Prospective, Multicenter Cohort Study on Primary Biliary Cholangitis
PBC-Cohort
1 other identifier
observational
1,200
1 country
50
Brief Summary
The German PBC Cohort is a multi-centric, observational (non-interventional) study with three parallel groups. The main objective of this observational study is to describe the course of Primary biliary cholangitis (PBC) in patients in Germany under routine treatment with approved drugs. Therefore, the effectiveness and safety/tolerability of PBC treatment options in a real-life setting will be evaluated.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Sep 2019
Longer than P75 for all trials
50 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 29, 2019
CompletedFirst Posted
Study publicly available on registry
September 3, 2019
CompletedStudy Start
First participant enrolled
September 19, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2024
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2024
CompletedJune 7, 2023
June 1, 2023
4.5 years
August 29, 2019
June 6, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Systematic registry
A primary outcome measure is not applicable as usual, since this data acquisition is performed to built a newly developed systematic registry which serves to describe - for the first time in Germany - the characteristics and the recent state of usual clinical care of the respective population. Within the 18 months of recruitment and 3 years of individual follow-up for every patient regular analyses will be performed and published, based on a statistical analysis plan which may be yearly updated on request to address the main questions of the responsible PBC consortium. After the end of data acquisition hepatologic scientists may apply with detailed proposals to further use available data. A scientific consortium will than decided on further analyses of data.
from baseline to 36 months after baseline (observational period)
Secondary Outcomes (7)
Comprehensive clinical characterization of German PBC patients
from baseline to 36 months after baseline
Characterization of PBC therapies
from baseline to 36 months after baseline
Treatment response to PBC therapies after 12 months and during longer courses of application
from baseline to 12 months after baseline and to 36 months after baseline
Application and analyses of existing prognostic PBC scores to provide information on patients' prognosis.
from baseline to 36 months after baseline
Concomitant Medications
from baseline to 36 months after baseline
- +2 more secondary outcomes
Study Arms (3)
Group 1 - Incomplete Responder
1. Primary Incomplete Responder: PBC patients demonstrating an insufficient response to the standard therapy with ursodeoxycholic acid (UDCA) after a minimum of 12 months of treatment (Paris II criteria). 2. Secondary Incomplete Responder: PBC patients demonstrating a satisfactory initial response to UDCA after a minimum of 12 months of treatment (Paris II criteria) followed by a re-increase of ALP ≥1.5 ULN, or AST ≥1.5 ULN, or bilirubin \>1 mg/dl at any later time point during continuous UDCA-treatment.
Group 2 - Responder
PBC patients demonstrating a satisfactory initial and contin-ued response to UDCA after a minimum of 12 months of treatment (Paris II criteria) without a re-increase of ALP ≥1.5 ULN, or AST ≥1.5 ULN, or bilirubin \>1 mg/dl at any later time point during continuous UDCA-treatment.
Group 3
Patients newly diagnosed for PBC receiving an approved PBC therapy for the first time. Patients are considered to be newly diagnosed if the initial diagnosis took place no later than six months prior to inclusion into the study.
Interventions
Eligibility Criteria
The patients will be recruited in gastroenterology specialized practices and outpatient clinics which provide routine treatment for PBC patients.
You may qualify if:
- Age ≥ 18 years
- Diagnosis of PBC PBC diagnosis (consistent with AASLD and EASL practice guidelines), as demonstrated by the presence of at least two of the following three diagnostic factors:
- History of elevated ALP levels for 6 months.
- Positive anti-mitochondrial antibody (AMA) titer or if AMA negative or in low titer (\<1:80) =\> PBC-specific antibodies:
- anti-GP210 and/or
- anti-SP100 and/or
- antibodies against the major M2 components \[PDC-E2, 2-oxo-glutaric acid dehydrogenase complex (OADC-E2), branched-chain-2-oxo-acid-dehydrogenase complex, (BCOADC-E2)\].
- Liver biopsy consistent with PBC.
- Medication-based treatment with at least one drug approved in Germany for the treatment of PBC
- Availability of all following essential parameters at the initial diagnosis of PBC prior to the initiation of treatment with UDCA, 12 months after initiation of UDCA and if applicable at time point of secondary incomplete response:
- Platelet count
- Alkaline Phosphatase (ALP)
- Total Bilirubin
- Aspartate aminotransferase (AST/GOT)
- Age at initial diagnosis of PBC
- +2 more criteria
You may not qualify if:
- Current participation in a phase I to IV interventional clinical trial for PBC or participation in another PBC registry.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- University of Leipziglead
- RWTH Aachen Universitycollaborator
- Zentrum für Klinische Studien Leipzigcollaborator
- Intercept Pharma Europe Limited (IPEL)collaborator
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)collaborator
- University Hospital Erlangencollaborator
- Medical care center for Gastroenterology, Berlincollaborator
- Institute for Interdisciplinary Medicine, Hamburgcollaborator
- Leberhilfe Projekt gUG, Colognecollaborator
- Hannover Medical Schoolcollaborator
Study Sites (50)
University Hospital Aachen
Aachen, Germany
Charité - Campus Benjamin Franklin
Berlin, Germany
Charité-Campus Virchow-Klinikum
Berlin, Germany
Internal Practice
Berlin, Germany
Liver Center Checkpoint
Berlin, Germany
MVZ Gastroenterology
Berlin, Germany
Hospital Chemnitz
Chemnitz, Germany
University Hospital Cologne
Cologne, Germany
Internal Practice
Dornstadt, Germany
MVZ Düsseldorf
Düsseldorf, Germany
University hospital Düsseldorf
Düsseldorf, Germany
University Hospital Erlangen
Erlangen, Germany
St. Josef- Hospital Kupferdreh
Essen, Germany
University Hospital Essen
Essen, Germany
Internal Practice
Frankfurt, Germany
University Hospital J.W. Goethe- Universität
Frankfurt am Main, Germany
University Hospital Freiburg
Freiburg im Breisgau, Germany
University Hospital Gießen
Giessen, Germany
Gastroenerological-Oncological Practice
Halle, Germany
University Hospital Halle
Halle, Germany
Liver Center Hamburg - Asklepios Clinic St. Georg
Hamburg, Germany
Internal Practice
Hamelin, Germany
MHH
Hanover, Germany
University Hospital Heidelberg
Heidelberg, Germany
Gastroenerological Practice
Herne, Germany
University hospital Saarland
Homburg, Germany
University Hospital Jena
Jena, Germany
Gastroenerological Practice
Kassel, Germany
Center for Gastroenterology and Hepatology Kiel
Kiel, Germany
University Hospital Schleswig-Holstein Campus Kiel
Kiel, Germany
Eugastro - Gastroenerological Practice
Leipzig, 04129, Germany
University Hospital Leipzig
Leipzig, Germany
MVZ Leverkusen
Leverkusen, Germany
University hospital Schleswig-Holstein
Lübeck, Germany
Internal Practice Hepatology
Magdeburg, Germany
University hospital Magdeburg
Magdeburg, Germany
University Hospital Mainz
Mainz, Germany
University Hospital Mannheim
Mannheim, Germany
Hospital LMU
Munich, Germany
Liver Center Munich
Munich, Germany
Technical University - Klinikum rechts der Isar
Munich, Germany
University Hospital Münster
Münster, Germany
Hospital Nuremberg
Nuremberg, Germany
Internal Practice
Potsdam, Germany
University Hospital Regensburg
Regensburg, Germany
Diakonie-Klinikum Schwäbisch Hall
Schwäbisch Hall, Germany
Internal Practice
Schwerin, Germany
University Hospital Tübingen
Tübingen, Germany
University Hospital Ulm
Ulm, Germany
St. Josefs-Hospital
Wiesbaden, Germany
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
Thomas Berg, Prof.Dr.
University of Leipzig
- PRINCIPAL INVESTIGATOR
Johannes Wiegand, Prof.Dr.
University of Leipzig
- PRINCIPAL INVESTIGATOR
Christian Trautwein, Prof.Dr.
RWTH Aachen University
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Prof. Dr. Thomas Berg
Study Record Dates
First Submitted
August 29, 2019
First Posted
September 3, 2019
Study Start
September 19, 2019
Primary Completion
March 1, 2024
Study Completion
March 1, 2024
Last Updated
June 7, 2023
Record last verified: 2023-06