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 Donor Umbilical Cord Blood Transplant By Injection Into the Bone Marrow in Treating Patients With Hematologic Cancer (NCT00295880), a na phase 1, phase 2 study. The primary outcome is Median Number of Days to Neutrophil Engraftment.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00295880 |
| Phase | Phase 1, Phase 2 |
| Condition | Myeloproliferative Disorders, Leukemia, Lymphoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 12 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Masonic Cancer Center, University of Minnesota |
| Results posted | Yes |
2. Clinical Question
The study asks whether donor umbilical cord blood transplantation administered directly into the bone marrow can be used in patients with hematologic cancer, with attention to treatment-related effects and neutrophil recovery. The record describes a phase I/II study and lists myeloproliferative disorders, leukemia, lymphoma, and myelodysplastic syndromes among the conditions.
Population
Patients with hematologic cancer covered by the listed conditions: myeloproliferative disorders, leukemia, lymphoma, and myelodysplastic syndromes. Enrollment was 12. The record does not report further eligibility details or the characteristics of the participants enrolled.
Intervention
Umbilical cord blood transplantation. The title and intervention description specify administration by injection into bone marrow. The primary endpoint description, however, refers to two umbilical cord blood units administered i.v.; the registry record does not explain this difference in wording.
Comparator
There was no separate comparator group. The study is recorded as single-group, with allocation listed as not applicable and masking listed as none. The transplant patients are not a control group against which a treatment effect can be estimated.
Primary question
The primary endpoint is the median number of days to neutrophil engraftment. Neutrophil recovery is defined as the first of 3 consecutive days with ANC at least 500/ul, assessed daily through Day 60 post transplant. The registry record does not report the observed median or range.
Secondary outcomes listed include neutrophil recovery, evidence of engraftment, acute and chronic graft-versus-host disease, transplant-related mortality, and survival at Day 100 and 1 Year. These outcomes indicate the areas the study intended to describe; their results are not included in the registry record. The clinical question is therefore about the experience of a small, single-group transplant cohort, not a comparative estimate of benefit or harm.
3. Trial Design
This was a phase I/II, single-group study of umbilical cord blood transplantation. The registry lists an enrollment of 12 and one arm, Transplant Patients, described as patients receiving umbilical cord blood transplantation. Allocation is recorded as not applicable, and masking as none. There is no randomized or concurrent control group identified in the registry record.
The study concerns patients with hematologic cancers, with myeloproliferative disorders, leukemia, lymphoma, and myelodysplastic syndromes listed as conditions. The brief summary describes chemotherapy and total-body irradiation before transplantation as part of the treatment rationale. It says the trial was studying side effects and how well the approach works, but further details of the conditioning regimen, eligibility criteria, transplant procedures, or participant selection are not reported in the registry record.
The listed intervention is umbilical cord blood transplantation, and the study title specifies injection into bone marrow. There is a wording difference in the primary endpoint description, which refers to recipients of two cord blood units administered i.v. The record does not clarify how these descriptions relate to one another. It also does not report whether the intervention or procedures changed during the study.
The study started in 2005-06, had primary completion in 2008-08, and is recorded as terminated. The reason for termination is not reported in the registry record. These dates and status do not establish how many participants completed follow-up or why recruitment or study activity ended.
Without a comparator group, the design can describe outcomes among the enrolled participants, but it cannot separate effects associated with the transplant approach from other aspects of care, participant characteristics, or the underlying conditions. The registry record does not report a protocol-level statistical plan, a sample-size justification, or details of analysis populations.
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 | Umbilical Cord Blood Transplant Patients |
|---|---|
| STARTED | 12 |
| COMPLETED | 10 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | Umbilical Cord Blood Transplant Patients |
|---|---|
| IBMI unit could not be volume reduced | 1 |
| Unit randomization did not occur | 1 |
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 | Umbilical Cord Blood Transplant Patients |
|---|---|---|
| Participants analysed | 12 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 12 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | years | 34 (SD 8) |
| Sex: Female, Male: Female | Participants | 5 |
| Sex: Female, Male: Male | Participants | 7 |
| Region of Enrollment: United States | participants | 12 |
6. Primary Endpoint
The registry lists the primary outcome as Median Number of Days to Neutrophil Engraftment, measured over Daily through Day 60 post transplant.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Full Range |
| Unit | Days |
| Endpoint classification | Time-to-Event Analysis |
The primary endpoint is the median number of days to neutrophil engraftment. The record defines neutrophil recovery as the first of 3 consecutive days on which ANC is at least 500/ul, with assessment daily through Day 60 post transplant. This is a time-to-event measure: the event is reaching the specified recovery threshold, and the measured time is the number of days from transplantation to that event.
As a general statistical concept, a median time describes the middle of the distribution of times. It can summarize when recovery occurred in a group without requiring an assumption that times follow a symmetric distribution. The accompanying full range, when reported, would describe the lowest and highest observed times; it can be strongly affected by unusually early or late observations and does not show how densely observations fall between those endpoints.
Neutrophil recovery is used as a clinical and biological marker of blood-cell recovery after transplantation. It is a defined outcome rather than a complete measure of transplant success or overall health. The registry also lists separate outcomes for evidence of engraftment, graft-versus-host disease, transplant-related mortality, and survival, reflecting that these are distinct aspects of the post-transplant course.
No observed median, range, or participant-level endpoint result is reported in the registry record. The primary endpoint description's reference to intravenous administration also differs from the title's description of intra-bone marrow injection, and the record does not resolve that distinction.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a primary endpoint and a cohort, but it does not provide the observed median time to neutrophil engraftment or its full range. Consequently, the posted record does not support a numerical description of how quickly neutrophil recovery occurred in this cohort, nor how variable the observed times were. Naming an endpoint is not the same as reporting its result.
Enrollment was 12 in a single-group study. Without a separate comparator group, an observed outcome in this cohort would be descriptive. It could not, by itself, establish what would have happened to similar patients under a different transplant procedure or other care. Differences in patients, disease, clinical management, or follow-up could also be relevant, and the registry record does not provide detail to assess them.
The study is listed as terminated, but the reason is not reported in the registry record. Termination alone does not show whether the study stopped for safety, feasibility, recruitment, administrative reasons, or another cause. The record lists 8 outcome measures and 0 analyses. It names several planned secondary outcomes, but their numerical results are not reported in the record.
The safety information needs to be read separately from the efficacy endpoint. The adverse-event record lists 12 participants at risk for serious events and 5 affected. It also lists 0 affected by other events in the corresponding group fields. The top serious-event terms include Death at 41.7%, Acute GVHD Grade 3 at 16.7%, and graft failure at 8.3%. The record gives no deaths value in the group summary. These entries do not establish that events were caused by the intervention, and the listed Death term cannot be treated as a complete accounting of deaths without further detail.
Statistically, the central limitation is not merely imprecision from a small cohort: the outcome estimates themselves are absent from the registry record. There is therefore no primary effect estimate or interval to interpret. Even if descriptive results were available, a single-group design would not provide a direct comparative treatment effect. The posted information can establish the study's stated question, design, and selected safety entries, but not a conclusion about comparative efficacy or a causal safety profile.
9. Statistical Methods Explained
Time-to-event endpoint. The primary outcome is framed as time from transplant to a defined recovery event. In general, a time-to-event analysis measures both whether an event occurred and when it occurred. Here, the event definition is the first of 3 consecutive days with ANC at least 500/ul, and the stated assessment period is daily through Day 60 post transplant. The registry record identifies the summary statistic as a median with a full range, but does not report the values or explain the method used to estimate the median.
A median is a measure of location: it describes the central point of a distribution of event times. It is often useful when event times are not well represented by a mean, which can be pulled by long or short observations. A full range gives the extremes, not the shape of the distribution or the precision of the median. General statistical interpretation would also consider whether some participants had not experienced recovery by the end of observation. The registry record does not report whether that occurred or how such observations were handled.
For time-to-event data, some participants may have incomplete event-time information because observation ends before the event occurs. General methods can account for this through censoring, but no censoring counts or handling method are reported in the registry record. It is therefore not possible to determine from the record whether the reported endpoint, if any, would be a simple median among observed recoveries or an estimate accounting for incomplete follow-up.
Single-group design. The study is recorded as single-group, with allocation not applicable and no masking. In general, a single-group study can describe outcomes in the participants treated, but it lacks a concurrent control for estimating a contrast between treatment strategies. A before-and-after or external comparison would require information not reported in the registry record. No sponsor-reported hazard ratios are reported in the registry record, and there are 0 analyses listed.
Multiplicity and multiple outcomes. The record identifies 8 outcome measures and lists secondary outcomes covering recovery, engraftment, graft-versus-host disease, transplant-related mortality, and survival. When several outcomes or statistical tests are considered, the chance of at least one apparently unusual result can increase. General approaches to multiplicity include prespecifying a primary endpoint, allocating significance thresholds, or treating secondary findings as descriptive. The registry record does not report a multiplicity strategy, hypothesis-testing plan, or analysis results for the listed secondary outcomes. It is therefore not possible to tell whether any planned adjustment was used.
Sample-size planning. The record includes assumptions for a planning illustration based on an observed effect: a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It describes the effect to detect as a stated fraction of the observed effect. Those are planning assumptions, not evidence of achieved precision or an observed treatment effect. The record does not report the trial's own sample-size justification. In general, planning estimates depend on the assumed effect and statistical model; they should not be read as observed power. The study itself is single-group, so a hypothetical equal-allocation comparison does not describe its enrolled design.
The sample-size note distinguishes time-to-event calculations, which use a total number of events, from other calculations, which use participants per arm. These are general planning conventions, not outcome findings. Since the registry record does not provide the observed primary endpoint result or an observed comparison, no clinically meaningful effect size can be assessed from those assumptions alone.
Uncertainty and precision. A confidence interval, in general, represents a range of values compatible with a statistical estimate under its model and assumptions. It helps readers assess precision rather than treating an estimate as exact. No confidence interval for the primary endpoint is reported in the registry record. With enrollment of 12, any cohort-level description would need to be interpreted in light of the limited amount of information, but a numerical precision statement cannot be reconstructed without the actual endpoint data and analysis method.
Safety summaries. Adverse-event summaries count participants at risk and affected and list serious event terms. General safety interpretation depends on the definitions, observation period, event attribution, and whether a participant could contribute more than one event. Those details are not reported in the registry record. The listed group totals and terms should therefore be read as registry entries, not as a complete causal analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 8 outcome measures, including the primary neutrophil-engraftment endpoint and secondary outcomes concerning engraftment, graft-versus-host disease, transplant-related mortality, and survival. Considering several outcomes can raise a multiplicity issue: when many comparisons are tested, the chance of at least one result appearing statistically notable can increase. General methods to address this include defining a primary endpoint in advance, adjusting testing thresholds, or treating secondary outcomes as exploratory. The registry record does not report how multiplicity was handled, whether formal hypothesis tests were planned, or whether any adjustment was applied.
The record lists 0 analyses and provides no numerical primary endpoint result. It also does not report results for the named secondary outcomes. Thus, there are no posted inferential results from which to assess the role of multiple testing or distinguish confirmatory from exploratory findings.
Missing-data methods and analysis populations are not reported in the registry record. It is not stated whether all enrolled participants were included in each endpoint analysis, how withdrawals or incomplete follow-up were handled, or whether any participants were excluded from an analysis. For the time-to-event endpoint, the record does not say whether any observations were censored or how censoring was incorporated. The group summary reports 12 at risk for serious adverse events, but this does not establish the analysis population for the primary endpoint or other outcomes.
The registry also does not report a detailed statistical analysis plan, interim analyses, or the handling of protocol deviations. These omissions limit reproducibility and make it difficult to judge whether outcome definitions and analyses were settled before results were known. They should not be filled in by assumption.
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 |
|---|---|---|---|
| Umbilical Cord Blood Transplant Patients | 5 | 12 | 41.7% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Umbilical Cord Blood Transplant Patients | 0 of 12 |
Most frequent serious adverse event terms
| Event term | Organ system | Umbilical Cord Blood Transplant Patients |
|---|---|---|
| Death | General disorders | 5 of 12 |
| Acute GVHD Grade 3 | Blood and lymphatic system disorders | 2 of 12 |
| graft failure | Blood and lymphatic system disorders | 1 of 12 |
Serious events with the highest arm-level rate: death (41.7%), acute gvhd grade 3 (16.7%), graft failure (8.3%).
The serious-adverse-event group summary lists 12 participants at risk and 5 affected. Its corresponding other-event fields list 12 at risk and 0 affected. The top serious-event terms are Death at 41.7%, Acute GVHD Grade 3 at 16.7%, and graft failure at 8.3%. These figures are registry entries and should be read in the context of the information the record actually supplies.
The percentages are listed for event terms, while the record does not provide event counts for each term or explain whether participants could contribute more than one event. The group summary gives the deaths field as null, so it does not provide a usable death count. The Death term in the serious-event list should not be treated as a substitute for that missing count or as proof that a death was caused by the transplant.
The registry record does not report event timing, duration, severity details beyond the named grade, relatedness assessments, or the period over which events were collected. Nor does it provide a complete account of how serious events were defined. These omissions prevent a fuller safety interpretation. The figures describe what is entered in the registry; they do not by themselves establish risk relative to another treatment or attribute an event to the intervention.
12. What the Result Does, and Does Not, Mean
The registry record describes a phase I/II, single-group study of umbilical cord blood transplantation in patients with hematologic cancer. It identifies neutrophil engraftment as the primary endpoint and defines the recovery threshold and observation schedule. It also lists secondary outcomes and selected serious adverse-event information. These details show what the study set out to examine and what safety terms appear in the record.
The record does not report the median time to neutrophil engraftment, its full range, or numerical findings for the listed secondary outcomes. As a result, the posted information cannot establish how often or how quickly neutrophil recovery occurred, or how the cohort fared on the other named outcomes. It also does not report a comparative estimate, confidence interval, or hazard ratio.
Because the design has no separate comparator group, the outcomes of enrolled participants cannot by themselves identify what would have occurred under another treatment approach. The absence of a comparison is a design limitation, not evidence for or against an effect. The record's status of terminated does not explain why the study ended; the reason is not reported in the registry record.
The serious-event entries provide some descriptive safety information, including 5 affected among 12 at risk for serious events and the listed serious-event terms. But the record does not provide enough detail to establish causation, event timing, or a comparative safety profile. The Death term and the null deaths field should be kept distinct.
In short, the registry record supports a description of the study's design, intended endpoint, and selected safety entries. It does not support a conclusion about whether this transplant approach improved outcomes, how it compared with an alternative, or the size and precision of any treatment effect. Those questions require results and methodological detail that are not reported in the registry record.
13. Why This Trial Matters Statistically
This study is statistically notable for the gap between a clearly named time-to-event endpoint and the absence of its numerical result in the registry record. The endpoint definition is specific enough to identify the event—neutrophil recovery at the stated ANC threshold—but the median and range needed to describe observed recovery are not reported. This illustrates why outcome definitions and outcome estimates serve different purposes.
The single-group design also makes the scope of inference important. A cohort can provide descriptive information about participants who received an intervention, but without a comparator it cannot estimate a treatment contrast. This distinction matters especially when a study aims both to examine side effects and to assess how well an approach works: descriptive outcomes alone do not establish what would have happened under another approach.
The record names several clinically distinct secondary outcomes, but does not give their results or describe how multiple outcomes were handled statistically. When many outcomes are considered, a prespecified analysis strategy helps readers understand which findings are central and how uncertainty should be interpreted. The absence of that strategy in the registry record limits the ability to evaluate the intended inferential framework.
There is also a difference between planning assumptions and observed evidence. The record describes a sample-size planning illustration, but those assumptions do not replace a trial-specific justification or outcome estimates. Planning calculations are not observed power, and they cannot resolve the absence of a reported endpoint result.
Finally, the intervention descriptions are not fully aligned: the title refers to injection into bone marrow, while the primary endpoint description says the two units were administered i.v. The registry record does not explain the difference. Recognizing this kind of ambiguity is part of statistical interpretation, because it affects what intervention the endpoint description can be understood to represent.
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.
Several limitations are specific to the information recorded for this study. Enrollment was 12, and the design was single-group with no comparator. This permits a cohort description but does not support a controlled estimate of treatment differences. The record does not report participant characteristics, eligibility criteria, or details needed to assess how representative the enrolled group was of the listed conditions.
The primary endpoint result is absent. Although the record specifies a median, full range, event definition, and observation schedule, it reports no numerical median or range. Results for the listed secondary outcomes are also not reported. This makes it impossible to assess the distribution of recovery times, the observed frequency of other outcomes, or the statistical precision of estimates.
The registry's intervention wording is inconsistent: the title describes transplantation by injection into bone marrow, while the primary endpoint description refers to two units administered i.v. The record does not clarify whether these refer to different aspects of the procedure or reflect a documentation issue. Treatment details and the reason for termination are also not reported in the registry record.
For safety, the record provides a group summary and top serious-event terms but does not describe event timing, attribution, recurrence, or collection period. The deaths field is null, despite Death appearing as a serious-event term. The information therefore does not provide a complete accounting of mortality or a causal assessment.
Finally, no detailed analysis plan, missing-data approach, analysis populations, interim analyses, or multiplicity strategy is reported. These gaps prevent a full assessment of how outcomes were analyzed and how uncertainty should be interpreted.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Donor Umbilical Cord Blood Transplant By Injection Into the Bone Marrow in Treating Patients With Hematologic Cancer — NCT00295880.
- Registry record last updated 2017-12-28; status last verified 2017-12.
- Results first posted 2009-09-03.
- 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.
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18. Record Summary
The registry record describes a phase I/II, single-group study of umbilical cord blood transplantation in patients with hematologic cancer. Enrollment was 12; allocation was not applicable and masking was none. The study started in 2005-06, had primary completion in 2008-08, and is listed as terminated. A reason for termination is not reported in the registry record.
The primary endpoint was median days to neutrophil engraftment, defined as the first of 3 consecutive days with ANC at least 500/ul and assessed daily through Day 60 post transplant. The observed median and full range are not reported. The registry lists 8 outcome measures and 0 analyses. It also lists serious-event information, including 5 affected among 12 at risk and selected serious-event terms, while the deaths field is null. No comparator group or numerical primary outcome result is reported.