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 The Use of Oral Budesonide and Rectal Hydrocortisone for the Treatment of Active Ulcerative Colitis (NCT00805285), a na phase 2 study. The primary outcome is Simple Clinical Colitis Disease Activity (SCCAI).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00805285 |
| Phase | Phase 2 |
| Condition | Inflammatory Bowel Disease, Ulcerative Colitis |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Maryland, Baltimore |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients with active ulcerative colitis. The official title describes extensive ulcerative colitis, while the brief description refers to active ulcerative colitis. The registry reports enrollment of one patient, who withdrew; it does not report analyzable primary outcome data. Further participant characteristics are not reported in the registry record.
Intervention
The intervention was a combination of oral budesonide and rectal hydrocortisone. The registry identifies this as a drug intervention but does not report doses, schedules, treatment duration, or other administration details.
Comparator
There was no concurrent comparator group. Prednisone appears in the study rationale as a standard steroid against which the combination's side effects were of interest, but the registered design is single-group and does not compare the combination with prednisone.
Primary question
The primary outcome was Simple Clinical Colitis Disease Activity (SCCAI), measured at 0, 2, 4, 6, and 8 weeks. The study aimed to evaluate whether the combination improved symptoms. Because the sole patient withdrew and there was no SCCAI data to analyze, the registry cannot answer whether scores changed or whether clinical remission was reached.
The study also described an interest in whether the combination had fewer and less severe side effects than prednisone. That comparison is a stated rationale, not a result of a controlled comparison in this trial. The registry lists ACTH Stimulation Test, adverse events, and C Reactive Protein among secondary outcomes. It does not report analyzable results for these outcomes.
3. Trial Design
This was a phase 2, single-group study sponsored by University of Maryland, Baltimore. Its official title identifies it as a pilot study of oral budesonide and rectal hydrocortisone for extensive ulcerative colitis. The study began in 2008-10, had a primary completion date of 2009-10, and is listed as terminated. The registry reports an enrollment of one.
Allocation is listed as NA, masking as none, and the model as single-group. Accordingly, the design does not contain randomized treatment assignment, masking, or a separate control arm. The registry's single intervention is the combination of oral budesonide and rectal hydrocortisone. Although the study rationale mentions standard steroids, specifically prednisone, the registered trial does not include a prednisone arm. The registry record does not report a like-for-like comparison between treatment groups.
The primary outcome was SCCAI, with planned assessment at 0, 2, 4, 6, and 8 weeks. The registry describes scores from 0-19, with higher scores indicating greater disease severity, and says a score less than 3 is consistent with clinical remission. However, the only enrolled patient withdrew, and the primary outcome description states there was no data to analyze. The registry lists five outcome measures and zero analyses.
The record does not report a detailed protocol, treatment regimen, recruitment procedures, eligibility criteria beyond the broad disease description, or a sample-size justification. It also does not report interim analyses or a prespecified statistical analysis plan. Those omissions limit the ability to reconstruct how the pilot was intended to be conducted beyond the registered design and outcome descriptions.
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 | Combination Oral Budesonide and Rectal Hydrocortisone |
|---|---|
| STARTED | 1 |
| COMPLETED | 0 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Combination Oral Budesonide and Rectal Hydrocortisone |
|---|---|
| Lack of Efficacy | 1 |
Analysis population for the primary outcome, as defined in the registry: The only patient enrolled withdrew; there was no data to analyze.
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: The only enrolled patient was withdrawn secondary to disease worsening.
| Characteristic | Unit | Combination Oral Budesonide and Rectal Hydrocortisone |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | years | 45 (SD 0) |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 0 |
| Region of Enrollment: United States | participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Simple Clinical Colitis Disease Activity (SCCAI), measured over 0, 2, 4, 6, and 8 weeks.
| 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 endpoint was Simple Clinical Colitis Disease Activity (SCCAI), a clinical score intended to describe ulcerative colitis disease activity. The registry states that scores range from 0-19, that higher scores indicate increased disease severity, and that a score less than 3 is consistent with clinical remission. The scheduled assessments were at 0, 2, 4, 6, and 8 weeks.
As a general statistical concept, repeated clinical scores can describe how symptoms or disease activity change over time within a participant. They can also provide information about whether a participant crosses a clinically defined threshold. Such interpretation depends on having observed scores at relevant time points and on knowing how missing assessments are handled.
In this study, the endpoint cannot be summarized as a change, trajectory, or remission result. The only patient enrolled withdrew, and the registry explicitly says there was no data to analyze. No baseline or follow-up SCCAI values, endpoint estimate, variability measure, or uncertainty interval is reported. The stated threshold therefore describes how the score is interpreted in general, not an outcome reached by this study.
The registry record does not explain whether the intended primary analysis would have used a particular score, change from baseline, time-point comparison, or repeated-measures model. It also does not report an endpoint-specific analysis population. These details are not reported in the registry record, so the stated measurement schedule and score description should not be mistaken for an available efficacy result.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical fact is that enrollment was one and the only patient withdrew. For the primary endpoint, the registry states there was no data to analyze. Consequently, there is no reported SCCAI estimate, no observed change from baseline, and no basis for describing the course of disease activity during the scheduled assessment period.
This absence of outcome data is different from observing no change. A result of no change would require measured scores that could be compared. Here, the registry does not report such scores. It is also different from evidence that the combination did or did not affect symptoms: the study supplies no analyzable primary outcome from which to estimate an effect. The appropriate interpretation is that the primary clinical question remains unanswered by the posted results.
The record lists five outcome measures and zero analyses. It names ACTH Stimulation Test, adverse events, and C Reactive Protein among secondary outcomes, but it does not provide analyzable results for those outcomes. Thus, the available record does not support comparisons across outcome measures or a broader estimate of treatment response.
The registered design was single-group, with no masking and no concurrent comparator. Even if outcome measurements had been available, a single-group study would not establish a treatment contrast against prednisone or another control group. The stated interest in comparing side effects with standard steroids is part of the study rationale; it is not a comparative finding from this design. Differences in disease activity over time in an uncontrolled study could not, by themselves, separate treatment effects from other explanations.
The safety listing reports one participant at risk and one participant affected for serious adverse events, and one at risk and one affected for other adverse events. Worsening of ulcerative colitis is listed among serious adverse event terms at 100.0%. These figures describe the registry's safety entries; they do not provide a comparative risk estimate or establish that the intervention caused the event. The death count is not reported in the registry record.
Precision cannot be assessed for a treatment effect because no primary effect estimate or analysis is reported. There is no confidence interval or other uncertainty interval for SCCAI in the record. In general, a confidence interval expresses the range of effect values compatible with the data and the specified method; no such interval can be interpreted when the primary endpoint has no analyzed observations. The posted results are therefore descriptive of study completion and data availability, not evidence for or against a clinical effect.
The record's terminated status and withdrawal are relevant context, but the reason for termination is not reported in the registry record. No explanation of the withdrawal, recruitment experience, or feasibility findings is reported. These gaps prevent a more specific account of why the study ended or what operational conclusions the investigators may have drawn.
9. Statistical Methods Explained
The registered design determines what kinds of statistical conclusions would be possible. This was a single-group study with allocation listed as NA and masking listed as none. There was no randomized comparator arm. Its registered intervention was oral budesonide combined with rectal hydrocortisone. The registry therefore does not define a between-group treatment contrast, and the study cannot yield a randomized estimate comparing the combination with prednisone.
The primary endpoint, SCCAI, was scheduled for assessment at 0, 2, 4, 6, and 8 weeks. As a general statistical concept, repeated measurements can be used to describe within-participant patterns over time. Depending on the research question and data, an analysis might focus on one follow-up time, change from a starting value, or the overall pattern across visits. Different approaches answer different questions and rely on different assumptions. The registry does not report which approach was planned. More importantly, the only patient withdrew and the registry says there was no primary outcome data to analyze, so no such analysis is reported.
A descriptive estimate would ordinarily summarize observed values using a suitable measure of location and, where available, a measure of variability. An inferential analysis could also estimate uncertainty around an effect. General statistical methods cannot recover missing clinical measurements from a record that contains no analyzable primary endpoint observations. There is no reported SCCAI summary, standard error, confidence interval, or test result to interpret. The registry lists zero analyses.
As a general explanation, a confidence interval communicates how precisely an effect has been estimated under a chosen statistical method and its assumptions. It is not a substitute for an outcome estimate. In this study, the registry does not report a primary effect estimate or interval, so neither the direction nor precision of an SCCAI effect can be assessed. A p-value is likewise not reported; no conclusion about statistical significance can be drawn.
The registry gives a general description of planning illustrations: they use 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. Time-to-event illustrations are described as total event counts, while other illustrations are described as participants per arm. These are planning assumptions, not details of the registered single-group design. The registry does not report a trial-specific sample-size justification, and no primary effect estimate is available in the results from which to interpret an observed effect. Any planning illustration should not be read as observed power or as evidence that the study tested a treatment effect with a particular precision.
As a general concept, power describes the probability that a specified design and analysis will detect an effect of a defined size under stated assumptions. It is not a measure of whether a result is true, and a post hoc calculation based on observed results is not a replacement for an analysis. Planning quantities can help illustrate design requirements, but they do not change the fact that the enrolled participant withdrew and there were no primary endpoint data to analyze. The registry does not report a power target or the assumptions used to justify the enrollment target.
Over-dispersion is a general issue in count outcomes: it occurs when observed variability exceeds what a chosen count model expects, and ignoring it can make uncertainty appear too small. It does not provide a relevant correction for the absent SCCAI outcome data in this record. The registry does not report an over-dispersion assessment, a count-model analysis, or a model-based primary result.
Multiplicity refers, in general, to the challenge of interpreting several statistical tests or outcomes together. The registry lists five outcome measures and names three secondary outcome areas: ACTH Stimulation Test, adverse events, and C Reactive Protein. It does not report a multiplicity adjustment, a hierarchy among secondary outcomes, or analyses of those outcomes. Since zero analyses are listed, there are no reported outcome-specific test results to adjust or compare.
Finally, an analysis population specifies which participants are included in an analysis and how departures from the intended treatment or follow-up are handled. This record does not define an analysis population. With one enrolled patient who withdrew and no primary data for analysis, the record does not support reconstructing an intention-to-treat, per-protocol, or other analysis set. Such details are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists five outcome measures and names ACTH Stimulation Test, adverse events, and C Reactive Protein as secondary outcomes. The primary endpoint is SCCAI. It lists zero analyses, and the primary outcome description states that the only patient enrolled withdrew and there was no data to analyze. No analyzed result is reported for the primary or secondary outcomes.
As a general statistical principle, testing several outcomes can increase the chance of finding at least one apparently notable result by chance. A prespecified outcome hierarchy or multiplicity adjustment can help define how multiple results should be interpreted. The registry does not report a testing hierarchy, adjustment method, or analysis plan for multiple outcomes. With no analyses listed, there are no p-values or outcome-level conclusions in the record for which to assess multiplicity handling.
Missing data are central to interpreting this trial. The registry explicitly connects withdrawal of the only enrolled patient with the absence of primary outcome data. It does not report the timing or reason for withdrawal, the number of assessments completed before withdrawal, or any imputation or other missing-data procedure. It is therefore not possible to determine whether any partial observations existed or how an analysis would have treated them.
The record also does not define analysis populations or explain how the withdrawal would have affected inclusion in an analysis. The mechanism behind the missing endpoint data cannot be established from the registry entry. These are not merely technical omissions: without observed primary values, no endpoint estimate can be calculated or interpreted. Details about missing-data handling, analysis populations, and multiplicity are not reported in 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 |
|---|---|---|---|
| Combination Oral Budesonide and Rectal Hydrocortisone | 1 | 1 | 100.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Combination Oral Budesonide and Rectal Hydrocortisone | 1 of 1 |
Most frequent serious adverse event terms
| Event term | Organ system | Combination Oral Budesonide and Rectal Hydrocortisone |
|---|---|---|
| Worsening of ulcerative colitis | Gastrointestinal disorders | 1 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Combination Oral Budesonide and Rectal Hydrocortisone |
|---|---|---|
| Portal vein and superior mesenteric vein thrombosis | Vascular disorders | 1 of 1 |
Serious events with the highest arm-level rate: worsening of ulcerative colitis (100.0%).
The safety entry lists one participant at risk and one participant affected for serious adverse events. It also lists one participant at risk and one affected for other adverse events. Worsening of ulcerative colitis is included among the serious adverse event terms at 100.0%. The death count is not reported in the registry record.
These entries should be read as registry counts, not as a comparative safety analysis. The one-person enrollment means that the listed affected-participant counts describe the entire enrolled group; they do not establish how often events would occur in a larger population. The serious and other adverse event listings may refer to different categories of reporting, and the record does not explain the event-level details or any overlap. No comparison group is available to estimate whether event frequency differed from another treatment.
The listed term does not establish that the intervention caused worsening of ulcerative colitis. Safety events observed during a study and events caused by a treatment are distinct concepts. The registry does not report a causal assessment, event timing, severity details beyond the serious-event classification, or a comparison with prednisone. The figures therefore describe what was entered in the safety record, but they cannot support a comparative conclusion about risks or a general estimate of treatment safety.
12. What the Result Does, and Does Not, Mean
The result that can be stated is narrow: the registry reports one enrolled patient, withdrawal of that patient, and no primary endpoint data to analyze. It also lists zero analyses. No SCCAI effect estimate, change over time, or remission result is reported. The posted results do not answer whether oral budesonide combined with rectal hydrocortisone improved symptoms in this study.
This is not the same as a null finding. A null finding would refer to an analysis of collected data that did not show a detectable effect, or to an estimate near no difference. Here, no primary outcome estimate is available. Nor is withdrawal itself evidence that the combination was ineffective or poorly tolerated: the reason for withdrawal is not reported in the registry record.
The clinical question also included an interest in side effects compared with standard steroids, including prednisone. The registered trial has a single group and no concurrent prednisone comparison. The safety entries cannot establish whether the combination had fewer or less severe side effects than prednisone. They also cannot establish a causal relationship between the intervention and the serious adverse event term listed in the record.
The study should not be used to estimate treatment benefit, comparative safety, or the likely response of a broader patient population. It does not provide an analyzed clinical outcome or a precision estimate. Its registry entries do establish that it was a phase 2, single-group pilot study and that the planned primary measure was SCCAI at 0, 2, 4, 6, and 8 weeks. Those design details describe the intended question, not an answer to it.
More generally, an incomplete study can still clarify what was intended to be measured and what was entered in the registry. But the distinction between a planned endpoint and an observed result is essential. In this case, the threshold for remission is described, but no score is reported to assess against it. The record supports a conclusion about the absence of analyzable primary data, not a conclusion about whether the treatment works or does not work.
13. Why This Trial Matters Statistically
This trial matters statistically because it illustrates the difference between a study question and an estimable treatment effect. The registry describes a clinically relevant question about symptoms and side effects, and identifies a primary clinical score with scheduled follow-up. Yet an endpoint can only inform an effect estimate if usable measurements are available. Here, the sole enrolled patient withdrew, and the registry reports no primary data to analyze.
It also demonstrates why study design and analysis must be interpreted together. A single-group design has no concurrent control for distinguishing treatment-associated changes from other influences. The study's rationale mentions prednisone, but the registered design does not contain a prednisone group. Therefore, even a complete set of within-group measurements would not be a direct comparison of the combination with standard steroids.
The registry's status as terminated, the enrollment of one, and the absence of analyses are important when reading any safety or outcome entries. A listed adverse event does not automatically establish causation, and a count from one enrolled participant does not provide a stable estimate for a wider population. Likewise, a planned remission threshold cannot be treated as a remission result when no SCCAI values are reported.
For biostatisticians and clinical researchers, the record highlights the importance of separating feasibility and data availability from efficacy inference. A pilot-study label does not itself imply that an effect was estimated or that the study completed its intended measurements. The registry record does not explain the termination or give a sample-size justification, so conclusions about why the study ended or what it established operationally would be speculative. Statistically, its clearest contribution is the documentation of a planned single-group question that remained unanswered in the posted results.
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 immediate limitation is that only one patient was enrolled and that patient withdrew. The registry says there was no primary outcome data to analyze. This prevents any estimate of SCCAI, any description of its change across the scheduled visits, and any evaluation of the stated remission threshold.
The single-group, unmasked design also limits causal interpretation. There was no concurrent control group and no randomized comparison. Prednisone is mentioned in the study rationale, but the registered intervention does not include a prednisone arm. The study therefore cannot provide a direct comparative estimate of symptoms or side effects against prednisone.
Important study details are not reported in the registry record. These include the reason for withdrawal, treatment doses and schedule, detailed eligibility criteria, a trial-specific sample-size justification, a statistical analysis plan, interim analyses, and defined analysis populations. The record also does not explain how missing data would have been handled or whether any partial outcome measurements were collected.
The safety entries are limited to the posted participant counts and the listed serious adverse event term. The death count is not reported in the registry record. Event timing, a causal assessment, and detailed comparative safety information are not reported. The record lists five outcome measures and zero analyses, so it does not provide analyzable findings for secondary outcomes either.
The study is listed as terminated, but the reason for termination is not reported in the registry record. Taken together, these limitations mean the entry documents the intended study question and some design and safety information, but it does not supply an interpretable clinical effect estimate or comparative safety result.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: The Use of Oral Budesonide and Rectal Hydrocortisone for the Treatment of Active Ulcerative Colitis — NCT00805285.
- Registry record last updated 2019-12-04; status last verified 2019-11.
- Results first posted 2013-05-13.
- 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
NCT00805285 is a phase 2, single-group pilot study of oral budesonide combined with rectal hydrocortisone for active ulcerative colitis. It was sponsored by University of Maryland, Baltimore, began in 2008-10, had a primary completion date of 2009-10, and is listed as terminated. The design was unmasked, with allocation listed as NA.
The primary outcome was SCCAI, scheduled at 0, 2, 4, 6, and 8 weeks. The registry describes higher scores as greater disease severity and a score less than 3 as consistent with clinical remission. One patient enrolled and withdrew; the registry states there was no primary outcome data to analyze. Five outcome measures and zero analyses are listed.
The record includes secondary outcomes of ACTH Stimulation Test, adverse events, and C Reactive Protein, as well as safety entries for the enrolled group. It does not report an analyzable treatment effect, a prednisone comparison, or the reason for withdrawal or termination.