Middlebury

Researchers are evaluating the safety, tolerability, and effectiveness of AP306 at fixed doses in adults with hyperphosphatemia who are receiving maintenance hemodialysis. Hyperphosphatemia is a common complication in advanced chronic kidney disease and is linked to serious risks such as cardiovascular problems, fractures, and mortality, especially in dialysis patients. This research is a randomized, double-blind, placebo-controlled Phase 2b study to better understand how AP306 affects phosphate levels in this population. Participants will receive AP306 in different fixed doses administered orally either twice or three times daily for 8 weeks, or a placebo given three times daily. The study includes seven different cohorts, six receiving various doses of AP306 and one receiving placebo. The treatment period aims to assess how these doses impact serum phosphate levels and the drugs safety and tolerability. During the study, participants will have their serum phosphate levels monitored to measure the drugs impact over the 8-week treatment period. Researchers will also assess the proportion of participants achieving target phosphate levels and the time it takes to see a response. Safety monitoring will include laboratory tests and assessments of adverse effects. The study expects full participation to span at least 8 weeks of treatment, with ongoing evaluation of tolerability and efficacy.

Researchers are conducting a combined phase 2b and phase 3 study to evaluate the drug CSL300 Clazakizumab in adults with end stage kidney disease ESKD who are undergoing dialysis. The study focuses on participants with systemic inflammation and either atherosclerotic cardiovascular disease ASCVD or diabetes. The trial aims to find the best dose of CSL300 and then assess its effects on cardiovascular outcomes and safety compared to a placebo. The study is divided into two parts Part 1 phase 2b is a dose-finding phase where different doses of CSL300 are given intravenously to participants to determine the optimal dose versus placebo. Part 2 phase 3 evaluates the efficacy and safety of the selected CSL300 dose compared to placebo over approximately five years. All treatments are administered by intravenous infusion. Participants will be involved in regular assessments including blood tests to measure inflammation markers such as high-sensitivity C-reactive protein hs-CRP, cardiovascular events tracking, and monitoring for adverse events. The phase 2b portion includes up to 24 weeks of follow-up for dose evaluation, while the phase 3 portion monitors cardiovascular outcomes like heart attacks or cardiovascular death over about five years. Safety and drug levels in the blood will also be closely observed throughout the study.

Researchers are evaluating the efficacy, safety, tolerability, and pharmacokinetics of VX-147 in adults and children aged 10 to 65 with apolipoprotein L1 APOL1-mediated proteinuric kidney disease. This condition involves kidney damage characterized by protein in the urine. The study is a combined Phase 23 adaptive, double-blind, placebo-controlled clinical trial sponsored by Vertex Pharmaceuticals Incorporated. Participants will be randomly assigned to receive different doses of VX-147 tablets or matching placebo tablets. They will take the assigned treatment for at least 96 weeks in Part A. Those who complete Part A will continue receiving VX-147 for an additional 96 weeks in Part B. The study uses a quadruple masking design to keep treatment assignments confidential. Throughout the study, participants will undergo regular assessments including urine protein to creatinine ratio UPCR and estimated glomerular filtration rate eGFR slopes to monitor kidney function. Safety is monitored by tracking adverse events and serious adverse events over several years. Blood samples will measure VX-147 levels, and treatment acceptability will be assessed in pediatric participants. Total participation may last up to approximately four years, with ongoing safety and efficacy evaluations.

Researchers are evaluating VX-407 in adults aged 18 to 65 with Autosomal Dominant Polycystic Kidney Disease ADPKD who have certain PKD1 gene variants. The study aims to assess the effect of VX-407 on height-adjusted total kidney volume htTKV, as well as its safety, tolerability, and how the body processes the drug. This is an open-label, single-arm Phase 2a trial sponsored by Vertex Pharmaceuticals Incorporated. Participants will receive VX-407 tablets orally for up to 52 weeks. There is only one treatment group in this study, and all participants will receive this investigational drug. The study includes a treatment period lasting about one year, during which researchers will monitor the effects of VX-407 on kidney volume and collect data on the drugs behavior in the body. During the study, participants will have MRI scans to measure htTKV from the start through week 52. Safety and tolerability will be tracked by noting any adverse events up to week 54. Blood samples will be taken to evaluate the drugs plasma concentration and overall exposure over time. Participants are expected to comply with scheduled visits and study procedures throughout their involvement, which lasts a little over one year.