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 CPI-613, Cytarabine, and Mitoxantrone Hydrochloride in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Granulocytic Sarcoma (NCT02484391), a na phase 1 study. The primary outcome is Feasibility of Administering CPI-613 in Combination With High Dose Cytarabine and Mitoxantrone During Induction, Consolidation and Maintenance Therapies, Defined as Percentage of Patients Eligible for Maintenance Therapy Who Complete at Least 3 Courses.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02484391 |
| Phase | Phase 1 |
| Condition | Granulocytic Sarcoma, Recurrent Adult Acute Myeloid Leukemia |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 48 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Wake Forest University Health Sciences |
| Results posted | Yes |
2. Clinical Question
Population
Patients with relapsed or refractory acute myeloid leukemia (AML) or granulocytic sarcoma. The registry record describes a phase I study with enrollment of 48.
Intervention
The treatment combined CPI-613, cytarabine, and mitoxantrone hydrochloride. The registry record also lists hematopoietic cell transplantation as an intervention, but does not specify in the summarized information who received it or how it was used.
Comparator
There was no concurrent comparison group: the study is listed as single-group, with allocation recorded as not applicable. The arms labeled 1500, 2000, and 2500 are not a randomized control comparison.
Primary question
The primary question was whether the regimen could be administered through induction, consolidation, and maintenance, with feasibility defined by maintenance completion. Specifically, the endpoint concerned the percentage of patients eligible for maintenance who completed at least 3 courses. The registry states that if at least 50% of eligible patients completed 3 cycles, the regimen would be considered feasible for future study. Its reported outcome says none of the participants completed three maintenance cycles.
3. Trial Design
This was a phase I, open-label study with a single-group design. The registry record lists allocation as not applicable and masking as none. As a result, the design does not provide a randomized, concurrent control group for estimating how outcomes would differ under another treatment. The arm labels 1500, 2000, and 2500 describe groups in the record, but they should not be read as randomized treatment and control arms.
The study evaluated CPI-613 given with high-dose cytarabine and mitoxantrone in patients with relapsed or refractory AML. The record also includes granulocytic sarcoma in its condition information and in the trial title. Hematopoietic cell transplantation is listed among the interventions; its role or use by participants is not described in the information summarized in the registry record.
Enrollment is listed as 48. The recorded start was 2015-09, primary completion was 2018-10, and status is completed. The primary endpoint concerned maintenance therapy over a timeframe of up to 12 weeks. The registry describes secondary outcomes that include toxicity, overall survival, and response rate assessed using standard AML criteria.
The open-label, single-group structure is relevant to interpretation. It can describe outcomes observed among participants receiving the regimen, but it cannot by itself establish a comparative treatment effect. The registry record does not report a statistical analysis plan, the methods used for the secondary outcomes, or the rationale for the enrollment target.
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 | 1500 | 2000 | 2500 |
|---|---|---|---|
| STARTED | 21 | 20 | 7 |
| Achieved Remission | 9 | 7 | 5 |
| Received Maintenance Therapy | 0 | 0 | 2 |
| COMPLETED | 0 | 0 | 0 |
| NOT COMPLETED | 21 | 20 | 7 |
Reasons for not completing this period:
| Reason | 1500 | 2000 | 2500 |
|---|---|---|---|
| Lack of Efficacy | 15 | 14 | 3 |
| Lost to Follow-up | 0 | 0 | 2 |
| Physician Decision | 1 | 3 | 0 |
| Went to Transplant | 5 | 3 | 2 |
Analysis population for the primary outcome, as defined in the registry: None of the 9 participants in the 1500 mg arm and the 7 participants in the 2000 mg arm started maintenance, only 2 out of the 5 participants in the 2500 mg arm started maintenance. None of participants completed three cycles of maintenance.
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 | 1500 | 2000 | 2500 | Total |
|---|---|---|---|---|---|
| Participants analysed | 21 | 20 | 7 | 48 | |
| Age, Continuous | years | 61 (35 to 85) | 65.5 (21 to 76) | 64 (27 to 78) | 64 (21 to 85) |
| Sex: Female, Male: Female | Participants | 9 | 8 | 3 | 20 |
| Sex: Female, Male: Male | Participants | 12 | 12 | 4 | 28 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 3 | 3 | 1 | 7 |
| Race (NIH/OMB): White | Participants | 18 | 16 | 6 | 40 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 1 | 0 | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Feasibility of Administering CPI-613 in Combination With High Dose Cytarabine and Mitoxantrone During Induction, Consolidation and Maintenance Therapies, Defined as Percentage of Patients Eligible for Maintenance Therapy Who Complete at Least 3 Courses, measured over Up to 12 weeks of maintenance therapy.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was feasibility of administering CPI-613 with high-dose cytarabine and mitoxantrone during induction, consolidation, and maintenance. It was defined as the percentage of patients eligible for maintenance who completed at least 3 courses. The registry record set a decision criterion: if at least 50% of eligible patients completed 3 cycles of maintenance, the regimen would be considered feasible for future study.
This endpoint focuses on treatment delivery rather than a direct measure of antileukemic response. In a feasibility assessment, completion can indicate whether participants were able to proceed through the planned treatment period. It does not, by itself, establish efficacy, safety, or the reason that some participants did not complete treatment. General statistical point: a percentage is interpretable only in relation to its denominator and a clearly defined outcome window. Here, the registry identifies maintenance eligibility as the denominator concept and specifies a timeframe of up to 12 weeks of maintenance, but does not provide a full statistical account of eligibility and completion by participant.
The posted outcome states that none of the participants completed three cycles of maintenance. It also reports that none of the 9 participants in the 1500 arm and the 7 participants in the 2000 arm started maintenance, while 2 out of the 5 participants in the 2500 arm started maintenance. These statements describe maintenance progression; they do not establish why participants did or did not proceed.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The principal reported result is that no participants completed three cycles of maintenance. The registry also reports that none of the 9 participants in the 1500 arm and the 7 participants in the 2000 arm started maintenance, and that 2 out of the 5 participants in the 2500 arm started maintenance. These are descriptive statements about treatment progression. They do not provide a comparison with an untreated group or another regimen.
The endpoint's stated feasibility criterion was completion of 3 maintenance cycles by at least 50% of patients eligible for maintenance. The reported absence of participants completing three cycles falls short of that stated criterion as described in the registry. However, the record does not provide a complete accounting of the endpoint denominator, eligibility decisions, or reasons for not starting or completing maintenance. Those omissions limit how precisely the feasibility outcome can be interpreted.
The study is single-group and open-label. The groups labeled 1500, 2000, and 2500 do not constitute a randomized comparison, so differences in their listed outcomes cannot be attributed to the group label or interpreted as controlled dose effects. The registry record does not report inferential analyses, confidence intervals, or p-values for the primary endpoint. There is therefore no reported estimate of statistical uncertainty around the feasibility result.
Safety information is reported by group, including counts for serious adverse events and selected serious-event terms. These figures describe events in this study; they do not establish whether an event was caused by treatment. The registry also lists death counts, but the information summarized does not identify causes or treatment relationships. Similarly, the record names overall survival and response rate as secondary outcomes but does not include their numerical results in the registry entries.
Overall, the posted results describe maintenance initiation and completion and provide selected safety counts. They do not support a controlled estimate of treatment benefit, a conclusion about comparative safety, or an explanation for treatment discontinuation. The sponsor's interpretation or analysis beyond these registry entries is not reported in the registry record.
9. Statistical Methods Explained
The statistical information in the registry record is limited. It describes a phase I, open-label, single-group trial and identifies a primary endpoint based on maintenance completion. No statistical analysis plan or inferential analyses are reported in the registry record. The explanations below give general context for understanding the endpoint and the types of statistical summaries that would ordinarily be relevant; they do not describe additional analyses conducted in this study.
What does a percentage endpoint represent?
General: a percentage is a proportion expressed on a percentage scale. It requires a numerator, such as participants meeting the completion definition, and a denominator, such as all participants who met the eligibility condition. The interpretation depends on exactly who is included in that denominator and how the outcome is defined. A denominator restricted to participants eligible for maintenance answers a different question from a denominator containing everyone enrolled.
In this trial, the primary endpoint is described as the percentage of maintenance-eligible patients who completed at least 3 courses. The stated criterion for considering the regimen feasible for future study was at least 50% completing 3 cycles. The registry narrative reports maintenance starters by arm and says none of the participants completed three cycles. It does not provide a complete analysis accounting for the endpoint denominator or individual eligibility. Without that accounting, readers cannot independently reconstruct the endpoint percentage from the narrative alone.
What would a confidence interval add?
General: a confidence interval expresses the statistical uncertainty around an estimate, under the assumptions of the method used. For a proportion, its width depends in part on the amount of information in the denominator and on the observed proportion. A confidence interval does not remove limitations in the underlying design or correct an unclear outcome definition. The registry record does not report a confidence interval for the primary endpoint, so the precision of an estimated completion percentage cannot be assessed from the reported information.
Why does the study design matter?
General: a randomized concurrent comparison helps distinguish differences associated with treatment assignment from differences that may arise because groups differ in other ways. In a single-group study, observed outcomes describe participants who received the study regimen, but there is no concurrent randomized control group to estimate what would have happened under another treatment. Open-label status also means masking is absent, as the registry records. These design features do not invalidate descriptive reporting, but they constrain causal and comparative interpretations.
The three group labels in the safety information should not be treated as randomized arms. Allocation is recorded as not applicable, and the trial model is single-group. The registry record does not describe a statistical method for comparing those groups. It would therefore be inappropriate to treat an apparent difference across labels as a controlled treatment effect.
How should the planning illustrations be read?
General: sample-size planning uses assumptions about the effect to detect, variability, allocation, and statistical error rates. A planning estimate is not an observed trial result and is not a measure of the probability that a result is true. The sample-size illustrations associated with this record are described as planning calculations based on fractions of the observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. For time-to-event outcomes, the stated quantities are total events; for other outcomes, they are participants per arm.
Those assumptions describe hypothetical planning, not the actual single-group trial design. In particular, equal allocation between two arms does not make the enrolled study a randomized two-arm trial. The record says the illustrations are not the trial's own design and that they are not observed power. Sample-size assumptions cannot substitute for a prespecified analysis plan or for uncertainty estimates around the study's actual outcomes.
What statistical detail is missing?
The registry record lists no analyses. It does not report the model or test used for the primary endpoint, how incomplete outcome data were handled, or whether any interim analysis was planned. It also does not provide numerical results for the listed response-rate and overall-survival outcomes in the registry record. Without these details, readers cannot assess the analytic choices or reproduce inferential results. General: transparent reporting of outcome definitions, denominators, analysis populations, missing-data handling, and uncertainty estimates is important because each can affect how a result should be read.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists no analyses and does not report a statistical plan for handling multiple outcomes. It identifies a primary endpoint and names secondary outcomes that include toxicity, overall survival, and response rate, but it does not say whether a hierarchy of tests, an adjustment for multiple comparisons, or another multiplicity strategy was planned. Accordingly, the risk of chance findings across multiple outcome analyses cannot be evaluated from the record.
The primary endpoint is defined among patients eligible for maintenance. The registry narrative provides counts related to starting maintenance in groups labeled 1500, 2000, and 2500, and reports no participants completing three cycles. It does not give a full account of eligibility, the denominator used for the endpoint percentage, or missing endpoint observations. The reasons participants did not start or complete maintenance are not reported in the registry record.
The analysis population is also not defined beyond the endpoint's maintenance-eligibility wording. The record does not state whether analyses used all enrolled participants, only participants who began maintenance, or another population. Nor does it describe imputation, censoring, or other missing-data procedures. These details matter because the estimated completion proportion can depend on which participants are counted and how unavailable outcomes are treated.
General: multiplicity concerns arise when several outcomes or comparisons are examined, because interpreting each as if it were the sole planned analysis can overstate how much evidence an individual result provides. Missing-data methods likewise rely on assumptions about why values are unavailable. Neither issue can be resolved by the descriptive counts alone. No multiplicity adjustment or missing-data method should be assumed in the absence of a report.
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 |
|---|---|---|---|
| 1500 | 21 | 21 | 100.0% |
| 2000 | 16 | 20 | 80.0% |
| 2500 | 4 | 7 | 57.1% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| 1500 | 6 of 21 |
| 2000 | 6 of 20 |
| 2500 | 1 of 7 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| 1500 | 21 of 21 |
| 2000 | 20 of 20 |
| 2500 | 7 of 7 |
Most frequent serious adverse event terms
| Event term | Organ system | 1500 | 2000 | 2500 |
|---|---|---|---|---|
| Lymphocyte count decreased | Investigations | 17 of 21 | 3 of 20 | 1 of 7 |
| Platelet count decreased | Investigations | 16 of 21 | 3 of 20 | 1 of 7 |
| White blood cell decreased | Investigations | 16 of 21 | 2 of 20 | 1 of 7 |
| Neutrophil count decreased | Investigations | 15 of 21 | 2 of 20 | 1 of 7 |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 5 of 21 | 2 of 20 | 0 of 7 |
| Sepsis | Infections and infestations | 4 of 21 | 0 of 20 | 0 of 7 |
| Hypoalbuminemia | Metabolism and nutrition disorders | 3 of 21 | 0 of 20 | 0 of 7 |
| Hyponatremia | Metabolism and nutrition disorders | 3 of 21 | 0 of 20 | 0 of 7 |
| Cardiac arrest | Cardiac disorders | 1 of 21 | 2 of 20 | 0 of 7 |
| Anemia | Blood and lymphatic system disorders | 1 of 21 | 0 of 20 | 1 of 7 |
Most frequent non-serious adverse event terms
| Event term | Organ system | 1500 | 2000 | 2500 |
|---|---|---|---|---|
| Hyperglycemia | Metabolism and nutrition disorders | 21 of 21 | 19 of 20 | 7 of 7 |
| Anemia | Blood and lymphatic system disorders | 20 of 21 | 19 of 20 | 6 of 7 |
| Hypoalbuminemia | Metabolism and nutrition disorders | 18 of 21 | 20 of 20 | 7 of 7 |
| Diarrhea | Gastrointestinal disorders | 16 of 21 | 20 of 20 | 7 of 7 |
| Hypokalemia | Metabolism and nutrition disorders | 18 of 21 | 17 of 20 | 7 of 7 |
| Disseminated intravascular coagulation | Blood and lymphatic system disorders | 15 of 21 | 18 of 20 | 7 of 7 |
| Fatigue | General disorders | 16 of 21 | 17 of 20 | 7 of 7 |
| Aspartate aminotransferase increased | Investigations | 18 of 21 | 15 of 20 | 7 of 7 |
| Anorexia | Metabolism and nutrition disorders | 16 of 21 | 17 of 20 | 6 of 7 |
| Hypomagnesemia | Metabolism and nutrition disorders | 15 of 21 | 18 of 20 | 6 of 7 |
Serious events with the highest arm-level rate: lymphocyte count decreased (81.0%), platelet count decreased (76.2%), white blood cell decreased (76.2%), neutrophil count decreased (71.4%), respiratory failure (23.8%).
The registry lists serious-event counts by group. For the group labeled 1500, 21 participants were listed as at risk and 21 as affected; for 2000, the corresponding counts were 20 and 16; for 2500, they were 7 and 4. These are registry counts, not a randomized comparison. They should be read as descriptive safety information within the recorded groups.
The leading serious-event terms listed include lymphocyte count decreased at 81.0%, platelet count decreased at 76.2%, white blood cell decreased at 76.2%, neutrophil count decreased at 71.4%, respiratory failure at 23.8%, and sepsis at 19.0%. The registry record does not provide further context about event timing, recurrence, severity beyond the serious-event classification, or how each percentage was calculated.
Death counts are listed as 6 for the 1500 group, 6 for the 2000 group, and 1 for the 2500 group. The record does not identify causes of death or whether the deaths were judged related to treatment. General: an event recorded after treatment is not automatically caused by treatment; determining attribution requires clinical assessment and a defined reporting method. These counts alone do not establish comparative risk or treatment causality.
12. What the Result Does, and Does Not, Mean
The primary result describes maintenance completion: the registry reports that none of the participants completed three cycles. It also describes how many participants started maintenance in the groups labeled 1500, 2000, and 2500. These observations address treatment progression, which is the focus of the feasibility endpoint, but the record does not explain why participants did not start or complete maintenance.
The endpoint's stated decision criterion was that at least 50% of eligible patients complete 3 cycles for the regimen to be considered feasible for future study. The reported lack of three-cycle completion does not meet that criterion as stated. Interpretation is constrained by the absence of a complete endpoint denominator and eligibility account in the registry information. No confidence interval or inferential analysis is reported for this outcome.
The result does not establish whether CPI-613 with cytarabine and mitoxantrone is more effective or safer than another treatment. The study has a single-group, open-label design and no randomized concurrent comparator. It also does not establish why maintenance was not initiated or completed, whether the observed safety events were caused by treatment, or how the listed death counts relate to treatment.
The registry names response rate, overall survival, and toxicity among secondary outcomes, but numerical results for response rate and overall survival are not reported in the registry record for this interpretation. Their mention as planned or listed outcomes should not be mistaken for evidence of a particular result. The safety counts provide descriptive information, not a controlled estimate of risk.
In general, a feasibility result can inform whether a regimen proceeded through a defined treatment process in a study population. It is not, on its own, a measure of clinical benefit. The posted information supports a narrow description of maintenance initiation and completion, while leaving clinical efficacy, comparative safety, and reasons for treatment progression unresolved.
13. Why This Trial Matters Statistically
This trial illustrates why an endpoint's operational definition and denominator are central to statistical interpretation. The primary endpoint was not simply whether patients responded; it concerned the percentage of maintenance-eligible patients completing at least 3 courses. The registry also states a threshold of at least 50% completion as its criterion for considering the regimen feasible for future study. Readers therefore need to distinguish the endpoint's target population from all enrolled participants and from those who actually started maintenance.
The record reports that none of the participants completed three cycles and provides maintenance-start information by group label. At the same time, it does not give a complete denominator and eligibility accounting for the endpoint percentage. This makes the distinction between a clear descriptive statement and a fully quantified endpoint estimate especially important.
The study also demonstrates the limits of interpreting group-level counts when allocation is not randomized and the model is single-group. The groups labeled 1500, 2000, and 2500 are not controlled comparison arms. Their safety counts can be reported descriptively, but they do not isolate the effect of a dose or regimen from other possible differences among participants.
Finally, the record names several outcomes but does not report an analysis plan or statistical analyses. Without that information, readers cannot determine how outcome multiplicity, missing data, or analysis populations were handled. These are not minor technical details: they shape how precisely and confidently clinical-trial results can be interpreted. The case is a useful reminder that outcome definitions, complete denominators, and transparent analyses are necessary companions to reported counts.
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 does not provide a full accounting of who was eligible for maintenance, the denominator used to calculate the primary percentage, or the reasons participants did not start or complete maintenance. Although it reports that none of the participants completed three cycles and gives counts of maintenance starters by group label, those details do not explain treatment progression or provide a complete endpoint analysis.
The trial is listed as open-label and single-group, with allocation not applicable and no masking. There is no concurrent randomized control group in the reported design. Consequently, descriptive outcomes cannot be used as controlled estimates of comparative efficacy or safety, and the group labels cannot be interpreted as randomized dose comparisons.
The registry record does not report a statistical analysis plan, inferential analyses, confidence intervals, or p-values for the primary endpoint. It does not describe analysis populations, missing-data procedures, or multiplicity handling. Numerical results for the named secondary outcomes of overall survival and response rate are not included in the registry record. The record also does not provide causes of death or treatment-attribution information for the safety events and deaths.
The enrollment figure and completion status establish the scope and status recorded for the study, but they do not resolve these reporting limitations. The sample-size illustrations are planning calculations and are not the trial's design or observed power. These constraints mean the available record is most informative as a descriptive account of maintenance progression and listed safety events, not as a comparative treatment-effect analysis.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: CPI-613, Cytarabine, and Mitoxantrone Hydrochloride in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Granulocytic Sarcoma — NCT02484391.
- Registry record last updated 2024-09-19; status last verified 2024-08.
- Results first posted 2024-09-19.
- 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
NCT02484391 is a completed phase I, open-label, single-group study sponsored by Wake Forest University Health Sciences. Enrollment is listed as 48. The recorded start was 2015-09 and primary completion was 2018-10. The study evaluated CPI-613 with high-dose cytarabine and mitoxantrone in patients with relapsed or refractory AML or granulocytic sarcoma.
The primary endpoint concerned the percentage of patients eligible for maintenance who completed at least 3 courses, with a stated feasibility criterion of at least 50% completing 3 cycles. The registry reports that none of the participants completed three cycles. It also provides counts of participants starting maintenance in groups labeled 1500, 2000, and 2500.
Serious adverse-event counts and selected event terms are listed, along with death counts. The record does not report a statistical analysis plan, inferential analyses, or numerical results for the named response-rate and overall-survival outcomes. The single-group design supports descriptive interpretation but not a randomized comparison.