Evaluation of Calcineurin-inhibitor Reduction With Conversion at 2 Months to Everolimus/Reduced Tacrolimus in Renal Transplant Recipients Following Campath® Induction: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Evaluation of Calcineurin-inhibitor Reduction With Conversion at 2 Months to Everolimus/Reduced Tacrolimus in Renal Transplant Recipients Following Campath® Induction (NCT01935128), a na phase 4 study. The primary outcome is Renal Function.

2. Clinical Question

These details define the clinical question as a study of a treatment strategy in renal transplant recipients, rather than a direct estimate of how that strategy compares with a separate control regimen. The registry describes a pilot study at a single center. Its enrollment is 55, but the record does not report how many participants contributed data to the primary endpoint at the stated time point. Nor does it report participant-level clinical characteristics that would help readers judge how closely the study population resembles other transplant populations.

The study aim and its endpoint are related but not identical: the aim mentions nephrotoxicity and graft survival, while the primary endpoint is an estimated measure of renal function. The endpoint can describe kidney function at the specified assessment, but the record does not establish a comparative effect on graft survival. That distinction matters when interpreting the clinical question and the evidence actually posted.

3. Trial Design

The registry describes a Phase 4, single-center, pilot, open-label study. Its design fields identify a single-group model, allocation as NA, and no masking. Enrollment is listed as 55. The study status is completed, with a start date of 2013-07-03 and primary completion recorded as 2020-07. The official title calls the study a 24-month controlled trial, but the registry design fields do not list a separate control arm or randomized allocation.

The intervention arm is named Everolimus/Reduced dose tacrolimus. According to the arm description, myfortic® is weaned off quickly, everolimus is initiated toward the stated target level, and tacrolimus is reduced after everolimus reaches a therapeutic dose. These are descriptions of the planned treatment strategy; they are not evidence that every participant received or maintained the stated doses or levels. Details about adherence, changes to treatment, and actual exposure are not reported in the registry record.

The primary outcome is renal function at 2 years. The registry specifies a mean and standard deviation for this continuous measure, and identifies the intent-to-treat population with last observation carried forward imputation. Secondary outcomes listed include impaired glucose tolerance, proteinuria, lipid levels, mouth ulcers, gastrointestinal complaints, leukopenia, thrombocytopenia, and neurotoxicity. The record also lists 20 outcome measures, but outcome-specific results are not reported.

The absence of a listed comparator shapes what the design can answer. A single-group study may describe outcomes among participants who received the strategy, but without a concurrent comparison arm it cannot by itself separate treatment-associated changes from other explanations, such as changes over time or differences in participants’ clinical circumstances. The registry record does not report a protocol-level explanation for the design, the planned sample-size justification, or further details of the analysis plan.

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: 50 participants met the inclusion/exclusion criteria and were enrolled in the treatment arm.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Intent to treat population (all participants assigned to treatment arm). Last observation carried forward (LOCF) imputation method

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 Function, measured over 2 years.

The primary endpoint is renal function at 2 years, measured as estimated glomerular filtration rate using the Modified Diet Renal Disease estimation. The unit is mL/min/1.73 m2, and the registry specifies that the result is summarized as a mean with standard deviation. GFR is an estimate of how effectively the kidneys filter blood; it is used as a measure of remaining kidney function. The registry’s description explains that the estimate uses a mathematical formula involving serum creatinine and personal characteristics.

A continuous kidney-function measure can capture variation across participants rather than reducing the assessment to a simple event category. In general, a mean summarizes the central level of a continuous measure, while a standard deviation describes how dispersed individual measurements are around that mean. Neither summary alone shows the full distribution of participant values or how much uncertainty surrounds the estimated mean. A confidence interval, if reported, would help convey uncertainty in the estimate; no primary endpoint confidence interval is reported in the registry record.

The endpoint is relevant to the stated study aim because the brief summary frames increased creatinine clearance as a way to assess reduced nephrotoxicity. However, the primary outcome specifically recorded is MDRD-estimated GFR. The registry record does not report an observed mean, standard deviation, change from an earlier assessment, or comparison with another treatment group. It therefore describes how renal function was to be assessed, but does not show the endpoint result at 2 years.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The central statistical feature of this record is that the registered primary endpoint is continuous. It is estimated GFR, summarized as a mean and standard deviation at 2 years. In general, continuous outcomes can take a range of values, so analysts often report a measure of central tendency and a measure of variability. The mean describes the average level in the analyzed group. The standard deviation describes how much individual observations vary around that mean. Neither should be confused with a treatment effect: a treatment effect requires a defined contrast, such as a difference between groups or a change from a baseline value.

What does the endpoint summary tell us?

A mean is useful for a concise description, but it does not show whether observations are clustered near the mean or whether the distribution has a long tail. The standard deviation gives information about spread, but not the precision of the estimated mean. In general, a confidence interval around a mean or a contrast gives a range of values compatible with the data under the analysis assumptions. Its width depends on the amount of information and variability, among other factors. The registry record does not report a confidence interval or the primary endpoint mean and standard deviation, so the precision of the renal-function estimate cannot be assessed from these entries.

For a comparative treatment question, an analysis would also need to define the contrast being estimated and how it is calculated. A randomized comparison can help distinguish a treatment-group difference from other causes of differing outcomes, subject to the trial’s conduct and analysis. Here, the record lists a single-group model and no separate comparator arm. It therefore does not report a randomized contrast. A value from the intervention group alone, even if posted, would describe that group; it would not by itself establish what would have happened under another treatment strategy.

Why does design affect interpretation?

Allocation describes how participants are assigned to interventions, and masking describes whether participants or study personnel are kept unaware of treatment assignment. The record lists allocation as NA and masking as none. In general, random allocation can reduce systematic differences between groups at the start of a trial, while masking can reduce some influences on treatment delivery or outcome assessment. These concepts do not guarantee unbiased results, and their relevance depends on the particular design and outcome. Because the registry describes a single group and no masking, readers should not interpret this study as a blinded randomized comparison.

The official title calls the study controlled, while the design fields list a single-group model and the intervention listing contains one arm. Those descriptions are not enough to infer an unlisted control group. The registry record does not report a comparator regimen, allocation procedure, or a statistical comparison with a control group. For interpretation, the design fields and listed arms are the available basis for describing the posted evidence.

What does last observation carried forward mean?

The primary endpoint population is described as the intent-to-treat population, with last observation carried forward imputation. In general, an intent-to-treat approach aims to analyze participants according to their assigned treatment group, regardless of subsequent treatment changes, when the design includes assigned groups. This record describes a single arm and does not provide further detail about how the population definition was applied. Last observation carried forward is a method for filling a missing later measurement using an earlier observed value. It can preserve a participant in an analysis, but it treats the earlier value as if it represented the later assessment. That assumption may be unrealistic when the outcome can change over time. The record does not report the amount or pattern of missing primary endpoint data, the timing of carried-forward measurements, or sensitivity analyses addressing missing data.

Imputation is not the same as observing the endpoint. An analysis using carried-forward values combines measured observations with values substituted under an assumption. In general, the credibility of an imputed analysis depends on why data are missing and whether the imputation assumptions are reasonable. The registry record’s identification of LOCF does not tell readers how many values were imputed or how much the reported outcome, if available, would depend on that choice.

How should the sample-size illustrations be read?

The sample-size section contains planning illustrations rather than a reconstruction of the trial’s enrollment decision. The stated assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the target effect expressed as a fraction of the observed effect. In general, sample-size planning asks how much information may be needed to detect a defined effect under specified assumptions. Such a calculation is sensitive to the assumed effect, variability, design, and analysis method. If the assumed effect is based on an observed estimate, uncertainty in that estimate also matters.

These illustrations are not observed power. Observed power is not a substitute for reporting an effect estimate and its uncertainty, and a planned sample-size calculation does not show what the completed study found. The two-arm assumptions also do not match the registry’s listed single-group model. The registry record does not report the trial’s own sample-size justification, so the illustrations should not be read as the protocol’s original rationale or as proof that the completed study had a particular level of precision.

For continuous outcomes, general analyses may use models that account for baseline measurements or repeated observations, but no such model is specified in the registry record. It does not report whether the primary analysis used an endpoint value, a change from baseline, repeated measurements, covariate adjustment, or another approach. Those choices can affect the estimate and its uncertainty. They should not be inferred from the endpoint description alone.

10. Confidence Intervals for Each Arm

No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.

R code and output

arm <- c("Arm 1 Everolimus/Reduced Dose Tacrolimus")
n   <- c(50)
m   <- c(65.7)
sp  <- c(20.04)
sd  <- sp      # posted standard deviations
for (i in seq_along(n)) {
  se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
  cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}

CARM  Arm 1 Everolimus/Reduced Dose Tacrolimus  50  65.700  20.040  60.005  71.395

11. Multiplicity, Missing Data and Analysis Populations

The registry lists secondary outcomes spanning metabolic, renal, gastrointestinal, blood, and neurologic measures, and reports 20 outcome measures in total. When a study examines multiple outcomes, there are multiple opportunities for apparently notable findings to arise by chance. In general, multiplicity methods can control or otherwise address that issue, but the appropriate approach depends on which outcomes are confirmatory, how they are related, and the analysis plan. The registry record does not report a multiplicity adjustment, a hierarchy of outcomes, or which secondary outcomes were designated as confirmatory.

The record identifies the primary endpoint and its planned summary, but does not report its result or the results of the listed secondary outcomes. It also lists zero analysis entries. This means that the posted registry information does not offer an analysis-level account of estimates, uncertainty, or statistical tests. It does not establish whether analyses were conducted elsewhere or whether any particular outcome was omitted; those details are not reported in the registry record.

The stated analysis population is intent to treat, with last observation carried forward for the primary endpoint. In general, this approach attempts to retain participants in the analysis while using an earlier measurement in place of a missing later one. The registry record does not report the number of participants with missing endpoint data, the reasons for missingness, the amount of follow-up available, or any sensitivity analysis. Without these details, readers cannot judge how the missing-data method may have influenced an endpoint estimate.

Enrollment is listed as 55, while safety denominators are listed separately for the reported arm. The registry record does not explain the relationship between those denominators and enrollment or provide the number contributing to the primary endpoint. These counts should therefore not be treated as interchangeable. Nor should the total of listed outcomes be interpreted as evidence that all outcome data were complete or analyzed.

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 4% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: venous thrombosis (8.0%), pyrexia (8.0%), dyspnea (6.0%), nephrectomy (4.0%), influenza a (2.0%).

The safety section lists the arm as Everolimus/Reduced Dose Tacrolimus. It reports serious adverse events among 18 of 50 participants at risk, other adverse events among 50 of 50 at risk, and 1 death. The record lists venous thrombosis and pyrexia at 8.0% each, dyspnea at 6.0%, nephrectomy at 4.0%, and influenza A and gastroenteritis at 2.0% each as the top serious-event terms.

These figures are descriptive registry reports. The “at risk” denominators identify the numbers used for the event summaries, but the record does not explain their relationship to the enrollment figure. The listed terms and percentages do not, by themselves, show whether events occurred in distinct participants or whether a participant experienced more than one event. The registry record also does not report event timing, duration, outcome, or a comparison group’s safety data in these entries.

Serious adverse events and other adverse events are separate categories in the record; the counts should not be merged. A reported event after treatment is not automatically caused by treatment. Without a suitable comparator and further clinical detail, these figures cannot establish whether the regimen changed the likelihood of an event relative to another strategy. They should be read as the safety information posted for this arm, not as a comparative risk estimate.

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

This record documents a study of an everolimus and reduced-dose tacrolimus strategy in adult renal transplant recipients following Campath® induction and steroid avoidance. It identifies estimated GFR at 2 years as the primary endpoint and lists several safety and secondary outcomes. It also provides serious and other adverse-event counts for the listed arm. These registry entries describe the study question and the information recorded in the registry.

The record does not report the primary endpoint mean or standard deviation, an estimate of change in renal function, or a confidence interval. It therefore does not show how renal function was observed at the primary assessment or how precisely it was estimated. No primary endpoint result should be inferred from the study’s completion status, enrollment, or treatment description.

The design information does not support a randomized comparison. The model is single group, allocation is NA, and no separate comparator arm is listed. Even if a within-group outcome were reported, change within a group would not isolate the effect of treatment from other influences without a suitable comparison or other justified design and analysis. The title’s reference to a controlled trial does not establish a control group in the posted design details.

The safety counts do not establish that the intervention caused the reported events, and the record does not give a parallel regimen’s event counts for comparison. They are useful as a description of what was reported for the arm, but cannot answer a comparative safety question on their own. Likewise, the list of secondary outcomes does not constitute evidence about those outcomes when corresponding results are absent.

In general, a clinical trial result is most informative when readers can see the outcome estimate, its uncertainty, the population analyzed, missing-data handling, and the comparison that supports the inference. Several of these elements are not reported in the registry record. The defensible interpretation is consequently limited: the record describes a completed, single-group study and its planned kidney-function endpoint, but does not report the primary result needed to assess the observed renal-function outcome.

14. Why This Trial Matters Statistically

The study illustrates why statistical interpretation begins with the design rather than the treatment name or the study title. The registry calls the study controlled in the official title, but lists a single-group model, allocation as NA, and one intervention arm. Those design details determine whether a direct comparison is available. Without a listed comparator, readers should not treat the study as a randomized head-to-head test of treatment strategies.

It also highlights the distinction between an outcome definition and an outcome result. Estimated GFR is a clinically relevant continuous measure for describing renal function, and the registry specifies a mean and standard deviation at 2 years. Yet those details do not reveal the observed value, change, or uncertainty. A clear endpoint definition supports interpretation only when the corresponding estimates and analysis details are available.

Missing-data choices are another important statistical feature. The registry identifies last observation carried forward for the primary endpoint. As a general method, carrying an earlier value forward makes an assumption about what a later unobserved value would have been. Reporting the extent of missingness and checking how conclusions respond to alternative assumptions would help readers understand the robustness of an analysis; those details are not reported in the registry record.

Finally, the safety entries show why event summaries need context. Counts and listed terms describe reported events in the study arm, but without comparative safety data they do not quantify excess risk attributable to the regimen. The tables, plots, and planning illustrations can help organize information, but they cannot supply an unreported endpoint result or turn a single-group record into a randomized comparison. The statistical value of the registry entry is in clarifying what was planned and what was posted, as well as making the gaps in interpretable results visible.

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.

The main limitation for interpreting this specific study is that the registry record does not report the primary renal-function result. It gives the endpoint definition and timeframe, but no observed mean, standard deviation, change, or confidence interval. Without those estimates, readers cannot assess the level or precision of renal function at the primary assessment.

The design fields identify a single-group study, with allocation recorded as NA and no masking. A separate comparator arm is not listed. This limits direct comparative inference: outcomes among participants in the intervention arm cannot, by themselves, establish what would have occurred under another regimen. Although the official title describes a controlled trial, the registry entries do not specify a control group or a randomized comparison.

The registry gives enrollment as 55, but does not report the number included in the primary endpoint analysis or the degree of follow-up for that outcome. It specifies LOCF, yet does not report how much data were missing, why observations were missing, or whether the results were examined under different missing-data assumptions. These omissions make it difficult to judge the potential influence of imputation.

The record lists 20 outcome measures and multiple secondary outcomes, but does not report their analysis results or a multiplicity strategy. It also does not report further statistical-model details, such as whether baseline values or repeated measurements were included. The protocol’s sample-size justification is not reported in the registry record. Planning illustrations elsewhere should not be mistaken for that justification or for observed power.

Safety information is also limited to the posted event summaries. The record does not explain the relation between the safety denominators and enrollment, provide comparative event rates, or give detailed timing and outcomes for the listed terms. These gaps limit both causal interpretation and assessment of how complete the safety reporting is.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Evaluation of Calcineurin-inhibitor Reduction With Conversion at 2 Months to Everolimus/Reduced Tacrolimus in Renal Transplant Recipients Following Campath® Induction — NCT01935128.
• Registry record last updated 2022-05-26; status last verified 2022-05.
• Results first posted 2022-05-26.
• 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 describes a completed Phase 4, single-center, pilot, open-label study of everolimus with reduced-dose tacrolimus in adult renal transplant recipients following Campath® induction and steroid avoidance. Enrollment is 55. The study started on 2013-07-03, and primary completion is recorded as 2020-07.

The primary outcome is renal function at 2 years, assessed by MDRD-estimated GFR and summarized as a mean with standard deviation. The registry describes an intent-to-treat population and last observation carried forward imputation. It lists secondary outcomes and safety-event summaries for the intervention arm.

The model is listed as single group, allocation as NA, and masking as none. No separate comparator arm is listed. Most importantly, the primary endpoint result and outcome-level analyses are not reported in the registry record. The entry therefore supports a description of the study design and planned assessment, but not a conclusion about the observed renal-function result or a comparative treatment effect.