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