Sirolimus for Massive Polycystic Liver

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Brief Title

Sirolimus for Massive Polycystic Liver

Official Title

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

Brief 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.
    

Detailed 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.
    

Study Phase

Phase 2/Phase 3

Study Type

Interventional


Primary Outcome

Total liver volume

Secondary Outcome

 Total liver volume

Condition

Polycystic Kidney Diseases

Intervention

Sirolimus

Study Arms / Comparison Groups

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

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

44

Start Date

September 2011

Completion Date

August 2015

Primary Completion Date

September 2014

Eligibility 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
      

Gender

All

Ages

18 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

Curie Ahn, MD, PhD, 82-2-2072-2222, [email protected]

Location Countries

Korea, Republic of

Location Countries

Korea, Republic of

Administrative Informations


NCT ID

NCT01680250

Organization ID

SILVER


Responsible Party

Sponsor

Study Sponsor

Seoul National University Hospital

Collaborators

 Wyeth is now a wholly owned subsidiary of Pfizer

Study Sponsor

Curie Ahn, MD, PhD, Principal Investigator, Seoul National University Hospital


Verification Date

September 2012