Study to evaluate the efficacy, safety, and tolerability of investigational drug obeticholic acid (OCA) in combination with the investigational drug bezafibrate (BZF) in participants with Primary Biliary Cholangitis (PBC).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
75
5 mg tablet of OCA once daily titrating up to a maximum of 10 mg OCA once daily
200 mg IR tablet of Bezafibrate once daily for the remainder of the study
One tablet daily for the remainder of the study
Change in Alkaline Phosphatase (ALP) From Baseline in the Double-Blind Treatment Period
Serum samples were collected at scheduled visits during the double-blind treatment period. Changes in ALP over time will be analyzed using a mixed model for repeated measures (MMRM) to assess treatment group effects across study visits. Baseline was defined as the mean of all available evaluations prior to the first administration of investigational product in the DB period. 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 in the Double-Blind Treatment Period
Responders were defined as participants achieving a ≥10%, ≥20%, ≥30%, or ≥40% reduction from baseline in serum ALP at Week 12 with non-responder imputation applied. Baseline ALP was defined as the mean of all available evaluations prior to the first administration of investigational product in the DB period. 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: Week 12
Normalization Rates of ALP at Week 12 in the Double-Blind Treatment Period
Percentage of participants achieving a response in serum ALP at Week 12 was assessed using non-responder imputation. Analyses of ALP response rates and normalization rates were performed using a Cochran-Mantel-Haenszel test stratified by the randomization stratification factor.
Time frame: Week 12
Normalization Rates of Biochemical Disease Markers in the Double-Blind Treatment Period
Percentage of participants achieving a response in biochemical disease markers including Alanine Aminotransferase (ALT), Gamma-Glutamyl Transpeptidase (GGT), Aspartate Aminotransferase (AST), Total and conjugated Bilirubin and lipid panel (cholesterol, high density lipoprotein \[HDL\] and low-density lipoprotein \[LDL\]) at Week 12 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 daily for the remainder of the study
400 mg SR tablet of Bezafibrate once daily for the remainder of the study
One tablet daily for the remainder of the study
OCA one tablet will be administered.
Bezafibrate one tablet will be administered.
Flinders Medical Centre
Bedford Park, Perth, Australia
Royal Adelaide Hospital
Adelaide, Australia
UZ Gasthuisberg
Leuven, Belgium
Clinical Hospital Dubrava
Zagreb, Croatia
Zagreb University Hospital Center
Zagreb, Croatia
Hepato-Gastroenterologie HK, s.r.o.
Hradec Králové, Czechia
Artroscan s.r.o., Gastroenterologicka ambulance
Ostrava, Czechia
Research Site s.r.o.
Pilsen, Czechia
Tartu University Hospital
Tartu, Estonia
Hôpital Henri Mondor
Créteil, France
...and 23 more locations
Time frame: Week 12
Change From Baseline in GGT, ALT and AST Levels in the Double-Blind Treatment Period
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 in the DB period. 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 in the Double-Blind Treatment Period
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 in the DB period. 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 in the Double-Blind Treatment Period
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 in the DB period. Change from Baseline was calculated as post Baseline value minus Baseline value.
Time frame: Baseline and at Week 12
Change From Baseline in 7 Alpha (α) Hydroxy 4 Cholesten-3 One (C4) in the Double-Blind Treatment Period
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 change equals post baseline value minus baseline value.
Time frame: Baseline and at Week 12
Change From Baseline in Bile Acid in the Double-Blind Treatment Period
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. Change from Baseline was calculated as change equals post Baseline value minus Baseline value.
Time frame: Baseline and at Week 12