Shenandoah

This research aims to evaluate the safety, tolerability, and impact on albuminuria of the drug MZE829 in adults who have proteinuric chronic kidney disease and carry the APOL1 high-risk genotype. This Phase 2 open-label study focuses on participants with specific genetic markers associated with kidney disease to better understand treatment effects. Participants will receive MZE829 in the form of oral capsules. The study involves monitoring the participants over a 12-week period to assess the drug's safety and how well patients tolerate it. Researchers will also measure changes in albuminuria, which reflects kidney function. During the study, participants will be closely monitored for any adverse events from the first day through week 12. Safety assessments and laboratory tests will be performed to track the drug’s effects. The main goal is to determine how safe and tolerable MZE829 is, along with its impact on kidney disease markers over the treatment duration.

Researchers are evaluating the safety and effectiveness of VIA Disc NP, a non-surgical treatment designed to supplement damaged nucleus pulposus tissue in people with lumbar discogenic pain linked to degenerative disc disease (DDD). This randomized, sham-controlled, multi-center, double-blind clinical trial includes an open-label roll-in period with one participant per site. The study focuses on adults aged 22 to 85 years who have moderate to severe DDD and chronic low-back pain that has not improved with conservative care. Participants will receive one VIA Disc NP treatment per affected disc level, up to two levels. Those enrolled after the roll-in phase will be randomly assigned in a 2:1 ratio to either the VIA Disc NP intradiscal injection or a sham procedure that mimics the injection without penetrating the disc. Participants in the sham group who continue to experience symptoms after 12 months may cross over to receive VIA Disc NP and will follow an additional 12 months of study visits. During the study, participants will undergo assessments including pain severity and disability scores, physical tests, and imaging to monitor the treatment's effects and safety. Researchers will track the proportion of participants achieving meaningful pain improvement and report any treatment-related adverse events over 12 months. Those crossing over will be monitored for an additional year, ensuring close safety follow-up and evaluation of long-term outcomes.

Researchers are conducting a two-part, phase 2b/3 study to evaluate CSL300 (Clazakizumab) in adults with end stage kidney disease (ESKD) undergoing dialysis who have systemic inflammation and either atherosclerotic cardiovascular disease (ASCVD) or diabetes. The study aims to determine the best dose of CSL300 and assess its effects on cardiovascular outcomes and safety in this population. This multicenter, randomized, double-blind, placebo-controlled trial targets patients with elevated inflammation markers and significant health risks due to their conditions. In the first part (phase 2b), the study focuses on finding the appropriate dose of CSL300 compared to placebo. CSL300 is given through intravenous (IV) administration. The second part (phase 3) evaluates the impact of CSL300 on cardiovascular events such as heart attack or cardiovascular death over approximately 5 years, continuing to compare CSL300 to placebo for safety and efficacy. The placebo matches CSL300's excipient content but lacks the active drug. Participants will undergo baseline and regular assessments for inflammation markers like high-sensitivity C-reactive protein (hs-CRP) up to 12 weeks in phase 2b, and long-term monitoring for cardiovascular outcomes in phase 3. The study involves ongoing safety evaluations and efficacy measurements during the entire follow-up period. This comprehensive approach helps researchers understand how CSL300 affects inflammation and cardiovascular health in patients with ESKD on dialysis.

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 evaluating VX-147 for its effectiveness, safety, tolerability, and how the body processes it in adults and children aged 10 to 65 who have apolipoprotein L1 (APOL1)-mediated proteinuric kidney disease. This study is a Phase 2/3 trial designed to better understand treatment options for this specific kidney condition. Participants will receive either VX-147 or a placebo, both given as oral tablets. The study is double-blind and placebo-controlled, meaning neither participants nor researchers know who receives the active drug or placebo during the treatment period. The trial consists of two parts: Part A focuses on treatment effects over at least 48 weeks, while Part B involves continued safety and tolerability observation for approximately four years after the last participant enrolls. Throughout the study, participants will undergo regular assessments including measurements of urine protein to creatinine ratio and kidney function via estimated glomerular filtration rate (eGFR). Safety is monitored by tracking adverse events and serious adverse events. Data will be collected during the treatment period and followed long-term to evaluate both efficacy and safety outcomes, with some measures assessed at interim and final analyses over at least two years.

Researchers are evaluating the Shield blood test as a screening tool for colorectal cancer (CRC) in people aged 45 to 81 who are at average risk for CRC. This study aims to assess how well the Shield test performs during a second round of testing, using colonoscopy as the standard comparison. Colorectal cancer is a common and serious disease, especially in older adults, and early detection through screening can reduce mortality by catching cancer at earlier, more treatable stages. Participants will undergo the Shield blood test as part of their standard care. The study focuses on average-risk individuals who do not have symptoms or high-risk factors for CRC. The performance of the Shield test will be monitored over a period of 33 to 42 months after enrollment to evaluate its effectiveness compared to colonoscopy results. During the study, participants will follow study procedures and standard care assessments. Researchers will measure the performance of the Shield test in detecting colorectal cancer and its precursors during the second testing interval. This includes ongoing monitoring and data collection to understand the test's accuracy and reliability in a real-world setting, with a total follow-up period extending beyond two and a half 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.