This trial is active, not recruiting.

Condition primary biliary cirrhosis
Treatment obeticholic acid (oca)
Phase phase 2
Sponsor Intercept Pharmaceuticals
Start date November 2013
End date August 2014
Trial size 25 participants
Trial identifier NCT01865812, 747-205


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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose basic science
obeticholic acid, oral administration, 10mg, 8 weeks
obeticholic acid (oca) 6α-ethyl chenodeoxycholic acid (6-ECDCA); INT-747
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 study period, subjects will be offered the opportunity to enter an open label long term safety extension for up to years.

Primary Outcomes

Change from baseline in High-density lipoprotein (HDL) Metabolism
time frame: Week 4, Week 8 and Week 12

Secondary Outcomes

Change from baseline in Lipoprotein Metabolism
time frame: Week 4, Week 8 and Week 12
Change from baseline Reverse Cholesterol Transport
time frame: Week 4, Week 8 and Week 12
Pharmacokinetic parameters of OCA and OCA conjugates
time frame: Week 8

Eligibility Criteria

Male or female participants at least 18 years old.

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, or colestipol - 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), omega-3 fatty acid containing dietary supplements - 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

Additional Information

Official title A Phase 2 Clinical Trial Investigating the Effects of Obeticholic Acid on Lipoprotein Metabolism in Subjects With Primary Biliary Cirrhosis
Trial information was received from ClinicalTrials.gov and was last updated in April 2015.
Information provided to ClinicalTrials.gov by Intercept Pharmaceuticals.