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Sirolimus for Massive Polycystic Liver

NCT ID: NCT01680250

Last Updated: 2012-09-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE2/PHASE3

Total Enrollment

44 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-09-30

Study Completion Date

2015-08-31

Brief Summary

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

Detailed Description

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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.

Conditions

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Polycystic Kidney Diseases

Keywords

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Total liver volume Liver cyst

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Sirolimus

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

Group Type EXPERIMENTAL

Sirolimus

Intervention Type DRUG

Sirolimus administration for 12 months followed by conventional therapy alone for additional 12 months

Interventions

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Sirolimus

Sirolimus administration for 12 months followed by conventional therapy alone for additional 12 months

Intervention Type DRUG

Other Intervention Names

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Rapamune

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Wyeth is now a wholly owned subsidiary of Pfizer

INDUSTRY

Sponsor Role collaborator

Seoul National University Hospital

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Curie Ahn, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Seoul National University Hospital

Locations

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Seoul National University Hospital

Seoul, , South Korea

Site Status RECRUITING

Countries

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South Korea

Central Contacts

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Curie Ahn, MD, PhD

Role: CONTACT

Phone: 82-2-2072-2222

Email: [email protected]

Facility Contacts

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Curie Ahn, MD, PhD

Role: primary

Other Identifiers

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SILVER

Identifier Type: -

Identifier Source: org_study_id