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 The 'MADe IT' Clinical Trial: Molecular Analyses Directed Individualized Therapy for Advanced Non-Small Cell Lung Cancer (NCT00215930), a na phase 2 study. The primary outcome is Best Disease Response After a Maximum of Six Cycles..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00215930 |
| Phase | Phase 2 |
| Condition | Carcinoma, Non-Small-Cell Lung |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 53 |
| Arms in the primary outcome | n/a |
| Lead sponsor | H. Lee Moffitt Cancer Center and Research Institute |
| Results posted | Yes |
2. Clinical Question
Population
Participants had newly diagnosed advanced non-small-cell lung cancer. The official title specifies stage IV or stage IIIB disease with malignant pleural effusion. Enrollment was 53 participants, and the primary outcome description says participants were analyzed according to Response Evaluation Criteria in Solid Tumors (RECIST).
Intervention
The intervention was a chemotherapy doublet assigned using tumor molecular analysis. Before treatment, expression of ERCC1 and RRM1 was measured; the registry says the assignment was based on those gene-expression results. The listed drugs are vinorelbine, docetaxel, gemcitabine, and carboplatin. The registry does not report which specific doublet each participant received.
Comparator
There was no listed comparison group. The design is identified as single group, with allocation recorded as not applicable and masking as none. Therefore, the record does not describe a randomized or like-for-like control comparison that would support a direct estimate of the intervention’s effect relative to another treatment.
Primary question
The primary question was how many participants fell into each best-disease-response category after a maximum of six cycles of molecular-analysis-directed chemotherapy. The outcome timeframe is listed as 24 months. The registry record does not report the response-category counts, so the observed response distribution cannot be described from this record.
3. Trial Design
This was a phase II clinical trial of molecular-analysis-directed chemotherapy for advanced non-small-cell lung cancer. The registry lists 53 participants and a single-group model. Allocation is recorded as not applicable, and masking as none. The study is marked completed; its listed start was 2004-02 and its primary completion was 2009-10.
The treatment strategy used tumor expression of ERCC1 and RRM1 to guide assignment to a doublet chemotherapy regimen. The listed drugs are vinorelbine, docetaxel, gemcitabine, and carboplatin. The registry describes the arm as “Double Agent Chemotherapy” and says assignment was based on gene expression of ERCC1 and RRM1. It does not specify the mapping from individual molecular profiles to particular drug combinations, or how many participants received each combination.
The primary outcome was best disease response after a maximum of six cycles, with a 24-month timeframe. The outcome description says tumor expression was measured before treatment and that participants were analyzed according to RECIST. Overall survival and progression-free survival are listed as secondary outcomes. However, the registry does not report numerical results for these outcomes in the record.
Because there is no control group, the design can describe what happened among participants treated under this strategy, but it cannot by itself separate treatment effects from the natural course of disease, participant characteristics, or other influences. The record does not report a sample-size justification, a detailed statistical analysis plan, or interim-analysis procedures. These details should not be inferred from the phase label or the single-group design.
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: Assignments: High ERCC1 expression and High RRM1 Expression - vinorelbine plus docetaxel (DV); High ERCC1 expression but NOT High RRM1 expression - docetaxel plus gemcitabine (GD); High ERCC1 expression, but HAVE High RRM1 - carboplatin plus docetaxel (DC); High ERCC1 expression and do NOT have High RRM1 - carboplatin plus gemcitabine (GC)
Overall Study
| Stage | Double Agent Chemotherapy |
|---|---|
| STARTED | 53 |
| COMPLETED | 53 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: All participants were analyzed according to Response Evaluation Criteria in Solid Tumors (RECIST).
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 | Double Agent Chemotherapy |
|---|---|---|
| Participants analysed | 53 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 37 |
| Age, Categorical: >=65 years | Participants | 16 |
| Age, Customized | years | 63 (38 to 78) |
| Sex: Female, Male: Female | Participants | 22 |
| Sex: Female, Male: Male | Participants | 31 |
| Region of Enrollment: United States | participants | 53 |
6. Primary Endpoint
The registry lists the primary outcome as Best Disease Response After a Maximum of Six Cycles., measured over 24 Months.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants in each best-disease-response category after a maximum of six chemotherapy cycles. The registry gives a 24-month timeframe and states that participants were evaluated according to RECIST. It describes measuring tumor expression of ERCC1 and RRM1 before treatment and assigning a chemotherapy doublet on that basis. The response categories themselves and the number of participants in each category are not reported in the registry record.
Counting participants by response category gives a descriptive account of tumor response observed during the study. It is useful for summarizing the range of outcomes in a phase II study and for showing how frequently each recorded category occurred. Such counts do not, on their own, establish that the treatment strategy caused an outcome or that it performs better than another strategy. That distinction is especially important in a single-group study without a control arm.
The registry describes the outcome as a number of participants, rather than reporting a modeled treatment effect. No confidence interval, response estimate, or statistical test for this endpoint is reported in the registry record. As a result, the outcome’s precision and the uncertainty around the response distribution cannot be assessed from the posted endpoint information.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a molecularly guided chemotherapy strategy and identifies best disease response as the primary endpoint. It reports enrollment of 53 participants and says that response was assessed according to RECIST. However, it does not report how many participants were in each response category. Consequently, the principal clinical outcome cannot be characterized from the registry record: there is no reported response distribution to interpret, and no reported estimate of its uncertainty.
The single-group design shapes what an eventual response count could mean. A count among participants treated under the strategy would describe outcomes in that group. Without a concurrent comparator, it would not estimate the difference between molecularly guided assignment and another treatment approach. It would also not establish that gene-expression-guided assignment, rather than other participant or care factors, explains any observed outcomes. The registry itself notes no randomized comparison.
Overall survival and progression-free survival are listed as secondary outcomes, but their numerical results are not reported in the registry record. No analysis estimates, confidence intervals, p-values, or event counts for those outcomes are listed. Their inclusion as outcome names alone does not show whether they were measured completely, how they were analyzed, or what the results were.
The safety information is more numerical but remains limited. For the double-agent chemotherapy group, the registry lists 53 participants at risk and 29 affected for serious adverse events. It lists 53 at risk and 5 affected for other adverse events. It also names several serious adverse-event terms with percentages. Those entries describe the safety information as recorded; they do not provide a comparison with an untreated or alternative-treatment group, nor do they establish the cause of an event.
The record lists no analyses. It does not report how the primary response counts were handled statistically, how missing observations were treated, or whether any adjustment was planned for multiple outcomes or tests. Thus, the most defensible interpretation is limited to the design, outcome definitions, and posted safety counts. The missing response and survival results prevent an assessment of the study’s observed efficacy outcomes or their precision.
9. Statistical Methods Explained
The registry identifies this study as phase II, single group, with allocation not applicable and no masking. Those features describe the structure of the study, not the statistical procedure used to estimate an effect. No statistical analyses are listed in the registry record, and a detailed analysis plan is not reported in the registry record. The method used for any unposted analysis therefore cannot be reconstructed from the record.
What does a single-group design estimate?
In general, a single-group study can summarize outcomes among the participants who received the study strategy. For a categorical endpoint such as best response, the basic descriptive result would be a count in each response category. If a proportion were also reported, it would describe the share of the analyzed group in that category. Such a within-group description is not the same as a comparative treatment effect: without a comparator, there is no concurrent outcome distribution against which to estimate a between-group difference.
For this trial, the registry says the endpoint is the number of participants in each response category after a maximum of six cycles and that all participants were analyzed according to RECIST. It does not give the category counts or explain how participants without an assessable response were handled. No response proportions, uncertainty intervals, or tests are reported in the registry record. General statistical practice would distinguish observed counts from estimates and would report uncertainty when making inferences beyond the enrolled group, but the record does not establish that any particular inferential method was used.
What would a confidence interval contribute?
In general, a confidence interval expresses uncertainty around an estimate under the assumptions of the method used. For a response proportion, it can indicate how much the estimate may vary because only a limited group of participants was observed. It does not correct for a missing control group, bias in who entered the study, or errors in measurement. No confidence interval is reported for the primary endpoint in the registry record, so its precision cannot be evaluated from the posted results.
How should survival outcomes be read?
Overall survival and progression-free survival are listed as secondary outcomes. In general, time-to-event outcomes require a definition of the event, a time origin, and an approach to participants whose event has not been observed by the end of follow-up. Methods may account for such incomplete follow-up, but the appropriate details depend on the protocol and data. The registry record does not report the outcome definitions in enough detail to establish these analysis choices, nor does it give event counts, estimates, confidence intervals, or survival curves. The methods used for these outcomes are therefore not reported in the registry record.
What does the safety information count?
The safety entries give numbers at risk and numbers affected for serious and other adverse events, alongside selected serious-event terms and percentages. In general, a count of participants affected is distinct from a count of event episodes: one participant may experience more than one event. The registry record does not clarify whether the term-level percentages represent participants or episodes, or provide the detailed coding and tabulation rules. The listed figures should therefore be read as posted safety summaries, not as a complete event-level analysis.
What cannot be inferred about the statistical plan?
The record does not report the sample-size justification, a prespecified hypothesis test, the method for handling missing outcome data, multiplicity adjustments, interim analyses, or a detailed definition of analysis populations beyond the statement that participants were analyzed according to RECIST. Their absence from the record does not show that a procedure was or was not used; it means the procedure is not reported in the registry record. General explanations of statistical methods cannot fill in those study-specific details.
10. Multiplicity, Missing Data and Analysis Populations
The record lists best disease response as the primary outcome and overall survival and progression-free survival as secondary outcomes. It does not report a detailed statistical analysis plan or identify a method for controlling multiplicity across outcomes, response categories, or any additional analyses. No analyses are listed. It is therefore not possible to determine whether the investigators used a prespecified testing hierarchy, adjusted significance thresholds, or treated the outcomes as descriptive. These methods are not reported in the registry record.
The primary outcome description says participants were analyzed according to RECIST, but the response counts by category are not posted. The record does not explain how a participant without an evaluable response was classified, whether any observations were excluded, or how missing assessments were handled. The number of participants with missing primary-outcome data is also not reported in the registry record. Accordingly, the stated analysis population does not provide enough detail to assess completeness or the potential influence of missing data.
For survival outcomes, the registry does not provide numerical results or analysis details such as event definitions, follow-up handling, or censoring rules. Those choices affect interpretation of time-to-event estimates, but should not be assumed. The registry also does not report interim analyses or a sample-size justification. No conclusion about these procedures can be drawn from the completed status or the phase designation alone.
In general, multiple outcome analyses can increase the chance that at least one apparent finding arises by chance when many tests are conducted. Missing data can also affect estimates if the availability of an outcome is related to participants’ condition or treatment experience. These are general statistical considerations; whether they affected this study cannot be assessed from the registry record.
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 |
|---|---|---|---|
| Double Agent Chemotherapy | 29 | 53 | 54.7% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 2% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Double Agent Chemotherapy | 5 of 53 |
Most frequent serious adverse event terms
| Event term | Organ system | Double Agent Chemotherapy |
|---|---|---|
| Lymphocytes - Grade 3 | Blood and lymphatic system disorders | 11 of 53 |
| Leukocytes - Grade 3 | Blood and lymphatic system disorders | 7 of 53 |
| Neutrophils - Grade 3 | General disorders | 5 of 53 |
| Fatigue - Grade 3 | General disorders | 4 of 53 |
| Hyponatremia - Grade 3 | General disorders | 3 of 53 |
| Dyspnea - Grade 3 | Respiratory, thoracic and mediastinal disorders | 2 of 53 |
| FN-ANC <1.0/Fever >=38.5C - Grade 3 | General disorders | 2 of 53 |
| Hand/Foot Syndrome - Grade 3 | General disorders | 2 of 53 |
| Hemoglobin - Grade 3 | Blood and lymphatic system disorders | 2 of 53 |
| Hyperglycemia - Grade 3 | Blood and lymphatic system disorders | 2 of 53 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Double Agent Chemotherapy |
|---|---|---|
| Hemoglobin - Grade 1 | Blood and lymphatic system disorders | 1 of 53 |
| Hypokalemia - Grade 1 | Blood and lymphatic system disorders | 1 of 53 |
| Leukocytes - Grade 1 | Blood and lymphatic system disorders | 1 of 53 |
| Lymphocytes - Grade 1 | Blood and lymphatic system disorders | 1 of 53 |
| Platelet Count - Grade 1 | Blood and lymphatic system disorders | 1 of 53 |
Serious events with the highest arm-level rate: lymphocytes - grade 3 (20.8%), leukocytes - grade 3 (13.2%), neutrophils - grade 3 (9.4%), fatigue - grade 3 (7.5%), hyponatremia - grade 3 (5.7%).
The registry lists 53 participants at risk for serious adverse events and 29 affected in the Double Agent Chemotherapy group. For other adverse events, it lists 53 at risk and 5 affected. These are posted counts; the record does not provide a control group for comparison. The counts therefore describe the recorded safety experience in this study group and do not establish that chemotherapy caused any particular event.
The listed serious-event terms and percentages are lymphocytes - Grade 3, 20.8%; leukocytes - Grade 3, 13.2%; neutrophils - Grade 3, 9.4%; fatigue - Grade 3, 7.5%; hyponatremia - Grade 3, 5.7%; and dyspnea - Grade 3, 3.8%. The registry presents these as selected top serious terms. It does not give the full event listing or explain whether the percentages count participants, event episodes, or another denominator. They should not be treated as a complete account of all adverse events.
In general, adverse-event summaries depend on definitions, collection procedures, severity grading, and whether repeated events are counted separately. Detailed rules for these summaries are not reported in the registry record. The safety counts also do not indicate whether an event led to treatment discontinuation or how long participants were observed for safety outcomes.
12. What the Result Does, and Does Not, Mean
The record establishes that a phase II, single-group study enrolled 53 participants and evaluated a chemotherapy strategy assigned using tumor expression of ERCC1 and RRM1. It identifies best disease response after a maximum of six cycles as the primary outcome and lists overall survival and progression-free survival as secondary outcomes. It also reports selected safety counts and serious adverse-event terms.
Those details describe the study’s design and the information entered in the registry. They do not provide the response-category counts, survival estimates, or statistical analyses needed to characterize the observed efficacy outcomes. As a result, the registry record does not support a conclusion about the size, precision, or comparative meaning of any response or survival result. The absence of numerical outcomes in this record is not itself evidence for a particular clinical outcome.
Even if response counts were available, a single-group study would not provide a randomized comparison with another treatment strategy. The observed results could not, on their own, identify the effect of molecularly guided assignment relative to another approach. The record does not report an alternative arm or a control group, and it does not report enough statistical methods to assess adjusted analyses or other sources of uncertainty.
The safety entries show the counts and selected terms recorded for the study group, but they do not establish causation or comparative safety. The record does not give a detailed event-level account or the rules behind the summaries. Overall, the study record conveys the question and design, but leaves key outcome results and analysis details unreported.
13. Why This Trial Matters Statistically
This trial raises a statistical question about using tumor molecular measurements to assign treatment: how should outcomes be described when treatment selection is individualized but there is no control group? The registry says that ERCC1 and RRM1 expression guided assignment to a chemotherapy doublet. This makes the treatment strategy more than a fixed drug regimen; interpretation would also depend on how molecular results translated into particular assignments. That assignment rule is not reported in detail.
The single-group structure is important because it limits what a response count can establish. A descriptive count can show the outcomes observed among participants treated under the strategy, but it cannot isolate the effect of the strategy from other explanations. A comparison group, preferably established through a design that makes groups comparable, is needed for a direct comparative estimate. The registry record explicitly does not describe a randomized comparison.
The endpoint also illustrates the difference between defining an outcome and reporting its results. The record specifies best response, the RECIST framework, and a treatment-cycle limit, but does not post the category counts. Without those counts and an uncertainty estimate, readers cannot judge either the observed response distribution or its precision. Listing survival outcomes without their numerical results likewise does not permit statistical interpretation of survival.
Finally, the posted safety counts show why denominators and event definitions matter. Counts of participants affected and counts of event episodes answer different questions, and the record does not fully explain the term-level percentages. These are general reporting concerns; they cannot be resolved without further study details. The statistical importance of the trial lies in the design and reporting questions it presents, not in an outcome conclusion that the registry record does not report.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The main limitation for interpreting outcomes is that the registry record does not report the primary response-category counts. Although best disease response is identified as the primary endpoint, there is no posted distribution from which to describe the observed response or assess its precision. Overall survival and progression-free survival are listed as secondary outcomes, but their numerical results and analysis details are also not reported.
The study is single group, with allocation recorded as not applicable and no masking. No control group is described, so the study cannot provide a direct randomized comparison with another treatment approach. The design also means that any descriptive outcomes would be difficult to attribute specifically to molecularly guided assignment rather than other influences.
The registry names four chemotherapy drugs and says assignment was based on ERCC1 and RRM1 expression, but it does not report the specific treatment mapping, the number receiving each doublet, or details of the molecular-assignment procedure. These omissions limit interpretation of what treatment was actually received across the study group.
The record does not report a sample-size justification, a detailed statistical analysis plan, missing-data procedures, multiplicity handling, interim analyses, or detailed survival-analysis methods. It also does not describe the denominator and event-counting rules for the adverse-event term percentages. These items are not reported in the registry record and should not be inferred. The registry’s completed status does not resolve these limitations.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: The 'MADe IT' Clinical Trial: Molecular Analyses Directed Individualized Therapy for Advanced Non-Small Cell Lung Cancer — NCT00215930.
- Registry record last updated 2017-03-23; status last verified 2011-03.
- Results first posted 2011-02-23.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
NCT00215930 is a completed phase II, single-group study of molecular-analysis-directed chemotherapy in advanced non-small-cell lung cancer. Enrollment was 53. The registry lists vinorelbine, docetaxel, gemcitabine, and carboplatin, with assignment based on ERCC1 and RRM1 expression. Masking is listed as none; allocation is not applicable.
The primary outcome is the number of participants in each best-disease-response category after a maximum of six cycles, with a 24-month timeframe. The record says participants were analyzed according to RECIST, but it does not report the response counts. Overall survival and progression-free survival are listed as secondary outcomes, with no numerical results reported in the registry record.
Safety summaries list 29 affected for serious adverse events and 5 affected for other adverse events, each among 53 at risk. The record lists selected serious-event terms and percentages. No analyses are listed, and statistical-plan details are not reported in the registry record.