Clinical Trial: Randomized, Double-Blind, Safety and Efficacy Study of RE-021 (Sparsentan) in Focal Segmental Glomerulosclerosis

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Efficacy and Safety of RE-021, a Dual Endothelin Receptor and Angiotensin Receptor Blocker, in Patients With Focal Segmental Glomerulosclerosis (FSGS): a Randomized, Doubl

Brief Summary: This study will investigate whether RE-021 (Sparsentan), a selective dual-acting receptor antagonist with affinity for endothelin (A type) and angiotensin II receptors (Type 1), is safe and effective in treating patients with focal segmental glomerulosclerosis (FSGS).

Detailed Summary: Focal segmental glomerulosclerosis (FSGS) is a rare glomerular disorder which results in frank proteinuria and progression to end-stage kidney disease (ESKD) over 5-10 years. Proteinuria reduction is widely regarded to be beneficial, and is considered the primary goal of treatment in FSGS and slowing its progressive course (D'Agati, et. al, 2011). Patients are currently treated with steroids, calcineurin inhibitors, angiotensin receptor blockers (ARB) and angiotensin converting inhibitors (ACE) to lower proteinuria (Cameron, 2003). Despite these therapies, many patients have nephrotic range proteinuria and new therapeutic agents are needed (Kiffel, et. al, 2011). Endothelin receptor antagonists (ERA) have been shown to lower proteinuria in clinical trials of diabetic nephropathy (Kohan, et. al, 2011) (Mann, et. al 2010) and have been speculated to be effective in FSGS (Barton, 2010).
Sponsor: Retrophin, Inc.

Current Primary Outcome: Evaluate change in urine protein/creatinine (Up/C). [ Time Frame: 8 weeks ]

Primary efficacy objective is to determine the change in Up/C in FSGS patients receiving RE-021 (Sparsentan) over a range of dose levels compared to treatment with irbesartan as active control.


Original Primary Outcome: Change in albumin excretion rate (AER) from baseline achieved with fixed doses (100 mg, 200 mg, 400 mg, and 800 mg) of RE-021 to ARB therapy after 6 weeks of treatment. [ Time Frame: 6 weeks ]

Current Secondary Outcome:

Original Secondary Outcome: Determine the PK characteristics of RE-021 in patients with FSGS and proteinuria [ Time Frame: 6 weeks ]

Information By: Retrophin, Inc.

Dates:
Date Received: June 4, 2012
Date Started: December 2013
Date Completion: January 2019
Last Updated: October 18, 2016
Last Verified: October 2016