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 Trial of High Dose Lenalidomide in Patients With MDS and AML With Trilineage Dysplasia (NCT00867308), a non-randomized phase 2 study. The primary outcome is Response Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00867308 |
| Phase | Phase 2 |
| Condition | Myelodysplastic Syndrome |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 32 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins |
| Results posted | Yes |
2. Clinical Question
Population
The trial title and brief summary describe patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) with trilineage dysplasia. The arm descriptions characterize participants as having high-risk MDS, regardless of 5q deletion status. These descriptions are not identical, and the registry record does not further clarify how the eligibility descriptions relate to each other.
Intervention
Participants were assigned to lenalidomide 15 mg or lenalidomide 50 mg. The arm descriptions specify oral dosing on days 1-28 of a 42-day cycle for 2 cycles. The brief summary says participants would receive two cycles and that those who responded might receive additional cycles until disease progression. Details of continuing therapy are incomplete in the arm descriptions.
Comparator
The record identifies two dose groups, but describes the study as nonrandomized and single-group. It does not establish a randomized control group or a like-for-like comparison between the doses. Any differences between the groups therefore should not be interpreted as estimates from a randomized comparison.
Primary question
The primary endpoint is the number of participants with a complete or partial response according to International Working Group 2006 criteria, measured at 15 weeks. The statistical question is whether response was observed in the study population under the trial regimen. The registry record does not report the response count, so it does not permit a numerical answer to that question.
3. Trial Design
This is a phase II trial listed as nonrandomized, with no masking and a single-group model. The registry record identifies two lenalidomide dose arms: 15 mg and 50 mg. Because allocation was nonrandomized, the arms are not a like-for-like control comparison. The record does not explain how participants were allocated between doses or whether allocation followed particular clinical or logistical criteria.
The arm descriptions specify lenalidomide by mouth on days 1-28 of a 42-day cycle for 2 cycles. They describe the participants as having high-risk MDS, regardless of 5q deletion status. The brief summary also refers to patients with AML with trilineage dysplasia and says that participants who respond may receive additional cycles until disease progression. The arm descriptions stop partway through their description of continuing therapy, so the full details are not reported in the registry record.
The listed enrollment is 32. The registry record lists a start in 2009-07, primary completion in 2014-05, and a terminated status. It does not report why the study was terminated or provide a completion explanation. The sponsor is Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins.
The trial's primary outcome is response at 15 weeks. A secondary outcome is Grade 3-4 Toxicity. The record lists two outcome measures, but does not provide a primary response count or a statistical analysis for the primary outcome. The reported safety counts describe participants at risk and affected in each dose group; they do not establish that any event was caused by lenalidomide.
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: 2 participants on the 15mg arm and 3 participants on the 50mg arm were screen failures.
Overall Study
| Stage | Lenalidomide 15 mg | Lenalidomide 50 mg |
|---|---|---|
| STARTED | 9 | 18 |
| COMPLETED | 0 | 0 |
| NOT COMPLETED | 9 | 18 |
Reasons for not completing this period:
| Reason | Lenalidomide 15 mg | Lenalidomide 50 mg |
|---|---|---|
| Death | 1 | 0 |
| Adverse Event | 1 | 4 |
| Lack of Efficacy | 6 | 12 |
| Withdrawal by Subject | 1 | 2 |
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 | Lenalidomide 15 mg | Lenalidomide 50 mg | Total |
|---|---|---|---|---|
| Participants analysed | 9 | 18 | 27 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 3 | 4 | 7 |
| Age, Categorical: >=65 years | Participants | 6 | 14 | 20 |
| Age, Continuous | years | 77 (47 to 88) | 70 (53 to 79) | 72 (47 to 88) |
| Sex: Female, Male: Female | Participants | 1 | 4 | 5 |
| Sex: Female, Male: Male | Participants | 8 | 14 | 22 |
| Race and Ethnicity Not Collected | Participants | — | — | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Response Rate, measured over 15 weeks.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the count of participants with a complete or partial response according to International Working Group 2006 criteria. Its stated timeframe is 15 weeks. It combines complete and partial response into one endpoint, so the measure is the number meeting either response category rather than separate counts for each category. The registry record does not report the count of participants who met the endpoint.
In general, a response endpoint is used to describe whether participants meet prespecified criteria for a change in disease status during a defined observation period. A count can communicate how many participants met the criteria, but interpretation also depends on the population denominator and how response was assessed. The registry record does not report the analysis population, the number assessed for response, or details about assessment procedures beyond naming the criteria.
The stated 15-week timeframe identifies when the outcome is assessed; it does not, by itself, establish that every participant had an assessment at that time. Missing assessments and the handling of participants without an assessment are not reported in the registry record. Because the response result is absent, the endpoint definition alone does not show how many participants responded or how precisely a response rate was estimated.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record describes a phase II study with two lenalidomide dose groups and a primary endpoint based on complete or partial response at 15 weeks. It does not report the primary response count. Consequently, the response finding itself, its precision, and any comparison of response between doses cannot be described from the registry record.
The study is listed as nonrandomized, with no masking and a single-group model. Although two dose groups are named, the record explicitly cautions that these are not a like-for-like control comparison. Any observed difference between the groups, if available elsewhere, could reflect differences in the participants or other factors as well as dose. The registry record does not report an adjusted comparison or an analysis that addresses such differences.
The safety information lists serious adverse events among 3 of 9 participants at risk in the 15 mg group and 9 of 18 in the 50 mg group. Other adverse events are listed among 7 of 9 and 13 of 18 participants, respectively. The record also lists deaths: 3 in the 15 mg group and 2 in the 50 mg group. These counts describe events recorded for the groups; they do not establish the cause of an event, whether it was related to treatment, or whether the groups differ because of dose. The registry record does not give further details about event timing or attribution.
The listed serious event terms include Pneumonia at 16.7%, Weakness - leg at 11.1%, Endocarditis at 11.1%, Epistaxis at 11.1%, Febrile neutropenia at 11.1%, and Hypoxia at 11.1%. The record does not clarify the denominators or group-specific distribution for those percentages in the registry record reported. They should therefore be read as listed event frequencies, not as a basis for comparing dose groups.
No inferential analysis is listed. The record reports zero analyses, and statistical methods for the primary endpoint are not reported in the registry record. These omissions prevent assessment of whether the study's results were tested against a prespecified target or how uncertainty was quantified. The registry status is terminated, but the reason is not reported in the registry record.
9. Statistical Methods Explained
What does the design imply?
In general, random allocation helps balance measured and unmeasured participant characteristics across groups, on average. This trial is listed as nonrandomized, so that protection does not apply. The two dose groups should not be treated as though they were randomized arms of a controlled comparison. Differences between groups could reflect how participants entered or were assigned to each group, alongside any dose-related differences. The registry record does not describe the allocation process or report an adjusted analysis.
The record also identifies a single-group model, even though two dose groups are named. That classification and the nonrandomized allocation matter when interpreting comparisons: the listed groups are not described as a randomized control comparison. The registry record does not report a no-treatment or placebo group. Therefore, counts from this study alone cannot establish what would have happened to the same participants under an alternative intervention.
What does a response count convey?
In general, a binary response endpoint classifies participants according to whether they meet the specified response definition. The primary outcome counts participants with either a complete or partial response under International Working Group 2006 criteria, at 15 weeks. The count is an absolute quantity. To interpret it as a rate, a denominator must also be specified, such as the number of participants eligible for the analysis or the number assessed. The primary outcome record gives no response count or analysis denominator, so a response proportion and its uncertainty cannot be described.
A confidence interval, in general, is a range of values compatible with the observed data under the statistical method used. It communicates precision as well as the point estimate. The registry record does not report a confidence interval, a response estimate, or an analysis method for the primary endpoint. No claim about the precision of the response result is possible from the posted primary outcome information.
What do the safety counts mean?
For each dose group, the safety figures distinguish participants at risk from participants affected. The serious adverse event figures are 3 affected among 9 at risk for lenalidomide 15 mg, and 9 affected among 18 at risk for lenalidomide 50 mg. The other adverse event figures are 7 affected among 9 at risk and 13 affected among 18 at risk, respectively. In general, these are descriptive counts. They do not adjust for differences between groups, account for the timing or duration of exposure, or establish that an event was caused by treatment. Such details are not reported in the registry record.
The death counts are 3 for the 15 mg group and 2 for the 50 mg group. A death count is not, by itself, a treatment-attribution analysis. The record does not state whether deaths were considered related to treatment, when they occurred, or how deaths were handled in the primary response analysis.
How should the planning assumptions be read?
The record describes sample-size illustrations as planning calculations based on the observed effect, not as the trial's own design. It specifies 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 illustrations describe hypothetical sample sizes under those assumptions. They are not the observed number of participants, and they are not observed power. The registry record does not report the trial's sample-size justification, so the illustrations should not be taken as evidence that the study was designed to achieve a particular level of power.
In general, over-dispersion means that observed variability is greater than a statistical model assumes. When relevant, that mismatch can make uncertainty estimates too small if not addressed. The registry record does not report a model for over-dispersion, and the primary endpoint results and analysis methods are absent. No conclusion about whether over-dispersion arose or was addressed can be drawn.
What inferential methods are reported?
The record lists zero analyses. It does not report a hypothesis test, adjustment for covariates, confidence intervals, or a statistical model for the primary outcome. A significance level appears in the sample-size illustration assumptions, but that does not establish that a hypothesis test using that level was conducted for the trial endpoint. In general, a planning assumption and a completed analysis serve different purposes.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists a primary outcome, Response Rate, and a secondary outcome, Grade 3-4 Toxicity. It identifies two outcome measures, but does not report an analysis of the primary response endpoint. It also does not report a statistical testing plan, a hierarchy among outcomes, or any adjustment for multiple comparisons. Multiplicity refers, in general, to the added chance of a false-positive finding when several hypotheses or outcomes are tested. Without the analysis plan and results, it is not possible to determine whether multiplicity was relevant to the reported conclusions or how it was handled.
Missing-data methods are not reported in the registry record. The record does not say how participants without a response assessment at 15 weeks were handled, whether any assessments were missing, or how incomplete safety follow-up was treated. These details matter because an endpoint count can depend on whether the analysis includes all enrolled participants, only those assessed, or another defined group.
The analysis populations are also not reported in the registry record. No intention-to-treat, per-protocol, or safety population is identified for the response endpoint. The listed safety denominators are 9 participants in the 15 mg group and 18 in the 50 mg group, but the record does not explain how those denominators relate to the listed enrollment of 32. The reason for any difference between enrollment and safety denominators is not reported in the registry record.
Because allocation was nonrandomized, the record's lack of an analysis plan also leaves unclear whether any attempt was made to account for differences between dose groups. No adjusted analysis is listed. These gaps limit interpretation of both the response endpoint and comparisons between groups.
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 |
|---|---|---|---|
| Lenalidomide 15 mg | 3 | 9 | 33.3% |
| Lenalidomide 50 mg | 9 | 18 | 50.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Lenalidomide 15 mg | 3 of 9 |
| Lenalidomide 50 mg | 2 of 18 |
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 |
|---|---|
| Lenalidomide 15 mg | 7 of 9 |
| Lenalidomide 50 mg | 13 of 18 |
Most frequent serious adverse event terms
| Event term | Organ system | Lenalidomide 15 mg | Lenalidomide 50 mg |
|---|---|---|---|
| Pneumonia | Infections and infestations | 0 of 9 | 3 of 18 |
| Febrile neutropenia | Infections and infestations | 1 of 9 | 1 of 18 |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | 1 of 9 | 1 of 18 |
| Acute kidney injury | Renal and urinary disorders | 0 of 9 | 1 of 18 |
| Weakness - leg | Nervous system disorders | 1 of 9 | 0 of 18 |
| Clostridium difficile infection | Infections and infestations | 0 of 9 | 1 of 18 |
| Endocarditis | Infections and infestations | 1 of 9 | 0 of 18 |
| Epistaxis | General disorders | 1 of 9 | 0 of 18 |
| Fungal pneumonia | Infections and infestations | 0 of 9 | 1 of 18 |
| Hypotension | Cardiac disorders | 0 of 9 | 1 of 18 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Lenalidomide 15 mg | Lenalidomide 50 mg |
|---|---|---|---|
| Fatigue | General disorders | 5 of 9 | 8 of 18 |
| Pruritis | Skin and subcutaneous tissue disorders | 1 of 9 | 8 of 18 |
| Rash | Skin and subcutaneous tissue disorders | 3 of 9 | 6 of 18 |
| Edema | Cardiac disorders | 1 of 9 | 7 of 18 |
| Constipation | Gastrointestinal disorders | 1 of 9 | 6 of 18 |
| Cough | General disorders | 3 of 9 | 4 of 18 |
| Epistaxis | Blood and lymphatic system disorders | 4 of 9 | 3 of 18 |
| Bleeding | Blood and lymphatic system disorders | 3 of 9 | 3 of 18 |
| Myalgia | General disorders | 0 of 9 | 6 of 18 |
| Weakness | Nervous system disorders | 0 of 9 | 6 of 18 |
Serious events with the highest arm-level rate: pneumonia (16.7%), weakness - leg (11.1%), endocarditis (11.1%), epistaxis (11.1%), febrile neutropenia (11.1%).
The safety figures are counts of participants affected and counts at risk in each dose group. For serious adverse events, the record lists 3 affected among 9 at risk with lenalidomide 15 mg, and 9 affected among 18 at risk with lenalidomide 50 mg. For other adverse events, it lists 7 affected among 9 at risk and 13 affected among 18 at risk, respectively. The record also lists 3 deaths in the 15 mg group and 2 in the 50 mg group.
The listed serious adverse event terms and percentages are Pneumonia, 16.7%; Weakness - leg, 11.1%; Endocarditis, 11.1%; Epistaxis, 11.1%; Febrile neutropenia, 11.1%; and Hypoxia, 11.1%. The registry record does not provide further detail about the denominators, timing, or group-specific distribution for these terms.
These figures are descriptive. They do not show whether events were treatment-related, how long participants were followed, or whether participants could have more than one event. Because the study is nonrandomized, the dose groups are not a controlled comparison of safety.
12. What the Result Does, and Does Not, Mean
The registry record documents a phase II study of lenalidomide in two nonrandomized dose groups and defines a response endpoint at 15 weeks. It also records serious and other adverse event counts and deaths for each group. Those details describe the study and the safety information listed in the record, but the primary response result is not reported.
Without a response count, an analysis denominator, or an uncertainty estimate, the record does not show how many participants met the response criteria or how precisely that outcome was estimated. It also cannot establish whether one dose group had a higher response than the other. Even if a difference were reported elsewhere, nonrandomized allocation would mean that the groups could differ in ways that complicate a causal interpretation.
The adverse event counts likewise do not establish that lenalidomide caused an event or death. The registry record does not report event attribution, timing, or a comparative statistical analysis. Counts across the two dose groups should not be treated as evidence that one dose caused more or fewer events, especially because the allocation was not randomized.
In general, a trial result applies most directly to the population and procedures studied, and broader interpretation depends on details of eligibility, assessment, follow-up, and analysis. The record's descriptions of the population are not fully aligned: the title and brief summary mention MDS and AML with trilineage dysplasia, while the arm descriptions refer to high-risk MDS regardless of 5q deletion status. The registry record does not resolve this difference.
The terminated status is recorded, but the reason is not reported in the registry record. Neither that status nor the listed safety counts can substitute for the missing primary endpoint result or a statistical analysis.
13. Why This Trial Matters Statistically
This trial illustrates why design labels and outcome reporting matter when interpreting clinical data. The record lists two lenalidomide doses, but also identifies nonrandomized allocation and a single-group model. Those features prevent treating the arms as randomized control groups. A numerical difference between the dose groups would not, on its own, isolate the effect of dose.
The primary endpoint is clearly named and tied to a defined timeframe: participants with complete or partial response under International Working Group 2006 criteria at 15 weeks. Yet the response count is not reported. This distinction is important: a well-defined endpoint does not itself provide an outcome result. Without the count, denominator, and uncertainty measure, readers cannot assess the magnitude or precision of the primary finding.
The safety information also shows why denominators and event context are essential. The record gives affected counts and participants at risk, along with selected serious event terms and percentages. But attribution, timing, follow-up, and the distribution of listed event terms by group are not reported in the registry record. Those missing details constrain interpretation of the counts.
Finally, the record lists zero analyses and does not report the primary statistical method, missing-data handling, analysis population, or sample-size justification. This prevents a reader from evaluating how the endpoint was analyzed or whether the analysis followed a prespecified plan. The sample-size assumptions are described as planning illustrations rather than the trial's design. Taken together, the record is useful for identifying the intended question and broad study structure, while leaving the primary efficacy result and important analytical details 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 primary limitation for interpreting efficacy is that the registry record does not report the response count for the primary endpoint. It also does not provide an analysis denominator, confidence interval, or statistical test. The number of participants who were assessed for response at 15 weeks is not reported in the registry record.
The trial is nonrandomized, and the two dose groups are not a like-for-like control comparison. The record does not describe the allocation process or report an adjusted analysis. Therefore, group differences cannot be interpreted as an unbiased estimate of the effect of dose.
The population descriptions differ: the title and brief summary refer to MDS and AML with trilineage dysplasia, whereas the arm descriptions specify high-risk MDS regardless of 5q deletion status. The registry record does not clarify this difference. The arm descriptions also end during the description of continuing therapy, leaving those details incomplete.
The listed enrollment is 32, while the safety counts give at-risk denominators of 9 and 18. The registry record does not explain how these figures relate. It does not report analysis populations, missing-data handling, duration of safety follow-up, event attribution, or the timing of deaths. The reason for the terminated status is also not reported in the registry record.
Finally, no statistical analyses are listed. The record does not report the trial's sample-size justification, interim analyses, or a statistical analysis plan. These omissions limit assessment of both the intended inferential framework and the strength of any conclusions that might be drawn from the posted safety information.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Trial of High Dose Lenalidomide in Patients With MDS and AML With Trilineage Dysplasia — NCT00867308.
- Registry record last updated 2018-10-17; status last verified 2018-09.
- Results first posted 2018-10-17.
- 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
NCT00867308 is a phase II, nonrandomized, unmasked study listed with a single-group model and an enrollment of 32. It evaluates lenalidomide 15 mg and 50 mg in a population described in the title and brief summary as having MDS and AML with trilineage dysplasia. The arm descriptions refer to high-risk MDS regardless of 5q deletion status.
The primary outcome is the number of participants with complete or partial response by International Working Group 2006 criteria at 15 weeks. The response count and statistical analyses are not reported in the registry record. Safety counts are listed by dose group, and the record lists Grade 3-4 Toxicity as a secondary outcome. The study is marked terminated; the reason is not reported in the registry record.