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 A Study of E7820 in People With Bone Marrow (Myeloid) Cancers (NCT05024994), a na phase 2 study. The primary outcome is Median Overall Survival.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT05024994 |
| Phase | Phase 2 |
| Condition | Acute Myeloid Leukemia, Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 12 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Memorial Sloan Kettering Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
People with relapsed or refractory myeloid malignancies involving mutations in splicing-factor genes. The listed conditions are acute myeloid leukemia, myelodysplastic syndromes, and chronic myelomonocytic leukemia. The registry record does not give further eligibility details or identify the mutations included.
Intervention
E7820, administered daily. The described starting dose is 100 mg daily, with the dose potentially reduced if excessive toxicity is encountered. The registry record does not report how many participants received each dose or whether dose reductions occurred.
Comparator
There is no separate comparator group in the registered design. The study is listed as single-group, with allocation marked NA and no masking. It therefore does not make a randomized, like-for-like comparison between E7820 and another treatment or control.
Primary question
The primary outcome is median overall survival, with a stated timeframe of 1 year. The study description says it aims to assess 1 year overall survival. The registry record does not report the median estimate, its confidence interval, or an analysis result, so the registered question cannot be answered from the posted results alone.
This is a study of a defined clinical setting rather than a broad assessment of all myeloid cancers: participants were to have relapsed or refractory disease and mutations in splicing-factor genes. The record names three disease categories, but does not state how participants were distributed among them. Enrollment was 12. That small cohort means the registered experience is specific to the people enrolled and cannot, by itself, establish how outcomes would compare with those under another treatment. No comparative effect estimate is reported.
3. Trial Design
The registry describes a Phase II, single-group clinical trial sponsored by Memorial Sloan Kettering Cancer Center. Enrollment was 12. The conditions listed are acute myeloid leukemia, myelodysplastic syndromes, and chronic myelomonocytic leukemia, in people with relapsed or refractory disease and mutations in splicing-factor genes. The study status is completed; the recorded start date is 2021-08-13 and the primary completion date is 2025-06-19.
All participants were assigned to the E7820 group in the registered design. E7820 is listed as a drug intervention, administered daily. The arm description gives a starting dose of 100 mg daily and says the dose could subsequently be reduced if excessive toxicity was encountered. The registry record does not report the doses actually taken, treatment duration, adherence, or the number of dose reductions.
Allocation is marked NA, masking is listed as none, and the model is single-group. These characteristics mean the design does not include a randomized control arm or a blinded treatment comparison. Outcomes describe the enrolled group; they cannot isolate a treatment effect from other influences using a concurrent randomized comparison. There is no like-for-like control comparison in this design.
The primary outcome measure is titled Median Overall Survival, with months as the unit, a 95% confidence interval as the dispersion measure, and a timeframe of 1 year. Its description is to assess 1 year overall survival. Although the record lists this outcome, it does not post the median, confidence limits, or an analysis result. The statistical analysis plan, eligibility criteria beyond the summary, follow-up details, and handling of participants with incomplete follow-up 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 | E7820 |
|---|---|
| STARTED | 12 |
| COMPLETED | 12 |
| NOT COMPLETED | 0 |
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 | E7820 |
|---|---|---|
| Participants analysed | 12 | |
| Age, Continuous | years | 77 (71 to 85) |
| Sex: Female, Male: Female | Participants | 2 |
| Sex: Female, Male: Male | Participants | 10 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 11 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 1 |
| Race (NIH/OMB): Asian | Participants | 0 |
| 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 | 9 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Region of Enrollment: United States | Participants | 12 |
6. Primary Endpoint
The registry lists the primary outcome as Median Overall Survival, measured over 1 year.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 95% Confidence Interval |
| Unit | months |
| Endpoint classification | Time-to-Event Analysis |
Overall survival describes the time from a defined starting point until death from any cause. The registry identifies the primary outcome as median overall survival, measured in months, and gives a timeframe of 1 year. The median is the point in the estimated survival-time distribution at which half the group has experienced the event and half has not; it is a summary of time, not a percentage alive at a particular time. This is a general explanation of the measure.
Overall survival is used in clinical research because death is an event that can be defined without deciding whether a particular cause is related to the disease or treatment. Time-to-event measures can also make use of follow-up information when participants have different lengths of observation, provided the analysis handles censoring appropriately. The method used to estimate survival and the rules for censoring are not reported in the registry record.
The record says that the endpoint is intended to assess 1 year overall survival, but it does not report a median estimate or its 95% confidence interval. It is therefore not possible to describe the observed survival time or its precision from the posted results. The registry record also does not specify the time origin used for measuring survival.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The available record describes a completed, single-group Phase II study with enrollment of 12 and a primary endpoint of median overall survival. It does not report the median overall-survival estimate, the 95% confidence interval, or a statistical analysis result. Consequently, no numerical conclusion about the observed survival time or the precision of that estimate can be drawn from the posted results. The absence of a result in the registry record should not be mistaken for a value of zero or for evidence that no events occurred.
The design is also important to interpretation. A single-group study has no concurrent randomized comparator. Any outcome observed among participants would describe their experience during the study, but would not provide a randomized estimate of how outcomes differ from those under another treatment. Differences from outcomes in other populations or studies would be difficult to interpret because patient characteristics, eligibility, follow-up, and other aspects of care may differ. The registry record does not identify a comparison group or report a comparative effect estimate.
The registered endpoint is time-to-event in nature, and the record specifies a confidence interval as its dispersion measure. In general, a confidence interval expresses uncertainty around an estimate under the assumptions of the analysis. A narrower interval indicates greater statistical precision than a wider interval, but neither its width nor its location can be discussed for this study because the interval itself is not reported. The method used to calculate it is also not reported in the registry record.
The safety entries give counts for the E7820 group: 12 participants at risk for serious adverse events, 9 affected by serious adverse events, 12 at risk for other adverse events, 10 affected by other adverse events, and 5 deaths. These are descriptive counts in a small enrolled group. The record lists serious-event terms and percentages, but does not provide event-level details, timing, severity grades, or causal assessments. The count of deaths does not by itself establish that the deaths were caused by E7820; the registry does not report that attribution.
The record lists one outcome measure and no analysis entries. It does not report an analysis plan, the analysis population, missing-data procedures, interim analyses, or a sample-size justification. These omissions limit assessment of how the endpoint was analyzed and how complete the follow-up was. The study status is completed, but completion status does not substitute for a posted endpoint result.
A separate sample-size illustration may describe hypothetical planning under stated assumptions, including equal allocation between two arms. Such an illustration is not the design of this single-group trial, does not represent observed power, and cannot supply a missing survival result. The registry record does not report an actual comparative effect or the numerical estimates needed to interpret such a calculation. Overall, the posted record supports a description of the study design and reported safety counts, but not a numerical assessment of the primary survival endpoint or a comparative treatment effect.
9. Statistical Methods Explained
The registry identifies median overall survival as the primary outcome and a 95% confidence interval as its dispersion measure. It does not give an outcome estimate, confidence limits, or analysis entries. The descriptions below explain general statistical concepts; they do not identify methods actually used in this study unless the registry record states them.
What does a median survival measure?
In general, a time-to-event analysis follows participants from a specified starting point until an event occurs or follow-up ends. For overall survival, the event is death from any cause. The median survival time is the time at which the estimated survival distribution reaches its midpoint. It summarizes the timing of events, rather than counting only how many events occurred, and it does not show the individual experiences within the group.
Time-to-event methods are useful when participants have varying lengths of follow-up. Some may still be alive when observation ends or may stop contributing follow-up for another reason. Such observations are commonly treated as censored: the event time is not known, but the participant contributes information up to the last point at which their status is known. This is a general description. The registry record does not report how censoring was defined or handled, whether anyone was censored, or the time origin for overall survival.
What does a confidence interval communicate?
A confidence interval describes statistical uncertainty around an estimated quantity under a specified analysis and its assumptions. In general, it gives a range of values compatible with the observed data and the interval procedure. It is not a guarantee that the true value falls inside that particular interval, and it does not account automatically for every source of uncertainty, such as limitations in study design or incomplete reporting.
The registered dispersion measure is a 95% confidence interval, but the interval endpoints are not reported. The estimation method, assumptions, and treatment of censored observations are also not reported in the registry record. Without an estimate and its interval, the survival result cannot be assessed for precision or compared with a clinically relevant threshold.
Why does the single-group design matter?
In general, randomization to a concurrent comparator can help balance measured and unmeasured characteristics between groups, supporting a less biased estimate of a treatment contrast. This trial is registered as single-group, with allocation marked NA and no masking. It therefore provides no randomized contrast. An outcome in the E7820 group is descriptive of that enrolled group and cannot, on its own, establish what would have happened to the same participants under a different treatment.
The design also means that a two-arm planning calculation should not be confused with the trial's actual design. The record describes an illustration based on two-sided significance level 0.05, equal allocation between two arms, and a normal approximation, with effects expressed as fractions of an observed effect. For time-to-event calculations, the stated planning quantity is the total number of events under the Schoenfeld method; other calculations are described in participants per arm. These are planning assumptions, not a description of the enrolled single-group study. No numerical planning values are stated in the registry record summarized for this analysis, and such values would not be observed power.
What do event counts and percentages tell the reader?
Adverse-event counts describe how many participants in the listed group were at risk and how many were recorded as affected. They do not necessarily describe the total number of individual episodes, since one participant may have more than one event. The registry presents serious adverse-event terms with percentages, but does not explain the counting rules, whether the terms refer to participants or episodes, or the duration of observation used as the denominator. The percentages should therefore be read as reported rather than reinterpreted as incidence rates or comparative risks.
General statistical interpretation also distinguishes an event occurring during a study from an event caused by the intervention. Attribution requires clinical assessment and defined reporting procedures. The listed safety data do not provide causal assessments, and the count of deaths should not be treated as a count of deaths caused by E7820.
What cannot be evaluated from the analysis record?
The registry lists one outcome measure and no analysis entries. It does not report the survival estimation procedure, confidence-interval method, statistical model, covariate adjustment, handling of missing data, analysis population, or any planned interim analysis. It also does not report a protocol-level statistical plan or a sample-size justification. These omissions prevent a check of whether the reported endpoint was analyzed as planned or how sensitive the result might be to analytic choices. Since no numerical survival result is posted, there is no effect estimate to recalculate or compare with a sponsor analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one outcome measure, Median Overall Survival, and no analysis entries. That record does not establish that the study had only one outcome or one statistical test in its full protocol; it describes what is registered in the posted outcome information. A multiplicity plan, including any hierarchy among outcomes or adjustment for multiple tests, is not reported in the registry record.
In general, multiplicity arises when several hypotheses or outcomes are tested and the chance of at least one apparently positive result can increase across those tests. Statistical plans may address this by defining a primary comparison, controlling an error rate, or treating additional analyses as exploratory. No such approach is reported for this trial. Because the primary outcome result itself is not posted, there are no reported tests or p-values to interpret for multiplicity.
The analysis population is not reported. The registry does not say whether analyses were planned for all enrolled participants, those who began treatment, or another defined group. It also does not describe whether participants who discontinued, had incomplete follow-up, or lacked endpoint information were included in any analysis.
Missing-data methods and the extent of missing endpoint information are not reported in the registry record. For a time-to-event endpoint, incomplete follow-up can be handled through censoring under assumptions about how follow-up ends and how that relates to future event risk. This is a general point, not a claim about the methods used in this study. The record does not report censoring rules, the amount of follow-up, or the number of participants with unknown status.
Interim analyses and stopping rules are also not reported. The study status is completed, but that status alone does not indicate whether interim monitoring occurred or how completion decisions were made.
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 |
|---|---|---|---|
| E7820 | 9 | 12 | 75.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| E7820 | 5 of 12 |
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 |
|---|---|
| E7820 | 10 of 12 |
Most frequent serious adverse event terms
| Event term | Organ system | E7820 |
|---|---|---|
| Death NOS | General disorders | 3 of 12 |
| Lung infection | Infections and infestations | 3 of 12 |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 2 of 12 |
| Sepsis | Infections and infestations | 2 of 12 |
| Anemia | Blood and lymphatic system disorders | 1 of 12 |
| Dysphagia | Gastrointestinal disorders | 1 of 12 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 1 of 12 |
| Pruritus | Skin and subcutaneous tissue disorders | 1 of 12 |
| Urinary tract pain | Renal and urinary disorders | 1 of 12 |
| Vaginal discharge | Reproductive system and breast disorders | 1 of 12 |
Most frequent non-serious adverse event terms
| Event term | Organ system | E7820 |
|---|---|---|
| Diarrhea | Gastrointestinal disorders | 6 of 12 |
| Cough | Respiratory, thoracic and mediastinal disorders | 4 of 12 |
| Non-cardiac chest pain | General disorders | 3 of 12 |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | 3 of 12 |
| Fall | Injury, poisoning and procedural complications | 3 of 12 |
| Nausea | Gastrointestinal disorders | 3 of 12 |
| Anemia | Blood and lymphatic system disorders | 2 of 12 |
| Neutropenia | Blood and lymphatic system disorders | 2 of 12 |
| Abdominal pain | Gastrointestinal disorders | 2 of 12 |
| Fever | General disorders | 2 of 12 |
Serious events with the highest arm-level rate: death nos (25.0%), lung infection (25.0%), respiratory failure (16.7%), sepsis (16.7%), anemia (8.3%).
The registry reports safety figures for the E7820 group. It lists 12 participants at risk for serious adverse events and 9 affected; for other adverse events, it lists 12 at risk and 10 affected. The listed death count is 5. These are participant counts, not comparative estimates, because the study has no separate control group. The record does not report event timing or the duration over which participants were monitored.
The serious adverse-event terms with the highest listed percentages are Death NOS and Lung infection, each 25.0%; Respiratory failure and Sepsis, each 16.7%; and Anemia and Dysphagia, each 8.3%. These percentages are reproduced as registry entries. The record does not explain whether terms count participants or episodes, whether a participant could contribute to multiple terms, or the exact denominator used for each percentage. They should not be added together or interpreted as distinct proportions of the group.
Serious adverse events and other adverse events are separate reporting categories in the record. A serious event is not necessarily caused by the study drug, and an event occurring during treatment does not by itself establish causation. The registry does not report causal assessments, severity grades, individual case details, or whether the deaths were considered treatment-related. The safety information therefore describes reported counts and terms, not a causal comparison or a complete account of risk.
12. What the Result Does, and Does Not, Mean
The registry record documents a completed Phase II, single-group study of daily E7820 in people with relapsed or refractory myeloid malignancies with splicing-factor gene mutations. It records enrollment of 12 and identifies median overall survival as the primary outcome, with a 1 year timeframe and a 95% confidence interval as the intended measure of dispersion. The posted record does not give the median, confidence interval, or an analysis result.
That absence means the primary endpoint cannot be quantified from the posted results. It is not appropriate to infer that survival was short or long, that the endpoint was met or not met, or that an unreported result has a particular direction. The record also does not provide an analysis population, follow-up description, or methods for handling incomplete observation, all of which would help readers evaluate a time-to-event result.
The single-group design does not provide a randomized comparison with another treatment. Even if an outcome estimate were available, it would describe the enrolled group and would not by itself show what would have happened under a different treatment. The registry does not report a hazard ratio or other comparative estimate. A hypothetical two-arm planning illustration is not evidence of a comparison observed in this study.
The adverse-event figures describe reported safety counts and selected terms, including 5 deaths, but do not establish that E7820 caused any event. The record does not state whether deaths were attributed to treatment or provide the detailed context needed to assess individual events. Those counts should be read as descriptive rather than causal.
In general, a study's interpretation depends on its design, the completeness of outcome reporting, the analysis methods, and the precision of estimates. Here, the design and some safety counts are available, but the primary survival estimate and important analysis details are not reported in the registry record. The posted information therefore supports a description of the study and its reported safety entries, but not a conclusion about the magnitude or comparative effect of E7820 on overall survival.
13. Why This Trial Matters Statistically
This trial is statistically notable because it asks a time-to-event question in a small, single-group Phase II study. The registered primary outcome, median overall survival, is a summary of event timing, and its interpretation depends on follow-up and censoring as well as on the observed deaths. A confidence interval would help communicate uncertainty around the estimate, but neither the estimate nor its interval is reported in the registry record.
The single-group design places a clear boundary on what the data can establish. It can describe outcomes among enrolled participants, but it cannot produce a randomized comparison with a control group. This distinction matters when readers encounter planning calculations that assume two arms: those calculations are hypothetical illustrations, not a representation of the registered trial's allocation or an observed treatment contrast.
Small enrollment also makes transparent reporting especially important. The registry records enrollment of 12, but does not post the primary endpoint estimate or explain the analysis population, missing-data approach, or survival estimation method. Without those details, readers cannot judge precision, assess how incomplete follow-up was handled, or reproduce an analysis from the registry information.
The safety entries further illustrate the difference between descriptive reporting and causal inference. Counts and listed event terms can describe what was recorded in the E7820 group, but without a comparator and without reported attribution they cannot establish excess risk or drug causation. The death count is particularly important to read in that descriptive context.
Statistically, the record is most useful for defining the intended population, intervention, design, and endpoint, and for showing which safety counts were posted. Its limitations are also informative: a registered outcome without a posted estimate cannot answer the endpoint question, and a single group cannot supply a randomized treatment contrast.
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 primary endpoint is identified, but its result is not reported in the registry record. No median overall-survival estimate, 95% confidence interval, or analysis result is available. The record also does not specify the time origin for measuring survival, the survival estimation method, or how censoring was handled. These omissions prevent evaluation of the primary outcome's magnitude, precision, and analysis assumptions.
Enrollment was 12, and the study has no separate comparator group. It is therefore limited to describing the enrolled group and does not provide a randomized estimate of a difference from another treatment. The record does not give the number of participants in each disease category, the detailed mutation eligibility criteria, or additional participant characteristics. Generalizability beyond the defined relapsed or refractory population with splicing-factor gene mutations cannot be assessed from the registry information alone.
The arm description states a starting dose of 100 mg daily and allows dose reduction if excessive toxicity is encountered. The registry record does not report actual treatment exposure, dose reductions, treatment duration, adherence, or reasons for discontinuation. It also does not describe the frequency or duration of safety monitoring.
Safety reporting includes counts and selected serious-event terms, but does not provide event-level records, severity grades, timing, or causal attribution. The denominator and counting rules for the listed percentages are not explained. The five reported deaths cannot be assumed to be treatment-related.
Finally, the registry does not report the analysis population, missing-data procedures, multiplicity approach, interim analyses, or sample-size justification. Study completion status does not fill these reporting gaps. The planning assumptions for hypothetical two-arm calculations should not be interpreted as the trial's design or as observed evidence.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study of E7820 in People With Bone Marrow (Myeloid) Cancers — NCT05024994.
- Registry record last updated 2026-06-17; status last verified 2025-06.
- Results first posted 2026-06-17.
- 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
NCT05024994 is a completed Phase II, single-group study sponsored by Memorial Sloan Kettering Cancer Center. It enrolled 12 people with relapsed or refractory myeloid malignancies involving splicing-factor gene mutations. Listed conditions are acute myeloid leukemia, myelodysplastic syndromes, and chronic myelomonocytic leukemia.
The intervention was daily E7820, with a starting dose of 100 mg and possible dose reduction for excessive toxicity. The design is unmasked and has no separate comparator group. The recorded start date is 2021-08-13, and the primary completion date is 2025-06-19.
The primary outcome is median overall survival, measured in months, with a 95% confidence interval and a stated timeframe of 1 year. The registry record does not report the endpoint estimate, confidence limits, or analysis result. Safety entries list 9 of 12 participants affected by serious adverse events, 10 of 12 affected by other adverse events, and 5 deaths; the record does not establish causation. Statistical analysis details and missing-data methods are not reported in the registry record.