Developmental and epileptic encephalopathy

This trial tested a single dose of CAP-002 gene therapy in children aged 18 months to 8 years with STXBP1 encephalopathy, a rare genetic brain disorder causing seizures and developmental delays. The study aimed to check safety and whether it could reduce seizures and improve skil…

This study looked at the long-term safety of soticlestat when added to standard seizure medicines for people with Dravet syndrome or Lennox-Gastaut syndrome. It included 352 children and adults who had already been in earlier studies. The trial was terminated early, so results ar…

This study looked at the long-term safety of the drug NBI-921352 for people with a rare genetic seizure disorder called SCN8A-DEE. It was an extension of an earlier study, and participants took the drug alongside their usual seizure medications. The study was stopped early and on…

This study tested the drug fenfluramine (FINTEPLA) in adults with Dravet syndrome whose seizures were not controlled by other medications. The trial was open-label and added fenfluramine to existing treatments. It aimed to see if the drug could reduce monthly seizures by at least…

This study looked at the long-term safety of soticlestat, an experimental drug, in children and adults with severe forms of epilepsy like Dravet syndrome and Lennox-Gastaut syndrome. Participants took soticlestat twice a day along with their usual seizure medicines. The study was…

This study looked at the long-term safety of a drug called NBI-827104 in children with a rare epilepsy that causes seizures during sleep. The trial was stopped early and only enrolled 19 children, so the results are limited. The main goal was to see how many children had serious …

This study tested an experimental drug called NBI-921352 in people aged 2 to 21 with a rare, severe form of epilepsy caused by a change in the SCN8A gene. The goal was to see if adding this drug to their current seizure medicines could reduce how often they had seizures. The tria…

This study tested whether lorcaserin, when added to current medications, could reduce convulsive seizures in people with Dravet syndrome, a severe form of epilepsy. The trial planned to enroll about 22 participants aged 2 and older, but it was terminated early. The main goal was …

This study tested a cannabidiol (CBD) oral solution in children under 2 years old with tuberous sclerosis complex, Dravet syndrome, or Lennox-Gastaut syndrome who had uncontrolled seizures. The goal was to see if CBD is safe and can reduce seizures. However, the study was termina…