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 Safety and Efficacy Study of Single Weekly Bortezomib in Newly Diagnosed Multiple Myeloma (NCT01090921), a na phase 2 study. The primary outcome is Number of Participants at Each Response Category (Stable Disease, Minimal Response, Partial Response, Very Good Partial Response, Near Complete Response/Complete Resonse).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01090921 |
| 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 | Boston VA Research Institute, Inc. |
| Results posted | Yes |
2. Clinical Question
Population
People with newly diagnosed multiple myeloma who had not yet received treatment. The endpoint description specifies patients ineligible for transplant or eligible but wishing to postpone autologous stem cell transplant. The registry record lists an enrollment of 50.
Intervention
Weekly bortezomib administered with dexamethasone. The arm description gives bortezomib as 1.6mg/m2 by IV push once a week for four weeks, followed by one week off; that five-week period is a treatment cycle. The dexamethasone schedule is described only in part in the registry record.
Comparator
There was no separate comparator group. The study is identified as single-group, with allocation recorded as NA and masking as none. The registry record therefore does not describe a randomized comparison or a like-for-like control group.
Primary question
The primary endpoint asks how many participants fell into each listed response category during participation: stable disease, minimal response, partial response, very good partial response, or near complete response/complete response. The registry record does not report the number of participants in these categories, so the response distribution cannot be described from the posted results.
In statistical terms, this is a descriptive question about the distribution of response categories among participants receiving the study regimen. It is not a direct estimate of how outcomes compare with another treatment or with no treatment. Without a concurrent comparison group, any observed response pattern would not by itself distinguish treatment effects from other explanations, including the course of disease or participant characteristics. The registry record describes the intended population and endpoint, but does not provide the category counts or an analysis of them.
3. Trial Design
NCT01090921 is listed as a completed Phase II study sponsored by Boston VA Research Institute, Inc. The title describes a study of single weekly bortezomib in newly diagnosed multiple myeloma. The registry record lists 50 participants and identifies the design as single-group, with no masking and allocation recorded as NA. No separate control arm is described, so this design does not provide a randomized between-group treatment comparison.
The single arm is described as receiving bortezomib and dexamethasone. Bortezomib is specified at 1.6mg/m2 by IV push over 3 to 5 seconds, once a week for four weeks followed by one week off. The registry record calls this five-week interval a treatment cycle. It gives a partial dexamethasone schedule of 40mg daily on the day of and the day after each dos, but that wording is incomplete in the registry record and should not be extended by inference.
The study began in 2007-05, reached primary completion in 2015-03, and is marked completed. The primary endpoint covers response categories over each subject’s participation. Its timeframe description says participation averaged 4.8 cycles. That average describes exposure time, not the number of participants with a response or the duration of benefit.
The record also lists a secondary outcome concerning participants with dose reductions in bortezomib, dexamethasone, or both. It lists two outcome measures, but no analyses. The protocol’s sample-size justification, detailed statistical plan, analysis populations, and handling of missing endpoint data are not reported in the registry record. The enrollment figure alone does not show whether the study was sized to estimate response with a particular precision.
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 | Bortezomib and Dexamethasone |
|---|---|
| STARTED | 50 |
| COMPLETED | 23 |
| NOT COMPLETED | 27 |
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 | Bortezomib and Dexamethasone |
|---|---|---|
| Participants analysed | 50 | |
| Age, Customized: Age | years | 70.9 (50 to 89) |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 50 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 50 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 50 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants at Each Response Category (Stable Disease, Minimal Response, Partial Response, Very Good Partial Response, Near Complete Response/Complete Resonse), measured over Data was collected for each subject for the duration of the participation in the study, which was an average of 4.8 cycles..
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants in five response categories: stable disease, minimal response, partial response, very good partial response, and near complete response/complete response. It is therefore a categorical endpoint, recorded as participant counts rather than as a continuous measurement. The endpoint description says it was intended to evaluate the response rate for weekly bortezomib plus dexamethasone in newly diagnosed patients who were ineligible for transplant or wished to postpone autologous stem cell transplant.
Response categories can be useful because they describe differing levels of observed disease response in a way that is clinically interpretable. Reporting the distribution across categories can show whether participants had responses of differing degrees or stable disease during study participation. However, the registry record does not report the count in any category. It consequently does not support a description of the observed response distribution, nor does it show how response was assessed or adjudicated.
The endpoint timeframe is each subject’s participation in the study, with an average of 4.8 cycles. A participant-level category count is not the same as a time-to-event measure: it does not, by itself, describe when a response began, how long it lasted, or the time until progression. Those details are not reported in the registry record. Because the study has a single group, even a reported response distribution would describe outcomes among enrolled participants and would not establish a comparative treatment effect.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a 50-participant, single-group Phase II study of bortezomib with dexamethasone in newly diagnosed multiple myeloma. Its primary endpoint is a count of participants in each of five response categories. However, the category counts are not reported in the registry record. The posted information therefore does not establish how many participants had stable disease, minimal response, partial response, very good partial response, or near complete response/complete response. No response proportion or uncertainty interval can be described from the listed details.
The design also limits the type of conclusion that could be drawn from response counts alone. With no separate comparator group, the study can describe outcomes observed in the treated group, but it cannot directly estimate a difference relative to another treatment. A single-group result cannot separate the regimen’s contribution from other explanations for the observed outcomes. This is a design limitation, not a claim about whether the regimen was beneficial or harmful.
The registry lists the study as completed and gives primary completion as 2015-03. Completion status does not substitute for reported endpoint results. The record lists two outcome measures and zero analyses; it does not provide the primary response counts, inferential analysis, confidence intervals, or a statistical analysis plan. The endpoint timeframe states that participation averaged 4.8 cycles, but that average does not indicate the response distribution or provide a measure of response duration.
The safety record reports 28 serious adverse events among 50 participants at risk in the Bortezomib and Dexamethasone group, and six deaths. These figures should be read as safety descriptions in a single treatment group, not as estimates of excess risk attributable to treatment. The registry also lists serious adverse event terms and their percentages, but those terms do not replace the missing efficacy results or provide a comparator for assessing causation.
Planning illustrations based on an observed effect are not observed power and are not the study’s original design justification. Their stated assumptions include a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. Those assumptions describe a hypothetical planning exercise and do not change the actual single-group design. The observed effect and numeric planning results are not reported in the registry record. Overall, the available record describes the study question and design, but does not provide the primary outcome results needed to characterize responses.
9. Statistical Methods Explained
The study’s primary endpoint is a set of participant counts across response categories. The registry record describes the endpoint but does not report the category counts or an analysis. The concepts below are general statistical explanations; they do not imply that a particular method was used in this study.
What does a response-category count describe?
A categorical outcome places each participant into one of a defined set of groups. Here, the listed groups range from stable disease through several response categories, ending with near complete response/complete response. A count in each category describes the observed distribution in the study group. If the categories are mutually exclusive and each participant is assigned one category, the counts can account for the participants with an assessed outcome. Whether the categories were handled that way, and how participants without an assessment were treated, are not reported in the registry record.
Counts are descriptive. A count alone does not communicate precision, particularly when the group is limited in size. A confidence interval is a range of values calculated from data under a statistical method; it conveys uncertainty around an estimate. For a proportion, an interval would describe uncertainty about the underlying proportion compatible with the observed data and the method’s assumptions. No response counts, proportions, or confidence intervals are reported in the registry record, so none can be interpreted for this endpoint.
What would a comparison require?
A between-group treatment comparison requires outcomes from distinct groups and a defined contrast, such as a difference in response proportions. This study is identified as single-group and has no separate comparator. Thus, its design does not support a randomized treatment contrast. General statistical adjustment cannot recreate a randomized control group in the registry record in the registry record. A comparison with external or historical data would require details about those data and methods for addressing differences between populations; such a comparison is not described in the registry record.
Even in a study with a comparator, an observed contrast is an estimate rather than a guarantee of a treatment effect. Its interpretation depends on the design, endpoint definition, analysis population, missing data, and uncertainty. In a single-group study, a response count can describe what was observed among participants, but cannot establish what would have happened to those same participants under a different treatment.
Why do denominators and missing outcomes matter?
A participant count needs a clear denominator. The enrolled number is 50, but the registry record does not say how many participants had an evaluable primary endpoint or whether all enrolled participants were included in each response category. If participants with missing assessments are omitted, the reported distribution could differ from a distribution using all enrolled participants as the denominator. If missingness relates to health status, treatment discontinuation, or response, a complete-case estimate may not represent the full enrolled group. These are general concerns; the registry record does not report its missing-data approach or endpoint analysis population.
General analyses may address missing outcomes through prespecified assumptions or sensitivity analyses, but those approaches depend on information about why outcomes are missing and the analysis plan. No such methods are reported in the registry record. It is therefore not possible to tell whether missing data were present, how they were handled, or whether results were tested under alternative assumptions.
What does over-dispersion mean?
For count data, a statistical model may assume a particular relationship between the mean and variability. Over-dispersion occurs when observed variability is greater than the model allows. If ignored, it can make uncertainty estimates too narrow and tests appear more decisive than warranted. This is a general issue for some count-model analyses. The endpoint in this study is described as participant counts across categories, and the registry record does not report a count-model analysis or any assessment of over-dispersion. It should not be assumed that such a model was used or required.
How should planning calculations be read?
Sample-size planning asks how many participants might be needed under specified assumptions to detect a stated effect with a chosen statistical threshold and power. Such calculations depend on the effect size, outcome variability, allocation, and analysis method. The planning description associated with this record specifies a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It says the effect to detect is a stated fraction of the observed effect. These are planning assumptions, not observed trial outcomes.
The actual study is single-group, so equal allocation between two arms is not its design. A planning illustration framed around two arms is hypothetical and does not describe how the enrolled study was conducted. The record does not report the observed effect or numeric sample-size illustrations in the registry record for interpretation. Such planning estimates must not be treated as observed power, nor as evidence that the original study was designed around those assumptions. The study’s own sample-size justification is not reported in the registry record.
What can be inferred from the analysis listing?
The record lists two outcome measures and zero analyses. That means the listed record does not supply an inferential analysis for the response categories. It does not identify a statistical test, model, adjustment strategy, confidence-interval method, or prespecified hypothesis. The absence of a listed analysis is not evidence that no analysis was conducted outside the registry; it means those methods are not reported in the registry record. Accordingly, interpretation should remain limited to the design and outcome descriptions actually recorded.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists two outcome measures: the primary response-category endpoint and a secondary outcome concerning dose reductions in bortezomib, dexamethasone, or both. It does not report a statistical analysis plan, a hierarchy of hypotheses, or a method for addressing multiplicity. When several outcomes or comparisons are tested, the chance of at least one apparently notable finding can increase. General approaches include prespecifying a primary test or adjusting inference across tests, but no particular approach is reported for this study.
The record lists zero analyses. It does not provide hypothesis tests, confidence intervals, or an account of whether outcomes were analyzed as planned. The protocol’s sample-size justification and any interim analyses are not reported in the registry record. Neither the presence nor absence of interim monitoring should be inferred from the completed status.
Missing-data handling is also not reported. The primary endpoint is described over each participant’s study participation, but the record does not say how many participants had evaluable response assessments, how missing assessments were classified, or which participants were included in analysis. It also does not report whether the primary analysis used all enrolled participants or a defined subset. These details matter because different denominator rules can change a response distribution.
These omissions limit reproducibility and assessment of selective reporting, but they do not establish that the study’s investigators used any particular unreported method. The registry record supports a description of the intended outcomes and design; it does not support conclusions about multiplicity control, missing-data assumptions, interim testing, or analysis populations.
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 |
|---|---|---|---|
| Bortezomib and Dexamethasone | 28 | 50 | 56.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Bortezomib and Dexamethasone | 6 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 |
|---|---|
| Bortezomib and Dexamethasone | 0 of 50 |
Most frequent serious adverse event terms
| Event term | Organ system | Bortezomib and Dexamethasone |
|---|---|---|
| Lymphopenia | Blood and lymphatic system disorders | 9 of 50 |
| Hyperglycemia | Endocrine disorders | 9 of 50 |
| Asthenia | General disorders | 4 of 50 |
| Anemia | Blood and lymphatic system disorders | 2 of 50 |
| Diarrhea | Gastrointestinal disorders | 2 of 50 |
| Constipation | Gastrointestinal disorders | 2 of 50 |
| Acute renal failure | Renal and urinary disorders | 2 of 50 |
| Thrombocytopenia | Blood and lymphatic system disorders | 1 of 50 |
| Vomiting/Nausea | Gastrointestinal disorders | 1 of 50 |
| Elevated creatinine | Renal and urinary disorders | 1 of 50 |
Serious events with the highest arm-level rate: lymphopenia (18.0%), hyperglycemia (18.0%), asthenia (8.0%), anemia (4.0%), diarrhea (4.0%).
For the Bortezomib and Dexamethasone group, the safety record lists 50 participants at risk for serious adverse events and 28 participants affected. It lists 50 participants at risk for other adverse events and zero affected. The record also reports six deaths. These figures describe events recorded in the study group; they do not by themselves show that treatment caused an event or death. There is no separate control group for a comparative safety estimate.
The listed serious adverse event terms are lymphopenia (18.0%), hyperglycemia (18.0%), asthenia (8.0%), anemia (4.0%), diarrhea (4.0%), and constipation (4.0%). The registry record presents these as percentages. It does not specify in the registry record whether participants could appear under multiple terms, so the percentages should not be added or treated as mutually exclusive categories. These terms also do not describe all possible adverse events.
A serious adverse event is a safety classification, not a statement that an event was caused by treatment. The record’s group-level counts do not provide event timing, duration, severity beyond the serious classification, or individual-level clinical context. The safety data should therefore be interpreted descriptively and separately from the unreported primary response counts.
12. What the Result Does, and Does Not, Mean
The record establishes that NCT01090921 was a completed Phase II, single-group study enrolling 50 participants with newly diagnosed multiple myeloma. It describes weekly bortezomib with dexamethasone and a primary endpoint based on participant counts in response categories. It also provides a study timeframe, an average participation duration of 4.8 cycles, and selected safety figures. These registry entries characterize the design and what the study intended to measure.
The record does not report the response-category counts. Consequently, it is not possible to state how participants were distributed across the endpoint categories or to describe the size or precision of any response proportion. The registry record also does not report an inferential analysis, a statistical analysis plan, or handling of missing endpoint data. The endpoint description alone is not an outcome result.
Even if response counts were available, the single-group design would support description of outcomes among participants, not a direct estimate of how the regimen compares with another treatment. There is no randomized comparator, and allocation is recorded as NA. A response observed during treatment would not, on its own, establish that treatment caused the response. The same caution applies to safety: the serious adverse event and death counts describe the group, but do not establish attribution or comparative risk.
The study’s completed status is not a substitute for posted efficacy results. Likewise, a planning illustration based on an observed effect would not be evidence of observed power or a result of the enrolled single-group study. The record does not provide the observed effect or the study’s original sample-size justification.
In sum, the registry record supports a clear description of the study question, treatment schedule as recorded, design, and selected safety counts. It does not support a conclusion about the response distribution, comparative efficacy, treatment causality, or the precision of an efficacy estimate. Those limits reflect the information reported and the design, rather than a claim for or against the treatment.
13. Why This Trial Matters Statistically
This study is statistically informative as an example of the distinction between an outcome definition and an outcome result. The registry record carefully names response categories and describes the population and treatment question, but does not report the counts in those categories. Without those counts, readers cannot assess the observed response distribution or its uncertainty. A stated endpoint does not supply evidence about the endpoint’s observed values.
The single-group design also clarifies the difference between describing outcomes and estimating comparative effects. A treated group can be described, but without a concurrent comparator there is no randomized contrast. This is particularly important when interpreting response and safety data: counts in a treatment group are not themselves estimates of what would have occurred under another treatment.
The record also illustrates why transparent reporting of analysis details matters. The registry lists two outcome measures and zero analyses, while the protocol’s sample-size justification, missing-data handling, analysis populations, and multiplicity approach are not reported. Without these details, readers cannot evaluate the planned inferential framework or determine how incomplete endpoint assessments may have affected interpretation.
Finally, planning exercises and actual study design must be kept distinct. The planning assumptions describe a hypothetical two-arm calculation, while the enrolled study is single-group. Planning values are not observed power and cannot replace reported results. These distinctions help prevent descriptive safety counts, design assumptions, and missing efficacy results from being blended into a stronger conclusion than the registry record supports.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The principal limitation for interpretation is that the primary response-category counts are not reported in the registry record. The record therefore does not allow readers to assess the primary outcome distribution, calculate or interpret response proportions, or evaluate their precision. It lists the intended categories and the timeframe, but not the endpoint values or an analysis.
The single-group design has no separate comparator. It cannot provide a randomized estimate of a treatment difference, and outcomes observed in the group cannot by themselves establish causation. The record’s allocation field is NA and masking is none; the study is identified as single-group. These features should shape interpretation of both efficacy and safety descriptions.
Several details relevant to reproducibility are also not reported in the registry record: the statistical analysis plan, sample-size justification, interim analyses, analysis populations, missing-data methods, and multiplicity handling. The primary endpoint is described over each subject’s participation, averaging 4.8 cycles, but the record does not state how incomplete participation or missing response assessments were handled. The dexamethasone schedule is incomplete in the arm description, ending with the word “dos,” and should not be completed by assumption.
The safety information is limited to the recorded group counts, six deaths, and selected serious adverse event terms with percentages. It does not give a comparator or establish event attribution. These limitations do not show that unreported procedures or analyses were absent; they show that they are not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Safety and Efficacy Study of Single Weekly Bortezomib in Newly Diagnosed Multiple Myeloma — NCT01090921.
- Registry record last updated 2020-09-09; status last verified 2020-08.
- Results first posted 2020-08-20.
- 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
NCT01090921 is a completed Phase II, single-group study sponsored by Boston VA Research Institute, Inc., with an enrollment of 50. It concerns weekly bortezomib with dexamethasone for newly diagnosed multiple myeloma. The record lists no masking and allocation as NA. The study began in 2007-05 and reached primary completion in 2015-03.
The primary endpoint counts participants across five response categories during participation, which averaged 4.8 cycles. The category counts and analyses are not reported in the registry record. A secondary outcome concerns participants with dose reductions in bortezomib, dexamethasone, or both.
The safety record lists 28 of 50 participants affected by serious adverse events, zero of 50 affected by other adverse events, and six deaths. These are descriptive figures for the single study group, not comparative or causal estimates.