Clinical trial NCT05819866: A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
Official title: A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Cerebral Adrenoleukodystrophy (cALD)
Lead sponsor: Minoryx Therapeutics, S.L.
Sponsor class: INDUSTRY
First posted: 2023-04-19
Enrollment: 40
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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


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.

Primary outcomes:
measure: The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
timeFrame: Interim analysis 1 (at 18 months of treatment)
measure: The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
timeFrame: Interim analysis 2 (at 27 months of treatment)
measure: The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
timeFrame: Final analysis (at 36 months of treatment)
Secondary outcomes:
measure: Change from Baseline in Loes Score.
timeFrame: Interim analysis 1 (at 18 months of treatment)
measure: Change from Baseline in Loes Score
timeFrame: Interim analysis 2 (at 27 months of treatment)
measure: Change from Baseline in Loes Score
timeFrame: Final analysis (at 36 months of treatment)
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Stanford University Medical Center
state: California
city: Gainesville
country: United States
facility: Neuro Medicine Hospital / UF Health
state: Florida
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Minnesota
country: United States
facility: University of Minnesota
state: Minnesota
city: Salt Lake City
country: United States
facility: Health University of Utah
state: Utah
city: Buenos Aires
country: Argentina
facility: Hospital Austral
city: São Paulo
country: Brazil
facility: Federal University of Sao Paulo
city: Paris
country: France
facility: ICM La Pitie Salpetriere
city: Leipzig
country: Germany
facility: Klinik und Poliklinik für Neurologie-Leipzig
city: New Delhi
country: India
facility: Sir Ganga Ram Hospital
city: Madrid
country: Spain
facility: Hospital 12 de Octubre
city: London
country: United Kingdom
facility: National Hospital for Neurology and Neurosurgery
Study officials:
References: