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 Cetuximab + Taxotere With Low Dose Fractionated Radiation for Head and Neck Carcinoma (NCT01794845), a na phase 2 study. The primary outcome is Overall Response Rate (ORR) of Participants.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01794845 |
| Phase | Phase 2 |
| Condition | Squamous Cell Carcinoma, Head and Neck Cancer, Recurrent Disease |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 5 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Miami |
| Results posted | Yes |
2. Clinical Question
Population
Participants had recurrent, unresectable locally advanced head and neck carcinoma. The listed conditions include squamous cell carcinoma, head and neck cancer, and recurrent disease. Five participants were enrolled; the primary outcome description states that data for 4 participants were analyzed because 1 participant withdrew before receiving protocol therapy.
Intervention
The study regimen combined Erbitux, Taxotere and low-dose fractionated radiation therapy. The registry record presents these interventions together as the regimen under study; it does not provide a separate outcome estimate for any one component.
Comparator
This was a single-group study, with no randomized allocation and no masking. A separate control group or comparator regimen is not reported in the registry record, so the study does not provide a like-for-like comparison between treatments.
Primary question
The primary question was whether the combined regimen was associated with complete or partial tumor response, assessed using RECIST v1.0, over the stated timeframe of up to 6 months from end of treatment, about 9 months. The registry record does not report the observed overall response rate.
The clinical question is therefore a single-group response question, not a comparative treatment-effect question. The trial aimed to examine response among people with recurrent unresectable locally advanced head and neck carcinoma receiving the combined regimen. Its brief summary frames the question as whether adding low-dose radiation to Taxotere and Erbitux improves response rate. However, the design described in the registry record has no control group, so it cannot directly estimate how response would differ without the radiation component or under another regimen.
The primary outcome definition groups complete response and partial response as qualifying responses. It does not describe a separate primary question for each response category. The record also lists treatment-related toxicity, estimated progression-free survival and estimated overall survival as secondary outcomes, but does not report their results in the registry record for this analysis. As a result, the documented question can be described, while its clinical answer cannot be quantified from the registry record.
3. Trial Design
This was a Phase II, single-group trial sponsored by the University of Miami. The registry record lists an enrollment of 5 and a status of terminated. The start date was 2013-06-03, and the primary completion date was 2016-06-07. The reason for termination is not reported in the registry record.
The intervention group is described as receiving Erbitux, Taxotere and low-dose fractionated radiation therapy. Allocation is listed as not applicable, consistent with the stated single-group model. Masking is listed as none. There is no randomized comparison, and there are no arms described as an active control or usual-care comparator. Consequently, any observed response in the group would be a within-group result rather than an estimate of the difference between two treatment strategies.
The primary outcome was overall response rate, defined as the rate of participants achieving complete response or partial response according to RECIST v1.0. The stated timeframe was up to 6 months from end of treatment, about 9 months. The registry outcome description says that 4 of 5 participants were analyzed because 1 subject withdrew before receiving protocol therapy. It does not state how the response status of the remaining participants was assessed or how missing response information was handled.
The record lists four outcome measures and no analysis entries. Beyond the endpoint definition and the population note, a statistical analysis plan, a sample-size justification, interim analyses, and detailed operational features are not reported in the registry record. The available design details establish the broad structure of the study, but do not support reconstructing additional protocol procedures.
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 | Erbitux, Taxotere, LD Fractionated RT |
|---|---|
| STARTED | 5 |
| COMPLETED | 4 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Erbitux, Taxotere, LD Fractionated RT |
|---|---|
| Withdrawal by Subject | 1 |
Analysis population for the primary outcome, as defined in the registry: Data for 4 of 5 participants analyzed due to 1 subject withdrawing prior to receiving protocol therapy.
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 | Erbitux, Taxotere, LD Fractionated RT |
|---|---|---|
| Participants analysed | 5 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 2 |
| Age, Categorical: >=65 years | Participants | 2 |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 4 |
| Region of Enrollment: United States | participants | 5 |
| Site of Head and Neck Carcinoma: Larynx | Participants | 2 |
| Site of Head and Neck Carcinoma: Floor of Mouth | Participants | 1 |
| Site of Head and Neck Carcinoma: Tonsil | Participants | 1 |
| Surgery | Participants | 1 |
| Chemotherapy | Participants | 3 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Response Rate (ORR) of Participants, measured over Up to 6 months from End of Treatment, about 9 months.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is overall response rate, expressed as the percentage of participants with a complete response or partial response. Response was defined according to RECIST v1.0, and the stated assessment period was up to 6 months from end of treatment, about 9 months. The endpoint therefore turns each participant’s tumor response assessment into a response category and summarizes the proportion meeting either of the two qualifying categories.
General: A binary response endpoint can provide a direct summary of how many participants meet a prespecified response definition within a study group. Combining complete and partial responses produces one overall response measure, rather than separate estimates for each category. Such a measure is interpretable only in relation to its assessment criteria, timeframe and analysis population; those details define what counts as a response and which participants contribute to the estimate.
The registry record does not report the observed overall response rate, the number of complete responses, or the number of partial responses. It also does not report an uncertainty interval for the response estimate. The fact that 4 of 5 participants were analyzed because 1 withdrew before protocol therapy describes the analysis population, not the response result. Without the outcome result, the endpoint’s value and its precision cannot be assessed from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a Phase II study of a combined regimen in a single group, with 5 participants enrolled. For the primary endpoint, data for 4 of 5 participants were analyzed; the outcome description attributes the exclusion of 1 participant to withdrawal before receiving protocol therapy. These figures describe enrollment and the analysis population. They do not establish how many participants achieved complete or partial response.
The registry record does not report the overall response rate, a confidence interval, a p-value, or an analysis estimate for the primary endpoint. It therefore does not provide a numerical response result to interpret. The record lists four outcome measures but no analysis entries. The absence of a reported estimate means that the response frequency and the precision of any estimate cannot be evaluated from this record.
The single-group design also limits what a response result, if reported, could establish. A proportion of responders in one group would describe outcomes observed among participants receiving the combined regimen. Without a control group, it would not directly quantify a difference relative to another regimen or isolate the contribution of low-dose fractionated radiation from the other treatments. The record’s clinical question mentions adding radiation, but the recorded design does not provide a concurrent group receiving the other agents without radiation.
Four participants contributed to the primary outcome analysis. In a small analyzed group, the response status of each participant has substantial influence on the group estimate; this is a general feature of proportions based on few observations. An uncertainty interval would help communicate the range of values compatible with the observed data, but none is reported. The overall response rate itself is also not reported, so neither its magnitude nor its precision can be described.
The secondary outcomes listed are treatment-related toxicity, estimated progression-free survival and estimated overall survival. Their results and analysis details are not reported in the registry record. The adverse-event figures are available as counts for the intervention group, but they do not supply the missing primary response outcome. Taken together, the record permits a description of the trial’s question, design and limited participant accounting, but not a quantitative conclusion about response or a comparison of treatment effects.
9. Statistical Methods Explained
The statistical interpretation is constrained by the form of the registry record. It defines a binary primary endpoint and gives the number analyzed, but it does not report an outcome estimate or analysis entry. The methods concepts below explain how such an endpoint is generally read; they do not describe undocumented procedures used by the investigators.
What does a response proportion represent?
General: For a binary outcome, each analyzed participant is classified according to whether the outcome occurred. The response proportion is the number classified as responders relative to the number included in the analysis. In this trial, a responder was defined as having complete response or partial response according to RECIST v1.0. The registry record identifies 4 analyzed participants, but does not give the response count or the resulting proportion. No rate can therefore be reconstructed from the record.
A percentage summarizes the observed group, but does not by itself show how stable that estimate may be in another group of similar participants. General: with a small number of observations, each participant’s classification can materially affect the estimated proportion. The record’s small analysis population makes it especially important to distinguish an observed estimate from the uncertainty around it. Because neither the estimate nor an interval is reported, that uncertainty cannot be quantified.
What would a confidence interval add?
General: A confidence interval is a range calculated from sample data to express uncertainty around an estimated quantity. For a response proportion, it communicates that the observed group result is not known with perfect precision and that plausible values may extend beyond the single estimate. The interval’s width depends on factors such as the amount of information in the data and the observed response proportion. No confidence interval for the primary endpoint is reported in the registry record, so the precision of the endpoint cannot be assessed.
A confidence interval would not remove the design limitation. General: an interval around a single-group response proportion describes uncertainty about that group’s response rate; it does not create a comparison group or establish what would have happened under a different regimen. A comparative treatment effect requires a suitable contrast, which is not part of the recorded single-group design.
Why does the analysis population matter?
The outcome description states that data for 4 of 5 participants were analyzed because 1 subject withdrew before receiving protocol therapy. This identifies a reason the enrolled count differs from the analyzed count for the primary endpoint. The record does not describe a broader analysis-population definition, such as whether all enrolled participants were included in any other endpoint analysis. It also does not explain whether any response data were missing among the 4 analyzed participants or how such missing information would be treated.
General: defining the analysis population matters because an endpoint estimate depends on which participants are included. Excluding a participant before treatment and excluding a participant after treatment begins can have different implications for interpretation. The recorded reason for this exclusion is withdrawal before protocol therapy; further details about timing, follow-up or the planned handling of withdrawals are not reported in the registry record.
How should the design shape interpretation?
The study is listed as single-group, with allocation not applicable and no masking. General: a single-group study can describe outcomes among participants receiving the intervention, but it does not estimate a between-group contrast. Without a concurrent comparator, changes or responses cannot be separated statistically from other possible explanations through a randomized comparison. In this record, that limitation is particularly relevant because the clinical question refers to adding low-dose radiation to two drugs, while the recorded intervention is the combined regimen.
No p-value or inferential analysis is reported for the primary endpoint. General: a p-value, when used, summarizes how compatible data are with a specified statistical model or null hypothesis; it is not a measure of clinical importance and does not replace an effect estimate or interval. Since there is no reported response estimate or analysis, no significance assessment can be made from the registry record.
What do planning illustrations mean?
General: sample-size planning explores how many participants might be needed under stated assumptions to detect a specified effect with a chosen statistical procedure. Any planning illustration based on an observed effect is hypothetical: it uses that effect as an input and does not show the probability that the completed trial found a true effect. It is not observed power and is not evidence of an outcome.
The recorded planning assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and effects specified as fractions of the observed effect. The record also states that time-to-event planning uses total events, while other planning values use participants per arm. These assumptions do not describe the actual trial design, which is single-group. The registry record does not report a trial-specific sample-size justification or the numerical planning values. Because the observed primary effect is also not reported, planning values cannot be interpreted as evidence about the trial’s response result.
General: over-dispersion refers to variability greater than a chosen model expects. It matters when a statistical model’s uncertainty calculations rely on an assumption about how variable observations are; if actual variability is greater, standard errors and intervals based on the narrower assumption can be too small. No over-dispersion assessment or model-based analysis is reported in the registry record. The primary endpoint is defined as a response percentage, but the record does not state which estimation procedure, if any, was used.
Overall, the general methods concepts clarify the distinction between an endpoint definition, an observed estimate, its uncertainty and a comparative effect. Only the endpoint definition and analysis count are available for the primary outcome. The observed response, interval, hypothesis test and detailed analysis method are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists four outcome measures: overall response rate, treatment-related toxicity, estimated progression-free survival and estimated overall survival. Overall response rate is identified as the primary outcome. The record does not report how the secondary outcomes were analyzed, or whether any adjustment for multiple outcome testing was planned or used. A multiplicity strategy is therefore not reported in the registry record.
General: when several outcomes or statistical tests are assessed, the chance of at least one result appearing unusual under a null model can depend on how many analyses are conducted and how they are related. Prespecified priorities and any planned adjustment help readers interpret a set of results. Without a reported analysis plan, it is not possible to determine how the investigators intended to handle multiple outcomes, repeated assessments or any subgroup analyses. Subgroup analyses and repeated assessments are not reported in the registry record.
The record gives a primary-outcome analysis population of 4 participants out of 5 enrolled, with 1 withdrawal before protocol therapy. It does not report a detailed rule for missing data, the status of other outcome observations, or whether participants were analyzed according to any broader assigned or treated population. The handling of missing response data beyond the stated withdrawal is not reported in the registry record.
General: missing-data handling can affect an estimate when the people without outcome information differ in relevant ways from those with observed outcomes. A transparent account usually identifies who was included, what information was unavailable and what assumptions or procedures were used. Here, the stated withdrawal reason is documented, but further missing-data methods are not. The record also does not report an interim analysis plan, a stopping rule, or the statistical analysis plan. Those details cannot be inferred from the enrollment count, the terminated status or the reported endpoint definition.
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 |
|---|---|---|---|
| Erbitux, Taxotere, LD Fractionated RT | 1 | 4 | 25.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Erbitux, Taxotere, LD Fractionated RT | 1 of 4 |
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 |
|---|---|
| Erbitux, Taxotere, LD Fractionated RT | 4 of 4 |
Most frequent serious adverse event terms
| Event term | Organ system | Erbitux, Taxotere, LD Fractionated RT |
|---|---|---|
| Hematoma | Vascular disorders | 1 of 4 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Erbitux, Taxotere, LD Fractionated RT |
|---|---|---|
| Rash acneiform | Skin and subcutaneous tissue disorders | 3 of 4 |
| Dry mouth/salivary gland (xerostomia) | Gastrointestinal disorders | 2 of 4 |
| Dysphagia | Gastrointestinal disorders | 2 of 4 |
| Anemia | Blood and lymphatic system disorders | 1 of 4 |
| Constipation | Gastrointestinal disorders | 1 of 4 |
| Dehydration | Gastrointestinal disorders | 1 of 4 |
| Dermatitis radiation | Injury, poisoning and procedural complications | 1 of 4 |
| Dry skin | Skin and subcutaneous tissue disorders | 1 of 4 |
| Dysgeusia | Nervous system disorders | 1 of 4 |
| Ear pain | Ear and labyrinth disorders | 1 of 4 |
Serious events with the highest arm-level rate: hematoma (25.0%).
The adverse-event figures are reported for the group receiving Erbitux, Taxotere and low-dose fractionated radiation therapy. The registry record lists 4 participants at risk for serious adverse events and 1 affected; it lists 4 at risk for other adverse events and 4 affected. It also reports 1 death. Hematoma is listed as a top serious adverse-event term at 25.0%.
These figures describe counts or a reported percentage within the registry’s adverse-event record. They do not establish that an event was caused by the study regimen. The registry record does not report the cause of the death or whether it was considered treatment-related. It also does not provide further event details, such as timing, duration, severity grading beyond the serious-event classification, or the number of events per participant. The listed hematoma percentage is reported as given and should not be read as a separate estimate of overall serious-event risk.
General: safety summaries depend on definitions, follow-up duration and the way events are counted. The record lists treatment-related toxicity as a secondary outcome, but the available figures do not by themselves clarify the attribution of each event. With only 4 participants at risk in the reported group, these counts describe this small group and should not be treated as a precise estimate of event frequency in a broader population.
12. What the Result Does, and Does Not, Mean
The record establishes that a Phase II, single-group study evaluated Erbitux, Taxotere and low-dose fractionated radiation therapy for recurrent unresectable locally advanced head and neck carcinoma. It defines overall response rate as the proportion achieving complete or partial response according to RECIST v1.0, with assessment up to 6 months from end of treatment, about 9 months. It also states that 4 of 5 participants were analyzed for the primary endpoint because 1 withdrew before receiving protocol therapy.
Those details describe the research question and the available participant accounting; they do not provide the response result. The overall response rate, counts of complete and partial responses, and uncertainty around the result are not reported in the registry record. Consequently, the posted data do not support a quantitative statement about the frequency of response in the analyzed group. The registry record also does not report analysis results for the listed secondary outcomes.
Even if a single-group response estimate were available, it would describe outcomes in participants who received the combined regimen. It would not, by itself, show how outcomes compare with a different regimen or identify the contribution of any one component. The recorded design has no randomized comparison or control group. The clinical question mentions whether low-dose radiation improves response when added to Taxotere and Erbitux, but the listed design cannot directly estimate that added effect.
The safety counts are likewise limited to the reported group and do not establish causal attribution. The record lists one participant affected by a serious adverse event among four at risk, four affected by other adverse events among four at risk, one death, and hematoma at 25.0% among the top serious terms. Further context, including attribution of the death, is not reported in the registry record.
In short, the record supports a description of the study’s aim, design, endpoint definition and limited safety counts. It does not provide the primary outcome result or the comparative evidence needed to determine an added treatment effect. The reported information should be interpreted as incomplete for efficacy assessment, not as a positive or negative response finding.
13. Why This Trial Matters Statistically
This trial illustrates why design and result reporting need to be read together. The clinical question concerns whether adding low-dose radiation to Taxotere and Erbitux improves response, but the recorded study is single-group. A response proportion from one group could describe that group’s observed outcome, but a comparative claim about the effect of adding radiation requires a comparison that the registry design does not include.
It also illustrates the importance of separating a defined endpoint from an endpoint result. The primary outcome is clearly described as overall response rate based on complete or partial response under RECIST v1.0. The record also states the assessment timeframe and explains why the primary analysis included 4 of 5 enrolled participants. Yet it does not report the response proportion or an analysis entry. Readers therefore have some information about what was intended to be measured and who contributed, but not the numerical finding needed to assess the endpoint.
The small analysis population makes transparent reporting of both the estimate and its uncertainty especially important. General: when few participants contribute information, each individual outcome can have a substantial effect on the group estimate. An interval would show the uncertainty around a reported estimate, while a clearly stated analysis population would help readers understand its basis. Neither a primary response estimate nor an uncertainty interval is reported in the registry record.
The record further distinguishes enrollment from analysis: five participants were enrolled, while four were analyzed for the primary outcome after one withdrew before protocol therapy. Reporting that reason is useful, but fuller descriptions of missing-data handling, the analysis plan and results for secondary outcomes are not reported in the registry record. The safety counts add descriptive information, but do not substitute for the missing response analysis or establish causation.
Statistically, the central lesson is that an endpoint definition, a study design and a reported result answer different questions. This record makes the first two reasonably clear at a broad level, while leaving the primary outcome estimate and key analytical details unreported. That distinction is essential when interpreting a trial entry without overstating what its available results establish.
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 primary limitation is that the registry record does not report the primary outcome result. Although overall response rate is defined and the analysis population is stated, the response percentage, response counts and uncertainty interval are not reported. This prevents assessment of the observed response frequency and its precision.
The single-group design limits causal and comparative interpretation. There is no randomized control group, so the record cannot directly estimate whether adding low-dose fractionated radiation changes response relative to Taxotere and Erbitux without radiation or another regimen. It also cannot separate the contribution of each intervention in the combination.
The primary outcome includes data for 4 of 5 enrolled participants. The record says the excluded participant withdrew before receiving protocol therapy, but it does not report additional details about the withdrawal or whether there were other missing observations. The broader analysis-population definitions and missing-data methods are not reported in the registry record.
The status is listed as terminated, but the reason for termination is not reported in the registry record. A statistical analysis plan, sample-size justification, interim analyses and outcome-analysis details are also not reported. The record lists four outcome measures but no analysis entries. These omissions limit appraisal of how the study planned to interpret its outcomes and whether reported analyses followed prespecified methods.
The safety information is limited to the reported counts and the listed serious-event term. The record does not provide the cause of the death, detailed event narratives or clear attribution for each event. The small number at risk means the figures describe the enrolled group, rather than supplying a precise estimate for a wider population. These limitations are specific to the information recorded for this trial and do not establish why the study was terminated or what its unreported results would have been.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Cetuximab + Taxotere With Low Dose Fractionated Radiation for Head and Neck Carcinoma — NCT01794845.
- Registry record last updated 2017-05-11; status last verified 2017-03.
- Results first posted 2017-05-11.
- 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
This University of Miami-sponsored Phase II study evaluated Erbitux, Taxotere and low-dose fractionated radiation therapy in recurrent unresectable locally advanced head and neck carcinoma. The registry record lists a single-group design, no masking, enrollment of 5, and status as terminated. The start date was 2013-06-03, and the primary completion date was 2016-06-07.
The primary endpoint was overall response rate, defined as complete or partial response according to RECIST v1.0, assessed up to 6 months from end of treatment, about 9 months. Data for 4 of 5 participants were analyzed because 1 withdrew before protocol therapy. The response result and its statistical analysis are not reported in the registry record. Listed secondary outcomes include treatment-related toxicity, estimated progression-free survival and estimated overall survival.
The adverse-event record lists 1 participant with a serious event among 4 at risk, 4 with other adverse events among 4 at risk, 1 death, and hematoma at 25.0% among top serious terms. These figures do not establish causal attribution. The single-group design does not provide a randomized comparison, and the reason for termination is not reported in the registry record.