Use of Low Dose Pioglitazone to Treat Autosomal Dominant Polycystic Kidney Disease
Condition: Polycystic Kidney Disease
MeSH terms: Kidney Diseases; Polycystic Kidney Diseases; Polycystic Kidney, Autosomal Dominant
Intervention: Pioglitazone; Placebo
Intervention description: Pioglitazone; Placebo
Intervention MeSH terms: Pioglitazone
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Double (Participant, Investigator)
Enrollment: 18.0
Sex: All
Minimum age: 18 Years
Maximum age: 55 Years
Healthy volunteers: No

Summary:

      Funding Source - FDA OOPD

      Pioglitazone is currently used in clinical practice to treat diabetes and this study will
      examine the potential use of a low dose of the same drug for the treatment of polycystic
      kidney disease. The purpose of this study is to determine whether the diabetes drug
      pioglitazone (Actos) is a safe and effective treatment of autosomal dominant polycystic
      kidney disease when treated in its early stages. Pioglitazone is approved by the FDA for the
      treatment of diabetes. Pre-clinical models of polycystic kidney disease have shown that low
      dose treatment with pioglitazone decreases the growth of the cysts. The studies also suggest
      that effective pioglitazone dosing for polycystic kidney disease may be lower than that used
      to treat diabetes. The purpose of this study is to see if pioglitazone might slow cyst
      disease in humans.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male or female patients with autosomal dominant polycystic kidney disease (ADPKD) aged
             18-55

          -  estimate glomerular filtration rate (GFR) at or above ≥ 50 ml/min/1.73 m2 by any GFR
             formula

          -  Normal liver enzymes (ALT/AST)

          -  fasting blood glucose between 70 and120

          -  for female patients, a willingness to use double contraception to avoid pregnancy
             while in study

          -  able to give informed consent

          -  In the opinion of the investigator, high likelihood of progressive kidney disease

        Exclusion Criteria:

          -  diabetes, defined as any of the following: fasting blood sugar > 130 times two, HgbA1C
             > 7, on any blood sugar lowering medication, or past diagnosis of diabetes not
             occurring during pregnancy

          -  uncontrolled hypertension as determined by the examining physician

          -  history of impaired systolic function (ejection fraction < 50%) by previous
             echocardiogram or known ischemic cardiovascular disease

          -  findings suggestive of a kidney disease other than ADPKD

          -  systemic illness requiring immunosuppressive or anti-inflammatory agents

          -  congenital absence of a kidney or history of a total nephrectomy

          -  history of cyst reduction or partial nephrectomy

          -  history of renal cyst aspiration within the previous year

          -  History of bladder cancer, or gross hematuria

          -  inability to undergo MRI due to implantable devices or foreign objects that preclude
             MRI

          -  active renal transplant

          -  allergy or sensitivity to any of the components of the test materials

          -  institutionalized

          -  currently pregnant or plans to become pregnant during the study