Small Bowel Capsule Endoscopy Findings in Patients Receiving Cellcept®: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Small Bowel Capsule Endoscopy Findings in Patients Receiving Cellcept® (NCT00652834), a na phase 4 study. The primary outcome is GI Mucosal Lesions Change and Clinical Symptoms Using The Gastrointestinal Symptom Rating Scale (GSRS) Score.

2. Clinical Question

The registry record frames the study around gastrointestinal symptoms and lesions associated with MMF use and the changes observed after conversion to EC-MPS. The participants were kidney transplant recipients with GI symptoms, rather than a broad sample of all people taking mycophenolic acid. This population definition matters when interpreting how broadly the findings might apply.

The clinical question is a before-and-after question within one group. It is not a question about whether one treatment is better than another in a randomized, concurrent comparison. Without a separate control group, any change observed over the study interval could not by itself distinguish a treatment-related change from other explanations, such as the passage of time or changes in symptoms that would have occurred without switching.

The record describes a four-week study and an assessment around 30 days after the switch. It names GSRS symptom scores and SBCE mucosal examination, but the posted primary outcome results are not reported in the registry record. Therefore, the direction, size, and precision of any change cannot be assessed from the registry record.

3. Trial Design

This was a Phase 4, single-group study sponsored by the University of California, Los Angeles. The registry lists enrollment of 23. The study began in 2009-04, had primary completion in 2011-03, and is recorded as completed. These administrative details describe the record but do not supply evidence about the magnitude or precision of an outcome.

The study group consisted of kidney transplant recipients with gastrointestinal symptoms who were using MMF. The arm description says participants switched from MMF to EC-MPS and characterizes the investigation as lasting four weeks. The intervention list includes myfortic and small bowel capsule endoscopy. The stated purpose was to examine gastrointestinal mucosal lesions and changes in clinical symptoms after the switch.

Allocation is listed as not applicable, masking as none, and the model as single group. There is no randomized allocation and no concurrent control group described. Consequently, the design does not support a like-for-like comparison between independent treatment groups. Instead, the intended clinical assessment concerns changes within the participant group around the treatment switch.

The primary outcome is described as GI mucosal lesions change and clinical symptoms using the GSRS score, with a one-month timeframe. The record states that GSRS is a seven-point graded Likert-type scale, with higher scores indicating worse symptoms. The description also refers to a difference between D30 and last SBCE scores. The record does not explain in detail how those measurements were combined or how mucosal findings were quantified for the primary analysis.

The completed status does not mean that outcome estimates are available in the registry record. The primary estimate, its confidence interval, and an analysis result are not reported in the registry record. The statistical analysis plan, sample-size justification, interim analyses, and details of the analysis population are also not reported in the registry record.

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

Reasons for not completing this period:

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: of 23 participants, 18 participants completed the study.

6. Primary Endpoint

The registry lists the primary outcome as GI Mucosal Lesions Change and Clinical Symptoms Using The Gastrointestinal Symptom Rating Scale (GSRS) Score, measured over one month.

The named primary outcome concerns change in gastrointestinal mucosal lesions and clinical symptoms, with symptoms assessed using the Gastrointestinal Symptom Rating Scale (GSRS). The registry gives the measure as a mean GSRS score and describes its dispersion as a 95% confidence interval. The timeframe is one month.

The GSRS is described as a seven-point graded Likert-type scale. A score of 1 represents absence of troublesome symptoms, while 7 represents very troublesome symptoms. Thus, higher scores indicate worse symptoms. The registry states that a difference between D30 and last SBCE scores greater or equal to 0.3 can be considered a clinically significant improvement in symptoms. This is a criterion stated in the record, not a reported study result.

Patient-reported symptom scales can capture experiences that may not be fully represented by an examination alone. SBCE is separately named as the procedure used to examine the small-bowel mucosa. Together, symptom assessment and mucosal examination address distinct aspects of gastrointestinal status: experienced symptoms and observed lesions.

The outcome description does not report the actual mean score, change estimate, confidence interval, or numerical SBCE findings. It also does not make clear how mucosal lesion findings were incorporated into the named mean GSRS measure. Those outcome details are not reported in the registry record, so the observed change and its precision cannot be characterized.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The study’s central statistical feature is its single-group, before-and-after framing. Participants were described as switching from MMF to EC-MPS, with symptom and mucosal assessments over one month. This design differs from a randomized comparison, in which concurrent groups are allocated to different treatment conditions. Here, the registry does not identify a separate control arm.

What does a within-group change estimate?

In general, a within-group change compares measurements from the same group at different times. It describes how the measured outcome changed over the assessment period. It does not by itself show what would have happened over the same period if participants had not switched treatments. Without that counterfactual comparison, observed changes may reflect the switch, natural variation, other events during follow-up, or a combination of influences. This is a design limitation, not a calculation error.

The registry describes the primary measure as a mean GSRS score and identifies a 95% confidence interval as the dispersion measure. In general, a mean summarizes the average score in a group, while the confidence interval expresses uncertainty around an estimate under the analysis assumptions. The record does not provide the mean, a change estimate, or the confidence interval. Therefore, neither the average symptom level nor the precision of a change can be evaluated from the registry outcome entry.

How should the GSRS scale be read?

The record describes a seven-point graded Likert-type scale, with 1 indicating absence of troublesome symptoms and 7 indicating very troublesome symptoms. Higher values therefore represent worse symptoms. The registry states that a difference between D30 and last SBCE scores greater or equal to 0.3 can be considered a clinically significant improvement. This threshold gives context for interpreting a reported difference, but it cannot establish that such a difference occurred. The numerical outcome is not reported in the registry record.

GSRS is a symptom measure, whereas SBCE is an examination procedure used to assess the small-bowel mucosa. They address related but distinct outcomes. A symptom score should not be treated as a direct count or measure of mucosal lesions, and the record does not describe a numerical lesion scale or a statistical model for lesion findings. The exact relationship between the mucosal assessment and the named mean GSRS outcome is not reported in the registry record.

What can a confidence interval establish?

In general, a confidence interval communicates the uncertainty around an estimated quantity, conditional on the data and method used. It is not a guarantee that the true value lies within the interval, and its width depends on factors including variability and the amount of information available. For a clinical study, the estimate and interval are important together: the estimate indicates the observed direction and size, while the interval shows how precisely that size is estimated. Neither is posted for the primary outcome in this registry record.

The analysis method used to produce a mean or confidence interval is not reported in the registry record. The record also does not state whether the analysis accounted for repeated measurements, how missing observations were handled, or whether any adjustments were made. No detailed statistical plan or interim analysis information is reported. Consequently, it is not possible to assess whether the reported analysis, if any, followed a prespecified approach.

Why does the design affect causal interpretation?

In general, a concurrent control group helps distinguish changes associated with an intervention from changes that occur over time for other reasons. Random allocation can also reduce systematic differences between groups. This study has no randomized comparison according to the registry record. A pre-to-post change, if reported, would therefore be an association with the period following the switch, not an isolated causal effect of EC-MPS.

The record includes a planning illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. Such assumptions describe a hypothetical planning exercise. A planning sample size is not the number of participants analyzed, and it is not observed power. In general, observed power calculated from an observed effect does not provide a substitute for reporting the effect estimate and its confidence interval. No planning outputs or underlying observed effect are reported in the registry record.

There are no recalculated outcome estimates to interpret from the registry entries. No discrepancy between a sponsor analysis and a simple recalculation is identified. The primary result and detailed analysis approach remain unavailable in the registry record, which limits any statistical conclusion beyond describing the design and measurement definitions.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists one outcome measure and one analysis. That count alone does not describe whether the investigators tested multiple components, time points, or subgroups within that analysis. The record does not report a multiplicity adjustment, a hierarchy of outcomes, or a prespecified strategy for handling multiple statistical tests. It is therefore not possible to determine how any multiplicity was addressed.

Missing-data methods are not reported in the registry record. The entry does not state whether all enrolled participants contributed primary outcome measurements, how incomplete GSRS assessments or SBCE examinations were treated, or whether any imputation or other missing-data approach was used. The enrollment figure should not be assumed to equal the number analyzed for the primary outcome.

The analysis population is also not described. The registry does not say whether the primary analysis included all enrolled participants, only those who completed follow-up, or another defined group. It does not report exclusions, protocol deviations, or a participant flow accounting. Those details matter because conclusions can depend on which observations are included and how missing outcomes are handled.

The record does not report the detailed statistical analysis plan, interim analyses, or sample-size justification. It describes a planning illustration based on an observed effect, but that does not establish the original trial’s design rationale or analysis choices. Without the primary estimate and information about missingness and analysis populations, the robustness of any outcome finding cannot be assessed from the registry record.

11. 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 0% reporting threshold.

The registry lists one group, titled Kidney Transplant Recipients With GI Symptoms, with 23 participants at risk for serious adverse events and 0 affected. It also lists 23 at risk for other adverse events and 0 affected. The serious adverse event terms list is empty.

These entries are counts recorded in the registry. They indicate that the record reports zero affected participants in each of those two categories for the listed group. They do not provide event-level descriptions, severity gradations beyond the serious versus other categories, timing, or a detailed account of surveillance. Such details are not reported in the registry record.

A count of zero is not evidence that every possible event was actively excluded or that events could not have occurred. Interpretation depends on how events were defined, collected, and followed, and those procedures are not reported in the registry record. The registry entries also do not establish how complete the safety follow-up was for the group.

The figures should be read as recorded counts over the study’s safety reporting, not as a comparative estimate. With no separate control group, they do not allow a relative safety comparison between MMF and EC-MPS. The listed figures alone cannot establish the presence or absence of a treatment-related safety effect.

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

The registry record establishes that a completed, single-group study examined kidney transplant recipients with GI symptoms who switched from MMF to EC-MPS. It describes symptom assessment using GSRS and small-bowel mucosal examination using SBCE over one month. It also provides the scale’s direction and a threshold that the record characterizes as potentially clinically significant improvement.

What the record does not establish is the actual change in symptoms or mucosal findings. The primary outcome estimate, confidence interval, and numerical SBCE findings are not reported in the registry record. The direction and size of any observed change therefore cannot be stated, and the stated GSRS threshold cannot be applied to an unreported estimate.

Even if a before-and-after change were available, the design would not establish that the treatment switch caused it. A single-group study has no concurrent control to indicate what would have happened over the same period without the switch. In general, changes over time may have multiple explanations. This limitation applies regardless of whether the observed score moves in a favorable or unfavorable direction.

The registry records zero participants affected by serious adverse events and zero affected by other adverse events in the listed group. These are safety counts as entered in the registry, not proof that adverse effects were impossible or that safety is equivalent to another treatment. Detailed safety definitions and follow-up procedures are not reported in the registry record.

Accordingly, this record is useful for understanding the population, treatment transition, outcome concepts, and study structure. It does not support a conclusion about the amount or statistical precision of symptom or mucosal change, nor a causal treatment comparison. Those conclusions would require outcome estimates and analysis details that are not reported in the registry record.

13. Why This Trial Matters Statistically

This study raises a statistically important question about how symptom reports and observed mucosal findings relate after a treatment switch. Those are different outcome types: one reflects participants’ reported experience, while the other concerns findings from an examination. Keeping the measures distinct helps avoid treating symptom scores as if they directly quantified intestinal lesions.

The design also illustrates the interpretive limits of a single-group pre-to-post study. Such a study can describe changes during follow-up, but the absence of a concurrent comparator means the change cannot be separated from other influences over time. This distinction matters in clinical research because a numerical change and a causal treatment effect are not the same claim.

Precision is another central issue. The record names a mean GSRS score and a 95% confidence interval, but does not report the estimate or interval. A study’s enrollment and completion status do not replace those results. Without the estimate and its uncertainty, readers cannot judge whether a change was small or large, precise or uncertain, or whether it crossed the stated threshold for clinically significant improvement.

The registry also illustrates why outcome definitions and analysis details matter. It identifies a symptom scale and mentions SBCE, but does not report a numerical mucosal endpoint or explain how those measures were combined in the primary analysis. Missing-data handling, analysis populations, and multiplicity methods are not reported in the registry record. These omissions limit assessment of analytic reliability.

Finally, safety counts are most informative when their definitions and ascertainment are clear. The recorded zero counts give a concise description of the registry entries, but not a comparative safety estimate or a complete account of monitoring. The study’s statistical importance lies in the questions it attempts to address and in the need to distinguish measurement, association, precision, and causation when evaluating clinical evidence.

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 principal limitation is that the primary outcome results are not reported in the registry record. No mean GSRS score, change estimate, confidence interval, p-value, or numerical SBCE finding is available. As a result, the magnitude and precision of the intended outcomes cannot be assessed.

The study is described as a single-group investigation with no randomized allocation and no concurrent control. Any within-group change over the follow-up interval, if observed, would not by itself distinguish an effect of the switch from time-related or other influences. The record does not describe a design feature that addresses this limitation.

The endpoint description combines GI mucosal lesions and clinical symptoms, while identifying GSRS as the score measure and SBCE as the examination procedure. It does not specify how mucosal lesions were quantified or how the two types of outcome were integrated into the primary analysis. The relation between D30 and last SBCE scores is not explained in further detail.

The analysis plan, handling of repeated measurements, missing-data methods, and analysis population are not reported in the registry record. Nor are participant flow details or reasons for any missing measurements. Without those details, it is not possible to evaluate the completeness or robustness of the analysis.

Finally, the registry’s adverse-event entries do not describe event definitions, surveillance procedures, or follow-up completeness. The safety counts therefore have limited interpretive context. The record’s completed status and enrollment figure do not resolve these outcome and methods limitations.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Small Bowel Capsule Endoscopy Findings in Patients Receiving Cellcept® — NCT00652834.
• Registry record last updated 2016-03-04; status last verified 2014-12.
• Results first posted 2016-03-04.
• 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.

18. Record Summary

NCT00652834 is a completed Phase 4, single-group study sponsored by the University of California, Los Angeles. It lists enrollment of 23 and concerns kidney transplant recipients with gastrointestinal symptoms who switched from MMF to EC-MPS. The record gives a study start of 2009-04 and primary completion of 2011-03.

The study examined gastrointestinal symptoms using the GSRS and mucosal findings using SBCE over one month. The GSRS is described as a seven-point scale on which higher scores indicate worse symptoms. The registry record does not report the primary outcome results, the detailed statistical analysis plan, missing-data methods, or the analysis population.

For the listed group, the registry records 0 participants affected by serious adverse events and 0 affected by other adverse events. These counts do not constitute a comparative safety analysis. Overall, the record describes the study question and design but does not provide the outcome estimates needed to evaluate symptom or mucosal change.