Self-management Intervention for Children With Chronic Medical Complexity: Pilot Feasibility Trial: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Self-management Intervention for Children With Chronic Medical Complexity: Pilot Feasibility Trial (NCT04470193), a randomized na study. The primary outcome is Patient Quality of Life (QOL).

2. Clinical Question

The trial asked whether outcomes differed between children whose parents or patients used the MyChildCMC app and those receiving usual care. The registry describes a pilot intended to explore online home monitoring and its preliminary impact. The primary outcome was child quality of life (QOL), assessed at baseline and at follow-up. Other outcomes included emergency department visits or hospitalizations, hospital days, and parent or caregiver satisfaction with care.

The app was intended to support daily monitoring of common symptoms and recognition of early warning signs. The trial's description links this approach to the possibility of avoiding acute events, but the registry record does not report numerical findings for emergency visits, hospitalizations, hospital days, or caregiver satisfaction. It also does not report a numerical measure of app feasibility or scalability. The trial therefore frames relevant questions, but the recorded information does not establish whether app use was feasible in practice or whether outcomes differed between groups.

3. Trial Design

This was a randomized, parallel-group pilot feasibility trial. The registry lists an enrollment of 50 and describes two groups: MyChildCMC intervention and usual care. The study was unmasked, so participants and care providers were not blinded to group assignment. The study status is completed. The recorded start date is 2019-06-10, and the primary completion date is 2020-03-19.

In the intervention group, parents or patients were to use the app to monitor daily symptoms during the study period of 3 months. The usual care group was instructed to continue regular care and symptom monitoring without the app. The registry describes the app as an online phone application intended to facilitate ongoing monitoring of symptoms such as respiratory distress, inadequate feeding or fluid intake, fever, altered mental status, pain, and seizure status. The intervention description also mentions a daily form with 12 questions, including areas such as vitals, pain, seizures, and mood.

The primary outcome was patient QOL. The registry describes assessment at baseline and comparison with follow-up at 1 month and 3 months from the start of the study or intervention. Secondary outcomes listed were patient emergency department visits or hospitalization, number of hospital days, and parent or caregiver satisfaction with care. The registry record does not provide numerical results for these outcomes.

Although the study is identified as a pilot feasibility trial, the specific criteria used to judge feasibility, such as app-use thresholds or recruitment targets, are not reported in the registry record. The randomization procedure, allocation concealment, and any stratification factors are also not reported in the registry record. The record does not state whether the analysis followed the randomized groups regardless of adherence, or identify another analysis population. These details matter when interpreting a randomized study because they clarify how assignment was generated and how participants were included in outcome comparisons.

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: Of the 68 subjects whose medical records were reviewed, 6 did NOT meet eligibility criteria (5 non-English speaking parents and 1 was on palliative care) and were NOT invited to participate. Of the 62 POTENTIALLY eligible subjects who were invited to participate in the study, 12 DECLINED to participate and WERE NOT enrolled, and 50 subjects AGREED to participate and were enrolled. Of the 50 subjects who were enrolled, 24 were randomized to MyChildCMC and 26 to usual care.

Overall Study

Analysis population for the primary outcome, as defined in the registry: Analysis included 50 participants at baseline (including 24 participants in the intervention group and 26 in the usual care group ), 40 participants at 1 months (including 16 in the intervention group and 24 in the usual care group), and 37 participants at 3 months (including 15 participants in the intervention and 22 participants in the usual care group).

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: Baseline population includes 50 participants (who AGREED to participate in the study and were enrolled ): 24 in the MyChildCMC group and 26 in the usual care group.

6. Primary Endpoint

The registry lists the primary outcome as Patient Quality of Life (QOL), measured over Quality of Life assessed at baseline, then compared to 1 month and 3 months from start of study/intervention.

The primary endpoint was patient QOL, measured with the Ellzey et al. questionnaire for children with complex medical conditions. The instrument assesses several domains, including physical and mental health, sleep, pain, activities, and general QOL. The registry states that the total score was standardized to a scale ranging from 20 to 100, with higher scores representing better QOL.

The endpoint was summarized as a mean, with standard deviation as the measure of dispersion. The primary comparison was defined as the difference between study groups in mean QOL change from baseline to each follow-up assessment. Change from baseline focuses attention on how scores shift over time, while a comparison between groups asks whether those shifts differ between the app and usual care groups. The registry describes assessments at baseline, 1 month, and 3 months.

QOL is relevant to this trial because the intervention was designed to help families monitor the health of children with complex medical conditions, and the study description identifies child QOL as a preliminary outcome of interest. The score's range and direction help interpret a reported value: higher scores indicate better QOL according to the registry. However, the registry record does not report the group means, standard deviations, changes from baseline, or between-group estimates. It also does not report confidence intervals or p-values for this endpoint. As a result, the direction, magnitude, and precision of any QOL difference cannot be assessed from the recorded results.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry identifies a continuous primary outcome summarized using a mean and standard deviation. It describes the comparison as mean QOL change from baseline to each follow-up between the intervention and usual care groups. The specific statistical test or model used for this comparison is not reported in the registry record. The concepts below are general explanations of the statistics that would be relevant to this kind of endpoint, not a description of an unreported analysis.

What does a mean change represent?

A mean is an average across participants with outcome measurements. For a change-from-baseline outcome, each participant's follow-up score is considered in relation to that participant's baseline score, and the group mean summarizes those changes. A between-group comparison of mean changes then contrasts the average change in one group with the average change in the other. This is different from comparing follow-up scores alone, although the approaches can be related under particular analysis plans.

The QOL scale has a stated range from 20 to 100, and higher scores represent better QOL. That direction helps explain the meaning of a reported score, but it cannot determine the direction of an unreported group contrast. The registry record does not provide the numerical scores or mean changes needed to describe the observed result.

What does the standard deviation describe?

A standard deviation describes how dispersed individual observations are around the group mean. A smaller standard deviation indicates that values are more concentrated around the mean; a larger one indicates more spread. It describes variability among observations, not the precision of the mean by itself. Precision also depends on how many participants contribute data and on the analysis used. The registry record identifies standard deviation as the dispersion measure but does not report its values for the QOL outcome.

How would a confidence interval help?

In general, a confidence interval gives a range of values compatible with an estimate under the assumptions of the statistical method. For a between-group mean-change estimate, it would convey both the estimated contrast and its uncertainty. A relatively narrow interval indicates greater precision than a wide interval, while the interval's position in relation to no difference can help readers assess which contrasts remain compatible with the data. A confidence interval is not a guarantee that the true value lies within the particular interval. No confidence interval for the QOL comparison is reported in the registry record, so its precision cannot be judged.

What does a p-value answer?

In general, a p-value describes how compatible the observed data are with a specified null hypothesis, under the assumptions of the analysis. It is not the probability that the null hypothesis is true, and it does not measure the size or practical importance of an effect. A p-value alone also does not communicate precision as directly as an estimate with a confidence interval. The registry record does not report a p-value for the primary comparison.

Why do repeated assessments need an analysis plan?

QOL was assessed at baseline and at follow-up at 1 month and 3 months. Measurements from the same participant over time are related rather than independent. General statistical methods for repeated observations account for this relationship, while separate analyses at each time point address contrasts individually. Different approaches can produce different estimates and uncertainty, especially when some participants lack measurements at later assessments. The statistical method used for the repeated assessments is not reported in the registry record, so no particular model or test should be assumed.

There are multiple follow-up assessments and several listed secondary outcomes. In general, examining multiple outcomes or time points can increase the chance of finding a nominally unusual result by chance if many tests are conducted. Multiplicity procedures can address this issue, but no adjustment strategy or testing hierarchy is reported in the registry record. This absence does not establish whether an adjustment was or was not applied in an analysis outside the record; it means the record does not document one.

How should sample-size illustrations be understood?

General planning calculations can estimate the number of participants per arm needed to detect an assumed effect under stated conditions. The registry's sample-size assumptions describe an illustration based on an observed effect, a two-sided significance level of 0.05, equal allocation, and a normal approximation; the effect to detect is described as a stated fraction of the observed effect. These are planning assumptions, not evidence that the trial was designed around those calculations. The assumptions also specify that non-time-to-event planning quantities are participants per arm. The resulting numerical planning values are not reported in the registry record addressed by these registry entries.

Such calculations are not observed power. They do not show the probability that the completed trial detected an effect, and they do not substitute for a confidence interval or the observed estimate. Because the numerical QOL results and the resulting planning values are not reported in the registry record, readers cannot use them to evaluate the magnitude or precision of the comparison.

Finally, the distinction between a randomized design and an analysis method matters. Randomization assigns participants to groups, while the statistical analysis summarizes and compares outcomes after assignment. The registry identifies random allocation and a parallel model, but it does not identify the inferential method used for QOL or the other outcomes. General descriptions of means, standard deviations, confidence intervals, and repeated-measures methods therefore provide context only; they should not be read as claims that a particular procedure was used in this trial.

10. Multiplicity, Missing Data and Analysis Populations

The primary QOL outcome was assessed at baseline and at two follow-up times, and the registry also lists emergency department visits or hospitalization, hospital days, and parent or caregiver satisfaction with care as secondary outcomes. Considering several outcomes and follow-up comparisons raises a general multiplicity issue: the more statistical tests performed, the greater the chance that at least one result may appear unusual under a null model. Researchers may address this through a prespecified hierarchy, adjustment, or other strategy. The registry record does not report a multiplicity strategy, a testing hierarchy, or whether any adjustment was used.

The primary outcome analysis counts vary by assessment. It included 50 participants at baseline, 40 at 1 month, and 37 at 3 months. The group counts reported are 24 intervention and 26 usual care at baseline, and 16 intervention and 24 usual care at 1 month. At 3 months, 15 participants were in the intervention group; the usual care count is not reported in the registry record. These are analysis counts, not an explanation of missingness. Reasons that participants did not contribute to a particular assessment, whether missingness differed by group, and whether the observed data were considered complete are not reported in the registry record.

The record does not specify an analysis population, such as whether participants were analyzed according to randomized assignment, nor does it describe imputation, complete-case analysis, or sensitivity analyses for missing data. These omissions limit assessment of how attrition and incomplete outcome data may have affected the estimates. The registry record also does not report the numerical endpoint estimates required to evaluate that issue empirically.

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% reporting threshold.

The registry reports zero serious adverse events among 24 participants at risk in the MyChildCMC intervention group and zero among 26 participants at risk in the usual care group. These counts describe what was recorded for the specified serious adverse event category; they do not establish that no health events of any kind occurred. The record lists no serious adverse event terms.

Deaths are reported separately: 2 in the intervention group and 0 in the usual care group. The registry information does not report the cause, timing, circumstances, or relationship to the intervention for these deaths. It also does not explain how the death counts relate to the serious adverse event counts. They should therefore be described as reported counts, without attributing cause or drawing conclusions about treatment safety.

Safety counts are most informative when considered alongside the period of observation, event definitions, completeness of reporting, and exposure to the intervention. Those details are not fully reported in the registry record. The counts should not be converted into rates or comparisons without the necessary person-time and analysis details. In particular, zero recorded serious adverse events is not proof that the intervention has no risk, and the reported death counts alone do not establish a causal relationship.

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

The registry record establishes that this was a randomized, parallel-group pilot comparing use of MyChildCMC with usual care in children with medical complexity. It identifies child QOL as the primary outcome and describes follow-up assessments at 1 month and 3 months. It also lists emergency department visits or hospitalization, hospital days, and parent or caregiver satisfaction with care as secondary outcomes. These details clarify what the investigators intended to study.

The record does not report numerical QOL scores, mean changes, between-group estimates, confidence intervals, or p-values. It also does not report numerical results for the secondary outcomes. Therefore, the posted information does not show whether the groups differed, how large any difference was, or how precisely it was estimated. The absence of reported estimates is not evidence that the groups had the same outcomes, and it cannot support a conclusion that the app did or did not change QOL.

The study description presents the app as a tool for daily symptom monitoring and early recognition of warning signs. It frames feasibility, scalability, and preliminary impact as areas of interest. However, specific feasibility measures and their results are not reported in the registry record. The description of an intended purpose is not itself evidence that the purpose was achieved.

The trial's pilot status and enrollment of 50 provide context for its scale, but the registry does not report a sample-size justification or the analysis needed to judge the precision of the primary comparison. The outcome counts at successive assessments also show that the number included in the QOL analysis varied over time. Reasons for this variation and methods for handling missing data are not reported in the registry record.

Accordingly, the record supports a description of the trial design, intervention, comparator, outcomes, and some analysis counts. It does not support a quantitative estimate of the comparative effect, a claim of equivalence, or a causal interpretation of the separately listed deaths. Those judgments require reported outcome estimates and fuller analysis and safety details.

13. Why This Trial Matters Statistically

This trial addresses a statistical question that is important in studies of complex care: how to compare a monitoring intervention with usual care when outcomes are collected repeatedly and may be available for fewer participants at later assessments. The primary endpoint, child QOL, is a continuous measure, and the registry defines a comparison of mean change from baseline. That makes the distinction between the size of an estimated group contrast and its precision central to interpretation.

The randomized parallel design provides a framework for comparing the app group with usual care. Random assignment can reduce systematic differences between groups on average, but it does not guarantee that groups are identical in a particular trial. The registry record does not describe the randomization procedure, allocation concealment, or the statistical model, so important features of the comparison cannot be evaluated from this information alone.

The differing QOL analysis counts across assessments make missing-data handling relevant. A complete interpretation would need to connect the estimates to the participants contributing data and explain how incomplete follow-up was handled. The record lists several outcomes and follow-up times as well, making it useful to know whether the analysis followed a prespecified hierarchy or addressed multiplicity. Neither the missing-data approach nor the multiplicity strategy is reported in the registry record.

Finally, a pilot can be informative about the conduct of a later study as well as about preliminary outcomes. Here, the stated interest in feasibility and scalability is not accompanied in the registry record by a reported feasibility definition or numerical result. The record thus identifies the issues a subsequent evaluation would need to quantify, while the absence of outcome estimates means the comparative clinical findings cannot be assessed from these registry entries.

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 main limitation for interpreting this trial record is that numerical results for the primary QOL outcome are not reported in the registry record. Group means, standard deviations, mean changes, between-group estimates, confidence intervals, and p-values are not available in the registry record for this analysis. Without these, the direction, size, and precision of the primary comparison cannot be evaluated. Numerical results for the listed secondary outcomes are also not reported in the registry record.

The registry provides QOL analysis counts at baseline and follow-up, but it does not report why the counts differ across assessments or how missing observations were handled. The number in the usual care group at 3 months is not reported in the registry record. This prevents a complete description of group-specific follow-up counts and limits assessment of the potential effects of incomplete outcome data.

Further methodological information is not reported in the registry record, including the statistical test or model, the randomization procedure, allocation concealment, the analysis population, multiplicity handling, and sensitivity analyses. The record also does not provide a sample-size justification. Although it includes assumptions for planning illustrations, the numerical results of those illustrations are not available in the registry record, and such illustrations would not be observed trial outcomes.

The feasibility and scalability aims are described, but specific feasibility criteria and results are not reported in the registry record. The intervention and usual care descriptions also do not give a complete account of implementation or adherence. Finally, deaths are listed for the intervention group, but cause, timing, and relationship to the intervention are not reported. These limits preclude attributing the deaths to the app or making a complete safety assessment from the recorded counts.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Self-management Intervention for Children With Chronic Medical Complexity: Pilot Feasibility Trial — NCT04470193.
• Registry record last updated 2024-10-04; status last verified 2024-07.
• Results first posted 2024-10-04.
• 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 describes NCT04470193, a completed randomized, parallel-group pilot feasibility trial sponsored by the University of Utah. Enrollment was 50. The study compared the MyChildCMC app, used for daily symptom monitoring during a 3-month study period, with usual care. The primary outcome was patient QOL, assessed at baseline and at 1 month and 3 months; secondary outcomes included emergency department visits or hospitalization, hospital days, and parent or caregiver satisfaction with care.

The record reports QOL analysis counts of 50 at baseline, 40 at 1 month, and 37 at 3 months. It reports zero serious adverse events in both groups and lists 2 deaths in the intervention group and 0 in usual care. Numerical QOL and secondary outcome results, details of missing-data handling, and the statistical analysis method are not reported in the registry record.