Sirolimus (Rapamune®) for Autosomal Dominant Polycystic Kidney Disease (ADPKD)
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ClinicalTrials.gov Identifier: NCT00346918 |
Recruitment Status :
Completed
First Posted : June 30, 2006
Last Update Posted : February 27, 2014
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Drug: Sirolimus Other: Standard | Phase 3 |
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 |

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) |
- renal volume measured by high resolution magnetic resolution imaging [ Time Frame: 1.5 yrs ]
- GFR [ Time Frame: 1.5 yrs ]
- Adverse event [ Time Frame: 1.5 yrs ]

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Ages Eligible for Study: | 18 Years to 40 Years (Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

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
Switzerland | |
University Hospital | |
Zurich, Switzerland, 8091 |
Principal Investigator: | Andreas L. Serra, MD | University of Zurich |
Publications of Results:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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 |
ADPKD autosomal dominant polycystic kidney disease Sirolimus volumetry |
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 |