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 Bortezomib, Lenalidomide, and Dexamethasone in Treating Patients With Multiple Myeloma Undergoing Stem Cell Transplant (NCT02353468), a na phase 2 study. The primary outcome is Event Free Survival Rates by Land-mark Analysis.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02353468 |
| Phase | Phase 2 |
| Condition | Refractory Plasma Cell Myeloma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 3 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Colorado, Denver |
| Results posted | Yes |
2. Clinical Question
Population
Patients with symptomatic multiple myeloma following autologous transplantation are the population described in the official study title. The condition is listed as refractory plasma cell myeloma, while the brief summary describes patients with multiple myeloma undergoing stem cell transplant. Detailed eligibility criteria and the characteristics of enrolled participants are not reported in the registry record.
Intervention
The study examines a regimen of bortezomib, lenalidomide, and dexamethasone. Its official title describes treatment with the combination followed by lenalidomide and dexamethasone as post-transplant consolidation and maintenance. The registry describes a consolidation regimen, but the full treatment schedule and the details of later treatment are not reported in the registry record.
Comparator
There is no separate comparison group in this single-group study. Allocation is listed as not applicable, and masking is listed as none. The record therefore does not support a like-for-like comparison between the regimen and another treatment or control condition.
Primary question
The stated primary endpoint is event-free survival rates by landmark analysis, with a timeframe of up to 5 years. The registry says the study was terminated and the endpoints were not reached. It does not report event-free survival estimates, the landmark time points, or a comparison of outcomes between groups.
In statistical terms, the study was intended to describe outcomes after treatment in the transplant setting, rather than estimate a contrast between randomized treatment groups. A single-group outcome can describe what happened among the participants observed, but it cannot isolate the effect of a regimen from other influences, such as participants’ clinical characteristics, other care, or the course of disease. Those influences cannot be assessed from the posted endpoint information.
The enrollment listed in the record is 3. That small enrolled group and the fact that the stated endpoints were not reached sharply limit what can be learned about the planned survival outcome. No participant-level outcome data or endpoint estimates are reported in the registry record, so the primary question cannot be answered from the posted results.
3. Trial Design
This is a phase II, single-group study sponsored by the University of Colorado, Denver. It concerns bortezomib, lenalidomide, and dexamethasone in patients with multiple myeloma in the setting of stem cell transplantation. The official title describes combination therapy followed by lenalidomide and dexamethasone as post-transplant consolidation and maintenance. The registry lists the condition as refractory plasma cell myeloma and describes the intended population as patients with symptomatic multiple myeloma following autologous transplantation.
The design has no separate control arm: allocation is recorded as not applicable, and the model is single group. Masking is recorded as none. Thus, the registry does not describe a randomized comparison or a blinded assessment. Any outcome description would concern the participants treated in the study, not a contrast between independently assigned groups.
The registry lists enrollment as 3 and status as terminated. The start and primary completion dates are recorded as 2009-12 and 2011-10. The record does not explain why the study was terminated. It also does not state whether treatment was completed by all enrolled participants or give participant flow, follow-up completeness, or reasons for discontinuation.
The primary outcome is event-free survival rates by landmark analysis, with a timeframe of up to 5 years. Its description says that a Kaplan-Meier curve would have been used to describe the distribution. The population field states that the study was terminated and study endpoints were not reached. Accordingly, the registry does not present the planned endpoint estimates or a survival curve.
The registry lists 1 outcome measure and 0 analyses. It does not report a detailed statistical analysis plan, a sample-size justification, or the planned definition of an event for event-free survival. These omissions matter for interpreting how the endpoint was to be constructed and analyzed. The record also does not report whether any protocol changes occurred or what analyses, if any, were completed before termination.
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: 35 days following autologous transplant, patients were evaluated for eligibility by the standard tests, including paraprotein assessment, bone marrow biopsy and aspiration, MRI scan, and PET/CT scan that is indicated for patients with hypo- or non-secretary myeloma.
Overall Study
| Stage | Enzyme Inhibitor, Biological Therapy, Chemotherapy |
|---|---|
| STARTED | 3 |
| COMPLETED | 0 |
| NOT COMPLETED | 3 |
Reasons for not completing this period:
| Reason | Enzyme Inhibitor, Biological Therapy, Chemotherapy |
|---|---|
| Study terminated | 3 |
Analysis population for the primary outcome, as defined in the registry: The study was terminated, study endpoints were not reached.
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: The study had planned to enroll sixty-two patients in order to achieve sufficient power, 81% to detect an improvement in 3-year event-free survival.
| Characteristic | Unit | Enzyme Inhibitor, Biological Therapy, Chemotherapy |
|---|---|---|
| Participants analysed | 3 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 3 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | Years | 54.3 (SD 3.8) |
| Sex: Female, Male: Female | Participants | 2 |
| Sex: Female, Male: Male | Participants | 1 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 3 |
| Race (NIH/OMB): White | Participants | 0 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 3 |
6. Primary Endpoint
The registry lists the primary outcome as Event Free Survival Rates by Land-mark Analysis, measured over up to 5 years.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
Event-free survival is a time-to-event outcome: in general, it describes the time until a participant experiences a prespecified event. The exact events that would count for this study are not reported in the registry record. Without that definition, readers cannot determine which clinical occurrences would trigger an event or how deaths, progression, or other outcomes would be handled.
The registry names the primary endpoint as event-free survival rates by landmark analysis and gives a timeframe of up to 5 years. A landmark estimate describes the proportion of participants who remain event-free at a specified time point. The particular landmark time points are not reported in the registry record. The description says a Kaplan-Meier curve would have been used to describe the distribution. In general, this method uses information from participants’ follow-up times and can account for observation ending before an event is recorded, provided censoring and event information are available.
Time-to-event endpoints are often used when both whether an event occurs and when it occurs are relevant. They can retain information about follow-up timing rather than reducing outcomes to a simple event/no-event classification over an unspecified period. That general advantage depends on having adequate event and follow-up data, a clear endpoint definition, and a prespecified analysis approach.
For this study, the registry states that the endpoints were not reached after termination. It reports no landmark estimates, event counts, follow-up distribution, or Kaplan-Meier results. Therefore, the intended endpoint cannot be interpreted as an observed estimate of event-free survival from the posted results.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The main interpretive fact is that the registry does not report results for the primary endpoint. It states that the study was terminated and that study endpoints were not reached. Although the planned endpoint was event-free survival rates by landmark analysis, no event-free survival rate, event count, median, confidence interval, or curve is reported. Readers therefore cannot assess the magnitude or precision of the intended outcome from this record.
The enrollment is 3, but enrollment alone does not establish what happened to participants or how much follow-up was available. The record does not report participant-level data, the number with evaluable endpoint information, or the duration of follow-up. It is not possible to infer that participants had no events merely because endpoint results are absent. Nor does the record establish that the planned time horizon was completed for any participant.
The study’s single-group design also constrains interpretation. Even if an outcome estimate had been reported, it would describe outcomes in the treated group and would not, by itself, show how outcomes would have differed under another treatment or no study regimen. Since there is no comparator group and no reported endpoint estimate, neither a comparative treatment effect nor a precise within-group survival description can be drawn from the posted results.
The safety record lists 3 participants at risk for serious adverse events and 0 affected. It also lists 3 at risk for other adverse events and 0 affected. These are registry counts, not evidence that the regimen has no safety risks: the enrolled group is small, and the record does not give a detailed reporting window, event definitions, or participant-level safety information. The death count is not reported as a numeric value, so it should not be read as zero.
The record’s status is terminated, but the reason for termination is not reported in the registry record. No conclusion about the reason, the conduct of the study, or the clinical implications of termination should be inferred from status alone. The posted information supports a narrow conclusion: the registry describes the study’s intended question and design, but it does not provide the planned primary outcome results.
Statistical precision cannot be judged without endpoint estimates and their uncertainty. For a time-to-event outcome, confidence intervals would ordinarily communicate how compatible the data are with a range of underlying survival values. No such interval is reported. The absence of an estimate is not an estimate with wide uncertainty; it is a lack of reported endpoint results.
9. Statistical Methods Explained
What does the single-group design allow?
In general, a single-group study describes outcomes among participants who receive the study regimen. It can provide a record of treatment and observed outcomes when those outcomes are collected and reported. It does not create a control group for estimating what would have happened to comparable participants under another treatment. Differences between an observed outcome and an external expectation may reflect factors other than treatment, so a single-group design cannot establish a comparative causal effect on its own.
For this study, the registry identifies a single-group model, no masking, and allocation as not applicable. Those design features mean there is no randomized arm contrast to interpret. The record does not report a formal causal or comparative analysis, and it lists 0 analyses. The primary endpoint was not reached according to the registry, so the record provides no endpoint data with which to describe the planned outcome.
How would a Kaplan-Meier analysis work in general?
Kaplan-Meier estimation is a general method for describing time-to-event data. It combines the timing of observed events with the time participants were known to remain under observation. A participant who has no recorded event before follow-up ends can contribute information through the last known follow-up time; that observation is generally treated as censored rather than as proof that an event never occurred.
That approach relies on accurately recorded event times, follow-up times, and censoring. It also relies on a defined event and a defensible assumption about how censoring relates to the event process. The registry says a Kaplan-Meier curve would have been used, but does not report an actual curve, event or censoring counts, or the endpoint definition. Therefore, readers cannot examine whether the required information was available or evaluate the resulting estimates.
Landmark analysis refers generally to reporting an outcome at specified time points. A landmark rate is interpretable only when the time point, the outcome definition, and the relevant population are clear. The registry gives a timeframe of up to 5 years but does not identify the particular landmark times or provide rates. It also does not report how participants with incomplete follow-up would have been handled in a landmark summary.
What does a confidence interval convey?
In general, a confidence interval expresses statistical uncertainty around an estimate under the assumptions of the analysis. A narrower interval indicates greater precision in the estimate, while a wider interval indicates that the data are compatible with a broader range of values. A confidence interval does not remove design limitations, establish causation, or account for sources of bias that were not included in the analysis.
No confidence interval or endpoint estimate is reported for this study. Because the primary outcome results are absent, it is not possible to describe the precision of the intended survival analysis. Enrollment by itself cannot substitute for an estimate and its uncertainty: precision depends on the relevant outcome and follow-up information, as well as the analysis.
How should the planning illustrations be read?
The planning assumptions describe an illustration based on an effect, with a two-sided significance level of 0.05, equal allocation between two hypothetical arms, and a normal approximation. The stated effect to detect is a fraction of the observed effect. For time-to-event planning, the assumptions specify total events; for other outcomes, they specify participants per arm. These are planning conventions, not evidence that the trial enrolled or compared participants in two arms.
Such sample-size calculations are prospective illustrations of the amount of information that might be needed under their assumptions. They are not observed power, do not show that a study had a particular probability of detecting an effect after results are known, and do not establish that the assumptions match a trial’s original design. In this registry record, the primary endpoint results are not reported and no sample-size justification is reported. The illustration therefore should not be treated as a trial-specific analysis plan or as a substitute for endpoint data.
Over-dispersion is a general concern for some count outcomes: the observed variability can exceed what a simpler statistical model assumes. When that happens, methods that fail to account for the extra variability can understate uncertainty. This is a general statistical consideration, not a reported feature or analysis of this study. The registry does not describe a count outcome or report a model addressing over-dispersion.
What can be concluded from the posted analysis information?
The registry lists 1 outcome measure and 0 analyses, and says the endpoints were not reached. It does not report an analysis population, missing-data method, censoring rules, adjustment variables, or a complete statistical plan. These gaps prevent readers from reproducing or auditing the planned endpoint analysis. Statistical methods can explain what a Kaplan-Meier analysis or a confidence interval would mean in general, but they cannot supply the absent study results or resolve the limitations of a single-group design.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity concerns arise when a study tests or reports multiple outcomes, time points, treatment comparisons, or subgroup analyses. In general, testing many questions can increase the chance of observing a nominally unusual result by chance. Prespecified priorities and appropriate statistical procedures can help readers distinguish a primary analysis from additional exploratory analyses.
The registry lists 1 outcome measure and 0 analyses. The primary endpoint is event-free survival rates by landmark analysis, but the landmark time points and the endpoint event definition are not reported in the registry record. The record does not describe a multiplicity adjustment, a hierarchy among outcomes, or whether any additional analyses were planned. It would be inappropriate to infer that multiplicity was or was not controlled beyond what the registry states.
Missing data are also important for time-to-event outcomes. In general, follow-up can end before an event is observed, and the analysis needs to distinguish this censoring from an observed event. Other missing information, such as an unknown event status or absent follow-up, may require separate consideration. The registry does not report missingness, censoring counts, procedures for handling missing data, or follow-up completeness. It says only that the study was terminated and endpoints were not reached.
The analysis population is not described in the posted results. The enrollment is 3, but the record does not say which participants would have been included in an endpoint analysis, whether any were excluded, or how deviations from the intended treatment were handled. These details are not reported in the registry record. As a result, neither the population underlying a primary endpoint estimate nor the effect of missing follow-up can be evaluated.
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 |
|---|---|---|---|
| Enzyme Inhibitor, Biological Therapy, Chemotherapy | 0 | 3 | 0.0% |
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 |
|---|---|
| Enzyme Inhibitor, Biological Therapy, Chemotherapy | 0 of 3 |
The safety record lists 3 participants at risk for serious adverse events and 0 affected. For other adverse events, it likewise lists 3 at risk and 0 affected. These counts describe what is recorded in the registry; they do not establish that no adverse events occurred outside the reported categories or reporting period.
The record does not describe the safety observation window, adverse-event definitions, collection procedures, severity grading, or completeness of reporting. It also does not list any top serious adverse-event terms. Those omissions limit interpretation of the zero affected counts, particularly in a study with enrollment of 3. A small recorded group cannot rule out uncommon events or provide a precise estimate of event frequency.
Deaths are listed with no numeric count. That missing value is not equivalent to a recorded count of 0. The record also does not provide participant-level safety information or a detailed account of events leading to treatment discontinuation. The counts should therefore be read narrowly as registry-reported figures, not as a comprehensive assessment of safety.
12. What the Result Does, and Does Not, Mean
The registry record documents a phase II, single-group study of bortezomib, lenalidomide, and dexamethasone in the stem cell transplant setting for multiple myeloma. It identifies event-free survival rates by landmark analysis as the primary endpoint and says a Kaplan-Meier curve would have been used. It also records that the study was terminated and the endpoints were not reached.
What the record does not provide is equally important: no event-free survival estimate, landmark rate, event count, curve, or uncertainty interval is reported. The posted results therefore do not establish what event-free survival was among participants. The absence of outcome results should not be interpreted as evidence of no events or as evidence that treatment did or did not change outcomes.
Because the design is single group, there is no concurrent comparator for estimating a treatment contrast. Even with a reported outcome, an observed result would describe the study participants and would not, by itself, show what would have happened under another treatment. The registry also does not report why the study was terminated, so the status cannot be used to infer the reason.
The safety counts show 0 affected for serious adverse events and 0 affected for other adverse events among the recorded at-risk groups, each listed as 3. These figures are limited by the absence of details about reporting and follow-up. The death count is not reported numerically. The record therefore does not support a broad conclusion about safety.
Overall, the registry provides information about the planned clinical question, design, and endpoint, but not the primary outcome results needed to assess the intended survival question. Any interpretation should remain within those limits.
13. Why This Trial Matters Statistically
This study illustrates why a registry’s design information and outcome results need to be read separately. The record identifies a clinically relevant time-to-event endpoint and a Kaplan-Meier approach, but it also states that the study was terminated before the endpoints were reached. A planned method is not the same as an observed analysis, and a named endpoint is not evidence that an estimate was obtained.
The single-group design is another important statistical feature. Without a separate comparison group, outcomes cannot be interpreted as a randomized treatment contrast. That distinction remains important even when an intervention and a follow-up outcome are clearly specified. Design determines which questions the data can address; analysis cannot create a control group that the study did not have.
The record also shows how a small enrollment and absent endpoint results affect precision and interpretation. Enrollment is recorded as 3, while the primary outcome estimates and their uncertainty are not reported. Readers cannot assess the amount of event information, follow-up, or precision. A planning illustration cannot fill that gap: it is not observed power and does not replace an endpoint estimate.
Finally, the safety figures show why counts require context. Zero affected is a reported count for the listed categories, but the record does not describe reporting windows or completeness, and the death count is not numeric. Statistical reading therefore depends not just on the value, but on who was observed, for how long, and what was counted. Those details are not reported in the registry record.
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 primary limitation is that the registry states the study was terminated and the endpoints were not reached. It provides no event-free survival rates, event counts, estimates of uncertainty, or Kaplan-Meier results. The reason for termination is not reported in the registry record, so no explanation should be inferred.
Enrollment is listed as 3, and the study has no comparator group. This limits the amount and type of information the study could provide: the record does not support a treatment comparison, and it reports no endpoint data for a descriptive survival estimate. Participant characteristics, treatment completion, follow-up duration, and participant flow are not reported in the registry record.
The primary endpoint is not fully operationalized in the record. It does not specify which events count toward event-free survival or identify the landmark time points. It describes a Kaplan-Meier curve as the planned way to describe the distribution, but no event or censoring information is available to evaluate how that method was applied.
Statistical analysis details are limited. The record lists 0 analyses and does not report an analysis population, missing-data approach, censoring rules, multiplicity procedures, or a detailed statistical plan. It also does not report a sample-size justification. The planning assumptions are illustrative and should not be treated as the original design or as observed power.
Safety reporting has additional limitations. The registry lists 0 affected for serious and other adverse events, with 3 at risk in each category, but provides no reporting window or detailed event definitions. The death count is not reported as a numeric value. These gaps prevent a fuller assessment of safety information.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Bortezomib, Lenalidomide, and Dexamethasone in Treating Patients With Multiple Myeloma Undergoing Stem Cell Transplant — NCT02353468.
- Registry record last updated 2015-03-09; status last verified 2015-01.
- Results first posted 2015-03-09.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
The record describes a phase II, single-group study sponsored by the University of Colorado, Denver, of bortezomib, lenalidomide, and dexamethasone in the setting of autologous stem cell transplantation for multiple myeloma. It lists enrollment as 3, masking as none, allocation as not applicable, and status as terminated.
The primary endpoint is event-free survival rates by landmark analysis, with a timeframe of up to 5 years. The registry says a Kaplan-Meier curve would have been used, but also states that the study was terminated and the endpoints were not reached. No primary endpoint results are reported.
The safety record lists 0 affected for serious adverse events and 0 affected for other adverse events, with 3 at risk in each category. The death count is not reported numerically. The registry does not report why the study was terminated or provide a detailed statistical analysis plan.