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

BMC Nephrology

Study Protocol

Michael Somers¹  Howard Trachtman²  Suzanne Vento²  Debbie Gipson³  Larysa Wickman³  Virginia Savin⁴  Jennifer Gassman⁵  Maury Pinsk⁶  Melanie Joy⁷  Tom Greene⁸ 

[1] Boston Children's Hospital, Boston, MA, USA;Cohen Children's Medical Center of New York, North Shore Long Island Jewish Health System, New Hyde Park, NY, USA;Department of Pediatrics, University of Michigan, Ann Arbor, MI, USA;Kansas City VA Medical Center, Kansas City, MO, USA;The Cleveland Clinic, Cleveland, OH, USA;University of Alberta, Edmonton, AB, Canada;University of North Carolina, Chapel Hill, NC, USA;University of Utah, Salt Lake City, UT, USA;

关键词: Proteinuria;    Atorvastatin;    Adalimumab;    Losartan;    Rosiglitazone;   

DOI  :  10.1186/1471-2369-12-8

 received in 2011-01-19, accepted in 2011-02-10,  发布年份 2011

来源: Springer

【 摘 要 】

BackgroundThe 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/DesignThe 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.DiscussionThis 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 diseasesTrial RegistrationClinicalTrials.gov, NCT00814255

【 授权许可】

CC BY   
© Trachtman et al; licensee BioMed Central Ltd. 2011