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Title 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
Therapeutic Area Alports Syndrome, Chronic Kidney Disease, Focal Semental Glomeroulsclerosis, Glomerular Disease, Kidney Diseases, Proteinuria
Principal Investigator Lesley Inker, MD
Min Age 12 Years
Gender Any
Contact Teya Lovell
More Information


The purpose of this Phase II trial is to see if R3R01 is safe in patients with Alport Syndrome and Focal Segmental Glomerulosclerosis. This will be done by evaluating how efficient R3R01 is in decreasing proteinuria levels. The study will consists of a screening period, a treatment period, and a follow-up period. If you qualify, you will receive the study drug R3R01 for 12 weeks. We will then follow up with you after for an additional 12 weeks.

Study Details

Inclusion Criteria

1. Age 12 years or older

2. Diagnosis of Alport Syndrome (confirmed by genetic testing and/or kidney biopsy) / Primary FSGS (confirmed by renal biopsy) 

Exclusion Criteria

1. Uncontrolled diabetes mellitus or hypertension

2. History of organ transplant or possibility of transplant in the six months after screening

If you aren't sure if you qualify, please call us and we will help determine your eligibility. 

Study Requirements

If you qualify, you will receive R3R01 for 12 weeks. We will then follow up with you for an additional 12 weeks following completion of the study drug treatment.