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 Bortezomib and Rituximab for Patients With Waldenstrom's Macroglobulinemia (NCT00492050), a na phase 2 study. The primary outcome is Response Rate After 2 Cycles of Treatment With Bortezomib and Rituximab.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00492050 |
| Phase | Phase 2 |
| Condition | Waldenstrom's Macroglobulinemia |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 46 |
| Arms in the primary outcome | n/a |
| Lead sponsor | M.D. Anderson Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
People with Waldenstrom's Macroglobulinemia were eligible for this research. The registry lists enrollment as 46. Its primary-outcome description notes that 1 subject did not complete 2 cycles of therapy and was not evaluable for response. Other details about the characteristics of the enrolled population are not reported in the registry record.
Intervention
The study intervention was bortezomib with rituximab. The listed regimen gives bortezomib 1.6 mg/m² intravenously weekly on Days 1, 8, 15 and 22, and rituximab 375 mg/m² intravenously on Day 8 and 22. Valacyclovir 500 mg orally daily, or acyclovir 200 mg orally twice daily, is also specified. The research description mentions stem-cell collection and assessment of safety and tolerability.
Comparator
There is no concurrent comparison group in the listed design. The study is recorded as single-group, with allocation listed as NA and masking listed as none. It therefore does not provide a randomized, like-for-like comparison of the regimen against another treatment or a control condition.
Primary question
The primary outcome is the count of participants classified by response after 2 (35 day) cycles of treatment with bortezomib and rituximab. The registry describes criteria for partial response, complete response and stable disease. However, the response count or response rate is not reported in the registry record. The available entry therefore identifies what was intended to be assessed, but does not establish how participants were classified or what the observed response result was.
3. Trial Design
This is a phase 2 clinical study of bortezomib and rituximab for people with Waldenstrom's Macroglobulinemia. The registry lists M.D. Anderson Cancer Center as the sponsor, an enrollment of 46, a start date of 2006-08-02 and a primary completion date of 2021-02-28. Its status is active, not recruiting. These record details describe the study's registration and status; they do not by themselves supply the response findings.
The design is listed as SINGLE GROUP. Allocation is NA and masking is NONE. There is no concurrent control arm in this record, so the study is not a randomized comparison. Its results, if reported, would describe outcomes among participants receiving the study regimen, rather than estimate a treatment contrast against another group. The record does not report a separate control intervention or a like-for-like comparator.
The listed regimen gives bortezomib 1.6 mg/m² IV weekly on Days 1, 8, 15 and 22, with rituximab 375 mg/m² IV on Day 8 and 22. It also lists daily valacyclovir 500 mg orally, with acyclovir 200 mg orally twice daily as an alternative. The primary response assessment is timed after 2 (35 day) cycles. The brief description also refers to collecting stem cells and examining safety and tolerability, including following treatment with other drug combinations. Further details about those later treatments are not reported in the registry record.
The registry lists 3 outcome measures and 0 analyses. The listed primary measure concerns participants, and the outcome is a count rather than a continuous measurement. The outcome description identifies a participant who did not complete 2 cycles and was not evaluable for response. The record does not report the overall response result, the number in each response category, or the analysis population used for a final estimate. Those omissions limit what can be concluded about outcomes from the entry.
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: 46 Participants consented, 9 screen failures.
Overall Study
| Stage | Bortezomib + Rituximab |
|---|---|
| STARTED | 37 |
| Inadequate Response to First Two Cycles | 25 |
| Inadequate Response to First 3 Cycles | 12 |
| If Adequate Response to First Two Cycles-participants Proceed to Stem Cell Collection | 30 |
| COMPLETED | 28 |
| NOT COMPLETED | 9 |
Reasons for not completing this period:
| Reason | Bortezomib + Rituximab |
|---|---|
| Adverse Event | 1 |
| Lack of Efficacy | 1 |
| Withdrawal by Subject | 3 |
| Unable to collect SCT | 2 |
| Progressive Disease | 2 |
Analysis population for the primary outcome, as defined in the registry: 1 subject didn't complete 2 cycles of therapy and isn't evaluable for response
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 | All Participants |
|---|---|---|
| Participants analysed | 37 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 28 |
| Age, Categorical: >=65 years | Participants | 9 |
| Age, Continuous | years | 61 (42 to 77) |
| Sex: Female, Male: Female | Participants | 9 |
| Sex: Female, Male: Male | Participants | 28 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 2 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 35 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 2 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 3 |
| Race (NIH/OMB): White | Participants | 30 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 2 |
| Region of Enrollment: United States | participants | 37 |
| Newly diagnosed patient with Waldenstrom Macroglobulinemia with no prior treatment | Participants | 37 |
6. Primary Endpoint
The registry lists the primary outcome as Response Rate After 2 Cycles of Treatment With Bortezomib and Rituximab, measured over After 2 (35 day) cycles of treatment.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is titled “Response Rate After 2 Cycles of Treatment With Bortezomib and Rituximab.” It is expressed as a count of participants and is assessed after 2 (35 day) cycles. The registry describes partial response as requiring at least > 50 % reduction of serum monoclonal IgM concentration, or > 50% decrease in adenopathy or organomegaly, with no new symptoms or signs of active disease. Complete response requires disappearance of serum and urine monoclonal protein, absence of malignant cells in bone marrow, resolution of adenopathy or organomegaly confirmed by CT, and no signs or symptoms attributable to WM. Stable disease is described as neither growth nor shrinkage of the amount of disease.
General: A response endpoint can make a treatment study's intended clinical outcome explicit by defining categories based on measurements and clinical findings. Clear definitions matter because different rules for classifying response can produce different counts. Here, the registry provides category definitions, but does not report the observed count or response rate. The outcome description also says participants will be followed for < 6 months after receiving bortezomib/rituximab or rituximab-modified hyper-CVAD to be considered unresponsive in accordance with the stated consensus guidelines. The record does not explain how that follow-up relates to the primary assessment beyond the wording in the outcome description.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The primary outcome is specified, but its result is not reported in the registry record. There is no response count, response rate, uncertainty interval or test result in the outcome information. Consequently, the posted record does not allow readers to assess how many participants had partial response, complete response, stable disease or another outcome. The fact that the endpoint is called a response rate does not supply the missing estimate.
The response assessment is intended to take place after 2 (35 day) cycles. The outcome description states that 1 subject did not complete 2 cycles and was not evaluable for response. That is relevant when interpreting any response estimate, because the number of participants included and the handling of people without an evaluable response can affect the reported result. The analysis population and the treatment of this participant in any final analysis are not reported in the registry record. It would not be appropriate to infer an evaluable sample size from the enrollment field and this note.
The design also shapes interpretation. This is a single-group study, with allocation listed as NA and masking listed as none. Without a concurrent randomized control group, an outcome among treated participants cannot be interpreted as a randomized estimate of the difference between the regimen and another approach. General: outcomes in a single group may reflect a range of influences, including participant characteristics and the course of the condition, as well as the intervention. The record does not provide a comparative estimate that separates those influences.
The registry's serious adverse event entry lists 37 at risk and 14 affected. The leading listed serious-event terms include Anemia and Hypotension at 5.4% each; Diarrhea, Elevated Alkaline Phosphate, Fever and Gram negative bacteremia are each listed at 2.7%. Deaths are listed as 0. These figures describe the posted safety record, but the entry does not provide the event-level details or an outcome comparison needed to interpret relative safety against another group.
The registry lists 3 outcome measures and 0 analyses. The endpoint result and analysis details are therefore not available in the record summarized by these registry entries. General: an estimate is more informative when accompanied by an uncertainty interval and a description of how the analysis population was defined. Neither can be assessed for this primary outcome from the reported entry. No conclusion about the magnitude or precision of the response outcome can be drawn from the endpoint definition alone.
9. Statistical Methods Explained
The record identifies a single-group phase 2 study and a count-based primary outcome. It does not report a statistical analysis plan, inferential method, confidence interval, hypothesis test or adjustment method for the primary outcome. Statistical interpretation should therefore distinguish the endpoint definition from an analysis result: a detailed definition describes what would count as response, but does not itself provide an estimate or its uncertainty.
What does a count-based outcome represent?
The primary measure is described as a count of participants. General: a count records how many people meet a specified outcome definition. A response rate expresses that count in relation to a defined analysis population, so both the numerator and the denominator need to be clear. The registry title uses the phrase “Response Rate,” while the measure is recorded as a participant count. The actual count, denominator and rate are not reported in the registry record. The entry therefore does not allow a reader to reconstruct a rate or judge its precision.
The outcome definition divides responses into categories, including partial response, complete response and stable disease. General: categories depend on operational rules, such as laboratory findings, imaging or examination, and symptoms. The rules help make classifications reproducible, but they do not guarantee that every participant can be classified. The record explicitly notes that 1 subject did not complete 2 cycles and was not evaluable for response. It does not say whether that subject was excluded, counted in another category, or handled in some other way in an analysis.
Why does the single-group design matter?
General: random allocation to treatment groups is one way to support a fair comparison, because it aims to balance participant characteristics across groups. This record lists a single group and allocation as NA, so no randomized group comparison is available. An outcome proportion in one group can describe what happened among participants included in that outcome analysis, but it cannot on its own estimate what would have happened to comparable participants under a different treatment. The record does not report a comparative effect estimate.
Masking is listed as NONE. General: masking can reduce the influence of knowledge about treatment assignment on some assessments or decisions. In a study without masking, awareness of treatment may be relevant to judgments or reporting, depending on how outcomes are assessed. The registry record does not provide details that would establish whether or how such influences were addressed for the response categories.
What does a confidence interval add?
General: a confidence interval describes the statistical uncertainty around an estimate under a specified analysis. A narrower interval generally indicates greater precision, while a wider interval indicates less precision. An interval does not resolve design limitations, missing outcome information or differences between treated and untreated groups. No confidence interval for the primary response outcome is reported in the registry record, so the precision of an observed response estimate cannot be assessed.
How should planning calculations be read?
The planning assumptions state that sample-size illustrations use a two-sided significance level of 0.05, equal allocation between two arms and a normal approximation, with the effect to detect expressed as a stated fraction of the observed effect. They also specify that time-to-event calculations use total events, while other calculations use participants per arm. These are planning assumptions, not evidence that this single-group study had two randomized arms. The record does not report the study's own sample-size justification or statistical plan.
General: a planning calculation estimates the amount of information a future design might need under chosen assumptions. Such a calculation depends on the assumed effect, outcome variability or event frequency, allocation, and the analysis method. It is not an observed treatment effect and does not show the probability that a completed study's result is correct. The planning material is explicitly an illustration based on the observed effect, rather than the trial's own design. Sample-size illustrations are not observed power. Since the primary response result is not reported in the registry record, readers cannot evaluate the observed response estimate against those planning assumptions from this record.
What analysis details are missing?
The registry lists 0 analyses. The statistical plan, handling of missing outcome data, analysis population, method for estimating uncertainty and any planned subgroup analyses are not reported in the registry record. General: these details help readers understand which observations contribute to an estimate and how conclusions depend on analytic choices. In their absence, the endpoint definition and the note about an unevaluable subject should not be treated as a complete account of the analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 3 outcome measures and 0 analyses. It does not report a statistical plan describing which outcomes were primary for inference, how multiple outcomes were to be handled, or whether any adjustment for multiple testing was planned. Although the primary measure is identified by name, that alone does not show how testing across outcomes was addressed.
General: when a study evaluates several outcomes or performs several statistical tests, the chance of at least one apparently notable result can increase. A prespecified testing strategy or an adjustment can address that issue, but the appropriate approach depends on the study questions and analysis plan. The registry record does not report such a strategy, so the consequences of multiplicity cannot be assessed.
The outcome description notes that 1 subject did not complete 2 cycles and was not evaluable for response. The record does not report a missing-data procedure, the analysis population, or whether the participant was excluded or otherwise included in the response analysis. General: excluding people without an observed outcome can affect interpretation if their outcome experience differs from that of participants who are evaluable. The direction and size of any such effect cannot be determined from this entry.
The record does not report whether an intention-to-treat, per-protocol or other analysis population was used. It also does not give a missing-data sensitivity analysis or specify how response status was determined when information was incomplete. These omissions mean the response estimate's dependence on analysis choices is not available for evaluation.
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 |
|---|---|---|---|
| All Participants | 14 | 37 | 37.8% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| All Participants | 0 of 37 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 1% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| All Participants | 14 of 37 |
Most frequent serious adverse event terms
| Event term | Organ system | All Participants |
|---|---|---|
| Anemia | Blood and lymphatic system disorders | 2 of 37 |
| Hypotension | Cardiac disorders | 2 of 37 |
| Diarrhea | Gastrointestinal disorders | 1 of 37 |
| Elevated Alkaline Phosphate | Gastrointestinal disorders | 1 of 37 |
| Fever | Infections and infestations | 1 of 37 |
| Gram negative bacteremia | Infections and infestations | 1 of 37 |
| Hypercalcemia | Metabolism and nutrition disorders | 1 of 37 |
| Neutropenia | Blood and lymphatic system disorders | 1 of 37 |
| Neutrophenic fever | Infections and infestations | 1 of 37 |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | 1 of 37 |
Most frequent non-serious adverse event terms
| Event term | Organ system | All Participants |
|---|---|---|
| Fatigue | General disorders | 6 of 37 |
| Pain (joint) | Musculoskeletal and connective tissue disorders | 4 of 37 |
| Pain (muscle) | Musculoskeletal and connective tissue disorders | 4 of 37 |
| Neutropenia | Blood and lymphatic system disorders | 4 of 37 |
| Diarrhea | Gastrointestinal disorders | 3 of 37 |
| Dry eye | Eye disorders | 3 of 37 |
| Leukopenia | Blood and lymphatic system disorders | 3 of 37 |
| Dizziness | Nervous system disorders | 2 of 37 |
| Vomiting | Gastrointestinal disorders | 2 of 37 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 2 of 37 |
Serious events with the highest arm-level rate: anemia (5.4%), hypotension (5.4%), diarrhea (2.7%), elevated alkaline phosphate (2.7%), fever (2.7%).
The registry's serious adverse event summary lists 37 participants at risk and 14 affected. It lists 0 deaths. The most frequent serious-event terms named are Anemia and Hypotension, each at 5.4%. Diarrhea, Elevated Alkaline Phosphate, Fever and Gram negative bacteremia are each listed at 2.7%. These figures are the entries in the registry record; no comparison group is listed for interpreting them against another regimen.
General: an “at risk” count identifies the group used for an adverse-event summary, while an “affected” count identifies participants recorded as having an event under the relevant summary definition. The registry also gives 37 at risk and 14 affected for other adverse events. Those are descriptive safety summaries, not estimates of the difference in risk between treatments. The record does not report timing, duration, severity grading details, recurrence, attribution to treatment, or participant-level event histories for these terms.
The listed percentages should be read as the registry's reported values, not as evidence that all events were captured or that the same monitoring period applied to every participant. Details about exposure time and the safety analysis methods are not reported in the registry record. The absence of a listed death count does not answer questions about other safety outcomes, and the single-group design does not support a randomized safety comparison.
12. What the Result Does, and Does Not, Mean
The record establishes that the study was designed to assess response after 2 cycles of bortezomib and rituximab in people with Waldenstrom's Macroglobulinemia. It also provides response-category definitions and a note that 1 subject did not complete 2 cycles and was not evaluable for response. These details clarify the intended endpoint and identify a limitation in outcome ascertainment.
The record does not report the primary response count or rate. It therefore does not show the proportion classified as having partial response, complete response or stable disease, nor does it report an uncertainty interval for the endpoint. No conclusion about the magnitude or precision of the response finding can be drawn from the endpoint title or its definition.
The study's single-group design means its reported outcomes, if available, would be descriptive of the participants analyzed. It would not provide a randomized comparison against a control group. General: a treatment outcome observed in one group is not by itself a causal estimate of what the same participants would have experienced under another approach. The registry record does not report a comparative result or an analysis that could address that question.
The serious adverse event summary records 14 affected among 37 at risk, with 0 deaths listed and several event terms with reported percentages. These entries describe the safety information in the record; they do not establish a relative safety profile because there is no concurrent control group and event details are limited. The registry also does not report the statistical plan, response analysis population, or handling of missing response data.
Overall, the registry record supports a description of study design, regimen, endpoint definitions and selected safety entries. It does not provide the primary outcome result needed to assess the study's observed response, and it does not support claims about comparative benefit or risk.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of why a clearly named endpoint and a reported result are different things. The registry specifies response categories and an assessment time after 2 (35 day) cycles, but does not report the response count or rate. Without the result and its analysis denominator, the central endpoint cannot be evaluated quantitatively.
The design also matters. The record identifies a single-group phase 2 study, with no masking and no randomized comparator. Such a design can describe outcomes in the group receiving the regimen, but does not produce a like-for-like treatment contrast. General: a comparator helps distinguish an outcome associated with a treatment from the outcome that might otherwise have occurred. Without one, readers must avoid interpreting a within-group result as a randomized estimate of treatment effect.
The note that 1 subject did not complete 2 cycles and was not evaluable highlights the importance of analysis-population definitions. General: an outcome estimate is easier to interpret when the record explains who was included, how unavailable outcomes were handled, and how uncertainty was quantified. Those methods are not reported. The registry also lists 3 outcome measures but 0 analyses, leaving multiplicity and analytic choices unresolved.
Finally, the safety entries illustrate the difference between reporting adverse events and comparing safety. Event terms and participant counts can describe what was recorded, but a single-group summary cannot determine how those outcomes compare with an alternative. The record's value is in documenting the study question, regimen and selected outcomes; its gaps limit statistical conclusions about response and comparative effects.
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 limitation of the registry entry is that the primary response result is not reported. Although the endpoint and response definitions are available, the record gives no response count, response rate, uncertainty interval or inferential result. A reader cannot assess the observed response from these details alone.
The study is listed as single-group, with allocation NA and masking none. It has no concurrent randomized comparison in the record. Any outcome description would therefore lack a like-for-like control comparison, and the entry does not provide a comparative effect estimate. The record does not report participant characteristics that would allow assessment of how broadly the study population represents other groups.
The endpoint description states that 1 subject did not complete 2 cycles and was not evaluable for response. The analysis population and handling of that missing response are not reported. The registry also does not provide the statistical analysis plan, sample-size justification, missing-data methods, multiplicity strategy or details of the method used to calculate uncertainty.
The safety summary is limited to the listed counts, percentages and terms. Event timing, duration, severity details, attribution, follow-up exposure and comparative safety results are not reported in the registry record. The entry's reference to stem-cell collection and later drug combinations does not provide further results about those activities. These limitations constrain interpretation to what the record explicitly documents; they do not establish that unreported analyses or information were absent from all study materials.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Bortezomib and Rituximab for Patients With Waldenstrom's Macroglobulinemia — NCT00492050.
- Registry record last updated 2026-03-11; status last verified 2026-02.
- Results first posted 2022-05-04.
- 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
This phase 2 study, sponsored by M.D. Anderson Cancer Center, concerns bortezomib and rituximab for Waldenstrom's Macroglobulinemia. It is recorded as single-group, with allocation NA and masking none. The registry lists enrollment as 46, a start date of 2006-08-02, a primary completion date of 2021-02-28 and status active, not recruiting.
The primary outcome is a participant count for response after 2 (35 day) cycles. The registry defines partial response, complete response and stable disease, and notes that 1 subject did not complete 2 cycles and was not evaluable for response. The response result and analysis methods are not reported in the registry record.
The safety summary lists 37 at risk and 14 affected, with 0 deaths. The record lists 3 outcome measures and 0 analyses. Its design does not include a concurrent randomized comparator.