Sunitinib Malate in Refractory Germ Cell Tumors: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Sunitinib Malate in Refractory Germ Cell Tumors (NCT00912912), a na phase 2 study. The primary outcome is 12 Week Progression Free Survival Rate in Refractory Germ Cell Tumors Treated With Sunitinib Malate.

2. Clinical Question

This question is narrower than whether the intervention changes outcomes compared with another treatment or with no treatment. A single-group study can describe outcomes among participants who received the intervention, but without a concurrent comparator it cannot by itself separate treatment effects from other explanations for those outcomes. The registry record also does not report a statistical analysis of the endpoint.

3. Trial Design

This was a Phase II, single-group study of sunitinib malate in refractory germ cell tumors. The registry record lists enrollment of 5 participants. Allocation is listed as NA, and masking is listed as none. No concurrent comparison group is described, so the study design does not support a like-for-like comparison between treatment arms.

The intervention description specifies 50 mg capsules taken by mouth once a day for 4 weeks in a row in a 6 week cycle. The registry record does not report how long each participant received treatment, whether treatment was interrupted, or how closely participants followed the schedule.

The study start is listed as 2009-05 and primary completion as 2014-12. Its status is terminated. The record does not give a termination date or explain why the study was terminated, and the dates alone do not establish how long any participant was followed.

The primary outcome measure was the 12-week progression-free survival rate, expressed as a percentage of participants. The endpoint description specifies how progression was defined and how deaths or loss to follow-up during therapy were classified. However, the registry record does not report the number of participants who were progression-free at 12 weeks, the observed percentage, or any analysis results. It also does not report the protocol, a sample-size justification, or details of the statistical plan.

As a single-group study, this design can describe outcomes in the enrolled group if those results are reported. It cannot provide a direct randomized estimate of the difference between sunitinib malate and a comparator. The small enrollment also means that any reported percentage would need to be read in light of how little information a small group can provide about the range of outcomes in a wider population.

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.

Before assignment: Study was closed early due to low rate of response and slow accrual.

Overall Study

Reasons for not completing this period:

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 12 Week Progression Free Survival Rate in Refractory Germ Cell Tumors Treated With Sunitinib Malate, measured over 12 weeks.

The primary endpoint is the percentage of participants who were progression-free at 12 weeks. Progression-free survival is a time-to-event concept: it concerns the time until disease progression or death. In this registry record, the reported measure is framed as a percentage at a specified time point rather than as a full account of event times.

The description says that measurable disease or response was recorded from the start of treatment until disease progression or recurrence. It defines progressive disease as at least a 20% increase in the sum of the longest diameter of target lesions, using the smallest recorded sum since treatment started as the reference, or the appearance of one or more new lesions, or unequivocal progression of existing non-target lesions. Response evaluation was to follow Response Evaluation Criteria in Solid Tumors (RECIST) guidelines. Participants who died during therapy or were lost to follow-up were to be counted as having progressive disease.

In general, a time-specific endpoint can make the clinical question concrete by asking about status at a defined follow-up point. Its interpretation depends on the stated event definitions and on whether follow-up is sufficiently complete. Here, the registry record does not report the endpoint result, the follow-up completeness, or the number of participants whose status was determined at 12 weeks. The endpoint definition therefore describes how the measure was intended to be constructed, not what outcome was observed.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does the endpoint measure?

The primary measure is a percentage of participants at a fixed follow-up point: 12 weeks. The endpoint description treats progression, recurrence, death during therapy, and loss to follow-up according to its stated rules. In general, a binary endpoint classifies each participant into one of two outcome categories, such as meeting or not meeting a specified condition by a specified time. The registry record gives the intended classification rules but does not report the number in either category.

When a percentage is reported, its interpretation depends on both the numerator (the participants meeting the endpoint) and the denominator (the participants included in the calculation). Neither the endpoint count nor an analysis population is reported. The listed enrollment is 5, but the record does not state how many participants contributed to the primary outcome calculation.

What would an uncertainty interval add?

In general, a confidence interval describes the statistical uncertainty around an estimated quantity under a specified method and its assumptions. For a percentage based on a small group, an interval can show that the true underlying rate may be compatible with a broad range of values, even when a single point estimate is available. The registry record does not report a confidence interval or the endpoint percentage, so no estimate of precision can be read from this record.

A confidence interval is not a guarantee that the true value lies inside the interval, nor does its width resolve limitations in study design. Even a precisely estimated single-group rate would not, on its own, provide a randomized comparison. The registry record lists no interval or inferential analysis to assess.

What can a single-group design establish?

The study is described as single-group, with allocation listed as NA and no concurrent comparator listed. In general, a single-group study can describe the outcomes observed among enrolled participants. A comparative treatment effect, by contrast, requires a comparison that estimates how outcomes differ between treatment conditions. Without a concurrent control group, the observed course cannot be distinguished from the course that might have occurred without the intervention or under another intervention.

Masking is listed as none. The record does not explain how outcome assessments were conducted or whether any safeguards were used to reduce assessment bias. RECIST is named as the response evaluation framework, and the endpoint includes specific progression criteria, but the record does not provide participant-level assessments or the final outcome determinations.

What does the analysis record contain?

The registry record lists one outcome measure and no analyses. It does not report a point estimate, confidence interval, hypothesis test, model, or adjustment method for the primary outcome. Therefore, statistical significance cannot be assessed, and no analysis-specific assumptions can be evaluated. The protocol and full statistical analysis plan are not reported in the registry record.

In general, the analysis plan specifies how outcomes are summarized and how uncertainty is quantified. It can also define the analysis population, the treatment of missing observations, and any adjustments for multiple comparisons. Without those details, readers should not assume that a particular method was prespecified or used.

How should planning illustrations be read?

The registry record's sample-size note describes planning illustrations using a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect to detect expressed as a fraction of the observed effect. It distinguishes time-to-event illustrations, which use a total number of events, from other illustrations, which use participants per arm. These assumptions describe planning calculations, not the design actually listed for this study.

The study itself is single-group, and the registry record does not report an observed endpoint effect or numerical planning estimates. The illustrations therefore cannot be read as the trial's planned enrollment, its observed power, or evidence that the study had a particular probability of detecting an effect. In general, sample-size calculations depend on assumptions about the effect, variability, significance level, and design. Changing those assumptions changes the planning result; it does not change what was observed.

Overall, the available statistical information establishes the endpoint definition and a few design characteristics, but not an analyzed outcome. No reported estimate or uncertainty measure is available to support a quantitative conclusion about progression-free status at 12 weeks.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Sunitinib Malate")
n      <- c(5)
events <- c(1)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Sunitinib Malate  5  1  20.0  0.5  71.6  3.6  62.4  4.3  64.1

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists one outcome measure and no analyses. It does not report whether other outcomes were collected, whether multiple hypotheses were considered, or whether any multiplicity strategy was planned. The protocol and statistical analysis plan are not reported in the registry record. It would therefore be inappropriate to infer that multiplicity was addressed, or that it was irrelevant, from the listed outcome alone.

In general, multiplicity arises when several outcomes, time points, subgroups, or statistical tests are examined. Considering many possible results can increase the chance of finding an apparently notable result by chance. A prespecified analysis plan can clarify which questions are primary and how multiple testing is handled. No such details are available, and no statistical test results are posted.

The endpoint description states that participants who died during therapy or were lost to follow-up were to be counted as having progressive disease. This is a stated endpoint rule, not a report of how many participants experienced either circumstance. The record does not report the number lost to follow-up, the completeness of 12-week assessments, or any other missing-data handling procedures.

The registry record also does not identify an analysis population or state how many enrolled participants were included in the primary endpoint calculation. Enrollment is reported as 5, but that fact alone does not establish the denominator used for the outcome. In general, missingness and analysis-population definitions can affect an estimate, particularly when the sample is small. The available record does not permit assessment of those issues for this endpoint.

12. 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: pain (mid-back) (20.0%), pain (left-chest) (20.0%), pain (thoracic back) (20.0%), headache (20.0%), hemorrhage (20.0%).

The safety information lists 5 participants at risk for serious adverse events and 3 participants affected in the Sunitinib Malate group. For other adverse events, 5 participants were listed at risk and 5 affected. These are group-level counts; the record does not provide participant-level event histories or a description of how events were collected.

The listed serious adverse event terms are PAIN (MID-BACK), PAIN (LEFT-CHEST), PAIN (THORACIC BACK), HEADACHE, HEMORRHAGE, and DEATH, each with a reported value of 20.0%. These percentages should be read as the registry's term-level figures, not as a complete description of the timing, severity, duration, or clinical context of each event. The record does not say whether the same participant contributed to more than one term, so the terms should not be added or treated as distinct participants.

The deaths count is not reported in the registry record, even though DEATH appears among the serious event terms. The term-level percentage should not be used as a substitute for a separately reported deaths count. The safety information is descriptive and, with no comparator group, does not establish comparative safety. It also does not provide enough detail to assess causality or attribute an event to the intervention.

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

The registry record documents a Phase II, single-group study of sunitinib malate in refractory germ cell tumors and identifies a 12-week progression-free survival rate as its primary outcome. It also gives the endpoint's progression criteria and the intervention schedule. Those details establish what the study set out to describe, but the record does not report the endpoint result or any statistical analysis.

As a result, the posted information does not show what percentage of participants were progression-free at 12 weeks, how uncertain that percentage would be, or how many participants experienced progression, death, or loss to follow-up under the endpoint rules. It is not possible to draw a numerical conclusion about the primary outcome from the record.

Even if a single-group outcome estimate were available, it would describe the enrolled participants rather than directly compare outcomes with another treatment or a control condition. No comparator is listed, and allocation is NA. In general, that design limitation prevents a direct estimate of the difference attributable to the intervention. The available record also does not describe enough about participant characteristics or follow-up to judge how broadly any observed outcome might apply.

The safety listing reports affected participants and serious event terms, but it does not give sufficient detail to establish causes, event histories, or comparative risks. Its death term should be distinguished from the separate deaths count, which is not reported in the registry record.

The appropriate conclusion is therefore about the limits of the posted evidence: the study's design and intended endpoint are described, but the primary result and its precision are absent. Neither a favorable nor an unfavorable treatment conclusion follows from the information reported.

14. Why This Trial Matters Statistically

This study illustrates why an endpoint definition and an endpoint result are different kinds of information. The registry record explains how progression was to be classified and identifies a 12-week percentage as the primary measure. Without the observed percentage, however, readers cannot assess the outcome itself or its statistical precision.

The design also highlights the distinction between describing an outcome and estimating a comparative effect. A single-group study can provide a description of the enrolled participants' outcomes if results are reported. It does not supply a concurrent estimate of what would have happened under another condition. This distinction matters when interpreting any single-group treatment study, regardless of whether the outcome measure is clearly defined.

Enrollment of 5 further emphasizes the importance of uncertainty and denominator transparency. In general, small groups yield limited information about the range of outcomes that may occur in a broader population. Reporting the participant count for each outcome, an estimate, and an uncertainty interval would help readers understand the evidence's precision. Those outcome details are not reported in the registry record.

The record also shows why safety terms need careful reading. Several serious event terms have the same reported percentage, but term-level figures are not necessarily distinct participants and do not provide event context or causality. A separate deaths count is not reported, despite a DEATH term in the list.

Finally, planning illustrations should not be confused with observed results or actual study power. The listed assumptions describe hypothetical calculations, while the trial is single-group and the primary outcome result is absent. Taken together, these points make the record useful for understanding the intended question and design, but insufficient for a quantitative assessment of the primary outcome.

15. 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 of the registry record is that it does not report the primary endpoint result or any analyses. No observed 12-week progression-free survival percentage, endpoint count, confidence interval, or hypothesis test is listed. Without these, the primary outcome's magnitude and precision cannot be assessed.

Enrollment was 5, so the study involved a small group. Even with a reported estimate, limited enrollment would constrain the information available about outcome variability and generalizability. The registry record does not provide the endpoint denominator or state how many participants completed the relevant assessment.

The single-group design and absence of a concurrent comparator limit interpretation to outcomes in the enrolled group, if those outcomes are reported. The record cannot support a direct comparison with another treatment or control condition. Masking is listed as none, but the record does not describe outcome-assessment procedures or safeguards against assessment bias.

Follow-up and missing-data details are also limited. The endpoint definition counts participants who die during therapy or are lost to follow-up as having progressive disease, but the record does not report how many participants fell into those categories or how complete follow-up was. It does not identify the analysis population or provide participant-level outcome information.

The study is listed as terminated, but the reason and termination date are not reported in the registry record. A protocol, sample-size justification, detailed statistical plan, multiplicity approach, and explanation of the absence of analysis results are also not reported. The safety information is limited to group counts and event terms, without timing, context, or a separately reported deaths count.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Sunitinib Malate in Refractory Germ Cell Tumors — NCT00912912.
• Registry record last updated 2016-04-15; status last verified 2016-03.
• Results first posted 2016-01-27.
• 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.

19. Record Summary

NCT00912912 is a Phase II study titled Sunitinib Malate in Refractory Germ Cell Tumors, sponsored by M.D. Anderson Cancer Center. The registry record describes a single-group study with enrollment of 5 and no masking. The intervention was oral sunitinib malate, 50 mg once a day for 4 weeks in a 6 week cycle.

The study start is listed as 2009-05, primary completion as 2014-12, and status as terminated. Its primary outcome was the 12-week progression-free survival rate, measured as a percentage of participants. The record gives progression criteria but does not report the endpoint result or any analysis. The reason for termination, protocol, and detailed statistical plan are not reported in the registry record.