A Phase II, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in Alport Syndrome Patients with Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients with Primary Steroid-Resistant Focal Segmental Glomerulosclerosis

Alport Syndrome (AS) and Primary Steroid-Resistent Focal Segmental Glomerulosclerosis (FSGS)

Safety and tolerability endpoints will be assessed by occurrence of adverse events (AE), changes in physical examinations, vital signs, ECGs and clinical laboratory parameters., For AS patients: The change from baseline in UPCR at 12 weeks for all patients with AS., For FSGS patients: The change from baseline in UPCR at 12 weeks for all patients with FSGS.

For all patients: • Change in quality-of-life assessment from baseline to end of treatment and to the end of the follow-up period by cohort., Population pharmacokinetic analysis to derive primary PK parameters (absorption rate (ka), apparent Clearance (Cl/F), apparent Volume of distriburion (V/F)), and secondary PK parameters (maximum concentration (Cmax), time of Cmax (tmax), minimum plasma concentrations (Cmin), area under the concentration time curve, elimination half-life (t1/2)., For AS patients: • Number of AS patients with complete response, a partial response for proteinuria (categorical assessment) at weeks 4, 8, 12, 16, 20 and 24., For FSGS patients: • Number of FSGS patients with complete remission, a partial remission or a modified partial remission for proteinuria (categorical assessment) at weeks 4, 8, 12, 16, 20 and 24.

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