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New hope for rare bleeding disorder: experimental drug aims to prevent bleeds

NCT ID NCT06211634

First seen Jan 02, 2026 · Last updated May 19, 2026 · Updated 28 times

Summary

This early-stage trial tests a new medicine called HMB-001 in 57 adults with Glanzmann thrombasthenia, a rare bleeding condition. The study checks if the drug is safe and if it can reduce the number and severity of bleeding episodes. Participants receive single or multiple doses over several months to find the best dose and schedule.

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Contacts and locations

Locations

  • AP-HM - Hopital de la Timone

    Marseille, France

  • AP-HP Hopital Bicetre (Part B/C)

    Le Kremlin-Bicêtre, France

  • AP-HP Hopital Necker-Enfants Malades (Part B/C)

    Paris, France

  • Azienda Ospedaliero-Universitaria Careggi (Part B/C)

    Florence, Italy

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano (Part B/C)

    Milan, Italy

  • Hemophilia Center of Western Pennsylvania (HCWP) (Part B/C)

    Pittsburgh, Pennsylvania, 15213, United States

  • Leeds Teaching Hospitals NHS Trust

    Leeds, United Kingdom

  • Mayo Clinic - Rochester (Part B/C)

    Rochester, Minnesota, 55905, United States

  • Queen Elizabeth Hospital Birmingham (Part B/C)

    Birmingham, United Kingdom

  • Richmond Pharmacology Ltd (Part A/B/C)

    London, United Kingdom

  • Royal Free London NHS Foundation Trust (Part B/C)

    London, United Kingdom

  • The Royal London Hospital (Part B/C)

    Whitechapel, United Kingdom

  • Tulane University Medical Center (Part B/C)

    New Orleans, Louisiana, 70112, United States

  • Universitair Medisch Centrum Utrecht (Part B/C)

    Utrecht, Netherlands

  • University Hospital Leuven - Campus Gasthuisberg (Part B/C)

    Leuven, Belgium

  • University of California, San Diego (UCSD) (Part B/C)

    La Jolla, California, 92093, United States

  • Washington Institute for Coagulation (Part B/C)

    Seattle, Washington, 98101, United States

Conditions

Explore the condition pages connected to this study.