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This is a Phase II/III, randomized, double-blind, placebo-controlled, multicenter, two parallel-group study in male patients with the AMN phenotype of X-linked adrenoleukodystrophy (X-ALD) to assess the efficacy and safety of MIN-102 treatment. Study sites will consist of specialist referral centers experienced in the management of adrenoleukodystrophy (ALD).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Active | Active Comparator |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MIN-102 | Drug | MIN-102 treatment |
| |
| Placebos |
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate the efficacy of MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patients as determined by a motor function test. | in 96 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate the efficacy of MIN-102 in terms of patient reported outcomes. | in 96 weeks | |
| SSPROM (Severity Score System for Progressive Myelopathy ) | in 96 weeks | |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford University Medical Center | Stanford | California | 94304 | United States | ||
| Kennedy Krieger Institute |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36681445 | Derived | Kohler W, Engelen M, Eichler F, Lachmann R, Fatemi A, Sampson J, Salsano E, Gamez J, Molnar MJ, Pascual S, Rovira M, Vila A, Pina G, Martin-Ugarte I, Mantilla A, Pizcueta P, Rodriguez-Pascau L, Traver E, Vilalta A, Pascual M, Martinell M, Meya U, Mochel F; ADVANCE Study Group. Safety and efficacy of leriglitazone for preventing disease progression in men with adrenomyeloneuropathy (ADVANCE): a randomised, double-blind, multi-centre, placebo-controlled phase 2-3 trial. Lancet Neurol. 2023 Feb;22(2):127-136. doi: 10.1016/S1474-4422(22)00495-1. |
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| ID | Term |
|---|---|
| D000326 | Adrenoleukodystrophy |
| ID | Term |
|---|---|
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| Drug |
Placebo |
|
| EDSS (Expanded Disability Status Scale ) |
| in 96 weeks |
| Quality of life scales (Euroqol) | in 96 weeks |
| Incidence of cerebral inflammatory lesions | in 96 weeks |
| Baltimore |
| Maryland |
| 21205 |
| United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Hospital de la Pitié-Salpêtrière | Paris | France |
| Universitat Leipzig Klinik and Poliklinik für Neurologie | Leipzig | Germany |
| Institute of Genomic Medicine and Rare Disorders | Budapest | Hungary |
| Instituto Neurologico Carlo Besta | Milan | Italy |
| Academish Medisch Centrum | Amsterdam | Netherlands |
| Hospital Universitari Vall d'Hebrón | Barcelona | Spain |
| National hospital for Neurology and Neurosurgery Charles Dent Metabolic Unit) | London | United Kingdom |
| D009422 | Nervous System Diseases |
| D020279 | Hereditary Central Nervous System Demyelinating Diseases |
| D056784 | Leukoencephalopathies |
| D003711 | Demyelinating Diseases |
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D008661 | Metabolism, Inborn Errors |
| D018901 | Peroxisomal Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D000309 | Adrenal Insufficiency |
| D000307 | Adrenal Gland Diseases |
| D004700 | Endocrine System Diseases |