Hope for rare brain disease: new drug trial aims to stall deadly decline
NCT ID NCT05819866
First seen Jun 27, 2026 · Last updated Jul 30, 2026 · Updated 2 times
Summary
This Phase 3 study tests whether the drug leriglitazone can help adult men with cerebral adrenoleukodystrophy (cALD), a rare and serious brain disease. The main goal is to see if the drug delays 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.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Leriglitazone
- What this could lead to
- If it works, this could offer a treatment option to slow progression of cerebral adrenoleukodystrophy and delay severe disability or death.
- What could go wrong
- This is a small Phase 3 trial with only 40 participants, so results may not apply broadly. The drug may not show a significant benefit over placebo, and side effects are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Federal University of Sao Paulo
São Paulo, Brazil
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Hospital 12 de Octubre
Madrid, 28041, Spain
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Hospital Austral
Buenos Aires, Argentina
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ICM La Pitie Salpetriere
Paris, 75013, France
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Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
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Klinik und Poliklinik für Neurologie-Leipzig
Leipzig, 04103, Germany
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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National Hospital for Neurology and Neurosurgery
London, WC1N 3AX, United Kingdom
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Sir Ganga Ram Hospital
New Delhi, 110060, India
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Stanford University Medical Center
Palo Alto, California, 94304-5978, United States
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University of Minnesota
Minneota, Minnesota, 55455, United States