Roseville

Researchers are conducting a Phase 3 clinical trial to study the effects of relutrigine in people with developmental and epileptic encephalopathies (DEEs). The study is designed to evaluate the drug's efficacy, safety, tolerability, and how the body processes it compared to a placebo. This trial includes participants aged 2 to 65 years who have experienced seizure onset before age 12 and have a confirmed diagnosis of DEE. Participants will be randomly assigned to receive one of two doses of relutrigine (1.0 mg/kg/day or 1.5 mg/kg/day) or a placebo. The medication is given once daily either by mouth or through a gastrostomy or jejunostomy tube. After the initial double-blind treatment period, there is an open-label extension where all participants may receive the study drug. During the study, participants will be monitored for changes in seizure frequency over 16 weeks as the primary outcome. Safety and tolerability will also be assessed throughout the trial. Various evaluations, including heart monitoring and seizure tracking, will be conducted to ensure participant well-being. The overall study will track participants from screening through treatment and follow-up phases to gather comprehensive data about relutrigine's effects.

Researchers are evaluating the effectiveness and safety of BHV-7000 in adults with refractory focal onset epilepsy, a condition where seizures originate in one area of the brain and do not respond well to current treatments. This Phase 2/3 clinical trial aims to determine whether BHV-7000 can reduce seizure frequency in this population. The study is divided into two parts. In Part A, participants are randomly assigned to receive either 25 mg or 50 mg of BHV-7000, or a matching placebo, taken once daily. After completing Part A, participants move to Part B, where they are randomized to receive 75 mg of BHV-7000 or a matching placebo, also taken once daily. Both parts are randomized and double-blinded to ensure unbiased results. Participants will be monitored from Week 8 to Week 20 of each part for changes in average seizure frequency, serious adverse events, discontinuations due to side effects, and laboratory abnormalities. Researchers will track seizure diaries and assess safety and tolerability throughout the study. The total duration includes both study parts with regular evaluations to measure the drug’s impact and participant safety.

Researchers are conducting a multicenter, Phase 3, randomized, double-blind, placebo-controlled trial to evaluate the effectiveness and safety of clemizole HCl (EPX-100) as an additional treatment for children and adults diagnosed with Lennox-Gastaut syndrome (LGS). This study focuses on participants aged 2 to 55 years who experience this severe form of epilepsy characterized by multiple types of seizures and abnormal brain activity. Participants must have a history of abnormal cognitive development and seizure onset at age 11 or younger. Participants will be randomly assigned to receive either clemizole HCl or a placebo, both given as oral solutions. The study includes an Observational Period, followed by a Double-Blind Period where neither participants nor researchers know who receives the active drug or placebo. After this, there is an option to enter an Open-Label Extension Period where all participants may receive clemizole HCl. This design allows researchers to closely monitor the drug's effects compared to placebo over time. During the study, participants will undergo various assessments to measure changes in their seizure frequency and severity, specifically using the Percent Change in CMMS-28 from baseline up to 16 weeks. Safety and tolerability will also be monitored throughout the trial. Consent from participants or their legal representatives is required, and the study is designed to carefully track treatment adherence and any adverse effects over the course of the study periods.

Researchers are studying tuberous sclerosis complex (TSC) and lymphangioleiomyomatosis (LAM) through the TSC Alliance TSC Biosample Repository and Natural History Database. This project aims to better understand these conditions, which can affect various organs like the brain, kidneys, heart, lungs, and skin, potentially leading to new treatment options. The Natural History Database collects clinical information from individuals with TSC over their lifetime, including retrospective and prospective data. Participants may choose to provide biological samples such as blood, cheek swabs, or tissue following routine medical procedures. These samples are collected at recognized clinical sites, participants' homes, educational meetings, or other approved locations. The collected samples are sent to the Van Andel Institute Biorepository for processing and storage, following approved procedures. Genetic testing may be performed on these samples, with participants having the option to receive their genetic results and counseling. Throughout the study, researchers link clinical data with biosamples to deepen understanding of TSC and LAM. The project allows distribution of these samples and data to approved researchers for further study. Participation includes providing samples and clinical information, with an average follow-up period of about 15 years. The study welcomes individuals of all ages diagnosed with TSC or sporadic LAM.