Abstract: TH-PO605
Sparsentan (SPAR) in Pediatric Patients with Rare Proteinuric Kidney Disease: Preliminary Findings from the EPPIK Study
Session Information
- Membranous Nephropathy, FSGS, and Minimal Change Disease
October 24, 2024 | Location: Exhibit Hall, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Glomerular Diseases
- 1402 Glomerular Diseases: Clinical, Outcomes, and Therapeutics
Authors
- Trachtman, Howard, University of Michigan, Ann Arbor, Michigan, United States
- Coppo, Rosanna, Fondazione Ricerca Molinette, Regina Margherita Hospital, Turin, Italy
- Masthan Ahmed, Nuhira Ahmed, Travere Therapeutics, Inc., San Diego, California, United States
- Lieberman, Kenneth V., Joseph M. Sanzari Children's Hospital of the Hackensack Meridian Health, Hackensack, New Jersey, United States
- Mercer, Alex, JAMCO Pharma Consulting, Stockholm, Sweden
- Rheault, Michelle N., University of Minnesota Medical School, Minneapolis, Minnesota, United States
- Saleem, Moin A., University of Bristol & Bristol Royal Hospital for Children, Bristol, United Kingdom
- Komers, Radko, Travere Therapeutics, Inc., San Diego, California, United States
Background
SPAR is a nonimmunosuppressive, dual endothelin angiotensin receptor antagonist (DEARA) approved (US and EU) for adults with IgA nephropathy (IgAN) at risk of rapid disease progression and is being investigated for focal segmental glomerulosclerosis (FSGS). The phase 2 EPPIK study (NCT05003986) evaluates safety and long-term antiproteinuric and nephroprotective effects of SPAR in pediatric patients (pts) with FSGS, minimal change disease (MCD), IgAN, IgA vasculitis (IgAV), and Alport syndrome (AS). We report preliminary findings per individual diagnoses.
Methods
This open-label, single-arm trial examines SPAR safety, efficacy, and pharmacokinetics in ≈30 pts 1-<18 y with FSGS and/or MCD histological patterns (population 1) and ≈27 pts 2-<18 y with IgAN, IgAV, or AS (population 2) over 108 wk and 4-wk safety follow-up. SPAR is given once daily (liquid form, dose adjusted for weight); pts on a renin-angiotensin system inhibitor undergo 2-wk washout before study medication (baseline [BL]). Primary endpoints include safety and efficacy (change in urine protein/creatinine ratio [UP/C]).
Results
At data cutoff (2/15/2024), 34 pts received ≥1 SPAR dose (BL characteristics in Table). Over 12 wk, proteinuria (UP/C) decreased by 51% and 27% in pts with MCD and FSGS and 54% and 61% in pts with IgAN/IgAV and AS (Figure). SPAR was safe and generally well tolerated.
Conclusion
SPAR is well tolerated in pediatric pts with various glomerular diseases, consistent with adult trials in FSGS and IgAN. Proteinuria decreased in each indication, even in those not previously studied, with about 50% overall reduction over 12 wk of treatment.
Funding
- Commercial Support – Travere Therapeutics, Inc.