Use of Low Dose Pioglitazone to Treat Autosomal Dominant Polycystic Kidney Disease



Status:Active, not recruiting
Conditions:Renal Impairment / Chronic Kidney Disease
Therapuetic Areas:Nephrology / Urology
Healthy:No
Age Range:18 - 55
Updated:3/1/2019
Start Date:January 26, 2016
End Date:October 2020

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Use of Low Dose Pioglitazone to Treat Autosomal Dominant Polycystic Kidney

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.

Patients will be randomize to placebo or 15 mg pioglitazone for 12 months, and then be
crossed over to the other arm. Patients will undergo MRI of the liver and kidney and
MRspectroscopy of the lumbar spine (if they choose as this is ancillary study) three times
during the study. Assessments will be every 3 months and include blood work, blood pressure,
and body water assessments.

Inclusion Criteria:

- Male or female ADPKD patients aged 18-55

- eGFR 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 ECHO 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
We found this trial at
1
site
Indianapolis, Indiana 46202
Phone: 317-944-7580
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mi
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Indianapolis, IN
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