An observational study in Primary Sclerosing Cholangitis, sponsored by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). Recruiting at 1 site in United States. Open to participants aged 18 Years to 90 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2026-10-29.
Sponsored by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) · Observational
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.
Study Description:
We hypothesize that primary sclerosing cholangitis (PSC) develops as a consequence of a genetically driven aberrant immune response to commensal or pathogenic bacteria, and that unique genetic-immunemicrobial associations may underlie development of distinct disease patterns. We intend to conduct a thorough radiologic, endoscopic, histologic and microbiological investigation of patients with PSC in order to determine potential associations.
Objectives:
Primary Objective:
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 phenotypic presentation in one exceptionally well-characterized set of patients.
Secondary Objectives:
Endpoints:
Primary Endpoint:
149 studies on the registry are indexed under Cholangitis, Sclerosing; 39 are open to participants now.
This study's planned enrollment of 143 is below the median of 200 across 46 observational studies indexed under Cholangitis, Sclerosing.
Browse Cholangitis, Sclerosing studies →National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) is the lead sponsor of 538 studies on the registry; 55 are open to participants now.
Of its 80 completed or terminated interventional studies of FDA-regulated products, 51 (64%) have results posted.
Counted across the registry records on this site, refreshed daily.
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.
INCLUSION CRITERIA:
In order to be eligible to participate in this study, an individual must meet all of the following criteria:
EXCLUSION CRITERIA:
An individual who meets any of the following criteria will be excluded from participation in this 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 is \<60 microl/min/1.73m\^2, chronic
symptomatic heart failure or severe .
greater than or equal to 1.5, PTT greater thna or equal to 1.3 times control and/or any known history of disease associated with
increased bleeding diathesis.
CONTROLS:
INCLUSION CRITERIA:
In order to be eligible to participate in this study, an individual must meet all of the following criteria:
EXCLUSION CRITERIA:
An individual who meets any of the following criteria will be excluded from participation in this study:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Chronic obstructive pulmonary disease: long-term lung disease that makes it hard to breathe out, including emphysema and chronic bronchitis.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
COPD
Chronic obstructive pulmonary disease: long-term lung disease that makes it hard to breathe out, including emphysema and chronic bronchitis.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
INR, PT and aPTT (clotting tests)
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
40 patients with PSC diagnosed by standard clinical, biochemical, or imaging features (including up to 30 with a known diagnosis of IBD)
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.
Time frame: End of Study
To collect comprehensive data on distinct patterns of disease expression, through single cell sequencing and microRNA and transcriptome profiling.
Single cell sequencing, microRNA and transcriptome profiling
Time frame: End of Study
To conduct extensive phenotypic characterization of cellular and humoral immune responses as well as microbiome signatures at multiple anatomic sites
Immune phenotyping, immune repertoire sequencing and cytokine profiling techniques, as well as next generation sequencing of microbiota in saliva, stool, blood, bile, small intestine, colon and liver tissue
Time frame: End of Study
To evaluate metabolic signatures or biomarkers for PSC diagnosis and prognostication
High throughput metabolomics screening of portal and systemic blood, liver tissue and bile in search of bile acids and other target metabolites
Time frame: End of Study
To generate a humanized mouse model of each subject s disease by obtaining bone marrow aspirate
The study of immune phenotype and function in a host (in this case the mouse) na(SqrRoot) ve to immunosuppressive therapy
Time frame: End of Study
Plan to share: No
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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