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 Haploidentical Allogeneic Transplant With Post-transplant Infusion of Regulatory T-cells (NCT01050764), a na phase 1, phase 2 study. The primary outcome is Maximum-tolerated Dose (MTD) of Regulatory and Conventional T-cells.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01050764 |
| Phase | Phase 1, Phase 2 |
| Condition | Leukemia, Acute, Chronic Myelogenous Leukemia (CML), Myelodysplastic Syndrome (MDS) |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 10 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Everett Meyer |
| Results posted | Yes |
2. Clinical Question
Population
Patients with hematologic malignancies undergoing myeloablative hematopoietic cell transplantation from haploidentical-related donors were the intended population. The listed conditions include acute leukemia, chronic myelogenous leukemia, myelodysplastic syndrome, non-Hodgkin lymphoma, and chronic lymphocytic leukemia. The primary-endpoint population is described as participants who received HSCT.
Intervention
The study evaluated post-transplant infusion of allogeneic regulatory T-cells and conventional T-cells following myeloablative treatment and stem cell rescue. The listed interventions also include melphalan, thiotepa, fludarabine, rabbit anti-thymocyte globulin, and the CliniMACS CD34 Reagent System. The registry describes a T-reg cell infusion after allogeneic stem cell transplant group.
Comparator
No concurrent comparator group is described. The record identifies a single-group model and no allocation method. Accordingly, the study is not a randomized comparison, and the posted information does not support a like-for-like comparison of study arms.
Primary question
The primary question was what maximum-tolerated dose of regulatory and conventional T-cells could be identified from safety and feasibility across a pre-determined set of cellular dose-level combinations. The assessment was planned for 30 days after HSCT infusion. The registry record does not report the dose combinations, the observed endpoint result, or a selected maximum-tolerated dose.
The trial was registered as phase 1 and phase 2, with an enrollment of 10 and a status of terminated. The clinical question is therefore one of early feasibility and dose finding, rather than a comparison of treatment effects against a control group. The record also lists secondary outcomes concerning acute and chronic graft-versus-host disease, overall survival, and serious infections. It does not provide analyses for those outcomes.
3. Trial Design
This was a single-group phase 1 and phase 2 study of post-transplant allogeneic T-cell infusion in patients undergoing myeloablative hematopoietic cell transplantation from haploidentical-related donors. Allocation is recorded as not applicable, and masking is recorded as none. No randomized or masked treatment comparison is described.
The intervention plan involved regulatory T-cell and conventional T-cell infusion after transplant. The brief description states that patients would receive myeloablative chemotherapy followed by stem cell rescue using bone marrow or hematopoietic peripheral blood stem cells collected by apheresis from a filgrastim-mobilized haploidentical related donor. Other listed interventions include melphalan, thiotepa, fludarabine, rabbit anti-thymocyte globulin, and the CliniMACS CD34 Reagent System. The registry does not provide a detailed sequence or dosing schedule for these interventions in the posted information.
The primary objective was to determine a maximum-tolerated dose using safety and feasibility across pre-determined combinations of regulatory and conventional T-cell dose levels. Its timeframe was 30 days after HSCT infusion, and the endpoint population was described as participants who received HSCT. The actual dose combinations and the operational criteria used to decide that a dose was tolerated are not reported in the registry record.
The registry lists an enrollment of 10, a start date of 2009-06, a primary completion date of 2012-08, and a terminated status. The reason for termination is not reported in the registry record. The listed secondary outcomes include acute graft-versus-host disease, overall survival at 1 year, median overall survival, incidence and severity of acute and chronic graft-versus-host disease, and serious infections. Although six outcome measures are recorded, the registry lists no analyses. It does not report a control group, a statistical analysis plan, or results for the primary endpoint.
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 | Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT) |
|---|---|
| STARTED | 10 |
| Received HSCT Graft | 8 |
| Received Infusion of T-reg Cells | 7 |
| Received Infusion of T-con Cells | 7 |
| COMPLETED | 7 |
| NOT COMPLETED | 3 |
Reasons for not completing this period:
| Reason | Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT) |
|---|---|
| Death, before HSCT | 1 |
| Donor mobilization failure, death | 1 |
| Death after HSCT, before T-cell infusion | 1 |
Analysis population for the primary outcome, as defined in the registry: Includes all participants that received HSCT
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 | Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT) |
|---|---|---|
| Participants analysed | 10 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 10 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 6 |
| Sex: Female, Male: Male | Participants | 4 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 4 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 6 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 |
| Race (NIH/OMB): White | Participants | 7 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Maximum-tolerated Dose (MTD) of Regulatory and Conventional T-cells, measured over 30 days after HSCT infusion.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | cells/kg |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was the maximum-tolerated dose of regulatory and conventional T-cells, expressed in cells/kg. The registry describes the intended assessment as determining this dose from safety and feasibility observed across a pre-determined set of combinations of regulatory T-cell and conventional T-cell dose levels. The timeframe was 30 days after HSCT infusion, and the endpoint population was participants who received HSCT.
In general, a maximum-tolerated-dose endpoint is used in early-phase research to guide dose selection by relating dose levels to observed tolerability and feasibility. It is not, by itself, a measure of comparative clinical benefit. Interpreting such an endpoint depends on the dose levels tested, the rules used to classify dose-limiting safety events, the observation period, and how incomplete follow-up is handled. Those protocol details are not reported in the registry record, so the endpoint’s operational definition cannot be fully assessed from the posted information.
The registry record does not report the dose combinations administered, the number of participants evaluated at each dose, any safety or feasibility findings used in the decision, or a selected maximum-tolerated dose. It also does not provide a numerical primary-endpoint result. Therefore, the endpoint is described as a study objective, not as a reported finding. The listed population includes participants who received HSCT; the record does not clarify how that population relates to the enrollment of 10.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record describes an early-phase, single-group dose-finding study and gives the intended primary endpoint, but it does not report the endpoint result. In particular, no maximum-tolerated dose, dose-level findings, or analyses are listed. The stated objective was to determine a tolerated dose from safety and feasibility across pre-determined regulatory and conventional T-cell dose combinations, assessed 30 days after HSCT infusion. Without the observed findings and decision criteria, the dose-finding question cannot be answered from the registry record.
The study has an enrollment of 10 and is marked terminated. Enrollment describes the registered study size; it does not establish how many participants received HSCT, received an infusion, completed the endpoint observation period, or contributed to each outcome. The endpoint population is described as participants who received HSCT, but the corresponding number is not reported in the registry record. The reason for termination is also not reported, so termination alone does not establish why recruitment or study activity ended.
The safety information should be read as posted counts and listed adverse-event terms, not as a controlled estimate of excess risk. The adverse-event group reports 7 participants at risk and 7 affected for both serious and other events, while deaths are listed as 9. The registry does not explain how the death count relates to those group counts. It lists progressive disease, relapse, or recurrence at 100.0%, adenoviral infection at 57.1%, acute graft-versus-host disease at 42.9%, bacteremia at 42.9%, acute renal failure at 28.6%, and diffuse alveolar hemorrhage at 28.6% among the top serious terms. The record does not provide enough context to interpret those percentages as comparative treatment effects or to determine the timing and duration of observation.
The secondary outcomes include acute and chronic graft-versus-host disease, overall survival, and serious infections, but no analyses are listed. There are no posted effect estimates, confidence intervals, or p-values for those outcomes. Consequently, precision and uncertainty for the secondary outcomes cannot be evaluated from the registry record.
Planning illustrations associated with the posted results use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; for time-to-event outcomes they use a total-event approach. These are planning assumptions, not a description of the single-group trial design and not observed power. The numerical planning outputs are not reported in the registry record. They cannot substitute for missing trial results or provide evidence of a treatment effect.
9. Statistical Methods Explained
The trial’s statistical objective is described as dose finding. The primary endpoint was the maximum-tolerated dose of regulatory and conventional T-cells, with dose expressed in cells/kg. According to the registry, the decision was to be based on safety and feasibility across a pre-determined set of dose-level combinations, with assessment 30 days after HSCT infusion. That description identifies the broad purpose of the endpoint, but it does not specify the decision algorithm or the event definitions used to classify tolerability.
What does dose finding require?
In general, a dose-finding study evaluates outcomes at planned dose levels and uses a prespecified rule to decide whether a dose is acceptable, too poorly tolerated, or merits further evaluation. The conclusions depend on which participants are evaluable, how adverse events are attributed and graded, when events count toward the decision, and how missing observations are treated. When dose combinations involve more than one cell type, the combinations and the rules for moving among them are especially important to interpretation. The registry record does not report these details, including the dose combinations, dose-escalation rules, or formal safety criteria. It is therefore not possible to reconstruct a dose-finding method from the stated objective alone.
What can be interpreted without a reported endpoint result?
A statistical method describes how observations are turned into an estimate or decision; it cannot supply the observations themselves. The registry lists no analyses and does not report a selected maximum-tolerated dose or numerical primary-endpoint result. No estimate of uncertainty for that endpoint is available in the posted information. The study’s enrollment of 10 does not reveal how many participants contributed to the endpoint or how observations were distributed across dose combinations. These missing details prevent assessment of the strength or precision of any dose-finding conclusion.
How should a confidence interval be understood?
In general, a confidence interval expresses uncertainty around an estimated quantity under the assumptions of the analysis method. A narrower interval indicates greater statistical precision under those assumptions; it does not remove concerns about bias, missing data, or study design. No confidence interval is reported for the primary endpoint or the listed secondary outcomes. As a result, statistical precision cannot be evaluated from the registry record.
What do the planning assumptions mean?
The planning illustrations are described as using the observed effect as their reference and considering a stated fraction of that effect. They assume a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. For time-to-event outcomes, the illustration is based on the total number of events; for other outcomes, it is expressed as participants per arm. These are planning assumptions, not the design of this single-group study. They do not indicate that participants were assigned equally to two arms, and they are not observed power. The numerical planning outputs and the effect values used for them are not reported in the registry record, so their magnitude cannot be assessed.
Over-dispersion is a general statistical concern when outcome variability is greater than a model assumes. If it is ignored, uncertainty estimates can be too small and intervals can appear more precise than the data warrant. Whether over-dispersion is relevant depends on the outcome and statistical model. The registry record does not identify a model for the primary endpoint or report any model checks, so no conclusion can be made about whether over-dispersion was assessed.
What can be learned from the safety listings?
In general, safety summaries describe the number of participants with events and the types of events recorded. Their interpretation depends on the population at risk, event definitions, observation time, and whether repeated events in one participant are counted separately. Percentages may also depend on the denominator used. The registry lists group counts and percentages for selected serious adverse-event terms, but it does not describe the analysis conventions or exposure time in the posted information. The safety data therefore provide a descriptive listing, not an adjusted or comparative estimate.
No inferential methods, adjustment procedures, interim analyses, or multiplicity procedures are reported in the registry record. Without a stated analysis plan and results, it is not possible to determine whether analyses were prespecified, how any uncertainty was quantified, or whether changes were made during the study. General statistical principles can clarify what information would matter, but they cannot replace the unreported methods and results for this trial.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists six outcome measures, including the primary dose-finding endpoint and secondary outcomes concerning graft-versus-host disease, overall survival, and serious infections. It does not report a multiplicity strategy. In general, when several outcomes or analyses are evaluated, multiplicity procedures can limit the chance of interpreting an isolated result as persuasive when many statistical tests have been conducted. Whether such adjustment is appropriate depends on the study’s aims and analysis plan. No inferential analyses are listed, so the record does not show whether multiplicity was addressed or whether any secondary findings were considered exploratory.
Missing-data methods are not reported in the registry record. The primary endpoint population is described as participants who received HSCT, but the number meeting that description and the number with endpoint observations are not given. The registry does not state how participants who did not receive HSCT, missed follow-up, or lacked safety or feasibility assessments would be handled. In general, missing observations can affect both which participants contribute to an analysis and how representative the analyzed group is; the direction and size of any effect depend on why data are missing and the method used.
The record also does not provide an analysis population definition for the listed secondary outcomes, or explain how incomplete follow-up was handled for survival outcomes. No interim-analysis plan, stopping rule, or statistical analysis plan is reported in the registry record. These omissions limit assessment of prespecification and of the possible influence of missing observations or repeated looks at the data. They do not establish that any particular method was or was not used outside what is recorded.
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 |
|---|---|---|---|
| Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT) | 7 | 7 | 100.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT) | 9 of 9 |
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 |
|---|---|
| Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT) | 7 of 7 |
Most frequent serious adverse event terms
| Event term | Organ system | Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT) |
|---|---|---|
| Progressive disease / relapse / recurrence | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 7 of 7 |
| Adenoviral infection | Infections and infestations | 4 of 7 |
| Acute graft vs host disease (GvHD) | Immune system disorders | 3 of 7 |
| Bacteremia | Infections and infestations | 3 of 7 |
| Acute renal failure | Renal and urinary disorders | 2 of 7 |
| Diffuse alveolar hemorrhage | Respiratory, thoracic and mediastinal disorders | 2 of 7 |
| Multi-organ failure | General disorders | 2 of 7 |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 2 of 7 |
| Adult respiratory distress syndrome (ARDS) | Respiratory, thoracic and mediastinal disorders | 1 of 7 |
| Cardiorespiratory failure | General disorders | 1 of 7 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT) |
|---|---|---|
| Infection | Infections and infestations | 7 of 7 |
| Tachypnea | Respiratory, thoracic and mediastinal disorders | 4 of 7 |
| aGvHD, skin | Immune system disorders | 3 of 7 |
| Neutropenic fever | Blood and lymphatic system disorders | 2 of 7 |
| Hemorrhagic cystitis | Gastrointestinal disorders | 2 of 7 |
| Mucositis | Gastrointestinal disorders | 2 of 7 |
| Acute graft vs host disease (aGvHD), gut | Immune system disorders | 1 of 7 |
| aGvHD, liver | Immune system disorders | 1 of 7 |
| Edema, lower extremity | General disorders | 1 of 7 |
| Pain, abdominal | General disorders | 1 of 7 |
Serious events with the highest arm-level rate: progressive disease / relapse / recurrence (100.0%), adenoviral infection (57.1%), acute graft vs host disease (gvhd) (42.9%), bacteremia (42.9%), acute renal failure (28.6%).
The safety group lists 7 participants at risk and 7 affected for serious adverse events, and likewise 7 at risk and 7 affected for other adverse events. The same group entry lists 9 deaths. The registry record does not explain how the death count relates to the participant counts, so these entries should be reported as recorded rather than reconciled or treated as interchangeable measures.
The top serious terms are listed as progressive disease, relapse, or recurrence at 100.0%; adenoviral infection at 57.1%; acute graft-versus-host disease at 42.9%; bacteremia at 42.9%; acute renal failure at 28.6%; and diffuse alveolar hemorrhage at 28.6%. These are descriptive registry listings. The record does not state the event observation period, clarify the denominator for each listed term, or provide a comparison group. The percentages therefore should not be read as estimates of comparative risk, nor do they by themselves establish that an event was caused by the intervention. The posted safety information does not support a treatment-effect conclusion.
12. What the Result Does, and Does Not, Mean
The registry record shows that the study was designed to investigate the feasibility and safety of post-transplant regulatory and conventional T-cell infusion and to identify a maximum-tolerated dose. It also shows a single-group design, no masking, enrollment of 10, and a terminated status. The endpoint was to be assessed 30 days after HSCT infusion among participants who received HSCT. These registry entries describe the study’s registered purpose and structure.
The record does not report the primary endpoint result, a selected dose, or any statistical analyses. It therefore does not establish what dose, if any, was identified as maximum tolerated, how safety and feasibility varied across dose combinations, or how precise an endpoint estimate might be. The registry also lists secondary outcomes but provides no analyses for them. Descriptive adverse-event entries are available, but their denominators and relationship to the death count are not explained in the posted information.
The single-group design does not provide a concurrent control for a like-for-like comparison. General statistical reasoning distinguishes a description of events observed in a group from an estimate of how outcomes differ between treatments. The former may characterize what was recorded during the study; the latter requires an appropriate comparison and interpretable analysis. No treatment-effect estimate is reported.
The status of terminated is not itself an explanation for why the trial ended, and the reason is not reported in the registry record. Nor does the record establish whether missing information reflects unavailable analyses, incomplete follow-up, or another circumstance. The defensible interpretation is limited to the stated design, planned endpoint, enrollment, status, and posted safety listings. It does not support a conclusion about comparative efficacy or a definitive dose-finding outcome.
13. Why This Trial Matters Statistically
This trial illustrates a statistical distinction between a clearly stated objective and a reported result. The registry defines a dose-finding question: assess safety and feasibility across pre-determined combinations of regulatory and conventional T-cell doses and identify a maximum-tolerated dose. Yet the registry record does not report the dose combinations, the decision rules, or a primary-endpoint result. Without those elements, readers cannot evaluate the dose-finding decision, even though the endpoint’s general purpose is clear.
The single-group design is also important to interpretation. It can describe observations made among participants receiving the study regimen, but it does not provide a concurrent control group for estimating a comparative treatment effect. This distinction matters when reading the safety listings: observed event terms and percentages are not equivalent to evidence that the intervention changed event risk.
Finally, the record demonstrates why analysis populations, follow-up, and denominators are central to safety and outcome reporting. The primary endpoint refers to participants who received HSCT, while the registry separately reports enrollment of 10 and safety counts of 7 at risk and 9 deaths. The record does not explain how these figures relate. Clear participant flow and outcome-specific denominators would be needed to understand who contributed to each result. Since no analyses are listed, statistical uncertainty and the handling of missing observations cannot be assessed. These are not merely technical details: they determine what the posted findings can support.
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 registry record leaves several trial-specific details unavailable. It does not report the pre-determined cellular dose combinations, the number of participants treated at each combination, the dose-escalation or dose-selection rule, or the criteria for defining an intolerable event. These omissions prevent an independent assessment of how the maximum-tolerated dose was intended to be selected.
The primary endpoint is described for participants who received HSCT, but the record does not report how many participants met that description or how many had an evaluable 30-day assessment. It does not state how participants without endpoint observations were handled. Although enrollment is 10, the endpoint-specific denominator is not reported in the registry record.
The safety entry has 7 participants at risk and 7 affected for both serious and other events, alongside 9 deaths. The relationship among these counts is not explained. The record also does not specify the observation time for the adverse-event listings, the denominator used for each percentage, or whether listed terms refer to participants or events. These details limit interpretation of the safety summaries.
The trial is marked terminated, but the reason is not reported in the registry record. No statistical analysis plan, interim-analysis information, multiplicity procedure, or missing-data method is reported. There is no concurrent comparator group and no listed analysis of the primary or secondary outcomes. The record consequently does not support an assessment of comparative effects, endpoint precision, or the robustness of any dose-finding conclusion.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Haploidentical Allogeneic Transplant With Post-transplant Infusion of Regulatory T-cells — NCT01050764.
- Registry record last updated 2018-06-27; status last verified 2018-06.
- Results first posted 2017-05-11.
- 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
This phase 1 and phase 2 trial evaluated post-transplant infusion of allogeneic regulatory and conventional T-cells in patients with hematologic malignancies undergoing myeloablative transplantation from haploidentical-related donors. It was registered as a single-group, unmasked study, with enrollment of 10 and a terminated status. The start date was 2009-06 and the primary completion date was 2012-08.
The primary objective was to determine a maximum-tolerated dose from safety and feasibility across pre-determined cell-dose combinations, assessed 30 days after HSCT infusion. The registry record does not report the dose combinations, endpoint result, or any analyses. Safety listings include 7 participants at risk and 7 affected for serious and other events, and 9 deaths; the record does not explain how these counts relate. The reason for termination is not reported in the registry record.