San Luis

Researchers are evaluating the clinical efficacy, safety, and tolerability of XEN1101 as an additional treatment for people with focal-onset seizures in a Phase 3 randomized, double-blind, placebo-controlled study. This trial aims to compare two doses of XEN1101 with a placebo to see how well the medication can reduce seizure frequency in patients who continue their current antiseizure medications. The study involves adults diagnosed with focal epilepsy who have tried at least two antiseizure medicines without achieving seizure freedom. About 360 participants will be randomly assigned to receive either 25 mg or 15 mg of XEN1101 or a placebo once daily with an evening meal. The study includes up to 9.5 weeks of baseline monitoring to track seizure frequency followed by 12 weeks of blinded treatment. Participants maintaining the study drug can then join an open-label extension to continue treatment or enter an 8-week follow-up after treatment ends. Throughout the study, participants will keep accurate seizure diaries and continue their stable antiseizure medications. Researchers will measure the median percentage change in seizure frequency from baseline through the 12-week treatment period, along with secondary outcomes like the proportion of participants with at least a 50% reduction in seizures and patient-reported improvement. Safety will be monitored from screening until 56 days after the last dose. Overall, participants are involved for the baseline, treatment, and follow-up phases lasting several months.

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 problems. People with AS face a high risk of chronic kidney disease CKD, which gradually reduces kidney function and can lead to end-stage kidney disease. A common sign of worsening kidney function is proteinuria, the presence of excess protein in the urine. This study evaluates the effects of BAY 3401016, a monoclonal antibody designed to block the protein Semaphorin 3A Sema3A, which may contribute to kidney damage in AS. Participants are randomly assigned to receive either BAY 3401016 or a placebo once weekly for 24 weeks, alongside their background therapy. The study includes an extension phase to further assess the treatments safety and efficacy. Participants will be involved for at least 24 weeks of treatment, followed by a 90-day follow-up period. Researchers will monitor kidney function by measuring the urinary albumin creatinine ratio UACR at several points during treatment and after its completion. Safety and tolerability of BAY 3401016 will also be carefully assessed throughout the study period.

Researchers are studying children and young adults aged 1 to 18 years with chronic kidney disease CKD and proteinuria, a condition where the kidneys leak protein into the urine. This study aims to evaluate the long-term safety of finerenone when added to standard treatments called ACE inhibitors or angiotensin receptor blockers ARBs, which are commonly used to control blood pressure and protect kidney function. The research also seeks to understand how well finerenone can reduce protein levels in urine and support kidney health over time. Participants will receive finerenone in doses adjusted by age and body weight, taken orally for up to 18 months alongside their usual ACEI or ARB treatment. The study includes patients who previously took part in a related trial and will follow them for about 19 months, including a one-month follow-up after treatment ends. The research involves one group receiving finerenone openly without placebo or comparison groups. During the study, participants will attend at least 8 to 12 visits depending on their treatment start status. At these visits, doctors will measure vital signs like blood pressure, heart rate, weight, and height perform physical exams collect blood and urine samples to monitor kidney function and protein levels and conduct heart tests using electrocardiograms and echocardiography. Participants and their caregivers will also answer questions about medication use, side effects, and overall well-being. Safety will be closely monitored by tracking any medical problems that arise during the trial.

Researchers are investigating a new treatment approach for children with chronic kidney disease CKD and proteinuria, conditions that affect kidney function and cause protein leakage into the urine. The study focuses on whether adding a drug called finerenone to existing treatments with angiotensin-converting enzyme inhibitors ACEI or angiotensin receptor blockers ARB can better control kidney problems related to overactivity of a system that regulates blood pressure and fluid balance. This Phase 3 study aims to see if finerenone can reduce protein levels in the urine more effectively than a placebo. Participants in this trial will receive either finerenone or a placebo alongside their usual ACEI or ARB medication. The study treatment lasts about 180 days, with doses adjusted for age and body weight. Before starting treatment, children must pass screening visits to confirm eligibility. During treatment, participants will attend at least seven visits where various health checks, blood and urine tests, heart exams, and questionnaires about medication experience and side effects will be performed. Throughout the study, researchers will monitor kidney function, electrolyte levels, and how the body processes finerenone. They will also track any medical problems participants experience. After completing treatment, participants will have a follow-up visit about 30 days later to assess their health. The main measure of success is the change in the urinary protein-to-creatinine ratio from before treatment to about six months later, helping to understand the treatments impact on proteinuria.

This study evaluates the long-term safety and tolerability of pelacarsen TQJ230 in people with established cardiovascular disease and elevated Lipoproteina who completed a previous related study. It is an open-label extension trial, meaning all participants receive the study drug without placebo comparison. The trial is sponsored by Novartis Pharmaceuticals and focuses on continued treatment after the completion of the parent study. Participants receive monthly injections of pelacarsen 80 mg subcutaneously for up to 36 months during this extension phase. This phase is designed to provide access to the study drug after the initial trial and to monitor participants closely. The study does not involve randomization or blinding, and all enrolled participants receive the active drug. During the study, participants will undergo regular assessments including monitoring for adverse events and cardiovascular events, as well as measuring Lipoproteina levels at baseline and several time points over 36 months. Safety and tolerability will be closely tracked throughout the treatment period. The total duration of participation corresponds to the length of the extension phase, up to three years.

Researchers are evaluating the efficacy, safety, and pharmacokinetics of INM004 in children with Hemolytic Uremic Syndrome caused by Shiga toxin-producing Escherichia coli STEC-HUS. This study aims to assess how well INM004, when added to standard care, improves kidney function and reduces complications, mortality, and hospital stay in affected pediatric patients. Participants will be randomly assigned to receive either two doses of INM004, an Anti-Shiga Toxin Hyperimmune Equine Immunoglobulin fragment, or two doses of a placebo solution. Each dose is given intravenously 24 hours apart, with infusion times adjusted based on body mass index. The study includes a 28-day acute phase and further assessments up to 90 days to monitor recovery and other health outcomes. During the study, children will undergo evaluations of kidney function, blood tests for markers of disease activity, and safety assessments. Researchers will measure the time to kidney function recovery as the primary outcome, along with mortality rates, dialysis needs, and hospital stay length. The trial lasts for at least 90 days, including close monitoring of health status and medication effects throughout this period.

Researchers are tracking patients with Fabry disease through an ongoing international observational program called the Fabry Registry. This registry collects routine clinical outcomes for patients regardless of whether they are receiving treatment. The study aims to better understand the diseases variability, progression, and natural history, including in women who carry one copy of the gene, and to help improve patient care by developing monitoring recommendations and reporting outcomes. Additionally, the registry evaluates the long-term safety and effectiveness of Fabrazyme4, a treatment used in Fabry disease. The registry includes a special pregnancy sub-registry for women with Fabry disease who are pregnant or have been pregnant. This sub-registry observes pregnancy outcomes and infant growth up to 36 months after birth, collecting medical and obstetric history and treatment details. No experimental treatments are given participants continue to receive their usual care as determined by their physicians. Data from both registries support regulatory requirements and ongoing research. Participants undergo regular clinical assessments and receive standard care from their doctors throughout the study. The research team collects data on disease progression, treatment effectiveness, pregnancy outcomes, and infant development. The study is observational, meaning no study drugs or procedures are administered. The total participation can last up to 33 years, allowing for long-term monitoring of safety and outcomes related to Fabry disease and pregnancy.

Researchers are evaluating the efficacy and safety of the drug levosimendan, taken orally, compared to a placebo in adults with pulmonary hypertension associated with heart failure with preserved left ventricular ejection fraction PH-HFpEF. The main goal is to measure the change in the 6-Minute Walk Distance, which reflects exercise capacity. This phase 3, randomized, double-blind study aims to improve understanding of treatment options for this condition. About 540 participants will be randomly assigned in a 21 ratio to receive either oral levosimendan or a matching placebo. Those who complete the initial study period of 26 weeks may be eligible to join an open-label extension lasting 52 weeks, during which all participants can receive levosimendan. The study carefully monitors participants throughout both phases to assess the effects of the treatment. Participants will undergo various assessments including the 6-Minute Walk Distance test, questionnaires like the Kansas City Cardiomyopathy Questionnaire to evaluate health status, and classification of physical ability using the New York Heart Association scale. Screening includes heart catheterization, echocardiograms, ambulatory heart rhythm monitoring, and exercise testing. Safety and clinical worsening events will be tracked. The total involvement may last beyond a year for those in the extension phase, ensuring thorough evaluation of outcomes and safety.

Researchers are evaluating an organ dysfunction score adapted specifically for pregnant and early postpartum patients up to 3 days after birth admitted to intensive care units ICU. This observational study aims to determine if this adjusted score, called SOFA-OBS, better predicts ICU mortality compared to the general SOFA score. The study also assesses whether a non-invasive pulse oximeter can effectively evaluate respiratory function instead of the standard arterial blood gas test, which is more painful and invasive. The study involves following pregnant and postpartum patients admitted to the ICU for at least 24 hours. Researchers will collect routine clinical data and laboratory results already used in ICU care, without requiring extra interventions. The SOFA-OBS score adjusts kidney function and blood pressure measurements to reflect the physiological changes during pregnancy and postpartum. It also replaces arterial blood gas oxygen measurements with pulse oximetry when blood gas analysis is unavailable. Data will be recorded daily during the ICU stay. Participants will be monitored for organ dysfunction and mortality outcomes during their ICU stay or up to 28 days after enrollment. Researchers will evaluate and compare the predictive ability of the SOFA-OBS and general SOFA scores for ICU mortality and sepsis-related death. Data quality will be carefully checked, and privacy will be protected by anonymizing patient information. The study is approved by ethical boards and requires informed consent from participants, with a planned enrollment of 130 patients.

Researchers are evaluating iptacopan LNP023 alongside standard care to study its effectiveness, safety, and tolerability in adults with active lupus nephritis Class III-IV, with or without Class V. This phase 2 trial is randomized, double-blind, and placebo-controlled, aiming to explore dosing and treatment outcomes in this patient group. Participants receive either iptacopan combined with standard care, placebo matching iptacopan with standard care, or iptacopan with placebo as part of the standard care. Each treatment is taken for 52 weeks. The study proceeds in two parts, with different combination regimens of iptacopan, placebo, and standard care, including oral corticosteroids or their absence. During the study, participants undergo assessments at baseline and weeks 24 and 52 to measure kidney response, protein levels in urine, fatigue, and lupus disease activity using specific scoring tools. Researchers monitor the proportion of patients achieving complete renal response without flares and other kidney and health outcomes. Safety and tolerability are also evaluated over the course of treatment and follow-up.