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.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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About 40 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jul 2023
- Expected to finish
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May 2027
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Male participants only
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Key Inclusion Criteria: * Subject is male and aged ≥18 years. * Subject has progressive cALD, defined as GdE+ brain lesions. * Subjects for whom HSCT is not recommended by the investigator or subject is not willing to undergo HSCT. * Subject has a Loes score ≥0.5 and ≤12 at Screening. * Subject does not have major functional disability in the Major Functional Disabilities-Neurological Function Score (MFD-NFS), except for "wheelchair bound" or "total incontinence", which will be allowed as these are considered expected symptoms of AMN in the time course of the disease * Subject does not have major cognitive impairment which would impair his ability to take part in the study as determined by the investigator at screening. Key Exclusion Criteria: * Subject who had previous bone marrow transplantation (HSCT) or treatment with ex-vivo gene therapy (eli-Cel). * Subject has known type 1 or type 2 diabetes. * Subject has known hypersensitivity or intolerance to pioglitazone or any other thiazolidinedione. * Subject is taking or has taken honokiol, pioglitazone, or other thiazolidinediones within 3 months prior to Screening. * Subject with current participation in another interventional clinical study or within 1 month prior to Screening. * Subject with other medical, neuropsychiatric or social conditions that, in the opinion of the investigator, are likely to adversely affect the risk-benefit of study participation, interfere with study compliance, or confound the study results.
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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
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