A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
- Conditions
- Cerebral Adrenoleukodystrophy (cALD)
- Interventions
- Drug: Placebo
- Registration Number
- NCT05819866
- Lead Sponsor
- Minoryx Therapeutics, S.L.
- Brief Summary
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adults Male Subjects with Cerebral Adrenoleukodystrophy.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- Male
- Target Recruitment
- 40
- 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
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo Placebo Leriglitazone Leriglitazone Leriglitazone Treatment
- Primary Outcome Measures
Name Time Method The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo. Final analysis (at 36 months of treatment) The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
- Secondary Outcome Measures
Name Time Method Change from Baseline in Loes Score Final analysis (at 36 months of treatment) Change from Baseline in Loes Score. Loes Score Minimum = 0 Maximum =34 0 better outcome (healthy) 34 worst osutcome
Change from Baseline in Loes Score. Interim analysis 1 (at 18 months of treatment) Change from Baseline in Loes Score. Loes Score Minimum = 0 Maximum =34 0 better outcome (healthy) 34 worst osutcome
Trial Locations
- Locations (13)
Stanford University Medical Center
🇺🇸Palo Alto, California, United States
Neuro Medicine Hospital / UF Health
🇺🇸Gainesville, Florida, United States
University of Minnesota
🇺🇸Minnesota, Minnesota, United States
Kennedy Krieger Institute
🇺🇸Baltimore, Maryland, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Health University of Utah
🇺🇸Salt Lake City, Utah, United States
Hospital Austral
🇦🇷Buenos Aires, Argentina
National Hospital for Neurology and Neurosurgery
🇬🇧London, United Kingdom
Federal University of Sao Paulo
🇧🇷São Paulo, Brazil
ICM La Pitie Salpetriere
🇫🇷Paris, France
Sir Ganga Ram Hospital
🇮🇳New Delhi, India
Hospital 12 de Octubre
🇪🇸Madrid, Spain
Klinik und Poliklinik für Neurologie-Leipzig
🇩🇪Leipzig, Germany