Sirolimus in Treating Patients With Angiomyolipoma of the Kidney

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00126672
Recruitment Status : Unknown
Verified October 2008 by National Cancer Institute (NCI).
Recruitment status was:  Active, not recruiting
First Posted : August 4, 2005
Last Update Posted : February 2, 2010
National Cancer Institute (NCI)
Information provided by:
National Cancer Institute (NCI)

August 2, 2005
August 4, 2005
February 2, 2010
June 2005
April 2010   (Final data collection date for primary outcome measure)
  • Objective response as assessed by RECIST criteria
  • Toxicity as measured by NCI CTC
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Complete list of historical versions of study NCT00126672 on Archive Site
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Sirolimus in Treating Patients With Angiomyolipoma of the Kidney
A Phase II Multi-Center Study of Rapamycin for Treating Kidney Angiomyolipomas in TSC or LAM Patients

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.



  • 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.


  • 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.

Phase 2
Masking: None (Open Label)
Primary Purpose: Treatment
Nonmalignant Neoplasm
Drug: sirolimus
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*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Unknown status
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April 2010   (Final data collection date for primary outcome measure)


  • 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



  • 18 to 65

Performance status

  • Not specified

Life expectancy

  • Not specified


  • Hematocrit > 27%
  • ANC > 1,500
  • Platelet count > 100,000


  • SGOT and SGPT < 2 times normal
  • Bilirubin < 2 times normal
  • Alkaline phosphatase < 2 times normal


  • eGFR 30 or higher
  • No evidence of accelerating renal dysfunction
  • No acute renal failure


  • No history of coronary artery disease


  • See Disease Characteristics


  • 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


Biologic therapy

  • Not specified


  • Not specified

Endocrine therapy

  • Not specified


  • Not specified


  • Not specified


  • 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
Sexes Eligible for Study: All
18 Years to 65 Years   (Adult)
Contact information is only displayed when the study is recruiting subjects
United States
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Sandra Dabora, Dana-Farber/Brigham and Women's Cancer Center
Dana-Farber Cancer Institute
National Cancer Institute (NCI)
Study Chair: Sandra Dabora, MD, PhD Dana-Farber/Brigham and Women's Cancer Center
National Cancer Institute (NCI)
October 2008

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP