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 Evaluating the Pharmacokinetic and Mass Balance of Single Dose [14C] AZD9291 in Volunteers (NCT02096679), a non-randomized phase 1 study. The primary outcome is Percentage of Radioactive Dose of [14C] Radiolabelled AZD9291 Recovered in Urine, Faeces, and in Total..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02096679 |
| Phase | Phase 1 |
| Condition | Healthy Volunteers |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 8 |
| Arms in the primary outcome | n/a |
| Lead sponsor | AstraZeneca |
| Results posted | Yes |
2. Clinical Question
Population
Healthy male volunteers were eligible for this pharmacokinetic and mass-balance study. The registry record lists an enrollment of 8. The primary outcome population is described as participants who received one dose of [14C]-AZD9291 and had at least one postdose pharmacokinetic measurement, without important protocol deviations or events thought to significantly affect pharmacokinetics.
Intervention
Participants received a single oral dose of AZD9291 as a solution. The described dose was 20mg of [14C]-AZD9291, containing nominal 1 μCi activity. This radiolabelled dose allowed measurement of radioactive material in collected samples.
Comparator
There was no randomized or concurrent control group. The study is listed as non-randomized and single-group, with no masking. Its design therefore describes measurements after dosing in the enrolled volunteers, not a like-for-like comparison between treatment arms.
Primary question
The primary question was what percentage of the radioactive dose was recovered in urine, faeces, and in total, up to Day 85. The registry record identifies the mean and standard deviation as the intended summary statistics, but numerical results for this endpoint are not reported in the registry record.
3. Trial Design
This was a phase I, single-centre, non-randomized, open-label pharmacokinetic and mass-balance study in healthy male volunteers. It is listed as a single-group study, with an enrollment of 8. The registry record identifies AstraZeneca as the sponsor and lists the study as completed. The recorded start was 2014-05, and primary completion was 2014-08.
Participants were assigned no randomized comparator in the design described by the record. Each received a single oral dose of [14C]-AZD9291 as a solution. The arm description specifies 20mg, with nominal 1 μCi activity. Open-label means masking was not used; the registry also lists masking as none. These design features are relevant when interpreting the study as a descriptive assessment of drug-related radioactivity and pharmacokinetics, rather than as a controlled comparison of treatment effects.
The primary outcome concerns the percentage of the radioactive dose recovered in urine, faeces, and in total, up to Day 85. The registry describes extensive planned sampling: blood measurements at specified post-dose times, urine collections across specified intervals, and faecal collections beginning over the first 24 hours and then following the urine schedule. The schedule indicates the intended collection framework; it does not establish that every planned sample was obtained or quantify completeness of collection.
Other listed outcomes address pharmacokinetic measures for AZD9291, its metabolites, and whole-blood and plasma radioactivity. These include areas under the concentration-time curve over different intervals, maximum concentration, time to maximum concentration, lag time, apparent oral clearance, and apparent volume of distribution. The record lists 20 outcome measures overall, but numerical results for the primary endpoint and detailed results for these other measures are not reported in the registry record. It lists 0 analyses.
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.
Before assignment: 8 evaluable patients were planned to be enrolled. These 8 evaluable patients were assigned to take 20 mg \[14C\]-AZD9291 oral solution (free base equivalent) containing a nominal dose 1 μCi (0.037 MBq) activity as a single administration on Day 1
Overall Study
| Stage | AZD9291 20mg |
|---|---|
| STARTED | 8 |
| COMPLETED | 8 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Pharmacokinetic population - all subjects who received 1 dose of \[14C\]-AZD9291 and had at least 1 postdose PK measurement without important protocol deviations/violations or events thought to significantly affect the PK of the investigational product
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 | AZD9291 20mg |
|---|---|---|
| Participants analysed | 8 | |
| Age, Continuous | Years | 42 (SD 13) |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 8 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 8 |
| 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 | 3 |
| Race (NIH/OMB): White | Participants | 5 |
| 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 Percentage of Radioactive Dose of [14C] Radiolabelled AZD9291 Recovered in Urine, Faeces, and in Total., measured over Blood samples (hrs) - 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 18, 24, 36, 48, every 24 to 168, 336, 504, 648, 816, 904 and 1992. Urine (h): 0-4, 4-8, 8-12, 12-24, every 24 hrs to 504, 648-72, 816-40, 984-1008 and 1992-2016. Faeces: 0-24h and then as per urine..
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | Percentage radioactive dose recovered |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is the percentage of the radioactive dose of [14C]-labelled AZD9291 recovered in urine, faeces, and in total, with assessment described up to Day 85. It is a continuous measure of dose recovery. The registry record designates the mean and standard deviation as the summary statistics, and describes the relevant population as participants who received one dose and had at least one postdose pharmacokinetic measurement, without important protocol deviations or events thought to significantly affect pharmacokinetics.
In general, a mass-balance assessment tracks where administered radiolabel is recovered in collected samples. Measuring radioactivity in urine and faeces can describe the recovery of radiolabel through those collection routes. The total is intended to represent recovery across the specified routes; it should not be read as a complete accounting unless collection and measurement are sufficiently complete. The registry record does not report collection-completeness details or numerical recovery results.
The stated endpoint is distinct from the concentration-based pharmacokinetic outcomes. Those listed outcomes include concentration-time summaries, peak concentration and its timing, and apparent clearance and distribution measures. The primary endpoint’s mean and standard deviation are specified as the intended descriptive summaries, but their numerical values are not reported in the registry record. Consequently, the endpoint’s observed magnitude and its precision cannot be assessed from the posted results.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a single-group study of 20mg oral [14C]-AZD9291 in healthy male volunteers, with the main outcome defined as the percentage of radioactive dose recovered in urine, faeces, and in total up to Day 85. The record also specifies that the mean and standard deviation were the intended summaries. It does not report the numerical mean, standard deviation, or other primary endpoint results. No conclusion about the amount recovered, the pattern of recovery, or the precision of the estimate can therefore be drawn from the posted results.
This absence matters because a definition of an endpoint is not an outcome result. The sampling schedule and endpoint description explain what investigators intended to measure, but do not show the values observed. The registry lists concentration-based secondary outcomes, including several area-under-the-curve measures, maximum concentration, timing measures, apparent oral clearance, and apparent volume of distribution. Numerical results for these measures are not reported in the registry record either. The record lists 20 outcome measures and 0 analyses, but the count of listed measures does not supply results for them.
The design also limits the kind of inference available. There was no randomized comparator and no masking. The study can be described as a single-group pharmacokinetic and mass-balance assessment, not as a randomized treatment comparison. Without a control group, variation over time or differences from another population cannot be separated through a concurrent comparison. The study’s stated population was healthy male volunteers; results from that population, even if available, would not by themselves establish what would be observed in other populations or clinical settings.
The registry reports enrollment of 8, but enrollment is not necessarily the number contributing to every endpoint. The primary outcome population is defined using receipt of dose, at least one postdose pharmacokinetic measurement, and the absence of important deviations or events thought to affect pharmacokinetics. The number meeting that definition is not reported in the registry record. Nor are endpoint-specific denominators, missing samples, or exclusions described. Without those details and the numerical outcome summaries, precision and potential influence of incomplete observations cannot be judged.
Safety figures are also limited to the entries in the record: 0 serious adverse events among 8 at risk and 7 with other adverse events among 8 at risk. These counts are descriptive for this study group. They do not answer the primary mass-balance question, and the record does not report a comparator safety rate. The deaths field is not reported in the registry record.
Any planning illustration based on an observed effect would require an effect estimate and assumptions that are not quantified in the registry record. The stated assumptions identify a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a fraction of an observed effect. Those are planning assumptions, not a description of the single-group study design. No numerical planning estimates are reported in the registry record, and such illustrations would not represent observed power.
9. Statistical Methods Explained
The study’s primary endpoint is continuous: it expresses recovered radioactive dose as a percentage. The registry record identifies the mean and standard deviation as the planned descriptive summaries. In general, a mean describes the centre of a set of measurements, while a standard deviation describes how spread out individual measurements are around that centre. They answer different questions: the mean describes the average observed recovery, and the standard deviation describes variation among observations. Neither numerical summary is reported for this endpoint in the registry record.
What does a confidence interval communicate?
In general, a confidence interval gives a range of values compatible with a statistical estimation procedure and the observed data, under the method’s assumptions. A narrower interval indicates greater precision for the estimate, while a wider interval indicates more uncertainty. The interval is not a guarantee that the true value lies within its limits, nor does it describe the range containing every participant’s measurement. No confidence interval for the primary outcome is reported in the registry record, so its precision cannot be assessed from the posted results.
Why does the single-group design matter?
The study is listed as non-randomized and single-group, with no masking. In general, random allocation can help balance known and unknown participant characteristics between comparison groups. A single-group study has no such concurrent randomized contrast. Its endpoint can describe measurements in the participants assessed, but it does not estimate a randomized between-group difference. In this study, the principal question concerns dose recovery after administration, rather than a comparative effect against a control arm.
Open-label status means that masking was not used, according to the record. For objective laboratory measures, the implications of awareness can differ from those for subjective assessments, but the registry record does not describe laboratory procedures, blinding of assay personnel, or safeguards against measurement bias. It would therefore be inappropriate to assume either that masking was needed or that its absence had no effect.
How should repeated measurements be understood?
The registry describes collection at multiple times for blood, urine, and faeces. In general, repeated observations from one participant are related, not independent observations. Pharmacokinetic analyses often use the timing and sequence of concentrations to characterize exposure and related parameters. AUC measures summarize concentration over a specified interval; Cmax represents a maximum concentration, and Tmax the time at which it occurs. Tlag refers to a lag before quantifiable concentrations are observed. Apparent oral clearance and apparent volume of distribution are derived pharmacokinetic quantities. The record lists these outcome types but does not provide their numerical results or enough analysis detail to reconstruct their estimation.
The collection schedule is extensive, but a planned schedule does not establish that all samples were collected, measurable, or included in analyses. For a mass-balance endpoint, recovery depends on the observations and the collection intervals included. In general, incomplete collection can make apparent recovery lower than total recovery, while measurement and timing issues can add uncertainty. The record does not report collection completeness, assay performance, handling of samples below quantification limits, or the extent of missing observations.
What can be inferred from the stated summaries?
The record names the mean and standard deviation but does not report their values. Without those values, readers cannot assess the observed average, between-participant variability, or whether estimates are dominated by unusually high or low observations. A small study group can make estimates sensitive to individual observations in general, but the registry record does not supply participant-level data or summaries that would show whether that occurred. No numerical precision measure is reported.
The record lists 0 analyses. It does not describe an inferential model, covariate adjustment, transformation, treatment of outliers, or formal hypothesis test for the primary endpoint. Those details are not reported in the registry record. The absence of reported analysis details should not be filled in by assuming a conventional method; only the endpoint type and planned mean and standard deviation are explicit.
The stated sample-size assumptions refer to a planning illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The effect to detect is described as a stated fraction of the observed effect. Those assumptions do not describe this single-group, non-randomized trial’s actual design, and the numerical planning results are not reported in the registry record. A sample-size calculation is a prospective planning exercise, not observed power. In general, observed power calculated after seeing an estimate does not replace an interval that communicates estimation uncertainty.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 20 outcome measures, including the primary mass-balance endpoint and several pharmacokinetic measures. It also lists 0 analyses. The record does not report a multiplicity adjustment, a hierarchy for secondary outcomes, or a plan for controlling the chance of false-positive findings across multiple statistical tests. Those features are not reported in the registry record. Because numerical outcome results and inferential tests are not reported, the impact of multiplicity on any claimed findings cannot be evaluated.
In general, testing multiple outcomes can increase the chance that at least one result appears statistically notable by chance alone. A prespecified hierarchy or adjustment can address some multiplicity concerns, but neither should be assumed when not described. This study’s listed outcomes include related measures calculated over different time intervals and for different analytes, so interpretation would depend on the actual analysis plan and the role assigned to each outcome. The registry does not report those details.
Missing-data information is also limited. The primary population definition requires one dose, at least one postdose pharmacokinetic measurement, and no important protocol deviations or events thought to significantly affect pharmacokinetics. The record does not state how many participants met that definition, how many contributed to each endpoint, whether any samples were missing, or how missing or unquantifiable measurements were handled. It does not report exclusions or sensitivity analyses for incomplete data.
In general, missing samples can affect estimates when missingness is related to the values that would have been observed or to the collection process. For a mass-balance outcome, completeness of urine and faeces collections is particularly relevant to interpreting recovery. The record describes collection intervals but does not report completeness. The degree or direction of any resulting bias cannot be inferred from the registry record.
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 |
|---|---|---|---|
| AZD9291 20mg | 0 | 8 | 0.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 1% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| AZD9291 20mg | 7 of 8 |
Most frequent non-serious adverse event terms
| Event term | Organ system | AZD9291 20mg |
|---|---|---|
| Headache | Nervous system disorders | 2 of 8 |
| Asthenopia | Eye disorders | 1 of 8 |
| Visual impairment | Eye disorders | 1 of 8 |
| Abdominal pain upper | Gastrointestinal disorders | 1 of 8 |
| Constipation | Gastrointestinal disorders | 1 of 8 |
| Nausea | Gastrointestinal disorders | 1 of 8 |
| Non-cardiac chest pain | General disorders | 1 of 8 |
| Otitis media | Infections and infestations | 1 of 8 |
| Laceration | Injury, poisoning and procedural complications | 1 of 8 |
| Back pain | Musculoskeletal and connective tissue disorders | 1 of 8 |
The registry lists 0 serious adverse events among 8 participants at risk in the AZD9291 20mg group. It lists 7 with other adverse events among 8 at risk. These are descriptive counts for the study group, not a comparison with another group. The record lists no serious adverse event terms among its top serious terms.
The distinction between the serious-event count and the other adverse-event count is important: the 7 other adverse events should not be described as serious events. The registry record does not provide event-level details for those other events in the registry record, so their nature, timing, severity, and relationship to treatment cannot be characterized. It also does not report a comparison rate or enough information to estimate a comparative safety effect.
The deaths field is not reported in the registry record. That should not be converted into a claim that no deaths occurred. More generally, a small descriptive safety record cannot establish the absence of uncommon harms. The figures should be read only as the entries recorded for this group, with the registry’s reported at-risk denominator.
12. What the Result Does, and Does Not, Mean
This study was designed to characterize pharmacokinetics and mass balance after a single oral dose of [14C]-AZD9291 in healthy male volunteers. The primary endpoint specifies recovery of radioactive dose in urine, faeces, and in total up to Day 85. The registry record also identifies planned pharmacokinetic measures for AZD9291, its metabolites, and whole-blood and plasma radioactivity. These details describe the questions and measurements of interest.
However, the registry record does not report numerical results for the primary endpoint. It identifies mean and standard deviation as summary statistics but gives neither value. The record also does not report numerical results for the listed secondary pharmacokinetic outcomes. Accordingly, the posted results do not establish how much radioactivity was recovered, how variable recovery was, or what the measured pharmacokinetic values were. An endpoint definition and sampling schedule cannot substitute for those observed results.
The design is not a randomized comparison. It cannot, on its own, provide a like-for-like estimate of a difference between AZD9291 and a control treatment. Its setting and population are also specific: healthy male volunteers in a phase I, single-centre study. The record does not establish that findings would generalize to other populations or settings.
The safety entries report 0 serious adverse events and 7 other adverse events among 8 at risk. These descriptive counts do not determine whether an event was caused by treatment, and the registry record does not report a comparative safety assessment. The deaths field is not reported in the registry record.
Overall, the record supports a description of the study design, its intended measurements, and limited safety counts. It does not support a quantitative conclusion about mass-balance recovery or pharmacokinetic results because the numerical endpoint results are not reported in the registry record. Nor should planning assumptions be mistaken for observed findings or evidence of observed power.
13. Why This Trial Matters Statistically
Mass-balance studies address a different statistical question from trials that compare clinical outcomes between treatment groups. Their focus is the measured disposition and recovery of a labelled dose across collection routes. This study’s primary endpoint was defined as the percentage of radioactive dose recovered in urine, faeces, and in total up to Day 85. The registry’s description makes clear that the intended evidence depends on timed collections and measurement of radioactivity, not on a randomized comparison of clinical benefit.
That distinction matters when interpreting a study record. A detailed schedule and named endpoint may suggest a comprehensive assessment, but the actual numerical results are needed to evaluate recovery and uncertainty. Here, the record identifies mean and standard deviation as planned summaries but does not report their values. It also lists multiple pharmacokinetic outcomes without numerical results. Statistical interpretation must distinguish the measurement plan from evidence about what was observed.
The record also illustrates why analysis populations and collection completeness matter. It defines a pharmacokinetic population based on dose receipt, postdose measurement, and the absence of important deviations or events thought to affect pharmacokinetics. But it does not report how many participants met that definition or the completeness of samples. For timed pharmacokinetic data and cumulative recovery, those details help readers understand which observations inform an estimate and how missing measurements may affect it.
Finally, the study’s single-group, non-randomized design and healthy male volunteer population set boundaries on interpretation. The listed safety counts are descriptive, and the study does not provide a concurrent control comparison. These features do not make the measurements uninformative in principle; they define the questions the design can address. The missing numerical results prevent quantitative assessment of the central endpoint in the registry record.
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 main limitation for interpreting the posted results is that numerical results for the primary mass-balance endpoint are not reported in the registry record. Although the endpoint is defined and mean and standard deviation are named as summary statistics, their values are absent. Numerical results for the listed pharmacokinetic outcomes are also not reported. This prevents assessment of observed recovery, variability, and estimation precision.
The study was non-randomized, open-label, single-centre, and single-group, with no concurrent comparator. These design characteristics mean it does not provide a randomized between-group contrast. The registry record does not describe masking of laboratory personnel or other measurement safeguards. It also does not report the number contributing to each outcome, the number meeting the defined pharmacokinetic population criteria, or how missing and unquantifiable samples were handled.
For the primary outcome, the record provides a collection schedule extending to Day 85 but does not report whether collections were complete. That matters because incomplete urine or faeces collection could affect apparent dose recovery. The registry does not report assay details or completeness checks. It also does not report the statistical analysis plan, endpoint-specific methods, inferential procedures, or any multiplicity approach.
The safety information is limited to aggregate counts, and the deaths field is not reported in the registry record. The listed population comprises healthy male volunteers, so the record does not establish generalizability to other populations. These limitations concern what can be concluded from the registry entry; they do not establish that unreported procedures or analyses were not performed.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study Evaluating the Pharmacokinetic and Mass Balance of Single Dose [14C] AZD9291 in Volunteers — NCT02096679.
- Registry record last updated 2015-10-21; status last verified 2015-10.
- Results first posted 2015-07-30.
- 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
NCT02096679 is a completed phase I, single-centre, non-randomized, open-label, single-group pharmacokinetic and mass-balance study sponsored by AstraZeneca. It enrolled 8 healthy male volunteers and assessed a single oral dose of 20mg [14C]-AZD9291 as a solution, with nominal 1 μCi activity. The recorded start was 2014-05 and primary completion was 2014-08.
The primary endpoint was the percentage of radioactive dose recovered in urine, faeces, and in total up to Day 85. The registry identifies mean and standard deviation as the intended summaries, but numerical results are not reported in the registry record. The record lists 20 outcome measures and 0 analyses. Safety entries include 0 serious adverse events and 7 other adverse events among 8 at risk; deaths are not reported in the registry record.