Pirfenidone to Treat Kidney Disease (Focal Segmental Glomerulosclerosis)
Status:
Completed
Trial end date:
2008-10-01
Target enrollment:
Participant gender:
Summary
This study will examine the effectiveness of the drug pirfenidone in treating focal segmental
glomerulosclerosis (FSGS). Patients with this disease have kidney fibrosis (scarring) and
proteinuria (excessive excretion of protein in the urine). About half of patients with FSGS
eventually require kidney dialysis or transplant. Steroids, which are currently used to treat
the disease, are effective in only a minority of patients. Other drugs, such as cyclosporin
and cyclophosphamide, improve proteinuria in a very small percentage of patients and have
serious side effects.
Patients with FSGS who wish to participate in this study will undergo pre-study evaluation
with blood and urine tests. Patients must be on a stable dose of an ACE inhibitor (a drug
that lowers blood pressure and reduces proteinuria) for at list 6 months before starting
pirfenidone therapy. (Patients who are not already taking an ACE inhibitor will be started on
the drug; those who cannot tolerate ACE inhibitors will be given a different drug.) Patients
with elevated cholesterol will take a cholesterol-lowering drug. A diet containing
approximately 1 gram of protein per kilogram of body weight per day will be recommended.
Patients will take pirfenidone by mouth 3 times a day for 12 months. Blood and urine will be
tested once a month, either at NIH or by the patient's local kidney specialist. They will
collect two 24-hour urine samples at the beginning of the treatment period, at 2-month
intervals throughout the study, and at a 6-month follow-up. Patients will also be asked to
give three to five tubes of blood and urine samples for analysis during the study.
In animal studies, pirfenidone improved kidney function and proteinuria and reduced kidney
scarring in rats with a disease similar to FSGS. In human studies, pirfenidone improved
breathing and survival in patients with lung fibrosis.
Phase:
Phase 2
Details
Lead Sponsor:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)