A Phase II Multi-Center Study of Rapamycin for Treating Kidney Angiomyolipomas in TSC or LAM Patients
- Determine the efficacy of sirolimus, in terms of objective response rate, in patients
with angiomyolipoma of the kidney secondary to tuberous sclerosis (TSC) or
- Determine the toxicity of this drug in these patients.
- Determine changes in other TSC lesions (e.g., tubers, subependymal giant cell
astrocytomas, facial angiofibromas, or kidney cysts) in patients with TSC treated with
- Determine changes in pulmonary disease in patients with LAM treated with this drug.
OUTLINE: This is a multicenter study.
Patients receive oral sirolimus once daily for 12 months in the absence of unacceptable
After completion of study treatment, patients are followed at 6 months and 1 year.
PROJECTED ACCRUAL: A total of 13-35 patients will be accrued for this study within 3-30
Masking: Open Label, Primary Purpose: Treatment
Objective response as assessed by RECIST criteria
Sandra Dabora, MD, PhD
Dana-Farber/Brigham and Women's Cancer Center
United States: Federal Government
|Cincinnati Children's Hospital Medical Center||Cincinnati, Ohio 45229-3039|
|University of Texas Southwestern Medical Center at Dallas||Dallas, Texas 75235-8897|
|Massachusetts General Hospital||Boston, Massachusetts 02114-2617|
|Connecticut Children's Medical Center||Hartford, Connecticut 06106|
|New York University Medical Center||New York, New York 10016|
|Tuberous Sclerosis Clinic at Loma Linda University Medical Center||Loma Linda, California 92350|