Cerebral Adrenoleukodystrophy (cALD)
Conditions
Brief summary
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adults Male Subjects with Cerebral Adrenoleukodystrophy.
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
Sponsors
Minoryx Therapeutics, S.L.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
MALE
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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. | Interim analysis 1 (at 18 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
| Measure | Time frame | Description |
|---|---|---|
| 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 |
| Change from Baseline in Loes Score | Interim analysis 2 (at 27 months of treatment) | Change from Baseline in Loes Score. Loes Score Minimum = 0 Maximum =34 0 better outcome (healthy) 34 worst osutcome |
Countries
Argentina, Brazil, France, Germany, India, Spain, United Kingdom, United States
Outcome results
None listed