Sitravatinib in Advanced Liposarcoma and Other Soft Tissue Sarcomas: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Sitravatinib in Advanced Liposarcoma and Other Soft Tissue Sarcomas (NCT02978859), a na phase 2 study. The primary outcome is Number of Participants Who Were Progression Free.

2. Clinical Question

This is a Phase II trial sponsored by Columbia University. Its stated purpose was to evaluate sitravatinib for advanced liposarcoma, while the arm description also refers to locally advanced and unresectable or metastatic sarcoma. The wording matters: the registry’s stated treatment group is broader than the two conditions listed in the condition fields, and the entry does not give enough participant-level detail to establish exactly how enrolled patients were distributed across disease types.

The clinical question is consequently about the observed experience of a treated group, not about a treatment contrast against usual care or another therapy. Without a comparator, the record cannot establish how the same participants would have fared under an alternative treatment. The endpoint can describe disease status at a specified time, but its value is not posted in the record.

3. Trial Design

The record describes a Phase II, single-group study of sitravatinib, also called MGCD516, in advanced liposarcoma and other soft tissue sarcomas. Enrollment is listed as 29. The study is marked completed, with a start date of 2016-11 and primary completion in 2020-01. Masking is listed as none, and allocation as NA. These entries describe an open-label, non-randomized treatment group rather than a blinded, randomized comparison.

The intervention is listed as a drug. The brief summary describes sitravatinib as an oral small molecular receptor tyrosine kinase inhibitor. The arm description says patients with locally advanced and unresectable or metastatic sarcoma would receive MGCD516 at the principal investigator’s discretion until disease progression, unacceptable toxicity or adverse event(s), or withdrawal of consent. The record does not report a dosing schedule, dose-modification rules, or additional treatment details.

The primary outcome is the number of participants alive and without evidence of disease progression at the 12-week scan after starting treatment, assessed using RECIST criteria v1.1. Secondary outcomes listed are treatment-related adverse events, overall response rate, progression-free survival, and overall survival. Five outcome measures are listed in total, but numerical results for the primary endpoint are not reported in the registry record.

Because the design has no control group, the posted outcomes can describe what was recorded among participants receiving MGCD516 but cannot, by themselves, separate treatment effects from the natural course of disease, participant selection, or other influences. The registry record does not report the protocol’s sample-size justification, planned statistical analysis, or details about how outcome assessments were conducted beyond the primary endpoint description.

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

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 Number of Participants Who Were Progression Free, measured over 12 weeks.

The primary endpoint is a count of participants who were alive and had no evidence of disease progression at the 12-week scan after starting treatment. Progression is defined using RECIST criteria v1.1. The endpoint is therefore a status assessment at a specified time, rather than a measure of how much an individual tumor changed or how long each participant remained progression-free.

A count can be useful for describing how many participants in a treated group met a prespecified clinical and imaging condition at that assessment. Its interpretation depends on the number of participants included in the analysis and on whether each participant’s status at the assessment is known. The registry record lists enrollment as 29, but does not report the primary endpoint count, the number assessed at the 12-week scan, or how missing assessments were handled. Thus, the endpoint is defined, but its observed result and precision cannot be evaluated from the registry entry.

In general, a fixed-time status endpoint provides a snapshot at the chosen assessment point. It does not by itself describe the timing of progression before or after that point, and it should not be treated as interchangeable with progression-free survival, which is separately listed as a secondary outcome. The record does not report a confidence interval or other measure of uncertainty for the primary count. Without those results, readers cannot assess how precisely the endpoint was estimated or compare it with a prespecified benchmark.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes the primary endpoint as a count: the number of participants alive and without evidence of disease progression at the 12-week scan after treatment began. The assessment uses RECIST criteria v1.1. The endpoint’s reported unit is participants, and the record classifies the measure as a count of participants with an event. In this context, the event is meeting the defined status at the specified assessment, not experiencing an adverse event.

What does a fixed-time count represent?

In general, a fixed-time count summarizes how many participants meet a stated condition at a particular assessment point. It can be easy to communicate, but it does not preserve all information about when progression occurred or how long each person remained free of progression. Those are different questions, which is consistent with the registry listing progression-free survival separately as a secondary outcome. The record does not report the numerical primary count, so the endpoint’s observed frequency cannot be interpreted.

A count also needs a clearly identified analysis population. For example, interpretation depends on whether the analysis includes all enrolled participants or only those with an assessment. The registry entries list 29 enrolled but do not report the number assessed at 12 weeks, the analysis population, or the handling of participants without an evaluable scan. No assumption about those choices can be made from the endpoint description alone.

What can the single-group design estimate?

The study is listed as a single group, with allocation NA and masking none. In general, a single-group study can describe outcomes among people who received the intervention. It does not directly estimate a treatment difference against a concurrent control group, because no such group is identified in this record. Differences between the observed outcomes and what might have occurred under another treatment remain unresolved. This is why a descriptive count must not be read as a causal treatment effect.

The record names MGCD516 as the intervention and describes treatment continuation at the principal investigator’s discretion until progression, unacceptable toxicity or adverse event(s), or withdrawal of consent. It does not report the dose, adherence, dose changes, or any statistical adjustments. The description therefore establishes the general treatment context but not the full operational details needed to reproduce or evaluate an analysis.

How should uncertainty be considered?

In general, an estimate is more informative when accompanied by an uncertainty measure. A confidence interval describes a range of values compatible with the observed data under the method’s assumptions; it does not guarantee that the true value lies in the interval. The registry record does not report a confidence interval for the primary endpoint or a statistical test. Because the primary count itself is also not reported, neither the size of the estimate nor its precision can be assessed.

The registry lists enrollment as 29. That figure is the study’s enrollment, not a substitute for the analysis denominator at the 12-week assessment. The record does not state how many participants contributed to each secondary outcome, whether outcome follow-up was complete, or whether the analysis accounted for different follow-up durations. These omissions matter because outcome availability and timing can affect interpretation.

What is reported about the analysis plan?

The registry record does not report a statistical analysis plan, an interim analysis plan, or a sample-size justification. The listed count of analyses is 0. That entry does not provide an analysis result or explain what methods were intended. No adjustment method, model, test, or prespecified benchmark for the primary endpoint is reported. It would be inappropriate to infer such methods from the Phase II label or from the endpoint definition.

The study lists four secondary outcomes: treatment-related adverse events, overall response rate, progression-free survival, and overall survival. In general, when a study considers multiple outcomes, the interpretation of statistical evidence can depend on which outcomes were prespecified as primary and whether adjustments for multiple testing were planned. The registry identifies the primary endpoint and secondary outcomes, but does not report a multiplicity strategy or analysis results for them. The existence of several listed outcomes alone does not establish that any particular adjustment was made.

What do the planning assumptions mean?

In general, sample-size calculations are planning tools whose meaning depends on the assumed effect, error rate, allocation, and analysis method. The recorded planning assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect defined as a stated fraction of an observed effect. They also distinguish total events for time-to-event calculations from participants per arm for other calculations. These are planning illustrations, not observed power and not evidence that the trial used a two-arm design. The study itself is recorded as a single-group trial, and no numerical sample-size illustration or effect value is included in the registry record.

In general, a planning calculation based on an observed effect is retrospective as a design illustration and can be sensitive to the observed estimate. It should not be presented as a justification for the original study design. The registry record does not report the trial’s own sample-size rationale, so the planning assumptions should not be attributed to the protocol. With no primary outcome value posted, the observed effect referenced by the planning description cannot be evaluated from this record.

Taken together, the methods information supports identifying the endpoint’s definition and the single-group structure. It does not establish the full analysis population, missing-data approach, inferential method, multiplicity handling, or primary endpoint result. Those are material elements of a statistical analysis and are not reported in the registry record.

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("MGCD516")
n      <- c(29)
events <- c(12)
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  MGCD516  29  12  41.4  23.5  61.1  25.5  59.3  25.5  59.4

11. Multiplicity, Missing Data and Analysis Populations

The registry identifies one primary outcome and lists four secondary outcomes: treatment-related adverse events, overall response rate, progression-free survival, and overall survival. Having multiple outcomes raises a general interpretive issue: if several statistical claims are tested, the chance of finding an apparently notable result by chance can depend on how many claims are examined and how the analysis is conducted. The registry record does not report whether any adjustment for multiple testing was planned or performed, or how the secondary outcomes were prioritized.

Missing data are also relevant to the primary endpoint because it is defined by status at the 12-week scan. The record does not report how many of the 29 enrolled participants had an evaluable scan, how missing assessments were classified, or whether participants who discontinued treatment before that assessment were included in the endpoint analysis. It also does not describe missing-data methods for the secondary outcomes. No assumptions about these choices should be made.

The registry record does not report the analysis populations used for efficacy or safety outcomes, any interim analyses, or a statistical analysis plan. It lists 0 analyses, and the numerical primary outcome result is not reported. As a result, readers cannot determine whether the reported participants, if any, were analyzed according to an intention-to-treat, per-protocol, or other approach. These omissions limit reproducibility and make it difficult to assess how incomplete follow-up might affect the estimates.

In general, multiplicity and missingness affect interpretation in different ways: multiplicity concerns the context for evaluating multiple statistical findings, while missingness concerns which observations contribute to an estimate and why others are absent. The registry does not provide enough detail to evaluate either issue for this trial’s outcomes.

12. Safety Results

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

Deaths

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: hypertension (grade 3 and 4) (24.1%), fatigue (grade 3) (6.9%), oral mucositis (grade 3) (3.4%), anemia (grade 3) (3.4%), weight loss (grade 3) (3.4%).

The registry safety entry lists 29 participants at risk for serious adverse events, with 12 affected. It also lists 29 at risk for other adverse events, with 17 affected. These are separate categories in the posted safety information. They should not be combined or treated as interchangeable with the primary endpoint count. The registry also lists 21 deaths, but does not report their causes, timing, or relationship to treatment.

Serious-event terms listed include hypertension (Grade 3 and 4) at 24.1%, fatigue (Grade 3) at 6.9%, oral mucositis (Grade 3) at 3.4%, anemia (Grade 3) at 3.4%, and weight loss (Grade 3) at 3.4%. These are the percentages attached to the listed terms in the record. The entry does not provide further detail about the calculation or denominator for each percentage, so they should be quoted as reported rather than reinterpreted.

In general, an adverse-event count is descriptive: it records events or affected participants under the study’s reporting definitions. It does not by itself establish that treatment caused an event. The secondary outcome list includes treatment-related adverse events, but the safety counts do not identify which events were considered treatment-related. The record also does not provide event onset, duration, outcome, or participant-level detail. These limits preclude a fuller account of the safety profile or a comparison with another treatment.

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

The record establishes that a completed Phase II, single-group study evaluated MGCD516 in advanced liposarcoma and other soft tissue sarcomas. It specifies a primary endpoint based on the number of participants alive and without disease progression at the 12-week scan using RECIST criteria v1.1. It also records enrollment of 29 and lists several secondary outcomes and safety entries.

However, the numerical result for the primary endpoint is not reported in the registry record. The entry therefore cannot show how many participants met that endpoint, how precise any estimate was, or whether the result met a target. Numerical results for the listed secondary outcomes are not included in the registry record used for this analysis. The completion status indicates that the study is marked completed; it does not supply the missing endpoint results.

Even if a primary count were available, the single-group design would make it a description of outcomes among treated participants, not a randomized comparison. A comparison with another treatment or with no treatment is not reported. In general, outcomes in a treated group can reflect multiple influences, including participant characteristics and the course of disease, so a single-group result alone cannot isolate a treatment effect.

The safety entries provide counts and listed serious-event terms, but the record does not report the causes or timing of deaths, the relationship of each event to treatment, or detailed methods for the safety analysis. The listed percentages should be understood as registry-reported figures, not as a complete account of all safety outcomes.

Accordingly, the record supports a description of study design, endpoint definition, enrollment, and selected safety reporting. It does not support a conclusion about the size or precision of the primary outcome, a causal treatment effect, or comparative safety. The absence of a posted numerical endpoint result is a substantive limit on what can be inferred from this registry entry.

14. Why This Trial Matters Statistically

This trial is statistically informative as an example of why design and reporting need to be read together. Its endpoint is clearly framed as a fixed-time count: participants alive and without evidence of progression at the 12-week scan using RECIST criteria v1.1. But an endpoint definition is not an outcome estimate. Because the numerical count is not reported in the registry record, readers can understand what was intended to be measured without being able to assess what was observed.

The study is also explicitly single-group, with no allocation to a comparator and no masking. That structure sets a boundary on interpretation. It can support description of the treated group, but it does not create the counterfactual comparison needed to attribute an observed outcome to treatment. This distinction is important when clinical outcome measures are discussed as evidence of an effect.

The safety entries reinforce another statistical distinction: counts of participants affected, serious-event terms, treatment-related outcomes, and deaths are not identical measures. The record lists each in different ways and does not provide the detail needed to connect them fully. Careful reading avoids combining distinct categories or treating descriptive safety counts as evidence of causation.

Finally, the registry identifies several secondary outcomes but does not report their numerical results, the analysis populations, missing-data methods, multiplicity handling, or a statistical analysis plan. Those gaps limit reproducibility and constrain what can be inferred. The record therefore illustrates why transparent outcome reporting and uncertainty estimates matter alongside a trial’s design and endpoint definitions.

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 most direct limitation is that the registry record does not report the numerical result for the primary endpoint. It defines the 12-week progression-free count but gives no observed count or uncertainty estimate. The study is marked completed, yet completion alone does not make the outcome interpretable. Numerical results for the listed secondary outcomes are also not included in the registry record.

The study’s single-group design offers no concurrent control group. Consequently, even if outcome values were posted, they would describe participants receiving MGCD516 and would not provide a randomized estimate of a difference relative to another treatment. Masking is listed as none, and the record does not provide further details about outcome assessment procedures beyond the RECIST criteria for the primary endpoint.

The registry lists enrollment as 29 but does not report the number included in the primary analysis or the number with an evaluable 12-week assessment. It does not describe how missing scans, treatment discontinuation, or incomplete follow-up were handled. The record also does not report participant characteristics, disease-subtype distribution, dosing details, or the statistical analysis plan. These omissions limit assessment of who contributed to the outcomes and how the analyses were conducted.

Safety interpretation is limited by the lack of information about event definitions, attribution, timing, and the causes or timing of the 21 deaths. The record lists serious-event terms and percentages but does not supply further denominator detail for those percentages. It also does not report multiplicity handling for the listed outcomes, interim analysis methods, or the trial’s sample-size justification. These are trial-specific gaps in the registry record, not evidence that a particular method was or was not used.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Sitravatinib in Advanced Liposarcoma and Other Soft Tissue Sarcomas — NCT02978859.
• Registry record last updated 2024-07-18; status last verified 2024-07.
• Results first posted 2024-07-18.
• 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

NCT02978859 is a completed Phase II, single-group study sponsored by Columbia University, evaluating MGCD516, also called sitravatinib, in advanced liposarcoma and other soft tissue sarcomas. Enrollment is listed as 29. The study started in 2016-11, with primary completion in 2020-01. Masking is listed as none and allocation as NA.

The primary endpoint is the number of participants alive and without evidence of disease progression at the 12-week scan, using RECIST criteria v1.1. Its numerical result is not reported in the registry record. Secondary outcomes include treatment-related adverse events, overall response rate, progression-free survival, and overall survival.

Safety entries list 12 participants affected by serious adverse events among 29 at risk and 17 affected by other adverse events among 29 at risk. The record lists 21 deaths and several serious-event terms, but does not report causes or timing of deaths. The single-group design and absent primary result limit the conclusions that can be drawn from this record.