This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for A Phase II Study of Akt Inhibitor MK2206 in the Treatment of Recurrent Platinum-Resistant Ovarian, Fallopian Tube, or Peritoneal Cancer (NCT01283035), a na phase 2 study. The primary outcome is Efficacy (as Measured by Objective Response Rate) of Akt Inhibitor MK2206 in Patients With Recurrent High-grade Platinum-resistant Serous Ovarian, Fallopian Tube, or Primary Peritoneal Cancer.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01283035 |
| Phase | Phase 2 |
| Condition | Ovarian Sarcoma, Recurrent Fallopian Tube Carcinoma, Recurrent Ovarian Carcinoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 6 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Cancer Institute (NCI) |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients with recurrent high-grade serous platinum-resistant ovarian, fallopian tube, or primary peritoneal cancer. The registry’s broader condition list also includes ovarian sarcoma and recurrent forms of these cancers. Its summary describes interest in tumors with mutations in P13K or AKT, or low levels of PTEN. Five patients started study treatment; the registry enrollment is 6.
Intervention
The intervention was Akt inhibitor MK2206, described in the treatment plan as an oral drug taken once a week for four weeks per cycle. Treatment was to continue while a participant was benefiting from the study drug. Laboratory biomarker analysis was also listed as an intervention.
Comparator
There was a single treatment group and no control arm. Allocation is listed as not applicable, and masking as none. Consequently, the study does not provide a randomized, like-for-like comparison against another treatment or a control condition.
Primary question
The primary endpoint was objective response rate: whether participants had a complete or partial tumor response assessed using RECIST v1.0. The registry describes a planned decision rule based on responses among a final set of 29 patients. It does not report the observed response count or response rate, so the treatment’s observed efficacy cannot be determined from the registry record.
The clinical question is therefore about tumor response in a defined recurrent, platinum-resistant cancer population treated with MK2206, rather than a comparative effect between treatments. Biomarker outcomes were also planned: the listed secondary outcomes include associations between selected biomarkers and response, progression-free survival, and overall survival, as well as biopsy analyses of feedback-loop activation and target inhibition in selected patients. The record also lists survival duration and toxicities as secondary outcomes. Those aims give the study a biological and safety context, but they do not replace the primary response question. The registry describes the trial as phase II and completed. Completion status alone does not provide an efficacy conclusion, and the registry record does not report the primary response result.
3. Trial Design
This was a phase II, single-group study sponsored by the National Cancer Institute (NCI). Allocation is listed as not applicable, masking as none, and the model as single group. The intervention group received MK2206; laboratory biomarker analysis was also listed. With no comparator arm, the design can describe outcomes among treated participants but cannot isolate a treatment effect from other explanations through a randomized comparison.
The registry lists enrollment of 6, while its primary endpoint population description says that five patients started study treatment. These are different registry figures and should not be treated as interchangeable. The record does not explain the difference. It describes the planned primary endpoint timeframe as up to 3 years. Treatment was planned as oral dosing once a week for four weeks per cycle, continuing while a participant was benefiting from the drug.
The primary endpoint description sets out a decision rule for a final set of 29 patients: if 4 or more demonstrated a response, the stated null hypothesis of a response rate at or below 5% could be rejected in favor of an alternative of at least 20%, using alpha of 0.05 and beta of 0.20, corresponding to 80% power. This is a design statement, not a report of observed responses. The registry does not explain why enrollment was 6 or why five patients started treatment, and it does not report whether the planned final set was reached.
Secondary aims included biomarker associations, biopsy-based analyses in selected patients, overall and progression-free survival, and toxicities assessed using the active version of NCI CTCAE v4.0. The record lists 6 outcome measures and 0 analyses. The study status is completed; its listed start is 2011-04 and primary completion is 2014-12. These administrative details do not establish that each planned outcome was analyzed or reported.
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 | Treatment (Akt Inhibitor MK2206) |
|---|---|
| STARTED | 6 |
| COMPLETED | 5 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Treatment (Akt Inhibitor MK2206) |
|---|---|
| Subject later found ineligible | 1 |
Analysis population for the primary outcome, as defined in the registry: Five patients started study treatment.
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 | Treatment (Akt Inhibitor MK2206) |
|---|---|---|
| Participants analysed | 5 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 3 |
| Age, Categorical: >=65 years | Participants | 2 |
| Sex: Female, Male: Female | Participants | 5 |
| Sex: Female, Male: Male | Participants | 0 |
| Region of Enrollment: United States | participants | 5 |
6. Primary Endpoint
The registry lists the primary outcome as Efficacy (as Measured by Objective Response Rate) of Akt Inhibitor MK2206 in Patients With Recurrent High-grade Platinum-resistant Serous Ovarian, Fallopian Tube, or Primary Peritoneal Cancer, measured over Up to 3 years.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is objective response rate, a binary outcome: each participant is classified according to whether an objective tumor response occurred. The registry specifies RECIST v1.0 criteria for target lesions assessed by MRI. Complete response means disappearance of all target lesions. Partial response means a decrease of at least 30% in the sum of the longest diameters of target lesions. Progression is defined by an increase of 20% in that sum, a measurable increase in a non-target lesion, or appearance of new lesions. Stable disease is neither sufficient shrinkage for partial response nor sufficient increase for progression.
Objective response is used to summarize tumor change as a participant-level outcome. A response-rate endpoint can be interpreted as the proportion of participants meeting the complete- or partial-response criteria, rather than as an average change in tumor size. In this record, however, no observed response count or response rate is reported. The planned threshold of 4 or more responses among a final set of 29 patients is a decision rule, not the trial’s observed endpoint result.
The distinction between endpoint definition and endpoint result matters. The criteria specify how a response would be classified; they do not indicate how many participants met those criteria. Nor does the planned timeframe of up to 3 years establish the duration of any response. The registry record does not report the observed primary outcome or the response-assessment results needed to evaluate it.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a single-arm phase II study and a planned objective-response decision rule, but does not report the observed primary response result. It gives the rule as 4 or more responses in a final set of 29 patients to reject a null hypothesis of a response rate at or below 5% in favor of an alternative of at least 20%, with alpha of 0.05 and beta of 0.20. Those values describe the planned statistical criterion. They are not observed response data and cannot establish whether the criterion was met.
The record lists enrollment of 6 and separately states that five patients started study treatment. It does not report why these figures differ, whether every treated patient had evaluable response data, or how many participants completed follow-up. It therefore does not support calculation of an observed response proportion or an uncertainty interval. It also does not report the result of a comparison with another group, since there was no comparator arm.
The single-group design limits interpretation even if an outcome count were available. A response observed after treatment would describe the treated participants, but without a concurrent control group it would not by itself show how outcomes would have differed under another treatment or no study treatment. Differences in participant characteristics, disease course, assessment, or follow-up could not be separated through random assignment in this design.
The secondary outcome list indicates that the trial intended to examine biomarkers, survival, and toxicity, among other outcomes. The registry record does not report the corresponding outcome estimates or analysis results. It also lists 0 analyses. The absence of posted analyses should not be interpreted as evidence that no analyses were conducted outside the registry; it means the registry record does not provide them.
Safety entries report that 4 of 5 participants at risk were affected by serious adverse events and that 5 of 5 were affected by other adverse events. Rash maculo-papular and colonic obstruction are listed among the top serious-event terms, with percentages of 80.0% and 20.0%, respectively. These figures describe the registry’s reported safety entries, not a comparison against another group. Deaths are not reported in the registry record. The available counts do not establish event severity beyond the serious-event classification, timing, duration, or attribution to treatment.
Overall, the record supports describing the study design, planned response criterion, and some safety counts. It does not support a conclusion about the observed objective-response rate, survival outcomes, biomarker findings, or comparative treatment effects. The planned 80% power is a design property under its stated assumptions; it is not observed power and does not substitute for reporting the actual outcomes.
9. Statistical Methods Explained
The primary endpoint is a binary participant-level outcome. A participant either meets the registry’s criteria for objective response, through complete or partial response, or does not. The endpoint description uses RECIST v1.0 definitions for target lesions assessed by MRI. Complete response is disappearance of all target lesions; partial response is a decrease of at least 30% in the sum of the longest diameters of target lesions. Progression is defined as an increase of 20% in that sum, a measurable increase in a non-target lesion, or new lesions. Stable disease falls between the stated partial-response and progression thresholds.
For a binary endpoint, the response rate is conceptually the fraction of evaluable participants who meet the response definition. The registry does not report the observed number of responders or an observed rate, so neither can be stated. It also does not specify in the record how missing or unevaluable response assessments were handled. The distinction is important: the endpoint definition tells readers what counts as response, whereas analysis rules determine which participants enter the denominator and how incomplete assessments are treated.
What does the planned decision rule say?
The registry describes a planned rule for a final set of 29 patients. If 4 or more demonstrated a response, the stated null hypothesis of a response rate at or below 5% could be rejected in favor of an alternative of at least 20%. The record states alpha of 0.05 and beta of 0.20, corresponding to 80% power. This is a prespecified design criterion as described in the registry, not a report that the required number of responses occurred.
In general, alpha is the chosen probability threshold for rejecting a null hypothesis when that null is true, under the assumptions of the test. Beta is the probability of failing to reject the null under a specified alternative; power is one minus beta. These concepts describe operating characteristics of a planned procedure under assumptions. They do not measure the probability that the treatment is effective, and they do not reveal the actual endpoint result. The registry record does not provide further detail about the test implementation or its assumptions beyond the stated threshold and design values.
Why does the single-group design matter?
A single-group study estimates outcomes among participants receiving the intervention. It does not randomize participants to a concurrent control group, so it cannot directly estimate a between-group treatment contrast. The planned comparison is against response-rate hypotheses stated in the record, not against outcomes from a randomized control arm in this trial. Historical or benchmark assumptions can inform a design, but this record does not identify a concurrent comparator or report comparative results.
The lack of masking is also recorded: masking is listed as none. The registry does not explain what safeguards, if any, were used in response assessment. In general, knowledge of treatment assignment can affect some judgments or behaviors; objective criteria may standardize assessment, but they do not remove every possible source of bias. No additional assessment procedures should be inferred from the registry record.
How should the safety counts be understood?
Safety outcomes are reported as participant counts affected, rather than as a complete account of event episodes. The registry lists 4 of 5 participants at risk for serious adverse events as affected, and 5 of 5 at risk for other adverse events as affected. These are not counts of distinct event types or event episodes. The serious-event terms listed include rash maculo-papular at 80.0% and colonic obstruction at 20.0%; the record does not make the percentage denominator explicit in the listed information. The deaths entry is not reported in the registry record.
In general, safety percentages depend on their denominator and on how repeated events are handled. A participant may experience more than one event, and a term-level percentage is not necessarily the percentage of participants experiencing any serious event. Readers should keep the all-serious-event affected count distinct from the listed term percentages. These safety observations also do not establish causality or comparative risk without additional information.
What statistical details remain unspecified?
The registry lists 0 analyses and does not report an observed primary outcome, analysis population, confidence interval, missing-data procedure, or detailed model. The listed secondary endpoints include overall survival and progression-free survival, but the record does not report their estimates or analysis methods. General statistical practice may use uncertainty intervals to show the range of values compatible with data under a model; no such interval is reported for the primary endpoint.
Over-dispersion is relevant to some count models when variability exceeds what a simple model assumes, but the primary endpoint is described as a binary response count, and the registry does not report a count model. No conclusion about over-dispersion or model diagnostics is warranted. Likewise, the record does not report interim analyses, stopping rules, or additional statistical-plan details. The stated response threshold and error rates should not be expanded into an assumed protocol beyond what is recorded.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 6 outcome measures, including the primary objective-response endpoint and secondary outcomes involving biomarkers, biopsies, survival, and toxicities. It does not report how statistical multiplicity across these outcomes was handled. Multiplicity arises when several hypotheses or outcomes are examined: even if each test uses a conventional threshold, the chance of at least one false-positive finding can increase across a family of tests. General approaches can adjust thresholds or distinguish primary, secondary, and exploratory questions, but the record does not state that any particular approach was used.
The primary endpoint description does state alpha of 0.05 and beta of 0.20 for its planned response decision rule. That information does not establish the multiplicity strategy for secondary outcomes or biomarker analyses. The registry does not report interim analyses or additional testing rules, so none should be assumed.
Missing-data handling and analysis populations are also not reported in the registry record. The record gives enrollment of 6 and says five patients started treatment, but does not explain the difference, report a participant flow, or clarify how many patients were evaluable for response. It does not say whether participants without an assessment were counted as nonresponders, excluded, or handled in another way. These choices can affect a binary response estimate, particularly in a small study. The registry lists 0 analyses and does not provide an observed endpoint estimate or its uncertainty. These omissions limit interpretation; they do not establish that missing data or multiplicity were handled in any particular way 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 |
|---|---|---|---|
| Treatment (Akt Inhibitor MK2206) | 4 | 5 | 80.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Treatment (Akt Inhibitor MK2206) | 5 of 5 |
Most frequent serious adverse event terms
| Event term | Organ system | Treatment (Akt Inhibitor MK2206) |
|---|---|---|
| Rash maculo-papular | Skin and subcutaneous tissue disorders | 4 of 5 |
| Colonic Obstruction | Gastrointestinal disorders | 1 of 5 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Treatment (Akt Inhibitor MK2206) |
|---|---|---|
| Fatigue | General disorders | 3 of 5 |
| Diarrhea | Gastrointestinal disorders | 2 of 5 |
| Oral mucositis | Gastrointestinal disorders | 2 of 5 |
| Cough | Respiratory, thoracic and mediastinal disorders | 1 of 5 |
| Hyperglycemia | Metabolism and nutrition disorders | 1 of 5 |
| Abdominal Pain | Gastrointestinal disorders | 1 of 5 |
| Chelitis | Gastrointestinal disorders | 1 of 5 |
| Conjunctivitis | Eye disorders | 1 of 5 |
| Dysgeusia | Nervous system disorders | 1 of 5 |
| Increased urea | Investigations | 1 of 5 |
Serious events with the highest arm-level rate: rash maculo-papular (80.0%), colonic obstruction (20.0%).
The registry lists one treatment group. For serious adverse events, 4 of 5 participants at risk were affected; for other adverse events, 5 of 5 participants at risk were affected. These are participant-level affected counts in the registry entry, not totals of event episodes. The record lists rash maculo-papular at 80.0% and colonic obstruction at 20.0% among the top serious-event terms. It does not make the denominator for those term percentages explicit in the reported information, so they should not be treated as a direct calculation from the affected-participant counts.
The two affected counts should not be added together: the groups describe serious and other adverse events, and the same participant could be represented in both. Nor do the listed terms establish how many episodes occurred, when events began, how long they lasted, or whether investigators attributed them to MK2206. Deaths are not reported in the registry record. Without a comparator group, these figures cannot provide a comparative estimate of safety. They describe the safety information posted for this single treatment group, with the limitations of the reported detail.
12. What the Result Does, and Does Not, Mean
The registry record documents a phase II, single-group study of MK2206 and specifies objective response as its primary endpoint. It also describes a planned rule requiring 4 or more responses in a final set of 29 patients to reject a null hypothesis of a response rate at or below 5% in favor of an alternative of at least 20%, with alpha of 0.05 and beta of 0.20. These are planned design values, not outcome findings.
The record does not report the actual number of participants who had a complete or partial response, the observed response rate, or its uncertainty. Although the record lists enrollment of 6 and says five patients started treatment, it does not say whether the planned final set of 29 was reached. Thus, readers cannot use the posted information to determine whether the decision threshold was met or to quantify the observed response.
The study also cannot make a randomized treatment comparison because it had one group and no comparator arm. Even a reported response in treated participants would not, by itself, show how outcomes would have differed under another treatment. The record’s secondary outcome list identifies questions about biomarkers, survival, and toxicity, but the corresponding results are not reported in the registry record.
The safety entries indicate that serious and other adverse events were reported among participants at risk, with specific serious-event terms listed. They do not establish a comparative risk, causal attribution, or a full account of event timing and severity. The death count is not reported. These findings should be read as limited descriptive information, not as a complete assessment of either benefit or harm.
In short, the registry supports a description of the study’s population, intervention, endpoint definition, planned response criterion, and some safety counts. It does not provide the outcome data needed to conclude whether the response criterion was reached, what the survival outcomes were, or how the intervention compared with another approach. A planned power level is not evidence that the study achieved that power in practice or that an effect was observed.
13. Why This Trial Matters Statistically
This trial illustrates the difference between a statistical design and a statistical result. The registry states a decision threshold for objective response, along with alpha and beta values, but those features describe how the study was planned to evaluate a response count. They do not reveal the response count that occurred. Without the endpoint result, the central efficacy question remains unanswered by the registry record.
The distinction between enrollment and the primary endpoint population is also important. The registry lists enrollment of 6 but states that five patients started study treatment. It does not explain the difference. A reader cannot assume that every enrolled participant received treatment, that every treated participant had an evaluable response, or that the planned final set of 29 was reached. Clear participant flow and explicit analysis populations are essential for interpreting a response proportion.
The single-group design offers a further statistical lesson. A one-arm study can describe outcomes under the study intervention and evaluate them against a stated benchmark, but it does not produce a randomized comparison with a control arm. The interpretation therefore depends on the planned criterion and on assumptions about the benchmark, while remaining different from a causal comparison generated by random allocation.
Finally, a registry can contain detailed endpoint definitions and safety entries while still not reporting outcome analyses. Here, the primary response result, survival estimates, biomarker results, analysis populations, and missing-data methods are not reported in the registry record. Recognizing which details are available—and which are not—prevents a planned hypothesis, a completion status, or a safety count from being mistaken for a treatment-effect estimate.
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 principal limitation for interpretation is that the registry record does not report the primary objective-response result. It provides the endpoint definition and planned threshold, but not the observed response count, rate, or uncertainty. The record also does not report results for the listed secondary outcomes, including survival, biomarker associations, biopsy analyses, and toxicities beyond the posted safety entries.
The enrollment figure is 6, while the primary endpoint description says five patients started treatment. The registry does not explain this discrepancy or provide participant flow. It also does not state how many patients were evaluable for response, define the analysis population, or describe handling of missing assessments. These omissions prevent assessment of the response denominator and of how incomplete follow-up may have affected the endpoint.
The single-group design has no randomized comparator, and masking is listed as none. As a result, the study cannot furnish a direct randomized estimate of treatment effect relative to a control condition. The record does not report further safeguards or details about response assessment that would clarify potential sources of bias.
Although the primary endpoint description gives a planned threshold for a final set of 29 patients, the registry enrollment is 6 and no analysis is listed. The record does not establish whether the planned final set was reached, whether the stated rule was applied, or why the posted enrollment differs from the planned set. It also does not report multiplicity procedures, interim analyses, a detailed statistical plan, confidence intervals, or missing-data methods. These are limitations of the available registry information; the record does not establish what may have been documented elsewhere.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Phase II Study of Akt Inhibitor MK2206 in the Treatment of Recurrent Platinum-Resistant Ovarian, Fallopian Tube, or Peritoneal Cancer — NCT01283035.
- Registry record last updated 2016-06-24; status last verified 2016-05.
- Results first posted 2016-06-24.
- 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
This completed phase II study evaluated Akt inhibitor MK2206 in a single group of patients with recurrent platinum-resistant ovarian, fallopian tube, or primary peritoneal cancer. The registry lists enrollment of 6 and states that five patients started study treatment. MK2206 was planned as an oral treatment once a week for four weeks per cycle, continuing while a participant was benefiting from the drug.
The primary endpoint was objective response assessed using RECIST v1.0 criteria. The registry describes a planned decision rule based on 4 or more responses among a final set of 29 patients, with alpha of 0.05 and beta of 0.20. The observed response result is not reported in the registry record. Secondary outcomes included biomarker, survival, and toxicity questions; the record lists 0 analyses.
Safety entries report serious adverse events affecting 4 of 5 participants at risk and other adverse events affecting 5 of 5. Rash maculo-papular and colonic obstruction are listed among the top serious-event terms. Deaths are not reported in the registry record. With no comparator group and no posted primary outcome result, the record does not establish an observed treatment effect.