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 Rituximab, Cyclophosphamide, Bortezomib, and Prednisone in Patients With Stage III/IV FL or MZL (NCT00772668), a na na study. The primary outcome is Overall Response Rate, According to the International Workshop Criteria (IWC) for Non-Hodgkin's Lymphoma (NHL).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00772668 |
| Phase | NA |
| Condition | Lymphoma, Follicular Lymphoma, Marginal Zone Lymphoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 3 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Miami |
| Results posted | Yes |
2. Clinical Question
Population
People with stage III or stage IV follicular lymphoma or marginal zone lymphoma were the intended population. The registry record lists lymphoma, follicular lymphoma, and marginal zone lymphoma as conditions. Enrollment was 3. The record does not report further details about the characteristics of participants who enrolled.
Intervention
The study examined RCVELP, a regimen of rituximab, cyclophosphamide, bortezomib, and prednisone, as first-line therapy. The registry describes a single-group study. The available regimen description is incomplete, so the full treatment schedule is not reported in the registry record.
Comparator
There was no separate control group in this single-group design. Allocation is listed as NA, and masking as none. Consequently, the study does not provide a randomized, like-for-like comparison with another treatment or with a control intervention.
Primary question
The primary outcome was overall response rate according to International Workshop Criteria for non-Hodgkin's lymphoma. It included complete response, complete response unconfirmed, and partial response. The intended assessment schedule was after cycles 2, 4, 6, and 8, then every 3 months, for about 2 years. The response data were not analyzed because the required minimum enrollment of 30 was not met and the study ended early due to lack of funding.
3. Trial Design
This was a pilot study of RCVELP as first-line therapy for follicular lymphoma and marginal zone lymphoma. It is recorded as a single-group study, with no masking. Allocation is listed as NA; the record does not describe an assignment process or a concurrent comparison group. The study is not a randomized comparison, so its design cannot estimate a treatment contrast against a control arm.
The registry lists enrollment as 3 and the study status as terminated. The start date is 2009-09-25, and primary completion is recorded as 2010-12. The registry states that the primary outcome required a minimum enrollment of 30 participants and that the data were not analyzed because that requirement was not met and the study was terminated early due to lack of funding. A planned sample size or a rationale for that minimum is not reported in the registry record.
The intervention combined rituximab, cyclophosphamide, bortezomib, and prednisone. The registry's available intervention description includes only part of the regimen schedule, so other dosing and administration details are not reported in the registry record. The listed secondary outcomes were progression-free survival, overall survival, and toxicity. Although the registry lists four outcome measures, it reports no analyses. The study phase is listed as NA.
These design features define the scope of interpretation. The record documents a small pilot with no control group and no analyzed primary response outcome. It therefore does not provide an estimate of comparative efficacy. The absence of an analysis also means there is no reported primary response estimate or precision interval to interpret. The listed outcomes should be distinguished from outcomes with reported results: listing an outcome does not establish that it was analyzed.
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 | RCVELP |
|---|---|
| STARTED | 3 |
| COMPLETED | 0 |
| NOT COMPLETED | 3 |
Reasons for not completing this period:
| Reason | RCVELP |
|---|---|
| Physician Decision | 3 |
Analysis population for the primary outcome, as defined in the registry: A minimum enrollment of 30 participants was required for this outcome measure. Due this minimum requirement not being met, and the early termination of the study due to lack of funding, the data for this outcome measure were not analyzed.
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 | RCVELP |
|---|---|---|
| Participants analysed | 3 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 3 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 2 |
| Sex: Female, Male: Male | Participants | 1 |
| Region of Enrollment: United States | participants | 3 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Response Rate, According to the International Workshop Criteria (IWC) for Non-Hodgkin's Lymphoma (NHL), measured over Post cycles 2,4,6,8 and then every 3 months, about 2 years.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was overall response rate under the International Workshop Criteria for non-Hodgkin's lymphoma. It groups complete response, complete response unconfirmed, and partial response. In general, a response endpoint is used to describe whether disease measurements meet prespecified criteria for a level of tumor reduction or disappearance. It can provide a structured assessment of activity over a defined period, but it is not by itself a comparison with another treatment or a complete account of longer-term outcomes.
The registry describes complete response as disappearance of detectable clinical and radiographic disease, disappearance of disease-related symptoms if present before therapy, and normalization of biochemical abnormalities attributable to lymphoma. Complete response unconfirmed allows the stated residual lymph node mass condition or indeterminate bone marrow. Partial response includes a >= 50% decrease in the sum of the product of the perpendicular diameters of the six largest dominant nodes or masses, with no increase in other nodes, liver, or spleen. These categories rely on specified clinical and imaging criteria.
Assessments were planned after cycles 2, 4, 6, and 8, and then every 3 months, for about 2 years. The registry says the primary response data were not analyzed because the required minimum enrollment of 30 was not reached and the trial was terminated early due to lack of funding. Thus, the endpoint is defined, but no response rate, response category count, or uncertainty estimate is reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central result is the absence of an analyzed primary response outcome. The registry defines overall response rate using complete response, complete response unconfirmed, and partial response, but explicitly states that the data for this outcome were not analyzed. There is therefore no posted estimate of response rate, no confidence interval for that rate, and no inferential test of the primary endpoint to interpret.
Enrollment was 3, while the registry states that a minimum enrollment of 30 participants was required for the primary outcome. The record attributes the lack of analysis to failure to meet that minimum and early termination due to lack of funding. The study status is terminated. These registry entries explain why the primary result is unavailable, but they do not establish what the response outcome would have been with further enrollment or follow-up.
The design was single-group, with no masking and no separate comparator. Even if an outcome estimate had been reported, a single-group result would describe observations in the treated group rather than a randomized contrast. Without a control group, observed outcomes cannot distinguish the intervention's contribution from other influences on the outcome. Because the primary outcome was not analyzed, even a descriptive response estimate is absent.
Progression-free survival, overall survival, and toxicity are listed as secondary outcomes. The registry reports no analyses. An outcome's presence in the registry does not mean its results were measured, analyzed, or posted. Interpretations of time-to-event outcomes or their precision would require results and analysis details that are not reported in the registry record.
The safety listing is descriptive. It records serious adverse event information for the RCVELP group, including diverticulitis, Grade 3, at 33.3%, with 1 serious event affected and 3 at risk; deaths are recorded as 0. These observations are limited by the small group and should not be treated as a stable estimate of event frequency or as a comparative safety result. The registry also records 0 other adverse events affected among 3 at risk. Those figures do not provide a complete safety profile or support comparison with another regimen.
Overall, the registry record documents that the pilot was conducted and terminated, but does not supply an analyzed primary efficacy result. Any planning illustrations based on assumed statistical inputs are not trial findings and are not observed power. They cannot fill the gap left by the unreported response analysis. The interpretation should remain confined to the design, enrollment, termination, and descriptive safety information recorded.
9. Statistical Methods Explained
What does a single-group design estimate?
In general, a single-group study describes outcomes observed among participants receiving the study intervention. It does not directly estimate a difference between that intervention and a concurrent control. A randomized comparison helps balance known and unknown influences across groups; without that comparison, changes in outcomes may have more than one explanation. This is a general design principle, not an additional finding about the participants in this study.
The registry classifies this study as single-group, with no masking and allocation listed as NA. Its record does not describe a comparator arm. The listed enrollment is 3. These details matter because a response proportion, if it had been analyzed, would have represented the observed group under the study conditions, not a causal treatment effect established by a controlled contrast.
What does an overall response rate represent?
In general, an overall response rate is the proportion of an evaluated group meeting one of the response definitions specified in advance. The categories used were complete response, complete response unconfirmed, and partial response under International Workshop Criteria. The endpoint definition sets the criteria for classifying response; an endpoint definition alone does not give the observed response rate. That requires analyzed participant-level outcome data and a clearly described denominator.
The registry specifies criteria involving clinical and radiographic disease, symptoms, biochemical abnormalities, lymph node measurements, and other disease sites. The stated partial-response criterion includes a >= 50% decrease in the sum of the product of perpendicular diameters for the six largest dominant nodes or masses, with no increase in size of other nodes, liver, or spleen. These criteria make the endpoint more standardized than an informal judgment, but classification still depends on assessment and data collection. Whether independent review, blinded assessment, or adjudication was used is not reported in the registry record.
Why does precision matter?
In general, a confidence interval describes uncertainty around an estimated quantity under the analysis assumptions. A narrow interval indicates greater statistical precision than a wide interval, although precision does not by itself establish clinical importance or eliminate bias. For a proportion, uncertainty depends in part on how many observations contribute and how the outcome is distributed. When the registry reports that the primary outcome was not analyzed, there is no response estimate or interval from which to assess precision.
Small enrollment also means that any descriptive observations can be highly sensitive to individual outcomes. This general point does not create an estimate for this study. The record does not report a response count, response denominator, or primary analysis. It is therefore not appropriate to infer a response rate or to construct an interval from the enrollment figure alone.
What is the role of hypothesis tests?
In general, a hypothesis test evaluates how compatible observed data are with a specified null model, given the assumptions of the test. A p-value does not measure the probability that a treatment works, nor does it measure the size or importance of an effect. Interpretation also requires the analysis population, endpoint definition, model, and handling of missing observations. No primary analysis or p-value is reported in the registry record.
The record does not report a statistical analysis plan, the test intended for the primary endpoint, or whether any interim analyses were planned or conducted. Those details should not be inferred from the endpoint description or from a pilot label. The registry lists zero analyses, which reinforces that the outcome listings should not be read as evidence that statistical tests were completed.
How should planning calculations be read?
In general, a sample-size calculation is a planning exercise based on assumptions about a target effect, variability, significance level, allocation, and analysis method. It does not measure the probability that an already completed study has detected an effect. Sample-size illustrations are not observed power and do not substitute for an analyzed result.
The planning assumptions associated with the statistical material use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; time-to-event calculations are described in terms of total events, while other calculations are expressed per arm. Those are planning assumptions, not a description of the actual single-group study design. They should not be mistaken for evidence that this study had two arms or for a trial-specific efficacy analysis. The registry record does not report the study's own statistical plan or sample-size justification.
What does over-dispersion mean in general?
Over-dispersion occurs when observed variability is greater than a statistical model expects. In general, ignoring it can make standard errors too small and confidence intervals too narrow, making results appear more precise than warranted. Appropriate modeling depends on the outcome type and data structure. The registry record does not identify an over-dispersion model or provide analyzed outcomes for which its relevance could be assessed.
These concepts explain how results would ordinarily be evaluated, but they cannot recover results that were not analyzed. Here, the primary endpoint has an operational definition and planned assessment times, while its analysis and associated uncertainty are absent from the registry record.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity arises when several hypotheses or outcomes are tested, increasing the chance of at least one apparently unusual result if each is interpreted separately. In general, an analysis plan may identify a primary endpoint, define how secondary outcomes will be handled, and specify any adjustment or prioritization strategy. The registry identifies overall response rate as the primary outcome and lists progression-free survival, overall survival, and toxicity as secondary outcomes. Whether multiplicity adjustments or a testing hierarchy were planned is not reported in the registry record.
The primary response data were not analyzed, and the registry lists zero analyses. It therefore reports no primary or secondary hypothesis tests for which multiplicity handling can be evaluated. The analysis plan, if any, and the intended approach to multiple outcomes are not reported in the registry record. No interim analysis plan or interim results are reported in the registry record.
Missing data also affect interpretation. In general, missing outcome assessments can alter an estimated response proportion, particularly if availability of follow-up is related to health status or treatment experience. A complete-case analysis, an imputation method, and a sensitivity analysis answer different questions and rely on different assumptions. The registry record does not describe missing response assessments, reasons for missingness, an imputation method, or a sensitivity analysis. Since the outcome was not analyzed, the record does not establish how participants without an evaluable response would have been handled.
The analysis population is likewise not reported in detail. The registry does not state whether the intended population was all enrolled participants, participants who began treatment, or participants with an evaluable response. It would be inappropriate to assume an intention-to-treat or per-protocol analysis. The reported enrollment of 3 should not be treated as an endpoint denominator because the registry states that the endpoint data were not analyzed.
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 |
|---|---|---|---|
| RCVELP | 1 | 3 | 33.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| RCVELP | 0 of 3 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| RCVELP | 0 of 3 |
Most frequent serious adverse event terms
| Event term | Organ system | RCVELP |
|---|---|---|
| Diverticulitis, Grade 3 | Gastrointestinal disorders | 1 of 3 |
Serious events with the highest arm-level rate: diverticulitis, grade 3 (33.3%).
The registry records safety information for the RCVELP group, with 3 at risk for serious adverse events and 1 affected. The listed serious adverse event term is diverticulitis, Grade 3, at 33.3%. Deaths are recorded as 0. For other adverse events, the registry records 0 affected among 3 at risk. These are descriptive registry figures, not estimates from a controlled safety comparison.
The serious event count and the percentage describe a very small group, so they should not be interpreted as a stable estimate of how often an event would occur in a broader population. With no comparator arm, the record cannot show whether the event frequency differs from another treatment or from background risk. The registry information also does not establish causality for the reported event. The listed term, grade, and frequency should be read as the reported safety record, not as proof of a treatment-related effect.
The registry lists toxicity as a secondary outcome, but reports no analyses. The serious and other adverse event figures therefore do not amount to a complete analyzed safety assessment. The record does not report a broader analysis of exposure, duration, event timing, or participant-level context. These limitations are especially important when reading percentages based on a very small at-risk group.
12. What the Result Does, and Does Not, Mean
The record establishes that a pilot study of RCVELP as first-line therapy for stage III/IV follicular lymphoma or marginal zone lymphoma was registered as a single-group study and enrolled 3. It also establishes that the study was terminated and that the primary response outcome was not analyzed. The registry attributes the absence of primary outcome analysis to the unmet minimum enrollment requirement and early termination due to lack of funding.
It does not establish an overall response rate. Although the response categories and planned assessment schedule are specified, no analyzed response data, response estimate, or confidence interval is reported. The primary endpoint cannot be interpreted as positive or negative from the registry record. A missing estimate is not evidence that no responses occurred, and it is not evidence that responses did occur.
The record also does not establish comparative effectiveness. The design had no separate control group, so there is no randomized contrast. The listed secondary outcomes, including progression-free survival and overall survival, have no reported analyses. The available safety entries describe events in the RCVELP group but cannot establish comparative risk or a causal relationship.
General statistical explanations and planning calculations should not be confused with observed trial results. Planning illustrations depend on assumptions and do not replace the primary analysis. Nor do they provide observed power. The registry record does not report the protocol's statistical analysis plan or a justification for the stated minimum enrollment.
The appropriate interpretation is limited: this was a small, terminated pilot with a defined but unanalyzed primary endpoint, no control group, and sparse descriptive safety information. The record does not resolve whether the regimen changes response, survival, or toxicity compared with another approach.
13. Why This Trial Matters Statistically
This trial is statistically informative because it illustrates the difference between defining an outcome and producing an analyzable estimate. The registry gives a detailed definition of overall response and planned assessment times, but reports that the primary outcome data were not analyzed. An endpoint can be clinically meaningful and carefully specified while still yielding no result available for estimation or inference.
It also highlights how study conduct and design determine what can be learned. The single-group structure offers no randomized comparator, and the small enrollment did not meet the stated minimum requirement for the primary outcome. Early termination due to lack of funding prevented the planned outcome analysis described in the record. These are not merely technical details: they limit both the precision of any descriptive account and the ability to attribute outcomes to an intervention.
The registry further shows why outcome listings must be distinguished from outcome results. Progression-free survival, overall survival, and toxicity appear as secondary outcomes, but the record reports no analyses. Likewise, descriptive safety counts should not be mistaken for a comparison or a stable estimate of risk.
For clinical researchers, this case underscores the value of transparent reporting of analysis populations, missing-data handling, statistical plans, and reasons for non-analysis. Those details can clarify what an absent result means and whether any planned inference was possible. In this record, the documented details support a clear account of the design and the reason the primary endpoint was unavailable, while leaving efficacy and comparative safety unresolved.
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 main limitation is that the primary endpoint was not analyzed. The registry says a minimum enrollment of 30 was required for the overall response outcome, while enrollment was 3, and attributes non-analysis to early termination due to lack of funding. No response estimate, confidence interval, or test is reported.
The single-group design has no concurrent comparator, and masking is listed as none. Therefore, even a reported outcome would not provide a randomized treatment contrast. The record does not report participant characteristics, an analysis population, response-evaluability rules, missing-data methods, or sensitivity analyses. These omissions prevent assessment of how enrolled participants would have contributed to an endpoint analysis.
The trial is identified as a pilot, but the registry record does not report a sample-size justification or a statistical analysis plan. It also does not report whether interim analyses were planned, how secondary outcomes were to be analyzed, or how multiplicity would be handled. The intervention description is incomplete, so the full regimen schedule cannot be reconstructed from the record.
Safety information is limited to the reported event entries, with very few at risk, and there are no comparative safety results. The registry lists progression-free survival and overall survival but does not report analyses or follow-up results for those outcomes. Consequently, both efficacy and broader safety interpretation remain limited by absent outcome analyses and the lack of a control group.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Rituximab, Cyclophosphamide, Bortezomib, and Prednisone in Patients With Stage III/IV FL or MZL — NCT00772668.
- Registry record last updated 2017-12-11; status last verified 2017-11.
- Results first posted 2013-02-21.
- 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
NCT00772668 is a University of Miami pilot study of RCVELP as first-line therapy for stage III/IV follicular lymphoma or marginal zone lymphoma. The study was single-group, unmasked, and listed with phase NA. Enrollment was 3. The recorded start date is 2009-09-25, primary completion is 2010-12, and status is terminated.
The primary outcome was overall response rate under International Workshop Criteria, assessed after cycles 2, 4, 6, and 8 and then every 3 months, for about 2 years. The registry states that the data were not analyzed because the required minimum enrollment of 30 was not met and the study ended early due to lack of funding. Secondary outcomes listed were progression-free survival, overall survival, and toxicity; the registry reports no analyses.
The safety record lists diverticulitis, Grade 3, at 33.3%, with 1 serious event affected among 3 at risk, and 0 deaths. No controlled comparison or analyzed primary efficacy result is reported.