Clinical trials: CDKL5 Deficiency Disorder
CDKL5 Deficiency Disorder
Efficacy and Safety of Daily Home-based Hyperthermic Baths for Reducing Epileptic Seizures in CDKL5 Deficiency Disorder: A PROBE Clinical Trial
The primary objective of this research is to study the efficacy and safety of daily, home-based hyperthermic baths for reducing epileptic seizures in CDKL5 Deficiency Disorder.
- Trial name
- NCT07602205
- Testing
- Home-based hyperthermic Baths
- Volunteers
- 34 people
- Sponsor
- Xuanwu Hospital, Beijing
Cyclin-Dependent Kinase-Like 5 Deficiency Disorder
Observational Study in Patients With Cyclin-dependent Kinase-like 5 Deficiency Disorder
Observational, noninterventional, 3-year study to examine the presence of detectable differences in neurocognitive, developmental, motor, neurophysiologic, and quality of life measures over time in patients with cyclin-dependent…
- Trial name
- CANDID (NCT05373719)
- Testing
- No intervention; observational
- Where
- 23 sites in United States, Canada and 5 more
- Volunteers
- 112 people
- Sponsor
- Loulou Foundation
Epilepsy in Children, Dyskinesias
A Retrospective Survey-based Multicenter Study to Delineate the Molecular and Phenotypic Spectrum of Epilepsy-dyskinesia Syndromes
The Epilepsy-Dyskinesia Study aims to advance the understanding of the clinical and molecular spectrum of epilepsy-dyskinesia syndromes, monogenic diseases that cause both movement disorders and epilepsy.
- Conditions
- Epilepsy in Children, Dyskinesias, Movement Disorders in Children, Neurologic Disorder
- Trial name
- NCT06585605
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- Boston Children's Hospital
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
Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
This is a phase 2, crossover study of Ataluren for the treatment of nonsense mutation Dravet syndrome or cyclin-dependent kinase-like 5 (CDKL5) deficiency, resulting in drug-resistant epilepsy.
- Trial name
- NCT02758626
- Testing
- ataluren
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- NYU Langone Health
Dravet Syndrome, Lennox Gastaut Syndrome
Intermediate-Size Expanded Access Protocol (EAP) for LP352
This is an intermediate-size expanded access program (EAP) study. The purpose of this EAP is to provide continued access to LP352, an investigational drug product being investigated in participants with DEEs.
- Conditions
- Dravet Syndrome, Lennox Gastaut Syndrome, Developmental and Epileptic Encephalopathies
- Trial name
- NCT06149663
- Testing
- LP352
- Where
- 22 sites in United States, Australia
- Sponsor
- Longboard Pharmaceuticals