Autologous Stem Cell Treatment for Chronic Lung Disease Study: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Autologous Stem Cell Treatment for Chronic Lung Disease Study (NCT03044431), a registered phase not stated study. The primary outcome is Change in FEV1 From Baseline Among COPD Patients.

2. Clinical Question

In total, 207 participants were enrolled. The record identifies COPD and interstitial lung disease as the conditions of interest, but does not report how enrollment was distributed across those diagnoses. It also does not report a control group against which any observed change could be compared. The clinical question is therefore primarily descriptive: what change in FEV1 was recorded among the small number of COPD participants who completed and returned follow-up testing, and what adverse-event counts were reported for the treated group? The registry record does not include a numerical FEV1 result or an analysis estimating that change.

3. Trial Design

The registry record describes a completed study sponsored by Lung Institute, with 207 participants enrolled. The recorded study start was 2016-04-01, and primary completion was 2017-06-01. The intervention is listed as a procedure called cell therapy. The arm description says that all enrolled participants would undergo cell therapy; no control arm is described in the available record.

This is not a randomized comparison. Allocation is not stated, and the record identifies the arms as not being a like-for-like control comparison. Consequently, the record does not support a treatment-versus-control estimate. Any change from baseline, if reported, would be a before-and-after description rather than a randomized estimate of the difference attributable to treatment. Changes over time could also reflect factors not described in the record.

The primary endpoint concerns change in FEV1 from baseline among participants with COPD. Measurements were planned before treatment and at six months after treatment. The registry says all patients with COPD were asked to provide a six-month pulmonary function test, but only 5 participants completed and returned the test. The number of COPD participants asked to complete testing is not reported in the registry record.

The study summary describes harvesting a patient's own cells and platelet-rich plasma through venous or bone marrow collection techniques, followed by same-day return of the product. Beyond that summary, details such as procedural standardization, co-interventions, follow-up procedures, and criteria for participation are not reported in the registry record. The record lists seven outcome measures and no recorded analysis entries. It does not state a formal sample-size justification, a prespecified statistical analysis plan, or interim analyses. The study's completed status does not by itself establish that each planned measurement or analysis was completed.

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: No patients in the study received placebo. All patients received autologous, adult stem cell therapy.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: All patients with COPD were asked to provide a 6 month post-treatment pulmonary function test. 5 participants completed and returned the test.

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.

Population: All patients who underwent cell therapy were included in the data analysis.

6. Primary Endpoint

The registry lists the primary outcome as Change in FEV1 From Baseline Among COPD Patients, measured over Measurements pre-treatment and then at 6 months post- treatment.

The primary endpoint is change in FEV1 from baseline among participants with COPD, measured by pulmonary function testing or spirometry. The planned assessment points were before treatment and six months after treatment. The registry identifies the outcome unit as percentage of change in FEV1 and names the summary parameter as the mean, with full range as the dispersion measure.

FEV1 is commonly used in respiratory research because spirometry provides a quantitative measure of airflow during a forced exhalation. As a general statistical point, measuring the same outcome at baseline and follow-up can describe how that measure changes over time in the people assessed. It does not, on its own, establish what would have happened without the intervention. A comparator group and an appropriate analysis would be needed to address that causal question more directly.

The registry says all participants with COPD were asked to return a six-month test, but only 5 completed and returned it. No numerical baseline value, follow-up value, mean change, range, or confidence interval is reported in the registry record. It is therefore not possible to describe the direction or magnitude of the recorded FEV1 change from the posted results. Nor can the precision of an estimate be assessed from the information recorded.

The endpoint is specific to the COPD subgroup and should not be treated as an outcome for all 207 participants or for participants with interstitial lung disease. The number of COPD participants in the study and the characteristics of those who returned testing are not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a mean change describe?

The registry identifies the primary endpoint as mean change in FEV1, expressed as percentage of change, from baseline among participants with COPD. In general, a mean summarizes the average value in the analyzed observations. For a before-and-after endpoint, a mean change would summarize the individual changes for participants with measurements at both time points, if that is how the analysis were defined. The registry record does not state the exact calculation rule or analysis set.

A mean can be informative, but it does not show how much individual responses vary. The registry names full range as the dispersion measure. In general, a range describes the smallest and largest observed values, making it sensitive to extreme observations and not a complete description of the distribution. Here, the record does not provide the mean or range values, so neither the average nor the observed spread can be evaluated.

What would a confidence interval add?

General explanation: a confidence interval expresses the statistical uncertainty around an estimate under the assumptions of the analysis. A narrower interval indicates greater precision, while a wider interval indicates less precision. It does not guarantee that the true value lies within the interval, and it does not resolve problems such as missing follow-up or lack of a comparison group.

No confidence interval for FEV1 change is reported in the registry record. Because no numerical estimate or interval is posted, the precision of the endpoint cannot be judged. The small number of returned tests is relevant to how much information the follow-up sample can offer, but it is not a substitute for a reported estimate and uncertainty interval.

Why does the design affect interpretation?

General explanation: random allocation can make treatment groups more comparable on average, while a concurrent control group helps distinguish change over time from change associated with an intervention. This record does not describe random allocation or a like-for-like control group. The intervention arm includes participants who underwent cell therapy, so the registry results do not provide a direct treatment-versus-control contrast.

In an uncontrolled before-and-after study, a change from baseline is not automatically attributable to treatment. Other influences and measurement variation can contribute to observed change. The record does not describe analyses that would address these issues. No treatment-effect estimate can be recovered from the information posted.

How should planning illustrations be read?

General explanation: a sample-size calculation can illustrate how many participants might be needed under stated assumptions to detect a specified effect with a chosen statistical procedure. It is a planning exercise, not an observed result and not a measure of the probability that a completed study detected an effect. The record's planning assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the target effect defined as a fraction of the observed effect.

Those assumptions do not describe the study's own design: the registry record describes all participants as receiving cell therapy and does not identify a like-for-like control arm. Planning illustrations therefore should not be mistaken for evidence that the study was randomized, that the assumed allocation occurred, or that a formal sample-size justification was used. The record does not report such a justification. No numerical planning results are stated in the registry record, and no sample-size illustration can replace the missing primary outcome estimate.

What is the role of hypothesis tests?

General explanation: a hypothesis test evaluates how compatible observed data are with a specified null model under stated assumptions. A p-value is not the probability that a treatment is effective or ineffective, and it does not describe the size or practical importance of an effect. Interpretation ordinarily considers an effect estimate and its uncertainty as well as the test result.

The registry record lists no analysis entries and reports no p-value or test for the primary endpoint. No inferential conclusion can be drawn from a significance test because no such result is posted. The absence of a reported test does not mean that a test found no difference; the result is simply not reported in the registry record.

Why does missing follow-up matter?

General explanation: when outcome measurements are missing, analyses may represent only people with observed data unless a method for handling missingness is specified. If participants with and without follow-up differ in ways related to the outcome, complete-case results may not represent the full eligible group. Whether such differences occurred cannot be determined from the registry record.

For the primary endpoint, 5 participants completed and returned the six-month test. The record does not report the number of COPD participants asked, the reasons for missing tests, or an approach to missing data. It also does not state whether baseline measurements were available for all participants who returned a test. These omissions limit assessment of both the representativeness of follow-up and the statistical analysis.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists seven outcome measures, while the primary endpoint is change in FEV1 among participants with COPD. A study that examines multiple outcomes can raise a multiplicity issue: in general, the more statistical tests considered, the greater the chance of obtaining at least one apparently unusual result by chance if each is judged separately. Whether that issue was addressed depends on the analysis plan and the role assigned to each endpoint.

A multiplicity strategy, including any hierarchy of outcomes or adjustment to statistical tests, is not reported in the registry record. There are no analysis entries, p-values, or confidence intervals posted for the outcomes. It is therefore not possible to assess how multiple outcomes were handled or to distinguish confirmatory analyses from exploratory ones.

Missing data are especially relevant to the primary endpoint. All COPD participants were asked to provide a six-month pulmonary function test, but only 5 participants completed and returned it. The number of COPD participants asked, reasons for non-return, and the method used to handle missing measurements are not reported in the registry record. No analysis population is defined, and the record does not clarify whether participants were analyzed only when both baseline and follow-up measurements were available.

The record also does not report an interim-analysis plan or whether any interim analyses occurred. It does not state a prespecified statistical analysis plan, a sample-size justification, or the rules used to determine which participants and observations entered each analysis. These details are needed to evaluate whether reported results follow a planned approach; no outcome analysis is posted to assess against such a plan.

11. 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 0.2% reporting threshold.

Most frequent non-serious adverse event terms

The registry reports serious adverse events for the cell therapy treated group: 207 participants were listed at risk, and 0 were listed as affected. For other adverse events, 207 were listed at risk and 10 were listed as affected. The record also reports 4 deaths. These are descriptive counts, not comparisons with an untreated or control group.

No serious adverse-event terms are listed. The record does not provide event descriptions, timing, duration, severity grading beyond the serious versus other categories, or an assessment of whether events were considered related to the procedure. It also does not explain the relationship between the death count and the adverse-event categories. These details are not reported in the registry record.

A count of 0 participants with reported serious adverse events does not establish that serious events could not occur, nor does it establish absence of risk. Similarly, the other adverse-event count and deaths cannot, by themselves, indicate whether the intervention caused an event. Interpretation depends on complete ascertainment, follow-up duration, event definitions, and comparison data; those details are not reported in the registry record. The counts should be read as the safety information recorded for the treated group, without inferring causality or comparative safety.

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

The registry record establishes that the study enrolled 207 participants and describes cell therapy for chronic lung disease, including COPD and interstitial lung disease. It identifies a primary endpoint of change in FEV1 among participants with COPD, with measurement planned at baseline and six months after treatment. It also reports that 5 participants completed and returned a six-month pulmonary function test. These registry entries describe the study and the availability of follow-up data, but they do not provide the FEV1 result itself.

Without a posted numerical estimate, there is no basis in the record to say whether average FEV1 increased or decreased, or to quantify any change. Without an uncertainty interval, the precision of a possible estimate cannot be assessed. The posted results also do not give a control-group comparison. A before-and-after observation, even when available, would describe change among those measured rather than isolate an effect attributable to treatment.

The safety information consists of 0 participants with serious adverse events among 207 at risk, 10 with other adverse events among 207 at risk, and 4 deaths. The record does not supply event terms or establish relatedness. Those counts do not establish that the procedure caused or prevented events, and they cannot be compared with another treatment from the information recorded.

The registry's stated hypotheses include several outcomes beyond FEV1, such as perceived quality of life and use of oxygen or rescue inhalers. Stated hypotheses are not results. Although seven outcome measures are listed, no analysis entries are recorded. The posted information therefore does not quantify those hypothesized outcomes or show how they were analyzed.

In statistical terms, the record offers a study description and limited outcome reporting, not a complete estimate of benefit or harm. Its completed status does not fill in the missing outcome estimates. The conclusions that can be drawn are bounded by what is actually posted: a single treated group, limited returned lung-function testing, adverse-event counts, and no reported primary-endpoint analysis.

13. Why This Trial Matters Statistically

This study illustrates why a clearly named endpoint and a completed study status are not enough to assess a result. The registry identifies a clinically interpretable primary measure—change in FEV1 among participants with COPD—but does not post the numerical endpoint estimate. Without that result, readers cannot evaluate the direction, magnitude, or precision of the primary outcome.

The difference between an intended measurement and an observed analysis is important. The record says all COPD participants were asked to return a six-month pulmonary function test, but only 5 completed and returned it. The size and composition of the group asked to follow up are not reported. That makes it difficult to understand how representative the available measurements might be, even before considering the absence of an outcome summary.

The design also matters. All enrolled participants were assigned to cell therapy according to the arm description, and no like-for-like control comparison is described. This means the record does not offer a concurrent comparison to distinguish change over time from change associated with treatment. A descriptive result and a comparative treatment effect answer different statistical questions.

Finally, the safety counts show why event totals need context. The record lists serious and other adverse events and deaths, but does not provide event terms or relatedness assessments. Counts alone cannot establish cause or comparative safety. The combination of limited outcome reporting, missing follow-up detail, and no comparator demonstrates how registry information can define the question and identify reporting gaps without resolving the treatment-effect question.

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 primary endpoint is reported as a planned measure, but no numerical FEV1 result is present in the registry record. The record gives no baseline or follow-up values, mean change, full range, confidence interval, or test result. The primary endpoint therefore cannot be quantitatively interpreted.

Only 5 participants completed and returned the six-month pulmonary function test. The number of COPD participants asked to complete testing, reasons for non-return, and characteristics of those with and without follow-up are not reported in the registry record. The number of participants with usable paired baseline and follow-up measurements is also not stated.

The record describes all participants as receiving cell therapy and does not identify a like-for-like control group. Allocation is not stated. As a result, a treatment-versus-control effect cannot be estimated from the posted design and results. The record also does not report procedural details beyond the summary of collection and return of participants' own cells and platelet-rich plasma.

Safety reporting is limited to counts. Serious adverse-event terms are not listed, and event timing, relatedness, and the relationship between deaths and the event categories are not explained. Follow-up completeness for safety outcomes is not described.

Seven outcome measures are listed, but no analysis entries are recorded. The statistical analysis plan, multiplicity approach, analysis populations, missing-data methods, interim analyses, and sample-size justification are not reported in the registry record. These limitations prevent a full assessment of the analysis strategy and leave the principal efficacy question unanswered by the posted results.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Autologous Stem Cell Treatment for Chronic Lung Disease Study — NCT03044431.
• Registry record last updated 2018-10-15; status last verified 2018-10.
• Results first posted 2017-10-25.
• 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.

18. Record Summary

The registry record describes a completed study sponsored by Lung Institute, with 207 participants enrolled and cell therapy as the intervention. COPD and interstitial lung disease are listed as conditions. The recorded study start was 2016-04-01, and primary completion was 2017-06-01.

The primary endpoint is mean change in FEV1 from baseline to six months among participants with COPD, with full range named as the dispersion measure. The record says all COPD participants were asked to return a six-month pulmonary function test; 5 completed and returned it. No numerical primary-endpoint result or analysis is reported.

For safety, the record lists 0 serious adverse events among 207 at risk, 10 other adverse events among 207 at risk, and 4 deaths. Serious-event terms are not listed. Seven outcome measures are recorded, with no analysis entries. A like-for-like control comparison, statistical analysis plan, missing-data approach, and sample-size justification are not reported in the registry record.