Umbilical Cord Blood (UCB) Transplant, Fludarabine, Melphalan, and Anti-thymocyte Globulin (ATG) in Treating Patients With Hematologic Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Umbilical Cord Blood (UCB) Transplant, Fludarabine, Melphalan, and Anti-thymocyte Globulin (ATG) in Treating Patients With Hematologic Cancer (NCT00827099), a na phase 2 study. The primary outcome is Number of Participants With 100 Day Transplant-related Mortality (TRM).

2. Clinical Question

The trial asked whether umbilical cord blood transplantation following reduced-intensity conditioning could be used in adults with hematologic malignancies, with the primary endpoint focused on transplant-related mortality within 100 days. The registry describes treatment with fludarabine, melphalan, and anti-thymocyte globulin alongside transplantation. Tacrolimus and mycophenolate mofetil are also listed among the interventions.

The registry’s rationale describes conditioning chemotherapy as intended to suppress cancer-cell growth and reduce immune rejection of donor cells. It also describes the possibility of graft-versus-tumor activity and graft-versus-host disease, with tacrolimus and mycophenolate mofetil listed in the treatment regimen. These statements describe the rationale, not findings from this trial. The posted record does not establish how often engraftment occurred, whether relapse was reduced, or whether survival improved. Its primary clinical question is an early safety outcome, and the absence of a control group limits what can be concluded about comparisons with other approaches.

3. Trial Design

This was a phase II, single-group trial with enrollment of 5 participants. The registry lists allocation as NA and masking as none. There is no randomized comparator arm described, so the study is not a like-for-like comparison of treatments. It was sponsored by Northside Hospital, Inc., started in 2006-06, and had primary completion in 2009-11. Its current registry status is terminated; the reason for termination is not reported in the registry record.

The intervention record includes umbilical cord blood transplantation, anti-thymocyte globulin, fludarabine phosphate, melphalan, tacrolimus, and mycophenolate mofetil. The official title describes transplantation of two partially matched umbilical cord blood units following reduced-intensity conditioning. The registry’s brief summary explains the intended rationale for conditioning and post-transplant immunosuppression, but does not provide a detailed protocol schedule or dose information. Those details are not reported in the registry record.

The primary outcome is the number of participants with transplant-related mortality within 100 days, defined as death within 100 days from transplant-related complications. The record lists six outcome measures in total and includes secondary outcomes concerning engraftment, donor and host chimerism, graft-versus-host disease, relapse, and survival. The status of each outcome and its measurement details should be read from the posted record rather than inferred from the study title.

Because the trial has a single group, its results can describe what was observed among enrolled participants, if outcome results are posted, but cannot directly estimate a treatment contrast against a concurrent control group. The registry lists no analysis records. A detailed statistical analysis plan, the intended analysis population, and the reason for termination are 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.

Before assignment: There were no group assignments for this study. It was a single arm cord blood transplant study.

Overall Study

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 100 Day Transplant-related Mortality (TRM), measured over 100 days.

The primary endpoint is the number of participants with 100-day transplant-related mortality. The registry defines this as death within 100 days from transplant-related complications. It is a count outcome: it asks how many participants experienced the event, rather than measuring a continuous change or time-to-event effect over the full follow-up period.

Early transplant-related mortality is used as a safety and treatment-delivery outcome because it captures deaths attributed to transplant complications during an explicitly defined early period. In general, a defined endpoint period helps make the outcome interpretable: the reader knows which deaths the measure is intended to include. Attribution also matters. The registry definition ties the endpoint to transplant-related complications, rather than to every death from any cause.

The endpoint name and definition are present, but the number of participants who met it is not reported in the registry record. The serious adverse-event figures elsewhere in the record are not a substitute for this endpoint: they are presented as adverse-event counts and do not establish the primary outcome result or its cause. Without a posted primary endpoint result, this record does not show the observed frequency of 100-day transplant-related mortality.

In general, an event count from a small single-group study can be described for the enrolled participants, while uncertainty around the underlying event rate may remain substantial. The registry does not report an estimate with a confidence interval for this endpoint. It also does not report further detail on how transplant-related cause of death was adjudicated.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes a phase II single-group study and names a count endpoint: the number of participants with transplant-related mortality by 100 days. It does not report a statistical analysis plan or analysis records. The points below explain general statistical concepts relevant to reading this type of record; they are not claims about methods used by the investigators unless the registry explicitly states them.

What does a count endpoint show?

A count records how many participants experienced an event in the defined group and period. For this endpoint, the event is death within 100 days from transplant-related complications. A count is directly understandable, but its meaning depends on the number of participants observed and on complete ascertainment. In general, an event count alone does not describe the uncertainty in the underlying risk. A proportion or interval estimate can provide additional context, but neither is reported for the primary endpoint in the registry record.

What can a single-group design estimate?

A single-group study can describe outcomes among the participants who received the intervention. It does not include a concurrent control group, so it cannot directly estimate the difference between the regimen and another treatment. In general, random allocation helps balance known and unknown factors between groups. This study is not recorded as randomized, and no control comparison is listed. Therefore, even a complete outcome count would be descriptive rather than a randomized estimate of comparative effect.

That distinction is important when interpreting outcomes after transplantation. General changes in health, participant characteristics, supportive care, and other influences can affect outcomes. In a single-group design, the study cannot separate these influences from the intervention through a concurrent randomized comparison. This is a design limitation, not evidence that a particular influence affected these participants.

How should uncertainty be considered?

A confidence interval is a general statistical tool for expressing the range of values compatible with an estimate under a specified method. It helps readers assess precision, rather than treating a point estimate as exact. The registry record does not report a confidence interval for the primary endpoint, and it does not identify an interval method. With enrollment of 5, the amount of information available for estimating an event frequency is limited in general. No interval should be reconstructed from the registry figures when the primary endpoint count itself is absent.

In general, an estimate’s precision depends on the amount of information and the pattern of observed events. Small samples can produce wide uncertainty, and small changes in observed counts can materially change an estimate. This is why the endpoint count, denominator, follow-up completeness, and an appropriate uncertainty measure are all useful. Here, the enrollment count is given, but the primary endpoint count and its uncertainty are not reported in the registry record.

What do the planning illustrations mean?

The page includes sample-size planning illustrations, which are distinct from trial results. The stated illustration framework uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; it considers the effect stated as a fraction of the observed effect. For time-to-event tables, the stated quantity is total events, while other tables express participants per arm. These are planning assumptions, not a description of this trial’s single-group enrollment or a record of an actual randomized comparison.

In general, a sample-size calculation asks how much information a hypothetical study might need under stated assumptions. It is not a measure of the probability that this completed study found a true effect. The illustrations are planning tools, not observed power. They should not be interpreted as evidence that the treatment worked, as proof that the study was adequately sized, or as an explanation of why enrollment was 5. The registry does not report a trial-specific sample-size justification, so one should not infer that these planning illustrations formed part of the protocol.

What is not available for a full analysis?

The registry lists 0 analyses. It does not report an analysis model, a prespecified statistical plan, an approach to missing outcomes, an analysis population, or a method for handling competing events or cause-of-death attribution beyond the endpoint definition. These omissions prevent a reader from assessing whether a posted estimate would be adjusted, how uncertainty would be calculated, or whether all enrolled participants were included in each outcome analysis. The absence of this information does not show that investigators did or did not use a particular method; those details are not reported in the registry record.

General statistical interpretation should keep the design, endpoint, and evidence distinct. The design is single-group, the endpoint is defined, and the primary outcome result is unavailable in the registry record. A planned calculation, an adverse-event count, or the biological rationale cannot be substituted for that missing result.

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("Umbilical Cord Blood Transplant")
n      <- c(5)
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  Umbilical Cord Blood Transplant  5  0  0.0  0.0  52.2  0.0  43.4  0.4  45.9

11. Multiplicity, Missing Data and Analysis Populations

The registry lists six outcome measures, including the primary 100-day transplant-related mortality outcome and secondary outcomes concerning engraftment, chimerism, graft-versus-host disease, relapse, and survival. When a study examines multiple outcomes, general statistical practice considers whether and how to account for multiplicity. Testing many outcomes can increase the chance of finding an apparently notable result by chance. The registry record does not report a multiplicity strategy, such as a hierarchy among outcomes or an adjustment procedure.

The number of analyses listed is 0. This means the posted record does not provide analysis entries to inspect; it does not establish which analyses were or were not conducted outside the registry. A prespecified analysis plan, any changes to that plan, and the handling of multiple endpoints are not reported in the registry record.

Missing-data methods and analysis populations are also not described. The record does not state how participants with incomplete follow-up would be handled, whether every enrolled participant contributed to each outcome, or how missing cause-of-death information would be treated. Those details matter in general because incomplete outcome ascertainment can affect both a count and its interpretation. The registry provides enrollment of 5 but does not report endpoint-specific denominators or follow-up completeness.

Without these details, readers cannot assess whether the outcome analyses were complete, whether the reported endpoints were analyzed as planned, or how robust any estimate would be to missing information. These are reporting gaps, not evidence that data were missing or that an analysis was inappropriate.

12. Safety Results

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

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent non-serious adverse event terms

The adverse-event record lists the Umbilical Cord Blood Transplantation group with 5 participants at risk for serious adverse events and 0 affected. It also lists 5 participants at risk for other adverse events and 5 affected. These are the posted group-level figures and should be read as counts, not as estimates of comparative safety. The registry does not list serious adverse-event terms in the top serious terms field.

The field for deaths is null, so a death count in this adverse-event section is not reported in the registry record. A blank or null entry should not be interpreted as a count of zero. Similarly, the serious-event count does not answer the primary endpoint question: transplant-related mortality by 100 days has its own endpoint definition, and its result is not reported in the registry record.

In general, adverse-event summaries depend on the reporting period, event definitions, attribution rules, and completeness of reporting. Those details are not fully. The record also has no comparator group, so these counts cannot show whether event frequency was higher or lower than it would have been under another treatment. Given enrollment of 5, the counts describe only the participants represented in this record and should not be generalized as precise estimates of safety.

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

This record documents a phase II, single-group investigation of umbilical cord blood transplantation with a conditioning regimen that includes fludarabine, melphalan, and anti-thymocyte globulin. It identifies transplant-related mortality within 100 days as the primary endpoint and gives a definition for that outcome. It also lists other outcomes that address engraftment, chimerism, graft-versus-host disease, relapse, and survival.

What the record does not show is the primary endpoint result. The number of participants with 100-day transplant-related mortality is not reported in the registry record, and there is no associated estimate of precision. The listed adverse-event counts are not a substitute for that result. Nor do the stated biological rationale and intervention list demonstrate that the regimen achieved its intended effects.

The single-group design means there is no concurrent randomized control comparison. In general, outcomes observed in a treated group cannot by themselves show what would have happened to similar participants under another treatment. Therefore, the record cannot establish a comparative effect, whether favorable or unfavorable. The status of terminated does not supply a clinical conclusion or explain why the trial ended; the reason is not reported in the registry record.

The posted information is useful for identifying the intended clinical question, the treatment components, the population, and the outcome framework. It is not sufficient to determine the trial’s observed primary outcome, quantify its uncertainty, or draw a causal conclusion about treatment effects. The sample-size planning illustrations are hypothetical planning material and not observed power or trial results. Readers should distinguish these design details from outcome evidence and avoid filling the missing result with assumptions.

14. Why This Trial Matters Statistically

This trial illustrates a basic distinction in clinical research: a clear clinical question does not guarantee that a registry record contains the results needed to answer it. Here, the question concerns early transplant-related mortality, and the registry defines the endpoint as death within 100 days from transplant-related complications. Yet the result for that endpoint is not reported in the registry record. Statistical interpretation must acknowledge that gap rather than infer an outcome from the study rationale or other fields.

The study also shows why design matters. A single-group trial can describe the experience of its enrolled participants, but it cannot provide a randomized comparison with an alternative treatment. This distinction remains important even when a study has a named primary endpoint and several clinically relevant secondary outcomes. A treatment effect is a comparative concept; without a suitable comparator, the record does not establish one.

Enrollment of 5 highlights the need to consider precision as well as the direction of any result. In general, estimates from small groups carry limited information, and a complete report should make the event count and uncertainty visible. In this record, the primary result and an interval estimate are absent, so readers cannot judge the endpoint’s observed frequency or precision.

Finally, the sample-size illustrations demonstrate why planning calculations must be kept separate from analysis results. They use stated assumptions for hypothetical planning and do not describe the design actually recorded. They are not observed power. Together, these points make the trial a useful example of how design, outcome reporting, and statistical uncertainty shape what can responsibly be concluded from a registry entry.

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 important limitation for interpretation is that the primary endpoint result is not reported in the registry record. Although the endpoint is defined, the record does not state how many participants experienced 100-day transplant-related mortality or provide a precision estimate. This prevents direct assessment of the trial’s primary outcome.

The trial is single-group, with allocation recorded as NA and masking recorded as none. There is no concurrent control group, so the design cannot estimate a randomized contrast against another treatment. Enrollment was 5, which limits the amount of information available for describing outcomes and makes generalization especially uncertain. These design features do not indicate the direction of any outcome.

The registry status is terminated, but the reason for termination is not reported in the registry record. The record also does not report a trial-specific sample-size justification, a detailed statistical analysis plan, an analysis population, missing-data methods, endpoint-specific denominators, or follow-up completeness. There are 0 analyses listed in the registry. Thus, readers cannot assess from this entry how analyses were specified or whether outcomes were fully observed.

The adverse-event information is limited to group-level counts and does not list serious-event terms. The deaths field is null, which is not the same as reporting zero deaths. Those adverse-event data do not supply the missing primary endpoint result. The listed secondary outcomes also do not provide evidence of their own results in the information. Overall, the registry supports a description of the study design and intended outcomes, but not a complete efficacy or safety assessment.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Umbilical Cord Blood (UCB) Transplant, Fludarabine, Melphalan, and Anti-thymocyte Globulin (ATG) in Treating Patients With Hematologic Cancer — NCT00827099.
• Registry record last updated 2012-03-23; status last verified 2012-03.
• Results first posted 2011-08-03.
• 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 phase II trial evaluated umbilical cord blood transplantation with fludarabine, melphalan, and anti-thymocyte globulin in participants with hematologic cancer. It was a single-group study with enrollment of 5, no masking, and allocation recorded as NA. The primary endpoint was the number of participants with transplant-related mortality within 100 days, defined as death within 100 days from transplant-related complications.

The registry lists secondary outcomes related to engraftment, chimerism, graft-versus-host disease, relapse, and survival. It reports 0 serious adverse events among 5 at risk and 5 other adverse events among 5 at risk; the deaths field is null. The primary endpoint result, the reason for termination, and a detailed statistical plan are not reported in the registry record. The study has no concurrent control group, and the record lists 0 analyses.