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

NCT05239468 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: 747-214
• Study Type: interventional
• Target: 72
• Locations: 4 countries
• Sites: 26

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).

Conditions

Primary Biliary Cholangitis

Keywords

Primary Biliary Cholangitis; Primary Biliary Cirrhosis; Hepatic Impairment; Cirrhosis; Liver

Outcome Measures

Primary Outcomes (1)

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

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

Secondary Outcomes (9)

• 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

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

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

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

• Change from Baseline in biochemical disease marker GGT

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

• Change from Baseline in biochemical disease marker ALT

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

• Change from Baseline in biochemical disease marker AST

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

Study Arms (5)

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

ACTIVE COMPARATOR

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

Drug: Bezafibrate 100 mg; Drug: Obeticholic Acid placebo; Drug: Bezafibrate Placebo

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

ACTIVE COMPARATOR

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

Drug: Bezafibrate 200 mg; Drug: Obeticholic Acid placebo

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

EXPERIMENTAL

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

Drug: Bezafibrate 100 mg; Drug: Obeticholic Acid 5 mg; Drug: Bezafibrate Placebo

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

EXPERIMENTAL

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

Drug: Bezafibrate 200 mg; Drug: Obeticholic Acid 5 mg

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

EXPERIMENTAL

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

Drug: Bezafibrate 200 mg; Drug: Obeticholic Acid 5 mg

Interventions

Bezafibrate 100 mg DRUG

One tablet of bezafibrate 100 mg IR once daily

Double Blind (DB) Phase Treatment A: BZF 100 milligrams (mg) Immediate Release (IR) tablet; Double Blind (DB) Phase Treatment C: OCA 5 mg + BZF 100 mg IR

Bezafibrate 200 mg DRUG

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

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

Obeticholic Acid 5 mg DRUG

One tablet of obeticholic acid 5 mg tablet once daily.

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

Obeticholic Acid placebo DRUG

One tablet of obeticholic acid placebo tablet once daily

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

Bezafibrate Placebo DRUG

One tablet of bezafibrate placebo tablet once daily

Double Blind (DB) Phase Treatment A: BZF 100 milligrams (mg) Immediate Release (IR) tablet; Double Blind (DB) Phase Treatment C: OCA 5 mg + BZF 100 mg IR

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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 farnesoid X receptor (FXR) agonists, or peroxisome proliferator activated receptor (PPAR)-agonists within 3 months before Screening
• Unable to tolerate BZF or other fibrates, treatment with commercially available fibrates, or participation in a previous study involving fibrate within 3 months before Screening.

Sponsors & Collaborators

• Intercept Pharmaceuticals: lead

Study Sites (26)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

Location

Southern California Gastrointestinal (GI) and Liver Centers (SCLC) - Coronado

Coronado, California, 92118, United States

Location

Facey Medical Group

Mission Hills, California, 91345, United States

Location

Schiff Center for Liver Diseases / University of Miami

Miami, Florida, 33136, United States

Location

Tampa General Medical Group

Tampa, Florida, 33606, United States

Location

Piedmont Atlanta Hospital

Atlanta, Georgia, 30309, United States

Location

Loyola University Medical Center

Maywood, Illinois, 60459, United States

Location

Ochsner Medical Center

New Orleans, Louisiana, 70121, United States

Location

Beth Israel Deaconess Medical Center Harvard Liver Research Center

Boston, Massachusetts, 02215, United States

Location

NYU Langone Medical Center

New York, New York, 10016-6402, United States

Location

Wake Endoscopy Center

Raleigh, North Carolina, 27607, United States

Location

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

Gastro One

Cordova, Tennessee, 38018, United States

Location

Gastrointestinal Associates of Northeast Tennessee

Johnson City, Tennessee, 37604, United States

Location

Methodist Clinical Research Institute (CRI)

Dallas, Texas, 75203, United States

Location

Houston Methodist Cancer Center

Houston, Texas, 77030-2717, United States

Location

American Research Corporation at the Texas Liver Institute

San Antonio, Texas, 78215, United States

Location

Hospital Italiano La Plata

La Plata, Buenos Aires, Argentina

Location

DIM Clinical Privada

Ramos Mejía, Buenos Aires, Argentina

Location

Hospital Italiano de Buenos Aires

Buenos Aires, Argentina

Location

Hospital Universitario Austral

Pilar, Argentina

Location

Hospital Provincial del Centenario

Rosario, Argentina

Location

The Northern Alberta Clinical Trials and Research Centre

Edmonton, Alberta, T6G 287, Canada

Location

Pacific Gastroenterology Associates

Vancouver, British Columbia, V6Z 2K5, Canada

Location

University of Montreal

Montreal, Quebec, Canada

Location

Azienda Ospedaliero-Universitaria di Bologna Policlinico S. Orsola-Malpighi

Bologna, Italy

Location

MeSH Terms

Conditions

Liver Cirrhosis, Biliary; Fibrosis

Interventions

Bezafibrate; obeticholic acid

Condition Hierarchy (Ancestors)

Cholestasis, Intrahepatic; Cholestasis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Liver Diseases; Liver Cirrhosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Benzamides; Amides; Organic Chemicals; Fibric Acids; Isobutyrates; Butyrates; Acids, Acyclic; Carboxylic Acids; Benzoates; Acids, Carbocyclic; Chlorobenzoates; Phenyl Ethers; Ethers; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Phenols

Study Officials

• Lynda Szczech, M.D.

  Intercept Pharmaceuticals, Inc

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 28, 2022
• First Posted: February 15, 2022
• Study Start: March 21, 2022
• Primary Completion: September 1, 2025
• Study Completion: September 1, 2025
• Last Updated: September 25, 2025

Record last verified: 2025-09

Locations

CA (3); US (17); IT (1); AR (5)