This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Randomized HaploCord Blood Transplantation vs. Double Umbilical Cord Blood Transplantation for Hematologic Malignancies (NCT01745913), a randomized phase 2 study. The primary outcome is Rate of Neutrophil Engraftment After Combined Haplo-identical Cord With That of Umbilical Cord Blood Transplantation..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01745913 |
| Phase | Phase 2 |
| Condition | Acute Myelogenous Leukemia, Myelodysplastic Syndrome, Acute Lymphocytic Leukemia |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 2 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Weill Medical College of Cornell University |
| Results posted | Yes |
2. Clinical Question
Population
Patients with hematologic malignancies were eligible for the study. The registry lists acute myelogenous leukemia, myelodysplastic syndrome, acute lymphocytic leukemia, Hodgkin's lymphoma and non-Hodgkin's lymphoma as conditions of interest. The record reports enrollment of 2, but does not describe the enrolled participants' diagnoses or other baseline characteristics. The primary-outcome population is described as not evaluable: the registry says no patients completed the study, subject data were only evaluable up to month 3, and the primary-outcome population data were not collected because patients died.
Intervention
The investigational arm was haplo-identical cord transplantation, described in the record as Haplo-Cord SCT. The study summary also mentions reduced-intensity conditioning and lists CliniMACS CD34 Reagent System, fludarabine, melphalan and rabbit ATG among the interventions. The detailed regimen and the treatment actually received by each participant are not reported in the registry record.
Comparator
The comparison arm was double umbilical cord blood transplantation, described as UCB SCT. The study summary characterizes this as a procedure using cells from one or two umbilical cords and calls it the standard arm. The record describes cord-unit selection criteria, but does not provide participant-level treatment details or results that allow a comparison of outcomes between arms.
Primary question
The primary question was whether the rate of neutrophil engraftment differed between combined haplo-identical cord transplantation and umbilical cord blood transplantation. The stated timeframe was an estimation of engraftment rates over 24 months. The registry reports that the primary outcome was not evaluable, so it does not establish an outcome contrast between the randomized arms.
3. Trial Design
This was a randomized, parallel-group phase 2 study with no masking. It compared haplo-identical cord transplantation with double umbilical cord blood transplantation in patients with hematologic malignancies. The sponsor was Weill Medical College of Cornell University. The registry gives a start date of 2012-10-26, a primary completion date of 2015-04-29 and a terminated status.
Enrollment was 2. The record does not describe the sequence-generation method, allocation concealment, the reasons for termination, or whether the randomization resulted in the intended assignments. It also does not report the number assigned to each arm in a separate allocation summary. No assumptions about those details can be made from the parallel randomized designation alone.
The study summary states hypotheses about engraftment, graft-versus-host disease, infections, transfusion requirements, hospital stay and long-term outcomes. These are hypotheses, not reported findings. The registry lists secondary outcome domains including platelet recovery, transfusion requirements, transplant-related mortality, relapse, survival, progression-free survival, graft-versus-host disease and opportunistic infections. The primary outcome was neutrophil engraftment, but the record says it was not evaluable. The trial's small enrollment and lack of evaluable primary results prevent an informative comparison of the planned outcome domains.
Although randomized allocation can help balance prognostic factors in a sufficiently informative trial, randomization alone does not make an outcome comparison interpretable when outcome data are unavailable or too incomplete to evaluate. The registry record does not report a statistical analysis plan, a sample-size justification or methods for handling incomplete follow-up. It also does not report whether any analyses were conducted beyond the posted outcome records.
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
| Stage | Haplo-Cord SCT | UCB SCT |
|---|---|---|
| STARTED | 1 | 1 |
| COMPLETED | 0 | 0 |
| NOT COMPLETED | 1 | 1 |
Analysis population for the primary outcome, as defined in the registry: Data not collected. Patients died.
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.
| Characteristic | Unit | Haplo-Cord SCT | UCB SCT | Total |
|---|---|---|---|---|
| Participants analysed | 1 | 1 | 2 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 | 1 | 2 |
| Age, Categorical: >=65 years | Participants | 0 | 0 | 0 |
| Sex: Female, Male: Female | Participants | 0 | 1 | 1 |
| Sex: Female, Male: Male | Participants | 1 | 0 | 1 |
| Region of Enrollment: United States | participants | 1 | 1 | 2 |
6. Primary Endpoint
The registry lists the primary outcome as Rate of Neutrophil Engraftment After Combined Haplo-identical Cord With That of Umbilical Cord Blood Transplantation., measured over estimation of 24 months to determine engraftment rates for all subjects.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was the rate of neutrophil engraftment after transplantation. Neutrophil engraftment is a clinically relevant marker of blood-cell recovery after a transplant. As a general statistical concept, an engraftment rate expresses how often a defined recovery event is observed in a study population over a specified period. Comparing rates can describe whether the event was observed differently across treatment groups, but interpretation depends on a clear event definition, complete follow-up and enough observations to estimate the rates with useful precision.
The registry states that the intended timeframe was an estimation of engraftment rates over 24 months. It does not provide a numerical rate, a group-level estimate, a confidence interval or a test of the treatment contrast. Instead, it says no patients completed the study and were therefore inevaluable for this outcome; subject data were only evaluable up to month 3. The primary-outcome population data are described as not collected because patients died.
Consequently, the endpoint's clinical purpose can be described, but the posted record does not permit estimation of the rate or comparison of engraftment between the arms. The endpoint is not a substitute for later outcomes such as survival or infection, which were listed separately among the study's outcome domains.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry documents a randomized comparison, but it does not report a primary-outcome result that can answer the trial's central question. The study enrolled 2, was terminated, and the primary outcome is explicitly described as inevaluable. The record says no patients completed the study, with subject data only evaluable up to month 3, although the endpoint timeframe was intended to estimate engraftment over 24 months. No arm-specific engraftment rates, effect estimate, confidence interval or inferential test are reported.
This means the posted record does not quantify either the direction or magnitude of a difference in neutrophil engraftment. It also does not support a conclusion that the two transplant approaches had similar or different outcomes. A missing comparison is not evidence of no difference. In general, an effect estimate describes the observed contrast, while a confidence interval describes the range of values reasonably compatible with the data and analysis. Neither is available for the primary endpoint in the registry record.
The record's hypotheses concerning engraftment, graft-versus-host disease, infections, transfusion requirements, hospital stay and long-term outcomes should be kept distinct from results. The listed secondary outcome domains do not come with reported analyses in the registry record. There are no posted estimates to assess precision, and the record does not provide enough outcome data to judge the possible size or clinical importance of any effects.
The safety record contains counts for each arm: one participant at risk for each reported category, zero serious adverse events affecting participants in either arm, one other adverse event affecting a participant in each arm, and one death in each arm. These limited counts describe what the record lists; they do not establish that a treatment caused or prevented an event. With such sparse information and no accompanying event descriptions or attribution details, comparisons should not be treated as reliable estimates of relative safety.
The registry record does not report why the study was terminated, the circumstances surrounding the deaths, or a statistical analysis plan. It also does not report a primary-outcome analysis or the reasons individual participants became inevaluable. Those omissions limit the interpretation of both the study's conduct and its outcomes. The most defensible reading is that the registry describes the planned comparison and reports that the primary endpoint could not be evaluated, rather than reporting evidence for or against either treatment approach.
9. Statistical Methods Explained
Statistical methods help translate observed outcomes into estimates of treatment differences and quantify the uncertainty around those estimates. In a randomized comparison, the intended contrast is between outcomes in the assigned groups. Interpretation still depends on outcome definitions, follow-up, missing-data handling and a prespecified analysis. The registry record does not report a statistical analysis plan, and it lists no analyses. The explanations below describe general statistical concepts rather than methods confirmed to have been used in this trial.
What does a rate estimate show?
In general, a rate or proportion summarizes the frequency of an event in a defined group over a specified observation period. For this study, the planned event was neutrophil engraftment and the registry stated an assessment horizon of 24 months. To compare groups, the event needs a consistent definition and the record needs enough outcome observations in each group. A rate without a clear denominator or follow-up description can be difficult to interpret, because participants may have different opportunities for the event to be observed.
The registry does not report numerical engraftment rates or group-specific denominators for an evaluable endpoint analysis. It states that the outcome was not evaluable and that subject data were only evaluable up to month 3. Therefore, there is no reported rate contrast to interpret. It would not be appropriate to reconstruct one from the trial's enrollment or safety counts.
What does a confidence interval contribute?
As a general concept, a confidence interval communicates the uncertainty in an estimated effect. A narrow interval indicates greater precision under the assumptions of the analysis; a wide interval indicates that the data are compatible with a broader range of effects. The interval does not by itself establish whether an effect is clinically important, and it does not replace scrutiny of the study design or missingness. The registry record reports no confidence interval for the primary outcome.
A p-value, when used, summarizes how compatible the observed data are with a specified null hypothesis under a statistical model. It is not the probability that the null hypothesis is true, nor does it measure the size or importance of an effect. No primary-outcome p-value or test result is reported in this record. The record also does not report whether any inferential tests were planned or performed.
Why does incomplete follow-up matter?
For an outcome observed over time, incomplete follow-up can leave uncertainty about whether an event would have occurred later. If participants are not observed through the intended period, an analysis needs a clearly stated approach to censoring, competing events and missing outcome information. The appropriate approach depends on the endpoint and data collection. These are general statistical considerations; the registry does not describe an approach for this trial.
The record states that the primary endpoint was inevaluable, no patients completed the study, and data were evaluable only up to month 3. It also describes the primary-outcome population data as not collected because patients died. Death may prevent later observation of engraftment or otherwise affect the interpretation of the endpoint; without participant-level timing and a stated analysis method, the effect cannot be characterized from this record. No assumptions should be made about the reasons for missing data or how an analysis would have handled them.
What do the planning assumptions mean?
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 arms and a normal approximation. It says the effect to detect is expressed as a fraction of the observed effect. Such a calculation is a planning exercise: it asks how many participants per arm might be needed under stated assumptions to detect a target effect. It is not an observed trial result and is not observed power.
Here, no primary-outcome effect estimate is reported, and the primary comparison was not evaluable. As a result, no numerical planning illustration can be interpreted as a meaningful estimate of requirements for a future study from the registry record alone. A calculation based on an unstable or unavailable observed effect would not resolve the absence of primary endpoint data. For time-to-event outcomes, the record notes that planning calculations use the total number of events; other calculations use participants per arm. This distinction is general to the stated planning approach and does not constitute evidence that a corresponding analysis was conducted.
Why can over-dispersion matter?
As a general statistical concept, over-dispersion occurs when observed variation is greater than a model assumes. A model that underestimates variability can produce intervals that are too narrow and tests that appear more decisive than the data warrant. Whether over-dispersion is relevant depends on the outcome and the chosen model. The registry does not report a statistical model, an over-dispersion assessment or sufficient outcome detail to evaluate this issue for the trial.
Likewise, adjustment for baseline characteristics can sometimes improve precision or address prespecified prognostic factors, but adjusted results depend on the model and available data. No adjusted or unadjusted primary effect estimate is reported. There is therefore no basis for describing an adjusted analysis or comparing adjusted and unadjusted estimates.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 6 outcome measures and identifies several secondary domains: platelet recovery, transfusion requirements, transplant-related mortality, relapse, survival, progression-free survival, graft-versus-host disease and opportunistic infections. Testing or estimating many outcomes can raise multiplicity concerns. In general, multiplicity refers to the increased chance of a misleading positive finding when many statistical questions are examined. Prespecified priorities, adjustment procedures and transparent reporting can help readers understand how multiple comparisons were handled.
The registry record does not report a multiplicity strategy, a hierarchy among secondary outcomes or a statistical analysis plan. It lists no analyses. Since there are no reported outcome estimates or tests to assess, the potential effect of multiplicity cannot be evaluated. The hypotheses in the study summary are not analysis results and should not be treated as such.
Missing data are central to interpretation. The registry says no patients completed the study and were inevaluable for the primary outcome. It reports that subject data were evaluable only up to month 3 and that primary-outcome population data were not collected because patients died. The reasons for termination, participant-level follow-up, missingness patterns and any imputation or censoring methods are not reported in the registry record.
The analysis population is also not defined beyond these outcome notes. The record does not report whether analyses were planned by randomized assignment, treatment received, or another population definition. These distinctions matter in randomized trials because each analysis population answers a different question and may be affected differently by nonadherence or missing outcomes. Without the analysis plan and evaluable endpoint data, no population-specific treatment effect can be interpreted.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Haplo-Cord SCT | 0 | 1 | 0.0% |
| UCB SCT | 0 | 1 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Haplo-Cord SCT | 1 of 1 |
| UCB SCT | 1 of 1 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 1% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Haplo-Cord SCT | 1 of 1 |
| UCB SCT | 1 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Haplo-Cord SCT | UCB SCT |
|---|---|---|---|
| chills | General disorders | 0 of 1 | 1 of 1 |
| Hypomagnesemia | Metabolism and nutrition disorders | 0 of 1 | 1 of 1 |
| rigors | General disorders | 0 of 1 | 1 of 1 |
| nausea | Gastrointestinal disorders | 0 of 1 | 1 of 1 |
| insomnia | Psychiatric disorders | 0 of 1 | 1 of 1 |
| generalized muscle weakness | Musculoskeletal and connective tissue disorders | 0 of 0 | 1 of 1 |
| headache | Nervous system disorders | 0 of 1 | 1 of 1 |
| fever | General disorders | 0 of 1 | 1 of 1 |
| abdominal pain | Gastrointestinal disorders | 0 of 1 | 1 of 1 |
| abdominal cramping | Gastrointestinal disorders | 0 of 1 | 1 of 1 |
The registry's safety figures are sparse. In each arm, the record lists one participant at risk for serious adverse events and zero participants affected by a serious adverse event. It also lists one participant at risk for other adverse events and one participant affected in each arm. The death count is one in each arm. These are the counts as recorded; they are not estimates of comparative risk.
The record does not provide descriptions, timing, severity beyond the serious-versus-other categorization, or attribution of the events. It does not state whether the deaths were considered related to transplantation or study treatment. A death recorded alongside an adverse-event summary does not, by itself, demonstrate treatment causation. Similarly, zero serious adverse events in the listed data does not establish that serious harms were absent beyond the information captured in this limited record.
With only the listed at-risk counts, the safety summary is too limited to characterize event patterns or make a stable comparison between arms. The registry does not report additional contextual information, follow-up duration for the safety observations, or analyses of adverse events. These figures should be read as descriptive registry counts, not as evidence that one approach is safer.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a randomized phase 2 comparison of haplo-identical cord transplantation and double umbilical cord blood transplantation was conducted and later terminated. It reports enrollment of 2 and identifies neutrophil engraftment as the primary outcome, with a planned timeframe of 24 months. It also records that no patients completed the study and that the primary outcome was inevaluable, with subject data only evaluable up to month 3.
What the record does not establish is a treatment effect. No arm-specific engraftment results, effect estimate, confidence interval or statistical test are reported. The absence of an evaluable comparison does not mean that the treatments have equivalent outcomes, nor does it show that one is better. It means the primary question cannot be answered from the posted outcome data.
The hypotheses listed in the study summary concern possible differences in engraftment and other clinical outcomes. Hypotheses describe what investigators expected to examine; they are not findings. The secondary outcome domains likewise do not amount to results when no numerical analyses are reported. Safety counts should also be interpreted cautiously: the registry lists sparse event and death counts but does not provide attribution or enough contextual information for comparative conclusions.
General statistical interpretation depends on both the estimated effect and its precision. Here, neither is available for the primary endpoint. The registry also does not report why the trial ended, the reasons for incomplete follow-up or the statistical methods intended for analysis. Those gaps prevent a more specific account of how the study's results might have been analyzed or why the primary outcome could not be evaluated.
The appropriate conclusion is limited: the trial record describes a planned randomized comparison but does not provide evaluable primary-outcome evidence to resolve that comparison. This is a statement about the information reported in the registry, not a medical recommendation or a conclusion about the relative merits of either transplant approach.
13. Why This Trial Matters Statistically
This trial illustrates an important distinction between a randomized design and an informative result. Randomization specifies how a comparison is intended to be made, but it cannot compensate for a primary outcome that is not evaluable. For readers assessing clinical evidence, the availability and completeness of outcome data are as important as the planned design.
The primary endpoint was chosen to address engraftment, a recovery outcome relevant to transplantation. The registry's stated 24-month timeframe makes follow-up an essential part of interpreting the intended endpoint. Yet the record reports that subject data were only evaluable up to month 3 and that no patients completed the study. This gap between the planned endpoint timeframe and the reported evaluability is central to understanding why no primary comparison can be drawn.
The record also highlights the need to distinguish hypotheses, outcome listings and results. The study summary describes anticipated benefits across several clinical domains, and the registry lists secondary outcomes. Neither provides evidence of an observed treatment effect. Statistical conclusions require analyzable outcomes, transparent methods and an account of uncertainty.
Finally, the safety counts demonstrate why small, sparse summaries need context. Counts can be reported accurately while still being insufficient to establish differences between groups or the cause of events. The registry's terminated status and lack of a reported explanation further limit interpretation of the trial's course. Statistically, this study is informative chiefly as a reminder that a clear trial question does not guarantee that the available record can answer it.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The most consequential limitation is that the registry reports no evaluable primary outcome. Although neutrophil engraftment was the primary endpoint and the intended timeframe was 24 months, the record states that no patients completed the study and subject data were only evaluable up to month 3. It does not report group-specific endpoint counts, rates or estimates.
Enrollment was 2, leaving the record with very limited participant information. The registry does not provide baseline characteristics, participant-level outcomes, detailed follow-up or an account of how participants were assigned and treated. The reasons for termination are not reported in the registry record, so the circumstances of the study's ending cannot be assessed.
The record does not report the statistical analysis plan, a sample-size justification, an analysis population definition, handling of missing data, or a multiplicity approach. It also reports no analyses. The planning assumptions described in the record do not supply an interpretable effect estimate when the primary outcome itself is not evaluable.
Safety information is similarly limited. Counts are listed for serious and other adverse events and deaths, but event descriptions, timing, attribution and safety follow-up are not reported. The registry's safety summary therefore cannot support a robust comparison between arms.
These limitations apply to interpretation of the registry record; they do not establish why the trial was terminated or why outcome data were unavailable. Those explanations are not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Randomized HaploCord Blood Transplantation vs. Double Umbilical Cord Blood Transplantation for Hematologic Malignancies — NCT01745913.
- Registry record last updated 2018-07-11; status last verified 2018-05.
- Results first posted 2017-08-09.
- 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.
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18. Record Summary
NCT01745913 is a randomized, parallel-group, unmasked phase 2 study sponsored by Weill Medical College of Cornell University. It compared haplo-identical cord transplantation with double umbilical cord blood transplantation in patients with hematologic malignancies. The registry reports enrollment of 2, a start date of 2012-10-26, a primary completion date of 2015-04-29 and terminated status.
The primary outcome was the rate of neutrophil engraftment, with a stated assessment timeframe of 24 months. The record says no patients completed the study, the outcome was inevaluable and subject data were only evaluable up to month 3. It reports no primary-outcome analysis. Safety counts are limited, and the registry does not report the reason for termination or a statistical analysis plan. The posted record therefore describes the study question and its incomplete outcome information, but does not establish a comparative treatment effect.