Clinical trials: Developmental and Epileptic Encephalopathy
Developmental and Epileptic Encephalopathy
A Clinical Trial of CAP-002 Gene Therapy in Pediatric Patients With Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy
The goal of this clinical trial is to learn about the safety of CAP-002 gene therapy in children with Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy.
- Trial name
- NCT06983158
- Testing
- gene therapy
- Where
- 4 sites in United States
- Volunteers
- 1 people
- Sponsor
- Capsida Biotherapeutics, Inc.
Tuberous Sclerosis, STXBP1 Encephalopathy With Epilepsy
Gait in Rare Diseases
The aim of this pilot study is to explore whether the knowledge and experience gained during the T-GaiD project (Treatment of Gait Disorders in Dravet Syndrome - NCT03857451) can be transferred to other populations with similar…
- Trial name
- GAGA (NCT05161494)
- Where
- Belgium
- Volunteers
- 41 people
- Sponsor
- Universiteit Antwerpen
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Epileptic Encephalopathy
A First-in-human Study of S230815 in Pediatric Participants With KCNT1-related Developmental and Epileptic Encephalopathy
Study CL1-230815-001 (KANDLE) is a Phase Ib/II, First In Human, multicentre, open-label, multiple ascending dose study to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamic (PD) effect of S230815 in…
- Trial name
- KANDLE (NCT07227857)
- Testing
- S230815- Starting dose A, S230815- Dose B, S230815- Dose C
- Where
- 15 sites in United States, France and 3 more
- Volunteers
- 20 people
- Sponsor
- Institut de Recherches Internationales Servier
Developmental and Epileptic Encephalopathies, Epileptic Spasms
Ketogenic Diet for Prevention of Epileptic Spasms in Infantile Onset Genetic Epilepsies
Epileptic spasms (ES) are a predominantly infantile seizure type observed frequently in certain genetic disorders. Ketogenic diet (high ratio of fat to carbohydrate/protein) is an established non-medication treatment for…
- Conditions
- Developmental and Epileptic Encephalopathies, Epileptic Spasms, Genetic Epilepsy, Neonatal and Infant Epilepsy
- Trial name
- NCT06700811
- Testing
- Ketogenic diet
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Heather Olson
Epilepsy, Dravet Syndrome (DS)
A Study of Soticlestat in Adults and Children With Rare Epilepsies
The main aim is to assess the long-term safety and tolerability of soticlestat when used along with other anti-seizure treatment. Participants will receive soticlestat twice a day.
- Conditions
- Epilepsy, Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS)
- Trial name
- Endymion 1 (NCT03635073)
- Testing
- Soticlestat
- Where
- 59 sites in United States, Australia and 6 more
- Volunteers
- 156 people
- Sponsor
- Takeda
SCN2A Encephalopathy, SCN8A Encephalopathy
A Clinical Trial of PRAX-562 in Subjects With Developmental and Epileptic Encephalopathies (DEE)
A Clinical Trial of PRAX-562 in Subjects With Developmental and Epileptic Encephalopathies (DEE)
- Trial name
- EMBOLD (NCT05818553)
- Testing
- PRAX-562
- Where
- 8 sites in United States, Israel and 2 more
- Volunteers
- 77 people
- Sponsor
- Praxis Precision Medicines
STXBP1 Encephalopathy With Epilepsy, SLC6A1 Neurodevelopmental Disorder, Developmental and Epileptic Encephalopathy
Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy
This study is to evaluate the use of glycerol phenylbutyrate for monogenetic developmental epileptic encephalopathies (DEEs). DEEs are characterized by epilepsy and developmental delay in early life.
- Trial name
- NCT04937062
- Testing
- Glycerol Phenylbutyrate 1100 MG/ML [Ravicti]
- Where
- 2 sites in United States
- Volunteers
- 50 people
- Sponsor
- Weill Medical College of Cornell University
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
GNAO1, Dystonia
Prospective Pilot Trial to Address Feasibility and Safety of Oral Zinc in GNAO1 Associated Disorders
The goal of this clinical trial is to investigate feasibility and safety of an oral therapy with zinc in patients affected by Guanine nucleotide-binding protein G(o) subunit alpha (GNAO1) associated disorders.
- Conditions
- GNAO1, Dystonia, Epilepsy, Development Delay
- Trial name
- ZINCGNAO1 (NCT06412653)
- Testing
- Zinc Acetate Dihydrate
- Where
- Germany
- Volunteers
- 13 people
- Sponsor
- Children's University Hospital Cologne, Germany
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
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