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

Trial Summary

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 data supports the effectiveness of the treatment Proton Beam Radiation Therapy for Neuroblastoma?

Proton therapy for neuroblastoma may help protect nearby healthy organs better than traditional X-ray therapy, and it is considered a promising option because it can reduce side effects while still effectively treating the cancer. ^{1 2 3 4 5}

Is proton radiation therapy safe for treating neuroblastoma in children?

Proton radiation therapy for neuroblastoma in children is generally considered safe, with studies showing few acute side effects and successful completion of treatment without delays. It may also reduce the risk of damage to nearby healthy organs compared to traditional radiation methods. ^{1 3 6 7 8}

How is proton radiation therapy different from other treatments for neuroblastoma?

Proton radiation therapy is unique because it targets tumors more precisely than conventional radiation, reducing damage to surrounding healthy tissues and potentially lowering side effects, which is especially important in treating children with neuroblastoma. ^{1 2 3 4 5}

What is the purpose of this trial?

This research study is evaluating a therapy called proton beam radiation therapy (PBRT) as a possible treatment for neuroblastoma. Neuroblastoma most commonly occurs in and around the adrenal glands, which are located at the top of the kidneys. However, it can also occur in other areas where groups of nerve cells exist, such as other areas of the abdomen, neck and near the spine. Conventional radiation therapy with photons is used as standard treatment for many patients with neuroblastic tumors. In this research study, the investigators are looking at another type of radiation called proton radiation which is known to spare surrounding tissues and organs from unnecessary radiation. Proton radiation delivers radiation to the area requiring radiation. This may reduce side effects that patients would normally experience with standard radiation therapy or other means of delivering proton radiation therapy. In this research study, the investigators are evaluating the effectiveness of using proton radiation delivered to reduce side effects associated with radiation treatment. The investigators will also be assessing the late side effects experienced by participants in each treatment group.

Research Team

Shannon MacDonald, MD

Principal Investigator

Massachusetts General Hospital

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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 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]

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]

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 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]

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]

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]

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]

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

Proton Beam Therapy for Children With Neuroblastoma: Experiences From the Prospective KiProReg Registry. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Efficacy of proton therapy in children with high-risk and locally recurrent neuroblastoma. [2020]

3. United States pubmed.ncbi.nlm.nih.gov

Proton versus photon radiation therapy for patients with high-risk neuroblastoma: the need for a customized approach. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Assessment of Proton Beam Therapy Use Among Patients With Newly Diagnosed Cancer in the US, 2004-2018. [2022]

5. England pubmed.ncbi.nlm.nih.gov

Clinical results of proton beam therapy for advanced neuroblastoma. [2022]

6. United States 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. [2021]

7. United States pubmed.ncbi.nlm.nih.gov

Outcomes After Proton Therapy for Treatment of Pediatric High-Risk Neuroblastoma. [2021]

8. United States pubmed.ncbi.nlm.nih.gov

Acute toxicity of proton beam radiation for pediatric central nervous system malignancies. [2013]