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A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
Active Not RecruitingPhase 3
Sponsor: Minoryx Therapeutics, S.L.
Conditions: Cerebral Adrenoleukodystrophy (cALD)
Interventions: Leriglitazone, Placebo
Countries: United States, Argentina, Brazil, France, Germany, India, Spain, United Kingdom
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adults Male Subjects with Cerebral Adrenoleukodystrophy.
Eligibility overview
Sex: MALE
Age: 18 Years to —
Healthy volunteers: No
Study type: INTERVENTIONAL
Eligibility 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.
Locations (11)
- Palo Alto, California, United States
- Baltimore, Maryland, United States
- Boston, Massachusetts, United States
- Minneota, Minnesota, United States
- Buenos Aires, Argentina
- São Paulo, Brazil
- Paris, France
- Leipzig, Germany
- New Delhi, India
- Madrid, Spain
- London, United Kingdom