This study will evaluate the safety, tolerability and influence on itching, bile acids and liver enzyme changes in patients with PBC (Primary Biliary Cirrhosis) treated with A4250
Who can participate
Age range18 Years – 80 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Major Inclusion Criteria:
* Diagnosis of PBC or PBC-Autoimmune hepatitis overlap as established according to American Association for the Study of Liver Diseases/European Association for the Study of Liver (AASLD/EASL) definitions. Definite or probable PBC diagnosis, as demonstrated by the presence of ≥ 2 of the following 3 diagnostic factors:
* History of elevated alkaline phosphatase (ALP) levels (\>1.67 ULN) for at least 6 months prior to Day 1
* Positive antimitochondrial antibodies (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)
* Liver biopsy consistent with PBC;
* Ursodeoxycholic acid (UDCA) non-responders defined as \>6 months of UDCA and at the time of enrolment a serum ALP \>1.67 ULN;
* Laboratory markers of cholestasis identified within 3 months of Visit 1;
* Treatment with cholestyramine at a dose \>4g BID or colestipol \> 5mg for at least 3 months;
* The patient has a VAS-Itch of at least 30 mm during the day before baseline (Visit 2);
* The patient is a male or non-pregnant female ≥18 years of age and ≤80 years of age with body mass index (BMI) ≥18.5 but \<35 kg/m2;
Major Exclusion Criteria:
* Any condition that, in the opinion of the Investigator constitutes a risk for the patient or a contraindication for participation and completion of the study, or could interfere with study objectives, conduct…
What they're measuring
1
•Safety and Tolerability assessed by the occurrence of treatment-emergent SAEs during the four weeks of treatment with A4250