UrApp for Childhood Nephrotic Syndrome Management (Incident Cohort)

• ClinicalTrials.gov Identifier: NCT04075656
• Recruitment Status: Recruiting
• First Posted: September 3, 2019
• Last Update Posted: July 18, 2022
• Sponsor: Emory University
• Collaborator: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
• Information provided by (Responsible Party): Chia-shi Wang, Emory University

Tracking Information

• First Submitted Date: August 29, 2019
• First Posted Date: September 3, 2019
• Last Update Posted Date: July 18, 2022
• Actual Study Start Date: September 17, 2019
• Estimated Primary Completion Date: August 31, 2024 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: August 29, 2019)

• Change in Medication Adherence [ Time Frame: Baseline, Month 6, Month 12 ]
  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.
• Change in Urine Monitoring Adherence [ Time Frame: Baseline, Month 6, Month 12 ]
  Adherence with urine protein monitoring is defined as checking, on average, at least 2 times a week in the month preceding the assessment.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: August 29, 2019)

• Change in Self-efficacy [ Time Frame: Baseline, Month 6, Month 12 ]
  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.
• Change in Patient-Reported Outcomes Measurement Information System - Pediatric Quality of Life Inventory (PedsQL) Score [ Time Frame: Baseline, Month 6, Month 12 ]
  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.
• Change in Delayed Relapse Detection [ Time Frame: Month 6, Month 12 ]
  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.
• Change in Hospitalizations [ Time Frame: Month 6, Month 12 ]
  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.

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: UrApp for Childhood Nephrotic Syndrome Management (Incident Cohort)
• Official Title: A Pilot Trial of UrApp, a Novel Mobile Application for Childhood Nephrotic Syndrome Management
• Brief Summary: 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.

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
• Study Phase: Not Applicable
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Prevention
• Condition: Idiopathic Nephrotic Syndrome

Intervention

• 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.

Study Arms

• 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.
  Interventions:

  • Behavioral: Standard of Care
  • Behavioral: UrApp
• Active Comparator: Standard of Care
  Participants randomized to this study arm will use receive the standard of care for one year.
  Intervention: Behavioral: Standard of Care

• 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.

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: August 29, 2019): 60
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: August 31, 2024
• Estimated Primary Completion Date: August 31, 2024 (Final data collection date for primary outcome measure)

Eligibility Criteria

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)

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Margret Kamel, PhD; 404-712-9923; mkamel@emory.edu

• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT04075656
• Other Study ID Numbers: IRB00108912; 1K23DK118189-01A1 ( U.S. NIH Grant/Contract )
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: Yes
• Plan Description: All of the individual participant data collected during the trial will be available for sharing, after deidentification.
• Supporting Materials: Study Protocol
• Supporting Materials: Statistical Analysis Plan (SAP)
• Supporting Materials: Informed Consent Form (ICF)
• Supporting Materials: Clinical Study Report (CSR)
• Supporting Materials: Analytic Code
• Time Frame: Data will be available for sharing immediately following publication, with no end date.
• 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.

• Current Responsible Party: Chia-shi Wang, Emory University
• Original Responsible Party: Chia-shi Wang, Emory University, Assistant Professor
• Current Study Sponsor: Emory University
• Original Study Sponsor: Same as current
• Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Investigators

• Principal Investigator: Chia-shi Wang, MD, MS; Emory University

• PRS Account: Emory University
• Verification Date: July 2022