Brief description of trial (Data source: BASEC)
In dieser Studie wird die primär biliäre Cholangitis (auch bekannt als primär biliäre Zirrhose oder PBC) untersucht. Wir führen diese Studie durch, um zusätzliche Informationen über Obeticholsäure und ihre Wirkung auf Ihre PBC-Erkrankung zu gewinnen, wenn diese gemeinsam mit Ursodeoxycholsäure verabreicht wird und der Sponsor möchte den klinischen Nutzen für Patienten bestätigen. Darüber hinaus bewertet der Sponsor die Sicherheit des Arzneimittels sowie die Verteilung und den Abbau des Studienmedikaments. Dies wird Pharmakokinetik genannt.
Health conditions investigated(Data source: BASEC)
primär biliäre Zirrhose und mäßige bis schwerer Leberfunktionsstörung
Health conditions
(Data source: WHO)
Liver Cirrhosis, Biliary
Rare disease
(Data source: BASEC)
No
Intervention investigated (e.g. drug, therapy or campaign)
(Data source: BASEC)
Der Zweck dieser Studie besteht darin, herauszufinden, wie das Prüfpräparat, im Körper absorbiert und verteilt wird und wie das Prüfpräparat aus dem Körper ausgeschieden wird. Dies wird als Pharmakokinetik bezeichnet. Zudem wird auch die Sicherheit des Prüfpräparats untersuchen.
Interventions
(Data source: WHO)
Drug: Obeticholic Acid (OCA);Drug: Placebo
Criteria for participation in trial
(Data source: BASEC)
1. Eine definitive oder wahrscheinliche Diagnose von primärer biliäre Cholangitis
2. Hinweise auf eine Zirrhose
3. Leberfunktionsstörungen während der Voruntersuchung
Exclusion criteria
(Data source: BASEC)
1. Nicht-zirrhotische oder zirrhotische biliäre Cholangitis
2. Lebertransplantation oder Organtransplantation in der Krankengeschichte
3. Alkohol- oder Drogenmissbrauch innerhalb von 12 Monaten vor der Voruntersuchung
Inclusion/Exclusion Criteria
(Data source: WHO)
Inclusion Criteria:
1. A definite or probable diagnosis of PBC (consistent with American Association for the
Study of Liver Diseases [AASLD] and European Association for the Study of the Liver
[EASL] Practice Guidelines [Lindor 2009, EASL 2009]), defined as having =2 of the
following 3 diagnostic factors:
- History of elevated ALP levels for at least 6 months
- Positive antimitochondrial antibody (AMA) titer or if AMA negative or 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)
- Liver biopsy consistent with PBC (collected at any time prior to Screening)
2. Evidence of cirrhosis including at least one of the following:
- Biopsy results consistent with PBC Stage 4
- Liver stiffness as assessed by Transient Elastography (TE) Median Value =16.9kPa
- Clinical evidence in the absence of acute liver failure consistent with cirrhosis
including: gastroesophageal varices, ascites, radiological evidence of cirrhosis
(nodular liver or enlargement of portal vein and splenomegaly)
- Combined low platelet count (<140 000/mm3) with
- persistent decrease in serum albumin, or
- elevation in prothrombin time /INR (not due to antithrombotic agent use), or
- elevated bilirubin (2× ULN)
3. Satisfy the criteria of the modified CP classification for hepatic impairment during
Screening:
- Moderate: CP-B (Scores 7 to 9) or
- Severe: CP-C (Scores 10 to 12)
4. MELD score of 6 to 24 at Screening
5. Taking UDCA for at least 12 months (stable dose for =3 months) prior to Day 1, or
unable to tolerate or unresponsive to UDCA (no UDCA for =3 months)
Exclusion Criteria:
1. Non-cirrhotic or cirrhotic CP-A (Mild; Score 5 to 6)
2. History of liver transplant or organ transplant
3. History of alcohol or drug abuse within 12 months prior to Screening
4. Hepatic encephalopathy (as defined by a West Haven score of =2 [AASLD, EASL 2014])
5. History or presence of other concomitant liver diseases including:
- Hepatitis C virus infection and RNA positive
- Active hepatitis B infection; however, patients who have seroconverted (hepatitis
B surface antigen and hepatitis B e antigen negative) may be included in this
study after consultation with the medical monitor
- Primary sclerosing cholangitis
- Alcoholic liver disease
- Definite autoimmune liver disease or overlap hepatitis
- Gilbert's Syndrome
6. In the opinion of the Investigator, fluctuating or rapidly deteriorating hepatic
function prior to randomization
-
Further information on trial
Date trial registered
Apr 8, 2018
Incorporation of the first participant
Jun 22, 2018
Recruitment status
Active, not recruiting
Academic title
(Data source: WHO)
A Phase 4, Double-Blind, Randomized, Placebo-Controlled Study Evaluating the Pharmacokinetics and Safety of Obeticholic Acid in Patients With Primary Biliary Cholangitis and Moderate to Severe Hepatic Impairment
Type of trial
(Data source: WHO)
Interventional
Design of the trial
(Data source: WHO)
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
Phase
(Data source: WHO)
Phase 4
Primary end point
(Data source: WHO)
Evaluate safety and tolerability as assessed by the incidence of treatment emergent adverse events and serious treatment emergent adverse events comparing OCA to placebo;Evaluate area under the concentration curve versus time curve from 0 to 24 hours (AUC 0-24) of OCA, its conjugates and total OCA;Evaluate maximum concentration (Cmax) of OCA, its conjugates and total OCA (sum of OCA and its conjugates)
Secundary end point
(Data source: WHO)
Evaluate the effect of OCA treatment compared to placebo on plasma bile acids (µmol/L);. Evaluate the effect of OCA treatment compared to placebo on 7a hydroxy-4-cholesten-3-one (ng/mL);Evaluate the effect of OCA treatment compared to placebo on fibroblast growth factor-19 (pg/mL);Evaluate the effect of OCA treatment compared to placebo on platelets (109/L);Evaluate the effect of OCA treatment compared to placebo on alkaline phosphatase (U/L), alanine aminotransferase (U/L), aspartate transaminase (U/L), and gamma glutamyl transaminase (U/L);Evaluate the effect of OCA treatment compared to placebo on total bilirubin (mg/dL) and direct bilirubin (mg/dL);Evaluate the effect of OCA treatment compared to placebo on Child-Pugh score and its components;Evaluate the effect of OCA treatment compared to placebo on the model of end-stage liver disease (MELD) and its components
Contact information
(Data source: WHO)
Please refer to primary and secondary sponsors
Trial results
(Data source: WHO)
Results summary
no information available yet
Link to the results in the primary register
no information available yet
Information on the availability of individual participant data
no information available yet
Trial sites
Trial sites in Switzerland
(Data source: BASEC)
Bern
Countries
(Data source: WHO)
Switzerland might not appear as site of trial if it has not yet been entered as such in the WHO primary registry.
Argentina, Australia, Belgium, Brazil, Canada, Estonia, Germany, Hungary, Italy, Lithuania, Spain, United States
Contact for further information on the trial
Details of contact in Switzerland
(Data source: BASEC)
Beat Weiss
+41-31-543 01 19
b.weiss@weiss-company.ch
Contact for general information
(Data source: WHO)
Christian Weyer, M.D.
Intercept Pharmaceuticals
Contact for scientific information
(Data source: WHO)
Christian Weyer, M.D.
Intercept Pharmaceuticals
Authorisation by the ethics committee (Data source: BASEC)
Name of the authorising ethics committee (for multicentre studies only the lead committee)
Kantonale
Ethikkommission Bern
Date of authorisation by the ethics committee
07.04.2020
Further trial identification numbers
Trial identification number of the ethics committee (BASEC-ID)
(Data source: BASEC)
2019-00181
Secondary ID (Data source: WHO)
747-401
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