Exploring experiences of patients and parents dealing with swelling from Nephrotic Syndrome

Preparing a Clinical Outcomes Assessment Set for Nephrotic Syndrome

This study talks to patients and parents about their experiences with swelling from Nephrotic Syndrome to help create better ways to measure how new treatments work.

Quick facts

• Study type: Observational
• Enrollment: 150 (estimated)
• Ages: 2 Years and up
• Sex: All
• Sponsor: University of Michigan (other)
• Locations: 1 site (Ann Arbor, Michigan)
• Trial ID: NCT05505500 on ClinicalTrials.gov

What this trial studies

This observational study involves interviews with adult patients and parents of children suffering from Nephrotic Syndrome, focusing on their experiences with swelling and fluid overload. Conducted by researchers from the University of Michigan and Northwestern University, the study aims to gather qualitative data that will inform the development of a survey for evaluating new medications targeting Nephrotic Syndrome. The interviews will help create core outcome measures that reflect patient and observer perspectives on edema associated with the condition.

Who should consider this trial

Good fit: Ideal candidates include parents of children aged 2-11 with a documented diagnosis of idiopathic Nephrotic Syndrome and current edema.

Not a fit: Patients who are on dialysis or do not have a current diagnosis of Nephrotic Syndrome will not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved assessment tools for evaluating treatments for Nephrotic Syndrome, ultimately enhancing patient care.

How similar studies have performed: While similar qualitative studies have been conducted, this specific approach to developing outcome measures for Nephrotic Syndrome is relatively novel.

Eligibility criteria

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 NS-associated edema
4. The child must have native kidney function
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
4. Current NS-associated edema
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
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

Where this trial is running

Ann Arbor, Michigan

• University of Michigan — Ann Arbor, Michigan, United States (RECRUITING)

Study contacts

• Principal investigator: Eloise Salmon, M.D. — University of Michigan
• Study coordinator: Ashley Rahimi

Conditions: Fluid Overload, Glomerulosclerosis, Focal Segmental, Edema, Membranous Nephropathy, Minimal Change Disease, Minimal Change Nephrotic Syndrome, IgM Nephropathy, Nephrotic Syndrome