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 Assess Safety & Efficacy of Selumetinib When Given in Combination With Standard First Line Treatment for Advanced Non-small Cell Lung Cancer (NCT01809210), a na phase 1 study. The primary outcome is Dose Limiting Toxicity (DLT) Events in Chemotherapy in Combination With Selumetinib.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01809210 |
| Phase | Phase 1 |
| Condition | Locally Advanced or Metastatic NSCL Cancer Stage IIIB IV |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 55 |
| Arms in the primary outcome | n/a |
| Lead sponsor | AstraZeneca |
| Results posted | Yes |
2. Clinical Question
Population
Patients with locally advanced or metastatic non-small cell lung cancer, recorded as stage IIIB IV. The study description identifies the treatment setting as advanced or metastatic disease and the primary safety population as patients who received at least 1 dose of selumetinib.
Intervention
Oral selumetinib given with first-line chemotherapy. Cohorts used gemcitabine with cisplatin or carboplatin, or pemetrexed with cisplatin or carboplatin, at different selumetinib dose levels.
Comparator
There was no concurrent control group identified. The registry lists a single-group model and allocation as not applicable. The cohorts are not a randomized, like-for-like comparison.
Primary question
How tolerable is selumetinib when combined with the selected chemotherapy regimens, as assessed by dose-limiting toxicity during the initial treatment period? The study also planned to assess preliminary activity, but a primary endpoint result is not reported in the registry record.
The clinical question is primarily about finding and assessing combination doses, rather than estimating a treatment effect against a control. The registry describes a Phase I, open-label, multicentre study with dose finding and optional cohort expansion. Its stated purpose was to investigate an optimal selumetinib dose in combination with selected standard first-line double-platinum chemotherapy regimens, with tolerability as an initial consideration and preliminary evidence of activity also to be assessed.
That purpose matters when interpreting the cohort information. The chemotherapy backbone and selumetinib dose varied across cohorts, and there is no randomized allocation to make those cohorts comparable as treatment groups. Consequently, their outcomes cannot be read as a controlled estimate of the effect of one combination versus another. The record lists several secondary efficacy and pharmacokinetic outcomes, but does not report their results.
3. Trial Design
This was a Phase I, open-label, multicentre, single-group study sponsored by AstraZeneca. The registry lists the study as completed and records enrollment of 55. The recorded start date is 2013-04-04 and the primary completion date is 2016-01-04. The trial title and description identify patients with advanced or metastatic non-small cell lung cancer receiving selumetinib alongside first-line chemotherapy.
The study used multiple cohorts combining different selumetinib dose levels with chemotherapy regimens. The listed backbones included gemcitabine with cisplatin or carboplatin and pemetrexed with cisplatin or carboplatin. The design description characterizes the work as dose finding with optional cohort expansion. It also states that all patients would be assessed for anticancer efficacy of the combination.
Allocation is listed as not applicable, masking as none, and the model as single group. Thus, this is not a blinded, randomized trial with a control arm. Cohort differences in dose and chemotherapy regimen mean that between-cohort contrasts would not isolate the effect of a single treatment difference. The registry record does not report a protocol for comparing cohorts or a statistical plan for such comparisons.
The primary outcome is dose-limiting toxicity during the early treatment period. The safety analysis set is defined as patients who received at least 1 dose of selumetinib. The registry also lists secondary outcomes covering objective response, changes in target-lesion size, and pharmacokinetic measures. Results for those secondary outcomes are not reported in the registry record. The record also does not report details of the dose-escalation decision rules or the criteria used to select a dose for expansion.
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: Data cut off (DCO) was 7 Jan 16. DCO was defined as the earliest of 12 (±1) weeks after last patient started, or 28 days after final patient discontinued, selumetinib or chemotherapy doublet regimen.
Only the first 6 of 7 groups are shown.
Overall Study
| Stage | Cohort 1 sel50, Gem, Cis | Cohort 2 sel50, Gem, Carb | Cohort 3 sel75, Gem, Cis | Cohort 4 sel150, Pem, Carb | Cohort 5 sel75, Pem, Carb | Cohort 6 sel75, Pem, Cis |
|---|---|---|---|---|---|---|
| STARTED | 3 | 9 | 7 | 3 | 6 | 15 |
| COMPLETED | 2 | 8 | 4 | 2 | 4 | 12 |
| NOT COMPLETED | 1 | 1 | 3 | 1 | 2 | 3 |
Reasons for not completing this period:
| Reason | Cohort 1 sel50, Gem, Cis | Cohort 2 sel50, Gem, Carb | Cohort 3 sel75, Gem, Cis | Cohort 4 sel150, Pem, Carb | Cohort 5 sel75, Pem, Carb | Cohort 6 sel75, Pem, Cis |
|---|---|---|---|---|---|---|
| Withdrawal by Subject | 0 | 0 | 1 | 1 | 1 | 0 |
| Death | 1 | 1 | 2 | 0 | 1 | 3 |
Analysis population for the primary outcome, as defined in the registry: Safety analysis set - all patients who received at least 1 dose of selumetinib.
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.
Only the first 6 of 8 groups are shown.
| Characteristic | Unit | Cohort 1 sel50, Gem, Cis | Cohort 2 sel50, Gem, Carb | Cohort 3 sel75, Gem, Cis | Cohort 4 sel150, Pem, Carb | Cohort 5 sel75, Pem, Carb | Cohort 6 sel75, Pem, Cis |
|---|---|---|---|---|---|---|---|
| Participants analysed | 3 | 9 | 7 | 3 | 6 | 15 | |
| Age, Continuous | Years | 58.7 (SD 3.79) | 69.2 (SD 3.53) | 55.1 (SD 8.65) | 64.7 (SD 7.23) | 59.5 (SD 5.28) | 61.8 (SD 5.86) |
| Sex: Female, Male: Female | Participants | 1 | 5 | 3 | 1 | 4 | 5 |
| Sex: Female, Male: Male | Participants | 2 | 4 | 4 | 2 | 2 | 10 |
| Race/Ethnicity, Customized: Black Or African American | Participants | 1 | 0 | 0 | 0 | 1 | 1 |
| Race/Ethnicity, Customized: Other | Participants | 0 | 0 | 0 | 0 | 0 | 1 |
| Race/Ethnicity, Customized: White | Participants | 2 | 9 | 7 | 3 | 5 | 13 |
6. Primary Endpoint
The registry lists the primary outcome as Dose Limiting Toxicity (DLT) Events in Chemotherapy in Combination With Selumetinib, measured over The first dose on Cycle 1 Day 1 up to the time before dosing on Cycle 2 Day 1, assessed up to 3 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants with dose-limiting toxicity events during chemotherapy given in combination with selumetinib. The specified assessment window begins with the first dose on Cycle 1 Day 1 and continues to the time before dosing on Cycle 2 Day 1, assessed up to 3 weeks. A qualifying toxicity is described as one considered related to the combination, not attributable to the disease or disease-related processes, and occurring within that window at a dose-limiting level.
Counting participants with an event is a direct way to describe how often a specified safety event occurred in the defined population and period. In a dose-finding study, early tolerability information is relevant to choosing which doses and combinations merit further assessment. The endpoint is not a measure of tumor response or survival; those are distinct outcomes.
The registry record does not report the number of participants who experienced the primary endpoint, nor does it provide an estimate of uncertainty for that outcome. Therefore, the endpoint definition and assessment window can be described, but the primary safety result cannot be interpreted from the posted record. The defined safety analysis set includes all patients who received at least 1 dose of selumetinib; further details about handling participants with incomplete endpoint observation are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record sets out a dose-finding safety question for selumetinib combined with first-line chemotherapy in advanced non-small cell lung cancer. It does not provide a numerical result for the primary endpoint of dose-limiting toxicity. The primary outcome entry supplies the event definition, population, and observation window, but not an event count or an estimate of uncertainty. No conclusion about the frequency of the primary endpoint can therefore be drawn from this record alone.
The registry does report serious adverse event counts by cohort. These are descriptive safety data, not the primary endpoint result: the event definitions and observation periods need not be identical. Counts are small within several cohorts, and the regimens and selumetinib dose levels vary. Such cohort-level figures can describe what was recorded, but they do not support a randomized comparison or a reliable ranking of regimens. The record gives no common control group against which to interpret them.
Several secondary outcomes are named, including best objective response, objective response rate, target-lesion changes, and pharmacokinetic measures. Their outcome results are not reported in the registry record. The study description says that preliminary activity was to be assessed, but a stated intention to measure an outcome is not itself evidence of its result. No response rate or change in lesion size should be inferred from the outcome titles.
No confidence interval, p-value, or effect estimate for a comparative treatment contrast is reported in the registry record. A confidence interval, in general, expresses the statistical uncertainty around an estimate; without a reported estimate and interval, the precision of a primary endpoint result cannot be assessed. Likewise, a significance test would not create a meaningful control comparison where the design has no randomized control arm.
The registry identifies enrollment of 55, but the primary outcome population is defined by receipt of at least 1 dose and the number of participants contributing to that endpoint is not reported. Enrollment should not be assumed to equal the number analyzed for every outcome. The safest reading is therefore that the record describes the design and selected safety counts, while leaving the primary endpoint result, efficacy results, and their precision unreported.
9. Statistical Methods Explained
The main statistical task in a dose-finding study is to describe tolerability at the doses and combinations that were actually administered. The primary endpoint is a participant count for dose-limiting toxicity over a defined early-treatment window. In general, a count is interpretable only alongside its population, event definition, and time period. Those elements are described in the registry, but the primary endpoint count is not reported in the registry record.
What does a participant count tell the reader?
A count of participants with an event is an absolute description of how many people in a defined analysis set experienced that event. It is not automatically a rate that can be compared across studies or cohorts. The number at risk, follow-up period, eligibility for the analysis set, and completeness of outcome observation all affect interpretation. The registry defines the primary safety set as patients who received at least 1 dose of selumetinib and specifies an assessment period beginning on Cycle 1 Day 1 and ending before Cycle 2 Day 1, assessed up to 3 weeks. It does not report the primary count or the number contributing complete observation.
Why does the design affect comparisons?
The study is listed as single group, with allocation not applicable and no masking. It has no randomized concurrent control group. The cohorts differ in selumetinib dose and chemotherapy regimen, so a difference in observed counts could reflect several features at once, including the regimen, dose, or cohort composition. General statistical principle: random allocation helps balance both known and unknown participant characteristics across treatment groups. Without it, an observed cohort contrast is descriptive and cannot be treated as a causal estimate of the effect of changing one treatment component.
Over-dispersion is a general issue when counts vary more across groups than a simple statistical model expects. It can lead to uncertainty estimates that are too narrow if ignored. The posted primary outcome is a participant count, but the registry does not report a model for the outcome or enough primary endpoint results to assess variation across cohorts. No model choice should be inferred from the endpoint title alone.
How should uncertainty be assessed?
In general, an estimate is more informative when accompanied by an interval that communicates its precision. A confidence interval gives a range of values compatible with the data under the model and assumptions used; it does not remove limitations in the design or guarantee that the model is correct. No confidence interval for the primary endpoint is reported in the registry record. The record also does not report a comparative estimate or p-value for the primary endpoint. As a result, readers cannot assess the precision of a primary safety effect estimate from the record.
The registry lists sample-size planning assumptions for an illustration based on an observed effect, including a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. These are planning assumptions, not a description of the single-group study's observed evidence. Any sample-size values based on such assumptions are illustrations for planning and must not be interpreted as observed power or as proof that the study could detect a particular effect. The registry record does not report the trial's own sample-size justification.
What is the role of the other outcomes?
The secondary outcomes include best objective response, objective response rate, target-lesion changes, and pharmacokinetic measures such as AUC (0-tau), Cmax,ss, Tmax,ss, and CL/F. These outcomes answer different questions. Tumor response measures describe disease activity, while pharmacokinetic measures characterize drug exposure or disposition. Listing an outcome does not establish that a result was available or analyzed. The record does not report results for these secondary outcomes, and it lists no outcome analyses.
General statistical principle: a study should distinguish descriptive summaries from inferential comparisons. Descriptive statistics present the observed data; inferential methods quantify uncertainty or test a specified hypothesis under assumptions. In this registry record, the reported cohort safety counts can be read descriptively. A treatment comparison is not justified by the design, and the primary endpoint lacks a posted count and uncertainty estimate. No unreported method or analysis can be reconstructed from the study title or outcome list.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists several secondary outcomes spanning tumor response, target-lesion change, and pharmacokinetics. When multiple outcomes are assessed, the chance of at least one apparently notable result can increase if many statistical tests are conducted. General statistical practice may address this through a prespecified hierarchy, adjustment, or a clearly exploratory interpretation. A multiplicity strategy is not reported in the registry record, and secondary outcome results are not reported.
The record lists no outcome analyses. It does not report whether interim analyses were planned or conducted, nor does it report decision thresholds for dose escalation or expansion. These details should not be inferred from the Phase I designation or the study description.
The primary safety population is described as all patients who received at least 1 dose of selumetinib. The number analyzed for the primary endpoint and the handling of missing or incomplete observations are not reported in the registry record. There is no reported imputation method, missing-data sensitivity analysis, or explanation of exclusions from the safety set. The listed enrollment of 55 therefore should not be assumed to be the denominator for each endpoint.
The registry record also does not report an efficacy analysis population or a statistical analysis plan for the listed secondary outcomes. These omissions limit assessment of whether outcome definitions, analysis choices, and missing-data handling were prespecified or applied consistently.
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 |
|---|---|---|---|
| Cohort 1 sel50, Gem, Cis | 1 | 3 | 33.3% |
| Cohort 2 sel50, Gem, Carb | 6 | 9 | 66.7% |
| Cohort 3 sel75, Gem, Cis | 3 | 7 | 42.9% |
| Cohort 4 sel150, Pem, Carb | 2 | 3 | 66.7% |
| Cohort 5 sel75, Pem, Carb | 3 | 6 | 50.0% |
| Cohort 6 sel75, Pem, Cis | 9 | 15 | 60.0% |
| Cohort 7 sel100, Pem, Carb | 9 | 12 | 75.0% |
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 |
|---|---|
| Cohort 1 sel50, Gem, Cis | 3 of 3 |
| Cohort 2 sel50, Gem, Carb | 9 of 9 |
| Cohort 3 sel75, Gem, Cis | 7 of 7 |
| Cohort 4 sel150, Pem, Carb | 3 of 3 |
| Cohort 5 sel75, Pem, Carb | 6 of 6 |
| Cohort 6 sel75, Pem, Cis | 15 of 15 |
Most frequent serious adverse event terms
| Event term | Organ system | Cohort 1 sel50, Gem, Cis | Cohort 2 sel50, Gem, Carb | Cohort 3 sel75, Gem, Cis | Cohort 4 sel150, Pem, Carb | Cohort 5 sel75, Pem, Carb | Cohort 6 sel75, Pem, Cis |
|---|---|---|---|---|---|---|---|
| Thrombocytopenia | Blood and lymphatic system disorders | 0 of 3 | 4 of 9 | 0 of 7 | 0 of 3 | 0 of 6 | 0 of 15 |
| Nausea | Gastrointestinal disorders | 0 of 3 | 0 of 9 | 2 of 7 | 0 of 3 | 0 of 6 | 1 of 15 |
| Neutropenia | Blood and lymphatic system disorders | 0 of 3 | 1 of 9 | 0 of 7 | 0 of 3 | 0 of 6 | 1 of 15 |
| Myocardial infarction | Cardiac disorders | 0 of 3 | 1 of 9 | 0 of 7 | 0 of 3 | 0 of 6 | 1 of 15 |
| Large intestine perforation | Gastrointestinal disorders | 1 of 3 | 0 of 9 | 1 of 7 | 0 of 3 | 0 of 6 | 0 of 15 |
| Pyrexia | General disorders | 0 of 3 | 1 of 9 | 0 of 7 | 0 of 3 | 1 of 6 | 0 of 15 |
| Lower respiratory tract infection | Infections and infestations | 0 of 3 | 1 of 9 | 0 of 7 | 0 of 3 | 0 of 6 | 1 of 15 |
| Pneumonia | Infections and infestations | 0 of 3 | 1 of 9 | 1 of 7 | 0 of 3 | 0 of 6 | 0 of 15 |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | 0 of 3 | 0 of 9 | 1 of 7 | 0 of 3 | 0 of 6 | 1 of 15 |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | 1 of 3 | 0 of 9 | 1 of 7 | 0 of 3 | 0 of 6 | 0 of 15 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Cohort 1 sel50, Gem, Cis | Cohort 2 sel50, Gem, Carb | Cohort 3 sel75, Gem, Cis | Cohort 4 sel150, Pem, Carb | Cohort 5 sel75, Pem, Carb | Cohort 6 sel75, Pem, Cis |
|---|---|---|---|---|---|---|---|
| Nausea | Gastrointestinal disorders | 3 of 3 | 5 of 9 | 5 of 7 | 3 of 3 | 5 of 6 | 8 of 15 |
| Diarrhoea | Gastrointestinal disorders | 2 of 3 | 5 of 9 | 4 of 7 | 1 of 3 | 3 of 6 | 9 of 15 |
| Fatigue | General disorders | 2 of 3 | 6 of 9 | 3 of 7 | 1 of 3 | 2 of 6 | 9 of 15 |
| Vomiting | Gastrointestinal disorders | 3 of 3 | 4 of 9 | 4 of 7 | 1 of 3 | 4 of 6 | 6 of 15 |
| Constipation | Gastrointestinal disorders | 1 of 3 | 5 of 9 | 4 of 7 | 2 of 3 | 2 of 6 | 6 of 15 |
| Rash | Skin and subcutaneous tissue disorders | 0 of 3 | 3 of 9 | 5 of 7 | 2 of 3 | 1 of 6 | 8 of 15 |
| Decreased appetite | Metabolism and nutrition disorders | 1 of 3 | 4 of 9 | 2 of 7 | 0 of 3 | 3 of 6 | 4 of 15 |
| Lethargy | Nervous system disorders | 0 of 3 | 2 of 9 | 3 of 7 | 1 of 3 | 2 of 6 | 5 of 15 |
| Periorbital oedema | Eye disorders | 0 of 3 | 3 of 9 | 2 of 7 | 2 of 3 | 1 of 6 | 2 of 15 |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | 1 of 3 | 4 of 9 | 0 of 7 | 1 of 3 | 3 of 6 | 1 of 15 |
Serious events with the highest arm-level rate: thrombocytopenia (44.4%), large intestine perforation (33.3%), musculoskeletal chest pain (33.3%), pain in extremity (33.3%), urinary retention (33.3%).
The registry reports serious adverse event counts for seven named cohorts. The entries list 1 affected among 3 at risk in Cohort 1, 6 among 9 in Cohort 2, 3 among 7 in Cohort 3, 2 among 3 in Cohort 4, 3 among 6 in Cohort 5, 9 among 15 in Cohort 6, and 9 among 12 in Cohort 7. These are cohort-level descriptions, not randomized comparisons. The cohorts used different selumetinib dose levels and chemotherapy combinations, and the record does not provide a common control group.
The registry also lists serious-event terms with percentages: Thrombocytopenia, 44.4%; Large intestine perforation, 33.3%; Musculoskeletal chest pain, 33.3%; Pain in extremity, 33.3%; Urinary retention, 33.3%; and Pulmonary embolism, 33.3%. The record does not report the denominators or further details needed to interpret those percentages, such as the cohort distribution for each term. They should not be treated as estimates of comparative risk.
Death counts are not reported in the registry record; a blank or unavailable count must not be read as zero. Serious adverse events are distinct from the primary endpoint of dose-limiting toxicity, which has its own definition and assessment window. The posted serious-event figures describe reported safety information, but do not establish a causal comparison between regimens.
12. What the Result Does, and Does Not, Mean
This record is useful for understanding the intended clinical and statistical role of the study: a Phase I investigation of selumetinib with first-line chemotherapy, focused initially on tolerability and dose finding. It identifies the primary safety outcome and its assessment window, describes the treatment combinations, and reports serious adverse event counts for the listed cohorts. Those features help readers understand what the study set out to examine.
The record does not report the primary dose-limiting toxicity count or a corresponding estimate of precision. It also does not report results for its listed response, target-lesion, or pharmacokinetic outcomes. The study description's intention to evaluate preliminary activity cannot substitute for observed outcome results. Accordingly, the registry record does not establish a treatment effect on tumor response or any other efficacy outcome.
The single-group, open-label design has no randomized control arm. Differences among the cohorts cannot be interpreted as unbiased comparisons of chemotherapy combinations or selumetinib doses, because the regimens and doses vary and cohort assignment was not randomized. The safety counts can be read as descriptions of what the registry records, but they do not by themselves show that one regimen is safer or more effective than another.
In general, a study's result is most informative when its endpoint definition, analyzed population, observed estimate, and uncertainty are all available. Here, the endpoint definition is present, but its numerical result and uncertainty are not. The registry record also does not report the analysis plan, missing-data procedures, or rationale for the planned enrollment. These gaps prevent a full assessment of the statistical evidence and its limitations. The posted information therefore supports a description of the design and selected safety data, not a definitive efficacy or comparative safety conclusion.
13. Why This Trial Matters Statistically
Phase I combination studies address an important statistical challenge: several treatment components may vary at the same time while the study seeks information about tolerability. This record includes different selumetinib dose levels and multiple chemotherapy backbones. The design context is essential because a cohort count is not an isolated estimate of the effect of a single drug or dose.
The primary endpoint is also tied to a specific early-treatment window. Defining the population, toxicity criteria, and observation period makes the intended safety question clearer and highlights what must be reported to interpret it. In this registry record, those definitions are available, but the primary endpoint count is not. The contrast between a detailed endpoint definition and an absent result illustrates why outcome reporting needs both methods and observed data.
The study's single-group structure also makes a useful statistical distinction visible: describing events is not the same as estimating a comparative treatment effect. The cohort safety counts convey information about recorded events in each cohort, but differences cannot be attributed to regimen or dose without a suitable comparison and careful accounting for cohort differences. General statistical reasoning can clarify that limitation without treating the counts as a ranking.
Finally, the record lists both efficacy and pharmacokinetic outcomes alongside safety outcomes. These measures address different questions and should not be conflated. The absence of their results means that planned measurement cannot be mistaken for evidence. For clinical researchers and biostatisticians, the record underscores the value of transparent endpoint results, analysis populations, uncertainty estimates, and analysis plans when interpreting early-phase combination trials.
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 most important limitation of the posted record is that it does not report the primary outcome result. The endpoint definition and timeframe are present, but the dose-limiting toxicity count and its precision are not. Without those results, the central safety question cannot be answered from the registry record.
The study has no randomized control group and uses multiple cohorts with differing selumetinib doses and chemotherapy combinations. This prevents a like-for-like comparison and makes any informal contrast between cohorts vulnerable to differences in regimen and participant composition. Open-label conduct is recorded, but details about outcome assessment procedures are not reported in the registry record.
Although the registry lists enrollment of 55, it does not report how many participants contributed to each endpoint analysis. The safety set is defined by receipt of at least 1 dose, but outcome-specific denominators, missing-data handling, and exclusions are not reported. The serious adverse event entries therefore cannot be assumed to describe a common follow-up period or identical analysis populations across outcomes.
The listed secondary outcomes have no reported results, and the registry does not report an analysis plan, multiplicity approach, interim-analysis plan, or the trial's sample-size justification. The serious adverse event term percentages are listed without denominator details. Death counts are not reported in the registry record. These limitations restrict interpretation of both the safety summaries and the planned efficacy assessment.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Assess Safety & Efficacy of Selumetinib When Given in Combination With Standard First Line Treatment for Advanced Non-small Cell Lung Cancer — NCT01809210.
- Registry record last updated 2018-03-13; status last verified 2018-03.
- Results first posted 2018-03-13.
- 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
The registry record describes a completed, Phase I, open-label, multicentre, single-group study sponsored by AstraZeneca, with enrollment of 55. It evaluated selumetinib with first-line chemotherapy regimens in patients with advanced or metastatic non-small cell lung cancer, using dose-finding cohorts and optional expansion.
The primary endpoint was dose-limiting toxicity during the early treatment period, among patients who received at least 1 dose of selumetinib. Its definition and timeframe are recorded, but its numerical result is not reported in the registry record. Serious adverse event counts are listed by cohort, while secondary efficacy and pharmacokinetic outcomes are named without results. Because the cohorts are not randomized comparisons, the posted counts are descriptive rather than comparative evidence.