Trial Outcomes & Findings for UrApp for Childhood Nephrotic Syndrome Management (Incident Cohort) (NCT NCT04075656)

NCT ID: NCT04075656

Last Updated: 2025-11-06

Results Overview

Adherence to medications is evaluated via caregiver survey with the validated 4-question Morisky, Green, and Levine (MGL) Adherence Scale. Response options are Yes (0) or No (1) and total scores range from 0 to 4 with higher scores indicating better medication adherence. Adherence will be defined as a score of ≥3 on the MGL scale.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

58 participants

Primary outcome timeframe

Baseline, Month 6, Month 12

Results posted on

2025-11-06

Participant Flow

Caregiver participants were recruited from Children's Healthcare of Atlanta, in Atlanta, Georgia, Oregon Health and Science University, in Portland, Oregon, and the University of Minnesota Children's Hospital in Minneapolis, Minnesota, USA. Participant enrollment began September 17, 2019, and all follow-up assessments were completed by April 25, 2025.

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.

Participant milestones

Participant milestones
Measure
Caregivers Using UrApp with Standard of Care
Caregiver participants randomized to this study arm use the UrApp mobile application for one year, in addition to receiving the standard of care. UrApp: Caregivers/parents download UrApp at the baseline visit. UrApp contains instructional videos to guide users. The telephone number of the patient's provider is entered into UrApp and participants are able to call their providers directly through UrApp. The email address of the Emory study staff is also entered. UrApp automatically e-mails test results to the research staff when elected by the users. Participants are asked by study staff, and reminded by the app, to call their providers and send urine testing results to the study staff whenever there is a relapse or remission. When the study staff receives alerts of a relapse/remission via UrApp, the information is communicated to the treating physician within 1 business day.
Caregivers Receiving the Standard of Care
Caregiver participants randomized to this study arm receive the standard of care for one year. Standard of Care: Participants are provided a folder of educational material on nephrotic syndrome (NS), including general information on symptoms, treatments, and possible complications; healthy diet for children taking corticosteroids; and low sodium diet. Site research staff demonstrate how to check urine for protein with test strips and educate patients on the definitions of disease relapse and remission. Urine test strips and urine protein logs are provided to ensure that each participant can check their urine daily for protein. Participants are instructed to check their urine daily for protein and call their provider within 1 business day for relapses and remissions.
Overall Study
STARTED
30
28
Overall Study
COMPLETED
25
23
Overall Study
NOT COMPLETED
5
5

Reasons for withdrawal

Reasons for withdrawal
Measure
Caregivers Using UrApp with Standard of Care
Caregiver participants randomized to this study arm use the UrApp mobile application for one year, in addition to receiving the standard of care. UrApp: Caregivers/parents download UrApp at the baseline visit. UrApp contains instructional videos to guide users. The telephone number of the patient's provider is entered into UrApp and participants are able to call their providers directly through UrApp. The email address of the Emory study staff is also entered. UrApp automatically e-mails test results to the research staff when elected by the users. Participants are asked by study staff, and reminded by the app, to call their providers and send urine testing results to the study staff whenever there is a relapse or remission. When the study staff receives alerts of a relapse/remission via UrApp, the information is communicated to the treating physician within 1 business day.
Caregivers Receiving the Standard of Care
Caregiver participants randomized to this study arm receive the standard of care for one year. Standard of Care: Participants are provided a folder of educational material on nephrotic syndrome (NS), including general information on symptoms, treatments, and possible complications; healthy diet for children taking corticosteroids; and low sodium diet. Site research staff demonstrate how to check urine for protein with test strips and educate patients on the definitions of disease relapse and remission. Urine test strips and urine protein logs are provided to ensure that each participant can check their urine daily for protein. Participants are instructed to check their urine daily for protein and call their provider within 1 business day for relapses and remissions.
Overall Study
Withdrawal by Subject
5
5

Baseline Characteristics

UrApp for Childhood Nephrotic Syndrome Management (Incident Cohort)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
UrApp
n=30 Participants
Caregiver participants randomized to this study arm use the UrApp mobile application for one year, in addition to receiving the standard of care. UrApp: Caregivers/parents download UrApp at the baseline visit. UrApp contains instructional videos to guide users. The telephone number of the patient's provider is entered into UrApp and participants are able to call their providers directly through UrApp. The email address of the Emory study staff is also entered. UrApp automatically e-mails test results to the research staff when elected by the users. Participants are asked by study staff, and reminded by the app, to call their providers and send urine testing results to the study staff whenever there is a relapse or remission. When the study staff receives alerts of a relapse/remission via UrApp, the information is communicated to the treating physician within 1 business day.
Standard of Care
n=28 Participants
Caregiver participants randomized to this study arm receive the standard of care for one year. Standard of Care: Participants are provided a folder of educational material on nephrotic syndrome (NS), including general information on symptoms, treatments, and possible complications; healthy diet for children taking corticosteroids; and low sodium diet. Site research staff demonstrate how to check urine for protein with test strips and educate patients on the definitions of disease relapse and remission. Urine test strips and urine protein logs are provided to ensure that each participant can check their urine daily for protein. Participants are instructed to check their urine daily for protein and call their provider within 1 business day for relapses and remissions.
Total
n=58 Participants
Total of all reporting groups
Age, Categorical
<=18 years
0 Participants
n=49 Participants
0 Participants
n=50 Participants
0 Participants
n=50 Participants
Age, Categorical
Between 18 and 65 years
30 Participants
n=49 Participants
28 Participants
n=50 Participants
58 Participants
n=50 Participants
Age, Categorical
>=65 years
0 Participants
n=49 Participants
0 Participants
n=50 Participants
0 Participants
n=50 Participants
Sex: Female, Male
Female
11 Participants
n=49 Participants
9 Participants
n=50 Participants
20 Participants
n=50 Participants
Sex: Female, Male
Male
19 Participants
n=49 Participants
19 Participants
n=50 Participants
38 Participants
n=50 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
3 Participants
n=49 Participants
4 Participants
n=50 Participants
7 Participants
n=50 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
27 Participants
n=49 Participants
24 Participants
n=50 Participants
51 Participants
n=50 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=49 Participants
0 Participants
n=50 Participants
0 Participants
n=50 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=49 Participants
0 Participants
n=50 Participants
0 Participants
n=50 Participants
Race (NIH/OMB)
Asian
6 Participants
n=49 Participants
2 Participants
n=50 Participants
8 Participants
n=50 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=49 Participants
0 Participants
n=50 Participants
0 Participants
n=50 Participants
Race (NIH/OMB)
Black or African American
5 Participants
n=49 Participants
7 Participants
n=50 Participants
12 Participants
n=50 Participants
Race (NIH/OMB)
White
18 Participants
n=49 Participants
16 Participants
n=50 Participants
34 Participants
n=50 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=49 Participants
0 Participants
n=50 Participants
0 Participants
n=50 Participants
Race (NIH/OMB)
Unknown or Not Reported
1 Participants
n=49 Participants
3 Participants
n=50 Participants
4 Participants
n=50 Participants
Region of Enrollment
United States
30 Participants
n=49 Participants
28 Participants
n=50 Participants
58 Participants
n=50 Participants

PRIMARY outcome

Timeframe: Baseline, Month 6, Month 12

Population: 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.

Adherence to medications is evaluated via caregiver survey with the validated 4-question Morisky, Green, and Levine (MGL) Adherence Scale. Response options are Yes (0) or No (1) and total scores range from 0 to 4 with higher scores indicating better medication adherence. Adherence will be defined as a score of ≥3 on the MGL scale.

Outcome measures

Outcome measures
Measure
UrApp
n=25 Participants
Caregiver participants randomized to this study arm use the UrApp mobile application for one year, in addition to receiving the standard of care.
Standard of Care
n=22 Participants
Caregiver participants randomized to this study arm receive the standard of care for one year.
Number of Participants Reporting Medication Adherence
Participants with score ≥3 at Month 6
19 Participants
18 Participants
Number of Participants Reporting Medication Adherence
Participants with score ≥3 at Month 12
15 Participants
16 Participants
Number of Participants Reporting Medication Adherence
Participants with score ≥3 at Baseline
25 Participants
21 Participants

PRIMARY outcome

Timeframe: Baseline, Month 6, Month 12

Population: 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.

Caregiver adherence with urine protein monitoring is assessed by asking caregivers on average how often they checked their child's urine for protein over the past month. Possible responses are "once a week", "2-4 times a week" or " 5-7 times a week." Adherence is defined as checking urine at least 2 times per week.

Outcome measures

Outcome measures
Measure
UrApp
n=25 Participants
Caregiver participants randomized to this study arm use the UrApp mobile application for one year, in addition to receiving the standard of care.
Standard of Care
n=23 Participants
Caregiver participants randomized to this study arm receive the standard of care for one year.
Number of Participants Reporting Adherence With Urine Monitoring
Adherence at Baseline
21 Participants
18 Participants
Number of Participants Reporting Adherence With Urine Monitoring
Adherence at Month 6
19 Participants
18 Participants
Number of Participants Reporting Adherence With Urine Monitoring
Adherence at Month 12
14 Participants
14 Participants

SECONDARY outcome

Timeframe: Baseline, Month 6, Month 12

Population: 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.

Self-efficacy of caregivers for managing nephrotic syndrome of pediatric patients is assessed with a 3-item instrument adapted from the Chronic Disease Self-Efficacy Scales. Responses are given on a 10-point scale where 0 = not confident at all and 10 = totally confident. Total scores range from 0 to 30 with higher scores indicating increased self-efficacy.

Outcome measures

Outcome measures
Measure
UrApp
n=25 Participants
Caregiver participants randomized to this study arm use the UrApp mobile application for one year, in addition to receiving the standard of care.
Standard of Care
n=23 Participants
Caregiver participants randomized to this study arm receive the standard of care for one year.
Caregiver Self-efficacy Score
Baseline
8.97 score on a scale
Standard Deviation 1.84
9.35 score on a scale
Standard Deviation 1.26
Caregiver Self-efficacy Score
Month 6
9.65 score on a scale
Standard Deviation 1.26
9.42 score on a scale
Standard Deviation 0.81
Caregiver Self-efficacy Score
Month 12
9.21 score on a scale
Standard Deviation 1.51
9.49 score on a scale
Standard Deviation 0.88

SECONDARY outcome

Timeframe: Baseline, Month 6, Month 12

Population: 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.

The 23-item PedsQL was developed as part of the NIH Roadmap Initiative to create universal measures for patient-reported outcomes and contains questions in the domains of social-peer, depression, anxiety, mobility, and function. Responses are given on a 5-point scale where 0 = never and 4 = almost always. Items are reverse scored and linearly transformed to a scale of 0 to 100, where higher total mean scores indicate a better quality of life.

Outcome measures

Outcome measures
Measure
UrApp
n=25 Participants
Caregiver participants randomized to this study arm use the UrApp mobile application for one year, in addition to receiving the standard of care.
Standard of Care
n=23 Participants
Caregiver participants randomized to this study arm receive the standard of care for one year.
Patient-Reported Outcomes Measurement Information System - Pediatric Quality of Life Inventory (PedsQL) Score for Parents
Baseline
83 score on a scale
Standard Deviation 16
85 score on a scale
Standard Deviation 12
Patient-Reported Outcomes Measurement Information System - Pediatric Quality of Life Inventory (PedsQL) Score for Parents
Month 6
86 score on a scale
Standard Deviation 18
84 score on a scale
Standard Deviation 14
Patient-Reported Outcomes Measurement Information System - Pediatric Quality of Life Inventory (PedsQL) Score for Parents
Month 12
88 score on a scale
Standard Deviation 13
76 score on a scale
Standard Deviation 17

SECONDARY outcome

Timeframe: Baseline, Month 6, Month 12

Population: This analysis includes pediatric patients who completed the study and responded to this survey. Survey data are missing for some pediatric patients. If a caregiver participant missed a study visit they were sent the survey electronically but some pediatric patients did not complete this survey.

The 23-item PedsQL was developed as part of the NIH Roadmap Initiative to create universal measures for patient-reported outcomes, and contains questions in the domains of social-peer, depression, anxiety, mobility, and function. Responses are given on a 5-point scale where 0 = never and 4 = almost always. Items are reverse scored and linearly transformed to a scale of 0 to 100, where higher total mean scores indicate a better quality of life.

Outcome measures

Outcome measures
Measure
UrApp
n=24 Participants
Caregiver participants randomized to this study arm use the UrApp mobile application for one year, in addition to receiving the standard of care.
Standard of Care
n=23 Participants
Caregiver participants randomized to this study arm receive the standard of care for one year.
Patient-Reported Outcomes Measurement Information System - Pediatric Quality of Life Inventory (PedsQL) Score for Pediatric Patients
Baseline
91 score on a scale
Standard Deviation 7
83 score on a scale
Standard Deviation 15
Patient-Reported Outcomes Measurement Information System - Pediatric Quality of Life Inventory (PedsQL) Score for Pediatric Patients
Month 6
89 score on a scale
Standard Deviation 10
84 score on a scale
Standard Deviation 7
Patient-Reported Outcomes Measurement Information System - Pediatric Quality of Life Inventory (PedsQL) Score for Pediatric Patients
Month 12
94 score on a scale
Standard Deviation 5
81 score on a scale
Standard Deviation 14

SECONDARY outcome

Timeframe: Month 6, Month 12

Population: Information for this outcome measure was collected from medical records. Some participants did not attend clinic visits corresponding with the time frames of this study. The overall number of participants analyzed reflects caregiver participants who contributed data at either the 6 month or 12 month time points, while the number of participants analyzed at each time point are pediatric patients who had a least one relapse during the time frame.

Relapses detected only after patients experienced symptoms of relapse, indicating delayed detection, is compared between study arms. Patient medical charts were reviewed for occurrence and frequency of delayed relapse reporting which is defined as a relapse that was not reported to the treating physician until clinical manifestations or complications occurred and/or only discovered during planned or unplanned visits or hospitalizations.

Outcome measures

Outcome measures
Measure
UrApp
n=23 Participants
Caregiver participants randomized to this study arm use the UrApp mobile application for one year, in addition to receiving the standard of care.
Standard of Care
n=22 Participants
Caregiver participants randomized to this study arm receive the standard of care for one year.
Number Pediatric Patients With Delayed Detection of Relapses
Month 6
2 Participants
7 Participants
Number Pediatric Patients With Delayed Detection of Relapses
Month 12
1 Participants
4 Participants

SECONDARY outcome

Timeframe: Month 6, Month 12

Population: Information for this outcome measure was collected from medical records. Some participants did not attend clinic visits corresponding with the time frames of this study. The overall number of participants analyzed reflects caregiver participants who contributed data at either the 6 month or 12 month time points, while the number of participants analyzed at each time point are pediatric patients who had medical record data to be reviewed for the indicated study time point.

Medical records were reviewed for the primary reason for admission and NS disease complications, such as bacterial peritonitis, septicemia, shock, blood clot(s), acute kidney injury, and seizures from hyponatremia or hypertension.

Outcome measures

Outcome measures
Measure
UrApp
n=23 Participants
Caregiver participants randomized to this study arm use the UrApp mobile application for one year, in addition to receiving the standard of care.
Standard of Care
n=20 Participants
Caregiver participants randomized to this study arm receive the standard of care for one year.
Number of Hospitalizations Among Pediatric Patients
Month 6
3 count of hospitalizations
7 count of hospitalizations
Number of Hospitalizations Among Pediatric Patients
Month 12
4 count of hospitalizations
10 count of hospitalizations

Adverse Events

UrApp

Serious events: 5 serious events
Other events: 0 other events
Deaths: 0 deaths

Standard of Care

Serious events: 9 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
UrApp
n=30 participants at risk
Pediatric patients of caregiver participants randomized to this study arm use the UrApp mobile application for one year, in addition to receiving the standard of care.
Standard of Care
n=28 participants at risk
Pediatric patients of caregiver participants randomized to this study arm receive the standard of care for one year.
Renal and urinary disorders
Hospitalization due to anasarca
13.3%
4/30 • Number of events 4 • Information on adverse events among pediatric patients was collected beginning one month after initiating the intervention and Months 6 and 12 (up to 11 months).
During study visits, caregivers were asked if their child had any serious illnesses or trips to the hospital since the last study visit. Information on non-serious adverse events among pediatric patients was not collected. Information about adverse events impacting caregiver participants was not collected.
21.4%
6/28 • Number of events 11 • Information on adverse events among pediatric patients was collected beginning one month after initiating the intervention and Months 6 and 12 (up to 11 months).
During study visits, caregivers were asked if their child had any serious illnesses or trips to the hospital since the last study visit. Information on non-serious adverse events among pediatric patients was not collected. Information about adverse events impacting caregiver participants was not collected.
Infections and infestations
Hospitalization due to fever, concern for infection, or immunosuppression
10.0%
3/30 • Number of events 3 • Information on adverse events among pediatric patients was collected beginning one month after initiating the intervention and Months 6 and 12 (up to 11 months).
During study visits, caregivers were asked if their child had any serious illnesses or trips to the hospital since the last study visit. Information on non-serious adverse events among pediatric patients was not collected. Information about adverse events impacting caregiver participants was not collected.
21.4%
6/28 • Number of events 6 • Information on adverse events among pediatric patients was collected beginning one month after initiating the intervention and Months 6 and 12 (up to 11 months).
During study visits, caregivers were asked if their child had any serious illnesses or trips to the hospital since the last study visit. Information on non-serious adverse events among pediatric patients was not collected. Information about adverse events impacting caregiver participants was not collected.
Infections and infestations
Hospitalization due to peritonitis
0.00%
0/30 • Information on adverse events among pediatric patients was collected beginning one month after initiating the intervention and Months 6 and 12 (up to 11 months).
During study visits, caregivers were asked if their child had any serious illnesses or trips to the hospital since the last study visit. Information on non-serious adverse events among pediatric patients was not collected. Information about adverse events impacting caregiver participants was not collected.
3.6%
1/28 • Number of events 1 • Information on adverse events among pediatric patients was collected beginning one month after initiating the intervention and Months 6 and 12 (up to 11 months).
During study visits, caregivers were asked if their child had any serious illnesses or trips to the hospital since the last study visit. Information on non-serious adverse events among pediatric patients was not collected. Information about adverse events impacting caregiver participants was not collected.
Renal and urinary disorders
Hospitalization due to acute kidney injury
6.7%
2/30 • Number of events 2 • Information on adverse events among pediatric patients was collected beginning one month after initiating the intervention and Months 6 and 12 (up to 11 months).
During study visits, caregivers were asked if their child had any serious illnesses or trips to the hospital since the last study visit. Information on non-serious adverse events among pediatric patients was not collected. Information about adverse events impacting caregiver participants was not collected.
7.1%
2/28 • Number of events 2 • Information on adverse events among pediatric patients was collected beginning one month after initiating the intervention and Months 6 and 12 (up to 11 months).
During study visits, caregivers were asked if their child had any serious illnesses or trips to the hospital since the last study visit. Information on non-serious adverse events among pediatric patients was not collected. Information about adverse events impacting caregiver participants was not collected.
Renal and urinary disorders
Hospitalization due to electrolyte derangements
0.00%
0/30 • Information on adverse events among pediatric patients was collected beginning one month after initiating the intervention and Months 6 and 12 (up to 11 months).
During study visits, caregivers were asked if their child had any serious illnesses or trips to the hospital since the last study visit. Information on non-serious adverse events among pediatric patients was not collected. Information about adverse events impacting caregiver participants was not collected.
3.6%
1/28 • Number of events 1 • Information on adverse events among pediatric patients was collected beginning one month after initiating the intervention and Months 6 and 12 (up to 11 months).
During study visits, caregivers were asked if their child had any serious illnesses or trips to the hospital since the last study visit. Information on non-serious adverse events among pediatric patients was not collected. Information about adverse events impacting caregiver participants was not collected.
Endocrine disorders
Hospitalization due to high blood sugar
0.00%
0/30 • Information on adverse events among pediatric patients was collected beginning one month after initiating the intervention and Months 6 and 12 (up to 11 months).
During study visits, caregivers were asked if their child had any serious illnesses or trips to the hospital since the last study visit. Information on non-serious adverse events among pediatric patients was not collected. Information about adverse events impacting caregiver participants was not collected.
3.6%
1/28 • Number of events 1 • Information on adverse events among pediatric patients was collected beginning one month after initiating the intervention and Months 6 and 12 (up to 11 months).
During study visits, caregivers were asked if their child had any serious illnesses or trips to the hospital since the last study visit. Information on non-serious adverse events among pediatric patients was not collected. Information about adverse events impacting caregiver participants was not collected.
Infections and infestations
Hospitalization due to gastrointestinal parasite
0.00%
0/30 • Information on adverse events among pediatric patients was collected beginning one month after initiating the intervention and Months 6 and 12 (up to 11 months).
During study visits, caregivers were asked if their child had any serious illnesses or trips to the hospital since the last study visit. Information on non-serious adverse events among pediatric patients was not collected. Information about adverse events impacting caregiver participants was not collected.
3.6%
1/28 • Number of events 1 • Information on adverse events among pediatric patients was collected beginning one month after initiating the intervention and Months 6 and 12 (up to 11 months).
During study visits, caregivers were asked if their child had any serious illnesses or trips to the hospital since the last study visit. Information on non-serious adverse events among pediatric patients was not collected. Information about adverse events impacting caregiver participants was not collected.

Other adverse events

Adverse event data not reported

Additional Information

Chia-shi Wang, MD, MS

Emory University

Phone: 404-727-5728

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place