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Open Access Highly Accessed Study protocol

Novel therapies for resistant focal segmental glomerulosclerosis (FONT) phase II clinical trial: study design

Howard Trachtman1*, Suzanne Vento1, Debbie Gipson2, Larysa Wickman2, Jennifer Gassman3, Melanie Joy4, Virginia Savin5, Michael Somers6, Maury Pinsk7 and Tom Greene8

Author Affiliations

1 Cohen Children's Medical Center of New York, North Shore Long Island Jewish Health System, New Hyde Park, NY, USA

2 Department of Pediatrics, University of Michigan, Ann Arbor, MI, USA

3 The Cleveland Clinic, Cleveland, OH, USA

4 University of North Carolina, Chapel Hill, NC, USA

5 Kansas City VA Medical Center, Kansas City, MO, USA

6 Boston Children's Hospital, Boston, MA, USA

7 University of Alberta, Edmonton AB, Canada

8 University of Utah, Salt Lake City, UT, USA

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BMC Nephrology 2011, 12:8  doi:10.1186/1471-2369-12-8

Published: 10 February 2011

Abstract

Background

The lack of adequate randomized clinical trials (RCT) has hindered identification of new therapies that are safe and effective for patients with primary focal segmental glomerulosclerosis (FSGS), especially in patients who fail to respond to corticosteroids and immunosuppressive therapies. Recent basic science advances have led to development of alternative treatments that specifically target aberrant pathways of fibrosis which are relevant to disease progression in FSGS. There is a need for a flexible Phase II study design which will test such novel antifibrotic strategies in order to identify agents suitable for phase III testing.

Methods/Design

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.

Discussion

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 diseases

Trial Registration

ClinicalTrials.gov, NCT00814255