Clinical trial NCT05200871: Humanistic Burden of (FSGS) Focal Segmental Glomerulosclerosis and IgAN (Immunoglobulin A Nephropathy)
Official title: Humanistic Burden of Rare Kidney Diseases: Understanding the Impact of FSGS and IgAN on Patients and Caregivers Study (HONUS) - A Multi-National, Cross-Sectional Survey Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Focal Segmental Glomerulosclerosis
Immunoglobulin A Nephropathy
Keywords: Observational
Cross-Sectional
Survey
Health-related quality of life (HRQoL)
Patients
Care-Partners
Humanistic
Burden
Lead sponsor: Travere Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2022-01-21
Enrollment: 487
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population will include adult patients with FSGS or IgAN and their adult care-partners, as well as parents/care-partners of children/adolescents with FSGS or IgAN, who meet specified inclusion/exclusion criteria

Brief summary: The aim of this observational study is to assess humanistic burden among adults and children/adolescents with FSGS and IgAN as well as the burden and impact for patient care-partners in six countries (United States \[US\], United Kingdom \[UK\], France, Germany, Italy and Spain).


Eligibility criteria: Inclusion Criteria:

* Adult patients and their adult care-partners: At least 18 years old; Either have a physician-provided diagnosis of FSGS or IgAN (with renal biopsy confirmation of the diagnosis) or being a care-partner for someone with a physician-provided diagnosis of FSGS or IgAN (with renal biopsy confirmation of the diagnosis);
* Able to provide informed consent;
* Located in the United States (US), United Kingdom (UK), Germany, France, Spain or Italy.

  \*Care-partners (paired with adult patients) (defined as the individual \[e.g., spouse, parent, sibling, relative, or friend\] providing direct disease-related support to the adult patient.

  \*\*All patient groups including chronic kidney disease (CKD) stage 1-5, with or without dialysis, and with or without kidney transplant will be included.
* Care-partners/parents of children/adolescents: At least 18 years old;
* Being a care-partner of children/adolescents with physician-provided diagnosis of FSGS or IgAN (with renal biopsy or genetic confirmation of the diagnosis);
* Able to provide informed consent; Located in the US, UK, Germany, France, Spain or Italy.

  * Care-partners will be defined as family members who provide disease-related support and unpaid care to child/adolescent patients

Exclusion Criteria:

* Patient has FSGS or IgAN secondary to another condition;
* Patient has a history of malignancy other than adequately treated basal cell or squamous cell skin cancer;
* Patient has a co-existing glomerular disease (e.g., membranous nephropathy, lupus nephritis);
* Patient is currently participating in a kidney disease clinical trial, and potentially receiving active treatment as part of the trial.

Primary outcomes:
measure: Demographics
timeFrame: Day 1, day of enrollment
measure: Disease history.
timeFrame: Day 1, day of enrollment
measure: Adult patient health-related quality of life.
timeFrame: Day 1, day of enrollment
measure: Pediatric patient health-related quality of life (reported by parent/care-partner).
timeFrame: Day 1, day of enrollment
measure: Adult patient care-partner and pediatric patient parent/care-partner health-related quality of life.
timeFrame: Day 1, day of enrollment
measure: Adult patient, adult patient care-partner and pediatric patient parent/care-partner anxiety.
timeFrame: Day 1, day of enrollment
measure: Adult patient, adult patient care-partner and pediatric patient parent/care-partner depression.
timeFrame: Day 1, day of enrollment
measure: Adult patient cognition.
timeFrame: Day 1, day of enrollment
measure: Adult patient symptoms.
timeFrame: Day 1, day of enrollment
measure: Pediatric patient symptoms (reported by parent/care-partner).
timeFrame: Day 1, day of enrollment
measure: Adult patient, pediatric patient (reported by parent/care-partner), adult patient care-partner and pediatric patient parent/care-partner fear and anxiety for the future.
timeFrame: Day 1, day of enrollment
measure: Adult patient productivity impairment.
timeFrame: Day 1, day of enrollment
measure: Adult patient care-partner and pediatric patient parent/care-partner productivity impairment.
timeFrame: Day 1, day of enrollment
measure: Adult patient, adult patient care-partner and pediatric patient parent/care-partner impact of disease.
timeFrame: Day 1, day of enrollment
measure: Pediatric/adolescent patient impact of disease (reported by parent/care-partner).
timeFrame: Day 1, day of enrollment
Secondary outcomes:
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: Travere Investigational Site
state: California
city: Belmar
country: United States
facility: Travere Investigational Site
state: New Jersey
city: Chapel Hill
country: United States
facility: Travere Investigational Site
state: North Carolina
city: King Of Prussia
country: United States
facility: Travere Investigational Site
state: Pennsylvania
city: Villingen-Schwenningen
country: Germany
facility: Travere Investigational Site
city: Madrid
country: Spain
facility: Travere Investigational Site
Study officials:
affiliation: Travere Therapeutics
name: Leah Conley, MBA
role: STUDY_DIRECTOR
References: