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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: Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome, Early Infantile Epileptic Encephalopathy, FOXG1 Syndrome, KBG Syndrome, Noonan Syndrome, Severe Hemophilia A, Short Bowel Syndrome, Beta-Propeller Protein-Associated Neurodegeneration, Brain Injury of Prematurity With Periventricular Leukomalacia, Chromosome 17p13.3 Microdeletion Syndrome, Chromosome 1q43-1q44 Deletion, Cockayne Syndrome, Congenital Diaphragmatic Hernia, End-Stage Renal Disease With Cloacal Anomaly, Mitochondrial Depletion Disorder, Severe Factor VII Deficiency
Interventions: Family Centered pediatric palliative care for family caregivers of children with rare diseases.; Behavioral
Lead sponsor: Children's National Research Institute; Other
Eligibility: 12 Months to 99 Years
Enrollment: 480 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2029
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

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: Epileptic Encephalopathy, Continuous Spike and Wave During Sleep
Interventions: NBI-827104; Drug
Lead sponsor: Neurocrine Biosciences; Industry
Eligibility: 4 Years to 12 Years
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 7
States / cities: Orange, California • Aurora, Colorado • Washington D.C., District of Columbia + 4 more

Conditions: Developmental and Epileptic Encephalopathies, Epileptic Spasms, Genetic Epilepsy, Neonatal and Infant Epilepsy
Interventions: Ketogenic diet; Drug
Lead sponsor: Heather Olson; Other
Eligibility: 0 Days to 9 Months
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Epilepsy, Epileptic Encephalopathy
Interventions: Exome and/or whole genome sequencing; Genetic
Lead sponsor: Boston Children's Hospital; Other
Eligibility: Not listed
Enrollment: 5,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2030
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: SCN8A Developmental and Epileptic Encephalopathy Syndrome
Interventions: NBI-921352; Drug
Lead sponsor: Neurocrine Biosciences; Industry
Eligibility: 2 Years to 22 Years
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 4
States / cities: San Francisco, California • Washington D.C., District of Columbia • Rochester, New York + 1 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: SCN2A-DEE, Epilepsy
Interventions: Not listed
Lead sponsor: Praxis Precision Medicines; Industry
Eligibility: 1 Year to 16 Years
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2023
U.S. locations: 1
States / cities: Atlanta, Georgia

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/or Epileptic Encephalopathies
Interventions: TAK-935, Placebo; Drug
Lead sponsor: Takeda; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2018
U.S. locations: 11
States / cities: Phoenix, Arizona • Port Charlotte, Florida • Tampa, Florida + 8 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: Developmental and Epileptic Encephalopathy 1
Interventions: 1.0mg/kg/day PRAX-562, 1.5mg/kg/day PRAX-562, Placebo; Drug
Lead sponsor: Praxis Precision Medicines; Industry
Eligibility: 2 Years to 65 Years
Enrollment: 160 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 6
States / cities: La Jolla, California • Gulf Breeze, Florida • Chevy Chase, Maryland + 3 more

Conditions: Epileptic Encephalopathy, SCN2A Encephalopathy
Interventions: 1mg elsunersen, 0.5mg elsunersen; Drug
Lead sponsor: Praxis Precision Medicines; Industry
Eligibility: 1 Day to 18 Years
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 6
States / cities: San Diego, California • Aurora, Colorado • Chicago, Illinois + 3 more

Conditions: Neonatal Seizure, Hypoxic-Ischemic Encephalopathy, Stroke, Intracranial Hemorrhage, Epilepsy, Gene Abnormality
Interventions: Not listed
Lead sponsor: University of California, San Francisco; Other
Eligibility: Not listed
Enrollment: 300 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 8
States / cities: San Francisco, California • Washington D.C., District of Columbia • Boston, Massachusetts + 4 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: Neonatal Seizure, Hypoxic-Ischemic Encephalopathy, Stroke, Intracranial Hemorrhages, Epilepsy, Cerebral Palsy, Intellectual Disability
Interventions: Not listed
Lead sponsor: University of California, San Francisco; Other
Eligibility: 2 Years to 8 Years
Enrollment: 188 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2025
U.S. locations: 9
States / cities: San Francisco, California • Stanford, California • Washington D.C., District of Columbia + 5 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: Nonconvulsive Status Epilepticus, Subclinical Seizure, Altered Mental Status, Nonepileptic Seizures, Encephalopathy
Interventions: EEG Cap, Anti-seizure medication, Standard EEG; Device · Drug · Procedure
Lead sponsor: Mayo Clinic; Other
Eligibility: 18 Years and older
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2018
U.S. locations: 1
States / cities: Jacksonville, Florida

Conditions: Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome, Mitochondrial Encephalopathy (MELAS), Pontocerebellar Hypoplasia Type 6 (PCH6), Alpers Disease, Alpers Syndrome
Interventions: Vatiquinone, Placebo; Drug · Other
Lead sponsor: PTC Therapeutics; Industry
Eligibility: Up to 20 Years
Enrollment: 68 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2023
U.S. locations: 12
States / cities: San Diego, California • Stanford, California • New Haven, Connecticut + 7 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: Chronic Pain, Chronic Pain Syndrome, Chronic Pain Due to Injury, Chronic Pain Due to Trauma, Fibromyalgia, Seizures, Hepatitis C, Cancer, Crohn Disease, HIV/AIDS, Multiple Sclerosis, Traumatic Brain Injury, Sickle Cell Disease, Post Traumatic Stress Disorder, Tourette Syndrome, Ulcerative Colitis, Glaucoma, Epilepsy, Inflammatory Bowel Diseases, Parkinson Disease, Amyotrophic Lateral Sclerosis, Chronic Traumatic Encephalopathy, Anxiety, Depression, Insomnia, Autism, Opioid-use Disorder, Bipolar Disorder, Covid19, SARS-CoV Infection, COVID-19, Corona Virus Infection, Coronavirus
Interventions: Cannabis, Medical, RYAH-Medtech Inhaler; Drug · Device
Lead sponsor: OMNI Medical Services, LLC; Network
Eligibility: 7 Years and older
Enrollment: 200,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2025
U.S. locations: 17
States / cities: Boca Raton, Florida • Bradenton, Florida • Fort Lauderdale, Florida + 13 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

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

Palliative Care Needs of Children With Rare Diseases and Their Families

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

Extension Study to Evaluate NBI-827104 in Pediatric Participants With Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep

Ketogenic Diet for Prevention of Epileptic Spasms in Infantile Onset Genetic Epilepsies

Genetics of Epilepsy and Related Disorders

Extension Study to Evaluate How Safe and Tolerable NBI-921352 is as an Adjunctive Therapy for Participants With SCN8A-DEE

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

A Prospective, Remote Observational Study in Pediatric Participants With Early-Onset SCN2A-Developmental and Epileptic Encephalopathy

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

Study of TAK-935 as an Adjunctive Therapy in Participants With Developmental and/or Epileptic Encephalopathies

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

A Clinical Trial for Participants With DEE to Assess Efficacy, Safety, Tolerability, and PK of Relutrigine

A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety

Neonatal Seizure Registry, GEnetics of Post-Neonatal Epilepsy

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

Neonatal Seizure Registry - Developmental Functional EValuation

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

EEG Cap for Identification of Non-Convulsive Status Epilepticus

A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

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

Outcomes Mandate National Integration With Cannabis as Medicine

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