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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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 Summary: 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.
Sponsor: University of Zurich

Current Primary Outcome: renal volume measured by high resolution magnetic resolution imaging [ Time Frame: 1.5 yrs ]

Original Primary Outcome: renal volume measured by high resolution magnetic resolution imaging

Current Secondary Outcome:

  • GFR [ Time Frame: 1.5 yrs ]
  • Adverse event [ Time Frame: 1.5 yrs ]


Original Secondary Outcome:

  • GFR
  • Adverse event


Information By: University of Zurich

Dates:
Date Received: June 22, 2006
Date Started: June 2006
Date Completion:
Last Updated: February 26, 2014
Last Verified: February 2014