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Trial record 1 of 1 for:    NCT00346918
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Sirolimus (Rapamune®) for Autosomal Dominant Polycystic Kidney Disease (ADPKD)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
University of Zurich
ClinicalTrials.gov Identifier:
NCT00346918
First received: June 22, 2006
Last updated: February 26, 2014
Last verified: February 2014

June 22, 2006
February 26, 2014
June 2006
January 2010   (final data collection date for primary outcome measure)
renal volume measured by high resolution magnetic resolution imaging [ Time Frame: 1.5 yrs ] [ Designated as safety issue: No ]
renal volume measured by high resolution magnetic resolution imaging
Complete list of historical versions of study NCT00346918 on ClinicalTrials.gov Archive Site
  • GFR [ Time Frame: 1.5 yrs ] [ Designated as safety issue: Yes ]
  • Adverse event [ Time Frame: 1.5 yrs ] [ Designated as safety issue: Yes ]
  • GFR
  • Adverse event
Not Provided
Not Provided
 
Sirolimus (Rapamune®) for Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Sirolimus (Rapamune®) for Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD): a Randomized Controlled Study.

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.

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.

Interventional
Phase 3
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Outcomes Assessor)
Primary Purpose: Treatment
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
  • Drug: Sirolimus
    Standard plus Sirolimus
    Other Name: Rapamune (R)
  • Other: Standard
    Standard
    Other Name: Treatment of hypertension, cyst infections and flank pain
  • Active Comparator: 1
    Treatment of hypertension, cyst infections and flank pain
    Intervention: Other: Standard
  • Active Comparator: 2
    Sirolimus plus Standard Treatment
    Intervention: Drug: Sirolimus

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
100
June 2010
January 2010   (final data collection date for primary outcome measure)

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
Both
18 Years to 40 Years
No
Contact information is only displayed when the study is recruiting subjects
Switzerland
 
NCT00346918
EK-1246
Yes
University of Zurich
University of Zurich
Not Provided
Principal Investigator: Andreas L. Serra, MD University of Zurich
University of Zurich
February 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP