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 After Allo-Hematopoietic Cell Transplant (HCT) in Acute Myelogenous Leukemia (AML) and Myelodysplastic Syndromes (MDS) Subjects With Minimal Residual Disease (NCT02370888), a na phase 1 study. The primary outcome is Maximum Tolerated Dose (MTD) of Lenalidomide.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02370888 |
| Phase | Phase 1 |
| Condition | Leukemia, Myeloid, Myelodysplastic Syndromes |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 11 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Florida |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned people with acute myelogenous leukemia (AML) or myelodysplastic syndromes (MDS) after allogeneic hematopoietic cell transplant (allo-HCT), with minimal residual disease detected by CD34+ mixed chimerism analysis. The registry record does not report further eligibility details or the characteristics of participants enrolled at the tested dose level.
Intervention
The intervention was lenalidomide after allo-HCT. The record describes a planned course of 42 days and lists increasing dose levels, including a first dose level of 2.5 mg by mouth every other day on days 1–21 of each 28-day cycle for two cycles. The dose-level description is incomplete after the start of its third level, and the record says only one dose level was tested.
Comparator
There was no separate comparator group. The study is recorded as single-group, with allocation listed as not applicable and masking as none. Its design therefore does not provide a concurrent control for comparisons of outcomes or harms.
Primary question
The primary question was whether a maximum tolerated dose (MTD) of lenalidomide could be determined, alongside assessment of dose-limiting toxicities and safety, in this post-transplant population. The primary outcome timeframe was up to 72 days. The registry record reports that cohort requirements for analysis were not met, only a single dose level was tested, and no dose escalation was performed; consequently, MTD could not be assessed.
3. Trial Design
This was a phase I, single-group clinical trial of lenalidomide after allo-HCT in people with AML or MDS and minimal residual disease detected by CD34+ mixed chimerism analysis. The registry record lists the University of Florida as sponsor, an enrollment of 11, a start date of 2016-05-16, a primary completion date of 2019-05-31, and a terminated status. The enrollment figure is a registry characteristic; it should not be mistaken for the number included in an analysis of the primary endpoint.
The planned intervention involved increasing lenalidomide doses. The record describes the first dose level as 2.5 mg by mouth every other day on days 1–21 of each 28-day cycle for two cycles. It also gives a second level of 2.5 mg by mouth daily on days 1–21 of each 28-day cycle for two cycles. The description of the third dose level begins with 5 mg daily on days 1–21 of each 28-day cycle but ends before the full regimen is stated. The record reports that only one dose level was tested and that there was no dose escalation, so the planned sequence should not be read as a sequence actually administered.
The study was not randomized, was unmasked, and had no control arm. These features are consistent with an early-phase dose and safety investigation rather than a comparative trial. The record lists the primary outcome as MTD, with an assessment timeframe up to 72 days. Its primary outcome description says that cohort requirements for analysis were not met and that MTD could not be assessed. CD34+ mixed chimerism is listed as a secondary outcome, but results for that outcome are not reported in the registry record. The detailed protocol, dose-escalation rules, and reasons for termination are 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.
Before assignment: Per protocol, enrollment is completion of informed consent. 11 subjects were enrolled- 2 did not meet eligibility and 9 did. Of the 9 eligible subjects- 3 received protocol treatment assignment, 5 did not have their chimerism drop, per protocol, below 90% at day 60 or 90 post-transplant, and 1 withdrew consent prior to assignment.
Overall Study
| Stage | Dose Escalation of Lenalidomide |
|---|---|
| STARTED | 9 |
| Proceed to Treatment | 3 |
| COMPLETED | 1 |
| NOT COMPLETED | 8 |
Reasons for not completing this period:
| Reason | Dose Escalation of Lenalidomide |
|---|---|
| disease progression | 2 |
| did not meet chimerism criteria | 6 |
Analysis population for the primary outcome, as defined in the registry: Cohort requirements for analysis not met. MTD could not be assessed as only a single dose level was tested. No dose escalation was performed.
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: Subsequent cohort requirements for analysis not met.
| Characteristic | Unit | Dose Level 1 | Dose Level 2 | Dose Level 3 | Dose Level 4 | Total |
|---|---|---|---|---|---|---|
| Participants analysed | 3 | 0 | 0 | 0 | 3 | |
| Age, Customized | Participants | 3 | — | — | — | 3 |
| Sex: Female, Male: Gender – Female | Participants | 1 | — | — | — | 1 |
| Sex: Female, Male: Gender – Male | Participants | 2 | — | — | — | 2 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | — | — | — | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 3 | — | — | — | 3 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | — | — | — | 0 |
| Number of Participants with CD34 donor chimerism drop | Participants | 3 | — | — | — | 3 |
6. Primary Endpoint
The registry lists the primary outcome as Maximum Tolerated Dose (MTD) of Lenalidomide, measured over Up to 72 days.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was the maximum tolerated dose of lenalidomide after allo-HCT in people with AML or MDS whose minimal residual disease was detected by CD34+ mixed chimerism analysis. The stated timeframe was up to 72 days. In general, an MTD endpoint is used in early-phase studies to identify a dose that can be carried forward while taking dose-limiting toxicity into account. It is a dose-finding objective, not a measure of comparative treatment benefit.
Interpreting an MTD result depends on how toxicities are defined, which observation period is used, how many participants are assessed at each dose, and what dose-escalation decisions follow the observed toxicities. These operational details and the trial’s dose-limiting toxicity rules are not reported in the registry record. The record instead states that cohort requirements for analysis were not met, only one dose level was tested, and no dose escalation was performed. It therefore explicitly says that MTD could not be assessed.
The record also lists CD34+ mixed chimerism as a secondary outcome. Results for this outcome are not reported in the registry record. Neither the primary MTD endpoint nor the listed secondary outcome can be used from this record to establish a treatment effect.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central result is that the intended dose-finding question was unresolved. The registry record states that cohort requirements for analysis were not met and that MTD could not be assessed because only a single dose level was tested and no escalation occurred. This is not an estimate of an MTD, and it does not identify a dose as tolerable or intolerable according to a completed dose-escalation analysis.
The registry lists an enrollment of 11, but this does not establish how many participants contributed to the primary endpoint. The primary outcome population is described as not meeting cohort requirements for analysis. A reader should distinguish the overall enrollment entry from endpoint-specific analysis; the latter is not reported in the registry record. The tested adverse-event group lists 3 at risk. For that group, the record reports 0 with serious adverse events, 3 with other adverse events, and 0 deaths. Those counts describe the recorded safety entries for the dose level, not the full toxicity profile of every enrolled participant.
The absence of a comparator matters for interpretation. Because the study was single-group and unmasked, its results cannot provide a randomized comparison with another dose, standard care, or no lenalidomide. The record also does not report outcome estimates for the secondary measure, CD34+ mixed chimerism, or statistical analyses. It is therefore not possible to infer an effect on disease status, transplant outcomes, or any other clinical outcome from the listed results.
In general, early-phase dose studies rely on observed toxicities at successive doses to inform dose decisions. Here, the record says no dose escalation occurred, so it does not document a dose-response evaluation. The registry does not report why escalation did not occur, whether a particular safety finding prompted termination, or whether administrative or other factors contributed. Those explanations should not be inferred from the status alone.
Precision is also limited by the lack of a reported endpoint analysis and by the small safety-group count recorded at the tested dose. A confidence interval, where available, would describe uncertainty around an estimated quantity; no such estimate or interval is reported for MTD in this record. The zero serious-event entry should be read as a count in the recorded group, not as proof that serious toxicity is impossible or absent in a broader population. Similarly, the other-adverse-event count indicates that events were recorded, but their nature and severity are not stated in the posted results.
The most defensible interpretation is descriptive: the registry records an intended phase I dose-escalation study, reports testing at one dose level without escalation, and concludes that MTD could not be assessed. It provides limited safety counts for the recorded dose-level group and no reported analysis of the listed secondary outcome. It does not answer whether lenalidomide changes clinical outcomes or how its effects compare with an alternative.
9. Statistical Methods Explained
Statistical methods should be interpreted in light of the objective and the information actually reported. This trial was registered as a phase I, single-group study whose primary endpoint was MTD, not a randomized comparison of clinical outcomes. The registry record does not report a statistical analysis plan, the dose-escalation algorithm, a formal definition of dose-limiting toxicity, or a planned method for selecting the MTD. Those details are important because MTD is not calculated from a single universal formula; the conclusion depends on the protocol’s toxicity criteria and dose-decision rules.
What does dose escalation contribute?
In general, a dose-escalation design observes safety at one dose and uses prespecified rules to decide whether to remain at that dose, move to another dose, or stop. A sequence of tested doses can help describe how toxicity changes across dose levels. A study that tests only one level cannot use observed outcomes across levels to locate a maximum tolerated dose. The registry record explicitly says that only a single dose level was tested and no dose escalation was performed. It also says cohort requirements for analysis were not met. Accordingly, the primary MTD endpoint is reported as not assessed, rather than as a numerical estimate.
How should safety counts be read?
Counts of participants with events are descriptive. The adverse-event record lists a dose-level group with 3 at risk, 0 with serious adverse events, 3 with other adverse events, and 0 deaths. Such counts do not, by themselves, quantify the probability of an event in a wider population or establish a causal relationship to treatment. They also do not show the timing, duration, recurrence, or clinical details of events. The specific serious-event terms are not reported in the registry record. General statistical practice is to interpret an event count together with its definition, observation period, analysis population, and method of collection; several of those details are not reported in the registry record.
What do uncertainty measures add?
In general, a confidence interval conveys a range of values reasonably compatible with an estimate under the assumptions of the analysis. A narrow interval indicates greater precision than a wide interval, while neither guarantees that the underlying assumptions or study design are appropriate. The registry record does not report an MTD estimate, an endpoint-specific analysis population, or a confidence interval for the primary outcome. Without an estimand and an estimate, there is no interval to interpret for MTD. The small recorded safety group also means that raw event counts should not be treated as precise estimates of population risks.
What about power and sample-size illustrations?
General sample-size planning asks how much information a future study might need to detect a specified effect under stated assumptions. Such calculations are planning illustrations, not observed power and not evidence that a trial did or did not succeed. The registry record’s planning assumptions describe an illustration based on an observed effect, two-sided significance level 0.05, equal allocation between two arms, and a normal approximation. That framework is not a description of this single-group trial’s design: there were no two randomized arms in the study. The record does not report an endpoint estimate from which an observed effect for that planning exercise can be evaluated. Any illustration based on those assumptions should therefore be kept separate from conclusions about this trial.
For time-to-event outcomes, general methods often use event counts because the amount of information depends on events as well as enrollment and follow-up. This record does not report a time-to-event analysis for the primary MTD endpoint. The listed secondary outcome is CD34+ mixed chimerism, but results and an analysis method for it are not reported in the registry record. No inference about time to an event or event-free outcomes is warranted from the listed information.
What can be concluded from the analysis record?
The registry record lists two outcome measures and reports no analysis entries. It does not describe statistical tests, adjustment for covariates, handling of repeated measurements, missing-data procedures, or sensitivity analyses. Those methods should not be presumed. The primary result is instead a registry statement about feasibility of assessment: cohort requirements were not met, the study tested one dose level, and dose escalation did not occur. That statement is the relevant endpoint interpretation, rather than an unreported statistical estimate.
For any future dose-finding study, prespecifying toxicity definitions, observation windows, escalation and stopping rules, and analysis populations would make the dose decision transparent. This is a general methodological point, not a description of procedures used in this trial. The registry record does not provide enough information to determine whether such procedures were specified in a separate protocol.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity arises when a study evaluates several outcomes, time points, groups, or statistical hypotheses. In general, testing multiple hypotheses can increase the chance of a false-positive finding unless the analysis plan addresses that issue. The registry record does not report a multiplicity strategy, and it does not report inferential analyses for the listed outcomes. The primary outcome was MTD; CD34+ mixed chimerism is listed as a secondary outcome. The outcome list alone does not show that multiple statistical tests were conducted.
Missing-data handling and analysis populations are also not described in the registry record. The primary outcome text says cohort requirements for analysis were not met, but it does not identify those requirements, state which participants were eligible for an endpoint analysis, or explain whether any observations were missing. The registry enrollment entry of 11 should not be substituted for an endpoint-specific denominator. For the recorded safety group, 3 are listed as at risk; the record does not explain how that safety population was defined or whether it represents all enrolled participants.
The registry lists two outcome measures and reports no analysis entries. It does not report imputation, exclusion rules, a modified or per-protocol population, or sensitivity analyses. These details are not reported in the registry record, so missingness cannot be assumed to be absent, nor can its potential effect on results be quantified. The lack of reported analyses also means there is no documented multiplicity-adjusted or unadjusted result to interpret.
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 |
|---|---|---|---|
| Dose Level 1: 2.5 mg PO QOD Day 1-21 for 28 Day Cycle X 2 | 0 | 3 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Dose Level 1: 2.5 mg PO QOD Day 1-21 for 28 Day Cycle X 2 | 0 of 3 |
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 |
|---|---|
| Dose Level 1: 2.5 mg PO QOD Day 1-21 for 28 Day Cycle X 2 | 3 of 3 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Dose Level 1: 2.5 mg PO QOD Day 1-21 for 28 Day Cycle X 2 |
|---|---|---|
| Blood and lymphatic system disorders | Blood and lymphatic system disorders | 2 of 3 |
| General disorders and administration site conditions | General disorders | 2 of 3 |
| Investigations | Investigations | 2 of 3 |
| Metabolism and nutrition disorders | Metabolism and nutrition disorders | 2 of 3 |
| Skin and subcutaneous tissue disorders | Skin and subcutaneous tissue disorders | 2 of 3 |
| Gastrointestinal disorders | Gastrointestinal disorders | 1 of 3 |
| Infections and infestations | Infections and infestations | 1 of 3 |
| Nervous system disorders | Nervous system disorders | 1 of 3 |
The safety record lists one dose-level group: 3 participants were at risk, 0 had a serious adverse event recorded, 3 had other adverse events recorded, and 0 deaths were recorded. The record does not give specific serious adverse-event terms. These entries are counts for the listed group and should not be interpreted as a complete characterization of safety across all enrolled participants, because the relationship between the safety group and the overall enrollment is not explained.
In general, an adverse-event count describes events observed and recorded under a study’s collection and classification rules. A zero count does not establish that an event could never occur, and it does not provide a precise estimate of risk in other settings. The non-serious or other-event count does not identify event types, severity, duration, or whether events were considered related to lenalidomide. Those details are not reported in the registry record.
These safety figures also do not answer the primary dose-finding question. The record states that MTD could not be assessed because only one dose level was tested and no escalation occurred. The safety counts should therefore be read as descriptive entries, not as confirmation of a maximum tolerated dose.
12. What the Result Does, and Does Not, Mean
The result means that this registry record does not establish a maximum tolerated dose of lenalidomide in the study population. The primary endpoint was not assessed: cohort requirements for analysis were not met, one dose level was tested, and no dose escalation occurred. The trial therefore did not produce the dose-finding conclusion its primary question sought.
The record does document a phase I, single-group study of lenalidomide after allo-HCT in AML and MDS with minimal residual disease detected by CD34+ mixed chimerism analysis. It also reports limited safety counts for the listed dose-level group: 0 serious adverse events, 3 other adverse events, and 0 deaths among 3 at risk. These counts are descriptive and do not establish a comparative safety profile or determine the likelihood of events in a broader population.
The result does not show that lenalidomide improves or worsens disease control, survival, transplant outcomes, or any other clinical endpoint. There was no comparator group, and the registry record reports no analysis of the secondary outcome CD34+ mixed chimerism. It also does not explain why dose escalation did not occur or why the study was terminated. Those omissions limit interpretation but should not be filled by speculation.
In general, an unsuccessful or incomplete dose-finding assessment is different from evidence that a drug is effective, ineffective, safe, or unsafe. It indicates that the intended dose-selection question remains unanswered by the reported study results. The posted information may describe what was planned and some recorded safety counts, but it does not support a treatment-effect estimate or a definitive dose recommendation.
13. Why This Trial Matters Statistically
This study is statistically notable because its primary endpoint was a dose-selection objective, while the registry record says the conditions needed to assess that endpoint were not met. MTD estimation depends on evidence collected across dose levels and on explicit rules for interpreting dose-limiting toxicity. With one tested dose level and no escalation, the key comparison needed to identify a maximum tolerated dose was not made. The record therefore demonstrates why a trial’s achieved conduct must be distinguished from its planned dose sequence.
The study also illustrates the importance of separating enrollment from analysis populations. The registry reports enrollment of 11, yet the primary endpoint population is described as not meeting cohort requirements for analysis. The adverse-event group separately lists 3 at risk. Without an explanation of these populations, an enrollment figure cannot stand in for the denominator relevant to every outcome.
Finally, the record shows the limits of interpreting safety counts in a single-group phase I study. The posted counts provide a narrow descriptive view, but there is no comparator and no reported statistical analysis of the primary or secondary outcomes. The study’s terminated status and the absence of dose escalation make transparency about reasons, protocol rules, and analysis populations especially important; those details are not reported in the registry record. These limitations are not merely technical: they determine which questions the data can answer and which remain open.
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.
Several limitations are specific to the record. The primary endpoint could not be assessed because cohort requirements for analysis were not met; the registry does not state what those requirements were. Only one dose level was tested and no dose escalation occurred, preventing the planned comparison across increasing doses needed for dose finding. The reason that escalation did not occur is not reported in the registry record.
The overall enrollment entry of 11 does not identify the number contributing to primary endpoint assessment. The safety entry lists 3 at risk, but the record does not explain how that group relates to the overall enrollment or define its observation population. Serious adverse-event terms are not listed, and details about the other adverse events are not reported. The secondary outcome CD34+ mixed chimerism has no results reported in the registry record.
The study had no comparator and was unmasked, so the record cannot provide a controlled estimate of treatment effects. Statistical analysis methods, missing-data handling, dose-limiting toxicity criteria, and the dose-escalation plan are not reported in the registry record. The full regimen for the third listed dose level is incomplete in the dose description. The registry status is terminated, but the reason for termination is not reported. These gaps prevent a more complete account of conduct, endpoint analysis, and safety.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Lenalidomide After Allo-Hematopoietic Cell Transplant (HCT) in Acute Myelogenous Leukemia (AML) and Myelodysplastic Syndromes (MDS) Subjects With Minimal Residual Disease — NCT02370888.
- Registry record last updated 2020-01-27; status last verified 2020-01.
- Results first posted 2020-01-13.
- 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 phase I, single-group study of lenalidomide after allo-HCT in people with AML or MDS and minimal residual disease detected by CD34+ mixed chimerism analysis. The primary objective was to assess MTD, dose-limiting toxicities, and safety, with an MTD timeframe up to 72 days. The record reports enrollment of 11, a start date of 2016-05-16, a primary completion date of 2019-05-31, and terminated status.
The primary outcome could not be assessed: cohort requirements for analysis were not met, only one dose level was tested, and no dose escalation occurred. The listed safety group has 3 at risk, 0 serious adverse events, 3 other adverse events, and 0 deaths. CD34+ mixed chimerism is listed as a secondary outcome, but its results are not reported in the registry record. The record does not provide a comparator, statistical analyses, or the reason for termination.