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

Primary Purpose

Polycystic Kidney Disease

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Pioglitazone

Placebo

About this trial

This is an interventional treatment trial for Polycystic Kidney Disease

Eligibility Criteria

18 Years - 55 Years (Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Indiana University Health

Arms of the Study

Arm 1

Arm 2

Arm Type

Placebo Comparator

Active Comparator

Arm Label

Placebo Arm

Pioglitazone Arm

Arm Description

Subject will be on placebo

Subject will be on pioglitazone

Outcomes

Primary Outcome Measures

Safety: Total Body Water

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

Efficacy: Percent Change in Total Kidney Volume

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

Secondary Outcome Measures

Safety: Hypoglycemia

number of patients with blood sugar < 70 mg/dl

Safety: Elevated Liver Function Tests

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

Efficacy: Glomerular Filtration Rate

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

Efficacy Blood Pressure

mean systolic and diastolic blood pressure

Bone Marrow Fat

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.

Full Information

NCT ID

NCT02697617

First Posted

October 30, 2015

Last Updated

December 22, 2020

Sponsor

Indiana University

1. Study Identification

Unique Protocol Identification Number

NCT02697617

Brief Title

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

Acronym

PIOPKD

Official Title

Use of Low Dose Pioglitazone to Treat Autosomal Dominant Polycystic Kidney

Study Type

Interventional

2. Study Status

Record Verification Date

December 2020

Overall Recruitment Status

Completed

Study Start Date

January 26, 2016 (Actual)

Primary Completion Date

October 2019 (Actual)

Study Completion Date

January 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Indiana University

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Polycystic Kidney Disease

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Crossover Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

18 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Placebo Arm

Arm Type

Placebo Comparator

Arm Description

Subject will be on placebo

Arm Title

Pioglitazone Arm

Arm Type

Active Comparator

Arm Description

Subject will be on pioglitazone

Intervention Type

Drug

Intervention Name(s)

Pioglitazone

Other Intervention Name(s)

Actos

Intervention Description

Pioglitazone

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

Placebo

Primary Outcome Measure Information:

Title

Safety: Total Body Water

Description

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

Time Frame

average of 4 measures in each 12 month arm

Title

Efficacy: Percent Change in Total Kidney Volume

Description

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

Time Frame

Baseline, end of year 1, and end of year 2

Secondary Outcome Measure Information:

Title

Safety: Hypoglycemia

Description

number of patients with blood sugar < 70 mg/dl

Time Frame

measured quarterly for 12 months in pioglitazone and same in placebo

Title

Safety: Elevated Liver Function Tests

Description

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

Time Frame

measured quarterly over 12 months for each arm

Title

Efficacy: Glomerular Filtration Rate

Description

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

Time Frame

average of 4 values over 12 months

Title

Efficacy Blood Pressure

Description

mean systolic and diastolic blood pressure

Time Frame

average of 4 measures over 12 months

Title

Bone Marrow Fat

Description

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.

Time Frame

Baseline, end of year 1, and end of year 2

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

55 Years

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Sharon Moe, 317-944-7580

Organizational Affiliation

Indiana University

Official's Role

Principal Investigator

Facility Information:

Facility Name

Indiana University Health

City

Indianapolis

State/Province

Indiana

ZIP/Postal Code

46202

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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