Prospective, Multicenter Cohort Study on Primary Biliary Cholangitis

• Register: ClinicalTrials.gov
• Last refreshed on: 20 June 2023
• Main ID: NCT04076527
• Date of registration: 29/08/2019
• Prospective Registration: Yes
• Primary sponsor: University of Leipzig
• Public title: Prospective, Multicenter Cohort Study on Primary Biliary Cholangitis PBC-Cohort
• Scientific title: Prospective, Multicenter Cohort Study on Primary Biliary Cholangitis
• Date of first enrolment: September 19, 2019
• Target sample size: 1200
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT04076527
• Study type: Observational

Countries of recruitment

Germany

Contacts

• Name: Thomas Berg, Prof.Dr.

• Name: Johannes Wiegand, Prof.Dr.
• Affiliation: University of Leipzig

• Name: Christian Trautwein, Prof.Dr.
• Affiliation: RWTH Aachen University

• Name: Thomas Berg, Prof.Dr.
• Affiliation: University of Leipzig

Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Primary Biliary Cholangitis

PBC

Intervention(s)

Drug: UDCA

Drug: Ocaliva

Primary Outcome(s)

Systematic registry [Time Frame: from baseline to 36 months after baseline (observational period)]

Secondary Outcome(s)

Concomitant Non-Autoimmune Diseases [Time Frame: from baseline to 36 months after baseline]

Characterization of PBC therapies [Time Frame: from baseline to 36 months after baseline]

Concomitant Autoimmune Diseases [Time Frame: from baseline to 36 months after baseline]

Application and analyses of existing prognostic PBC scores to provide information on patients' prognosis. [Time Frame: from baseline to 36 months after baseline]

Comprehensive clinical characterization of German PBC patients [Time Frame: from baseline to 36 months after baseline]

Concomitant Medications [Time Frame: from baseline to 36 months after baseline]

Treatment response to PBC therapies after 12 months and during longer courses of application [Time Frame: from baseline to 12 months after baseline and to 36 months after baseline]

Secondary ID(s)

2.0

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

RWTH Aachen University

Leberhilfe Projekt gUG, Cologne

Intercept Pharma Europe Limited (IPEL)

Hannover Medical School

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

Medical care center for Gastroenterology, Berlin

University Hospital Erlangen

Institute for Interdisciplinary Medicine, Hamburg

Zentrum für Klinische Studien Leipzig