Irradiated Donor Cells Following Stem Cell Transplant in Controlling Cancer in Patients With Hematologic Malignancies: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Irradiated Donor Cells Following Stem Cell Transplant in Controlling Cancer in Patients With Hematologic Malignancies (NCT03272633), a non-randomized early/phase 1 study. The primary outcome is Number of Participants Who Received Irradiated Haploidentical Cells (IHC) With Disease Response.

2. Clinical Question

3. Trial Design

This was an early-phase 1, non-randomized, parallel-cohort clinical trial sponsored by Rutgers, The State University of New Jersey. The record lists enrollment of 2 and gives a study start date of 2020-10-26 and a primary completion date of 2022-06-30. Its status is terminated. Masking is listed as none. The registry record does not report why the study was terminated.

The study examined irradiated haploidentical cells after hematopoietic stem cell transplantation. Cohort I is described as receiving initial IHC within 42 days after hematopoietic engraftment following autologous transplantation. For participants without evidence of relapse or progressive disease, treatment could be given every 8-12 weeks for up to 3 doses. Cohort II covers initial IHC within 70 days after engraftment following allogeneic transplantation for high-risk disease, or initial treatment after documented relapse for participants treated for relapsed disease. The Cohort II description ends mid-sentence in the registry record, so no further details can be established from that description.

These cohorts differ in transplant context and treatment timing. Because allocation was non-randomized, comparisons between them would not isolate a treatment effect: differences could also reflect the patient groups, disease status, transplant type, or timing. The record does not report a control arm designed to provide a like-for-like comparison. Nor does it report a statistical analysis plan, allocation procedures beyond the non-randomized designation, or reasons for the termination.

The primary outcome is described for Cohort I, but the record explicitly says no data were collected for it. The registry also lists an outcome concerning induction of host T cells reactive with tumor-associated epitopes. It does not report an analysis for that outcome. Thus, the design and intended questions can be described, but the record does not support an estimate of efficacy or a comparison of cohort outcomes.

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

Analysis population for the primary outcome, as defined in the registry: no data was collected. Refers to the Cohort 1 Arm

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: No participants qualified for cohort I

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants Who Received Irradiated Haploidentical Cells (IHC) With Disease Response, measured over Up to 8 weeks after last protocol treatment.

The primary endpoint is the number of participants who received irradiated haploidentical cells and had a disease response. It is a count of participants, rather than a continuous measurement such as change in tumor size. The registry defines response using RECIST v1.0 criteria for target lesions assessed by MRI. A complete response means disappearance of all target lesions. A partial response means at least a 30% decrease in the sum of the longest diameter of target lesions. Overall response is defined as complete response plus partial response.

The stated assessment window is up to 8 weeks after the last protocol treatment. A response endpoint of this kind can provide a concise summary of whether participants meet prespecified imaging criteria during a defined follow-up window. General statistical context: converting imaging findings into response categories can make results easier to describe, but it also means the endpoint depends on the assessment rules and the availability and timing of evaluations.

The registry identifies the primary-outcome population as Cohort I and states that no data were collected for that outcome. Consequently, the record contains the endpoint definition but no observed response count or response proportion. It cannot establish how many participants met the criteria, nor how precise an estimate would have been.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What kind of endpoint is this?

The primary endpoint is a participant count: the number of people who received IHC and met the disease-response definition. General statistical context: binary outcomes classify each participant according to whether an event or criterion occurred. They are often reported as a count and a proportion, but the count and denominator must be known and clearly tied to the outcome population. The registry gives the endpoint definition and says no primary-outcome data were collected, so it does not supply those outcome quantities.

How are the response categories defined?

The registry uses RECIST v1.0 criteria for target lesions assessed by MRI. Complete response is defined as disappearance of all target lesions; partial response requires at least a 30% decrease in the sum of the longest diameter of target lesions. Overall response combines complete and partial responses. General statistical context: a composite category can capture more than one type of favorable classification, but its interpretation depends on the component definitions. The record states the categories but provides no observed classification results.

What does the follow-up window mean?

The endpoint timeframe is up to 8 weeks after the last protocol treatment. This sets the period in which the response outcome is intended to be assessed. General statistical context: a defined assessment window helps make outcome ascertainment more consistent, while differences in when assessments occur or whether they are available can affect the information used to classify participants. The registry record does not report assessment completion or a missing-assessment rule for this endpoint.

Why does non-randomized allocation matter?

The study is listed as non-randomized, with parallel cohorts. Cohort I and Cohort II have different treatment-timing descriptions, and Cohort II includes different transplant and disease-status contexts. General statistical context: random allocation can help balance both measured and unmeasured characteristics between comparison groups. Without it, group differences may reflect who entered each cohort or the circumstances of treatment, as well as any treatment-related difference. The registry explicitly warns that the arms are not a like-for-like control comparison. No adjustment strategy is reported.

What can uncertainty measures tell a reader?

General statistical context: a confidence interval conveys the uncertainty around an estimated effect or outcome value, under the assumptions of the statistical method. Its width depends on the information available and the method used. A narrow interval indicates greater precision than a wide one, but neither interval establishes causality on its own. The registry record does not report a response estimate or confidence interval, and it lists no statistical analyses. There is therefore no primary-endpoint interval to interpret.

What is over-dispersion, and is it relevant?

General statistical context: over-dispersion occurs when observed variability is greater than a statistical model expects. It can make standard errors too small if the model does not account for that extra variability. Its relevance depends on the outcome type and model. The primary endpoint is described as a participant count, but the registry record does not report a model or an analysis. It is not possible to say that over-dispersion was assessed or that a particular modeling approach was used.

How should planning illustrations be distinguished from results?

General statistical context: sample-size calculations are planning exercises based on assumptions about the effect to detect, the significance level, allocation, and statistical method. They are not observed outcomes and do not show the probability that a completed study detected an effect. The registry's planning assumptions describe a two-sided significance level of 0.05, equal allocation, a normal approximation, and effects expressed as fractions of an observed effect. The record does not provide a primary-outcome effect estimate because no primary-outcome data were collected. Any planning illustration based on an effect assumption should therefore not be read as a trial result or as observed power.

The registry does not report an inferential model, hypothesis-testing procedure, adjustment for cohort differences, or analysis population details beyond the Cohort I designation for the primary outcome. These methods cannot be reconstructed from the endpoint definition alone.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Cohort II (Initial IHC Within 70 Days or After Rel")
n      <- c(2)
events <- c(2)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Cohort II (Initial IHC Within 70 Days or After Rel  2  2  100.0  15.8  100.0  34.2  100.0  29.2  99.2

11. Multiplicity, Missing Data and Analysis Populations

The record lists 3 outcome measures, including the primary disease-response endpoint and a secondary outcome concerning induction of host T cells reactive with tumor-associated epitopes. It reports no statistical analyses. A statistical plan for handling multiple outcomes or multiple comparisons is not reported in the registry record. Without such a plan or analysis results, it is not possible to determine whether any multiplicity adjustment was intended or used.

The primary endpoint description identifies Cohort I as its population and explicitly states that no data were collected. The registry does not describe why those data are absent, whether assessments were attempted, or how missing response assessments would have been handled. It also does not report imputation, sensitivity analyses, or rules for excluding participants from an endpoint analysis. These procedures are not reported in the registry record.

General statistical context: examining multiple outcomes can increase the chance of finding an apparently notable result by chance if each is tested separately. Prespecified priorities and transparent analysis methods help readers distinguish planned tests from exploratory analyses. Missing outcome data can also affect interpretation, particularly when the reasons for missingness are related to disease status or treatment. Those general concerns cannot be quantified for this study from the available registry entries.

The record does not describe a separate intention-to-treat or per-protocol analysis population for the primary endpoint. The enrollment figure should not be assumed to be the denominator for an outcome when the record says no outcome data were collected.

12. 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 serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: musculoskeletal and connective tissue disorders (0.0%).

The serious adverse-event entries list 0 participants at risk and 0 affected in Cohort I. For Cohort II, the record lists 2 participants at risk and 0 affected by serious adverse events. Deaths are listed as 0 in both cohorts. The top serious-event term is musculoskeletal and connective tissue disorders, listed as 0.0%.

The entries for adverse events other than serious adverse events list 0 at risk and 0 affected in Cohort I, and 2 at risk and 2 affected in Cohort II. These figures are descriptive registry entries, not a comparison between randomized groups. Cohort I has zero participants recorded at risk, so its zero event count should not be read as evidence that no events occurred among an observed group.

General statistical context: safety counts are most informative when the risk population, event definitions, observation period, and reporting completeness are clear. The registry figures do not provide detailed event descriptions or an explanation of the zero at-risk entry for Cohort I. They should not be interpreted as a complete characterization of safety or as a causal comparison between cohorts.

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

The record documents a study of irradiated haploidentical cells given after stem cell transplantation, with different timing descriptions for its non-randomized cohorts. It also documents the intended primary response criteria and a safety summary. The primary-outcome population is identified as Cohort I, but the registry states that no data were collected for that outcome.

Accordingly, the record does not establish whether participants met the RECIST response criteria. It provides no observed response count, no response estimate, and no statistical uncertainty around such an estimate. The enrollment figure alone cannot answer the primary question. The outcome definition is not an outcome result.

The study's non-randomized structure also limits comparisons between cohorts. Their differing transplant contexts and treatment timing mean that any observed contrast would not automatically represent the effect of IHC. The registry itself notes that the arms are not a like-for-like control comparison. No comparative statistical analysis is reported.

The safety entries describe the events recorded for the cohorts, including zero serious adverse events and zero deaths in the listed entries. They should be read alongside the at-risk entries and the limitations of registry reporting, especially the zero at-risk entry for Cohort I. They do not establish comparative safety or explain why the study was terminated.

General statistical context: a study record can provide useful information about intended eligibility, treatment, endpoint definitions, and reporting even when it cannot provide an effect estimate. The distinction between an unanswered research question and evidence of no effect is important. The registry's absence of primary-outcome data is not evidence that the intervention did or did not change disease response. The reason those data are absent is not reported in the registry record.

14. Why This Trial Matters Statistically

This trial is statistically notable because it illustrates the difference between an outcome being defined and an outcome being observed. The registry specifies a response endpoint, assessment method, and timeframe, but reports that no data were collected for the primary outcome. Without observations, there is no response estimate to interpret and no basis for quantifying its precision.

It also illustrates why design labels matter. A non-randomized study with parallel cohorts may describe treatment experience, but its cohorts are not automatically valid controls for each other. Here, cohort descriptions differ in transplant setting, disease status, and timing of initial IHC. Those design features constrain any interpretation of between-cohort differences, even if results were available.

The record further separates efficacy questions from safety reporting. Safety counts are present, but they do not answer the primary disease-response question. Their interpretation depends on the listed at-risk populations, and a zero at-risk entry is not equivalent to observing a group with no events.

General statistical context: clear outcome definitions, complete follow-up, transparent analysis populations, and a prespecified statistical approach all help readers judge what a study can support. The registry record gives some design and endpoint details but does not report a statistical analysis plan, missing-data methods, or an explanation for termination. Recognizing these gaps prevents a study's stated aim from being mistaken for evidence of an outcome.

15. 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 is that the registry record states no data were collected for the primary outcome. As a result, the intended response endpoint cannot be estimated from this record. The entry does not explain why outcome data were not collected, and the reason for termination is also not reported in the registry record.

The study is non-randomized, and its parallel cohorts differ in their stated treatment timing and clinical context. The record cautions that they are not a like-for-like control comparison. There is no reported randomized comparator or adjustment analysis that would support a causal comparison between cohorts.

The primary endpoint is associated with Cohort I, but the safety entries show 0 participants at risk for that cohort. The registry does not explain this entry or give additional details that would clarify the relationship between enrollment, outcome assessment, and safety reporting. Cohort II's description is incomplete in the registry record, ending mid-sentence.

Statistical methods for the primary endpoint, missing data, multiplicity, and analysis populations are not reported. The registry also does not provide response estimates, uncertainty intervals, or analysis results. These limits mean that the record supports a description of intended design and recorded safety entries, but not conclusions about disease response or comparative effects.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Irradiated Donor Cells Following Stem Cell Transplant in Controlling Cancer in Patients With Hematologic Malignancies — NCT03272633.
• Registry record last updated 2023-06-26; status last verified 2023-06.
• Results first posted 2023-06-26.
• 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.

19. Record Summary

This is an early-phase 1, non-randomized, parallel-cohort study sponsored by Rutgers, The State University of New Jersey, with enrollment of 2. The record lists a start date of 2020-10-26, primary completion date of 2022-06-30, no masking, and terminated status.

The study examined irradiated haploidentical cells after stem cell transplantation. Cohort I and Cohort II have different timing and clinical-context descriptions. The primary outcome is the number of participants with disease response by RECIST v1.0 criteria assessed by MRI, up to 8 weeks after the last protocol treatment. The registry states that no data were collected for this primary outcome.

The registry lists 3 outcome measures and no statistical analyses. Safety entries include zero serious adverse events and zero deaths in both cohorts, with different at-risk entries by cohort. The reason for termination and the explanation for the absence of primary-outcome data are not reported in the registry record.