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 Phase II Study of Sorafenib + Carboplatin and Docetaxel in First Line Treatment of Stage IIIB/IV NSCLC (NCT00647426), a na phase 2 study. The primary outcome is The Number of Patients With Stage IIIB/IV NSCLC Response Rate to the Combination Therapy of Sorafenib and Docetaxel/Carboplatin.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00647426 |
| Phase | Phase 2 |
| Condition | Non-Small Cell Lung Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 7 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Abramson Cancer Center at Penn Medicine |
| Results posted | Yes |
2. Clinical Question
Population
Patients with stage IIIB/IV non-small cell lung cancer receiving first-line treatment. The registry record describes the study as enrolling patients with advanced non-small cell lung cancer, and records enrollment of 7 participants. It also states that sufficient data was not collected for the primary outcome population.
Intervention
Sorafenib in combination with docetaxel and carboplatin. The arm description specifies 400 mg po BID Sorafenib and 75 mg/m2 IV Docetaxel on day 1, plus AUC 6 on Carboplatin on day 1 of each 21 day cycle. The summary describes sorafenib as continuing daily and chemotherapy as repeating every 21 days for a total of four cycles, subject to disease progression, unacceptable or prohibitive toxicity, or withdrawal as described in the record.
Comparator
There is no separate comparator group. The study is recorded as a single-group study with allocation listed as NA, so it does not provide a like-for-like control comparison. Comparisons with other treatments or with an external standard are not reported in the registry record.
Primary question
The primary question was the response rate to the combination therapy in patients with stage IIIB/IV non-small cell lung cancer, with a stated timeframe of 30 months. The record defines best overall response using tumor measurements and confirmation criteria, but says sufficient data was not collected. A numerical response result is therefore not reported in the registry record.
3. Trial Design
This was a phase II, open-label, single-institution study of sorafenib combined with docetaxel and carboplatin in first-line treatment of stage IIIB/IV non-small cell lung cancer. The registry lists the design as single group, with allocation recorded as NA and masking as none. There was no randomized control arm or other concurrent comparator group in the registry record.
The recorded enrollment was 7. The study status is completed. The registry lists a start of 2007-11 and primary completion of 2011-08. Those dates describe the record’s study timeline; they do not supply a response result or explain how long each participant was followed.
The intervention is listed as Sorafenib + Docetaxel/Carboplatin. The arm description gives 400 mg po BID Sorafenib, 75 mg/m2 IV Docetaxel on day 1, and AUC 6 on Carboplatin on day 1 of each 21 day cycle. The brief summary says chemotherapy courses were intended to repeat every 21 days for a total of four cycles, while sorafenib was to continue until progression, prohibitive toxicity, or withdrawal. These descriptions summarize the planned regimen; they do not establish what each participant received.
The primary outcome concerns response rate, with a stated timeframe of 30 months. Secondary outcomes include progression-free survival, serious adverse events, and biomarkers of sorafenib activity and toxicity. The record lists four outcome measures, but does not report sufficient data for the primary outcome or provide statistical analyses. Treatment assignment, outcome results, follow-up details, and analysis populations beyond the record’s brief descriptions should not be inferred.
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 | Sorafinb + Docetoxel+Carboplatin |
|---|---|
| STARTED | 7 |
| COMPLETED | 5 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | Sorafinb + Docetoxel+Carboplatin |
|---|---|
| Withdrawal by Subject | 2 |
Analysis population for the primary outcome, as defined in the registry: Sufficient data was not collected.
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 | Sorafinb + Docetoxel+Carboplatin |
|---|---|---|
| Participants analysed | 7 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 6 |
| Age, Categorical: >=65 years | Participants | 1 |
| Sex: Female, Male: Female | Participants | 2 |
| Sex: Female, Male: Male | Participants | 5 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 6 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 6 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Region of Enrollment: United States | Participants | 7 |
6. Primary Endpoint
The registry lists the primary outcome as The Number of Patients With Stage IIIB/IV NSCLC Response Rate to the Combination Therapy of Sorafenib and Docetaxel/Carboplatin, measured over 30 months.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is described as the number of patients with a response to the combination therapy. Its stated timeframe is 30 months. The record defines best overall response as the best response recorded from treatment start until disease progression or recurrence, using the smallest measurements recorded since treatment began as the reference for progressive disease.
For target lesions, complete response is defined as 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 the baseline sum. Progressive disease requires at least a 20% increase relative to the smallest sum recorded since treatment started, or the appearance of one or more new lesions. The record notes that response assignment depends on measurement and confirmation criteria, while the referenced details of those criteria are not reported in the registry record.
Response is used in clinical trials as a way to describe tumor change during treatment. It is distinct from outcomes such as progression-free survival, overall survival, or symptom improvement. Those outcomes answer different questions and cannot be substituted for a response result. In this record, the population field says sufficient data was not collected. No response count, response rate, or estimate of its precision is reported in the registry record, so the endpoint definition alone cannot establish how participants responded.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a treatment regimen and a primary response-rate question, but it does not provide a numerical primary-outcome result. It explicitly states that sufficient data was not collected for the primary outcome population. Consequently, there is no reported response count or rate to interpret, and no estimate of uncertainty around a response result. The record’s completion status does not fill that gap: study completion and availability of analyzable endpoint data are separate matters.
The single-group design is important for interpretation. Any outcome observed among participants receiving the combination would describe that group, but without a concurrent comparator it would not show how outcomes differed from another treatment or from no treatment. Differences in patient characteristics, disease course, follow-up, and other care could not be separated from treatment effects using a control comparison that this design does not contain. The registry also gives no adjusted or unadjusted effect estimate for a treatment contrast.
Enrollment is recorded as 7, but the record does not state how many participants contributed evaluable primary endpoint data. The statement that sufficient data was not collected makes it especially important not to treat enrollment as the denominator for a response calculation. The number enrolled cannot be used to reconstruct a response rate when response status and the analysis population are not reported.
The adverse-event record lists 7 at risk and 0 affected for serious adverse events, and 7 at risk and 0 affected for other adverse events in the listed group. These are registry counts, not a complete description of event types, observation time, or ascertainment. The record lists no serious adverse-event terms, and deaths are recorded as null rather than as a numeric count. These entries should be read as the posted safety fields, not as proof that no events occurred outside the captured data.
There are no statistical analyses listed in the registry record. No confidence interval, hypothesis test, adjusted model, or analysis population is reported. Thus, the record supports a description of the design, planned endpoint, and posted adverse-event counts, but not a quantitative conclusion about response or comparative treatment effects.
9. Statistical Methods Explained
What does a single-group design estimate?
In a single-group study, outcomes are described among participants receiving the intervention. The registry classifies this study as single group, with allocation listed as NA and masking as none. Because no separate comparator arm is recorded, the design does not directly estimate a difference between treatments. A response observed in the treated group, if it had been reported, would describe outcomes in that group under the study’s conditions; it would not by itself identify what would have happened to the same participants under an alternative treatment.
This distinction is general to trial interpretation. Randomized comparisons can help balance measured and unmeasured factors between groups, while an uncontrolled group does not provide that protection. A single-group result can still be descriptive, but causal interpretation requires caution. No causal or comparative treatment estimate is reported in the registry record.
How is the response endpoint defined?
The record uses best overall response, assessed from treatment start until progression or recurrence, and specifies measurement-based criteria for complete response, partial response, and progressive disease. These categories turn tumor measurements into an outcome classification. Complete response means disappearance of all target lesions. Partial response uses at least a 30% decrease in the sum of longest diameters from baseline. Progressive disease uses at least a 20% increase from the smallest sum recorded since treatment began or the appearance of new lesions.
As a general statistical concept, categorical response criteria make outcomes easier to summarize, but the resulting response proportion depends on having a defined analysis population and adequately captured response status. Measurement and confirmation rules can affect classification. The registry refers to confirmation criteria but does not report those details. It also states that sufficient data was not collected for the primary outcome, so no response estimate can be reconstructed from the record.
What do precision measures communicate?
Generally, a confidence interval describes the uncertainty around an estimated quantity under the assumptions of the analysis. A narrower interval indicates greater statistical precision, while a wider interval indicates less precision. An interval does not correct for missing outcome data, bias from the design, or uncertainty about whether the analysis population represents the intended population. No confidence interval or response estimate is reported in the registry record, so precision for the primary endpoint cannot be assessed.
Likewise, a hypothesis test addresses a specified statistical question under a model and analysis plan. A p-value is not a measure of clinical importance, and a non-significant result would not establish that groups are equivalent. In this study, no treatment contrast is listed and no statistical analyses are reported. There is therefore no reported hypothesis test or p-value to interpret.
What do planning illustrations mean?
General sample-size calculations describe the number of participants or events that a hypothetical design might require under stated assumptions. The planning assumptions associated with this record specify a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a stated fraction of the observed effect. For time-to-event outcomes, the stated approach uses the total number of events; for other outcomes, it expresses the quantity as participants per arm.
These are planning assumptions, not evidence that the original trial used a two-arm design. The actual record describes a single-group study. The illustrations are not observed power, do not establish that the study was adequately sized, and do not replace a result. No numerical analysis result or effect estimate appears in the registry record from which to judge the precision of a primary response estimate. The registry also does not report the protocol’s sample-size justification or statistical plan.
Why can over-dispersion matter?
In general, over-dispersion occurs when count data vary more than a statistical model assumes. If ignored, standard errors can be too small and confidence intervals or tests can be misleading. It is relevant only when the selected outcome and model involve counts with that feature. The registry record does not report a count model, an over-dispersion assessment, or a statistical analysis for the primary outcome. It would therefore be inappropriate to claim that over-dispersion was present or that a particular method addressed it.
10. Multiplicity, Missing Data and Analysis Populations
The record lists four outcome measures. The primary outcome is response rate, and the listed secondary outcomes concern progression-free survival, serious adverse events, and biomarkers of sorafenib activity and toxicity. Looking at multiple outcomes can raise a multiplicity question: when many hypotheses are tested, the chance of at least one apparently notable result can increase. General approaches include prespecifying a primary outcome, adjusting for multiple tests, or treating secondary analyses as exploratory. Which approach was planned for this study is not reported in the registry record.
No analyses are listed in the record. It does not report a multiplicity adjustment, an analysis hierarchy, or a statistical analysis plan. There are no reported estimates or tests across the listed outcomes to evaluate for multiplicity.
Missing outcome information is a separate concern. The primary outcome’s population field states that sufficient data was not collected. The record does not explain why data were insufficient, which participants had missing response assessments, how missingness was handled, or whether any sensitivity analyses were conducted. It also does not report the analysis population used for response or progression-free survival. Enrollment should not be assumed to equal the number analyzed.
These gaps limit what can be inferred from the registry entry. They do not show that a particular missing-data method or multiplicity procedure was used; neither is reported. The absence of posted analyses also means readers cannot assess how endpoint definitions, missing observations, or multiple outcomes affected any trial conclusions.
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 |
|---|---|---|---|
| Sorafenib + Docetaxel | 0 | 7 | 0.0% |
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 |
|---|---|
| Sorafenib + Docetaxel | 0 of 7 |
The adverse-event fields list a group titled Sorafenib + Docetaxel. For serious adverse events, the record lists 7 at risk and 0 affected. For other adverse events, it also lists 7 at risk and 0 affected. No serious adverse-event terms are listed. The deaths field is null, which is not a numeric death count and should not be interpreted as zero.
These figures describe the entries in the registry, not necessarily the full safety experience. The record does not give event-level detail, severity grades, observation time, or a narrative of how adverse events were collected. It also does not state whether every participant was followed for the same duration. A count of zero affected in a posted field does not establish that no events occurred outside the data captured in that field.
The study has no comparator group, so the safety counts cannot be used to estimate a difference in risk relative to another treatment. They should be read as descriptive registry counts for the listed group. The group title does not include carboplatin, while the intervention description names sorafenib with docetaxel and carboplatin; the record does not explain this labeling difference.
12. What the Result Does, and Does Not, Mean
The record documents a completed phase II study of sorafenib with docetaxel and carboplatin for first-line treatment of stage IIIB/IV non-small cell lung cancer. It describes an open-label, single-group design, records enrollment of 7, and defines response rate as the primary outcome with a stated timeframe of 30 months. It also lists secondary outcomes involving progression-free survival, serious adverse events, and biomarkers.
What the record does not establish is the primary response result. The outcome population field says sufficient data was not collected, and no response count, response rate, or confidence interval is reported. The absence of an outcome result does not show that participants did or did not respond; it means the registry entry does not provide enough information to quantify response.
Nor can this design establish comparative effectiveness. With no separate control arm, the record cannot show whether the combination led to better or worse outcomes than an alternative. The registry also contains no reported statistical analyses or treatment-effect estimate. The safety counts are limited to posted fields and do not provide a complete comparative safety assessment.
The study’s stated regimen and endpoint remain useful for understanding what investigators intended to examine. However, a treatment description, an endpoint definition, and a completed status are not substitutes for reported results. Interpretation should remain confined to the design and fields that the registry actually records, without inferring unreported outcomes or analyses.
13. Why This Trial Matters Statistically
This study illustrates why design and outcome reporting must be read together. A phase II label and a clearly defined response endpoint indicate the kind of question the investigators set out to examine, but they do not guarantee that an interpretable estimate is available. Here, the registry states that sufficient data was not collected for the primary endpoint population. That limitation prevents readers from estimating response from the posted record.
The single-group design also marks an important inferential boundary. Outcomes from a treated group can be described, but without a concurrent control group they cannot provide a direct estimate of how outcomes differ under another treatment. This matters especially when a reader encounters a regimen and response criteria that appear precise: precision in the endpoint definition does not remove the absence of a comparator or an outcome estimate.
The safety fields provide a further lesson in registry reading. Posted zero counts and a null deaths field are not interchangeable, and neither should be expanded into claims about events beyond what the record captured. Clear distinctions among a numeric zero, an unreported value, and an unavailable result are central to responsible interpretation.
Finally, planning illustrations are not trial results. Assumptions about significance level, allocation, and statistical approximation can help frame hypothetical designs, but they do not establish the original study’s sample-size rationale or its achieved precision. The registry does not report its statistical plan or analyses. Keeping planned questions, design constraints, and reported findings separate is essential when summarizing this study.
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 most direct limitation is that the registry says sufficient data was not collected for the primary outcome population. It reports no numerical response result, so neither the magnitude nor precision of response can be evaluated. The record does not explain why the data were insufficient, whether response assessments were unavailable for some participants, or how missing observations were handled.
The study is listed as single group, with allocation recorded as NA and masking as none. Without a comparator, any reported outcome would be descriptive rather than a direct comparison of treatments. In this record, no response result is reported in any case. The enrollment of 7 does not establish the number of participants with an evaluable outcome.
The registry does not report the statistical analysis plan, sample-size justification, analysis populations, or any statistical analyses. It also does not provide detailed follow-up information, despite listing a 30-month timeframe for the primary outcome. The response description references measurement and confirmation criteria whose details are not reported in the registry record.
For safety, the posted counts do not include event-level descriptions or observation time. The deaths value is null, and no serious adverse-event terms are listed. These entries leave important details unreported and should not be treated as a complete account of safety. The intervention and adverse-event group labels also differ: the intervention includes carboplatin, while the adverse-event group title names sorafenib and docetaxel. The registry does not explain the discrepancy in labeling.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Phase II Study of Sorafenib + Carboplatin and Docetaxel in First Line Treatment of Stage IIIB/IV NSCLC — NCT00647426.
- Registry record last updated 2019-10-22; status last verified 2019-09.
- Results first posted 2019-10-22.
- 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.
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
This completed phase II study evaluated sorafenib with docetaxel and carboplatin as first-line treatment for stage IIIB/IV non-small cell lung cancer. It was open-label, conducted at a single institution, and recorded as a single-group study without a comparator. Enrollment was 7.
The primary endpoint was response rate, with a 30-month timeframe and measurement-based definitions for complete response, partial response, and progressive disease. The record states that sufficient data was not collected for the primary outcome population and reports no numerical response result or statistical analysis.
Secondary outcomes listed include progression-free survival, serious adverse events, and biomarkers of sorafenib activity and toxicity. The posted adverse-event fields record 0 affected for serious events and 0 affected for other events, with 7 at risk in each field; deaths are null. These fields do not supply a complete safety narrative or comparative assessment.