Use of Low Dose Pioglitazone to Treat Autosomal Dominant Polycystic Kidney
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 18
- 试验地点
- 1
- 主要终点
- Safety: Total Body Water
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 55 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
Placebo Arm
Subject will be on placebo
干预措施: Placebo (Drug)
Pioglitazone Arm
Subject will be on pioglitazone
干预措施: Pioglitazone (Drug)
结局指标
主要结局
Safety: Total Body Water
时间窗: 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
时间窗: 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
次要结局
- Safety: Elevated Liver Function Tests(measured quarterly over 12 months for each arm)
- Safety: Hypoglycemia(measured quarterly for 12 months in pioglitazone and same in placebo)
- Efficacy: Glomerular Filtration Rate(average of 4 values over 12 months)
- Efficacy Blood Pressure(average of 4 measures over 12 months)
- Bone Marrow Fat(Baseline, end of year 1, and end of year 2)
研究者
Sharon Moe
MD, Stuart A. Kleit Professor of Medicine, Director, Division of Nephrology
Indiana University