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 BMS 247550 to Treat Kidney Cancer (NCT00030992), a na phase 2 study. The primary outcome is Response Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00030992 |
| Phase | Phase 2 |
| Condition | Renal Cell Carcinoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 102 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Cancer Institute (NCI) |
| Results posted | Yes |
2. Clinical Question
Population
Adults with renal cell carcinoma were the intended study population. The registry summary describes eligibility for people 18 years of age or older and excludes some participants with central nervous system spread, with an exception for a stable brain tumor after treatment. The registry lists enrollment of 102 participants; further details about the analyzed population are not reported in the registry record.
Intervention
Participants received BMS-247550, also called ixabepilone in the brief summary. The arm description specifies a one-hour infusion on five successive days every three weeks, with a starting dose of 6 mg/m^2/day. Ranitidine and diphenhydramine are also listed as interventions, but their roles are not described in the registry record.
Comparator
There was no concurrent control arm: the trial is listed as a single-group study, with allocation recorded as NA. Consequently, the registry record does not provide a randomized comparison between BMS-247550 and another treatment or a control condition.
Primary question
The primary endpoint asks what percentage of participants had a response assessed using RECIST at 6 weeks. The response categories include complete response and partial response; stable disease and progressive disease are defined separately. The registry record gives the endpoint definition but does not report the number or percentage of participants who responded.
This is a question about observed tumor response in a treated group, not a direct estimate of how the drug compares with another treatment. The single-group design can describe outcomes among participants in the study, but without a concurrent comparator it cannot isolate the effect of treatment from other explanations for those outcomes. The registry lists 102 participants as enrolled, but the number included in the primary endpoint analysis and the handling of participants without an assessment are not reported in the registry record. Accordingly, the available record establishes what response was intended to be measured and when, but does not establish the observed response rate or its precision.
3. Trial Design
This was a Phase II, single-group clinical trial of BMS-247550 in renal cell carcinoma, sponsored by the National Cancer Institute. The registry lists 102 participants enrolled. The study began in 2002-02, had primary completion in 2009-01, and is recorded as completed. These dates describe the registry record; they do not by themselves explain the duration of follow-up for any participant.
The intervention description specifies an infusion lasting one hour on five consecutive days in each three-week cycle, with a starting dose of 6 mg/m^2/day and a total per-cycle starting dose of 30 mg/m^2. The brief summary says the number of cycles could vary according to individual clinical circumstances. The registry lists ranitidine and diphenhydramine as additional drug interventions, but does not explain their use. Masking is listed as none, and allocation as NA.
A single-group design means that participants receive the study intervention without a concurrent comparison arm in the registry record. The design can describe response and safety observations in the study group, but it does not provide a like-for-like control comparison. In particular, it cannot estimate a treatment contrast against an untreated group or another therapy from the listed design alone.
The primary endpoint is response rate at 6 weeks, defined using RECIST. The registry also lists the number of participants with adverse events as a secondary outcome. Although the record specifies these outcomes, it does not report a primary endpoint analysis, an analysis population, or a statistical analysis plan. Details such as the intended enrollment rationale, eligibility criteria beyond the brief summary, and any interim analyses are not reported in 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 | BMS-247550 |
|---|---|
| STARTED | 102 |
| COMPLETED | 102 |
| 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 | BMS-247550 |
|---|---|---|
| Participants analysed | 102 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 79 |
| Age, Categorical: >=65 years | Participants | 23 |
| Age Continuous | years | 57.21 (SD 10.81) |
| Sex: Female, Male: Female | Participants | 24 |
| Sex: Female, Male: Male | Participants | 78 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 2 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 100 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race/Ethnicity, Customized: Caucasian | Participants | 84 |
| Race/Ethnicity, Customized: African American | Participants | 12 |
| Race/Ethnicity, Customized: Asian | Participants | 5 |
| Race/Ethnicity, Customized: Hispanic | Participants | 1 |
| Region of Enrollment: United States | participants | 102 |
6. Primary Endpoint
The registry lists the primary outcome as Response Rate, measured over 6 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the percentage of participants with a response assessed at 6 weeks using the Response Evaluation Criteria in Solid Tumors, or RECIST. The registry definition identifies complete response as disappearance of all target lesions and partial response as at least a 30% decrease in the sum of the longest diameter of target lesions. It defines progressive disease as at least a 20% increase in that sum or the appearance of one or more new lesions. Stable disease is neither sufficient shrinkage for partial response nor sufficient increase for progressive disease.
A response-rate endpoint reduces assessments of tumor change to a defined outcome category. In general, this can provide a clear way to describe how many participants meet prespecified response criteria at a particular assessment time. It does not, by itself, describe how long a response lasts, how symptoms change, or what happens after the specified assessment. Those outcomes require separate measures and analyses.
The registry record does not report the number of participants in each response category, the response percentage, or a confidence interval. It also does not report the number assessed at 6 weeks or how missing assessments were handled. Therefore, the endpoint definition is available, but its observed result and statistical precision cannot be evaluated from this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a response-rate endpoint at 6 weeks and defines response using RECIST, but it does not report the observed response rate. No response count, percentage, confidence interval, or statistical test is listed. The record also lists zero analyses. Thus, the central efficacy result cannot be reconstructed or interpreted from the available registry entries. The absence of a reported estimate is not evidence that the response rate was zero.
The study has a single-group design. Any response observed among participants would describe outcomes in that group, but there is no randomized control group in the record against which to compare those outcomes. This distinction matters: an observed outcome in a treated group is not the same as an estimated difference attributable to treatment. The record does not support a comparative estimate.
The planned assessment time is 6 weeks. The record does not state the number of participants who completed that assessment, whether any participants were excluded from the endpoint analysis, or the analysis population used. Without these details and the actual response result, neither the response frequency nor its uncertainty can be assessed. A confidence interval, in general, expresses the precision of an estimate; no interval for response is reported.
The safety entries provide a separate kind of information. In the BMS-247550 group, the registry lists 102 participants at risk and 40 participants with serious adverse events. It also lists 102 at risk and 99 with other adverse events. These are descriptive counts, not comparisons with another group. The registry lists serious-event terms with percentages, including Fever at 7.8%, Infection without neutropenia at 6.9%, Dehydration at 5.9%, Dyspnea (shortness of breath) at 5.9%, Hypotension at 5.9%, and Diarrhea (without colostomy) at 4.9%. The record does not provide further details about event timing, duration, recurrence, or attribution.
These safety counts should not be combined into a single total: the registry does not state whether participants with serious and other events overlap. Nor do the listed terms establish that each event was caused by BMS-247550. The deaths field has no reported value. Overall, the record gives some descriptive safety figures but does not provide enough detail to assess comparative safety or to connect the listed events to particular treatment cycles.
9. Statistical Methods Explained
Statistical methods determine how trial observations are converted into estimates and how uncertainty is described. The registry identifies a percentage-based primary outcome and a single-group design, but the analysis details and actual efficacy estimates are not reported in the registry record. The explanations below are general and distinguish the meaning of the endpoint from information that is absent from this record.
What does a response percentage represent?
In general, a response percentage is the number of participants classified as having a response divided by the number included in the relevant analysis population. To interpret it, a reader needs both the numerator and denominator, along with the rules used to decide which participants enter that analysis. The registry defines response categories using RECIST and specifies assessment at 6 weeks, but does not report the response numerator, the analyzed denominator, or the handling of participants without an assessment. No response percentage can therefore be stated from this record.
How should a confidence interval be understood?
In general, a confidence interval describes the uncertainty around an estimated quantity under a stated statistical method. A narrower interval indicates greater precision than a wider interval, though precision does not resolve limitations in design or measurement. For a response percentage, an interval would help show how compatible the observed data are with a range of underlying response frequencies. The registry record does not report an interval for the primary endpoint, so its precision cannot be assessed.
What does the single-group structure allow?
In general, a single-group study can summarize outcomes among the participants who received the intervention. It does not provide a concurrent control group for estimating a treatment difference. Even a clearly reported response frequency would be a description of the study group, rather than a randomized comparison. Differences in participant characteristics, disease course, assessment, or other influences cannot be separated from treatment effects using a single group alone.
The registry records allocation as NA and the model as single group. It does not identify a randomized allocation process or a control arm. Therefore, comparative methods that estimate differences between randomized arms do not apply to the design as recorded. No adjustment method or model for the primary response outcome is reported.
What can the safety counts tell readers?
In general, an adverse-event count describes how many participants in a stated group had an event under the registry's reporting definitions. A count alone does not show event duration, severity beyond the category reported, recurrence, timing, or causality. For this trial, the record lists 40 participants with serious adverse events among 102 at risk and 99 participants with other adverse events among 102 at risk. It gives several serious-event terms and percentages, but does not give details on how event terms were selected or whether participants could contribute to more than one category.
The percentages attached to serious-event terms should be read as registry-reported figures, not recalculated estimates. The record does not explain their denominator or provide participant-level details. It also does not report a comparison group or a statistical analysis of safety outcomes. General comparisons of adverse-event frequencies would require comparable groups, definitions, observation periods, and reporting practices.
What do planning assumptions mean?
General sample-size calculations can illustrate how many participants per arm might be needed under specified assumptions about the effect to detect, significance level, allocation, and statistical approximation. Such illustrations are planning aids, not observed trial outcomes and not evidence that a study had a particular probability of detecting an effect. The registry notes that any planning illustration is based on an observed effect and assumptions of two-sided significance level 0.05, equal allocation, and a normal approximation. It also notes that such illustrations are not the trial's own design. The trial itself is single group, so a two-arm planning illustration does not describe its observed design. No sample-size justification for the actual trial is reported in the registry record.
More broadly, a prespecified statistical plan helps clarify the endpoint definition, analysis population, missing-data approach, and any planned subgroup or interim analyses. Those details are not reported in the registry record. Without them and without posted primary endpoint results, readers cannot check whether an analysis followed a protocol or determine how robust the result is to alternative analysis choices.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary response-rate endpoint and a secondary outcome concerning the number of participants with adverse events. It does not report how many hypotheses were tested, whether any adjustment for multiple comparisons was planned, or whether additional endpoints or subgroup analyses were specified. The approach to multiplicity is therefore not reported in the registry record. In general, multiplicity matters when several outcomes or comparisons are tested, because interpreting each test in isolation can overstate the strength of evidence across the full set of analyses.
The record does not report a primary endpoint analysis or response results. It also does not state how missing 6-week assessments were handled, whether participants were analyzed according to a defined analysis population, or how participants who discontinued treatment were counted. These details matter because exclusions and missing outcome assessments can affect the denominator and interpretation of a response percentage. No assumptions about missing outcomes can be inferred from the endpoint definition alone.
Likewise, interim analyses, stopping rules, and the statistical analysis plan are not reported in the registry record. The listed enrollment does not establish the size of the primary analysis population. The safety denominators are separately given as 102 at risk for each listed adverse-event grouping, but that does not establish the analysis population for the response endpoint. Consequently, the registry record supports a description of the intended endpoint and some safety counts, but not a full assessment of analysis choices, missing data, or multiplicity.
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 |
|---|---|---|---|
| BMS-247550 | 40 | 102 | 39.2% |
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 |
|---|---|
| BMS-247550 | 99 of 102 |
Most frequent serious adverse event terms
| Event term | Organ system | BMS-247550 |
|---|---|---|
| Fever | General disorders | 8 of 102 |
| Infection without neutropenia | Infections and infestations | 7 of 102 |
| Dehydration | Metabolism and nutrition disorders | 6 of 102 |
| Dyspnea (shortness of breath) | Respiratory, thoracic and mediastinal disorders | 6 of 102 |
| Hypotension | Vascular disorders | 6 of 102 |
| Diarrhea (without colostomy) | Gastrointestinal disorders | 5 of 102 |
| Neutrophils/granulocytes (ANC/AGC) | Investigations | 5 of 102 |
| Hemoglobin (hgb) | Blood and lymphatic system disorders | 4 of 102 |
| Fatigue (lethargy, malaise, asthenia) | General disorders | 3 of 102 |
| Hematuria (in the absence of vaginal bleeding) | Renal and urinary disorders | 3 of 102 |
Most frequent non-serious adverse event terms
| Event term | Organ system | BMS-247550 |
|---|---|---|
| Fatigue (lethargy, malaise, asthenia) | General disorders | 73 of 102 |
| Hemoglobin (hgb) | Blood and lymphatic system disorders | 65 of 102 |
| Hypoalbuminemia | Metabolism and nutrition disorders | 62 of 102 |
| Alopecia | Skin and subcutaneous tissue disorders | 61 of 102 |
| Anorexia | Metabolism and nutrition disorders | 56 of 102 |
| Neuropathy-sensory | Nervous system disorders | 50 of 102 |
| Nausea | Gastrointestinal disorders | 48 of 102 |
| Neutrophils/granulocytes (ANC/AGC) | Investigations | 46 of 102 |
| Diarrhea (without colostomy) | Gastrointestinal disorders | 42 of 102 |
| Taste disturbance (dysgeusia) | Nervous system disorders | 40 of 102 |
Serious events with the highest arm-level rate: fever (7.8%), infection without neutropenia (6.9%), dehydration (5.9%), dyspnea (shortness of breath) (5.9%), hypotension (5.9%).
The registry lists 102 participants at risk in the BMS-247550 group, with 40 participants recorded as having serious adverse events and 99 as having other adverse events. These counts describe the safety entries as recorded; they are not evidence of a comparison with another treatment. The record also lists serious adverse-event terms and their percentages: Fever, 7.8%; Infection without neutropenia, 6.9%; Dehydration, 5.9%; Dyspnea (shortness of breath), 5.9%; Hypotension, 5.9%; and Diarrhea (without colostomy), 4.9%.
These percentages are registry-reported, and the record does not explain the denominator for each term or whether participants could experience multiple terms. The serious and other adverse-event groups may overlap; the record does not say whether they do. The listed terms should not be interpreted as proof that the intervention caused an event. Event timing, duration, recurrence, and attribution are not reported in the registry record. The deaths value is also not reported, so no conclusion about deaths can be drawn from that field.
12. What the Result Does, and Does Not, Mean
The record documents a completed Phase II, single-group study of BMS-247550 in renal cell carcinoma, with 102 participants enrolled. It defines a primary response-rate endpoint using RECIST at 6 weeks and lists adverse-event outcomes. Those details establish the study's intended question and some descriptive safety information.
The record does not report the primary response count or percentage, a confidence interval, or an analysis. It therefore does not establish what response was observed in the study group or how precisely that response was estimated. Missing response results should not be treated as a finding of no response. The record also does not provide the information needed to assess missing assessments or define the endpoint analysis population.
Because the trial is single group and has no listed comparator, its design does not support a randomized estimate of how outcomes differ from another treatment or control condition. This limitation applies even if a response estimate were available. A response observed in a treated group would be descriptive, not by itself proof that the intervention caused the response.
The adverse-event counts and listed serious-event terms provide a limited descriptive account. They do not establish comparative safety, causation, or the full clinical course of events. The registry does not report event timing, recurrence, or attribution, and the deaths field has no value. Taken together, the record supports statements about the design, outcome definitions, enrollment, and posted safety counts, but not a conclusion about treatment efficacy or comparative safety.
13. Why This Trial Matters Statistically
This trial is statistically useful to examine because it makes clear the difference between defining an endpoint and reporting its result. The registry specifies a response-rate outcome, a 6-week assessment time, and RECIST criteria. Those definitions provide a framework for interpreting a response estimate if one is reported. However, the record does not include the actual response result or an analysis, so the central endpoint cannot be quantified.
The single-group design also illustrates why study structure constrains interpretation. A study can collect outcomes after an intervention and describe them, but a treatment comparison requires a comparator and an appropriate design. The registry lists no concurrent control arm, so it does not provide a randomized treatment contrast. This distinction is important when readers encounter efficacy language in a trial description: the stated aim and the evidentiary scope of the design are not the same thing.
The safety entries offer another statistical lesson. Counts and term-specific percentages can summarize reported events, but interpretation depends on definitions, denominators, time at risk, overlap, and attribution. Several of those details are not reported in the registry record. The entries should therefore be read as descriptive registry data rather than as a complete comparative safety analysis.
Finally, this record shows why analysis populations, missing-data handling, and statistical plans matter. Without these details and a posted endpoint estimate, readers cannot evaluate precision, analysis decisions, or sensitivity to missing outcomes. A transparent endpoint definition is valuable, but it cannot replace an analyzed result and the information needed to interpret it.
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 limitations specific to this record restrict interpretation. First, the primary endpoint is defined, but its observed response count and percentage are not reported. No confidence interval, test result, or primary analysis is listed. The record therefore does not allow the primary clinical outcome to be assessed statistically.
Second, the study is recorded as single group, with allocation listed as NA and masking as none. No concurrent comparator is listed. Any outcomes in the intervention group consequently cannot be interpreted as a randomized comparison against another treatment or control condition.
Third, the registry lists 102 participants enrolled, but the number assessed for response at 6 weeks and the primary endpoint analysis population are not reported. The handling of missing assessments, treatment discontinuations, and participants who were not evaluable is also not reported in the registry record. Those omissions prevent an assessment of the denominator and the potential influence of missing data.
Fourth, safety information is limited to the listed counts and serious-event terms. The record does not explain whether participants appear in both adverse-event groupings, how term-specific percentages were calculated, or the events' timing, duration, recurrence, and attribution. The deaths field has no reported value.
Finally, the registry record does not report a sample-size justification, a statistical analysis plan, multiplicity procedures, or interim analyses. These are gaps in the record, not grounds for assuming that any particular method was or was not used. They limit assessment of how the study was planned and how its outcomes were analyzed.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: BMS 247550 to Treat Kidney Cancer — NCT00030992.
- Registry record last updated 2012-08-20; status last verified 2012-06.
- Results first posted 2012-07-20.
- 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
NCT00030992 is a completed Phase II, single-group study of BMS-247550 in renal cell carcinoma, sponsored by the National Cancer Institute. The registry lists 102 participants enrolled, no masking, and no allocation to a concurrent comparison arm. The primary endpoint is the percentage of participants with a RECIST response at 6 weeks.
The registry record does not report the response result or an analysis. It lists 40 participants with serious adverse events and 99 with other adverse events, each among 102 at risk, along with selected serious-event terms and percentages. Further details about response analysis, missing data, event attribution, and comparative outcomes are not reported in the registry record.