AMG 706 in Treating Patients With Persistent or Recurrent Ovarian Epithelial Cancer, Fallopian Tube Cancer, or Primary Peritoneal Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for AMG 706 in Treating Patients With Persistent or Recurrent Ovarian Epithelial Cancer, Fallopian Tube Cancer, or Primary Peritoneal Cancer (NCT00574951), a na phase 2 study. The primary outcome is Number of Patients With Objective Tumor Response Rate (Complete Response [CR] or Partial Response [PR]) Using RECIST Version 1.0.

2. Clinical Question

The clinical question was whether an objective tumor response was observed among eligible and treated participants receiving AMG 706. The study was a phase II evaluation in patients with persistent or recurrent disease across the listed cancer types. This is a treatment-group question, not a comparative-effect question: with no control group, the record cannot show how outcomes would have differed under another treatment or without AMG 706.

The response definition was based on changes in measurable disease and required confirmation on two disease assessments at least 4 weeks apart. Complete response required disappearance of target and non-target lesions and no evidence of new lesions. Partial response required at least a 30% decrease in the sum of longest dimensions of target measurable lesions from baseline, with no unequivocal progression of non-target lesions and no new lesions. A special rule applied when the only target lesion was a solitary pelvic mass assessed by physical examination and not radiographically measurable: a 50% decrease in its longest dimension was required.

These criteria make the primary question more specific than whether a participant’s disease changed in any way. They set rules for classifying response and require confirmation, while the endpoint itself is a count of participants meeting either response category. The registry record does not report the resulting count, the denominator used for the analysis, or an estimate of response uncertainty. It therefore documents the question and its measurement rules without establishing the observed response frequency.

3. Trial Design

The registry identifies this as a phase II study of AMG 706 in patients with persistent or recurrent ovarian epithelial cancer, fallopian tube cancer, or primary peritoneal cancer. Its design is listed as single group, with no masking and allocation marked not applicable. Enrollment is listed as 23. The record names the Gynecologic Oncology Group as sponsor and lists the study status as terminated. The start is recorded as 2007-12 and primary completion as 2013-07.

The intervention is identified as motesanib diphosphate, with an arm description of AMG 706 daily. No comparator arm is recorded. Accordingly, the study can describe outcomes among participants in the treatment group, but it cannot directly estimate a treatment contrast against a concurrent control. Differences between this group and any outside population would not be a randomized comparison and are not reported as study results.

The primary endpoint was the number of eligible and treated participants with complete or partial tumor response by RECIST version 1.0. Tumor assessments were scheduled by CT scan or MRI every other cycle for the first 6 months, then every 3 months thereafter, and at other times if clinically indicated, up to 5 years. The record also lists duration of overall survival, grade 3 or higher adverse effects assessed by Common Terminology Criteria for Adverse Events version 3.0, and duration of progression-free survival as secondary outcomes.

These design details describe how the trial was organized and what it intended to assess; they do not establish how many participants completed follow-up or contributed data to each outcome. The reason for termination, the protocol’s stopping rules, and the timing or rationale for any study closure are not reported in the registry record. Nor is a protocol-level account of the intended statistical analysis reported. The registry’s enrollment figure should not be assumed to equal the analysis population for every endpoint.

With a single group, the primary result would ordinarily be interpreted as a response frequency within the enrolled study population, subject to the endpoint definition and available follow-up. Without a concurrent comparison group, response counts alone cannot separate a treatment-associated pattern from other influences on outcomes. That limitation is part of the design, not a statistical adjustment that can be resolved from the information recorded.

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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Eligible and treated patients.

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: Eligible and treated patients.

6. Primary Endpoint

The registry lists the primary outcome as Number of Patients With Objective Tumor Response Rate (Complete Response [CR] or Partial Response [PR]) Using RECIST Version 1.0, measured over CT scan or MRI every other cycle for the first 6 months; then every 3 months thereafter; and at any other time if clinically indicated up to 5 years..

The primary endpoint counts eligible and treated participants classified as having an objective tumor response, defined as complete response or partial response using RECIST version 1.0. It is a participant count rather than a time-to-event measure. The registry record specifies that complete response requires disappearance of all target and non-target lesions, no new lesions, and confirmation on two assessments at least 4 weeks apart. Partial response requires at least a 30% decrease in the sum of the longest dimensions of target measurable lesions relative to baseline, without unequivocal progression of non-target lesions or new lesions, also confirmed on two assessments at least 4 weeks apart.

For a participant whose only target lesion was a solitary pelvic mass measured by physical examination and not radiographically measurable, the record specifies a 50% decrease in the longest dimension. This rule allows that particular assessment situation to be classified within the response framework.

General: A response endpoint is useful because it applies prespecified criteria to changes in tumor measurements, rather than treating any change as a response. Requiring confirmation can reduce reliance on a single assessment. A count is easy to interpret as the number of participants meeting the criteria, but a response rate also requires a clearly specified denominator and complete outcome information.

The imaging schedule was CT scan or MRI every other cycle for the first 6 months, then every 3 months thereafter, with additional assessments if clinically indicated, up to 5 years. The registry record does not report the primary endpoint count or rate, the confidence interval, or the analysis denominator. Thus, it records the endpoint’s definition and assessment schedule, but not the observed primary result.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

General: Statistical methods should match the kind of outcome being measured. The primary endpoint is a count of participants meeting a binary classification: objective response or no objective response. A time-to-event outcome, such as duration of overall survival or progression-free survival, has a different structure because the timing of an event and the length of observation matter. Adverse-event counts are also distinct from tumor-response outcomes and should be interpreted using their own denominators and definitions.

What a response count can establish

For a binary endpoint, a participant is classified according to the endpoint rules, and the analysis counts how many participants fall into the response category. A response rate expresses that count relative to a specified population. The registry record identifies eligible and treated patients as the primary endpoint population, but does not report the observed count or analysis denominator. Without both, a response rate cannot be recovered from the record. Enrollment alone is not a substitute for the reported analysis denominator, because the record does not establish that every enrolled participant contributed an evaluable primary endpoint.

The response classification depends on RECIST version 1.0. Complete and partial response require specific changes in lesions and confirmation by two assessments at least 4 weeks apart. As a general statistical point, explicit classification rules help make an outcome more reproducible: the reader can understand what counted as an event rather than relying on an informal judgment. However, a well-defined outcome does not guarantee that its observed result, denominator, or uncertainty will be reported.

How uncertainty is usually described

General: A confidence interval is a range of values compatible with the observed data under a specified statistical procedure. For a response proportion, it communicates how imprecisely the underlying response frequency is estimated. A narrow interval indicates greater precision than a wide one, while neither interval width nor its interpretation establishes causation. The registry record does not report a confidence interval for the primary endpoint, so no trial-specific precision estimate can be described.

General: A p-value evaluates how compatible observed data are with a specified null hypothesis under assumptions of the analysis. It is not the probability that a treatment works, nor does it measure the size or clinical importance of an effect. No p-value or inferential analysis is reported in the registry record. In a single-group trial, any test would also depend on a reference value or other explicit statistical hypothesis; such a hypothesis is not reported in the registry record.

Why the design affects inference

General: Random allocation creates a basis for comparing groups because assignment is not chosen according to participant characteristics. In this study, allocation is listed as not applicable and the model is single group. There is therefore no randomized control arm in the record. Descriptive outcomes in the treatment group do not provide the same information as a randomized between-group comparison, and statistical methods cannot create a missing concurrent control group from the recorded details.

The record lists masking as none. General: Masking can reduce the influence of treatment knowledge on some assessments or decisions, but its role varies by outcome and trial procedures. The registry does not report a more detailed account of response assessment procedures or safeguards. The RECIST criteria and confirmation schedule are recorded, but further implementation details are not reported in the registry record.

Interpreting safety counts

General: Safety counts should be read with their at-risk population, event definition, and observation period in mind. A participant count indicates how many people experienced an event, not necessarily how many event episodes occurred. Different participants can experience the same event term, and one participant can experience more than one type of event. The registry reports 22 at risk for serious adverse events and 11 affected, as well as 22 at risk for other adverse events and 21 affected. It also lists selected serious-event terms and percentages. The record does not describe event duration, repeated episodes, individual-level overlap among terms, or a comparison group.

General: Over-dispersion is a situation in which count outcomes vary more than a statistical model expects. It can matter when analyzing counts of repeated events, because treating every event as independent may understate uncertainty if multiple events come from the same participant. The registry’s posted safety figures are participant counts and listed terms; it does not report a repeated-event model or an analysis of event counts per participant. No trial-specific conclusion about over-dispersion can therefore be drawn.

Planning illustrations and observed power

The planning assumptions described for any sample-size illustration use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the target effect expressed as a stated fraction of the observed effect. For time-to-event outcomes, such illustrations concern the total number of events; for other outcomes, they concern participants per arm for 80% power or another stated planning level. General: Such values are planning illustrations, not observed power and not evidence that the trial achieved a particular power. They are not the trial’s own design justification. The record does not report the protocol’s sample-size justification. Because the primary observed effect is not reported, a trial-specific interpretation of illustrations based on that effect is not possible from the registry record.

No analysis is reported in the registry record. The intended statistical plan, including any model choices, confidence-interval procedure, interim analyses, multiplicity strategy, or sensitivity analyses, is not reported in the registry record. General explanations of these methods clarify what they can do, but they should not be mistaken for methods documented as having been used in this trial.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary response endpoint and secondary outcomes of duration of overall survival, incidence of grade 3 or higher adverse effects, and duration of progression-free survival. When several outcomes are assessed, multiplicity can arise if multiple statistical tests are used to support conclusions. General: Without a prespecified strategy, repeated testing can increase the chance of a nominally unusual result. A multiplicity plan may define a primary outcome, adjust significance thresholds, or specify a hierarchy of tests. No such strategy is reported in the registry record, and no analyses are reported.

The primary endpoint population is described as eligible and treated patients. The record lists enrollment of 23, but does not report the number analyzed for the primary endpoint or the analysis populations for the secondary outcomes. It does not report how participants without an evaluable assessment were handled, whether missing outcome data occurred, or whether any imputation or sensitivity analysis was used. These details are important because the interpretation of a count depends on who was included and how unavailable outcomes were treated.

The record also does not report interim analyses, stopping rules, or a statistical analysis plan. The study status is terminated, but the reason for termination and its relationship, if any, to outcome review or analysis are not reported in the registry record. These omissions prevent assessment of how the planned analysis addressed multiplicity, incomplete follow-up, or early study closure.

General: Missing-data methods cannot recover information without assumptions. Analyses that exclude participants with unavailable outcomes may describe only those with observed assessments, while imputation methods rely on assumptions about unobserved outcomes. The appropriate interpretation depends on the specific endpoint and reason data are missing. Since the registry does not describe these matters, no particular missing-data approach should be attributed to the study.

11. Safety Results

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

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: obstruction, gi - small bowel nos (9.1%), cholecystitis (9.1%), hypertension (4.5%), death no ctcae term - disease progression nos (4.5%), vomiting (4.5%).

The registry lists 22 participants at risk for serious adverse events, of whom 11 are recorded as affected. It also lists 22 at risk for other adverse events, with 21 affected. The record’s selected serious-event terms include obstruction, GI - small bowel NOS and cholecystitis, each listed at 9.1%; hypertension, death no CTCAE term - disease progression NOS, vomiting, and creatinine, each listed at 4.5%.

These figures are descriptive. The listed terms should not be read as a complete account of all safety findings, nor do the percentages establish the number of distinct participants across different terms. The registry record does not explain overlap between terms, repeated events, event timing, or whether the selected terms represent all serious events meeting the reporting criteria.

A serious adverse event is not automatically an event caused by the intervention. The registry record does not provide a control group or a comparative safety estimate, and the listed event terms alone cannot determine causation. The record identifies grade 3 or higher adverse effects assessed by Common Terminology Criteria for Adverse Events version 3.0 as a secondary outcome, but does not report a full analysis of that outcome. No conclusion about relative safety can be drawn from these counts alone.

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

The record establishes that a phase II, single-group study evaluated daily AMG 706 in patients with persistent or recurrent ovarian epithelial, fallopian tube, or primary peritoneal cancer. It specifies a primary endpoint based on complete or partial response under RECIST version 1.0, the imaging schedule, and several secondary outcomes. It also records enrollment of 23 and selected adverse-event counts for 22 participants at risk.

What the record does not establish is the observed number of participants who met the primary response criteria. Without that result and the analysis denominator, the response frequency and its precision are not available. The presence of a defined endpoint should not be confused with a reported endpoint finding. Nor does the record provide estimates for overall survival, progression-free survival, or the listed grade 3 or higher adverse effects.

The single-group design means that even a reported response count would describe outcomes in the treatment group rather than provide a randomized comparison with another group. Allocation is not applicable, masking is none, and no comparator is recorded. A single-group result cannot by itself show how outcomes would have differed under another treatment or under a control condition. The record also does not report the statistical analysis plan, the sample-size justification, or the reason for termination.

The safety entries document that adverse events were recorded, including serious adverse events and selected terms. They do not establish that the intervention caused those events or how its safety compares with another treatment. The registry does not provide the detailed event-level information or comparative design needed for those interpretations.

Accordingly, the defensible reading is narrow: the registry describes the study’s question, eligibility group, treatment, endpoint rules, follow-up schedule, and selected safety counts. It does not report enough analyzed outcome information to quantify the primary response result or establish a comparative treatment effect. Statistical interpretation should distinguish the documented design and measurements from results that are not reported in the registry record.

13. Why This Trial Matters Statistically

This study illustrates an important distinction in clinical-trial reporting: a clearly specified endpoint is not the same as a reported result. The registry describes objective response using RECIST version 1.0, including measurement rules and confirmation assessments. Yet the primary response count and analysis denominator are not reported in the registry record. Readers therefore know what was intended to be counted, but cannot determine how many participants met the endpoint or how precisely the response frequency was estimated.

It also illustrates the limits of single-group evidence. The study is listed as a single-group phase II trial, with no comparator and allocation marked not applicable. Such a design can describe outcomes in its treatment group, but it does not provide a randomized estimate of a difference between treatments. This distinction is central when interpreting both tumor response and safety counts.

The record also highlights why denominators and analysis populations matter. Enrollment is 23, while the adverse-event risk group is 22; the registry does not report whether the primary endpoint used all enrolled participants or a different denominator. The difference between a trial’s enrollment figure and the population contributing to a particular outcome should not be assumed away.

Finally, the record lists important secondary outcomes and selected adverse-event terms without reporting their inferential analyses. This makes it possible to describe what was planned or recorded, but not to reconstruct a full statistical assessment. The study is therefore useful as an example of how design, endpoint definitions, denominators, and result reporting jointly determine what conclusions can be supported. Where a key estimate is absent, a careful statistical reading identifies that gap rather than replacing it with an assumption.

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.

Several trial-specific limitations constrain interpretation. The registry lists a single group, no masking, and allocation as not applicable. There is no concurrent comparator, so the record cannot support a randomized comparison of outcomes. The enrollment figure is 23, but the primary endpoint population is described only as eligible and treated patients; the analysis denominator is not reported in the registry record.

The central limitation is that the observed primary response count and response rate are not reported. The endpoint criteria and assessment schedule are detailed, but they do not supply the outcome itself. The registry also does not report a confidence interval or other inferential analysis for the primary endpoint. Although overall survival, progression-free survival, and grade 3 or higher adverse effects are listed as secondary outcomes, their results are not reported in the record.

The reason for termination is not reported in the registry record. Neither the protocol’s sample-size justification nor its statistical analysis plan is reported. The record also does not describe interim analyses, stopping rules, missing-data methods, or the analysis populations for the secondary endpoints. Consequently, it is not possible to assess whether the planned methods addressed incomplete outcome data or multiple outcomes.

The adverse-event figures have their own limits. The record gives participant counts at risk and affected, plus selected serious-event terms and percentages, but does not provide details about overlap, recurrence, timing, or comparative rates. These entries describe recorded events and do not establish causation. The record also does not provide enough information to evaluate how representative the enrolled group was of other patients with the listed conditions. These limitations are specific to the information documented for this study and should be kept separate from the RECIST endpoint definition itself.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: AMG 706 in Treating Patients With Persistent or Recurrent Ovarian Epithelial Cancer, Fallopian Tube Cancer, or Primary Peritoneal Cancer — NCT00574951.
• Registry record last updated 2018-01-11; status last verified 2015-05.
• Results first posted 2018-01-11.
• 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.

18. Record Summary

This registry record concerns NCT00574951, a phase II, single-group study of AMG 706, identified as motesanib diphosphate, for persistent or recurrent ovarian epithelial cancer, fallopian tube cancer, or primary peritoneal cancer. The intervention was described as AMG 706 daily. Enrollment was 23; the study is listed as terminated. The sponsor was the Gynecologic Oncology Group.

The primary endpoint counted eligible and treated participants with complete or partial response under RECIST version 1.0. Assessments were scheduled by CT scan or MRI every other cycle for the first 6 months, then every 3 months thereafter and when clinically indicated, up to 5 years. Secondary outcomes included overall survival, progression-free survival, and grade 3 or higher adverse effects.

The registry records 22 participants at risk for serious and other adverse events, with 11 and 21 affected, respectively. It does not report the primary response count, outcome analyses, the reason for termination, or the statistical analysis plan. The available record therefore documents design and measurement details, but does not permit quantification of the primary response result or a comparative treatment-effect estimate.