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 A Phase II Study of Bevacizumab and Bortezomib in Patients With Relapsed/Refractory Multiple Myeloma (NCT00464178), a na phase 2 study. The primary outcome is Median Number of Cycles to Tumor Progression (TTP).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00464178 |
| Phase | Phase 2 |
| Condition | Multiple Myeloma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 7 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Hackensack Meridian Health |
| Results posted | Yes |
2. Clinical Question
Population
The study concerns patients with relapsed or refractory multiple myeloma. The registry record gives an enrollment of 7, but does not report further eligibility details or describe the characteristics of the enrolled participants.
Intervention
The arm is named “Bortezomib and bevacizumab.” Its description specifies bortezomib administration on Days 1, 4, 8, and 11, with response assessment after each cycle. Although the arm description names both drugs, the interventions list names bortezomib only.
Comparator
The study is recorded as single-group, with allocation listed as NA and masking as none. No concurrent control group or randomized comparison is described. Accordingly, the registry record does not present a like-for-like comparison against another treatment or a control condition.
Primary question
The primary outcome is the median number of cycles before tumor progression, with a timeframe of 18 months. The study’s stated purpose was to determine whether the drug combination had increased efficacy in relapsed/refractory multiple myeloma. Without a comparator and without a reported endpoint result, the record does not establish a comparative treatment effect.
3. Trial Design
This is a Phase II study recorded as single-group, with allocation listed as NA and masking as none. The registry record names the study arm “Bortezomib and bevacizumab.” It describes bortezomib administration on Days 1, 4, 8, and 11 and says response would be assessed after each 21-day cycle. The arm description says a total of 8 cycles were planned and that patients would be removed after Cycle 2 if progression was documented.
The interventions list names bortezomib as a drug, while the arm name and description refer to the combination with bevacizumab. The record does not list bevacizumab in the interventions field or give its administration schedule there. This difference in how the combination is represented should be kept in mind when reading the registry entry.
Enrollment is recorded as 7. The study started in 2007-04, had a primary completion date of 2009-02, and is listed as terminated. These administrative details describe the study record; they do not indicate why the study was terminated or whether the planned treatment course was completed by participants. The reason for termination is not reported in the registry record.
The primary outcome is the median number of cycles to tumor progression, with a stated timeframe of 18 months. No control arm is described, so the design does not support a randomized, direct comparison. The record does not report a statistical analysis plan, the treatment exposure actually received, or the number of participants contributing data to the primary outcome.
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: Seven subjects were consented, six were treated and one screen failed. Of the six treated, 4 completed, one subject was taken off study because of non compliance to protocol, one withdrew
Overall Study
| Stage | Bortezomib and Bevacizumab |
|---|---|
| STARTED | 6 |
| COMPLETED | 4 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | Bortezomib and Bevacizumab |
|---|---|
| Protocol Violation | 1 |
| Withdrawal by Subject | 1 |
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: 7 subjects were consented 6 proceeded with treatment, 4 completed study: one failed screening and one was dropped due to non-compliance with protocol, one withdrew consent
| Characteristic | Unit | Drug Combination |
|---|---|---|
| Participants analysed | 6 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 5 |
| Age, Categorical: >=65 years | Participants | 1 |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 5 |
6. Primary Endpoint
The registry lists the primary outcome as Median Number of Cycles to Tumor Progression (TTP), measured over 18 months.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Full Range |
| Unit | Cycles Prior to Tumor Progression |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the median number of cycles to tumor progression, expressed as cycles prior to progression. It is a time-to-event measure recorded in cycles rather than calendar time. The stated timeframe is 18 months, and the study description says response was assessed after each 21-day cycle.
The median describes the central point in the observed distribution: it is the point at which the ordered observations have at least half of the observations on either side, subject to the details of the analysis. The registry lists full range as the measure of dispersion, which describes the spread from the lowest to the highest observed value. These summaries are different from an average and do not, by themselves, show how precisely the endpoint has been estimated.
Progression is described using the Bladé criteria. The record mentions an increase of >25% in paraprotein or urinary light chain excretion, or marrow plasma cell percentage, but the description ends before the definition is complete. The full operational definition and any rules for handling participants who did not have documented progression are not reported in the registry record.
The registry identifies the endpoint but does not report its median, range, or other result. Therefore, the endpoint’s value and the precision of its estimate cannot be assessed from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes that the study was a Phase II, single-group investigation of a combination named as bevacizumab and bortezomib in relapsed/refractory multiple myeloma. It records enrollment of 7 and lists median cycles to tumor progression as the primary outcome. It does not report the numerical result for that endpoint. Without the observed median and its range, the posted record cannot show how long participants remained free of documented progression or how variable those observations were.
The design also matters for interpretation. No concurrent comparator is described, and the study is not recorded as randomized. An outcome observed in a single group cannot by itself distinguish the effects of the regimen from other explanations, such as characteristics of the participants or the course of the disease. The registry’s stated aim of assessing increased efficacy is a purpose, not evidence of a comparative effect. No treatment effect estimate or comparative analysis is reported.
The study is listed as terminated, but the reason is not reported in the registry record. Termination alone does not identify whether the reason was related to recruitment, safety, feasibility, administration, or another consideration. Nor does the record establish whether the planned 8 cycles were delivered, or why any participant may have stopped treatment. The arm description only states that participants would be removed after Cycle 2 if progression was documented.
The enrollment figure is important context for the scope of the evidence, but it should not be converted into a claim about precision without endpoint results and an analysis. The record does not give the number of participants included in the primary outcome analysis, the individual progression observations, or a confidence interval. Consequently, it is not possible to judge the uncertainty around the endpoint estimate from this record.
The record lists 2 outcome measures and 0 analyses. The absence of a posted analysis means there is no reported inferential result to interpret, such as a confidence interval or significance test. Any planning illustrations based on an observed effect should not be mistaken for results: the endpoint effect itself is not reported in the registry record, and the planning assumptions are not a substitute for an observed analysis.
Overall, the record describes a planned clinical question and a study structure, but it does not provide the primary outcome result needed to assess what was observed. It also does not provide a control comparison. Conclusions about efficacy, comparative benefit, or the size and precision of an effect are not supported by the information reported.
9. Statistical Methods Explained
The registry record identifies a primary endpoint but does not report a statistical analysis of that endpoint. The concepts below explain how such summaries are generally read; they do not imply that a particular analysis was performed in this study.
What does a median endpoint summarize?
For an endpoint recorded as cycles to progression, the median describes the central location of the observed cycle counts. Unlike a mean, the median is not pulled as strongly by unusually high or low observations. It can be useful when a distribution is skewed or contains a small number of extreme values. Its interpretation still depends on which participants contribute observations and how incomplete follow-up is handled.
The registry identifies full range as the dispersion measure. In general, a range gives the lowest and highest observed values and indicates the span of the observations. It is sensitive to extreme values and does not describe the distribution between those endpoints. The median and range together do not show every participant’s outcome or quantify uncertainty in the median. The registry does not report either numerical summary for this outcome.
Why does the time-to-event framing matter?
Progression is an event, and participants may have different lengths of observation. In general, time-to-event methods can account for participants whose event has not been observed by the end of follow-up, or whose follow-up ends for another reason, through censoring. The assumptions and rules for censoring can affect the result. The registry record does not state how censoring, incomplete follow-up, or participants without documented progression were handled. It therefore does not establish whether a simple median of observed cycles or a time-to-event estimator was used.
The endpoint is expressed in cycles, while its timeframe is 18 months. The schedule describes 21-day cycles and assessment after each cycle. This gives context for how progression was to be assessed, but does not supply the actual observation times or clarify the analysis rules for participants with differing follow-up. The full progression definition is also not present: the description refers to an increase of >25% in specified measures, then ends before completing the definition.
What would a confidence interval add?
In general, a confidence interval presents a range of values compatible with the observed data under a stated statistical procedure. It communicates precision as well as the estimated endpoint value. A narrow interval generally indicates greater precision than a wide interval, while an interval can still be compatible with a range of clinically different interpretations. A confidence interval does not correct for limitations in study design, such as the absence of a comparator.
No confidence interval for the primary endpoint is reported in the registry record. There is also no endpoint estimate to which an interval could be attached. Thus the record does not permit assessment of statistical precision, and the enrollment figure alone cannot supply that information.
How should planning calculations be distinguished from results?
Sample-size calculations are planning illustrations rather than evidence that a study achieved a particular level of power. The stated assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The calculation is described as based on a fraction of the observed effect. For time-to-event outcomes, the stated planning convention concerns the total number of events; for other outcomes, it concerns participants per arm.
These assumptions describe a hypothetical two-arm planning framework, whereas this study is recorded as single-group. The registry record does not report an observed primary effect, an analysis comparing arms, or a trial-specific design justification. No sample-size planning illustration should be interpreted as observed power or as evidence that the study could detect a stated effect. Because the endpoint result is not reported, the planning assumptions cannot be connected to an observed estimate in this record.
What can be inferred from the analysis record?
The registry records 0 analyses. That does not establish that no analyses were performed outside the registry; it means the registry record does not report an analysis. There is no reported inferential test, model, adjustment, confidence interval, or endpoint estimate to evaluate. The distinction is important: a registered outcome definition describes what was intended to be assessed, while a reported analysis describes what the data showed under a specified method.
The single-group design also limits the role of comparative methods. Without a control group, a between-group contrast cannot be estimated from the study as recorded. Descriptive summaries could still characterize the participants’ observed outcomes, but the registry does not provide those summaries for the primary endpoint. Statistical methods cannot recover missing results or remove the interpretive limits of the design.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 2 outcome measures, but only the primary endpoint is described in the available record: median number of cycles to tumor progression. It does not report the other outcome measure’s details in the registry record recorded. The presence of multiple outcomes can raise multiplicity concerns when several hypotheses are tested, because repeated testing can increase the chance of a statistically significant finding arising by chance. Whether that concern applies depends on the analyses planned and reported.
The registry records 0 analyses and does not report a multiplicity strategy, a hierarchy of outcomes, or adjustments for multiple comparisons. Since there is no reported test result, the record does not support a conclusion about whether multiplicity was addressed or whether any finding would remain after adjustment. The lack of reported methods should not be taken as proof that a particular strategy was or was not used outside the registry record.
Missing-data handling is not reported in the registry record. The record does not say how incomplete endpoint observations, missing assessments, withdrawals, or participants without documented progression were treated. For a progression endpoint, these details can matter because follow-up may end before the event is observed. Censoring definitions and follow-up procedures are also not reported.
Analysis populations are not reported. The record does not specify whether the endpoint was analyzed among all enrolled participants, those who started treatment, or another population. It also does not report how many participants contributed to the primary endpoint analysis. Therefore, the enrollment figure should not be assumed to equal the analysis population.
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 |
|---|---|---|---|
| Drug Combination | 0 | 5 | 0.0% |
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 |
|---|---|
| Drug Combination | 0 of 5 |
The registry lists a Drug Combination group and reports 5 at risk for the serious-event category, with 0 affected. For the other-event category, it also reports 5 at risk and 0 affected. These are category-specific figures; the record does not explain whether the same participants contributed to both categories. They should not be added together or treated as separate groups of participants.
The death count is null, so a death result is not reported in the registry record. The serious-event figures describe what is recorded for the stated group and categories. They do not establish that no safety events occurred outside those categories, or that every participant had complete safety follow-up. The registry does not provide the serious-event terms, event timing, duration, severity details beyond the category, or an explanation of how events were collected.
These safety figures are descriptive and do not compare the combination with another treatment. The record does not report a control group or a comparative safety analysis. A count of 0 affected in a category should therefore be read narrowly as the posted count for that category, rather than as proof of absence of risk.
12. What the Result Does, and Does Not, Mean
The record shows that a Phase II, single-group study of the named bevacizumab and bortezomib combination was registered for relapsed/refractory multiple myeloma. It identifies median cycles to tumor progression as the primary endpoint and describes a planned assessment schedule. It also records enrollment of 7 and a terminated status. These details establish the study’s stated purpose and design, not its clinical outcome.
The primary endpoint result is not reported in the registry record. There is no median, range, or confidence interval to indicate the observed progression timing or its precision. Consequently, the record does not show whether the observed outcome was consistent with the study’s objective, how much variation there was, or how uncertain an estimate might be.
The study has no described comparator. Even if a single-group outcome were reported, it would not provide a randomized comparison with another treatment or control condition. The stated aim of determining whether the combination had increased efficacy cannot be answered by the aim alone. A comparative conclusion would require appropriate comparative evidence, which is not reported in this record.
The record also does not explain why the study was terminated or whether all planned treatment and assessments were completed. It does not report the analysis population, missing-data procedures, or a statistical analysis of the primary endpoint. These omissions constrain interpretation independently of the small enrollment figure.
Accordingly, the registry record supports a description of the study’s design, planned regimen, and intended outcome. It does not support a conclusion that the regimen did or did not work, nor does it provide a quantitative estimate of efficacy or comparative safety. The safety counts should likewise be interpreted only as recorded category counts, not as a complete assessment of risk.
13. Why This Trial Matters Statistically
This record illustrates why a clearly defined endpoint and a reported endpoint result are both necessary for statistical interpretation. The registry identifies tumor progression in cycles as the primary outcome and gives a progression definition in part. However, without the observed median and range, readers cannot assess the outcome itself or its variability. An endpoint description alone is not a result.
The single-group design is another central statistical feature. It can describe outcomes among participants who receive a regimen, but without a comparator it cannot isolate a comparative treatment effect. This distinction is especially important because the study’s stated purpose refers to increased efficacy. A research question may be comparative even when the design, as recorded, does not include a concurrent comparison.
The registry also illustrates the difference between trial planning assumptions and evidence from an analysis. Planning calculations may depend on assumptions about an effect and a design, but they do not show what happened to participants. Here, the stated planning framework refers to two-arm allocation, while the study is recorded as single-group. With no endpoint result reported, those planning assumptions cannot be evaluated against observed data.
Finally, the record’s status and safety counts require careful, limited interpretation. Termination is recorded, but its reason is not. Safety categories have counts, but the death count is not reported and details about event collection are absent. Keeping these distinctions explicit prevents administrative entries or incomplete numerical summaries from being mistaken for efficacy or safety conclusions.
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 registry record does not report the primary endpoint result, its range, or an uncertainty interval. This prevents evaluation of the outcome and its precision. Although progression is defined in part, the definition is truncated after referring to an increase of >25% in specified measures. The complete criteria and adjudication procedures are not reported.
The study is listed as single-group, with no control group or randomized comparison described. This limits the kinds of conclusions the design could support, even if outcome summaries were available. The record also does not state why the study was terminated, whether participants completed the planned treatment, or how many contributed data to the endpoint.
The arm description names bevacizumab and bortezomib, but the interventions list names bortezomib only. The record does not provide a bevacizumab schedule in the interventions entry. The schedule information available describes bortezomib and planned cycles, not actual treatment received.
Analysis methods, analysis populations, missing-data handling, censoring procedures, and multiplicity methods are not reported in the registry record. The serious and other event categories each list 5 at risk and 0 affected, but the death count is null and further safety details are absent. These gaps mean neither the endpoint nor the safety entries can be treated as a comprehensive account of participant outcomes.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Phase II Study of Bevacizumab and Bortezomib in Patients With Relapsed/Refractory Multiple Myeloma — NCT00464178.
- Registry record last updated 2022-07-20; status last verified 2022-06.
- Results first posted 2022-07-20.
- 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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Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
NCT00464178 is a Phase II study of bevacizumab and bortezomib in relapsed/refractory multiple myeloma. It is recorded as single-group, with allocation listed as NA and masking as none. Enrollment is 7. The study started in 2007-04, had a primary completion date of 2009-02, and is listed as terminated.
The primary endpoint is median cycles to tumor progression, with a timeframe of 18 months. The arm description specifies bortezomib administration on Days 1, 4, 8, and 11, response assessment after each 21-day cycle, and 8 planned cycles. The registry record does not report the primary outcome result or a statistical analysis. It lists 2 outcome measures and 0 analyses. The reason for termination, analysis population, missing-data procedures, and complete progression definition are not reported in the registry record.