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Phase 2 Study on Effects of Obeticholic Acid (OCA) on Lipoprotein Metabolism in Subjects With Primary Biliary Cirrhosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01865812
Recruitment Status : Completed
First Posted : May 31, 2013
Results First Posted : October 19, 2016
Last Update Posted : March 14, 2018
Information provided by (Responsible Party):
Intercept Pharmaceuticals

Brief Summary:
The purpose of this study is to determine if obeticholic acid (OCA) has an effect on cholesterol levels in the blood in patients with PBC.

Condition or disease Intervention/treatment Phase
Primary Biliary Cirrhosis Drug: obeticholic acid (OCA) Phase 2

Detailed Description:
This was a phase 2, open-label, multicenter study evaluating the effects of OCA on lipoprotein metabolism in subjects with PBC; in particular, OCA's effects on high density lipoprotein (HDL) cholesterol. Nuclear magnetic resonance (NMR) spectroscopy was utilized to quantify the changes in lipoprotein particle sizes and concentrations. Components of reverse cholesterol transport were also assessed.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 26 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
Official Title: A Phase 2 Clinical Trial Investigating the Effects of Obeticholic Acid on Lipoprotein Metabolism in Subjects With Primary Biliary Cirrhosis
Study Start Date : November 2013
Actual Primary Completion Date : August 2014
Actual Study Completion Date : September 2016

Arm Intervention/treatment
Experimental: OCA: 10 mg
obeticholic acid, oral administration, 10 mg, 8 weeks
Drug: obeticholic acid (OCA)
All subjects will be treated with OCA (oral administration, 10 mg, once daily) for 8 weeks and should continue their prestudy dose of ursodeoxycholic acid (UDCA). After completion of the 8 week Primary Treatment Phase of the study and the 4 week follow up period, during which time subjects do not take OCA, all eligible subjects will be offered the opportunity to enter an open label long term safety extension phase, during which they will receive 10 mg OCA daily for up to 2 years.
Other Name: 6α-ethyl chenodeoxycholic acid (6-ECDCA); INT-747

Primary Outcome Measures :
  1. Absolute Change From Baseline in High-density Lipoprotein (HDL) Cholesterol Concentration [ Time Frame: Baseline, Week 8 ]
  2. Absolute Change From Baseline in High-density Lipoprotein (HDL) Particle Size [ Time Frame: Baseline, Week 8 ]
  3. Absolute Change From Baseline in High-density Lipoprotein (HDL) Particle Concentration [ Time Frame: Baseline, Week 8 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Definite or probable primary biliary cirrhosis (PBC) diagnosis as demonstrated by the presence of ≥ 2 of the following 3 diagnostic factors:

    • History of elevated alkaline phosphatase (ALP) levels for at least 6 months
    • A positive anti-microbial antibody (AMA) titer or, if AMA negative or in low titer (<1:80), PBC specific antibodies
    • Liver biopsy consistent with PBC
  2. Age ≥ 18 years
  3. Taking UDCA for at least 12 months (stable dose for ≥ 3 months) prior to Day 0 or unable to tolerate UDCA (no UDCA for ≥ 3 months prior to Day 0)
  4. Contraception: Female subjects must be postmenopausal, surgically sterile, or if premenopausal, be prepared to use ≥ 1 effective (≤ 1% failure rate) method of contraception during the trial and until at least 30 days after the last dose of Investigational Product.
  5. Must provide written informed consent and agree to comply with the trial protocol.

Exclusion Criteria:

  1. Subjects with decompensated PBC (as determined by the Investigator)
  2. Severe pruritus or systemic treatment for pruritus (e.g. treatment with bile acid sequestrants or rifampicin) within 2 months of Day 0
  3. History or presence of other significant liver diseases including:

    • Active or chronic Hepatitis B or C virus (HBV, HCV) infection
    • Primary sclerosing cholangitis (PSC)
    • Alcoholic liver disease
    • Definite autoimmune liver disease or overlap hepatitis
    • Nonalcoholic steatohepatitis (NASH)

    NOTE: Subjects with Gilbert's disease or those with a history of hepatitis B who are currently antigen negative and seroconverted should not be considered exclusionary

  4. Uncontrolled diabetes or other uncontrolled or unstable medical condition that may interfere with trial results
  5. Administration of any of the following medications as specified below:

    • Prohibited 28 days prior to Day 0: bile acid sequestrants (BAS) including cholestyramine, colesevelam, colestipol or omega-3 fatty acid containing dietary supplements
    • Prohibited 3 months prior to Day 0 and throughout trial participation: serum-lipid modifying agents including 3-hydroxy-3-methylglutaryl-coenzyme A (HMG CoA) reductase inhibitors, fenofibrate or other fibrates, nicotinic acid and derivatives, ezetimibe, Vitamin E (other than as standard dietary supplement)
    • Prohibited 6 months prior to Day 0 and throughout the trial participation: azathioprine, colchicine, cyclosporine, methotrexate, mycophenolate mofetil, pentoxifylline; budesonide and other systemic corticosteroids; potentially hepatotoxic drugs (including α-methyl-dopa, sodium valproic acid, isoniazide, or nitrofurantoin)
    • Prohibited 12 months prior to Day 0 and throughout the trial participation: antibodies or immunotherapy directed against interleukins or other cytokines or chemokines
  6. Planned change in diet or exercise habits during participation in the trial
  7. Presence or history of clinically significant cardiac arrhythmias that may prohibit the subject from participating in the trial
  8. If female: known pregnancy, or has a positive urine pregnancy test (confirmed by a positive serum pregnancy test), or lactating
  9. Recent (3 months prior to day 0) participation in another trial involving OCA or participation in another investigational trial (30 days prior to Day 0) and during the trial

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01865812

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United States, California
Scripps Clinic
La Jolla, California, United States, 92037
University of California, Davis Medical Center
Sacramento, California, United States, 95817
United States, Florida
University of Miami
Miami, Florida, United States, 33136
United States, Indiana
Indiana University Medical Center
Indianapolis, Indiana, United States, 46202
United States, New York
Beth Israel Medical Center
New York, New York, United States, 10003
United States, Virginia
Richmond, Virginia, United States, 23249
United States, Washington
Swedish Medical Center
Seattle, Washington, United States, 98104
Sponsors and Collaborators
Intercept Pharmaceuticals
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Study Director: David Shapiro, MD Intercept Pharmaceuticals
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Responsible Party: Intercept Pharmaceuticals Identifier: NCT01865812    
Other Study ID Numbers: 747-205
First Posted: May 31, 2013    Key Record Dates
Results First Posted: October 19, 2016
Last Update Posted: March 14, 2018
Last Verified: March 2018
Additional relevant MeSH terms:
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Liver Cirrhosis
Liver Cirrhosis, Biliary
Pathologic Processes
Liver Diseases
Digestive System Diseases
Cholestasis, Intrahepatic
Bile Duct Diseases
Biliary Tract Diseases
Chenodeoxycholic Acid
Gastrointestinal Agents