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

NCT ID: NCT00920309

Last Updated: 2014-04-14

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 21 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2009-06-30
• Study Completion Date: 2010-07-31

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.

Conditions

Autosomal Dominant Polycystic Kidney Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Rapamycin

Intervention Type: DRUG

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.

Standard of Care-Placebo

Standard of Care

Group Type: PLACEBO_COMPARATOR

Standard of Care-Placebo

Intervention Type: OTHER

Interventions

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.

Intervention Type: DRUG

Standard of Care-Placebo

Intervention Type: OTHER

Other Intervention Names

sirolimus

Eligibility 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Yale University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Neera K Dahl, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Yale University

Locations

Yale Center for Clinical Investigation

New Haven, Connecticut, United States

Countries

United States

Other Identifiers

HIC#0903004934

Identifier Type: -

Identifier Source: org_study_id