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
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
Daniela Stols-Gonsalves
EmailSite Name
AmsterdamUMC
Meibergdreef 9 Amsterdam, 1105AZ
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
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.
Eligibility Criteria
Patient Population
Alport syndrome and primary focal segmental glomerulosclerosis (FSGS)
Age
12 — 26+
History of transplant allowed?
No
History of dialysis allowed?
No
eGFR
45 — > 60
UPCR
1.0 or Less — 3.0 or Above
Permitted medication history
Ace Inhibitors/ARB, Prednisone (Steroids), Abatacept, Acthar, Cellcept (AKA Mycophenolate), Cytoxan (Cyclophosphamide), Prograf (AKA Tacrolimus), Rituximab (AKA Rituxan)
Patient should be
Treatment Resistant, Steroid Dependent
About the Drug
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.
Sponsor
R3R Corp
Estimated End Date
Last patient enrolled July 2024
About the Trial
Study Drug
R3R01
Study Goal
The goal of this 12-week open-label study is to determine if R3R01 reduces proteinuria.
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.