TRPC6 Inhibitor for Primary Focal Segmental Glomerulosclerosis (pFSGS)

Study Overview

A Phase 3, multicenter, randomized, double-blind, placebo-controlled study evaluating an oral TRPC6 inhibitor in adults and adolescents with primary FSGS or genetic FSGS related to TRPC6 gene variants. The study aims to demonstrate reduction in proteinuria and preservation of kidney function. TRPC6 inhibition targets the podocyte directly, addressing the underlying mechanism of glomerular injury.

Inclusion Criteria

• ✓Adults and adolescents ≥12 years old
• ✓Weight ≥40 kg and BMI ≤40 kg/m²
• ✓Biopsy-confirmed pFSGS or genetic FSGS with TRPC6 gain-of-function gene mutation
• ✓UPCR ≥1500 mg/g at screening
• ✓eGFR ≥25 mL/min/1.73 m²

Exclusion Criteria

• ✗Known monogenic or syndromic causes of FSGS (except TRPC6 mutations)
• ✗Clinical or histologic evidence of secondary FSGS
• ✗FSGS of undetermined cause (FSGS-UC)
• ✗History of organ transplantation or planned transplant during the trial
• ✗Use of IV immunosuppressive agents (rituximab, cyclophosphamide) in last 6 months