UrApp for Childhood Nephrotic Syndrome Management (Incident Cohort): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for UrApp for Childhood Nephrotic Syndrome Management (Incident Cohort) (NCT04075656), a randomized na study. The primary outcome is Number of Participants Reporting Medication Adherence.

2. Clinical Question

This is a question about reported adherence at multiple assessment times, not a single endpoint value. A full comparison would need the adherence counts, the number of respondents at each time, and an analysis that accounts for the way the observations are organized across time. Those results are not reported in the registry record. The registry also lists secondary outcomes concerning caregiver self-efficacy, parent- and patient-reported pediatric quality of life, delayed detection of relapses, and hospitalizations. Their presence gives context to the broader self-management question, but does not substitute for the missing primary endpoint results.

3. Trial Design

The registry describes a randomized, parallel-group trial with no masking. The two arms were UrApp and standard of care. Participants assigned to UrApp were to use the application for one year in addition to standard of care; the comparison arm was to receive standard of care for one year. The registry classifies both interventions as behavioral. The phase is listed as NA.

The trial concerns idiopathic nephrotic syndrome, described in the registry as a chronic kidney disease affecting children and associated with relapses and complications. Its rationale emphasizes the demands of self-management for families and the possibility of nonadherence. UrApp is intended to assist families with this management; the trial's stated purpose is to examine self-management and disease outcomes rather than to report a clinical treatment comparison.

The enrollment recorded in the registry is 58. The brief description states that the study will include 60 caregivers and describes randomization in a 1:1 ratio. These figures are both retained as registry statements; the record does not explain the difference between the planned description and the enrollment entry. The study status is completed. The recorded start date is 2019-09-17, and the primary completion date is 2025-04-25.

These design features describe how the comparison was intended to be organized, but they do not establish how many participants contributed data at each assessment or whether the intended allocation was achieved in the final analyzed population. The primary endpoint description specifies an analysis population of study completers who responded to the survey, with survey data missing for some participants. Final arm-specific analysis counts and the actual allocation counts for the primary endpoint are not reported in the registry record. Details beyond the randomized, parallel, unmasked design—including methods used to conceal allocation or monitor the study—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.

Before assignment: Caregivers of pediatric patients were enrolled into the study, randomized to a study arm, and took part in the study intervention. Data for outcome measures and adverse events were collected about and from pediatric patients but the study participants are the caregivers rather than pediatric patients.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: This analysis includes participants who completed the study and responded to this survey. Survey data are missing for some participants. If a participant missed a study visit they were sent the survey electronically but some participants did not complete every survey.

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 Number of Participants Reporting Medication Adherence, measured over Baseline, Month 6, Month 12.

The primary endpoint is the number of participants reporting medication adherence. Adherence is assessed through a caregiver survey using the validated 4-question Morisky, Green, and Levine Adherence Scale. Each question has Yes or No response options, coded 0 or 1, and the total score ranges from 0 to 4. Higher scores indicate better medication adherence. The registry defines adherence as a score of at least 3.

The endpoint is assessed at baseline, Month 6, and Month 12. A participant count gives the number meeting the stated threshold; it is not, by itself, a complete account of the proportion of eligible respondents meeting the threshold. Interpreting a count requires knowing how many participants provided usable responses at that assessment. The endpoint description notes that the analysis includes study completers who responded to the survey, and that some participants missed visits or did not complete every survey.

A threshold-based endpoint turns the scale into a classification: scores meeting the cutoff count as adherent, and scores below it do not. This can make the outcome straightforward to describe, while losing detail about differences in scores within either category. The scale's validity and the prespecified threshold are part of the endpoint definition, not evidence about the results. The registry does not report the observed adherence counts, the respondent counts at each time, or an estimated comparison between arms. Those results are not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary outcome is a count of participants meeting a binary rule: a survey score at least 3 is classified as adherence. The underlying score has several possible values, but the analysis definition reduces it to whether each respondent meets the cutoff. A count can be useful for communicating how many participants satisfy a criterion. For a comparison between arms, however, counts need context: the number of participants assessed and the amount of missing data determine what population the observed count represents.

What does a confidence interval express?

In general, a confidence interval describes the statistical uncertainty around an estimated quantity under the assumptions of the analysis. A narrow interval indicates greater precision than a wide interval, while an interval that includes values compatible with little or no difference may leave the size and direction of an effect uncertain. A confidence interval does not show the probability that a particular treatment effect is true. No confidence interval for the primary outcome is reported in the registry record, so its precision cannot be evaluated.

What would a test of the arms address?

In general, a statistical test evaluates how compatible the observed data are with a specified null hypothesis under a stated model. A p-value does not measure the size or clinical importance of an effect, and it does not replace an estimate with an uncertainty interval. The registry lists no analyses, and it does not report a primary endpoint test or p-value. The statistical plan, including the planned hypothesis test and any adjustment for repeated assessment, is not reported in the registry record.

Why do repeated assessments matter?

Baseline, Month 6, and Month 12 responses from the same participant are not independent observations in the way responses from unrelated participants may be. General methods for repeated measurements account for this within-participant relationship, or may define a particular follow-up time as the main comparison. Different choices can produce estimates with different interpretations. The registry specifies the assessment times but does not report which method was used, whether the primary comparison was at a particular time, or whether change from baseline was analyzed.

Why is missingness important?

When survey responses are unavailable, the observed data describe respondents rather than necessarily describing everyone assigned to an arm. Missingness can reduce precision, and it can also introduce bias if the likelihood of responding is related to adherence or other relevant characteristics. General statistical approaches can use assumptions or models to address missing observations, but those approaches rely on choices that should be described. The record notes missing surveys and defines the endpoint population as completers who responded. It does not report imputation, weighting, sensitivity analyses, or assumptions about why data are missing.

How should the planning illustrations be read?

In general, a sample-size calculation is a planning exercise based on assumptions about an effect, variability, significance level, allocation, and analysis method. It is not an observed result and does not show the achieved power of a completed trial. The registry describes its planning illustrations as based on fractions of the observed effect, with a two-sided significance level of 0.05, equal allocation, and a normal approximation. It also distinguishes total event counts for time-to-event illustrations from participants per arm for other illustrations. These descriptions do not establish the trial's original design justification or its final analysis. The sample-size justification and the full statistical plan are not reported in the registry record.

More generally, over-dispersion occurs when observed variability is greater than a statistical model assumes. If a model underestimates variability, standard errors may be too small and uncertainty may appear narrower than it should. The registry does not report a model for the primary endpoint or evidence that over-dispersion was assessed; this general issue should not be taken as a description of the analysis used in this trial.

Because the primary outcome is a thresholded count observed at multiple times, several statistical summaries could be appropriate for different questions. A count alone does not specify a risk, rate, or treatment effect, and it does not provide a measure of uncertainty. The registry record does not report the values needed to reconstruct an unadjusted comparison, so no recalculation is available. The sponsor's own analysis cannot be compared with a recalculation because neither a primary analysis nor the required endpoint summaries are reported.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary adherence outcome assessed at baseline, Month 6, and Month 12, as well as secondary outcomes involving caregiver self-efficacy, parent and pediatric quality of life, delayed relapse detection, and hospitalizations. Considering several outcomes or assessment times can create multiplicity: the more comparisons examined, the greater the chance that at least one appears notable by chance if each is interpreted without regard to the others. General approaches include identifying a primary comparison in advance or adjusting inference across a defined family of tests. The registry record does not report a multiplicity strategy, a hierarchy among time points, or which secondary outcomes were formally tested.

The record describes missing survey data and specifies that the primary analysis includes participants who completed the study and responded to the survey. Participants who missed a visit were sent the survey electronically, but some did not complete every survey. This means the number contributing to the endpoint may vary across assessment times. Arm-specific response totals and reasons for missingness are not reported in the registry record.

Missing data can affect both precision and interpretation. General methods may use complete cases, imputation, models, or sensitivity analyses, each with assumptions and limitations. The endpoint population suggests a respondent-based analysis, but the record does not describe whether further methods were used. Imputation, sensitivity analyses, and a missing-data assumption are not reported in the registry record.

The registry lists zero analyses. It therefore does not provide a basis for evaluating how the multiple outcomes and time points were handled, which participants were included in each analysis, or whether any adjustment was made. The analysis populations beyond the primary endpoint's stated completer-and-responder group are not reported in the registry record.

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.

Most frequent serious adverse event terms

Serious events with the highest arm-level rate: hospitalization due to anasarca (21.4%), hospitalization due to fever, concern for infection, or immunosuppression (21.4%), hospitalization due to acute kidney injury (7.1%), hospitalization due to peritonitis (3.6%), hospitalization due to electrolyte derangements (3.6%).

The registry reports serious adverse events among participants at risk in both arms. In the UrApp group, 5 of 30 participants at risk had a serious adverse event; in the standard-of-care group, 9 of 28 participants at risk had one. No deaths are listed, and no other adverse events are listed in either arm. These are descriptive counts in the registry record, not an adjusted comparison or a test of differences between groups.

The record lists serious-event terms including hospitalization due to anasarca (21.4%) and hospitalization due to fever, concern for infection, or immunosuppression (21.4%). Other listed terms are hospitalization due to acute kidney injury (7.1%), peritonitis (3.6%), electrolyte derangements (3.6%), and high blood sugar (3.6%). The record does not clarify in this listing the denominator underlying each percentage, so the percentages should not be converted into participant counts or interpreted as arm-specific risks.

Serious-event counts do not by themselves show whether events were caused by an intervention, and a difference in counts is not necessarily evidence of a difference in underlying risk. Interpreting safety requires context about event definitions, ascertainment, exposure, and uncertainty. Those details and a statistical comparison are not reported in the registry record.

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

The registry record establishes that a randomized, parallel trial compared UrApp plus standard of care with standard of care for childhood idiopathic nephrotic syndrome. It identifies medication adherence as the primary endpoint and explains how adherence is defined from caregiver survey responses. It also records the assessment times and describes survey nonresponse. These details clarify what the trial intended to measure and why the available respondent population matters.

The record does not report the primary endpoint counts or an estimate comparing the arms. It therefore cannot establish whether adherence differed, how large any difference was, or how precisely such a difference was estimated. A completed status does not itself supply the outcome results. The absence of inferential analyses in the registry record also means there is no reported test to interpret. No conclusion about the direction, size, or statistical significance of the adherence comparison follows from the design description alone.

The listed serious adverse event counts are descriptive. They do not answer the primary adherence question, establish causation, or give an adjusted safety comparison. The listed event percentages also lack a stated denominator in the record, limiting what can be inferred from them.

More broadly, a randomized trial can support a comparison of assigned groups when the results, analysis population, and methods are clearly reported. Here, key information needed for that assessment—including the primary outcome data, uncertainty estimates, treatment of missing surveys, and analysis methods—is not reported in the registry record. This does not show that an effect is absent; it means the record does not supply the results needed to quantify or judge it.

13. Why This Trial Matters Statistically

This trial addresses a statistical problem that arises in many studies of self-management: the outcome is reported by participants or caregivers, measured repeatedly, and subject to incomplete survey response. The endpoint definition is explicit, but a threshold-based count only has a clear interpretation when its denominator and analysis population are also understood. Reporting counts alongside respondent totals at each assessment would help readers distinguish the number meeting the adherence definition from the likelihood of meeting it among those assessed.

The repeated assessments also make the choice of comparison important. Baseline, Month 6, and Month 12 can be used to describe different aspects of adherence, but the record does not report which comparison was designated for inference or how the association among responses from the same participant was handled. Stating the estimand, analysis method, uncertainty, and missing-data approach would make the statistical question and its answer easier to assess.

The trial's registry record also illustrates the difference between documenting a design and reporting results. Randomization and a defined endpoint describe how evidence was intended to be generated. They do not replace arm-level outcome data or the analysis required to estimate an effect. Likewise, serious-event counts are useful descriptive information, but are not a substitute for an outcome analysis or a causal safety assessment.

For clinical researchers and biostatisticians, these distinctions matter when evaluating evidence from a completed study. The registry provides a useful account of the intended comparison, the adherence scale, and some safety observations. At the same time, it does not report the primary endpoint results or the methods needed to assess their precision, leaving the central statistical question unanswered in the record.

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 principal limitation of the available record is that primary endpoint results are not reported. There are no adherence counts by arm or assessment time, no respondent totals for those assessments, and no estimated arm comparison or uncertainty interval. The record lists zero analyses, so the primary outcome cannot be statistically interpreted from the registry information.

The endpoint population is restricted to participants who completed the study and responded to the survey, and the record notes incomplete survey data. This can limit how representative the observed respondents are of all randomized participants. The number of missing surveys in each arm and at each time, reasons for missingness, and any methods used to address it are not reported in the registry record.

The primary endpoint is measured at baseline, Month 6, and Month 12, but the record does not specify the inferential comparison or the method for repeated observations. It also does not report a multiplicity approach for the several assessments and listed secondary outcomes. The planned analysis, sample-size justification, and any interim analyses are not reported in the registry record.

The registry enrollment is 58, while the brief description states that the study will include 60 caregivers. The record does not explain the difference. Although the status is completed, the final number analyzed for the primary endpoint is not reported. The serious adverse event percentages are listed without a clear denominator in the term listing, and the record does not provide a statistical safety comparison. These limitations concern what can be concluded from the registry record; they do not establish what information may exist elsewhere.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: UrApp for Childhood Nephrotic Syndrome Management (Incident Cohort) — NCT04075656.
• Registry record last updated 2025-11-06; status last verified 2025-10.
• Results first posted 2025-11-06.
• 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 a completed, randomized, parallel, unmasked trial of UrApp for childhood idiopathic nephrotic syndrome. Participants in the UrApp arm were to use the application for one year in addition to standard of care; the comparison arm was assigned standard of care for one year. Enrollment is recorded as 58, while the brief description refers to 60 caregivers. The primary outcome is the number of participants meeting a caregiver-survey medication-adherence threshold at baseline, Month 6, and Month 12.

The primary endpoint results and inferential analyses are not reported in the registry record. The endpoint population includes study completers who responded, and the record notes missing surveys. Serious adverse event counts are listed for both arms, along with serious-event terms and percentages. These observations are descriptive and do not resolve the primary adherence question. The record does not report the statistical plan, missing-data methods, or multiplicity approach.