Safety, Tolerability, and Efficacy of Etrasimod (APD334) in Patients With Primary Biliary Cholangitis

• Status: Terminated
• Trial end date: 2019-01-31
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this open-label, pilot, proof of concept study is to evaluate the safety, tolerability, and efficacy of oral etrasimod (APD334) in patients with primary biliary cholangitis (PBC).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Arena Pharmaceuticals

Criteria

Key Inclusion Criteria:

• Males or females aged 18 to 80 years (inclusive) at the time of screening, with confirmed PBC diagnosis based upon at least 2 of 3 criteria:

• AMA titer >1:40 on immunofluorescence or M2 positive by enzyme-linked immunosorbent assay (ELISA) or positive PBC-specific antinuclear antibodies (anti-GP210 and/or anti-SP100)

• ALP >1.5 x ULN for at least 6 months

• Liver biopsy findings consistent with PBC

• Use of UDCA for at least 6 months prior to screening (stable dose for at least 3 months immediately prior to screening)

• Patients must have ALP >1.5 x ULN but <10 x ULN, ALT and AST <5 x ULN, and total bilirubin- AST, ALT, ALP, and total bilirubin must have 2 values at least 4 weeks apart that are within 20% of each other

Key Exclusion Criteria:

• Chronic liver disease of a non-PBC etiology. However, PBC patients accompanied with primary Sjögren's syndrome (pSS) are eligible to be enrolled.

• History or evidence of clinically significant hepatic decompensation

• Medical conditions that may cause non-hepatic increases in ALP (e.g., Paget's disease)

• Clinically significant infections within 6 weeks prior to treatment start, or infection with hepatitis C virus anytime in the past

• Immunosuppressive, immunomodulating, or investigational agents within 30 days prior to treatment start

• Treatment with OCA within 30 days prior to Day 1

Note: Other protocol defined Inclusion/Exclusion criteria may apply