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 Study of Velcade and Temsirolimus for Relapsed or Refractory Non-Hodgkin Lymphoma (NCT01281917), a na phase 2 study. The primary outcome is Overall Response Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01281917 |
| Phase | Phase 2 |
| Condition | Non-Hodgkins Lymphoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 40 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Wisconsin, Madison |
| Results posted | Yes |
2. Clinical Question
Population
People with relapsed or refractory B-cell non-Hodgkin lymphoma. The registry record gives an enrollment of 40 and lists non-Hodgkins lymphoma as the condition.
Intervention
Velcade combined with temsirolimus. The recorded regimen is Velcade 1.6 mg/m2 weekly on days 1, 8, 15, and 22, with temsirolimus 25mg IV weekly on days 1, 8, 15, 22, and 29. Treatment was planned for up to 6 cycles, each 35 days long.
Comparator
No comparator arm is listed. This is a single-group study, not a randomized comparison.
Primary question
Whether the combination provides benefit as assessed by overall response rate, defined as complete response plus partial response.
The clinical question is about tumor response among people whose B-cell non-Hodgkin lymphoma had relapsed or was refractory. The registry record describes complete response as disappearance of all target lesions and partial response as a decrease of 30% or greater in the sum of the longest diameters of target lesions. The assessment criteria are identified as RECIST v1.0, with target lesions assessed by MRI.
The record frames the study as an evaluation of the combination, not a direct test of whether it is better than another treatment. Because no comparison group is listed, the study cannot estimate a between-group treatment effect from the design described in the record. Overall response rate can describe observed tumor responses in the treated study group, but interpreting that rate depends on the reported estimate, its uncertainty, and the analysis population. The primary response estimate and its confidence interval are not reported in the registry record.
3. Trial Design
The registry record describes a phase II, single-group study sponsored by the University of Wisconsin, Madison. Allocation is listed as not applicable and masking as none. The study status is completed, and the recorded enrollment is 40. The record gives a start of 2011-02 and primary completion of 2013-10.
The listed intervention is Velcade plus temsirolimus. The regimen description specifies Velcade 1.6 mg/m2 weekly on days 1, 8, 15, and 22, and temsirolimus 25mg IV weekly on days 1, 8, 15, 22, and 29. Treatment was to continue for up to 6 cycles, with cycles lasting 35 days. These are regimen details in the record; adherence, dose modifications, treatment exposure, and the number completing treatment are not reported in the registry record.
A single-group design can characterize outcomes among participants receiving the regimen, but it has no concurrent control group for separating treatment-associated outcomes from the course of disease or other influences. The record does not describe random assignment, and the absence of masking is consistent with its listing of masking as none. No comparative treatment effect can be inferred from the design alone.
The record lists safety, complete response rate, tolerability, duration of response, and overall survival as secondary outcomes. It identifies seven outcome measures, but the registry record does not provide a statistical analysis entry. The protocol, eligibility details beyond the condition and relapsed or refractory status, and the planned methods for handling treatment discontinuation are not reported in the registry record.
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: Forty participants were enrolled from 10 sites within the Wisconsin Oncology Network (WON) over 2 years. One participant withdrew consent immediately after enrollment and was never treated. Therefore, results are reported on the remaining 39 participants.
Overall Study
| Stage | Velcade Plus Temsirolimus |
|---|---|
| STARTED | 40 |
| COMPLETED | 39 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Velcade Plus Temsirolimus |
|---|---|
| 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: The study enrolled patients with relapsed and refractory B-cell non-Hodgkin lymphoma.
| Characteristic | Unit | Velcade Plus Temsirolimus |
|---|---|---|
| Participants analysed | 39 | |
| Age, Continuous | years | 68 (38 to 89) |
| Sex: Female, Male: Female | Participants | 11 |
| Sex: Female, Male: Male | Participants | 28 |
| Region of Enrollment: United States | participants | 39 |
| Number of prior treatments | Prior treatments | 4 (1 to 11) |
| Histology: DLBCL | Participants | 18 |
| Histology: Follicular lymphoma | Participants | 9 |
| Histology: Mantle cell lymphoma | Participants | 7 |
| Histology: Small lymphocytic lymphoma | Participants | 3 |
| Histology: Marginal zone lymphoma | Participants | 2 |
| ECOG Performance Status: 0 | Participants | 17 |
| ECOG Performance Status: 1 | Participants | 16 |
| ECOG Performance Status: 2 | Participants | 6 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Response Rate, measured over Up to 60 months.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 95% Confidence Interval |
| Unit | Percentage of participants |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is overall response rate, expressed in the registry record as a percentage of participants. The stated definition is the sum of participants with a complete response and those with a partial response. A complete response is described as disappearance of all target lesions; a partial response is described as a decrease of 30% or greater in the sum of the longest diameters of target lesions. The record identifies RECIST v1.0 and MRI assessment for target lesions.
In general, an overall response rate condenses tumor response into a summary of whether participants meet a response criterion during the specified assessment process. It can describe the observed frequency of complete or partial responses in a study group. Its interpretation depends on which participants are included in the denominator, how response is assessed over time, and how missing or unevaluable assessments are handled. Those details are not reported in the registry record.
The primary endpoint timeframe is listed as up to 60 months. The record labels the measure as a median with a 95% confidence interval, but does not report a numerical response estimate or interval. Accordingly, the endpoint definition is available, while the primary result and its precision are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the study question and endpoint definition, but it does not provide a numerical overall response rate. It also does not report a confidence interval for that endpoint. As a result, the posted record does not allow readers to assess the observed frequency of complete or partial response or the precision of an estimate. No estimate should be inferred from the enrollment or the endpoint description.
The design matters to interpretation. This is a phase II single-group study, with no comparator arm listed. An observed response rate, if reported, would describe the study group under the response criteria used. It would not by itself establish how that outcome compares with another regimen, usual care, or the natural course of disease. The record does not list random allocation, and the skip note explicitly states that the study is not a randomized comparison.
The primary endpoint is framed around response, while the record also lists duration of response and overall survival among secondary outcomes. These outcomes address different aspects of experience: response concerns whether a response criterion is met, whereas duration of response and overall survival concern time. Their interpretation requires outcome definitions, follow-up information, and analysis details. Numerical results and analysis methods for these outcomes are not reported in the registry record.
Several adverse-event counts are recorded for the Velcade plus temsirolimus group. The safety record lists 18 participants at risk for serious adverse events and 18 affected; for other adverse events, it lists 39 at risk and 35 affected. It also lists 16 deaths. These are descriptive registry figures. The record does not establish from these counts whether an event was caused by treatment, nor does it provide a comparison group that would support a comparative safety estimate.
The record lists seven outcome measures and zero analysis entries. This does not provide a statistical analysis of the primary endpoint or secondary outcomes. A sample-size calculation, a p-value, an adjusted estimate, and an analysis population are not reported in the registry record. Thus, interpretation is limited to the design, definitions, and descriptive safety counts that are explicitly recorded; efficacy and precision cannot be judged from a result that is not posted.
9. Statistical Methods Explained
The registry record identifies a phase II, single-group study with no masking. These design features determine the kinds of statistical conclusions the study can support. The explanations below describe general statistical principles; they do not imply that a particular method was used in this study unless the registry record says so.
What can a single-group design estimate?
In general, a single-group study can summarize outcomes observed among participants who receive the intervention. For a response endpoint, the main descriptive quantity is the response frequency under the study's assessment rules. That summary depends on the denominator: for example, whether it includes all enrolled participants, only participants who began treatment, or only those with an evaluable assessment. The denominator rule for the primary endpoint is not reported in the registry record, and no response estimate is reported.
Without a concurrent comparator, a single-group result cannot isolate the effect of treatment from other explanations for observed outcomes. Differences in disease course, participant characteristics, assessment practices, and care outside the study can affect outcomes. A randomized comparison is designed to support a more direct contrast between treatment groups; this record does not describe such a comparison. The record itself warns that its arms are not a like-for-like control comparison.
What does a confidence interval communicate?
In general, a confidence interval expresses statistical uncertainty around an estimate. A narrower interval indicates greater precision under the method's assumptions; a wider interval indicates less precision. It does not remove potential bias or make a study comparable with a control group. The primary endpoint is labelled as having a 95% confidence interval, but neither the numerical estimate nor its interval is reported in the registry record. Therefore, the precision of the primary result cannot be evaluated from the posted record.
How does the response definition work?
The endpoint definition combines complete and partial responses. The record describes complete response as disappearance of all target lesions, and partial response as a decrease of 30% or greater in the sum of the longest diameters of target lesions. In general, applying a response rule requires consistent assessment and a specified approach to participants whose response cannot be evaluated. The record identifies RECIST v1.0 and MRI, but does not report the assessment schedule, confirmation requirements, independent review procedures, or missing-assessment rules.
The registry record calls the primary measure a percentage of participants and labels its parameter as a median with a 95% confidence interval. No numerical value accompanies that description. The endpoint's denominator, timing of response classification, and the relationship between the percentage measure and the median label are not clarified in the registry record, so these should not be resolved by assumption.
Why do analysis populations matter?
In general, an analysis population specifies which participants contribute to an outcome estimate. Including all enrolled participants can answer a different question from including only participants with evaluable response assessments. Excluding participants without an assessment can affect an estimate if missingness is related to prognosis or treatment experience. The registry record does not identify an analysis population for the primary endpoint or describe how missing data were handled.
The enrollment is recorded as 40, while the safety record lists 39 at risk for other adverse events and 18 at risk for serious adverse events. These figures refer to the enrollment and safety categories as recorded; the registry record does not explain the differences in the listed at-risk counts. They should not be treated as a substitute for a defined primary-endpoint analysis population.
What can be concluded from the posted analysis information?
The registry record lists zero analysis entries. It therefore does not report a statistical model, inferential test, adjustment strategy, handling of repeated assessments, or methods for time-to-event outcomes. In general, a statistical plan would specify the estimand, analysis population, endpoint calculation, uncertainty method, and approach to missing data. Whether such a plan existed outside the registry record is not reported in the registry record.
The registry record's listed secondary outcomes include safety, complete response rate, tolerability, duration of response, and overall survival. Each requires a clear operational definition and analysis method. For time-to-event outcomes, general methods account for the fact that some participants may not have experienced the event by the end of observation; the record does not state which method, censoring rules, or follow-up procedures were used. No outcome estimates or analysis details are posted for these measures.
Consequently, the available statistical interpretation is descriptive and limited. The study design and endpoint definition can be discussed, as can the adverse-event counts in their recorded categories. The record does not support reconstructing an unreported primary result, deriving uncertainty, or deciding whether an analysis was prespecified. Any further conclusion about efficacy or comparative safety would go beyond what is reported.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists the primary endpoint as overall response rate and names secondary outcomes covering safety, complete response rate, tolerability, duration of response, and overall survival. When a study evaluates multiple outcomes or repeated comparisons, general statistical practice considers multiplicity: the chance of at least one apparently notable result can rise as more tests are examined. A prespecified hierarchy or adjustment can help address that issue. The registry record does not report a multiplicity strategy, testing hierarchy, or inferential analysis.
The record lists seven outcome measures and zero analysis entries. It does not specify whether all listed outcomes were analyzed, how outcomes were selected for reporting, or whether any interim analyses occurred. Interim analyses and their decision rules are not reported in the registry record. No p-values or outcome estimates are reported in the record available for interpretation.
Missing-data methods are also not reported in the registry record. For a response endpoint, missing or unevaluable assessments can affect both the number of participants included and the resulting estimate. In general, a transparent report states the analysis population, the number with evaluable outcomes, and how missing assessments are treated. The record does not provide those details for the primary endpoint.
Likewise, the registry record does not describe analysis populations for the secondary outcomes. The listed safety risk sets differ across serious and other adverse events, but the record does not explain how participants entered those categories or how missing safety information was handled. These omissions limit assessment of outcome completeness and the possible influence of missing data.
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 |
|---|---|---|---|
| Velcade Plus Temsirolimus | 18 | 39 | 46.2% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Velcade Plus Temsirolimus | 16 of 39 |
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 |
|---|---|
| Velcade Plus Temsirolimus | 35 of 39 |
Most frequent serious adverse event terms
| Event term | Organ system | Velcade Plus Temsirolimus |
|---|---|---|
| Urinary Tract Infection | Renal and urinary disorders | 3 of 39 |
| Acute Kidney Injury | Renal and urinary disorders | 2 of 39 |
| Constipation | Gastrointestinal disorders | 2 of 39 |
| Diarrhea | Gastrointestinal disorders | 2 of 39 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 2 of 39 |
| Fatigue | General disorders | 2 of 39 |
| Fever | General disorders | 2 of 39 |
| Heart Failure | Cardiac disorders | 2 of 39 |
| Sepsis | Infections and infestations | 2 of 39 |
| Atrial fibrillation | Cardiac disorders | 1 of 39 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Velcade Plus Temsirolimus |
|---|---|---|
| Platelet count decreased | Investigations | 24 of 39 |
| Fatigue | General disorders | 20 of 39 |
| Neutrophil count decreased | Investigations | 18 of 39 |
| Nausea | Gastrointestinal disorders | 18 of 39 |
| Diarrhea | Gastrointestinal disorders | 17 of 39 |
| Anorexia | Metabolism and nutrition disorders | 16 of 39 |
| Anemia | Blood and lymphatic system disorders | 13 of 39 |
| Peripheral sensory neuropathy | Nervous system disorders | 12 of 39 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 12 of 39 |
| Vomiting | Gastrointestinal disorders | 11 of 39 |
Serious events with the highest arm-level rate: urinary tract infection (7.7%), acute kidney injury (5.1%), constipation (5.1%), diarrhea (5.1%), dyspnea (5.1%).
The registry record lists a single safety group, Velcade Plus Temsirolimus. For serious adverse events, it records 39 participants at risk and 18 affected. For other adverse events, it records 39 at risk and 35 affected. The record also lists 16 deaths. These are descriptive counts, not comparative estimates.
The serious adverse-event terms with the largest listed percentages are Urinary Tract Infection at 7.7%, and Acute Kidney Injury, Constipation, Diarrhea, Dyspnea, and Fatigue at 5.1% each. The percentages are reproduced as recorded. The registry record does not give additional event-level detail in the registry record for these terms, such as timing, duration, severity grade, or whether a participant experienced more than one event. The percentages therefore should not be read as a complete account of each participant's safety experience.
In general, an adverse event occurring during a study is not automatically evidence that the treatment caused it. A causal interpretation requires more information than a count, including clinical assessment and a suitable comparison when the aim is to estimate relative risk. No comparator group is listed. The record does not report attribution of the listed events or the deaths to treatment, so these figures should be read as recorded safety observations rather than causal conclusions.
12. What the Result Does, and Does Not, Mean
The record describes a completed phase II study of Velcade plus temsirolimus in relapsed or refractory B-cell non-Hodgkin lymphoma, with overall response rate named as the primary endpoint. It specifies how complete and partial responses are defined and provides regimen details. It also records selected safety counts and secondary outcome topics. These elements explain what the study set out to examine and what information is available in the registry record.
The primary response result is not reported in the registry record. There is no numerical overall response rate or confidence interval from which to judge the observed response frequency or its precision. The record also contains zero analysis entries, so it does not provide an inferential analysis of the primary or secondary outcomes. The completed status of the study does not itself supply a result.
The single-group design also limits what an observed outcome could establish. In general, a response in a treated group is a description of that group under the study's assessment rules; without a comparator, it is not a randomized estimate of the difference between treatments. The registry record does not list a control arm, and it warns that this is not a randomized comparison.
The adverse-event figures provide descriptive information about events recorded in the safety categories, but do not establish that treatment caused those events or deaths. The record does not report how missing primary outcome assessments were handled, how the primary analysis population was defined, or the methods used for secondary outcomes. These gaps mean the posted information does not support a full assessment of efficacy, precision, or comparative safety. The appropriate statistical reading is bounded by the outcome definitions and descriptive data recorded, without treating unreported results as either favorable or unfavorable.
13. Why This Trial Matters Statistically
This study is statistically useful to examine because its question is clear while its available results are limited. It asks about overall response to a drug combination in relapsed or refractory B-cell non-Hodgkin lymphoma and defines response using complete and partial response criteria. A reader can identify the intended endpoint and study group, but cannot evaluate the endpoint's observed value or precision because the primary result is not reported in the registry record.
The design also illustrates the distinction between describing an outcome and estimating a comparative treatment effect. A single-group phase II study can collect evidence about responses and safety in its study group. Without a comparator, however, the design does not provide a like-for-like basis for attributing differences in outcomes to treatment. Recognizing that distinction prevents a descriptive result from being interpreted as a randomized comparison.
The record also makes the importance of analysis reporting visible. A response definition alone is not enough to reproduce or fully interpret an estimate. Readers need to know the denominator, analysis population, handling of missing assessments, and method for quantifying uncertainty. Those details are not reported in the registry record, and the record lists zero analysis entries.
Finally, the safety information demonstrates why counts need context. Serious and other adverse events are recorded with different at-risk counts, and deaths are listed, but attribution and comparison are not reported. Statistically, the figures can be described as recorded observations; they cannot by themselves distinguish treatment-related events from other causes. The trial therefore matters as an example of how design, endpoint definitions, analysis transparency, and result availability jointly shape what can be concluded.
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 for interpretation is that the registry record does not report a numerical result for the primary endpoint, despite naming overall response rate as the primary outcome. No confidence interval is reported, so neither the observed response frequency nor its statistical precision can be assessed.
The single-group design has no listed comparator and is not a randomized comparison. Even if response or safety estimates were available, the design would not support a direct randomized contrast with another treatment. The record lists allocation as not applicable and masking as none.
Important details needed to interpret the response endpoint are not reported in the registry record. These include the denominator and analysis population, assessment schedule, handling of missing or unevaluable assessments, and any confirmation or review procedures. Although the record identifies RECIST v1.0 and MRI, it does not provide those additional operational details.
The registry record lists seven outcome measures but zero analysis entries. It does not report inferential methods, multiplicity handling, interim analyses, or methods for the secondary outcomes. It also does not explain the differences between the safety at-risk counts or report event attribution. These limitations restrict interpretation of the posted safety figures and prevent a comprehensive assessment of outcome completeness.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study of Velcade and Temsirolimus for Relapsed or Refractory Non-Hodgkin Lymphoma — NCT01281917.
- Registry record last updated 2019-12-13; status last verified 2017-07.
- Results first posted 2017-08-21.
- 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
The registry record describes a completed phase II, single-group study of Velcade plus temsirolimus for relapsed or refractory B-cell non-Hodgkin lymphoma. Enrollment is recorded as 40. The regimen specifies Velcade 1.6 mg/m2 weekly and temsirolimus 25mg IV weekly, with treatment planned for up to 6 cycles of 35 days each.
Overall response rate is the primary endpoint, defined as complete response plus partial response using RECIST v1.0 criteria with target lesions assessed by MRI. The primary timeframe is listed as up to 60 months. The numerical primary result and its confidence interval are not reported in the registry record.
The record lists secondary outcome topics and descriptive safety counts, including 18 affected by serious adverse events, 35 affected by other adverse events, and 16 deaths. It lists zero analysis entries. No comparator arm is listed, and the record does not report analysis populations, missing-data methods, or outcome analyses.