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 Hematopoietic Stem Cell Transplantation (HSCT) Using CD34 Selected Mismatched Related Donor and One Umbilical Cord Unit (NCT01050946), a na phase 2 study. The primary outcome is The Primary Objective is to Estimate the Overall Survival, Separately in the Two Risk Strata..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01050946 |
| Phase | Phase 2 |
| Condition | Leukemia, Lymphocytic, Acute, Leukemia, Lymphocytic, Chronic, Leukemia, Myelocytic, Acute |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Medical College of Wisconsin |
| Results posted | Yes |
2. Clinical Question
Population
The study addressed patients considered candidates for stem cell transplantation who did not have an appropriate donor. The registry lists acute and chronic lymphocytic leukemia, acute and chronic myeloid leukemia, non-Hodgkin lymphoma, and Hodgkin lymphoma among the conditions. Only one participant enrolled, so the posted record cannot characterize a broader patient population or show how outcomes might differ across these diagnoses.
Intervention
The intervention was a combined haploidentical and cord blood transplant. Blood-forming cells from a partially HLA-matched related donor were collected along with cells from an umbilical cord unit. The intervention description specifies that the preconditioning regimen was at the treating physician’s discretion. The study summary describes chemotherapy, and sometimes total body irradiation, as components of conditioning intended to prepare the recipient for transplantation.
Comparator
There was no randomized or control group listed. This was a single-group study, with allocation recorded as not applicable. The registry therefore does not provide a like-for-like comparison with another transplant approach or with no transplant. Any interpretation concerns the enrolled participant and the posted record, not a comparative treatment effect.
Primary question
The primary objective was to estimate overall survival separately in two risk strata, with a three-year timeframe. The registry states that the sole enrolled participant died before that timeframe and that the outcome could not be assessed. Consequently, the record does not provide an estimate of overall survival for either stratum, or an estimate of how survival might compare between approaches.
3. Trial Design
This was a phase II study with a single-group design. The registry lists one participant enrolled and identifies the study status as terminated. Allocation was not applicable, and masking was none. These entries describe a study without a randomized assignment or blinded treatment allocation. The study began in 2009-07, with primary completion recorded as 2013-03; the registry record does not state a termination date.
The intervention was a haploidentical/cord transplant, combining cells from a partially HLA-matched related donor with an umbilical cord unit. The brief summary frames the approach as a possible transplant option for patients who lack an appropriate donor, and describes conditioning before the transplant. The arm description says the precondition regimen was at the treating physician’s discretion. The registry record does not give a standardized regimen, dose schedule, or detailed rules for choosing conditioning.
The primary objective was to estimate overall survival separately in two risk strata over three years. The registry does not report the risk-stratum definitions, their planned enrollment, or results for each stratum. It states that the participant died before the primary outcome’s timeframe and that the outcome could not be assessed. The record lists secondary outcomes concerning relapse, neutrophil and platelet engraftment, acute graft-versus-host disease, transplant-related mortality, and disease-free survival. It does not report analyses for these outcomes.
Because the study has no control group, it cannot directly estimate a comparative effect against another transplant strategy. The one-person enrollment also means that the record offers no meaningful basis for describing between-participant variability. The registry does not report a protocol-level statistical plan, sample-size justification, interim analyses, or details of the intended 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 | Haploidentical/Cord Transplant |
|---|---|
| STARTED | 1 |
| COMPLETED | 1 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Only one patient enrolled on study. Patient died prior to time frame of 3 years. It is not possible to assess this outcome measure.
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 | Haploidentical/Cord Transplant |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | years | 61 |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 0 |
6. Primary Endpoint
The registry lists the primary outcome as The Primary Objective is to Estimate the Overall Survival, Separately in the Two Risk Strata., measured over 3 years.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
Overall survival concerns whether a participant is alive over a stated follow-up period. The registry’s primary objective was to estimate this outcome separately in two risk strata, using a three-year timeframe. Overall survival is a broad endpoint: unlike an outcome focused on a particular complication or transplant milestone, it reflects survival regardless of the specific cause of death.
In general, a time-to-event analysis of overall survival uses the time from a defined starting point to death, while accounting for participants whose status is not observed through the full follow-up period. The registry describes the study’s timeframe but does not report the planned time origin, censoring rules, estimator, or analysis method. The primary endpoint’s description also does not specify how risk strata were defined.
The record states that the only participant enrolled died before the three-year timeframe, making it impossible to assess the stated outcome. It therefore contains no estimate of three-year overall survival for either stratum. The clinical importance of overall survival does not overcome the absence of an assessable estimate: the posted record cannot establish the survival experience that the study set out to describe.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The most important statistical feature of this record is the gap between the primary objective and the available evidence. The study aimed to estimate overall survival separately in two risk strata, but only one participant enrolled. The registry states that this participant died before the three-year timeframe and that the primary outcome could not be assessed. No stratum-specific survival estimate or uncertainty interval is reported. The record therefore does not answer the primary question.
This is not a comparative trial result. The design was single-group, allocation was not applicable, and there was no control arm. Even if an outcome estimate had been available, the design would not support a direct comparison with another transplant approach. With only one participant, the posted record also cannot describe the range of outcomes among a group of recipients or distinguish a typical outcome from an unusual one.
The registry lists seven outcome measures and describes several intended secondary outcomes, including relapse, engraftment, acute graft-versus-host disease, transplant-related mortality, and disease-free survival. It reports no analyses. The primary outcome’s stated timeframe cannot be evaluated, and the record does not give numerical estimates for the secondary outcomes. The outcome descriptions alone are not results.
The serious adverse event listing reports one participant at risk and one affected in the transplant group. It identifies Acute Respiratory Distress Syndrome, Acute Renal Injury, Confusion, and Death at 100.0% each. These figures describe the posted record for a group of one; they do not estimate event rates in a wider population. The registry also does not provide event timing, duration, attribution, or a detailed account of the clinical course.
In general, estimates from very small samples are highly limited: a single participant’s course can dominate the reported experience, and the data cannot establish how much outcomes vary across patients. That concern is especially acute when the primary outcome itself is reported as not assessable. The registry record supports a description of enrollment, design, and the posted event information, but it does not support a reliable estimate of survival, a treatment effect, or generalization to other patients.
9. Statistical Methods Explained
The registry identifies this as a single-group study with no masking and allocation recorded as not applicable. Those design features determine what statistical conclusions are possible. There is no randomized comparator, so there is no basis for estimating a between-group treatment contrast. The record also lists no analyses. A description of intended outcomes should not be mistaken for a completed statistical analysis.
What does an overall-survival estimate represent?
In general, overall survival is a time-to-event outcome: the event is death, and the analysis concerns the time from a defined starting point until that event. When participants have different lengths of follow-up, methods can account for people whose event status is not known through the full observation period. Such methods depend on clearly stated rules for the time origin, follow-up, and censoring. The registry gives a three-year timeframe but does not report these analysis details or an estimator. It says the sole participant died before that timeframe and that the outcome could not be assessed. No survival estimate or confidence interval is reported.
A confidence interval, in general, describes the uncertainty around an estimated quantity under the assumptions of the method used. It is not a guarantee that the true value lies within the interval, and it does not remedy a lack of usable outcome information. The registry record reports no confidence interval for the primary outcome. Given the stated inability to assess the outcome, there is no posted estimate to interpret for either risk stratum.
Why does the single-group design matter?
In general, random allocation to comparison groups can help balance measured and unmeasured prognostic factors, although it does not guarantee identical groups. This study did not use a randomized comparison. Its single-group design means the registry can describe the enrolled participant’s course but cannot isolate the effect of the transplant approach from other influences or compare that course with an alternative. The record says the precondition regimen was at the treating physician’s discretion, but does not provide details that would allow its contribution to be evaluated.
The enrollment of one participant also prevents meaningful estimation of variation between participants. A group-level estimate based on a single person would not show whether that person’s experience is common or atypical. This is distinct from a question of statistical power: the registry’s main limitation is that the stated primary outcome was not assessable, and there are no reported outcome analyses to evaluate.
What do the planning assumptions mean?
General sample-size calculations are planning tools. The registry’s planning description specifies a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It says the effect to detect is a stated fraction of an observed effect. For time-to-event calculations, the planning description uses a total number of events; other calculations are expressed as participants per arm.
Those assumptions describe a planning illustration, not this study’s observed design or results. The actual study is single-group, with one participant enrolled, rather than an equal-allocation, two-arm comparison. The registry record does not report a protocol sample-size justification or establish that the planning assumptions governed the study. Any resulting sample-size figures should therefore be read as hypothetical planning illustrations, not as evidence of observed power or a post hoc measure of whether the trial could detect an effect.
What can the adverse-event percentages establish?
In general, a percentage is easiest to interpret alongside its numerator, denominator, event definition, and observation period. Here, the registry lists one participant at risk and one affected in the transplant group, and lists four serious event terms at 100.0% each. With a group of one, the percentages describe that record and are not stable estimates of the likelihood of these events among other recipients. The registry does not report further details needed to evaluate event timing, duration, attribution, or whether events overlapped in time.
The registry does not report an inferential method, adjustment strategy, or uncertainty calculation for these outcomes. Those omissions mean that statistical precision cannot be assessed beyond recognizing the extremely limited enrollment and the explicit statement that the primary outcome could not be evaluated.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one primary objective and seven outcome measures. It does not report a multiplicity strategy, such as a rule for controlling the chance of false-positive findings when several outcomes are tested. Because the record reports no analyses, there are no posted outcome tests or p-values to interpret. The presence of several endpoint descriptions alone does not show that statistical testing took place.
Missing-data methods are not reported in the registry record. The primary outcome description says the sole participant died before the three-year timeframe and that the outcome could not be assessed. The record does not explain whether any other outcome data were missing, how incomplete follow-up was handled, or what rules would have applied to censoring.
Analysis populations are also not reported in the registry record. No intention-to-treat, per-protocol, or safety population is defined. With one enrolled participant, the record does not support an analysis of differences between such populations in any case. The registry does not describe exclusions, protocol deviations, or how participants would have been assigned to the stated risk strata.
In general, multiplicity rules, missing-data methods, and analysis-population definitions help clarify how results were generated and how uncertainty should be interpreted. Their absence prevents assessment of those aspects of the statistical plan. It should not be read as evidence that any particular method was or was not used outside what the registry reports.
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 |
|---|---|---|---|
| Haploidentical/Cord Transplant | 1 | 1 | 100.0% |
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 |
|---|---|
| Haploidentical/Cord Transplant | 1 of 1 |
Most frequent serious adverse event terms
| Event term | Organ system | Haploidentical/Cord Transplant |
|---|---|---|
| Acute Respiratory Distress Syndrome | Respiratory, thoracic and mediastinal disorders | 1 of 1 |
| Acute Renal Injury | Renal and urinary disorders | 1 of 1 |
| Confusion | Psychiatric disorders | 1 of 1 |
| Death | Infections and infestations | 1 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Haploidentical/Cord Transplant |
|---|---|---|
| Acute Respiratory Distress Syndrome | Respiratory, thoracic and mediastinal disorders | 1 of 1 |
| Acute Renal Injury | Renal and urinary disorders | 1 of 1 |
| Confusion | Psychiatric disorders | 1 of 1 |
| Death | Infections and infestations | 1 of 1 |
Serious events with the highest arm-level rate: acute respiratory distress syndrome (100.0%), acute renal injury (100.0%), confusion (100.0%), death (100.0%).
The serious adverse event record lists the Haploidentical/Cord Transplant group with one participant at risk and one affected. It lists Acute Respiratory Distress Syndrome, Acute Renal Injury, Confusion, and Death at 100.0% each. These are descriptions of the registry’s recorded events in a group of one, not estimates of how often such events occur in a larger population.
Percentages based on one participant are especially limited: each recorded event can account for the entire group’s percentage. The figures do not communicate event timing, duration, severity beyond the serious-event classification, relationship to the transplant, or whether one event preceded another. The registry record does not report those details. The death is also consistent with the primary outcome description, which states that the participant died before the three-year timeframe, but the record does not give the timing or cause of death in that outcome description.
The adverse-event listing can therefore be read as a report of what was recorded for the enrolled participant. It cannot establish a general safety profile or compare safety with another approach, since the study had no control group and enrolled only one participant.
12. What the Result Does, and Does Not, Mean
The record documents a phase II, single-group study of combined haploidentical and cord blood transplantation, with one participant enrolled and a terminated status. It also records a primary objective of estimating overall survival separately in two risk strata over three years. The registry states that the participant died before the specified timeframe and that the outcome could not be assessed. The central statistical meaning is that the intended survival estimate is unavailable.
This absence is not evidence that survival would have been favorable or unfavorable in a larger group, nor does it establish the effect of the transplant approach. There was no randomized comparator, and the record reports no outcome analyses. The study’s one-person enrollment cannot describe variability among recipients, support a reliable population estimate, or separate the contribution of the transplant from other aspects of care.
The serious adverse event listing reports four terms at 100.0% in a group of one. That is a description of the enrolled participant’s posted record, not a general rate. It does not establish that the same events would occur in other patients, and the registry does not report details such as timing or attribution.
The record also does not provide a detailed statistical plan, risk-stratum definitions, analysis populations, missing-data methods, or a protocol sample-size justification. These omissions limit evaluation of how the study was intended to estimate its outcomes. They do not warrant filling in a likely protocol or analysis method.
Overall, the registry supports a narrow account of study design, enrollment, stated objectives, and recorded events. It does not provide an assessable estimate of the primary endpoint or evidence for a comparative treatment effect. Any broader clinical or statistical conclusion would go beyond what the posted record establishes.
13. Why This Trial Matters Statistically
This study illustrates how an intended endpoint and an interpretable estimate are not the same thing. Overall survival was the primary objective, with a three-year timeframe and separate estimates planned for two risk strata. But the registry reports one enrolled participant, who died before that timeframe, and explicitly states that the outcome could not be assessed. The statistical question is therefore unresolved in the posted record.
The record also shows why design matters when interpreting results. A single-group study can describe what happened to its participants, but it cannot provide a randomized comparison. The enrollment of one participant sharply limits what can be learned about variation, uncertainty, and the experience of other patients. A percentage reported for a group of one can look precise while describing only that individual record.
The study’s planning description makes a separate statistical point. It describes assumptions for a two-arm planning illustration, while the registered study is single-group. Planning calculations are not observed results, and they do not substitute for a reported analysis or a trial’s own design justification. The registry does not report such a justification.
Finally, the record distinguishes endpoint listings from endpoint findings. It names relapse, engraftment, acute graft-versus-host disease, transplant-related mortality, and disease-free survival, but reports no analyses for them. For statistical interpretation, that difference is fundamental: an outcome’s inclusion in a registry does not itself establish that a result was collected, analyzed, or estimable. This record is a clear example of why conclusions must follow the reported data and their limitations.
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 primary limitation is that only one participant enrolled, and the registry says the primary overall-survival outcome could not be assessed because that participant died before the three-year timeframe. There is no reported survival estimate for either risk stratum. The record does not define those strata, report their enrollment, or present a risk-specific analysis.
The study was single-group, with no randomized comparator. It cannot directly compare the combined transplant approach with another strategy. The registry records the precondition regimen as being at the treating physician’s discretion, but does not provide enough detail to assess how variation in conditioning might relate to outcomes.
The record lists secondary outcomes but reports no analyses. It does not provide outcome-specific estimates, uncertainty intervals, or a detailed account of follow-up. Statistical methods for survival, censoring, missing data, and analysis populations are not reported in the registry record. A protocol sample-size justification and interim analyses are also not reported in the registry record.
Safety information is limited to the serious-event listing, which records one participant at risk and one affected, with four terms at 100.0%. The registry does not give event timing, duration, attribution, or additional clinical detail. These figures cannot characterize risk in a broader population.
The study status is listed as terminated, but the registry record does not report a termination date or explain the reason for termination. The record also does not establish whether the stated planning assumptions were part of the study’s original statistical design. These limitations prevent conclusions beyond the narrow description of the posted record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Hematopoietic Stem Cell Transplantation (HSCT) Using CD34 Selected Mismatched Related Donor and One Umbilical Cord Unit — NCT01050946.
- Registry record last updated 2015-11-20; status last verified 2015-10.
- Results first posted 2015-09-25.
- 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 describes a phase II study of combined haploidentical and cord blood transplantation for patients considered transplant candidates without an appropriate donor. It was single-group, unmasked, and had allocation recorded as not applicable. The intervention’s preconditioning regimen was at the treating physician’s discretion.
One participant enrolled, and the study status is terminated. The primary objective was to estimate overall survival separately in two risk strata over three years. The registry states that the participant died before the timeframe and that the outcome could not be assessed. It lists secondary outcomes but reports no analyses.
The serious adverse event listing records one participant at risk and one affected, with Acute Respiratory Distress Syndrome, Acute Renal Injury, Confusion, and Death each listed at 100.0%. These figures describe the single-participant record and do not establish event rates in a wider population. The registry does not report a detailed statistical plan, analysis populations, or a protocol sample-size justification.