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Researchers are evaluating the safety and effects of marstacimab, a study medicine, for potential treatment of hemophilia in boys and male children aged 1 to 17 years. The trial focuses on those with severe Hemophilia A or moderately severe to severe Hemophilia B, including participants with or without inhibitors. The study aims to compare participants experiences with marstacimab to their historical bleeding episodes without the medicine. All participants will receive weekly subcutaneous injections of marstacimab. The first dose is administered at the study site by staff, and subsequent doses can be given at home or by study staff during the 12-month treatment period. Enrollment opens sequentially by age groups, starting with adolescents 12-17 years, followed by children 6-11 years, and finally children 1-5 years. The study includes about 1 month of screening, 12 months of treatment, and 1 month of follow-up. Participants will visit the study site at least 10 times during the study, with the option for two visits to occur at home if allowed. They will also receive six phone calls approximately every two months. Researchers will monitor bleeding rates, adverse events, injection site reactions, and immune responses. The overall participation lasts about 14 months, including screening, treatment, and follow-up periods.

Researchers are evaluating the effects of nipocalimab on children aged 2 to less than 18 years with generalized myasthenia gravis gMG, a condition causing muscle weakness. The study aims to assess the safety, tolerability, and how the body processes nipocalimab in pediatric participants who have not responded well to stable standard treatments. This open-label, multicenter trial is sponsored by Janssen Research & Development, LLC and focuses on both pharmacokinetics and pharmacodynamics over an extended period. Participants are divided into two age groups children aged 2 to less than 12 years and adolescents aged 12 to less than 18 years, with US sites only including those 8 to less than 18 years old. All will receive nipocalimab through an intravenous infusion every two weeks for 24 weeks. After this treatment period, participants may choose to join a long-term extension phase to continue monitoring. Throughout the study, participants will undergo regular evaluations including blood tests to measure total serum immunoglobulin G IgG levels and monitor for adverse events, laboratory abnormalities, vital signs, and physical examinations. Additional assessments include measuring drug concentration and immune response markers. The study also tracks changes in myasthenia gravis symptoms and quality of life over a period of up to three years, with ongoing safety and pharmacokinetic monitoring during this time.

Researchers are evaluating whether the combination of vicadrostat BI 690517 and empagliflozin helps adults with heart failure who have symptoms and a left ventricular ejection fraction LVEF of 40% or more. This phase III study is designed to compare the effects of vicadrostatempagliflozin tablets versus placeboempagliflozin tablets on heart failure outcomes. The study aims to understand if this combined treatment improves health and reduces heart-related events. Participants are randomly assigned to one of two groups one group takes vicadrostat plus empagliflozin tablets once a day, and the other takes placebo plus empagliflozin tablets once a day. The study has no fixed duration and continues as long as participants benefit and tolerate the treatment. Throughout the study, participants visit their doctors regularly for health checks, and study staff may also contact them by phone to monitor well-being and any side effects. During the study, researchers monitor participants health through regular doctor visits and phone contacts. They collect data on heart-related events such as cardiovascular death, hospitalizations for heart failure, and urgent visits for heart failure over up to 42 months. Participants also answer questions about their symptoms and well-being. The study carefully tracks safety and treatment tolerance while gathering information to determine if the combined treatment helps people with heart failure.

This research evaluates the effects of the Close Collaboration with Parents, a family-centered care approach for neonatal intensive care unit NICU staff, on parents of newborns in the NICU. The study compares experiences before and after this intervention, focusing on family-centered care quality as rated by parents, parental anxiety symptoms, and readiness for the infants discharge. The intervention is planned to be implemented from December 2023 through December 2024. The Close Collaboration with Parents intervention educates NICU healthcare staff to better observe infants individual needs, support parents in understanding and participating in their infants care, and involve parents in decision-making from early care through discharge preparation. Data will be collected during three phases before the intervention September to December 2023, during the intervention January to December 2024, and after January to April 2025. Parents and newborns admitted before and after the intervention form two groups for comparison. Participants will be parents of infants admitted to the NICU for at least two weeks. Researchers will collect data on family-centered care quality, parental anxiety, and discharge readiness around the infants discharge at about one month of age. Data will be pseudonymized and securely managed, with statistical analyses adjusting for factors like length of stay and parental mental health history. Participation spans from pre-intervention data collection through post-intervention follow-up, lasting over a year.

This research aims to create a detailed registry called the Tiny Baby Collaborative Multicenter Inventory of Neonatal-Perinatal Interventions MINI minimum dataset. It focuses on outcomes and practices for infants born extremely prematurely at 22 to 23 weeks gestation and admitted to intensive care at participating hospitals. The goal is to improve understanding of treatments and outcomes in this vulnerable population. The study collects observational baseline data about both mothers and infants. This includes demographics, mothers health and delivery details, as well as the infants health, medical interventions, and clinical outcomes during neonatal intensive care. Participating centers may also optionally include data on infants born before 22 weeks gestation admitted to NICU. The collected data help hospitals with quality improvement, evaluate treatment-outcome associations, and monitor trends in neonatal care. Participants information is gathered as part of routine care from birth through intensive care admission and early life, up to 1 year old. Researchers compile a longitudinal database with uniform collection of characteristics and outcomes for all extremely premature births and admissions. This ongoing registry supports reporting, analysis, and tracking of neonatal-perinatal interventions and their results through January 2028 and study completion in 2030.

Researchers are evaluating the long-term safety, tolerability, and effectiveness of marstacimab as a preventive treatment in males aged 1 to 74 years with severe hemophilia A or moderately severe to severe hemophilia B, including those with or without inhibitors. This open-label extension study follows participants who completed prior Phase 3 studies without early termination, aiming to gather extended data on marstacimabs impact over several years. Participants receive marstacimab as a subcutaneous injection using a prefilled pen or syringe. Those aged 12 years and older start with a 300 mg loading dose followed by 150 mg weekly, with possible dose escalation to 300 mg weekly. Children aged 6 to under 12 years receive a 150 mg loading dose followed by 75 mg weekly, with the option to increase to 150 mg weekly. The study provides devices for administration and allows some flexibility based on participant needs and country-specific availability. During the study, participants undergo regular assessments including monitoring of adverse events, laboratory tests, coagulation factor consumption, bleeding rates, joint health, and quality of life questionnaires over a period of up to seven years. Researchers will track safety outcomes such as thrombotic events, injection site reactions, and immune responses to the drug. This extended follow-up helps understand marstacimabs long-term effects and supports dosing decisions in younger age groups.

This research aims to evaluate the effects of combining baxdrostat with dapagliflozin compared to dapagliflozin alone in adults aged 40 and older who have type 2 diabetes, established cardiovascular disease, a history of hypertension with a systolic blood pressure of at least 130 mmHg, and at least one additional risk factor for heart failure. The study is a phase III, randomized, placebo-controlled trial focusing on preventing heart failure events and cardiovascular death. Participants will be randomly assigned to receive either baxdrostat with dapagliflozin or placebo with dapagliflozin. Those starting the baxdrostatdapagliflozin treatment may begin with a lower baxdrostat dose that can be increased if certain criteria are met. A run-in period with dapagliflozin alone for 4 to 6 weeks may occur for those not previously treated or treated less than 4 weeks with SGLT2 inhibitors. Treatment visits will occur at about 2, 4, 8, 16, and 34 weeks after randomization, then every 4 months until study closure. Participants will undergo screening for eligibility within a 14-day period, with an optional pre-screening phase that does not require site visits or consent. During the study, regular assessments including monitoring for heart failure events and cardiovascular outcomes will be conducted. If participants stop the blinded treatment early, they may continue with open-label dapagliflozin unless specific discontinuation criteria apply. The study will continue until a predetermined number of cardiovascular events occur, with ongoing data collection and visits according to protocol.