Try our beta test site
IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...

Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to Therapy

This study has been terminated.
(This study was stopped after larger studies published in the NEJM failed to show a benefit in treating ADPKD)
Information provided by (Responsible Party):
Yale University Identifier:
First received: June 12, 2009
Last updated: March 7, 2014
Last verified: March 2014

Currently the only approved use for rapamycin (sirolimus) is for immunosuppression after renal transplantation.

This trial is designed to determine whether rapamycin is safe and effective treatment for patients with polycystic kidney disease (ADPKD). Patients will be followed by volumetric magnetic resonance imaging (MRI) to observe for change in kidney (and cyst) size. Blood and urine samples will also be collected to evaluate for change in biomarkers with treatment.

Condition Intervention Phase
Autosomal Dominant Polycystic Kidney Disease
Drug: Rapamycin
Other: Standard of Care-Placebo
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Rapamycin as Treatment for ADPKD: The Role of Biomarkers in Predicting a Response to Therapy

Resource links provided by NLM:

Further study details as provided by Yale University:

Primary Outcome Measures:
  • Total Kidney Volume (mL) [ Time Frame: 2 years ]

Secondary Outcome Measures:
  • Glomerular Filtration Rate (Kidney Function) [ Time Frame: 2 years ]

Enrollment: 21
Study Start Date: June 2009
Study Completion Date: July 2010
Primary Completion Date: July 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Rapamycin

Drug: Rapamycin

Other Names:

sirolimus The starting dose of rapamycin will be 1 mg daily. The dose will be increased as needed to achieve a 24 hour trough level of 4-6 ng/ml.

Drug: Rapamycin
The starting dose of rapamycin will be 1 mg daily. The dose will be increased as needed to achieve a 24 hour trough level of 4-6 ng/ml.
Other Name: sirolimus
Placebo Comparator: Standard of Care-Placebo
Standard of Care
Other: Standard of Care-Placebo

Detailed Description:
This is a 2 year, open label trial to evaluate the role of rapamycin as treatment for ADPKD. Patients will be randomized 2:1 to the rapamycin arm or to standard therapy. The dose of rapamycin will be adjusted so that patients obtain 24 trough levels of 4-6ng/ml. There will be a volumetric MRI measurement at the start and end of the treatment period. Patients will be monitored every 4 months throughout the study.

Ages Eligible for Study:   18 Years to 70 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes

Inclusion Criteria:

  • adult ADPKD patients aged 18-70
  • combined kidney volume >1200 ml
  • estimated creatinine clearance >60 ml/min
  • absence of implanted ferromagnetic objects

Exclusion Criteria:

  • Age >70
  • uncontrolled hyperlipidemia
  • Proteinuria >500 mg/day
  • unstable cerebral aneurysm
  • active coronary artery disease
  • diagnosis of another systemic condition affecting kidney function (ie: diabetes, hepatitis B or C, HIV)
  • diagnosis of cancer other than skin cancer
  • pregnancy or lactation
  • presence of implanted ferromagnetic objects
  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 identifier: NCT00920309

United States, Connecticut
Yale Center for Clinical Investigation
New Haven, Connecticut, United States, 06520
Sponsors and Collaborators
Yale University
Principal Investigator: Neera K Dahl, MD, PhD Yale University
  More Information

Responsible Party: Yale University Identifier: NCT00920309     History of Changes
Other Study ID Numbers: HIC#0903004934
Study First Received: June 12, 2009
Results First Received: August 29, 2013
Last Updated: March 7, 2014

Keywords provided by Yale University:
Autosomal Dominant Polycystic Kidney Disease

Additional relevant MeSH terms:
Kidney Diseases
Polycystic Kidney, Autosomal Dominant
Polycystic Kidney Diseases
Urologic Diseases
Kidney Diseases, Cystic
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs processed this record on May 24, 2017