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 GSK1120212+GSK2141795 for Cervical Cancer (NCT01958112), a na phase 2 study. The primary outcome is Response Rate for the Combination of GSK1120212 (Trametinib) and GSK2141795 in Patients With Recurrent or Persistent Cervical Cancer..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01958112 |
| Phase | Phase 2 |
| Condition | Cervical Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 14 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Dana-Farber Cancer Institute |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned people with persistent or recurrent cervical cancer. The registry record lists cervical cancer as the condition and describes the response population as participants with recurrent or persistent disease. Enrollment was 14. Tumor samples were to be tested for mutations in genes that might be associated with sensitivity to the drug combination.
Intervention
The study treatment combined GSK1120212, also called trametinib, with GSK2141795. The recorded regimen was trametinib 1.5 mg QD plus GSK2141795 50 mg QD in 28 day cycles. The summary describes trametinib as a MEK inhibitor and GSK2141795 as an AKT inhibitor; both are discussed in relation to signaling pathways in cancer cells.
Comparator
There was no separate comparator group. The registry record identifies a single-group model and lists allocation as NA. Consequently, this study does not provide a randomized, like-for-like comparison between the combination and another treatment or control condition.
Primary question
The primary question was whether the combination produced responses in persistent or recurrent cervical cancer. Response rate was to be assessed by RECIST version 1.1 over a timeframe of 2 Years. The posted record does not report the number of participants who responded, a response-rate estimate, or an uncertainty interval. It also describes an interest in whether tumor genetic make-up might relate to response, but does not report results for that question.
3. Trial Design
This was a phase II, single-arm, single-stage study of GSK1120212 (trametinib) and GSK2141795 in persistent or recurrent cervical cancer. The registry record lists enrollment as 14 and the study status as terminated. The recorded start was 2013-10, and primary completion was 2017-11. A reason for termination is not reported in the registry record.
The single-group design means that participants received the combination regimen rather than being assigned among randomized study arms. The recorded dose was trametinib 1.5 mg QD plus GSK2141795 50 mg QD in 28 day cycles. Masking was listed as none. Allocation was listed as NA, consistent with the absence of a randomized comparator group. The study therefore describes outcomes observed among participants receiving the combination, but does not provide a concurrent control group for estimating how those outcomes compare with another treatment or with no study treatment.
The primary outcome was the response rate for the combination, assessed by RECIST version 1.1, with a stated timeframe of 2 Years. Secondary outcomes included duration of progression-free survival, toxicity measured by the number of participants with adverse events, mutation and co-mutation rates in PI3K and RAS ERK signaling pathway genes, and overall survival. The registry record lists five outcome measures, but does not provide posted analysis results for the primary response outcome.
The record does not report the protocol’s sample-size justification, planned statistical analysis details, or a reason for ending the trial. The study’s single-arm structure is important when interpreting any outcomes: a response count or rate, even if available, would describe this treated group and would not by itself establish a comparative treatment effect.
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 | GSK1120212 (Trametinib) and GSK2141795 |
|---|---|
| STARTED | 14 |
| COMPLETED | 14 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Response rate assessed by RECIST version 1.1.
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 | GSK1120212 (Trametinib) and GSK2141795 |
|---|---|---|
| Participants analysed | 14 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 9 |
| Age, Categorical: >=65 years | Participants | 5 |
| Sex: Female, Male: Female | Participants | 14 |
| Sex: Female, Male: Male | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 14 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 14 |
6. Primary Endpoint
The registry lists the primary outcome as Response Rate for the Combination of GSK1120212 (Trametinib) and GSK2141795 in Patients With Recurrent or Persistent Cervical Cancer., measured over 2 Years.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was response rate among participants with recurrent or persistent cervical cancer. The record says response was assessed using RECIST version 1.1. In general, a response endpoint classifies participants according to whether their disease meets prespecified criteria for response; a response rate is the proportion of the assessed group classified as responders. This kind of endpoint can summarize tumor response during a defined assessment period, but its meaning depends on the response criteria, the participants included in the analysis, and the completeness and timing of assessments.
The registry record gives a timeframe of 2 Years. It does not report the number of participants classified as responders, the denominator used for a response-rate calculation, or a response-rate estimate. No confidence interval or other uncertainty estimate for this endpoint is reported in the registry record. As a result, the endpoint definition and assessment method are known, but the posted information does not establish what response was observed or how precisely it was estimated.
Response rate is distinct from outcomes such as duration of progression-free survival and overall survival, which are listed as secondary outcomes. A participant-level response classification does not by itself describe how long a response lasts, whether disease later progresses, or survival. Those questions require their own outcome definitions and analyses; results for them are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies response rate as the primary outcome, with assessment by RECIST version 1.1 over 2 Years. It does not report a response count or rate. Without those results, there is no posted estimate of how many participants responded, and no basis in the record for describing the magnitude or precision of the response outcome. A confidence interval, p value, and primary-outcome analysis result are not reported in the registry record.
Enrollment was 14, and the study was single-arm. The absence of a comparator matters independently of the missing response result: even a reported response rate would describe outcomes in the treated group rather than a randomized contrast against another group. The record cannot separate the contribution of the combination from other explanations for observed outcomes, such as the natural course of disease or differences in participant characteristics. These are general limitations of interpreting single-arm outcome data, not findings about what happened to participants in this trial.
The registry record lists secondary outcomes for progression-free survival duration, adverse events, mutation and co-mutation rates, and overall survival. It does not report analyses for those outcomes. The safety information does include counts: among 14 participants at risk, 3 were recorded with a serious adverse event and 1 with another adverse event; deaths were recorded for 8 participants. These counts are descriptive. The record does not establish that an adverse event or death was caused by the study drugs, and it does not provide the timing or causes of the deaths.
The listed serious-event terms include thromboembolic event at 14.3%, colonic perforation at 7.1%, hypophosphatemia at 7.1%, and retinal pigment epithelial detachment at 7.1%. The record does not give the event-level details needed to determine whether participants experienced more than one listed term or how each event related to treatment. These percentages should therefore be read as registry-listed term frequencies, not as evidence of causation or comparative risk.
The study status is terminated, but the reason is not reported in the registry record. Termination alone does not explain whether recruitment, safety, efficacy, operational issues, or another consideration led to the decision. Nor does it supply a missing response result. The most defensible interpretation is therefore limited: the registry describes a small single-group study, its intended response assessment, and selected safety counts, while leaving the primary efficacy estimate and several analysis details unreported.
9. Statistical Methods Explained
What does a binary response endpoint represent?
The primary endpoint is a participant-level response classification assessed by RECIST version 1.1. In general, a binary outcome places each assessed participant into one of two broad categories, such as meeting or not meeting a defined response criterion. A response rate then describes the share of the analyzed participants classified as responders. That summary is only interpretable alongside its numerator, denominator, assessment rules, and uncertainty. The registry record does not report the response count, rate, or denominator used for an analysis, so these quantities cannot be described for this trial.
Response rate also answers a different question from time-to-event outcomes. A response classification says whether a participant met the response definition; duration of progression-free survival and overall survival concern time until specified events. The latter outcomes are listed among the study’s secondary outcomes, but analyses of them are not reported in the registry record. No assumptions about their event definitions or statistical handling can be made from the listed outcome names alone.
Why does the single-arm design affect interpretation?
In a single-arm study, outcomes are recorded for a group receiving the study regimen without a randomized concurrent comparator. The design can describe what happened in that group, but it cannot directly estimate a randomized difference between treatments. General explanations for outcomes may include treatment, participant characteristics, disease course, and other factors. Without a comparison group, those explanations cannot be disentangled by a treatment-versus-control contrast. The registry record lists no allocation to comparison arms, and its skip note explicitly cautions that there is no randomized comparison.
For a binary endpoint, a single-arm analysis might report the observed response proportion and an interval describing sampling uncertainty. A confidence interval is a range of values compatible with the observed data under the specified statistical procedure; it communicates precision, not certainty that the true value lies within a particular range. The record does not report a response estimate or interval, and the statistical procedure for such an interval is not reported in the registry record. No inference about the response outcome can be reconstructed from enrollment alone.
What is known about the analysis plan?
The registry record names the primary outcome, its RECIST version 1.1 assessment, and its 2 Years timeframe. It does not report the analysis population, handling of missing response assessments, rules for evaluability, or the planned statistical test or interval method. It also does not report interim analyses or a sample-size justification. These details can affect the meaning of a response estimate, especially when outcome assessments are unavailable or participants do not complete follow-up. In their absence, it is not possible to determine how the investigators planned to handle those situations.
The record lists mutation and co-mutation assessment as a secondary outcome and describes testing tumor samples for genetic features that might relate to response. In general, an analysis of whether a biomarker is associated with outcome needs a defined biomarker classification, an analysis population, and a statistical method. The registry record does not report the genetic analysis results or enough details to specify how such an association was assessed. The stated interest in tumor genetics should not be mistaken for a demonstrated predictive relationship.
How should planning calculations be distinguished from trial results?
Any sample-size figures associated with planning are illustrations, not observed trial outcomes and not observed power. The stated planning framework uses a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect to detect expressed as a stated fraction of the observed effect. For time-to-event planning, the described quantity is a total event count; for other outcomes, it is participants per arm for 80% power or another stated power level. Such assumptions describe a hypothetical calculation framework. They do not describe this single-arm study’s enrollment design, which had 14 participants and no randomized two-arm comparison. The protocol’s own sample-size justification is not reported in the registry record.
In general, over-dispersion refers to variability in observed data that exceeds what a simple model assumes. It can make uncertainty estimates based on that simpler model too narrow if not accounted for. No over-dispersion parameter or model is reported for this binary endpoint, and it should not be assumed to have been used. More broadly, statistical methods should be interpreted only when their relationship to the actual protocol and analysis is documented; the registry record does not provide enough detail to identify a full primary-outcome analysis procedure.
The main statistical distinction is between the intended measurement and the reported evidence. The endpoint definition identifies what the trial planned to assess; the posted counts or estimates would be needed to describe the observed response. In this record, the endpoint is defined, but its result is not reported. The design and the limitations of the available safety counts can be described, while efficacy conclusions and unreported analysis choices cannot.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists one primary outcome and several secondary outcomes: duration of progression-free survival, toxicity measured by adverse-event counts, mutation and co-mutation rates in genes from the PI3K and RAS ERK signaling pathways, and overall survival. It does not report a multiplicity plan. In general, testing several outcomes or subgroup relationships can increase the chance of at least one apparently notable result when each is assessed separately. A prespecified strategy can explain whether and how multiple tests are handled. No such strategy is reported in the registry record, so it is not possible to determine whether any adjustment or hierarchy was planned.
The record also does not report an analysis population for the primary endpoint, rules for missing response assessments, or how incomplete follow-up was to be handled for time-to-event outcomes. Those decisions matter because the participants included in an analysis and the treatment of unavailable outcome data can affect estimates. The listed enrollment of 14 does not establish that all enrolled participants contributed to every endpoint or completed all assessments.
No analysis results are listed in the registry record. It therefore does not report whether interim analyses were planned or performed, or whether any missing-data methods were used. These omissions do not show that a particular procedure was or was not used; they mean that the procedure cannot be identified from the registry 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 |
|---|---|---|---|
| GSK1120212 (Trametinib) and GSK2141795 | 3 | 14 | 21.4% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| GSK1120212 (Trametinib) and GSK2141795 | 8 of 14 |
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 |
|---|---|
| GSK1120212 (Trametinib) and GSK2141795 | 1 of 14 |
Most frequent serious adverse event terms
| Event term | Organ system | GSK1120212 (Trametinib) and GSK2141795 |
|---|---|---|
| Thromboembolic event | Blood and lymphatic system disorders | 2 of 14 |
| colonic perforation | Gastrointestinal disorders | 1 of 14 |
| Hypophosphatemia | Investigations | 1 of 14 |
| Retinal pigment epithelial detachment | Eye disorders | 1 of 14 |
Most frequent non-serious adverse event terms
| Event term | Organ system | GSK1120212 (Trametinib) and GSK2141795 |
|---|---|---|
| rash acneiform | Skin and subcutaneous tissue disorders | 1 of 14 |
Serious events with the highest arm-level rate: thromboembolic event (14.3%), colonic perforation (7.1%), hypophosphatemia (7.1%), retinal pigment epithelial detachment (7.1%).
The safety information lists 14 participants at risk for serious adverse events and 3 participants affected. It lists 14 at risk for other adverse events and 1 affected, and records 8 deaths. These are counts from a study group of 14, not comparisons between treatment groups. The registry record does not provide timing, duration, severity grades, causes of death, or participant-level event histories.
The listed serious-event terms are thromboembolic event (14.3%), colonic perforation (7.1%), hypophosphatemia (7.1%), and retinal pigment epithelial detachment (7.1%). These percentages are reported as term frequencies. The record does not state whether terms could overlap within participants, whether every event was treatment-related, or how the terms were selected for listing. They should not be added together or interpreted as mutually exclusive categories.
An adverse event recorded during a trial is not automatically caused by the treatment. The registry record does not report a causal assessment for these events. The counts are useful as descriptive safety information, but the small single-group study and lack of a comparator limit comparative interpretation. The number of deaths is reported without causes or timing, so it cannot by itself establish the relationship between death and treatment.
12. What the Result Does, and Does Not, Mean
The record establishes that a phase II, single-arm study evaluated trametinib plus GSK2141795 in persistent or recurrent cervical cancer, with response assessed by RECIST version 1.1. It also establishes the recorded regimen, enrollment, study status, and selected adverse-event counts. These details describe the study and the information posted about it; they do not supply the primary efficacy result.
The key missing item is the response outcome. No response count, rate, confidence interval, or statistical test is reported in the registry record. As a result, the posted information does not show how many participants met the response criteria or how precisely a response rate could be estimated. The absence of a posted result should not be converted into a claim that no responses occurred, just as the treatment’s biological rationale should not be treated as evidence that responses occurred.
Even if a response rate were available, this study’s single-arm design would leave no randomized comparator for estimating a relative effect. An observed outcome in the treated group would be descriptive and could not, by itself, establish what would have happened under another treatment or control condition. The registry record also does not report results for the listed secondary outcomes, including progression-free survival duration, overall survival, and mutation analyses.
The safety counts deserve similarly bounded interpretation. They record adverse events and deaths in the study group, but provide no treatment-causality assessment or comparative rates. Termination status is recorded, but the reason for termination is not reported in the registry record. The overall meaning is therefore that the registry documents a small, uncontrolled trial and selected safety information while leaving efficacy outcomes, important analysis details, and the explanation for termination unresolved.
13. Why This Trial Matters Statistically
This trial illustrates why study design and result availability are central to statistical interpretation. The primary outcome is clearly named as response rate, with a defined assessment approach, but the registry record does not give the response result. Knowing the endpoint definition is not equivalent to knowing the observed outcome. Without the response count, denominator, and an uncertainty estimate, the primary question cannot be answered from the posted results.
The single-arm design is another important feature. Such a design can describe outcomes among participants receiving a regimen, but does not offer a randomized comparison that isolates differences between treatments. That distinction matters when readers encounter claims about benefit or harm: a within-group observation is not the same as a comparative treatment effect. The record itself identifies that the arms are not a like-for-like control comparison.
The record also shows how outcome families can raise analysis questions. Response, progression-free survival, overall survival, safety, and tumor mutations address different aspects of the study. The registry lists these outcomes but does not report analyses or a plan for handling multiple outcomes. Likewise, the absence of reported analysis-population and missing-data methods limits the ability to assess what any eventual estimates would represent.
Finally, the safety counts demonstrate the difference between describing events and attributing them to treatment. Counts and listed term frequencies are useful pieces of evidence, but causal and comparative conclusions require context not reported in the registry record. Taken together, these features make the trial statistically informative as an example of how design, endpoint reporting, uncertainty, and causal interpretation should be considered separately.
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 main trial-specific limitation is the lack of a reported primary response result. The registry record specifies RECIST version 1.1 and a timeframe of 2 Years, but gives no response count, rate, interval, or analysis. Without those results, the intended efficacy outcome cannot be summarized statistically.
The study enrolled 14 participants and used a single-group design without a randomized comparator. This limits both precision and the ability to make comparative inferences. No protocol sample-size justification is reported in the registry record, so the rationale for the enrollment target cannot be assessed. A reason for termination is also not reported; the status alone does not explain why the study ended.
Several details needed to interpret endpoint results are absent, including the primary analysis population, handling of missing assessments, planned inferential methods, and any interim analysis plan. The registry lists secondary outcomes but does not report their analyses or a multiplicity strategy. The tumor mutation objective is described, but no mutation results or analysis details are reported.
Safety interpretation is limited by the absence of event timing, causes of death, treatment-relatedness assessments, and participant-level overlap among listed serious-event terms. The record reports counts and term frequencies but no comparator group. These constraints mean that safety findings can be described as recorded events, not as comparative risks or established effects of the regimen.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: GSK1120212+GSK2141795 for Cervical Cancer — NCT01958112.
- Registry record last updated 2019-05-31; status last verified 2019-05.
- Results first posted 2019-01-02.
- 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
The registry record describes a phase II, single-stage, single-arm study of GSK1120212 (trametinib) plus GSK2141795 for persistent or recurrent cervical cancer. Enrollment was 14; the status is terminated. The recorded regimen was trametinib 1.5 mg QD plus GSK2141795 50 mg QD in 28 day cycles. The primary outcome was response rate assessed by RECIST version 1.1 over 2 Years.
The registry record does not report the primary response result, analysis methods, analysis population, missing-data handling, or a reason for termination. It lists secondary outcomes for progression-free survival, adverse events, mutation and co-mutation rates, and overall survival, without posted analyses. Safety information records 3 participants with serious adverse events, 1 with other adverse events, and 8 deaths among 14 participants. These counts are descriptive; causality and comparative safety are not established by the record.