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

NCT ID: NCT02697617

Last Updated: 2021-01-15

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 18 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-01-26
• Study Completion Date: 2020-01-31

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.

Conditions

Polycystic Kidney Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Placebo Arm

Subject will be on placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo

Pioglitazone Arm

Subject will be on pioglitazone

Group Type: ACTIVE_COMPARATOR

Pioglitazone

Intervention Type: DRUG

Pioglitazone

Interventions

Pioglitazone

Pioglitazone

Intervention Type: DRUG

Placebo

Placebo

Intervention Type: DRUG

Other Intervention Names

Actos

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 55 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Indiana University

OTHER

Sponsor Role: lead

Responsible Party

Sharon Moe

MD, Stuart A. Kleit Professor of Medicine, Director, Division of Nephrology

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Sharon Moe, 317-944-7580

Role: PRINCIPAL_INVESTIGATOR

Indiana University

Locations

Indiana University Health

Indianapolis, Indiana, United States

Countries

United States

References

St Pierre K, Cashmore BA, Bolignano D, Zoccali C, Ruospo M, Craig JC, Strippoli GF, Mallett AJ, Green SC, Tunnicliffe DJ. Interventions for preventing the progression of autosomal dominant polycystic kidney disease. Cochrane Database Syst Rev. 2024 Oct 2;10(10):CD010294. doi: 10.1002/14651858.CD010294.pub3.

Reference Type: DERIVED

PMID: 39356039 (View on PubMed)

Provided Documents

Document Type: Informed Consent Form

View Document

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

FD-R-004826-01-A2

Identifier Type: OTHER

Identifier Source: secondary_id

IndianaU 1308084213

Identifier Type: -

Identifier Source: org_study_id