Interventional Study
a clinical trial in which a new drug or device is used to measure a specific outcome.
R3R01: AS-FSGS
R3R01: AS-FSGS
Brief Description
A Phase II, Multi-center, Open-Label Study to Assess the 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.
Trial Physician / Study Coordinator
Oscar Lemus
EmailSite Name
Academic Medical Research Institute
5830 E Whittier Blvd. Los Angeles, 90022, California
Sponsor
R3R Corp
Study Drug
R3R01
Estimated enrollment
20 AS and 30 FSGS patients
Estimated end date
Last patient enrolled July 2024
Interventional Study
a clinical trial in which a new drug or device is used to measure a specific outcome.
R3R01: AS-FSGS
R3R01: AS-FSGS
Brief Description
A Phase II, Multi-center, Open-Label Study to Assess the 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.
Trial is for people with
Alport syndrome and primary focal segmental glomerulosclerosis (FSGS)
Study Goal
The goal of this 12-week open-label study is to determine if R3R01 reduces proteinuria.
What is involved for the Patient?
All patients will receive R3R01 as an oral tablet twice daily for 12 weeks. Blood and urine will be collected, physical exams including vital signs and electrocardiograms will be performed. Patients will be required to attend study visits weekly for the first month, then monthly for the remainder of treatment and 3-month follow-up.
About the drug or intervention
R3R01 is an orally administered small molecule that targets a novel mechanism by increasing levels of functional ABCA1 and cholesterol efflux with the aim of reducing pathologic levels of intracellular (kidney) lipids.