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Clinical trial information and results are updated daily from ClinicalTrials.gov. The latest data update was conducted on 01/20/2021.

NAtural Course and Prognosis of PFIC and Effect of Biliary Diversion

Clinicaltrials.gov identifier NCT03930810

Recruitment Status Enrolling by invitation

First Posted April 29, 2019

Last update posted April 29, 2019

Study Description

Brief summary:

The natural course of PFIC syndromes and the effect of diversion techniques, have so far not been characterized in a rigorous manner within a larger population of patients. In fact, the clinical or biochemical parameters which most directly define and/or predict the success of reduced enterohepatic circulation (either by surgical diversion or medically) are still unclear. The present project aims to: 1. Define the natural course of disease in genetically defined PFIC1, and PFIC2 patients, with respect to relevant biochemical and clinical parameters (and if available, histological). Included will be patients homozygous for a known, disease-causing mutation, patients compound homozygous for two disease-causing mutations or heterozygous for one disease-causing mutation in combination with the clinical phenotype of Bsep-deficiency or FIC1-deficiency. 2. Define the change in the natural course of disease in response to biliary diversion surgery and or liver transplantation, based on short- and long(er)-term changes in biochemical (if available, histological) and clinical parameters, including outcome measures. Follow up after transplantation will be limited to max 3 months after transplant surgery, follow up after surgical biliary diversion will be as long as possible. 3. Assessment of biochemical variables as possible surrogate endpoints for clinical hard endpoints. If possible this allows for identification of low-risk to high-risk patients early during follow-up. 4. If patient numbers permit, to establish genotype-phenotype relationships for the most common genetic mutations causing Bsep-deficiency or FIC1-deficiency. Based on this project it is anticipated that the investigators are able: - to characterize the variation in natural course of disease (whether or not genotype dependent) to allow clinicians to rationally select a target population for assessing the effect of medical intervention, rather than surgical biliary diversion); - to identify and qualify one or more biomarkers that independently predict either improved or poor clinical outcomes of surgical biliary diversion; - to investigate if the identified biomarker(s) can be used as surrogate end point(s) for assessing and predicting outcomes with novel interventional strategies.

  • Condition or Disease:Progressive Familial Intrahepatic Cholestasis
  • Intervention/Treatment: Procedure: Surgical biliary diversion
  • Phase: N/A
Detailed Description


Study Design
  • Study Type: Observational
  • Estimated Enrollment: 582 participants
  • Observational Model: Cohort
  • Time Perspective: Retrospective
  • Official Title: NAtural Course and Prognosis of PFIC and Effect of Biliary Diversion (NAPPED Study), Meta-analysis of Individual Patient Data of PFIC Before and After Surgery (Bile Diversion or Liver Transplantation)
  • Actual Study Start Date: January 2017
  • Estimated Primary Completion Date: January 2027
  • Estimated Study Completion Date: January 2027
Groups and Cohorts
Groups/Cohorts Intervention/treatment
: FIC1-deficiency and Bsep-deficiency
Procedure: Surgical biliary diversion
Surgical interruption of enterohepatic circulation
Outcome Measures
  • Primary Outcome Measures: 1. Number of participants with liver transplantation [ Time Frame: at 18 years of age ]
    Underwent liver transplant
  • 2. Number of participants that succumbed [ Time Frame: at 18 years of age ]
  • Secondary Outcome Measures: 1. Number of participant undergoing a surgical biliary diversion [ Time Frame: at 5, 10, 15 and 18 years of age ]
    Underwent surgical biliary diversion
Eligibility Criteria
  • Ages Eligible for Study: up to 65 / (64 years+)
  • Sexes Eligible for Study: All
  • Accepts Healthy Volunteers: No
  • Sampling Method: Probability Sample
  • Study Population: The diagnosis of PFIC needs to be performed according to the international guidelines, based on (episodes with a) low gamma-GT cholestasis and identification of disease-causing mutations in PFIC-1 (ATP8B1) or PFIC 2 (ABCB11) genes.

Inclusion Criteria:

- Clinical suspicion for Bsep- or FIC1-deficiency

Contacts and Locations

University Medical Center Groningen

Sponsors and Collaborators

University Medical Center Groningen

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