Study to determine if the investigational drug obeticholic acid (also known as OCA) in combination with the investigational drug bezafibrate (BZF), has an effect on Primary Biliary Cholangitis (also known as Primary Biliary Cirrhosis or PBC).
- Conditions
- Primary Biliary Cholangitis (PBC)MedDRA version: 21.0Level: LLTClassification code 10036680Term: Primary biliary cirrhosisSystem Organ Class: 100000004871Therapeutic area: Diseases [C] - Digestive System Diseases [C06]
- Registration Number
- EUCTR2018-002575-17-GR
- Lead Sponsor
- Intercept Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 78
- A definite or probable diagnosis of PBC (consistent with the European Association for the Study of the Liver [EASL] Practice Guidelines and the American Association for the Study of Liver Diseases; [Lindor 2009a, EASL 2017])
- Qualifying ALP and/or bilirubin liver biochemistry values
- Age =18 years
- Taking UDCA for at least 12 months (stable dose for =3 months) before Day 1 or unable to tolerate or unresponsive to UDCA (no UDCA for =3 months) before Day 1
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 65
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10
- History or presence of other concomitant liver diseases
- Clinical complications of PBC
- History or presence of decompensating events
- History of or current gallbladder diseases
- If female, known pregnancy, or has a positive urine pregnancy test
(confirmed by a positive serum pregnancy test), or lactating
- Treatment with commercially available OCA or participation in a
previous study involving OCA
Note: Other protocol defined Inclusion/Exclusion criteria may apply
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The primary objective is to assess the effects of the combination of OCA and BZF on alkaline phosphatase (ALP) in comparison to BZF alone in subjects with PBC.;Secondary Objective: The secondary objectives are to assess the effects of the combination of OCA and BZF in comparison to BZF alone in subjects with PBC on the following:<br>- Biochemical disease markers, including ALP, GGT, ALT, AST, total and conjugated bilirubin and a lipid panel<br>- Biomarkers of bile acid synthesis and homeostasis, including 7a-hydroxy-4-cholesten-3-one (C4) and bile acids<br>- Safety and tolerability;Primary end point(s): Change in ALP from baseline to Week 12 in the DB Period;Timepoint(s) of evaluation of this end point: baseline to week 12
- Secondary Outcome Measures
Name Time Method Secondary end point(s): - The response rates of =10%, =20%, =30% and =40% reduction from baseline and normalization rates of ALP at Week 12 <br>- Normalization rates at Week 12 of GGT, ALT, AST, total and conjugated bilirubin, and a lipid panel<br>- Change from baseline to Week 12 in GGT, ALT, AST, and total and conjugated bilirubin, and a lipid panel<br>- Change from baseline to Week 12 in 7a-hydroxy-4-cholesten-3-one (C4) and bile acids;Timepoint(s) of evaluation of this end point: Baseline to End of study