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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
International
Study Type
Interventional
Intervention
Obeticholic acid
Bezafibrate 200 MG
OCA Placebo
Bezafibrate 200 mg Placebo
Bezafibrate 400 MG
Bezafibrate 400 mg Placebo
OCA
Bezafibrate
Sponsored by
Intercept Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Biliary Cholangitis focused on measuring Primary Biliary Cholangitis, Primary Biliary Cirrhosis, PBC, 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 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
  • 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 participation in a previous study involving OCA

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Sites / Locations

  • Flinders Medical Centre
  • Royal Adelaide Hospital
  • Medizinische Universität Wien
  • Hopital Erasme
  • UZ Gasthuisberg
  • Clinical Hospital Dubrava
  • Zagreb University Hospital Center
  • Hepato-Gastroenterologie HK, s.r.o.
  • Artroscan s.r.o., Gastroenterologicka ambulance
  • Research Site s.r.o.
  • Tartu University Hospital
  • Hôpital Henri Mondor
  • Centre Hospitalier Universitaire Grenoble
  • CHRU de Lille
  • Hôpital de la Croix-Rousse - Hospices Civils de Lyon
  • Hôpital Saint Eloi - CHU Montpellier
  • Groupe Hospitalier Pitié Salpêtrière - Assistance publique - Hôpitaux de Paris
  • CHU Paris Est - Hopital Saint Antoine
  • Hôpital Haut Leveque CHU de Bordeaux
  • Hospitalier Universitaire de Poitiers
  • CHU Rennes - Hopital Pontchaillou
  • Universitatsklinikum Bonn
  • Universtitaetsklinikum Essen
  • Universitatsklinikum Hamburg-Eppendorf UKE
  • Medizinische Hochschule Hannover
  • Liver Study centre Kiel
  • University of Athens - Hippokration General Hospital
  • Department of Medicine and Research Laboratory of Internal Medicine, University Hospital of Larissa
  • Budai Hepatologiai Centrum (BHC)
  • Bekes Megyei Kozponti Korhaz Dr. Rethy Pal Tagkorhaz
  • DEOEC II. sz. Belgyógyászati Klinika
  • Hadassah Ein-Karem Medical Center - Liver unit
  • Tel Aviv Surasky Medical Center
  • The Catholic University of Korea, Daejeon St. Mary's Hospital
  • The Catholic University of Korea, Bucheon St. Mary's Hospital
  • Seoul National University Bundang Hospital
  • Pusan National University Hospital
  • Kyungpook National University Hospital
  • Seoul National University Hospital
  • Vsl Klaipedos jurininku ligonine
  • Vlinius University
  • Academisch Medisch Centrum
  • UMC Utrecht Locatie AZU
  • Universitetet i Oslo - Akershus Universitetssykehus (AHUS)
  • Narodowy Instytut Onkologii, Klinika Gastroenterologii Onkologicznej
  • Narodowy Instytut Onkologii, Klinika Gastroenterologii Onkologicznej
  • Fundacio Clinic Per La Recerca Biomedica
  • Hospital General Universitario Gregorio Marañón
  • Consorcio Hospital General Universitario
  • Hospital Universitario Rio Hortega
  • Hull University Teaching Hospitals NHS Trust
  • Institute of Cellular Medicine, Newcastle University
  • John Radcliffe Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm Type

Active Comparator

Active Comparator

Experimental

Experimental

Experimental

Arm Label

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

Treatment B: BZF 400 mg SR

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

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

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

Arm Description

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

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

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

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

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.

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Response rates of ≥10%, ≥20%, ≥30% and ≥40% reduction, and normalization of biochemical disease marker Alkaline Phosphatase (ALP)
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
Change in GGT from baseline to Week 12
Change in ALT from baseline to Week 12
Change in AST from baseline to Week 12
Change in total and conjugated bilirubin from baseline to Week 12
Change in lipid panel from baseline to Week 12
Change in 7 alpha (α) hydroxy 4 cholesten-3 one (C4) from baseline to Week 12
Change in bile acid from baseline to Week 12

Full Information

First Posted
July 14, 2020
Last Updated
August 4, 2023
Sponsor
Intercept Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT04594694
Brief Title
Study of OCA in Combination With BZF Evaluating Efficacy, Safety, and Tolerability in Participants With PBC
Official Title
A Phase 2, Double-Blind, Randomized, Parallel Group Study Evaluating the Efficacy, Safety, and Tolerability of Obeticholic Acid Administered in Combination With Bezafibrate in Subjects With Primary Biliary Cholangitis Who Had an Inadequate Response or Who Were Unable to Tolerate Ursodeoxycholic Acid
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
October 2, 2019 (Actual)
Primary Completion Date
October 2024 (Anticipated)
Study Completion Date
December 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
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes

5. Study Description

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).

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, PBC, 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
72 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Treatment A: BZF 200 milligrams (mg) Immediate release (IR)
Arm Type
Active Comparator
Arm Description
Participants will receive Bezafibrate (BZF) 200 mg IR + OCA Placebo + BZF 400 mg Placebo
Arm Title
Treatment B: BZF 400 mg SR
Arm Type
Active Comparator
Arm Description
Participants will receive BZF 400 mg SR + OCA Placebo + BZF 200 mg Placebo
Arm Title
Treatment C: OCA 5 mg to 10 mg + BZF 200 mg IR
Arm Type
Experimental
Arm Description
Participants will receive OCA 5 mg to 10 mg + BZF 200 mg IR + BZF 400 mg Placebo
Arm Title
Treatment D: OCA 5 mg to 10 mg + BZF 400 mg SR
Arm Type
Experimental
Arm Description
Participants will receive OCA 5 mg to 10 mg + BZF 400 mg SR + BZF 200 mg Placebo
Arm Title
Long-term safety extension (LTSE) phase: OCA + BZF
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Obeticholic acid
Intervention Description
5 mg tablet of OCA once daily titrating up to a maximum of 10 mg OCA once daily
Intervention Type
Drug
Intervention Name(s)
Bezafibrate 200 MG
Intervention Description
200 mg IR tablet of Bezafibrate once daily for the remainder of the study
Intervention Type
Drug
Intervention Name(s)
OCA Placebo
Intervention Description
One tablet daily for the remainder of the study
Intervention Type
Drug
Intervention Name(s)
Bezafibrate 200 mg Placebo
Intervention Description
One tablet daily for the remainder of the study
Intervention Type
Drug
Intervention Name(s)
Bezafibrate 400 MG
Intervention Description
400 mg SR tablet of Bezafibrate once daily for the remainder of the study
Intervention Type
Drug
Intervention Name(s)
Bezafibrate 400 mg Placebo
Intervention Description
One tablet daily for the remainder of the study
Intervention Type
Drug
Intervention Name(s)
OCA
Intervention Description
OCA one tablet will be administered.
Intervention Type
Drug
Intervention Name(s)
Bezafibrate
Intervention Description
Bezafibrate one tablet will be administered.
Primary Outcome Measure Information:
Title
Change in Alkaline Phosphatase (ALP) from baseline to Week 12 in the DB Treatment Period
Time Frame
Baseline, Day 1, and Weeks 4, 8, and 12
Secondary Outcome Measure Information:
Title
Response rates of ≥10%, ≥20%, ≥30% and ≥40% reduction, and normalization of biochemical disease marker Alkaline Phosphatase (ALP)
Time Frame
Baseline, Day 1, and Weeks 2, 4, 6, 8, and 12
Title
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
Time Frame
Baseline, Day 1, and Weeks 2, 4, 6, 8, and 12
Title
Change in GGT from baseline to Week 12
Time Frame
Baseline, Day 1, and Weeks 4, 8, and 12
Title
Change in ALT from baseline to Week 12
Time Frame
Baseline, Day 1, and Weeks 4, 8, and 12
Title
Change in AST from baseline to Week 12
Time Frame
Baseline, Day 1, and Weeks 4, 8, and 12
Title
Change in total and conjugated bilirubin from baseline to Week 12
Time Frame
Baseline, Day 1, and Weeks 4, 8, and 12
Title
Change in lipid panel from baseline to Week 12
Time Frame
Baseline, Day 1, and Weeks 4, 8, and 12
Title
Change in 7 alpha (α) hydroxy 4 cholesten-3 one (C4) from baseline to Week 12
Time Frame
Baseline, Day 1, and Weeks 4, 8, and 12
Title
Change in bile acid from baseline to Week 12
Time Frame
Baseline, Day 1, and Weeks 4,8, and 12

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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 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. Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Antonio Civitarese, M.D.
Organizational Affiliation
Intercept Pharmaceuticals, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Flinders Medical Centre
City
Bedford Park
State/Province
Perth
ZIP/Postal Code
5042
Country
Australia
Facility Name
Royal Adelaide Hospital
City
Adelaide
ZIP/Postal Code
5000
Country
Australia
Facility Name
Medizinische Universität Wien
City
Vienna
ZIP/Postal Code
1090
Country
Austria
Facility Name
Hopital Erasme
City
Bruxelles
ZIP/Postal Code
1070
Country
Belgium
Facility Name
UZ Gasthuisberg
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
Clinical Hospital Dubrava
City
Zagreb
ZIP/Postal Code
10000
Country
Croatia
Facility Name
Zagreb University Hospital Center
City
Zagreb
ZIP/Postal Code
10000
Country
Croatia
Facility Name
Hepato-Gastroenterologie HK, s.r.o.
City
Hradec Kralove
ZIP/Postal Code
500 12
Country
Czechia
Facility Name
Artroscan s.r.o., Gastroenterologicka ambulance
City
Ostrava
ZIP/Postal Code
722 00
Country
Czechia
Facility Name
Research Site s.r.o.
City
Plzen
ZIP/Postal Code
301 00
Country
Czechia
Facility Name
Tartu University Hospital
City
Tartu
ZIP/Postal Code
51014
Country
Estonia
Facility Name
Hôpital Henri Mondor
City
Créteil
ZIP/Postal Code
940000
Country
France
Facility Name
Centre Hospitalier Universitaire Grenoble
City
Grenoble
ZIP/Postal Code
38043
Country
France
Facility Name
CHRU de Lille
City
Lille
ZIP/Postal Code
59000
Country
France
Facility Name
Hôpital de la Croix-Rousse - Hospices Civils de Lyon
City
Lyon
ZIP/Postal Code
69317
Country
France
Facility Name
Hôpital Saint Eloi - CHU Montpellier
City
Montpellier
ZIP/Postal Code
34295
Country
France
Facility Name
Groupe Hospitalier Pitié Salpêtrière - Assistance publique - Hôpitaux de Paris
City
Paris
ZIP/Postal Code
75651
Country
France
Facility Name
CHU Paris Est - Hopital Saint Antoine
City
Paris
ZIP/Postal Code
Paris 12
Country
France
Facility Name
Hôpital Haut Leveque CHU de Bordeaux
City
Pessac
ZIP/Postal Code
33600
Country
France
Facility Name
Hospitalier Universitaire de Poitiers
City
Poitiers
ZIP/Postal Code
86021
Country
France
Facility Name
CHU Rennes - Hopital Pontchaillou
City
Rennes
ZIP/Postal Code
35033
Country
France
Facility Name
Universitatsklinikum Bonn
City
Bonn
ZIP/Postal Code
53127
Country
Germany
Facility Name
Universtitaetsklinikum Essen
City
Essen
ZIP/Postal Code
Essen
Country
Germany
Facility Name
Universitatsklinikum Hamburg-Eppendorf UKE
City
Hamburg
ZIP/Postal Code
20246
Country
Germany
Facility Name
Medizinische Hochschule Hannover
City
Hannover
ZIP/Postal Code
30625
Country
Germany
Facility Name
Liver Study centre Kiel
City
Kiel
ZIP/Postal Code
24146
Country
Germany
Facility Name
University of Athens - Hippokration General Hospital
City
Athens
ZIP/Postal Code
18794
Country
Greece
Facility Name
Department of Medicine and Research Laboratory of Internal Medicine, University Hospital of Larissa
City
Larissa
ZIP/Postal Code
41110
Country
Greece
Facility Name
Budai Hepatologiai Centrum (BHC)
City
Budapest
ZIP/Postal Code
1111
Country
Hungary
Facility Name
Bekes Megyei Kozponti Korhaz Dr. Rethy Pal Tagkorhaz
City
Békéscsaba
ZIP/Postal Code
5600
Country
Hungary
Facility Name
DEOEC II. sz. Belgyógyászati Klinika
City
Debrecen
ZIP/Postal Code
4032
Country
Hungary
Facility Name
Hadassah Ein-Karem Medical Center - Liver unit
City
Jerusalem
ZIP/Postal Code
91120
Country
Israel
Facility Name
Tel Aviv Surasky Medical Center
City
Tel Aviv
ZIP/Postal Code
6423906
Country
Israel
Facility Name
The Catholic University of Korea, Daejeon St. Mary's Hospital
City
Junggu
State/Province
Daejeon
ZIP/Postal Code
34943
Country
Korea, Republic of
Facility Name
The Catholic University of Korea, Bucheon St. Mary's Hospital
City
Bucheon-si
State/Province
Gyeonggi-do
ZIP/Postal Code
14647
Country
Korea, Republic of
Facility Name
Seoul National University Bundang Hospital
City
Seongnam-si
State/Province
Gyeonggi-do
ZIP/Postal Code
13620
Country
Korea, Republic of
Facility Name
Pusan National University Hospital
City
Busan
ZIP/Postal Code
602-739
Country
Korea, Republic of
Facility Name
Kyungpook National University Hospital
City
Daegu
ZIP/Postal Code
41944
Country
Korea, Republic of
Facility Name
Seoul National University Hospital
City
Seoul
ZIP/Postal Code
3080
Country
Korea, Republic of
Facility Name
Vsl Klaipedos jurininku ligonine
City
Klaipėda
ZIP/Postal Code
92288
Country
Lithuania
Facility Name
Vlinius University
City
Vilnius
ZIP/Postal Code
08661
Country
Lithuania
Facility Name
Academisch Medisch Centrum
City
Amsterdam
ZIP/Postal Code
1105 AZ
Country
Netherlands
Facility Name
UMC Utrecht Locatie AZU
City
Utrecht
ZIP/Postal Code
3584 CX
Country
Netherlands
Facility Name
Universitetet i Oslo - Akershus Universitetssykehus (AHUS)
City
Loerenskog
ZIP/Postal Code
1478
Country
Norway
Facility Name
Narodowy Instytut Onkologii, Klinika Gastroenterologii Onkologicznej
City
Katowice
ZIP/Postal Code
40-752
Country
Poland
Facility Name
Narodowy Instytut Onkologii, Klinika Gastroenterologii Onkologicznej
City
Warszawa
ZIP/Postal Code
02-781
Country
Poland
Facility Name
Fundacio Clinic Per La Recerca Biomedica
City
Barcelona
ZIP/Postal Code
08036
Country
Spain
Facility Name
Hospital General Universitario Gregorio Marañón
City
Madrid
ZIP/Postal Code
28007
Country
Spain
Facility Name
Consorcio Hospital General Universitario
City
Valencia
ZIP/Postal Code
46010
Country
Spain
Facility Name
Hospital Universitario Rio Hortega
City
Valladolid
ZIP/Postal Code
47012
Country
Spain
Facility Name
Hull University Teaching Hospitals NHS Trust
City
Hull
ZIP/Postal Code
HU3 2JZ
Country
United Kingdom
Facility Name
Institute of Cellular Medicine, Newcastle University
City
Newcastle Upon Tyne
ZIP/Postal Code
NE2 4HH
Country
United Kingdom
Facility Name
John Radcliffe Hospital
City
Oxford
ZIP/Postal Code
OX3 9DU
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
19554543
Citation
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.
Results Reference
background
PubMed Identifier
19501929
Citation
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.
Results Reference
background

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

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