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 Trial Of PF-00299804 In Patients With Advanced Refractory Lung Cancer (NCT00553254), a non-randomized phase 2 study. The primary outcome is Recommended Phase 2 Dose (RP2D) - Phase 1.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00553254 |
| Phase | Phase 2 |
| Condition | Carcinoma, Non Small Cell Lung |
| Allocation | Non-Randomized |
| Design model | Not Stated |
| Masking | None (open label) |
| Enrollment | 55 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Pfizer |
| Results posted | Yes |
2. Clinical Question
Population
People in Korea with advanced non-small cell lung cancer whose disease was refractory to chemotherapy and erlotinib or gefitinib. The primary endpoint’s as-treated population included enrolled participants who received at least 1 dose of study medication.
Intervention
PF-00299804, a drug studied at 30 mg and 45 mg in the phase 1 groups and at 45 mg in the phase 2 group.
Comparator
There was no randomized comparator. The registry describes a non-randomized, open-label, single-arm trial, so outcomes are not a comparison of PF-00299804 with a control treatment.
Primary question
Which dose met the registry’s toxicity-based criteria for the recommended phase 2 dose? The primary endpoint was the Recommended Phase 2 Dose, assessed from baseline up to Day 21 using dose-limiting toxicities.
The registry’s brief summary states that the study assessed safety and efficacy in people with advanced lung cancer. Its official title describes a phase 1/2 study, while the phase field lists phase 2. The primary endpoint is a dose-selection measure, not a direct estimate of comparative efficacy. Secondary outcomes include progression-free survival and overall survival at Month 6 in phase 2, objective response in phase 1, biomarker and mutation measures, and pharmacokinetic measures. These listed outcomes identify what the study set out to assess; the registry record does not report their numerical results in the information available for this analysis.
Because treatment allocation was not randomized and there was no control group, the central question is descriptive: what dose was designated using the specified toxicity rule, and what outcomes were recorded among treated participants? The posted information gives safety counts for the dose groups but does not state the selected recommended phase 2 dose or provide a numerical result for the primary endpoint. It therefore does not establish a treatment contrast or quantify an effect relative to another therapy.
3. Trial Design
The registry describes a phase 1/2, open-label, single-arm study of PF-00299804 in people in Korea with advanced non-small cell lung cancer refractory to chemotherapy and erlotinib or gefitinib. The phase field lists PHASE2, while the official title identifies the study as phase 1/2 and distinguishes phase 1 dose determination from phase 2 efficacy evaluation. Allocation is non-randomized and masking is none. These design features mean that participants and investigators were not assigned across randomized treatment groups, and there is no blinded assessment described in the record.
Enrollment was 55. Safety groups are listed for PF-00299804 30 mg in phase 1, PF-00299804 45 mg in phase 1, and PF-00299804 45 mg in phase 2. The corresponding numbers at risk for the adverse-event summaries were 6, 6, and 43. These are distinct registry groups, not randomized arms intended for a like-for-like comparison. In particular, comparing phase 1 and phase 2 counts as if they arose from a concurrent controlled design would ignore the different study phases and the absence of randomization.
The primary measure was Recommended Phase 2 Dose, with a timeframe from baseline up to Day 21. The stated rule was to designate as both the maximum tolerated dose and the recommended phase 2 dose the highest dose at which less than 33 percent (%) of participants experienced dose-limiting toxicities. The registry defines the qualifying toxicities, but the primary endpoint’s observed result is not reported in the registry record.
The listed secondary outcomes span survival, response, biomarkers, mutation status, and drug concentration and timing measures. There are 22 outcome measures recorded, while the analyses count is 0. That combination does not establish that no analyses were conducted; it means the registry entry does not enumerate analyses in that field. The study status is completed. Its start date is 2008-02-05 and its primary completion date is 2010-08-03. Further details about the recruitment process, dose assignment sequence, follow-up schedule, and conduct of analyses are not reported 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.
Overall Study
| Stage | PF-00299804 30 mg - Phase 1 | PF-00299804 45 mg - Phase 1 | PF-00299804 45 mg - Phase 2 |
|---|---|---|---|
| STARTED | 6 | 6 | 43 |
| COMPLETED | 0 | 0 | 0 |
| NOT COMPLETED | 6 | 6 | 43 |
Reasons for not completing this period:
| Reason | PF-00299804 30 mg - Phase 1 | PF-00299804 45 mg - Phase 1 | PF-00299804 45 mg - Phase 2 |
|---|---|---|---|
| Death | 3 | 6 | 25 |
| Lost to Follow-up | 0 | 0 | 2 |
| Withdrawal by Subject | 2 | 0 | 2 |
| Study terminated by Sponsor | 0 | 0 | 1 |
| Other | 1 | 0 | 13 |
Analysis population for the primary outcome, as defined in the registry: As-treated population included all enrolled participants who received at least 1 dose of study medication.
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: Data for baseline characteristics was pre-specified in statistical analysis plan to be analyzed and summarized per phase.
| Characteristic | Unit | PF-00299804 - Phase 1 All Participants | PF-00299804 45 mg - Phase 2 | Total |
|---|---|---|---|---|
| Participants analysed | 12 | 43 | 55 | |
| Age, Continuous | years | 52.6 (SD 12.3) | 57.0 (SD 8.7) | 56.0 (SD 9.6) |
| Sex: Female, Male: Female | Participants | 9 | 23 | 32 |
| Sex: Female, Male: Male | Participants | 3 | 20 | 23 |
6. Primary Endpoint
The registry lists the primary outcome as Recommended Phase 2 Dose (RP2D) - Phase 1, measured over Baseline up to Day 21.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | mg |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the Recommended Phase 2 Dose (RP2D), measured in mg. It asks which dose met the registry’s prespecified toxicity criterion during the period from baseline up to Day 21. The stated rule identifies the highest dose at which less than 33 percent (%) of participants had dose-limiting toxicities; that dose was to be designated both the maximum tolerated dose and the recommended phase 2 dose.
The registry defines dose-limiting toxicity using specified adverse events. These include Grade 3/4 nausea, vomiting, or diarrhea despite adequate or maximal medical intervention and/or prophylaxis; Grade 3 or higher non-hematological toxicity or death related to an adverse event; treatment-related toxicity delaying scheduled treatment for more than 14 days; Grade 4 neutropenia, including the stated absolute neutrophil count and duration criteria or febrile neutropenia; and Grade 4 thrombocytopenia or bleeding requiring platelet transfusion. The registry’s original threshold and event definitions are part of how the endpoint is interpreted.
General statistical context: dose-selection endpoints are used in early development to guide which dose is carried forward for later evaluation. They focus on tolerability under the study’s definitions and observation period, rather than directly measuring whether a treatment improves an outcome relative to a comparator. The endpoint’s observed value, the number of dose-limiting toxicities by dose, and the resulting dose designation are not reported in the registry record. Consequently, the stated rule can be explained, but its application to the participants cannot be independently assessed from the reported primary result.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a completed, non-randomized study with 55 participants enrolled and a single-arm treatment structure. It lists three safety groups: PF-00299804 30 mg in phase 1, PF-00299804 45 mg in phase 1, and PF-00299804 45 mg in phase 2. The reported numbers at risk are 6, 6, and 43, respectively. These group counts provide context for the adverse-event summaries, but they do not create a randomized comparison between doses or phases.
For serious adverse events, the counts of participants affected are 1 in the phase 1 30 mg group, 0 in the phase 1 45 mg group, and 9 in the phase 2 45 mg group. For other adverse events, the corresponding affected counts are 6, 6, and 41. The serious-event counts are descriptive. Without a control group, they cannot be interpreted as excess risk attributable to PF-00299804 compared with another treatment or with no treatment. Nor do counts alone explain event duration, severity beyond the serious-event classification, recurrence, or clinical course.
The registry lists pleural effusion at 16.7% and disease progression at 11.6% among the top serious adverse-event terms. Febrile neutropenia, diarrhoea, pneumonia, and hydrocephalus are each listed at 2.3%. The denominator and specific method used to calculate these percentages are not reported in the registry record. These percentages should therefore be read as registry-listed summaries, not reinterpreted as rates using a denominator selected by the reader.
The primary endpoint is a toxicity-based dose recommendation, but its result is not reported in the registry record. The available information does not say which dose met the criterion, how many participants at each dose had dose-limiting toxicities, or whether the recommended phase 2 dose was formally assigned. Thus the primary question cannot be answered from the posted endpoint description alone.
The secondary outcomes include progression-free survival and overall survival at Month 6, objective response, biomarkers, mutation status, and pharmacokinetic measures. Their presence in the outcome list does not supply the results. Numerical estimates, uncertainty intervals, and analysis details for those outcomes are not reported in the registry record. The registry also records 22 outcome measures and 0 analyses, so the record does not give a detailed account of how individual outcomes were analyzed.
General statistical context: a count or percentage describes the observed participants in a defined group; it does not by itself show how precisely a population-level rate has been estimated. Small group sizes can make estimates sensitive to a small number of events. Since no confidence intervals or inferential analyses are listed in the available record, the precision of the adverse-event summaries cannot be assessed from those summaries alone.
9. Statistical Methods Explained
The record’s central statistical feature is that the primary endpoint is dose selection based on toxicity, not a comparison of treatment effects between randomized groups. Its stated rule is to choose the highest dose at which less than 33 percent (%) of participants experienced dose-limiting toxicities during baseline up to Day 21. The maximum tolerated dose and recommended phase 2 dose were to be designated using this same rule. The record defines which toxicities qualify, but does not report the observed dose-limiting toxicity results or the dose selected.
What does the dose-selection rule mean?
General statistical context: a threshold rule turns observed participant outcomes into a decision category. Here, the classification depends on whether the proportion with qualifying toxicities at a dose falls below the stated boundary, as well as on which dose is the highest one satisfying that condition. The rule is only interpretable alongside the event definition, the observation window, and the number of participants assessed at each dose. The registry describes the event definition and window, but the primary endpoint result and dose-specific dose-limiting toxicity counts are not reported in the registry record. The decision therefore cannot be reconstructed from the posted information.
The toxicity definition includes several different clinical events: severe gastrointestinal events despite medical intervention or prophylaxis; severe non-hematological toxicity or related death; delayed recovery from treatment-related toxicity; specified severe neutropenia or febrile neutropenia; and severe thrombocytopenia or bleeding requiring platelet transfusion. Because these events differ in type and timing, the rule treats them as qualifying events under a shared endpoint definition. The record does not describe adjudication procedures, the handling of multiple qualifying events in one participant, or any additional operational details.
How should the group counts be read?
The safety summaries identify 6 participants at risk in the 30 mg phase 1 group, 6 in the 45 mg phase 1 group, and 43 in the 45 mg phase 2 group. These figures are denominators for the adverse-event group summaries, not evidence of randomized allocation. The record labels the trial non-randomized, open-label, and single-arm. General statistical context: randomization helps make treatment groups comparable on average, while an uncontrolled study does not provide that design-based protection. Differences in event counts across the listed groups may reflect their different sizes and phases, as well as participant characteristics or observation patterns not described in the record. They should not be treated as a causal comparison.
The serious-event counts and other adverse-event counts are reported as numbers of affected participants. A participant count is not the same as a count of event episodes: the record does not explain whether people with repeated events are counted once or whether event-level counts are also available. The top serious-event terms are accompanied by percentages, but their denominator and calculation method are not reported in the registry record. General statistical context: percentages require a defined denominator and a clear unit of analysis, such as participants or events, to support precise interpretation.
What can the listed analyses establish?
The outcome list includes survival and response outcomes, biomarkers, mutation status, and pharmacokinetic measurements. These are different outcome types and ordinarily require methods suited to their measurement scales and follow-up structure. General statistical context: time-to-event outcomes account for the timing of an event and may also need to account for participants whose event status is not observed by the end of follow-up. The registry lists progression-free survival and overall survival at Month 6, but does not report definitions, censoring rules, estimates, or uncertainty intervals in the information. Those analytical details are not reported in the registry record.
The record lists 22 outcome measures and 0 analyses. This is a registry-field count, not a complete statistical analysis plan. It does not establish whether models, adjustments, subgroup analyses, or interim analyses were used. Those details are not reported in the registry record. Likewise, no protocol-based sample-size justification is reported in the registry record.
The record describes a planning illustration based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It says the effect to detect is a stated fraction of the observed effect; time-to-event illustrations use total events, while other illustrations use participants per arm. These are planning assumptions, not evidence that the trial itself randomized participants equally between two arms. The trial is listed as single-arm and non-randomized. Such calculations are not observed power, and the numerical planning outputs and the fractions used are not reported in the registry record.
Why does uncertainty matter?
General statistical context: an estimate is most useful when accompanied by an indication of its precision. A confidence interval expresses a range of values compatible with the data under a specified statistical method and assumptions. Wider intervals indicate less precision; narrower intervals indicate greater precision. No confidence intervals for the primary endpoint or the reported adverse-event counts are given in the registry information available for this analysis. The small phase 1 group sizes also mean that the event counts describe only a limited number of participants, without allowing a reliable precision assessment from counts alone.
General statistical context: over-dispersion arises when outcome variability is greater than expected under a simple statistical model. Accounting for it can prevent an analysis from overstating precision when observations vary more than the model assumes. The registry does not report a model for the primary endpoint or secondary outcomes, and it does not identify over-dispersion as an issue. There is therefore no basis to say whether such a method was used or needed in any analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 22 outcome measures, including a primary dose-selection endpoint and secondary outcomes covering survival, objective response, biomarkers, mutation status, and pharmacokinetics. When a study examines multiple outcomes, there can be more than one opportunity to observe an apparently notable result by chance. General statistical context: multiplicity procedures can control the overall error rate across a defined family of tests, while a prespecified hierarchy can give some outcomes priority over others. The registry information does not report whether any multiplicity adjustment or testing hierarchy was used.
The record reports 0 analyses in its analyses count. This does not establish that the study had no statistical analyses; it means that analyses are not enumerated in that registry field. A full statistical analysis plan, model specifications, hypotheses, and any adjustment for multiple testing are not reported in the registry record. No outcome-level estimates or inference results are available to evaluate how multiple outcomes were handled.
Missing data are also not described. The record does not report the amount or pattern of missing outcome data, how incomplete follow-up was handled, whether participants were excluded from particular analyses, or whether sensitivity analyses were conducted. General statistical context: missingness can affect interpretation when the availability of an outcome is related to participants’ characteristics or outcomes. For survival measures, the handling of incomplete observation is especially relevant, but censoring definitions and rules are not reported in the registry record.
The primary endpoint population is described as the as-treated population: enrolled participants who received at least 1 dose of study medication. This definition does not specify the analysis population for each secondary outcome. The registry record does not report whether all enrolled participants contributed to every analysis, nor does it describe any intention-to-treat population or per-protocol population. The reported safety group denominators should therefore be read as group-specific safety summaries, not as a complete account of analysis populations across all outcomes.
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 |
|---|---|---|---|
| PF-00299804 30 mg - Phase 1 | 1 | 6 | 16.7% |
| PF-00299804 45 mg - Phase 1 | 0 | 6 | 0.0% |
| PF-00299804 45 mg - Phase 2 | 9 | 43 | 20.9% |
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 |
|---|---|
| PF-00299804 30 mg - Phase 1 | 6 of 6 |
| PF-00299804 45 mg - Phase 1 | 6 of 6 |
| PF-00299804 45 mg - Phase 2 | 41 of 43 |
Most frequent serious adverse event terms
| Event term | Organ system | PF-00299804 30 mg - Phase 1 | PF-00299804 45 mg - Phase 1 | PF-00299804 45 mg - Phase 2 |
|---|---|---|---|---|
| Disease progression | General disorders | 0 of 6 | 0 of 6 | 5 of 43 |
| Febrile neutropenia | Blood and lymphatic system disorders | 0 of 6 | 0 of 6 | 1 of 43 |
| Diarrhoea | Gastrointestinal disorders | 0 of 6 | 0 of 6 | 1 of 43 |
| Pneumonia | Infections and infestations | 0 of 6 | 0 of 6 | 1 of 43 |
| Hydrocephalus | Nervous system disorders | 0 of 6 | 0 of 6 | 1 of 43 |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | 1 of 6 | 0 of 6 | 0 of 43 |
| Hypotension | Vascular disorders | 0 of 6 | 0 of 6 | 1 of 43 |
Most frequent non-serious adverse event terms
| Event term | Organ system | PF-00299804 30 mg - Phase 1 | PF-00299804 45 mg - Phase 1 | PF-00299804 45 mg - Phase 2 |
|---|---|---|---|---|
| Dermatitis acneiform | Skin and subcutaneous tissue disorders | 4 of 6 | 6 of 6 | 35 of 43 |
| Diarrhoea | Gastrointestinal disorders | 2 of 6 | 6 of 6 | 36 of 43 |
| Paronychia | Infections and infestations | 3 of 6 | 3 of 6 | 29 of 43 |
| Stomatitis | Gastrointestinal disorders | 2 of 6 | 3 of 6 | 20 of 43 |
| Palmar-plantar erythrodysaesthesia syndrome | Skin and subcutaneous tissue disorders | 2 of 6 | 3 of 6 | 13 of 43 |
| Dry skin | Skin and subcutaneous tissue disorders | 0 of 6 | 1 of 6 | 16 of 43 |
| Pruritus | Skin and subcutaneous tissue disorders | 2 of 6 | 0 of 6 | 15 of 43 |
| Decreased appetite | Metabolism and nutrition disorders | 0 of 6 | 2 of 6 | 14 of 43 |
| Mucosal inflammation | General disorders | 2 of 6 | 3 of 6 | 7 of 43 |
| Cough | Respiratory, thoracic and mediastinal disorders | 3 of 6 | 0 of 6 | 9 of 43 |
Serious events with the highest arm-level rate: pleural effusion (16.7%), disease progression (11.6%), febrile neutropenia (2.3%), diarrhoea (2.3%), pneumonia (2.3%).
The safety summaries list serious adverse events and other adverse events separately for three PF-00299804 groups. For PF-00299804 30 mg in phase 1, 1 of 6 participants at risk was affected by a serious adverse event, and 6 of 6 were affected by other adverse events. For PF-00299804 45 mg in phase 1, 0 of 6 were affected by a serious adverse event, and 6 of 6 were affected by other adverse events. For PF-00299804 45 mg in phase 2, 9 of 43 were affected by a serious adverse event, and 41 of 43 were affected by other adverse events.
The registry also lists pleural effusion at 16.7% and disease progression at 11.6% among the top serious adverse-event terms. Febrile neutropenia, diarrhoea, pneumonia, and hydrocephalus are each listed at 2.3%. The denominator for these term percentages is not reported in the registry record. They should not be assumed to use the group denominators unless the record specifies that calculation.
General statistical context: these figures describe adverse events recorded in a single-arm study. They do not compare event frequency with a control group, and they do not by themselves establish causation. The registry record does not give confidence intervals, event timing, repeated-event counts, or additional information about exposure duration. Consequently, the counts are useful as descriptive safety summaries, but their precision and comparative meaning are limited.
12. What the Result Does, and Does Not, Mean
The registry record documents a completed single-arm study of PF-00299804 in advanced non-small cell lung cancer. It gives a toxicity-based rule for selecting the recommended phase 2 dose and describes the adverse-event groups. It also lists a broad set of secondary outcomes. The record does not, however, report the primary dose-selection result or numerical results for the listed efficacy and other secondary outcomes. The main question of which dose met the rule therefore remains unanswered by the outcome information.
The safety counts describe participants affected within the listed groups: 1 serious adverse event in the 30 mg phase 1 group, 0 in the 45 mg phase 1 group, and 9 in the 45 mg phase 2 group. Other adverse events affected 6, 6, and 41 participants, respectively. These figures do not establish that one dose is safer than another. The groups belong to different phases, are not randomized comparators, and have different numbers at risk. No controlled treatment contrast is available.
The listed serious adverse-event terms and percentages add descriptive detail, but the denominator for the percentages is not reported in the registry record. The absence of outcome estimates and confidence intervals also means that the precision of the listed summaries cannot be quantified from the registry information.
General statistical context: evidence from a single-arm study can describe outcomes among participants treated under the study conditions. It cannot, by itself, separate treatment effects from the natural course of disease, participant selection, or other influences. A randomized control group can help address such comparisons, but this trial is explicitly non-randomized and single-arm.
The record’s outcome list should not be mistaken for a set of demonstrated findings. Progression-free survival, overall survival, response, biomarkers, mutation status, and pharmacokinetics are named as measures, but their numerical results are not reported in the registry record. The available information supports a description of the trial’s design, endpoint definitions, and adverse-event counts—not a conclusion about comparative efficacy or the size of a treatment effect.
13. Why This Trial Matters Statistically
This study illustrates an important distinction in clinical trial statistics: a primary endpoint can guide a development decision without being a comparative measure of efficacy. The Recommended Phase 2 Dose endpoint is defined by a dose-limiting toxicity threshold during a stated period. The rule makes the decision criterion explicit, but interpretation still depends on observed toxicity counts and the dose actually selected. Those results are not reported in the registry record.
The record also demonstrates why study design determines what a result can answer. Although safety information is presented for more than one dose and study phase, the trial is non-randomized and single-arm. The listed groups are not a like-for-like control comparison. Descriptive counts can document what was observed in each group, but they cannot establish a causal difference between dose groups or against another treatment.
A further statistical lesson is the value of complete outcome reporting. The registry lists 22 outcome measures, yet its analyses count is 0, and numerical results for the primary endpoint and secondary outcomes are not reported in the information available for this analysis. Without estimates, uncertainty intervals, and analysis details, readers cannot evaluate the precision of outcomes or the methods used to address missing data and multiple outcomes.
Finally, safety summaries require attention to both numerator and denominator. The record reports affected-participant counts and group sizes, while the denominator for the listed serious-event term percentages is not reported. Clear denominators and definitions are essential for readers to interpret rates consistently. Together, these features make the study a useful example of how endpoint definition, design, and reporting completeness shape the conclusions that can be drawn from a clinical trial 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 statistical interpretation is the lack of a reported primary endpoint result. The registry defines the Recommended Phase 2 Dose rule, but does not state which dose met it, provide dose-limiting toxicity counts by dose, or report the resulting recommended dose. The endpoint’s stated timeframe is baseline up to Day 21, but the record does not give further detail about assessment completeness or the handling of participants without an evaluable assessment.
The study is non-randomized, open-label, and single-arm. The safety summaries include phase 1 and phase 2 groups, but they are not randomized comparison groups. The different group sizes and study phases make direct comparisons particularly difficult to interpret. There is no control-group result in the registry record against which to assess the adverse-event counts or any potential efficacy outcome.
The registry lists secondary outcomes but does not report their numerical results in the information. It also does not provide confidence intervals, analysis methods, details of follow-up, missing-data procedures, or a full account of the analysis populations. The number of outcome measures is recorded as 22 and the analyses count as 0, but this does not substitute for outcome-specific analysis details.
The serious adverse-event term percentages are listed without a reported denominator. The safety record does not provide additional information about timing, duration, recurrence, or exposure for the adverse events. These omissions restrict interpretation of both the percentages and the event counts. The registry’s as-treated population definition applies to the primary endpoint, but populations used for the secondary outcomes are not reported in the registry record.
The record does not report a protocol-based sample-size justification, interim analysis plan, or statistical analysis plan. The described planning illustration is distinct from the trial’s single-arm design and is not a report of observed power. These reporting gaps limit assessment of the study’s planned and executed statistical procedures.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Trial Of PF-00299804 In Patients With Advanced Refractory Lung Cancer — NCT00553254.
- Registry record last updated 2020-10-19; status last verified 2020-09.
- Results first posted 2020-10-19.
- 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
NCT00553254 is a completed, open-label, non-randomized, single-arm phase 1/2 study of PF-00299804 in people in Korea with advanced non-small cell lung cancer refractory to chemotherapy and erlotinib or gefitinib. The phase field lists PHASE2. Enrollment was 55. The primary endpoint was Recommended Phase 2 Dose, defined using a dose-limiting toxicity threshold from baseline up to Day 21. Its observed result is not reported in the registry record.
Safety groups are listed for 30 mg in phase 1, 45 mg in phase 1, and 45 mg in phase 2, with 6, 6, and 43 participants at risk. Serious adverse events affected 1, 0, and 9 participants; other adverse events affected 6, 6, and 41. The record lists 22 outcome measures and 0 analyses. Numerical results for the primary and secondary outcomes, analysis methods, and the denominator for serious-event term percentages are not reported in the registry record.