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 Eculizumab to Prevent Antibody-mediated Rejection in ABO Blood Group Incompatible Living Donor Kidney Transplantation (NCT01095887), a na phase 1, phase 2 study. The primary outcome is Number of Subjects With Antibody-Mediated Rejection (AMR) Within 3 Months of Kidney Transplant.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01095887 |
| Phase | Phase 1, Phase 2 |
| Condition | Kidney Transplant |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 6 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Mayo Clinic |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients undergoing kidney transplantation from a living donor with a different ABO blood group. The registry lists kidney transplant as the condition and describes ABO-incompatible living-donor transplantation in the study title. Enrollment was 6. Further participant characteristics, eligibility details, and the characteristics of the enrolled group are not reported in the registry record.
Intervention
The intervention was eculizumab, described as being added to conventional treatment. The registry says eculizumab was given on Day 0, day 1, and weekly for the first four weeks after transplant. Details of the conventional treatment and any changes to the dosing schedule are not reported in the registry record.
Comparator
There was no separate comparator group in the listed study design. The registry identifies a single-group model and no masking; allocation is listed as not applicable. The intervention group therefore cannot be read as a randomized comparison against a control group. Any within-study interpretation is descriptive rather than a like-for-like treatment contrast.
Primary question
The primary endpoint was the number of participants with antibody-mediated rejection within 3 months of kidney transplant surgery. This asks whether rejection was recorded during a defined period after transplantation among participants receiving the study regimen. The registry record does not report the primary endpoint result, so it does not establish how many participants experienced rejection during that period.
3. Trial Design
This was a single-center, open-label study listed as phase 1 and phase 2. The sponsor was Mayo Clinic. The registry gives a start date of 2010-05 and a primary completion date of 2014-04. The study status is terminated, and enrollment is listed as 6. The reason for termination is not reported in the registry record.
The study evaluated eculizumab added to conventional treatment in ABO-incompatible living-donor kidney transplantation. The intervention description states that eculizumab was administered on Day 0, day 1, and weekly for the first four weeks after transplant. Details of conventional treatment, participant eligibility, and any protocol changes are not reported in the registry record.
The listed model is single group, masking is none, and allocation is not applicable. Those features matter when interpreting results: there is no listed randomized control group against which to estimate a treatment contrast, and participants and study personnel were not masked. The registry record does not describe a comparator arm. The posted record lists one primary outcome measure and no analyses. It does not report a primary outcome result or explain why the study was terminated. The design and available results therefore support a description of what was planned and recorded, but not a controlled estimate of the effect of eculizumab.
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 | Eculizumab |
|---|---|
| STARTED | 6 |
| COMPLETED | 6 |
| NOT COMPLETED | 0 |
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 | Eculizumab |
|---|---|---|
| Participants analysed | 6 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 4 |
| Age, Categorical: >=65 years | Participants | 2 |
| Sex: Female, Male: Female | Participants | 3 |
| Sex: Female, Male: Male | Participants | 3 |
| Region of Enrollment: United States | participants | 6 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Subjects With Antibody-Mediated Rejection (AMR) Within 3 Months of Kidney Transplant, measured over 3 months after kidney transplant surgery.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was the number of participants with antibody-mediated rejection within 3 months of kidney transplant surgery. It is a binary outcome at the participant level: each participant either is or is not recorded as having the event during the stated timeframe. The registry labels the measure as a number of participants, rather than a rate of events over time.
This endpoint addresses the study’s stated question about preventing antibody-mediated rejection after ABO-incompatible living-donor kidney transplantation. As a general statistical point, an endpoint tied to a defined clinical event and follow-up period makes the outcome question explicit and permits a count of participants who meet that definition. The registry does not give further details about how rejection was defined, how it was confirmed, or how events were adjudicated.
The posted results do not report the primary endpoint count. Consequently, the registry record does not show the observed frequency of rejection in the study group, and no estimate of uncertainty around that outcome is reported. A count alone would also not describe the timing or severity of events; those details are not reported as part of the primary endpoint result.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a small, single-group study, with enrollment of 6, and identifies antibody-mediated rejection within 3 months as the primary endpoint. However, the primary endpoint result is not reported. The record therefore does not say how many participants met the endpoint, and it contains no reported analysis for that outcome. It is not possible to judge the observed endpoint frequency or its precision from the registry record.
The design is also important. Because the study is listed as single group, with allocation not applicable, there is no randomized control group for a direct treatment comparison. An outcome observed in participants receiving the study regimen, even if reported, would describe that group; by itself, it would not separate the contribution of eculizumab from conventional treatment, participant characteristics, or other aspects of care. The open-label design means masking was absent, but the registry does not describe whether this affected outcome assessment.
The safety record lists 5 participants affected by serious adverse events among 6 at risk, and 1 participant affected by other adverse events among 6 at risk. These are safety figures, not the missing primary endpoint result. The serious-event terms are listed with percentages, but the record does not provide enough detail to infer causal attribution or to treat those terms as a measure of treatment effect.
In general, statistical precision depends on both the observed outcome and the amount of information available. A confidence interval can express uncertainty around an estimate, but no primary endpoint estimate or interval is reported. The small enrollment also means that any estimate, if available, would need cautious interpretation; the record does not provide a formal uncertainty analysis. It reports no p-value, adjusted estimate, or analysis plan. The sample-size illustrations use planning assumptions and should not be read as observed power or as evidence of a treatment effect. Overall, the posted results do not establish whether the regimen changed rejection outcomes.
9. Statistical Methods Explained
What does a single-group design allow?
The registry lists a single-group model, no masking, and allocation as not applicable. In general, a single-group study can describe outcomes among participants who receive the study regimen. It does not, on its own, estimate what would have happened to comparable participants receiving a different regimen. Without a control group, differences between observed outcomes and an external expectation can reflect factors other than the intervention. This study’s record does not identify a concurrent comparator, and it should not be interpreted as a randomized treatment comparison.
How should the primary endpoint be understood?
The endpoint is a count of participants with antibody-mediated rejection within 3 months after kidney transplant surgery. In general, this kind of binary outcome records whether each participant experienced a specified event during a defined period. The measure is not an event rate per unit of follow-up time. The registry does not report the endpoint count, event definition details, or a statistical analysis of the outcome. A count, if available, would describe observed participants; it would not by itself establish a causal treatment effect.
What does statistical uncertainty mean?
As a general explanation, an estimate describes the result in the observed study group, while a confidence interval conveys how uncertain that estimate is under the statistical model. Wider intervals indicate less precision; narrow intervals indicate greater precision, conditional on the assumptions used. No primary endpoint estimate or confidence interval is reported in the registry record. There is therefore no reported interval to interpret, and no basis for recovering one without making calculations from information that is not present.
For binary outcomes, methods may be used to estimate an event proportion and its uncertainty. The choice of method can matter, particularly when the amount of information is limited or events are sparse. The registry record does not state which method, if any, was used for the primary endpoint. No p-value or inferential result is reported. These absences should not be filled by assuming a conventional test or analysis.
What do the planning assumptions mean?
The registry’s sample-size assumptions describe a planning illustration, not the study’s own design. They specify a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect to detect defined as a stated fraction of the observed effect. These assumptions describe a hypothetical planning comparison; they do not change the listed study design, which is single group. The record does not report a trial-specific sample-size justification.
In general, planning calculations ask how much information a future comparison might require under stated assumptions. Their outputs depend on the assumed effect and statistical method. The illustrations are not observed power: observed power is not a substitute for interpreting an estimated effect and its uncertainty. The registry record does not report the primary outcome result needed to assess the observed effect from the trial itself. The planning calculations should therefore not be treated as evidence that the trial could detect a particular effect, nor as a summary of what happened among enrolled participants.
Why does over-dispersion matter?
In general, over-dispersion occurs when variability in observed counts exceeds what a statistical model assumes. If a count model underestimates variability, its uncertainty estimates can be too small and inference can appear more precise than warranted. This study’s primary endpoint is described as a participant count for a binary event, and the registry does not report a count model or an over-dispersion assessment. Over-dispersion is a general statistical consideration, not a reported feature or analysis of this trial.
The registry lists no analyses for the outcome measure. It does not specify a statistical model, adjustment variables, hypothesis test, confidence-interval method, or analysis population. The interpretation must therefore remain descriptive of the information recorded, without attributing unreported methods or calculations to the investigators.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists one primary outcome measure and no analyses. It does not report a multiplicity plan, such as how the investigators would handle inference across multiple outcomes or repeated statistical tests. With no reported primary endpoint analysis, there is no reported significance testing for which a multiplicity adjustment can be assessed. The record also does not describe interim analyses or stopping rules; these should not be inferred from the terminated status.
Missing-data methods are not reported in the registry record. It does not state whether primary endpoint status was available for every enrolled participant, how incomplete follow-up was handled, or whether any participants were excluded from an analysis. The enrollment figure alone does not establish the size of an analysis population. No imputation method, sensitivity analysis, or missingness summary is reported for the primary endpoint.
The analysis population is also not defined in the available record. It does not specify whether the intended analysis included all enrolled participants, only participants who received treatment, or another population. These distinctions can affect interpretation because the analyzed group may differ from the group initially enrolled. No participant flow or endpoint-specific denominator is reported for the primary outcome. Consequently, the registry record does not support assumptions about completeness of follow-up, exclusions, or the handling of missing outcomes.
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 |
|---|---|---|---|
| Eculizumab | 5 | 6 | 83.3% |
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 |
|---|---|
| Eculizumab | 1 of 6 |
Most frequent serious adverse event terms
| Event term | Organ system | Eculizumab |
|---|---|---|
| Partial small bowel obstruction | Gastrointestinal disorders | 2 of 6 |
| Elevated creatinine | Renal and urinary disorders | 2 of 6 |
| Allograft Nephrectomy | Renal and urinary disorders | 2 of 6 |
| Antibody-mediated rejection of transplant | Renal and urinary disorders | 2 of 6 |
| Right hemicolectomy | Surgical and medical procedures | 2 of 6 |
| Prolonged hospitalization | Cardiac disorders | 1 of 6 |
| Native kidney mass | Renal and urinary disorders | 1 of 6 |
| Native Nephrectomy | Renal and urinary disorders | 1 of 6 |
| Splenectomy | Surgical and medical procedures | 1 of 6 |
| Hyperkalemia | Cardiac disorders | 1 of 6 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Eculizumab |
|---|---|---|
| Atrial Fibrillation | Cardiac disorders | 1 of 6 |
| Anemia | Blood and lymphatic system disorders | 1 of 6 |
| High-volume serous drainage from surgery incision site | Surgical and medical procedures | 1 of 6 |
| Wound infection | Infections and infestations | 1 of 6 |
| Lymphocele | Surgical and medical procedures | 1 of 6 |
| Debilitation | General disorders | 1 of 6 |
Serious events with the highest arm-level rate: partial small bowel obstruction (33.3%), elevated creatinine (33.3%), allograft nephrectomy (33.3%), antibody-mediated rejection of transplant (33.3%), right hemicolectomy (33.3%).
The registry lists 6 participants at risk for serious adverse events, with 5 affected, and 6 at risk for other adverse events, with 1 affected. These are participant-level figures as labeled in the record; they should not be interpreted as counts of every event episode. The record lists serious-event terms with percentages: Partial small bowel obstruction, 33.3%; Elevated creatinine, 33.3%; Allograft Nephrectomy, 33.3%; Antibody-mediated rejection of transplant, 33.3%; Right hemicolectomy, 33.3%; and Prolonged hospitalization, 16.7%.
The registry does not explain the denominator used for each listed term, whether terms overlap within participants, or how relatedness to eculizumab was assessed. The percentages should therefore be read as reported, not converted into event counts or added together. The death count is not reported in the registry record. Adverse-event listings describe observed safety information; without a control group and further attribution details, they do not establish that the intervention caused an event or that an event was caused by something else.
12. What the Result Does, and Does Not, Mean
The registry record establishes that this was an open-label, single-group study of eculizumab added to conventional treatment in ABO-incompatible living-donor kidney transplantation. It records enrollment of 6, a primary endpoint of antibody-mediated rejection within 3 months, and a terminated study status. The posted safety figures report 5 participants affected by serious adverse events among 6 at risk, and 1 affected by other adverse events among 6 at risk.
The record does not report the primary endpoint result, any outcome analysis, or a comparator group. It therefore does not show how many participants experienced rejection during the defined period, whether that outcome differed from another treatment strategy, or how precise an estimate might be. It also does not explain the reason for termination. None of these missing details can be inferred from the listed enrollment or safety information.
As a general principle, an observed outcome in a single group is not equivalent to a controlled estimate of treatment effect. Clinical outcomes can reflect multiple influences, and a comparison group is needed to distinguish the intervention’s contribution from other explanations. Here, the registry provides no such comparison. The safety terms and percentages likewise should not be treated as proof of causation.
The study record can be read as a limited account of the design, planned endpoint, and recorded safety data. It cannot establish whether eculizumab prevented rejection, whether it changed risk, or whether the reported safety events were attributable to treatment. Those questions require outcome and analysis information that is not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically notable because its central clinical question is clear, while the registry record does not report the primary endpoint result needed to answer it. The endpoint is antibody-mediated rejection within 3 months after kidney transplant surgery, but no count or analysis is available. This distinction illustrates why a stated outcome and an outcome result are not interchangeable: the endpoint defines what investigators intended to measure, whereas the result shows what was observed.
The single-group design also highlights the difference between describing outcomes and estimating a treatment contrast. A single-group study can record what happened among participants receiving the regimen, but it does not provide a randomized comparator for estimating what would have happened under another strategy. This limits causal interpretation independently of the statistical method used.
The registry’s sample-size assumptions offer a further lesson. Planning illustrations depend on assumptions about the comparison, effect, and statistical approach. Here, the illustration describes equal allocation between two arms even though the listed trial model is single group. That is a planning scenario, not a description of the enrolled design, and it is not observed power. The absence of a reported primary outcome estimate also prevents readers from assessing the observed effect that such an illustration references.
Finally, safety counts and listed event terms require careful separation from efficacy outcomes. They describe reported adverse-event information but do not resolve the missing primary endpoint result or establish causation. The record therefore illustrates the importance of reporting outcome counts, analysis methods, uncertainty, participant accounting, and reasons for study status when presenting a clinical trial.
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 trial-specific limitations constrain interpretation. The study is listed as single group, with no masking and allocation not applicable, so the registry does not provide a randomized control comparison. Any outcomes in the treated group could not be separated from other influences using a within-trial comparator. The enrollment was 6, limiting the amount of information available for describing outcomes.
Most importantly, the primary endpoint result is not reported. The record identifies the endpoint and timeframe but gives no count, analysis, or estimate of uncertainty. The event definition and method of outcome assessment are also not reported in the registry record. Details of participant characteristics, eligibility, follow-up completeness, and the analysis population are absent, as are missing-data methods and a statistical analysis plan.
The study status is terminated, but the reason is not reported. It is not appropriate to infer that the study stopped because of efficacy, safety, recruitment, or another cause. The safety record lists affected participants and event terms, but does not describe event attribution, overlap, or the denominator for each term. The death count is not reported in the registry record. These gaps prevent a fuller assessment of the safety findings and the trial’s conduct.
Finally, the sample-size assumptions are explicitly planning illustrations rather than the trial’s own design. They do not replace a trial-specific justification or provide observed evidence about the endpoint. Together, these reporting limitations mean the registry record supports only a cautious description of design and recorded safety figures, not an estimate of treatment effect.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Eculizumab to Prevent Antibody-mediated Rejection in ABO Blood Group Incompatible Living Donor Kidney Transplantation — NCT01095887.
- Registry record last updated 2015-07-20; status last verified 2015-06.
- Results first posted 2015-06-19.
- 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
NCT01095887 is a Mayo Clinic, single-center, open-label study listed as phase 1 and phase 2. The registry reports a start date of 2010-05, a primary completion date of 2014-04, terminated status, and enrollment of 6. The study evaluated eculizumab added to conventional treatment in ABO-incompatible living-donor kidney transplantation. Eculizumab was described as given on Day 0, day 1, and weekly for the first four weeks after transplant.
The primary endpoint was the number of participants with antibody-mediated rejection within 3 months of kidney transplant surgery. The registry record reports no analyses and does not report the endpoint result. Safety listings include serious and other adverse-event figures and serious-event terms, but do not provide the causal attribution needed to connect events to treatment. The design has no listed comparator group, and the reason for termination is not reported. These limits preclude a controlled estimate of treatment effect from the registry record.