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 Genetic Variates of Response to Cisplatin, Vinblastine, and Temozolomide (CVT) in Patients With Metastatic Melanoma (NCT00885534), a na phase 2 study. The primary outcome is Overall Response to CVT Chemotherapy..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00885534 |
| Phase | Phase 2 |
| Condition | Melanoma, Skin Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 7 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Memorial Sloan Kettering Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
Patients with stage III or IV melanoma, measurable disease, no prior cytotoxic chemotherapy, and disease not thought to be curable by surgery. The registry record reports enrollment of 7.
Intervention
The chemotherapy combination of cisplatin, vinblastine, and temozolomide, referred to as CVT.
Comparator
There was no comparator arm. The trial is described as single-group, and allocation is listed as not applicable.
Primary question
What overall response to CVT was recorded, using RECIST v1.0 criteria for target lesions assessed by MRI over 2 years?
The investigators also aimed to learn whether tumor gene activity could help predict response to CVT. The brief summary describes an interest in identifying genes that are turned on or off in tumors and using that information to understand which tumors might respond. The registry record does not report results for a genetic predictor or a gene-expression analysis.
The response question is a treatment-outcome question within the enrolled group, not a comparison of CVT with another treatment or with no treatment. Without a comparator, the study can describe the responses recorded among participants, but it cannot estimate a relative treatment effect against a control group. The clinical question is also distinct from the genetic prediction aim: an overall response count alone would not establish whether any gene-based measure predicts response.
The brief summary mentions that investigators had previously observed tumors becoming smaller in 30-40% of cases with CVT. That statement is background context, not a result from this trial. The response outcome for the current study is not reported in the registry record, so the extent of response in these enrolled participants cannot be determined from the posted results.
3. Trial Design
The registry describes a completed phase II study at a single institution, with a single-group model and no masking. Allocation is listed as not applicable. The intervention was cisplatin, vinblastine, and temozolomide, administered as the chemotherapy combination CVT. Enrollment is reported as 7. The registry dates the study start to 2009-04 and primary completion to 2013-10.
The arm description identifies a population with stage III or IV melanoma, measurable disease, no prior cytotoxic chemotherapy, and disease not thought to be curable by surgery. The primary endpoint is overall response to CVT, assessed over 2 years. The registry specifies RECIST v1.0 criteria for target lesions assessed by MRI. It defines overall response as complete response plus partial response.
This design can describe outcomes among participants who received the regimen. It does not include a concurrent comparator, so a difference between CVT and another strategy cannot be estimated from this trial. The registry also does not describe random allocation. These features matter when interpreting any response count: observed outcomes in a single group do not by themselves separate treatment effects from the course of disease or other influences.
The record lists 1 outcome measure and no analysis entries. Although the study's stated aims include learning about tumor genes and response prediction, a genetic analysis result is not reported in the registry record. The primary response result is also not reported. Thus, the design and endpoint can be described, but the posted record does not permit an assessment of the trial's response estimate, its uncertainty, or whether the genetic aim was achieved.
The adverse-event listing includes a CVT group with 6 participants at risk for serious events and 2 affected, as well as 6 at risk for other events and 6 affected. These safety counts are distinct from the unreported primary response outcome. The registry status is completed, but completion status alone does not supply outcome estimates or show which planned analyses were performed.
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 | Cisplatin, Vinblastine, Temozolomide |
|---|---|
| STARTED | 7 |
| COMPLETED | 6 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Cisplatin, Vinblastine, Temozolomide |
|---|---|
| Patient deemed ineligible | 1 |
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 | Cisplatin, Vinblastine, Temozolomide |
|---|---|---|
| Participants analysed | 7 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 3 |
| Age, Categorical: >=65 years | Participants | 4 |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 6 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Response to CVT Chemotherapy., measured over 2 years.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is overall response to CVT chemotherapy, with a timeframe of 2 years. It is defined using RECIST v1.0 criteria for target lesions assessed by MRI. A complete response means disappearance of all target lesions. A partial response means at least 30% decrease in the sum of the longest diameter of target lesions. Overall response combines complete and partial responses.
This endpoint classifies whether a participant's measured target lesions meet response criteria. It provides a structured way to describe tumor-size change using specified imaging criteria rather than relying only on an informal impression. Because the endpoint is reported as a number of participants, its interpretation would depend on the response count and the group being evaluated.
The registry record does not report the number of participants who achieved overall response. It therefore does not provide the trial's response proportion, nor an estimate of uncertainty around that result. The response definition is available, but the result needed to apply that definition to this study is not reported in the registry record.
Overall response is also not the same as a genetic prediction result. A response endpoint can describe tumor outcomes, while evaluating prediction would additionally require reported genetic measurements and an analysis relating those measurements to response. Such results are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The main interpretive limitation is that the registry record gives the primary endpoint definition but not its result. There is no posted count of complete responses, partial responses, or overall responses. Without that count, the response experience of participants in this study cannot be quantified from the registry record. No confidence interval or other precision estimate for the primary endpoint is reported either.
The trial is single-group and has no comparator. Even if the response count were available, it would describe outcomes among participants who received CVT; it would not show how those outcomes compare with a different treatment or with no treatment. The design does not support a randomized treatment contrast. The absence of a comparator is especially important when interpreting an observed response: a response count in a treated group is not, by itself, an estimate of benefit relative to an alternative.
The enrollment is 7, while the safety listing reports 6 at risk for each of its event categories. The registry record does not explain the relationship between the enrollment figure and those safety denominators. The reported safety counts should therefore be read with their stated denominators and not assumed to represent the same analysis population as the primary endpoint.
The brief summary reports earlier experience in which tumors became smaller in 30-40% of cases. This is background information about prior use of CVT, not the result of the current study. It cannot fill the gap in the current trial's response reporting, and it does not establish how the enrolled participants responded.
The stated objective also includes identifying tumor genes that might help predict response. No genetic results, predictive model, or association between gene activity and response is reported in the registry record. The genetic objective should therefore be treated as an aim, not as an achieved finding.
General statistical context: a confidence interval expresses the range of values compatible with an estimate under the assumptions of the analysis. It helps show precision, rather than only whether an estimate is statistically significant. Because the response estimate itself is not reported, no such precision assessment can be made for this endpoint from the registry record. A non-significant result, if one existed, would not automatically establish no effect; however, no test result is reported to interpret.
The record lists no analysis entries. It does not report an analysis method, a hypothesis test, a p-value, an adjusted estimate, or a missing-data approach for the primary outcome. The lack of these details does not show that no analyses were performed; it means the registry record does not document them. Accordingly, interpretation should remain limited to the recorded design, endpoint definition, enrollment, and safety figures.
9. Statistical Methods Explained
The outcome is binary at the participant level: a participant either meets the registry's overall-response definition or does not. The registry describes the endpoint as a number of participants, not as a continuous measure of tumor-size change. The response definition combines complete and partial responses, while the registry record does not report the count in either category or their combined total.
General statistical context: for a binary outcome, the basic descriptive result is often a count and the proportion of participants meeting the definition. A proportion is easier to interpret when its numerator and denominator are both clear. The denominator should represent the participants included in the outcome analysis, which may not always be identical to enrollment. Here, the response denominator and response count are not reported in the registry record, so neither the proportion nor its precision can be evaluated.
General statistical context: uncertainty around a proportion can be expressed with a confidence interval. Such an interval reflects sampling variation and gives readers a sense of how precisely the underlying response rate is estimated. With limited information about the outcome count and analysis population, no valid interval can be interpreted from this record. It would not be appropriate to infer one from the enrollment figure alone.
The endpoint uses RECIST v1.0 criteria for target lesions assessed by MRI. The complete-response criterion is disappearance of all target lesions; the partial-response criterion is at least 30% decrease in the sum of the longest diameter of target lesions. Combining these categories into overall response simplifies the primary result into a response count, but it also means that the registry's primary endpoint does not separately describe the complete-response and partial-response counts in the posted results.
The trial's single-group structure determines what comparisons can be made. General statistical context: random allocation can balance measured and unmeasured factors between treatment groups, on average, and permits a direct comparison under the trial's design. This study has no comparator arm and allocation is listed as not applicable. As a result, any outcome count would be descriptive for the treated group rather than a randomized contrast. No adjustment method or comparison with an external group is reported in the registry record.
The registry lists no analysis entries. A statistical analysis plan, including the exact estimator, hypothesis test, confidence-interval method, and any covariate adjustment, is not reported in the registry record. It is therefore not possible to determine which statistical procedures were intended or used. No inference about statistical significance can be made from a response endpoint definition alone.
The sample-size assumptions describe a planning illustration based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They state that the effect to detect is a fraction of the observed effect. This illustration is not the trial's own design: the registered study is single-group, not an equal-allocation, two-arm comparison. Any participant counts calculated for that illustration are planning quantities, not observed power and not evidence that the trial had a particular probability of detecting an effect. The registry record does not report the trial's own sample-size justification.
General statistical context: over-dispersion occurs when variation in data exceeds what a model assumes. For binary outcomes, the main concern is whether the model and sampling structure appropriately represent the outcome and participant population; for other outcome types, additional sources of variability may need explicit modeling. No model for over-dispersion or any other statistical model is reported for this trial. Given the absent primary outcome count and analysis details, there is no basis to assess model fit or the robustness of a model-based estimate.
Missing observations can affect an endpoint estimate if participants without an evaluable assessment are handled differently from participants with observed outcomes. General statistical context: analyses may use complete cases, imputation, or other prespecified approaches, and conclusions can depend on those choices. The registry record does not report the number with an evaluable response assessment or how missing response data were handled. It is not possible to determine whether any participants lacked an endpoint assessment or how that might influence a result.
The record also does not report an analysis population definition, such as whether the primary analysis included all enrolled participants or only those meeting particular eligibility or assessment conditions. Such definitions influence both the numerator and denominator of a binary response estimate. The available enrollment figure alone does not establish the endpoint analysis population.
What can be concluded from the posted methods?
The record identifies a single-group phase II design and a clearly defined imaging-based response endpoint. It does not provide the primary endpoint result or enough analysis information to assess an estimate, its precision, or a statistical test. The descriptions of general statistical methods explain what such analyses can do, but they are not claims that those methods were applied in this trial.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 1 outcome measure, the overall response to CVT chemotherapy. The study summary also describes a broader aim of assessing tumor genes and predicting response, but genetic outcomes and analyses are not reported in the registry record. A formal multiplicity strategy, including any adjustment for testing multiple endpoints, genes, or hypotheses, is not reported.
Multiplicity matters when several statistical hypotheses are examined: the chance of at least one apparently notable result can increase as more tests are conducted. General statistical context: investigators may address this by prespecifying a primary hypothesis, using an adjustment, or treating additional analyses as exploratory. The record does not describe which approach, if any, was planned for the genetic objective. It is not possible to assess the scope of testing or the interpretation of any genetic findings from the registry record.
The missing-data approach for the response endpoint is not reported in the registry record. The number of participants with an evaluable MRI assessment, the handling of participants without an assessment, and the analysis population definition are also not reported. These details matter because a response count depends on who is included and how unavailable outcomes are treated.
No analysis entries are listed. This does not establish that analyses were not performed; it indicates that analysis details are not documented in the registry record. The record also does not report interim analyses, stopping rules, or a statistical analysis plan. Those design and analysis details should not be inferred from the completed status or the endpoint description.
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 |
|---|---|---|---|
| Cisplatin, Vinblastine, Temozolomide | 2 | 6 | 33.3% |
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 |
|---|---|
| Cisplatin, Vinblastine, Temozolomide | 6 of 6 |
Most frequent serious adverse event terms
| Event term | Organ system | Cisplatin, Vinblastine, Temozolomide |
|---|---|---|
| Dehydration | General disorders | 2 of 6 |
| Constipation | Gastrointestinal disorders | 1 of 6 |
| Hypotension | Cardiac disorders | 1 of 6 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Cisplatin, Vinblastine, Temozolomide |
|---|---|---|
| Lymphopenia | Blood and lymphatic system disorders | 2 of 6 |
| Glucose, high (hyperglycemia) | General disorders | 2 of 6 |
| Hemoglobin decreased | Blood and lymphatic system disorders | 2 of 6 |
| Creatinine increased | Blood and lymphatic system disorders | 1 of 6 |
| Urogenital disorder | Renal and urinary disorders | 1 of 6 |
| ALT, SGPT | Blood and lymphatic system disorders | 1 of 6 |
| Fever (in the absence of neutropenia) | General disorders | 1 of 6 |
| Potassium, high (hyperkalemia) | Metabolism and nutrition disorders | 1 of 6 |
| Bilirubin (hyperbilirubinemia) | Blood and lymphatic system disorders | 1 of 6 |
| INR increased | Blood and lymphatic system disorders | 1 of 6 |
Serious events with the highest arm-level rate: dehydration (33.3%), constipation (16.7%), hypotension (16.7%).
The adverse-event listing reports 2 serious-event-affected participants among 6 at risk in the CVT group. It also reports 6 affected among 6 at risk for other events. These are separate registry categories; the record does not define the precise distinction between the serious and other-event entries in this listing. They should not be combined into a single total.
The most frequently listed serious-event terms are dehydration at 33.3%, constipation at 16.7%, and hypotension at 16.7%. These percentages should be read as the registry reports them. The listing does not provide further detail about event severity, timing, duration, recurrence, or whether events were considered related to treatment.
The safety denominators are 6, while enrollment is reported as 7. The registry record does not explain this difference. It also does not provide a detailed account of missing safety information or establish whether all enrolled participants contributed to each safety category. The counts describe the posted safety listing, not necessarily every event experienced during the study.
These figures do not establish a comparative safety profile because the study has no comparator arm. They are descriptive safety data for the CVT group, with the limitations of the registry reporting.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a completed phase II, single-group study evaluated CVT in patients with metastatic melanoma and specified overall response as its primary endpoint. It gives the response criteria and the assessment timeframe of 2 years. It also provides enrollment and a limited safety listing.
The record does not report the primary response count or any response estimate. Consequently, it does not show how many participants had complete response, partial response, or overall response. It also does not report uncertainty around the primary outcome. The endpoint definition alone cannot establish whether responses occurred or how often.
The record's background statement that earlier investigators observed tumors becoming smaller in 30-40% of cases is not the result of this trial. It should not be interpreted as an estimate from the enrolled participants. Likewise, the study's stated interest in tumor genes and response prediction is an objective, not evidence that a predictive marker was identified. Genetic results are not reported in the registry record.
Because there is no comparator arm, the design does not provide a randomized comparison of CVT with another treatment. A response count, if reported, would describe outcomes among people treated in this study; it would not by itself demonstrate what would have happened under an alternative. The record also does not report enough statistical analysis detail to evaluate adjustment, precision, missing outcomes, or the analysis population.
General statistical context: statistical evidence depends on both the estimated effect and its uncertainty, as well as on the design and assumptions behind the analysis. A treatment result should not be judged from a design label, an endpoint definition, or a planning calculation alone. For this trial, the missing primary result is the central limitation: the posted registry information supports description of the question and design, but not a conclusion about the observed response outcome.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of the distinction between a clearly described endpoint and a reported endpoint result. The registry specifies how overall response was defined, which imaging criteria were used, and the timeframe for assessment. Those details make the intended outcome interpretable in principle. However, without the response count or an analysis estimate, readers cannot judge the observed response frequency or its precision.
The single-group design also makes the target of inference important. The study can describe outcomes within its treated group, but it has no concurrent control group for estimating a comparative treatment effect. That difference is not repaired by the phase II label or by the stated objective to learn about response. Design determines which comparisons the data can support.
The study's genetic objective illustrates a separate statistical challenge: prediction requires more than identifying a biological aim. It requires reported measurements and an analysis linking those measurements to the clinical outcome, with attention to how many candidate genes or hypotheses were examined. Such results and multiplicity details are not reported in the registry record.
The adverse-event listing further shows why denominators and outcome categories need to be read explicitly. The serious-event and other-event entries have stated risk and affected counts, while the relationship between those denominators and enrollment is not explained. Clear reporting of participant flow, analysis populations, and outcome handling would help readers assess the meaning and limitations of both efficacy and safety summaries.
The record therefore matters as a reminder that registry status, design, and endpoint definitions do not substitute for results. Statistical interpretation depends on transparent reporting of outcomes, uncertainty, and analytic choices.
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 primary limitation for interpretation is that the registry record does not report the response result. It provides no count of participants meeting the complete-response, partial-response, or overall-response definition. No response estimate or uncertainty interval is reported, preventing assessment of the central endpoint.
The study has no comparator arm, so it cannot provide a randomized contrast with another treatment. The enrollment of 7 is small in absolute terms, but the registry record does not report the trial's sample-size justification, and the missing endpoint result prevents assessment of the precision actually achieved. The planning illustration uses assumptions for equal allocation between two arms, which do not match the registered single-group design; those planning assumptions are not the trial's own design description.
The record does not report a statistical analysis plan, primary analysis method, analysis population, or missing-data handling. It does not report genetic results or explain how any genetic testing and analysis were conducted. Multiplicity considerations for a genetic prediction aim are also not reported. These omissions limit assessment of both the clinical endpoint and the study's broader prediction objective.
The safety listing reports denominators of 6, whereas enrollment is 7, without explaining the difference. Additional event details, including timing, duration, severity beyond the serious-event categorization, and treatment relatedness, are not reported in the registry record. The listed safety counts therefore offer a limited description rather than a complete comparative safety assessment.
The registry status is completed, but completion does not resolve the absence of these outcome and analysis details. The conclusions available from the record remain limited to the design, stated objectives, endpoint definition, enrollment, and posted safety figures.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Genetic Variates of Response to Cisplatin, Vinblastine, and Temozolomide (CVT) in Patients With Metastatic Melanoma — NCT00885534.
- Registry record last updated 2015-05-06; status last verified 2015-04.
- Results first posted 2015-05-06.
- 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.
Continue through the Clinical Biostats statistical pathway
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18. Record Summary
This is a completed phase II, single-group study of cisplatin, vinblastine, and temozolomide in patients with stage III or IV melanoma and measurable disease. The registry reports enrollment of 7, a study start of 2009-04, and primary completion in 2013-10. There was no comparator arm, and masking was none.
The primary endpoint was overall response over 2 years, defined by RECIST v1.0 criteria for target lesions assessed by MRI. The registry record does not report the primary response count or a statistical analysis of that endpoint. The study also aimed to explore tumor genes as possible predictors of response, but genetic findings are not reported.
The safety listing reports serious events affecting 2 of 6 at risk and other events affecting 6 of 6 at risk. Listed serious-event terms include dehydration, constipation, and hypotension. The registry does not explain why the safety denominators differ from enrollment or provide a comparative safety analysis.