This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Study of GSK2132231A Antigen-Specific Cancer Immunotherapeutic in Patients With Inoperable Metastatic Cutaneous Melanoma (NCT00896480), a na phase 2 study. The primary outcome is Number of Patients With Objective Tumor Response (OR) to GSK2132231A Study Treatment.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00896480 |
| Phase | Phase 2 |
| Condition | Melanoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 24 |
| Arms in the primary outcome | n/a |
| Lead sponsor | GlaxoSmithKline |
| Results posted | Yes |
2. Clinical Question
Population
The registry record describes patients with unresectable and progressive metastatic cutaneous melanoma. The primary endpoint population is the Total Treated population: patients who received at least one dose of GSK2132231A. The arm description specifies male or female participants aged 18 years or older. The record lists an enrollment of 24.
Intervention
The intervention was the GSK2132231A cancer immunotherapeutic, administered intramuscularly. The arm description says participants received up to 24 doses in 4 cycles. It gives some cycle timing and dose information, but the available description ends partway through the schedule.
Comparator
There was no comparator group. The study is recorded as a single-group study with allocation marked NA and masking listed as none. Consequently, the design does not provide a concurrent control group for comparison with GSK2132231A.
Primary question
The primary question was how many treated participants had an objective tumor response, defined as a best overall response of complete response or partial response. The endpoint timeframe extended from pre-treatment to study end, identified as Year 4, with censoring at the first report of disease progression in assessed lesions. The registry record does not report the observed response count.
3. Trial Design
This is a completed Phase 2, single-group study of GSK2132231A in patients with unresectable and progressive metastatic cutaneous melanoma. The registry record lists GlaxoSmithKline as the sponsor, an enrollment of 24, a start date of 2009-05-19, and a primary completion date of 2014-11-03. Allocation is recorded as NA, and masking as none. There is no randomized or concurrent control arm in the design described by the record.
The intervention is identified as a biological product. The arm description says participants aged 18 years or older received intramuscular doses in 4 cycles, up to 24 doses. It describes Cycle 1 as ending at Week 13, with 6 doses at 2-week intervals, and Cycle 2 as ending at Week 32, with 6 doses at 3-week intervals. It starts to describe Cycle 3, but the available arm description is incomplete. The full schedule, including the omitted details, is not reported in the registry record.
The stated clinical objectives include tumor response and time to treatment failure, along with safety and immune response. Translational objectives concern biological variables. These objectives describe the scope of planned evaluation; they do not establish what results were observed. The registry record lists 33 outcome measures, but no analysis entries. The primary endpoint is a participant count based on best overall tumor response. The record does not describe a comparator-based treatment effect estimate, and the single-group design cannot directly estimate a difference against an untreated or alternative-treatment group.
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.
Before assignment: During the screening the following steps occurred: check for inclusion criteria, contraindications/precautions, medical history of the subjects and signing informed consent forms.
Overall Study
| Stage | GSK2132231A Group |
|---|---|
| STARTED | 24 |
| COMPLETED | 3 |
| NOT COMPLETED | 21 |
Reasons for not completing this period:
| Reason | GSK2132231A Group |
|---|---|
| Disease progression/recurrence | 21 |
Analysis population for the primary outcome, as defined in the registry: The Total Treated population included all patients who have received at least one Dose of the GSK2132231A 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 | GSK2132231A Group |
|---|---|---|
| Participants analysed | 24 | |
| Age, Continuous | Years | 65.4 (SD 12.4) |
| Sex: Female, Male: Female | Participants | 17 |
| Sex: Female, Male: Male | Participants | 7 |
| Race and Ethnicity Not Collected | Participants | — |
6. Primary Endpoint
The registry lists the primary outcome as Number of Patients With Objective Tumor Response (OR) to GSK2132231A Study Treatment, measured over From Pre-treatment (up to 4 weeks before first treatment) to study end (Year 4), each patient being censored out of the analysis at 1st report of disease progression in assessed lesions.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants with an objective tumor response to GSK2132231A. Objective response is defined as the best overall response in a participant being complete response or partial response. The registry description places these categories within a response classification that also includes stable disease, SD/PR, progressive disease, and non-evaluable status. Complete response means disappearance of all target lesions. Partial response requires at least 30% decrease in the sum of the longest diameters of target lesions. The endpoint uses RECIST v1.0 criteria for target lesions assessed by magnetic-resonance imaging.
A response count is a way to describe how many treated participants met the specified tumor-response definition during the assessment period. It is clinically interpretable as a summary of observed response under the study's assessment rules, but it is not by itself a comparison with another treatment or with no treatment. The registry defines the timeframe as pre-treatment, up to 4 weeks before the first treatment, through study end, identified as Year 4. Each patient is censored from the analysis at the first report of disease progression in assessed lesions.
The registry record does not report the number with complete response, partial response, or objective response. It therefore does not permit a description of the observed primary endpoint result or its precision.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies the primary outcome as a count of participants with objective tumor response, but it does not report the observed count. It also does not report a response estimate, confidence interval, or statistical test for this endpoint. The main conclusion supported by the record is therefore about the study question and design, not the magnitude of observed tumor response. No response rate or treatment effect can be stated from the record information available.
The enrollment is listed as 24, and the endpoint population is defined as the Total Treated population: participants who received at least one dose. Enrollment and the endpoint population definition are not interchangeable. The record does not provide the number who received treatment, the number assessed for response, or the number included in the primary endpoint analysis. It also does not provide the response-category counts. Those omissions prevent assessment of how the enrolled group contributed to the endpoint.
The study has no control group. A response count in a single group can describe outcomes observed under the study conditions, but it cannot by itself distinguish effects of the study treatment from other influences on tumor response. Nor does the record provide a comparative estimate for time to treatment failure. The stated study objectives include that outcome, but its definition and result are not reported in the registry record information presented.
General statistical context: estimates based on participant counts have sampling uncertainty. A confidence interval, when reported, expresses a range of values compatible with the data and the statistical method; it is not a guarantee that the true value lies in that range. Without the observed count and an uncertainty estimate, the precision of the primary result cannot be evaluated. A small or large-looking count, considered without its denominator and assessment details, would not be enough to judge precision. The registry record does not give the denominator analyzed for the endpoint.
Safety information is reported separately: the record lists participants affected in serious and other adverse-event categories, along with serious adverse-event terms. These entries describe recorded events, not an estimate of causal risk relative to a comparator. Because the study is single-group and the record does not provide causal attribution details, the safety figures should not be interpreted as proof that the intervention caused an event or prevented one.
Overall, the registry record supports a description of the trial's intended primary endpoint, eligibility context, treatment schedule as far as recorded, and selected safety counts. It does not support a numerical conclusion about objective response, comparative benefit, or the precision of the endpoint estimate. The sponsor's own endpoint analysis is not included in the registry information, so no recalculated result is available to set alongside it.
9. Statistical Methods Explained
Endpoint type and estimand. The primary endpoint is a count of participants meeting a binary response definition: best overall response is complete response or partial response. A binary endpoint places each evaluated participant into a category for the purpose of the outcome, even when the underlying clinical assessment has multiple response categories. The registry describes several categories, including stable disease, SD/PR, progressive disease, and non-evaluable status, while defining objective response as complete response plus partial response. It does not report how many participants fell into any category.
In general, an estimand specifies the treatment condition, population, outcome, and handling of events or missing assessments that define the clinical question. The record identifies the intervention, the Total Treated population, the response definition, and a timeframe. It also states censoring at the first report of disease progression in assessed lesions. Details that would fully clarify how all intercurrent events and missing response assessments were handled are not reported in the registry record. That matters because outcome rules can affect which observations contribute to a summary and how the result should be interpreted.
Response assessment. The registry defines complete response as disappearance of all target lesions and partial response as at least 30% decrease in the sum of the longest diameters of target lesions. Stable disease is described as neither sufficient shrinkage for partial response nor sufficient increase for progressive disease. Progressive disease is defined using at least 20% increase in the sum of largest diameters for target lesions. The assessment is described as using RECIST v1.0 for target lesions and magnetic-resonance imaging. These criteria help make response classification more explicit, but the record does not report assessment timing beyond the overall timeframe, the number of assessments, or adjudication procedures.
What a count can tell us. A participant count directly reports how many individuals met a specified outcome, provided both the count and analysis denominator are known. A proportion can sometimes be useful for interpreting a binary outcome, but the record does not report the primary outcome count or its analysis denominator. No proportion should be inferred from enrollment alone: the primary population is defined by treatment received, and the record does not state how many enrolled participants received treatment or were evaluable. No count, proportion, or uncertainty interval is recalculated.
General statistical context: a confidence interval is an uncertainty summary around an estimate. For a binary endpoint, interval methods account for the fact that outcomes are counts and that the estimated proportion is not known without uncertainty. Different methods can yield different intervals, particularly when counts are sparse. The registry record does not report a confidence interval or identify a method for one. It also does not report a hypothesis test or p-value for the primary endpoint.
Single-group design. The study is recorded as single-group, with allocation marked NA and masking listed as none. In a single-group design, the observed outcome is described within the treated group; there is no concurrent control group to provide a direct estimate of what would have happened under another treatment or without the study treatment. Historical comparisons, if made elsewhere, would require information about comparability and methods not reported in the registry record. No such comparison is described in the available record.
General statistical context: random allocation can help balance measured and unmeasured prognostic factors between groups on average, while masking can reduce some assessment or reporting biases. Those design features are not listed for this study as a randomized or masked comparison. Their absence does not determine the direction or size of any bias, but it limits the kinds of causal conclusions a single-group result can support.
Time-to-event outcomes. The trial objectives mention time to treatment failure, but the primary endpoint described in this record is a participant count for objective tumor response. Time-to-event analysis generally concerns the time until a defined event and must specify how participants without an event by the end of observation are handled. The event definition, censoring details, analysis method, and results for time to treatment failure are not reported in the registry record information available. No time-to-event estimate can therefore be interpreted from these registry entries.
Planning and observed data. A sample-size calculation is a planning exercise based on assumptions such as a target effect, significance level, allocation, and statistical approximation. It is not an observed result and is not a measure of the study's achieved statistical power. No trial-specific design justification or sample-size calculation is reported in the registry record. The record's enrollment figure does not by itself establish whether the study was adequately sized for its objectives. Any planning illustrations associated with other results would need to be read as hypothetical planning scenarios, not as observed evidence or as the trial's original design rationale.
General statistical context: over-dispersion is relevant when observed variability exceeds what a chosen model assumes. It can make standard errors too small if ignored. The primary outcome is described as a participant count, but the record does not report a model, dispersion estimate, or analysis details that would permit assessment of over-dispersion. It would not be appropriate to assume a particular model from the endpoint label alone.
Analyses and reporting. The registry record lists 33 outcome measures and no analysis entries. That does not establish which analyses were conducted outside the registry, whether results were published elsewhere, or whether any prespecified analysis was changed. The statistical plan, interim analyses, adjustment methods, and model specifications are not reported in the registry record. Without those details and without the observed endpoint result, readers cannot assess the analysis choices or reproduce an inferential result from the record.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 33 outcome measures, while no analysis entries are listed. This creates an important distinction between the number of outcomes registered and the number of analyses reported in this record. The record does not identify which outcomes were designated as primary beyond the objective tumor response measure, nor does it state whether multiple hypotheses were tested, whether outcomes were analyzed at multiple time points, or whether any multiplicity adjustment was used. The statistical analysis plan and any interim-analysis plan are not reported in the registry record.
General statistical context: when many outcomes or comparisons are tested, the chance of at least one apparently unusual result can increase. Multiplicity procedures can address that issue, but the appropriate approach depends on the planned questions and analysis strategy. Without the analysis plan and reported analyses, it is not possible to determine whether multiplicity was relevant to the reported conclusions or how it was handled.
Information about missing endpoint assessments is also not reported. The endpoint population is defined as participants who received at least one dose, but the record does not state how many participants had response data, how many were non-evaluable, or how missing assessments were treated in the primary analysis. Non-evaluable is included as a response classification, but its count and analysis handling are not given. The registry also does not report whether sensitivity analyses assessed alternative assumptions about missing data.
General statistical context: missing outcomes can affect interpretation when the likelihood of missingness is related to health status or response. The direction and magnitude of any resulting bias depend on the pattern of missingness and the analysis method. Since those details are not reported, the extent to which missing data could affect the endpoint cannot be assessed 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 |
|---|---|---|---|
| GSK2132231A Group | 2 | 24 | 8.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| GSK2132231A Group | 0 of 24 |
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 |
|---|---|
| GSK2132231A Group | 23 of 24 |
Most frequent serious adverse event terms
| Event term | Organ system | GSK2132231A Group |
|---|---|---|
| Cardiac disorder | Cardiac disorders | 1 of 24 |
| Metastatic malignant melanoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 24 |
Most frequent non-serious adverse event terms
| Event term | Organ system | GSK2132231A Group |
|---|---|---|
| Injection site pain | General disorders | 12 of 24 |
| Injection site reaction | General disorders | 12 of 24 |
| Pyrexia | General disorders | 10 of 24 |
| Asthenia | General disorders | 9 of 24 |
| Fatigue | General disorders | 7 of 24 |
| Headache | Nervous system disorders | 7 of 24 |
| Influenza like illness | General disorders | 7 of 24 |
| Nausea | Gastrointestinal disorders | 7 of 24 |
| Chills | General disorders | 4 of 24 |
| Injection site erythema | General disorders | 4 of 24 |
Serious events with the highest arm-level rate: cardiac disorder (4.2%), metastatic malignant melanoma (4.2%).
The registry record lists 24 participants at risk in the GSK2132231A Group for the serious adverse-event category, with 2 affected. For the other adverse-event category, it lists 24 at risk and 23 affected. The record lists 0 deaths. These are recorded counts; the available information does not explain event timing, severity grading beyond the serious classification, recurrence, or causal attribution to the study treatment.
The serious adverse-event terms with the highest listed percentages are cardiac disorder at 4.2% and metastatic malignant melanoma at 4.2%. These percentages are registry entries and should not be treated as comparative risks. The record has no control group, and it does not provide a comparison of adverse-event frequency under another treatment or under no treatment. It also does not clarify whether the listed terms refer to participants with an event, event episodes, or how overlapping terms were handled. The percentages should therefore be read as descriptive registry figures, not as evidence that the intervention caused or prevented these events.
General statistical context: an adverse event is an observation during a study and is not automatically attributable to the intervention. Seriousness is a classification, not a statement of causality. A fuller safety interpretation would require definitions, exposure and follow-up context, event-level details, and attribution information, which are not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The trial asks whether participants with unresectable and progressive metastatic cutaneous melanoma experienced an objective tumor response during treatment with GSK2132231A. The registry defines that response using complete response or partial response as the best overall response, with RECIST v1.0 criteria for target lesions. It also specifies a treatment-received population and an assessment timeframe extending to study end, identified as Year 4.
What the registry record does not establish is the observed answer to that primary question. The response count, response-category counts, analysis denominator, and an uncertainty estimate are not reported. The record therefore cannot support a numerical statement about how often objective response occurred or how precisely it was estimated. It also cannot establish a treatment effect relative to another option, because the design is single-group and has no comparator.
The intervention schedule is described as intramuscular administration in 4 cycles, with up to 24 doses, but the available arm description is incomplete. The record states safety and immunogenicity objectives, among other objectives, but the information available does not include their full results. The safety entries can be reported descriptively, while causal interpretation remains limited by the absence of a comparison group and event-attribution details.
General statistical context: a trial result is most informative when the endpoint definition, analysis population, observed estimate, uncertainty, and analysis method can be considered together. A registered objective is not itself a finding, and an enrollment figure is not a substitute for an endpoint result. Likewise, a count without its analysis denominator or uncertainty does not show how stable the estimate may be.
The record supports a careful account of the study's design and intended questions, but not a conclusion that GSK2132231A did or did not produce a particular level of response. The absence of a reported primary outcome in this registry record should be distinguished from evidence of no response. It means the outcome result is not reported in the registry record, rather than showing that the outcome was zero.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of the difference between a registered clinical question and a reported result. The record gives a clearly stated primary endpoint based on objective tumor response, with defined response categories and an assessment framework. Yet the endpoint count and its analysis details are not reported. Readers therefore need to distinguish a specified outcome from an available estimate.
The single-group design also makes the target of interpretation important. A participant count can describe outcomes within the treated group, but without a comparator it cannot directly quantify a difference attributable to treatment. This distinction is especially relevant when an endpoint is framed as clinical activity: evidence about observed activity within a group and evidence about comparative treatment effects answer different statistical questions.
The registry record also illustrates why population definitions matter. The primary endpoint population includes participants who received at least one dose, while the record's enrollment figure does not state how many participants were treated or assessed. Without the analysis denominator, even a response count would be difficult to interpret fully. Reporting participant flow, evaluability, and missing outcome handling would help readers understand how the endpoint population relates to enrollment.
Finally, the record includes safety counts and several outcome measures but no analysis entries. That makes it important to separate descriptive event reporting from causal claims and to recognize that the statistical plan and multiplicity handling are not reported. These are not grounds for assuming that analyses were absent in all settings; they are limitations of what can be assessed from the registry record. The trial matters statistically because it underscores how design, endpoint definitions, denominators, uncertainty, and complete results reporting jointly determine what conclusions a clinical study can support.
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 limitation for interpreting the primary endpoint is that the registry record does not report the observed objective response count. It also does not give the number analyzed, response-category counts, a confidence interval, or a statistical test. The endpoint is defined, but its result and precision cannot be evaluated from the record information available.
The design is single-group, with no concurrent comparator. Any outcome observed in the treated group would not, by itself, establish a difference from another treatment or from no treatment. Masking is listed as none, and the record does not describe independent response review or other procedures that might address assessment subjectivity. Such procedures should not be assumed to have been used.
The endpoint population is defined by receipt of at least one dose, but participant flow and the number with evaluable response assessments are not reported. The handling of non-evaluable assessments, missing data, discontinuations, and progression-related censoring beyond the stated rule is not described. The registry also does not provide sufficient detail to evaluate outcome timing and assessment frequency.
The arm description gives a partial treatment schedule and ends partway through its description of Cycle 3. The complete schedule is not reported in the registry record information available. Although the record lists multiple outcome measures, it does not provide analysis entries or the statistical analysis plan. Consequently, the approach to multiplicity, interim analyses, and model selection cannot be assessed. The safety information is limited to aggregate entries and selected serious-event terms; event-level details and causal attribution are not reported. These limitations restrict interpretation of both efficacy and safety, without implying that unreported information was not collected elsewhere.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study of GSK2132231A Antigen-Specific Cancer Immunotherapeutic in Patients With Inoperable Metastatic Cutaneous Melanoma — NCT00896480.
- Registry record last updated 2021-04-08; status last verified 2021-03.
- Results first posted 2019-09-27.
- 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 completed Phase 2, single-group study of GSK2132231A in unresectable and progressive metastatic cutaneous melanoma. GlaxoSmithKline is listed as sponsor, enrollment is 24, and the record gives study start as 2009-05-19 and primary completion as 2014-11-03. Allocation is NA and masking is none.
The primary endpoint is the number of treated participants with an objective tumor response, defined as complete response or partial response. The endpoint result, analysis denominator, and uncertainty estimate are not reported in the registry record. The record lists 33 outcome measures and no analysis entries.
Safety entries list 2 affected among 24 at risk for serious adverse events, 23 affected among 24 at risk in the other adverse-event category, and 0 deaths. Cardiac disorder and metastatic malignant melanoma are listed as serious adverse-event terms, each at 4.2%. The single-group design and limited reporting support descriptive interpretation only; a comparative treatment effect cannot be determined from this record.