Clinical trials: Dravet Syndrome
Epilepsy, Dravet Syndrome
A Dose-ranging Pharmacokinetics and Safety Study of GWP42003-P in Children With Dravet Syndrome (GWPCARE1)
To evaluate the safety and pharmacokinetics (PK) of multiple doses of GWP42003-P compared with placebo in children with Dravet syndrome.
- Trial name
- NCT02091206
- Testing
- GWP42003-P 5 mg/kg/day Dose, GWP42003-P 10 mg/kg/day Dose, GWP42003-P 20 mg/kg/day Dose
- Where
- 11 sites in United States, United Kingdom
- Volunteers
- 34 people
- Sponsor
- Jazz Pharmaceuticals
Dravet Syndrome, Lennox Gastaut Syndrome
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
This is an open label study to evaluate the safety of ZX008 (fenfluramine) in patients with Dravet syndrome (DS) or Lennox Gastaut syndrome (LGS) who are being administered cannabidiol (CBD).
- Trial name
- NCT03467113
- Testing
- ZX008 0.2 to 0.8 mg/kg/day, Cannabidiol
- Where
- 2 sites in United States
- Volunteers
- 9 people
- Sponsor
- Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
CDKL5 Deficiency Disorder
Study of Adjunctive Ganaxolone Treatment in Children and Young Adults With CDKL5 Deficiency Disorder
A clinical study to evaluate the efficacy, safety, and tolerability of adjunctive ganaxolone therapy compared to placebo for the treatment of seizures in children and young adults with genetically confirmed CDKL5 gene mutation.
- Trial name
- Marigold (NCT03572933)
- Testing
- ganaxolone
- Where
- 36 sites in United States, Australia and 6 more
- Volunteers
- 101 people
- Sponsor
- Marinus Pharmaceuticals
Epilepsy, Dravet Syndrome
An Open Label Extension Study of Cannabidiol (GWP42003-P) in Children and Adults With Dravet or Lennox-Gastaut Syndromes
To investigate the potential antiepileptic effects of cannabidiol (GWP42003-P) in children and adults with Dravet or Lennox-Gastaut syndromes.
- Conditions
- Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- GWPCARE5 (NCT02224573)
- Testing
- GWP42003-P
- Volunteers
- 681 people
- Sponsor
- GW Research Ltd
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
Dravet Syndrome
Treatment Plan to Provide Expanded Access to Stiripentol for Patients With Dravet Syndrome
Expanded access to Stiripentol for patients with Dravet Syndrome.
- Trial name
- NCT01983722
- Testing
- Stiripentol
- Where
- United States
- Sponsor
- Children's Hospital Medical Center, Cincinnati
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
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
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
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
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
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
Rett Syndrome, MECP2 Duplication
Biobanking of Rett Syndrome and Related Disorders
The overarching purpose of this study is to advance understanding of the natural history of Rett syndrome (RTT), MECP2-duplication disorder (MECP2 Dup), RTT-related disorders including CDKL5, FOXG1, and individuals with MECP2…
- Conditions
- Rett Syndrome, MECP2 Duplication, CDKL5, FOXG1 Disorders
- Trial name
- NCT02705677
- Where
- 12 sites in United States
- Volunteers
- 752 people
- Sponsor
- University of Alabama at Birmingham
Dravet Syndrome
Stiripentol in Dravet Syndrome
The patient has failed all other available agents and has intractable epilepsy due to Dravet Syndrome. Stiripentol is highly efficacious in Dravet Syndrome.
- Trial name
- NCT01533506
- Testing
- stiripentol
- Where
- United States
- Sponsor
- Mayo Clinic
Dravet Syndrome
Cannabidiol Oral Solution as an Adjunctive Therapy for Treatment of Participants With Inadequately Controlled Dravet Syndrome
This Phase 3 study will enroll participants diagnosed with Dravet Syndrome (DS) who are still experiencing at least one tonic-clonic, clonic, and/or focal seizures with motor components (FSMC) per week, despite ongoing treatment…
- Trial name
- NCT02318563
- Testing
- Cannabidiol Oral Solution
- Sponsor
- INSYS Therapeutics Inc
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 (Australia Only)
WAYFINDER is a Phase 1/2 study in Australia to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged 6 to <84 months. The study follows an open-label, dose-escalation design.
- Trial name
- NCT06112275
- Testing
- ETX101
- Where
- Australia
- Volunteers
- 4 people
- Sponsor
- Encoded Therapeutics
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
An Open-Label Study to Investigate the Safety of Single and Multiple Ascending Doses in Children and Adolescents With Dravet Syndrome
Stoke Therapeutics is evaluating the safety and tolerability of single and multiple ascending doses of STK-001 in patients with Dravet syndrome.
- Trial name
- NCT04442295
- Testing
- STK-001 - Single Ascending Doses, STK-001 - Multiple Ascending Doses
- Where
- 18 sites in United States
- Volunteers
- 62 people
- Sponsor
- Stoke Therapeutics, Inc
15q Duplication Syndrome, CDKL5 Deficiency Disease
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)
The purpose of this study is to investigate the effect of soticlestat on the frequency of motor seizures for participants with Dup15q or CDD during the Maintenance Period.
- Trial name
- NCT03694275
- Testing
- Soticlestat
- Where
- 8 sites in United States
- Volunteers
- 20 people
- Sponsor
- Takeda
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