A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adults Male Subjects with Cerebral Adrenoleukodystrophy.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
40
Leriglitazone at a strength of 15 mg/ml. Once-daily dosing at an initial volume of 10 ml
Placebo will match the study drug visually and by taste
Stanford University Medical Center
Palo Alto, California, United States
RECRUITINGNeuro Medicine Hospital / UF Health
Gainesville, Florida, United States
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.
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.
Time frame: Interim analysis 1 (at 18 months of treatment)
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.
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.
Time frame: Interim analysis 2 (at 27 months of treatment)
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.
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.
Time frame: Final analysis (at 36 months of treatment)
Change from Baseline in Loes Score.
Change from Baseline in Loes Score. Loes Score Minimum = 0 Maximum =34 0 better outcome (healthy) 34 worst osutcome
Time frame: Interim analysis 1 (at 18 months of treatment)
Change from Baseline in Loes Score
Change from Baseline in Loes Score. Loes Score Minimum = 0 Maximum =34 0 better outcome (healthy) 34 worst osutcome
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Kennedy Krieger Institute
Baltimore, Maryland, United States
RECRUITINGMassachusetts General Hospital
Boston, Massachusetts, United States
RECRUITINGUniversity of Minnesota
Minneota, Minnesota, United States
RECRUITINGHealth University of Utah
Salt Lake City, Utah, United States
WITHDRAWNHospital Austral
Buenos Aires, Argentina
RECRUITINGFederal University of Sao Paulo
São Paulo, Brazil
RECRUITINGICM La Pitie Salpetriere
Paris, France
RECRUITINGKlinik und Poliklinik für Neurologie-Leipzig
Leipzig, Germany
RECRUITING...and 3 more locations
Time frame: Interim analysis 2 (at 27 months of treatment)
Change from Baseline in Loes Score
Change from Baseline in Loes Score. Loes Score Minimum = 0 Maximum =34 0 better outcome (healthy) 34 worst osutcome
Time frame: Final analysis (at 36 months of treatment)