Revlimid® as Consolidation Treatment Chronic Lymphocytic Leukemia: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Revlimid® as Consolidation Treatment Chronic Lymphocytic Leukemia (NCT01600053), a na phase 2 study. The primary outcome is Eradication of Residual Disease From the Marrow.

2. Clinical Question

3. Trial Design

This was a phase II, single-group study of lenalidomide for residual disease in people with chronic lymphocytic leukemia. The registry lists an enrollment of 11 and identifies lenalidomide as the drug intervention. The stated regimen was oral treatment on days 1–21 for up to six 28-day cycles. The brief summary likewise describes a course of six cycles of consolidation treatment.

The design did not include a like-for-like control group: allocation is recorded as not applicable, the model is single-group, and no comparator arm is listed. Masking is recorded as none. These features matter when interpreting the aim. Without a concurrent comparison group, the study was not set up to estimate a randomized treatment contrast. The registry record also does not report a separate control group for comparison with the treatment arm.

The study status is terminated. Its primary completion is recorded as 2014-05, and the start is recorded as 2011-11. The registry’s primary-outcome description states that interim analysis showed the trial would not meet the interim endpoint. It also states that data were not collected from the 11 participants before termination. Thus, the planned clinical question and the evidence actually available in the record are different: the record describes an intended assessment, but does not provide participant-level endpoint data.

The intervention description refers to up to six 28-day cycles, while the primary endpoint timeframe refers to the end of 12 cycles (12 months) or disease progression, whichever came first. These are both stated in the registry, but the record does not explain how the different durations were intended to relate to treatment and endpoint assessment. Further protocol detail, including a fuller account of the design and 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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: This study has been terminated. Interim analysis showed that the trial will not meet the interim endpoint. Data were not collected from the 11 participants before study termination.

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 Eradication of Residual Disease From the Marrow, measured over From date of first dose until then end of 12 cycles of treatment (12 months) or progression of disease, whichever comes first..

The primary endpoint is titled “Eradication of Residual Disease From the Marrow.” Its purpose was to capture whether residual CLL in bone marrow was eradicated after consolidation treatment. In general, a marrow-based endpoint can address disease remaining at a specified site rather than relying only on symptoms or other measures. That is a general explanation of the endpoint type, not a description of results from this study.

The registry gives the timeframe as from the first dose until the end of 12 cycles of treatment (12 months) or disease progression, whichever came first. This timeframe differs from the intervention description, which says lenalidomide would be taken for up to six 28-day cycles. The record does not explain the relationship between those two durations or provide further operational details about when and how marrow status would be assessed.

No unit, measurement parameter, or dispersion measure is listed for the primary outcome. The registry’s endpoint description states that interim analysis showed the trial would not meet the interim endpoint and that data were not collected from the 11 participants before termination. No participant-level endpoint values, response count, or estimate of eradication are reported. Consequently, the endpoint can be described as the intended outcome, but its observed value cannot be summarized from this record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a single-group design estimate?

In general, a single-group study follows one group receiving the intervention and describes outcomes within that group. It does not estimate a randomized difference between treatment and control. This distinction is important because a change observed over time can have more than one explanation; without a concurrent comparison, the study design cannot by itself distinguish those explanations. In this record, the model is single-group, allocation is not applicable, and masking is none. No comparison arm is listed.

That general design explanation should not be mistaken for a description of completed analysis. The registry reports that data were not collected from the 11 participants before termination. There are no posted participant-level observations from which to calculate a descriptive outcome rate, and the analysis count is zero. Thus, the design tells us what kind of study was planned, while the termination and lack of collected data determine what can actually be interpreted.

What would an endpoint estimate and confidence interval mean?

In general, an estimate summarizes the observed outcome in a study, while a confidence interval expresses uncertainty around that estimate under specified statistical assumptions. A narrower interval generally indicates greater precision, and a wider interval indicates less precision. Neither an estimate nor its interval, by itself, establishes a causal treatment effect. In a single-group study, an interval around an outcome rate would describe uncertainty about that group’s observed rate, not a randomized comparison.

The registry does not report an endpoint estimate or confidence interval for marrow disease eradication. It also does not list a unit, measurement parameter, or dispersion measure for the primary outcome. Therefore, there is no numerical interval to explain, and it would be inappropriate to infer precision from the enrollment figure alone. The absence of endpoint data means that the intended outcome cannot be quantified in the posted results.

How should the interim statement be read?

The record states that interim analysis showed the trial would not meet the interim endpoint. In general, an interim analysis is an assessment before the planned endpoint time or final analysis. The registry does not report the interim result, the statistical boundary or decision rule, the timing or details of the analysis, or whether a particular stopping rule was used. Those elements are not reported in the registry record and should not be inferred from the fact of termination.

The statement does not provide an effect estimate, a p-value, or a confidence interval. It also does not say that the primary endpoint was measured for the 11 participants: the same outcome description says data were not collected from them before termination. The available text supports reporting the sponsor’s stated interim conclusion, but not reconstructing its statistical basis.

What are the sample-size illustrations?

In general, a sample-size calculation illustrates how the amount of information needed can depend on assumptions about the outcome, the effect to detect, the significance level, allocation, and analysis method. The planning illustrations are not the study’s observed power and should not be interpreted as proof that the trial could or could not detect an effect. They are calculations under stated assumptions, not observed trial outcomes.

The assumptions described for the illustrations include a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The registry describes this actual study as single-group, with no comparison arm. The assumed equal allocation between two arms therefore does not match the design recorded for this trial. The illustrations are not a sample-size justification for the study; the protocol’s sample-size justification is not reported in the registry record. Since the trial did not collect participant data before termination, any displayed planning scenario should remain separate from claims about observed effects or achieved power.

Why can over-dispersion matter?

In general, over-dispersion means that observed variability is greater than a model expects. When a statistical model assumes less variability than is present, standard errors and uncertainty intervals can be too small, making results appear more precise than the data support. The appropriate model depends on the outcome’s measurement scale and distribution, and on the analysis plan. This is general statistical context; the registry does not report an over-dispersion assessment or a model for the primary endpoint.

More broadly, the statistical analysis plan, model specifications, covariate adjustments, and missing-data procedures are not reported in the registry record. No analysis results are listed. The posted record therefore supports an explanation of general concepts and the study’s stated design, but not a reconstruction of a statistical analysis that is not documented.

10. Multiplicity, Missing Data and Analysis Populations

Multiplicity arises when an analysis considers more than one endpoint, comparison, subgroup, or time point. In general, testing many questions can increase the chance of a false-positive finding unless the analysis plan addresses that issue. This is general statistical context. The registry lists a primary outcome titled “Eradication of Residual Disease From the Marrow” and a secondary outcome of recording the occurrence of adverse events, but does not report a multiplicity strategy, hierarchy of tests, or adjustment method. Those details are not reported in the registry record.

The primary outcome description states that interim analysis showed the trial would not meet the interim endpoint, but the record does not provide the interim result or its analysis details. It does not report whether the interim analysis was planned, what stopping criteria applied, or how any repeated assessment was accounted for. The reason for termination beyond the stated interim conclusion is also not reported in the registry record.

Missing data require particular care in this entry. The record does not merely describe a few unavailable measurements among an otherwise analyzed cohort; it says data were not collected from the 11 participants before study termination. No analysis population, imputation method, or other missing-data approach is reported. It is therefore not possible to determine whether there was an intention-to-treat population, a treated population, or an evaluable population used for an endpoint analysis. These details are not reported in the registry record.

The analysis count is zero, and no primary endpoint estimate is posted. As a result, multiplicity and missing-data methods cannot be evaluated from reported analysis outputs. The registry describes the planned outcomes and a termination statement, but not a completed statistical analysis of those outcomes.

11. Safety Results

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

Other (non-serious) adverse events

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

Most frequent non-serious adverse event terms

The adverse-event summary lists the lenalidomide group with 11 participants at risk for serious adverse events and zero affected. For other adverse events, it lists 11 at risk and 11 affected. These are the counts recorded in the registry. The record does not list any top serious adverse-event terms, so it does not provide details about event types, timing, severity beyond the serious-event classification, or attribution to treatment.

The deaths value is not reported in the registry record. A zero serious adverse-event count should be read as the count shown in this summary, not as proof that serious-event risk was absent. The other-adverse-event count also cannot be interpreted fully without event descriptions and collection details. In particular, the registry does not give a breakdown by event, participant, or treatment exposure, and the trial was terminated before data were collected from the 11 participants for the primary outcome.

These safety figures should be kept separate from the primary endpoint. They do not show whether residual marrow disease was eradicated, and they do not explain the interim conclusion. The limited reporting means that the adverse-event counts can be stated, but cannot support a more detailed assessment of safety patterns.

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

This record documents a phase II study intended to examine lenalidomide as consolidation treatment for residual CLL, with eradication of residual disease from the marrow as the primary endpoint. It also documents that the study was terminated, that interim analysis showed it would not meet the interim endpoint, and that data were not collected from the 11 participants before termination. These registry entries describe the status and stated purpose of the trial; they do not amount to a completed estimate of the endpoint.

The result does not establish an observed rate of marrow disease eradication. No endpoint values, effect estimate, confidence interval, or analysis result are reported. It also does not establish that eradication did not occur. Without collected participant data, the registry record cannot distinguish among possible participant outcomes or quantify their uncertainty.

The single-group design also limits the type of conclusion that would have been possible even if outcome data had been collected. A single group can be described, but it does not provide the randomized comparison needed to estimate a treatment contrast against a concurrent control. In this case, no descriptive primary outcome result is available either, because the record says data were not collected before termination.

The adverse-event counts are separate posted safety information and are limited in detail. They should not be interpreted as efficacy results or as a complete account of safety. The registry does not give event terms for the serious adverse-event summary or a death count.

In short, the record is informative about the planned research question, design, termination, and limits of the posted results. It does not support a conclusion about whether lenalidomide did or did not eradicate residual marrow disease. That distinction between a study’s intended question and the result actually documented is central to reading this entry.

13. Why This Trial Matters Statistically

This trial illustrates why registry status and outcome availability matter when reading clinical research. A named primary endpoint can make a study’s intended question clear, but the existence of that endpoint does not mean that an estimate was obtained. Here, the record identifies marrow disease eradication as the primary outcome, then states that the trial was terminated and participant data were not collected before termination. That makes the absence of an endpoint result part of the statistical interpretation, rather than a minor reporting detail.

It also shows the distinction between a study design and a treatment comparison. The record describes a single-group study, with no listed comparator. Statistical summaries from one group, even if available, would not be equivalent to a randomized estimate of the difference between treatments. In this entry, the lack of collected endpoint data prevents even a descriptive summary of the intended primary outcome.

The interim statement is another useful example of the boundary between what a registry says and what a reader can independently assess. The record reports that interim analysis showed the trial would not meet its interim endpoint, but does not provide the estimate, decision criteria, or analysis details. A careful interpretation can report the statement without inventing its statistical basis.

Finally, the safety summary demonstrates why counts need context. The registry lists separate counts for serious and other adverse events, but offers little detail about the events and does not report deaths. Statistical reading involves conveying these limits alongside the counts, rather than treating a sparse summary as a full safety analysis. Collectively, these features make the record valuable for understanding what was planned and what is documented, while also showing why not every registered study yields interpretable efficacy estimates.

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 that the registry says data were not collected from the 11 participants before study termination. The record consequently contains no reported primary endpoint measurements or analysis results. It states that interim analysis showed the trial would not meet the interim endpoint, but gives no numerical result or supporting analysis details. The reasons for termination beyond that statement are not reported in the registry record.

The study was single-group and had no listed comparator. This limits the kind of treatment contrast that could be estimated from the design. Masking is recorded as none, and the record does not describe further steps intended to reduce assessment bias. The analysis plan, model, covariate adjustment, analysis populations, and missing-data procedures are not reported in the registry record.

There is also a difference between the intervention duration described as up to six 28-day cycles and the primary outcome timeframe described as until the end of 12 cycles (12 months) or progression, whichever came first. The registry does not explain this difference or detail the endpoint assessment schedule. It provides no unit, measurement parameter, or dispersion measure for the primary outcome.

Safety reporting is limited to summary counts. The record lists no top serious adverse-event terms and does not report a death count. It does not provide event descriptions or a detailed account of attribution and timing. These limitations mean the posted entry supports only a narrow account of the trial’s planned question, termination, and summary safety counts.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Revlimid® as Consolidation Treatment Chronic Lymphocytic Leukemia — NCT01600053.
• Registry record last updated 2016-12-15; status last verified 2016-10.
• Results first posted 2016-12-15.
• 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.

18. Record Summary

The registry describes a phase II, single-group study of lenalidomide as consolidation treatment for residual chronic lymphocytic leukemia. The intervention was oral lenalidomide on days 1–21 for up to six 28-day cycles. The primary outcome was eradication of residual disease from the marrow, with a timeframe extending to the end of 12 cycles (12 months) or disease progression, whichever came first.

The study enrolled 11 and is listed as terminated. The record states that interim analysis showed the trial would not meet the interim endpoint and that data were not collected from the 11 participants before termination. No primary endpoint analysis is reported. The adverse-event summary lists zero affected for serious adverse events and 11 affected for other adverse events among 11 at risk in each category; deaths are not reported in the registry record.

The entry therefore documents the planned question and the reason stated for the trial’s outcome status, but it does not provide a completed estimate of marrow disease eradication.