IRB Number:

155-05

Trial Status:

Open for Enrollment

Phase: III

Why is this study being done?

This study is being done to:
- Find out the most effective treatment with the least amount of therapy that will still cure patients with low risk rhabdomyosarcoma

Results of past research studies with low-risk rhabdomyosarcoma patients show that using the three chemotherapy medicines (including cyclophosphamide) helps to cure more patients, but cyclophosphamide at the high doses that were used in these past studies can cause late effects.

The goals of this study are to see if:
1. Lower doses of cyclophosphamide used together with current standard doses of vincristine and dactinomycin can be as effective or better in curing patients as using the standard doses of vincristine and dactinomycin
2. The length of treatment can be shortened

Who is Eligible to Participate in the Study?

-Under age 50
-Diagnosed with embryonal rhabdomyosarcoma (or a related type called botryoid or spindle cell
rhabdomyosarcoma or embryonal ectomesenchymoma) that has a low risk of coming back after treatment (this is called recurrence).

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

What is Involved With this Study?

Standard Medical Tests
Before treatment on this study begins, and while getting treatment, patients will have a series of standard medical tests:
- Physical exam
- Blood tests
- Urine tests
- Pregnancy test for women able to become pregnant
- Various scans (CT/MRI)

All patients will begin treatment with the drugs vincristine, dactinomycin, and cyclophosphamide (together called VAC). The doctors treating the patient will decide if they will get radiation therapy or surgery, or both, along with the chemotherapy. This phase of treatment is called local control.

If patients are assigned to Subset 1 they will get treatment on Regimen A. Patients will go on to have three more cycles of VAC treatment. At Week 12 the patient's response to treatment will be looked at. It may be decided by their treating doctors that they will get radiation therapy and/or surgery at Week 13; there may be a different schedule for surgery and/or radiation in some cases, but that will be decided by the doctors giving the treatment.

Chemotherapy will continue with four cycles of VA (VAC minus the cyclophosphamide) and then an evaluation at Week 24. This will be the end of treatment for patients on Regimen A.

If patients are assigned to Subset 2 they will get treatment on Regimen B. Patients will have three more cycles of VAC treatment and an evaluation at Week 12, with possible radiation therapy and/or surgery starting at Week 13 also. However, Regimen B patients will continue with up to a total of twelve cycles of VA treatment (with periodic evaluations) and a final evaluation at Week 48.

If the tumor gets bigger while the patient is on VAC or VA treatment, at any of the evaluations, they will be taken off study treatment. The doctors will talk to the patient about other treatments.

Methods for Giving Drugs
Drugs on this study will be given by needle put into a vein (intravenous or IV).
Dactinomycin - given IV over one minute
Cyclophosphamide - given IV over 30-60 minutes (patients are advised to empty their bladder every 2 hours for the 12 hours following the cyclophosphamide dose)
Vincristine - given IV push over one minute
MESNA - given IV with cyclophosphamide to prevent bladder irritation and bleeding in the urine

Radiation Therapy
Some patients on this study will have radiation therapy.

Surgery
Some patients on this study may have surgery.

How long will the Study run?

Patients will be in the study for about 24 weeks if they are treated on Regimen A, and about
48 weeks if treated on Regimen B. However, patients will continue to have physical exams and blood tests for a few years after treatment so that researchers can keep on looking for any side effects of treatment. In addition, the research staff would like to collect some information about how the patient is doing for about ten years after the study closes to new patients.

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

Study Activation/Registration Date: 02/24/2005

IRB Review and Approval Date: 01/28/2005

Study Type: Treatment

Projected Accrual: The plan is to have up to 360 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: 05/07/2008