EVALUATION OF AMIFOSTINE FOR MUCOSAL AND HEMOPOETIC PROTECTION AND CARBOPLATIN, TAXOL, RADIOTHERAPY IN THE MANAGEMENT OF PATIENTS WITH HEAD AND NECK CANCER.(GCC 0202): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for EVALUATION OF AMIFOSTINE FOR MUCOSAL AND HEMOPOETIC PROTECTION AND CARBOPLATIN, TAXOL, RADIOTHERAPY IN THE MANAGEMENT OF PATIENTS WITH HEAD AND NECK CANCER.(GCC 0202) (NCT00270790), a non-randomized phase 2 study. The primary outcome is Participants With Mucositis and Hematological Toxicities With the Addition of Radioprotector Amifostine.

2. Clinical Question

3. Trial Design

This is a phase 2, single-site evaluation, identified as NCT00270790. The official title describes concurrent carboplatin, Taxol and radiotherapy in patients with advanced locoregional squamous cell carcinomas of the head and neck, with amifostine evaluated for mucosal and hematopoietic protection. The registry lists the study as completed, with a start date of 2002-05 and primary completion in 2005-12. Mohan Suntharalingam is listed as sponsor.

The design is non-randomized, unmasked and single-group. The listed regimen combines amifostine, carboplatin, Taxol and radiotherapy; there is no separate control arm in the record. Consequently, the design can describe outcomes observed among participants receiving this regimen, but it cannot isolate the contribution of amifostine through a direct between-group comparison. Differences from an external or historical experience would not be equivalent to a randomized comparison, and no such comparison is reported.

The registry records 21 patients enrolled. Its primary-outcome description says that 16 patients were analyzed because 5 patients withdrew. Reasons for withdrawal, the timing of withdrawal and the handling of data after withdrawal are not reported in the registry record. The record describes the primary outcome as participants with mucositis and hematological toxicities and gives a timeframe of 3 years. It also lists response rates based on the study regimen as a secondary outcome.

The treatment was intended to address toxicity concerns associated with chemotherapy and radiotherapy while also examining tumor response. That purpose is distinct from an observed conclusion: the study design and stated aim do not themselves show whether toxicity was reduced or what cancer response occurred. The registry does not report a randomized allocation process, masking procedures, a comparator regimen or a detailed statistical analysis plan.

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

Analysis population for the primary outcome, as defined in the registry: 21 patients enrolled, however only 16 patients were analyzed due to 5 patients withdrawals.

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.

6. Primary Endpoint

The registry lists the primary outcome as Participants With Mucositis and Hematological Toxicities With the Addition of Radioprotector Amifostine, measured over 3 years.

The primary endpoint is titled as the number of participants with mucositis and hematological toxicities with the addition of amifostine. It is a participant-count outcome: the intended summary is how many people experienced the defined toxicities, rather than a continuous measure such as a laboratory value averaged across the group. The registry gives a timeframe of 3 years.

The endpoint description says blood work, identified as CMP, was collected before chemotherapy administration and evaluated for neutropenia, leukopenia and anemia. Toxicity levels were assessed using Common Terminology Criteria for Adverse Events, version 3.0, and monitored based on the dose of amifostine given. These details indicate that the endpoint was intended to capture clinically graded toxicity as well as blood-related effects during treatment.

Such an endpoint is relevant to the stated study purpose because mucosal and hematological toxicities are among the harms the regimen was intended to examine. A participant count can make the frequency of recorded events easier to interpret, but its meaning depends on consistent definitions, observation and complete follow-up. The registry record does not give the actual primary-endpoint count, toxicity grades, event-specific results or the rules used to combine mucositis with hematological toxicities. It therefore describes the measure but not its observed value.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

For a binary participant outcome, the basic descriptive result is a count of participants meeting the event definition, interpreted relative to the participants assessed for that outcome. The registry describes the primary measure as participants with mucositis and hematological toxicities, so the intended outcome is a count rather than a measurement of average toxicity severity. A count alone is not sufficient to assess how common an event was unless its denominator and the outcome definition are also clear. In this record, the primary result itself is not reported, so no primary event frequency can be interpreted.

Confidence intervals are a general statistical tool for expressing uncertainty around an estimate. For an event proportion, an interval communicates how much uncertainty remains about the underlying event frequency given the observed data and the analysis assumptions. It is not a range that contains a fixed proportion of individual participants, and it does not establish that an intervention caused an outcome. No primary-endpoint confidence interval is reported in the registry record. It is therefore not possible to assess the precision of the intended toxicity estimate from this record.

Hypothesis tests and p-values address compatibility between observed data and a specified statistical model or null hypothesis. They do not measure the size or clinical importance of an effect, and they do not repair limitations in study design. The registry lists 0 analyses and reports no p-value for the primary outcome. A significance threshold or a test procedure for the primary endpoint is not reported in the registry record. No statistical evidence for or against the stated toxicity question can be reconstructed from the endpoint description alone.

The study is listed as a single-group, non-randomized evaluation. In a single-group design, a descriptive event count can characterize the participants observed under the regimen, but it does not provide the direct treatment contrast available from concurrent randomized groups. Randomization is a general method for balancing measured and unmeasured factors between treatment groups on average. Since this study has no randomized comparator, that protection against confounding does not apply. This does not make descriptive reporting impossible; it limits what causal interpretation can be supported.

The primary endpoint description refers to toxicity levels assessed using Common Terminology Criteria for Adverse Events, version 3.0, and monitoring based on amifostine dose. In general, standardized grading rules can improve consistency in how events are classified. For interpretation, however, it is also important to know the specific grades counted, whether the endpoint includes any grade or a selected severity threshold, how repeated events are handled, and whether different toxicity types are combined. Those details are not reported in the registry record. The record also does not provide event-specific primary results that would allow readers to distinguish mucositis from the named blood-related outcomes.

Missing data can affect an estimate when participants do not complete follow-up or outcome measurements are unavailable. The registry says that 21 patients enrolled and 16 were analyzed because 5 withdrew. In general, excluding participants with incomplete follow-up can produce a biased result if withdrawal is related to health status, toxicity or another factor associated with the outcome. That possibility cannot be evaluated: reasons and timing for withdrawal, the amount of outcome data missing, and any method for addressing missing observations are not reported. The analyzed population is identified, but the analysis population rules beyond that description are not reported.

The registry's sample-size assumptions describe planning illustrations based on an observed effect, not the design rationale of this trial. They specify a two-sided significance level of 0.05, equal allocation between two arms and a normal approximation, with the effect to detect expressed as a stated fraction of the observed effect. The trial itself is single-group, so those two-arm assumptions should not be read as its actual allocation or analysis design. The record does not report the trial's own sample-size justification. A planning illustration is not observed power, and no sample-size illustration can replace a reported estimate with uncertainty from the actual outcome data.

Over-dispersion is a general concern when observed variability in data is greater than a chosen statistical model expects. It can make standard errors too small if the model does not account for that extra variability. For a simple binary count, interpretation still depends on the outcome definition, denominator and independence assumptions; more complex clustering or repeated observations may require a different model. The registry does not report a model for the primary outcome, repeated-measure handling or an over-dispersion assessment. No such method should be inferred from the endpoint description.

Multiplicity refers generally to the increased chance of false-positive findings when several outcomes or hypotheses are tested without an appropriate plan. The record lists 2 outcome measures, including the primary toxicity outcome and a secondary response-rate outcome. It does not report whether multiplicity adjustments or a hierarchy of tests were planned. Since no analyses are listed, there are no reported test results to interpret in light of multiplicity. The registry also does not report subgroup analyses or interim analyses.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("AMIFOSTINE +CARBOPLATIN, TAXOL +RT")
n      <- c(16)
events <- c(16)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  AMIFOSTINE +CARBOPLATIN, TAXOL +RT  16  16  100.0  79.4  100.0  80.6  100.0  80.5  99.9

11. Multiplicity, Missing Data and Analysis Populations

The record lists 2 outcome measures: the primary outcome concerning mucositis and hematological toxicities, and a secondary outcome concerning response rates based on the study regimen. It records 0 analyses. Whether the investigators planned a hierarchy of outcomes, adjusted for multiple testing or conducted any interim analyses is not reported in the registry record. Without analysis results or a stated statistical plan, it is not possible to assess how multiple outcomes were handled or whether selective reporting affected the available results.

For the primary outcome, the registry says that 21 patients enrolled and 16 were analyzed because 5 withdrew. It does not report why or when they withdrew, whether all remaining participants had complete toxicity follow-up, or how missing endpoint data were treated. The reasons for withdrawal could matter if they were related to toxicity or clinical status, but the record does not permit that assessment. No imputation method, sensitivity analysis or missing-data assumption is reported.

The stated analyzed population is not accompanied by detailed analysis-population rules. The registry does not report whether the primary outcome was assessed in all enrolled patients, only those with evaluable observations, or another defined group beyond the statement that 16 were analyzed. No intention-to-treat analysis is reported. These omissions limit interpretation of both the denominator and the potential influence of withdrawals on the endpoint estimate. Because the numerical primary result is also absent, neither the size nor the direction of any missing-data impact can be assessed.

12. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent non-serious adverse event terms

The adverse-event summary lists one treatment group, with 21 participants at risk for serious events and 0 affected. It also lists 21 participants at risk for other events and 21 affected. These are registry counts, not a comparison between treatment groups. The record does not provide serious-event terms, and the other-event terms are not identified. The figure for other events therefore cannot be interpreted as a count for a particular symptom, toxicity type or severity level.

The primary endpoint is about mucositis and hematological toxicities, while the adverse-event categories are reported separately. The registry does not establish that the categories use the same definitions, observation period or analysis population as the primary outcome. The adverse-event counts should not be substituted for the missing primary endpoint result, nor do they describe the severity or duration of events.

The deaths entry is null, so a death count is not reported in the registry record. A reported count of 0 affected in the serious-event category is a descriptive registry entry; it does not establish that no safety concerns occurred, particularly when event terms and fuller follow-up details are unavailable. These figures should be read as limited safety reporting for the listed group, not as a comparative estimate of safety.

13. What the Result Does, and Does Not, Mean

The study addresses a specific statistical question: whether amifostine, given with carboplatin, Taxol and radiotherapy, could be associated with fewer mucosal and hematological toxicities in patients with head and neck cancer. The registry also identifies cancer response as an outcome of interest. These aims explain what the investigators intended to study, but they are not results. The registry record does not report the numerical primary-endpoint result or the secondary response result.

The available design information supports a description of a phase 2, single-site, single-group, non-randomized evaluation. It does not support a direct estimate of how outcomes would differ with versus without amifostine. In general, a single-group result can describe observed experience under a regimen, but a causal claim requires a credible comparison and assumptions that address other explanations for the observed outcomes. No concurrent comparator is reported.

The record identifies 21 enrolled patients and 16 analyzed after 5 withdrawals, but does not explain the withdrawals or missing-data handling. The separate adverse-event summary reports serious and other event counts, yet does not identify the other-event terms and does not report the primary outcome. Those entries offer only a limited view of safety reporting and cannot fill the gap in the endpoint result.

Accordingly, the record establishes the study question, regimen, design and some participant-flow and adverse-event information. It does not establish the frequency of the primary toxicities in the analyzed population, the precision of that frequency, or whether adding amifostine changed toxicity or response. A null or positive treatment effect should not be inferred from the absence of a reported primary result. The appropriate conclusion is that the registry describes an evaluation but does not provide enough outcome analysis to answer its central comparative-sounding question.

14. Why This Trial Matters Statistically

This trial matters statistically because its stated aim concerns whether adding a protective agent changes toxicity, while its listed design is a single-group evaluation. That distinction is fundamental. Describing outcomes among people receiving a regimen can be informative about what was observed in that group, but it does not by itself separate the effects of one component from the effects of the other treatments or from patient and follow-up differences.

The registry also illustrates why an endpoint definition and an endpoint result are not interchangeable. It identifies mucositis and hematological toxicities, describes blood work and toxicity grading, and gives a timeframe. Yet it does not report the primary count or an analysis. Without those results, readers cannot assess the event frequency, uncertainty or variation across toxicity types. An outcome's clinical relevance does not compensate for missing numerical reporting.

Participant flow is also important to statistical interpretation. The record states that 21 patients enrolled and 16 were analyzed after 5 withdrawals. Without reasons, timing or missing-data methods, readers cannot tell whether the analyzed group adequately represents all enrolled participants for the toxicity question. This uncertainty is separate from the limitations inherent in having no comparator.

Finally, the registry separates adverse-event reporting from the stated primary endpoint. Counts in broad serious and other categories do not establish the frequency of the specifically defined mucosal and blood-related toxicities. Clear event definitions, denominators, follow-up and analysis results are needed to connect safety reporting to the research question. The study is therefore a useful example of how design and reporting determine what can be concluded, independently of the importance of the clinical question.

15. Limitations

• The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
• Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.

The main limitation for interpretation is the absence of a numerical result for the primary endpoint. The registry defines the outcome and its timeframe, but does not give the number of participants with mucositis or hematological toxicities, toxicity grades, event-specific counts or an uncertainty estimate. The secondary response-rate outcome is listed without a numerical result as well.

The single-group, non-randomized design has no concurrent comparator. It therefore cannot provide a direct treatment contrast for the addition of amifostine, and any observed outcomes would be difficult to attribute to one component of the regimen rather than the combined treatment or other influences. The record does not report masking.

The population description notes that 16 patients were analyzed after 5 withdrawals among 21 enrolled. Reasons and timing for withdrawal, completeness of follow-up and methods for handling missing data are not reported. The record does not give enough detail to evaluate selection into the analyzed group or determine how withdrawals affected endpoint assessment.

Other specific limitations include the absence of a reported statistical analysis plan, outcome-analysis results, multiplicity approach and detailed endpoint rules. Although the endpoint description names mucositis, neutropenia, leukopenia and anemia, it does not state which grades counted or how these conditions were combined. The adverse-event summary does not identify the other-event terms, and the death count is not reported in the registry record. These gaps prevent a fuller assessment of both efficacy-related outcomes and safety.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: EVALUATION OF AMIFOSTINE FOR MUCOSAL AND HEMOPOETIC PROTECTION AND CARBOPLATIN, TAXOL, RADIOTHERAPY IN THE MANAGEMENT OF PATIENTS WITH HEAD AND NECK CANCER.(GCC 0202) — NCT00270790.
• Registry record last updated 2017-02-01; status last verified 2016-12.
• Results first posted 2016-08-08.
• 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.

19. Record Summary

NCT00270790 is a completed phase 2, single-site, non-randomized, unmasked, single-group evaluation of amifostine with carboplatin, Taxol and radiotherapy for head and neck cancer. The stated target population is patients with advanced locoregional squamous cell carcinomas of the head and neck. The registry lists a start date of 2002-05, primary completion in 2005-12 and Mohan Suntharalingam as sponsor.

The primary outcome concerns participants with mucositis and hematological toxicities over 3 years. The description refers to blood work, named blood-related toxicities and CTCAE 3.0 grading. The registry records 21 enrolled and 16 analyzed after 5 withdrawals. It lists response rates as a secondary outcome, 2 outcome measures and 0 analyses. No numerical primary or secondary outcome result is reported. The adverse-event summary reports 0 affected in the serious category and 21 affected in the other category, both among 21 at risk; the other-event terms are not identified.