Novel Therapies for Resistant FSGS (FONTII): Phase II Clinical Trial

Inclusion Criteria: * Primary FSGS confirmed by renal biopsy OR documentation of a genetic mutation in a podocyte protein associated with the disease * Failure to respond to prior therapy at least one of the following immunosuppressive medications -- cyclosporine, tacrolimus, mycophenolate mofetil, sirolimus - or other agents prescribed to lower proteinuria * Age 1-65 years at onset of proteinuria * Age 1-65 years at time of randomization * Estimated GFR ≥40 mL/min/1.73 m2 using Schwartz (age \<18 yr) or Cockroft-Gault (age \<18 yr) formula at screening and ≥30 mL/min/1.73 m2 at the end of the Run-In Period and at the time of randomization * Up/c \> 1.0 g/g creatinine on first morning void * Steroid resistance defined as failure to achieve sustained Up/c \< 1.0 following a standard course of prednisone/prednisolone/methylprednisolone prescribed for FSGS therapy, OR contraindication/anticipated intolerance to steroid therapy defined as severe obesity, documented decreased bone density, family history of diabetes, or a psychiatric disorder. * Willingness to follow the protocol, including medications, baseline and follow-up visits, and procedures. Exclusion Criteria: * Lactation, pregnancy, or refusal of birth control in women of child bearing potential * Participation in another therapeutic trial involving protocol mandated administration of a immunosuppressive medication concurrently or 30 days prior to randomization * Active/serious infection (including, but not limited to…