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 Study of ICT-107 Immunotherapy in Glioblastoma Multiforme (GBM) (NCT01280552), a randomized phase 2 study. The primary outcome is Overall Survival (OS).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01280552 |
| Phase | Phase 2 |
| Condition | Glioblastoma Multiforme |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Triple |
| Enrollment | 124 |
| Arms in the primary outcome | 2 |
| Lead sponsor | Precision Life Sciences Group |
| Results posted | Yes |
2. Clinical Question
Population
Newly diagnosed patients with glioblastoma multiforme (GBM) following resection and chemoradiation. The study description says patients had not yet received chemoradiation at entry. The primary analysis population is described as all randomized patients under an intent-to-treat approach.
Intervention
ICT-107, an immunotherapy made from a patient's own dendritic cells exposed to immunogenic peptides from tumor antigens. The study description says the vaccine was intended to stimulate an immune response against remaining tumor cells after surgery and chemotherapy.
Comparator
Control dendritic cells, described as autologous dendritic cells not pulsed with antigens. The registry also names this intervention as Placebo DC. The reported control arm is titled Control Dendritic Cells.
Primary question
How did overall survival compare between patients assigned to ICT-107 and those assigned to control dendritic cells? Overall survival is defined as time from randomization until death, or until the last date a patient was known to be alive if death was not observed. The posted arm summaries report median survival and 95% confidence intervals.
The trial record describes a randomized, double-blind, controlled phase IIb study with a parallel-group design. Its stated objective was to compare overall survival after treatment with ICT-107 or control. The registry lists an enrollment of 124 and reports results for both randomized arms. Randomization is intended to make assigned groups comparable on average at the time of allocation; it does not guarantee that every prognostic characteristic is balanced in a particular trial.
The primary question is about survival time, not simply whether a participant was alive at one fixed visit. This distinction matters because participants can have different lengths of follow-up, and some may be alive when observation ends. The endpoint definition accounts for that by using the last date known alive when a death is not observed. The registry record does not report the detailed follow-up distribution or the number of deaths in each arm.
The results describe median overall survival of 18.3 months for ICT-107 and 16.7 months for Control Dendritic Cells. Those estimates are accompanied by broad 95% confidence intervals, and the record does not post a hazard ratio or a p-value for the primary comparison. The medians alone therefore do not establish the statistical significance or clinical importance of a treatment difference.
3. Trial Design
This was a randomized, double-blind, controlled phase IIb study with a parallel design. Participants were assigned to ICT-107 or control dendritic cells. The ICT-107 product was described as autologous dendritic cells pulsed with immunogenic peptides from tumor antigens; the control product was described as autologous dendritic cells without antigen pulsing. The masking level is recorded as triple, but which individuals or groups were masked is not reported in the registry record.
The study concerned newly diagnosed glioblastoma multiforme after resection and chemoradiation. Its brief description says eligible patients had not yet received chemoradiation at the time of entry and describes the vaccine as being given after surgery and chemotherapy. The record identifies the trial as phase 2 and reports an enrollment of 124. The primary endpoint was overall survival, defined from randomization until death or the last date known alive for participants without an observed death. The stated primary analysis population was all randomized patients, described as intent to treat.
Random assignment supports a comparison of outcomes according to assigned group and helps limit confounding from baseline differences. A parallel design means each participant is assigned to one study arm rather than receiving both study interventions in sequence. Blinding can reduce the influence of expectations on care or outcome assessment, although the registry record does not specify who was masked or how masking was maintained.
The registry lists progression-free survival and progression-free survival in HLA-A2 patients as secondary outcomes. It reports four outcome measures and two analyses, but does not give enough detail to identify the complete analysis plan, the definitions for all secondary measures, or how those analyses were conducted. The protocol's sample-size justification, interim monitoring plan, and detailed allocation procedures are not reported in the registry record.
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 | ICT-107 | Placebo |
|---|---|---|
| STARTED | 81 | 43 |
| COMPLETED | 75 | 42 |
| NOT COMPLETED | 6 | 1 |
Analysis population for the primary outcome, as defined in the registry: Intent to treat include all randomized patients
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | ICT-107 | Placebo | Total |
|---|---|---|---|---|
| Participants analysed | 81 | 43 | 124 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 59 | 29 | 88 |
| Age, Categorical: >=65 years | Participants | 22 | 14 | 36 |
| Sex: Female, Male: Female | Participants | 37 | 12 | 49 |
| Sex: Female, Male: Male | Participants | 44 | 31 | 75 |
| Region of Enrollment: United States | participants | 81 | 43 | 124 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Survival (OS), measured over 2 -3 years.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 95% Confidence Interval |
| Unit | months of survival |
| Endpoint classification | Time-to-Event Analysis |
The primary endpoint is overall survival, measured in months from randomization until death. When a death was not observed, the endpoint definition uses the last date the participant was known to be alive. This is a time-to-event outcome: the event is death, and the analysis must also account for participants whose event has not been observed by the end of their available follow-up.
The posted results summarize each arm with a median and a 95% confidence interval. A median survival is the estimated time by which half of the relevant population has experienced the event and half has not, under the survival-analysis definition. It is not the average survival time, and it does not indicate that every participant survived for that duration.
Overall survival is commonly used in trials because it captures a direct outcome over time rather than relying only on a measure of tumor status or symptoms. That general rationale does not establish how the endpoint was selected for this particular protocol; the protocol's detailed rationale is not reported in the registry record. The listed timeframe is “2 -3 years,” but that alone does not describe the completeness or length of individual follow-up.
The record reports median overall survival of 18.3 months for ICT-107 and 16.7 months for control, with respective 95% confidence intervals. It does not report a primary-comparison hazard ratio, p-value, or detailed survival curve information in the available results. The medians therefore provide a concise description of the observed survival distributions, but not a complete estimate of the relative treatment effect.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry reports median overall survival of 18.3 months in the ICT-107 arm, with a 95% confidence interval from 14.9 to 21.2 months. In the Control Dendritic Cells arm, the median was 16.7 months, with a 95% confidence interval from 12.3 to 23.0 months. These are sponsor-posted arm summaries for the trial's stated intent-to-treat population. The registry record does not report a hazard ratio or p-value for the primary comparison.
A median describes the center of a survival distribution, but the two medians do not describe the full shapes of those distributions. They do not show how survival varied across participants, when events occurred, or how much follow-up was available for those who had not died. A comparison based only on the two medians also does not account for the timing of every event in the way a full time-to-event analysis can.
The confidence intervals communicate uncertainty around each arm's median estimate. The interval for the control arm is broad, extending from 12.3 to 23.0 months, while the ICT-107 interval extends from 14.9 to 21.2 months. Both intervals cover a range of plausible values for the underlying arm-specific median, given the estimation procedure and its assumptions. They are not ranges containing a fixed proportion of individual participants' survival times.
Confidence intervals for each arm should not be treated as a formal test of the difference between arms. Their overlap, or lack of overlap, is not a substitute for a between-group analysis. The registry record does not post a hazard ratio, a confidence interval for a comparative effect, or a p-value for overall survival. As a result, the available summaries do not establish whether the primary comparison met a prespecified statistical threshold, and the threshold itself is not reported in the record.
The randomization and stated intent-to-treat population support interpreting the results as a comparison by assigned group, provided the analysis was conducted as described. The record does not give details about adherence, treatment received after allocation, or handling of participants with incomplete outcome information. These details can affect interpretation of a trial result, especially when the endpoint depends on follow-up over time.
The numerical medians should not be read as proof that one intervention is better or worse. They describe the posted arm estimates, with substantial uncertainty, and do not by themselves indicate causation or clinical importance. Overall, the registry supplies an arm-level survival summary, but not the comparative statistics and analysis details needed for a complete assessment of the primary treatment contrast.
9. Statistical Methods Explained
What does a median survival estimate mean?
For a time-to-event endpoint, the median is the point on the estimated survival distribution at which the estimated proportion still free of the event reaches one half. In this trial, the event is death. A median is not an arithmetic average and does not imply that all participants experienced an event at that time. Some participants may still be alive when their follow-up ends; their available information is treated as censored rather than as a death at the final observation date.
The registry reports a median of 18.3 months for ICT-107 and 16.7 months for Control Dendritic Cells. These are separate summaries for the two assigned arms. The record does not describe the estimator, the number of deaths, the number censored, or the survival probabilities at particular time points. Those omissions limit the ability to reconstruct or independently assess the survival curves from the medians alone.
How should the confidence intervals be read?
A confidence interval expresses uncertainty about an estimated quantity. In repeated use of a specified method under its assumptions, a 95% confidence interval is designed to include the true parameter in 95% of such intervals. It does not mean there is a 95% probability that the particular interval contains the fixed true value, and it is not a prediction interval for individual survival times.
The ICT-107 median is reported with a 95% confidence interval from 14.9 to 21.2 months. The control median is reported with a 95% confidence interval from 12.3 to 23.0 months. These intervals convey uncertainty in each arm's median, not uncertainty in a direct difference between arms. A formal comparative interval would need to be calculated from a between-group analysis; such a result is not reported in the registry record.
Why is a time-to-event comparison different from comparing medians?
Time-to-event methods use event timing and can incorporate participants who have not experienced the event by the time their observation ends. A common comparative summary in this setting is a hazard ratio, which describes the relative event rate over time under the model used. Its interpretation depends on the analysis and assumptions, including whether the relative rate is reasonably stable over time. The registry record does not report a hazard ratio for this primary endpoint, so no such estimate can be interpreted from the posted results.
A comparison of medians compresses each survival distribution to one point. It can conceal differences in the shape of the curves, including whether separation varies over follow-up. It also does not by itself provide a test of the null hypothesis of no treatment effect. A p-value, when reported, addresses compatibility of data with a specified null model under stated assumptions; it does not measure the probability that the null hypothesis is true or the size of a clinically meaningful effect. No primary p-value is reported in the registry record.
What role does randomization play?
Randomization assigns participants to groups by chance. Its purpose is to reduce systematic differences between arms at allocation, including differences in both measured and unmeasured prognostic factors, on average over repeated trials. It does not ensure exact balance in any one trial. The randomized design makes an assigned-group comparison relevant, but sound interpretation also depends on following participants and analyzing their outcomes appropriately.
The primary population is described as intent to treat and includes all randomized patients. In general, an intent-to-treat analysis retains participants in the groups to which they were assigned, regardless of later treatment adherence or changes in care. That approach preserves the benefits of randomization for the main comparison. The registry record does not describe the detailed implementation, exclusions, or handling of incomplete survival information.
Why can over-dispersion matter in general?
Over-dispersion is a general statistical issue that arises when the variability in data is greater than a model assumes. If a model underestimates variability, standard errors can be too small and confidence intervals too narrow, which may make evidence appear more precise than it is. Appropriate modeling or robust variance methods can address this in settings where it is relevant. The registry record does not report an over-dispersion issue or the use of a method to address one; this general concept should not be taken as a description of the trial's analysis.
For survival analyses, other assumptions can also matter, including how censoring relates to outcomes and whether the chosen model is appropriate. The record does not provide enough detail to evaluate those assumptions. The arm-specific confidence intervals communicate some estimation uncertainty, but they cannot replace a full account of the analysis method and its diagnostics.
What can be concluded from the posted statistics?
The posted statistics support a descriptive statement about the two arm-specific medians and their uncertainty. They do not establish statistical significance, a hazard ratio, or the magnitude of a comparative effect. The trial's detailed statistical analysis plan, model specification, and any sensitivity analyses are not reported in the registry record. A careful reading therefore separates the reported descriptive estimates from comparative claims that require statistics not posted in the record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists progression-free survival and progression-free survival in HLA-A2 patients as secondary outcomes. It also reports four outcome measures and two analyses. The record does not describe the full set of hypotheses tested, the order in which outcomes were analyzed, or whether any testing hierarchy was used. It does not report a multiplicity adjustment or explain how the risk of false-positive findings across multiple outcomes or analyses was handled.
Multiplicity is a general concern when investigators evaluate several outcomes or perform several statistical tests. The more tests considered, the greater the chance that at least one result may appear statistically notable by chance if each is judged separately. Prespecified testing strategies can help control that risk. The absence of a reported strategy does not establish that no strategy was used; it means the approach is not reported in the registry record.
The primary endpoint description states that all randomized patients are included in an intent-to-treat analysis. This identifies the intended primary population, but the record does not describe the amount or pattern of missing outcome information, the methods used to handle incomplete follow-up, or any sensitivity analyses. For survival outcomes, participants without an observed death may contribute information up to their last known-alive date, as described in the endpoint definition. The details of censoring and any other data-handling decisions are not reported.
The record does not report a sample-size justification, interim analysis plan, stopping rule, or detailed statistical analysis plan. These omissions prevent an assessment of whether the enrollment target was based on a particular assumed effect or how the study was intended to manage repeated looks at the data. The posted results also do not include a primary comparative p-value or hazard ratio. These missing details should not be replaced by assumptions about what the protocol might have specified.
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 |
|---|---|---|---|
| ICT-107 | 8 | 80 | 10.0% |
| Placebo | 7 | 43 | 16.3% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5.0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| ICT-107 | 59 of 80 |
| Placebo | 40 of 43 |
Most frequent serious adverse event terms
| Event term | Organ system | ICT-107 | Placebo |
|---|---|---|---|
| seizure | Nervous system disorders | 8 of 80 | 7 of 43 |
| infection | Infections and infestations | 4 of 80 | 4 of 43 |
| intracranial hemorrhage | Nervous system disorders | 4 of 80 | 0 of 43 |
| cerebral edema | Nervous system disorders | 2 of 80 | 1 of 43 |
| pulmonary embolism | Respiratory, thoracic and mediastinal disorders | 0 of 80 | 3 of 43 |
| increased intracranial pressure | Nervous system disorders | 2 of 80 | 0 of 43 |
Most frequent non-serious adverse event terms
| Event term | Organ system | ICT-107 | Placebo |
|---|---|---|---|
| fatigue | General disorders | 12 of 80 | 11 of 43 |
| convulsion | Nervous system disorders | 11 of 80 | 7 of 43 |
| nausea | Gastrointestinal disorders | 6 of 80 | 4 of 43 |
| headache | Nervous system disorders | 2 of 80 | 7 of 43 |
| musular weakness | Musculoskeletal and connective tissue disorders | 2 of 80 | 6 of 43 |
| urinary tract infection | Infections and infestations | 5 of 80 | 2 of 43 |
| insomnia | Psychiatric disorders | 3 of 80 | 4 of 43 |
| thrombocytopenia | Blood and lymphatic system disorders | 3 of 80 | 3 of 43 |
| hemiparesis | Nervous system disorders | 4 of 80 | 2 of 43 |
| partial seizure | Nervous system disorders | 5 of 80 | 1 of 43 |
Serious events with the highest arm-level rate: seizure (16.3%), infection (9.3%), pulmonary embolism (7.0%), intracranial hemorrhage (5.0%), cerebral edema (2.5%).
The registry lists serious adverse events for 8 of 80 participants at risk in the ICT-107 group and 7 of 43 participants at risk in the placebo group. It separately lists other adverse events for 59 of 80 participants at risk in the ICT-107 group and 40 of 43 participants at risk in the placebo group. These are reported event counts and denominators, not a complete account of event timing, duration, severity grades, or relationship to study treatment.
The record lists seizure at 16.3%, infection at 9.3%, pulmonary embolism at 7.0%, intracranial hemorrhage at 5.0%, cerebral edema at 2.5%, and increased intracranial pressure at 2.5% among the top serious terms. It does not specify in this summary how those term percentages were calculated or provide arm-specific counts for each listed term. The listed terms should not be assumed to be mutually exclusive; one participant may have experienced more than one event.
The registry does not report deaths within the adverse-event summary. The counts do not establish that an event was caused by a study intervention, and the record does not give the attribution criteria or detailed safety-analysis methods. Cross-arm comparisons of these summaries should therefore be cautious, particularly without detail on exposure time, event definitions, and follow-up.
12. What the Result Does, and Does Not, Mean
The posted results give a descriptive account of overall survival in a randomized phase IIb study: median survival was 18.3 months in the ICT-107 arm and 16.7 months in the control dendritic-cell arm. The confidence intervals indicate uncertainty around each arm's median. The registry record does not report a hazard ratio, a comparative confidence interval, or a primary p-value. Accordingly, the arm summaries alone do not establish the statistical significance of a difference between treatments.
These results also do not show that every participant in either arm had a survival time near the reported median. A median is a feature of a group distribution, and individual outcomes can vary. The available record does not include the full survival curves, event counts, or detailed follow-up information needed to describe that variation fully.
Randomization strengthens the basis for comparing outcomes by assigned group, and the primary population is described as intent to treat. However, the registry record does not provide enough detail about treatment adherence, incomplete follow-up, or the full statistical analysis to assess every potential source of uncertainty. Nor does it report a protocol-based sample-size justification or the planned approach to interim monitoring and multiplicity.
The safety summaries report serious and other adverse-event counts, along with several serious-event terms and percentages. They do not establish treatment causation, and they do not provide a complete safety profile or enough detail for a formal comparison of risk. Interpretation should distinguish the recorded events from claims about why they occurred.
Thus, the posted record supports a limited conclusion: it documents arm-specific survival medians and confidence intervals, as well as selected adverse-event summaries. It does not, on its own, establish whether ICT-107 changed overall survival, whether any apparent difference is statistically reliable, or how important that difference might be to patients. Those questions require comparative analyses and clinical context not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically notable as a randomized comparison of an individualized dendritic-cell immunotherapy with control dendritic cells in newly diagnosed glioblastoma multiforme. Randomization and a stated intent-to-treat primary population are important features because they define a comparison by assigned treatment and aim to preserve the value of random allocation. The trial also uses overall survival, a time-to-event endpoint that requires attention to event timing and incomplete follow-up.
The posted median estimates illustrate why arm-level summaries are not the same as a treatment-effect estimate. Each median has its own 95% confidence interval, but the registry record does not report a hazard ratio or other formal comparative estimate for overall survival. A reader can describe the two observed medians, but cannot infer statistical significance from the medians or their separate intervals alone.
The record also lists progression-free survival outcomes and selected adverse-event summaries. Considering multiple outcomes raises general questions about prespecification and multiplicity, while time-to-event results raise questions about censoring and analysis methods. The registry record does not provide the detailed statistical plan needed to answer those questions. This makes the distinction between posted results and unreported analysis details especially important.
More broadly, the trial demonstrates how a registry entry can communicate important design and outcome information while leaving gaps that matter for statistical interpretation. The randomized design, endpoint definition, group medians, confidence intervals, and safety counts are useful details. They do not replace a complete comparative analysis, detailed follow-up accounting, or transparent reporting of missing-data and multiplicity methods.
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 registry record does not report a hazard ratio, confidence interval for a comparative effect, or p-value for the primary overall-survival comparison. It reports arm-specific medians and confidence intervals, which are not sufficient to establish the significance or size of a between-group effect. It also does not provide survival curves, numbers of deaths, censoring counts, or a detailed account of follow-up.
The primary endpoint description specifies an intent-to-treat population of all randomized patients, but the record does not give details of missing outcome data, censoring procedures, or sensitivity analyses. The treatment received after assignment, adherence, and any deviations from the intended intervention are not described in the results. The record also does not state which individuals or groups were included in the triple masking.
The trial lists secondary progression-free survival outcomes, including one in HLA-A2 patients, but does not report their results in the registry record for this analysis. Although four outcome measures and two analyses are recorded, the complete outcome set, statistical hierarchy, and multiplicity strategy are not described. The protocol's sample-size justification, interim-analysis plan, stopping rules, and detailed statistical analysis plan are also not reported in the registry record.
The adverse-event summary gives counts for serious and other events and lists selected serious-event terms. It does not provide detailed definitions, event timing, exposure-adjusted summaries, treatment attribution, or arm-specific counts for each listed term. The record does not report deaths in this safety summary. These limitations constrain both the safety interpretation and comparison between groups.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study of ICT-107 Immunotherapy in Glioblastoma Multiforme (GBM) — NCT01280552.
- Registry record last updated 2017-03-20; status last verified 2017-02.
- Results first posted 2014-10-07.
- 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 a randomized, double-blind, controlled phase IIb study of ICT-107 versus control dendritic cells in newly diagnosed glioblastoma multiforme. The design is parallel, and the primary endpoint is overall survival from randomization until death or the last date known alive. The primary population is described as all randomized patients under an intent-to-treat approach.
Median overall survival was 18.3 months for ICT-107, with a 95% confidence interval from 14.9 to 21.2 months, and 16.7 months for Control Dendritic Cells, with a 95% confidence interval from 12.3 to 23.0 months. A primary comparative hazard ratio and p-value are not reported in the registry record. Safety summaries include serious and other adverse-event counts and selected serious-event terms. Details of the statistical plan, missing-data methods, and multiplicity handling are not reported.