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 Study of ENMD-2076 in Patients With Advanced/Metastatic Soft Tissue Sarcoma (NCT01719744), a na phase 2 study. The primary outcome is 6-month Progression-free Survival Rate (PFS).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01719744 |
| Phase | Phase 2 |
| Condition | Advanced, Metastatic, Soft Tissue Sarcoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 25 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University Health Network, Toronto |
| Results posted | Yes |
2. Clinical Question
Population
Patients with advanced or metastatic soft tissue sarcoma. The registry record lists these conditions and describes the study population in those terms; further eligibility details are not reported in the registry record.
Intervention
ENMD-2076 capsules, 275 mg once daily by mouth. The study is described as a phase 2 investigation of this drug in the stated population.
Comparator
No concurrent comparator is listed. The design is single-group, allocation is recorded as NA, and masking is recorded as none. This is not a randomized comparison, so the study does not provide a like-for-like control group.
Primary question
The primary endpoint asks how many participants had no disease progression at six months after starting study treatment, with progression or death defining the endpoint event. The registry lists a 95% confidence interval as the dispersion measure, but its numerical estimate and interval are not reported in the registry record.
In practical terms, the study was set up to describe a six-month progression-free survival outcome among people receiving ENMD-2076, alongside safety and tolerability. That question is distinct from whether the drug improves outcomes compared with another treatment or with no treatment: without a control group, the registry record cannot make a direct comparative estimate.
The record also lists objective response rate and adverse events as secondary outcomes. It does not give numerical results for objective response rate in the registry record for this analysis. The safety summary reports participant counts for serious and other adverse events, and lists selected serious adverse-event terms with percentages. These observations describe the treated group; they do not by themselves establish that an event was caused by treatment.
3. Trial Design
This was a phase 2, single-group study sponsored by University Health Network, Toronto. The registry record gives planned enrollment as 25 and status as completed. The recorded start is 2013-01, and primary completion is 2015-12. The intervention was ENMD-2076, administered as 275 mg capsules once daily by mouth.
The design has no listed allocation process or masking: allocation is recorded as NA and masking as none. With a single group and no concurrent control, results can describe outcomes among participants who received ENMD-2076, but the design cannot isolate the effect of treatment from other influences on outcome. Differences in disease course, participant characteristics, supportive care, and the timing of assessment cannot be separated from treatment effects through a randomized comparison in this study.
The primary outcome is the six-month progression-free survival rate, represented in the registry as the number of participants with no progression at six months. Its stated timeframe runs from the start of study treatment until disease progression or death, whichever occurs first, up to six months. The record also lists objective response rate and the number and severity of adverse events per participant as secondary outcomes.
The registry lists 3 outcome measures and 0 analyses. The record does not report a detailed statistical analysis plan, the analysis population, the method used to handle censoring, or the procedures used to calculate the endpoint estimate and confidence interval. Sample-size justification is not reported in the registry record. Consequently, the stated enrollment and endpoint identify the study’s scope, but do not establish whether the study was designed to detect a particular effect or how the planned analysis was to be conducted.
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 | ENMD-2076 |
|---|---|
| STARTED | 25 |
| COMPLETED | 25 |
| NOT COMPLETED | 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 | ENMD-2076 |
|---|---|---|
| Participants analysed | 25 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 21 |
| Age, Categorical: >=65 years | Participants | 4 |
| Age, Continuous | years | 54 (22 to 73) |
| Sex: Female, Male: Female | Participants | 18 |
| Sex: Female, Male: Male | Participants | 7 |
| Region of Enrollment: Canada | participants | 25 |
6. Primary Endpoint
The registry lists the primary outcome as 6-month Progression-free Survival Rate (PFS), measured over From start of study treatment until disease progression or death, whichever occurs first, up to 6 months..
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the six-month progression-free survival rate. The registry describes it as the number of patients with no progression of disease at six months. The endpoint timeframe begins at the start of study treatment and ends at progression or death, whichever occurs first, with follow-up limited to up to six months.
Progression-free survival is used in clinical studies to capture time without either disease progression or death. It can reflect disease control over a defined period and includes death as an event, rather than counting progression alone. For this record, the primary outcome is expressed as a participant count, and a 95% confidence interval is specified as the measure of dispersion. The numerical count and interval are not reported in the registry record, so the observed six-month outcome and its precision cannot be described from these registry entries.
When progression-free survival is analyzed as a time-to-event outcome, participants may have different follow-up durations, and some may not have experienced an event by the time observation ends. Statistical methods generally account for such incomplete event times through censoring. The registry entries do not report how censoring was defined or handled, whether the endpoint was estimated using a time-to-event method, or how participants with incomplete six-month follow-up were treated. Those details matter for interpreting a rate or estimate, particularly if follow-up was not complete for every participant.
The stated endpoint is clinically interpretable as an outcome over a defined period, but its meaning is limited by the absent result and analysis details. It is not possible to assess the observed rate, the width of its confidence interval, or the extent to which the estimate represents all enrolled participants.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry identifies the intended primary outcome but does not provide its numerical result. Although it specifies a six-month progression-free survival rate and a 95% confidence interval, neither the count of participants without progression nor the interval limits are reported in the registry record. Therefore, the magnitude of the observed outcome and its statistical precision cannot be assessed from the registry entries available for this analysis.
This absence is important for interpretation. An endpoint definition tells readers what was to be measured, not what was observed. Without the estimate and interval, there is no basis for describing whether the observed six-month result was high or low, or whether the estimate is precise or uncertain. No effect comparison can be made because the design has no comparator group. The registry records a single-group study, allocation NA, and no masking; it is explicitly not a randomized comparison.
The enrollment is listed as 25, but enrollment alone does not reveal how many participants contributed to the primary endpoint, how many had complete follow-up, or how missing observations were treated. These details are not reported in the registry record. The listed number of outcome measures is 3 and the number of analyses is 0, but the registry entries do not include an analysis population or an account of the statistical procedures actually used.
The safety summary reports that 7 of 25 participants had serious adverse events and that 25 of 25 had other adverse events. The record lists dehydration, diarrhea, vomiting, and lung infection at 8.0% each among the top serious terms, and agitation and increased alanine aminotransferase at 4.0% each. These summaries describe events recorded in the treated group. They do not show whether the events were judged related to treatment, how events were collected, or whether participants had more than one event. Deaths are not reported in the registry record.
The sponsor’s own analysis takes precedence if any recalculated result conflicts with that analysis. No numerical primary-outcome result or sponsor analysis is included in the registry record, so no such comparison can be made. Any sample-size figures associated with planning are illustrations rather than observed power; they cannot substitute for the missing endpoint estimate, its uncertainty, or a prespecified design justification.
Overall, the record supports a description of the study’s question, intervention, and selected safety counts, but not an efficacy conclusion. A single-group result, even if fully reported, would describe outcomes during treatment without establishing what would have happened to comparable participants under a different treatment or without the study intervention.
9. Statistical Methods Explained
The primary endpoint is stated as a participant count: the number of patients with no progression at six months. The registry also names a 95% confidence interval as the dispersion measure. Those are endpoint specifications, not a numerical result. The count and interval are not reported in the registry record, and the statistical method used to produce them is not reported in the registry record.
What does a confidence interval communicate?
In general, a confidence interval expresses uncertainty around an estimate using a method with a stated long-run coverage property. A narrow interval indicates greater statistical precision under the assumptions of the method; a wide interval indicates less precision. The interval does not guarantee that the true value lies within its particular limits, and it does not remove bias from study design or measurement. In this study, the record specifies a 95% confidence interval, but its limits are unavailable. Precision therefore cannot be judged.
Why does the single-group design matter?
A single-group design observes outcomes in participants receiving the study intervention without a concurrent control group. It can describe what occurred in that group, but it cannot directly estimate a treatment contrast against an alternative. A change or outcome during follow-up may reflect treatment, underlying disease, other care, participant selection, measurement, or other factors. Random allocation in a controlled design can help balance factors between groups; this study has no such comparison. The registry records allocation as NA and masking as none.
How should a six-month progression-free endpoint be analyzed?
In general, progression-free survival is a time-to-event outcome: the event is progression or death, and the time origin is the start of treatment. Participants who have not had an event by the end of their observed follow-up may be censored, meaning their event time is only known to extend beyond their last observation. Time-to-event methods use the available event-time information while accounting for such censoring, subject to assumptions about how censoring relates to the chance of an event. Alternatively, a fixed-time participant count needs a clear rule for participants whose six-month status is unknown. The registry record does not report the censoring rules, the analysis method, or the handling of incomplete follow-up.
These distinctions affect what a reported six-month rate represents. A simple count among participants with known status may differ in interpretation from an estimated time-to-event probability that uses follow-up before the six-month point. The endpoint description alone does not establish which approach was used. Since neither the estimate nor analysis details are reported in the registry record, readers cannot determine how incomplete observation influenced the result.
What can the safety counts establish?
Event counts describe how many participants were recorded as affected in the stated categories. The registry lists 7 of 25 participants with serious adverse events and 25 of 25 with other adverse events. These categories do not, by themselves, identify causation. General safety interpretation also depends on event definitions, collection procedures, timing, severity grading, attribution, and whether repeated events in one participant are counted separately. Those details are not reported in the registry record. The serious-event term percentages should be read as the listed percentages for those terms, not added together to obtain a participant-level total.
What do planning assumptions mean?
The planning illustration is described as using 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 the observed effect. Those assumptions do not describe this study’s single-group design, and no resulting sample-size figures or observed effect are included in the registry record for this analysis. Any such planning quantities are illustrations, not observed power and not a report of the trial’s own design rationale. The trial’s sample-size justification is not reported in the registry record. The stated planning framework should therefore not be mistaken for evidence that the study enrolled enough participants to answer its primary question with a particular precision.
The record lists 3 outcome measures but 0 analyses. It does not report a multiplicity strategy, a prespecified hierarchy among outcomes, an analysis population, an intention-to-treat definition, or a statistical plan. Without these details, readers cannot determine how outcome selection, exclusions, missing data, or multiple statistical tests were addressed.
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.
| Arm | Participants | Events | Observed % | Clopper-Pearson 95% CI | Wilson 95% CI | Bayesian 95% credible interval |
|---|---|---|---|---|---|---|
| ENMD-2076 | 25 | 12 | 48.0% | 27.8% to 68.7% | 30.0% to 66.5% | 29.9% to 66.6% |
R code and output
arm <- c("ENMD-2076")
n <- c(25)
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 ENMD-2076 25 12 48.0 27.8 68.7 30.0 66.5 29.9 66.611. Multiplicity, Missing Data and Analysis Populations
The registry lists 3 outcome measures: the primary six-month progression-free survival outcome, objective response rate, and the number and severity of adverse events per participant. The record lists 0 analyses. A detailed statistical analysis plan, including any testing hierarchy or adjustment for multiple comparisons, is not reported in the registry record.
Multiplicity matters when several outcomes or statistical tests are examined. In general, repeated testing can increase the chance of finding an apparently noteworthy result by chance unless the analysis plan accounts for the testing strategy. It is not possible to determine from the registry entries whether multiplicity was relevant to the reported analyses, because analyses and their results are not reported. The record does not provide numerical results for the primary endpoint or objective response rate.
The analysis population is not reported in the registry record. It is therefore unclear which enrolled participants were included in each endpoint analysis, or whether any participants were excluded after enrollment. The handling of missing outcome data, incomplete follow-up, and withdrawals is also not reported in the registry record. For a six-month endpoint, these details can affect how many participants have an ascertainable outcome and how a rate is estimated.
The registry entries do not state whether the primary analysis was intention-to-treat, per-protocol, or based on another defined population. They also do not describe how protocol deviations were handled. These omissions limit reproducibility and make it difficult to assess whether the analyzed group corresponds to the participants originally enrolled.
12. 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 |
|---|---|---|---|
| ENMD-2076 | 7 | 25 | 28.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| ENMD-2076 | 25 of 25 |
Most frequent serious adverse event terms
| Event term | Organ system | ENMD-2076 |
|---|---|---|
| Dehydration | Metabolism and nutrition disorders | 2 of 25 |
| Diarrhea | Gastrointestinal disorders | 2 of 25 |
| Vomiting | Gastrointestinal disorders | 2 of 25 |
| Lung infection | Infections and infestations | 2 of 25 |
| Agitation | Psychiatric disorders | 1 of 25 |
| Alanine aminotransferase increased | Investigations | 1 of 25 |
| Aspartate aminotransferase increased | Investigations | 1 of 25 |
| Colitis | Gastrointestinal disorders | 1 of 25 |
| Confusion | Psychiatric disorders | 1 of 25 |
| Depressed level of consciousness | Nervous system disorders | 1 of 25 |
Most frequent non-serious adverse event terms
| Event term | Organ system | ENMD-2076 |
|---|---|---|
| HYPERTENSION | Vascular disorders | 19 of 25 |
| HYPOALBUMINEMIA | Metabolism and nutrition disorders | 17 of 25 |
| FATIGUE | General disorders | 16 of 25 |
| LYMPHOCYTE COUNT DECREASED | Investigations | 14 of 25 |
| NAUSEA | Gastrointestinal disorders | 13 of 25 |
| PROTEINURIA | Renal and urinary disorders | 13 of 25 |
| CONSTIPATION | Gastrointestinal disorders | 12 of 25 |
| DIARRHEA | Gastrointestinal disorders | 11 of 25 |
| ALANINE AMINOTRANSFERASE INCREASED | Investigations | 11 of 25 |
| PLATELET COUNT DECREASED | Investigations | 11 of 25 |
Serious events with the highest arm-level rate: dehydration (8.0%), diarrhea (8.0%), vomiting (8.0%), lung infection (8.0%), agitation (4.0%).
The safety summary lists 7 of 25 participants in the ENMD-2076 group as having serious adverse events, and 25 of 25 as having other adverse events. These are participant-level group counts as recorded. A participant can experience more than one event, so participant counts should not be interpreted as a count of distinct episodes.
The top serious-event terms are listed as dehydration at 8.0%, diarrhea at 8.0%, vomiting at 8.0%, lung infection at 8.0%, agitation at 4.0%, and alanine aminotransferase increased at 4.0%. These percentages are the registry’s listed figures for the terms. They should not be summed to infer the proportion of participants with any serious event, because the record does not say whether participants could appear under multiple terms. Nor do the listed terms establish that an event was caused by ENMD-2076.
The record does not report the number of deaths. It also does not provide event timing, relatedness assessments, severity grading details, or the methods used to collect and classify adverse events. Those omissions limit comparisons with other studies and prevent a fuller account of the safety profile. The figures describe recorded events in the study group, not a comparative safety effect.
13. What the Result Does, and Does Not, Mean
The registry record establishes that a phase 2 study examined ENMD-2076 in patients with advanced or metastatic soft tissue sarcoma. It identifies the planned primary endpoint as the number of participants without progression at six months and specifies a 95% confidence interval. The record also gives selected adverse-event counts and lists objective response rate as a secondary outcome.
What it does not establish is the numerical primary result. The six-month count and confidence interval are not reported in the registry record. Objective response results are not reported in the registry record, and the numerical primary outcome cannot be reconstructed from the study design or safety information. Without the endpoint estimate, readers cannot assess its magnitude or statistical precision.
The single-group design also limits what any outcome could mean. There is no concurrent comparator, so outcomes among participants receiving ENMD-2076 cannot be directly contrasted with outcomes under another treatment. Even a clearly estimated progression-free survival rate would be descriptive of this treated group, not a randomized estimate of a treatment effect. The design alone does not establish that an observed outcome was caused by the intervention.
The serious and other adverse-event counts indicate that events were recorded among participants, while the listed serious-event terms provide additional descriptive detail. They do not establish causality, comparative risk, or the full timing and severity pattern. Deaths are not reported in the registry record.
The record also leaves important analytic details unreported, including the analysis population, missing-data handling, censoring method, and statistical plan. Those details affect how the endpoint and its uncertainty should be interpreted. The appropriate reading is therefore limited: the record describes the study design and selected safety observations, but does not report the numerical primary efficacy result or enough analysis information to support a comparative conclusion.
14. Why This Trial Matters Statistically
This trial is statistically informative as an example of the distinction between an endpoint specification and an endpoint result. The registry names a six-month progression-free survival outcome and a 95% confidence interval, but does not report either the estimate or interval limits. Knowing what investigators intended to measure is not enough to judge the observed outcome or its precision.
The single-group design also makes the interpretation of results especially important. Descriptive outcomes can characterize participants receiving ENMD-2076, but no concurrent control group is available to separate treatment-associated outcomes from the natural course of disease or other influences. The record’s allocation and masking entries reinforce that this was not a randomized, masked comparison.
The safety summary illustrates a separate issue: event counts, event terms, and causality are not interchangeable. Participant counts and listed percentages can describe recorded events, but without details on relatedness, collection, timing, and repeated events, they cannot establish that the intervention caused an event or quantify a comparative risk.
Finally, the registry’s lack of a reported analysis population, missing-data approach, censoring method, and statistical plan limits reproducibility. These are not merely technical details; they help readers understand which participants inform an estimate and how uncertainty is calculated. The record lists 3 outcome measures and 0 analyses, leaving the reader without numerical outcome analyses to evaluate. For clinical research interpretation, that gap illustrates why complete reporting of estimates, uncertainty, and analytic methods is essential alongside a clear study question.
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 a numerical result for the primary six-month progression-free survival endpoint or its specified 95% confidence interval. Without these, neither the observed endpoint nor its precision can be assessed. Numerical objective response results are also not reported in the registry record.
The study has a single-group design and no concurrent comparator. As a result, the record cannot support a like-for-like comparison or distinguish outcomes associated with treatment from other possible influences. Allocation is listed as NA and masking as none.
Several analytic details are absent. The registry does not report the analysis population, treatment of missing data, handling of incomplete six-month follow-up, censoring rules, or a detailed statistical analysis plan. It also does not report a sample-size justification. These omissions make it difficult to evaluate the endpoint methods, participant inclusion, and uncertainty.
The safety summary gives group counts and selected serious-event terms, but does not report deaths, event timing, attribution, or full adverse-event collection and classification procedures. The listed adverse-event information is therefore descriptive and incomplete for assessing causality or comparative risk.
Finally, the record lists 3 outcome measures and 0 analyses. With no numerical outcome analyses reported in the registry record for this analysis, the study’s endpoint findings cannot be independently interpreted or compared with its stated objectives.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: Study of ENMD-2076 in Patients With Advanced/Metastatic Soft Tissue Sarcoma — NCT01719744.
- Registry record last updated 2023-08-01; status last verified 2023-07.
- Results first posted 2017-11-07.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
19. Record Summary
The registry record describes a completed phase 2, single-group study of oral ENMD-2076 in patients with advanced or metastatic soft tissue sarcoma. Planned enrollment was 25. Treatment was listed as 275 mg capsules once daily by mouth. The recorded study start was 2013-01, with primary completion in 2015-12.
The primary endpoint was the number of participants with no disease progression at six months, with progression or death defining the endpoint event and a 95% confidence interval specified. The numerical primary result and interval are not reported in the registry record. Objective response rate and adverse events were listed as secondary outcomes; numerical objective response results are not reported in the registry record.
The safety summary lists serious adverse events in 7 of 25 participants and other adverse events in 25 of 25. It also lists selected serious-event terms with percentages. The record does not report deaths. It lists 3 outcome measures and 0 analyses, and does not report the analysis population, missing-data methods, censoring approach, or a detailed statistical plan.