One-Shot gene therapy tested to fix bleeding disorder

NCT ID NCT03370172

Summary

This early-stage study tested a single intravenous infusion of an experimental gene therapy called BAX 888 in adult men with severe hemophilia A. The main goals were to check for side effects and find a safe and effective dose. Researchers hoped the therapy would help the body produce its own missing clotting factor, potentially reducing or eliminating the need for regular factor VIII injections.

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Contacts and locations

Locations

  • AKH - Medizinische Universität Wien

    Vienna, 1090, Austria

  • CHU Rennes - Hopital Pontchaillou

    Rennes, Ille Et Vilaine, 35000, France

  • CHU Tours - Hôpital Trousseau

    Tours, Indre Et Loire, 37044, France

  • CHU de Nantes Site Hotel Dieu

    Nantes, Loire Atlantique, 44093, France

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Groupement Hospitalier Est- Hôpital Louis Pradel

    Bron, Rhone, 69677, France

  • Gulf States Hemophilia and Thrombophilia Center

    Houston, Texas, 77030-4009, United States

  • Hopital Jeanne de Flandre - CHU Lille

    Lille, Nord, 59037, France

  • Hospital Clinico Universitario de Salamanca

    Salamanca, 37007, Spain

  • Hospital Regional Universitario de Malaga

    Málaga, 29010, Spain

  • Hospital Universitari Vall d'Hebron

    Barcelona, 08035, Spain

  • Hospital Universitari i Politecnic La Fe

    Valencia, 46026, Spain

  • Hospital Universitario La Paz

    Madrid, 28046, Spain

  • Hôpital Bicêtre

    Le Kremlin-Bicêtre, Val De Marne, 94275, France

  • Hôpital Morvan

    Brest, Finistere, 29609, France

  • Hôpital de la Timone

    Marseille, Bouches-du-Rhône, 13385, France

  • Klinikum der Johann Wolfgang Goethe-Universitaet

    Frankfurt am Main, Hesse, 60590, Germany

  • Medical University of South Carolina (MUSC)

    Charleston, South Carolina, 29425, United States

  • Mount Sinai Medical Center

    New York, New York, 10029, United States

  • Orthopaedic Hospital DBA Orthopaedic Hemophilia Treatment Center

    Los Angeles, California, 90007, United States

  • Phoenix Childrens Hospital

    Phoenix, Arizona, 85016, United States

  • Semmelweis Egyetem

    Budapest, 1083, Hungary

  • UC Davis Medical Center

    Sacramento, California, 95817, United States

  • Universitaetsklinikum Carl Gustav Carus TU Dresden

    Dresden, Saxony, 01307, Germany

  • University of Colorado Hemophilia & Thrombosis Center

    Aurora, Colorado, 80045, United States

  • Vivantes Klinikum im Friedrichshain

    Berlin, 10249, Germany

Conditions

Explore the condition pages connected to this study.