927 Sparsentan for treatment of pediatric patients with selected proteinuric glomerular diseases: design of the phase 2 EPPIK study. (17th August 2022)

• Record Type: Journal Article
• Authors: Trachtman, Howard; Saleem, Moin; Coppo, Rosanna; Rheault, Michelle; He, Ping; Komers, Radko
• Is Part Of: Archives of disease in childhood. Volume 107(2022)Supplement 2
• Page Start: A96
• Page End: A97
• Publication Date: 2022-08-17
• Subjects: Children -- Diseases -- Periodicals; Infants -- Diseases -- Periodicals; 618.920005
• DOI: 10.1136/archdischild-2022-rcpch.157
• Languages: English
• ISSNs: 0003-9888

Abstract

Abstract : Aims: Sparsentan is a novel Dual Endothelin Angiotensin Receptor Antagonist (DEARA) being investigated for focal segmental glomerulosclerosis (FSGS) and IgA nephropathy (IgAN). It is a dual acting, highly selective antagonist of both the endothelin A receptor (ETA R) and the angiotensin II subtype 1 receptor (AT1 R). The Phase 2 EPPIK study will examine the long-term antiproteinuric and nephroprotective potential and safety of sparsentan in pediatric patients with FSGS, minimal change disease (MCD), IgAN, IgA vasculitis (IgAV), and Alport syndrome (AS). Methods: The global, open-label, single-arm, multicenter study will evaluate the safety, efficacy, and pharmacokinetics (PK) of sparsentan in ~57 patients (aged ≥1 to <18 years), including ~30 with FSGS and/or MCD (population 1) and ~27 with IgAN, IgAV, or AS (population 2) over 108 weeks ( figure 1 ). See table 1 for inclusion/exclusion criteria. Sparsentan will be administered in a novel liquid formulation at a dose adjusted to body weight. Results: Primary endpoints include safety (incidence of treatment-emergent adverse events) and efficacy (change in urine protein/creatinine ratio [UP/C] from baseline over 108 weeks) with sparsentan treatment. Secondary endpoints include PK outcomes, change from baseline over 108 weeks in albumin/creatinine ratio and estimated glomerular filtration rate (eGFR), and the proportion of patients with FSGS/MCD who achieve partial remission (defined as UP/C ≤1.5 g/g and >40% reduction in UP/C). Conclusion: This Phase 2 study will evaluate the long-term safety, antiproteinuric, and nephroprotective effects of sparsentan in pediatric patients with FSGS, MCD, IgAN, IgAV, and AS.