New hope for fabry patients: experimental drug enters human trials

NCT ID NCT06858397

First seen Nov 01, 2025 · Last updated May 09, 2026 · Updated 26 times

Summary

This early-stage study tests a new medicine called HM15421 in 18 adults with Fabry disease, a rare genetic condition that causes pain, organ damage, and other serious problems. The main goal is to see if the drug is safe and to find the right dose. Researchers will also check if it helps control the disease by measuring drug levels in the blood and watching for side effects.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

  • Contact

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

Locations

  • Centro Medico IPAM

    RECRUITING

    Rosario, Santa Fe Province, S2000, Argentina

    Contact

  • Children's Hospital Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229-3039, United States

    Contact

  • David Geffen School of Medicine UCLA, UCLA Health

    RECRUITING

    Los Angeles, California, 90095, United States

    Contact

  • Hospital Italiano de Buenos Aires

    RECRUITING

    Buenos Aires, C1199ABB, Argentina

    Contact

  • Lysosomal and Rare Disorders Research and Treatment Center

    RECRUITING

    Fairfax, Virginia, 22030, United States

    Contact

  • Pusan National University Children's Hospital in Yangsan

    RECRUITING

    Yangsan, Gyeongsangnam-do, 50612, South Korea

    Contact

  • University of Kansas School of Medicine

    RECRUITING

    Kansas City, Kansas, 66160-8500, United States

    Contact

  • University of Minnesota

    RECRUITING

    Minneapolis, Minnesota, 55455, United States

    Contact

  • University of Pittsburgh Medical Center Children's Hoispital of Pittsburgh

    RECRUITING

    Pittsburgh, Pennsylvania, 15224, United States

    Contact

  • Yonsei University, College of Medicine

    RECRUITING

    Seoul, 03722, South Korea

    Contact

Conditions

Explore the condition pages connected to this study.