Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases — EPPIK  

Focal Segmental Glomerulosclerosis Clinical Trial

Official title: 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

Clinical Trial 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.

Clinical Trial 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 ;

Related Conditions & MeSH terms

• Alport Syndrome
• Focal Segmental Glomerulosclerosis
• Glomerulonephritis, IGA
• Glomerulosclerosis, Focal Segmental
• IgA Vasculitis
• Immunoglobulin A Nephropathy
• Minimal Change Disease
• Nephritis, Hereditary
• Nephrosis, Lipoid
• Vasculitis

• NCT number: NCT05003986
• Study type: Interventional
• Source: Travere Therapeutics, Inc.
• Contact: Travere Call Center
• Phone: 1-877-659-5518
• Email: medinfo@travere.com
• Status: Recruiting
• Phase: Phase 2
• Start date: August 12, 2021
• Completion date: June 1, 2025