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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02308111




Registration number
NCT02308111
Ethics application status
Date submitted
10/11/2014
Date registered
4/12/2014
Date last updated
20/05/2021

Titles & IDs
Public title
Phase 4 Study of Obeticholic Acid Evaluating Clinical Outcomes in Patients With Primary Biliary Cholangitis
Scientific title
A Phase 4, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Effect of Obeticholic Acid on Clinical Outcomes in Patients With Primary Biliary Cholangitis
Secondary ID [1] 0 0
747-302
Universal Trial Number (UTN)
Trial acronym
COBALT
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Liver Cirrhosis, Biliary 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
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Obeticholic Acid (OCA)
Treatment: Drugs - Placebo

Experimental: Obeticholic Acid (OCA) 5 mg to 10 mg - Obeticholic Acid (OCA) 5 mg for a minimum 3 months and then titrating up to a maximum 10 mg for the remainder of the trial (based on tolerability and Child Pugh Score).

Placebo Comparator: Placebo -


Treatment: Drugs: Obeticholic Acid (OCA)
Non-cirrhotic and classified as Child-Pugh Class A: 5 mg tablet of OCA once daily titrating up to a maximum of 10 mg OCA once daily based on tolerability at 3 months for the duration of the study (majority of patients).
Cirrhotic and classified as Child-Pugh Class B and C: 5 mg tablet of OCA once weekly for at least 3 months, subsequently titrating up to a maximum dose and frequency of 10 mg OCA twice weekly based on tolerability and biochemical response for the duration of the study.

Treatment: Drugs: Placebo
One tablet daily (or a lower frequency depending on Child Pugh score) for the remainder of the study

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Composite endpoint of any of the five listed adjudicated events - Primary endpoint events include:
death
liver transplant
MELD score =15
uncontrolled ascites
hospitalization for new onset or recurrence of any of the following:
variceal bleed
hepatic encephalopathy
spontaneous bacterial peritonitis
Timepoint [1] 0 0
Time to accrue approximately 127 primary endpoint events, estimated to be approximately 10 years
Secondary outcome [1] 0 0
First occurrence of each of the listed individual events - Individual events include:
death
liver transplant
MELD score >15
uncontrolled ascites
hospitalization for new onset or recurrence of any of the following:
variceal bleed
hepatic encephalopathy
spontaneous bacterial peritonitis
Timepoint [1] 0 0
Time to first occurrence from date of randomization until the date of first documented progression or date of death from any cause, whichever came first, up to 10 years
Secondary outcome [2] 0 0
First occurrence of liver-related death - To assess the effect of OCA compared to placebo on time to occurrence of liver-related death
Timepoint [2] 0 0
Time to first occurrence from date of randomization until the date of first documented progression or date of death from any cause, whichever came first, up to 10 years
Secondary outcome [3] 0 0
Progression to cirrhosis - To assess the effect of OCA compared to placebo on progression to cirrhosis
Timepoint [3] 0 0
Time to first occurrence from date of randomization until the date of first documented progression or date of death from any cause, whichever came first, up to 10 years
Secondary outcome [4] 0 0
Time to occurrence of hepatocellular carcinoma (HCC) - To assess the effect of OCA compared to placebo on time to occurrence of hepatocellular carcinoma (HCC)
Timepoint [4] 0 0
Time to first occurrence from date of randomization until the date of first documented progression or date of death from any cause, whichever came first, up to 10 years
Secondary outcome [5] 0 0
Changes from Baseline in bilirubin as a marker of liver biochemistry - bilirubin (total and conjugated)
Timepoint [5] 0 0
Samples will be measured at Baseline and Months 3, 6, 9 and 12 for 10 Years
Secondary outcome [6] 0 0
Changes from Baseline in aspartate aminotransferase (AST) as a marker of liver biochemistry - aspartate aminotransferase (AST)
Timepoint [6] 0 0
Samples will be measured at Baseline and Months 3, 6, 9 and 12 for 10 Years
Secondary outcome [7] 0 0
Changes from Baseline in alanine aminotransferase (ALT) as a marker of liver biochemistry - alanine aminotransferase (ALT)
Timepoint [7] 0 0
Samples will be measured at Baseline and Months 3, 6, 9 and 12 for 10 Years
Secondary outcome [8] 0 0
Changes from Baseline in alkaline phosphatase (ALP) as a marker of liver biochemistry - alkaline phosphatase (ALP)
Timepoint [8] 0 0
Samples will be measured at Baseline and Months 3, 6, 9 and 12 for 10 Years
Secondary outcome [9] 0 0
Changes from Baseline in gamma-glutamyl transferase (GGT) as a marker of liver biochemistry - gamma-glutamyl transferase (GGT)
Timepoint [9] 0 0
Samples will be measured at Baseline and Months 3, 6, 9 and 12 for 10 Years
Secondary outcome [10] 0 0
Change from Baseline in IgM as a marker of inflammation - IgM
Timepoint [10] 0 0
Samples will be measured at Baseline and Months 6 and 12 for up to 10 Years
Secondary outcome [11] 0 0
Change from Baseline in C-reactive protein (CRP) as a marker of inflammation - C-reactive protein (CRP)
Timepoint [11] 0 0
Samples will be measured at Baseline and Months 6 and 12 for up to 10 Years
Secondary outcome [12] 0 0
Change from Baseline in tumor necrosis factor-alpha (TNF-a) as a marker of inflammation - tumor necrosis factor-alpha (TNF-a)
Timepoint [12] 0 0
Samples will be measured at Baseline and Months 6 and 12 for up to 10 Years
Secondary outcome [13] 0 0
Change from Baseline in fibroblast growth factor-19 (FGF-19) as a marker of inflammation - fibroblast growth factor-19 (FGF-19)
Timepoint [13] 0 0
Samples will be measured at Baseline and Months 6 and 12 for up to 10 Years
Secondary outcome [14] 0 0
Change from Baseline in cytokeratin-18 (CK-18) as a marker of liver fibrosis - cytokeratin-18 (CK-18)
Timepoint [14] 0 0
Samples will be measured at baseline and Months 6 and 12 for up to 10 Years
Secondary outcome [15] 0 0
Change from Baseline in enhanced liver fibrosis (ELF) test as a marker of liver fibrosis - enhanced liver fibrosis (ELF) test
Timepoint [15] 0 0
Samples will be measured at baseline and Months 6 and 12 for up to 10 Years
Secondary outcome [16] 0 0
Change from Baseline in transient elastography as a marker of liver fibrosis - Liver fibrosis measured using transient Elastography with Fibroscan®
Timepoint [16] 0 0
Liver fibrosis will be measured at baseline and Month 12 for up to 10 Years

Eligibility
Key inclusion criteria
Key

1. Definite or probable PBC diagnosis (consistent with American Association for the Study
of Liver Diseases [AASLD] and the European Association for the Study of the Liver
[EASL] practice guidelines; Lindor 2009; EASL 2009), as demonstrated by the presence
of =2 of the following 3 diagnostic factors:

- History of elevated Alkaline phosphatase levels for at least 6 months

- Positive antimitochondrial antibody (AMA) titer or if AMA negative or in low
titer (<1:80) PBC-specific antibodies (anti-GP210 and/or anti-SP100 and/or
antibodies against the major M2 components [PDC-E2, 2-oxo-glutaric acid
dehydrogenase complex])

- Liver biopsy consistent with PBC

2. A mean total bilirubin >ULN and =5x ULN and/or a mean ALP >3x ULN

3. Either is not taking UDCA (no UDCA dose in the past 3 months) or has been taking UDCA
for at least 12 months with a stable dose for =3 months prior to Day 0
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. History or presence of other concomitant liver diseases including:

- Hepatitis C virus infection

- Active Hepatitis B infection; however, subjects who have seroconverted (hepatitis
B surface antigen and hepatitis B e antigen negative) may be included in this
study after consultation with the medical monitor

- Primary sclerosing cholangitis (PSC)

- Alcoholic liver disease

- Definite autoimmune liver disease or overlap hepatitis

- Nonalcoholic steatohepatitis (NASH)

- Gilbert's Syndrome

2. Presence of clinical complications of PBC or clinically significant hepatic
decompensation, including:

- History of liver transplant, current placement on a liver transplant list, or
current Model of End Stage Liver Disease (MELD) score >12. Subjects who are
placed on a transplant list despite a relatively early disease stage (for example
per regional guidelines) may be eligible as long as they do not meet any of the
other exclusion criteria

- Cirrhosis with complications, including history (within the past 12 months) or
presence of:

- Variceal bleed

- Uncontrolled ascites

- Encephalopathy

- Spontaneous bacterial peritonitis

- Known or suspected HCC

- Prior transjugular intrahepatic portosystemic shunt procedure

- Hepatorenal syndrome (type I or II) or screening (Visit 1 or 2) serum creatinine
>2 mg/dL (178 µmol/L)

3. Mean total bilirubin >5x ULN

4. Subjects who have undergone gastric bypass procedures (gastric lap band is acceptable)
or ileal resection or plan to undergo either of these procedures

5. Other medical conditions that may diminish life expectancy, including known cancers
(except carcinomas in situ or other stable, relatively benign conditions)

6. If female: plans to become pregnant, known pregnancy or a positive urine pregnancy
test (confirmed by a positive serum pregnancy test), or lactating

7. Known history of human immunodeficiency virus infection

8. Medical conditions that may cause nonhepatic increases in ALP (eg, Paget's disease or
fractures within 3 months)

9. Other clinically significant medical conditions that are not well controlled or for
which medication needs are anticipated to change during the study

10. History of alcohol abuse or other substance abuse within 1 year prior to Day 0

11. Participation in another investigational product, biologic, or medical device study
within 30 days prior to Screening. Participation in a previous study of OCA is allowed
with 3 months washout prior to enrollment in this study

12. Mental instability or incompetence, such that the validity of informed consent or
ability to be compliant with the study is uncertain

13. History of known or suspected clinically significant hypersensitivity to OCA or any of
its components

14. UDCA naïve (unless contraindicated)

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 4
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
AW Morrow Gastroenterology and Liver Centre, Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
Department of Gastroenterology & Hepatology, Nepean Hospital - Kingswood
Recruitment hospital [3] 0 0
Gallipoli Medical Research Foundation - Brisbane
Recruitment hospital [4] 0 0
Department of Gastroenterology and Hepatology, Royal Adelaide Hospital - Adelaide
Recruitment hospital [5] 0 0
Flinders Medical Centre - Adelaide
Recruitment hospital [6] 0 0
Box Hill Hospital - Box Hill
Recruitment hospital [7] 0 0
St. Vincent's Hospital Melbourne - Fitzroy
Recruitment hospital [8] 0 0
Austin Hospital - Heidelberg
Recruitment hospital [9] 0 0
Fiona Stanley Hospital, Gastroenterology Department - Murdoch
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
2747 - Kingswood
Recruitment postcode(s) [3] 0 0
4120 - Brisbane
Recruitment postcode(s) [4] 0 0
5000 - Adelaide
Recruitment postcode(s) [5] 0 0
5042 - Adelaide
Recruitment postcode(s) [6] 0 0
3128 - Box Hill
Recruitment postcode(s) [7] 0 0
3065 - Fitzroy
Recruitment postcode(s) [8] 0 0
3084 - Heidelberg
Recruitment postcode(s) [9] 0 0
6150 - Murdoch
Recruitment outside Australia
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Nottingham

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

Ethics approval
Ethics application status

Summary
Brief summary
Primary Biliary Cholangitis (PBC) is a serious, life-threatening, bile acid related liver
disease of unknown cause. Without treatment, it frequently progresses to liver fibrosis and
eventual cirrhosis requiring liver transplantation or resulting in death. The investigational
drug, Obeticholic Acid (OCA) is a modified bile acid and FXR agonist that is derived from the
primary human bile acid chenodeoxycholic acid. The key mechanisms of action of OCA, including
its choleretic, anti-inflammatory, and anti-fibrotic properties, underlie its
hepatoprotective effects and result in attenuation of injury and improved liver function in a
cholestatic liver disease such as PBC. The study will assess the effect of OCA compared to
placebo, combined with stable standard care, on clinical outcomes in PBC patients.
Trial website
https://clinicaltrials.gov/show/NCT02308111
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.
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.
Public notes

Contacts
Principal investigator
Name 0 0
George Harb, MD
Address 0 0
Intercept Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Steven Lauder
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
steven.lauder@interceptpharma.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT02308111