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 MAGE-A4ᶜ¹º³²T for Multi-Tumor (NCT03132922), a non-randomized phase 1 study. The primary outcome is Adverse Events (AE) Including Serious Adverse Events (SAE).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03132922 |
| Phase | Phase 1 |
| Condition | Urinary Bladder Cancer, Melanoma, Head and Neck Cancer |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 71 |
| Arms in the primary outcome | n/a |
| Lead sponsor | USWM CT, LLC |
| Results posted | Yes |
2. Clinical Question
Population
Participants with HLA-A2 and MAGE-A4-positive tumors who received afamitresgene autoleucel or low dose radiation with afamitresgene autoleucel. Listed tumor types include urinary bladder cancer, melanoma, head and neck cancer, ovarian cancer, non-small cell lung cancer, esophageal cancer, gastric cancer, synovial sarcoma, myxoid round cell liposarcoma, and gastroesophageal junction tumors.
Intervention
Autologous genetically modified MAGE-A4ᶜ¹º³²T cells. A substudy also evaluates the cell therapy in combination with low dose radiation.
Comparator
There is no randomized control group. The radiation substudy is a combination-treatment group, not a like-for-like control comparison.
Primary question
Among participants who received study treatment, how many experienced adverse events, including serious adverse events, during the specified observation period?
The central question is about safety and tolerability, not a randomized estimate of benefit compared with another treatment. The primary outcome counts participants with adverse events, including serious adverse events. The registry definition includes untoward medical occurrences regardless of whether they were considered related to study intervention.
The study also lists antitumor activity and disease-course outcomes, including overall response rate, best overall response, time to response, duration of response, duration of stable disease, progression-free survival, and overall survival. However, the registry record lists no analyses for these outcomes. Their presence as planned outcomes does not itself establish that results were posted or that an effect was observed.
The population is defined by tumor antigen and HLA status as well as treatment received. The registry record does not report further details about eligibility criteria in the registry record for this analysis. The findings therefore concern the recorded treatment groups and cannot be assumed to describe people outside those groups.
3. Trial Design
This is a Phase 1, non-randomized, unmasked, single-group study of genetically engineered MAGE-A4ᶜ¹º³²T cells in people with MAGE-A4-positive tumors and HLA-A2. The registry lists enrollment of 71. The study began on 2017-05-15, and the primary completion date is 2022-12-27. Its status is active, not recruiting.
The intervention is autologous genetically modified MAGE-A4ᶜ¹º³²T cells, also identified in the registry as afamitresgene autoleucel. The brief summary describes taking a participant’s T cells and giving them a T cell receptor protein that recognizes and attacks tumors. A substudy examines the cell therapy with low dose radiation in up to 10 subjects. The record lists a radiation combination group, but does not provide a detailed account of the treatment sequence or allocation process.
The study includes participants with a range of tumor types: urinary bladder cancer, melanoma, head and neck cancer, ovarian cancer, non-small cell lung cancer, esophageal cancer, gastric cancer, synovial sarcoma, myxoid round cell liposarcoma, and gastroesophageal junction tumors. This multi-tumor design means the enrolled population is not described as one single cancer type. The registry record does not report subgroup-specific outcome estimates in the registry record for this analysis.
Because allocation is non-randomized and the model is single group, the design does not create a randomized comparison against a control treatment. The low dose radiation group receives a combination intervention and is not a control group that differs only by the presence or absence of the cell therapy. Differences among groups, if considered, would not by themselves establish a causal treatment effect.
The registry identifies adverse events, including serious adverse events, as the primary outcome. Its stated timeframe runs from the start of lymphodepleting chemotherapy until the end of the interventional phase, up to 3.2 years. Further details of dose escalation, dose selection, stopping decisions, and the statistical plan 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.
Before assignment: The main study could enroll up to 18 participants in the dose escalation phase (data cut off 01Sep2020). Following the dose escalation phase, the study was expanded to enroll up to approximately 30 participants at the selected dose range (inclusive of those treated during dose escalation at that dose range) across all the eligible tumor types to better characterize and assess overall safety. The sub study planned to enroll up to 10 participants (data cut off 13Jan2022).
Overall Study
| Stage | Afamitresgene Autoleucel, Group 1 | Afamitresgene Autoleucel, Group 2 | Afamitresgene Autoleucel, Group 3 + Expansion | Substudy: Low Dose Radiation (LDR) in Combination With Afamitresgene Autoleucel |
|---|---|---|---|---|
| STARTED | 3 | 3 | 32 | 4 |
| Received Afamitresgene Autoleucel | 3 | 3 | 32 | 4 |
| COMPLETED | 3 | 3 | 29 | 4 |
| NOT COMPLETED | 0 | 0 | 3 | 0 |
Reasons for not completing this period:
| Reason | Afamitresgene Autoleucel, Group 1 | Afamitresgene Autoleucel, Group 2 | Afamitresgene Autoleucel, Group 3 + Expansion | Substudy: Low Dose Radiation (LDR) in Combination With Afamitresgene Autoleucel |
|---|---|---|---|---|
| Ongoing in Interventional phase at data cut-off (1Sep2020) | 0 | 0 | 3 | 0 |
Analysis population for the primary outcome, as defined in the registry: Participants who received afamitresgene autoleucel or LDR/ afamitresgene autoleucel
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.
Population: Participants who received afamitresgene autoleucel or LDR/ afamitresgene autoleucel
| Characteristic | Unit | Afamitresgene Autoleucel, Group 1 | Afamitresgene Autoleucel, Group 2 | Afamitresgene Autoleucel, Group 3 + Expansion | Substudy: Low Dose Radiation (LDR) in Combination With Afamitresgene Autoleucel | Total |
|---|---|---|---|---|---|---|
| Participants analysed | 3 | 3 | 32 | 4 | 42 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 | 3 | 24 | 3 | 31 |
| Age, Categorical: >=65 years | Participants | 2 | 0 | 8 | 1 | 11 |
| Sex: Female, Male: Female | Participants | 3 | 3 | 10 | 3 | 19 |
| Sex: Female, Male: Male | Participants | 0 | 0 | 22 | 1 | 23 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 1 | 1 | 0 | 2 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 3 | 2 | 31 | 4 | 40 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 3 | 0 | 3 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 | 1 | 1 |
| Race (NIH/OMB): White | Participants | 3 | 3 | 29 | 3 | 38 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Adverse Events (AE) Including Serious Adverse Events (SAE), measured over From the start of lymphodepleting chemotherapy until end of interventional phase (up to 3.2 years)..
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants with adverse events, including serious adverse events. The registry defines an adverse event as an untoward medical occurrence in a clinical study participant who received a pharmaceutical product, regardless of causality. This includes an unfavorable or unintended sign, an abnormal laboratory finding, a symptom, or a new or worsened disease that occurs in temporal association with study intervention, whether or not it is considered related to that intervention.
The endpoint is reported as a count of participants rather than as a measure of how severe an event was, how long it lasted, or how many separate events an individual experienced. A participant-level count can show how many people in a recorded group had an event, but it does not convey the full event burden. A person with one event and a person with several events may each contribute once to a count of participants with an event.
The recorded population is participants who received afamitresgene autoleucel or low dose radiation with afamitresgene autoleucel. The observation period is from the start of lymphodepleting chemotherapy until the end of the interventional phase, up to 3.2 years. A defined observation period matters because adverse-event counts depend on how long participants are followed and when events are captured.
Safety outcomes are used to describe the types and frequency of unfavorable medical occurrences during a study. They do not, on their own, show whether an event was caused by treatment. The registry definition explicitly includes events regardless of causality, and the counts should be read in that context.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry reports safety counts across four treatment groups. In Afamitresgene Autoleucel, Group 1, 3 participants were at risk for serious adverse events and 2 were affected; 3 were at risk for other adverse events and 3 were affected, and the death count is 2. In Group 2, the corresponding figures are 3 at risk and 2 affected for serious adverse events, 3 at risk and 3 affected for other adverse events, and 1 death. In Group 3 + Expansion, 32 participants were at risk and 23 were affected for serious adverse events; 32 were at risk and 32 were affected for other adverse events, and the death count is 15. In the low dose radiation combination group, 4 participants were at risk and 3 were affected for serious adverse events; 4 were at risk and 4 were affected for other adverse events, and the death count is 2.
These are descriptive counts. They identify how many participants in each recorded group were affected according to the registry categories, but they do not establish that the intervention caused an event or death. The registry’s adverse-event definition includes events whether or not they were considered related to study intervention. The record does not provide causes of death in the registry record for this analysis, nor does it specify whether each death was considered treatment-related.
The group sizes differ, and the groups do not constitute randomized, like-for-like comparisons. In particular, the low dose radiation group receives a combination intervention. It is not appropriate to read differences among the group counts as evidence that one regimen is safer or less safe than another. The registry record does not report an adjusted comparison, uncertainty interval, or statistical test for these counts.
The serious-event terms listed by the registry are cytokine release syndrome, sepsis, pancytopenia, atrial fibrillation, encephalopathy, and syncope. Each is listed at 33.3%. The denominator and the precise interpretation of these percentages are not reported in the registry record. They should therefore be treated as registry-listed percentages, not as a basis for inferring the number of affected participants or comparing risks across groups.
The registry lists several antitumor and survival outcomes, but the record reports 0 analyses. No estimates for response, time to response, duration of response, progression-free survival, or overall survival are available in the registry record for this analysis. Consequently, the posted safety counts cannot answer whether the treatment changed tumor outcomes. The registry record also does not describe missing-data handling, confidence intervals, or the method used to analyze the safety counts.
Overall, the posted information provides a descriptive account of adverse events and deaths in the listed groups. Precision is limited by the group counts and by the absence of uncertainty estimates and fuller event detail. These limitations make the counts useful for describing what the registry records, but not for drawing comparative or causal conclusions.
9. Statistical Methods Explained
The outcomes are counts of participants with events. The explanations below distinguish what the registry reports from general statistical concepts that help interpret such counts. The registry record does not report the full statistical analysis plan.
What does a participant count describe?
A participant-level event count records how many people in a group experienced an event during the specified observation period. It differs from an event count: one participant can experience multiple events, while a participant count records that person as affected. The primary outcome is explicitly described as the number of participants with adverse events, including serious adverse events. The registry also supplies at-risk and affected counts for the groups. These figures describe the recorded groups; they do not explain the severity, duration, recurrence, or clinical course of every event.
In general, a proportion can be calculated by relating the number of affected participants to the number at risk, but no new proportions are calculated in this interpretation. Reporting a numerator and denominator together helps readers understand the group size behind a count. It still does not reveal how precisely the underlying risk has been estimated. An uncertainty interval would help describe that precision, but no such interval is reported in the registry record.
Why does the design matter?
Random allocation is one way to make treatment groups more comparable on average, including with respect to characteristics that may affect outcomes. This study is non-randomized and single group, and the radiation group receives a combination intervention. Therefore, group differences may reflect differences in participants, treatment context, follow-up, or other factors rather than a treatment effect. The registry record does not report a randomized control comparison. The recorded counts should be interpreted descriptively rather than as estimates from a controlled efficacy comparison.
Masking is listed as none. In general, masking can reduce the possibility that knowledge of treatment assignment influences some assessments or reporting. Its absence does not establish that a particular bias occurred, but it is relevant when considering subjective outcomes and event assessment. The registry record does not provide additional details on event adjudication or assessment procedures.
How should uncertainty be understood?
In general, a confidence interval gives a range of values compatible with the data under a specified statistical method and assumptions. It helps distinguish a relatively precise estimate from one that remains uncertain. A count alone does not provide that information. The registry record does not report confidence intervals for the adverse-event figures in the registry record for this analysis, so their statistical precision cannot be assessed from those counts alone.
Safety outcomes can also involve different event types, severity levels, and exposure times. A participant-level count simplifies those details. It may be useful for describing whether participants experienced events, but it does not capture how many events occurred in total or how their timing varied. The specified timeframe, from the start of lymphodepleting chemotherapy until the end of the interventional phase, up to 3.2 years, is therefore part of the meaning of the endpoint. The registry record does not report person-time rates or the timing of each event.
Over-dispersion is a general statistical concern when event counts vary more than a model assumes. It is most directly relevant to analyses of event counts, rather than to a simple participant count. In general, ignoring over-dispersion can make uncertainty estimates too narrow. The record does not report a count model or an assessment of over-dispersion, and the participant-level outcome should not be mistaken for an analysis of repeated event counts.
What does the sample-size illustration mean?
The planning illustrations are based on the observed effect and assume a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. For non-time-to-event outcomes, the illustrations are expressed as participants per arm; time-to-event illustrations are expressed as total events. Such calculations describe sample sizes under stated assumptions for detecting an effect of a specified fraction of the observed effect. They are planning illustrations, not the study’s own design justification and not observed power.
Those assumptions do not turn this non-randomized, single-group study into a two-arm trial, nor do they supply a comparator that the design lacks. The registry record does not report the study’s sample-size justification, a planned effect size, or a detailed analysis plan. No numerical planning estimates are because the registry entries available for this analysis contain no such estimates.
What can the registry analyses establish?
The registry lists 0 analyses. That means the registry entries available for this interpretation do not include statistical analyses or estimates for the listed outcomes. It does not justify filling in missing results or inferring that a result is zero. General statistical methods can clarify what an estimate or interval would mean, but they cannot replace outcome results that are not reported in the registry record. The same caution applies to the listed secondary outcomes: outcome names describe what is assessed, not what the study found.
10. Exact Estimates for Each Arm
No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.
| Arm | Participants | Events | Observed % | Clopper-Pearson 95% CI | Wilson 95% CI | Bayesian 95% credible interval |
|---|---|---|---|---|---|---|
| Afamitresgene Autoleucel, Group 1 | 3 | 3 | 100.0% | 29.2% to 100.0% | 43.9% to 100.0% | 39.8% to 99.4% |
| Afamitresgene Autoleucel, Group 2 | 3 | 3 | 100.0% | 29.2% to 100.0% | 43.9% to 100.0% | 39.8% to 99.4% |
| Afamitresgene Autoleucel, Group 3 + Expansion | 32 | 32 | 100.0% | 89.1% to 100.0% | 89.3% to 100.0% | 89.4% to 99.9% |
| Low Dose Radiation (LDR) in Combination With Afami | 4 | 4 | 100.0% | 39.8% to 100.0% | 51.0% to 100.0% | 47.8% to 99.5% |
R code and output
arm <- c("Afamitresgene Autoleucel, Group 1", "Afamitresgene Autoleucel, Group 2", "Afamitresgene Autoleucel, Group 3 + Expansion", "Low Dose Radiation (LDR) in Combination With Afami")
n <- c(3, 3, 32, 4)
events <- c(3, 3, 32, 4)
for (i in seq_along(n)) {
ci <- binom.test(events[i], n[i])$conf.int # Clopper-Pearson exact interval
cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i]) # Bayesian, uniform Beta(1,1) prior
wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}
ARM Afamitresgene Autoleucel, Group 1 3 3 100.0 29.2 100.0 43.9 100.0 39.8 99.4
ARM Afamitresgene Autoleucel, Group 2 3 3 100.0 29.2 100.0 43.9 100.0 39.8 99.4
ARM Afamitresgene Autoleucel, Group 3 + Expansion 32 32 100.0 89.1 100.0 89.3 100.0 89.4 99.9
ARM Low Dose Radiation (LDR) in Combination With Afami 4 4 100.0 39.8 100.0 51.0 100.0 47.8 99.511. Multiplicity, Missing Data and Analysis Populations
The registry lists secondary outcomes for overall response rate, best overall response, time to response, duration of response, duration of stable disease, progression-free survival, and overall survival. It also lists 10 outcome measures and 0 analyses. The registry entries available for this analysis do not include estimates or tests for those outcomes.
When a study evaluates multiple outcomes, there is a general multiplicity concern: conducting several statistical tests can increase the chance of at least one apparently notable result arising by chance. Studies may address this with a prespecified hierarchy, adjustment procedure, or other strategy. The multiplicity strategy for this study is not reported in the registry record. Because no analyses are listed, there are no posted test results in the registry record to interpret for multiplicity.
The registry identifies the primary safety population as participants who received afamitresgene autoleucel or low dose radiation with afamitresgene autoleucel. Beyond this description, the analysis-population definitions are not reported in the registry record. The registry entries do not specify how participants who started but did not complete treatment, or who had incomplete follow-up, were handled.
Missing-data methods and the extent of missing outcome data are not reported in the registry record. The record also does not state how participants with differing follow-up time were handled in the safety summaries. These omissions matter because an observed count depends on outcome capture and the period during which participants could be observed. Without those details, the counts can be described as posted, but the completeness of follow-up cannot be assessed.
12. 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 |
|---|---|---|---|
| Afamitresgene Autoleucel, Group 1 | 2 | 3 | 66.7% |
| Afamitresgene Autoleucel, Group 2 | 2 | 3 | 66.7% |
| Afamitresgene Autoleucel, Group 3 + Expansion | 23 | 32 | 71.9% |
| Low Dose Radiation (LDR) in Combination With Afamitresgene Autoleucel | 3 | 4 | 75.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Afamitresgene Autoleucel, Group 1 | 2 of 3 |
| Afamitresgene Autoleucel, Group 2 | 1 of 3 |
| Afamitresgene Autoleucel, Group 3 + Expansion | 15 of 32 |
| Low Dose Radiation (LDR) in Combination With Afamitresgene Autoleucel | 2 of 4 |
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 |
|---|---|
| Afamitresgene Autoleucel, Group 1 | 3 of 3 |
| Afamitresgene Autoleucel, Group 2 | 3 of 3 |
| Afamitresgene Autoleucel, Group 3 + Expansion | 32 of 32 |
| Low Dose Radiation (LDR) in Combination With Afamitresgene Autoleucel | 4 of 4 |
Most frequent serious adverse event terms
| Event term | Organ system | Afamitresgene Autoleucel, Group 1 | Afamitresgene Autoleucel, Group 2 | Afamitresgene Autoleucel, Group 3 + Expansion | Low Dose Radiation (LDR) in Combination With Afamitresgene Autoleucel |
|---|---|---|---|---|---|
| Cytokine release syndrome | Immune system disorders | 1 of 3 | 0 of 3 | 8 of 32 | 0 of 4 |
| Pyrexia | General disorders | 0 of 3 | 0 of 3 | 5 of 32 | 0 of 4 |
| Pancytopenia | Blood and lymphatic system disorders | 0 of 3 | 1 of 3 | 3 of 32 | 0 of 4 |
| Pneumonia | Infections and infestations | 0 of 3 | 0 of 3 | 2 of 32 | 0 of 4 |
| Sepsis | Infections and infestations | 0 of 3 | 1 of 3 | 1 of 32 | 0 of 4 |
| Atrial fibrillation | Cardiac disorders | 1 of 3 | 0 of 3 | 1 of 32 | 0 of 4 |
| Platelet count decreased | Investigations | 0 of 3 | 0 of 3 | 2 of 32 | 0 of 4 |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | 0 of 3 | 1 of 3 | 1 of 32 | 0 of 4 |
| Anal abscess | Infections and infestations | 0 of 3 | 0 of 3 | 1 of 32 | 0 of 4 |
| Cellulitis | Infections and infestations | 0 of 3 | 0 of 3 | 1 of 32 | 0 of 4 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Afamitresgene Autoleucel, Group 1 | Afamitresgene Autoleucel, Group 2 | Afamitresgene Autoleucel, Group 3 + Expansion | Low Dose Radiation (LDR) in Combination With Afamitresgene Autoleucel |
|---|---|---|---|---|---|
| Lymphocyte count decreased | Investigations | 3 of 3 | 3 of 3 | 31 of 32 | 4 of 4 |
| White blood cell count decreased | Investigations | 3 of 3 | 3 of 3 | 28 of 32 | 4 of 4 |
| Neutrophil count decreased | Investigations | 3 of 3 | 3 of 3 | 27 of 32 | 4 of 4 |
| Anaemia | Blood and lymphatic system disorders | 2 of 3 | 2 of 3 | 23 of 32 | 3 of 4 |
| Nausea | Gastrointestinal disorders | 3 of 3 | 2 of 3 | 19 of 32 | 2 of 4 |
| Fatigue | General disorders | 3 of 3 | 2 of 3 | 19 of 32 | 2 of 4 |
| Pyrexia | General disorders | 1 of 3 | 3 of 3 | 18 of 32 | 3 of 4 |
| Cytokine release syndrome | Immune system disorders | 1 of 3 | 0 of 3 | 20 of 32 | 3 of 4 |
| Platelet count decreased | Investigations | 2 of 3 | 1 of 3 | 18 of 32 | 0 of 4 |
| Vomiting | Gastrointestinal disorders | 2 of 3 | 2 of 3 | 15 of 32 | 1 of 4 |
Serious events with the highest arm-level rate: cytokine release syndrome (33.3%), sepsis (33.3%), pancytopenia (33.3%), atrial fibrillation (33.3%), encephalopathy (33.3%).
The serious adverse-event figures are participant counts, with an at-risk count and an affected count for each treatment group. For example, the registry records 23 affected participants among 32 at risk in Afamitresgene Autoleucel, Group 3 + Expansion. The other groups and the low dose radiation combination group have their own recorded counts. These figures describe the registry categories and do not indicate how many separate serious events each participant experienced.
The registry separately records counts for other adverse events and deaths. The death counts are not identified in the registry record for this analysis as treatment-related or unrelated. Likewise, adverse events are counted regardless of whether they were considered related to study intervention. The counts should not be interpreted as causal effects.
The serious-event terms listed are cytokine release syndrome, sepsis, pancytopenia, atrial fibrillation, encephalopathy, and syncope, each with a registry-listed percentage of 33.3%. The denominator for these percentages is not reported in the registry record. They therefore cannot be used to infer the number of cases, the proportion of all participants affected, or differences among treatment groups.
Group sizes are not equal, and the study is not randomized. These figures are descriptive safety information, not a comparative test of the regimens.
13. What the Result Does, and Does Not, Mean
The posted information establishes that the registry records adverse-event counts, other adverse-event counts, and deaths in four treatment groups. It also identifies several serious adverse-event terms. This is useful descriptive information about what was recorded during the study’s stated observation period. The endpoint definition includes events regardless of whether they were considered related to study intervention, so the counts should not be read as events proven to have been caused by treatment.
The figures do not establish a comparative safety effect. The study is non-randomized, the design is listed as single group, and the radiation group receives a combination intervention. There is no randomized control group against which to estimate what would have happened under another treatment. Differences in group counts cannot be attributed to treatment without a suitable comparison and appropriate analysis.
The figures also do not provide a complete account of safety burden. Participant counts do not show the number of events per participant, event duration, timing, severity details beyond the serious-event category, or whether an event led to treatment changes. The registry record does not report causes of death or whether deaths were considered related to the intervention in the registry record for this analysis.
Nor do the safety counts answer the antitumor questions listed as secondary outcomes. The record lists response and survival outcomes but reports 0 analyses. No conclusion about response, disease control, or survival can be drawn from outcome names alone. The confidence intervals, missing-data approach, and detailed statistical analysis plan are not reported in the registry record.
Accordingly, the most defensible reading is narrow: the registry posts descriptive event counts for the recorded treatment groups and lists serious-event terms. Those data are not a randomized estimate of benefit or harm, and they do not establish causation. They provide neither a complete account of event severity and timing nor posted statistical estimates for the listed efficacy outcomes.
14. Why This Trial Matters Statistically
This study is statistically distinctive because it examines a genetically modified autologous cell therapy across multiple tumor types and includes a low dose radiation combination substudy. The eligibility context involves HLA-A2 and MAGE-A4-positive tumors. Such a multi-tumor setting makes it important to understand exactly which participants and treatment groups a reported count represents; a single pooled figure may not describe each tumor type equally.
The Phase 1, non-randomized, unmasked, single-group design is central to interpreting the record. It is suited to describing experience in the recorded treatment groups, but it does not provide a randomized control comparison. Safety counts can be informative in their own right, yet without a control group they cannot isolate the contribution of the study intervention from other explanations for events.
The primary outcome’s participant-count format is also important. It communicates how many people were affected, while leaving out event frequency per person, timing, and duration. The registry’s separate serious-event terms add descriptive detail, but their listed percentages have no reported denominator in the record. Statistical interpretation depends not only on the displayed value, but also on its population, definition, follow-up period, and uncertainty.
Finally, this record illustrates the distinction between listed outcomes and analyzed results. Response and survival outcomes appear among the secondary outcomes, but the registry lists 0 analyses. The presence of an endpoint does not supply an estimate, and a planning illustration is not observed power or evidence that a treatment effect was demonstrated. Keeping those distinctions clear prevents descriptive safety data from being overread as comparative evidence.
15. 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 is the absence of a randomized comparator. The non-randomized, single-group design cannot establish how the recorded safety counts would compare with outcomes under another treatment. The low dose radiation group is a combination-treatment group, not a like-for-like control. Differences among groups may reflect factors other than intervention.
The registry record gives participant counts but, in the registry record for this analysis, does not report confidence intervals, statistical tests, adjusted comparisons, or a detailed analysis plan. It lists 0 analyses. Thus, the precision of the counts and any inferential conclusions cannot be assessed from the registry entries.
Safety detail is limited. The primary endpoint counts participants with adverse events, including serious adverse events, but does not describe the number of individual events, their timing, duration, or full severity profile. Death counts are recorded without causes or attribution details in the registry record for this analysis. The listed serious-event percentages have no reported denominator, limiting their interpretation.
The registry describes the primary population as participants who received the cell therapy or the low dose radiation combination, but further analysis-population definitions are not reported. Missing-data handling and the extent of missing follow-up are also not reported in the registry record. These details matter when interpreting event counts across different follow-up periods.
The study includes several tumor types, while tumor-specific outcome estimates are not reported in the registry record for this analysis. The secondary outcomes are listed, but no analyses are posted. The results summarized by the registry therefore do not support conclusions about antitumor response or survival.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: MAGE-A4ᶜ¹º³²T for Multi-Tumor — NCT03132922.
- Registry record last updated 2026-06-04; status last verified 2026-05.
- Results first posted 2026-05-07.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
19. Record Summary
NCT03132922 is a Phase 1, non-randomized, unmasked, single-group study of autologous genetically modified MAGE-A4ᶜ¹º³²T cells in participants with HLA-A2 and MAGE-A4-positive tumors. A substudy evaluates the cell therapy with low dose radiation. The registry lists enrollment of 71 and an active, not recruiting status.
The primary outcome counts participants with adverse events, including serious adverse events, from the start of lymphodepleting chemotherapy until the end of the interventional phase, up to 3.2 years. The registry posts group counts for serious adverse events, other adverse events, and deaths, along with a list of serious-event terms. The event definition includes occurrences regardless of causality.
The registry lists several antitumor and survival outcomes but reports 0 analyses. A randomized comparison, detailed statistical plan, missing-data methods, and uncertainty estimates are not reported in the registry record. The posted safety counts are descriptive and do not establish causation or comparative treatment effects.