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 Phase I/II Trial of the Combination of Lenalidomide (Revlimid) and Nab-paclitaxel (Abraxane) in the Treatment of Relapsed/Refractory Multiple Myeloma (NCT02075021), a na phase 1, phase 2 study. The primary outcome is Maximum Tolerated Dose (Phase I).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02075021 |
| Phase | Phase 1, Phase 2 |
| Condition | Relapsed or Refractory Multiple Myeloma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 3 |
| Arms in the primary outcome | n/a |
| Lead sponsor | NYU Langone Health |
| Results posted | Yes |
2. Clinical Question
Population
People with relapsed or refractory multiple myeloma. The registry record reports enrollment of 3 participants.
Intervention
A combination of lenalidomide (Revlimid) and nab-paclitaxel (Abraxane). The described regimen used Abraxane weekly for 3 weeks and Revlimid daily for 21 days in a 4-week cycle, with planned dose escalation and dose de-escalation as needed.
Comparator
There was no separate comparator group. The registry record identifies a single-group study, with allocation listed as not applicable and no masking.
Primary question
The Phase I primary outcome concerned the maximum tolerated dose, assessed through dose-limiting toxicity over 4 weeks using CTCAE version 4.0. The registry record says the results were not analyzed because there was insufficient data. A secondary Phase II outcome was the number of participants achieving complete or partial response, but no response result is reported.
The clinical question was therefore a dose-finding question first, with a planned response assessment in Phase II. It was not a comparison of the combination against another treatment or a control group. The record describes the study as currently in the Phase I stage in its summary, while its status is terminated. It does not report an analyzed estimate of the maximum tolerated dose or a Phase II response result. Consequently, the posted information does not establish what dose was tolerated or how often a response occurred.
3. Trial Design
This was a Phase I/II, single-group trial of lenalidomide plus nab-paclitaxel in relapsed or refractory multiple myeloma. The registry lists the study as terminated and reports enrollment of 3. The listed start is 2014-03, and the primary completion is 2015-11. Allocation is recorded as not applicable, and masking is listed as none; there was no separate control group.
The described treatment used 100 mg/m^2 of Abraxane weekly for 3 weeks and Revlimid daily for 21 days, beginning at 10 mg with planned escalation to 15 mg and 25 mg. One cycle was 4 weeks. The description also says dose de-escalations could be performed for both drugs as necessary, and treatment would continue until disease progression. The summary describes standard cohorts of 3 for escalation and a planned expansion to 25 participants at the dose below the maximum tolerated dose for a pilot Phase II trial. The registry record does not establish that this expansion occurred.
The summary describes assessments at least at the 2, 4 and 6 month timepoints, then monthly until progression. The primary outcome was maximum tolerated dose over 4 weeks, based on dose-limiting toxicity assessed using CTCAE version 4.0. A secondary outcome concerned the number achieving complete or partial response in Phase II. The record reports no outcome analyses and says the primary results were not analyzed because of insufficient data; it also notes that the principal investigator left the institution.
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 | Lenalidomide and Nab-paclitaxel |
|---|---|
| STARTED | 3 |
| COMPLETED | 3 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Results were not analyzed due to lack of sufficient data. PI left the institution.
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: 3 Subjects accrued and completed follow up. Data was not analyzed because PI left institution.
| Characteristic | Unit | Lenalidomide and Nab-paclitaxel |
|---|---|---|
| Participants analysed | 3 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 3 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 2 |
| Sex: Female, Male: Male | Participants | 1 |
| Region of Enrollment: United States | Participants | 3 |
6. Primary Endpoint
The registry lists the primary outcome as Maximum Tolerated Dose (Phase I), measured over 4 weeks.
| 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 maximum tolerated dose in Phase I, with a 4-week timeframe. The registry description links this outcome to dose-limiting toxicity assessed using CTCAE version 4.0. In general, a dose-finding endpoint helps characterize the highest dose considered tolerable under a study’s specified rules. Toxicity grading provides a structured way to classify adverse events, while the study’s dose-escalation rules determine how those observations inform dose selection.
The registry record does not report the dose-limiting toxicity observations, the rules used to classify a dose as the maximum tolerated dose, or an estimated maximum tolerated dose. It states that results were not analyzed because of insufficient data. Therefore, the endpoint’s purpose can be described, but its value cannot be inferred from the posted results.
The record also names a Phase II secondary outcome: the number of participants who achieve complete response or partial response. Such response outcomes describe tumor response under specified criteria, but the response criteria and an analyzed result are not reported in the registry record. This secondary outcome does not replace the primary dose-finding endpoint.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central result is a lack of an analyzed primary endpoint, not an estimate of a tolerated dose. The registry record explicitly says that the maximum-tolerated-dose results were not analyzed because of insufficient data and notes that the principal investigator left the institution. No dose-limiting toxicity count, dose-specific safety summary, or maximum tolerated dose is reported. The planned dose-escalation approach therefore cannot be evaluated from these results.
The record reports enrollment of 3, but this figure alone does not show how many participants received each dose, completed the 4-week assessment, or contributed evaluable toxicity data. Those details are not reported in the registry record. Without them, readers cannot assess how much information was available at each dose level or whether observed toxicities could have been attributed to a particular regimen level.
The secondary outcome was the number achieving complete or partial response in Phase II. No response count or response analysis is reported. The summary’s description of a planned Phase II expansion does not demonstrate that the expansion took place, and the record’s summary says the trial was in Phase I. The posted information therefore does not support an estimate of response frequency.
The adverse-event record lists 0 serious adverse events, 0 other adverse events, and 0 deaths for the treatment group, with 3 at risk for each event category. These are the recorded counts, not proof that the regimen caused no adverse events. The registry does not report event definitions beyond the serious and other categories, observation completeness, or participant-level follow-up for safety. Zero recorded events in a small, incompletely characterized dataset offer limited information about the range or frequency of potential harms.
Because this was a single-group study without masking, there is no concurrent control comparison. Even if an outcome estimate had been reported, a single-group result would describe participants receiving the combination rather than isolate its effect relative to another treatment. The posted record contains no inferential analysis, uncertainty interval, or treatment-effect estimate. Any interpretation should remain descriptive and account for the absence of analyzed efficacy and dose-finding results.
9. Statistical Methods Explained
What does a single-group design estimate?
In general, a single-group study observes outcomes among participants receiving a study regimen without a concurrent comparison group. It can describe what happened in the enrolled group, provided the relevant outcomes are collected and analyzed. It does not directly estimate the difference between the regimen and an alternative. Without a comparator, changes over time cannot be separated from other influences using a randomized contrast.
This registry record identifies a single-group design and no masking. It does not report an analyzed clinical outcome or a comparative effect estimate. The study’s primary aim was dose finding, rather than a randomized comparison. The fact that the registry lists a response outcome does not create a comparison group or establish a treatment effect.
How does dose escalation inform a maximum tolerated dose?
In general, dose-finding studies use rules to guide whether dose levels are escalated, maintained, or reduced in response to observed toxicities. A maximum tolerated dose is an outcome of applying those rules to the available safety observations. It is not simply the highest dose administered. The result depends on how dose-limiting toxicities are defined, the observation period, which participants are evaluable, and the decision rules specified for the study.
The registry summary describes cohorts of 3 and planned escalation of Revlimid, with dose de-escalations for both drugs as necessary. It also describes a 4-week primary-outcome timeframe and CTCAE version 4.0 for toxicity assessment. However, the registry record does not report the operational dose-limiting toxicity definition, the complete escalation algorithm, evaluability rules, or the observations at each dose. Most importantly, it says the primary results were not analyzed because of insufficient data. The maximum tolerated dose cannot be reconstructed from the planned design description alone.
What does an uncertainty interval tell a reader?
In general, a confidence interval expresses the statistical uncertainty around an estimate under a specified analysis and its assumptions. A narrower interval indicates greater precision under that method; a wider interval indicates less precision. An interval is not a guarantee that the true value lies within its endpoints, and its interpretation depends on how the data were collected and modeled.
No confidence interval, analyzed primary estimate, or inferential result is reported in the registry record. Thus, there is no precision estimate to interpret for the maximum tolerated dose or the response outcome. The absence of an interval is not evidence that an outcome is precisely known; it reflects that the relevant result is not reported.
Why can small datasets be difficult to interpret?
In general, estimates based on limited observations can be highly sensitive to individual outcomes. This is especially important for uncommon events and dose-finding outcomes, where a small change in the observed toxicity pattern may affect a dose decision. A small enrollment figure also does not reveal the amount of usable data for a particular endpoint. Participants may not all contribute outcome information at the same time or in the same way, and those details must be reported to assess the analysis.
The registry reports enrollment of 3, but does not give the number evaluable for the primary endpoint or the distribution of participants across dose levels. The reported enrollment should not be treated as a denominator for response or dose-limiting toxicity outcomes, because those results are not reported. It also cannot establish the precision of an unreported estimate.
How should planning calculations be distinguished from observed evidence?
In general, a sample-size calculation is a planning exercise based on assumptions about an outcome, variability, significance level, allocation, and the effect a future study aims to detect. It does not describe how much power the completed study actually had after its outcomes were observed. A calculation based on an effect estimate is also dependent on that estimate and its uncertainty.
Any planning illustration associated with the posted results should not be interpreted as an observed trial outcome or as the study’s original design. The registry record does not report the trial’s own statistical analysis plan or a completed inferential analysis. In particular, an illustration assuming a two-arm allocation does not change the registered study’s single-group design. The posted data do not supply an analyzed effect estimate for the primary or secondary outcome that could support conclusions about treatment benefit.
General statistical principles can explain what a dose-finding endpoint or confidence interval is intended to convey, but they cannot fill in missing trial observations. The registry record’s statement that the primary results were not analyzed is decisive for interpreting the reported evidence: no maximum tolerated dose estimate is available to assess.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity arises when multiple outcomes, comparisons, timepoints, or subgroup analyses are examined. In general, testing many questions can increase the chance of finding an apparently notable result by chance unless the analysis plan addresses that issue. The registry lists a Phase I primary outcome and a Phase II response outcome, but it does not report a multiplicity strategy, a hierarchy for testing outcomes, or an inferential analysis. The record also reports 0 analyses.
Missing-data procedures are not reported in the registry record. It does not describe whether endpoint information was absent for some participants, how evaluability was defined, or how missing observations would have been handled. The statement that primary results were not analyzed because of insufficient data indicates that the primary result is unavailable; it does not specify the underlying data pattern or the reason each observation was unavailable.
The analysis populations are also not reported in the registry record. In particular, the record does not identify which participants were considered evaluable for the 4-week toxicity endpoint or for the Phase II response outcome. Enrollment of 3 should not be assumed to equal the analysis population for either endpoint.
These omissions limit both reproducibility and interpretation. Without analysis-population definitions, missing-data rules, and an analysis plan, readers cannot determine how participants were selected for an outcome analysis or whether any planned statistical adjustment was used. No adjustment or missing-data method should be inferred from the study’s phase or design.
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 and Nab-paclitaxel | 0 | 3 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Lenalidomide and Nab-paclitaxel | 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 |
|---|---|
| Lenalidomide and Nab-paclitaxel | 0 of 3 |
The adverse-event record lists the treatment group as having 0 serious adverse events among 3 at risk, 0 other adverse events among 3 at risk, and 0 deaths. These are registry counts. They should be read as recorded events for the listed group, not as proof that no adverse effects occurred or that the combination is free of risk.
In general, event counts depend on how events are defined, collected, classified, and followed over time. The registry record does not report the observation period used for these adverse-event counts, the completeness of safety follow-up, or additional details about event grading and attribution. It also does not provide a dose-specific breakdown. The absence of listed events is therefore difficult to interpret beyond the recorded counts.
The primary toxicity endpoint differs from these aggregate adverse-event counts: it concerns dose-limiting toxicity over 4 weeks using CTCAE version 4.0. The record says that primary results were not analyzed because of insufficient data. The adverse-event figures do not supply the missing dose-limiting toxicity analysis and cannot be used to infer the maximum tolerated dose.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a Phase I/II, single-group study of lenalidomide and nab-paclitaxel was registered for relapsed or refractory multiple myeloma, enrolled 3 participants, and was terminated. It describes a dose-escalation approach and identifies maximum tolerated dose as the Phase I primary outcome. It also names a Phase II response outcome. These registry entries describe the study’s purpose and registered structure.
The record does not report an analyzed maximum tolerated dose, dose-limiting toxicity result, or count of complete or partial responses. It states that the primary results were not analyzed because of insufficient data. Accordingly, the posted data do not establish how the combination performed on the primary endpoint or the secondary response outcome. They also do not provide a basis for estimating comparative benefit, since the study had no control group.
The recorded adverse-event counts are 0 serious adverse events, 0 other adverse events, and 0 deaths in the listed group. These counts are not a comprehensive estimate of risk, particularly because the record does not report safety follow-up completeness or detailed event data. They should not be converted into a claim that treatment was safe or that adverse events did not occur outside the recorded categories or observation.
More generally, statistical conclusions depend on outcome data, defined analysis populations, and a specified analysis. When those elements are unavailable, the appropriate conclusion is that the outcome is not established by the posted record. This is different from concluding that an effect is absent. The registry’s terminated status and lack of analyzed results leave the principal clinical questions unresolved in this record.
13. Why This Trial Matters Statistically
This trial is statistically instructive because its registered purpose and its reported results are not the same thing. A dose-finding design can be described in detail, including planned escalation and a toxicity assessment window, while still yielding no analyzable maximum tolerated dose. The record’s statement that results were not analyzed makes the absence of an estimate central to interpretation.
The design also illustrates why a planned Phase II expansion should not be mistaken for completed evidence. The summary describes an expansion plan after dose finding, but the record does not report that the expansion occurred or provide a Phase II response result. Planned cohorts and assessments explain intended procedures; they do not establish that those procedures were completed or that outcomes were analyzed.
Another statistical lesson is that enrollment alone does not tell readers how much endpoint information exists. The record reports enrollment of 3, but does not state how many participants were evaluable for toxicity, the dose distribution, or the analysis population. These are important details for interpreting dose-finding data, even before considering precision or uncertainty.
Finally, a single-group design can describe observations but cannot provide a controlled comparison. In this record, that limitation is compounded by the absence of analyzed primary and secondary outcomes. Clear separation between the registered plan, recorded event counts, and analyzed estimates helps prevent an intended study question from being mistaken for a result.
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 record says the primary outcome was not analyzed because of insufficient data. No maximum tolerated dose or dose-limiting toxicity result is available. The record also reports no response result for the Phase II secondary outcome. As a result, the central dose-finding and response questions cannot be answered from the posted results.
Enrollment was 3, and the study was single-group with no masking. There was no concurrent comparator, so the design could not estimate a randomized contrast against another treatment. The record does not report the number of participants treated at each dose, the number evaluable for each endpoint, or the analysis populations. The planned escalation and Phase II expansion do not establish what was actually completed.
Important analysis details are not reported in the registry record, including the full dose-limiting toxicity definition, the escalation decision rules, missing-data handling, and statistical analysis plan. The record also does not describe safety follow-up completeness or provide detailed adverse-event data. These gaps prevent assessment of endpoint precision, the treatment of incomplete observations, and the interpretation of the listed zero event counts.
The record notes that the principal investigator left the institution, but it does not provide further detail about the consequences for study conduct or data availability. No explanation beyond insufficient data is reported for the lack of primary analysis. These limitations should be kept distinct from evidence of no toxicity or no response: the outcomes are unreported, not established as absent.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Phase I/II Trial of the Combination of Lenalidomide (Revlimid) and Nab-paclitaxel (Abraxane) in the Treatment of Relapsed/Refractory Multiple Myeloma — NCT02075021.
- Registry record last updated 2018-10-15; status last verified 2018-09.
- Results first posted 2017-09-05.
- 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
NCT02075021 is a terminated Phase I/II, single-group trial of lenalidomide and nab-paclitaxel in relapsed or refractory multiple myeloma. The registry reports enrollment of 3, a start of 2014-03, and primary completion in 2015-11. The primary outcome was maximum tolerated dose over 4 weeks, assessed through dose-limiting toxicity using CTCAE version 4.0.
The treatment description included planned dose escalation and de-escalation, with a 4-week cycle. A secondary Phase II outcome concerned the number achieving complete or partial response. The registry says the primary results were not analyzed because of insufficient data and reports no analyses. It lists 0 serious adverse events, 0 other adverse events, and 0 deaths in the treatment group. No maximum tolerated dose or response result is reported.