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 India tracker

Sponsor

Minoryx Therapeutics, S.L.

Phase

Phase 3

Started

2023-07-12

Last updated

2026-07-29

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

Sex

MALE

Min age

18 Years

Max age

—

Healthy volunteers

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
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 India