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Title Metformin as a Novel Therapy for Autosomal Dominant Polycystic Kidney Disease
Therapeutic Area Polycystic Kidney Disease
Principal Investigator Ronald D. Perrone, MD
Gender Both
Contact Margaret Healy 617-636-8117
Carly Tucker 617-636-7914, ctucker@tuftsmedicalcenter.orgorg
More Information


This study will involve patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD) and is supported by the Peer Reviewed Medical Research Program of the Congressionally Directed Medical Research Program. The study has started at Tufts Medical Center and the University of Maryland (Baltimore). Drs. Ronald Perrone and Dana Miskulin of Tufts Medical Center and Drs. Terry Watnick and Steve Seliger of the University of Maryland will conduct the study at these clinical sites. The study will test the safety and tolerability of the antidiabetic drug metformin, which has been shown in animal models to slow the progression of PKD. Metformin is a widely used generic drug that is FDA approved for the treatment of diabetes.

Study Details

Inclusion Criteria

  • Glomerular filtration rate (GFR) greater than 50 mL/minute
  • Confirmed diagnosis of ADPKD

Exclusion Criteria

  • Have been on dialysis
  • Previous kidney transplant
  • Pregnant or planning to become pregnant in the next two years

Study Requirements

Study participation will last 26 months, and include a screening visit, baseline visit, and follow-up visits every 3 months, for a total of 12 visits. Each study visit will last approximately one hour.

Study visits at Tufts Medical center will include surveys of symptoms, a blood draw, and optional blood and urine collection for biobanking at the University of Maryland as well as testing at the University of Southern California.

The baseline visit as well as the 6 month follow-up visits (Months 0, 6, 12, 18, 24) will also include an MRI.

Between follow-up visits, patients will be asked to have intermediate blood draws either at Tufts Medical Center or at a convenient Quest lab to monitor kidney function and lactic acid levels.

After the baseline visit, patients will be assigned to the metformin group or placebo group, and will increase dose gradually based on tolerability of side effects.