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 Assessing the Safety/Efficacy of Asacol® Given Every 12 Hours to Children and Adolescents With Active Ulcerative Colitis (NCT00713310), a randomized phase 3 study. The primary outcome is Treatment Success PUCAI (Pediatric Ulcerative Colitis Activity Index), mITT/Modified Intent to Treat Population.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00713310 |
| Phase | Phase 3 |
| Condition | Ulcerative Colitis |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Triple |
| Enrollment | 83 |
| Arms in the primary outcome | 2 |
| Lead sponsor | Warner Chilcott |
| Results posted | Yes |
2. Clinical Question
Population
Children and adolescents with mildly-to-moderately active ulcerative colitis. The primary analysis population is described as the modified intent-to-treat population: randomized participants who took at least one dose of study medication.
Intervention
High-dose Asacol, given as 400 mg delayed-release tablets every 12 hours for 6 weeks. The dose range was 2.0 to 4.8 g/day, dependent on body weight.
Comparator
Low-dose Asacol, also given as 400 mg delayed-release tablets every 12 hours for 6 weeks. The dose range was 1.2 to 2.4 g/day, dependent on body weight.
Primary question
Among participants in the modified intent-to-treat population, what proportion met the registry’s PUCAI treatment-success definition at 6 weeks, and how did the posted success rates compare between the high-dose and low-dose groups?
The comparison is between two dose ranges of the same named study drug, not between Asacol and a placebo or an untreated group. The registry describes treatment success using the Pediatric Ulcerative Colitis Activity Index (PUCAI), with assessments at baseline and 6 weeks. The main statistical contrast is the difference in the percentage of participants meeting that success definition in each dose group.
This is a question about the observed endpoint and the precision of the dose-group comparison. It is not, by itself, a complete account of all potential benefits or harms, and the registry record does not report every detail that would be needed to assess the trial’s conduct or analysis plan.
3. Trial Design
The registry identifies this as a Phase 3, randomized, triple-masked, parallel-group study. Participants were assigned to a low-dose or high-dose Asacol group. Both groups used 400 mg delayed-release tablets administered every 12 hours for 6 weeks, with the daily dose dependent on body weight. The registry lists enrollment as 83 and marks the study completed.
The primary outcome is a binary treatment-success measure: each participant either met the specified PUCAI success definition or did not. The posted primary analysis is for the modified intent-to-treat population, defined in the record as randomized participants who took at least one dose of study medication. The registry reports 41 participants in each group for the primary outcome.
The trial began in 2008-12, and primary completion was recorded as 2011-03. These dates describe the registry record; they do not establish how long each participant was followed beyond the stated 6-week treatment period. Further follow-up duration is not reported in the registry record.
The registry names Warner Chilcott as sponsor and identifies ulcerative colitis as the condition. It does not report the details of sequence generation, allocation concealment, the practical masking procedures, eligibility criteria, or the planned sample-size justification. The design labels establish the broad structure of the trial but do not answer those more specific methodological questions.
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 | Low-Dose | High-Dose |
|---|---|---|
| STARTED | 41 | 42 |
| mITT Population | 41 | 41 |
| COMPLETED | 36 | 36 |
| NOT COMPLETED | 5 | 6 |
Reasons for not completing this period:
| Reason | Low-Dose | High-Dose |
|---|---|---|
| Adverse Event | 5 | 2 |
| Lack of Efficacy | 0 | 2 |
| Withdrawal by Subject | 0 | 2 |
Analysis population for the primary outcome, as defined in the registry: miTT includes subjects who were randomized and took at least one dose of study medication
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 | Low-Dose | High-Dose | Total |
|---|---|---|---|---|
| Participants analysed | 41 | 42 | 83 | |
| Age Continuous | years | 13.0 (SD 3.2) | 12.8 (SD 3.0) | 12.9 (SD 3.1) |
| Age, Customized: 5-8 years | participants | 4 | 4 | 8 |
| Age, Customized: 9-17 years | participants | 37 | 38 | 75 |
| Sex: Female, Male: Female | Participants | 22 | 23 | 45 |
| Sex: Female, Male: Male | Participants | 19 | 19 | 38 |
| Region of Enrollment: United States | participants | 26 | 23 | 49 |
| Region of Enrollment: Canada | participants | 0 | 4 | 4 |
| Region of Enrollment: Poland | participants | 9 | 10 | 19 |
| Region of Enrollment: Romania | participants | 2 | 3 | 5 |
| Region of Enrollment: Croatia | participants | 4 | 2 | 6 |
6. Primary Endpoint
The registry lists the primary outcome as Treatment Success PUCAI (Pediatric Ulcerative Colitis Activity Index), mITT/Modified Intent to Treat Population, measured over Baseline and 6 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | % participants with treatment success |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the percentage of participants with treatment success on the PUCAI in the modified intent-to-treat population, assessed from baseline to 6 weeks. The PUCAI is a score from 0 to 85 incorporating abdominal pain, rectal bleeding, stool consistency, bowel-movement frequency, nocturnal bowel movements, and activity level. The registry describes remission as a score below 10, with ranges for mild, moderate, and severe activity.
For this endpoint, success at week 6 was defined in either of two ways: a week-6 score below 10, described as complete success; or a reduction of at least 20 points from baseline to week 6 with a week-6 score of at least 10, described as partial success. Thus, the endpoint counts participants meeting either criterion, rather than measuring the average change in score.
A binary endpoint can provide an interpretable summary of how many participants meet a prespecified clinical-state or improvement rule. It also discards some detail: participants just inside and outside the success definition are classified differently even if their scores are close. The registry does not report a rationale for selecting this threshold or information about how the success definition was chosen.
7. Results Posted in the Registry
- High Dose: 56.1% (23/41) versus 56.1% (23/41) on control; risk difference 0.0 percentage points (95% CI -21.5 to 21.5), risk ratio 1.00 (0.68–1.47); unadjusted two-proportion z-test p = 1.000.
Risk difference: High Dose
95% CI: -21.5 to 21.5 percentage points
Recalculated from the posted summary statistics
8. Secondary Efficacy Endpoints
The registry also posts results for secondary outcomes. For each outcome below, the endpoint type, the test applied to the posted summary statistics, the result and its interpretation are given in turn. Secondary endpoints are usually not adjusted for multiple testing, so they support rather than confirm the primary finding.
| Secondary outcome | Type | Effect measure | Estimate (95% CI) | Planning size, 80% power |
|---|---|---|---|---|
| Treatment Success PUCAI Amended Endpoint (5 Point Scale Abdominal Pain), mITT | Binary Endpoint | Risk difference | +2.4 pts (-19.0 to 23.8) | 6,455 per arm |
Treatment Success PUCAI Amended Endpoint (5 Point Scale Abdominal Pain), mITT
- Endpoint type: Binary: the percentage of participants with the event (counts rebuilt from the posted percentages and group sizes).
- Test: Unadjusted two-proportion z-test, with a Wald 95% confidence interval for the risk difference.
- Result:
- High Dose: 58.5% (24/41) versus 56.1% (23/41) on control; risk difference 2.4 percentage points (95% CI -19.0 to 23.8), risk ratio 1.04 (0.72–1.52); unadjusted two-proportion z-test p = 0.823.
- Interpretation:
- The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between High Dose and the control. The interval gives the range of effects that remain compatible with the data.
The registry posts one secondary treatment-success endpoint, using an amended PUCAI definition with a 5-point abdominal-pain scale. Its reported comparison follows the same broad pattern as the primary result: the group percentages are close, the estimated difference is small, and the confidence interval spans zero and extends in both directions. This adds context to the primary endpoint but does not resolve its uncertainty.
The secondary outcome is related to the primary outcome because both classify treatment success using the PUCAI at week 6. The amended definition changes the abdominal-pain scale, so the outcome is not an entirely independent test of a different clinical construct. Similar patterns across these related definitions can be informative about sensitivity to the endpoint specification, but should not be mistaken for independent replication.
The registry reports an unadjusted secondary p-value and a sponsor-reported analysis. It does not report a multiplicity adjustment, a testing hierarchy, or whether the secondary analysis was prespecified in a way that would support confirmatory interpretation. In general, when multiple outcomes or analyses are examined, unadjusted p-values do not account for the increased opportunity for chance findings. This matters even when the posted results are not especially striking: secondary findings generally carry less evidential weight than a clearly prespecified primary analysis unless the statistical plan establishes otherwise.
The secondary endpoint’s sample-size illustrations are planning exercises based on its observed effect, not evidence about the trial’s achieved power. They should not be read as changing the interpretation of the trial results or as proof that a future study must use those group sizes.
Secondary outcomes registered for this trial, for reference (only those above were recalculated):
- Treatment Success PUCAI Amended Endpoint (5 Point Scale Abdominal Pain), mITT
9. Statistical Interpretation
The posted primary success rates are 56.1% for low dose and 55.0% for high dose. The registry’s reported analysis gives a high-dose minus low-dose success-rate difference of -1.1 percentage points, with a 95% confidence interval from -22.7 to 20.5 and p = 0.924. This estimate is close to zero, while the interval is wide enough to include differences in either direction.
The posted unadjusted recalculation gives a risk difference of 0.0 and p = 1.0, based on 23 successes among 41 participants in each group. This is not the same analysis as the registry’s reported Cochran-Mantel-Haenszel comparison. The registry does not explain why the displayed group percentages and the two analyses differ slightly, so they should be read as distinct posted results rather than treated as interchangeable calculations.
The registry’s secondary amended-endpoint analysis reports success rates of 56.1% for low dose and 57.5% for high dose. Its reported high-dose minus low-dose difference is 1.4 percentage points, with a 95% confidence interval from -20.2 to 23.0 and p = 0.819. The unadjusted recalculation for that endpoint has p = 0.823. As with the primary comparison, the interval includes zero and allows for differences in either direction.
These results describe uncertainty around the observed dose-group comparisons. They do not establish that the dose groups are equivalent: an equivalence conclusion would require an appropriate design and a prespecified margin, neither of which is reported in the registry record. Nor do the results establish that either group’s outcome was caused by treatment, because the posted figures alone do not provide the full context needed for that causal assessment.
The high-dose versus low-dose contrast is the central comparison. The similar posted success percentages and confidence intervals spanning zero mean the record does not show a precise separation between the groups on these endpoints. The registry’s p-values are not evidence that the true difference is exactly zero; p-values and confidence intervals instead describe how the observed data relate to a statistical model and a range of possible differences.
10. Statistical Methods Explained
What does a treatment-success percentage represent?
For a binary endpoint, each participant is classified as having met or not met the success definition. The percentage is the share classified as successful in a group. It summarizes the frequency of the endpoint, but it does not show the distribution of individual PUCAI scores or how far participants were from the success threshold. The primary result therefore concerns the chance of meeting the stated rule, not the average score change.
What is a risk difference?
A risk difference subtracts the event proportion in one group from the proportion in the comparison group. For the registry’s reported contrast, the direction is high dose minus low dose. A positive value means the posted success proportion is higher in the high-dose group; a negative value means it is lower. A value near zero indicates little observed separation in the two proportions, but does not by itself demonstrate that the underlying group outcomes are identical.
The registry also posts recalculated unadjusted comparisons from group summaries. These are simple comparisons of the observed group proportions and do not necessarily reproduce a sponsor-reported analysis that uses a named statistical method. The primary recalculation reports a risk difference of 0.0 and p = 1.0; the registry analysis reports a difference of -1.1 percentage points and p = 0.924. Those outputs answer closely related questions but should be kept distinct because the registry does not provide enough detail to explain the small differences between them.
What does the Cochran-Mantel-Haenszel label tell us?
The registry identifies the reported analysis as Cochran-Mantel-Haenszel and labels the parameter as the high-dose minus low-dose difference in success rates. In general, this method can combine comparisons across strata while accounting for the stratification used in an analysis. The registry record does not report the strata, any covariates, or further implementation details. It is therefore not possible to determine from the record whether or how stratification affected these estimates.
How should a p-value be read?
In general, a p-value measures how unusual data at least as incompatible with a specified null model would be, assuming that model and the analysis assumptions hold. It is not the probability that the null hypothesis is true, and it does not measure the size or importance of a treatment difference. The registry’s reported primary p-value is 0.924; the unadjusted recalculation is 1.0. Both are consistent with the observed data not providing a clear statistical separation under their respective analyses, but neither proves equality.
The secondary endpoint also has a reported p-value and an unadjusted recalculation. Because the registry does not report a multiplicity plan for the secondary outcomes, those p-values should be treated as exploratory rather than as independent confirmatory evidence. Repeated testing across outcomes can increase the chance of finding an apparently notable result by chance if no adjustment or testing hierarchy is used.
Why do confidence intervals matter?
In general, a confidence interval conveys the range of effect estimates compatible with the observed data under the method’s assumptions. A narrower interval indicates greater precision; a wider interval indicates less precision. For a difference in success rates, an interval crossing zero includes the possibility of no difference as well as differences in either direction. It also shows which magnitudes remain compatible with the data, which a p-value alone does not communicate.
The primary registry interval runs from -22.7 to 20.5 percentage points. The secondary registry interval runs from -20.2 to 23.0 percentage points. Both include zero and extend materially in both directions. Their breadth is important: the data do not pin down the size or direction of a dose-group difference with high precision.
What are the planning illustrations for?
The posted planning illustrations use the observed effect from the amended secondary endpoint, with assumptions of a two-sided significance level of 0.05, equal allocation, and a normal approximation. They list participants per arm for 80% power and participants per arm for 90% power when planning for 100%, 75%, or 50% of the observed effect. For example, planning for 75% of the observed effect gives 11,497 participants per arm for 80% power and 15,390 participants per arm for 90% power.
These are hypothetical planning calculations based on the observed effect, not the trial’s own sample-size justification and not evidence of the trial’s observed power. The registry does not report the trial’s original sample-size assumptions or justification. Planning estimates can also be highly sensitive to the effect size used, so the values should be interpreted as illustrations under the stated assumptions, not as a prescription for a future study.
11. Confidence Intervals and Precision
The registry’s primary 95% confidence interval for the high-dose minus low-dose difference extends from -22.7 to 20.5 percentage points. Its center, -1.1 percentage points, is close to no observed separation, but the interval is broad. It includes lower success in the high-dose group as well as higher success in that group. The data therefore do not identify the direction of a possible difference with precision.
For the amended secondary endpoint, the reported 95% confidence interval extends from -20.2 to 23.0 percentage points around a reported difference of 1.4 percentage points. This interval also crosses zero and includes a broad range of possible group differences. The primary and secondary intervals should not be treated as proof that the dose groups are equivalent; the registry does not report an equivalence margin or an equivalence-testing plan.
In general, a confidence interval is an estimate of uncertainty around an effect, conditional on the analysis method and its assumptions. The interval is not a range containing a known fixed proportion of individual participant outcomes, nor does it assign a probability to the true effect after the data have been observed. Its practical message is that the estimated group differences are imprecise relative to the range of effects still compatible with the reported analyses.
The recalculated primary result is based on equal posted success counts, while the registry’s analysis reports a small nonzero difference. The record does not give enough methodological detail to resolve that distinction. Reading the registry’s reported analysis alongside the unadjusted recalculation makes the analytic contrast visible without implying that either interval or point estimate is more precise than the posted information supports.
12. Analyses Reported by the Sponsor
| Method | Estimate | 95% CI | P-value |
|---|---|---|---|
| Cochran-Mantel-Haenszel | High-Low Dose Difference Success Rates -1.1 | -22.7 to 20.5 | 0.924 |
The registry reports the primary comparison using the Cochran-Mantel-Haenszel method. It gives a high-dose minus low-dose success-rate difference of -1.1 percentage points, a 95% confidence interval from -22.7 to 20.5, and p = 0.924. The unadjusted recalculation from the posted counts gives a risk difference of 0.0 and p = 1.0. For the amended secondary endpoint, the registry reports a difference of 1.4 percentage points, a 95% confidence interval from -20.2 to 23.0, and p = 0.819; the unadjusted recalculation gives p = 0.823.
These comparisons are close in broad interpretation: neither gives a precise indication of a difference between dose groups, and both reported intervals include zero. Their estimates and p-values are not identical. The registry names the sponsor analysis method but does not report enough detail to reproduce it or explain the small numerical differences from the unadjusted recalculations. The recalculations are not substitutes for the registry’s reported analysis, and the registry record does not flag a discrepancy.
13. Multiplicity, Missing Data and Analysis Populations
The registry record reports a primary endpoint and one posted secondary endpoint, but a full multiplicity strategy is not reported in the registry record. It is not reported whether there was a prespecified hierarchy for testing outcomes, whether any adjustment was planned, or how the sponsor intended to interpret the secondary analysis alongside the primary analysis. Without that information, the secondary p-value is best treated as an unadjusted result rather than as a separate confirmatory finding.
The record defines the primary analysis population as the modified intent-to-treat population: randomized participants who took at least one dose of study medication. This excludes randomized participants who did not take a dose by definition, but the registry does not report how many such participants there were or how exclusions were handled in detail. The phrase modified intent-to-treat should therefore be interpreted using the record’s stated definition, rather than assumed to mean that every randomized participant was included.
Missing-data methods are not reported in the registry record. It is not stated whether missing week-6 assessments were treated as failures, imputed, analyzed using another method, or handled in a different way. The record also does not report the amount or pattern of missing outcome data. For a binary endpoint, the approach to missing assessments can affect the estimated success percentage, so this omission limits assessment of robustness.
The registry does not provide the complete statistical analysis plan, sample-size justification, or details of interim analyses. Those elements should not be inferred from the final posted outcome statistics. The planning illustration associated with the secondary endpoint is explicitly an illustration based on the observed effect, not the trial’s own design justification.
14. 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 |
|---|---|---|---|
| Low Dose | 5 | 41 | 12.2% |
| High Dose | 2 | 41 | 4.9% |
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 |
|---|---|
| Low Dose | 20 of 50 |
| High Dose | 15 of 41 |
Most frequent serious adverse event terms
| Event term | Organ system | Low Dose | High Dose |
|---|---|---|---|
| Colitis Ulcerative | Gastrointestinal disorders | 1 of 41 | 1 of 41 |
| Sinusitis | Infections and infestations | 1 of 41 | 0 of 41 |
| Abdominal Pain | Gastrointestinal disorders | 1 of 41 | 0 of 41 |
| Body Mass Index Decreased | Investigations | 1 of 41 | 0 of 41 |
| Adenovirus Infection | Infections and infestations | 1 of 41 | 0 of 41 |
| Diarrhoea Haemorrhagic | Gastrointestinal disorders | 1 of 41 | 0 of 41 |
| Cholangitis Sclerosing | Hepatobiliary disorders | 1 of 41 | 0 of 41 |
| Pancreatitis | Gastrointestinal disorders | 1 of 41 | 0 of 41 |
| Anaemia | Blood and lymphatic system disorders | 0 of 41 | 1 of 41 |
| Syncope | Nervous system disorders | 0 of 41 | 1 of 41 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Low Dose | High Dose |
|---|---|---|---|
| Nasopharyngitis | Infections and infestations | 4 of 50 | 4 of 41 |
| Colitis Ulcerative | Gastrointestinal disorders | 5 of 50 | 2 of 41 |
| Headache | Nervous system disorders | 4 of 50 | 2 of 41 |
| Fatigue | General disorders | 1 of 50 | 3 of 41 |
| Dizziness | Nervous system disorders | 3 of 50 | 1 of 41 |
| Pyrexia | General disorders | 0 of 50 | 3 of 41 |
| Sinusitis | Infections and infestations | 3 of 50 | 0 of 41 |
Serious events with the highest arm-level rate: sinusitis (2.4%), abdominal pain (2.4%), body mass index decreased (2.4%), colitis ulcerative (2.4%), adenovirus infection (2.4%).
The registry lists serious adverse events for 5 participants in the low-dose group, with 41 at risk, and for 2 participants in the high-dose group, also with 41 at risk. The listed serious adverse-event terms each have a reported value of 2.4%: sinusitis, abdominal pain, body mass index decreased, ulcerative colitis, adenovirus infection, and haemorrhagic diarrhoea. These are posted event figures; the record does not provide enough detail to determine whether individual participants experienced more than one event or how the listed terms relate to the group-level counts.
For other adverse events, the registry lists 20 affected among 50 at risk for low dose and 15 affected among 41 at risk for high dose. The low-dose denominator differs from the primary outcome group size, and the registry does not explain the safety denominator in the record. These adverse-event figures should not be directly compared as if all denominators and reporting rules were necessarily the same.
Deaths are not reported in the registry record. The event counts alone do not establish that an event was caused by treatment or permit a complete safety comparison. The registry figures should be read as descriptive reporting, alongside the stated at-risk denominators and the limitations of the available detail.
15. What the Result Does, and Does Not, Mean
The posted results show similar treatment-success percentages in the low-dose and high-dose groups on the primary PUCAI endpoint, with the sponsor-reported difference close to zero. The 95% confidence interval is broad and includes both negative and positive differences. The unadjusted recalculation also gives a risk difference of 0.0. Together, these statistics describe an observed comparison that does not precisely distinguish the two dose groups on this endpoint.
That finding is not the same as demonstrating that the dose groups are equivalent or that their effects are identical. The confidence interval leaves a range of possible differences compatible with the registry analysis, and the record does not report an equivalence margin or a design intended to establish equivalence. A p-value near one does not mean there is a high probability that the treatments are the same.
The results also do not establish that either dose caused the observed success rate. The randomized design is relevant to causal comparison, but a complete interpretation also depends on trial conduct, missing outcomes, the analysis plan, and the details of participant follow-up. Several of those details are not reported in the registry record.
The secondary endpoint yields a broadly similar pattern, but it is related to the primary outcome and its multiplicity handling is not reported. It therefore provides context, not a fully independent confirmation. The safety counts are descriptive and have their own denominator and reporting limitations.
In general, statistical estimates are most useful when read with their uncertainty, outcome definitions, and analysis population. These registry results describe the posted comparisons for this trial; they do not provide a complete basis for decisions about an individual’s care or for conclusions beyond the studied population and reported outcomes.
16. Why This Trial Matters Statistically
This trial illustrates why a difference between observed percentages should be considered together with its uncertainty. The primary group percentages are close, but the reported confidence interval is wide enough to include potentially meaningful differences in either direction. A small point estimate is not automatically precise evidence of a small underlying effect.
It also shows that statistical results depend on the analysis being reported. The registry’s Cochran-Mantel-Haenszel analysis and the unadjusted recalculation produce slightly different estimates and p-values. These are not necessarily contradictory: methods can target related comparisons while using different analytical details. Because the registry does not provide the full implementation details, readers should avoid attributing the difference to a particular adjustment or assumption.
The endpoint definition matters as well. Treatment success combines a threshold for a low week-6 score with a separate improvement rule for participants whose score remains at or above 10. That binary rule makes the result interpretable as a success proportion, while concealing variation in score changes and closeness to the threshold. The amended secondary definition demonstrates that even a related endpoint specification should be recognized as a separate analysis.
Finally, planning calculations based on the observed secondary effect are not observed power and are not the original design rationale. Separating observed results, uncertainty estimates, and planning illustrations is essential for a sound statistical reading of clinical-trial evidence.
17. 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.
- Participant counts were reconstructed from posted percentages and arm sizes, so small rounding differences are possible.
The registry record does not report the full protocol or statistical analysis plan. Details of randomization procedures, allocation concealment, masking implementation, stratification, and the sample-size justification are not reported in the registry record. Although the study is labeled randomized and triple-masked, those labels do not supply the missing operational detail.
Missing outcome data and their handling are not reported in the registry record. The primary analysis population excludes randomized participants who did not take at least one dose, according to its stated definition, but the record does not give further details about exclusions or the sensitivity of results to analysis-population choices. This limits evaluation of possible bias from missing data or deviations from assigned treatment.
The safety record uses different at-risk denominators for other adverse events than those posted for the primary outcome, and the reason is not reported in the registry record. Deaths are not reported in the registry record; that wording should not be interpreted as evidence that no deaths occurred. The serious-event listing also does not provide enough information to reconstruct participant-level overlap among events.
The primary and secondary confidence intervals are wide, and the secondary endpoint is related to the primary definition. Multiplicity handling is not reported, limiting the weight that can be placed on secondary analyses. The registry also does not report follow-up beyond the stated 6-week endpoint timeframe. These limitations restrict how fully the posted results can be interpreted, without changing the descriptive values recorded for the trial.
18. Related Tutorials
19. Related Statistical Calculators
20. Sources
- ClinicalTrials.gov: Assessing the Safety/Efficacy of Asacol® Given Every 12 Hours to Children and Adolescents With Active Ulcerative Colitis — NCT00713310.
- Registry record last updated 2012-04-05; status last verified 2012-04.
- Results first posted 2012-04-04.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
21. Record Summary
This completed Phase 3 study was randomized, triple-masked, and parallel-group. It compared weight-dependent low-dose and high-dose Asacol administered as 400 mg delayed-release tablets every 12 hours for 6 weeks in children and adolescents with mildly-to-moderately active ulcerative colitis. Enrollment was recorded as 83.
The primary endpoint was PUCAI treatment success at 6 weeks in the modified intent-to-treat population. The registry reports success rates of 56.1% for low dose and 55.0% for high dose. Its reported difference was -1.1 percentage points, with a 95% confidence interval from -22.7 to 20.5 and p = 0.924. The confidence interval is broad and includes zero.
The record also posts one related secondary treatment-success endpoint and descriptive adverse-event figures. Details of missing-data handling, multiplicity, and the full statistical plan are not reported in the registry record.