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

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Trial ID: NCT05819866

Purpose

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

Official Title

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

Stanford Investigator(s)

Jacinda Sampson
Jacinda Sampson

Clinical Professor, Neurology & Neurological Sciences

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.

Intervention(s):

drug: Leriglitazone

drug: Placebo

Recruiting

I'm Interested

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Jacinda Sampson
650-723-0993