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 to Assess the Effect of AZD9291 on the Blood Levels of Rosuvastatin, in Patients With EGFRm+ Non-small Cell Lung Cancer (NCT02317016), a na phase 1 study. The primary outcome is Assessment of Maximum Plasma Concentration (Cmax) for Rosuvastatin After a Single Dose Alone and in Combination With AZD9291.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02317016 |
| Phase | Phase 1 |
| Condition | Non Small Cell Lung Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 44 |
| Arms in the primary outcome | n/a |
| Lead sponsor | AstraZeneca |
| Results posted | Yes |
2. Clinical Question
Population
Patients with EGFR mutation-positive non-small cell lung cancer whose disease had progressed following prior therapy with an approved EGFR tyrosine kinase inhibitor. The planned enrollment was 44.
Intervention
AZD9291 was administered in a sequential treatment design. The study assessed rosuvastatin after a single dose alone and after a single dose given with AZD9291.
Comparator
Rosuvastatin alone in an earlier treatment period was compared with rosuvastatin given together with AZD9291 in a later period. This was not a randomized, parallel-group comparison.
Primary question
How did rosuvastatin’s maximum plasma concentration, or Cmax, compare when rosuvastatin was taken alone versus in combination with AZD9291?
The study’s central question is pharmacokinetic: whether the measured peak concentration of rosuvastatin differed between the two treatment conditions. It is not a trial designed to compare cancer outcomes between AZD9291 and another cancer treatment. Rather, it evaluates rosuvastatin exposure in the presence and absence of AZD9291 in a specific patient population.
In Part A, patients received treatments in sequence: rosuvastatin alone, AZD9291 alone, and then rosuvastatin with AZD9291. The primary endpoint concerns the rosuvastatin doses given alone and in combination. Blood samples were collected at multiple times after rosuvastatin dosing on Days 1 and 32. The planned analysis population included dosed patients with at least one quantifiable post-dose plasma concentration and no important deviations or events that would exclude them.
The registry describes a primary endpoint and its measurement plan, but the numerical Cmax results and a numerical estimate of the difference between treatment conditions are not reported in the registry record. The clinical question can therefore be described, but the size and precision of the observed pharmacokinetic contrast cannot be read from this record.
3. Trial Design
This was a Phase I, open-label, non-randomised, multicentre, single-group study. The registry lists a planned enrollment of 44 and describes the study as having two parts. The study began on 2015-03-10, had a primary completion date of 2015-07-11, and is listed as completed.
In Part A, patients received rosuvastatin alone, AZD9291 alone, and then rosuvastatin with AZD9291 in sequential treatment periods. The primary pharmacokinetic comparison uses a single rosuvastatin dose alone in Period 1 and a single rosuvastatin dose together with AZD9291 in Period 3. AZD9291 was administered as multiple oral doses in the fasted state during the pharmacokinetic part. The listed primary sampling schedule collected blood on Days 1 and 32 before dosing and at multiple post-dose times through 72 hours.
Part B allowed patients who completed Part A to continue to receive AZD9291 80 mg once daily as a single agent, with additional safety data collection. The registry describes continuation until disease progression, no longer deriving clinical benefit, or another reason. Part B therefore has a different role from the controlled pharmacokinetic periods: it provides further access to AZD9291 and additional safety observation.
Because treatments were given sequentially to one group, the comparison is within the treatment sequence rather than between randomized groups. The registry lists no allocation and no masking. That structure can help compare measurements from the same participants, but treatment condition is also tied to period and sequence. The registry record does not report a separate randomized control group, a sample-size justification, or details of methods used to account for period effects.
Several secondary outcomes describe other rosuvastatin pharmacokinetic measures, including Tmax, AUC0-t, apparent clearance, apparent volume of distribution, and terminal elimination half-life. Other listed outcomes describe AZD9291 and its metabolites. The record does not give numerical results for those measures in 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.
Part A: Day 1-3 (Rosuvastatin Alone)
| Stage | Rosuvastatin and AZD9291 (Part A); AZD9291 Alone (Part B) |
|---|---|
| STARTED | 44 |
| COMPLETED | 44 |
| NOT COMPLETED | 0 |
Part A: Day 4-31 (AZD9291 Alone)
| Stage | Rosuvastatin and AZD9291 (Part A); AZD9291 Alone (Part B) |
|---|---|
| STARTED | 44 |
| COMPLETED | 43 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Rosuvastatin and AZD9291 (Part A); AZD9291 Alone (Part B) |
|---|---|
| Adverse Event | 1 |
Part A: Day 32-34 (Rosuvastatin+AZD9291)
| Stage | Rosuvastatin and AZD9291 (Part A); AZD9291 Alone (Part B) |
|---|---|
| STARTED | 43 |
| COMPLETED | 41 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | Rosuvastatin and AZD9291 (Part A); AZD9291 Alone (Part B) |
|---|---|
| Condition under investigation worsened | 1 |
| Death | 1 |
Part B: Day 35 to End Part B (AZD9291)
| Stage | Rosuvastatin and AZD9291 (Part A); AZD9291 Alone (Part B) |
|---|---|
| STARTED | 42 |
| COMPLETED | 11 |
| NOT COMPLETED | 31 |
Reasons for not completing this period:
| Reason | Rosuvastatin and AZD9291 (Part A); AZD9291 Alone (Part B) |
|---|---|
| Condition under investigation worsened | 23 |
| Adverse Event | 1 |
| Withdrawal by Subject | 2 |
| Death | 5 |
Analysis population for the primary outcome, as defined in the registry: The PK analysis set included all dosed patients who had at least 1 quantifiable plasma concentration collected post-dose without any important deviations or events that would exclude the patient.
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 in the safety analysis set who received at least one dose of AZD9291 or rosuvastatin (n = 44) were included in the baseline analysis.
| Characteristic | Unit | Rosuvastatin and AZD9291 (Part A); AZD9291 Alone (Part B) |
|---|---|---|
| Participants analysed | 44 | |
| Age, Continuous | years | 61.5 (SD 11.37) |
| Sex: Female, Male: Female | Participants | 30 |
| Sex: Female, Male: Male | Participants | 14 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 2 |
| Race (NIH/OMB): White | Participants | 41 |
| 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 Assessment of Maximum Plasma Concentration (Cmax) for Rosuvastatin After a Single Dose Alone and in Combination With AZD9291, measured over Blood samples collected on Days 1 and 32 at pre-dose, 0.5, 1, 1.5, 2, 3, 4, 5, 6, 8, 10, 12, 24, 36, 48, 60, and 72 hours post rosuvastatin dose in Part A..
| Element | Registry entry |
|---|---|
| Result type | Geometric Mean |
| Measure of spread | Geometric Coefficient of Variation |
| Unit | nanogram per millilitre (ng/mL) |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is rosuvastatin maximum plasma concentration, or Cmax, measured in nanograms per millilitre. Cmax is the highest measured concentration in the blood after a dose. Here, it is intended to characterize the peak exposure to rosuvastatin after a single dose given alone and after a single dose given with AZD9291.
The endpoint is relevant to a pharmacokinetic interaction question because a change in drug exposure can be assessed by comparing concentration profiles under different treatment conditions. Cmax focuses on the peak, rather than the total exposure across the sampling period. Other listed outcomes, such as area under the concentration-time curve, address exposure over time; they are distinct measures and should not be treated as interchangeable with Cmax.
The primary endpoint is described using a geometric mean and geometric coefficient of variation. These summaries are commonly used for concentration measures that can be skewed, since positive values may vary multiplicatively rather than symmetrically. In general, a geometric mean summarizes the central level after working on a logarithmic scale and returning to the original scale. A geometric coefficient of variation describes relative spread on that scale. Those are general statistical explanations; the registry record does not report the detailed calculation procedure used in this study.
Although the endpoint, unit, sampling schedule, and analysis population are described, the numerical Cmax summaries for each condition and an estimated contrast with an uncertainty interval are not reported in the registry record. Without those results, the endpoint definition alone does not establish the magnitude or precision of any difference.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the question this study was designed to examine: rosuvastatin Cmax after a dose alone compared with Cmax after a dose given with AZD9291. It also describes the sequential treatment periods, the blood-sampling plan, and the intended pharmacokinetic analysis population. These details identify the design and outcome, but they do not disclose the numerical primary result.
The Cmax estimates for rosuvastatin under the two treatment conditions are not reported in the registry record. A numerical contrast, confidence interval, and p-value for the primary comparison are also not reported in the registry record. As a result, this record does not show how large the observed change was, which condition had the higher estimate, or how precisely the comparison was estimated. Those omissions prevent a result-focused interpretation of the primary endpoint from this record alone.
In general, a confidence interval gives a range of values compatible with the observed data and the method used to construct it. A narrow interval indicates more precision than a wide interval, but neither width nor location can be assessed because a primary confidence interval is not reported in the registry record. A p-value, if reported, would address how compatible the data are with a specified null model; it would not by itself describe the size or practical importance of a pharmacokinetic contrast. No primary p-value is reported in the registry record.
The study’s sequential, non-randomised design also matters when interpreting any within-person contrast. Measuring participants in more than one treatment period can reduce the influence of stable differences between people. However, treatment condition follows a fixed sequence in the described design, so treatment and period are not separated by random allocation. Changes over time, carryover, or other period-related influences could matter in principle. The registry record does not report whether or how such issues were assessed.
Enrollment of 44 refers to planned study enrollment, not necessarily the number contributing to every pharmacokinetic endpoint. The registry’s analysis-set description requires dosing, at least one quantifiable post-dose concentration, and no important exclusionary deviations or events. The number included in the primary pharmacokinetic analysis is not reported in the registry record.
The safety figures are descriptive, and the overall, Part A, and Part B safety populations are not independent parallel groups. Part B follows the Part A pharmacokinetic phase for patients who continued. Its counts should not be read as a randomized comparison, and the group totals should not be added to produce a unique-participant total. The registry record reports serious and other adverse-event counts by safety population, but these figures do not supply the missing Cmax result.
9. Statistical Methods Explained
What does the endpoint summarize?
The primary endpoint is Cmax, the maximum measured plasma concentration of rosuvastatin after a single dose. The registry describes Cmax using a geometric mean and geometric coefficient of variation, with concentration expressed in nanograms per millilitre. The comparison is between rosuvastatin alone and rosuvastatin administered together with AZD9291. This is a pharmacokinetic endpoint: it describes drug concentration, not a clinical cancer outcome.
In general, concentration data are often right-skewed: a smaller number of high values can make the distribution asymmetric. A logarithmic scale can make multiplicative differences easier to model, and a geometric mean can summarize the central concentration on the original scale. The geometric coefficient of variation describes relative variability. These are general explanations of the statistical terms; the registry record does not report the study’s exact formulas or estimation procedures.
Why use repeated sampling?
Cmax is determined from concentrations observed over time, so the sampling schedule is central to its measurement. The registry lists blood collection on Days 1 and 32, before dosing and at multiple post-dose times through 72 hours. More than one sampling time helps describe the concentration profile and locate the highest measured value. In general, the observed maximum depends on when samples are taken; the schedule does not guarantee that a sample is taken at the exact biological peak.
The record also lists Tmax, which concerns the time to maximum concentration, and several measures of overall exposure and drug disposition. These outcomes address related but distinct aspects of the concentration profile. A higher Cmax would not, on its own, establish a change in total exposure, just as a measure of total exposure would not determine the peak concentration.
What is the comparison structure?
Participants received treatments in sequence, with rosuvastatin alone in an earlier period and rosuvastatin with AZD9291 in a later period. This is not a randomized comparison between separate treatment arms. In general, within-person comparisons can account for stable characteristics shared by measurements from the same person. At the same time, a fixed treatment sequence can make treatment condition difficult to distinguish from period effects. The registry record does not report whether the analysis adjusted for period, sequence, or carryover, or whether those effects were assessed.
The registry describes a pharmacokinetic analysis set: dosed patients with at least one quantifiable post-dose plasma concentration and without important deviations or events that would exclude them. This definition indicates that inclusion depends on data availability and eligibility for analysis. The number of participants meeting that definition is not reported in the registry record.
What can uncertainty measures tell us?
In general, an estimated treatment contrast is more informative when paired with an uncertainty interval. A confidence interval communicates the range of effect values reasonably compatible with the observed data under the chosen method. Its interpretation depends on the analysis and assumptions; it is not a guarantee that the true value lies in the interval. A p-value can address compatibility with a null hypothesis, but it does not quantify the size of an effect or its practical importance.
The registry record does not report a numerical Cmax contrast, confidence interval, or p-value. It therefore does not allow a reader to judge either the direction or precision of the primary comparison. The analysis method used to estimate a contrast is also not reported in the registry record. The endpoint description identifies geometric summaries, but it does not specify a model, transformation, adjustment set, or inferential procedure.
Why does over-dispersion matter?
In general, over-dispersion occurs when observed variability is greater than a statistical model expects. If it is ignored, standard errors can be too small and uncertainty intervals too narrow. That issue is especially relevant to count outcomes when a model assumes a particular relationship between the mean and variance. This trial’s primary endpoint is a continuous pharmacokinetic measure, and the registry record does not identify a count model or report an over-dispersion assessment. Over-dispersion should not be presumed to have affected this Cmax analysis.
What do the safety counts represent?
The adverse-event section reports safety populations and numbers affected by serious and other adverse events. These are descriptive counts for the named populations, not estimates of a randomized treatment contrast. The overall safety population and the Part A and Part B populations relate to stages of the same study and may include participants observed across sequential parts. General statistical interpretation therefore requires attention to the population definitions and possible overlap. The registry does not provide a randomized comparison from which to infer comparative safety effects.
Overall, the registry describes the endpoint and study structure but leaves key inferential details unavailable. The sample-size justification, specific statistical analysis plan, handling of missing pharmacokinetic observations, and any interim analyses are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The study lists a primary Cmax outcome and multiple secondary pharmacokinetic outcomes. These include other rosuvastatin measures and measures for AZD9291 and its metabolites. When a study evaluates several outcomes, the chance of at least one apparently unusual result can increase if each is tested separately without adjustment. In general, multiplicity procedures can control the overall error rate or define a hierarchy for interpreting tests. The registry record does not report whether any multiplicity adjustment or testing hierarchy was used.
The registry identifies the primary outcome and gives its sampling timeframe and intended analysis population, but does not report the detailed statistical analysis plan. The prespecified significance threshold, any adjustment for repeated endpoints, and whether analyses were exploratory or confirmatory are not reported in the registry record. The number of listed outcomes alone does not establish that each was formally tested.
The primary analysis set includes dosed patients with at least one quantifiable post-dose concentration and no important deviations or events that would exclude them. This is an endpoint-specific population description, not a full account of how missing data were handled. The registry record does not report how many participants lacked usable measurements, why observations might be missing, or whether any imputation or sensitivity analysis was used.
In general, missing concentration measurements can affect a pharmacokinetic estimate if missingness is related to a participant’s concentrations or treatment experience. Excluding participants or observations can also change the population represented by the analysis. Here, the stated analysis-set criteria indicate some exclusions may apply, but their number and impact are not reported in the registry record.
The record also does not report an interim analysis plan or a sample-size justification. The planned enrollment is 44, but that figure alone does not establish the precision intended for the primary comparison or the number ultimately analyzed. These omissions limit assessment of the analysis decisions and the reliability of inferences beyond the endpoint description.
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 |
|---|---|---|---|
| Overall Safety Population | 7 | 44 | 15.9% |
| Part A Safety Population | 2 | 44 | 4.5% |
| Part B Safety Population | 6 | 42 | 14.3% |
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 |
|---|---|
| Overall Safety Population | 40 of 44 |
| Part A Safety Population | 34 of 44 |
| Part B Safety Population | 29 of 42 |
Most frequent serious adverse event terms
| Event term | Organ system | Overall Safety Population | Part A Safety Population | Part B Safety Population |
|---|---|---|---|---|
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | 1 of 44 | 1 of 44 | 1 of 42 |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | 1 of 44 | 0 of 44 | 1 of 42 |
| Fatigue | General disorders | 1 of 44 | 0 of 44 | 1 of 42 |
| Pneumonia | Infections and infestations | 1 of 44 | 0 of 44 | 1 of 42 |
| Pneumonia pseudomonal | Infections and infestations | 1 of 44 | 0 of 44 | 1 of 42 |
| Blood creatine phosphokinase increased | Investigations | 1 of 44 | 1 of 44 | 0 of 42 |
| Transaminases increased | Investigations | 1 of 44 | 0 of 44 | 1 of 42 |
| Transient ischaemic attack | Nervous system disorders | 1 of 44 | 0 of 44 | 1 of 42 |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | 1 of 44 | 0 of 44 | 1 of 42 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Overall Safety Population | Part A Safety Population | Part B Safety Population |
|---|---|---|---|---|
| Decreased appetite | Metabolism and nutrition disorders | 16 of 44 | 9 of 44 | 9 of 42 |
| Diarrhoea | Gastrointestinal disorders | 13 of 44 | 9 of 44 | 9 of 42 |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | 12 of 44 | 8 of 44 | 4 of 42 |
| Cough | Respiratory, thoracic and mediastinal disorders | 11 of 44 | 4 of 44 | 8 of 42 |
| Asthenia | General disorders | 9 of 44 | 4 of 44 | 7 of 42 |
| Fatigue | General disorders | 8 of 44 | 5 of 44 | 3 of 42 |
| Nausea | Gastrointestinal disorders | 7 of 44 | 4 of 44 | 3 of 42 |
| Constipation | Gastrointestinal disorders | 6 of 44 | 4 of 44 | 3 of 42 |
| Arthralgia | Musculoskeletal and connective tissue disorders | 6 of 44 | 2 of 44 | 5 of 42 |
| Pyrexia | General disorders | 6 of 44 | 3 of 44 | 4 of 42 |
Serious events with the highest arm-level rate: gastrooesophageal reflux disease (2.4%), fatigue (2.4%), pneumonia (2.4%), pneumonia pseudomonal (2.4%), transaminases increased (2.4%).
The safety section reports an overall safety population of 44, with 7 affected by serious adverse events and 40 affected by other adverse events. For Part A, the safety population is 44, with 2 affected by serious adverse events and 34 by other adverse events. For Part B, the safety population is 42, with 6 affected by serious adverse events and 29 by other adverse events.
These are counts of participants affected in the named safety populations, not measures of event severity beyond the serious-event classification and not randomized comparisons. Part A and Part B are stages within a sequential study, and the record does not establish that their populations are independent. The counts should not be added across groups to infer a total number of unique participants.
The registry lists six serious-event terms at 2.4% each: gastrooesophageal reflux disease, fatigue, pneumonia, pseudomonal pneumonia, increased transaminases, and transient ischaemic attack. These percentages are reported as presented in the registry; the record does not give a fuller event-level breakdown. The number of deaths is not reported in the registry record, so a blank or unavailable death entry should not be interpreted as zero deaths.
Safety counts describe what was recorded in these study populations. Without a randomized control group and further detail on event timing and attribution, they do not establish a comparative safety effect.
12. What the Result Does, and Does Not, Mean
This study addresses a focused pharmacokinetic question in patients with EGFR mutation-positive non-small cell lung cancer whose disease had progressed after an approved EGFR tyrosine kinase inhibitor. Its primary endpoint is the maximum plasma concentration of rosuvastatin after a single dose alone and after a single dose with AZD9291. The treatment sequence and concentration-sampling schedule are described in the registry record.
What the record does not provide is the numerical Cmax result. It does not report the condition-specific estimates, a contrast between conditions, a confidence interval, or a p-value for that primary comparison. Therefore, it is not possible from this record to describe the observed direction, size, or statistical precision of the primary result. The absence of those values is not evidence that the concentrations were the same, nor is it evidence that they differed.
The study’s single-group, non-randomised structure is important. It compares treatment periods in sequence rather than comparing separate randomized groups. In general, this can offer within-person information, but the fixed sequence also means that period-related changes may be entangled with treatment condition unless addressed by design or analysis. Whether the analysis addressed such issues is not reported in the registry record.
The listed adverse-event counts provide descriptive safety information for the overall and part-specific populations. They do not answer the primary pharmacokinetic question, and they should not be interpreted as randomized comparisons. The record also does not report the number included in the primary analysis or how missing measurements were handled.
Thus, the study record makes the intended question and endpoint clear, but the result details needed to judge the primary comparison are unavailable. It supports an explanation of the design and methods, not a conclusion about the magnitude or importance of a rosuvastatin exposure change.
13. Why This Trial Matters Statistically
This trial is statistically interesting because it frames a drug-interaction question using measured plasma concentrations rather than a clinical outcome. Its primary endpoint, rosuvastatin Cmax, captures the peak of the concentration profile after a single dose. The design also includes other pharmacokinetic measures, which can describe different features of exposure and disposition rather than repeating the same endpoint.
The sequential design highlights a common trade-off in pharmacokinetic studies. Comparing conditions within the same participants can reduce the influence of stable differences between people. But when treatment conditions occur in a fixed order, the comparison can also be influenced by period or sequence. Understanding such a design requires knowing not only the endpoint, but also how treatment periods were handled analytically. That detail is not reported in the registry record.
The record illustrates why an endpoint definition is not the same as a reported result. The Cmax measure, unit, sampling times, and analysis-set description are available, while the numerical estimates and uncertainty for the primary contrast are not reported in the registry record. Without those, a reader cannot assess the size or precision of the comparison. A complete statistical interpretation depends on both the design and the estimates with their uncertainty.
The safety section also shows why populations and study stages need careful reading. Overall, Part A, and Part B figures describe related populations in a sequential study, rather than independent randomized groups. Treating those counts as separate arms would misrepresent the design.
For clinical researchers and biostatisticians, the key lesson is to distinguish the question a study was designed to answer, the method used to measure it, and the evidence actually available in its record. Here, the first two are described more fully than the numerical primary result.
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 this study from the registry record is that numerical Cmax results are not reported. The record does not provide condition-specific estimates, the treatment contrast, an uncertainty interval, or a p-value. Consequently, the primary comparison’s direction, magnitude, and precision cannot be assessed from this record.
The design was open-label, non-randomised, and single-group, with treatments administered in sequence. This is not a randomized parallel-group comparison. In general, repeated measurements within participants can be useful, but treatment condition can be entangled with period in a fixed sequence. The registry record does not report whether period effects, carryover, or sequence-related considerations were addressed.
Although the analysis population is described, the number of participants included in the primary pharmacokinetic analysis is not reported in the registry record. The number and reasons for missing or non-quantifiable measurements, the handling of missing data, and sensitivity analyses are also not reported. Planned enrollment of 44 does not establish the number contributing to the endpoint.
The record does not report the sample-size justification, detailed statistical analysis plan, multiplicity strategy, interim analysis plan, or specific inferential method for the Cmax comparison. It also does not provide numerical results for the listed secondary outcomes in the registry record. These omissions restrict evaluation of the study’s precision, analysis choices, and consistency across pharmacokinetic measures.
Finally, the safety populations correspond to the overall study and its two parts. Their counts should be interpreted descriptively and not as results from independent treatment groups. The death entry is not reported in the registry record, and therefore does not establish that no deaths occurred.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study to Assess the Effect of AZD9291 on the Blood Levels of Rosuvastatin, in Patients With EGFRm+ Non-small Cell Lung Cancer — NCT02317016.
- Registry record last updated 2018-07-31; status last verified 2018-06.
- Results first posted 2016-07-14.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
This completed Phase I study enrolled patients with EGFR mutation-positive non-small cell lung cancer whose disease had progressed after prior therapy with an approved EGFR tyrosine kinase inhibitor. It was open-label, non-randomised, multicentre, and single-group, with a planned enrollment of 44.
Part A used sequential treatments of rosuvastatin alone, AZD9291 alone, and rosuvastatin with AZD9291. The primary endpoint was rosuvastatin Cmax after a single dose alone and in combination. Part B allowed patients completing Part A to continue AZD9291 and contributed additional safety data.
The registry describes the primary endpoint, sampling schedule, analysis-set criteria, and safety counts. Numerical Cmax results and the detailed statistical analysis plan are not reported in the registry record, so the primary comparison’s size and precision cannot be assessed.