Proton radiation therapy for treating neuroblastoma

Phase II Study of Proton Radiation Therapy for Neuroblastoma

This study tests if proton radiation therapy can effectively treat neuroblastoma and ganglioneuroblastoma in children while causing less harm to healthy tissues compared to regular radiation.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 30 (estimated)
• Ages: 6 Months to 25 Years
• Sex: All
• Sponsor: Massachusetts General Hospital (Academic / other)
• Drugs / interventions: chemotherapy, radiation
• Locations: 1 site (Boston, Massachusetts)
• Trial ID: NCT02112617 on ClinicalTrials.gov

What this trial studies

This research evaluates proton beam radiation therapy (PBRT) as a treatment for neuroblastoma and ganglioneuroblastoma, which are cancers that primarily affect children. The study aims to determine the effectiveness of PBRT, which is designed to minimize radiation exposure to surrounding healthy tissues compared to conventional photon radiation therapy. Participants will receive daily outpatient treatments over 3-4 weeks, with regular assessments to monitor side effects and overall health. Follow-up visits will occur for up to five years post-treatment to evaluate long-term outcomes.

Who should consider this trial

Good fit: Ideal candidates are children and young adults aged 3 to 25 years with confirmed neuroblastoma or ganglioneuroblastoma.

Not a fit: Patients with other types of cancer or those who do not meet the specific eligibility criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could lead to fewer side effects and improved outcomes for children with neuroblastoma.

How similar studies have performed: Other studies have shown promise with proton radiation therapy in pediatric populations, suggesting potential for success in this approach.

Eligibility criteria

Inclusion Criteria:

* Participants must have histologically confirmed neuroblastoma or ganglioneuroblastoma or elevated urinary catecholamine metabolites. If tumor tissue was obtained, pathological review of surgical specimen at the Massachusetts General Hospital or other DF/HCC institution is required, but preliminary report only required prior to enrollment. If no tumor tissue was obtained, urinary catecholamine metabolites are required.
* Participants do not need to have measurable disease at the time of radiation. Age ≥ 3 and ≤ 25 years at the time of diagnosis because this study evaluates this disease entity in the pediatric population which may differ from the adult population.
* Life expectancy of greater than 12 months.
* ECOG performance status ≤2 (Karnofsky ≥60%, Lansky ≥60%, see Appendix A).
* Diagnostic Imaging MRI and/or CT of the area to be treated within 8 weeks of any treatment. Baseline bone marrow biopsy and bone scan (with 99mTc-diphosphonate or MIBG scan (131I-MIBG or 123I-MIBG) from time of original diagnosis is required.
* Because radiation is known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
* Ability of the patient or the patient's legal guardian to understand and the willingness to sign a written informed consent document.
* Participants or their legal guardian must sign consent prior to the initiation of radiation therapy.

Exclusion Criteria:

* Patients will be ineligible if any prior therapeutic radiation therapy \> 500 cGy has been delivered.
* Patients will be ineligible if chemotherapy was completed ≥ 1 year from the planned start date of radiation therapy or if the patient is referred for radiation therapy after a relapse following a regimen with chemotherapy alone.
* Individuals with a history of a different malignancy are ineligible except for the following circumstances. Individuals with a history of other malignancies are eligible if they have been disease-free for at least 5 years and are deemed by the investigator to be at low risk for recurrence of that malignancy. Individuals with the following cancers are eligible if diagnosed and treated within the past 5 years: cervical cancer in situ, and basal cell or squamous cell carcinoma of the skin.
* Pregnant females are excluded. Women of childbearing age/menstruating must have a negative pregnancy test prior to initiation of radiation therapy.

Where this trial is running

Boston, Massachusetts

• Massachusetts General Hospital — Boston, Massachusetts, United States (Recruiting)

Study contacts

• Principal investigator: Shannon M. MacDonald, MD — Massachusetts General Hospital
• Study coordinator: Shannon M MacDonald, MD

Conditions Neuroblastoma; Ganglioneuroblastoma; Childhood neuroblastoma; Pediatric neuroblastoma