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ClinicalTrials.gov public records Last synced Oct 26, 2026, 6:21 PM EDT

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Showing 1–24 of 67 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Dravet Syndrome Clear all
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 26, 2026, 6:21 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 26, 2026, 6:21 PM 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 14, 2023 · Synced Oct 26, 2026, 6:21 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 26, 2026, 6:21 PM EDT
Completed No phase listed Observational

Natural History of Rett Syndrome & Related Disorders

NCT02738281
View directory record Official record
Conditions
Rett Syndrome, MECP2 Duplication dIsorder, CDKL5 Disorder, FOXG1 Syndrome
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
Not listed
Enrollment
1,044 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
14
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 11 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2021 · Synced Oct 26, 2026, 6:21 PM EDT
Active, not recruiting Phase 3 Interventional

A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome

NCT06872125
View directory record Official record
Conditions
Dravet Syndrome
Interventions
zorevunersen, Sham Comparator
Drug · Other
Lead sponsor
Stoke Therapeutics, Inc
Industry
Eligibility
2 Years to 17 Years
Enrollment
170 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
28
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 23 more
Source: ClinicalTrials.gov public record
Updated Oct 20, 2026 · Synced Oct 26, 2026, 6:21 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 26, 2026, 6:21 PM 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 14, 2026 · Synced Oct 26, 2026, 6:21 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 26, 2026, 6:21 PM EDT
Completed No phase listed Observational

Biobanking of Rett Syndrome and Related Disorders

NCT02705677
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 5, 2021 · Synced Oct 26, 2026, 6:21 PM EDT
No Longer Available No phase listed Expanded access

Stiripentol in Dravet Syndrome

NCT01533506
View directory record Official record
Conditions
Dravet Syndrome
Interventions
stiripentol
Drug
Lead sponsor
Mayo Clinic
Other
Eligibility
11 Years and older · Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 28, 2013 · Synced Oct 26, 2026, 6:21 PM EDT
No Longer Available No phase listed Expanded access

Extended Access Program With Lorcaserin For The Treatment of Dravet Syndrome and Other Refractory Epilepsies

NCT04457687
View directory record Official record
Conditions
Epilepsies, Myoclonic, Drug Resistant Epilepsy
Interventions
Lorcaserin
Drug
Lead sponsor
Eisai Inc.
Industry
Eligibility
2 Years and older
U.S. locations
18
States / cities
Birmingham, Alabama • Los Angeles, California • Aurora, Colorado + 14 more
Source: ClinicalTrials.gov public record
Updated Oct 19, 2026 · Synced Oct 26, 2026, 6:21 PM EDT
Completed Phase 1Phase 2 Interventional

An Open-Label Study to Investigate the Safety of Single and Multiple Ascending Doses in Children and Adolescents With Dravet Syndrome

NCT04442295
View directory record Official record
Conditions
Dravet Syndrome
Interventions
STK-001 - Single Ascending Doses, STK-001 - Multiple Ascending Doses
Drug
Lead sponsor
Stoke Therapeutics, Inc
Industry
Eligibility
2 Years to 18 Years
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
18
States / cities
San Francisco, California • Aurora, Colorado • Washington D.C., District of Columbia + 15 more
Source: ClinicalTrials.gov public record
Updated May 18, 2025 · Synced Oct 26, 2026, 6:21 PM EDT
Completed Phase 2 Interventional Results available

A Multicenter, Open-label, Pilot Study of Soticlestat (TAK-935/OV935) in Participants With 15Q Duplication Syndrome (Dup 15q) or Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder (ARCADE STUDY)

NCT03694275
View directory record Official record
Conditions
15q Duplication Syndrome, CDKL5 Deficiency Disease
Interventions
Soticlestat
Drug
Lead sponsor
Takeda
Industry
Eligibility
2 Years to 55 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
8
States / cities
Los Angeles, California • Aurora, Colorado • Norcross, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 10, 2026 · Synced Oct 26, 2026, 6:21 PM EDT
Completed No phase listed Observational

Genetic and Physical Characteristics of Rett Syndrome

NCT00299312
View directory record Official record
Conditions
Rett Syndrome, MECP2 Duplication Disorder, Rett-related Disorder
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
11
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 16, 2017 · Synced Oct 26, 2026, 6:21 PM 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 4, 2026 · Synced Oct 26, 2026, 6:21 PM 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 24, 2015 · Synced Oct 26, 2026, 6:21 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 26, 2026, 6:21 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 26, 2026, 6:21 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 26, 2026, 6:21 PM EDT
No Longer Available No phase listed Expanded access

Expanded Access Use of Stiripentol in Dravet Syndrome or Sodium Channel Mutation Epileptic Encephalopathies

NCT02239276
View directory record Official record
Conditions
Dravet Syndrome, Epileptic Encephalopathies Associated With SCN1A Mutations
Interventions
Stiripentol
Drug
Lead sponsor
Cook Children's Health Care System
Other
Eligibility
6 Months to 18 Years
U.S. locations
1
States / cities
Fort Worth, Texas
Source: ClinicalTrials.gov public record
Updated Feb 5, 2020 · Synced Oct 26, 2026, 6:21 PM EDT