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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

Tracking Information
First Submitted Date  ICMJE May 11, 2017
First Posted Date  ICMJE May 16, 2017
Results First Submitted Date  ICMJE January 28, 2022
Results First Posted Date  ICMJE March 24, 2022
Last Update Posted Date March 24, 2022
Actual Study Start Date  ICMJE December 29, 2017
Actual Primary Completion Date January 31, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 28, 2022)
  • Change in Serum Alkaline Phosphatase (ALP) Concentration [ Time Frame: Baseline, Week 24 ]
    Reduction in ALP concentration is a surrogate marker of slower disease progression.
  • 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.
Original Primary Outcome Measures  ICMJE
 (submitted: May 15, 2017)
  • Change from baseline to Week 24 in serum ALP concentration. [ Time Frame: Week 24 ]
    Assess the change in ALP concentration following administration of APD334.
  • 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
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 28, 2022)
  • Change in Serum ALP Concentration [ Time Frame: Baseline, Week 12 ]
    Reduction in ALP concentration is a surrogate marker of slower disease progression.
  • Pharmacokinetic Parameters of Etrasimod, and Its Metabolites [ Time Frame: Up to Week 24 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: May 15, 2017)
  • Change from baseline to Week 12 in serum ALP concentration. [ Time Frame: Week 12 ]
    Assess the change in ALP concentration following administration of APD334.
  • 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 quality of life [ Time Frame: Weeks 12 and 24 ]
    measured by the PBC-40 scale
  • Change from baseline to Week 12 and 24 in fatigue [ Time Frame: Weeks 12 and 24 ]
    measured by the PBC-40 scale
  • Change from baseline to Week 12 and 24 in pruritus [ Time Frame: Weeks 12 and 24 ]
    measured by the 5-D scale
  • 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
  • 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
  • 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 concentration of serum ALT [ 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 GGT [ 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 immunoglobulin [ 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 AMA [ Time Frame: Weeks 12 and 24 ]
Current Other Pre-specified Outcome Measures
 (submitted: January 28, 2022)
  • Exploratory - Change in Complete Blood Count [ Time Frame: Baseline, Week 12, Week 24 ]
  • Exploratory - Change in Incidence of Fatigue as Assessed by Peripheral Biliary Cholangitis (PBC-40) Scale [ Time Frame: Baseline, Week 12, Week 24 ]
  • Exploratory - Change in Incidence of Pruritus as Assessed by 5-Dimensions (5-D) Itch Scale [ Time Frame: Baseline, Week 12, Week 24 ]
  • Exploratory - Change in Schirmer Test Outcome [ Time Frame: Baseline, Week 12, Week 24 ]
  • Exploratory - Change in Tear Film Break-Up Time [ Time Frame: Baseline, Week 12, Week 24 ]
  • Exploratory - Change in Concentration of Serum High Sensitivity C-Reactive Protein (hsCRP) [ Time Frame: Baseline, Week 12, Week 24 ]
  • Exploratory - Change in Concentration of Serum Alanine Transaminase (ALT) [ Time Frame: Baseline, Week 12, Week 24 ]
  • Exploratory - Change in Concentration of Serum Aspartate Transaminase (AST) [ Time Frame: Baseline, Week 12, Week 24 ]
  • Exploratory - Change in Concentration of Serum Gamma-Glutamyl Transferase (GGT) [ Time Frame: Baseline, Week 12, Week 24 ]
  • Exploratory - Change in Concentration of Serum C4 [ Time Frame: Baseline, Week 12, Week 24 ]
  • Exploratory - Change in Concentration of Serum Immunoglobulin [ Time Frame: Baseline, Week 12, Week 24 ]
  • Exploratory - Change in Concentration of Serum GP73 [ Time Frame: Baseline, Week 12, Week 24 ]
  • Exploratory - Change in Concentration of Serum Anti-Mitochondrial Antibodies (AMA) [ Time Frame: Baseline, Week 12, Week 24 ]
  • Exploratory - Change in Quality of Life [ Time Frame: Baseline, Week 12, Week 24 ]
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety, Tolerability, and Efficacy of Etrasimod (APD334) in Participants With Primary Biliary Cholangitis
Official Title  ICMJE 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
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).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description:
Open label
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Primary Biliary Cholangitis
Intervention  ICMJE Drug: APD334
APD334 active treatment for 24 weeks.
Other Name: etrasimod
Study Arms  ICMJE Experimental: APD334
APD334 active treatment for 24 weeks.
Intervention: Drug: APD334
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: May 14, 2019)
2
Original Estimated Enrollment  ICMJE
 (submitted: May 15, 2017)
20
Actual Study Completion Date  ICMJE January 31, 2019
Actual Primary Completion Date January 31, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   New Zealand,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03155932
Other Study ID Numbers  ICMJE APD334-010
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Arena Pharmaceuticals
Study Sponsor  ICMJE Arena Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Arena CT.gov Administrator Arena Pharmaceuticals
PRS Account Arena Pharmaceuticals
Verification Date February 2022

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP