Primary Sclerosing CholangitisNCT04181138
Primary Sclerosing Cholangitis in Children
An observational study of Primary Sclerosing Cholangitis and Liver Diseases, run by Arbor Research Collaborative for Health at 12 sites in 2 countries.
Trial summary
Primary sclerosing cholangitis (PSC) is a rare liver disease that damages the liver's bile ducts. Bile ducts are tiny tubes that carry bile from the liver to the small intestine. Bile is a liquid produced by the liver that helps us absorb and use the nutrients in the food we eat. Read more about this study
- Who is it for?
- Ages 2 to 25 years
- Conditions the study is for
- Primary Sclerosing Cholangitis, Liver Diseases, Cholangitis, Sclerosing
- How many people
- 1,000 participants
- Where
- 12 sitesin 2 countries
- Study dates
- 30 Dec 2021to 31 May 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Participants are eligible for enrollment into the ChiLDReN PSC Study if they meet the inclusion criteria and if none of the exclusion criteria apply. Every effort will be made to enroll as diverse a patient population (racial, ethnic, sex, age, etc) as possible.
Who may be able to join
- Patients with the clinical diagnosis of large or small duct PSC made at any time prior to enrollment are screened for eligibility to participate in this prospective cohort study. The site PI will determine eligibility following review of MRCP or ERCP images with the site radiologist to confirm presence of an abnormal cholangiogram at the time of diagnosis of large duct PSC. Liver histopathology obtained at the time of diagnosis of small duct PSC will be reviewed with the site pathologist prior to enrollment.
- Individuals must meet all of the Inclusion criteria in order to be eligible to participate in the study:
- Aged 2 through 25 years at time of screening.
- Diagnosis of large duct PSC based on review of cholangiogram by MRC, ERC, or intraoperative cholangiogram (IOC) by the site radiologist and interpreted to be consistent with PSC, based on one or more of the following:
- Focal structuring of the bile duct(s)
- Dominant stricture of the common bile duct
Show all 21Show fewer
- Saccular dilatation of bile duct(s)
- Beaded appearance of bile duct(s)
- Pruning appearance of the distal bile duct branches
- AND/OR
- Diagnosis of small duct PSC based on review of liver histopathology by the site pathologist and interpreted to be compatible with PSC:
- Probable small duct PSC: biopsy with ≥3 of 5 criteria: periductal edema, concentric inflammation, bile duct injury, ductular reaction, and neutrophils in bile ducts (cholangitis) OR...
- Definitive small duct PSC: Periductal fibrosis/ "onion skinning" around interlobular bile ducts or smaller profiles
- Stated willingness to comply with all study procedures and availability for the duration of the study.
- Able to provide informed consent/assent
- Participants for the imaging study are eligible if they are:
- Aged 8 through 25 years at the time of screening
- No absolute contraindication to MRI
- No skin condition that could be aggravated by MREL
- Meet all other eligibility criteria of the PSC Observational Study
- For whom none of the exclusion criteria apply
Who cannot join
- An individual who meets any of the following criteria at baseline will be excluded from participation in this study.
- History of liver transplantation
- History bone marrow transplantation
- History of primary or acquired immunodeficiency predisposing to secondary sclerosing cholangitis, for instance: hyper-IgM syndrome, severe combined immunodeficiency (SCID) syndrome, common variable immunodeficiency (CVID) syndrome, cartilage hair hypoplasia syndrome, or HIV/AIDS
- History of histiocytosis, including Langerhans cell histiocytosis (LCH), or hemophagocytic lymphohistiocytosis (HLH)
- History of ischemic cholangitis
Show all 19Show fewer
- History of portal vein thrombosis with biliopathy, veno-occlusive disease, or abdominal radiation vasculopathy
- History of recurrent pyogenic cholangitis
- History of biliary tract surgery for cholecystolithiasis prior to cholangiogram/liver biopsy evaluated to determine enrollment
- History of biliary tract surgery for choledochal cyst
- History of hepatocellular carcinoma, or hepatoblastoma
- History of surgical biliary trauma
- History of congenital cytomegalovirus (CMV) hepatitis
- History of Sickle Cell Disease
- History of cystic fibrosis, biliary atresia, Caroli disease/congenital hepatic fibrosis, or progressive familial intrahepatic cholestasis type 3/MDR3 disease
- History of cardiac hepatopathy.
- History of metabolic disorders, including Wilson's disease, glycogen storage disorder, Alpha-1 Antitrypsin deficiency
- Diagnosis of systemic lupus erythematosus (SLE)
- Concurrent pregnancy at the time of enrollment -
Trial locations and contacts
12 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
12 sites in 2 countries
United States11 sites, 9 recruiting
- Recruiting
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Jordyn Turner, 404-785-3690, jordyn.turner@choa.org
Katelynn Harris, 4047850421, katelynn.harris@choa.org
- Recruiting
Children's Hospital Colorado
Aurora, Colorado, 80045
Cynthia Castanon, 720-777-0587, Cynthia.castanon@childrenscolorado.org
Michelle Landis, 720-777-8884, michelle.landis@childrenscolorado.org
- Recruiting
Ann & Robert H Lurie Children's Hospital
Chicago, Illinois, 60611
Angela Anthony, 312-227-4559, aanthony@luriechildrens.org
Jacqueline Alexander, 312-227-3523, jaalexander@luriechildrens.org
- Recruiting
Cincinnati Children's Hospital Medical
Cincinnati, Ohio, 45229
Erin Chapman, BAS, 513-803-7482, erin.chapman@cchmc.org
Jennifer Hawkins, MS, 513-636-7818, jennifer.hawkins@cchmc.org
- Recruiting
Texas Children's Hospital (Baylor College of Medicine)
Houston, Texas, 77030
Alyia Thomas, 832-824-6646, alyia.thomas@bcm.edu
Telma Gomez, 832-824-3244, telma.gomez@bcm.edu
- Completed
Riley Hospital for Children
Indianapolis, Indiana, 46202
- Recruiting
Children's Hospital of Los Angeles
Los Angeles, California, 90027
Sirikorn Boonsawat, 323-361-2181, sboonsawat@chla.usc.edu
Rohit Kohli, MD
- Recruiting
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Caitlin Griffiths, MS, 267-577-9888, griffithc2@chop.edu
Chigozie Amonu, 267-603-2396, amonuc@chop.edu
- Completed
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
- Recruiting
The University of Utah
Salt Lake City, Utah, 84113
Ann Rutherford, BS, CCRP, 801.585.9495, ann.rutherford@hsc.utah.edu
Natalie Fillerup, 801-587-5670, natalie.fillerup@hsc.utah.edu
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105
Melissa Young, 206.987.1037, melissa.young@seattlechildrens.org
Teresa Chen, 206-987-6649, teresa.chen@seattlechildrens.org
About this study
Official title: Prospective Observational Study of Primary Sclerosing Cholangitis (PSC) in Children
Primary sclerosing cholangitis (PSC) is a rare liver disease that damages the liver's bile ducts. Bile ducts are tiny tubes that carry bile from the liver to the small intestine. Bile is a liquid produced by the liver that helps us absorb and use the nutrients in the food we eat. In people with PSC, the bile backs up into the liver and will damage it, causing scarring of the liver.
The purposes of this study are to:
- Collect medical and other data to learn more about PSC, how it progresses, and identify factors that may cause the disease to progress more quickly.
- Ask questions about how PSC symptoms affect your child's life to learn more about its impact on your child's daily functioning
- Children with PSC who are seen at one of the participating clinical sites in the Childhood Liver Disease Research Network (ChiLDReN) will be asked to contribute information, DNA, and other specimens. The information and specimens will be available to investigators to carry out approved research aimed at learning more about the possible causes and long-term effects of PSC.
Read the detailed description
Pediatric primary sclerosing cholangitis (PSC) is a rare autoimmune biliary fibrosing disease that leads to significant morbidity, the need for liver transplantation in ~50% of patients, and an increased risk for biliary and colorectal cancers in adulthood. The progression of the biliary disease in children is variable and risk factors associated with a more rapid progression of disease have not been adequately studied. Importantly, pediatric hepatologists have never previously collaborated with inflammatory bowel disease (IBD) specialists to rigorously explore interactions between colonic inflammation and liver disease. New non-invasive imaging modalities to measure fibrosis have not been explored in pediatric PSC. Furthermore, the impact that PSC has on the global functioning of children is not well understood, and likely underappreciated.
The natural history of pediatric PSC is poorly understood. This study aims to determine risk factors, including activity of co-existent IBD, associated with more rapid progression of disease, characterize the impact of PSC on global functioning, define the spectrum and prognostic value of biliary tract disease and liver fibrosis based on novel imaging techniques, and establish a biobank of specimens for future mechanistic studies aimed at discovering biomarkers pertaining to etiology and severity of PSC and novel mechanisms of immunopathogenesis of disease. This comprehensive observational and longitudinal study will delineate unique aspects of the natural history and severity of pediatric PSC and of associated IBD and provide necessary data for future therapeutic trials. It aims to provide a platform to discover and validate circulating and imaging biomarkers, which may serve as surrogate endpoints in future interventional studies.
- Keywords
- Pediatric Liver Disease, Primary Sclerosing Cholangitis
- Medical subject headings
- Cholangitis, Sclerosing, Liver Diseases
What is being measured
The primary outcome is the main question the study is designed to answer.
To characterize the major phenotypes of PSC including patients with large duct or small duct disease, with and without Inflammatory Bowel Disease (IBD), and with and without Auto Immune Hepatitis (AIH)
Data will be collected on all phenotypes of PSC but attention is focused on how the intestinal inflammation and clinical activity of Inflammatory Bowel Disease (IBD) affect the progression of PSC, better classification of patients with features of Auto Immune Hepatitis (AIH), and the implications of bacterial cholangitis amongst all PSC phenotypes.
Collection of retrospective clinical and laboratory data from the time of diagnosis of PSC and annual timepoints thereafter. Information regarding clinically important timepoints, laboratory data and FibroScan Liver Stiffness Measurements (LSM) are collected prospectively.
Slides/images (if available) from each liver biopsy obtained at the time of diagnosis of PSC and thereafter and from the explanted liver recovered at the time of liver transplantation will undergo central review.
Cholangiography results (MRCP, ERCP) will also undergo central review.
Measured: up to 10 years
Secondary outcomes (3)
To identify the symptoms of PSC in children and the affects of those symptoms on the functional health of children.
Identify deficits in the functional health of children with PSC and explore the association of these functional parameters with biochemical markers of liver disease severity and IBD activity. Symptoms and the impact the symptoms on the functional health of children is measured through the use of "Patient Reported Outcomes" and performance-based metrics.
The burden of disease on the quality of life for children with PSC is measured utilizing the Peds-QL questionnaires (parent proxy and self-report). Child self-report: 8-12 years, and 13-18 years. Young adult self-report: 18-25 years. Parent proxy report: 2-4 years, 5-7 years, 8-12 years, and 13-17 years.
Itch and fatigue identified as predominant symptoms in PSC are measured through the administration (at every visit) of the 5-D ITCH scale and the PedsQL multidimensional fatigue and PROMIS Sleep Scales.
Frailty parameters in children with PSC are measured through the Fried frailty criteria at every visit.
Measured: up to 10 years
Utilizing imaging modalities measuring liver fibrosis, and large duct injury to correlate with other markers of fibrosis and biliary injury and predict progression of disease.
The imaging modalities are utilized to explore the two pathophysiological processes of PSC, liver fibrosis and bile duct damage. Quantitative MRI techniques may be more sensitive to disease progression than standard clinical and laboratory tests, as the liver and bile ducts are being explored directly.
All participants will undergo a liver stiffness measurement (LSM) at baseline and annually. In addition to the LSM, participants who are eligible (eligibility criteria are different than those for the overall study) will be asked to undergo a research MRI at the baseline, Year 1 and Year 5 visits.
Measured: up to 10 years
Development of a repository for formalin fixed paraffin embedded (FFPE) liver biopsy tissue, serum, plasma, peripheral blood mononuclear cells (PBMCs), DNA and stool.
Samples of peripheral blood mononuclear cells (PBMC), serum, plasma, DNA and stool are collected from participants at baseline and serum/plasma will be collected annually. In addition, any liver tissue previously collected or collected for clinical purposes in the future will be analyzed along with the PBMC, serum, plasma, DNA and stool within future mechanistic ancillary studies related to the diagnostic/prognostic biomarkers, and genetic, immune and microbial theories of pathogenesis.
Measured: up to 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Nov 2019
- Study start30 Dec 2021
- Primary completion31 May 2029 (estimated)
- Study completion31 May 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (14)Papers the sponsor has linked to this study.
- Other trials for Primary Sclerosing CholangitisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Arbor Research Collaborative for Health
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Cara Mack, MD, Study chair, Medical College of Wisconsin-MilwaukeeEd Doo, MD, Study director, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)Katrina Loh, MD, Study director, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)John Magee, MD, Principal investigator, University of MichiganLisa Henn, PhD, Principal investigator, Arbor Research Collaborative for Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04181138
- Sponsor's protocol ID
- PSC Study - ChiLDReN Network
- Other IDs
- 2U24DK062456
- Status last verified
- Jul 2026
- First submitted
- 6 Aug 2019
- Last updated
- 14 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Hirschfield GM, Karlsen TH, Lindor KD, Adams DH. Primary sclerosing cholangitis. Lancet. 2013 Nov 9;382(9904):1587-99. doi: 10.1016/S0140-6736(13)60096-3. Epub 2013 Jun 28.
PubMed 23810223Eaton JE, Talwalkar JA, Lazaridis KN, Gores GJ, Lindor KD. Pathogenesis of primary sclerosing cholangitis and advances in diagnosis and management. Gastroenterology. 2013 Sep;145(3):521-36. doi: 10.1053/j.gastro.2013.06.052. Epub 2013 Jul 1.
PubMed 23827861Bambha K, Kim WR, Talwalkar J, Torgerson H, Benson JT, Therneau TM, Loftus EV Jr, Yawn BP, Dickson ER, Melton LJ 3rd. Incidence, clinical spectrum, and outcomes of primary sclerosing cholangitis in a United States community. Gastroenterology. 2003 Nov;125(5):1364-9. doi: 10.1016/j.gastro.2003.07.011.
PubMed 14598252Deneau M, Jensen MK, Holmen J, Williams MS, Book LS, Guthery SL. Primary sclerosing cholangitis, autoimmune hepatitis, and overlap in Utah children: epidemiology and natural history. Hepatology. 2013 Oct;58(4):1392-400. doi: 10.1002/hep.26454. Epub 2013 Aug 13.
PubMed 23686586Toy E, Balasubramanian S, Selmi C, Li CS, Bowlus CL. The prevalence, incidence and natural history of primary sclerosing cholangitis in an ethnically diverse population. BMC Gastroenterol. 2011 Jul 18;11:83. doi: 10.1186/1471-230X-11-83.
PubMed 21767410Olsson R, Danielsson A, Jarnerot G, Lindstrom E, Loof L, Rolny P, Ryden BO, Tysk C, Wallerstedt S. Prevalence of primary sclerosing cholangitis in patients with ulcerative colitis. Gastroenterology. 1991 May;100(5 Pt 1):1319-23.
PubMed 2013375Ricciuto A, Kamath BM, Griffiths AM. The IBD and PSC Phenotypes of PSC-IBD. Curr Gastroenterol Rep. 2018 Mar 28;20(4):16. doi: 10.1007/s11894-018-0620-2.
PubMed 29594739Mieli-Vergani G, Vergani D, Baumann U, Czubkowski P, Debray D, Dezsofi A, Fischler B, Gupte G, Hierro L, Indolfi G, Jahnel J, Smets F, Verkade HJ, Hadzic N. Diagnosis and Management of Pediatric Autoimmune Liver Disease: ESPGHAN Hepatology Committee Position Statement. J Pediatr Gastroenterol Nutr. 2018 Feb;66(2):345-360. doi: 10.1097/MPG.0000000000001801.
PubMed 29356770Debray D, Pariente D, Urvoas E, Hadchouel M, Bernard O. Sclerosing cholangitis in children. J Pediatr. 1994 Jan;124(1):49-56. doi: 10.1016/s0022-3476(94)70253-5.
PubMed 8283375Smolka V, Karaskova E, Tkachyk O, Aiglova K, Ehrmann J, Michalkova K, Konecny M, Volejnikova J. Long-term follow-up of children and adolescents with primary sclerosing cholangitis and autoimmune sclerosing cholangitis. Hepatobiliary Pancreat Dis Int. 2016 Aug;15(4):412-8. doi: 10.1016/s1499-3872(16)60088-7.
PubMed 27498582Feldstein AE, Perrault J, El-Youssif M, Lindor KD, Freese DK, Angulo P. Primary sclerosing cholangitis in children: a long-term follow-up study. Hepatology. 2003 Jul;38(1):210-7. doi: 10.1053/jhep.2003.50289.
PubMed 12830004Miloh T, Arnon R, Shneider B, Suchy F, Kerkar N. A retrospective single-center review of primary sclerosing cholangitis in children. Clin Gastroenterol Hepatol. 2009 Feb;7(2):239-45. doi: 10.1016/j.cgh.2008.10.019. Epub 2008 Oct 30.
PubMed 19121649Valentino PL, Wiggins S, Harney S, Raza R, Lee CK, Jonas MM. The Natural History of Primary Sclerosing Cholangitis in Children: A Large Single-Center Longitudinal Cohort Study. J Pediatr Gastroenterol Nutr. 2016 Dec;63(6):603-609. doi: 10.1097/MPG.0000000000001368.
PubMed 27504812Wilschanski M, Chait P, Wade JA, Davis L, Corey M, St Louis P, Griffiths AM, Blendis LM, Moroz SP, Scully L, et al. Primary sclerosing cholangitis in 32 children: clinical, laboratory, and radiographic features, with survival analysis. Hepatology. 1995 Nov;22(5):1415-22.
PubMed 7590657
Study at a glance
From the registry record, last updated by the sponsor on 14 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Observational Study of Primary Sclerosing Cholangitis (PSC) in Children
- ClinicalTrials.gov ID
- NCT04181138
- Acronym / sponsor ID
- PSC Study - ChiLDReN Network
- Keywords
- Pediatric Liver Disease, Primary Sclerosing Cholangitis
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 1,000 participants (estimated)
- Lead sponsor
- Arbor Research Collaborative for HealthUniversity, hospital or other
- Collaborators
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Study start
- 30 Dec 2021 (actual)
- Primary completion
- 31 May 2029 (estimated)
- Study completion
- 31 May 2029 (estimated)
- First posted
- 29 Nov 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Primary sclerosing cholangitis (PSC) is a rare liver disease that damages the liver's bile ducts. Bile ducts are tiny tubes that carry bile from the liver to the small intestine. Bile is a liquid produced by the liver that helps us absorb and use the nutrients in the food we eat. In people with PSC, the bile backs up into the liver and will damage it, causing scarring of the liver.
The purposes of this study are to:
- Collect medical and other data to learn more about PSC, how it progresses, and identify factors that may cause the disease to progress more quickly.
- Ask questions about how PSC symptoms affect your child's life to learn more about its impact on your child's daily functioning
- Children with PSC who are seen at one of the participating clinical sites in the Childhood Liver Disease Research Network (ChiLDReN) will be asked to contribute information, DNA, and other specimens. The information and specimens will be available to investigators to carry out approved research aimed at learning more about the possible causes and long-term effects of PSC.
Detailed description
Pediatric primary sclerosing cholangitis (PSC) is a rare autoimmune biliary fibrosing disease that leads to significant morbidity, the need for liver transplantation in ~50% of patients, and an increased risk for biliary and colorectal cancers in adulthood. The progression of the biliary disease in children is variable and risk factors associated with a more rapid progression of disease have not been adequately studied. Importantly, pediatric hepatologists have never previously collaborated with inflammatory bowel disease (IBD) specialists to rigorously explore interactions between colonic inflammation and liver disease. New non-invasive imaging modalities to measure fibrosis have not been explored in pediatric PSC. Furthermore, the impact that PSC has on the global functioning of children is not well understood, and likely underappreciated.
The natural history of pediatric PSC is poorly understood. This study aims to determine risk factors, including activity of co-existent IBD, associated with more rapid progression of disease, characterize the impact of PSC on global functioning, define the spectrum and prognostic value of biliary tract disease and liver fibrosis based on novel imaging techniques, and establish a biobank of specimens for future mechanistic studies aimed at discovering biomarkers pertaining to etiology and severity of PSC and novel mechanisms of immunopathogenesis of disease. This comprehensive observational and longitudinal study will delineate unique aspects of the natural history and severity of pediatric PSC and of associated IBD and provide necessary data for future therapeutic trials. It aims to provide a platform to discover and validate circulating and imaging biomarkers, which may serve as surrogate endpoints in future interventional studies.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 2 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Participants are eligible for enrollment into the ChiLDReN PSC Study if they meet the inclusion criteria and if none of the exclusion criteria apply. Every effort will be made to enroll as diverse a patient population (racial, ethnic, sex, age, etc) as possible.
Key inclusion criteria (21 listed)
- Patients with the clinical diagnosis of large or small duct PSC made at any time prior to enrollment are screened for eligibility to participate in this prospective cohort study. The site PI will determine eligibility following review of MRCP or ERCP images with the site radiologist to confirm presence of an abnormal cholangiogram at the time of diagnosis of large duct PSC. Liver histopathology obtained at the time of diagnosis of small duct PSC will be reviewed with the site pathologist prior to enrollment.
- Individuals must meet all of the Inclusion criteria in order to be eligible to participate in the study:
- Aged 2 through 25 years at time of screening.
- Diagnosis of large duct PSC based on review of cholangiogram by MRC, ERC, or intraoperative cholangiogram (IOC) by the site radiologist and interpreted to be consistent with PSC, based on one or more of the following:
- AND/OR
Key exclusion criteria (19 listed)
- An individual who meets any of the following criteria at baseline will be excluded from participation in this study.
- History of liver transplantation
- History bone marrow transplantation
- History of primary or acquired immunodeficiency predisposing to secondary sclerosing cholangitis, for instance: hyper-IgM syndrome, severe combined immunodeficiency (SCID) syndrome, common variable immunodeficiency (CVID) syndrome, cartilage hair hypoplasia syndrome, or HIV/AIDS
- History of histiocytosis, including Langerhans cell histiocytosis (LCH), or hemophagocytic lymphohistiocytosis (HLH)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
To characterize the major phenotypes of PSC including patients with large duct or small duct disease, with and without Inflammatory Bowel Disease (IBD), and with and without Auto Immune Hepatitis (AIH)
Time frame: up to 10 years
Data will be collected on all phenotypes of PSC but attention is focused on how the intestinal inflammation and clinical activity of Inflammatory Bowel Disease (IBD) affect the progression of PSC, better classification of patients with features of Auto Immune Hepatitis (AIH), and the implications of bacterial cholangitis amongst all PSC phenotypes. Collection of retrospective clinical and laboratory data from the time of diagnosis of PSC and annual timepoints thereafter. Information regarding clinically important timepoints, laboratory data and FibroScan Liver Stiffness Measurements (LSM) are collected prospectively. Slides/images (if available) from each liver biopsy obtained at the time of diagnosis of PSC and thereafter and from the explanted liver recovered at the time of liver transplantation will undergo central review. Cholangiography results (MRCP, ERCP) will also undergo central review.
3 secondary outcomes
To identify the symptoms of PSC in children and the affects of those symptoms on the functional health of children.
Time frame: up to 10 years
Utilizing imaging modalities measuring liver fibrosis, and large duct injury to correlate with other markers of fibrosis and biliary injury and predict progression of disease.
Time frame: up to 10 years
Development of a repository for formalin fixed paraffin embedded (FFPE) liver biopsy tissue, serum, plasma, peripheral blood mononuclear cells (PBMCs), DNA and stool.
Time frame: up to 10 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jul 2026.
- Background: PubMed 23810223Hirschfield GM, Karlsen TH, Lindor KD, Adams DH. Primary sclerosing cholangitis. Lancet. 2013 Nov 9;382(9904):1587-99. doi: 10.1016/S0140-6736(13)60096-3. Epub 2013 Jun 28.
- Background: PubMed 23827861Eaton JE, Talwalkar JA, Lazaridis KN, Gores GJ, Lindor KD. Pathogenesis of primary sclerosing cholangitis and advances in diagnosis and management. Gastroenterology. 2013 Sep;145(3):521-36. doi: 10.1053/j.gastro.2013.06.052. Epub 2013 Jul 1.
- Background: PubMed 14598252Bambha K, Kim WR, Talwalkar J, Torgerson H, Benson JT, Therneau TM, Loftus EV Jr, Yawn BP, Dickson ER, Melton LJ 3rd. Incidence, clinical spectrum, and outcomes of primary sclerosing cholangitis in a United States community. Gastroenterology. 2003 Nov;125(5):1364-9. doi: 10.1016/j.gastro.2003.07.011.
- Background: PubMed 23686586Deneau M, Jensen MK, Holmen J, Williams MS, Book LS, Guthery SL. Primary sclerosing cholangitis, autoimmune hepatitis, and overlap in Utah children: epidemiology and natural history. Hepatology. 2013 Oct;58(4):1392-400. doi: 10.1002/hep.26454. Epub 2013 Aug 13.
- Background: PubMed 21767410Toy E, Balasubramanian S, Selmi C, Li CS, Bowlus CL. The prevalence, incidence and natural history of primary sclerosing cholangitis in an ethnically diverse population. BMC Gastroenterol. 2011 Jul 18;11:83. doi: 10.1186/1471-230X-11-83.
- Background: PubMed 2013375Olsson R, Danielsson A, Jarnerot G, Lindstrom E, Loof L, Rolny P, Ryden BO, Tysk C, Wallerstedt S. Prevalence of primary sclerosing cholangitis in patients with ulcerative colitis. Gastroenterology. 1991 May;100(5 Pt 1):1319-23.
- Background: PubMed 29594739Ricciuto A, Kamath BM, Griffiths AM. The IBD and PSC Phenotypes of PSC-IBD. Curr Gastroenterol Rep. 2018 Mar 28;20(4):16. doi: 10.1007/s11894-018-0620-2.
- Background: PubMed 29356770Mieli-Vergani G, Vergani D, Baumann U, Czubkowski P, Debray D, Dezsofi A, Fischler B, Gupte G, Hierro L, Indolfi G, Jahnel J, Smets F, Verkade HJ, Hadzic N. Diagnosis and Management of Pediatric Autoimmune Liver Disease: ESPGHAN Hepatology Committee Position Statement. J Pediatr Gastroenterol Nutr. 2018 Feb;66(2):345-360. doi: 10.1097/MPG.0000000000001801.
- Background: PubMed 8283375Debray D, Pariente D, Urvoas E, Hadchouel M, Bernard O. Sclerosing cholangitis in children. J Pediatr. 1994 Jan;124(1):49-56. doi: 10.1016/s0022-3476(94)70253-5.
- Background: PubMed 27498582Smolka V, Karaskova E, Tkachyk O, Aiglova K, Ehrmann J, Michalkova K, Konecny M, Volejnikova J. Long-term follow-up of children and adolescents with primary sclerosing cholangitis and autoimmune sclerosing cholangitis. Hepatobiliary Pancreat Dis Int. 2016 Aug;15(4):412-8. doi: 10.1016/s1499-3872(16)60088-7.
- Background: PubMed 12830004Feldstein AE, Perrault J, El-Youssif M, Lindor KD, Freese DK, Angulo P. Primary sclerosing cholangitis in children: a long-term follow-up study. Hepatology. 2003 Jul;38(1):210-7. doi: 10.1053/jhep.2003.50289.
- Background: PubMed 19121649Miloh T, Arnon R, Shneider B, Suchy F, Kerkar N. A retrospective single-center review of primary sclerosing cholangitis in children. Clin Gastroenterol Hepatol. 2009 Feb;7(2):239-45. doi: 10.1016/j.cgh.2008.10.019. Epub 2008 Oct 30.
- Childhood Liver Disease Research Network (ChiLDReN) websiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Cara Mack, MD study chair
Medical College of Wisconsin-Milwaukee
Ed Doo, MD study director
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Katrina Loh, MD study director
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
12 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Primary Sclerosing Cholangitis and Liver Diseases, run by Arbor Research Collaborative for Health at 12 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 21 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 14 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Melissa Sexton, BBA, CCRP (734-693--3811, melissa.sexton@arborresearch.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To characterize the major phenotypes of PSC including patients with large duct or small duct disease, with and without Inflammatory Bowel Disease (IBD), and with and without Auto Immune Hepatitis (AIH) (time frame: up to 10 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04181138, which also lists 14 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jul 2026.