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ARST08P1 (Pilot 2) A Pilot Study to Evaluate Novel Agent Temozolomide in Combination with Intensive Multi-Agent Interval Compressed Therapy for Patients with High-Risk Rhabdomyosarcoma

Purpose of study:

The standard treatment for subjects with high risk rhabdomyosarcoma is surgery to remove the tumor (when possible), anti-cancer drugs (chemotherapy), and high energy x-rays (radiation therapy). The standard drugs used for rhabdomyosarcoma are vincristine, dactinomycin and cyclophosphamide, and are often called VAC therapy.

Current standard therapy does not work as well with tumors that have already spread at the time a subject is diagnosed. Past studies have been done using higher doses of cyclophosphamide but that has not increased the treatment success for high risk subjects. We chose the drug temozolomide because it has been shown to increase the effect of other anticancer drugs (chemotherapy) as well as high energy x-rays (radiation therapy). The combination of VAC and interval compression of Ifosfamide, etoposide, doxorubicin and irinotecan has been well-tolerated in children and adolescents with rhabdomyosarcoma, and other kinds of sarcomas.

It is not known if adding temozolomide to high intensity chemotherapy will be helpful for high risk rhabdomyosarcoma, and it may increase the side effects of treatment.
The overall goals of this study:
To find out the effects, good and/or bad, of giving high intensity chemotherapy containing temozolomide to subjects with high risk rhabdomyosarcoma or ectomesenchymoma.
To see if early exposure to high intensity chemotherapy containing temozolomide will get rid of the cancer for as long as possible for high risk rhabdomyosarcoma subject.

Who can participate:


<50 years of age at time of enrollment


Patients with newly diagnosed, biopsy-proven metastatic rhabdomyosarcoma or ectomesenchymoma.
Additional screening for eligibility will be required.

What will happen during the study:

The overall study has three different treatment plans, and each plan is called a 'Pilot'. Subjects are placed in a specific pilot based on when they enroll in the study. Currently, we are enrolling subjects in Pilot 2.

High intensity chemotherapy containing temozolomide that will be given on this study is experimental. It is not known how subjects with high risk rhabdomyosarcoma will do with this new approach to chemotherapy. It is not known if this combination (high intensity chemotherapy containing temozolomide) will get rid of the cancer for as long as possible.

Various methods will be used to give drugs:

  • PO - Drug is given by tablet or liquid swallowed through the mouth.
  • IV - Drug is given using a needle or tubing inserted into a vein. It can be given by IV push over several minutes or by infusion over minutes or hours.
  • SubQ - Drug is given by inserting a needle into the tissue just under the skin.

You will get some supportive care drugs to help with the effects of the chemotherapy. These drugs are commonly used with chemotherapy drugs. These drugs will be:

  • Myeloid growth factors (for example, filgrastim) to help your blood counts recover from some of the chemotherapy drugs,
  • Dexrazoxane, which helps protect your heart when you get doxorubicin,
  • Mesna, which helps protect your bladder from bleeding when you get ifosfamide or cyclophosphamide.

You will have additional tests and procedures during this study to assess your response to this treatment.

This study is expected to last 54 weeks. During this time, subjects in this clinical trial are expected to receive treatment on this pilot study for about 51 weeks. After treatment, subjects will have follow-up examinations and medical tests (3 weeks after the end of treatment and thereafter). We will continue to collect some medical information about how you are doing for 5 years after you start the study.?

Who to contact:

PI: Mark Ranalli, MD

Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000