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 Paclitaxel and Bevacizumab With or Without Emactuzumab in Treating Patients With Platinum-Resistant Ovarian, Fallopian Tube, or Primary Peritoneal Cancer (NCT02923739), a randomized phase 2 study. The primary outcome is Part 1: To Evaluate the Safety of Administration of Paclitaxel, Bevacizumab and Emactuzumab Over 4 Weeks..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02923739 |
| Phase | Phase 2 |
| Condition | Fallopian Tube Adenocarcinoma, Fallopian Tube Clear Cell Adenocarcinoma, Fallopian Tube Endometrioid Adenocarcinoma |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 9 |
| Arms in the primary outcome | n/a |
| Lead sponsor | M.D. Anderson Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
Patients with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer were the intended population. The registry lists multiple cancer types and histologies, including recurrent disease. Nine participants were enrolled, but the record does not give a breakdown of their diagnoses or describe the characteristics of the enrolled participants.
Intervention
The experimental regimen combined paclitaxel and bevacizumab with emactuzumab. In the planned regimen, paclitaxel and bevacizumab were administered on scheduled treatment days in repeating 28-day courses, and emactuzumab was added on days 1 and 15. Treatment was to continue in the absence of disease progression or unacceptable toxicity.
Comparator
The comparator was paclitaxel plus bevacizumab without emactuzumab, also given in repeating 28-day courses. The study was registered as randomized, open-label, and parallel-group. However, the registry lists no participants in either randomized arm, so the posted record does not provide a randomized comparison of the regimens.
Primary question
The primary objective was to evaluate the safety of administering paclitaxel, bevacizumab, and emactuzumab over 4 weeks. Safety was defined as having no required dose alterations during the first cycle because of a related adverse event of grade 3 or greater. The study was terminated early, and the registry says no data were collected for this objective in a way that allowed it to be fully evaluated.
3. Trial Design
This was a phase II, randomized, parallel-group trial with no masking. Its official title describes an induction discontinuation design of emactuzumab following paclitaxel and bevacizumab. The registry also describes a safety lead-in and lists planned induction and randomized arms. The treatment plans distinguished paclitaxel plus bevacizumab from the same regimen with emactuzumab added.
For the planned paclitaxel and bevacizumab regimen, paclitaxel was given intravenously over 60 minutes on days 1, 8, 15, and 22, while bevacizumab was given intravenously over 90 minutes on days 1 and 15. Courses repeated every 28 days in the absence of disease progression or unacceptable toxicity. In the emactuzumab regimen, emactuzumab was given intravenously over 30 minutes on days 1 and 15 in addition to the other drugs.
The registry records enrollment of 9 and status as terminated. The study started on 2017-05-05, and its primary completion date is 2022-09-27. The record attributes early termination to the sponsor and states that the primary objective could not be fully evaluated. Although the study was registered as randomized, the posted participant counts are in the safety lead-in group: 9 at risk in that group and 0 in each listed induction or randomized group. Thus, the registry does not document a completed randomized comparison or give participant flow explaining how enrollment related to the planned trial stages.
The registry identifies M.D. Anderson Cancer Center as the sponsor. It does not report a detailed statistical analysis plan, randomization method, allocation concealment procedure, or a justification for the planned sample size. Those omissions limit how fully the design can be assessed from the registry record.
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 | Safety Lead-In | Induction Arm | Randomized Arm 1 | Randomized Arm 2 |
|---|---|---|---|---|
| STARTED | 9 | 0 | 0 | 0 |
| COMPLETED | 4 | 0 | 0 | 0 |
| NOT COMPLETED | 5 | 0 | 0 | 0 |
Reasons for not completing this period:
| Reason | Safety Lead-In | Induction Arm | Randomized Arm 1 | Randomized Arm 2 |
|---|---|---|---|---|
| Withdrawal by Subject | 2 | 0 | 0 | 0 |
| Failed Screening | 3 | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: The study was terminated early by the sponsor and no data was collected for the study objective was unable to be fully evaluated.
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.
Population: The study was terminated early by the sponsor
| Characteristic | Unit | Safety Lead-In | Induction Arm | Randomized Arm 1 | Randomized Arm 2 | Total |
|---|---|---|---|---|---|---|
| Participants analysed | 9 | 0 | 0 | 0 | 9 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 5 | 0 | 0 | 0 | 5 |
| Age, Categorical: >=65 years | Participants | 4 | 0 | 0 | 0 | 4 |
| Age, Continuous | Years | 66 (51 to 77) | — | — | — | 66 (51 to 77) |
| Sex: Female, Male: Female | Participants | 9 | 0 | 0 | 0 | 9 |
| Sex: Female, Male: Male | Participants | 0 | 0 | 0 | 0 | 0 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 3 | 0 | 0 | 0 | 3 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 6 | 0 | 0 | 0 | 6 |
| 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 | 0 | 0 | 0 |
| 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 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 7 | 0 | 0 | 0 | 7 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 2 | 0 | 0 | 0 | 2 |
| Region of Enrollment: United States | participants | 9 | — | — | — | 9 |
6. Primary Endpoint
The registry lists the primary outcome as Part 1: To Evaluate the Safety of Administration of Paclitaxel, Bevacizumab and Emactuzumab Over 4 Weeks., measured over Up to 4 weeks.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint focused on whether the three-drug regimen could be administered through the first treatment cycle without dose alterations required because of a related adverse event of grade 3 or greater. The registry defines the observation period as up to 4 weeks. This is a safety and treatment-delivery measure: it combines the occurrence and attribution of adverse events with whether those events required a change to the planned dosing.
Such an endpoint can be useful in an early-phase study because it asks whether a regimen can be delivered as planned during an initial period of treatment. It is not, by itself, a measure of tumor response, survival, or comparative benefit. Nor does the definition count every adverse event as an endpoint event: the event must be related, reach the stated severity threshold, and lead to a required dose alteration.
The registry states that the sponsor terminated the study early and that no data were collected for the study objective in a way that allowed it to be fully evaluated. Consequently, the record does not report a primary endpoint result or an estimate of the frequency of participants meeting the safety definition. The adverse-event entries that are posted do not substitute for a complete assessment of this endpoint, because the registry does not connect those entries to required dose alterations during the first cycle.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive point is that the registered primary safety question has no evaluable result in the registry record. The endpoint required assessing whether related adverse events of grade 3 or greater caused required dose alterations during the first 4 weeks. The record says the study was terminated early and that the objective could not be fully evaluated. It therefore does not support an estimate of the proportion meeting the endpoint, a confidence interval for that proportion, or a treatment comparison for this outcome.
The registry does include adverse-event information for the safety lead-in group. It lists 9 participants at risk, 4 with serious adverse events, 4 with other adverse events, and 5 deaths. These figures describe the posted safety entries, but they do not establish whether the primary endpoint was met. In particular, the registry does not say that every reported event was related, that it occurred during the endpoint window, or that it required a dose alteration. The listed serious adverse event term CPK increased is reported at 44.4%. That term-level entry should be read as a reported safety detail, not as a measure of the primary endpoint or a comparison between regimens.
The distinction between the planned trial and the posted record matters. The study was registered as randomized and intended to compare paclitaxel plus bevacizumab with the same regimen plus emactuzumab. Yet the listed participant counts show no participants in the induction or randomized groups. The registry therefore does not provide outcome data from the intended randomized comparison. No relative effect, uncertainty estimate, or treatment-group contrast can be inferred from the record.
The small recorded enrollment and early termination also leave the scope of the safety information limited. There are no posted analyses, and the registry does not report how participants were selected for the safety lead-in, whether all enrolled participants contributed to each outcome, or how missing outcome information was handled. The recorded counts are descriptive entries rather than a basis for claims about comparative safety or efficacy. They should not be interpreted as showing that either regimen is safer or more effective.
Overall, the registry establishes that the study was designed to assess early-cycle safety, planned a randomized comparison, enrolled 9 participants, and terminated before the primary objective could be fully evaluated. It documents a limited set of safety counts, but not the primary endpoint result. The posted record therefore describes an incomplete trial rather than a completed test of the treatment question.
9. Statistical Methods Explained
The record identifies the trial as phase II, randomized, unmasked, and parallel-group. These design labels describe the intended structure: participants were to be assigned to treatment groups, the regimens were organized as separate groups, and masking was not used. They do not establish that the planned randomization was completed. In this record, the listed counts are 0 for the induction and randomized groups, while the safety lead-in group has 9 participants at risk. The registry does not report the randomization sequence, allocation concealment, or the number assigned to each intended randomized regimen.
What the primary endpoint captures
The primary endpoint is a binary safety-and-delivery criterion: whether a participant had no required dose alteration during the first cycle because of a related adverse event of grade 3 or greater. A binary endpoint classifies each participant according to whether the defined condition was met. To estimate its frequency, the record would need both a clearly defined analysis population and the number of participants meeting the criterion. Neither an endpoint count nor a rate is reported. The registry instead states that the objective could not be fully evaluated after early termination.
How to think about uncertainty
In general, a confidence interval describes the range of effect estimates reasonably compatible with the observed data under the analysis assumptions. It communicates precision as well as the central estimate; a wide interval indicates less precision, while a narrow interval indicates more. Here, the primary endpoint estimate is not reported, so there is no registry-based confidence interval to interpret. The posted record also reports no analyses. An absence of an estimate is not the same as an estimate showing no effect or no safety concern.
General statistical methods can only be applied after an outcome and analysis population are defined and the required observations are available. For example, a comparison of the two planned regimens would require outcome data from both treatment groups and a stated method for comparing them. The registry provides neither a completed randomized-group comparison nor an analysis result. No treatment effect or precision measure should be inferred from the overall enrollment or the safety lead-in counts.
Why planning estimates are different
A sample-size calculation is a planning exercise based on assumptions about the outcome, the target effect, allocation, and statistical error. It is not an observed result and does not establish that a study had a particular probability of detecting an effect after the fact. The registry does not report a trial-specific sample-size justification. It describes a generic planning illustration based on an observed effect and specified assumptions, but the primary endpoint was not fully evaluated and no single treatment comparison is available from the posted results. There is therefore no observed primary effect in the registry to support interpreting such a planning exercise as a finding from this trial. Any planning illustration must be understood as hypothetical, not as observed power.
Power is a design concept: in general, it is the probability that a specified analysis will detect a specified effect under its assumptions. It depends on the assumed effect and variability, the sample size, and the testing procedure. Power calculated from planning assumptions is not a measure of how convincing the trial's eventual result is. In this case, early termination and the absence of a reported primary endpoint result prevent a meaningful assessment of the planned comparison's precision or evidential strength.
Over-dispersion and outcome structure
In general, over-dispersion occurs when observed variability in count data exceeds what a chosen statistical model expects. It can make standard errors too small if a model assumes less variability than the data contain. That concept is relevant when analyzing count outcomes, but the registry does not provide a count-based treatment comparison or a model specification for this trial. It would be inappropriate to claim that over-dispersion was present or that a particular correction was used.
The registry lists two outcome measures and no posted analyses. It does not describe a statistical model, test procedure, covariate adjustment, confidence interval method, or rules for handling repeated measurements. It also does not report interim analysis procedures or stopping boundaries. The early termination is recorded, but the statistical rationale or decision process is not described. These details are not reported in the registry record.
In general, a useful analysis description specifies the outcome definition, analysis population, handling of missing data, model or test, and uncertainty estimates. Those details help readers understand which observations support a result and what assumptions connect the data to the estimate. Here, the registry has a clear definition for the primary safety endpoint but no evaluable primary result and no detailed analysis plan. As a result, the statistical interpretation must remain descriptive and limited to the fields actually reported.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists two outcome measures and no analyses. It does not report a multiplicity strategy, such as a hierarchy of outcomes or an adjustment for testing several hypotheses. Multiplicity concerns arise when multiple outcomes or comparisons are tested, because the chance of at least one apparently notable result can increase across tests. Without posted analyses or an analysis plan, it is not possible to determine how the trial would have addressed that issue. No adjustment should be assumed.
The registry does not report an analysis population for the primary endpoint, nor does it describe intention-to-treat, per-protocol, or safety-population rules. In general, these populations can differ in which participants are included and can therefore answer different questions. The participant counts identify 9 at risk in the safety lead-in group and 0 in the listed induction and randomized groups, but the record does not explain participant flow or how these group labels relate to each outcome.
Missing-data methods are not reported in the registry record. The record does not say how unavailable endpoint information, treatment discontinuation, or incomplete follow-up would have been handled in an analysis. It also does not report whether outcome data were missing for particular participants or reasons for missingness. Since the primary objective was not fully evaluated, these omissions prevent assessment of how missing observations might affect a primary endpoint estimate.
Early termination and absent analysis results mean that no multiplicity-adjusted inference, missing-data sensitivity analysis, or population-specific estimate can be assessed. These are gaps in the registry information, not evidence that a particular method was or was not used outside the record.
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 |
|---|---|---|---|
| Safety Lead-In | 4 | 9 | 44.4% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Safety Lead-In | 5 of 9 |
| Induction Arm | 0 of 0 |
| Randomized Arm 1 | 0 of 0 |
| Randomized Arm 2 | 0 of 0 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 1% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Safety Lead-In | 4 of 9 |
| Induction Arm | 0 of 0 |
| Randomized Arm 1 | 0 of 0 |
| Randomized Arm 2 | 0 of 0 |
Most frequent serious adverse event terms
| Event term | Organ system | Safety Lead-In | Induction Arm | Randomized Arm 1 | Randomized Arm 2 |
|---|---|---|---|---|---|
| CPK increased | Investigations | 4 of 9 | 0 of 0 | 0 of 0 | 0 of 0 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Safety Lead-In | Induction Arm | Randomized Arm 1 | Randomized Arm 2 |
|---|---|---|---|---|---|
| LDH increased | Investigations | 4 of 9 | 0 of 0 | 0 of 0 | 0 of 0 |
| Anemia | Blood and lymphatic system disorders | 4 of 9 | 0 of 0 | 0 of 0 | 0 of 0 |
| SGOT increased | Investigations | 4 of 9 | 0 of 0 | 0 of 0 | 0 of 0 |
| CPK increased | Investigations | 4 of 9 | 0 of 0 | 0 of 0 | 0 of 0 |
| Fatigue | General disorders | 4 of 9 | 0 of 0 | 0 of 0 | 0 of 0 |
| Hypertension | Vascular disorders | 4 of 9 | 0 of 0 | 0 of 0 | 0 of 0 |
| Diarrhea | Gastrointestinal disorders | 2 of 9 | 0 of 0 | 0 of 0 | 0 of 0 |
| Edema - face | General disorders | 2 of 9 | 0 of 0 | 0 of 0 | 0 of 0 |
| Mucositis oral | Gastrointestinal disorders | 2 of 9 | 0 of 0 | 0 of 0 | 0 of 0 |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | 2 of 9 | 0 of 0 | 0 of 0 | 0 of 0 |
Serious events with the highest arm-level rate: cpk increased (44.4%).
The safety entries are reported for the safety lead-in group, with 9 participants at risk. The record lists 4 participants with serious adverse events and 4 with other adverse events, as well as 5 deaths. These are distinct registry categories; the information does not establish whether the participants represented in one category also appear in another. The counts should not be added together or treated as mutually exclusive groups.
CPK increased is the most frequent serious adverse event term listed, at 44.4%. The registry does not give enough detail in this entry to determine timing, attribution, duration, or the clinical circumstances of the event. It also does not connect the term to a required dose alteration under the primary endpoint definition.
These figures are descriptive safety information from the posted record. They are not a comparison between the randomized regimens: the registry lists 0 participants in each randomized group. Nor do the counts establish whether an event was caused by treatment. The death count should not be interpreted as a treatment effect or attributed to a particular regimen from these entries alone. The record does not provide additional context needed to make those judgments.
12. What the Result Does, and Does Not, Mean
The registry record documents a planned early-phase study of paclitaxel and bevacizumab, with or without emactuzumab, in platinum-resistant ovarian, fallopian tube, or primary peritoneal cancer. It also documents that the study was terminated early and that its primary safety objective could not be fully evaluated. That is the main conclusion that can be drawn about the trial's result: the registered primary question remains unanswered in the posted record.
The safety entries give descriptive counts for the safety lead-in group, including serious adverse events, other adverse events, deaths, and a listed serious event term. These records may describe what was entered for that group, but they do not provide a complete analysis of the primary endpoint. The registry does not report whether events were related, whether they prompted dose changes, or whether the first-cycle endpoint was met. The figures therefore should not be interpreted as a definitive estimate of the regimen's safety.
The record also does not show a completed randomized comparison. Although randomization was part of the design, no participants are listed in the randomized arms. There is no treatment-group result, effect estimate, or measure of uncertainty from which to compare the regimens. Accordingly, the posted data do not establish whether adding emactuzumab changed safety, tumor response, or any other outcome.
These limitations do not mean that the regimens have identical effects, or that they have no effects. They mean that the registry record does not contain the information needed to estimate or compare those effects. A null finding would require an analyzed outcome and uncertainty assessment; neither is reported for the primary objective. The appropriate reading is therefore that the trial was incomplete for its stated primary question, with some safety counts posted but no evaluable primary result or randomized treatment contrast.
13. Why This Trial Matters Statistically
This trial illustrates why design labels and results need to be read separately. A registry can describe a randomized, parallel-group design and detailed treatment schedules, but those plans do not themselves constitute evidence from a completed comparison. In this record, the randomized groups have no listed participants, while a safety lead-in group contains the recorded enrollment. Keeping planned design distinct from achieved enrollment prevents an intended comparison from being mistaken for an observed one.
It also shows that endpoint definitions matter. The primary question was not simply whether adverse events occurred. It required assessing whether a related event of grade 3 or greater led to a dose alteration during the first cycle. The posted safety counts do not directly answer that question. Without linking severity, attribution, timing, and dose changes, general adverse-event totals cannot be substituted for the defined endpoint.
Early termination is statistically important because it can leave a trial without the observations needed to estimate its planned outcomes. Here the registry explicitly says the primary objective could not be fully evaluated, and it provides no analysis results. That makes precision, treatment effects, and the intended randomized contrast unavailable for interpretation. The record is consequently informative about the limits of the evidence as well as about the planned study.
Finally, the record highlights the difference between a safety description and a comparative inference. Counts from one group can be reported without supporting conclusions about differences between regimens. Transparent reporting of enrollment, endpoint results, analysis populations, and uncertainty would be needed to move from descriptive safety information to a statistical comparison.
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 most consequential limitation is that the study was terminated early and the registry states that the primary objective could not be fully evaluated. No primary endpoint result is reported. The posted record therefore cannot answer the endpoint question as defined, even though it describes the intended treatment schedules and safety criterion.
The registry lists 9 participants at risk in the safety lead-in group and 0 in each listed induction and randomized group. It does not explain participant flow or report outcomes by the two planned randomized regimens. Thus, the intended comparison cannot be assessed from the posted results. The enrollment count alone does not reveal which participants received which treatment or how many contributed information to each outcome.
The safety entries are limited in context. The record lists serious and other adverse-event counts, deaths, and a serious event term, but does not report timing, attribution, dose changes, or the relationship of these entries to the primary endpoint. The safety information therefore cannot be treated as a full endpoint analysis or as a comparative safety assessment.
Additional statistical details are not reported in the registry record, including a trial-specific sample-size justification, a detailed analysis plan, analysis populations, missing-data methods, multiplicity procedures, and interim analysis procedures. The record also does not describe the reason or statistical process for early termination. These gaps limit assessment of design assumptions, possible bias, and the precision of any intended analysis. They do not establish that a particular method was absent outside the registry; they indicate that the posted record does not document it.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Paclitaxel and Bevacizumab With or Without Emactuzumab in Treating Patients With Platinum-Resistant Ovarian, Fallopian Tube, or Primary Peritoneal Cancer — NCT02923739.
- Registry record last updated 2024-03-13; status last verified 2024-03.
- Results first posted 2023-10-10.
- 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
NCT02923739 was a phase II randomized, unmasked, parallel-group trial sponsored by M.D. Anderson Cancer Center. It planned to evaluate paclitaxel and bevacizumab with or without emactuzumab in patients with platinum-resistant ovarian, fallopian tube, or primary peritoneal cancer. The primary objective was a first-cycle safety measure based on whether related adverse events of grade 3 or greater required dose alterations during 4 weeks.
The registry records enrollment of 9 and status as terminated. It reports 9 participants at risk in the safety lead-in group and 0 in the listed induction and randomized groups. The sponsor terminated the study early, and the primary objective could not be fully evaluated. Safety entries include 4 participants with serious adverse events, 4 with other adverse events, and 5 deaths; CPK increased is listed as a serious event term at 44.4%.
No analyses are posted, and the registry does not report a primary endpoint result or randomized comparison. The record supports a description of the intended design and limited safety entries, but not an estimate of the primary safety outcome or a comparison of the planned regimens.