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

Sponsor
University of Michigan (Other)
Overall Status
Recruiting
CT.gov ID
NCT05505500
Collaborator
Northwestern University (Other), Food and Drug Administration (FDA) (U.S. Fed)
150
1
47.4
3.2

Study Details

Study Description

Brief Summary

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.

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 Design

Study Type:
Observational
Anticipated Enrollment :
150 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
Preparing a Clinical Outcomes Assessment Set for Nephrotic Syndrome
Actual Study Start Date :
Apr 18, 2022
Anticipated Primary Completion Date :
Apr 1, 2026
Anticipated Study Completion Date :
Apr 1, 2026

Arms and Interventions

Arm 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.

Outcome Measures

Primary Outcome Measures

  1. Patient Reported Impact of Fluid Overload on symptoms and functioning [Interviews are 60 minutes]

    Themes generated through qualitative analysis from semi-structured interviews

  2. Observer Reported Impact of Fluid Overload on symptoms and functioning [Interviews are 60 minutes]

    Themes generated through qualitative analysis from semi-structured interviews

Eligibility Criteria

Criteria

Ages Eligible for Study:
2 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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.

  1. 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
  1. Current or history of NS-associated edema within the past 3 months

  2. Kidney function with most recent estimated Glomerular Filtration Rate (eGFR) > 25 ml/min/1.73m2

  3. 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

Contacts and Locations

Locations

Site City State Country Postal Code
1 University of Michigan Ann Arbor Michigan United States 48109

Sponsors and Collaborators

  • University of Michigan
  • Northwestern University
  • Food and Drug Administration (FDA)

Investigators

  • Principal Investigator: Debbie Gipson, M.D., University of Michigan
  • Principal Investigator: John Peipert, Ph.D., Northwestern University

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Debbie Gipson, MD, Professor of Pediatrics, University of Michigan
ClinicalTrials.gov Identifier:
NCT05505500
Other Study ID Numbers:
  • HUM00208148
  • 5UG3FD007308-02
First Posted:
Aug 17, 2022
Last Update Posted:
Aug 22, 2022
Last Verified:
Aug 1, 2022
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by Debbie Gipson, MD, Professor of Pediatrics, University of Michigan
Additional relevant MeSH terms:

Study Results

No Results Posted as of Aug 22, 2022