Interview Study of Adult and Child Patients and Parents of Children With Swelling Due to Nephrotic Syndrome. (Prepare-NS)

Preparing a Clinical Outcomes Assessment Set for Nephrotic Syndrome

Researchers from the University of Michigan and Northwestern University are studying people's experiences with swelling caused by Nephrotic Syndrome. Interviews with patients (child and adult) and parents of young children will be conducted. The information collected from the interviews will be used to develop a survey to use when testing new medications for Nephrotic Syndrome.

Please consider participating in a 1-hour long interview with the Prepare-NS research study to discuss children and adults experiences with swelling.

Study Overview

• Status: Recruiting
• Conditions: Edema; Fluid Overload; Glomerulosclerosis, Focal Segmental; Nephrotic Syndrome; Glomerular Disease; Minimal Change Disease; Membranous Nephropathy; Nephrotic Syndrome in Children; Nephrotic Syndrome, Minimal Change; FSGS; Minimal Change Nephrotic Syndrome; IgM Nephropathy; Nephrotic Syndrome With Edema (Diagnosis)
• Intervention / Treatment: Behavioral: Interview

Detailed Description

This registration includes concept elicitation, analysis and creation of complementary survey measures focused on edema/fluid overload of nephrotic syndrome. The measures to be developed include an Observer Reported Outcome (ObsRO) and Patient Reported Outcome (PRO). The ObsRO and PRO substudies are Institutional Review Board (IRB) approved at release.

The researcher's goal is to produce core outcome sets of patient-rated and observer reported Fluid Overload (edema) measures that can be applied across the fluid overload severity continuum, fit to help drug development in this area.

• Study Type: Observational
• Enrollment (Estimated): 150

Contacts and Locations

• Study Contact: Name: Tina Creguer; Phone Number: 734-936-3590; Email: tcreguer@umich.edu

Study Locations

• United States
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • Recruiting
      • University of Michigan
      • Principal Investigator: Eloise Salmon, M.D.
      • Contact: Tina Creguer; Phone Number: 734-936-3590; Email: tcreguer@umich.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

ObsRO Study Population: Data collection will involve approximately 20-40 parents/guardians of children ages 2-11.999 with NS

PRO Study Population: Data collection will involve N = 60 to 95 patients ≥8 years of age with NS (depending on when concept saturation is achieved; see section 9.0 Statistical Considerations for more detail). Recruitment of patients with NS will occur across the United States. Enrollment will take place at the University of Michigan.

Description

Criteria for the Observer Reported Outcomes (ObsRO) cohort of the study:

Inclusion Criteria:

1. Parents/guardians must be able to read and understand English;
2. Parents/guardians must be caring for a child (ages 2-11.999) with a medically documented diagnosis of idiopathic (primary) Nephrotic Syndrome (NS) or primary or monogenic NS associated kidney disease. Populations with Primary NS Conditions: Focal segmental glomerulosclerosis (FSGS), Minimal Change Disease (MCD), Immunoglobulin M (IgM) Nephropathy, Membranous Nephropathy (MN), and childhood - onset nephrotic syndrome not biopsied;
3. The child must have a current or history of NS-associated edema within the past 3 months;
4. The child may have native kidney function or may be kidney transplant dependent with a history of post-transplant NS recurrence;
5. Parents/guardians must provide informed consent.

Exclusion Criteria:

1. Index case with dialysis dependence throughout the 3-month pre-enrollment period

Criteria for the Patient Reported Outcomes (PRO) cohort of the study:

Inclusion Criteria:

1. ≥8 years of age
2. Able to read and understand English

3. Primary (idiopathic) kidney disease that causes NS or monogenic NS associated kidney disease.

   i. Populations with Primary Nephrotic Syndrome (NS) Conditions include: FSGS, MCD, IgM nephropathy, MN, and childhood - onset nephrotic syndrome not biopsied ii. Kidney disease in the native kidneys or recurrent in kidney transplant

4. Current or history of NS-associated edema within the past 3 months
5. Kidney function with most recent estimated Glomerular Filtration Rate (eGFR) > 25 ml/min/1.73m2
6. Informed Consent: For patients ≥8 to <18 years of age: a parent or legal guardian provide informed consent and the patient must provide assent. Patients ≥18 years of age must provide informed consent.

Exclusion Criteria:

1. Native kidney disease participant with dialysis dependence during the 3-month pre-enrollment period OR kidney transplant recipient with ongoing dialysis therapy at the time of enrollment.
2. Co-existing significant chronic or severe acute health condition that has the potential to influence how the participant feels or functions as related to fluid overload in NS

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Parent/Guardian of child with Nephrotic Syndrome (NS); This group will help create the Observer Reported Outcome (ObsRO) tool.	Behavioral: Interview; Concept Elicitation Interviews are used to create tools followed by Cognitive Debrief Interviews that are used to review created tools.
Person with Nephrotic Syndrome (NS); This group will help create the Patient Reported Outcome (PRO) tool.	Behavioral: Interview; Concept Elicitation Interviews are used to create tools followed by Cognitive Debrief Interviews that are used to review created tools.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Patient Reported Impact of Fluid Overload on symptoms and functioning	Themes generated through qualitative analysis from semi-structured interviews	Interviews are 60 minutes
Observer Reported Impact of Fluid Overload on symptoms and functioning	Themes generated through qualitative analysis from semi-structured interviews	Interviews are 60 minutes

Collaborators and Investigators

• Sponsor: University of Michigan
• Collaborators: Northwestern University; Food and Drug Administration (FDA)
• Investigators: Principal Investigator: John Peipert, Ph.D., Northwestern University; Principal Investigator: Eloise Salmon, M.D., University of Michigan

Study record dates

Study Major Dates

• Study Start (Actual): April 18, 2022
• Primary Completion (Estimated): April 1, 2026
• Study Completion (Estimated): April 1, 2026

Study Registration Dates

• First Submitted: August 16, 2022
• First Submitted That Met QC Criteria: August 16, 2022
• First Posted (Actual): August 17, 2022

Study Record Updates

• Last Update Posted (Actual): September 13, 2023
• Last Update Submitted That Met QC Criteria: September 11, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Keywords: Child; Edema; Adult; Nephrotic Syndrome; Membranous Nephropathy; FSGS; Minimal Change Disease; Fluid Overload; IgM Nephropathy
• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Urologic Diseases; Disease; Nephritis; Glomerulonephritis; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Syndrome; Kidney Diseases; Edema; Glomerulosclerosis, Focal Segmental; Nephrotic Syndrome; Nephrosis; Glomerulonephritis, Membranous; Nephrosis, Lipoid
• Other Study ID Numbers: HUM00208148; 5UG3FD007308-02 (U.S. FDA Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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