Clinical Trial Regarding MPNSTs

SARC Clinical Trial for People with Malignant Peripheral Nerve Sheath Tumors (MPNST)

SARC (Sarcoma Alliance for Research through Collaboration), a not-for-profit consortium that facilitates clinical trials involving sarcomas, is conducting a clinical trial for people with MPNSTs. Dr. Brigitte Widemann, Investigator, Pediatric Oncology Branch, National Cancer Institute, is the Principal Investigator for this SARC clinical trial that is exploring treatment of patients with malignant peripheral nerve sheath tumors (MPNST).

Trial Background
MPNST is a type of soft tissue sarcoma (a type of cancer) that develops in cells that form a protective covering (sheath) around peripheral nerves. Peripheral nerves are those that are outside of the central nervous system (brain and spinal cord). These tumors most commonly occur in the arms and legs. However, they can also arise in the abdomen as well as other locations. This tumor can be associated with a condition called neurofibromatosis type 1 (NF1).

Neurofibromatosis type 1 is a genetic disorder that primarily affects the development and growth of nerve tissues. This disorder can cause tumors to grow on the nerves and produce other abnormalities such as skin changes and bone deformities, and can affect other organ systems.

MPNSTs are rare in the general population, but occur with greater frequency in people who have NF1. MPNSTs comprise approximately 5-10% of all soft tissue sarcomas. Half of these will occur in people with NF1. The best treatment for MPNSTs is complete surgical resection. For MPNSTs that cannot be completely resected the question arises, if chemotherapy can help shrink the MPNST in order to make surgery feasible. It is not known, to date, how well MPNSTs respond to chemotherapy. There is some data to suggest that an MPNST that occurs in people with NF1 versus an MPNST that occurs in people who do not have NF1 may respond differently to treatment.

This study is designed to define the response to treatment with chemotherapy in MPNSTs, and to explore if there is a difference in response between people with an MPNST who have or do not have NF1.

Responses (tumor shrinkage) of MPNSTs to doxorubicin/ifosfamide and ifosfamide/etoposide chemotherapy have been reported suggesting that an MPNST may be responsive to standard chemotherapy drugs used to treat other sarcomas. The main goal of this study is to determine the clinical response rate of high grade, unresectable or metastatic MPNSTs, or potentially resectable MPNSTs not previously treated with chemotherapy for this malignancy. Response will be separately evaluated in people who have NF1 and do not have NF1. Children and adults with measurable high-grade, unresectable, or metastatic MPNSTs, or those with a potentially resectable MPNST for which neoadjuvant chemotherapy is determined to be the best treatment option are eligible to participate in this trial.

In this trial, combination chemotherapy of doxorubicin and ifosfamide and etoposide and ifosfamide will be given to consented, eligible and enrolled patients. These drugs are all Food and Drug Administration approved drugs for the treatment of various types of cancer. As part of this study, up to 4 cycles of each combination (total of 8 cycles) will be given. After the 4th cycle, patients may undergo local control measures at the discretion of their doctor, which could be surgery, radiation treatment, or both. Information (data) from patients with NF and MPNST will be compared with information (data) from patients who have MPNST but do not have NF1 to determine if their response to treatment is different.

Participation in the trial*
To be eligible to participate in this trial a person must:

1. Have confirmed diagnosis of MPNST (NF1 associated or sporadic)
2. Have not received previous treatment with chemotherapy for this MPNST
3. Must have disease which is measurable on imaging studies such as CT or MRI
4. Can be any age
5. Be active, able to perform light physical activity (such as light housework or office work)

People would NOT be able to participate in the study if they are pregnant or breast feeding females.

*This represents only a partial list of requirements to be included or excluded from the study. A complete list is available from SARC or Dr. Widemann.

For more information contact SARC at 734.930-7600, or Dr. Widemann at:, phone: 301-496-7387, fax: 301-480-8871

This trial is registered at  (clinical identifier: NCT0030483)

There are several sites across the United States conducting this trial.  To find a site near you, call SARC at 734-930-7600 or email:

The full title is: Phase II trial of chemotherapy in sporadic and neurofibromatosis type 1 associated high grade malignant peripheral nerve sheath tumors (SARC006)

Neoadjuvant therapy: treatment given before surgery is done

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