Brain TumorNCT01063114
Proton Beam Radiotherapy for Medulloblastoma and Pineoblastoma
A study testing proton beam radiation in people with Brain Tumor and Medulloblastoma, run by Massachusetts General Hospital at 2 sites in United States.
Trial summary
There are two types of external radiation treatments (proton beam and photon beam). As part of the participant's treatment, they will receive radiation to the entire central nervous system (CNS); this is known as craniospinal irradiation (CSI). In the past, photon radiation therapy has been used for CSI. Read more about this study
- Who is it for?
- Ages 3 to 25 years
- Conditions the study is for
- Brain Tumor, Medulloblastoma, Pineoblastoma
- What is being tested?
- proton beam radiation
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- 2 sitesUnited States
- Study dates
- 8 Apr 2010to Nov 2021
- Phase
- Interventional
- 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 3 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Participants must have undergone biopsy or attempted surgical resection and must have histologically confirmed medulloblastoma or pineoblastoma.
- Participants may have had a gross total resection, sub-total resection or biopsy only.
- For patients with no prior chemotherapy, treatment must start within 35 days of definitive surgery or as indicated if enrolled on therapeutic study
- Age range between 3 and 25 at the time of enrollment
- Life expectancy of greater than 3 months
- Blood laboratory values as outlined in the protocol
- 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
- Patients with more than one previous chemotherapy regimen
- Patients with recurrent or progressive disease after one or more regimens of pre-radiation chemotherapy
- Patients with prior radiation therapy
- Any major uncontrolled or poorly controlled intercurrent illness that would limit compliance with study requirements
- Pregnant women
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Massachusetts General Hospital
Boston, Massachusetts, 02115
MD Anderson Cancer Center
Houston, Texas, 77030
About this study
Official title: Proton Beam Radiotherapy for Medulloblastoma and Pineoblastoma: An Assessment of Acute Toxicity and Long Term Neurocognitive, Neuroendocrine and Ototoxicity Outcomes
There are two types of external radiation treatments (proton beam and photon beam). As part of the participant's treatment, they will receive radiation to the entire central nervous system (CNS); this is known as craniospinal irradiation (CSI). In the past, photon radiation therapy has been used for CSI. In this study we will be examining the effects of proton beam radiation therapy. Studies have suggested that this kind of radiation can cause less damage to normal tissue than photon radiation therapy. The physical characteristics of proton beam radiation let the doctor safely deliver the amount of radiation delivered to the tumor that is normally delivered through standard therapy but spare more normal tissue in the process.
Read the detailed description
- Before beginning radiation therapy, participants will have scans done in order to prepare for the radiation treatment. Doctors will use information gathered from these scans to plan the best way to deliver radiation to the tumor.
- The following procedures will be performed either before or during the radiotherapy: Hearing exam; neurocognitive exam; blood tests and cerebral spinal fluid test.
- Not everyone who participates in this study will receive the same amount of proton radiation therapy. The length of time and amount of radiation received will depend upon the condition of the participant's disease. Radiation treatment will be given once a day, 5 days a week (Monday-Friday). The overall treatment course will be approximately 6 weeks.
- Conditions
- Brain Tumor, Medulloblastoma, Pineoblastoma
- Being tested
- proton beam radiation
- Keywords
- proton beam radiation
- Medical subject headings
- Brain Neoplasms, Medulloblastoma, Pinealoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
proton beam radiationRadiation
Given once a day, 5 days a week (Monday-Friday)
| Study group | Role | What they receive |
|---|---|---|
| Proton Beam Radiation | Experimental | Proton Beam Radiation Receives: Radiation: proton beam radiation |
What is being measured
The primary outcome is the main question the study is designed to answer.
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 RT (radiation treatment)
Measured: 3- and 5- years post radiation treatment
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 Effects
Difference in Full-Scale Intelligence Quotient (FSIQ) between baseline and latest follow-up neurocognitive test. Tests used include WISC (Wechsler Intelligence Scale for Children) IV, WISC V, WAIS (Wechsler Adult Intelligence Scale) III, WAIS IV. The test measures the 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: Baseline and Follow-up (median of 6 years)
Secondary outcomes (3)
Progression Free Survival
3- and 5-year progression free survival rate of pediatric medulloblastoma and pineoblastoma patients treated with proton CSI treatment.
Measured: 3- and 5-years post radiation treatment
Treatment Efficiency
To modify the speed of treating patients requiring cranial spinal irradiation (CSI) and improve the safety of treating patients under anesthesia
Measured: 2 years
Percentage of Participants Who Experienced an Acute Toxicity by Grade
All patients shall be assessed for acute toxicity on a weekly basis during craniospinal irradiation (CSI). Percentage of participants who experienced an acute toxicity by grade. Acute toxicities are graded according to the Common Terminology Criteria for Adverse Events (CTCAE) v4.0. Toxicities are graded on a scale of 1 to 5. A higher grade indicates a worse outcome with 1 being mild, 4 being life-threatening, and 5 being death.
Measured: 3 months after radiation therapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2010 (estimated)
- Study start8 Apr 2010
- Primary completionNov 2021
- Study completionNov 2021
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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 90 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- M.D. Anderson Cancer Center, 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
- NCT01063114
- Sponsor's protocol ID
- 09-361
- Other IDs
- P01CA021239
- Status last verified
- Aug 2025
- First submitted
- 3 Feb 2010
- Last updated
- 13 Aug 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 13 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Proton Beam Radiotherapy for Medulloblastoma and Pineoblastoma: An Assessment of Acute Toxicity and Long Term Neurocognitive, Neuroendocrine and Ototoxicity Outcomes
- ClinicalTrials.gov ID
- NCT01063114
- Acronym / sponsor ID
- 09-361
- Conditions
- Brain Tumor, Medulloblastoma, Pineoblastoma
- Keywords
- proton beam radiation
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 90 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Collaborators
- M.D. Anderson Cancer Center, National Cancer Institute (NCI)
- Study start
- 8 Apr 2010 (actual)
- Primary completion
- Nov 2021 (actual)
- Study completion
- Nov 2021 (actual)
- First posted
- 5 Feb 2010 (estimated)
- Results
- Posted 13 Aug 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
There are two types of external radiation treatments (proton beam and photon beam). As part of the participant's treatment, they will receive radiation to the entire central nervous system (CNS); this is known as craniospinal irradiation (CSI). In the past, photon radiation therapy has been used for CSI. In this study we will be examining the effects of proton beam radiation therapy. Studies have suggested that this kind of radiation can cause less damage to normal tissue than photon radiation therapy. The physical characteristics of proton beam radiation let the doctor safely deliver the amount of radiation delivered to the tumor that is normally delivered through standard therapy but spare more normal tissue in the process.
Detailed description
- Before beginning radiation therapy, participants will have scans done in order to prepare for the radiation treatment. Doctors will use information gathered from these scans to plan the best way to deliver radiation to the tumor.
- The following procedures will be performed either before or during the radiotherapy: Hearing exam; neurocognitive exam; blood tests and cerebral spinal fluid test.
- Not everyone who participates in this study will receive the same amount of proton radiation therapy. The length of time and amount of radiation received will depend upon the condition of the participant's disease. Radiation treatment will be given once a day, 5 days a week (Monday-Friday). The overall treatment course will be approximately 6 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Proton Beam Radiation | Experimental | Radiation: proton beam radiationProton Beam Radiation |
Who may qualify
- Ages
- 3 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Participants must have undergone biopsy or attempted surgical resection and must have histologically confirmed medulloblastoma or pineoblastoma.
- Participants may have had a gross total resection, sub-total resection or biopsy only.
- For patients with no prior chemotherapy, treatment must start within 35 days of definitive surgery or as indicated if enrolled on therapeutic study
- Age range between 3 and 25 at the time of enrollment
- Life expectancy of greater than 3 months
Key exclusion criteria (5 listed)
- Patients with more than one previous chemotherapy regimen
- Patients with recurrent or progressive disease after one or more regimens of pre-radiation chemotherapy
- Patients with prior radiation therapy
- Any major uncontrolled or poorly controlled intercurrent illness that would limit compliance with study requirements
- Pregnant women
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 beam radiationGiven once a day, 5 days a week (Monday-Friday) | Radiation | Proton Beam Radiation |
Primary outcome
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 RT (radiation treatment)
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 Effects
Time frame: Baseline and Follow-up (median of 6 years)
Difference in Full-Scale Intelligence Quotient (FSIQ) between baseline and latest follow-up neurocognitive test. Tests used include WISC (Wechsler Intelligence Scale for Children) IV, WISC V, WAIS (Wechsler Adult Intelligence Scale) III, WAIS IV. The test measures the 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
Progression Free Survival
Time frame: 3- and 5-years post radiation treatment
Treatment Efficiency
Time frame: 2 years
Percentage of Participants Who Experienced an Acute Toxicity by Grade
Time frame: 3 months after radiation therapy
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Aug 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 study testing proton beam radiation in people with Brain Tumor and Medulloblastoma, 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 13 Aug 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.
Are results available for this study?
Yes. Results are posted in the registry (first posted 13 Aug 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?
Cumulative Incidence of Ototoxicity (time frame: 3- and 5- years post radiation treatment). The study lists 3 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 NCT01063114, 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 13 Aug 2025.