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/Bortezomib/Dexamethasone for Multiple Myeloma (MM) (NCT01782963), a na phase 2 study. The primary outcome is Objective Response Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01782963 |
| Phase | Phase 2 |
| Condition | Multiple Myeloma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 50 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Massachusetts General Hospital |
| Results posted | Yes |
2. Clinical Question
Population
The study concerns patients with newly diagnosed multiple myeloma who were not eligible for transplant. The registry records an enrollment of 50 participants. Further eligibility details and characteristics of the enrolled participants are not reported in the registry record.
Intervention
The treatment combined lenalidomide, bortezomib and dexamethasone. During induction cycles 1-9, lenalidomide was taken orally on days 1-21, and bortezomib was given by injection on days 1, 8, 15 and 22. Dexamethasone schedules differed by age: for participants 75 years of age or younger, it was taken orally on days 1, 2, 8, 9, 15, 16, 22 and 23; for those greater than 75 years of age, it was taken orally on days 1, 8, 15 and 22. Dose amounts and the complete treatment schedule beyond the described induction period are not reported in the registry record.
Comparator
There was no separate comparator group. The study is recorded as a single-group trial with no allocation to different groups and no masking. Its design therefore does not directly compare the combination with another treatment or with a control condition.
Primary question
The primary endpoint was objective response rate over 2 years. An objective response was defined using International Myeloma Working Group uniform response criteria, including stringent complete response, complete response, very good partial response and partial response. The registry record does not report how many participants achieved an objective response or a response-rate estimate. Thus, the question is clear, but its answer cannot be quantified from the posted results.
Statistically, this is a question about the frequency of a defined binary outcome in a treated group: each participant either meets the response definition or does not. The trial's single-group structure can describe response among participants in the study, if those outcome data are available. It cannot, by itself, distinguish the effect of the combination from other explanations for outcomes, because it has no concurrent comparator. The registry record identifies the study as a Phase II trial and describes the combination as investigational. It does not report the primary endpoint result, details of the analysis population, or a comparison that would support a treatment-effect estimate relative to another group.
3. Trial Design
The registry identifies NCT01782963 as a Phase II study sponsored by Massachusetts General Hospital. The study is titled as an investigation of modified lenalidomide, bortezomib and dexamethasone in transplant-ineligible patients with newly diagnosed multiple myeloma. Its recorded enrollment is 50. The listed study status is completed; the recorded study start is 2013-03 and primary completion is 2016-12.
The design is single-group, with allocation recorded as not applicable and masking recorded as none. Participants were assigned to a treatment arm, rather than randomized between treatments. The absence of a control arm is central to interpreting the results: outcome counts in the treated group would describe what happened in this cohort, but would not establish how those outcomes compare with a different treatment or with no study treatment. No like-for-like control comparison is available in the registry record.
The intervention consists of lenalidomide, bortezomib and dexamethasone. The described induction schedule covers cycles 1-9. Lenalidomide was taken orally on days 1-21, bortezomib was injected on days 1, 8, 15 and 22, and dexamethasone was scheduled differently according to age. The registry description is incomplete after the beginning of the consolidation schedule, so the full treatment course is not reported in the registry record. Dose amounts, dose modifications and treatment adherence are also not reported in the registry record.
The trial's primary outcome is an objective response assessed over 2 years. Other listed outcomes include treatment-related adverse events, survival measures, time to response, response by cytogenetic characteristics, plasma bortezomib concentration and pharmacogenomic markers of neuropathy. Although these outcomes indicate a range of clinical and laboratory interests, the registry record does not provide their numerical results or a detailed statistical analysis plan. The record describes the trial design and endpoints, but not enough outcome data to assess response or survival estimates.
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 | Treatment Arm |
|---|---|
| STARTED | 50 |
| COMPLETED | 50 |
| NOT COMPLETED | 0 |
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 | Treatment Arm |
|---|---|---|
| Participants analysed | 50 | |
| Age, Continuous | years | 73 (65 to 91) |
| Sex: Female, Male: Female | Participants | 27 |
| Sex: Female, Male: Male | Participants | 23 |
| Race/Ethnicity, Customized: White | Participants | 42 |
| Race/Ethnicity, Customized: Black or African-American | Participants | 2 |
| Race/Ethnicity, Customized: Asian | Participants | 3 |
| Race/Ethnicity, Customized: Other | Participants | 3 |
| Region of Enrollment: United States | Participants | 50 |
| ISS Stage at Diagnosis: I | Participants | 19 |
| ISS Stage at Diagnosis: II | Participants | 17 |
| ISS Stage at Diagnosis: III | Participants | 14 |
| Durie-Salmon Stage: I | Participants | 16 |
| Durie-Salmon Stage: II | Participants | 16 |
| Durie-Salmon Stage: III | Participants | 18 |
| ECOG Performance Status: 0 | Participants | 25 |
| ECOG Performance Status: 1 | Participants | 18 |
| ECOG Performance Status: 2 | Participants | 7 |
| ECOG Performance Status: 3 | Participants | 0 |
| ECOG Performance Status: 4 | Participants | 0 |
| ECOG Performance Status: 5 | Participants | 0 |
| High-Risk Cytogenetics: Yes | Participants | 6 |
| High-Risk Cytogenetics: No | Participants | 42 |
| High-Risk Cytogenetics: Unknown | Participants | 2 |
| Serum Heavy/Light Chain: IgG | Participants | 34 |
| Serum Heavy/Light Chain: IgA | Participants | 9 |
| Serum Heavy/Light Chain: Light-chain only | Participants | 5 |
| Serum Heavy/Light Chain: Unknown | Participants | 2 |
| Body Mass Index (BMI) | Kg/m^2 | 28 (18 to 45) |
6. Primary Endpoint
The registry lists the primary outcome as Objective Response Rate, measured over 2 years.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint, objective response rate, measures how many participants meet a prespecified definition of tumor response during the 2 years specified for the endpoint. The registry uses International Myeloma Working Group uniform response criteria. These include several response categories: stringent complete response, complete response, very good partial response and partial response. The categories rely on measures such as serum and urine M-protein, bone marrow plasma cells, free light chain ratio and, when applicable, plasmacytomas. The precise criteria differ by response category.
Combining the qualifying categories into an objective response endpoint gives a binary outcome for each participant: response criteria are met or they are not. This kind of endpoint is used to describe the proportion of a study group that reaches a defined response threshold. It can make clinical outcomes easier to summarize than reporting each participant's many laboratory measurements separately. The response definition also makes clear that the endpoint is not simply a change in one measurement; it depends on the specified criteria.
The endpoint's interpretation depends on how participants were assessed, how missing assessments were handled and which analysis population was used. Those details are not reported in the registry record. Most importantly, the record does not report the number of participants who met the objective response definition, a response-rate estimate or an interval quantifying its precision. The endpoint is defined, but its observed result cannot be evaluated from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a single-group Phase II study enrolling 50 participants and identifies objective response rate over 2 years as the primary endpoint. It does not report the primary endpoint result. There is no response count, response-rate estimate or confidence interval in the recorded results. Consequently, the frequency of objective response and the precision of any estimate cannot be assessed from this record. The absence of a reported estimate is not evidence that no responses occurred; it means the result is not reported in the registry record.
The design also limits what a response result could establish, even if a count were available. A single treated group can show the outcomes observed among its participants, but without a concurrent comparator it cannot isolate a treatment effect from other possible explanations. There is no randomized or otherwise assigned comparison group in the registry record. The recorded allocation is not applicable, and masking is none. Thus, this trial does not yield a like-for-like comparison between treatment strategies.
The registry lists other outcomes, including median progression-free survival, median overall survival, median time to response and response by cytogenetic characteristics. It also lists adverse-event and pharmacology outcomes. Numerical results for those measures are not reported in the registry record. In particular, listing a survival outcome does not reveal a median survival time, and listing a subgroup outcome does not establish that subgroup estimates were calculated or that they are precise.
The safety summary contains counts for participants affected by serious adverse events and other adverse events, as well as serious-event terms. These data should be read separately from the primary efficacy endpoint. An event can be serious without necessarily being treatment-related, and the registry's summary does not provide enough detail to infer causality, event timing or severity grades for each term. The registry also records zero deaths in the adverse-event summary, but that entry does not replace a reported overall survival analysis.
Statistical precision cannot be judged for the primary endpoint because no estimate or interval is reported. An interval around an estimate would ordinarily convey the range of values compatible with the observed data under the analysis assumptions; a narrow interval generally indicates more precision than a wide one. This is a general statistical explanation, not a description of an interval in this trial. No confidence interval for objective response is reported in the registry record.
Overall, the record supports a description of the trial's design, population focus, treatment schedule and endpoint definitions. It does not support a numerical conclusion about the primary response outcome or the listed survival outcomes. Nor does its single-group structure support a direct comparative treatment-effect conclusion.
9. Statistical Methods Explained
The methods that can be described from the registry record are limited to the design and endpoint definitions. The study is recorded as a single-group Phase II trial with an enrollment of 50. The primary endpoint is a participant count for objective response, assessed over 2 years. The record does not describe the planned statistical test, estimator, confidence-interval method, handling of missing outcome data, analysis population or detailed analysis plan. These items are not reported in the registry record.
What does a single-group design estimate?
In a single-group study, the direct descriptive target is the frequency of an outcome among participants in that group. For a binary outcome such as meeting or not meeting the objective response criteria, an analysis can count participants in each outcome category and estimate the proportion meeting the criteria. This is a general description of binary-outcome analysis; the registry record does not report the response count or estimate for this study.
A descriptive proportion is not, on its own, a comparative treatment effect. A treatment-effect comparison requires outcomes under a comparator or another defensible source of comparison. This study is recorded as single-group, with no allocation to a separate comparison arm. Therefore, no between-arm contrast can be estimated from the trial design recorded in the registry. The study may describe the experience of its enrolled cohort, but its design does not provide a randomized comparison.
Why does precision matter?
An estimate is more informative when its uncertainty is also reported. A confidence interval is a general statistical device for expressing uncertainty around an estimate under specified assumptions. It is not a guarantee about an individual participant's outcome, and it does not correct for design limitations such as the lack of a comparison group. No confidence interval for objective response is reported in the registry record, so the precision of that endpoint cannot be assessed.
The number of enrolled participants alone cannot establish precision for a particular endpoint. Precision also depends on how many participants have outcome data and on the observed distribution of outcomes. The record gives an enrollment of 50 but does not report the primary response count or an endpoint-specific analysis population. It is therefore not possible to recover a response estimate or its uncertainty from the enrollment figure alone.
What can the listed planning illustrations tell readers?
Sample-size calculations are planning tools, not observed trial outcomes. The planning illustrations associated with the posted results are described as using a two-sided significance level of 0.05, equal allocation between two arms and a normal approximation. They are based on a stated fraction of the observed effect; time-to-event illustrations use a total number of events, while other illustrations express participants per arm. These are general planning assumptions and do not describe the trial's recorded single-group design.
Those illustrations should not be read as evidence that the actual study had a particular power, or as observed power. A planning calculation addresses a hypothetical design and effect under its assumptions. It does not show that the effect occurred, and it cannot replace the missing primary result. In this case, the registry record does not report the observed objective response effect needed to interpret such illustrations, nor does it report that the two-arm assumptions were the trial's own design assumptions. The registry record does not report a sample-size justification for the actual study.
Why can count outcomes be over-dispersed?
For count outcomes, a simple statistical model may assume that variability follows a particular relationship with the average count. Over-dispersion means that the observed variation is greater than that model expects. In general, ignoring over-dispersion can make uncertainty look smaller than it is, so methods that allow extra variation may be needed. This is a general statistical point. The primary endpoint in this trial is a participant count for a binary response status, not a recurrent-event count, and no over-dispersion analysis is reported in the registry record.
Related outcomes can require different methods. Time-to-event endpoints such as progression-free survival and overall survival concern the time until an event, and may also involve participants whose event has not occurred by the end of observation. A median for such an endpoint cannot be inferred from the endpoint name; the event and follow-up data are needed. The registry lists median progression-free survival and median overall survival but does not report their results or the methods used to estimate them.
What methods are not described?
The registry record does not report interim analyses, stopping rules, adjustment for multiple outcomes, covariate adjustment or a detailed missing-data strategy. It also does not describe whether analyses followed an intention-to-treat principle, whether participants were excluded from particular analyses, or whether sensitivity analyses were planned. These details can affect interpretation and should not be inferred from a Phase II designation or from the endpoint list. Without the analysis plan and numerical outcome results, readers cannot independently evaluate the statistical analysis of the primary endpoint.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary endpoint and multiple other outcomes, including adverse events, progression-free survival, overall survival, time to response, cytogenetic response, bortezomib concentration and pharmacogenomic markers of neuropathy. When a study assesses several outcomes, the chance of obtaining at least one apparently notable result can depend on how many analyses are performed and how they are interpreted. This is the general multiplicity concern. The registry record does not report a strategy for controlling or otherwise addressing multiplicity, so it is not possible to determine whether any adjustment was planned or used.
Missing-data handling is also not reported in the registry record. The record does not say how participants without an evaluable response assessment would be classified, how incomplete follow-up would be treated for survival outcomes, or whether any imputation or sensitivity analysis was used. These decisions matter because the analyzed participants and their outcome availability can affect estimates. They cannot be reconstructed from the enrollment count alone.
The analysis populations are not described in detail. The record does not state whether every enrolled participant was included in each endpoint analysis, whether exclusions occurred, or whether participants were analyzed according to treatment received. No interim analyses or detailed statistical plan are reported in the registry record. These omissions do not establish that a particular procedure was or was not used; they limit what can be evaluated from the recorded information.
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 |
|---|---|---|---|
| Treatment Arm | 34 | 50 | 68.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Treatment Arm | 0 of 50 |
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 |
|---|---|
| Treatment Arm | 50 of 50 |
Most frequent serious adverse event terms
| Event term | Organ system | Treatment Arm |
|---|---|---|
| Hypophosphatemia | Metabolism and nutrition disorders | 15 of 50 |
| Fatigue | General disorders | 8 of 50 |
| Adrenal insufficiency | Endocrine disorders | 2 of 50 |
| Dehydration | Metabolism and nutrition disorders | 2 of 50 |
| Generalized muscle weakness | Musculoskeletal and connective tissue disorders | 2 of 50 |
| Hyperglycemia | Metabolism and nutrition disorders | 2 of 50 |
| Hypertension | Vascular disorders | 2 of 50 |
| Hyponatremia | Metabolism and nutrition disorders | 2 of 50 |
| Neutrophil count decreased | Investigations | 2 of 50 |
| Platelet count decreased | Investigations | 2 of 50 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Treatment Arm |
|---|---|---|
| Fatigue | General disorders | 41 of 50 |
| Peripheral sensory neuropathy | Nervous system disorders | 32 of 50 |
| Anemia | Blood and lymphatic system disorders | 30 of 50 |
| Hypophosphatemia | Metabolism and nutrition disorders | 27 of 50 |
| Diarrhea | Gastrointestinal disorders | 25 of 50 |
| Edema limbs | General disorders | 23 of 50 |
| Constipation | Gastrointestinal disorders | 22 of 50 |
| Back pain | Musculoskeletal and connective tissue disorders | 20 of 50 |
| Hyperglycemia | Metabolism and nutrition disorders | 20 of 50 |
| Hypertension | Vascular disorders | 20 of 50 |
Serious events with the highest arm-level rate: hypophosphatemia (30.0%), fatigue (16.0%), adrenal insufficiency (4.0%), dehydration (4.0%), generalized muscle weakness (4.0%).
The registry's adverse-event summary lists 50 participants at risk for serious adverse events, with 34 affected. It also lists 50 participants at risk for other adverse events, with 50 affected. The summary records zero deaths. These are counts in the treatment arm; they are not a comparison with an untreated or alternative-treatment group.
The serious-event terms listed with percentages are hypophosphatemia, 30.0%; fatigue, 16.0%; adrenal insufficiency, 4.0%; dehydration, 4.0%; generalized muscle weakness, 4.0%; and hyperglycemia, 4.0%. These figures describe the terms as recorded in the registry summary. The summary does not establish that these terms are mutually exclusive, so they should not be added together to estimate the number of affected participants.
Seriousness and treatment relatedness are distinct concepts. The registry lists a separate outcome for participants with Grade 3 or higher treatment-related adverse events, but the serious-event counts do not by themselves identify whether an event was caused by treatment or its grade. Event definitions, timing, duration and participant-level detail are not reported in the registry record. The safety counts describe recorded events; they do not provide a controlled comparison of risk.
12. What the Result Does, and Does Not, Mean
The trial record establishes that a Phase II study of lenalidomide, bortezomib and dexamethasone was conducted in a single group of transplant-ineligible patients with newly diagnosed multiple myeloma, with 50 participants enrolled. It identifies objective response over 2 years as the primary endpoint and describes the response criteria. The record also lists survival, safety, pharmacology and other outcomes.
What the record does not establish is the observed objective response rate. The primary response count and estimate are not reported in the registry record. Without those results, there is no basis for describing the proportion who responded, the precision of that proportion or how it relates to the study's stated objective. This absence should not be interpreted as a null result or as evidence that no participants responded.
The single-group design also means that the trial does not provide a randomized treatment comparison. Even a reported response estimate would describe outcomes in the enrolled group rather than establish how outcomes would differ under another treatment. A comparator would be needed for a direct comparative assessment, and none is recorded.
The listed survival endpoints also cannot be interpreted without their numerical results and follow-up information. The registry record does not report median progression-free survival, median overall survival or median time to response. It likewise does not report numerical findings for the other listed efficacy or laboratory outcomes. Safety counts are available in the adverse-event summary, but their interpretation is limited by the absence of detailed definitions, event-level information and a control group.
In statistical terms, the record supports a description of the trial's question and design, not a conclusion about the magnitude or precision of its primary outcome. The distinction is important: a stated endpoint is not itself a result, and a single-group outcome is not a comparative effect. Any broader claim about benefit, harm or relative performance would go beyond what the registry record reports.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of the distinction between a carefully defined question and a reported answer. The registry specifies an objective response endpoint, a 2-year timeframe and response criteria based on the International Myeloma Working Group uniform response framework. That level of definition clarifies what outcome the study intended to measure. Yet the primary result is not reported in the registry record, so the defined endpoint cannot be converted into a response estimate or a statement about its statistical precision.
The single-group design also illustrates why study structure matters as much as outcome choice. An observed response frequency, if reported, would characterize the participants in the treatment arm. Without a comparison group, it would not quantify the difference between the regimen and another treatment. This is not a defect that can be remedied by a more elaborate analysis of the same single-group data; it is a limit on the comparative question the design can answer.
Finally, the record shows why registry entries should be read as a collection of design details, outcome definitions and reported results, rather than treating every listed outcome as a finding. The study lists several survival, safety and exploratory outcomes, but numerical results for most are not reported. Clear separation of endpoint, design and result helps prevent overinterpretation. Here, the most defensible statistical account is that the study and its outcome questions are documented, while the primary efficacy estimate and its uncertainty are unavailable in the registry record.
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 important trial-specific limitation in interpreting efficacy is that the registry record does not report the primary objective response result. It gives the endpoint definition and timeframe, but not the number of participants meeting the response criteria or an estimate with uncertainty. The result therefore cannot be quantified, and its precision cannot be assessed.
The study is recorded as a single group, with no allocation to a separate comparison arm and no masking. Its results cannot provide a direct randomized comparison with another treatment. The registry identifies transplant-ineligible patients with newly diagnosed multiple myeloma as the target population, but additional eligibility criteria and participant characteristics are not reported in the registry record. Generalizability beyond the enrolled cohort cannot be assessed from the available detail.
The treatment description gives induction schedules for lenalidomide, bortezomib and age-specific dexamethasone, but the registry description of the regimen is incomplete after the beginning of consolidation. Dose amounts, modifications and adherence are not reported in the registry record. The record also does not describe the primary endpoint analysis population, missing-data rules, interim analyses or a detailed statistical analysis plan.
Numerical findings for the listed survival and other secondary outcomes are not reported in the registry record. Safety information is summarized as counts and serious-event terms, without detailed event definitions, timing, duration or participant-level data. These omissions constrain assessment of outcome completeness, analysis choices and the context of reported events. They should be treated as limits of the registry information rather than as proof that the corresponding information was not collected during the study.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Lenalidomide/Bortezomib/Dexamethasone for Multiple Myeloma (MM) — NCT01782963.
- Registry record last updated 2018-10-31; status last verified 2018-10.
- Results first posted 2018-03-13.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
NCT01782963 is a completed Phase II, single-group study sponsored by Massachusetts General Hospital. It enrolled 50 participants with newly diagnosed multiple myeloma who were not eligible for transplant, according to the study title. The intervention combined lenalidomide, bortezomib and dexamethasone. The primary endpoint was objective response over 2 years, defined using International Myeloma Working Group uniform response criteria.
The registry record does not report the primary response result, a response estimate or its precision. The listed progression-free survival, overall survival and other outcome results are also not reported in the registry record. The adverse-event summary records 34 participants affected by serious adverse events among 50 at risk, 50 affected by other adverse events among 50 at risk, and zero deaths. Because the study has no comparator group, its recorded outcomes do not support a direct treatment comparison.