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Phase 2 Study of the mTOR Inhibitor Everolimus in Combination With Bevacizumab in Patients With Sporadic and Neurofibromatosis Type 1 (NF1) Related Refractory Malignant Peripheral Nerve Sheath Tumors

Phase 2
18 Years
Open (Enrolling)
Malignant Peripheral Nerve Sheath Tumors, MPNST, Sarcoma

Thank you

Trial Information

Phase 2 Study of the mTOR Inhibitor Everolimus in Combination With Bevacizumab in Patients With Sporadic and Neurofibromatosis Type 1 (NF1) Related Refractory Malignant Peripheral Nerve Sheath Tumors

Inclusion Criteria:

- Patients 18 or older

- Unresectable or metastatic sporadic or NF1 associated high-grade MPNST

- Experienced progression after one or more prior regimens of cytotoxic chemotherapy

- Patients must be able to swallow tablets

- Patients must have measurable disease, defined as at least one tumor that is

- Patients who develop a recurrence or progression (WHO criteria) of an MPNST in a
previously radiated field may be enrolled if it has been at least 4 weeks since the
last dose of radiation therapy

- Patients must have recovered from the toxic effects of all prior therapy before
entering this study

- Adequate organ function

- ECOG performance status of 0, 1, or 2

- Patents who received an anthracycline prior to enrollment must have an ejection
fraction ≥ 50%

- Subjects of childbearing potential requires acceptable form of birth control

- Informed consent

Exclusion Criteria:

- Patients currently receiving anticancer therapies or who have received anticancer
therapies within 3 weeks of the start of study drug or patients receiving prior
treatment with investigational drugs 4 weeks of the start of study drug

- Patients may not be currently receiving strong inhibitors of CYP3A4, and may not have
received these medications within 1 week of entry

- Prior radiotherapy within 4 weeks of the start of study drug

- Patients who have had a major surgery or significant traumatic injury within 4 weeks
of start of study drug,

- Patients who have not recovered from the side effects of any major surgery

- Patients that may require major surgery during the course of the study

- Less than 7 days have passed from core biopsies or other minor surgical procedures
excluding placement of a vascular access device

- Patients receiving chronic, systemic treatment with corticosteroids or another
immunosuppressive agent(Topical or inhaled corticosteroids are allowed)

- Uncontrolled brain or leptomeningeal metastases, including patients who continue to
require glucocorticoids for brain or leptomeningeal metastases

- Other malignancies within the past 3 years except for adequately treated carcinoma of
the cervix or basal or squamous cell carcinomas of the skin

- Patients who have any severe and/or uncontrolled medical conditions or other
conditions that could affect their participation in the study

- Female patients who are pregnant or breast feeding

- Patients who have received prior treatment with an mTOR inhibitor or bevacizumab

- Patients with known hypersensitivity to rapamycins

- concurrent use of anti-coagulant drugs

- Patients using Seville orange, star fruit, grapefruit and their juices, and St.
John's Wort

- Patients taking enzyme inducing anticonvulsants

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Evaluation of lesions for progression or response via Who criteria

Outcome Time Frame:

great than or equal to 4 months

Safety Issue:


Principal Investigator

Brigitte C. Widemann, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

National Cancer Institute (NCI)


United States: Food and Drug Administration

Study ID:




Start Date:

September 2012

Completion Date:

September 2016

Related Keywords:

  • Malignant Peripheral Nerve Sheath Tumors
  • Sarcoma
  • malignant peripheral nerve sheath tumors
  • RAD001
  • Everolimus
  • Bevacizumab
  • Sarcoma
  • Avastin
  • mTOR inhibitor
  • Neurofibromatosis 1
  • Neurofibromatoses
  • Nerve Sheath Neoplasms
  • Neurofibrosarcoma
  • Neurilemmoma
  • Sarcoma



University of Iowa Iowa City, Iowa  52242
University of Michigan Ann Arbor, Michigan  48109-0624
Children's Hospital of Philadelphia Philadelphia, Pennsylvania  19104
Children's National Medical Center Washington, District of Columbia  20010-2970
Cincinnati Children's Hospital Medical Center Cincinnati, Ohio  45229-3039
Pennsylvania Oncology Hematology Associates Philadelphia, Pennsylvania  19107
University of Alabama at Birmingham Birmingham, Alabama  35294-3300
Johns Hopkins Baltimore, Maryland  21231
Children's Hospital of Boston Boston, Massachusetts  02115
Washington University in St. Louis St. Louis, Missouri  63110
National Cancer Institute Bethesda, Maryland  20892-1922
Ann and Robert Lurie Children's Hospital of Chicago Chicago, Illinois  60611