Lennox-Gastaut Syndrome

Researchers are evaluating RAP-219, an investigational drug, for adults with refractory focal epilepsy, a condition involving difficult-to-control focal seizures. This open-label, multi-center study aims to assess the long-term safety, tolerability, how the drug behaves in the body, and its antiseizure activity in adults who have not responded well to previous treatments. Participants will receive RAP-219 starting with one 0.125 mg capsule daily for 3 days, then one 0.25 mg tablet daily for 28 days, followed by one 0.75 mg tablet daily for the rest of the treatment period. The study is open-label, meaning all participants know they are receiving RAP-219, and the treatment period may last up to 112 weeks with ongoing monitoring. During the study, participants will be regularly assessed for treatment-related side effects and seizure frequency compared to their baseline before treatment. Researchers will monitor seizure-free days, longest seizure-free intervals, and other seizure-related measures using clinical assessments and RNS system data. Safety will be tracked through adverse event reporting up to 8 weeks after the last dose. The total participation duration can be up to about 2 years, with ongoing evaluations throughout.

Researchers are developing a multicenter registry to collect and share data on pediatric patients who have undergone deep brain stimulation (DBS) for movement disorders such as dystonia, epilepsy, Tourette syndrome, and mood disorders. The study aims to improve understanding of DBS safety and effectiveness in children, as current data are limited and individual centers often have too few cases for strong research. This registry will support large-scale analyses and help refine DBS as a treatment option for hyperkinetic movement disorders in the pediatric population. The study involves gathering both retrospective and prospective clinical data from multiple pediatric centers. The registry will collect information on surgical techniques, patient outcomes, implant sites, and long-term effects of DBS. This collaborative data-sharing approach enables comprehensive evaluation of which patients benefit most from DBS and how it impacts their quality of life over time. Participants include children aged 0 to 18 years who have already received or are scheduled to receive DBS for neurological movement disorders. Data will be collected over five years to monitor safety, efficacy, and quality of life outcomes. The study does not involve treatment administration but focuses on gathering and analyzing clinical information. Parental or legal guardian consent is required for prospective participation.

Researchers are investigating epilepsy-dyskinesia syndromes, which are rare genetic diseases causing both movement disorders and epilepsy in children. This multinational retrospective survey, supported by the International Parkinson and Movement Disorder Society, aims to collect detailed clinical and molecular data to better understand these conditions. The study focuses on identifying patterns in disease features, progression, and genetic links to improve knowledge and support precision medicine. The study collects previously recorded data from multiple countries, harmonizing information on clinical features, disease progression, age of onset, genetic variants, and coexisting neurological conditions. By standardizing this data, the survey addresses challenges in rare disease research like small, dispersed patient groups and inconsistent protocols. The goal is to build a shared clinical database and analyze how movement and seizure disorders relate at both clinical and molecular levels. Participants are children aged 0 to 18 years with diagnosed movement disorders linked to specific genetic variants. The study reviews existing medical records and genetic information without new treatments or interventions. Researchers will assess the disease spectrum, how movement disorders affect quality of life, and the effectiveness of symptomatic treatments over one year. The study encourages international collaboration to advance understanding and improve care for these rare conditions.

Researchers are evaluating the safety, tolerability, and initial effectiveness of low-intensity focused ultrasound (LIFU) neuromodulation using the NaviFUS System in patients with drug-resistant unilateral or bilateral temporal lobe epilepsy (DR-TLE). This pilot study involves adults with epilepsy that has not responded to standard medications, aiming to reduce abnormal brain activity and seizures. Participants will undergo six LIFU treatment sessions over three consecutive weeks. Each treatment uses specific ultrasound exposure settings delivered by the NaviFUS System. Before treatment, patients will be observed and asked to keep a seizure diary for 8 weeks. After completing treatments, there is a 12-week follow-up period to monitor outcomes. Patients may continue their anti-seizure medications throughout the study. Participants will keep a seizure diary throughout the study to track seizure frequency and severity. Researchers will also assess safety by monitoring adverse events for up to 23 weeks. Additional evaluations include EEG scans to observe changes in brain activity, and questionnaires that measure anxiety, depression, and the personal impact of epilepsy. The total study duration includes a 2-month baseline, 3 weeks of treatment, and 12 weeks of follow-up.

Researchers are evaluating the efficacy of perampanel as an additional therapy for children and adolescents with pediatric epileptic syndrome and partial-onset seizures. The study aims to measure how well perampanel reduces seizure frequency, focusing on the 50% responder rate during the maintenance phase of the core study. This phase 2, open-label trial includes participants from 1 month to less than 18 years old with specific epilepsy diagnoses. The study includes a Core Study and two Extension Phases. The Core Study starts with a 4-week screening period, followed by a 23-week treatment period divided into a 10-week titration phase and a 13-week maintenance phase. Participants receive perampanel oral suspension or tablets once daily for up to 56 weeks. Those completing the Core Study may join Extension Phase A, which has a 33-week treatment period and a 4-week follow-up. Extension Phase B is available for participants in areas without commercial perampanel or extended access programs, continuing treatment as deemed beneficial by investigators. Throughout the study, participants will undergo regular assessments including seizure frequency monitoring, cognitive and behavioral evaluations, growth and development measurements, and safety checks such as vital signs, laboratory tests, and electroencephalograms. Researchers will track responders who achieve seizure reductions and evaluate overall clinical impressions and drug effects. Participation may last up to about 60 weeks, including all phases and follow-up periods, with careful monitoring of adverse events and treatment impact.

Researchers are evaluating the use of Pulsed Low-Intensity Focused Ultrasound (PLIFU) to reduce or suppress seizure activity in adults experiencing non-convulsive or focal motor status epilepticus (SE) who have not responded to standard treatments. The study focuses on brain regions that generate or control seizures, aiming to assess the effects of this non-invasive brain stimulation in a hospital setting. Participants will receive up to two sessions of PLIFU treatment in the Intensive Care Unit. Each session lasts 10 minutes and uses a custom device that delivers ultrasound to specific brain areas without damaging tissue. Treatments will be given after anti-seizure medications have been administered, and the ultrasound is applied using safe, non-thermal settings. During the study, participants will be monitored continuously with surface EEG to measure epileptic brain activity before, during, and after PLIFU treatment. Researchers will observe changes in seizure activity and follow participants throughout their hospital stay. The main outcome is the change in epileptiform activity measured by EEG at baseline and 10 minutes after treatment. The study aims to gather preliminary data to support future research.

Researchers are evaluating the effectiveness of an AI-personalized mobile app called Medilepsy in improving medication adherence and transition readiness among underserved adolescents and young adults with epilepsy, ages 14 to 24, in Florida. This study compares the AI-enhanced version of the app with a standard version that does not include AI personalization. The research focuses on key outcomes including medical adherence, usability, and language experience. Participants are randomly assigned to one of two groups: the intervention group uses the AI-personalized Medilepsy app with an interactive Smart Chatbot that provides tailored prompts, adaptive education, and personalized support for epilepsy self-management. The comparison group receives a standard version of the Medilepsy app, which offers static prompts and general educational content without personalized feedback. The study lasts 3 to 6 months, during which participants use their assigned app version. During the study, participants will be monitored for medication adherence and transition readiness skills through app usage and assessments. Researchers will evaluate the usability of the app at the end of the 3 to 6 month period. The study collects data on how well the AI features support epilepsy management compared to the standard app version. Safety and participant engagement are also observed throughout the trial duration.

Researchers are evaluating the efficacy and safety of clemizole HCl as an additional treatment for children and adults aged 2 to 55 years with Lennox-Gastaut syndrome (LGS), a complex form of epilepsy. This multicenter, Phase 3, randomized, double-blind, placebo-controlled trial is designed to assess how well clemizole HCl works alongside other treatments in controlling seizures associated with LGS. Participants will be randomly assigned to receive either clemizole HCl or a placebo, both given as oral solutions, during the double-blind period. After completing this phase, eligible participants may choose to join an optional open-label extension where they will receive clemizole HCl for up to three years. The study includes an initial observational period, the double-blind treatment period, and the long-term extension phase. During the trial, participants will undergo various assessments including seizure counts, electroencephalogram (EEG) evaluations, and quality of life questionnaires. Researchers will track changes in seizure frequency and intensity, as well as global impressions of change by clinicians and caregivers. Safety will be monitored throughout the study, which may last up to approximately 172 weeks. Participants will be regularly evaluated for treatment effects and adverse events to understand the impact of clemizole HCl on LGS.

Researchers are studying patients with refractory or focal epilepsy who undergo pre-surgical evaluation using Stereoelectroencephalography (SEEG). The goal is to create a detailed brain atlas of human cognition by collecting brain recordings taken during cognitive tasks performed as part of routine clinical care. This atlas will provide high temporal and spatial resolution data to better understand how epilepsy and epilepsy surgery affect cognitive functions. Participants will have intracerebral electrodes implanted as part of their invasive SEEG assessment to locate epileptogenic zones and evaluate cognitive risks before surgery. During this procedure, brain activity is recorded while patients perform cognitive tasks. This study uses Dynamic Spectral Imaging (ISD) to map gamma oscillations in the brain, which are linked to complex cognitive functions. Behavioral and neuropsychological data may also be collected after surgery. During the study, participants complete cognitive tasks while SEEG signals are visually and quantitatively analyzed over about three weeks. Researchers measure brain activity through functional mapping and record composite behavioral data. The study monitors patients’ intellectual capacity to comply with cognitive testing and collects detailed brain function data to improve understanding of epilepsy's impact on cognition. Participation lasts through the clinical evaluation and data collection periods.

Researchers are evaluating the safety and effectiveness of BMB-101 in reducing seizure frequency in adults with Absence Epilepsy, including Epilepsy with Eyelid Myoclonia (Jeavons Syndrome), as well as Developmental Epileptic Encephalopathies such as Dravet and Lennox-Gastaut syndromes. This open-label Phase 2 study will last up to six months and aims to better understand how BMB-101 may impact these conditions. Participants will take BMB-101 liquid orally twice daily for up to three months. The study includes a one-month screening period, followed by the treatment phase with dose adjustments (titration and tapering/washout), and then a one-month follow-up period. There will be six clinic visits during the study to monitor progress and safety. Participants will be asked to complete seizure diaries and attend scheduled clinic visits for evaluations. Researchers will use 24-hour EEGs to measure changes in seizure activity and generalized spike-wave discharges, along with assessments of quality of life. The study will monitor safety, tolerability, and seizure frequency changes over a total participation period of about six months.