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 Study of Bavituximab, Axitinib, and Avelumab in Advanced Hepatocellular Carcinoma (NCT05249569), a na phase 2 study. The primary outcome is Response Rate of Combination Therapy.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT05249569 |
| Phase | Phase 2 |
| Condition | Hepatocellular Carcinoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Texas Southwestern Medical Center |
| Results posted | Yes |
2. Clinical Question
Population
People with advanced hepatocellular carcinoma (HCC) not previously treated with systemic therapy were the intended population for the primary objective response question. The registry record states that one participant signed consent and enrolled. That participant did not receive study treatment before the study was terminated, so the enrolled participant did not contribute treatment-response data.
Intervention
The study evaluated a combination of axitinib, avelumab, and bavituximab. The registered regimen was axitinib 5 mg PO BID, avelumab 10 mg/kg IV every 2 weeks (2 doses in a 4-week cycle), and bavituximab 3 mg/kg IV every 1 week (4 doses in a 4-week cycle). Treatment was to continue until disease progression, unacceptable toxicity, death, or another reason for stopping study treatment, up to 24 months.
Comparator
The design was single-group, with allocation recorded as NA and masking recorded as NONE. The registry record does not describe a separate comparator group. The combination was therefore not set up as a randomized or like-for-like comparison against another treatment in this study.
Primary question
The primary objective was to determine the objective response rate for the combination in advanced HCC not previously treated with systemic therapy. Response rate asks what proportion of treated participants meet the study’s criteria for an objective tumor response. The response criteria are not reported in the registry record. Because the enrolled participant did not receive treatment and data were not collected for this outcome, the record provides no observed response-rate result.
3. Trial Design
This was a multicenter, open-label Phase II study of axitinib, avelumab, and bavituximab in advanced hepatocellular carcinoma. The registered design is single-group, allocation is recorded as NA, and masking as NONE. No separate control arm or randomized comparison is described. The sponsor was the University of Texas Southwestern Medical Center.
The planned regimen combined axitinib 5 mg PO BID, avelumab 10 mg/kg IV every 2 weeks (2 doses in a 4-week cycle), and bavituximab 3 mg/kg IV every 1 week (4 doses in a 4-week cycle). Study treatment was to continue until disease progression, unacceptable toxicity, death, or discontinuation for another reason, up to 24 months. These details describe the registered plan, not treatment received: the registry record states that the participant enrolled did not receive treatment before termination.
The primary objective was objective response rate in advanced HCC not previously treated with systemic therapy. Listed secondary outcomes were disease control rate, overall survival, 6-month progression-free survival, duration of response, and safety outcomes covering adverse events and laboratory abnormalities graded by NCI CTCAE v5.0. The registry record reports no collected primary-outcome data and no analyses. It does not report results for the secondary outcomes.
The study started on 2022-11-04, had a primary completion date of 2022-12-30, and is recorded as TERMINATED. One participant signed consent and enrolled. The record does not report why the study was terminated or provide further details about the circumstances. The intended design and planned regimen should therefore be distinguished from an evaluation of treatment: there is no treated study population in the registry results.
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 | Axitinib / Avelumab /Bavituximab |
|---|---|
| STARTED | 1 |
| COMPLETED | 0 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Axitinib / Avelumab /Bavituximab |
|---|---|
| Sponsor pulled support for one of the study drugs. | 1 |
Analysis population for the primary outcome, as defined in the registry: One patient signed consent and was enrolled on study. The participant that was enrolled on the study did not receive treatment before the study was terminated. Data was not collected for this outcome measure.
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: One patient signed consent and was enrolled on study. However, the sponsor that was planning to provide one of the study drugs decided they no longer wanted to supply the drug for this project. The participant that was enrolled on the study did not receive treatment before the study was terminated.
| Characteristic | Unit | Axitinib / Avelumab /Bavituximab |
|---|---|---|
| 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 | 0 |
| Sex: Female, Male: Male | Participants | 1 |
| 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 |
6. Primary Endpoint
The registry lists the primary outcome as Response Rate of Combination Therapy, measured over Study treatment will continue until disease progression, unacceptable toxicity, death, or discontinuation from the study treatment for any other reason up to 24 months..
| 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 was the response rate of the combination therapy. The stated objective was to determine the objective response rate of axitinib, avelumab, and bavituximab in advanced HCC not previously treated with systemic therapy. An objective response endpoint is intended to describe tumor response using prespecified assessment criteria. In general, objective response rate commonly refers to the proportion of participants whose tumors meet criteria for a complete or partial response. The registry record does not report the specific response criteria or assessment procedures, so their details cannot be characterized.
Response rate is used in clinical trials to summarize observed tumor shrinkage under the endpoint’s definition. It is distinct from outcomes such as disease control, duration of response, progression-free survival, and overall survival, which address different aspects of disease status or time. Those outcomes were listed as secondary measures in this study, but their results are not reported in the registry record.
No response-rate estimate can be obtained from this record. The enrolled participant did not receive treatment before the study was terminated, and the record states that data were not collected for the primary outcome. Thus, the endpoint identifies the question the trial was intended to address, not a measured result about response to the combination.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record supports a narrow interpretation: the study was designed to evaluate the objective response rate of a three-drug combination, but it did not generate a reported primary-outcome estimate. One participant signed consent and enrolled; that participant did not receive study treatment before termination. The record explicitly states that data were not collected for the response-rate outcome. It therefore contains no observed response result to interpret.
This is not evidence that the combination did or did not produce tumor responses. A response rate requires treatment and outcome assessments made under the endpoint definition. Neither treatment exposure nor primary-outcome observations are recorded for the enrolled participant. It would be inaccurate to treat the absence of collected data as a response, a nonresponse, or an estimate of treatment effect.
The same distinction applies to the listed secondary outcomes. Disease control rate, overall survival, 6-month progression-free survival, duration of response, and safety outcomes were planned or listed, but results for them are not reported in the registry record. The adverse-event summary records zero participants at risk and zero affected for its displayed safety categories. Because the enrolled participant did not receive study treatment, those entries do not establish a safety profile during exposure to the regimen.
The design was single-group and open-label, without a registered comparator. Even if outcome data had been available, a single-group result would describe outcomes among participants receiving the regimen rather than provide a randomized comparison against another treatment. Here, there are no primary outcome data to describe even within a treated group. No analyses are recorded.
The registry record does not report the statistical analysis plan, response criteria, handling of missing outcome data, analysis populations, or multiplicity procedures. These omissions limit what can be said about how the planned endpoint would have been estimated and interpreted. They do not change the central finding in the record: the primary endpoint was not measured for a treated participant, and no response-rate result is reported.
The termination and lack of treatment exposure are essential context when reading the study’s planned design. The planned regimen and outcomes describe what the trial set out to study; they should not be mistaken for evidence that the regimen was administered or evaluated. In statistical terms, the record offers no estimate whose precision or uncertainty can be assessed for the primary question.
9. Statistical Methods Explained
What does an objective response rate represent?
In general, an objective response rate is a proportion based on participants who meet the trial’s criteria for tumor response. The response criteria determine what counts as a response, how it is assessed, and how observations are categorized. The registry record names objective response rate as the primary endpoint and its description mentions the intended population, but does not report the response criteria or assessment schedule. Those methodological details are therefore not reported in the registry record.
A response proportion is interpretable only when the relevant population and outcome observations are defined. The record states that one participant enrolled, did not receive study treatment, and had no primary-outcome data collected. There is consequently no observed numerator or evaluable treated population from which to estimate response. No response-rate value or uncertainty interval is reported.
Why does the single-group design matter?
General: a single-group study follows participants receiving a regimen without a separate concurrent control group. It can describe outcomes observed in that group if treatment and outcome measurements are available. It does not, by its design alone, establish how those outcomes compare with what would have occurred under another treatment or without the study regimen. The registry record classifies this study as single-group and does not describe a comparator.
Open-label means masking was not used, as also recorded in the registry. General: lack of masking can matter when outcomes involve judgment, behavior, or decisions influenced by knowledge of treatment. The record does not provide enough detail about response assessment to determine how masking or its absence would affect assessment in this study. In any event, no response assessment data were collected.
What can be learned from the safety entries?
The listed safety outcomes concern participants with serious or non-serious adverse events and participants with laboratory abnormalities, graded by NCI CTCAE v5.0. The registry’s adverse-event summary reports zero at risk and zero affected in the displayed categories. General: safety summaries rely on exposure and observation over a defined period. A zero count among a group with no participants at risk is not evidence that treatment caused no adverse events; it indicates that the summary contains no at-risk participants for those categories. The enrolled participant did not receive study treatment, so the registry does not report safety observations during treatment exposure.
What do the listed secondary outcomes add?
Disease control rate, overall survival, 6-month progression-free survival, duration of response, and safety were listed as secondary outcomes. General: each outcome answers a different question. Disease control combines response-related categories according to a study definition; time-to-event outcomes concern the timing of events or follow-up status; duration of response concerns how long a response persists among participants who respond. The record does not provide operational definitions or outcome data for these measures. It is not possible to infer their results from the list of planned outcomes.
What is the role of statistical analysis?
General: an analysis plan specifies how outcomes will be summarized or modeled, which participants will be included, how missing observations will be handled, and what uncertainty measures will accompany estimates. For a proportion, a confidence interval can describe the range of values compatible with the data under the chosen method and its assumptions. An interval based on no collected response observations cannot provide useful precision about the treatment’s response rate. The registry record does not report a statistical analysis plan or any analyses.
The registry includes an assumption statement for planning illustrations, but it does not describe those illustrations as the trial’s own design analysis. Such calculations are not observed outcomes and cannot substitute for response data. No sample-size justification, interim-analysis approach, or detailed statistical plan is reported in the registry record. No conclusion about statistical power or the adequacy of a planned sample can be drawn from the recorded results.
Multiplicity is relevant when several outcomes or comparisons are tested, because repeated testing can affect the chance of false-positive findings. General: methods to address multiplicity depend on the intended inferential questions and analysis plan. The record lists a primary outcome and secondary outcomes but does not report testing procedures or adjustment methods. Since no analyses are recorded, there are no reported statistical tests to interpret.
Missingness also requires context. General: an unobserved outcome is not automatically equivalent to a favorable or unfavorable outcome. The registry states that primary-outcome data were not collected because the enrolled participant did not receive treatment before termination. This is a structural absence of treatment-response data in the record, not a reported response classification. The record does not state a planned missing-data method or define analysis populations.
10. Multiplicity, Missing Data and Analysis Populations
The registry record identifies objective response rate as the primary outcome and lists secondary outcomes covering disease control, overall survival, 6-month progression-free survival, duration of response, and safety. It does not report whether statistical tests were planned for these outcomes, whether any multiplicity adjustment was specified, or how outcomes would be prioritized for interpretation. Multiplicity procedures are therefore not reported in the registry record.
Missing data are central to interpretation of the primary endpoint. The record says that one participant signed consent and enrolled, did not receive treatment before the study was terminated, and had no data collected for the response-rate outcome. No response status is reported. The record does not describe a missing-data strategy, imputation procedure, or sensitivity analysis. General: absent outcome data should not be treated as a response or nonresponse unless the study’s analysis rules explicitly define that classification.
The registry record does not report analysis populations, such as a defined treated population or a population intended for a particular endpoint analysis. It also does not explain how participants who did not start treatment would have been handled in an analysis. With no treatment exposure and no primary-outcome data reported, there is no analysis population from which a response rate can be interpreted. No analyses are recorded for the primary or secondary outcomes.
These omissions do not imply that a particular procedure was or was not used outside the registry record. They mean that the record does not document these aspects of the statistical plan, and no outcome analysis is available to evaluate.
11. What the Result Does, and Does Not, Mean
The record establishes what the trial intended to study: a combination of axitinib, avelumab, and bavituximab in advanced HCC, with objective response rate as the primary outcome. It also records that the study was open-label and single-group, and that it was terminated. One participant enrolled, but did not receive treatment before termination.
The record does not establish a response rate, disease-control result, survival result, duration-of-response result, or treatment safety estimate. Primary-outcome data were not collected, and analyses are not recorded. The safety entries with zero at risk and zero affected do not demonstrate an absence of treatment-related events because the record reports no treatment exposure.
General: a planned endpoint describes a question, whereas an endpoint result requires observations collected under an analysis definition. Likewise, a treatment regimen in a registry describes the intended intervention; it does not show that participants received it. Here, the registered dose and schedule should be understood as planned regimen details, not as evidence of administration.
The study cannot support a conclusion about whether the combination changes outcomes in advanced HCC. It also cannot support a comparison with another treatment because no comparator group is described. The absence of a result is not a negative treatment result. It reflects the lack of treatment and outcome data reported for this terminated study.
For readers, the key distinction is between the clinical question and the evidence actually recorded. The question concerned response to the combination in a specified population. The available record documents enrollment without treatment and no primary endpoint data. Any stronger claim about efficacy, lack of efficacy, or safety would go beyond the posted results.
12. Why This Trial Matters Statistically
This trial is statistically informative as an example of the difference between a planned study and an analyzable clinical result. The registry describes a Phase II, open-label, single-group evaluation, its intended combination regimen, and its primary response-rate question. But the enrolled participant did not receive treatment, and the primary outcome was not collected. The design alone cannot supply an estimate when no endpoint observations are reported.
That distinction matters because a study’s title, phase, and planned outcomes can create an expectation of treatment evidence. Statistical interpretation must instead begin with what was observed. In this record, there is no response-rate estimate to compare with a benchmark, no uncertainty interval to assess, and no treated group outcome to characterize. The absence of such an estimate is not evidence for either benefit or lack of benefit.
The record also illustrates why safety counts need denominators and exposure context. General: a count of events is interpreted in relation to participants at risk and the observation conditions. Here the adverse-event summary records zero at risk, while the participant enrolled did not receive treatment. The counts therefore cannot be read as a treatment safety profile.
Finally, the record leaves important statistical methods undocumented, including the analysis plan, analysis populations, missing-data handling, and multiplicity procedures. With no analyses reported, those omissions cannot be resolved by examining results. The most defensible statistical account is consequently limited but useful: the trial posed a clinical question, but the registry record contains no treatment-response data with which to answer it.
13. 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 the absence of treatment exposure and primary-outcome data. The registry states that the participant who enrolled did not receive study treatment before termination and that no data were collected for the objective response rate outcome. There is therefore no response estimate or treatment-effect precision to assess.
The study was single-group and open-label, with no registered comparator. Even if outcomes had been reported, the design would not provide a randomized comparison against another treatment. The registry does not report response criteria, assessment procedures, or the statistical analysis plan, so the intended operational and inferential details cannot be evaluated.
Although several secondary outcomes were listed, their results are not reported in the registry record. The safety summary has zero participants at risk and zero affected in its displayed categories; given the absence of treatment exposure, it cannot characterize safety during treatment. The record also does not report analysis populations, methods for handling missing outcomes, multiplicity procedures, or a reason for termination.
These limitations are specific to the recorded study information and should not be filled by assumptions about protocol conduct beyond what the registry states. The planned regimen and endpoint identify the study’s intent, but the record does not contain observations that answer the primary clinical question.
14. Related Tutorials
15. Related Statistical Calculators
16. Sources
- ClinicalTrials.gov: Study of Bavituximab, Axitinib, and Avelumab in Advanced Hepatocellular Carcinoma — NCT05249569.
- Registry record last updated 2023-05-03; status last verified 2023-04.
- Results first posted 2023-05-03.
- 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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17. Record Summary
The registry record describes a multicenter, open-label Phase II study of axitinib, avelumab, and bavituximab in advanced hepatocellular carcinoma. Its primary objective was to determine objective response rate among people with advanced HCC not previously treated with systemic therapy. The design was single-group, with no comparator described.
The planned regimen was axitinib 5 mg PO BID, avelumab 10 mg/kg IV every 2 weeks (2 doses in a 4-week cycle), and bavituximab 3 mg/kg IV every 1 week (4 doses in a 4-week cycle). The record states that one participant enrolled but did not receive treatment before the study was terminated. Primary-outcome data were not collected, and no analyses are reported.
Secondary outcomes were listed, but results are not reported in the registry record. The available information therefore describes the study’s intent and termination, not an observed response or safety result.