Primary Biliary CirrhosisPhase 2NCT01865812

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

A Phase 2 study testing Obeticholic Acid in people with Primary Biliary Cirrhosis, run by Intercept Pharmaceuticals at 7 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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). Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Primary Biliary Cirrhosis
What is being tested?: Obeticholic Acid
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 27 participants
Where: 7 sitesUnited States
Study dates: 3 Dec 2013to 12 Sep 2016
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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.

Who cannot join

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

Show all 19Show fewer

Definite autoimmune liver disease or overlap hepatitis
Nonalcoholic steatohepatitis
Note: Participants with Gilbert's disease or those with a history of hepatitis B who were currently antigen negative and seroconverted were not considered exclusionary.
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.
Presence or history of clinically significant cardiac arrhythmias that may have prohibited the participant from participating in the trial.
If female: known pregnancy, or had a positive urine pregnancy test (confirmed by a positive serum pregnancy test), or lactating.
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.

Trial locations and contacts

7 sites in 1 country.

City, postcode or hospital

7 sites in 1 country

United States7 sites

Indiana University Medical Center
Indianapolis, Indiana, 46202
Scripps Clinic
La Jolla, California, 92037
University of Miami
Miami, Florida, 33136
Beth Israel Medical Center
New York, New York, 10003
McGuire DVAMC
Richmond, Virginia, 23249
University of California, Davis Medical Center
Sacramento, California, 95817
Swedish Medical Center
Seattle, Washington, 98104

Other trials in United States

About this study

Official title: A Phase 2 Clinical Trial Investigating the Effects of Obeticholic Acid on Lipoprotein Metabolism in Subjects With Primary Biliary Cirrhosis

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).

Read the detailed description

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

Conditions: Primary Biliary Cirrhosis
Being tested: Obeticholic Acid
Medical subject headings: Liver Cirrhosis, Biliary

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Obeticholic AcidDrug
Also called 6α-Ethyl chenodeoxycholic acid (6-ECDCA), INT-747
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.

Study groups in NCT01865812
Study group	Role	What they receive
OCA: 10 mg	Experimental	Obeticholic acid, oral administration, 10 milligrams (mg), 8 weeks Receives: Drug: Obeticholic Acid

What is being measured

The primary outcome is the main question the study is designed to answer.

Absolute Change From Baseline In High-density Lipoprotein (HDL) Cholesterol Concentration
Measured: Baseline, Week 8
Absolute Change From Baseline In HDL Particle Size
Measured: Baseline, Week 8
Absolute Change From Baseline In HDL Particle Number
Measured: Baseline, Week 8

Secondary outcomes (53)

Median Change From Baseline In HDL Cholesterol Concentration At Weeks 4, 8, and 12
Measured: Baseline, Week 4, Week 8, Week 12
Median Change From Baseline In HDL Particle Size At Weeks 4, 8, and 12
Measured: Baseline, Week 4, Week 8, Week 12
Median Change From Baseline In HDL Particle Number At Weeks 4, 8, and 12
Measured: Baseline, Week 4, Week 8, Week 12
Median Change From Week 8 In HDL Cholesterol Concentration At Week 12
Measured: Week 8, Week 12
Median Change From Week 8 In HDL Particle Size At Week 12
Measured: Week 8, Week 12
Median Change From Week 8 In HDL Particle Number At Week 12
Measured: Week 8, Week 12
Maximum Plasma Concentration (Cmax) Of OCA And Conjugates
Results are reported in nanograms per milliliter (ng/mL).
Measured: Week 8
Time To Reach Cmax (Tmax) For OCA And Conjugates
Results are reported in hours (h).
Measured: Week 8
Area Under The Concentration-time Curve From Hour 0 To Last Sampling Time (Hour 6) (AUC0-6) For OCA And Conjugates
Results are reported in hour*nanograms per milliliter (h*ng/mL).
Measured: Week 8
Median Change From Baseline In Total Cholesterol
Measured: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In Total Triglycerides
Measured: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In Low-density Lipoprotein (LDL) Cholesterol (Direct)
Measured: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In LDL Particle Size
Measured: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In Total LDL Particles
Results are reported in nanomoles per liter (nmol/L).
Measured: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In Very Low-density Lipoprotein (VLDL) Cholesterol
Results are reported in milligrams per deciliter (mg/dL).
Measured: Baseline, Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In VLDL Particle Size
Measured: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In VLDL Particles
Measured: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In Apolipoprotein A1 (ApoA1)
Results are reported in grams per liter (g/L).
Measured: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In Apolipoprotein B (ApoB)
Measured: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In ApoA1/ApoB Ratio
Measured: Baseline, Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In Apolipoprotein E
Measured: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In Lipoprotein-a
Measured: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In Lecithin-cholesterol Acyltransferase Activity
Results are reported in nanomoles/milliliter/hour (nmol/mL/h).
Measured: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Cholesteryl Ester Transfer Protein
Results are reported in picomole/milliliter/minute (pmol/mL/min).
Measured: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Prebeta-1 HDL Concentration
Results are reported in microgram/milliliter (ug/mL).
Measured: Baseline, Week 4, Week 8/End of Treatment (EOT), Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Macrophage Cholesterol Efflux
Results are reported as a percentage of cholesterol.
Measured: Baseline, Week 4, Week 8/End of Treatment (EOT), Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In C-reactive Protein
Measured: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Glycoprotein A
Results are reported in picograms/milliliter (pg/mL).
Measured: Baseline, Week 12, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Fibroblast Growth Factor-19
Measured: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT
Participants With Lipoprotein X
Lipoprotein samples were assessed using nuclear magnetic resonance spectroscopy for the presence/absence of Lipoprotein X. Lipoprotein X sometimes appears with advanced cholestasis and can confound assessment of other lipoprotein concentrations, particularly LDL.
Measured: Week 12 and Last Dose
Median Change From Baseline In Alkaline Phosphatase
Results are reported in units/Liter (U/L).
Measured: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Gamma-glutamyl Transferase
Measured: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Alanine Aminotransferase
Measured: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Aspartate Aminotransferase
Measured: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Total And Unconjugated (Direct) Bilirubin
Measured: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Albumin
Measured: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Prothrombin Time
Results are reported in seconds (sec).
Measured: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Prothrombin International Normalized Ratio
Measured: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Enhanced Liver Fibrosis (ELF) Score
Change in ELF was calculated as ELF score at the end of the study minus ELF score prior to the intervention (at baseline). A decrease in the ELF score was considered good as it reflected a decrease in liver fibrosis, and an increase in ELF score was considered bad as it reflected an increase in liver fibrosis.
Change in ELF scores ranged from -0.56 (good) to + 0.68 (bad).
Measured: Baseline, Month 12, Month 24/EOT
Median Change From Baseline In Hyaluronic Acid
Measured: Baseline, Month 12, Month 24/EOT
Median Change From Baseline In Amino-terminal Propeptide Of Type III Procollagen
Results are reported in micrograms/Liter (ug/L).
Measured: Baseline, Month 12, Month 24/EOT
Median Change From Baseline In Tissue Inhibitor Of Metalloproteinases 1
Measured: Baseline, Month 12, Month 24/EOT
Median Change From Baseline In Hepatic Stiffness
Results are reported in kilopascal (kPa).
Measured: Baseline, Month 12, Month 24/EOT
Median Change From Baseline In Total Bile Acids
Measured: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Total Endogenous Bile Acid
Measured: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Total UDCA
Measured: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Total Chenodeoxycholic Acid
Measured: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Total Lithocholic Acid
Measured: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Total Cholic Acid
Measured: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Total Deoxycholic Acid
Measured: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Absolute Change From Baseline In HDL Cholesterol Concentration
Measured: Baseline, Month 24/EOT
Absolute Change From Baseline In HDL Particle Size
Measured: Baseline, Month 24/EOT
Absolute Change From Baseline In HDL Particle Number
Measured: Baseline, Month 24/EOT

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

3 Dec 201312 Sep 2016

First posted31 May 2013 (estimated)
Study start3 Dec 2013
Primary completion13 Aug 2014
Study completion12 Sep 2016

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Primary Biliary CirrhosisStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Basic science
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 27 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01865812
Sponsor's protocol ID: 747-205
Status last verified: Jul 2022
First submitted: 23 May 2013
Last updated: 24 Aug 2022
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 24 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2 Clinical Trial Investigating the Effects of Obeticholic Acid on Lipoprotein Metabolism in Subjects With Primary Biliary Cirrhosis
ClinicalTrials.gov ID: NCT01865812
Acronym / sponsor ID: 747-205
Conditions: Primary Biliary Cirrhosis
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 27 participants (actual)
Lead sponsor: Intercept PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: 3 Dec 2013 (actual)
Primary completion: 13 Aug 2014 (actual)
Study completion: 12 Sep 2016 (actual)
First posted: 31 May 2013 (estimated)
Results: Posted 19 Oct 2016

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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).

Detailed description

Study design

Primary purpose: Basic science
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
OCA: 10 mg	Experimental	Drug: Obeticholic AcidObeticholic acid, oral administration, 10 milligrams (mg), 8 weeks

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (7 listed)

Definite or probable PBC diagnosis as demonstrated by the presence of ≥ 2 of the following 3 diagnostic factors:
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.

Key exclusion criteria (19 listed)

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:
Note: Participants with Gilbert's disease or those with a history of hepatitis B who were currently antigen negative and seroconverted were not considered exclusionary.
Uncontrolled diabetes or other uncontrolled or unstable medical condition that may have interfered with trial results.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Obeticholic AcidAlso: 6α-Ethyl chenodeoxycholic acid (6-ECDCA), INT-747All 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.	Drug	OCA: 10 mg

Primary outcome

Absolute Change From Baseline In High-density Lipoprotein (HDL) Cholesterol Concentration
Time frame: Baseline, Week 8
Absolute Change From Baseline In HDL Particle Size
Time frame: Baseline, Week 8
Absolute Change From Baseline In HDL Particle Number
Time frame: Baseline, Week 8

53 secondary outcomes

Median Change From Baseline In HDL Cholesterol Concentration At Weeks 4, 8, and 12
Time frame: Baseline, Week 4, Week 8, Week 12
Median Change From Baseline In HDL Particle Size At Weeks 4, 8, and 12
Time frame: Baseline, Week 4, Week 8, Week 12
Median Change From Baseline In HDL Particle Number At Weeks 4, 8, and 12
Time frame: Baseline, Week 4, Week 8, Week 12
Median Change From Week 8 In HDL Cholesterol Concentration At Week 12
Time frame: Week 8, Week 12
Median Change From Week 8 In HDL Particle Size At Week 12
Time frame: Week 8, Week 12
Median Change From Week 8 In HDL Particle Number At Week 12
Time frame: Week 8, Week 12
Maximum Plasma Concentration (Cmax) Of OCA And Conjugates
Time frame: Week 8
Time To Reach Cmax (Tmax) For OCA And Conjugates
Time frame: Week 8
Area Under The Concentration-time Curve From Hour 0 To Last Sampling Time (Hour 6) (AUC0-6) For OCA And Conjugates
Time frame: Week 8
Median Change From Baseline In Total Cholesterol
Time frame: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In Total Triglycerides
Time frame: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In Low-density Lipoprotein (LDL) Cholesterol (Direct)
Time frame: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In LDL Particle Size
Time frame: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In Total LDL Particles
Time frame: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In Very Low-density Lipoprotein (VLDL) Cholesterol
Time frame: Baseline, Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In VLDL Particle Size
Time frame: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In VLDL Particles
Time frame: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In Apolipoprotein A1 (ApoA1)
Time frame: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In Apolipoprotein B (ApoB)
Time frame: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In ApoA1/ApoB Ratio
Time frame: Baseline, Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In Apolipoprotein E
Time frame: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In Lipoprotein-a
Time frame: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT, Last Dose
Median Change From Baseline In Lecithin-cholesterol Acyltransferase Activity
Time frame: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Cholesteryl Ester Transfer Protein
Time frame: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Prebeta-1 HDL Concentration
Time frame: Baseline, Week 4, Week 8/End of Treatment (EOT), Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Macrophage Cholesterol Efflux
Time frame: Baseline, Week 4, Week 8/End of Treatment (EOT), Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In C-reactive Protein
Time frame: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Glycoprotein A
Time frame: Baseline, Week 12, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Fibroblast Growth Factor-19
Time frame: Baseline, Week 4, Week 8/End of Treatment (EOT), Week 12, Month 6, Month 12, Month 18, Month 24/EOT
Participants With Lipoprotein X
Time frame: Week 12 and Last Dose
Median Change From Baseline In Alkaline Phosphatase
Time frame: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Gamma-glutamyl Transferase
Time frame: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Alanine Aminotransferase
Time frame: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Aspartate Aminotransferase
Time frame: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Total And Unconjugated (Direct) Bilirubin
Time frame: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Albumin
Time frame: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Prothrombin Time
Time frame: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Prothrombin International Normalized Ratio
Time frame: Baseline, Month 6, Month 12, Month 18, Month 24/EOT
Median Change From Baseline In Enhanced Liver Fibrosis (ELF) Score
Time frame: Baseline, Month 12, Month 24/EOT
Median Change From Baseline In Hyaluronic Acid
Time frame: Baseline, Month 12, Month 24/EOT

Results and safety

Results posted to the registry on 19 Oct 2016, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Primary Treatment Phase	27	25
Long-term Safety Extension Phase	0	0

Primary outcome: Absolute Change From Baseline In High-density Lipoprotein (HDL) Cholesterol Concentration

Time frame: Baseline, Week 8. Least squares mean (95% Confidence Interval). Unit: mmol/L

Measure	Primary Treatment Phase n = 23
Value	-0.38 (-0.51 to -0.24)

Primary Treatment Phase: Mean Difference (Net): -0.38 (95% CI -0.51 to -0.24)

Population analysed: Intent-to-treat: all enrolled participants who received at least 1 dose of OCA during the study. All efficacy and safety endpoints were analyzed using the intent-to-treat population.

Primary outcome: Absolute Change From Baseline In HDL Particle Size

Time frame: Baseline, Week 8. Least squares mean (95% Confidence Interval). Unit: nm

Measure	Primary Treatment Phase n = 23
Value	-0.44 (-0.63 to -0.25)

Primary Treatment Phase: Mean Difference (Net): -0.44 (95% CI -0.63 to -0.25)

Primary outcome: Absolute Change From Baseline In HDL Particle Number

Time frame: Baseline, Week 8. Least squares mean (95% Confidence Interval). Unit: umol/L

Measure	Primary Treatment Phase n = 23
Value	-0.06 (-1.58 to 1.46)

Primary Treatment Phase: Mean Difference (Net): -0.06 (95% CI -1.58 to 1.46)

53 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse event data were collected from the time of the first dose of investigational product until the participant fully completed participation in the study, up to 2 years.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Primary Treatment Phase	1 of 26 (3.8%)	18 of 26 (69.2%)	0 of 26
Long-term Safety Extension Phase	5 of 21 (23.8%)	17 of 21 (81%)	0 of 21

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 24 Aug 2022.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

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RecruitingPhase 3
Seladelpar in Subjects With Primary Biliary Cholangitis (PBC) and Compensated Cirrhosis
Primary Biliary Cholangitis
RecruitingPhase 3
FIREFLY Trial: Fenofibrate Intervention---Randomized Evaluation in First-Line PBC Therapy
Primary Biliary Cholangitis (PBC)
RecruitingPhase 3
Fenofibrate for Patients With Primary Biliary Cirrhosis Who Had An Inadequate Response to Ursodeoxycholic Acid
Primary Biliary Cirrhosis
RecruitingPhase 2/3
Fenofibrate in Patients With Primary Biliary Cholangitis (PBC)
Primary Biliary Cholangitis

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

George Harb, MD study director

Intercept Pharmaceuticals, Inc.

7 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Obeticholic Acid in people with Primary Biliary Cirrhosis, run by Intercept Pharmaceuticals at 7 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 24 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 19 Oct 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Absolute Change From Baseline In High-density Lipoprotein (HDL) Cholesterol Concentration (time frame: Baseline, Week 8). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01865812.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Aug 2022.