UrApp for Childhood Nephrotic Syndrome Management (Incident Cohort)

A Pilot Trial of UrApp, a Novel Mobile Application for Childhood Nephrotic Syndrome Management

Idiopathic nephrotic syndrome is one of the most common chronic kidney diseases in children. Patients suffer from frequent disease relapses and complications. Self-management is difficult for families and nonadherence is common, with adverse effects on the children's health. UrApp is a mobile application designed to assist families with nephrotic syndrome management. This study will examine whether providing the children's caregivers (or adolescent patients) with UrApp improves self-management and disease outcomes. This study will include 60 caregivers of children with newly diagnosed nephrotic syndrome. Participants will be randomized 1:1 to UrApp or standard of care and followed for 1 year.

Study Overview

• Status: Recruiting
• Conditions: Idiopathic Nephrotic Syndrome
• Intervention / Treatment: Behavioral: Standard of Care; Behavioral: UrApp

Detailed Description

Idiopathic nephrotic syndrome (NS) is one of the most common chronic kidney diseases in children, with a prevalence of approximately 16 cases per 100,000 children. NS is characterized by heavy urinary losses of protein leading to hypoalbuminemia, edema, and hyperlipidemia. Children are treated with high-dose corticosteroids on presentation, and >80% respond to treatment with resolution of proteinuria and symptoms. However, 80-90% of the children initially sensitive to corticosteroids will experience disease relapse, with more than half relapsing frequently or becoming dependent on corticosteroids to maintain remission. During a relapse, patients can suffer from anasarca, acute kidney injury, serious infections, or thromboembolic events.

Management of children with NS entails long-term outpatient surveillance and treatment. Home care includes the important standard-of-care task of urine monitoring to follow the relapsing-remitting nature of the disease. New proteinuria signals disease relapse before the development of overt symptoms such as edema. Thus, patients are instructed to alert their providers to the occurrence of proteinuria in a timely manner so that corticosteroids can be initiated or adjusted to treat each relapse and prevent acute disease complications. It is also important for the patients to track urine protein for resolution so that corticosteroids can be stopped or reduced to minimize steroid toxicity.

Not unlike other chronic, relapsing-remitting pediatric disorders, self-management is difficult for NS patients and their caregivers. Mobile health (mHealth) is a promising, rapidly growing field in disease management. In NS, there are numerous aspects of self-management that may be facilitated by a mobile app. First, the visual analysis of a urine test strip is subject to human error, including reading the wrong reagent block and erroneous assessments of color. This can be improved through using a smartphone's camera and computer to read and analyze test strip results. Second, caregivers must remember to check their child's urine, recall results, and understand what the results mean: the demands are taxing in that disease relapse is defined as urine protein ≥2+ for 3 consecutive days and remission is defined as negative/trace urine protein for 3 consecutive days. Apps, with their inherent interactivity, can provide reminders for urine testing, capture the results, and analyze trends to detect disease relapse/remission. Apps can alert a caregiver to seek medical attention and directly transmit test results to providers. Lastly, apps can provide medication reminders for NS patients, who are on highly complex medication regimens.

UrApp was iteratively developed by an expert panel of two pediatric nephrologists and three research engineers with expertise in human-computer interaction. App features were devised by the clinicians to support elements of chronic care management according to the Chronic Care Model and tasks that are challenging for caregivers. This study will include 60 caregivers of children with newly diagnosed nephrotic syndrome. Participants will be randomized 1:1 to UrApp or standard of care and followed for 1 year. In addition to the study outcome measures, user feedback will be collected via survey, interview, and by stakeholder meetings to inform app refinement.

• Study Type: Interventional
• Enrollment (Estimated): 60
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Margret Kamel, PhD; Phone Number: 404-712-9923; Email: mkamel@emory.edu

Study Locations

• United States
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Recruiting
      • Children's Healthcare of Altanta
  • Minnesota
    • Minneapolis, Minnesota, United States, 55454
      • Recruiting
      • University of Minnesota Children's Hospital
      • Contact: Michelle Rheault, MD; Phone Number: 612-626-2922; Email: rheau002@umn.edu
      • Principal Investigator: Michelle Rheault, MD
  • Oregon
    • Portland, Oregon, United States, 97239
      • Recruiting
      • Oregon Health and Science University
      • Contact: Amira Al-Uzri, MD; Phone Number: 503-494-7327; Email: aluzria@ohsu.edu
      • Principal Investigator: Amira Al-Uzri, MD
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Suspended
      • Medical University of South Carolina

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Caregivers of patients ages 1-17 with steroid sensitive nephrotic syndrome (clinical diagnosis with edema, nephrotic range proteinuria [urine protein to creatinine ratio >2 mg/mg, or ≥ 300 mg/dL or ≥ 3+ protein on urine dipstick], and hypoalbuminemia ≤ 2.5 g/dL; resolution of proteinuria [negative/trace protein on urine dipstick] within 4 weeks of corticosteroid treatment)
• Caregivers of pediatric patients with steroid sensitive nephrotic syndrome diagnosed within 42 days at the time of enrollment
• Access to internet/wireless fidelity (Wi-Fi) in the home
• Caregiver proficiency with the English language

Exclusion Criteria:

• Caregivers of pediatric patients with end-stage kidney disease
• Caregivers of pediatric patients with renal transplantation
• Caregivers of pediatric patients with clinical or histologic evidence of secondary nephrotic syndrome (e.g., systemic lupus erythematosus)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: UrApp; Participants randomized to this study arm will use the UrApp mobile application for one year, in addition to receiving the standard of care.	Behavioral: Standard of Care; Participants will be provided a folder of educational material on NS, including general information on symptoms, treatments, and possible complications; healthy diet for children taking corticosteroids; and low sodium diet. Site research staff will 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 will be provided to ensure that each participant can check their urine daily for protein. Participants will be instructed to check their urine daily for protein and call their provider within 1 business day for relapses and remissions.; Behavioral: UrApp; Caregivers/parents will download UrApp at the baseline visit. UrApp contains instructional videos to guide users. The telephone number of the patient's provider will be entered into UrApp and participants will be able to call their providers directly through UrApp. The email address of the Emory study staff will also be entered. UrApp will automatically e-mail test results to the research staff when elected by the users. Participants will be 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 will be communicated to the treating physician within 1 business day.
Active Comparator: Standard of Care; Participants randomized to this study arm will use receive the standard of care for one year.	Behavioral: Standard of Care; Participants will be provided a folder of educational material on NS, including general information on symptoms, treatments, and possible complications; healthy diet for children taking corticosteroids; and low sodium diet. Site research staff will 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 will be provided to ensure that each participant can check their urine daily for protein. Participants will be instructed to check their urine daily for protein and call their provider within 1 business day for relapses and remissions.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Medication Adherence	Adherence to medications will be 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.	Baseline, Month 6, Month 12
Change in Urine Monitoring Adherence	Adherence with urine protein monitoring is defined as checking, on average, at least 2 times a week in the month preceding the assessment.	Baseline, Month 6, Month 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Self-efficacy	Self-efficacy for managing nephrotic syndrome will be 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.	Baseline, Month 6, Month 12
Change in Patient-Reported Outcomes Measurement Information System - Pediatric Quality of Life Inventory (PedsQL) Score	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.	Baseline, Month 6, Month 12
Change in Delayed Relapse Detection	The number of delayed relapses detected will be compared between study arms. Patient medical charts will be 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.	Month 6, Month 12
Change in Hospitalizations	The number of hospitalizations will be compared between study arms. Medical records will be 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.	Month 6, Month 12

Collaborators and Investigators

• Sponsor: Emory University
• Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
• Investigators: Principal Investigator: Chia-shi Wang, MD, MS, Emory University

Publications and helpful links

General Publications

• Wang CS, Escoffery C, Patzer RE, McCracken C, Ross D, Rheault MN, Al-Uzri A, Greenbaum LA. A dual efficacy-implementation trial of a novel mobile application for childhood nephrotic syndrome management: the UrApp for childhood nephrotic syndrome management pilot study protocol (UrApp pilot study). BMC Nephrol. 2020 Apr 9;21(1):125. doi: 10.1186/s12882-020-01778-w.

Study record dates

Study Major Dates

• Study Start (Actual): September 17, 2019
• Primary Completion (Estimated): August 31, 2024
• Study Completion (Estimated): August 31, 2024

Study Registration Dates

• First Submitted: August 29, 2019
• First Submitted That Met QC Criteria: August 29, 2019
• First Posted (Actual): September 3, 2019

Study Record Updates

• Last Update Posted (Actual): September 13, 2023
• Last Update Submitted That Met QC Criteria: September 8, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Keywords: Pediatrics; mHealth; Behavioral intervention
• Additional Relevant MeSH Terms: Pathologic Processes; Kidney Diseases; Urologic Diseases; Disease; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Syndrome; Nephrotic Syndrome; Nephrosis
• Other Study ID Numbers: IRB00108912; 1K23DK118189-01A1 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: All of the individual participant data collected during the trial will be available for sharing, after deidentification.
• IPD Sharing Time Frame: Data will be available for sharing immediately following publication, with no end date.
• IPD Sharing Access Criteria: Data will be available for sharing with researchers who provide a methodologically sound proposal, in order to achieve aims in the approved proposal. Proposals should be directed to chia-shi.wang@emory.edu. To gain access, data requesters will need to sign a data access agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; ANALYTIC_CODE; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No