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 Conversion of CellCept to Myfortic: A Prospective Study in Liver Transplant Recipients (NCT00336895), a na na study. The primary outcome is Gastrointestinal Side Effects and Quality of Life (Total Score of GSRS).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00336895 |
| Phase | NA |
| Condition | Immunosuppression |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 29 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Pittsburgh |
| Results posted | Yes |
2. Clinical Question
Population
Liver transplant recipients receiving CellCept who had gastrointestinal side effects. The registry record lists enrollment as 29 and describes the study population as liver transplant subjects.
Intervention
Participants were to discontinue CellCept and switch to Myfortic. The intervention description specifies Myfortic 360mg or 720 mg BID for 90 days.
Comparator
There was no concurrent comparison group in the registry design: the study is listed as single-group, with allocation recorded as NA. The proposed comparison is with participants’ experience on CellCept, not with a separately assigned control arm.
Primary question
The study asked whether Myfortic would be tolerable and safe for liver transplant recipients, with gastrointestinal symptoms assessed using the total score of the Gastrointestinal Symptom Rating Scale (GSRS). Its stated hypothesis was that Myfortic would have fewer gastrointestinal side effects and comparable effectiveness to CellCept. The registry record does not report results that establish either claim.
The study is described as prospective and focuses on people already receiving CellCept who experienced gastrointestinal side effects. Its stated objective is to assess tolerability and safety after conversion to Myfortic. The primary outcome is a continuous symptom score measured at screening and at follow-up time points. However, the registry record does not report the observed GSRS scores, changes over time, or an analysis comparing symptoms on Myfortic with symptoms on CellCept. The clinical question can therefore be described from the planned design and hypothesis, but the posted record does not answer whether symptoms changed after the switch or whether the two medicines had comparable effectiveness.
3. Trial Design
This is a prospective, single-group study listed as completed. The registry record identifies the sponsor as the University of Pittsburgh, the condition as immunosuppression, and enrollment as 29. Phase is listed as NA. Allocation is also listed as NA, and masking is listed as none. The study began in 2006-11, with primary completion in 2008-11.
All subjects were assigned to the intervention described as Myfortic. The arm description states that subjects would receive Myfortic 360mg or 720 mg BID for 90 days. Participants were selected from liver transplant recipients receiving CellCept who had gastrointestinal side effects, according to the study summary. There is no separate CellCept arm or other concurrent control group in the registry design. Thus, the design does not provide a randomized, like-for-like comparison between treatment groups.
The primary outcome was the total score of the GSRS, with assessments scheduled at screening, 2, 6 and 12 weeks. The record lists three outcome measures but no analyses. It does not report how participants were assigned to either stated Myfortic dose, whether dose selection changed during the study, or what analysis was planned for repeated assessments. Those details are not reported in the registry record.
The study’s status is completed, but completion status alone does not provide evidence about symptom changes, safety, or effectiveness. The numerical outcome results and the study’s statistical analysis are 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.
Before assignment: 30 participants were screened only 29 participants were enrolled
Overall Study
| Stage | Liver Transplant Subjects |
|---|---|
| STARTED | 30 |
| COMPLETED | 26 |
| NOT COMPLETED | 4 |
Reasons for not completing this period:
| Reason | Liver Transplant Subjects |
|---|---|
| diarrhea | 1 |
| nausea and upset stomach | 1 |
| Physician Decision | 1 |
| screen failed | 1 |
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: 30 potential participants were screened but only 29 enrolled.
| Characteristic | Unit | Liver Transplant Subjects |
|---|---|---|
| Participants analysed | 29 | |
| Age, Continuous | years | 52.2 (SD 13.0) |
| Gender: Female | Participants | 13 |
| Gender: Male | Participants | 16 |
| Region of Enrollment: United States | participants | 29 |
| Abdominal Pain- screening | units on a scale | 10.7778 (SD 5.09399) |
| Reflux screening | units on a scale | 5.0000 (SD 3.67423) |
| Indigestion screening | units on a scale | 12.6800 (SD 7.11056) |
| Diarrhea screening | units on a scale | 10.3200 (SD 5.57315) |
| Constipation screening | units on a scale | 7.2000 (SD 4.52769) |
6. Primary Endpoint
The registry lists the primary outcome as Gastrointestinal Side Effects and Quality of Life (Total Score of GSRS), measured over screening, 2, 6 and 12 weeks.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | units on a scale |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is the total score of the Gastrointestinal Symptom Rating Scale (GSRS), a measure of gastrointestinal symptoms. The record describes 15 items, each rated on a seven-point scale from 1= No discomfort at all to 7= Very severe discomfort. The total score ranges from a minimal value of 15, representing no discomfort at all, to a maximum of 105, representing very severe discomfort. The endpoint is specified as a mean, with standard deviation as the measure of dispersion.
The GSRS covers abdominal symptoms, reflux, diarrhea, indigestion and constipation. The registry description says the item groupings are based on a factor analysis. In this study, a total score was intended to track gastrointestinal symptom burden around the conversion from CellCept to Myfortic. The scheduled assessments were screening, 2, 6 and 12 weeks.
As a general statistical rationale, a continuous symptom score can describe the average level of reported symptoms and can be followed over time. The standard deviation describes how spread out individual scores are around the mean; it is not a measure of uncertainty in the mean. Interpreting a change requires the actual scores, the number of observations at each assessment, and an analysis that accounts for the study design. Those outcome values and analysis details are not reported in the registry record, so the size and precision of any change cannot be assessed from this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a planned evaluation of gastrointestinal symptoms after switching from CellCept to Myfortic, but it does not report the primary outcome results. No mean GSRS scores, standard deviations, changes between assessments, confidence intervals, or p-values are reported in the registry record. The record also lists no analyses. Consequently, there is no numerical estimate of the observed symptom pattern to interpret and no reported inferential analysis to assess.
The endpoint schedule included screening, 2, 6 and 12 weeks. These time points indicate when assessments were intended, not what participants experienced. Without scores at those assessments, it is not possible to tell whether symptoms improved, worsened, or remained similar after conversion. Nor can the precision of a change be evaluated. A confidence interval, in general, expresses the uncertainty around an estimated effect; no such interval is reported for the GSRS outcome.
The study is a single-group design, not a randomized comparison. Any observed before-and-after pattern, if it were reported, would describe change over time among participants receiving Myfortic. It would not by itself isolate the effect of switching from other possible explanations for a change. A concurrent control group and random assignment can help support a treatment comparison; neither is part of the design recorded. The registry’s stated hypothesis about fewer gastrointestinal side effects and comparable effectiveness is a hypothesis, not a reported finding.
The record describes a population with gastrointestinal side effects while receiving CellCept. This makes the planned symptom assessment relevant to the study objective, but the record does not report entry scores or participant-level data. It is also not possible to assess whether results were consistent across the stated Myfortic doses, because dose-specific outcomes are not reported in the registry record.
The adverse-event record lists a serious-event group with 30 at risk and 0 affected, and an other-event group with 30 at risk and 0 affected. These entries should be interpreted as registry counts, not as a complete account of all possible adverse effects. The record does not provide event terms, follow-up completeness, or an explanation for the listed at-risk count in relation to the enrollment figure of 29.
In short, the posted record describes the question, design, outcome scale, schedule, and certain adverse-event counts. It does not provide the outcome data needed to estimate symptom change or determine its precision. The completion status does not substitute for those results, and no conclusion about comparative effectiveness can be drawn from the information reported.
9. Statistical Methods Explained
The registry identifies the primary outcome as a continuous measure summarized by a mean and standard deviation. In general, a mean describes the average score in the group being analyzed. A standard deviation describes variation among individual scores around that average. These summaries answer different questions: the mean locates the group’s average score, while the standard deviation describes how dispersed participants’ scores are. Neither quantity is reported for the primary outcome in the registry record.
What would a change in the mean describe?
For a continuous outcome measured at several times, an analysis might describe average scores at each assessment or estimate change over time. In general, an estimated change is most informative when its uncertainty is also reported, for example with a confidence interval. A confidence interval gives a range of values compatible with the data and analysis under its assumptions; a narrower interval indicates greater precision than a wider one. The registry lists screening, 2, 6 and 12 weeks as assessment times, but the observed means, changes and confidence intervals are not reported in the registry record.
The study has one treatment group. Therefore, a within-group change would not be equivalent to a randomized treatment contrast. General statistical interpretation distinguishes change observed after an intervention from change caused by that intervention. Without a concurrent control group, other influences over time cannot be separated from the intervention using a between-group comparison. This is especially important when a study’s hypothesis refers to a comparison with the prior treatment: the registry describes CellCept use before conversion but does not report a formal comparative analysis.
Why does the analysis population matter?
In general, analysis results depend on which participants and observations are included. A complete-case analysis uses participants with observed data for the relevant assessment, while other approaches may use additional information to address missing outcomes. Each approach relies on assumptions. The registry record does not report the analysis population, the number of observed GSRS scores at each time, or how missing measurements were handled. Therefore, the completeness of follow-up and the potential influence of missing data cannot be assessed.
The planned outcome is measured repeatedly in the same study population. As a general matter, measurements from the same participant may be related; analyses of repeated observations can account for that dependence. The record does not specify whether the planned analysis treated time points separately, modeled repeated measurements, or used another approach. It is not appropriate to assume a particular statistical method from the assessment schedule alone.
What do significance tests and sample-size illustrations mean?
As a general concept, a p-value describes how surprising data would be under a specified null hypothesis and statistical model. It does not measure the probability that a hypothesis is true, nor does it establish clinical importance. No p-value or test result for the primary outcome is reported in the registry record.
The registry’s sample-size assumptions describe a planning illustration based on an observed effect: a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect to detect expressed as a stated fraction of the observed effect. The record also distinguishes participants per arm for outcomes other than time-to-event outcomes. These assumptions describe an illustration, not the recorded single-group design. They are not evidence that the study used this approach for its own planning, and the sample-size justification is not reported in the registry record. Any sample-size illustration should be treated as planning information, not observed power or evidence about the outcome.
For a mean-based endpoint, general analysis assumptions can include how the outcome is distributed and whether the model adequately represents its variability. Over-dispersion, in general, means that observed variation exceeds what a chosen model assumes; accounting for it can affect standard errors and uncertainty. The registry does not report a model, distributional checks, or adjustments for variability. No recalculated outcome estimates are available because the record provides no primary outcome summary statistics.
10. Multiplicity, Missing Data and Analysis Populations
The primary endpoint was assessed at screening, 2, 6 and 12 weeks. Multiple assessments create choices about which time point or contrast is the main inferential focus. If several tests are conducted, the chance of at least one apparently notable result can increase; multiplicity methods are designed to address that issue when appropriate. The registry record does not report a multiplicity plan, a primary follow-up contrast, or whether the repeated assessments were analyzed jointly. It also lists three outcome measures, but details of their testing and any adjustment are not reported in the registry record.
Missing data are another important consideration for a repeated symptom measure. If some participants lack scores at particular assessments, the group mean at each time may reflect a different set of observed participants. General methods for handling missing outcomes rely on assumptions about why observations are missing and how missingness relates to observed or unobserved scores. The registry record does not report the number of available GSRS observations at each scheduled assessment, reasons for missingness, or the method used to address missing data.
The analysis population is not reported in the registry record. It is therefore unclear which participants were included in any outcome analysis, whether all enrolled participants were analyzed, or whether any exclusions occurred. The registry lists enrollment as 29, but this does not establish the number contributing data at any particular assessment. The record lists no analyses and does not provide outcome estimates with which to evaluate the effect of multiplicity or missing-data decisions.
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 |
|---|---|---|---|
| Liver Transplant Subjects | 0 | 30 | 0.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Liver Transplant Subjects | 0 of 30 |
The adverse-event record lists the Liver Transplant Subjects group with 30 at risk and 0 affected for serious events. It also lists 30 at risk and 0 affected in the other-event category. No serious-event terms are listed. These figures are registry entries and should be read within the scope of the information recorded, rather than as proof that no adverse events of any kind occurred.
The death count is not reported in the registry record. The record does not describe the definitions used for the event categories, the completeness of event collection, or the follow-up time represented by these counts. It also lists enrollment as 29 while the adverse-event group has 30 at risk; the reason for that difference is not reported in the registry record. The counts should not be silently reconciled or treated as a statement about distinct participants.
In general, a count of affected participants is not enough to characterize the nature, timing, severity, or duration of events. Here, the posted adverse-event details do not provide event terms beyond the serious-event category heading. Accordingly, the figures can be reported as recorded, but they cannot support a broader description of safety or establish the absence of risk.
12. What the Result Does, and Does Not, Mean
The record establishes that investigators planned a prospective study of conversion from CellCept to Myfortic in liver transplant recipients with gastrointestinal side effects. It identifies a single-group design, a Myfortic intervention, a GSRS total score as the primary outcome, and assessment times at screening, 2, 6 and 12 weeks. It also records the study as completed and lists adverse-event counts.
It does not report the GSRS results. There is therefore no posted estimate of average symptom burden, no estimate of change after conversion, and no measure of the precision of any such change. Without these data, the registry record does not establish whether gastrointestinal symptoms changed or whether any observed change would be large or precise.
The stated hypothesis concerns fewer gastrointestinal side effects and comparable effectiveness relative to CellCept. The design recorded is not a randomized comparison with a concurrent CellCept group, and no comparative effectiveness results are reported. The hypothesis should not be mistaken for a result. A completed study status indicates the registry’s status entry, not that a particular claim was supported.
The listed adverse-event counts are limited registry information. They do not describe event types, and the death count is not reported in the registry record. The at-risk count also differs from the enrollment figure, with no explanation recorded. Those limitations prevent a comprehensive interpretation of safety from these entries alone.
Thus, the record is informative about the study’s intended question and measurement plan, but not about the observed primary outcome or a comparative treatment effect. It should be read as a description of the registered design and available results, with the absence of reported outcome analyses kept distinct from evidence of no effect.
13. Why This Trial Matters Statistically
This study raises a useful statistical distinction between a clinical hypothesis and the evidence needed to evaluate it. The hypothesis concerns symptom changes after switching treatments and comparable effectiveness relative to CellCept. The registered design is single-group, so it does not include a concurrent randomized comparator. That distinction matters because a before-and-after pattern, even if available, would not by itself provide the same treatment contrast as a controlled comparison.
The endpoint also illustrates why measurement plans and outcome results are not interchangeable. The record describes the GSRS content, scale range, summary measure and assessment schedule in detail. Those elements define what investigators intended to measure, but they do not reveal the actual scores or their uncertainty. Without the numerical results and an analysis, the reader cannot judge the magnitude or precision of symptom change.
Repeated assessments make analysis choices important. General questions include how observations from the same participant are handled, how missing scores are addressed, and how several assessments or outcomes are interpreted. The registry record does not report those choices. Their absence limits reproducibility and makes it difficult to assess how robust any result would be, even though no primary outcome result is posted.
The adverse-event entries also show why denominators and record consistency deserve attention. The listed at-risk count is not the same as the enrollment figure, and no explanation is reported. This should prompt cautious reading rather than an unrecorded reconciliation. Overall, the study is statistically instructive as an example of a defined clinical question and outcome schedule with insufficient posted outcome analysis to answer that question.
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.
Several limitations are specific to the information recorded for this study. The registry identifies the study as single-group and lists no randomized comparison. As a result, the design does not provide a concurrent control group for distinguishing a change after conversion from other changes over time. The stated comparison with CellCept is part of the study hypothesis, not a reported controlled contrast.
The registry record does not report primary outcome values, standard deviations, estimates of change, confidence intervals, or p-values. It also lists no analyses. The effect and its precision therefore cannot be evaluated. Although the GSRS assessments were scheduled at screening, 2, 6 and 12 weeks, the observed data at those times are not reported in the registry record.
The record does not describe the analysis population, missing-data handling, or approach to repeated measurements. It does not report a multiplicity plan despite listing three outcome measures and multiple assessment times. These omissions prevent assessment of important analytic choices and the completeness of follow-up.
The Myfortic arm description includes 360mg or 720 mg BID for 90 days, but dose-specific assignment and outcomes are not reported in the registry record. The sample-size justification and the study’s own statistical plan are also not reported in the registry record. Planning assumptions recorded for an illustration should not be mistaken for evidence about how this study was designed or analyzed.
Finally, the adverse-event entry lists 30 at risk, while enrollment is listed as 29. The reason is not reported in the registry record. Serious-event terms are absent, and the death count is not reported in the registry record. These limitations constrain interpretation of both efficacy-related outcomes and safety.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Conversion of CellCept to Myfortic: A Prospective Study in Liver Transplant Recipients — NCT00336895.
- Registry record last updated 2016-11-07; status last verified 2016-11.
- Results first posted 2016-11-07.
- 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.
18. Record Summary
NCT00336895 is a completed, prospective, single-group study of conversion from CellCept to Myfortic in liver transplant recipients with gastrointestinal side effects. The registry lists enrollment as 29 and identifies the University of Pittsburgh as sponsor. The intervention description specifies Myfortic 360mg or 720 mg BID for 90 days.
The primary outcome is the total GSRS score, summarized as a mean with standard deviation and scheduled at screening, 2, 6 and 12 weeks. The record lists three outcome measures and no analyses. Primary outcome results and the statistical plan are not reported in the registry record.
The adverse-event entry lists 30 at risk and 0 affected in both serious and other-event categories; the death count is not reported in the registry record. The reason the adverse-event at-risk count differs from enrollment is not reported in the registry record. The posted information describes the study question and design but does not provide outcome estimates that answer its hypothesis.