Asthma and Obesity: Pilot Study: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Asthma and Obesity: Pilot Study (NCT04113746), a randomized na study. The primary outcome is Beliefs About Illness Perception Questionnaire (BIPQ).

2. Clinical Question

3. Trial Design

The registry record describes Asthma and Obesity: Pilot Study as a completed, randomized, single-masked, parallel-group study. It lists an enrollment of 27 and identifies the sponsor as Icahn School of Medicine at Mount Sinai. The record gives a start date of 2019-10-03 and a primary completion date of 2021-08-30. The phase is listed as NA.

The two arms were Asthma and Exercise Lifestyle Change and Asthma Education. Participants in the lifestyle-change arm received asthma and lifestyle-change education related to exercise; the asthma-education arm did not receive lifestyle-change education. The record’s brief summary describes an aim to develop and pilot test four theory-based educational modules integrating asthma and obesity counseling to promote greater physical activity among people with asthma. The stated aim describes the program’s purpose; it does not establish that the program changed activity or any other outcome.

Single masking is the registry’s design classification. Which people were masked, and how masking was implemented, are not reported in the registry record. The method used to generate or conceal random assignments is also not reported. The record does not provide further details about recruitment, the setting, eligibility criteria, treatment delivery, or adherence to the assigned education.

The primary outcome is a continuous BIPQ score assessed at the 30-day post-pilot follow-up visit, with the stated timeframe extending up to 2 months. The posted arm-level outcome samples are 9 for Asthma and Exercise Lifestyle Change and 10 for Asthma Education. These are endpoint counts, not a description of all enrolled participants. The registry record does not explain the difference between those counts and the enrollment figure.

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

Reasons for not completing this period:

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 Beliefs About Illness Perception Questionnaire (BIPQ), measured over At 30 days Post-pilot follow-up visit, up to 2 months.

The primary endpoint is the Beliefs About Illness Perception Questionnaire (BIPQ), recorded as a mean score on a scale. The questionnaire is described as covering five cognitive domains: Identity, Cause, Timeline, Consequences, and Cure-Control. The item in each domain is scored from 0 (none) to 10 (extreme), and the full scale ranges from 0-80. Higher scores reflect a more threatening view of the illness.

This measure is used to assess how participants cognitively and emotionally represent their illness. In this study, that makes it a patient-reported endpoint relevant to the educational program’s focus on asthma, obesity, and lifestyle. It is not a direct measure of physical activity, asthma control, medication adherence, or quality of life. Those topics appear among the secondary outcomes, which include step counts per day, time in physical activity, the Asthma Control Questionnaire, the Asthma Quality of Life Questionnaire, the Medication Adherence Rating Scale, and medication beliefs.

The registry lists the BIPQ assessment at the 30-day post-pilot follow-up visit, up to 2 months. The endpoint is continuous, and its posted summaries are means with standard deviations. The outcome description does not report a minimally important difference or identify a threshold that would define a meaningful individual change. Such a threshold is not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary outcome is continuous: it is a score on a scale, summarized in each arm by a mean and standard deviation. A mean describes the average score among participants contributing to that arm’s outcome summary. A standard deviation describes the spread of individual scores around that arm’s mean. It is not the standard error of the mean and does not directly show the precision of a between-arm comparison.

As a general statistical concept, a confidence interval for a treatment contrast describes a range of values compatible with the data and the method used to calculate the interval. Its width depends on factors including the amount of information in the data and the variability in the outcome. A confidence interval for an arm difference is not reported in the registry record. Nor is a comparative p-value reported. The record specifically notes that values are missing or not estimable for a two-arm comparison of this continuous endpoint, so the posted arm-level means should not be mistaken for an inferential comparison.

Randomization is intended, in general, to support fair comparisons by assigning participants to study arms through a chance process. It does not ensure that every characteristic is balanced in a small study, and it does not remove the need to report how assignment was generated, how outcomes were analyzed, or how missing values were handled. The registry record calls the study randomized, but does not report the sequence-generation or allocation-concealment methods. The record classifies masking as single, without identifying who was masked.

The posted sample-size assumptions describe a planning illustration based on the observed effect, rather than the trial’s own design. The illustration uses a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation. It considers the effect to detect as a stated fraction of the observed effect. For non-time-to-event outcomes, the planning quantities are participants per arm for 80% power or other stated power levels. These values are planning illustrations, not observed power, and do not establish what power the completed pilot study had. The registry record does not report a sample-size justification for the trial itself.

General power calculations depend on assumptions about the effect to be detected, outcome variability, allocation, and the chosen significance level. A planning calculation based on an observed effect is especially sensitive to uncertainty in that observed effect. In a pilot study, a planning illustration may help frame the scale of a later study, but it should not be read as evidence that the pilot was designed to detect a specified effect. The registry record does not report a protocol-based target effect or a formal sample-size calculation for this trial.

For a continuous outcome, a simple unadjusted contrast would compare the arm means. However, no such recalculated contrast is posted as an estimable comparison in the record, and no new numerical contrast can be inferred from the descriptive summaries without doing a calculation. The record’s note that a two-arm comparison is not estimable is therefore central: the descriptive arm summaries do not supply the inferential result needed to quantify a difference with uncertainty. No discrepancy between the sponsor’s analysis and a recalculated analysis is identified.

Other outcomes listed are medication adherence, step counts per day, time in physical activity, asthma control, asthma-related quality of life, and beliefs about medication necessity and concerns. These measures cover different constructs and may have different scales and distributions. As a general statistical point, analysis methods should match the outcome type and data structure. The registry record does not describe the statistical methods used for these outcomes or post analyses for them in the registry record for this assessment.

Multiplicity arises when multiple outcomes or comparisons are examined: the more questions tested, the greater the chance of at least one apparently unusual result under a collection of null hypotheses. General approaches include defining a primary question in advance and using an adjustment or hierarchy when confirmatory claims depend on multiple tests. Here, the record identifies a primary outcome and lists secondary outcomes, but it does not report a multiplicity procedure or a statistical analysis plan. With no posted analyses, there is no reported set of inferential tests to interpret or adjust.

For safety outcomes, counts are presented with an at-risk denominator for each arm. General interpretation requires attention to both the event count and the population in which events were assessed. A zero count in the recorded group does not establish that the risk is zero outside the observed participants, nor does it establish safety in settings or populations not represented by the study. The registry record does not provide the safety observation period or further details of event collection.

10. Confidence Intervals for Each Arm

No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.

R code and output

arm <- c("Asthma and Exercise Lifestyle Change", "Asthma Education")
n   <- c(9, 10)
m   <- c(42.6, 36.2)
sp  <- c(11.4, 8.2)
sd  <- sp      # posted standard deviations
for (i in seq_along(n)) {
  se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
  cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}

CARM  Asthma and Exercise Lifestyle Change  9  42.600  11.400  33.837  51.363
CARM  Asthma Education  10  36.200  8.200  30.334  42.066

11. Multiplicity, Missing Data and Analysis Populations

The registry identifies the BIPQ as the primary outcome and lists six secondary outcomes: medication adherence on the Medication Adherence Rating Scale, step counts per day, time in physical activity, asthma control on the Asthma Control Questionnaire, asthma-related quality of life on the Asthma Quality of Life Questionnaire, and beliefs about medication necessity and concerns on the Beliefs About Medications Questionnaire. The record lists 7 outcome measures in total. The existence of several outcomes raises a general multiplicity issue when investigators make claims based on multiple tests, because examining many results can increase the chance of a seemingly notable finding by chance.

The registry record reports no analyses and does not report a statistical analysis plan, multiplicity adjustment, testing hierarchy, or which outcomes were included in any inferential procedure. It is therefore not possible to determine from the record whether multiplicity was addressed or how any secondary findings were intended to be interpreted. No secondary outcome results are included in the registry record for this analysis.

Missing-data handling is also not reported. The posted primary endpoint counts are 9 and 10, while enrollment is 27. The record does not say why the endpoint counts differ from enrollment, whether missing measurements occurred, or what assumptions or methods were used for incomplete data. The safety denominators differ from the endpoint counts, and the reason is not reported.

The analysis populations are not described. In particular, the registry record does not state whether the primary outcome was analyzed by randomized assignment, among participants who completed follow-up, or according to another population definition. Without these details, the descriptive outcome counts cannot be interpreted as a documented intention-to-treat or per-protocol analysis.

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 posted safety summaries report no serious adverse events and no other adverse events in either arm. For Asthma and Exercise Lifestyle Change, the serious-event denominator is 13 and the other-event denominator is 13; the reported affected counts are 0 for both. For Asthma Education, each denominator is 14 and each affected count is 0. The record also reports 0 deaths in each arm. No top serious adverse-event terms are listed.

These are counts within the recorded safety groups, not evidence that the risk of an event is zero. General interpretation of a zero count depends on how many participants were assessed, how long they were followed, what events were collected, and how they were defined. The safety observation period, event definitions, and ascertainment procedures are not reported in the registry record. The safety denominators also differ from the posted primary-outcome counts, and the reason for that difference is not reported.

The counts alone do not support a comparative safety estimate or a conclusion about rare events. They describe what the registry reports for these groups and should be read within the limits of the recorded denominators and the unreported details of safety monitoring.

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

The record gives a limited descriptive view of BIPQ scores after two different educational approaches. The mean score was 42.6 in the Asthma and Exercise Lifestyle Change arm and 36.2 in the Asthma Education arm. Because higher BIPQ scores reflect a more threatening view of illness, the first arm had a higher posted mean. The record does not provide an estimable two-arm comparison, so those summaries do not show whether the observed pattern represents a reliable difference between the approaches.

The reported outcome is a measure of illness perception, not a direct measure of physical activity or asthma control. The study’s educational goal involved promoting physical activity, and the listed secondary outcomes include activity, medication adherence, asthma control, quality of life, and medication beliefs. However, the registry entries available for this analysis do not include posted findings for those secondary outcomes. Their inclusion as outcomes should not be confused with evidence of change on them.

The randomized design is relevant to how a comparative effect could be assessed, but the record does not provide an analysis of the primary contrast, details of randomization, the masked party, or the handling of missing data. The endpoint counts are smaller than enrollment, and the reason is not reported. Without a documented comparative estimate and its uncertainty, the size and precision of any effect remain unknown from the posted results.

The study is described as a pilot whose purpose was to develop and pilot test educational modules. Its record can inform readers about the intervention concept, the outcome selected, and the descriptive scores posted for participants with endpoint data. It does not establish that the program changed participants’ illness perceptions, activity, asthma outcomes, or medication behavior. Nor does the absence of reported adverse events establish that no risks exist. The appropriate interpretation is confined to the information and summaries in the registry record.

14. Why This Trial Matters Statistically

This trial is statistically notable for the distinction between a randomized design and an estimable reported comparison. Randomization can support a causal comparison when allocation and analysis are appropriately documented, but the record does not post an estimable two-arm analysis of the continuous primary outcome. The arm means are informative descriptions, yet they do not alone answer whether the groups differ beyond the observed data.

The pilot context also matters. The record describes a program-development and pilot-testing purpose, and the outcome counts for the primary score are 9 and 10. A pilot can contribute practical and descriptive information, but its results should not automatically be treated as a precise estimate for a larger confirmatory study. No sample-size justification for the trial itself is reported. The separate planning illustration is based on an observed effect and is not observed power.

Several related outcomes were identified, ranging from activity to asthma control, quality of life, medication adherence, and illness or medication beliefs. Having a defined primary outcome helps distinguish the central question from secondary ones, but the registry record does not report analyses, a multiplicity approach, or findings for the secondary outcomes. Readers therefore cannot assess a family of outcome results or the consistency of effects across measures.

The record also illustrates why endpoint and safety denominators should be read separately. The posted primary-outcome counts differ from the safety denominators and from the enrollment figure, while the reasons are not reported. Transparent reporting of analysis populations and missing-data methods would help readers understand who contributed to each result. These gaps limit the statistical conclusions that can be drawn without diminishing the value of clear descriptive reporting for a pilot study.

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 posted primary outcome has arm-level means and standard deviations, but the registry record says values are missing or not estimable for a two-arm comparison. No comparative estimate, confidence interval, or p-value is reported. The descriptive summaries therefore cannot quantify the magnitude or precision of a difference between arms.

Enrollment is listed as 27, while the primary endpoint summaries include 9 participants in Asthma and Exercise Lifestyle Change and 10 in Asthma Education. The registry record does not explain the difference or describe missing-data handling. The safety denominators are 13 and 14, and the record does not explain why these differ from the endpoint counts. Analysis populations are not reported.

Important design and analysis details are also absent from the record details. Although the trial is classified as randomized and single-masked, the method of random assignment, allocation concealment, and identity of the masked party are not reported. The statistical analysis plan, sample-size justification, multiplicity method, and outcome-analysis details are not reported. The registry lists six secondary outcomes but does not post analyses for them in the registry record.

The record gives no further detail about participant eligibility, characteristics, delivery or receipt of the educational materials, follow-up completeness, or the safety observation period. It also does not report a threshold for a meaningful BIPQ change. These omissions limit judgments about generalizability, the interpretation of the score summaries, and the completeness of safety reporting. They should not be filled in by assumption.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Asthma and Obesity: Pilot Study — NCT04113746.
• Registry record last updated 2022-11-14; status last verified 2022-10.
• Results first posted 2022-11-14.
• 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

Asthma and Obesity: Pilot Study is a completed randomized, single-masked, parallel-group study sponsored by Icahn School of Medicine at Mount Sinai. It enrolled 27 and compared asthma and exercise lifestyle-change education with asthma education without lifestyle-change content. Its primary outcome was the BIPQ score at the 30-day post-pilot follow-up visit, up to 2 months.

The posted BIPQ mean was 42.6 (standard deviation 11.4; 9 participants) for Asthma and Exercise Lifestyle Change and 36.2 (standard deviation 8.2; 10 participants) for Asthma Education. The record states that a two-arm comparison is not estimable. It reports no analyses, and the reasons for the differences between enrollment, endpoint counts, and safety denominators are not reported in the registry record.