Neonatal/infantile-onset epilepsy syndrome with developmental and epileptic encephalopathy

This Phase 3 study tests a drug called zorevunersen in 170 children aged 2 to 17 with Dravet syndrome, a severe form of epilepsy. The drug is given as an injection into the spine and compared to a sham procedure. The goal is to see if it reduces seizures and improves daily living…

This Phase 3 trial tests a single inhaled dose of alprazolam (a fast-acting sedative) to stop prolonged seizures in people aged 12 and older. The goal is to see if it can end a seizure within 90 seconds and prevent it from coming back for at least 2 hours. About 350 participants …

This study tests an experimental drug called EPX-100 (clemizole) in 150 children and adults with Dravet syndrome, a severe form of epilepsy. The goal is to see if adding EPX-100 to current treatments can safely reduce the number of motor seizures. Participants will be randomly as…

This early-stage trial tests a gene therapy called RC001 in children aged 2 to 18 with Dravet syndrome, a severe form of epilepsy caused by a genetic mutation. The therapy is given as a single injection into the spinal fluid. The main goal is to check safety and how the body proc…

This study tests a gene therapy called ETX101 for children with Dravet syndrome, a severe form of epilepsy caused by a genetic change. The treatment uses a harmless virus to deliver a working copy of the gene to help control seizures. The trial includes infants and children up to…

This study tests a new medicine called ION337 in children aged 2 to 12 with Dravet syndrome, a severe form of epilepsy. The main goal is to see if the drug is safe and tolerable when given as a spinal injection. Researchers will also measure how the drug moves through the body an…

This study tests whether the drug LP352 can safely reduce seizures in children and adults with Dravet syndrome, a severe form of epilepsy. About 160 participants will receive either LP352 or a placebo, and researchers will track changes in seizure frequency over several months. T…

This study is testing the safety of fenfluramine, a drug already approved for children 2 and older with Dravet syndrome, in younger children aged 12 to 23 months. Dravet syndrome is a severe genetic epilepsy that starts in the first year of life. The study will enroll 5 children …

This study tests an investigational drug called relutrigine in 160 children with a severe form of epilepsy called developmental and epileptic encephalopathy (DEE). The goal is to see if the drug can safely reduce monthly seizure frequency compared to a placebo. After the initial …

This study tests the drug fenfluramine in 10 children aged 12 to 36 months whose infantile spasms did not get better with standard treatments. The children take fenfluramine for 21 days, and doctors check brain waves and seizure diaries to see if the spasms stop. If it helps, the…

This early-phase study tests whether a ketogenic (high-fat) diet can prevent epileptic spasms in infants with genetic epilepsy. Ten babies under 9 months old will receive the diet and be monitored with regular EEGs. The goal is to see if this approach is safe and feasible, potent…

This study investigates how rehearsal (repeating information) and sleep help solidify memories in people with epilepsy. Participants learn object-location pairs, with some pairs rehearsed and others not, and their memory is tested after a short delay and again after 12 hours—eith…

This study uses PET and MRI scans to look at serotonin pathways in the brainstem of adults with Dravet syndrome, a severe form of epilepsy. Researchers want to see if these pathways are different in Dravet patients compared to those with other drug-resistant epilepsy and healthy …

This study follows 1,000 children and teens with rare epilepsies (like West and Dravet syndromes) to see how different treatments affect their seizures and thinking skills. Researchers will compare care across hospitals to find what works best. The goal is to give families cleare…

This study follows 50 children and young adults with Dravet syndrome caused by SCN1A gene mutations over four years. Researchers will use standard tests to measure changes in thinking, movement, and daily skills. They will also look for chemical markers in the blood that might re…

This study uses powerful MRI scanners to look at the brains and spinal cords of 700 people, including healthy volunteers and those with conditions like multiple sclerosis, Alzheimer's, and Parkinson's. The goal is to understand why some brains stay healthy while others decline. R…