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 Adoptive Cell Transfer of Autologous Tumor Infiltrating Lymphocytes and High-Dose Interleukin 2 in Select Solid Tumors (NCT03991741), a na phase 1 study. The primary outcome is Dose Limiting Toxicity.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03991741 |
| Phase | Phase 1 |
| Condition | Metastatic Melanoma, Locally Advanced Refractory/Recurrent Melanoma, Metastatic Head and Neck Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 3 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Gregory Daniels |
| Results posted | Yes |
2. Clinical Question
The trial addressed whether autologous tumor-infiltrating lymphocytes, given with high-dose interleukin 2 after lymphodepletion, could be administered in selected solid tumors and whether dose-limiting toxicity would occur during the stated observation period. The registry describes the cell treatment as being taken from participants’ tumors, grown in a laboratory, and returned to participants. The primary endpoint is dose-limiting toxicity, measured as a count of participants over 2 months.
This is a phase I question about treatment administration and safety in a small group, not a randomized comparison of treatment strategies. The listed conditions include metastatic and locally advanced refractory or recurrent melanoma, and metastatic and locally advanced refractory or recurrent head and neck cancer. The registry does not report a separate primary endpoint result by condition or provide efficacy outcomes in the record.
Population
Participants with the listed metastatic or locally advanced refractory or recurrent melanoma or head and neck cancer conditions. Enrollment was 3.
Intervention
Autologous tumor-infiltrating lymphocytes and high-dose interleukin 2, following lymphodepletion as described in the official trial title.
Comparator
There was no separate control group: the registry describes a single-group design and allocation as not applicable.
Primary question
How many participants experienced dose-limiting toxicity during the 2-month primary endpoint timeframe?
3. Trial Design
The registry classifies this study as phase I, single-group, and unmasked. Allocation is listed as not applicable. These features mean that the record does not describe random assignment to competing treatment groups or masking of participants or study personnel. The intervention list names autologous tumor-infiltrating lymphocytes and high-dose interleukin 2; the official title describes lymphodepletion before cell transfer and interleukin 2.
The study enrolled 3 participants. Its status is terminated. The registry lists 2020-10-07 as the start date and 2023-01-26 as the primary completion date. The record does not explain why the trial was terminated, so no reason can be inferred from the status alone. It also does not report whether enrollment ended as planned or whether the listed participants all completed the primary endpoint observation period.
The conditions listed cover metastatic melanoma, locally advanced refractory or recurrent melanoma, metastatic head and neck cancer, and locally advanced refractory or recurrent head and neck cancer. The arm description is “Solid Tumor.” The registry does not report participant characteristics, the distribution of participants across the listed conditions, or a more detailed account of treatment delivery in the outcome record.
A single-group design can describe what was recorded among participants who received the study regimen, but it does not provide a concurrent control estimate. Differences between treated participants and an external population cannot be attributed to treatment using this design alone. No protocol-level sample-size justification, interim-analysis plan, or detailed statistical analysis plan is 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 | Solid Tumor |
|---|---|
| STARTED | 3 |
| COMPLETED | 3 |
| NOT COMPLETED | 0 |
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 | Solid Tumor |
|---|---|---|
| Participants analysed | 3 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 2 |
| Age, Categorical: >=65 years | Participants | 1 |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 3 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 2 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 2 |
| Race (NIH/OMB): More than one race | Participants | 1 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Dose Limiting Toxicity, measured over 2 months.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is titled “Dose Limiting Toxicity,” with the unit specified as participants and the measure defined as a count of participants. Its timeframe is 2 months. In general, a dose-limiting toxicity endpoint is used in early-phase research to describe whether participants experience toxicity considered important to treatment delivery or dose evaluation within a specified observation period. The registry record does not give a more detailed operational definition of DLT, including the criteria used to classify an event as dose limiting.
A participant count is a binary-outcome summary at the participant level: a participant is counted according to whether the endpoint occurred, rather than counting each episode as a separate participant. This distinction matters because multiple events in one participant do not necessarily represent multiple participants with the endpoint. The registry record does not state whether repeated events were handled in any particular way, or whether the endpoint required independent adjudication.
Although the record identifies one primary outcome measure, it does not post the number of participants with DLT. Therefore, the endpoint’s observed result cannot be described from this record. The enrollment count does not substitute for that missing outcome count, and the listed adverse-event figures should not be treated as the DLT result: the registry does not identify those figures as dose-limiting toxicity.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted record supports a limited description of the study: it was a phase I, single-group trial with enrollment of 3, and it was terminated. The registry lists a primary endpoint of dose-limiting toxicity measured as a participant count over 2 months, but the endpoint result itself is not reported in the registry record. Consequently, the number of participants who experienced DLT cannot be stated, and no estimate of the frequency of DLT can be interpreted from the record.
Safety information is recorded separately. For the Solid Tumor group, the registry lists 3 participants at risk and 3 affected by serious adverse events. It also lists 3 participants at risk and 3 affected by other adverse events, and reports 3 deaths. The serious adverse-event terms with the highest reported frequencies are pericardial effusion, lung infection, and sepsis, each listed as 33.3%. These are descriptive registry figures. They do not establish that the events were caused by the intervention, and the record does not state whether any of them met the DLT definition.
The small enrollment means that the record describes only a very limited group of participants. Even when a count is available, a count from a small group would give limited precision for estimating how often an outcome occurs in a broader population. General statistical principle: an estimate based on few observations is sensitive to each individual outcome, and uncertainty should be conveyed rather than treating the observed experience as a precise population rate. Here, the primary endpoint count and an uncertainty interval are not reported, so neither a DLT estimate nor its precision can be assessed.
The single-group design also limits comparative interpretation. Without a control group, the record cannot estimate how outcomes would have differed under another treatment or under no study intervention. The registry lists no outcome analyses, and no efficacy endpoint result is included in the record. The safety counts therefore should not be read as comparative effects or evidence of treatment benefit or lack of benefit.
The termination status is relevant context, but it does not explain why the study stopped. The reason is not reported in the registry record. Likewise, the record does not establish whether the planned enrollment was reached, whether all participants had complete follow-up, or whether missing outcomes influenced the posted results. These uncertainties limit interpretation beyond the descriptive information that is explicitly recorded.
9. Statistical Methods Explained
The registry identifies a binary primary endpoint: dose-limiting toxicity counted as participants, with a 2-month timeframe. A binary endpoint records whether a defined condition occurred for a participant. In general, a count is useful because it communicates how many participants met the endpoint, but interpretation also depends on a clear endpoint definition, a stated analysis population, and an account of missing observations. Those details, along with the actual DLT count, are not reported in the registry record.
What does a participant count tell the reader?
A count of participants with an event differs from a count of event episodes. If a participant has more than one episode, a participant-level endpoint may still count that person once, depending on the prespecified definition. The registry says the primary measure is a count of participants, but does not report its detailed counting rules. It also does not post the primary endpoint result. The enrollment count alone cannot reveal how many participants met the DLT endpoint.
General statistical principle: an estimate of an event frequency is more informative when accompanied by an uncertainty interval. A confidence interval represents a range of values compatible with the data and the statistical method under its assumptions; it does not guarantee that the true value lies in that range. Neither a DLT estimate nor a confidence interval for that endpoint is reported in the registry record. No p-value or inferential analysis for the primary endpoint is reported either.
Why does the design matter?
The trial is listed as single-group, with allocation not applicable and masking none. A single-group result is descriptive of the participants studied. It does not provide a randomized comparison, and it cannot separate the intervention’s effects from other influences on outcomes. General statistical principle: random allocation can help balance measured and unmeasured factors between comparison groups, while masking can reduce some forms of knowledge-related influence on assessment. Neither a randomized comparator nor masking is described for this trial.
The listed interventions are autologous tumor-infiltrating lymphocytes and high-dose interleukin 2, while the official title also describes lymphodepletion before cell transfer. The registry record does not provide a statistical comparison that separates the contribution of each component. It also does not report a dose-escalation method or a specific rule for selecting a dose. Such details should not be inferred from the phase label or the endpoint name.
What can the planning illustration establish?
The stated assumptions for planning illustrations use a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect defined as a stated fraction of the observed effect. The instructions distinguish total events for time-to-event outcomes from participants per arm for other outcomes. These are assumptions for illustrations, not evidence that the trial used those methods in its own design. In particular, the actual study is listed as single-group, whereas the illustration assumes two arms. No calculated participant-per-arm values are present in the registry record.
General statistical principle: sample-size calculations depend on assumptions about the outcome, the effect a study aims to detect, variability, and the analysis. A planning illustration can show how such assumptions relate to a hypothetical design, but it does not describe the number of participants actually observed and is not observed power. The registry does not report its own sample-size justification or enough primary endpoint results to determine an observed effect for this trial.
Why are the safety counts not a DLT analysis?
The adverse-event record lists participants affected by serious and other adverse events, as well as deaths. Those summaries are not labeled as the primary endpoint and do not identify events as dose limiting. General statistical principle: safety categories and endpoint classifications can overlap, but they answer different questions unless the protocol explicitly links them. Because that linkage and the DLT criteria are not reported, the serious adverse-event counts cannot be substituted for the missing DLT count.
The record lists no outcome analyses. It does not report an analysis population, a method for handling missing observations, covariate adjustment, or interim analyses. It also does not report whether any confidence intervals or hypothesis tests were planned for safety outcomes. Without those details, a reader can describe the registry’s recorded counts and design classifications but cannot reconstruct an analysis plan or add unreported inferential results.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity concerns arise when several outcomes, time points, groups, or statistical tests are assessed. In general, conducting many tests can increase the chance of a seemingly notable result arising by chance, so a study may prespecify how such tests are prioritized or adjusted. The registry identifies one primary outcome measure, dose-limiting toxicity, but lists no outcome analyses. Any formal multiplicity strategy, hierarchy of secondary outcomes, or adjustment method is not reported in the registry record.
The record does not report an interim-analysis plan, stopping boundaries, or whether the termination decision followed a prespecified statistical rule. The status is terminated, but the reason for termination is not reported in the registry record. It would be inappropriate to infer that termination was driven by safety, efficacy, futility, recruitment, or a statistical threshold.
Missing-data handling is also not reported in the registry record. The record does not state whether the primary endpoint was available for every enrolled participant, how incomplete follow-up was handled, or whether any participants were excluded from an analysis. Enrollment of 3 does not establish that 3 participants contributed complete primary endpoint data.
The analysis populations are not reported in the registry record. There is no stated intention-to-treat, treated-participant, or evaluable-participant population, and no analysis set can be inferred. These omissions matter because counts and estimates can depend on which participants are included and how endpoint availability is defined. With no posted primary endpoint count and no analyses, conclusions should remain descriptive of the registry entries that are recorded.
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 |
|---|---|---|---|
| Solid Tumor | 3 | 3 | 100.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Solid Tumor | 3 of 3 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Solid Tumor | 3 of 3 |
Most frequent serious adverse event terms
| Event term | Organ system | Solid Tumor |
|---|---|---|
| Pericardial effusion | Cardiac disorders | 1 of 3 |
| Lung infection | Respiratory, thoracic and mediastinal disorders | 1 of 3 |
| Sepsis | Infections and infestations | 1 of 3 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Solid Tumor |
|---|---|---|
| Febrile neutropenia | Blood and lymphatic system disorders | 3 of 3 |
| Nausea | Gastrointestinal disorders | 3 of 3 |
| Fatigue | General disorders | 3 of 3 |
| Platelet count decreased | Investigations | 3 of 3 |
| Anemia | Blood and lymphatic system disorders | 2 of 3 |
| Constipation | Gastrointestinal disorders | 2 of 3 |
| Diarrhea | Gastrointestinal disorders | 2 of 3 |
| Cytokine release syndrome | Immune system disorders | 2 of 3 |
| Sepsis | Infections and infestations | 2 of 3 |
| Alanine aminotransferase increased | Investigations | 2 of 3 |
Serious events with the highest arm-level rate: pericardial effusion (33.3%), lung infection (33.3%), sepsis (33.3%).
For the Solid Tumor group, the registry lists 3 participants at risk for serious adverse events and 3 affected. It also lists 3 at risk for other adverse events and 3 affected. The record reports 3 deaths. These are participant counts, not estimates of comparative risk, and the registry does not state that the events or deaths were caused by the study intervention.
The most frequently listed serious adverse-event terms are pericardial effusion, lung infection, and sepsis, each reported as 33.3%. The registry record does not clarify whether participants could appear under more than one term, whether terms were coded at a particular level of detail, or whether the terms were considered treatment-related. The percentages should therefore be read as the registry’s reported frequencies, not as evidence about cause.
Serious adverse events, other adverse events, deaths, and dose-limiting toxicity are distinct reporting categories. The primary DLT result is not reported in the registry record, and the serious-event counts do not establish which events, if any, met the DLT definition. The record also does not report event timing, severity grades, resolution, or attribution. The small enrollment further means that these figures describe only the participants recorded in this study and cannot characterize safety in a broader population.
12. What the Result Does, and Does Not, Mean
The registry record establishes that this was a phase I, single-group study of autologous tumor-infiltrating lymphocytes and high-dose interleukin 2 in selected solid tumors, with lymphodepletion described in the official title. It records enrollment of 3, a terminated status, a primary DLT endpoint with a 2-month timeframe, and safety counts for the Solid Tumor group. Those are descriptive details about the registered study.
The primary endpoint result is not reported in the registry record. The record therefore does not show how many participants experienced DLT, and it does not permit an estimate of the frequency or precision of that endpoint. The adverse-event counts cannot fill that gap because the record does not identify them as DLT outcomes. Nor does the record provide comparative efficacy results.
General statistical principle: a single-group study cannot by itself show what would have happened to the same participants under a different treatment. It can describe observed outcomes in the group, but it does not provide a concurrent counterfactual comparison. Even if an outcome count were available, interpretation would need to account for the small enrollment, endpoint definition, completeness of follow-up, and uncertainty.
The listed serious adverse-event frequencies and death count warrant careful descriptive reading, but their relationship to treatment is not stated. The registry does not report causes of death or event attribution, and it does not give the reason for termination. These registry entries should not be turned into claims that the regimen caused or prevented an outcome.
Accordingly, the record does not establish treatment efficacy, comparative safety, or a generalizable rate of DLT. It also does not establish that the study’s intended enrollment or analysis plan was completed. The appropriate interpretation is narrower: the registry documents a small, terminated, single-group trial and selected safety summaries, while leaving the primary endpoint result and several key analysis details unreported.
13. Why This Trial Matters Statistically
This trial is statistically notable because it illustrates the difference between a registered endpoint and a reported endpoint result. The registry names dose-limiting toxicity, specifies participants as the unit, and gives a 2-month timeframe, but does not report the count. Without that result, readers cannot assess the primary endpoint’s observed frequency or its precision. An endpoint label alone is not an analysis result.
The design also sets a clear boundary on interpretation. The study is listed as single-group and unmasked, with allocation not applicable. Its observations can describe the participants recorded, but there is no randomized control group against which to estimate a treatment contrast. This distinction is important when readers encounter safety counts or other descriptive information: those figures do not answer a comparative question.
Enrollment was 3, which makes the scope of the record especially narrow. General statistical principle: in a small study, each participant’s outcome can have substantial influence on a descriptive summary, and estimates tend to be imprecise. A precise-looking percentage does not remove that limitation. The posted serious-event percentages should be read alongside the participant counts and the absence of causal attribution.
The stated planning assumptions also illustrate why calculations must be kept separate from the trial’s actual design and evidence. The assumptions describe a hypothetical two-arm planning setup, while the trial is registered as single-group. Such illustrations are not observed results, and they do not replace a reported sample-size justification or primary endpoint analysis. The record does not report the rationale for termination, the analysis population, or missing-data 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.
Several trial-specific gaps restrict interpretation. The most consequential is that the registry does not report the primary dose-limiting toxicity count, despite identifying the endpoint, its participant-count unit, and its 2-month timeframe. The endpoint’s operational criteria are also not reported in the registry record. Without both a result and a detailed definition, the primary safety question cannot be evaluated.
Enrollment was 3, and the record does not give participant-level descriptions or the distribution of participants across the listed cancer conditions. The Solid Tumor arm label does not resolve that uncertainty. The record also does not report whether every enrolled participant completed follow-up or contributed to the primary endpoint analysis.
The trial was terminated, but the reason is not reported in the registry record. No analysis populations, missing-data procedures, interim analyses, or formal statistical analysis methods are reported. The record does not provide a trial-specific sample-size justification. The planning assumptions described for illustrations use two-arm allocation, unlike this study’s registered single-group design, and no calculated planning values are reported in the record.
The safety record does not report event attribution, timing, severity grading, or causes of death. It does not identify whether serious adverse events met the DLT definition. Because there is no concurrent control group, the record cannot provide a randomized comparative estimate. These limitations mean that the available figures should be treated as registry descriptions, not as a complete account of the trial’s clinical or statistical findings.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Adoptive Cell Transfer of Autologous Tumor Infiltrating Lymphocytes and High-Dose Interleukin 2 in Select Solid Tumors — NCT03991741.
- Registry record last updated 2025-07-03; status last verified 2025-06.
- Results first posted 2025-07-03.
- 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 phase I, unmasked, single-group trial of autologous tumor-infiltrating lymphocytes and high-dose interleukin 2 in selected solid tumors. The official title describes lymphodepletion before cell transfer. Enrollment was 3, and the study status is terminated. The listed primary endpoint is dose-limiting toxicity, measured as a participant count over 2 months.
The primary endpoint result is not reported in the registry record, and the record lists no outcome analyses. For the Solid Tumor group, it records 3 participants at risk and 3 affected by serious adverse events, 3 at risk and 3 affected by other adverse events, and 3 deaths. Pericardial effusion, lung infection, and sepsis are each listed among the leading serious-event terms at 33.3%. The record does not report event attribution or the reason for termination.