Study to determine the effect of the investigational drug bezafibrate (BZF) alone and in combination with the investigational drug obeticholic acid (OCA) in participants with Primary Biliary Cholangitis (PBC).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
72
One tablet of bezafibrate 100 mg IR once daily
Two tablets of bezafibrate 200 mg IR once daily for BZF 400 mg IR
One tablet of obeticholic acid 5 mg tablet once daily.
Change From Baseline in Alkaline Phosphatase (ALP)
Serum samples were collected at scheduled visits during the double-blind treatment period. Changes in ALP were evaluated using a mixed-effects repeated-measures model (MMRM) to assess the effects of treatment groups over time. Baseline was defined as the mean of all available evaluations prior to the first administration of investigational product. Change from Baseline was calculated as post Baseline value minus Baseline value.
Time frame: Baseline to Week 12
Percentage of Participants With Response Rates of ≥10%, ≥20%, ≥30% and ≥40% Reduction From Baseline in ALP
Responders were defined as participants achieving a ≥10%, ≥20%, ≥30%, or ≥40% reduction from baseline in serum ALP at week 12 during the DB treatment period with non-responder imputation applied. Baseline ALP was defined as the last evaluations prior to the first administration of investigational product. The percentage of responders were summarized by treatment group for each response threshold and compared using a Cochran Mantel Haenszel test stratified by the randomization stratification factor.
Time frame: At Week 12
Normalization Rates of ALP at Week 12
Percentage of participants achieving a response in serum ALP at week 12 during the DB treatment period was assessed using non-responder imputation. Analyses of ALP normalization rates were performed using a Cochran-Mantel-Haenszel test stratified by the randomization stratification factor.
Time frame: At Week 12
Normalization Rates of Biochemical Disease Markers
Percentage of participants achieving a response in biochemical disease markers including ALP, alanine amino transferase (ALT), aspartate amino transferase (AST), gamma-glutamyl transferase (GGT), total and conjugated bilirubin and lipid panel (cholesterol, high density lipoprotein \[HDL\] and low density lipoprotein \[LDL\]) at Week 12 during the DB treatment period has been presented. Analyses of response rates and normalization rates were performed using a Cochran-Mantel-Haenszel test stratified by the randomization stratification factor.
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One tablet of obeticholic acid placebo tablet once daily
One tablet of bezafibrate placebo tablet once daily
University of Alabama at Birmingham
Birmingham, Alabama, United States
Southern California Gastrointestinal (GI) and Liver Centers (SCLC) - Coronado
Coronado, California, United States
Facey Medical Group
Mission Hills, California, United States
Schiff Center for Liver Diseases / University of Miami
Miami, Florida, United States
Tampa General Medical Group
Tampa, Florida, United States
Piedmont Atlanta Hospital
Atlanta, Georgia, United States
Loyola University Medical Center
Maywood, Illinois, United States
Ochsner Medical Center
New Orleans, Louisiana, United States
Beth Israel Deaconess Medical Center Harvard Liver Research Center
Boston, Massachusetts, United States
NYU Langone Medical Center
New York, New York, United States
...and 16 more locations
Time frame: At Week 12
Change From Baseline in in GGT, ALT and AST Levels
Blood samples were collected at indicated timepoint and Change from Baseline in GGT, ALT and AST were analyzed using the MMRM model and results were summarized in standard International System of Units (SI) by treatment group using descriptive statistics at baseline and at each on-study evaluation. Baseline was defined as the mean of all available evaluations prior to the first administration of investigational product. Change from baseline was calculated as post Baseline value minus Baseline value.
Time frame: Baseline and At Week 12
Change From Baseline in Total and Conjugated Bilirubin
Blood samples were collected at indicated timepoints and the changes in total and conjugated bilirubin were evaluated using MMRM to assess the effects of treatment groups over time. Baseline was defined as the mean of all available evaluations prior to the first administration of investigational product. Change from baseline was calculated as post Baseline value minus Baseline value.
Time frame: Baseline and At Week 12
Change From Baseline in Lipid Panel
Blood samples were collected at indicated timepoints and the changes in lipid panel including cholesterol, HDL and LDL were evaluated using MMRM to assess the effects of treatment groups over time. Baseline was defined as the mean of all available evaluations prior to the first administration of investigational product. Change from baseline was calculated as post Baseline value minus Baseline value.
Time frame: Baseline and At Week 12
Change From Baseline of the Plasma Value of 7 Alpha (α) Hydroxy 4 Cholesten-3 One (C4)
Blood samples were collected at indicated timepoints for the assessment of C4. Baseline was defined as the mean of all available evaluations prior to the first administration of investigational product. Change from baseline was calculated as post Baseline value minus Baseline value.
Time frame: Baseline and At Week 12
Number of Participants With Clinically Significant Changes From Baseline in Bile Acids
Blood samples were collected for the assessment of bile acids. Baseline was defined as the mean of all available evaluations prior to the first administration of investigational product.
Time frame: At Week 12