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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00346918
First Posted: June 30, 2006
Last Update Posted: February 27, 2014
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
University of Zurich
  Purpose
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.

Condition Intervention Phase
Autosomal Dominant Polycystic Kidney Disease (ADPKD) Drug: Sirolimus Other: Standard Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Sirolimus (Rapamune®) for Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD): a Randomized Controlled Study.

Resource links provided by NLM:


Further study details as provided by University of Zurich:

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

Secondary Outcome Measures:
  • GFR [ Time Frame: 1.5 yrs ]
  • Adverse event [ Time Frame: 1.5 yrs ]

Enrollment: 100
Study Start Date: June 2006
Study Completion Date: June 2010
Primary Completion Date: January 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: 1
Treatment of hypertension, cyst infections and flank pain
Other: Standard
Standard
Other Name: Treatment of hypertension, cyst infections and flank pain
Active Comparator: 2
Sirolimus plus Standard Treatment
Drug: Sirolimus
Standard plus Sirolimus
Other Name: Rapamune (R)

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.
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 40 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00346918


Locations
Switzerland
University Hospital
Zurich, Switzerland, 8091
Sponsors and Collaborators
University of Zurich
Investigators
Principal Investigator: Andreas L. Serra, MD University of Zurich
  More Information

Additional Information:
Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: University of Zurich
ClinicalTrials.gov Identifier: NCT00346918     History of Changes
Other Study ID Numbers: EK-1246
First Submitted: June 22, 2006
First Posted: June 30, 2006
Last Update Posted: February 27, 2014
Last Verified: February 2014

Keywords provided by University of Zurich:
ADPKD
autosomal dominant polycystic kidney disease
Sirolimus
volumetry

Additional relevant MeSH terms:
Polycystic Kidney Diseases
Polycystic Kidney, Autosomal Dominant
Kidney Diseases
Urologic Diseases
Kidney Diseases, Cystic
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn
Sirolimus
Everolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs