Ex Vivo-activated Autologous Lymph Node Lymphocytes in Treating Patients With Chronic Lymphocytic Leukemia: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Ex Vivo-activated Autologous Lymph Node Lymphocytes in Treating Patients With Chronic Lymphocytic Leukemia (NCT02530515), a na phase 2 study. The primary outcome is Treatment Success and Feasibility of Autologous Activated T-cells Infusion, Determined by Number of Participants That Achieved Target-Activated T-cell Dose Without DLT..

2. Clinical Question

The clinical question is about whether the cell infusion could be delivered at the target dose without the specified toxicities, rather than about a randomized comparison with another treatment. The registry defines success using both a dose criterion and the absence of DLT. Feasibility is defined as meeting those conditions in >50% of enrolled patients. That threshold is a criterion in the endpoint definition; it is not a reported finding. The registry record does not report how many participants met the target dose, how many experienced DLT, or how many met the combined success definition.

Immune reconstitution, overall response rates, and incidence of infections are listed as secondary outcomes. Their presence indicates additional clinical and biological questions, but does not establish that corresponding results are reported. The posted record identifies no comparator, so any outcome counts would describe this treated group and would not by themselves estimate how outcomes differ from those under another treatment or usual care.

3. Trial Design

This is a phase II, single-group trial of ex vivo-activated autologous lymph node lymphocytes in patients with CLL. The registry lists M.D. Anderson Cancer Center as the sponsor, enrollment of 8, and status as completed. The recorded start date is 2015-12-18, and the primary completion date is 2018-04-30. Masking is listed as none, so the design is unmasked. Allocation is listed as NA, consistent with the single-group model rather than a randomized allocation.

The intervention description identifies a biological treatment, with laboratory biomarker analysis also listed. The treatment-arm description says that patients receive an intravenous infusion of ex vivo-activated autologous lymph node lymphocytes over 10-30 minutes on day 0. The registry record does not describe a concurrent control group. It is therefore important not to interpret the arm labels in the adverse-event data as evidence of randomized or comparable groups: the record lists Arm A and Arm B with no participants at risk, and Arm C with 8 at risk.

The stated primary endpoint concerns the number of participants who achieve the target activated T-cell dose without DLT. Its timeframe is enrollment up to day 100 post T-cell infusion for each arm, while the endpoint definition describes DLT criteria occurring by day +90 after infusion. The record lists immune reconstitution, overall response rates, and incidence of infections as secondary outcomes. It does not report a detailed statistical analysis plan, sample-size justification, interim analyses, or outcome-analysis populations. The absence of those details means the design can be described from its registry entries, but cannot be supplemented with assumptions about how outcomes were intended to be analyzed.

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: Patients who qualify for lymphodepleting chemotherapy and who do not have 17p deletion are randomized to Arm A or Arm B. Those patients who do not qualify by those criteria are assigned to Arm C.

Overall Study

Analysis population for the primary outcome, as defined in the registry: Patients with CLL.

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: All patients were enrolled in Arm C at the physician's discretion and there is no analysis to compare between the lenalidomide Arm A vs the non-lenalidomide Arm B

6. Primary Endpoint

The registry lists the primary outcome as Treatment Success and Feasibility of Autologous Activated T-cells Infusion, Determined by Number of Participants That Achieved Target-Activated T-cell Dose Without DLT., measured over Enrollment up to day 100 post T cell infusion for each arm..

The primary endpoint is a count: the number of participants who achieve the target activated T-cell dose without dose-limiting toxicity. The target is stated as 1x108 +/-20%. Success requires both achieving that dose and having no DLT under the trial definition. DLT includes specified severe non-hematologic, allergy/immunology, autoimmune, or hematologic toxicities judged related to T cells and occurring within the defined post-infusion period; the definition also excludes certain preexisting adverse events due to prior treatment or disease. The registry describes the primary endpoint timeframe as enrollment up to day 100 post T-cell infusion for each arm.

This endpoint is used to assess whether the intended cell dose can be delivered without the specified toxicities. The registry defines feasibility as achieving the target dose without DLT in >50% of patients enrolled. That threshold is a prespecified definition in the record, not evidence that the threshold was reached. The registry record does not report the number achieving the target dose, the number with DLT, or the number meeting the endpoint. It therefore does not establish the observed success or feasibility result.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry record identifies a single-group phase II study and a primary endpoint expressed as a participant count. It does not list outcome analyses or provide an inferential analysis plan. The statistical interpretation should therefore distinguish between the endpoint definition, the descriptive adverse-event counts, and any statistical estimate that might have been produced elsewhere. Only the first two are described in the posted record.

What does a participant count represent?

A count records how many people meet a defined condition. For this trial, the condition combines receipt of the target activated T-cell dose with the absence of DLT. A count is directly interpretable only when its definition and denominator are clear. The registry states a feasibility criterion based on the proportion of enrolled patients, but the number meeting the endpoint is not reported. The endpoint cannot be reconstructed from the enrollment figure alone: enrollment does not tell how many reached the target dose or avoided DLT.

In general, a proportion places an event count in relation to a denominator, while an interval estimate expresses uncertainty around that proportion under a statistical model. Neither the primary count nor an interval estimate is reported for this endpoint. No new estimate can therefore be drawn from the registry description. The feasibility threshold of >50% is a criterion specified by the endpoint definition; it is not a statistical finding.

Why does the single-group design matter?

In general, a single-group study can describe outcomes among participants who receive the intervention, but it does not directly estimate a contrast with a concurrent control group. A comparison requires a suitable comparator and a defined analysis of outcomes between groups. This registry record lists a single-group model, no masking, and allocation as NA. It does not describe a like-for-like control group. The adverse-event labels Arm A, Arm B, and Arm C do not alter that design description, particularly because only Arm C has participants at risk in the listed safety data.

Without a comparison group, changes or events observed in participants cannot be separated statistically from what might have occurred without the intervention using this record alone. General statistical reasoning also cautions that outcomes in a small group can be sensitive to the experience of individual participants. That does not make descriptive counts unusable; it limits the scope of conclusions they can support.

What is the role of DLT criteria?

The endpoint combines a dose target with safety criteria. The record defines DLT using specified toxicity categories, grades, attribution to T cells, and a post-infusion period. Such operational definitions help determine consistently which observations count toward an endpoint. Here, the registry also notes exceptions for preexisting adverse events due to prior treatment or disease. The record does not report how many events met each criterion, how attribution was adjudicated, or how many participants met the combined endpoint. Those details would be needed to understand the endpoint count more fully.

The primary endpoint timeframe is described as enrollment up to day 100 post T-cell infusion for each arm, while the DLT definition refers to events by day +90 after infusion. The record does not explain how those time references were reconciled in outcome reporting. They should be quoted as stated rather than treated as proof of a particular analysis window.

How should uncertainty and planning be understood?

In general, uncertainty intervals around counts or proportions indicate how much the estimate could vary because of sampling, conditional on the statistical assumptions. A narrow interval suggests greater precision than a wide interval, but does not remove design limitations such as the absence of a comparator. No primary endpoint estimate or interval is reported in the registry record, so its precision cannot be evaluated.

Sample-size planning is also distinct from an observed result. In general, planning calculations use assumptions about an effect, variability, significance level, allocation, and analysis method to illustrate how much information a future study might need. Such calculations are not observed power and do not demonstrate that a completed study succeeded. The registry describes planning illustrations based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It does not report the calculated planning values in the registry record, and its single-group design should not be mistaken for a two-arm randomized comparison.

In general, over-dispersion occurs when count data vary more than a simple statistical model expects; accounting for it can prevent uncertainty from being understated. That concept does not establish that over-dispersion was relevant to this trial. The registry record does not report a model for count outcomes, an over-dispersion assessment, confidence intervals, hypothesis tests, or adjusted estimates. No such method should be attributed to the investigators based on the posted information.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary endpoint and secondary outcomes of immune reconstitution, overall response rates, and incidence of infections. Considering multiple outcomes can raise a multiplicity issue: in general, testing many hypotheses can increase the chance of at least one apparently notable result by chance. Analysts may address this through a prespecified testing strategy or adjustment, depending on the study aims. The registry record does not report a multiplicity strategy, so it is not possible to say whether any adjustment was planned or used. The record also lists 0 outcome analyses, and does not provide reported results for the named secondary outcomes.

Missing-data handling is not reported in the registry record. There is no description of how incomplete measurements, missed assessments, withdrawal, or unavailable endpoint classifications were treated. The enrollment figure of 8 is not enough to establish the denominator used for an outcome analysis or whether every enrolled participant was evaluated for each outcome. It would be inappropriate to assume that all participants had complete follow-up or that missing observations were handled using a particular method.

The analysis populations are also not reported in the registry record. For example, the record does not state whether primary endpoint reporting would include all enrolled participants, only participants who received the infusion, or another defined group. These distinctions matter because the endpoint includes both a target dose and absence of DLT. The protocol's intended analysis population, the number included in any result, and reasons for exclusions are not available in the registry description.

In general, prespecified definitions of outcome populations, missingness, and multiplicity help readers understand what an estimate represents and how strongly to interpret it. Their absence does not show that investigators failed to plan these issues; it means they are not reported in the registry record. Interpretation should remain limited to the design and counts that are actually posted.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent non-serious adverse event terms

The serious adverse-event entries list Arm C with 8 participants at risk and 0 affected; deaths are listed as 0. Arms A and B each have 0 participants at risk and 0 affected. These are registry counts, not evidence of a comparative safety difference, because the trial is described as single group and the arm entries do not provide a like-for-like comparison.

For other adverse events, Arm C has 8 participants at risk and 8 affected. The registry information does not identify the event terms, grades, timing, seriousness, or attribution for those entries. The count therefore indicates that other adverse events were recorded for all 8 participants at risk in that category, but does not show what those events were or how clinically important they were.

Zero recorded serious adverse events and zero deaths in this group should be read as observed counts, not proof that such events cannot occur. In general, the ability of a study to characterize uncommon harms depends on the amount and completeness of safety observation. The registry record does not provide further detail on safety follow-up completeness or event-level summaries, so broader safety conclusions cannot be drawn from these figures alone.

12. What the Result Does, and Does Not, Mean

The registry record describes a study designed to examine whether a target dose of activated autologous T cells could be delivered without the specified DLTs. It defines feasibility as achieving the target dose without DLT in >50% of enrolled patients. However, the number of participants meeting that combined endpoint is not reported in the registry record. The central feasibility result therefore cannot be determined from the posted results.

The adverse-event entries do report 0 serious adverse events and 0 deaths among 8 participants at risk in Arm C, while 8 participants at risk are listed as affected by other adverse events. Those counts are descriptive and do not establish cause. The specific other adverse events are not reported in the information described, and the empty Arm A and Arm B entries do not create comparator groups. The design is single group, not a randomized comparison.

The listed secondary outcomes point to additional questions about immune reconstitution, overall response rates, and infections. Their listing does not mean that findings for those outcomes are available. No outcome analyses are listed, and the record does not provide effect estimates, confidence intervals, or a detailed analysis plan. Consequently, claims about response, comparative efficacy, or the size and precision of treatment effects are not supported by the registry information.

In general, statistical results need to be interpreted in light of design, measurement, and uncertainty. A single-group count may describe what occurred in the enrolled participants, but it cannot by itself establish what would have happened under a different treatment. Likewise, absence of a reported result is not evidence that an endpoint was failed or achieved. The defensible conclusion is limited: the registry defines the feasibility and toxicity question, reports enrollment and some safety counts, but does not report the primary endpoint result needed to answer that question.

13. Why This Trial Matters Statistically

This trial is statistically notable because its primary question is framed as feasibility rather than as a comparison between treatments. Feasibility endpoints can be useful when a study needs to establish whether an intervention can be delivered at a target dose while meeting specified safety conditions. The registry makes that objective concrete by defining a target dose, DLT criteria, and a threshold of >50% of enrolled patients for feasibility.

That clarity in the endpoint definition also highlights the importance of reporting the observed endpoint count. A definition says what qualifies as success; the count says how many participants met it. Since the count is not reported in the registry record, readers cannot judge whether the stated feasibility threshold was reached. This illustrates why complete outcome reporting is essential even when a study's main purpose is feasibility rather than estimation of a comparative treatment effect.

The single-group structure also shapes the statistical claims available. Descriptive results can characterize participants in the study, but without a concurrent comparator they do not estimate a treatment contrast. The registry's Arm A, Arm B, and Arm C safety labels need to be read alongside the single-group design and the fact that only Arm C has participants at risk in the listed safety data. Treating those labels as equivalent randomized arms would misrepresent the design.

Finally, the record illustrates the difference between a planned threshold, a reported observation, and an inferential conclusion. The feasibility threshold is stated, some adverse-event counts are posted, but the primary endpoint result and outcome analyses are not. Keeping these elements separate allows readers to recognize what the study set out to assess without overstating what the registry record establishes.

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 direct limitation for interpretation is that the registry record does not report the primary endpoint count. Without the number achieving the target dose without DLT, the feasibility definition cannot be evaluated. The record also does not provide an endpoint estimate or confidence interval, so the precision of any feasibility result is unavailable.

Enrollment was 8, and the study is single group. These design features limit what can be concluded about comparisons with other treatments; the record does not describe a concurrent control. The adverse-event data list Arm A and Arm B with no participants at risk and Arm C with 8 at risk, so the labels should not be interpreted as balanced comparison arms.

The safety information is incomplete for detailed interpretation. Although serious adverse events and deaths are listed as 0 in Arm C, the record does not provide event-level descriptions for the 8 participants affected by other adverse events. It also does not report severity, timing, attribution, or completeness of safety follow-up in the information described.

Further limitations arise from reporting gaps. The registry lists secondary outcomes but does not report their results, and it lists 0 outcome analyses. Missing-data methods, analysis populations, multiplicity handling, sample-size justification, and interim analyses are not reported in the registry record. The record's differing descriptions of the primary endpoint timeframe and DLT window are not reconciled in outcome reporting. These omissions constrain interpretation; they do not establish that any particular procedure was or was not used.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Ex Vivo-activated Autologous Lymph Node Lymphocytes in Treating Patients With Chronic Lymphocytic Leukemia — NCT02530515.
• Registry record last updated 2019-10-29; status last verified 2019-10.
• Results first posted 2019-10-29.
• 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.

18. Record Summary

NCT02530515 is a completed phase II, single-group study sponsored by M.D. Anderson Cancer Center, with enrollment of 8. It evaluates infusion of ex vivo-activated autologous lymph node lymphocytes in patients with CLL. The recorded start date is 2015-12-18, and the primary completion date is 2018-04-30. The study is unmasked, and allocation is listed as NA.

The primary endpoint counts participants achieving a target activated T-cell dose without DLT. Feasibility is defined as meeting that condition in >50% of enrolled patients. The registry record does not report the primary endpoint result or any outcome analyses. It lists secondary outcomes for immune reconstitution, overall response rates, and incidence of infections.

For Arm C, the safety entries list 8 participants at risk for serious adverse events, 0 affected, and 0 deaths; 8 are listed at risk and affected for other adverse events. The record does not identify those other events in the information described. The single-group design does not support treating the arm labels as a control comparison.