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Conditions: Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome, Seizure in Participants With Lennox-Gastaut Syndrome
Interventions: GWP42003-P; Drug
Lead sponsor: Jazz Pharmaceuticals; Industry
Eligibility: 1 Month to 23 Months
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2025
U.S. locations: 6
States / cities: Little Rock, Arkansas • Los Angeles, California • Chicago, Illinois + 3 more

Conditions: Dravet Syndrome
Interventions: Clobazam; Drug
Lead sponsor: H. Lundbeck A/S; Industry
Eligibility: 1 Year to 16 Years
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015
U.S. locations: 8
States / cities: Los Angeles, California • Orange, California • Orlando, Florida + 4 more

Conditions: Dravet Syndrome
Interventions: Retrospective study; Other
Lead sponsor: Biocodex; Industry
Eligibility: Not listed
Enrollment: 100 participants
Timeline: 2022 – 2024
U.S. locations: 1
States / cities: Rochester, Minnesota

Conditions: Dravet Syndrome
Interventions: zorevunersen (STK-001); Drug
Lead sponsor: Stoke Therapeutics, Inc; Industry
Eligibility: 30 Months and older
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2029
U.S. locations: 17
States / cities: San Francisco, California • Aurora, Colorado • Washington D.C., District of Columbia + 14 more

Conditions: Epilepsies, Myoclonic, Drug Resistant Epilepsy
Interventions: Lorcaserin; Drug
Lead sponsor: Eisai Inc.; Industry
Eligibility: 2 Years and older
U.S. locations: 18
States / cities: Birmingham, Alabama • Los Angeles, California • Aurora, Colorado + 14 more

Conditions: Epilepsy, Dravet Syndrome
Interventions: GWP42003-P 20 mg/kg/day Dose, Placebo control; Drug
Lead sponsor: Jazz Pharmaceuticals; Industry
Eligibility: 2 Years to 18 Years
Enrollment: 120 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015
U.S. locations: 13
States / cities: Miami, Florida • Orlando, Florida • Atlanta, Georgia + 10 more

Conditions: Myoclonus Epilepsies, Progressive
Interventions: AUT00201, Placebo; Drug
Lead sponsor: Autifony Therapeutics Limited; Industry
Eligibility: 18 Years and older
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2024
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
Interventions: TAK-935, Placebo; Drug
Lead sponsor: Takeda; Industry
Eligibility: 2 Years to 17 Years
Enrollment: 141 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 15
States / cities: Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 12 more

Conditions: Dravet Syndrome
Interventions: stiripentol; Drug
Lead sponsor: Mayo Clinic; Other
Eligibility: 11 Years and older · Female only
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2013
U.S. locations: 1
States / cities: Rochester, Minnesota

Conditions: Dravet Syndrome, Epileptic Encephalopathies Associated With SCN1A Mutations
Interventions: Stiripentol; Drug
Lead sponsor: Cook Children's Health Care System; Other
Eligibility: 6 Months to 18 Years
U.S. locations: 1
States / cities: Fort Worth, Texas

Conditions: CDKL5 Deficiency Disorder (CDD)
Interventions: Survey Instrument; Other
Lead sponsor: University of Pennsylvania; Other
Eligibility: Not listed
Enrollment: 500 participants
Timeline: 2018 – 2028
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Unverricht-Lundborg Disease
Interventions: Placebo, BRV 2.5 mg, BRV 25 mg, BRV 50 mg; Other · Drug
Lead sponsor: UCB Pharma; Industry
Eligibility: 16 Years and older
Enrollment: 56 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2008
U.S. locations: 4
States / cities: San Francisco, California • Gainesville, Florida • New York, New York + 1 more

Conditions: Dravet Syndrome
Interventions: Clobazam, Placebo; Drug
Lead sponsor: H. Lundbeck A/S; Industry
Eligibility: 1 Year to 16 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015
U.S. locations: 8
States / cities: Los Angeles, California • Orlando, Florida • Rochester, Minnesota + 4 more

Conditions: Dravet Syndrome
Interventions: EPX-100 (Clemizole Hydrochloride), Placebos; Drug
Lead sponsor: Epygenix; Industry
Eligibility: 18 Years to 50 Years
Enrollment: 24 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2019
U.S. locations: 1
States / cities: Fair Lawn, New Jersey

Conditions: Dravet Syndrome, Lennox Gastaut Syndrome
Interventions: ZX008 0.2 to 0.8 mg/kg/day, Cannabidiol; Drug
Lead sponsor: Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.; Industry
Eligibility: 2 Years to 18 Years
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 2
States / cities: Atlanta, Georgia • Tacoma, Washington

Conditions: Familial Dementia With Neuroserpin Inclusion Bodies, Nervous System Heredodegenerative Disorder
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 10 Years and older
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2009
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Healthy, Myoclonic Epilepsy
Interventions: Not listed
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: Not listed
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2001
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Inherited Mitochondrial Disease
Interventions: Vatiquinone; Drug
Lead sponsor: PTC Therapeutics; Industry
Eligibility: Not listed
Enrollment: 101 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 14
States / cities: La Jolla, California • Stanford, California • New Haven, Connecticut + 10 more

Conditions: Developmental and Epileptic Encephalopathy, Dravet Syndrome, Lennox Gastaut Syndrome
Interventions: LP352, Placebo; Drug
Lead sponsor: Longboard Pharmaceuticals; Industry
Eligibility: 12 Years to 65 Years
Enrollment: 52 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2023
U.S. locations: 29
States / cities: Tucson, Arizona • Little Rock, Arkansas • Downey, California + 24 more

Conditions: Dravet Syndrome
Interventions: Not listed
Lead sponsor: University of Colorado, Denver; Other
Eligibility: Up to 50 Years
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2017
U.S. locations: 1
States / cities: Denver, Colorado

Conditions: Seizures Associated With EMAS
Interventions: GWP42003-P, Placebo; Drug
Lead sponsor: Jazz Pharmaceuticals; Industry
Eligibility: 1 Year to 18 Years
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2023
U.S. locations: 10
States / cities: Birmingham, Alabama • Sacramento, California • Atlanta, Georgia + 7 more

Conditions: Seizures
Interventions: Cannabidiol Oral Solution; Drug
Lead sponsor: INSYS Therapeutics Inc; Industry
Eligibility: 1 Year to 18 Years
Enrollment: 52 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2017
U.S. locations: 11
States / cities: San Francisco, California • Miami, Florida • Pensacola, Florida + 8 more

Conditions: Cyclin-Dependent Kinase-Like 5 Deficiency Disorder
Interventions: No intervention; observational; Other
Lead sponsor: Loulou Foundation; Other
Eligibility: Up to 55 Years
Enrollment: 112 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 9
States / cities: Los Angeles, California • Aurora, Colorado • Norcross, Georgia + 6 more

Conditions: Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy, Hyperinsulinemic Hypoglycemia, Familial 1, Diabetes Mellitus, Adrenoleukodystrophy, Neonatal, Medium-chain Acyl-CoA Dehydrogenase Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Beta-ketothiolase Deficiency, Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency, Primary Hyperoxaluria Type 1, Congenital Bile Acid Synthesis Defect Type 2, Pyridoxine-Dependent Epilepsy, Hereditary Fructose Intolerance, Hypophosphatasia, Hyperargininemia, Mucopolysaccharidosis Type 6, Argininosuccinic Aciduria, Citrullinemia, Type I, Wilson Disease, Maple Syrup Urine Disease, Type 1A, Maple Syrup Urine Disease, Type 1B, Biotinidase Deficiency, Neonatal Severe Primary Hyperparathyroidism, Intrinsic Factor Deficiency, Usher Syndrome Type 1D/F Digenic (Diagnosis), Cystic Fibrosis, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Carbamoyl Phosphate Synthetase I Deficiency Disease, Carnitine Palmitoyl Transferase 1A Deficiency, Carnitine Palmitoyltransferase II Deficiency, Cystinosis, Chronic Granulomatous Disease, Cerebrotendinous Xanthomatoses, Maple Syrup Urine Disease, Type 2, Severe Combined Immunodeficiency Due to DCLRE1C Deficiency, Thyroid Dyshormonogenesis 6, Thyroid Dyshormonogenesis 5, Supravalvar Aortic Stenosis, Factor X Deficiency, Hemophilia A, Hemophilia B, Tyrosinemia, Type I, Fructose 1,6 Bisphosphatase Deficiency, Glycogen Storage Disease Type I, G6PD Deficiency, Glycogen Storage Disease II, Galactokinase Deficiency, Mucopolysaccharidosis Type IV A, Galactosemias, Guanidinoacetate Methyltransferase Deficiency, Agat Deficiency, Glutaryl-CoA Dehydrogenase Deficiency, Gtp Cyclohydrolase I Deficiency, Hyperinsulinism-Hyperammonemia Syndrome, Primary Hyperoxaluria Type 2, 3-Hydroxyacyl-CoA Dehydrogenase Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Mitochondrial Trifunctional Protein Deficiency, Sickle Cell Disease, Beta-Thalassemia, Holocarboxylase Synthetase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, Primary Hyperoxaluria Type 3, Hermansky-Pudlak Syndrome 1, Hermansky-Pudlak Syndrome 4, Apparent Mineralocorticoid Excess, HSDB, CBAS1, Mucopolysaccharidosis Type 2, Mucopolysaccharidosis Type 1, Severe Combined Immunodeficiency, X Linked, Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency, Diabetes Mellitus, Permanent Neonatal, Isovaleric Acidemia, Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder), Jervell and Lange-Nielsen Syndrome 2, Hyperinsulinemic Hypoglycemia, Familial, 2, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Jervell and Lange-Nielsen Syndrome 1, Lysosomal Acid Lipase Deficiency, CblF, 3-Methylcrotonyl CoA Carboxylase 1 Deficiency, 3-Methylcrotonyl CoA Carboxylase 2 Deficiency, Waardenburg Syndrome Type 2A, Methylmalonic Aciduria cblA Type, Methylmalonic Aciduria cblB Type, Methylmalonic Aciduria and Homocystinuria Type cblC, MAHCD, Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency, Congenital Disorder of Glycosylation Type 1B, Mthfr Deficiency, Methylcobalamin Deficiency Type Cbl G (Disorder), Methylcobalamin Deficiency Type cblE, Usher Syndrome, Type 1B, N-acetylglutamate Synthase Deficiency, Ornithine Transcarbamylase Deficiency, Phenylketonurias, Waardenburg Syndrome Type 1, Congenital Hypothyroidism, Propionic Acidemia, Usher Syndrome, Type 1F, Pancreatic Agenesis 1, Hereditary Hypophosphatemic Rickets, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, MOWS, Epilepsy, Early-Onset, Vitamin B6-Dependent, Pyridoxal Phosphate-Responsive Seizures, Pituitary Hormone Deficiency, Combined, 1, Ptsd, Dihydropteridine Reductase Deficiency, Severe Combined Immunodeficiency Due to RAG1 Deficiency, Severe Combined Immunodeficiency Due to RAG2 Deficiency, Retinoblastoma, Multiple Endocrine Neoplasia Type 2B, Pseudohypoaldosteronism, Type I, Liddle Syndrome, Biotin-Responsive Basal Ganglia Disease, SCD, DIAR1, GSD1C, Acrodermatitis Enteropathica, Thyroid Dyshormonogenesis 1, Riboflavin Transporter Deficiency, Waardenburg Syndrome, Type 2E, SRD, Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency, Barth Syndrome, Adrenocorticotropic Hormone Deficiency, Transcobalamin II Deficiency, Thyroid Dyshormonogenesis 3, Segawa Syndrome, Autosomal Recessive, Autosomal Recessive Nonsyndromic Hearing Loss, Thyroid Dyshormonogenesis 2A, Congenital Isolated Thyroid Stimulating Hormone Deficiency, Hypothyroidism Due to TSH Receptor Mutations, Usher Syndrome Type 1C, Usher Syndrome Type 1G (Diagnosis), Von Willebrand Disease, Type 3, Combined Immunodeficiency Due to ZAP70 Deficiency, Adenine Phosphoribosyltransferase Deficiency, Metachromatic Leukodystrophy, Canavan Disease, Menkes Disease, Carbonic Anhydrase VA Deficiency, Developmental and Epileptic Encephalopathy 2, 17 Alpha-Hydroxylase Deficiency, Smith-Lemli-Opitz Syndrome, Krabbe Disease, Glutathione Synthetase Deficiency, Mucopolysaccharidosis Type 7, Rett Syndrome, Molybdenum Cofactor Deficiency, Type A, Niemann-Pick Disease, Type C1, Niemann-Pick Disease Type C2, Ornithine Aminotransferase Deficiency, 3-Phosphoglycerate Dehydrogenase Deficiency, Leber Congenital Amaurosis 2, Dravet Syndrome, Mucopolysaccharidosis Type 3 A, Ornithine Translocase Deficiency, Carnitine-acylcarnitine Translocase Deficiency, Glucose Transporter Type 1 Deficiency Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type A, Pitt Hopkins Syndrome, Tuberous Sclerosis 1, Tuberous Sclerosis 2, Ataxia With Isolated Vitamin E Deficiency, Angelman Syndrome, Prader-Willi Syndrome, Homocystinuria, Permanent Neonatal Diabetes Mellitus, Transient Neonatal Diabetes Mellitus, Factor VII Deficiency, Glycogen Storage Disease Type IXA1, Glycogen Storage Disease, Type IXA2, Glycogen Storage Disease IC, Glycogen Storage Disease Type IB, Central Hypoventilation Syndrome With or Without Hirschsprung Disease
Interventions: Confirmatory Testing; Diagnostic Test
Lead sponsor: RTI International; Other
Eligibility: 1 Day to 31 Days
Enrollment: 30,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2025
U.S. locations: 1
States / cities: Research Triangle Park, North Carolina

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Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures

Safety and Tolerability of Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome

Retrospective Non-interventional Study of Stiripentol Use in Dravet Patients in the USA

An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome

Extended Access Program With Lorcaserin For The Treatment of Dravet Syndrome and Other Refractory Epilepsies

Antiepileptic Efficacy Study of GWP42003-P in Children and Young Adults With Dravet Syndrome (GWPCARE1)

Safety, Blood Levels and Effects of AUT00201 in Patients With MEAK

A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies

Stiripentol in Dravet Syndrome

Expanded Access Use of Stiripentol in Dravet Syndrome or Sodium Channel Mutation Epileptic Encephalopathies

International CDKL5 Registry

Brivaracetam as add-on Treatment of Unverricht-Lundborg Disease (ULD) in Adolescents and Adults

Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome

Study of Safety and Pharmacokinetics of Oral Doses of EPX-100 in Healthy Subjects.

A Study to Assess the Safety and Tolerability of ZX008 in Children and Young Adults With Dravet Syndrome or Lennox Gastaut Syndrome Currently Taking Cannabidiol

Clinical and Genetic Studies of Familial Presenile Dementia With Neuronal Inclusion Bodies

Effect of Levetiracetam on Brain Excitability

A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies

Genetic Analysis Between Charlotte's Web Responders Versus Non- Responders in a Dravet Population

Efficacy and Safety of GWP42003-P Oral Solution in Children With Epilepsy With Myoclonic-atonic Seizures

Cannabidiol Oral Solution as an Adjunctive Treatment for Treatment-resistant Seizure Disorder

Observational Study in Patients With Cyclin-dependent Kinase-like 5 Deficiency Disorder

Early Check: Expanded Screening in Newborns