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 Gemcitabine and Imatinib Mesylate in Treating Patients With Recurrent or Metastatic Non-Small Cell Lung Cancer (NCT00323362), a na phase 2 study. The primary outcome is Percentage of Patients Who Meet Critieria for Response.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00323362 |
| Phase | Phase 2 |
| Condition | Lung Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 17 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Rutgers, The State University of New Jersey |
| Results posted | Yes |
2. Clinical Question
Population
Patients with recurrent or metastatic non-small cell lung cancer. The registry record reports enrollment of 17 and says 14 subjects were evaluable for response; three were not assessed.
Intervention
Gemcitabine hydrochloride given with imatinib mesylate. The registry record identifies both as drug interventions.
Comparator
There was no separate comparator arm in this single-group study. Allocation is recorded as not applicable, so the study does not make a randomized comparison with another treatment or a control group.
Primary question
What proportion of patients met the registry's criteria for response within the stated two-year timeframe? Response was defined as complete response or partial response under RECIST. The registry record does not report the observed response result.
The study's stated purpose was to examine the combination in recurrent or metastatic non-small cell lung cancer. Its primary outcome was response, rather than a direct randomized comparison of the combination against another regimen. The listed secondary outcomes were time to progression and one-year survival. The registry record does not report results for those outcomes either.
This design can describe outcomes among the patients studied, but it cannot by itself distinguish an effect of treatment from other explanations for observed outcomes. Without a separate comparison group, any response rate would be a description of the study cohort, not an estimate of how outcomes differed from those under another treatment. In this case, even that cohort response result is not reported in the registry record.
3. Trial Design
This was a phase II study of gemcitabine hydrochloride and imatinib mesylate for recurrent or metastatic non-small cell lung cancer. Its registry model is single group, and allocation is recorded as not applicable. The record lists one combined-treatment group and no separate comparator arm. Masking is recorded as none, so the study was not masked according to the registry entry.
The registry reports enrollment of 17. For the primary response outcome, it states that 14 subjects were evaluable and three were not assessed. These response-evaluation counts describe the endpoint population; the registry record does not explain why some subjects were not assessed or report how those subjects were treated in any analysis.
The study started in 2006-04, and primary completion is recorded as 2008-10. Its status is terminated. The reason for termination, the timing of termination, and whether it changed the planned analyses are not reported in the registry record. The sponsor is Rutgers, The State University of New Jersey.
Time to progression and one-year survival are listed as secondary outcomes, alongside the primary response outcome. The registry indicates three outcome measures but no analyses. It therefore does not give enough analysis detail to establish how the outcomes were summarized, what statistical tests were planned, or whether any adjusted estimates were intended. Those details are not reported in the registry record.
The study design supports a descriptive account of a treated group, but not a randomized treatment contrast. Since there is no concurrent control group, comparisons with external populations would require information and assumptions not reported in the registry record. The design and the missing outcome results should guide how narrowly the study can 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 | Gemcitabine Hydrochloride and Imatinib Mesylate |
|---|---|
| STARTED | 17 |
| COMPLETED | 17 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Fourteen subjects were evaluable for response. Three subjects were not assessed.
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 | Gemcitabine Hydrochloride and Imatinib Mesylate |
|---|---|---|
| Participants analysed | 17 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 13 |
| Age, Categorical: >=65 years | Participants | 4 |
| Age, Continuous | years | 59.5 (SD 8.5) |
| Sex: Female, Male: Female | Participants | 10 |
| Sex: Female, Male: Male | Participants | 7 |
| Region of Enrollment: United States | participants | 17 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Patients Who Meet Critieria for Response, measured over 2 years.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | percentage of patients who responded |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the percentage of patients who meet criteria for response, assessed over a stated timeframe of two years. Response includes complete response or partial response under RECIST. A complete response means disappearance of all target lesions. A partial response means at least a 30% decrease in the sum of the longest diameters of target lesions, using the baseline sum as the reference.
The endpoint's RECIST definitions also distinguish progressive disease and stable disease. Progressive disease is defined as at least a 20% increase in the sum of the longest diameters, using the smallest sum recorded since treatment began as the reference, or the appearance of one or more new lesions. Stable disease is neither sufficient shrinkage for partial response nor sufficient increase for progressive disease, using the smallest sum since treatment began as the reference. These categories clarify which assessments count as response: complete and partial response, not stable disease.
Response is a categorical measure of tumor change, and a response proportion can describe how often the qualifying categories occurred in the assessed group. Such an endpoint is useful for summarizing tumor response in a clinical study, but it does not by itself describe how long a response lasts or establish a comparative treatment effect. The registry record does not report the observed number or percentage of responders, a confidence interval, or a statistical test. It also does not report the assessment schedule or further details about how response assessments were conducted.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive limitation is that the registry record defines a primary response outcome but does not report its observed result. It identifies 14 subjects as evaluable for response and three as not assessed, but gives no number of complete or partial responses and no response percentage. Consequently, the posted record does not support a numerical conclusion about the frequency of response in this study group.
The response definition is clear at the category level: complete response or partial response under RECIST counts as response. The listed criteria specify disappearance of all target lesions for complete response and at least a 30% decrease in the sum of the longest diameters of target lesions for partial response, relative to baseline. The registry also defines progressive disease and stable disease, but neither category meets the primary response definition. These rules tell readers how the endpoint is classified; they do not reveal how participants were classified in practice.
Even if a response result were available, a single-group design would limit what it could establish. A response proportion would describe the assessed study group. Without a concurrent control group, it would not measure a randomized difference from another treatment or from no treatment. Differences in patient characteristics, care, assessment, or other factors could not be separated from treatment effects using this design alone. The registry record does not identify an alternative comparison that would resolve those issues.
Precision also cannot be assessed from the posted response results because no estimate or uncertainty interval is reported. In general, a confidence interval expresses a range of values compatible with the data and statistical assumptions; a wider interval signals less precision. Here, there is no response estimate or interval to interpret. The number evaluable is reported, but it cannot be used to recover the missing response result.
The secondary outcomes, time to progression and one-year survival, are listed without results. The record does not report event counts, time-to-event estimates, confidence intervals, or analysis methods for them. It also indicates no analyses. A listed outcome is not itself evidence of a finding, and the absence of a posted result should not be read as either a favorable or an unfavorable result.
The safety record reports 10 subjects affected by serious adverse events and 17 affected by other adverse events in the combined-treatment group, with 17 at risk in each category. It also lists selected serious adverse-event terms with percentages. These are safety descriptions, not a randomized comparison, and they do not establish that the study drugs caused the events. The record's death field is not populated with a count; the listed term concerning death and disease progression should not be treated as a complete mortality result.
Overall, the registry record describes the study design, endpoint definitions, assessment population, and some safety counts, but not the primary or secondary outcome results. Interpretations about response, progression, survival, or comparative benefit are therefore not supported by the posted results.
9. Statistical Methods Explained
Statistical interpretation begins with the design. This study is recorded as single group, with allocation not applicable and no masking. That means the main outcome is not a contrast between randomized arms. A response proportion, if reported, would summarize the observed response categories among the assessed group. It would not by itself estimate a causal difference between treatments.
What does the primary outcome classify?
The primary outcome is response under RECIST, defined as complete response or partial response. The complete-response category requires disappearance of all target lesions. Partial response requires at least a 30% decrease in the sum of the longest diameters of target lesions relative to baseline. The criteria also describe progressive disease and stable disease, which help distinguish response from other observed states. These definitions standardize what counts as a response, but do not provide the study's observed classifications.
The registry describes the endpoint as a percentage of patients who responded and sets a two-year timeframe. It reports 14 subjects evaluable for response and three not assessed. It does not report how many met either response category, how assessments were scheduled, or the result of any statistical analysis. Thus, no response proportion or its precision can be interpreted from the record.
What would an uncertainty interval mean?
In general, a confidence interval is a statistical range intended to convey uncertainty around an estimate under a specified method and assumptions. For a response proportion, it would help readers judge how precisely the study estimates response in the population represented by the study. It would not correct the lack of a control group, remove bias from missing assessments, or establish that treatment caused the observed responses. The registry record does not report a response estimate or confidence interval, so there is no interval to assess.
Why does the single-group design matter?
A randomized comparison uses concurrent groups to estimate a difference while reducing some sources of imbalance through random assignment. This study has no such comparison. Its allocation is listed as not applicable, and its model is single group. As a result, the observed outcomes, if reported, would be descriptive of the people assessed under this study's conditions. A comparison with another study or population would involve differences in eligibility, care, follow-up, and measurement that are not resolved by the registry record.
Masking is recorded as none. In general, masking can reduce the risk that knowledge of treatment assignment affects assessments or other study processes. Its absence does not prove that an assessment was biased, but it means the record does not describe masking as a safeguard. The registry does not report further procedures that might bear on assessment consistency.
What can the secondary outcomes establish?
Time to progression and one-year survival are listed as secondary outcomes. Time-to-event outcomes generally concern the timing of an event and may depend on how follow-up, censoring, and event definitions are handled. In general, censoring occurs when a participant's event time is not observed during the available follow-up, so the analysis uses the information available up to that point. The record does not provide the event definitions, follow-up details, censoring rules, estimates, or analyses for these outcomes. No conclusion about progression or survival can be drawn from their listing alone.
The registry indicates three outcome measures and zero analyses. That does not say that no analysis was ever performed; it says the registry record does not contain analysis entries. The statistical plan, including any prespecified tests, adjustment approach, or interim analyses, is not reported in the registry record. It would be inappropriate to infer those procedures from the endpoint names or study phase.
How should planning illustrations be read?
Planning illustrations based on an observed effect are not results from this trial and are not observed power. The stated illustration assumptions use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect set as a fraction of the observed effect. The record does not give an observed response effect from which to judge those illustrations. More importantly, the assumptions describe a two-arm planning setup, while this trial is single group. Such illustrations therefore should not be mistaken for the study's design, an analysis of its data, or a basis for a causal comparison.
In general, sample-size planning asks how many participants a future study might need under specified assumptions about the outcome, effect, variability, error rates, and design. Changing those assumptions changes the planning result. A planning value is not an estimate of how likely the completed study was to detect an effect; that would be an observed-power interpretation, which is not appropriate to substitute for an outcome estimate and its uncertainty. The registry record does not report the trial's own sample-size justification or statistical planning procedure.
Why does over-dispersion matter in general?
For count outcomes, a simple statistical model may assume that variability follows a particular relationship to the expected count. Over-dispersion means that the observed variability is greater than that assumption allows. If ignored, uncertainty can be understated and statistical tests may appear more decisive than warranted. This is a general methods consideration, not a reported feature of this study. The registry does not provide a count-outcome analysis or dispersion estimate, so no conclusion about over-dispersion in this trial is possible.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary response outcome and two secondary outcomes: time to progression and one-year survival. It does not report a statistical analysis plan, testing hierarchy, multiplicity adjustment, or whether any outcomes were designated for formal hypothesis testing. Those details are not reported in the registry record. Because several outcomes are listed, readers should not assume that each was tested, that tests were adjusted for multiple comparisons, or that any one result was the sole basis for interpretation.
The record indicates three outcome measures but no analyses. It therefore contains definitions and outcome listings without corresponding analysis results. The reason no analyses are recorded is not reported. The absence of registry analysis entries does not establish that investigators did not analyze data outside the registry; it means those analyses cannot be evaluated from this record.
For missing response assessment, the record states that 14 subjects were evaluable and three were not assessed. It does not give reasons, timing, or outcome status for the subjects not assessed. It also does not describe imputation, sensitivity analyses, or a missing-data assumption. No method for incorporating the unassessed subjects into the primary outcome is reported in the registry record.
The record does not specify whether the primary analysis population was based on enrollment, evaluability, treatment receipt, or another rule. The phrase “evaluable for response” identifies the reported assessment group, but does not establish a full analysis-population definition. It is therefore not possible to assess whether exclusions from the response evaluation could have affected the result, especially since the response result itself is absent.
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 |
|---|---|---|---|
| Gemcitabine Hydrochloride and Imatinib Mesylate | 10 | 17 | 58.8% |
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 |
|---|---|
| Gemcitabine Hydrochloride and Imatinib Mesylate | 17 of 17 |
Most frequent serious adverse event terms
| Event term | Organ system | Gemcitabine Hydrochloride and Imatinib Mesylate |
|---|---|---|
| Death not associated with CTCAE term - Disease progression NOS | General disorders | 4 of 17 |
| Dyspnea (shortness of breath) | Respiratory, thoracic and mediastinal disorders | 3 of 17 |
| Pleural effusion (non-malignant) | Respiratory, thoracic and mediastinal disorders | 3 of 17 |
| Platelets | Blood and lymphatic system disorders | 2 of 17 |
| Infection with normal ANC or Grade 1 or 2 neutrophils - Skin (cellulitis) | Infections and infestations | 2 of 17 |
| Hemoglobin | Blood and lymphatic system disorders | 1 of 17 |
| Fatigue (asthenia, lethargy, malaise) | General disorders | 1 of 17 |
| Hemorrhage, pulmonary/upper respiratory - Nose | Vascular disorders | 1 of 17 |
| Other toxicity | General disorders | 1 of 17 |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | 1 of 17 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Gemcitabine Hydrochloride and Imatinib Mesylate |
|---|---|---|
| Leukocytes (total WBC) | Blood and lymphatic system disorders | 11 of 17 |
| Neutrophils/granulocytes (ANC/AGC) | Blood and lymphatic system disorders | 11 of 17 |
| Platelets | Blood and lymphatic system disorders | 9 of 17 |
| Dyspnea (shortness of breath) | Respiratory, thoracic and mediastinal disorders | 9 of 17 |
| Hemoglobin | Blood and lymphatic system disorders | 8 of 17 |
| Fatigue (asthenia, lethargy, malaise) | General disorders | 8 of 17 |
| Cough | Respiratory, thoracic and mediastinal disorders | 7 of 17 |
| Nausea | Gastrointestinal disorders | 5 of 17 |
| Pain - Bone | General disorders | 4 of 17 |
| Pleural effusion (non-malignant) | Respiratory, thoracic and mediastinal disorders | 3 of 17 |
Serious events with the highest arm-level rate: death not associated with ctcae term - disease progression nos (23.5%), dyspnea (shortness of breath) (17.6%), pleural effusion (non-malignant) (17.6%), platelets (11.8%), infection with normal anc or grade 1 or 2 neutrophils - skin (cellulitis) (11.8%).
The safety record lists 17 subjects at risk for serious adverse events and 10 affected in the combined gemcitabine hydrochloride and imatinib mesylate group. It also lists 17 at risk for other adverse events and 17 affected. These are separate safety summaries. The registry does not provide a randomized comparator, so the counts cannot be used to estimate a comparative safety difference.
The record lists selected serious adverse-event terms with percentages: “Death not associated with CTCAE term - Disease progression NOS” at 23.5%; “Dyspnea (shortness of breath)” and “Pleural effusion (non-malignant)” at 17.6% each; “Platelets” and “Infection with normal ANC or Grade 1 or 2 neutrophils - Skin (cellulitis)” at 11.8% each; and “Hemoglobin” at 5.9%. These are registry-listed terms and percentages, not evidence by themselves that either study drug caused an event.
The record does not give event-level narratives, timing, severity details for each term, or a complete interpretation of attribution. The death field is not populated with a count. The listed disease-progression term should not be read as a reported overall death count or as an adverse effect caused by treatment. Safety counts describe what the registry records; causal conclusions require more information than these summaries contain.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a phase II, single-group study evaluated gemcitabine hydrochloride with imatinib mesylate for recurrent or metastatic non-small cell lung cancer. It defines the primary outcome as response under RECIST, with complete response or partial response counting as response, and gives a two-year timeframe. It also lists time to progression and one-year survival as secondary outcomes.
What the record does not establish is the observed response result. Although it reports that 14 subjects were evaluable and three were not assessed, it gives no response count or percentage. It also gives no results for time to progression or one-year survival. Without those results, the record cannot support a conclusion about how often response occurred, how long participants remained progression-free, or survival at one year.
Even a reported response rate from this study would describe the study group rather than a treatment difference. The single-group design has no separate control arm, and allocation is recorded as not applicable. It cannot isolate the role of the combination from other influences on outcomes. The lack of masking is also recorded, although the registry does not describe further assessment procedures or establish that bias occurred.
The safety information records serious and other adverse-event counts and selected serious-event terms. Those counts should be read as descriptive registry data, not proof of cause. The absence of a populated death count and of detailed event information limits what can be inferred about mortality and safety patterns.
Accordingly, this record is informative about the study's intended question, endpoint definitions, design, and some safety reporting. It is not sufficient to judge the magnitude or precision of a treatment outcome, and it does not establish a comparative treatment effect. The reason the study was terminated is not reported in the registry record, so termination should not be interpreted as evidence of a particular outcome or safety concern.
13. Why This Trial Matters Statistically
This trial illustrates why endpoint definitions and outcome results need to be read separately. The registry gives detailed RECIST criteria for complete response, partial response, progressive disease, and stable disease. Those definitions tell readers which categories count toward the primary response outcome. But without the observed number of responders, the definition alone cannot answer the clinical question.
It also demonstrates the inferential limits of a single-group study. A response proportion can be useful as a description of the group studied, but a treatment effect is inherently comparative. With no separate control arm, outcomes cannot be contrasted under the same study conditions against another treatment or control. Recognizing this distinction prevents a descriptive result, if one were posted, from being interpreted as a randomized treatment benefit.
The response-evaluation counts raise a further statistical issue: the record distinguishes subjects evaluable for response from those not assessed, but does not explain why assessments are missing or how missing outcomes were handled. Missingness can affect interpretation if the people not assessed differ in relevant ways from those assessed. The available record does not show whether that concern applies, so it cannot be resolved by assuming that the evaluable group represents everyone enrolled.
Finally, the listing of secondary outcomes and safety terms does not replace analysis results. Time to progression and one-year survival require their own definitions and follow-up details, while adverse-event counts do not establish causation. The record contains useful design and measurement information, but its absent outcome analyses and results sharply limit what readers can conclude. These distinctions are central to transparent interpretation of clinical trial records.
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.
Several limitations are specific to the registry record for this study. It is marked terminated, but the reason for termination and its implications are not reported in the registry record. The record also does not describe whether termination affected follow-up, the planned sample, or the outcome analyses. No explanation should be inferred from the status alone.
The enrollment is 17, and the registry reports 14 subjects evaluable for response, with three not assessed. The reasons for non-assessment, the characteristics of those not assessed, and the handling of their outcomes are not reported. This leaves uncertainty about the response-assessment population. Since the response result itself is absent, the extent to which missing assessments might have influenced the estimate cannot be evaluated.
The trial is single group, has no masking, and has no separate comparator arm. Those features limit comparative interpretation and may leave outcome assessment open to influences that a control group or masking could help address. The record does not report procedures that would permit further evaluation of those concerns.
The primary response outcome has an explicit RECIST definition, but the record does not give an observed response count, percentage, or uncertainty interval. The secondary outcomes are named without results, event definitions, or analysis details. The registry contains no analysis entries, and the statistical plan, multiplicity approach, sample-size justification, and interim-analysis procedures are not reported in the registry record.
Safety reporting includes counts and selected serious-event terms, but the registry does not provide enough detail to establish causation or give a complete account of mortality. These limits concern what can be learned from the record; they should not be converted into claims about the study's unreported findings.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Gemcitabine and Imatinib Mesylate in Treating Patients With Recurrent or Metastatic Non-Small Cell Lung Cancer — NCT00323362.
- Registry record last updated 2014-05-22; status last verified 2014-04.
- Results first posted 2014-05-22.
- 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
The registry record describes a phase II, single-group study of gemcitabine hydrochloride and imatinib mesylate in recurrent or metastatic non-small cell lung cancer. Enrollment is 17; the record reports 14 subjects evaluable for response and three not assessed. The study started in 2006-04, primary completion is recorded as 2008-10, and status is terminated.
The primary outcome is response under RECIST within a two-year timeframe. Time to progression and one-year survival are listed as secondary outcomes. The registry record does not report observed results for these outcomes and contains no analysis entries. Safety information includes serious and other adverse-event counts and selected serious-event terms. The reason for termination, missing-response handling, statistical plan, and analysis details are not reported in the registry record.