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 as First-Line Therapy in Patients With Locally Adv. or Metastatic Pancreatic Cancer (NCT00161213), a na phase 2 study. The primary outcome is Progression-free Survival.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00161213 |
| Phase | Phase 2 |
| Condition | Pancreatic Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 44 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Medicine and Dentistry of New Jersey |
| Results posted | Yes |
2. Clinical Question
Population
Patients with locally advanced or metastatic pancreatic cancer receiving first-line treatment. The registry reports that 44 patients were enrolled; one withdrew consent before treatment, and one was later found to have ampullary cancer and was evaluable for toxicity but not response.
Intervention
Gemcitabine hydrochloride together with imatinib mesylate, listed in the record as gemcitabine and imatinib.
Comparator
No concurrent comparator is reported. The study is described as a single-group phase II trial, with allocation listed as NA.
Primary question
What was the progression-free survival for patients receiving this first-line combination, with progression defined using RECIST v1.0? The registry gives the endpoint definition and a four-year timeframe, but the median and its 95% confidence interval are not reported in the registry record.
The study record frames the clinical question around the combination of two drugs in first-line treatment, rather than around a randomized comparison between treatments. Its stated primary endpoint is progression-free survival, a time-to-event outcome describing the time until disease progression or death according to the endpoint definition and analysis used. The record also lists response rate, one-year survival rate, and overall survival as secondary outcomes.
The record does not provide a primary-endpoint estimate, a confidence interval, or a statistical test result. Consequently, it is not possible to describe the observed duration of progression-free survival or its precision from the information reported. Nor can the results establish how outcomes would compare with another treatment: no control group is reported. The question the record can address is therefore limited to the outcomes observed and reported for this single group.
3. Trial Design
This is a completed phase II study of gemcitabine and imatinib mesylate as first-line therapy for pancreatic cancer. The record describes the study model as single group, lists allocation as NA, and states that masking was none. The listed enrollment is 44. The study began in 2005-09 and had primary completion in 2010-10.
The treatment group is named Gemcitabine and Imatinib. The interventions are gemcitabine hydrochloride and imatinib mesylate. No separate control arm or randomized treatment assignment is reported. The absence of a concurrent comparator is important for interpreting outcomes: a change over time or an observed outcome in this group cannot, by itself, identify what would have happened to comparable patients under another treatment.
The primary endpoint is progression-free survival in months, with a median and 95% confidence interval specified as the intended summary. Its stated timeframe is four years. Progression is defined using Response Evaluation Criteria in Solid Tumors Criteria (RECIST v1.0): a 20% increase in the sum of the longest diameter of target lesions, a measurable increase in a non-target lesion, or the appearance of new lesions.
The record reports that one patient withdrew consent before starting treatment. It also reports that one patient was determined after initiating treatment to have ampullary cancer; that patient was evaluable for toxicity but not response. These details matter because the population enrolled, treated, and evaluated for a particular outcome may differ. The registry record does not report further details of the analysis populations or how each outcome handled these participants.
The record lists response rate, one-year survival rate, and overall survival as secondary outcomes. It does not report the results for these outcomes in the information available for this analysis. The protocol’s statistical plan, sample-size justification, and details of follow-up or censoring 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 | Gemcitabine and Imatinib |
|---|---|
| STARTED | 44 |
| COMPLETED | 42 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | Gemcitabine and Imatinib |
|---|---|
| Withdrawal by Subject | 1 |
| Evaluable for toxicity but not response | 1 |
Analysis population for the primary outcome, as defined in the registry: A total of 44 patients were enrolled. One patient withdrew consent before starting treatment. One patient was determined, after initiating treatment, to have an ampullary cancer and was evaluable for toxicity but not for response.
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 and Imatinib |
|---|---|---|
| Participants analysed | 44 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 24 |
| Age, Categorical: >=65 years | Participants | 20 |
| Age, Continuous | years | 63 (SD 10) |
| Sex: Female, Male: Female | Participants | 22 |
| Sex: Female, Male: Male | Participants | 22 |
| Region of Enrollment: United States | participants | 44 |
6. Primary Endpoint
The registry lists the primary outcome as Progression-free Survival, measured over 4 years.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 95% Confidence Interval |
| Unit | months |
| Endpoint classification | Time-to-Event Analysis |
Progression-free survival is a time-to-event endpoint. It concerns the time from a defined starting point until disease progression or death, as specified by the study’s analysis. The record reports the endpoint in months and identifies the median and 95% confidence interval as its summary. The starting point used in the analysis and the handling of censoring are not reported in the registry record.
Progression is defined using RECIST v1.0. The stated criteria include a 20% increase in the sum of the longest diameter of target lesions, a measurable increase in a non-target lesion, or the appearance of new lesions. This makes the endpoint dependent on how tumor measurements and new lesions are assessed, as well as on the timing and completeness of assessments. The registry record does not provide further assessment procedures.
In general, progression-free survival is used to summarize how long participants remain alive without a recorded progression event. It can capture disease progression before an overall-survival event occurs, but it is not interchangeable with overall survival or response rate. Its interpretation depends on the event definition, follow-up, and analysis methods.
Although the record specifies a median and a 95% confidence interval, it does not report either value. It therefore does not allow a reader to assess the observed progression-free survival or the precision of its estimate. The endpoint definition and four-year timeframe are reported; the primary result is not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a single-group phase II study and identifies progression-free survival as its primary endpoint. It does not report the primary endpoint’s median or 95% confidence interval. Without those results, no account of the observed progression-free survival or its statistical precision can be made from the record. The outcome results listed for response rate, one-year survival rate, and overall survival are also not reported in the registry record.
The study enrolled 44 patients. One withdrew consent before starting treatment, while one was later found to have ampullary cancer and was evaluable for toxicity but not response. These statements describe enrollment and outcome eligibility, but they do not establish the denominator used for the primary endpoint. The analysis population and endpoint-specific denominators are not reported in the registry record.
There is no concurrent comparator: the model is single group, allocation is listed as NA, and masking is none. Thus, even if an outcome estimate were available, it would describe this study group and would not by itself provide a randomized estimate of the difference between the combination and another treatment. Differences in patient characteristics, care, or follow-up across studies could also affect comparisons with external results; the record does not report an external comparison.
The record specifies RECIST v1.0 criteria for progression, including a 20% increase in the sum of the longest diameter of target lesions, a measurable increase in a non-target lesion, or new lesions. In general, time-to-event results also depend on follow-up and on how participants without a recorded event are handled. The registry record does not report those analysis details, so readers cannot assess their contribution to the endpoint estimate.
The record lists serious adverse event figures for the Gemcitabine and Imatinib group: 6 affected among 43 at risk. It lists 39 affected among 43 at risk for other adverse events. These categories should be kept separate, and the figures do not establish causation. The record also lists serious adverse event terms with percentages, but it does not provide a full account of event timing, duration, or attribution in the information. Deaths are not reported in the adverse-event summary.
These limitations do not show that an outcome was favorable or unfavorable; they limit what can be concluded from the posted record. The record documents the study design, endpoint definition, enrollment, and selected safety figures, but does not report the efficacy results or enough analysis detail to quantify uncertainty beyond the stated endpoint plan.
9. Statistical Methods Explained
Methods for interpreting a trial depend on the design, endpoint, analysis population, and details of how outcomes are recorded. The registry record identifies a phase II, single-group study with no masking and no randomized allocation. It names progression-free survival as the primary endpoint and specifies a median with a 95% confidence interval. The actual endpoint estimate and interval are not reported in the registry record.
What does a single-group design mean?
In a single-group study, outcomes are observed in participants receiving the study treatment without a concurrent randomized control group. The resulting description can characterize what was observed in the enrolled group, but it cannot isolate a treatment effect from other possible explanations. For example, a comparison with outcomes from another study would not have the protection of randomization; differences between the groups or their care could matter. This is a general design principle, not a claim about what happened in this study.
Here, the record lists one treatment group, Gemcitabine and Imatinib. Allocation is NA, and masking is none. No like-for-like control comparison is reported. Accordingly, any interpretation should stay within the evidence that a single group can provide. The registry record does not report an adjusted comparative effect or sponsor-reported hazard ratio.
How should the median and confidence interval be understood?
For a time-to-event outcome, the median is a point on the estimated event-time distribution: in general, it marks the time by which half of the population represented by that estimate has experienced the event, subject to the analysis method and censoring. A 95% confidence interval expresses uncertainty around the estimated median under the statistical method used. It is not a guarantee about the range of individual participants’ outcomes. These are general explanations; the record does not state the method used to estimate the median or interval, and it does not report their values.
Time-to-event analyses commonly account for participants whose event has not been observed by their last follow-up through censoring. In general, censoring allows partial follow-up information to contribute, but interpretation relies on assumptions about how follow-up ends. The registry record does not report its censoring rules, follow-up distribution, or the number of endpoint events. Those details cannot be inferred from the stated timeframe.
What does the progression definition contribute?
The record uses RECIST v1.0 and defines progression through specified changes in target lesions, non-target lesions, or new lesions. A fixed operational definition supports consistent classification of events. In general, however, application also depends on assessment schedules, measurement procedures, and review practices. Such details are not reported in the registry record. The stated four-year timeframe does not itself describe how often assessments took place or how long each participant was followed.
What can the posted safety counts establish?
The registry lists 6 affected among 43 at risk for serious adverse events, and 39 affected among 43 at risk for other adverse events. These are descriptive figures for the listed group, not a randomized comparison. The record separately lists selected serious adverse event terms with percentages. Those percentages should be read as registry-reported values, not as a basis for deriving unreported counts. In general, adverse-event summaries depend on definitions, collection periods, coding, and whether events are counted by participants or occurrences; the full details are not reported in the registry record.
What about statistical significance and planning?
The registry record does not report a primary-endpoint test result, p-value, hazard ratio, or confidence interval value. No claim about statistical significance or precision can therefore be made. It also does not report the protocol’s sample-size justification, planned interim analyses, or full statistical analysis plan. A planning illustration based on an observed effect, if considered separately, is not observed power and cannot substitute for trial design documentation or missing endpoint results. The record’s methods information is insufficient to reconstruct a statistical analysis.
10. Multiplicity, Missing Data and Analysis Populations
The record lists progression-free survival as the primary outcome and lists response rate, one-year survival rate, and overall survival as secondary outcomes. It does not report results for these outcomes in the information available for this analysis. Nor does it report a multiplicity strategy, such as how inference across multiple outcomes was planned or whether any adjustment was used. The protocol’s statistical plan is not reported in the registry record.
Multiplicity matters in general because examining several outcomes can create more opportunities for apparently notable findings, especially when many tests are considered. A prespecified hierarchy or adjustment can help define how results should be interpreted. Without the analysis plan and outcome results, it is not possible to determine whether or how multiplicity was handled in this study. This is a limitation of the reported information, not evidence that a particular procedure was or was not performed.
The record states that 44 patients were enrolled. One patient withdrew consent before starting treatment. Another was identified after treatment initiation as having ampullary cancer and was evaluable for toxicity but not response. The record does not report the analysis populations for progression-free survival, overall survival, or the other outcomes, nor does it describe how missing outcome information was handled.
In general, missing follow-up can affect time-to-event estimation if the reasons for loss to follow-up are related to outcomes. The assumptions and methods used to address missing data or censoring are not reported in the registry record. The endpoint-specific denominators, numbers of events, and completeness of follow-up are also not reported. Consequently, the enrollment count and the listed eligibility details should not be treated as a full description of the analyzed population for every endpoint.
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 and Imatinib | 6 | 43 | 14.0% |
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 and Imatinib | 39 of 43 |
Most frequent serious adverse event terms
| Event term | Organ system | Gemcitabine and Imatinib |
|---|---|---|
| Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10e9/L) | General disorders | 2 of 43 |
| Pain - Abdomen not otherwise specified | Gastrointestinal disorders | 2 of 43 |
| Nausea | Gastrointestinal disorders | 2 of 43 |
| Dyspnea (shortness of breath) | Respiratory, thoracic and mediastinal disorders | 1 of 43 |
| Thrombosis/thrombus/embolism - pulmonary embolus | Vascular disorders | 1 of 43 |
| Mood alteration - Depression - pre-existing condition | Psychiatric disorders | 1 of 43 |
| Neutrophils/granulocytes (ANC/AGC) | Blood and lymphatic system disorders | 1 of 43 |
| Infection with normal ANC or Grade 1 or 2 neutrophils - Biliary tree | Infections and infestations | 1 of 43 |
| ALT, SGPT (serum glutamic pyruvic transaminase) | Metabolism and nutrition disorders | 1 of 43 |
| Bilirubin (hyperbilirubinemia) | Metabolism and nutrition disorders | 1 of 43 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Gemcitabine and Imatinib |
|---|---|---|
| Neutrophils/granulocytes (ANC/AGC) | Blood and lymphatic system disorders | 25 of 43 |
| Nausea | Gastrointestinal disorders | 22 of 43 |
| Leukocytes (total WBC) | Blood and lymphatic system disorders | 20 of 43 |
| Fatigue (asthenia, lethargy, malaise) | General disorders | 19 of 43 |
| Hemoglobin | Blood and lymphatic system disorders | 17 of 43 |
| Constipation | Gastrointestinal disorders | 12 of 43 |
| Platelets | Blood and lymphatic system disorders | 12 of 43 |
| Anorexia | Gastrointestinal disorders | 11 of 43 |
| Pain - Abdomen NOS | General disorders | 11 of 43 |
| Diarrhea | Gastrointestinal disorders | 9 of 43 |
Serious events with the highest arm-level rate: fever (in the absence of neutropenia, where neutropenia is defined as anc <1.0 x 10e9/l) (4.7%), pain - abdomen not otherwise specified (4.7%), nausea (4.7%), dyspnea (shortness of breath) (2.3%), thrombosis/thrombus/embolism - pulmonary embolus (2.3%).
The registry reports serious adverse event figures for the Gemcitabine and Imatinib group: 6 affected among 43 at risk. For other adverse events, it reports 39 affected among 43 at risk. These are separate categories in the record and should not be combined. They describe events recorded in the study group; without a concurrent comparator, they do not establish whether the combination caused an event or whether the rate differs from another treatment.
The record lists the following serious adverse event terms and percentages: Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10e9/L), Pain - Abdomen not otherwise specified, and Nausea, each at 4.7%; Dyspnea (shortness of breath), Thrombosis/thrombus/embolism - pulmonary embolus, and Mood alteration - Depression - pre-existing condition, each at 2.3%. These are registry-reported percentages. The information available does not establish event timing, duration, attribution, or whether a percentage refers to participants or occurrences.
Deaths are not reported in the adverse-event summary. The figures should be interpreted as descriptive safety reporting, not as a comparison of risks or a complete account of all possible harms.
12. What the Result Does, and Does Not, Mean
The record documents a completed phase II study of first-line gemcitabine and imatinib mesylate in patients with locally advanced or metastatic pancreatic cancer. It identifies progression-free survival as the primary endpoint, defines progression using RECIST v1.0, and specifies a median with a 95% confidence interval. It also records enrollment and selected adverse-event figures.
It does not report the primary endpoint estimate or its confidence interval. It also does not report results for the listed secondary outcomes in the information available for this analysis. Therefore, the posted record cannot support a description of the observed progression-free survival, response rate, one-year survival rate, or overall survival. It does not permit an assessment of the statistical precision of the primary result.
The design is single group, with allocation listed as NA and no masking. It does not provide a randomized comparison with another treatment. Even a reported outcome estimate from this group would describe the study participants and would not, on its own, establish what would have happened to comparable patients receiving a different treatment. The record should not be read as demonstrating that the combination does or does not work.
General statistical interpretation also depends on the analysis population, event counts, follow-up, censoring, and handling of missing data. These details are not reported in the registry record. Enrollment and the stated eligibility details do not show which participants contributed to each endpoint analysis.
The adverse-event figures offer a limited descriptive account: 6 affected among 43 at risk for serious adverse events and 39 affected among 43 at risk for other adverse events, alongside selected serious-event terms and percentages. They do not establish causation or comparative safety. In short, the record describes the study’s intent, design, endpoint definition, and some safety reporting, while leaving the principal efficacy results and several key analysis details unreported.
13. Why This Trial Matters Statistically
This study is statistically informative as an example of how design constrains interpretation. The record identifies a phase II, single-group study, not a randomized comparison. That distinction determines the scope of the evidence: outcomes can describe the group studied, but treatment differences cannot be separated from other explanations without a suitable comparator.
The primary endpoint is time-to-event, and the record specifies a median and 95% confidence interval. This is a useful distinction between an estimate and its uncertainty: in general, a point estimate alone does not communicate how precisely an outcome has been estimated. In this record, neither the median nor the interval is reported, so the stated endpoint plan cannot be translated into a quantitative result.
The progression definition is also important. The use of RECIST v1.0 makes the event criteria explicit, including a 20% increase in the sum of the longest diameter of target lesions, measurable increase in a non-target lesion, or new lesions. In general, clear event definitions support consistent analysis, but readers also need information about assessment timing and endpoint handling. Those details are not reported in the registry record.
The enrollment and eligibility notes illustrate why analysis populations need to be described by outcome. One patient withdrew consent before treatment, and one was evaluable for toxicity but not response after being found to have ampullary cancer. The record does not state the analysis populations or denominators for the primary endpoint. This limits the ability to connect enrollment to the endpoint estimates.
Finally, the gap between a specified endpoint and an unreported result is itself consequential for evidence interpretation. The record lists outcome aims and selected safety figures, but does not report the primary efficacy estimate or secondary outcome results. Statistical interpretation should reflect that reporting boundary rather than treating an endpoint description as a result.
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 central limitation for interpreting efficacy is that the registry record does not report the primary progression-free survival median or its 95% confidence interval. It also does not report the listed secondary outcome results. As a result, the record does not permit a quantitative assessment of the study’s efficacy outcomes.
The single-group design has no concurrent comparator. Allocation is listed as NA and masking as none. This prevents a randomized, like-for-like treatment comparison and limits conclusions to descriptions of the study group. The record does not provide a sponsor-reported hazard ratio or adjusted comparison.
Several details needed to evaluate the time-to-event analysis are not reported in the registry record: the analysis population, number of progression or death events, endpoint-specific denominators, follow-up distribution, censoring rules, and handling of missing outcome data. The record identifies the endpoint timeframe as four years, but does not report the assessment schedule or participant-level follow-up.
The record notes that one patient withdrew consent before treatment and one patient was evaluable for toxicity but not response after an ampullary cancer determination. It does not explain how these participants were handled in each analysis, or identify the full populations used for progression-free survival and other outcomes.
The protocol’s statistical analysis plan, sample-size justification, interim analysis procedures, and multiplicity strategy are not reported in the registry record. Safety reporting is also limited to the listed affected figures and selected serious adverse event terms and percentages; deaths are not reported in the adverse-event summary. These gaps constrain interpretation but do not establish that unreported procedures or outcomes were absent from the study itself.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Gemcitabine and Imatinib Mesylate as First-Line Therapy in Patients With Locally Adv. or Metastatic Pancreatic Cancer — NCT00161213.
- Registry record last updated 2023-08-14; status last verified 2023-07.
- Results first posted 2012-12-27.
- 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 completed phase II, single-group study of gemcitabine and imatinib mesylate as first-line therapy for locally advanced or metastatic pancreatic cancer. Enrollment was 44. The record lists no masking and allocation as NA.
Progression-free survival was the primary endpoint, defined using RECIST v1.0, with a median and 95% confidence interval specified over a four-year timeframe. The median and confidence interval are not reported in the registry record. Response rate, one-year survival rate, and overall survival are listed as secondary outcomes, but their results are not reported in the information available for this analysis.
The record notes one withdrawal before treatment and one participant evaluable for toxicity but not response. For the Gemcitabine and Imatinib group, it reports 6 affected among 43 at risk for serious adverse events and 39 affected among 43 at risk for other adverse events. Deaths are not reported in the adverse-event summary. No concurrent comparator or detailed statistical analysis plan is reported.