A Study of RG-012 in Subjects With Alport Syndrome

This is a Phase 1, multi-center study of the safety, pharmacodynamics, and pharmacokinetics of RG-012 administered to subjects with Alport syndrome. During this open-label study, all eligible subjects will receive RG-012. The study consists of two parts (Part A and Part B). During Part A, half of the participants will receive a single dose of RG-012 and half will receive 4 doses of RG-012 (one dose every other week for 6 weeks). All subjects will undergo two renal biopsies, one before and one after receiving RG-012, to assess the effect of RG-012 on the kidney. After completing Part A, subjects will be able to enter Part B of the study. During Part B, all subjects will receive RG-012 every other week for 48 weeks.

Inclusion criteria

• Males or females, ages 18 to 65 years
• Confirmed diagnosis of Alport syndrome
• eGFR between 40 and 90 mL/min/1.73m2
• Proteinuria of at least 300 mg protein/g creatinine
• For subjects taking an ACE inhibitor or an ARB, the dosing regimen should be stable for at least 30 days prior to screening
• Willing to comply with contraception requirements

Exclusion criteria

• Causes of chronic kidney disease aside from Alport syndrome (such as diabetic nephropathy, hypertensive nephropathy, lupus nephritis, or IgA nephropathy)
• End stage renal disease (ESRD) as evidenced by ongoing dialysis therapy or history of renal transportation
• Any other condition that may pose a risk to the subject's safety and well-being
• Female subjects who are pregnant or lactating