Proton Radiotherapy for Pediatric Brain Tumors Requiring Partial Brain Irradiation

June 2, 2025 updated by: Torunn Yock, MD, Massachusetts General Hospital

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

100

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Dana-Farber Cancer Institute
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 25 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Proton Radiotherapy
Radiation: Proton radiotherapy
5 days a week

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Endocrine Dysfunction
Time Frame: 3- and 5- years post radiation treatment
Cumulative incidence (estimated percentage participants) that developed endocrine dysfunction at 3- and 5-years following completion of proton radiation therapy.
3- and 5- years post radiation treatment
Neurocognitive Sequelae
Time Frame: 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.
The overall change in neurocognitive outcomes between treatment and last-follow-up as assessed by Wechsler Intelligence Scale for Children version 4 (WISC-IV). The test measures the Full-Scale Intelligence Quotient (FSIQ) of children through four indices; the Verbal Comprehension Index (VCI), Perceptual Reasoning Index (PRI), working memory test, and a processing speed test. FSIQ and the four indices are all assessed on a bell curve scale with an average score of 100 and standard deviation of 15. Higher scores represent higher intelligence and lower scores represent reduced intelligence.
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Disease Control
Time Frame: 3- and 5- years post radiation treatment

Three and 5 year local and distant disease control survival probabilities. Participants were assessed each follow-up and categorized in the following ways: 1) disease controlled no evidence of progression, 2) disease not controlled, tumor has progressed 3) patient has suffered a second malignancy (new tumor unrelated to the one the participant was treated for). Disease failure (recurrence of primary tumor) can occur locally (at or near the site of the original tumor), distally (located further away from the original tumor site), or both.

Participants who did not fail locally or in a distant site were censored at the date of their last follow-up, including death due to other causes. Disease control shows the percent probability of remaining recurrence free (disease is controlled) in follow-up post radiation treatment completion.
3- and 5- years post radiation treatment
Cumulative Incidence of Grade 3+ Toxicities
Time Frame: 3- and 5- years post radiation treatment
Percentage of participants who experienced a toxicity following completion of radiation treatment as measured by the NCI Common Terminology Criteria for Adverse Events Version 4.0 (CTCAE 4.0) after the completion of radiation therapy. Cumulative incidence is shown at 3- and 5-years post treatment. The descriptions and grading scales found in the NCI Common Terminology Criteria
3- and 5- years post radiation treatment
Cumulative Incidence of Ototoxicity
Time Frame: 3- and 5- years post radiation treatment
Cumulative incidence (estimated percentage participants) who experienced ototoxicity defined as either grade 3 or 4 hearing loss in either ear after the completion of radiation therapy in the overall participant population. Cumulative incidence and 95% confidence intervals are shown at 3- and 5-years post radiation treatment.
3- and 5- years post radiation treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Massachusetts General Hospital

Collaborators

National Cancer Institute (NCI)
Dana-Farber Cancer Institute

Investigators

  • Principal Investigator: Torunn I. Yock, MD, Massachusetts General Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 1, 2011

Primary Completion (Actual)

September 1, 2016

Study Completion (Actual)

September 1, 2016

Study Registration Dates

First Submitted

February 1, 2011

First Submitted That Met QC Criteria

February 1, 2011

First Posted (Estimated)

February 2, 2011

Study Record Updates

Last Update Posted (Actual)

June 3, 2025

Last Update Submitted That Met QC Criteria

June 2, 2025

Last Verified

June 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 10-206

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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