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 Chemotherapy for Patients With Non-Small Cell Lung Cancer (NCT00380718), a non-randomized phase 4 study. The primary outcome is Proportion of Participants With a Complete or Partial Response (Objective Response Rate [ORR]).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00380718 |
| Phase | Phase 4 |
| Condition | Non-small Cell Lung Cancer |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 33 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Eli Lilly and Company |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned Taiwanese patients with advanced non-small cell lung cancer who had received prior chemotherapy. The primary outcome population was defined as participants who received at least one dose of study drug. The registry record does not give further eligibility details or the number in this outcome population.
Intervention
The intervention was pemetrexed. The study purpose describes dosing tailored to individual patient tolerance. The exact dose and schedule are not reported in the registry record.
Comparator
There was no concurrent comparator group. The study was open-label, non-randomized, and single-group, so the posted results do not provide a randomized treatment contrast.
Primary question
The primary question was what proportion of participants achieved a complete or partial response according to RECIST. These responses were combined as the objective response rate. The registry record does not report the observed response proportion or its confidence interval.
The clinical question is therefore descriptive: among the treated participants meeting the stated outcome population definition, how often was a best response of complete response or partial response recorded? This differs from asking whether pemetrexed caused a change compared with another treatment or with no treatment. Without a concurrent comparator, response observations cannot by themselves separate treatment effects from other explanations, including the course of disease or participant characteristics.
The registry also names disease control rate, overall survival, progression-free survival, duration of response, time to treatment failure, and time to tumor progression as secondary outcomes. Those outcomes concern different aspects of disease status and follow-up; they do not substitute for the primary response result. Numerical results for those outcomes are not reported in the registry record.
3. Trial Design
This was an open-label, single-group Phase IV study with non-randomized allocation. The registry lists pemetrexed as the intervention and gives enrollment as 33. The study status is completed, with a start date of 2006-11 and primary completion in 2008-09. These registry dates describe the study record; they do not by themselves establish how long each participant was followed.
Because there was no randomized comparator, the design does not support a like-for-like comparison between treatment groups. The study can describe outcomes recorded among participants who received pemetrexed, but a change or response within a single group cannot establish what would have happened to those same participants under a different treatment or without treatment. Open-label conduct also means the study was not masked. The registry record does not describe additional procedures intended to reduce potential assessment or reporting bias.
The primary outcome was the proportion of participants with a best response of complete response or partial response, assessed using RECIST. The stated population for that outcome was participants who received at least one dose. Participants classified as Unknown were considered nonresponders rather than missing. Tumor assessments were scheduled every 2 cycles during therapy and every 6-8 weeks during post-therapy follow-up, until documented disease progression or up to 18 months after enrollment.
The registry names several secondary outcomes, including disease control rate and time-to-event outcomes. The actual dose and schedule, detailed assessment procedures, analysis methods, and any interim analyses are not reported in the registry record. The enrollment figure should not be taken as the number assessed for every outcome: the record does not state the number who received at least one dose or the number contributing to each secondary analysis.
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: Thirty-seven participants were screened; 3 did not meet inclusion/exclusion criteria and one withdrew.
Overall Study
| Stage | Pemetrexed |
|---|---|
| STARTED | 33 |
| Received at Least One Dose of Study Drug | 33 |
| Received Escalated Dose in Cycle 2 | 25 |
| COMPLETED | 15 |
| NOT COMPLETED | 18 |
Reasons for not completing this period:
| Reason | Pemetrexed |
|---|---|
| Adverse Event | 1 |
| Death after 30-day post-therapy followup | 17 |
Analysis population for the primary outcome, as defined in the registry: Number of participants who received at least one dose of study drug. Participants who were classified as "Unknown" were considered nonresponders rather than missing.
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 | Pemetrexed |
|---|---|---|
| Participants analysed | 33 | |
| Age Continuous | years | 58.0 (SD 11.27) |
| Sex: Female, Male: Female | Participants | 13 |
| Sex: Female, Male: Male | Participants | 20 |
| Region of Enrollment: Taiwan | participants | 33 |
| Disease Characteristic: Basis for Diagnosis: Histopathological | participants | 17 |
| Disease Characteristic: Basis for Diagnosis: Cytological | participants | 16 |
| Disease Characteristic: Disease Stage at Study Entry: Stage IIIB | participants | 3 |
| Disease Characteristic: Disease Stage at Study Entry: Stage IV | participants | 30 |
| Disease Characteristic: Eastern Cooperative Oncology Group Performance Status: 0 - Fully Active | participants | 9 |
| Disease Characteristic: Eastern Cooperative Oncology Group Performance Status: 1 - Ambulatory, Restricted Strenuous Activity | participants | 24 |
| Disease Characterstic: Pathological Diagnosis Code: Adenocarcinoma of Lung | participants | 23 |
| Disease Characterstic: Pathological Diagnosis Code: Large Cell Carcinoma of Lung | participants | 1 |
| Disease Characterstic: Pathological Diagnosis Code: Mixed Cell | participants | 0 |
| Disease Characterstic: Pathological Diagnosis Code: Squamous Cell Carcinoma of Lung | participants | 8 |
| Disease Characterstic: Pathological Diagnosis Code: Non-Small Cell Lung Carcinoma | participants | 1 |
| Race/Ethnicity: East/Southeast Asian | participants | 33 |
| Smoking Status: Ever Smoking | participants | 20 |
| Smoking Status: Never Smoking | participants | 13 |
| 24 Hour Creatinine Clearance | milliliters per minute (ml/min) | 81.142 (SD 24.9265) |
| Body Temperature | degrees Celcius (°C) | 36.25 (SD 0.336) |
| Disease Characteristic: Time from Initial Diagnosis to Prior Chemotherapy Failure | months | 12.04 (SD 23.764) |
| Disease Characteristic: Time from Initial Diagnosis to Study Entry | months | 11.54 (SD 20.829) |
| Disease Characteristic: Time from Prior Chemotherapy Failure to Study Entry | months | 1.00 (SD 0.486) |
6. Primary Endpoint
The registry lists the primary outcome as Proportion of Participants With a Complete or Partial Response (Objective Response Rate [ORR]), measured over baseline to measured progressive disease (Tumor assessments were performed every 2 cycles during therapy and 6-8 weeks during post-therapy until documented disease progression, or up to 18 months after enrollment).
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | 95% Confidence Interval |
| Unit | proportion of responders |
| Endpoint classification | Continuous Endpoint |
The primary endpoint was the objective response rate, defined as the proportion of participants whose best response was complete response or partial response under RECIST. The registry defines the calculation as the number of complete and partial responses divided by the number of participants. Participants classified as Unknown were counted as nonresponders rather than treated as missing for this endpoint.
Response is a categorical assessment of tumor status. Combining complete and partial responses into a proportion gives a compact summary of how many participants met the specified response criteria during the assessment period. As a general statistical point, a proportion can describe the observed frequency of an outcome, while a confidence interval conveys uncertainty around the estimated frequency. The registry specifies a 95% confidence interval for the primary endpoint, but the observed proportion and its interval are not reported in the registry record.
The assessment window ran from baseline to measured progressive disease. Tumor assessments were performed every 2 cycles during therapy and every 6-8 weeks during post-therapy follow-up until documented progression, or up to 18 months after enrollment. The endpoint is not the same as overall survival, progression-free survival, or duration of response; those are listed separately as secondary outcomes. A response proportion also does not, on its own, show whether an observed response was caused by treatment, particularly in a single-arm study without a concurrent comparator.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record defines an efficacy endpoint but does not report its observed value. It therefore does not provide the primary response proportion, the numbers of complete or partial responses, or the stated 95% confidence interval. Without those values, the magnitude of the response estimate and its statistical precision cannot be evaluated from the registry record. The listed secondary outcomes likewise have no numerical results reported in the registry record.
The distinction between an endpoint definition and an endpoint result is important. A clear definition tells readers what was intended to be counted and who was eligible for the primary analysis. It does not reveal how many participants met the response criteria. The registry says that participants who received at least one dose formed the primary outcome population and that Unknown classifications were treated as nonresponses. The number meeting the treated-population definition is not reported in the registry record, so the denominator for the response calculation cannot be established from the posted information.
Even if a response estimate were available, its interpretation would be limited by the single-group design. The study had no concurrent control arm and no random allocation. A response observed during follow-up would describe outcomes in this group; it would not quantify a treatment difference against another option. Open-label assessment is another design feature to consider when interpreting outcomes that involve clinical measurement or classification. The registry does not describe masking or additional safeguards against assessment bias.
The record specifies a 95% confidence interval. In general, a confidence interval communicates the uncertainty associated with an estimated quantity under the analysis method. Its width depends on the observed data and statistical approach. Since neither the response estimate nor its interval is reported, the precision of the primary result cannot be judged. No estimate should be reconstructed from enrollment alone: the response count and the relevant analysis denominator are not stated.
For safety, the record lists 13 participants with serious adverse events among 33 at risk, and 33 participants with other adverse events among 33 at risk. It also lists serious adverse event terms with percentages. These are descriptive safety entries, not a randomized comparison. They do not establish that pemetrexed caused any particular event, and the registry record does not report deaths in the adverse-event entry. Detailed interpretation of these safety figures requires keeping event categories, participant counts, and the lack of a comparator distinct.
Overall, the registry supplies useful information about the study question, design, endpoint definition, and some safety counts, but it does not provide the primary efficacy result. Conclusions about the observed response frequency or its precision cannot be drawn from the record. The design also means that the posted results, even if complete, would describe a treated group rather than estimate a controlled treatment effect.
9. Statistical Methods Explained
The statistical concepts relevant to this study begin with the outcome type. Each participant’s best response was classified according to RECIST, and the primary endpoint summarized the proportion classified as complete or partial responders. This is a participant-level categorical outcome summarized as a proportion. The registry describes the response numerator and denominator, specifies a 95% confidence interval, and states that Unknown classifications were treated as nonresponses.
What does the response proportion represent?
A proportion is a summary of how frequently an outcome occurs in the analysis population. In this study, the intended numerator combined complete and partial responses, while the denominator was the participants who received at least one dose. The registry definition is helpful because it identifies how Unknown classifications were handled: they were included as nonresponders rather than excluded as missing for the primary endpoint. However, the response count, the number in the treated population, and the resulting proportion are not reported in the registry record. Consequently, the endpoint cannot be independently interpreted from its definition alone.
As a general explanation, estimates based on proportions are subject to sampling uncertainty: another group of participants from the same underlying population might yield a different observed proportion. A confidence interval is a way of expressing uncertainty around an estimate under specified statistical assumptions. The registry lists a 95% confidence interval for the primary measure, but does not report the interval’s endpoints or the method used to construct it. There is therefore no interval width to assess and no basis for judging precision from the posted primary outcome information.
Why does the study design matter?
In a randomized comparison, random allocation helps create groups that can be compared while reducing systematic differences at baseline. This study was non-randomized and single-group, with no concurrent comparator. Its response proportion is therefore a description of outcomes in the enrolled and treated group, not a between-group treatment effect. General statistical interpretation should distinguish an observed outcome from a causal contrast: an outcome occurring after treatment does not, by itself, establish what would have happened under an alternative.
The study was open-label and had no masking. In general, masking can reduce the possibility that knowledge of treatment assignment influences assessments or reporting. The registry does not describe whether any other procedures were used to limit those influences. This limitation is especially relevant to outcomes requiring clinical assessment, although the record does not provide enough detail to characterize how response decisions were made beyond their stated basis in RECIST.
How should planning assumptions be read?
The registry’s sample-size assumptions describe a planning illustration computed from the observed effect, using a two-sided significance level 0.05, equal allocation between two arms, and a normal approximation. This is not the design of the study itself: the trial was single-group and had no two-arm randomized comparison. The assumptions describe an illustration, not observed power, and do not supply evidence about the trial’s efficacy result. The numerical planning outputs and the effect used for the illustration are not reported in the registry record.
As a general point, a normal approximation is a mathematical shortcut for estimating uncertainty or planning under certain conditions. Its suitability depends on the data and the particular calculation. Proportions are bounded quantities derived from categorical outcomes, so the behavior of an approximation can depend on the observed response frequency and sample size. The response frequency is absent here, so the adequacy of a particular approximation for the endpoint cannot be evaluated. The registry also does not report a full statistical analysis plan or the method used for the confidence interval.
What do secondary outcomes add?
The registry lists disease control rate, overall survival, progression-free survival, duration of response, time to treatment failure, and time to tumor progression. These outcomes address distinct questions and may require distinct estimands, follow-up definitions, and analysis methods. For example, a time-to-event analysis generally concerns the timing of an event and may account for participants whose event has not been observed by the end of follow-up. The registry record does not give numerical results or detailed analysis methods for these secondary outcomes, so their handling of follow-up and censoring cannot be assessed.
The listed sample-size assumptions distinguish time-to-event planning, which uses a total event count, from other planning illustrations, which use participants per arm. Those are general planning conventions in the record and should not be mistaken for observed trial outcomes. The study itself had no two-arm allocation, and the registry does not report a trial-specific sample-size justification.
What remains unavailable?
The record does not report the primary response estimate, its confidence interval, the response counts, or the number of participants in the primary analysis population. It also does not report the statistical method for the primary confidence interval, the analysis methods for secondary outcomes, or any interim analysis information. These absences limit the reproducibility and evaluation of the statistical results. General methodological principles can explain what the endpoint and interval are intended to convey, but they cannot recover the missing estimates.
10. Multiplicity, Missing Data and Analysis Populations
The registry names the objective response rate as the primary outcome and lists secondary outcomes covering disease control, survival, progression, response duration, treatment failure, and tumor progression. Testing or interpreting multiple outcomes raises a general multiplicity concern: the more outcomes examined, the greater the opportunity for chance findings if each is treated as independently confirmatory. The registry record does not report a multiplicity adjustment, a hierarchy among secondary outcomes, or a statistical analysis plan explaining how those outcomes were to be interpreted.
The record lists 0 analyses. Numerical results for the primary and secondary efficacy outcomes are not reported in the registry record. It is therefore not possible to assess whether planned analyses were carried out, whether any adjustment was applied, or how inferential claims were intended to be controlled. The absence of reported methods should not be filled in by assuming a conventional approach.
The primary outcome population was participants who received at least one dose of study drug. Participants classified as Unknown were considered nonresponders rather than missing for the response endpoint. This is a stated handling rule, but it does not reveal how many participants were classified as Unknown or how many received a dose. The registry record does not report a full accounting of enrollment, treatment initiation, follow-up, and outcome availability.
Handling of missing observations for the secondary outcomes is not reported in the registry record. Nor does the record describe whether analyses used additional populations, how withdrawals were handled, or what assumptions were made about incomplete follow-up. As a general statistical point, missing-data assumptions can affect estimates and their uncertainty, especially when reasons for missingness are related to outcomes. Without the relevant counts and methods, the impact cannot be assessed for this study.
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 |
|---|---|---|---|
| Pemetrexed | 13 | 33 | 39.4% |
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 |
|---|---|
| Pemetrexed | 33 of 33 |
Most frequent serious adverse event terms
| Event term | Organ system | Pemetrexed |
|---|---|---|
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | 2 of 33 |
| Anaemia | Blood and lymphatic system disorders | 1 of 33 |
| Leukopenia | Blood and lymphatic system disorders | 1 of 33 |
| Neutropenia | Blood and lymphatic system disorders | 1 of 33 |
| Cardiac tamponade | Cardiac disorders | 1 of 33 |
| Pericardial effusion | Cardiac disorders | 1 of 33 |
| Pyrexia | General disorders | 1 of 33 |
| Cellulitis | Infections and infestations | 1 of 33 |
| Infection | Infections and infestations | 1 of 33 |
| Sepsis | Infections and infestations | 1 of 33 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Pemetrexed |
|---|---|---|
| Fatigue | General disorders | 11 of 33 |
| Pruritus | Skin and subcutaneous tissue disorders | 11 of 33 |
| Alanine aminotransferase increased | Investigations | 10 of 33 |
| Cough | Respiratory, thoracic and mediastinal disorders | 10 of 33 |
| Vomiting | Gastrointestinal disorders | 9 of 33 |
| Chest pain | General disorders | 9 of 33 |
| Anorexia | Metabolism and nutrition disorders | 9 of 33 |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | 9 of 33 |
| Nausea | Gastrointestinal disorders | 8 of 33 |
| Pyrexia | General disorders | 8 of 33 |
Serious events with the highest arm-level rate: pneumonitis (6.1%), anaemia (3.0%), leukopenia (3.0%), neutropenia (3.0%), cardiac tamponade (3.0%).
The registry lists 33 participants at risk for serious adverse events and 13 participants affected. For other adverse events, it lists 33 participants at risk and 33 affected. These are participant counts in separate safety categories; they should not be added together, because the same participant may appear in more than one category. The registry does not clarify the extent of overlap.
The serious adverse event terms listed include pneumonitis at 6.1%, and anaemia, leukopenia, neutropenia, cardiac tamponade, and pericardial effusion at 3.0% each. These percentages are reported as written in the registry. They describe listed events, not a comparison with an untreated group or another treatment. The record does not establish that the intervention caused any specific event.
The deaths entry is not reported in the registry record. The safety counts are descriptive and should be read alongside the study’s single-arm design and the absence of a comparator. The registry does not provide enough detail to determine whether an event occurred once or more than once in a participant, or to assess individual event timing and clinical course.
12. What the Result Does, and Does Not, Mean
The registry record establishes that the study was designed to describe response and safety in participants with advanced non-small cell lung cancer after prior chemotherapy who received pemetrexed. It defines the primary response outcome using RECIST, gives the intended assessment schedule, and specifies how Unknown response classifications were handled. It also reports participant counts for serious and other adverse events and lists several serious adverse event terms with percentages.
The primary response result itself is not reported in the registry record. The response proportion, response counts, and 95% confidence interval are unavailable, so the observed response frequency and its precision cannot be described. The record also does not give numerical results for the named secondary outcomes. This means the endpoint definition should not be mistaken for evidence about the size or direction of an observed outcome.
The study’s single-arm, non-randomized design is central to interpretation. A response observed in participants receiving pemetrexed would be a descriptive finding in that group, not a randomized estimate of the difference between pemetrexed and another approach. The design does not provide a concurrent counterfactual comparison. Open-label conduct and unreported details of outcome assessment add further limits to the interpretation of clinical measurements.
The safety entries likewise describe counts and event terms in the study group. They are not comparative rates and do not establish causation. The record’s missing deaths entry and lack of detailed event-level information further constrain safety interpretation.
In general, statistical evidence is most informative when the estimate, its uncertainty, analysis population, and method are all available and considered together. Here, the registry supplies some definitions and descriptive safety information but not the primary efficacy estimate or its uncertainty. The appropriate interpretation is therefore limited to what the design and reported safety entries document, without extending those details to claims about comparative effects.
13. Why This Trial Matters Statistically
This study illustrates why a clearly defined endpoint and an available endpoint result are both needed for statistical interpretation. The registry explains that objective response rate combines complete and partial responses under RECIST, identifies the intended treated population, and states how Unknown classifications were handled. Those details make the intended measure understandable. Yet the response count, estimate, and confidence interval are not reported in the registry record, so readers cannot assess the magnitude or precision of the primary outcome.
It also demonstrates the distinction between describing a clinical outcome and estimating a treatment effect. A single-group study can document what happened among its participants, but without a concurrent comparator it cannot provide a randomized contrast. The non-randomized, open-label design therefore shapes what can be inferred regardless of how the response endpoint is defined.
The record also makes visible the importance of separating efficacy reporting from safety reporting. It includes counts for participants affected by serious and other adverse events, alongside selected serious event terms and percentages. Those entries convey descriptive information, but they do not replace efficacy results or establish causation. Missing deaths information and the absence of more detailed event descriptions limit interpretation further.
Finally, the study is a useful example of why statistical plans and analysis populations matter. The registry record does not report a detailed statistical analysis plan, methods for the confidence interval, handling of missingness for secondary outcomes, or a multiplicity approach. Without those details and the actual estimates, a reader cannot evaluate precision, robustness, or the intended inferential role of secondary outcomes. These are general considerations when reading clinical trial records, particularly when the design does not include a control group.
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 trial-specific limitation is the absence of the primary efficacy result. Although the registry defines objective response rate and specifies a 95% confidence interval, it does not report the response counts, estimated proportion, or interval. The number who received at least one dose is also not reported, so the stated primary analysis denominator cannot be confirmed from the record.
The single-group, non-randomized design provides no concurrent comparison. The study was also open-label, and the registry record does not describe additional safeguards for outcome assessment. As a result, the design limits comparative interpretation, while the available information does not allow a detailed assessment of potential assessment or reporting influences.
Important treatment and analysis details are not reported in the registry record. These include the exact pemetrexed dose and schedule, detailed eligibility criteria, baseline participant characteristics, the statistical method for the primary confidence interval, and analysis methods for secondary outcomes. The record also does not describe missing-data handling for those secondary outcomes, analysis populations beyond the primary population definition, or interim analyses. Numerical results for the secondary outcomes are not reported.
The safety information is limited to participant counts and selected serious adverse event terms. Deaths are not reported in the registry record, and detailed event-level timing, recurrence, overlap, and attribution information is not given. These limitations mean the posted safety counts are descriptive rather than a complete account of event experience. The registry’s stated enrollment of 33 should not be assumed to equal the analysis population for every outcome.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Chemotherapy for Patients With Non-Small Cell Lung Cancer — NCT00380718.
- Registry record last updated 2010-11-16; status last verified 2010-11.
- Results first posted 2009-10-06.
- 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
NCT00380718 is a completed, open-label, non-randomized, single-group Phase IV study of pemetrexed in Taiwanese patients with advanced non-small cell lung cancer after prior chemotherapy. Enrollment was 33. The study began in 2006-11 and reached primary completion in 2008-09.
The primary outcome was objective response rate, defined as the proportion with a best response of complete or partial response under RECIST. The registry specified a 95% confidence interval and counted Unknown classifications as nonresponders. The primary estimate and interval are not reported in the registry record. Secondary outcomes included disease control rate, overall survival, progression-free survival, duration of response, time to treatment failure, and time to tumor progression; their numerical results are not reported.
The registry lists 13 participants with serious adverse events among 33 at risk, and 33 with other adverse events among 33 at risk. Serious event terms and percentages are listed, while deaths are not reported in the registry record. The single-arm design permits descriptive interpretation but provides no randomized comparator.