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 AZD1775 Plus Carboplatin-Paclitaxel in Squamous Cell Lung Cancer (NCT02513563), a na phase 2 study. The primary outcome is Progression Free Survival (PFS).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02513563 |
| Phase | Phase 2 |
| Condition | Lung Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 42 |
| Arms in the primary outcome | n/a |
| Lead sponsor | H. Lee Moffitt Cancer Center and Research Institute |
| Results posted | Yes |
2. Clinical Question
Population
The trial concerns participants with squamous cell lung cancer, as stated in its title. The primary endpoint population is described as evaluable participants. The registry record lists Lung Cancer as the condition and reports enrollment of 42 participants; it does not give the number included in the evaluable population.
Intervention
The intervention is a combination of AZD1775, carboplatin, and paclitaxel. The registry describes treatment twice daily on days 1 and 2 and once on day 3, for a total of 5 doses during each 21 day cycle of treatment. The trial was intended to examine effects, both good and bad, of this combination in participants and their cancer.
Comparator
The trial is recorded as single group, with allocation listed as NA and masking as none. It therefore does not define a separate control group for a like-for-like treatment comparison. The registry specifically notes that this is not a randomized comparison.
Primary question
The primary endpoint is median progression-free survival, measured in months, with an 80% confidence interval. The registry describes follow-up up to 2 years. The posted record does not report the median, its interval, or a numerical treatment-effect estimate, so the primary question cannot be answered from the registry results described in this record.
3. Trial Design
This is a phase II clinical trial recorded as completed. It enrolled 42 participants and used a single-group model. Allocation is listed as NA, and masking is listed as none. The registry record identifies H. Lee Moffitt Cancer Center and Research Institute as the sponsor. The recorded study start date is 2015-10-13, and primary completion is recorded as 2022-11-27.
All participants in the treatment group received the combination of carboplatin, paclitaxel, and AZD1775. The registry describes AZD1775 as being taken twice daily on days 1 and 2 and once on day 3, for a total of 5 doses in each 21 day cycle. It does not report a separate arm receiving only the chemotherapy combination or another control treatment. As a result, the design does not provide a concurrent randomized comparison between treatment strategies.
The primary endpoint is progression-free survival among evaluable participants, with a timeframe of up to 2 years. Secondary outcomes named in the record are overall survival, duration of overall response, and the percentage of participants with disease control. The record lists four outcome measures, but does not provide numerical results for them in the information available for this analysis.
The registry record does not report the protocol’s sample-size justification, its planned statistical analysis, interim analyses, or the criteria used to define evaluability. The completion status describes the trial’s administrative status; it does not by itself indicate whether a particular endpoint result was favorable, precise, or statistically conclusive.
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 | Carboplatin/Paclitaxel/AZD1775 |
|---|---|
| STARTED | 42 |
| COMPLETED | 33 |
| NOT COMPLETED | 9 |
Reasons for not completing this period:
| Reason | Carboplatin/Paclitaxel/AZD1775 |
|---|---|
| Death | 4 |
| Withdrawal by Subject | 4 |
| Physician Decision | 1 |
Analysis population for the primary outcome, as defined in the registry: Evaluable participants
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 | Carboplatin/Paclitaxel/AZD1775 |
|---|---|---|
| Participants analysed | 42 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 19 |
| Age, Categorical: >=65 years | Participants | 23 |
| Sex: Female, Male: Female | Participants | 14 |
| Sex: Female, Male: Male | Participants | 28 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 2 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 38 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 2 |
| 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 | 40 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Region of Enrollment: United States | participants | 42 |
6. Primary Endpoint
The registry lists the primary outcome as Progression Free Survival (PFS), measured over Up to 2 years.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 80% Confidence Interval |
| Unit | months |
| Endpoint classification | Time-to-Event Analysis |
Progression-free survival (PFS) is the time from the date of first study treatment until death, disease progression, or the last follow-up data, whichever comes first, according to the registry definition. Progression is defined in the record using changes in target-lesion diameters and the appearance of new lesions. The endpoint is assessed in evaluable participants, with a stated timeframe of up to 2 years.
The primary summary is the median PFS in months, accompanied by an 80% confidence interval. In general, a median time-to-event measure describes the time by which half of the relevant participants have experienced the defined event, subject to the endpoint’s rules for follow-up and censoring. It gives a time-based summary without requiring every participant to have experienced progression or death during observation.
In general, PFS is used to summarize how long participants remain alive without documented disease progression. It combines progression and death in one endpoint, so it is not a measure of either outcome separately. Its interpretation depends on how progression is assessed, how follow-up is handled, and who is included in the analysis. The registry record does not report the median PFS or its confidence interval, so the size and precision of the observed time-to-event result cannot be described.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry identifies median PFS as the primary endpoint and specifies an 80% confidence interval, but it does not report a median estimate, confidence limits, or a hazard ratio. It also lists 0 analyses. Consequently, the posted results described in this record do not provide a numerical primary result to interpret. No conclusion about the direction or size of the observed PFS outcome can be drawn from the endpoint definition alone.
In general, a median time-to-event estimate is a summary of the observed event-time distribution. Its confidence interval conveys uncertainty in that estimate under the analysis assumptions. A narrower interval would generally indicate greater precision than a wider interval, but no interval limits are reported. The specified 80% confidence level should be kept distinct from any claim about whether a treatment effect is established; an interval alone does not answer a comparative question, especially when there is no control group.
The single-group design is central to interpretation. Without a concurrent control group, the registry record cannot directly estimate how PFS under the combination compares with PFS under another treatment or with no study treatment. Outcomes observed in this group could be described, if numerical results were posted, but the design does not support a randomized between-group treatment contrast. The registry itself flags that the arms are not a like-for-like control comparison.
The title identifies the intended disease as squamous cell lung cancer, while the condition field lists Lung Cancer. The endpoint population is described as evaluable participants, but the size and definition of that population are not reported in the registry record. Enrollment of 42 does not establish that all enrolled participants contributed to the primary endpoint analysis.
Several additional outcomes are named, including overall survival, duration of overall response, and disease control. Their names indicate the topics measured, not the results. The record does not provide numerical findings for those outcomes. Nor does it report an analysis plan, adjustment approach, or explanation of how multiple outcomes were handled. Interpretation should therefore remain limited to the design, endpoint definition, and status information that the registry records, rather than implying an efficacy result from the trial’s completion or its stated purpose.
9. Statistical Methods Explained
Time-to-event methods address outcomes measured as elapsed time until a defined event, while allowing for participants whose event time is not observed during their available follow-up. For this trial, the registry defines PFS from first study treatment to death, progression, or last follow-up data, whichever comes first. The record describes progression using target-lesion changes and new lesions. These definitions determine what counts as an endpoint event and what information contributes to the time-to-event summary.
What does the median summarize?
In general, the median time-to-event estimate is the point in the event-time distribution at which half of the population represented by the analysis has had the event. When some participants have no recorded event before follow-up ends, their information is typically treated as censored rather than as an event at the end of observation. Censoring preserves information about the time they were observed, while acknowledging that their later event time is unknown. The registry record does not state the specific estimator or computational method used for the reported median.
In general, Kaplan–Meier methods are commonly used to estimate time-to-event distributions when follow-up times vary and some observations are censored. That is a general description of a method, not a claim about the method used in this trial: the analysis method is not reported in the registry record. Without a stated method, event counts, or numerical estimate, readers cannot independently assess how the PFS summary was constructed.
How should the confidence interval be read?
The registry specifies an 80% confidence interval around the median PFS. In general, a confidence interval expresses uncertainty around an estimated quantity under the assumptions of the method used. It is not a range containing every participant’s PFS time, nor does it describe the spread of individual outcomes. The interval limits are not reported in this record, so its precision cannot be assessed. The registry record also does not explain why this confidence level was selected.
Hazard ratios are another general way to summarize time-to-event comparisons. A hazard ratio compares event rates over follow-up between groups under the model used, but it does not directly state a difference in median times. This trial is recorded as single group, and no sponsor-reported hazard ratio is included in the registry record for this analysis. The record therefore does not support interpretation of a hazard ratio or a comparative time-to-event effect.
What do the planning assumptions mean?
The registry-related planning illustration is described as using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It states that the effect to detect is a stated fraction of the observed effect. For time-to-event illustrations, the calculation is described as a total number of events using the Schoenfeld method; other illustrations are described as participants per arm. These are hypothetical planning assumptions, not a report of the trial’s actual design or observed statistical power.
Those planning assumptions describe a two-arm allocation, whereas this trial is recorded as single group and the registry flags that there is no randomized, like-for-like control comparison. The illustrations should not be read as the trial’s own sample-size justification or as evidence that the study had a particular probability of detecting an effect. The record does not report the protocol’s sample-size rationale, and it does not provide the numerical planning outputs in the registry record for this analysis. No observed power can be inferred from such planning calculations.
In general, over-dispersion matters when observed variability in count data exceeds what a statistical model assumes; failing to account for it can make uncertainty appear too small. That concept is not a reported feature of this trial’s primary endpoint, which is a median time-to-event measure rather than a count outcome. The registry record does not report model diagnostics or other assumptions for the primary analysis.
The record identifies the primary population as evaluable participants. In general, an analysis population definition specifies which participants contribute to a particular estimate, and may differ from everyone enrolled. The registry does not define evaluability or state how missing endpoint information was handled. Thus, the enrollment figure alone cannot establish the analysis denominator or the amount of censoring.
10. Multiplicity, Missing Data and Analysis Populations
The registry names PFS as the primary outcome and lists overall survival, duration of overall response, and percentage of participants with disease control as secondary outcomes. It also states that there are four outcome measures and 0 analyses. The record does not report numerical estimates for these outcomes or describe a plan for controlling the risk of false-positive findings when multiple outcomes are assessed. Whether any multiplicity adjustment was planned or applied is not reported in the registry record.
In general, multiplicity arises when several outcomes or comparisons are examined: considering many results can increase the chance that at least one appears noteworthy by chance. A prespecified hierarchy or adjustment can help define how such results should be interpreted. No such approach is described in the registry information used here, so the relationship between the primary and secondary outcomes cannot be assessed statistically.
The registry describes the primary population as evaluable participants, but it does not report the number of evaluable participants or define the criteria for inclusion in that population. It also does not report how missing PFS assessments, incomplete follow-up, withdrawals, or other missing observations were handled. In general, missingness can affect a time-to-event estimate if the availability of follow-up is related to a participant’s outcome. The endpoint definition refers to last follow-up data, but that wording alone does not specify the censoring rules or establish how missing data were addressed.
The registry record does not report the analysis plan, any interim analyses, or the planned statistical treatment of secondary outcomes. Those details should not be inferred from the phase, completion status, or stated purpose of the trial.
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 |
|---|---|---|---|
| Carboplatin/Paclitaxel/AZD1775 | 26 | 42 | 61.9% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Carboplatin/Paclitaxel/AZD1775 | 4 of 42 |
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 |
|---|---|
| Carboplatin/Paclitaxel/AZD1775 | 40 of 42 |
Most frequent serious adverse event terms
| Event term | Organ system | Carboplatin/Paclitaxel/AZD1775 |
|---|---|---|
| Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 6 of 42 |
| Lung infection | Infections and infestations | 5 of 42 |
| Abdominal pain | Gastrointestinal disorders | 4 of 42 |
| Death NOS | General disorders | 3 of 42 |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 3 of 42 |
| Dehydration | Metabolism and nutrition disorders | 3 of 42 |
| Febrile neutropenia | Blood and lymphatic system disorders | 3 of 42 |
| Hypotension | Vascular disorders | 3 of 42 |
| Respiratory, thoracic and mediastinal disorders -Other | Respiratory, thoracic and mediastinal disorders | 3 of 42 |
| Acute kidney injury | Renal and urinary disorders | 3 of 42 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Carboplatin/Paclitaxel/AZD1775 |
|---|---|---|
| Diarrhea | Gastrointestinal disorders | 21 of 42 |
| Fatigue | General disorders | 21 of 42 |
| Nausea | Gastrointestinal disorders | 20 of 42 |
| Anemia | Blood and lymphatic system disorders | 17 of 42 |
| Vomiting | Gastrointestinal disorders | 12 of 42 |
| Pain | General disorders | 10 of 42 |
| Abdominal Pain | Gastrointestinal disorders | 9 of 42 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 9 of 42 |
| Constipation | Gastrointestinal disorders | 7 of 42 |
| Anorexia | Metabolism and nutrition disorders | 7 of 42 |
Serious events with the highest arm-level rate: neoplasms benign, malignant and unspecified (incl cysts and polyps) - other (14.3%), lung infection (11.9%), abdominal pain (9.5%), death nos (7.1%), respiratory failure (7.1%).
The registry’s adverse-event summary lists 42 participants at risk for serious events and 26 affected, and 42 at risk for other events and 40 affected. It also records 4 deaths. These are group-level figures for the Carboplatin/Paclitaxel/AZD1775 group. They describe the reported counts, not whether an event was caused by the study treatment. The record does not provide a causal assessment in the information.
The listed serious-event terms and percentages are Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, 14.3%; Lung infection, 11.9%; Abdominal pain, 9.5%; Death NOS, 7.1%; Respiratory failure, 7.1%; and Dehydration, 7.1%. These are registry-listed terms, and the percentages should be read as reported rather than converted into counts or treated as proof of a treatment relationship.
In general, participants can experience more than one adverse event, and the same participant may contribute to more than one event category. Therefore, category totals should not be added to estimate the number of participants affected. The registry summary also does not, by itself, describe event timing, duration, resolution, or the clinical context of each event. Deaths recorded in a safety summary should not be assumed to have been caused by treatment unless the record states that relationship.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a completed phase II, single-group study evaluated a combination of AZD1775, carboplatin, and paclitaxel in a trial titled for squamous cell lung cancer. It specifies PFS as the primary endpoint and defines how progression, death, and follow-up enter that measure. It also describes the treatment schedule and lists several secondary outcomes.
The posted information does not establish the numerical PFS result. A median and 80% confidence interval are identified as the intended summary, but their values are not reported in the registry record. The record also lists 0 analyses. Without estimates and uncertainty measures, the observed PFS experience cannot be characterized for magnitude or precision. Completion status is not a substitute for an outcome result.
The study’s single-group design also limits what can be concluded comparatively. Even if a group-level outcome estimate were available, it would describe participants receiving the combination rather than isolate the combination’s effect relative to a concurrent control. Changes over time or outcomes in a single group cannot, by themselves, rule out other explanations for the observed course.
The adverse-event summary reports participant counts and selected serious-event terms, but it does not establish that the combination caused those events. The registry-listed categories are descriptive and should not be interpreted as a comparative safety assessment because there is no control group in this design.
In general, clinical interpretation requires the endpoint estimates, their uncertainty, the analysis population, and the methods used to handle follow-up and missing information. Several of those elements are not reported in this registry record. The appropriate reading is therefore limited: the record describes the trial’s design, treatment, endpoint definitions, and some safety summaries, but it does not provide enough outcome analysis to quantify a treatment effect or judge its precision.
13. Why This Trial Matters Statistically
This trial illustrates why study design and outcome reporting need to be read together. The registry identifies a clinically defined treatment combination and a time-to-event primary endpoint, but the design is single group. That distinction matters because an endpoint can describe what occurred among treated participants while still not providing a controlled estimate of how outcomes differ under another strategy.
It also highlights the difference between specifying an endpoint and reporting its result. The PFS definition is detailed, and the record identifies median PFS with an 80% confidence interval. Yet the numerical median and interval are not reported in the registry record. A clearly defined outcome makes the intended measurement understandable, but does not reveal its observed value or precision.
The record also provides a useful example of why planning calculations must be separated from trial evidence. The stated planning assumptions describe a hypothetical two-arm calculation, whereas the actual trial is recorded as single group. Such illustrations are not observed power and should not be mistaken for the trial’s own sample-size rationale or a test of its results.
Finally, the safety summary illustrates the limits of descriptive event listings. Counts and percentages can identify what the registry recorded, but causal attribution and comparative safety require context that these figures alone do not supply. Together, these features show the importance of distinguishing design details, planned statistical quantities, observed outcome estimates, and causal conclusions.
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 principal limitation for statistical interpretation is that the registry record does not report the numerical primary PFS result, its 80% confidence interval, or a sponsor-reported hazard ratio. Although the study is marked completed, the information does not permit an assessment of the observed time-to-event outcome or its precision.
The trial is recorded as single group, with allocation listed as NA and masking as none. There is no randomized concurrent control group for a treatment comparison, and the registry explicitly cautions that the arms are not a like-for-like control comparison. Accordingly, any group-level observations would not alone establish a comparative treatment effect.
The primary endpoint is assigned to evaluable participants, but the number and definition of evaluable participants are not reported in the registry record. Details about missing assessments, loss to follow-up, censoring, analysis methods, and protocol-defined statistical procedures are also not reported. The enrollment of 42 participants should not be treated as the analysis population size.
The record names secondary outcomes but does not provide their numerical results or explain how multiple outcomes were handled. It also does not report a sample-size justification or interim analyses. The safety summary is descriptive and does not establish causality. These omissions constrain interpretation; they do not establish that an analysis or procedure was absent from the underlying study, only that it is not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: AZD1775 Plus Carboplatin-Paclitaxel in Squamous Cell Lung Cancer — NCT02513563.
- Registry record last updated 2026-02-19; status last verified 2026-02.
- Results first posted 2024-01-10.
- 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 a completed phase II study titled AZD1775 Plus Carboplatin-Paclitaxel in Squamous Cell Lung Cancer. It is recorded as a single-group trial with no masking, enrollment of 42, and a treatment group receiving AZD1775 with carboplatin and paclitaxel. The listed primary outcome is median PFS in months, with an 80% confidence interval and a timeframe of up to 2 years.
The registry defines PFS using death, progression, and last follow-up data, and identifies evaluable participants as the primary endpoint population. Overall survival, duration of overall response, and disease control are listed as secondary outcomes. The record includes adverse-event summaries, but no numerical primary outcome estimate or analysis is reported in the information. The registry does not report the statistical plan, sample-size justification, missing-data handling, or multiplicity approach.