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 Evaluation of a New Anti-cancer Immunotherapy After Chemotherapy in Adult Patients With Acute Myeloid Leukemia (AML) (NCT00725283), a na phase 1 study. The primary outcome is Number of Patients With Severe Toxicities.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00725283 |
| Phase | Phase 1 |
| Condition | Leukaemia, Myelocytic, Acute |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 34 |
| Arms in the primary outcome | n/a |
| Lead sponsor | GlaxoSmithKline |
| Results posted | Yes |
2. Clinical Question
Population
Adults with WT1-positive acute myeloid leukemia in first complete remission after chemotherapy are the population described in the study summary. The treatment-group description refers to patients with cytologically proven AML defined by the World Health Organization classification. The safety analysis population is described as patients who started treatment and received at least one dose. The registry record does not give further eligibility details or explain how WT1 positivity was established.
Intervention
The intervention was GSK Biologicals’ recombinant WT1 Antigen-Specific Cancer Immunotherapeutic, also identified as GSK2130579A. It was given as post-consolidation therapy. The group description says patients received 24 doses over approximately 4 years. The study summary identifies safety and the induction of an immune response specific to the malignancy as questions of interest.
Comparator
There was no comparator group in this single-group study. Allocation is recorded as not applicable, and masking as none. The study therefore does not make a randomized, like-for-like comparison between treatment strategies. Its posted counts describe patients in the GSK2130579A Group, rather than a treatment effect estimated against a control group.
Primary question
The primary endpoint asks how many treated patients experienced severe toxicities during treatment, from Day 0 to Month 48, under a prespecified definition based on CTCAE Version 3.0 and attribution to study product. The endpoint is a safety count, not a comparison of treatment effectiveness. The registry record does not report the primary endpoint count or a statistical analysis of it.
3. Trial Design
This was a Phase 1, single-group study, with allocation recorded as not applicable and masking recorded as none. The record describes GSK2130579A as post-consolidation therapy for adults with acute myeloid leukemia. The stated purpose was to evaluate safety in adults with WT1-positive AML in first complete remission and to assess the extent to which treatment induced an immune response specific to the malignancy. These aims describe what the study set out to assess; the immune-response results are not included in the registry record.
The registry records enrollment of 34 and describes one GSK2130579A Group. Its arm description says that patients received a standard dose and 24 doses over approximately 4 years. The treatment-period endpoint spans Day 0 to Month 48. The registry lists the study as completed, with a start date of 2008-10-01 and a primary completion date of 2016-06-22. Those dates describe the registered study timeline and do not establish the follow-up duration of each participant.
Because there was only one group and no randomized comparator, the design supports a descriptive account of events observed among treated participants. It does not provide a randomized estimate of how outcomes would differ under another treatment or no treatment. The record does not report a blinding procedure, a control arm, or an allocation scheme beyond the entries noted above. Further protocol details, including dose amount and schedule, are not reported in the registry record. The record identifies GlaxoSmithKline as sponsor.
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 | GSK2130579A Group |
|---|---|
| STARTED | 34 |
| COMPLETED | 10 |
| NOT COMPLETED | 24 |
Reasons for not completing this period:
| Reason | GSK2130579A Group |
|---|---|
| Missing Code | 3 |
| Death | 5 |
| Other | 6 |
| Withdrawal by Subject | 10 |
Analysis population for the primary outcome, as defined in the registry: This analysis was performed on the Total treated population, which included all patients who had started treatment and received at least one dose of the study product, which were included in the overall safety analyses.
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 | GSK2130579A Group |
|---|---|---|
| Participants analysed | 34 | |
| Age, Continuous | Years | 60.1 (SD 12.3) |
| Sex: Female, Male: Female | Participants | 18 |
| Sex: Female, Male: Male | Participants | 16 |
| Race and Ethnicity Not Collected | Participants | — |
6. Primary Endpoint
The registry lists the primary outcome as Number of Patients With Severe Toxicities, measured over During the study treatment period (From Day 0 to Month 48).
| 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 patients with severe toxicities during study treatment, from Day 0 to Month 48. The registry defines the analysis population as the Total treated population: patients who started treatment and received at least one dose, included in the overall safety analyses. The endpoint is a count of participants meeting the toxicity definition, not a count of all individual episodes.
The definition uses CTCAE Version 3.0 and requires an event to be study product-related or possibly study product-related. It includes Grade 4 toxicity, with an exception for Grade 4 fatigue unless it lasted at least 48 hours; Grade 3 toxicity lasting at least 48 hours, with exceptions for myalgia, arthralgia, headache, and fever regardless of duration; and Grade 2 allergic reaction or hypersensitivity, including rash, flushing, urticaria, and dyspnea. Drug fever is excluded. It also includes a decrease in renal function with calculated creatinine clearance below 40 mL/min and Grade 2 cardiac ischemia or infarction, including asymptomatic testing suggesting ischemia or stable angina.
A defined safety endpoint helps specify which events contribute to a summary, rather than treating every reported event as severe toxicity. The registry record does not report the observed count for this primary endpoint, so its frequency in the treated population cannot be described from this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted results characterize adverse events in the GSK2130579A Group. The registry reports 34 patients at risk for serious adverse events and 8 patients with any serious adverse event. It also reports 34 patients at risk for other adverse events and 33 with any such event. These are descriptive counts from a single treatment group. They do not establish how event occurrence compares with another treatment, and they do not by themselves show that treatment caused an event.
The record lists 5 deaths. It does not state in the registry record whether the deaths were related to treatment, nor does it provide timing, causes, or a comparison group. The count should therefore be read as a recorded safety figure, not as a causal estimate. Likewise, the listed serious-event terms—thrombocytopenia, myocardial infarction, abdominal pain, oesophagitis, small intestinal obstruction, and hypersensitivity—each have a reported frequency of 2.9%. The registry record does not provide further detail about the circumstances, duration, or treatment attribution of these individual terms.
The primary endpoint is specifically severe toxicity judged related or possibly related to study product under the stated CTCAE criteria. It should not be conflated with the broader measure of any serious adverse event: the definitions differ, and the record does not provide a cross-tabulation showing which patients met both definitions. The primary severe-toxicity count itself is not reported in the registry record. Consequently, the posted adverse-event counts cannot substitute for that missing primary result.
No statistical analyses are listed in the registry record. There is no reported estimate of comparative effect, confidence interval, or hypothesis test for the primary endpoint. In general, a confidence interval communicates uncertainty around an estimated quantity; without a reported estimate and interval, the precision of the primary result cannot be assessed. The absence of a reported analysis also means that no statistical conclusion about treatment effects can be drawn from these registry entries.
The planned sample-size illustrations described for the posted material are not the study’s own design justification. They 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 fraction of an observed effect. Those assumptions do not match the registry’s single-group design. Such figures are planning illustrations, not observed power and not evidence that this trial was designed or analyzed as a two-arm comparison. The registry record does not report the study’s sample-size justification, interim analyses, or statistical plan.
9. Statistical Methods Explained
The registry describes a single-group Phase 1 study and lists no statistical analyses. The methods information therefore supports a description of the design and endpoints, but not a reconstruction of an inferential analysis. The distinctions below explain how to read the recorded quantities without treating them as evidence of a randomized treatment effect.
What does a participant count represent?
A participant count records how many people meet a defined condition. For the primary endpoint, that condition is having at least one qualifying severe toxicity during the stated treatment period. This differs from counting event episodes: one participant could have more than one event, while the endpoint title asks for the number of patients with severe toxicities. The registry’s population definition is the Total treated population, meaning patients who started treatment and received at least one dose. The primary endpoint result is not reported in the registry record, so the number meeting that definition is unknown from this record.
The adverse-event entries also use participant counts. The record gives counts at risk and affected for serious adverse events and other adverse events. These summaries answer how many patients in the recorded safety group had an event under those categories. They do not reveal the number of episodes per patient, event duration, or whether events overlapped across categories. The registry record does not report those details in the registry record.
What can a one-group study estimate?
In general, a single-group study can describe outcomes observed among people who received an intervention. A comparison intended to estimate how outcomes differ between interventions requires a suitable comparator and an analysis that addresses the design. Here, allocation is listed as not applicable and there is one treatment group. The adverse-event counts are consequently descriptive. Without a control group, differences that might have occurred under another treatment cannot be estimated from these counts.
This design distinction matters even when a count is precise as a record of observed events. A count does not isolate the effect of the intervention from other influences on the outcome. In general, random allocation can help make groups comparable with respect to measured and unmeasured factors, but this study has no randomized comparison. No causal effect estimate is reported. The registry also does not report a statistical model or adjustment strategy.
How should uncertainty be understood?
In general, a confidence interval is a range of values compatible with an estimate under specified statistical assumptions. It communicates uncertainty in addition to the point estimate. A small number of observed events can leave substantial uncertainty about the underlying event frequency, but the registry record does not provide a confidence interval for the primary endpoint or the listed safety counts. No interval should be inferred from the counts alone in this explanation.
In general, a hypothesis test evaluates data against a stated null hypothesis under a specified analysis plan. A p-value is not a measure of the size or clinical importance of an effect. The registry lists no analyses, and no p-value for the primary endpoint is reported. It is therefore not possible to describe a sponsor analysis, a test result, or an adjustment for covariates from this record. The missing analysis details are not evidence that no analysis was conducted; the registry record simply does not report them.
How do the endpoint definitions affect interpretation?
The primary severe-toxicity endpoint is narrower than a general adverse-event count. It is restricted to specified grades, durations, event types, and treatment-attribution categories under CTCAE Version 3.0. For example, some Grade 3 events qualify based on duration, while the definition gives named exceptions. A Grade 2 hypersensitivity event is included under the described criteria, but other Grade 2 events are not automatically included on that basis. Renal and cardiac criteria are also specified. The definition therefore determines which events count, and the broad label “severe toxicity” should be interpreted through those criteria.
Serious adverse events are reported separately. Seriousness and severity are distinct concepts in safety reporting: in general, a seriousness category concerns specified consequences or regulatory criteria, whereas severity describes intensity. The registry’s primary endpoint is based on severity grades and additional rules, while the posted adverse-event summary includes patients with any serious adverse event. The two measures should not be treated as interchangeable. The registry record does not report a mapping between them.
What do the planning illustrations mean?
In general, sample-size planning asks how many participants would be needed under chosen assumptions to detect a target effect with a specified statistical procedure. The planning illustrations described for the posted material use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They use a target effect defined as a stated fraction of an observed effect. These are planning assumptions, not results from the trial and not observed power.
Those assumptions describe a hypothetical two-arm planning setup, whereas this registry record describes a single-group study. They should not be read as evidence that the trial used equal allocation, had a comparator, or was designed around those calculations. The registry record does not report its own sample-size justification or statistical design calculations. It also does not report an interim-analysis plan or analysis details that would explain how the primary endpoint was handled.
General statistical concepts can clarify why design and definition matter, but they cannot fill in absent study results. For this record, the primary severe-toxicity count, its uncertainty, and any formal statistical analysis remain not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 7 outcome measures and no analyses. It does not report how outcomes were prioritized beyond identifying the severe-toxicity count as primary, or whether any formal adjustment was planned for examining multiple outcomes. The registry record does not report a multiplicity strategy, such as a method for controlling error across several statistical tests. Because analyses are not listed, it is not possible to determine whether formal tests were conducted or adjusted.
In general, examining multiple outcomes or multiple comparisons can increase the chance of finding an apparently notable result by chance if each is assessed separately. A prespecified primary outcome can help focus interpretation, while adjustment methods can address some consequences of multiple testing. These are general statistical principles, not claims about the analysis actually used in this study. No specific multiplicity method should be attributed to the investigators from this record.
Missing-data handling is also not reported in the registry record. The safety population is defined as patients who started treatment and received at least one dose, but the record does not explain how incomplete follow-up, withdrawals, missed assessments, or unavailable outcome values were handled. The treatment period is stated as Day 0 to Month 48, but that does not establish that every patient had complete observation across that interval.
The stated safety population definition clarifies who could enter the overall safety analyses; it does not, by itself, document the number assessed at each time point or how missing observations affected the primary endpoint. The registry record also does not describe sensitivity analyses for missing data. These omissions limit assessment of whether the posted counts reflect complete observation or how robust any unreported analysis might be.
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 |
|---|---|---|---|
| GSK2130579A Group | 8 | 34 | 23.5% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| GSK2130579A Group | 5 of 34 |
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 |
|---|---|
| GSK2130579A Group | 33 of 34 |
Most frequent serious adverse event terms
| Event term | Organ system | GSK2130579A Group |
|---|---|---|
| Thrombocytopenia | Blood and lymphatic system disorders | 1 of 34 |
| Myocardial infarction | Cardiac disorders | 1 of 34 |
| Abdominal pain | Gastrointestinal disorders | 1 of 34 |
| Oesophagitis | Gastrointestinal disorders | 1 of 34 |
| Small intestinal obstruction | Gastrointestinal disorders | 1 of 34 |
| Hypersensitivity | Immune system disorders | 1 of 34 |
| Encephalitis | Infections and infestations | 1 of 34 |
| Pneumonia | Infections and infestations | 1 of 34 |
| Upper respiratory tract infection | Infections and infestations | 1 of 34 |
| Viral infection | Infections and infestations | 1 of 34 |
Most frequent non-serious adverse event terms
| Event term | Organ system | GSK2130579A Group |
|---|---|---|
| Injection site pain | General disorders | 22 of 34 |
| Fatigue | General disorders | 17 of 34 |
| Dizziness | Nervous system disorders | 16 of 34 |
| Thrombocytopenia | Blood and lymphatic system disorders | 10 of 34 |
| Diarrhoea | Gastrointestinal disorders | 10 of 34 |
| Upper respiratory tract infection | Infections and infestations | 10 of 34 |
| Pain in extremity | Musculoskeletal and connective tissue disorders | 10 of 34 |
| Constipation | Gastrointestinal disorders | 9 of 34 |
| Nausea | Gastrointestinal disorders | 9 of 34 |
| Back pain | Musculoskeletal and connective tissue disorders | 8 of 34 |
Serious events with the highest arm-level rate: thrombocytopenia (2.9%), myocardial infarction (2.9%), abdominal pain (2.9%), oesophagitis (2.9%), small intestinal obstruction (2.9%).
The registry reports 8 patients with any serious adverse event among 34 at risk in the GSK2130579A Group. For other adverse events, it reports 33 patients affected among 34 at risk. These are separate summaries; the registry entries do not establish whether the same patients appear in both categories. The record also lists 5 deaths, without further details about cause or relationship to study treatment in the registry record.
The serious-event terms listed are thrombocytopenia, myocardial infarction, abdominal pain, oesophagitis, small intestinal obstruction, and hypersensitivity. Each is reported with a frequency of 2.9%. The registry does not provide additional event-level detail in the registry record, so the terms and frequencies should not be interpreted as a complete account of timing, duration, recurrence, or attribution.
These figures are not the primary severe-toxicity result. Serious adverse events and severe toxicities are different outcome definitions, and the primary endpoint count is not reported in the registry record. In this single-group study, the counts describe events recorded among treated patients; they do not establish that the intervention caused an event or show how event rates compare with another group.
12. What the Result Does, and Does Not, Mean
The record documents a Phase 1 study of GSK2130579A as post-consolidation therapy in adults with AML. It describes a safety objective and an immune-response objective, a single treatment group, and a treatment course of 24 doses over approximately 4 years. The posted safety figures indicate that serious and other adverse events were recorded among treated participants, and the record includes a list of serious-event terms with reported frequencies. Those observations describe the experience recorded in the study group.
They do not provide a randomized comparison. The study has no comparator group, so the recorded counts cannot show whether outcomes would have been different under another treatment strategy. Counts alone also do not establish that the intervention caused particular events. The registry record does not report event timing, causal assessments for the deaths, or a comparative statistical estimate in the registry record.
The primary endpoint asks for the number of treated patients with severe toxicities meeting its specific CTCAE-based definition. That result is not reported in the registry record. Neither a confidence interval nor a statistical test for that endpoint is listed. As a result, the record does not support a conclusion about the frequency or precision of the primary endpoint, beyond explaining what it was designed to measure.
The study summary says that immune response specific to the malignancy would also be assessed, but no immune-response results are included in the registry record. Similarly, the record does not report a statistical plan, sample-size justification, or missing-data approach. Those absences should be treated as limits on what can be concluded from the registry record, not as proof that the corresponding work was not done.
Overall, the available information supports a descriptive reading of a single-group safety record. It does not establish comparative efficacy, treatment causation, or a complete assessment of the primary safety endpoint. The distinction between documented study aims, posted adverse-event counts, and unreported analyses is central to interpreting the record.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of how the design determines the questions that can be answered. A single-group study can record the frequency of events among treated participants, but without a comparator it cannot estimate a randomized difference between treatment strategies. The design label is therefore essential context for reading the adverse-event counts: they describe one group rather than a controlled contrast.
The primary endpoint also illustrates why operational definitions matter. The count is not simply a tally of all symptoms or all adverse events. It applies CTCAE Version 3.0 grades, duration rules, named exceptions, treatment attribution, and specified renal and cardiac criteria. A reader needs those details to understand what qualifies as a primary event and why the primary count cannot be substituted with a broader serious-event summary.
The registry record further demonstrates the difference between an endpoint being defined and its result being reported. Although the primary safety outcome is described, its observed count and statistical analysis are not reported. The separate adverse-event figures do not fill that gap, and the listed planning assumptions do not constitute observed power or a description of the trial’s own statistical design.
For biostatisticians and clinical researchers, these distinctions help prevent overinterpretation. Design, outcome definition, analysis reporting, and uncertainty all shape the meaning of a result. In this record, the registry entries permit a cautious description of the study and its posted safety counts, while leaving the primary endpoint result and important analysis details unresolved.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The most direct limitation for interpreting the primary safety question is that the observed severe-toxicity count is not reported in the registry record. The record describes the endpoint in detail but does not provide its result, uncertainty, or statistical analysis. The adverse-event counts cannot be used as substitutes because they refer to different categories.
The single-group design is another important limitation for interpretation. There is no randomized comparator, so the record cannot estimate a treatment-versus-control difference from the posted counts. Attribution of individual events is also limited in the information: the primary endpoint includes events judged related or possibly related to study product, but the primary result is absent, and further attribution details for the listed serious events are not reported.
The record gives the treatment period as Day 0 to Month 48 and describes 24 doses over approximately 4 years. It does not report how complete follow-up was for each participant, how missing observations were handled, or whether outcome ascertainment differed over time. It also does not provide event timing, duration, recurrence, or detailed causes for the listed deaths in the registry record.
No analysis methods, multiplicity approach, interim-analysis plan, or sample-size justification are reported in the registry record. The listed planning assumptions describe a hypothetical two-arm illustration and should not be mistaken for the design or analysis of this single-group study. These limits mean that the record supports only a descriptive interpretation of the posted safety figures and a clear account of what the primary endpoint was intended to measure.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Evaluation of a New Anti-cancer Immunotherapy After Chemotherapy in Adult Patients With Acute Myeloid Leukemia (AML) — NCT00725283.
- Registry record last updated 2018-08-07; status last verified 2017-06.
- Results first posted 2018-08-07.
- 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 describes NCT00725283 as a completed Phase 1 study sponsored by GlaxoSmithKline. It evaluated GSK2130579A, a WT1 antigen-specific cancer immunotherapeutic, as post-consolidation therapy in adults with AML. The design is single-group, with allocation not applicable and no masking. The record lists enrollment of 34 and describes 24 doses over approximately 4 years.
The primary outcome is the number of patients with defined severe toxicities during treatment from Day 0 to Month 48. The primary outcome count and any statistical analysis are not reported in the registry record. Posted safety figures include 8 patients with any serious adverse event among 34 at risk, 33 with other adverse events among 34 at risk, and 5 deaths. These are descriptive figures from a single group, not a randomized comparison.