Bethlehem

Researchers are evaluating the safety and effects of a gene therapy called 4D-150 in adults aged 50 years and older with neovascular (wet) age-related macular degeneration (AMD) who are currently receiving anti-VEGF treatments and have shown a positive response. This Phase 1/2 trial includes a dose-escalation stage, a randomized controlled expansion phase, and additional cohorts to optimize steroid use and extend the population studied. The study also includes substudies to assess dosing safety in the opposite eye and to examine vector shedding. Participants receive a single injection of 4D-150 into the eye on Day 1 at assigned dose levels. Some participants receive aflibercept injections as an active comparator. Following the injection, participants are monitored monthly for 24 months to track safety and treatment effects. Those who receive 4D-150 enter a long-term follow-up period lasting up to five years to evaluate extended safety and the duration of the gene therapy’s activity. The substudies include one-time dosing in the contralateral eye and assessments of vector shedding, with safety monitored for one year and continued follow-up through five years. Throughout the study, participants undergo regular eye function and structure tests, including visual acuity and retinal thickness measurements using imaging technologies. Researchers track adverse events and the need for additional aflibercept injections, as well as changes in vision and retinal health. Participants must comply with study procedures and agree to use barrier methods during and after treatment to prevent fluid transmission. The study ensures comprehensive safety monitoring, with evaluations continuing for up to five years after treatment administration.

Researchers are conducting a non-interventional, observational study to understand the short-term progression of geographic atrophy (GA) caused by age-related macular degeneration (AMD) in people aged 55 and older. The study aims to identify participants with progressing GA to measure structural and functional changes and explore how these relate to genetic or lifestyle factors. This research is sponsored by Complement Therapeutics and involves multiple centers. Participants with confirmed bilateral GA secondary to AMD will be observed without receiving any study treatment. The study uses imaging techniques such as fundus autofluorescence (FAF) and optical coherence tomography (OCT) to assess GA lesion size and progression. Evaluations occur at the start, 3 months, and 6 months to monitor changes in GA area and related functional vision measures. During the study, participants will undergo visual acuity tests, microperimetry to measure retinal sensitivity, and imaging scans to track GA progression. Researchers will also collect and analyze genetic and lifestyle data to understand factors influencing the disease. The primary outcome focuses on short-term GA progression measured at baseline, month 3, and month 6. The total study duration and safety monitoring details are based on these scheduled visits.

Researchers are evaluating real-world patient characteristics, treatment methods, and long-term outcomes in people with symptomatic obstructive hypertrophic cardiomyopathy (HCM) in the United States and Europe. The study includes patients receiving mavacamten, other treatments, or no treatment due to intolerance or treatment failure. The US sub-study focuses on safety of mavacamten, while the European sub-study assesses both safety and effectiveness of mavacamten in everyday care. Participants may receive mavacamten or standard treatments like beta blockers, non-dihydropyridine calcium channel blockers, or disopyramide as prescribed by their doctors. The study observes these groups over time in routine clinical settings without altering their care. The study includes two main groups based on treatment type and monitors outcomes up to five years. Participants undergo evaluations including echocardiograms, heart function assessments using New York Heart Association (NYHA) class, left ventricular outflow tract gradient measurements, and patient-reported health questionnaires. Researchers also track heart failure events, arrhythmias, major cardiovascular events, hospitalizations, mortality, and biomarkers like NT-proBNP and cardiac troponin. Follow-up varies by region, lasting up to 18 months in Europe and up to 5 years in the United States.

Researchers are evaluating the safety, tolerability, and effectiveness of a one-time intravitreal injection of SAR446597 in participants with Geographic Atrophy (GA) caused by Age-related Macular Degeneration (AMD). This is a Phase 1/2, two-part, multicenter study that aims to understand how this treatment affects participants with this condition over time. Participants will receive a one-time injection of SAR446597 or a sham injection depending on the study part and group assignment. Multiple dose levels of SAR446597 will be tested in successive cohorts during Part I, followed by dose-specific groups and a sham control group in Part II. The core study phase lasts about 2 years, after which participants enter an Extended Follow-Up phase for an additional 3 years to monitor long-term outcomes. During the study, participants will undergo regular assessments including eye exams, visual acuity tests, and monitoring for any adverse events related to the treatment. Researchers will measure changes in the size of GA lesions and visual acuity at several time points up to 2 years, with safety follow-up extending to 5 years. This approach helps evaluate treatment effects and long-term safety in those receiving SAR446597 or sham injections.

Researchers are evaluating the effectiveness and safety of combining divarasib and pembrolizumab compared to pembrolizumab with pemetrexed and carboplatin or cisplatin for first-line treatment in adults with KRAS G12C-mutated advanced or metastatic non-squamous non-small cell lung cancer (NSCLC). This phase III study focuses on patients who have not received prior systemic treatment for this type of lung cancer and aims to provide new options for this specific mutation. Participants are randomly assigned to one of two groups. One group receives an oral daily dose of divarasib along with pembrolizumab given by intravenous infusion every three weeks. The other group receives pembrolizumab combined with pemetrexed and either carboplatin or cisplatin, also administered intravenously every three weeks. Treatments continue according to the study schedule to assess how well each combination works and their safety profiles. Throughout the study, participants will be closely monitored for progression-free survival and overall survival for up to approximately five years. Additional assessments include tumor response, quality of life related to lung cancer symptoms, duration of response, and side effects reported by patients. Safety is also tracked by recording adverse events and their impact on daily activities. This comprehensive monitoring helps researchers understand the full effects of the treatments over time.

Researchers are evaluating the real-world experience of participants treated with BRIUMVI4 (ublituximab-xiiy) for relapsing multiple sclerosis (RMS). The study aims to assess the safety, effectiveness, and overall treatment experience of this medication in everyday clinical practice. The trial is observational, focusing on patients who have been prescribed BRIUMVI and tracking their outcomes over time. Participants in this study will receive BRIUMVI4 as an intravenous (IV) infusion specifically for RMS treatment. The study includes participants who have not previously received BRIUMVI infusions before the study begins, allowing observation from the start of their treatment. This approach helps understand how the medication performs outside of controlled clinical trial settings. During the study, researchers will monitor participants for up to 96 weeks, measuring the annualized relapse rate (ARR) to evaluate disease activity. They will also track adverse events, serious adverse events, and infusion-related reactions at each infusion visit. Participants will undergo regular assessments to capture their health status and any side effects, providing a comprehensive view of treatment safety and patient experience over nearly two years.

Researchers are evaluating the safety and effectiveness of the 3M20V.A.C.AE Peel and Place dressing when used with 3M20V.A.C.AE Therapy for treating various wound types. This study collects post-market data to better understand how well this dressing works and how safe it is for patients with open or closed wounds. The study is prospective, conducted at multiple centers, and involves a single group of participants. Participants will receive the 3M20V.A.C.AE Peel and Place dressing applied along with 3M20V.A.C.AE Therapy. The study plans to enroll about 90 subjects, each with one study wound, including 60 open wounds and 30 closed or covered wounds. Treatment includes an initial dressing application, with dressing changes as needed but no more than 7 days apart, followed by an end-of-treatment visit between 7 and 14 days after the first application. Participants will be involved for up to 20 days, beginning with a screening period up to 5 days. During the study, researchers will monitor the incidence of adverse device effects and assess wound healing, including the closure of surgical incisions and skin flaps, skin graft success, and overall wound healing rates. Safety and healing outcomes will be evaluated at 14 days, with multiple clinical visits for dressing changes and assessments.

Researchers are studying the effects of experimental drugs called pozelimab and cemdisiran on people with Geographic Atrophy (GA), a late stage of Age-related Macular Degeneration (AMD) that affects central vision. The trial aims to compare the rate at which GA progresses in patients receiving cemdisiran alone, pozelimab combined with cemdisiran, or a placebo. The study also explores possible side effects, how much of the drugs remain in the blood over time, and whether the body develops antibodies that might impact treatment. Participants are randomly assigned to one of three groups: one receiving both pozelimab and cemdisiran, one receiving cemdisiran alone, and one receiving a placebo. All treatments are given by subcutaneous injection. The study is conducted in a double-masked manner to ensure unbiased results. Vaccinations against meningococcal and pneumococcal infections are required before participation. During the study, participants will attend regular clinic visits where eye imaging and vision tests will be performed to monitor the size of GA lesions and changes in visual acuity. Blood samples will be taken to measure drug levels and antibody development. The primary outcome is the growth rate of GA lesion area over 52 weeks. Safety will be monitored throughout and up to nearly six years. The overall study duration allows detailed assessment of treatment effects and tolerability.

This trial investigates whether adding intismeran autogene to pembrolizumab after surgery can help people with non-small cell lung cancer (NSCLC) remain cancer-free longer than those receiving pembrolizumab with a placebo. It focuses on patients whose tumors did not completely respond to treatment before surgery. The study is a Phase 3, randomized, double-blind trial assessing adjuvant therapy following neoadjuvant treatment and surgery in resectable Stage II to IIIB NSCLC without complete pathological response. Participants first receive neoadjuvant treatment with pembrolizumab and chemotherapy every 3 weeks for up to 4 cycles, lasting about 12 weeks. After surgery, they get adjuvant treatment with pembrolizumab every 6 weeks for up to 7 cycles, combined with either intismeran autogene or placebo given by intramuscular injection every 3 weeks for up to 9 doses, for a total of about 42 weeks. The study compares these two groups to evaluate the impact on cancer recurrence. During the trial, participants undergo regular evaluations including disease-free survival monitoring for up to approximately 97 months, and other measures like overall survival, metastasis-free survival, and quality of life assessments for up to around 129 months. Safety is tracked by recording adverse events and therapy discontinuations. Follow-up involves questionnaires and clinical tests to assess health status and treatment effects over the long term.

Researchers are evaluating the addition of Tersolisib (LY4064809/STX-478) to other anti-cancer drugs as a first treatment for adults with advanced hormone receptor-positive (HR+) and human epidermal growth factor receptor 2-negative (HER2-) breast cancer that has a specific genetic mutation called PIK3CA. This phase 3 study aims to assess both the effectiveness and safety of this combination treatment. Participants can remain in the study as long as the treatment is helping control the cancer without causing unbearable side effects. Participants will receive Tersolisib orally in combination with an oral CDK4/6 inhibitor and endocrine therapy (ET), which may be given orally or by intramuscular injection. The study includes different groups: one receiving two different doses of Tersolisib plus CDK4/6 inhibitor and ET, another receiving one dose of Tersolisib plus CDK4/6 inhibitor and ET, and a placebo group receiving placebo with CDK4/6 inhibitor and ET. The CDK4/6 inhibitors used include ribociclib, palbociclib, or abemaciclib, while ET options include anastrozole, letrozole, exemestane, or fulvestrant. During the study, participants will undergo evaluations that include measuring tumor response, progression-free survival, overall survival, and quality of life using questionnaires. Researchers will monitor participants regularly through scans, lab tests, and clinical assessments to track treatment response and side effects. The study may last up to 7 years to observe long-term outcomes, and participants will be closely followed to assess how the cancer responds and how the treatments affect their health and well-being over time.