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 Clobetasol for Oral Graft-Versus-Host Disease (NCT01557517), a randomized phase 2 study. The primary outcome is Percentage of Participants With a Response as Assessed by the 273-point Oral Mucositis Rating Scale (OMRS) Who Received Topical Clobetasol 0.05% Oral Rinse for Oral Chronic Graft-versus-host-disease (cGVHD) During a Four-week Treatment Period.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01557517 |
| Phase | Phase 2 |
| Condition | Oral Chronic Graft vs Host Disease |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Double |
| Enrollment | 40 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Cancer Institute (NCI) |
| Results posted | Yes |
2. Clinical Question
Population
People with oral chronic graft-versus-host disease were the target population. The registry summary describes eligibility as age 12 years or older, with oral disease and no clobetasol allergy. The primary endpoint description says 18/19 participants in the clobetasol group were analyzed because one participant declined before treatment began.
Intervention
The active treatment was topical clobetasol 0.05% oral rinse. The arm description specifies 10cc held in the oral cavity for 2 minutes, 3 times a day. The primary endpoint is described as assessing response during a four-week treatment period, while the summary describes rinsing for 2 weeks; the registry record does not reconcile these durations.
Comparator
The comparator was a placebo oral rinse. Its arm description specifies 10cc for 2 minutes, 3 times a day. The registry record also names a placebo-first group during a clobetasol phase and a placebo group during a 2-week placebo phase. It does not explain how these phase labels relate to the listed parallel design.
Primary question
The primary endpoint concerns the percentage of participants meeting response criteria on the 273-point Oral Mucositis Rating Scale (OMRS) after active treatment. The record describes categories for progression, partial response, complete response and stable disease, but no single comparison, response counts, effect estimate or uncertainty interval is reported.
Oral chronic graft-versus-host disease can involve changes in the mouth’s tissues as well as symptoms such as pain, sensitivity and dryness, as described in the trial summary. The OMRS endpoint focuses on rated mucosal changes. It is therefore a measure of tissue findings rather than a direct measure of every symptom or of overall quality of life.
The registry lists a randomized, double-blind, parallel study and a placebo rinse comparator. However, its primary outcome record does not provide a numerical result for the comparison. It also describes multiple response categories, and the analysis record does not reduce them to a single comparison. Consequently, the clinical question is identifiable, but the posted record does not give the result needed to quantify how the groups differed on that question.
3. Trial Design
The registry identifies this as a phase 2, randomized, double-blind, parallel pilot study sponsored by the National Cancer Institute (NCI). It records enrollment of 40 and a completed status. The record gives a study start date of 2012-03-28 and a primary completion date of 2017-06-09. These are registry dates, not evidence about the timing of an individual participant’s treatment or follow-up.
The listed interventions were clobetasol oral rinse and placebo oral rinse. The clobetasol arm description specifies 10cc of clobetasol 0.05% for 2 minutes, 3 times a day; the placebo description gives the same volume, duration and frequency. The brief summary describes rinsing after meals for 2 weeks. In contrast, the primary endpoint title refers to a four-week treatment period and assessment at 4 weeks on active treatment. The registry record does not clarify the relationship between these durations.
There is also a design-description issue. Alongside the parallel-model designation, the adverse-event groups include “Placebo First (During Clobetasol Phase Only)” and a separate “Placebo (During 2-week Placebo Phase Only)” group. These labels suggest phase-specific reporting, but the record does not explain the sequence or how these groups map to the stated parallel allocation. The design should therefore be described using the registry’s classifications without assuming an unreported treatment sequence.
The summary describes screening with medical history, examination, laboratory testing and oral assessment, including a mouth tissue biopsy. It also describes follow-up testing and examination. The full schedule and details of follow-up after treatment are not reported in the registry record. The record likewise does not explain allocation procedures, concealment, or the operational details of masking.
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 | Clobetasol Followed by Assessment + 2 Weeks Clobetasol | Placebo Followed by Clobetasol |
|---|---|---|
| STARTED | 19 | 21 |
| COMPLETED | 17 | 17 |
| NOT COMPLETED | 2 | 4 |
Reasons for not completing this period:
| Reason | Clobetasol Followed by Assessment + 2 Weeks Clobetasol | Placebo Followed by Clobetasol |
|---|---|---|
| Death | 1 | 0 |
| Refused further treatment | 0 | 2 |
| Adverse Event | 0 | 2 |
| Pt declined before treatment started | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: 18/19 are analyzed in the Clobetasol arm/group because one participant declined participation before treatment started.
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 | Clobetasol | Placebo | Total |
|---|---|---|---|---|
| Participants analysed | 19 | 21 | 40 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 18 | 17 | 35 |
| Age, Categorical: >=65 years | Participants | 1 | 4 | 5 |
| Age, Continuous | years | 44.09 (SD 14.52) | 41.4 (SD 16.53) | 42.75 (SD 15.53) |
| Sex: Female, Male: Female | Participants | 6 | 11 | 17 |
| Sex: Female, Male: Male | Participants | 13 | 10 | 23 |
| Race/Ethnicity, Customized: Black or African American | Participants | 3 | 3 | 6 |
| Race/Ethnicity, Customized: White | Participants | 15 | 13 | 28 |
| Race/Ethnicity, Customized: Asian | Participants | 0 | 3 | 3 |
| Race/Ethnicity, Customized: Unknown | Participants | 1 | 0 | 1 |
| Race/Ethnicity, Customized: Multiple Race | Participants | 0 | 2 | 2 |
| Race/Ethnicity, Customized: Mexican, Puerto Rican, Cuban, Central or So. Amer. | Participants | 3 | 3 | 6 |
| Race/Ethnicity, Customized: Not meeting definition for Hispanic or Latino | Participants | 16 | 18 | 34 |
| Region of Enrollment: United States | Participants | 19 | 21 | 40 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Participants With a Response as Assessed by the 273-point Oral Mucositis Rating Scale (OMRS) Who Received Topical Clobetasol 0.05% Oral Rinse for Oral Chronic Graft-versus-host-disease (cGVHD) During a Four-week Treatment Period, measured over At 4 weeks on active treatment.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the percentage of participants with a response assessed using the 273-point Oral Mucositis Rating Scale (OMRS). The scale rates oral tissue changes from 0 to 3 relative to normal tissue: 0 means normal or no change, and 3 means severe change. The total score is the sum of the items, with a possible range of 0-273. Lower scores indicate more normal oral mucosa.
The endpoint is intended to capture changes in oral mucosal findings during treatment. A structured scale can make those findings more consistent than an unstructured description, and using a change-based response category can distinguish improvement, worsening and no qualifying change. These are general reasons for using a scored clinical endpoint; they do not establish how consistently the scale was applied in this trial.
The registry says there is no standard definition of response in this field and describes study-specific criteria: progression is tied to a 25% change in the initial score, partial response is defined by a decrease, complete response adds a score of 0 on the erythema and ulceration components, and stable disease meets neither progression nor response criteria. The wording of the progression and partial-response definitions is incomplete or unclear in the record, so the thresholds cannot be fully reconstructed from it.
The primary timeframe is given as 4 weeks on active treatment, while the summary describes a 2-week rinse course. No participant counts by response category, aggregate percentage, group contrast or uncertainty estimate is reported for the primary endpoint. Thus, the outcome definition is partly described, but the posted record does not permit a numerical assessment of the primary result.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies the trial’s primary outcome as response on the OMRS, but it does not report the response counts or percentages for the treatment groups. It also does not report a treatment contrast, confidence interval, p-value or other inferential result for that outcome. The primary outcome has several response categories or time points and is not reduced to a single comparison in the record. As a result, the direction, size and precision of the primary treatment difference cannot be described from the posted information.
This absence should not be mistaken for evidence that the groups were the same. A finding of no difference would require results that quantify the comparison and its uncertainty. The registry record does not provide those results. It is equally not possible to conclude from this record that one group had a higher response rate. No numerical primary result is available to support either statement.
The trial is identified as a pilot study, but the record does not report a formal sample-size justification or a statistical plan that would establish what precision or treatment difference the study was designed to detect. The enrollment figure alone does not show whether the study could estimate the primary effect precisely. Any planning illustration based on a posted observed effect would be a planning calculation, not a description of the trial’s observed power; in this case, the primary effect needed for such a calculation is not reported.
The registry lists nine outcome measures, including changes in pain, sensitivity and dryness, clobetasol concentrations and pharmacokinetic measures, and adverse events. Their listing does not show that results are available for each outcome. No analysis entries are reported. The adverse-event counts are available by the listed groups, but they are not a substitute for the missing primary efficacy comparison. They also use group labels that refer to different phases, so they should be read as recorded rather than combined into a single treatment contrast.
Several interpretive details remain uncertain: the relationship between the two-week rinse schedule and the four-week primary assessment, the meaning of the placebo-first phase labels alongside the parallel-design classification, and the complete response definitions. These are limitations in the registry description, not evidence about what occurred clinically. The posted record supports a description of the question and the measurement approach, but not a conclusion about the primary treatment effect.
9. Statistical Methods Explained
Statistical interpretation starts by distinguishing the outcome definition from the result. The OMRS is a score of oral tissue findings, and the primary outcome converts those findings into response categories. A result would ordinarily report how many participants in each relevant group met each category, along with a clearly defined comparison. The registry record does not report those primary response counts or a single comparison. It therefore does not support a numerical estimate of the treatment effect.
What does a response percentage represent?
In general, a response percentage describes the proportion of participants in an analysis group who satisfy a prespecified response definition. Its meaning depends on the numerator, denominator, response rules and assessment time. A percentage without those details can be difficult to interpret; a category-based endpoint can also lose information compared with analyzing the underlying score. These are general statistical considerations. For this trial, the response categories are described but the category counts and corresponding percentages are not reported in the registry record.
The record says the endpoint is the percentage of participants with a response, but also lists progression, partial response, complete response and stable disease. These categories do not automatically define one binary outcome unless the analysis specifies which categories count as response and how they are compared. The registry record does not provide a single reduced comparison. The response wording itself is also incomplete: the progression definition refers to a 25% change in the initial score, and the partial-response wording does not state the full threshold. Without a fully specified rule, reproducing a response classification from the description would not be possible.
Why does the scale’s direction matter?
The OMRS ranges from 0-273, and lower scores indicate more normal oral mucosa. That direction is important when interpreting a change: a lower score is not worse simply because the numerical value has decreased. The record gives score anchors from normal or no change to severe change and says the total is formed by summing the items. General measurement considerations include whether a scale captures change meaningfully and whether the scoring is applied consistently. The registry description does not report reliability assessments or validation results for this trial, so none can be inferred.
How should uncertainty be assessed?
In general, a confidence interval describes a range of values compatible with the observed data under the statistical method used. It communicates precision as well as the direction and magnitude of an estimate. A p-value, when reported, addresses compatibility with a specified null hypothesis under a model; it does not measure the size or clinical importance of an effect. Neither a confidence interval nor a p-value for the primary outcome is reported in the registry record. No uncertainty statement can therefore be attached to a primary comparison.
For a binary response outcome, estimates can be sensitive to the number of participants and events in each group. General methods may use different interval or test procedures, and the appropriate choice depends on the design and analysis plan. The registry record does not report the primary analysis method, model, test, or interval procedure. It would be inappropriate to assign one retrospectively based only on the endpoint title.
What does over-dispersion mean in general?
Over-dispersion occurs when the variability in data is greater than a statistical model expects. If a model assumes less variation than the data contain, uncertainty may be understated and statistical conclusions may be too confident. Allowing for extra variability can improve uncertainty estimates when the model and outcome call for it. This is a general concept; the registry record does not report an over-dispersion assessment or a model for the primary endpoint, and the binary response description alone does not establish that over-dispersion applies.
Planning calculations can illustrate how many participants per arm might be required under stated assumptions, but they are not observed trial results or observed power. The planning description names a two-sided significance level of 0.05, equal allocation, a normal approximation and effects defined as fractions of an observed effect. The registry record does not provide the primary observed effect needed to interpret such illustrations. It also does not report that these planning assumptions were the design assumptions used by the sponsor. A planning result should therefore not be read as proof that the trial was adequately powered.
What can the adverse-event counts tell us?
The adverse-event record reports counts of participants affected in the listed groups and the numbers at risk. Such descriptive counts can show what was recorded within each group or phase. They do not by themselves establish whether an event was caused by treatment, and comparisons can be hard to interpret when the groups represent different treatment phases or exposure periods. The registry record does not describe an inferential safety analysis, adjustment for exposure time, or a method for attributing events to treatment.
For any analysis, the target population and handling of incomplete observations also matter. The primary description notes that 18/19 were analyzed in the clobetasol arm because one participant declined before treatment started. The record does not state the corresponding analysis population for the comparator, the approach to missing outcome measurements, or whether participants were analyzed according to randomized assignment. Those omissions limit evaluation of both bias and precision.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists nine outcome measures, including the OMRS response endpoint, symptom ratings for pain, sensitivity and dryness, pharmacokinetic measures, and adverse events. Multiple outcomes can create multiple opportunities to observe apparently notable results. In general, a prespecified multiplicity strategy can help interpret a family of statistical tests, for example by controlling the chance of false-positive findings across a defined set of comparisons. The registry record does not report a multiplicity adjustment, a hierarchy of endpoints, or a statistical plan specifying which comparisons were primary or secondary.
The primary outcome description includes multiple response categories, and the record says the outcome has several categories or time points rather than reducing it to one comparison. It does not give a single primary contrast or the counts needed to assess categories separately. The analysis record lists no analyses. Therefore, the record does not show how multiple categories, time points or outcomes were handled, or whether any adjustment was planned.
Missing-data methods are not reported in the registry record. The primary endpoint description states that 18/19 participants in the clobetasol arm were analyzed because one participant declined participation before treatment started. It does not provide a corresponding comparator analysis population or explain whether any other endpoint measurements were missing. The record does not say how missing observations were handled, whether any imputation was used, or what population definition governed each analysis.
These omissions matter because complete-case results, imputed results and analyses based on randomized assignment can answer different questions and rely on different assumptions. This is a general methodological point, not a claim about what the investigators did. Without the analysis populations and missing-data procedures, the primary estimate and its susceptibility to missingness cannot be evaluated from the registry record.
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 |
|---|---|---|---|
| Clobetasol | 3 | 18 | 16.7% |
| Placebo First (During Clobetasol Phase Only) | 4 | 21 | 19.0% |
| Placebo (During 2-week Placebo Phase Only) | 0 | 21 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Clobetasol | 1 of 18 |
| Placebo First (During Clobetasol Phase Only) | 0 of 21 |
| Placebo (During 2-week Placebo Phase Only) | 0 of 21 |
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 |
|---|---|
| Clobetasol | 16 of 18 |
| Placebo First (During Clobetasol Phase Only) | 21 of 21 |
| Placebo (During 2-week Placebo Phase Only) | 6 of 21 |
Most frequent serious adverse event terms
| Event term | Organ system | Clobetasol | Placebo First (During Clobetasol Phase Only) | Placebo (During 2-week Placebo Phase Only) |
|---|---|---|---|---|
| Diarrhea | Gastrointestinal disorders | 2 of 18 | 0 of 21 | 0 of 21 |
| Hypertension | Vascular disorders | 1 of 18 | 1 of 21 | 0 of 21 |
| Lung infection | Infections and infestations | 1 of 18 | 1 of 21 | 0 of 21 |
| Cough | Respiratory, thoracic and mediastinal disorders | 1 of 18 | 0 of 21 | 0 of 21 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 1 of 18 | 0 of 21 | 0 of 21 |
| Enterocolitis infectious | Infections and infestations | 0 of 18 | 1 of 21 | 0 of 21 |
| Respiratory, thoracic and mediastinal disorders - Other, specify | Respiratory, thoracic and mediastinal disorders | 1 of 18 | 0 of 21 | 0 of 21 |
| Acute kidney injury | Renal and urinary disorders | 1 of 18 | 0 of 21 | 0 of 21 |
| Adrenal insufficiency | Endocrine disorders | 1 of 18 | 0 of 21 | 0 of 21 |
| Anemia | Blood and lymphatic system disorders | 1 of 18 | 0 of 21 | 0 of 21 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Clobetasol | Placebo First (During Clobetasol Phase Only) | Placebo (During 2-week Placebo Phase Only) |
|---|---|---|---|---|
| Adrenal insufficiency | Endocrine disorders | 10 of 18 | 11 of 21 | 0 of 21 |
| Lymphocyte count decreased | Investigations | 6 of 18 | 8 of 21 | 0 of 21 |
| Insomnia | Psychiatric disorders | 5 of 18 | 7 of 21 | 1 of 21 |
| Dysgeusia | Nervous system disorders | 3 of 18 | 8 of 21 | 0 of 21 |
| Mucosal infection | Infections and infestations | 5 of 18 | 6 of 21 | 0 of 21 |
| Hypophosphatemia | Metabolism and nutrition disorders | 1 of 18 | 7 of 21 | 0 of 21 |
| Hypertension | Vascular disorders | 4 of 18 | 2 of 21 | 0 of 21 |
| Mucositis oral | Gastrointestinal disorders | 3 of 18 | 2 of 21 | 0 of 21 |
| Nausea | Gastrointestinal disorders | 1 of 18 | 3 of 21 | 1 of 21 |
| Upper respiratory infection | Infections and infestations | 2 of 18 | 3 of 21 | 0 of 21 |
Serious events with the highest arm-level rate: diarrhea (11.1%), cough (5.6%), dyspnea (5.6%), respiratory, thoracic and mediastinal disorders - other, specify (5.6%), acute kidney injury (5.6%).
The registry reports serious and non-serious adverse-event counts separately for the listed groups. In the Clobetasol group, 3 participants were affected by serious adverse events among 18 at risk, and 16 were affected by other adverse events among 18 at risk; 1 death is reported. In “Placebo First (During Clobetasol Phase Only),” 4 participants were affected by serious adverse events among 21 at risk, and 21 were affected by other adverse events among 21 at risk; 0 deaths are reported. In “Placebo (During 2-week Placebo Phase Only),” 0 participants were affected by serious adverse events among 21 at risk, and 6 were affected by other adverse events among 21 at risk; 0 deaths are reported.
These are descriptive reports for the registry’s named groups, not evidence that treatment caused an event. The group labels distinguish phases, and the record does not explain how the phases relate to the listed parallel design. The counts should not be added across groups or treated as a single randomized comparison. Exposure time, attribution rules and an inferential safety analysis are not reported in the registry record.
The registry also lists serious-event terms, including diarrhea at 11.1%, and cough, dyspnea, respiratory, thoracic and mediastinal disorders - Other, specify, acute kidney injury, and adrenal insufficiency at 5.6% each. These entries describe reported terms; they do not establish comparative risk or causation.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a randomized, double-blind pilot study compared topical clobetasol oral rinse with placebo for oral chronic graft-versus-host disease. It describes the OMRS as the primary measure of mucosal response and gives a study-specific response framework. It also records adverse-event counts and several secondary outcomes. These details clarify what the study set out to evaluate and what kinds of measurements it included.
The record does not provide the primary response counts, a comparison between groups, or an estimate with uncertainty. It therefore does not show the magnitude or precision of any treatment difference on the primary endpoint. The lack of a posted comparison is not evidence that there was no difference, and it is not evidence that there was a benefit. Neither conclusion can be drawn from the information in the registry record.
The endpoint is based on oral tissue ratings, not solely on pain, dryness or sensitivity. Those symptoms are listed as separate secondary outcomes. Even a reported difference on the OMRS would answer a specific question about scored mucosal changes; it would not automatically establish an effect on every symptom, long-term outcomes, or patient experience. General interpretation requires matching each conclusion to the outcome that was measured.
The pilot designation and enrollment record do not, by themselves, establish how precise an estimate would be. The registry does not report the sample-size justification, primary statistical method, missing-data strategy or multiplicity plan. The description also leaves unresolved the difference between the 2-week treatment schedule in the summary and the four-week primary endpoint timeframe, as well as the phase-specific group labels alongside a parallel-design classification.
Accordingly, the record is useful for understanding the study question, intervention and outcome definitions, but it is insufficient for judging the primary comparative result. The adverse-event entries can be read as descriptive counts for the listed groups, with the same caution about phase labels and lack of causal attribution. A clinical conclusion about the primary response would require results and analysis details not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of why a clearly named endpoint is not enough to evaluate a clinical result. The registry identifies a 273-point oral mucosal scale, gives its direction and describes response categories. Yet without counts by category, a defined group contrast and an uncertainty estimate, the primary effect cannot be quantified. Statistical interpretation depends on both the measurement and the way results are reported.
The response definition also illustrates the importance of operational detail. The record says there is no standard response definition in this field and describes study-specific criteria, but parts of the wording do not fully specify the thresholds. If the rules for classifying participants are ambiguous, readers cannot confidently reproduce the outcome or know exactly which observations count as response. A fully specified definition is especially important when a continuous score is converted into categories.
The timing descriptions raise a related design issue. The summary describes a 2-week rinse course, while the primary endpoint refers to a four-week treatment period and assessment at 4 weeks on active treatment. The registry also labels the model as parallel while listing phase-specific placebo groups. These details matter because interpretation of an outcome depends on when it is measured and which treatment exposure the group represents.
Finally, safety counts and outcome listings should not be mistaken for a complete comparative analysis. Counts can describe recorded events, but phase labels, exposure periods and attribution methods affect what comparisons mean. For biostatisticians and clinical researchers, the key lesson is that transparent reporting of analysis populations, estimands, missing-data handling, multiplicity, and effect precision is needed to connect a trial question to a defensible conclusion.
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 limitation of the registry record for interpretation is that it does not report the primary response results. No response counts, group percentages, treatment contrast, confidence interval or p-value are available. The listed analysis count is 0, and the primary outcome is not reduced to one comparison. This prevents assessment of the size, direction and precision of the primary result.
The response criteria are not fully clear in the record. It describes progression as a 25% change in initial score and partial response as a decrease, but does not supply a complete, unambiguous threshold for each category. The relationship between response categories and the single percentage endpoint is also not stated. This makes it difficult to reconstruct how individual participants would be classified.
There is a discrepancy in timing descriptions: the summary says participants rinse for 2 weeks, while the primary outcome title refers to a four-week treatment period and the timeframe says 4 weeks on active treatment. The registry record does not reconcile these statements. It also lists a parallel model but names placebo-first and placebo-phase groups in the safety record. Their relationship to the stated design is not explained.
The registry does not report the primary statistical method, sample-size justification, missing-data approach, multiplicity strategy, or full analysis populations. It notes that 18/19 in the clobetasol group were analyzed because one participant declined before treatment started, but does not give the corresponding comparator analysis details. These reporting gaps limit evaluation of bias, uncertainty and reproducibility.
Safety information is descriptive, and the record does not report exposure-adjusted comparisons, event attribution methods or an inferential safety analysis. The recorded event counts should therefore not be interpreted as causal effects or as a complete comparison of treatment risks.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Clobetasol for Oral Graft-Versus-Host Disease — NCT01557517.
- Registry record last updated 2025-06-15; status last verified 2025-06.
- Results first posted 2019-01-03.
- 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
The registry describes NCT01557517, a completed phase 2 randomized, double-blind pilot study of clobetasol 0.05% oral rinse for oral chronic graft-versus-host disease. It records enrollment of 40 and identifies the National Cancer Institute (NCI) as sponsor. The listed comparator is placebo oral rinse.
The primary endpoint is the percentage of participants with an OMRS response, assessed at 4 weeks on active treatment. The scale ranges from 0-273, with lower scores indicating more normal oral mucosa. The record describes study-specific response categories but does not report a single primary comparison or its numerical results.
The registry lists nine outcome measures and adverse-event counts for three named groups. It reports 18/19 analyzed in the clobetasol arm because one participant declined before treatment began. The record does not report the primary statistical method, missing-data strategy, multiplicity approach or sample-size justification. It also does not reconcile the 2-week treatment description with the four-week endpoint timeframe or explain the phase labels alongside the parallel-design classification.