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 Docetaxel in Treating Patients With Recurrent or Metastatic Head and Neck Cancer (NCT00425750), a non-randomized phase 2 study. The primary outcome is Patient Response to Treatment.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00425750 |
| Phase | Phase 2 |
| Condition | Head and Neck Cancer |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 25 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Vanderbilt-Ingram Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
The trial concerned patients with recurrent and/or metastatic head and neck squamous cell carcinoma. The registry record lists head and neck cancer as the condition and states the more specific disease in the official trial title. It records 25 enrolled participants. Further eligibility details and characteristics of the enrolled participants are not reported in the registry record, so the population represented by the enrollment figure cannot be described more narrowly.
Intervention
The intervention was a combination of bortezomib and docetaxel. The arm description specifies docetaxel at 40 mg/m2 by intravenous infusion over 30 minutes on days 1 and 8 of a 21-day cycle, with the first dose held on day 1 of cycle 1. Bortezomib was specified at 1.6 mg/m2 as an intravenous push on days 1 and 8 of a 21-day cycle; it was given as a single agent on day 1 of cycle 1. Laboratory biomarker analysis and a pharmacological study are also listed as interventions, but their role in the primary response result is not detailed.
Comparator
There was no separate comparator arm: the trial is identified as single-group and non-randomized. The registry record does not describe a concurrent control group or a randomized comparison. Consequently, the posted results cannot establish how outcomes under the combination compare with outcomes under another treatment or with outcomes in an untreated group.
Primary question
The primary outcome was patient response to treatment, classified using changes in the sum of the longest diameters of target lesions. The categories were complete response, partial response, stable disease, and progressive disease. The registry record gives the category definitions and an average on-study-to-off-study duration of 7.55 months, but does not report how many participants had each response category. The central question is therefore the response outcome the trial set out to assess, not a numerical estimate of response that can be recovered from the registry entry.
3. Trial Design
This was a phase II, non-randomized, single-group trial of bortezomib combined with docetaxel in recurrent and/or metastatic head and neck squamous cell carcinoma. The registry record lists 25 enrolled participants and describes the study as completed. Its recorded start is 2005-08, and its primary completion is 2009-06. These dates describe the registry timeline; they do not supply details about when individual participants entered or completed treatment.
The treatment arm description gives a dosing schedule for both drugs. Docetaxel was specified as 40 mg/m2 by intravenous infusion over 30 minutes on days 1 and 8 of a 21-day cycle, except that the first dose was held on day 1 of cycle 1. Bortezomib was specified as 1.6 mg/m2 by intravenous push on days 1 and 8 of a 21-day cycle, with bortezomib alone on day 1 of cycle 1. The registry also lists laboratory biomarker analysis and a pharmacological study. It does not explain how those activities relate to the primary outcome analysis.
Masking is listed as none. With no random allocation and no separate control arm, this design describes outcomes among participants receiving the study regimen rather than estimating a randomized treatment contrast. The registry lists overall survival and progression-free survival as secondary outcomes, but does not report their results. The primary response outcome is also described without its observed category counts. The sample-size justification, detailed statistical plan, and any interim analyses 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: Twenty-seven patients consented, two of which were ineligible.
Overall Study
| Stage | Bortezomib; Docetaxel |
|---|---|
| STARTED | 25 |
| COMPLETED | 0 |
| NOT COMPLETED | 25 |
Reasons for not completing this period:
| Reason | Bortezomib; Docetaxel |
|---|---|
| Death | 1 |
| Withdrawal by Subject | 3 |
| Adverse Event | 3 |
| Progression of disease | 18 |
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Bortezomib; Docetaxel |
|---|---|---|
| Participants analysed | 25 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 21 |
| Age, Categorical: >=65 years | Participants | 4 |
| Age Continuous | years | 55 (SD 1) |
| Sex: Female, Male: Female | Participants | 7 |
| Sex: Female, Male: Male | Participants | 18 |
| Region of Enrollment: United States | participants | 25 |
6. Primary Endpoint
The registry lists the primary outcome as Patient Response to Treatment, measured over 7.55 months (average duration, on study to off study).
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is described as patient response to treatment, measured in participants and classified using target-lesion diameter changes. Complete response means disappearance of all target lesions. Partial response is defined as a decrease of at least 30% in the sum of the longest diameters from baseline. Progressive disease is defined as an increase of at least 20% from the smallest sum recorded since treatment began. Stable disease is the category for changes meeting neither the partial-response nor progressive-disease definition.
These categories distinguish tumor-size patterns using stated thresholds and reference points. The partial-response comparison uses baseline, while the progressive-disease comparison uses the smallest recorded sum after treatment started. Stable disease is defined by exclusion from the other specified change categories. The endpoint is useful for describing whether participants met the registry’s response classifications during the study; it is not, by itself, a randomized comparison or a complete account of survival.
The registry gives an average duration of 7.55 months from on study to off study as the timeframe for the primary outcome. It does not report the observed number of participants in each response category, the assessment schedule, or a response estimate with a confidence interval. As a result, the endpoint definition can be explained, but the primary response outcome itself cannot be quantified from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a clinically focused phase II study of a two-drug regimen in recurrent and/or metastatic head and neck squamous cell carcinoma. It establishes that 25 participants were enrolled and describes the response categories used as the primary outcome. It does not report the number of participants with complete response, partial response, stable disease, or progressive disease. Without those outcome counts, the primary response result and its precision cannot be assessed from this record.
The design is single-group and non-randomized, with no separate control arm. Accordingly, even a complete descriptive response result would characterize outcomes observed in the treated group; it would not, by itself, isolate the effect of the regimen from other explanations for outcomes. The registry record does not present a randomized comparison or a like-for-like control group. No causal treatment contrast should be inferred from the listed design.
Overall survival and progression-free survival are named as secondary outcomes. Their definitions, results, and uncertainty estimates are not reported in the registry record. The 7.55 months listed for the primary outcome is an average on-study-to-off-study duration, not a reported response rate or a survival result. It should not be interpreted as the duration of response or as an estimate of time to progression.
In general, a confidence interval describes the range of estimates compatible with the data and the stated statistical method; it helps readers judge precision as well as the central estimate. No confidence interval for response is reported in the registry record, and the response count needed to evaluate one is absent. The absence of a reported estimate does not establish that no responses occurred; it means the registry entry does not provide the numerical result needed to describe them.
The safety summary records serious adverse events among 11 of the 25 participants at risk, and records zero affected participants in the other adverse-event category. Those summaries are separate from the efficacy endpoint and do not show whether a serious event was caused by treatment. The listed serious-event terms include Death and Vomitting, among others, but the registry does not give event-level attribution or enough detail to interpret individual cases. Overall, the posted information supports a description of the study design, endpoint rules, and reported safety counts, but not a numerical conclusion about treatment response or comparative benefit.
9. Statistical Methods Explained
The design determines what statistical claims are available. This trial is listed as single-group, non-randomized, and unmasked. It therefore has no randomized contrast between the combination and another arm. A descriptive analysis could report the frequency of each response category among participants assessed, but a comparison of treatment strategies would require a suitable comparator and a defined analysis. Neither is described in the registry record. The actual response counts are also not reported, so no descriptive response estimate can be reconstructed from this entry.
What does a response category represent?
The primary outcome is categorical: participants are classified as having complete response, partial response, stable disease, or progressive disease using the stated lesion-size rules. The rules do not assign a numerical response result to the group on their own. To summarize the outcome, the record would need to state how many participants met each category and what population was assessed. Those details are not reported in the registry record. In general, categorical outcomes are commonly described with counts and proportions, accompanied by uncertainty when appropriate; no such primary outcome summary is present.
How should uncertainty be considered?
In general, an estimate from a clinical study is subject to sampling variability. A confidence interval conveys uncertainty around an estimate under a specified statistical method and assumptions. A narrower interval generally indicates greater precision, while a wider interval indicates less precision. The record does not report a response estimate, confidence interval, or the method used to calculate uncertainty for the primary outcome. Therefore, the precision of the response result cannot be evaluated.
The number enrolled is not a substitute for the number included in an endpoint analysis. Participants may have different amounts of outcome information, and the record does not say how many were evaluable for response or how missing assessments were treated. The reported average on-study-to-off-study duration does not resolve those questions. It describes a timeframe, not the analysis denominator, follow-up distribution, or response classification.
What can a single-group study estimate?
In general, a single-group study can describe outcomes observed among its participants under the study regimen. Without a concurrent randomized comparator, it cannot separate treatment effects from other influences on outcome, such as differences in participant characteristics, disease course, or care received. Those are general design considerations, not findings about this trial’s participants. The registry record does not report an adjusted analysis or a statistical method intended to address such comparisons.
The trial is unmasked. In general, lack of masking can matter when outcome assessment or decisions depend on judgment, although the response rules are defined using lesion measurements. The registry record does not explain how measurements were assessed, whether reviewers were independent, or whether any safeguards were used. It is therefore not possible to characterize the degree to which assessment procedures could have influenced classification.
What do the planning assumptions mean?
The record describes a planning illustration based on the observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It states that the effect to detect is a stated fraction of the observed effect. Such an illustration is a hypothetical sample-size calculation, not a description of the trial’s actual design: this trial is listed as single-group and non-randomized. The values of the planning illustrations are not reported in the registry record. They should not be read as observed power or as evidence that the trial had a particular probability of detecting an effect.
In general, over-dispersion occurs when variation in data exceeds what a statistical model assumes. Accounting for it can matter in outcomes such as counts, because uncertainty may otherwise be understated. The primary outcome is a response classification, not a reported count outcome, and the registry does not state that an over-dispersed model was used. No model specification, significance testing procedure, or confidence-interval procedure for the primary outcome is reported.
The registry lists overall survival and progression-free survival as secondary outcomes. These are time-to-event outcomes in general, and their analysis may depend on follow-up and censoring rules. The record does not report their operational definitions, censoring approach, estimates, or statistical methods. Consequently, no time-to-event interpretation can be made from the outcome names alone.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists patient response to treatment as the primary outcome and overall survival and progression-free survival as secondary outcomes. It does not report a testing hierarchy, adjustment for multiple comparisons, or a plan for interpreting several outcomes together. When multiple outcomes are examined, multiplicity can affect the chance of a false-positive finding across the set of tests; in general, a prespecified approach helps readers understand how results were evaluated. Whether any such approach was used in this trial is not reported in the registry record.
Missing-data handling is also not reported. The entry does not state how participants without an evaluable response assessment were classified, whether incomplete follow-up affected the endpoint denominator, or how missing information was treated for the secondary outcomes. The enrollment figure alone does not establish the number included in each analysis. Analysis populations, including any intention-to-treat or evaluable-participant population, are not reported in the registry record.
The registry gives response definitions and an average on-study-to-off-study timeframe, but does not provide the response counts, secondary outcome results, or analysis methods. It also does not report an interim-analysis plan or a statistical-analysis plan. These omissions limit assessment of outcome selection, the handling of incomplete observations, and the degree to which analyses followed prespecified procedures.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Bortezomib; Docetaxel | 11 | 25 | 44.0% |
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 |
|---|---|
| Bortezomib; Docetaxel | 0 of 25 |
Most frequent serious adverse event terms
| Event term | Organ system | Bortezomib; Docetaxel |
|---|---|---|
| Death | General disorders | 2 of 25 |
| Vomitting | Gastrointestinal disorders | 2 of 25 |
| Hypercalcemia | Metabolism and nutrition disorders | 1 of 25 |
| Seizure | Nervous system disorders | 1 of 25 |
| Diarrhea | Gastrointestinal disorders | 1 of 25 |
| Dry mouth | Gastrointestinal disorders | 1 of 25 |
| Mucositis | Gastrointestinal disorders | 1 of 25 |
| Nausea | Gastrointestinal disorders | 1 of 25 |
| Hemorrhage, GI | Gastrointestinal disorders | 1 of 25 |
Serious events with the highest arm-level rate: death (8.0%), vomitting (8.0%), hypercalcemia (4.0%), seizure (4.0%), diarrhea (4.0%).
The registry safety summary lists 25 participants at risk for serious adverse events and 11 affected participants. It lists 25 participants at risk in the other adverse-event category and zero affected participants. These are participant-level summary counts as recorded; they do not establish whether an event was caused by bortezomib, docetaxel, the combination, or another factor. The registry record does not report attribution or event-level clinical detail.
The listed serious adverse-event terms and percentages are Death, 8.0%; Vomitting, 8.0%; Hypercalcemia, 4.0%; Seizure, 4.0%; Diarrhea, 4.0%; and Dry mouth, 4.0%. The record does not provide participant counts alongside each term or explain the denominator used for these percentages. The terms and percentages should not be added to infer a number of unique participants, since one participant may have more than one event. The separate deaths field is not reported, so the term Death should not be used to infer an overall death count or a treatment-related death count.
In general, a serious adverse event is a safety classification and is not synonymous with a side effect proven to be caused by treatment. These entries describe what the registry records, not comparative safety: there is no control arm for a like-for-like comparison, and details such as event timing, severity grades, and attribution are not reported.
12. What the Result Does, and Does Not, Mean
The trial record establishes that a phase II, single-group study of bortezomib and docetaxel enrolled 25 participants with recurrent and/or metastatic head and neck squamous cell carcinoma. It defines the primary outcome using complete response, partial response, stable disease, and progressive disease categories, and it gives the treatment schedule and a summary of serious adverse events. Those details describe the study and the information recorded about it.
The registry record does not report the number of participants in any primary response category. It therefore does not permit a numerical estimate of response or an assessment of how precise such an estimate might be. The secondary outcomes, overall survival and progression-free survival, are named but their results are not reported. The recorded average duration of 7.55 months is the average time from on study to off study, not a response estimate or a survival result.
Even if response counts were available, the single-group non-randomized design would support a description of observed outcomes rather than a randomized comparison against another treatment. No like-for-like control group is described. The record also does not report an analysis plan, missing-data methods, or an analysis population, all of which would be relevant when evaluating the reliability and applicability of outcome estimates.
The safety counts and serious-event terms provide a registry summary, but do not establish causation or comparative risk. The listed term Death is not accompanied by a reported death count in the separate deaths field or an attribution statement. Thus, the entry supports careful reporting of its recorded design and safety information, while leaving the principal efficacy result and important analytical details unavailable in the registry record.
13. Why This Trial Matters Statistically
This study illustrates why a trial’s design and its reported results need to be read together. A phase II label indicates the study’s stated phase, but it does not by itself tell readers the magnitude or precision of a treatment outcome. Here, the record provides detailed response-category definitions but does not report the category counts. The endpoint rules alone cannot show how participants were classified.
The single-group, non-randomized structure is equally important. It means the study is described as observing outcomes under a regimen, not as comparing randomized treatment groups. A response summary, if reported, would still need to be interpreted in the context of that design. The registry record does not provide a comparator that could support a direct treatment contrast.
The entry also shows the distinction between a planning calculation and observed evidence. Its stated planning assumptions describe a hypothetical sample-size illustration, including equal allocation between two arms, even though this trial is identified as single-group. Such calculations are not observed power and do not replace reported outcomes. The numeric planning illustrations are not reported in the registry record.
Finally, endpoint counts, analysis populations, missing-data methods, and uncertainty estimates all matter for evaluating a result. Their absence limits what can be concluded even when the design and outcome definitions are available. The record is informative about the trial’s structure and stated safety summary, but its main response result cannot be evaluated from the registry entry.
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 trial-specific limitation in the registry record is the absence of primary response counts. Although the response categories and lesion-size thresholds are defined, the record does not state how many participants met each category or provide an overall response estimate. It also does not report a confidence interval or the method used to calculate uncertainty. The outcome’s precision therefore cannot be assessed.
The study is listed as single-group and non-randomized, with no separate comparator arm. Findings from such a design would not provide a randomized estimate of the difference between the combination and another treatment. In addition, the record does not report participant characteristics or detailed eligibility criteria, limiting description of who was represented beyond recurrent and/or metastatic head and neck squamous cell carcinoma.
Overall survival and progression-free survival are named as secondary outcomes, but their definitions and results are not reported. The record also does not state the response analysis population, how missing assessments were handled, or whether outcome assessors followed any particular assessment procedure. The average on-study-to-off-study duration does not answer those questions.
Safety reporting is limited to the recorded participant counts and serious-event terms. The entry does not report event attribution, timing, or detailed severity information, and its separate deaths field is not reported. Finally, the statistical plan, multiplicity approach, sample-size justification, and interim-analysis details are not reported in the registry record. These gaps restrict interpretation of both efficacy and safety summaries.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Bortezomib and Docetaxel in Treating Patients With Recurrent or Metastatic Head and Neck Cancer — NCT00425750.
- Registry record last updated 2011-11-16; status last verified 2011-11.
- Results first posted 2011-04-04.
- 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
NCT00425750 is a completed phase II trial of bortezomib and docetaxel in recurrent and/or metastatic head and neck squamous cell carcinoma. It is listed as non-randomized, unmasked, and single-group, with 25 participants enrolled. The registry records a start of 2005-08 and primary completion in 2009-06.
The primary outcome was patient response to treatment, classified by target-lesion changes as complete response, partial response, stable disease, or progressive disease. The registry gives an average on-study-to-off-study duration of 7.55 months, but does not report the primary response counts or results. Overall survival and progression-free survival are listed as secondary outcomes without reported results.
The safety summary records serious adverse events in 11 participants and zero affected participants in the other adverse-event category. It lists several serious-event terms with percentages. The registry record does not report a comparator, response analysis methods, missing-data handling, or event attribution.