Sirolimus for Massive Polycystic Liver

• Status: Unknown status
• Trial end date: 2015-08-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to evaluate the effectiveness and safety of Sirolimus in reducing liver volume in autosomal dominant polycystic kidney disease.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Seoul National University Hospital
• Collaborator: Wyeth is now a wholly owned subsidiary of Pfizer
• Treatments: Everolimus; Sirolimus

Criteria

Inclusion Criteria:

• Age 18 - 65

• Patients diagnosed as ADPKD based upon the unified criteria for ultrasonographic diagnosis of ADPKD

• Polycystic liver with total liver volume > 2500 mL or symptomatic polycystic liver

• Estimated glomerular filtration rate (IDMS-traceable MDRD equation) >= 30 mL/min/1.73m2

Exclusion Criteria:

• Concomitant systemic renal parenchymal or urinary tract disease (random urine albumin-to-creatinine ratio > 500 mg/g)

• WBC < 4,000/uL, platelet < 100,000/uL, or hemoglobin < 10.0 g/dL

• Diabetes mellitus, cancer, or psychiatric disorder

• Increased liver enzymes (2-fold above normal value)

• Hypercholesterolemia (fasting cholesterol > 200mg/dL) or hypertriglyceridemia (>150 mg/dL) not controlled by lipid lowering therapy

• Infection with hepatitis B, C, HIV

• Any condition that could prevent full comprehension of the purpose and risks of the study

• Pregnant or lactating women or fertile women without effective contraception

• History of intervention, such as cyst aspiration or embolization in past 1 year