Marlboro Township

Researchers are conducting a global Phase 3 study to evaluate the safety and effectiveness of clemizole hydrochloride (EPX-100) as an additional treatment for children and adults with Dravet syndrome, a severe form of epilepsy. The study focuses on participants with seizures that are not fully controlled by anti-epileptic drugs and includes those with a confirmed SCN1A gene mutation and other specific clinical features of Dravet syndrome. This randomized, double-blind, placebo-controlled trial aims to provide important data on this investigational therapy. Participants will receive either clemizole hydrochloride or a placebo, both given as oral solutions. The study includes a 4-week observational period without treatment changes, followed by a 16-week double-blind treatment phase where participants receive either the study drug or placebo. After this, there is an open-label extension period where all participants can receive clemizole hydrochloride. The treatment periods are designed to assess the drug's effect on seizure control over time. During the study, participants will be closely monitored for changes in the frequency of countable motor seizures compared to their baseline levels. Evaluations will include seizure tracking over 28-day periods during the titration, maintenance, and baseline phases. Safety and tolerability will also be assessed throughout the trial. Overall, the trial lasts about 20 weeks, with additional follow-up during the extension phase to gather long-term information about the treatment's effects.

Researchers are evaluating the safety, tolerability, pharmacokinetics, and efficacy of TP-05 in healthy adults who are at high risk of tick exposure and Lyme disease. This Phase 2b randomized, double-blind, placebo-controlled study aims to understand how TP-05 performs compared to a placebo in preventing Lyme borreliosis. The study enrolls adults aged 18 to 70 years who are overtly healthy and able to comply with study procedures. Participants will be randomly assigned to receive either a low dose or high dose of TP-05 or a matching placebo, all administered orally according to a predefined dosing schedule. The study includes a screening period, a treatment period lasting up to 24 weeks, and a safety follow-up period. During treatment, participants will be monitored closely for any adverse effects and signs of Lyme borreliosis. Throughout the study, participants will undergo safety assessments including monitoring of adverse events, clinical laboratory tests, vital signs, physical exams, and electrocardiograms. Researchers will follow participants for approximately 15 months to track safety outcomes and any tick bites or symptoms of Lyme disease. Key measures include changes from baseline in laboratory results, vital signs, and ECG parameters, ensuring thorough safety evaluation over the study course.

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 conducting a Phase 3, multicenter, double-blind, randomized, placebo-controlled clinical trial to evaluate the efficacy and safety of vormatrigine in adults with focal seizures. The study focuses on adults aged 18 to 85 years who have focal onset epilepsy and are currently taking one to three anti-seizure medications (ASMs). The trial aims to compare vormatrigine to placebo in reducing the frequency of focal seizures over a 12-week period. Participants will receive once-daily oral doses of vormatrigine at 20 mg, 30 mg, or 40 mg, or a placebo, for 12 weeks. The study includes multiple treatment groups to assess different dose levels of vormatrigine. The administration is daily, and patients will continue their stable ASM regimen during the trial. During participation, individuals will be monitored for seizure frequency using seizure diaries, which must be kept for at least 80% of days during an observation period before randomization. Researchers will evaluate the efficacy of vormatrigine by measuring the change in focal seizure frequency. Safety assessments and monitoring will be conducted throughout the study. The total participation duration includes a 12-week treatment period, with ongoing evaluation of seizure activity and safety.

This research aims to study female patients with acute thoracolumbar compression fractures that occurred within the past 3 months or less. The focus is on understanding how pain and daily function improve following treatment. The study particularly evaluates pain reduction and quality of life using various patient-reported outcome measures over a six-month period. Participants will receive treatment with the commercially available SPRINT Peripheral Nerve Stimulation (PNS) system for 60 days. Pain levels will be monitored using the Numeric Rating Scale (NRS) and Brief Pain Inventory (BPI), while quality of life will be assessed using the PROMIS questionnaire. Measurements will be taken one week before the intervention and then at 2 weeks, 3 months, and 6 months after PNS implantation. During the study, participants will complete pain and quality of life questionnaires and have their functional outcomes tracked through a digital data collection system called CareSense. The primary outcome is the change in pain from before treatment to multiple follow-up points. Researchers will also analyze functional improvements and overall quality of life. Safety and effectiveness will be monitored throughout the six months post-treatment.

Researchers are evaluating the effectiveness of BHV-7000 in treating adults with refractory focal onset epilepsy, a condition where seizures originate in specific areas of the brain and have not responded to previous treatments. This Phase 2/3 trial aims to assess the safety, tolerability, and ability of BHV-7000 to reduce seizure frequency in participants who continue to have seizures despite using anti-seizure medications. The study follows classification criteria set by the International League Against Epilepsy and includes participants aged 18 to 75 years. Participants will be randomly assigned to receive either BHV-7000 at doses of 50 mg or 75 mg once daily, or a matching placebo, in a double-blind setup where neither participants nor researchers know which treatment is given. The treatment period focuses on monitoring changes in seizure frequency over 28-day averages from baseline through weeks 8 to 16. The study design includes careful control and comparison to evaluate the investigational drug's impact. During the study, participants will keep accurate seizure diaries to track their seizures. Researchers will measure changes in the average number of seizures over 28-day periods as the primary outcome. Safety and tolerability will also be monitored closely. The study requires participants to be currently treated with one to three anti-seizure medications and to meet specific epilepsy criteria. Overall participation includes screening, treatment, and follow-up to assess the drug's effects and participant safety.