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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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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
Dravet Syndrome
Interventions
Clemizole HCl, Placebo
Drug
Lead sponsor
Epygenix
Industry
Eligibility
2 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2029
U.S. locations
32
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Los Angeles, California + 27 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
Lennox-Gastaut Syndrome (LGS), Dravet Syndrome (DS)
Interventions
CBD: GT4 Transdermal Delivery System
Drug
Lead sponsor
Alexander Rotenberg
Other
Eligibility
2 Years to 55 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
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
Dravet Syndrome
Interventions
ION337
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 12 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
3
States / cities
Ann Arbor, Michigan • Rochester, Minnesota • Memphis, Tennessee
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
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Early Check: Expanded Screening in Newborns

NCT03655223
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
Epilepsy, Dravet Syndrome
Interventions
GWP42003-P, Placebo Control
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
2 Years to 18 Years
Enrollment
199 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
23
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Los Angeles, California + 20 more
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
Rett Syndrome, MECP2 Duplication Disorder, Rett-related Disorder
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
11
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 8 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
Dravet Syndrome (DS), Lennox Gastaut Syndrome (LGS)
Interventions
Soticlestat
Drug
Lead sponsor
Takeda
Industry
Eligibility
2 Years to 56 Years
Enrollment
352 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
26
States / cities
Phoenix, Arizona • Tucson, Arizona • Los Angeles, California + 19 more
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
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
Dravet Syndrome
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
87 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
5
States / cities
San Francisco, California • Aurora, Colorado • Chicago, Illinois + 2 more
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
Rett Syndrome, MECP2 Duplication dIsorder, CDKL5 Disorder, FOXG1 Syndrome
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
Not listed
Enrollment
1,044 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
14
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 11 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, 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
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