Rheumatic Heart Disease

Researchers are evaluating a predictive tool called the Multifactorial Dynamic Perfusion Index MDPI to estimate the risk of acute kidney injury after cardiac surgery involving cardiopulmonary bypass CPB. This observational study involves adults and infants undergoing cardiac surgery, aiming to validate the MDPI in adults, develop a version for infants under 20 kg, and explore if the MDPI can predict other complications including mortality. The MDPI combines seven variables measured during CPB, many of which can be modified by the surgical team to potentially improve patient outcomes. The study is divided into three parts first, validating the MDPI in 800 adult cardiac surgery patients from two centers using existing CPB monitors that collect data such as hematocrit, oxygen delivery, arterial pressure, and transfusions second, developing a modified MDPI for 200 infants under 20 kg undergoing congenital heart surgery with additional infant-specific variables and third, investigating whether the MDPI can predict other postoperative issues like low cardiac output, longer ICU stays, bleeding complications, and 30-day mortality. Data collection includes parameters recorded during surgery and postoperative creatinine values. Participants will undergo cardiac surgery with CPB, during which relevant physiological data will be continuously recorded by specialized monitors. After surgery, kidney function and other outcomes will be monitored for up to 48 hours, with extended follow-up for complications and mortality over 30 days. Researchers will analyze how well the MDPI predicts acute kidney injury and other clinical outcomes, aiming to incorporate the MDPI into real-time monitoring devices to guide therapeutic decisions during surgery.

Researchers are evaluating whether stopping secondary antibiotic prophylaxis SAP is not worse than continuing SAP in preventing worsening of rheumatic heart disease RHD in children and adolescents. This Phase 3 trial focuses on participants aged 5 to 20 years who have mild RHD and have already received at least two years of SAP. The goal is to see if stopping SAP affects heart disease progression compared to continuing treatment. The study is randomized and controlled with a single masking approach. Participants will be randomly assigned to either stop SAP or continue receiving monthly intramuscular benzathine benzylpenicillin G BPG injections for two years. Dosage is based on weight, with children under 30 kg receiving 600,000 IU and those 30 kg or above receiving 1.2 million IU every 28 days. The control group continues the standard care of monthly BPG injections, while the intervention group receives no prophylaxis for the same period. During the study, participants will be monitored for two years to assess the progression of RHD using echocardiographic evaluations based on the 2023 World Heart Federation criteria. Blinded adjudicators will review heart imaging results to determine disease changes. Researchers will also analyze outcomes for subgroups with either normalized or stable mild RHD and explore differences based on prior prophylaxis routes. The trial spans from enrollment through the end of the 2-year treatment period, with safety and disease progression closely followed.

Researchers are collecting clinical data on patients with symptomatic mitral valve disease, including mitral regurgitation, mitral stenosis, and mixed mitral valve disease. This observational registry aims to improve understanding of the effects of correcting symptomatic mitral valve disease on patient outcomes, particularly in those who may be candidates for Transcatheter Mitral Valve Replacement TMVR. The data may also support future regulatory or reimbursement decisions related to the Cephea Mitral Valve System. Participants include patients with symptomatic mitral valve disease who could be considered for transcatheter mitral valve replacement, based on evaluation by a specialized heart team. The study records information without assigning treatments, focusing on those judged more suitable for TMVR than conventional surgery or other therapies like transcatheter edge-to-edge repair TEER. There are no specific interventions administered as this is an observational registry. Participants provide informed consent and agree to follow study requirements, allowing ongoing data collection during their clinical care. The study monitors patients from enrollment through up to two years of follow-up to assess clinical outcomes related to mitral valve disease management. Data collected includes health status, treatment decisions, and other relevant clinical information to better understand the impact of TMVR candidacy on patient health over time.

This research aims to evaluate the feasibility, sustainability, and public health impact of a district-based program for secondary prevention of Rheumatic Heart Disease RHD in Uganda. It is a non-randomized study testing the implementation of decentralized RHD preventive services, overseen by the Uganda Ministry of Health and District Health Offices in partnership with a technical quality assurance team. The study uses the RE-AIM framework to measure program reach, effectiveness, adoption, implementation, and maintenance while also estimating cost-effectiveness and budget impact. The study involves the ADUNU program, which integrates echocardiographic screening for RHD in children and young adults through community and facility-based testing. The program also includes registry-based secondary prophylaxis injections of Benzathine penicillin G BPG administered at local health centers HCIII and HCIV in the participating districts. This approach is designed to improve access to care and maintain patients linked to treatment. Participants and healthcare providers residing or working in the involved districts will engage with the program over several years. Researchers will assess the proportion of persons reached, enrolled, retained, and adherent to treatment at multiple time points up to five years. They will also evaluate program adoption and implementation at organizational, provider, and patient levels through surveys and interviews, alongside a cost analysis of the program at three years. The study ensures ongoing monitoring of adherence and outcomes to understand the programs public health impact.

Heart valve diseases are a major cause of illness and death, following coronary artery disease, hypertension, and heart failure. Mitral stenosis, a condition where the mitral valve narrows and restricts blood flow, leads to symptoms like shortness of breath, fatigue, and decreased exercise capacity. Patients often experience reduced physical activity, impaired respiratory function, and a decline in quality of life. Most prior studies focused on patients after percutaneous mitral balloon valvuloplasty, and groups studied were not uniform. This research compares exercise capacity, muscle oxygenation, aortic stiffness, and quality of life between patients who have had mitral valve surgery for mitral stenosis and healthy individuals. It also assesses respiratory muscle strength and endurance, pulmonary function, and physical activity levels. Measurements include cardiopulmonary exercise testing, near-infrared spectroscopy for muscle oxygenation, spirometry, mouth pressure devices for respiratory strength, incremental loading tests, arteriography for aortic stiffness, physical activity monitors, and quality of life surveys. Participants undergo evaluations lasting about one year, including tests of exercise capacity, muscle and respiratory function, aortic stiffness, and physical activity monitoring. Researchers measure primary outcomes like maximal exercise capacity, muscle oxygenation, aortic stiffness, and quality of life, as well as secondary outcomes such as respiratory muscle strength and endurance, various pulmonary function parameters, and physical activity level. The study involves both patients after mitral valve intervention and healthy controls to provide comparative data on these health indicators.

Researchers are exploring the natural history and genetics of autoinflammatory and autoimmune diseases, including VEXAS syndrome and related disorders. This study aims to discover genetic factors contributing to these inflammatory conditions and to enhance understanding of their immune system mechanisms. It also seeks to describe clinical features of poorly characterized diseases through chart reviews of routine medical care. Participants include individuals with known or suspected autoinflammatory diseases, family members related by blood or marriage, and healthy volunteers. Samples such as blood, saliva, hair, nails, or buccal swabs may be collected for genetic and immunologic studies. Some participants might undergo skin biopsies. The study involves genetic analysis and functional immunologic tests to identify disease-associated variants and features. During the study, participants provide samples for laboratory analysis and may have their medical records reviewed. Researchers measure the number and types of genetic variants linked to inflammatory diseases over up to five years. Study involvement includes mail-in sample collection and possible clinical evaluations. The total participation time can extend up to the study end date in 2032, with ongoing data collection and analysis to improve understanding of these conditions.

Researchers are evaluating the preliminary safety and effectiveness of the Cephea Mitral Valve System in treating symptomatic patients with mitral valve disease, including mitral regurgitation, mitral stenosis, and mixed mitral valve disease. The study focuses on patients for whom transcatheter therapy is considered more suitable than open heart surgery. This feasibility study is sponsored by Abbott Medical Devices and involves adult participants aged 18 years and older. Participants will receive treatment using the Cephea Mitral Valve System, a device designed to address mitral valve issues via a minimally invasive transcatheter approach. The study does not involve a control group or placebo and is not blinded. The primary outcomes measured 30 days after implant include freedom from all-cause mortality and reduction of mitral regurgitation to Grade I or less. During the study, participants will be monitored for safety and treatment effectiveness. Evaluations include assessments of mitral valve function and survival status at 30 days post-implant. Follow-up will help researchers understand the devices impact on mitral valve disease symptoms and patient outcomes. The total duration of participation may vary depending on individual follow-up schedules and assessments.

Researchers are exploring a new way to personalize treatment for autoimmune and autoinflammatory rheumatic diseases. This study aims to develop and use a special cell-based test that helps predict how well patients will respond to disease-modifying antirheumatic drugs DMARDs. By identifying the best treatment for each person, the study hopes to improve treatment success, shorten the time to see benefits, and lower healthcare costs. The approach focuses on analyzing immune cells from patients to better understand their response to different therapies. The study includes several groups newly diagnosed patients without prior treatment, patients already on immunomodulatory therapy, and healthy individuals for comparison. Blood samples are collected at different times depending on the group. Researchers use advanced laboratory methods to test immune cell responses to DMARDs outside the body, measuring specific immune signals and gene activity. Archived tissue samples are also studied to link immune profiles with disease features. This design allows detailed analysis of immune function related to treatment. Participants will provide blood samples and undergo clinical assessments over time, including baseline and follow-up visits at 6 and 12 weeks, with additional optional visits if disease worsens or treatment changes. The study measures changes in immune cell types and activity using the ex vivo assay and other tests. Data are stored securely, and the study will compare predicted responses with actual clinical outcomes to validate the tests usefulness. Participants involvement may last up to a year, with ongoing monitoring and data collection to support personalized therapy development.

Researchers are evaluating the safety and effectiveness of the VDyne Transcatheter Tricuspid Valve Replacement System for treating tricuspid regurgitation, a heart valve disorder. The study aims to gather data to support the devices approval under Conformit Europenne CE marking. This clinical investigation is led by VDyne, Inc. and focuses on adults with severe tricuspid valve problems who are candidates for this treatment. Participants will receive treatment using the VDyne System, a device designed to replace the tricuspid valve via a minimally invasive procedure. The study includes one treatment group using this system. Key assessments will occur from baseline to one month after the procedure, with extended follow-up up to one year to track device-related major adverse events, valve function, quality of life, symptom status, and physical capacity. Throughout the study, participants will undergo imaging evaluations, functional tests like the 6-minute walk test, and quality of life questionnaires. Researchers will monitor safety and effectiveness at multiple time points, including 30 days, 3 months, 6 months, and one year after the procedure. The total duration of participation varies, with ongoing assessments to ensure comprehensive data collection and patient monitoring.

Researchers are evaluating the safety and clinical effects of the VDyne Transcatheter Tricuspid Valve Replacement System for treating people with symptomatic severe tricuspid regurgitation, a heart valve disorder. This study aims to assess how well the device works and its safety profile in improving symptoms and heart function in affected patients. The study involves implanting the VDyne system, which includes a bioprosthetic tricuspid valve and delivery tools, via a transfemoral approach while the heart is beating. The valve can be repositioned or retrieved during the procedure if needed. There is one treatment group receiving this device, and the procedure is guided by imaging techniques without the use of cardiopulmonary bypass. Participants will be monitored from the time of implant through one year after the procedure. Researchers will assess major adverse events, changes in valve function, symptom status, exercise capacity, and quality of life at various timepoints up to one year. Safety and effectiveness will be evaluated through imaging, clinical assessments, and questionnaires. The study starts with screening, implantation, and includes follow-up visits for ongoing evaluation.