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

NCT04594694 | Terminated Phase 2 DMC

• 1 other identifier: 747-213
• Study Type: interventional
• Target: 75
• Locations: 16 countries
• Sites: 31

Brief Summary

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).

Conditions

Primary Biliary Cholangitis

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Change in Alkaline Phosphatase (ALP) from baseline to Week 12 in the DB Treatment Period

  Baseline, Day 1, and Weeks 4, 8, and 12

Secondary Outcomes (9)

• Response rates of ≥10%, ≥20%, ≥30% and ≥40% reduction, and normalization of biochemical disease marker Alkaline Phosphatase (ALP)

  Baseline, Day 1, and Weeks 2, 4, 6, 8, and 12

• Number of participants with normalization rates of biochemical disease marker Alanine Aminotransferase (ALT), Gamma-Glutamyl Transpeptidase (GGT), Aspartate Aminotransferase (AST), total and conjugated bilirubin and lipid panel

  Baseline, Day 1, and Weeks 2, 4, 6, 8, and 12

• Change in GGT from baseline to Week 12

  Baseline, Day 1, and Weeks 4, 8, and 12

• Change in ALT from baseline to Week 12

  Baseline, Day 1, and Weeks 4, 8, and 12

• Change in AST from baseline to Week 12

  Baseline, Day 1, and Weeks 4, 8, and 12

Study Arms (5)

Treatment A: BZF 200 milligrams (mg) Immediate release (IR)

ACTIVE COMPARATOR

Participants will receive Bezafibrate (BZF) 200 mg IR + OCA Placebo + BZF 400 mg Placebo

Drug: Bezafibrate 200 MG; Drug: OCA Placebo; Drug: Bezafibrate 400 mg Placebo

Treatment B: BZF 400 mg SR

ACTIVE COMPARATOR

Participants will receive BZF 400 mg SR + OCA Placebo + BZF 200 mg Placebo

Drug: OCA Placebo; Drug: Bezafibrate 200 mg Placebo; Drug: Bezafibrate 400 MG

Treatment C: OCA 5 mg to 10 mg + BZF 200 mg IR

EXPERIMENTAL

Participants will receive OCA 5 mg to 10 mg + BZF 200 mg IR + BZF 400 mg Placebo

Drug: Obeticholic acid; Drug: Bezafibrate 200 MG; Drug: Bezafibrate 400 mg Placebo

Treatment D: OCA 5 mg to 10 mg + BZF 400 mg SR

EXPERIMENTAL

Participants will receive OCA 5 mg to 10 mg + BZF 400 mg SR + BZF 200 mg Placebo

Drug: Obeticholic acid; Drug: Bezafibrate 200 mg Placebo; Drug: Bezafibrate 400 MG

Long-term safety extension (LTSE) phase: OCA + BZF

EXPERIMENTAL

Participants will continue the original treatment assignment allocated during the DB Period. The OCA and BZF dose may be optimized based on safety and efficacy during the DB period.

Drug: OCA; Drug: Bezafibrate

Interventions

Obeticholic acid DRUG

5 mg tablet of OCA once daily titrating up to a maximum of 10 mg OCA once daily

Treatment C: OCA 5 mg to 10 mg + BZF 200 mg IR; Treatment D: OCA 5 mg to 10 mg + BZF 400 mg SR

Bezafibrate 200 MG DRUG

200 mg IR tablet of Bezafibrate once daily for the remainder of the study

Treatment A: BZF 200 milligrams (mg) Immediate release (IR); Treatment C: OCA 5 mg to 10 mg + BZF 200 mg IR

OCA Placebo DRUG

One tablet daily for the remainder of the study

Treatment A: BZF 200 milligrams (mg) Immediate release (IR); Treatment B: BZF 400 mg SR

Bezafibrate 200 mg Placebo DRUG

One tablet daily for the remainder of the study

Treatment B: BZF 400 mg SR; Treatment D: OCA 5 mg to 10 mg + BZF 400 mg SR

Bezafibrate 400 MG DRUG

400 mg SR tablet of Bezafibrate once daily for the remainder of the study

Treatment B: BZF 400 mg SR; Treatment D: OCA 5 mg to 10 mg + BZF 400 mg SR

Bezafibrate 400 mg Placebo DRUG

One tablet daily for the remainder of the study

Treatment A: BZF 200 milligrams (mg) Immediate release (IR); Treatment C: OCA 5 mg to 10 mg + BZF 200 mg IR

OCA DRUG

OCA one tablet will be administered.

Long-term safety extension (LTSE) phase: OCA + BZF

Bezafibrate DRUG

Bezafibrate one tablet will be administered.

Long-term safety extension (LTSE) phase: OCA + BZF

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
• Clinical complications of PBC
• History or presence of hepatic 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 other farnesoid X receptor (FXR) agonists, or participation in a previous study involving OCA within 3 months before Screening.

Sponsors & Collaborators

• Intercept Pharmaceuticals: lead

Study Sites (33)

Flinders Medical Centre

Bedford Park, Perth, 5042, Australia

Location

Royal Adelaide Hospital

Adelaide, 5000, Australia

Location

UZ Gasthuisberg

Leuven, 3000, Belgium

Location

Clinical Hospital Dubrava

Zagreb, 10000, Croatia

Location

Zagreb University Hospital Center

Zagreb, 10000, Croatia

Location

Hepato-Gastroenterologie HK, s.r.o.

Hradec Králové, 500 12, Czechia

Location

Artroscan s.r.o., Gastroenterologicka ambulance

Ostrava, 722 00, Czechia

Location

Research Site s.r.o.

Pilsen, 301 00, Czechia

Location

Tartu University Hospital

Tartu, 51014, Estonia

Location

Hôpital Henri Mondor

Créteil, 940000, France

Location

Centre Hospitalier Universitaire Grenoble

Grenoble, 38043, France

Location

CHRU de Lille

Lille, 59000, France

Location

Groupe Hospitalier Pitié Salpêtrière - Assistance publique - Hôpitaux de Paris

Paris, 75651, France

Location

CHU Paris Est - Hopital Saint Antoine

Paris, Paris 12, France

Location

Universitatsklinikum Hamburg-Eppendorf UKE

Hamburg, 20246, Germany

Location

Medizinische Hochschule Hannover

Hanover, 30625, Germany

Location

Department of Medicine and Research Laboratory of Internal Medicine, University Hospital of Larissa

Larissa, 41110, Greece

Location

Budai Hepatologiai Centrum (BHC)

Budapest, 1111, Hungary

Location

DEOEC II. sz. Belgyógyászati Klinika

Debrecen, 4032, Hungary

Location

Hadassah Ein-Karem Medical Center - Liver unit

Jerusalem, 91120, Israel

Location

Tel Aviv Surasky Medical Center

Tel Aviv, 6423906, Israel

Location

Hospital of Lithuanian University of Health Sciences, Kauno Klinikos

Kaunas, 50161, Lithuania

Location

Vlinius University

Vilnius, 08661, Lithuania

Location

Academisch Medisch Centrum

Amsterdam, 1105 AZ, Netherlands

Location

Universitetet i Oslo - Akershus Universitetssykehus (AHUS)

Loerenskog, 1478, Norway

Location

Narodowy Instytut Onkologii, Klinika Gastroenterologii Onkologicznej

Warsaw, 02-781, Poland

Location

Pusan National University Hospital

Busan, 602-739, South Korea

Location

Kyungpook National University Hospital

Daegu, 41944, South Korea

Location

Fundacio Clinic Per La Recerca Biomedica

Barcelona, 08036, Spain

Location

Consorcio Hospital General Universitario

Valencia, 46010, Spain

Location

Hull University Teaching Hospitals NHS Trust

Hull, HU3 2JZ, United Kingdom

Location

Institute of Cellular Medicine, Newcastle University

Newcastle upon Tyne, NE2 4HH, United Kingdom

Location

John Radcliffe Hospital

Oxford, OX3 9DU, United Kingdom

Location

Related Publications (2)

• Lindor KD, Gershwin ME, Poupon R, Kaplan M, Bergasa NV, Heathcote EJ; American Association for Study of Liver Diseases. Primary biliary cirrhosis. Hepatology. 2009 Jul;50(1):291-308. doi: 10.1002/hep.22906. No abstract available.

  PMID: 19554543 BACKGROUND

• European Association for the Study of the Liver. EASL Clinical Practice Guidelines: management of cholestatic liver diseases. J Hepatol. 2009 Aug;51(2):237-67. doi: 10.1016/j.jhep.2009.04.009. Epub 2009 Jun 6. No abstract available.

  PMID: 19501929 BACKGROUND

MeSH Terms

Conditions

Liver Cirrhosis, Biliary; Fibrosis

Interventions

obeticholic acid; Bezafibrate

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

  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: July 14, 2020
• First Posted: October 20, 2020
• Study Start: October 2, 2019
• Primary Completion: October 14, 2025
• Study Completion: October 14, 2025
• Last Updated: November 17, 2025

Record last verified: 2025-11

Locations

FR (5); DE (2); ES (1); CZ (3); HU (2); GR (1); IL (2); BE (1); LI (2); AU (2); NL (1); NO (1); GB (3); CR (2); KR (2); PL (1)