Novel therapies for resistant focal segmental glomerulosclerosis (FONT) phase II clinical trial: study design
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Trachtman, Howard, et al. Novel Therapies for Resistant Focal Segmental Glomerulosclerosis (font) Phase Ii Clinical Trial: Study Design. BioMed Central Ltd, 2011. https://doi.org/10.17615/fpt2-0a66APA
Trachtman, H., Vento, S., Gipson, D., Wickman, L., Gassman, J., Joy, M., Savin, V., Somers, M., Pinsk, M., & Greene, T. (2011). Novel therapies for resistant focal segmental glomerulosclerosis (FONT) phase II clinical trial: study design. BioMed Central Ltd. https://doi.org/10.17615/fpt2-0a66Chicago
Trachtman, Howard, Suzanne Vento, Debbie Gipson, Larysa Wickman, Jennifer Gassman, Melanie Joy, Virginia Savin et al. 2011. Novel Therapies for Resistant Focal Segmental Glomerulosclerosis (font) Phase Ii Clinical Trial: Study Design. BioMed Central Ltd. https://doi.org/10.17615/fpt2-0a66- Creator
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Trachtman, Howard
- Other Affiliation: Cohen Children's Medical Center of New York, North Shore Long Island Jewish Health System, New Hyde Park, NY, USA
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Vento, Suzanne
- Other Affiliation: Cohen Children's Medical Center of New York, North Shore Long Island Jewish Health System, New Hyde Park, NY, USA
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Gipson, Debbie
- Other Affiliation: Department of Pediatrics, University of Michigan, Ann Arbor, MI, USA
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Wickman, Larysa
- Other Affiliation: Department of Pediatrics, University of Michigan, Ann Arbor, MI, USA
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Gassman, Jennifer
- Other Affiliation: The Cleveland Clinic, Cleveland, OH, USA
- Joy, Melanie
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Savin, Virginia
- Other Affiliation: Kansas City VA Medical Center, Kansas City, MO, USA
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Somers, Michael
- Other Affiliation: Boston Children's Hospital, Boston, MA, USA
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Pinsk, Maury
- Other Affiliation: University of Alberta, Edmonton AB, Canada
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Greene, Tom
- Other Affiliation: University of Utah, Salt Lake City, UT, USA
- Abstract
- 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
- Date of publication
- February 10, 2011
- DOI
- Identifier
- Resource type
- Article
- Rights statement
- In Copyright
- Rights holder
- Howard Trachtman et al.; licensee BioMed Central Ltd.
- License
- Journal title
- BMC Nephrology
- Journal volume
- 12
- Journal issue
- 1
- Page start
- 8
- Language
- English
- Is the article or chapter peer-reviewed?
- Yes
- ISSN
- 1471-2369
- Bibliographic citation
- BMC Nephrology. 2011 Feb 10;12(1):8
- Publisher
- BioMed Central Ltd
- Access right
- Open Access
- Date uploaded
- July 15, 2016
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