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 JNJ-40346527 in Treating Participants With Relapsed or Refractory Acute Myeloid Leukemia (NCT03557970), a na phase 2 study. The primary outcome is Best Objective Response Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03557970 |
| Phase | Phase 2 |
| Condition | Recurrent Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 3 |
| Arms in the primary outcome | n/a |
| Lead sponsor | OHSU Knight Cancer Institute |
| Results posted | Yes |
2. Clinical Question
Population
Participants with recurrent or refractory acute myeloid leukemia. The registry record describes a Phase 2 study and records enrollment of 3 participants. It does not report further population details such as age or other eligibility characteristics in the information available for this analysis.
Intervention
JNJ-40346527, also called edicotinib in the brief summary. The treatment description specifies oral dosing twice daily on days 1-28, with 28-day cycles repeated in the absence of disease progression or unacceptable toxicity. A pharmacokinetic study is also listed.
Comparator
There is no separate control group: the study is listed as single-group, with allocation recorded as not applicable and no masking. The assay-positive and assay-negative labels in the adverse-event data are not presented as randomized treatment groups or as a like-for-like control comparison.
Primary question
Among participants who meet the protocol’s evaluability criteria, what is the best objective response during the first 2 cycles of study drug? The record defines response as a partial response or any listed type of complete response. It indicates that no participant was evaluable for this endpoint.
3. Trial Design
This was a Phase 2, open-label, single-group study of JNJ-40346527 in participants with relapsed or refractory acute myeloid leukemia. The sponsor was OHSU Knight Cancer Institute. The registry record lists enrollment of 3 and describes one treatment regimen: JNJ-40346527 by mouth twice daily on days 1-28. Cycles were to repeat every 28 days if disease progression or unacceptable toxicity did not occur. A pharmacokinetic study is also listed as an intervention.
Allocation is recorded as not applicable, masking as none, and the model as single group. There is therefore no concurrent control arm described for comparing response or safety. The assay-positive and assay-negative labels in the adverse-event record do not change that design: they are not identified as randomized groups, and they should not be read as a treatment-versus-control contrast.
The study began on 2018-10-05, and the primary completion date is 2020-09-28. Its status is terminated. The reason for termination is not reported in the registry record. Nor is the planned enrollment target or a sample-size justification reported in the registry record. Enrollment is a record of the participants entered, not evidence that the endpoint analysis included that number.
The primary outcome was best objective response during the first 2 cycles. The registry description says participants needed to complete at least 1 cycle and have response measurements to be evaluable. The record states that the only participant with a recorded disease-response designation did not complete 1 cycle, and it indicates that there were no evaluable participants for objective response. Secondary outcomes listed include adverse events, duration of response, event-free survival, and overall survival. Results analyses for those outcomes are not reported in the registry record in the information available for this analysis.
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.
Before assignment: All enrolled participants were included in the study and assigned to 1 of 2 arms according to their ex vivo sensitivity to study drug. Consented patients with indeterminate ex vivo sensitivity were considered screen failures. Although this was designed as a 2-arm trial (for purposes of stopping early for futility at the end of stage 1 of the study and of correlating pre-treatment ex vivo sensitivity with post-treatment clinical response), the study drug regimen was identical in the 2 arms.
Overall Study
| Stage | Assay Positive | Assay Negative |
|---|---|---|
| STARTED | 1 | 2 |
| COMPLETED | 0 | 0 |
| NOT COMPLETED | 1 | 2 |
Reasons for not completing this period:
| Reason | Assay Positive | Assay Negative |
|---|---|---|
| Study closure | 1 | 0 |
| Physician Decision | 0 | 1 |
| Death | 0 | 1 |
Analysis population for the primary outcome, as defined in the registry: According to the protocol, only those participants who have completed at least 1 cycle of study agent and have response measurements will be evaluable for objective response. Since the only participant with a recorded disease response designation did not complete 1 cycle of study drug, there are no evaluable participants for this trial's primary outcome.
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 | Assay Positive | Assay Negative | Total |
|---|---|---|---|---|
| Participants analysed | 1 | 2 | 3 | |
| Age, Continuous | years | 72 (72 to 72) | 60 (57 to 63) | 63 (57 to 72) |
| Sex: Female, Male: Female | Participants | 1 | 0 | 1 |
| Sex: Female, Male: Male | Participants | 0 | 2 | 2 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 0 | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 1 | 2 | 3 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 1 | 2 | 3 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Best Objective Response Rate, measured over first 2 cycles of study drug.
| 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 endpoint, best objective response rate, is intended to describe the proportion of evaluable participants whose best recorded disease status meets the response definition during the first 2 cycles of study drug. The registry defines an objective response as a partial response or any listed type of complete response: CR, CRm, CRc, or CRi. It states that each participant’s best response designation during that period would be used to compute the rate, alongside an exact confidence interval.
Response endpoints can summarize whether disease measurements meet prespecified response criteria over a defined assessment window. They are not the same as duration of response, event-free survival, or overall survival, which address different aspects of outcomes over time. The registry lists those as secondary outcomes, but their results are not reported in the registry record in the information available for this analysis.
Here, the endpoint cannot be estimated from the recorded response information. The registry says the only participant with a recorded disease-response designation did not complete 1 cycle, while the protocol required completion of at least 1 cycle and response measurements for evaluability. The record indicates no evaluable participants. Consequently, no objective response rate or exact confidence interval is reported in the registry record. This absence should not be interpreted as a response rate of zero: there is no qualifying evaluable population from which to calculate the endpoint.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The main statistical feature of this record is not an estimated response rate but the absence of evaluable data for the primary endpoint. The registry describes best objective response during the first 2 cycles and requires at least 1 completed cycle plus response measurements for evaluability. It also states that the only participant with a recorded disease-response designation did not complete 1 cycle. The record indicates there were no evaluable participants. Without an evaluable denominator and response classifications within that population, the response rate and its uncertainty cannot be estimated.
This is different from observing no responses among a group of evaluable participants. A zero response rate would require an evaluable denominator and a report that no one in that denominator met the endpoint definition. The registry record does not provide that combination of information. It therefore supports neither a numerical response estimate nor a conclusion that the intervention did or did not produce responses.
The study is single-group and open-label, with no masking and allocation recorded as not applicable. Even if endpoint data had been available, a single-group result would describe outcomes in participants treated under this study’s conditions; it would not by itself provide a randomized comparison with another treatment or usual care. In this record, the lack of primary endpoint data makes even a descriptive response estimate unavailable. No causal comparison should be inferred from the assay-positive and assay-negative labels in the safety data, which are not described as randomized treatment arms.
The enrollment figure is 3, but it should not be treated as the denominator for the primary response calculation. The protocol’s stated evaluability rule and the recorded completion information are relevant to that endpoint, and the registry indicates no participants were evaluable. The reason for termination, details of follow-up, and any further explanation for missing endpoint data are not reported in the registry record.
Safety information is recorded separately. The adverse-event figures include small counts in assay-positive and assay-negative categories, including a recorded death in the assay-negative category. These figures are descriptive registry entries; the record does not establish a causal relationship between an adverse event and treatment or explain the circumstances of the death. The catheter-related infection is listed among the top serious terms. The available information does not support broader safety conclusions.
Planning illustrations based on an observed effect are not a substitute for observed endpoint evidence. In this study, the primary response endpoint has no evaluable participants, and the registry does not report an observed effect for that endpoint. The record’s stated illustration assumptions do not establish the study’s original design rationale, a clinically meaningful target, or observed power. Those details are not reported in the registry record.
9. Statistical Methods Explained
What does an objective response rate represent?
As a general statistical concept, a rate for a binary endpoint is the number of participants meeting a defined outcome divided by the number eligible for that analysis. Both pieces matter: the outcome definition determines which observations count as responses, and the analysis population determines who belongs in the denominator. A rate without a clear denominator is not interpretable as an estimate of the response probability in a defined group.
For this study, the registry specifies that response means a partial response or one of the listed complete-response categories during the first 2 cycles. It also describes an evaluability condition: completion of at least 1 cycle and availability of response measurements. The only participant with a recorded response designation did not complete 1 cycle, and the record indicates there were no evaluable participants. Thus, the primary endpoint’s calculation cannot be made from the recorded evaluable population.
Why would a confidence interval accompany the rate?
As a general concept, a confidence interval expresses the uncertainty around an estimate that could vary from sample to sample. It does not remove uncertainty, guarantee that the true value lies within a particular interval, or correct for bias in how participants entered the study. The registry says an exact confidence interval would accompany the response rate for each arm separately. Because there are no evaluable participants for this endpoint, the record does not report a rate or interval to interpret.
Exact methods are often used for a binary outcome when the data are sparse, because methods based on large-sample approximations can behave poorly when counts are small. This is a general explanation, not a statement about additional methods used in this study. The registry does not report a calculated interval or further details of the calculation.
How does the study design shape interpretation?
In general, a single-group study can describe outcomes among participants receiving the study regimen, but it does not create a concurrent control-group estimate of what would have happened under another treatment. Open-label means masking is absent as recorded in the registry. The record also lists allocation as not applicable. These features matter because observed outcomes in a single group cannot, on their own, separate treatment effects from other influences on outcome. In this case, the primary endpoint is not estimable in any event.
The registry describes the regimen as oral dosing twice daily on days 1-28, with 28-day cycles repeated in the absence of progression or unacceptable toxicity. That description records the intended treatment schedule; it does not establish how much treatment each participant received, whether doses were missed, or why a participant did not complete 1 cycle. Those details are not reported in the registry record.
What do the safety counts describe?
As a general concept, adverse-event counts should be read with their group definitions and denominators in mind. A count of affected participants is not the same as a count of separate events, and a percentage can be difficult to interpret if its denominator or counting method is unclear. Here, the adverse-event record labels categories Assay Positive and Assay Negative and provides at-risk and affected counts for serious and other events. These categories should not be treated as randomized treatment groups: the trial is recorded as single-group, with no allocation to a comparator.
The registry lists catheter-related infection as a top serious term with 100.0%. The information available does not explain the term’s precise denominator or whether the percentage refers to events or participants. As a result, it should be read as a registry-reported figure, not converted into a broader risk estimate. The adverse-event entries do not establish that treatment caused an event.
What do planning illustrations mean?
As a general statistical concept, a sample-size calculation depends on assumptions about the effect to detect, the variability or event pattern, the significance level, power, and allocation. An illustration based on an observed effect is conditional on the effect estimate being meaningful and available; it is not a measure of the probability that the completed study found a real effect. Sample-size planning is also distinct from observed power, which should not be used to reinterpret a result after the fact.
The registry’s stated illustration assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect specified as a fraction of the observed effect. It also says time-to-event illustrations give a total number of events, while other illustrations give participants per arm. These are descriptions of planning illustrations, not evidence that the trial itself used two arms or that these were its original design assumptions. Since the primary endpoint has no evaluable participants and no observed response effect is reported, these illustrations cannot supply missing clinical evidence. The study’s own sample-size justification and statistical plan are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary outcome and several secondary outcomes. The primary outcome is best objective response rate during the first 2 cycles. Secondary outcomes include treatment-related and non-treatment-related adverse events, duration of response, event-free survival, and overall survival. Listing multiple outcomes raises a general statistical issue: when many hypotheses are tested, the chance of at least one apparently unusual result can increase unless the analysis plan addresses multiplicity. The registry record does not report a multiplicity adjustment or a hierarchy for interpreting secondary outcomes.
The record reports no analyses for the outcome measures in the information available for this analysis. For the primary response endpoint, it states that the only participant with a recorded disease-response designation did not complete 1 cycle, despite the requirement to complete at least 1 cycle and have response measurements. The registry indicates there were no evaluable participants. This is missing endpoint information for the defined analysis population, not evidence that the outcome was negative.
The registry does not report a missing-data strategy, an imputation method, or sensitivity analyses for the primary endpoint. It also does not report the detailed analysis populations used for the secondary outcomes. These omissions limit the ability to assess how incomplete observation, treatment discontinuation, or follow-up might affect outcome estimates. The reason for termination and the reason the recorded response participant did not complete 1 cycle are not reported in the registry record.
In general, missingness can affect conclusions when the likelihood of having an outcome recorded is related to the outcome itself or to participant characteristics. That is a methodological concern, not a claim that a particular mechanism occurred in this study. With no evaluable participants for the primary endpoint and no reported analysis addressing missingness, a response estimate cannot be recovered from the registry information.
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 |
|---|---|---|---|
| Assay Positive | 1 | 1 | 100.0% |
| Assay Negative | 0 | 2 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Assay Positive | 0 of 1 |
| Assay Negative | 1 of 2 |
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 |
|---|---|
| Assay Positive | 1 of 1 |
| Assay Negative | 2 of 2 |
Most frequent serious adverse event terms
| Event term | Organ system | Assay Positive | Assay Negative |
|---|---|---|---|
| Catheter related infection | Infections and infestations | 1 of 1 | 0 of 2 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Assay Positive | Assay Negative |
|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | 1 of 1 | 0 of 2 |
| Bone pain (worsening) | Musculoskeletal and connective tissue disorders | 0 of 1 | 1 of 2 |
| Diarrhea | Gastrointestinal disorders | 0 of 1 | 1 of 2 |
| Generalized muscle weakness | Musculoskeletal and connective tissue disorders | 0 of 1 | 1 of 2 |
| Hyperkalemia | Metabolism and nutrition disorders | 1 of 1 | 0 of 2 |
| Mucositis oral | Gastrointestinal disorders | 0 of 1 | 1 of 2 |
| Nausea | Gastrointestinal disorders | 1 of 1 | 0 of 2 |
| Pain in extremity | General disorders | 1 of 1 | 0 of 2 |
| Thromboembolic event | Vascular disorders | 1 of 1 | 0 of 2 |
Serious events with the highest arm-level rate: catheter related infection (100.0%).
The serious adverse-event data are divided into Assay Positive and Assay Negative categories. For Assay Positive, the record lists 1 at risk and 1 affected for serious events; it also lists 1 at risk and 1 affected for other events, with 0 deaths. For Assay Negative, it lists 2 at risk and 0 affected for serious events; for other events it lists 2 at risk and 2 affected, with 1 death. These are registry counts, not a randomized comparison between treatment groups.
Catheter-related infection is the listed top serious term, reported as 100.0%. The registry information available does not clarify the denominator or whether this percentage is based on participants or events, so it should not be treated as a general incidence estimate. The data do not establish whether any event was caused by JNJ-40346527. The relationship of the recorded death to treatment or to a particular event is not reported in the registry record. Small descriptive counts do not support stable comparisons between these assay categories.
12. What the Result Does, and Does Not, Mean
The registry record documents a Phase 2, open-label, single-group study of JNJ-40346527 in recurrent or refractory acute myeloid leukemia. It describes a treatment schedule and a primary response endpoint, but indicates that no participants were evaluable for that endpoint. The participant with a recorded disease-response designation did not complete 1 cycle, while the protocol description required completion of at least 1 cycle and response measurements. Therefore, the registry information does not yield an objective response rate or its confidence interval.
This does not mean the response rate was zero. A zero estimate would require a defined evaluable group and a record that none met the response criteria. Nor does the absence of an estimate demonstrate that the intervention had no effect. The endpoint is not estimable from the registry record, so the record does not answer the primary efficacy question.
The design also limits what could be concluded from a response estimate even if one had been reported. A single-group study has no concurrent comparator, and open-label status means masking was absent. Such data can describe outcomes under the study regimen but cannot, by themselves, establish the difference attributable to treatment relative to another approach.
The safety counts provide descriptive information in assay-positive and assay-negative categories, including a recorded death in the latter category and a catheter-related infection listed as a top serious term. They do not establish cause, treatment attribution, or a reliable contrast between groups. The registry does not report the circumstances of the death, the reason the study was terminated, or the detailed analysis methods needed to interpret other outcomes.
Overall, the record supports a description of the study design, treatment schedule, endpoint definition, and limited adverse-event entries. It does not provide an interpretable primary response result. Any broader conclusion about efficacy, comparative effectiveness, or treatment-related safety would go beyond what the registry record reports.
13. Why This Trial Matters Statistically
This study illustrates why the definition of an analysis population is as important as the outcome definition. The primary endpoint is clearly described as best objective response during the first 2 cycles, with evaluability requiring at least 1 completed cycle and response measurements. But the only participant with a recorded response designation did not meet the completion condition, and the registry indicates there were no evaluable participants. Without an eligible denominator, a rate cannot be calculated or meaningfully compared with another estimate.
It also shows the distinction between a study’s enrollment figure and the population contributing to a particular endpoint. Enrollment is recorded as 3, but the response analysis population depends on the endpoint’s evaluability criteria. Treating enrollment as the response denominator would disregard the registry’s stated rules and would misrepresent what the record reports.
The design context matters as well. A single-group, open-label study can provide descriptive experience, but it does not contain a randomized control comparison. The assay categories in the safety data should not be mistaken for such a comparison. This separation between group labels and trial allocation is important when reading adverse-event counts.
Finally, the record demonstrates that trial status, endpoint availability, and safety entries answer different questions. The study is terminated, but its reason is not reported in the registry record. Safety counts are present, while the primary response estimate is not. Neither the study status nor the safety counts can fill the missing endpoint information. Statistically, the most defensible conclusion is that the response question remains unanswered by the reported data.
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 limitation is that no participants are reported as evaluable for the primary response endpoint. The registry’s criteria require at least 1 completed cycle and response measurements; the only participant with a recorded response designation did not complete 1 cycle. Accordingly, the endpoint rate and its exact confidence interval are not reported in the registry record.
Enrollment is recorded as 3, but the registry information does not show that those participants formed an evaluable population for response. The reason for the incomplete cycle, the reason for study termination, and the extent or timing of outcome follow-up are not reported in the registry record. No missing-data approach or sensitivity analysis is reported.
The study has no control group and no masking. The design is therefore not a randomized comparison, and the assay-positive and assay-negative safety categories should not be interpreted as treatment arms. The small descriptive counts and unclear denominator for the listed catheter-related infection percentage further constrain safety interpretation. The record does not establish event causality or explain the recorded death.
The registry lists secondary outcomes but does not report outcome analyses for them in the information available for this analysis. The statistical plan, multiplicity approach, sample-size justification, and detailed reasons for termination are not reported in the registry record. Together, these limitations leave the primary efficacy question unanswered and restrict conclusions to the study description and recorded safety entries.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: JNJ-40346527 in Treating Participants With Relapsed or Refractory Acute Myeloid Leukemia — NCT03557970.
- Registry record last updated 2021-12-02; status last verified 2021-12.
- Results first posted 2021-12-02.
- 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
NCT03557970 is a terminated Phase 2, open-label, single-group study sponsored by OHSU Knight Cancer Institute. It evaluated JNJ-40346527, also called edicotinib, in participants with recurrent or refractory acute myeloid leukemia. The registry records enrollment of 3 and describes oral dosing twice daily on days 1-28 in repeating 28-day cycles, subject to disease progression or unacceptable toxicity.
The primary endpoint was best objective response during the first 2 cycles. The registry indicates no participants were evaluable: the only participant with a recorded response designation did not complete 1 cycle, while the protocol description required at least 1 cycle and response measurements. No response rate or confidence interval is reported. Secondary outcomes include adverse events, duration of response, event-free survival, and overall survival.
Adverse-event entries are reported for assay-positive and assay-negative categories, with a catheter-related infection listed as a top serious term. The reason for termination, treatment attribution of events, and detailed analyses are not reported in the registry record.