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

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ClinicalTrials.gov Identifier: NCT02697617
Recruitment Status : Completed
First Posted : March 3, 2016
Results First Posted : January 15, 2021
Last Update Posted : January 15, 2021
Sponsor:
Information provided by (Responsible Party):
Sharon Moe, Indiana University

Brief 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.


Condition or disease Intervention/treatment Phase
Polycystic Kidney Disease Drug: Pioglitazone Drug: Placebo Phase 2

Detailed Description:
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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 18 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: Use of Low Dose Pioglitazone to Treat Autosomal Dominant Polycystic Kidney
Actual Study Start Date : January 26, 2016
Actual Primary Completion Date : October 2019
Actual Study Completion Date : January 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Kidney Diseases

Arm Intervention/treatment
Placebo Comparator: Placebo Arm
Subject will be on placebo
Drug: Placebo
Placebo

Active Comparator: Pioglitazone Arm
Subject will be on pioglitazone
Drug: Pioglitazone
Pioglitazone
Other Name: Actos




Primary Outcome Measures :
  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 Outcome Measures :
  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.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 55 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02697617


Locations
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United States, Indiana
Indiana University Health
Indianapolis, Indiana, United States, 46202
Sponsors and Collaborators
Indiana University
Investigators
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Principal Investigator: Sharon Moe, 317-944-7580 Indiana University
  Study Documents (Full-Text)

Documents provided by Sharon Moe, Indiana University:
Informed Consent Form  [PDF] May 31, 2017

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Responsible Party: Sharon Moe, MD, Stuart A. Kleit Professor of Medicine, Director, Division of Nephrology, Indiana University
ClinicalTrials.gov Identifier: NCT02697617    
Other Study ID Numbers: IndianaU 1308084213
FD-R-004826-01-A2 ( Other Identifier: FDA Orphan Products Development Ad Hoc Panel Review )
First Posted: March 3, 2016    Key Record Dates
Results First Posted: January 15, 2021
Last Update Posted: January 15, 2021
Last Verified: December 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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Kidney Diseases
Polycystic Kidney Diseases
Polycystic Kidney, Autosomal Dominant
Urologic Diseases
Kidney Diseases, Cystic
Abnormalities, Multiple
Congenital Abnormalities
Ciliopathies
Genetic Diseases, Inborn
Pioglitazone
Hypoglycemic Agents
Physiological Effects of Drugs