Proton Radiotherapy for Pediatric Brain Cancer

No longer recruiting at 1 trial location

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Massachusetts General Hospital

Disqualifiers: Prior radiotherapy, Metastases, Pregnancy, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests proton radiotherapy, a type of radiation treatment, to determine if it can better target brain tumors in children and young adults while causing fewer side effects than standard radiation. Proton therapy aims to spare more healthy tissue by focusing the radiation precisely on the tumor. The trial includes participants aged 1 to 25 with specific brain tumors, such as gliomas or medulloblastomas, who have not received previous radiation at the treatment site. Participants should not have distant cancer spread and must be able to engage in neurocognitive assessments in English or Spanish. As a Phase 2 trial, this research measures how well the treatment works in an initial, smaller group, offering participants a chance to contribute to advancements in targeted cancer therapies.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What prior data suggests that proton radiotherapy is safe for pediatric brain cancer?

Research has shown that proton radiotherapy is generally safe for treating brain tumors in children. Studies have found that it controls tumors and helps children survive as effectively as traditional radiation, but with fewer side effects. For instance, one study found that children treated with proton therapy had a low risk of serious brain issues. This indicates that proton therapy is less likely to damage healthy brain tissue compared to traditional radiation. Additionally, proton therapy has been used for other types of childhood cancers, demonstrating better long-term results and fewer side effects. Overall, this suggests that proton radiotherapy is a well-tolerated option for children with brain cancer. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for brain cancer?

Proton radiotherapy is unique because it targets pediatric brain cancer with remarkable precision. Unlike traditional radiation therapy, which often affects surrounding healthy tissues, proton radiotherapy delivers high doses of radiation directly to the tumor with minimal impact on nearby areas. This precision reduces the risk of side effects, which is crucial for young patients whose brains are still developing. Researchers are excited about this treatment because it holds the potential to improve outcomes while preserving the quality of life for children battling brain cancer.

What evidence suggests that proton radiotherapy might be an effective treatment for pediatric brain cancer?

A previous study showed that proton radiotherapy effectively treats brain tumors in children. Research indicates that 5-year survival rates for patients with certain brain tumors, such as ependymomas, reached 81% with this treatment. Studies have also found that proton therapy can improve cognitive skills, such as IQ, in children with brain cancer. Proton therapy targets the tumor more precisely and protects healthy tissue better than standard radiation. By reducing harm to normal tissues, proton therapy may lessen both immediate and long-term side effects. ^{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 aged 1-25 with certain brain tumors (like high-grade glioma, but not GBM, or non-disseminated ATRT) who haven't had radiotherapy in the same area before. They should have a life expectancy over a year and be willing to use contraception if applicable. It's not for those with metastases, uncontrolled illnesses that could affect participation, pregnant/breastfeeding women, or those unable to contribute to neurocognitive outcomes.

Inclusion Criteria

I have a confirmed diagnosis of a specific brain tumor type.

I have a high-grade glioma (not GBM) or ATRT with complete surgery and no spread.

I am between 1 and 25 years old.

Exclusion Criteria

I have cancer that has spread to my spine or other distant areas.

I do not have any illnesses that would stop me from following the study's requirements.

I have received radiotherapy on the area that needs treatment.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Proton Radiotherapy

Participants receive proton radiotherapy 5 days per week at the Francis H. Burr Proton Therapy Center. The duration depends on tumor type, location, and treatment tolerance.

Weekly physical exams during treatment

Follow-up

Participants are monitored for neurocognitive, neuroendocrine, and ototoxicity outcomes, as well as disease control and auditory function.

5 years

What Are the Treatments Tested in This Trial?

Interventions

• Proton radiotherapy

Trial Overview The study tests proton radiotherapy on pediatric brain tumors needing partial brain irradiation. Proton therapy targets tumors more precisely than standard radiation, potentially sparing healthy tissue and reducing side effects. The effectiveness of this technique compared to conventional methods is being evaluated.

How Is the Trial Designed?

Group I: Proton Radiotherapy Experimental Treatment 1 Intervention

Proton Radiotherapy

Who Is Running the Clinical Trial?

Massachusetts General Hospital

Lead Sponsor

Trials

3,066

Recruited

13,430,000+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

Dana-Farber Cancer Institute

Collaborator

Trials

1,128

Recruited

382,000+

Published Research Related to This Trial

Proton radiotherapy offers a safer treatment option for pediatric cancers by effectively sparing normal tissues, which helps reduce both acute and long-term side effects.

Over the past decade, more than 30 proton therapy facilities have opened in the U.S., allowing approximately 3,000 children annually to access this advanced treatment, with ongoing research to compare its efficacy and toxicity against traditional photon radiotherapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The role of proton therapy in pediatric malignancies: Recent advances and future directions. Greenberger, BA., Yock, TI.[2020]

Proton beam therapy, while having a better overall side effects profile compared to photon radiation therapy, is associated with a higher incidence of moyamoya syndrome, nearly double that of photon therapy, particularly in pediatric patients.

Children under 5 years old who receive proton beam therapy for brain tumors are at a greater risk of developing moyamoya syndrome, with most diagnoses occurring within the first 2 years after treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The incidence of radiation-induced moyamoya among pediatric brain tumor patients who received photon radiation versus those who received proton beam therapy: a systematic review. Elkatatny, A., Ismail, M., Ibrahim, KMM., et al.[2023]

In a study of 294 pediatric patients treated with proton beam therapy (PBT) for intracranial tumors, the 3-year overall survival rate was 82.7%, indicating promising long-term outcomes for this treatment.

While PBT showed good efficacy, 17 patients experienced grade 3 adverse events, including two cases of vision loss, highlighting the need for careful monitoring of side effects during long-term follow-up.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Proton Beam Therapy for Pediatric Tumors of the Central Nervous System-Experiences of Clinical Outcome and Feasibility from the KiProReg Study. Peters, S., Frisch, S., Stock, A., et al.[2022]

Proton radiation therapy provides similar tumor control as traditional photon radiation therapy for pediatric brain tumors, but with potentially fewer long-term side effects.

The superior physical properties of proton beams make them less likely to cause late effects, offering a safer treatment option for children undergoing radiation therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

New technologies in radiation therapy for pediatric brain tumors: the rationale for proton radiation therapy. Kirsch, DG., Tarbell, NJ.[2009]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12554329/

Therapeutic Outcomes and Toxicity Mitigation of Proton Beam ... Overall, the included studies provide strong evidence supporting proton therapy as an effective treatment for pediatric brain neoplasms, ...

2. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0967586824005162

Evaluating the safety and efficacy of proton radiotherapy for ... Proton radiotherapy is effective for pediatric intracranial ependymomas. 5-year overall survival reached 81% in treated patients.

3. journals.plos.org journals.plos.org/plosone/article?id=10.1371/journal.pone.0318194

Proton or photon? Comparison of survival and toxicity of two ... 3. Neurocognitive domains, for example IQ level, can be increased by using proton radiotherapy at children with brain cancers (MD: 11.91, 95% CI: 1.91–21.90).

4. advancesradonc.org advancesradonc.org/article/S2452-1094(23)00102-1/fulltext

The Pediatric Proton and Photon Therapy Comparison ... The physical properties of protons lower doses to surrounding normal tissues compared with photons, potentially reducing acute and long-term ...

5. link.springer.com link.springer.com/article/10.1007/s12519-023-00726-6

Neurocognitive outcomes in pediatric brain tumors after ... Pediatric brain tumor patients who receive PBRT achieve significantly higher scores on most neurocognitive outcomes than those who receive XRT.

6. mayoclinic.org mayoclinic.org/medical-professionals/pediatrics/news/proton-beam-therapy-for-pediatric-cancers/mac-20576795

Proton beam therapy for pediatric cancers Proton beam therapy is transforming pediatric cancer care, with improved outcomes and reduced long-term side effects.

7. mdpi.com mdpi.com/2072-6694/16/21/3655

Brainstem Toxicity Following Proton Beam Radiation ... This study revealed a low incidence of symptomatic brainstem toxicity and related mortality among pediatric brain tumor patients undergoing proton beam ...