Trial · NCT02308111
Phase 4 Study of Obeticholic Acid Evaluating Clinical Outcomes in Patients With Primary Biliary Cholangitis
NCT02308111, Phase 4, Terminated, Intercept Pharmaceuticals; results first posted 2023-03-09
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Phase 4 Study of Obeticholic Acid Evaluating Clinical Outcomes in Patients With Primary Biliary Cholangitis. Trials, record NCT02308111. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02308111
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase 4 Study of Obeticholic Acid Evaluating Clinical Outcomes in Patients With Primary Biliary Cholangitis
- NCT number
- NCT02308111
- Status
- Terminated
- Phases
- Phase 4
- Enrollment
- 334 participants, actual
- Lead sponsor
- Intercept Pharmaceuticals
- Conditions
- Liver Cirrhosis, Biliary
- Interventions
- Obeticholic Acid (OCA), Placebo
- First posted
- 2014-12-04
- Last updated
- 2023-03-09
- Results first posted
- 2023-03-09
- Limitations and caveats
The estimated effect of treatment from the ITT population was underpowered and potentially biased, resulting in difficulties in the interpretation of the tests of hypotheses for the primary and key secondary endpoints.
Source ClinicalTrials.gov
Registered outcomes136
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Time to the First Occurrence of Composite Endpoint | 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 the Kaplan-Meier methodology. Point estimates and 95% confidence intervals (CIs) for the clinical events distribution percentiles (25th and 50th) are provided. |
| Primary | Time to the First Occurrence of Primary Clinical Event (Expanded Endpoint) | 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 without decompensation (for participants without decompensation or clinical evidence of portal hypertension at baseline). 71 endpoint events were observed in the OCA arm, and 80 were observed in the Placebo arm. The clinical 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. |
| Secondary | Time To First Occurrence Of Severe Decompensating Events of Expanded Composite Endpoint | 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 methodology. Point estimates and 95% CIs for the clinical events distribution percentiles (25th and 50th) are provided. |
| Secondary | Time To Liver Transplant Or Death (All-cause) | 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. |
| Secondary | Time to First Occurrence of Fatal Event (All-Cause) | 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 |
| Secondary | Time to First Occurrence of Liver Transplant | 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. |
| Secondary | Time to First Occurrence of Hospitalization Due to Hepatic Events | 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. |
| Secondary | Time to First Occurrence of Uncontrolled or Refractory Ascites | 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. |
| Secondary | Time to First Occurrence of MELD Score ≥15 | 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 was summarized through CIF estimate at 5 years. |
| Secondary | Time To Development Of Varix/Varices | 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. |
1 to 10 of 136Source ClinicalTrials.gov
See all 136 registered outcomesOutcomes136
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Time to the First Occurrence of Composite Endpoint | Time to first occurrence from date of randomization until the time to accrue approximately 127 primary endpoint events (up to 7 years) | The Intent-to-Treat (ITT) population included all randomized participants who received any dosage of OCA or placebo. | days | NUMBER |
| PRIMARY | Time to the First Occurrence of Primary Clinical Event (Expanded Endpoint) | Time to first occurrence from date of randomization until the time to accrue approximately 127 primary endpoint events (up to 7 years) | The ITT population included all randomized participants who received any dosage of OCA or placebo. | days | NUMBER |
| SECONDARY | Time To First Occurrence Of Severe Decompensating Events of Expanded Composite Endpoint | 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 ITT population included all randomized participants who received any dosage of OCA or placebo. | days | NUMBER |
| SECONDARY | Time To Liver Transplant Or Death (All-cause) | 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 ITT population included all randomized participants who received any dosage of OCA or placebo. | days | NUMBER |
| SECONDARY | Time to First Occurrence of Fatal Event (All-Cause) | Time to first occurrence from date of randomization until the date of death from any cause (up to 7 years) | The ITT population included all randomized participants who received any dosage of OCA or placebo. | days | NUMBER |
| SECONDARY | Time to First Occurrence of Liver Transplant | 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 ITT population included all randomized participants who received any dosage of OCA or placebo. | proportion of participants | NUMBER |
| SECONDARY | Time to First Occurrence of Hospitalization Due to Hepatic Events | 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) | The ITT population included all randomized participants who received any dosage of OCA or placebo. | proportion of participants | NUMBER |
| SECONDARY | Time to First Occurrence of Uncontrolled or Refractory Ascites | 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) | The ITT population included all randomized participants who received any dosage of OCA or placebo. | proportion of participants | NUMBER |
| SECONDARY | Time to First Occurrence of MELD Score ≥15 | 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 ITT population included all randomized participants who received any dosage of OCA or placebo. | proportion of participants | NUMBER |
| SECONDARY | Time To Development Of Varix/Varices | 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 ITT population included all randomized participants who received any dosage of OCA or placebo. | proportion of participants | NUMBER |
1 to 10 of 136Source ClinicalTrials.gov
See all 136 outcomesResults by group456
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Time to the First Occurrence of Composite Endpoint | Obeticholic Acid | 25th Percentile | 1092 | 95% Confidence Interval 670 to 1464 | days |
| Time to the First Occurrence of Composite Endpoint | Placebo | 25th Percentile | 970 | 95% Confidence Interval 688 to 1342 | days |
| Time to the First Occurrence of Composite Endpoint | Obeticholic Acid | 50th Percentile | NA | 95% Confidence Interval 1910 to NA | days |
| Time to the First Occurrence of Composite Endpoint | Placebo | 50th Percentile | NA | 95% Confidence Interval NA to NA | days |
| Time to the First Occurrence of Primary Clinical Event (Expanded Endpoint) | Obeticholic Acid | 25th Percentile | 370 | 95% Confidence Interval 261 to 638 | days |
| Time to the First Occurrence of Primary Clinical Event (Expanded Endpoint) | Placebo | 25th Percentile | 450 | 95% Confidence Interval 358 to 569 | days |
| Time to the First Occurrence of Primary Clinical Event (Expanded Endpoint) | Obeticholic Acid | 50th Percentile | 1827 | 95% Confidence Interval 1198 to NA | days |
| Time to the First Occurrence of Primary Clinical Event (Expanded Endpoint) | Placebo | 50th Percentile | 1102 | 95% Confidence Interval 841 to NA | days |
| Time To First Occurrence Of Severe Decompensating Events of Expanded Composite Endpoint | Obeticholic Acid | 25th Percentile | 1092 | 95% Confidence Interval 670 to 1408 | days |
| Time To First Occurrence Of Severe Decompensating Events of Expanded Composite Endpoint | Placebo | 25th Percentile | 929 | 95% Confidence Interval 679 to 1342 | days |
1 to 10 of 456Source ClinicalTrials.gov
See all 456 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Obeticholic Acid | 168 | |
| Overall Study | STARTED | Placebo | 166 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Obeticholic Acid | 168 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Placebo | 166 | |
| Overall Study | COMPLETED | Obeticholic Acid | 0 | |
| Overall Study | COMPLETED | Placebo | 0 | |
| Overall Study | NOT COMPLETED | Obeticholic Acid | 168 | |
| Overall Study | NOT COMPLETED | Placebo | 166 |
Source ClinicalTrials.gov
Adverse events340
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Pruritus | Skin and subcutaneous tissue disorders | Obeticholic Acid | 131 | 168 | |
| other | Pruritus | Skin and subcutaneous tissue disorders | Placebo | 85 | 166 | |
| other | Rash | Skin and subcutaneous tissue disorders | Obeticholic Acid | 10 | 168 | |
| other | Rash | Skin and subcutaneous tissue disorders | Placebo | 6 | 166 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Obeticholic Acid | 25 | 168 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Placebo | 12 | 166 | |
| other | Nausea | Gastrointestinal disorders | Obeticholic Acid | 25 | 168 | |
| other | Nausea | Gastrointestinal disorders | Placebo | 21 | 166 | |
| other | Abdominal pain | Gastrointestinal disorders | Obeticholic Acid | 21 | 168 | |
| other | Abdominal pain | Gastrointestinal disorders | Placebo | 17 | 166 |
1 to 10 of 340Source ClinicalTrials.gov
See all 340 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Obeticholic Acid | 16 | 168 |
| Total, all-cause mortality | Placebo | 12 | 166 |
| Total, other adverse events | Obeticholic Acid | 156 | 168 |
| Total, other adverse events | Placebo | 146 | 166 |
| Total, serious adverse events | Obeticholic Acid | 53 | 168 |
| Total, serious adverse events | Placebo | 53 | 166 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Obeticholic Acid vs Placebo: deaths from any cause | Trial comparisons |
| Obeticholic Acid vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
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