Pilot Study Evaluating Safety & Efficacy of DCBT: NiCord® & UNM CBU to Patients With Hematological Malignancies: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Pilot Study Evaluating Safety & Efficacy of DCBT: NiCord® & UNM CBU to Patients With Hematological Malignancies (NCT01221857), a na phase 1, phase 2 study. The primary outcome is Acute Toxicity Associated With the Infusion of NiCord.

2. Clinical Question

The study was designed as a pilot evaluation of safety and efficacy in people with hematological malignancies. Its stated primary outcome concerns acute toxicity associated with NiCord infusion. The registry describes monitoring for events such as fever, chills, allergic reactions, changes in heart rate or blood pressure, gastrointestinal symptoms, breathing or oxygenation problems, hemoglobinuria, infection, flank pain, and other possible toxicity manifestations.

The registry also lists acute graft-versus-host disease by grade and non-relapse mortality as secondary outcomes. Those outcomes address events after transplantation rather than only the immediate infusion period. The record lists four outcome measures overall, but it does not report the results or analysis details for those secondary measures.

Because this is a single-group study, the central question is descriptive: what safety outcomes were recorded among participants treated in this study? The design does not directly estimate how those outcomes compare with a separate treatment or control group. It also cannot, on its own, distinguish effects associated with NiCord from events related to transplantation, underlying illness, or other aspects of care.

3. Trial Design

The registry describes a phase 1/2, completed, single-group pilot study. Enrollment was 12 participants. The intervention is NiCord, used in combination with a second, unmanipulated cord blood unit for allogeneic stem cell transplantation. Although the registry lists NiCord as the intervention, the study title and description characterize the treatment as a co-transplantation approach.

The allocation entry is listed as NA, and the study model is single group. Masking is listed as none. There is therefore no randomized treatment allocation or masked comparison described in the registry record. The record does not give a separate control group, so the outcomes are interpreted as observations in the treated group rather than as contrasts between treatment strategies.

The listed conditions are acute lymphoblastic leukemia, acute myelogenous leukemia, myelodysplastic syndrome, non-Hodgkin's lymphoma, and Hodgkin's disease. The primary outcome is acute toxicity associated with NiCord infusion, measured as a count of participants. Its timeframe field says 180 days post-transplant, but the outcome description identifies adverse events within 24 hours post-infusion as the acute toxicity period. This difference in wording leaves the precise relationship between the stated timeframe and the acute event definition unclear.

The study start is listed as 2010-11, and primary completion as 2012-09. The registry status is completed. The sponsor is Gamida Cell ltd. The registry does not report the full protocol, eligibility details, treatment administration details, or the rationale for the enrollment target. It also does not describe the study's statistical analysis plan. Those omissions limit how fully the design and intended analyses can be reconstructed from the registry record alone.

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: 12 patients were enrolled/transplanted; efficacy results were summarized for 11 patients treated with NiCord. 1 patient was transplanted with unmanipulated cord only. Safety results were summarized for all patients.

Overall Study

Analysis population for the primary outcome, as defined in the registry: Patients transplanted with NiCord

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 Acute Toxicity Associated With the Infusion of NiCord, measured over 180 days post-transplant.

The primary endpoint is the count of participants with acute toxicity associated with NiCord infusion. It is expressed as a participant count rather than as a continuous measurement. The outcome description defines the acute toxicity period as the 24 hours after infusion and lists adverse events that were specifically monitored, including fever, chills, allergic reactions, changes in heart rate or blood pressure, nausea, vomiting, diarrhea, breathing difficulties, hypoxia, hemoglobinuria, infection, flank pain, and other manifestations.

In general, an event-count endpoint is useful when the study question is whether participants experienced a defined type of event. It can communicate the observed burden in direct terms, particularly in an early-stage safety study. It does not by itself describe event severity, duration, recurrence, or the time until an event occurred unless those features are separately defined and analyzed.

The registry’s timeframe field states 180 days post-transplant, whereas its description specifies events within 24 hours post-infusion as the acute toxicity period. The registry record does not clarify this difference. It also does not report a numerical result for the primary endpoint or details of how events were attributed, adjudicated, or handled if a participant experienced multiple events. Those details matter when interpreting a count and comparing it with outcomes from other studies.

The registry identifies participants transplanted with NiCord as the endpoint population. The primary endpoint is therefore relevant to describing acute safety observations in this treatment group, but the single-group design does not provide a concurrent comparison for estimating relative risk.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a participant count represent?

The primary outcome is defined as a count of participants with an event. In general, this type of measure records whether each participant experienced the event of interest during the specified observation period. It is different from a count of all individual event episodes: one participant with repeated episodes may still contribute one participant to an outcome defined as the number of participants affected. The registry record does not provide enough detail to determine how repeated events were treated in the primary outcome.

Participant counts are often easy to interpret, but their meaning depends on a clear event definition and observation window. Here, the endpoint description identifies adverse events within 24 hours after infusion, while the timeframe field states 180 days post-transplant. The record does not explain how those two time references are reconciled. Without clarification, it is important not to assume that the primary endpoint covers one period or the other beyond the wording in the record.

What can a single-group design estimate?

In general, a single-group design can describe outcomes observed among participants who receive a study intervention. It does not directly estimate a treatment contrast against a control group. Differences between groups are the basis for many comparative treatment-effect estimates; without a separate comparator, the observed event counts cannot show what would have happened to similar participants under another approach.

This distinction is especially important in transplantation studies, where illness, transplant procedures, and other care may also be relevant to observed events. The registry lists NiCord in combination with a second, unmanipulated cord blood unit, but does not provide a comparison that separates the contribution of either component. The reported adverse-event information should therefore be interpreted as a description of the study group, not as an isolated causal effect of NiCord.

How should uncertainty be understood?

In general, a confidence interval describes a range of values compatible with the observed data under a specified statistical method. For a participant count or event proportion, an interval can communicate uncertainty that a point estimate alone does not show. No confidence interval for the primary endpoint is reported in the registry record, and no primary analyses are listed. No inferential estimate should be inferred from the reported counts.

With 12 participants enrolled, each observation can matter substantially to a descriptive summary. This is a general feature of small studies: a result may change noticeably when a small number of outcomes differ. That does not make the observations uninformative, but it means they should be read as limited evidence about this study group rather than as a precise estimate for a wider population.

What do the planning assumptions mean?

In general, power calculations are planning tools used to assess how many participants might be needed to detect an effect under specified assumptions. They do not measure whether a completed study had “observed power,” and they do not turn an observed result into evidence of efficacy. The registry’s stated planning illustration uses a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and effects defined as fractions of an observed effect. It is explicitly described as a planning illustration rather than the trial’s own design.

The study itself is single-group, so assumptions involving equal allocation between two arms do not describe its actual treatment allocation. Any sample-size illustrations based on those assumptions should be understood as hypothetical planning scenarios, not as a record of how this pilot was designed or conducted. The registry does not report a sample-size justification for the enrolled group of 12, and the specific planning estimates are not described in the registry record.

What can be inferred from the safety summaries?

The registry reports 8 of 12 participants at risk as affected by serious adverse events and 11 of 12 as affected by other adverse events. These are descriptive participant-level summaries. In general, they do not convey the timing, duration, recurrence, severity within each category, or causal relationship of each event unless those details are separately reported. The listed serious adverse event terms and percentages add some detail about recorded event types but do not substitute for participant-level event histories or an adjudication method.

The registry also reports 3 deaths. A death count is an important safety observation, but it is not an estimate of treatment-attributable mortality without information on causes, timing, attribution, and an appropriate comparison. The record does not report those details. The secondary outcomes include non-relapse mortality, but the registry does not provide a numerical result or analysis for that outcome.

What does the absence of analyses imply?

The registry lists four outcome measures and zero analyses. This means the record does not present statistical analyses for those outcomes. It does not establish that no analyses were performed outside the registry, but those analyses are not reported in the registry record. No p-values, adjusted estimates, confidence intervals, or model specifications for the primary endpoint are available to interpret from this record.

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("NiCord")
n      <- c(11)
events <- c(0)
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  NiCord  11  0  0.0  0.0  28.5  -0.0  25.9  0.2  26.5

11. Multiplicity, Missing Data and Analysis Populations

The registry lists four outcome measures, including acute toxicity as the primary outcome and acute graft-versus-host disease by grade and non-relapse mortality among the secondary outcomes. It does not report a multiplicity strategy, such as whether or how statistical testing across outcomes would be adjusted. Because no analyses are listed, the record does not establish that formal hypothesis tests were conducted for these outcomes. A multiplicity adjustment should not be assumed.

Missing-data procedures are not reported in the registry record. The record does not explain how participants with incomplete follow-up, unavailable event information, or other missing measurements would have been handled. Nor does it describe whether any participants were excluded from an outcome analysis or how the analysis population was defined for each outcome.

The safety summaries use 12 participants at risk as the denominator for both the serious and other adverse-event counts. The registry does not provide a detailed account of follow-up completeness for these summaries. It also does not report a separate denominator for the primary endpoint, whose numerical result is not presented.

In general, missing-data rules and analysis-population definitions can affect the interpretation of trial results. For example, a result based on all enrolled participants may differ from one based only on participants with complete observations. No such analysis choices are described, so they cannot be reconstructed from the registry record. The lack of reported analysis details also means that the record does not show whether outcome assessments were handled consistently across the listed conditions or across the follow-up period.

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: gastrointestinal disorder (16.7%), hypertension (16.7%), pneumonia (8.3%), depression (8.3%), altered mental status (8.3%).

For the NiCord group, the registry reports that 8 of 12 participants at risk were affected by serious adverse events and that 11 of 12 were affected by other adverse events. It also reports 3 deaths. These are observed safety summaries for the study group, not estimates of a difference between treatments.

The registry lists gastrointestinal disorder and hypertension at 16.7% each among the serious adverse event terms, and pneumonia, depression, altered mental status, and hemoptysis at 8.3% each. The record does not explain whether participants could appear under multiple terms, how events were coded, or how these term-specific percentages relate to the participant-level counts. They should not be added together or interpreted as mutually exclusive categories.

In general, an adverse event is an event observed during a study; its presence alone does not prove that the intervention caused it. The registry does not provide causal attribution details for these events or deaths. It also does not report timing, duration, or outcomes for individual events. The listed safety figures therefore support a descriptive reading of what was recorded, but not a causal or comparative conclusion.

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

The registry record describes a completed pilot study of NiCord used with a second, unmanipulated cord blood unit in 12 participants with hematological malignancies. Its primary question concerns acute toxicity associated with NiCord infusion. The record also reports serious and other adverse-event counts and a death count for the study group.

Those observations matter as a description of what occurred among the enrolled participants. They do not demonstrate that the intervention caused a particular event, nor do they establish how outcomes would compare with another transplantation approach. There is no separate control group and no randomized comparison described. Other clinical factors may be relevant to the observed safety experience, and the registry does not provide information that would isolate the contribution of NiCord or the second cord blood unit.

The primary endpoint is not accompanied by a numerical result or an analysis in the registry record. The record’s timeframe wording is also not fully consistent: it gives 180 days post-transplant in the timeframe field but defines acute toxicity as events within 24 hours post-infusion in the description. This limits certainty about how the endpoint should be operationalized.

In general, a small pilot study can provide descriptive information and help identify questions for later research, but it is not necessarily designed to provide precise comparative estimates. The registry does not report a sample-size justification, a statistical analysis plan, or uncertainty intervals. It therefore does not support conclusions about the precision of an effect estimate, and it does not report a formal test of treatment efficacy.

The study’s title includes safety and efficacy, but the available registry information does not report efficacy results. The listed secondary outcomes include acute graft-versus-host disease by grade and non-relapse mortality, yet no numerical results or analyses for those outcomes are presented. The evidence available in the registry is consequently limited to the design description and the posted safety summaries.

14. Why This Trial Matters Statistically

Statistically, this trial is useful for illustrating the distinction between describing outcomes and estimating comparative effects. A single-group pilot can document events among participants exposed to a treatment approach. Without a comparator group, it cannot estimate how those outcomes differ from what would have occurred under another approach. That distinction is central when interpreting safety observations in a complex clinical setting.

The registry’s primary endpoint is a participant count, making the intended outcome understandable in broad terms. At the same time, the record’s differing timeframe statements show why operational definitions matter. A clear event definition and observation window are necessary for consistent measurement, reproducibility, and comparison with other studies. The record does not resolve whether the stated acute toxicity outcome is tied to the 24-hour period in its description or the 180-day timeframe field.

The reported safety counts also show why denominators and event categories matter. The participant-level counts indicate how many people were affected in the study group, while the term-specific percentages describe particular listed serious adverse event categories. The record does not explain how terms overlap or how event attribution was determined. A careful interpretation keeps these summaries descriptive and avoids treating them as treatment effects.

Finally, the record lists no analyses for its four outcome measures. Without reported estimates, uncertainty intervals, or a statistical plan, readers cannot assess the precision or inferential basis of the primary result. The trial remains statistically informative as a case in how design, endpoint definitions, and reporting determine what can be concluded—not as a comparative estimate of treatment effect.

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 principal limitation is the single-group design. There is no separate comparator and no randomized allocation described, so the registry cannot support a direct estimate of how outcomes differ from another treatment strategy. The observed adverse-event counts should not be interpreted as causal effects attributable to NiCord.

Enrollment was 12 participants. In general, a small study provides limited information about the range of outcomes that may occur in a broader population, and individual observations can have considerable influence on descriptive summaries. The registry does not report a sample-size justification or a precision target for the study.

The primary endpoint has an unresolved timeframe discrepancy. Its field lists 180 days post-transplant, while its description defines acute toxicity as adverse events within 24 hours post-infusion. The record does not clarify which period governs the endpoint. It also does not report the primary endpoint result, despite naming it as the primary outcome.

Analysis details are limited: the registry lists four outcome measures but no analyses. It does not report a statistical analysis plan, missing-data procedures, outcome-specific analysis populations, confidence intervals, or hypothesis tests. Although acute graft-versus-host disease by grade and non-relapse mortality are listed as secondary outcomes, their results are not reported in the registry record.

Safety reporting is also limited for interpretation. The record provides participant counts, selected serious adverse event terms and percentages, and a death count, but does not describe event timing, duration, attribution, coding rules, or whether term categories overlap. These gaps restrict what can be inferred from the safety summaries.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Pilot Study Evaluating Safety & Efficacy of DCBT: NiCord® & UNM CBU to Patients With Hematological Malignancies — NCT01221857.
• Registry record last updated 2021-08-03; status last verified 2021-08.
• Results first posted 2019-02-26.
• 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 completed phase 1/2 pilot study enrolled 12 participants with hematological malignancies. It evaluated NiCord, an ex vivo expanded cord blood-derived stem and progenitor cell product, in combination with a second, unmanipulated cord blood unit. The study was single group, with no masking and no randomized comparison described.

The primary outcome was a count of participants with acute toxicity associated with NiCord infusion. Its description defines the acute toxicity period as within 24 hours after infusion, while its timeframe field states 180 days post-transplant. The registry lists four outcome measures and zero analyses; it does not report a numerical result for the primary endpoint.

For the NiCord group, the registry reports serious adverse events in 8 of 12 participants at risk, other adverse events in 11 of 12, and 3 deaths. The record does not establish causal attribution for these events. It lists acute graft-versus-host disease by grade and non-relapse mortality as secondary outcomes but does not report their results or analyses.