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Sirolimus in Treating Patients With Angiomyolipoma of the Kidney

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified October 2008 by National Cancer Institute (NCI).
Recruitment status was:  Active, not recruiting
National Cancer Institute (NCI)
Information provided by:
National Cancer Institute (NCI) Identifier:
First received: August 2, 2005
Last updated: February 1, 2010
Last verified: October 2008

August 2, 2005
February 1, 2010
June 2005
April 2010   (final data collection date for primary outcome measure)
  • Objective response as assessed by RECIST criteria [ Designated as safety issue: No ]
  • Toxicity as measured by NCI CTC [ Designated as safety issue: Yes ]
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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: 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
18 Years to 65 Years   (Adult)
Contact information is only displayed when the study is recruiting subjects
United States
CDR0000440080, DFCI-04298, WYETH-C-0468H1-101899
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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