An observational study in PBC and Primary Biliary Cholangitis, sponsored by University of Leipzig. Recruiting at 50 sites in Germany. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-06-07.
Sponsored by University of Leipzig · Observational
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Among 32 studies in Liver Cirrhosis, Biliary with a site in Germany — 4 open to participants now, 9 under way, 4 of them phase 3. Liver Cirrhosis, Biliary · Germany.
Among the 32 studies in Liver Cirrhosis, Biliary with a site in Germany, this one is one of 2 that started in 2019, is one of 5 observational ones, and was the 17th registered.
214 studies on the registry are indexed under Liver Cirrhosis, Biliary; 48 are open to participants now.
Browse Liver Cirrhosis, Biliary studies →4 of the 32 studies in Liver Cirrhosis, Biliary with a site in Germany are open now. Recruiting first, the newest first:
Assistance Publique - Hôpitaux de Paris · Sites in Germany, Belgium, France and 2 more countries
Ages 18–70
Assistance Publique - Hôpitaux de Paris · Sites in Germany, Austria, Belgium and 4 more countries
Ages 18–70
Gilead Sciences · Sites in Germany, United States, Argentina and 26 more countries
Ages 18+
A study can be open while its sites in Germany have not opened yet or have stopped. Only those recruiting at a site in Germany →
Of the 34 Liver Cirrhosis, Biliary studies recruiting now, 16 (47%) set no upper age limit and 11 (32%) stop at 75 or younger; 3 take children under 18; 4 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
32 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 1 · Phase 2 13 · Phase 3 11 · Phase 4 3
First in 2007; this study’s year is marked. 2 started in 2019.
By drug class, each counting the studies that test any of its medicines:
This study's planned enrollment of 1,200 is above the median of 380 across 54 observational studies indexed under Liver Cirrhosis, Biliary.
Median enrollment 119 across the 27 interventional studies, 670 across the 5 observational. Of the 23 that completed or were terminated, 18 (78%) have results posted.
This tab as a report to print or save as PDF → With an account.
28 of 32 have an industry lead sponsor. The 10 most frequent:
University of Leipzig is the lead sponsor of 148 studies on the registry; 25 are open to participants now.
University of Leipzig has 1 study filed under Liver Cirrhosis, Biliary — fewer than ten, so there is no comparison to draw.
1 of the studies in Liver Cirrhosis, Biliary with a site in Germany that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05050136Mirum Pharmaceuticals, Inc.A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis | 2027-01 |
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Among the 32 studies in Liver Cirrhosis, Biliary with a site in Germany, from their registry updates:
This tab as a report to print or save as PDF → With an account.
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.
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.
The patients will be recruited in gastroenterology specialized practices and outpatient clinics which provide routine treatment for PBC patients.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
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:
Positive anti-mitochondrial antibody (AMA) titer or if AMA negative or in low titer (\<1:80) => PBC-specific antibodies:
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:
Exclusion Criteria:
Current participation in a phase I to IV interventional clinical trial for PBC or participation in another PBC registry.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Drug: UDCA · Drug: Ocaliva
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.
Drug: UDCA
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.
Drug: UDCA · Drug: Ocaliva
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.
Time frame: from baseline to 36 months after baseline (observational period)
Comprehensive clinical characterization of German PBC patients
i.e. demographics, biochemical markers, ultrasound, transient elastography, stage of the disease
Time frame: from baseline to 36 months after baseline
Characterization of PBC therapies
Characterization of UDCA as first line therapy and characterization of approved second-line treatment options such as OCALIVA. Furthermore, safety data on the PBC medications used will be systematically gathered and reported on high-level aggregation with regard to any causality with PBC treatment per cohort. The respective results will be reviewed against the known safety profiles.
Time frame: from baseline to 36 months after baseline
Treatment response to PBC therapies after 12 months and during longer courses of application
To evaluate the natural progression under PBC drug therapy with respect to response to treatment changes in laboratory results (as ratios of the upper/lower limits of normal or differences to BL values) and characteristics of liver function will be described, e.g.: 1. frequency of hepatic decompensation (occurrence of variceal hemorrhage, ascites, encephalopathy, and/or hepatocellular carcinoma), 2. frequency of liver transplants, 3. frequency of deaths in total and from a liver-related cause, and 4. details on further events of special interest, e.g. for the first time serum bilirubin \> ULN and/or alkaline phosphatase \> 1.5 ULN, or transient elastography \> 9.6 kPa.
Time frame: 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.
Details on typical therapeutic measures in treating PBC and patient compliance will be presented. This allows making a statement on quality of PBC treatment in Germany. Effectiveness will be assessed per cohort and compared between physicians' practices and hospital outpatient departments, aggregating data over all participating sites of the respective structure of health care. This refers to GLOBE score and/or Paris II criteria, frequency of hepatic decompensation or frequency of abnormal surrogate parameter (i.e. alkaline phosphatase, bilirubin, ALAT, ASAT or transient elastography). The respective results will be discussed against the results provided from clinical trials to verify everyday suitability of the different PBC therapies.
Time frame: from baseline to 36 months after baseline
Concomitant Medications
Assessment of selected concomitant medications (e.g. symptomatic treatment of pru-ritus)
Time frame: from baseline to 36 months after baseline
Concomitant Autoimmune Diseases
Assessment of selected concomitant autoimmune diseases (e.g. overlap syndrome with autoimmune hepatitis)
Time frame: from baseline to 36 months after baseline
Concomitant Non-Autoimmune Diseases
Assessment of selected concomitant non-autoimmune diseases (i.e. cardiovascular disease, non-alcoholic fatty liver disease, metabolic syndrome, chronic kidney diseases)
Time frame: from baseline to 36 months after baseline
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Plan to share: Undecided
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Started Sep 2019 (actual). Primary completion is estimated for Mar 2024, 54 months after the start.
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