NCT05003986

Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases

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

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 67 (estimated)
• Sponsor: Travere Therapeutics, Inc. · Industry
• Sex: All
• Age: 1 Year – 17 Years
• Healthy volunteers: Not accepted

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.

Detailed 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 biopsy-confirmed IgAN The study will evaluate long-term safety, tolerability, and efficacy with pharmacokinetic (PK) evaluations at Day 1 (Baseline), Day 2 (Visit 4), and Week 12 (Visit 9) in Population 1 and Population 2. In Population 3, PK values will be evaluated at Day 1 (Baseline) and at Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, and 96. For Population 1 and Population 2, subjects will be enrolled in 3 cohorts based on age ranges. For Population 3, subjects will be enrolled in one cohort. Study Enrollment: * Population 1: FSGS and/or MCD (30 subjects total) 1. Cohort 1 (6 subjects): ≥8 years to \<18 years 2. Cohort 2 (18 subjects): ≥3 years to \<8 years 3. Cohort 3 (6 subjects): ≥1 year to \<3 years * Population 2: IgAN, IgAV, or AS (27 subjects total) 1. Cohort 1 (9 subjects): ≥8 years to \<18 years 2. Cohort 2 (12 subjects): ≥5 years to \<8 years 3. Cohort 3 (6 subjects): ≥2 years to \<5 years * Population 3: IgAN (10 subjects total) 1. 10 subjects: ≥8 years to \<18 years

Conditions

• Focal Segmental Glomerulosclerosis
• Minimal Change Disease
• Immunoglobulin A Nephropathy
• IgA Vasculitis
• Alport Syndrome

Interventions

Type	Name	Description
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)

Timeline

• Start date: 2021-08-12
• Primary completion: 2027-03-12
• Completion: 2027-04-12
• First posted: 2021-08-13
• Last updated: 2025-12-23

Locations

47 sites across 8 countries: United States, Germany, Italy, Netherlands, Poland, Spain, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study