RecruitingPhase 3NCT05819866

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


Sponsor

Minoryx Therapeutics, S.L.

Enrollment

40 participants

Start Date

Jul 12, 2023

Study Type

INTERVENTIONAL

Conditions

Summary

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


Eligibility

Sex: MALEMin Age: 18 Years

Inclusion Criteria6

  • 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 Criteria6

  • 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.

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Interventions

DRUGLeriglitazone

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

DRUGPlacebo

Placebo will match the study drug visually and by taste


Locations(13)

Stanford University Medical Center

Palo Alto, California, United States

Neuro Medicine Hospital / UF Health

Gainesville, Florida, United States

Kennedy Krieger Institute

Baltimore, Maryland, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

University of Minnesota

Minneota, Minnesota, United States

Health University of Utah

Salt Lake City, Utah, United States

Hospital Austral

Buenos Aires, Argentina

Federal University of Sao Paulo

São Paulo, Brazil

ICM La Pitie Salpetriere

Paris, France

Klinik und Poliklinik für Neurologie-Leipzig

Leipzig, Germany

Sir Ganga Ram Hospital

New Delhi, India

Hospital 12 de Octubre

Madrid, Spain

National Hospital for Neurology and Neurosurgery

London, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT05819866


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