Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Safety, Tolerability, and Efficacy of Etrasimod (APD334) in Patients 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
Last Update Posted : May 16, 2019
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 patients with primary biliary cholangitis (PBC).

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

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
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 15, 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 from baseline to Week 24 in serum ALP concentration. [ Time Frame: Week 24 ]
    Assess the change in ALP concentration following administration of APD334.

  2. Number of patients with adverse events and abnormal clinical laboratory tests (including hematology, serum chemistry, coagulation and urinalysis). [ Time Frame: Week 26 ]
    Safety and tolerability of APD334


Secondary Outcome Measures :
  1. Change from baseline to Week 12 in serum ALP concentration. [ Time Frame: Week 12 ]
    Assess the change in ALP concentration following administration of APD334.

  2. Change from baseline to Week 12 and 24 in complete blood counts [ Time Frame: Weeks 12 and 24 ]
  3. Change from baseline to Week 12 and 24 in quality of life [ Time Frame: Weeks 12 and 24 ]
    measured by the PBC-40 scale

  4. Change from baseline to Week 12 and 24 in fatigue [ Time Frame: Weeks 12 and 24 ]
    measured by the PBC-40 scale

  5. Change from baseline to Week 12 and 24 in pruritus [ Time Frame: Weeks 12 and 24 ]
    measured by the 5-D scale

  6. Change from baseline to Week 12 and 24 in Schirmer test outcome [ Time Frame: Weeks 12 and 24 ]
    Schirmer test in patients with abnormal results at screening

  7. Change from baseline to Week 12 and 24 in tear film break-up time [ Time Frame: Weeks 12 and 24 ]
    Tear film break-up time in patients with abnormal results at screening

  8. Change from baseline to Week 12 and 24 in concentration of serum HsCRP [ Time Frame: Weeks 12 and 24 ]
  9. Change from baseline to Week 12 and 24 in concentration of serum ALT [ Time Frame: Weeks 12 and 24 ]
  10. Change from baseline to Week 12 and 24 in concentration of serum AST [ Time Frame: Weeks 12 and 24 ]
  11. Change from baseline to Week 12 and 24 in concentration of serum GGT [ Time Frame: Weeks 12 and 24 ]
  12. Change from baseline to Week 12 and 24 in concentration of serum C4 [ Time Frame: Weeks 12 and 24 ]
  13. Change from baseline to Week 12 and 24 in concentration of serum immunoglobulin [ Time Frame: Weeks 12 and 24 ]
  14. Change from baseline to Week 12 and 24 in concentration of serum GP73 [ Time Frame: Weeks 12 and 24 ]
  15. Change from baseline to Week 12 and 24 in concentration of serum AMA [ Time Frame: Weeks 12 and 24 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
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 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 <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 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


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
Layout table for location information
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
Australia, Queensland
Sunshine Coast University Hospital
Birtinya, Queensland, Australia
Australia, Victoria
Alfred Health
Melbourne, Victoria, Australia, 3004
New Zealand
Auckland City Hospital
Grafton, Auckland, New Zealand
Christchurch Clinical Studies Trust
Christchurch, New Zealand
Sponsors and Collaborators
Arena Pharmaceuticals

Layout table for additonal information
Responsible Party: Arena Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03155932     History of Changes
Other Study ID Numbers: APD334-010
First Posted: May 16, 2017    Key Record Dates
Last Update Posted: May 16, 2019
Last Verified: May 2019

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
Layout table for MeSH terms
Cholangitis
Liver Cirrhosis, Biliary
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Cholestasis, Intrahepatic
Cholestasis
Liver Diseases
Liver Cirrhosis