Brain TumorPhase 2NCT01288235
Proton Radiotherapy for Pediatric Brain Tumors Requiring Partial Brain Irradiation
A Phase 2 study testing Proton radiotherapy in people with Brain Tumor and Low Grade Glioma, run by Massachusetts General Hospital at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year to 25 years
- Conditions the study is for
- Brain Tumor, Low Grade Glioma, Astrocytoma
- What is being tested?
- Proton radiotherapy
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 2 sitesUnited States
- Study dates
- Feb 2011to Sep 2016
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115
About this study
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
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.
Read the 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.
- Conditions
- Brain Tumor, Low Grade Glioma, Astrocytoma, Ependymoma, Ganglioglioma
- Being tested
- Proton radiotherapy
- Keywords
- proton radiotherapy
- Medical subject headings
- Brain Neoplasms, Astrocytoma, Ependymoma, Ganglioglioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Proton radiotherapyRadiation
5 days a week
| Study group | Role | What they receive |
|---|---|---|
| Proton Radiotherapy | Experimental | Proton Radiotherapy Receives: Radiation: Proton radiotherapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Endocrine Dysfunction
Cumulative incidence (estimated percentage participants) that developed endocrine dysfunction at 3- and 5-years following completion of proton radiation therapy.
Measured: 3- and 5- years post radiation treatment
Neurocognitive Sequelae
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.
Measured: 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 (3)
Disease Control
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.
Measured: 3- and 5- years post radiation treatment
Cumulative Incidence of Grade 3+ Toxicities
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
Measured: 3- and 5- years post radiation treatment
Cumulative Incidence of Ototoxicity
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.
Measured: 3- and 5- years post radiation treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2011 (estimated)
- Study startFeb 2011
- Primary completionSep 2016
- Study completionSep 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Brain TumorStudies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Dana-Farber Cancer Institute, National Cancer Institute (NCI)
- Responsible party
- Principal investigator, Torunn Yock, MD, Massachusetts General Hospital
- Investigators
- Torunn I. Yock, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01288235
- Sponsor's protocol ID
- 10-206
- Status last verified
- Jun 2025
- First submitted
- 1 Feb 2011
- Last updated
- 3 Jun 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
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.
PubMed 30118397
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01288235
- Acronym / sponsor ID
- 10-206
- Conditions
- Brain Tumor, Low Grade Glioma, Astrocytoma, Ependymoma, Ganglioglioma
- Keywords
- proton radiotherapy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Collaborators
- Dana-Farber Cancer Institute, National Cancer Institute (NCI)
- Study start
- Feb 2011 (actual)
- Primary completion
- Sep 2016 (actual)
- Study completion
- Sep 2016 (actual)
- First posted
- 2 Feb 2011 (estimated)
- Results
- Posted 3 Jun 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Proton Radiotherapy | Experimental | Radiation: Proton radiotherapyProton Radiotherapy |
Who may qualify
- Ages
- 1 year to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Proton radiotherapy5 days a week | Radiation | Proton Radiotherapy |
Primary outcome
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.
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.
3 secondary outcomes
Disease Control
Time frame: 3- and 5- years post radiation treatment
Cumulative Incidence of Grade 3+ Toxicities
Time frame: 3- and 5- years post radiation treatment
Cumulative Incidence of Ototoxicity
Time frame: 3- and 5- years post radiation treatment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 30118397Vatner 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Torunn I. Yock, MD principal investigator
Massachusetts General Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Proton radiotherapy in people with Brain Tumor and Low Grade Glioma, run by Massachusetts General Hospital at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 3 Jun 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Endocrine Dysfunction (time frame: 3- and 5- years post radiation treatment). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01288235, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jun 2025.