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 Infusion of Expanded Cord Blood Cells in Addition to Single Cord Blood Transplant in Treating Patients With Acute Leukemia, Chronic Myeloid Leukemia, or Myelodysplastic Syndromes (NCT03399773), a na phase 2 study. The primary outcome is Number of Participants With Graft Failure.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03399773 |
| Phase | Phase 2 |
| Condition | Acute Biphenotypic Leukemia, Acute Lymphoblastic Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 31 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Fred Hutchinson Cancer Center |
| Results posted | Yes |
2. Clinical Question
The study examines the use of off-the-shelf ex vivo expanded cryopreserved progenitor cells, identified as dilanubicel, in the setting of a single cord blood transplant. Participants have hematologic malignancies, including the listed leukemia and myelodysplastic syndrome conditions. The transplant approach also involves chemotherapy and total-body irradiation; the listed interventions include fludarabine, cyclophosphamide, thiotepa, and umbilical cord blood transplantation.
The primary clinical question is whether participants experience graft failure, defined in the registry record as primary graft failure or rejection by up to day 45 post-transplant. The definition covers no neutrophil recovery, regardless of donor chimerism, or autologous recovery: neutrophil recovery with < 10% donor chimerism in blood and bone marrow. This endpoint focuses on whether the transplant has achieved the specified early recovery and donor-cell criteria.
The registry record describes two treatment regimens, but the study is identified as a single-group trial, with allocation listed as not applicable. The regimens are not a like-for-like control comparison. Consequently, the record does not support interpreting any difference between regimen groups as a randomized treatment effect. The listed secondary outcomes include engraftment timing, graft-versus-host disease, non-relapse mortality, and adverse events.
Population
Participants with the listed acute and chronic leukemia conditions or myelodysplastic syndromes who undergo the study transplant approach. Participants who did not proceed to transplant were not analyzed for the primary endpoint.
Intervention
Single cord blood transplantation with ex vivo expanded cord blood progenitor cells, identified as dilanubicel, following the treatment regimen.
Comparator
No randomized control group is reported. Regimen A and regimen B are not like-for-like control arms.
Primary question
How many analyzed participants experience primary graft failure or rejection by up to day 45 post-transplant under the stated definition?
3. Trial Design
This is a phase II pilot study sponsored by Fred Hutchinson Cancer Center. The registry lists a single-group model and allocation as not applicable; masking is none. The title and study description concern adding expanded cord blood progenitor cells to single cord blood transplantation for participants with hematologic malignancies. Enrollment is listed as 31.
The treatment description identifies regimen A and regimen B. Regimen A is described in the adverse-event record as a high-dose regimen consisting of 1320 cGy total-body irradiation, fludarabine, and cyclophosphamide, followed by umbilical cord blood and dilanubicel. Regimen B is described as an intermediate-dose regimen consisting of 400 cGy total-body irradiation, fludarabine, cyclophosphamide, and thiotepa, followed by umbilical cord blood and dilanubicel. The detailed arm description is incomplete in the registry record, so further regimen procedures should not be inferred from it.
These treatment groups do not constitute a randomized comparison. The record explicitly cautions that the arms are not a like-for-like control comparison. Group differences could reflect factors other than the regimen, and the record does not establish a causal comparison between them. The study therefore primarily describes outcomes in participants receiving the study approach, rather than estimating a contrast against a concurrent control treatment.
The record lists a start date of 2022-05-10, a primary completion date of 2025-06-09, and a status of active, not recruiting. Those registry entries describe the study's recorded status and timeline; they do not establish that all outcomes have been collected or analyzed. The number of participants who proceeded to transplant, and therefore entered the primary endpoint analysis, is not reported in the registry record. The protocol's allocation rationale, sample-size justification, and detailed statistical analysis plan 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.
Overall Study
| Stage | Regimen A: High Dose Consisting of 1320 cGy TBI, Flu, Cy, Followed by UCB and Dilanubicel | Regimen B: Intermediate Dose Consisting of 400 cGy TBI, Flu, Cy, TT, Followed by UCB and Dilanubicel |
|---|---|---|
| STARTED | 16 | 15 |
| Study Treatment | 7 | 9 |
| COMPLETED | 6 | 7 |
| NOT COMPLETED | 10 | 8 |
Reasons for not completing this period:
| Reason | Regimen A: High Dose Consisting of 1320 cGy TBI, Flu, Cy, Followed by UCB and Dilanubicel | Regimen B: Intermediate Dose Consisting of 400 cGy TBI, Flu, Cy, TT, Followed by UCB and Dilanubicel |
|---|---|---|
| On Study Follow-Up (<2 years post-transplant) | 7 | 5 |
| Death | 0 | 1 |
| Relapse Requiring Alternative Therapy | 1 | 1 |
| Physician Decision | 2 | 1 |
Analysis population for the primary outcome, as defined in the registry: Participants who did not proceed to transplant on the study were not analyzed.
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 | Regimen A: High Dose Consisting of 1320 cGy TBI, Flu, Cy, Followed by UCB and Dilanubicel | Regimen B: Intermediate Dose Consisting of 400 cGy TBI, Flu, Cy, TT, Followed by UCB and Dilanubicel | Total |
|---|---|---|---|---|
| Participants analysed | 16 | 15 | 31 | |
| Age, Categorical: <=18 years | Participants | 3 | 1 | 4 |
| Age, Categorical: Between 18 and 65 years | Participants | 13 | 14 | 27 |
| Age, Categorical: >=65 years | Participants | 0 | 0 | 0 |
| Sex: Female, Male: Female | Participants | 7 | 8 | 15 |
| Sex: Female, Male: Male | Participants | 9 | 7 | 16 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 1 | 1 |
| Race (NIH/OMB): Asian | Participants | 2 | 0 | 2 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 1 | 0 | 1 |
| Race (NIH/OMB): Black or African American | Participants | 1 | 2 | 3 |
| Race (NIH/OMB): White | Participants | 10 | 8 | 18 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 2 | 4 | 6 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 6 | 4 | 10 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 10 | 11 | 21 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Disease Diagnosis: Acute Myeloid Leukemia (AML) | Participants | 7 | 9 | 16 |
| Disease Diagnosis: Biphenotypic Acute Leukemia (BAL) or Mixed-Lineage Leukemia (MPAL) | Participants | 1 | 0 | 1 |
| Disease Diagnosis: Acute Lymphoblastic Leukemia (ALL) | Participants | 7 | 5 | 12 |
| Disease Diagnosis: Myelodysplasia (MDS) | Participants | 0 | 1 | 1 |
| Disease Diagnosis: Chronic Myeloid Leukemia (CML) | Participants | 1 | 0 | 1 |
| Comorbidity Score: 0 | Participants | 7 | 4 | 11 |
| Comorbidity Score: 1 | Participants | 2 | 5 | 7 |
| Comorbidity Score: 2 | Participants | 1 | 1 | 2 |
| Comorbidity Score: 3 | Participants | 6 | 2 | 8 |
| Comorbidity Score: 4 | Participants | 0 | 2 | 2 |
| Comorbidity Score: 5 | Participants | 0 | 1 | 1 |
| Donor/graft characteristics: Fully matched, 6/6 | Participants | 0 | 0 | 0 |
| Donor/graft characteristics: Single mismatch, 5/6 | Participants | 2 | 4 | 6 |
| Donor/graft characteristics: Two mismatches, 4/6 | Participants | 14 | 11 | 25 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Graft Failure, measured over Up to day 45 post-transplant.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants with graft failure up to day 45 post-transplant. The registry defines primary graft failure or rejection as no neutrophil recovery, regardless of donor chimerism, or autologous recovery, meaning neutrophil recovery with < 10% donor chimerism in blood and bone marrow. This is a binary participant-level outcome: each analyzed participant either meets the definition or does not.
Neutrophil recovery and donor chimerism represent different parts of the endpoint definition. Requiring the definition to account for both helps distinguish a lack of neutrophil recovery from recovery that is not sufficiently donor-derived under the stated threshold. The endpoint is limited to the specified period after transplant, so it describes early graft outcomes rather than all later transplant outcomes.
Graft failure is used as a direct measure of whether the transplant meets the specified early recovery criteria. It is not interchangeable with the secondary outcomes, which include time to neutrophil engraftment, time to platelet engraftment, graft-versus-host disease, and non-relapse mortality. Those outcomes address other aspects of recovery and complications.
The record states that participants who did not proceed to transplant were not analyzed for this endpoint. It does not report how many participants were analyzed or the number who experienced graft failure. Thus, the endpoint definition and timeframe are available, but the primary outcome result is not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes the trial design and primary endpoint but does not report the primary graft-failure count. It also lists zero statistical analyses. As a result, the posted record does not establish how many analyzed participants met the graft-failure definition, nor does it provide an estimate of the frequency or precision of that outcome. No conclusion about the observed primary endpoint can be drawn from the registry record.
The enrollment is 31, while the primary endpoint population excludes participants who did not proceed to transplant. Since the number who proceeded is not reported, enrollment should not be treated as the primary analysis denominator. A count without its denominator would be difficult to interpret, and a denominator without a clear account of exclusions could also misrepresent the outcome population.
The study includes regimen A and regimen B, but its model is single-group and allocation is not applicable. The record cautions that these are not like-for-like control arms. Any descriptive difference between their outcomes would not, by itself, show that one regimen caused a better or worse result. The record does not report a randomized treatment contrast or statistical adjustment intended to make the groups comparable.
The adverse-event record lists participants at risk and affected in each regimen group. For serious events, the recorded counts are 8 of 16 in regimen A and 7 of 15 in regimen B. For other events, the counts are 16 of 16 and 14 of 15, respectively. These are descriptive counts from the registry record, not proof that the study product caused the events. The recorded death counts are 0 and 1, but the record does not give cause or attribution for those deaths.
A confidence interval, when reported, describes uncertainty around an estimated quantity under the assumptions of the analysis. It does not correct for non-random group assignment, incomplete outcome reporting, or limitations in the analyzed population. No confidence interval or statistical analysis for the primary endpoint is reported in the registry record. The available details therefore support description of the design, endpoint definition, and adverse-event counts, but not a quantitative estimate of the primary endpoint or a comparative efficacy claim.
9. Statistical Methods Explained
Statistical methods are useful only when the target outcome, analysis population, and comparison are clear. The registry defines a participant-level binary primary endpoint: graft failure is either recorded as present or absent under the stated definition and timeframe. For such an endpoint, a complete report would generally identify the number of participants meeting the definition and the number included in the analysis. Those primary result counts are not reported in the registry record.
What a binary outcome can describe
For a binary outcome, a count communicates how many participants experienced the event. A proportion places that count in relation to the analyzed population. These quantities answer different questions: a count alone describes the number of events, while a proportion describes how common the event was among the participants in the denominator. The appropriate denominator matters, particularly when some enrolled participants do not proceed to the treatment or are otherwise excluded from the analysis.
Here, the primary endpoint population excludes participants who did not proceed to transplant. The number of participants who proceeded is not reported in the registry record. The enrollment figure therefore cannot be assumed to be the endpoint denominator. The outcome definition also includes a specific post-transplant timeframe, so the result would apply to that window and definition rather than to all later outcomes.
Precision and confidence intervals
A confidence interval is a range of values compatible with the observed data under a specified statistical method and its assumptions. A narrow interval indicates greater statistical precision than a wide interval, though precision is distinct from whether an estimate is clinically important. Intervals can also help readers see whether the data are compatible with a range of plausible event frequencies. The registry record does not report a primary endpoint estimate or confidence interval, so its precision cannot be assessed.
In general, estimates from small study populations tend to have more uncertainty than estimates from larger populations when other features are comparable. That general principle does not supply an estimate for this study. The analyzed population size is not reported, and no primary endpoint count is available. The enrollment figure alone cannot resolve those gaps.
Why the design affects interpretation
A randomized comparison is designed to make treatment groups comparable, on average, with respect to factors that could affect outcomes. This study is listed as single-group, with allocation not applicable, and the registry cautions that regimen A and regimen B are not like-for-like control groups. Therefore, comparisons between those regimen groups are descriptive. They cannot be interpreted as the effect of assigning one regimen rather than the other without further design and analysis information.
In a non-randomized comparison, differences may reflect patient characteristics, clinical decisions, or other factors associated with group membership, as well as treatment. Statistical adjustment can sometimes address measured differences, but it cannot guarantee removal of unmeasured confounding. The registry record does not report an adjusted comparative analysis or its assumptions.
Over-dispersion and outcome type
Over-dispersion is a concern when the variability in count data is greater than a statistical model expects. It can make standard errors too small if the model assumes less variability than the data contain. This concept is relevant to some count and rate analyses, but the primary endpoint is specified as a count of participants with a binary event. The registry record does not report a model for the primary endpoint, a dispersion estimate, or an over-dispersion assessment. It would therefore be inappropriate to claim that over-dispersion was tested or handled.
Time-to-event outcomes, such as time to neutrophil or platelet engraftment, require methods that account for when events occur and how follow-up ends. The registry lists these as secondary outcomes but does not report analysis methods or results for them. General methods for censored time-to-event data cannot be assumed to have been used in this trial.
Planning illustrations are not observed power
The record describes planning illustrations based on a fraction of the observed effect, with a two-sided significance level of 0.05, equal allocation, and a normal approximation. It distinguishes participant counts per arm for most outcomes from a total event count for time-to-event outcomes. Such calculations are hypothetical planning quantities under stated assumptions. They are not the probability that this completed or ongoing study will find a statistically significant result, and they are not observed power.
The registry record does not report the trial's own sample-size justification or the effect estimate used to generate the illustrations. The primary endpoint result is also not reported, so readers cannot evaluate the observed effect from this record. The planning assumptions should not be mistaken for a protocol-defined analysis plan or evidence about the study's actual statistical power.
Finally, a reported p-value would address compatibility with a specified null model under the analysis assumptions; it would not establish causality in a non-randomized comparison or communicate the size and precision of an effect by itself. No primary endpoint p-value or analysis is reported in the registry record. The most defensible statistical reading is consequently a description of what was planned and defined, alongside a clear acknowledgment that the primary result and its uncertainty are unavailable in the record.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity arises when several outcomes, groups, timepoints, or statistical tests are examined. The more tests considered, the greater the chance that at least one result appears noteworthy by chance if each test is judged separately. Statistical plans may specify a primary outcome, testing hierarchy, or adjustment method to address this issue. The registry lists one primary outcome and multiple secondary outcomes, but multiplicity procedures and a detailed statistical analysis plan are not reported in the registry record. No adjustment or testing hierarchy should be assumed.
The record lists nine outcome measures and zero analyses. Secondary outcomes include serious adverse events related to study product, neutrophil and platelet engraftment timing, acute and chronic graft-versus-host disease, and non-relapse mortality. The outcome list alone does not reveal which analyses were completed, whether all planned measures were collected, or how any multiple comparisons were handled.
Missing-data methods are also not reported in the registry record. The primary endpoint population excludes participants who did not proceed to transplant, but the record does not provide their number or describe the handling of missing endpoint information among participants who proceeded. It does not report whether endpoint status was complete for all analyzed participants or how any unavailable observations were treated.
The registry's statement about the primary analysis population is important: the endpoint is not defined over everyone enrolled, but over those who proceeded to transplant. Without the size and disposition of that population, the extent and possible implications of exclusions cannot be assessed. No assumptions about imputation, censoring, or sensitivity analyses are justified by the record.
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 |
|---|---|---|---|
| Regimen A: High Dose Consisting of 1320 cGy TBI, Flu, Cy, Followed by UCB and Dilanubicel | 8 | 16 | 50.0% |
| Regimen B: Intermediate Dose Consisting of 400 cGy TBI, Flu, Cy, TT, Followed by UCB and Dilanubicel | 7 | 15 | 46.7% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Regimen A: High Dose Consisting of 1320 cGy TBI, Flu, Cy, Followed by UCB and Dilanubicel | 0 of 16 |
| Regimen B: Intermediate Dose Consisting of 400 cGy TBI, Flu, Cy, TT, Followed by UCB and Dilanubicel | 1 of 15 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Regimen A: High Dose Consisting of 1320 cGy TBI, Flu, Cy, Followed by UCB and Dilanubicel | 16 of 16 |
| Regimen B: Intermediate Dose Consisting of 400 cGy TBI, Flu, Cy, TT, Followed by UCB and Dilanubicel | 14 of 15 |
Most frequent serious adverse event terms
| Event term | Organ system | Regimen A: High Dose Consisting of 1320 cGy TBI, Flu, Cy, Followed by UCB and Dilanubicel | Regimen B: Intermediate Dose Consisting of 400 cGy TBI, Flu, Cy, TT, Followed by UCB and Dilanubicel |
|---|---|---|---|
| Fever | General disorders | 3 of 16 | 1 of 15 |
| Diarrhea | Gastrointestinal disorders | 1 of 16 | 1 of 15 |
| Disseminated adenovirus (ADV) | Infections and infestations | 1 of 16 | 1 of 15 |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | 2 of 16 | 0 of 15 |
| Abdominal pain | Renal and urinary disorders | 0 of 16 | 1 of 15 |
| Acute kidney injury | Renal and urinary disorders | 0 of 16 | 1 of 15 |
| Cardiac arrest | Cardiac disorders | 0 of 16 | 1 of 15 |
| Conjunctivitis | Infections and infestations | 0 of 16 | 1 of 15 |
| Ejection fraction decreased | Investigations | 0 of 16 | 1 of 15 |
| Encephalitis infection | Infections and infestations | 0 of 16 | 1 of 15 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Regimen A: High Dose Consisting of 1320 cGy TBI, Flu, Cy, Followed by UCB and Dilanubicel | Regimen B: Intermediate Dose Consisting of 400 cGy TBI, Flu, Cy, TT, Followed by UCB and Dilanubicel |
|---|---|---|---|
| Febrile neutropenia | Blood and lymphatic system disorders | 13 of 16 | 11 of 15 |
| Anorexia | Metabolism and nutrition disorders | 3 of 16 | 8 of 15 |
| Mucositis oral | Gastrointestinal disorders | 6 of 16 | 3 of 15 |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | 5 of 16 | 4 of 15 |
| Enterocolitis infectious | Infections and infestations | 3 of 16 | 5 of 15 |
| Abdominal pain | Gastrointestinal disorders | 3 of 16 | 2 of 15 |
| Sepsis | Infections and infestations | 3 of 16 | 2 of 15 |
| Urinary tract infection | Infections and infestations | 2 of 16 | 3 of 15 |
| Cytomegalovirus (CMV) infection reactivation | Infections and infestations | 3 of 16 | 1 of 15 |
| Acute kidney injury | Renal and urinary disorders | 0 of 16 | 3 of 15 |
Serious events with the highest arm-level rate: fever (18.8%), hypoxia (12.5%), abdominal pain (6.7%), acute kidney injury (6.7%), cardiac arrest (6.7%).
The adverse-event record separates serious and other events by regimen group. For serious events, 16 participants were at risk in regimen A and 8 were affected; 15 were at risk in regimen B and 7 were affected. For other events, the corresponding at-risk and affected counts are 16 and 16 for regimen A, and 15 and 14 for regimen B. These counts describe the registry entries; they do not by themselves establish that events were caused by dilanubicel or another study intervention.
The record lists serious event terms with percentages: fever, 18.8%; hypoxia, 12.5%; abdominal pain, 6.7%; acute kidney injury, 6.7%; cardiac arrest, 6.7%; and conjunctivitis, 6.7%. The percentages are reported as event-term figures; the record does not provide further denominator detail alongside this list. They should not be read as comparative risks between regimens.
Deaths are recorded as 0 in regimen A and 1 in regimen B. Cause, timing, and attribution are not reported in the registry record. The listed secondary outcome includes grade ≥3 adverse events related to study product, but the group counts should not be assumed to represent only events judged related to the study product. Safety interpretation requires keeping event seriousness, attribution, and the population at risk distinct.
12. What the Result Does, and Does Not, Mean
The record establishes that this is a phase II, single-group study of expanded cord blood progenitor cells used with single cord blood transplantation. It defines a primary endpoint of graft failure up to day 45 post-transplant and specifies which participants are excluded from that endpoint population. These details make the intended outcome and its scope understandable.
However, the primary graft-failure count and analysis are not reported in the registry record. The record therefore does not show how often the endpoint occurred, how precise an estimate would be, or whether the result is compatible with a broad range of event frequencies. The study's enrollment figure is not a substitute for the number analyzed because participants who did not proceed to transplant were excluded.
The registry identifies two regimens, but it does not describe a randomized control comparison. It explicitly cautions that the regimens are not like-for-like control arms. Their outcome counts, if considered, can describe the recorded groups but cannot establish that regimen differences caused any outcome differences.
The secondary outcome and adverse-event listings offer additional context about what the study tracks. They do not fill the gap in the primary result, and the safety figures alone do not establish causality. The record does not report detailed methods for missing data, multiplicity, or adjusted analyses.
In general, a study's result is most informative when the endpoint definition, analysis population, event count, denominator, and uncertainty are all reported. Here, the endpoint definition is available, but key result and analysis details are not. The record can therefore support a description of the study's question and design, but not a conclusion that the intervention does or does not produce a particular clinical outcome.
13. Why This Trial Matters Statistically
This trial is statistically notable because its primary endpoint is precisely defined while its primary result is absent from the registry record. A clear definition helps readers understand what would count as graft failure, including the timeframe and donor chimerism criterion. But without the number of participants analyzed and the event count, the endpoint cannot be quantified or evaluated for precision.
The distinction between enrollment and analysis population is especially important. The registry says participants who did not proceed to transplant were not analyzed for the primary endpoint. Readers therefore need the number who proceeded, along with a complete outcome count, to understand what population the result represents. That denominator is not reported in the registry record.
The study also illustrates why design labels matter. A single-group study with two regimen groups is not the same as a randomized comparison. The record explicitly warns against treating the regimens as like-for-like controls. Descriptive group figures should not be converted into causal claims simply because they appear side by side.
Finally, the record lists a range of secondary outcomes and adverse events, but does not report analyses or a detailed statistical plan. This makes it important to distinguish the outcomes a study intends to assess from outcomes for which results and uncertainty are actually available. The registry record provides useful design and endpoint context, while leaving the principal quantitative question unanswered.
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 main trial-specific limitation for interpretation is that the primary graft-failure result is not reported in the registry record. The number of participants who proceeded to transplant and entered that endpoint analysis is also not reported. Although enrollment is listed as 31, the enrolled population cannot be treated as the primary analysis population because participants who did not proceed to transplant were excluded.
The design is single-group, and the allocation field is listed as not applicable. Regimen A and regimen B are not identified as like-for-like control arms. Consequently, the record does not support causal comparisons between those regimens. The detailed description of the treatment arms is incomplete, further limiting assessment of how the regimens were implemented.
The registry reports zero analyses. It does not report a primary endpoint estimate, confidence interval, or p-value, nor does it give the statistical analysis plan, sample-size justification, multiplicity approach, or missing-data methods. The planning assumptions described in the record are not a substitute for those details or for outcome results.
The adverse-event record gives group counts and selected serious-event terms, but it does not provide all details needed to interpret event attribution, timing, or cause of death. The recorded death counts do not include cause or attribution. The event-term percentages are listed without further denominator detail in the record. These limits do not establish whether an event was related to study treatment.
Finally, the status is active, not recruiting, and the registry lists a primary completion date of 2025-06-09. Registry status and dates do not establish that all outcomes have been reported. Readers should distinguish the available record from a complete account of the trial's results.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Infusion of Expanded Cord Blood Cells in Addition to Single Cord Blood Transplant in Treating Patients With Acute Leukemia, Chronic Myeloid Leukemia, or Myelodysplastic Syndromes — NCT03399773.
- Registry record last updated 2026-07-10; status last verified 2026-06.
- Results first posted 2026-07-10.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
The registry describes a phase II pilot study of dilanubicel with single cord blood transplantation in participants with hematologic malignancies. It lists a single-group model, no masking, enrollment of 31, and two treatment regimens that are not like-for-like controls. The primary endpoint is the number of participants with graft failure up to day 45 post-transplant, using a definition based on neutrophil recovery and donor chimerism.
Participants who did not proceed to transplant were excluded from the primary endpoint analysis, but the analyzed population size is not reported. The primary graft-failure count and statistical analyses are not reported in the registry record. Adverse-event counts are listed by regimen, along with selected serious-event terms and death counts; attribution and other interpretive details are limited. The record is active, not recruiting, and gives a primary completion date of 2025-06-09.