Actively Recruiting
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
Led by Minoryx Therapeutics, S.L. · Updated on 2025-11-17
40
Participants Needed
13
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the efficacy and safety of Leriglitazone in adult male subjects with cerebral adrenoleukodystrophy (cALD), a progressive neurological condition. This phase 3 clinical trial aims to compare Leriglitazone treatment with a placebo to better understand its impact on disease progression and patient outcomes. Participants included are adult males with confirmed progressive cALD who are not recommended or willing to undergo hematopoietic stem cell transplantation (HSCT). Participants will be randomly assigned to receive either Leriglitazone at a dose of 15 mg/ml once daily, starting at 10 ml volume, or a placebo matching the study drug in appearance and taste. The study uses a triple masking design to ensure unbiased results. The treatment period spans up to 36 months, with interim analyses at 18 and 27 months to assess outcomes. Throughout the study, participants will be monitored for the primary outcome of time to death or becoming bedridden with permanent ventilatory support, whichever occurs first. Secondary outcomes include changes in the Loes score, which measures brain lesion severity. Safety and functional status will also be assessed, alongside monitoring for cognitive ability and overall neurological function. The trial is expected to conclude in May 2027.
CONDITIONS
Brief Title
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Subject is male and aged 6 years or older.
- Subject has progressive cerebral adrenoleukodystrophy with GdE+ brain lesions.
- Hematopoietic stem cell transplantation (HSCT) is not recommended or the subject is not willing to undergo HSCT.
- Loes score between 0.5 and 12 at screening.
- No major functional disability except wheelchair bound or total incontinence as expected symptoms.
- No major cognitive impairment that would affect study participation as judged by the investigator.
You will not qualify if you...
- Previous bone marrow transplantation (HSCT) or ex-vivo gene therapy (eli-Cel).
- Known type 1 or type 2 diabetes.
- Known hypersensitivity or intolerance to pioglitazone or other thiazolidinediones.
- Use of honokiol, pioglitazone, or other thiazolidinediones within 3 months prior to screening.
- Current participation in another interventional clinical study or within 1 month prior to screening.
- Other medical, neuropsychiatric, or social conditions that may affect risk-benefit, compliance, or study results as judged by the investigator.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 36 months
Participants receive once-daily dosing of leriglitazone or placebo to assess efficacy and safety in adult males with cerebral adrenoleukodystrophy.
Trial Site Locations
Total: 13 locations
1
Stanford University Medical Center
Palo Alto, California, United States, 94304-5978
Actively Recruiting
2
Neuro Medicine Hospital / UF Health
Gainesville, Florida, United States, 32608
Withdrawn
3
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205
Actively Recruiting
4
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Actively Recruiting
5
University of Minnesota
Minneota, Minnesota, United States, 55455
Actively Recruiting
6
Health University of Utah
Salt Lake City, Utah, United States, 84112
Withdrawn
7
Hospital Austral
Buenos Aires, Argentina
Actively Recruiting
8
Federal University of Sao Paulo
São Paulo, Brazil
Actively Recruiting
9
ICM La Pitie Salpetriere
Paris, France, 75013
Actively Recruiting
10
Klinik und Poliklinik für Neurologie-Leipzig
Leipzig, Germany, 04103
Actively Recruiting
11
Sir Ganga Ram Hospital
New Delhi, India, 110060
Actively Recruiting
12
Hospital 12 de Octubre
Madrid, Spain, 28041
Actively Recruiting
13
National Hospital for Neurology and Neurosurgery
London, United Kingdom, WC1N 3AX
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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