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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03155932
Recruitment Status : Terminated (Sponsor decision)
First Posted : May 16, 2017
Results First Posted : March 24, 2022
Last Update Posted : March 24, 2022
Sponsor:
Information provided by (Responsible Party):
Arena Pharmaceuticals

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).

Condition or disease Intervention/treatment Phase
Primary Biliary Cholangitis Drug: APD334 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Open label
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : December 29, 2017
Actual Primary Completion Date : January 31, 2019
Actual Study Completion Date : January 31, 2019


Arm Intervention/treatment
Experimental: APD334
APD334 active treatment for 24 weeks.
Drug: APD334
APD334 active treatment for 24 weeks.
Other Name: etrasimod




Primary Outcome Measures :
  1. Change in Serum Alkaline Phosphatase (ALP) Concentration [ Time Frame: Baseline, Week 24 ]
    Reduction in ALP concentration is a surrogate marker of slower disease progression.

  2. Number of Participants With Adverse Events [ Time Frame: Up to Week 26 ]
    Safety was assessed by monitoring adverse events and clinically relevant changes in vital signs and clinical laboratory results.


Secondary Outcome Measures :
  1. Change in Serum ALP Concentration [ Time Frame: Baseline, Week 12 ]
    Reduction in ALP concentration is a surrogate marker of slower disease progression.

  2. Pharmacokinetic Parameters of Etrasimod, and Its Metabolites [ Time Frame: Up to Week 24 ]

Other Outcome Measures:
  1. Exploratory - Change in Complete Blood Count [ Time Frame: Baseline, Week 12, Week 24 ]
  2. Exploratory - Change in Incidence of Fatigue as Assessed by Peripheral Biliary Cholangitis (PBC-40) Scale [ Time Frame: Baseline, Week 12, Week 24 ]
  3. Exploratory - Change in Incidence of Pruritus as Assessed by 5-Dimensions (5-D) Itch Scale [ Time Frame: Baseline, Week 12, Week 24 ]
  4. Exploratory - Change in Schirmer Test Outcome [ Time Frame: Baseline, Week 12, Week 24 ]
  5. Exploratory - Change in Tear Film Break-Up Time [ Time Frame: Baseline, Week 12, Week 24 ]
  6. Exploratory - Change in Concentration of Serum High Sensitivity C-Reactive Protein (hsCRP) [ Time Frame: Baseline, Week 12, Week 24 ]
  7. Exploratory - Change in Concentration of Serum Alanine Transaminase (ALT) [ Time Frame: Baseline, Week 12, Week 24 ]
  8. Exploratory - Change in Concentration of Serum Aspartate Transaminase (AST) [ Time Frame: Baseline, Week 12, Week 24 ]
  9. Exploratory - Change in Concentration of Serum Gamma-Glutamyl Transferase (GGT) [ Time Frame: Baseline, Week 12, Week 24 ]
  10. Exploratory - Change in Concentration of Serum C4 [ Time Frame: Baseline, Week 12, Week 24 ]
  11. Exploratory - Change in Concentration of Serum Immunoglobulin [ Time Frame: Baseline, Week 12, Week 24 ]
  12. Exploratory - Change in Concentration of Serum GP73 [ Time Frame: Baseline, Week 12, Week 24 ]
  13. Exploratory - Change in Concentration of Serum Anti-Mitochondrial Antibodies (AMA) [ Time Frame: Baseline, Week 12, Week 24 ]
  14. Exploratory - Change in Quality of Life [ Time Frame: Baseline, Week 12, Week 24 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03155932


Locations
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United States, California
Gastroenterology and Hepatology, UC Davis Medical Center
Sacramento, California, United States, 95817
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
Texas Liver Institute
San Antonio, Texas, United States, 78215
United States, Washington
Swedish Medical Center
Seattle, Washington, United States, 98104
Australia, New South Wales
Royal Prince Alfred Hospital
Camperdown, New South Wales, Australia, 2050
Australia, Queensland
Sunshine Coast University Hospital
Birtinya, Queensland, Australia, 4575
Australia, Victoria
Alfred Health
Melbourne, Victoria, Australia, 3004
New Zealand
Auckland City Hospital
Grafton, Auckland, New Zealand, 1023
Christchurch Clinical Studies Trust
Christchurch, New Zealand, 8011
Sponsors and Collaborators
Arena Pharmaceuticals
Investigators
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Study Director: Arena CT.gov Administrator Arena Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Arena Pharmaceuticals:
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Responsible Party: Arena Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03155932    
Other Study ID Numbers: APD334-010
First Posted: May 16, 2017    Key Record Dates
Results First Posted: March 24, 2022
Last Update Posted: March 24, 2022
Last Verified: February 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cholangitis
Liver Cirrhosis, Biliary
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Cholestasis, Intrahepatic
Cholestasis
Liver Diseases
Liver Cirrhosis
Fibrosis
Pathologic Processes