A Phase 2, Open-Label, Single-Arm, Cohort Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Sparsentan Treatment in Pediatric Subjects With Selected Proteinuric Glomerular Diseases

Travere Therapeutics, Inc.

Summary

To evaluate the safety, efficacy and tolerability of sparsentan oral suspension and tablets, and assess changes in proteinuria after once-daily dosing over 108 weeks.

Description

This is a multicenter, open-label, 112-week study of sparsentan in approximately 67 pediatric subjects aged ≥1 year to \<18 years with selected proteinuric glomerular diseases, divided into 3 populations, defined as follows: * Population 1: Subjects with selected proteinuric glomerular diseases associated with Focal Segmental Glomerulosclerosis (FSGS) and Minimal Change Disease (MCD) histological patterns * Population 2: Subjects with kidney biopsy-confirmed immunoglobulin A nephropathy (IgAN), immunoglobulin A vasculitis (IgAV), or Alport syndrome (AS) * Population 3: Subjects with kidney bi…

Eligibility

Age range: 1–17 years
Sex: All
Healthy volunteers: No

Detailed criteria

Inclusion Criteria for All Subjects (All Three Populations): A subject must meet all of the following criteria to be eligible for participation in this study: * The subject or parent/legal guardian (as appropriate) is willing and able to provide signed informed consent/assent, and where required, the subject is willing to provide assent before any screening procedures per local requirements. * The subject has an estimated glomerular filtration rate (eGFR) ≥30 mL/min/1.73 m2 at screening. * The subject has a mean seated blood pressure between the 5th and 95th percentile for sex and height. I…

Interventions

Drug
Sparsentan
Population 1: 800 mg Sparsentan (oral suspension)
Drug
Sparsentan
Population 2: 400 mg Sparsentan (oral suspension)
Drug
Sparsentan
Population 3: 400 mg Sparsentan (tablets)

Locations (47)

Cedars-Sinai Medical Center
Los Angeles, California
Nemours Children's Hospital
Wilmington, Delaware
University of Miami, Leonard M. Miller School of Medicine
Miami, Florida
Nicklaus Children's Hospital
Miami, Florida
University of Iowa, Stead Family Children's Hospital
Iowa City, Iowa
Floating Hospital for Children at Tufts Medical Center
Boston, Massachusetts