DBT for Metastatic Lung Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for DBT for Metastatic Lung Cancer (NCT04973436), a na na study. The primary outcome is Feasibility as Measured by Study Accrual.

2. Clinical Question

3. Trial Design

This completed study, sponsored by Duke University, is registered as NCT04973436 and titled DBT for Metastatic Lung Cancer. The registry lists its phase as NA, allocation as NA, masking as none, and model as single group. These entries describe a pilot without a randomized control arm; they do not establish a comparative treatment effect.

The study description outlines an adaptation effort followed by a pilot. Phase I aimed to use focus groups and interviews with metastatic lung cancer patients, thoracic oncology providers, and clinicians with relevant survivorship and behavioral symptom-management expertise. The described aims include adapting the skills-training material and preparing a manualized protocol called LiveWell. Phase II aimed to pilot test LiveWell for feasibility, acceptability, and pre-to-post intervention outcomes. The registry summary describes planned participant counts for those activities, while the overall recorded enrollment is 31. The record does not explain how that enrollment is divided across study activities.

The intervention is listed as behavioral. The primary outcome is study accrual, and secondary outcomes include fatigue, dyspnea, pain severity and interference, emotion regulation, intolerance of uncertainty, and DBT skill use. The registry lists 13 outcome measures but records 0 analyses. The study start date is 2022-05-18, the primary completion date is 2024-03-11, and the status is completed. Completion status indicates the registry’s study status; it does not substitute for outcome results.

With no control group and no masking recorded, changes over time cannot be separated from other influences using a concurrent comparison. The registry record does not report a detailed statistical analysis plan, the conduct of any interim analyses, or a sample-size justification for the study’s own 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: One participant was lost to contact prior to baseline assessment.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Individuals who were eligible for consent.

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.

6. Primary Endpoint

The registry lists the primary outcome as Feasibility as Measured by Study Accrual, measured over Post-intervention (approximately 8 weeks).

The primary outcome is feasibility measured by study accrual. It is defined as a count of participants among individuals eligible for consent, with a stated target of N = 30 for the single-arm pilot. The timeframe is post-intervention, approximately 8 weeks. This is a process outcome: it asks whether the study enrolled enough eligible participants according to its stated target, rather than whether symptoms or other clinical measures changed.

Accrual is used in a pilot because the ability to recruit participants is relevant to whether a larger or further study could be conducted. In general, a target-based accrual measure is interpreted against the target and the outcome’s defined population. The registry records total enrollment of 31, but does not report the primary outcome count or clarify whether total enrollment corresponds to the eligible-for-consent population used for the endpoint. Therefore, enrollment alone cannot establish the reported primary endpoint result.

The registry description presents feasibility as the primary focus and also names acceptability and pre-to-post intervention outcomes among the pilot aims. It does not report an endpoint estimate, uncertainty interval, or test result for accrual. Without those results, the endpoint’s observed performance relative to its target is not available in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical interpretation depends first on the design and the quantity being measured. This is a single-group pilot, and its listed primary outcome is an accrual count. The registry record does not report an inferential analysis for that outcome or for the other outcomes. The concepts below explain how such data are generally approached; they should not be read as a description of analyses conducted in this study.

What does a count measure?

A count records how many participants meet a definition, rather than how far a continuous score changes. For this primary outcome, the count is intended to describe accrual among individuals eligible for consent, in relation to a stated target of N = 30. The registry’s overall enrollment figure is 31, but an overall enrollment total and the endpoint count need not represent the same defined population. The endpoint result is not reported in the registry record.

In general, a target-based feasibility outcome can be reported by giving the observed count, the target, and a clear definition of who was included. If uncertainty is relevant, an interval can describe the range of population values compatible with the observed data under stated assumptions. A confidence interval is not a guarantee that the true value lies inside its bounds; it is a measure of estimation uncertainty under the method used. No interval or statistical method for the accrual outcome is reported in the registry record.

Why does the single-group design matter?

A single-group study observes participants receiving the intervention without a concurrent control group. If an outcome is measured before and after intervention, a change score describes the difference across those assessments for the measured participants. It does not, by itself, identify what would have happened over the same period without the intervention. In general, a randomized control group can help distinguish intervention-related differences from changes that may occur over time or for other reasons. This study has no such comparison group, and the registry does not report randomized allocation.

The record lists masking as none. Masking, in general, is intended to limit the influence of knowledge about treatment assignment on care, reporting, or assessment. In a single-group study there is no treatment assignment comparison to conceal. The registry does not describe additional procedures for reducing expectation or measurement effects.

How should change measures be understood?

The listed secondary measures cover fatigue, dyspnea, pain severity and interference, emotion regulation, intolerance of uncertainty, and DBT skill use. Each outcome represents a different construct, and an interpretation would depend on its scoring rules, assessment times, and direction of favorable change. The registry names instruments for several outcomes, including the Fatigue Symptom Inventory, Brief Pain Inventory-Short Form, Emotion Regulation Scale, Intolerance of Uncertainty Short Form, and DBT Ways of Coping Checklist skills subscale. Results and scoring details are not reported in the registry record.

For continuous outcomes, general summaries may include a mean or median, a measure of spread, and an estimate of change. The spread describes variation among observations; an interval around an estimate describes uncertainty about the estimated quantity. For counts of participants with events, the numerator and the population at risk should be clearly defined. These principles do not imply that any particular summary or model was used in this trial.

What can multiple outcomes tell us?

The registry lists 13 outcome measures. When multiple outcomes are examined, there are more opportunities to observe an apparently notable result by chance if each is tested separately. In general, a multiplicity strategy may prioritize a primary outcome, adjust inference across a family of tests, or treat secondary findings as exploratory. The registry record does not report a multiplicity procedure, a hierarchy among secondary outcomes, or outcome-specific analysis methods. It also records 0 analyses, so no multiplicity-adjusted or unadjusted findings are available.

Over-dispersion is a general issue for count data: observed variability may exceed what a simple count model assumes. When relevant, an analysis may use a model that accommodates the extra variability or report a robust uncertainty estimate. The registry does not report a model for the accrual count, and its stated outcome is a target-based count rather than a reported modeled rate. There is no basis to claim that over-dispersion was assessed or that a particular count model was used.

Finally, the registry does not report the study’s own sample-size justification, missing-data method, analysis populations, interim analyses, or detailed statistical plan. The absence of these details limits assessment of how measurements were handled and how results would have been interpreted. It does not establish that a particular method was or was not used outside what the registry record reports.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("LiveWell: A Dialectical Behavioral Therapy Skills ")
n      <- c(34)
events <- c(31)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  LiveWell: A Dialectical Behavioral Therapy Skills   34  31  91.2  76.3  98.1  77.0  97.0  76.9  96.8

11. Multiplicity, Missing Data and Analysis Populations

The registry lists 13 outcome measures, including the primary accrual measure and outcomes spanning symptoms, emotion regulation, uncertainty, and DBT skill use. It records 0 analyses. A multiplicity plan—how inference is handled when several outcomes are considered—is not reported in the registry record. In general, multiple outcome tests can increase the chance of finding an apparently notable result by chance; a prespecified hierarchy or adjustment can help define how results should be interpreted. No such procedure can be attributed to this study from the registry entry.

Missing-data procedures are not reported in the registry record. The record does not explain whether participants missed assessments, how incomplete questionnaires were handled, or whether any imputation or other method was planned. Analysis populations are also not reported. In particular, the endpoint refers to individuals eligible for consent, while the record’s enrollment total is not identified as the endpoint analysis population.

The record does not report the study’s detailed statistical analysis plan, interim analyses, or its own sample-size justification. Without these details and without posted outcome analyses, it is not possible to assess how missing observations, multiple outcomes, or analysis populations affected the results. These are reporting gaps, not evidence that any specific problem occurred.

12. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

The registry lists one LiveWell group for adverse-event reporting. It records 0 participants with a serious adverse event among 30 at risk and 0 participants with other adverse events among 30 at risk. It also records 2 deaths. These are separate reported counts; the registry does not say that the deaths were intervention-related, and no causal attribution should be inferred from the counts alone.

The at-risk counts describe the denominators recorded for the adverse-event entries. They do not establish that every participant had the same observation period or that all events were captured in the same way. The registry does not provide further detail on event definitions, timing, causes of death, follow-up duration, or the relationship of events to the intervention. No serious adverse-event terms are listed.

In general, safety counts are most informative when interpreted alongside event definitions, exposure and follow-up information, and a clear account of attribution. Those details are not reported in the registry record. The posted counts should therefore be treated as limited descriptive safety information, not as a comparative safety analysis.

13. What the Result Does, and Does Not, Mean

The registry record describes a completed pilot of a behavioral skills-training protocol for people with metastatic lung cancer. Its primary focus was feasibility measured through accrual, with a stated target of N = 30. The registry records enrollment of 31 but does not report the primary endpoint result or clarify that the enrollment total is the eligible-for-consent count used by the endpoint definition. The record therefore does not establish whether the primary feasibility target was met according to that measure.

The study description also names acceptability and pre-to-post outcomes as aims, and lists measures related to symptoms, emotion regulation, uncertainty, and DBT skill use. Because the registry records 0 analyses and provides no outcome estimates, it does not show the observed direction or size of any change. It also provides no uncertainty intervals with which to judge precision.

The study has no control group. Even if change estimates were available, a single-group pre-to-post comparison would not establish that any change was caused by LiveWell. The registry information also cannot establish comparative effectiveness, because it includes no like-for-like control arm.

The adverse-event entries are descriptive counts, including 2 deaths, with no reported attribution or further contextual detail. They do not establish whether events were caused by the intervention. In short, the record documents the pilot’s purpose and design and includes limited enrollment and safety information, but it does not supply analyzed efficacy, feasibility, or safety conclusions.

14. Why This Trial Matters Statistically

Pilot studies often address whether a larger study can be conducted and whether an intervention can be adapted for a particular population. This study’s stated aims included adapting DBT skills training for metastatic lung cancer patients and pilot testing LiveWell. Its primary outcome—accrual against a stated target—reflects a practical feasibility question rather than a clinical effectiveness comparison.

That distinction matters statistically. A count of enrolled participants can inform recruitment only when it matches the endpoint’s population definition and is reported as the endpoint result. Here, the registry records overall enrollment of 31 but does not report the accrual outcome among individuals eligible for consent. The record therefore leaves the primary feasibility result unresolved.

The single-group design also sets a limit on what the study can establish. It may be suitable for describing a pilot process, but it cannot estimate a controlled difference between LiveWell and an alternative. The registry’s 0 analyses means that the named symptom and behavioral outcomes cannot be used to judge observed change or its precision from this record.

For biostatisticians and clinical researchers, this example highlights the value of reporting endpoint definitions alongside observed results, analysis populations, missing-data handling, and planned methods. These details are especially important when a pilot has several outcomes and no concurrent comparator. Completion status and enrollment are useful study descriptors, but they are not substitutes for outcome estimates or a transparent account of their uncertainty.

15. 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 principal limitation for interpretation is that the registry lists 0 analyses and does not report the primary accrual result or any secondary outcome estimates. Although total enrollment is recorded as 31 and the primary target is N = 30, the endpoint is defined for individuals eligible for consent. The relationship between the total enrollment figure and that endpoint population is not reported in the registry record.

The single-group design has no control arm, so it cannot support a controlled comparison. The registry lists masking as none and allocation as NA. These features limit interpretation of any pre-to-post changes as intervention effects, even if such estimates were available. No outcome changes or causal effects can be inferred from the listed aims alone.

The record lists 13 outcome measures but does not report analysis methods, a multiplicity approach, missing-data procedures, analysis populations, or an interim-analysis plan. It also does not report a sample-size justification for the study’s own design. The summary describes planned adaptation and pilot activities, but does not provide enough information to determine how participants and measurements were distributed across those activities.

Safety reporting is also limited. The registry gives event counts and numbers at risk, but does not report event timing, causes, intervention attribution, or further follow-up context. The 2 deaths therefore cannot be interpreted as intervention-related or unrelated from the posted information. These reporting limitations restrict conclusions; they do not demonstrate that a particular outcome or safety issue occurred.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: DBT for Metastatic Lung Cancer — NCT04973436.
• Registry record last updated 2025-04-27; status last verified 2025-04.
• Results first posted 2025-04-27.
• 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.

19. Record Summary

NCT04973436, titled DBT for Metastatic Lung Cancer, is a completed, single-group study sponsored by Duke University. The registry describes adaptation and pilot testing of LiveWell, a dialectical behavioral therapy skills-training protocol for people with metastatic lung cancer. It records enrollment of 31, with study dates of 2022-05-18 and 2024-03-11 for start and primary completion.

The primary outcome is study accrual, with a stated target of N = 30 and a post-intervention timeframe of approximately 8 weeks. The registry lists 13 outcome measures and 0 analyses. It records 0 serious adverse events among 30 at risk, 0 other adverse events among 30 at risk, and 2 deaths in the LiveWell group. The primary endpoint result, outcome analyses, and further context for the safety counts are not reported in the registry record.