Primary Focal Segmental Glomerulosclerosis is a serious renal disease, accounting for nearly 10-15% of all pediatric and adult patients requiring chronic dialysis or transplantation. In response to this urgent situation, a national multicenter NIH-funded randomized clinical trial is underway to compare the efficacy of cyclosporine, the current standard of care, with a combination of mycophenolate mofetil and oral dexamethasone pulses in patients with steroid-resistant FSGS. It is anticipated that a significant number of patients will be ineligible for this trial because of prior treatment with one of the test therapies or will fail to respond to the experimental treatment. There is a pressing need to develop novel therapies to treat these refractory patients. This proposal will evaluate the safety and efficacy of two novel agents that may have the capacity to reduce renal fibrosis and slow the rate of deterioration of disease in patients with resistant FSGS.  We are currently in the initial phase of the Phased Innovation Award.  During this initial stage, the R21 Phase, the safety, tolerance, and pharmacokinetic profile of two novel therapies – a TNF-α antagonist ( Adalimumab,Humira) and a PPAR γ agonist (Rosiglitazone, Avandia) -- will be tested.

The trial is currently enrolling patients between the ages of 2 and 42 with primary FSGS, estimated GFR of ≥ 40ml/min/1.73m2 and not transplanted. Further information about the trial or a participating site near you may be obtained by contacting one of the two core coordinating centers for the trial. 

Howard Trachtman, MD                                         Debbie Gipson MD, MSPH

Schneider Children Hospital (SCH)                         University North Carolina (UNC)

Clinical Coordinating Center                                    Data Coordinating Center

Division of Nephrology                                            Division of Nephrology

269-01 76th Avenue                                               7012 Burnett-Womack, CB 7155

New Hyde Park NY 11040                                    Chapel Hill, NC 27599-7155

TEL: 718-470-3491                                                TEL: 919-966-2561

FAX: 718-470-0887                                               FAX: 919-966-4104

E-mail: trachtma@lij.edu                                          TOLL FREE NUMBER: 866-462-9371

Coordinator:  Suzzane Vento, RN                            E-mail: Debbie_Gipson@med.unc.edu

E-mail: svento@lij.edu                                             Coordinator: Amber Thompson, RN

                                                                                                          E-mail: amber_thompson@med.unc.edu