Clinical trials: CDKL5 Disorder
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, 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 (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
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
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, Lennox Gastaut Syndrome
Research on Cognitive Effect of Cannabidiol on Dravet Syndrome and Lennox-Gastaut SyndromeGastaut Syndrome
The clinical trial "A Prospective Single-Center Single-Arm Clinical Trial on Cognitive Effect of Cannabidiol (CBD-OS®) on Dravet syndrome and Lennox-Gastaut Syndrome" is a single-group phase III study done in single tertiary…
- Trial name
- NCT04611438
- Testing
- Cannabidiol
- Where
- South Korea
- Volunteers
- 107 people
- Sponsor
- Yonsei University
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
Dravet Syndrome (DS)
A Study to Evaluate the Safety and Pharmacokinetics of RC001 in Children With Dravet Syndrome
This is an open-label, single-center study to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of intrathecal RC001 in patients with Dravet syndrome aged 2 to 18 years.
- Trial name
- NCT07675746
- Testing
- RC001 injection-Dose Escalation Cohort, RC001 injection-Fixed Dose Cohort
- Where
- China
- Volunteers
- 8 people
- Sponsor
- Second Affiliated Hospital of Guangzhou Medical University
Epilepsy, Dravet Syndrome
A PET-MRI Study of Serotoninergic Brainstem Pathway in Patients With Dravet Syndrome
Dravet Syndrome (DS) is a severe neurodevelopmental disease, which is predominantly caused by mutations of SCN1A, the gene coding for Nav1.1 voltage-gated sodium channels.
- Conditions
- Epilepsy, Dravet Syndrome, Drug Resistant Epilepsy, Healthy Controls
- Trial name
- DRAPETONINE (NCT07013331)
- Testing
- [18F]MPPF PET-MRI
- Where
- France
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Hospices Civils de Lyon
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
Dravet Syndrome
A 2-Part Study to Investigate the Dose-Ranging Safety and Pharmacokinetics, Followed by the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy in Children ≥ 2 Years Old and Young Adults With Dravet Syndrome
The primary purpose of this study is to evaluate the safety, tolerability, and efficacy of ZX008 (fenfluramine hydrochloride) when added to adjunctive antiepileptic stiripentol treatment in children and young adults with Dravet…
- Trial name
- NCT02926898
- Testing
- ZX008 (Fenfluramine Hydrochloride)
- Where
- 28 sites in United States, Canada and 5 more
- Volunteers
- 87 people
- Sponsor
- Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
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.
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
Dravet Syndrome
Transcranial Magnetic Stimulation to Measure Cortical Excitability in Dravet Syndrome
Dravet syndrome (DS) is an epileptic encephalopathy caused by pathogenic variants in the SCN1A gene resulting in medically refractory epilepsy and psychomotor delays.
- Trial name
- NCT04614506
- Testing
- Transcranial Magnetic Stimulation (TMS), Electroencephalogram (EEG)
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Boston Children's Hospital
Dravet Syndrome
Study of Safety and Pharmacokinetics of Oral Doses of EPX-100 in Healthy Subjects.
This is a placebo-controlled, double-blind, 2-period study in 3 sequential groups of 8 healthy subjects each. The safety and pharmacokinetics of escalating single and multiple oral doses of EPX-100 will be assessed in fasting…
- Trial name
- NCT04069689
- Testing
- EPX-100 (Clemizole Hydrochloride)
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Epygenix
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
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.
Dravet Syndrome
A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome
This is a multicenter, Phase 3, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of clemizole hydrochloride (EPX-100) as adjunctive therapy in children and adult participants with…
- Trial name
- ARGUS (NCT04462770)
- Testing
- Clemizole HCl
- Where
- 68 sites in United States, Argentina and 8 more
- Volunteers
- 150 people
- Sponsor
- Epygenix
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