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

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.
 
ClinicalTrials.gov Identifier: NCT00346918
Recruitment Status : Completed
First Posted : June 30, 2006
Last Update Posted : February 27, 2014
Sponsor:
Information provided by (Responsible Party):
University of Zurich

Study Description
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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.

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

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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 100 participants
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.
Study Start Date : June 2006
Actual Primary Completion Date : January 2010
Actual Study Completion Date : June 2010

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Kidney Diseases
Drug Information available for: Sirolimus

Arms and Interventions
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Arm Intervention/treatment
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)


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

Secondary Outcome Measures :
  1. GFR [ Time Frame: 1.5 yrs ]
  2. Adverse event [ Time Frame: 1.5 yrs ]

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

Publications of Results:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: University of Zurich
ClinicalTrials.gov Identifier: NCT00346918    
Other Study ID Numbers: EK-1246
First Posted: June 30, 2006    Key Record Dates
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:
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Arthrogryposis
Kidney Diseases
Polycystic Kidney Diseases
Polycystic Kidney, Autosomal Dominant
Urologic Diseases
Joint Diseases
Musculoskeletal Diseases
Muscular Diseases
Musculoskeletal Abnormalities
Congenital Abnormalities
Kidney Diseases, Cystic
Abnormalities, Multiple
 Ciliopathies
Genetic Diseases, Inborn
Sirolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs