NAtural Course and Prognosis of PFIC and Effect of Biliary Diversion (NAPPED)
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|ClinicalTrials.gov Identifier: NCT03930810|
Recruitment Status : Enrolling by invitation
First Posted : April 29, 2019
Last Update Posted : January 10, 2023
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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:
- 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.
- 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.
- 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.
- 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||Intervention/treatment|
|Progressive Familial Intrahepatic Cholestasis||Procedure: Surgical biliary diversion|
|Study Type :||Observational|
|Estimated Enrollment :||582 participants|
|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 26, 2017|
|Estimated Primary Completion Date :||January 1, 2032|
|Estimated Study Completion Date :||January 1, 2032|
|FIC1-deficiency and Bsep-deficiency||
Procedure: Surgical biliary diversion
Surgical interruption of enterohepatic circulation
- Number of participants with liver transplantation [ Time Frame: at 18 years of age ]Underwent liver transplant
- Number of participants that succumbed [ Time Frame: at 18 years of age ]Succumbed
- Number of participant undergoing a surgical biliary diversion [ Time Frame: at 5, 10, 15 and 18 years of age ]Underwent surgical biliary diversion
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|Ages Eligible for Study:||0 Years to 65 Years (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
|Sampling Method:||Probability Sample|
- Clinical suspicion for Bsep- or FIC1-deficiency
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03930810
|University Medical Center Groningen|
|Responsible Party:||Prof. Dr. Henkjan J. Verkade, Principal Investigator, Professor of Pediatrics, University Medical Center Groningen|
|Other Study ID Numbers:||
|First Posted:||April 29, 2019 Key Record Dates|
|Last Update Posted:||January 10, 2023|
|Last Verified:||January 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||Undecided|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases