Adoptive Cell Therapy Across Cancer Diagnoses: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Adoptive Cell Therapy Across Cancer Diagnoses (NCT03296137), a na phase 1, phase 2 study. The primary outcome is Number of Participants and Type of Reported Adverse Events.

2. Clinical Question

The central statistical question is descriptive: what adverse events were recorded among patients treated in this single-group study? Without a comparator, the record cannot estimate how event frequency differs from what would have occurred under another treatment or no study treatment. It also does not establish whether a reported event was caused by a particular component of the treatment. The scope is further limited by the registry’s lack of detailed eligibility criteria, diagnosis-specific counts, and outcome analyses. The enrollment listed in the record is 25; the record does not clarify recruitment or follow-up for each outcome.

3. Trial Design

This is a phase 1 and phase 2, single-group study titled “Adoptive Cell Therapy Across Cancer Diagnoses.” The registry lists the study status as completed, enrollment as 25, and the condition as cancer. The start date is 2017-10-13, and the primary completion date is 2020-03-13. The sponsor is listed as Inge Marie Svane.

The intervention is described as TIL-based adoptive T-cell therapy combined with checkpoint inhibition. The registry lists autologous TILs, ipilimumab, nivolumab, proleukin, cyclophosphamide, and Fludara. The arm description refers to TIL therapy with checkpoint inhibitors, but the record does not give treatment schedules, doses, rules for selecting agents, or information about whether every participant received each listed intervention.

There is no randomized allocation, masking, or separate control arm recorded. This is therefore a descriptive treatment group rather than a randomized comparison. The registry does not report a protocol for allocation, a sample-size justification, or details of how participants were selected across cancer diagnoses. The number and type of outcome measures are recorded as four measures, with no analyses listed. The primary outcome concerns the number of participants and types of reported adverse events, with a timeframe of up to 2,5 years from the beginning of the study. Time to disease progression, overall survival, and overall response rate are listed as secondary outcomes. Their definitions, analysis methods, and results are not reported in the registry record.

The single-group structure matters for interpretation. Adverse events can be described in the treated group, but no between-group treatment effect can be estimated from this design. Changes in disease status or survival, if reported, would also need to be interpreted without a concurrent control group. The record does not provide additional design details that would resolve these limitations.

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

Analysis population for the primary outcome, as defined in the registry: Patients who received TIL therapy

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: Patients who received TIL therapy

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants and Type of Reported Adverse Events, measured over Up to 2,5 years from begin of study.

The primary outcome is titled “Number of Participants and Type of Reported Adverse Events.” It is measured in participants, and its description specifies reporting grade >2 adverse events according to CTCAE v. 4.0 among patients who received TIL therapy. The listed timeframe is up to 2,5 years from the beginning of the study. The endpoint is intended to characterize safety by recording how many participants experienced events and what types of events were reported.

Participant counts are useful for describing the burden of reported events in a treated group. Event types also help distinguish the kinds of experiences recorded rather than reducing safety reporting to a single overall count. However, a count does not by itself describe event duration, severity beyond the stated grading threshold, timing, recurrence, or relationship to a particular treatment component. Those details are not reported in the registry record.

The endpoint’s grade threshold should not be treated as interchangeable with the separate serious-adverse-event summary. “Serious” and a CTCAE grade threshold are different ways of classifying events; the record does not state that the categories are identical. Nor does the primary outcome title alone establish that every event listed in the safety summary meets the primary endpoint definition. The posted record gives participant counts for serious and other adverse events, but does not report a complete grade-specific result for the primary endpoint.

Safety endpoints are used to describe events observed during treatment and follow-up. In a single-group study, such descriptions can inform what was recorded among treated participants, but they cannot isolate the effects of treatment from other possible causes.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a participant count describe?

A participant count reports how many people in a defined group were recorded as experiencing an outcome. For safety reporting, counts can be presented for participants with at least one event, as well as for particular event types. These are not necessarily the same as counts of all event episodes: a participant can experience more than one event, and the registry may report people rather than episodes. The primary outcome is explicitly expressed in participants, but the posted record does not provide an event-level dataset or a detailed counting rule.

A count is most interpretable when its denominator is clear. The serious and other adverse-event summaries each list 25 participants at risk; the respective affected counts are 9 and 25. These summaries do not tell the reader whether the same people appear in both categories, how exposure time was handled, or whether follow-up was complete for everyone. The enrollment listing is 25, but enrollment and the number contributing complete outcome data are not necessarily interchangeable. The record does not explain this distinction for each outcome.

Why are event percentages not treatment effects?

A percentage for an event term describes the frequency reported under the registry’s counting convention. It does not measure a difference between treatments. For example, the listed percentages for serious event terms are descriptive values for this study record. Without a comparator group and a clearly specified denominator, they cannot establish whether a particular event is more or less common than under another treatment.

In general, comparisons require groups that can support a meaningful contrast and methods that account for the design. This study is recorded as single-group, with allocation listed as not applicable. There is no randomized control arm to provide a contemporaneous reference. Even where an event count is complete, potential differences in diagnosis, prior treatment, disease status, exposure, and follow-up can affect observed frequencies. The registry does not report analyses addressing these factors.

What would a confidence interval add?

In general, a confidence interval expresses the statistical uncertainty around an estimate under a stated model and sampling process. A relatively wide interval indicates less precision, while a narrower interval indicates greater precision under those assumptions. It does not resolve bias or create a comparison that the study design lacks. The registry record does not report confidence intervals for the adverse-event counts or percentages, so the precision of these values cannot be evaluated from the posted results.

Likewise, a hypothesis test or p-value would address a specified statistical question, not the broader question of whether an intervention caused an event. The record lists no analyses and reports no hypothesis tests. No inferential result should be inferred from the event counts alone.

How should the planning assumptions be read?

General sample-size planning asks how much information a future study would need to detect a defined effect under stated assumptions. The planning description associated with the registry refers to a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It also describes planning for stated fractions of an observed effect. Such calculations are illustrations based on assumptions; they are not observed power and do not establish that this trial was designed or powered in that way.

The planning description is not the same as the recorded trial design. The study itself is single-group, while the planning assumptions describe two-arm calculations. The registry does not report a numerical sample-size justification for the single-group study, an effect estimate used for planning, or the resulting planning values. Thus, the assumptions cannot be treated as evidence about the trial’s original design rationale or its ability to answer a comparative question.

In general, over-dispersion occurs when variability in count data exceeds what a simple model assumes. If over-dispersion is present and ignored, uncertainty can be understated. This concept is relevant to some event-count analyses, but the registry does not report a count model, a dispersion estimate, or analyses of event counts that would allow its importance to be assessed for this study.

What do the outcome labels establish?

The primary outcome description specifies grade >2 adverse events according to CTCAE v. 4.0, and the adverse-event summary separately organizes information into serious and other events. A grading system and a seriousness classification answer related but distinct questions. A grade describes severity under a grading framework; seriousness is a separate event classification. The posted record does not provide enough detail to map each serious event term to its grade or to determine exactly how the primary endpoint was tabulated.

The registry lists time to disease progression, overall survival, and overall response rate as secondary outcomes. In general, time-to-event outcomes require information about event times and censoring, while response-rate outcomes require a response definition and assessment rules. Those details and outcome results are not reported in the registry record. No analysis should be inferred from the outcome names alone.

Overall, the reported numbers support a descriptive account of selected adverse-event summaries. They do not supply the uncertainty estimates, participant-level detail, outcome definitions, or comparative analyses needed for more elaborate statistical interpretation.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary adverse-event outcome and three secondary outcomes: time to disease progression, overall survival, and overall response rate. The record gives the number of outcome measures as 4, but lists no analyses. A plan for controlling multiplicity across outcomes, event types, or time points is not reported in the registry record. Multiplicity matters in general because examining many outcomes or comparisons can increase the chance of apparently notable findings arising by chance. Without an analysis plan, it is not possible to tell whether any adjustment was intended or how outcomes were prioritized in practice.

No hypothesis tests or inferential analyses are posted, so there are no reported multiplicity-adjusted or unadjusted results to interpret. The absence of analysis results also means there is no basis to determine whether secondary outcomes were assessed, whether any planned outcomes were omitted, or whether results were selected for reporting.

Missing-data procedures are not reported in the registry record. The record does not describe how incomplete follow-up, unavailable outcome assessments, withdrawals, or missing adverse-event information were handled. It also does not give outcome-specific denominators or a flow of participants through follow-up. Consequently, the listed enrollment and adverse-event at-risk counts should not be taken to establish complete observation for every endpoint.

The primary outcome population is described as patients who received TIL therapy. The registry does not report a broader analysis population, an intention-to-treat approach, exclusions from analysis, or rules for handling participants who did not receive or complete treatment. These omissions limit assessment of selection and attrition. Without those details, the reported counts can be read as registry summaries, but the completeness and representativeness of each analysis population remain unclear.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 4% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: colitis (8.0%), elevated bilirubin (4.0%), urinary tract infection (4.0%), refractory trombocytopenia (4.0%), chylos (4.0%).

The safety summary lists 25 participants at risk for serious adverse events, with 9 affected; for other adverse events, it lists 25 at risk and 25 affected. It also records 23 deaths. These are registry figures for the treated group. The record does not report the timing or causes of deaths, whether deaths were considered treatment-related, or how deaths relate to the adverse-event categories. No causal interpretation should be attached to the death count from the information posted.

Colitis is listed among the serious adverse-event terms at 8.0%. Elevated bilirubin, urinary tract infection, refractory trombocytopenia, chylos, and neutropenic fever are each listed at 4.0%. The registry does not specify the denominator or counting rule for each term, and the percentages should not be treated as mutually exclusive categories. A participant may potentially contribute to more than one event type, but participant-level overlap is not reported.

Serious events and grade >2 events are not interchangeable classifications. The primary outcome description specifies the latter, while the summary reports serious and other events. The record does not provide the detail needed to reconcile those summaries or determine whether all listed terms meet the primary outcome definition. The figures describe reported events; without a comparison group, follow-up detail, and causal assessment, they do not establish comparative safety or attribution to any specific intervention.

12. What the Result Does, and Does Not, Mean

The posted results support a limited descriptive statement: the registry records adverse-event counts and selected serious-event terms for participants in a single treated group. The serious-event summary lists 9 affected participants among 25 at risk, and the other-event summary lists 25 affected among 25 at risk. It also records 23 deaths. Selected serious-event terms and their reported percentages add detail about the kinds of events listed. The record does not report uncertainty estimates for these figures.

These observations do not establish that the treatment caused any particular event, nor do they show how the event burden compares with another treatment or with no treatment. A single-group study has no concurrent control group against which to estimate a treatment contrast. In addition, the registry does not provide event timing, attribution, diagnosis-specific results, or the completeness of follow-up. These missing details constrain interpretation even of the descriptive counts.

The primary endpoint is framed around grade >2 adverse events according to CTCAE v. 4.0, while the posted safety summary separately identifies serious and other events. The relationship between these classifications is not given. The registry also does not post results for time to disease progression, overall survival, or overall response rate. Their listing as secondary outcomes is not evidence of a particular finding.

The record therefore cannot establish comparative efficacy, comparative safety, or a treatment effect across cancer diagnoses. Nor does it support generalization to every cancer diagnosis, because the diagnosis mix and diagnosis-specific outcomes are not reported. The enrollment entry is 25, but that alone does not show how many participants had evaluable data for each endpoint or how long each person was followed.

The most defensible interpretation is that the registry contains a safety-oriented descriptive summary, with important details and analyses absent. It can identify what the record reports, but not resolve causation, precision, comparative benefit or harm, or the consistency of outcomes across diagnoses. Those questions would require additional study details and results that are not reported in the registry record.

13. Why This Trial Matters Statistically

This study is statistically notable for the gap between its broad clinical scope and the narrow conclusions supported by its recorded design and results. It is described as spanning cancer diagnoses and combines TIL-based adoptive cell therapy with checkpoint inhibition, but the registry records a single group and does not provide diagnosis-specific outcome summaries. Statistical interpretation must therefore distinguish a broad treatment description from evidence that can be compared or generalized across diagnoses.

The record also illustrates why endpoint definitions matter. The primary outcome description refers to grade >2 adverse events under CTCAE v. 4.0, while the safety summary reports serious and other adverse events. Those labels capture different classifications, and the registry does not explain their relationship. Without a clear connection between the endpoint definition and the summary counts, readers cannot assume the figures fully represent the primary outcome as defined.

Another important issue is the difference between a descriptive safety summary and an inferential analysis. Counts and event terms can document observations in a treated group, but without a comparator they cannot estimate relative risks or treatment effects. The record does not provide confidence intervals or analyses that would quantify statistical precision. Its listed secondary outcomes likewise have no posted results.

The planning description refers to assumptions for two-arm calculations, whereas the recorded study design is single-group. This contrast underscores that planning illustrations should not be confused with the actual design or evidence of the trial’s statistical rationale. The registry does not report a sample-size justification for the study itself. The record is therefore most useful as a reminder that design, endpoint definition, analysis population, and completeness of reporting all shape what safety data can establish.

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 principal trial-specific limitation is the single-group design. With no randomized comparison group, reported event counts cannot establish how outcomes differ under an alternative treatment, and observed events cannot be attributed to a particular intervention from the registry information alone. Allocation is listed as not applicable and masking as none.

The study spans cancer diagnoses, but the registry does not report the diagnosis mix, diagnosis-specific enrollment, or outcomes by diagnosis. Treatment details are also limited: several interventions are listed, but schedules, doses, participant-level exposure, and the use of each agent are not described. These omissions make it difficult to interpret variation in outcomes or relate an event to a particular component.

The primary outcome description specifies grade >2 adverse events according to CTCAE v. 4.0. However, the safety summary separates serious and other events, and the registry does not explain how those summaries map to the primary endpoint. Event timing, duration, recurrence, attribution, and participant-level overlap are not reported. The meaning and denominator of each listed serious-event percentage are also not detailed.

Although the registry lists an enrollment of 25 and an adverse-event at-risk count of 25 for each reported category, it does not provide outcome-specific follow-up completeness or missing-data procedures. The listed deaths are not accompanied by timing or cause. Results for the secondary outcomes are absent, as are analyses, confidence intervals, and hypothesis tests. Multiplicity handling, the analysis population beyond patients who received TIL therapy, and the sample-size justification are not reported in the registry record.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Adoptive Cell Therapy Across Cancer Diagnoses — NCT03296137.
• Registry record last updated 2024-10-26; status last verified 2024-10.
• Results first posted 2024-10-26.
• 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.

18. Record Summary

The registry record for NCT03296137 describes “Adoptive Cell Therapy Across Cancer Diagnoses,” a completed phase 1 and phase 2, single-group study. Enrollment is listed as 25. The recorded start date is 2017-10-13, and the primary completion date is 2020-03-13. The intervention description concerns TIL-based adoptive T-cell therapy with checkpoint inhibition and lists autologous TILs, ipilimumab, nivolumab, proleukin, cyclophosphamide, and Fludara.

The primary outcome concerns the number of participants and types of reported adverse events, with a description specifying grade >2 events according to CTCAE v. 4.0. The safety summary records serious and other adverse-event counts and selected serious-event terms. Time to disease progression, overall survival, and overall response rate are listed as secondary outcomes, but results for these outcomes are not reported in the registry record. No analyses are listed. The record does not report a comparator, analysis plan, missing-data procedures, or sample-size justification for the study.