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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Pivotal Study of N-acetyl-L-leucine for CACNA1ACongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Neurologic Manifestations, Spinocerebellar Ataxias, Spinocerebellar Degenerations, atactic disorder, non-human animal, brain disorder, central nervous system disorder, cerebellar ataxia, cerebellar disorder, episodic ataxia type 2, hereditary disease, inherited neurodegenerative disorder, nervous system disorder, neurodegenerative disease, spinal cord disorder, spinocerebellar ataxia type 6RecruitingPhase 32026-09-08NCT07221292
A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type CBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, histiocytosis, inborn errors of metabolism, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosisRecruitingPhase 32026-04-03NCT05163288
A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Dyskinesias, Neurologic Manifestations, Nutritional and Metabolic Diseases, Primary Immunodeficiency Diseases, Spinocerebellar Ataxias, atactic disorder, non-human animal, ataxia telangiectasia, brain disorder, cardiovascular disorder, central nervous system disorder, cerebellar ataxia, cerebellar disorder, hereditary disease, immune system disorder, inborn error of immunity, metabolic disease, nervous system disorder, neurocutaneous syndrome, telangiectasis, vascular disorderActive, not recruitingPhase 32025-07-03NCT06673056