One-Time gene infusion could change hemophilia treatment

NCT ID NCT03370172

First seen Oct 31, 2025 · Last updated Apr 25, 2026 · Updated 25 times

Summary

This study tested a new gene therapy called BAX 888 for men with severe hemophilia A. Four adult males received a single infusion to help their bodies produce more clotting factor. The main goal was to check safety and find the right dose. While promising, patients may still need ongoing care, so this is not a cure.

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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.