Setauket- East Setauket

Researchers are evaluating the safety and early effectiveness of an experimental drug called CS5001 in patients with advanced solid tumors and lymphomas. This first-in-human Phase 1 study includes patients with diseases that have progressed despite prior treatments. The study aims to find the appropriate dose of CS5001 when used alone or alongside other systemic therapies. Participants will receive CS5001, with the dose and schedule determined by a safety committee. The drug may be given alone or combined with other treatments such as Rituximab, Gemcitabine, Oxaliplatin, Lenalidomide, Cyclophosphamide, Doxorubicin, Vincristine, and Prednisone. The study has a dose escalation phase lasting about 6 months to identify the maximum tolerated dose and recommended phase 2 dose, followed by a dose expansion phase that may last up to 2 years. During the study, patients will undergo various assessments including tumor evaluations using standardized criteria, safety monitoring for adverse events up to 90 days after the last dose, and pharmacokinetic studies. Researchers will track treatment responses and tolerability throughout the study period to better understand the effects and safety of CS5001 in this patient population.

Researchers are evaluating the safety and immune response of a group B streptococcus (GBS) vaccine in healthy pregnant women and their babies in this Phase 3 randomized, placebo-controlled, double-blinded trial. The study includes pregnant women aged 49 or younger between 24 and 36 weeks of gestation with uncomplicated singleton pregnancies and no major fetal abnormalities. Participants must also have documented negative tests for HIV, syphilis, and hepatitis B during this pregnancy. The goal is to learn how the vaccine works and to monitor safety for both mothers and their infants. Participants will receive one injection of either the GBS6 vaccine or a saline placebo. Pregnant women will be followed for up to 14 months, including 6 months after delivery. Their babies will be followed for about 12 months after birth. A subset of infants will also receive routine vaccinations such as diphtheria toxoid-containing vaccines and pneumococcal vaccines according to their country's immunization schedule, with blood samples collected one month after completing primary and toddler booster doses. Mothers will be monitored for local and systemic reactions within 7 days after vaccination, adverse events through 1 month, and serious or medically attended events up to 6 months postpartum. Infants will be observed for adverse events from birth through at least one year, with serious and medically attended events tracked through 6 months. Researchers will also measure antibody levels in infants at birth to assess the vaccine's potential to protect against early and late onset GBS disease. Mothers will attend at least 3 to 4 study visits, some via telephone, to support ongoing safety and immunogenicity assessments.

This research aims to learn about the safety, how the body processes, and how well the study medicine called nirmatrelvir/ritonavir works for treating COVID-19 in children under 18 years old who are not hospitalized but are at risk for severe illness. The study focuses on pediatric patients confirmed to have COVID-19, with early symptoms and risk factors for worsening disease. Participants will receive the study medicine, which is given by mouth, to evaluate its effects. The trial is open-label, meaning both researchers and participants know the treatment being given. The study is designed as a single-arm Phase 2/3 trial conducted at multiple centers. During the study, researchers will measure medicine levels in the blood at specific times to understand drug absorption and processing. They will monitor for any side effects or adverse events up to 34 days after starting treatment. Vital signs and other health indicators will be regularly checked to assess safety and overall health. The total participation period includes treatment and follow-up to gather comprehensive safety and effectiveness data.

Overweight and obesity are major health challenges in the United States, contributing to conditions like heart disease, stroke, and type 2 diabetes. Physicians often lack training in Weight Management Counseling (WMC), which hinders addressing these issues during clinical visits. This research evaluates the MRWeight curriculum, designed to improve medical residents' skills in WMC using a structured, evidence-based approach during their residency training. Participants include postgraduate year 1 residents in Internal Medicine residency programs. The MRWeight program involves several components over 12 months: a 45-minute foundational didactic session covering key WMC concepts at the start; a series of six email modules over months 2 to 8 called the 3Ps Program (Prepare, Practice, Process) that use video-based scenarios for skill development; a second 45-minute didactic session in months 8 to 10 addressing barriers to WMC; and two email reinforcements during months 10 to 12. Residents in the comparison group receive only a foundational course PowerPoint on WMC without the additional sessions or modules. Residents participate in three assessments over 18 months to measure changes in WMC skills and their adoption in clinical practice. These include video communication assessments at months 1 and 12, and skill assessments at months 1, 12, and 18. Researchers will evaluate the effectiveness of the MRWeight program on improving counseling skills and explore factors influencing these outcomes, with the overall study lasting 18 months per participant.

Researchers are evaluating surgical and minimally invasive treatments for lumbar spinal stenosis (LSS) by comparing Medicare patients who received the MILD procedure against those who had interspinous process decompression (IPD). The study focuses on outcomes such as the rate of harms related to the initial procedure and the frequency of additional surgical or minimally invasive interventions within 24 months after treatment. Enrollment includes patients treated from January 1, 2017, onward, with continuation until the sponsor decides to stop. The MILD procedure involves percutaneous image-guided lumbar decompression, performed under fluoroscopy through a dorsal approach to partially remove tissue and bone at the affected spinal level. The control group receives the IPD procedure for LSS. Both groups are monitored for a 24-month period post-index procedure using Medicare claims data to track reoperations and any harms. Participants contribute data through Medicare claims without needing prior enrollment or consent, as the study is exempt from IRB oversight. Researchers collect and analyze information on procedure-related harms and subsequent interventions over two years. This approach allows evaluation of long-term safety and effectiveness outcomes for patients treated with either MILD or IPD.

Researchers are investigating the effectiveness, safety, and tolerability of iptacopan (LNP023) alongside standard care in adults with active lupus nephritis Class III-IV, with or without Class V. This Phase 2 trial aims to assess how well iptacopan works in this condition by comparing it with placebo combined with standard treatments. The study is carefully designed to explore different doses and their impact on kidney health in participants with biopsy-confirmed active lupus nephritis. Participants receive either iptacopan or placebo along with their usual care, including corticosteroids and immunosuppressive drugs like MMF or MPS, for 52 weeks. The study is divided into two parts, both lasting 52 weeks, during which participants take the assigned medications. The treatment is given in a double-blind manner, meaning neither the participants nor the researchers know who is receiving the active drug or placebo. Throughout the study, researchers monitor kidney function and disease activity, focusing on the proportion of patients achieving complete renal response by week 24 without kidney flares. Participants undergo regular assessments including lab tests and clinical evaluations to track their response and safety. They are followed closely during the 52 weeks of treatment to ensure careful observation of effects and any side effects, supporting an in-depth understanding of iptacopan's role in managing lupus nephritis.

Researchers are investigating plasminogen deficiency, also known as hypoplasminogenemia, to better understand its natural history, the variety in how it appears in different people, and to find markers that predict how the disease progresses. This international study involves a large group of affected individuals and their first-degree family members to gather detailed information and improve future treatments. The study includes both retrospective data from the past year and prospective data collected over three years. Participants come from various regions, with data and samples collected primarily through two centers: the Indiana Hemophilia & Thrombosis Center in the Americas and the University of Milan in Europe. Samples like blood and urine will be analyzed centrally in Italy, while urine tests are done locally. The study will analyze plasminogen activity, genetics, and other related factors, creating a biobank of specimens for current and future research. The study also explores links with certain bacterial strains and altered plasminogen products. Each participant will be involved for three years, with in-person visits at the start and end of the study to collect samples and information. Follow-up calls will occur every six months, and additional data may be gathered during unscheduled visits if needed. The main outcomes include tracking the natural course of the disease over two years, understanding factors affecting disease severity over five years, and building a long-term specimen biobank over 15 years.

Researchers are evaluating the effectiveness of ravulizumab compared to a placebo in reducing proteinuria and improving kidney function in adults with Immunoglobulin A Nephropathy (IgAN) who are at risk of disease progression. This Phase 3 study involves participants who have a confirmed diagnosis of IgAN and are receiving stable standard treatments for their condition. The study aims to provide important information about the impact of ravulizumab on kidney health over time. About 510 eligible participants will join the study. Around 450 will be randomly assigned to receive either ravulizumab or a placebo through weight-based intravenous infusions. Participants will continue their stable IgAN treatments during the study. An additional group of approximately 60 participants with more advanced kidney disease will also be enrolled. After Week 106, all participants have the option to enter an open-label phase to receive ravulizumab. Participants will be monitored through urine tests measuring protein levels and kidney function assessments over the course of the study. Key outcomes include changes in proteinuria at Week 34 and kidney filtration rate at Week 106. Safety and treatment effects will be closely observed throughout the study and during any extended access periods. The study includes thorough screening and follow-up assessments to track progress and ensure participant well-being.