NCT05267262

Study to Evaluate R3R01 in Patients With Alport Syndrome and Patients With Focal Segmental Glomerulosclerosis

A Phase II, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in AS Patients With Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients With Primary Steroid-Resistant FSGC

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 43 (actual)
• Sponsor: River 3 Renal Corp. · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 2, 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

Detailed description

R3R01 is investigational small molecule designed to decrease fat levels in certain cells in the kidney and therefore may improve kidney function and reduce damage in the kidney. This is a single arm open-label study enrolling patients in three cohorts. Cohort 1 will include 5 adult (≥18 y/o) patients from Cohorts 2 and 3 (including at least one patient from Cohort 2 and at least one patient from Cohort 3). Cohort 2 will include approximately 20 male and female patients from 12 years and older with X-linked Alport Syndrome (AS), and male and female patients with autosomal inherited AS. Cohort 3 will include approximately 30 male and female patients from age 12 to 75 years with a biopsy proven diagnosis who present with primary steroid-resistant focal segmental glomerulosclerosis (FSGS) with proteinuria. All eligible patients will be enrolled to receive R3R01 over a treatment period of 12 weeks with a primary efficacy outcome as the percentage change in proteinuria from baseline to the end of treatment (Day 84) in each cohort as a whole

Conditions

• Alport Syndrome
• Focal Segmental Glomerulosclerosis

Interventions

Type	Name	Description
DRUG	R3R01	R3R01 administered orally for 12 weeks

Timeline

• Start date: 2022-06-15
• Primary completion: 2025-05-21
• Completion: 2025-08-19
• First posted: 2022-03-04
• Last updated: 2025-09-29

Locations

22 sites across 6 countries: United States, Belgium, France, Germany, Netherlands, United Kingdom

Regulatory

• FDA-regulated drug study