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

• Status: Completed
• Trial end date: 2010-06-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Zurich
• Treatments: Everolimus; Sirolimus

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