Clinical trials: CDKL5
CDKL5, CDKL5 Deficiency Disorder
International CDKL5 Clinical Research Network
Pathogenic variants in the Cyclin-dependent kinase like 5 (CDKL5) gene cause CDKL5 deficiency disorder (CDD, MIM 300672, 105830), a severe developmental and epileptic encephalopathy associated with cognitive and motor impairments…
- Conditions
- CDKL5, CDKL5 Deficiency Disorder, CDD
- Trial name
- ICCRN (NCT05558371)
- Testing
- No intervention.
- Where
- 9 sites in United States, Australia
- Volunteers
- 1,000 people
- Sponsor
- University of Colorado, Denver
Epilepsy
Cardiac Arrhythmias in Dravet Syndrome
SUMMARY Rationale: People with Dravet Syndrome (DS), a rare epilepsy syndrome, have a high risk of Sudden Unexpected Death in Epilepsy (SUDEP).
- Trial name
- NCT02415686
- Where
- 3 sites in Germany, Netherlands and 1 more
- Volunteers
- 59 people
- Sponsor
- Stichting Epilepsie Instellingen Nederland
Developmental and Epileptic Encephalopathy
A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathy (DEE)
This (DEEp OLE Study) is a multicentre, open-label study to investigate the long-term safety, efficacy, tolerability, and pharmacokinetics (PK) of LP352 in the treatment of seizures in children and adults with DEE who completed…
- Trial name
- NCT06908226
- Testing
- LP352
- Where
- 88 sites in United States, Australia and 15 more
- Volunteers
- 324 people
- Sponsor
- Longboard Pharmaceuticals
CDKL5 Deficiency Disorder
The Association Between Fever and CDKL5 Deficiency Disorder: a Nationwide Survey
The primary objective of this research is to study the effects of fever on seizure activity in patients with CDKL5 deficiency disorder
- Trial name
- NCT06663163
- Testing
- Observation
- Where
- China
- Volunteers
- 131 people
- Sponsor
- Xuanwu Hospital, Beijing
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
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
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, Seizure Disorder
A Trial of Two Fixed Doses of ZX008 (Fenfluramine HCl) in Children and Young Adults With Dravet Syndrome
Study 1 and Study 3 are the prospective, merged analyses of 2 identical double-blind, placebo-controlled studies, ZX008-1501 and ZX008-1502, to assess the efficacy, safety, and pharmacokinetics of ZX008 when used as adjunctive…
- Trial name
- NCT02682927
- Testing
- ZX008 (Fenfluramine Hydrochloride)
- Where
- 55 sites in United States, Australia and 9 more
- Volunteers
- 262 people
- Sponsor
- Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
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, 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, Severe Myoclonic Epilepsy of Infancy
Treatment of Gait Disorders in Children With Dravet Syndrome
Dravet syndrome is a severe infantile onset epilepsy syndrome with a prevalence of 1/15.000 to 1/30.000. An infant with an apparently normal development presents around 6 months of age with a convulsive status epilepticus.
- Trial name
- T-GaiD (NCT03857451)
- Testing
- Clinical gait analysis
- Where
- 3 sites in Belgium
- Volunteers
- 50 people
- Sponsor
- Universiteit Antwerpen
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
Dravet Syndrome
Neuronal Excitability of HCN1 Channel Mutations in Dravet Syndrome
This study addresses the changes in the axonal excitability parameters. It will compare these changes in patients with early infantile epileptic encephalopathy with HCN1 channel mutation and in control patients, with and without…
- Trial name
- EXCIDRAH (NCT02896608)
- Testing
- measure of neuronal excitability
- Where
- France
- Volunteers
- 92 people, healthy volunteers welcome
- Sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
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
Lennox-Gastaut Syndrome, Dravet Syndrome
The Effects of Cannabidiol (CBD) on Electrical and Autonomic Cardiac Function in Children With Severe Epilepsy
The investigators propose to study the effects of cannabidiol (CBD) on cardiac electrical function and the autonomic nervous system in children with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS), when the CBD is…
- Trial name
- CBD1 (NCT02815540)
- Testing
- 12-Lead ECG, Cannabidiol
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Gillette Children's Specialty Healthcare
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.
Dravet Syndrome
Genetic Analysis Between Charlotte's Web Responders Versus Non- Responders in a Dravet Population
There is tremendous curiosity about medical marijuana and the treatment of epilepsy. In a specific genetic epilepsy known as Dravet Syndrome, a mutation occurs affecting the SCN1A gene.
- Trial name
- NCT02229032
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- University of Colorado, Denver
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
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
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.