Assessing Leriglitazone for Adult Males with Cerebral Adrenoleukodystrophy
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
This study is testing if a new medication called Leriglitazone can help adult men with a rare brain condition called Cerebral Adrenoleukodystrophy feel better.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 40 (estimated) |
| Ages | 18 Years and up |
| Sex | Male |
| Sponsor | Minoryx Therapeutics, S.L. Industry-sponsored |
| Locations | 13 sites (Palo Alto, California and 12 other locations) |
| Trial ID | NCT05819866 on ClinicalTrials.gov |
What this trial studies
This clinical study evaluates the efficacy and safety of Leriglitazone in adult male patients diagnosed with progressive Cerebral Adrenoleukodystrophy (cALD). Participants will receive either Leriglitazone or a placebo, and their progress will be monitored to determine the treatment's effectiveness. The study specifically targets males aged 18 and older who have certain disease characteristics and are not candidates for hematopoietic stem cell transplantation (HSCT). The study aims to provide insights into a potential new treatment option for this rare neurological condition.
Who should consider this trial
Good fit: Ideal candidates for this study are adult males aged 18 and older with progressive cALD and specific disease characteristics.
Not a fit: Patients who have undergone previous bone marrow transplantation or have significant cognitive impairments may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could offer a new therapeutic option for adult males suffering from cALD, potentially improving their quality of life and disease outcomes.
How similar studies have performed: While this approach is novel for treating adult males with cALD, similar studies in related conditions have shown promise in exploring new therapeutic avenues.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Palo Alto, California and 12 other locations
- Stanford University Medical Center — Palo Alto, California, United States (Recruiting)
- Neuro Medicine Hospital / UF Health — Gainesville, Florida, United States (Withdrawn)
- Kennedy Krieger Institute — Baltimore, Maryland, United States (Recruiting)
- Massachusetts General Hospital — Boston, Massachusetts, United States (Recruiting)
- University of Minnesota — Minneota, Minnesota, United States (Recruiting)
- Health University of Utah — Salt Lake City, Utah, United States (Withdrawn)
- Hospital Austral — Buenos Aires, Argentina (Recruiting)
- Federal University of Sao Paulo — São Paulo, Brazil (Recruiting)
- ICM La Pitie Salpetriere — Paris, France (Recruiting)
- Klinik und Poliklinik für Neurologie-Leipzig — Leipzig, Germany (Recruiting)
- Sir Ganga Ram Hospital — New Delhi, India (Recruiting)
- Hospital 12 de Octubre — Madrid, Spain (Recruiting)
- National Hospital for Neurology and Neurosurgery — London, United Kingdom (Recruiting)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.