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 Sunitinib in Treating Patients With Metastatic Germ Cell Tumors That Have Relapsed or Not Responded to Treatment (NCT00453310), a na phase 2 study. The primary outcome is Confirmed Objective Response Rate (Complete and Partial Response) as Measured by RECIST Criteria After 2 Courses of Treatment.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00453310 |
| Phase | Phase 2 |
| Condition | Extragonadal Germ Cell Tumor, Ovarian Cancer, Teratoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 10 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Memorial Sloan Kettering Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients with metastatic germ cell tumors that had relapsed or had not responded to treatment. The registry lists extragonadal germ cell tumor, ovarian cancer, teratoma, and testicular germ cell tumor among the conditions. It does not report further eligibility criteria or describe the characteristics of enrolled participants.
Intervention
The intervention was sunitinib malate, given as a continuous daily oral dose of 37.5 mg for 6 weeks. The registry describes a therapy cycle as 42 days, or 6 weeks. It does not report whether participants could receive additional cycles or how treatment was modified if an adverse event occurred.
Comparator
This was a single-group study, with no randomized comparison arm. Allocation is recorded as not applicable, and masking is recorded as none. The study therefore does not provide a like-for-like control group against which to compare response or adverse events.
Primary question
The primary endpoint was the number of participants with a confirmed complete or partial objective response by RECIST criteria after 2 courses of treatment. The registry also gives a timeframe of 2 years. It does not report the observed response count, response rate, or an analysis of this endpoint, so the clinical question is stated but its answer cannot be read from the registry record.
3. Trial Design
This was a phase II, single-group study sponsored by Memorial Sloan Kettering Cancer Center. The registry lists an enrollment of 10 and marks the study as completed. Its recorded start was 2007-03, and its primary completion was 2008-11. These dates describe the registry record; they do not establish when individual participants entered or finished treatment.
Participants received sunitinib malate. The recorded regimen was a continuous daily oral dose of 37.5 mg for 6 weeks, with a therapy cycle described as 42 days, or 6 weeks. The record does not give participant-level treatment duration, adherence, dose reductions, discontinuations, or the number of cycles actually received.
The design has no randomized comparator: allocation is marked not applicable and the study model is single group. Masking is recorded as none. Accordingly, the study can describe outcomes among participants treated under the study regimen, but it cannot estimate a randomized treatment contrast. Changes in disease status cannot be separated from other influences using a concurrent control group in this design.
The primary outcome is described as confirmed objective response, combining complete and partial response as measured by RECIST criteria after 2 courses of treatment. The endpoint record separately lists a timeframe of 2 years. How that timeframe relates to the assessment after 2 courses is not explained in the registry record. Although one outcome measure is listed, no analysis entries are recorded, and the primary response result is not reported in the registry record.
The registry also includes 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. That illustration is not the design of this single-group trial: its equal-allocation assumption describes a hypothetical two-arm planning calculation, not actual assignment in this study. The resulting sample-size values are not reported in the registry record. Such planning values are not observed power.
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 Malate |
|---|---|
| STARTED | 10 |
| COMPLETED | 0 |
| NOT COMPLETED | 10 |
Reasons for not completing this period:
| Reason | Sunitinib Malate |
|---|---|
| Progression of Disease | 10 |
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 Malate |
|---|---|---|
| Participants analysed | 10 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 10 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 10 |
6. Primary Endpoint
The registry lists the primary outcome as Confirmed Objective Response Rate (Complete and Partial Response) as Measured by RECIST Criteria After 2 Courses of Treatment, measured over 2 years.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants with a confirmed complete or partial objective response under RECIST criteria. It combines two response categories into a binary outcome: a participant either meets the endpoint definition or does not. This makes the measure a direct summary of whether tumor measurements meet the specified response criteria, rather than a measure of how long response lasts or how long participants live.
Response criteria provide a structured way to classify changes in measurable disease. In general, standardized criteria can make assessment more consistent than an informal judgment, although the registry does not report details of assessment procedures, who reviewed scans, or whether response was independently confirmed. The endpoint title says response was to be assessed after 2 courses of treatment; the separately recorded timeframe is 2 years. The relationship between these timing descriptions is not reported in the registry record.
A response count can be expressed as a proportion of the assessed participants, but that interpretation depends on knowing who was assessed and how missing assessments were handled. Neither the response count nor the analysis population is reported in the registry record. The endpoint definition is therefore available, but the observed response result and its precision cannot be evaluated from the posted results.
This endpoint is not itself a comparative effect. In a single-group study, an observed response proportion describes outcomes in the treated group; without a control group, it does not by itself establish what would have happened under another treatment or without the study treatment.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies the study question and the endpoint but does not report the primary response outcome. It lists an enrollment of 10, one primary outcome measure, and no analysis entries. The number of participants who had a confirmed complete or partial response, the response rate, and any uncertainty interval are not reported in the registry record. It is therefore not possible to quantify the observed response or how precisely it was estimated from this record.
The design is single group, with no randomized comparison arm. This matters for interpretation even if an outcome result were available. A response observed after treatment would describe what happened in the enrolled group; it would not be a randomized estimate of the difference between sunitinib and another treatment. Differences in patient characteristics, disease course, prior treatment, and other influences could not be balanced through random assignment in this study design. The registry does not describe these factors in detail.
The response endpoint combines complete and partial response under RECIST criteria, and its title refers to assessment after 2 courses. The timeframe field says 2 years. Because the record does not explain how these timing descriptions fit together, readers should not assume a particular assessment schedule beyond what is explicitly stated. The registry also does not report the number assessed, the handling of unevaluable participants, or whether any response was confirmed.
The adverse-event record is descriptive. For the Sunitinib Malate group, it lists 1 participant with a serious adverse event among 10 at risk and 8 participants with other adverse events among 10 at risk. The listed top serious term is “Hemorrhage, Abdomen NOS” at 10.0%. These fields do not establish that an event was caused by treatment. The deaths field is not reported in the registry record.
The sample-size planning illustration uses assumptions for a hypothetical two-arm calculation, including equal allocation between arms, despite the actual study being single group. The assumptions are planning parameters rather than evidence that the study had a particular power. The registry does not report the illustration's resulting sample-size values, and the primary response estimate needed to evaluate that illustration is not available in the record.
In general, a confidence interval would express uncertainty around an estimated response proportion, with wider intervals indicating less precision. No interval is reported. In the absence of the endpoint count and its analysis, neither the magnitude nor the precision of the primary result can be described. The record supports a description of the study's design and stated outcome, not a conclusion about the size or reliability of a response result.
9. Statistical Methods Explained
This study's outcome is binary at the participant level: the primary definition classifies participants according to whether they had a confirmed complete or partial objective response. In general, a binary outcome can be summarized by reporting the number meeting the definition and the proportion among an explicitly defined analysis group. The registry gives neither the primary response count nor the denominator used for an analysis. Enrollment alone does not show how many participants were assessed for this endpoint.
What does the response definition capture?
RECIST criteria are a structured framework for classifying objective tumor response. The endpoint title specifies complete and partial response and says assessment occurred after 2 courses of treatment. A response endpoint of this kind focuses on whether a participant crosses a defined category threshold. It does not, by itself, describe the duration of response, time to progression, survival, or changes in symptoms. Those are distinct outcomes and are not listed as the primary endpoint in this record.
The registry's timeframe is 2 years, while the endpoint title refers to assessment after 2 courses. The record does not explain whether the timeframe refers to follow-up, the period over which responses were collected, or another timing convention. It also does not report scan schedules, confirmation procedures beyond the word “confirmed,” or the personnel responsible for response assessment. Those details should not be inferred from the endpoint title.
How does a single-group design affect estimation?
In a single-group study, a response proportion describes the experience of the enrolled group under the study regimen. It is not a treatment-versus-control contrast. A randomized comparison can support a different question because random assignment is intended to make treatment groups comparable on average. This trial has no randomized comparison arm, so that design-based protection is absent. A numerical response result, if reported, would need to be interpreted as a within-group description rather than a randomized estimate of treatment effect.
General statistical inference for a binary outcome uses the observed count and the denominator to estimate an underlying response probability. An uncertainty interval would show the range of values reasonably compatible with the data under the chosen method and assumptions. The interval is important because a point estimate alone does not show how much it could vary due to sampling. No response estimate, interval, or method for constructing an interval is reported in the registry record.
What does the planning illustration mean?
The registry describes a sample-size planning illustration based on the observed effect. It specifies a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is described as a stated fraction of the observed effect. The resulting sample-size values and the fractions used are not reported in the registry record.
These assumptions concern a hypothetical two-arm planning exercise, not the actual single-group study. Equal allocation between two arms does not describe the enrollment structure in this trial. A planning calculation asks how many participants might be needed under stated assumptions for a future comparison. It does not show the probability that this completed study detected an effect, and it is not observed power. Without the observed primary endpoint result and the actual calculation values, the illustration cannot be evaluated as a numerical planning result.
In general, a normal approximation simplifies calculations by using an approximate distribution for an estimator. Whether an approximation is adequate depends on features such as the event frequency and sample size. The registry does not report the response frequency, so the adequacy of that approximation for the planning illustration cannot be assessed from the posted results. This general point does not imply that the study used a particular analysis method.
What analysis details are absent?
The registry records no analysis entries. It does not report an inferential model, an uncertainty interval, a hypothesis test for the primary endpoint, or adjustments for participant characteristics. It also does not report the intended analysis population, missing-outcome handling, or how participants who did not complete treatment would be classified. These omissions prevent a reconstruction of the primary analysis and its assumptions.
For safety outcomes, the record provides counts of participants at risk and affected, plus a listed serious-event term and percentage. These are descriptive summaries. The record does not report a method for comparing adverse-event frequencies, a treatment attribution assessment, or a detailed time-at-risk analysis. In general, adverse-event counts are most informative when interpreted with clear definitions, observation periods, and denominators; those details are not fully specified in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one outcome measure, identified as the confirmed objective response endpoint, and records no analysis entries. It does not report whether any additional outcomes were assessed but not entered, or whether multiple statistical tests were planned. The analysis plan, including any approach to multiplicity, is not reported in the registry record.
In general, multiplicity arises when several outcomes, comparisons, or repeated tests are considered. Without an analysis plan, readers cannot determine whether any adjustment was intended or whether the listed endpoint was the only outcome used for formal inference. No hypothesis-test result is available in the registry record, so there is no reported multiplicity adjustment to interpret.
The registry does not report the number of participants with an evaluable response assessment, how missing assessments were handled, or whether participants who stopped treatment remained in the primary analysis. The analysis population is not reported in the registry record. Enrollment of 10 is not enough to establish the endpoint denominator, because participation in treatment and availability of an assessment are not described participant by participant.
In general, missing outcomes can affect a response estimate when the probability of being assessed is related to disease status or treatment experience. Different missing-data rules can lead to different estimates. No missing-data rule or sensitivity analysis is reported in the registry record, so the influence of missing assessments cannot be evaluated.
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 Malate | 1 | 10 | 10.0% |
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 Malate | 8 of 10 |
Most frequent serious adverse event terms
| Event term | Organ system | Sunitinib Malate |
|---|---|---|
| Hemorrhage, Abdomen NOS | Gastrointestinal disorders | 1 of 10 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Sunitinib Malate |
|---|---|---|
| Leukocytes (total WBC) | Blood and lymphatic system disorders | 4 of 10 |
| ALT, SGPT | Blood and lymphatic system disorders | 2 of 10 |
| Lymphopenia | Blood and lymphatic system disorders | 2 of 10 |
| Mucositis (func/sympt)- Oral cavity | General disorders | 2 of 10 |
| Glucose, high (hyperglycemia) | Blood and lymphatic system disorders | 1 of 10 |
| Hemorrhage, Nose | General disorders | 1 of 10 |
| Hypertension | Cardiac disorders | 1 of 10 |
| Neuropathy: motor | Nervous system disorders | 1 of 10 |
| Neuropathy: sensory | Nervous system disorders | 1 of 10 |
| Pain - Extremity-limb | General disorders | 1 of 10 |
Serious events with the highest arm-level rate: hemorrhage, abdomen nos (10.0%).
For the Sunitinib Malate group, the registry lists 10 participants at risk for serious adverse events and 1 participant affected. For other adverse events, it lists 10 at risk and 8 affected. The top serious adverse-event term is “Hemorrhage, Abdomen NOS,” listed at 10.0%. These figures are registry summaries, not a comparison with an untreated or alternative-treatment group.
The at-risk and affected fields describe participants, rather than the total number of event episodes. The record does not give a full event-level listing or explain the observation period for each participant. The serious-event term is listed with a percentage, but the registry does not give additional detail about its clinical course or attribution. A listed event during a study does not, on its own, establish that the study treatment caused it.
The deaths field is not reported in the registry record. The record also does not report a detailed safety analysis, exposure-adjusted event rates, or a comparison of risk between groups. Since the study has a single group, these summaries can describe recorded events among participants but cannot estimate a relative safety difference against a control treatment.
12. What the Result Does, and Does Not, Mean
The study record establishes that a phase II, single-group investigation of sunitinib was conducted in patients with relapsed or refractory metastatic germ cell tumors. It specifies an oral daily regimen of 37.5 mg for 6 weeks and identifies confirmed complete or partial objective response by RECIST criteria as the primary endpoint. It also records adverse-event counts for the Sunitinib Malate group.
The record does not report the primary response count, response rate, or uncertainty interval. It therefore does not establish from the posted results how often the endpoint occurred or how precisely that frequency was estimated. The absence of an analysis entry also means that no primary statistical analysis can be reviewed. These gaps limit interpretation of the endpoint, regardless of the stated study purpose.
Even a reported response proportion in this design would describe outcomes among treated participants and would not provide a randomized comparison. The lack of a control group means that a treatment contrast cannot be estimated from this trial alone. The record does not provide information that would allow the response experience to be separated from other influences on disease course.
The adverse-event summaries show that events were recorded among participants, including a serious adverse-event count and a listed serious term. They do not show whether the events were caused by sunitinib, how long participants were observed, or how event risks compare with another treatment. The registry record also does not report deaths in the corresponding field.
In general, clinical interpretation depends on both the estimate and its uncertainty, the population analyzed, the outcome definition, and the design used to generate the evidence. Here, the endpoint definition and design are available, but the primary estimate and analysis details are not. The study record should therefore be read as a description of the trial setup and posted safety summaries, not as a complete account of the primary response findings.
13. Why This Trial Matters Statistically
This trial is statistically notable because its stated clinical question concerns objective response, while its single-group design does not include a randomized comparator. That distinction separates description from comparative inference. A response count in the treated group can summarize observed outcomes, but a randomized contrast requires a comparison structure that this record does not have.
The endpoint is clearly framed as confirmed complete or partial response under RECIST criteria, but its timing is not fully explained: the title refers to assessment after 2 courses, while the timeframe field says 2 years. Such details matter because response estimates depend on the assessment schedule and on which participants are considered evaluable. Those operational and analysis details are not reported in the registry record.
The contrast between the actual single-group design and the stated two-arm sample-size planning assumptions is also important. The planning illustration specifies equal allocation between two arms, a two-sided significance level of 0.05, and a normal approximation. Those are assumptions for a hypothetical planning exercise, not features of actual assignment in this study. Planning calculations should not be mistaken for observed power or a result of the trial.
Finally, the registry record lists adverse-event counts but no primary analysis entries. This leaves a gap between a defined endpoint and an interpretable estimate of that endpoint. For readers learning to assess clinical evidence, the study illustrates why design, outcome definition, denominator, missing-data handling, and reported uncertainty all matter alongside a trial's stated purpose.
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 central limitation for interpreting efficacy is that the primary response result is not reported in the registry record. The registry defines the outcome but gives no response count, rate, or uncertainty interval. No analysis entries are listed, so the method used to analyze the endpoint cannot be reviewed.
The study is single group and has no randomized comparator. Its design therefore cannot provide a randomized comparison with another treatment or a control condition. The registry reports no detailed participant characteristics or eligibility criteria beyond the disease context and condition labels, limiting assessment of whom the enrolled group represents.
Important endpoint details are also absent. The title specifies response assessment after 2 courses, whereas the timeframe is listed as 2 years; the relationship between these descriptions is not explained. The record does not state how many participants had an evaluable assessment, how response confirmation was conducted, or how missing or unevaluable outcomes were handled. The analysis population is not reported in the registry record.
The record does not report treatment exposure beyond the planned daily regimen, including actual treatment duration, adherence, dose modifications, or discontinuation. For safety, it provides selected participant counts and a top serious-event term, but the observation period, event-level details, attribution, and deaths are not reported in the registry record.
The sample-size illustration uses equal allocation between two arms, whereas the actual study is single group. Its resulting values are not reported, and the primary observed effect needed to interpret the illustration is unavailable. These limitations mean the registry record supports a description of the trial structure and selected safety summaries, but not a complete statistical appraisal of its primary result.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Sunitinib in Treating Patients With Metastatic Germ Cell Tumors That Have Relapsed or Not Responded to Treatment — NCT00453310.
- Registry record last updated 2015-10-27; status last verified 2015-09.
- Results first posted 2015-10-27.
- 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 NCT00453310 as a completed phase II, single-group study sponsored by Memorial Sloan Kettering Cancer Center. Its recorded start was 2007-03, primary completion was 2008-11, and enrollment was 10. The study examined sunitinib malate in patients with relapsed or refractory metastatic germ cell tumors.
The planned regimen was a continuous daily oral dose of 37.5 mg for 6 weeks, described as a 42-day cycle. The primary endpoint was confirmed complete or partial objective response by RECIST criteria after 2 courses, with a listed timeframe of 2 years. The response result and analysis are not reported in the registry record.
The safety record lists 1 participant with a serious adverse event among 10 at risk and 8 with other adverse events among 10 at risk. “Hemorrhage, Abdomen NOS” is listed as the top serious term at 10.0%. The record has no randomized comparison group and no analysis entries.