Epilepsy syndrome

This study tests whether adding perampanel (Fycompa) to current epilepsy medications can reduce seizures in children aged 1 month to 18 years. About 100 children with specific epilepsy syndromes or partial-onset seizures will receive the drug. The main goal is to see if at least …

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 phase 3 trial is testing the safety and tolerability of fenfluramine (Fintepla) in 25 infants aged 1 to 2 years with Dravet syndrome, a severe form of epilepsy. The drug is given as an oral solution twice daily alongside other seizure medications. Researchers will monitor he…

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 study tests the long-term safety and effectiveness of XEN1101 (azetukalner) in 880 adults with focal or tonic-clonic seizures. Participants who completed earlier studies will take XEN1101 capsules for up to 6 years. Researchers will track side effects and changes in monthly …

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 pilot study tests whether deep brain stimulation (DBS) of specific brain pathways can safely reduce seizures in people with Lennox-Gastaut syndrome, a severe form of epilepsy. Five participants receive the electrical stimulation as an add-on to their usual treatment. The goa…

This study tests a custom-made drug called an antisense oligonucleotide (ASO) designed for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare genetic disorder that affects movement and brain function. The drug aims to reduce the harmful effects of the mutated AT…

This study tests a custom-made genetic medicine (called an antisense oligonucleotide) designed for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare inherited brain disorder. The treatment aims to reduce seizures and improve quality of life. Only one participan…

This study tests whether brivaracetam, taken as an oral solution, can stop absence seizures in people aged 2 to 25 with childhood or juvenile absence epilepsy. Participants receive either the drug or a placebo, and seizure freedom is checked using a 24-hour EEG after 14 days. The…

This phase 2 trial tests whether high-dose chemotherapy followed by a stem cell transplant can stop the immune system from attacking the nervous system in people with autoimmune neurologic diseases like multiple sclerosis and stiff person syndrome. The treatment aims to weaken th…

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 study tests a new medicine called PRAX-562 in 77 children with rare genetic epilepsies (SCN2A or SCN8A). The goal is to see if it safely reduces motor seizures. The trial has two parts: a double-blind phase where some children get the drug and some get a placebo, followed by…

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 brain-responsive neurostimulation system in 24 people aged 12 and older with Lennox-Gastaut syndrome whose seizures are not controlled by medication. The device detects and responds to seizure activity in the brain to reduce drop seizures. The goal is to see if…

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 is testing the long-term safety of a once-daily drug called vormatrigine in 700 adults with focal or generalized tonic-clonic seizures. Participants must have already taken vormatrigine in a previous trial or program. The main goal is to check for side effects and chan…

This pilot study tests whether deep brain stimulation (DBS) of a brain region called the subiculum can safely reduce seizures in 6 people with drug-resistant temporal lobe epilepsy and bilateral hippocampal sclerosis. Participants receive a DBS device, and researchers track seizu…

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 program provides continued access to the seizure medication perampanel (Fycompa) for people who completed certain earlier studies and are still benefiting from the drug. It runs until the drug is commercially available in their country or no participants remain. Only those a…

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 whether an AI tool can help radiologists read brain CT and MRI scans more accurately and quickly. Researchers will compare how well doctors, AI alone, and doctors using AI together can spot abnormalities, urgent findings, and classify diseases. The goal is to red…

This study planned to test whether a brain-computer interface (BCI) could help children with drug-resistant epilepsy improve their attention. Participants would have used brain electrodes already implanted for epilepsy surgery to train a decoder that gives real-time feedback when…

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 small study tests whether low-intensity focused ultrasound (LIFU) can safely reduce seizures in people with temporal lobe epilepsy that doesn't improve with medication. Six participants with implanted brain electrodes will receive both real and sham ultrasound sessions. The …

This study looked at how sounds played during sleep affect brain waves and memory in children with Rolandic epilepsy. Researchers used brain scans (MRI, MEG/EEG) to understand which brain circuits are involved. The goal was to find new treatment ideas for this type of childhood e…

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 study will collect and analyze genetic data from 1000 people with suspected inherited metabolic diseases, including conditions like epilepsy and mitochondrial disorders. Researchers at Karolinska University Hospital aim to improve diagnosis by using advanced genetic testing …

This study looks at whether specific brain wave patterns on an EEG can predict how well the drug fenfluramine works for people with Lennox-Gastaut Syndrome (LGS), a severe form of epilepsy. Researchers will follow 20 children and adults aged 2 to 35 who are already taking fenflur…

This study looks at gene activity in brain tissue removed during surgery from 10 people with temporal lobe epilepsy and hippocampal sclerosis. The goal is to find which genes are linked to the disease and may help predict outcomes. No new treatment is being tested; instead, resea…

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 compares a new type of brain scanner (OPM-MEG) to the standard MEG scanner used to plan epilepsy surgery in children. The new scanner uses a lightweight hat instead of a rigid helmet, making it more comfortable and better fitting for kids. Researchers will check if the…

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…

This pilot study at the University of Chicago is testing whether a non-invasive brain stimulation technique called TMS can improve memory and reduce seizure-related brain activity in people with mesial temporal lobe epilepsy (MTLE). Thirty-four adults with drug-resistant MTLE wil…

This study gathers MRI, CT, and ultrasound images from 300 adults—some healthy, some with kidney or brain disease—to build a collection for future not-for-profit research. No new treatments are being tested; the goal is to make medical images available to scientists for advancing…