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 Effect of Heavy Alcohol Consumption on Farnesoid X Receptor (FXR) Signaling (NCT02654236), a randomized na study. The primary outcome is Change in Bile Salt Metabolism (C4 )Levels to Determine Effect of FXR.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02654236 |
| Phase | NA |
| Condition | Alcohol Consumption |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Quadruple |
| Enrollment | 30 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Suthat Liangpunsakul |
| Results posted | Yes |
2. Clinical Question
Population
The study concerns heavy drinkers, with non-drinking healthy controls also described in the registry record. Enrollment is listed as 30. The record does not report further eligibility details, such as how heavy drinking was defined or which clinical characteristics were required.
Intervention
The intervention is 10 mg Obeticholic Acid (OCA), administered orally once daily for 4 weeks. The study description frames the investigation around heavy alcohol consumption and FXR signaling in the liver, but the registry record does not report how adherence to the medication schedule was assessed.
Comparator
The medication comparator is placebo, with an arm described as heavy drinkers on placebo. Non-drinking healthy controls are also listed as a group. The record does not explain whether those controls were included in the randomized medication comparison or how comparisons involving them were planned.
Primary question
The primary endpoint is change in bile salt metabolism, measured using C4 levels from baseline to 28 days, to determine the effect of FXR. The registry record states that zero participants were analyzed because the data were not collected. It therefore gives no analyzed estimate of change and cannot answer whether the intervention altered this endpoint.
3. Trial Design
The registry record describes a randomized, quadruple-masked, parallel study, with enrollment of 30 and status listed as completed. The recorded start is 2016-04 and primary completion is 2019-09. The phase is listed as NA. The interventions include placebo and 10 mg Obeticholic Acid (OCA); the arm descriptions also include non-drinking healthy controls.
In general, random allocation is intended to reduce systematic differences between treatment groups, while masking is intended to limit the influence of treatment knowledge on conduct or assessment. The record labels masking as quadruple but does not identify which roles were masked or explain how masking was maintained. A parallel design means the assigned groups are compared within the same study period; the registry record does not provide further details about visit schedules beyond the primary endpoint timeframe.
The OCA description specifies oral administration once daily for 4 weeks. The duration and schedule of placebo administration are not separately described. The record does not report the allocation ratio, recruitment locations, eligibility criteria, or how the non-drinking controls were selected. Although the study is marked completed, completion status does not itself establish that the planned outcomes were measured or analyzed: the primary outcome entry explicitly says no participants were analyzed because data were not collected.
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.
Overall Study
| Stage | Placebo | 10 mg Obeticholic Acid (OCA) | Non-drinking Controls |
|---|---|---|---|
| STARTED | 9 | 6 | 15 |
| COMPLETED | 9 | 6 | 13 |
| NOT COMPLETED | 0 | 0 | 2 |
Reasons for not completing this period:
| Reason | Placebo | 10 mg Obeticholic Acid (OCA) | Non-drinking Controls |
|---|---|---|---|
| Withdrawal by Subject | 0 | 0 | 2 |
Analysis population for the primary outcome, as defined in the registry: Zero participants were analyzed due to data not being collected
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.
| Characteristic | Unit | Placebo | 10 mg Obeticholic Acid (OCA) | Non-drinking Controls | Total |
|---|---|---|---|---|---|
| Participants analysed | 9 | 6 | 15 | 30 | |
| Age, Continuous | Years | 31 (21 to 63) | 49 (30 to 65) | 47 (27 to 60) | 39 (21 to 65) |
| Sex: Female, Male: Female | Participants | 5 | 2 | 10 | 17 |
| Sex: Female, Male: Male | Participants | 4 | 4 | 5 | 13 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 0 | 1 | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 9 | 6 | 14 | 29 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 3 | 3 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 6 | 3 | 4 | 13 |
| Race (NIH/OMB): White | Participants | 3 | 2 | 7 | 12 |
| Race (NIH/OMB): More than one race | Participants | 0 | 1 | 1 | 2 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 |
| Region of Enrollment: United States | participants | 9 | 6 | 15 | 30 |
6. Primary Endpoint
The registry lists the primary outcome as Change in Bile Salt Metabolism (C4 )Levels to Determine Effect of FXR, measured over Baseline to 28 days.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is change in bile salt metabolism, assessed through C4 levels from baseline to 28 days. The endpoint title says the measure is intended to determine the effect of FXR. In general, a measured level can provide a way to track a biological process of interest; in this study, the registry record identifies C4 as the measure associated with bile salt metabolism and FXR signaling.
The endpoint is a biological measure rather than a reported clinical outcome such as symptoms or liver injury. The study summary gives the scientific context: abnormal FXR signaling may lead to liver injury in some people who drink heavily. That context explains the pathway of interest, but it does not establish that a change in C4 would correspond to a change in clinical outcomes. The record does not report the unit, measurement procedure, threshold for a meaningful change, or planned interpretation of the C4 result.
Most importantly, the primary outcome entry states that zero participants were analyzed because data were not collected. Thus the registry record contains no observed change in C4 for this endpoint. Its stated timeframe and intended purpose do not substitute for an outcome estimate.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record gives the study question and design, but it does not provide an analyzed primary outcome. The primary endpoint is change in C4 levels from baseline to 28 days, and the entry states that zero participants were analyzed because data were not collected. There is consequently no reported treatment-group estimate, measure of uncertainty, or statistical test for this endpoint. The record cannot show whether C4 changed, how large any change was, or whether groups differed.
The record lists 13 outcome measures and 0 analyses. Secondary outcomes include fasting serum bile salt levels, oxidative stress measured by malondialdehyde, CYP2E1 activity measured by chlorzoxazone clearance, gut permeability measured by a lactulose/mannitol test, bacterial translocation measures, intestinal inflammation measured by stool calprotectin, and innate immune activation measured by TNF-alpha. These are registered outcomes, not results. Their presence does not establish that they were measured, and their findings are not reported in the registry record.
There is also no reported estimate that would support an assessment of precision. A confidence interval, in general, describes a range of values compatible with the data and the specified statistical method. Without an analyzed estimate and its uncertainty, readers cannot judge whether the data would be compatible with small or large changes. No p-value or other inferential result for the primary endpoint is reported either.
Adverse-event counts are recorded separately. The registry lists 0 serious adverse events in each listed group, while other adverse events are recorded for the placebo and OCA groups and not for non-drinking controls. These counts are descriptive registry entries; they do not supply an estimate of the primary endpoint, and they do not resolve the absence of collected primary outcome data.
The status is completed, but that administrative status should not be interpreted as evidence that the primary endpoint was successfully measured. The registry's explicit statement about data collection governs interpretation of the primary result. No conclusion about the intervention's effect on FXR signaling or bile salt metabolism can be drawn from an endpoint with no analyzed participants.
9. Statistical Methods Explained
The design labels describe how the trial was organized, but the registry record does not give a detailed statistical analysis plan. The study is listed as randomized, quadruple-masked, and parallel. In general, randomization assigns participants to study groups using a chance process; its purpose is to help balance prognostic characteristics across groups rather than guarantee identical groups. Masking can reduce the influence of knowledge about assignment on participant behavior, care, or outcome assessment. The record does not name the roles covered by quadruple masking or explain the randomization procedure.
What would an endpoint comparison mean?
The primary endpoint is change in C4 levels from baseline to 28 days. In general, a change score compares a participant's measurement at follow-up with that participant's baseline measurement. A treatment comparison could then summarize the changes in the relevant study groups. Such an estimate needs measured observations and a defined analysis population. Here, the registry record says zero participants were analyzed because the data were not collected, so no endpoint comparison is available.
The record does not state whether a future or unreported analysis would compare final values, change scores, or an adjusted model. It also does not report the statistical test, covariates, treatment-effect scale, handling of repeated measurements, or rules for excluding observations. These details matter because different reasonable analysis choices can produce estimates with different interpretations and uncertainty.
How should uncertainty be understood?
In general, an estimate describes the observed effect in the analyzed data, while a confidence interval communicates the precision of that estimate under the chosen method. A narrow interval indicates more precision than a wide interval, but neither interval alone establishes clinical importance. A p-value, in general, assesses how compatible the data are with a specified null model under the test assumptions; it is not a measure of effect size or proof that a result is important. The registry record reports no primary estimate, confidence interval, or p-value, so those concepts cannot be applied to an observed primary result.
When data are sparse or highly variable, estimates may be imprecise. In general, over-dispersion means that observed variation exceeds what a model assumes; a model that ignores it can understate uncertainty. The registry record does not identify an outcome distribution or report evidence of over-dispersion. This is a general consideration, not a claim about these trial data.
What do the planning assumptions describe?
The record describes planning illustrations based on the observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The effect to detect is described as a stated fraction of the observed effect. For time-to-event settings, the stated planning quantity is the total number of events; for other settings, it is participants per arm. These are design-planning assumptions, not observed findings. A planning sample size is not observed power, and it cannot replace a reported analysis.
The primary outcome was not collected, and the registry record does not give a primary effect estimate. The assumptions alone therefore do not provide evidence about the treatment effect or the precision actually achieved. The record also says that the primary outcome has several categories or time points and was not reduced to one comparison. It does not identify a selected contrast or report calculations for a specific primary comparison.
What remains unspecified?
The registry record does not report an intention-to-treat definition, a per-protocol population, a missing-data method, an interim analysis plan, or a stopping rule. It does not say whether the trial used adjusted or unadjusted analyses for any registered outcome. No statistical method should be attributed to the investigators when it is not documented in the registry record. General explanations of randomization, masking, estimates, and uncertainty describe statistical concepts; they do not fill these missing protocol details.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 13 outcome measures, including the primary C4 endpoint and several secondary measures related to bile salts, oxidative stress, enzyme activity, gut permeability, bacterial translocation, intestinal inflammation, and innate immunity. When a study evaluates multiple outcomes or comparisons, general statistical practice must consider multiplicity: conducting many tests can increase the chance of obtaining at least one apparently unusual result by chance. Methods such as defining a primary endpoint in advance or adjusting inference can address particular multiplicity concerns, but the registry record does not report a multiplicity strategy.
The record lists 0 analyses. For the primary endpoint, it explicitly reports zero participants analyzed because the data were not collected. Results for the secondary endpoints are not reported in the registry record, and the entry does not explain whether their data were collected. No claims about secondary outcome effects or statistical significance are therefore supported by the record.
The analysis populations are not reported in the registry record. It is not stated whether analyses were intended to include all randomized participants, only participants who completed treatment, or another defined population. The record also does not describe how missing measurements would have been handled. Because the primary outcome was not collected, this is more fundamental than ordinary partial missingness: no primary endpoint participants were analyzed. The record gives no imputation method, sensitivity analysis, or explanation beyond the statement that data were not collected.
These omissions prevent assessment of how the investigators intended to control false-positive findings across outcomes, who would have been included in analyses, and how incomplete data might affect estimates. They do not justify assuming that an unreported method was or was not used.
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 |
|---|---|---|---|
| Placebo | 0 | 9 | 0.0% |
| 10 mg Obeticholic Acid (OCA) | 0 | 6 | 0.0% |
| Non-drinking Controls | 0 | 15 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Placebo | 0 of 9 |
| 10 mg Obeticholic Acid (OCA) | 0 of 6 |
| Non-drinking Controls | 0 of 15 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Placebo | 4 of 9 |
| 10 mg Obeticholic Acid (OCA) | 3 of 6 |
| Non-drinking Controls | 0 of 15 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Placebo | 10 mg Obeticholic Acid (OCA) | Non-drinking Controls |
|---|---|---|---|---|
| Headache | Nervous system disorders | 1 of 9 | 2 of 6 | 0 of 15 |
| Nausea | Gastrointestinal disorders | 1 of 9 | 1 of 6 | 0 of 15 |
| Upper respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | 0 of 9 | 1 of 6 | 0 of 15 |
| Burns first degree | Injury, poisoning and procedural complications | 0 of 9 | 1 of 6 | 0 of 15 |
| Diarrhea | Gastrointestinal disorders | 1 of 9 | 0 of 6 | 0 of 15 |
| Thirst decreased | General disorders | 1 of 9 | 0 of 6 | 0 of 15 |
| Gastrointestinal pain | Gastrointestinal disorders | 1 of 9 | 0 of 6 | 0 of 15 |
| Aspartate aminotransferase increased | Investigations | 1 of 9 | 0 of 6 | 0 of 15 |
| Alanine aminotransferase increased | Investigations | 1 of 9 | 0 of 6 | 0 of 15 |
| throat irritation | Respiratory, thoracic and mediastinal disorders | 1 of 9 | 0 of 6 | 0 of 15 |
The registry record lists serious adverse events separately from other adverse events and deaths. For Placebo, 0 serious adverse events are recorded among 9 at risk, and 4 other adverse events are recorded among 9 at risk; deaths are recorded as 0. For 10 mg Obeticholic Acid (OCA), 0 serious adverse events are recorded among 6 at risk, and 3 other adverse events are recorded among 6 at risk; deaths are recorded as 0. For Non-drinking Controls, 0 serious adverse events are recorded among 15 at risk, and 0 other adverse events are recorded among 15 at risk; deaths are recorded as 0.
These are counts as entered in the registry, not comparative risk estimates. They do not establish whether the groups differed in safety, and they should not be interpreted as proof that no events could occur. The record does not provide event descriptions for the listed other adverse events, severity details, timing, attribution, or a statistical comparison. The absence of listed serious-event terms does not add detail beyond the recorded counts. Safety counts also do not answer the primary question about change in C4 or FXR signaling.
12. What the Result Does, and Does Not, Mean
The record documents a completed randomized, quadruple-masked, parallel study concerning heavy alcohol consumption and FXR signaling. It identifies placebo and 10 mg Obeticholic Acid (OCA) as interventions and also describes non-drinking healthy controls. It states that the primary endpoint was change in bile salt metabolism, assessed through C4 levels from baseline to 28 days.
What the record does not establish is the primary outcome result. The primary outcome entry says zero participants were analyzed because data were not collected. No effect estimate, confidence interval, or statistical test is reported. Therefore, the record cannot quantify a change in C4 or indicate whether the intervention altered the endpoint. It also cannot establish that the hypothesized pathway was changed or that any pathway change affected liver injury.
The registry lists secondary outcome measures, but their results are not reported in the registry record. Those entries show what outcomes were of interest, not what happened to them. The adverse-event entries provide counts, but they are not a substitute for the missing endpoint analysis and lack the detail needed for a full comparative assessment.
In general, a completed study may still have outcomes that were not collected or analyzed. Completion status describes the registry's study status; it is not a result. Likewise, randomization and masking describe design features, not evidence of an effect. The registry record supports a description of the intended question, design, and recorded safety counts, while leaving the central efficacy question unanswered.
The scope of interpretation is therefore limited to what is documented. It is not possible to infer benefit, lack of benefit, or equivalence from an endpoint with no analyzed observations. Nor should missing results be treated as evidence that the groups had the same outcomes.
13. Why This Trial Matters Statistically
This trial matters statistically because its registered design and its available primary outcome record do not align in a way that permits an effect estimate. Randomization and masking can strengthen a comparison when outcomes are collected and analyzed according to a clear plan. They cannot compensate for the absence of measurements on the primary endpoint. The explicit note that zero participants were analyzed because data were not collected is central to interpreting the registry entry.
The study also illustrates why a registered outcome is distinct from a reported result. A detailed endpoint title and timeframe communicate what investigators intended to assess, but they do not provide the estimate, uncertainty, or analysis needed to evaluate the result. The record lists 13 outcome measures and 0 analyses, so the existence of multiple planned measures should not be mistaken for a set of findings.
Statistically, a missing primary result prevents examination of both effect size and precision. Readers cannot determine whether the endpoint would have shown a small or large change, or whether any estimate would have been precise. General planning assumptions cannot answer those questions: planning calculations concern hypothetical design performance and are not observed power or outcome evidence.
The entry is also a reminder that design features and administrative status must be interpreted separately from outcome data. The trial is marked completed and described as randomized and quadruple-masked, but these registry entries do not establish what happened to the primary measure. Accurate interpretation requires keeping the study question, the planned endpoint, the collected data, and the analyzed result distinct.
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.
Important design and reporting details are not reported in the registry record. Eligibility criteria and the definition of heavy drinking are not given, so the population represented by the heavy-drinker groups cannot be characterized beyond the wording of the arm descriptions. Recruitment setting, allocation ratio, and the method used to generate and conceal assignments are also not reported. The record identifies quadruple masking without specifying who was masked.
The role of the non-drinking healthy controls is not fully explained. The registry describes them as a group, but does not clarify their relationship to the randomized placebo and OCA comparison or state which contrasts were planned. The placebo schedule is not detailed separately, and the record does not explain adherence assessment or treatment discontinuation.
Measurement and analysis details are limited. The endpoint unit and measurement procedure are not reported, nor are the target effect, statistical model, covariate adjustment, analysis population, or missing-data strategy. The primary outcome entry states that zero participants were analyzed because data were not collected. Results for the registered secondary outcomes are not reported in the registry record, and the reason those results are unavailable is not specified.
The registry does record serious and other adverse-event counts, but it does not provide event-level descriptions, timing, severity, attribution, or comparative analyses. These limitations restrict both efficacy interpretation and detailed assessment of safety. They should be distinguished from evidence of no effect or no risk: the record leaves those questions unresolved rather than answering them.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Effect of Heavy Alcohol Consumption on Farnesoid X Receptor (FXR) Signaling — NCT02654236.
- Registry record last updated 2023-04-25; status last verified 2023-04.
- Results first posted 2023-04-25.
- 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
The registry record identifies NCT02654236 as a completed, randomized, quadruple-masked, parallel study titled “Effect of Heavy Alcohol Consumption on Farnesoid X Receptor (FXR) Signaling.” Enrollment is listed as 30. The recorded start is 2016-04, and primary completion is 2019-09.
The interventions are placebo and 10 mg Obeticholic Acid (OCA), described as oral once-daily treatment for 4 weeks. Non-drinking healthy controls are also listed. The primary endpoint concerns change in bile salt metabolism using C4 levels from baseline to 28 days. The registry states that zero participants were analyzed for this endpoint because data were not collected. It lists 13 outcome measures and 0 analyses.
Serious adverse-event counts are recorded as 0 in each group. Other adverse events are recorded as 4 for Placebo, 3 for 10 mg Obeticholic Acid (OCA), and 0 for Non-drinking Controls. These counts do not supply the missing primary endpoint result. The registry record therefore documents the study design and selected safety counts but does not report an analyzed primary efficacy outcome.