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 Gemcitabine and Cisplatin as Neoadjuvant Chemotherapy in Patients With High-Grade Upper Tract Urothelial Carcinoma (NCT01261728), a na phase 2 study. The primary outcome is Pathologic Response Rate (<pT2).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01261728 |
| Phase | Phase 2 |
| Condition | Urothelial Carcinoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 57 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Memorial Sloan Kettering Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
Patients with high-grade upper tract urothelial carcinoma who were candidates for radical nephroureterectomy or distal ureterectomy. The registry record lists 57 participants enrolled.
Intervention
Neoadjuvant gemcitabine and cisplatin before surgery. The brief summary describes four 21 day cycles, for a total of 12 weeks.
Comparator
No concurrent comparator group is listed. This is a single-group study, and the allocation field is recorded as NA.
Primary question
What was the pathologic response rate after neoadjuvant chemotherapy, using the study definition of absence of high-grade carcinoma (<pT2 disease) and absence of microscopic lymph node metastases (N0) in the final surgical specimen?
The clinical question concerns treatment before surgery, rather than a comparison of two assigned regimens. The study description says chemotherapy was intended to shrink the tumor before surgery for kidney cancer. The listed intervention is gemcitabine and cisplatin, and the arm description identifies patients eligible for radical nephroureterectomy or distal ureterectomy.
The primary endpoint is defined by findings in the final nephroureterectomy specimen. That definition combines the absence of high-grade carcinoma at or beyond the stated threshold with the absence of microscopic lymph node metastases. The registry record does not report the number of participants who met this definition or a pathologic response rate. It therefore does not answer the response question numerically. The named secondary outcomes include progression-free survival rate, overall survival, and participants evaluated for toxicity; numerical results for the survival outcomes are not reported in the registry record.
3. Trial Design
This is a Phase II, single-group study of gemcitabine and cisplatin as neoadjuvant chemotherapy for patients with high-grade upper tract urothelial carcinoma. The registry record lists 57 participants enrolled and identifies Memorial Sloan Kettering Cancer Center as the sponsor. The study status is completed. The listed start date is 2010-12-14, and the primary completion date is 2025-08-06.
The intervention is gemcitabine and cisplatin. The brief summary describes four 21 day cycles over a total of 12 weeks before surgery. The arm description states that participants were candidates for radical nephroureterectomy or distal ureterectomy. Masking is recorded as none, and allocation is recorded as NA. These details are consistent with a study without a listed randomized control arm; they do not establish how participants were selected or whether other treatment decisions followed a common protocol.
The primary outcome is pathologic response rate, assessed using the final nephroureterectomy specimen and the stated criteria for tumor and lymph node findings. Its timeframe is listed as up to 2 years. The registry record lists progression-free survival rate, overall survival, and number of participants evaluated for toxicity as secondary outcomes. It records four outcome measures, but does not report numerical findings for the primary endpoint or the named survival outcomes.
A Phase II label describes the study phase, not the certainty of an effect. The registry record does not report a randomized comparison, a control-group result, or a statistical analysis for the primary endpoint. It also does not report the protocol’s sample-size justification, the intended analysis population, or details of any interim analyses. Those omissions limit how fully the design and analysis can be reconstructed from the registry record.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Gemcitabine and Cisplatin |
|---|---|
| STARTED | 57 |
| COMPLETED | 57 |
| 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 | Gemcitabine and Cisplatin |
|---|---|---|
| Participants analysed | 57 | |
| Age, Continuous | years | 66 (58 to 71) |
| Sex: Female, Male: Female | Participants | 21 |
| Sex: Female, Male: Male | Participants | 36 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 11 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 43 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 3 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 2 |
| Race (NIH/OMB): White | Participants | 54 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | Participants | 57 |
6. Primary Endpoint
The registry lists the primary outcome as Pathologic Response Rate (<pT2), measured over up to 2 years.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the pathologic response rate, expressed as a count of participants in the registry outcome description. Response is defined as absence of high-grade carcinoma, specified as <pT2 disease, and absence of microscopic lymph node metastases, specified as N0, in the final nephroureterectomy specimen. The outcome timeframe is listed as up to 2 years.
This endpoint uses pathology from the surgical specimen to assess whether the prespecified tumor and lymph node criteria are met after neoadjuvant chemotherapy. In general, a pathologic endpoint can describe findings in tissue removed during surgery, rather than relying only on a clinical assessment before surgery. The combined definition matters: a participant must satisfy both the tumor criterion and the lymph node criterion to meet the stated response definition.
The registry record does not report how many participants met the response definition, the denominator used for the response rate, or an estimated rate with a measure of uncertainty. The enrolled count of 57 should not be assumed to be the endpoint denominator, because the analysis population and the number with evaluable surgical specimens are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the study question and the endpoint definition, but it does not provide a numerical primary-endpoint result. It is therefore not possible to describe the observed pathologic response rate, its precision, or how many participants met the combined tumor and node criteria. Enrollment of 57 participants is not itself evidence of a particular response rate.
The study is single-group, with no concurrent comparator listed. Any response observed in such a design would describe outcomes among participants who received the regimen in the study context. Without a control group, the registry record cannot support a direct estimate of how outcomes would differ under another regimen or no neoadjuvant chemotherapy. This design feature does not invalidate a descriptive outcome, but it constrains causal comparisons.
The registry record names progression-free survival and overall survival as secondary outcomes, but numerical findings for them are not reported. It also lists the number of participants evaluated for toxicity. The available safety counts include 22 participants with serious adverse events among 57 at risk, 57 participants with other adverse events among 57 at risk, and 11 deaths. These are separate safety counts; the registry record does not establish that deaths were caused by treatment, nor does it provide the timing or causes of death.
The serious adverse event terms listed most prominently include decreased lymphocyte count (33.3%), neutrophil count decrease (31.6%), and hyperglycemia (14.0%). Febrile neutropenia, WBC decreased, and platelet count decreased are each listed as 7.0%. These entries describe the registry’s reported safety information; they do not supply a comparison with another treatment or background rate.
Precision cannot be assessed for the primary endpoint because no estimate or confidence interval is reported. A confidence interval, in general, describes a range of values compatible with the data under a specified method; without the endpoint result and its analysis details, that uncertainty cannot be characterized. The registry record also reports zero analyses, so it does not provide an inferential analysis for the primary endpoint. The available information should be read as a record of the study design, endpoint definition, and selected safety counts, rather than as a complete report of efficacy findings.
9. Statistical Methods Explained
What does the study design allow?
In general, a single-group study describes outcomes among participants receiving the intervention, without a concurrent randomized control group. The registry record identifies this study as single-group and records allocation as NA. Consequently, the design as listed supports a description of outcomes in the enrolled study population, but does not provide a randomized estimate of the difference between gemcitabine and cisplatin and another treatment. Differences between this group and outcomes from another source could reflect differences in participants, care, follow-up, or measurement, as well as treatment.
The study is labeled Phase II. That phase designation does not, by itself, specify the planned statistical model, the target effect, or a threshold for deciding whether the endpoint was met. Those details are not reported in the registry record. The record also does not report the rationale for the enrollment target or the method used to select participants.
How should a count endpoint be understood?
The primary outcome is a count of participants meeting a binary response definition: each evaluable participant either meets both specified pathologic criteria or does not. In general, a response rate is the count meeting the definition divided by the appropriate analysis denominator. That denominator could depend on prespecified rules for participants who did not undergo surgery or lacked an evaluable specimen. Those rules, the number evaluable, and the response count are not reported in the registry record, so a rate cannot be reconstructed.
For a binary endpoint, statistical uncertainty depends on the number meeting the definition and the denominator, as well as the method used to calculate an interval. A confidence interval communicates the precision of an estimate under its assumptions; it is not a guarantee that the true value lies within the reported limits. No primary response estimate or interval is reported in the registry record, so neither the magnitude nor the precision of the response can be assessed.
What can be inferred from the listed analyses?
The registry record lists zero analyses. It therefore does not report a statistical test, model-based estimate, confidence interval, or adjustment for participant characteristics for the primary outcome. It is not possible to infer that no analysis was performed outside the registry record; the appropriate statement is that such analyses are not reported in the registry record.
In general, over-dispersion refers to greater variability in data than a statistical model assumes. It can affect uncertainty estimates and tests when the model does not account for that extra variation. For a simple participant-level binary outcome, the key information needed to assess the response rate is the number meeting the definition and the analysis denominator. The registry record does not identify a model or report enough endpoint data to assess whether over-dispersion was considered or relevant to the sponsor’s analysis.
How should planning illustrations be read?
General sample-size calculations are planning exercises, not observed findings. The stated planning assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the target effect expressed as a fraction of the observed effect. The stated method says that time-to-event planning counts total events, while other planning counts refer to participants per arm. These assumptions describe hypothetical planning calculations; they are not the design of this single-group study and should not be read as the number of participants actually needed or as observed power.
Observed power is not a substitute for an estimate and its uncertainty. In general, a planning calculation asks how much information might be needed under specified assumptions, whereas an analysis of observed data estimates an outcome from the data collected. The registry record does not report the observed primary response effect or a sponsor analysis, so the assumptions cannot establish the study’s actual precision or efficacy result. No recalculated primary result is available to interpret, and there is no sponsor analysis in the registry record to compare with a recalculation.
What do the outcome timeframes imply?
The primary endpoint timeframe is listed as up to 2 years. A timeframe identifies the period associated with an outcome in the record; by itself, it does not explain the exact assessment schedule, handling of participants with shorter follow-up, or whether all participants had complete ascertainment. Those details are not reported in the registry record. General statistical interpretation of a time-to-event outcome also depends on event definitions, censoring rules, and follow-up completeness. Numerical progression-free survival and overall survival results, along with those analysis details, are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists four outcome measures, including pathologic response as the primary outcome and progression-free survival rate, overall survival, and number of participants evaluated for toxicity as secondary outcomes. It does not report a multiplicity strategy, such as whether any secondary outcomes were intended to be confirmatory or how statistical error across multiple tests would be handled. Because zero analyses are listed, no test results are available to assess.
In general, multiplicity arises when several hypotheses are tested or several outcomes are examined. Without an analysis plan, readers cannot determine which comparisons were prespecified, whether the primary endpoint had a designated testing procedure, or how secondary results should be interpreted in relation to it. The registry record does not report an interim-analysis plan or stopping boundaries.
The primary endpoint requires a final surgical specimen, but the registry record does not report the number of participants with an evaluable specimen, the analysis population, or how participants without an assessment were handled. The enrolled count of 57 is not necessarily the denominator for the endpoint. Missing-data rules, including any imputation or sensitivity analyses, are not reported in the registry record.
For survival outcomes, interpretation would also depend on follow-up duration, event definitions, and the treatment of participants whose outcome status was not known at the end of observation. These details and numerical survival results are not reported in the registry record. The absence of these details does not establish that data were missing or that a particular method was used; it means those matters cannot be evaluated from the registry record.
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 |
|---|---|---|---|
| Gemcitabine and Cisplatin | 22 | 57 | 38.6% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Gemcitabine and Cisplatin | 11 of 57 |
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 |
|---|---|
| Gemcitabine and Cisplatin | 57 of 57 |
Most frequent serious adverse event terms
| Event term | Organ system | Gemcitabine and Cisplatin |
|---|---|---|
| Lymphocyte count decreased | Investigations | 19 of 57 |
| Neutrophil count decrease | Investigations | 18 of 57 |
| Hyperglycemia | Metabolism and nutrition disorders | 8 of 57 |
| Febrile Neutropenia | Blood and lymphatic system disorders | 4 of 57 |
| WBC decreased | Investigations | 4 of 57 |
| Platelet count decreased | Investigations | 4 of 57 |
| Hypocalcemia | Metabolism and nutrition disorders | 4 of 57 |
| Thromboembolic event | Vascular disorders | 4 of 57 |
| Anemia | Blood and lymphatic system disorders | 3 of 57 |
| Creatinine increased | Investigations | 3 of 57 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Gemcitabine and Cisplatin |
|---|---|---|
| Hyperglycemia | Metabolism and nutrition disorders | 53 of 57 |
| Fatigue | General disorders | 48 of 57 |
| Hypocalcemia | Metabolism and nutrition disorders | 45 of 57 |
| White blood cell decreased | Investigations | 45 of 57 |
| Creatinine increased | Investigations | 42 of 57 |
| Platelet count decreased | Blood and lymphatic system disorders | 41 of 57 |
| Hypoalbuminemia | Metabolism and nutrition disorders | 36 of 57 |
| Neutrophil count decreased | Investigations | 36 of 57 |
| Constipation | Gastrointestinal disorders | 30 of 57 |
| Hypomagnesemia | Metabolism and nutrition disorders | 30 of 57 |
Serious events with the highest arm-level rate: lymphocyte count decreased (33.3%), neutrophil count decrease (31.6%), hyperglycemia (14.0%), febrile neutropenia (7.0%), wbc decreased (7.0%).
The registry record lists 57 participants at risk for serious adverse events and 22 participants affected. It also lists 57 participants at risk for other adverse events and 57 affected. These counts describe the recorded safety groups; they do not compare the regimen with a control group.
The most frequently listed serious adverse event terms are lymphocyte count decreased (33.3%), neutrophil count decrease (31.6%), and hyperglycemia (14.0%). Febrile neutropenia, WBC decreased, and platelet count decreased are each listed as 7.0%. The record does not give additional event-level details here, such as timing, severity grades, or whether a participant could contribute to more than one term. The percentages should therefore be read as the registry’s reported term frequencies, not as mutually exclusive categories.
The record also lists 11 deaths. It does not report their causes, timing, or relationship to treatment. These counts alone cannot establish that the regimen caused an adverse event or death, and they do not quantify risk relative to another treatment. No medical advice or treatment conclusion follows from these registry figures.
12. What the Result Does, and Does Not, Mean
The registry record documents a Phase II, single-group study of gemcitabine and cisplatin before surgery in patients with high-grade upper tract urothelial carcinoma. It defines pathologic response using findings in the final nephroureterectomy specimen: absence of high-grade carcinoma at the stated <pT2 threshold and absence of microscopic lymph node metastases, N0. The record also describes a 12-week course consisting of four 21 day cycles.
What the record does not report is the numerical primary response result. Without the number meeting the definition and the number evaluable, readers cannot determine the observed response rate or its precision. The named progression-free survival and overall survival outcomes also have no numerical results reported in the registry record. A completed status and an enrollment count do not substitute for outcome estimates.
The safety information includes counts of participants affected and selected serious adverse event terms, as well as 11 deaths. These details can describe what was recorded in the study group, but do not identify causation or provide a comparison with another group. The lack of a concurrent comparator also means that a treatment effect relative to an alternative cannot be estimated from the listed design.
In general, a study can contribute information about feasibility, observed outcomes, and safety reporting even when it does not provide a randomized comparison. The strength of any interpretation depends on complete outcome reporting, a clearly defined analysis population, and suitable estimates of uncertainty. Those elements are not reported for the primary endpoint in the registry record. Accordingly, the record supports a description of the question asked and some safety counts, but not a conclusion about the size or precision of the pathologic response or comparative benefit.
13. Why This Trial Matters Statistically
This study raises a statistical question that is distinct from a treatment comparison: how often did the specified pathologic response occur among patients receiving neoadjuvant gemcitabine and cisplatin in this study? A single-group design can describe that frequency if the outcome count and denominator are reported. The registry record provides the response definition but not the response result, so the central quantity needed to answer the question is unavailable.
The endpoint combines tumor and lymph node criteria. This makes the definition clinically specific, but also means that the response count depends on complete assessment of both parts in the surgical specimen. The registry record does not report how many participants had specimens suitable for assessment or how missing assessments were handled. Those details are essential to understanding the denominator and any resulting estimate.
The design also illustrates why phase and enrollment alone do not establish evidence strength. The study is Phase II and lists 57 participants, but the record does not report the primary analysis, an uncertainty interval, or a concurrent control. Without those elements, readers cannot quantify the response estimate’s precision or compare outcomes under alternative treatment conditions.
The safety entries show a different aspect of statistical interpretation: counts and listed event frequencies can be informative descriptions while still falling short of causal evidence. The adverse event and death counts do not establish attribution or comparative risk. Clear reporting of outcome denominators, follow-up, analysis populations, and uncertainty would help readers distinguish what was observed from what can be inferred.
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 limitation for interpretation is that the registry record does not report a numerical result for the primary pathologic response endpoint. It gives the response definition and a timeframe of up to 2 years, but not the number meeting the criteria, the analysis denominator, or an uncertainty estimate. The endpoint’s precision and observed magnitude therefore cannot be assessed.
The study is listed as single-group, with allocation recorded as NA and masking as none. There is no concurrent comparator listed, so the design does not provide a direct randomized comparison with another regimen. Any descriptive outcome would apply to the study group and would not, by itself, establish what would have occurred under an alternative treatment.
The registry record lists 57 enrolled participants, but does not report how many were treated, proceeded to surgery, had an evaluable specimen, or entered the primary analysis. It also does not report rules for missing assessments, the analysis population, or whether participants who did not complete the described course were included. These omissions make it impossible to determine the appropriate endpoint denominator.
Numerical results for progression-free survival and overall survival are not reported. Follow-up details and analysis methods for those outcomes are also not reported in the registry record. For safety, the record gives selected counts and term frequencies but not causes or timing of the 11 deaths, or further event-level context. These specific reporting limits constrain conclusions about efficacy, precision, comparative effects, and attribution of safety events.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Gemcitabine and Cisplatin as Neoadjuvant Chemotherapy in Patients With High-Grade Upper Tract Urothelial Carcinoma — NCT01261728.
- Registry record last updated 2026-09-02; status last verified 2025-08.
- Results first posted 2026-09-02.
- 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 record describes a completed Phase II, single-group study of gemcitabine and cisplatin as neoadjuvant chemotherapy for high-grade upper tract urothelial carcinoma. It lists 57 participants enrolled, no masking, and allocation as NA. The brief summary describes four 21 day cycles over 12 weeks before surgery.
The primary outcome is pathologic response, defined by absence of high-grade carcinoma (<pT2 disease) and absence of microscopic lymph node metastases (N0) in the final nephroureterectomy specimen. Its timeframe is listed as up to 2 years. The registry record does not report a numerical response result or an analysis for that endpoint. Progression-free survival rate and overall survival are named secondary outcomes, but numerical results are not reported.
Safety information lists 22 participants with serious adverse events among 57 at risk, 57 participants with other adverse events among 57 at risk, and 11 deaths. The record does not provide a randomized comparator or enough detail to establish cause or comparative risk.