Proton Radiation Therapy for Neuroblastoma

Overseen By Shannon M MacDonald, MD

Age: < 65

Sex: Any

Trial Phase: Academic

Sponsor: Massachusetts General Hospital

Disqualifiers: Prior radiation, Recent chemotherapy, Pregnancy, others

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

Approved in 6 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 3

Duration: 3-4 weeks

30 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a type of radiation treatment called proton beam radiation therapy (PBRT) for individuals with neuroblastoma, a cancer that often affects nerve tissue around the adrenal glands. PBRT targets tumors more precisely, reducing radiation exposure to healthy tissues and potentially lowering side effects compared to standard treatments. Suitable participants should have neuroblastoma or related conditions and should not have received significant prior radiation therapy. As an unphased trial, this study provides patients the opportunity to contribute to pioneering research that could enhance future treatment options.

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 beam radiation therapy is safe for treating neuroblastoma?

Research has shown that proton beam radiation therapy (PBRT) is generally safe and well-tolerated in children with neuroblastoma. In one study, after three years, 97.7% of the treated areas showed no cancer growth, and 90.9% of the children remained alive. These results indicate that the treatment is both effective and safe.

Proton beam therapy reduces long-term side effects by targeting the tumor precisely, thus protecting nearby healthy tissues and organs. This precision is particularly beneficial for children, as it minimizes the risk of damaging healthy, growing tissues.

Overall, evidence suggests that PBRT is a promising and safer option for treating neuroblastoma in children compared to traditional radiation methods. It effectively controls cancer while causing fewer side effects. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Proton Beam Radiation Therapy (PBRT) is unique because it specifically targets tumors with high precision, which helps spare surrounding healthy tissues from damage. Unlike traditional radiation therapies, which use X-rays, PBRT uses protons that can be controlled more precisely, making it particularly promising for treating sensitive areas in neuroblastoma patients. Researchers are excited about PBRT because it has the potential to reduce side effects and improve outcomes for young patients, offering a more focused approach compared to conventional methods.

What evidence suggests that proton beam radiation therapy is effective for neuroblastoma?

Studies have shown that proton beam radiation therapy (PBRT), which participants in this trial will receive, effectively treats high-risk neuroblastoma. In one study, three years after treatment, 97.7% of patients had their cancer controlled at the original site, and 90.9% were still alive. PBRT precisely targets tumors, protecting nearby healthy tissues and reducing side effects compared to traditional radiation. This precision makes it a promising option for children with neuroblastoma, as it can help maintain their quality of life by minimizing long-term side effects. Research suggests that PBRT provides strong disease control and survival rates, making it a valuable treatment to consider. ^{1 3 6 7 8}

Who Is on the Research Team?

Shannon MacDonald, MD

Principal Investigator

Massachusetts General Hospital

Are You a Good Fit for This Trial?

This trial is for children and young adults aged 3 to 25 with neuroblastoma or ganglioneuroblastoma. Participants must have confirmed diagnosis, agree to use contraception if of child-bearing age, and have a life expectancy over 12 months. Excluded are those who've had certain prior treatments or cancers, except specific low-risk cases.

Inclusion Criteria

My cancer does not need to be measurable for radiation treatment.

I've had an MRI or CT scan of the area needing treatment within the last 8 weeks.

Participants or their legal guardian must sign consent prior to the initiation of radiation therapy

Exclusion Criteria

Pregnant females are excluded. Women of childbearing age/menstruating must have a negative pregnancy test prior to initiation of radiation therapy.

I have not received radiation therapy above 500 cGy.

I had cancer before, but it's been 5 years and it was not likely to come back, or it was a minor skin cancer or cervical cancer in situ treated within the last 5 years.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive proton radiation therapy daily for 3-4 weeks, Monday through Friday, with each session lasting 30-45 minutes

3-4 weeks

Daily visits (Monday-Friday)

Follow-up

Participants are monitored for late side effects and overall health, with visits 3-6 months post-treatment and annually for up to 5 years

5 years

Initial visit 3-6 months post-treatment, then annual visits

What Are the Treatments Tested in This Trial?

Interventions

• Proton Beam Radiation Therapy

Trial Overview

The study tests proton beam radiation therapy (PBRT) as a treatment for neuroblastoma, aiming to reduce side effects compared to standard photon radiation by sparing surrounding tissues from unnecessary exposure.

How Is the Trial Designed?

Group I: Proton Beam Radiation Therapy (PBRT) Experimental Treatment 1 Intervention

Proton radiation will be delivered daily for 3-4 weeks, depending on the dose prescribed by study doctor. Treatment is delivered (Monday - Friday) for 5 days (no weekends or holidays). Each treatment the participant will lie on a table for 30-45 minutes.

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

Approved in European Union as Proton Therapy for:

• Esophageal adenocarcinoma
• Esophageal squamous cell carcinoma
• Gastroesophageal junction tumors

Approved in United States as Proton Therapy for:

• Esophageal adenocarcinoma
• Esophageal squamous cell carcinoma
• Gastroesophageal junction tumors

Approved in Canada as Proton Therapy for:

• Esophageal adenocarcinoma
• Esophageal squamous cell carcinoma
• Gastroesophageal junction tumors

Approved in Japan as Proton Therapy for:

• Esophageal adenocarcinoma
• Esophageal squamous cell carcinoma
• Gastroesophageal junction tumors

Approved in China as Proton Therapy for:

• Esophageal adenocarcinoma
• Esophageal squamous cell carcinoma
• Gastroesophageal junction tumors

Approved in Switzerland as Proton Therapy for:

• Esophageal adenocarcinoma
• Esophageal squamous cell carcinoma
• Gastroesophageal junction tumors

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+

Published Research Related to This Trial

Proton beam therapy (PBT) showed promising efficacy in treating pediatric patients with advanced neuroblastoma, with a 3-year locoregional control rate of 82% and 7 out of 14 patients remaining alive without disease after treatment.

PBT was well-tolerated, with no severe acute radiotoxicity reported, suggesting it may be a safer alternative to traditional photon radiotherapy for difficult-to-treat cases of neuroblastoma.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical results of proton beam therapy for advanced neuroblastoma. Oshiro, Y., Mizumoto, M., Okumura, T., et al.[2022]

In a study of 45 patients with high-risk neuroblastoma treated with proton radiation therapy (PRT), there was a remarkable 97% freedom from primary site recurrence at 3, 4, and 5 years, indicating strong efficacy of this treatment.

No patients experienced severe long-term renal or liver toxicity, suggesting that PRT is a safe option that effectively spares normal tissues while treating high-risk neuroblastoma.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Outcomes After Proton Therapy for Treatment of Pediatric High-Risk Neuroblastoma. Hill-Kayser, CE., Tochner, Z., Li, Y., et al.[2021]

In a study of 13 high-risk neuroblastoma patients, proton therapy demonstrated excellent target coverage while significantly sparing organs at risk, particularly the contralateral kidney and bowel, compared to intensity-modulated X-ray therapy (IMXT).

No patients experienced local recurrence or clinical organ toxicity, indicating that proton therapy is a safe and effective treatment option for high-risk neuroblastoma, although IMXT may be better for certain patients regarding renal sparing.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Proton versus photon radiation therapy for patients with high-risk neuroblastoma: the need for a customized approach. Hill-Kayser, C., Tochner, Z., Both, S., et al.[2022]

A study of 48 children with malignant brain tumors treated with proton beam therapy (PBT) showed that acute toxicities were generally low-grade and manageable, with fatigue, alopecia, and dermatitis being the most common side effects.

The research indicates that PBT is well tolerated in pediatric patients, and acute toxicities can be effectively managed with supportive care, suggesting a favorable safety profile for this treatment approach.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Acute toxicity of proton beam radiation for pediatric central nervous system malignancies. Suneja, G., Poorvu, PD., Hill-Kayser, C., et al.[2013]

Proton beam therapy (PBT) was found to be an effective and well-tolerated treatment for children with high-risk neuroblastoma, with a three-year overall survival rate of 90.9% and minimal severe acute toxicity reported during treatment.

In a study of 44 patients, PBT resulted in high local control (97.7%) and manageable side effects, with only seven cases of higher-grade hematologic toxicity and no long-term severe toxicities observed, suggesting it may reduce treatment-related side effects compared to conventional radiotherapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Proton Beam Therapy for Children With Neuroblastoma: Experiences From the Prospective KiProReg Registry. Jazmati, D., Butzer, S., Hero, B., et al.[2022]

Proton beam therapy (PBT) usage in the US increased from 0.4% in 2004 to 1.2% in 2018, particularly among patients with cancers recommended for PBT by the American Society of Radiation Oncology, indicating growing acceptance and application of this treatment.

While PBT use for prostate cancer temporarily decreased from 2011 to 2014, it rebounded by 2018, suggesting fluctuations in treatment preferences or insurance coverage that may influence patient access to this advanced therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Assessment of Proton Beam Therapy Use Among Patients With Newly Diagnosed Cancer in the US, 2004-2018. Nogueira, LM., Jemal, A., Yabroff, KR., et al.[2022]

Proton therapy for high-risk neuroblastoma showed excellent local control rates, with 94% at two years and 87% at five years for the primary site, and no failures at treated distant metastatic sites, based on a study of 18 patients with a median follow-up of 60.2 months.

The treatment resulted in a five-year overall survival rate of 94% and a progression-free survival rate of 64%, with no reported cases of radiation-related nephropathy or hepatopathy, indicating a favorable safety profile.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of proton therapy in children with high-risk and locally recurrent neuroblastoma. Bagley, AF., Grosshans, DR., Philip, NV., et al.[2020]

Proton beam therapy (PBT) for high-risk neuroblastoma was found to be safe and feasible, with all 18 patients completing treatment without delays and experiencing few acute adverse events.

The treatment resulted in a 3-year overall survival rate of 71% and a local control rate of 100%, indicating that PBT is an effective option for managing high-risk neuroblastoma, even when patients need to transfer between institutions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Patient Transfer to Receive Proton Beam Therapy During Intensive Multimodal Therapy is Safe and Feasible for Patients With Newly Diagnosed High-risk Neuroblastoma. Hosaka, S., Fukushima, H., Nakao, T., et al.[2021]

Citations

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pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC6588416/

Efficacy of Proton Therapy in Children with High Risk and ...

Our study demonstrates that for high-risk neuroblastoma patients, proton therapy provides excellent local and locoregional disease control both at the primary ...

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pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC7855697/

Proton Beam Therapy for Children With Neuroblastoma

At three years, the estimated local control, distant metastatic free survival, progression free survival, and overall survival was 97.7, 84.1, 81.8, and 90.9%, ...

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jamanetwork.com

jamanetwork.com/journals/jamaotolaryngology/fullarticle/2840479

Proton Beam vs Intensity-Modulated Radiotherapy in ...

Meaning PBRT may not improve overall survival, recurrence-free survival, or radiation toxic effects relative to IMRT for patients with ONB.

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sciencedirect.com

sciencedirect.com/science/article/pii/S2772610X25000303

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This review explores the development of PBT, its dosimetric advantages over conventional x-ray-based radiotherapy, and the rationale behind its use in ...

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advancesradonc.org

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

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Modeling studies indicate that proton beam scanning results in the lowest out-of-field dose while data for photon treatments and passive ...

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mayoclinic.org

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pmc.ncbi.nlm.nih.gov

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Pediatric Experience and Outcomes from the First Single-Vault ...

Our data indicate that proton therapy allowed for the growth of our pediatric radiotherapy service with an increase in referral of pediatric ...

8.

redjournal.org

redjournal.org/article/S0360-3016(19)30190-7/abstract

Outcomes After Proton Therapy for Treatment of Pediatric ...

We observed 97% freedom from primary site recurrence at 3, 4, and 5 years. Overall survival rates were 89%, 80%, and 80% and disease-free survival rates were 77 ...