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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

Tracking Information
First Submitted Date  ICMJE October 30, 2015
First Posted Date  ICMJE March 3, 2016
Results First Submitted Date  ICMJE October 19, 2020
Results First Posted Date  ICMJE January 15, 2021
Last Update Posted Date January 15, 2021
Actual Study Start Date  ICMJE January 26, 2016
Actual Primary Completion Date October 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 22, 2020)
  • 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
  • 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
Original Primary Outcome Measures  ICMJE
 (submitted: February 26, 2016)
To provide information on the safety of pioglitazone therapy in human ADPKD patients over a one year period assessed by edema, tolerance of drug, hematuria, and blood sugar levels. [ Time Frame: Quarterly (every 4 months) until the end of the study (predicted study end date is 12/31/2019) ]
Assessment of edema, tolerance of drug, hematuria, and blood sugar levels.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 22, 2020)
  • Safety: Hypoglycemia [ Time Frame: measured quarterly for 12 months in pioglitazone and same in placebo ]
    number of patients with blood sugar < 70 mg/dl
  • 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
  • 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
  • Efficacy Blood Pressure [ Time Frame: average of 4 measures over 12 months ]
    mean systolic and diastolic blood pressure
  • 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.
Original Secondary Outcome Measures  ICMJE
 (submitted: February 26, 2016)
  • To provide information on the efficacy of pioglitazone therapy in human ADPKD patients over a one year period using MRI monitoring of total kidney volume. [ Time Frame: Baseline, end of year 1, and end of year 2 ]
    We will assess change in kidney volume by MRI, change in liver volume by MRI.
  • To provide information on bone marrow fat content [ 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
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Use of Low Dose Pioglitazone to Treat Autosomal Dominant Polycystic Kidney Disease
Official Title  ICMJE Use of Low Dose Pioglitazone to Treat Autosomal Dominant Polycystic Kidney
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.

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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Polycystic Kidney Disease
Intervention  ICMJE
  • Drug: Pioglitazone
    Pioglitazone
    Other Name: Actos
  • Drug: Placebo
    Placebo
Study Arms  ICMJE
  • Placebo Comparator: Placebo Arm
    Subject will be on placebo
    Intervention: Drug: Placebo
  • Active Comparator: Pioglitazone Arm
    Subject will be on pioglitazone
    Intervention: Drug: Pioglitazone
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 16, 2018)
18
Original Estimated Enrollment  ICMJE
 (submitted: February 26, 2016)
28
Actual Study Completion Date  ICMJE January 2020
Actual Primary Completion Date October 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 55 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02697617
Other Study ID Numbers  ICMJE IndianaU 1308084213
FD-R-004826-01-A2 ( Other Identifier: FDA Orphan Products Development Ad Hoc Panel Review )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Sharon Moe, Indiana University
Study Sponsor  ICMJE Indiana University
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Sharon Moe, 317-944-7580 Indiana University
PRS Account Indiana University
Verification Date December 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP