Phase 4 Study of Obeticholic Acid Evaluating Clinical Outcomes in Patients With Primary Biliary Cholangitis



Status:Recruiting
Conditions:Gastrointestinal
Therapuetic Areas:Gastroenterology
Healthy:No
Age Range:18 - Any
Updated:3/21/2019
Start Date:December 2014
End Date:April 2025
Contact:Lisa Latvin
Email:lisa.latven@interceptpharma.com

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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

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.

This Phase 4, double-blind, randomized, placebo-controlled, multicenter study is being
undertaken at up to 170 sites internationally to evaluate the effect of OCA on clinical
outcomes in 428 subjects with PBC. The study will include a screening period of up to 8
weeks, requiring two clinic visits. Eligible participants will be randomly allocated (1:1) to
treatment with either OCA 5 mg or matching placebo tablets, taken orally once daily for the
majority of subjects; dose and frequency will be modified for subjects with cirrhosis and
classified as Child-Pugh B or C. Randomization will be stratified by standard treatment with
UDCA (yes/no) and baseline liver function. The treatment period involves clinic visits
approximately every 3 months. At the 3 month visit or any study visit thereafter, if study
treatment is tolerated, participants' dose should be titrated per protocol in a blinded
manner eg for participants who are non-cirrhotic or classified as Child-Pugh A and randomized
to OCA, they should receive the maximum daily dose of 10 mg OCA, those on placebo continue to
receive placebo. Subsequently, daily dosage may return to 5 mg if necessary for these
participants who are non-cirrhotic or classified as Child-Pugh A, but should be increased to
10 mg if possible, based on tolerability and clinical judgment. Safety and tolerability will
be assessed by monitoring adverse events and vital signs, and blood and urine testing. The
study is event driven and total duration will be determined by the time required to accrue
approximately 127 primary endpoint events, estimated to be approximately 10 years. Subjects
are expected to have a minimum follow-up time of approximately 6 years.

Inclusion Criteria:

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 prior to
Day 0

- 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. Age ≥18 years

4. 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

5. Contraception: Female subjects of childbearing potential must use ≥1 effective method
of contraception during the trial and until 30 days following the last dose of
investigational product. Effective methods of contraception are considered to be:

- Hormonal (e.g. contraceptive pill, patch, intramuscular implant or injection; or

- Double barrier method, i.e. (a) condom (male or female) or (b) diaphragm, with
spermicide; or

- Intrauterine device (IUD); or

- Vasectomy (partner); or

- Abstinence, if in line with the preferred and usual lifestyle of the subject

6. Must provide written informed consent and agree to comply with the study protocol

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 such as
chronic lymphocytic leukemia)

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 prior to Day 0)

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)
We found this trial at
44
sites
3660 Vista Avenue
Saint Louis, Missouri 63104
Principal Investigator: Bruce Bacon, M.D.
Phone: 314-977-7067
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3500 Gaston Avenue
Dallas, Texas 75246
1.800.422.9567
Principal Investigator: Jacqueline O'Leary, MD
Phone: 214-818-7703
Baylor University Medical Center Baylor University Medical Center in Dallas, TX is ranked nationally in...
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3551 Roger Brooke Dr
Fort Sam Houston, Texas 78234
(210) 916-4141
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425 University Blvd.
Indianapolis, Indiana 46202
(317) 274-4591
Principal Investigator: Raj Vuppalanchi, M.D.
Phone: 317-278-6215
Indiana University INDIANA UNIVERSITY is a major multi-campus public research institution, grounded in the liberal...
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8700 Beverly Blvd # 8211
Los Angeles, California 90048
(1-800-233-2771)
Principal Investigator: Vinay Sundaram, M.D.
Phone: 310-423-6000
Cedars Sinai Med Ctr Cedars-Sinai is known for providing the highest quality patient care. Our...
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3400 Spruce St
Philadelphia, Pennsylvania 19104
 (215) 662-4000
Principal Investigator: K. Rajender Reddy, MD
Phone: 215-360-0836
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601 Elmwood Avenue
Rochester, New York 14642
(585) 275-2100
Principal Investigator: Jonathan Huang, D.O.
Phone: 585-275-4763
Univ of Rochester Medical Center One of the nation's top academic medical centers, the University...
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Atlanta, Georgia 30309
Principal Investigator: Raymond Rubin, M.D.
Phone: 404-605-4618
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1364 Clifton Rd NE
Atlanta, Georgia 30322
(404) 712-2000
Principal Investigator: Ryan Ford, MD
Phone: 404-712-4117
Emory University Hospital As the largest health care system in Georgia and the only health...
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Aurora, Colorado 80045
Principal Investigator: Lisa Forman, M.D.
Phone: 303-724-1866
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345 St Paul Pl
Baltimore, Maryland 21202
(410) 332-9000
Principal Investigator: Paul Thuluvath, M.D.
Phone: 410-576-5389
Mercy Medical Center "Mercy Medical Center" is a hospital located in Baltimore, Maryland. The landmark...
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303 E Chicago Ave
Chicago, Illinois 60611
(312) 503-8194
Principal Investigator: Steven Flamm, MD
Phone: 312-684-0264
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5841 S Maryland Ave
Chicago, Illinois 60637
(773) 702-1000
Principal Investigator: K. Gautham Reddy, M.D.
Phone: 773-702-4477
University of Chicago Medical Center The University of Chicago Medicine has been at the forefront...
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Dallas, Texas 75203
Principal Investigator: Parvez Mantry, M.D.
Phone: 214-947-4366
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Dallas, Texas 75231
Principal Investigator: Jacqueline O'Leary, MD
Phone: 817-424-1525
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2201 Inwood Rd
Dallas, Texas 75235
(214) 645-8300
Principal Investigator: Marlyn Mayo, MD
Phone: 214-648-2725
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Fort Worth, Texas 76104
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Gainesville, Florida 32610
Principal Investigator: Roberto Firpi-Morell, M.D.
Phone: 352-273-9464
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Houston, Texas 77030
Principal Investigator: Moises Nevah Rubin, MD
Phone: 713-500-6670
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6620 Main Street
Houston, Texas 77030
Principal Investigator: John Vierling, M.D.
Phone: 713-798-5349
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Indianapolis, Indiana 46237
Principal Investigator: David Pound, MD
Phone: 312-865-2959
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Jackson, Mississippi 39216
Principal Investigator: Brian Borg, MD
Phone: 601-940-3341
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425 Volker Boulevard
Kansas City, Missouri 64131
Principal Investigator: Bradley Freilich, MD
Phone: 816-759-5274
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La Plata, Buenos Aires
Principal Investigator: Raul Eduardo Adrover, MD
Phone: 54-221-483-8537
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500 S Preston St
Louisville, Kentucky
(502) 852-5555
Principal Investigator: Matthew Cave, M.D.
Phone: 502-852-2043
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Marietta, Georgia 30060
Principal Investigator: Aasim Sheikh, M.D
Phone: 678-388-2050
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Miami, Florida 33136
Principal Investigator: Cynthia Levy, M.D.
Phone: 305-243-4648
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Murray, Utah 84123
Principal Investigator: Terry Box, MD
Phone: 801-441-2106
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Nashville, Tennessee 37211
Principal Investigator: Robert Herring, M.D.
Phone: 615-832-5530
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New Orleans, Louisiana 70112
Principal Investigator: Fredric Regenstein, MD
Phone: 504-988-6902
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New York, New York 10021
Principal Investigator: Sonal Kumar, MD, MPH
Phone: 646-962-5302
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New York, New York 10003
Principal Investigator: Jahavi Naik, MD
Phone: 212-420-2354
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Novi, Michigan 48377
Principal Investigator: Stuart Gordon, M.D.
Phone: 248-344-2364
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703 Main Street
Paterson, New Jersey 07503
973.754.2000
Principal Investigator: Maliha Ahmad, MD
Phone: 973-754-2315
Saint Joseph's Regional Medical Center Rich in history, St. Joseph's Healthcare System has evolved from...
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Phoenix, Arizona 85054
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Rialto, California 92377
Principal Investigator: Zeid Kayali, M.D.
Phone: 909-883-2999
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Richmond, Virginia 23249
Principal Investigator: Velmir Luketic, MD
Phone: 804-675-6924
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1 Shields Ave
Sacramento, California 95616
(530) 752-1011
Principal Investigator: Christopher Bowlus, M.D.
Phone: 916-734-2911
University of California-Davis As we begin our second century, UC Davis is poised to become...
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Saint Paul, Minnesota 55114
Principal Investigator: Donald Zogg, M.D.
Phone: 612-870-5596
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San Antonio, Texas 78215
Principal Investigator: Eric Lawitz, M.D.
Phone: 210-253-3426
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Seattle, Washington 98104
Principal Investigator: Kris Kowdley, MD
Phone: 206-386-3201
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Southlake, Texas 76092
Principal Investigator: James Trotter, M.D.
Phone: 469-930-3125
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450 Serra Mall
Stanford, California 94305
(650) 723-2300
Principal Investigator: Aijaz Ahmed, MD
Phone: 650-721-4288
Stanford University Stanford University, located between San Francisco and San Jose in the heart of...
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Worcester, Massachusetts 01655
Principal Investigator: Gyongyi Szabo, M.D., Ph.D.
Phone: 774-442-1982
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