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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.
ClinicalTrials.gov 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)

Brief Summary:

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.

Condition or disease Intervention/treatment Phase
Nonmalignant Neoplasm Drug: sirolimus Phase 2

Detailed Description:



  • 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.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 36 participants
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Multi-Center Study of Rapamycin for Treating Kidney Angiomyolipomas in TSC or LAM Patients
Study Start Date : June 2005
Estimated Primary Completion Date : April 2010

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Primary Outcome Measures :
  1. Objective response as assessed by RECIST criteria
  2. Toxicity as measured by NCI CTC

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00126672

United States, California
Tuberous Sclerosis Clinic at Loma Linda University Medical Center
Loma Linda, California, United States, 92350
United States, Connecticut
Connecticut Children's Medical Center
Hartford, Connecticut, United States, 06106
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
United States, New York
New York University Medical Center
New York, New York, United States, 10016
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229-3039
United States, Texas
University of Texas Southwestern Medical Center at Dallas
Dallas, Texas, United States, 75390
Sponsors and Collaborators
Dana-Farber Cancer Institute
National Cancer Institute (NCI)
Study Chair: Sandra Dabora, MD, PhD Dana-Farber/Brigham and Women's Cancer Center

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Sandra Dabora, Dana-Farber/Brigham and Women's Cancer Center
ClinicalTrials.gov Identifier: NCT00126672     History of Changes
Other Study ID Numbers: CDR0000440080
First Posted: August 4, 2005    Key Record Dates
Last Update Posted: February 2, 2010
Last Verified: October 2008

Keywords provided by National Cancer Institute (NCI):

Additional relevant MeSH terms:
Neoplasms, Adipose Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Perivascular Epithelioid Cell Neoplasms
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs