Proton Radiation Therapy (RT) for the Treatment of Pediatric Rhabdomyosarcoma

A Phase II Trial of Proton RT for the Treatment of Pediatric Rhabdomyosarcoma

The main purpose of this study is to see if using proton beam radiation therapy instead of photon beam radiation therapy can reduce side effects from radiation treatment for rhabdomyosarcoma. Photon beam radiation is the standard type of radiation for treating most rhabdomyosarcoma and many other types of cancer. Photon beam radiation enters the body and passes through healthy tissue, encounters the tumor, then leaves the body through healthy tissue. A beam of proton radiation enters the body and passes through healthy tissue, encounters tumor, but then stops. This means that less healthy tissue is affected by proton beam radiation than by photon beam radiation.

Study Overview

• Status: Completed
• Conditions: Rhabdomyosarcoma
• Intervention / Treatment: Radiation: Proton Beam Radiation

Detailed Description

• A special device is made for each participant to help them hold still during the treatment. This may be in the form of a mask or a custom made foam cradle depending on the area to be treated.
• Radiation treatments will be given once per day, 5 days a week for a total of 4 to 6 weeks, depending upon how much total dose the tumor requires.
• Participants will be seen once per week by their radiation doctor to monitor health and record any side effects from treatment.
• After the radiation treatments are completed, participants will be required to undergo further tests and evaluations for several years following treatment.

• Study Type: Interventional
• Enrollment (Actual): 115
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Dana-Farber Cancer Institute
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
  • Texas
    • Houston, Texas, United States, 77030
      • University of Texas MD Anderson Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 21 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Patients with biopsy proven newly diagnosed rhabdomyosarcoma.
• Patients less than or equal to 30 years of age.
• Patients must be treated with a standardly accepted chemotherapy regimen.
• May not have metastatic disease unless aged 2-10 with embryonal histology.
• Must be willing to receive follow-up care for a minimum of five years after treatment at MGH and annual visits unless it is too difficult to return to MGH for follow-up care. In that event, they must be willing to have their outside medical information released to us to track the results.
• Timing of radiation must be according to the IRB protocol upon which the patient is treated within either 35 days of last chemotherapy or surgery.

Exclusion Criteria:

• Life expectancy of less than 2 years.
• Co-morbidities that would make the use of radiation too toxic to deliver safely, such as serious local injury or collagen vascular disease.
• Patients who are pregnant
• Previous treatment with radiation therapy.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Proton Beam Radiation	Radiation: Proton Beam Radiation; Once per day, 5 days a week for a total of 4 to 6 weeks.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cumulative Incidence (Estimated Percent of Participants) With Grade 3 or Higher Toxicity in Follow-up	Cumulative incidence of participants who experienced a toxicity defined as either grade 3, 4, or 5 after the completion of radiation therapy in pediatric patients with pediatric rhabdomyosarcomas. Late toxicity is scored on a yearly follow-up basis for at least five years. Late toxicities will be graded according to the Common Terminology Criteria for Adverse Events (CTCAE), Version 3.0. Cumulative incidence is shown at 5 years follow-up.	5 years from the start of radiation treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants Who Experienced an Acute Toxicity by Grade	Percentage of participants who experienced an acute toxicity following completion of radiation treatment by grade. Acute toxicity will be scored at weekly status check visits by the radiation oncology nurse or doctor. In addition, acute toxicity will be scored at least one time within the 3 months following completion of radiation therapy. The recommended evaluation time for acute toxicity after radiotherapy was 6 weeks following completion, but may be at any time during the 3 months after treatment. Acute toxicities are graded according to the Common Terminology Criteria for Adverse Events (CTCAE), Version 3.0. Toxicities are graded on a scale of 1 to 5. A higher grade indicates a worse outcome with 1 being mild, 4 being life-threatening, and 5 being death.	3 months from radiation treatment
Dosimetric Comparison	Comparison of dose distribution to tumor and surrounding normal structures using dose volume histograms (DVH) generated from the proton plan used to treat the patient and the photon plan generated for comparison purposes. Percent of normal tissue spared was calculated using the following equation: (100 x (mean x-ray dose - mean proton dose))/mean X-ray dose. No measure of dispersion was calculated for these estimates. The data is no longer available.	4 years from the start of radiation treatment
Local Control	Estimated survival probability (defined as being free of a local recurrence) was calculated using Grays formula for competing risks	5 years from the start of radiation therapy

Collaborators and Investigators

• Sponsor: Massachusetts General Hospital
• Collaborators: M.D. Anderson Cancer Center; National Cancer Institute (NCI); Dana-Farber Cancer Institute; Boston Children's Hospital; National Institutes of Health (NIH); Brigham and Women's Hospital
• Investigators: Principal Investigator: Torunn Yock, MD, Massachusetts General Hospital

Publications and helpful links

Helpful Links

• Preliminary results of a phase II trial of proton radiotherapy for pediatric rhabdomyosarcoma

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2004
• Primary Completion (Actual): August 1, 2010
• Study Completion (Actual): August 1, 2010

Study Registration Dates

• First Submitted: December 28, 2007
• First Submitted That Met QC Criteria: December 28, 2007
• First Posted (Estimated): January 14, 2008

Study Record Updates

• Last Update Posted (Actual): June 18, 2025
• Last Update Submitted That Met QC Criteria: June 16, 2025
• Last Verified: June 1, 2025

More Information

Terms related to this study

• Keywords: pediatric rhabdomyosarcoma; proton radiation therapy
• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Sarcoma; Neoplasms, Connective and Soft Tissue; Neoplasms, Muscle Tissue; Myosarcoma; Rhabdomyosarcoma
• Other Study ID Numbers: 04-188; P01CA021239 (U.S. NIH Grant/Contract)