Sirolimus (Rapamune®) for Autosomal Dominant Polycystic Kidney Disease (ADPKD)

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Brief Title

Sirolimus (Rapamune®) for Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Official Title

Sirolimus (Rapamune®) for Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD): a Randomized Controlled Study.

Brief Summary

      The aim of our study is to investigate whether Rapamune used at a low dose (2 mg/d) retards
      cyst growth and slows renal functional deterioration in patients with ADPKD.
    

Detailed Description

      Currently there is no treatment for ADPKD other than supportive care and blood pressure
      control. Usually dialytic treatment or renal transplantation becomes necessary when the
      disease has progressed to end-stage renal failure.We and others could demonstrate that
      rapamycin, a classical mTOR inhibitor, retards cyst growth and preserves renal function in a
      rodent model of ADPKD. The aim of our study is to investigate whether Rapamune (2 mg/d)
      retards cyst growth and slows renal functional deterioration in patients with ADPKD. We
      anticipate that we can slow disease progression and delay the need for chronic renal
      replacement therapy by the inhibition of mTOR with Rapamune. This is a 24-month prospective,
      controlled, open label study with 2 parallel groups in patients with ADPKD. Patients will be
      randomized at a 1:1 ratio to one of the 2 treatment arms. Primary endpoint is percentage
      change of renal volume measured by high resolution magnetic resolution imaging.
    

Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

renal volume measured by high resolution magnetic resolution imaging

Secondary Outcome

 GFR

Condition

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Intervention

Sirolimus

Study Arms / Comparison Groups

 1
Description:  Treatment of hypertension, cyst infections and flank pain

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

100

Start Date

June 2006

Completion Date

June 2010

Primary Completion Date

January 2010

Eligibility Criteria

        Inclusion Criteria:

          -  Male or female ADPKD patient between 18 and 40 years of age

          -  measured GFR higher than 70 ml/min 1.73m2

          -  documented kidney volume progression

          -  informed consent

        Exclusion Criteria:

          -  Female of childbearing potential who is planning to become pregnant, who is pregnant
             and/or lactating, who is unwilling to use effective means of contraception

          -  increased liver enzymes (2-fold above normal values)

          -  hypercholesterolemia (fasting cholesterol > 8 mmol/l) or hypertriglyceridaemia (> 5
             mmol/l) not controlled by lipid lowering therapy

          -  granulocytopenia (white blood cell < 3,000/mm3) or thrombocytopenia (platelets <
             100,000/mm3),

          -  infection with hepatitis B or C, HIV

          -  any past or present malignancy

          -  mental illness that interfere with the patient ability to comply with the protocol

          -  drug or alcohol abuse within one year of baseline

          -  co-medication with strong inhibitor of CYP3A4 and or P-gp like voriconazole,
             ketoconazole, diltiazem, verapamil, erythromycin

          -  co-medication with strong CYP3A4 and or P-gp inductor like rifampicin

          -  known hypersensitivity to macrolides or Rapamune
      

Gender

All

Ages

18 Years - 40 Years

Accepts Healthy Volunteers

No

Contacts

Andreas L. Serra, MD, , 

Location Countries

Switzerland

Location Countries

Switzerland

Administrative Informations


NCT ID

NCT00346918

Organization ID

EK-1246


Responsible Party

Sponsor

Study Sponsor

University of Zurich


Study Sponsor

Andreas L. Serra, MD, Principal Investigator, University of Zurich


Verification Date

February 2014