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 Evaluation of Efficacy and Mechanisms of an Antiinflammatory Intervention for Chemotherapy Related Mucosal Injury (NCT00031551), a randomized phase 2 study. The primary outcome is What is the Clinical Efficacy of an Etanercept Mouthwash Used for the Treatment of Autologous or Allogeneic Peripheral Blood Stem Cell Transplant or Bone Marrow Transplant Treatment-related Stomatitis?.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00031551 |
| Phase | Phase 2 |
| Condition | Stomatitis |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Triple |
| Enrollment | 27 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Institute of Nursing Research (NINR) |
| Results posted | Yes |
2. Clinical Question
Population
The trial concerned people with chemotherapy-related stomatitis in the setting of autologous or allogeneic peripheral blood stem cell transplant or bone marrow transplant treatment. The record describes a pilot study and a main study. Pilot participants contributed descriptive information about pain perception and laboratory techniques; the main study focused on the clinical question. The registry enrollment is 27, but the record says participants withdrew and analyses were terminated before any data were collected. Enrollment should not be read as the number analyzed.
Intervention
The main-study intervention was etanercept mouthwash. The registry describes etanercept 2.5 mg in 20cc mouthwash, swished and spit every 6 hours. It was to begin one day before conditioning chemotherapy. The record describes continuing treatment until oral pain intensity and stomatitis severity scores were both 0, or by bone marrow transplant day.
Comparator
The comparator was placebo mouthwash, described as 20cc and used by swishing and spitting every 6 hours. It was also to begin one day before conditioning chemotherapy. The registry describes the same stopping conditions for the placebo mouthwash. The record does not report an observed comparison between the two groups.
Primary question
The planned primary question was the clinical efficacy of etanercept mouthwash for treatment-related stomatitis. The stated measures were stomatitis grade and oropharyngeal pain, with a timeframe recorded as 2 years. The registry states that analyses ended before any data were collected. It therefore does not establish whether the intervention and comparator differed on either measure.
3. Trial Design
The registry classifies this as a phase 2, randomized, triple-masked, parallel-design study. Randomization means assignment to study groups was intended to use a chance-based process. Triple masking indicates that the record uses that masking classification, but it does not identify which people were masked. The parallel model means the intended groups received their assigned approaches rather than switching between them. These design labels describe the planned structure; they do not indicate that a comparison was completed.
The record distinguishes a pilot study from a main study. The pilot was intended to collect descriptive information about pain perception and assess laboratory techniques for measuring TNF-alpha gene expression in unstimulated saliva, plasma, and mucosal epithelial cells among patients with chemotherapy-related stomatitis. The main study description frames the clinical research around inflammation and etanercept mouthwash. The registry lists etanercept and placebo as the interventions and describes the main-study mouthwash regimens.
Enrollment is listed as 27. The study start is recorded as 2002-03 and primary completion as 2010-08. The status is terminated. The primary-outcome population statement says participants withdrew and analyses were terminated before any data were collected. These details mean that the planned design and enrollment record should be kept separate from completed outcome evidence: the record describes what the trial set out to do, but it does not provide an analyzed clinical comparison.
The sponsor is the National Institute of Nursing Research (NINR). The registry record does not report a detailed account of the reasons for withdrawal or termination, nor does it identify which roles were masked. Those details should not be inferred from the design labels or status.
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 | Main Study Participants | Pilot Study |
|---|---|---|
| STARTED | 2 | 25 |
| COMPLETED | 0 | 25 |
| NOT COMPLETED | 2 | 0 |
Reasons for not completing this period:
| Reason | Main Study Participants | Pilot Study |
|---|---|---|
| Withdrew due to chemotherapy effects | 2 | 0 |
Analysis population for the primary outcome, as defined in the registry: Participants withdrew and analyses were terminated before any data collected.
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 | Main Study Participants | Pilot Study | Total |
|---|---|---|---|---|
| Participants analysed | 2 | 25 | 27 | |
| Age, Continuous | years | 59 (54 to 64) | 46 (32 to 68) | NA (NA to NA) |
| Sex: Female, Male: Female | Participants | 0 | 13 | 13 |
| Sex: Female, Male: Male | Participants | 2 | 12 | 14 |
| Race/Ethnicity, Customized: White | participants | 2 | 16 | 18 |
| Race/Ethnicity, Customized: Hispanic | participants | 0 | 6 | 6 |
| Race/Ethnicity, Customized: Black | participants | 0 | 2 | 2 |
| Race/Ethnicity, Customized: Asian/Pacific Islander | participants | 0 | 1 | 1 |
| Region of Enrollment: United States | participants | 2 | 25 | 27 |
| Education: Grade School | participants | 0 | 2 | 2 |
| Education: High School | participants | 0 | 5 | 5 |
| Education: Some College | participants | 0 | 7 | 7 |
| Education: College Graduate | participants | 1 | 7 | 8 |
| Education: Graduate School | participants | 1 | 4 | 5 |
| Cancer Diagnosis: Breast Cancer | participants | 0 | 7 | 7 |
| Cancer Diagnosis: Non-Hodgkin's Lymphoma | participants | 1 | 8 | 9 |
| Cancer Diagnosis: Hodgkin's Lymphoma | participants | 0 | 4 | 4 |
| Cancer Diagnosis: Multiple Myeloma | participants | 1 | 2 | 3 |
| Cancer Diagnosis: Leukemia | participants | 0 | 4 | 4 |
| Cancer stage: Stage II | participants | 0 | 1 | 1 |
| Cancer stage: Stage III | participants | 1 | 13 | 14 |
| Cancer stage: Stage IV | participants | 0 | 5 | 5 |
| Cancer stage: Unstaged | participants | 0 | 1 | 1 |
| Cancer stage: First Remission | participants | 0 | 1 | 1 |
| Cancer stage: Second Remission | participants | 0 | 1 | 1 |
| Cancer stage: Accelerated Phase | participants | 0 | 1 | 1 |
| Cancer stage: Unknown | participants | 1 | 2 | 3 |
6. Primary Endpoint
The registry lists the primary outcome as What is the Clinical Efficacy of an Etanercept Mouthwash Used for the Treatment of Autologous or Allogeneic Peripheral Blood Stem Cell Transplant or Bone Marrow Transplant Treatment-related Stomatitis?, measured over 2 years.
| 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 was intended to assess clinical efficacy by measuring stomatitis grade and oropharyngeal pain. Stomatitis is described in the registry as inflammation of the mucous membranes of the oral cavity and oropharynx, with features including tissue erythema, edema, and atrophy, which may progress to ulceration. The record does not specify a single unit, summary statistic, or numerical endpoint value. Its stated timeframe is 2 years.
In general, measuring both condition severity and pain can address related but distinct aspects of a clinical problem: a grading measure characterizes the observed condition, while a pain measure captures the participant’s symptom experience. That is a general explanation of why the two types of measure may be informative together, not a claim about the measures’ performance in this trial. The registry does not report the assessment instrument, scoring details, or a prespecified rule for combining or prioritizing the two measures.
The outcome was not measured in the completed analysis described by the record. The primary-outcome population statement says that participants withdrew and analyses were terminated before any data were collected. Consequently, there are no reported endpoint values, between-group estimates, or uncertainty intervals on which to judge the planned clinical comparison.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical fact is that the registry reports no collected primary-outcome data. The planned efficacy measures were stomatitis grade and oropharyngeal pain, but the record states that participants withdrew and analyses were terminated before data collection. There is therefore no reported estimate of the difference between etanercept mouthwash and placebo mouthwash on either measure. There is also no reported confidence interval, p-value, or other inferential result for the primary question.
This absence is not evidence that the groups had the same outcomes, nor does it establish that one regimen was better. A comparison requires outcome observations from the groups and a defined analysis of those observations. Without such data, an effect cannot be estimated from the registry record. The enrollment figure of 27 describes the enrollment recorded for the study; it does not replace outcome data and should not be treated as an analyzed sample size.
In general, a confidence interval communicates the range of effect values compatible with an estimate under the analysis assumptions. A narrow interval can indicate greater precision, while a wide interval indicates more uncertainty. Here, the record reports no primary-outcome estimate or interval to interpret. Likewise, a p-value cannot be used to evaluate the planned comparison because no primary-outcome analysis is reported. The registry lists no analyses.
The record includes adverse-event figures for the main-study participants, but those are safety counts and do not answer the primary efficacy question. They also have a very small recorded at-risk group. Such counts can describe what the registry recorded for those participants, but they do not support a broad safety conclusion. The pilot-study adverse-event fields report zero at risk and zero affected; this does not provide an efficacy comparison.
The study status is terminated, and the record says that withdrawals occurred before data collection and analysis ended. That history explains why no clinical efficacy estimate is available in the registry record, but the record does not give further details about why participants withdrew or why the study ended. No inference about the direction or magnitude of an unobserved treatment effect is justified.
Any planning calculation is distinct from trial evidence. In general, sample-size illustrations describe hypothetical design requirements under stated assumptions; they do not create observed results or demonstrate observed power. The registry characterizes its planning assumptions as illustrations, not as the trial’s own design. Since the primary outcome has no reported data, such calculations cannot fill the evidentiary gap.
9. Statistical Methods Explained
The record identifies randomization, triple masking, and a parallel model. These labels describe intended design features, not the quality or completeness of an analysis. The registry does not report a detailed statistical analysis plan, the randomization procedure, or which people were masked. Those particulars are not reported in the registry record.
What does randomization contribute?
General explanation: random assignment is used to reduce systematic differences between groups at the start of a study, on average. It does not guarantee that groups will be identical, especially when the number of participants is limited. Randomization also does not prevent bias introduced later by missing outcomes, deviations from assigned treatment, or choices in analysis. For this trial, the registry says the study was randomized but reports no collected primary outcome data, so the intended benefit of randomization cannot yield an efficacy estimate.
What does masking mean?
General explanation: masking is intended to limit the influence of knowledge about treatment assignment on care, reporting, or assessment. The record’s classification is triple-masked, but it does not say which parties were masked or how masking was maintained. No conclusion about the success of masking can be drawn from the label alone. In particular, masking does not substitute for outcome collection or resolve the absence of analyzed results.
How should the parallel design be understood?
General explanation: in a parallel trial, participants are assigned to one study group and outcomes are compared across groups. The registry describes an etanercept mouthwash group and a placebo mouthwash group for the main study. It does not report an observed comparison. The pilot is described separately as a source of descriptive information about pain perception and laboratory techniques, so its purpose should not be confused with the main study’s planned clinical efficacy question.
What would an effect estimate and interval ordinarily tell readers?
General explanation: an effect estimate summarizes the observed contrast between groups for a defined outcome. A confidence interval expresses uncertainty around that estimate under the statistical model and assumptions used. The estimate concerns the size and direction of a contrast; the interval helps show how precisely it is estimated. A p-value, when reported, addresses compatibility between data and a specified null hypothesis under an analysis model. None of these concepts can be applied to a trial comparison without outcome data and a reported analysis. The registry provides no such efficacy estimate, interval, or p-value.
What is over-dispersion, and is it relevant?
General explanation: over-dispersion occurs when observed variability is greater than a statistical model expects. If an analysis assumes less variability than the data actually have, uncertainty can be understated and inference may be unreliable. Whether over-dispersion would matter depends on the outcome scale and the model used. The record does not report an outcome model or data distribution, so it is not possible to determine whether over-dispersion arose or how it was handled in this trial.
How do the planning assumptions fit?
The registry describes planning calculations as illustrations rather than as the trial’s original design. The stated assumptions include a two-sided significance level of 0.05, equal allocation, and a normal approximation; the effect to detect is characterized as a stated fraction of the observed effect. These are planning assumptions, not observed trial findings. The record also says analyses ended before any data were collected for the primary outcome. Consequently, the assumptions do not supply an efficacy result, and any sample-size calculation should be understood as a planning illustration rather than observed power.
General explanation: power is a design-stage probability tied to a specified effect, variability, sample size, and analysis assumptions. It is not a measure of whether a completed study found an effect, and a post hoc calculation based on an observed estimate does not replace an interval or a transparent analysis. The registry does not report a trial-specific statistical plan or a completed primary-outcome analysis, so the details needed to assess those choices are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists four outcome measures and identifies a primary outcome concerning clinical efficacy. It also lists a secondary question about toxicity of etanercept mouthwash for transplant-related stomatitis. The record does not report how the outcomes were ordered for analysis, whether any multiplicity adjustment was planned, or how conclusions across multiple measures would be controlled. A formal multiplicity strategy is not reported in the registry record.
In general, multiplicity refers to the added risk of misleading findings when many hypotheses, outcomes, time points, or subgroup comparisons are assessed. A prespecified strategy can distinguish primary from supportive analyses and can account for repeated testing where appropriate. The registry identifies a primary question, but the trial ended before primary-outcome data were collected; therefore no multiplicity procedure can be evaluated from reported efficacy results.
Missing outcome data and no outcome data collected are different situations. Missing-data methods address incomplete observations when at least some outcome information exists. Here, the primary-outcome statement says participants withdrew and analyses were terminated before any data were collected. The registry does not describe a missing-data method, imputation approach, sensitivity analysis, or reasons for individual withdrawal. These items are not reported in the registry record.
The record also does not specify an intention-to-treat population, a per-protocol population, or another analysis population for the primary comparison. The enrollment figure does not establish how many participants would have been included in an analysis. In the absence of outcome observations and a reported population definition, there is no analysis population or missing-data assumption to assess for the primary efficacy question.
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 |
|---|---|---|---|
| Main Study Participants | 0 | 2 | 0.0% |
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 |
|---|---|
| Main Study Participants | 2 of 2 |
| Pilot Study | 0 of 0 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Main Study Participants | Pilot Study |
|---|---|---|---|
| Nausea | Gastrointestinal disorders | 2 of 2 | 0 of 0 |
| Diarrhea | Gastrointestinal disorders | 2 of 2 | 0 of 0 |
| Fatigue | General disorders | 1 of 2 | 0 of 0 |
The registry reports adverse-event figures separately for Main Study Participants and Pilot Study. For Main Study Participants, the serious-adverse-event fields report 2 at risk and 0 affected. The other-adverse-event fields report 2 at risk and 2 affected. For Pilot Study, the record reports 0 at risk and 0 affected in both the serious and other adverse-event fields. These are the registry’s recorded counts; they should not be expanded into rates or combined across groups.
The deaths field is null for both groups, so deaths are not reported in the registry record. No serious adverse-event terms are listed. The counts do not give event descriptions, timing, severity beyond the serious-versus-other classification, or a comparison suitable for estimating relative risk. The small recorded at-risk group for the main study also limits what can be learned from these entries.
In general, adverse-event counts describe events recorded among people at risk during a defined observation period. Their interpretation depends on complete follow-up, clear event definitions, and an appropriate denominator. The registry does not provide additional detail on those points in the entries. These figures therefore document the record’s safety reporting but do not establish the overall safety profile of either mouthwash regimen.
12. What the Result Does, and Does Not, Mean
The record establishes that a randomized phase 2 study was planned to investigate etanercept mouthwash for treatment-related stomatitis, with placebo mouthwash as the comparator. Its primary clinical measures were stomatitis grade and oropharyngeal pain. It also records a pilot component intended to collect descriptive information about pain perception and laboratory techniques. These registry entries describe the research question and intended approach.
The record does not establish whether etanercept mouthwash improved, worsened, or had no effect on either primary measure. It reports that participants withdrew and analyses were terminated before any primary-outcome data were collected. With no outcome values or analysis, there is no estimated treatment contrast, no precision measure, and no inferential basis for deciding between possible effects. The lack of a result should not be mistaken for evidence of no difference.
The adverse-event fields provide limited descriptive counts for the recorded groups. They do not supply a comparative safety analysis or enough context to characterize the safety of the intervention. A null entry for deaths is not a reported count of zero; it means that deaths are not reported in the registry record.
More generally, a trial’s design can make a research question testable, but design labels and enrollment alone cannot answer it. Evidence about a treatment contrast depends on observed outcomes, a defined comparison, and analysis with uncertainty described. Those elements are not reported for the primary endpoint in this record. Any planning assumptions remain planning assumptions and cannot stand in for observed clinical evidence.
The appropriate reading is therefore limited: the registry records an intended clinical investigation and its early termination before primary-outcome data collection, rather than a completed efficacy evaluation. It does not support a conclusion about the direction, size, or precision of an effect.
13. Why This Trial Matters Statistically
This record illustrates why trial registration details and trial outcome evidence answer different questions. The design information describes the intended comparison: randomized assignment, triple masking, a parallel model, etanercept mouthwash, and placebo mouthwash. The outcome information, by contrast, determines whether the planned clinical question can be answered. In this record, the primary-outcome statement says that analyses ended before any data were collected.
That distinction matters statistically because a carefully described design does not guarantee an estimable treatment effect. Randomization can support a fair comparison when outcome data are collected and analyzed appropriately, but it cannot recover outcomes that were never collected. Nor can enrollment alone establish precision, power, or efficacy. Readers need estimates and measures of uncertainty to judge what the data can say; this record reports none for the primary clinical question.
The record also demonstrates why safety counts must be read in context. Event counts without richer information about follow-up, definitions, and comparative analysis should not be treated as comprehensive evidence. Here, the small recorded at-risk group and the absence of reported deaths data or event terms further constrain interpretation.
Finally, a registry record can identify unanswered methodological questions as well as planned methods. The exact masking roles, statistical analysis plan, missing-data strategy, and analysis populations are not reported. Recognizing these omissions prevents readers from filling them with assumptions. The statistical importance of this trial record lies less in a treatment estimate than in the clear boundary it sets: the planned comparison is described, but the primary clinical result is unavailable.
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 main limitation is that the registry states participants withdrew and analyses were terminated before any data were collected for the primary outcome. There are no reported values for stomatitis grade or oropharyngeal pain, and no efficacy estimates or uncertainty measures. The planned clinical comparison therefore cannot be evaluated from the record.
The record gives an enrollment figure of 27, but it does not identify an analyzed population or explain how enrollment relates to the pilot and main study components. The enrollment figure should not be treated as the denominator for an efficacy analysis. The status is terminated, but further detail about the reasons for withdrawal and termination is not reported in the registry record.
Several methodological details are also absent. The registry does not report which parties were masked, the randomization procedure, a statistical analysis plan, the scoring instrument for stomatitis or pain, the method for combining or prioritizing the primary measures, or the planned handling of missing data. It does not identify an intention-to-treat or other analysis population. These details cannot be reconstructed from the design labels.
The safety entries are limited: the record provides counts for two groups, but does not report event terms, deaths, or broader context for follow-up and event ascertainment. The deaths fields are not reported as numeric values. These limitations restrict interpretation of safety as well as efficacy.
The primary outcome timeframe is recorded as 2 years, but the record does not establish that outcome assessment continued for that period. A stated timeframe is not evidence of completed follow-up, particularly given the statement that no primary-outcome data were collected.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Evaluation of Efficacy and Mechanisms of an Antiinflammatory Intervention for Chemotherapy Related Mucosal Injury — NCT00031551.
- Registry record last updated 2017-12-06; status last verified 2017-11.
- Results first posted 2015-12-30.
- 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
NCT00031551 is a phase 2 randomized, triple-masked, parallel study sponsored by the National Institute of Nursing Research (NINR). The registry records enrollment of 27, a start of 2002-03, primary completion of 2010-08, and status as terminated. It describes a pilot study and a main study, with etanercept mouthwash compared with placebo mouthwash in the main study.
The planned primary outcome was clinical efficacy measured by stomatitis grade and oropharyngeal pain, with a timeframe recorded as 2 years. The registry states that participants withdrew and analyses were terminated before any data were collected. No primary efficacy estimate or analysis is reported. Adverse-event counts are recorded for the main study and pilot study, but do not establish a comparative safety result.