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

Phase 2

Terminated

• Conditions: Autosomal Dominant Polycystic Kidney Disease
• Interventions: Other: Standard of Care-Placebo; Drug: Rapamycin

• Registration Number: NCT00920309
• Lead Sponsor: Yale University

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.

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 Timeline

• Study Start: 2009-06
• Study First Submitted: 2009-06-12
• Study First Submitted QC: 2009-06-12
• Study First Posted: 2009-06-15
• Primary Completion: 2010-07
• Study Completion: 2010-07
• Results First Submitted: 2013-08-29
• Status Verified: 2014-03
• Results First Submitted QC: 2014-03-07
• Last Update Submitted: 2014-03-07
• Results First Posted: 2014-04-14
• Last Update Posted: 2014-04-14

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 21

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Standard of Care-Placebo	Standard of Care-Placebo	Standard of Care
Rapamycin	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.

Primary Outcome Measures

Name	Time	Method
Total Kidney Volume (mL)	2 years

Secondary Outcome Measures

Name	Time	Method
Glomerular Filtration Rate (Kidney Function)	2 years

Trial Locations

Locations (1)

Yale Center for Clinical Investigation; 🇺🇸 New Haven, Connecticut, United States