Little River

Researchers are evaluating the effect of ION775 on fasting triglyceride levels in adults with hypertriglyceridemia and severe hypertriglyceridemia. This phase 2, open-label study aims to assess the safety, tolerability, and efficacy of ION775 in reducing triglyceride levels in these participants. The study is sponsored by Ionis Pharmaceuticals, Inc. Participants will be randomly assigned to one of three groups receiving ION775 by subcutaneous injection. The study includes a 4 to 6 week screening period, followed by a 6-month treatment period where participants receive ION775, and then a 6-month post-treatment follow-up period to monitor outcomes and safety. The study measures changes in fasting triglycerides and other cholesterol-related markers. During the study, participants will have assessments at baseline and at month 6 to measure triglyceride levels and other cholesterol components such as apolipoprotein C-III, very low-density lipoprotein cholesterol, remnant cholesterol, and non-high-density lipoprotein cholesterol. Safety is monitored through treatment-emergent adverse events and laboratory evaluations up to 12 months. Participants are involved in regular visits during the treatment and follow-up periods to evaluate the effects and safety of ION775.

Researchers are conducting a multicenter, randomized, double-blind Phase 2 study to evaluate the pharmacodynamics, safety, and tolerability of a combination of QCZ484 and inclisiran compared to QCZ484 alone, inclisiran alone, and placebo in adults with hypertension and hypercholesterolemia. The study focuses on understanding how these treatments affect certain blood markers and blood pressure in this population. Participants receive a single dose of either the combination of QCZ484 and inclisiran, QCZ484 alone, inclisiran alone, or placebo on Day 1. The study lasts up to 12 months and includes a screening period with two visits to confirm eligibility, followed by the treatment administration at the baselinerandomization visit. After treatment, participants enter a safety follow-up phase, with an option to join an open-label extension study instead of the follow-up. During the study, participants undergo various assessments including measurements of PCSK9 and AGT levels at baseline and Month 3, blood pressure and LDL cholesterol levels at baseline, Month 3, and Month 6, along with monitoring for treatment-emergent adverse events and changes in laboratory tests and vital signs for up to 12 months. These evaluations help researchers understand the effects and safety of the treatments over time.

Researchers are evaluating the effectiveness, safety, and tolerability of a vaccine designed to reduce Clostridioides difficile C. difficile infections in adults aged 65 years and older. This phase 3 study compares the vaccine to a placebo in a group of older adults who have recent or planned contact with healthcare systems or recent antibiotic use. The purpose is to understand how well the vaccine works to prevent infections and to monitor any side effects or reactions. Participants will receive either the C. difficile vaccine or a placebo shot injected into the upper arm muscle. The study is randomized, double-blinded, and placebo-controlled. The vaccination period includes two doses and participants will be monitored for up to about three and a half years. Follow-up includes three planned clinical visits and three phone visits initially, then yearly clinic visits until the study ends. Participants are asked to report and save stool samples if they experience three or more loose stools in 24 hours to check for possible infection. During the study, researchers will track local and systemic reactions to the vaccine within seven days after each shot, adverse events up to one month after each vaccination, and serious adverse events for up to 18 months after the last dose. The main measurement is the occurrence of medically attended primary C. difficile infections from two weeks after the second vaccination through the surveillance period. Safety monitoring and annual visits will continue until the study is completed, which could be sooner or later depending on infection rates.

Researchers are evaluating the effect of muvalaplin in lowering cardiovascular risks among adults with elevated lipoproteina who either have atherosclerotic cardiovascular disease or are at risk of a first heart attack or stroke. This phase 3, randomized, double-blind study aims to investigate whether muvalaplin can reduce major adverse cardiovascular events compared to placebo in this high-risk population. Participants are randomly assigned to receive either muvalaplin or a placebo, both given orally. The study is designed with parallel groups and will last about 5.25 years, during which the occurrence of cardiovascular events and changes in lipoproteina levels will be closely monitored. Throughout the study, participants will undergo regular assessments including measurement of lipoproteina levels, monitoring of cardiovascular events such as heart attacks or strokes, and evaluation of healthcare resource use. The primary outcome is the time to first major adverse cardiac event, tracked from baseline until the study ends. Safety and pharmacokinetics of muvalaplin will also be evaluated during the trial period.

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 olpasiran, compared to a placebo, to see how it affects the risk of coronary heart disease death, heart attacks, or urgent coronary revascularization in people at risk for their first major cardiovascular event who have high levels of lipoproteina. This Phase 3 study focuses on participants aged 50 to 105 years with multiple cardiovascular risk factors or evidence of atherosclerosis. Participants will be randomly assigned to receive either olpasiran or a placebo through subcutaneous injections. The study is double-blind, so neither participants nor researchers know who receives the active drug or placebo. Treatments and evaluations will continue for up to approximately 6.2 years. Throughout the study, participants will be monitored for heart-related events such as heart attacks, cardiovascular death, strokes, and coronary revascularizations. Researchers will also measure changes in lipoproteina levels at baseline and at Week 48, along with tracking adverse events. The total participation duration can extend up to about 6.2 years, with ongoing assessments to evaluate the treatments effect over time.

Researchers are evaluating the effect of MAR001 compared to a placebo in adults with elevated triglycerides TG and remnant cholesterol RC. This Phase 2b randomized, double-blind, placebo-controlled study aims to understand how MAR001 influences TG levels in participants with these conditions. The study includes two parts with different participant groups based on their baseline TG levels. Participants are assigned randomly to receive either MAR001 or placebo through subcutaneous injections every 4 weeks. Part A includes about 216 participants with baseline TG between 150 to 880 mgdL, treated with three different doses of MAR001 or placebo at a 31 ratio. Part B enrolls around 100 participants with baseline TG between 450 and 2000 mgdL, using the dose selected from Part A data, assigned at a 11 ratio. The study timeline consists of up to 8 weeks of screening, a 24-week treatment period, and a 12-week safety follow-up with clinic visits at weeks 28 and 36. During the study, participants will undergo assessments including blood tests and clinical evaluations to monitor TG and RC levels and safety outcomes. The primary outcome is measured at 12 weeks, with additional secondary outcomes assessed at various time points. Participants adherence to stable diet and medication regimens is monitored. The total participation duration extends through the screening, treatment, and follow-up periods, with safety monitored until study completion.