This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Phase 4 Study of Obeticholic Acid Evaluating Clinical Outcomes in Patients With Primary Biliary Cholangitis (NCT02308111), a randomized phase 4 study. The primary outcome is Time to the First Occurrence of Composite Endpoint.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02308111 |
| Phase | Phase 4 |
| Condition | Liver Cirrhosis, Biliary |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Quadruple |
| Enrollment | 334 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Intercept Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
Participants with primary biliary cholangitis (PBC) were eligible for the trial. The primary endpoint description defines the Intent-to-Treat population as randomized participants who received any dosage of obeticholic acid (OCA) or placebo. The registry record does not report additional eligibility criteria in the registry record recorded for this analysis.
Intervention
The intervention was OCA alongside established local standard of care. The described regimen began at 5 mg for a minimum 3 months, followed by titration up to a maximum 10 mg for the remainder of the trial, based on tolerability and CP Score.
Comparator
The comparison group received placebo in conjunction with established local standard of care. The record does not give further details of the placebo regimen.
Primary question
The trial asked whether OCA, compared with placebo alongside standard care, affected the time until the first occurrence of a composite clinical endpoint. The endpoint combined death, liver transplant, MELD of 15 or higher, uncontrolled ascites, and hospitalization for specified hepatic events. The registry record does not report the comparative endpoint estimates.
This question concerns the timing of a first qualifying event, rather than a change in a laboratory measure or a count of every event during follow-up. A participant could meet the composite endpoint through any one of its listed components. The record describes an event-driven observation period: follow-up was planned from randomization until approximately 127 primary endpoint events accrued, with follow-up up to 7 years. That description does not establish how long any individual participant was followed.
The study was registered as a Phase 4, randomized, double-blind, placebo-controlled, multicenter trial. Its stated objective was to assess clinical outcomes among people with PBC. The available registry entries describe the question and endpoint, but do not provide an estimate of the treatment comparison or its precision.
3. Trial Design
This was a Phase 4, randomized, parallel-group, placebo-controlled trial with quadruple masking. The official title describes it as double-blind; the registry’s masking field specifies quadruple masking. The sponsor was Intercept Pharmaceuticals. The condition listed was biliary liver cirrhosis, and the trial concerned participants with primary biliary cholangitis.
The registry lists enrollment of 334. Enrollment is not necessarily the same as the number included in a particular analysis. For the primary endpoint, the record defines the Intent-to-Treat population as all randomized participants who received any dosage of OCA or placebo. It does not report the number meeting that population definition or the number included in each endpoint analysis.
Participants were assigned to OCA or placebo. The OCA arm description specifies 5 mg for a minimum 3 months, then titration up to a maximum 10 mg for the remainder of the trial, depending on tolerability and CP Score. Both groups were to receive the established local standard of care. The placebo arm description gives no further dosing detail.
The registry records a start date of 2014-12-26 and a primary completion date of 2021-12-23. Trial status is listed as terminated. The reason for termination, and whether termination altered follow-up or analysis plans, are not reported in the registry record.
The primary outcome was defined as time from randomization to the first occurrence of a composite endpoint. The timeframe was described as continuing until approximately 127 primary endpoint events accrued, up to 7 years. The record says clinical-event distribution percentiles were estimated using Kaplan-Meier methodology, with point estimates and 95% confidence intervals for the 25th and 50th percentiles. The registry record does not provide those estimates. It also does not report the allocation ratio or details of the randomization procedure.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Before assignment: A total of 334 participants were randomized into the study. The study was terminated early as the Data Monitoring Committee made the recommendation not to pursue further enrollment, given the lack of feasibility for this post-marketing study as designed. At termination, the study randomized \<80% of planned enrollment.
Overall Study
| Stage | Obeticholic Acid | Placebo |
|---|---|---|
| STARTED | 168 | 166 |
| Received at Least 1 Dose of Study Drug | 168 | 166 |
| COMPLETED | 0 | 0 |
| NOT COMPLETED | 168 | 166 |
Reasons for not completing this period:
| Reason | Obeticholic Acid | Placebo |
|---|---|---|
| Adverse Event | 31 | 19 |
| Death | 9 | 6 |
| Lost to Follow-up | 2 | 7 |
| Physician Decision | 6 | 17 |
| Protocol Violation | 0 | 1 |
| Withdrawal by Subject | 28 | 38 |
| Study Terminated by Sponsor | 72 | 61 |
| Initiated Commercial OCALIVA | 6 | 8 |
| Site Closure | 3 | 3 |
| Non-compliance with Study Drug | 3 | 1 |
| COVID-19 Pandemic Limitation | 2 | 0 |
| Liver Transplant | 3 | 3 |
Analysis population for the primary outcome, as defined in the registry: The Intent-to-Treat (ITT) population included all randomized participants who received any dosage of OCA or placebo.
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
Population: All randomized participants who received at least 1 dose of the study drug.
| Characteristic | Unit | Obeticholic Acid | Placebo | Total |
|---|---|---|---|---|
| Participants analysed | 168 | 166 | 334 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 140 | 140 | 280 |
| Age, Categorical: >=65 years | Participants | 28 | 26 | 54 |
| Age, Continuous | years | 53.4 (SD 10.28) | 53.9 (SD 10.41) | 53.7 (SD 10.33) |
| Sex: Female, Male: Female | Participants | 151 | 149 | 300 |
| Sex: Female, Male: Male | Participants | 17 | 17 | 34 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 24 | 18 | 42 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 138 | 139 | 277 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 6 | 9 | 15 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 1 | 2 | 3 |
| Race (NIH/OMB): Asian | Participants | 11 | 9 | 20 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 2 | 2 | 4 |
| Race (NIH/OMB): White | Participants | 146 | 143 | 289 |
| Race (NIH/OMB): More than one race | Participants | 4 | 1 | 5 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 4 | 9 | 13 |
6. Primary Endpoint
The registry lists the primary outcome as Time to the First Occurrence of Composite Endpoint, measured over Time to first occurrence from date of randomization until the time to accrue approximately 127 primary endpoint events (up to 7 years).
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | days |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint measures the time from randomization to the first occurrence of any event in a composite: death, liver transplant, MELD score of 15 or higher, uncontrolled ascites, or hospitalization for new onset or recurrence of variceal bleed, hepatic encephalopathy, or spontaneous bacterial peritonitis. For hepatic encephalopathy, the endpoint description specifies a West Haven score of 2 or higher. The events were described as adjudicated.
A time-to-event endpoint retains information about when a first event occurs, not only whether an event occurred by a single fixed time. Kaplan-Meier methods can estimate the distribution of event times when participants have different follow-up durations. In general, a composite endpoint can capture several clinically important types of outcomes in one analysis, but its components may differ in seriousness, frequency, and timing. A composite estimate therefore does not by itself show which component accounts for an observed pattern.
The registry record says the clinical-event distribution was estimated using Kaplan-Meier methodology and that the 25th and 50th percentiles, with 95% confidence intervals, were to be reported. It gives the endpoint definition and planned event-driven timeframe, but not the percentile estimates, confidence limits, or component-specific results. The endpoint description is therefore informative about what was to be measured, but does not establish the observed timing of events or the comparison between groups.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies the primary comparison and the statistical quantity intended to describe it, but does not report the primary endpoint estimates. It names time to the first occurrence of the composite endpoint and states that Kaplan-Meier estimates of the clinical-event distribution were used. It also specifies that estimates and 95% confidence intervals for the 25th and 50th percentiles were to be provided. Those numerical results are not reported in the registry record. Consequently, the posted information does not show the estimated time to either percentile in either group, nor a treatment contrast or confidence interval for such a contrast.
The distinction between an endpoint definition and a result is essential. The listed events tell readers what counted as the first primary event; they do not establish how often events occurred, how soon they occurred, or whether the groups differed. Likewise, the planned accrual of approximately 127 primary endpoint events is a description of the study timeframe, not a report that this number was reached or an outcome result.
The record lists enrollment of 334, but enrollment alone cannot be used to infer the number of people contributing to the primary analysis. The endpoint population is defined as randomized participants who received any dosage of OCA or placebo. The number meeting that definition and the numbers analyzed by group are not reported in the registry record.
The safety information lists serious adverse events affecting 53 participants in each group, with 168 at risk in the OCA group and 166 at risk in the placebo group. It also lists other adverse events affecting 156 and 146 participants, respectively, with the same group-specific at-risk counts, and deaths of 16 and 12. These are descriptive counts as recorded. Without the corresponding analysis methods, event-level details, follow-up information, or a reported inferential comparison, they do not establish whether a difference is meaningful or attributable to treatment. The listed serious-event terms and percentages are not assigned to a treatment group in the recorded details.
The trial is marked terminated, but the reason is not reported in the registry record. Termination alone does not explain the available outcome reporting and should not be interpreted as evidence for or against an effect. Overall, the posted information supports a description of the trial design, endpoint, and selected safety counts, but not a conclusion about the primary treatment comparison. No effect estimate, hypothesis test, or precision measure for that comparison is available in the registry record.
9. Statistical Methods Explained
What does a time-to-event endpoint represent?
The primary outcome is time from randomization to the first qualifying event. Each participant’s relevant observation ends at the first event that satisfies the composite definition, rather than counting later occurrences for the primary endpoint. The listed qualifying events are death, liver transplant, MELD score of 15 or higher, uncontrolled ascites, and hospitalization for specified hepatic events. This endpoint structure makes the timing of the first event central to the analysis.
In general, time-to-event methods are useful when participants may have different follow-up durations. Some may experience the event during observed follow-up; others may not have an event recorded before follow-up ends. When an event has not been observed by the end of available follow-up, the participant’s event time is not known exactly. Time-to-event analyses can use the observed follow-up without treating every such participant as if an event had occurred or as if follow-up had continued indefinitely. The registry entries do not report the censoring rules or individual follow-up details used in this trial.
What does Kaplan-Meier methodology estimate?
The registry states that the clinical-event distribution was estimated using Kaplan-Meier methodology. In general, the Kaplan-Meier method estimates the probability of remaining without the event over time while accounting for participants whose event time is not observed during their available follow-up. The resulting curve describes the event-free distribution over follow-up; it is not, by itself, a causal explanation for differences between groups.
The record specifies estimates for the 25th and 50th percentiles of the clinical-event distribution, together with 95% confidence intervals. In general, a percentile of an event-time distribution locates the time by which a stated portion of the distribution has reached the event. The 50th percentile is often called the median event time when it can be estimated. A percentile may not be estimable from the observed data if the distribution does not reach that point during follow-up. The registry record does not provide the percentile values or state whether either was estimable.
A confidence interval expresses uncertainty around an estimate under the assumptions of the statistical method. In general, a wider interval indicates less precision than a narrower interval, while the interval’s coverage interpretation depends on the method and its assumptions. A 95% confidence interval is not a guarantee that the true value lies within the particular reported interval. No confidence limits for the primary endpoint are reported in the registry record, so its precision cannot be assessed from the registry entries.
Why use a composite endpoint?
The primary outcome combines several types of clinical events into one endpoint, and the first one to occur determines the time-to-event outcome. In general, combining events can make an endpoint capture a wider range of clinically consequential outcomes. It also means a single summary can conceal different patterns among its components. For example, a composite result could be influenced more by one component than another, so component-specific information can help explain what the overall measure represents. The registry record lists the components but does not report their separate results.
The endpoint description identifies the events as adjudicated. Adjudication generally refers to a structured review used to determine whether a reported event meets the endpoint definition. The registry entries do not provide further details about the adjudication process, criteria, or review procedures.
How should the event-driven timeframe be read?
The record describes follow-up from randomization until approximately 127 primary endpoint events accrued, up to 7 years. This is an event-driven timeframe: the endpoint observation period was linked to the accumulation of events, subject to the stated maximum duration. It is not a reported count of events observed, and it does not indicate that all participants had equal follow-up. The registry entries do not state whether the target event count was reached or how many events occurred in each group.
In general, event-driven follow-up can allow information to accumulate until a planned amount of endpoint experience is available. Interpretation still depends on the observed events, follow-up, analysis population, and statistical plan. The registry record does not report the target’s role in a formal testing procedure, the analysis timing, or whether any interim analyses were conducted.
What is not available about the analysis?
The registry describes the Kaplan-Meier estimation of percentiles, but does not report a comparative effect measure, hypothesis test, or model comparing treatment groups. It also does not report assumptions or procedures for missing data, censoring, or handling of events that might affect observation of another endpoint component. Such details can matter in time-to-event analyses; their absence means readers should not infer the exact analysis procedure beyond the methods explicitly described.
General statistical interpretation also distinguishes estimates from precision. A group-specific event-time estimate describes the data under the method, while an uncertainty interval communicates how precisely it is estimated. A comparison between groups requires an explicitly reported contrast or suitable comparative analysis; group estimates alone do not automatically supply one. Since neither the primary estimates nor a group comparison are reported, the endpoint’s direction, magnitude, and precision cannot be assessed.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 136 outcome measures and 16 analyses. These counts indicate that the record contains multiple outcome and analysis entries, but do not establish how many were prespecified as primary, how they were prioritized, or which formed a confirmatory testing family. The primary endpoint is identified as time to the first occurrence of a composite endpoint. No statistical plan for controlling multiplicity is reported in the registry record.
In general, evaluating multiple outcomes or repeated analyses can increase the chance of a seemingly notable finding arising by chance if each is tested without adjustment. Multiplicity procedures can address this issue, but the appropriate approach depends on the planned hypotheses and analysis strategy. The registry entries do not identify any adjustment method, testing hierarchy, or interpretation rule for multiple results. Because primary endpoint results are not reported, there is no posted primary comparison to interpret in relation to other outcomes.
The endpoint population is defined as randomized participants who received any dosage of OCA or placebo. That definition does not tell readers how missing outcome information was handled, how participants with incomplete follow-up were treated, or what sensitivity analyses were conducted. These procedures are not reported in the registry record. The record also does not describe the extent or pattern of missing data.
These omissions do not establish that multiplicity or missing data were mishandled; they mean the relevant procedures cannot be evaluated from the registry entries. The sample-size assumptions describe an illustration based on an observed effect, a two-sided significance level of 0.05, equal allocation, and a normal approximation. The registry entries do not state the illustration’s numerical outputs, and those planning assumptions do not substitute for the trial’s statistical plan or observed results.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Obeticholic Acid | 53 | 168 | 31.5% |
| Placebo | 53 | 166 | 31.9% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Obeticholic Acid | 16 of 168 |
| Placebo | 12 of 166 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Obeticholic Acid | 156 of 168 |
| Placebo | 146 of 166 |
Most frequent serious adverse event terms
| Event term | Organ system | Obeticholic Acid | Placebo |
|---|---|---|---|
| Oesophageal varices haemorrhage | Gastrointestinal disorders | 4 of 168 | 5 of 166 |
| Hepatic encephalopathy | Nervous system disorders | 3 of 168 | 5 of 166 |
| Ascites | Gastrointestinal disorders | 2 of 168 | 4 of 166 |
| Urinary tract infection | Infections and infestations | 3 of 168 | 1 of 166 |
| Hepatic failure | Hepatobiliary disorders | 2 of 168 | 2 of 166 |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | 0 of 168 | 3 of 166 |
| Urosepsis | Infections and infestations | 0 of 168 | 3 of 166 |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | 1 of 168 | 2 of 166 |
| Disease progression | General disorders | 2 of 168 | 1 of 166 |
| Oedema peripheral | General disorders | 2 of 168 | 1 of 166 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Obeticholic Acid | Placebo |
|---|---|---|---|
| Pruritus | Skin and subcutaneous tissue disorders | 131 of 168 | 85 of 166 |
| Arthralgia | Musculoskeletal and connective tissue disorders | 23 of 168 | 29 of 166 |
| Urinary tract infection | Infections and infestations | 20 of 168 | 30 of 166 |
| Oedema peripheral | General disorders | 31 of 168 | 18 of 166 |
| Diarrhoea | Gastrointestinal disorders | 21 of 168 | 26 of 166 |
| Varices oesophageal | Gastrointestinal disorders | 20 of 168 | 27 of 166 |
| Nausea | Gastrointestinal disorders | 25 of 168 | 21 of 166 |
| Headache | Nervous system disorders | 23 of 168 | 21 of 166 |
| Blood bilirubin increased | Investigations | 19 of 168 | 25 of 166 |
| Fatigue | General disorders | 18 of 168 | 25 of 166 |
Serious events with the highest arm-level rate: oesophageal varices haemorrhage (3.0%), hepatic encephalopathy (3.0%), ascites (2.4%), gastrointestinal haemorrhage (1.8%), urosepsis (1.8%).
The safety listing reports serious adverse events for the OCA and placebo groups. In each group, 53 participants are listed as affected; the corresponding at-risk counts are 168 for OCA and 166 for placebo. The listing also reports other adverse events affecting 156 participants in the OCA group and 146 in the placebo group, with at-risk counts of 168 and 166, respectively. Deaths are listed as 16 and 12. These are the counts in the registry record; they should be read as descriptive information, not as a treatment-effect estimate.
Counts alone do not show the duration of observation, whether each affected participant had one or multiple events, or how the categories were defined and collected. The registry entries do not report those details or a statistical comparison of the safety groups. They also do not state whether the deaths are included among the serious adverse event counts. No percentage should be inferred from these counts for this interpretation.
The record lists serious adverse event terms with percentages: oesophageal varices haemorrhage, 3.0%; hepatic encephalopathy, 3.0%; ascites, 2.4%; gastrointestinal haemorrhage, 1.8%; urosepsis, 1.8%; and urinary tract infection, 1.8%. The registry entries do not assign these percentages to a specific group or state their denominators, so they should not be treated as group-specific rates. The listing provides safety context, but does not by itself establish a comparative safety conclusion.
12. What the Result Does, and Does Not, Mean
The trial’s registry record describes a randomized comparison of OCA plus standard care with placebo plus standard care in participants with PBC. Its primary question concerns time to the first event in a composite of death, liver transplant, MELD score of 15 or higher, uncontrolled ascites, or hospitalization for specified hepatic events. The record identifies Kaplan-Meier estimation and percentile confidence intervals as part of the planned presentation of the primary endpoint.
What the posted information does not show is the primary endpoint estimate, the confidence intervals, or a comparative treatment effect. Therefore it cannot establish whether the observed timing of the composite endpoint differed between groups, how large any difference may have been, or how precisely it was estimated. The endpoint definition and event-driven follow-up description are not substitutes for results.
The safety listing provides group-specific counts of participants affected by serious and other adverse events, group-specific at-risk counts, and deaths. It also names serious event terms with percentages. These descriptive entries are not accompanied by a reported comparative analysis, and the term percentages are not assigned to a group in the registry record. They do not, by themselves, establish a treatment-related cause or a comparative safety effect.
In general, interpretation of a clinical trial depends on both the design and the reported estimates, their uncertainty, and the analysis methods. Here, the design and endpoint are described, but details about the statistical plan, missing data, multiplicity, and the reason for trial termination are not reported in the registry record. The record’s terminated status should not be read as a result or an explanation of the outcome reporting.
Accordingly, the available information supports a clear account of the research question, trial structure, endpoint definition, and selected safety counts. It does not support a conclusion about whether OCA changed the primary endpoint or about the size and precision of any such effect. The trial’s findings on the stated primary question are not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically notable for defining its primary outcome as a time-to-event composite rather than a single event or a fixed-time measurement. The endpoint combines death, liver transplant, MELD score of 15 or higher, uncontrolled ascites, and hospitalization for specified hepatic events. Such an endpoint requires readers to understand both the timing analysis and the mix of components contributing to the first event.
The record describes Kaplan-Meier estimation of the clinical-event distribution and confidence intervals for the 25th and 50th percentiles. These methods focus on event timing and can accommodate varying observed follow-up in general. The timeframe was described as continuing until approximately 127 primary endpoint events accrued, up to 7 years. This event-driven framing makes the number and timing of observed events central to interpretation, but the registry entries do not report whether the target was reached or the observed event distribution.
The trial also illustrates why design details and numerical results must be distinguished. Randomization, masking, a placebo comparator, and a defined Intent-to-Treat population provide important context for evaluating a treatment comparison. However, without group estimates, confidence intervals, and analysis details, the primary comparison cannot be evaluated for magnitude or precision. The registry’s termination status is a study-status fact, not an explanation of what the results mean.
Finally, the safety entries show why raw counts require context. At-risk counts and affected counts are reported, but the registry entries do not provide follow-up duration, event-level detail, or a comparative statistical analysis. The record therefore offers a useful account of the intended clinical question and selected safety information while leaving the main efficacy estimate and several interpretive details unavailable.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The principal limitation of the registry information is that it does not report the primary endpoint estimates, confidence intervals, or a group comparison. Although the endpoint definition states that the 25th and 50th percentiles were to be estimated, their values are not reported in the registry record. The status of the event-driven target of approximately 127 events is also not reported, so the amount of endpoint information actually accrued cannot be determined from these registry entries.
The registry marks the trial as terminated but does not report why it was terminated or how termination affected follow-up, data collection, or analysis. The reason should not be inferred from the status alone. The record gives enrollment of 334 and an Intent-to-Treat population definition, but does not report the analysis population counts or the number analyzed in each group.
Important statistical details are not reported, including the comparative model or test, missing-data procedures, censoring rules, multiplicity strategy, and any sensitivity analyses. The record describes Kaplan-Meier estimation, but the registry entries do not provide enough detail to assess the full implementation or its assumptions in this trial. The composite endpoint’s components are listed, but component-specific results are not reported.
The safety entries provide affected and at-risk counts and deaths, but the registry entries do not describe follow-up duration, repeated events, or a formal between-group analysis. The listed serious-event percentages have no group assignment or denominator stated in the registry record. These limitations constrain interpretation of both the primary endpoint and safety information; they do not establish that any particular analysis was inappropriate.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Phase 4 Study of Obeticholic Acid Evaluating Clinical Outcomes in Patients With Primary Biliary Cholangitis — NCT02308111.
- Registry record last updated 2023-03-09; status last verified 2023-02.
- Results first posted 2023-03-09.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
NCT02308111 is a Phase 4, randomized, parallel-group, placebo-controlled, quadruple-masked trial of obeticholic acid in participants with primary biliary cholangitis. The record lists enrollment of 334, sponsor Intercept Pharmaceuticals, and status terminated. It describes OCA beginning at 5 mg for a minimum 3 months, with titration up to a maximum 10 mg for the remainder of the trial based on tolerability and CP Score, alongside standard care.
The primary outcome is time from randomization to the first occurrence of a composite clinical endpoint. The endpoint includes death, liver transplant, MELD score of 15 or higher, uncontrolled ascites, and hospitalization for specified hepatic events. The record states that Kaplan-Meier methods were used to estimate the clinical-event distribution and that 25th and 50th percentile estimates with 95% confidence intervals were to be provided.
Primary endpoint estimates and comparative results are not reported in the registry record. Safety entries include serious and other adverse event counts, at-risk counts, deaths, and selected serious event terms. Further details about the statistical plan, missing data, multiplicity, and the reason for termination are not reported in the registry record.