Setauket- East Setauket

Researchers are gathering real-world data from patients with Philadelphia chromosome-positive chronic myelogenous leukemia in the chronic phase Ph-CML-CP who are treated with tyrosine kinase inhibitors TKIs approved for first or second line therapy. This registry study aims to collect evidence on the tolerability, safety, effectiveness, and patient-reported outcomes over a prospective follow-up period of 5 years to understand long-term treatment results in routine healthcare settings. Participants will be those receiving TKI treatments including asciminib, bosutinib, dasatinib, imatinib, or nilotinib as prescribed in standard medical care, either as initial therapy or after one prior TKI therapy. The study includes patients who began these treatments on or after specified dates and those continuing asciminib after participating in an interventional trial. There is no treatment allocation instead, patients are observed based on their current prescribed TKI therapy. During the 5-year follow-up, participants treatment continuation and discontinuation due to adverse events will be monitored, along with the occurrence of adverse events, treatment switches, molecular and hematological responses, survival outcomes, and patient-reported measures of health, symptoms, and medication adherence. Data will be collected from electronic medical records and claims data with patient consent, ensuring ongoing safety and effectiveness observations in real-world care up to 5 years.

Researchers are evaluating the safety and early effects of an experimental drug called CS5001 in people with advanced solid tumors and lymphomas. This study is a first-in-human trial, meaning it is the first time CS5001 is being tested in humans. The study aims to find safe dosage levels and observe how well the drug works alone or combined with other cancer treatments. Participants may receive CS5001 either alone or together with other systemic therapies such as Rituximab, Gemcitabine, Oxaliplatin, Lenalidomide, Cyclophosphamide, Doxorubicin, Vincristine, or Prednisone. The doses and schedules are determined by a safety committee. The study includes a dose escalation phase to find appropriate dosing, followed by a dose expansion phase to further evaluate the drugs effects. During the study, participants will be closely monitored for side effects and treatment responses using criteria specific to solid tumors and lymphomas. Researchers will assess the maximum tolerated dose, recommended dose for future studies, adverse events, and objective response rate up to two years. Blood tests will measure drug concentrations and antibody levels. Safety is followed until 90 days after the last dose or until new cancer treatments begin. The total duration of participation varies by individual treatment and response.

The BEATRIX study focuses on healthy pregnant women aged 49 or younger between 24 and 36 weeks of pregnancy to evaluate the safety and immune response of a group B streptococcus GBS vaccine. Researchers aim to understand how this vaccine works in pregnant women and their babies, assessing various safety measures and immune responses related to GBS. This study is a Phase 3, randomized, placebo-controlled, double-blinded trial sponsored by Pfizer. Participants will receive a single injection of either the GBS vaccine or a placebo saline. After birth, a subset of infants will receive routine vaccines according to each countrys immunization schedule, including vaccines for diphtheria, pneumococcal disease, and others. Some infants will have blood samples taken after completing their primary and toddler vaccine doses to evaluate immune responses. Pregnant participants will visit the study site at least three to four times, with some visits possibly conducted by phone, and may stay involved for up to 14 months, including six months after delivery. Their babies will be followed for about 12 months, with a subset participating for up to 19 months. Researchers will monitor local and systemic reactions, adverse events, and antibody levels in both mothers and infants to assess safety and immunogenicity of the vaccine throughout the study period.

Researchers are evaluating the safety, how the body processes, and how well the study medicine nirmatrelvirritonavir works for treating COVID-19 in children under 18 years old who are not hospitalized but at risk of severe illness. This trial is a Phase 3, open-label study focusing on nonhospitalized pediatric patients with COVID-19 to better understand the effects of this oral medication. The study medicine is given by mouth as tablets or powder twice daily for 5 days, totaling 10 doses. Different dosing forms and amounts are used depending on the participants age and weight, ranging from under 1 month old up to 18 years. Participants are assigned to one of several groups based on these factors to receive the medicine accordingly. During the study, participants will have blood samples taken to measure drug levels at specific times, and researchers will monitor for any side effects or changes in vital signs up to 34 days after starting treatment. They will also measure viral load through nasal swabs at several points over 28 days and assess hospitalizations or deaths related to COVID-19. Participants feedback on the medicines taste and acceptability will be collected during treatment.

This research aims to improve how medical residents learn to counsel patients on weight management. It addresses the challenge that many physicians lack training in this area during their residency. The study evaluates the MRWeight curriculum, which uses spaced education and the 5As framework to teach residents weight management counseling WMC skills, comparing it to a standard obesity and weight management course. The study is a randomized controlled trial involving Internal Medicine residency programs and focuses on how well residents adopt and apply WMC skills in clinical practice. Participants are divided into two groups. The intervention group attends two educational sessions and receives six email modules with video-based communication assessments over 12 months, reinforcing WMC concepts and practice. The comparison group receives only a foundational course via PowerPoint slides without the additional sessions or email modules. Both groups participate in three assessments over 18 months to evaluate their WMC skills and adoption in patient encounters. During the study, residents complete surveys and video communication assessments at baseline, 12 months, and 18 months. The researchers measure changes in WMC skills and how residents adopt these skills in clinical settings. The study monitors residents through multiple cohorts and collects data on the effectiveness of the training methods. Participation involves online and in-class evaluations, with the overall study lasting 18 months.

Researchers are comparing the rates of surgical and minimally invasive interventions, as well as any harms, in Medicare beneficiaries treated with the MILD procedure versus those treated with interspinous process decompression IPD for lumbar spinal stenosis with neurogenic claudication. This observational study uses Medicare claims data to follow patients for 24 months after their initial procedure starting from January 1, 2017. The purpose is to evaluate outcomes between these two types of procedures without requiring prior patient enrollment or consent. The study includes two groups patients who received MILD, which is a percutaneous image-guided lumbar decompression performed under fluoroscopic guidance through a dorsal approach to the spine, and patients who received IPD, a different device-based decompression procedure. Data on reoperations and complications will be collected for both groups over a 24-month follow-up period using Medicare claims. Enrollment continues until the sponsor decides to stop. Participants involvement is passive as the study uses existing Medicare claims data. Researchers will monitor rates of harms related to the initial procedure and subsequent surgical or minimally invasive interventions over two years. No direct patient visits or interventions are conducted, and the study is exempt from institutional review board oversight. The total study duration extends to December 2026, covering cases treated since early 2017.

Researchers are evaluating iptacopan LNP023 alongside standard care to study its effectiveness, safety, and tolerability in adults with active lupus nephritis Class III-IV, with or without Class V. This phase 2 trial is randomized, double-blind, and placebo-controlled, aiming to explore dosing and treatment outcomes in this patient group. Participants receive either iptacopan combined with standard care, placebo matching iptacopan with standard care, or iptacopan with placebo as part of the standard care. Each treatment is taken for 52 weeks. The study proceeds in two parts, with different combination regimens of iptacopan, placebo, and standard care, including oral corticosteroids or their absence. During the study, participants undergo assessments at baseline and weeks 24 and 52 to measure kidney response, protein levels in urine, fatigue, and lupus disease activity using specific scoring tools. Researchers monitor the proportion of patients achieving complete renal response without flares and other kidney and health outcomes. Safety and tolerability are also evaluated over the course of treatment and follow-up.

Researchers are conducting an international retrospective and prospective study to better understand hyposplasminogenemia, a condition caused by plasminogen deficiency. This study aims to define the natural history of the disease, explore the variability in how it presents in different people, identify markers that predict disease progression, and help develop improved treatments. The research is being conducted across two main centers in the Americas and Europe, gathering data from affected individuals and their first-degree family members. The study will collect both retrospective data from the past year and prospective data for three years on approximately 100 affected individuals and about 500 family members. Biological samples such as DNA and plasma will be collected and analyzed centrally in Italy, except for urine samples that will be analyzed locally. The study will test plasminogen activity and antigen levels, genetic mutations, and other factors influencing disease expression. Follow-up will include in-person visits at the start and end of the study, with telephone check-ins every six months and unscheduled visits as needed based on clinical care. Participants will be involved in providing samples and clinical information at baseline and at the end of their three-year study period. The researchers will monitor disease progression and gather data through regular telephone follow-ups and clinical visits. The main outcomes measured include the natural history of plasminogen deficiency, factors influencing disease severity, and the creation of a biobank for future research. The study is designed to improve understanding of this rare condition over a long-term observation period.