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 AZD9150, a STAT3 Antisense Oligonucleotide, in People With Malignant Ascites (NCT02417753), a na phase 2 study. The primary outcome is Changes in Immune Parameters in the Malignant Ascites of Patients With Advanced Cancer Following Therapy With AZD9150.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02417753 |
| Phase | Phase 2 |
| Condition | Ovarian Cancer, Ovarian Neoplasms, Ascites |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Cancer Institute (NCI) |
| Results posted | Yes |
2. Clinical Question
Population
The planned population was adults with a gastrointestinal malignancy or metastatic ovarian cancer and ascites. The registry describes the study as involving people with malignant ascites and advanced cancer. The record reports enrollment of 1 participant, so the posted experience represents a very limited basis for describing outcomes in the broader eligible population.
Intervention
The intervention was AZD9150, a STAT3 antisense oligonucleotide. The registry describes intravenous administration over a 28-day cycle. The brief summary planned six administrations in the first cycle and four in each later cycle. These are planned treatment details; the registry record does not establish how much treatment the participant received.
Comparator
There was no control group. The study is recorded as single-group, with allocation listed as not applicable and masking listed as none. As a result, the design does not provide a like-for-like comparison with another treatment or with an untreated group. Any changes observed during treatment could not, from this design alone, be separated from other influences.
Primary question
The primary question concerned changes in immune parameters in malignant ascites following AZD9150 therapy. The planned measurements included percentages of memory CD8+ cells, regulatory T cells, plasmacytoid dendritic cells, B cells, and natural killer cells in ascitic fluid. The registry says this outcome was not done because the one patient did not make it to one scan after 8 weeks.
In statistical terms, this was an exploratory question about biological measurements before and after treatment, not a randomized treatment-effect comparison. The registry record does not report a primary endpoint estimate or a statistical test for those immune parameters.
3. Trial Design
This was a phase 2 pilot study sponsored by the National Cancer Institute. The registry classifies the design as single-group, with no masking and allocation recorded as not applicable. The study began on 2015-04-03, had a primary completion date of 2016-04-07, and is listed as terminated. The record reports enrollment of 1 participant.
The planned intervention was AZD9150, administered intravenously over a 28-day cycle. The brief summary describes six administrations in the first cycle and four in subsequent cycles. Planned study procedures included screening, physical examinations, blood tests, paracentesis to collect ascitic fluid, and tumor imaging after every 2 cycles. Some participants might also have had a tumor biopsy. These descriptions concern the intended procedures; the record does not specify which were completed for the enrolled participant.
The study aimed to examine immune parameters in ascitic fluid after AZD9150 therapy. Secondary outcomes concerned STAT3-associated signaling, p-STAT3 expression, response rate, adverse events, progression-free survival, and overall survival. The registry record describes seven outcome measures, but it lists no analyses. The primary immune-parameter outcome is explicitly recorded as not done because the participant did not make it to one scan after 8 weeks.
Because there was no comparison group, the design could describe measurements and events observed during the study but could not directly estimate a comparative treatment effect. The registry record does not report a statistical analysis plan, a sample-size justification, or interim analyses. It also does not establish the amount or duration of treatment actually received.
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 | AZD9150 in People With Malignant Ascites |
|---|---|
| STARTED | 1 |
| COMPLETED | 0 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | AZD9150 in People With Malignant Ascites |
|---|---|
| Death | 1 |
Analysis population for the primary outcome, as defined in the registry: This outcome measure was not done because the one patient did not make it to one scan after 8 weeks.
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 | AZD9150 in People With Malignant Ascites |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | years | 60.4 (SD 0) |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 1 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 1 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | 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 | 1 |
| Race (NIH/OMB): White | Participants | 0 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | Participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Changes in Immune Parameters in the Malignant Ascites of Patients With Advanced Cancer Following Therapy With AZD9150, measured over 1.5 years.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint concerned changes in immune parameters in malignant ascites following AZD9150 therapy. The planned procedure was to collect ascitic fluid by research paracentesis and process it for flow cytometry. The measures were the percentages of memory CD8+ cells, regulatory T cells, plasmacytoid dendritic cells, B cells, and natural killer cells. The stated timeframe was 1.5 years.
These measurements were intended to characterize immune-cell composition in the ascites. In general, a before-and-after biomarker endpoint can help describe whether measured biological features change during treatment. Such a change is not by itself proof that treatment caused it, particularly without a comparison group. It also does not directly establish a change in symptoms, tumor control, or survival.
The registry states that this outcome was not done because the one patient did not make it to one scan after 8 weeks. It reports no baseline or follow-up values, no change estimate, and no uncertainty interval for the primary endpoint. Therefore, the planned biological question remains unanswered by the posted primary-outcome results. The record does not report whether any of the planned fluid samples or other endpoint-related procedures were completed.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The most important interpretive fact is that the primary immune-parameter outcome was not done. The registry attributes this to the participant not making it to one scan after 8 weeks. Consequently, there are no posted primary endpoint measurements to interpret as evidence of a change in the ascitic-fluid immune-cell percentages. No effect estimate, confidence interval, or primary endpoint test is reported.
The record reports enrollment of 1 participant and lists the study as terminated. This limits what can be learned about outcomes in the intended population. Even if measurements or clinical events were available, a single-group study would not distinguish changes associated with treatment from changes related to disease course, other care, measurement variability, or chance. A concurrent control group was not part of the recorded design.
The registry lists secondary outcomes covering signaling markers, response, adverse events, progression-free survival, and overall survival. It records no analyses. Their inclusion as planned outcomes should not be mistaken for reported outcome estimates. The registry record does not report numerical results for response, survival, or the biological secondary endpoints.
The safety record lists 1 participant at risk and 1 affected for serious adverse events, and 1 at risk and 1 affected for non-serious adverse events. It also records 1 death. Listed serious-event terms include ascites, atrial fibrillation, death NOS, trunk edema, generalized muscle weakness, and hypotension, each shown as 100.0%. These entries describe what the registry reports; they do not establish that AZD9150 caused an event. The record does not provide event-level timing, clinical context, or an attribution assessment sufficient to interpret causality.
Any inferential precision is necessarily very limited when the recorded enrollment is 1 and the primary endpoint was not completed. The registry record does not report confidence intervals, p-values, adjusted analyses, or a statistical model for the outcomes. Without those results, readers cannot assess the magnitude or precision of an endpoint effect. The record supports a description of the study design, its termination, and its reported safety entries, but not a quantitative conclusion about the primary biological question.
Planning calculations based on an observed effect, where displayed, should not be interpreted as observed power or as evidence that the study was adequately sized. The registry's planning assumptions are illustrative and do not substitute for a completed endpoint analysis. In particular, the primary endpoint has no reported observed effect on which to base an outcome interpretation.
9. Statistical Methods Explained
Statistical interpretation depends on separating what the study planned to measure from what the registry reports as completed. The study planned a biological assessment of immune-cell percentages in ascitic fluid. The registry explicitly says that the primary outcome was not done. Thus, no primary endpoint estimate can be interpreted, regardless of the methods that might ordinarily be used for such measurements.
What does a single-group design allow?
In general, a single-group study can describe outcomes observed among people receiving an intervention. If measurements are collected before and after treatment, the within-person pattern can be described. But without a concurrent comparison group, that pattern does not isolate a treatment effect. Disease progression, other treatments, changes over time, and measurement variability can also contribute to observed differences.
Here, the registry records a single-group design, no masking, and allocation as not applicable. These details mean the design was not a randomized comparison. There is no control arm in the record against which to interpret changes. The study's pilot label and phase do not supply missing comparative evidence or establish the precision of any estimate.
What would an endpoint estimate mean?
In general, an endpoint estimate summarizes a measured quantity, such as a change in a biomarker or a proportion of participants with an outcome. A confidence interval describes uncertainty around an estimate under the assumptions of the analysis; it does not repair limitations in study design or missing measurements. For this trial, the primary endpoint has no posted values or estimate, and the registry reports no confidence interval for it. There is therefore no primary effect magnitude or precision to discuss.
The planned flow-cytometry measures were percentages of several immune-cell types. Those measurements would need to be interpreted as biological outcomes, not automatically as evidence of clinical benefit. The registry record does not report the planned marker values, their variability, or comparisons before and after therapy.
Why does the recorded enrollment matter?
The registry reports enrollment of 1 participant. In general, a very small amount of participant-level information provides little basis for estimating how outcomes vary across a target population. It also makes estimates sensitive to the experience of the individual enrolled. In this record, the primary outcome was not done, so there is no completed primary measurement to summarize even at the individual level.
The registry record does not report a sample-size justification. A sample-size calculation ordinarily depends on a defined target effect, variability assumptions, significance level, and desired power. Without a reported rationale, readers cannot assess how the planned enrollment was linked to the scientific question. Any sample-size planning illustrations should be read as hypothetical calculations, not as observed power or as evidence about the study's design adequacy.
How should the planning assumptions be read?
The registry's planning illustration uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It describes an effect to detect as a stated fraction of the observed effect. These are assumptions for planning calculations, not a description of the recorded trial design, which is single-group. A planning illustration asks what sample size might be associated with specified assumptions; it does not show that the trial achieved a particular probability of detecting an effect.
For time-to-event planning calculations, the stated method concerns a total number of events; other planning calculations are expressed as participants per arm. These conventions describe the hypothetical planning outputs, not outcomes observed in this study. The registry record does not report a trial-specific statistical plan that would connect those illustrative assumptions to the actual conduct of the study.
What is not available for evaluation?
The registry reports no analyses. It does not report an analysis population, a method for handling missing measurements, an approach to multiplicity, or an interim-analysis plan. It also does not report p-values, confidence intervals, endpoint estimates, or a statistical model. These are not details that can be inferred from the list of planned outcomes.
In general, multiplicity becomes relevant when researchers test or interpret several endpoints or several components of an endpoint. A prespecified approach can help control the risk of overinterpreting chance findings. Here, the registry lists multiple biological and clinical outcomes but does not report how they were to be analyzed together or whether any adjustment was planned. No conclusion about the use or absence of a particular adjustment can be drawn beyond noting that the plan is not reported in the registry record.
Likewise, missing-data methods matter when planned measurements are unavailable. The primary outcome is recorded as not done, but the registry does not describe an imputation method, a sensitivity analysis, or another missing-data procedure. The responsible interpretation is that the primary result is unavailable, not that a particular unreported method was used.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists several outcome domains, including immune-cell measures, STAT3-related signaling, p-STAT3 expression, response, adverse events, progression-free survival, and overall survival. It records seven outcome measures and no analyses. A collection of endpoints raises a general multiplicity issue: when many outcomes are tested or emphasized, chance findings can be harder to distinguish from reliable signals. The registry record does not report a multiplicity strategy, such as a hierarchy of outcomes or an adjustment for multiple testing.
The primary endpoint itself included several cell populations. The record does not say how these components were to be combined, prioritized, or tested. It also does not report a statistical analysis plan, so readers cannot determine whether these choices were prespecified elsewhere or how they would affect interpretation.
Missingness is particularly consequential because the registry says the primary outcome was not done. It attributes this to the one patient not making it to one scan after 8 weeks. No primary endpoint values or analysis are reported. The record does not describe a missing-data method, an imputation procedure, or sensitivity analyses. The absent endpoint should therefore be treated as unavailable rather than as evidence of no biological change.
The registry record also does not define an analysis population for the secondary outcomes or report how incomplete follow-up was handled. It does not state whether any interim analyses were planned. These details are not reported in the registry record, so conclusions about analysis choices cannot be drawn from the planned outcome list alone.
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 |
|---|---|---|---|
| AZD9150 in People With Malignant Ascites | 1 | 1 | 100.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| AZD9150 in People With Malignant Ascites | 1 of 1 |
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 |
|---|---|
| AZD9150 in People With Malignant Ascites | 1 of 1 |
Most frequent serious adverse event terms
| Event term | Organ system | AZD9150 in People With Malignant Ascites |
|---|---|---|
| Ascites | Gastrointestinal disorders | 1 of 1 |
| Atrial fibrillation | Cardiac disorders | 1 of 1 |
| Death NOS | General disorders | 1 of 1 |
| Edema trunk | General disorders | 1 of 1 |
| Generalized muscle weakness | Musculoskeletal and connective tissue disorders | 1 of 1 |
| Hypotension | Vascular disorders | 1 of 1 |
| Ileus | Gastrointestinal disorders | 1 of 1 |
| Nausea | Gastrointestinal disorders | 1 of 1 |
| Upper gastrointestinal hemorrhage | Gastrointestinal disorders | 1 of 1 |
| Vomiting | Gastrointestinal disorders | 1 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | AZD9150 in People With Malignant Ascites |
|---|---|---|
| Activated partial thromboplastin time prolonged | Investigations | 1 of 1 |
| Alanine aminotransferase increased | Investigations | 1 of 1 |
| Anemia | Blood and lymphatic system disorders | 1 of 1 |
| Anorexia | Metabolism and nutrition disorders | 1 of 1 |
| Aspartate aminotransferase increased | Investigations | 1 of 1 |
| Constipation | Gastrointestinal disorders | 1 of 1 |
| Creatinine increased | Investigations | 1 of 1 |
| Dehydration | Metabolism and nutrition disorders | 1 of 1 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 1 of 1 |
| Edema limbs | General disorders | 1 of 1 |
Serious events with the highest arm-level rate: ascites (100.0%), atrial fibrillation (100.0%), death nos (100.0%), edema trunk (100.0%), generalized muscle weakness (100.0%).
The registry safety entries list 1 participant at risk and 1 affected for serious adverse events, and 1 participant at risk and 1 affected for non-serious adverse events. It also records 1 death. The listed serious-event terms are ascites, atrial fibrillation, death NOS, trunk edema, generalized muscle weakness, and hypotension; each term is shown with 100.0%.
These figures should be read as registry-reported event information, not as an estimate of comparative risk. There was no control group, and the record reports enrollment of 1 participant. The percentages do not establish how an event relates to treatment or whether it was caused by the drug. The registry does not provide enough event-level context to determine timing, duration, severity details beyond the serious/non-serious classifications, or attribution to AZD9150.
In general, adverse-event reports describe events occurring in the study context; they do not, by themselves, prove causation. The registry entries also do not support a stable estimate of how often events would occur in a larger population. The listed event terms and death should therefore be described as reported safety information, with uncertainty about interpretation and no comparative treatment conclusion.
12. What the Result Does, and Does Not, Mean
The record documents a phase 2, single-group pilot study of AZD9150 in people with malignant ascites. Its central scientific aim was to assess changes in immune parameters in ascitic fluid after therapy. That primary outcome was not done, and the registry attributes this to the participant not making it to one scan after 8 weeks. No primary biomarker values, effect estimate, or uncertainty interval are reported.
The study therefore does not provide a reported primary endpoint result from which to judge the direction, size, or precision of a change in immune-cell percentages. The planned secondary outcomes also should not be mistaken for findings: the registry lists those outcomes but reports no analyses. The safety entries are reportable as registry records, but they do not establish that AZD9150 caused the listed events.
These limitations do not show that the intervention had no biological effect, nor do they show that it had one. The outcome needed to address the main biological question was not completed in the record. Similarly, the recorded death and adverse events cannot be interpreted as a comparative treatment effect because the study had no control group and the record lacks event-level attribution details.
In general, an uncontrolled pilot study may help describe feasibility or generate questions for further study, but those purposes require adequate reporting of what was completed and observed. The registry record does not provide a full account of treatment exposure or completed procedures. It also does not report a statistical analysis plan or endpoint analyses. The sound conclusion is narrow: the registry describes the planned study and a limited set of reported safety entries, while the primary immune-marker result remains unavailable.
13. Why This Trial Matters Statistically
This trial illustrates why planned endpoints and completed results must be distinguished. The registry describes a focused biological question: whether immune-cell parameters in malignant ascites would change following AZD9150 therapy. Yet the primary endpoint is recorded as not done. Without measured values, there is no effect estimate to evaluate and no basis for assessing its precision.
The record also shows the interpretive limits of a single-group pilot design. A treatment-associated pattern, if observed, would not by itself separate the intervention's contribution from other changes over time or from disease-related influences. Here, the absence of a comparator is coupled with enrollment of 1 participant and no reported primary endpoint result. Those features sharply limit the scope of statistical conclusions.
The distinction between safety reporting and causal interpretation is also important. Serious and non-serious event entries and a death are listed, but the registry record does not provide a comparison group or enough context to establish treatment attribution. Event counts and percentages should not be transformed into general risk estimates from this record.
Finally, the record lists multiple planned biological and clinical outcomes but reports no analyses or multiplicity approach. This makes it especially important not to treat a list of intended outcomes as evidence that results were obtained. The study matters statistically as an example of how design, endpoint completion, and transparent analysis reporting jointly determine what a trial can support.
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 for interpreting efficacy-related outcomes is that the primary immune-parameter endpoint was not done. The registry says this was because the one patient did not make it to one scan after 8 weeks. No measurements, estimates, or uncertainty intervals are reported for the primary question.
Enrollment was 1 participant, and the study had no comparison group. The results therefore cannot provide a like-for-like estimate of treatment effect or support broad generalization from the recorded experience. The registry also lists the study as terminated, but does not report a detailed reason for termination beyond the primary-outcome note.
The planned treatment and procedures are described, but the record does not establish which were completed or how much treatment was received. The registry lists secondary outcomes but reports no analyses. Statistical methods for endpoint estimation, missing data, multiplicity, analysis populations, and interim analyses are not reported in the registry record. Nor is a sample-size justification reported.
Safety entries are limited in interpretive detail. The record lists serious and non-serious adverse events and a death, but does not provide sufficient event-level information to establish causation or to estimate comparative risks. The single-group design further limits safety comparisons. These limitations mean the registry can describe the study's planned question and recorded status, but cannot provide a completed statistical answer to the primary biological question.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: AZD9150, a STAT3 Antisense Oligonucleotide, in People With Malignant Ascites — NCT02417753.
- Registry record last updated 2017-05-15; status last verified 2017-05.
- Results first posted 2017-05-15.
- 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
NCT02417753 is a phase 2 pilot study sponsored by the National Cancer Institute, examining AZD9150 in people with malignant ascites. The registry records a single-group, unmasked design and enrollment of 1 participant. The study began on 2015-04-03, had a primary completion date of 2016-04-07, and is listed as terminated.
The primary endpoint concerned changes in immune parameters in ascitic fluid. The registry states that it was not done because the one patient did not make it to one scan after 8 weeks. No analyses are recorded. Secondary outcomes covered biological markers, response, safety, and survival, but numerical analyses for those outcomes are not reported. Safety entries list serious and non-serious adverse events and 1 death; the record does not establish causation.