Abstract: SA-OR84
Preliminary Findings from the Phase 2 EPPIK Study of Sparsentan in Pediatric Patients with Selected Proteinuric Glomerular Diseases
Session Information
- Pediatric Nephrology: Clinical and Genetic Studies
November 04, 2023 | Location: Room 105, Pennsylvania Convention Center
Abstract Time: 05:24 PM - 05:33 PM
Category: Pediatric Nephrology
- 1900 Pediatric Nephrology
Authors
- Lieberman, Kenneth V., Joseph M. Sanzari Children's Hospital of the Hackensack Meridian Health, Hackensack, New Jersey, United States
- Trachtman, Howard, University of Michigan, Ann Arbor, Michigan, United States
- Coppo, Rosanna, Fondazione Ricerca Molinette, Regina Margherita Hospital, Turin, Italy
- He, Ping, Travere Therapeutics Inc, San Diego, California, United States
- Mercer, Alex, JAMCO Pharma Consulting, Stockholm, Sweden
- Rheault, Michelle N., University of Minnesota Medical School, Minneapolis, Minnesota, United States
- Saleem, Moin, University of Bristol & Bristol Royal Hospital for Children, Bristol, United Kingdom
- Shafer, Teresa, Travere Therapeutics Inc, San Diego, California, United States
- Komers, Radko, Travere Therapeutics Inc, San Diego, California, United States
Background
Sparsentan (SPAR) is a novel, non-immunosuppressive, single molecule Dual Endothelin Angiotensin Receptor Antagonist (DEARA) approved by the US FDA for the treatment of adults with IgA nephropathy (IgAN) at risk of rapid disease progression and is being investigated for focal segmental glomerulosclerosis (FSGS). The ongoing Phase 2 EPPIK study is examining the safety and long-term antiproteinuric and nephroprotective potential of SPAR in pediatric patients with FSGS, minimal change disease (MCD), IgAN, IgA vasculitis (IgAV), and Alport syndrome (AS). Here we report preliminary findings.
Methods
This open-label, single-arm, multicenter trial is evaluating the safety, efficacy, and pharmacokinetics of SPAR in ~30 patients aged 1-<18 years with FSGS and/or MCD (Population 1) and ~27 aged 2-<18 years with IgAN, IgAV, or AS (Population 2) over 108 weeks with a 4-week safety follow-up. SPAR is administered once daily in a liquid formulation with dose adjusted to body weight. Patients receiving RAASi undergo a 2-week washout prior to study medication start (baseline). Primary endpoints include safety and efficacy (change in urine protein/creatinine ratio [UP/C] from baseline over 108 weeks).
Results
At data cutoff (4/5/23) 23 participants had received ≥1 dose of SPAR. Baseline characteristics are shown in Table. UP/C decreased over 12 weeks by 26% and 52% in Populations 1 and 2 and 35% overall (Figure). SPAR has been safe and generally well-tolerated.
Conclusion
SPAR treatment reduced proteinuria over the initial 12 weeks in pediatric patients with a range of proteinuric glomerular diseases. SPAR was safe and generally well-tolerated, consistent with findings from ongoing FSGS and adult IgAN trials.
Funding
- Commercial Support – Travere Therapeutics, Inc.