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
EPPIK
What is the goal of the study?
Currently, there are no approved treatment options for pediatric subjects with proteinuric kidney conditions. The current study (Protocol RTRX-RE021-201) is a Phase 2, single-arm, safety, efficacy, and pharmacokinetic (PK) trial in children ≥1 to <18 years treated for up to 108 weeks with sparsentan. this study is designed to provide safety descriptive efficacy and pk data in a broad proteinuric pediatric population in need of therapeutic intervention and as a bridge to sparsentan efficacy using a partial extrapolation approach with matching exposure as established in the ongoing phase 2 and 3 studies. this is a multicenter open-label 108-week study of sparsentan in approximately 57 pediatric subjects aged 1 year to><18 years with selected proteinuric glomerular diseases. primary objectives: evaluate the safety and tolerability of sparsentan oral suspension assess changes in proteinuria after once-daily dosing of sparsentan oral suspension over the 108-week treatment period.>
Who can participate in the study?
Everyone