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 CPX-351 or CLAG-M Regimen for the Treatment of Acute Myeloid Leukemia or Other High-Grade Myeloid Neoplasms in Medically Less-Fit Patients (NCT04195945), a randomized phase 2 study. The primary outcome is 3-month Overall Survival (OS).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT04195945 |
| Phase | Phase 2 |
| Condition | Acute Myeloid Leukemia, Myeloid Neoplasm |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 60 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Fred Hutchinson Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
The trial enrolled medically less-fit adults with acute myeloid leukemia (AML) or other high-grade myeloid neoplasms. These are the conditions named in the registry record. The record describes the primary endpoint population as participants assessed after starting protocol therapy. Further eligibility details and the criteria used to define “medically less-fit” are not reported in the registry record.
Intervention
One randomized arm received CPX-351, also named liposome-encapsulated daunorubicin-cytarabine in the intervention list. The registry describes the trial as evaluating doses typically used for medically fit adults with AML. Detailed treatment descriptions are present for induction, but the registry record does not provide enough complete arm text to characterize all subsequent treatment.
Comparator
The other randomized arm received CLAG-M: cladribine, cytarabine, G-CSF, and mitoxantrone. The registry’s primary-outcome description also refers to attenuated-dose CPX-351 used in a previous institutional trial. That historical comparison is distinct from the two arms listed as randomized in this trial; further details about the earlier trial are not reported in the registry record.
Primary question
The primary endpoint asks for the number of participants alive, deceased, or with unknown status at 3 months after starting protocol therapy. The trial description frames the research question as whether CPX-351 or CLAG-M at doses typically used for medically fit adults improves 3-month overall survival relative to attenuated-dose CPX-351 used previously. The registry record does not report the outcome counts or a statistical comparison, so it does not establish how the randomized arms differed on this endpoint.
3. Trial Design
This is a randomized, open-label, parallel-group phase 2 trial sponsored by Fred Hutchinson Cancer Center. The registry lists enrollment of 60 participants and two treatment arms: CPX-351 and CLAG-M. The adverse-event records list 30 participants at risk in each arm. The parallel design assigns participants to one of the two treatment groups, rather than having each participant receive both regimens in sequence. The allocation method and details of how the random sequence was generated are not reported in the registry record.
The trial was not masked. Participants and trial personnel therefore were not blinded to the assigned regimen according to the registry’s masking entry. This can matter for outcomes that involve judgment or reporting, such as adverse events or questionnaire responses; the record does not describe procedures intended to reduce those risks. The trial includes secondary outcomes for complete remission, MRD-negative complete remission, response duration, relapse-free survival, adverse events, mortality at specified times, and quality of life.
The registry dates the study start to 2020-05-27 and primary completion to 2025-06-06, and lists the status as completed. The registry does not report a sample-size justification or details of the randomization process. Although enrollment is recorded as 60, that number alone does not reveal how many participants contributed data to each endpoint or whether all randomized participants were included in any particular analysis. Those analysis-population details 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 | CPX-351 Induction Therapy | CLAG-M Induction Therapy |
|---|---|---|
| STARTED | 30 | 30 |
| Completed 1 Cycle of Therapy | 23 | 27 |
| Completed 2 Cycles of Therapy | 6 | 1 |
| Completed 3 Cycles of Therapy | 0 | 1 |
| Completed 4 Cycles of Therapy | 1 | 0 |
| Completed 5 Cycles of Therapy | 0 | 1 |
| Completed 6 Cycles of Therapy | 0 | 0 |
| COMPLETED | 30 | 30 |
| NOT COMPLETED | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: Count of participants (and percentage) alive, deceased, and unknown at 3-months after start of protocol therapy
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 | CPX-351 Induction Therapy | CLAG-M Induction Therapy | Total |
|---|---|---|---|---|
| Participants analysed | 30 | 30 | 60 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 5 | 7 | 12 |
| Age, Categorical: >=65 years | Participants | 25 | 23 | 48 |
| Age, Continuous | Years | 72 (58 to 89) | 72 (40 to 83) | 72 (40 to 89) |
| Sex: Female, Male: Female | Participants | 11 | 6 | 17 |
| Sex: Female, Male: Male | Participants | 19 | 24 | 43 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 2 | 0 | 2 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 2 | 2 |
| Race (NIH/OMB): White | Participants | 27 | 26 | 53 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 | 2 | 3 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 | 2 | 3 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 29 | 27 | 56 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 1 | 1 |
| Region of Enrollment: United States | Participants | 30 | 30 | 60 |
6. Primary Endpoint
The registry lists the primary outcome as 3-month Overall Survival (OS), measured over Up to 3 months from date of start of protocol therapy.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is 3-month overall survival, described as the count of participants who are alive, deceased, or of unknown status 3 months after starting protocol therapy. It is a snapshot at a specified time: the record’s stated measure is participant status at that time, not a reported survival curve or a time-to-event estimate. The unit is participants, and the registry description also refers to a percentage for each status category.
General: Overall survival is used to describe whether participants remain alive over a defined period, without restricting the assessment to a particular cause of death. A fixed-time assessment can be clinically interpretable, but a count alone does not show how precisely an arm-level estimate is known or whether observed differences are compatible with chance. Confidence intervals and a comparison between arms would help convey that uncertainty. Neither estimates nor confidence intervals for the primary endpoint are reported in the registry record.
The endpoint is listed as primary, but the registry record does not give the counts of participants alive, deceased, or unknown at the 3-month assessment. It also does not report how unknown status was handled in an analysis. Consequently, the endpoint definition can be described, but its observed result and comparative interpretation cannot be reconstructed from this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a randomized comparison between CPX-351 and CLAG-M in medically less-fit adults with AML or other high-grade myeloid neoplasms. It lists 60 participants enrolled and gives 3-month overall survival as the primary outcome. However, no analysis results are recorded: the number of analyses is listed as zero, and the primary outcome is flagged as having several categories or time points that were not reduced to one comparison. The record therefore does not provide an arm-level estimate or a single statistical contrast for this endpoint.
That absence is important. The trial’s stated objective concerns whether either regimen, at doses typically used for medically fit adults with AML, improves 3-month survival relative to attenuated-dose CPX-351 used in a previous institutional trial. The randomized arms listed for this study are CPX-351 and CLAG-M. The record does not report results for either arm on the primary endpoint, nor does it report a comparison with the historical attenuated-dose group. No conclusion about the direction, magnitude, or precision of a survival difference can be drawn from the registry record.
General: An estimate describes the observed result, while a confidence interval describes uncertainty around that estimate under the statistical model used. A p-value, if reported, would address compatibility of the data with a specified null hypothesis; it would not by itself show the size or clinical importance of an effect. Here, an effect estimate, confidence interval, and p-value for the primary comparison are not reported in the registry record. The listed enrollment and the safety-group counts cannot substitute for those endpoint results.
The record does report adverse-event counts. In the CPX-351 induction group, 2 participants were recorded as affected by serious adverse events among 30 at risk; in the CLAG-M induction group, 6 were affected among 30 at risk. It also lists 22 deaths in the CPX-351 group and 25 in the CLAG-M group. These figures are not identified as the 3-month primary endpoint counts, and the time frame and analysis definition for the adverse-event death counts are not reported in the registry record. They should not be treated as a substitute for the missing primary outcome analysis.
The appropriate statistical reading is therefore descriptive and limited. The registry identifies the design, planned endpoint, treatment groups, and selected safety counts, but not the trial’s primary outcome results. Without outcome estimates and uncertainty measures, the record does not establish whether the groups differed or how precisely any difference was estimated.
9. Statistical Methods Explained
The registry describes a phase 2, randomized, parallel-group trial with no masking. Randomization is a design method intended to make treatment assignment independent of participant characteristics, on average. It supports a causal comparison when the analysis preserves the randomized groups and follow-up is adequately accounted for. The registry does not describe the sequence-generation method, concealment of assignment, stratification factors, or the planned analysis approach. Those details are not reported in the registry record.
General: A parallel-group design compares outcomes between groups assigned to different interventions. In an open-label study, knowledge of assignment can influence care, reporting, or assessment. The possibility is especially relevant to outcomes that depend on judgment or participant reporting. It does not establish that bias occurred, but it is one reason to examine how outcomes were defined and assessed. The registry lists a quality-of-life questionnaire outcome and adverse-event outcomes, but the assessment procedures and any safeguards against assessment bias are not reported in the registry record.
The primary measure is a categorical status count at a fixed time: alive, deceased, or unknown at 3 months after protocol therapy starts. The registry’s skip note says that the primary outcome has several categories or time points and was not reduced to one comparison. It follows that no single binary contrast, such as alive versus not alive, should be attributed to the posted results. Such a recoding would require an analysis definition and data that are not reported in the registry record.
General: A categorical outcome can be summarized using counts and proportions in each group. A comparison may use a test or a model, but the choice depends on the prespecified question, the outcome structure, sample size, and assumptions. For a fixed-time survival status, the analysis must define how unknown status is treated and identify the denominator for each reported proportion. The registry does not report these analysis decisions or the primary outcome counts. No test statistic, p-value, confidence interval, or adjusted estimate for the primary endpoint is reported.
Overall survival can also be analyzed as time to death, with participants who have not experienced the event by the end of follow-up treated as censored under an appropriate method. Such an analysis uses event timing and follow-up information, not only a status count at one time point. The registry defines the primary outcome as status at 3 months and does not report a time-to-event analysis for it. The analysis type should therefore not be inferred from the phrase “overall survival” alone.
The record lists secondary outcomes including complete remission, MRD-negative complete remission, response duration, relapse-free survival, adverse events, mortality at 30 and 60 days, and quality of life. These outcomes have different data structures and may require different summaries or models. For example, binary response outcomes concern whether a participant met a response definition; time-to-event outcomes concern both whether and when an event occurred; and questionnaire outcomes depend on score definitions and assessment timing. The registry record does not report results or the statistical methods for these secondary outcomes.
The record lists 11 outcome measures and zero analyses. That means the registry entry does not give a set of analysis results from which the primary comparison can be assessed. The protocol’s statistical analysis plan, including covariate adjustment, handling of incomplete observations, and any interim analyses, is not reported in the registry record. It would be inappropriate to infer those choices from the trial phase or design.
General: Over-dispersion arises when observed variability exceeds what a chosen model assumes. If present, standard errors from a model that ignores it can be too small, producing confidence intervals that are too narrow and tests that are too liberal. Whether over-dispersion is relevant depends on the outcome and model. The registry record does not identify a model for the primary outcome, so no claim about over-dispersion or its handling can be made for this trial.
The sample-size assumptions describe planning illustrations based on an observed effect, with a two-sided significance level of 0.05, equal allocation, and a normal approximation; the effect to detect is a stated fraction of the observed effect. For time-to-event illustrations, the convention is total events, while other illustrations use participants per arm. These are planning illustrations, not observed power and not evidence that the trial had a particular probability of detecting an effect. The registry record does not report the trial’s own sample-size justification or the observed effect needed to interpret such planning scenarios.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 11 outcome measures, spanning the primary 3-month overall-survival outcome and secondary measures such as remission, response duration, relapse-free survival, adverse events, mortality at 30 and 60 days, and quality of life. Considering many outcomes or time points can create a multiplicity issue: when many statistical tests are performed, the chance of at least one apparently notable result can increase. General statistical approaches include prespecifying a primary comparison, controlling an error rate across selected tests, or treating secondary findings as exploratory. The multiplicity strategy for this trial is not reported in the registry record.
The record lists zero analyses, and it does not provide results for the primary endpoint or secondary outcomes. It also does not report whether all planned comparisons were conducted, whether any outcomes were changed, or whether interim analyses were performed. These omissions prevent evaluation of how the reported outcome set relates to the protocol’s intended testing plan.
Missing-data handling is also not reported in the registry record. This matters for the primary endpoint because the outcome definition includes an unknown status category. The record does not state whether unknown status was retained as a category, excluded from a denominator, imputed, or handled in another way. Nor does it report how missing questionnaire responses or incomplete follow-up were addressed.
The analysis populations are not reported in the registry record. In particular, the record does not state whether analyses used all randomized participants, participants who started treatment, or another defined group. It does not give endpoint-specific denominators beyond the safety records’ at-risk counts. Without these details, readers cannot determine how exclusions or missing observations may have affected estimates, if any were calculated.
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 |
|---|---|---|---|
| CPX-351 Induction Therapy | 2 | 30 | 6.7% |
| CLAG-M Induction Therapy | 6 | 30 | 20.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| CPX-351 Induction Therapy | 22 of 30 |
| CLAG-M Induction Therapy | 25 of 30 |
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 |
|---|---|
| CPX-351 Induction Therapy | 30 of 30 |
| CLAG-M Induction Therapy | 30 of 30 |
Most frequent serious adverse event terms
| Event term | Organ system | CPX-351 Induction Therapy | CLAG-M Induction Therapy |
|---|---|---|---|
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 2 of 30 | 3 of 30 |
| Bacteremia | Infections and infestations | 0 of 30 | 1 of 30 |
| Lung infection | Infections and infestations | 0 of 30 | 1 of 30 |
| Sepsis | Infections and infestations | 0 of 30 | 1 of 30 |
| Tumor lysis syndrome | Metabolism and nutrition disorders | 0 of 30 | 1 of 30 |
| Encephalopathy | Nervous system disorders | 0 of 30 | 1 of 30 |
| Stroke | Nervous system disorders | 0 of 30 | 1 of 30 |
| Hematoma | Vascular disorders | 0 of 30 | 1 of 30 |
Most frequent non-serious adverse event terms
| Event term | Organ system | CPX-351 Induction Therapy | CLAG-M Induction Therapy |
|---|---|---|---|
| Febrile neutropenia | Blood and lymphatic system disorders | 14 of 30 | 15 of 30 |
| Hypertension | Vascular disorders | 6 of 30 | 7 of 30 |
| Bacteremia | Infections and infestations | 2 of 30 | 11 of 30 |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | 6 of 30 | 5 of 30 |
| Sepsis | Infections and infestations | 2 of 30 | 5 of 30 |
| Diarrhea | Gastrointestinal disorders | 1 of 30 | 5 of 30 |
| Hypotension | Vascular disorders | 2 of 30 | 4 of 30 |
| Lung infection | Infections and infestations | 3 of 30 | 2 of 30 |
| Mucositis oral | Gastrointestinal disorders | 3 of 30 | 2 of 30 |
| Acute kidney injury | Renal and urinary disorders | 1 of 30 | 3 of 30 |
Serious events with the highest arm-level rate: respiratory failure (10.0%), bacteremia (3.3%), lung infection (3.3%), sepsis (3.3%), tumor lysis syndrome (3.3%).
The registry lists serious adverse-event information by induction group. For CPX-351, 2 participants were affected among 30 at risk; for CLAG-M, 6 were affected among 30 at risk. The record separately lists 30 participants at risk and 30 affected by other adverse events in each group. These are descriptive counts in the registry record, not adjusted comparisons. No confidence intervals or statistical tests for these safety counts are reported.
The listed deaths are 22 in the CPX-351 induction group and 25 in the CLAG-M induction group. The registry does not identify these counts as the primary 3-month overall-survival outcome, and the time frame and definition for the adverse-event death counts are not reported in the registry record. They should not be interpreted as the trial’s 3-month alive, deceased, and unknown categories.
The most frequent serious-event terms listed include respiratory failure at 10.0%, and bacteremia, lung infection, sepsis, tumor lysis syndrome, and encephalopathy at 3.3% each. These percentages are reproduced as recorded; the record does not provide enough detail to infer the full event-specific denominators or establish a comparative risk. Serious-event counts describe what was recorded, but without timing, attribution, and analysis details they do not establish whether an event was caused by a regimen.
12. What the Result Does, and Does Not, Mean
The registry record supports a description of the trial’s design and the outcomes it intended to assess. It identifies a randomized phase 2 comparison of CPX-351 and CLAG-M, an enrollment of 60, and a primary endpoint defined as participant status 3 months after starting protocol therapy. It also records selected adverse-event counts. These registry entries describe the study structure and some posted safety information, but they do not provide the primary outcome results.
The record does not show the number alive, deceased, or unknown at 3 months in either arm. It does not report an estimate of the difference between arms, a confidence interval, or a statistical test for the primary endpoint. As a result, it cannot establish the direction or size of a difference in 3-month overall survival, or the precision with which a difference was estimated. The registry’s stated objective and endpoint definition are not themselves evidence of an observed treatment effect.
The adverse-event figures also have limits. Serious-event counts and listed deaths are descriptive, and the death counts are not identified as the primary endpoint results. Without a reported time frame, definitions, or comparative analysis, these numbers cannot answer the primary survival question. The listed event percentages likewise do not, by themselves, establish that one regimen caused more or fewer events than the other.
General: A randomized design can strengthen a treatment comparison, but interpretation still depends on actual outcome data, complete follow-up, clearly defined analysis populations, and suitable uncertainty estimates. Statistical significance, if available, would not alone determine clinical importance; effect size and precision would also matter. In this record, those primary efficacy results are not reported, so neither statistical nor clinical conclusions about comparative efficacy can be drawn.
Accordingly, the defensible conclusion is narrow: the registry documents a completed trial with a defined randomized comparison and a stated 3-month endpoint, while leaving the endpoint analysis unavailable. The record does not show whether either regimen improved survival or whether the groups had similar outcomes.
13. Why This Trial Matters Statistically
This trial raises a useful statistical distinction between a clearly stated research question and an assessable result. The registry describes a randomized comparison and specifies a primary endpoint, but the endpoint has several status categories and the record does not reduce them to a single comparison. Without arm-specific counts and an analysis, readers cannot quantify the outcome or its uncertainty. This makes transparent outcome reporting essential to interpreting a clinical trial.
The design also illustrates why the target population and comparator need to be kept clear. The randomized arms are CPX-351 and CLAG-M, while the primary-outcome description refers to comparison with attenuated-dose CPX-351 used in a previous institutional trial. A historical reference and a concurrently randomized group are different kinds of comparison. The registry record does not provide enough information to estimate or evaluate the historical comparison.
General: A trial’s enrollment does not, by itself, show whether it was large enough to estimate an effect precisely. Precision depends on the number of observed outcomes, allocation, follow-up, and the analysis method, among other factors. Planning illustrations based on an observed effect should not be interpreted as observed power. The record does not report the trial’s own sample-size justification or primary outcome estimates, so the adequacy of the design for its stated endpoint cannot be assessed from the registry entry.
The safety records offer descriptive information, but they also demonstrate the need for context: event definitions, follow-up windows, attribution, and denominators shape interpretation. These considerations apply especially when counts are read alongside an overall-survival endpoint but are not themselves the endpoint analysis. Clear reporting of these details, together with the planned analysis and outcome estimates, would be needed for a fuller statistical assessment.
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 central limitation of the registry record is the absence of primary-outcome results. Although 3-month overall survival is identified as the primary endpoint, the record does not give the counts of participants alive, deceased, or with unknown status, or a comparison between randomized arms. No effect estimate, confidence interval, or test result for the primary endpoint is reported. This prevents assessment of both the observed outcome and its precision.
The record also leaves important design and analysis details unspecified. The randomization method, allocation concealment, eligibility criteria beyond the broad population description, sample-size justification, analysis populations, and handling of missing data are not reported in the registry record. The protocol’s statistical analysis plan and any interim analyses are also not reported. The trial is listed as open-label, but the record does not describe outcome-assessment safeguards.
Some arm descriptions are incomplete in the registry entry, limiting a full account of treatment after induction. The primary-outcome description refers to a historical attenuated-dose CPX-351 comparison, but corresponding historical results and comparative methods are not reported. The adverse-event counts do not specify the time frame for the listed deaths, and the record does not provide inferential safety comparisons.
Finally, the registry lists secondary outcomes but does not report their results or analysis methods. These limitations mean the record can establish the trial’s stated design and selected descriptive information, but it cannot support a comprehensive efficacy or safety assessment.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: CPX-351 or CLAG-M Regimen for the Treatment of Acute Myeloid Leukemia or Other High-Grade Myeloid Neoplasms in Medically Less-Fit Patients — NCT04195945.
- Registry record last updated 2026-06-29; status last verified 2026-06.
- Results first posted 2026-05-14.
- 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
NCT04195945 is a completed randomized, open-label, parallel-group phase 2 trial sponsored by Fred Hutchinson Cancer Center. It enrolled 60 medically less-fit adults with AML or other high-grade myeloid neoplasms. The two listed arms received CPX-351 or CLAG-M. The registry gives 3-month overall survival as the primary endpoint, defined by counts of participants alive, deceased, or unknown at 3 months after protocol therapy began.
The registry lists 11 outcome measures and zero analyses. It does not report the primary endpoint counts, an arm comparison, or uncertainty estimates. Safety records list serious adverse events affecting 2 of 30 participants in the CPX-351 group and 6 of 30 in the CLAG-M group, as well as death counts of 22 and 25, respectively; the death-count time frame is not reported. These descriptive entries do not replace the missing primary endpoint analysis.