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Everolimus in Treating Patients With Stage IV Melanoma

This study has been completed.
Information provided by:
National Cancer Institute (NCI) Identifier:
First received: December 7, 2004
Last updated: June 17, 2012
Last verified: January 2007

December 7, 2004
June 17, 2012
April 2005
July 2007   (final data collection date for primary outcome measure)
Proportion of patients with progression-free disease at 16 weeks [ Designated as safety issue: No ]
Not Provided
Complete list of historical versions of study NCT00098553 on Archive Site
  • Median overall survival [ Designated as safety issue: No ]
  • Tumor response rate for 2 consecutive evaluations at least 8 weeks apart [ Designated as safety issue: No ]
  • Toxicity as measured by CTCAE v. 3.0 [ Designated as safety issue: Yes ]
  • Efficacy as measured by fludeoxyglucose F 18 uptake at baseline and at weeks 8 and 16 by positron-emission tomography imaging [ Designated as safety issue: No ]
  • Efficacy as measured by the intensity of tissue vascular endothelial growth factor (VEGF) based on percentage of tumor cells positive for stain and the intensity of staining and microvessel density at 8 weeks and at diseae progression [ Designated as safety issue: No ]
  • Efficacy as measured by serum VEGF levels at baseline, 8 weeks, and at disease progression [ Designated as safety issue: No ]
  • Effect of therapy on the immune system at 8 weeks [ Designated as safety issue: No ]
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Everolimus in Treating Patients With Stage IV Melanoma
Phase II Trial Of RAD-001 In Metastatic Malignant Melanoma

RATIONALE: Drugs used in chemotherapy, such as everolimus, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Everolimus may also stop the growth of melanoma by blocking blood flow to the tumor.

PURPOSE: This phase II trial is studying how well everolimus works in treating patients with stage IV melanoma.



  • Determine the median time to disease progression in patients with stage IV malignant melanoma treated with everolimus.


  • Determine the median overall survival of patients treated with this drug.
  • Determine the clinical benefit rates (i.e., stable disease, partial remission, and complete response rates) in patients treated with this drug.
  • Determine the toxicity profile of this drug in these patients.
  • Determine changes in serum vascular endothelial growth factor levels in patients treated with this drug.

OUTLINE: This is a multicenter study.

Patients receive oral everolimus once daily for 8 weeks. Courses repeat every 8 weeks in the absence of disease progression or unacceptable toxicity.

Patients are followed every 2 months until disease progression and then every 4 months for up to 5 years after registration.

PROJECTED ACCRUAL: A total of 73 patients will be accrued for this study.

Phase 2
Masking: Open Label
Primary Purpose: Treatment
Melanoma (Skin)
Drug: everolimus
Not Provided
  • Rao RD, Allred JB, Windschitl HE, et al.: N0377: results of NCCTG phase II trial of the mTOR inhibitor RAD-001 in metastatic melanoma. [Abstract] J Clin Oncol 25 (Suppl 18): A-8530, 479s, 2007.
  • Rao RD, Windschitl HE, Allred JB, et al.: Phase II trial of the mTOR inhibitor everolimus (RAD-001) in metastatic melanoma. [Abstract] J Clin Oncol 24 (Suppl 18): A-8043, 463s, 2006.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Not Provided
July 2007   (final data collection date for primary outcome measure)


  • Histologically confirmed malignant melanoma for which no known standard or potentially curative therapy exists or has been proven to extend life expectancy

    • Stage IV disease
  • Measurable disease

    • At least 1 lesion ≥ 20 mm by CT scan or MRI OR ≥ 10 mm by spiral CT scan
  • No intracranial disease



  • 18 and over

Performance status

  • ECOG 0-2

Life expectancy

  • At least 12 weeks


  • Absolute neutrophil count ≥ 1,500/mm^3
  • Platelet count ≥ 100,000/mm^3
  • Hemoglobin ≥ 9.0 g/dL
  • No bleeding diathesis


  • AST ≤ 3 times upper limit of normal (ULN)
  • Alkaline phosphatase ≤ 3 ULN
  • INR ≤ 1.5


  • Creatinine ≤ 1.5 times ULN


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • Willing to refrain from foods high in fat content
  • No uncontrolled infection
  • No immunosuppression from any cause (e.g., known HIV infection)
  • No other malignancy within the past 5 years except basal cell or squamous cell skin cancer treated with local resection only
  • No other severe condition that would preclude study participation or compliance


Biologic therapy

  • More than 4 weeks since prior immunotherapy or biologic therapy


  • More than 4 weeks since prior chemotherapy (6 weeks for nitrosoureas or mitomycin) and recovered
  • No prior sirolimus or its analogues for any indication
  • No other concurrent chemotherapy

Endocrine therapy

  • No concurrent steroids


  • More than 4 weeks since prior radiotherapy to head and neck area
  • More than 4 weeks since prior radiosurgery
  • No prior radiotherapy to > 30% of bone marrow
  • No concurrent radiotherapy


  • Not specified


  • At least 1 week since prior and no concurrent CYP3A4 inducers
  • No concurrent warfarin
  • No concurrent cytotoxic agents
  • No other concurrent experimental drugs
  • No other concurrent immunosuppressive therapy
18 Years and older
Contact information is only displayed when the study is recruiting subjects
United States
CDR0000402871, NCCTG-N0377
Not Provided
Not Provided
North Central Cancer Treatment Group
National Cancer Institute (NCI)
Study Chair: Ravi D. Rao, MD, MBBS Mayo Clinic
Investigator: Harold E. Windschitl, MD Coborn Cancer Center
Investigator: William J. Maples, MD Mayo Clinic
Investigator: Michael K. Gornet, MD Mayo Clinic
Investigator: James N. Ingle, MD Mayo Clinic
Investigator: Edward T. Creagan, MD Mayo Clinic
Investigator: Judith S. Kaur, MD Mayo Clinic
Investigator: Barbara A. Pockaj, MD Mayo Clinic Hospital
Investigator: Evanthia Galanis, MD Mayo Clinic
Investigator: Charles L. Loprinzi, MD Mayo Clinic
Investigator: Henry C. Pitot, MD Mayo Clinic
Investigator: Lori A. Erickson, MD Mayo Clinic
Investigator: Val J. Lowe, MD Mayo Clinic
National Cancer Institute (NCI)
January 2007

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