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 Docetaxel, Cisplatin, and Fluorouracil Followed By Cetuximab and Radiation Therapy in Treating Patients With Locally Advanced Head and Neck Cancer (NCT00721513), a na phase 2 study. The primary outcome is Progression-free Survival.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00721513 |
| Phase | Phase 2 |
| Condition | Head and Neck Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 20 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Wake Forest University Health Sciences |
| Results posted | Yes |
2. Clinical Question
Population
Patients with locally advanced squamous cell carcinoma of the head and neck were the intended population. The registry lists head and neck cancer as the condition and records enrollment of 20 participants. It does not give further eligibility details or describe the characteristics of participants who enrolled.
Intervention
The study evaluated a sequence of treatment: docetaxel, cisplatin, and fluorouracil chemotherapy, followed by cetuximab given with radiation therapy. The registry describes the radiation approach as 3-dimensional conformal radiation therapy. The listed interventions also include computed tomography, positron emission tomography, and quality-of-life assessment procedures.
Comparator
There was no separate control group. The study is listed as single-group, with allocation marked NA and masking marked none. The results therefore describe the enrolled group’s experience; they do not provide a randomized, like-for-like comparison against another treatment or a control condition.
Primary question
The primary outcome was progression-free survival, defined in the registry as a median time-to-event measure in months, with a standard error and a timeframe of up to 2 years. In general, progression-free survival concerns the time until disease progression or death, according to the study’s outcome definition. The registry does not report the median estimate, its standard error, or other primary-outcome results, so the study’s observed progression-free survival cannot be described from this record.
In practical terms, the study was intended to characterize outcomes and toxicity after this treatment sequence in a small group of patients with locally advanced disease. The registry lists secondary outcomes that include objective and best overall response rates, overall survival, distant metastasis, local-regional control, and several quality-of-life or functional measures. Those outcomes could add context about tumor response, disease control, survival, and patient-reported or functional experience, but their results are not reported in the registry record. The record therefore identifies the clinical questions the study set out to examine without furnishing the estimates needed to answer them quantitatively.
3. Trial Design
This was a phase II study sponsored by Wake Forest University Health Sciences. The registry lists a single-group model, enrollment of 20, allocation marked NA, and no masking. The study status is completed. Its recorded start is 2008-09 and its primary completion is 2014-08. These dates describe the registry record and do not, by themselves, give the follow-up duration for individual participants.
The single group received the listed sequence of TPF chemotherapy followed by concomitant cetuximab and radiation therapy. Since there was no separate control arm, the design can describe outcomes among participants who received the regimen but cannot isolate the regimen’s effect by comparing it with a concurrent alternative. Differences from outcomes in other populations or studies would not be a randomized comparison and could reflect differences in eligibility, care, measurement, or follow-up.
The registry lists nine outcome measures, including the primary progression-free survival outcome and secondary measures covering response, survival, metastasis, local-regional control, quality of life, performance status, and swallowing-related assessment. The record does not provide the detailed protocol, eligibility criteria, treatment schedules, follow-up procedures, or analysis plan. Those details are not reported in the registry record and should not be inferred from the study title or the list of interventions.
Enrollment of 20 describes the study’s recorded size, not how many participants contributed to each outcome analysis. Outcome-specific analysis populations and the number of participants with usable data are not reported. The registry also lists zero analyses. That registry entry indicates that analyses are not represented in the record; it does not establish what analyses may have been conducted outside the registry. Without outcome estimates or analysis details, the design can be characterized, but the quantitative findings cannot be assessed.
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 | TPFChemotherapy + Concomitant Cetuximab & RT |
|---|---|
| STARTED | 20 |
| COMPLETED | 20 |
| NOT COMPLETED | 0 |
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 | TPFChemotherapy + Concomitant Cetuximab & RT |
|---|---|---|
| Participants analysed | 20 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 18 |
| Age, Categorical: >=65 years | Participants | 2 |
| Age, Continuous | years | 54.6 (SD 6.49) |
| Sex: Female, Male: Female | Participants | 4 |
| Sex: Female, Male: Male | Participants | 16 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 20 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 10 |
| Race (NIH/OMB): White | Participants | 10 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | Participants | 20 |
6. Primary Endpoint
The registry lists the primary outcome as Progression-free Survival, measured over up to 2 years.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Standard Error |
| Unit | months |
| Endpoint classification | Time-to-Event Analysis |
Progression-free survival is the primary endpoint named in the registry. In general, a time-to-event endpoint measures the time from a defined starting point until a specified event occurs. Progression-free survival is commonly used to capture the period until disease progression or death, bringing disease worsening and death into a single outcome. The precise event definition and time origin for this study are not reported in the registry record.
The registry specifies a median, in months, with standard error, and gives a timeframe of up to 2 years. A median time-to-event estimate describes the point at which the estimated event-free proportion reaches its midpoint. A standard error conveys the statistical uncertainty of an estimate; it is not the same as a confidence interval, although it can contribute to uncertainty calculations under an appropriate method. General explanation: time-to-event methods can account for participants whose event status is not known through the full follow-up period, provided their censoring information is available and handled according to the analysis plan.
The median, standard error, confidence interval, number of events, and any supporting survival curve are not reported in the registry record. The timeframe alone is not an outcome result. Without those estimates and the endpoint definition, it is not possible to describe the observed progression-free survival or its precision. The registry also does not state how progression was assessed or how incomplete follow-up was handled.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record establishes that a completed phase II, single-group study enrolled 20 participants and evaluated TPF chemotherapy followed by cetuximab and radiation therapy. It identifies progression-free survival as the primary endpoint and lists several secondary outcomes. It does not report the primary endpoint estimate or results for the secondary outcomes. Consequently, no numerical conclusion about progression-free survival, response, overall survival, disease control, or quality of life can be drawn from this registry record.
The absence of a separate control group matters for interpretation. A single-group study can describe outcomes observed among participants receiving the regimen, but it cannot directly compare those outcomes with outcomes under another treatment. Even if an outcome estimate were available, it would not by itself establish what would have happened to comparable participants under a different approach. Factors such as participant selection, disease characteristics, supportive care, and follow-up could affect outcomes; the registry does not provide enough detail to assess these factors.
The record lists an enrollment of 20, but it does not report how many participants were included in each outcome analysis, how many events occurred, or how complete follow-up was. Those details are important for understanding both the stability of an estimate and its precision. With no primary estimate, standard error, or confidence interval reported, the degree of uncertainty cannot be quantified from this record.
The safety listing records one death in the study group and gives serious adverse-event terms with percentages. The record does not state whether the death was related to treatment or provide enough context to interpret its cause. The percentages describe entries in the adverse-event listing; they do not substitute for a complete safety analysis, which would require details such as event definitions, observation period, severity, attribution, and the number of participants assessed.
Overall, the registry supplies useful design and outcome-label information but not the outcome data needed to judge the observed results. The status of completed means that the study is recorded as completed, not that results are necessarily posted. Any account of efficacy, comparative benefit, or the precision of treatment effects would go beyond what is reported in the registry record.
9. Statistical Methods Explained
The registry identifies progression-free survival as a median time-to-event outcome and names standard error as its measure of dispersion. These labels describe the intended form of the primary outcome, but the record does not report the estimate or the analysis details. The following explanations are general statistical context; they should not be read as a description of methods confirmed for this study.
What does a time-to-event endpoint represent?
Time-to-event analysis uses information about both whether an event occurred and when it occurred. For progression-free survival, the event is generally progression or death, although the exact event definition must be specified by the trial. A participant who has not had a recorded event by the last available assessment may contribute follow-up up to that point and be censored. Censoring means that the event time is not known beyond the last observed time; it does not mean that the participant remained event-free indefinitely.
Appropriate interpretation depends on the time origin, event definition, assessment schedule, and censoring rules. If follow-up differs across participants, time-to-event methods can use the available follow-up without treating everyone as if they had the same observation period. That advantage does not remove the need to understand why follow-up ended or whether censoring could be related to participants’ prognosis. The registry does not report these details for this study.
What does a median and standard error convey?
A median is a summary of the time-to-event distribution, not a statement that every participant experienced the event at that time. In general, the median is the time by which half of the estimated population has experienced the event, when the data support estimating that point. The standard error describes the sampling uncertainty of an estimate under the method used. It is not an individual-level range and should not be interpreted as a confidence interval. Confidence intervals are often more directly useful for showing a range of estimates compatible with the data and method, but no interval is reported for this primary outcome.
Precision depends on the amount and timing of event information, as well as the analysis method. Enrollment alone does not determine precision: the number of observed events and the pattern of follow-up also matter. Because the registry does not give an estimate, standard error, event count, or confidence interval, precision cannot be evaluated.
How do hazard ratios differ from medians?
In general, a hazard ratio compares event rates over follow-up between groups, under a specified model. It is a relative comparison and is not the same quantity as a difference between median survival times. Hazard-ratio interpretation also depends on assumptions about how the relative event rates behave over time. The registry identifies a single-group model and does not report a sponsor hazard ratio for the outcomes described. A comparative hazard ratio would require a comparison group and an analysis that the record does not provide.
The registry’s statistical description says that only sponsor-reported hazard ratios are used for time-to-event calculations. No such reported hazard ratio appears in this record. Therefore, no hazard-ratio result or recalculated comparison can be presented. A calculation from incomplete or absent summary statistics would not be justified.
What do planning calculations mean?
The registry’s planning assumptions describe an illustration based on an observed effect, using a two-sided significance level of 0.05, equal allocation, and a normal approximation; for time-to-event outcomes, the stated approach uses a total event count based on the Schoenfeld method. These are planning assumptions, not evidence that the study used that design or achieved a particular level of power. The study itself is recorded as single-group, so an equal-allocation, two-arm planning illustration should not be mistaken for its actual design.
In general, sample-size calculations answer a prospective planning question under specified assumptions about an effect, variability, allocation, and statistical test. They do not describe the probability that a completed study has established a result, and they do not replace an estimate with its uncertainty. When calculations are presented for fractions of an observed effect, they illustrate planning for effects of those stated sizes; they are not observed power. No numerical planning outputs are reported in the registry record.
Recalculated estimates, when available, are simple unadjusted calculations from posted summary statistics and should be distinguished from the sponsor’s own analysis. If the two disagree, the sponsor’s own analysis takes precedence. In this record, no primary outcome summary statistics or sponsor analysis are reported, so there is no basis for a recalculated effect estimate.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists nine outcome measures, with progression-free survival as the primary endpoint and several secondary outcomes. When a study evaluates multiple outcomes, general statistical practice considers whether the analysis plan addresses multiplicity: the possibility that testing many outcomes can increase the chance of at least one apparently notable result. Approaches may include prespecifying a primary outcome, ordering tests, adjusting thresholds, or treating secondary findings as exploratory. The registry does not report a multiplicity strategy, so it is not possible to determine how the study handled this issue.
Missing-data handling and analysis populations are also not described. The record does not state whether outcomes were analyzed in all enrolled participants, in a defined subset, or according to another population rule. It does not report the number contributing data to each endpoint, the extent or reasons for missing measurements, or any method for handling missing values. For time-to-event outcomes, censoring rules and follow-up completeness are especially relevant; those details are not reported in the registry record.
The registry lists zero analyses. This means the record contains no posted analysis entries for interpreting the listed outcomes. It does not establish whether analyses were undertaken elsewhere. Without an analysis population, endpoint-specific denominators, methods for missing data, and outcome estimates, readers cannot assess the robustness of results or whether the reported outcomes reflect all enrolled participants.
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 |
|---|---|---|---|
| TPFChemotherapy + Concomitant Cetuximab & RT | 20 | 20 | 100.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| TPFChemotherapy + Concomitant Cetuximab & RT | 1 of 20 |
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 |
|---|---|
| TPFChemotherapy + Concomitant Cetuximab & RT | 20 of 20 |
Most frequent serious adverse event terms
| Event term | Organ system | TPFChemotherapy + Concomitant Cetuximab & RT |
|---|---|---|
| Lymphopenia | Investigations | 17 of 20 |
| hyponatremia | Investigations | 13 of 20 |
| Low Hemoglobin | Investigations | 10 of 20 |
| Low ANC | Investigations | 9 of 20 |
| hypokalemia | Investigations | 9 of 20 |
| Low WBC | Investigations | 7 of 20 |
| Mucositis/stomatitis Oral cavity | Infections and infestations | 6 of 20 |
| Dental problems | General disorders | 5 of 20 |
| Anorexia | General disorders | 4 of 20 |
| hypophosphatemia | Investigations | 4 of 20 |
Most frequent non-serious adverse event terms
| Event term | Organ system | TPFChemotherapy + Concomitant Cetuximab & RT |
|---|---|---|
| Nausea | Gastrointestinal disorders | 18 of 20 |
| hypocalcemia | Investigations | 18 of 20 |
| hypoalbuminemia | Investigations | 18 of 20 |
| hyperglycemia | Investigations | 17 of 20 |
| Fatigue | General disorders | 16 of 20 |
| hypomagnesemia | Investigations | 16 of 20 |
| Vomiting | Gastrointestinal disorders | 15 of 20 |
| Constipation | Gastrointestinal disorders | 15 of 20 |
| low Platelets | Investigations | 14 of 20 |
| Diarrhea | Gastrointestinal disorders | 13 of 20 |
Serious events with the highest arm-level rate: lymphopenia (85.0%), hyponatremia (65.0%), low hemoglobin (50.0%), low anc (45.0%), hypokalemia (45.0%).
The adverse-event listing is associated with the single study group. It records 20 participants at risk and 20 affected for the serious-event category, and 20 at risk and 20 affected for the other-event category. These are the registry’s recorded group values; the record does not provide further detail on the observation period or how the categories were defined.
The listed serious adverse-event terms and percentages are lymphopenia, 85.0%; hyponatremia, 65.0%; low hemoglobin, 50.0%; low ANC, 45.0%; hypokalemia, 45.0%; and low WBC, 35.0%. These percentages should be read as the values recorded for the listed terms, not as a complete account of all possible harms. The registry does not provide event-level details, severity grades, duration, recurrence, or treatment attribution alongside these figures.
One death is recorded. The registry does not state its cause or whether it was considered related to treatment. A count alone cannot establish attribution. Because there is no comparison group, these safety figures also do not support a comparative estimate of risk. Interpretation would require the event definitions, assessment period, and fuller safety reporting, which are not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The registry record supports a description of the study’s design and intended questions: a completed phase II, single-group evaluation of TPF chemotherapy followed by cetuximab and radiation therapy, with enrollment of 20 and progression-free survival named as the primary outcome. It also records secondary outcomes and a set of adverse-event entries. These registry entries describe what was registered and listed; they do not provide the numerical primary or secondary outcome results.
Because the primary progression-free survival estimate and its uncertainty are not reported, the record cannot show the observed median or how precisely it was estimated. The same limitation applies to objective response, overall survival, metastasis, local-regional control, quality of life, and the other listed secondary measures. The record’s completion status does not fill this gap: completion indicates the study is recorded as completed, not that outcome results are present.
The single-group design also limits what could be concluded even if outcome estimates were available. Observed outcomes in the treated participants would not establish how outcomes compare with a different regimen or with no treatment, because no concurrent comparator is listed. A treatment effect relative to an alternative requires a valid comparison; this record does not contain one.
The adverse-event entries give some safety information, including the listed serious-event percentages and one death, but the record does not establish the death’s cause or treatment relationship. The entries also lack enough detail to characterize the full safety profile or compare risks. Taken together, the record identifies the population, treatment sequence, outcome framework, and some safety listings, while leaving the results and their uncertainty unreported. Conclusions about efficacy, comparative effects, or the clinical importance of outcomes would therefore exceed the evidence recorded.
13. Why This Trial Matters Statistically
This study is statistically informative as an example of the distinction between a registered research question and a reported result. The record clearly identifies a phase II, single-group design, a treatment sequence, and progression-free survival as the primary endpoint. Yet it does not include the primary estimate or results for the listed secondary outcomes. A reader can understand what was intended to be evaluated without being able to quantify what was observed.
The single-group design is another important statistical feature. It can describe outcomes in participants receiving the treatment sequence, but it does not supply a concurrent estimate of outcomes under an alternative. This distinction matters when interpreting any outcome as evidence of a treatment effect. Descriptive outcome data and comparative evidence answer different questions.
The record also illustrates why enrollment alone is not enough to judge the strength of a time-to-event result. For interpretation, readers need the event definition, follow-up information, event counts, analysis population, estimate, and uncertainty. Those details are not reported. A median without a measure of precision would be incomplete; in this record, even the median is absent.
Finally, the safety listing demonstrates the need to distinguish event counts or percentages from causal conclusions. The recorded adverse-event terms and death are relevant descriptions, but the record does not give attribution or enough context to estimate comparative risk. Statistical interpretation therefore depends not only on the design and endpoint labels, but also on complete reporting of methods, denominators, and results. This registry record supplies some of that framework, but not the outcome evidence needed for quantitative conclusions.
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 interpreting this study from the registry record is that no primary or secondary outcome estimates are reported. The primary endpoint is named, but its median, standard error, confidence interval, and event count are absent. Results for the listed response, survival, metastasis, local-regional control, and quality-of-life outcomes are also not reported in the registry record.
The design is single-group, with no separate comparator. This means the record cannot support a direct randomized comparison with another treatment. Enrollment is recorded as 20, but endpoint-specific numbers analyzed and follow-up completeness are not given. The registry also does not describe eligibility criteria, participant characteristics, the event definition, time origin, assessment schedule, or censoring approach.
Analysis details are limited. The record lists zero analyses and does not report analysis populations, missing-data procedures, multiplicity handling, or a statistical analysis plan. It is therefore not possible to evaluate whether the outcome analyses followed prespecified methods or how incomplete observations were addressed. The planning assumptions described in the record should not be treated as the trial’s actual design or as observed power.
Safety interpretation is constrained by the lack of event definitions, observation period, severity and attribution details, and context for the recorded death. The adverse-event percentages alone do not establish treatment causation or comparative risk. These limitations are specific to the information available in the registry record; additional details are not reported there and should not be inferred.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Docetaxel, Cisplatin, and Fluorouracil Followed By Cetuximab and Radiation Therapy in Treating Patients With Locally Advanced Head and Neck Cancer — NCT00721513.
- Registry record last updated 2020-04-07; status last verified 2020-03.
- Results first posted 2019-07-12.
- 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
NCT00721513 is recorded as a completed phase II, single-group study sponsored by Wake Forest University Health Sciences, with enrollment of 20. The study evaluated docetaxel, cisplatin, and fluorouracil followed by cetuximab and radiation therapy in patients with locally advanced head and neck cancer. The registry names progression-free survival as the primary endpoint, with a median time-to-event measure in months and a timeframe of up to 2 years.
Nine outcome measures are listed, including response, survival, disease-control, and quality-of-life measures. The registry does not report outcome estimates or analyses. Its safety listing includes serious adverse-event terms and one death, without reported attribution for the death. The single-group design has no separate comparator, and the available record does not provide the details needed to assess outcome precision, missing data, or analysis populations.