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 Photodynamic Therapy in Treating Patients With Resectable Non-Small Cell Lung Cancer That Has Spread to the Pleura (NCT00601848), a na phase 2 study. The primary outcome is Toxicity Assessment of Pleural Photodynamic Therapy.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00601848 |
| Phase | Phase 2 |
| Condition | Lung Cancer, Metastatic Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 9 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Abramson Cancer Center at Penn Medicine |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients with resectable non-small cell lung cancer that had spread to the pleura. The registry lists lung cancer and metastatic cancer as conditions. It does not report further eligibility details or describe the characteristics of the participants enrolled.
Intervention
The trial examined photodynamic therapy given during surgery. The registry lists porfimer sodium, conventional surgery, chemotherapy, spectroscopy, immunohistochemistry staining, and laboratory biomarker analysis among its interventions. Their precise roles and how they were used are not reported in the registry record.
Comparator
This was a single-group study, not a randomized comparison. The registry describes one arm, called Photodynamic Therapy, and gives its description as PDT. There is no like-for-like control arm, so the study does not directly compare the intervention with another treatment or with no photodynamic therapy.
Primary question
The primary outcome was a toxicity assessment of pleural photodynamic therapy over one year, with toxicities defined by CTCAE v4.0. The study purpose also refers to examining how well treatment works, and progression-free survival and Photofrin® uptake are listed as secondary outcomes. However, primary-outcome data are unavailable, and no outcome analyses are posted.
3. Trial Design
NCT00601848 is a phase II study titled A Phase II Study of Pleural Photodynamic Therapy for Patients With Non-small Cell Lung Cancer and Pleural Spread. It was sponsored by Abramson Cancer Center at Penn Medicine. The registry lists a single-group model, no masking, and allocation as not applicable. Enrollment is listed as 9, and the study status is terminated. The recorded start is 2004-11 and the primary completion is 2012-02.
A single-group design follows participants receiving the intervention of interest without assigning a comparison group. It can describe outcomes observed in that group, but it cannot by itself separate effects associated with treatment from other explanations, such as participants' underlying disease or other care. The registry specifies one arm, Photodynamic Therapy, described as PDT. It does not report a randomized control group or a parallel comparator.
The study rationale describes photodynamic therapy as using a drug, such as porfimer sodium, that is absorbed by tumor cells and activated by light. The stated purpose was to assess side effects and how well photodynamic therapy given during surgery works in patients with resectable non-small cell lung cancer that has spread to the pleura. The registry also lists chemotherapy, conventional surgery, spectroscopy, immunohistochemistry staining, and laboratory biomarker analysis as interventions, but the exact treatment sequence and each intervention's role are not reported in the registry record.
The study was terminated. The registry does not report why it was terminated, whether enrollment reached its intended target, or the reasons participants may or may not have completed follow-up. A sample-size justification, detailed protocol, and statistical analysis plan are not reported in the registry record. The enrollment value describes the number listed in the record; it does not establish that complete outcome data are available for those participants.
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 | Photodynamic Therapy |
|---|---|
| STARTED | 9 |
| COMPLETED | 0 |
| NOT COMPLETED | 9 |
Analysis population for the primary outcome, as defined in the registry: The study was terminated. Despite all possible efforts to contact the PI/study team members, no data are available to be reported
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 | Single Arm |
|---|---|---|
| Participants analysed | 9 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 3 |
| Age, Categorical: >=65 years | Participants | 6 |
| Age, Continuous | years | 71 (SD 10.35) |
| Sex: Female, Male: Female | Participants | 4 |
| Sex: Female, Male: Male | Participants | 5 |
| 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 | 1 |
| Race (NIH/OMB): White | Participants | 7 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Region of Enrollment: United States | participants | 9 |
6. Primary Endpoint
The registry lists the primary outcome as Toxicity Assessment of Pleural Photodynamic Therapy, measured over One year.
| 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 outcome is titled Toxicity Assessment of Pleural Photodynamic Therapy. It is defined as toxicities of PDT under CTCAE v4.0, with a timeframe of one year. Toxicity outcomes are used to characterize unwanted effects observed during or after an intervention. A defined grading framework can make event descriptions more consistent, although the registry does not provide the specific toxicity grades, event counts, or assessment schedule beyond the one-year timeframe.
The registry's primary-outcome description states that the study was terminated and, despite efforts to contact the study team, no data are available to be reported. Consequently, the endpoint's observed results, including the number and severity of toxicities, are not reported in the registry record. The endpoint definition identifies what was intended to be assessed; it does not establish what was observed.
The registry lists progression-free survival and Photofrin® uptake as secondary outcomes. It does not report their definitions, measurement procedures, analysis populations, or results. These outcomes therefore cannot be interpreted as evidence about disease progression or drug uptake. The available record supports describing the intended outcome measures, but not estimating their frequency, distribution, or relationship to treatment.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive fact is that no primary-outcome data are available in the registry record. The intended primary assessment was toxicity of pleural photodynamic therapy, defined using CTCAE v4.0 over one year. The record does not provide toxicity observations or an analysis of this endpoint. Thus, the stated endpoint and timeframe describe what the study intended to assess, not a reported result.
The study is listed as phase II, single group, and terminated, with enrollment of 9. Its single-arm design is important for interpretation. Even if outcome data were available, observations in one group would describe that group; without a concurrent comparator, they would not establish what would have happened under another treatment or without photodynamic therapy. The registry has no randomized comparison, and its own notes caution that the arms are not a like-for-like control comparison.
The adverse-event record lists one group with 9 at risk for serious events and 0 affected, and 9 at risk for other events and 0 affected. It also lists 0 deaths. These are the posted counts, but they need to be read alongside the primary-outcome statement that no data are available to be reported. The record does not explain the relationship between that missing primary endpoint and the adverse-event entries, or provide event definitions, follow-up completeness, or supporting details. The zeros should therefore not be taken as proof that no toxicity occurred or that safety was fully assessed.
No outcome analyses are posted. The record gives no estimates of progression-free survival or Photofrin® uptake, despite listing them as secondary outcomes. There are no reported effect estimates, confidence intervals, or hypothesis-test results for these outcomes. Without an estimate and a measure of uncertainty, neither the magnitude nor the precision of any treatment-associated outcome can be assessed.
Overall, the registry supports a description of the study's question, design, intended outcomes, status, and limited posted safety counts. It does not support a conclusion about the observed toxicity profile or treatment effect. That distinction is especially important in a small, terminated, single-group study with unavailable primary-outcome data: absence of reported results is not itself evidence of either benefit or harm.
9. Statistical Methods Explained
The registry describes a phase II, single-group study with no masking and allocation listed as not applicable. Those design features establish the broad structure, but the statistical methods used for outcome analysis are not reported in the registry record. No analysis results are posted. The explanations below describe general statistical concepts and should not be read as claims about methods used in this study.
What can a single-group design estimate?
In general, a single-group study can summarize what happened among the participants observed. For a binary outcome, an analysis might report the number of participants with an event and the number assessed. For a time-to-event outcome, an analysis might describe how long participants were followed and when events occurred. Which summaries are appropriate depends on the endpoint and the data collected. The registry does not report endpoint-level data or the analysis procedures needed to determine which summaries were conducted.
A comparison of treatment strategies requires a suitable contrast. In general, a concurrent control group can help distinguish differences associated with treatment assignment from changes related to time, disease course, or other care. In a single-group study, those explanations cannot be separated through a randomized between-group comparison. This is a design limitation, not a statistical adjustment that can be resolved from the posted record.
What does uncertainty mean?
In general, a confidence interval describes a range of values compatible with the observed data and the assumptions of a statistical method. Its width reflects precision: less information or greater variability tends to produce less precise estimates. An estimate without an uncertainty measure can give an incomplete picture, while a confidence interval does not eliminate design limitations or establish causation. No outcome estimates or confidence intervals are reported for this study, so its statistical precision cannot be assessed.
A p-value, in general, measures how unusual the data would be under a specified statistical model and null hypothesis. It does not measure the probability that a treatment works, and it does not describe the size or importance of an effect. A p-value also cannot compensate for missing data or an absent control group. The registry does not report hypothesis tests or p-values for the study outcomes.
How should time-to-event outcomes be approached?
Progression-free survival is listed as a secondary outcome, but its definition and results are not reported in the registry record. In general, time-to-event methods account for both the timing of an event and the length of observation. Participants who have not had the event by the end of their observed follow-up may be censored: their eventual event time is unknown, but their observed time still contributes information under the method's assumptions. Reliable interpretation depends on clear event definitions, follow-up procedures, censoring information, and the number of events. Those details are not reported for this outcome.
In general, a hazard ratio can compare event rates over time between groups, but it requires a defined comparison and model assumptions. A single-group design does not provide a randomized treatment contrast. No hazard ratio, survival estimate, event count, or time-to-event analysis is posted for progression-free survival, so none can be described as a result of this study.
Why do event counts and denominators matter?
For toxicity, the number of participants assessed and the number experiencing each event help readers understand the observed frequency. The event definition, severity grading, follow-up duration, and completeness of reporting are also important. A zero count can be informative only in the context of a clear denominator and adequate ascertainment. Here, the adverse-event entries list 9 at risk and 0 affected for serious events, and 9 at risk and 0 affected for other events; the primary outcome separately states that no data are available to be reported. The registry does not clarify this difference or provide event-level detail.
In general, a confidence interval around an event proportion can show how uncertain a frequency estimate is, especially when the number observed is small. The registry does not report such intervals for the safety figures. No new estimate can be reconstructed from the available record without making assumptions about the meaning, completeness, and analysis of those entries.
What is known about planning?
The registry does not report the trial's sample-size justification, its statistical analysis plan, or any interim-analysis plan. Planning calculations are distinct from observed results. In general, an illustrative sample size based on an assumed effect is a planning aid, not observed power and not evidence that a study detected an effect. Any planning illustration associated with the record is described as using a stated fraction of an observed effect, a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; time-to-event illustrations use a total event count. That description is not the design of this study, which is single group, and the registry does not supply an observed effect estimate for interpreting such an illustration.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists five outcome measures, including toxicity assessment as the primary outcome and progression-free survival and Photofrin® uptake as secondary outcomes. The specific definitions and results for the secondary outcomes are not reported in the registry record. No outcome analyses are posted.
Multiplicity arises in general when several outcomes, time points, subgroups, or statistical tests are examined. Testing many hypotheses can increase the chance that at least one result appears statistically notable by chance. A study may address this through a prespecified hierarchy, adjustment, or careful interpretation, but the appropriate approach depends on its design and analysis plan. A multiplicity strategy is not reported in the registry record. There is also no posted set of outcome estimates or tests from which to assess how multiple comparisons were handled.
Missing data require knowing which measurements were planned, which were obtained, and why observations are absent. The primary outcome description states that no data are available to be reported, but the registry does not explain the extent or reasons for missing outcome data, the follow-up status of participants, or whether any imputation or sensitivity analysis was performed. Those methods are not reported in the registry record.
Analysis populations are also not described. The record does not say whether analyses were intended to include all enrolled participants, only those who received the intervention, or another defined group. Enrollment of 9 should not be treated as confirmation that 9 participants contributed complete data to every outcome. Without a reported analysis population and outcome-specific denominators, completeness and selection cannot be assessed.
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 |
|---|---|---|---|
| Single Arm | 0 | 9 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Single Arm | 0 of 9 |
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 |
|---|---|
| Single Arm | 0 of 9 |
The adverse-event entries list a Single Arm group. For serious adverse events, 9 participants are listed as at risk and 0 as affected. For other adverse events, 9 are listed as at risk and 0 as affected. The entries also list 0 deaths, and no top serious-event terms are listed.
These figures are registry entries, not a detailed safety analysis. The record does not define the event categories further, describe event severity beyond the distinction between serious and other events, or provide event-level narratives. It also does not explain the completeness of follow-up or how the entries relate to the primary-outcome statement that no data are available to be reported.
Accordingly, the reported zero counts should be stated as posted, but they do not establish that no adverse events occurred or that toxicity was fully assessed. The primary outcome was a one-year toxicity assessment under CTCAE v4.0; outcome data for that assessment are unavailable. The safety entries and the missing primary-outcome information together leave the completeness and interpretation of safety reporting uncertain.
12. What the Result Does, and Does Not, Mean
The registry record identifies a phase II study of pleural photodynamic therapy during surgery for resectable non-small cell lung cancer with pleural spread. It describes a single-group design, enrollment of 9, and a primary goal of assessing toxicity over one year using CTCAE v4.0. It also lists progression-free survival and Photofrin® uptake as secondary outcomes. These registry entries establish the study's intended scope, but they are not outcome findings.
The primary outcome description says that the study was terminated and that no data are available to be reported. The registry posts no outcome analyses. As a result, readers cannot estimate toxicity, progression-free survival, or Photofrin® uptake from this record, and cannot judge the precision of any treatment-related estimate. The adverse-event entries show 0 affected for serious and other events among 9 listed at risk, with 0 deaths, but the record does not reconcile these entries with the missing primary-outcome data. They should not be interpreted as demonstrating an absence of toxicity.
The design also limits what the study could establish. With one group and no randomized comparator, observed outcomes would not provide a direct estimate of how participants would have fared under another treatment. Even complete single-group observations would need cautious interpretation; unavailable outcome data make conclusions still more limited.
These limitations do not mean the study had no scientific purpose. An assessment of feasibility and toxicity can be an important part of evaluating an intervention, and the registry records the intended clinical question and outcome framework. However, the posted record does not permit a conclusion about treatment benefit, treatment harm, or comparative effectiveness. It documents the study design and a lack of reportable primary-outcome data rather than an interpretable estimate of the intervention's effect.
13. Why This Trial Matters Statistically
This study illustrates a basic distinction in clinical research: a trial's question and design are not the same as its result. The registry describes an intervention, a target population, a single-group structure, and a primary toxicity outcome. Yet the primary-outcome description reports no data available, and no analyses are posted. Statistical interpretation depends on observed outcome data as well as the design; an endpoint definition alone cannot show what happened.
It also illustrates why denominators and reporting context matter. The safety entries list 9 at risk and 0 affected for serious events, and the same at-risk and affected counts for other events. But the record does not explain how those entries relate to its statement that primary-outcome data are unavailable. Statistical readers should preserve that distinction rather than convert a posted zero into a claim of no toxicity.
The single-group design is another useful teaching point. A study can describe outcomes within its participants, but a randomized comparator is needed for a direct randomized contrast. Without that contrast, differences from other populations or historical expectations may reflect factors besides treatment. This limitation remains even when an outcome is measured consistently.
Finally, a terminated study with a small listed enrollment and unavailable primary-outcome data demonstrates why precision, missingness, and analysis populations are central to evidence appraisal. The registry does not report a sample-size justification, analysis plan, or outcome estimates. That absence prevents evaluation of statistical uncertainty and limits the conclusions that can responsibly be drawn from the record.
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 registry lists the study as terminated but does not report the reason for termination. It records enrollment of 9, yet the primary-outcome description states that no data are available to be reported. The record does not explain whether outcome data were never collected, could not be obtained, or were unavailable for another reason. The extent and pattern of missing information therefore cannot be assessed.
The study has a single-group design, with no randomized control arm. This means there is no direct randomized comparison for interpreting outcomes. The registry does not provide further design details such as eligibility criteria, participant characteristics, treatment timing, follow-up procedures, or the exact roles of all listed interventions.
The primary outcome is described as toxicity under CTCAE v4.0 over one year, but no toxicity results, grades, or endpoint-level denominators are reported. The adverse-event entries list zero affected participants, while the primary-outcome description says no data are available; the record does not clarify this apparent difference. Progression-free survival and Photofrin® uptake are listed as secondary outcomes without definitions or results.
A statistical analysis plan, sample-size justification, multiplicity approach, analysis populations, and methods for handling missing data are not reported in the registry record. There are no posted outcome estimates or uncertainty measures. These omissions prevent assessment of analysis choices, precision, and the extent to which the posted entries represent complete follow-up.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Photodynamic Therapy in Treating Patients With Resectable Non-Small Cell Lung Cancer That Has Spread to the Pleura — NCT00601848.
- Registry record last updated 2020-11-24; status last verified 2020-04.
- Results first posted 2020-11-24.
- 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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Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
NCT00601848 is a phase II, single-group study of pleural photodynamic therapy for patients with resectable non-small cell lung cancer and pleural spread. The registry lists Abramson Cancer Center at Penn Medicine as sponsor, enrollment of 9, a start of 2004-11, primary completion of 2012-02, and status as terminated.
The primary outcome was toxicity assessment over one year under CTCAE v4.0. The record states that no primary-outcome data are available to be reported, and no outcome analyses are posted. Progression-free survival and Photofrin® uptake are listed as secondary outcomes without reported results. The adverse-event entries list 0 affected for serious and other events among 9 at risk, and 0 deaths; their relationship to the unavailable primary-outcome data is not explained.
The single-group design provides no randomized comparator. The registry does not report the reason for termination, a sample-size justification, a statistical analysis plan, or analysis populations.