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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: Lennox Gastaut Syndrome, Dravet Syndrome, Tuberous Sclerosis Complex
Interventions: Cannabidiol; Drug
Lead sponsor: Jazz Pharmaceuticals; Industry
Eligibility: 1 Year and older
Enrollment: 154 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2028
U.S. locations: 26
States / cities: Little Rock, Arkansas • Downey, California • Long Beach, California + 22 more

Conditions: Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome, Tuberous Sclerosis, Focal Seizures
Interventions: Turmeric; Dietary Supplement
Lead sponsor: NYU Langone Health; Other
Eligibility: 1 Year to 70 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018
U.S. locations: 1
States / cities: New York, New York

Conditions: Dravet Syndrome
Interventions: No Intervention; Other
Lead sponsor: Encoded Therapeutics; Industry
Eligibility: 6 Months to 60 Months
Enrollment: 58 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 12
States / cities: Los Angeles, California • San Francisco, California • Aurora, Colorado + 9 more

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: 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: Lennox-Gastaut Syndrome, Dravet Syndrome
Interventions: 12-Lead ECG, Cannabidiol; Procedure · Drug
Lead sponsor: Gillette Children's Specialty Healthcare; Other
Eligibility: 2 Years to 30 Years
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2018
U.S. locations: 1
States / cities: Saint Paul, Minnesota

Conditions: Dravet Syndrome
Interventions: Fenfluramine; Drug
Lead sponsor: University of California, Los Angeles; Other
Eligibility: 2 Years and older
U.S. locations: 1
States / cities: Los Angeles, California

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: Dravet Syndrome, Lennox Gastaut Syndrome, Developmental and Epileptic Encephalopathies
Interventions: LP352; Drug
Lead sponsor: Longboard Pharmaceuticals; Industry
Eligibility: 2 Years to 65 Years
U.S. locations: 19
States / cities: Downey, California • Los Angeles, California • San Francisco, California + 15 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-Dyskinesia, Epilepsy, Dyskinesia, EDS, Epilepsy-Dyskinesia Syndomes, Epilepsy in Children, Dyskinesias, Movement Disorders in Children, Neurologic Disorder, Chorea, Myoclonus, Ataxia, Dystonia Disorder, Movement Disorders
Interventions: Not listed
Lead sponsor: Boston Children's Hospital; Other
Eligibility: 0 Years to 30 Years
Enrollment: 700 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2030
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Dravet Syndrome
Interventions: Verapamil; Drug
Lead sponsor: Gillette Children's Specialty Healthcare; Other
Eligibility: 2 Years to 25 Years
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2015
U.S. locations: 4
States / cities: Chicago, Illinois • Rochester, Minnesota • Saint Paul, Minnesota + 1 more

Conditions: Epilepsy, Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS)
Interventions: Soticlestat; Drug
Lead sponsor: Takeda; Industry
Eligibility: 2 Years and older
Enrollment: 156 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2025
U.S. locations: 27
States / cities: Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 18 more

Conditions: Dravet Syndrome
Interventions: Stiripentol; Drug
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 6 Months and older
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Dravet Syndrome
Interventions: fenfluramine; Drug
Lead sponsor: UCB BIOSCIENCES, Inc.; Industry
Eligibility: 1 Year to 23 Months
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 3
States / cities: Memphis, Tennessee • Dallas, Texas • Seattle, Washington

Conditions: Dravet Syndrome, Seizure Disorder
Interventions: ZX008 (Fenfluramine Hydrochloride), Matching Placebo; Drug
Lead sponsor: Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.; Industry
Eligibility: 2 Years to 18 Years
Enrollment: 262 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2020
U.S. locations: 19
States / cities: Phoenix, Arizona • Tucson, Arizona • San Diego, California + 15 more

Conditions: Dravet Syndrome
Interventions: STK-001 - Single Ascending Doses, STK-001 - Multiple Ascending Doses; Drug
Lead sponsor: Stoke Therapeutics, Inc; Industry
Eligibility: 2 Years to 18 Years
Enrollment: 62 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2023
U.S. locations: 18
States / cities: San Francisco, California • Aurora, Colorado • Washington D.C., District of Columbia + 15 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: 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 – 2026
U.S. locations: 1
States / cities: Research Triangle Park, North Carolina

Conditions: CDKL5 Deficiency Disorder, Generalized Tonic Clonic Seizure, Epileptic Spasm, Refractory Seizures
Interventions: Fenfluramine, Placebo; Drug
Lead sponsor: Zogenix, Inc.; Industry
Eligibility: 1 Year to 35 Years
Enrollment: 87 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 13
States / cities: Birmingham, Alabama • Los Angeles, California • San Francisco, California + 10 more

Conditions: Dravet Syndrome
Interventions: LP352, Placebo; Drug
Lead sponsor: Longboard Pharmaceuticals; Industry
Eligibility: 2 Years to 65 Years
Enrollment: 104 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 25
States / cities: Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 20 more

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: Epilepsy, Dravet Syndrome
Interventions: GWP42003-P 5 mg/kg/day Dose, Placebo control, GWP42003-P 10 mg/kg/day Dose, GWP42003-P 20 mg/kg/day Dose; Drug
Lead sponsor: Jazz Pharmaceuticals; Industry
Eligibility: 4 Years to 10 Years
Enrollment: 34 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2015
U.S. locations: 8
States / cities: Miami, Florida • Atlanta, Georgia • Chicago, Illinois + 5 more

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Stiripentol in Dravet Syndrome

Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution

Turmeric as Treatment in Epilepsy

Natural History Study of Infants and Children With Developmental and Epileptic Encephalopathies

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

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

The Effects of Cannabidiol (CBD) on Electrical and Autonomic Cardiac Function in Children With Severe Epilepsy

Treatment of Dravet Syndrome With Fenfluramine (Expanded Access Protocol)

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

Intermediate-Size Expanded Access Protocol (EAP) for LP352

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

Registry and Natural History of Epilepsy-Dyskinesia Syndromes

Verapamil as Therapy for Children and Young Adults With Dravet Syndrome

A Study of Soticlestat in Adults and Children With Rare Epilepsies

Treatment Plan to Provide Expanded Access to Stiripentol for Patients With Dravet Syndrome

A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome

A Trial of Two Fixed Doses of ZX008 (Fenfluramine HCl) in Children and Young Adults With Dravet Syndrome

An Open-Label Study to Investigate the Safety of Single and Multiple Ascending Doses in Children and Adolescents With Dravet Syndrome

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

Early Check: Expanded Screening in Newborns

A Phase 3 Study to Examine the Efficacy and Safety of ZX008 in Subjects With CDKL5 Deficiency Disorder.

A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)

International CDKL5 Registry

A Dose-ranging Pharmacokinetics and Safety Study of GWP42003-P in Children With Dravet Syndrome (GWPCARE1)