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 Safety And Efficacy Study Of Sunitinib Malate As First-Line Systemic Therapy In Chinese Patients With Metastatic Renal Cell Carcinoma (NCT00706706), a non-randomized phase 4 study. The primary outcome is Progression-free Survival (PFS).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00706706 |
| Phase | Phase 4 |
| Condition | Carcinoma, Renal Cell |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 105 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Pfizer |
| Results posted | Yes |
2. Clinical Question
Population
Chinese patients with metastatic renal cell carcinoma. The registry record lists an enrollment of 105; the primary endpoint population is described as participants who had taken at least one dose of study drug.
Intervention
Single-agent sunitinib malate used as first-line systemic therapy. The study is described as a post-approval commitment study.
Comparator
There was no separate control group. The trial was nonrandomized and used a single-group model, so it cannot provide a randomized, like-for-like comparison with another treatment.
Primary question
The primary endpoint was progression-free survival, measured in weeks from the start of study treatment to documented tumor progression or death from any cause. The registry record specifies a median and 95% confidence interval, but does not report their numerical values.
The clinical question was whether first-line single-agent sunitinib could be characterized for safety and efficacy in this patient population after approval. The registry describes the study as open-label and multicenter, with one treatment group. Participants were followed for progression-free survival using oncologic assessment data and adverse-event data when the event outcome was death.
Progression-free survival captures the time until either tumor progression or death, rather than measuring tumor response alone. The registry also lists objective response, overall survival, and one-year survival probability as secondary outcomes. Their numerical results are not reported in the registry record. With no concurrent comparator, the study can describe outcomes observed among treated participants, but cannot isolate a treatment effect from other explanations for those outcomes. The registry record does not report further details about eligibility criteria, the number of participating centers, or the numerical efficacy results.
3. Trial Design
This was a completed, Phase IV, open-label, multicenter, nonrandomized study with a single treatment group. The registry lists an enrollment of 105 and identifies sunitinib malate as the intervention. The study title and brief summary describe first-line systemic therapy for Chinese patients with metastatic renal cell carcinoma. Pfizer is listed as the sponsor. The record gives a start date of 2008-07 and a primary completion date of 2011-08.
A single-group design means that participants received the study intervention without a separate concurrent control arm. Nonrandom allocation and the absence of masking are consistent with the registry’s description of the study as open-label. Consequently, outcome summaries can describe the treated group, but the design does not supply a randomized comparison with another treatment or with no treatment. Any cross-study comparison would also require assumptions that the registry record does not establish.
The stated primary endpoint was progression-free survival, with a median and 95% confidence interval as the planned summary format. The endpoint timeframe describes assessments at baseline and Day 28 of Cycles 1, 2, 3, 4 and even cycles thereafter until disease progression, or every 2 months until death, up to 88 weeks. The population specified for the primary endpoint is the safety population: participants who had taken at least one dose of study drug.
The registry lists three secondary outcomes: percentage of participants with objective response, overall survival, and one-year survival probability. It lists four outcome measures in total. The numerical results for these efficacy outcomes, the statistical analysis plan, and details about follow-up completion are not reported in the registry record. The enrollment figure is a record characteristic; it should not be read as evidence that the study was powered to detect a particular effect.
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 | Sunitinib |
|---|---|
| STARTED | 105 |
| COMPLETED | 0 |
| NOT COMPLETED | 105 |
Reasons for not completing this period:
| Reason | Sunitinib |
|---|---|
| Death | 53 |
| Lost to Follow-up | 7 |
| Study endpoint had reached | 45 |
Analysis population for the primary outcome, as defined in the registry: Safety population included those participants who had taken at least one dose of the study drug.
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 | Sunitinib |
|---|---|---|
| Participants analysed | 105 | |
| Age Continuous | Years | 54.6 (SD 12.9) |
| Sex: Female, Male: Female | Participants | 26 |
| Sex: Female, Male: Male | Participants | 79 |
6. Primary Endpoint
The registry lists the primary outcome as Progression-free Survival (PFS), measured over Baseline, Day 28 of Cycles 1, 2, 3, 4 and even cycles thereafter until disease progression or every 2 months until death (up to 88 weeks).
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 95% Confidence Interval |
| Unit | Weeks |
| Endpoint classification | Time-to-Event Analysis |
Progression-free survival is the time from the start of study treatment to the first documentation of objective tumor progression or death from any cause. In this study it was expressed in weeks, with the median specified as the primary summary and a 95% confidence interval as its measure of uncertainty. The registry defines the time calculation as the first event date minus the date of the first study-drug dose plus 1, divided by 7.
This endpoint is used in clinical research because it combines two clinically relevant events into a time-to-event measure. It includes participants who experience progression and those who die before documented progression. In general, time-to-event methods can account for participants whose event has not been observed by the end of their available follow-up, provided that appropriate censoring information and rules are available. The registry record does not report the censoring rules or the number of participants censored.
Progression was determined from oncologic assessment data meeting the criteria for progressive disease, or from adverse-event data when the outcome was death. The specified assessment schedule describes measurements over treatment cycles and follow-up, but the actual assessment completeness is not reported. Nor are the median progression-free survival estimate or its confidence limits reported. The endpoint definition therefore clarifies what was intended to be measured, but does not establish the magnitude or precision of the observed result.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies progression-free survival as the primary endpoint and specifies that it would be summarized by a median and 95% confidence interval. However, it does not report the median or the confidence interval limits. Without those numerical results, the observed progression-free survival cannot be described, and its precision cannot be evaluated from the registry record. The endpoint definition and assessment schedule alone do not indicate how long participants remained progression-free.
The same limitation applies to the secondary efficacy outcomes. Objective response, overall survival, and one-year survival probability are listed, but their results are not reported in the registry record. No sponsor-reported hazard ratios or other comparative effect estimates appear in the registry record of the record. There is therefore no basis for describing an estimated treatment effect or for quantifying uncertainty around one.
The study’s single-group design is central to interpretation. A summary of outcomes in participants who received sunitinib can describe what was observed in that group. It cannot, by itself, show what would have happened to comparable participants under another treatment or without study treatment. Nonrandom allocation means the trial does not create a randomized control comparison. The open-label design also means that treatment assignment was not masked, although the registry record does not report whether particular assessments or analyses were independently reviewed.
The registry lists an enrollment of 105, while the primary endpoint population is defined as participants who took at least one dose. The number who received a dose, the number with evaluable endpoint information, and the number included in the reported efficacy analysis are not reported in the registry record. Enrollment alone should not be treated as the denominator for every endpoint without those details.
The record lists serious adverse event terms and percentages, but these safety summaries do not supply the missing efficacy estimates. Nor do the listed event terms establish whether an event was caused by treatment. The record’s serious-event figures describe recorded events among participants at risk, while the relevant attribution and analysis details are not reported.
Overall, the available registry information supports a description of the study question, design, endpoint definition, and some adverse-event summaries. It does not support a numerical account of progression-free survival or the secondary efficacy results. Any interpretation beyond those descriptive details would require outcome data and analysis details that are not reported in the registry record.
9. Statistical Methods Explained
What does a median time-to-event result describe?
In general, a time-to-event endpoint records how long it takes for a defined event to occur. For progression-free survival, the event is the first documented tumor progression or death from any cause. The median is the point in the estimated time distribution at which half of the relevant population’s event experience lies on either side, subject to the method used to estimate the time-to-event curve. It is not an average, and it does not describe the experience of every participant.
The registry specifies the median as the primary summary and gives weeks as the unit. It also describes the event-time calculation from the first dose to the first event. But the numerical median is not reported in the registry record. Details of the estimator used, the number of events, and the treatment of participants without an observed event are also not reported. Thus, the endpoint description does not permit reconstruction of the result.
What does a confidence interval contribute?
In general, a confidence interval expresses statistical uncertainty around an estimate under the assumptions of the analysis. A narrower interval indicates greater precision than a wider interval, but an interval does not remove bias from study design or measurement. It also does not make a single-group estimate into a comparative treatment effect. The registry specifies a 95% confidence interval for the median, but its endpoints are not reported in the registry record. The width and location of that interval therefore cannot be assessed.
How does censoring matter?
In general, some participants may not have a documented event during their observed follow-up. Time-to-event analyses can retain information from such participants by censoring them at a specified time, rather than treating them as if an event had occurred. The validity of the estimate depends in part on appropriate censoring rules and assumptions. The registry gives an assessment schedule and states that progression-free survival ends at progression or death, but it does not report the censoring rules, follow-up completeness, or number censored. These details are important to judging the reliability of a time-to-event estimate.
What can be inferred from a single-group study?
In general, a single-group study can summarize the outcomes observed among participants receiving an intervention. Without a concurrent control group, it cannot estimate a randomized contrast between treatments. Differences between an observed outcome and an outcome from another source may reflect differences in patient characteristics, follow-up, outcome definitions, or other factors. Those potential explanations cannot be resolved by the design described in this record. No comparative hazard ratio is reported in the registry record.
How should planning illustrations be distinguished from trial analysis?
In general, a sample-size calculation explores how many participants or events might be needed under chosen assumptions. Such a calculation is a planning illustration, not an observed trial result and not evidence that the study had a particular probability of detecting an effect. The planning assumptions described for the illustrations use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is stated as a fraction of the observed effect. For time-to-event illustrations, the calculation is based on the total number of events; other illustrations are expressed as participants per arm.
Those assumptions are not reported as the trial’s own design. The study itself is single-arm, so the equal-allocation two-arm assumption in an illustration should not be mistaken for the actual allocation. The registry record does not report the trial’s sample-size justification, a target effect, or a protocol-based power calculation. The observed effect needed to interpret such illustrations is also not numerically reported in the registry record. Accordingly, no planning value should be read as an observed result or as observed power.
What does the registry report about analyses?
The registry lists zero analyses. This describes the registry record and does not establish that no analysis was performed. The analysis methods, model specifications, confidence-interval procedure, adjustment variables, and analysis dates are not reported in the registry record. The available information identifies the intended endpoint and summary type, but not the computational details required to reproduce or audit the efficacy estimate.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists progression-free survival as the primary outcome and objective response, overall survival, and one-year survival probability as secondary outcomes. Considering several outcomes raises a general statistical issue: when many hypotheses are tested, the chance of at least one apparently notable finding can increase unless the analysis plan anticipates this. The record does not report a multiplicity adjustment, a testing hierarchy, or another strategy for controlling interpretation across endpoints. It is therefore not possible to determine how the sponsor handled multiplicity.
The registry lists zero analyses, but that entry should not be taken to mean that no analyses were conducted. The analysis plan and the numerical results for the listed efficacy outcomes are not reported in the registry record. The method used to handle missing outcome assessments, incomplete follow-up, or participants who discontinued treatment is also not reported. For a time-to-event endpoint, the number censored and the censoring rules are relevant, but these details are not available.
The primary endpoint population is described as the safety population, consisting of participants who had taken at least one dose of study drug. The registry does not report a separate intention-to-treat population, an endpoint-specific analysis population, or counts of participants included in each analysis. It also does not report how participants with no post-baseline assessment were handled. These omissions limit assessment of possible selection from enrollment to analysis and of how broadly the results represent the enrolled group.
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 |
|---|---|---|---|
| Sunitinib | 13 | 105 | 12.4% |
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 |
|---|---|
| Sunitinib | 102 of 105 |
Most frequent serious adverse event terms
| Event term | Organ system | Sunitinib |
|---|---|---|
| Disease progression | General disorders | 3 of 105 |
| Pneumonia | Infections and infestations | 2 of 105 |
| Right ventricular dysfunction | Cardiac disorders | 1 of 105 |
| Death | General disorders | 1 of 105 |
| Blood creatinine increased | Investigations | 1 of 105 |
| Platelet count decreased | Investigations | 1 of 105 |
| Cerebral haemorrhage | Nervous system disorders | 1 of 105 |
| Haematuria | Renal and urinary disorders | 1 of 105 |
| Renal failure | Renal and urinary disorders | 1 of 105 |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | 1 of 105 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Sunitinib |
|---|---|---|
| Palmar-plantar erythrodysaesthesia syndrome | Skin and subcutaneous tissue disorders | 67 of 105 |
| White blood cell count decreased | Investigations | 55 of 105 |
| Fatigue | General disorders | 54 of 105 |
| Platelet count decreased | Investigations | 54 of 105 |
| Diarrhoea | Gastrointestinal disorders | 51 of 105 |
| Decreased appetite | Metabolism and nutrition disorders | 45 of 105 |
| Neutrophil count decreased | Investigations | 41 of 105 |
| Haemoglobin decreased | Investigations | 39 of 105 |
| Hypertension | Vascular disorders | 39 of 105 |
| Mouth ulceration | Gastrointestinal disorders | 30 of 105 |
Serious events with the highest arm-level rate: disease progression (2.9%), pneumonia (1.9%), right ventricular dysfunction (1.0%), death (1.0%), blood creatinine increased (1.0%).
The registry lists 105 participants at risk for serious adverse events and 13 participants affected. It also lists 105 participants at risk for other adverse events and 102 participants affected. These are recorded safety counts; they do not establish that an event was caused by sunitinib. The registry record does not report the attribution criteria or provide further information about event duration, recurrence, or severity beyond the serious-event classification.
The listed serious adverse event terms and percentages are Disease progression, 2.9%; Pneumonia, 1.9%; Right ventricular dysfunction, 1.0%; Death, 1.0%; Blood creatinine increased, 1.0%; and Platelet count decreased, 1.0%. These are the registry’s reported terms and percentages; they should not be interpreted as comparative risks because there is no control group. The record does not provide a full event-by-event narrative or an assessment of causal relationship.
The deaths field is not populated in the safety summary. The separate listed serious-event term Death, 1.0%, does not supply a complete mortality analysis. Details such as timing, cause, and relation to treatment are not reported in the registry record. The event figures therefore describe reported safety summaries, not a complete comparative safety assessment.
12. What the Result Does, and Does Not, Mean
The registry record describes a completed Phase IV investigation of single-agent sunitinib malate as first-line systemic therapy in Chinese patients with metastatic renal cell carcinoma. It specifies a primary endpoint, progression-free survival, and gives the endpoint definition, time unit, assessment schedule, and intended summary as a median with a 95% confidence interval. It also lists three secondary outcomes and selected serious adverse event summaries.
Those details do not reveal the numerical primary result. The median progression-free survival and confidence interval are not reported in the registry record. Results for objective response, overall survival, and one-year survival probability are also not reported. As a result, the record does not establish the observed magnitude or precision of the primary endpoint, nor does it allow numerical interpretation of the listed secondary endpoints.
The trial’s single-arm, nonrandomized design matters for what can be concluded. Outcomes among participants treated in the study can be described, but the design does not provide a concurrent randomized comparison. Even a numerical outcome estimate would describe the treated group rather than identify what would have happened to comparable patients under an alternative. The absence of masking and the lack of reported analysis details add further limits to assessment.
The listed adverse-event counts and terms are safety summaries, not proof of treatment causation. Without a comparator, they cannot establish whether an event occurred more or less often than it would under another treatment. The record also does not report enough detail to interpret the deaths field or assess missing safety information.
In general, registry entries can document intended endpoints and selected results, but interpretation depends on complete outcome reporting and transparent methods. For this study, the available record supports a careful description of the design and endpoint definitions. It does not support a claim that treatment did or did not improve outcomes, nor does it support a comparative estimate of efficacy or safety.
13. Why This Trial Matters Statistically
This trial is statistically notable as a single-arm, post-approval study with a defined time-to-event primary endpoint. Its registry entry makes clear that progression-free survival was intended to be summarized by a median and a 95% confidence interval, and it defines progression or death as the events of interest. Those specifications help distinguish the primary question from the listed secondary outcomes of objective response, overall survival, and one-year survival probability.
The design also illustrates a key distinction between describing outcomes and estimating comparative effects. A treatment group’s observed progression-free survival can be informative as a description, but nonrandomized single-group data do not create a like-for-like control comparison. Statistical precision around a single-group estimate, even if reported, would not address that design limitation.
Another important issue is the difference between a registry record’s stated outcome and the numerical result needed to interpret it. Here, the primary estimate and its confidence interval are not reported in the registry record. The endpoint definition is useful for understanding what was planned to be measured, but it cannot substitute for the estimate, uncertainty interval, event counts, or analysis details.
The record also shows why planning calculations must remain distinct from trial results. Illustrations based on an observed effect and specified assumptions are not evidence that the study used those assumptions in its design, nor are they observed power. The trial’s own sample-size justification is not reported in the registry record. Together, these points make the study a useful example of how design, endpoint definition, reporting completeness, and causal interpretation are separate statistical questions.
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 registry record does not report the numerical median progression-free survival or its 95% confidence interval. It also does not report numerical results for the secondary outcomes. This prevents assessment of the magnitude and statistical precision of the efficacy findings from the record.
The study used a single-group, nonrandomized design with no like-for-like control arm. Thus, it cannot provide a randomized comparative estimate. The open-label design is reported, but the record does not describe any independent or blinded outcome review. The number of participating centers is not reported beyond the multicenter designation.
The record does not report eligibility criteria in detail, the number who received at least one dose, the number included in each endpoint analysis, or the extent of follow-up. Although the primary endpoint population is described as the safety population, endpoint-specific analysis populations are not reported. Censoring rules, missing-data handling, and the number of censored participants are also not reported, limiting evaluation of the time-to-event analysis.
Further limitations include the absence of reported statistical analysis methods, a sample-size justification, and a multiplicity strategy. The registry lists zero analyses, but this does not establish that no analysis was conducted. Safety summaries list affected participants and selected serious event terms, but do not report attribution criteria or a complete mortality analysis. These gaps restrict both reproducibility and the scope of conclusions that can be drawn from the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Safety And Efficacy Study Of Sunitinib Malate As First-Line Systemic Therapy In Chinese Patients With Metastatic Renal Cell Carcinoma — NCT00706706.
- Registry record last updated 2013-01-18; status last verified 2013-01.
- Results first posted 2012-12-20.
- 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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Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
The registry describes a completed, open-label, multicenter, nonrandomized Phase IV study of single-agent sunitinib malate as first-line systemic therapy in Chinese patients with metastatic renal cell carcinoma. It lists an enrollment of 105, with Pfizer as sponsor, a start date of 2008-07, and a primary completion date of 2011-08.
Progression-free survival was the primary endpoint, defined as time from the first dose to documented tumor progression or death from any cause. The intended summary was a median in weeks with a 95% confidence interval. Objective response, overall survival, and one-year survival probability were listed as secondary outcomes. Numerical efficacy results and detailed analysis methods are not reported in the registry record. Selected serious adverse event counts and terms are listed, but do not provide a controlled comparison or establish causation.