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

Minoryx

Status and phase

Enrolling

Phase 3

Conditions

Cerebral Adrenoleukodystrophy (cALD)

Treatments

Drug: Leriglitazone

Drug: Placebo

Study type

Interventional

Funder types

Industry

Identifiers

MT-3-01

Details and patient eligibility

About

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

Enrollment

40 estimated patients

Sex

Male

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

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.