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A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adults Male Subjects with Cerebral Adrenoleukodystrophy.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Leriglitazone | Active Comparator | Leriglitazone Treatment |
|
| Placebo | Placebo Comparator | Placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Leriglitazone | Drug | Leriglitazone at a strength of 15 mg/ml. Once-daily dosing at an initial volume of 10 ml |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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. | 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. | Final analysis (at 36 months of treatment) |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | Interim analysis 1 (at 18 months of treatment) |
| Change from Baseline in Loes Score |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford University Medical Center | Recruiting | Palo Alto | California | 94304-5978 | United States |
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| Placebo | Drug | Placebo will match the study drug visually and by taste |
|
Change from Baseline in Loes Score. Loes Score Minimum = 0 Maximum =34 0 better outcome (healthy) 34 worst osutcome
| 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 | Final analysis (at 36 months of treatment) |
| Neuro Medicine Hospital / UF Health | Withdrawn | Gainesville | Florida | 32608 | United States |
| Kennedy Krieger Institute | Recruiting | Baltimore | Maryland | 21205 | United States |
|
| Massachusetts General Hospital | Recruiting | Boston | Massachusetts | 02114 | United States |
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| University of Minnesota | Recruiting | Minneota | Minnesota | 55455 | United States |
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| Health University of Utah | Withdrawn | Salt Lake City | Utah | 84112 | United States |
| Hospital Austral | Recruiting | Buenos Aires | Argentina |
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| Federal University of Sao Paulo | Recruiting | São Paulo | Brazil |
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| ICM La Pitie Salpetriere | Recruiting | Paris | 75013 | France |
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| Klinik und Poliklinik für Neurologie-Leipzig | Recruiting | Leipzig | 04103 | Germany |
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| Sir Ganga Ram Hospital | Recruiting | New Delhi | 110060 | India |
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| Hospital 12 de Octubre | Recruiting | Madrid | 28041 | Spain |
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| National Hospital for Neurology and Neurosurgery | Recruiting | London | WC1N 3AX | United Kingdom |
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| ID | Term |
|---|---|
| C000720427 | leriglitazone |
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