Phase 2
N=18
Use of Low Dose Pioglitazone to Treat Autosomal Dominant Polycystic Kidney Disease
Polycystic Kidney Disease
Bottom Line
View on ClinicalTrials.gov: NCT02697617 ↗Enrolled (actual)
18
Serious AEs
8.3%
Results posted
Jan 2021
Primary outcome: Primary: Safety: Total Body Water — 45.78; 44.17 Ohms — p=0.024
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Pioglitazone (Drug); Placebo (Drug)
- Age
- Adult · 18+ yrs
- Sex
- All
- Sponsor
- Indiana University
- Primary completion
- Oct 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety: Total Body Water |
45.78; 44.17 | 0.024 sig |
| PRIMARY Efficacy: Percent Change in Total Kidney Volume |
4.35; 7.85 | 0.14 |
| SECONDARY Safety: Hypoglycemia |
1; 1 | — |
| SECONDARY Safety: Elevated Liver Function Tests |
0; 1 | — |
| SECONDARY Efficacy: Glomerular Filtration Rate |
75.5; 78.1 | 0.15 |
| SECONDARY Efficacy Blood Pressure |
127; 129; 83; 82 | 0.4 |
| SECONDARY Bone Marrow Fat |
— | — |
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.
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
Data sourced from ClinicalTrials.gov (NCT02697617). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.