A Pilot Study of the Therapeutic Potential of Stem Cell Educator Therapy in Type 1 Diabetes: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Pilot Study of the Therapeutic Potential of Stem Cell Educator Therapy in Type 1 Diabetes (NCT02624804), a na phase 1 study. The primary outcome is Number of Participants With Treatment Related Adverse Events.

2. Clinical Question

The primary endpoint is a participant count, not a measure of change in diabetes control. The registry describes the study as assessing safety, feasibility, and efficacy, but the posted outcome information identifies treatment-related adverse events as the primary endpoint. It also lists the number of patients unable to complete therapy as a secondary outcome. The registry record does not report an analysis of that secondary outcome.

Because everyone in the study received the intervention and there was no control group, the central question is descriptive: what safety events were recorded among the participants represented in the posted safety results? A single-group pilot can document experience with an intervention, but it cannot by itself establish what would have happened to similar participants without it. Nor does the registry record provide an efficacy result that can be compared between groups.

3. Trial Design

This was a prospective, single-arm, open-label, single-center pilot study of Stem Cell Educator therapy in type 1 diabetes. The registry lists the study as Phase 1 and completed, with enrollment of 13. Its listed start date is 2017-06-27 and its primary completion date is 2022-11-11. The sponsor is Hackensack Meridian Health.

In a single-arm study, participants receive the intervention without a separate control group. The registry lists allocation as NA and masking as none. Accordingly, the results do not represent a randomized comparison, and the intervention group should not be treated as directly comparable with an untreated or alternative-treatment group. Open-label means the study was not masked; details about outcome assessment procedures are not reported in the registry record.

The intervention description states that patients have apheresis performed and then have their own blood returned with “educated” lymphocytes. Further procedural details, including how the treatment was delivered beyond that description, are not reported in the registry record. The brief summary says the pilot was intended to assess safety, feasibility, and efficacy. The registry’s primary endpoint, however, is the number of participants with treatment-related adverse events, assessed during therapy and over a 12-month follow-up period.

The registry lists two outcome measures, including the primary safety endpoint and the secondary outcome “Number of Patients Unable to Complete Therapy.” It reports zero analyses. Detailed eligibility criteria, participant flow, treatment completion counts, protocol deviations, and the statistical plan are not reported in the registry record. The enrollment figure and the safety-result denominators are not the same; how those figures relate to one another is not reported in the registry record.

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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Patients Experiencing Treatment Related Adverse Events

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.

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants With Treatment Related Adverse Events, measured over 12 months.

The primary endpoint is the number of participants with treatment-related adverse events. The unit is participants, and the registry describes the measure as a count of participants. Its timeframe is 12 months. The endpoint description says adverse events occurring during therapy, particularly those requiring temporary or permanent discontinuation, and during the 12-month follow-up period will be assessed.

A participant count is a safety summary: it indicates how many participants are reported as experiencing an event under the endpoint definition. It is not itself a measure of event severity, duration, or causal certainty, and a participant count does not necessarily communicate how many separate events occurred. Those details are not reported in the registry record for the primary endpoint.

Safety endpoints are used to describe harms observed during and after an intervention, which is especially relevant in a pilot study described as assessing safety and feasibility. General statistical context: counting participants with at least one event can make the result easier to interpret than counting every event when some participants experience more than one. However, the registry record does not state the detailed event-counting rules or provide an analyzed result for this primary endpoint. It lists safety counts separately, but does not report a primary-endpoint analysis or clarify whether those counts use exactly the endpoint’s treatment-related definition and 12-month timeframe.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a participant count describe?

The primary outcome is defined as the number of participants with treatment-related adverse events. In general, a participant-level count records whether participants experienced an outcome, rather than tallying every occurrence. That distinction matters because one participant could, in principle, experience more than one event. The registry record does not report the detailed rules used to classify or count events, nor does it give an analyzed result for this primary endpoint. The separately posted safety counts should therefore be read according to their listed categories, without assuming that they are a complete analysis of the primary endpoint.

The record describes the primary timeframe as 12 months and says events during therapy and the follow-up period are assessed. General statistical context: defining a timeframe helps establish which events belong in an endpoint, but interpretation also depends on how follow-up was handled, when participants entered or left observation, and how events were attributed. Those procedural and attribution details are not reported in the registry record.

Why does study design affect interpretation?

This study is single-group, open-label, and non-randomized, with allocation listed as NA. There is no concurrent control group in the registry record. General statistical context: random assignment can help make treatment groups comparable, while a control group gives a reference for interpreting outcomes. Without those features, observed events cannot be contrasted with outcomes under an alternative treatment or no intervention. The study can describe events recorded among the participants represented in its safety results, but it does not estimate a treatment-versus-control difference.

Open-label status means that masking was absent. General statistical context: knowledge of treatment assignment can affect reporting, observation, or classification of outcomes, depending on how those outcomes are collected. The registry record does not report details of event ascertainment or whether any outcome assessment was independently reviewed, so those aspects cannot be evaluated from the posted information.

What do the safety denominators mean?

The registry lists 8 participants at risk for serious adverse events, with 1 affected, and 8 at risk for other adverse events, with 4 affected. It also lists 0 deaths. These figures are counts as posted; no new rates or comparisons are calculated. The enrollment is 13, but the record does not explain why the safety denominators are 8 or whether the serious and other-event groups include the same participants. It also does not describe whether “affected” represents participants with at least one event, or provide event-level counts. Such details are important because participant counts and event counts answer different questions.

General statistical context: a denominator defines the group to which a count pertains. If the denominator or observation period is unclear, the scope of the count is also unclear. The registry record does not report enough detail to resolve these questions for the posted safety data.

What can confidence intervals and significance tests add?

General statistical context: a confidence interval expresses the uncertainty around an estimate under a specified statistical model. A significance test evaluates how compatible data are with a null hypothesis under its assumptions. Neither is reported for the primary endpoint in this record. With no reported analysis and no control group, there is no posted comparative effect estimate to interpret through a confidence interval or a treatment-comparison significance test.

A confidence interval would not correct limitations in the design or missing outcome information. It would describe uncertainty for the quantity estimated and model used. The registry record does not report a confidence interval, an endpoint estimate, or an inferential procedure for the primary endpoint. It is therefore not possible to characterize its precision statistically from the posted results.

How should the planning illustration be read?

The record describes a sample-size planning illustration based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It says the effect to detect is a stated fraction of the observed effect and distinguishes participant counts per arm from total event counts for time-to-event outcomes. No numerical planning results are reported in the record. The illustration is not the trial’s own design; it should not be mistaken for a sample-size justification for this single-arm pilot or as a measure of observed power.

General statistical context: planning calculations depend on assumptions about the target effect, variability, allocation, and analysis. If those assumptions differ from the actual design or data structure, the resulting illustration does not describe the achieved precision of the trial. The record does not report the trial’s own sample-size justification, so that rationale cannot be evaluated.

Finally, the registry reports zero analyses. The statistical analysis plan, model specifications, handling of missing outcomes, and analysis populations are not reported in the registry record. Without those details and the primary-endpoint results, the posted information supports a descriptive account of the listed safety counts, not a broader statistical inference.

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("Stem Cell Educator Therapy")
n      <- c(8)
events <- c(1)
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  Stem Cell Educator Therapy  8  1  12.5  0.3  52.7  2.2  47.1  2.8  48.2

11. Multiplicity, Missing Data and Analysis Populations

The registry lists two outcome measures: the primary count of participants with treatment-related adverse events and the secondary count of patients unable to complete therapy. It reports zero analyses. Results for the secondary outcome are not reported in the registry record, and an analyzed result for the primary endpoint is also not reported. The record does not provide a statistical analysis plan or state whether multiple outcomes were considered in any inferential analysis.

General statistical context: when several outcomes are tested, the chance of at least one apparently unusual result can increase if each is assessed separately. Methods for addressing this issue depend on the analysis plan and the purpose of the outcomes. Since no analyses or multiplicity procedure are reported, it is not possible to determine whether multiplicity was assessed or adjusted for.

Missing-data methods, reasons for missing observations, and follow-up completeness are not reported in the registry record. The relationship between the enrollment of 13 and the safety denominators of 8 is also not explained. This does not establish that data were missing; it means the record does not give enough information to assess who contributed to each posted count or how unobserved outcomes were handled.

The analysis population is not reported in the registry record. It is therefore unclear whether the counts refer to all enrolled participants, participants who started therapy, participants with safety follow-up, or another defined group. No imputation method, sensitivity analysis, or participant-level flow information is reported. These gaps limit assessment of whether the posted results represent all enrolled participants and how robust any outcome interpretation would be to incomplete follow-up.

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 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: depression (12.5%).

The registry lists 8 participants at risk for serious adverse events and 1 affected. For other adverse events, it lists 8 at risk and 4 affected. It reports 0 deaths and identifies Depression at 12.5% as the top serious adverse-event term. These figures should be read as registry-reported safety information, not as a comparison with another group.

The record does not explain the relationship between the 8-person safety denominators and the enrollment of 13. It also does not specify whether the serious and other-event categories include overlapping participants, how many separate events occurred, or the precise denominator and counting rule for the Depression percentage. The percentage should not be converted into a participant count without those details.

General statistical context: “serious” describes an event category, not necessarily its causal relationship to treatment. The registry lists serious adverse events but does not report an analysis establishing whether they were caused by therapy. The posted counts also do not indicate how events were distributed over the 12-month endpoint timeframe. They describe the safety information recorded in the registry; the completeness and follow-up basis are not reported.

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

The posted information establishes that this was a completed, single-arm pilot study of Stem Cell Educator therapy in type 1 diabetes, with 13 participants enrolled. The registry lists safety counts for a group of 8 participants at risk: 1 affected for serious adverse events and 4 affected for other adverse events. It also lists 0 deaths and identifies Depression at 12.5% as the top serious adverse-event term. These figures describe the information in the registry record; they do not provide a controlled estimate of treatment effect.

The record does not report an analysis of the primary endpoint, despite defining it as the number of participants with treatment-related adverse events over 12 months. It also does not report a result for the secondary outcome concerning inability to complete therapy. Although the brief summary says the study assessed efficacy, no efficacy analysis is reported in the registry record. Thus the available results do not establish whether diabetes-related outcomes changed or whether any change differed from what would occur without the intervention.

General statistical context: an event observed after treatment is not, by timing alone, evidence that the treatment caused it. A single-arm design has no concurrent control group to help separate treatment-related events from events that might occur for other reasons. Likewise, a small pilot’s descriptive experience does not automatically predict event frequency in a broader population.

The safety denominators are smaller than the enrollment, and the registry does not explain why. This limits confidence about which participants are represented in the safety counts. The record also does not report confidence intervals, detailed event definitions, missing-data methods, or analysis populations. Accordingly, the posted information supports a cautious description of the safety counts, but not a conclusion about comparative safety, efficacy, or the overall benefit-harm balance.

14. Why This Trial Matters Statistically

This trial is statistically informative as an example of the distinction between a study’s stated aims and the results available for analysis. The brief summary describes an assessment of safety, feasibility, and efficacy, while the registry identifies a participant count for treatment-related adverse events as the primary endpoint. The posted record includes some safety figures but reports zero analyses and no efficacy results. Readers therefore need to distinguish the existence of a planned question from the availability of an analyzed answer.

The single-arm design also illustrates the limits of descriptive safety evidence. Counts among treated participants can document what the registry reports, but without a control group they cannot quantify how outcomes compare with an alternative. This remains true even when a study is prospective and has a specified follow-up timeframe. The record lists 12 months for the primary endpoint, but does not report enough detail to assess follow-up completeness or the population represented by each safety denominator.

The enrollment of 13 alongside safety denominators of 8 makes transparent reporting of analysis populations and participant flow especially important. The registry does not explain the relationship between these figures. That absence prevents a reader from determining whether the counts represent all enrolled participants or a subset, and whether the two adverse-event categories use the same people.

General statistical context: pilot studies can contribute descriptive information and help identify questions for later research, but their findings should be interpreted according to the design and completeness of reporting. The stated sample-size illustration is not the study’s design and cannot substitute for an analysis of observed outcomes. This record demonstrates why effect estimates, uncertainty measures, outcome definitions, and clear denominators are needed before drawing broader statistical conclusions.

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 reports zero analyses and does not provide an analyzed result for the primary endpoint. It also does not report the secondary outcome result or an efficacy analysis, despite describing efficacy as one of the study’s aims. The posted information therefore cannot answer all the questions identified in the brief summary.

The single-group, open-label design has no randomized or masked comparator. Consequently, the safety counts cannot be used to estimate a comparative effect, and the design cannot distinguish intervention-related outcomes from other explanations. The registry record does not report detailed event attribution procedures or independent outcome assessment.

Enrollment is 13, while the serious and other adverse-event denominators are each 8. The registry does not explain the difference or define the analysis population. It also does not clarify whether those categories overlap, what “at risk” means, or whether “affected” counts participants or events. The Depression figure of 12.5% is not accompanied by a detailed denominator or counting explanation.

Eligibility criteria, participant flow, treatment completion results, missing-data handling, follow-up completeness, and the statistical analysis plan are not reported in the registry record. Although the primary timeframe is 12 months, details about the timing and completeness of safety observation are not reported. These gaps constrain interpretation of both the scope and precision of the posted safety information.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: A Pilot Study of the Therapeutic Potential of Stem Cell Educator Therapy in Type 1 Diabetes — NCT02624804.
• Registry record last updated 2026-04-03; status last verified 2026-03.
• Results first posted 2024-10-10.
• 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

NCT02624804 is a completed Phase 1, prospective, single-arm, open-label, single-center pilot study sponsored by Hackensack Meridian Health. It enrolled 13 participants with type 1 diabetes. The listed start date is 2017-06-27, and the primary completion date is 2022-11-11.

The intervention was Stem Cell Educator therapy, described as apheresis followed by returning the patient’s own blood with “educated” lymphocytes. The primary endpoint was the number of participants with treatment-related adverse events over 12 months. The secondary outcome was the number of patients unable to complete therapy.

The registry reports zero analyses. Safety figures list 8 at risk and 1 affected for serious adverse events, 8 at risk and 4 affected for other adverse events, and 0 deaths. Depression at 12.5% is listed as the top serious adverse-event term. The record does not explain the safety denominators or report an analyzed primary or efficacy result.