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 Transfer of iNKT Cells for Treating Patients With Relapsed/Advanced HCC (NCT03175679), a non-randomized phase 1 study. The primary outcome is Number of Adverse Events.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03175679 |
| Phase | Phase 1 |
| Condition | Hepatocellular Carcinoma |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 10 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Beijing YouAn Hospital |
| Results posted | Yes |
2. Clinical Question
Population
The study enrolled patients with relapsed or advanced hepatocellular carcinoma, described as BCLC stage C. The registry says the cancer had relapsed or metastasized after standard treatment, or that patients could not receive standard treatment under their current conditions. The enrollment listed in the record is 10.
Intervention
The intervention was autologous, in vitro expanded invariant natural killer T cells, used with IL-2 and lymphodepleting chemotherapy with Tegafur. The registry lists iNKT cell loading doses of 3x10^7/m2, 6x10^7/m2, 9x10^7/m2, and 1x10^10/m2. It describes IL-2 at 25,000 IU/kg/day for 5-14 days. The Tegafur dosing details are not reported in the registry record.
Comparator
There was no randomized control group. The study is recorded as non-randomized and single-group, although it lists groups receiving different iNKT cell loading doses. These dose groups do not constitute a like-for-like control comparison, and the record does not report a concurrent untreated or standard-treatment comparator.
Primary question
The primary endpoint is the number of adverse events. The study description says its purpose included finding the largest iNKT cell dose that was safe and tolerated, assessing how long the cells remained in the body, learning about immune response and side effects, and exploring whether the treatment might help patients. The posted primary endpoint does not itself establish comparative efficacy. Results for the named secondary outcomes are not reported in the registry record.
3. Trial Design
This completed Phase 1 study is registered as non-randomized, unmasked, and single-group. Enrollment is listed as 10. The record gives a start date of 2017-04-01 and a primary completion date of 2018-12-30. Beijing YouAn Hospital is named as the sponsor. The condition is hepatocellular carcinoma.
The study description concerns patients with relapsed or advanced HCC, identified as BCLC stage C, whose cancer relapsed or metastasized following standard treatment or who could not receive standard treatment under current conditions. The intervention combined autologous in vitro expanded iNKT cells with IL-2 and Tegafur. The record lists iNKT cell loading doses of 3x10^7/m2, 6x10^7/m2, 9x10^7/m2, and 1x10^10/m2. IL-2 is described at 25,000 IU/kg/day for 5-14 days. The Tegafur description is incomplete, so its dose and administration details are not reported in the registry record.
The listed dose groups do not change the fundamental design into a randomized comparison. The record does not describe random allocation, masking, or a concurrent control arm. It also does not report how patients were assigned among the dose groups, whether assignment followed a specified escalation rule, or whether the groups were intended for formal between-group testing. Those details are not reported in the registry record.
The registry lists Progression-Free Survival, the number of participants with stabilized or progressive disease, and Overall Survival as secondary outcomes. It does not provide results for those outcomes in the information summarized for this analysis. Consequently, the design and posted endpoint information support a description of the study’s safety focus, but not an estimate of treatment effects relative to another intervention.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Before assignment: A total of 10 patients were recruited in this study from April 2017 to May 2018. Patient 1-8 received cell transfusion in stages according to the plan of 10%，30%，60% of them were used per infusion every other day, and patient 9 and 10 infused all of the cells at one-time.
Overall Study
| Stage | iNKT Cell Loading Dose:3x10^7/m2 | iNKT Cell Loading Dose:6x10^7/m2 | iNKT Cell Loading Dose:9x10^7/m2 | iNKT Cell Loading Dose:1x10^10/m2. |
|---|---|---|---|---|
| STARTED | 3 | 3 | 3 | 1 |
| COMPLETED | 3 | 3 | 3 | 1 |
| NOT COMPLETED | 0 | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: Adverse events defined as the occurrence of toxicity events within 4 weeks after iNKT cells infusion. The severity of adverse events were divided into 5 levels.
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: A total of 10 patients were enrolled in the experiment from April 2017 to May 2018.
| Characteristic | Unit | iNKT Cell Loading Dose:3x10^7/m2 | iNKT Cell Loading Dose:6x10^7/m2 | iNKT Cell Loading Dose:9x10^7/m2 | iNKT Cell Loading Dose:1x10^10/m2 | Total |
|---|---|---|---|---|---|---|
| Participants analysed | 3 | 3 | 3 | 1 | 10 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 3 | 3 | 3 | 1 | 10 |
| Age, Categorical: >=65 years | Participants | 0 | 0 | 0 | 0 | 0 |
| Age, Continuous | years | 50 (50 to 60) | 51 (45 to 59) | 52 (37 to 59) | 53 (53 to 53) | 51.5 (37 to 60) |
| Sex: Female, Male: Female | Participants | 0 | 0 | 1 | 0 | 1 |
| Sex: Female, Male: Male | Participants | 3 | 3 | 2 | 1 | 9 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 3 | 3 | 3 | 1 | 10 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 |
| BCLC (Barcelona Clinic Liver Cancer ) stage: BCLC stage B | Participants | 0 | 0 | 1 | 1 | 2 |
| BCLC (Barcelona Clinic Liver Cancer ) stage: BCLC stage C | Participants | 3 | 3 | 2 | 0 | 8 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Adverse Events, measured over During the first 12 weeks, participants were assessed for adverse events every 2-4 weeks after infusion; after the first 12 weeks, participants were assessed for adverse events every 3 months, up to 20 months..
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | events |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is titled Number of Adverse Events and uses events as its unit. The registry defines adverse events as signs or symptoms, laboratory toxicities, and clinical events considered possibly, likely, or definitely related to study treatment. It says severity was assessed according to NCI-CTCAE v4.0 criteria 2. The study description therefore treats the endpoint as a way to record treatment-related toxicity and clinical events, rather than as a direct measure of tumor response.
Safety-event monitoring is used in early-phase research to characterize what happens during and after exposure to an investigational intervention. Recording event types and severity can help describe the tolerability experience in the enrolled participants. A count of events is not necessarily the same as a count of participants: a participant may experience more than one event, and a single event may have more than one clinical feature. This distinction is general and matters when interpreting an endpoint whose unit is events alongside registry figures described in terms of participants affected.
The registry gives an assessment schedule of every 2-4 weeks after infusion during the first 12 weeks, then every 3 months up to 20 months. It separately describes the adverse-event population using occurrence of toxicity events within 4 weeks after iNKT cell infusion. These descriptions do not specify how the endpoint count reconciles the shorter event definition with the longer assessment schedule. That relationship is not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted safety information is organized by iNKT cell loading dose. For the groups titled 3x10^7/m2 and 6x10^7/m2, the registry reports 1 serious adverse event affected count among 3 at risk in each group. For 9x10^7/m2, it reports 2 affected among 3 at risk. For 1x10^10/m2, it reports 1 affected among 1 at risk. The other-adverse-event figures are 3 affected among 3 at risk for each of the first three listed dose groups, and 1 affected among 1 at risk for the 1x10^10/m2 group. The record also lists deaths of 1, 1, 2, and 1 across those groups, respectively.
These entries describe the posted group-level counts; they do not establish that the treatment caused each event or death. The adverse-event definition includes events considered possibly, likely, or definitely related to study treatment, but the posted group counts do not provide individual event narratives or a separate causal assessment for every outcome. The record’s serious-event terms include spontaneous rupture of the tumor at 100.0%, cirrhosis and upper gastrointestinal bleeding at 66.7%, and refractory bile duct infection and toxic megacolon at 33.3%. The denominator used for those percentages is not reported in the registry record, so they should not be interpreted as rates among all enrolled participants.
The group counts are descriptive. Because allocation was non-randomized and there was no like-for-like control comparison, differences between dose groups cannot by themselves identify a dose effect. The registry does not report how participants were assigned to dose groups, and the group counts are limited. In general, estimates from small groups can be imprecise and sensitive to individual observations; that is a statistical caution, not a newly calculated result.
The primary endpoint is a number of adverse events, but the posted information includes figures for affected participants as well as event terminology. The registry does not provide a complete event-level dataset or a statistical analysis that links these summaries. It reports no analyses in the record. Confidence intervals, hypothesis tests, adjusted estimates, and a prespecified comparison for the primary endpoint are not reported in the registry record.
The registry names Progression-Free Survival, stabilized or progressive disease, and Overall Survival as secondary outcomes, but does not report their results in the record. The safety counts therefore should not be treated as evidence about tumor response or survival. Nor do these counts provide a comparative estimate against standard care or another treatment. They describe recorded adverse-event and death figures in the listed study groups, with substantial uncertainty about the event-level detail and the comparison those groups can support.
9. Statistical Methods Explained
There is no reported inferential analysis for the primary endpoint in the registry record. The record identifies a count outcome, but does not state a statistical model, a method for comparing dose groups, a confidence interval procedure, or a hypothesis test. It is therefore possible to describe the posted counts, but not to attribute an unreported adjusted or model-based estimate to the study.
What does a count endpoint represent?
In general, count data record how many events occur over a defined observation period. Counts can be zero or positive, and the distribution of counts may be uneven: many participants may have few events while some have more. A simple count does not show how long each person was observed, whether events recurred, or how severity varied unless those details are reported separately. For this trial, the endpoint is labeled in events, while the posted adverse-event summaries also contain at-risk and affected counts. Those are different forms of information and should not be treated as interchangeable.
In general, count outcomes are sometimes analyzed with models that allow for counts and differences in observation time. If the variation in the data exceeds what a basic count model expects, over-dispersion can matter: a model that assumes too little variability may produce confidence intervals that are too narrow and tests that appear too conclusive. This is general statistical context only. The registry does not report an over-dispersion assessment or a count model for this endpoint.
Why is a direct dose-group comparison difficult?
General statistical interpretation of group contrasts depends on how groups were formed and whether their participants are reasonably comparable. Random allocation can help balance measured and unmeasured characteristics on average, whereas non-randomized assignment may leave differences between groups that affect outcomes. This study is recorded as non-randomized and single-group, and no control comparison is reported. Although the registry lists different loading-dose groups, the method used to allocate patients to them is not reported in the registry record. A crude difference in counts between groups would not, by itself, distinguish a dose relationship from differences in participant characteristics, observation, or other aspects of care.
The record does not state a statistical plan for comparing dose levels or identify a primary contrast among them. It also does not report whether event counts were analyzed per participant, per treatment exposure, or with another unit of analysis. Without that information, a reader should not infer a formal comparison from the descriptive figures.
What do uncertainty estimates normally add?
In general, a confidence interval expresses the range of estimates compatible with the observed data under a specified statistical method and its assumptions. It helps distinguish a precise estimate from one that is highly uncertain. A p-value, when used, describes how compatible data are with a specified null hypothesis under a model; it does not measure the probability that a treatment is effective or establish causality. Neither an interval nor a test can repair a non-comparative design or missing outcome information. Confidence intervals and p-values for the primary endpoint are not reported in the registry record.
The sample-size illustrations associated with the posted material are planning calculations, not observed power. Their stated assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the target effect is a stated fraction of the observed effect. The record describes the calculations as planning illustrations rather than the trial’s own design. For time-to-event outcomes, the stated method reports total events; for other outcomes, it reports participants per arm. These assumptions do not establish that the trial used those methods or that it was designed around a particular comparison. The registry does not report a sample-size justification for the trial itself.
There is an additional interpretive issue when a planning calculation depends on an observed effect: the target is tied to the estimate used in the calculation. In general, an estimate from limited data may be unstable, and a planning illustration based on it should not be mistaken for a guarantee of future precision. No observed effect or inferential analysis for the primary endpoint is reported in the registry record, so the illustrations cannot be read as evidence that this study had adequate power for a safety or efficacy question.
Finally, the endpoint descriptions give different time scopes: the assessment schedule extends up to 20 months, while the adverse-event population definition refers to events within 4 weeks after infusion. The registry does not explain whether the primary count uses the shorter window, the full follow-up period, or a particular reconciliation of the definitions. In general, an analysis needs a clear outcome definition and observation window for its estimates to be interpretable. That detail is not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry names a primary adverse-event endpoint and lists Progression-Free Survival, stabilized or progressive disease, and Overall Survival as secondary outcomes. It does not describe a multiplicity strategy for interpreting several endpoints, dose groups, event categories, or repeated assessments. It is not reported in the registry record whether any outcome was designated for formal hypothesis testing or whether adjustments to significance levels were planned.
The registry record lists no analyses. Statistical methods for handling incomplete follow-up, missing outcome observations, withdrawal, or censoring are not reported in the registry record. The record does not state how missing safety assessments would be handled, whether participants with incomplete records were included in each analysis, or whether any sensitivity analyses were planned.
Analysis populations are also not described. The registry does not report whether the safety summaries include everyone who received any study intervention, everyone enrolled, or another defined population. It does not report how exposure to the different components of treatment was handled in the analysis. These omissions limit interpretation of the at-risk and affected counts because a reader cannot establish the exact analysis denominator and inclusion rules from the registry record.
In general, multiplicity matters when several outcomes or comparisons are examined because repeated testing can increase the chance of apparently notable findings. Missing-data methods matter because conclusions can depend on why observations are absent and how the analysis handles them. These are general statistical concerns; the registry record does not report a plan addressing them for this study.
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 |
|---|---|---|---|
| iNKT Cell Loading Dose:3x10^7/m2 | 1 | 3 | 33.3% |
| iNKT Cell Loading Dose:6x10^7/m2 | 1 | 3 | 33.3% |
| iNKT Cell Loading Dose:9x10^7/m2 | 2 | 3 | 66.7% |
| iNKT Cell Loading Dose:1x10^10/m2 | 1 | 1 | 100.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| iNKT Cell Loading Dose:3x10^7/m2 | 1 of 3 |
| iNKT Cell Loading Dose:6x10^7/m2 | 1 of 3 |
| iNKT Cell Loading Dose:9x10^7/m2 | 2 of 3 |
| iNKT Cell Loading Dose:1x10^10/m2 | 1 of 1 |
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 |
|---|---|
| iNKT Cell Loading Dose:3x10^7/m2 | 3 of 3 |
| iNKT Cell Loading Dose:6x10^7/m2 | 3 of 3 |
| iNKT Cell Loading Dose:9x10^7/m2 | 3 of 3 |
| iNKT Cell Loading Dose:1x10^10/m2 | 1 of 1 |
Most frequent serious adverse event terms
| Event term | Organ system | iNKT Cell Loading Dose:3x10^7/m2 | iNKT Cell Loading Dose:6x10^7/m2 | iNKT Cell Loading Dose:9x10^7/m2 | iNKT Cell Loading Dose:1x10^10/m2 |
|---|---|---|---|---|---|
| Cirrhosis and upper gastrointestinal bleeding | Gastrointestinal disorders | 0 of 3 | 1 of 3 | 2 of 3 | 0 of 1 |
| Refractory bile duct infection and toxic megacolon | Gastrointestinal disorders | 1 of 3 | 0 of 3 | 0 of 3 | 0 of 1 |
| Spontaneous rupture of the tumor | Gastrointestinal disorders | 0 of 3 | 0 of 3 | 0 of 3 | 1 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | iNKT Cell Loading Dose:3x10^7/m2 | iNKT Cell Loading Dose:6x10^7/m2 | iNKT Cell Loading Dose:9x10^7/m2 | iNKT Cell Loading Dose:1x10^10/m2 |
|---|---|---|---|---|---|
| Increased blood bilirubin level | Hepatobiliary disorders | 3 of 3 | 1 of 3 | 3 of 3 | 0 of 1 |
| Decreased platelet count | Blood and lymphatic system disorders | 1 of 3 | 1 of 3 | 1 of 3 | 1 of 1 |
| Increased aspartate transaminase level | Hepatobiliary disorders | 1 of 3 | 0 of 3 | 2 of 3 | 1 of 1 |
| Fever | Immune system disorders | 3 of 3 | 0 of 3 | 0 of 3 | 0 of 1 |
| Decreased white blood cell count | Blood and lymphatic system disorders | 1 of 3 | 0 of 3 | 1 of 3 | 1 of 1 |
| Chills | Immune system disorders | 2 of 3 | 0 of 3 | 0 of 3 | 0 of 1 |
| Fatigue | General disorders | 1 of 3 | 0 of 3 | 1 of 3 | 0 of 1 |
| Anemia | Blood and lymphatic system disorders | 1 of 3 | 0 of 3 | 1 of 3 | 0 of 1 |
| Increased alanine transaminase level | Hepatobiliary disorders | 0 of 3 | 0 of 3 | 1 of 3 | 1 of 1 |
| Pain | Gastrointestinal disorders | 1 of 3 | 0 of 3 | 0 of 3 | 0 of 1 |
Serious events with the highest arm-level rate: spontaneous rupture of the tumor (100.0%), cirrhosis and upper gastrointestinal bleeding (66.7%), refractory bile duct infection and toxic megacolon (33.3%).
The safety summaries report serious adverse-event and other-adverse-event figures by listed iNKT cell loading dose. For the 3x10^7/m2 group, the serious-event figures are 1 affected and 3 at risk; for 6x10^7/m2, they are 1 and 3; for 9x10^7/m2, they are 2 and 3; and for 1x10^10/m2, they are 1 and 1. The corresponding other-event figures are 3 affected and 3 at risk, 3 and 3, 3 and 3, and 1 and 1. Deaths are listed as 1, 1, 2, and 1 in the same group order.
These are registry counts, not comparative risk estimates. The record does not provide a control group, event-level details, or a separate causal assessment for the deaths. The serious-event terms are listed with percentages: spontaneous rupture of the tumor, 100.0%; cirrhosis and upper gastrointestinal bleeding, 66.7%; and refractory bile duct infection and toxic megacolon, 33.3%. Their percentage denominator is not reported in the registry record.
The endpoint is labeled as a number of events, whereas these summaries distinguish those at risk from those affected. A participant count and an event count answer different questions, particularly if events can recur. The record also gives an assessment schedule through 20 months but defines the adverse-event population around toxicity within 4 weeks after infusion. The precise relationship between those time descriptions is not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The registry record documents a Phase 1 study intended to examine iNKT cell treatment in relapsed or advanced HCC, with a stated focus on dose safety and tolerability as well as immune response, persistence, side effects, and possible benefit. The posted safety information gives adverse-event and death counts for the listed dose groups. It also identifies several serious-event terms and their percentages. These descriptions are useful for understanding what the record reports, but they are not a complete account of each participant’s clinical course.
The record does not establish that the intervention caused every listed adverse event or death. The adverse-event definition includes events assessed as possibly, likely, or definitely related to study treatment; the group summaries do not show the individual judgments or details underlying each entry. The figures also cannot show whether an event was more or less common than it would have been without treatment, because no concurrent control group is reported.
The record does not report results for the named secondary outcomes of Progression-Free Survival, stabilized or progressive disease, or Overall Survival. The posted safety figures therefore do not answer whether tumor disease was controlled or whether survival differed under treatment. No comparative treatment effect, adjusted estimate, or inferential analysis is reported for the primary endpoint.
In general, findings from a non-randomized study can describe the observed experience of its participants, while conclusions about what would have happened under another treatment require a defensible comparison. The listed dose groups do not provide a randomized, like-for-like control comparison. The registry also does not report how assignment to dose groups occurred. For these reasons, the group counts should be read as descriptive safety information, not as proof of a dose-response relationship or a treatment effect.
The record’s limitations in endpoint timing, analysis methods, missing-data handling, and analysis populations further constrain interpretation. The strongest supported reading is that the registry records adverse-event and death figures in the listed groups, while leaving the comparative meaning, uncertainty, and full outcome profile unreported.
13. Why This Trial Matters Statistically
This study is statistically notable because its central endpoint is safety-focused and its design is non-randomized and single-group. That combination makes descriptive reporting especially important: readers need to know what was counted, over what period, and in which analysis population. The registry identifies the endpoint as a number of adverse events and provides group-level affected and at-risk figures, but it does not report an analysis method that connects those summaries to a formal estimate or comparison.
The record also illustrates why a list of dose groups is not equivalent to a controlled dose comparison. Without random allocation or a reported assignment method, apparent differences between groups could reflect factors other than dose. The posted counts should therefore be interpreted as descriptions of the recorded experience, not as a causal estimate.
There is also a material distinction between event counts and participant counts. An event endpoint can capture repeated experiences, while an affected-participant summary indicates whether people experienced events. Clear definitions and observation windows are needed to understand how these quantities relate. Here, the registry gives both a longer adverse-event assessment schedule and a population definition referring to toxicity within 4 weeks after infusion, without explaining how the two scopes relate.
Finally, the record names clinically relevant secondary outcomes but does not report their results. This limits the statistical account to the available safety summaries and prevents a fuller assessment of disease outcomes. These features show why transparent reporting of endpoint definitions, analysis populations, uncertainty, and comparator structure is central to interpreting early-phase clinical research.
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 registry describes a primary adverse-event assessment schedule during the first 12 weeks and then every 3 months up to 20 months, while its adverse-event population definition refers to toxicity within 4 weeks after iNKT cell infusion. The record does not clarify which time window governs the primary event count or how later assessments contribute. This leaves the duration represented by the endpoint uncertain.
The intervention descriptions list iNKT cells with IL-2 and Tegafur, but the Tegafur administration details are incomplete. The record does not report the method used to assign participants to loading-dose groups. It also does not explain how the listed deaths relate to treatment exposure, event timing, or the secondary Overall Survival outcome.
The serious-event terms have percentages, but their denominator is not reported in the registry record. The registry gives aggregate counts rather than individual event histories, so event recurrence, timing, duration, and severity by group cannot be assessed from these summaries. The endpoint’s event unit and the affected-participant figures should not be assumed to represent the same quantity.
Statistical methods, uncertainty estimates, analysis populations, and handling of missing observations are not reported in the registry record. The secondary outcome results are also not reported. These gaps, together with the absence of a randomized control group, prevent the safety figures from supporting a comparative estimate or a broader conclusion about tumor response or survival.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Adoptive Transfer of iNKT Cells for Treating Patients With Relapsed/Advanced HCC — NCT03175679.
- Registry record last updated 2024-09-19; status last verified 2024-09.
- Results first posted 2024-09-19.
- 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 completed Phase 1 study sponsored by Beijing YouAn Hospital, with enrollment listed as 10. It began on 2017-04-01 and has a primary completion date of 2018-12-30. The design is non-randomized, unmasked, and single-group.
The study concerns relapsed or advanced hepatocellular carcinoma and evaluates autologous expanded iNKT cells with IL-2 and Tegafur across listed iNKT cell loading-dose groups. The primary endpoint is Number of Adverse Events. The registry also names Progression-Free Survival, stabilized or progressive disease, and Overall Survival as secondary outcomes.
Posted safety information includes serious and other adverse-event figures, deaths, and serious-event terms. Statistical analyses and results for the secondary outcomes are not reported in the registry record. The counts are descriptive and do not provide a randomized comparison.