Prospective, Multicenter Cohort Study on Primary Biliary Cholangitis

NCT ID: NCT04076527

Last Updated: 2023-06-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

1200 participants

Study Classification

OBSERVATIONAL

Study Start Date

2019-09-19

Study Completion Date

2024-03-31

Brief Summary

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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.

Detailed Description

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Primary biliary cholangitis (PBC) is a chronic autoimmune cholestatic liver disease. The course of the disease is characterized by a slow destruction of bile ducts, and progressive cholestasis. Prognosis depends on the development of cirrhosis and its complications. Ursodeoxycholic acid (UDCA) has been established as standard therapy for PBC and improves patients' long-term outcome. However, UDCA is not a uniformly effective drug, and the prognosis of PBC patients insufficiently responding to treatment is markedly worse. For patients with suboptimal treatment response to UDCA obeticholic acid (OCA) as newly approved medication (OCALIVA®) is available as second line treatment.

Due to the low prevalence and the slowly progressive course of the disease it is very difficult to investigate the prognosis of subgroups of PBC patients or to evaluate the effectivness of therapeutic interventions on clinical outcomes. Therefore, several national or international registries (UK-PBC Consortium or the Global PBC Study Group) were founded to better characterize the clinical course of PBC patients.

Since in Germany a registry for PBC does not exist, the German PBC Cohort is being implemented as observational study to collect data on treatment progress and success in clinical routine that reflects real world conditions in Germany as closely as possible. The effectiveness and safety/tolerability of PBC treatment options (UDCA as standard therapy and second-line treatment options like OCALIVA in case of inadequate UDCA treatment response) will be evaluated.

In approximatly 40 sites in Germany routine data is collected. There are no specifications for the diagnosis, therapy and monitoring of the PBC patients. The documentation of the routine data is carried out alongside with guideline recommended treatment intervals of the patients.

Furthermore, a critical criterion for the German PBC Cohort study is the involvement of a sufficient number of gastroenterology specialized practices and outpatient clinics that have consciously not been selected based on the strict specifications of a clinical trial and which provide routine treatment for PBC patients. In addition, patient access is designed to be open. Data will be collected on patient groups that represent a majority of the PBC patients in Germany, but who are not being investigated in clinical trials.

Conditions

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PBC Primary Biliary Cholangitis

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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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.

UDCA

Intervention Type DRUG

Routine data is collected for UDCA therapy.

Ocaliva

Intervention Type DRUG

Routine data is collected for OCA therapy.

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.

UDCA

Intervention Type DRUG

Routine data is collected for UDCA therapy.

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.

UDCA

Intervention Type DRUG

Routine data is collected for UDCA therapy.

Ocaliva

Intervention Type DRUG

Routine data is collected for OCA therapy.

Interventions

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UDCA

Routine data is collected for UDCA therapy.

Intervention Type DRUG

Ocaliva

Routine data is collected for OCA therapy.

Intervention Type DRUG

Other Intervention Names

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ursodeoxycholic acid obeticholic acid

Eligibility Criteria

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Inclusion Criteria

1. Age ≥ 18 years
2. 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.
3. Medication-based treatment with at least one drug approved in Germany for the treatment of PBC
4. 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
5. Patients must meet criteria of one of the cohorts (group 1/2/3) within this NIS according to design
6. written statement of informed consent

Exclusion Criteria

Current participation in a phase I to IV interventional clinical trial for PBC or participation in another PBC registry.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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RWTH Aachen University

OTHER

Sponsor Role collaborator

Zentrum für Klinische Studien Leipzig

OTHER

Sponsor Role collaborator

Intercept Pharma Europe Limited (IPEL)

UNKNOWN

Sponsor Role collaborator

Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

OTHER

Sponsor Role collaborator

University Hospital Erlangen

OTHER

Sponsor Role collaborator

Medical care center for Gastroenterology, Berlin

UNKNOWN

Sponsor Role collaborator

Institute for Interdisciplinary Medicine, Hamburg

UNKNOWN

Sponsor Role collaborator

Leberhilfe Projekt gUG, Cologne

UNKNOWN

Sponsor Role collaborator

Hannover Medical School

OTHER

Sponsor Role collaborator

University of Leipzig

OTHER

Sponsor Role lead

Responsible Party

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Thomas Berg

Prof. Dr. Thomas Berg

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Thomas Berg, Prof.Dr.

Role: STUDY_CHAIR

University of Leipzig

Johannes Wiegand, Prof.Dr.

Role: PRINCIPAL_INVESTIGATOR

University of Leipzig

Christian Trautwein, Prof.Dr.

Role: PRINCIPAL_INVESTIGATOR

RWTH Aachen University

Locations

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University Hospital Aachen

Aachen, , Germany

Site Status RECRUITING

Charité - Campus Benjamin Franklin

Berlin, , Germany

Site Status RECRUITING

Charité-Campus Virchow-Klinikum

Berlin, , Germany

Site Status RECRUITING

Internal Practice

Berlin, , Germany

Site Status RECRUITING

Liver Center Checkpoint

Berlin, , Germany

Site Status NOT_YET_RECRUITING

MVZ Gastroenterology

Berlin, , Germany

Site Status RECRUITING

Hospital Chemnitz

Chemnitz, , Germany

Site Status RECRUITING

University Hospital Cologne

Cologne, , Germany

Site Status WITHDRAWN

Internal Practice

Dornstadt, , Germany

Site Status RECRUITING

MVZ Düsseldorf

Düsseldorf, , Germany

Site Status WITHDRAWN

University hospital Düsseldorf

Düsseldorf, , Germany

Site Status NOT_YET_RECRUITING

University Hospital Erlangen

Erlangen, , Germany

Site Status RECRUITING

St. Josef- Hospital Kupferdreh

Essen, , Germany

Site Status RECRUITING

University Hospital Essen

Essen, , Germany

Site Status NOT_YET_RECRUITING

Internal Practice

Frankfurt, , Germany

Site Status WITHDRAWN

University Hospital J.W. Goethe- Universität

Frankfurt am Main, , Germany

Site Status RECRUITING

University Hospital Freiburg

Freiburg im Breisgau, , Germany

Site Status RECRUITING

University Hospital Gießen

Giessen, , Germany

Site Status RECRUITING

Gastroenerological-Oncological Practice

Halle, , Germany

Site Status RECRUITING

University Hospital Halle

Halle, , Germany

Site Status RECRUITING

Liver Center Hamburg - Asklepios Clinic St. Georg

Hamburg, , Germany

Site Status WITHDRAWN

Internal Practice

Hamelin, , Germany

Site Status WITHDRAWN

MHH

Hanover, , Germany

Site Status RECRUITING

University Hospital Heidelberg

Heidelberg, , Germany

Site Status RECRUITING

Gastroenerological Practice

Herne, , Germany

Site Status RECRUITING

University hospital Saarland

Homburg, , Germany

Site Status RECRUITING

University Hospital Jena

Jena, , Germany

Site Status RECRUITING

Gastroenerological Practice

Kassel, , Germany

Site Status WITHDRAWN

Center for Gastroenterology and Hepatology Kiel

Kiel, , Germany

Site Status RECRUITING

University Hospital Schleswig-Holstein Campus Kiel

Kiel, , Germany

Site Status RECRUITING

Eugastro - Gastroenerological Practice

Leipzig, , Germany

Site Status RECRUITING

University Hospital Leipzig

Leipzig, , Germany

Site Status RECRUITING

MVZ Leverkusen

Leverkusen, , Germany

Site Status RECRUITING

University hospital Schleswig-Holstein

Lübeck, , Germany

Site Status RECRUITING

Internal Practice Hepatology

Magdeburg, , Germany

Site Status RECRUITING

University hospital Magdeburg

Magdeburg, , Germany

Site Status RECRUITING

University Hospital Mainz

Mainz, , Germany

Site Status RECRUITING

University Hospital Mannheim

Mannheim, , Germany

Site Status RECRUITING

Hospital LMU

Munich, , Germany

Site Status RECRUITING

Liver Center Munich

Munich, , Germany

Site Status WITHDRAWN

Technical University - Klinikum rechts der Isar

Munich, , Germany

Site Status RECRUITING

University Hospital Münster

Münster, , Germany

Site Status RECRUITING

Hospital Nuremberg

Nuremberg, , Germany

Site Status RECRUITING

Internal Practice

Potsdam, , Germany

Site Status RECRUITING

University Hospital Regensburg

Regensburg, , Germany

Site Status WITHDRAWN

Diakonie-Klinikum Schwäbisch Hall

Schwäbisch Hall, , Germany

Site Status WITHDRAWN

Internal Practice

Schwerin, , Germany

Site Status WITHDRAWN

University Hospital Tübingen

Tübingen, , Germany

Site Status RECRUITING

University Hospital Ulm

Ulm, , Germany

Site Status NOT_YET_RECRUITING

St. Josefs-Hospital

Wiesbaden, , Germany

Site Status RECRUITING

Countries

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Germany

Central Contacts

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Thomas Berg, Prof.Dr.

Role: CONTACT

+49 341 97 12 330

Johannes Wiegand, Prof.Dr.

Role: CONTACT

+49 341 97 12 330

Facility Contacts

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Christian Trautwein, Prof.Dr.

Role: primary

Marion Muche, Dr.

Role: primary

Tobias Müller, Dr.

Role: primary

Uwe Naumann, Dr.

Role: primary

Renate Heyne, Dr.

Role: primary

Wolf Peter Hofmann, Prof. Dr.

Role: primary

Ulrich Stölzel, Prof. Dr.

Role: primary

Nektarios Dikopoulos, Prof. Dr.

Role: primary

Hans Bock, Prof.

Role: primary

Andreas Kremer, Dr. Dr.

Role: primary

Susanne Beckebaum, Prof. Dr.

Role: primary

Sandra Christoph, Dr

Role: primary

Stefan Zeuzem, Prof. Dr.

Role: primary

Christoph Neumann-Haefelin, Dr.

Role: primary

Elke Roeb, Prof. Dr.

Role: primary

Rüdiger Behrens, Dr

Role: primary

Greinert, Dr.

Role: primary

Heike Bantel, Prof. Dr.

Role: primary

Theresa Hippchen, Dr.

Role: primary

Matthias Hinz, Dr.

Role: primary

Marcin Krawczyk, Prof. Dr.

Role: primary

Philipp Reuken, Dr.

Role: primary

Holger Hinrichsen, MD

Role: primary

Rainer Günther, Dr.

Role: primary

Ingolf Schiefke, Prof.

Role: primary

Thomas Berg, Prof. Dr.

Role: primary

Johannes Wiegand, Prof.Dr.

Role: backup

Karl- Georg Simon, Dr.

Role: primary

Henrike Dobbermann, Dr.

Role: primary

Kerstin Stein, Dr.

Role: primary

Verena Keitel-Anselmino, Prof. Dr.

Role: primary

Jörn Schattenberg, PD Dr

Role: primary

Andreas Teufel, Prof.Dr.Dr.

Role: primary

Gerald Denk, PD Dr

Role: primary

Ursula Tanase, Dr.

Role: primary

Hauke Heinzow, Prof. Dr.

Role: primary

Andreas Weber, Dr.

Role: primary

Harald Grümmer, Dr.

Role: primary

Christoph Berg, Prof. Dr.

Role: primary

Eugen Zizer, Dr.

Role: primary

Christoph Sarrazin, Prof. Dr.

Role: primary

Other Identifiers

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2.0

Identifier Type: -

Identifier Source: org_study_id

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