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 A Study of CellCept (Mycophenolate Mofetil) in Patients With Lupus Nephritis. (NCT00425438), a randomized phase 3 study. The primary outcome is Complete Response (CR) by the End of Treatment - Percentage of Participants With an Event.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00425438 |
| Phase | Phase 3 |
| Condition | Lupus Nephritis |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 52 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Hoffmann-La Roche |
| Results posted | Yes |
2. Clinical Question
The trial was designed to compare two treatment strategies for people with lupus nephritis. One strategy used CellCept, also called mycophenolate mofetil, with corticosteroids. The other used cyclophosphamide with corticosteroids during induction, followed by azathioprine with corticosteroids during maintenance. The primary question concerned the percentage of participants with complete response by the end of treatment.
Population
Patients with lupus nephritis were eligible for the study. The registry record reports enrollment of 52 participants.
Intervention
Mycophenolate mofetil plus corticosteroids. The study summary describes a planned induction period followed by a maintenance period using mycophenolate mofetil with corticosteroids.
Comparator
Cyclophosphamide plus corticosteroids during induction, followed by azathioprine plus corticosteroids during maintenance.
Primary question
What percentage of participants achieved complete response by the end of treatment under each strategy?
Complete response was defined using urinary protein, urine findings, and serum creatinine relative to baseline. The registry record gives the definition, but reports that efficacy data were not analyzed because the study was terminated early for administrative reasons. No participants completed all planned study visits, so the intended primary comparison is unavailable. The record therefore does not establish which strategy had a higher response rate or how uncertain any difference might have been.
3. Trial Design
This was a randomized, open-label, parallel-group Phase 3 study sponsored by Hoffmann-La Roche. Participants were assigned to one of two treatment strategies. The registry record lists 52 participants as enrolled. The study started in 2007-03, and primary completion is recorded as 2008-04. Its status is terminated.
The planned comparison was between mycophenolate mofetil plus corticosteroids and cyclophosphamide plus corticosteroids followed by azathioprine. The study summary describes a 24-week induction phase and a subsequent 24-week maintenance phase. It says response rate would be assessed at the end of induction and at the end of the study. These are descriptions of the study plan, not evidence that participants completed those treatment periods or assessments.
Randomization is intended to reduce systematic differences between treatment groups at assignment. In an open-label study, participants and study personnel are not masked to assigned treatment. Open-label status can matter when outcomes involve clinical judgments or decisions, although the registry record does not report whether any particular assessment was affected in this trial. The parallel design means participants were assigned to one strategy rather than crossing between strategies.
The registry record does not report a detailed randomization procedure, allocation concealment method, or a statistical analysis plan. It also does not explain the administrative reason for termination beyond identifying it as administrative. The record’s stated target sample size was 100-500 individuals, while enrollment was 52. These figures describe the target and the eventual enrollment; they do not, on their own, explain the study’s statistical planning or the consequences of early termination.
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 | Mycophenolate Mofetil | Cyclophosphamide, Azathioprine |
|---|---|---|
| STARTED | 25 | 27 |
| COMPLETED | 0 | 0 |
| NOT COMPLETED | 25 | 27 |
Reasons for not completing this period:
| Reason | Mycophenolate Mofetil | Cyclophosphamide, Azathioprine |
|---|---|---|
| Termination by Sponsor | 25 | 27 |
Analysis population for the primary outcome, as defined in the registry: Efficacy data were not analyzed because of study termination. The study was terminated early for administrative reasons and no participants completed all planned study visits; therefore efficacy analyses could not be performed.
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: Safety population: all participants who recieved at least one dose of study drug.
| Characteristic | Unit | Mycophenolate Mofetil | Cyclophosphamide, Azathioprine | Total |
|---|---|---|---|---|
| Participants analysed | 25 | 27 | 52 | |
| Age, Continuous | years | 33.4 (SD 9.8) | 31.6 (SD 8.0) | 32.4 (SD 8.9) |
| Sex: Female, Male: Female | Participants | 22 | 23 | 45 |
| Sex: Female, Male: Male | Participants | 3 | 4 | 7 |
6. Primary Endpoint
The registry lists the primary outcome as Complete Response (CR) by the End of Treatment - Percentage of Participants With an Event, measured over Screening, Day 0, Weeks 2, 4, 8, 12, 16, 20, 24, 32, 40, and 48.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was the percentage of participants with a complete response by the end of treatment. Complete response required urinary protein below 500 mg per 24 hours, no hematuria or cellular casts in urine, and a stable serum creatinine within ±25% of baseline or some improvement. The registry record lists assessments across screening, Day 0, and scheduled study weeks through Week 48.
In general, a response endpoint combines clinical measurements into a rule for classifying participants. A rule can make the outcome easier to interpret as the proportion meeting a defined response criterion, but its meaning depends on the exact components and thresholds. Here, the definition uses kidney-related urine findings and serum creatinine relative to baseline. The record does not report separate primary results for those components.
The percentage was intended to compare the frequency of complete response between the treatment strategies. However, the registry record states that efficacy data were not analyzed because the study ended early and no participants completed all planned study visits. Thus, the outcome’s definition is available, but an analyzed primary response percentage or treatment comparison is not reported in the registry record. The stated assessment schedule should not be read as proof that every scheduled assessment was completed.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The main statistical finding is the absence of an analyzed efficacy result. The registry record explicitly says efficacy data were not analyzed because the study was terminated early for administrative reasons and no participants completed all planned study visits. Accordingly, there is no reported primary response percentage, estimated treatment difference, confidence interval, or hypothesis test to interpret. The record does not show whether either strategy produced a higher complete-response rate.
Enrollment was 52, whereas the stated target sample size was 100-500 individuals. This indicates that enrollment did not reach the stated target range. It does not identify why the target was set, whether recruitment stopped before or after a planned milestone, or what precision would have been expected under the original plan. Those details are not reported in the registry record.
The safety summaries are descriptive and should be read separately from the unavailable efficacy analysis. The mycophenolate mofetil group lists 25 participants at risk for serious adverse events and 8 affected; the cyclophosphamide/azathioprine group lists 27 at risk and 5 affected. For other adverse events, the corresponding counts are 25 and 13, and 27 and 18. These are counts within groups, not adjusted comparisons. The registry record does not provide a statistical comparison or uncertainty interval for them. The number of deaths is not reported in the registry record.
In general, an estimate describes the observed result, while a confidence interval describes the range of values compatible with the data and the analysis assumptions. Neither can be interpreted for the primary endpoint without a reported efficacy estimate and its uncertainty. Likewise, a p-value cannot substitute for an effect estimate or recover an analysis that was not performed. No primary efficacy p-value is reported in the registry record.
The registry lists secondary outcomes including response, decreases in glomerular filtration rate, treatment termination, and time to treatment failure. Their presence in the outcome list does not establish that results were analyzed. The registry record reports six outcome measures overall, but does not provide inferential analyses. The defensible conclusion is therefore about the limits of the available results: the intended efficacy comparison cannot be evaluated from the registry record, while the posted adverse-event counts can be described without treating them as proof of a treatment difference.
9. Statistical Methods Explained
The registry identifies a randomized, parallel-group study with no masking. It does not report enough detail to reconstruct the statistical methods used or planned. The concepts below explain how such methods are generally understood; they should not be mistaken for procedures documented for this trial.
What randomization contributes
In general, random assignment aims to make treatment groups comparable at the start of a trial with respect to measured and unmeasured characteristics. It supports a fair comparison when participants are analyzed according to an appropriate plan and follow-up is sufficiently complete. Randomization does not guarantee balance in a particular study, especially when the number of participants is limited. The registry record does not describe the random sequence generation, allocation concealment, or baseline comparisons for this trial.
How a response proportion is interpreted
For a binary endpoint such as complete response, each participant is classified as meeting or not meeting the stated response criteria, if the needed observations are available. A group response proportion describes the share classified as responders under the analysis definition. Comparing such proportions can be done using several statistical approaches. The approach depends on the study plan, data structure, and assumptions. The registry record does not report the method for estimating or comparing response proportions, and it reports that efficacy analyses were not performed.
In general, an effect estimate gives the direction and size of a treatment contrast, while a confidence interval communicates its precision under the chosen method. A wide interval indicates that the data are compatible with a broad range of effects; a narrow interval indicates greater precision. A p-value addresses compatibility with a specified null hypothesis under model assumptions, but does not measure clinical importance. No primary effect estimate, confidence interval, or p-value is reported for this trial, so these quantities cannot be interpreted for its primary endpoint.
Why follow-up and missing outcomes matter
General statistical practice distinguishes an outcome that was not measured from an outcome that was measured but not reported or not included in an analysis. Early termination can leave planned measurements unavailable and can reduce the information available for estimating treatment effects. Missingness may also differ across participants or groups. The registry record says no participants completed all planned visits and that efficacy data were not analyzed. It does not give the number of participants with each measurement, reasons for missed visits, or methods for handling missing data.
Analysis populations are another important part of trial interpretation. For example, a plan may define whether all randomized participants are included, or whether analysis is restricted according to treatment received or availability of outcomes. Different choices can answer different questions and can be affected differently by incomplete follow-up. The registry record does not report a defined efficacy analysis population. The safety group counts are reported separately and should not be assumed to identify the efficacy population.
Multiplicity and repeated assessments
Multiplicity arises when several outcomes, contrasts, or assessment times are tested. In general, conducting many tests can increase the chance of at least one apparently notable result if no adjustment or prespecified testing strategy is used. A study may address this through a hierarchy of outcomes, adjusted significance thresholds, or another stated procedure. The registry record lists one primary outcome with a schedule of assessments and several secondary outcomes, but does not report a multiplicity strategy. Because no efficacy analyses were performed, there are no reported primary or secondary efficacy tests to interpret.
Dispersion and model choice
For outcomes that count events or measure time until an event, the distribution of observations and the amount of variation can influence model choice. In general, over-dispersion means that count data vary more than a simple model assumes; ignoring it can make uncertainty estimates unreliable. Time-to-event analysis also depends on event timing and censoring information. The registry lists time to treatment failure among secondary outcomes, but does not report an analysis method, event count, or estimate for it. No claim about a particular model or its assumptions is supported by the registry record.
Planning calculations are not trial results
A separate planning illustration uses a two-sided significance level of 0.05, equal allocation, a normal approximation, and effects defined as fractions of an observed effect. For time-to-event planning it uses a total event count, while other planning quantities are expressed as participants per arm. These are planning assumptions, not a description of the trial’s original design, and they are not observed power. The registry record does not report the trial’s sample-size justification or the methods used to choose its stated target range. Since no primary efficacy analysis was performed, the planning illustration should not be treated as evidence of the trial’s observed treatment effect.
10. Multiplicity, Missing Data and Analysis Populations
The trial included a primary complete-response outcome and multiple secondary outcomes. The primary outcome was scheduled for assessment at several visits, while the secondary outcomes included response, glomerular filtration rate decreases, treatment termination, and time to treatment failure. In general, examining several outcomes or time points creates a multiplicity question: a statistical plan should clarify which comparisons are primary and whether any adjustment or testing hierarchy will be used. The registry record does not report such a strategy.
The record says efficacy data were not analyzed because the study was terminated early for administrative reasons, and that no participants completed all planned study visits. Consequently, the issue is not merely that a reported efficacy analysis used one missing-data method rather than another: no efficacy analyses are reported. The number of participants with usable observations at each visit, the reasons for incomplete follow-up, and any imputation or other missing-data procedures are not reported in the registry record.
In general, missing outcomes can affect both the number of observations contributing to an estimate and the validity of the comparison. The consequences depend on why data are missing and on assumptions used in the analysis. Incomplete planned follow-up does not establish that no observations were collected at all; it means the record does not document completion of the planned visit schedule, and it explicitly says efficacy analyses could not be performed.
The registry record also does not define an efficacy analysis population or say how participants were to be handled if they stopped treatment, switched treatment, or missed assessments. It lists safety counts by group, but those counts should not be treated as a substitute for an efficacy population or analysis.
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 |
|---|---|---|---|
| Mycophenolate Mofetil | 8 | 25 | 32.0% |
| Cyclophosphamide, Azathioprine | 5 | 27 | 18.5% |
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 |
|---|---|
| Mycophenolate Mofetil | 13 of 25 |
| Cyclophosphamide, Azathioprine | 18 of 27 |
Most frequent serious adverse event terms
| Event term | Organ system | Mycophenolate Mofetil | Cyclophosphamide, Azathioprine |
|---|---|---|---|
| Lung infection | Infections and infestations | 1 of 25 | 2 of 27 |
| Pyrexia | General disorders | 1 of 25 | 1 of 27 |
| Gastroenteritis | Gastrointestinal disorders | 1 of 25 | 1 of 27 |
| Nausea | Gastrointestinal disorders | 1 of 25 | 0 of 27 |
| Vomiting | Gastrointestinal disorders | 1 of 25 | 0 of 27 |
| Liver disorder | Hepatobiliary disorders | 0 of 25 | 1 of 27 |
| Infection | Infections and infestations | 1 of 25 | 0 of 27 |
| Urinary tract infection | Infections and infestations | 1 of 25 | 0 of 27 |
| Acute renal failure | Renal and urinary disorders | 1 of 25 | 0 of 27 |
| Herpes zoster | Skin and subcutaneous tissue disorders | 1 of 25 | 0 of 27 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Mycophenolate Mofetil | Cyclophosphamide, Azathioprine |
|---|---|---|---|
| Vomiting | Gastrointestinal disorders | 0 of 25 | 4 of 27 |
| Upper respiratory tract infection | Infections and infestations | 1 of 25 | 3 of 27 |
| Alanine aminotransferase increased | Investigations | 1 of 25 | 3 of 27 |
| Palpitations | Cardiac disorders | 0 of 25 | 3 of 27 |
| Nausea | Gastrointestinal disorders | 2 of 25 | 1 of 27 |
| Pyrexia | General disorders | 3 of 25 | 0 of 27 |
| Dizziness | Nervous system disorders | 2 of 25 | 1 of 27 |
| Cough | Respiratory, thoracic and mediastinal disorders | 3 of 25 | 0 of 27 |
| Oedema peripheral | Cardiac disorders | 1 of 25 | 1 of 27 |
| Abdominal distension | Gastrointestinal disorders | 2 of 25 | 0 of 27 |
Serious events with the highest arm-level rate: lung infection (7.4%), pyrexia (4.0%), gastroenteritis (4.0%), nausea (4.0%), vomiting (4.0%).
The registry reports serious adverse events for 25 participants at risk in the mycophenolate mofetil group, with 8 affected, and for 27 participants at risk in the cyclophosphamide/azathioprine group, with 5 affected. For other adverse events, the reported counts are 13 affected among 25 at risk and 18 affected among 27 at risk, respectively. These are descriptive group counts. The registry record does not report an adjusted comparison, confidence interval, or statistical test for these figures.
The listed serious-event terms and percentages are Lung infection, 7.4%; Pyrexia, 4.0%; Gastroenteritis, 4.0%; Nausea, 4.0%; Vomiting, 4.0%; and Infection, 4.0%. The record does not specify in this listing how these terms are distributed by treatment group, so group attribution should not be inferred. The percentages should be read as posted rather than converted into counts or treated as mutually exclusive categories.
In general, adverse-event counts describe events observed and recorded during the available follow-up; they do not by themselves establish that treatment caused an event or that one treatment differs from another. The number of deaths is not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The record establishes that a randomized comparison of two lupus nephritis treatment strategies was initiated and that 52 participants were enrolled. It also documents that the study was terminated early for administrative reasons. The intended primary outcome was complete response by the end of treatment, defined using urinary protein, urine findings, and serum creatinine relative to baseline.
It does not establish an efficacy result. The registry record states that efficacy data were not analyzed and that no participants completed all planned study visits. There is therefore no reported primary response rate, treatment contrast, confidence interval, or p-value from which to assess the direction, magnitude, or precision of an efficacy difference. The absence of an analyzed result is not evidence that the treatment strategies were equivalent, nor is it evidence that one was better.
The safety counts provide a descriptive record of participants affected by serious and other adverse events in each group. Without an inferential comparison and fuller information about event definitions, follow-up, and analysis, those counts should not be interpreted as establishing a causal difference between strategies. The reported serious-event terms and percentages add detail about recorded events, but the listing does not give their treatment-group distribution. Deaths are not reported in the registry record.
In general, a trial’s planned design and its completed analyses are distinct. A protocol can specify outcomes and assessment times, but those plans do not become evidence of an outcome when the relevant analysis is not available. Likewise, an enrollment target is a planning quantity, not a guarantee that the study will produce a precise estimate. Here, the stated target range was 100-500 individuals, while enrollment was 52; the record does not report the original statistical justification for the target.
The appropriate reading is limited: the registry documents the intended comparison and the early termination, while leaving the primary efficacy question unanswered. It cannot support a claim about comparative efficacy or equivalence. The posted safety counts can be described as observed registry summaries, with the limitations noted above.
13. Why This Trial Matters Statistically
This trial illustrates an important distinction between a planned clinical question and an answer supported by analyzed data. The registry gives a clear primary outcome definition and identifies the two treatment strategies, but early termination meant that the intended efficacy analysis was not performed. A design description alone cannot answer the comparative question.
From a statistical perspective, incomplete follow-up and a smaller-than-target enrollment can limit the amount of information available. In general, less information tends to reduce precision, but the record does not report an efficacy estimate or its uncertainty, so the actual precision of a treatment contrast cannot be quantified. The stated target range also does not reveal the statistical assumptions or justification used when planning the study.
The case also highlights why trial status and analysis availability need to be considered together. The study is recorded as terminated, and the primary completion date is 2008-04; however, no participants completed all planned study visits and efficacy data were not analyzed. Those details make it important to distinguish administrative completion milestones from the availability of a completed endpoint analysis.
Finally, the safety summaries demonstrate the limits of descriptive counts. Counts can convey what was recorded in each group, but without a reported comparative analysis and sufficient context they do not establish a treatment effect. The most useful statistical conclusion is therefore not a ranking of the strategies, but a precise account of what the record does and does not allow readers to infer.
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 central limitation is that the registry record reports no efficacy analysis. No participants completed all planned study visits, and the study ended early for administrative reasons. The primary endpoint is defined, but an analyzed response rate and comparative estimate are absent. It is therefore not possible to assess the magnitude or precision of the intended efficacy contrast from the registry record.
Enrollment was 52, compared with a stated target range of 100-500 individuals. The record does not explain the sample-size justification, recruitment progress, or whether early termination was related to enrollment. It also does not report the detailed statistical analysis plan, methods for handling missing outcomes, or a defined efficacy analysis population.
The study was open label, and the record does not report masking or independent assessment procedures beyond identifying masking as none. Open-label designs can raise concerns about assessment or management decisions in general, but the registry record does not establish that such effects occurred. Randomization is reported, while details of sequence generation and allocation concealment are not reported in the registry record.
The safety information is limited to group counts and a list of serious-event terms with percentages. The record does not provide comparative uncertainty estimates for adverse events, the group attribution of the listed terms, or a reported death count. These limitations prevent stronger interpretation of the safety summaries.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study of CellCept (Mycophenolate Mofetil) in Patients With Lupus Nephritis. — NCT00425438.
- Registry record last updated 2014-09-25; status last verified 2014-09.
- Results first posted 2014-09-25.
- 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
This Phase 3 study was a randomized, open-label, parallel-group comparison in patients with lupus nephritis. It compared mycophenolate mofetil plus corticosteroids with cyclophosphamide plus corticosteroids followed by azathioprine. The registry lists 52 participants enrolled, a start date of 2007-03, primary completion in 2008-04, and a status of terminated.
The primary outcome was the percentage of participants with complete response by the end of treatment. The registry record states that the study was terminated early for administrative reasons, no participants completed all planned study visits, and efficacy data were not analyzed. The primary efficacy comparison is therefore unavailable. Descriptive serious and other adverse-event counts are reported by group; deaths are not reported in the registry record.