Clinical Trial: Phase 3 Study of Obeticholic Acid in Patients With Primary Biliary Cirrhosis

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Phase 3, Double Blind, Placebo Controlled Trial and Long Term Safety Extension of Obeticholic Acid in Patients With Primary Biliary Cirrhosis

Brief Summary: The investigational drug, Obeticholic Acid (OCA) is a modified bile acid and FXR agonist. FXR is a key regulator of bile acid synthesis and transport. Bile acids are used by the body to help with digestion. It is hypothesized that regular treatment with OCA will improve liver function in persons with Primary Biliary Cirrhosis (PBC).

Detailed Summary:
Sponsor: Intercept Pharmaceuticals

Current Primary Outcome: Composite Endpoint Alkaline Phosphatase and Total Bilirubin, 10 mg OCA vs. Placebo [ Time Frame: 12 months ]

Proportion of subjects at Month 12 with ALP < 1.67x ULN and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline.


Original Primary Outcome:

  • Composite endpoint Alkaline Phosphatase and total bilirubin [ Time Frame: 6 months and 12 months ]
    Alkaline phosphatase less than 1.67 times upper limit normal and total bilirubin within normal limits
  • alkaline phosphatase [ Time Frame: 6 months and 12 months ]
    Greater than or equal 15% decrease in alkaline phosphatase


Current Secondary Outcome:

  • Composite Endpoint Alkaline Phosphatase and Total Bilirubin, 10 mg vs. Placebo [ Time Frame: 6 months ]
    Proportion of subjects at Month 6 with ALP < 1.67x ULN and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline.
  • Composite Endpoint Alkaline Phosphatase and Total Bilirubin, 5-10 mg vs. Placebo [ Time Frame: 12 Months ]
    Proportion of subjects at Month 12 with ALP < 1.67x ULN and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline.
  • Composite Endpoint Alkaline Phosphatase and Total Bilirubin, 5-10 mg vs. Placebo [ Time Frame: 6 Months ]
    Proportion of subjects at Month 6 with ALP < 1.67x ULN and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline.
  • Alkaline Phosphatase Absolute Change From Baseline to Month 12 [ Time Frame: 12 months ]
    Alkaline Phosphatase Absolute Change from Baseline to Month 12
  • Total Bilirubin Absolute Change From Baseline to Month 12 [ Time Frame: 12 months ]
    Total Bilirubin Absolute Change from Baseline to Month 12
  • Direct Bilirubin Absolute Change From Baseline to Month 12 [ Time Frame: 12 months ]
    Direct Bilirubin Absolute Change from Baseline to Month 12
  • Alanine Aminotransferase (ALT) Absolute Change From Baseline to Month 12 [ Time Frame: 12 months ]
    Alanine Aminotransferase (ALT) Absolute Change from Baseline to Month 12
  • Aspartate Aminotransferase (AST) Absolute Change From Baseline to Month 12 [ Time Frame: 12 months ]
    Aspartate Aminotransferase (AST) Absolute Change from Baseline to Month 12
  • Gamma-glutamyltransferase (GGT) Absolute Change From Baseline to Month 12 [ Time Frame: 12 months ]
    Gamma-glutamyltransferase (GGT) Absolute Change from Baseline to Month 12


Original Secondary Outcome:

  • Alkaline phosphatase [ Time Frame: 12 months ]
    Alkaline phosphatase response rates of 10%, 20% and 40% change
  • Alkaline phosphatase/aspartate aminotransferase/bilirubin [ Time Frame: 12 months ]
    Alkaline phosphatase less than or equal to 3 times upper limit noraml and aspartate aminotransferase less than or equal to 2 times upper limit normal and normal bilirubin
  • Alkaline phosphatase/aspartate aminotransferase/bilirubin [ Time Frame: 12 months ]
    Alkaline phosphatase less than or equal to 1.5 times upper limit noraml and aspartate aminotransferase less than or equal to 1.5 times upper limit normal and normal bilirubin
  • Bilirubin and albumin [ Time Frame: 12 months ]
    Normal bilirubin and normal albumin


Information By: Intercept Pharmaceuticals

Dates:
Date Received: November 14, 2011
Date Started: January 2012
Date Completion: June 2018
Last Updated: December 20, 2016
Last Verified: December 2016