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 Phase II Study of Alternating Sunitinib and Temsirolimus (NCT01517243), a na phase 2 study. The primary outcome is Progression Free Survival.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01517243 |
| Phase | Phase 2 |
| Condition | Metastatic Renal Cell Carcinoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 37 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Dartmouth-Hitchcock Medical Center |
| Results posted | Yes |
2. Clinical Question
The study concerns patients with metastatic renal cell carcinoma and evaluates a regimen that alternates sunitinib with temsirolimus. The registry describes sunitinib as targeting the vascular endothelial growth factor receptor and temsirolimus as an inhibitor of mammalian target of rapamycin. The treatment schedule alternates periods of each drug with rest periods.
The central clinical question is whether progression-free survival can be characterized in patients treated with this alternating targeted-therapy regimen. The registry’s description says the study aimed to determine time to disease progression. Its stated hypothesis was that alternating the agents would double progression-free survival compared with the agents given sequentially. That hypothesis is a rationale recorded for the study, not a result: the registry record does not report an observed progression-free survival estimate or a comparison with sequential treatment.
Population
Patients with metastatic renal cell carcinoma. The registry records enrollment of 37.
Intervention
Alternating sunitinib and temsirolimus. The described cycle uses sunitinib 50mg by mouth daily for 4 weeks, followed by a two week rest, then temsirolimus 25mg IV weekly for 4 weeks, followed by a two week rest.
Comparator
No concurrent control group is described. Allocation is listed as NA and the study model as single group, so the registry does not support a like-for-like comparison with sequential treatment.
Primary question
What is the time to disease progression, expressed as progression-free survival, among patients treated with alternating targeted therapy? The registry’s timeframe also includes death from any cause.
The record also lists clinical response rate and toxicity characterization as secondary outcomes. It does not report the results for these outcomes. Accordingly, the clinical question can be described, but its answer cannot be inferred from the record alone.
3. Trial Design
This is a Phase II study with a single-group model. The registry lists the allocation as NA and masking as none. The study is marked completed and records enrollment of 37. Dartmouth-Hitchcock Medical Center is identified as the sponsor. The recorded start date is 2010-09-22, and the primary completion date is 2018-08-20.
The intervention is a sequence of alternating drug periods. A cycle is described as sunitinib 50mg by mouth daily for 4 weeks, followed by a two week rest, then temsirolimus 25mg IV weekly for 4 weeks, followed by a two week rest. The record names both drugs as interventions and describes one arm, Alternating Sunitinib and Temsirolimus. It does not describe a concurrent control arm.
The design has an important consequence for interpretation: results from a single group can describe outcomes among those treated under the study regimen, but cannot by themselves isolate the regimen’s effect relative to another treatment. The stated hypothesis refers to progression-free survival with alternating treatment compared with the agents given sequentially, but the registry does not describe a sequential-treatment comparison group. A direct comparison would require information from a suitable comparator, which is not reported in the registry record.
The primary endpoint’s timeframe refers to measurement from the date of randomization, with evaluation every 12 weeks throughout study treatment. Yet the design fields identify a single group and allocation as NA. Whether randomization applied in another sense, or how the endpoint’s starting point was implemented, is not reported in the registry record. The registry also does not give a fuller account of eligibility criteria, recruitment, treatment adherence, or follow-up after treatment.
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 | Alternating Sunitinib and Temsirolimus |
|---|---|
| STARTED | 19 |
| COMPLETED | 19 |
| 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 | Number of Participants Classified by Tumor Histology/Cytology |
|---|---|---|
| Participants analysed | 19 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 10 |
| Age, Categorical: >=65 years | Participants | 9 |
| Age, Continuous | Years | 63.0 (SD 10.6) |
| Sex: Female, Male: Female | Participants | 3 |
| Sex: Female, Male: Male | Participants | 16 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 19 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 19 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 19 |
| Tumor Histology/Cytology: Clear Cell | participants | 16 |
| Tumor Histology/Cytology: Papillary | participants | 1 |
| Tumor Histology/Cytology: Unclassified | participants | 2 |
6. Primary Endpoint
The registry lists the primary outcome as Progression Free Survival, measured over From the date of randomization until the first documented disease progression or date of death from any cause, evaluated every 12 weeks throughout duration of study treatment.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 95% Confidence Interval |
| Unit | Months |
| Endpoint classification | Time-to-Event Analysis |
The primary endpoint is progression-free survival, reported as a median in months with a 95% confidence interval. The registry defines the interval as beginning at the date of randomization and ending at the first documented disease progression or death from any cause. It says evaluations occurred every 12 weeks throughout study treatment.
Progression-free survival is a time-to-event endpoint: it measures how long it takes until a specified event occurs. Combining disease progression and death into the event definition gives a measure that captures either outcome, rather than focusing only on tumor response. A median describes the time by which half of the relevant observed event-time distribution has reached the event, subject to the survival-analysis method and available follow-up. A confidence interval communicates uncertainty around the estimated median; it is not a range containing every individual patient’s outcome.
Time-to-event methods can make use of information from people whose event has not been observed by the end of their available follow-up. Such observations are generally treated as censored at the last time their status is known, rather than as if an event occurred then. That general interpretation depends on the analysis handling censoring appropriately. The registry does not report the observed median, its confidence interval, the analysis population, or details of censoring and event ascertainment. Therefore, the endpoint’s definition is available, but its numerical result and precision are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies progression-free survival as the primary endpoint and gives its definition, units, and evaluation schedule. It does not provide the observed median or its 95% confidence interval. Without those estimates, the magnitude and precision of the primary outcome cannot be described from the record. The record also lists no analyses, and it contains no sponsor-reported hazard ratio values. No treatment effect estimate should be inferred from the stated study hypothesis.
The study is described as single group, with allocation listed as NA and masking as none. Its structure can support a descriptive account of outcomes in the group receiving alternating sunitinib and temsirolimus, if the outcome estimates are available. It cannot, by itself, establish how outcomes compare with sequential use of the agents or another treatment. Differences between a single group’s observed outcomes and an external benchmark could reflect differences in patient characteristics, follow-up, measurement, or other aspects of care, rather than treatment alone.
The brief summary gives the rationale that the agents can be given sequentially and states a hypothesis about doubling progression-free survival. This is an expectation motivating the study, not evidence that the expectation was met. The registry does not report a concurrent sequential-treatment group, a comparative estimate, or a test of that hypothesis. It also does not report a numerical response-rate result or a toxicity analysis beyond the adverse-event entries described separately.
The registry’s adverse-event fields list 4 participants affected by serious events in the group, with 19 at risk, and list 19 affected by other events, with 19 at risk. It also records 1 death. These are registry entries, not an adjusted comparison between treatment groups. The top serious terms are listed with percentages, but the record does not provide enough detail to interpret overlap between terms or to connect each term to a unique person. The listed serious-event terms should therefore be read as event reporting, not as a complete account of the regimen’s safety.
Planning illustrations based on an observed effect are not observed trial results and are not a measure of power achieved in this study. The registry describes general assumptions for such illustrations, but the numerical planning outputs are not included in the record. No conclusion about the primary endpoint, comparative benefit, or the adequacy of study size can be drawn from those assumptions alone. The most defensible reading is limited to the design, endpoint definition, and event entries that the registry actually records.
9. Statistical Methods Explained
Statistical concepts help clarify what the registry’s endpoint and analysis descriptions would mean, but they cannot fill in estimates that are absent. In this record, the primary outcome is time to progression or death, expressed as a median, and the record does not provide the median or its confidence interval.
What does a median time-to-event estimate describe?
A time-to-event outcome records both the time until an event and whether the event was observed during follow-up. The median is a summary of the event-time distribution, not an average duration for every participant and not a guarantee about an individual outcome. In studies with censoring, some participants have not had the event by their last known follow-up. Survival-analysis methods can retain information from those participants up to that point, rather than treating the last contact as an event.
Progression-free survival commonly treats progression or death as an event, in line with this registry’s definition. How progression is determined, how deaths are verified, and how incomplete follow-up is handled can affect the result. The record states that evaluation was every 12 weeks during study treatment but does not report detailed assessment procedures or censoring rules. It also does not report the number of events or the number included in the endpoint analysis.
How should a confidence interval be read?
A confidence interval expresses statistical uncertainty around an estimate under the assumptions of the method used. For a median time-to-event estimate, the interval helps show how precisely the median is estimated. A narrower interval generally indicates greater precision than a wider interval, though precision alone does not establish clinical importance or remove bias. The registry specifies a 95% confidence interval for the primary endpoint but does not report its limits. Therefore, neither the estimated median nor its precision can be assessed from this record.
What can a single-group design establish?
A single-group study describes outcomes observed among participants who received the study regimen. Because the registry lists no concurrent control group, there is no within-study contrast that separates the regimen’s association with outcomes from other influences. A before-and-after interpretation or comparison with historical experience would require additional information and assumptions; neither such analysis nor its results are reported in the registry record. The study’s stated hypothesis about comparison with sequential treatment should not be treated as if that comparison was performed.
Masking is listed as none. In general, lack of masking can matter when outcome assessment involves judgment, because expectations may influence assessment or reporting. For a time-to-event endpoint, this concern can depend on how progression is assessed and whether assessments are standardized. The registry says evaluation occurred every 12 weeks, but does not report assessment procedures or independent review. The extent to which lack of masking affected outcome ascertainment is therefore not reported in the registry record.
What do hazard ratios and event counts mean?
A hazard ratio is a relative comparison of event rates over time between groups, under the model used to estimate it. It is not the same as a ratio of medians, and it does not directly state the length of time an individual will remain progression-free. A hazard ratio requires an appropriate comparison; this registry describes a single group and includes no sponsor-reported hazard ratio values. The record’s general note says only sponsor-reported hazard ratios are used, but no such numerical estimates are recorded.
For time-to-event planning, event totals can be more informative than participant counts because information accrues as events occur. The registry’s planning note describes a Schoenfeld approach for total events in time-to-event illustrations. It also describes a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect specified as a fraction of the observed effect. These are planning assumptions, not evidence that the study had a particular achieved power or that a two-arm comparison occurred. The numerical planning outputs and the observed effect used for them are not reported in the registry record.
More generally, a sample-size illustration answers a conditional question: under stated assumptions, what scale of study might be needed to detect a chosen effect? It does not show the probability that a completed study has found a real effect, and it cannot substitute for a prespecified design rationale. The registry does not report its own sample-size justification, so the planning illustrations should not be read as the trial’s original design calculation.
Why does over-dispersion matter in general?
For count outcomes, a basic statistical model may assume that the variability is tied closely to the mean. If observed variation is greater than that assumption allows, the data are over-dispersed. Ignoring over-dispersion can make uncertainty estimates too small and statistical evidence appear stronger than warranted. This is a general issue in count-data analysis; the registry does not report a count model or an over-dispersion analysis for this trial.
Overall, the record identifies an endpoint and some design fields but does not describe the statistical analysis in enough detail to assess model choices, covariate adjustment, censoring, or the handling of multiple outcomes. Those elements are not reported in the registry record and should remain unknown rather than be reconstructed from general practice.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary endpoint of progression-free survival and two secondary outcomes: clinical response rate measured by best response achieved, and characterization of the toxicity profile. It records 3 outcome measures but lists 0 analyses. The record does not report numerical results for the primary or secondary outcomes.
When a study evaluates several outcomes or performs several statistical tests, the chance of at least one apparently unusual result can increase if no adjustment or prioritization is used. This is the general issue of multiplicity. A prespecified hierarchy, adjustment method, or clear distinction between confirmatory and exploratory outcomes can help readers understand how multiple results were interpreted. The registry record does not report a multiplicity strategy, the number of statistical tests, or a hierarchy among the endpoints beyond identifying progression-free survival as primary.
Missing-data handling is also not described. For a time-to-event endpoint, incomplete observation can lead to censoring; how and when censoring occurs, and whether assumptions behind the method are plausible, matter to interpretation. The record does not report censoring rules, loss to follow-up, missing outcome data, or sensitivity analyses. It is not possible to determine from the record how incomplete assessments were handled.
The analysis population is not specified. The registry does not state whether the primary analysis used all enrolled participants, only participants who started treatment, or another defined group. It also does not report exclusions from analysis or protocol deviations. The enrollment field records 37, but enrollment alone does not establish the number included in any particular outcome analysis.
These omissions do not prove that no statistical plan or analysis existed; they mean that the relevant details are not reported in the registry record. The reported design and endpoint definition should be distinguished from the unreported analysis decisions needed to judge the reliability and scope of the results.
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 |
|---|---|---|---|
| Alternating Sunitinib and Temsirolimus | 4 | 19 | 21.1% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Alternating Sunitinib and Temsirolimus | 1 of 19 |
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 |
|---|---|
| Alternating Sunitinib and Temsirolimus | 19 of 19 |
Most frequent serious adverse event terms
| Event term | Organ system | Alternating Sunitinib and Temsirolimus |
|---|---|---|
| Dysarthia | Nervous system disorders | 1 of 19 |
| Ischemia Cerebrovascular (stroke) | Vascular disorders | 1 of 19 |
| Muscle Weakness- right sided | Musculoskeletal and connective tissue disorders | 1 of 19 |
| Atrial Fibrillation | Cardiac disorders | 1 of 19 |
| Urosepsis | Renal and urinary disorders | 1 of 19 |
| Pneumonia (w/ sepsis) | Respiratory, thoracic and mediastinal disorders | 1 of 19 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Alternating Sunitinib and Temsirolimus |
|---|---|---|
| Fatigue | General disorders | 14 of 19 |
| Mucositis oral | Gastrointestinal disorders | 10 of 19 |
| Anemia | Investigations | 9 of 19 |
| Diarrhea | Gastrointestinal disorders | 9 of 19 |
| Creatinine increased | Investigations | 8 of 19 |
| Palmar-plantar erythrodysesthesia syndrome | Skin and subcutaneous tissue disorders | 8 of 19 |
| Dysgeusia | Nervous system disorders | 7 of 19 |
| Allergic reaction (rash) | Immune system disorders | 6 of 19 |
| Headache | Nervous system disorders | 6 of 19 |
| Platelet count decreased | Investigations | 6 of 19 |
Serious events with the highest arm-level rate: dysarthia (5.3%), ischemia cerebrovascular (stroke) (5.3%), muscle weakness- right sided (5.3%), atrial fibrillation (5.3%), urosepsis (5.3%).
The adverse-event record lists one group, Alternating Sunitinib and Temsirolimus. For serious adverse events, it records 19 at risk and 4 affected. For other adverse events, it records 19 at risk and 19 affected. The record lists 1 death. These are counts as entered in the registry; they should not be treated as a comparison between treatments.
The listed top serious-event terms are Dysarthia, Ischemia Cerebrovascular (stroke), Muscle Weakness- right sided, Atrial Fibrillation, Urosepsis, and Pneumonia (w/ sepsis). Each is reported as 5.3%. The registry does not explain whether a participant could contribute more than one term, give event-level timing, or report severity grades and attribution in this listing. A percentage attached to a term is not sufficient to establish how many unique participants experienced any serious event, nor whether the listed terms cover all reported serious events.
Adverse-event entries describe events recorded during the study; they do not, by themselves, establish that treatment caused an event. The record does not provide a control group for comparative safety interpretation. It also does not report a full account of duration, recurrence, treatment discontinuation, or how events were judged related to the regimen. The counts and terms are therefore best read as registry safety entries with limited context, not as a complete comparative safety analysis.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a completed Phase II, single-group study evaluated alternating sunitinib and temsirolimus in metastatic renal cell carcinoma. It identifies progression-free survival as the primary endpoint and describes how that endpoint was defined. It also records enrollment, the treatment schedule, secondary outcomes, and adverse-event entries.
The record does not report the observed progression-free survival median or its 95% confidence interval. It does not report a hazard ratio, a numerical response-rate result, or a comparative analysis. Consequently, the record cannot establish whether the stated hypothesis about progression-free survival was supported. The hypothesis is not an observed finding, and the single-group design does not supply a concurrent sequential-treatment comparison.
The safety entries provide limited descriptive information: serious events affected 4 participants in the group, with 19 at risk; other events affected 19, with 19 at risk; and 1 death is recorded. The top serious-event terms are listed with percentages. These entries do not resolve event attribution or provide a treatment comparison, and they should not be expanded into claims about overall safety.
Statistical precision and clinical importance are different questions. A precise estimate may still be of limited clinical importance, and an imprecise estimate may leave a wide range of plausible outcomes. Here, the endpoint’s estimate and confidence interval are not reported, so neither precision nor the magnitude of the observed outcome can be assessed. The record’s planning assumptions do not supply the missing results and do not indicate the power achieved.
The appropriate conclusion is narrow: the registry describes a study design and planned outcome framework, but does not give the primary numerical result needed to evaluate the endpoint. No claim about benefit, lack of benefit, or comparative safety follows from the information recorded.
13. Why This Trial Matters Statistically
This trial is statistically notable because its stated hypothesis refers to a comparison with sequential therapy, while the registry describes a single-group study with allocation listed as NA. That distinction matters: a hypothesis can motivate a study without the study design directly testing every comparison named in the rationale. Without a concurrent comparator, the registry can describe outcomes in the treated group but cannot isolate a comparative treatment effect.
The primary endpoint is time-to-event, a design choice that can use information from participants with differing follow-up durations, provided censoring and event ascertainment are handled appropriately. The endpoint combines progression and death and is reported as a median with a confidence interval. Those choices make the definition and analysis plan important. The record provides the endpoint definition and evaluation schedule, but not the estimate, uncertainty limits, event counts, or details of censoring and analysis.
The record also illustrates why registration details and outcome reporting serve different purposes. Design fields identify the intended study structure, while results are needed to judge what was observed. Here, the record marks the study completed, but lists 0 analyses and provides no numerical primary endpoint result. Completion status alone does not tell readers what outcomes occurred or how precisely they were estimated.
Finally, planning assumptions should not be mistaken for evidence. An illustration based on a stated significance level, allocation assumption, and target fraction of an observed effect is conditional, and the record does not report numerical planning outputs or the trial’s own sample-size justification. Such planning cannot replace an outcome estimate or establish that the study was adequately powered. The case underscores the value of reporting the design, analysis population, event handling, estimates, and uncertainty together.
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.
Interpretation is constrained first by the absence of the primary endpoint result. The registry identifies progression-free survival as the primary outcome but does not report its median or 95% confidence interval. The listed outcome measures have no accompanying numerical results in the record, and the analyses field lists 0 analyses.
The single-group design limits comparative interpretation. Although the stated study rationale refers to sequential treatment, the registry does not describe a concurrent comparison group. Allocation is listed as NA, and the design is identified as single group. The record therefore does not support a direct estimate of how alternating treatment compares with sequential therapy or another regimen.
The endpoint timeframe refers to the date of randomization, despite the single-group model and allocation entry. The record does not clarify how that starting point was defined or applied. It notes evaluation every 12 weeks during study treatment, but does not describe progression review procedures, censoring rules, or follow-up after treatment. The analysis population, missing-data handling, and statistical model are also not reported in the registry record.
The safety information is limited to registry counts and listed terms. It does not report event-level detail, attribution, severity grading, or a comparative safety analysis. The record lists 1 death but does not provide context that would establish cause or relationship to treatment. The top serious-event terms are shown as percentages, but overlap between terms is not explained.
Finally, the record does not report its own sample-size justification or a multiplicity plan. Planning assumptions described for illustrations are not substitutes for either. These limitations mean that the record can establish the study’s general design and endpoint definitions, but not the magnitude, precision, or comparative meaning of its outcomes.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Phase II Study of Alternating Sunitinib and Temsirolimus — NCT01517243.
- Registry record last updated 2023-09-21; status last verified 2023-07.
- Results first posted 2023-09-21.
- 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 identifies NCT01517243 as a completed Phase II study of alternating sunitinib and temsirolimus for metastatic renal cell carcinoma. The single-group study lists enrollment of 37, no masking, and Dartmouth-Hitchcock Medical Center as sponsor. Its recorded start date is 2010-09-22, and its primary completion date is 2018-08-20.
Progression-free survival is the primary endpoint, defined from the date of randomization to documented progression or death from any cause, with evaluation every 12 weeks during study treatment. The registry does not report the primary estimate or confidence interval. Clinical response rate and toxicity characterization are listed as secondary outcomes, but their results are not reported.
The adverse-event entries include serious and other event counts, 1 death, and six top serious-event terms, each listed as 5.3%. The record describes no concurrent comparator and lists 0 analyses. Important statistical details, including the analysis population, missing-data handling, and multiplicity plan, are not reported in the registry record.