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 Combination of Taxotere and Oxaliplatin in Squamous Cell Carcinoma of the Head and Neck (NCT00184028), a non-randomized phase 2 study. The primary outcome is Tumor Response.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00184028 |
| Phase | Phase 2 |
| Condition | Carcinoma of the Head and Neck |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 12 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Southern California |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned subjects with squamous cell cancer of the head and neck that was not solely treatable with surgery or radiation. The registry lists the condition as carcinoma of the head and neck. It does not provide further eligibility details or a description of the enrolled participants.
Intervention
Participants received a chemotherapy combination of Taxotere followed by oxaliplatin. The registry describes treatment on Day 1 of each cycle, repeated every 21 days. The combination was identified as experimental in the study summary.
Comparator
There was no separate comparator group. The trial was non-randomized, unmasked, and used a single-group model, so the posted record does not provide a concurrent control against which to compare outcomes.
Primary question
The primary outcome was tumor response, recorded as a number of participants. The study sought to assess the effectiveness of the drug combination in this population. Response categories were complete response, partial response, stable disease, progressive disease, and early death from malignant disease. Numerical results for those categories are not reported in the registry record, so the central response question cannot be answered from the posted outcome data.
3. Trial Design
This was a Phase II, non-randomized, unmasked, single-group trial sponsored by the University of Southern California. The registry reports enrollment of 12 participants and a status of terminated. The study summary had described an expected enrollment of approximately 54 subjects. The record does not state why enrollment was lower than that estimate or why the study was terminated.
The treatment was Taxotere followed by oxaliplatin. On Day 1 of each treatment cycle, Taxotere was given as a 1-hour IV infusion at 60 mg/m2, followed by oxaliplatin at 100 mg/m2, administered IV over 2 hours at a rate of 10mg/m2/min. The regimen was to be repeated every 21 days. The registry does not report how many cycles participants received or whether all enrolled participants completed treatment.
The primary outcome was tumor response, measured as the number of participants in response categories. Its timeframe was 6 months after the last subject enrolled has gone off study. A secondary outcome listed the number of participants with serious adverse events. The record lists two outcome measures, but does not report numerical tumor-response results or statistical analyses. The registry's sample-size justification, any interim analyses, and the reasoning behind the design 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: The trial had no pre-assignment criteria. All subjects were given the same treatment.
Overall Study
| Stage | Taxotere Followed by Oxaliplatin |
|---|---|
| STARTED | 12 |
| COMPLETED | 2 |
| NOT COMPLETED | 10 |
Reasons for not completing this period:
| Reason | Taxotere Followed by Oxaliplatin |
|---|---|
| Death | 1 |
| Lack of Efficacy | 4 |
| Adverse Event | 2 |
| Withdrawal by Subject | 1 |
| Non-compliance | 2 |
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 | Taxotere Followed by Oxaliplatin |
|---|---|---|
| Participants analysed | 12 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 10 |
| Age, Categorical: >=65 years | Participants | 2 |
| Sex: Female, Male: Female | Participants | 3 |
| Sex: Female, Male: Male | Participants | 9 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 2 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 10 |
| 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 | 2 |
| Race (NIH/OMB): White | Participants | 6 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 2 |
| Region of Enrollment: United States | participants | 12 |
6. Primary Endpoint
The registry lists the primary outcome as Tumor Response, measured over 6 months after the last subject enrolled has gone off study.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was tumor response, expressed as a number of participants. The registry defines five categories: complete response, partial response, stable disease, progressive disease, and early death from malignant disease. The descriptions for complete response, partial response, stable disease, and progressive disease refer to changes in target or nontarget lesions under RECIST criteria. The record does not give a separate definition for the early-death category.
In general, a response endpoint organizes observed disease status into categories that can describe tumor change during a study. Reporting participant counts by category would show how outcomes were distributed in the treated group. It would not, by itself, establish that treatment caused those outcomes, particularly in a single-group study without a concurrent comparator.
The registry states that all eligible patients who received the first dose of Taxotere would be included in the analysis. It gives the assessment timeframe as 6 months after the last subject enrolled has gone off study. However, numerical counts for the response categories are not reported in the registry record. The endpoint is therefore described, but its observed results and precision cannot be evaluated from the posted record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record establishes the design and enrollment context: this was a single-group Phase II trial of Taxotere followed by oxaliplatin, with 12 participants enrolled. It also provides some adverse-event counts. It does not report numerical results for the primary tumor-response endpoint, despite specifying response categories and an assessment timeframe. No tumor-response estimate, uncertainty interval, or inferential test is available in the registry record.
That absence limits interpretation of the study's main research question. The study summary says its purpose was to assess the effectiveness of the combination, but without the response counts, the posted record cannot show how many participants had complete response, partial response, stable disease, progressive disease, or early death from malignant disease. It is not possible to characterize the response distribution or its precision from the available outcome information.
The safety figures describe participants recorded with adverse events in the treatment group. Serious adverse events were reported for 7 of 12 participants at risk, and other adverse events were reported for 11 of 12 participants at risk. These counts indicate that events were recorded among participants, but the record does not provide event-level detail, timing, duration, severity grades, or attribution to treatment for these group totals. The listed serious-event terms include anorexia, dyspnea, leukocytes (total WBC), allergic reaction/hypersensitivity, serum-high calcium, and Death NOS, with percentages stated in the registry. The term list does not establish whether an event was caused by the study drugs.
There was no randomized comparator. Any observed outcome in this group would therefore lack a concurrent control for separating treatment effects from other influences, such as the course of disease or other care. This design can describe outcomes among enrolled participants, but it cannot support a controlled treatment contrast.
The registry marks the study as terminated and records enrollment of 12, whereas the summary had anticipated approximately 54 subjects. The reason for termination and the reason for the difference in enrollment are not reported in the registry record. These registry entries are important context, but they do not explain the missing response results or establish what outcomes would have been observed with a different enrollment.
9. Statistical Methods Explained
What does a single-group design show?
In general, a single-group study observes participants who receive a treatment without assigning a separate comparison group. It can describe what happened among those participants, provided outcomes are reported, but it does not directly compare their outcomes with what would have happened under another treatment or under no study treatment. In this trial, the registry identifies a single-group model and non-randomized allocation. There is consequently no randomized treatment contrast to interpret.
In general, random allocation helps make treatment groups comparable with respect to both measured and unmeasured characteristics. Without randomization and a control group, differences between outcomes in separate settings cannot be attributed to treatment alone. Here, there is no second group in the registry record, so no between-group effect estimate is available.
What does the response endpoint measure?
The endpoint counts participants in tumor-response categories. The registry describes the categories using RECIST criteria, including disappearance of lesions for complete response, a decrease in target-lesion measurements for partial response, neither sufficient shrinkage nor increase for stable disease, and an increase or new lesions for progressive disease. These are categorical outcomes rather than a continuous average measurement. In general, category counts describe how many participants fell into each defined status; they do not convey all the detail of individual tumor measurements.
The record specifies an analysis population consisting of eligible patients who received the first dose of Taxotere. It does not report how many participants met that definition, nor does it report numerical category counts. The stated timeframe is 6 months after the last subject enrolled has gone off study. The record does not clarify whether outcomes were assessed at additional timepoints or how assessments outside the stated timeframe would be handled.
How should precision be understood?
In general, a confidence interval expresses the range of values compatible with an estimate under a statistical model and its assumptions. A narrow interval indicates greater precision than a wide interval, while neither width nor the point estimate alone resolves limitations in study design. The registry record does not report a confidence interval or a numerical primary estimate, so precision for tumor response cannot be assessed.
In general, inferential tests evaluate data against a specified statistical hypothesis and rely on a defined analysis plan. A p-value is not a measure of clinical importance and does not show the probability that a treatment works. No inferential analyses are listed in the registry record. The statistical plan, including any prespecified test, adjustment, or interim analysis, is not reported in the registry record.
What do the planning calculations represent?
In general, sample-size calculations are planning exercises based on assumptions about the design, outcome, effect to detect, and statistical method. They are not results from enrolled participants and do not represent observed power. The posted assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect set as a stated fraction of the observed effect. For time-to-event calculations, the stated quantity is a total number of events; other calculations are expressed as participants per arm.
Those assumptions describe a hypothetical two-arm planning framework, while the actual trial is registered as single-group. They should not be read as the trial's original sample-size justification or as evidence that the study had a particular level of power. The registry's own sample-size justification is not reported in the registry record. Moreover, numerical primary response results are not reported, so the posted record does not offer a tumor-response estimate on which to base an interpretation of an observed effect.
Why does over-dispersion matter in general?
In general, over-dispersion occurs when observed variability is greater than a statistical model expects. If a model assumes less variability than the data contain, standard errors may be too small and uncertainty may be understated. Whether over-dispersion is relevant depends on the outcome and model. The registry does not report a model-based analysis of tumor response or a dispersion estimate, so over-dispersion cannot be assessed for this trial.
More broadly, a statistical method cannot compensate for missing outcome results or the lack of a control group. The central methodological reading is therefore limited: the design and endpoint are specified, but the registry does not report the primary numerical outcome or an inferential analysis.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity concerns arise when a study conducts multiple tests or makes multiple outcome-based claims. In general, testing many hypotheses without an appropriate plan can increase the chance of a false-positive finding. The registry lists a primary tumor-response outcome and a secondary outcome concerning participants with serious adverse events. It does not report an analysis plan, a multiplicity adjustment, or whether additional analyses were planned. The registry record does not report interim analyses.
The primary outcome is described as a count across response categories, but no numerical results for those categories are reported. The record does not explain whether the categories were to be analyzed separately, combined in any way, or assessed with a particular statistical test. No inferential analyses are listed. Accordingly, no conclusion about multiplicity control or the treatment of multiple response comparisons can be drawn.
The registry specifies that eligible patients who received the first dose of Taxotere would be included in the primary analysis. It does not report how many participants met this definition, whether any participants were excluded from the analysis, or how missing or unevaluable response assessments were handled. The record also does not report whether participants who stopped treatment or left the study early were included in the outcome analysis. These missing-data procedures are not reported in the registry record.
In general, missing outcome data can affect an estimate if participants without recorded outcomes differ from those with observed outcomes. The extent and direction of any such influence cannot be assessed without the numbers and handling of missing assessments. The registry does not report those details for tumor response.
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 |
|---|---|---|---|
| Taxotere Followed by Oxaliplatin | 7 | 12 | 58.3% |
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 |
|---|---|
| Taxotere Followed by Oxaliplatin | 11 of 12 |
Most frequent serious adverse event terms
| Event term | Organ system | Taxotere Followed by Oxaliplatin |
|---|---|---|
| Anorexia | Metabolism and nutrition disorders | 3 of 12 |
| Dyspnea (shortness of breath) | Respiratory, thoracic and mediastinal disorders | 2 of 12 |
| Leukocytes (total WBC) | Investigations | 2 of 12 |
| Allergic reaction/hypersensitivity (including drug fever) | Immune system disorders | 1 of 12 |
| Calcium, serum-high (hypercalcemia) | Metabolism and nutrition disorders | 1 of 12 |
| Death not associated with CTCAE term (Death NOS) | General disorders | 1 of 12 |
| Fatigue | General disorders | 1 of 12 |
| Hemoglobin | Blood and lymphatic system disorders | 1 of 12 |
| Infection (documented clinically or microbiologically) | Infections and infestations | 1 of 12 |
| Insomnia | Psychiatric disorders | 1 of 12 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Taxotere Followed by Oxaliplatin |
|---|---|---|
| Hemoglobin | Blood and lymphatic system disorders | 9 of 12 |
| Glucose, serum-high (hyperglycemia) | Metabolism and nutrition disorders | 8 of 12 |
| Insomnia | Psychiatric disorders | 6 of 12 |
| Hair loss/alopecia (scalp or body) | Skin and subcutaneous tissue disorders | 5 of 12 |
| Constipation | Gastrointestinal disorders | 4 of 12 |
| Taste alteration (dysgeusia) | Nervous system disorders | 4 of 12 |
| Dyspnea (shortness of breath) | Respiratory, thoracic and mediastinal disorders | 4 of 12 |
| Dehydration | Metabolism and nutrition disorders | 4 of 12 |
| Leukocytes (total WBC) | Investigations | 3 of 12 |
| Pain (Head/headache) | Nervous system disorders | 3 of 12 |
Serious events with the highest arm-level rate: anorexia (25.0%), dyspnea (shortness of breath) (16.7%), leukocytes (total wbc) (16.7%), allergic reaction/hypersensitivity (including drug fever) (8.3%), calcium, serum-high (hypercalcemia) (8.3%).
The registry reports 7 of 12 participants at risk with serious adverse events and 11 of 12 participants at risk with other adverse events in the Taxotere-followed-by-oxaliplatin group. These are counts of participants, not a comparison of treatment groups. Because the trial had a single group, the figures do not provide a controlled estimate of how event rates differ from another treatment or from no study treatment.
The registry lists serious-event terms with percentages: anorexia, 25.0%; dyspnea (shortness of breath), 16.7%; leukocytes (total WBC), 16.7%; allergic reaction/hypersensitivity (including drug fever), 8.3%; calcium, serum-high (hypercalcemia), 8.3%; and Death NOS, 8.3%. These listed percentages should be read as the registry's reported term figures, not as evidence that the events were caused by treatment. The record does not describe event attribution or provide further detail on timing, duration, or severity grades.
A serious adverse event is not synonymous with an event judged to be treatment-related. The registry reports no separate death count, and the appearance of Death NOS in the serious-event term list does not establish additional details about deaths. The safety figures describe recorded events in this group; they do not establish a comparative safety effect.
12. What the Result Does, and Does Not, Mean
The record describes a small, single-group Phase II study of Taxotere and oxaliplatin in squamous cell carcinoma of the head and neck. It documents a treatment schedule, a tumor-response endpoint, an intended analysis population, and participant counts for adverse events. It also records that the study was terminated after enrollment of 12 participants, while the study summary had anticipated approximately 54 subjects.
What the record does not establish is the primary tumor-response result. No numerical counts for the response categories, effect estimate, confidence interval, or statistical test are reported in the registry record. The stated goal of assessing effectiveness is therefore not answered by the posted outcome data. The absence of a reported result should not be interpreted as a favorable or unfavorable response finding.
Even if response counts were available, the single-group, non-randomized design would limit causal interpretation. Without a concurrent comparator, the study could describe outcomes among its participants but could not isolate the contribution of the drug combination from other influences. The record also does not report the reason for termination, the reason enrollment was below the anticipated figure, or details about missing response assessments.
The adverse-event figures show that serious and other adverse events were recorded among participants. They do not establish that the study drugs caused those events, and there is no comparison group to quantify a treatment-related difference. The registry does not report a separate death count.
In general, a trial record is most informative when it includes both a clear outcome definition and the observed results with uncertainty and analysis details. This record provides outcome definitions and some safety counts, but not the numerical primary result or statistical analysis needed to assess the response findings and their precision.
13. Why This Trial Matters Statistically
This trial illustrates why a stated clinical question and endpoint are not enough to interpret a study statistically. The registry defines tumor response and describes the categories, but numerical results for those categories are not reported. Without those results, readers cannot assess the response distribution or its precision, regardless of how clearly the endpoint is specified.
It also illustrates the interpretive limits of a single-group design. The registry identifies no comparator and no randomization, so the study does not provide a controlled contrast. In general, outcomes observed in one treated group can be described, but the design does not show what outcomes would have been under another treatment or in a comparable untreated group.
The enrollment context matters as well. The record reports 12 participants and says the study summary anticipated approximately 54 subjects. The study is marked terminated, but the reason for termination and the reason for the enrollment difference are not reported in the registry record. These details are relevant to assessing the record's completeness, but they do not explain the missing primary outcome data.
Finally, the safety counts and listed serious-event terms demonstrate the difference between describing events and making causal claims. The counts identify events recorded among participants, but do not show that the drugs caused them. The record contains no statistical analyses, so it also offers no inferential result for the primary endpoint. Together, these features make the trial a useful example of how design, outcome reporting, and uncertainty shape what can be concluded from a registry record.
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 direct limitation is that numerical results for the primary tumor-response outcome are not reported in the registry record. Although the response categories and timeframe are specified, the record does not give counts in those categories or an estimate of response. There is therefore no primary-result precision to evaluate.
The trial was non-randomized and used a single group without a comparator. This prevents a direct controlled comparison and limits interpretation of any observed outcomes as effects of treatment. The registry also lists no statistical analyses. The statistical plan, any interim analyses, and the trial's sample-size justification are not reported in the registry record.
Enrollment was 12, compared with approximately 54 subjects anticipated in the study summary, and the study status is terminated. The reasons for the enrollment difference and termination are not reported in the registry record. The record does not state how many enrolled participants met the primary analysis population definition, how many had evaluable response assessments, or how missing assessments were handled.
For safety, the registry provides participant counts and a list of serious-event terms, but no attribution to treatment or detailed account of timing and duration. A separate death count is not reported. These reporting gaps limit interpretation of both the response endpoint and safety experience.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Combination of Taxotere and Oxaliplatin in Squamous Cell Carcinoma of the Head and Neck — NCT00184028.
- Registry record last updated 2014-05-22; status last verified 2014-05.
- Results first posted 2013-05-06.
- 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 NCT00184028, a Phase II, non-randomized, unmasked, single-group trial sponsored by the University of Southern California. The study concerned Taxotere followed by oxaliplatin for squamous cell carcinoma of the head and neck. Enrollment was 12, and the study status is terminated. The record lists a study start of 2004-09 and primary completion of 2009-07.
The primary outcome was tumor response, recorded as the number of participants in specified categories, with a timeframe of 6 months after the last subject enrolled has gone off study. Numerical response results and statistical analyses are not reported in the registry record. The safety record reports serious adverse events for 7 of 12 participants at risk and other adverse events for 11 of 12 participants at risk. The reason for termination and the registry's sample-size justification are not reported.