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 Mechanisms and Treatment of Chronic Allograft Injury (CAI) Due to Calcineurin Inhibitor (CNI) Toxicity (NCT01473732), a randomized na study. The primary outcome is Change in eGFR (Creatinine Clearance) as Measured by Serum Creatinine Blood Test.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01473732 |
| Phase | NA |
| Condition | Chronic Allograft Injury, Calcineurin Inhibitor Toxicity |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 2 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Montefiore Medical Center |
| Results posted | Yes |
2. Clinical Question
Population
People with chronic kidney injury associated with Prograf or Neoral use. The registry describes the condition as chronic allograft injury associated with calcineurin inhibitor toxicity. It reports that 2 participants enrolled, but neither reached the one-year assessment.
Intervention
Everolimus (Zortress) combined with mycophenolic acid (Myfortic). The study description frames this as an alternative regimen for patients with chronic kidney injury associated with Prograf or Neoral use.
Comparator
Reduced-dose tacrolimus (Prograf) combined with mycophenolic acid (Myfortic). The brief summary describes this as the current drug regimen, including a low Prograf dose and Myfortic.
Primary question
The planned primary question was whether the regimens differed in change in eGFR, described as creatinine clearance measured by serum creatinine blood test, from baseline to one year. The registry reports that the participants did not complete the study and there was no data to analyze, so it does not provide an estimate of the comparison.
3. Trial Design
The registry describes a randomized, parallel-group study with no masking. The two listed treatment groups were everolimus (Zortress) plus mycophenolic acid (Myfortic), and reduced-dose tacrolimus (Prograf) plus mycophenolic acid (Myfortic). The record lists Montefiore Medical Center as the sponsor and the phase as NA. Enrollment was 2.
The study began in 2012-03, had a primary completion date of 2012-08, and is recorded as terminated. Those dates describe the registry record; they do not establish why the study ended or how long each participant was followed. The registry says that the only two patients enrolled did not reach the one-year mark and did not complete the study.
Random allocation is intended to reduce systematic differences between treatment groups at the time of assignment. In a parallel design, participants are assigned to a treatment group and evaluated in that group. Neither design feature can ensure that the groups will be comparable when enrollment is extremely limited, and neither can replace outcome data. With no analyzable primary endpoint reported, the record cannot show how the randomized comparison turned out.
The registry’s brief summary describes an interest in both how well the regimens work and their safety. The record lists a primary outcome and adverse-event counts, but does not report an analysis of the primary outcome. It also does not report enough detail to establish the timing or completeness of adverse-event collection. The status is terminated; the reason for termination is not reported in the registry record.
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 | Everolimus (Zortress)+ Mycophenolic Acid(Myfortic) | Reduced Dose Prograf+ Mycophenolic Acid(Myfortic) |
|---|---|---|
| STARTED | 2 | 0 |
| COMPLETED | 0 | 0 |
| NOT COMPLETED | 2 | 0 |
Reasons for not completing this period:
| Reason | Everolimus (Zortress)+ Mycophenolic Acid(Myfortic) | Reduced Dose Prograf+ Mycophenolic Acid(Myfortic) |
|---|---|---|
| Adverse Event | 1 | 0 |
| Death | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: The only two patients enrolled did not reach the one year mark; they did not complete the study so there was no data to analyze.
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: We did not analyze the data due to no patient enrolled in reduced dose prograf group and 2 patients enrolled in everolimus group did not complete the study due to death and adverse event
| Characteristic | Unit | Everolimus (Zortress)+ Mycophenolic Acid(Myfortic) | Reduced Dose Prograf+ Mycophenolic Acid(Myfortic) | Total |
|---|---|---|---|---|
| Participants analysed | 2 | 0 | 2 | |
| Age, Continuous | years | 63 (SD 2) | — | 63 (SD 2) |
| Sex: Female, Male: Female | Participants | 2 | — | 2 |
| Sex: Female, Male: Male | Participants | 0 | — | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | — | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | — | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | — | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 | — | 1 |
| Race (NIH/OMB): White | Participants | 0 | — | 0 |
| Race (NIH/OMB): More than one race | Participants | 1 | — | 1 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | — | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Change in eGFR (Creatinine Clearance) as Measured by Serum Creatinine Blood Test, measured over Baseline, One year.
| 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 described as change in eGFR (creatinine clearance) measured by a serum creatinine blood test, assessed at baseline and one year. eGFR is an estimate of kidney filtration and is used as an indicator of kidney function. The registry describes a normal eGFR value for healthy people as 80-120ml/min and says that for transplants, 60-80 ml/min is expected.
Assessing change over time can describe whether kidney function has shifted from a participant’s baseline during follow-up. A baseline measure provides a reference for interpreting a later measure; comparing changes between randomized groups would address the trial’s stated treatment question. These are general reasons for using a change endpoint, not findings from this study.
The registry does not report a value, unit, summary statistic, dispersion measure, or treatment comparison for the primary outcome. It explicitly states that the 2 enrolled participants did not reach the one-year mark, did not complete the study, and had no data to analyze. Consequently, neither the amount of change nor the precision of a between-group estimate can be assessed from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive fact is that the registry reports no analyzable primary outcome. The endpoint was change in eGFR from baseline to one year, but the only two enrolled participants did not reach the one-year mark and did not complete the study. No group-level change, between-group difference, confidence interval, or primary-outcome test result is reported. The record therefore does not quantify the comparative effect of the two regimens on eGFR.
This absence of an estimate is different from a finding of no difference. A null result would require outcome data and an analysis that estimated a difference, even if that estimate were close to zero or too imprecise to distinguish from other possibilities. No such analysis is available in the registry record. It is not possible to infer that the treatments were equivalent, that one was superior, or that either did or did not alter kidney function.
The registry does list adverse-event counts. For the everolimus plus mycophenolic acid group, it reports 2 at risk and 2 with serious adverse events; it also reports 2 at risk for other adverse events and 0 affected. For the reduced-dose Prograf plus mycophenolic acid group, it reports 0 at risk and 0 affected for both serious and other adverse events. The record lists 1 death in the everolimus group and 0 in the reduced-dose Prograf group. These counts need cautious interpretation: the registry record does not explain the zero at-risk entries for the comparator group, provide a safety analysis, or establish whether an event was caused by a treatment.
The listed serious-event terms are proteinuria, 50.0%, and Small bowel obstruction leading to death, 50.0%. These entries describe the terms and percentages recorded, not comparative risks or causal effects. With such limited enrollment and no reported primary outcome data, the posted information supports description of the record only. It cannot support a reliable estimate of treatment benefit, harm, or comparative safety.
The study is marked terminated. The reason, any planned interim review, and any decisions made on the basis of the available observations are not reported in the registry record. The termination status and the absence of one-year outcomes should therefore be kept distinct: the record establishes that the planned primary assessment is unavailable, but it does not explain why the study stopped.
9. Statistical Methods Explained
The methods information in the registry is limited. It identifies random allocation, no masking, a parallel model, the primary endpoint, and the baseline-to-one-year timeframe. It does not report the statistical analysis used for the primary outcome, the method of randomization, or details of the analysis population. The following concepts explain how such methods generally work; they are not claims about procedures actually used in this study.
What does random allocation contribute?
Random allocation uses chance to assign participants to treatment groups. Its purpose is to make treatment assignment independent of measured and unmeasured participant characteristics, on average, across repeated assignments. In a large enough study, this can reduce confounding and make a group comparison more interpretable. Randomization does not guarantee balance in a particular small study, does not correct incomplete follow-up, and does not create outcome data where none were collected. The registry identifies the allocation as randomized but does not describe the sequence generation or concealment procedures.
What does an unmasked design mean?
The masking entry is NONE. In an unmasked study, participants or study personnel may know which regimen is assigned, depending on the procedures used. Awareness of assignment can influence behavior, co-interventions, reporting, or assessment. The potential for such influences depends on the outcome and study conduct. The registry does not describe which people knew assignments, whether outcome assessment was standardized, or whether any safeguards were used. The primary endpoint is based on a laboratory measure, but that fact alone does not establish how measurement or data handling was conducted.
How should the primary outcome comparison be understood?
The endpoint is change in eGFR from baseline to one year. In general, a change comparison evaluates how much the measured outcome shifts over time in each treatment group and then compares those changes. The analysis may account for baseline values or other prespecified factors, but the selected method depends on the protocol and data. The registry does not report a statistical model, adjustment variables, handling of repeated measurements, or a calculation for a treatment contrast. It also does not report an estimate or its uncertainty. Since the participants did not reach the one-year assessment, the registry states that there was no data to analyze for the primary outcome.
What do confidence intervals and p-values ordinarily convey?
In general, a confidence interval describes uncertainty around an estimated effect under the assumptions of the analysis. A narrower interval indicates greater precision, while a wider interval indicates less precision. A p-value is a measure calculated under a specified statistical model and a null hypothesis; it is not the probability that a treatment works or that the null hypothesis is true. Neither measure is reported for this primary outcome. Without an analyzed outcome estimate, there is no primary comparison to interpret for precision or statistical compatibility with possible effects.
What is over-dispersion, and is it relevant?
Over-dispersion is a general issue in count-data models: observed variation can exceed what a chosen model assumes. If not addressed where present, standard errors and resulting uncertainty measures may be misleading. The record does not report a count-based primary outcome or a count-data model. The primary endpoint is an eGFR change, and no primary analysis is reported, so there is no basis for asserting that over-dispersion was present or required a particular adjustment in this trial.
What do the planning assumptions mean?
The registry’s sample-size assumptions describe a planning illustration, not the trial’s own design: a two-sided significance level of 0.05, equal allocation between the two arms, a normal approximation, and an effect to detect stated as a fraction of the observed effect. It also distinguishes time-to-event illustrations, which give total events, from other illustrations, which give participants per arm. These are assumptions for interpreting planning calculations, not evidence that the trial used those procedures or enrolled to a particular target. No sample-size justification or actual statistical plan is reported in the registry record. Because no primary outcome effect was analyzed, the assumptions cannot supply a valid observed treatment effect or observed power.
In general, sample-size calculations estimate how much information a future study might need under stated assumptions. They do not demonstrate that an existing study was adequately powered, and a planning illustration is not an observed result. Any such calculation depends on assumptions about the effect, variability, significance level, allocation, and analysis. The registry does not report the trial’s own sample-size justification or enough primary outcome information to assess the assumptions against an observed comparison.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity arises when a study conducts several hypothesis tests or estimates several outcomes, increasing the chance that at least one result appears notable by chance. How to address that issue depends on the prespecified objectives and analysis plan. The registry lists 1 outcome measure and identifies a primary endpoint, but the statistical plan, any secondary outcomes, and any multiplicity adjustment are not reported in the registry record. No primary outcome analysis is reported, so there are no reported hypothesis-test results to assess for multiplicity.
The main missing-data issue is that the participants did not reach the one-year assessment and did not complete the study. The registry explicitly says there was no data to analyze for the primary endpoint. It does not report when the participants left follow-up, why they did not complete the study, whether baseline eGFR values were recorded, or whether any earlier measurements were available. It also does not describe imputation, sensitivity analyses, or other methods for handling missing outcomes. The missingness mechanism cannot be inferred from the record.
An analysis population describes which participants are included in a particular analysis, such as all randomized participants or those who completed follow-up. The registry does not report an analysis population or a primary endpoint analysis. Enrollment was 2, but the record does not describe the allocation of those participants or give an analyzed group count for the primary outcome. No interim analyses, stopping rules, or statistical decision process are reported in the registry record. The terminated status does not establish whether any such procedures occurred.
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 |
|---|---|---|---|
| Everolimus (Zortress)+ Mycophenolic Acid(Myfortic) | 2 | 2 | 100.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Everolimus (Zortress)+ Mycophenolic Acid(Myfortic) | 1 of 2 |
| Reduced Dose Prograf+ Mycophenolic Acid(Myfortic) | 0 of 0 |
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 |
|---|---|
| Everolimus (Zortress)+ Mycophenolic Acid(Myfortic) | 0 of 2 |
| Reduced Dose Prograf+ Mycophenolic Acid(Myfortic) | 0 of 0 |
Most frequent serious adverse event terms
| Event term | Organ system | Everolimus (Zortress)+ Mycophenolic Acid(Myfortic) | Reduced Dose Prograf+ Mycophenolic Acid(Myfortic) |
|---|---|---|---|
| proteinuria | Renal and urinary disorders | 1 of 2 | 0 of 0 |
| Small bowel obstruction leading to death | Gastrointestinal disorders | 1 of 2 | 0 of 0 |
Serious events with the highest arm-level rate: proteinuria (50.0%), small bowel obstruction leading to death (50.0%).
The registry lists serious and other adverse-event counts by treatment group. For everolimus (Zortress) plus mycophenolic acid (Myfortic), it reports 2 at risk and 2 affected for serious adverse events, and 2 at risk and 0 affected for other adverse events. It lists 1 death in that group. For reduced-dose Prograf plus mycophenolic acid (Myfortic), the record reports 0 at risk and 0 affected for serious and other adverse events, and 0 deaths.
The serious-event terms are proteinuria, 50.0%, and Small bowel obstruction leading to death, 50.0%. These are registry-listed terms and percentages. The record does not report the event-level timing, duration of observation, event ascertainment procedures, or a statistical comparison. It also does not explain why the reduced-dose group is listed with 0 at risk, despite the study enrollment of 2. These counts therefore should not be interpreted as a complete estimate of comparative safety or as evidence that a treatment caused an event. The death is a reported safety count, not by itself proof of treatment attribution.
12. What the Result Does, and Does Not, Mean
The record supports a narrow description: a randomized, unmasked, parallel study was registered to compare everolimus plus mycophenolic acid with reduced-dose tacrolimus plus mycophenolic acid in people with chronic kidney injury associated with Prograf or Neoral use. Its primary outcome was change in eGFR from baseline to one year. The registry says the 2 enrolled participants did not reach that assessment, did not complete the study, and had no data to analyze.
Accordingly, no estimate of the primary treatment contrast is available. The record does not establish whether eGFR changed in either group, whether the regimens differed, or how precise any estimated difference would have been. The absence of an analysis is not evidence of no effect, equivalence, or lack of effect. Nor does the registry support a conclusion that either regimen is better or worse.
The adverse-event entries document what the registry lists for the groups, including serious-event counts and 1 death in the everolimus group. They do not establish treatment causality or a reliable difference in risk. The lack of an explanation for the comparator group’s zero at-risk entries further limits interpretation.
More broadly, these registry results cannot resolve the clinical question posed in the study description. They also cannot determine why the study terminated, whether the participants received the assigned regimens as intended, or whether any additional data exist outside the registry record. Those matters are not reported in the registry record. The appropriate reading is that the planned primary comparison is unavailable, while the listed safety counts remain descriptive and limited.
13. Why This Trial Matters Statistically
This study illustrates an important distinction between a trial’s question and the evidence its record can answer. The registry describes a clinically motivated comparison of two drug combinations and specifies a kidney-function endpoint. But the endpoint is useful for comparative inference only when follow-up measurements and an analysis are available. The record states that the enrolled participants did not reach the one-year mark and that there was no primary outcome data to analyze.
Randomization is a valuable design feature because it is intended to support a fair comparison. It cannot overcome the absence of outcome observations. Nor can the design label alone establish balance between groups or make descriptive adverse-event counts a comparative safety analysis. The record’s adverse-event entries, including a reported death, warrant careful description but do not resolve treatment attribution or comparative risk.
For biostatisticians and clinical researchers, the record also underscores why trial status, follow-up, analysis populations, missing-data handling, and statistical plans matter. A planned endpoint and a randomized design do not guarantee an interpretable estimate. When these details and outcome results are missing, readers need to distinguish what the registry records from what remains unknown. Here, the posted information documents a study objective and limited safety counts, but not an analyzed estimate of the primary treatment effect.
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 most direct limitation is the absence of analyzable primary outcome data. The registry states that the 2 enrolled participants did not reach the one-year mark and did not complete the study. No eGFR results, treatment contrast, uncertainty interval, or test result is reported. Thus, the primary comparison cannot be interpreted statistically.
Enrollment of 2 also means that the record contains very limited information about the study population and the treatment groups. The allocation of participants is not reported. The adverse-event listing gives 0 at risk for the reduced-dose Prograf group, but the record does not explain this entry. The available information therefore does not establish the safety follow-up denominator for that group.
Other limitations include the absence of a reported statistical analysis plan, analysis population, missing-data method, randomization details, masking procedures beyond the NONE entry, and reason for termination. The registry does not report whether there were interim analyses, a sample-size justification, or a stopping rule. These omissions prevent assessment of how the study was intended to handle incomplete follow-up or make statistical decisions. They should not be filled by assumptions about usual practice.
The safety entries are descriptive, with no reported comparative analysis or information about event timing and ascertainment. The listed serious-event terms and death cannot establish causality. The registry record is therefore limited both for estimating eGFR effects and for comparing safety outcomes.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Mechanisms and Treatment of Chronic Allograft Injury (CAI) Due to Calcineurin Inhibitor (CNI) Toxicity — NCT01473732.
- Registry record last updated 2018-10-16; status last verified 2018-09.
- Results first posted 2018-09-18.
- 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
NCT01473732, titled “Mechanisms and Treatment of Chronic Allograft Injury (CAI) Due to Calcineurin Inhibitor (CNI) Toxicity,” is recorded as a randomized, unmasked, parallel study sponsored by Montefiore Medical Center. It enrolled 2 participants and is listed as terminated. The registered comparison was everolimus plus mycophenolic acid versus reduced-dose tacrolimus plus mycophenolic acid.
The primary endpoint was change in eGFR from baseline to one year. The registry says the only two enrolled patients did not reach the one-year mark, did not complete the study, and had no data to analyze. It lists adverse-event counts, including 1 death in the everolimus group, but does not provide a comparative safety analysis. The reason for termination and the study’s statistical plan are not reported in the registry record.