New drug trial offers hope for kids with rare hormone disorder

NCT ID NCT03708900

First seen Nov 20, 2025 · Last updated May 08, 2026 · Updated 14 times

Summary

This study tests a drug called osilodrostat in children and teens aged 2 to 18 with Cushing's syndrome, a condition where the body makes too much cortisol. The goal is to see how the drug works in the body, how well it lowers cortisol levels, and if it is safe. About 12 participants will take the drug for up to 12 weeks to help control their disease.

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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: •••••@•••••

  • Contact

    Phone: •••-•••-••••

Locations

  • ABMED Clinical Research Corp

    RECRUITING

    Cape Coral, Florida, 33914, United States

    Contact Email: •••••@•••••

  • Alder Hey Childrens NHS Foundation Trust

    WITHDRAWN

    Liverpool, L12 2AP, United Kingdom

  • Ann & Robert H. Lurie Children's Hospital

    RECRUITING

    Chicago, Illinois, 60611, United States

    Contact Email: •••••@•••••

    Contact

  • Aziendal Ospedaliero Universitaria Pisana Presidio Ospedale di Cisanello

    COMPLETED

    Pisa, PI, 56124, Italy

  • CHU Bicetre APHP Paris Saclay

    COMPLETED

    Paris, 94270, France

  • Hospital Necker Enfants Malades

    COMPLETED

    Paris, 75015, France

  • Multiprofile Hospital for Active Treatment Sveta Marina EAD

    WITHDRAWN

    Varna, 9010, Bulgaria

  • National Institute of Child Health and Human Development

    COMPLETED

    Bethesda, Maryland, 20892, United States

  • Ospedale Bambino Gesu

    COMPLETED

    Roma, 00165, Italy

  • Robert Debre Hospital

    COMPLETED

    Paris, 75019, France

  • Texas Valley Clinical Research

    RECRUITING

    Weslaco, Texas, 78596, United States

    Contact Email: •••••@•••••

  • The Royal London Childrens Hospital

    WITHDRAWN

    London, E11BB, United Kingdom

  • UZ Brussel

    COMPLETED

    Jette, Brussels Capital, 1090, Belgium

  • University Clinical Center Ljubljana

    WITHDRAWN

    Ljubljana, 1525, Slovenia

  • University of California San Francisco UCSF

    RECRUITING

    San Francisco, California, 94143, United States

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

Conditions

Explore the condition pages connected to this study.