Feasibility Study of Metformin Therapy in ADPKD

Patients with ADPKD are still in need for a well-tolerated treatment that can be used
long-term to prevent cyst growth and kidney function decline. Metformin has a long track
record of a low risk-to-benefit profile in patients with diabetes or at risk for diabetes.
Metformin inhibits two key processes responsible for the growth of polycystic kidneys, i.e.
fluid secretion and cell proliferation, as shown in cell cultures and animal models of ADPKD.
Experiments in animal models of chronic kidney disease demonstrate that metformin
administration prevents kidney fibrosis and preserves kidney function. Diabetic patients who
are treated with metformin appear to develop less kidney failure and live longer than
patients who are treated with other anti-diabetic medications. Therefore this drug is
promising for people with ADPKD, with the potential to slow cyst enlargement and preserve
kidney function.

Inclusion Criteria:

- Autosomal Dominant Polycystic Kidney Disease and

- An estimated glomerular filtration (GFR) rate of 50-80 ml/min/1.73 m2;

- Subject is able to sign an Informed Consent

Exclusion Criteria:

- Diabetes mellitus,

- Active infection,

- Congestive heart failure,

- Liver disease,

- Alcohol or substance dependence,

- Cigarette smoking within the last 12 months;

- Females who are pregnant or breast feeding, or

- Are unwilling to use contraception;

- Are unable to undergo magnetic resonance imaging, or

- Have a contraindication to the use of metformin