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 Caphosol Study: Prevention of Oral Mucositis in Children, Adolescents and Young Adults Receiving Chemotherapy (NCT02539342), a randomized phase 3 study. The primary outcome is Measure the Development of Oral Mucositis (Grade >/= 2) in Children, Adolescents and Young Adults..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02539342 |
| Phase | Phase 3 |
| Condition | Oral Mucositis |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Medical College of Wisconsin |
| Results posted | Yes |
2. Clinical Question
Population
Children, adolescents and young adults receiving chemotherapy were the intended population. The registry describes the primary outcome population as children receiving chemotherapy, and reports that one participant enrolled in the Caphosol arm and none in the control arm.
Intervention
The intervention was Caphosol rinse, intended to begin at the start of chemotherapy. The arm description specifies use 4 times per day, with duration linked to chemotherapy completion, blood count recovery and resolution of oral mucositis.
Comparator
The control intervention was Biotene, also described as being used 4 times per day. Participants in that arm were also to keep a study diary. No participants were enrolled in the control arm.
Primary question
The trial asked whether Caphosol could prevent oral mucositis of grade 2 or higher, assessed using CTCAE 4.0. With no control participants and only one participant overall, the registry results cannot establish a comparative effect.
The study was designed as a randomized, controlled trial in people receiving chemotherapy, with the intended comparison between Caphosol and Biotene. The clinical question concerns prevention: whether the Caphosol regimen changes the occurrence of oral mucositis at the specified severity threshold. The primary outcome was a count of participants, rather than a continuous measurement or a time-to-event estimate.
The arm descriptions outline different mouth-rinse regimens, but the registry does not report enough enrolled participants to compare their outcomes. It reports one participant in the Caphosol arm and none in the control arm. The primary outcome population description also states those enrollment counts. Consequently, the question remains clinically relevant as the study’s objective, but the posted results do not provide an estimate of the difference between the interventions. The reason for termination is not reported in the registry record.
3. Trial Design
The registry identifies this as a Phase 3, randomized, open-label trial with a parallel-group model and no masking. The sponsor was Medical College of Wisconsin. The study began in 2016-10, its primary completion date was 2017-03-02, and its status is listed as terminated. Enrollment is recorded as 1.
The intended comparison assigned participants to a Caphosol arm or a control arm using Biotene. The Caphosol regimen was described as use 4 times per day, beginning at chemotherapy initiation and continuing according to the timing and clinical conditions described in the arm entry. The control regimen was Biotene 4 times per day, with a study diary for recording doses and symptoms. Both descriptions refer to use during the chemotherapy period and to oral mucositis or blood-count-related conditions, although the control description is truncated in the registry entry. Additional details about the intended treatment schedule are not reported in the registry record.
Open-label means that the trial was not masked; the registry lists masking as none. In general, lack of masking can affect participant-reported symptoms, care decisions or assessment, depending on how outcomes are collected. The registry specifies use of CTCAE 4.0 for evaluating the primary outcome but does not report who assessed it or whether assessors were independent.
The intended randomized parallel comparison could not be meaningfully evaluated with the enrollment reported. One participant was enrolled in the Caphosol arm, and none in the control arm. The randomization method, allocation concealment, reasons for termination, recruitment progress and any amendments are not reported in the registry record. The status and enrollment counts describe the trial’s recorded conduct; they do not explain why enrollment was limited or why the study ended.
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 | Caphosol Arm | Control Arm |
|---|---|---|
| STARTED | 1 | 0 |
| COMPLETED | 1 | 0 |
| NOT COMPLETED | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: We only had 1 patient enrolled on the Caphosol Arm and none on the Control Arm
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.
Population: We had only 1 enrollment overall and that was to the Caphosol arm.
| Characteristic | Unit | Caphosol Arm | Control Arm | Total |
|---|---|---|---|---|
| Participants analysed | 1 | 0 | 1 | |
| Age, Categorical: <=18 years | Participants | 1 | 0 | 1 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 | 0 | 0 |
| Age, Categorical: >=65 years | Participants | 0 | 0 | 0 |
| Sex: Female, Male: Female | Participants | 0 | 0 | 0 |
| Sex: Female, Male: Male | Participants | 1 | 0 | 1 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 0 | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 1 | 0 | 1 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 1 | 0 | 1 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Region of Enrollment: United States | participants | 1 | — | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Measure the Development of Oral Mucositis (Grade >/= 2) in Children, Adolescents and Young Adults., measured over 24 months.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was the development of oral mucositis at grade 2 or higher, assessed using CTCAE 4.0. The registry expresses the outcome as a count of participants and gives a timeframe of 24 months. The outcome is intended to capture whether participants experienced mucositis reaching the stated severity threshold while receiving chemotherapy.
In general, a binary endpoint records whether each participant did or did not experience an event that meets a defined criterion. Counting participants with the event can support comparisons between randomized arms when the event status and the number assessed are available in each arm. A clearly specified severity threshold also distinguishes clinically graded mucositis from less severe findings that do not meet the endpoint definition.
The registry’s outcome population description states that one participant enrolled in the Caphosol arm and none in the control arm. With no control participants, an arm-to-arm comparison of event frequency cannot be estimated. The posted results do not provide a usable comparative effect estimate or its uncertainty. The registry does not report further operational details about endpoint ascertainment, such as assessment timing within the stated timeframe, handling of repeated assessments, or the treatment of participants with incomplete outcome information.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical fact is the very limited enrollment: the registry reports one participant in the Caphosol arm and none in the control arm. The study was intended to compare Caphosol with Biotene, but a randomized comparison requires outcome information from participants in both arms. Because no control participants were enrolled, the posted results cannot estimate how the outcome differed between the assigned regimens.
The primary endpoint was a participant count for oral mucositis of grade 2 or higher, assessed using CTCAE 4.0 over a stated timeframe of 24 months. The registry does not provide an inferential analysis of this endpoint. Its analysis count is listed as 0, and no comparative effect estimate, confidence interval or p-value is reported. This means the results do not quantify either the direction or precision of a treatment contrast. The absence of a reported comparison should not be interpreted as evidence that the interventions have the same effect.
With only one enrolled participant, any observation from the Caphosol arm would describe that participant rather than establish a pattern in the intended population. The zero enrollment in the control arm makes even a descriptive between-arm contrast unavailable. A confidence interval, in general, expresses the uncertainty around an estimated quantity; without an estimable comparative quantity, there is no reported interval to interpret. Statistical significance cannot be assessed from the registry entries.
The randomized design describes the intended method for reducing baseline differences between groups, but randomization cannot produce a reliable comparison when one arm has no participants. The registry does not report details of the random sequence, allocation process or analysis population. It also does not report a reason for termination or a reason for the low enrollment. These omissions prevent an explanation of why recruitment stopped and whether any design or analysis decisions changed.
The registry lists two outcome measures but no analyses. The primary outcome entry itself says it was not reduced to one comparison because it has several categories or time points. No category-specific results are available in the registry record recorded for this analysis. Therefore, neither the primary outcome nor any secondary outcome can support a treatment-effect conclusion from the posted information.
The adverse-event entries report zero serious events and zero other events for the one participant at risk in the Caphosol arm. The control arm has zero participants at risk. Those counts are descriptive and do not support a meaningful comparison of safety between arms. In particular, no event count in an arm with no enrolled participants can be read as evidence about that intervention’s safety.
Overall, the posted data document a trial that was randomized in design but terminated with enrollment too limited for the planned clinical question to be answered. They do not establish benefit, lack of benefit, harm or equivalence. The appropriate statistical reading is that the treatment contrast is not estimable from the registry results, and the uncertainty is substantial because the intended comparative evidence was not generated.
9. Statistical Methods Explained
The outcome is recorded as a count of participants with an event. In general, this is a binary outcome: each participant is classified according to whether oral mucositis reaches the specified grade threshold during the outcome timeframe. A comparison of binary outcomes usually relies on both the number of participants with the event and the number assessed in each arm. The registry reports one participant enrolled in the Caphosol arm and none in the control arm, so the intended two-arm contrast cannot be calculated from the enrollment information.
What does randomization contribute?
In general, randomization assigns participants to treatment groups by chance. When enrollment is adequate and follow-up is available, this approach can help balance factors that might otherwise influence outcomes, including factors that were not measured. It does not guarantee balance in a very small study, and it cannot compensate for an arm with no participants. The registry labels the study randomized, but the randomization sequence, method of allocation and concealment procedures are not reported in the registry record.
The trial is described as parallel-group, meaning the intended design compares separate groups assigned to the different interventions. It is open-label, with masking listed as none. In general, masking can limit the influence of knowledge about assigned treatment on outcome assessment or participant reporting. Its absence does not automatically invalidate results, but it can matter when outcome evaluation involves judgment or reporting. The registry specifies CTCAE 4.0 for the primary outcome but does not report the assessor, assessment procedures or any safeguards against assessment bias.
Why are effect estimates and confidence intervals absent?
An effect estimate summarizes the contrast between interventions, while a confidence interval describes the statistical uncertainty around that estimate. For this trial, the control arm has no enrolled participants, so there is no observed control-group outcome distribution with which to form the planned comparison. The registry reports no comparative estimate, confidence interval or p-value. This is not a finding of no difference; it is a lack of estimable comparative evidence.
In general, small samples yield imprecise estimates, even when both arms contain participants. Here the missing control arm means the problem is more fundamental than ordinary imprecision: the intended contrast cannot be formed from the reported enrollment. Standard inferential procedures cannot recover information that was not collected from a comparison group.
How should the sample-size assumptions be read?
The registry describes sample-size planning illustrations based on the observed effect, with a two-sided significance level of 0.05, equal allocation between arms and a normal approximation. It also describes the target effect as a stated fraction of the observed effect. These are planning illustrations, not a report of the trial’s original design justification and not observed power. No numerical sample-size estimates are reported in the registry record for interpretation.
In general, sample-size planning asks how many participants would be needed under specified assumptions to detect a chosen effect with a chosen error rate. Such planning depends on assumptions about the effect, outcome variability or event frequency, allocation and analysis method. An illustration tied to an observed effect is especially difficult to interpret when the trial has no usable between-arm estimate. It should not be treated as evidence that the trial had a particular probability of detecting an effect. The registry does not report the original sample-size justification, the target enrollment rationale or a protocol-based power calculation.
For binary outcomes, the event rate and the number of participants assessed in each group are central to precision. No event rates or comparative estimate are available in the posted results. General statistical explanations about confidence intervals, significance testing and sample-size planning therefore describe concepts; they do not supply missing inferential results for this trial.
What do over-dispersion and model choice mean?
Over-dispersion is a general concern when observed variability is greater than a statistical model expects. It is especially relevant for some count or clustered-data models. This endpoint is specified as a count of participants with an event, a binary outcome rather than a reported rate or repeated count process. The registry does not identify a fitted model or report a dispersion estimate. No claim about over-dispersion or model adequacy can therefore be made for the posted analysis.
The registry lists no analyses. It does not report whether the planned comparison would have used a risk difference, relative measure, regression model or another method. Nor does it state how the stated timeframe was incorporated into analysis. Without a documented analysis and adequate outcome data in both arms, there is no statistical model result to interpret.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists two outcome measures and an analysis count of 0. The primary outcome entry notes that it has several categories or time points and was not reduced to one comparison. No category-specific outcome results or multiplicity adjustment are reported in the registry record.
In general, testing multiple outcomes, time points or categories creates more opportunities to find an apparently notable result by chance. A statistical plan may identify a primary comparison, specify how related tests will be handled, or use an adjustment. The registry does not report a multiplicity strategy, a hierarchy of outcomes or a plan for selecting among time points. Since no analyses are recorded, there are no reported test results for which the consequences of multiplicity can be evaluated.
Missing-data methods are also not reported in the registry record. It is not stated how the analysis would have handled participants with missing endpoint assessments, incomplete diaries, withdrawal or unavailable follow-up. The enrollment count does not itself establish whether the participant who enrolled completed all outcome assessments.
The registry does not describe an intention-to-treat population, a per-protocol population or another analysis population. In general, an intention-to-treat analysis evaluates participants according to randomized assignment, while other analysis populations may use additional eligibility or adherence rules. The record does not say which approach was planned or used. Given the absence of control participants and the absence of recorded analyses, the posted information cannot establish how a prespecified analysis population or missing-data method affected the results.
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 |
|---|---|---|---|
| Caphosol Arm | 0 | 1 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Caphosol Arm | 0 of 1 |
| Control Arm | 0 of 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 |
|---|---|
| Caphosol Arm | 0 of 1 |
| Control Arm | 0 of 0 |
The adverse-event entries report zero serious events among 1 participant at risk in the Caphosol arm, and zero other events among 1 participant at risk in that arm. The recorded death count is 0. For the control arm, the number at risk is 0, and the recorded counts for serious events, other events and deaths are also 0.
These are counts, not comparative safety estimates. Zero events in the Caphosol arm means that no events of those listed types were recorded among its one participant. It does not establish that events cannot occur or provide a precise estimate of event frequency. The control arm had no participants, so its zero counts do not describe observed safety experience with Biotene and cannot be compared with the Caphosol arm.
The registry lists no serious adverse-event terms. The available safety data are too limited to characterize comparative harms, and the record does not report further detail about adverse-event collection, definitions, follow-up completeness or attribution. The counts should be read strictly as the recorded event totals, not as evidence that either regimen is safer.
12. What the Result Does, and Does Not, Mean
The registry results establish that this Phase 3 randomized trial was terminated and had enrollment of 1. One participant was enrolled in the Caphosol arm, and none were enrolled in the control arm. Those details describe the amount of comparative information available; they do not answer whether Caphosol prevents oral mucositis of grade 2 or higher.
The intended endpoint was clinically focused: development of oral mucositis at a defined severity threshold in people receiving chemotherapy. However, an endpoint definition and a randomized design do not by themselves produce an interpretable treatment comparison. Such a comparison requires outcome information in both groups. Because the control arm has no participants, the effect of Caphosol relative to Biotene cannot be estimated from the posted results.
The findings do not show that the interventions are equivalent, that Caphosol has no effect, or that it prevents mucositis. They also do not establish a safety difference. The adverse-event entries record zero events for the one participant at risk in the Caphosol arm, while there were no control participants at risk. These observations are too limited to support broad conclusions about either intervention.
In general, a well-specified trial question can remain unanswered when recruitment or follow-up does not produce enough data. That is the statistical situation reflected in this record. The outcome timeframe is stated as 24 months, but additional detail about outcome completeness is not reported in the registry record. No inferential results are listed, so there is no p-value, confidence interval or comparative effect estimate to interpret.
The practical conclusion is about the evidence, not about treatment choice: this trial’s registry results do not resolve its prevention question. The reason for termination and the reasons for limited enrollment are not reported. Readers should distinguish the study’s objective from what its posted data can establish.
13. Why This Trial Matters Statistically
This trial illustrates a basic requirement of comparative research: both arms need participants with outcome information if the planned contrast is to be estimated. The study was randomized and intended to compare Caphosol with Biotene, but one participant enrolled in the Caphosol arm and none in the control arm. The design identifies the question the investigators sought to answer; the enrollment determines that the posted results cannot answer it.
The example also distinguishes a recorded outcome from an inferential result. The primary endpoint is defined as oral mucositis of grade 2 or higher, assessed using CTCAE 4.0, and its timeframe is listed as 24 months. Yet an endpoint definition is not itself evidence of a treatment effect. Without an evaluable comparison, neither the magnitude nor the precision of a contrast can be reported.
For biostatisticians, the record underscores why estimates should be read with their denominators and study context. A zero count in one arm may appear reassuring in isolation, but the Caphosol arm had only one participant at risk and the control arm had none. Those counts provide no basis for comparing event frequencies or generalizing to the intended population.
The record also makes clear that a sample-size illustration is different from the trial’s original design justification and from observed power. Statistical planning assumptions cannot substitute for collected data. When recruitment is limited, planned methods and potential analyses do not create information that the study did not generate.
Finally, the trial’s status is part of the interpretation. It is listed as terminated, but the reason is not reported in the registry record. Recognizing what is absent helps keep the conclusion appropriately bounded: this study documents an attempted randomized comparison, not a resolved clinical question.
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 important trial-specific limitation is the enrollment: one participant was assigned to the Caphosol arm and none to the control arm. This prevents the intended comparison with Biotene and leaves the primary clinical question unanswered. The limited enrollment also means that the experience of the enrolled participant cannot characterize outcomes for the intended population.
The registry lists the trial as terminated but does not report why it ended, why enrollment was limited, or whether recruitment was stopped for operational or other reasons. The record also does not report whether the enrolled participant completed the stated follow-up or had a complete primary endpoint assessment. These omissions limit interpretation of the outcome and the conduct of the study.
The trial was open-label, with masking listed as none. The registry does not identify who assessed mucositis or describe procedures intended to reduce bias in assessment. It specifies CTCAE 4.0 but does not provide further detail about assessment timing, repeated measurements or adjudication. The control-arm description is truncated, so its full regimen cannot be recovered from the registry entry.
No analysis is recorded, and the registry does not report a comparative estimate, confidence interval, p-value, missing-data approach, analysis population or multiplicity plan. The registry lists two outcome measures, while the primary outcome is not reduced to one comparison because it includes several categories or time points. The absence of category-level results adds to the uncertainty about outcome reporting.
Finally, the adverse-event counts arise from one participant in the Caphosol arm and no participants in the control arm. They are insufficient to characterize safety or compare arms. The original sample-size justification and reason for termination are not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Caphosol Study: Prevention of Oral Mucositis in Children, Adolescents and Young Adults Receiving Chemotherapy — NCT02539342.
- Registry record last updated 2020-03-09; status last verified 2020-02.
- Results first posted 2019-08-01.
- 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
NCT02539342 is a Phase 3 randomized, open-label, parallel-group study sponsored by Medical College of Wisconsin. It examined Caphosol rinse for prevention of oral mucositis in children, adolescents and young adults receiving chemotherapy, with Biotene as the control intervention. The primary outcome was development of oral mucositis of grade 2 or higher, assessed using CTCAE 4.0, with a stated timeframe of 24 months.
The study began in 2016-10, reached primary completion on 2017-03-02 and is listed as terminated. Enrollment was 1: one participant in the Caphosol arm and none in the control arm. The registry lists two outcome measures and no analyses. It does not report a comparative effect estimate or explain the termination. The posted results therefore do not establish whether the interventions differed in prevention of the primary outcome or in safety.