San Luis

Researchers are conducting the X-TOLE3 Phase 3 clinical trial to evaluate the safety, tolerability, and effectiveness of XEN1101 as an additional treatment for adults with focal-onset seizures. The study focuses on measuring changes in seizure frequency when XEN1101 is added to existing antiseizure medications compared to placebo. Participants must have a confirmed diagnosis of focal epilepsy and have tried at least two antiseizure medications without achieving seizure freedom. About 360 participants will be randomly assigned in equal groups to receive either XEN1101 at 25 mg, 15 mg, or a placebo. The study includes up to 9.5 weeks of baseline observation to record seizure frequency, followed by 12 weeks of double-blind treatment where participants take the assigned capsules once daily with an evening meal. Those who complete this period may join a separate open-label extension to continue XEN1101 treatment, while others will enter an 8-week follow-up after treatment ends. During the study, participants will maintain accurate seizure diaries and continue stable doses of 1 to 3 antiseizure medications. Researchers will monitor seizure frequency changes from baseline through the 12-week treatment. Safety and tolerability will also be assessed throughout the trial. The total participation includes baseline, treatment, and follow-up periods to ensure thorough evaluation of the treatment's impact.

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. The study aims to understand the safety of a treatment called finerenone when used together with standard medicines called ACE inhibitors or angiotensin receptor blockers (ARBs). These medicines help control kidney function and blood pressure by targeting a system called the renin-angiotensin-aldosterone system (RAAS), which is often overactive in CKD. Finerenone may help control this system more effectively alongside ACEI or ARB. Participants will receive finerenone in doses adjusted by age and body weight for up to 18 months. The study is open-label and single-arm, meaning all participants receive the treatment. Some participants already took finerenone in a previous study and will continue, while others will start anew. The treatment period lasts about 540 days with a 1-month follow-up after the last dose. Visits are planned at least 12 times for new finerenone users and 8 times for continuing users. During visits, participants will have their blood pressure, heart rate, temperature, height, and weight measured. Blood and urine samples will be collected to monitor kidney function and protein levels. Heart function will be checked using electrocardiograms and echocardiography. Participants and their guardians will answer questions about medication use, side effects, and well-being. Researchers will track any medical problems during the study and check health about 30 days after treatment ends. The main focus is safety, including monitoring adverse events, potassium levels, and blood pressure changes over about 19 months.

Researchers are investigating a new treatment approach for children aged 6 months to less than 18 years who have chronic kidney disease (CKD) and proteinuria, a condition where the kidneys leak protein into the urine. CKD causes the kidneys to work less effectively, leading to waste buildup and high blood pressure. Current treatments include ACE inhibitors (ACEI) or angiotensin receptor blockers (ARB), which help regulate a system involved in blood pressure and kidney function. However, these treatments do not work for all patients. This study is focused on seeing if adding finerenone to ACEI or ARB can better control this system and improve kidney function. Participants will receive either finerenone or a placebo for about 180 days, alongside their usual ACEI or ARB medication. The study will adjust finerenone doses based on age and body weight. Before starting treatment, participants will attend up to two screening visits within 104 days to check eligibility. During the treatment phase, participants will make at least seven visits to the study site for ongoing care and monitoring. Throughout the study, doctors will measure participants' blood pressure, heart rate, temperature, height, and weight. They will collect blood and urine samples to monitor kidney function and protein levels. Heart health will be checked using electrocardiograms and echocardiography. Participants or their parents will answer questions about medication use, side effects, and how they feel. Researchers will track any medical problems that occur during the study and will follow up about 30 days after treatment ends. The main goal is to see how much the protein in urine changes from the start to day 180.

This research aims to evaluate the long-term safety and tolerability of pelacarsen (TQJ230) in adults with established cardiovascular disease and elevated Lipoprotein(a) who have completed the parent trial CTQJ230A12301. The study is an open-label extension following the phase 3 parent study, providing participants continued access to pelacarsen after the initial trial. Participants will receive pelacarsen 80 mg by subcutaneous injection once a month during this open-label extension. The study is single-arm and multicenter, focusing on continued treatment with pelacarsen for up to 36 months after completion of the parent study. Throughout the study, participants will be monitored regularly to assess safety and tolerability, with particular attention to adverse events occurring up to 36 months. Researchers will collect data on health status throughout this period to understand the long-term effects of pelacarsen in this patient population.

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 effectiveness, safety, and how the body processes INM004 in children with Hemolytic Uremic Syndrome caused by infection with Shiga toxin-producing Escherichia coli (STEC-HUS). The study aims to see if adding INM004 to the usual treatment helps improve kidney function and reduce complications, mortality, and hospital stay time. This is a Phase III study focusing on pediatric patients with this condition. Participants will receive either INM004 or a placebo, both given as two intravenous infusions 24 hours apart. INM004 involves doses of Anti-Shiga Toxin Hyperimmune Equine Immunoglobulin F(ab´)2 fragments calculated by the child's weight, infused over 50 minutes or 100 minutes for those with a body mass index over 30. The placebo matches the infusion schedule and appearance but contains no active drug. During the study, participants will be closely monitored for kidney function recovery over 28 days and other health outcomes. Assessments include laboratory tests for blood and kidney function, safety evaluations, and pharmacokinetics of INM004. Hospitalization will occur at the participating institution, with informed consent and pregnancy testing as part of the process. The study includes children from 9 months to under 18 years old and lasts through the acute phase recovery period.

Researchers are tracking patients with Fabry disease through an ongoing international, multi-center observational program called the Fabry Registry. This program collects routine clinical data from patients regardless of their treatment status to better understand the disease's variability, progression, and natural history. It also focuses on enhancing patient care by supporting the development of monitoring recommendations and evaluating the long-term safety and effectiveness of Fabrazyme, a treatment for Fabry disease. The study includes a Fabry Pregnancy Sub-registry, which is a voluntary, international, longitudinal observation program that monitors pregnancy outcomes for women enrolled in the Fabry Registry who are pregnant or have been pregnant. This sub-registry collects medical and obstetric history, pregnancy, and birth data, along with infant growth information up to 36 months postpartum, regardless of the specific treatment received. No experimental treatments are administered in either registry; patients continue receiving routine care as determined by their physicians. Participants contribute data through clinical assessments and standard care evaluations performed by their doctors. The study measures long-term outcomes including safety and effectiveness of Fabrazyme over up to 33 years, as well as pregnancy outcomes and infant growth data. The program helps fulfill regulatory requirements and supports research while tracking patient health over extended periods without altering their usual care.

Researchers are studying the drug levosimendan taken orally to evaluate its safety and effectiveness 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 see if levosimendan improves exercise ability, measured by the change in the distance walked in six minutes. This is a Phase 3 clinical trial involving approximately 540 participants. Participants will be randomly assigned in a 2:1 ratio to receive either oral levosimendan or a matching placebo. After the initial study period, those who qualify may join an open-label extension lasting 52 weeks to continue receiving the study drug. The study carefully monitors the participants' health and responses during these phases. Throughout the trial, participants will undergo various assessments including right heart catheterization, echocardiograms, and a six-minute walk test to measure exercise capacity. Heart rhythm will be monitored using a 48-hour ambulatory cardiac monitor during screening. Safety and effectiveness are tracked over at least 26 weeks, with the primary measurement being the six-minute walk distance. Participants may be followed for up to a year if they enter the extension phase.

Researchers are evaluating an organ dysfunction scoring system adapted specifically for pregnant and early postpartum patients admitted to intensive care units (ICUs). The study aims to develop and validate this obstetric SOFA score (SOFA-OBS) to better predict ICU mortality and sepsis-related mortality in this group. The current general SOFA score does not account for physiological changes during pregnancy and early postpartum, such as reduced creatinine levels and lower blood pressure, which may affect its accuracy in these patients. This study will include about 130 female participants who are either pregnant at any stage or within three days after giving birth and require ICU care. The study will use the new SOFA-OBS score that adjusts kidney and cardiovascular measures to reflect pregnancy changes and simplifies respiratory function evaluation by using a non-invasive pulse oximeter when arterial blood gases are unavailable. The original neurologic, liver, and platelet assessments remain unchanged. Researchers will collect data routinely gathered during ICU care without additional interventions. They will track SOFA and SOFA-OBS scores daily during ICU stays, up to discharge or death, focusing on the first 24 and 48 hours, and assess various organ functions and sepsis definitions adapted to pregnancy stages. Participants will be monitored throughout their ICU stay with data recorded on organ function, infection status, interventions, and outcomes. The study will compare SOFA-OBS with the general SOFA score for predicting mortality and sepsis outcomes. Data collection will include laboratory tests, vital signs, and clinical assessments using standardized forms and electronic systems. Safety and privacy will be maintained, and consent obtained before participation. The total duration depends on ICU stay, with daily evaluations until discharge or death, or up to 28 days post-enrollment for mortality outcomes.

Researchers are investigating the effectiveness, safety, and tolerability of iptacopan (LNP023) alongside standard care in adults with active lupus nephritis Class III-IV, with or without Class V. This Phase 2 trial aims to assess how well iptacopan works in this condition by comparing it with placebo combined with standard treatments. The study is carefully designed to explore different doses and their impact on kidney health in participants with biopsy-confirmed active lupus nephritis. Participants receive either iptacopan or placebo along with their usual care, including corticosteroids and immunosuppressive drugs like MMF or MPS, for 52 weeks. The study is divided into two parts, both lasting 52 weeks, during which participants take the assigned medications. The treatment is given in a double-blind manner, meaning neither the participants nor the researchers know who is receiving the active drug or placebo. Throughout the study, researchers monitor kidney function and disease activity, focusing on the proportion of patients achieving complete renal response by week 24 without kidney flares. Participants undergo regular assessments including lab tests and clinical evaluations to track their response and safety. They are followed closely during the 52 weeks of treatment to ensure careful observation of effects and any side effects, supporting an in-depth understanding of iptacopan's role in managing lupus nephritis.