Clinical trials: Generalized Epilepsy, open to volunteers
Absence Epilepsy, Ketogenic Dieting
Ketogenic Diet for New-Onset Absence Epilepsy
The ketogenic diet is a medical therapy for epilepsy that is used nearly predominantly for refractory epilepsy (after 2-3 drugs have been tried and failed).
- Conditions
- Absence Epilepsy, Ketogenic Dieting, Epilepsy, Absence
- Trial name
- NCT04274179
- Testing
- Modified Atkins Diet, Absence epilepsy medications
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Johns Hopkins University
Lafora Disease
A Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease
This study will test the safety and efficacy of multiple doses of ION283 administered as intrathecal (IT) injections by lumbar puncture (LP). All subjects will receive ION283.
- Trial name
- Lafora (NCT06609889)
- Testing
- ION283
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Berge Minassian
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
- 86 sites in United States, Argentina and 9 more
- Volunteers
- 150 people
- Sponsor
- Epygenix
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, Quality of Life
Idiopathic Generalized Epilepsy Cognitive and Emotional Profile
The primary goal of the study is to explore the neurocognitive, emotional-behavioral functioning, and quality of life of adolescents with IGE, identifying key factors that affect their overall well-being.
- Conditions
- Epilepsy, Quality of Life, Neuropathology, Emotional Problem
- Trial name
- NCT06782074
- Testing
- emotional and neuropsychological assessment
- Where
- Italy
- Volunteers
- 96 people
- Sponsor
- IRCCS National Neurological Institute "C. Mondino" Foundation
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
Progressive Myoclonus Epilepsy Type 1, EPM1
Registry and Natural History Study for Progressive Myoclonus Epilepsy Type 1 (EPM1)
The Registry and Natural History Study for Progressive Myoclonus Epilepsy Type 1 (EPM1) is focused on gathering longitudinal clinical data as well as biological samples (blood and/or urine) from male and female patients, of all…
- Conditions
- Progressive Myoclonus Epilepsy Type 1, EPM1, CSTB-related Disease, Myoclonus Epilepsies, Progressive
- Trial name
- EPM1 (NCT06593951)
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Boston Children's Hospital
Epilepsy, Seizure Disorder
Epilepsy Learning Healthcare System (ELHS)
The Epilepsy Learning Health System (ELHS) is a quality improvement and research network to improve outcomes for people with epilepsy.
- Conditions
- Epilepsy, Seizure Disorder, Neurologic Disorder, Rare Diseases
- Trial name
- ELHS (NCT06265103)
- Testing
- Clinical care and quality improvement
- Where
- 13 sites in United States
- Volunteers
- 100,000 people
- Sponsor
- Epilepsy Foundation of America
Infantile Spasm
Characterization by Automated System on Infantile Spasmes
Infantile spasms are defined by the occurrence of epileptic episodes characterized by the appearance of very specific motor seizures, made up of rapid, repeated contractions in flexion or extension known as spasms.
- Trial name
- REASSESS (NCT06266234)
- Where
- France
- Volunteers
- 5,000 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Lennox-Gastaut Syndrome (LGS), Dravet Syndrome (DS)
CBD Delivery with the A-Synaptic GT4 Transdermal Delivery System in with Dravet Syndrome And/or Lennox-Gastaut Syndrome
This study is a preliminary open-label, single-arm Phase II investigation into the safety and efficacy of transdermal cannabidiol (CBD) delivered using GT4 skin bream technology in individuals diagnosed with Dravet and/or…
- Trial name
- NCT06738732
- Testing
- CBD: GT4 Transdermal Delivery System
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Alexander Rotenberg
PolG, Primary Mitochondrial Myopathy
Prospective Natural History Study of POLG Disease
The PIONEER study is a prospective, natural history study dedicated to characterizing the clinical progression of POLG-related disorders.
- Trial name
- PIONEER (NCT07775872)
- Where
- 21 sites in United States, Australia and 7 more
- Volunteers
- 300 people
- Sponsor
- The POLG Foundation
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
Dravet Syndrome
GABA Biomarkers in Dravet Syndrome
This study will non-invasively obtain levels of GABA in the brain of children with SCN1A+DS and neurodeveloping children through evoked and induced cortical responses, correlate them with the BOLD responses, and with the levels…
- Trial name
- NCT05651204
- Testing
- GABA Blood Level
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Cook Children's Health Care System
Epilepsy, West Syndrome
Multicentre Real-life Follow-up Study of Rare Epileptic Syndromes in Children and Adolescents
Rare epilepsies as a whole account for 20-30% of epilepsies, but knowledge about prognostic factors is currently limited. This means that it is difficult to provide adequate information to families at diagnosis and during…
- Conditions
- Epilepsy, West Syndrome, Dravet Syndrome
- Trial name
- EPIRARE (NCT05126914)
- Where
- 11 sites in France
- Volunteers
- 1,000 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
SLC6A1
A Fully Remote Feasibility Trial of Phenylbutyrate for SLC6A1-Related Disorders
The purpose of this study is to evaluate the feasibility of a site-less (fully remote) clinical trial using phenylbutyrate for SLC6A1-related disorders.
- Trial name
- NCT07847918
- Testing
- Glycerol Phenylbutyrate
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Scripps Translational Science Institute
Epilepsy, Epilepsy, Idiopathic Generalized
Idiopathic Generalized Epilepsy Syndromes
The idiopathic generalized epilepsies (IGEs) have historically included the syndromes childhood absence epilepsy (CAE), juvenile absence epilepsy (JAE), juvenile myoclonic epilepsy (JME), and epilepsy with generalized…
- Trial name
- NCT06388174
- Where
- China
- Volunteers
- 200 people
- Sponsor
- Shen Chun-Hong
Status Epilepticus
Exogenous Ketone Esters for Refractory Status Epileptics
This study aims to investigate the efficacy of add-on exogenous ketone esters for the treatment of children with refractory generalized convulsive status epilepticus
- Trial name
- EKERSE (NCT05674552)
- Testing
- Exogenous ketone ester
- Where
- Egypt
- Volunteers
- 50 people
- Sponsor
- Sohag University
Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis
National Registry of Rare Kidney Diseases
The goal of this National Registry is to is to collect information from patients with rare kidney diseases, so that it that can be used for research.
- Conditions
- Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis, AHL Amyloidosis, AL Amyloidosis
- Trial name
- RaDaR (NCT06065852)
- Where
- United Kingdom
- Volunteers
- 35,000 people
- Sponsor
- UK Kidney Association
Mitochondrial Disorders, Mitochondrial Genetic Disorders
North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
The North American Mitochondrial Disease Consortium (NAMDC) maintains a patient contact registry and tissue biorepository for patients with mitochondrial disorders.
- Conditions
- Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes
- Trial name
- NAMDC (NCT01694940)
- Where
- 17 sites in United States, Canada
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Columbia University
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