%0 Journal Article
%T Novel therapies for resistant focal segmental glomerulosclerosis (FONT) phase II clinical trial: study design
%A Howard Trachtman
%A Suzanne Vento
%A Debbie Gipson
%A Larysa Wickman
%A Jennifer Gassman
%A Melanie Joy
%A Virginia Savin
%A Michael Somers
%A Maury Pinsk
%A Tom Greene
%J BMC Nephrology
%D 2011
%I BioMed Central
%R 10.1186/1471-2369-12-8
%X The Novel Therapies for Resistant Focal Segmental Glomerulosclerosis (FONT) project is a multicenter Phase I/II RCT designed to investigate the potential efficacy of novel therapies for resistant FSGS. Adalimumab and galactose will be evaluated against conservative therapy consisting of the combination of lisinopril, losartan and atorvastatin. The sample size is defined to assure that if one of the treatments has a superior response rate compared to that of the other treatments, it will be selected with high probability for further evaluation. Comparison of primary and secondary endpoints in each study arm will enable a choice to be made of which treatments are worthy of further study in future Phase III RCT.This report highlights the key features of the FONT II RCT including the two-step outcome analysis that will expedite achievement of the study objectives. The proposed phase II study design will help to identify promising agents for further testing while excluding ineffective agents. This staged approach can help to prevent large expenditures on unworthy therapeutic agents in the management of serious but rare kidney diseasesClinicalTrials.gov, NCT00814255The goal of therapy in proteinuric diseases such as primary FSGS is complete remission of proteinuria and preservation of renal function. However, this is rarely achieved in patients with FSGS that is resistant to standard treatment [1,2]. When corticosteroids and immunosuppressive therapy fail to induce remission in patients with primary FSGS, a number of agents are used as renoprotective therapy to delay progression of chronic kidney disease (CKD) to end stage kidney disease (ESKD). Angiotensin converting enzyme inhibitor (ACEi) and angiotensin II receptor blocker (ARB) are two such therapies that reduce proteinuria when used alone, with an additive effect when prescribed in combination [3-12]. Prescription of 3-hydroxy-3-methyl-glutaryl coenzyme A (HMG-CoA) reductase inhibitors in doses designed to treat dys
%U http://www.biomedcentral.com/1471-2369/12/8