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ClinicalTrials.gov public records Last synced Oct 2, 2026, 2:57 PM EDT

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Showing 25–48 of 80 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Epilepsies, Myoclonic Clear all
Completed Phase 3 Interventional Results available

GWPCARE2 A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet Syndrome

NCT02224703
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 28, 2022 · Synced Oct 2, 2026, 2:57 PM 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 Dec 1, 2023 · Synced Oct 2, 2026, 2:57 PM EDT
Suspended No phase listed Observational

International CDKL5 Registry

NCT04486768
View directory record Official record
Conditions
CDKL5 Deficiency Disorder (CDD)
Interventions
Survey Instrument
Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
Not listed
Enrollment
500 participants
Timeline
2018 – 2028
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 21, 2026 · Synced Oct 2, 2026, 2:57 PM EDT
Terminated Phase 2Phase 3 Interventional

A Study to Determine if BHV-7000 is Effective and Safe in Adults With Idiopathic Generalized Epilepsy With Generalized Tonic-clonic Seizures

NCT06425159
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced Oct 2, 2026, 2:57 PM EDT
Not listed No phase listed Observational

Transcranial Magnetic Stimulation to Measure Cortical Excitability in Dravet Syndrome

NCT04614506
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 22, 2022 · Synced Oct 2, 2026, 2:57 PM EDT
Recruiting No phase listed Observational

Registry and Natural History Study for Progressive Myoclonus Epilepsy Type 1 (EPM1)

NCT06593951
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 18, 2026 · Synced Oct 2, 2026, 2:57 PM EDT
Completed Phase 2 Interventional Results available

Verapamil as Therapy for Children and Young Adults With Dravet Syndrome

NCT01607073
View directory record Official record
Conditions
Dravet Syndrome
Interventions
Verapamil
Drug
Lead sponsor
Gillette Children's Specialty Healthcare
Other
Eligibility
2 Years to 25 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
4
States / cities
Chicago, Illinois • Rochester, Minnesota • Saint Paul, Minnesota + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2021 · Synced Oct 2, 2026, 2:57 PM EDT
Completed Phase 2 Interventional Results available

Open-Label Study to Assess Lacosamide Safety as Add-on Therapy for Primary Generalized Tonic-Clonic Seizures in Subjects With Epilepsy

NCT01118949
View directory record Official record
Conditions
Epilepsy
Interventions
Lacosamide
Drug
Lead sponsor
UCB BIOSCIENCES, Inc.
Industry
Eligibility
16 Years to 65 Years
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
25
States / cities
Alabaster, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 22 more
Source: ClinicalTrials.gov public record
Updated Jul 17, 2018 · Synced Oct 2, 2026, 2:57 PM 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 15, 2026 · Synced Oct 2, 2026, 2:57 PM 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 7, 2024 · Synced Oct 2, 2026, 2:57 PM 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 15, 2026 · Synced Oct 2, 2026, 2:57 PM EDT
Completed Phase 2 Interventional Results available

Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease

NCT02023866
View directory record Official record
Conditions
Inherited Mitochondrial Disease, Including Leigh Syndrome
Interventions
Cysteamine Bitartrate
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 17 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
5
States / cities
San Diego, California • Stanford, California • Akron, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2024 · Synced Oct 2, 2026, 2:57 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene Mutation

NCT06706388
View directory record Official record
Conditions
Dentatorubral-Pallidoluysian Atrophy
Interventions
nL-ATN1-002
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
17 Years to 17 Years · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 9, 2026 · Synced Oct 2, 2026, 2:57 PM EDT
Terminated Phase 3 Interventional Results available

A Study to Assess the Usability of the Embrace Seizure Detection Watch in Children and Young Adults With Dravet Syndrome

NCT03299842
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 13, 2023 · Synced Oct 2, 2026, 2:57 PM EDT
Terminated Phase 2Phase 3 Interventional Results available

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

NCT04378075
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 31, 2026 · Synced Oct 2, 2026, 2:57 PM EDT
Recruiting No phase listed Observational

Biology of Juvenile Myoclonic Epilepsy

NCT03400371
View directory record Official record
Conditions
Juvenile Myoclonic Epilepsy
Interventions
Blood draw, Existing samples
Other
Lead sponsor
King's College London
Other
Eligibility
10 Years to 40 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
3
States / cities
New York, New York • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 5, 2025 · Synced Oct 2, 2026, 2:57 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

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

NCT04069689
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 2, 2026, 2:57 PM EDT
Active, not recruiting Phase 4 Interventional

Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution

NCT05044819
View directory record Official record
Conditions
Lennox Gastaut Syndrome, Dravet Syndrome, Tuberous Sclerosis Complex
Interventions
Cannabidiol
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
1 Year and older
Enrollment
154 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
26
States / cities
Little Rock, Arkansas • Downey, California • Long Beach, California + 22 more
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Oct 2, 2026, 2:57 PM EDT
Recruiting Phase 1Phase 2 Interventional

ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome

NCT07531745
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 2, 2026, 2:57 PM EDT
Completed Phase 2 Interventional Results available

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

NCT02091206
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 28, 2022 · Synced Oct 2, 2026, 2:57 PM EDT
Terminated Phase 2 Interventional Results available

A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease

NCT02473445
View directory record Official record
Conditions
Mitochondrial Diseases
Interventions
Cysteamine Bitartrate
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 17 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
5
States / cities
San Diego, California • Stanford, California • Akron, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2024 · Synced Oct 2, 2026, 2:57 PM EDT
Completed Phase 1Phase 2 Interventional

Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD).

NCT06051448
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 29, 2024 · Synced Oct 2, 2026, 2:57 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease

NCT06609889
View directory record Official record
Conditions
Lafora Disease
Interventions
ION283
Genetic
Lead sponsor
Berge Minassian
Other
Eligibility
10 Years to 18 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 18, 2025 · Synced Oct 2, 2026, 2:57 PM EDT