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 Erlotinib (Tarceva) During First Line Standard Platinum Containing Chemo for Advanced Squamous Cell Head and Neck Cancer (NCT00448240), a na phase 2, phase 3 study. The primary outcome is Tumor Response Rate of Intermittent Tarceva During First Line Standard Platinum Containing Chemotherapy.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00448240 |
| Phase | Phase 2, Phase 3 |
| Condition | Head and Neck Cancer, Carcinoma, Squamous Cell |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 6 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Henry Ford Health System |
| Results posted | Yes |
2. Clinical Question
Population
The study concerns people with advanced squamous cell carcinoma of the head and neck. The brief summary describes the intended population as having metastatic, recurrent, or persistent disease. Further eligibility details, including clinical characteristics of enrolled participants, are not reported in the registry record.
Intervention
The intervention is intermittent erlotinib, also called Tarceva, during first-line standard platinum-containing chemotherapy. The brief summary names erlotinib, cisplatin or carboplatin, and paclitaxel as the combination being evaluated. The intervention listing itself identifies erlotinib; details about dosing and the chemotherapy actually received are not reported in the registry record.
Comparator
No separate comparator group is identified. The study is classified as single-group, with allocation listed as not applicable. It therefore does not make a randomized, like-for-like comparison between treatment groups.
Primary question
The primary question is how many participants have a tumor response to intermittent Tarceva during first-line standard platinum-containing chemotherapy. Response was to be assessed with radiologic testing using RECIST 1.0. The registry record does not report the observed response count, so the result of that question cannot be determined from the posted record.
3. Trial Design
NCT00448240 is registered as a phase 2 and phase 3 study sponsored by Henry Ford Health System. It is classified as a single-group study, and allocation is listed as not applicable. Masking is listed as none. These entries describe a study without a blinded assignment or a separate randomized control group; they do not establish further details about how participants were recruited, treated, or followed.
The registry record lists an enrollment of 6 and a status of terminated. The recorded start is 2007-02, and primary completion is listed as 2012-01. The reason for termination, the timing of the decision, and whether the planned enrollment was reached are not reported in the registry record. The enrollment figure describes the record's enrollment, not necessarily the number with evaluable tumor imaging or complete endpoint data.
The stated study purpose is to determine whether combination erlotinib, cisplatin or carboplatin, and paclitaxel are effective as first-line treatment for metastatic, recurrent, and persistent squamous cell carcinoma of the head and neck. The title describes intermittent erlotinib during standard platinum-containing chemotherapy. The intervention listing names erlotinib, but detailed treatment schedules, chemotherapy choices, dose modifications, and treatment exposure are not reported in the registry record.
The primary outcome is tumor response rate, expressed as a count of participants. It was to be evaluated at 6-week intervals, up to an average of 18 weeks, using radiologic testing and RECIST 1.0. The record lists one outcome measure and zero analyses. It does not report an analysis plan, endpoint results, or a reason for the absence of posted analyses. Thus, the design and stated question can be described, but the study's observed response cannot be reconstructed from the registry information.
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 | Erlotinib |
|---|---|
| STARTED | 6 |
| COMPLETED | 6 |
| 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 | Erlotinib |
|---|---|---|
| Participants analysed | 6 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 5 |
| Age, Categorical: >=65 years | Participants | 1 |
| Sex: Female, Male: Female | Participants | 3 |
| Sex: Female, Male: Male | Participants | 3 |
6. Primary Endpoint
The registry lists the primary outcome as Tumor Response Rate of Intermittent Tarceva During First Line Standard Platinum Containing Chemotherapy, measured over Evaluated at 6-week intervals, up to an average of 18 weeks..
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the tumor response rate for intermittent Tarceva during first-line standard platinum-containing chemotherapy. It is described as a count of participants, with response assessed by radiologic testing under RECIST 1.0. The planned assessment schedule is at 6-week intervals, up to an average of 18 weeks. The record does not give the response count or explain how participants without evaluable imaging were handled.
As a general statistical concept, a tumor-response endpoint converts imaging findings into categories defined by a response-assessment framework. A count of participants with a response is a binary outcome at the participant level: each participant is classified according to whether the defined response occurred. A rate would ordinarily relate that count to an appropriate set of participants, but the registry record does not report the endpoint numerator, its denominator, or a percentage estimate.
Response is used as an outcome because it records whether tumors meet specified imaging criteria during the assessment period. It is not, by itself, a complete description of how long a response lasts or of other clinical outcomes. Those additional outcomes are not identified as reported results in this record. Without the observed count and the analysis details, readers cannot judge the magnitude or precision of the primary endpoint result. The registered outcome definition tells readers what was intended to be measured, not what the study found.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes that the study enrolled 6 participants and was terminated. It identifies tumor response as the primary outcome, but it does not report the response count, a response rate, an uncertainty interval, or an analysis result. The central efficacy question therefore remains unanswered by the posted registry results. The absence of an endpoint result is not evidence that there were no responses, and it is not evidence that every participant responded.
The single-group design affects what could be inferred even if a response count were available. Without a separate comparator group, the record cannot support a randomized comparison of the regimen with another treatment. An observed response in a single group would describe outcomes among participants receiving the study regimen; it would not, on its own, isolate the contribution of erlotinib from the accompanying chemotherapy or from other influences on observed outcomes. This is a general limitation of uncontrolled treatment-group data.
The enrollment of 6 also means that any estimate based on the enrolled group would be based on a small number of participants. In general, estimates from small groups can be imprecise, and a small change in the participant count can materially affect a count-based result. Here, no response estimate or confidence interval is reported, so its precision cannot be assessed. The record also does not state how many participants had evaluable imaging or whether endpoint data were missing.
The record's status is terminated, but it does not explain why the study ended or whether termination affected endpoint collection. The dates listed are 2007-02 for start and 2012-01 for primary completion; those entries do not supply the missing outcome data or clarify the study's operational history. The listed phase entries are phase 2 and phase 3, but the registry record does not explain the relationship between those designations or report phase-specific analyses.
The safety information is separate from the missing tumor-response result. It reports zero serious adverse events among 6 at risk, four participants with other adverse events among 6 at risk, and zero deaths. These counts describe the posted safety fields; they do not provide the type, timing, severity detail, or causal attribution of the other adverse events. Taken together, the registry information supports a description of the planned question, design, enrollment, and recorded safety counts, but not a conclusion about tumor response or comparative treatment effect.
9. Statistical Methods Explained
The registry describes a binary primary outcome recorded as a count of participants. In general, a binary outcome classifies each participant according to whether a defined event occurred. For tumor response, the event definition is tied to radiologic assessment using RECIST 1.0. A count is the number classified as having the event; a rate or proportion additionally depends on a defined denominator. Although the endpoint is called a response rate, the posted record does not give the response count, denominator, or calculated rate.
What does the assessment schedule mean?
The endpoint description says response was evaluated at 6-week intervals, up to an average of 18 weeks. In general, repeated assessments allow response status to be considered across a treatment period rather than at a single imaging visit. The registry does not specify how the response endpoint combined findings across visits, which visit determined a participant's final classification, or how missing or unevaluable scans were handled. The phrase average of 18 weeks is part of the stated timeframe; further details about individual follow-up are not reported in the registry record.
How should a confidence interval be understood?
In general, a confidence interval expresses the statistical uncertainty around an estimate under a specified method and its assumptions. A wider interval indicates less precision than a narrower interval. For a count-based response outcome, an interval around a proportion would help readers see how uncertain the estimate is, particularly when participant numbers are limited. No confidence interval or response estimate is reported in the registry record, so precision cannot be judged from the posted endpoint data. This general explanation does not imply that a particular interval was calculated for this study.
Why does the design matter?
The study is classified as single-group, with allocation listed as not applicable and masking listed as none. In general, an uncontrolled study can describe outcomes observed in the treated group, but it cannot create the same direct treatment contrast as a randomized controlled comparison. If a tumor response were reported, it would be an outcome in the enrolled group; without a comparator, it would not quantify the difference attributable to adding erlotinib. The registry record lists no like-for-like control arm, and the study-design description explicitly excludes a randomized comparison.
What do the planning illustrations represent?
The planning description says the sample-size illustrations use an observed effect as the effect to detect, with two-sided significance level 0.05, equal allocation between two arms, and a normal approximation. These are planning assumptions, not a report of the study's actual design or of achieved statistical power. The illustrations are not observed power. In general, a planning calculation estimates how many participants might be needed under stated assumptions; it does not show that a study has detected an effect or that its result is precise. The registry record describes this illustration as a two-arm calculation, whereas the trial itself is classified as single-group. It should therefore not be read as evidence that the trial had two randomized arms.
The posted record does not report an endpoint estimate from which a reader could evaluate the effect used in those illustrations. Consequently, the illustrated planning scenarios cannot substitute for missing tumor-response results. They also cannot establish that the enrollment of 6 was adequate, because the registry record does not report a study-specific sample-size justification or enough endpoint information to assess the assumptions.
What can be inferred from the analysis listing?
The registry lists zero analyses. This indicates that no analysis results are listed in that record; it does not identify which statistical procedures were planned or whether analyses were conducted outside the registry. The statistical analysis plan, including any model, adjustment strategy, handling of repeated assessments, and rules for missing data, is not reported in the registry record. No recalculated estimate is available from the posted primary outcome information. In general, a recalculation from summary data would describe a simple unadjusted result and would not replace a sponsor's own analysis when a discrepancy is identified. The record does not present a sponsor analysis or a discrepancy to evaluate.
Over-dispersion is a general issue in count-data models when variability exceeds what a chosen model assumes. It matters because using a model that understates variability can make uncertainty appear too small. The primary endpoint is recorded as a participant count, but the registry does not identify a count-data model or report dispersion. There is no basis in the record for claiming that over-dispersion was assessed or that a particular model was used. More broadly, statistical labels and planning calculations cannot fill the gap left by unreported endpoint data and an unreported analysis plan.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists one primary outcome measure, tumor response rate, and zero analyses. It does not report whether additional outcomes were prespecified in the protocol, whether multiple time points or response definitions were analyzed, or whether any multiplicity adjustment was planned. Therefore, the record does not establish whether multiplicity was a practical issue in the full study analysis or how it would have been addressed. In general, when several outcomes or repeated opportunities to test a hypothesis are considered, the chance of a false-positive finding can increase unless the analysis plan accounts for that multiplicity. This is a general statistical concern, not a claim about an analysis conducted in this study.
Missing-data handling is also not reported. The enrollment was 6, but the record does not say whether all enrolled participants had post-baseline imaging, were included in the response analysis, or were evaluable at the scheduled assessments. It does not state how withdrawal, unavailable scans, or incomplete follow-up affected endpoint classification. The listed timeframe—evaluated at 6-week intervals, up to an average of 18 weeks—does not specify the analysis population or missing-data rules.
The registry does not identify an intention-to-treat population, a per-protocol population, or another analysis set. It also does not provide participant flow, exclusions from the endpoint analysis, or a statistical analysis plan. These omissions prevent assessment of whether the result, if available elsewhere, would represent all enrolled participants or a selected subset. General methods for handling missing outcomes require assumptions; the record does not identify assumptions or methods used. With no reported response result and no analysis details, multiplicity, missingness, and population definitions remain unresolved aspects of the evidence in the registry record.
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 |
|---|---|---|---|
| Erlotinib | 0 | 6 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Erlotinib | 0 of 6 |
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 |
|---|---|
| Erlotinib | 4 of 6 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Erlotinib |
|---|---|---|
| lymphopenia | Blood and lymphatic system disorders | 2 of 6 |
| anemia | Blood and lymphatic system disorders | 2 of 6 |
| Neutropenia | Blood and lymphatic system disorders | 2 of 6 |
| hearing loss | Ear and labyrinth disorders | 1 of 6 |
| lymphopenia | Blood and lymphatic system disorders | 1 of 6 |
| Neutropenic fever | Blood and lymphatic system disorders | 1 of 6 |
The safety fields list 6 participants at risk for serious adverse events and zero affected. For other adverse events, the record lists 6 at risk and four affected. Deaths are listed as zero. These are participant counts in the registry record; they do not describe the tumor-response endpoint.
The serious-adverse-event term list is empty, so no specific serious event terms are reported in the registry record. The record does not provide the identities or descriptions of the other adverse events, their severity, timing, duration, or attribution to treatment. It also does not explain how adverse events were collected or whether participants had the same length of safety follow-up.
In general, an observed count of zero serious adverse events in a small group does not establish that serious events are impossible or that risk is absent. It describes only the recorded count for the participants included in that safety field. Likewise, the count for other adverse events is not a measure of tumor response, and it cannot establish that an event was caused by erlotinib or by chemotherapy. These safety counts should be read as limited descriptive registry data, not as a comparative safety analysis.
12. What the Result Does, and Does Not, Mean
The registry record answers some descriptive questions: the study was single-group, enrolled 6 participants, was terminated, and specified tumor response as its primary outcome. It also gives the planned response-assessment approach—radiologic testing using RECIST 1.0 at 6-week intervals, up to an average of 18 weeks. These details identify what the study set out to assess, but they do not report the primary outcome result.
The posted data do not show how many participants had a tumor response, the denominator used to calculate a rate, or an estimate of uncertainty. They therefore do not establish the direction or magnitude of the observed response. No response result cannot be interpreted as zero responses, nor can it be interpreted as evidence of a response. The cause of termination and the completeness of endpoint follow-up are also not reported.
The design does not provide a randomized comparator. Even if a response count were available, an outcome in a single treated group would not isolate the effect of erlotinib from the chemotherapy combination or other influences. The record also does not report the treatment exposure details or statistical analysis plan needed to understand how the endpoint was analyzed.
The safety entries describe zero serious adverse events among 6 at risk, four with other adverse events among 6 at risk, and zero deaths. Those counts are descriptive and lack event details, follow-up context, and a comparison group. They do not establish causation or permit a comparative safety conclusion.
In short, the registry supports a limited account of the study's stated question, design, enrollment, and recorded safety counts. It does not support a conclusion about the primary response outcome, the precision of an effect estimate, or the comparative effect of the regimen. The planning illustrations are not observed power and cannot replace missing results.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of the distinction between a registered research question and a reported result. The registry defines a primary outcome, identifies an assessment method, and supplies a timeframe. Yet the record lists no analyses and does not report the response count. A clearly stated endpoint is important, but it cannot answer the research question without observed outcome data.
The study also illustrates how design limits interpretation. A single-group study can describe outcomes among participants receiving the regimen, but a randomized comparison is needed to estimate a direct difference between treatment groups under comparable conditions. This distinction matters particularly for a combination regimen: an outcome observed in the group would not, by itself, attribute an effect to erlotinib separately from the accompanying chemotherapy.
The enrollment of 6 makes precision an important concern even if an endpoint estimate were available. In general, limited participant numbers leave less information for estimating response and can make estimates sensitive to individual outcomes. Because no estimate or confidence interval is posted, readers cannot quantify that uncertainty in this record.
The planning illustrations use assumptions for a two-arm calculation, while the registered trial is single-group. Recognizing that difference helps prevent a planning scenario from being mistaken for the actual design or for evidence of statistical power. More generally, sample-size calculations depend on assumptions and do not demonstrate that a result has been observed.
Finally, the record shows why outcome completeness and analysis transparency matter. It does not explain termination, identify endpoint analysis populations, describe missing-data handling, or give the statistical analysis plan. These omissions constrain what can be concluded from registry data and underscore the value of reporting outcome estimates with their uncertainty and clear analysis details.
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.
Several limitations are specific to the information recorded for this study. The registry lists 6 enrolled participants, but does not report how many had evaluable imaging or contributed to the primary endpoint. The tumor-response count, denominator, rate, and uncertainty interval are not reported in the registry record. This prevents an assessment of both the observed outcome and its precision.
The study is classified as single-group, with allocation not applicable and no masking. There is no randomized comparator in the record, so the design cannot provide a like-for-like treatment contrast. The study evaluates a regimen that includes intermittent erlotinib during platinum-containing chemotherapy, while the intervention listing names erlotinib. Details of the actual chemotherapy, dosing, adherence, and treatment exposure are not reported.
The registry status is terminated, but the reason and its implications for recruitment, treatment, follow-up, and endpoint collection are not reported. The listed primary completion date does not explain those matters. The relationship between the phase 2 and phase 3 designations is also not reported.
The record does not describe eligibility criteria beyond the broad disease description, participant characteristics, analysis populations, missing-data procedures, or statistical analysis plan. It does not report whether any interim analyses occurred. These elements should not be inferred from the study's phase, status, or endpoint description. The listed safety counts also lack event terms for serious events and details for other events. Together, these record-specific gaps make the available information insufficient for evaluating tumor response, comparative benefit, or detailed safety patterns.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Erlotinib (Tarceva) During First Line Standard Platinum Containing Chemo for Advanced Squamous Cell Head and Neck Cancer — NCT00448240.
- Registry record last updated 2025-03-18; status last verified 2025-02.
- Results first posted 2025-03-18.
- 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
NCT00448240 is a terminated, single-group study sponsored by Henry Ford Health System, with phase 2 and phase 3 listed. The record gives an enrollment of 6, a start of 2007-02, and primary completion of 2012-01. The study concerns intermittent erlotinib during first-line standard platinum-containing chemotherapy for advanced squamous cell carcinoma of the head and neck.
The primary outcome is tumor response, assessed with radiologic testing using RECIST 1.0 at 6-week intervals, up to an average of 18 weeks. The registry lists one outcome measure and zero analyses, but does not report a primary response result or explain why the study was terminated.
Safety fields list zero serious adverse events among 6 at risk, four with other adverse events among 6 at risk, and zero deaths. Event details are not reported. The single-group design has no randomized comparator, and the posted record does not support a comparative treatment-effect conclusion.