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 Lenalidomide in Previously Treated Patients With Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (NCT00439231), a na phase 2 study. The primary outcome is To Establish the Overall Response Rate Measured at 24 Weeks After First Dose of Lenalidomide Using This Dosing Regimen.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00439231 |
| Phase | Phase 2 |
| Condition | Chronic Lymphocytic Leukemia (CLL), Small Lymphocytic Lymphoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 33 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Georg Aue, M.D. |
| Results posted | Yes |
2. Clinical Question
Population
Participants had chronic lymphocytic leukemia or small lymphocytic lymphoma and had previously received standard treatment. The study description says eligible patients were 21 years of age and older and had relapsed after initial treatment. The registry reports enrollment of 33 participants; further details about the enrolled population, such as disease characteristics or the number with each diagnosis, are not reported in the registry record.
Intervention
Participants received lenalidomide, also identified as Revlimid. The study description says capsules were taken once a day for 21 days, followed by 21 days off treatment, with this schedule constituting a cycle. Treatment was intended to continue for four cycles if tolerated, with continuation after that point depending on response. The actual treatment received by each participant is not reported in the registry record.
Comparator
There was no comparison group. The registry identifies the study as single-group, with allocation listed as not applicable and masking as none. Consequently, the study does not provide a like-for-like comparison of lenalidomide with another treatment, placebo, or no treatment.
Primary question
The primary question was whether an overall response could be established at 24 weeks after the first lenalidomide dose. The endpoint description says response was assessed using peripheral blood measures, lymphadenopathy, hepatomegaly, splenomegaly, constitutional symptoms, and bone marrow biopsy. The number or proportion meeting the response definition is not reported in the registry record, so the primary result cannot be described from this record.
3. Trial Design
This was a phase II, single-group study of lenalidomide in previously treated patients with chronic lymphocytic leukemia or small lymphocytic lymphoma. The registry lists enrollment of 33 and identifies the intervention as lenalidomide. It lists no allocation and no masking. Those features mean that the study was not a randomized comparison and that the participants and investigators were not masked to treatment assignment.
The study description outlines a treatment schedule of capsules once a day for 21 days followed by 21 days off, which constituted one cycle. Participants were to continue for four cycles if the medicine was tolerated. After four cycles, those described as having a complete response were to continue for another two cycles; those with a partial response were to continue for another four cycles; and those without a response were to stop treatment but continue safety follow-up. These are the described treatment rules, not evidence that participants completed the schedule or achieved any particular response.
The primary endpoint was framed around response measured at 24 weeks after the first dose. The record gives no primary outcome result and lists no analyses. The registry records the study as completed, with a start date of 2007-02 and primary completion in 2010-09. Additional operational details, including the actual analysis population and the handling of participants who stopped treatment or did not have an endpoint assessment, are not reported in the registry record.
Because all participants were assigned to the same treatment group, the study can describe outcomes among those treated but cannot by itself separate treatment effects from changes that might also occur over time or from other influences. It also cannot estimate a comparative effect against an alternative treatment. No further design or statistical-plan details are 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 | CLL Subjects Response to Lenalidomide (Revlimid) |
|---|---|
| STARTED | 33 |
| COMPLETED | 5 |
| NOT COMPLETED | 28 |
Reasons for not completing this period:
| Reason | CLL Subjects Response to Lenalidomide (Revlimid) |
|---|---|
| Lack of Efficacy | 28 |
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.
| Characteristic | Unit | CLL Subjects Response to Lenalidomide (Revlimid) |
|---|---|---|
| Participants analysed | 33 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 19 |
| Age, Categorical: >=65 years | Participants | 14 |
| Sex: Female, Male: Female | Participants | 7 |
| Sex: Female, Male: Male | Participants | 26 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 29 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 4 |
| 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 | 4 |
| Race (NIH/OMB): White | Participants | 26 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 3 |
6. Primary Endpoint
The registry lists the primary outcome as To Establish the Overall Response Rate Measured at 24 Weeks After First Dose of Lenalidomide Using This Dosing Regimen, measured over 24 weeks of lenalidomide therapy.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was the overall response rate assessed at 24 weeks after the first dose of lenalidomide. Its description identifies several kinds of evidence used in the response assessment: peripheral blood measures, lymphadenopathy, hepatomegaly, splenomegaly, constitutional symptoms, and bone marrow biopsy. The registry names the outcome as a number of participants, but does not report the observed number meeting the response definition or the overall response rate.
In general, an overall response endpoint is intended to classify whether participants meet criteria for improvement using specified clinical or laboratory findings. Such an endpoint can provide a structured summary across several aspects of disease assessment rather than relying on a single measurement. The particular criteria, thresholds, confirmation rules, and adjudication procedures used for this study are not reported in the registry record, so the exact classification process cannot be reconstructed from the description alone.
A fixed assessment time makes clear when the endpoint is intended to be evaluated. Here, that time is 24 weeks after the first dose. However, the record does not state how participants without an assessment at that time were handled, or whether another assessment could be used. Without the response count and the analysis denominator, the endpoint’s observed result and its precision cannot be evaluated.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a primary response question but does not report the primary response result. It gives the assessment time and the clinical and laboratory domains named in the endpoint description, but no response count, rate, confidence interval, or test result. Therefore, the record does not establish how many participants responded or how precisely a response rate was estimated. A numerical efficacy conclusion cannot be drawn from the posted registry information.
The study enrolled 33 participants and used a single-group design. This gives a description of outcomes within a group receiving lenalidomide, but there is no concurrent control group for comparison. Without such a group, observed changes cannot be attributed to treatment alone. The record lists no masking, so expectations and assessment practices could also be relevant considerations, particularly for components of response assessment that involve clinical judgment. The record does not describe safeguards or independent review procedures.
The listed adverse-event counts are a separate part of the record. Serious adverse events affected 8 of 33 participants, and other adverse events affected 32 of 33 participants. The record also lists tumor flare syndrome at 21.2% and DVT at 3.0% among the top serious terms. These figures describe recorded events in this study group; they do not provide a comparison with another treatment or establish that lenalidomide caused each event. The definitions, observation period, and detailed event-level information are not reported in the registry record.
The enrollment count should not be confused with the number included in a primary endpoint analysis. The actual endpoint denominator and the handling of missing or unevaluable assessments are not reported in the registry record. Likewise, no primary analysis method or statistical test result is listed. Without those details and the endpoint result, it is not possible to assess precision, statistical evidence, or the sensitivity of conclusions to analysis choices.
The overall reading is therefore constrained: the registry documents a phase II, single-group investigation, its intended response assessment, and selected safety counts, but not the primary efficacy result. The record supports describing the design and the reported event counts. It does not support a quantified claim about the response rate or a causal comparison with other care.
9. Statistical Methods Explained
The statistical information in the registry record is limited. It identifies a binary outcome, with the outcome unit expressed as participants and the event meaning described as participants with an event (counts). It does not list any analyses. The primary endpoint title refers to an overall response rate, but the recorded outcome format is a number of participants. No observed response count, denominator for the primary analysis, confidence interval, or test result is reported in the registry record.
What does a response count represent?
In general, a count records how many participants meet a defined outcome criterion. A rate or proportion relates that count to a specified denominator, such as participants assessed for the endpoint. The denominator matters: it determines which participants the result describes. For a response endpoint, the definition must also specify how the different clinical and laboratory findings are combined and how missing assessments are treated. Those operational details are not reported in the registry record. Since neither a response count nor an analysis denominator is posted, a response rate cannot be interpreted from this record.
How would uncertainty be described?
In general, a confidence interval expresses the range of values compatible with an estimate under a specified statistical procedure. It helps readers judge precision: a more precise estimate has a narrower interval, while a less precise estimate has a wider interval. A confidence interval is not a guarantee that the true value lies within its endpoints. No interval for the primary response outcome is reported in the registry record, so its precision cannot be assessed. No p-value for that endpoint is reported either.
What does a p-value tell readers?
In general, a p-value measures how incompatible observed data are with a stated null model, assuming that model and the analysis assumptions are appropriate. It is not the probability that a treatment works, nor does it measure the size or clinical importance of an effect. A p-value also does not replace an estimate and its uncertainty. The registry record lists no analysis results for the primary endpoint, so there is no reported p-value to interpret.
Why does the single-group design matter?
A single-group study can describe outcomes observed among participants receiving the study intervention. It does not supply a concurrent untreated or alternative-treatment group. General causal interpretation depends on comparing what happened under treatment with what would have happened under a suitable alternative; a single group does not observe both conditions. Thus, even if an outcome result were available, this design alone would not establish a comparative treatment effect. The registry explicitly identifies the study as single-group, with allocation not applicable and no masking.
How should the planning illustration be read?
The registry describes a planning illustration based on the observed effect, rather than the trial's own design. Its stated assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is a stated fraction of the observed effect. Such a calculation is a planning exercise, not an observed study result and not observed power. It does not change the fact that the actual study was single-group and had no like-for-like control comparison. The registry record does not report a sample-size justification as part of the original study design. The illustration's numerical outputs are not described in the registry entry summarized by the record.
What is over-dispersion?
In general, over-dispersion occurs when observed variation is greater than a statistical model expects. If a model assumes less variation than is present, its uncertainty estimates can be too small and its inferential results can be misleading. This concept is relevant to some count and rate analyses, but the registry does not report a model for the primary endpoint, an over-dispersion assessment, or any such adjustment. It would therefore be inappropriate to infer that a particular model was used.
What information is missing for a fuller statistical appraisal?
The record does not report the primary analysis population, the statistical test or estimation method, a missing-data strategy, or the handling of participants who discontinued or lacked a 24-week assessment. It also does not report endpoint-level results. These omissions prevent reconstruction of the analysis and evaluation of how robust the result might be to alternative reasonable choices. General statistical principles can explain why these items matter, but they cannot fill in the study's unreported methods or findings.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists one outcome measure and no analyses. It does not report a statistical analysis plan, a hierarchy of endpoints, adjustment for multiple comparisons, or a rationale for whether multiplicity was considered. The primary endpoint is described, but the absence of analysis results means that no conclusion can be made about how it was tested or estimated. Whether other outcomes were collected but not entered in the registry record is not reported in the registry record.
Missing data are also not addressed in the available record. It does not state how participants without a 24-week response assessment were handled, whether they were counted as nonresponders, or whether another method was used. The number included in an endpoint analysis is not reported in the registry record. The study description says that participants who did not respond after four cycles would stop treatment but continue safety follow-up; it does not specify how such participants were handled in the primary endpoint analysis.
The analysis population is not reported in the registry record. For example, the record does not say whether the response analysis included everyone enrolled, only participants who began treatment, or only those with an evaluable assessment. These choices can affect the meaning of a result, so the enrollment count should not be assumed to be the primary endpoint denominator. Without a stated analysis population, missing-data approach, and endpoint results, readers cannot assess the impact of missing assessments or determine whether multiplicity affected interpretation.
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 |
|---|---|---|---|
| CLL Subjects Treated With Lenalidomide (Revlimid) | 8 | 33 | 24.2% |
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 |
|---|---|
| CLL Subjects Treated With Lenalidomide (Revlimid) | 32 of 33 |
Most frequent serious adverse event terms
| Event term | Organ system | CLL Subjects Treated With Lenalidomide (Revlimid) |
|---|---|---|
| Tumor flare syndrome | Blood and lymphatic system disorders | 7 of 33 |
| DVT | Blood and lymphatic system disorders | 1 of 33 |
Most frequent non-serious adverse event terms
| Event term | Organ system | CLL Subjects Treated With Lenalidomide (Revlimid) |
|---|---|---|
| decrease in neutrophils | Blood and lymphatic system disorders | 19 of 33 |
| decrease in platelets | Blood and lymphatic system disorders | 19 of 33 |
| fatigue | General disorders | 19 of 33 |
| upper respiratory infection | Respiratory, thoracic and mediastinal disorders | 16 of 33 |
| skin rash | Skin and subcutaneous tissue disorders | 14 of 33 |
| Tumor flare syndrome | Blood and lymphatic system disorders | 13 of 33 |
| syncope | Nervous system disorders | 12 of 33 |
| dyspnea | Respiratory, thoracic and mediastinal disorders | 10 of 33 |
| diarrhea | Gastrointestinal disorders | 9 of 33 |
| weight loss | General disorders | 9 of 33 |
Serious events with the highest arm-level rate: tumor flare syndrome (21.2%), dvt (3.0%).
The registry reports serious adverse events in 8 of 33 participants and other adverse events in 32 of 33 participants in the lenalidomide group. These are participant counts, not counts of all individual event episodes. The record does not provide event-level detail, definitions, timing, duration, severity beyond the serious-event category, or the observation period used for these summaries. Deaths are not reported in the registry record.
The listed top serious terms are tumor flare syndrome at 21.2% and DVT at 3.0%. These are registry-listed percentages; the record does not provide further detail about how the terms were coded or the individual cases. The counts and terms describe events recorded among participants in this group. They do not, by themselves, show that treatment caused an event or indicate how the event frequency compares with another treatment, since there was no comparison group.
Serious adverse events and other adverse events are distinct reporting categories in the record. The figures should not be combined or treated as measures of the same severity. The registry entry does not provide enough detail to infer whether participants experienced multiple events or to reconstruct a complete safety profile.
12. What the Result Does, and Does Not, Mean
The study record establishes that a phase II, single-group study of lenalidomide enrolled 33 previously treated participants with chronic lymphocytic leukemia or small lymphocytic lymphoma. It specifies an intended primary assessment of overall response at 24 weeks after the first dose and provides selected adverse-event counts. It does not report the primary response count or rate. That missing result is central: no efficacy estimate or measure of its precision can be described from the registry record.
Even if the response result were available, the design would remain noncomparative. With no control group, the observed course of participants could not be directly contrasted with the course under another treatment or no study treatment. The single-group result would describe outcomes in the enrolled group, not establish what caused them. No masking is listed, and the record does not describe further procedures for limiting potential assessment bias.
The safety figures likewise describe only the recorded events in this study group. They do not establish comparative risk, and the record does not contain enough detail to judge event timing, recurrence, or attribution. The event counts should therefore be read as descriptive registry information rather than as a complete account of safety or a causal estimate.
The record also does not state the primary endpoint analysis population, the handling of missing assessments, or the statistical methods used. As a result, the enrollment count cannot automatically be treated as the response-analysis denominator, and the precision or robustness of a response estimate cannot be assessed. The appropriate conclusion is limited to the documented design, intended endpoint, and posted safety counts; the primary efficacy finding is not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of the difference between a planned clinical question and a reported result. The registry identifies overall response at 24 weeks as the primary question, but does not report the response count or rate. A clearly named endpoint does not, on its own, provide evidence about the outcome. Readers need the observed result, its denominator, and an account of uncertainty to evaluate what the study found.
The single-group design also illustrates an important boundary on inference. Descriptive outcomes can characterize participants in the treated group, but without a concurrent comparison group they do not estimate a treatment contrast. This distinction matters even when a study has a defined endpoint and a completed status. The registry lists no allocation and no masking, reinforcing that this was not a randomized, blinded comparison.
The adverse-event information offers another useful statistical distinction: participant counts and percentages describe what was recorded in the study group, but do not establish causation or comparative safety. The registry’s serious-event counts and listed terms cannot stand in for event-level analysis or a control-group comparison. Finally, the record illustrates why analysis populations and missing-data procedures matter. Without them, readers cannot identify the denominator behind an endpoint estimate or assess the consequences of unavailable assessments.
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 efficacy is that the primary response result is not reported in the registry record. Although the endpoint and its assessment time are identified, there is no response count, rate, confidence interval, or analysis result. The primary analysis denominator and analysis population are also not reported, preventing assessment of how many enrolled participants contributed to that endpoint.
The single-group design has no concurrent comparator, and the registry lists no masking. Consequently, the record cannot support a direct comparative estimate, and it does not describe procedures that might reduce bias in outcome assessment. The response definition is described through several clinical and laboratory domains, but the precise criteria, thresholds, confirmation process, and assessment procedures are not reported in the registry record.
There is no reported approach to missing data, discontinuations, or unevaluable response assessments. The treatment description includes response-dependent continuation and stopping, but does not say how these treatment pathways were incorporated into endpoint analysis. The registry also lists no analyses and does not report a statistical analysis plan or multiplicity approach.
Safety interpretation is limited to the posted participant counts and listed serious terms. Detailed event descriptions, timing, duration, attribution, and deaths are not reported in the registry record. These limitations mean the entry supports a description of the study design and selected recorded safety figures, but not a complete efficacy or safety evaluation.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Lenalidomide in Previously Treated Patients With Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma — NCT00439231.
- Registry record last updated 2014-01-13; status last verified 2013-11.
- Results first posted 2014-01-13.
- 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 registry describes a completed phase II, single-group study of lenalidomide in previously treated patients with chronic lymphocytic leukemia or small lymphocytic lymphoma. Enrollment was 33. The record lists no allocation and no masking, and identifies overall response at 24 weeks after the first dose as the primary endpoint.
The endpoint description names peripheral blood measures, lymphadenopathy, hepatomegaly, splenomegaly, constitutional symptoms, and bone marrow biopsy. The primary response result and analyses are not reported in the registry record. Serious adverse events were reported for 8 of 33 participants, and other adverse events for 32 of 33. Tumor flare syndrome at 21.2% and DVT at 3.0% are listed among the top serious terms. These data are descriptive and provide no comparison group.