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

NCT03155932 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: APD334-010
• Study Type: interventional
• Target: 2
• Locations: 3 countries
• Sites: 9

Brief 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 participants with primary biliary cholangitis (PBC).

Conditions

Primary Biliary Cholangitis

Outcome Measures

Primary Outcomes (2)

• Change in Serum Alkaline Phosphatase (ALP) Concentration

  Reduction in ALP concentration is a surrogate marker of slower disease progression.

  Baseline, Week 24

• Number of Participants With Adverse Events

  Safety was assessed by monitoring adverse events and clinically relevant changes in vital signs and clinical laboratory results.

  Up to Week 26

Secondary Outcomes (2)

• Change in Serum ALP Concentration

  Baseline, Week 12

• Pharmacokinetic Parameters of Etrasimod, and Its Metabolites

  Up to Week 24

Other Outcomes (14)

• Exploratory - Change in Complete Blood Count

  Baseline, Week 12, Week 24

• Exploratory - Change in Incidence of Fatigue as Assessed by Peripheral Biliary Cholangitis (PBC-40) Scale

  Baseline, Week 12, Week 24

• Exploratory - Change in Incidence of Pruritus as Assessed by 5-Dimensions (5-D) Itch Scale

  Baseline, Week 12, Week 24

Study Arms (1)

APD334

EXPERIMENTAL

APD334 active treatment for 24 weeks.

Drug: APD334

Interventions

APD334 DRUG

APD334 active treatment for 24 weeks.

Also known as: etrasimod

APD334

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Arena Pharmaceuticals: lead

Study Sites (9)

Gastroenterology and Hepatology, UC Davis Medical Center

Sacramento, California, 95817, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Texas Liver Institute

San Antonio, Texas, 78215, United States

Location

Swedish Medical Center

Seattle, Washington, 98104, United States

Location

Royal Prince Alfred Hospital

Camperdown, New South Wales, 2050, Australia

Location

Sunshine Coast University Hospital

Birtinya, Queensland, 4575, Australia

Location

Alfred Health

Melbourne, Victoria, 3004, Australia

Location

Auckland City Hospital

Grafton, Auckland, 1023, New Zealand

Location

Christchurch Clinical Studies Trust

Christchurch, 8011, New Zealand

Location

MeSH Terms

Conditions

Liver Cirrhosis, Biliary

Interventions

etrasimod

Condition Hierarchy (Ancestors)

Cholestasis, Intrahepatic; Cholestasis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Liver Diseases; Liver Cirrhosis; Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Arena CT.gov Administrator
• Organization: Arena Pharmaceuticals, Inc.

ct.gov@arenapharm.com

+1 855-218-9153

Study Officials

• Arena CT.gov Administrator

  Arena Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open label

Study Record Dates

• First Submitted: May 11, 2017
• First Posted: May 16, 2017
• Study Start: December 29, 2017
• Primary Completion: January 31, 2019
• Study Completion: January 31, 2019
• Last Updated: March 24, 2022
• Results First Posted: March 24, 2022

Record last verified: 2022-02

Data Sharing

• IPD Sharing: Will not share

Locations

AU (3); NZ (2); US (4)