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 Advancing Renal TRANSplant eFficacy and Safety Outcomes With an eveRolimus-based regiMen (TRANSFORM) (NCT01950819), a randomized phase 4 study. The primary outcome is Incidence of Failure on the Composite of Treated Biopsy-proven Acute Rejection (tBPAR) or Estimated Glomerular Filtration Rate (eGFR) < 50 mL/Min/1.73m2..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01950819 |
| Phase | Phase 4 |
| Condition | End Stage Renal Disease (ESRD), Chronic Kidney Disease (CKD), Hemodialysis |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 2037 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Novartis Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
Adult recipients of a de novo renal transplant were the population described in the trial summary. The primary outcome analysis population is identified as the full analysis set. The registry record lists end stage renal disease, chronic kidney disease, hemodialysis, renal replacement therapy and renal transplantation among the conditions associated with the study. Further eligibility details are not reported in the registry record.
Intervention
The experimental regimen combined everolimus with reduced exposure to a calcineurin inhibitor, abbreviated CNI. Everolimus had a target trough level of 3-8 ng/mL. The CNI could be cyclosporine or tacrolimus. Induction therapy and corticosteroids were also listed as interventions, but further details of their use are not reported in the registry record.
Comparator
The comparator combined mycophenolate with standard exposure to a calcineurin inhibitor. Mycophenolate could be mycophenolic acid sodium or mycophenolate mofetil, and the CNI could be cyclosporine or tacrolimus. Specific dosing information for this regimen is not reported in the registry record.
Primary question
The primary question concerned the incidence of failure on a composite endpoint: treated biopsy-proven acute rejection, or estimated glomerular filtration rate (eGFR) below 50 mL/min/1.73m2. Month 12 is identified as the primary time point, with Month 24 secondary. The registry record does not report event counts or a numerical comparison between the treatment groups for this endpoint.
3. Trial Design
TRANSFORM was a Phase 4, randomized, parallel-group, open-label study in de novo renal transplantation. The record describes it as a multicenter study of everolimus with reduced calcineurin inhibitor exposure compared with mycophenolate with standard calcineurin inhibitor exposure. The brief summary characterizes the study as lasting 2 years. Enrollment was 2037. The record lists 2013-12-03 as the study start and 2017-02-01 as primary completion, and the study status is completed.
The two regimens differed in both the antimetabolite or immunosuppressive agent and the intended level of CNI exposure. In the everolimus arm, everolimus was used with reduced exposure to cyclosporine or tacrolimus; the target everolimus trough level was 3-8 ng/mL. In the comparator arm, mycophenolate was paired with standard exposure to cyclosporine or tacrolimus. The record also lists induction therapy and corticosteroids among the interventions. Details such as specific dosing schedules, treatment adherence, changes over follow-up and criteria for modifying therapy are not reported in the registry record.
Random allocation is intended to balance prognostic factors between groups on average, while the parallel design assigns participants to a regimen for comparison over the study period. The study was open-label, meaning masking was not used. The record does not explain how the lack of masking was addressed in outcome assessment or clinical management. It also does not report the randomization procedure, allocation concealment, stratification factors, or the planned handling of treatment changes and withdrawals. These details should not be inferred from the broad design labels.
The registry identifies a full analysis set for the primary outcome and describes a primary Month 12 assessment with Month 24 as secondary. It lists 22 outcome measures and one analysis, but a detailed statistical analysis plan is not reported in the registry record. The primary endpoint is a composite of treated biopsy-proven acute rejection or eGFR below the specified threshold; other listed outcomes include additional rejection, graft loss, death and renal-function measures. The record describes the study design and outcomes, but does not provide a numerical primary-endpoint comparison.
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: 2226 patients screened; 2037 patients transplanted and randomized to treatment.
Overall Study
| Stage | EVR+rCNI | MPA+sCNI |
|---|---|---|
| STARTED | 1022 | 1015 |
| COMPLETED | 893 | 881 |
| NOT COMPLETED | 129 | 134 |
Reasons for not completing this period:
| Reason | EVR+rCNI | MPA+sCNI |
|---|---|---|
| Graft Loss | 35 | 30 |
| Death | 29 | 34 |
| Subject / Gardian decision | 54 | 51 |
| technical problems | 2 | 0 |
| Pregnancy | 0 | 2 |
| Lost to Follow-up | 9 | 17 |
Analysis population for the primary outcome, as defined in the registry: full analysis set
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: Full Analysis set
| Characteristic | Unit | EVR+rCNI | MPA+sCNI | Total |
|---|---|---|---|---|
| Participants analysed | 1022 | 1015 | 2037 | |
| Age, Continuous | years | 48.79 (SD 14.123) | 48.75 (SD 14.515) | 48.77 (SD 14.316) |
| Sex: Female, Male: Female | Participants | 312 | 308 | 620 |
| Sex: Female, Male: Male | Participants | 710 | 707 | 1417 |
| Race/Ethnicity, Customized: Hispany or Latino | Participants | 157 | 132 | 289 |
| Race/Ethnicity, Customized: not Hispanic or Latino | Participants | 646 | 661 | 1307 |
| Race/Ethnicity, Customized: Unknown or not reported | Participants | 219 | 222 | 441 |
| Race/Ethnicity, Customized: Caucasian | Participants | 743 | 735 | 1478 |
| Race/Ethnicity, Customized: Asian | Participants | 136 | 157 | 293 |
| Race/Ethnicity, Customized: Black | Participants | 43 | 35 | 78 |
| Race/Ethnicity, Customized: Other | Participants | 100 | 88 | 188 |
6. Primary Endpoint
The registry lists the primary outcome as Incidence of Failure on the Composite of Treated Biopsy-proven Acute Rejection (tBPAR) or Estimated Glomerular Filtration Rate (eGFR) < 50 mL/Min/1.73m2., measured over Month 12 is Primary, Month 24 secondary.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint combines treated biopsy-proven acute rejection (tBPAR) with eGFR below 50 mL/min/1.73m2. It measures whether a participant experiences either of these events by the assessment time. Because the endpoint is a composite, a participant can meet it through rejection, through renal function falling below the threshold, or through both. The primary assessment is identified as Month 12; Month 24 is secondary.
Composite endpoints are used in clinical trials to capture more than one clinically relevant type of outcome within a single measure. They can also increase the number of participants who experience the defined endpoint compared with using a less frequent component alone. However, a composite result does not automatically mean that each component has a similar frequency, importance or treatment-group pattern. Component-specific counts and analyses are needed to understand what contributes to a composite difference. The registry lists related outcomes, including rejection, graft loss, death and eGFR measures, but the numerical primary result and component results are not reported in the registry record.
The endpoint is based on participants, rather than a continuous average eGFR value. The threshold-based component classifies whether eGFR is below a prespecified level; it does not by itself describe the size or trajectory of renal-function changes. A continuous eGFR measure or change over time can answer different questions about the distribution and evolution of renal function. The record lists mean eGFR and eGFR slope among secondary measures, but does not report their results in the registry record described for this analysis. The clinical meaning of a composite therefore depends on both the event definition and the component-level evidence, neither of which can be fully assessed from the primary endpoint description alone.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a randomized comparison of two immunosuppressive regimens and specifies a binary primary outcome: the incidence of failure on a composite of treated biopsy-proven acute rejection or eGFR below 50 mL/min/1.73m2. It gives Month 12 as the primary time point and Month 24 as secondary. However, it does not report the number of participants meeting the primary endpoint in either arm, an effect estimate, a confidence interval, or a p-value. Consequently, the direction, magnitude and statistical precision of the primary comparison cannot be determined from the registry record.
The primary comparison is described as not reduced to one comparison because the outcome has several categories or time points. That means no single numerical contrast is available for interpreting the main endpoint from this record. The absence of a reported estimate is not evidence that the groups had the same outcomes, nor does it establish that one regimen had better or worse results. A null finding, a meaningful difference and an imprecise estimate cannot be distinguished without the underlying results.
The registry lists an enrollment of 2037, while the safety information gives at-risk denominators of 1014 for Everolimus Plus@Reduced CNI and 1012 for MPA Plus Standard@CNI. These are distinct types of information: enrollment describes the overall study enrollment, while the denominators attached to safety results describe the groups at risk for those safety summaries. The record does not explain the relationship between these figures or provide a participant flow account in the registry record. The figures should not be used to reconstruct an unreported primary-endpoint analysis.
Serious adverse event summaries report 593 affected participants among 1014 at risk in the everolimus plus reduced CNI group, and 613 among 1012 at risk in the mycophenolate plus standard CNI group. Other adverse events are reported for 951 among 1014 and 942 among 1012, respectively. The registry also reports deaths of 20 and 29, respectively. These are descriptive safety counts; the record does not provide a statistical comparison or uncertainty interval for them. No additional rates, contrasts or tests can be established from the counts alone without calculations, and no treatment conclusion should be drawn from them in isolation.
Among the listed serious adverse event terms, urinary tract infection is reported as 9.2%, diarrhoea and pneumonia as 5.4% each, blood creatinine increased as 4.9%, cytomegalovirus infection as 4.7%, and acute kidney injury as 4.5%. The record does not specify in the information described whether these percentages are arm-specific or pooled. They therefore cannot be assigned to a particular regimen. The listing also does not show how these events were distributed over time, how severity was defined, or how missing safety observations were handled.
Overall, the record supports a description of the study question, design and selected safety counts, but not an efficacy conclusion. A complete statistical interpretation would require the primary outcome counts or estimate, the method used to compare groups, uncertainty around that estimate, and the relevant analysis population and missing-data details. These are not reported in the registry record.
9. Statistical Methods Explained
Randomization and statistical comparison
Randomization assigns participants to treatment groups by chance. In general, this helps reduce systematic differences between groups at the start of a trial, although it does not guarantee identical groups. A treatment-effect estimate compares the outcome between randomized groups according to a defined analysis. For a binary endpoint such as the primary composite, results might be reported as group event counts and a comparative estimate with a confidence interval. The interval communicates the range of effect sizes that remain compatible with the observed data under the analysis assumptions. The registry record does not report a primary-endpoint estimate, confidence interval, p-value or the exact comparison method.
The study is described as randomized and parallel-group, but the randomization sequence generation, concealment procedures and any stratification are not reported in the registry record. These are important implementation details: sequence generation concerns how assignments are created, while concealment concerns whether the next assignment can be known before enrollment. The general purpose of allocation concealment is to reduce the possibility that knowledge of future assignment affects enrollment decisions. The record does not provide enough detail to assess these procedures.
Open-label design and outcome assessment
The study had no masking. In an open-label trial, participants and treating clinicians know the assigned regimen. General methodological concerns include possible differences in co-interventions, participant reporting, follow-up and decisions to investigate or treat an event. These concerns are particularly relevant to outcomes involving clinical judgment, such as treated rejection. Objective laboratory measures may be less directly affected by expectations, although measurement, timing and missingness still matter. The registry record does not describe blinded outcome adjudication or other procedures used to limit bias in outcome assessment.
Binary endpoints and composites
The primary measure is a participant-level binary outcome: a participant either meets the composite failure definition by the specified assessment or does not. The composite includes treated biopsy-proven acute rejection and eGFR below 50 mL/min/1.73m2. General interpretation of a composite requires attention to its components. A summary comparison can be driven mainly by a more frequent component, and the components can differ in clinical importance. A composite estimate should therefore be read alongside component-specific results when available. The registry lists several related secondary outcomes, but the component counts and primary comparison are not reported in the registry record.
Time point and follow-up
The primary endpoint is assessed at Month 12, with Month 24 designated secondary. A fixed-time binary analysis generally summarizes whether each participant experienced the event by the chosen time. A time-to-event analysis instead uses the timing of events and may account for differing follow-up, subject to its assumptions. The registry record does not report the primary analysis method, event timing, censoring approach, follow-up completeness or whether the primary comparison used a fixed-time or time-to-event framework. It is therefore not appropriate to infer these features from the endpoint wording.
Multiplicity and planned comparisons
When a trial evaluates several outcomes, time points, treatment contrasts or subgroups, repeated testing can raise the chance of at least one apparently notable result by chance. General approaches include identifying one primary outcome in advance, adjusting for multiple tests, or using a prespecified testing hierarchy. The record identifies a primary outcome and a secondary time point, and lists 22 outcome measures. It does not report a multiplicity-control strategy, a testing hierarchy, or how secondary outcomes were intended to support interpretation. The existence of multiple listed outcomes alone does not establish which, if any, adjustments were planned.
Sample-size planning and power
Sample-size planning depends on assumptions about the outcome frequency, the effect considered important, variability, allocation, significance level and desired power. The planning assumptions described for derived illustrations use a two-sided significance level of 0.05, equal allocation between the two arms, a normal approximation, and an effect defined as a stated fraction of the observed effect. Such illustrations are conditional planning exercises, not observed trial results and not observed power. They do not establish the trial's original design assumptions. The sponsor's own sample-size justification is not reported in the registry record. No sample-size illustration should be read as evidence that the trial detected a particular effect, and no planning value substitutes for an observed treatment-effect estimate.
Missing data and analysis populations
Missing outcome information can affect a participant-level endpoint if event status cannot be established at the planned assessment. General strategies include prespecified imputation, sensitivity analyses, or analyses that use available observations, but each relies on assumptions. The registry identifies the full analysis set for the primary endpoint but does not define its membership, explain the treatment of withdrawals or missing eGFR measurements, or report sensitivity analyses. The statistical plan for handling intercurrent events, treatment discontinuation or crossover is also not reported in the registry record. Without these details, the consequences of missingness and departures from assigned treatment cannot be assessed.
Safety summaries
Safety counts describe how many participants were at risk and how many were recorded as affected for the reported summaries. Such counts are descriptive unless accompanied by a defined comparison and statistical uncertainty. If participants have different follow-up durations, raw counts may not reflect differences in time at risk; the record does not report person-time or a method addressing this issue. General safety interpretation also distinguishes participants with at least one event from the total number of event episodes. The registry information lists affected-participant counts, but further details of event counting, follow-up and analysis are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry record identifies one primary outcome, a composite of treated biopsy-proven acute rejection or eGFR below 50 mL/min/1.73m2, with Month 12 designated primary and Month 24 secondary. It also lists 22 outcome measures, including other composites, rejection outcomes, graft loss, death and several measures of renal function. Because multiple outcomes and assessment times can generate multiple statistical comparisons, prespecified control of multiplicity can matter when interpreting inferential results. The record does not report a multiplicity adjustment, a gatekeeping procedure, a testing hierarchy or the planned role of secondary outcomes. The registry entries therefore do not establish how the family of comparisons was intended to be interpreted.
The primary outcome population is described as the full analysis set. The record does not define that set or state how many participants contributed to the primary analysis. It does not report the handling of missing outcome values, missing eGFR measurements, withdrawal, treatment discontinuation, crossover or other intercurrent events. These omissions prevent assessment of whether all randomized participants were included as intended, how missingness could affect the endpoint, or whether sensitivity analyses tested the robustness of the findings. A detailed statistical analysis plan is not reported in the registry record.
The primary endpoint result itself is not reported as a single comparison. As a result, multiplicity and missing-data methods cannot be evaluated against an observed primary estimate, and the precision of the primary analysis cannot be assessed. These are limitations of what can be learned from the registry record, rather than evidence that any particular 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 |
|---|---|---|---|
| Everolimus Plus@Reduced CNI | 593 | 1014 | 58.5% |
| MPA Plus Standard@CNI | 613 | 1012 | 60.6% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Everolimus Plus@Reduced CNI | 20 of 1014 |
| MPA Plus Standard@CNI | 29 of 1012 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Everolimus Plus@Reduced CNI | 951 of 1014 |
| MPA Plus Standard@CNI | 942 of 1012 |
Most frequent serious adverse event terms
| Event term | Organ system | Everolimus Plus@Reduced CNI | MPA Plus Standard@CNI |
|---|---|---|---|
| Urinary tract infection | Infections and infestations | 78 of 1014 | 93 of 1012 |
| Pneumonia | Infections and infestations | 55 of 1014 | 36 of 1012 |
| Blood creatinine increased | Investigations | 50 of 1014 | 36 of 1012 |
| Acute kidney injury | Renal and urinary disorders | 46 of 1014 | 37 of 1012 |
| Diarrhoea | Gastrointestinal disorders | 27 of 1014 | 55 of 1012 |
| Pyrexia | General disorders | 35 of 1014 | 36 of 1012 |
| Transplant rejection | Immune system disorders | 45 of 1014 | 25 of 1012 |
| Complications of transplanted kidney | Injury, poisoning and procedural complications | 34 of 1014 | 28 of 1012 |
| Pyelonephritis | Infections and infestations | 24 of 1014 | 33 of 1012 |
| Lymphocele | Vascular disorders | 34 of 1014 | 21 of 1012 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Everolimus Plus@Reduced CNI | MPA Plus Standard@CNI |
|---|---|---|---|
| Oedema peripheral | General disorders | 347 of 1014 | 244 of 1012 |
| Diarrhoea | Gastrointestinal disorders | 242 of 1014 | 318 of 1012 |
| Constipation | Gastrointestinal disorders | 245 of 1014 | 242 of 1012 |
| Hypertension | Vascular disorders | 240 of 1014 | 230 of 1012 |
| Urinary tract infection | Infections and infestations | 219 of 1014 | 249 of 1012 |
| Anaemia | Blood and lymphatic system disorders | 227 of 1014 | 238 of 1012 |
| Nausea | Gastrointestinal disorders | 191 of 1014 | 225 of 1012 |
| Hypophosphataemia | Metabolism and nutrition disorders | 190 of 1014 | 167 of 1012 |
| Hyperkalaemia | Metabolism and nutrition disorders | 164 of 1014 | 184 of 1012 |
| Hypomagnesaemia | Metabolism and nutrition disorders | 134 of 1014 | 168 of 1012 |
Serious events with the highest arm-level rate: urinary tract infection (9.2%), diarrhoea (5.4%), pneumonia (5.4%), blood creatinine increased (4.9%), cytomegalovirus infection (4.7%).
The serious adverse event summary reports 593 affected participants among 1014 at risk in the Everolimus Plus@Reduced CNI group, and 613 among 1012 at risk in the MPA Plus Standard@CNI group. The other adverse event summary reports 951 among 1014 and 942 among 1012, respectively. Death counts are 20 and 29, respectively. These figures are descriptive counts, not a reported statistical comparison. No confidence intervals or p-values for these safety comparisons are reported in the registry record.
The record also lists urinary tract infection at 9.2%, diarrhoea at 5.4%, pneumonia at 5.4%, blood creatinine increased at 4.9%, cytomegalovirus infection at 4.7%, and acute kidney injury at 4.5% among the top serious terms. The information does not clarify whether these percentages refer to one arm or both arms, so they should not be assigned to a particular regimen. A term-specific percentage is also not necessarily comparable with the overall serious-event count unless the denominator and counting rules are the same.
In general, safety summaries need context about event definitions, follow-up duration, recurrence, severity and exposure time. The registry record does not report those details in the information. Counts alone do not establish a causal relationship with treatment, and they do not show whether differences are statistically precise.
12. What the Result Does, and Does Not, Mean
The TRANSFORM record describes a randomized comparison between everolimus with reduced CNI exposure and mycophenolate with standard CNI exposure in de novo renal transplantation. Its primary outcome is a composite of treated biopsy-proven acute rejection or eGFR below 50 mL/min/1.73m2, assessed primarily at Month 12. This describes the clinical question and endpoint, but it does not provide a numerical primary result. Therefore, the posted information does not establish the size or direction of the primary treatment-group difference, nor how precisely that difference was estimated.
The safety summaries provide affected-participant counts for serious and other adverse events, along with deaths and selected serious-event terms. These numbers characterize what the registry reports, but without an inferential comparison, uncertainty estimates, clearer event definitions and exposure-time information, they do not establish whether one regimen has a different safety profile. The listed serious-event percentages are not identified as arm-specific in the information available, so they cannot be used to compare the regimens.
More generally, a result from a randomized trial applies to the specific population, regimens, outcome definitions, follow-up and analysis used. The record describes adult de novo renal transplant recipients and identifies a full analysis set for the primary outcome, but detailed eligibility criteria and the analysis procedures are not reported in the registry record. The absence of these details limits assessment of how well the findings would generalize to other populations or clinical settings.
These limitations do not demonstrate that the regimens are equivalent, that either is superior, or that the study failed to find a difference. They mean that the available registry information does not contain the primary effect estimate required to support those conclusions. Interpretation of the trial's efficacy question would require the outcome counts or comparative estimate, its uncertainty, component-specific results and analysis details. The statistical record supports a clear description of the trial question and selected safety data, but not a definitive efficacy claim.
13. Why This Trial Matters Statistically
This trial is statistically notable for comparing two treatment strategies that differ in both the named immunosuppressive agent and the intended calcineurin inhibitor exposure. That makes the contrast a comparison of regimens rather than an isolated test of everolimus against mycophenolate with all other treatment features held fixed. The assigned strategy includes the accompanying CNI exposure, so the trial question concerns the overall regimens as described.
The primary endpoint combines a treated rejection event with a threshold-based renal-function outcome. Composite outcomes can capture more than one relevant event, but interpretation depends on the frequency and contribution of each component. In this case, the registry lists related outcomes but does not report the primary comparison or component-specific results. This illustrates why a clear endpoint definition is necessary but not sufficient: numerical results and component detail are needed to understand the evidence.
The study is randomized but open-label. Randomization is an important design feature for reducing confounding in the group comparison, while open-label conduct can matter for treatment decisions and outcomes requiring clinical judgment. The registry record does not describe outcome adjudication or procedures intended to address possible bias. Statistical interpretation must therefore distinguish the design label from implementation details that are not reported.
Finally, the record includes substantial enrollment and several safety summaries, yet the primary efficacy comparison is not reported as a single estimate. A large enrollment figure alone does not establish the precision or conclusiveness of a particular endpoint result. Statistical interpretation depends on the outcome data, analysis method and uncertainty, not only on how many participants entered the study. The record is useful for understanding the planned clinical contrast and endpoints, while also demonstrating the limits of interpreting a completed trial when its primary result and analysis details are unavailable.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The principal limitation for interpreting this trial from the registry record is that no numerical primary-endpoint comparison is reported. The outcome is described, as are its primary and secondary time points, but event counts by group, an effect estimate, a confidence interval and a p-value are not reported. The record also flags that the primary outcome has several categories or time points and is not reduced to one comparison. As a result, efficacy magnitude, direction and precision cannot be assessed.
The primary endpoint is a composite of treated biopsy-proven acute rejection and eGFR below 50 mL/min/1.73m2. The record does not provide the component-specific contribution to the endpoint. It therefore cannot establish whether any hypothetical composite difference would be driven by rejection, renal function or both. Although related secondary measures are listed, their results are not included in the registry record described for this analysis.
Additional limitations include the open-label design and the absence of information on outcome adjudication, randomization procedures, allocation concealment, treatment adherence and changes to assigned regimens. The record identifies the full analysis set but does not define it or explain missing outcome data, withdrawals or sensitivity analyses. It also does not report the detailed statistical analysis plan, multiplicity approach or sponsor sample-size justification. These elements affect assessment of the reliability and robustness of a trial estimate, but their absence from the registry record should not be treated as proof that a particular method was not used.
The safety information is limited to affected-participant counts, deaths and selected serious-event terms. The registry does not report comparative uncertainty, event exposure time or, for the listed percentages, whether they are arm-specific. These limitations prevent a complete comparison of safety outcomes. The record also does not provide further eligibility detail, limiting assessment of how closely the enrolled population represents other transplant settings.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Advancing Renal TRANSplant eFficacy and Safety Outcomes With an eveRolimus-based regiMen (TRANSFORM) — NCT01950819.
- Registry record last updated 2019-01-30; status last verified 2019-01.
- Results first posted 2019-01-30.
- 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
TRANSFORM (NCT01950819) was a completed Phase 4 randomized, parallel-group, open-label study with enrollment of 2037. It compared everolimus with reduced CNI exposure against mycophenolate with standard CNI exposure in de novo renal transplantation. The study start is listed as 2013-12-03 and primary completion as 2017-02-01.
The primary outcome was failure on a composite of treated biopsy-proven acute rejection or eGFR below 50 mL/min/1.73m2. Month 12 was primary and Month 24 secondary; the primary analysis population is described as the full analysis set. The registry record does not report a numerical primary comparison. Safety summaries include serious and other adverse-event counts and deaths, but do not provide comparative uncertainty. The detailed analysis plan, missing-data methods, multiplicity strategy and sample-size justification are not reported in the registry record.