Clinical trial NCT01288235: Proton Radiotherapy for Pediatric Brain Tumors Requiring Partial Brain Irradiation
Official title: A Phase II Study of Proton Radiotherapy for Pediatric Brain Tumors Requiring Partial Brain Irradiation: An Assessment of Long Term Neurocognitive, Neuroendocrine Adn Ototoxicity Outcomes
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Brain Tumor
Low Grade Glioma
Astrocytoma
Ependymoma
Ganglioglioma
Keywords: proton radiotherapy
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
Collaborators: name: Dana-Farber Cancer Institute
name: National Cancer Institute (NCI)
First posted: 2011-02-02
Enrollment: 100
Sex: ALL
Minimum age (years): 1
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

Detailed description: - Participants will receive proton radiotherapy at the Francis H. Burr Proton Therapy Center which is located at the Massachusetts General Hospital. They will receive the proton radiotherapy 5 days per week. The number of weeks the participant will be receiving proton radiotherapy depends upon the tumor type and location and how well they are tolerating the treatment. Participant's will have a physical exam weekly during proton radiotherapy treatment.

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.

Primary outcomes:
measure: Endocrine Dysfunction
timeFrame: 3- and 5- years post radiation treatment
measure: Neurocognitive Sequelae
timeFrame: From the start of radiation treatment (baseline) to study completion. Participants were assessed annually up to 5 years following treatment completion. The median follow-up is 4.7 years.
Secondary outcomes:
measure: Disease Control
timeFrame: 3- and 5- years post radiation treatment
measure: Cumulative Incidence of Grade 3+ Toxicities
timeFrame: 3- and 5- years post radiation treatment
measure: Cumulative Incidence of Ototoxicity
timeFrame: 3- and 5- years post radiation treatment
Other outcomes:
Locations:
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Boston
country: United States
facility: Dana-Farber Cancer Institute
state: Massachusetts
Study officials:
affiliation: Massachusetts General Hospital
name: Torunn I. Yock, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Vatner RE, Niemierko A, Misra M, Weyman EA, Goebel CP, Ebb DH, Jones RM, Huang MS, Mahajan A, Grosshans DR, Paulino AC, Stanley T, MacDonald SM, Tarbell NJ, Yock TI. Endocrine Deficiency As a Function of Radiation Dose to the Hypothalamus and Pituitary in Pediatric and Young Adult Patients With Brain Tumors. J Clin Oncol. 2018 Oct 1;36(28):2854-2862. doi: 10.1200/JCO.2018.78.1492. Epub 2018 Aug 17.