Proton Beam Radiation for Rhabdomyosarcoma

No longer recruiting at 2 trial locations

Overseen By Torunn Yock, MD

Age: < 65

Sex: Any

Trial Phase: Academic

Sponsor: Massachusetts General Hospital

Must be taking: Chemotherapy

Disqualifiers: Pregnancy, Previous radiation, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 5

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether proton beam radiation (also known as proton therapy) can reduce side effects compared to the usual photon beam radiation in treating rhabdomyosarcoma, a type of cancer. Proton beam radiation is believed to affect less healthy tissue, potentially leading to fewer side effects. It is suitable for patients up to 30 years old who have recently been diagnosed with rhabdomyosarcoma and are already receiving standard chemotherapy. Participants must commit to follow-up care for at least five years. As an unphased trial, this study offers patients the opportunity to contribute to research that could enhance future cancer treatments.

Do I have to stop taking my current medications for the trial?

The trial protocol does not specify whether you need to stop taking your current medications. However, you must be treated with a standardly accepted chemotherapy regimen.

Will I have to stop taking my current medications?

The trial protocol does not specify whether you need to stop taking your current medications. However, you must be treated with a standard chemotherapy regimen, so it's best to discuss your current medications with the trial team.

What prior data suggests that proton beam radiation is safe for treating rhabdomyosarcoma?

Research has shown that proton beam radiation is a safe treatment for rhabdomyosarcoma, a type of cancer that develops in soft tissue. Studies have found that patients generally tolerate this treatment well, experiencing fewer side effects than with the more common photon beam radiation.

One study discovered that proton beam therapy results in fewer immediate and long-term side effects, meaning patients experience less harm during and after treatment. Proton beams can more precisely target tumors, affecting less healthy tissue. Additionally, proton beam therapy is linked to better survival rates in patients who do not undergo surgery.

Overall, evidence suggests that proton beam radiation is a promising and safe option for treating rhabdomyosarcoma, with an emphasis on reducing unwanted side effects. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Proton Beam Radiation is unique because it uses a precise form of radiation therapy that targets tumors while minimizing damage to surrounding healthy tissue. Unlike traditional radiation therapy, which can affect both cancerous and healthy cells, proton beams can be controlled more accurately, potentially reducing side effects. Researchers are excited about this treatment for rhabdomyosarcoma because it offers the possibility of effective tumor control with fewer long-term complications, especially important for young patients whose bodies are still developing.

What evidence suggests that proton beam radiation is effective for reducing side effects in rhabdomyosarcoma?

Studies have shown that proton beam radiation, the treatment being tested in this trial, is a promising option for rhabdomyosarcoma, a type of cancer. Research indicates that this therapy effectively controls the tumor and improves survival rates. Specifically, after five years, 83% of patients had their cancer under control, 80% did not experience cancer progression, and 84% remained alive. Importantly, proton beam radiation affects less healthy tissue compared to traditional radiation, reducing side effects. This makes proton beam therapy a safer option with fewer long-term complications. ^{1 2 4 6 7}

Who Is on the Research Team?

Torunn Yock, MD

Principal Investigator

Massachusetts General Hospital

Are You a Good Fit for This Trial?

This trial is for children and young adults up to 21 years old with newly diagnosed rhabdomyosarcoma. They must be on a standard chemotherapy regimen and able to follow-up for five years post-treatment. It's not suitable for those with metastatic disease (except certain cases), previous radiation therapy, life-threatening co-morbidities, or if pregnant.

Inclusion Criteria

I am receiving a widely accepted chemotherapy treatment.

I am aged 2-10 with a specific type of cancer, allowing for metastatic disease.

Patients 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

Exclusion Criteria

I have health conditions that make radiation unsafe for me.

I have undergone radiation therapy before.

Life expectancy of less than 2 years

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Radiation Treatment

Participants receive proton beam radiation therapy once per day, 5 days a week for 4 to 6 weeks

4-6 weeks

5 visits per week (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, with further tests and evaluations for several years

4 years

What Are the Treatments Tested in This Trial?

Interventions

• Proton Beam Radiation

Trial Overview The study tests proton beam radiation against the standard photon beam radiation in treating pediatric rhabdomyosarcoma. Proton radiation aims to minimize damage to healthy tissue by stopping at the tumor site rather than passing through it.

How Is the Trial Designed?

Group I: Proton Beam Radiation Experimental Treatment 1 Intervention

Proton Beam Radiation

Proton Beam Radiation is already approved in European Union, United States, Japan, Canada for the following indications:

Approved in European Union as Proton Therapy for:

• Cancer of the brain and spinal cord
• Eye melanoma
• Head and neck cancers
• Breast cancer
• Lung cancer
• Liver cancer
• Pancreatic cancer
• Prostate cancer

Approved in United States as Proton Beam Therapy for:

• Brain tumors
• Eye melanoma
• Head and neck cancers
• Breast cancer
• Lung cancer
• Liver cancer
• Pancreatic cancer
• Prostate cancer
• Pediatric cancers

Approved in Japan as Proton Radiotherapy for:

• Brain tumors
• Eye melanoma
• Head and neck cancers
• Breast cancer
• Lung cancer
• Liver cancer
• Pancreatic cancer
• Prostate cancer

Approved in Canada as Proton Therapy for:

• Brain tumors
• Eye melanoma
• Head and neck cancers
• Breast cancer
• Lung cancer
• Liver cancer
• Pancreatic cancer
• Prostate cancer

Who Is Running the Clinical Trial?

Massachusetts General Hospital

Lead Sponsor

Trials

3,066

Recruited

13,430,000+

Brigham and Women's Hospital

Collaborator

Trials

1,694

Recruited

14,790,000+

National Institutes of Health (NIH)

Collaborator

Trials

2,896

Recruited

8,053,000+

Boston Children's Hospital

Collaborator

Trials

801

Recruited

5,584,000+

M.D. Anderson Cancer Center

Collaborator

Trials

3,107

Recruited

1,813,000+

Dana-Farber Cancer Institute

Collaborator

Trials

1,128

Recruited

382,000+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

Published Research Related to This Trial

Proton radiotherapy (PRT) for pediatric patients with rhabdomyosarcoma (RMS) showed promising overall survival rates of 91.9% at 1 year and 84.8% at 2 years, with particularly high survival rates in low-risk groups (100%).

While there were 153 adverse events, mostly hematologic toxicities, proton-specific toxicity was not observed, indicating that PRT has a tolerable safety profile similar to traditional photon radiotherapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Preliminary results of proton radiotherapy for pediatric rhabdomyosarcoma: a multi-institutional study in Japan. Mizumoto, M., Murayama, S., Akimoto, T., et al.[2022]

Proton radiation therapy (PRT) was successfully used in two patients with orbital rhabdomyosarcoma, resulting in both being disease-free after 2.5 to 3.4 years, with excellent visual acuity and minimal cosmetic side effects.

PRT demonstrated superior sparing of critical normal tissues, such as the lens and pituitary gland, while effectively targeting the tumor, which may reduce the risk of long-term complications and second malignancies in childhood cancer survivors.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fractionated, three-dimensional, planning-assisted proton-radiation therapy for orbital rhabdomyosarcoma: a novel technique. Hug, EB., Adams, J., Fitzek, M., et al.[2019]

Proton beam therapy is a viable option for urgent radiotherapy in children with parameningeal rhabdomyosarcoma that extends into the cranium, as demonstrated in a case series of 3 patients aged 6 to 12 years.

Timely transfer to facilities offering proton beam therapy can minimize delays in treatment, highlighting the importance of quick communication between hospitals when intracranial involvement is suspected.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Urgent Proton Beam Therapy With Interinstitutional Transfer for Patients With Intracranial Rhabdomyosarcoma: Report of 3 Cases. Inaba, M., Nakao, T., Hosaka, S., et al.[2021]

Rhabdomyosarcoma, the most common soft tissue sarcoma in children, has seen improved clinical outcomes due to enhanced treatment strategies developed by the Intergroup Rhabdomyosarcoma Study Group.

Advanced radiotherapy techniques, such as intensity-modulated radiotherapy and proton therapy, are being utilized to target tumors near critical structures, aiming to reduce radiation exposure and minimize long-term side effects while maintaining treatment efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Radiotherapy for rhabdomyosarcoma: indications and outcome. Terezakis, SA., Wharam, MD.[2012]

In a study of 57 children with rhabdomyosarcoma treated with proton radiotherapy and chemotherapy, the five-year local control rate was 81%, with event-free survival at 69% and overall survival at 78%, indicating effective disease management.

The treatment was associated with low toxicity, as no acute or late adverse effects exceeded grade 3, suggesting that proton radiation is a safe option for pediatric patients compared to traditional photon radiation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Preliminary results of a phase II trial of proton radiotherapy for pediatric rhabdomyosarcoma. Ladra, MM., Szymonifka, JD., Mahajan, A., et al.[2022]

Proton radiotherapy for children with parameningeal rhabdomyosarcoma (PM-RMS) resulted in a 5-year failure-free survival rate of 59% and an overall survival rate of 64%, indicating effective tumor control and survival outcomes comparable to historical controls.

The late side effects observed in patients treated with proton therapy, such as mild facial hypoplasia and dental issues, were fewer and less severe compared to those reported in patients treated with traditional photon therapy, suggesting a potentially safer profile for proton radiotherapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Proton radiotherapy for parameningeal rhabdomyosarcoma: clinical outcomes and late effects. Childs, SK., Kozak, KR., Friedmann, AM., et al.[2021]

In a study of 37 infants under 24 months with non-metastatic rhabdomyosarcoma treated with proton therapy, the 5-year local control, progression-free survival, and overall survival rates were 83%, 78%, and 83%, respectively, indicating promising outcomes for this age group.

Despite the young age being traditionally viewed as a negative prognostic factor, the results suggest that with appropriate local therapy, infants can achieve survival rates comparable to older children, although the classification of nasal ala as an unfavorable site may need reconsideration.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Proton radiotherapy for infant rhabdomyosarcoma: Rethinking young age as an adverse prognostic factor. Parekh, AD., Indelicato, DJ., Vega, RBM., et al.[2021]

Proton beam therapy (PBT) was safely and effectively used to treat a 1-year-old girl with stage IV alveolar rhabdomyosarcoma, resulting in a 95% reduction in tumor volume after treatment, despite the tumor's challenging location and previous resistance to chemotherapy.

This case highlights the potential of PBT as a viable treatment option for pediatric patients with tumors that are difficult to manage with conventional radiotherapy, particularly when there are concerns about surrounding organ tolerance.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Proton beam therapy for a patient with large rhabdomyosarcoma of the body trunk. Takizawa, D., Oshiro, Y., Mizumoto, M., et al.[2022]

In a study of 83 children with rhabdomyosarcoma treated with pencil beam scanning proton therapy, the 5-year overall survival rate was 80.6%, indicating effective treatment outcomes.

The treatment resulted in minimal late non-ocular toxicity (only 3.6% incidence of grade 3 toxicity) and improved quality of life scores after therapy, highlighting its safety profile.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tumour control and Quality of Life in children with rhabdomyosarcoma treated with pencil beam scanning proton therapy. Leiser, D., Calaminus, G., Malyapa, R., et al.[2022]

In a study of 94 pediatric patients with rhabdomyosarcoma treated with proton beam therapy (PBT) and chemotherapy, the 4-year overall survival rate was 71% and the progression-free survival rate was 63%, indicating that PBT is an effective treatment option.

The study found that larger tumor size and delayed delivery of radiation therapy negatively impacted local control, progression-free survival, and overall survival, suggesting that timely treatment and possibly higher doses for larger tumors may improve outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Disease Control and Patterns of Failure After Proton Beam Therapy for Rhabdomyosarcoma. Buszek, SM., Ludmir, EB., Grosshans, DR., et al.[2021]

In a study of 66 children with nonmetastatic rhabdomyosarcoma treated with proton therapy, the 2-year local control rate was 88%, indicating that proton therapy is effective in managing this type of cancer.

All recurrences occurred within the treated radiation field, suggesting that the precise targeting of proton therapy does not increase the risk of marginal failures, even in patients with unfavorable risk features.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Patterns of Failure in Pediatric Rhabdomyosarcoma After Proton Therapy. Vern-Gross, TZ., Indelicato, DJ., Bradley, JA., et al.[2017]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9942395/

Efficacy and safety of proton beam therapy for ... The data showed that PBT is a feasible, safe, and effective modality for RMS, showing promising LC, OS, PFS, and lower acute and late toxicities ...

2. link.springer.com link.springer.com/article/10.1007/s10147-025-02794-2

Systematic review and meta-analysis of photon ... Proton beam therapy (PBT) is currently utilized due to its potential to reduce long-term complications; however, data on its impact on tumor ...

3. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0360301620300110

Outcomes Following Proton Therapy for Group III Pelvic ... With a median follow-up of 4.2 years, the 5-year local control, progression-free survival, and overall survival rates were 83%, 80%, and 84%, respectively.

4. frontiersin.org frontiersin.org/journals/oncology/articles/10.3389/fonc.2025.1490098/full

Proton beam therapy for a patient with prostatic ... The combination of radiotherapy and immunotherapy has demonstrated significant synergistic effects, enhancing both local and systemic tumor ...

5. ro-journal.biomedcentral.com ro-journal.biomedcentral.com/articles/10.1186/s13014-023-02223-6/tables/4

Table 4 Survival outcomes, toxicity incidence and prognostic ... This study aimed to evaluate and conduct a meta-analysis on the efficacy and safety of proton beam therapy (PBT) for rhabdomyosarcoma (RMS).

6. frontiersin.org frontiersin.org/journals/oncology/articles/10.3389/fonc.2025.1644829/full

Proton beam radiation therapy vs. photon ... Our study found that, among patients who did not undergo surgery, proton beam therapy (PBT) was associated with improved overall survival ...

7. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0360301620343522

Disease Control and Patterns of Failure After Proton Beam ... We report disease control and patterns of failure for pediatric RMS patients across all anatomic sites treated with PBT at our institution.