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 Treatment of Milademetan Versus Trabectedin in Patient With Dedifferentiated Liposarcoma (NCT04979442), a randomized phase 3 study. The primary outcome is Compare Progression-free Survival (PFS) as Determined by Blinded Independent Central Review (BICR) Between the Milademetan Treatment Arm and Trabectedin Control Arm.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT04979442 |
| Phase | Phase 3 |
| Condition | Dedifferentiated Liposarcoma |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 175 |
| Arms in the primary outcome | 2 |
| Lead sponsor | Rain Oncology Inc |
| Results posted | Yes |
2. Clinical Question
Population
Participants had unresectable or metastatic dedifferentiated liposarcoma that had progressed on one or more prior systemic therapies, including at least one anthracycline-based therapy. The study description characterizes unresectability as resection judged to cause unacceptable morbidity or mortality.
Intervention
Milademetan, identified in the registry record as RAIN-32, was given orally at 260 mg once daily on Days 1 to 3 and Days 15 to 17 of each 28-day cycle.
Comparator
Trabectedin was given as a 1.5 mg/m2 body surface area dose by 24-hour intravenous infusion every 3 weeks.
Primary question
The primary question was whether progression-free survival, determined by blinded independent central review, differed between the milademetan treatment arm and the trabectedin control arm. Progression-free survival was measured from randomization to documented radiographic progression or death due to any cause.
This is a treatment comparison in a population with advanced disease and prior systemic treatment. The registry record identifies the study as randomized, multicenter, open-label, and Phase 3. Its stated purpose was to evaluate safety and efficacy; the posted primary result focuses on median progression-free survival. The posted arm-level medians and confidence intervals describe the observed time-to-event results, but the registry record does not report a comparative hazard ratio or another sponsor-reported estimate of the relative progression-free survival effect. The distinction matters: comparing arm medians alone does not quantify the full time-to-event contrast or establish how likely the observed difference is to have arisen by chance.
3. Trial Design
The registry record describes a randomized, multicenter, open-label Phase 3 registration study with a parallel-group design. Participants were assigned to milademetan or trabectedin. Masking was not used for treatment assignment, but the primary endpoint was assessed by blinded independent central review. This separation can reduce the influence of knowledge of treatment assignment on the central assessment of radiographic progression, although it does not make the treatment trial itself masked.
The recorded enrollment was 175. The primary outcome population is described as all randomized participants through the final analysis cut-off date, 01 March 2023. The registry record reports 86 participants in the milademetan arm and 89 in the trabectedin arm for the primary outcome. The study status is terminated. A reason for termination is not reported in the registry record, and the record does not state whether termination altered the planned analysis or follow-up.
The recorded study start was 2021-07-14 and the primary completion date was 2023-10-01. These dates provide the registry timeline; they do not, by themselves, explain the duration of follow-up for each participant. The primary endpoint timeframe is from randomization to documented progression or death, up to 13 months. The registry record does not report the randomization procedure, allocation concealment, stratification factors, or the planned sample-size justification. Those design details should not be inferred from the fact that the study was randomized or from the posted enrollment.
The study also listed overall survival, disease control rate, objective response rate, investigator-assessed progression-free survival, and treatment-emergent adverse events among its secondary outcomes. Results are posted for disease control rate and objective response rate. The registry record does not include reported analyses for the listed secondary outcomes as a group, and it does not supply sponsor-reported comparative analyses for the primary time-to-event outcome.
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: A total of 86 subjects were enrolled to the Milademetan arm and a total of 89 subjects were enrolled to the Trabectedin arm. Of the 89 subjects in the trabectedin arm, 79 subjects were treated.
Overall Study
| Stage | Milademetan | Trabectedin |
|---|---|---|
| STARTED | 86 | 89 |
| Received Treatment | 86 | 79 |
| COMPLETED | 34 | 42 |
| NOT COMPLETED | 52 | 47 |
Reasons for not completing this period:
| Reason | Milademetan | Trabectedin |
|---|---|---|
| Death | 43 | 30 |
| Withdrawal by Subject | 8 | 13 |
| Physician Decision | 1 | 1 |
| Lost to Follow-up | 0 | 2 |
| do not want to continue | 0 | 1 |
Analysis population for the primary outcome, as defined in the registry: Analysis population included all the randomized participants up to up to final analysis cut-off date (01 March 2023).
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 | Milademetan | Trabectedin | Total |
|---|---|---|---|---|
| Participants analysed | 86 | 89 | 175 | |
| Age, Continuous | years | 59.3 (SD 13.08) | 61.8 (SD 11.92) | 60.6 (SD 12.53) |
| Sex: Female, Male: Female | Participants | 40 | 36 | 76 |
| Sex: Female, Male: Male | Participants | 46 | 53 | 99 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 3 | 6 | 9 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 71 | 72 | 143 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 12 | 11 | 23 |
| Race/Ethnicity, Customized: Race – American Indian or Alaska Native | Participants | 1 | 0 | 1 |
| Race/Ethnicity, Customized: Race – Asian | Participants | 15 | 12 | 27 |
| Race/Ethnicity, Customized: Race – Black or African American | Participants | 0 | 1 | 1 |
| Race/Ethnicity, Customized: Race – Native Hawaiian or Other Pacific Islander | Participants | 0 | 1 | 1 |
| Race/Ethnicity, Customized: Race – White | Participants | 59 | 63 | 122 |
| Race/Ethnicity, Customized: Race – Unknown | Participants | 11 | 12 | 23 |
| Race/Ethnicity, Customized: Race – Other | Participants | 0 | 0 | 0 |
| Region: North America | Participants | 27 | 37 | 64 |
| Region: Europe | Participants | 47 | 42 | 89 |
| Region: Rest of World | Participants | 12 | 10 | 22 |
6. Primary Endpoint
The registry lists the primary outcome as Compare Progression-free Survival (PFS) as Determined by Blinded Independent Central Review (BICR) Between the Milademetan Treatment Arm and Trabectedin Control Arm, measured over from the randomization date to date of documented progression or death, up to 13 months.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 95% Confidence Interval |
| Unit | months |
| Endpoint classification | Time-to-Event Analysis |
Progression-free survival (PFS) is a time-to-event outcome. In this study it runs from randomization until the earliest of the first objective radiographic documentation of progression or death from any cause. Progression was defined using RECIST v1.1: a 20% increase in the sum of the longest diameter of target lesions, a measurable increase in a non-target lesion, or the appearance of new lesions. The primary assessment was by blinded independent central review, and the stated timeframe extended up to 13 months.
PFS is used in trials of cancer treatments because it captures the time participants remain alive without documented disease progression. It includes more information about timing than a simple count of participants who progressed, but its analysis depends on follow-up and on how participants without a recorded event are handled. The registry record does not report the censoring rules or the specific statistical procedure used to estimate the medians and confidence intervals.
For each arm, the registry record reports a median PFS and a 95% confidence interval. A median is the point in the estimated event-time distribution at which half the observations are on either side; it is not the average time for every participant and does not describe each individual’s course. A confidence interval conveys uncertainty around the estimated median. It is not a range containing a fixed proportion of individual participant outcomes. The arm-specific median estimates are descriptive; without a reported comparative time-to-event estimate, they do not alone establish the size or precision of the treatment contrast.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted primary results give a median PFS of 3.6 months for milademetan, with a 95% confidence interval from 2.1 to 4.1 months. For trabectedin, the median is 2.2 months, with a 95% confidence interval from 1.9 to 4.2 months. These are arm-specific estimates in the analysis population described by the registry record, through the final analysis cut-off date of 01 March 2023.
The intervals overlap substantially. That overlap is a reminder that each median is estimated with uncertainty, but overlap alone is not a formal test of the between-arm difference. Nor does the apparent ordering of the medians establish a statistically reliable or clinically important treatment effect. A comparison of medians is not equivalent to comparing the entire time-to-event distributions, particularly when the registry record does not provide a sponsor-reported hazard ratio, a confidence interval for a relative effect, or a reported test for the primary comparison.
The registry record lists no reported analyses for the primary outcome comparison. Its analysis count is recorded as zero, and the record notes that values are missing or not estimable for a two-arm comparison of this median-time endpoint. Thus, the posted information supports describing the arm-specific medians and their uncertainty, but not attributing a formal comparative conclusion to the sponsor. A recalculated contrast based only on the posted summaries would not substitute for the sponsor’s intended time-to-event analysis; no such recalculation is presented for this endpoint.
The study status is terminated, but the reason is not reported in the registry record. The primary completion date and analysis cut-off are recorded, yet these registry entries do not establish whether the termination was related to efficacy, safety, recruitment, operational considerations, or another reason. No such explanation should be inferred.
For the posted binary secondary outcomes, the arm-level estimates are directionally higher for milademetan, but the registry record does not report sponsor analyses for them. The calculated unadjusted comparisons are exploratory and do not carry the same weight as a prespecified primary comparison. Their uncertainty and the absence of a reported multiplicity plan limit their ability to confirm the primary result or support a separate definitive claim.
Overall, the registry record presents descriptive primary results with substantial uncertainty and without a reported comparative time-to-event analysis. The data should therefore be read as estimates of observed PFS in each arm, not as proof that one treatment is superior, equivalent, or inferior.
9. Statistical Methods Explained
Progression-free survival is analyzed as a time-to-event outcome because participants can experience progression or death at different times after randomization. The primary endpoint combines the first qualifying radiographic progression with death due to any cause. The registry record specifies RECIST v1.1 criteria for radiographic progression and blinded independent central review for the primary assessment. It does not report the statistical method used to estimate the survival curves, medians, confidence intervals, or a between-arm effect.
What does a median time-to-event estimate describe?
In general, a median time-to-event estimate is the time at which an estimated event-time distribution reaches its midpoint. It summarizes the distribution rather than giving the time for every participant. Participants who have not had a recorded event by the end of observation may contribute information up to their last observation, depending on the analysis and censoring rules. The registry record does not report those rules, so the posted median should be interpreted as reported rather than reconstructed from individual follow-up.
The primary median estimates are 3.6 months for milademetan and 2.2 months for trabectedin. Their respective 95% confidence intervals are 2.1 to 4.1 months and 1.9 to 4.2 months. In general, a confidence interval expresses the precision of an estimate under the assumptions of the analysis. It is not an interval for individual outcomes, and it does not directly provide the probability that one treatment is better. A confidence interval for each arm’s median also differs from a confidence interval for a between-arm contrast.
Why is a comparative time-to-event estimate useful?
In general, a hazard ratio can summarize the relative event rate over follow-up, subject to assumptions about how that relative rate behaves over time. Other methods can compare time-to-event distributions without relying on the same assumptions. The registry record does not report a sponsor hazard ratio or other comparative time-to-event result for PFS. It reports the arm-specific medians and intervals, but those summaries cannot fully describe how the distributions compare throughout follow-up. The record also does not report a test statistic or p-value for the primary comparison.
The registry record identifies PFS as measured from randomization to progression or death, with follow-up up to 13 months. The analysis population is described as all randomized participants through the final analysis cut-off date. These statements identify the endpoint and population, but do not specify how participants with incomplete assessments, withdrawal, or other missing follow-up were handled. Censoring rules, assessment schedules, sensitivity analyses, and missing-data procedures are not reported in the registry record.
How should the secondary binary outcomes be understood?
Disease control rate and objective response rate are binary outcomes expressed as percentages of participants. In general, a binary outcome classifies each participant according to whether a defined event or response occurred within the stated assessment period. The registry record reports arm-level percentages and 95% confidence intervals for these outcomes, and posts recalculated unadjusted risk differences with their own confidence intervals and unadjusted p-values. These recalculations use the posted summary counts and are not reported sponsor analyses. They are not adjusted for other trial features or for testing multiple outcomes.
A risk difference expresses the absolute contrast between the proportions in two arms. In general, a confidence interval for a risk difference that includes zero is compatible with no absolute difference as well as with effects in either direction under the calculation’s assumptions. An unadjusted p-value addresses a single comparison without accounting for the number of outcomes or analyses considered. The registry record does not report a multiplicity strategy, so the posted unadjusted secondary comparisons should be treated as exploratory rather than confirmatory.
What do the planning illustrations mean?
The sample-size illustrations are calculations based on observed effects, not the trial’s original design justification and not observed power. The stated assumptions are a two-sided significance level of 0.05, equal allocation, and a normal approximation; the illustrations use specified fractions of the observed effect. In general, such calculations show how a hypothetical future study’s required participants per arm could vary with the effect chosen for detection. They do not show that the current study had a particular probability of detecting an effect, and they do not establish that the observed effect is reliable. The trial’s own sample-size justification is not reported in the registry record.
Finally, the designation of the trial as randomized does not reveal how the sequence was generated or concealed. Open-label treatment assignment means masking was not used for participants or study staff as recorded, while blinded central review was specified for the primary endpoint. The registry record does not report whether other safeguards, stratification procedures, interim analyses, or sensitivity analyses were planned or conducted. These omissions limit how fully the statistical analysis can be evaluated from the posted record.
10. Multiplicity, Missing Data and Analysis Populations
The registry record reports no analyses for the primary time-to-event comparison and no reported analyses for the listed secondary outcomes as a group. It gives the primary analysis population as all randomized participants through the final analysis cut-off date of 01 March 2023. It does not state how missing outcome assessments, incomplete follow-up, withdrawals, or deviations from assigned treatment were handled. It also does not report censoring rules for PFS or identify sensitivity analyses addressing missing data.
A missing-data plan matters because participants may have different amounts of follow-up or may leave observation before progression or death is documented. In general, time-to-event analyses use censoring to represent incomplete observation, and the assumptions behind that treatment of follow-up affect interpretation. The registry record does not describe those assumptions or provide enough information to assess their plausibility. The record’s analysis population statement does not, by itself, explain how every randomized participant contributed to each endpoint.
Multiplicity is also not described. The study listed several secondary outcomes, but the registry record does not report a testing hierarchy, adjustment procedure, or plan for controlling the chance of false-positive findings across outcomes. The posted unadjusted secondary comparisons therefore should not be treated as confirmatory. No interim analyses or stopping rules are reported in the registry record.
The record identifies randomization and blinded independent central review for the primary endpoint, but does not report allocation concealment, the randomization method, or stratification factors. It also does not report the primary analysis model or a sponsor-reported comparative PFS estimate. These omissions leave important aspects of the analysis plan and its execution unclear. The reason the study was terminated is not reported in the registry record, so the relationship, if any, between termination and missing analyses cannot be determined.
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 |
|---|---|---|---|
| Milademetan | 31 | 86 | 36.0% |
| Trabectedin | 38 | 79 | 48.1% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Milademetan | 43 of 86 |
| Trabectedin | 30 of 89 |
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 |
|---|---|
| Milademetan | 86 of 86 |
| Trabectedin | 78 of 79 |
Most frequent serious adverse event terms
| Event term | Organ system | Milademetan | Trabectedin |
|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | 14 of 86 | 15 of 79 |
| Thrombocytopenia | Blood and lymphatic system disorders | 8 of 86 | 3 of 79 |
| platelet count decreased | Investigations | 3 of 86 | 3 of 79 |
| Acute kidney injury | Renal and urinary disorders | 0 of 86 | 5 of 79 |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | 3 of 86 | 1 of 79 |
| Sepsis | Infections and infestations | 2 of 86 | 1 of 79 |
| Small intestinal obstruction | Gastrointestinal disorders | 2 of 86 | 1 of 79 |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | 1 of 86 | 2 of 79 |
| Febrile neutropenia | Blood and lymphatic system disorders | 0 of 86 | 3 of 79 |
| Neutropenia | Blood and lymphatic system disorders | 0 of 86 | 3 of 79 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Milademetan | Trabectedin |
|---|---|---|---|
| Nausea | Gastrointestinal disorders | 61 of 86 | 46 of 79 |
| Anemia | Blood and lymphatic system disorders | 38 of 86 | 29 of 79 |
| Fatigue | General disorders | 30 of 86 | 26 of 79 |
| Vomiting | Gastrointestinal disorders | 38 of 86 | 15 of 79 |
| Asthenia | General disorders | 28 of 86 | 19 of 79 |
| Diarrhea | Gastrointestinal disorders | 24 of 86 | 21 of 79 |
| Thrombocytopenia | Blood and lymphatic system disorders | 31 of 86 | 11 of 79 |
| Decreased appetite | Metabolism and nutrition disorders | 25 of 86 | 17 of 79 |
| Neutrophil count decreased | Investigations | 21 of 86 | 15 of 79 |
| platelet count decreased | Investigations | 22 of 86 | 9 of 79 |
Serious events with the highest arm-level rate: anaemia (19.0%), thrombocytopenia (9.3%), acute kidney injury (6.3%), platelet count decreased (3.8%), febrile neutropenia (3.8%).
The registry record reports serious adverse events in 31 of 86 participants at risk in the milademetan arm and 38 of 79 at risk in the trabectedin arm. It reports other adverse events in 86 of 86 at risk in the milademetan arm and 78 of 79 at risk in the trabectedin arm. Death counts are 43 and 30, respectively. These are posted counts; the record does not establish that the deaths were caused by study treatment or provide a cause-specific breakdown in these figures.
The listed serious adverse event terms include anaemia at 19.0%, thrombocytopenia at 9.3%, acute kidney injury at 6.3%, platelet count decreased at 3.8%, febrile neutropenia at 3.8%, and neutropenia at 3.8%. These percentages should be read as registry-reported term frequencies, not as a complete description of each participant’s safety experience. The record does not provide the exposure time or additional context needed to compare rates adjusted for time on treatment. It also does not explain the difference between the outcome-specific at-risk counts and the primary outcome arm counts. Counts and term frequencies alone do not establish causation or determine the balance of benefits and harms.
12. What the Result Does, and Does Not, Mean
The registry record supports a descriptive account of the posted primary outcome: the reported median PFS is 3.6 months in the milademetan arm and 2.2 months in the trabectedin arm, with the stated 95% confidence intervals. These estimates concern the analysis population described through the final cut-off date and use blinded independent central review for progression assessment. They do not, by themselves, establish a reliable comparative treatment effect.
The primary endpoint is clinically relevant as a measure of time to documented progression or death, but its interpretation depends on follow-up, event ascertainment, censoring, and the statistical comparison. The registry record does not report the sponsor’s comparative PFS analysis, a hazard ratio, or a test of the primary contrast. A difference in medians is not the same as a formal treatment comparison, and the overlapping intervals do not resolve that question.
The posted secondary binary results provide additional descriptive information, but they are unadjusted recalculations and the registry record does not report a multiplicity plan. They therefore should not be treated as confirmation of the primary outcome. The record also lists other secondary endpoints without corresponding reported analyses in the record described. Missing analyses are not evidence that an outcome was unchanged.
The trial is recorded as terminated, but the reason is not reported in the registry record. No interpretation about the reason for termination, the intended sample size, the study’s statistical power, or any interim decision can be drawn from the status alone. The sample-size illustrations are planning calculations based on observed effects, not observed power and not evidence about the original study design.
These results should not be read as individualized predictions or as medical advice. They describe group-level outcomes in a particular population with unresectable or metastatic dedifferentiated liposarcoma after prior systemic therapy. The registry record alone does not establish how the results apply to people outside that population, nor does it show that either treatment is superior, equivalent, or inferior.
13. Why This Trial Matters Statistically
This study raises several statistical issues common to randomized clinical trials. First, the primary outcome is time-to-event, while the registry record reports arm-specific medians but no sponsor-reported comparative estimate. A median can describe a central point in each outcome distribution, yet a treatment comparison generally requires an analysis that uses the time-to-event information and addresses incomplete follow-up. Without the comparative result, the posted medians are informative but incomplete evidence about the between-arm contrast.
Second, the primary endpoint was assessed by blinded independent central review in an otherwise open-label study. That distinction is relevant because central review can reduce the influence of treatment knowledge on radiographic assessment, while open-label assignment can still matter for other aspects of trial conduct and participant experience. The registry record does not describe additional procedures that might address those concerns.
Third, secondary outcomes offer different views of disease status, but their interpretation depends on how many outcomes were tested and whether analyses were prespecified. The registry record does not report a multiplicity plan or sponsor analyses for the secondary comparisons. Unadjusted recalculations can help describe the posted results but cannot replace a planned confirmatory analysis.
Finally, the study is recorded as terminated, with no reason reported in the registry record. The status makes it especially important not to infer the purpose or consequences of stopping from the posted outcome values alone. Sample-size planning illustrations based on observed effects are not observed power and do not reveal the original design assumptions. Overall, the record illustrates why treatment comparisons require more than a comparison of point estimates: the analysis plan, uncertainty around contrasts, missing-data handling, and multiplicity all affect the strength of conclusions.
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 interpretive limitation is that the registry record reports arm-specific median PFS estimates and confidence intervals but no sponsor-reported comparative time-to-event analysis. There is no reported hazard ratio, confidence interval for a relative effect, or primary comparison test. As a result, the posted information does not quantify the primary between-arm contrast using the trial’s analysis.
Important statistical details are not reported in the registry record. These include the method used to estimate PFS, censoring rules, missing-data procedures, sensitivity analyses, randomization method, allocation concealment, stratification factors, and the original sample-size justification. The registry record also does not describe interim analyses, stopping rules, or the reason for termination. These omissions prevent a complete assessment of how the primary result was planned and analyzed.
Follow-up for the primary endpoint is stated as up to 13 months. The record does not give the distribution of follow-up or explain how participants without a recorded event were handled. The analysis population is described as all randomized participants through the cut-off date, but that statement does not establish that every participant had complete outcome assessment.
The secondary evidence is limited: sponsor analyses are not reported for the listed outcomes, and the recalculated binary comparisons are unadjusted. The registry record does not specify a multiplicity plan. Safety counts are also difficult to compare fully without treatment exposure duration and additional context for the reported at-risk denominators. The reason for the differing safety denominators is not reported in the registry record.
Finally, the findings concern the registry-defined population: participants with unresectable or metastatic dedifferentiated liposarcoma after prior systemic treatment, including anthracycline-based therapy. They should not be generalized beyond that population on the basis of this record alone.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Treatment of Milademetan Versus Trabectedin in Patient With Dedifferentiated Liposarcoma — NCT04979442.
- Registry record last updated 2025-01-14; status last verified 2025-01.
- Results first posted 2025-01-14.
- 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
NCT04979442 is a randomized, multicenter, open-label Phase 3 parallel-group study of milademetan versus trabectedin in participants with unresectable or metastatic dedifferentiated liposarcoma after prior systemic therapy. The recorded enrollment is 175, and the study status is terminated.
The primary outcome is median progression-free survival by blinded independent central review, measured from randomization to radiographic progression or death. The posted medians are 3.6 months for milademetan and 2.2 months for trabectedin, with 95% confidence intervals of 2.1 to 4.1 months and 1.9 to 4.2 months, respectively. A sponsor-reported comparative analysis of the primary outcome is not reported in the registry record.
Disease control rate and objective response rate are posted as secondary outcomes, but the registry record does not report sponsor analyses for the secondary comparisons or a multiplicity plan. The reason for study termination, missing-data procedures, and original sample-size justification are not reported in the registry record.