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Novel therapies for resistant focal segmental glomerulosclerosis (FONT) phase II clinical trial: study design.
- Source :
-
BMC nephrology [BMC Nephrol] 2011 Feb 10; Vol. 12, pp. 8. Date of Electronic Publication: 2011 Feb 10. - Publication Year :
- 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.<br />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.<br />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.
- Subjects :
- Adalimumab
Adolescent
Adult
Angiotensin II Type 1 Receptor Blockers adverse effects
Angiotensin II Type 1 Receptor Blockers therapeutic use
Angiotensin-Converting Enzyme Inhibitors adverse effects
Angiotensin-Converting Enzyme Inhibitors therapeutic use
Anti-Inflammatory Agents adverse effects
Antibodies, Monoclonal adverse effects
Antibodies, Monoclonal, Humanized
Anticholesteremic Agents adverse effects
Anticholesteremic Agents therapeutic use
Atorvastatin
Child
Child, Preschool
Drug Resistance
Drug Therapy, Combination
Follow-Up Studies
Galactose adverse effects
Heptanoic Acids adverse effects
Heptanoic Acids therapeutic use
Humans
Infant
Lisinopril adverse effects
Lisinopril therapeutic use
Losartan adverse effects
Losartan therapeutic use
Middle Aged
Pyrroles adverse effects
Pyrroles therapeutic use
Treatment Outcome
Young Adult
Anti-Inflammatory Agents therapeutic use
Antibodies, Monoclonal therapeutic use
Galactose therapeutic use
Glomerulosclerosis, Focal Segmental drug therapy
Subjects
Details
- Language :
- English
- ISSN :
- 1471-2369
- Volume :
- 12
- Database :
- MEDLINE
- Journal :
- BMC nephrology
- Publication Type :
- Academic Journal
- Accession number :
- 21310077
- Full Text :
- https://doi.org/10.1186/1471-2369-12-8