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 Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases (NCT02231710), a na phase 1 study. The primary outcome is Adverse Events.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02231710 |
| Phase | Phase 1 |
| Condition | Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Bellicum Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned people with non-malignant diseases undergoing haploidentical, related, T cell-depleted hematopoietic cell transplantation. The registry lists primary immune deficiency disorders, hemophagocytic lymphohistiocytosis, inherited bone marrow failure syndrome, hemoglobinopathies, and metabolic disorders as conditions of interest. One subject was dosed.
Intervention
The intervention was BPX-501 gene-modified donor T cells after transplantation, followed by rimiducid (AP1903) on Day 7. The arm description specifies a single administration of BPX-501 T cells after partially mismatched, related, T cell-depleted HCT, then a rimiducid infusion.
Comparator
There was one study group and no comparator arm. Allocation is recorded as NA and masking as none. Consequently, the record does not offer a like-for-like control group for separating effects associated with the intervention from other events after transplantation.
Primary question
The primary outcome was adverse events over 24 months, with safety described in relation to Grade III-IV GVHD and response to rimiducid. The record says one subject received 5 x 106 cells of BPX-501 T cells and 0.4 mg/kg of rimiducid on Day 7. It also says no efficacy data were gathered and that the subject was withdrawn by the investigator's physician. The study was terminated early by the sponsor due to lack of enrolment. The available record therefore frames a safety question but does not provide an efficacy evaluation or a comparison between treatment strategies.
3. Trial Design
This was a Phase 1, single-group study sponsored by Bellicum Pharmaceuticals. Its official title describes evaluation of BPX-501 T cells and AP1903 for prevention of GVHD after haploidentical, related, T cell-depleted hematopoietic cell transplantation for non-malignant diseases. The planned treatment sequence was BPX-501 T cells after transplantation, followed by rimiducid on Day 7. The registry lists one intervention, BPX-501 and Rimiducid, and one study group.
The registry records allocation as NA, masking as none, and the model as single group. These design features mean the available record has no randomized assignment and no masked comparison. The enrollment was 1. The study started in 2015-02, had a primary completion date of 2015-09, and is recorded as terminated. The reason given is lack of enrolment; the record also says the sponsor terminated the study early.
The primary outcome was adverse events over 24 months. Secondary outcomes listed in the registry include GVHD, immune reconstitution, infection rates, graft rejection, rimiducid activity, and high-grade toxicity. The record lists 8 outcome measures, but provides no analyses. It does not report a detailed statistical analysis plan, a sample-size justification, or interim analyses.
With one participant and no control group, the study record cannot support a comparative estimate between BPX-501 and rimiducid treatment and an alternative strategy. Nor does the enrollment describe a completed assessment across the intended range of conditions. The record instead documents a limited exposure and a study that ended before efficacy data were gathered. These design and completion details are essential context for reading the safety outcome and the absence of an efficacy result.
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 | BPX-501 and Rimiducid |
|---|---|
| STARTED | 1 |
| COMPLETED | 0 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | BPX-501 and Rimiducid |
|---|---|
| Physician Decision | 1 |
Analysis population for the primary outcome, as defined in the registry: One subject was dosed with 5 x 106 cells of BPX-501 T cells and 0.4 mg/kg of rimiducid on Day 7. No efficacy data was gathered for this patient and patient was withdrawn from the Study by investigator's physician. The study was terminated early by Sponsor due to lack of enrolment
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 | BPX-501 and Rimiducid |
|---|---|---|
| Participants analysed | 1 | |
| Age, Continuous | years | 12 (12 to 12) |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 1 |
| Race and Ethnicity Not Collected | Participants | — |
| Region of Enrollment: United States | participants | 1 |
| sickle cell disease | Participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Adverse Events, measured over 24 months.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary outcome is titled “Adverse Events” and has a timeframe of 24 months. Its description frames safety in terms of Grade III-IV GVHD that is non-responsive to rimiducid after HCT using HLA-haploidentical CD34+ selected peripheral blood stem cell grafts and BPX-501 T cells, followed by scheduled rimiducid on Day 7. The description then says the measure is the number of patients who experienced Grade III-IV GVHD that was “not non-responsive” to rimiducid administration. Those phrases are not equivalent: one refers to non-responsive GVHD, while the other appears to refer to GVHD that was not non-responsive. The registry record does not resolve this wording difference.
Safety outcomes are used to describe harms or complications observed during treatment and follow-up. In general, a clearly defined safety endpoint helps readers understand which events count, how they are classified, and when they are assessed. Here, the endpoint title is broad, while its description focuses on a particular GVHD event and its relation to rimiducid. The record gives a 24-month timeframe but does not report an endpoint result that can resolve the ambiguity. It also explicitly states that no efficacy data were gathered for the participant.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry reports a very limited clinical experience: one subject was dosed with BPX-501 T cells and rimiducid. The subject was withdrawn from the study by the investigator's physician, and the record says no efficacy data were gathered. The study was terminated early by the sponsor due to lack of enrolment. These registry entries sharply limit what can be inferred from the posted results.
The adverse-event group records 1 participant at risk and 1 participant with a serious adverse event; it also records 1 participant at risk and 1 with an adverse event in the other category. Deaths are recorded as 0. Haemolysis is listed among the top serious terms at 100.0%. These entries describe the registry's event reporting, not a comparison with another group. The record does not establish that the intervention caused an event, and the absence of a control group means that the event counts cannot be contrasted with a control rate.
The primary endpoint description is not fully consistent in its wording. It first characterizes safety as non-responsive Grade III-IV GVHD to rimiducid, then describes the reported measure as Grade III-IV GVHD that was not non-responsive to rimiducid. The record does not provide enough information to resolve that distinction or to state a primary endpoint estimate beyond the wording and group-level safety information recorded.
No efficacy data were gathered for the participant, so the record cannot answer whether the treatment sequence affected engraftment, GVHD prevention, or any other efficacy outcome. Although the registry lists secondary outcomes, it does not provide analyses for them. There are no reported estimates of treatment effects, confidence intervals, or inferential tests in the registry record.
In general, a confidence interval conveys the range of effect estimates compatible with the observed data under the statistical model used. It would also make uncertainty visible. No confidence interval is reported, and with one participant a stable estimate of event frequency or treatment effect is not available. The record should therefore be read as an account of a single participant's exposure and recorded adverse events, not as evidence that would establish a safety rate or comparative effect.
Sample-size planning illustrations, if considered alongside these results, are not observed power and do not restore the missing comparison or efficacy information. The registry describes them as planning calculations based on an observed effect, but separately says no efficacy data were gathered. That context makes them unsuitable as a substitute for trial evidence about treatment effects.
9. Statistical Methods Explained
Several basic design and analysis concepts help explain the limits of the record. The study is listed as Phase 1 and single group. In general, a single-group design observes people receiving the intervention without a concurrent control group. Such a design may describe what happened to participants who received treatment, but it cannot by itself distinguish treatment-related events from events associated with the underlying disease, transplantation, or other aspects of care. The registry's allocation value is NA, and masking is recorded as none; no randomized or masked comparison is described.
The outcome is an adverse-event measure with a 24-month timeframe. A timeframe defines the period over which the outcome is assessed. In general, a safety outcome needs an operational definition: the event criteria, severity classification, method of attribution, and follow-up rules should be clear enough for consistent interpretation. The registry names adverse events and describes a specific GVHD-related safety endpoint, but its wording differs between non-responsive and “not non-responsive” GVHD. The record does not clarify whether this is a wording error or which interpretation was intended.
The registry reports no analyses. In general, a statistical analysis can describe event counts, estimate rates or treatment effects, and quantify uncertainty. For a treatment comparison, a model or other method would need to account for the design and outcome type. No such analysis, effect estimate, confidence interval, or inferential test is reported in this record. The absence of an analysis is especially consequential when the study has only one participant and no control group: there is no meaningful basis for estimating a comparative treatment effect.
A confidence interval is a statistical range intended to express uncertainty around an estimate under a specified model. Its width depends on the data and assumptions. A narrow interval would indicate greater precision under that model, while a wide interval would indicate less precision. No confidence interval is reported. General statistical concepts cannot supply one in the absence of an applicable reported estimate and analysis.
Over-dispersion is a general issue in count data: it occurs when variability is greater than a simple model assumes. When present, standard errors from a model that ignores it may be too small, making results appear more precise than they are. Whether over-dispersion was assessed or relevant to any particular outcome is not reported in the registry record. The record does not specify a count-data model or another statistical method for the listed outcomes.
The registry lists 8 outcome measures and names secondary outcomes that include GVHD, immune reconstitution, infection rates, graft rejection, rimiducid activity, and high-grade toxicity. A list of outcomes is not itself an analysis plan. The record does not state which secondary outcomes were tested, how they were measured, which timepoints applied to each, or how missing observations would be handled. It also does not report whether any analyses were adjusted for multiple comparisons.
The sample-size assumptions describe planning illustrations using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect stated as a fraction of an observed effect. They are planning illustrations, not the trial's own design or observed power. For time-to-event outcomes, the description refers to total events; for other outcomes, it refers to participants per arm. Those assumptions do not describe this study's single-group design, and the record does not report a trial-specific sample-size justification. Because the record says no efficacy data were gathered, an observed efficacy effect is not available from this participant to support a treatment-effect interpretation.
In general, a planning calculation asks how many participants or events might be needed under specified assumptions. It does not show the probability that a completed study has proved an effect, and it cannot compensate for absent outcome data. Planning illustrations should therefore be distinguished from actual enrollment, observed events, and an analysis of treatment effects.
Finally, the event summaries are descriptive. Counts indicate how many participants were recorded in the relevant categories, while a listed event term identifies an event reported as serious. With one participant, such entries characterize this registry record, but they do not estimate how often an event would occur in a broader population. No generalization beyond the recorded participant is justified by the reported data alone.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 8 outcome measures and several secondary outcome areas, but it does not report an analysis plan specifying which outcomes were confirmatory, how many hypotheses were tested, or whether any adjustment for multiple testing was used. In general, multiplicity arises when several outcomes or comparisons are evaluated: the chance of at least one apparently notable result can increase as more tests are considered. Adjustments or a prespecified hierarchy can address that issue, but the registry record does not say whether either approach was planned or used.
The record reports no analyses and no efficacy data for the participant. It does not describe a primary or secondary analysis population, such as whether all enrolled participants or only treated participants would be included in a particular analysis. It also does not report methods for handling missing data, withdrawals, incomplete follow-up, or outcomes that could not be assessed. These details are important because analysis populations and missing-data rules affect how outcome summaries should be interpreted.
The record says the subject was withdrawn by the investigator's physician and that no efficacy data were gathered. It does not state which assessments were completed before withdrawal or whether the 24-month primary-outcome follow-up was completed. The study's early termination due to lack of enrolment further limits outcome availability. No assumptions about the reasons for missing efficacy observations or their statistical treatment can be made from the record.
In general, missing-data methods rely on stated assumptions about why information is absent and how that absence relates to measured or unmeasured outcomes. Without the analysis population, missing-data approach, and endpoint results, it is not possible to assess whether missingness could have affected a reported estimate. No such estimate is reported.
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 |
|---|---|---|---|
| BPX-501 and Rimiducid | 1 | 1 | 100.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| BPX-501 and Rimiducid | 0 of 1 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 1% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| BPX-501 and Rimiducid | 1 of 1 |
Most frequent serious adverse event terms
| Event term | Organ system | BPX-501 and Rimiducid |
|---|---|---|
| haemolysis | Blood and lymphatic system disorders | 1 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | BPX-501 and Rimiducid |
|---|---|---|
| Platelet Count Decreased | Investigations | 1 of 1 |
| Haematocrit Decreased | Investigations | 1 of 1 |
| Activated Partial Thromboplastin Time abnormality | Investigations | 1 of 1 |
| Hypersensitivity | Immune system disorders | 1 of 1 |
| Febrile Neutropenia | Blood and lymphatic system disorders | 1 of 1 |
| Disseminated Intravascular Coagulation | Blood and lymphatic system disorders | 1 of 1 |
Serious events with the highest arm-level rate: haemolysis (100.0%).
The adverse-event summary records 1 participant at risk and 1 participant with a serious adverse event in the BPX-501 and Rimiducid group. It also records 1 at risk and 1 affected in the other adverse-event category, with deaths recorded as 0. Haemolysis is listed as a top serious term at 100.0%. The registry does not provide further detail in the record about event timing, severity beyond the serious classification, duration, outcome, or attribution to the intervention.
These figures should be read as descriptive entries from a study with one dosed subject, not as stable estimates of event frequency. The percentage for haemolysis is a registry summary, not evidence that the same percentage would occur in a larger group. Nor does the listing establish that BPX-501 or rimiducid caused the event. The single-group design has no control arm for comparison, and the record does not report an attribution analysis.
The primary outcome description also focuses on Grade III-IV GVHD in relation to rimiducid, but its wording alternates between non-responsive and “not non-responsive.” The adverse-event summary does not resolve that ambiguity or provide an endpoint result for the specific GVHD measure. The recorded serious-event figures and the primary GVHD endpoint should therefore be treated as distinct pieces of information.
12. What the Result Does, and Does Not, Mean
The result does mean that the registry documents one subject receiving BPX-501 T cells after transplantation and rimiducid on Day 7. The adverse-event summary records one participant with a serious adverse event, one with an event in the other category, 0 deaths, and haemolysis as a top serious term at 100.0%. It also records that the subject was withdrawn by the investigator's physician and that no efficacy data were gathered.
The result does not establish a rate of serious adverse events for a broader population. A single participant cannot represent the range of outcomes that might occur among people with the listed non-malignant conditions. Without a comparison group, these entries also cannot show whether the treatment sequence changed the risk of an event relative to another approach. The registry does not establish a causal link between the intervention and haemolysis or any other event.
The study record also does not show whether the treatment affected engraftment, GVHD prevention, immune reconstitution, infection rates, graft rejection, rimiducid activity, or high-grade toxicity. Those subjects appear among the outcome measures, but the record says no efficacy data were gathered and reports no analyses. The wording of the primary GVHD endpoint is ambiguous, and no outcome estimate is reported to clarify it.
In general, descriptive observations can identify what was recorded for an individual, while treatment effects require an appropriate design, outcome data, and analysis. A planning calculation is not a result and is not observed power. The posted record is therefore informative about the limited exposure and the recorded safety entries, but it cannot support a conclusion about comparative efficacy, a population-level safety profile, or the frequency of harms.
13. Why This Trial Matters Statistically
This trial matters statistically because it illustrates the difference between a registered research question and an answer supported by outcome data. The record describes a Phase 1 safety study with a defined intervention sequence and a 24-month primary outcome timeframe. Yet enrollment was 1, the participant was withdrawn, no efficacy data were gathered, and the study was terminated early due to lack of enrolment. The intended question and the information ultimately available are therefore not the same.
The record also shows why design details matter. With one study group and no comparator, observed events cannot be contrasted with events under another strategy. Without a sufficiently informative set of observations, counts cannot provide a dependable estimate of event frequency. These are limits of the design and available data, not merely limitations of a statistical calculation.
Outcome wording matters as well. The primary measure is described in terms of non-responsive Grade III-IV GVHD, but the reporting sentence says “not non-responsive.” That distinction affects what event is being counted. A reader cannot resolve it statistically without a clearer endpoint definition and reported outcome data.
Finally, sample-size planning illustrations should not be mistaken for evidence from a completed analysis. Statistical planning depends on assumptions, while conclusions about safety or efficacy depend on observed outcomes and appropriate analyses. The record makes the separation especially visible: it includes planning assumptions but reports no efficacy data or analyses. This is a useful reminder that statistical precision cannot be created by calculation when the underlying clinical information is absent.
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 limitation is the very small enrollment: the record reports 1 subject dosed. The study was terminated early due to lack of enrolment, so the record does not describe a completed assessment across the intended population. No efficacy data were gathered for the participant, who was withdrawn from the study by the investigator's physician. The record does not state which assessments were completed before withdrawal.
The study had a single group, with no comparator arm, allocation recorded as NA, and masking recorded as none. As a result, the registry offers no concurrent control for interpreting adverse events or evaluating treatment effects. The recorded safety counts are observations from this study, not comparative estimates.
The primary endpoint's description is ambiguous about whether it counts Grade III-IV GVHD that is non-responsive or “not non-responsive” to rimiducid. Although the timeframe is 24 months, the record does not report a primary endpoint result that resolves this wording. It also does not report the statistical methods, analysis populations, missing-data procedures, or multiplicity approach. Those items are not reported in the registry record.
The adverse-event summary contains limited detail about event timing, severity beyond the serious classification, outcomes, and attribution. The record lists haemolysis as a top serious term, but does not supply further clinical context. Taken together, the single participant, early termination, absent efficacy data, ambiguous endpoint wording, and lack of reported analyses restrict interpretation to a description of the registry entries. They do not support broad safety or efficacy conclusions.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases — NCT02231710.
- Registry record last updated 2024-03-25; status last verified 2023-09.
- Results first posted 2024-03-25.
- 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
This Phase 1 study evaluated BPX-501 gene-modified donor T cells followed by rimiducid after haploidentical transplantation for non-malignant diseases. It used a single-group design, with no masking and allocation recorded as NA. The registry records enrollment of 1 and an intervention of BPX-501 and Rimiducid.
The primary outcome was adverse events over 24 months, described in relation to Grade III-IV GVHD and rimiducid response. One subject was dosed and later withdrawn by the investigator's physician; no efficacy data were gathered. The study was terminated early by the sponsor due to lack of enrolment. The adverse-event summary records 1 participant with a serious adverse event, 1 in the other adverse-event category, 0 deaths, and haemolysis as a top serious term at 100.0%.
The registry lists 8 outcome measures but reports no analyses. Its endpoint wording does not clearly resolve whether the primary measure concerns GVHD that is non-responsive or “not non-responsive” to rimiducid. The available information supports a limited descriptive account of one participant, not an estimate of comparative treatment effects or population-level event rates.