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 Gastrointestinal and Health-related Quality of Life Outcomes in Patients With Autoimmune Diseases Treated With Mycophenolate (NCT00351377), a non-randomized phase 3 study. The primary outcome is Changes in GI Symptom Severity After Conversion From Mycophenolate Mofetil (MMF) to Enteric-coated Mycophenolate Sodium (EC-MPS).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00351377 |
| Phase | Phase 3 |
| Condition | Autoimmune Disease |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 111 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Novartis Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
Patients with autoimmune diseases who were receiving mycophenolate mofetil before study entry. The primary outcome population is defined as the intention-to-treat population: enrolled participants who received study medication.
Intervention
Conversion to enteric-coated mycophenolate sodium, administered orally in divided doses twice daily. The dose was equimolar to the mycophenolate mofetil dose being taken at study entry. Planned treatment duration was 6 to 8 weeks.
Comparator
The registry describes a single-group, nonrandomized study, not a concurrent control group. The primary outcome compares gastrointestinal symptom scores at baseline with scores at 6 - 8 weeks after conversion.
Primary question
How did the mean Gastrointestinal Symptom Rating Scale total score change from baseline to 6 - 8 weeks after conversion from mycophenolate mofetil to enteric-coated mycophenolate sodium? The registry record does not report the observed change.
The study addresses gastrointestinal symptoms and health-related quality of life in patients with autoimmune diseases treated with mycophenolate. Its stated aims include assessing the proportion of patients experiencing gastrointestinal complaints under mycophenolate mofetil-based immunosuppressive treatment and assessing whether a switch to enteric-coated mycophenolate sodium is followed by changes in gastrointestinal symptoms or quality of life.
The primary outcome is a before-and-after measure in the converted group. It can describe how scores changed over the study interval, but, without a concurrent control group, it cannot by itself separate a treatment-associated change from other influences over time. The record also lists secondary measures relating to gastrointestinal symptoms, gastrointestinal-related quality of life, psychological well-being, and overall treatment effects assessed by physicians or patients.
3. Trial Design
This was a phase 3, nonrandomized, unmasked, single-group study. The registry lists enrollment of 111 and status as completed. The record gives a start date of 2006-06 and a primary completion date of 2009-06. It identifies Novartis Pharmaceuticals as the sponsor.
Participants receiving mycophenolate mofetil were converted to enteric-coated mycophenolate sodium. The tablets were administered orally in divided doses twice daily, at a dose equimolar to the mycophenolate mofetil dose taken at study entry. Planned treatment duration was 6 to 8 weeks. The primary outcome timeframe is baseline and 6 - 8 weeks.
Because the study is described as single-group and nonrandomized, the registry does not describe a randomized allocation to treatment or a concurrent control arm. The main comparison for the primary endpoint is therefore within participants, across the baseline and follow-up assessments. The absence of masking means that the study is recorded as unmasked; details of how this may have affected assessments are not reported in the registry record.
The primary outcome is change in the Gastrointestinal Symptom Rating Scale total score. Secondary outcomes include changes in symptom severity subscales, the Gastrointestinal Quality of Life Index and its subscales, and the Psychological General Well-Being Index and its subscales. The record also includes overall treatment effects for gastrointestinal symptoms assessed by the physician and patient, and for health-related quality of life assessed by the patient.
The registry record does not report a detailed protocol for allocation, recruitment, follow-up, or statistical analysis. The planned treatment interval and outcome schedule describe the study’s intended assessment window, but do not establish how many participants completed follow-up or contributed to each outcome.
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 | Enteric-coated Mycophenolate Sodium |
|---|---|
| STARTED | 111 |
| COMPLETED | 104 |
| NOT COMPLETED | 7 |
Reasons for not completing this period:
| Reason | Enteric-coated Mycophenolate Sodium |
|---|---|
| Adverse Event | 5 |
| Death | 1 |
| Unsatisfactory therapeutic effect | 1 |
Analysis population for the primary outcome, as defined in the registry: Intention to treat (ITT) population consisting of all enrolled participants who received 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 | Enteric-coated Mycophenolate Sodium |
|---|---|---|
| Participants analysed | 111 | |
| Age Continuous | years | 49.5 (SD 14.1) |
| Sex: Female, Male: Female | Participants | 82 |
| Sex: Female, Male: Male | Participants | 29 |
6. Primary Endpoint
The registry lists the primary outcome as Changes in GI Symptom Severity After Conversion From Mycophenolate Mofetil (MMF) to Enteric-coated Mycophenolate Sodium (EC-MPS), measured over Baseline and 6 - 8 weeks.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | Scores on a scale |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is change in gastrointestinal symptom severity, measured as change in the Gastrointestinal Symptom Rating Scale total score from baseline to the visit at 6 - 8 weeks. The total score is the mean of 15 individual items, each scored from 1-7. The total score also ranges from 1-7, with higher scores indicating more unfavorable conditions.
This is a continuous measure: it retains information about the degree of reported symptoms rather than reducing the outcome to a simple present-or-absent category. A mean can describe the average score in the analysis population, while the standard deviation describes how spread out individual scores are around that mean. The registry identifies the endpoint parameter as a mean and the dispersion measure as a standard deviation.
Assessing change from baseline is relevant to a conversion study because it describes whether participants’ reported symptom scores shifted over the follow-up interval. The scale’s direction is important when interpreting any change: a higher score represents less favorable symptom status. However, the registry record does not report the baseline mean, follow-up mean, mean change, standard deviation, confidence interval, or test result. The direction and size of the observed change therefore cannot be determined from the registry record.
The endpoint is patient-reported symptom severity, not an objective measure of gastrointestinal function or a direct measure of the cause of symptoms. The record describes the score construction and interpretation, but does not report additional information about how item-level responses were handled when missing.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record defines a clinically relevant descriptive question: whether gastrointestinal symptom scores changed after conversion from mycophenolate mofetil to enteric-coated mycophenolate sodium. It does not report the primary endpoint result. There is no baseline or follow-up score, estimated mean change, measure of uncertainty, or statistical test result in the record. Consequently, the direction, magnitude, and precision of any observed change are not reported in the registry record.
The study design also limits what a before-and-after comparison can establish. In a single-group study, participants serve as their own reference across time, but there is no concurrent group to show what would have happened over the same interval without conversion. A change could be associated with the switch, but changes in symptoms over time or other influences cannot be distinguished from a treatment effect using this design alone. This is a general limitation of uncontrolled before-and-after comparisons, not evidence that any particular influence occurred in this study.
The outcome is measured on a scale where higher values indicate more unfavorable conditions. If a mean change were reported, the scale direction would be essential to its interpretation. The registry does not provide that estimate, so it is not possible to say whether the posted results suggest an increase, decrease, or little change in symptom severity. It is also not possible to judge how variable individual scores were or how precisely the mean change was estimated.
The registry lists 9 outcome measures, including the primary gastrointestinal symptom measure and secondary measures of symptoms, quality of life, psychological well-being, and overall treatment effects. Considering multiple outcomes can create challenges in interpreting isolated findings, especially when the statistical plan for controlling false-positive findings is unknown. The registry record does not report an analysis plan, adjustment for multiple comparisons, or outcome-level analyses.
The safety information lists 111 participants at risk for serious adverse events and 6 affected, as well as 111 at risk for other adverse events and 38 affected. These are descriptive counts in the registry record. They do not, on their own, establish whether an event was caused by study medication, and the record does not provide a comparative safety group.
Overall, the registry describes the study question and outcome framework but does not supply outcome estimates needed to judge the observed symptom change. The design can describe changes among participants who were assessed, but a causal comparison and a precise estimate of the primary outcome cannot be reconstructed from the registry record.
9. Statistical Methods Explained
The registry describes a continuous primary endpoint analyzed as a mean, with standard deviation as the dispersion measure. It does not report the analysis method used for the primary outcome, nor does it provide the numerical results. The explanations below are general statistical context, not a description of methods confirmed to have been used in this study.
What does a mean change describe?
A mean change summarizes the average difference between measurements over time for the participants included in an analysis. In a before-and-after study, the natural unit of analysis is often each participant’s paired measurements: baseline and follow-up. A paired approach accounts for the fact that measurements from the same person are related. The registry identifies a change endpoint but does not state the exact calculation or statistical test used.
A mean is useful for conveying the average direction and size of change on the original scale. It can, however, conceal differences between participants. Some participants may improve, some may worsen, and others may have little change, while the average alone does not show that distribution. The registry’s specified standard deviation would describe spread among scores or changes, depending on what was summarized; the record does not clarify which result-level standard deviations were reported because no outcome results are listed.
What does the standard deviation tell the reader?
Standard deviation describes variability among observations. It is not the same as the standard error, which reflects the precision of an estimated mean, and it is not itself a confidence interval. A confidence interval is a general way of expressing a range of values compatible with the data and a chosen statistical procedure. Its width depends on the variability, the amount of information, and the analysis method. No confidence interval or standard error for the primary endpoint is reported in the registry record, so the precision of the estimate cannot be assessed.
Scale interpretation also matters. The Gastrointestinal Symptom Rating Scale total is the average of 15 single items, with each item ranging from 1-7 and the total score ranging from 1-7. Higher values indicate more unfavorable conditions. Any reported difference would need to be read in light of that direction. The registry does not report an observed difference, and no additional threshold for a meaningful change is reported.
Why does the study design matter to the analysis?
In general, a within-person comparison can account for stable characteristics of participants because each person’s follow-up score is compared with their own baseline score. But the design does not control for factors that may change over time. Without a concurrent control group, the statistical analysis cannot by itself determine whether a change was caused by conversion rather than other time-related influences. This distinction concerns the design’s ability to support causal interpretation, not the correctness of calculating a mean change.
The study is recorded as nonrandomized, unmasked, and single-group. The registry does not report a randomization procedure, a control group, or masking. It also does not report adjustment for baseline characteristics, a model for repeated measurements, or a strategy for handling departures from statistical assumptions. Those methods should not be presumed.
What does over-dispersion mean in general?
Over-dispersion refers to variability greater than a statistical model expects. It is especially relevant to count or categorical outcomes modeled under assumptions that constrain their variance. The primary outcome in this record is a continuous score, and the registry does not state that an over-dispersion model was used. More generally, when data are more variable than a model assumes, standard errors can be underestimated and uncertainty can appear too small unless the analysis accounts for the extra variation. This is a general statistical principle, not a finding about this study.
How should planning illustrations be read?
The record’s planning assumptions describe illustrations based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The effect to detect is described as a stated fraction of the observed effect. These are planning assumptions, not a description of the study’s single-group design or its observed results. The record does not report numerical planning outputs in the information available for this analysis, and the primary outcome estimate needed to interpret such illustrations is not reported in the registry record.
Sample-size illustrations are prospective planning aids: they indicate how a hypothetical future design might be sized under stated assumptions. They are not observed power and do not establish the probability that the completed study detected an effect. The registry record does not report a sample-size justification for the study or clarify whether the planning assumptions formed part of its original design.
Finally, statistical significance and clinical importance are different concepts. A test result, if reported, would address compatibility with a statistical null hypothesis under the chosen method. It would not by itself establish that a change matters to patients. Interpretation would also require the size and precision of the estimated change and relevant clinical context, which are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 9 outcome measures, spanning gastrointestinal symptoms, gastrointestinal-related quality of life, psychological well-being, and overall treatment effects. When several outcomes are examined, the chance of observing an apparently unusual result somewhere among them can increase. Statistical plans sometimes address this multiplicity by defining a hierarchy, adjusting significance thresholds, or treating secondary results as exploratory. The registry record does not report whether any such approach was planned or used, and it lists no outcome analyses.
The registry defines the primary analysis population as the intention-to-treat population consisting of all enrolled participants who received study medication. This definition identifies who is intended for inclusion but does not report how many contributed primary endpoint data at baseline or follow-up. It also does not explain how participants who discontinued treatment, missed a visit, or had incomplete questionnaire items were handled.
Missing-data methods are not reported in the registry record. There is no description of imputation, complete-case analysis, or sensitivity analysis. These choices can matter when follow-up is incomplete, because the analyzed participants may differ from those without outcome data. The record does not provide the information needed to assess the amount, pattern, or likely impact of missing outcome data.
The primary endpoint is scheduled from baseline to 6 - 8 weeks, and the record describes the intended population and timeframe. It does not provide participant flow, outcome-specific denominators, or a statistical analysis plan. As a result, neither the handling of missing observations nor the relationship between the stated analysis population and the actual outcome data can be assessed.
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 |
|---|---|---|---|
| Enteric-coated Mycophenolate Sodium | 6 | 111 | 5.4% |
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 |
|---|---|
| Enteric-coated Mycophenolate Sodium | 38 of 111 |
Most frequent serious adverse event terms
| Event term | Organ system | Enteric-coated Mycophenolate Sodium |
|---|---|---|
| Nephrogenic anaemia | Blood and lymphatic system disorders | 1 of 111 |
| Thrombocytopenia | Blood and lymphatic system disorders | 1 of 111 |
| Diverticulum | Gastrointestinal disorders | 1 of 111 |
| Bronchitis | Infections and infestations | 1 of 111 |
| Endocarditis | Infections and infestations | 1 of 111 |
| Pneumonia | Infections and infestations | 1 of 111 |
| Septic shock | Infections and infestations | 1 of 111 |
| Dementia | Nervous system disorders | 1 of 111 |
| Disturbance in attention | Nervous system disorders | 1 of 111 |
| Dizziness | Nervous system disorders | 1 of 111 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Enteric-coated Mycophenolate Sodium |
|---|---|---|
| Diarrhoea | Gastrointestinal disorders | 16 of 111 |
| Nausea | Gastrointestinal disorders | 13 of 111 |
| Vomiting | Gastrointestinal disorders | 10 of 111 |
| Nasopharyngitis | Infections and infestations | 10 of 111 |
| Abdominal distension | Gastrointestinal disorders | 8 of 111 |
| Dyspepsia | Gastrointestinal disorders | 8 of 111 |
Serious events with the highest arm-level rate: nephrogenic anaemia (0.9%), thrombocytopenia (0.9%), diverticulum (0.9%), bronchitis (0.9%), endocarditis (0.9%).
The registry lists 111 participants at risk for serious adverse events, with 6 affected. It also lists 111 participants at risk for other adverse events, with 38 affected. These are counts as recorded; they should not be interpreted as event rates beyond what the record states, and no additional rate has been calculated.
The serious adverse event terms listed are nephrogenic anaemia, thrombocytopenia, diverticulum, bronchitis, endocarditis, and pneumonia, each reported as 0.9%. The record does not provide event-specific counts alongside those percentages, nor does it describe timing, severity grading, recurrence, or whether events led to treatment discontinuation.
Adverse event reporting is descriptive. The listed counts do not establish that an event was caused by enteric-coated mycophenolate sodium. The study is single-group, and the registry record does not present a concurrent safety comparison. The record also does not report an assessment attributing events to study treatment. These limitations prevent a causal interpretation of the safety figures.
The at-risk figures and affected counts are registry summaries, not a substitute for detailed event narratives or comparative analyses. They should be read as the safety information recorded for this study, with attribution and comparative risk not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The study was designed to assess changes in gastrointestinal symptoms and health-related quality of life after conversion from mycophenolate mofetil to enteric-coated mycophenolate sodium. Its primary endpoint is a patient-reported score measured at baseline and 6 - 8 weeks. The registry record reports the scale’s construction and direction, but not the observed outcome estimate.
Without a reported baseline score, follow-up score, mean change, variability, or confidence interval, the registry record does not establish whether symptoms changed, how large any change was, or how precisely it was estimated. The absence of reported outcome analyses also means that statistical significance cannot be evaluated from the registry record. No conclusion about the direction or size of the result should be inferred from the study aims alone.
Even if a within-person change were available, this single-group, nonrandomized design would describe change over time among assessed participants rather than establish that the medication switch caused the change. A concurrent control group could help distinguish a treatment-associated change from other influences over the same period; such a group is not described in the registry record.
The registry lists secondary measures that could provide broader context about symptoms and quality of life, along with physician- and patient-assessed overall treatment effects. Their results are not reported in the registry record. The listed safety counts likewise describe events but do not show whether those events were caused by treatment or how they compare with another group.
Thus, the record establishes the study question, treatment approach, endpoint definition, and reported safety counts. It does not provide the result data needed to determine what changed or to quantify uncertainty. The interpretation is consequently limited to the planned assessment and the structure of the available registry information.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of a before-and-after question in a clinical setting where outcomes are patient-reported. The primary endpoint uses a continuous score, so a mean change could describe the average symptom shift while a measure of dispersion could show how much responses varied. Such summaries answer different questions: an average describes the center of the observed change, while dispersion reflects differences across participants.
The design makes the distinction between describing change and attributing change especially important. A single-group comparison can characterize scores before and after conversion, but it does not supply the counterfactual comparison needed to determine what would have happened without conversion. This is a general causal-inference limitation of uncontrolled studies, even when the outcome measurement is clearly defined.
The record also illustrates why outcome definitions alone are not enough for statistical interpretation. The scale range, direction, timeframe, and analysis population are described, but the primary endpoint estimate, its uncertainty, and the analysis method are not reported. Without those results, readers cannot assess the magnitude or precision of change, or compare the statistical finding with a threshold of practical importance.
Multiple outcome domains add another interpretive consideration. Gastrointestinal symptoms, quality of life, psychological well-being, and global assessments may provide complementary perspectives, but a reader needs the analysis plan and results to understand how those outcomes were prioritized and interpreted. The registry record lists the outcome domains but does not report the statistical strategy or analyses.
The central statistical lesson is that a well-defined endpoint and a completed study do not by themselves supply evidence about the result. Design, missing-data handling, multiplicity, estimates, and uncertainty all shape what can be concluded. In this record, those result-level details are not reported, so the study’s observed statistical findings cannot be characterized.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The most important limitation is that the registry record does not report the primary endpoint result or any outcome-level analyses. There is no estimate of mean change, no standard deviation attached to a reported result, and no confidence interval or test result. Consequently, the magnitude, direction, and precision of the primary outcome cannot be evaluated.
The single-group, nonrandomized design provides no concurrent comparator. Changes between baseline and follow-up therefore cannot be separated from other influences that may change over time. The study is also recorded as unmasked; the record does not explain how that design feature was managed for patient-reported or clinician-assessed outcomes.
The planned primary endpoint timeframe is baseline and 6 - 8 weeks, with planned treatment duration of 6 to 8 weeks. Longer-term outcomes are not described in the primary endpoint information. The registry record does not report follow-up completion, outcome-specific denominators, or the handling of missing questionnaire responses. The impact of attrition or incomplete data cannot therefore be assessed.
Further details about the participants’ autoimmune diagnoses, baseline symptom distribution, recruitment setting, concomitant treatments, and eligibility criteria are not reported in the registry record. The record also does not provide a detailed statistical analysis plan, sample-size justification, multiplicity approach, or methods for dealing with deviations from model assumptions. These omissions limit assessment of both the population represented and the robustness of any analysis.
The adverse-event summary does not provide a concurrent comparison or treatment-attribution analysis. It lists affected counts and serious-event terms, but not event timing, recurrence, or detailed participant-level context. Accordingly, the safety information cannot establish comparative risk or causality.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Gastrointestinal and Health-related Quality of Life Outcomes in Patients With Autoimmune Diseases Treated With Mycophenolate — NCT00351377.
- Registry record last updated 2011-04-21; status last verified 2011-04.
- Results first posted 2011-01-12.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
The registry record describes a completed phase 3, nonrandomized, unmasked, single-group study sponsored by Novartis Pharmaceuticals, with enrollment of 111. Participants receiving mycophenolate mofetil were converted to enteric-coated mycophenolate sodium at an equimolar dose, administered orally in divided doses twice daily. Planned treatment duration was 6 to 8 weeks.
The primary endpoint was change in the Gastrointestinal Symptom Rating Scale total score from baseline to 6 - 8 weeks. Higher scores indicate more unfavorable conditions. Secondary outcomes address gastrointestinal symptoms, quality of life, psychological well-being, and overall treatment effects.
The record lists serious and other adverse-event counts but does not report the primary endpoint results or outcome analyses. It also does not report the statistical analysis plan or methods for missing data. The study can therefore be described by its design, endpoint, and recorded safety information, but the primary outcome’s direction, size, and precision are not reported in the registry record.