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

NCT01865812 | Completed Phase 2 Results DMC

• 1 other identifier: 747-205
• Study Type: interventional
• Target: 27
• Locations: 1 country
• Sites: 7

Brief Summary

The purpose of this study was to determine if OCA had an effect on cholesterol levels in the blood in participants with primary biliary cirrhosis (PBC).

Conditions

Primary Biliary Cirrhosis

Outcome Measures

Primary Outcomes (3)

• Absolute Change From Baseline In High-density Lipoprotein (HDL) Cholesterol Concentration

  Baseline, Week 8

• Absolute Change From Baseline In HDL Particle Size

  Baseline, Week 8

• Absolute Change From Baseline In HDL Particle Number

  Baseline, Week 8

Secondary Outcomes (53)

• Median Change From Baseline In HDL Cholesterol Concentration At Weeks 4, 8, and 12

  Baseline, Week 4, Week 8, Week 12

• Median Change From Baseline In HDL Particle Size At Weeks 4, 8, and 12

  Baseline, Week 4, Week 8, Week 12

• Median Change From Baseline In HDL Particle Number At Weeks 4, 8, and 12

  Baseline, Week 4, Week 8, Week 12

• Median Change From Week 8 In HDL Cholesterol Concentration At Week 12

  Week 8, Week 12

• Median Change From Week 8 In HDL Particle Size At Week 12

  Week 8, Week 12

Study Arms (1)

OCA: 10 mg

EXPERIMENTAL

Obeticholic acid, oral administration, 10 milligrams (mg), 8 weeks

Drug: Obeticholic Acid

Interventions

Obeticholic Acid DRUG

All participants were treated with OCA (oral administration, 10 mg, once daily \[QD\]) for 8 weeks and continued 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 participants did not take OCA, all eligible participants were offered the opportunity to enter an open-label, long-term safety extension phase, during which they could receive 10 mg OCA QD for up to 2 years.

Also known as: 6α-Ethyl chenodeoxycholic acid (6-ECDCA), INT-747

OCA: 10 mg

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Definite or probable PBC diagnosis as demonstrated by the presence of ≥ 2 of the following 3 diagnostic factors:
• History of elevated alkaline phosphatase 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
• 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).
• Contraception: Female participants must have been postmenopausal, surgically sterile, or if premenopausal, were 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.
• Must have provided written informed consent and agreed to comply with the trial protocol.

You may not qualify if:

• Participants with decompensated PBC (as determined by the Investigator).
• Severe pruritus or systemic treatment for pruritus (for example, treatment with bile acid sequestrants or rifampicin) within 2 months of Day 0.
• History or presence of other significant liver diseases including:
• Active or chronic Hepatitis B or C virus infection
• Primary sclerosing cholangitis
• Alcoholic liver disease
• Definite autoimmune liver disease or overlap hepatitis
• Nonalcoholic steatohepatitis
• Uncontrolled diabetes or other uncontrolled or unstable medical condition that may have interfered with trial results.
• Administration of any of the following medications as specified below:
• Prohibited 28 days prior to Day 0: bile acid sequestrants 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 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
• Planned change in diet or exercise habits during participation in the trial.

Sponsors & Collaborators

• Intercept Pharmaceuticals: lead

Study Sites (7)

Scripps Clinic

La Jolla, California, 92037, United States

Location

University of California, Davis Medical Center

Sacramento, California, 95817, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

Indiana University Medical Center

Indianapolis, Indiana, 46202, United States

Location

Beth Israel Medical Center

New York, New York, 10003, United States

Location

McGuire DVAMC

Richmond, Virginia, 23249, United States

Location

Swedish Medical Center

Seattle, Washington, 98104, United States

Location

MeSH Terms

Conditions

Liver Cirrhosis, Biliary

Interventions

obeticholic acid

Condition Hierarchy (Ancestors)

Cholestasis, Intrahepatic; Cholestasis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Liver Diseases; Liver Cirrhosis; Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Medical Information
• Organization: Intercept Pharmaceuticals, Inc.

medinfo@interceptpharma.com

+1 844-782-4278

Study Officials

• George Harb, MD

  Intercept Pharmaceuticals, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 23, 2013
• First Posted: May 31, 2013
• Study Start: December 3, 2013
• Primary Completion: August 13, 2014
• Study Completion: September 12, 2016
• Last Updated: August 24, 2022
• Results First Posted: October 19, 2016

Record last verified: 2022-07

Locations

US (7)