis, Minnesota, 55455, United States

Conditions

Explore the condition pages connected to this study.