Neonatal Care

Preterm birth, defined as birth before 37 weeks of gestation, occurs in about 8 percent of pregnancies in Canada and is linked to many health challenges, especially when it happens before 29 weeks. At this early stage, infants often face breathing difficulties and may require resuscitation. This trial compares resuscitation using either low (30%) or high (60%) oxygen levels to determine which approach results in better survival and neurodevelopmental outcomes at around 24 months of age. The study uses a cluster randomized crossover design where hospitals alternate between starting resuscitation with 30% or 60% oxygen for groups of 30 infants. Resuscitation includes standard care steps like lung expansion and ventilation support as needed. Oxygen is initially given at the assigned concentration for the first 5 minutes, then adjusted based on the infant's oxygen saturation levels and heart rate over the next 5 minutes to maintain target saturation ranges. This approach aims to balance risks of too much or too little oxygen. Participants are infants born between 23 and 28 weeks gestation who receive full resuscitation at participating centers. During the study, infants have oxygen saturation monitored continuously, and adjustments to oxygen concentration are made carefully. Researchers will evaluate survival and major neurodevelopmental outcomes at 24 months corrected age, along with several safety and health measures during the neonatal intensive care stay. The trial is expected to provide important evidence to guide oxygen use during resuscitation of extremely preterm infants.

This clinical trial studies the use of a linear cognitive aid app designed to help emergency medical service (EMS) teams respond to pediatric emergencies. Researchers want to see if this new tool is practical and well accepted by EMS teams in both urban and rural settings. The study compares current standard cognitive aids with the new linear cognitive aid during simulated critical resuscitation scenarios for children. Participants will be randomized into two groups: one using their current standard cognitive aids, and the other using the linear cognitive aid app. Each team will perform two high-fidelity, in situ simulations of critical pediatric resuscitation emergencies. The linear cognitive aid app offers minimal input, linear step-by-step guidance, tailored recommendations based on the patient’s age and scenario, and audio prompts timed to the resuscitation process. During the study, researchers will assess the time it takes teams to reach American Heart Association (AHA) technical milestones, teamwork quality, and participants' perceived cognitive load during each 10-minute simulation. The study aims to evaluate how well teams perform and how the different cognitive aids affect their experience. The trial is led by Beth Israel Deaconess Medical Center and includes adult EMS providers who speak English and are actively practicing.

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 conducting a nationwide, multicenter observational study in China to investigate the severity and challenges faced by Small Vulnerable Newborns (SVN), including preterm, small for gestational age, and low birth weight infants. The study aims to provide a clear and unified description of these newborns' issues and to develop targeted prevention strategies to improve care and outcomes. This comprehensive research gathers data across multiple centers to better understand the scope of these conditions. In this study, participating hospitals across China will collect clinical data on vulnerable newborns under their care throughout 2024. Detailed questionnaires will be completed to support thorough data collection. The study does not involve any treatment interventions; instead, it focuses on observation and data gathering to assess the incidence, complications, and mortality rates of these newborns. Participants in the study will have their clinical information recorded from hospital admission until discharge or death, with follow-up up to one year. Researchers will measure the number of small vulnerable newborns, their mortality rates, and the incidence of related complications. This observational approach aims to provide valuable insights to guide prevention efforts and improve care for these at-risk infants.

Researchers are conducting a study to systematically screen newborns in the Normandy region for lysosomal storage diseases such as Mucopolysaccharidosis type I and Pompe disease. This observational study aims to evaluate the occurrence and epidemiology of these diseases using dried blood samples collected from newborns. The study is based on previous pilot work and seeks to include about 100,000 newborns over a period of three years. All newborns born in Normandy maternity hospitals who are participating in the national neonatal screening program will have additional blood samples collected on blotting paper for this study. The screening occurs within the first few days after birth, typically from day 2 to day 4. The study will continue until the target number of participants is reached. Participants will have blood samples collected as part of routine neonatal screening, with extra samples taken specifically for this research. The main outcome measured is the number of newborns screened relative to the number of samples collected. Secondary outcomes include the number of positive cases detected for Mucopolysaccharidosis type I and Pompe disease. The study involves parental consent and monitors newborns during these early days, with no further intervention or long-term follow-up described.

Many premature newborns have trouble breathing after birth and receive support with a breathing machine called nasal continuous positive airway pressure (NCPAP). When their breathing worsens despite NCPAP, they may be treated with surfactant medication inserted into the windpipe using either a ventilation tube or a thin catheter. This research is studying the use of video laryngoscopes, which have a camera and display the airway on a screen, compared to standard direct laryngoscopes that require doctors to look directly into the baby's mouth to insert the tube or catheter. The study aims to see if video laryngoscopy improves the success rate of inserting the thin catheter on the first attempt without causing drops in oxygen or heart rate. The study observes infants who need a thin endotracheal catheter inserted for surfactant administration. It compares two approaches: using a video laryngoscope versus a direct laryngoscope during the insertion. This is done across multiple hospitals, each switching from direct to video laryngoscopy over time. The study does not assign which method is used but records which laryngoscope was used for each insertion attempt to compare outcomes. The main focus is on successful first attempts without physiologic instability, as well as secondary measures like number of attempts, duration, oxygen levels, heart rate, and any complications. Participants are newborn infants up to 28 days old who require thin catheter insertion for surfactant treatment and whose parents have consented to share data. Researchers will collect detailed information during and after the insertion attempt, including heart rate, oxygen saturation, duration of the procedure, and any adverse events such as oral trauma or need for chest compressions. Data will be analyzed to evaluate the success and safety of using video versus direct laryngoscopy. The total participation time depends on when the catheter insertion is performed and the follow-up assessments within 72 hours after the procedure.

Researchers are evaluating the safety and effectiveness of a liposomal mitoxantrone combination regimen in treating relapsed and refractory solid tumors in children, adolescents, and young adults aged 6 to 24 years. This prospective, single-arm, multicenter clinical study aims to improve the objective response rate compared to historical data and to assess related side effects. The study focuses on specific tumor types including bone and soft tissue tumors and neuroblastoma among others. Participants will receive a treatment regimen combining mitoxantrone liposome, capecitabine, and anlotinib over 4 to 6 cycles, each lasting 21 days. Capecitabine is given twice daily on days 1 to 14, while anlotinib dosage is adjusted based on body weight and administered for 14 days per cycle. Those showing partial response or better may receive additional local treatments such as surgery or radiotherapy. During the study, participants will have regular checkups and tests to monitor tumor status, blood parameters, and other relevant health indicators. The main measurement is the objective response rate after up to 6 treatment cycles. Other outcomes include disease control rate, progression-free survival, overall survival, and safety monitoring for up to one year after treatment. The total observation period includes follow-up assessments to evaluate lasting effects and adverse events.

Many newborn babies experience breathing difficulties that require intubation, a procedure where a tube is inserted into the windpipe to help with breathing support. This trial compares two methods used by doctors to perform intubation: the traditional direct laryngoscopy (DL), where doctors look directly into the baby's mouth with a standard laryngoscope, and video laryngoscopy (VL), which uses a camera and screen to view the windpipe. The study aims to determine if VL leads to more successful first attempts without causing drops in oxygen levels or heart rate compared to DL. The trial uses a stepped-wedge cluster randomized design across multiple hospitals. Initially, each hospital uses the standard DL method for intubation. At randomly assigned intervals, hospitals switch to using the VL device, the C-MAC video laryngoscope, which includes a monitor and special blades. Doctors receive training and practice with this device before switching. Other intubation practices, such as drug use and supplemental oxygen, remain the same throughout the study. Participants in the trial are newborn infants undergoing intubation. Researchers will collect information about intubation attempts, including success rates, oxygen levels, heart rates, and any complications like oral trauma or need for chest compressions. The main outcome is whether babies are successfully intubated on the first try without physiological instability within five minutes. Additional measures include procedure time, number of attempts, and adverse events. Data will be compared between the periods using DL and VL to assess effectiveness and safety over the course of the study, which lasts until August 2026.

Researchers are studying the physical, nutritional, pulmonary, and mental health of females with cystic fibrosis (CF) who are planning pregnancy, are pregnant, or in early parenthood. They are also examining the health of their children from infancy through early childhood to see if new screening or care methods should be included in clinical guidelines. This observational study focuses on the impact of pregnancy and parenthood in the era of CFTR modulators, treatments that target the cause of CF. The study, called MATRIARCH_CF, includes three related sub-studies: "Mama" follows females with CF aged 16 or older who are planning pregnancy or pregnant, involving up to eight visits with tests like blood work, lung function, imaging, and interviews. "Mini" enrolls biological offspring from birth to age two, with up to four visits including blood tests, sweat tests, and brain ultrasounds. "Midi" studies children aged three to six with up to two visits involving lung function tests and lung MRI. The study does not provide or change treatments but monitors health through these investigations. Participants will attend multiple visits for physical and psychological assessments, including lung function, imaging, and blood tests for mothers, and various tests for children depending on their age group. Researchers will measure outcomes such as lung function changes, pulmonary complications during pregnancy, premature deliveries, liver dysfunction, congenital abnormalities, and CF diagnoses in children. The study aims to gather detailed health information over several years without altering clinical care, lasting up to three years for some outcomes.

Researchers are studying aficamten in children and adolescents aged 6 to under 18 years with symptomatic obstructive hypertrophic cardiomyopathy (oHCM), a condition causing thickened heart muscle and outflow obstruction. The study aims to evaluate the drug's effectiveness, safety, and how the body processes it in this pediatric group. Adolescents and children will be studied sequentially, starting with adolescents to establish safe and effective dosing before proceeding to younger children. The trial is a Phase 2/3 randomized, double-blind, placebo-controlled study with additional open-label extension periods for long-term assessment. Participants will be assigned randomly to receive either oral aficamten or a placebo once daily. The initial treatment period lasts 12 weeks and is double-blinded. After this, all participants receive aficamten for 52 weeks during an open-label extension, followed by a long-term extension of 144 weeks to further assess safety, tolerability, and efficacy. Doses range from 5 mg to 20 mg and are adjusted based on echocardiography findings. This staged approach ensures careful monitoring and dosing adjustments throughout the study. During the study, participants will undergo regular evaluations including heart ultrasounds to measure heart function and obstruction, blood tests for heart biomarkers, and assessments of heart failure symptoms using the New York Heart Association functional class. Researchers will monitor how aficamten affects the heart's outflow tract gradient over 12 weeks and track long-term safety over several years. The study includes multiple visits for clinical assessments, laboratory tests, and drug level measurements to understand treatment effects and ensure participant safety.