Clinical trial NCT04594694: 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
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Primary Biliary Cholangitis
Keywords: Primary Biliary Cholangitis
Primary Biliary Cirrhosis
PBC
Hepatic Impairment
Cirrhosis
Liver
Lead sponsor: Intercept Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2020-10-20
Enrollment: 72
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).


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.

Primary outcomes:
measure: Change in Alkaline Phosphatase (ALP) from baseline to Week 12 in the DB Treatment Period
timeFrame: Baseline, Day 1, and Weeks 4, 8, and 12
Secondary outcomes:
measure: Response rates of ≥10%, ≥20%, ≥30% and ≥40% reduction, and normalization of biochemical disease marker Alkaline Phosphatase (ALP)
timeFrame: Baseline, Day 1, and Weeks 2, 4, 6, 8, and 12
measure: 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
timeFrame: Baseline, Day 1, and Weeks 2, 4, 6, 8, and 12
measure: Change in GGT from baseline to Week 12
timeFrame: Baseline, Day 1, and Weeks 4, 8, and 12
measure: Change in ALT from baseline to Week 12
timeFrame: Baseline, Day 1, and Weeks 4, 8, and 12
measure: Change in AST from baseline to Week 12
timeFrame: Baseline, Day 1, and Weeks 4, 8, and 12
measure: Change in total and conjugated bilirubin from baseline to Week 12
timeFrame: Baseline, Day 1, and Weeks 4, 8, and 12
measure: Change in lipid panel from baseline to Week 12
timeFrame: Baseline, Day 1, and Weeks 4, 8, and 12
measure: Change in 7 alpha (α) hydroxy 4 cholesten-3 one (C4) from baseline to Week 12
timeFrame: Baseline, Day 1, and Weeks 4, 8, and 12
measure: Change in bile acid from baseline to Week 12
timeFrame: Baseline, Day 1, and Weeks 4,8, and 12
Other outcomes:
Locations:
city: Bedford Park
country: Australia
facility: Flinders Medical Centre
state: Perth
city: Adelaide
country: Australia
facility: Royal Adelaide Hospital
city: Leuven
country: Belgium
facility: UZ Gasthuisberg
city: Zagreb
country: Croatia
facility: Clinical Hospital Dubrava
city: Zagreb
country: Croatia
facility: Zagreb University Hospital Center
city: Hradec Kralove
country: Czechia
facility: Hepato-Gastroenterologie HK, s.r.o.
city: Ostrava
country: Czechia
facility: Artroscan s.r.o., Gastroenterologicka ambulance
city: Plzen
country: Czechia
facility: Research Site s.r.o.
city: Tartu
country: Estonia
facility: Tartu University Hospital
city: Créteil
country: France
facility: Hôpital Henri Mondor
city: Grenoble
country: France
facility: Centre Hospitalier Universitaire Grenoble
city: Lille
country: France
facility: CHRU de Lille
city: Paris
country: France
facility: Groupe Hospitalier Pitié Salpêtrière - Assistance publique - Hôpitaux de Paris
city: Paris
country: France
facility: CHU Paris Est - Hopital Saint Antoine
city: Hamburg
country: Germany
facility: Universitatsklinikum Hamburg-Eppendorf UKE
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Larissa
country: Greece
facility: Department of Medicine and Research Laboratory of Internal Medicine, University Hospital of Larissa
city: Budapest
country: Hungary
facility: Budai Hepatologiai Centrum (BHC)
city: Debrecen
country: Hungary
facility: DEOEC II. sz. Belgyógyászati Klinika
city: Jerusalem
country: Israel
facility: Hadassah Ein-Karem Medical Center - Liver unit
city: Tel Aviv
country: Israel
facility: Tel Aviv Surasky Medical Center
city: Busan
country: Korea, Republic of
facility: Pusan National University Hospital
city: Daegu
country: Korea, Republic of
facility: Kyungpook National University Hospital
city: Kaunas
country: Lithuania
facility: Hospital of Lithuanian University of Health Sciences, Kauno Klinikos
city: Vilnius
country: Lithuania
facility: Vlinius University
city: Amsterdam
country: Netherlands
facility: Academisch Medisch Centrum
city: Loerenskog
country: Norway
facility: Universitetet i Oslo - Akershus Universitetssykehus (AHUS)
city: Warszawa
country: Poland
facility: Narodowy Instytut Onkologii, Klinika Gastroenterologii Onkologicznej
city: Barcelona
country: Spain
facility: Fundacio Clinic Per La Recerca Biomedica
city: Valencia
country: Spain
facility: Consorcio Hospital General Universitario
city: Hull
country: United Kingdom
facility: Hull University Teaching Hospitals NHS Trust
city: Newcastle Upon Tyne
country: United Kingdom
facility: Institute of Cellular Medicine, Newcastle University
city: Oxford
country: United Kingdom
facility: John Radcliffe Hospital
Study officials:
affiliation: Intercept Pharmaceuticals, Inc.
name: Antonio Civitarese, M.D.
role: STUDY_DIRECTOR
References:
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.
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.