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Scientists launch deep dive into rare liver disease PSC

NCT ID NCT04685200

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 17, 2026 · Updated 17 times

Summary

This study aims to understand what causes primary sclerosing cholangitis (PSC), a rare chronic liver disease. About 143 adults with PSC will provide blood, saliva, urine, stool, and tissue samples, plus undergo imaging and procedures like colonoscopy and liver biopsy. No treatment is given; the goal is to gather data to uncover the disease's biological roots.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 143 people

The number the study aims to enrol. It can still change while the study runs.

Started

Mar 2023

Expected to finish

Oct 2026

An estimate. End dates often move.

Lead sponsor

A government research agency

The lead sponsor is the US National Institutes of Health.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

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.

Ages

18 to 90 years

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

* PSC SUBJECTS: INCLUSION CRITERIA: In order to be eligible to participate in this study, an individual must meet all of the following criteria: 1. Stated willingness to comply with all study procedures and availability for the duration of the study 2. Male or nonpregnant female, greater than or equal to 18 years of age 3. Evidence of PSC established by biochemical testing and either MRCP or ERCP. Participant must have evidence of large duct disease on imaging. 4. Agreement to adhere to Lifestyle Considerations throughout study duration. EXCLUSION CRITERIA: An individual who meets any of the following criteria will be excluded from participation in this study: 1. Pregnant or lactating women or females of child-bearing age not taking measures to prevent pregnancy during the period of study. 2. History of clinical, serologic, or histopathologic evidence supporting etiologies of chronic liver disease other than PSC 3. History of liver transplantation 4. Diagnosis consistent with secondary sclerosing cholangitis (cholelithiasis, bile duct strictures secondary to ischemia, HIV cholangiopathy, etc.). 5. Current or past clinical evidence of decompensated liver disease (e.g. ascites, bleeding esophageal varices, spontaneous bacterial peritonitis, encephalopathy, etc.). 6. History of liver or bile duct lesions concerning for malignancy. 7. Ca-19-9 \>130 U/microL 8. Alpha-fetoprotein level greater than 200 ng/microL. 9. Patients with active bacterial, viral, or fungal, systemic or localized infection. 10. Unwillingness to refrain from ingesting probiotics during study. 11. 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. 12. 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. 13. History of portal vein thrombosis 14. Patients with severe allergic reactions to iodine or other contrast, which cannot be controlled by premedication with antihistamines or steroids. 15. History of gastric and/or proximal small bowel surgery including bariatric surgery such as Roux-en-Y gastric bypass 16. Contraindication to monitored anesthesia care and/or medications that are commonly used for conscious sedation during GI Endoscopy 17. Use of anti-coagulant and anti-platelet agents excluding aspirin and NSAIDs 18. Contraindications to completing MRCP or MRI 19. Absolute neutrophil count below 1000/mm\^3 20. Hemoglobin level below 10.0 g/dl 21. Platelet count lower than 50,000/mm\^3. 22. INR 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. 23. Inability to provide informed consent CONTROLS: INCLUSION CRITERIA: In order to be eligible to participate in this study, an individual must meet all of the following criteria: 1. Male or female greater than or equal to 18 years of age 2. Any individual who is either a parent, sibling or child of a PSC patient enrolled to the study, or an unrelated person who has been living with the patient for at least 3 consecutive months before study enrollment EXCLUSION CRITERIA: An individual who meets any of the following criteria will be excluded from participation in this study: 1. History suggestive of PSC 2. History of chronic liver disease (except for steatosis) 3. Patients with history of any malignancy in the 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. 4. History of Inflammatory Bowel Disease 5. Antibiotic use within the last 6 weeks 6. Pregnancy 7. Inability to provide informed consent

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

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