Clinical trials: Dravet Syndrome, active
Dravet Syndrome
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
ENDEAVOR is a Phase 1/2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to <36 months (Part 1A), aged ≥48 months to <18 years (Part 1B), and…
- Trial name
- ENDEAVOR (NCT05419492)
- Testing
- ETX101
- Where
- 14 sites in United States, Australia and 1 more
- Volunteers
- 113 people
- Sponsor
- Encoded Therapeutics
Dravet Syndrome
A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome
The purpose of the study is to evaluate the efficacy, safety, and tolerability of zorevunersen in Patients with Dravet syndrome.
- Trial name
- NCT06872125
- Testing
- zorevunersen
- Where
- 61 sites in United States, China and 6 more
- Volunteers
- 170 people
- Sponsor
- Stoke Therapeutics, Inc
Dravet Syndrome (DS), Lennox Gastaut Syndrome (LGS)
Clinical Utility of Reduced EEG Home Monitoring in Fenfluramine Titration for Dravet and LGS
The goal of this real world data study is to evaluate the clinical utility of remote patient monitoring solution (using Byteflies' EpiCare@Home with reduced EEG montage and other vital signs) with Dravet and LGS patients (ages 3…
- Trial name
- TETRIS (NCT07225231)
- Testing
- Byteflies Remote patient monitoring solution (with reduced EEG)
- Where
- 3 sites in United States
- Volunteers
- 20 people
- Sponsor
- Byteflies
Dravet Syndrome
A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)
This (DEEp SEA Study) is a double-blind, randomized, placebo-controlled, multicenter study to investigate the efficacy, safety, and tolerability of LP352 in the treatment of seizures in children and adults with DS.
- Trial name
- NCT06660394
- Testing
- LP352
- Where
- 105 sites in United States, Australia and 16 more
- Volunteers
- 96 people
- Sponsor
- Longboard Pharmaceuticals
Dravet Syndrome
A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome
This is a multicenter, Phase 3, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of clemizole hydrochloride (EPX-100) as adjunctive therapy in children and adult participants with…
- Trial name
- ARGUS (NCT04462770)
- Testing
- Clemizole HCl
- Where
- 68 sites in United States, Argentina and 8 more
- Volunteers
- 150 people
- Sponsor
- Epygenix
Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS)
A Study to Investigate the Transition of Children From 'Artisanal" Cannabidiol (CBD) to Epidiolex
The goal of this clinical trial is to learn the best way to switch children with Lennox-Gastaut Syndrome (LGS) or Dravet Syndrome (DS) taking 'artisanal' (non pharmaceutical-grade) cannabidiol (CBD) to Epidiolex for treatment of…
- Trial name
- CANN-SWITCH (NCT06924827)
- Testing
- Epidiolex 100 mg/mL Oral Solution
- Where
- Canada
- Volunteers
- 25 people
- Sponsor
- Elizabeth Donner
Dravet Syndrome
ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
The primary purpose of this study is to evaluate the safety and tolerability of ION337 in participants with Dravet syndrome (DS).
- Trial name
- NCT07531745
- Testing
- ION337
- Where
- 6 sites in United States, Canada
- Volunteers
- 32 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Dravet Syndrome (DS)
Biomarkers of Neurodegeneration, Synaptic Plasticity and Neuroinflammation in Dravet Syndrome
Dravet syndrome (DS) is a developmental and epileptic encephalopathy, usually caused by de novo pathogenic SCN1A variants, characterized by early-onset prolonged febrile seizures, subsequent drug-resistant polymorphic epilepsy…
- Trial name
- TEMBO-DS (NCT07801404)
- Where
- 9 sites in Italy
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Lennox Gastaut Syndrome, Dravet Syndrome
Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution
This study will monitor for potential chronic liver injury and liver fibrosis, in participants treated with cannabidiol oral solution.
- Conditions
- Lennox Gastaut Syndrome, Dravet Syndrome, Tuberous Sclerosis Complex
- Trial name
- NCT05044819
- Testing
- Cannabidiol
- Where
- 26 sites in United States
- Volunteers
- 154 people
- Sponsor
- Jazz Pharmaceuticals
Dravet Syndrome
The FINTEPLA as an Anti-SUDEP Therapy in Dravet Syndrome Project
This study investigates cerebrovascular reactivity (CVR) and functional brain connectivity in Dravet Syndrome (DS) patients with convulsive seizures.
- Trial name
- FAST-DS (NCT07112365)
- Testing
- Fenfluramine treatment (Fintepla), Hypercapnia Challenge using the device (RespirAct)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- The University of Texas Health Science Center, Houston
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Dravet Syndrome (DS)
A Study to Evaluate the Safety and Pharmacokinetics of RC001 in Children With Dravet Syndrome
This is an open-label, single-center study to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of intrathecal RC001 in patients with Dravet syndrome aged 2 to 18 years.
- Trial name
- NCT07675746
- Testing
- RC001 injection-Dose Escalation Cohort, RC001 injection-Fixed Dose Cohort
- Where
- China
- Volunteers
- 8 people
- Sponsor
- Second Affiliated Hospital of Guangzhou Medical University
Dravet Syndrome
A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome
EXPEDITION is a Phase 1/2 study in the UK to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet Syndrome aged 6 to < 48 months. The study follows and open-label, dose-escalation design.
- Trial name
- UK Only (NCT06283212)
- Testing
- ETX101
- Where
- 3 sites in United Kingdom
- Volunteers
- 5 people
- Sponsor
- Encoded Therapeutics
Lennox Gastaut Syndrome, Dravet Syndrome
Epidyolex® in Lennox Gastaut, Dravet Syndrome and Tuberous Sclerosis Complex: an Observational Study in ITALY
This is a prospective, observational study on approximately 70-100 Real World participants affected by LGS, DS, or TSC treated with Epidyolex® as prescribed in the summary of product characteristics.
- Conditions
- Lennox Gastaut Syndrome, Dravet Syndrome, Tuberous Sclerosis Complex
- Trial name
- EpiTALY (NCT05485831)
- Testing
- Epidiolex 100 mg/mL Oral Solution
- Where
- 18 sites in Italy
- Volunteers
- 111 people
- Sponsor
- Jazz Pharmaceuticals
Dravet Syndrome (DS), Children Under 2 Years
Assessment of Safety of the Use of Fenfluramine in Children With Dravet Syndrome Under 24 Months of Age
Dravet syndrome is a genetic epilepsy associated with pathogenic variants in SCN1A that codes for Nav1.1, a protein necessary for sodium channels.
- Trial name
- NCT06598449
- Testing
- fenfluramine
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- University of Colorado, Denver
Epilepsy, Dravet Syndrome
A PET-MRI Study of Serotoninergic Brainstem Pathway in Patients With Dravet Syndrome
Dravet Syndrome (DS) is a severe neurodevelopmental disease, which is predominantly caused by mutations of SCN1A, the gene coding for Nav1.1 voltage-gated sodium channels.
- Conditions
- Epilepsy, Dravet Syndrome, Drug Resistant Epilepsy, Healthy Controls
- Trial name
- DRAPETONINE (NCT07013331)
- Testing
- [18F]MPPF PET-MRI
- Where
- France
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Hospices Civils de Lyon
Epilepsy, West Syndrome
Multicentre Real-life Follow-up Study of Rare Epileptic Syndromes in Children and Adolescents
Rare epilepsies as a whole account for 20-30% of epilepsies, but knowledge about prognostic factors is currently limited. This means that it is difficult to provide adequate information to families at diagnosis and during…
- Conditions
- Epilepsy, West Syndrome, Dravet Syndrome
- Trial name
- EPIRARE (NCT05126914)
- Where
- 11 sites in France
- Volunteers
- 1,000 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Dravet Syndrome
An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome
Stoke Therapeutics is evaluating the long-term safety \& tolerability of repeated doses of zorevunersen (STK-001) in patients with Dravet syndrome who previously participated in studies of zorevunersen.
- Trial name
- NCT04740476
- Testing
- zorevunersen (STK-001)
- Where
- 17 sites in United States
- Volunteers
- 60 people
- Sponsor
- Stoke Therapeutics, Inc
Dravet Syndrome
Longitudinal Study of Phenotypic and Developmental Severity in Patients With Dravet Syndrome With SCN1A Gene Mutation
Dravet syndrome with SCN1A gene mutation is a developmental and epileptic encephalopathy characterized by treatment-resistant epilepsy and global developmental delay.
- Trial name
- LONG-DS (NCT07251673)
- Where
- France
- Volunteers
- 50 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Dravet Syndrome, Epilepsy
Neurodevelopmental Impact of Epilepsy on Autonomic Function in Dravet Syndrome
Dravet Syndrome (DS) is a severe epileptic encephalopathy, which main cause is mutations of SCN1A, the gene coding for the Nav1.1 voltage-gated sodium channel.
- Trial name
- AUTONOMIC (NCT05472389)
- Testing
- Video-electroencephalography, Blood Samples
- Where
- 4 sites in Belgium, France and 1 more
- Volunteers
- 92 people
- Sponsor
- Hospices Civils de Lyon