A Study to Evaluate the Impact of Adalimumab on Quality of Life, Health Care Utilization and Costs of Ulcerative Colitis Subjects in the Usual Clinical Practice Setting: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Study to Evaluate the Impact of Adalimumab on Quality of Life, Health Care Utilization and Costs of Ulcerative Colitis Subjects in the Usual Clinical Practice Setting (NCT01550965), a na phase 3 study. The primary outcome is Mean Change From Baseline in Short Inflammatory Bowel Disease Questionnaire (SIBDQ): Total Score.

2. Clinical Question

3. Trial Design

This was an open-label, multicenter, Phase 3 study of adalimumab among people with ulcerative colitis. The study title and description place the work in the usual clinical practice setting and identify quality of life, health care utilization and costs as areas of interest. The registry record lists 463 participants as enrolled. The study began in 2012-05, reached primary completion in 2015-04, and is listed as completed.

The design is recorded as single-group, with allocation not applicable and masking absent. Participants received adalimumab; there is no concurrent control arm in the registry record. As a result, the design can describe participants’ outcomes during the study, but it does not create a randomized contrast that separates treatment-associated changes from other changes over time. The record does not report a protocol rationale for selecting this design.

The listed dosing began with 160 mg at the Baseline Visit, then 80 mg at the Week 2 Visit, and 40 mg every other week beginning at Week 4. Non-responders were to discontinue treatment at Week 8. The registry’s arm description is incomplete after its mention of possible dose escalation, so the remaining dosing details are not reported in the registry record.

The primary endpoint is mean change in total SIBDQ score between Week 0 (baseline) and Week 26. The listed primary analysis population is the ITT population with evaluable data. The record also lists outcomes concerning costs, health care use, treatment satisfaction, absence of blood in stool, SIBDQ over time and physician assessment. These are registered outcomes, but their numerical findings are not reported in the available record. The record does not state a sample-size justification or a statistical analysis plan.

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.

Before assignment: A total of 463 participants were enrolled: 461 in the intent to treat (ITT) population were analyzed for efficacy (excluding 2 due to lack of post-baseline measurement data); 463 were analyzed for safety (participants who had received at least one dose of study drug).

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: All participants in the ITT population with evaluable data.

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: Intention to Treat (ITT) population: all participants who had received at least one dose of study drug and had data for at least one post-baseline assessment of any effectiveness measurement \[up to and including Week 26\] while receiving treatment with adalimumab.

6. Primary Endpoint

The registry lists the primary outcome as Mean Change From Baseline in Short Inflammatory Bowel Disease Questionnaire (SIBDQ): Total Score, measured over Week 0 (baseline) and Week 26.

The primary endpoint is mean change from baseline in the total score on the Short Inflammatory Bowel Disease Questionnaire (SIBDQ), assessed at Week 0 and Week 26. The SIBDQ is a disease-specific health-related quality-of-life measure for inflammatory bowel disease. Its ten questions address physical, social and emotional status. Each question is scored from 1, representing poor quality of life, to 7, representing optimum quality of life; the total score ranges from 10 to 70. A higher score indicates better health-related quality of life.

A change score expresses the difference between a participant’s follow-up measurement and baseline measurement. Averaging those changes gives a group-level summary of how scores shifted during the observation period. This can make a patient-reported outcome easier to interpret than a follow-up score alone because it directly references each participant’s starting value. In general, the mean describes the average change, while a measure of dispersion describes how much individual changes vary. The endpoint specifies standard deviation as its dispersion measure.

The registry describes the SIBDQ as able to detect and define meaningful clinical changes in inflammatory bowel disease. That rationale explains why it was selected as an outcome; it does not establish that a particular observed change occurred in this study. The registry record does not report the mean change, standard deviation, number of participants contributing data, or an estimate of uncertainty for the primary endpoint. The magnitude, direction and precision of the observed result are therefore not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The methods information in the registry is limited. It identifies the primary outcome as a mean change in a continuous measure, gives the assessment times and names standard deviation as the dispersion measure. It also identifies the primary analysis population as the ITT population with evaluable data. It does not report the statistical model, test, confidence interval method, missing-data procedure or covariate adjustment. The explanations below describe general statistical concepts and do not imply that a particular unreported method was used.

What does a mean change represent?

For a continuous outcome such as the SIBDQ total score, a change score represents a participant’s follow-up value relative to their baseline value. A group mean change averages the individual changes among those included in the analysis. The sign and size of that estimate would convey the average direction and magnitude of score movement. Here, the registry record defines the intended endpoint but does not report its numerical estimate, so that interpretation cannot be applied to an observed result.

A mean is useful as a compact description of the group’s average, but it does not show the full pattern of individual responses. Participants can have different changes even when the average is near zero, and a group mean can conceal variation across participants. The standard deviation describes the spread of observed values around the mean; it is not itself a measure of how precisely the mean has been estimated. The registry specifies standard deviation as the dispersion measure, but does not report its value for the primary endpoint.

How should uncertainty be understood?

In general, a confidence interval describes uncertainty around an estimate under a specified statistical procedure and its assumptions. A relatively narrow interval indicates greater precision than a wide interval, while the interval’s location helps show which values remain compatible with the data. It is not a guarantee that the true value lies within the interval, nor does it replace considering the study design. The registry record does not report a confidence interval for the SIBDQ change.

A p-value is a measure derived from a statistical test under a null hypothesis and test assumptions. It is not the probability that the null hypothesis is true, nor does it describe the size or practical importance of a change. Since no p-value for the primary outcome is reported, the record does not support claims about statistical significance. More broadly, statistical significance and clinical importance are different questions: the first concerns compatibility with a statistical null model, while the second concerns the meaning of a change for patients. The registry provides the score’s directionality and intended role, but no observed change to evaluate on either dimension.

Why does the single-group design matter?

In a single-group study, outcomes before and after treatment can be described, but the participants do not provide a concurrent untreated or alternative-treatment comparison. General influences on outcomes over time can therefore be mixed with any treatment-associated change. Examples include changes in disease status, other care, expectations and patterns of follow-up. Without a control group, those influences are not isolated by random allocation. This does not make descriptive change uninformative, but it limits causal interpretation.

The study is open-label, meaning masking is listed as none. In general, knowledge of treatment can affect participant reports, clinician assessments and care decisions. This is particularly relevant to outcomes that involve self-reported quality of life or physician assessment. It does not show that such bias occurred, and the registry does not report an assessment of it.

What does the sample-size illustration mean?

The record describes planning illustrations based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms and a normal approximation; the effect to detect is a stated fraction of the observed effect. Those are planning assumptions, not a description of the single-group trial’s allocation, which is listed as not applicable. A hypothetical two-arm calculation does not turn this study into a controlled comparison. Such sample-size figures are planning illustrations and should not be read as observed power. The registry does not report a sample-size justification for the trial itself, and the observed primary effect needed to interpret the illustration is not reported in the registry record.

What can be inferred from the analysis counts?

The registry lists 16 outcome measures and 45 analyses. Multiple outcomes or analyses can raise multiplicity concerns: when many statistical tests are performed, the chance of at least one apparently notable result can increase. General approaches include defining a primary analysis in advance, controlling error rates, or treating secondary findings as exploratory. The record does not report which approach was used, whether adjustments were made, or how the 45 analyses relate to the listed outcomes. It is therefore not possible to determine the inferential status of individual analyses from these counts alone.

10. Confidence Intervals for Each Arm

No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.

R code and output

arm <- c("Participants Receiving Adalimumab")
n   <- c(460)
m   <- c(17.4)
sp  <- c(14.48)
sd  <- sp      # posted standard deviations
for (i in seq_along(n)) {
  se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
  cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}

CARM  Participants Receiving Adalimumab  460  17.400  14.480  16.073  18.727

11. Multiplicity, Missing Data and Analysis Populations

The registry lists 16 outcome measures and 45 analyses, but does not report a multiplicity strategy. When several outcomes or analyses are examined, the collection of results can be harder to interpret than a single prespecified test. In general, a protocol may define a primary endpoint and a hierarchy of secondary endpoints, adjust for multiple testing, or identify secondary analyses as exploratory. The registry record does not state whether such a hierarchy or adjustment was used, or which of the listed analyses were considered confirmatory.

The record identifies the primary analysis population as participants in the ITT population with evaluable data. ITT generally refers to analyzing participants according to their assigned group, but this study is single-group and has no randomized allocation. The additional requirement for evaluable data means that outcome availability matters to the stated population. The registry does not report the number with evaluable SIBDQ measurements at Week 0 or Week 26, the extent or pattern of missing data, or the method used to handle missing measurements. It also does not say whether any sensitivity analyses assessed the effect of missingness assumptions.

Missing data can affect both the size and representativeness of an estimate if participants with observed follow-up differ from those without it. This is a general concern, not evidence that missingness biased this study’s result. Without information on outcome completeness and the analysis method, readers cannot assess how robust the endpoint estimate would be to missing data. The numerical primary result itself is not reported in the registry record, further limiting assessment.

The registry also does not report a statistical analysis plan, prespecified model, covariate adjustment, or interim-analysis plan. These details should not be inferred from the outcome definitions or analysis counts. The record therefore gives a basic outline of what was measured and who was intended for the primary analysis, while leaving important questions about multiplicity and missing-data handling unanswered.

12. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: colitis ulcerative (8.9%), anaemia (0.4%), abortion induced (0.4%), pancytopenia (0.2%), thrombocytopenia (0.2%).

The adverse-event record lists 463 participants at risk for serious adverse events and 67 participants affected by serious adverse events. It also lists 463 participants at risk for other adverse events and 178 affected. These are counts for the adalimumab group; the study has no concurrent control group for a comparative safety assessment. The registry record does not report the number of deaths.

The listed serious adverse-event terms include colitis ulcerative at 8.9%, anaemia at 0.4%, abortion induced at 0.4%, pancytopenia at 0.2%, thrombocytopenia at 0.2%, and silent myocardial infarction at 0.2%. These percentages describe the listed terms in the adverse-event record. The registry entry does not provide further detail in this information about event timing, duration, severity beyond the serious-event classification, or whether an event was judged related to treatment.

Counts of participants affected and counts of event terms are not necessarily interchangeable: one participant may experience more than one event, and the record does not provide participant-level event histories. A serious adverse event is a safety classification, not by itself proof that treatment caused the event. With no control group, these figures describe reported events among treated participants but cannot establish whether the event frequency differs from another treatment or from the background rate. The registry record does not report a comparative safety estimate.

13. What the Result Does, and Does Not, Mean

The study’s primary question concerns change in disease-specific quality of life during adalimumab treatment, measured by the SIBDQ from Week 0 to Week 26. The registry provides the scale description, assessment times and intended analysis population. It does not report the numerical mean change or its variability. Accordingly, the direction, magnitude and precision of the primary result are not reported in the registry record. No conclusion about the observed quality-of-life change can be drawn from the endpoint description alone.

The design also defines what the study can mean. A single-group, open-label study can describe outcomes among enrolled participants receiving adalimumab in usual clinical practice. It cannot, on its own, establish how those outcomes would compare with a different treatment or with no treatment. A before-and-after change may reflect treatment as well as other influences over time; the design does not separate those explanations through randomization or a concurrent control arm.

The registry lists economic, utilization, satisfaction and additional clinical outcomes, but does not report their numerical findings in the available record. The existence of a registered outcome is not evidence of a particular result. The adverse-event information reports affected-participant counts and selected serious-event terms, but the lack of a comparator and the absence of attribution details limit what can be inferred about treatment-related risk.

These limits do not mean the study question is unimportant. Quality of life and health care use are meaningful areas of investigation, and describing outcomes in routine practice can contribute information about the experience of treated participants. The distinction is between describing what was planned or counted and estimating a treatment effect. The registry supports the former in part, but its reported information is insufficient to quantify the primary change or to establish a causal comparison.

14. Why This Trial Matters Statistically

This trial is statistically notable for the gap between a clearly described clinical question and the limited outcome information reported in the registry. It identifies a patient-reported quality-of-life endpoint, assessment times, score interpretation and an intended analysis population. Those details help define what an outcome estimate would represent. But without the mean change, standard deviation or an uncertainty interval, readers cannot assess either the observed magnitude or its precision.

The single-group design makes the distinction between change over time and treatment effect especially important. Repeated measurement can describe whether scores moved among participants with available assessments, but an uncontrolled comparison does not identify what would have happened over the same period without adalimumab or under another treatment. This is a general issue of causal interpretation in before-and-after studies, not a judgment about the direction of this trial’s unreported result.

The study also spans several domains, including quality of life, costs, health care utilization, treatment satisfaction and clinical measures. A broad set of outcomes can illuminate different aspects of care, while also making it important to know which analyses were prespecified and how multiplicity was handled. The registry reports 16 outcome measures and 45 analyses but does not explain the statistical plan or multiplicity approach. That leaves the status of secondary findings difficult to evaluate.

Finally, an outcome population defined by evaluable data makes completeness consequential. The registry does not report the number of participants contributing primary endpoint measurements or its handling of missing data. Together, the absence of numerical primary results, incomplete analysis details and lack of a comparator mean the registry record is most informative about the study’s design and measurement intentions, rather than a quantified estimate of treatment effect.

15. 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 direct limitation of the registry information is that it does not report the numerical primary endpoint result. The mean SIBDQ change, standard deviation, number analyzed and confidence interval are not reported in the registry record. The numerical findings for the registered secondary outcomes are also not reported in the available information. This prevents assessment of the magnitude, direction and precision of the study’s outcomes.

The single-group, open-label design limits causal interpretation. There is no concurrent control group, and allocation is listed as not applicable. Changes observed during follow-up cannot be separated from other influences over time through a randomized contrast. The open-label design also means masking is absent; the registry does not report whether or how this was addressed for patient-reported or clinician-assessed outcomes.

The primary population is described as the ITT population with evaluable data, but the record does not provide the number of participants with evaluable baseline and Week 26 scores. Missing-data extent, reasons for missingness, imputation or other handling methods, and sensitivity analyses are not reported in the registry record. The record also does not report a sample-size justification, the statistical analysis plan, covariate adjustment, interim analyses, or a multiplicity strategy.

The available regimen description is incomplete after it mentions possible dose escalation after Week 8. Details beyond that point are not reported in the registry record. The listed adverse-event counts and serious-event terms lack a concurrent comparator in this study, and the available information does not provide event attribution or further participant-level detail. These limitations concern what can be interpreted from the registry record; they do not establish that unreported procedures or analyses were absent from the full study documentation.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: A Study to Evaluate the Impact of Adalimumab on Quality of Life, Health Care Utilization and Costs of Ulcerative Colitis Subjects in the Usual Clinical Practice Setting — NCT01550965.
• Registry record last updated 2018-07-02; status last verified 2016-05.
• Results first posted 2016-11-01.
• Data retrieved from ClinicalTrials.gov on 2026-11-09.
• Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.

19. Record Summary

NCT01550965 is a completed, open-label, multicenter, Phase 3, single-group study of adalimumab in ulcerative colitis. It enrolled 463 participants and is described as taking place in the usual clinical practice setting. The study began in 2012-05 and reached primary completion in 2015-04. The sponsor is listed as AbbVie (prior sponsor, Abbott).

The primary outcome is mean change in total SIBDQ score from Week 0 to Week 26 among participants in the ITT population with evaluable data. The registry explains the questionnaire and its scoring, but does not report the numerical primary result. It also lists economic, health care utilization, satisfaction and clinical secondary outcomes without reporting their numerical findings in the available record.

The design has no concurrent comparator, and the record does not report the statistical analysis plan, missing-data methods or multiplicity strategy. Safety information lists 67 participants affected by serious adverse events and 178 affected by other adverse events, among 463 at risk in each category. The registry does not report a death count.