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 Exploring Novel Interventions to Improve Adherence in Children With Cystic Fibrosis (NCT02301377), a randomized na study. The primary outcome is Medication Adherence.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02301377 |
| Phase | NA |
| Condition | Cystic Fibrosis |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 5 |
| Arms in the primary outcome | 2 |
| Lead sponsor | University of Michigan |
| Results posted | Yes |
2. Clinical Question
Population
Children with cystic fibrosis were enrolled. The registry record reports an enrollment of 5. It does not report further eligibility details or a more specific description of the analysis population for the primary outcome.
Intervention
The intervention group received a Spiro PD personal spirometer. The device was intended to let participants measure lung function at home and receive medication reminders. Participants were instructed to use it to check lung function once a week. The registry description of the reminder instructions is incomplete, so further details are not reported in the registry record.
Comparator
The control group was asked to complete quality-of-life questionnaires at enrollment and at the end of the study, and to attend quarterly clinic visits. The record describes routine clinic measurements but does not report that this group received the personal spirometer or its reminders.
Primary question
The primary question was whether medication adherence, measured using prescription-refill data over 3 months, differed between the randomized groups. The measure covered inhaled hypertonic saline, dornase alfa and CF multivitamins. The outcome is a percentage based on filled prescriptions relative to the number expected from the prescribed amount. The registry record posts group means and standard deviations, but no inferential comparison or confidence interval.
3. Trial Design
This completed study was a randomized, parallel-group pilot in children with cystic fibrosis. The registry lists allocation as randomized and masking as none. Its phase is listed as NA. The sponsor was the University of Michigan. The recorded start was 2014-11, and primary completion was 2015-03.
The intervention group received a Spiro PD personal spirometer, described as enabling home lung-function measurement and medication reminders. Participants were instructed to use the device once a week to check lung function. The control-group description includes quality-of-life questionnaires at enrollment and study end, as well as quarterly clinic visits with a pediatric pulmonologist. The arm descriptions in the registry record are incomplete, so additional procedures should not be inferred.
The registry reports an enrollment of 5. The posted primary outcome data include 3 participants in the intervention group and 2 in the control group. This is a very small pilot dataset. The study summary describes the work as a pilot intended to assess the feasibility of providing the device to children with cystic fibrosis. A target enrollment, recruitment details, and a sample-size justification are not reported in the registry record.
The primary outcome was medication adherence over 3 months. Two secondary outcomes are listed: child and parent treatment-burden domain scores from the Cystic Fibrosis Questionnaire-Revised. The record lists three outcome measures in total, but secondary numerical results are not included in the posted outcome data. It also lists no posted analyses. The protocol’s analysis populations and any analysis plan are not reported in the registry record.
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 | Intervention Group | Control |
|---|---|---|
| STARTED | 3 | 2 |
| COMPLETED | 3 | 2 |
| NOT COMPLETED | 0 | 0 |
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 | Intervention Group | Control | Total |
|---|---|---|---|---|
| Participants analysed | 3 | 2 | 5 | |
| Age, Continuous | years | 11.51 (SD 0.64) | 11.93 (SD 1.29) | 11.67 (SD 0.82) |
| Sex: Female, Male: Female | Participants | 3 | 1 | 4 |
| Sex: Female, Male: Male | Participants | 0 | 1 | 1 |
| Forced expiratory volume in 1 second (FEV1) | %predicted | 74.33 (SD 16.65) | 93 (SD 1.41) | 81.8 (SD 15.61) |
| Body Mass Index (BMI) Percentile | Percentile | 64.33 (SD 19.14) | 41.5 (SD 16.26) | 55.2 (SD 20.14) |
| Cystic Fibrosis Questionnaire-Revised (CFQ-R) Treatment Burden Domain Score (Child) | units on a scale | 74.33 (SD 6.35) | 78 (SD 31.11) | 75.8 (SD 16.31) |
| Cystic Fibrosis Questionnaire-Revised (CFQ-R) Treatment Burden Domain (Parent) | units on a scale | 59.33 (SD 17.24) | 61 (SD 24.04) | 60 (SD 17.14) |
6. Primary Endpoint
The registry lists the primary outcome as Medication Adherence, measured over 3 months.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | Percent Adherence |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is medication adherence, expressed as percent adherence over 3 months. It combines refill information for inhaled hypertonic saline, dornase alfa and CF multivitamins. For each medication, the number of prescriptions filled during the period was divided by the number expected from the prescribed amount, and that result was multiplied by 100. The registry describes the endpoint as overall adherence across these medications.
This measure uses prescription-refill records rather than direct observation of medication use. As a general statistical and measurement point, refill-based adherence can indicate whether prescriptions were obtained as expected, but it does not by itself establish when or how a medication was taken. The endpoint therefore measures a specific aspect of adherence, not every aspect of medication-taking behavior.
The outcome is reported as a continuous measure, with the group mean and standard deviation. The intervention group mean was 91.6, with a standard deviation of 14.43 among 3 participants. The control group mean was 100.0, with a standard deviation of 0.0 among 2 participants. The registry does not report a confidence interval or a formal statistical comparison for this endpoint. The reasons for selecting this endpoint and its measurement properties are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted primary outcome summaries are descriptive. The intervention group had a mean adherence value of 91.6 and standard deviation of 14.43, based on 3 participants. The control group had a mean of 100.0 and standard deviation of 0.0, based on 2 participants. These summaries describe the observed participants and should not be read as a reliable estimate of the difference expected in a wider population.
The group sizes are particularly important to interpretation. With only 3 participants in one group and 2 in the other, individual observations can strongly influence group summaries. The control group’s standard deviation of 0.0 means the reported values did not vary within that group; it does not show that adherence is invariably the same among children with cystic fibrosis or that the measure is precise in a broader population. The intervention group’s standard deviation describes the spread of its observed values, not uncertainty about the true effect by itself.
No confidence interval, p-value, or formal between-group test is posted for the primary endpoint. Consequently, the registry record does not quantify the uncertainty around a group contrast or establish whether the observed summaries are compatible with a range of possible effects. Statistical significance cannot be inferred from the means and standard deviations alone. No adjusted analysis is posted, and the analysis approach is not reported in the registry record.
The study summary frames the work as a pilot to assess the feasibility of providing a home spirometer. The outcome summaries do not report feasibility measures such as device uptake, adherence to device use, acceptability, or implementation barriers. Those results are not reported in the registry record. Nor do the posted data establish that reminders or home lung-function measurement caused the observed adherence values: random allocation is reported, but the very small groups and absence of a reported inferential analysis leave substantial uncertainty.
The two listed secondary outcomes concern treatment burden as rated by children and parents. Their numerical results are not posted in the registry record. A conclusion about those outcomes cannot be drawn from the primary adherence summaries. The posted data support a narrow description of the recorded refill-based adherence values in these participants; they do not support a broad conclusion about effectiveness, safety, feasibility, or outcomes in other settings.
9. Statistical Methods Explained
What does a group mean describe?
A mean is the arithmetic average of the observed values in a group. In this trial, the reported mean is the average percent-adherence measure among participants with posted data in that group. It describes the center of those observations. It is not a guarantee that each participant had that value, and it does not describe all children with cystic fibrosis.
The intervention-group mean is 91.6, and the control-group mean is 100.0. These are descriptive summaries. A comparison of means can be useful for expressing a group contrast, but an inferential conclusion also requires information about uncertainty and a specified analysis. The registry record does not post a formal comparison or a confidence interval for the primary outcome.
What does the standard deviation describe?
A standard deviation summarizes how dispersed individual observations are around the group mean. The intervention group has a standard deviation of 14.43, while the control group has a standard deviation of 0.0. The latter indicates no observed spread in the posted control values. It is not a measure of how certain the mean is, and with 2 participants it provides very limited information about variability beyond those observations.
As a general statistical explanation, the standard deviation and the standard error answer different questions. The standard deviation describes variation among observations; the standard error describes the estimated sampling variability of a statistic under a model. The registry record does not report a standard error, confidence interval, or model-based estimate for this comparison. No new uncertainty estimate can be recovered from the posted summaries without making additional analytic choices.
Why does the small sample matter?
With 3 participants in the intervention group and 2 in the control group, the estimates are highly sensitive to the observed values. A single participant’s result can materially affect a mean or a dispersion summary. Small groups also make assumptions used by many standard inferential procedures difficult to assess. This does not make the descriptive values invalid; it limits how confidently they can be generalized and how finely uncertainty can be characterized.
Randomization helps define the intended comparison by assigning participants to groups, but it cannot ensure that small groups are balanced on every relevant characteristic. Baseline characteristics and group comparability are not reported in the registry record. The allocation method and any stratification details are also not reported. Therefore, the posted outcome data do not permit an assessment of baseline balance or a more detailed reconstruction of the randomized comparison.
What can a confidence interval add?
As a general explanation, a confidence interval gives a range of estimates compatible with the data under a specified statistical procedure. Its width conveys precision: a wider interval indicates less precision. An interval for a between-group contrast would be more informative than group means alone because it would show uncertainty in the contrast, not just the two observed centers. No such interval is reported in the registry record. A confidence interval should not be interpreted as the probability that a fixed true effect lies within a particular calculated interval.
What do planning calculations mean?
As a general planning concept, sample-size calculations ask how many participants may be needed to detect an effect under chosen assumptions. The planning illustration associated with these results is described as using the observed effect, a two-sided significance level of 0.05, equal allocation, and a normal approximation. It considers a stated fraction of the observed effect. For non-time-to-event outcomes, such planning quantities are expressed as participants per arm for 80% power or participants per arm for 90% power.
These are planning illustrations rather than observed trial results. They are not observed power, and they are not evidence that the trial was designed to achieve a particular power. Calculations based on an effect estimated from very small groups can be unstable; a different underlying effect would lead to different planning requirements. The trial’s own sample-size justification is not reported in the registry record. No planning count should be taken as an enrollment target or as a replacement for the actual posted group sizes.
What is not available for reanalysis?
The record posts group-level means, standard deviations and participant counts for the primary endpoint, but no individual observations. It reports no formal primary-outcome analysis and no secondary-outcome numerical summaries. Without individual data, the analysis plan, and additional details about missing observations, a more complete reanalysis cannot be reconstructed from the registry record. The group summaries should therefore be read as the reported descriptive results, not as a substitute for a sponsor’s inferential analysis.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity arises when a study evaluates several outcomes or makes several statistical comparisons. As a general principle, the more inferences considered, the greater the chance that at least one may appear unusual by chance unless the analysis plan accounts for the multiplicity. This registry record lists medication adherence as the primary outcome and two secondary outcomes: child and parent treatment-burden domain scores from the Cystic Fibrosis Questionnaire-Revised. It does not report a multiplicity adjustment, a hierarchy for testing outcomes, or a prespecified approach to interpreting multiple results. No statistical analysis plan is reported in the registry record.
The record lists no posted analyses. It provides primary-outcome summaries but no formal test or confidence interval, and it does not post numerical results for the two secondary outcomes. As a result, there is no set of reported inferential results from which to assess multiplicity-adjusted conclusions. The reason the secondary results are absent is not reported in the registry record.
Missing-data handling is also not reported. The record does not explain whether all randomized participants contributed to the primary endpoint, whether any measurements were unavailable, or how unavailable values were treated. It does not describe imputation, sensitivity analyses, or a rule for excluding observations. The posted primary outcome counts are 3 for the intervention group and 2 for the control group; the registry does not state whether these counts represent all randomized participants or a defined analysis population.
The analysis population is not reported in the registry record. In particular, the record does not specify whether the analysis followed assigned groups regardless of device use, included only participants with outcome data, or used another population definition. These omissions limit assessment of how the reported summaries relate to the randomization and how missing outcomes might affect interpretation.
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 |
|---|---|---|---|
| Intervention Group | 0 | 3 | 0.0% |
| Control | 0 | 2 | 0.0% |
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 |
|---|---|
| Intervention Group | 0 of 3 |
| Control | 0 of 2 |
The registry lists 3 participants at risk for serious adverse events in the intervention group and 2 in the control group. It reports 0 participants affected by serious adverse events in each group. For other adverse events, 3 intervention participants and 2 control participants were listed as at risk, with 0 affected in either group. These figures describe what was posted for the study; they do not establish that no adverse events occurred outside the registry’s reporting scope.
The death counts are not reported in the registry record. No serious adverse-event terms are listed. The record does not provide event descriptions, event timing, severity grading beyond the serious/non-serious distinction, or details about how adverse events were collected. It also does not give enough information to characterize the duration or completeness of safety monitoring.
Zero affected participants in these small groups should be interpreted as an absence of reported affected participants, not as proof that the device or study procedures carry no risk. The figures cannot estimate uncommon harms with precision, and they should not be generalized beyond the participants and reporting period represented by the registry record.
12. What the Result Does, and Does Not, Mean
The posted result is a descriptive account of refill-based medication adherence in a very small randomized pilot. The intervention-group mean was 91.6, with standard deviation 14.43 among 3 participants. The control-group mean was 100.0, with standard deviation 0.0 among 2 participants. Those values do not, by themselves, establish the size or precision of a treatment effect. No inferential comparison or confidence interval is reported.
The outcome reflects prescription refills for inhaled hypertonic saline, dornase alfa and CF multivitamins during 3 months. It does not directly measure every aspect of medication use, and it does not establish why a prescription was or was not filled. The record does not post supporting measurements that would explain participant behavior or show how the device was used.
The study summary identifies feasibility as the pilot’s purpose, but the posted outcome data do not include quantitative feasibility results. The record also does not provide numerical results for the listed child and parent treatment-burden outcomes. Therefore, the posted data cannot establish whether device provision was feasible or how participants’ treatment-burden scores changed.
Randomization is an important design feature, but the groups are extremely small, and information about baseline characteristics, missing outcomes and analysis populations is not reported. These limits make the results imprecise and constrain generalization. A difference between the observed group summaries should not be treated as proof of benefit or lack of benefit, and the data do not establish that the intervention caused any particular adherence pattern.
The appropriate interpretation is narrow: these are the recorded summary values for the participants included in the primary outcome results. They are useful as pilot data and as a record of what was measured, but they are not a definitive estimate of comparative effectiveness, a complete account of feasibility, or a comprehensive safety assessment.
13. Why This Trial Matters Statistically
This trial is statistically notable because it illustrates the distinction between a randomized design and a precise estimate. Random allocation defines a comparative framework, but it does not eliminate uncertainty when the groups are very small. The posted group counts—3 in the intervention group and 2 in the control group—mean that the descriptive summaries can be strongly influenced by individual observations.
The endpoint also shows how the choice of measurement shapes interpretation. Prescription-refill data provide a practical record-based measure of one component of medication adherence. They are not the same as directly observing medication use. Readers need to keep the operational definition in view when interpreting the means: the outcome is based on filled prescriptions relative to the number expected from prescribed amounts during 3 months.
The record identifies the study as a pilot focused on feasibility, yet its posted outcomes center on adherence and list child and parent treatment-burden scores as secondary outcomes. The absence of posted secondary numerical results and feasibility measures means the registry record does not resolve those questions. This distinction matters: a pilot’s aims, collected outcomes and posted results do not necessarily provide the same kinds of evidence.
Finally, the absence of a reported inferential analysis is itself important. Means and standard deviations can describe the recorded data, but they do not show the uncertainty around a comparative effect. A confidence interval, analysis plan and explanation of missing data would be needed for a fuller statistical interpretation. The record offers a transparent but limited snapshot, and its value lies in understanding the measurement and scale of the pilot rather than treating its estimates as definitive.
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 most immediate limitation is the small number of participants with primary outcome summaries: 3 in the intervention group and 2 in the control group. The registry reports an enrollment of 5, but it does not clarify whether the outcome counts include all randomized participants or whether any participants were excluded from the endpoint analysis. Small groups provide little information about between-participant variation and make results sensitive to each observation.
The registry record does not report baseline characteristics, details of allocation procedures, or the analysis population. It is therefore not possible to assess group comparability from the posted outcome information or determine whether the analysis followed assigned groups. The treatment and control descriptions are incomplete, including an unfinished description of reminder instructions and limited detail about control procedures.
No formal statistical comparison, confidence interval, or analysis method is reported for the primary outcome. Handling of missing data, protocol deviations, and multiplicity is also not reported. The two secondary outcomes are listed but have no posted numerical results. The registry record does not explain whether those results were unavailable, not collected, or omitted for another reason.
The endpoint relies on prescription-refill data. This provides a defined measure of medication acquisition relative to expected refills, but does not directly establish medication-taking behavior. The registry record does not report validation details for the measure or explain how adherence across the three medications was combined beyond the stated overall calculation.
Finally, the record does not post quantitative feasibility outcomes, detailed adverse-event descriptions, or death counts. The posted data are therefore limited for judging implementation and safety. These constraints are specific to the information recorded for this pilot and should temper any attempt to generalize its results.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Exploring Novel Interventions to Improve Adherence in Children With Cystic Fibrosis — NCT02301377.
- Registry record last updated 2015-07-16; status last verified 2015-06.
- Results first posted 2015-07-16.
- 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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18. Record Summary
NCT02301377 is a completed randomized, parallel-group pilot sponsored by the University of Michigan. The registry lists no masking, phase NA, enrollment of 5, a start in 2014-11 and primary completion in 2015-03. The intervention was a Spiro PD personal spirometer intended to support home lung-function measurement and provide medication reminders.
The primary outcome was prescription-refill medication adherence over 3 months for inhaled hypertonic saline, dornase alfa and CF multivitamins. The posted mean was 91.6 with standard deviation 14.43 for 3 intervention participants, and 100.0 with standard deviation 0.0 for 2 control participants. No formal comparison or confidence interval is reported. Two secondary treatment-burden outcomes are listed without numerical results. No statistical analyses are posted, and analysis populations, missing-data methods, sample-size justification and detailed feasibility findings are not reported in the registry record.