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Showing 1–24 of 70 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Developmental and Epileptic Encephalopathy 2 Clear all
Completed Phase 2 Interventional Results available

Fenfluramine in CDKL5 Deficiency Disorder (CDD)

NCT03861871
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 3, 2023 · Synced Sep 17, 2026, 5:14 AM EDT
Completed Phase 3 Interventional Results available

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

NCT02318602
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 25, 2018 · Synced Sep 17, 2026, 5:14 AM EDT
Recruiting Phase 1Phase 2 Interventional

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

NCT05419492
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 20, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

A Clinical Trial of PRAX-562 in Subjects With Developmental and Epileptic Encephalopathies (DEE)

NCT05818553
View directory record Official record
Conditions
SCN2A Encephalopathy, SCN8A Encephalopathy
Interventions
PRAX-562
Drug
Lead sponsor
Praxis Precision Medicines
Industry
Eligibility
1 Year to 18 Years
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
4
States / cities
Atlanta, Georgia • Chicago, Illinois • Minneapolis, Minnesota + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 28, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Completed Phase 3 Interventional Results available

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

NCT04940624
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Not yet recruiting Phase 1Phase 2 Interventional

CBD Delivery with the A-Synaptic GT4 Transdermal Delivery System in with Dravet Syndrome And/or Lennox-Gastaut Syndrome

NCT06738732
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 17, 2024 · Synced Sep 17, 2026, 5:14 AM EDT
Recruiting No phase listed Observational

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

NCT06585605
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Completed Phase 1Phase 2 Interventional

Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies

NCT05364021
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 7, 2024 · Synced Sep 17, 2026, 5:14 AM EDT
Withdrawn Phase 3 Interventional

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

NCT02174094
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 23, 2015 · Synced Sep 17, 2026, 5:14 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives

NCT05635266
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 6, 2024 · Synced Sep 17, 2026, 5:14 AM EDT
Completed Phase 3 Interventional Results available

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

NCT03936777
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 16, 2025 · Synced Sep 17, 2026, 5:14 AM EDT
Active, not recruiting Phase 2 Interventional

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

NCT04740476
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 3, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Enrolling by invitation Phase 3 Interventional

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

NCT06908226
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Terminated Phase 2 Interventional Results available

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

NCT04873869
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 15, 2025 · Synced Sep 17, 2026, 5:14 AM EDT
Recruiting Phase 3 Interventional

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

NCT06660394
View directory record Official record
Conditions
Dravet Syndrome
Interventions
LP352, Placebo
Drug
Lead sponsor
Longboard Pharmaceuticals
Industry
Eligibility
2 Years to 65 Years
Enrollment
104 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
25
States / cities
Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 20 more
Source: ClinicalTrials.gov public record
Updated Aug 9, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Completed Phase 1 Interventional

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

NCT03467113
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 30, 2023 · Synced Sep 17, 2026, 5:14 AM EDT
Completed Phase 2 Interventional Results available

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

NCT02358538
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 20, 2023 · Synced Sep 17, 2026, 5:14 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome

NCT06118255
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 6, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Terminated Phase 3 Interventional Results available

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

NCT05163314
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Completed Phase 2 Interventional Results available

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

NCT03650452
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 13, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Recruiting No phase listed Observational

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

NCT06092346
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Completed Phase 3 Interventional Results available

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

NCT03572933
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 13, 2023 · Synced Sep 17, 2026, 5:14 AM EDT
Recruiting Phase 3 Interventional

A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome

NCT04462770
View directory record Official record
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
31
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Los Angeles, California + 27 more
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Sep 17, 2026, 5:14 AM EDT
Terminated Phase 2 Interventional Results available

A Study of Soticlestat in Adults and Children With Rare Epilepsies

NCT03635073
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Sep 17, 2026, 5:14 AM EDT