Last updated 9 months ago
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
40 patients around the world
Available in United States, Argentina, Brazil
Minoryx Therapeutics, S.L.
2Research sites
40Patients around the world
Requirements for the patient
From 18 Years
Male
Medical requirements
- 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 diseaseSubject does not have major cognitive impairment which would impair his ability to take part in the study as determined by the investigator at screening.
- 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.
Sites
Hospital Universitario Austral - Pilar
Universidad Federal de São Paulo - Unifesp
StudyCALYX
SponsorMinoryx Therapeutics, S.L.
Requirements
From 18 YearsMale
Unique study IDclinicaltrials.gov
NCT05819866
