[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"biliary-atresia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:biliary-atresia":26},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,19,0,[8,50,76,93,120,144,168,189,220,252,274,295,316,342,750,773,801,823,844],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":16,"sex":17,"minAge":4,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":23,"conditions":24,"keywords":28,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":38,"lastUpdatePostDateStruct":39,"startDateStruct":42,"completionDateStruct":44,"leadSponsor":46,"locationsCount":49},"100053278","the-dirty-nappy-study-100053278",false,"NCT07697872","The Dirty Nappy Study","The Dirty Nappy Study: Development and Evaluation of a Machine Learning Algorithm for the Early Detection of Cholestasis and Biliary Atresia From Parent-Provided Images of Dirty Nappies","Inclusion Criteria:\n\nAll Arms:\n\nInformed Consent: Parents\u002Fguardians can give informed consent for their child's participation in the study and can understand written English.\n\n1. Retrospective Cholestasis Arm:\n\n   All infants, of any gestation, male and female who were referred to Birmingham Liver Unit from any hospital for investigation of suspected cholestasis within the previous 10 years before the study launch. At the time of the referral to Birmingham Liver Unit the children were under the age of 6 months and the children were referred before the start date of the study.\n2. Prospective Cholestasis Arm:\n\n   All infants, of any gestation, male and female who were referred to Birmingham Liver Unit from any hospital for investigation of suspected cholestasis after the study launch. At the time of the referral to Birmingham Liver Unit the children were under the age of 6 months.\n3. Prospective Non-Cholestasis Arm All infants, of any gestation, male and female \\& aged less than 28 days, not thought to be cholestatic at the time of recruitment \\& with the birth registered at any of the participating hospital trusts.\n\nExclusion Criteria:\n\nIf the inclusion criteria as outlined are met, there is no exclusion criteria.",true,"ALL","11 Years",{"count":20,"type":21},5350,"ESTIMATED","OBSERVATIONAL","This observational study evaluates whether a machine-learning algorithm, a computer program that learns patterns from data, can accurately diagnose cholestasis in newborns. Cholestasis refers to reduced or blocked bile flow from the liver, which can lead to liver damage. A severe form of cholestasis is biliary atresia, a condition where the bile ducts are damaged or absent, requiring early treatment to prevent long-term harm.\n\nThe study involves infants from birth, both healthy and those potentially affected by cholestasis, recruited from four UK hospitals. It addresses two primary aims:\n\n* Accuracy of Diagnosis: Can the machine-learning algorithm accurately identify cholestasis and biliary atresia using parent-provided stool images? This will be assessed by measuring sensitivity (the ability to correctly detect true cases) and specificity (the ability to correctly identify infants without the condition).\n* Feasibility of Screening: Is using parent-provided images a feasible and acceptable screening method for early detection?\n\nTo evaluate these aims, researchers will compare two groups:\n\n* Infants with abnormal stool images who are subsequently diagnosed with cholestasis or biliary atresia.\n* Infants with normal stool images who do not develop biliary atresia.\n\nThis comparison will help determine the algorithm's ability to distinguish between infants with and without these conditions.\n\nParents will:\n\n* Take smartphone photos of their baby's dirty diapers at 14, 21, and 28 days of age.\n* Upload the images for analysis by the algorithm.\n* Provide feedback on their experience with this screening process.\n\nThe study seeks to determine if parent-submitted stool images can serve as a practical early screening tool for cholestasis, potentially enabling faster diagnosis and improved outcomes for affected infants.",[25,26,27],"Cholestasis in Newborn","Biliary Atresia","Neonatal Cholestasis",[29,30,31,32,33,34,35,36],"Neonatal cholestasis","Biliary atresia diagnosis","Newborn stool analysis","infant liver disease","screening","machine learning","AI","image analysis","RECRUITING","2026-07-06",{"date":40,"type":41},"2026-07-13","ACTUAL",{"date":43,"type":41},"2025-04-01",{"date":45,"type":21},"2026-09-30",{"name":47,"class":48},"Birmingham Women's and Children's NHS Foundation Trust","OTHER",1,{"id":51,"slug":52,"hasResults":11,"nctId":53,"briefTitle":54,"officialTitle":55,"acronym":56,"eligibilityCriteria":57,"healthyVolunteers":11,"sex":17,"minAge":58,"maxAge":59,"enrollmentInfo":60,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":62,"conditions":63,"keywords":64,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":66,"lastUpdatePostDateStruct":67,"startDateStruct":69,"completionDateStruct":71,"leadSponsor":73,"locationsCount":75},"100083583","biliary-atresia-study-in-infants-and-children-100083583","NCT00345553","Biliary Atresia Study in Infants and Children","Biliary Atresia Study in Infants and Children (BASIC)","BASIC","Inclusion Criteria:\n\n1. Participants need to have a confirmed diagnosis of BA determined by chart review including review of pertinent diagnostic biopsy reports, radiologic reports and surgical reports (if surgery was performed).\n2. Participants need to be \\>6 months of age up to and equal to the age of 20 (participants enrolled at 20 years of age will have one visit).\n3. Participants with their native liver.\n4. Parent, guardian or participant (if 18 years of age or older) is willing to provide informed consent and, when appropriate, the participant is willing to assent.\n\nExclusion Criteria:\n\n1. Currently participating in the ChiLDReN study PROBE.\n2. Inability to confirm original diagnostic evaluation of biliary atresia.\n3. Inability or unwillingness of family or participant to participate in all scheduled visits.\n4. History of liver transplantation.","6 Months","20 Years",{"count":61,"type":21},1265,"Little is known about the factors that cause biliary atresia nor the factors that influence disease progression. The purpose of this study is to collect the pertinent clinical information, genetic material and body fluid samples to enable investigators to address the following aims: To identify the gene or genes implicated in the etiology of BA; To characterize the natural history of the older, non-transplanted child with BA.",[26],[26,65],"Cholestasis","2026-06-03",{"date":68,"type":41},"2026-06-04",{"date":70,"type":41},"2006-05-16",{"date":72,"type":21},"2029-05-31",{"name":74,"class":48},"Arbor Research Collaborative for Health",16,{"id":77,"slug":78,"hasResults":11,"nctId":79,"briefTitle":80,"officialTitle":81,"acronym":82,"eligibilityCriteria":83,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":58,"enrollmentInfo":84,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":86,"conditions":87,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":66,"lastUpdatePostDateStruct":88,"startDateStruct":89,"completionDateStruct":91,"leadSponsor":92,"locationsCount":75},"100062542","a-prospective-database-of-infants-with-cholestasis-100062542","NCT00061828","A Prospective Database of Infants With Cholestasis","Childhood Liver Disease Research Network (ChiLDReN): A Prospective Database of Infants With Cholestasis","PROBE","INCLUSION CRITERIA\n\n* Infant's age less than or equal to 180 days at initial presentation at the ChiLDReN clinical site.\n* Diagnosis of cholestasis defined by serum direct or conjugated bilirubin greater than or equal to 2 mg\u002Fdl and suspected biliary atresia.\n* The subject's parent(s)\u002Fguardian(s) willing to provide informed written consent.\n\nEXCLUSION CRITERIA\n\n* Acute liver failure.\n* Previous hepatobiliary surgery with dissection or excision of biliary tissue.\n* Diagnoses of bacterial or fungal sepsis (except where associated with metabolic liver disease)\n* Diagnoses of hypoxia, shock or ischemic hepatopathy within the past two weeks (If the cholestasis persists beyond two weeks of the initiating event, the infant can be enrolled).\n* Diagnosis of any malignancy.\n* Presence of any primary hemolytic disease (except when diagnosed with biliary atresia or another cholestatic disease being studied by ChiLDREN).\n* Diagnosis of any drug or Total parenteral nutrition (TPN)-associated cholestasis (except when diagnosed with biliary atresia or another cholestatic disease being studied by ChiLDREN).\n* Diagnosis with Extracorporeal membrane oxygenation (ECMO)-associated cholestasis.\n* Birth weight less than 1500g (except when diagnosed with biliary atresia).",{"count":85,"type":21},1000,"Biliary atresia, idiopathic neonatal hepatitis, and specific genetic cholestatic conditions are the most common causes of jaundice and hyperbilirubinemia that continue beyond the newborn period. The long term goal of the Childhood Liver Disease Research Network (ChiLDReN) is to establish a database of clinical information and plasma, serum, and tissue samples from cholestatic children to facilitate research and to perform clinical, epidemiological and therapeutic trials in these important pediatric liver diseases.",[26],{"date":68,"type":41},{"date":90,"type":41},"2004-04-21",{"date":72,"type":21},{"name":74,"class":48},{"id":94,"slug":95,"hasResults":11,"nctId":96,"briefTitle":97,"officialTitle":98,"acronym":4,"eligibilityCriteria":99,"healthyVolunteers":11,"sex":17,"minAge":100,"maxAge":101,"enrollmentInfo":102,"targetDuration":4,"studyType":104,"phases":105,"briefSummary":107,"conditions":108,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":111,"lastUpdatePostDateStruct":112,"startDateStruct":114,"completionDateStruct":116,"leadSponsor":118,"locationsCount":49},"100510351","preventive-effect-of-prophylactic-oral-antibiotics-against-cholangitis-after-kasai-portoenterostomy-100510351","NCT05925309","Preventive Effect of Prophylactic Oral Antibiotics Against Cholangitis After Kasai Portoenterostomy","Preventive Effect of Prophylactic Oral Antibiotics Against Cholangitis After Kasai Portoenterostomy in Biliary Atresia: a Randomized Controlled Trial","Inclusion Criteria:\n\n* Patients whose age of operation is 14-90 d. Sex and race are not restricted;\n* Patients who are born with gestational age older than 36 weeks;\n* Patients whose body weight before operation \\> 2 kg;\n* Patients diagnosed of type-III BA and underwent KP in Children's Hospital of Fudan University;\n* The type-III BA diagnosis is based on cholangiography or operation;\n* Patients whose histological features of liver biopsies are reported. HE staining and Masson staining are required, and edema, inflammation, fibrosis, and hyperplasia of intrahepatic bile duct should be reported;\n* Patients who are not allergic to postoperative medications;\n* Patients who haven't accepted other antibiotic or probiotic therapy.\n\nExclusion Criteria:\n\n* Patients with cholestasis of non-BA disease;\n* Patients who have undergone KP at other institutions;\n* Patients whose pathohistological diagnosis is in doubt;\n* Patients who undergo liver transplantation immediately after KP;\n* Patients with other liver diseases or severe complications (e.g., severe pulmonary hypertension, renal failure, intracranial hemorrhage, etc.) requiring surgical intervention or other medical therapy;\n* Patients with severe cardiac, renal, or central nerve system malformations (e.g., tetralogy of Fallot, transposition of the great arteries, cerebral dysplasia, etc.) and have poor prognosis;\n* Patients judged by the researchers that they can not comply with the study requirements.","14 Days","90 Days",{"count":103,"type":21},356,"INTERVENTIONAL",[106],"NA","This study is non-inferiority trial design. This study aimed to investigate the effect of prophylactic oral antibiotics on preventing cholangitis in biliary atresia (BA) patients after Kasai portoenterostomy (KP) by comparing the cholangitis rate in BA patients who received prophylactic oral antibiotics and those who did not. The patients were followed up for 2 years after KP.",[26,109,110],"Cholangitis","Anti-Bacterial Agents","2026-05-12",{"date":113,"type":41},"2026-05-15",{"date":115,"type":41},"2023-07-01",{"date":117,"type":21},"2029-12-31",{"name":119,"class":48},"Children's Hospital of Fudan University",{"id":121,"slug":122,"hasResults":11,"nctId":123,"briefTitle":124,"officialTitle":124,"acronym":4,"eligibilityCriteria":125,"healthyVolunteers":11,"sex":17,"minAge":126,"maxAge":127,"enrollmentInfo":128,"targetDuration":4,"studyType":104,"phases":130,"briefSummary":133,"conditions":134,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":135,"lastUpdatePostDateStruct":136,"startDateStruct":138,"completionDateStruct":140,"leadSponsor":142,"locationsCount":49},"100632388","phase-1-use-of-indocyanine-green-icg-for-the-diagnosis-of-biliary-atresia-100632388","NCT07513038","Use of Indocyanine Green (ICG) for the Diagnosis of Biliary Atresia","Inclusion Criteria:\n\n1. age \\\u003C3 months viable infants\n2. cholestasis (defined as direct bilirubin \\>1)\n3. undergoing diagnostic HIDA scan to evaluate for biliary atresia\n\nExclusion Criteria\n\n1. kidney disease (eGFR \\\u003C60 mL\u002Fmin\u002F1.73m2)\n2. grossly bloody stools\n3. history of bowel surgery or disease\n4. history of allergic reaction to ICG, iodine, or shellfish\n5. unable to provide informed consent","0 Days","3 Months",{"count":129,"type":21},20,[131,132],"PHASE1","PHASE2","Biliary atresia is a rare, progressive liver disease that only affects infants. It leads to complete obstruction and scarring of the bile duct. Current non-invasive diagnostic tests have limited sensitivity.\n\nIndocyanine Green (ICG) is a non-radioactive, fluorescent compound with several clinical applications including angiography for ophthalmologic testing, visualization during surgery, and measurement of liver function. After excitation with near infrared light (750-810 nm), ICG emits near infrared light at 850 nm, which can be detected by a special camera. ICG is taken up by the liver exclusively and excreted in the bile, where it is removed from the body in the stool. The hypothesis is that after injection of ICG, participants with biliary atresia will not have any fluorescence detected in the stool. Investigators aim to use ICG as a functional test of bile duct patency in participants with cholestasis being evaluated for biliary atresia.",[26],"2026-04-03",{"date":137,"type":41},"2026-04-06",{"date":139,"type":41},"2024-08-01",{"date":141,"type":21},"2026-12",{"name":143,"class":48},"Boston Children's Hospital",{"id":145,"slug":146,"hasResults":11,"nctId":147,"briefTitle":148,"officialTitle":149,"acronym":4,"eligibilityCriteria":150,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":127,"enrollmentInfo":151,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":153,"conditions":154,"keywords":158,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":161,"lastUpdatePostDateStruct":162,"startDateStruct":163,"completionDateStruct":165,"leadSponsor":167,"locationsCount":49},"100444837","high-medium-chain-triglyceride-nutritional-support-in-infants-with-biliary-atresia-100444837","NCT05072626","High Medium-chain Triglyceride Nutritional Support in Infants With Biliary Atresia","Study on High Medium-chain Triglyceride Nutritional Support in Infants With Biliary Atresia After Kasai Portoenterostomy","Inclusion Criteria:\n\nThe Kasai procedure for infants with biliary atresia under the age of 3 months.\n\nExclusion Criteria:\n\n* Complicated with cirrhosis, hepatitis, or other hepatic disorders;\n* Complicated with other systemic serious diseases (such as congenital multiple malformations, chromosome abnormalities)",{"count":152,"type":21},300,"This study is a prospective, single center and observational open clinical study.",[26,155,156,157],"Infant","Nutrition Support","Medium-chain Triglyceride",[26,159,156,160],"Kasai portoenterostomy","medium-chain triglyceride","2026-03-30",{"date":135,"type":41},{"date":164,"type":41},"2021-10-11",{"date":166,"type":21},"2028-12-31",{"name":119,"class":48},{"id":169,"slug":170,"hasResults":11,"nctId":171,"briefTitle":172,"officialTitle":172,"acronym":4,"eligibilityCriteria":173,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":174,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":176,"conditions":177,"keywords":179,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":181,"lastUpdatePostDateStruct":182,"startDateStruct":184,"completionDateStruct":186,"leadSponsor":188,"locationsCount":49},"100504438","preoperative-serum-fgf19-in-the-prognosis-of-biliary-atresia-100504438","NCT05848310","Preoperative Serum FGF19 in the Prognosis of Biliary Atresia","Inclusion Criteria:\n\n* Samples diagnosed as biliary atresia in Children's Hospital of Fudan University\n* Samples with one-year follow-up records after surgery\n* Samples with a record of serum total bilirubin levels three months after surgery\n* Samples with preoperative serum left（volume \\> 500ul)\n\nExclusion Criteria:\n\n* None",{"count":175,"type":21},200,"To investigate the role of preoperative serum FGF19 level in the prognosis of biliary atresia.",[26,178],"Prognosis",[26,180],"FGF19","2026-03-24",{"date":183,"type":41},"2026-03-27",{"date":185,"type":41},"2023-10-15",{"date":187,"type":21},"2027-08",{"name":119,"class":48},{"id":190,"slug":191,"hasResults":11,"nctId":192,"briefTitle":193,"officialTitle":194,"acronym":4,"eligibilityCriteria":195,"healthyVolunteers":11,"sex":17,"minAge":196,"maxAge":197,"enrollmentInfo":198,"targetDuration":4,"studyType":104,"phases":200,"briefSummary":201,"conditions":202,"keywords":203,"overallStatus":210,"whyStopped":4,"lastUpdateSubmitDate":211,"lastUpdatePostDateStruct":212,"startDateStruct":214,"completionDateStruct":216,"leadSponsor":218,"locationsCount":4},"100536110","phase-1-tolerability-of-enteral-nac-in-infants-100536110","NCT06260566","Tolerability of Enteral NAC in Infants","A Phase 1 Trial of Tolerability of Enteral N-Acetylcysteine in Infants","Inclusion Criteria:\n\n* 122-273 days of life at time of enrollment\n* Confirmed diagnosis of biliary atresia based on intraoperative cholangiogram\n* Able to tolerate oral nutrition and medications and not on continuous tube feeds\n* Anticipated inpatient admission of at least 4 days\n* Legal guardian(s) consent to study enrollment after understanding the risks and investigational nature of the study\n\nExclusion Criteria:\n\n* Gestational age of \\\u003C32 weeks at birth\n* Inability or contraindication to taking oral nutrition\n* Neonatal intensive care unit admission\n* Short bowel, or other malabsorptive, syndrome\n* Decompensated liver disease (INR \\> 1.3 despite vitamin K administration)\n* Active respiratory infection\n* Severe concurrent illnesses that would interfere with the conduct and\u002For results of the study\n* Concurrent participation in another drug trial","122 Days","273 Days",{"count":199,"type":21},12,[131],"Biliary atresia (BA) is a neonatal liver disease characterized by impaired bile flow and is the most common indication for pediatric liver transplantation. BA can be treated with the Kasai portoenterostomy (KP), a procedure that attempts to restore bile flow and slow disease progression. However, success of the KP procedure is quite variable, and lack of adjuvant medical therapies following KP is a major gap in pediatric hepatology.\n\nThis study begins to explore oral N-acetylcysteine (NAC) as a potential medical therapy in BA by determining whether an oral formulation can be given to infants. The primary objective is to determine tolerability of the oral NAC formulation. The primary outcome is tolerating at least 3 out of 4 total doses without emesis. The Bayesian Optimal Interval Design (BOIN) trial design will be used to determine the maximum tolerated dose of oral NAC. Our secondary objective is to assess palatability of the oral NAC formulation by comparing facial expressions when taking oral NAC versus other medications commonly given to cholestatic infants.",[26],[26,204,205,206,207,208,209],"N-Acetylcysteine","glutathione","bile flow","serum bilirubin","serum bile acids","cholestasis","NOT_YET_RECRUITING","2026-03-02",{"date":213,"type":41},"2026-03-04",{"date":215,"type":21},"2026-10",{"date":217,"type":21},"2031-10",{"name":219,"class":48},"Sanjiv Harpavat",{"id":221,"slug":222,"hasResults":11,"nctId":223,"briefTitle":224,"officialTitle":224,"acronym":4,"eligibilityCriteria":225,"healthyVolunteers":16,"sex":17,"minAge":226,"maxAge":227,"enrollmentInfo":228,"targetDuration":4,"studyType":104,"phases":230,"briefSummary":232,"conditions":233,"keywords":236,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":243,"lastUpdatePostDateStruct":244,"startDateStruct":246,"completionDateStruct":248,"leadSponsor":250,"locationsCount":49},"100612228","early-phase-1-the-use-of-near-infrared-fluorescence-cholangiography-with-indocyanine-green-icg-in-the-work-up-of-neonatal-cholestasis-100612228","NCT07250854","The Use of Near-Infrared Fluorescence Cholangiography With Indocyanine Green (ICG) in the Work Up of Neonatal Cholestasis","Inclusion Criteria:\n\n* Infants admitted to the hospital (no matter their age, sex, race\u002Fethnicity) who a hepatologist deems warrants inpatient work up of neonatal cholestasis suspected to be due to biliary atresia.\n\nExclusion Criteria:\n\n* Infants that a hepatologist has not deemed to warrant inpatient admission for work up of neonatal cholestasis suspected to be due to biliary atresia.\n* Infants that are managed in the outpatient setting\n* Non-cholestatic infants\n* Patient who are on TPN AND NPO for reasons other than temporary imaging or surgical requirements\n* Infant with a history of bowel resection or other surgical procedures known to introduce blood into the gastrointestinal tract.","2 Weeks","4 Months",{"count":229,"type":21},15,[231],"EARLY_PHASE1","In infants that present with findings concerning for biliary atresia, along with other cholestatic work up which is standard, they will receive a one-time intravenous (IV) dose of Indocyanine Green (ICG). The infant's diapers will subsequently be examined for presence of the ICG, and if present, suggests bile flow. This was described as 97% accurate for assessing biliary patency and we would like to perform a similar study to assess biliary patency in the work up of neonatal cholestasis.",[26,234,25,235],"Kasai","Cholestasis in Newborn Infant",[237,238,209,239,240,241,242],"biliary atresia","kasai","cholestasis in neonate","cholestasis in newborn","direct bilirubin","acholic stool","2026-02-16",{"date":245,"type":41},"2026-02-18",{"date":247,"type":41},"2026-01-23",{"date":249,"type":21},"2027-03",{"name":251,"class":48},"University of Alabama at Birmingham",{"id":253,"slug":254,"hasResults":11,"nctId":255,"briefTitle":256,"officialTitle":257,"acronym":4,"eligibilityCriteria":258,"healthyVolunteers":11,"sex":17,"minAge":259,"maxAge":127,"enrollmentInfo":260,"targetDuration":4,"studyType":104,"phases":262,"briefSummary":263,"conditions":264,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":266,"lastUpdatePostDateStruct":267,"startDateStruct":269,"completionDateStruct":271,"leadSponsor":272,"locationsCount":49},"100574807","nutritional-intervention-for-biliary-atresia-100574807","NCT06764082","Nutritional Intervention for Biliary Atresia","Nutritional Intervention With High Chain Triglyceride Formulation During Perioperative Period of Kasai Surgery for Biliary Atresia: a Prospective Multicenter Trial","Inclusion Criteria:\n\n* Infants under 3 months old;\n* Children clinically diagnosed with biliary atresia and in need of Kasai surgery;\n* The patient themselves, legal representative or guardian have signed the informed consent form and are willing to actively cooperate with treatment and follow-up.\n\nExclusion Criteria:\n\n* Low birth weight infants or very low birth weight infants;\n* Complicated with life-threatening diseases of various organ systems;\n* Complicated with other severe digestive tract malformations or other diseases that may interfere with the treatment of the patient or the patient's compliance;\n* Patients who have participated in other clinical trials within the last month;\n* Any other conditions that the researcher deems unsuitable for participation in this trial.","1 Day",{"count":261,"type":21},120,[106],"Infants with biliary atresia (Biliary atresia, BA) have an increased risk of malnutrition due to insufficient dietary intake to maintain normal growth, impaired intestinal absorption, increased metabolic rate, and damage to some liver macronutrient metabolic pathways. The medium-chain triglyceride formula (MCT) in enteral nutrition has advantages: (1) It has a fast metabolism in the liver and possesses the advantage of being an innate energy source; (2) It can share metabolic pathways with some other fatty acids (DHA, EPA), and can promote the synthesis of phospholipids, etc. Therefore, EN containing the MCT formula is regarded as an important approach to alleviate growth retardation in BA children and improve the nutritional status of patients. This study aims to observe the effect of intensified enteral feeding with a high MCT formula during the perioperative period compared to traditional oral feeding on the prognosis of children with biliary atresia. The method adopted is a prospective, two-arm, open-label, multicenter, and interventional real-world study.",[265,26],"Nutrition Disorder, Infant","2025-12-15",{"date":268,"type":41},"2025-12-22",{"date":270,"type":41},"2025-02-01",{"date":166,"type":21},{"name":273,"class":48},"Tongji Hospital",{"id":275,"slug":276,"hasResults":11,"nctId":277,"briefTitle":278,"officialTitle":279,"acronym":280,"eligibilityCriteria":281,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":282,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":284,"conditions":285,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":286,"lastUpdatePostDateStruct":287,"startDateStruct":289,"completionDateStruct":291,"leadSponsor":293,"locationsCount":49},"100306732","mapping-disease-pathways-for-biliary-atresia-100306732","NCT03273049","Mapping Disease Pathways for Biliary Atresia","Coordinating Center- Mapping Disease Pathways for Biliary Atresia","BA","Inclusion Criteria:\n\n* living individuals who were diagnosed with Biliary Atresia and received or are about to receive a liver transplant from multiple participating centers (Children's Hospital of Pittsburgh, Kings College Hospital, Children's Hospital of Birmingham, and Hospital Sírio-Libanês).\n\nExclusion Criteria:\n\n* No child participant in the care of the state will be enrolled, nor will patients in the care of temporary or informal guardians be enrolled",{"count":283,"type":21},1100,"This project will primarily evaluate the developmental\u002Fgenetic basis of biliary atresia, the most common cause of liver failure at birth, and which accounts of half of all liver transplants performed worldwide in children.",[26],"2025-09-05",{"date":288,"type":41},"2025-09-09",{"date":290,"type":41},"2016-07-21",{"date":292,"type":21},"2035-07-21",{"name":294,"class":48},"University of Pittsburgh",{"id":296,"slug":297,"hasResults":11,"nctId":298,"briefTitle":299,"officialTitle":300,"acronym":4,"eligibilityCriteria":301,"healthyVolunteers":11,"sex":17,"minAge":259,"maxAge":101,"enrollmentInfo":302,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":304,"conditions":305,"keywords":4,"overallStatus":210,"whyStopped":4,"lastUpdateSubmitDate":307,"lastUpdatePostDateStruct":308,"startDateStruct":310,"completionDateStruct":312,"leadSponsor":314,"locationsCount":4},"100603684","stool-card-in-biliary-atresia-100603684","NCT07139717","Stool Card in Biliary Atresia","Evaluation of the Effectiveness of Stool Color Card in the Early Detection of Biliary Atresia: A Prospective Study.","Inclusion Criteria:\n\n1. Neonates and infants aged 1-90 days old.\n2. Neonates and infants with direct (conjugated) hyperbilirubinemia (Conjugated hyperbilirubinemia is generally defined as a conjugated or direct bilirubin level greater than 1 mg\u002FdL when the total bilirubin is less than 5 mg\u002FdL or more than 20% of the total bilirubin if the total bilirubin is greater than 5 mg\u002FdL) \\[12\\]\n3. No contraindication for liver biopsy as considered the gold standard for diagnosis of biliary atresia e.g. coagulopathy.\n\nExclusion Criteria:\n\n1 - Infants diagnosed with biliary atresia or other cholestatic liver diseases prior to enrollment.\n\n2-Infants aged more than 90 days 3-Neonates and infants with indirect hyperbilirubinemia. 4-Those with contraindication to do liver biopsy",{"count":303,"type":21},100,"To evaluate the effectiveness of using a stool color card as a non-invasive screening tool for the early detection of biliary atresia, with the objective of improving early diagnosis rates and facilitating timely surgical intervention.",[306,26],"Stool Color Card","2025-08-18",{"date":309,"type":41},"2025-08-24",{"date":311,"type":21},"2025-09-01",{"date":313,"type":21},"2026-12-01",{"name":315,"class":48},"Assiut University",{"id":317,"slug":318,"hasResults":11,"nctId":319,"briefTitle":320,"officialTitle":321,"acronym":4,"eligibilityCriteria":322,"healthyVolunteers":11,"sex":17,"minAge":126,"maxAge":101,"enrollmentInfo":323,"targetDuration":4,"studyType":104,"phases":325,"briefSummary":326,"conditions":327,"keywords":4,"overallStatus":210,"whyStopped":4,"lastUpdateSubmitDate":333,"lastUpdatePostDateStruct":334,"startDateStruct":336,"completionDateStruct":338,"leadSponsor":340,"locationsCount":49},"100593804","phase-1-clinical-outcomes-of-early-kasai-surgery-with-umbilical-cord-mscs-in-biliary-atresia-100593804","NCT07011199","Clinical Outcomes of Early Kasai Surgery With Umbilical Cord MSCs in Biliary Atresia","Evaluation of Clinical Outcomes in Biliary Atresia Patients Undergoing Kasai Surgery Before 90 Days of Age With Umbilical Cord-Derived Mesenchymal Stem Cell Therapy","Inclusion Criteria:\n\n* Infants diagnosed with biliary atresia confirmed by intraoperative findings.\n* Underwent Kasai portoenterostomy at an age less than 90 days.\n* Parents or legal guardians have provided informed consent to participate in the study and follow all study procedures.\n\nExclusion Criteria:\n\n* Infants with severe malnutrition.\n* Infants with major congenital anomalies other than biliary atresia.\n* Infants with positive tumor markers.",{"count":324,"type":21},14,[131],"The goal of this clinical trial with historical control is to evaluate whether umbilical cord-derived mesenchymal stem cell (UC-MSC) therapy can improve clinical outcomes in infants with biliary atresia undergoing Kasai surgery before 90 days of age.\n\nThe main questions it aims to answer are:\n\nDoes UC-MSC therapy improve liver function parameters (bilirubin, albumin, liver enzymes, coagulation profile)? Does UC-MSC therapy reduce complications such as anemia, ascites, jaundice, and improve PELD scores? Does UC-MSC therapy improve overall survival compared to standard Kasai surgery alone? Researchers will compare the group receiving UC-MSC in 2025-2027 with a historical control group of patients who previously underwent Kasai surgery without UC-MSC therapy.\n\nParticipants will:\n\nUndergo preoperative evaluation, including laboratory and imaging tests. Receive Kasai surgery combined with intraoperative trans-portal vein injection of 20 million UC-MSCs.\n\nBe monitored postoperatively through serial laboratory tests, imaging (Fibroscan), and clinical assessments at scheduled intervals.\n\nBe followed up for potential serious adverse events and survival outcomes.",[26,328,329,330,331,332],"Kasai Portoenterostomy","Mesenchymal Stem Cell Transplantation","Liver Function Disorders","Liver Fibrosis","Survival Rate","2025-05-30",{"date":335,"type":41},"2025-06-08",{"date":337,"type":21},"2025-06-17",{"date":339,"type":21},"2026-04-17",{"name":341,"class":48},"Universitas Diponegoro",{"id":343,"slug":344,"hasResults":11,"nctId":345,"briefTitle":346,"officialTitle":347,"acronym":348,"eligibilityCriteria":349,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":350,"targetDuration":352,"studyType":22,"phases":4,"briefSummary":353,"conditions":354,"keywords":697,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":740,"lastUpdatePostDateStruct":741,"startDateStruct":743,"completionDateStruct":745,"leadSponsor":747,"locationsCount":749},"100193378","rare-disease-patient-registry--natural-history-study---coordination-of-rare-diseases-at-sanford-100193378","NCT01793168","Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford","Coordination of Rare Diseases at Sanford","CoRDS","Inclusion Criteria:\n\n* Diagnosis of a rare disease, a disease of unknown prevalence, undiagnosed or an unaffected carrier of a rare\u002Funcommon disease\n\nExclusion Criteria:\n\n* Diagnosis of a disease which is not rare",{"count":351,"type":21},20000,"100 Years","CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases. This program allows patients and researchers to connect as easily as possible to help advance treatments and cures for rare diseases. The CoRDS team works with patient advocacy groups, individuals and researchers to help in the advancement of research in over 7,000 rare diseases. The registry is free for patients to enroll and researchers to access. Visit sanfordresearch.org\u002FCoRDS to enroll.",[355,356,357,358,359,360,361,362,363,364,365,366,367,368,369,370,371,372,373,374,375,376,377,378,379,380,381,382,383,384,385,386,387,388,389,390,391,392,393,394,395,396,397,398,399,400,401,402,403,404,405,406,407,408,409,410,411,412,413,414,415,416,417,418,419,420,421,422,423,424,425,426,427,428,429,430,431,432,433,434,435,436,437,438,439,440,441,442,443,444,445,446,447,448,449,450,451,452,453,454,455,456,457,458,459,460,461,462,463,464,465,466,467,468,469,470,471,472,473,474,475,476,477,478,479,480,481,482,483,484,485,486,487,488,489,490,491,492,493,494,495,496,497,498,499,500,501,502,503,504,505,506,507,508,509,510,511,512,513,514,515,516,517,518,519,520,521,522,523,524,525,526,527,528,529,530,531,532,533,534,535,536,537,538,539,540,541,542,543,544,545,546,547,548,549,550,551,552,553,554,555,556,557,558,559,560,561,562,563,564,565,566,567,568,569,570,571,572,573,574,575,576,577,578,579,580,581,582,583,584,585,586,587,588,589,590,591,592,593,594,595,596,597,598,599,600,601,602,603,604,605,606,607,608,609,610,611,612,613,614,615,616,617,618,619,620,621,622,623,624,625,626,627,628,629,630,631,632,633,634,635,636,637,638,639,640,641,642,643,644,645,646,647,648,649,650,651,652,653,654,655,656,657,658,659,660,661,662,663,664,665,666,26,667,668,669,670,671,672,673,674,675,676,677,678,679,680,681,682,683,684,685,686,687,688,689,690,691,692,693,694,695,696],"Rare Disorders","Undiagnosed Disorders","Disorders of Unknown Prevalence","Cornelia De Lange Syndrome","Prenatal Benign Hypophosphatasia","Perinatal Lethal Hypophosphatasia","Odontohypophosphatasia","Adult Hypophosphatasia","Childhood-onset Hypophosphatasia","Infantile Hypophosphatasia","Hypophosphatasia","Kabuki Syndrome","Bohring-Opitz Syndrome","Narcolepsy Without Cataplexy","Narcolepsy-cataplexy","Hypersomnolence Disorder","Idiopathic Hypersomnia Without Long Sleep Time","Idiopathic Hypersomnia With Long Sleep Time","Idiopathic Hypersomnia","Kleine-Levin Syndrome","Kawasaki Disease","Leiomyosarcoma","Leiomyosarcoma of the Corpus Uteri","Leiomyosarcoma of the Cervix Uteri","Leiomyosarcoma of Small Intestine","Acquired Myasthenia Gravis","Addison Disease","Hyperacusis (Hyperacousis)","Juvenile Myasthenia Gravis","Transient Neonatal Myasthenia Gravis","Williams Syndrome","Lyme Disease","Myasthenia Gravis","Marinesco Sjogren Syndrome(Marinesco-Sjogren Syndrome)","Isolated Klippel-Feil Syndrome","Frasier Syndrome","Denys-Drash Syndrome","Beckwith-Wiedemann Syndrome","Emanuel Syndrome","Isolated Aniridia","Axenfeld-Rieger Syndrome","Aniridia-intellectual Disability Syndrome","Aniridia - Renal Agenesis - Psychomotor Retardation","Aniridia - Ptosis - Intellectual Disability - Familial Obesity","Aniridia - Cerebellar Ataxia - Intellectual Disability","Aniridia - Absent Patella","Aniridia","Peters Anomaly - Cataract","Peters Anomaly","Potocki-Shaffer Syndrome","Silver-Russell Syndrome Due to Maternal Uniparental Disomy of Chromosome 11","Silver-Russell Syndrome Due to Imprinting Defect of 11p15","Silver-Russell Syndrome Due to 11p15 Microduplication","Syndromic Aniridia","WAGR Syndrome","Wolf-Hirschhorn Syndrome","4p16.3 Microduplication Syndrome","4p Deletion Syndrome, Non-Wolf-Hirschhorn Syndrome","Autosomal Recessive Stickler Syndrome","Stickler Syndrome Type 2","Stickler Syndrome Type 1","Stickler Syndrome","Mucolipidosis Type 4","X-linked Spinocerebellar Ataxia Type 4","X-linked Spinocerebellar Ataxia Type 3","X-linked Intellectual Disability - Ataxia - Apraxia","X-linked Progressive Cerebellar Ataxia","X-linked Non Progressive Cerebellar Ataxia","X-linked Cerebellar Ataxia","Vitamin B12 Deficiency Ataxia","Toxic Exposure Ataxia","Unclassified Autosomal Dominant Spinocerebellar Ataxia","Thyroid Antibody Ataxia","Sporadic Adult-onset Ataxia of Unknown Etiology","Spinocerebellar Ataxia With Oculomotor Anomaly","Spinocerebellar Ataxia With Epilepsy","Spinocerebellar Ataxia With Axonal Neuropathy Type 2","Spinocerebellar Ataxia Type 8","Spinocerebellar Ataxia Type 7","Spinocerebellar Ataxia Type 6","Spinocerebellar Ataxia Type 5","Spinocerebellar Ataxia Type 4","Spinocerebellar Ataxia Type 37","Spinocerebellar Ataxia Type 36","Spinocerebellar Ataxia Type 35","Spinocerebellar Ataxia Type 34","Spinocerebellar Ataxia Type 32","Spinocerebellar Ataxia Type 31","Spinocerebellar Ataxia Type 30","Spinocerebellar Ataxia Type 3","Spinocerebellar Ataxia Type 29","Spinocerebellar Ataxia Type 28","Spinocerebellar Ataxia Type 27","Spinocerebellar Ataxia Type 26","Spinocerebellar Ataxia Type 25","Spinocerebellar Ataxia Type 23","Spinocerebellar Ataxia Type 22","Spinocerebellar Ataxia Type 21","Spinocerebellar Ataxia Type 20","Spinocerebellar Ataxia Type 2","Spinocerebellar Ataxia Type 19\u002F22","Spinocerebellar Ataxia Type 18","Spinocerebellar Ataxia Type 17","Spinocerebellar Ataxia Type 16","Spinocerebellar Ataxia Type 15\u002F16","Spinocerebellar Ataxia Type 14","Spinocerebellar Ataxia Type 13","Spinocerebellar Ataxia Type 12","Spinocerebellar Ataxia Type 11","Spinocerebellar Ataxia Type 10","Spinocerebellar Ataxia Type 1 With Axonal Neuropathy","Spinocerebellar Ataxia Type 1","Spinocerebellar Ataxia - Unknown","Spinocerebellar Ataxia - Dysmorphism","Non Progressive Epilepsy and\u002For Ataxia With Myoclonus as a Major Feature","Spasticity-ataxia-gait Anomalies Syndrome","Spastic Ataxia With Congenital Miosis","Spastic Ataxia - Corneal Dystrophy","Spastic Ataxia","Rare Hereditary Ataxia","Rare Ataxia","Recessive Mitochondrial Ataxia Syndrome","Progressive Epilepsy and\u002For Ataxia With Myoclonus as a Major Feature","Posterior Column Ataxia - Retinitis Pigmentosa","Post-Stroke Ataxia","Post-Head Injury Ataxia","Post Vaccination Ataxia","Polyneuropathy - Hearing Loss - Ataxia - Retinitis Pigmentosa - Cataract","Muscular Atrophy - Ataxia - Retinitis Pigmentosa - Diabetes Mellitus","Non-hereditary Degenerative Ataxia","Paroxysmal Dystonic Choreathetosis With Episodic Ataxia and Spasticity","Olivopontocerebellar Atrophy - Deafness","NARP Syndrome","Myoclonus - Cerebellar Ataxia - Deafness","Multiple System Atrophy, Parkinsonian Type","Multiple System Atrophy, Cerebellar Type","Multiple System Atrophy","Maternally-inherited Leigh Syndrome","Machado-Joseph Disease Type 3","Machado-Joseph Disease Type 2","Machado-Joseph Disease Type 1","Leigh Syndrome","Late-onset Ataxia With Dementia","Infection or Post Infection Ataxia","GAD Ataxia","Hereditary Episodic Ataxia","Gliadin\u002FGluten Ataxia","Friedreich Ataxia","Fragile X-associated Tremor\u002FAtaxia Syndrome","Familial Paroxysmal Ataxia","Exposure to Medications Ataxia","Episodic Ataxia With Slurred Speech","Episodic Ataxia Unknown Type","Episodic Ataxia Type 7","Episodic Ataxia Type 6","Episodic Ataxia Type 5","Episodic Ataxia Type 4","Episodic Ataxia Type 3","Episodic Ataxia Type 1","Epilepsy and\u002For Ataxia With Myoclonus as Major Feature","Early-onset Spastic Ataxia-neuropathy Syndrome","Early-onset Progressive Neurodegeneration - Blindness - Ataxia - Spasticity","Early-onset Cerebellar Ataxia With Retained Tendon Reflexes","Early-onset Ataxia With Dementia","Childhood-onset Autosomal Recessive Slowly Progressive Spinocerebellar Ataxia","Dilated Cardiomyopathy With Ataxia","Cataract - Ataxia - Deafness","Cerebellar Ataxia, Cayman Type","Cerebellar Ataxia With Peripheral Neuropathy","Cerebellar Ataxia - Hypogonadism","Cerebellar Ataxia - Ectodermal Dysplasia","Cerebellar Ataxia - Areflexia - Pes Cavus - Optic Atrophy - Sensorineural Hearing Loss","Brain Tumor Ataxia","Brachydactyly - Nystagmus - Cerebellar Ataxia","Benign Paroxysmal Tonic Upgaze of Childhood With Ataxia","Autosomal Recessive Syndromic Cerebellar Ataxia","Autosomal Recessive Spastic Ataxia With Leukoencephalopathy","Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay","Autosomal Recessive Spastic Ataxia - Optic Atrophy - Dysarthria","Autosomal Recessive Spastic Ataxia","Autosomal Recessive Metabolic Cerebellar Ataxia","Autosomal Dominant Spinocerebellar Ataxia Due to Repeat Expansions That do Not Encode Polyglutamine","Autosomal Recessive Ataxia, Beauce Type","Autosomal Recessive Ataxia Due to Ubiquinone Deficiency","Autosomal Recessive Ataxia Due to PEX10 Deficiency","Autosomal Recessive Degenerative and Progressive Cerebellar Ataxia","Autosomal Recessive Congenital Cerebellar Ataxia Due to MGLUR1 Deficiency","Autosomal Recessive Congenital Cerebellar Ataxia Due to GRID2 Deficiency","Autosomal Recessive Congenital Cerebellar Ataxia","Autosomal Recessive Cerebellar Ataxia-pyramidal Signs-nystagmus-oculomotor Apraxia Syndrome","Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to WWOX Deficiency","Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to TUD Deficiency","Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to KIAA0226 Deficiency","Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome","Autosomal Recessive Cerebellar Ataxia With Late-onset Spasticity","Autosomal Recessive Cerebellar Ataxia Due to STUB1 Deficiency","Autosomal Recessive Cerebellar Ataxia Due to a DNA Repair Defect","Autosomal Recessive Cerebellar Ataxia - Saccadic Intrusion","Autosomal Recessive Cerebellar Ataxia - Psychomotor Retardation","Autosomal Recessive Cerebellar Ataxia - Blindness - Deafness","Autosomal Recessive Cerebellar Ataxia","Autosomal Dominant Spinocerebellar Ataxia Due to a Polyglutamine Anomaly","Autosomal Dominant Spinocerebellar Ataxia Due to a Point Mutation","Autosomal Dominant Spinocerebellar Ataxia Due to a Channelopathy","Autosomal Dominant Spastic Ataxia Type 1","Autosomal Dominant Spastic Ataxia","Autosomal Dominant Optic Atrophy","Ataxia-telangiectasia Variant","Ataxia-telangiectasia","Autosomal Dominant Cerebellar Ataxia, Deafness and Narcolepsy","Autosomal Dominant Cerebellar Ataxia Type 4","Autosomal Dominant Cerebellar Ataxia Type 3","Autosomal Dominant Cerebellar Ataxia Type 2","Autosomal Dominant Cerebellar Ataxia Type 1","Autosomal Dominant Cerebellar Ataxia","Ataxia-telangiectasia-like Disorder","Ataxia With Vitamin E Deficiency","Ataxia With Dementia","Ataxia - Oculomotor Apraxia Type 1","Ataxia - Other","Ataxia - Genetic Diagnosis - Unknown","Acquired Ataxia","Adult-onset Autosomal Recessive Cerebellar Ataxia","Alcohol Related Ataxia","Multiple Endocrine Neoplasia","Multiple Endocrine Neoplasia Type II","Multiple Endocrine Neoplasia Type 1","Multiple Endocrine Neoplasia Type 2","Multiple Endocrine Neoplasia, Type IV","Multiple Endocrine Neoplasia, Type 3","Multiple Endocrine Neoplasia (MEN) Syndrome","Multiple Endocrine Neoplasia Type 2B","Multiple Endocrine Neoplasia Type 2A","Atypical Hemolytic Uremic Syndrome","Atypical HUS","Wiedemann-Steiner Syndrome","Breast Implant-Associated Anaplastic Large Cell Lymphoma","Autoimmune\u002FInflammatory Syndrome Induced by Adjuvants (ASIA)","Hemophagocytic Lymphohistiocytosis","Behcet's Disease","Alagille Syndrome","Inclusion Body Myopathy With Early-onset Paget Disease and Frontotemporal Dementia (IBMPFD)","Lowe Syndrome","Pitt Hopkins Syndrome","1p36 Deletion Syndrome","Jansen Type Metaphyseal Chondrodysplasia","Cockayne Syndrome","Chronic Recurrent Multifocal Osteomyelitis","CRMO","Malan Syndrome","Hereditary Sensory and Autonomic Neuropathy Type Ie","VCP Disease","Hypnic Jerking","Sleep Myoclonus","Mollaret Meningitis","Recurrent Viral Meningitis","CRB1","Leber Congenital Amaurosis","Retinitis Pigmentosa","Rare Retinal Disorder","KCNMA1-Channelopathy","Primary Biliary Cirrhosis","ZMYND11","Transient Global Amnesia","Glycogen Storage Disease","Alstrom Syndrome","White Sutton Syndrome","DNM1","EIEE31","Myhre Syndrome","Recurrent Respiratory Papillomatosis","Laryngeal Papillomatosis","Tracheal Papillomatosis","Refsum Disease","Nicolaides Baraitser Syndrome","Leukodystrophy","Tango2","Cauda Equina Syndrome","Rare Gastrointestinal Disorders","Achalasia-Addisonian Syndrome","Achalasia Cardia","Achalasia Icrocephaly Syndrome","Anal Fistula","Congenital Sucrase-Isomaltase Deficiency","Eosinophilic Gastroenteritis","Idiopathic Gastroparesis","Hirschsprung Disease","Rare Inflammatory Bowel Disease","Intestinal Pseudo-Obstruction","Scleroderma","Short Bowel Syndrome","Sacral Agenesis","Sacral Agenesis Syndrome","Caudal Regression","Scheuermann Disease","SMC1A Truncated Mutations (Causing Loss of Gene Function)","Cystinosis","Juvenile Nephropathic Cystinosis","Nephropathic Cystinosis","Kennedy Disease","Spinal Bulbar Muscular Atrophy","Warburg Micro Syndrome","Mucolipidoses","Mitochondrial Diseases","Mitochondrial Aminoacyl-tRNA Synthetases","Mt-aaRS Disorders","Hypertrophic Olivary Degeneration","Non-Ketotic Hyperglycinemia","Fish Odor Syndrome","Halitosis","Isolated Congenital Asplenia","Lambert Eaton (LEMS)","STAG1 Gene Mutation","Coffin Lowry Syndrome","Borjeson-Forssman-Lehman Syndrome","Blau Syndrome","Arginase 1 Deficiency","HSPB8 Myopathy","Beta-Mannosidosis","TBX4 Syndrome","DHDDS Gene Mutations","MAND-MBD5-Associated Neurodevelopmental Disorder","Constitutional Mismatch Repair Deficiency (CMMRD)","SPATA5 Disorder","SPATA5L1 Related Disorder","Acrodysostosis","Multi-systematic Smooth Muscle Dysfunction Syndrome","CRELD1 (Cysteine Rich With EGF Like Domains 1)","GNB1 Syndrome","Pyruvate Dehydrogenase Complex Deficiency Disease","Beta Mannosidosis","Kbg Syndrome","Labrune Syndrome","Metachromatic Leukodystrophy (MLD)","Moyamoya Disease","OPHN1 Syndrome","Oculopharyngeal Muscular Dystrophy (OPMD)","TUBB3 Mutation","WOREE (WWOX-related Epileptic Encephalopathy","SCAR12","Skraban-Deardorff Syndrome","Hereditary Myopathy With Early Respiratory Failure",[698,699,700,701,702,409,703,704,416,705,375,706,707,708,579,588,709,710,366,711,712,374,713,376,714,365,715,716,591,717,718,594,595,719,597,598,720,721,601,722,723,724,651,725,726,727,728,729,730,731,656,732,733,734,661,662,735,736,737,738,739],"Rare Diseases","Neglected Diseases","Orphan Diseases","Rare Disease Research","Registries","Ataxia","Cornelia de Lange Syndrome","Ataxia Telangiectasia","Batten Disease","Mucolipidosis IV","Klippel-Feil Syndrome","Undiagnosed","Uncommon Disease","Hypersomnia","Hyperacusis","Marinesco-Sjogren Syndrome","4p-\u002FWolf-Hirschhorn Syndrome","Narcolepsy","Wiedermann-Steiner Syndrome","Autoimmune\u002Finflammatory Syndrome Induced by Adjuvants (ASIA)","Hemophagocytic Lymphohistiocytosis (HLH)","Inclusion body myopathy with early-onset Paget disease and frontotemporal dementia (IBMPFD)","1p36 deletion syndrome","Jansen metaphyseal chondrodysplasia","Chronic recurrent multifocal osteomyelitis (CRMO)","Malan syndrome","Hereditary Sensory and Autonomic Neuropathy","Juvenile nephropathic cystinosis","Nephropathic infantile cystinosis","Ocular cystinosis","Kennedy disease","Spinal Bulbar Muscular Atrophy (SBMA)","SMC1A Truncated Mutations (causing loss of gene function)","Leigh syndrome","Mucolipidosis","Mitochondrial aminoacyl-tRNA synthetases (Mt-aaRS Disorders)","Shine Syndrome","Intestinal Bromhidrosis Syndrome","Fish odor syndrome","Autosomal recessive extra oral halitosis","CACNA1H mutation","Dimethylglycine dehydrogenase deficiency","2025-05-22",{"date":742,"type":41},"2025-05-29",{"date":744,"type":41},"2010-07",{"date":746,"type":21},"2100-12",{"name":748,"class":48},"Sanford Health",2,{"id":751,"slug":752,"hasResults":11,"nctId":753,"briefTitle":754,"officialTitle":754,"acronym":4,"eligibilityCriteria":755,"healthyVolunteers":16,"sex":17,"minAge":756,"maxAge":757,"enrollmentInfo":758,"targetDuration":4,"studyType":104,"phases":760,"briefSummary":761,"conditions":762,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":764,"lastUpdatePostDateStruct":765,"startDateStruct":767,"completionDateStruct":769,"leadSponsor":771,"locationsCount":49},"100570540","evaluation-of-the-use-of-granulocyte-colony-stimulating-factor-gcsf-in-post-kasai-type-3-biliary-atresia-100570540","NCT06708572","Evaluation of the Use of Granulocyte Colony Stimulating Factor (GCSF) in Post Kasai Type 3 Biliary Atresia","Inclusion Criteria:\n\n* Infants with initial diagnosis of biliary atresia with biliary atresia score \\> 23.927 will be allocated for Kasai porto-enterostomy with intra-operative cholangiogram reaching type 3 biliary atresia anatomy as a final diagnosis.\n\nExclusion Criteria:\n\n* Major cardiac, renal, pulmonary, neurological malformations or illnesses.\n* Hemoglobinopathies, such as sickle cell anemia\n* Active systemic infection.\n* White blood cell count \\> 20,000 cells\u002Fmm3.\n* Platelet count \\\u003C 40,000 cells\u002Fmm3 or ≥ 800,000 cells\u002Fmm3.\n* Purpura fulminans or unexplained vascular thrombotic conditions.","20 Days","1 Year",{"count":759,"type":21},40,[106],"The aim of the study is to evaluate the use of Granulocyte Colony Stimulating Factor (GCSF) on the clinical and biochemical outcome of type 3 biliary atresia post kasai.",[763,26],"Granulocyte Colony-stimulating Factor","2024-12-09",{"date":766,"type":41},"2024-12-11",{"date":768,"type":21},"2024-12",{"date":770,"type":21},"2026-10-30",{"name":772,"class":48},"National Liver Institute, Egypt",{"id":774,"slug":775,"hasResults":11,"nctId":776,"briefTitle":777,"officialTitle":778,"acronym":4,"eligibilityCriteria":779,"healthyVolunteers":11,"sex":17,"minAge":780,"maxAge":781,"enrollmentInfo":782,"targetDuration":4,"studyType":104,"phases":784,"briefSummary":785,"conditions":786,"keywords":789,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":792,"lastUpdatePostDateStruct":793,"startDateStruct":795,"completionDateStruct":797,"leadSponsor":799,"locationsCount":49},"100559485","stem-cell-applications-in-biliary-atresia-patients-100559485","NCT06564740","Stem Cell Applications in Biliary Atresia Patients","Umbilical Cord Derived Mesenchymal Stem Cell (UC-MSC) Transplantation in Infants with Biliary Atresia: a Prospective Randomized Controlled Trial","Inclusion Criteria:\n\n* Infants were diagnosed with liver cirrhosis due to biliary atresia following Kasai's operation.\n* The patients two months old or older and exhibited signs of cirrhosis after the procedure, including hepatomegaly, congestive splenomegaly, elevated liver enzymes, esophageal varices (confirmed by endoscopy), and cirrhosis (confirmed by liver biopsy).\n\nExclusion Criteria:\n\n* Epilepsy\n* Neurological disorders\n* Coagulation disorders\n* Diabetes\n* Syndromic type biliary atresia\n* Allergies to anesthetic agents\n* Severe health conditions such as cancer or failure of the heart, lungs, liver, or kidneys, active infections, and severe psychiatric disorders.","2 Months","18 Years",{"count":783,"type":21},64,[106],"Recently, mesenchymal stem cell (MSC) transplantation has emerged as a promising treatment for liver cirrhosis in adults. Additionally, bone marrow-derived stem cell transplantation has shown success in treating children with biliary atresia (BA). This study aims to evaluate the efficacy of Umbilical Cord-Derived Mesenchymal Stem Cell (UC-MSC) therapy in BA through a multicentric randomized controlled trial.",[26,787,788],"Fibrosis, Liver","Stem Cell",[237,790,791],"liver fibrosis","stem cell","2024-09-03",{"date":794,"type":41},"2024-09-19",{"date":796,"type":41},"2024-06-01",{"date":798,"type":21},"2026-06-01",{"name":800,"class":48},"Necmi Kadıoğlu Hospital",{"id":802,"slug":803,"hasResults":11,"nctId":804,"briefTitle":805,"officialTitle":806,"acronym":807,"eligibilityCriteria":808,"healthyVolunteers":16,"sex":17,"minAge":809,"maxAge":781,"enrollmentInfo":810,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":811,"conditions":812,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":814,"lastUpdatePostDateStruct":815,"startDateStruct":817,"completionDateStruct":819,"leadSponsor":821,"locationsCount":49},"100469970","bilaco-trial-biliary-atresia---a-severe-complex-congenital-liver-disease-100469970","NCT05399745","BILACO Trial: Biliary Atresia - a Severe Complex Congenital Liver Disease","BILACO Trial: Biliary Atresia - a Severe Complex Congenital Liver Disease With High Mortality, Compromised Neurological Development, Severe Malnutrition and Unknown Etiology","BILACO","Inclusion Criteria:\n\n* Biliary atresia\n* Tetralogy of Fallot\n* Healthy controls\n\nExclusion Criteria:\n\n\\- Not able to participate in exams","0 Years",{"count":303,"type":21},"Biliary atresia is the most severe form of cholestatic liver disease. The children have high morbidity and mortality and get devastating pruritus and fatigue, failure to thrive, progressive hepatic failure and impaired neurodevelopment. The etiology is mostly unknown. More than half need a new liver from a living or deceased donor during childhood. However, correct timing of the transplantation is extremely difficult because of lack of consensus based on clinical assessment tools. All though the incidence is low, the cost of this disease is tremendous from both a clinical and human perspective. So far, protocolized neurodevelopment tests, genetic profiling, precise malnutrition evaluation based on clinical appearance, biochemical markers and brain MRI-scans, body composition, immunological function, level of physical activity and optimal time of transplantation in cholestatic children are unknown.\n\nThe aim is to determine risk factors for neurocognitive impairment in children suffering from severe cholestasis in order to determine optimal time for liver transplantation from a brain perspective.\n\nIn a prospective study, the investigators will investigate risk factors related to brain-, heart-, gut- and immunological function in the Danish cohort. This cohort consists of 75 children aged 0-18 years. In addition, 30 aged and gender matched healthy and 20 tetra fallot children will serve as control groups. The children will undergo extensive and advanced liver function evaluation, genetic profiling, nutrition and immunological status, neuro-imaging and neurocognitive evaluation at time of diagnose, 2 years of age, pre-school, pre-teenage, and teenage. In case of a liver transplantation, additional neuro-cognitive tests will be performed",[26,813],"Cognitive Impairment","2022-05-30",{"date":816,"type":41},"2022-06-01",{"date":818,"type":41},"2020-03-01",{"date":820,"type":21},"2040-12-31",{"name":822,"class":48},"Rigshospitalet, Denmark",{"id":824,"slug":825,"hasResults":11,"nctId":826,"briefTitle":827,"officialTitle":827,"acronym":828,"eligibilityCriteria":829,"healthyVolunteers":16,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":830,"targetDuration":4,"studyType":104,"phases":831,"briefSummary":832,"conditions":833,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":834,"lastUpdatePostDateStruct":835,"startDateStruct":837,"completionDateStruct":839,"leadSponsor":841,"locationsCount":749},"100383427","molecular-characterization-for-understanding-biliary-atresia-100383427","NCT04272515","Molecular Characterization for Understanding Biliary Atresia","CAVB","Inclusion Criteria:\n\n* confirmed diagnosis of biliary atresia in patients\n* parents of BA patients\n\nExclusion Criteria:\n\n* no",{"count":303,"type":21},[106],"Although considered a rare disease, Biliary Atresia (BA) is the leading cause of neonatal cholestasis and liver transplantation in children. Little is known about the molecular mechanisms that drive BA. The purpose of this study is to collect the fluid samples, explanted liver tissue samples and dermal biopsy samples to enable investigators to perform the genetic and molecular analyses that might point to the gene(s) and cellular pathway involved in etiology of BA disease.",[26],"2021-06-03",{"date":836,"type":41},"2021-06-04",{"date":838,"type":41},"2021-02-07",{"date":840,"type":21},"2032-02-07",{"name":842,"class":843},"Institut National de la Santé Et de la Recherche Médicale, France","OTHER_GOV",{"id":845,"slug":846,"hasResults":11,"nctId":847,"briefTitle":848,"officialTitle":849,"acronym":4,"eligibilityCriteria":850,"healthyVolunteers":16,"sex":17,"minAge":226,"maxAge":851,"enrollmentInfo":852,"targetDuration":4,"studyType":104,"phases":853,"briefSummary":854,"conditions":855,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":857,"lastUpdatePostDateStruct":858,"startDateStruct":860,"completionDateStruct":861,"leadSponsor":863,"locationsCount":49},"100219718","phase-1-treating-primary-sclerosing-cholangitis-and-biliary-atresia-with-vancomycin-100219718","NCT02137668","Treating Primary Sclerosing Cholangitis and Biliary Atresia With Vancomycin","The Human Gastrointestinal Tract Microbiota in the Setting of Treating Primary Sclerosing Cholangitis and Biliary Atresia With Vancomycin","Inclusion Criteria:\n\n* Persons who have primary sclerosing cholangitis, biliary atresia, a chronic intestinal disorder or who are undergoing upper or lower endoscopy.\n* Persons who have primary sclerosing cholangitis or biliary atresia who are good candidates for vancomycin therapy.\n\nExclusion Criteria:\n\n* Patients that have taken antibiotics and\u002For immunomodulators within the last 3 months will be excluded as this will alter the original bacterial flora.\n* Females who are pregnant may not participate.","40 Years",{"count":175,"type":21},[131],"The purpose of this study is twofold. First, is to determine whether vancomycin is effective in the early treatment of Biliary Atresia (BA) and Primary Sclerosing Cholangitis (PSC), and if so, by what mechanism. Secondly, to characterize human intestinal microbial communities and their interactions with the host.",[856,26],"Primary Sclerosing Cholangitis","2016-11-10",{"date":859,"type":21},"2016-11-15",{"date":744,"type":4},{"date":862,"type":21},"2028-07",{"name":864,"class":48},"Sacramento Pediatric Gastroenterology"]