Safety and Efficacy of Basiliximab, Delayed Dose Tacrolimus Plus ECMPA (Enteric Coated Mycophenolic Acid) Following Liver Transplantation: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Safety and Efficacy of Basiliximab, Delayed Dose Tacrolimus Plus ECMPA (Enteric Coated Mycophenolic Acid) Following Liver Transplantation (NCT02123108), a randomized phase 4 study. The primary outcome is Renal Recovery/ Function.

2. Clinical Question

3. Trial Design

The registry identifies this as a Phase 4, randomized, parallel-group study with no masking. Its title concerns basiliximab, delayed-dose tacrolimus, and enteric-coated mycophenolic acid following liver transplantation, compared with standard-dose tacrolimus, enteric-coated mycophenolic acid, and corticosteroids. The arm descriptions distinguish tacrolimus with basiliximab induction from a tacrolimus group without basiliximab induction. The brief summary describes a reduced tacrolimus dose after kidney function has normalized, but the detailed treatment schedule and how that description maps to the outcome entries are not reported in the registry record.

The registry lists enrollment as 59. For the primary endpoint, it lists 28 participants in the Basiliximab arm and 26 in the Tacrolimus Group. Enrollment and the arm counts are distinct registry entries; the record does not explain the relationship between them, the disposition of participants, or which participants were included in the endpoint analysis. It also does not report whether all randomized participants were analyzed in their assigned groups.

The sponsor is the University of California, Los Angeles. The record describes the study as investigator initiated at UCLA and funded by Novartis. The start is listed as 2011-01, primary completion as 2015-01, and status as completed. These registry entries establish the broad design and setting, but they do not replace a full protocol or analysis plan. Allocation procedures beyond the designation randomized, concealment procedures, treatment adherence, and co-interventions are not reported in the registry record.

Randomization is intended to support a fair comparison by assigning participants to study groups, while a parallel design follows separate groups receiving the respective regimens. With no masking, participants and study personnel were not masked according to the registry designation. That feature can matter for outcomes involving clinical judgment or care decisions, but the record does not say how endpoint determination was standardized or whether outcome assessors were masked. The trial reports a kidney-related endpoint at 12 months post-transplant; the available entries do not describe additional follow-up or the complete schedule of assessments.

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

Reasons for not completing this period:

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.

6. Primary Endpoint

The registry lists the primary outcome as Renal Recovery/ Function, measured over 12 months post-transplant.

The primary endpoint is titled Renal Recovery/ Function and is measured as a count of participants. Its description includes participants who experienced dialysis independence or improvement based on kidney-function laboratory results after orthotopic liver transplant. The stated timeframe is 12 months post-transplant. The endpoint therefore combines a clinical status—dialysis independence—with improvement based on laboratory measures. The registry record does not specify the laboratory measures, thresholds for improvement, whether either criterion alone was sufficient, or how conflicting measurements were handled.

A count of participants meeting an endpoint can be used to compare the occurrence of a defined outcome between randomized groups. In this study, the outcome is intended to describe renal recovery or function, an issue emphasized in the brief summary. The usefulness of that comparison depends on a clear and consistently applied outcome definition, complete and comparable ascertainment, and an analysis that accounts for the randomized design. Those operational details are not reported in the registry record.

The posted values are 28 participants for Basiliximab and 26 for the Tacrolimus Group, with listed arm sizes of 28 and 26, respectively. The registry labels the result as a count of participants and does not give a separate laboratory-based summary or a breakdown by the endpoint's two stated routes. It is therefore not possible to tell from these entries how many participants met the dialysis-independence criterion, how many met the laboratory-improvement criterion, or whether both contributed for any participant. The endpoint should be read as the registry's combined outcome definition, not as a detailed account of kidney-function trajectories.

7. Results Posted in the Registry

• Tacrolimus Group: 100.0% (26/26) versus 100.0% (28/28) on control; risk difference 0.0 percentage points (95% CI 0.0 to 0.0), risk ratio 1.00 (1.00–1.00); unadjusted two-proportion z-test p = n/a.

8. Statistical Interpretation

9. Statistical Methods Explained

The comparison is between two randomized parallel groups. For a binary endpoint expressed as participants with an event, a group proportion describes the share of participants meeting the endpoint among those included in the analysis. A risk difference compares those proportions by subtracting one from the other. A risk difference of 0.0 means the posted proportions are equal on this scale. The registry also reports relative-risk values of 1.0, which likewise indicate equal proportions in the posted comparison. These are descriptions of the recorded endpoint, not proof that the underlying treatment effects are identical.

A confidence interval describes statistical uncertainty around an estimate under a specified method and its assumptions. Its width is often informative: a wider interval permits a broader range of values, while a narrower interval permits a more restricted range. In the posted result, the interval endpoints are both 0.0. The registry does not name the interval method or explain how this interval was produced. Because the interval has no displayed width despite the finite arm counts, its interpretation deserves particular caution. The record does not indicate whether rounding, a calculation convention, or another issue accounts for this display.

A p-value measures how compatible the observed data are with a null hypothesis under a statistical model. It is not the probability that the null hypothesis is true, nor does it measure clinical importance. The registry reports p=1.0 for the comparison. This agrees with the equal posted proportions under the reported calculation, but the record does not identify the test, its assumptions, or whether it was adjusted for any other factors. A p-value alone cannot establish equivalence, absence of meaningful benefit or harm, or the reliability of outcome ascertainment.

Random allocation can reduce systematic differences between groups at the start of a trial, on average. It does not automatically address incomplete follow-up, deviations from assigned treatment, outcome measurement choices, or selective reporting. The registry labels allocation as randomized, but the sequence-generation and allocation-concealment procedures are not reported in the registry record. Masking is listed as none. The consequences of this design feature depend partly on how the endpoint was defined and measured; the registry does not describe assessor masking or safeguards against differences in clinical assessment.

What does the endpoint count represent?

The endpoint is a participant count combining dialysis independence or improvement based on kidney-function laboratory results. A binary analysis treats each participant as meeting or not meeting the defined outcome. That simplifies the comparison, but it can hide differences in the degree or timing of recovery, as well as variation in laboratory values. The record does not specify a threshold for laboratory improvement, whether dialysis independence and laboratory improvement were handled as alternatives, or how repeated measurements informed the classification. Without these details, the endpoint's reproducibility cannot be assessed from the registry entry alone.

The registry lists 28 participants and a value of 28 for Basiliximab, and 26 participants and a value of 26 for the Tacrolimus Group. These entries support the reported equality of proportions. They do not explain how the endpoint classifications were made, whether the values refer to every randomized participant, or how the listed enrollment of 59 relates to the arm counts. No outcome-level accounting of missing observations is reported.

How should the planning assumptions be read?

The record describes a planning illustration based on the observed effect, with two-sided significance level 0.05, equal allocation, and a normal approximation. It describes the effect to detect as a stated fraction of the observed effect. Such an illustration asks what sample size might be required under selected assumptions; it is not an estimate of the trial's actual probability of detecting an effect and must not be treated as observed power. No planning sample-size result or design justification is reported in the registry record. The record also says time-to-event planning summaries use total events, while other planning summaries use participants per arm. The primary endpoint is a participant count, not a time-to-event measure.

Planning calculations depend on the event definition, anticipated effect, variability or event frequency, allocation, significance level, and desired power. If the observed effect is exactly zero, planning for a fraction of that effect has no straightforward useful interpretation. The posted record does not provide a separate target effect or an independent design assumption that would clarify the illustration. The sample-size material should therefore be read as a conditional planning exercise, not as evidence about the adequacy of enrollment or the trial's ability to detect a clinically important difference.

What cannot be confirmed?

The registry lists zero analyses and does not describe a statistical analysis plan, adjustment covariates, model specification, handling of multiplicity, or analysis populations. It does not report whether the primary analysis followed intention-to-treat principles, was restricted to participants with observed outcomes, or used another approach. These choices can affect interpretation, particularly when endpoint information is unavailable or treatment differs from assignment. No method for missing data is stated.

The registry's posted comparison should consequently be treated as a brief result rather than a full statistical report. Details needed to reproduce the result—including the test and confidence-interval method—are not reported in the registry record. The displayed zero difference and p=1.0 are the sponsor's posted analysis values. When a sponsor analysis and a simple recalculation from summary statistics differ, the sponsor's analysis takes precedence; the record does not flag a discrepancy. The central methodological point remains that the reported estimate is precisely equal at the displayed scale, while the completeness and uncertainty of the underlying analysis cannot be fully evaluated from the registry entries.

10. Confidence Intervals and Precision

The reported risk difference is 0.0, and the confidence interval is 0.0 to 0.0. The point estimate says the posted proportions are equal; the interval, as written, also places both limits at that same value. Ordinarily, an interval communicates a range of estimates compatible with the data and method. A zero-width interval does not display the uncertainty readers would usually expect from a comparison based on finite participant counts. The registry does not explain the calculation method or whether rounding or a reporting convention is responsible.

The arm entries list 28 participants and 28 endpoint events in Basiliximab, and 26 participants and 26 endpoint events in the Tacrolimus Group. The posted proportions are consequently equal, as reflected by the risk difference of 0.0 and relative-risk values of 1.0. Equality of the observed proportions does not make the estimate precise in the sense of excluding plausible differences. Precision depends on the statistical method and the information available, not solely on whether the observed values match.

The p-value is 1.0. It is consistent with the equality of the posted proportions, but does not resolve the interval's unusual display or establish equivalence. The registry does not report a clinically acceptable difference, a noninferiority or equivalence margin, or a precision target. Nor does it provide component-specific outcome counts, missingness information, or a detailed analysis population. These omissions make it difficult to judge how much uncertainty is present beyond the displayed interval.

The most defensible reading is therefore limited: the registry reports a point estimate of no difference for its combined endpoint and an interval with identical displayed endpoints. The record does not provide enough methodological detail to determine whether that interval fully represents uncertainty. The result should not be described as demonstrating that the regimens have the same effect.

11. Multiplicity, Missing Data and Analysis Populations

The registry lists five outcome measures, zero posted analyses, and zero secondary outcomes as posted. It identifies Renal Recovery/ Function as the primary outcome, but does not provide a statistical analysis plan explaining how the primary outcome was selected, how other outcomes were analyzed, or whether any comparisons were planned beyond the posted primary comparison. The record does not report a multiplicity strategy. If several outcomes or analyses are examined, the chance of a nominally unusual result can change; a prespecified approach helps readers interpret such findings. The available entries do not permit an assessment of that issue.

Missing outcome data are also not described. The record does not report how many participants had an unavailable endpoint, why information might be missing, or what method was used to address it. The primary endpoint arm counts are 28 and 26, while enrollment is listed as 59; the record does not explain the relationship between enrollment and those endpoint counts. It would be inappropriate to infer attrition, exclusions, or missingness from those entries alone.

Analysis populations are not stated. The record does not say whether all randomized participants were analyzed according to their assigned group, whether analysis was limited to participants with observed outcomes, or whether another population was used. These distinctions matter because exclusions after randomization can affect the comparability that random assignment is intended to support. The registry also does not report how protocol deviations or treatment discontinuations were handled.

Without a protocol or statistical analysis plan in the record, it is not possible to determine whether the outcome, time point, and analysis were prespecified in the form reported, or whether additional planned analyses were omitted. These are gaps in the registry record, not evidence that any particular practice occurred. The result is best interpreted as the posted primary comparison, with uncertainty about multiplicity, missing data, and analysis population left unresolved.

12. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent serious adverse event terms

Serious events with the highest arm-level rate: death (10.3%).

The registry reports serious adverse event figures separately for the two groups. In Basiliximab, 30 participants were listed as at risk for serious events and 2 as affected; in the Tacrolimus Group, 29 were listed as at risk and 3 as affected. For other adverse events, the at-risk figures are 30 and 29, with 0 affected in each group. The record lists deaths of 2 and 3, respectively. It also lists Death among the top serious terms at 10.3%.

These are descriptive registry entries, not evidence that a treatment caused or prevented an event. The record does not describe the event definitions, observation period for safety, severity grading, relatedness assessment, or whether individual participants could contribute to more than one event category. Deaths are reported as counts, but the registry entry does not establish their timing or relationship to study treatment. The 10.3% figure should be read as a listed serious-term summary, not as a comparative treatment effect.

The at-risk counts differ between the groups, and the registry does not explain how those safety populations relate to enrollment or to the primary endpoint populations. No adjusted comparison or uncertainty interval for safety is reported. Small descriptive counts cannot, by themselves, establish a reliable difference in risk. The serious adverse event entries should therefore be read as a record of reported counts with important context absent, rather than as a definitive comparative safety analysis.

13. What the Result Does, and Does Not, Mean

The result establishes that the registry records equal proportions for the primary Renal Recovery/ Function outcome: 28 participants in Basiliximab and 26 in the Tacrolimus Group, with matching listed arm sizes. The reported risk difference is 0.0, its confidence interval is 0.0 to 0.0, and p=1.0. These statistics summarize the outcome as entered in the registry. They do not answer whether the regimens are equivalent, whether one is preferable, or how kidney function changed over time.

The endpoint is a combined classification involving dialysis independence or improvement based on kidney-function laboratory results. Because no component counts, laboratory criteria, or trajectories are reported, the posted values do not show which aspect of the definition accounted for each classification. A single endpoint assessment at 12 months post-transplant also does not describe the complete course of renal function before that time or after it.

The registry record does not supply the analysis method, missing-data approach, participant flow, or analysis population. It lists enrollment as 59, but gives endpoint arm sizes of 28 and 26 without explaining the relationship. The confidence interval has identical limits, and its construction is not described. These uncertainties limit how strongly the numerical comparison can be interpreted.

Nor do the reported figures establish that either treatment strategy improves kidney outcomes or that either fails to do so. A result with no observed difference can arise for different reasons, including genuinely similar outcomes, limitations in measurement, or incomplete information; the registry entries do not distinguish among these possibilities. No clinical recommendation follows from the posted statistics. The appropriate interpretation is narrow and descriptive: identical observed proportions are recorded for this combined primary outcome, while important details needed to assess precision, completeness, and clinical meaning are not reported.

14. Why This Trial Matters Statistically

This trial raises a meaningful statistical question: how should a randomized comparison be interpreted when the posted binary outcome has identical proportions in the groups, yet the registry provides little detail about how the result was calculated? The risk difference of 0.0 and p=1.0 are easy to read at face value. The confidence interval from 0.0 to 0.0, however, does not display the uncertainty readers ordinarily need to assess how much difference remains compatible with the data. The contrast between a simple point estimate and limited methodological reporting is an important lesson in registry interpretation.

The endpoint also illustrates how a combined outcome can compress distinct clinical information. Dialysis independence and laboratory-based improvement are both included in the definition, but the registry does not report the component counts or laboratory criteria. A single participant count may be clear as a headline measure while still leaving readers unable to understand what kinds of recovery it represents. Statistical interpretation depends on the operational definition as well as the calculation.

Sample-size planning is another useful distinction. A planning illustration based on an observed effect and stated assumptions is not evidence of observed power or proof that the trial was adequately sized. The registry does not report a target sample-size justification or numerical planning result. Keeping design assumptions separate from observed results prevents a conditional planning exercise from being mistaken for a finding about the trial.

Finally, the registry lists 59 enrolled participants, arm-level primary outcome counts, safety counts, and no posted analyses. These entries demonstrate why transparent participant flow, analysis populations, missing-data methods, and prespecified statistical plans matter. Without them, readers can describe the recorded estimate but cannot fully assess its robustness. The statistical value of the trial record lies not only in its point estimate, but also in recognizing what the record leaves unresolved.

15. 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.

Several limitations specific to this registry record constrain interpretation. The primary endpoint combines dialysis independence with improvement based on kidney-function laboratory results, but the criteria for either component are not described. There are no component-specific counts, laboratory values, thresholds, or explanation of how repeated measurements were handled. This prevents readers from determining what the combined outcome represents in practice.

The registry reports enrollment of 59, while the primary endpoint arm entries list 28 and 26 participants. It does not explain the relationship between these figures, provide participant flow, or state whether the endpoint counts include all randomized participants. No missing-data or exclusion information is reported. These gaps make it difficult to assess the completeness of endpoint ascertainment or the population to which the result applies.

The risk difference is 0.0 with a confidence interval from 0.0 to 0.0, but the registry does not name the method used to calculate the interval or explain its zero width. It also does not identify the statistical test underlying p=1.0. The analysis plan, adjustment strategy, and handling of multiple outcomes are not reported. The registry lists five outcome measures but zero analyses and zero secondary outcomes as posted.

The study is labeled randomized and unmasked, but sequence generation, allocation concealment, endpoint-assessor masking, adherence, and treatment discontinuations are not described. The arm descriptions and study title identify regimens broadly, while detailed timing and implementation are not available in the outcome entries. Safety information is limited to counts and does not include event definitions, exposure time, relatedness, or comparative uncertainty. These are limitations of the available record and should not be treated as evidence about procedures or events that it does not describe.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Safety and Efficacy of Basiliximab, Delayed Dose Tacrolimus Plus ECMPA (Enteric Coated Mycophenolic Acid) Following Liver Transplantation — NCT02123108.
• Registry record last updated 2022-09-29; status last verified 2022-09.
• Results first posted 2022-09-29.
• 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.

19. Record Summary

The registry record identifies NCT02123108 as a completed Phase 4 randomized, parallel-group study with no masking, sponsored by the University of California, Los Angeles. Enrollment is listed as 59; the study start is 2011-01 and primary completion is 2015-01. The comparison is between tacrolimus with basiliximab induction and a tacrolimus group without basiliximab induction, described as standard of care.

The primary endpoint is Renal Recovery/ Function, measured as participants with dialysis independence or laboratory-based improvement at 12 months post-transplant. Posted counts are 28 and 26, with a risk difference of 0.0, confidence interval 0.0 to 0.0, and p=1.0. The registry also lists serious adverse event counts, but no posted analyses or secondary outcomes. The endpoint criteria, analysis population, missing-data handling, and statistical methods are not reported in the registry record.