- Conditions
- Dravet Syndrome
- Interventions
- Transcranial Magnetic Stimulation (TMS), Electroencephalogram (EEG)
- Device
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 6 Months to 30 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2022
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
Search Results
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Showing 25–48
of 24
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS, MERRF
- Interventions
- Promoting Resilience in Stress Management (PRISM), Clinical-focused narrative (CFN)
- Behavioral
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 16 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
- Developmental and Epileptic Encephalopathy
- Interventions
- LP352
- Drug
- Lead sponsor
- Longboard Pharmaceuticals
- Industry
- Eligibility
- 2 Years to 66 Years
- Enrollment
- 324 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 20
- States / cities
- Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 16 more
- Conditions
- Epilepsy
- Interventions
- Lacosamide
- Drug
- Lead sponsor
- UCB BIOSCIENCES, Inc.
- Industry
- Eligibility
- 16 Years to 65 Years
- Enrollment
- 39 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2012
- U.S. locations
- 19
- States / cities
- Phoenix, Arizona • Little Rock, Arkansas • Aurora, Colorado + 16 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
- 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
- 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
- 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
- 5
- States / cities
- Ann Arbor, Michigan • Rochester, Minnesota • Durham, North Carolina + 2 more
- Conditions
- Dravet Syndrome
- Interventions
- GABA Blood Level
- Diagnostic Test
- Lead sponsor
- Cook Children's Health Care System
- Other
- Eligibility
- Up to 18 Years
- Enrollment
- 36 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2027
- U.S. locations
- 1
- States / cities
- Fort Worth, Texas
- Conditions
- Dravet Syndrome, Lennox Gastaut Syndrome, Epileptic Encephalopathy
- Interventions
- ZX008 (Fenfluramine Hydrochloride)
- Drug
- Lead sponsor
- Zogenix, Inc.
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 412 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 26
- States / cities
- Tucson, Arizona • Los Angeles, California • San Diego, California + 23 more
- Conditions
- Lafora Disease
- Interventions
- Not listed
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 5 Years and older
- Enrollment
- 33 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Dallas, Texas
- 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
- 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
- Generalized Epilepsy
- Interventions
- BHV-7000, Placebo
- Drug
- Lead sponsor
- Biohaven Therapeutics Ltd.
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 40
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 34 more
- Conditions
- Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes, Deletion and Duplication of Mitochondrial DNA
- Interventions
- Not listed
- Lead sponsor
- Columbia University
- Other
- Eligibility
- Not listed
- Enrollment
- 1,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2026
- U.S. locations
- 16
- States / cities
- San Diego, California • Stanford, California • Aurora, Colorado + 12 more
- Conditions
- Dentatorubral-Pallidoluysian Atrophy
- Interventions
- Positive genetic test for pathological expansion in ATN1
- Other
- Lead sponsor
- University College, London
- Other
- Eligibility
- Not listed
- Enrollment
- 225 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2026
- U.S. locations
- 2
- States / cities
- New York, New York • Chapel Hill, North Carolina
- 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
- 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
- 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
- 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
- Progressive Myoclonus Epilepsy Type 1, EPM1, CSTB-related Disease, Myoclonus Epilepsies, Progressive, Unverricht-Lundborg Disease, Progressive Epilepsy and/or Ataxia With Myoclonus as a Major Feature, PME, Progressive Myoclonus-Epilepsies
- Interventions
- Not listed
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2030
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease, Amyloidosis, Ankylosing Spondylitis, Arthritis, Alopecia Areata, Asthma, Atopic Dermatitis, Autism, Autoimmune Hepatitis, Behcet's Disease, Beta-Thalassemia, Cancer, Celiac Disease, Kidney Diseases, COPD, Crohn Disease, Cystic Fibrosis, Diabetes, Dravet Syndrome, DMD, Fibromyalgia, Graves Disease, Thyroid Diseases, Hepatitis, Hidradenitis Suppurativa, ITP, Leukemia, ALS, Lupus or SLE, Lymphoma, Multiple Sclerosis, Myasthenia Gravis, Heart Diseases, Parkinson Disease, Pemphigus Vulgaris, Cirrhosis, Psoriasis, Schizophrenia, Scleroderma, Sickle Cell Disease, Stroke, Ulcerative Colitis, Vasculitis, Vitiligo
- Interventions
- Specimen sample
- Diagnostic Test
- Lead sponsor
- Sanguine Biosciences
- Industry
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 20,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2025
- U.S. locations
- 1
- States / cities
- Waltham, Massachusetts
- Conditions
- Lafora Disease
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2002
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland