NCT00346918

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

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 100 (actual)
• Sponsor: University of Zurich · Academic / Other
• Sex: All
• Age: 18 Years – 40 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Interventions

Type	Name	Description
DRUG	Sirolimus	Standard plus Sirolimus
OTHER	Standard	Standard

Timeline

• Start date: 2006-06-01
• Primary completion: 2010-01-01
• Completion: 2010-06-01
• First posted: 2006-06-30
• Last updated: 2014-02-27

Locations

1 site across 1 country: Switzerland