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

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ClinicalTrials.gov Identifier: NCT00920309
Recruitment Status : Terminated (This study was stopped after larger studies published in the NEJM failed to show a benefit in treating ADPKD)
First Posted : June 15, 2009
Results First Posted : April 14, 2014
Last Update Posted : April 14, 2014
Sponsor:
Information provided by (Responsible Party):
Yale University

Study Description
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Brief Summary:

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 or disease Intervention/treatment Phase
Autosomal Dominant Polycystic Kidney Disease Drug: Rapamycin Other: Standard of Care-Placebo Phase 2 Phase 3

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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 21 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Rapamycin as Treatment for ADPKD: The Role of Biomarkers in Predicting a Response to Therapy
Study Start Date : June 2009
Actual Primary Completion Date : July 2010
Actual Study Completion Date : July 2010

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Kidney Diseases

Arms and Interventions
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Arm Intervention/treatment
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


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

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

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

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
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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): NCT00920309


Locations
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United States, Connecticut
Yale Center for Clinical Investigation
New Haven, Connecticut, United States, 06520
Sponsors and Collaborators
Yale University
Investigators
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Principal Investigator: Neera K Dahl, MD, PhD Yale University
More Information
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Responsible Party: Yale University
ClinicalTrials.gov Identifier: NCT00920309    
Other Study ID Numbers: HIC#0903004934
First Posted: June 15, 2009    Key Record Dates
Results First Posted: April 14, 2014
Last Update Posted: April 14, 2014
Last Verified: March 2014
Keywords provided by Yale University:
Autosomal Dominant Polycystic Kidney Disease
rapamycin
ADPKD
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