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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03155932
• Date of registration: 11/05/2017
• Prospective Registration: Yes
• Primary sponsor: Arena Pharmaceuticals
• Public title: Safety, Tolerability, and Efficacy of Etrasimod (APD334) in Patients With Primary Biliary Cholangitis
• Scientific title: An Open-label, Pilot, Proof of Concept Study to Evaluate the Safety, Tolerability, and Efficacy of Oral Etrasimod (APD334) in Patients With Primary Biliary Cholangitis
• Date of first enrolment: December 15, 2017
• Target sample size: 2
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT03155932
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Australia, New Zealand, United States

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Primary Biliary Cholangitis

Intervention(s)

Drug: APD334

Primary Outcome(s)

Change from baseline to Week 24 in serum ALP concentration. [Time Frame: Week 24]

Number of patients with adverse events and abnormal clinical laboratory tests (including hematology, serum chemistry, coagulation and urinalysis). [Time Frame: Week 26]

Secondary Outcome(s)

Change from baseline to Week 12 and 24 in fatigue [Time Frame: Weeks 12 and 24]

Change from baseline to Week 12 in serum ALP concentration. [Time Frame: Week 12]

Change from baseline to Week 12 and 24 in concentration of serum GGT [Time Frame: Weeks 12 and 24]

Change from baseline to Week 12 and 24 in concentration of serum GP73 [Time Frame: Weeks 12 and 24]

Change from baseline to Week 12 and 24 in concentration of serum ALT [Time Frame: Weeks 12 and 24]

Change from baseline to Week 12 and 24 in concentration of serum AMA [Time Frame: Weeks 12 and 24]

Change from baseline to Week 12 and 24 in complete blood counts [Time Frame: Weeks 12 and 24]

Change from baseline to Week 12 and 24 in concentration of serum C4 [Time Frame: Weeks 12 and 24]

Change from baseline to Week 12 and 24 in concentration of serum HsCRP [Time Frame: Weeks 12 and 24]

Change from baseline to Week 12 and 24 in pruritus [Time Frame: Weeks 12 and 24]

Change from baseline to Week 12 and 24 in Schirmer test outcome [Time Frame: Weeks 12 and 24]

Change from baseline to Week 12 and 24 in tear film break-up time [Time Frame: Weeks 12 and 24]

Change from baseline to Week 12 and 24 in quality of life [Time Frame: Weeks 12 and 24]

Change from baseline to Week 12 and 24 in concentration of serum AST [Time Frame: Weeks 12 and 24]

Change from baseline to Week 12 and 24 in concentration of serum immunoglobulin [Time Frame: Weeks 12 and 24]

Secondary ID(s)

APD334-010

Source(s) of Monetary Support

Please refer to primary and secondary sponsors