Rollover Protocol for Prior SU011248 Protocols: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Rollover Protocol for Prior SU011248 Protocols (NCT00798889), a na na study. The primary outcome is Duration of Treatment.

2. Clinical Question

In practical terms, the outcome concerns how long participants remained on study treatment, rather than a randomized comparison of treatment strategies. Duration on treatment can describe treatment exposure in this enrolled population. It does not, on its own, establish why treatment ended or whether participants experienced a clinical benefit. The registry record does not give reasons for stopping treatment or details about how the duration was calculated. It also does not report a comparative estimate, such as a difference between groups, because no comparator group is listed. The result available in the registry record is therefore limited: the outcome definition and summary measure are specified, but the numerical result needed to describe the observed distribution is not reported in the registry record.

3. Trial Design

This is a completed, single-group rollover protocol sponsored by Pfizer. Its title identifies it as a protocol for patients continuing from prior SU011248 protocols. The brief summary states that its objective was to provide SU011248 treatment to patients who had participated in a SU011248 protocol and were eligible to enter this protocol. The intervention is listed as Sunitinib.

The registry records enrollment of 314 and the study status as completed. The recorded start is 2004-03, and primary completion is 2011-12. Phase is listed as NA. Allocation is listed as NA, masking as NONE, and the model as SINGLE GROUP. Those entries describe a record without a randomized allocation or a listed control arm; they should not be read as evidence of a treatment comparison. The registry record does not report additional details about how eligibility was assessed, which prior protocols participants came from, or how treatment decisions were made.

The primary outcome population is defined as the intent-to-treat population, consisting of participants enrolled in the study who received at least 1 dose of study medication. This definition is tied to receipt of study treatment, but the record does not state how many enrolled participants met that criterion. The serious and other adverse-event figures use 311 participants at risk; the registry record does not explain how that safety denominator relates to the enrollment count.

Because the protocol enrolled people already participating in prior protocols and had no listed comparator, its design addresses treatment continuation within this selected group, not a controlled estimate of what would have happened under another treatment or no treatment. The registry record does not report randomization, masking procedures beyond the NONE entry, or a comparative statistical analysis.

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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: The intent-to-treat population included all participants who were enrolled in the study and received at least 1 dose of study medication.

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.

6. Primary Endpoint

The registry lists the primary outcome as Duration of Treatment, measured over Baseline up to Day 28 after last dose of study treatment.

The primary outcome is duration of treatment, with days as the unit. The registry specifies a median and full range, and gives the timeframe as baseline up to Day 28 after the last dose of study treatment. The numerical median and range are not reported in the registry record. The record also does not describe the precise calculation rules, including how the start and end of the duration were assigned.

In general, treatment duration describes time on treatment. A median is a measure of the center of a distribution: it identifies the point at which the distribution is divided into lower and upper portions. A full range describes the observed extent from the lowest to the highest value. These summaries can communicate both a typical duration and the spread of observed durations, although the range can be sensitive to unusually low or high observations. These are general interpretations of the stated summary measures, not additional findings from this trial.

Duration of treatment is useful for describing treatment exposure and persistence in a cohort. It is not interchangeable with a measure of tumor response, survival, or comparative effectiveness. A longer or shorter observed duration could have different explanations, and the registry record does not report reasons for stopping treatment. Without the actual median and range, readers cannot characterize the observed duration distribution from this outcome entry.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes a single-group study and identifies duration of treatment as its primary outcome. Its summary measure is the median with a full range, expressed in days. The record does not report the median or range values, nor does it contain an analysis entry for the outcome. The methodological explanations below describe general statistical concepts and do not add findings to the registry record.

What does a median describe?

In general, the median is a measure of the center of a set of observations. It is less influenced by extreme values than an arithmetic mean. For a time measure such as treatment duration, the median can give a concise description of the middle of the observed distribution. It does not show the full shape of that distribution, and it does not explain why individual durations differ. The registry record specifies the median but does not report its value, so no numerical interpretation of the center is possible.

What does the full range add?

In general, the range reports the smallest and largest observed values. It can make the spread of durations visible alongside a median, but it gives little information about how observations are distributed between those endpoints. It can also be influenced by unusual observations. The registry record states that a full range was the dispersion measure, but neither endpoint of the range is reported. No statements about the shortest or longest treatment duration can therefore be made from the registry entry.

How should the outcome timeframe be read?

The stated timeframe is baseline up to Day 28 after the last dose of study treatment. This identifies the registry's timeframe language; it does not, by itself, provide a complete operational definition for deriving each participant's duration. The record does not describe the date rules, handling of interruptions, or any other calculation details. In general, time-based outcomes require clear definitions of when measurement begins and ends, as well as rules for observations that are incomplete. Those details are not reported in the registry record.

Why does study design affect interpretation?

A single-group design describes outcomes within a group receiving the listed intervention. Without a comparator, it cannot separate the observed course from what might have occurred under a different treatment or under no study treatment. Random allocation, when used in a comparative trial, is a method for balancing measured and unmeasured characteristics across groups on average. Allocation is listed as NA in this record, and no control group is described. Thus, the interpretation should remain descriptive rather than causal or comparative.

Masking is recorded as NONE. In general, masking can reduce the influence of knowledge about assigned treatment on assessments or other trial procedures. The registry entry does not describe the implications of the masking entry for outcome assessment or event reporting. It is therefore appropriate to report the recorded status without inferring additional safeguards or procedures.

What do planning calculations mean?

The registry materials describe a planning illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The stated purpose is to illustrate detection of a fraction of that observed effect. These assumptions describe an illustration, not the original design of this single-group rollover protocol. A planning sample size is not observed power, and it does not show that the study had a particular probability of detecting an effect. No numerical planning results are reported in the registry record considered for this interpretation.

In general, sample-size planning depends on the effect being targeted, variability or event behavior, the chosen significance level, allocation, and model assumptions. Changing an assumption can change a planning estimate. For a time-to-event outcome, event counts can be central to planning; other outcomes may be expressed in participants per arm. These general principles do not turn the planning illustration into evidence of a treatment effect or into a retrospective assessment of the protocol's design. The registry record does not report a sample-size justification for the original study.

What can be concluded from the listed analyses?

The registry lists zero analyses. This indicates that no analysis results are entered in the registry record for the listed outcome. It does not establish whether investigators conducted analyses elsewhere or whether analyses were planned but not posted. No hazard ratio, confidence interval, test result, or adjusted estimate is reported. In general, a confidence interval describes the range of estimates compatible with a statistical model and its assumptions; its width indicates precision, and its position relative to a null value can inform a comparison. Since no such estimate is reported for this outcome, precision cannot be assessed statistically from the registry entry.

For the same reason, no p-value or formal test of a treatment contrast can be interpreted. There is no listed comparator and no numerical primary outcome result. General statistical methods cannot compensate for these missing results: they explain how an analysis might be interpreted when reported, but they do not generate an estimate from absent data. The appropriate reading is that the registry specifies a descriptive primary outcome and its intended summary, while leaving the numerical result and analysis unreported.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists one outcome measure and zero analyses. It identifies duration of treatment as the primary outcome, but does not report a numerical result or an analysis entry. The record does not report a multiplicity strategy, such as an adjustment for multiple outcomes or repeated testing. With no analysis results in the registry entry, there is no set of reported tests from which to assess whether multiplicity affected interpretation. The absence of an analysis entry should not be taken as proof that no analysis was performed outside the registry record.

The primary outcome population is defined as enrolled participants who received at least 1 dose of study medication. This definition makes receipt of treatment part of the stated analysis population. However, the registry record does not report the number meeting this definition, the number with outcome data, or how missing outcome observations were handled. It does not describe imputation, censoring, exclusions, or sensitivity analyses. These methods are not reported in the registry record.

The safety entry uses 311 participants at risk for serious adverse events and 311 for other adverse events, while enrollment is recorded as 314. The registry record does not explain the difference. The available information does not establish whether it reflects eligibility, exposure, follow-up, or another reason; no explanation should be inferred. The record also does not state how participants with incomplete safety follow-up were handled.

In general, clear reporting of analysis populations and missing-data methods helps readers understand which observations contribute to a result and how incomplete information could affect it. Here, the population definition is stated, but counts and handling details are not reported in the registry record. Accordingly, the registered outcome cannot be assessed for the impact of missing data or exclusions.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: abdominal pain (4.5%), dehydration (4.2%), vomiting (3.5%), nausea (3.2%), disease progression (2.9%).

The registry reports serious adverse events affecting 118 of 311 participants at risk, and other adverse events affecting 299 of 311 participants at risk. These are separate registry categories and should not be combined or treated as a comparison between groups. The study has a single listed group, and the record does not provide a control group against which to judge event frequency.

The listed serious adverse-event terms are abdominal pain, 4.5%; dehydration, 4.2%; vomiting, 3.5%; nausea, 3.2%; disease progression, 2.9%; and dyspnoea, 2.6%. These are selected terms with associated percentages, not a complete account of all events. The registry record does not report the definitions used for these terms or further details about severity, timing, recurrence, or attribution. The percentages should be read as reported, without assuming a different denominator or deriving additional counts.

Deaths are not reported in the registry record. The safety figures describe events in the registered study group; they do not, by themselves, show that the study drug caused an event. A causal assessment would require context and methods that are not reported in the registry record. The record also does not explain why the safety population at risk differs from the enrollment entry.

12. What the Result Does, and Does Not, Mean

The result can establish only what the registry record reports: a completed, single-group rollover protocol intended to provide SU011248 treatment to eligible participants from prior SU011248 protocols; a primary outcome of duration of treatment; and selected safety figures. The primary outcome was to be summarized by a median and full range in days over the stated timeframe, but the median and range themselves are not reported in the registry record.

That missing numerical result means the observed duration cannot be characterized. It is not possible to state from this record whether treatment duration was relatively short or long, how much it varied, or how it differed across participant subgroups. No subgroup results are reported. The registry also does not give reasons for treatment discontinuation, so duration cannot be interpreted as a direct measure of response, tolerability, or any single cause.

The study design limits what the posted information can establish. The protocol is single-group, with allocation listed as NA and no comparator group. It therefore does not provide a randomized contrast or an estimate of what would have happened under another treatment. The record does not report a hazard ratio, confidence interval, p-value, or other comparative estimate. No conclusion about a treatment effect or its precision follows from the information reported.

The serious and other adverse-event figures are descriptive entries for the listed group. Without a comparison group and additional event details, they should not be treated as evidence that treatment caused or prevented events. The registry does not report deaths in its safety entry, and this absence of a reported value is not evidence that no deaths occurred.

General statistical interpretation distinguishes observed description from causal inference. A single-group outcome can describe the recorded experience of the enrolled cohort, while controlled comparisons are needed to estimate relative outcomes under alternative assignments. Even a complete duration summary would not on its own establish a comparative treatment effect. In this case, the numerical duration summary is also absent, so the interpretation is narrower still: the registry specifies the intended outcome and population, but does not report the primary outcome result.

13. Why This Trial Matters Statistically

This trial is statistically notable for the distinction between a rollover protocol and a comparative treatment study. Its stated purpose was to make SU011248 treatment available to eligible participants continuing from prior protocols. The registry records a single-group model, no allocation, and no comparator. That design is suited to recording experience in the enrolled group, but it does not create a randomized estimate of how outcomes differ under alternative treatment choices.

The primary outcome also illustrates the difference between defining an endpoint and reporting its result. The record specifies duration of treatment, days, a median, a full range, and a timeframe. Those details tell readers what the intended summary concerns. Yet the numerical median and range are not reported in the registry record, so the outcome cannot be interpreted quantitatively. A well-described endpoint is not a substitute for a reported estimate.

The record also makes clear why populations and denominators should be read separately. Enrollment is 314, the primary population is defined by enrollment and receipt of at least 1 dose, and safety figures use 311 at risk. The registry does not reconcile these entries. Keeping them distinct avoids imposing an explanation not in the record.

Finally, the entry provides an opportunity to distinguish descriptive safety reporting from causal conclusions. Event counts and listed percentages characterize what was recorded in the study group, but they do not establish attribution or comparative risk. These points are broadly relevant when interpreting registry records: design, endpoint definition, population, denominator, and completeness of results all shape the conclusions that can responsibly be drawn.

14. Limitations

• The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
• Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.

The most consequential limitation is that the registry record does not report the primary outcome's median or full range. Although duration of treatment is defined as the primary outcome, the numerical result is unavailable, preventing a description of its observed center or spread. The record also lists zero analyses and does not provide a test result or an uncertainty interval.

The single-group design provides no listed control or randomized comparison. It cannot separate the observed experience of participants in this rollover protocol from what might have occurred under another treatment strategy. Eligibility was based on participation in a prior SU011248 protocol and eligibility to enter the rollover protocol, but the registry does not give more detail about the selection process or characteristics of people who did not enter. The enrolled group should therefore be understood in that specific context.

The registry enrollment is 314, whereas the safety figures use 311 participants at risk. The record does not explain this difference. It also does not report the number included in the primary outcome population, the number with outcome data, or methods for handling missing observations. The operational calculation rules for treatment duration and reasons for treatment ending are not reported in the registry record.

For safety, selected serious adverse-event terms and percentages are listed, but the entry does not give further details on event timing, duration, attribution, or follow-up. Deaths are not reported in the registry record. These omissions limit interpretation of the safety summaries and do not support causal conclusions. Taken together, the record supports a limited descriptive account of the protocol and selected safety entries, but not a quantitative interpretation of the primary outcome or a comparative treatment effect.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Rollover Protocol for Prior SU011248 Protocols — NCT00798889.
• Registry record last updated 2013-01-21; status last verified 2012-12.
• Results first posted 2013-01-21.
• 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.

18. Record Summary

NCT00798889 is a completed, Pfizer-sponsored, single-group rollover protocol for patients continuing from prior SU011248 protocols. The registry lists Sunitinib as the intervention, enrollment of 314, a start of 2004-03, and primary completion in 2011-12. Phase and allocation are recorded as NA; masking is recorded as NONE.

The primary outcome is duration of treatment in days, summarized by a median and full range, over the stated timeframe from baseline up to Day 28 after the last dose of study treatment. The numerical median and range are not reported in the registry record. The primary population is defined as enrolled participants who received at least 1 dose of study medication.

Safety entries report serious adverse events affecting 118 of 311 participants at risk and other adverse events affecting 299 of 311 at risk. Selected serious adverse-event terms and percentages are listed; deaths are not reported in the registry record. The entry lists one outcome measure and zero analyses, and it does not provide a comparator or a reported comparative estimate.