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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Clinical Utility of Reduced EEG Home Monitoring in Fenfluramine Titration for Dravet and LGSCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Lennox-Gastaut syndrome, brain disorder, central nervous system disorder, epilepsy, hereditary disease, nervous system disorderEnrolling by invitationNot applicable2026-10-08NCT07225231
A Study to Evaluate the Safety and Efficacy of JZP926 Capsule for the Treatment of Juvenile Myoclonic EpilepsyEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, epilepsy, juvenile myoclonic epilepsy, nervous system disorderRecruitingPhase 2 / Phase 32026-10-06NCT07723963
A Fully Remote Feasibility Trial of Phenylbutyrate for SLC6A1-Related DisordersEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, epilepsy, nervous system disorderRecruitingPhase 22026-09-29NCT07847918
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet SyndromeEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, epilepsy, generalized epilepsy with febrile seizures plus, type 2, nervous system disorderRecruitingPhase 1 / Phase 22026-09-22NCT05419492
A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, epilepsy, nervous system disorder, neurodevelopmental disorder, psychiatric disorder, visual epilepsyActive, not recruitingPhase 32026-09-17NCT06660394
A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathy (DEE)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Lennox-Gastaut syndrome, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, developmental and epileptic encephalopathy, epilepsy, hereditary disease, nervous system disorder, neurodevelopmental disorder, psychiatric disorder, visual epilepsyEnrolling by invitationPhase 32026-09-15NCT06908226
A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet SyndromeEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, epilepsy, nervous system disorder, visual epilepsyRecruitingPhase 32026-09-15NCT04462770
A Study to Investigate the Transition of Children From 'Artisanal" Cannabidiol (CBD) to EpidiolexCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Lennox-Gastaut syndrome, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, epilepsy, hereditary disease, nervous system disorder, visual epilepsyNot yet recruitingPhase 42026-09-11NCT06924827
Biomarkers of Neurodegeneration, Synaptic Plasticity and Neuroinflammation in Dravet SyndromeEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, epilepsy, nervous system disorderNot yet recruitingNot stated2026-09-03NCT07801404
ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet SyndromeEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, epilepsy, nervous system disorderRecruitingPhase 1 / Phase 22026-09-03NCT07531745
Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral SolutionCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Fibrosis, Lennox-Gastaut syndrome, Malformations of Cortical Development, Group I, Neoplasms, Multiple Primary, Pathologic Processes, Pathological Conditions, Signs and Symptoms, brain disorder, central nervous system disorder, central nervous system malformation, cerebral cortical dysplasia, cirrhosis of liver, epilepsy, hamartoma, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, liver disorder, neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, tuberous sclerosisActive, not recruitingPhase 42026-08-26NCT05044819
A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet SyndromeEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, epilepsy, nervous system disorderRecruitingPhase 32026-08-24NCT06872125
Global Registry and Natural History Study for Mitochondrial DisordersAbnormalities, Multiple, Barth syndrome, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cardiovascular Abnormalities, Cerebral Small Vessel Diseases, Chronic Disease, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Kearns-Sayre syndrome, Leber hereditary optic neuropathy, Leigh syndrome, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, MELAS syndrome, MERRF syndrome, NARP syndrome, Neurologic Manifestations, Nutritional and Metabolic Diseases, Ocular Motility Disorders, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Pearson syndrome, Pyruvate Metabolism, Inborn Errors, Signs and Symptoms, X-linked disease, ataxia neuropathy spectrum, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, coenzyme Q10 deficiency, congenital heart disease, cranial nerve neuropathy, epilepsy, eye disorder, heart disorder, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inborn mitochondrial metabolism disorder, inborn mitochondrial myopathy, inherited retinal dystrophy, maternally-inherited diabetes and deafness, metabolic disease, mitochondrial encephalomyopathy, mitochondrial neurogastrointestinal encephalomyopathy, muscle tissue disorder, musculoskeletal system disorder, myelodysplastic syndrome, nervous system disorder, neuromuscular disease, ophthalmoplegia, palsy, progressive external ophthalmoplegia, progressive myoclonus epilepsy, retinal degeneration, retinal disorder, retinitis pigmentosa, spinocerebellar ataxia with epilepsy, vascular disorder, very long chain acyl-CoA dehydrogenase deficiencyRecruitingNot stated2026-08-07NCT05554835
The FINTEPLA as an Anti-SUDEP Therapy in Dravet Syndrome ProjectDeath, Death, Sudden, Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Sudden Unexpected Death in Epilepsy, brain disorder, central nervous system disorder, epilepsy, nervous system disorderRecruitingPhase 42026-07-15NCT07112365
Personalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene MutationEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, dentatorubral-pallidoluysian atrophy, epilepsy, nervous system disorder, progressive myoclonus epilepsyActive, not recruitingPhase 1 / Phase 22026-07-13NCT07084311
Early Check: Expanded Screening in Newborns3-hydroxy-3-methylglutaric aciduria, 3-hydroxyacyl-CoA dehydrogenase deficiency, 3-methylcrotonyl-CoA carboxylase 1 deficiency, 3-methylcrotonyl-CoA carboxylase 2 deficiency, 3-phosphoglycerate dehydrogenase deficiency, AGAT deficiency, Abnormalities, Multiple, Adrenal hyperplasia, congenital, type 5, Adrenoleukodystrophy, Neonatal, Angelman syndrome, Aortic Stenosis, Supravalvular, Arrhythmias, Cardiac, Barth syndrome, Bone Diseases, Developmental, Bone Diseases, Endocrine, Bone Diseases, Metabolic, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Canavan disease, Cardiac Conduction System Disease, Cardiovascular Abnormalities, Chronic Disease, Citrullinemia 1, Collagen Diseases, Congenital Abnormalities, Congenital Hyperinsulinism, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Digestive System Diseases, Disease Attributes, Duchenne muscular dystrophy, Dwarfism, Dyslipidemias, Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Eye Diseases, Hereditary, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Fructose Metabolism, Inborn Errors, G6PD deficiency, Glycogen Storage Disease, Type IXA2, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Hermansky-Pudlak syndrome, Hermansky-Pudlak syndrome 1, Hermansky-Pudlak syndrome 4, Hyperphenylalaninemia, BH4-Deficient, B, Hypophosphatemia, Familial, Imprinting Disorders, Infant, Newborn, Diseases, Jervell and Lange-Nielsen syndrome, Jervell and Lange-Nielsen syndrome 1, Jervell and Lange-Nielsen syndrome 2, Krabbe disease, Leber congenital amaurosis 2, Leukoencephalopathies, Liddle syndrome, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, MPI-congenital disorder of glycosylation, Male Urogenital Diseases, Maple syrup urine disease, type 1A, Maple syrup urine disease, type 1B, Maple syrup urine disease, type 2, Menkes disease, Methylmalonic aciduria cblB type, Mucopolysaccharidosis I, Mucopolysaccharidosis III, Mucopolysaccharidosis Type IV A, Muscular Disorders, Atrophic, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Multiple Primary, Neoplasms, Nerve Tissue, Neurobehavioral Manifestations, Neurologic Manifestations, Niemann-Pick disease, Niemann-Pick disease type A, Niemann-Pick disease type C, Niemann-Pick disease, type C1, Niemann-Pick disease, type C2, Nutritional and Metabolic Diseases, Overweight, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Peroxisomal Disorders, Pitt-Hopkins syndrome, Prader-Willi syndrome, Primary Immunodeficiency Diseases, Rett syndrome, Schnyder corneal dystrophy, Sex Chromosome Disorders, Skin and Connective Tissue Diseases, Smith-Lemli-Opitz syndrome, Stickler syndrome type 1, Stickler syndrome type 2, Stress Disorders, Traumatic, Sulfatidosis, Tyrosinemia, Type I, Urogenital Abnormalities, Urogenital Diseases, Usher syndrome type 1B, Usher syndrome type 1C, Usher syndrome, type 1C, Usher syndrome, type 1F, Ventricular Outflow Obstruction, Waardenburg Syndrome, Type 2E, Waardenburg syndrome, Waardenburg syndrome type 1, Waardenburg syndrome type 2, Waardenburg syndrome type 2A, Wilson disease, Wolman disease, X-Linked Combined Immunodeficiency Diseases, X-linked Alport syndrome, X-linked disease, X-linked hypophosphatemic rickets, acrodermatitis enteropathica, acute stress disorder, adenine phosphoribosyltransferase deficiency, albinism, anemia, aortic valve disorder, aortic valve stenosis, apparent mineralocorticoid excess, arginase deficiency, argininosuccinic aciduria, autosomal recessive Alport syndrome, basal ganglia disorder, beta thalassemia, beta-ketothiolase deficiency, biotin-responsive basal ganglia disease, biotinidase deficiency, blood coagulation disease, blood platelet disease, bone disorder, brain disorder, calcium metabolic disease, carbamoyl phosphate synthetase I deficiency disease, cardiovascular disorder, carnitine palmitoyl transferase 1A deficiency, carnitine palmitoyltransferase II deficiency, carnitine-acylcarnitine translocase deficiency, central nervous system disorder, cerebrotendinous xanthomatosis, cholesteryl ester storage disease, chromosomal disorder, chronic granulomatous disease, citrullinemia, coagulation protein disease, combined immunodeficiency due to ZAP70 deficiency, congenital bile acid synthesis defect 1, congenital bile acid synthesis defect 2, congenital heart disease, congenital hypothyroidism, congenital isolated adrenocorticotropic hormone deficiency, connective tissue disorder, creatine transporter deficiency, cutaneous mucinosis, cystic fibrosis, cystinosis, demyelinating disease, diabetes mellitus, disease, disorder of glycogen metabolism, disorder of pilosebaceous unit, dopa-responsive dystonia due to sepiapterin reductase deficiency, encephalopathy due to GLUT1 deficiency, endocrine system disorder, epilepsy, epilepsy, early-onset, vitamin B6-dependent, epithelial neoplasm, eye disorder, factor VII deficiency, factor X deficiency, familial hemolytic anemia, familial isolated deficiency of vitamin E, fragile X syndrome, fructose-1,6-bisphosphatase deficiency, galactosemia, glucose metabolism disease, glutaryl-CoA dehydrogenase deficiency, glycogen storage disease I, glycogen storage disease II, glycogen storage disease IXb, glycogen storage disease IXc, glycogen storage disease Ib, guanidinoacetate methyltransferase deficiency, heart disorder, heart valve disorder, hematologic disorder, hemolytic anemia, hemophilia A, hemophilia B, hemorrhagic disease, hereditary disease, hereditary fructose intolerance, hereditary neoplastic syndrome, hereditary nephritis, hereditary skin disorder, histiocytosis, holocarboxylase synthetase deficiency, homocystinuria, homocystinuria due to methylene tetrahydrofolate reductase deficiency, hyperammonemia due to N-acetylglutamate synthase deficiency, hyperhomocysteinemia, hyperinsulinemic hypoglycemia, familial, 2, hyperinsulinism, hyperinsulinism-hyperammonemia syndrome, hypoglycemia, hypophosphatasia, hypophosphatemia, hypophosphatemic rickets, hypopigmentation of the skin, hypothyroidism, hypothyroidism due to TSH receptor mutations, immune system disorder, inborn carbohydrate metabolic disorder, inborn disorder of amino acid metabolism, inborn error of immunity, inborn errors of metabolism, inborn metal metabolism disorder, inborn mitochondrial metabolism disorder, inherited blood coagulation disorder, inherited hemoglobinopathy, inherited neurodegenerative disorder, intellectual disability, isovaleric acidemia, kidney disorder, leukocyte disorder, liver disorder, lung disorder, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, malignant endocrine neoplasm, maturity-onset diabetes of the young type 4, medium chain acyl-CoA dehydrogenase deficiency, metabolic disease, metachromatic leukodystrophy, methylcobalamin deficiency type cblE, methylmalonic aciduria and homocystinuria, methylmalonic aciduria and homocystinuria type cblC, methylmalonic aciduria due to methylmalonyl-CoA mutase deficiency, methylmalonic aciduria, cblA type, mitochondrial trifunctional protein deficiency, motor neuron disorder, movement disorder, mucopolysaccharidosis, mucopolysaccharidosis type 1, mucopolysaccharidosis type 2, mucopolysaccharidosis type 3A, mucopolysaccharidosis type 4, mucopolysaccharidosis type 6, mucopolysaccharidosis type 7, multiple carboxylase deficiency, multiple endocrine neoplasia, multiple endocrine neoplasia type 2B, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, neonatal severe primary hyperparathyroidism, neoplasm, nephritis, nervous system disorder, neurodegenerative disease, neuroepithelial neoplasm, neuromuscular disease, non-Langerhans cell histiocytosis, nutritional deficiency disease, nutritional disorder, obesity disorder, ocular cancer, oculocutaneous albinism, optic choroid disorder, ornithine aminotransferase deficiency, ornithine carbamoyltransferase deficiency, ornithine translocase deficiency, overnutrition, pancreas disorder, pancreatic agenesis 1, permanent neonatal diabetes mellitus, phagocyte bactericidal dysfunction, phenylketonuria, phosphorus metabolism disease, pituitary hormone deficiency, combined, 1, platelet storage pool deficiency, post-traumatic stress disorder, primary hyperoxaluria type 1, primary hyperoxaluria type 2, primary hyperoxaluria type 3, primitive neuroectodermal tumor, propionic acidemia, pseudohypoaldosteronism, pseudohypoaldosteronism type 1, psychiatric disorder, pyridoxal phosphate-responsive seizures, pyridoxine-dependent epilepsy, renal tubular transport disease, respiratory system disorder, retinal cancer, retinal disorder, retinoblastoma, riboflavin transporter deficiency, rickets, severe combined immunodeficiency, severe combined immunodeficiency due to DCLRE1C deficiency, severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency, sickle cell disease, skin disorder, skin pigmentation disorder, sphingolipidosis, spinal cord disorder, spinal muscular atrophy, steroid inherited metabolic disorder, sulfite oxidase deficiency due to molybdenum cofactor deficiency type A, thalassemia, thyroid dyshormonogenesis 2A, thyroid dyshormonogenesis 3, thyroid dyshormonogenesis 5, thyroid dyshormonogenesis 6, thyroid gland disorder, transcobalamin II deficiency, transient neonatal diabetes mellitus, tuberous sclerosis 1, tuberous sclerosis 2, tyrosine hydroxylase deficiency, tyrosinemia, urea cycle disorder, urinary system disorder, uveal disorder, very long chain acyl-CoA dehydrogenase deficiency, vitamin D deficiency, vitamin deficiency disorder, von Willebrand disease (hereditary or acquired), von Willebrand disease 3, xanthomatosisActive, not recruitingNot stated2026-07-01NCT03655223
A Study to Evaluate the Safety and Pharmacokinetics of RC001 in Children With Dravet SyndromeEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, epilepsy, generalized epilepsy with febrile seizures plus, type 2, nervous system disorderRecruitingEarly phase 12026-06-30NCT07675746
A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet SyndromeEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, epilepsy, nervous system disorderActive, not recruitingPhase 1 / Phase 22026-06-08NCT06283212
Epidyolex® in Lennox Gastaut, Dravet Syndrome and Tuberous Sclerosis Complex: an Observational Study in ITALYCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Lennox-Gastaut syndrome, Malformations of Cortical Development, Group I, Neoplasms, Multiple Primary, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, central nervous system malformation, cerebral cortical dysplasia, epilepsy, hamartoma, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, tuberous sclerosis, visual epilepsyActive, not recruitingNot stated2026-06-03NCT05485831
Assessment of Safety of the Use of Fenfluramine in Children With Dravet Syndrome Under 24 Months of AgeEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, epilepsy, nervous system disorderRecruitingPhase 42026-05-15NCT06598449
A PET-MRI Study of Serotoninergic Brainstem Pathway in Patients With Dravet SyndromeDeath, Death, Sudden, Drug Resistant Epilepsy, Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Sudden Unexpected Death in Epilepsy, brain disorder, central nervous system disorder, epilepsy, nervous system disorderRecruitingNot applicable2026-05-08NCT07013331
Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene MutationEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, dentatorubral-pallidoluysian atrophy, epilepsy, nervous system disorder, progressive myoclonus epilepsyActive, not recruitingPhase 1 / Phase 22026-04-09NCT06706388
Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene MutationEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, dentatorubral-pallidoluysian atrophy, epilepsy, nervous system disorder, progressive myoclonus epilepsyActive, not recruitingPhase 1 / Phase 22026-04-07NCT07221760
Registry and Natural History Study for Progressive Myoclonus Epilepsy Type 1 (EPM1)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Myoclonus, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Unverricht-Lundborg syndrome, brain disorder, central nervous system disorder, epilepsy, hereditary disease, inherited neurodegenerative disorder, nervous system disorder, neurodegenerative disease, progressive myoclonus epilepsyRecruitingNot stated2026-03-18NCT06593951
Multicentre Real-life Follow-up Study of Rare Epileptic Syndromes in Children and AdolescentsEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Spasms, Infantile, brain disorder, central nervous system disorder, epilepsy, nervous system disorderRecruitingNot stated2026-02-24NCT05126914