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 Monotherapy Pazopanib in Subjects With Advanced Non-Small Cell Lung Cancer (NCT00549328), a non-randomized phase 2 study. The primary outcome is Percentage of Participants Who Achieved Either a Confirmed Complete Response or Partial Response Per RECIST Criteria.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00549328 |
| Phase | Phase 2 |
| Condition | Lung Cancer, Non-Small Cell |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 14 |
| Arms in the primary outcome | n/a |
| Lead sponsor | GlaxoSmithKline |
| Results posted | Yes |
2. Clinical Question
Population
Participants with advanced non-small cell lung cancer, specified in the study summary as Stage IIIB or IV. The primary-outcome population was the All Treated Population: participants who met inclusion criteria and consented to participate. The registry record reports enrollment of 14.
Intervention
Pazopanib monotherapy, listed as Pazopanib (GW786034). The arm is described as a single-arm, non-randomized, single-stage study; the safety group is titled Pazopanib 800 mg.
Comparator
There was no concurrent control arm. Allocation was non-randomized and the model was single-group, so the study does not provide a like-for-like treatment comparison.
Primary question
The primary endpoint asked what percentage of participants achieved a confirmed complete or partial response under the stated tumor-response criteria, from baseline through the end of study, up to 2 years. The registry record says no formal efficacy analyses were performed because the study terminated early; it does not report a primary response estimate.
In statistical terms, this is a single-group question about the observed response proportion in a defined study population. Such a proportion can describe what was observed among participants who received the study intervention, but without a concurrent comparator it cannot separate treatment-associated outcomes from other explanations, such as the natural course of disease or differences between participants. The registry record lists progression-free survival, overall survival, disease control, and biomarker measures among secondary outcomes, but it does not provide formal efficacy results for the primary endpoint. The record therefore establishes the question the trial was designed to address, not a quantified answer to that question.
3. Trial Design
The registry identifies this as a Phase II, multi-center, open-label, non-randomized, single-group study of pazopanib in advanced non-small cell lung cancer. Its arm description specifies a single-stage design and monotherapy. With no random allocation and no control arm, the design is descriptive rather than a randomized comparison of treatment strategies. Participants and investigators were not masked, according to the registry.
The study enrolled 14 participants and is listed as terminated. Its recorded start was 2008-02, and primary completion was 2009-04. The reason for termination is not reported in the registry record. The planned primary follow-up window extended from baseline through the end of study, up to 2 years; the record does not establish that every enrolled participant was followed for that full period.
The primary endpoint concerned confirmed complete or partial tumor response. Secondary outcomes included the number of participants with complete or partial response or stable disease, progression-free survival, overall survival, circulating plasma biomarkers, and intra-tumoral biomarker characterization. These listed outcomes describe the scope of planned assessment; their presence does not establish that results were analyzed or reported. The registry lists 6 outcome measures and 0 analyses. It also states directly that no formal efficacy analyses were performed due to early termination.
The record does not report the reason for early termination, a sample-size justification, a detailed statistical analysis plan, or the planned handling of interim analyses. Those omissions limit what can be inferred about the intended statistical workflow. The small enrolled group and absence of a control arm also constrain precision and comparative interpretation, regardless of the original design intentions.
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 | Pazopanib 800 mg |
|---|---|
| STARTED | 14 |
| COMPLETED | 0 |
| NOT COMPLETED | 14 |
Reasons for not completing this period:
| Reason | Pazopanib 800 mg |
|---|---|
| Adverse Event | 2 |
| Disease Progression | 10 |
| Death | 1 |
| Use of Prohibited Medication | 1 |
Analysis population for the primary outcome, as defined in the registry: All Treated Population: all participants who met inclusion criteria and willingly consented to participate in the study
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 | Pazopanib 800 mg |
|---|---|---|
| Participants analysed | 14 | |
| Age, Continuous | years | 63.6 (SD 7.97) |
| Sex: Female, Male: Female | Participants | 7 |
| Sex: Female, Male: Male | Participants | 7 |
| Race/Ethnicity, Customized: White | participants | 14 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Participants Who Achieved Either a Confirmed Complete Response or Partial Response Per RECIST Criteria, measured over Baseline through End of Study (up to 2 years).
| 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 was the percentage of participants who achieved either a confirmed complete response or partial response according to the stated tumor-response criteria. A complete response was defined as disappearance of all known lesions, confirmed at 4 weeks. A partial response was defined as at least a 30% decrease in the sum of the longest diameters of target lesions relative to the baseline sum, also confirmed at 4 weeks. The assessment period ran from baseline through the end of study, up to 2 years.
Response endpoints are used to describe tumor change using prespecified categories and measurement rules. Requiring confirmation is intended to distinguish a sustained change from an unconfirmed assessment. A percentage would summarize the proportion of the relevant study population meeting either response definition, but that estimate is not reported in the registry record. The record also states that no formal efficacy analyses were performed because the study terminated early.
The endpoint title refers to RECIST criteria, while the description spells out the response definitions and refers to Response Evaluation Criteria In Solid Tumors. The registry does not report further details of image review, assessment timing beyond confirmation, or handling of unevaluable assessments. Without a posted estimate and its uncertainty, the endpoint definition alone cannot show how often participants responded or how precisely a response proportion could be estimated.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical limitation is that the registry defines a primary response endpoint but reports no formal efficacy analysis. No percentage achieving confirmed complete or partial response is posted, and no confidence interval or test result for that endpoint is reported in the registry record. Consequently, the efficacy question cannot be answered quantitatively from the recorded primary results. Early termination is given as the reason formal efficacy analyses were not performed; the reason the study terminated is not reported in the registry record.
The enrollment of 14 is the context for reading the study, not a substitute for an outcome estimate. A response proportion calculated from a small group can be imprecise, but in this record there is no posted response count from which to describe observed efficacy or its precision. It would be inappropriate to infer either that responses did or did not occur from the absence of an analysis. The listed secondary outcomes likewise do not establish that progression-free survival, overall survival, disease control, or biomarker results were formally evaluated.
Because the study was single-group and non-randomized, even a reported response proportion would describe outcomes among participants in the pazopanib arm rather than estimate a contrast against an untreated or alternative-treatment group. Differences in patient characteristics, disease course, assessment practices, or other factors could not be separated through randomization in this design. There is therefore no basis for a causal treatment comparison in the registry results.
The safety record contains counts for adverse events in the Pazopanib 800 mg group. One participant is counted as having a serious adverse event, and 13 participants are counted in the category of other adverse events; these categories should be read according to the registry's separate labels. Hypertension is listed among the top serious adverse event terms at 7.1%. The record does not report a death count, so the null entry should not be interpreted as evidence that no deaths occurred.
Overall, the posted record supports a description of design, enrollment, endpoint definitions, and selected safety counts. It does not provide a formal primary efficacy result or a controlled comparison. These distinctions matter: absence of an efficacy analysis is not a finding of no effect, and safety counts in a small single-arm study do not establish comparative risk.
9. Statistical Methods Explained
Statistical interpretation begins with the design. This was a single-group, non-randomized study, so its primary endpoint is a response proportion within the treated group, not a difference between randomized arms. The registry record reports no formal efficacy analysis. It therefore supplies neither a calculated response estimate nor an inferential result for the primary endpoint. The concepts below are general explanations of how such analyses are usually interpreted; they do not describe methods confirmed for this study unless stated explicitly.
What a response proportion describes
In general, a response proportion is the number of participants meeting the endpoint definition divided by the number of participants included in the analysis population. Here the endpoint combines confirmed complete and partial responses, with the confirmation and tumor-measurement rules stated in the registry. The record identifies the All Treated Population as the primary-outcome population, defined as participants meeting inclusion criteria and consenting to participate. It does not report the number of participants in that population with a confirmed response, so the proportion cannot be reconstructed from the posted primary results.
A confidence interval is a range of values that expresses statistical uncertainty around an estimate under specified assumptions. For a response proportion, the interval would communicate how much uncertainty accompanies the observed proportion. The registry does not report a primary response estimate or confidence interval. General statistical caution is especially important when an enrolled group is small: each observed outcome can have a substantial influence on the estimated proportion, and the uncertainty may be wide. No interval should be inferred without the underlying response count and an analysis method.
Why the design limits inference
Randomization helps make treatment groups comparable, on average, with respect to measured and unmeasured factors. This study had no random allocation and no comparator arm, so it cannot estimate a randomized treatment contrast. A single-group response result, if reported, would describe observed outcomes in this enrolled group. It would not by itself show what would have happened to the same participants under another treatment or without the study intervention.
Open-label conduct means the study was not masked. In general, knowledge of treatment assignment can influence assessments or participant and investigator behavior, depending on the outcome and assessment process. The registry does not report detailed safeguards for response assessment, such as independent review, so those procedures should not be assumed.
Confirmation and outcome timing
The stated response definitions require confirmation at 4 weeks. In general, confirmation can reduce the influence of a transient or unverified change on response classification. The primary timeframe is baseline through the end of study, up to 2 years. That timeframe does not indicate that every participant had the same length of observation. The record does not report individual follow-up duration, the schedule of tumor assessments, or how assessments after withdrawal or early termination were handled.
Progression-free survival and overall survival are listed as secondary outcomes. General time-to-event analyses account for the timing of events and may also require rules for censoring participants whose event status is not observed by the end of follow-up. The registry record does not report event counts, estimates, confidence intervals, censoring rules, or formal analyses for those outcomes. No conclusions about survival results can be drawn from their listing alone.
Multiplicity, missingness, and analysis populations
When a study evaluates multiple outcomes or makes multiple statistical tests, multiplicity can increase the chance of at least one apparently unusual result arising by chance. A prespecified hierarchy or adjustment can help govern interpretation. The registry lists several secondary outcomes, but it does not report a multiplicity strategy or a complete testing plan. The absence of a stated strategy does not establish that no plan existed outside the registry; it means the strategy is not reported in the registry record.
Missing outcome data can affect an estimate if participants without assessments differ systematically from those with observed results. General methods may use explicit rules for missing assessments, but the registry does not report the study's missing-data approach or the number of missing primary assessments. It also does not report whether participants who discontinued early were treated as non-responders, excluded, or handled under another rule. These choices cannot be filled in by assumption.
The primary population is named as the All Treated Population. The record does not define additional efficacy populations or explain how deviations from eligibility or treatment exposure were handled. It is therefore important not to substitute a different analysis population for the one named, or to imply that a complete-case or intention-to-treat analysis was performed when that is not reported.
Planning calculations are not observed power
The sample-size assumptions describe planning illustrations using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with effects framed as fractions of an observed effect. The assumptions state that time-to-event illustrations count total events and other illustrations count participants per arm. These are planning concepts, not observed power for this single-group trial. No randomized two-arm comparison is described by this study, and the registry's early termination meant no formal primary efficacy analysis was performed. A planning calculation cannot supply the missing outcome estimate or establish the probability that the study detected an effect.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists multiple outcomes, including response and stable disease, progression-free survival, overall survival, and biomarker measures. When several outcomes are analyzed, multiplicity is a general concern: repeated statistical testing can increase the chance of a nominally notable result occurring by chance. A prespecified primary endpoint can help focus interpretation, while a testing hierarchy or adjustment can govern claims across multiple outcomes. The registry record does not report a multiplicity strategy, testing hierarchy, or detailed statistical analysis plan. It does state that no formal efficacy analyses were performed due to early termination.
The primary analysis population is named as the All Treated Population, defined as those who met inclusion criteria and consented to participate. The registry does not report the number with evaluable primary assessments, the number with missing response measurements, or how missing and unevaluable results were to be handled. It also does not report analysis populations for the secondary outcomes. Missingness can affect an estimate when participants without measurements differ from those with measurements; general statistical practice therefore requires explicit reporting of both missing data and the analysis rule. No such rule should be assumed.
The record lists 0 analyses, and it does not provide formal primary efficacy results. The registry does not report an interim-analysis plan or whether interim monitoring was intended. The study's termination reason is also not reported in the registry record. These gaps limit assessment of how outcomes were selected, handled, and interpreted, but they do not establish that any particular analysis or procedure was used outside the information recorded.
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 |
|---|---|---|---|
| Pazopanib 800 mg | 1 | 14 | 7.1% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Pazopanib 800 mg | 13 of 14 |
Most frequent serious adverse event terms
| Event term | Organ system | Pazopanib 800 mg |
|---|---|---|
| Hypertension | Vascular disorders | 1 of 14 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Pazopanib 800 mg |
|---|---|---|
| Fatigue | General disorders | 8 of 14 |
| Diarrhea | Gastrointestinal disorders | 6 of 14 |
| Nausea | Gastrointestinal disorders | 6 of 14 |
| Constipation | Gastrointestinal disorders | 4 of 14 |
| Dyspepsia | Gastrointestinal disorders | 4 of 14 |
| Vomiting | Gastrointestinal disorders | 4 of 14 |
| Anorexia | Metabolism and nutrition disorders | 4 of 14 |
| Stomatitis | Gastrointestinal disorders | 3 of 14 |
| Headache | Nervous system disorders | 3 of 14 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 3 of 14 |
Serious events with the highest arm-level rate: hypertension (7.1%).
The registry lists a Pazopanib 800 mg group with 14 participants at risk for serious adverse events and 1 participant affected. For other adverse events, it lists 14 participants at risk and 13 affected. These are separate registry categories; the counts should not be added or treated as mutually exclusive unless the record says how overlap was handled, and that detail is not reported in the registry record.
Hypertension is listed among the top serious adverse event terms at 7.1%. This term-level percentage is distinct from the group-level count of participants with a serious adverse event. The record does not provide enough detail to infer the number of events per participant, timing, duration, severity grading, or whether the listed term accounts for all serious adverse events. A listed adverse event does not by itself establish that the study intervention caused it.
The deaths entry is null. A null entry is not a reported zero; the death count is not reported in the registry record. These safety figures describe counts in a small, single-arm study and do not provide a comparison with another treatment or background event rates. They should therefore be read as descriptive registry data, not as a comparative estimate of risk.
12. What the Result Does, and Does Not, Mean
The record establishes that a Phase II study of pazopanib monotherapy enrolled 14 participants with advanced non-small cell lung cancer and was terminated. It also documents the intended primary response endpoint, its definitions, the primary population, and selected adverse-event counts. These registry entries describe the study and the information recorded about it; they do not amount to a formal efficacy finding.
The primary question was whether participants achieved a confirmed complete or partial response. The registry explicitly states that no formal efficacy analyses were performed because the study terminated early, and it reports no response estimate. This absence must not be converted into a conclusion that no participant responded, or that the intervention did or did not have an effect. The reason for termination is not reported in the registry record, and no formal efficacy conclusion can be inferred from termination alone.
Even if a response proportion had been reported, the single-group, non-randomized design would limit interpretation to outcomes observed in the treated participants. There was no concurrent control group to estimate what would have happened under another treatment or without the study intervention. Without randomization, observed outcomes cannot be interpreted as a causal contrast.
The safety counts provide descriptive information but not a comparative safety estimate. The null death entry is not evidence of zero deaths, and the listed hypertension term does not establish causality. The study's enrollment and early termination also mean the record cannot establish how precisely outcomes would be estimated under a larger or longer study.
Thus, the registry record is informative about design, endpoint definitions, enrollment, and recorded safety categories, while leaving the primary efficacy question unanswered. The appropriate statistical reading is bounded: distinguish an unreported or unanalyzed result from a negative result, and distinguish an observed count from a treatment effect.
13. Why This Trial Matters Statistically
This trial matters statistically because it illustrates the difference between a clearly defined endpoint and an analyzable result. The registry specifies a primary response question, defines complete and partial response, identifies the analysis population, and sets an observation window. Yet it also states that no formal efficacy analyses were performed due to early termination. Endpoint detail is useful, but it cannot replace an observed estimate and an account of its uncertainty.
The design also highlights why a single-arm result has a narrower interpretation than a randomized comparison. A response proportion can describe what happened in the enrolled group, but a control group and random allocation are needed for a direct randomized contrast. Because this study was non-randomized and single-group, the registry cannot support a comparative effect estimate, even if the primary response result had been posted.
The safety record offers another statistical lesson: counts, percentages, and missing entries are not interchangeable. The count of participants with serious adverse events, a term-level percentage for hypertension, and a null death entry each convey different information. Careful reading avoids treating the null as zero or treating a term listed among serious events as the complete safety profile.
Finally, the registry demonstrates why planned outcomes and completed analyses must be distinguished. Listing survival and biomarker outcomes does not show that their results were analyzed. Likewise, a planning illustration is not an observed power calculation or a substitute for trial results. A transparent account of what was measured, what was analyzed, and what remains unreported is essential for responsible interpretation.
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 consequential limitation is the absence of a formal efficacy analysis. The registry does not report the number of complete or partial responses, a response percentage, or an uncertainty interval. It states that early termination prevented formal efficacy analyses, but the reason for termination is not reported in the registry record. The primary endpoint therefore cannot be quantified from the posted results.
The study enrolled 14 participants and had no comparator arm. The small enrolled group limits the precision that could be achieved even if outcomes were available, while the single-group, non-randomized design prevents a randomized comparison. Open-label conduct is also recorded, but the registry does not report detailed response-assessment procedures or whether review was independent.
The registry's planned timeframe extended up to 2 years, but it does not report participant-level follow-up, completeness of assessments, or the handling of participants who discontinued. The missing-data method and analysis populations beyond the named All Treated Population are not reported in the registry record. The reason for early termination, sample-size justification, detailed statistical analysis plan, multiplicity approach, and interim-analysis plan are also not reported.
Several secondary outcomes are listed, but formal results for them are not described in the record. The safety data are limited to the posted group counts and a top serious adverse event term; details such as event timing, recurrence, and the death count are not reported. These limitations mean the record supports a narrow description of study conduct and selected safety information, not a comprehensive efficacy or comparative safety assessment.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Monotherapy Pazopanib in Subjects With Advanced Non-Small Cell Lung Cancer — NCT00549328.
- Registry record last updated 2014-11-19; status last verified 2014-11.
- Results first posted 2010-01-27.
- 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
This registry record describes a Phase II, open-label, non-randomized, single-group study of pazopanib monotherapy in advanced non-small cell lung cancer. Enrollment was 14, and the study is listed as terminated. The primary endpoint was the percentage achieving confirmed complete or partial response, assessed from baseline through the end of study, up to 2 years.
The registry states that no formal efficacy analyses were performed due to early termination. It lists secondary response, survival, and biomarker outcomes, but does not report formal efficacy results for them. The safety group lists 1 participant with a serious adverse event and 13 with other adverse events; hypertension is listed among the top serious adverse event terms at 7.1%. The death count is not reported in the registry record.
With no comparator arm and no formal primary efficacy result, the posted record does not provide a comparative treatment estimate or a quantified answer to the primary response question.