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 Azacitidine and Cisplatin for Patients With Recurrent or Metastatic Squamous Cell Carcinoma of the Head and Neck (NCT00443261), a na phase 1 study. The primary outcome is Evaluate the Safety and Toxicity of Azacitidine (5-azacytidine, Vidaza®) and Cisplatin Combination.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00443261 |
| Phase | Phase 1 |
| Condition | Squamous Cell Carcinoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Kansas Medical Center |
| Results posted | Yes |
2. Clinical Question
Population
The population of interest was patients with recurrent or metastatic squamous cell carcinoma of the head and neck. The registry lists squamous cell carcinoma as the condition and describes the study group as patients with recurrent or metastatic disease. It does not report further eligibility details or the characteristics of the enrolled patient.
Intervention
The intervention was the combination of azacitidine and cisplatin. The registry lists both as drug interventions and describes the study group as receiving both. Doses, schedules, dose levels, and any dose-escalation procedures are not reported in the registry record.
Comparator
There was no separate comparator group. The study is recorded as single-group, with allocation listed as not applicable and no masking. The posted record therefore does not support a treatment-versus-control comparison.
Primary question
The primary question was whether the azacitidine and cisplatin combination could be evaluated for safety and toxicity in this population. The primary outcome title names safety and toxicity, and its timeframe is Weeks 1-12, 24, 36. The registry states that the 1 patient enrolled died after cycle 1 with rapidly progressing cancer, so there were no data to analyze for the primary outcome measure.
3. Trial Design
This was a Phase I study of azacitidine in combination with cisplatin in patients with recurrent or metastatic squamous cell carcinoma of the head and neck. The registry identifies the design as single-group, with no masking and allocation listed as not applicable. It records 1 participant enrolled. The listed study group received both azacitidine and cisplatin; there is no separate control arm in the record.
The study start is recorded as 2007-02, and primary completion as 2008-06. Its status is listed as terminated. The record names the University of Kansas Medical Center as sponsor. It does not report the reason for termination, the date or circumstances of the termination decision, or further information about study conduct.
The brief summary states that the aim was to evaluate safety and toxicity of the combination. Phase I studies generally focus on assessing safety and tolerability, but details of this study’s dose plan, escalation rules, stopping criteria, or monitoring schedule are not reported in the registry record. It would therefore be inappropriate to infer how dose decisions were intended to be made.
The registry also describes imaging-based reassessment for patients with measurable disease, with evaluation every 8 weeks and confirmatory scans 4 weeks after an initial objective response. It explicitly says response was not the primary endpoint. The primary outcome timeframe is listed as Weeks 1-12, 24, 36, but no primary outcome data are available to analyze. The record does not report a randomized comparison or a blinding procedure.
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 | Arm 1: SCCHN |
|---|---|
| STARTED | 1 |
| Accrual in 10 Months | 1 |
| COMPLETED | 0 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Arm 1: SCCHN |
|---|---|
| Death | 1 |
Analysis population for the primary outcome, as defined in the registry: The 1 patient enrolled in the study died after cycle 1 with rapidly progressing cancer. Therefore, no data to analyze for primary outcome measure.
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 | 1: SCCHN |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | Years | 52 (SD 0) |
| Gender: Female | Participants | 1 |
| Gender: Male | Participants | 0 |
| Region of Enrollment: United States | participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Evaluate the Safety and Toxicity of Azacitidine (5-azacytidine, Vidaza®) and Cisplatin Combination, measured over Weeks 1-12, 24, 36.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary outcome is titled “Evaluate the Safety and Toxicity of Azacitidine (5-azacytidine, Vidaza®) and Cisplatin Combination.” Its stated timeframe is Weeks 1-12, 24, 36. The registry does not specify a unit, a numerical outcome parameter, or a dispersion measure. Those details are not reported in the registry record, so the primary endpoint cannot be described as a defined numerical measure such as a rate, score, or laboratory change.
In general, safety and toxicity outcomes are used in early-phase research to characterize unwanted effects and to inform whether a regimen can be studied further. The specific criteria and measurement rules for this study are not reported in the registry record. Although the outcome description discusses imaging and assessment of objective response for patients with measurable disease, it states that response was not the primary endpoint. The registry reports that the participant died after cycle 1 with rapidly progressing cancer and that no data were available to analyze for the primary outcome. Consequently, the record does not provide an evaluable primary safety or toxicity result.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical fact is that enrollment was 1 and the registry reports no data to analyze for the primary outcome measure. The participant died after cycle 1 with rapidly progressing cancer. This means the posted record does not supply an estimate of the primary safety or toxicity outcome, nor a measure of its uncertainty. It is not possible to infer a pattern of toxicity or to quantify the primary outcome from this record.
The study had a single group and no comparator. Even if an outcome estimate had been reported, this design would not provide a direct randomized comparison with another treatment or with no treatment. Observed events in a single group can describe what was recorded among enrolled participants, but cannot by themselves separate effects of the study drugs from the underlying illness or other possible causes. The registry does not provide enough outcome information to make even that descriptive assessment for the primary endpoint.
The adverse-event record lists 1 participant at risk for serious adverse events and 1 affected, with “Pnemonia” listed as the top serious term at 100.0%. It also lists 1 participant at risk for other adverse events and 0 affected. These entries should be read as limited recorded counts in a study with enrollment of 1, not as stable estimates of event frequency in a wider population. The adverse-event record’s deaths field is not reported in the registry record, while the narrative states that the enrolled patient died after cycle 1. The record does not establish that pneumonia caused the death or that either event was caused by treatment.
No outcome analysis entries are listed. There is no reported effect estimate, confidence interval, or statistical test for the primary outcome. A confidence interval, in general, describes the uncertainty around an estimate under a specified statistical model; without an analyzable endpoint estimate, it cannot be interpreted for the primary outcome. Likewise, no comparative effect can be calculated from the single-group design.
The appropriate reading is therefore limited: the registry describes a planned evaluation of combination safety and toxicity, reports 1 enrollment, and states that no primary outcome data could be analyzed. The record does not establish the safety profile, efficacy, or comparative performance of azacitidine and cisplatin.
9. Statistical Methods Explained
The registry records a Phase I, single-group design, with allocation listed as not applicable and masking listed as none. These design labels determine what statistical conclusions the record can support. There is no randomized allocation to different treatment groups and no control group. As a result, the record does not support an estimate of a between-group treatment effect. The study’s stated purpose was evaluation of safety and toxicity, not a randomized comparison.
What can a single-group study estimate?
In general, a single-group study can describe outcomes observed among participants who received the study intervention, if those outcomes are collected and reported. Such descriptions do not by themselves establish what would have happened to the same participants under a different intervention. Without a comparator, differences attributable to treatment cannot be separated statistically from differences related to the participants’ condition, their course of illness, or other influences. Here, the registry reports enrollment of 1 and no data to analyze for the primary outcome, so it does not provide an analyzable primary endpoint estimate even as a within-group description.
What does the primary outcome record contain?
The primary outcome title refers to safety and toxicity, and the timeframe is Weeks 1-12, 24, 36. The unit, parameter, and dispersion are not reported in the registry record. Those omissions matter because an outcome needs a defined measurement and a clear rule for how it is summarized before its numerical results can be interpreted. The record’s description also discusses imaging assessment for response, while explicitly stating that response was not the primary endpoint. No response outcome results are reported in the primary outcome entry.
In general, an estimate summarizes an observed quantity, while a measure of dispersion or a confidence interval communicates uncertainty or variability. Those quantities require outcome data and a specified analysis. No primary outcome estimate, dispersion measure, confidence interval, or analysis is reported. Thus, no inferential result can be reconstructed from the registry entry.
Why do the adverse-event counts need careful reading?
The adverse-event entry reports counts of participants at risk and affected. For serious adverse events it lists 1 at risk and 1 affected, and names “Pnemonia” as the top serious term at 100.0%. For other adverse events it lists 1 at risk and 0 affected. These are descriptive entries, not evidence of a precise underlying event probability. In general, rates based on very small groups are highly sensitive to each observed participant, and they provide little information about how often events might occur in a broader population. A percentage can look definitive while representing only the recorded count in this study.
Over-dispersion is a general statistical concern when variation in observed counts exceeds what a simple probability model expects. It can arise in some event-count settings because participants differ or events cluster, but this record does not provide enough data to assess over-dispersion. In particular, the event count and population are too limited for a meaningful model of variation. The reported percentage should therefore not be treated as a precise population estimate.
What can be inferred from the absence of an analysis?
The registry lists 1 outcome measure and 0 analyses. This indicates that no analysis results are posted in the record. It does not reveal why no analysis was entered beyond the primary outcome narrative, which says the participant died after cycle 1 and that there were no data to analyze for that measure. The record does not describe an analysis plan, model, adjustment method, interim analysis, or sample-size justification. None should be inferred.
In general, a statistical test evaluates data under stated assumptions, while an estimate and interval describe magnitude and uncertainty. Neither becomes meaningful merely because a study has a named endpoint. Analyzable observations, a defined endpoint, and a stated method are needed. For this record, the registry does not provide an analyzed primary outcome or a reported comparative test. Any numerical sample-size planning illustration associated with an observed effect would be a hypothetical planning calculation, not evidence of observed power or a substitute for outcome results.
The registry’s response-assessment description gives an imaging schedule for patients with measurable disease and a confirmatory scan procedure after an initial objective response. It does not provide response data or a statistical method for evaluating response. Since response is stated not to be the primary endpoint, these procedural details do not fill the missing primary safety and toxicity results.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 1 outcome measure and 0 analyses. It does not report a multiplicity adjustment, a hierarchy of endpoints, or a plan for handling multiple statistical tests. Multiplicity arises when several hypotheses or outcomes are tested: repeated testing can increase the chance of at least one false-positive result if no suitable approach is used. That general concern cannot be evaluated for this study because no analysis results or detailed testing plan are reported in the registry record.
The primary outcome entry states that the participant died after cycle 1 with rapidly progressing cancer and that there were no data to analyze for the primary outcome measure. The record does not explain whether any component observations were collected, whether any data were considered missing, or how missing observations would have been handled under the planned analysis. It also does not describe an analysis population, such as a defined safety population or an efficacy population. Those details are not reported in the registry record.
In general, missing-data methods depend on what is missing, why it is missing, and the outcome being analyzed. A participant’s death can prevent later assessments, but the registry does not state a planned approach for such circumstances. No imputation, censoring, or sensitivity-analysis strategy is reported. The available record therefore does not support an assessment of whether missingness could bias an estimate, or whether a particular statistical remedy was used.
The absence of reported analysis details should not be interpreted as evidence that a particular method was or was not used outside the record. It means that the posted information does not document those methods. The primary endpoint cannot be statistically interpreted without outcome data and a stated analysis approach.
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 |
|---|---|---|---|
| 1: SCCHN | 1 | 1 | 100.0% |
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 |
|---|---|
| 1: SCCHN | 0 of 1 |
Most frequent serious adverse event terms
| Event term | Organ system | 1: SCCHN |
|---|---|---|
| Pnemonia | Infections and infestations | 1 of 1 |
Serious events with the highest arm-level rate: pnemonia (100.0%).
The adverse-event entry lists 1 participant at risk for serious adverse events and 1 affected. “Pnemonia” is the listed top serious term, with 100.0% recorded. For other adverse events, the entry lists 1 participant at risk and 0 affected. These counts describe the registry’s entries for this study; they should not be read as precise estimates for other patients.
The denominator is particularly important: enrollment was 1. A percentage based on such a small study group can change markedly with a different observation, and it gives little information about variation in a larger population. The record does not state whether “Pnemonia” was considered related to either drug, and it does not establish causation. The adverse-event death count is not reported in the registry record. Separately, the primary outcome narrative says the participant died after cycle 1 with rapidly progressing cancer. The registry does not say that pneumonia caused the death.
12. What the Result Does, and Does Not, Mean
The record documents an early-phase study intended to evaluate safety and toxicity of azacitidine combined with cisplatin in recurrent or metastatic squamous cell carcinoma of the head and neck. It reports 1 participant enrolled and states that the participant died after cycle 1 with rapidly progressing cancer. The primary outcome entry says there were no data to analyze. These are the limits of the primary outcome evidence in the registry.
The adverse-event counts show what was recorded for the enrolled participant, including a serious adverse-event entry for “Pnemonia” and no affected participants in the other adverse-event category. Such entries are descriptive. They do not establish whether an event was caused by treatment, nor do they provide a dependable estimate of how common an event would be beyond this study. The registry does not report an analysis of causality or a primary safety estimate.
The record does not show whether the combination is safe or unsafe in a broader population, and it does not establish whether it improves or worsens cancer outcomes. No efficacy conclusion can be drawn from the imaging description because no response results are reported and response was not the primary endpoint. No treatment comparison is possible because the study had a single group and no comparator.
In general, a trial result needs an outcome measurement, an analysis, and enough information to understand uncertainty and context. Those elements are not available for the primary outcome in this record. The registry’s stated absence of analyzable primary outcome data is not equivalent to a finding of no toxicity or no effect. It means the primary question remains unanswered by the posted results.
13. Why This Trial Matters Statistically
This study illustrates why a trial’s design and its analyzable results must be considered together. A Phase I designation and a safety-focused objective describe the study’s intent, but they do not guarantee that a safety estimate will be available. The registry records enrollment of 1 and explicitly reports no data to analyze for the primary outcome after the participant died following cycle 1.
It also illustrates the limits of percentages derived from very small groups. A serious adverse-event entry of 100.0% may appear to convey a precise frequency, but in this record it accompanies a count of 1 affected participant among 1 at risk. It is a description of this study’s recorded experience, not a stable estimate for a broader clinical population. Counts, denominators, and the underlying study design all matter when interpreting adverse-event summaries.
The single-group structure is also important. Without a comparator, an event observed during the study cannot be attributed to the intervention through a controlled between-group comparison. The record provides no causal analysis and no comparative efficacy result. These limits remain even when an adverse event is clearly recorded.
Finally, the registry shows why transparency about endpoint definitions and analyses matters. The primary outcome title names safety and toxicity, but the record does not provide a unit, numerical parameter, dispersion measure, or analysis result. It also includes a response-assessment description while identifying response as non-primary. Readers need to distinguish a study’s aims and assessment procedures from results that can actually be estimated. In this case, the primary outcome remains unevaluable in the posted record.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The most consequential limitation is that enrollment was 1 and the registry reports no analyzable primary outcome data. The participant died after cycle 1 with rapidly progressing cancer. No primary safety or toxicity estimate is posted, so the central objective cannot be assessed from these results.
The study has no comparator and is recorded as single-group, with allocation not applicable and no masking. Consequently, there is no randomized treatment contrast, and the record cannot support a comparative estimate. The adverse-event counts are based on a very limited study group and do not establish event frequency in a wider population or causation by treatment.
The registry does not report the primary outcome’s unit, parameter, dispersion, detailed measurement rules, or a statistical analysis. It also does not report a sample-size justification, analysis plan, interim-analysis approach, missing-data method, or defined analysis populations. The reason for termination is not reported in the registry record. These gaps prevent a fuller assessment of how the study was intended to operate and how outcomes were meant to be interpreted.
The outcome description includes imaging assessment for response, but response is stated not to be the primary endpoint, and no response results are reported. The adverse-event death field is not reported in the registry record, even though the primary outcome narrative states that the enrolled patient died after cycle 1. The record does not explain this difference in reporting or link the death to a specific adverse event.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Azacitidine and Cisplatin for Patients With Recurrent or Metastatic Squamous Cell Carcinoma of the Head and Neck — NCT00443261.
- Registry record last updated 2017-02-27; status last verified 2017-01.
- Results first posted 2017-02-27.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
This Phase I study evaluated azacitidine and cisplatin in patients with recurrent or metastatic squamous cell carcinoma of the head and neck. It is recorded as single-group, with no masking, allocation not applicable, and enrollment of 1. The sponsor is the University of Kansas Medical Center. The study start is listed as 2007-02, primary completion as 2008-06, and status as terminated.
The primary objective concerned safety and toxicity, with a stated timeframe of Weeks 1-12, 24, 36. The registry reports that the participant died after cycle 1 with rapidly progressing cancer and that no data were available to analyze for the primary outcome. The adverse-event entry records 1 participant affected by a serious adverse event, lists “Pnemonia” at 100.0%, and records 0 affected for other adverse events. No analyses are listed. The record does not provide a comparative treatment result or an analyzable primary outcome estimate.