Clinical Trial: Focal Segmental Glomerulosclerosis Clinical Trial (FSGS-CT)
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Focal Segmental Glomerulosclerosis Clinical Trial
Brief Summary: The FSGS Clinical Trial is a multi-center, prospective, controlled, open label randomized trial designed to determine if treatment with mycophenolate mofetil (MMF) in conjunction with pulse steroids is superior to treatment with Cyclosporine-A (CSA) in inducing remission from proteinuria over 12 months.
Detailed Summary:
Background/Rationale. Primary FSGS is a leading cause of end stage renal disease in both children and adults, with complete loss of kidney function in 50% of patients over 10 years. Evidence-based treatment guidelines for FSGS have not been developed because of the lack of controlled studies and the small number of patients included in most reports. Over the past decade, a number of studies have reported therapeutic efficacy for treatment with Cyclosporine-A (CSA) in patients with nephrotic syndrome including patients with steroid resistant FSGS. There have been two controlled trials of treatment with CSA in steroid resistant FSGS, one in children and one in adult patients. Consequently, CSA is the only medication that has been documented to be efficacious in a controlled trial in both children and adults with steroid resistant FSGS. The experience with mycophenolate mofetil (MMF) in the treatment of patients with steroid resistant FSGS has been limited to uncontrolled trials in adult patients and children.
Patient Population. The patient population consists of children and adults between the ages of 2 and 40 years with steroid resistant FSGS.
Study Design. The experimental design is a multi-center, prospective, controlled, open label randomized trial comparing two treatment regimens, CSA vs. MMF/Pulse Steroids. The treatment regimens in both arms also include angiotensin converting enzyme inhibitor (ACEI) therapy and alternate day low dose prednisone. The CSA and MMF/Pulse steroid treatment regimens will be implemented over the first 26 weeks after randomization and continued to 52 weeks if a response in proteinuria occurs. The ACE inhibitor component continues for an additional 26 weeks after withdrawal of initial therapies.
Logistical Structure. Participants are recruite
Sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Current Primary Outcome: The primary outcome is a 6-level ordinal variable defined based on the achievement of remission from proteinuria during the first 52 weeks after randomization. [ Time Frame: first 52 weeks after randomization. ]
Original Primary Outcome: The primary outcome is a 6-level ordinal variable defined based on the achievement of remission from proteinuria during the first 52 weeks after randomization.
Current Secondary Outcome: The main secondary outcome is a 5-level ordinal variable defined based on the persistence of remissions after immunosuppressive agents are withdrawn. [ Time Frame: based on the participant's level of proteinuria during the period from week 52 through week 78 following withdrawal of CSA or MMF/Pulse steroids ]
Original Secondary Outcome: To main secondary outcome is a 5-level ordinal variable defined based on the persistence of remissions after immunosuppressive agents are withdrawn.
Information By: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Dates:
Date Received: August 24, 2005
Date Started: November 2004
Date Completion:
Last Updated: May 21, 2012
Last Verified: May 2012
