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Sirolimus (Rapamune®) for Autosomal Dominant Polycystic Kidney Disease (ADPKD)
This study has been completed.
Sponsor:
University of Zurich
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
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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 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:
Genetics Home Reference related topics:
polycystic kidney disease
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 ]
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| 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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Please refer to this study by its ClinicalTrials.gov identifier: NCT00346918
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:
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 |
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 July 21, 2017