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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: Dravet Syndrome (DS), Children Under 2 Years
Interventions: fenfluramine; Drug
Lead sponsor: University of Colorado, Denver; Other
Eligibility: 12 Months to 24 Months
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 1
States / cities: Aurora, Colorado

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: Dravet Syndrome
Interventions: ZX008 (Fenfluramine Hydrochloride); Drug
Lead sponsor: Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.; Industry
Eligibility: 2 Years to 35 Years
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2018
U.S. locations: 2
States / cities: San Francisco, California • Aurora, Colorado

Conditions: Dravet Syndrome
Interventions: ZX008 (Fenfluramine Hydrochloride); Drug
Lead sponsor: Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.; Industry
Eligibility: 2 Years to 35 Years
Enrollment: 375 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2023
U.S. locations: 24
States / cities: Phoenix, Arizona • Tucson, Arizona • Orange, California + 20 more

Conditions: Epilepsy
Interventions: Ganaxolone; Drug
Lead sponsor: Marinus Pharmaceuticals; Industry
Eligibility: 2 Years to 18 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2019
U.S. locations: 9
States / cities: Phoenix, Arizona • Sacramento, California • San Francisco, California + 6 more

Conditions: Rett Syndrome, MECP2 Duplication, CDKL5, FOXG1 Disorders
Interventions: Not listed
Lead sponsor: University of Alabama at Birmingham; Other
Eligibility: Not listed
Enrollment: 752 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2021
U.S. locations: 12
States / cities: Birmingham, Alabama • Oakland, California • San Diego, California + 9 more

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: 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: CDD
Interventions: Fenfluramine Hydrochloride; Drug
Lead sponsor: NYU Langone Health; Other
Eligibility: 2 Years to 18 Years
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2022
U.S. locations: 1
States / cities: New York, New York

Conditions: Dravet Syndrome
Interventions: zorevunersen, Sham Comparator; Drug · Other
Lead sponsor: Stoke Therapeutics, Inc; Industry
Eligibility: 2 Years to 17 Years
Enrollment: 170 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 28
States / cities: Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 23 more

Conditions: 15q Duplication Syndrome, CDKL5 Deficiency Disease
Interventions: Soticlestat; Drug
Lead sponsor: Takeda; Industry
Eligibility: 2 Years to 55 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 8
States / cities: Los Angeles, California • Aurora, Colorado • Norcross, Georgia + 3 more

Conditions: Dravet Syndrome
Interventions: Fenfluramine treatment (Fintepla), Hypercapnia Challenge using the device (RespirAct); Drug · Device
Lead sponsor: The University of Texas Health Science Center, Houston; Other
Eligibility: 16 Years and older
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 1
States / cities: Houston, Texas

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: Epilepsy
Interventions: Cannabidiol; Drug
Lead sponsor: University of Utah; Other
Eligibility: 2 Years to 25 Years
Enrollment: 26 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2019
U.S. locations: 1
States / cities: Salt Lake City, Utah

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: 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: 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: 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: 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

Conditions: Dravet Syndrome
Interventions: ETX101; Drug
Lead sponsor: Encoded Therapeutics; Industry
Eligibility: 6 Months to 17 Years
Enrollment: 47 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2033
U.S. locations: 11
States / cities: San Francisco, California • Aurora, Colorado • Miami, Florida + 8 more

Conditions: Epilepsy in Children, Dyskinesias, Movement Disorders in Children, Neurologic Disorder, Chorea, Myoclonus, Ataxia, Epilepsy, Dystonia Disorder, Movement Disorders
Interventions: Not listed
Lead sponsor: Boston Children's Hospital; Other
Eligibility: 0 Years to 18 Years
Enrollment: 500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2029
U.S. locations: 1
States / cities: Boston, Massachusetts

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: Epilepsy
Interventions: ataluren, Placebo; Drug
Lead sponsor: NYU Langone Health; Other
Eligibility: 2 Years to 12 Years
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2021
U.S. locations: 1
States / cities: New York, New York

Search by objective public record fields.

Early Check: Expanded Screening in Newborns

Assessment of Safety of the Use of Fenfluramine in Children With Dravet Syndrome Under 24 Months of Age

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

A Study to Assess the Usability of the Embrace Seizure Detection Watch in Children and Young Adults With Dravet Syndrome

An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution in Children and Young Adults With Dravet Syndrome

Open-Label PoC Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic Epilepsies

Biobanking of Rett Syndrome and Related Disorders

Verapamil as Therapy for Children and Young Adults With Dravet Syndrome

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

Fenfluramine in CDKL5 Deficiency Disorder (CDD)

A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome

A Multicenter, Open-label, Pilot Study of Soticlestat (TAK-935/OV935) in Participants With 15Q Duplication Syndrome (Dup 15q) or Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder (ARCADE STUDY)

The FINTEPLA as an Anti-SUDEP Therapy in Dravet Syndrome Project

Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies

Cannabidiol (CBD) to 27 Patients (Aged 2 Years - 19 Years) With Drug Resistant Epilepsy

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

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

Registry and Natural History of Epilepsy-Dyskinesia Syndromes

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

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

A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome

A Retrospective Survey-based Multicenter Study to Delineate the Molecular and Phenotypic Spectrum of Epilepsy-dyskinesia Syndromes

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

Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome