Clinical Trials in Spain / NCT05819866
Active, not recruiting
Phase 3
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
NCT05819866 · tracked via the Priya Life Science Spain tracker
Condition(s) studied
Cerebral Adrenoleukodystrophy (cALD)
Investigational drug(s) / intervention(s)
LeriglitazonePlacebo
Leriglitazone: Leriglitazone at a strength of 15 mg/ml. Once-daily dosing at an initial volume of 10 ml
Placebo: Placebo will match the study drug visually and by taste
Study summary
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adults Male Subjects with Cerebral Adrenoleukodystrophy.
Eligibility
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.
Primary outcome measure(s)
- 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. - 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 2 (at 27 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. - 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.
Trial sites (11)
| Facility | City | Region | Status |
|---|---|---|---|
| Stanford University Medical Center | Palo Alto | California | |
| Kennedy Krieger Institute | Baltimore | Maryland | |
| Massachusetts General Hospital | Boston | Massachusetts | |
| University of Minnesota | Minneota | Minnesota | |
| Hospital Austral | Buenos Aires | Argentina | |
| Federal University of Sao Paulo | São Paulo | Brazil | |
| ICM La Pitie Salpetriere | Paris | France | |
| Klinik und Poliklinik für Neurologie-Leipzig | Leipzig | Germany | |
| Sir Ganga Ram Hospital | New Delhi | India | |
| Hospital 12 de Octubre | Madrid | Spain | |
| National Hospital for Neurology and Neurosurgery | London | United Kingdom |
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05819866 on ClinicalTrials.gov ↗ ← All trials in Spain