A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy

NCT05819866 | Recruiting Phase 3 FDA Drug

• 1 other identifier: MT-3-01
• Study Type: interventional
• Target: 40
• Locations: 8 countries
• Sites: 13

Brief Summary

A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adults Male Subjects with Cerebral Adrenoleukodystrophy.

Conditions

Cerebral Adrenoleukodystrophy (cALD)

Outcome Measures

Primary Outcomes (3)

• 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 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)

Secondary Outcomes (3)

• Change from Baseline in Loes Score.

  Interim analysis 1 (at 18 months of treatment)

• Change from Baseline in Loes Score

  Interim analysis 2 (at 27 months of treatment)

• Change from Baseline in Loes Score

  Final analysis (at 36 months of treatment)

Study Arms (2)

Leriglitazone

ACTIVE COMPARATOR

Leriglitazone Treatment

Drug: Leriglitazone

Placebo

PLACEBO COMPARATOR

Placebo

Drug: Placebo

Interventions

Leriglitazone DRUG

Leriglitazone at a strength of 15 mg/ml. Once-daily dosing at an initial volume of 10 ml

Leriglitazone

Placebo DRUG

Placebo will match the study drug visually and by taste

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Minoryx Therapeutics, S.L.: lead

Study Sites (13)

Stanford University Medical Center

Palo Alto, California, 94304-5978, United States

RECRUITING

Neuro Medicine Hospital / UF Health

Gainesville, Florida, 32608, United States

WITHDRAWN

Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

RECRUITING

University of Minnesota

Minneota, Minnesota, 55455, United States

RECRUITING

Health University of Utah

Salt Lake City, Utah, 84112, United States

WITHDRAWN

Hospital Austral

Buenos Aires, Argentina

RECRUITING

Federal University of Sao Paulo

São Paulo, Brazil

RECRUITING

ICM La Pitie Salpetriere

Paris, 75013, France

RECRUITING

Klinik und Poliklinik für Neurologie-Leipzig

Leipzig, 04103, Germany

RECRUITING

Sir Ganga Ram Hospital

New Delhi, 110060, India

RECRUITING

Hospital 12 de Octubre

Madrid, 28041, Spain

RECRUITING

National Hospital for Neurology and Neurosurgery

London, WC1N 3AX, United Kingdom

RECRUITING

MeSH Terms

Interventions

leriglitazone

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 23, 2023
• First Posted: April 19, 2023
• Study Start: July 12, 2023
• Primary Completion (Estimated): May 1, 2027
• Study Completion (Estimated): May 1, 2027
• Last Updated: November 17, 2025

Record last verified: 2025-11

Locations

US (6); DE (1); BR (1); FR (1); ES (1); AR (1); IN (1); GB (1)