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)

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

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Contact Information

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