Chevy Chase

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.

The purpose of this study is to assess the long-term safety and tolerability after an intravitreal injection (a shot of medicine into the eye) of JNJ-81201887 administered in parent clinical studies.

Researchers are evaluating whether the drugs retatrutide and tirzepatide can prevent major adverse liver outcomes (MALO) in adults with metabolic dysfunction-associated steatotic liver disease (MASLD) who are at high risk. This Phase 3 trial enrolls about 4,500 adults with MASLD identified by non-invasive tests indicating an increased likelihood of developing serious liver problems. The study aims to understand how these treatments might affect liver health over time compared to a placebo. Participants will be randomly assigned to receive either retatrutide, tirzepatide, or a placebo, all given by subcutaneous injection. The study will last approximately 224 weeks, during which participants may attend 25 to 30 clinic visits for monitoring and assessment. After the main study, eligible participants can join an optional 2-year extension where all will receive either retatrutide or tirzepatide regardless of their original group. Throughout the trial, participants’ liver function and disease progression will be closely monitored through various health assessments. Researchers will track the time to the first major adverse liver event as the main outcome. Safety and health status will be evaluated regularly during clinic visits, ensuring thorough observation over the long study period.

Researchers are evaluating how well oral icotrokinra works, its safety, and how well patients tolerate it in adults and adolescents with moderately to severely active ulcerative colitis, a chronic condition where the colon lining becomes inflamed and develops ulcers. This is a Phase 3 study aimed at finding effective treatments for this condition using a rigorous comparison. Participants will receive either icotrokinra tablets or placebo tablets taken by mouth. The study includes an induction phase and a maintenance phase, with adults participating in a randomized, double-blind, placebo-controlled design, while adolescents join an open-label maintenance study. Throughout the study, researchers will monitor clinical remission rates at 12 weeks during induction and at 40 weeks during maintenance. Participants will undergo assessments including endoscopic evaluations and pregnancy tests for females of childbearing potential. Safety and tolerability will be closely observed, with the total study duration covering both induction and maintenance periods.

This research evaluates the safety, tolerability, and effectiveness of a single intravitreal injection of SAR446597 in people with Geographic Atrophy (GA) caused by Age-related Macular Degeneration (AMD). It is a Phase 1/2, two-part, multicenter study focusing on participants aged 60 years and older who have this eye condition. The study aims to assess the impact of this treatment on GA over a long period. Participants receive a one-time injection of SAR446597 directly into the eye, or a sham injection during Part II of the study. The core study phase lasts about two years for each participant, followed by an Extended Follow-Up (EFU) phase that continues for three more years to monitor long-term safety and effects. During the study, researchers monitor for ocular and non-ocular treatment-emergent adverse events and serious adverse events from Day 1 through Week 104. Participants undergo regular assessments of their eye health and vision, including visual acuity and lesion size evaluations. The total participation includes the core phase and extended follow-up, ensuring thorough observation of treatment safety and tolerability over five years.

Researchers are evaluating corneal endothelial cells in people aged 50 and older who have neovascular age-related macular degeneration (nAMD). The study focuses on participants treated with the Port Delivery System (PDS) refilled every 24 weeks. This Phase IV, open-label trial aims to understand changes in corneal endothelial cell density over time in the eye receiving treatment compared to the fellow eye. The study involves delivering ranibizumab 100 mg/mL via the PDS implant. Supplemental treatment with intravitreal injections of ranibizumab (0.5 mg of a 10 mg/mL formulation) in the study eye may be given if needed. If participants stop the study treatment, they may receive intravitreal ranibizumab injections based on the investigator's judgment. Treatment and monitoring occur over at least 48 weeks. Participants will undergo detailed eye examinations including specular microscopy to measure corneal endothelial cell density at baseline and week 48. Historical visual acuity and imaging data will be reviewed. Researchers will monitor safety, disease activity, and treatment response through visual acuity assessments, optical coherence tomography, and other imaging techniques. The main outcome is the percent change in corneal endothelial cell density in the treated eye compared to the fellow eye after 48 weeks.

Researchers are evaluating the safety and effectiveness of a new oral medicine called ALG-000184 compared with tenofovir disproxil fumarate (TDF) in adults with chronic hepatitis B virus (HBV) infection. This Phase 2 study includes people who have never been treated or are currently not treated, and it focuses on both HBeAg-positive and HBeAg-negative participants. The study aims to understand how well these treatments control the virus over time. Participants will receive either ALG-000184 or TDF as a once-daily oral tablet for 48 weeks in a randomized, double-blind setting. After this period, all participants have the option to continue treatment with ALG-000184 alone for an additional 48 weeks in an open-label extension. The study includes two parts: one for HBeAg-positive subjects and one for HBeAg-negative subjects, each with the possibility of joining a liver biopsy sub-study. During the study, participants will be regularly monitored for viral levels, specifically measuring HBV DNA to see if it falls below a certain limit after 48 weeks. Researchers will also check safety and liver health through blood tests and imaging. The total study involvement can last up to 96 weeks, including the treatment extension. The study looks closely at how the virus responds to treatment and the overall health of participants throughout this time.

Researchers are studying the effects of two experimental drugs, pozelimab and cemdisiran, in adults aged 50 to 85 who have Geographic Atrophy (GA) caused by Age-related Macular Degeneration (AMD), a condition that affects central vision. The study aims to compare how quickly GA progresses in patients treated with cemdisiran alone, a combination of pozelimab and cemdisiran, or a placebo. Additional goals include monitoring possible side effects, measuring drug levels in the blood, and checking for antibodies that might reduce drug effectiveness or cause side effects. Participants receive subcutaneous injections of either pozelimab combined with cemdisiran, cemdisiran alone, or a placebo. The study is randomized, double-masked, and placebo-controlled, conducted at multiple centers. Treatment schedules and dosing are managed as described in the protocol, with vaccinations for meningococcal and pneumococcal infections required prior to participation. Throughout the study, participants undergo regular clinic visits where eye imaging using Fundus Autofluorescence (FAF) tracks the progression of GA lesion area over 52 weeks. Researchers also monitor safety, side effects, and immune responses, ensuring adherence to study procedures. The main outcome measured is the growth rate of the GA lesion area over one year, helping to evaluate the potential benefits and risks of the study drugs.

Researchers are evaluating the effectiveness and safety of icotrokinra in adults with moderately to severely active Crohn's disease, a chronic condition causing severe inflammation in the intestinal tract. This Phase 2b/3 study aims to understand how well icotrokinra works compared to a placebo in improving symptoms and intestinal healing in this patient group. Participants will receive either icotrokinra or a matching placebo orally every day. The study includes both induction and maintenance phases where researchers assess clinical and endoscopic responses at specific time points, such as Week 12 and Week 40, to determine treatment effects over time. Throughout the study, participants will undergo various assessments including clinical evaluations, endoscopic exams, and safety monitoring. Researchers will measure outcomes like clinical response, clinical remission, and endoscopic healing at Weeks 12 and 40. The study involves regular monitoring to track the participants' health and treatment adherence over the duration of the trial.

Researchers are evaluating the safety and effectiveness of NNZ-2591 in children aged 3 to 12 years with Phelan-McDermid Syndrome, a rare genetic disorder caused by abnormalities in the SHANK3 gene. This Phase 3, randomized, double-blind, placebo-controlled study aims to compare NNZ-2591 with a placebo to understand its impact on symptom improvement and adaptive behavior. After providing consent, eligible pediatric participants will enter a 4-week screening period where their health, eligibility, and symptom severity will be assessed. Those who qualify will be randomly assigned to receive either NNZ-2591 or a matching placebo taken orally twice daily for 13 weeks. Following this treatment period, participants will be monitored during a 2-week safety follow-up phase. Throughout the study, children will undergo various evaluations including assessments of symptom changes using the Phelan-McDermid Syndrome Assessment of Change and the Vineland Adaptive Behavior Scales. Researchers will monitor safety through laboratory tests and ECGs during screening and follow-up. The total involvement for each participant lasts at least 19 weeks including screening, treatment, and safety monitoring.