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 Umbilical & Cord Blood (CB) Derived CAR-Engineered NK Cells for B Lymphoid Malignancies (NCT03056339), a na phase 1, phase 2 study. The primary outcome is Number of Events That Was Grade 3-4 Toxicities.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03056339 |
| Phase | Phase 1, Phase 2 |
| Condition | B-Lymphoid Malignancies, Acute Lymphocytic Leukemia, Chronic Lymphocytic Leukemia |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 49 |
| Arms in the primary outcome | n/a |
| Lead sponsor | M.D. Anderson Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
The study concerns patients with relapsed or refractory B-lymphoid malignancies, including acute lymphocytic leukemia, chronic lymphocytic leukemia, and non-Hodgkin lymphoma. The brief summary describes patients with relapsed or refractory B-cell lymphoma or leukemia who are stem cell transplant patients. The registry lists an enrollment of 49, while the brief summary says that up to 36 patients would take part. The record does not explain the difference between these figures.
Intervention
Participants received lymphodepleting chemotherapy with fludarabine and cyclophosphamide, with mesna before and after cyclophosphamide, followed by genetically modified umbilical cord blood-derived CAR-NK cells. The intervention list also includes AP1903. The arm description is truncated, so the record excerpt does not fully describe when or under what circumstances AP1903 was used.
Comparator
The record identifies a single-group model and does not describe a randomized comparator. The adverse-event results are organized by dose group, but these cohorts are not a like-for-like control comparison. Consequently, differences across the dose groups cannot be read as randomized treatment effects.
Primary question
The primary endpoint is the number of grade 3–4 toxicity events within 40 days after NK-cell infusion. This endpoint focuses on serious toxicity during a defined period after treatment. The registry record gives the endpoint definition but does not report its result. The listed secondary outcomes concern overall response rate and the number of participants achieving an objective response; their results are not included in the record details.
3. Trial Design
This is a Phase I/II, single-group, dose-escalation study of umbilical cord blood-derived CAR-engineered NK cells given in conjunction with lymphodepleting chemotherapy. Its stated goals include studying safety, assessing whether the disease improves, and identifying the highest tolerable CAR-NK-cell dose. The registry marks allocation as not applicable and masking as none. It therefore does not describe a randomized, masked comparison against a control group.
The listed intervention includes fludarabine, cyclophosphamide, mesna, iC9/CAR.19/IL15-transduced cord blood NK cells, and AP1903. The available arm description says fludarabine and cyclophosphamide are given on Days -5, -4, and -3; mesna is given before and after cyclophosphamide; and the NK cells are infused on Day 0. The rest of the arm description is cut off, so details beyond those statements are not reported in the registry record.
Adverse-event data are grouped by four listed dose cohorts: 1x105 NK Cells /kg, 1x106 NK Cells /kg, 1x107 NK Cells /kg, and an 8x108 flat NK cell dose. These groups provide descriptive information by dose. They are not a randomized control comparison, and any differences between their reported event counts could reflect factors other than dose. The record does not report the allocation process to these groups or a detailed statistical comparison among them.
The study status is completed. The registry lists an enrollment of 49, whereas the brief summary says that up to 36 patients would take part and that all would be enrolled at MD Anderson. The record does not reconcile those enrollment figures. It lists the start as 2017-06-21 and primary completion as 2023-03-06. Dates and completion status describe the study record, not the magnitude or certainty of its clinical findings.
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: Patient accession #3 and #21 are the same patient. The patient was taken off protocol and re-consented for a second infusion. Patient was only evaluated in regards to safety and efficacy as 1 subject.
Overall Study
| Stage | Group 1: 1x105 NK Cells /kg | Group 2: 1x106 NK Cells /kg | Group 3: 1x107 NK Cells /kg | Group 4: 8x108 Flat NK Cell Dose |
|---|---|---|---|---|
| STARTED | 3 | 6 | 23 | 17 |
| COMPLETED | 3 | 4 | 16 | 16 |
| NOT COMPLETED | 0 | 2 | 7 | 1 |
Reasons for not completing this period:
| Reason | Group 1: 1x105 NK Cells /kg | Group 2: 1x106 NK Cells /kg | Group 3: 1x107 NK Cells /kg | Group 4: 8x108 Flat NK Cell Dose |
|---|---|---|---|---|
| Failed Requirements | 0 | 2 | 7 | 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 | Group 1: 1x105 NK Cells /kg | Group 2: 1x106 NK Cells /kg | Group 3: 1x107 NK Cells /kg | Group 4: 8x108 Flat NK Cell Dose | Total |
|---|---|---|---|---|---|---|
| Participants analysed | 3 | 6 | 23 | 17 | 49 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 3 | 5 | 11 | 9 | 28 |
| Age, Categorical: >=65 years | Participants | 0 | 1 | 12 | 8 | 21 |
| Sex: Female, Male: Female | Participants | 1 | 3 | 8 | 6 | 18 |
| Sex: Female, Male: Male | Participants | 2 | 3 | 15 | 11 | 31 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 0 | 0 | 0 | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 2 | 6 | 21 | 16 | 45 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 1 | 0 | 2 | 1 | 4 |
| Region of Enrollment: United States | participants | 3 | 6 | 23 | 17 | 49 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Events That Was Grade 3-4 Toxicities, measured over 40 days.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | events |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint counts grade 3–4 toxicity events within 40 days of NK-cell infusion. In plain terms, it is a count of events meeting the registry’s specified severity grades during a defined post-infusion window. It is not, by definition, a count of all adverse events, a measure of disease response, or a summary of toxicity over an unspecified period. The unit is events, and the registry identifies the endpoint parameter as a number.
A defined safety endpoint is useful in an early-phase dose-escalation study because it focuses attention on substantial toxicities during a specified period after treatment. Such information can contribute to assessment of tolerability as doses are explored. The registry record does not explain a dose-limiting-toxicity rule, a threshold for selecting a dose, or how multiple events in the same participant were handled. Those details are not reported in the registry record.
Most importantly, the endpoint definition is not an endpoint result. The record details identify the measure and its 40-day timeframe, but do not give the number of grade 3–4 toxicity events observed. The serious and other adverse-event summaries are separate descriptive entries and cannot be substituted for that primary endpoint: they do not establish that their events meet the endpoint’s grade and timeframe definition. Without the reported primary count and its analysis context, the primary safety outcome cannot be quantified from this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a completed Phase I/II study with dose cohorts and a primary safety endpoint, but the record does not include the primary endpoint result. It defines the outcome as the number of grade 3–4 toxicity events within 40 days of NK-cell infusion. No count of those events, uncertainty interval, or formal test for that endpoint is reported in the registry record. Therefore, the posted details do not permit a numerical interpretation of the primary outcome.
The adverse-event entries give descriptive counts by dose group. In the 1x105 NK Cells /kg group, the record lists 1 participant affected by serious adverse events among 3 at risk and 2 affected by other adverse events among 3 at risk. In the 1x106 NK Cells /kg group, it lists 0 affected by serious events among 6 at risk and 6 affected by other events among 6 at risk. In the 1x107 NK Cells /kg group, it lists 0 affected by serious events among 23 at risk and 12 affected by other events among 23 at risk. In the 8x108 Flat NK Cell Dose group, it lists 0 affected by serious events among 17 at risk and 10 affected by other events among 17 at risk. Deaths are listed as 0 in each group.
These are cohort-level descriptions, not estimates from a randomized comparison. The registry explicitly identifies allocation as not applicable and the model as single group. Differences in counts between cohorts should not be treated as evidence that a dose caused more or fewer events: group sizes differ, and the record does not report an adjusted analysis or a statistical comparison. The counts also do not give event timing, severity details for the “other” category, or individual-level overlap between categories.
The record lists overall response rate and the number of participants achieving an objective response as secondary outcomes, but the associated results are not included in the information. The brief summary states that researchers aimed to learn whether treatment would improve disease, but that stated aim is not evidence of a response. Nor does completed status establish an efficacy or safety conclusion. The available information supports a narrow reading: the study record specifies the design and endpoint, and includes adverse-event counts by dose group, while leaving the primary endpoint result and the secondary outcome results unreported.
The record also contains an enrollment inconsistency: its enrollment field lists 49, while the brief summary says up to 36 patients would take part. No explanation is reported. This limits interpretation of how the planned enrollment relates to the posted safety denominators, and it is not appropriate to infer from the record that every enrolled participant contributed to every outcome.
9. Statistical Methods Explained
The registry identifies the trial as a Phase I/II, single-group dose-escalation study, with allocation marked not applicable and masking listed as none. These design features matter when interpreting the numerical results. There is no randomized control group in the record against which to estimate a comparative treatment effect. The dose cohorts are descriptive groups within the study, not interchangeable treatment and control arms.
What does a single-group design show?
General: A single-group study can describe what occurred among participants receiving the study regimen. It can provide information about observed safety events and, when reported, disease outcomes. Without a concurrent randomized comparator, however, it cannot separate the effect of the intervention from other explanations, such as differences in participant characteristics, disease course, or other care. The registry record does not report an adjusted comparative analysis that would address such differences.
The intervention combines lymphodepleting chemotherapy and CAR-NK cells, and the listed interventions include AP1903. That means a result for the overall regimen would not by itself identify the contribution of each component. The record does not report a design intended to isolate the separate effects of chemotherapy, the cell infusion, or AP1903.
What is a count endpoint?
The primary endpoint is specified as a number of grade 3–4 toxicity events within 40 days after infusion. General: A count endpoint records how many qualifying events occurred, rather than measuring a continuous quantity such as a laboratory value. Counts can be interpreted differently depending on whether the analysis counts events, participants with at least one event, or event rates over exposure time. The registry specifies events as the unit, but does not report whether repeat events in a participant were counted separately, how follow-up time was handled, or the statistical model used for the primary endpoint.
The separate adverse-event summary reports participants affected and participants at risk within each group. Those summaries therefore should not be assumed to reproduce the primary endpoint’s event count. They also do not establish that the reported events meet the primary endpoint’s severity-grade and timeframe criteria.
Why can counts be difficult to compare?
General: Count data may vary more than a simple statistical model expects, a feature often called over-dispersion. If over-dispersion is present, a model that assumes less variability can produce uncertainty estimates that are too narrow. Methods for count outcomes may account for this extra variability, but the choice depends on the data structure and analysis plan. The model and dispersion assumptions for the primary endpoint are not reported in the registry record, and the record gives no primary count result with which to assess them.
Counts can also be influenced by how long participants are observed and how events are defined. Here, the primary endpoint specifies a 40-day window, but no details are reported about incomplete observation, withdrawals, or the handling of events occurring outside that window. The adverse-event entries provide at-risk denominators, but do not state whether each participant had the same duration of observation for those summaries.
How should the dose groups be read?
The registry reports adverse events separately for four dose cohorts. General: In a dose-escalation study, cohorts can help describe safety across planned dose levels, but a numerical pattern across cohorts is not automatically a dose-response relationship. Cohort assignment may be related to study stage or eligibility, and group sizes may differ. Causal claims require design and analysis assumptions beyond a visual or numerical comparison of counts. The record does not report the rules for escalation, stopping, or selecting a dose, and it does not provide inferential comparisons among the cohorts.
The adverse-event summaries do not provide confidence intervals. General: A confidence interval communicates the statistical uncertainty around an estimated quantity under the assumptions of the analysis. It is not a guarantee that the true value lies within the interval, and its width depends in part on the information available. Because the record details do not include an estimate and interval for the primary endpoint, its precision cannot be evaluated from these details.
What do the planning assumptions mean?
The stated sample-size planning illustration uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; it describes the target effect as a stated fraction of the observed effect. The record characterizes such sample-size values as planning illustrations computed from the observed effect, not as the trial’s own design. It further distinguishes participants per arm for non-time-to-event outcomes from total events for time-to-event outcomes. These assumptions do not constitute observed power, and the record does not report a randomized two-arm design for this trial. The actual sample-size illustration results are not reported in the registry record details.
General: A planning calculation asks what study size might be needed under specified assumptions to detect a target effect with a chosen error rate. It does not show that a completed study had that probability of detecting an effect, and it does not alter the uncertainty in observed results. When the underlying effect estimate, target fraction, and resulting sample-size values are not available in the record, the calculation cannot be independently interpreted from those details.
The registry lists zero statistical analyses. That does not establish what analyses may have been conducted outside the registry; it means that statistical analysis results and methods are not listed in the registry record. The protocol’s statistical plan, sample-size justification, interim analyses, and analysis populations are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The record identifies a primary toxicity outcome and lists two secondary outcomes: overall response rate of participants analyzed and the number of participants achieving an objective response. When a study examines multiple outcomes, multiplicity can arise because repeated statistical tests increase the chance of at least one apparently notable result even if no underlying effect is present. General: Researchers may address this through a prespecified hierarchy, adjusted significance thresholds, or other methods, but the appropriate approach depends on the protocol and analysis plan.
A multiplicity procedure is not reported in the registry record. The record also lists zero statistical analyses, so it does not provide test results or an adjustment method for interpreting multiple outcomes. No p-values or confidence intervals for these outcomes are reported in the information.
Missing-data rules and analysis populations are also not reported in the registry record. The enrollment field lists 49, while the brief summary says up to 36 patients would take part; the record does not explain the discrepancy or say how the enrollment figure relates to the population analyzed for each outcome. The adverse-event summary supplies at-risk denominators by dose group, but does not explain exclusions, incomplete follow-up, or the treatment of missing event information. The record does not state whether analyses used all enrolled participants, participants who received the cell infusion, or another defined population.
These omissions matter because the interpretation of an outcome depends not only on its definition but also on who was included, how incomplete observations were handled, and whether outcome testing followed a prespecified plan. No interim-analysis plan or statistical analysis plan is reported in the registry record. Their absence from the record does not establish that such plans did not exist; their details 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 |
|---|---|---|---|
| Group 1: 1x105 NK Cells /kg | 1 | 3 | 33.3% |
| Group 2: 1x106 NK Cells /kg | 0 | 6 | 0.0% |
| Group 3: 1x107 NK Cells /kg | 0 | 23 | 0.0% |
| Group 4: 8x108 Flat NK Cell Dose | 0 | 17 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Group 1: 1x105 NK Cells /kg | 0 of 3 |
| Group 2: 1x106 NK Cells /kg | 0 of 6 |
| Group 3: 1x107 NK Cells /kg | 0 of 23 |
| Group 4: 8x108 Flat NK Cell Dose | 0 of 17 |
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 |
|---|---|
| Group 1: 1x105 NK Cells /kg | 2 of 3 |
| Group 2: 1x106 NK Cells /kg | 6 of 6 |
| Group 3: 1x107 NK Cells /kg | 12 of 23 |
| Group 4: 8x108 Flat NK Cell Dose | 10 of 17 |
Most frequent serious adverse event terms
| Event term | Organ system | Group 1: 1x105 NK Cells /kg | Group 2: 1x106 NK Cells /kg | Group 3: 1x107 NK Cells /kg | Group 4: 8x108 Flat NK Cell Dose |
|---|---|---|---|---|---|
| General disorders and administration site conditions | General disorders | 1 of 3 | 0 of 6 | 0 of 23 | 0 of 17 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Group 1: 1x105 NK Cells /kg | Group 2: 1x106 NK Cells /kg | Group 3: 1x107 NK Cells /kg | Group 4: 8x108 Flat NK Cell Dose |
|---|---|---|---|---|---|
| Fatigue | Cardiac disorders | 2 of 3 | 0 of 6 | 10 of 23 | 10 of 17 |
| Nausea | Gastrointestinal disorders | 1 of 3 | 2 of 6 | 12 of 23 | 7 of 17 |
| Investigations - (Other) | Investigations | 2 of 3 | 6 of 6 | 3 of 23 | 1 of 17 |
| Fever | General disorders | 1 of 3 | 0 of 6 | 8 of 23 | 1 of 17 |
| Infections and infestations | Infections and infestations | 2 of 3 | 3 of 6 | 4 of 23 | 1 of 17 |
| Respiratory, thoracic and mediastinal disorders | Respiratory, thoracic and mediastinal disorders | 0 of 3 | 0 of 6 | 10 of 23 | 0 of 17 |
| Constipation | Gastrointestinal disorders | 0 of 3 | 0 of 6 | 5 of 23 | 4 of 17 |
| Diarrhea | Gastrointestinal disorders | 0 of 3 | 0 of 6 | 2 of 23 | 6 of 17 |
| Cardiac disorders - (Other), specify | Cardiac disorders | 1 of 3 | 1 of 6 | 4 of 23 | 1 of 17 |
| Insomnia | Psychiatric disorders | 0 of 3 | 0 of 6 | 5 of 23 | 2 of 17 |
Serious events with the highest arm-level rate: general disorders and administration site conditions (33.3%).
The serious adverse-event summary is organized by dose cohort and gives the number at risk and the number affected. The 1x105 NK Cells /kg group lists 1 affected among 3 at risk. The 1x106 NK Cells /kg, 1x107 NK Cells /kg, and 8x108 Flat NK Cell Dose groups each list 0 affected; their at-risk counts are 6, 23, and 17, respectively. The registry lists 0 deaths in every group.
For other adverse events, the group entries list 2 affected among 3 at risk, 6 among 6, 12 among 23, and 10 among 17, in the same dose-group order. The record also names “General disorders and administration site conditions” as a top serious-event term and gives 33.3%. The context and denominator for that percentage are not reported in the registry record details, so it should not be used to infer a cohort-specific rate.
These entries are descriptive summaries. They are not the primary endpoint result, which counts grade 3–4 toxicity events within 40 days of infusion. The record does not show whether the serious or other adverse-event categories map to that endpoint’s definition or timeframe. Nor do the group counts constitute a randomized comparison. They should be read as reported safety counts, not as proof of a dose effect or as a complete account of each participant’s clinical course.
12. What the Result Does, and Does Not, Mean
The registry record establishes the study’s stated purpose, design, intervention, endpoint definition, and selected safety summaries. It describes a Phase I/II dose-escalation study of cord blood-derived CAR-engineered NK cells given with lymphodepleting chemotherapy to patients with relapsed or refractory B-lymphoid malignancies. The primary safety measure is the number of grade 3–4 toxicity events during the 40 days after NK-cell infusion.
The record does not provide the result for that primary endpoint. It also does not provide the secondary response results listed in the registry. As a result, the available record details cannot quantify the study’s primary toxicity outcome or establish a disease-response finding. The study’s completion status and stated research objectives do not substitute for reported outcome data.
The posted adverse-event summaries describe participants affected by serious and other events in each dose cohort, along with deaths listed as zero in each group. These data offer descriptive safety information, but they do not supply a randomized comparison, an adjusted estimate, or uncertainty intervals. A difference in counts between dose groups should not be interpreted as a causal effect of dose. The categories also cannot be assumed to match the primary endpoint’s grade and time-window criteria.
The record does not report the analysis populations, missing-data approach, statistical plan, or multiplicity procedure. It also gives inconsistent enrollment information: the enrollment field lists 49 and the brief summary says up to 36, without an explanation. These limitations prevent a complete reconstruction of how participants were included in the reported summaries.
Accordingly, the record supports description of a completed early-phase study and its listed safety counts, but not a numerical conclusion about the primary endpoint, a formal estimate of treatment effect, or a comparative claim about safety or response. Those conclusions would require reported outcomes and analysis details that are not reported in the registry record.
13. Why This Trial Matters Statistically
This study illustrates a common statistical challenge in early-phase clinical research: the central questions concern safety and dose exploration, while the design does not include a randomized control group. Descriptive information across dose cohorts can be relevant to understanding what was recorded during the study, but it does not have the same interpretation as a controlled treatment comparison. This distinction is especially important when cohort sizes differ and the record does not report inferential comparisons.
The primary endpoint is an event count within a defined follow-up window. That definition highlights why outcome labels and analysis details matter. A count of qualifying toxicity events is not interchangeable with the number of participants affected by any serious adverse event, and neither is automatically equivalent to the number of participants with other adverse events. Clear reporting of event definitions, repeat-event handling, follow-up, and uncertainty would help readers understand how the endpoint was measured.
The record also demonstrates the value of reporting complete outcome results. It identifies the primary safety question and secondary response outcomes, but the results for those endpoints are not included in the record details. Without those results, readers cannot assess the magnitude or precision of the primary outcome or the secondary outcomes. A completed study status alone does not fill that gap.
Finally, the difference between the listed enrollment of 49 and the brief summary’s statement of up to 36 participants makes transparent reporting of participant flow and analysis populations particularly relevant. The record does not explain this difference. Statistical interpretation depends on knowing which participants contributed to each outcome and how incomplete data were handled. These are not merely administrative details; they shape what can be inferred from the posted counts.
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 primary limitation for interpretation is that the registry record defines the primary endpoint but does not report its observed result. The listed secondary response outcomes also lack reported results in the information. No uncertainty intervals or formal statistical comparisons are included, and the record lists zero statistical analyses.
The design is single group, with allocation marked not applicable and masking listed as none. The dose cohorts therefore do not provide a randomized control comparison. Descriptive event counts cannot establish that dose caused any difference between groups. The record does not describe cohort assignment, escalation or stopping rules, or a method for comparing dose groups.
There is an unexplained enrollment discrepancy: the registry lists 49, whereas the brief summary says up to 36 participants would take part. The record does not say how this relates to the at-risk counts or to the populations used for each outcome. Analysis populations, missing-data handling, incomplete follow-up, and repeat-event counting for the primary event endpoint are not reported in the registry record.
The primary outcome’s 40-day window is stated, but the details do not describe how events outside that window were handled or whether observation was complete for all participants. The adverse-event summaries are not a replacement for the primary endpoint because their definitions and relation to its severity and timing criteria are not reported. The record names a top serious-event term with a percentage but does not give its denominator or further context in the available details.
The statistical plan, sample-size justification, interim analyses, and multiplicity procedure are not reported in the registry record. The planning assumptions described for sample-size illustrations do not establish the study’s design or observed power. Together, these reporting gaps constrain conclusions to a descriptive account of the study design and posted adverse-event counts.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Umbilical & Cord Blood (CB) Derived CAR-Engineered NK Cells for B Lymphoid Malignancies — NCT03056339.
- Registry record last updated 2024-03-25; status last verified 2024-03.
- Results first posted 2024-03-25.
- 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
The registry describes NCT03056339 as a completed Phase I/II, single-group dose-escalation study of umbilical cord blood-derived CAR-engineered NK cells with lymphodepleting chemotherapy in relapsed or refractory B-lymphoid malignancies. The primary endpoint is the number of grade 3–4 toxicity events within 40 days of NK-cell infusion. The registry record does not report that endpoint’s result or the listed secondary response results.
Adverse-event counts are listed for four dose cohorts, with zero deaths recorded in each. These are descriptive data, not a randomized comparison. The registry lists enrollment of 49, while the brief summary says up to 36 participants; the discrepancy is not explained. The statistical analysis plan, analysis populations, missing-data handling, and multiplicity methods are not reported in the registry record.