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 Autologous Tumor Infiltrating Lymphocytes MDA-TIL in Treating Patients With Recurrent or Refractory Ovarian Cancer, Colorectal Cancer, or Pancreatic Ductal Adenocarcinoma (NCT03610490), a na phase 2 study. The primary outcome is Objective Response Rate (ORR) in Participants With Ovarian Cancer (OVCA), PDAC (Pancreatic Cancer), and Colorectal Cancers (CRC) According to RECIST v1.1 in Subjects With Ovarian Cancer (OVCA), PDAC (Pancreatic Cancer), and Colorectal Cancers (CRC).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03610490 |
| Phase | Phase 2 |
| Condition | Malignant Solid Neoplasm, Metastatic Colorectal Adenocarcinoma, Metastatic Ovarian Carcinoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 16 |
| Arms in the primary outcome | n/a |
| Lead sponsor | M.D. Anderson Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
Participants with recurrent or refractory ovarian cancer, colorectal cancer, or pancreatic ductal adenocarcinoma. The registry lists an enrollment of 16 and describes the primary endpoint for participants with these three cancer types.
Intervention
Autologous expanded tumor-infiltrating lymphocytes, called MDA-TIL, administered intravenously. The treatment description includes cyclophosphamide and fludarabine as a lymphodepletion regimen before T-cell infusion. Interleukin-2 is listed as an intervention, but its administration details are not reported in the registry record.
Comparator
There is no separate comparator group. The study is registered as a single-group trial with no masking and no randomized allocation.
Primary question
What proportion of treated participants with ovarian cancer, pancreatic ductal adenocarcinoma, or colorectal cancer have a confirmed complete or partial response under RECIST v1.1 through 4 years?
This is a question about the frequency of an observed tumor response in a treated group, not a direct comparison between MDA-TIL and another treatment. The registry’s primary endpoint description defines objective response rate as the number of participants with a confirmed complete response or partial response divided by the All-Treated analysis set, expressed as a percentage. It specifies a 97.5% confidence interval for the endpoint.
The study combines three cancer types in its stated primary question. The registry record does not report whether results were intended to be presented separately by cancer type, how such subgroups would be handled, or what criteria determined eligibility beyond the listed recurrent or refractory diseases. It also does not report an observed response rate. Consequently, the record identifies the question and its measurement framework, but does not establish what response proportion was observed.
3. Trial Design
This is a phase II, single-group study sponsored by M.D. Anderson Cancer Center. The registry lists an enrollment of 16. Allocation is recorded as not applicable, masking as none, and the study model as single group. There is no randomized or concurrent control group in the design described by the record.
The treatment regimen includes a lymphodepletion phase followed by an intravenous infusion of autologous tumor-infiltrating lymphocytes. The arm description specifies cyclophosphamide intravenously over 2 hours on days -7 and -6, followed by fludarabine intravenously over 15-30 minutes on days -5 to -1, in the absence of disease progression or unacceptable toxicity. It then describes intravenous MDA-TIL infusion. Interleukin-2 and quality-of-life assessment are also listed as interventions, but further administration or assessment details are not reported in the registry record.
The registry lists the study start as 2018-10-16 and primary completion as 2024-08-21. Its status is terminated. The reason for termination and the number of participants who received treatment are not reported in the registry record. The enrollment figure should therefore be read as the registry’s enrollment value, not assumed to describe the number treated or the number included in each analysis.
The primary endpoint is objective response rate through 4 years, assessed according to RECIST v1.1. The endpoint description refers to confirmed complete and partial responses and an All-Treated analysis set. Secondary outcomes listed include the number of participants with stable disease and progression-free and overall survival. The registry record lists three outcome measures, but does not report statistical analyses for them. It also does not report a protocol-level sample-size justification, interim analysis plan, or detailed statistical analysis plan.
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 | Autologous TIL |
|---|---|
| STARTED | 16 |
| COMPLETED | 16 |
| 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 | Autologous TIL |
|---|---|---|
| Participants analysed | 16 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 15 |
| Age, Categorical: >=65 years | Participants | 1 |
| Age, Continuous | years | 54.4 (33 to 70) |
| Sex: Female, Male: Female | Participants | 8 |
| Sex: Female, Male: Male | Participants | 8 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 15 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 1 |
| 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 | 15 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 16 |
6. Primary Endpoint
The registry lists the primary outcome as Objective Response Rate (ORR) in Participants With Ovarian Cancer (OVCA), PDAC (Pancreatic Cancer), and Colorectal Cancers (CRC) According to RECIST v1.1 in Subjects With Ovarian Cancer (OVCA), PDAC (Pancreatic Cancer), and Colorectal Cancers (CRC), measured over Through 4 years.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 97.5% Confidence Interval |
| Unit | Precentage of Participants |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is objective response rate (ORR) among participants with ovarian cancer, pancreatic ductal adenocarcinoma, or colorectal cancer. The registry defines ORR as the number of participants with a confirmed complete response or partial response divided by the number of participants in the All-Treated analysis set, expressed as a percentage. Response is assessed according to RECIST v1.1 through 4 years. The endpoint is recorded with a 97.5% confidence interval.
ORR is a binary-outcome summary: each participant either meets the definition of a confirmed complete or partial response, or does not. The percentage describes the proportion meeting that response definition in the specified analysis population. A confidence interval conveys uncertainty around an estimated proportion; generally, it indicates a range of values compatible with the observed data under the statistical method used. It does not remove uncertainty or establish that the result would be the same in a different population.
Using a response endpoint can make tumor change a direct focus of assessment. However, the registry record does not report the observed response count, the estimated ORR, or the confidence interval. It also does not give details about response-assessment timing, confirmation requirements beyond the word confirmed, or handling of participants without an evaluable assessment. Those details are not reported in the registry record, so the endpoint definition alone cannot show the magnitude or precision of the result.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a phase II, single-group study and identifies ORR as its primary endpoint, but it does not post an ORR result. It therefore supports interpretation of the study question and its endpoint definition, not an estimate of response in the enrolled or treated group. The absence of a reported endpoint result is distinct from a result of zero responses: the record does not establish either outcome.
The stated endpoint concerns confirmed complete or partial responses according to RECIST v1.1, using the All-Treated analysis set as its denominator. The registry specifies a 97.5% confidence interval, but neither the response count nor the interval is reported. Without those values, the response proportion and its precision cannot be assessed. The interval’s stated confidence level does not by itself indicate whether the estimate is precise; that depends on the observed data and the method used.
Because the study has one group and no randomized allocation, the design does not offer a concurrent control for comparing outcomes. General statistical interpretation: an outcome observed after treatment in a single group cannot, by itself, distinguish an effect of the intervention from other explanations for the observed outcome. The record does not provide a basis for estimating a comparative treatment effect.
The study spans ovarian cancer, colorectal cancer, and pancreatic ductal adenocarcinoma. These are distinct disease groups, while the listed primary question describes an ORR across participants with these cancers. The record does not report cancer-specific response estimates or how the overall result would be interpreted in relation to the individual cancer types. With no posted response counts or subgroup results, neither pooled nor disease-specific response patterns can be characterized.
The listed enrollment is 16, and the safety grouping reports 16 participants at risk for serious and other adverse events. These figures do not establish that every enrolled participant was treated or included in the primary endpoint analysis. The primary endpoint specifically names an All-Treated analysis set, but its size is not reported in the registry record. The reason for the terminated status is also not reported. These omissions constrain interpretation independently of the endpoint’s statistical definition.
Secondary outcomes include stable disease and progression-free and overall survival. The registry record does not post their results or analyses. No statistical analysis records are listed. As a result, the posted information describes outcomes the study intended to assess but does not provide numerical evidence about those outcomes. Any conclusion about response, disease control, survival, or comparative benefit would go beyond what the registry record reports.
9. Statistical Methods Explained
The study is registered as a single-group phase II trial with no masking and no randomized allocation. Those design features determine the kinds of statistical questions the record can answer. The primary endpoint is a response proportion in a treated analysis set, rather than a contrast between randomized groups. General statistical explanation: a single-group proportion describes outcomes within that group; without a comparator, it does not estimate a difference attributable to treatment relative to another strategy.
What does the response proportion represent?
The registry defines objective response rate as the number of participants with a confirmed complete response or partial response divided by the number in the All-Treated analysis set, expressed as a percentage. This is a proportion based on a binary classification for each participant under the stated response criteria. The record identifies RECIST v1.1 and a timeframe through 4 years. It does not report the number classified as responders, the size of the All-Treated set, or the estimated proportion.
The distinction between an outcome definition and an outcome result matters. A clearly defined endpoint tells readers what was intended to be counted. It does not reveal how many participants met the definition. In this record, the result is not reported in the registry record, so the endpoint cannot be reconstructed from the available information.
How should the confidence interval be understood?
The registry specifies a 97.5% confidence interval for ORR. General statistical explanation: a confidence interval expresses sampling uncertainty around an estimate. Its width depends on the observed data and the interval method, among other factors. A narrower interval indicates greater precision under that method, while a wider interval indicates less precision. The stated confidence level alone does not reveal the interval width or whether the estimate is precise.
Neither the interval bounds nor the method for calculating the interval are reported in the registry record. No interval can therefore be interpreted from the endpoint description alone. The confidence interval is also not a guarantee that the true response proportion lies within a particular range; it is a statistical summary whose interpretation depends on the method and assumptions.
What can a single-group design establish?
The study has one treatment group, with allocation recorded as not applicable. General statistical explanation: random allocation in a comparative study can help balance measured and unmeasured factors across groups. In a single-group design there is no such concurrent comparison. The observed response proportion, if reported, would describe the participants in the analysis set. The design would not directly show how their outcomes compare with what would have happened under a different treatment or without the study treatment.
The record describes MDA-TIL infusion after a lymphodepletion regimen including cyclophosphamide and fludarabine. Interleukin-2 is listed among the interventions, but its schedule is not reported in the registry record. The record therefore describes a treatment regimen with multiple listed components, not an isolated comparison that could separate the contribution of each component.
What does the analysis population imply?
The endpoint definition names the All-Treated analysis set as its denominator. This signals that the stated ORR is intended to use that population, but the registry record does not define its membership or give its size. The registry lists enrollment of 16; enrollment and the analysis denominator are not interchangeable without further information. The number treated, the number with assessable responses, and the handling of participants who discontinue or lack response assessments are not reported in the registry record.
General statistical explanation: the choice of analysis population can affect the interpretation of a proportion. A denominator limited to participants who received treatment or had an assessment may differ from one that includes all participants who entered a study. Readers need a stated population definition and accounting of participants to understand exactly what a result represents. Those details are not available in the registry record.
Why do the listed secondary outcomes need separate results?
The record lists stable disease and progression-free and overall survival as secondary outcomes. Each outcome has a different definition and statistical interpretation. General statistical explanation: a count of participants with stable disease is a categorical outcome, while progression-free and overall survival are time-to-event outcomes. Time-to-event analyses account for the time until an event and may also account for participants whose event status is not observed during follow-up. The specific definitions, analysis methods, and numerical results for these outcomes are not reported in the registry record.
The record lists no statistical analyses. The analysis approach for the primary endpoint, survival outcomes, or subgroup summaries is therefore not reported in the registry record. No interim analyses, multiplicity procedures, or missing-data methods are described. General statistical explanation: analysis choices should be specified because they can affect which participants and observations contribute to estimates and how uncertainty is quantified. Their absence from the registry record means those choices cannot be evaluated from this record.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists ORR as the primary endpoint and identifies stable disease and progression-free and overall survival as secondary outcomes. It does not report a multiplicity procedure for evaluating more than one outcome, nor does it describe whether separate analyses were planned across ovarian, colorectal, and pancreatic cancer groups. The handling of multiple endpoints or subgroup comparisons is therefore not reported in the registry record.
General statistical explanation: when several hypotheses are assessed, the chance of at least one apparently notable result can increase if each is evaluated without accounting for the collection of tests. A multiplicity strategy may specify which outcomes are confirmatory, how error rates are controlled, or how results should be interpreted. The registry record does not state such a strategy. This does not establish that no strategy existed outside the record; it means the approach is not reported in the registry record.
Missing-data methods are also not reported. The record does not say how participants without a response assessment, those who discontinue treatment, or those with incomplete follow-up would contribute to the ORR or survival analyses. It does not provide the size of the All-Treated analysis set or a participant accounting for the primary endpoint.
The status is terminated, but the reason for termination and its relation, if any, to missing outcome data are not reported in the registry record. General statistical explanation: incomplete outcome information can affect an estimate if participants with missing data differ systematically from those with observed outcomes. Without counts and a stated handling rule, the direction or magnitude of any such effect cannot be assessed. No analysis of missingness or sensitivity analysis is reported.
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 |
|---|---|---|---|
| Autologous TIL | 1 | 16 | 6.2% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Autologous TIL | 2 of 16 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 1% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Autologous TIL | 16 of 16 |
Most frequent serious adverse event terms
| Event term | Organ system | Autologous TIL |
|---|---|---|
| Hepatobiliary disorder - drug induced liver toxicity | Hepatobiliary disorders | 1 of 16 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Autologous TIL |
|---|---|---|
| Chills | General disorders | 14 of 16 |
| Fatigue | General disorders | 14 of 16 |
| Platelet count decreased | Investigations | 13 of 16 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 12 of 16 |
| Nausea | Gastrointestinal disorders | 12 of 16 |
| Anemia | Blood and lymphatic system disorders | 9 of 16 |
| Anorexia | Metabolism and nutrition disorders | 8 of 16 |
| Constipation | Gastrointestinal disorders | 7 of 16 |
| Vomiting | Gastrointestinal disorders | 7 of 16 |
| Diarrhea | Gastrointestinal disorders | 6 of 16 |
Serious events with the highest arm-level rate: hepatobiliary disorder - drug induced liver toxicity (6.2%).
The safety summary lists one group, Autologous TIL, with 16 participants at risk for serious adverse events and 1 participant affected. For other adverse events, 16 participants were at risk and 16 were affected. The record lists 2 deaths. These figures describe the posted safety counts; they do not by themselves establish the cause of an event or a causal relationship to treatment.
The most frequently listed serious adverse event term is “Hepatobiliary disorder - drug induced liver toxicity,” reported as 6.2%. The record does not provide further detail about the event’s timing, severity, outcome, or attribution. A listed event term should not be read as proof that the treatment caused the event.
General statistical explanation: a count of participants affected is different from a count of total events, because one participant can experience more than one event. The registry record does not state whether the other-adverse-event count represents participants with at least one event or the total number of events beyond the label. Nor does this summary provide a comparative safety estimate, because there is no separate control group. The safety figures should be interpreted as descriptive counts within the listed group, with the limits of the information reported in the registry record.
12. What the Result Does, and Does Not, Mean
The registry record establishes that the study was designed to assess a response endpoint for autologous tumor-infiltrating lymphocytes among participants with ovarian cancer, colorectal cancer, or pancreatic ductal adenocarcinoma. It defines ORR using confirmed complete and partial responses under RECIST v1.1, with the All-Treated analysis set as the denominator and assessment through 4 years. It also records safety counts and lists secondary outcomes.
It does not report the primary endpoint result, confidence interval bounds, or statistical analyses. Therefore, the registry record does not show the observed response proportion or its precision. The absence of a posted result is not evidence that no participants responded, and it is not evidence that all participants responded. It is simply a lack of a reported result in this record.
The single-group design also sets a limit on what even a reported response proportion could mean. General statistical explanation: a proportion in one group describes the outcomes observed in that group; it does not provide a controlled estimate of how outcomes differ from those under another treatment. The design has no randomized comparator, so it cannot directly establish a comparative effect.
The registry lists an enrollment of 16 and a terminated status. It does not report the reason for termination, the number treated, or the number included in the primary analysis. It is therefore not possible to determine from the registry record whether the endpoint was analyzed in all enrolled participants or what follow-up was available for each participant.
The safety counts indicate that adverse events were recorded in the Autologous TIL group, including serious events and deaths. These are descriptive figures and do not establish causation. Overall, the record provides a study framework and limited safety information, but not the outcome results required to characterize response or survival. It cannot support a conclusion about whether MDA-TIL changes clinical outcomes relative to another approach.
13. Why This Trial Matters Statistically
This study raises a statistical question across three cancer types while using a single-group design. That combination makes clear definitions of the population and endpoint particularly important. The primary endpoint specifies confirmed complete or partial responses, a named response framework, an All-Treated denominator, and a follow-up timeframe. These details identify what the study intended to measure, even though the registry record does not report the result.
The record also illustrates why an endpoint definition and an endpoint estimate are different kinds of information. A defined response measure makes the intended calculation understandable; the count of responders, denominator, and uncertainty interval are needed to interpret the observed outcome. Those result details are not reported in the registry record.
Statistically, the absence of a concurrent comparison group matters. A response proportion could describe outcomes among treated participants, but a single-group result would not provide a randomized estimate of how outcomes differ under another treatment. This distinction is central when interpreting early-phase studies and prevents a within-group observation from being mistaken for a controlled comparison.
The listed secondary outcomes further emphasize the need for transparent analysis definitions. Stable disease and survival outcomes answer different questions from ORR, and each requires its own outcome definition and analysis approach. The registry lists these outcomes but does not post their methods or results. The record therefore helps identify the intended areas of measurement while leaving their statistical findings unavailable.
Finally, the termination status and the lack of a stated reason underscore the importance of participant accounting and reporting context. The registry enrollment value is 16, but treatment and analysis counts are not reported. These gaps constrain assessment of both precision and completeness. The statistical significance of this record lies in showing what is defined, what is not reported, and why design and reporting determine how far conclusions can go.
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 most important limitation for interpreting this trial from the registry record is that no primary endpoint result or statistical analysis is posted. The ORR definition and stated confidence interval level do not reveal the observed response count, estimate, or precision. Results for the listed secondary outcomes are also not reported.
The study is single group, has no masking, and has no randomized allocation. There is no concurrent comparator described, so the design does not support a controlled estimate of differences between treatments. The registry record also does not report whether results would be analyzed separately for ovarian, colorectal, and pancreatic cancer, despite including all three in the primary question.
The enrollment value is 16, but the number treated and the size and membership of the All-Treated analysis set are not reported. The record does not provide participant flow, response-assessment completeness, follow-up completeness, or methods for handling missing observations. This prevents assessment of how representative the analyzed participants may be of the enrolled group.
The study status is terminated, but the reason is not reported in the registry record. The record also does not report a detailed statistical analysis plan, sample-size justification, interim analysis plan, or multiplicity and missing-data procedures. These limitations concern what can be learned from the registry record; they do not establish what may have been specified or analyzed elsewhere.
The safety summary is limited to listed counts and a serious-event term with a percentage. It does not give event timing, individual outcomes, or a comparison group. These constraints mean the safety information is descriptive and cannot establish treatment attribution or comparative risk.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Autologous Tumor Infiltrating Lymphocytes MDA-TIL in Treating Patients With Recurrent or Refractory Ovarian Cancer, Colorectal Cancer, or Pancreatic Ductal Adenocarcinoma — NCT03610490.
- Registry record last updated 2025-04-17; status last verified 2025-04.
- Results first posted 2025-04-17.
- 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
NCT03610490 is a phase II, single-group study of autologous tumor-infiltrating lymphocytes in recurrent or refractory ovarian cancer, colorectal cancer, or pancreatic ductal adenocarcinoma. The registry lists M.D. Anderson Cancer Center as sponsor, an enrollment of 16, a start date of 2018-10-16, a primary completion date of 2024-08-21, and a terminated status.
The primary endpoint is ORR according to RECIST v1.1 through 4 years, defined using confirmed complete or partial responses in the All-Treated analysis set, with a 97.5% confidence interval. The record lists stable disease and progression-free and overall survival as secondary outcomes. It reports safety counts for the Autologous TIL group, but does not post efficacy results, confidence interval bounds, statistical analyses, or the reason for termination.