Idiopathic Focal Segmental Glomerulosclerosis (FSGS) and Treatment With ACTH

EXPERIMENTAL TREATMENT: Patients with biopsy proven FSGS will be treated with ACTH in an open-label pilot study of tolerability and efficacy. The investigators will recruit 18-20 patients to complete this study, over 2 years.

ACTH therapy: Patients will receive H.P. Acthar gel IM or SQ, initially at 40 IU SQ every week for 16 weeks of therapy. All patients will be treated to goal BP of <140/90mmHg with all patients treated with ACEi or ARB therapy as tolerated. H2 receptor blockade or proton pump inhibitor therapy will also be offered all patients. Dose will be titrated up to 160 IU SQ every week, if at 1 month the patient has had no substantial reduction in proteinuria, no deterioration of blood pressure and no development of hyperglycemia as well as no serious adverse events felt to be related to the medication.

CLINICAL OUTCOME: Patients will be followed for the primary outcome of remission of proteinuria. This will be defined as partial remission when the proteinuria is reduced below 50% of the initial, pre treatment value and as complete remission when the proteinuria is reduced to < 0.5 g/g or <500 mg/day on a 24 hour urine sample. Secondary outcomes will include effects on eGFR, effects on glucose levels, effects on blood pressure (total doses of antihypertensive medications and absolute changes in blood pressure) and on immune status. Outcomes will be determined by looking at 3 month and 6 month values of urine protein and eGFR following initiation of treatment.

IMMUNOLOGIC TESTING: In order to further assess the role of immunologic perturations on FSGS and the effect of ACTH on the immune system, all patients will bank blood and urine before the start of the study for cytokine analysis, RNA, DNA, protein and protoarray testing.

MONITORING AND SAFETY: All patients will undergo full informed consent per the Stanford Institutional Review Board. Contact numbers will be provided and study staff will be available at all times in case of any medical emergencies. All patients will continue with routine health monitoring with a minimal of monthly assessments. A comprehensive interview will be undertaken to assess for side effects, complete physical exams will be accomplished including vital signs, CBC, clinical chemistries (including electrolytes, creatinine, glucose and liver function tests), urine for protein and creatinine and fasting lipid profiles every 3 months. Also at screening and baseline.