Observational study on Primary Biliary Cholangitis in Germany

Prospective, Multicenter Cohort Study on Primary Biliary Cholangitis

Observational University of Leipzig · NCT04076527

This study is collecting information from patients with Primary Biliary Cholangitis in Germany to see how well different approved treatments work in real life.

Quick facts

Study typeObservational
Enrollment1200 (estimated)
Ages18 Years and up
SexAll
SponsorUniversity of Leipzig Academic / other
Locations50 sites (Aachen and 49 other locations)
Trial IDNCT04076527 on ClinicalTrials.gov

What this trial studies

This observational study aims to collect data on the progression and treatment outcomes of patients with Primary Biliary Cholangitis (PBC) in Germany. It involves three parallel groups of patients receiving routine treatment with approved medications, including Ursodeoxycholic acid (UDCA) and Obeticholic acid (Ocaliva). The study will evaluate the effectiveness, safety, and tolerability of these treatments in a real-world clinical setting. By establishing a national registry, the study seeks to better characterize the clinical course of PBC and improve understanding of treatment responses.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 and older diagnosed with Primary Biliary Cholangitis who are receiving approved medications for their condition.

Not a fit: Patients without a confirmed diagnosis of PBC or those not receiving approved treatments for the condition may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could enhance treatment strategies for PBC, leading to improved patient outcomes and management of the disease.

How similar studies have performed: Other national and international registries have shown success in characterizing PBC, suggesting that this approach could yield valuable insights as well.

Eligibility criteria

Show full 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.

Where this trial is running

Aachen and 49 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions PBCPrimary Biliary Cholangitisursodeoxycholic acidUDCAobeticholic acidOCALIVACohortobservational study
Last reviewed 2026-06-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.