Clinical trials: CDKL5
Dravet Syndrome
A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome
The primary purpose of this study is evaluate the safety and tolerability of fenfluramine hydrochloride (HCl) 0.2 to 0.8 mg/kg/day in infants 1 year to less than 2 years of age with Dravet syndrome.
- Trial name
- ORCHID (NCT06118255)
- Testing
- fenfluramine
- Where
- 12 sites in United States, Belgium and 3 more
- Volunteers
- 25 people
- Sponsor
- UCB BIOSCIENCES, Inc.
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
Natural History Study of Infants and Children With Developmental and Epileptic Encephalopathies
This is a multicenter, prospective, 2-year observational study in infants and children with developmental and epileptic encephalopathies (DEEs). The DEE currently being investigated is SCN1A-positive Dravet Syndrome.
- Trial name
- ENVISION (NCT04537832)
- Testing
- No Intervention
- Where
- 16 sites in United States, Australia and 2 more
- Volunteers
- 58 people
- Sponsor
- Encoded Therapeutics
Dravet Syndrome, Lennox Gastaut Syndrome
A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome
This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome, Lennox-Gastaut syndrome or epileptic encephalopathy
- Conditions
- Dravet Syndrome, Lennox Gastaut Syndrome, Epileptic Encephalopathy
- Trial name
- NCT03936777
- Testing
- ZX008 (Fenfluramine Hydrochloride)
- Where
- 71 sites in United States, Australia and 12 more
- Volunteers
- 412 people
- Sponsor
- Zogenix, 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
Epilepsy
Open-Label PoC Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic Epilepsies
To evaluate the efficacy of open-label ganaxolone as adjunctive therapy for uncontrolled seizures in female children with PCDH19 mutation and other rare genetic epilepsies in an open-label proof-of-concept study.
- Trial name
- NCT02358538
- Testing
- Ganaxolone
- Where
- 10 sites in United States, Italy
- Volunteers
- 30 people
- Sponsor
- Marinus Pharmaceuticals
Dravet Syndrome
ZX008 Expanded Access Protocol
The treatment plan for this Expanded Access Protocol is for patients with Dravet syndrome who do not qualify for participation in one of the ongoing ZX008 clinical trials.
- Trial name
- NCT03780127
- Testing
- Fenfluramine Hydrochloride
- Sponsor
- Zogenix, Inc.
Dravet Syndrome
Safety and Tolerability of Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome
To investigate the long-term safety and tolerability of clobazam when administered for 1 year as adjunctive therapy in paediatric patients aged ≥1 to ≤16 years with Dravet Syndrome.
- Trial name
- NCT02187809
- Testing
- Clobazam
- Where
- 9 sites in United States, Mexico
- Volunteers
- 3 people
- Sponsor
- H. Lundbeck A/S
CDKL5 Deficiency Disorder
Double-blind, Randomized, Placebo-controlled Trial of Ganaxolone in CDKL5 Deficiency Patients 6 Months to Less Than 2 Years Old
This study will assess the efficacy, safety, and tolerability of ganaxolone (GNX) compared with placebo (PBO) as adjunctive therapy to the participant's standard anti-epileptic medication for the treatment of seizures in…
- Trial name
- NCT05249556
- Testing
- Ganaxolone
- Volunteers
- 20 people
- Sponsor
- Immedica Pharma AB
Developmental and Epileptic Encephalopathy, Dravet Syndrome
Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies
The objective of this study is to assess the safety, tolerability, efficacy, and pharmacokinetics of adjunctive therapy of LP352 in adults and adolescents with developmental and epileptic encephalopathies.
- Conditions
- Developmental and Epileptic Encephalopathy, Dravet Syndrome, Lennox Gastaut Syndrome
- Trial name
- PACIFIC (NCT05364021)
- Testing
- LP352
- Where
- 34 sites in United States, Australia
- Volunteers
- 52 people
- Sponsor
- Longboard Pharmaceuticals
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
Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome
Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures
This study will be conducted to evaluate the safety, pharmacokinetics (PK), and efficacy of adjunctive GWP42003-P in participants < 2 years of age with tuberous sclerosis complex (TSC), Lennox-Gastaut syndrome (LGS), or Dravet…
- Conditions
- Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome, Seizure in Participants With Lennox-Gastaut Syndrome
- Trial name
- NCT04485104
- Testing
- GWP42003-P
- Where
- 11 sites in United States, Italy and 1 more
- Volunteers
- 3 people
- Sponsor
- Jazz Pharmaceuticals
Dravet Syndrome (DS)
A Study of Soticlestat as an Add-on Therapy in Children and Young Adults With Dravet Syndrome
The main aim of the study is to learn if soticlestat, when given as an add-on therapy, reduces the number of convulsive seizures in children and young adults with DS.
- Trial name
- NCT04940624
- Testing
- Soticlestat
- Where
- 66 sites in United States, Australia and 16 more
- Volunteers
- 144 people
- Sponsor
- Takeda
Epilepsy, Dravet Syndrome
GWPCARE2 A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet Syndrome
To investigate the potential antiepileptic effects of cannabidiol (GWP42003-P) in children and young adults with Dravet syndrome.
- Trial name
- NCT02224703
- Testing
- GWP42003-P
- Where
- 38 sites in United States, Australia and 4 more
- Volunteers
- 199 people
- Sponsor
- Jazz Pharmaceuticals
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.
Developmental and Epileptic Encephalopathy, Dravet Syndrome
Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy
The objective of this study is to assess the long-term safety, tolerability, and efficacy of adjunctive therapy of LP352 in subjects with developmental and epileptic encephalopathies who completed participation in Study LP352-201.
- Conditions
- Developmental and Epileptic Encephalopathy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- NCT05626634
- Testing
- LP352, bexicaserin
- Where
- 28 sites in United States, Australia
- Volunteers
- 41 people
- Sponsor
- Longboard Pharmaceuticals
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 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
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
An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution in Children and Young Adults With Dravet Syndrome
This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome.
- Trial name
- NCT02823145
- Testing
- ZX008 (Fenfluramine Hydrochloride)
- Where
- 67 sites in United States, Australia and 10 more
- Volunteers
- 375 people
- Sponsor
- Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.