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 Effects of Mycophenolate Mofetil (MMF) On Anti-HLA (Human Leukocyte Antigen)Antibody Levels In Patients Awaiting Cadaveric Renal Transplant. (NCT00446459), a non-randomized phase 2 study. The primary outcome is The Number of Subjects With a 10% Decrease in PRA Level at Month 8..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00446459 |
| Phase | Phase 2 |
| Condition | Kidney Failure, Chronic, Diabetic Nephropathies, Glomerulonephritis, IGA |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 45 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Washington |
| Results posted | Yes |
2. Clinical Question
Population
Patients on the kidney transplant list with high Panel of Reactive Antibody (PRA) levels. The registry lists chronic kidney failure, diabetic nephropathies, IgA glomerulonephritis, and renal hypertension among the conditions. Specific eligibility criteria and the threshold used to define high PRA are not reported in the registry record.
Intervention
Mycophenolate mofetil (MMF), also identified as CellCept. The registry describes the study as prospective, open label, and 12 months long. The dose and administration schedule are not reported in the registry record.
Comparator
There is no separate comparator group in the listed design. The study is described as non-randomized and single group, so it does not provide a like-for-like control comparison.
Primary question
How many participants had a 10% decrease in PRA level by month 8? The endpoint is listed as a participant count from enrollment to month 8. The registry does not report the count or further specify how the 10% decrease was defined.
The question concerns change in an antibody-related measure among people awaiting transplantation, rather than a randomized comparison of MMF with another treatment or with no treatment. The registry summary describes high PRA as relevant to waiting time, but the study record does not report a comparison of waiting times or establish that any change in PRA altered transplantation outcomes. Secondary outcomes include infection, kidney transplantation, low white blood cell count or immunoglobulin titers below range, and negative crossmatch at transplantation. Their numerical results are not reported in the registry record.
3. Trial Design
The record describes a prospective, phase II, open-label study of mycophenolate mofetil in patients awaiting cadaveric renal transplantation. It is marked completed, with an enrollment of 45. The listed study period begins in 2006-04, and primary completion is recorded as 2008-11. The sponsor is the University of Washington.
The design is non-randomized and single group, with no masking. Participants received MMF, and the primary endpoint counts participants with a 10% decrease in PRA level by month 8. Because no separate control group is listed, the design can describe outcomes observed among participants in the study but cannot isolate the effect of MMF from other influences through a randomized between-group comparison. Changes over time may have several possible explanations, and this design alone cannot distinguish them.
The registry lists five outcome measures: the primary endpoint and four secondary outcomes. The secondary outcomes concern significant infections up to month 12, kidney transplantation up to 12 months, low white blood cell count or immunoglobulin titers below range, and negative crossmatch at transplant. The registry record does not report numerical results for these outcomes. The dose, treatment schedule, detailed eligibility criteria, and handling of treatment discontinuation are not reported in the registry record.
The record states that the study is 12 months long, while the primary endpoint is assessed at month 8. The rationale for choosing that endpoint time and the planned timing of other measurements are not reported in the registry record. Although the study status is completed, completion status does not itself provide outcome estimates or confirm that every participant completed follow-up.
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: Subjects were consented, given a physical exan and laboratory tests including serum virologies current Hepatitis B immunization and Tuberculosis (TB) testing initially. Abnormalities in white or red blood cells, platelets, TB test or signs of infection or evidence of cancer were grounds for exclusion.
8 Months
| Stage | Mycophenolate Mofetil (MMF) Single Arm Study |
|---|---|
| STARTED | 34 |
| COMPLETED | 22 |
| NOT COMPLETED | 12 |
Reasons for not completing this period:
| Reason | Mycophenolate Mofetil (MMF) Single Arm Study |
|---|---|
| Protocol Violation | 12 |
12 Months
| Stage | Mycophenolate Mofetil (MMF) Single Arm Study |
|---|---|
| STARTED | 22 |
| COMPLETED | 4 |
| NOT COMPLETED | 18 |
Reasons for not completing this period:
| Reason | Mycophenolate Mofetil (MMF) Single Arm Study |
|---|---|
| Protocol Violation | 4 |
| 14 subjects to MMF + Rituximab Study | 14 |
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 | Mycophenolate Mofetil (MMF) Single Arm Study |
|---|---|---|
| Participants analysed | 34 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 34 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age Continuous | years | 46.5 (SD 11.2) |
| Sex: Female, Male: Female | Participants | 17 |
| Sex: Female, Male: Male | Participants | 17 |
| Region of Enrollment: United States | participants | 34 |
6. Primary Endpoint
The registry lists the primary outcome as The Number of Subjects With a 10% Decrease in PRA Level at Month 8., measured over Enrollment to month 8.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Participant |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the number of participants with a 10% decrease in PRA level at month 8, measured from enrollment to month 8. It is a count of participants meeting a stated change threshold, rather than a reported average change in PRA. The registry does not further clarify whether the 10% decrease is defined as an absolute or relative change, or how measurements were handled when a month-8 value was unavailable.
A threshold-based count can describe how many participants crossed a prespecified level of change. In general, such an endpoint may be easier to interpret as a participant outcome than a group average, but it does not show the size of change for each participant or the distribution of changes. Results can also depend on the threshold and the measurement procedures. Those details are not reported in the registry record beyond the stated 10% decrease.
The endpoint is used to assess change in PRA in a population described as having high PRA while awaiting transplantation. However, the registry record does not report the number who met the endpoint, the total number included in that analysis, or an estimate of uncertainty. It also does not report whether the threshold was prespecified in a statistical analysis plan. The endpoint therefore identifies the study’s primary question, but the posted record does not provide the result needed to quantify the observed response.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a single-group study with 45 enrolled participants and a primary outcome defined as the number with a 10% decrease in PRA by month 8. It does not report the primary outcome count, an effect estimate, or a confidence interval. As a result, the posted record does not establish how many participants met the endpoint or how precisely that quantity was estimated.
The design matters for interpretation. Participants were not randomized to MMF or a control condition, and there is no separate comparator group. Any changes in PRA observed during follow-up would therefore be descriptive of this study group. Without a randomized control comparison, a before-and-after change cannot by itself show that MMF caused the change. Other influences, including natural variation and changes in participants’ circumstances, cannot be separated using the listed design alone.
The primary endpoint is a threshold count, not a mean change or a direct measure of transplantation. A participant could meet the threshold with a change close to it or with a larger change, but the registry does not report individual or distributional results. The endpoint also does not, by itself, establish whether participants received transplants or experienced other clinical outcomes. Those topics appear among the secondary outcomes, but their results are not reported in the registry record.
The adverse-event record lists 37 participants at risk for serious and other adverse events, with 2 affected in the serious category and 6 in the other category. It lists myocardial infarction and retinal stroke among the top serious terms, each at 2.7%. These figures describe the posted safety record; the registry does not provide a comparator that would support a comparative safety estimate. The number of deaths is not reported in the registry record.
Overall, the record provides design information, outcome definitions, and selected adverse-event counts, but not the numerical primary or secondary outcome results. Conclusions about the magnitude of change, uncertainty, or differences attributable to treatment cannot be drawn from the posted results. The study’s completed status should not be confused with a complete public account of its findings.
9. Statistical Methods Explained
The registry describes a prospective, non-randomized, single-group study with no masking. These design features determine what statistical conclusions are supported. The primary outcome is a count of participants meeting a threshold for PRA decrease, while the listed secondary outcomes are also participant counts or categories. The record lists five outcome measures and zero analyses, and does not report numerical results for the primary endpoint.
What does a single-group design estimate?
In general, a single-group study can describe outcomes among enrolled participants, such as the proportion who meet an endpoint if both the count and the relevant analysis denominator are reported. It cannot use random assignment to make the treatment and control groups comparable, because no control group is listed. A change from enrollment to follow-up may reflect the intervention, natural changes, measurement variation, or other events during follow-up. The design does not separate these explanations.
The record gives an enrollment of 45, but does not report the primary endpoint numerator or analysis denominator. Enrollment alone is not enough to determine how many participants had evaluable month-8 measurements or how missing measurements were handled. The statistical analysis plan, including any adjusted analyses, is not reported in the registry record.
How should a threshold endpoint be read?
In general, a threshold endpoint turns a measured change into a yes-or-no classification: a participant either meets the defined threshold or does not. The primary outcome is stated as a 10% decrease in PRA level at month 8. This presents a count question rather than a continuous summary of the amount of PRA change. Thresholds can make a result easier to express as the number meeting a criterion, but they can conceal variation within each category. The registry does not report the exact operational definition of the decrease, the measurement method, or the endpoint count.
What do uncertainty estimates add?
In general, a confidence interval describes a range of values compatible with the observed data under the statistical method used. Its width reflects the precision of an estimate; it does not remove design limitations or establish causation. No confidence interval or other uncertainty estimate for the primary endpoint is reported in the registry record. No p-value or inferential analysis for the primary endpoint is reported either.
For a count endpoint, interpretation also depends on the denominator: the number of participants assessed for the outcome. A count without that denominator does not convey how common the endpoint was among those assessed. Here, neither the count meeting the primary endpoint nor its analysis denominator is reported. Consequently, the posted record does not support a quantitative estimate of the frequency of the primary outcome.
What do the planning assumptions mean?
The registry describes planning illustrations using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with effects expressed as fractions of an observed effect. Such calculations are general planning exercises, not observed power and not evidence that the actual study used a two-arm design. The listed trial itself is single group. Sample-size illustrations are hypothetical planning quantities; they do not represent participants observed to meet the endpoint. The registry record does not report the trial’s own sample-size justification or a statistical plan that explains whether these assumptions informed the study.
In general, over-dispersion occurs when count outcomes vary more than a statistical model assumes. If present, a model that does not account for it can understate uncertainty. The registry does not report a model-based analysis of the primary endpoint, so there is no basis in the record to assess whether over-dispersion was considered or relevant to the posted analysis.
What can the adverse-event counts show?
Adverse-event counts describe events or participants recorded in the safety population, according to the registry’s reporting categories. They are not automatically comparable with rates from another study, particularly when populations, follow-up, event definitions, and ascertainment differ. The record lists 37 participants at risk in the single group, with 2 affected for serious adverse events and 6 affected for other adverse events. It also lists two serious terms at 2.7% each. The precise event-level accounting and the method used to produce those percentages are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The record lists a primary outcome and four secondary outcomes, making five outcome measures in total. The primary measure concerns a 10% decrease in PRA by month 8. The secondary measures concern significant infections, kidney transplantation, white blood cell count or immunoglobulin titers below range, and negative crossmatch at transplant. Results for these measures are not reported in the registry record.
In general, assessing multiple outcomes can increase the chance of finding an apparently notable result by chance if many separate statistical tests are performed. Methods to address this include prespecifying a primary endpoint, adjusting for multiple comparisons, or treating secondary outcomes as exploratory. The registry identifies a primary endpoint, but a multiplicity procedure, hierarchy among secondary outcomes, and statistical analysis plan are not reported in the registry record. The record lists zero analyses, so no posted inferential results are available to assess.
Missing-data handling is also not reported in the registry record. The listed enrollment is 45, while the adverse-event group reports 37 participants at risk; the record does not explain the difference between these figures. The endpoint-specific analysis denominator, missing month-8 PRA measurements, reasons for missingness, and any imputation or sensitivity analyses are not reported. The record also does not specify whether the primary analysis included all enrolled participants or a defined subset.
In general, results can depend on which participants are included and how missing outcome measurements are handled. Without a stated analysis population and missing-data approach, it is not possible to determine whether the posted outcome estimates, if available elsewhere, would represent all enrolled participants or only those with observed measurements. The registry record does not report interim analyses or stopping rules.
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 (MMF) Single Arm Study | 2 | 37 | 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 |
|---|---|
| Mycophenolate Mofetil (MMF) Single Arm Study | 6 of 37 |
Most frequent serious adverse event terms
| Event term | Organ system | Mycophenolate Mofetil (MMF) Single Arm Study |
|---|---|---|
| Myocardial infarction | Cardiac disorders | 1 of 37 |
| Retinal stroke | Cardiac disorders | 1 of 37 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Mycophenolate Mofetil (MMF) Single Arm Study |
|---|---|---|
| Anemia | Blood and lymphatic system disorders | 3 of 37 |
| Peritonitis | Infections and infestations | 2 of 37 |
| Abscess | Infections and infestations | 2 of 37 |
Serious events with the highest arm-level rate: myocardial infarction (2.7%), retinal stroke (2.7%).
The registry reports one MMF single-arm group with 37 participants at risk for serious adverse events and 2 affected. For other adverse events, it likewise lists 37 at risk and 6 affected. The top serious terms are myocardial infarction at 2.7% and retinal stroke at 2.7%. These are descriptive entries for the study group, not comparisons with an untreated or alternative-treatment group.
The serious-event count and the listed terms are different kinds of information: the count describes participants affected in the serious category, while the terms identify reported serious event types. The registry record does not explain whether the same participant could contribute to more than one term, or provide further event-level detail. The listed percentages should be read as reported; no additional rate or comparison is inferred from them.
The number of deaths is not reported in the registry record. The record also does not provide detail about event ascertainment, follow-up completeness for safety, or a statistical comparison. These entries therefore describe what is recorded for this single group, but they cannot establish relative safety or determine whether MMF caused a particular event.
12. What the Result Does, and Does Not, Mean
The study is designed to describe PRA changes among patients awaiting cadaveric renal transplantation who received MMF. Its primary endpoint asks how many participants had a 10% decrease in PRA by month 8. The registry record does not report the endpoint result, its denominator, or an uncertainty estimate. The central numerical finding therefore cannot be interpreted from the posted results.
Even if a within-participant decrease were reported, the single-group, non-randomized design would not by itself show that MMF caused it. A control group assigned under a comparable study process is not listed. General changes over time, variation in measurements, or other events could contribute to observed changes. The study can offer descriptive information about its participants, but the listed design does not provide a controlled treatment-effect estimate.
The primary outcome is a PRA threshold measure, not a direct measure of transplantation, survival, or overall clinical benefit. Transplantation and crossmatch outcomes are listed as secondary measures, but their results are not reported in the registry record. The summary discusses the relationship between PRA and waiting time as background, but this trial record does not report an analysis demonstrating that a change in PRA led to a change in waiting time or transplant outcomes.
The safety entries document serious and other adverse events in one study group. They do not provide a comparison that can show whether event occurrence was higher or lower than it would have been under another treatment or without MMF. The number of deaths is not reported in the registry record.
Accordingly, the available record identifies the question and design, but it does not allow a conclusion about the size or precision of the primary result, or about a causal treatment effect. It also does not support a comparative safety conclusion. A completed study status indicates the registry’s status entry; it does not substitute for reported outcome data.
13. Why This Trial Matters Statistically
This trial illustrates why design and results must be considered together. A prospective study can follow participants over time, but when it is non-randomized and single group, observed changes remain descriptive unless supported by a suitable comparison. The primary endpoint is clearly framed as a participant count meeting a PRA decrease threshold, yet the registry does not report the count or analysis denominator needed to quantify it.
The record also demonstrates the distinction between an outcome definition and an outcome result. Naming the threshold and assessment time tells readers what the investigators intended to measure. It does not reveal how many participants met that criterion, how variable the measurements were, or how precise an estimate might be. Those quantities are not reported in the registry record.
Safety entries require similar care. Counts and event terms from one group provide useful descriptive context, but they do not estimate comparative risk. The 37 participants at risk and the reported affected counts are not a substitute for a control-group comparison, and the listed serious-event percentages should not be interpreted as evidence of a causal relationship.
Finally, the registry lists several secondary outcomes but does not report their results or a multiplicity approach. This limits assessment of how the outcomes fit together and whether any statistical findings were selected from a broader set of analyses. The example underscores that transparent reporting of denominators, missing-data handling, analysis methods, effect estimates, and uncertainty is essential for readers to judge what a clinical trial can establish.
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 immediate limitation of interpretation is that the registry record does not report numerical results for the primary endpoint or the listed secondary outcomes. The primary endpoint count, analysis denominator, and uncertainty estimate are not reported. The record therefore does not permit an assessment of the magnitude or precision of the primary finding.
The non-randomized, single-group design has no like-for-like control comparison. Any change observed over time could have explanations other than the intervention, and the design does not isolate a causal effect. The study is open label with no masking, as recorded; the registry does not describe outcome-assessment procedures that might clarify how subjective or measurement-related influences were managed.
The operational definition of a 10% decrease in PRA, the measurement procedures, and the primary endpoint analysis population are not reported in the registry record. Missing-data handling and reasons for missing outcome measurements are also not reported. Although enrollment is listed as 45 and the adverse-event group has 37 participants at risk, the record does not explain the difference or identify the primary endpoint denominator.
The registry describes the population as patients on the kidney transplant list with high PRA and lists several conditions, but detailed eligibility criteria and the threshold for high PRA are not reported. Dose and treatment schedule are not reported. The result record does not provide enough information to assess how representative the enrolled participants were of other transplant-list populations.
The registry does not report the statistical analysis plan, sample-size justification, multiplicity handling, interim analyses, or stopping rules. These omissions limit evaluation of planned versus reported analyses. Safety information is also limited to the posted group counts and terms; deaths and further event-level details are not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Effects of Mycophenolate Mofetil (MMF) On Anti-HLA (Human Leukocyte Antigen)Antibody Levels In Patients Awaiting Cadaveric Renal Transplant. — NCT00446459.
- Registry record last updated 2010-04-15; status last verified 2010-04.
- Results first posted 2010-03-30.
- 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
NCT00446459 is a completed phase II, prospective, open-label, non-randomized, single-group study sponsored by the University of Washington. It enrolled 45 participants and evaluated mycophenolate mofetil among patients awaiting cadaveric renal transplantation with high PRA levels. The study period is listed as beginning in 2006-04, with primary completion in 2008-11.
The primary outcome is the number of participants with a 10% decrease in PRA level at month 8. Four secondary outcomes address significant infections, kidney transplantation, low white blood cell count or immunoglobulin titers below range, and negative crossmatch at transplant. Numerical results for these outcomes are not reported in the registry record.
The safety record lists 37 participants at risk, 2 affected for serious adverse events, and 6 affected for other adverse events. Myocardial infarction and retinal stroke are each listed at 2.7% among the top serious terms. The registry does not report a comparator group, numerical primary endpoint result, or number of deaths.