Biliary Atresia

Researchers are conducting a long-term observational study to understand how physiological changes in children after biliary tract reconstruction affect their health over time. The study focuses on changes in liver function, metabolism, immune response, and other bodily functions as children grow. It also evaluates the risks these changes might pose for chronic illnesses like cardiovascular disease, diabetes, and liver cirrhosis in adulthood, as well as their effects on cognitive and musculoskeletal health. The goal is to identify early biomarkers that predict future health outcomes and to suggest preventive or intervention strategies to reduce long-term complications. The study follows children diagnosed with biliary atresia or choledochal cyst who have undergone biliary reconstruction surgery. A control group of healthy children matched by age and gender will also be observed for comparison. Researchers will collect samples and data at multiple time points: before surgery, then at 1 week, 1 month, 3 months, 6 months, 1 year, 2 years, and 3 years after surgery. These data include targeted metabolic components detected in feces and plasma, along with fecal metagenomic sequencing. Participants will be monitored over several years through scheduled assessments to track metabolic and microbiome changes. The study will measure specific metabolic components in fecal and blood samples at each time point to observe ongoing physiological changes. By following both surgical patients and healthy controls, researchers aim to understand the long-term impacts of biliary reconstruction on children's health and identify ways to reduce future health risks. The study enrollment starts in May 2025 and continues until April 2035.

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.

Biliary atresia is a severe liver disease affecting children, leading to high illness and death rates, with symptoms like intense itching, fatigue, poor growth, liver failure, and impaired brain development. The cause is mostly unknown, and over half of affected children need a liver transplant during childhood. Determining the best time for transplantation is difficult due to lack of consensus based on clinical tools. This research aims to find risk factors for brain-related problems in these children to help decide the optimal transplant timing from a neurological perspective. The study will observe 75 children aged 0 to 18 years with biliary atresia in Denmark, along with 30 healthy children and 20 with tetralogy of Fallot as comparison groups. Participants will undergo detailed evaluations including liver function tests, genetic profiling, nutrition and immune status assessments, brain imaging (MRI), and neurocognitive tests at diagnosis, ages 2, preschool, pre-teen, and teenage. Additional neurocognitive tests will be done if a liver transplant occurs. The study involves advanced monitoring of brain, heart, gut, and immune functions. Children in the study will have regular assessments of their neurocognitive abilities using various tests and brain MRIs at multiple ages. Liver and heart function will be closely evaluated through imaging and lab tests. Researchers will also analyze genetics and microbiome samples. The main outcomes include brain MRI and multiple neurocognitive tests to evaluate development and function. The study is observational and will follow participants over many years to track changes and outcomes related to biliary atresia and transplantation timing.

Researchers are studying biliary atresia, a liver condition in infants and children, to better understand what causes it and how it progresses over time. This observational study aims to identify the genes involved in biliary atresia and to describe the natural history of older children who still have their native liver. The study is conducted by the Childhood Liver Disease Research Network funded by the National Institute of Diabetes & Digestive and Kidney Diseases. The study involves collecting clinical information, genetic material, and body fluid samples from participants once, usually at the start of the study. Participants include children and young adults aged over 6 months up to 20 years who have a confirmed diagnosis of biliary atresia and still have their native liver. Researchers will follow these participants yearly to gather observational data about disease progression and quality of life. Participants will be asked to attend yearly follow-up visits where observational information will be collected, including health status and growth measurements. The primary measurement is to identify genetic factors linked to biliary atresia, while secondary outcomes include understanding disease progression and quality of life over time. The study tracks important health events, growth, and nutritional status to help predict outcomes. Participation may last several years, with annual visits to monitor the condition and collect data.

This research aims to establish a national cohort in China to study biliary dilatation, a complex benign bile duct disorder common in East Asia. By enrolling patients from 25 medical centers, the study will clarify the disease's epidemiology, pathological features, classification, progression, cancer risk, and the best timing for surgery. It will also compare outcomes among different types of biliary dilatation to develop standardized surgical treatment strategies. The study includes both retrospective and prospective cohorts. Retrospectively, it collects past clinical and prognostic data from previously diagnosed patients. Prospectively, it enrolls newly diagnosed patients and follows them over time to observe outcomes and surgical effects. The focus is on patients with Todani types I, IVa, and V biliary dilatation, analyzing perioperative risks, long-term complications, and quality of life after surgery. Participants will have their demographic, clinical, laboratory, imaging, and biomarker data collected throughout their care. Researchers will monitor disease progression, surgical decision-making, postoperative complications, malignant transformation, and patient quality of life over many years. The primary outcome is the rate of long-term complications after surgery, with secondary outcomes including cancer development and perioperative complications. The study will run through 2035, providing long-term follow-up data.

The Calgary Registry for Advanced and Therapeutic Endoscopy (CReATE) is a high-quality, prospective study collecting data on adult patients undergoing Endoscopic Retrograde Cholangiopancreatography (ERCP) for biliary and pancreatic diseases. The registry aims to gather detailed information from multiple centers to better understand outcomes and complications related to this advanced endoscopic procedure. Participants undergo ERCP, an endoscopic procedure used to access biliary and pancreatic structures. The study observes patients consecutively from September 2018 to August 2022, capturing all relevant data before, during, and after the procedure. This includes procedural details and patient outcomes, serving also as a platform for additional prospective studies and randomized trials. During the study, a full-time research assistant collects real-time data by observing procedures directly. Participants are followed up with medical record reviews and phone interviews 30 days after the procedure to assess outcomes such as unplanned healthcare visits and procedure-related complications including pancreatitis, bleeding, and infections. The registry monitors technical success immediately and tracks patients' health for up to 30 days post-procedure, contributing to ongoing research until the study ends in 2030.

Researchers are evaluating a percutaneous cholangioscopic assisted technique using the Spyglass mini-endoscopic system to treat bile duct diseases in participants who are not eligible for traditional endoscopic treatment. This study aims to determine whether this new approach can clear biliary stones and perform endobiliary biopsies more quickly than conventional methods. The study is a non-pharmacological, prospective, single-center interventional trial conducted at the Bologna University Hospital. Participants will undergo the percutaneous cholangioscopic-assisted treatment, which involves a preliminary puncture of the bile ducts followed by a cholangiographic study using the Spyglass system. This system provides direct viewing and allows guided lithotripsy or biopsy collection. Multiple treatment sessions may be scheduled every 15-20 days if residual stones remain, with percutaneous drainage left in place after each session to monitor complications and maintain access. During the study, participants will receive clinical laboratory monitoring and imaging tests as needed, with follow-up visits at 3, 6, 12, 24, and 36 months post-treatment. Researchers will collect clinical data and review anonymized imaging to evaluate outcomes such as number of treatments, duration of drainage, relapse-free interval, and length of hospitalization. The total study duration includes a 24-month enrollment phase followed by a 36-month follow-up period.

This research investigates patients with type IVa biliary dilatation (BD), a complex benign bile duct condition affecting both the intra- and extrahepatic bile ducts. The study aims to build a Chinese patient cohort by enrolling participants from 25 medical centers across China. It seeks to compare surgical treatment approaches for type IVa BD, focusing on perioperative risks, long-term health outcomes, and quality of life to help develop standardized surgical strategies for this condition. Participants with type IVa BD receive one of two surgical treatments: liver resection combined with extrahepatic bile duct removal and Roux-en-Y hepaticojejunostomy, or extrahepatic bile duct removal with Roux-en-Y hepaticojejunostomy alone. These two groups allow comparison of different surgical methods to evaluate their safety and effectiveness. The study continuously enrolls patients and collects comprehensive data throughout their treatment and follow-up periods. During the study, patients are observed for complications occurring within 30 or 90 days after surgery, including long-term complication-free survival. Researchers will also assess rates of malignant transformation, perioperative complications, severity of complications, and the need for reoperations. Quality of life and functional status are monitored using tools like the Mayo Score over an average of six months. The study follows participants up to one year or more, aiming to gather detailed outcome and safety data to inform future treatment decisions for type IVa BD.

Researchers are evaluating the safety and effectiveness of duloxetine in preventing pancreatitis after undergoing endoscopic retrograde cholangiopancreatography (ERCP), a key procedure used to diagnose and treat pancreato-biliary diseases. Post-ERCP pancreatitis (PEP) is a common and serious complication that can lead to significant health problems. This Phase 3 randomized, double-blind study aims to compare duloxetine with a placebo to see if it can reduce the risk of PEP in adults up to 65 years old. Participants will be randomly assigned to receive either a 60 mg dose of duloxetine or a placebo capsule two hours before the ERCP procedure. Duloxetine is a non-opioid drug known for managing neuropathic pain and has both central and peripheral analgesic effects. The placebo is designed to look identical to duloxetine to maintain blinding. Patients will be monitored closely during and after the procedure to assess outcomes. During the study, researchers will observe whether pancreatitis develops within 24 hours after ERCP, which is the primary outcome being measured. Participants must have normal blood amylase and lipase levels before the procedure. The study includes monitoring for safety and adverse effects during the follow-up period. The total participation duration is based on the timing around the ERCP procedure and the subsequent 24-hour monitoring window.

Researchers are evaluating the use of Granulocyte Colony Stimulating Factor (GCSF) in infants with type 3 biliary atresia after the Kasai procedure. Biliary atresia is a serious liver condition in infants causing bile duct damage and blockage, often leading to liver failure and the need for transplantation. This study aims to assess how GCSF affects clinical and biochemical outcomes in this population, focusing on liver health and disease progression. Participants will be divided into two groups: one group of 20 infants will receive subcutaneous GCSF at a dose of 10 micrograms per kilogram daily for three days, starting 3 to 4 days after the Kasai surgery, while the other group of 20 infants will not receive GCSF. The study compares these two groups to see if GCSF helps improve liver function and reduce complications related to biliary atresia. During the study, researchers will monitor participants for changes in total bilirubin levels at three months and the frequency of cholangitis episodes at six months. Various clinical and laboratory assessments will be conducted to evaluate liver function and inflammation. The study will continue until October 2026, with regular follow-ups to observe the infants' health and disease outcomes.