Sirolimus for Massive Polycystic Liver (SILVER)

Seoul National University

Status and phase

Unknown

Phase 3

Phase 2

Conditions

Polycystic Kidney Diseases

Treatments

Drug: Sirolimus

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT01680250

SILVER

Details and patient eligibility

About

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

Full description

Autosomal dominant polycystic kidney disease (ADPKD) is one of the most common causes of end stage renal disease (ESRD), affecting an estimated 0.2% of population. Of ADPKD patients, 58% in 15-24 year, 85% in 25-34 year, and 94% in 35-46 year olds suffer from polycystic liver in addition to polycystic kidneys. Several anti-proliferative drugs have been used in clinical trials to stop cyst growth both in liver and kidneys. Among them, octreotide and sirolimus have been shown to be one of the most promising drugs to reduce cyst volume. Sirolimus already has been used as one of the most potential oral immunosuppressants. Moreover, the serum trough level is quite easy to measure. Sirolimus is the mTOR inhibitor that has been proven to be effective in reducing cyst growth both in animal models. However, its efficacy and safety is not well proven in previous studies. This is a open-label, prospective study to evaluate the effectiveness and safety of Sirolimus to reduce cyst growth in ADPKD patients with massive polycystic liver.

Enrollment

44 estimated patients

Sex

All

Ages

18 to 65 years old

Volunteers

No Healthy Volunteers

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