Alabaster

Researchers are evaluating the effectiveness, safety, and behavior of a new treatment called sefaxersen (RO7434656), an Antisense Oligonucleotide (ASO) therapy, for people with primary IgA nephropathy (IgAN). The study focuses on participants who have a high risk of their kidney disease worsening despite receiving the best available supportive care. This is a Phase III, randomized, double-blind, placebo-controlled trial conducted at multiple centers. Participants will receive either sefaxersen or a matching placebo through subcutaneous injections according to a specified schedule. The study compares these two groups to see how the treatment affects kidney function over time. The intervention is designed to inhibit Complement Factor B, which is involved in the disease process. The study includes vaccination requirements and contraceptive use for women of childbearing potential to ensure safety. During the study, participants will be monitored for changes in their urine protein-to-creatinine ratio (UPCR) at baseline and at week 37, which is the primary measure of kidney function improvement. Other assessments include kidney biopsy results, kidney function tests estimating glomerular filtration rate (eGFR), and ongoing safety evaluations. The trial tracks participants' health closely to assess the treatment's effect and any side effects throughout the study period.

Researchers are evaluating the effects of a medicine called BI 764198 in adults and adolescents aged 12 years and older who have a kidney condition known as focal segmental glomerulosclerosis (FSGS). This Phase 3 study aims to understand whether BI 764198 can improve kidney health in people with primary FSGS or genetic FSGS related to TRPC6 gene variants by comparing changes in urine protein levels over 104 weeks. Participants are randomly assigned to one of two groups: one group takes BI 764198 tablets once daily, and the other group takes placebo tablets that look like the medicine but contain no active drug. All participants continue their usual FSGS treatments during the study, which lasts up to two years. During the study, participants visit the study site approximately every three months. They regularly provide urine samples to monitor kidney function, and doctors check their overall health and note any side effects. The main outcome measured is the change in the 24-hour urine protein-to-creatinine ratio from the start of the study to week 104, helping researchers understand the treatment's impact on kidney disease.

Researchers are evaluating the study medicine PF-08046054 compared to the standard chemotherapy drug docetaxel in adults with non-small cell lung cancer (NSCLC) that has spread or cannot be removed with surgery or radiation. Participants must have PD-L1 expression on 1% or more of their tumor cells and have experienced cancer progression during or after treatment with PD-L1 or PD-1 inhibitors, platinum-based chemotherapy, and targeted therapies for those with known genetic mutations. The trial is a Phase 3 randomized study to better understand how well PF-08046054 works alone compared to docetaxel alone. Participants will be randomly assigned to receive either PF-08046054 or docetaxel. Those in the PF-08046054 group will get intravenous (IV) infusions twice every 21-day cycle, while those in the docetaxel group will receive one IV infusion every 21 days. The treatment period may last up to 5 years if their NSCLC responds to the therapy. No other treatments are combined during the study period. Throughout the study, participants will have regular clinic visits for evaluations and monitoring to see how they respond to the treatment. Researchers will collect information on overall survival over approximately 5 years. They will also monitor safety and disease progression during these visits to understand the long-term effects and benefits of the treatments.

Researchers are evaluating the safety and effectiveness of AZD2373 in adults aged 18 to 65 diagnosed with APOL1-Mediated Kidney Disease (AMKD) who carry specific high-risk APOL1 genotypes (G1 and G2). This Phase 2b study aims to see if AZD2373 can reduce the urine albumin-creatinine ratio (UACR) more than a placebo by the 30th week of treatment. Participants must have significant kidney involvement as indicated by UACR and estimated glomerular filtration rate (eGFR) levels. The study is designed as a randomized, double-blind, placebo-controlled trial with multiple treatment groups. The study includes three treatment arms: two different doses of AZD2373 and a placebo, all delivered via accessorized pre-filled syringes as injections. Participants will be randomly assigned to one of these groups and neither they nor the study staff will know their assignment during the trial. Treatment will last for a minimum of 30 weeks, continuing until the last participant completes this period. The study also uses a specialized APOL1 genotyping test to confirm participants' eligibility based on their genetic profile. Participants will undergo screening with urine tests to confirm UACR levels and blood tests for kidney function before joining. During the study, researchers will monitor changes in UACR from baseline to week 30 to assess treatment effects. Safety and tolerability will also be closely observed throughout the treatment period. Around 96 participants will be enrolled, with about 32 in each group, and all will be followed until the last participant completes the 30 weeks of treatment.

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 VX-407 in adults aged 18 to 65 who have Autosomal Dominant Polycystic Kidney Disease (ADPKD) with specific PKD1 gene variants. This Phase 2a open-label study focuses on how VX-407 affects height-adjusted total kidney volume (htTKV), along with its safety, tolerability, and how the body processes the drug (pharmacokinetics). Participants will receive VX-407 tablets taken orally during the study. The trial involves monitoring changes in htTKV via MRI scans over a period from baseline up to 52 weeks. The study is designed as a single-arm trial without a comparison group. During the study, participants will undergo scheduled visits including abdominal MRI scans to measure kidney volume and assessments of kidney function through estimated glomerular filtration rate (eGFR). Researchers will track safety and collect pharmacokinetic data to understand drug behavior. The total study duration for each participant is about one year, with ongoing evaluations to monitor treatment effects and safety.

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.