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

NCT00920309 | Terminated Phase 2 Results DMC

• 1 other identifier: HIC#0903004934
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

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.

Conditions

Autosomal Dominant Polycystic Kidney Disease

Keywords

Autosomal Dominant Polycystic Kidney Disease; rapamycin; ADPKD

Outcome Measures

Primary Outcomes (1)

• Total Kidney Volume (mL)

  2 years

Secondary Outcomes (1)

• Glomerular Filtration Rate (Kidney Function)

  2 years

Study Arms (2)

Rapamycin

EXPERIMENTAL

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

Standard of Care-Placebo

PLACEBO COMPARATOR

Standard of Care

Other: Standard of Care-Placebo

Interventions

Rapamycin 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.

Also known as: sirolimus

Rapamycin

Standard of Care-Placebo OTHER

Standard of Care-Placebo

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Yale University: lead

Study Sites (1)

Yale Center for Clinical Investigation

New Haven, Connecticut, 06520, United States

Location

MeSH Terms

Conditions

Polycystic Kidney, Autosomal Dominant

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Polycystic Kidney Diseases; Kidney Diseases, Cystic; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ciliopathies; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Results Point of Contact

• Title: Dr. Neera Dahl
• Organization: Yale University School of Medicine

neera.dahl@yale.edu

203-785-4184

Study Officials

• Neera K Dahl, MD, PhD

  Yale University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 12, 2009
• First Posted: June 15, 2009
• Study Start: June 1, 2009
• Primary Completion: July 1, 2010
• Study Completion: July 1, 2010
• Last Updated: April 14, 2014
• Results First Posted: April 14, 2014

Record last verified: 2014-03

Locations

US (1)