Clinical trials: Dravet Syndrome
Dravet Syndrome
Verapamil as Therapy for Children and Young Adults With Dravet Syndrome
This study will assess how well the drug verapamil can improve control of seizures and dysautonomia symptoms in children and young adults diagnosed with Dravet syndrome.
- Trial name
- NCT01607073
- Testing
- Verapamil
- Where
- 4 sites in United States
- Volunteers
- 2 people
- Sponsor
- Gillette Children's Specialty Healthcare
Epilepsies, Myoclonic, Drug Resistant Epilepsy
Extended Access Program With Lorcaserin For The Treatment of Dravet Syndrome and Other Refractory Epilepsies
The primary purpose of this study is to provide continued access of lorcaserin to participants with Dravet syndrome and other refractory epilepsies.
- Trial name
- NCT04457687
- Testing
- Lorcaserin
- Where
- 22 sites in United States, Canada
- Sponsor
- Eisai 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
CDKL5 Disorder
Ganaxolone Expanded Access Program Compassionate Use
The primary objective is to provide GNX to patients ≥ 2 years with CDD-related seizures who are refractory to, or intolerant of, standard therapy.
- Trial name
- CDD (NCT04678479)
- Testing
- Ganaxolone
- Sponsor
- Marinus Pharmaceuticals
Rett Syndrome, MECP2 Duplication dIsorder
Natural History of Rett Syndrome & Related Disorders
The purpose of this study is to advance understanding of the natural history of Rett syndrome (RTT), MECP2-duplication disorder (MECP2 Dup), CDKL5, FOXG1, and individuals with MECP2 mutations who do not have RTT including the…
- Conditions
- Rett Syndrome, MECP2 Duplication dIsorder, CDKL5 Disorder, FOXG1 Syndrome
- Trial name
- NCT02738281
- Where
- 14 sites in United States
- Volunteers
- 1,044 people
- Sponsor
- University of Alabama at Birmingham
Epilepsy-Dyskinesia, Epilepsy
Registry and Natural History of Epilepsy-Dyskinesia Syndromes
The Registry and Natural History of Epilepsy-Dyskinesia Syndromes is focused on gathering longitudinal clinical data as well as biological samples (blood, urine, and/or skin/tissue) from male and female patients, of all ages, who…
- Conditions
- Epilepsy-Dyskinesia, Epilepsy, Dyskinesia, EDS
- Trial name
- NCT06967727
- Where
- United States
- Volunteers
- 700 people
- Sponsor
- Boston Children's Hospital
Dravet Syndrome
A Study to Assess the Usability of the Embrace Seizure Detection Watch in Children and Young Adults With Dravet Syndrome
This is an exploratory sub-study to ZX008-1503 [NCT02823145]. Subjects will be fitted with an Embrace seizure detection watch and seizures detected by the watch will be compared to those entered into an electronic seizure diary.
- Trial name
- NCT03299842
- Testing
- ZX008 (Fenfluramine Hydrochloride)
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
Epileptic Encephalopathy
Cannabidiol in Children With Refractory Epileptic Encephalopathy
This study will assess the safety and tolerability of a cannabidiol-enriched Cannabis Herbal Extract in a small group of children with refractory epileptic encephalopathy.
- Trial name
- CARE-E (NCT03024827)
- Testing
- CanniMed® 1:20
- Where
- 4 sites in Canada
- Volunteers
- 20 people
- Sponsor
- University of Saskatchewan
Epilepsy, Dravet Syndrome
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
The purpose of this study is to investigate the effect on the frequency of all seizures (convulsive and drop) in participants treated with TAK-935 compared to placebo.
- Conditions
- Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- ELEKTRA (NCT03650452)
- Testing
- TAK-935
- Where
- 44 sites in United States, Australia and 6 more
- Volunteers
- 141 people
- Sponsor
- Takeda
Dravet Syndrome
Retrospective Non-interventional Study of Stiripentol Use in Dravet Patients in the USA
The present study aims to collect data regarding the history of the disease, previous and current treatments, and the clinical status of Dravet patients during the 3 months prior to stiripentol initiation, the first 3 months on…
- Trial name
- NCT05544058
- Testing
- Retrospective study
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Biocodex
CDKL5 Deficiency Disorder, Generalized Tonic Clonic Seizure
A Phase 3 Study to Examine the Efficacy and Safety of ZX008 in Subjects With CDKL5 Deficiency Disorder.
This is a Phase 3 Study to examine the efficacy and safety of ZX008 in children and adults with cyclin-dependent kinase like-5 (CDKL5) deficiency disorder (CDD).
- Conditions
- CDKL5 Deficiency Disorder, Generalized Tonic Clonic Seizure, Epileptic Spasm, Refractory Seizures
- Trial name
- GEMZ (NCT05064878)
- Testing
- Fenfluramine
- Where
- 46 sites in United States, Austria and 12 more
- Volunteers
- 87 people
- Sponsor
- Zogenix, Inc.
Lennox-Gastaut Syndrome (LGS), Dravet Syndrome (DS)
CBD Delivery with the A-Synaptic GT4 Transdermal Delivery System in with Dravet Syndrome And/or Lennox-Gastaut Syndrome
This study is a preliminary open-label, single-arm Phase II investigation into the safety and efficacy of transdermal cannabidiol (CBD) delivered using GT4 skin bream technology in individuals diagnosed with Dravet and/or…
- Trial name
- NCT06738732
- Testing
- CBD: GT4 Transdermal Delivery System
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Alexander Rotenberg
Dravet Syndrome
Compassionate Use of Stiripentol in Dravet Syndrome
Compassionate use of Stiripentol in Dravet Syndrome. This is a treatment protocol, not a research study, therefore children will only be monitored on a clinical basis for seizure improvement predominantly by parent and caregiver…
- Trial name
- NCT01835314
- Testing
- Stiripentol
- Where
- United States
- Sponsor
- University of Colorado, Denver
Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS)
A Study Evaluating Soticlestat in Participants With Dravet Syndrome or Lennox-Gastaut Syndrome Who Have Been Exposed to Fenfluramine
The purpose of this study is to check how soticlestat impacts symptoms of Dravet syndrome [DS] and Lennox-Gastaut syndrome [LGS] in participants who have been exposed to fenfluramine.
- Trial name
- NCT06422377
- Testing
- Soticlestat
- Where
- Denmark
- Volunteers
- 1 people
- Sponsor
- Takeda
Dravet Syndrome, Lennox Gastaut Syndrome
The Pharmacokinetics of Cannabidiol (CBD) and Its Effects in Children With Severe Epilepsy
The purpose of this study is to determine if cannabidiol (CBD) obtained via the state of Minnesota reduces seizures in patients with severe intractable epilepsy (Dravet Syndrome or Lennox Gastaut Syndrome), and to measure blood…
- Trial name
- NCT02910297
- Sponsor
- Gillette Children's Specialty Healthcare
Dravet Syndrome
GABA Biomarkers in Dravet Syndrome
This study will non-invasively obtain levels of GABA in the brain of children with SCN1A+DS and neurodeveloping children through evoked and induced cortical responses, correlate them with the BOLD responses, and with the levels…
- Trial name
- NCT05651204
- Testing
- GABA Blood Level
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Cook Children's Health Care System
CDKL5 Deficiency Disorder (CDD)
International CDKL5 Registry
Owing to the recent classification of CDKL5 Deficiency Disorder (CDD) as a unique disorder, there is a limited understanding of overall disease natural history and meaningful outcome measures.
- Trial name
- NCT04486768
- Testing
- Survey Instrument
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- University of Pennsylvania
CDKL5
Targeting the Gut to Improve Seizure Control in CDKL5 Deficiency Disorder (CDD)
Standard anti-seizure medications have limited efficacy in seizure control in cyclin-dependent kinase-like 5 deficiency disorder (CDD).
- Trial name
- NUTRIENT (NCT06448663)
- Testing
- alpha-lactalbumin, fructooligosaccharides, inulin, alpha-lactalbumin, sodium butyrate, fructooligosaccharides, inulin
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- University of Milan
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, Epilepsy
Bioavailability of Stiripentol After Single Oral Dose of Capsule vs Suspension in Healthy Subjects (STILIQ)
This is a Phase I, open-label, randomized, single-center, two-way cross-over study evaluating the relative bioavailability, pharmacokinetics, safety, and palatability of two formulations of stiripentol (Diacomit®), indicated in…
- Conditions
- Dravet Syndrome, Epilepsy, Childhood, Epileptic Encephalopathy
- Trial name
- STILIQ (NCT07176832)
- Testing
- Stiripentol capsule (Diacomit®), Stiripentol oral suspension (Diacomit®)
- Where
- 2 sites in France
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Biocodex