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 A Study of Epacadostat, an IDO1 Inhibitor, in Combination With Pembrolizumab in Patients With Metastatic and/or Locally Advanced Sarcoma (NCT03414229), a non-randomized phase 2 study. The primary outcome is Number of Patients With Best Objective Response Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03414229 |
| Phase | Phase 2 |
| Condition | Sarcoma |
| Allocation | Non-Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 30 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Memorial Sloan Kettering Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
People with locally advanced and/or metastatic sarcoma are the population named in the study title. The registry lists sarcoma as the condition and divides participants into cohorts for UPS, liposarcoma or pleomorphic liposarcoma; leiomyosarcoma; vascular sarcoma subtypes; and other sarcoma. It does not provide further eligibility details in the record.
Intervention
The intervention is the combination of epacadostat and pembrolizumab. The cohort descriptions list epacadostat 100mg twice daily, continuously on days 1-21 of each 3 week cycle, and pembrolizumab 200mg every 3 weeks on day 1 of each 3 week cycle. The descriptions identify epacadostat as oral and pembrolizumab as an IV infusion.
Comparator
The study is nonrandomized and lists parallel cohorts. The registry record does not report a separate control treatment or a like-for-like comparator. The sarcoma subtype cohorts are not randomized treatment groups; the descriptions list the same combination for each cohort. Differences between cohorts therefore should not be read as controlled comparisons of treatments.
Primary question
The primary outcome asks how many participants have a best objective response by 24 weeks, assessed by RECIST 1.1. This is a count-based endpoint, not a reported comparison of the combination with another treatment. The registry record does not report the response count, response rate, or an analysis estimate. As a result, the posted information identifies the question the study set out to assess but does not establish the observed response finding.
3. Trial Design
This is a phase II, nonrandomized, unmasked, parallel study sponsored by Memorial Sloan Kettering Cancer Center. The registry lists an enrollment of 30 and describes participants with sarcoma in four subtype-based cohorts: UPS, liposarcoma or pleomorphic liposarcoma; leiomyosarcoma; vascular sarcoma subtypes; and other. These cohorts are not randomized arms for comparing different treatments. Each cohort description lists epacadostat with pembrolizumab.
The treatment descriptions specify epacadostat 100mg twice daily, continuously on days 1-21 of each 3 week cycle, and pembrolizumab 200mg every 3 weeks on day 1 of each 3 week cycle. They identify epacadostat as oral and pembrolizumab as an IV infusion. The registry record does not describe dose modifications, treatment duration beyond the stated schedule, or other treatment-management details.
The primary outcome is the number of participants with best objective response by 24 weeks, assessed by RECIST 1.1. Although the registry lists this outcome, it does not report an analysis or an observed response count. The status is active, not recruiting. The listed start date is 2018-01-23 and the listed primary completion date is 2024-09-09. Those registry fields describe the study record and do not supply an outcome result.
Because allocation is nonrandomized and there is no separate comparator reported, the design is suited to describing outcomes in the listed cohorts rather than establishing a randomized treatment contrast. The record does not report the protocol rationale for this design, the planned enrollment rationale, or further details of how participants were assigned among cohorts. Any differences across cohort results, if reported elsewhere, would not by themselves remove the limitations of nonrandom assignment.
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 | UPS, Liposarcoma or Pleomorphic Liposarcoma | Leiomyosarcoma | Vascular Sarcoma Subtypes | Other |
|---|---|---|---|---|
| STARTED | 10 | 5 | 5 | 10 |
| COMPLETED | 1 | 0 | 0 | 0 |
| NOT COMPLETED | 9 | 5 | 5 | 10 |
Reasons for not completing this period:
| Reason | UPS, Liposarcoma or Pleomorphic Liposarcoma | Leiomyosarcoma | Vascular Sarcoma Subtypes | Other |
|---|---|---|---|---|
| Adverse Event | 1 | 0 | 2 | 1 |
| Lack of Efficacy | 7 | 4 | 1 | 9 |
| Change in therapy | 1 | 0 | 0 | 0 |
| Pregnancy | 0 | 0 | 1 | 0 |
| Withdrawal by Subject | 0 | 1 | 1 | 0 |
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | UPS, Liposarcoma or Pleomorphic Liposarcoma | Leiomyosarcoma | Vascular Sarcoma Subtypes | Other | Total |
|---|---|---|---|---|---|---|
| Participants analysed | 10 | 5 | 5 | 10 | 30 | |
| Age, Continuous | years | 54 (35 to 78) | 62 (37 to 76) | 53 (34 to 71) | 45 (24 to 75) | 54 (24 to 78) |
| Sex: Female, Male: Female | Participants | 3 | 3 | 2 | 4 | 12 |
| Sex: Female, Male: Male | Participants | 7 | 2 | 3 | 6 | 18 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 0 | 1 | 0 | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 10 | 5 | 4 | 10 | 29 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 1 | 2 | 3 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 | 1 | 1 |
| Race (NIH/OMB): White | Participants | 9 | 5 | 2 | 7 | 23 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 | 0 | 2 | 0 | 3 |
| Region of Enrollment: United States | Participants | 10 | 5 | 5 | 10 | 30 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Patients With Best Objective Response Rate, measured over by 24 weeks.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants with best objective response by 24 weeks, assessed according to RECIST 1.1. In plain terms, the endpoint is a count of participants meeting the study's objective-response definition during the stated timeframe. The registry labels the outcome as a participant count; it does not report a response count or rate in the posted results.
Objective response is used in clinical research to capture tumor response according to prespecified measurement criteria rather than relying only on a subjective impression. RECIST 1.1 is the named assessment standard in this record. In general, using a common response framework helps make the outcome definition explicit, but interpretation still depends on details such as how assessments were scheduled, how missing assessments were handled, and how response was confirmed. Those details are not reported in the registry record.
The endpoint is not a measure of survival, symptom change, or quality of life. The registry lists deaths among safety information, but those counts do not substitute for the primary response endpoint. Nor does an endpoint definition itself tell the reader whether participants experienced a response. Without the observed response count, the registered endpoint describes what was to be measured, not the study's result.
The timeframe matters: the stated outcome is assessed by 24 weeks. A participant count tied to a specified assessment window has a different interpretation from a time-to-event measure or a response estimate taken over an unspecified period. The registry record does not report the exact analysis population, assessment schedule, response-evaluation details, or handling of participants without an assessment by that timeframe.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a clinically relevant descriptive question: the number of participants with best objective response by 24 weeks under a combination of epacadostat and pembrolizumab in locally advanced and/or metastatic sarcoma. However, the record does not report the observed response count, a response rate, an uncertainty interval, or a statistical analysis for that endpoint. Consequently, the posted information does not permit a quantitative interpretation of the primary outcome.
The study is phase II and nonrandomized. Its four subtype-defined cohorts receive the same listed combination, and no separate control treatment is reported. This means the design does not support a randomized comparison between the combination and another treatment. Even if outcomes were available by cohort, differences between groups could reflect differences in sarcoma subtype or other participant characteristics as well as any treatment-related factor. The record does not describe adjustment methods that might address such differences.
The enrollment is 30. That figure gives the registered study size, but it is not a response result and does not indicate how many participants were included in a particular endpoint analysis. The registry record does not report the number assessed for response, exclusions from analysis, or the amount of missing endpoint information. Without those details, readers cannot assess how completely the primary outcome was observed.
The adverse-event information should be read separately from response. The registry lists serious adverse events, other adverse events, and deaths for the four cohorts. These are descriptive counts, and the record does not provide a comparative safety analysis or attribution of events to treatment. A listed death count does not establish that a death was caused by study treatment. The listed serious-event terms are each reported as 10.0%, but the record does not specify their denominators or additional event-level context.
General statistical interpretation: estimates from a small, nonrandomized study can be imprecise, and a count alone does not communicate the range of plausible underlying response frequencies. A confidence interval, when reported, expresses uncertainty around an estimate under the analysis assumptions; it does not correct design limitations or guarantee that the estimate applies to other populations. No confidence interval for the primary endpoint is reported in the registry record.
Overall, this record establishes the study question, cohort structure, treatment schedule, and selected safety counts. It does not provide the primary response result or sufficient analysis detail to determine the magnitude or precision of response in the studied population. Conclusions about efficacy, comparative benefit, or treatment causation should not be inferred from the design description or safety counts alone.
9. Statistical Methods Explained
General: what the registered endpoint counts. A participant-count endpoint reports how many people meet a stated outcome definition. Here, the named outcome is best objective response by 24 weeks according to RECIST 1.1. A count is an absolute quantity; by itself, it does not convey the proportion of participants who responded unless the relevant analysis denominator is also reported. The registry record does not report either the response count or its analysis denominator. It therefore does not permit calculation or interpretation of an observed response proportion.
General: why the assessment rule matters. An objective-response endpoint depends on a definition for classifying response and on the timing and availability of assessments. A named standard such as RECIST 1.1 makes the assessment framework identifiable. Interpretation can also depend on whether assessments are repeated, whether responses require confirmation, and how participants who stop treatment or miss assessments are classified. Those operational details are not reported in the registry record, so no assumptions about them should be made.
General: uncertainty around an estimate. A point estimate gives a single summary of the observed data, while a confidence interval describes statistical uncertainty around that estimate under specified assumptions. A wider interval indicates less precision; a narrower interval indicates more precision, conditional on the analysis and data. Neither an interval nor a point estimate would, by itself, establish a causal treatment effect in a nonrandomized study. The registry record does not report a point estimate or confidence interval for the primary endpoint.
General: design and comparison. Randomization is used in comparative trials to allocate treatment in a way that can reduce systematic differences between groups. This study is explicitly nonrandomized and lists parallel, subtype-defined cohorts, with the same combination described for each. The registry record does not list a separate control treatment. Accordingly, the design description should not be interpreted as a controlled test of the combination against another intervention. Differences between cohorts could be associated with their underlying disease subtypes or other characteristics; the record does not report a method for separating those influences.
General: what a phase II label does and does not say. A phase label describes the registered development phase, not the magnitude or certainty of an outcome. The phase II designation does not itself establish that a treatment is effective or ineffective, and it does not reveal the planned statistical analysis. The registry record does not report a sample-size justification or detailed statistical analysis plan.
General: interpreting sample-size illustrations. Planning illustrations associated with the posted analysis use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is specified as a fraction of the observed effect. Such illustrations are conditional calculations, not the study's own design rationale. They are not observed power and do not describe how many participants actually responded. The registry record does not report a sample-size justification for this trial. Because this study is nonrandomized and does not list a separate comparator, hypothetical equal allocation between two arms should not be mistaken for the design used in the registry.
General: why over-dispersion can matter. For count outcomes, a simple statistical model may assume that variability follows a particular relationship to the average count. Over-dispersion means the observed variability is greater than that assumption allows. If it is ignored, uncertainty can be understated and inferential results may be too optimistic. The primary outcome in this record is a participant count, but the registry does not report a model for it or an assessment of over-dispersion. No conclusion about whether over-dispersion was relevant to the study analysis can be drawn.
General: analyses and multiplicity. When researchers test several outcomes, subgroups, or hypotheses, the chance of at least one apparently unusual finding can increase unless the analysis plan addresses multiplicity. The registry lists one primary outcome measure and no analyses. It does not report a hierarchy of outcomes, multiplicity adjustment, or interim analyses. The absence of those details from the registry record is not evidence that a particular method was or was not used; the analysis plan is simply not reported in this record.
General: analysis population and missingness. A result's meaning depends on which participants are included and how missing outcome measurements are handled. An analysis might use all enrolled participants, only those with an evaluable assessment, or another prespecified group. Missing assessments can affect a response count if the participants without assessment are handled differently from participants with observed response data. The registry record does not identify the analysis population or missing-data method for the primary endpoint. Thus, the enrolled count should not automatically be treated as the endpoint denominator.
General: safety summaries. Safety counts describe the events recorded for participants at risk in the stated cohort. Serious adverse events and other adverse events are separate registry categories. These counts do not automatically show whether an event was caused by treatment, how long participants were observed, or whether the groups had comparable exposure. The record does not report an adjusted safety comparison or event attribution, so the counts are descriptive rather than causal evidence.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary endpoint, the number of participants with best objective response by 24 weeks according to RECIST 1.1, and reports zero analyses. It does not report a statistical analysis plan, a hierarchy of outcomes, any multiplicity adjustment, or interim analyses. Without those details, it is not possible to determine how the study planned to manage the interpretation of multiple outcomes, subgroup findings, or repeated looks at the data. This absence should not be treated as evidence that a particular approach was used or not used outside the registry record.
The registry record also does not identify the analysis population for the primary endpoint. Although enrollment is 30, the record does not say how many participants had an evaluable response assessment by 24 weeks or how participants without an assessment were treated in the analysis. The enrolled number therefore cannot be assumed to be the primary endpoint denominator.
Missing outcome data can affect the interpretation of a participant count. For example, the result can differ depending on whether participants with unavailable assessments are excluded, classified in a particular way, or addressed using a statistical method. These are general possibilities, not descriptions of this trial's procedure. The registry record does not report its missing-data assumptions or method.
It also does not report further detail on protocol deviations, withdrawals, or analysis exclusions. These omissions limit assessment of how closely the analyzed population might correspond to the registered enrollment. The posted record defines the endpoint and study structure, but does not provide enough analysis information to evaluate these issues.
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 |
|---|---|---|---|
| UPS, Liposarcoma or Pleomorphic Liposarcoma | 1 | 10 | 10.0% |
| Leiomyosarcoma | 0 | 5 | 0.0% |
| Vascular Sarcoma Subtypes | 0 | 5 | 0.0% |
| Other | 1 | 10 | 10.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| UPS, Liposarcoma or Pleomorphic Liposarcoma | 7 of 10 |
| Leiomyosarcoma | 4 of 5 |
| Vascular Sarcoma Subtypes | 2 of 5 |
| Other | 9 of 10 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| UPS, Liposarcoma or Pleomorphic Liposarcoma | 10 of 10 |
| Leiomyosarcoma | 5 of 5 |
| Vascular Sarcoma Subtypes | 5 of 5 |
| Other | 10 of 10 |
Most frequent serious adverse event terms
| Event term | Organ system | UPS, Liposarcoma or Pleomorphic Liposarcoma | Leiomyosarcoma | Vascular Sarcoma Subtypes | Other |
|---|---|---|---|---|---|
| Hypertension | Vascular disorders | 0 of 10 | 0 of 5 | 0 of 5 | 1 of 10 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 1 of 10 | 0 of 5 | 0 of 5 | 0 of 10 |
| Fever | General disorders | 1 of 10 | 0 of 5 | 0 of 5 | 0 of 10 |
| Non-cardiac chest pain | General disorders | 1 of 10 | 0 of 5 | 0 of 5 | 0 of 10 |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | 1 of 10 | 0 of 5 | 0 of 5 | 0 of 10 |
Most frequent non-serious adverse event terms
| Event term | Organ system | UPS, Liposarcoma or Pleomorphic Liposarcoma | Leiomyosarcoma | Vascular Sarcoma Subtypes | Other |
|---|---|---|---|---|---|
| Fatigue | General disorders | 4 of 10 | 4 of 5 | 4 of 5 | 5 of 10 |
| Pain | General disorders | 5 of 10 | 3 of 5 | 0 of 5 | 5 of 10 |
| Anemia | Blood and lymphatic system disorders | 3 of 10 | 1 of 5 | 2 of 5 | 3 of 10 |
| Constipation | Gastrointestinal disorders | 3 of 10 | 3 of 5 | 1 of 5 | 2 of 10 |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | 4 of 10 | 1 of 5 | 2 of 5 | 2 of 10 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 3 of 10 | 1 of 5 | 1 of 5 | 3 of 10 |
| Alkaline phosphatase increased | Investigations | 1 of 10 | 0 of 5 | 3 of 5 | 3 of 10 |
| Cough | Respiratory, thoracic and mediastinal disorders | 2 of 10 | 1 of 5 | 1 of 5 | 3 of 10 |
| Hypertension | Vascular disorders | 2 of 10 | 2 of 5 | 1 of 5 | 2 of 10 |
| Headache | Nervous system disorders | 3 of 10 | 1 of 5 | 1 of 5 | 1 of 10 |
Serious events with the highest arm-level rate: hypertension (10.0%), dyspnea (10.0%), fever (10.0%), non-cardiac chest pain (10.0%), pneumothorax (10.0%).
The registry lists safety information separately for the four sarcoma cohorts. For UPS, liposarcoma or pleomorphic liposarcoma, 10 participants are listed as at risk for serious adverse events and 1 as affected; 10 are listed as at risk for other adverse events and 10 as affected; deaths are listed as 7. For leiomyosarcoma, the corresponding figures are 5 at risk and 0 affected for serious events, 5 at risk and 5 affected for other events, and 4 deaths. For vascular sarcoma subtypes, the figures are 5 at risk and 0 affected for serious events, 5 at risk and 5 affected for other events, and 2 deaths. For other sarcoma, the figures are 10 at risk and 1 affected for serious events, 10 at risk and 10 affected for other events, and 9 deaths.
The registry also lists hypertension, dyspnea, fever, non-cardiac chest pain, and pneumothorax among the top serious-event terms, each at 10.0%. The record does not give further denominator or event-level context for these terms. These figures are descriptive entries, not a comparative analysis. The record does not establish that events or deaths were caused by the combination, and death counts should not be interpreted as treatment-attributed deaths. Differences between cohorts should not be treated as treatment comparisons because the study is nonrandomized and does not list a separate control group.
12. What the Result Does, and Does Not, Mean
The registry record shows that a phase II, nonrandomized study was organized to assess epacadostat plus pembrolizumab in people with locally advanced and/or metastatic sarcoma. It identifies four sarcoma subtype cohorts, describes the combination schedule, and names best objective response by 24 weeks according to RECIST 1.1 as the primary outcome. These registry entries explain the study's question and structure.
The record does not report the observed primary endpoint result. It gives no response count, response rate, confidence interval, or analysis estimate. Therefore, it cannot establish how frequently objective response occurred in the enrolled participants or how precise any response estimate might be. The registered outcome definition is not itself an outcome finding.
The design also limits what can be inferred. Participants were not randomized, and a separate control treatment is not reported. The subtype-defined cohorts are not a randomized comparison. The record therefore does not establish whether the combination changes response compared with another treatment or with no treatment. General statistical principle: observed outcomes in a nonrandomized cohort can reflect participant and disease characteristics as well as treatment; without a suitable comparative design and analysis, causal attribution is not supported.
The adverse-event information gives cohort-level counts for serious events, other events, and deaths, along with selected serious-event terms. These entries are useful descriptive safety information, but the record does not report treatment attribution, comparative risk estimates, or enough context to interpret the listed deaths as caused by treatment. They also do not replace the missing response result.
Accordingly, the record supports describing the planned endpoint, study design, treatment schedule, and listed safety counts. It does not support a conclusion that the combination does or does not work, a comparison with another therapy, or a claim about individual treatment decisions. Those broader conclusions would require results and analysis details not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of the distinction between a registered research question and a reported result. The registry clearly names an objective-response endpoint, its assessment standard, and its timeframe. Yet the record does not report the response count or analysis. Without the endpoint result, readers cannot assess its magnitude or precision, regardless of how clearly the outcome is defined.
The nonrandomized, parallel cohort structure also matters. The listed cohorts reflect sarcoma subtypes, not a randomized treatment contrast. Recognizing this distinction helps prevent a descriptive comparison across cohorts from being mistaken for evidence that one treatment strategy is superior. The record describes the same combination in each cohort and does not list a separate control treatment.
The safety entries illustrate why event counts need context. Serious events, other adverse events, and deaths are distinct summaries, and the registry lists counts by cohort. Without event attribution, exposure context, and a comparative analysis, those entries should remain descriptive. A count is not by itself proof of causation or a treatment-related risk difference.
General statistical principle: precision and design answer different questions. More precise measurement of an outcome would not remove confounding created by nonrandom allocation, while randomization would not substitute for complete reporting of the endpoint estimate and its uncertainty. A useful interpretation requires both an appropriate design for the intended comparison and transparent outcome analysis.
The registry record also makes clear why analysis details matter. It does not report the endpoint analysis population, missing-data method, or multiplicity approach. These details affect how readers assess whether the reported result, if available, represents the enrolled participants and whether uncertainty has been handled appropriately. The trial therefore offers a concrete reminder that the registered endpoint alone is not enough to reconstruct the statistical evidence.
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 primary limitation for interpreting this record is that the primary response result is not reported. The endpoint is defined as best objective response by 24 weeks according to RECIST 1.1, but there is no response count, rate, interval estimate, or analysis. The statistical evidence for the primary question cannot be evaluated from the registry information.
The study is nonrandomized and lists no separate control treatment. Its cohorts are based on sarcoma subtype, and the record does not report methods to account for differences among participants or cohorts. Accordingly, the design does not provide a like-for-like controlled comparison of the combination with another treatment.
The registry record does not report the primary endpoint analysis population, the number with evaluable assessments, or how missing response assessments were handled. It also does not report the assessment schedule beyond the endpoint timeframe, response confirmation details, protocol deviations, or analysis exclusions. These omissions prevent assessment of the completeness and precise interpretation of the endpoint.
Further statistical limitations include the absence of a reported analysis plan, sample-size justification, multiplicity approach, and interim-analysis information. These items are not reported in the registry record, so no conclusion about what methods were used should be inferred. The enrollment of 30 is listed, but it does not establish the endpoint denominator or the precision of an unreported result.
Safety information is also limited to listed cohort counts and selected terms. The record does not provide event attribution, comparative analyses, or additional context for interpreting the deaths and serious adverse events. These figures should not be treated as causal estimates or as a substitute for the missing primary outcome analysis.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study of Epacadostat, an IDO1 Inhibitor, in Combination With Pembrolizumab in Patients With Metastatic and/or Locally Advanced Sarcoma — NCT03414229.
- Registry record last updated 2026-04-23; status last verified 2026-04.
- Results first posted 2025-05-02.
- 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 record describes NCT03414229, a phase II, nonrandomized, unmasked, parallel study sponsored by Memorial Sloan Kettering Cancer Center. Enrollment is listed as 30, and the status is active, not recruiting. The listed start date is 2018-01-23 and the primary completion date is 2024-09-09.
The study evaluates epacadostat with pembrolizumab in locally advanced and/or metastatic sarcoma. Four subtype-based cohorts are listed, with the same combination schedule described for each. The primary endpoint is the number of participants with best objective response by 24 weeks according to RECIST 1.1.
The registry lists cohort-level serious adverse-event counts, other adverse-event counts, deaths, and selected serious-event terms. It reports no analyses and does not report the primary response result, endpoint analysis population, missing-data method, or statistical analysis plan. The record therefore describes the study design and registered outcome but does not provide the analysis needed to interpret the primary result.