Reduced-dose Radiotherapy for Neuroblastoma

Overseen By Brian Kushner, MD

Age: < 65

Sex: Any

Trial Phase: Phase < 1

Sponsor: Memorial Sloan Kettering Cancer Center

Disqualifiers: Residual tumor, Prior radiotherapy, Others

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

Compensation: Varies

Number of Visits: 14

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a lower dose of proton beam radiation to treat high-risk neuroblastoma, a cancer that often affects children. The goal is to minimize long-term side effects. Participants will receive radiation therapy twice daily for 5-6 weekdays. Suitable candidates have high-risk neuroblastoma, have undergone chemotherapy and surgery, and have no remaining tumors after surgery. As an Early Phase 1 trial, this research aims to understand how the treatment works in people, offering participants a chance to be among the first to receive this innovative therapy.

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 team or your doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that proton beam radiotherapy (RT) is generally safe and well-tolerated in children with neuroblastoma, a type of cancer. One study found it both safe and effective in controlling tumors, making it a manageable treatment option. In another study with 99 patients, 81% survived after treatment, and 92% experienced no cancer recurrence in the treated area. These findings suggest that proton therapy effectively controls the disease and is safe.

While some side effects can occur, most children experience them within a tolerable range. For example, another study noted some short-term side effects, but these were usually manageable. Overall, the evidence supports proton therapy as a promising and safe option for this condition. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about proton beam radiotherapy for high-risk neuroblastoma because it offers a more precise way to target tumors while sparing surrounding healthy tissue compared to traditional radiation therapies. Most current treatments use conventional radiation, which can affect both cancerous and healthy cells. Proton beam therapy, on the other hand, delivers radiation directly to the tumor with minimal impact on nearby areas, potentially reducing side effects. This precision makes it particularly promising for treating neuroblastoma, where minimizing damage to growing children's bodies is crucial.

What evidence suggests that this trial's treatments could be effective for neuroblastoma?

Research has shown that proton beam therapy, which participants in this trial may receive, effectively treats high-risk neuroblastoma, a cancer often found in children. Studies indicate that 82% of patients who receive proton therapy survive, and 97% have no cancer at the original site. Proton therapy is considered safe and may yield better results than traditional radiation. It targets the cancer more precisely, protecting nearby healthy tissue. This precision makes it a promising treatment for neuroblastoma with fewer side effects. ^{3 5 6 7 8}

Who Is on the Research Team?

Suzanne Wolden, MD

Principal Investigator

Memorial Sloan Kettering Cancer Center

Are You a Good Fit for This Trial?

This trial is for children and young adults (1 month to 18 years old) with high-risk neuroblastoma who've had chemotherapy and surgery. They must not be pregnant, breastfeeding, or have any leftover large tumors after surgery. If they can have children, they need to use birth control and have a negative pregnancy test before starting.

Inclusion Criteria

I have stage 3 neuroblastoma with MYCN amplification or I am over 18 months old with unfavorable pathology.

My diagnosis of neuroblastoma has been confirmed by a specialist.

I am a woman who can have children and have a recent negative pregnancy test.

Exclusion Criteria

I still have visible tumor after surgery.

Patients with any concurrent medical or psychiatric condition or disease which, in the investigator's judgment, would make them inappropriate candidates for entry into this study.

I have had radiation therapy on or near where my tumor was located.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Radiation

Patients undergo external beam radiation therapy using IMRT or proton beam RT twice daily for 5-6 weekdays (10-12 treatments)

1-2 weeks

10-12 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, with evaluations at 6, 12, 18, and 24 months

24 months

4 visits (in-person)

Long-term follow-up

Event-free survival and assessment of toxicity will be monitored over 3 years

3 years

What Are the Treatments Tested in This Trial?

Interventions

• External Beam Radiotherapy
• Proton Beam RT

Trial Overview The study tests whether using a lower dose of radiation therapy (proton beam RT or external beam radiotherapy) can effectively treat neuroblastoma while reducing long-term side effects from the treatment.

How Is the Trial Designed?

Group I: patients with high-risk neuroblastoma Experimental Treatment 2 Interventions

Patients undergo external beam radiation therapy using IMRT or proton beam RT twice daily for 5-6 weekdays (10-12 treatments). Patients will be evaluated by physical exams, CT scan or MRI of the primary site, and MIBG at, 6, 12, 18 and 24 months (+/- 6 weeks).

Who Is Running the Clinical Trial?

Memorial Sloan Kettering Cancer Center

Lead Sponsor

Trials

1,998

Recruited

602,000+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/30509728/

The Evidence for External Beam Radiotherapy in High- ... Conclusions: There are data that show that radiotherapy is of some efficacy in the management of high-risk neuroblastoma, but there is no level one evidence ...

2. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0936655518305272

The Evidence for External Beam Radiotherapy in High ... Conclusions. There are data that show that radiotherapy is of some efficacy in the management of high-risk neuroblastoma, but there is no level one evidence ...

3. thegreenjournal.com thegreenjournal.com/article/S0167-8140(24)00654-6/fulltext

Implications for radiation dose and field Reduced-dose radiation therapy to the primary site is effective for high-risk neuroblastoma: results from a prospective trial. Int J Radiat ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9689739/

Efficacy and Feasibility of Proton Beam Therapy in Relapsed ... In general, the prognosis for patients with recurrent HR NB is poor, with survival rates reported between 3% and 15% only [4,5]. However, with ...

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

Outcomes After Proton Therapy for Treatment of Pediatric ... Overall survival rates were 89%, 80%, and 80% and disease-free survival rates were 77%, 70%, and 70%, at 3, 4, and 5 years, respectively.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12048667/

Impact of radiotherapy on survival outcomes in metastatic ... The 5-year survival rate is estimated to range from 75 to 98% for children with low- and intermediate-risk neuroblastoma, while it falls below ...

7. redjournal.org redjournal.org/article/S0360-3016(24)02303-4/fulltext

Characterization of Clinical Outcomes for Patients with ... Characterization of clinical outcomes for patients with relapsed high-risk neuroblastoma after external beam radiotherapy.

8. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/22749633/

Results of a quality assurance review of external beam ... This study reviewed the compliance of plans with the radiation therapy guidelines of the International Society of Paediatric Oncology (Europe) Neuroblastoma ...