Clinical Trial: MBX-8025 in Subjects With Primary Biliary Cholangitis (PBC) (8 Week, Dose Ranging, Open Label, Randomized Phase 2 With an Extension)

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: An 8-week, Dose Ranging, Open Label, Randomized, Phase 2 Study With an 18-week Extension, to Evaluate the Safety and Efficacy of MBX-8025 in Subjects With Primary Biliary Cholang

Brief Summary: An 8-week, dose ranging, open label, randomized, Phase 2 study with an 18-week extension, to evaluate the safety and efficacy of MBX-8025 in subjects with Primary Biliary Cholangitis (PBC) and an inadequate response to or intolerance to ursodeoxycholic acid (UDCA)

Detailed Summary:

Primary:

To evaluate the safety and efficacy of MBX-8025 5 mg, 10 mg, and 25 mg over 8 weeks of treatment

Secondary:

To evaluate the safety and efficacy of MBX-8025 5 mg, 10 mg, and 25 mg over 26 weeks of treatment

To evaluate the pharmacokinetics (PK) of MBX-8025

Exploratory:

To evaluate the effect of MBX-8025 on bile acids, additional markers of inflammation and renal function


Sponsor: CymaBay Therapeutics, Inc.

Current Primary Outcome:

  • Serum Alkaline Phosphatase (AP) [ Time Frame: over 8 weeks ]
  • Adverse events (AE) and Treatment Emergent Adverse Events (TEAEs), ECG, biochemistry, hematology and urinalysis (NCI CTCAE Version 4.0) [ Time Frame: over 8 weeks and over 26 weeks ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Aspartate Aminotransferase (AST) [ Time Frame: over 8 weeks and over 26 weeks ]
  • Alanine Aminotransferase (ALT) [ Time Frame: over 8 weeks and over 26 weeks ]
  • Gamma-glutamyl Transferase (GGT) [ Time Frame: over 8 weeks and over 26 weeks ]
  • 5'nucleotidase [ Time Frame: over 8 weeks and over 26 weeks ]
  • Bilirubin - Total Bilirubin [ Time Frame: over 8 weeks and over 26 weeks ]
  • Bilirubin - Conjugated Bilirubin [ Time Frame: over 8 weeks and over 26 weeks ]
  • Bilirubin - Unconjugated Bilirubin [ Time Frame: over 8 weeks and over 26 weeks ]
  • Bone Specific Alkaline Phosphatase (AP) [ Time Frame: over 8 weeks and over 26 weeks ]
  • Triglycerides (TG) [ Time Frame: over 8 weeks and over 26 weeks ]
  • Total Cholesterol (TC) [ Time Frame: over 8 weeks and over 26 weeks ]
  • High Density Lipoprotein Cholesterol (HDL-C) [ Time Frame: over 8 weeks and over 26 weeks ]
  • Low Density Lipoprotein Cholesterol (LDL-C) [ Time Frame: over 8 weeks and over 26 weeks ]
  • Composite Endpoint of AP and Total Bilirubin [ Time Frame: over 8 weeks and over 26 weeks ]
    • AP < 1.67 × upper limit of normal (ULN)
    • Total Bilirubin within normal limit
    • > 15% decrease in AP
  • Published PBC Response Criteria - Paris I [ Time Frame: over 8 weeks and over 26 weeks ]
  • Published PBC Response Criteria - Paris II [ Time Frame: over 8 weeks and over 26 weeks ]
  • Published PBC Response Criteria - Toronto I [ Time Frame: over 8 weeks and over 26 weeks ]
  • Published PBC Response Criteria - Toronto II [ Time Frame: over 8 weeks and over 26 weeks ]
  • Published PBC Response Criteria - UK-PBC Risk Score [ Time Frame: over 8 weeks and over 26 weeks ]
  • 5D-itch Scale [ Time Frame: over 8 weeks and over 26 weeks ]
  • Pruritus Visual Analog Score (VAS) [ Time Frame: over 8 weeks and over 26 weeks ]
  • PBC-40 Quality of Life (QoL) [ Time Frame: over 8 weeks and over 26 weeks ]
  • Serum Alkaline Phosphatase (AP) [ Time Frame: over 26 weeks ]
  • PK of MBX-8025 and Metabolites [ Time Frame: (at 0, 0.5, 1, 2, 4, 6 and 24 hours for Cmax, Tmax, T1/2 and AUC) ]


Original Secondary Outcome: Same as current

Information By: CymaBay Therapeutics, Inc.

Dates:
Date Received: November 2, 2016
Date Started: December 2016
Date Completion: February 2018
Last Updated: May 18, 2017
Last Verified: May 2017