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 Irradiated Donor Lymphocytes in Treating Patients With Metastatic Kidney Cancer (NCT00176501), a na phase 2 study. The primary outcome is Response Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00176501 |
| Phase | Phase 2 |
| Condition | Kidney Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 3 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Rutgers, The State University of New Jersey |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients with metastatic kidney cancer, described in the registry record as renal cell carcinoma. The record reports enrollment of 3. It does not report further eligibility details, such as disease history, prior treatment, or clinical characteristics of those enrolled.
Intervention
The intervention was therapeutic allogeneic lymphocytes, described in the study title and arm as irradiated donor lymphocytes. The stated rationale was that infused donor lymphocytes might help the patient's immune system kill tumor cells. The registry record does not provide additional treatment administration details in the arm description.
Comparator
This was a single-group study, with no randomized allocation and no like-for-like control group. The study therefore does not provide a direct comparison with another treatment or with an untreated group. Any interpretation concerns the experience recorded in the intervention group, not a comparative treatment effect.
Primary question
The primary outcome was response rate, assessed over a timeframe of 5 years. The study aimed to examine clinical or radiological response as well as side effects. It closed early because of slow accrual, and the registry says insufficient data were collected to analyze the primary outcome. The actual response rate and the response criteria are not reported in the registry record.
3. Trial Design
This was a phase II, open-label, single-group study of irradiated allogeneic lymphocytes for metastatic kidney cancer. The sponsor was Rutgers, The State University of New Jersey. The registry lists the study as starting in 2004-08, with primary completion in 2008-08, and as terminated. The enrollment recorded is 3. The registry does not explain whether that enrollment figure represents all participants who began treatment or another enrollment convention.
The design did not include random allocation or a control arm. Masking was listed as none. Consequently, the design can describe observations among participants receiving the intervention, but it cannot isolate a treatment effect from the natural course of disease, other care, or other influences through a concurrent comparison. The absence of a control group is a design feature, not evidence that the intervention was beneficial or harmful.
The planned primary analysis used a two-stage Simon design intended to minimize expected accrual. The plan specified a maximum sample size of 35 subjects. Stage 1 was to accrue 18 subjects; the trial would stop if 2 or fewer responses were seen, while 3 or more responses would lead to accrual of an additional 19 patients for stage 2. At the end of the trial, 6 or fewer responses out of 35 would mean no further investigation was warranted under that design. These were planned decision rules, not observed results. The registry says the study closed early because of slow accrual and insufficient data to analyze the primary outcome; it does not report that the planned stage thresholds were reached or applied.
Secondary outcomes were the rate and kinetics of clinical or radiological response and the rate of graft-versus-host disease. Definitions, assessment schedules, and analysis details for these outcomes are not reported in the registry record. The number of outcome measures listed does not establish that each could be analyzed.
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 | Irradiated Allogeneic Lymphocytes |
|---|---|
| STARTED | 3 |
| COMPLETED | 3 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: The study was closed early due to slow accrual and insufficient data were collected to analyse this outcome measure.
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 | Irradiated Allogeneic Lymphocytes |
|---|---|---|
| Participants analysed | 3 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 3 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age Continuous | years | 55.3 (SD 7.1) |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 2 |
| Region of Enrollment: United States | participants | 3 |
6. Primary Endpoint
The registry lists the primary outcome as Response Rate, measured over 5 years.
| 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 response rate, with a timeframe of 5 years. A response-rate endpoint is intended to express how often participants meet a prespecified definition of improvement in disease status. In cancer research, this can offer an interpretable measure of observed tumor response, but it depends on clear criteria for defining response, scheduled assessments, and rules for handling participants whose status cannot be evaluated.
The registry description also refers to clinical and radiological response. However, the specific response criteria, assessment procedures, and denominator for the reported response rate are not reported in the registry record. Nor is an observed response count or rate reported. The record explicitly states that the study closed early because of slow accrual and that insufficient data were collected to analyze this outcome.
The endpoint was paired with a planned Simon two-stage design. In general, such a design can set decision thresholds for whether to continue accrual after an initial stage and whether the observed response result justifies further investigation. The registry's planned thresholds describe a prospective decision framework; they do not establish that the study accumulated enough information to estimate response or that any threshold was met.
Because the study lacked a control group, even an analyzable response rate would describe outcomes in the treated group rather than a comparative effect. The registry record does not support a conclusion about how those outcomes differed from what would have happened under another treatment or without the intervention.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive fact is that the primary response-rate outcome was not analyzed. The registry states that the study closed early because accrual was slow and that insufficient data were collected for this endpoint. Enrollment was 3, while the planned design described a maximum sample size of 35. The posted results do not give an observed response count, response rate, confidence interval, or test result. The absence of these estimates is not evidence that there were no responses; it means the record does not report an analyzable outcome.
The planned Simon design was built around response counts at specified stages. It set a stopping rule after the initial accrual and a final decision threshold at the end of the planned study. Since the study ended early and no outcome analysis is reported, the design's planned thresholds cannot be treated as observed findings. The registry record does not say whether any formal stage decision was made before termination. Slow accrual is the stated reason for early closure; it should not be replaced with an assumed response-based stopping reason.
The single-group structure limits interpretation even apart from the incomplete endpoint. Without a concurrent control group, an observed response could not by itself show how outcomes compared with another treatment or with the course of disease without the intervention. The study was unmasked, so assessments were not reported as blinded. Whether this affected endpoint assessment is not reported in the registry record; it is a design consideration when interpreting clinical or radiological outcomes.
The secondary outcomes listed include response rate and kinetics and graft-versus-host disease. Their listing identifies topics of interest, not evidence that those outcomes were evaluated or analyzed. The registry does not provide outcome estimates, analysis details, or results for them. The total number of outcome measures listed therefore should not be read as the number of completed analyses; the record reports no analyses.
Safety information is limited to the serious adverse event summary. For the irradiated allogeneic lymphocyte group, 3 were at risk and 1 was affected by a serious adverse event; 3 were at risk and 2 were affected by other adverse events. The listed serious terms each carry a percentage of 33.3%. Those figures describe the posted safety record, but do not provide event timing, severity grades, attribution, or enough detail to establish whether terms overlapped within participants. They also do not support a comparison with another group.
In general, an estimate is more informative when its uncertainty is reported alongside it. A confidence interval describes a range of values compatible with the data and model, rather than guaranteeing that the true value lies within that range. No confidence interval is reported for the primary response outcome. With no reported endpoint estimate or uncertainty measure, the registry record does not establish the magnitude or precision of response in this study.
9. Statistical Methods Explained
What does the planned two-stage design do?
In general, a Simon two-stage design is a single-arm phase II approach that uses response counts to guide whether a study should continue. Its purpose can be to limit expected accrual when early results do not meet a prespecified response threshold, while allowing further accrual when the initial result meets a continuation rule. The registry says this design was optimized to minimize the expected number of patients accrued. Its planned rules were to stop after stage 1 with 2 or fewer responses, continue with an additional 19 patients after 3 or more responses, and conclude that further investigation was unwarranted if 6 or fewer responses were observed out of 35 by the end.
These rules are part of the planned design, not results. The registry reports early closure for slow accrual and insufficient data to analyze the response-rate outcome. It does not report whether stage 1 was completed, whether the continuation rule was applied, or whether a final response threshold was evaluated. No response count is reported. Thus the design cannot be used to infer a favorable or unfavorable result from the termination status alone.
What can a single-group study estimate?
In general, a single-group study can describe the outcomes observed among participants who receive the intervention. It does not directly estimate a contrast between the intervention and a comparator because no comparator group is included. Random allocation is also absent in this design. As a result, a response rate, if available, would be descriptive of the enrolled group under the study's procedures, rather than a randomized estimate of comparative benefit or harm.
The study is listed as unmasked. In an unmasked study, participants and study personnel are not described as blinded to the intervention. For an outcome involving clinical or radiological response, assessment can depend on definitions, timing, and judgment. The registry does not report whether independent review or standardized assessment procedures were used. These details matter for evaluating possible assessment bias, but their absence does not establish that bias occurred.
Why do response definitions and denominators matter?
In general, a response rate requires both a response definition and a rule for deciding which participants enter the denominator. A protocol may specify how to classify changes in disease, when assessments occur, and how to handle missing or unevaluable assessments. The registry identifies the primary endpoint as response rate over a timeframe of 5 years, and its description refers to clinical or radiological response. It does not report response criteria, assessment schedules, denominator rules, or an observed estimate. Those omissions prevent readers from reconstructing the endpoint or checking its calculation.
The distinction between a planned assessment timeframe and an observed follow-up period is important. The stated timeframe is 5 years, but it does not establish that every participant was followed for that duration. Actual follow-up completeness and duration are not reported in the registry record. Nor does the timeframe show when responses occurred or how quickly they developed; response kinetics is listed as a secondary outcome, but its results are not reported.
How should precision be assessed?
In general, a point estimate gives a central summary, while a confidence interval conveys uncertainty around that estimate under the statistical model. A narrow interval typically indicates greater precision than a wide interval, though interpretation also depends on study design and assumptions. The registry does not report a primary response estimate or confidence interval, so its precision cannot be evaluated. The small recorded enrollment and early closure mean that the planned response-rate analysis was not completed, but the record does not quantify what precision would have been achieved.
A p-value, when used, addresses how compatible observed data are with a specified null model; it is not the probability that a treatment works or that the null hypothesis is true. No p-value or primary-outcome analysis is reported. No model, covariate adjustment, or testing procedure for an outcome analysis is reported either. The statistical analysis plan beyond the Simon design is not reported in the registry record.
What do the posted safety counts describe?
For safety outcomes, a count of participants affected is distinct from a count of adverse-event episodes. One participant may experience more than one event, and an event term may be reported in more than one way depending on coding. The registry gives participants at risk and affected for serious and other adverse events, and lists serious-event terms with percentages. It does not explain the event-level counting method, overlap among terms, time at risk, or causal attribution. These safety numbers should therefore be read as a limited descriptive report, not as comparative rates or proof of causation.
In general, randomized, masked comparisons and prespecified analysis procedures can address some uncertainties that a single-group, unmasked study cannot resolve. This study had no randomized comparator, and its primary endpoint was not analyzed. The registry record does not report an alternative adjusted or exploratory analysis that could fill that gap. Any sample-size calculations elsewhere associated with the results are planning illustrations, not observed power and not a substitute for an outcome analysis. They are also not this trial's stated Simon design.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary response-rate outcome and secondary outcomes concerning the rate and kinetics of clinical or radiological response and the rate of graft-versus-host disease. When a study assesses several outcomes, multiplicity can arise because repeated tests or analyses increase the chance of finding an apparently notable result by chance. In general, a statistical plan may specify a primary outcome, testing hierarchy, or adjustment procedure to address that issue. No multiplicity adjustment or testing hierarchy is reported in the registry record.
The record reports no analyses, and it states that insufficient data were collected to analyze the primary outcome. The outcomes listed should therefore not be treated as completed tests or as a set of findings from which a positive result can be selected. The registry does not report whether secondary outcomes were analyzed, how missing assessments were handled, or whether any exploratory analyses were undertaken.
Analysis populations are also not described. The registry does not report whether the primary endpoint was to use all enrolled participants, only participants who received treatment, or another defined population. It does not state how participants with incomplete follow-up or unevaluable response status would be classified. The reason given for closure was slow accrual; participant-level reasons for missing outcome information are not reported.
In general, missing outcome information can affect both the size and interpretation of an estimate, especially when the reasons for missingness are related to health status or outcome. The registry does not give enough information to assess such patterns. No imputation method, sensitivity analysis, or missing-data assumption is reported. Because the primary result itself is unavailable, there is no basis for assessing whether a missing-data method changed an estimate.
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 |
|---|---|---|---|
| Irradiated Allogeneic Lymphocytes | 1 | 3 | 33.3% |
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 |
|---|---|
| Irradiated Allogeneic Lymphocytes | 2 of 3 |
Most frequent serious adverse event terms
| Event term | Organ system | Irradiated Allogeneic Lymphocytes |
|---|---|---|
| Diarrhea | Gastrointestinal disorders | 1 of 3 |
| Positive lymphocyte culture | Blood and lymphatic system disorders | 1 of 3 |
| Vomiting | Gastrointestinal disorders | 1 of 3 |
| Nausea | Gastrointestinal disorders | 1 of 3 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Irradiated Allogeneic Lymphocytes |
|---|---|---|
| Fatigue (asthenia, lethargy, malaise) | General disorders | 2 of 3 |
| Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10e9/L) | General disorders | 2 of 3 |
| Diarrhea | Gastrointestinal disorders | 1 of 3 |
| Nausea | Gastrointestinal disorders | 1 of 3 |
| Vomiting | Gastrointestinal disorders | 1 of 3 |
| Alkaline phosphatase | Investigations | 1 of 3 |
| ALT, SGPT (serum glutamic pyruvic transaminase) | Blood and lymphatic system disorders | 1 of 3 |
| AST, SGOT (serum glutamic oxaloacetic transaminase) | Blood and lymphatic system disorders | 1 of 3 |
| Pain - Back | General disorders | 1 of 3 |
| Pain - Kidney | General disorders | 1 of 3 |
Serious events with the highest arm-level rate: diarrhea (33.3%), positive lymphocyte culture (33.3%), vomiting (33.3%), nausea (33.3%).
For the irradiated allogeneic lymphocyte group, the registry reports 3 at risk for serious adverse events, with 1 affected. It reports 3 at risk for other adverse events, with 2 affected. These are participant counts as labeled in the record; they are not a comparison between treatment groups, because the study had a single group.
The listed serious adverse event terms are diarrhea, positive lymphocyte culture, vomiting, and nausea, each reported as 33.3%. The registry does not state whether these terms occurred in the same or different participants, whether percentages refer to participants or event reports, or how the terms relate to the serious-event count. They should not be added or interpreted as separate participants.
The record does not report event timing, duration, severity grading, treatment relatedness, or outcomes following the events. Deaths are not given as a numerical result. These details are needed for a fuller safety interpretation. The figures are descriptive and do not establish that the intervention caused an event or how its safety compares with another treatment.
12. What the Result Does, and Does Not, Mean
The registry record documents a phase II study of irradiated donor lymphocytes in metastatic kidney cancer, with enrollment of 3 and early termination because of slow accrual. Its primary outcome was response rate over a timeframe of 5 years, but the record says insufficient data were collected to analyze that outcome. The actual response count and rate are not reported. This means the record does not establish the observed response level; it does not mean that there were no responses.
The planned two-stage Simon design described decision thresholds for response counts, but the study's early closure should not be mistaken for a result under those thresholds. The registry does not state that a response-based stopping rule was reached. Nor does it report an outcome estimate with a confidence interval or other measure of precision. The primary question therefore remains unanswered in the posted results.
The single-group, unmasked design also sets limits on what could be concluded even if a response rate had been reported. Without a concurrent comparator, observed outcomes cannot show how the group would have fared under another treatment or without the intervention. The registry does not provide a causal estimate, a treatment contrast, or results from the listed secondary outcomes.
The serious and other adverse-event counts and listed terms offer limited descriptive safety information. They do not show comparative risks, establish causation, or resolve uncertainties about the event-level details. Overall, the registry supports describing the study's intent, design, early closure, and limited posted safety counts. It does not support a conclusion that the intervention is effective or ineffective, or a complete assessment of its safety.
13. Why This Trial Matters Statistically
This study illustrates why a planned statistical design and an analyzable result are different things. The Simon design set out a response-based framework for deciding whether accrual should continue and whether further investigation was warranted. But slow accrual led to early closure, and the registry says the primary response outcome could not be analyzed. A prespecified decision rule cannot answer the research question when the information needed to apply it is not available.
It also demonstrates the limits of interpreting an enrollment figure in isolation. The recorded enrollment was 3, while the planned design described a maximum sample size of 35 and stage-specific accrual targets. Those figures show a substantial gap between the planned design and the information ultimately available, but they do not reveal the response rate, the reason for each participant's outcome status, or the precision of an estimate. No endpoint estimate is reported.
The single-group structure is another important statistical feature. A response rate in a treated group may be useful as a descriptive observation, but without a concurrent comparator it does not estimate a comparative treatment effect. This distinction is especially important when registry entries use language about how well an intervention works: a study's stated purpose is not itself an outcome finding.
Finally, the record shows the value of distinguishing outcome listings from completed analyses. Response kinetics and graft-versus-host disease are named as secondary outcomes, while the registry reports no analyses. The listed safety counts are limited descriptions rather than evidence from a comparative safety study. Together, these features make the study a clear example of how design, accrual, outcome availability, and reporting determine what statistical conclusions can be drawn.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The most important trial-specific limitation is early closure due to slow accrual, with insufficient data collected to analyze the primary response-rate outcome. The registry gives an enrollment of 3 but does not report an observed response count, response rate, or confidence interval. Thus neither the direction nor the magnitude of the primary outcome can be assessed.
The study had a single group and no randomized allocation or comparator. It was unmasked. These design features limit comparative and causal interpretation and leave open the possibility of assessment influences, although the registry does not report whether any such influence occurred. Details of the response criteria, assessment schedule, denominator, and rules for unevaluable participants are not reported.
The record does not report actual follow-up duration or completeness, participant characteristics, treatment administration details, or reasons for missing endpoint information. It also does not report the analysis population, statistical methods beyond the planned Simon design, or handling of missing data. The planned accrual and response thresholds therefore cannot be assumed to describe what was achieved or analyzed.
Secondary outcome results are not reported, despite their listing. Safety reporting is limited to counts and serious-event terms, without timing, severity, attribution, or an explanation of possible overlap among terms. No comparative safety assessment is available. Finally, the registry reports no analyses, so there are no posted inferential results to evaluate for robustness or consistency with the design.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Irradiated Donor Lymphocytes in Treating Patients With Metastatic Kidney Cancer — NCT00176501.
- Registry record last updated 2014-01-06; status last verified 2013-11.
- Results first posted 2014-01-06.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
The registry describes a phase II, open-label, single-group study of irradiated allogeneic lymphocytes for metastatic kidney cancer, sponsored by Rutgers, The State University of New Jersey. It lists enrollment of 3, a start date of 2004-08, primary completion in 2008-08, and terminated status.
The primary endpoint was response rate over a timeframe of 5 years. The planned Simon two-stage design specified response-count rules, but the study closed early because of slow accrual. The registry states that insufficient data were collected to analyze the primary outcome. No response estimate or analysis is reported. Listed secondary outcomes concerned clinical or radiological response and graft-versus-host disease; their results are not reported.
The safety record gives counts of participants at risk and affected and lists serious adverse event terms, each with a percentage of 33.3%. The record does not provide further detail needed for a comparative or causal safety interpretation.