A Phase II Multi-Center Study of Rapamycin for Treating Kidney Angiomyolipomas in TSC or LAM Patients
18 Years
65 Years
Both
Nonmalignant Neoplasm
Trial Information
A Phase II Multi-Center Study of Rapamycin for Treating Kidney Angiomyolipomas in TSC or LAM Patients
OBJECTIVES:
Primary
- Determine the efficacy of sirolimus, in terms of objective response rate, in patients
with angiomyolipoma of the kidney secondary to tuberous sclerosis (TSC) or
lymphangioleiomyomatosis (LAM).
- Determine the toxicity of this drug in these patients.
Secondary
- Determine changes in other TSC lesions (e.g., tubers, subependymal giant cell
astrocytomas, facial angiofibromas, or kidney cysts) in patients with TSC treated with
this drug.
- 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
toxicity.
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
months.
Inclusion Criteria
DISEASE CHARACTERISTICS:
- Diagnosis of angiomyolipoma of the kidney secondary to tuberous sclerosis (TSC) or
lymphangioleiomyomatosis (LAM)*
- Tumor ≥ 2 cm by MRI NOTE: *Diagnosis of LAM by chest CT scan
- No angiomyolipoma-related bleeding or impending bleed
- No evidence of severe LAM, defined as dependence on continuous oxygen
- Untreated renal cell carcinoma
PATIENT CHARACTERISTICS:
Age
- 18 to 65
Performance status
- Not specified
Life expectancy
- Not specified
Hematopoietic
- Hematocrit > 27%
- ANC > 1,500
- Platelet count > 100,000
Hepatic
- SGOT and SGPT < 2 times normal
- Bilirubin < 2 times normal
- Alkaline phosphatase < 2 times normal
Renal
- eGFR 30 or higher
- No evidence of accelerating renal dysfunction
- No acute renal failure
Cardiovascular
- No history of coronary artery disease
Pulmonary
- See Disease Characteristics
Other
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective barrier contraception
- No unstable seizures, defined as a recent change in seizure pattern or change in
antiepileptic drug regimen
- No active infection
PRIOR CONCURRENT THERAPY:
Biologic therapy
- Not specified
Chemotherapy
- Not specified
Endocrine therapy
- Not specified
Radiotherapy
- Not specified
Surgery
- Not specified
Other
- More than 30 days since prior investigational agents
- More than 6 months since prior vascular embolization therapy for treatment of kidney
angiomyolipomas
- No concurrent chronic use of diltiazem, ketoconazole, or rifampin
- No other concurrent investigational agents
Type of Study:
Interventional
Study Design:
Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
Objective response as assessed by RECIST criteria
Safety Issue:
No
Principal Investigator
Sandra Dabora, MD, PhD
Investigator Role:
Study Chair
Investigator Affiliation:
Dana-Farber/Brigham and Women's Cancer Center
Authority:
United States: Federal Government
Study ID:
CDR0000440080
NCT ID:
NCT00126672
Start Date:
June 2005
Completion Date:
Related Keywords:
- Nonmalignant Neoplasm
- angiomyolipoma
- Neoplasms
- Angiomyolipoma
Name | Location |
|---|---|
| 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 |
