18 and older, any sex, with Liver Cirrhosis, Biliary. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Time to the First Occurrence of Composite EndpointPrimary· Time to first occurrence from date of randomization until the time to accrue approximately 127 primary endpoint events (up to 7 years)
To assess the effect of OCA, compared to placebo in conjunction with the established local standard of care, on clinical outcomes in participants with PBC as measured by time to the first occurrence of any of the following adjudicated events, derived as a composite event endpoint of death, liver transplant, model of end-stage liver disease (MELD) ≥15, uncontrolled ascites, or hospitalization for new onset or recurrence of variceal bleed, hepatic encephalopathy (as defined by a West Haven score of \>=2), or spontaneous bacterial peritonitis. The clinical events distribution was estimated using
25th Percentile
Group
Value
95% CI
Obeticholic Acid
1092
670 – 1464
Placebo
970
688 – 1342
50th Percentile
Group
Value
95% CI
Obeticholic Acid
NA
1910 – NA
Placebo
NA
NA – NA
Time to the First Occurrence of Primary Clinical Event (Expanded Endpoint)Primary· Time to first occurrence from date of randomization until the time to accrue approximately 127 primary endpoint events (up to 7 years)
Primary clinical outcome event is the first occurrence of the following events: death, liver transplant, MELD score \>=15 (MELD-Na score \>=12 baseline), MELD-Na score \>=15 (MELD-Na score \<12 baseline), hospitalization for new onset or recurrence of variceal bleed, hepatic encephalopathy, spontaneous bacterial peritonitis (confirmed by diagnostic paracentesis), or bacterial empyema, uncontrolled or refractory ascites (requiring large volume paracentesis), portal hypertension syndromes, progression to decompensated liver disease, and progression to clinical evidence of portal hypertension wit
25th Percentile
Group
Value
95% CI
Obeticholic Acid
370
261 – 638
Placebo
450
358 – 569
50th Percentile
Group
Value
95% CI
Obeticholic Acid
1827
1198 – NA
Placebo
1102
841 – NA
Time To First Occurrence Of Severe Decompensating Events of Expanded Composite EndpointSecondary· Time to first occurrence from date of randomization until the date of first documented progression or date of death from any cause, whichever came first (up to 7 years)
The first occurrence of the key secondary clinical event refers to the first occurrence of the following events: death, liver transplant, MELD-Na score \>=15 if MELD-Na\< 12 at baseline, MELD score \>=15 if MELD-Na \>=12 at baseline, uncontrolled or refractory ascites, portal hypertension syndromes (hepatorenal syndrome, portopulmonary syndrome, hepatopulmonary syndrome) or hospitalization for new onset or recurrence of variceal bleed, hepatic encephalopathy, spontaneous bacterial peritonitis, or bacterial empyema. The clinical events distribution was estimated using the Kaplan-Meier methodolo
25th Percentile
Group
Value
95% CI
Obeticholic Acid
1092
670 – 1408
Placebo
929
679 – 1342
50th Percentile
Group
Value
95% CI
Obeticholic Acid
NA
1910 – NA
Placebo
NA
NA – NA
Time To Liver Transplant Or Death (All-cause)Secondary· Time to first occurrence from date of randomization until the date of first documented liver transplant or date of death from any cause (up to 7 years)
The effect of OCA compared to placebo on time to liver transplant or death (all-cause) was assessed. The events distribution was estimated using the Kaplan-Meier methodology. Point estimates and 95% CIs for the clinical events distribution percentiles (25th and 50th) are provided.
25th Percentile
Group
Value
95% CI
Obeticholic Acid
1580
1275 – NA
Placebo
1803
1206 – NA
50th Percentile
Group
Value
95% CI
Obeticholic Acid
NA
NA – NA
Placebo
NA
NA – NA
Time to First Occurrence of Fatal Event (All-Cause)Secondary· Time to first occurrence from date of randomization until the date of death from any cause (up to 7 years)
The results represent the ratio of OCA to placebo. The fatal events distribution was estimated using the Kaplan-Meier methodology. Point estimates and 95% CIs for the fatal events distribution percentiles (25th and 50th) are provided
25th Percentile
Group
Value
95% CI
Obeticholic Acid
NA
NA – NA
Placebo
NA
NA – NA
50th Percentile
Group
Value
95% CI
Obeticholic Acid
NA
NA – NA
Placebo
NA
NA – NA
Time to First Occurrence of Liver TransplantSecondary· Time to first occurrence from date of randomization until the date of first documented liver transplant or date of death from any cause (up to 5 years)
The effect of OCA compared to placebo on time to occurrence of a liver transplant was assessed. The results represented the ratio of OCA to placebo. A hazard ratio \<1 indicated an advantage for OCA. The event of interest was summarized through Cumulative Incidence Function (CIF) estimate at 5 years.
Group
Value
95% CI
Obeticholic Acid
0.18
0.11 – 0.26
Placebo
0.16
0.09 – 0.23
Time to First Occurrence of Hospitalization Due to Hepatic EventsSecondary· Time to first occurrence from date of randomization until the date of hospitalization, liver transplant or death from any cause, whichever came first (up to 5 years)
Hospitalization events include new onset or recurrent variceal bleed, hepatic encephalopathy (as defined by a West Haven score of \>=2), spontaneous bacterial peritonitis (confirmed by diagnostic paracentesis OR presence of \>250/mm\^3 polymorph leucocytes \[PMNs\] in the ascitic fluid), bacterial empyema is confirmed by diagnostic thoracentesis OR presence of \>250/mm\^3 PMNs in the pleural fluid. The event of interest was summarized through CIF estimate at 5 years.
Group
Value
95% CI
Obeticholic Acid
0.11
0.06 – 0.17
Placebo
0.18
0.10 – 0.26
Time to First Occurrence of Uncontrolled or Refractory AscitesSecondary· Time to first occurrence from date of randomization until the date of first documented uncontrolled or refractory ascites, liver transplant or date of death from any cause, whichever came first (up to 5 years)
Uncontrolled or refractory ascites are defined as diuretic-resistant ascites requiring large-volume paracentesis. The effect of OCA compared to placebo on time to the first occurrence of uncontrolled or refractory ascites was assessed. The event of interest was summarized through CIF estimate at 5 years.
Group
Value
95% CI
Obeticholic Acid
0.02
0.01 – 0.05
Placebo
0.04
0.01 – 0.08
Time to First Occurrence of MELD Score ≥15Secondary· Time to first occurrence from date of randomization until the date of first documented MELD Score ≥15, liver transplant or date of death from any cause, whichever came first (up to 5 years)
The MELD score is useful in assessing participants with significant decompensation. The MELD score is now used by the United Network for Organ Sharing in the United States and Eurotransplants to manage organ allocation for liver transplantation. The MELD score is derived from the participant's serum total bilirubin, serum creatinine, and International Normalized Ratio (INR), as appropriate, to predict survival. The MELD score ranges from 6 to 40. The higher the score, the more likely a participant will receive a liver from a deceased donor when an organ becomes available. The event of interest
Group
Value
95% CI
Obeticholic Acid
0.13
0.07 – 0.19
Placebo
0.18
0.11 – 0.25
Time To Development Of Varix/VaricesSecondary· Time to first occurrence from date of randomization until the date of first documented development of varix/varices, liver transplant or death from any cause, whichever came first (up to 5 years)
The effect of OCA compared to placebo on time to development of varix/varices was assessed. The event of interest was summarized through CIF estimate at 5 years.
Group
Value
95% CI
Obeticholic Acid
0.07
0.03 – 0.12
Placebo
0.09
0.04 – 0.17
Time To Liver-Related DeathSecondary· Time to first occurrence from date of randomization until the date of first liver-related or non-liver- related death, whichever came first (up to 5 years)
The effect of OCA compared to placebo on time to liver-related death was assessed. The event of interest was summarized through CIF estimate at 5 years.
Group
Value
95% CI
Obeticholic Acid
0.03
0.01 – 0.07
Placebo
0.04
0.01 – 0.09
Time To Liver-Related Death Or Liver TransplantSecondary· Time to first occurrence from date of randomization until the date of liver transplant, liver-related death or non-liver-related death from any cause, whichever came first (up to 5 years)
The effect of OCA compared to placebo on time to liver-related death or liver transplant was assessed. The event of interest was summarized through CIF estimate at 5 years.
Group
Value
95% CI
Obeticholic Acid
0.20
0.13 – 0.29
Placebo
0.19
0.12 – 0.27
Adverse events — posted to ClinicalTrials.gov
Time frame: Baseline up to the last IP dose plus 30 days and prior to commercial OCA initiation date, approximately 7 years.
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Obeticholic Acid
Serious: 53/168 (32%)
Deaths: 16/168
Placebo
Serious: 53/166 (32%)
Deaths: 12/166
Serious adverse events (128 terms)
Reaction
System
Obeticholic Acid
Placebo
Oesophageal varices haemorrhage
Gastrointestinal disorders
—
—
Hepatic encephalopathy
Nervous system disorders
—
—
Ascites
Gastrointestinal disorders
—
—
Gastrointestinal haemorrhage
Gastrointestinal disorders
—
—
Urinary tract infection
Infections and infestations
—
—
Urosepsis
Infections and infestations
—
—
Small intestinal obstruction
Gastrointestinal disorders
—
—
Sepsis
Infections and infestations
—
—
Hepatic cirrhosis
Hepatobiliary disorders
—
—
Hepatic failure
Hepatobiliary disorders
—
—
Cholecystitis
Hepatobiliary disorders
—
—
Acute respiratory failure
Respiratory, thoracic and mediastinal disorders
—
—
Pleural effusion
Respiratory, thoracic and mediastinal disorders
—
—
Cerebrovascular accident
Nervous system disorders
—
—
Hip fracture
Injury, poisoning and procedural complications
—
—
Disease progression
General disorders
—
—
Oedema peripheral
General disorders
—
—
Thrombotic thrombocytopenic purpura
Blood and lymphatic system disorders
—
—
Coronary artery disease
Cardiac disorders
—
—
Breast cancer
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
—
Hepatocellular carcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
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 participants.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT03633227 — Study of Obeticholic Acid (OCA) Evaluating Pharmacokinetics and Safety in Participants With Primary Biliary Cholangitis
· Phase 4
· terminated
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Intercept Pharmaceuticals
Last refreshed: 9 March 2023
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02308111.