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

NCT ID: NCT05819866

Last Updated: 2025-11-17

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

40 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-07-12

Study Completion Date

2027-05-31

Brief Summary

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

Detailed Description

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Conditions

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Cerebral Adrenoleukodystrophy (cALD)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Leriglitazone

Leriglitazone Treatment

Group Type ACTIVE_COMPARATOR

Leriglitazone

Intervention Type DRUG

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

Placebo

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo will match the study drug visually and by taste

Interventions

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Leriglitazone

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

Intervention Type DRUG

Placebo

Placebo will match the study drug visually and by taste

Intervention Type DRUG

Eligibility Criteria

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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.

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.
Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Minoryx Therapeutics, S.L.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Stanford University Medical Center

Palo Alto, California, United States

Site Status RECRUITING

Neuro Medicine Hospital / UF Health

Gainesville, Florida, United States

Site Status WITHDRAWN

Kennedy Krieger Institute

Baltimore, Maryland, United States

Site Status RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

University of Minnesota

Minneota, Minnesota, United States

Site Status RECRUITING

Health University of Utah

Salt Lake City, Utah, United States

Site Status WITHDRAWN

Hospital Austral

Buenos Aires, , Argentina

Site Status RECRUITING

Federal University of Sao Paulo

São Paulo, , Brazil

Site Status RECRUITING

ICM La Pitie Salpetriere

Paris, , France

Site Status RECRUITING

Klinik und Poliklinik für Neurologie-Leipzig

Leipzig, , Germany

Site Status RECRUITING

Sir Ganga Ram Hospital

New Delhi, , India

Site Status RECRUITING

Hospital 12 de Octubre

Madrid, , Spain

Site Status RECRUITING

National Hospital for Neurology and Neurosurgery

London, , United Kingdom

Site Status RECRUITING

Countries

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United States Argentina Brazil France Germany India Spain United Kingdom

Facility Contacts

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Jacinda Sampson

Role: primary

650-723-0993

Eric Mallack

Role: primary

443-923-2750

Reza Seyedsadjadi

Role: primary

617-726-5175

Troy Lund

Role: primary

612-625-4185

Hernan Amartino

Role: primary

+542304387458

Paulo Sgobbi

Role: primary

+55(11)913323742

Fanny Mochel

Role: primary

+ 33 (0)1 57 27 41 23

Caroline Bergner

Role: primary

+49 341 97 11747

C.S Agrawal

Role: primary

+91 11 4225 1099

Montserrat Morales

Role: primary

630 742 050

Robin Lachman

Role: primary

+442034484778

Other Identifiers

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MT-3-01

Identifier Type: -

Identifier Source: org_study_id

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