Now Available: Final Rule for FDAAA 801 and NIH Policy on Clinical Trial Reporting
Text Size

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
History of Changes
  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
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Single Blind (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:

MedlinePlus related topics: Kidney Diseases
U.S. FDA Resources

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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • GFR [ Time Frame: 1.5 yrs ] [ Designated as safety issue: Yes ]
  • Adverse event [ Time Frame: 1.5 yrs ] [ Designated as safety issue: Yes ]
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
Ages Eligible for Study:   18 Years to 40 Years   (Adult)
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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:
Homepage Study Center  This link exits the ClinicalTrials.gov site

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 
Study First Received: June 22, 2006
Last Updated: February 26, 2014
Health Authority: Switzerland: Swissmedic

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

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

ClinicalTrials.gov processed this record on September 23, 2016