Study to investigate the efficacy and safety of two doses of K-808 (pemafribate) in subjects with PBC.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
46
Administered orally once daily
Administered orally once daily
Administered orally once daily
Percent change from baseline in serum alkaline phosphatase (ALP)
Two doses of K-808 compared to placebo after 12 weeks of treatment
Time frame: Baseline to Week 12
Achievement of normalization of ALP level
ALP ≤1 × upper limit of normal (ULN)
Time frame: Baseline to Week 12
Achievement of target levels of ALP and total bilirubin (TB)
After two doses of K-808
Time frame: Baseline to Weeks 12 and 64
Change from baseline in liver function parameters
liver function test results including ALP, total and conjugated bilirubin, albumin, international normalized ratio \[INR\], γ-GT, ALT, AST, albumin, platelets count
Time frame: Baseline to Weeks 12 and 64
Change from baseline in GLOBE risk score
calculated by GLOBE scoring system which is calculated based on serum values of bilirubin, ALP, albumin and platelet count.
Time frame: Baseline to Weeks 12 and 64
Change from baseline in UK-PBC score
PBC risk score developed by United Kingdom (UK)-PBC Consortium is a scoring system and the calculation is based on laboratory test measurements and upper limits of normal (ULN) for the total bilirubin (BIL12); alanine transaminase or aspartate transaminase (TA12), and alkaline phosphatase (ALP12) after at least 12 months of UDCA, and the laboratory test measurements and lower limits of normal (LLN) for the serum albumin and platelet count in the same timeframe. A high number is indicative of a worse score.
Time frame: Baseline to Weeks 12 and 64
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UA Thomas D. Boyer Liver Institute
Tucson, Arizona, United States
Southern California Research Center - Coronado
Coronado, California, United States
Cedars-Sinai Medical Center
Los Angeles, California, United States
Velocity Clinical Research
Santa Ana, California, United States
Peak Gastroenterology Associates Colorado Springs
Colorado Springs, Colorado, United States
University of Florida Hepatology Research at CTRB
Gainesville, Florida, United States
Florida Research Institute
Lakewood Rch, Florida, United States
University of Miami Leonard M. Miller School of Medicine
Miami, Florida, United States
Springfield Clinic
Springfield, Illinois, United States
Mercy Medical Center - Mcauley Plaza
Baltimore, Maryland, United States
...and 28 more locations
Incidence of Treatment Emergent Adverse Events (TEAEs)
Coded using the most recent version of Medical Dictionary of Regulatory Activities (MedDRA).
Time frame: Baseline to Week 68