Phase 2
N=100
Proton Radiotherapy for Pediatric Brain Tumors Requiring Partial Brain Irradiation
Brain Tumor · Low Grade Glioma · Astrocytoma · Ependymoma · Ganglioglioma
Bottom Line
View on ClinicalTrials.gov: NCT01288235 ↗Enrolled (actual)
100
Serious AEs
2.0%
Results posted
Jun 2025
Primary outcome: Primary: Endocrine Dysfunction — 14.5; 20.2 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Proton radiotherapy (Radiation)
- Age
- Pediatric, Adult · 1+ yrs
- Sex
- All
- Sponsor
- Massachusetts General Hospital
- Primary completion
- Sep 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Endocrine Dysfunction |
14.5; 20.2 | — |
| PRIMARY Neurocognitive Sequelae |
99.2; 100.3 | 0.543 |
| SECONDARY Disease Control |
89.9; 85.9; 97.0; 95.0 | — |
| SECONDARY Cumulative Incidence of Grade 3+ Toxicities |
12.2; 16.3 | — |
| SECONDARY Cumulative Incidence of Ototoxicity |
12.5; 12.5 | — |
Summary
Some patients with brain tumors receive standard radiation to help prevent tumor growth. Although standard radiation kills tumor cells, it can also damage normal tissue in the process and lead to more side effects. This research study is looking at a different form of radiation called proton radiotherapy which helps spare normal tissues while delivering radiation to the tumor or tumor bed. Proton techniques irradiate 2-3 times less normal tissue then standard radiation. This therapy has been used in treatment of other cancers and information from those other research studies suggests that this therapy may help better target brain tumors then standard radiation.
Eligibility Criteria
Inclusion Criteria
- Biopsy proven low grade glioma or astrocytoma, ependymoma, craniopharyngioma, meningioma, neurocytoma, medulloblastoma or gangliogliomas or other rare tumor requiring tumor bed or tumor irradiation. Patients with a presumed diagnosis of optic glioma or gliomas based on imaging and clinical characteristics will also be allowed on this trial.
- Patients with biopsy proven high grade glioma (excluding GBM) and a gross total resection and patients with non-disseminated atypical teratoid rhabdoid (ATRT) may also be included.
- Pathologic diagnosis must be based on pathology or pathology review by Department of Pathology at MGH or another DF/HCC institution.
- Age between 1-25 years.
- Life expectancy of greater than 1 year.
- ECOG Performance Status 0, 1, 2 or 3 or Lansky performance status 30 or greater.
- Girls and women of child-bearing potential and men must agree to use adequate contraception prior to study entry and for the duration of study participation.
Exclusion Criteria
- Participants who have had radiotherapy to the site to be treated.
- Participants with known spinal or distant metastases. Patients with ependymoma, medulloblastoma or germinoma must have metastatic workup including spine MRI to rule out metastases.
- Uncontrolled intercurrent illness that would limit compliance with study requirements.
- Pregnant or breastfeeding women.
- Patients who cannot participate in contributing to the neurocognitive outcomes due to severe neurologic impairment or language barrier (ie not English or Spanish speaking) will be excluded from this study.
Data sourced from ClinicalTrials.gov (NCT01288235). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.