Clinical trials: Epileptic Encephalopathy
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
Inherited Mitochondrial Disease, Including Leigh Syndrome
Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered at a target maintenance dose of 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in…
- Trial name
- MITO-001 (NCT02023866)
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 36 people
- Sponsor
- Amgen
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
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
Developmental and Epileptic Encephalopathy (DEE)
A Study to Evaluate the Safety and Efficacy of CBD-OS in Participants With DEE
The efficacy, safety, and tolerability of CBD-OS have been evaluated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and Tuberous sclerosis complex (TSC).
- Trial name
- NCT07723976
- Testing
- CBD-OS
- Volunteers
- 120 people
- Sponsor
- Jazz Pharmaceuticals
Epileptic Encephalopathy, SCN2A Encephalopathy
A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
A Multi-Center, Single-Arm Clinical Trial to Investigate the Efficacy and Safety of Elsunersen in Pediatric Participants with Early Onset SCN2A Developmental and Epileptic Encephalopathy
- Trial name
- NCT07019922
- Testing
- 1mg elsunersen, 0.5mg elsunersen
- Where
- 10 sites in United States, Brazil and 2 more
- Volunteers
- 40 people
- Sponsor
- Praxis Precision Medicines
Dravet Syndrome
A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)
This (DEEp SEA Study) is a double-blind, randomized, placebo-controlled, multicenter study to investigate the efficacy, safety, and tolerability of LP352 in the treatment of seizures in children and adults with DS.
- Trial name
- NCT06660394
- Testing
- LP352
- Where
- 105 sites in United States, Australia and 16 more
- Volunteers
- 108 people
- Sponsor
- Longboard Pharmaceuticals
Developmental and Epileptic Encephalopathy
A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathies (DEE)
This (DEEp OCEAN Study) is a double-blind, randomized, placebo-controlled, multicenter study to investigate the efficacy, safety, and tolerability of LP352 in the treatment of seizures in children and adults with DEE.
- Trial name
- NCT06719141
- Testing
- LP352
- Where
- 116 sites in United States, Australia and 15 more
- Volunteers
- 367 people
- Sponsor
- Longboard Pharmaceuticals
SCN2A-DEE, Epilepsy
A Clinical Trial of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy
The goal of this trial is to learn about the effect of PRAX-222 in pediatric participants with early onset SCN2A developmental and epileptic encephalopathy (DEE), aged 2 to 18 years.
- Trial name
- NCT05737784
- Testing
- PRAX-222 - Initial Dose, PRAX-222 - Initial Ascending Doses, PRAX-222 - Optional Ascending Doses
- Where
- 3 sites in United States, Brazil
- Volunteers
- 60 people
- Sponsor
- Praxis Precision Medicines
Developmental and/or Epileptic Encephalopathies
Study of TAK-935 as an Adjunctive Therapy in Participants With Developmental and/or Epileptic Encephalopathies
The purpose of this study is to characterize the multiple-dose safety and tolerability profile of TAK-935 in adult participants with developmental and/or epileptic encephalopathies.
- Trial name
- NCT03166215
- Testing
- TAK-935
- Where
- 11 sites in United States
- Volunteers
- 18 people
- Sponsor
- Takeda
Lennox-Gastaut Syndrome
Post-marketing Surveillance of Long-term Administration of Inovelon Tablets in Patients With Lennox-Gastaut Syndrome
This surveillance's objectives are 1. Unknown adverse reactions 2. Incidences of adverse drug reaction 3. Efficacy during long-term administration 4. Factors considered to have effect to safety and effectiveness 5.
- Trial name
- NCT02175173
- Testing
- Rufinamide
- Where
- 2 sites in Japan
- Volunteers
- 707 people
- Sponsor
- Eisai Co., Ltd.
Epileptic Encephalopathy
Prospective Cohort Study on the Efficacy of Vagus Nerve Stimulation for Children and Adolescents With Epilepsy(PVNS-CAE)
Prospective cohort study on the long-term effectiveness and safety of PINS vagus nerve stimulators for children and adolescent with refractory epilepsy.
- Trial name
- NCT02603991
- Testing
- PINS Vagus Nerve Stimulator, Levetiracetam
- Where
- China
- Volunteers
- 1 people
- Sponsor
- Beijing Pins Medical Co., Ltd
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
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, 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 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.
SLC6A1 Neurodevelopmental Disorder (NDD), Developmental and Epileptic Encephalopathies (DEE)
SLC6A1-NDD Prospective Longitudinal Natural History Study
The overall objective of this prospective longitudinal natural history study is to collect clinical data to characterize and evaluate the natural course of SLC6A1-NDD and assess the feasibility of certain assessments for the…
- Trial name
- SPIRIT (NCT07531511)
- Where
- 3 sites in United States, France and 1 more
- Volunteers
- 60 people
- Sponsor
- UCB Biopharma SRL
Epilepsy, Epilepsy in Children
An Open-Label Extension of the Study XEN496 (Ezogabine) in Children With KCNQ2-DEE
To assess the long-term safety and tolerability of XEN496 in pediatric subjects with KCNQ2 developmental and epileptic encephalopathy (KCNQ2-DEE) who had participated in the primary study (XPF-009-301).
- Conditions
- Epilepsy, Epilepsy in Children, Epilepsy; Seizure, Disease
- Trial name
- EPIK-OLE (NCT04912856)
- Testing
- XEN496
- Where
- 4 sites in United States, Australia and 1 more
- Volunteers
- 8 people
- Sponsor
- Xenon Pharmaceuticals Inc.
Epileptic Encephalopathy, Continuous Spike and Wave During Sleep
Extension Study to Evaluate NBI-827104 in Pediatric Participants With Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep
The primary objective for this study is to evaluate the long-term safety and tolerability of NBI-827104 in pediatric participants with epileptic encephalopathy with continuous spike-and-wave during sleep (EECSWS).
- Trial name
- Steamboat 2 (NCT05301894)
- Testing
- NBI-827104
- Where
- 12 sites in United States, Denmark and 3 more
- Volunteers
- 19 people
- Sponsor
- Neurocrine Biosciences
SCN8A Developmental and Epileptic Encephalopathy Syndrome
Extension Study to Evaluate How Safe and Tolerable NBI-921352 is as an Adjunctive Therapy for Participants With SCN8A-DEE
Extension study to evaluate how safe and tolerable the drug NBI-921352 is when used as adjunctive therapy in participants with SCN8A developmental and epileptic encephalopathy syndrome (SCN8A-DEE).
- Trial name
- NCT05226780
- Testing
- NBI-921352
- Where
- 4 sites in United States
- Volunteers
- 8 people
- Sponsor
- Neurocrine Biosciences