Charleston

Researchers are evaluating the efficacy and safety of rilvegostomig compared to pembrolizumab as first-line treatments for patients with metastatic non-small cell lung cancer (mNSCLC) whose tumors have high PD-L1 expression. This Phase III, randomized, double-blind, and global study focuses on participants with stage IV mNSCLC who do not have certain genetic mutations or rearrangements and are eligible for systemic therapy. Participants receive either rilvegostomig or pembrolizumab intravenously on Day 1 of each 21-day cycle. The study compares these two biological treatments given as monotherapy. Both groups will be monitored over time to assess treatment impact and safety. Throughout the study, participants undergo evaluations including tumor measurements by CT or MRI, performance status assessments, and organ function tests. Researchers will measure overall survival and progression-free survival for up to approximately five years. Tumor samples are collected before treatment for central testing, and participants’ health and treatment responses are closely followed during the trial period.

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.

This trial investigates the safety and effectiveness of rilvegostomig combined with fluoropyrimidine and trastuzumab deruxtecan (T-DXd) compared to trastuzumab, chemotherapy, and pembrolizumab in adults with HER2-positive locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 with a combined positive score of 1 or higher. Additionally, rilvegostomig combined with trastuzumab and chemotherapy is studied separately to understand each component's contribution. This Phase 2, randomized, open-label, global study is conducted at 200-250 sites in about 25 countries. Participants are randomly assigned to one of three arms: Arm A receives rilvegostomig, fluoropyrimidine, and T-DXd; Arm B receives trastuzumab, chemotherapy, and pembrolizumab; Arm C receives rilvegostomig, trastuzumab, and chemotherapy. Treatments are administered mostly by intravenous infusion every three weeks, with capecitabine given orally twice daily. The study compares these treatment regimens to evaluate their effects on the cancer. Throughout the study, participants undergo assessments including tumor measurements, organ function tests, and heart function evaluation to ensure safety and monitor disease progression. The main outcomes measured are progression-free survival and overall survival for up to approximately six years. Researchers will also monitor adverse events and overall health status during and after treatment.

Researchers are evaluating the safety and effectiveness of spinal cord induced muscle stimulation (MuscleSCS) to treat chronic low back pain. This prospective, multi-center study focuses on two groups of patients with chronic pain: those who already have spinal cord stimulation (SCS) devices providing significant pain relief, and those who are candidates for trial and potential permanent SCS implantation. The study targets individuals with persistent back pain and aims to assess MuscleSCS as a treatment option within this population. The study involves two groups: the Pre-existing SCS Group, including patients with an implanted SCS system and successful pain relief for more than six months, and the Trial SCS Group, including patients eligible for an SCS trial. Both groups use devices with percutaneous or paddle leads and BurstDR IPGs. Participants will be evaluated using MuscleSCS, with treatment and monitoring designed to assess safety and efficacy in managing chronic low back pain. Participants will be involved from enrollment through a three-month follow-up visit. During this time, researchers will measure pain intensity using the Numerical Rating Scale (NRS), disability via the Oswestry Disability Index (ODI), quality of life with EQ5D-5L, pain disability index (PDI), pain catastrophizing scale (PCS), and patient global impression of change (PGIC). The study includes regular assessments to monitor these outcomes and ensure participant safety throughout the trial period.

Researchers are evaluating a new approach to prevent cardiovascular events in patients at increased risk due to age and conditions like type 2 diabetes, prediabetes, or metabolic syndrome but without known symptomatic cardiovascular disease. The study compares a Cleerly Coronary Artery Disease (CAD) Staging System-based care strategy with standard risk factor-based care to see if the former can better reduce cardiovascular events. The Cleerly system uses imaging to visualize and quantify coronary artery disease and guides personalized treatment and education based on this assessment. The trial uses the Cleerly CAD Staging System device, which employs a proprietary algorithm to detect and stage coronary artery disease and generate a risk score to guide treatment decisions. Participants receive either this stage-based care or the usual care based on traditional risk factors. The study is prospective, randomized, and pragmatic, designed to follow patients over an average of 3.5 years to compare cardiovascular event outcomes between these two care approaches. Participants will be monitored through cardiovascular event tracking throughout the study period. Data collected includes imaging results, risk scores, and treatment adherence to evaluate the impact of the care strategies. The primary outcome is the comparison of cardiovascular event risk between the Cleerly stage-based care and risk factor-based care groups. The study also includes ongoing safety monitoring and personalized management by a cardiologist-led team via digital communication devices.

Researchers are evaluating the effectiveness of pembrolizumab combined with sacituzumab govitecan-hziy compared to the standard chemotherapy treatments in patients with locally advanced or metastatic urothelial cancer. This Phase III trial focuses on cancers that have spread to nearby tissues, lymph nodes, or other parts of the body. The study aims to compare overall survival and other outcomes such as progression-free survival, response rates, clinical benefits, duration of response, and treatment toxicity between the two treatment approaches. Quality of life and fatigue are also assessed as secondary measures. Participants are randomly assigned to one of two treatment groups. One group receives standard of care chemotherapy, which may include carboplatin or cisplatin combined with gemcitabine, or alternatively docetaxel or paclitaxel, administered intravenously in cycles every 21 days for up to six cycles, unless the disease progresses or side effects become unacceptable. The other group receives sacituzumab govitecan-hziy intravenously on days 1 and 8, along with pembrolizumab intravenously on day 1 of each 21-day cycle, continuing for up to 35 cycles or two years, unless there is disease progression or unacceptable toxicity. Throughout the study, participants undergo regular blood sample collections and imaging scans using computed tomography or magnetic resonance imaging to monitor their condition. Quality of life questionnaires are also completed to assess symptoms and fatigue over time. After treatment ends, patients are followed up 30 days later and then annually for up to five years to evaluate long-term outcomes and safety. The main outcome measured is overall survival from the time of randomization up to five years.

Researchers are evaluating the effectiveness of camizestrant compared to standard endocrine therapy in patients with early breast cancer that is estrogen receptor positive (ER+) and human epidermal growth factor receptor 2 negative (HER2-). These patients have an intermediate or high risk of cancer recurrence and have already completed local treatments such as surgery and possibly chemotherapy, alongside at least 2 years and up to 5 years of standard adjuvant endocrine therapy. The study is a Phase III, open-label trial designed to assess outcomes over a long term. Participants will be randomly assigned to receive either camizestrant, an oral selective estrogen receptor degrader, or one of several standard endocrine therapies including tamoxifen, anastrozole, letrozole, or exemestane, administered according to local approved guidelines. The treatment duration for both groups is planned to last 60 months. Eligible patients may have previously used CDK4/6 inhibitors, and the study will specifically include those with intermediate or high risk of recurrence as determined by clinical and biological markers. During the study, participants will be monitored for up to 10 years from the last patient's randomization to evaluate invasive breast cancer-free survival. Additional outcomes include invasive disease-free survival, distant relapse-free survival, overall survival, safety, and clinical outcome assessments. The study involves ongoing assessments of health status, treatment effects, and safety to determine the long-term benefits and risks of camizestrant compared to standard therapies.

Researchers are evaluating combination chemotherapy treatments for patients with newly diagnosed stage II-IV diffuse anaplastic Wilms tumors (DAWT) and relapsed favorable histology Wilms tumors (FHWT). This phase II trial aims to assess how adding vincristine and irinotecan to standard chemotherapy regimens affects event-free survival and overall survival compared to historical data. The study also explores kidney toxicity, tumor genetics, and radiation therapy techniques to reduce side effects in children with lung and liver metastases. Two chemotherapy regimens are studied. Arm I (Regimen UH-3) involves cycles of vincristine, doxorubicin, cyclophosphamide, carboplatin, etoposide, and irinotecan given intravenously on specific days every 21 days. Radiation therapy is given around week 7 of cycle 3 if needed. Arm II (Regimen ICE/Cyclo/Topo) includes cycles of ifosfamide, carboplatin, etoposide, cyclophosphamide, and topotecan given intravenously every 21 days, with surgery and/or radiation during certain cycles. Both arms include multiple cycles in absence of disease progression or unacceptable side effects. Participants undergo various scans like CT, PET, MRI, chest x-rays, ultrasounds, and bone scans throughout the trial. Blood samples and biopsies may be collected periodically. After treatment, patients are followed up every 3 months for the first 2 years, every 6 months for years 3-4, and once at year 5. The main outcomes measured are event-free survival and overall survival up to 5 years, along with monitoring kidney health and treatment effects.

This research aims to evaluate the effectiveness and safety of different dose escalation plans of retatrutide in adults who are overweight or have obesity but do not have type 2 diabetes. The study focuses on participants with specific weight-related health conditions or a history of unsuccessful weight loss attempts. This is a Phase 3b, randomized, controlled, double-blind trial designed to compare multiple dose strategies. Participants will receive retatrutide, a drug given by subcutaneous injection. The study investigates various dosing schedules to understand how different dose escalations impact safety and efficacy in managing weight. The intervention period includes dose escalation schemes to determine the optimal approach for this population. Participants will be involved for about 113 weeks, during which their body weight will be regularly monitored, with the primary measurement being the percent change from baseline to week 104. Throughout the study, safety and health parameters will be assessed to ensure participant well-being and to track the drug's effects. This long-term follow-up allows researchers to evaluate the sustained impact of retatrutide on body weight and safety over time.

Researchers are collecting baseline growth data in children with idiopathic short stature (ISS) to better understand their growth patterns. This observational study includes children diagnosed with ISS who meet specific height and growth criteria, aiming to gather detailed growth measurements and related variables over time. The study does not involve any treatment interventions but focuses on monitoring growth changes in children who may or may not be receiving human growth hormone (hGH) therapy. Participants include those who have never received hGH and those currently on treatment. The study observes growth parameters such as height, height Z-score, BMI, and BMI Z-score every six months. Participants are involved in regular assessments every six months, continuing up to 15 years. These evaluations measure annualized growth velocity, standing height, height Z-score, BMI, and BMI Z-score to track their growth progress. The study relies on ongoing monitoring to understand growth changes in children with ISS over an extended period.