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

ReclutandoFase 3ClinicalTrials.gov

ID: NCT05819866Tipo: INTERVENTIONALInicio: 12 de jul de 2023Fin estimado: 1 de may de 2027

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Resumen

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

Elegibilidad

Edad mínima: 18 YearsSexo: MALE

Criterios de inclusión

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.

Criterios de exclusión

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.

Intervenciones

drug

Leriglitazone

Leriglitazone at a strength of 15 mg/ml. Once-daily dosing at an initial volume of 10 ml

drug

Placebo

Placebo will match the study drug visually and by taste

Ubicaciones

Hospital Austral

Buenos Aires, Argentina

Hernan Amartino · carolina.acevedo@resolutioncrs.com · +542304387458

RECRUITING

Patrocinadores

PrincipalMinoryx Therapeutics, S.L.

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