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Purpose

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

Condition

Eligibility

Eligible Ages
Over 18 Years
Eligible Genders
Male
Accepts Healthy Volunteers
No

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.

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Triple (Participant, Care Provider, Investigator)

Arm Groups

ArmDescriptionAssigned Intervention
Active Comparator
Leriglitazone 		Leriglitazone Treatment
  • Drug: Leriglitazone
    Leriglitazone at a strength of 15 mg/ml. Once-daily dosing at an initial volume of 10 ml
Placebo Comparator
Placebo 		Placebo
  • Drug: Placebo
    Placebo will match the study drug visually and by taste

Recruiting Locations

Massachusetts General Hospital
Boston, Massachusetts 02114
Contact:
Reza Seyedsadjadi
919-280-5582
rseyedsadjadi@mgh.harvard.edu

More Details

Status
Recruiting
Sponsor
Minoryx Therapeutics, S.L.

Study Contact

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.