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

Arena Pharmaceuticals

Status and phase: Terminated; Phase 2

Conditions: Primary Biliary Cholangitis

Treatments: Drug: APD334

Study type: Interventional

Funder types: Industry

Identifiers

NCT03155932

APD334-010

Details and patient eligibility

About

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

Enrollment: 2 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

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

  • Anti-mitochondrial antibodies (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)
  • Alkaline phosphatase (ALP) >1.5 x upper limit of normal (ULN) for at least 6 months
  • Liver biopsy findings consistent with PBC

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

• Participants must have ALP >1.5 x ULN but <10 x ULN, alanine aminotransferase (ALT) and aspartate aminotransferase (AST) <5 x ULN, and total bilirubin <ULN, at all screening visits

• 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 participants 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 obeticholic acid (OCA) within 30 days prior to Day 1

Note: Other protocol defined Inclusion/Exclusion criteria may apply

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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2 participants in 1 patient group

APD334

Experimental group

Description:

APD334 active treatment for 24 weeks.

Treatment:

Drug: APD334