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

Purpose

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

Condition

  • Cerebral Adrenoleukodystrophy (cALD)

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