Hope for rare brain disease: new drug aims to delay death or paralysis

NCT ID NCT05819866

First seen Nov 17, 2025 · Last updated Apr 24, 2026 · Updated 14 times

Summary

This study tests a drug called leriglitazone in adult men with cerebral adrenoleukodystrophy (cALD), a rare and serious brain disease. The goal is to see if the drug can delay death or the need for permanent breathing support compared to a placebo. About 40 men who cannot or choose not to have a stem cell transplant will take part.

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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

Locations

  • Federal University of Sao Paulo

    RECRUITING

    São Paulo, Brazil

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

  • Health University of Utah

    WITHDRAWN

    Salt Lake City, Utah, 84112, United States

  • Hospital 12 de Octubre

    RECRUITING

    Madrid, 28041, Spain

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

  • Hospital Austral

    RECRUITING

    Buenos Aires, Argentina

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

  • ICM La Pitie Salpetriere

    RECRUITING

    Paris, 75013, France

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

  • Kennedy Krieger Institute

    RECRUITING

    Baltimore, Maryland, 21205, United States

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

  • Klinik und Poliklinik für Neurologie-Leipzig

    RECRUITING

    Leipzig, 04103, Germany

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

  • Massachusetts General Hospital

    RECRUITING

    Boston, Massachusetts, 02114, United States

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

  • National Hospital for Neurology and Neurosurgery

    RECRUITING

    London, WC1N 3AX, United Kingdom

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

  • Neuro Medicine Hospital / UF Health

    WITHDRAWN

    Gainesville, Florida, 32608, United States

  • Sir Ganga Ram Hospital

    RECRUITING

    New Delhi, 110060, India

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

  • Stanford University Medical Center

    RECRUITING

    Palo Alto, California, 94304-5978, United States

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

  • University of Minnesota

    RECRUITING

    Minneota, Minnesota, 55455, United States

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

Conditions

Explore the condition pages connected to this study.