Clinical trial NCT05505500: Interview Study of Adult and Child Patients and Parents of Children with Swelling Due to Nephrotic Syndrome.
Official title: Preparing a Clinical Outcomes Assessment Set for Nephrotic Syndrome
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Fluid Overload
Glomerulosclerosis, Focal Segmental
Edema
Membranous Nephropathy
Minimal Change Disease
Minimal Change Nephrotic Syndrome
IgM Nephropathy
Nephrotic Syndrome
Glomerular Disease
Nephrotic Syndrome, Minimal Change
Nephrotic Syndrome in Children
Nephrotic Syndrome with Edema (Diagnosis)
FSGS
Keywords: Nephrotic Syndrome
Child
Adult
Fluid Overload
Edema
FSGS
Minimal Change Disease
Membranous Nephropathy
IgM Nephropathy
Lead sponsor: University of Michigan
Sponsor class: OTHER
Collaborators: name: Northwestern University
name: Food and Drug Administration (FDA)
First posted: 2022-08-17
Enrollment: 150
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.

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

Primary outcomes:
measure: Patient Reported Impact of Fluid Overload on symptoms and functioning
timeFrame: Interviews are 60 minutes
measure: Observer Reported Impact of Fluid Overload on symptoms and functioning
timeFrame: Interviews are 60 minutes
Secondary outcomes:
Other outcomes:
Locations:
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
Study officials:
affiliation: University of Michigan
name: Eloise Salmon, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Northwestern University
name: John Peipert, Ph.D.
role: PRINCIPAL_INVESTIGATOR
References: