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 Pemetrexed as the First Treatment in Advanced or Metastatic Breast Cancer (NCT00106002), a non-randomized phase 2 study. The primary outcome is Overall Tumor Response.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00106002 |
| Phase | Phase 2 |
| Condition | Breast Cancer, Breast Neoplasms, Male, Carcinoma, Ductal |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 37 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Eli Lilly and Company |
| Results posted | Yes |
2. Clinical Question
The study asked whether pemetrexed could be used as first-line chemotherapy for people with advanced or metastatic breast cancer. Its stated purposes also included assessing side effects, determining how much pemetrexed should be given, and collecting DNA for future research; DNA collection was optional.
Population
The study concerned patients with advanced or metastatic breast cancer. The registry lists breast cancer, male breast neoplasms, and ductal carcinoma among the conditions. Detailed eligibility criteria and a description of the analyzed population are not reported in the registry record.
Intervention
The intervention was pemetrexed. The registry identifies the drug but does not report a dose, schedule, dose-adjustment rules, or other treatment details.
Comparator
There was no separate comparator group: the study is described as a single-group, non-randomized trial. It therefore does not make a randomized, like-for-like comparison with another treatment or with usual care.
Primary question
The primary outcome was the number of participants with an overall tumor response. Response was assessed using RECIST criteria, which classify whether disease improves, remains stable, or progresses. The observed response count and its analysis are not reported in the registry record.
This design can describe outcomes among participants treated in the study, but without a concurrent control group it cannot separate treatment-associated outcomes from other explanations, such as the course of the disease or differences among participants. The record does not report enough information to determine how closely the enrolled participants matched a broader patient population.
3. Trial Design
This was a phase II study sponsored by Eli Lilly and Company. The registry describes it as non-randomized, unmasked, and single-group. Enrollment was 37. The study status is completed; its recorded start was 2005-04 and its primary completion was 2007-12.
All participants were assigned to the pemetrexed group, with no randomized control arm described. The registry provides no arm-specific treatment description beyond the name of the intervention. It does not report the dose, administration schedule, treatment duration rules, or any dose changes. Although determining how much pemetrexed should be given was among the study’s stated purposes, the dose-finding approach and resulting dose information are not reported in the registry record.
The primary outcome was assessed every 3 cycles, approximately 6-7 weeks, or until a participant had disease progression. The record describes the outcome as the best response from the start of treatment until progression or recurrence, assessed using RECIST. It does not state how many assessments each participant received or how the best response was assigned when assessments differed.
Secondary outcomes listed in the registry were toxicity, duration of tumor response, progression-free survival time, and overall survival time. The toxicity outcome refers to adverse events graded 3 and 4 under the Common Terminology Criteria for Adverse Events and present in >5% of participants. Definitions, assessment schedules, analysis details, and results for these outcomes are not reported in the registry record.
The record does not report a randomized allocation process, masking procedures beyond the designation of no masking, or a control treatment. It also does not report the eligibility criteria, participant flow, treatment exposure, or reasons participants may have stopped treatment or left follow-up. These omissions limit assessment of how participants entered and remained in the study and of how the observed outcomes should be interpreted.
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 | Pemetrexed |
|---|---|
| STARTED | 37 |
| COMPLETED | 35 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | Pemetrexed |
|---|---|
| Did not take any study drug | 1 |
| Ineligible after Registration | 1 |
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 | 37 | |
| Age Continuous | years | 61.4 (39 to 82) |
| Sex: Female, Male: Female | Participants | 37 |
| Sex: Female, Male: Male | Participants | 0 |
| Region of Enrollment: United States | participants | 37 |
| Eastern Cooperative Oncology Group Scale Performance Status: 0 - Fully Active | participants | 19 |
| Eastern Cooperative Oncology Group Scale Performance Status: 1 - Ambulatory, Restricted Strenuous Activity | participants | 18 |
| Eastern Cooperative Oncology Group Scale Performance Status: 2 - Ambulatory, No Work Activities | participants | 0 |
| Eastern Cooperative Oncology Group Scale Performance Status: 3 - Partially Confined to Bed, Limited Self Care | participants | 0 |
| Eastern Cooperative Oncology Group Scale Performance Status: 4 - Completely Disabled | participants | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Tumor Response, measured over every 3 cycles (approximately 6-7 weeks) or until patient has disease progression.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was overall tumor response, recorded as a number of participants. The registry describes response as the best response observed from treatment start until disease progression or recurrence. Assessments were scheduled every 3 cycles, approximately 6-7 weeks, or until progression. RECIST criteria were used to classify whether participants improved, remained stable, or had progression.
In general, a response endpoint gives a categorical summary of tumor status rather than a continuous measurement. Reporting the number of participants who respond can make the endpoint straightforward to interpret, provided the response definition, assessment process, and analysis population are clear. The registry record does not give the response count, a denominator for the analysis, a response estimate, or a confidence interval. It also does not describe the specific response categories counted as an overall response or the handling of participants without an evaluable assessment. Those details are not reported in the registry record.
Response is only one way to characterize a cancer outcome. The registry separately lists duration of tumor response, progression-free survival time, and overall survival time as secondary outcomes. These address different questions: how long a response lasts, how long participants remain without progression, and how long they live. No results for those outcomes are reported in the registry record. A response result alone would not provide the time-to-event information represented by those secondary outcomes.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The available record identifies the study design and intended outcome but does not report the primary outcome result. In particular, it gives no number of participants with an overall tumor response, no analysis denominator, and no estimate of response. It also gives no confidence interval or statistical test for that outcome. The registry record therefore does not allow a reader to assess the observed response frequency or its precision.
The trial enrolled 37 participants in a single-group, non-randomized study. That enrollment figure describes the study, not the number whose response was evaluable or included in an analysis. The analyzed population and participant flow are not reported in the registry record. Without those details, an outcome count could not be interpreted against a known analysis denominator even if one were available.
In general, a confidence interval describes a range of values compatible with the data and the statistical method used. A narrow interval indicates greater precision than a wide interval, while neither interval width nor an estimate alone establishes that an effect is caused by treatment. No confidence interval is reported for the primary endpoint, so its precision cannot be assessed from the registry record.
The single-group design is important to interpretation. In general, an outcome observed after treatment in one group does not by itself establish what would have happened to the same participants under a different treatment or without the study treatment. Differences in participant characteristics, disease course, assessment timing, or other care may contribute to observed outcomes. A concurrent randomized control group can help address these issues through like-for-like comparison; this study is not described as having such a group.
The registry lists serious adverse events affecting 14 participants and other adverse events affecting 34 participants in the pemetrexed group. The denominators and analysis populations for these counts are not reported in the registry record, so the counts should not be converted into rates. The registry also lists nausea at 8.3%, and anaemia, vomiting, cellulitis, dizziness, and mental status changes each at 5.6%, among its top serious adverse-event terms. These entries describe the posted safety data; they do not establish how risks compare with another treatment.
Several clinically relevant secondary outcomes are named, including progression-free survival time and overall survival time, but their results are not reported in the registry record. The study’s stated purposes included determining a dose, yet the dose information is also absent. As a result, the record supports a description of the study’s intent and structure, but not a conclusion about the magnitude, precision, or comparative meaning of its clinical outcomes.
9. Statistical Methods Explained
The registry describes a binary primary outcome in terms of participants with an event, expressed as counts. Here, the event of interest is an overall tumor response as defined by the study’s RECIST assessments. A count is interpretable only alongside its denominator: the number of participants included in the relevant analysis. The denominator and observed response count are not reported in the registry record.
What does a response count capture?
In general, a binary endpoint places each participant into one of two analysis categories, such as having or not having the event being studied. For an overall response endpoint, the exact rule for combining response categories determines which participants count as responders. The registry describes improvement, stability, and progression, but does not specify the detailed rule used to derive the overall-response count. That rule is not reported in the registry record.
Best response uses information gathered over a period rather than relying on a single assessment. In general, this can capture the most favorable recorded status during the specified assessment period. Interpretation depends on the assessment schedule, the criteria used, the treatment and follow-up period, and how missing or unevaluable assessments are handled. The record gives the schedule as every 3 cycles, approximately 6-7 weeks, or until disease progression, and names RECIST. Rules for missing assessments and the specific selection of the best response are not reported in the registry record.
How should uncertainty be considered?
In general, an estimate based on a limited number of participants has sampling uncertainty: another study of similar participants could yield a different result. A confidence interval communicates uncertainty around an estimate under a stated method and assumptions. It does not account automatically for every possible bias, such as incomplete follow-up or differences between a study population and the population of interest. No primary-outcome estimate or confidence interval is reported in the registry record, so the endpoint’s statistical uncertainty cannot be evaluated.
A statistical test, when used, evaluates data against a specified null hypothesis under the test’s assumptions. A p-value is not an estimate of effect size and does not measure the probability that a treatment works. No primary-outcome test or p-value is reported in the registry record. The trial’s statistical analysis plan, including any model or test, is not reported in the registry record.
What does the planning illustration represent?
In general, a sample-size calculation depends on a target effect, a significance level, a desired power, an allocation plan, and assumptions about the outcome and analysis. The planning illustration associated with the registry uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It frames the effect to detect as a stated fraction of the observed effect. For time-to-event outcomes, its illustration concerns total events; for other outcomes, it concerns participants per arm.
These are planning assumptions, not a description of the actual trial allocation. This trial is recorded as a non-randomized, single-group study, not a two-arm randomized comparison. The illustration is not observed power and should not be interpreted as evidence that the trial had a particular probability of detecting an effect. The underlying observed effect, the illustrated sample-size values, and the trial’s own sample-size justification are not reported in the registry record.
Why does the design affect statistical interpretation?
In general, randomization helps create comparable groups by assigning participants through chance, reducing systematic differences in expectation. A single-group study has no concurrent group to provide a direct estimate of outcomes under an alternative treatment or control condition. Its findings can describe what happened among participants in the study, but an observed outcome cannot by itself isolate the effect of the intervention from other influences.
The registry lists several time-to-event outcomes. In general, these outcomes concern the time until an event, and their analysis needs information about event times and follow-up, including how participants who have not had an event by the end of observation are handled. The registry names duration of tumor response, progression-free survival time, and overall survival time, but does not report their event definitions, censoring rules, estimates, or analyses. No conclusions about those outcomes can be derived from their names alone.
For safety, the registry gives counts of participants with serious and other adverse events and lists selected serious-event terms with percentages. In general, safety summaries are most interpretable when the number exposed, the observation period, event definitions, and handling of repeat events are clear. The denominators and detailed safety-analysis rules are not reported in the registry record. The posted counts and percentages can therefore be described as recorded, but cannot be expanded into unreported rates or comparative estimates.
10. Multiplicity, Missing Data and Analysis Populations
The registry names a primary outcome and several secondary outcomes. When multiple outcomes are tested, in general, the chance of at least one apparently notable finding can increase if each is assessed separately without accounting for multiplicity. Statistical plans may designate a primary endpoint, define a testing hierarchy, or use another method to address multiple comparisons. The registry record does not report a multiplicity strategy, a testing hierarchy, or any outcome-level statistical tests.
The registry describes overall tumor response as the primary outcome and lists toxicity, duration of tumor response, progression-free survival time, and overall survival time as secondary outcomes. It does not report whether these outcomes were all analyzed, whether any were exploratory, or how the analyses were prioritized. The registry lists five outcome measures, but analysis details and results are not reported in the registry record.
Missing data can affect both the number of participants included in an analysis and the interpretation of its results. In general, an analysis should specify what counts as missing or unevaluable, whether follow-up ended early, and how such observations are handled. The record does not report missing-data methods, assessment completeness, reasons for missing observations, or the number included in each analysis.
Analysis populations also matter. A trial may define participants according to enrollment, treatment received, or availability of outcome measurements, but the chosen population and its rules should be stated. The registry record does not report an analysis population for the primary or secondary outcomes. Enrollment was 37, but that number should not be assumed to be the denominator for any outcome analysis.
Interim analyses and stopping rules can also affect interpretation when planned or conducted. Whether this study used interim analyses or prespecified stopping rules is not reported in the registry record. No statistical analysis plan is reported in the registry record.
11. What the Result Does, and Does Not, Mean
The record establishes that a completed phase II study evaluated pemetrexed in a single group of participants with advanced or metastatic breast cancer and designated overall tumor response as its primary outcome. It also identifies several secondary outcomes and gives selected safety summaries. These details describe the study’s purpose and structure, but the primary response result is not reported in the registry record.
Without the response count, analysis denominator, and an uncertainty estimate, the record does not show how often participants had an overall tumor response or how precisely that result was estimated. Without a comparator group, any outcome observed in the pemetrexed group would not constitute a randomized comparison against another treatment. The study design cannot establish from its own single-group outcomes what would have happened to comparable participants under an alternative approach.
The safety entries are also limited in what they can establish. The record reports 14 participants with serious adverse events and 34 with other adverse events, but does not report the relevant denominators. It lists selected serious-event terms with percentages, including nausea at 8.3% and anaemia, vomiting, cellulitis, dizziness, and mental status changes at 5.6% each. These entries do not provide a comparison with another group, and the registry does not report enough detail to derive further rates.
The named secondary outcomes could address duration of response, progression-free survival, overall survival, and toxicity, but their results are not reported in the registry record. Likewise, although determining how much pemetrexed should be given was among the stated purposes, the dose and dose-finding results are not reported. Conclusions about those outcomes or the dose cannot be drawn from the available record.
In general, statistical significance, if reported, would not by itself establish clinical importance; the estimated effect, its uncertainty, the design, and the population would all matter. No primary-outcome estimate or statistical test is reported in the registry record. The appropriate interpretation is therefore limited to the stated study design, outcomes, and posted safety entries, rather than a claim about comparative benefit or harm.
12. Why This Trial Matters Statistically
This trial illustrates an important distinction between describing an outcome and estimating a comparative treatment effect. A single-group study can record tumor response and adverse events among participants who receive an intervention. Without a concurrent comparison group, however, it cannot directly estimate how those outcomes differ from what would occur under another treatment or control condition.
The registry identifies a clinically recognizable primary endpoint: best overall tumor response assessed with RECIST over treatment and follow-up. It also lists duration of response, progression-free survival, and overall survival. These outcomes represent different statistical questions. A response count summarizes a categorical outcome, whereas time-to-event outcomes require information about event timing and follow-up. The registry’s descriptions make clear that these measures are not interchangeable.
The record also demonstrates why a count requires context. Enrollment was 37, but the number analyzed for each outcome is not reported in the registry record. A count without a clear analysis denominator does not establish an event frequency, and a frequency without an uncertainty measure does not show how precisely it is estimated. These distinctions are central when interpreting small clinical studies.
Safety summaries similarly depend on denominators and analysis rules. The record lists affected-participant counts and percentages for selected serious-event terms, but does not report the denominators or full methods behind those summaries. Reporting the entries as recorded, without calculating additional rates, preserves the distinction between posted data and quantities that cannot be established from the record.
Finally, the planning illustration uses assumptions for a hypothetical two-arm allocation, while the study itself is described as a single-group, non-randomized trial. This reinforces a general methodological point: planning calculations depend on the design and assumptions they represent. They are not evidence of observed power, and they do not substitute for a clearly reported trial analysis. The registry record does not report the trial’s own sample-size justification or statistical analysis plan.
13. 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 direct limitation for interpreting efficacy is that the registry record does not report the primary endpoint result. There is no response count, analysis denominator, estimate, confidence interval, or test. Consequently, the statistical size and precision of the primary outcome cannot be assessed from this record.
The study is recorded as non-randomized and single-group, with no comparator arm described. This limits comparative interpretation: outcomes observed in the pemetrexed group cannot be directly contrasted with outcomes under another treatment or control within the study. The record also does not describe a comparator-based estimate or adjustment for differences in participant characteristics.
Important details about the study population and treatment are absent. Detailed eligibility criteria, the analyzed population, treatment dose and schedule, dose modifications, and treatment exposure are not reported in the registry record. Although the stated purposes included determining how much pemetrexed should be given, the dose-finding method and results are not reported.
Information needed to evaluate outcome ascertainment is also limited. The primary outcome description names RECIST and the assessment timing, but the detailed rule for deriving overall response, assessment completeness, and handling of unevaluable or missing assessments are not reported in the registry record. Participant flow, follow-up completeness, and reasons for withdrawal or discontinuation are also not reported.
The registry lists secondary outcomes and selected adverse-event summaries but does not report their analysis methods or results in full. Safety denominators and detailed analysis rules are not reported. Multiplicity handling, interim analyses, stopping rules, and the statistical analysis plan are not reported in the registry record. These gaps restrict assessment of both the reliability of individual outcome summaries and the overall evidence represented by the study.
14. Related Tutorials
15. Related Statistical Calculators
16. Sources
- ClinicalTrials.gov: Pemetrexed as the First Treatment in Advanced or Metastatic Breast Cancer — NCT00106002.
- Registry record last updated 2009-06-01; status last verified 2009-04.
- Results first posted 2009-06-01.
- 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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17. Record Summary
NCT00106002 is a completed phase II study titled “Pemetrexed as the First Treatment in Advanced or Metastatic Breast Cancer,” sponsored by Eli Lilly and Company. It is recorded as non-randomized, unmasked, and single-group, with enrollment of 37. The recorded start was 2005-04 and primary completion was 2007-12.
The intervention was pemetrexed. Overall tumor response was the primary outcome, assessed using RECIST every 3 cycles, approximately 6-7 weeks, or until disease progression. Listed secondary outcomes included toxicity, duration of tumor response, progression-free survival time, and overall survival time.
The registry reports selected adverse-event summaries but does not report the primary response result, detailed outcome analyses, or the statistical analysis plan. It also does not report the treatment dose, analysis populations, missing-data methods, or the trial’s sample-size justification.