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

Polycystic Kidney Disease Clinical Trial

— PIOPKD  

Official title:

Use of Low Dose Pioglitazone to Treat Autosomal Dominant Polycystic Kidney

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02697617
• Other study ID #: IndianaU 1308084213
• Secondary ID: FD-R-004826-01-A
• Status: Completed
• Phase: Phase 2
• Start date: January 26, 2016
• Est. completion date: January 2020

Study information

• Verified date: December 2020
• Source: Indiana University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 18
• Est. completion date: January 2020
• Est. primary completion date: October 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 55 Years

Eligibility

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

Related Conditions & MeSH terms

• Kidney Diseases
• Polycystic Kidney Disease
• Polycystic Kidney Diseases
• Polycystic Kidney, Autosomal Dominant

Intervention

Drug:
• Pioglitazone
Pioglitazone
• Placebo
Placebo

Locations

Country	Name	City	State
United States	Indiana University Health	Indianapolis	Indiana

Sponsors (1)

Lead Sponsor	Collaborator
Indiana University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety: Total Body Water	Bioimpedance analysis (BIA)(Ohms); Increase in BIA in Ohms indicates a decrease in total body water	average of 4 measures in each 12 month arm
Primary	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	Baseline, end of year 1, and end of year 2
Secondary	Safety: Hypoglycemia	number of patients with blood sugar < 70 mg/dl	measured quarterly for 12 months in pioglitazone and same in placebo
Secondary	Safety: Elevated Liver Function Tests	Number of patients with elevated liver test (ALT or AST) > 2 times upper limit of normal	measured quarterly over 12 months for each arm
Secondary	Efficacy: Glomerular Filtration Rate	average estimated glomerular filtration rate by chronic kidney disease (CKD) epidemiologic (epi) formula measured quarterly	average of 4 values over 12 months
Secondary	Efficacy Blood Pressure	mean systolic and diastolic blood pressure	average of 4 measures over 12 months
Secondary	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.	Baseline, end of year 1, and end of year 2