IRB Number:

06-009658

Trial Status:

Open for Enrollment

Phase: III

Why is this study being done?

The current standard treatment for rhabdomyosarcoma (RMS) is surgery to remove as much tumor as possible, chemotherapy (anti-drug therapy) with vincristine, dactinomycin and cyclophosphamide (together called VAC therapy), and, for almost all tumors, radiation therapy (treatment with high energy X-rays).

Who is Eligible to Participate in the Study?

-Ages: under 50 years old
-Newly diagnosed embryonal rhabdomyosarcoma (RMS), botryoid or spindle cell variants of embryonal RMS, ectomesenchymoma, or alveolar RMS

*More specific, detailed eligibility and/ or exclusion criteria are associated with this trial.

What is Involved With this Study?

Subjects (people participating in the study) will get one of two different treatment plans. The treatment plan they get is decided by a process called randomization. Randomization means that the treatment is assigned based on chance. It is a lot like flipping a coin, except that it is done by computer to make sure that there are about the same number of people on each treatment plan of the study. Patients have an equal chance of being assigned to either treatment plan. A randomized study is the best way to find out if one treatment is better than another.

Some participants will be randomized to get Regimen A (which is VAC alone) and some will be randomized to get Regimen B (which is VAC plus VI). Some subjects may also have more surgery if it becomes possible to remove more tumor.

Supportive Care Drugs:
Drugs called growth factors (Filgrastim or Sargramostim) are given to help the blood counts recover from the chemotherapy. A drug called MESNA is given to help protect the bladder from bleeding during therapy with cyclophosphamide.

Standard tests and procedures
The following tests and procedures are part of regular cancer care and may be done even if the patient does not join the study.
- Frequent labs to monitor blood counts and blood chemistries
- Urine tests to measure how the kidneys are functioning
- Pregnancy test before treatment begins for females of childbearing age
- X-rays and scans to monitor response to treatment.
- Bone Marrow Aspiration and Biopsy to see if there are any cancer cells present
- Spinal taps (for cancers near the brain)
- Lymph node biopsy (for cancer in the arms or legs or the testicle of boys more than 10 years of age)

Subjects on this study will get a slightly lower dose of the standard drug cyclophosphamide.
For Regimen A subjects, therapy will be given on the same schedule as standard treatment with VAC. For Regimen B subjects, the experimental drug combination of irinotecan and vincristine is added.
For all patients on both arms of this study, radiation therapy will start at Week 4.
Evaluations (scans) to see how the tumor has changed will be done at Weeks 15, 30 and 43. If at any evaluation the patient's tumor is larger, they will be taken off protocol therapy and other treatment options will be discussed with the patient's doctor. While taking part in this research study, the patient's doctor may recommend having a special type of scan called a PET scan as part of the patient's regular care to help see their tumor better.

How long will the Study run?

Treatment on this study will last for about 43 weeks. After patients are done with treatment, the study doctor will ask them to return for follow-up exams every two to four months for four years and yearly for five to 10 years. The Children's Oncology Group would like to continue to find out information about the patient's health for up to ten years after the study closes to new patients. Keeping in touch with the patient and checking on how their health is every year for a while after the study closes helps researchers understand the long-term effects of the study.

Sponsor(s): Children's Oncology Group (COG)

Study Activation/Registration Date: 01/24/2007

IRB Review and Approval Date: 01/23/2007

Study Type: Treatment

Projected Accrual: The plan is to have up to 486 patients participate from multiple medical centers.

Costs of Study:There may be standard patient care costs related to participating in a cancer research study.

Principal Investigator: Carola Arndt, MD

Who can I Contact for Additional Information on this Trial?

Complete Request Form for Cancer Clinical Trial Information at Mayo Clinic or
For more information about this study or other clinical trial options, please contact our Mayo Clinic Cancer Center Clinical Trials Referral Office.

• Patients living within the United States, call 507-538-7623, or e-mail cancerclinicaltrials@mayo.edu


• Patients living outside the United States may call our International Office at 507-284-8884, or e-mail intl.mcr@mayo.edu.

What is/are the Locations of this Clinical Trial?

• Rochester, MN

Last updated: 05/08/2008