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

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 phase 3 study is testing the long-term safety and effectiveness of LP352 (Bexicaserin) in 324 children and adults with developmental and epileptic encephalopathy (DEE), including Dravet and Lennox-Gastaut syndromes. Participants who completed earlier studies will receive LP3…

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 follows 158 people in France who are prescribed Epidyolex (a cannabidiol-based medicine) for seizures as part of their normal care. Researchers will track how long people stay on the treatment, side effects, seizure frequency, and changes in daily functioning and quali…

This early-stage study tests a gene therapy called ETX101 in 5 children with Dravet syndrome, a severe form of epilepsy. The therapy delivers a gene that helps calm overactive brain cells. Researchers will check if it safely reduces seizure frequency and improves thinking and dai…

This study follows about 111 people with Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex who are taking Epidyolex (a CBD oral solution) for seizures. Researchers track how many stay on the drug for a year and how many seizure-free days they have. It's an o…

This Phase 3 study tests whether fenfluramine (ZX008) can safely reduce seizures in 87 children and adults with CDKL5 deficiency disorder, a rare genetic condition causing severe epilepsy. Participants receive either the drug or a placebo, followed by an open-label extension wher…

This study offers fenfluramine to people with Dravet syndrome whose seizures have not improved with other medications. The goal is to provide access to the drug and monitor its safety. Participants must have a genetic diagnosis of Dravet syndrome and have tried all available seiz…

This study looks at the long-term safety of an experimental drug called zorevunersen (STK-001) in 60 people with Dravet syndrome, a severe form of epilepsy. Participants must have completed an earlier STK-001 study. Researchers will monitor side effects, seizure frequency, and qu…

This program provides expanded access to the drug stiripentol for people aged 6 months and older with Dravet syndrome, a severe form of epilepsy. The goal is to help control seizures that do not respond to other treatments. Participants must have a confirmed diagnosis and meet sa…

This study tests a gene therapy called ETX101 in 4 children with Dravet syndrome, a severe seizure disorder caused by a gene change. The therapy uses a harmless virus to deliver a working copy of the gene to brain cells. The main goals are to see if it is safe and if it reduces s…

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This study tests the long-term safety of an inhaled medication called Staccato alprazolam for people aged 12 and older who have prolonged seizures. About 300 participants will use the inhaler when a seizure starts to see if it stops the seizure quickly and without serious side ef…

This study tests a program called FACE-Rare, designed to support family caregivers of children with rare, life-limiting diseases. The program includes three sessions to help families prepare for future medical decisions and improve their quality of life. Researchers will compare …

This study follows 154 people taking Epidiolex (cannabidiol) for seizures caused by Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex. Researchers will monitor for signs of chronic liver injury and fibrosis over time using blood tests and liver scans. The go…

This observational study aims to measure how many people in Portugal have Dravet syndrome (DS) or Lennox-Gastaut syndrome (LGS), two rare and severe forms of epilepsy. Researchers will review existing medical records from about three public hospitals to estimate the percentage of…

This study creates a registry for up to 500 people with CDKL5 Deficiency Disorder (CDD), a rare genetic condition that causes seizures and developmental delays. Patients and their caregivers will provide information about symptoms, treatments, and quality of life over several yea…

This study looks at how repeated seizures affect breathing and heart function in people with Dravet syndrome, a severe form of epilepsy. Researchers will monitor 92 children and adults for 24-48 hours using video, brain wave, heart, and breathing recordings. The goal is to find c…