Unraveling the Mechanisms Underlying Primary Sclerosing Cholangitis Through a Multidisciplinary, Integrative Research Approach
2 other identifiers
observational
143
1 country
1
Brief Summary
Background: Primary sclerosing cholangitis is a rare chronic liver disease. It affects the bile ducts of the liver. It can result in bile duct infections, cirrhosis, cancer, and end stage liver disease. Researchers want to learn more about this disease. Objective: To understand the biological causes of primary sclerosing cholangitis. Eligibility: Adults age 18 and older who have primary sclerosing cholangitis. Design: Participants will be screened with a medical history, physical exam, and blood tests. Participants will give blood, saliva, urine, and stool samples. They will have nasal swabs. They will complete surveys. Participants will get an intravenous (IV) catheter. A plastic tube is inserted into an arm vein. Participants will have a colonoscopy. A tube with a video camera at the end is inserted into the rectum. Participants will have an upper endoscopy. A scope with a light and camera at its tip is used to look inside the upper digestive tract. Participants will have a liver biopsy, entering through the chest wall or a neck vein. Blood is drawn from a blood vessel that carries blood to the liver. A liver tissue sample is taken. Participants will have magnetic resonance imaging or spectroscopy. They will get a contrast agent through an IV. Participants may have an optional bone marrow aspiration. A large needle is inserted into the hip to withdraw marrow. Participants will have a liver ultrasound. Participants will complete a 3-day food diary. They will have a nutrition assessment. Participants may give contact details for people who live with them, to also take part in this study. Participation will last for 12 months.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for all trials
Started Mar 2023
Typical duration for all trials
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 24, 2020
CompletedFirst Posted
Study publicly available on registry
December 28, 2020
CompletedStudy Start
First participant enrolled
March 31, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 31, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
October 31, 2026
April 24, 2026
April 1, 2026
3.6 years
December 24, 2020
April 23, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
The ultimate goal of this study is to generate understanding of how factors driving pathogenesis in PSC interact by capturing and integrating collated datasets from across relevant biologic systems and interpreting those in the context of phenot...
1\. Identification of immune signatures at multiple levels and from different anatomical sites and tissues 2. Characterization of microbiome signatures (taxonomic and functional), as well as identification of specific species. 3. Identification of metabolomic signatures from different anatomical sites and tissues as well as identification of different biomarkers correlating with disease progression. 4. MicroRNA profiling of portal and systemic blood.
End of Study
Secondary Outcomes (4)
To collect comprehensive data on distinct patterns of disease expression, through single cell sequencing and microRNA and transcriptome profiling.
End of Study
To conduct extensive phenotypic characterization of cellular and humoral immune responses as well as microbiome signatures at multiple anatomic sites
End of Study
To evaluate metabolic signatures or biomarkers for PSC diagnosis and prognostication
End of Study
To generate a humanized mouse model of each subject s disease by obtaining bone marrow aspirate
End of Study
Study Arms (2)
Controls
90 controls to collect epidemiological data as well as blood samples, nasal swabs, salivary samples and stool samples from family members and cohabitants of subjects affected with PSC.
PSC Pariticipants
40 patients with PSC diagnosed by standard clinical, biochemical, or imaging features (including up to 30 with a known diagnosis of IBD)
Eligibility Criteria
40 male and female adult patients 18 years of age or above with known PSC recruited from hospitals in the continental US. 90 healthy volunteers, male and female 18 years of age or above, who are either family members of participating patients with PSC or have been cohabitating with participating patients with PSC.
You may qualify if:
- In order to be eligible to participate in this study, an individual must meet all of the following criteria:
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Male or nonpregnant female, greater than or equal to 18 years of age
- Evidence of PSC established by biochemical testing and either MRCP or ERCP. Participant must have evidence of large duct disease on imaging.
- Agreement to adhere to Lifestyle Considerations throughout study duration.
You may not qualify if:
- An individual who meets any of the following criteria will be excluded from participation in this study:
- Pregnant or lactating women or females of child-bearing age not taking measures to prevent pregnancy during the period of study.
- History of clinical, serologic, or histopathologic evidence supporting etiologies of chronic liver disease other than PSC
- History of liver transplantation
- Diagnosis consistent with secondary sclerosing cholangitis (cholelithiasis, bile duct strictures secondary to ischemia, HIV cholangiopathy, etc.).
- Current or past clinical evidence of decompensated liver disease (e.g. ascites, bleeding esophageal varices, spontaneous bacterial peritonitis, encephalopathy, etc.).
- History of liver or bile duct lesions concerning for malignancy.
- Ca-19-9 \>130 U/microL
- Alpha-fetoprotein level greater than 200 ng/microL.
- Patients with active bacterial, viral, or fungal, systemic or localized infection.
- Unwillingness to refrain from ingesting probiotics during study.
- History of systemic disease not related to PSC that is poorly controlled or associated with declining functional status. Examples include but are not limited to: poorly controlled diabetes mellitus, chronic renal failure with eGFR is \<60 microl/min/1.73m\^2, chronic
- symptomatic heart failure or severe COPD.
- Patients with history of any gastrointestinal malignancy in the last 3 years prior to enrollment will be excluded. Patients with history of any malignancy in the last 3 years prior to enrollment other than those individuals who had undergone curative surgical therapy and deemed as low risk for recurrence by her/his treating physician would be excluded.
- History of portal vein thrombosis
- +23 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Theo Heller, M.D.
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- NIH
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 24, 2020
First Posted
December 28, 2020
Study Start
March 31, 2023
Primary Completion (Estimated)
October 31, 2026
Study Completion (Estimated)
October 31, 2026
Last Updated
April 24, 2026
Record last verified: 2026-04-01
Data Sharing
- IPD Sharing
- Will not share