Suspended

SILVERAn Open-label, Prospective Clinical Trial to Evaluate the Effectiveness and Safety of Sirolimus to Reduce Cyst Growth in ADPKD Patients With Massive Polycystic Liver

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What is being tested

Sirolimus

Drug
Who is being recruted

Congenital Abnormalities+10

+ Abnormalities, Multiple

+ Ciliopathies

From 18 to 65 Years
+13 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Treatment Study

Phase 2 & 3
Interventional
Study Start: September 2011
See protocol details

Summary

Principal SponsorSeoul National University Hospital
Study ContactCurie Ahn, MD, PhDMore contacts
Last updated: September 7, 2012
Sourced from a government-validated database.Claim as a partner

Study start date: September 1, 2011

Actual date on which the first participant was enrolled.

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.

NCT01680250
Principal SponsorSeoul National University Hospital
Study ContactCurie Ahn, MD, PhDMore contacts
Last updated: September 7, 2012
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

44 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 18 to 65 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Congenital AbnormalitiesAbnormalities, MultipleCiliopathiesUrogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsKidney DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesUrologic DiseasesGenetic Diseases, InbornKidney Diseases, CysticFemale Urogenital DiseasesMale Urogenital DiseasesPolycystic Kidney Diseases

Criteria

4 inclusion criteria required to participate
Age 18 - 65
Estimated glomerular filtration rate (IDMS-traceable MDRD equation) >= 30 mL/min/1.73m2
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
9 exclusion criteria prevent from participating
Any condition that could prevent full comprehension of the purpose and risks of the study
Concomitant systemic renal parenchymal or urinary tract disease (random urine albumin-to-creatinine ratio > 500 mg/g)
Diabetes mellitus, cancer, or psychiatric disorder
History of intervention, such as cyst aspiration or embolization in past 1 year
Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

One single intervention group is designated in this study

This study does not include a placebo group 

Treatment Groups

Group I

Experimental
Sirolimus administration group starting dose: 2mg/day target trough level: 4-10 ng/dL

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 1 location

Recruiting

Seoul National University Hospital

Seoul, South KoreaOpen Seoul National University Hospital in Google Maps
SuspendedOne Study Center