A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
Minoryx Therapeutics, S.L.
Summary
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adults Male Subjects with Cerebral Adrenoleukodystrophy.
Eligibility
- Age range
- 18+ years
- Sex
- Male
- Healthy volunteers
- No
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 m…
Interventions
- DrugLeriglitazone
Leriglitazone at a strength of 15 mg/ml. Once-daily dosing at an initial volume of 10 ml
- DrugPlacebo
Placebo will match the study drug visually and by taste
Locations (13)
- Stanford University Medical CenterPalo Alto, California
- Neuro Medicine Hospital / UF HealthGainesville, Florida
- Kennedy Krieger InstituteBaltimore, Maryland
- Massachusetts General HospitalBoston, Massachusetts
- University of MinnesotaMinneota, Minnesota
- Health University of UtahSalt Lake City, Utah