- authorisedPhase I and Phase II (Integrated)- First administration to humans
Phase 1-2, Open-Label, Single and Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION337 in Patients with Dravet Syndrome
This early clinical trial is testing an experimental medicine called ION337 for people with Dravet syndrome. Researchers want to find out if the drug is safe, how the body handles it, and whether it can help reduce the number of major seizures patients experience.
Spain · France · Belgium - recruitingHuman Pharmacology (Phase I)- Other
Bioavailability study of stiripentol after single oral administration of two different formulations (capsule and oral suspension) in 24 healthy subjects
This study is looking at how a medicine called stiripentol is absorbed by the body. It compares two ways of taking it – capsules and a liquid medicine – in healthy adults, to see which works best.
France - active not recruitingPHASE3
A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome
This study is testing a new medicine, zorevunersen, for children and teenagers with Dravet syndrome. We want to see if it can help reduce seizures and improve their overall health, using a safe and gentle approach.
United States · China · France - active not recruitingPHASE1, PHASE2
A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome
This study is looking at a new gene therapy, ETX101, for young children with a specific type of Dravet Syndrome. Doctors want to see if it's safe and helps reduce seizures in children aged 6 to 47 months.
United Kingdom - recruiting
SCN1A Horizons A Natural History Study of SCN1A-related Epilepsies in the United Kingdom
This study looks at how Dravet Syndrome and other epilepsy linked to the SCN1A gene affect people over three years. Researchers want to understand seizures, development, and behaviour, and how different gene changes might lead to different experiences.
United Kingdom - recruitingPHASE3
A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome
This study is looking into a new medication called EPX-100 (clemizole hydrochloride) for children and adults with Dravet syndrome. It aims to see if this medicine can help control seizures and is safe to use alongside their current treatments.
United States · Argentina · Brazil - recruiting
Multicentre Real-life Follow-up Study of Rare Epileptic Syndromes in Children and Adolescents
This study looks at how children and teenagers with rare types of epilepsy respond to treatments and how their learning and behaviour change over time. It aims to gather real-life information to help doctors better understand and manage these conditions, as there isn't much information available right now.
France - active not recruitingPHASE3
A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome
This study is looking at a medicine called fenfluramine in very young children (aged 1 to just under 2 years old) with a rare type of epilepsy called Dravet syndrome. It aims to check if the medicine is safe and well-tolerated at different doses.
United States · Belgium · Germany - active not recruitingPHASE3
A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)
This study is looking into a new medication called LP352 for Dravet Syndrome. We want to see if it can help reduce seizures and if it's safe for children and adults. Participants will either get LP352 or a dummy pill called a placebo.
United States · Australia · Belgium - recruitingPHASE1, PHASE2
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
This study is looking at a new medicine called ETX101 for babies and children with Dravet Syndrome. Researchers want to see if it's safe and helps reduce seizures. It includes different age groups, from 6 months up to 17 years.
United States · Australia · United Kingdom