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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04594694




Registration number
NCT04594694
Ethics application status
Date submitted
14/07/2020
Date registered
20/10/2020
Date last updated
18/04/2024

Titles & IDs
Public title
Study of OCA in Combination With BZF Evaluating Efficacy, Safety, and Tolerability in Participants With PBC
Scientific 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
Secondary ID [1] 0 0
747-213
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary Biliary Cholangitis 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Infection 0 0 0 0
Other infectious diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Obeticholic acid
Treatment: Drugs - Bezafibrate 200 MG
Treatment: Drugs - OCA Placebo
Treatment: Drugs - Bezafibrate 200 mg Placebo
Treatment: Drugs - Bezafibrate 400 MG
Treatment: Drugs - Bezafibrate 400 mg Placebo
Treatment: Drugs - OCA
Treatment: Drugs - Bezafibrate

Active Comparator: Treatment A: BZF 200 milligrams (mg) Immediate release (IR) - Participants will receive Bezafibrate (BZF) 200 mg IR + OCA Placebo + BZF 400 mg Placebo

Active Comparator: Treatment B: BZF 400 mg SR - Participants will receive BZF 400 mg SR + OCA Placebo + BZF 200 mg Placebo

Experimental: Treatment C: OCA 5 mg to 10 mg + BZF 200 mg IR - Participants will receive OCA 5 mg to 10 mg + BZF 200 mg IR + BZF 400 mg Placebo

Experimental: Treatment D: OCA 5 mg to 10 mg + BZF 400 mg SR - Participants will receive OCA 5 mg to 10 mg + BZF 400 mg SR + BZF 200 mg Placebo

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


Treatment: Drugs: Obeticholic acid
5 mg tablet of OCA once daily titrating up to a maximum of 10 mg OCA once daily

Treatment: Drugs: Bezafibrate 200 MG
200 mg IR tablet of Bezafibrate once daily for the remainder of the study

Treatment: Drugs: OCA Placebo
One tablet daily for the remainder of the study

Treatment: Drugs: Bezafibrate 200 mg Placebo
One tablet daily for the remainder of the study

Treatment: Drugs: Bezafibrate 400 MG
400 mg SR tablet of Bezafibrate once daily for the remainder of the study

Treatment: Drugs: Bezafibrate 400 mg Placebo
One tablet daily for the remainder of the study

Treatment: Drugs: OCA
OCA one tablet will be administered.

Treatment: Drugs: Bezafibrate
Bezafibrate one tablet will be administered.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in Alkaline Phosphatase (ALP) from baseline to Week 12 in the DB Treatment Period
Timepoint [1] 0 0
Baseline, Day 1, and Weeks 4, 8, and 12
Secondary outcome [1] 0 0
Response rates of =10%, =20%, =30% and =40% reduction, and normalization of biochemical disease marker Alkaline Phosphatase (ALP)
Timepoint [1] 0 0
Baseline, Day 1, and Weeks 2, 4, 6, 8, and 12
Secondary outcome [2] 0 0
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
Timepoint [2] 0 0
Baseline, Day 1, and Weeks 2, 4, 6, 8, and 12
Secondary outcome [3] 0 0
Change in GGT from baseline to Week 12
Timepoint [3] 0 0
Baseline, Day 1, and Weeks 4, 8, and 12
Secondary outcome [4] 0 0
Change in ALT from baseline to Week 12
Timepoint [4] 0 0
Baseline, Day 1, and Weeks 4, 8, and 12
Secondary outcome [5] 0 0
Change in AST from baseline to Week 12
Timepoint [5] 0 0
Baseline, Day 1, and Weeks 4, 8, and 12
Secondary outcome [6] 0 0
Change in total and conjugated bilirubin from baseline to Week 12
Timepoint [6] 0 0
Baseline, Day 1, and Weeks 4, 8, and 12
Secondary outcome [7] 0 0
Change in lipid panel from baseline to Week 12
Timepoint [7] 0 0
Baseline, Day 1, and Weeks 4, 8, and 12
Secondary outcome [8] 0 0
Change in 7 alpha (a) hydroxy 4 cholesten-3 one (C4) from baseline to Week 12
Timepoint [8] 0 0
Baseline, Day 1, and Weeks 4, 8, and 12
Secondary outcome [9] 0 0
Change in bile acid from baseline to Week 12
Timepoint [9] 0 0
Baseline, Day 1, and Weeks 4,8, and 12

Eligibility
Key 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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
2/10/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2025
Actual
Sample size
Target
72
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Pert
Recruitment hospital [1] 0 0
Flinders Medical Centre - Bedford Park
Recruitment hospital [2] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment postcode(s) [1] 0 0
5042 - Bedford Park
Recruitment postcode(s) [2] 0 0
5000 - Adelaide
Recruitment outside Australia
Country [1] 0 0
Belgium
State/province [1] 0 0
Leuven
Country [2] 0 0
Croatia
State/province [2] 0 0
Zagreb
Country [3] 0 0
Czechia
State/province [3] 0 0
Hradec Kralove
Country [4] 0 0
Czechia
State/province [4] 0 0
Ostrava
Country [5] 0 0
Czechia
State/province [5] 0 0
Plzen
Country [6] 0 0
Estonia
State/province [6] 0 0
Tartu
Country [7] 0 0
France
State/province [7] 0 0
Créteil
Country [8] 0 0
France
State/province [8] 0 0
Grenoble
Country [9] 0 0
France
State/province [9] 0 0
Lille
Country [10] 0 0
France
State/province [10] 0 0
Paris
Country [11] 0 0
Germany
State/province [11] 0 0
Hamburg
Country [12] 0 0
Germany
State/province [12] 0 0
Hannover
Country [13] 0 0
Greece
State/province [13] 0 0
Larissa
Country [14] 0 0
Hungary
State/province [14] 0 0
Budapest
Country [15] 0 0
Hungary
State/province [15] 0 0
Debrecen
Country [16] 0 0
Israel
State/province [16] 0 0
Jerusalem
Country [17] 0 0
Israel
State/province [17] 0 0
Tel Aviv
Country [18] 0 0
Korea, Republic of
State/province [18] 0 0
Busan
Country [19] 0 0
Korea, Republic of
State/province [19] 0 0
Daegu
Country [20] 0 0
Lithuania
State/province [20] 0 0
Kaunas
Country [21] 0 0
Lithuania
State/province [21] 0 0
Vilnius
Country [22] 0 0
Netherlands
State/province [22] 0 0
Amsterdam
Country [23] 0 0
Norway
State/province [23] 0 0
Loerenskog
Country [24] 0 0
Poland
State/province [24] 0 0
Warszawa
Country [25] 0 0
Spain
State/province [25] 0 0
Barcelona
Country [26] 0 0
Spain
State/province [26] 0 0
Valencia
Country [27] 0 0
United Kingdom
State/province [27] 0 0
Hull
Country [28] 0 0
United Kingdom
State/province [28] 0 0
Newcastle Upon Tyne
Country [29] 0 0
United Kingdom
State/province [29] 0 0
Oxford

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Intercept Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
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).
Trial website
https://clinicaltrials.gov/ct2/show/NCT04594694
Trial related presentations / publications
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.
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.
Public notes

Contacts
Principal investigator
Name 0 0
Antonio Civitarese, M.D.
Address 0 0
Intercept Pharmaceuticals, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04594694