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Study of OCA in Combination With BZF Evaluating Efficacy, Safety and Tolerability in Participants With PBC

Primary Purpose

Primary Biliary Cholangitis

Status

Active

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Bezafibrate 100 mg

Bezafibrate 200 mg

Obeticholic Acid 5 mg

Obeticholic Acid placebo

Bezafibrate Placebo

About this trial

This is an interventional treatment trial for Primary Biliary Cholangitis focused on measuring Primary Biliary Cholangitis, Primary Biliary Cirrhosis, Hepatic Impairment, Cirrhosis, Liver

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

A definite or probable diagnosis of PBC
Qualifying ALP and/or bilirubin liver biochemistry values
Taking ursodeoxycholic acid (UDCA) for at least 12 months or no UDCA for 3 months before Day 1

Exclusion Criteria:

History or presence of other concomitant liver diseases
Presence of clinical complications of PBC
History or presence of decompensating events
Current or history of gallbladder disease
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, or other FXR agonists, or peroxisome proliferator activated receptor (PPAR)-agonists within 3 months before Screening
Treatment with commercially available fibrates, or participation in a previous study involving fibrate within 3 months before Screening

Sites / Locations

University of Alabama at Birmingham
Southern California Gastrointestinal (GI) and Liver Centers (SCLC) - Coronado
Facey Medical Group
UC Davis Medical Center
Schiff Center for Liver Diseases / University of Miami
Tampa General Medical Group
Piedmont Atlanta Hospital
Loyola University Medical Center
University of Louisville - Human Subjects Protection Program
Ochsner Medical Center
Mercy Medical Center
Beth Israel Deaconess Medical Center Harvard Liver Research Center
Henry Ford Health System
Southwest Gastroenterology Associates, PC (SWGA)
NYU Langone Medical Center
Wake Endoscopy Center
University Hospitals Cleveland Medical Center
Gastro One
Gastrointestinal Associates of Northeast Tennessee
Methodist Clinical Research Institute (CRI)
Houston Methodist Cancer Center
American Research Corporation at the Texas Liver Institute

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm Type

Active Comparator

Experimental

Arm Label

Double Blind (DB) Phase Treatment A: BZF 100 milligrams (mg) Immediate Release (IR) tablet

Double Blind (DB) Phase Treatment B: BZF 400 mg IR tablet

Double Blind (DB) Phase Treatment C: OCA 5 mg + BZF 100 mg IR

Double Blind (DB) Phase Treatment D: OCA 5 mg + BZF 400 mg IR

Long Term Safety Extension (LTSE) Phase Treatment D of the DB phase: OCA 5 mg + BZF 400 mg IR

Arm Description

Each Participant will take one OCA placebo tablet, one BZF 100 mg IR tablet and one BZF placebo tablet daily.

Each Participant will take one OCA placebo tablet and two BZF 200 mg IR tablets (to achieve 400 mg dose) daily.

Each participant will take one OCA 5 mg tablet, one BZF 100 mg IR tablet and one BZF placebo tablet, daily.

Each participant will take one OCA 5 mg tablet and two BZF 200 mg IR tablets (to achieve 400 mg dose) daily.

Outcomes

Primary Outcome Measures

Change in Alkaline Phosphatase (ALP) from Baseline to Week 12

Secondary Outcome Measures

Change from Baseline in response rates of ≥10 percent, ≥20 percent, ≥30 percent and ≥40 percent reduction and normalization rates of biochemical disease marker ALP

Number of participants with normalization rates of alanine aminotransferase (ALT), gamma-glutamyl transferase (GGT), alanine aminotransferase (AST), total and conjugated bilirubin and lipid panel

Change from Baseline in biochemical disease marker GGT

Change from Baseline in biochemical disease marker ALT

Change from Baseline in biochemical disease marker AST

Change from Baseline in biochemical disease markers, total & conjugated bilirubin

Change from Baseline in lipid panel

Change from Baseline of the plasma value of 7 alpha (α) hydroxy 4 cholesten-3 one (C4)

Change from Baseline of the plasma value of bile acids, in unit of nanograms per milliliter (ng/ml)

Full Information

NCT ID

NCT05239468

First Posted

January 28, 2022

Last Updated

August 4, 2023

Sponsor

Intercept Pharmaceuticals

1. Study Identification

Unique Protocol Identification Number

NCT05239468

Brief Title

Study of OCA in Combination With BZF Evaluating Efficacy, Safety and Tolerability in Participants With PBC

Official Title

A Phase 2a, Double-Blind, Randomized, Active Controlled, Parallel Group Study Evaluating the Efficacy, Safety, and Tolerability of Bezafibrate Administered in Combination With Obeticholic Acid in Subjects With Primary Biliary Cholangitis

Study Type

Interventional

2. Study Status

Record Verification Date

August 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

March 21, 2022 (Actual)

Primary Completion Date

August 2024 (Anticipated)

Study Completion Date

August 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Intercept Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Primary Biliary Cholangitis

Keywords

Primary Biliary Cholangitis, Primary Biliary Cirrhosis, Hepatic Impairment, Cirrhosis, Liver

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

60 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Double Blind (DB) Phase Treatment A: BZF 100 milligrams (mg) Immediate Release (IR) tablet

Arm Type

Active Comparator

Arm Description

Each Participant will take one OCA placebo tablet, one BZF 100 mg IR tablet and one BZF placebo tablet daily.

Arm Title

Double Blind (DB) Phase Treatment B: BZF 400 mg IR tablet

Arm Type

Active Comparator

Arm Description

Each Participant will take one OCA placebo tablet and two BZF 200 mg IR tablets (to achieve 400 mg dose) daily.

Arm Title

Double Blind (DB) Phase Treatment C: OCA 5 mg + BZF 100 mg IR

Arm Type

Experimental

Arm Description

Each participant will take one OCA 5 mg tablet, one BZF 100 mg IR tablet and one BZF placebo tablet, daily.

Arm Title

Double Blind (DB) Phase Treatment D: OCA 5 mg + BZF 400 mg IR

Arm Type

Experimental

Arm Description

Each participant will take one OCA 5 mg tablet and two BZF 200 mg IR tablets (to achieve 400 mg dose) daily.

Arm Title

Long Term Safety Extension (LTSE) Phase Treatment D of the DB phase: OCA 5 mg + BZF 400 mg IR

Arm Type

Experimental

Arm Description

Each participant will take one OCA 5 mg tablet and two BZF 200 mg IR tablets (to achieve 400 mg dose) daily.

Intervention Type

Drug

Intervention Name(s)

Bezafibrate 100 mg

Intervention Description

One tablet of bezafibrate 100 mg IR once daily

Intervention Type

Drug

Intervention Name(s)

Bezafibrate 200 mg

Intervention Description

Two tablets of bezafibrate 200 mg IR once daily for BZF 400 mg IR

Intervention Type

Drug

Intervention Name(s)

Obeticholic Acid 5 mg

Intervention Description

One tablet of obeticholic acid 5 mg tablet once daily.

Intervention Type

Drug

Intervention Name(s)

Obeticholic Acid placebo

Intervention Description

One tablet of obeticholic acid placebo tablet once daily

Intervention Type

Drug

Intervention Name(s)

Bezafibrate Placebo

Intervention Description

One tablet of bezafibrate placebo tablet once daily

Primary Outcome Measure Information:

Title

Change in Alkaline Phosphatase (ALP) from Baseline to Week 12

Time Frame

Baseline, and at Weeks 2, 4, 6, 8, 10 and 12

Secondary Outcome Measure Information:

Title

Change from Baseline in response rates of ≥10 percent, ≥20 percent, ≥30 percent and ≥40 percent reduction and normalization rates of biochemical disease marker ALP

Time Frame