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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: SCN8A Developmental and Epileptic Encephalopathy Syndrome
Interventions: NBI-921352, Placebo; Drug
Lead sponsor: Neurocrine Biosciences; Industry
Eligibility: 2 Years to 21 Years
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2023
U.S. locations: 4
States / cities: San Francisco, California • Washington D.C., District of Columbia • Rochester, New York + 1 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: CDKL5 Deficiency Disorder
Interventions: ganaxolone, Placebo; Drug
Lead sponsor: Marinus Pharmaceuticals; Industry
Eligibility: 2 Years to 21 Years
Enrollment: 101 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2021
U.S. locations: 17
States / cities: Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 14 more

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

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: 19
States / cities: Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 15 more

Conditions: CDKL5, CDKL5 Deficiency Disorder, CDD
Interventions: No intervention.; Other
Lead sponsor: University of Colorado, Denver; Other
Eligibility: 1 Month to 100 Years
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 8
States / cities: Los Angeles, California • Aurora, Colorado • Boston, Massachusetts + 5 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: AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of, IMPDH1, OMIM *146690, Retinitis Pigmentosa Type 10, Leber Congenital Amauriosis Type 11, APRT, OMIM *102600, Adenine Phosphoribosyltransferase Deficiency, HPRT1, OMIM *308000 Lesch-Nyhan Disease, XDH, OMIM *607633, Xanthinuria Type 1, SLC2A9, OMIM *606142 Hypouricemia, SLC22A12, OMIM *607096 Hypouricemia, PRPS1 Def, OMIM *311850, Arts Syndrome; Charcot-Marie-Tooth Disease, PRPS1 SA, OMIM *311850 Gout, PRPS-related Phosphoribosylpyrophosphate Synthetase Superactivity, AMPD2, OMIM *102771, Spastic Paraplegia 63; Pontocerebellar Hypoplasia, ITPA, OMIM *147520, Inosine Triphosphatase Deficiency; Developmental and Epileptic Encephalopathy 35, ADSL, OMIM *608222, Adenylosuccinate Lyase Deficiency, PNP, OMIM *164050, Nucleoside Phosphorylase Deficiency, ADA2, OMIM *607575,Sneddon Syndrome; VAIHS, CAD, *1140120, Developmental and Epileptic Encephalopathy, UPB1, OMIM *606673, Beta-ureidopropionase Deficiency, DPYS, OMIM *613326, Dihydropyrimidinase Deficiency, DPYD, OMIM *274270, Dihydropyrimidine Dehydrogenase Deficiency, DHODH, OMIM *126064, Miller Syndrome (Postaxial Acrofacial Dysostosis), UMPS, OMIM *613891, Orotic Aciduria, NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency, UNG, OMIM *191525, Hyper-IgM Syndrome 5, AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2, Purine-Pyrimidine Metabolism, Metabolic Disease
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 1 Month to 100 Years
Enrollment: 999 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2099
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Dravet Syndrome (DS)
Interventions: Soticlestat, Placebo; Drug
Lead sponsor: Takeda; Industry
Eligibility: 2 Years to 21 Years
Enrollment: 144 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2024
U.S. locations: 10
States / cities: Phoenix, Arizona • Los Angeles, California • San Francisco, California + 7 more

Conditions: Dravet Syndrome
Interventions: LP352, Placebo; Drug
Lead sponsor: Longboard Pharmaceuticals; Industry
Eligibility: 2 Years to 65 Years
Enrollment: 160 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 24
States / cities: Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 20 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: 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: 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

Conditions: Epilepsies, Myoclonic
Interventions: Placebo, Lorcaserin; Drug
Lead sponsor: Eisai Inc.; Industry
Eligibility: 2 Years and older
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2024
U.S. locations: 25
States / cities: Birmingham, Alabama • Los Angeles, California • San Diego, California + 20 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: 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
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
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: 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, 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: 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, 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

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

Study to Evaluate NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)

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

Study of Adjunctive Ganaxolone Treatment in Children and Young Adults With CDKL5 Deficiency Disorder

Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome

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

A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathy (DEE)

International CDKL5 Clinical Research Network

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

A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders

A Study of Soticlestat as an Add-on Therapy in Children and Young Adults With Dravet Syndrome

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

Registry and Natural History of Epilepsy-Dyskinesia Syndromes

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

Transcranial Magnetic Stimulation to Measure Cortical Excitability in Dravet Syndrome

A Study of Lorcaserin as Adjunctive Treatment in Participants With Dravet Syndrome

Biobanking of Rett Syndrome and Related Disorders

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

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

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

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

A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome

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

Intermediate-Size Expanded Access Protocol (EAP) for LP352