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 TIL Therapy in Combination With Checkpoint Inhibitors for Metastatic Ovarian Cancer (NCT03287674), a na phase 1, phase 2 study. The primary outcome is Number of Participants With Reported Adverse Events by Type.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03287674 |
| Phase | Phase 1, Phase 2 |
| Condition | Metastatic Ovarian Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 7 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Inge Marie Svane |
| Results posted | Yes |
2. Clinical Question
Population
Patients with advanced ovarian, fallopian tube, or primary peritoneal cancer. The registry lists metastatic ovarian cancer as the condition and records enrollment of 7 participants.
Intervention
A single-group treatment program combining tumor-infiltrating lymphocyte (TIL) therapy with checkpoint inhibitors. Listed interventions include ipilimumab, nivolumab, TIL infusion, cyclophosphamide, fludarabine, and interleukin-2.
Comparator
There is no separate comparator group. The record describes all patients as receiving the same treatment, so the trial does not provide a like-for-like control comparison.
Primary question
How many participants experienced reported adverse events by type during follow-up of up to 12 months, as a way to describe the safety of TIL therapy combined with checkpoint inhibitors?
The central question is descriptive: what adverse events were recorded among participants receiving this treatment program? Because all participants were assigned to one group, the record cannot directly estimate how event rates compare with another treatment or with no treatment. It can report events observed in the treated group, but those observations alone do not establish that the treatment caused each event.
The registry also names treatment-related immune responses, objective response rate, overall survival, and progression-free survival as secondary outcomes. Their names indicate areas of interest, not findings. Outcome values for these measures are not reported in the registry record. The posted material therefore supports a reading of the safety summary, but it does not establish what tumor response or survival outcomes occurred.
3. Trial Design
This was a completed Phase 1/Phase 2 trial with a single-group model and no masking. The registry records enrollment of 7 participants. It lists a start date of 2017-10-09 and a primary completion date of 2020-06-01. Allocation is listed as not applicable because the trial was not a randomized comparison. The record does not describe a control group.
The treatment description is a combination program rather than a comparison of separate interventions. The brief summary describes TILs isolated from a participant’s tumor tissue, expanded and activated in vitro for around 4-6 weeks, and then infused after preconditioning chemotherapy with cyclophosphamide and fludarabine. It also describes interleukin-2 after infusion. The arm description says all patients receive the same treatment and states that one dose of ipilimumab is given 14 days before surgical removal of tumor tissue for TIL expansion. It lists nivolumab among the interventions. The remaining arm description is incomplete in the registry record, so the full schedule cannot be reconstructed from this entry.
The primary outcome is the number of participants with reported adverse events by type, with a timeframe of up to 12 months. This is a safety-focused outcome, not a measure of comparative efficacy. The registry lists objective response rate, treatment-related immune responses, overall survival, and progression-free survival as secondary outcomes, but their results are not reported in the registry record. The posted safety summary has a denominator of 6, which differs from the enrollment of 7; the reason for this difference is not reported in the registry record. The design and available results should therefore be read as a small descriptive account, not as a controlled estimate of treatment effects.
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 | TIL Treated Patients |
|---|---|
| STARTED | 7 |
| All Treated With TILs | 6 |
| COMPLETED | 6 |
| NOT COMPLETED | 1 |
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 | TIL Treated Patients |
|---|---|---|
| Participants analysed | 6 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 6 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 6 |
| Sex: Female, Male: Male | Participants | 0 |
| Race and Ethnicity Not Collected | Participants | — |
| Region of Enrollment: Denmark | participants | 6 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Reported Adverse Events by Type, measured over Up to 12 months.
| 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 reported adverse events by type. The registry specifies that adverse events are to be reported according to CTCAE v. 4.0, with a timeframe of up to 12 months. The reported measure is a participant count: it concerns how many people had an event, rather than the total number of times an event occurred.
A count of participants with events is useful in a safety-focused trial because it gives a direct description of how many treated participants were recorded as affected. Grouping events by type can also show which categories appear in the posted safety summary. However, this endpoint does not by itself describe event severity in full, duration, reversibility, timing, or whether an event was judged to be caused by treatment. Those details are not reported in the registry record for the summary.
The endpoint also has no comparison group. A count among treated participants cannot show how many events would have occurred under a different treatment or without the treatment program. The posted primary outcome is therefore best read as a descriptive safety measure within this trial. It is not a comparative estimate of risk, and it does not answer the secondary questions about response or survival. Results for those secondary outcomes are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record describes a completed, single-group Phase 1/Phase 2 trial with enrollment of 7. The posted safety summary, however, reports a group of 6 TIL-treated patients, with 6 at risk for serious adverse events and 6 affected. It also reports 6 at risk for other adverse events and 6 affected. The distinction between enrolled participants and the safety-summary denominator matters: the registry record does not explain why the figures differ, so the summary should not be assumed to include every enrolled participant.
The serious adverse-event terms most frequently listed include neutropenia, thrombocytopenia, and anemia, each at 100.0%. Fatigue, hyponatremia, and fever are each listed at 50.0%. These are the percentages printed in the registry record. They describe the listed safety group; they are not comparisons with another group. The record also lists deaths as 6. It does not give enough information to interpret the relationship between those deaths and the adverse-event categories, their timing, their causes, or whether they were considered treatment-related. Those details are not reported in the registry record.
These figures should be read with the small safety denominator in mind. In a small group, each participant represents a substantial portion of the observed count, so the observed percentages can be unstable and may not represent what would be seen in a larger group. The registry does not provide confidence intervals or other uncertainty estimates for these safety figures. The descriptive counts alone cannot distinguish events caused by treatment from events related to the underlying illness, other care, or other circumstances.
The primary endpoint concerns adverse events. Although the registry names treatment-related immune responses, objective response rate, overall survival, and progression-free survival as secondary outcomes, their results are not reported in the registry record. No efficacy conclusion can be drawn from outcome names alone. Similarly, the sponsor’s stated rationale that the combination might increase clinical effect is a motivation for the trial, not a posted efficacy result.
There is no randomized or masked comparator. Consequently, the record cannot establish whether the observed safety profile differs from another treatment, nor can it isolate the contribution of any one component of the multi-part regimen. The sample-size section accompanying this page is a planning illustration, not evidence of the trial’s observed power or a substitute for reported outcomes. Overall, the posted data support a narrow reading: the registry records adverse events in the treated group, but provides limited information for causal, comparative, or efficacy interpretation.
9. Statistical Methods Explained
The available primary outcome is a count of participants with reported adverse events by type. That makes the central statistical object a participant-level event count: each participant is classified according to whether an event of a given type was reported. This is different from counting every event episode. A participant with repeated episodes may still contribute as one participant to a measure defined as the number of participants affected. The registry record does not provide event-level detail for interpreting recurrence.
What does a participant count tell us?
As a general statistical explanation, a count is easiest to interpret alongside its denominator. The denominator identifies how many participants were considered at risk or included in the summary. Here, the posted safety group reports 6 at risk for serious events and 6 affected, and 6 at risk for other events and 6 affected. Enrollment is recorded as 7, but the reason the adverse-event denominator is 6 is not reported in the registry record. Those figures should be kept distinct rather than treated as interchangeable.
A participant count can describe what was observed in the group, but it does not automatically estimate a stable rate for a wider population. In a small sample, an observation about one participant can materially change the apparent pattern. General statistical practice therefore often pairs an estimate with an uncertainty interval. No confidence interval is reported for the safety figures. A confidence interval, in general, expresses a range of values compatible with the data under a specified method; it does not remove limitations in study design or make a small uncontrolled comparison causal.
Why does the study design matter?
This trial is recorded as single-group, with allocation not applicable and masking absent. The design provides no concurrent control group against which to compare counts. In general, random allocation can help make comparison groups similar in expectation, while masking can reduce some forms of measurement or assessment bias. Neither a randomized comparator nor masking is recorded. As a result, the observed safety counts cannot tell us whether events were more or less common than they would have been under another approach.
The treatment program also includes several listed components: TIL infusion, checkpoint inhibitors, chemotherapy agents, and interleukin-2. A single-group combination study cannot separate the effect of each component using the posted summary alone. The registry does not report a statistical model that adjusts for other factors or attributes events to individual components. It also does not report causality assessments for the listed events in the summary used.
What can be inferred from the posted numbers?
The serious-event terms listed at 100.0% are neutropenia, thrombocytopenia, and anemia; fatigue, hyponatremia, and fever are listed at 50.0%. These reported percentages describe the group as tabulated. They should not be interpreted as comparative risks or as precise population estimates. They also do not indicate when an event occurred, how long it lasted, or whether it was judged related to treatment. Such detail is not reported in the registry record.
The registry names secondary outcomes for immune responses, objective response, overall survival, and progression-free survival. The existence of an outcome name is not evidence that a result is available. Results for these measures are not reported in the registry record. No inferential analyses are listed, and the number of analyses is recorded as 0. The registry does not report a statistical analysis plan, adjustment methods, or criteria for handling multiple outcomes. It is therefore not possible to determine which inferential procedures, if any, were planned for outcomes beyond the descriptive safety count.
How should the planning calculations be read?
The accompanying sample-size section is explicitly a planning illustration computed from an observed effect, not the trial’s own design. Its stated assumptions include a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is expressed as a fraction of the observed effect. For time-to-event outcomes, the illustration is expressed as a total number of events, while other outcomes are expressed as participants per arm. These calculations are planning scenarios, not observed results, and they do not mean the trial had a particular level of observed power. The trial itself is single-group and does not have the two-arm allocation assumed by that illustration.
In general, a sample-size calculation asks how much information a planned design might need to detect a specified effect under stated assumptions. Its answer depends on those assumptions and on the effect used. It cannot be treated as proof that a result was detectable or that a non-significant result indicates no effect. The trial’s own sample-size justification is not reported in the registry record. No new calculation can be inferred from the posted safety counts alone.
General methods such as regression, time-to-event analysis, or methods for over-dispersed counts are useful only when suited to the outcome and design and when the relevant data and model are available. The registry record does not report use of those methods. For this page, the defensible interpretation is descriptive: identify the recorded group, event types, denominators, and missing statistical details without implying an unreported analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry names several outcomes: the primary safety measure and secondary measures covering treatment-related immune responses, objective response rate, overall survival, and progression-free survival. Considering multiple outcomes can raise a multiplicity issue. In general, if many statistical tests are conducted, the chance of at least one apparently noteworthy result can increase unless the analysis plan accounts for this. The registry record does not report whether any multiplicity adjustment was planned or used, and it does not report a statistical analysis plan or inferential results for the secondary outcomes.
Missing-data handling is also not described. The safety summary has 6 participants at risk, while total enrollment is 7, and the reason for the difference is not reported in the registry record. The available entry does not explain whether any participant lacked follow-up, was excluded from a safety set, or had incomplete event reporting. It would be inappropriate to assign a reason or to assume that the safety summary includes all enrolled participants.
The analysis populations are not described beyond the group identified as TIL-treated patients in the adverse-event summary. The registry record does not state whether outcomes were analyzed according to enrollment, treatment received, or another population definition. It also does not report interim analyses, stopping rules, or a missing-data strategy. These absences limit evaluation of how complete and consistent the outcome summaries are. They do not establish that particular methods were or were not used outside the registry; they mean those methods are not reported in the registry record.
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 |
|---|---|---|---|
| TIL Treated Patients | 6 | 6 | 100.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| TIL Treated Patients | 6 of 6 |
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 |
|---|---|
| TIL Treated Patients | 6 of 6 |
Most frequent serious adverse event terms
| Event term | Organ system | TIL Treated Patients |
|---|---|---|
| Neutropenia | Blood and lymphatic system disorders | 6 of 6 |
| Trombocytopenia | Blood and lymphatic system disorders | 6 of 6 |
| Anemia | Blood and lymphatic system disorders | 6 of 6 |
| Fatigue | General disorders | 3 of 6 |
| Hyponatremia | Renal and urinary disorders | 3 of 6 |
| Fever | General disorders | 3 of 6 |
| Colitis | Immune system disorders | 1 of 6 |
| Agammaglobulimia | Blood and lymphatic system disorders | 1 of 6 |
| Infection | Renal and urinary disorders | 1 of 6 |
| Infection | Respiratory, thoracic and mediastinal disorders | 1 of 6 |
Most frequent non-serious adverse event terms
| Event term | Organ system | TIL Treated Patients |
|---|---|---|
| diarrhea | Gastrointestinal disorders | 6 of 6 |
| nausea | General disorders | 5 of 6 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 4 of 6 |
| Vomiting | Gastrointestinal disorders | 3 of 6 |
| Oral mucositis | Gastrointestinal disorders | 3 of 6 |
| Infection | Infections and infestations | 2 of 6 |
| Vomiting | Gastrointestinal disorders | 2 of 6 |
| Obstipation | Gastrointestinal disorders | 2 of 6 |
| Thyroiditis | Endocrine disorders | 1 of 6 |
| Elevated liver enzymes | Hepatobiliary disorders | 1 of 6 |
Serious events with the highest arm-level rate: neutropenia (100.0%), trombocytopenia (100.0%), anemia (100.0%), fatigue (50.0%), hyponatremia (50.0%).
The safety summary lists 6 participants at risk for serious adverse events and 6 affected. It also lists 6 at risk for other adverse events and 6 affected. The group is identified as TIL-treated patients. The event terms listed include neutropenia, thrombocytopenia, and anemia at 100.0% each, and fatigue, hyponatremia, and fever at 50.0% each. These figures are the registry’s reported values, not comparisons against a control group.
The record lists deaths as 6. It does not report the timing or causes of these deaths in the safety summary, or clarify their relationship to adverse events or treatment. The listed counts also do not establish that an event was caused by the treatment. Attribution, event duration, and the detailed severity of each event are not reported in the registry record.
Enrollment is 7, whereas the adverse-event summary uses 6 as the number at risk. The reason for this difference is not reported. Read the safety percentages with that denominator in view, and do not assume the summary represents all enrolled participants. Because there is no comparator group and the safety denominator is small, these figures describe only the posted experience in this group; they do not quantify comparative risk.
12. What the Result Does, and Does Not, Mean
The posted result is a descriptive safety summary from a completed, single-group trial. It reports adverse-event counts and selected event types among the TIL-treated group. The listed serious-event terms include neutropenia, thrombocytopenia, and anemia at 100.0%, along with fatigue, hyponatremia, and fever at 50.0%. The summary also records 6 participants affected among 6 at risk for serious adverse events and 6 affected among 6 at risk for other adverse events. These are the observations recorded in the registry, not estimates from a controlled comparison.
The record does not show whether the events were caused by TIL therapy, checkpoint inhibitors, another component of the treatment program, the underlying disease, or other circumstances. It also does not provide enough detail to interpret the deaths listed in the summary. The posted figures do not establish the timing, duration, reversibility, or treatment attribution of the events.
The trial cannot answer whether this treatment program produces more or fewer adverse events than another approach because it has no comparator group. Its single-group design also cannot isolate the contribution of individual interventions. The difference between enrollment of 7 and the safety denominator of 6 is unexplained in the registry record.
Although the registry lists response and survival outcomes, their values are not reported in the registry record. Thus, the posted entry does not support a conclusion about tumor response, progression, or survival. The safety figures should not be taken as proof that treatment is effective or ineffective, nor should they be generalized as precise estimates for a broader population. They are a limited account of reported events in a small treated group, with important design and reporting constraints.
13. Why This Trial Matters Statistically
This trial raises statistical questions that are important in early-phase and small single-group research. Its primary outcome is safety-focused, and the registry reports event counts rather than a randomized comparison. Such summaries can make the observed experience visible, but interpreting them requires close attention to the denominator, outcome definition, and completeness of follow-up.
Here, enrollment is recorded as 7, while the adverse-event group has 6 participants at risk. That discrepancy in denominators is consequential for reading the reported percentages, yet its explanation is not reported in the registry record. The posted safety terms are also presented without the comparative context a control group would provide. A descriptive summary is not a comparative estimate, and it cannot determine which component of a combination regimen accounts for an event.
The entry also illustrates the difference between listing an outcome and reporting its result. Immune responses, objective response, overall survival, and progression-free survival are named as secondary outcomes, but values for them are not reported in the registry record. Likewise, the record does not document a statistical analysis plan or multiplicity approach. These absences limit assessment of how outcomes were analyzed and how uncertainty should be understood.
Finally, the sample-size material is a planning illustration based on stated assumptions, rather than evidence of the trial’s power or design justification. Keeping planning calculations separate from observed data is essential. The statistical value of this record lies in clarifying what it does show—reported safety observations in a treated group—and what it does not establish about comparisons, causality, or efficacy.
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 limitations specific to this trial affect interpretation. The study is single-group, unmasked, and has no control comparison. Therefore, the observed adverse-event counts cannot be compared directly with outcomes under another treatment, and the contribution of individual components in the combination program cannot be separated. The registry record does not report a statistical analysis plan, inferential methods, or confidence intervals for the safety summary.
The record lists enrollment of 7, while the adverse-event group includes 6 participants at risk. The reason for the different denominators is not reported in the registry record. This prevents a clear assessment of how the safety group relates to all enrolled participants. The registry lists deaths as 6 but does not provide the detail needed to interpret timing, cause, or attribution.
The arm description is incomplete, so the full treatment schedule cannot be reconstructed from this entry. Although the brief summary describes several treatment steps and the intervention list includes nivolumab, the available record does not supply a complete account of the regimen. Event timing and treatment-relatedness are also not reported in the posted safety summary.
Results for the named secondary outcomes are not reported in the registry record. The entry therefore cannot support conclusions about response or survival. Finally, the small safety group limits how precisely its observations can characterize a broader population. These are limitations of the available record and design; they do not establish what may have been collected or analyzed outside the registry.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: TIL Therapy in Combination With Checkpoint Inhibitors for Metastatic Ovarian Cancer — NCT03287674.
- Registry record last updated 2023-03-01; status last verified 2023-02.
- Results first posted 2021-03-22.
- 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.
18. Record Summary
This completed Phase 1/Phase 2 trial evaluated a single-group treatment program involving TIL therapy and checkpoint inhibitors for advanced ovarian, fallopian tube, or primary peritoneal cancer. The registry records enrollment of 7 and dates the study from 2017-10-09 to primary completion on 2020-06-01. The primary outcome is the number of participants with reported adverse events by type, through up to 12 months.
The adverse-event summary describes 6 TIL-treated patients at risk, with 6 affected for both serious and other adverse events. It lists neutropenia, thrombocytopenia, and anemia at 100.0% each; fatigue, hyponatremia, and fever at 50.0% each; and deaths as 6. The reason for the difference between enrollment and the safety denominator, and further details about the listed deaths, are not reported in the registry record.
The registry names immune responses, objective response, overall survival, and progression-free survival as secondary outcomes, but their results are not reported. With no comparator group and limited statistical detail, the posted record supports descriptive safety interpretation, not comparative or efficacy conclusions.