Chubbuck

Researchers are investigating the long-term safety and tolerability of open-label iptacopan in adults with primary IgA nephropathy who have previously completed specific clinical trials (CLNP023X2203 or CLNP023A2301). This extension study is designed to allow participants continued access to iptacopan until certain conditions are met, such as reaching three years from the last patient first visit, loss of treatment benefit, negative benefit-risk profile, initiation of dialysis or kidney transplant, or commercial availability of the drug. The study will also assess the drug's effects on disease progression every six months. Participants who completed the prior trials and meet inclusion criteria may receive oral iptacopan capsules at a dose of 200 mg twice daily. The study is open-label and non-randomized and will continue treatment under this regimen until one of the study-defined stopping points is reached. Supportive care with ACE inhibitors or ARBs is maintained as per clinical guidelines, and vaccination against certain infections is required before enrollment. During the study, participants will be monitored for safety, including serious adverse events, adverse events of special interest, vital sign abnormalities, ECG changes, and laboratory test abnormalities from the first day of treatment until seven days after the last dose. Efficacy assessments occur every six months to evaluate clinical effects on disease progression. The study aims to collect long-term safety and tolerability data while providing ongoing treatment access until the drug becomes commercially available or other stopping criteria apply.

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 effectiveness and safety of veverimer in adults with moderate-to-severe chronic kidney disease (CKD) and metabolic acidosis over a 26-week period. This Phase 3, randomized, double-blind study aims to compare veverimer to a placebo in improving health outcomes related to this condition. The study focuses on changes in serum bicarbonate levels and physical performance as key measures of treatment impact. Participants are randomly assigned to one of two groups: one receiving 9 grams of veverimer twice daily, and the other receiving a placebo twice daily. The study involves a treatment period lasting 26 weeks, during which participants take their assigned medication regularly. The study design ensures that neither the participants nor the researchers know which treatment is being given to maintain objectivity. During the study, participants undergo several assessments including blood tests to measure serum bicarbonate concentration and physical performance tests like the Sit-to-Stand 5 times test. These evaluations occur from the screening visit through Day 168. Researchers monitor adherence to the treatment and any side effects to ensure participant safety and to measure the effectiveness of veverimer compared to placebo throughout the study duration.

Alport syndrome (AS) is a rare genetic disorder caused by changes in specific genes that produce collagen, leading to kidney disease, hearing loss, and eye abnormalities. People with AS are at high risk of developing chronic kidney disease, where the kidneys gradually lose their function, often marked by excess protein in the urine called proteinuria. This study focuses on evaluating BAY 3401016, a monoclonal antibody designed to block the protein Semaphorin 3A, which may contribute to kidney damage in AS, aiming to slow kidney function loss in adults with rapidly progressing AS. Participants will receive either BAY 3401016 or a placebo in a randomized, double-blind, parallel group Phase 2a study. The study includes an extension phase to further assess the treatment's effects. The treatment duration covers at least 24 weeks with follow-up visits extending up to 90 days after the end of treatment. The study measures the urinary albumin creatinine ratio (UACR) over weeks 16, 20, and 24 to evaluate kidney function. During the study, participants will undergo regular assessments including kidney function tests and urine measurements to monitor protein levels. Safety and efficacy will be tracked throughout treatment and follow-up. The study includes adults aged 18 to 45 with specific criteria related to kidney function and proteinuria levels. Overall participation spans the treatment period plus a 90-day post-treatment follow-up, with researchers closely observing changes in kidney health and safety outcomes.

Researchers are evaluating the safety and effectiveness of up to two injections of REACT/rilparencel in adults with type 2 diabetes mellitus and chronic kidney disease. This phase 3 randomized controlled study divides participants into two groups to compare the effects of the actual treatment versus sham procedures mimicking kidney biopsy and injections. The goal is to monitor kidney function and clinical outcomes over time to understand the impact of this therapy on disease progression. Participants are randomly assigned before a kidney biopsy to either receive sham procedures or the real treatment involving a kidney biopsy followed by two rilparencel injections about 12 weeks apart, each into different kidneys. Those receiving sham procedures will undergo similar-sounding and looking activities without actual tissue removal or injection. All participants will be followed until the study's global end date, ensuring consistent long-term observation. During the study, participants will undergo kidney biopsies or sham procedures, followed by injections or sham injections. Researchers will assess kidney function by measuring the slope of estimated glomerular filtration rate (eGFR) over 18 months after the 135th participant's first injection or sham procedure. They will also track clinical events such as significant kidney function decline, need for dialysis or transplant, or renal and cardiovascular deaths for up to 94 months. Safety and efficacy will be monitored throughout the study to evaluate treatment impact.

Researchers are investigating the effects of iptacopan treatment on patients with IgA nephropathy (IgAN), a kidney disease. This Phase 2 study focuses on changes in immune system activity and kidney pathology, particularly looking at the reduction of complement C3c fragments in kidney tissue, which signal disease activity. The study also evaluates various biomarkers related to kidney function, damage, and disease progression, including the Oxford MEST-C score, to better understand both systemic and kidney-specific impacts of the treatment over 9 months. Participants undergo a screening period followed by a baseline visit with a kidney biopsy to confirm diagnosis and assess disease status. Eligible patients receive oral iptacopan capsules at a dose of 200 mg twice daily for 9 months without dose changes. At the end of the treatment period, a follow-up kidney biopsy is performed to evaluate structural changes. The study involves a single-arm design where all participants receive the investigational drug alongside their standard supportive care. During the trial, participants will have clinical assessments including kidney biopsies at baseline and month 9, laboratory tests to monitor kidney function and disease markers, and will be monitored for safety and treatment adherence. The primary outcome measures the proportion of patients showing at least a tenfold reduction in mesangial C3c deposits in kidney tissue after 9 months. The study collects data on kidney structure and function changes, clinical biomarkers, and adverse events to understand the treatment's effects and safety profile.