This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Safety, Tolerability and Efficacy of the BL-8040 and Atezolizumab for Maintenance Treatment in Subjects With Acute Myeloid Leukemia (AML) (NCT03154827), a na phase 1, phase 2 study. The primary outcome is Relapse Free Survival.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03154827 |
| Phase | Phase 1, Phase 2 |
| Condition | Acute Myeloid Leukemia |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | BioLineRx, Ltd. |
| Results posted | Yes |
2. Clinical Question
Population
The study concerns subjects with acute myeloid leukemia who are 60 years or older. The primary outcome population is described as all eligible patients who received at least 1 dose of study medications. The registry record does not report further eligibility details.
Intervention
The study evaluated a combination of BL-8040 and atezolizumab for maintenance treatment. The registry describes a single combination-treatment arm; it does not report separate treatment groups.
Comparator
There was no parallel comparator arm. Allocation is recorded as not applicable, and the study is listed as single-group and open-label. The record therefore does not define a randomized or like-for-like control comparison.
Primary question
The primary question was how long participants remained free from relapse or death after complete remission while receiving the combination as maintenance treatment. Relapse-free survival was measured from complete remission to relapse or death from any cause, whichever came first. The registry lists a timeframe of up to 5 years, but reports no outcome analysis.
In statistical terms, this is a question about the observed course of participants receiving the combination, not a direct estimate of how their outcomes compare with another treatment or with no treatment. Without a comparator, differences attributable to treatment cannot be separated from other explanations using this design alone. The registry record identifies safety and tolerability as the main goal and lists relapse-free survival, minimal residual disease, overall survival, and event-free survival among additional objectives. It does not report analyses for those objectives.
3. Trial Design
The BATTLE study is described as a Phase Ib/II, multicenter, single-arm, open-label study of BL-8040 combined with atezolizumab for maintenance treatment in subjects with acute myeloid leukemia who are 60 years or older. The registry lists phases PHASE1 and PHASE2. Its start date is 2018-01-08, its primary completion date is 2020-04-01, its status is TERMINATED, and enrollment is 1.
A single-arm design means the registry describes one group receiving the combination, rather than distinct groups assigned to different treatments. Open-label means masking is listed as none. Allocation is recorded as not applicable. As a result, the design does not provide a contemporaneous control group for a treatment comparison. The registry does not report the reason for termination, the planned enrollment, or the details of how enrollment reached 1.
The primary outcome is relapse-free survival, with a stated timeframe of up to 5 years. The outcome description defines the event as relapse or death from any cause, whichever comes first, measured from complete remission. The specified outcome population is all eligible patients who received at least 1 dose of study medications. The record lists one outcome measure and zero analyses. It does not report a numerical relapse-free survival estimate, event count, follow-up distribution, or statistical test.
These design features set clear limits on interpretation. The record can describe the planned study question, the recorded status, and the posted safety counts. It cannot support a randomized comparative estimate, and the absence of a posted outcome analysis means the primary endpoint cannot be summarized from 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 | Combination Treatment Single Arm |
|---|---|
| STARTED | 1 |
| COMPLETED | 0 |
| NOT COMPLETED | 1 |
Analysis population for the primary outcome, as defined in the registry: All eligible patients who received at least 1 dose of study medications
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.
Population: All patients who were eligible to the study
| Characteristic | Unit | Combination Treatment of BL-8040 With Atezolizumab |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 |
| Age, Categorical: >=65 years | Participants | 1 |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| 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 | 0 |
| Race (NIH/OMB): White | Participants | 1 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: Czechia | participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Relapse Free Survival, measured over Up to 5 years.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
Relapse-free survival (RFS) is the primary endpoint. The registry defines it as the time from complete remission until relapse or death from any cause, whichever occurs first. The stated timeframe is up to 5 years, and the specified population is all eligible patients who received at least 1 dose of study medications.
In general, a time-to-event endpoint represents both whether an event occurs and how long participants remain event-free during observed follow-up. Using relapse or death as the event captures two kinds of outcomes in a single endpoint; whichever occurs first determines the recorded event time. This can be useful when the clinical question concerns the duration of remission without either event, rather than only the proportion experiencing relapse at a fixed point.
Interpreting such an endpoint usually depends on information about event times, censoring, follow-up, and the analysis method. Censoring occurs when a participant's event status is not observed through the end of the analysis period; how it is handled can affect estimates. The registry record does not report an RFS estimate, event count, confidence interval, censoring details, or analysis method. Therefore, the endpoint definition is available, but its observed distribution and precision are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The principal statistical fact is that the registry describes a study with enrollment of 1, status TERMINATED, one listed outcome measure, and zero analyses. Although relapse-free survival is identified as the primary outcome, no numerical RFS result or statistical analysis is reported in the registry record. There is consequently no posted estimate to interpret, no uncertainty interval to assess, and no reported test of a hypothesis.
The study design also matters. The combination was evaluated in a single arm without a parallel control group. An outcome observed in that arm would describe the participants' experience during the study, but by itself would not establish what would have happened to comparable participants under another treatment or without the combination. That distinction is especially important for survival outcomes, which can reflect the underlying disease course and other influences as well as treatment exposure.
Enrollment of 1 means the registry record describes a very limited enrolled group. The safety fields list 1 participant at risk and 1 participant affected for serious adverse events, and also list 1 at risk and 1 affected for other adverse events. The record lists 0 deaths in the safety group. These counts should be read as registry entries, not as a stable estimate of how often events would occur in a broader population. A single participant's experience cannot establish a reliable frequency pattern or characterize variability across participants.
The serious adverse event term listed is bronchiolitis at 100.0%. That figure is attached to the posted serious-event term, but the registry does not explain the denominator or the precise aggregation basis for the percentage. It should not be interpreted as a rate applicable beyond this recorded study group, nor does the registry entry establish that the study medication caused the event.
Overall, the record supports a description of the intended question, the single-arm design, the enrollment and status, and the posted safety counts. It does not provide evidence from an analyzed RFS endpoint or a controlled comparison. The sponsor's rationale for termination, the extent of follow-up, and reasons for the limited enrollment are not reported in the registry record. Accordingly, no conclusion about treatment benefit, lack of benefit, or comparative safety follows from these entries.
9. Statistical Methods Explained
What does a single-arm study estimate?
In general, a single-arm study records outcomes among participants who receive the study treatment. It can describe what happened to that group, subject to how outcomes were collected and analyzed. It does not directly estimate a treatment contrast against a concurrent control group, because no such group is included in the design described by the registry. External comparisons may be affected by differences in eligibility, disease status, follow-up, and other factors; the registry record does not describe an external comparator or analysis.
For the BATTLE study, the registry lists a single combination-treatment arm, with allocation not applicable and masking listed as none. It reports enrollment of 1 and zero analyses. Consequently, a between-group treatment effect is not available, and the primary RFS analysis is not reported. The record does not identify a statistical model, an estimation procedure, or a hypothesis test used for the outcome.
How should relapse-free survival be understood?
In general, time-to-event analysis uses information about when an event occurs, rather than treating every participant only as an event or a non-event at a single endpoint. Participants whose event has not been observed by the end of follow-up may be censored: their observed time contributes information up to the point at which follow-up ends, without asserting that an event never occurs. Estimates therefore depend on event definitions, follow-up, censoring, and the analytic method.
The registry defines RFS from complete remission to relapse or death from any cause, whichever comes first, with a timeframe of up to 5 years. It does not report an event count, participant-level follow-up, censoring rules, or an RFS estimate. It also does not report whether an estimator such as a Kaplan-Meier method was used. Such methods may be common in general time-to-event analysis, but attributing a particular method to this study would go beyond the registry record.
A confidence interval, in general, expresses uncertainty around an estimate under the assumptions of the method used. A narrow interval indicates more precision than a wide interval, but neither guarantees that the design answers a causal question. No confidence interval for RFS is reported, so the precision of an outcome estimate cannot be assessed.
What does the planning illustration mean?
General sample-size planning asks how many participants or events might be needed to detect a specified effect under stated assumptions. The registry-associated planning description says its illustration is computed from the observed effect, uses a two-sided significance level of 0.05, assumes equal allocation between two arms, and uses a normal approximation. It says time-to-event illustrations concern total events, while other illustrations concern participants per arm. Those assumptions describe a planning illustration, not the design actually recorded for this study, which is single-arm.
Planning values are not observed power and do not show that a trial had a particular chance of success after it was conducted. They depend on the assumed effect and other design choices. The registry record does not report a trial-specific sample-size justification or a numerical planning estimate in the outcome information. It also does not report a comparison effect estimate for RFS. The planning assumptions therefore cannot be treated as evidence about the study's observed efficacy or its original design rationale.
Why does over-dispersion matter in general?
Over-dispersion refers to greater variability in data than a statistical model assumes. When it is present, methods that assume less variability can produce uncertainty estimates that are too small. It is relevant to some count-data analyses, but the registry record does not report a count-outcome model or an over-dispersion assessment for this study. It would be inappropriate to imply that either was used.
More broadly, statistical methods are only interpretable when their assumptions and inputs are clear. For this record, the endpoint definition is available, but the analysis population beyond its written description, the censoring approach, the model, and the uncertainty estimates are not reported in the registry record. The main method-related conclusion is therefore about the limits of what can be evaluated: the intended time-to-event question is identifiable, but its statistical answer is not posted.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists relapse-free survival as the primary outcome and describes minimal residual disease, overall survival, and event-free survival as additional objectives in the study summary. It reports one outcome measure and zero analyses. No outcome results for these objectives are reported in the registry record.
Multiplicity arises in general when several outcomes, time points, or statistical tests are examined. Testing many questions can increase the chance that at least one result appears notable by chance. A prespecified strategy may define which outcome is primary and whether or how statistical evidence is adjusted across analyses. The registry record does not report a multiplicity strategy, a hierarchy among additional objectives, or any adjustment method. Since analyses are not posted, it is not possible to assess how multiple questions were handled in practice.
The record defines the primary outcome population as all eligible patients who received at least 1 dose of study medications. It does not report the full analysis populations, the eligibility criteria in sufficient detail to reconstruct membership, or whether every enrolled participant was included in each analysis. The enrollment figure is 1, but no outcome analysis is reported.
Missing-data methods are also not reported in the registry record. In general, incomplete follow-up and missing outcome information can affect time-to-event estimates, particularly when the reasons for missingness relate to prognosis or study participation. The record does not describe censoring rules, imputation, sensitivity analyses, or the handling of withdrawals and unavailable measurements. No assumptions about these procedures should be inferred.
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 |
|---|---|---|---|
| Combination Treatment of BL-8040 With Atezolizumab | 1 | 1 | 100.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Combination Treatment of BL-8040 With Atezolizumab | 0 of 1 |
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 |
|---|---|
| Combination Treatment of BL-8040 With Atezolizumab | 1 of 1 |
Most frequent serious adverse event terms
| Event term | Organ system | Combination Treatment of BL-8040 With Atezolizumab |
|---|---|---|
| Bronchiolitis | Infections and infestations | 1 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Combination Treatment of BL-8040 With Atezolizumab |
|---|---|---|
| Rash | Skin and subcutaneous tissue disorders | 1 of 1 |
| Vomiting | Gastrointestinal disorders | 1 of 1 |
| Nausea | Gastrointestinal disorders | 1 of 1 |
| Cough | Respiratory, thoracic and mediastinal disorders | 1 of 1 |
| Rhinitis | Respiratory, thoracic and mediastinal disorders | 1 of 1 |
| Decreased appetite | Metabolism and nutrition disorders | 1 of 1 |
| Dermatitis allergic | Skin and subcutaneous tissue disorders | 1 of 1 |
| Anxiety | Psychiatric disorders | 1 of 1 |
| Constipation | Gastrointestinal disorders | 1 of 1 |
| Bronchitis | Infections and infestations | 1 of 1 |
Serious events with the highest arm-level rate: bronchiolitis (100.0%).
The safety group is labeled as receiving the combination of BL-8040 with atezolizumab. The registry lists 1 participant at risk and 1 affected for serious adverse events, and 1 participant at risk and 1 affected for other adverse events. It lists 0 deaths. These are counts in the registry record; they are not estimates of event frequency in a larger population.
Bronchiolitis is the serious adverse event term listed at 100.0%. The registry record does not explain the denominator or aggregation basis for this percentage. With only 1 participant listed at risk in the safety group, the entry should be understood as a description of the recorded safety data, not as a precise population rate. The record does not state whether the event was judged related to treatment, and the listing alone cannot establish causation.
In general, adverse-event interpretation depends on clear definitions, observation periods, exposure, event severity, attribution, and complete reporting. The registry record does not provide those details beyond the listed counts and term. It also does not report a comparative safety group, so these entries cannot establish how the combination's safety profile compares with another treatment or with no treatment.
12. What the Result Does, and Does Not, Mean
The record establishes that the BATTLE study evaluated BL-8040 with atezolizumab as maintenance treatment in a single-arm, open-label study for acute myeloid leukemia. It identifies relapse-free survival as the primary outcome and defines the event as relapse or death from any cause, whichever comes first, from complete remission. It also records enrollment of 1 and a TERMINATED status.
What the record does not establish is an analyzed result for the primary outcome. No RFS estimate, confidence interval, event count, or statistical test is reported. There is therefore no posted estimate of how long participants remained free from relapse or death, and no way to judge the precision of such an estimate from these registry entries.
The single-arm design cannot isolate a treatment effect by comparison with a control arm. Even if outcome data had been posted for the treated group, an observed course alone would not identify what would have occurred under an alternative treatment. The registry record also does not report the reason for termination, the planned enrollment, or a trial-specific sample-size justification. These missing details limit interpretation of the study's conduct and its ability to answer the intended question.
The safety counts document the entries recorded for the study group, including the bronchiolitis serious-event term. They do not show that treatment caused an event, and the small recorded group cannot establish a general event rate. A lack of a reported outcome analysis is not evidence of no events or of no treatment effect; it means the outcome analysis is not reported in the registry record.
In general, trial results require both an interpretable estimate and an assessment of its uncertainty, while causal treatment conclusions require a design and analysis capable of supporting comparison. Neither an analyzed primary endpoint nor a controlled treatment comparison is available in this record. The appropriate reading is descriptive and limited to the registry entries, without inferring benefit, lack of benefit, or comparative safety.
13. Why This Trial Matters Statistically
This record is statistically important because it makes the distinction between a stated research question and a reported answer especially clear. Relapse-free survival is explicitly identified as the primary outcome, with a defined event and a stated timeframe of up to 5 years. Yet the registry reports zero analyses. The endpoint definition alone is not an outcome result: it does not reveal event timing, the proportion remaining event-free, or uncertainty.
The design also illustrates why a single-arm study has a different inferential reach from a controlled comparison. It can be framed around outcomes among treated participants, but it lacks a concurrent group that could support a direct contrast. This matters when readers encounter safety counts or any potential outcome estimate: an observation in the treatment group is not itself a comparative treatment effect.
Enrollment of 1 makes the record useful as a case study in cautious reading of small-group safety entries. Counts and a listed percentage can appear precise while conveying little about the range of outcomes in a broader population. Precision depends on the amount and structure of information, not merely on the display of a percentage.
The terminated status and absent analysis also make transparency about missing information central. The record does not report why the study ended, the analysis methods, or the outcome findings. Statistical interpretation should therefore separate what is documented—the design, endpoint definition, status, enrollment, and safety entries—from what cannot be evaluated, including the primary endpoint result and comparative effects.
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.
Several trial-specific limitations restrict interpretation. The registry identifies the study as single-arm, open-label, with allocation not applicable and masking listed as none. There is no parallel control group, so the design does not support a direct randomized comparison. The record reports enrollment of 1, a limited basis for characterizing outcome patterns or safety frequencies.
The study status is TERMINATED, but the reason for termination is not reported in the registry record. Planned enrollment, recruitment history, and whether the recorded enrollment reflects the full intended study population are also not reported. Without those details, the context for the limited enrollment cannot be assessed.
Although RFS is the primary outcome, no analysis is posted. The record does not report an RFS estimate, event count, confidence interval, or test result. It does not report the censoring approach, follow-up completeness, or the statistical method used. The stated timeframe of up to 5 years does not establish how much follow-up was actually available.
The safety record lists 1 participant at risk and 1 affected for serious adverse events, as well as 1 at risk and 1 affected for other adverse events. The serious-event term is bronchiolitis at 100.0%, but its denominator and aggregation basis are not explained. The record does not report an attribution assessment or comparative safety data.
Finally, the registry does not report detailed eligibility criteria, the reason for termination, multiplicity procedures, missing-data methods, or the complete analysis populations. These gaps should not be filled by assumptions about the protocol or study conduct. They limit both reproducibility and the conclusions that can be drawn from the posted record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Safety, Tolerability and Efficacy of the BL-8040 and Atezolizumab for Maintenance Treatment in Subjects With Acute Myeloid Leukemia (AML) — NCT03154827.
- Registry record last updated 2024-09-05; status last verified 2024-08.
- Results first posted 2024-09-05.
- 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
The registry describes the BATTLE study, a Phase Ib/II, multicenter, single-arm, open-label evaluation of BL-8040 with atezolizumab as maintenance treatment in subjects with acute myeloid leukemia who are 60 years or older. The listed primary outcome is relapse-free survival from complete remission to relapse or death from any cause, whichever comes first, with a timeframe of up to 5 years.
The record lists a start date of 2018-01-08, a primary completion date of 2020-04-01, status TERMINATED, and enrollment of 1. It reports one outcome measure and zero analyses. No RFS result is reported in the registry record.
The safety group lists 1 participant at risk and 1 affected for serious adverse events, 1 at risk and 1 affected for other adverse events, and 0 deaths. Bronchiolitis is listed as a serious adverse event term at 100.0%. The reason for termination, outcome analysis methods, and comparative results are not reported.