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NCT Number: NCT02697617

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

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.

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Key information

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

pioglitazone

Drug

Pioglitazone

Other names: Actos

Placebo

Drug

Placebo

Primary outcomes

  1. Safety: Total Body Water

    Time frame: average of 4 measures in each 12 month arm

    Bioimpedance analysis (BIA)(Ohms); Increase in BIA in Ohms indicates a decrease in total body water

  2. Efficacy: Percent Change in Total Kidney Volume

    Time frame: Baseline, end of year 1, and end of year 2

    Change in total kidney volume by Magnetic Resonance Imaging (MRI) from beginning to end of the 12 months

Secondary outcomes

  1. Safety: Hypoglycemia

    Time frame: measured quarterly for 12 months in pioglitazone and same in placebo

    number of patients with blood sugar < 70 mg/dl

  2. Safety: Elevated Liver Function Tests

    Time frame: measured quarterly over 12 months for each arm

    Number of patients with elevated liver test (ALT or AST) > 2 times upper limit of normal

  3. Efficacy: Glomerular Filtration Rate

    Time frame: average of 4 values over 12 months

    average estimated glomerular filtration rate by chronic kidney disease (CKD) epidemiologic (epi) formula measured quarterly

  4. Efficacy Blood Pressure

    Time frame: average of 4 measures over 12 months

    mean systolic and diastolic blood pressure

  5. Bone Marrow Fat

    Time frame: Baseline, end of year 1, and end of year 2

    We will assess change in bone marrow fat by MR spectroscopy as an ancillary study to be done at the same time as MRI; will not be done due to person leaving institution.

Sponsors and collaborators

Lead sponsor

Indiana University

Other

Registry information

Official study title

Use of Low Dose Pioglitazone to Treat Autosomal Dominant Polycystic Kidney

Acronym: PIOPKD

Important dates

Study start
2016
Primary completion
2019
Study completion
2020
First posted
Mar 3, 2016
Registry last updated
Jan 15, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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