Hope for rare brain disease: new drug trial aims to stall deadly decline
NCT ID NCT05819866
First seen Jun 27, 2026 · Last updated Jun 27, 2026
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.
Disclaimer
Read more
Show less
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.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for CEREBRAL ADRENOLEUKODYSTROPHY (CALD) are added.
By submitting, you agree to our Terms of use
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
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Federal University of Sao Paulo
RECRUITINGSão Paulo, Brazil
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Health University of Utah
WITHDRAWNSalt Lake City, Utah, 84112, United States
-
Hospital 12 de Octubre
RECRUITINGMadrid, 28041, Spain
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Hospital Austral
RECRUITINGBuenos Aires, Argentina
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
ICM La Pitie Salpetriere
RECRUITINGParis, 75013, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Kennedy Krieger Institute
RECRUITINGBaltimore, Maryland, 21205, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Klinik und Poliklinik für Neurologie-Leipzig
RECRUITINGLeipzig, 04103, Germany
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Massachusetts General Hospital
RECRUITINGBoston, Massachusetts, 02114, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
National Hospital for Neurology and Neurosurgery
RECRUITINGLondon, WC1N 3AX, United Kingdom
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Neuro Medicine Hospital / UF Health
WITHDRAWNGainesville, Florida, 32608, United States
-
Sir Ganga Ram Hospital
RECRUITINGNew Delhi, 110060, India
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Stanford University Medical Center
RECRUITINGPalo Alto, California, 94304-5978, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
University of Minnesota
RECRUITINGMinneota, Minnesota, 55455, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••