This trial is active, not recruiting.

Condition nonmalignant neoplasm
Treatment sirolimus
Phase phase 2
Target mTOR
Sponsor Dana-Farber Cancer Institute
Collaborator National Cancer Institute (NCI)
Start date June 2005
End date April 2010
Trial size 36 participants
Trial identifier NCT00126672, CDR0000440080, DFCI-04298, WYETH-C-0468H1-101899


RATIONALE: Drugs used in chemotherapy, such as sirolimus, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.

PURPOSE: This phase II trial is studying how well sirolimus works in treating patients with angiomyolipoma of the kidney.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Masking open label
Primary purpose treatment

Primary Outcomes

Objective response as assessed by RECIST criteria
time frame:
Toxicity as measured by NCI CTC
time frame:

Eligibility Criteria

Male or female participants from 18 years up to 65 years old.

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

Additional Information

Official title A Phase II Multi-Center Study of Rapamycin for Treating Kidney Angiomyolipomas in TSC or LAM Patients
Description 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.
Trial information was received from ClinicalTrials.gov and was last updated in February 2010.
Information provided to ClinicalTrials.gov by National Cancer Institute (NCI).