Neonatal Jaundice

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 infants 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.

Researchers are conducting a Phase 3 clinical trial to evaluate the safety and effectiveness of AOC 1044, also known as delpacibart zotadirsen, for treating Duchenne Muscular Dystrophy DMD in boys aged 7 to 16 with specific gene mutations suitable for exon 44 skipping. This study is designed as a randomized, double-blind, placebo-controlled trial to assess the impact of this intravenous treatment on muscle function over time. Participants will be randomly assigned to receive either AOC 1044 or a placebo infusion every 6 weeks for 54 weeks, totaling 9 doses during the double-blind treatment period. After this, all participants can join an open-label extension where they receive AOC 1044 every 6 weeks for another 54 weeks, adding 9 more doses. Following the final dose at week 102, participants will have assessments at weeks 108 and 114 to evaluate safety and treatment effects. During the study, participants will undergo various assessments including tests for time to rise velocity, muscle strength, walking and climbing abilities, and quality of life measures. Muscle enzyme levels and global impressions of severity and change from both patients and caregivers will also be monitored. Safety and tolerability will be reviewed regularly by an independent committee. Overall participation lasts over two years, including screening, treatment, extension, and follow-up phases.

Researchers are studying infants with cholestasis, including conditions like biliary atresia and idiopathic neonatal hepatitis that cause prolonged jaundice and high bilirubin levels after the newborn period. The Childhood Liver Disease Research Network ChiLDReN aims to create a database of clinical information and biological samples from affected children to support research and clinical trials on these serious liver diseases in children. This project collects detailed clinical data, laboratory results, and biological samples such as blood and tissue from infants diagnosed or suspected to have biliary atresia or other neonatal liver diseases. Infants diagnosed with biliary atresia are followed closely during their first year, at 18 months, annually up to age 10, and then every two years, or until liver transplantation occurs. Infants with other cholestatic diagnoses exit the study after diagnosis is confirmed. Participants undergo routine clinical care and evaluations, with data entered into a secure national research database. Blood samples are collected during clinically indicated procedures when possible. Researchers measure disease progression over time at multiple intervals from baseline through 20 years of age. The study involves long-term follow-up of outcomes and data are securely stored and eventually transferred to a national data repository.

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 babys 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 MATRIARCHCF, 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 evaluating nipocalimab to reduce the risk of fetal anemia and other serious complications in pregnancies at high risk for severe Hemolytic Disease of the Fetus and Newborn HDFN. The study compares nipocalimab to a placebo in pregnant participants to see if it can decrease risks like fetal loss, the need for intrauterine transfusions, hydrops fetalis, or neonatal death. This phase 3 trial focuses on pregnancies with maternal alloantibody presence and previous severe HDFN history. Participants receive either nipocalimab or a matching placebo through weekly intravenous infusions starting at randomization until gestational week 35. The study is randomized and triple-masked, meaning neither participants nor researchers know who receives the drug or placebo. The treatment period covers the pregnancy phase where risk is highest, with careful monitoring throughout. During the study, participants undergo various assessments including lab tests, antibody titers, fetal antigen testing, and physical exams to monitor health. Researchers track pregnancy outcomes through delivery and up to 4 weeks after birth or 41 weeks postmenstrual age for newborns. Long-term infant health, including development and complications related to HDFN, is followed for up to 104 weeks. Safety and maternal outcomes are also closely observed until 24 weeks postpartum.

Researchers are studying pregnant individuals at risk for hemolytic disease of the fetus and newborn HDFN, a condition where incompatible blood types between mother and baby cause rapid breakdown of fetal or newborn red blood cells. This observational study aims to evaluate the risk of anemia in fetuses and newborns receiving standard care, tracking outcomes from pregnancy through two years after birth. The study looks at complications such as fetal loss, transfusions, and neonatal death to better understand HDFNs impact. Participants receive standard medical care for HDFN as per local clinical practice without additional study treatments. Data collection occurs throughout pregnancy and continues for two years after birth for the baby. Participants will provide medical information and complete questionnaires at set times during follow-up. Data is gathered from medical centers that routinely treat HDFN to capture comprehensive clinical outcomes. During the study, researchers will collect detailed medical records, including clinical outcome assessments reported by patients or observers. They will measure outcomes such as fetal loss, intrauterine transfusions, hydrops fetalis, neonatal death, and disease severity. The study tracks pregnancies from conception through the neonatal period and continues monitoring infants up to two years. This long-term follow-up helps understand the course and effects of HDFN under standard care conditions.

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 evaluating the safety and feasibility of lung transplants from blood group A2 donors to blood group O recipients. This study aims to increase the donor pool for blood group O lung transplant candidates, who often face longer wait times and higher mortality on the waitlist. It is the first prospective study worldwide to assess ABO-incompatible lung transplants in this way. The intervention involves lung transplant surgery using lungs from blood group A2 donors transplanted into blood group O recipients who have low anti-A antibody levels and a negative virtual crossmatch at the time of transplant. The study monitors the recipients closely after surgery to assess immune response and transplant success. Participants will be monitored through various evaluations including antibody levels on post-operative days 5 or 12, primary graft dysfunction grading in the first three days, and assessment for rejection or infection over the following years. Long-term follow-up up to 10 years will evaluate graft function, chronic lung problems, infections, malignancies, and survival outcomes. The total participation time varies depending on individual follow-up and outcomes.