Pilot Studies of Novel Therapies to Treat Resistant Focal Segmental Glomerulosclerosis (FSGS)
Status:
Completed
Trial end date:
2007-10-01
Target enrollment:
Participant gender:
Summary
The current management of primary FSGS is predicated on the assumption that the disease is
caused by an immune-mediated disturbance in glomerular barrier function. Therefore, most
treatment protocols have involved immunosuppressive drugs given singly or in combination.
However, the efficacy of this type of therapy has been disappointing and the long-term
prognosis for renal survival in patients with resistant FSGS is poor. An alternative approach
that targets the fibrosis pathway may represent a novel approach to the treatment of
resistant FSGS. In this R21, the investigators will test the hypothesis that two novel agents
- a tumor necrosis factor-alpha (TNF-α) antagonist and a peroxisome proliferator activator
receptor-gamma (PPARγ) agonist - can be administered safely to patients with resistant FSGS.
In the R21 feasibility/pilot phase, pharmacokinetic studies will be conducted to assess the
impact of proteinuria on the kinetics of the novel drugs in children and adults.
Specific Aim #1: To assess the safety and tolerability of two novel drugs - a TNF-α
antagonist and a PPARγ agonist - in patients with resistant FSGS.
Specific Aim #2: To conduct a pharmacokinetic (PK) assessment of the selected agents to
enable selection of medication regimens for investigation in a randomized Phase II study.