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 Saracatinib in Treating Patients With Recurrent or Metastatic Head and Neck Cancer (NCT00513435), a na phase 2 study. The primary outcome is Overall Response Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00513435 |
| Phase | Phase 2 |
| Condition | Metastatic Squamous Neck Cancer With Occult Primary Squamous Cell Carcinoma, Recurrent Metastatic Squamous Neck Cancer With Occult Primary, Recurrent Squamous Cell Carcinoma of the Hypopharynx |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 9 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Cancer Institute (NCI) |
| Results posted | Yes |
2. Clinical Question
Population
People with recurrent or metastatic head and neck squamous cell carcinoma were the intended population. The registry condition list spans multiple head and neck sites and disease stages, and includes recurrent disease and metastatic disease. Enrollment was 9 participants. The registry record does not give a more specific description of the participants who entered the trial, such as their baseline characteristics or the precise eligibility criteria applied.
Intervention
The intervention was saracatinib, described as an enzyme inhibitor. Participants received it by mouth or through a PEG tube once daily on days 1-56. Courses were to repeat every 8 weeks in the absence of disease progression or unacceptable toxicity. Laboratory biomarker analysis was also listed as an intervention; the record does not describe its procedures or role in the primary outcome.
Comparator
There was no comparator group. The study is listed as a single-group, non-randomized trial, so its observations do not provide a direct comparison with a control treatment or with usual care. Any interpretation is limited to the enrolled group and the outcomes recorded for it.
Primary question
The primary outcome was overall response rate, measured as a number of participants, from treatment start for up to 12 weeks. The registry description says response was determined as indicated in the protocol but does not specify the response criteria. It also does not report the number of participants who responded or an estimated response rate. Overall survival was listed as a secondary outcome, without a reported result in the registry record.
3. Trial Design
This was a phase II, single-group trial sponsored by the National Cancer Institute. The registry record lists the study as completed, with a start date of 2007-07 and a primary completion date of 2011-03. Enrollment was 9. Allocation was listed as not applicable, and masking was none. With no concurrent comparison group, the design describes outcomes among participants treated with saracatinib; it does not estimate a randomized treatment contrast.
The treatment description specifies saracatinib by mouth or PEG tube once daily on days 1-56. Courses were to repeat every 8 weeks if disease had not progressed and toxicity remained acceptable. The registry also lists laboratory biomarker analysis, but gives no further detail about how biomarkers were collected or analyzed. The stated treatment schedule is a protocol description, not evidence that every participant completed the planned treatment.
The primary outcome was overall response rate, with the unit recorded as participants and a timeframe from treatment start for up to 12 weeks. The description refers to response determination in the protocol, but the response definition is not reported in the registry record. Overall survival was the listed secondary outcome. Although the record identifies two outcome measures, it lists no analyses. No response count, survival estimate, or statistical test result is reported.
A single-group phase II design can describe observed outcomes and support further study, but without a control group it cannot separate treatment-associated observations from other explanations, such as the course of disease or differences among participants. The small enrollment also means that each participant's outcome can have substantial influence on the group-level summary. The registry record does not report a sample-size justification, a protocol statistical plan, or interim analyses.
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 | Treatment (Enzyme Inhibitor Therapy) |
|---|---|
| STARTED | 9 |
| COMPLETED | 9 |
| 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 | Treatment (Enzyme Inhibitor Therapy) |
|---|---|---|
| Participants analysed | 9 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 7 |
| Age, Categorical: >=65 years | Participants | 2 |
| Age, Continuous | years | 53.8 (SD 13.7) |
| Sex: Female, Male: Female | Participants | 2 |
| Sex: Female, Male: Male | Participants | 7 |
| Region of Enrollment: United States | participants | 9 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Response Rate, measured over From the start of treatment for up to 12 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
Overall response rate is intended to describe how many participants meet the study's definition of a tumor response during the specified assessment period. The registry records the endpoint as a participant count, with assessment from the start of treatment for up to 12 weeks. In general, a response endpoint can provide an early measure of tumor change and can be useful in a phase II study when the goal is to characterize an initial activity signal.
The record says response was determined as indicated in the protocol, but does not state the criteria, how assessments were made, or how missing or unevaluable assessments were handled. Those details matter because response classifications depend on prespecified definitions and assessment procedures. A participant count is also not by itself a response rate unless the denominator and analysis population are clear.
The registry record does not report the number of responders, a rate, an uncertainty interval, or an analysis for this primary outcome. Consequently, the endpoint's planned role can be described, but its observed result and precision cannot be assessed from the posted information. Overall survival appears as a secondary outcome, but its definition, follow-up summary, and result are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive limit is that the registry record identifies a primary endpoint but does not give its observed value. Overall response rate was defined as a participant count and assessed from treatment start for up to 12 weeks. The record does not report the count of participants meeting the response definition, a response rate, or an uncertainty interval. Therefore, the magnitude and precision of the primary outcome cannot be evaluated from these registry results.
Enrollment was 9 in a single-group study. With a small group, observed participant counts can be sensitive to individual outcomes, and any estimate based on them would ordinarily have substantial uncertainty. This is a general statistical point, not a calculation of the uncertainty in this trial. Since the response count and analysis population are not reported, no trial-specific interval or precision statement can be made.
The study did not include a control arm. That means any observed outcomes would describe this treated group and would not establish how outcomes compare with an alternative treatment or with no study treatment. A single-group design also cannot by itself distinguish a treatment effect from other influences on outcome. This limitation remains even if a response count were available.
The registry record lists overall survival as a secondary outcome but gives no survival result or analysis. No time-to-event estimate, follow-up summary, or uncertainty measure is reported. Thus, survival cannot be interpreted from the information recorded as results.
The posted safety information records serious adverse events among 5 of 9 participants and other adverse events among 9 of 9 participants in the treatment group. It also lists several serious adverse event terms with percentages. Those observations describe events recorded in this small group; they do not establish whether an event was caused by saracatinib. The listing includes “Death not assoc w CTCAE term- Sudden death,” but the record does not provide a death count in the corresponding field or further details that would support a causal interpretation.
Overall, the registry record documents the design, treatment schedule, enrollment and some safety observations, but it does not provide the primary efficacy result or a statistical analysis. Any conclusion about response or survival would go beyond the information recorded.
9. Statistical Methods Explained
The statistical information in a trial record helps distinguish a planned question from a reported result. In this study, the primary outcome is named as overall response rate, the unit is participants, and the assessment period is described. However, the registry record lists no analyses and does not report an outcome value. A defined endpoint does not, by itself, show how the endpoint was analyzed or what was observed.
What does an endpoint count mean?
A participant count records how many people meet an outcome definition. A rate additionally requires a denominator: the group of participants eligible for inclusion in the calculation. In general, response rates also depend on the response criteria and on rules for participants whose assessments are missing or cannot be classified. The record does not give the response count, denominator used for an analysis, criteria, or rules for unevaluable participants. It is therefore not possible to reconstruct or verify a response rate from the registry information.
The timeframe is stated as from treatment start for up to 12 weeks. A specified timeframe identifies the period relevant to the endpoint, but does not establish that every participant was assessed at the same time or completed assessment. Assessment timing and handling of incomplete observations are not reported in the registry record.
Why does a confidence interval matter?
In general, a confidence interval expresses the statistical uncertainty around an estimated quantity under a specified method. For a participant count or response rate, it can show how much uncertainty remains even when an observed estimate is available. It does not guarantee that the true value lies within the interval, and it does not address design limitations such as the absence of a control group. No confidence interval for the primary outcome is reported in this registry record, and no interval can be reconstructed without the required outcome information and analysis choices.
What can a single-group study estimate?
A single-group study can summarize outcomes within the enrolled participants. It has no randomized comparator, so it cannot directly estimate a between-group treatment contrast. General statistical methods cannot remove that structural limitation from an outcome count. In addition, participants may differ in disease history, prognosis, or other characteristics, but the registry record does not report baseline data with which to describe such variation. The design should therefore be read as descriptive of its participants rather than as a controlled test of relative treatment effects.
How should the enrollment be understood?
The recorded enrollment is 9. In general, a small enrollment makes estimates less stable because the outcome of an individual participant can have a noticeable influence on a group summary. The degree of uncertainty depends on the outcome data and the chosen statistical method; neither a trial-specific precision estimate nor the observed primary outcome is reported. A planning sample-size calculation is different from an observed result and does not show that a study had adequate information for a particular conclusion.
Planning illustrations based on an observed effect are not the trial's design justification. The stated assumptions for such illustrations describe a two-arm comparison, equal allocation, a two-sided significance level of 0.05, and a normal approximation. Those assumptions do not match this single-group design, which has no allocation between two arms. No trial-specific sample-size justification is reported in the registry record. Any planning illustration should therefore be understood only in the context of its stated assumptions, not as evidence of observed power or as a substitute for the missing trial analysis.
What is known about survival analysis?
Overall survival is listed as a secondary outcome. In general, survival analyses use both the time to an event and the status of participants whose event has not been observed by the end of follow-up. The analysis depends on follow-up duration, event definitions, censoring, and the population included. None of those analysis details or a survival result is reported in the registry record. A secondary outcome title alone does not reveal the estimate, precision, or method used.
How should adverse event summaries be read?
Safety summaries commonly count participants who experienced events and may also list event terms. A participant can experience more than one event, so participant counts and event counts are not interchangeable. The registry lists 5 of 9 participants with serious adverse events and 9 of 9 with other adverse events in the treatment group. It also lists serious adverse event terms with percentages. The record does not clarify all counting conventions or provide details that support attributing those events to treatment. The entries should be read as recorded safety observations, not as comparative risks.
Finally, the record lists no analyses. It does not describe a statistical analysis plan, any adjustment for multiple outcomes, interim analyses, analysis populations, or missing-data methods. These omissions prevent assessment of whether reported analyses followed a prespecified approach. They also mean that no inferential conclusion should be inferred from outcome labels or safety listings alone.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists overall response rate as the primary outcome and overall survival as a secondary outcome. It reports two outcome measures but no analyses. The record does not state whether there were additional planned comparisons, whether any multiplicity adjustment was planned, or how the primary and secondary outcomes were prioritized for statistical testing. Without an analysis plan or test results, the impact of multiple outcome testing cannot be assessed.
Missing-data handling is also not described. The registry record does not specify the analysis population for response, how participants without an assessment were classified, or whether any participants were excluded from an outcome calculation. It does not report the number of participants with evaluable response assessments. These details are important because the denominator and treatment of incomplete observations can affect a participant count or rate.
The record does not describe an intention-to-treat population, a per-protocol population, or another analysis set. It also does not report whether every enrolled participant started treatment or completed the planned assessment period. Those matters are not reported in the registry record and should not be inferred from the enrollment total or treatment schedule.
For overall survival, follow-up, censoring rules, and the analysis population are not reported. These omissions prevent evaluation of how incomplete follow-up was handled. Since the record lists no outcome analyses, the posted information does not permit an assessment of multiplicity, missingness, or deviations from a planned analysis.
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 |
|---|---|---|---|
| Treatment (Enzyme Inhibitor Therapy) | 5 | 9 | 55.6% |
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 |
|---|---|
| Treatment (Enzyme Inhibitor Therapy) | 9 of 9 |
Most frequent serious adverse event terms
| Event term | Organ system | Treatment (Enzyme Inhibitor Therapy) |
|---|---|---|
| Death not assoc w CTCAE term- Sudden death | General disorders | 3 of 9 |
| Lymphopenia | Blood and lymphatic system disorders | 2 of 9 |
| Inf norm ANC/gr1/2 neut-Cellulitis(skin) | Blood and lymphatic system disorders | 1 of 9 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Treatment (Enzyme Inhibitor Therapy) |
|---|---|---|
| Fatigue (asthenia, lethargy, malaise) | Metabolism and nutrition disorders | 6 of 9 |
| Hemoglobin decreased | Blood and lymphatic system disorders | 4 of 9 |
| Diarrhea | Gastrointestinal disorders | 4 of 9 |
| Nausea | Gastrointestinal disorders | 4 of 9 |
| AST, SGOT | Renal and urinary disorders | 4 of 9 |
| Alkaline phosphatase | Renal and urinary disorders | 4 of 9 |
| Constipation | Gastrointestinal disorders | 3 of 9 |
| Vomiting | Gastrointestinal disorders | 3 of 9 |
| Lymphopenia | Blood and lymphatic system disorders | 2 of 9 |
| Platelets | Blood and lymphatic system disorders | 2 of 9 |
Serious events with the highest arm-level rate: death not assoc w ctcae term- sudden death (33.3%), lymphopenia (22.2%), inf norm anc/gr1/2 neut-cellulitis(skin) (11.1%).
The treatment group safety summary records serious adverse events in 5 of 9 participants and other adverse events in 9 of 9 participants. These are participant-level counts in the group, not evidence that every event was caused by treatment. A participant may have more than one adverse event, and the registry record does not provide a detailed account of event onset, duration, severity beyond the serious-event classification, or attribution.
The listed serious adverse event terms include “Death not assoc w CTCAE term- Sudden death” at 33.3%, “Lymphopenia” at 22.2%, and “Inf norm ANC/gr1/2 neut-Cellulitis(skin)” at 11.1%. These are the terms and percentages recorded; the record does not explain their denominators or provide case-level details. The wording attached to sudden death should not be treated as a complete causal assessment. The corresponding death-count field is not populated, so a death count is not reported in the registry record.
Because there is no comparator group and enrollment was 9, these figures describe recorded safety observations in this group only. They cannot establish comparative safety or quantify a treatment-attributable risk. Adverse event reporting and causal attribution should not be conflated.
12. What the Result Does, and Does Not, Mean
The registry record establishes that this completed phase II study enrolled 9 participants in a single-group treatment design. Saracatinib was administered by mouth or PEG tube according to the recorded schedule, and overall response rate was named as the primary outcome. The posted information also includes safety summaries and lists overall survival as a secondary outcome.
What the record does not establish is the observed response result. It provides no number of participants who responded, no response rate, no statistical analysis, and no uncertainty interval. It also provides no overall survival result. A reader cannot use the endpoint title or treatment description as a substitute for those results.
Even if an outcome estimate were available, the absence of a control group would limit what it could establish. A single-group result could describe what happened among enrolled participants, but could not directly show how those outcomes compare with another treatment. This is a design limitation, not a matter that can be resolved by statistical significance alone.
The safety listings indicate that serious and other adverse events were recorded among participants, with specific serious-event terms listed. They do not establish that saracatinib caused those events, nor do they provide a comparison against another group. The small enrollment also limits the breadth of safety experience represented by the record.
The results should therefore be interpreted as an incomplete registry account of a small, non-randomized study: design and treatment details are present, some safety information is present, but the primary efficacy result and statistical analyses are not reported. No conclusion about treatment benefit, lack of benefit, or comparative safety follows from the registry information alone.
13. Why This Trial Matters Statistically
This trial illustrates why the design and reporting context matter as much as an endpoint label. Overall response rate is identified as the primary outcome, but the registry does not report the response count or an analysis. Without those results, readers cannot evaluate the size or precision of the primary outcome. The contrast between a named endpoint and an unavailable estimate is an important feature of registry-based interpretation.
The study also shows the limits of interpreting a single-group phase II design. Such a study can record outcomes among people receiving a treatment, but cannot provide a randomized comparison. The design's role is different from that of a controlled trial, and a descriptive result should not be treated as a comparative treatment effect. This distinction remains important even when a registry includes numerical safety observations.
Enrollment was 9, making the scope of the record clear: it describes a small participant group rather than a broad population. General statistical reasoning indicates that small groups can yield imprecise estimates, while the absent response result means precision cannot be quantified for this trial. The registry also identifies survival as secondary but does not report its result, demonstrating why outcome labels alone are not enough for analysis.
Finally, the absence of reported analyses, analysis populations, and missing-data methods limits reproducibility and critical appraisal. These are not merely technical details: they affect which participants and observations underlie an estimate. The record is therefore useful for understanding the study's structure and recorded safety information, while leaving the primary efficacy question unanswered in its posted results.
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 registry record does not provide the response criteria, assessment procedures, or rules for missing and unevaluable response assessments. Although it specifies a timeframe of up to 12 weeks from treatment start, it does not report whether assessments were completed for all participants or how the analysis denominator was determined. The primary endpoint's observed count and statistical analysis are also absent.
The study was single-group, with no randomized or concurrent control arm. Consequently, there is no direct basis for estimating a relative treatment effect from this record. Enrollment was 9, which also constrains how broadly the observed group could represent the listed range of recurrent, metastatic, and stage-specific conditions. Baseline characteristics and the final analysis population are not reported.
Overall survival is listed as a secondary outcome, but the registry record does not describe follow-up, censoring, an estimate, or an analysis. The record lists no analyses at all, and does not report a statistical plan, interim analyses, sample-size justification, multiplicity procedure, or missing-data strategy. The treatment schedule is described, but adherence, treatment exposure, and discontinuation details are not reported.
Safety information is limited to participant counts and selected serious adverse event terms. The record does not give event-level narratives or enough detail to assess causal attribution, timing, or recurrence. It also does not populate the death-count field. These reporting gaps limit interpretation of both efficacy and safety beyond the observations explicitly recorded.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Saracatinib in Treating Patients With Recurrent or Metastatic Head and Neck Cancer — NCT00513435.
- Registry record last updated 2014-04-30; status last verified 2013-08.
- Results first posted 2013-10-18.
- 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
NCT00513435 is a completed phase II, single-group study sponsored by the National Cancer Institute, with enrollment of 9. The registry lists a start date of 2007-07 and a primary completion date of 2011-03. Participants were scheduled to receive saracatinib by mouth or PEG tube once daily on days 1-56, with courses repeating every 8 weeks in the absence of progression or unacceptable toxicity.
Overall response rate was the primary outcome, measured as a participant count from treatment start for up to 12 weeks. Overall survival was listed as secondary. The registry record reports no analyses and does not provide an efficacy result. Safety summaries record serious adverse events in 5 of 9 participants and other adverse events in 9 of 9, alongside selected serious-event terms. The study has no comparator group, and the record does not report a statistical plan, analysis populations, or missing-data methods.