Overview

IBAT Inhibitor A4250 for Cholestatic Pruritus

Status:
Terminated
Trial end date:
2016-10-01
Target enrollment:
0
Participant gender:
All
Summary
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
Phase:
Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Sahlgrenska University Hospital, Sweden
Collaborator:
Albireo
Criteria
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, or evaluations;

- Jaundice of extrahepatic origin;

- The patient has a structural abnormality of the GI tract;

- The patient has a known, active, clinically significant acute or chronic infection, or
any major episode of infection requiring hospitalization or treatment with parenteral
anti infectives within 4 weeks of treatment start (study day 1) or completion of oral
anti-infective treatment within 2 weeks prior to start of screening period;

- The patient has unexplained and clinically significant GI alarm signals (e.g., lower
GI bleeding or heme-positive stool, iron-deficiency anaemia, unexplained weight loss)
or systemic signs of infection or colitis;