Ranica

Researchers are evaluating the effects of the drug zigakibart on the progression of Immunoglobulin A Nephropathy IgAN, a kidney disease. This open-label, multicenter study will randomize participants into two groups that differ only by the timing of an on-treatment kidney biopsy, either at the end of the first or second year of treatment. The study aims to understand how zigakibart influences disease markers over time. Participants will receive subcutaneous injections of zigakibart at a dose of 600 mg every two weeks for up to two years 104 weeks. The study includes a maximum screening period of 8 weeks, followed by the 104-week treatment period, and concludes with a 13-week safety follow-up. The two groups differ in the timing of kidney biopsies taken to assess treatment effects either at week 53 or week 105. During the study, participants will undergo various assessments including measurement of kidney function, protein levels in urine, and immune markers such as mesangial IgA deposition and complement components. Safety will be monitored through tracking of adverse events throughout treatment and follow-up. Blood samples will be collected at multiple time points to measure drug levels and antibodies against the drug. The total participation time can be up to approximately 125 weeks.

Researchers are studying felzartamab in adults with Immunoglobulin A nephropathy IgAN, a kidney disease caused by abnormal IgA antibodies building up in the kidneys leading to inflammation and damage. This Phase 3 clinical trial aims to understand how felzartamab affects proteinuria, the presence of protein in urine, and kidney function in people with IgAN. The safety and how the body processes felzartamab are also being evaluated. Participants will be randomly assigned to receive either felzartamab or a placebo through intravenous infusions during a 24-week treatment period. Some participants with lower kidney filtration rates will be grouped separately but also receive either felzartamab or placebo. After treatment, participants will enter an 80-week follow-up phase. In total, participants will have 17 study visits over about two years. Throughout the study, participants will have urine tests to measure proteinuria, blood tests to assess kidney filtration function, and monitoring for side effects. Researchers will also study felzartamab levels in the blood and check for immune reactions against the drug. Safety will be closely monitored using vital signs, laboratory tests, and physical exams during the entire 104-week period.

Researchers are tracking the course of primary membranous nephropathy PMN, a rare autoimmune kidney disease that causes nephrotic syndrome in adults. The study focuses on understanding long-term outcomes for patients in real-world clinical practice, especially in light of new treatment options such as monoclonal antibodies. The goal is to gain insights into disease progression and response to these therapies over time. Participants will be observed without any experimental treatment assigned by the study. The focus is on patients diagnosed with PMN and nephrotic syndrome who have been treated with rituximab or other monoclonal antibodies as part of their care. Researchers will collect data from routine clinical visits and follow patients longitudinally to capture disease remission, progression, and treatment effects. During the study, participants will provide consent for their medical data to be used for research. Researchers will monitor disease remission and progression, kidney function decline, blood parameters, and any adverse events related to treatment throughout the study, which typically spans about six months on average. The study will help understand outcomes and predictors in patients managed with current therapies and support better future care.

Researchers are studying idiopathic nephrotic syndrome INS, which includes minimal change disease MCD and focal segmental glomerulosclerosis FSGS, and comparing it to primary membranous nephropathy MN. The goal is to evaluate the levels of circulating anti-nephrin autoantibodies, which may play a role in INS and post-transplant FSGS relapse. INS is a serious kidney condition with unclear causes and limited treatment options, especially when it recurs after transplantation. The study involves testing stored blood samples from patients with biopsy-confirmed idiopathic MCD or FSGS and those with membranous nephropathy. These samples will be analyzed using specialized ELISA tests to measure anti-nephrin autoantibody levels. The tests use recombinant nephrin protein and antibodies to detect these autoantibodies in the blood, comparing patients with INS to those with MN. Participants have previously provided consent for their samples to be stored in a certified biobank. Researchers will analyze these samples retrospectively over about one year to measure antibody levels. The study focuses on laboratory analysis without direct patient involvement, aiming to better understand the potential role of anti-nephrin autoantibodies in nephrotic syndrome and its recurrence after kidney transplant.

Researchers are investigating membranous nephropathy MN, a common cause of nephrotic syndrome in adults, focusing on the role of autoreactive B cells that produce antibodies against the PLA2R receptor. The study aims to understand why some patients respond to B-cell depleting treatments while others do not, by comparing circulating immune cells in responders, non-responders, and healthy controls. This observational study will help identify differences in immune cell behavior related to treatment outcomes. The study includes patients with biopsy-confirmed idiopathic MN who are either planned to receive or have already received B-cell depleting therapy, as well as healthy volunteers without significant illnesses. Researchers will analyze blood samples using biochemical and flow cytometry assays to measure the frequency and activity of various B-cell subsets and other immune cells over a 24-month period. This includes assessments at baseline and multiple follow-up points at 3, 6, 9, 12, and 24 months. Participants will provide blood specimens that will be tested to evaluate the presence and function of autoreactive B cells and other immune cell types. The study will track changes in these immune cells over time, correlating findings with treatment response. The research involves no interventional treatments but focuses on extensive immune monitoring to better understand MN disease mechanisms and predict outcomes. Participation duration varies, with multiple follow-up visits for sample collection and immune profiling.

Researchers are exploring how RNA sequencing RNA-Seq can help diagnose rare genetic diseases in children and adults who show early symptoms but have negative results from previous DNA tests called whole-exome sequencing WES. This project aims to set up and validate RNA-Seq techniques using healthy volunteers and patients with known genetic changes affecting RNA. The study focuses on identifying RNA changes in skin cells and comparing these to blood samples to better understand diseases that are hard to diagnose. Participants include healthy adults, patients with diagnosed genetic diseases affecting RNA, and undiagnosed patients suspected of having rare genetic conditions. Skin biopsies will be taken to culture fibroblasts, which are then analyzed using RNA-Seq to detect RNA level and splicing alterations. Blood samples will also be collected to study genetic and RNA changes. The study includes a discovery group with patients whose WES results were inconclusive, aiming to use RNA-Seq as an additional diagnostic tool. Participants will undergo skin biopsies and blood collection, and their samples will be stored in a biobank for analysis. Researchers will assess RNA changes in fibroblasts and compare RNA profiles from skin and blood. The main outcomes measure the setup and validation of RNA analysis methods and the detection of RNA alterations in patients. Participants will provide consent before enrollment, and their involvement includes sample collection and clinical information gathering. The study is expected to continue through June 2026.

Researchers are evaluating the safety and effectiveness of switching from anti-C5 antibody treatment to iptacopan in adults diagnosed with atypical hemolytic uremic syndrome aHUS. This Phase 3, multicenter, single-arm, open-label study aims to provide important data on how iptacopan works in patients who have already responded to anti-C5 antibody treatment. The study is sponsored by Novartis Pharmaceuticals and focuses on key measures related to aHUS symptoms and kidney function. Participants will receive iptacopan at a dose of 200 mg twice daily during the treatment periods. The study includes an initial screening period lasting up to 14 weeks, followed by a 12-month core treatment phase and then a 12-month extension treatment phase. This design allows researchers to observe the effects of iptacopan over a full two years after switching from previous therapy. Throughout the study, participants will undergo regular assessments including blood tests for platelet count, LDH, hemoglobin, kidney function markers such as serum creatinine and eGFR, and urine protein levels. Researchers will monitor for signs of thrombotic microangiopathy TMA and kidney disease progression, as well as track the need for dialysis. Safety and clinical responses will be evaluated at multiple time points to understand the treatment impact and participant well-being.

Atypical Hemolytic Uremic Syndrome aHUS is a rare disease that causes destruction of red blood cells, low platelet counts, and blockage of small blood vessels, especially affecting the kidneys. Unlike the typical form caused by E. coli infection, aHUS may be linked to genetic mutations affecting the immune systems complement pathway or other genes like DGKE. This study aims to better understand the function of these rare genetic mutations in aHUS and explore potential personalized treatments. The study will include 110 children and adults diagnosed with aHUS who carry mutations in either the MCP or DGKE genes, along with 2 healthy volunteers as controls. Participants will provide blood and urine samples for laboratory analysis. Researchers will create patient-specific stem cells and turn them into endothelial cells to study their characteristics and viability. This process will help reveal differences between patients and healthy individuals. During the study, participants will undergo a single blood draw and urine test. Researchers will assess cell generation, differentiation, and function from the collected samples. The primary outcomes include the successful creation and detailed study of patient-derived cells. The study will monitor these laboratory measures once during the trial to better understand the disease mechanisms and identify possible drug targets. Participation length varies with no additional treatment given.

Researchers are evaluating the long-term effects of iptacopan in people with C3 glomerulopathy C3G or idiopathic immune-complex-membranoproliferative glomerulonephritis IC-MPGN. This open-label extension study focuses on assessing the safety, tolerability, and sustained efficacy of iptacopan over an extended period, following participants who completed earlier phase 2 or phase 3 studies. The study aims to provide important data to support regulatory submissions and continued access to treatment. Participants receive iptacopan capsules at a dose of 200 mg twice daily. The study includes different groups based on prior study participation and kidney status, including those with native kidneys and kidney transplants, as well as participants from core phase 3 studies who were randomized to placebo or iptacopan. This extension study is planned to continue until iptacopan becomes commercially available, the benefit-risk profile changes, or the program is discontinued, potentially lasting up to approximately 168 months. During the study, participants undergo regular efficacy and safety assessments, including monitoring of renal endpoints, adverse events, vital signs, electrocardiograms, and laboratory tests. Data collected includes urine protein and albumin levels, serum creatinine, estimated glomerular filtration rate, and complement C3 levels. The study expects participants to remain for a minimum of 60 months and up to 84 months, with the goal of understanding long-term treatment effects and tolerability.

Researchers are conducting an observational study to develop and test new advanced ultrasound imaging protocols focused on vascular health in the arm and brain. This study aims to establish normal reference values for ultrasound parameters, assess measurement consistency, and explore correlations between these measurements and factors like age and gender. It is performed exclusively for research purposes using a state-of-the-art ultrasound machine called Resona 9. The study uses the Resona 9 ultrasound system equipped with innovative features to capture detailed images and 3D models of blood vessels in the non-dominant arm and neck carotid arteries. Sixty adult participants, divided by gender and age groups, will undergo ultrasound examinations at enrollment, with consent sought for possible future repeat scans. This approach allows researchers to refine imaging protocols and collect data for comparison with pathological cases. Participants will visit once for ultrasound scans of the specified vascular regions, during which multiple measurements will be taken to evaluate the new protocols. The study will monitor the reproducibility of these measurements and analyze how age and gender relate to vascular imaging results. Participation involves non-invasive imaging procedures without treatment or medication, and subjects may be asked to return for additional scans if needed. The study is expected to run until March 2027.