Status:

RECRUITING

Unraveling the Mechanisms Underlying Primary Sclerosing Cholangitis Through a Multidisciplinary, Integrative Research Approach

Lead Sponsor:

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Conditions:

Primary Sclerosing Cholangitis

Eligibility:

All Genders

18-90 years

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 dise...

Detailed Description

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 un...

Eligibility Criteria

Inclusion

  • PSC SUBJECTS:
  • INCLUSION CRITERIA:
  • 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.
  • EXCLUSION CRITERIA:
  • 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
  • Patients with severe allergic reactions to iodine or other contrast, which cannot be controlled by premedication with antihistamines or steroids.
  • History of gastric and/or proximal small bowel surgery including bariatric surgery such as Roux-en-Y gastric bypass
  • Contraindication to monitored anesthesia care and/or medications that are commonly used for conscious sedation during GI Endoscopy
  • Use of anti-coagulant and anti-platelet agents excluding aspirin and NSAIDs
  • Contraindications to completing MRCP or MRI
  • Absolute neutrophil count below 1000/mm\^3
  • Hemoglobin level below 10.0 g/dl
  • Platelet count lower than 50,000/mm\^3.
  • 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.
  • 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:
  • Male or female greater than or equal to 18 years of age
  • 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:
  • History suggestive of PSC
  • History of chronic liver disease (except for steatosis)
  • 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 Inflammatory Bowel Disease
  • Antibiotic use within the last 6 weeks
  • Pregnancy
  • Inability to provide informed consent

Exclusion

    Key Trial Info

    Start Date :

    March 31 2023

    Trial Type :

    OBSERVATIONAL

    Allocation :

    ESTIMATED

    End Date :

    October 31 2026

    Estimated Enrollment :

    143 Patients enrolled

    Trial Details

    Trial ID

    NCT04685200

    Start Date

    March 31 2023

    End Date

    October 31 2026

    Last Update

    December 31 2025

    Active Locations (1)

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    National Institutes of Health Clinical Center

    Bethesda, Maryland, United States, 20892