Marlboro Township

Researchers are evaluating clemizole hydrochloride EPX-100 as an additional treatment for children and adults with Dravet syndrome DS, a severe form of epilepsy. This Phase 3, multicenter, randomized, double-blind, placebo-controlled study aims to assess the safety and effectiveness of clemizole hydrochloride in reducing seizures. The study includes participants with confirmed DS who continue to have seizures despite other treatments and have a documented SCN1A genetic mutation. Participants first undergo a 4-week Observational Period to establish baseline seizure activity. Then, they enter a 16-week Double-Blind Period where they receive either clemizole hydrochloride or a placebo as an oral solution. Following this, eligible participants who complete the double-blind phase may continue to an Open-Label Extension Period, receiving clemizole hydrochloride for up to 3 years. During the study, participants are regularly monitored for changes in countable motor seizures per 28 days and other seizure-related outcomes. Assessments include clinical evaluations and seizure tracking, with safety monitored throughout the trial. The primary outcome focuses on seizure reduction over 16 weeks, while secondary outcomes evaluate seizure frequency, seizure-free days, and clinical impressions of improvement. Participants are followed for up to approximately 172 weeks, ensuring long-term safety and efficacy data are collected.

Researchers are evaluating clemizole HCl as an additional treatment for children and adults aged 2 to 55 years who have Lennox-Gastaut syndrome LGS, a condition characterized by seizures and abnormal brain activity. This Phase 3 trial aims to assess how well clemizole HCl works and how safe it is when added to existing treatments. The study is randomized, double-blind, and placebo-controlled to ensure reliable results. Participants will go through three main phases an Observational Period, a Double-Blind Period, and an optional Open-Label Extension. During the Double-Blind Period, participants receive either clemizole HCl or a placebo as an oral solution. Those who complete this phase may continue to receive clemizole HCl for up to three years in the Open-Label Extension Phase. Throughout the study, participants will be monitored closely with various assessments, including measuring seizure frequency using the CMMS-28 scale over up to 16 weeks. Researchers will also check global impressions of change, quality of life scores, and record any treatment-emergent side effects. The studys overall duration may last up to several years, allowing for long-term safety and effectiveness monitoring.

Researchers are evaluating outcomes in female patients with acute thoracolumbar compression fractures, focusing on how pain is managed and how well patients can function after injury. This observational study tracks patients whose fractures occurred within the last 3 months or less. The study aims to measure pain levels using the Numeric Rating Scale NRS and Brief Pain Inventory BPI, and quality of life through the PROMIS questionnaire at multiple time points before and after intervention. Participants will receive treatment using the commercially available SPRINT Peripheral Nerve Stimulation PNS system for 60 days. Pain and quality of life will be assessed prior to the intervention, then again at 2 weeks, 3 months, and 6 months after PNS implantation. The primary focus is on pain reduction, while secondary outcomes include improvements in function and quality of life as reported by participants through digital data collection. During the study, participants will complete assessments of pain and function at specified intervals. These include questionnaires like the PROMIS and patient-reported outcome measures collected via CareSense, a digital system. Researchers will monitor changes in pain scores and quality of life over time. The total participation includes assessments up to 6 months following the nerve stimulation treatment, allowing for observation of both short- and longer-term effects.

Researchers are evaluating KL1333 in adults with primary mitochondrial disease PMD to assess its effects on fatigue symptoms, daily living impacts, and physical strength and endurance. This Phase 2 study compares KL1333 with a placebo over 48 weeks, focusing on how the medication may influence selected disease aspects and evaluating its safety and tolerability. Participants take either KL1333 or a matching placebo tablet twice daily during the 48-week treatment period. The study is divided into three parts an 8 to 12-week screening and baseline phase, the 48-week treatment phase, and a 5-week safety follow-up. Participants are randomly assigned to receive KL1333 or placebo, with a higher chance of receiving KL1333, and neither they nor the study team know which treatment is given. Throughout the study, participants will undergo assessments including patient-reported fatigue measures and the 30-second sit-to-stand test at the start and end of treatment. Additional evaluations cover quality of life, activity interference, global impressions of severity and change, and neurological function. Safety is monitored through physical examinations, ECGs, laboratory tests, and adherence to treatment and study procedures. Total participation lasts approximately 61 to 65 weeks.