NeuroblastomaNCT02112617
Phase II Study of Proton Radiation Therapy for Neuroblastoma
A study testing Proton Beam Radiation Therapy in people with Neuroblastoma and Ganglioneuroblastoma, run by Massachusetts General Hospital at 1 site in United States.
Trial summary
This research study is evaluating a therapy called proton beam radiation therapy (PBRT) as a possible treatment for neuroblastoma. Neuroblastoma most commonly occurs in and around the adrenal glands, which are located at the top of the kidneys. Read more about this study
- Who is it for?
- Ages 6 months to 25 years
- Conditions the study is for
- Neuroblastoma, Ganglioneuroblastoma
- What is being tested?
- Proton Beam Radiation Therapy
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2014to Mar 2028
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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 6 months to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Participants must have histologically confirmed neuroblastoma or ganglioneuroblastoma or elevated urinary catecholamine metabolites. If tumor tissue was obtained, pathological review of surgical specimen at the Massachusetts General Hospital or other DF/HCC institution is required, but preliminary report only required prior to enrollment. If no tumor tissue was obtained, urinary catecholamine metabolites are required.
- Participants do not need to have measurable disease at the time of radiation. Age ≥ 3 and ≤ 25 years at the time of diagnosis because this study evaluates this disease entity in the pediatric population which may differ from the adult population.
- Life expectancy of greater than 12 months.
- ECOG performance status ≤2 (Karnofsky ≥60%, Lansky ≥60%, see Appendix A).
- Diagnostic Imaging MRI and/or CT of the area to be treated within 8 weeks of any treatment. Baseline bone marrow biopsy and bone scan (with 99mTc-diphosphonate or MIBG scan (131I-MIBG or 123I-MIBG) from time of original diagnosis is required.
- Because radiation is known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
- Ability of the patient or the patient's legal guardian to understand and the willingness to sign a written informed consent document.
- Participants or their legal guardian must sign consent prior to the initiation of radiation therapy.
Who cannot join
- Patients will be ineligible if any prior therapeutic radiation therapy > 500 cGy has been delivered.
- Patients will be ineligible if chemotherapy was completed ≥ 1 year from the planned start date of radiation therapy or if the patient is referred for radiation therapy after a relapse following a regimen with chemotherapy alone.
- Individuals with a history of a different malignancy are ineligible except for the following circumstances. Individuals with a history of other malignancies are eligible if they have been disease-free for at least 5 years and are deemed by the investigator to be at low risk for recurrence of that malignancy. Individuals with the following cancers are eligible if diagnosed and treated within the past 5 years: cervical cancer in situ, and basal cell or squamous cell carcinoma of the skin.
- Pregnant females are excluded. Women of childbearing age/menstruating must have a negative pregnancy test prior to initiation of radiation therapy.
Trial locations and contacts
1 site in 1 country.
United States1 site
Massachusetts General Hospital
Boston, Massachusetts, 02114
About this study
This research study is evaluating a therapy called proton beam radiation therapy (PBRT) as a possible treatment for neuroblastoma.
Neuroblastoma most commonly occurs in and around the adrenal glands, which are located at the top of the kidneys. However, it can also occur in other areas where groups of nerve cells exist, such as other areas of the abdomen, neck and near the spine.
Read more
Conventional radiation therapy with photons is used as standard treatment for many patients with neuroblastic tumors. In this research study, the investigators are looking at another type of radiation called proton radiation which is known to spare surrounding tissues and organs from unnecessary radiation. Proton radiation delivers radiation to the area requiring radiation. This may reduce side effects that patients would normally experience with standard radiation therapy or other means of delivering proton radiation therapy.
In this research study, the investigators are evaluating the effectiveness of using proton radiation delivered to reduce side effects associated with radiation treatment. The investigators will also be assessing the late side effects experienced by participants in each treatment group.
Read the detailed description
Proton radiation will be delivered daily for 3-4 weeks, depending on the dose prescribed by study doctor. Treatment is delivered (Monday - Friday) for 5 days (no weekends or holidays). Each treatment the participant will lie on a table for 30-45 minutes.
The radiation therapy will be outpatient at Massachusetts General Hospital.
During radiation therapy, the participant will have the following weekly assessments and procedures:
- Physical Exam
- Assess for any side effects
- Routine blood tests (weekly or at least every other week)
Post-treatment Follow Up Visit (s):
The participant will be asked to return to the clinic 3-6 months after their last dose of radiation therapy and then annually for up to 5 years for the following procedures:
- Medical History
- Physical Exam
- Diagnostic Imaging
- Assess for side effects
- Lung Function Tests (if required)
- MUGA or ECHO (if required)
- Conditions
- Neuroblastoma, Ganglioneuroblastoma
- Being tested
- Proton Beam Radiation Therapy
- Keywords
- Neuroblastoma, Childhood neuroblastoma, Ganglioneuroblastoma, Pediatric neuroblastoma
- Medical subject headings
- Neuroblastoma, Ganglioneuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Proton Beam Radiation TherapyRadiation
Proton radiation will be delivered daily for 3-4 weeks, depending on the dose prescribed by study doctor. Treatment is delivered (Monday - Friday) for 5 days (no weekends or holidays). Each treatment the participant will lie on a table for 30-45 minutes.
| Study group | Role | What they receive |
|---|---|---|
| Proton Beam Radiation Therapy (PBRT) | Experimental | Proton radiation will be delivered daily for 3-4 weeks, depending on the dose prescribed by study doctor. Treatment is delivered (Monday - Friday) for 5 days (no weekends or holidays). Each treatment the participant will lie on a table for 30-45 minutes. Receives: Radiation: Proton Beam Radiation Therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Describe late complications of radiation therapy delivered with proton radiotherapy in place of photon radiation for patients with neuroblastoma
To describe late complications of radiation therapy delivered with proton radiotherapy in place of photon radiation for patients with neuroblastoma. Specifically, cardiac, pulmonary, gastrointestinal, endocrine, growth and second malignancies will be described.
Measured: 5 years
To evaluate acute and subacute toxicities of proton radiotherapy in place of photon radiation for patients with neuroblastoma.
To evaluate acute and subacute toxicities of proton radiotherapy in place of photon radiation for patients with neuroblastoma. Specifically, nausea, vomiting, diarrhea, radiation pneumonitis and skin toxicity will be described.
Measured: 5 years
Secondary outcomes (2)
Progression free survival
Measured: 10 years
Overall survival rates
Measured: 10 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Apr 2014 (estimated)
- Study startJun 2014
- Primary completion30 Jun 2026
- Study completionMar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeuroblastomaStudies 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
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Principal investigator, Kevin X. Liu, MD, DPHIL, Massachusetts General Hospital
- Investigators
- Shannon M. MacDonald, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02112617
- Sponsor's protocol ID
- 13-443
- Status last verified
- Aug 2026
- First submitted
- 10 Apr 2014
- Last updated
- 20 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Proton Radiation Therapy for Neuroblastoma
- ClinicalTrials.gov ID
- NCT02112617
- Acronym / sponsor ID
- 13-443
- Conditions
- Neuroblastoma, Ganglioneuroblastoma
- Keywords
- Neuroblastoma, Childhood neuroblastoma, Ganglioneuroblastoma, Pediatric neuroblastoma
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Jun 2014
- Primary completion
- 30 Jun 2026 (actual)
- Study completion
- Mar 2028 (estimated)
- First posted
- 14 Apr 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This research study is evaluating a therapy called proton beam radiation therapy (PBRT) as a possible treatment for neuroblastoma.
Neuroblastoma most commonly occurs in and around the adrenal glands, which are located at the top of the kidneys. However, it can also occur in other areas where groups of nerve cells exist, such as other areas of the abdomen, neck and near the spine.
Conventional radiation therapy with photons is used as standard treatment for many patients with neuroblastic tumors. In this research study, the investigators are looking at another type of radiation called proton radiation which is known to spare surrounding tissues and organs from unnecessary radiation. Proton radiation delivers radiation to the area requiring radiation. This may reduce side effects that patients would normally experience with standard radiation therapy or other means of delivering proton radiation therapy.
In this research study, the investigators are evaluating the effectiveness of using proton radiation delivered to reduce side effects associated with radiation treatment. The investigators will also be assessing the late side effects experienced by participants in each treatment group.
Detailed description
Proton radiation will be delivered daily for 3-4 weeks, depending on the dose prescribed by study doctor. Treatment is delivered (Monday - Friday) for 5 days (no weekends or holidays). Each treatment the participant will lie on a table for 30-45 minutes.
The radiation therapy will be outpatient at Massachusetts General Hospital.
During radiation therapy, the participant will have the following weekly assessments and procedures:
- Physical Exam
- Assess for any side effects
- Routine blood tests (weekly or at least every other week)
Post-treatment Follow Up Visit (s):
The participant will be asked to return to the clinic 3-6 months after their last dose of radiation therapy and then annually for up to 5 years for the following procedures:
- Medical History
- Physical Exam
- Diagnostic Imaging
- Assess for side effects
- Lung Function Tests (if required)
- MUGA or ECHO (if required)
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 Therapy (PBRT) | Experimental | Radiation: Proton Beam Radiation TherapyProton radiation will be delivered daily for 3-4 weeks, depending on the dose prescribed by study doctor. Treatment is delivered (Monday - Friday) for 5 days (no weekends or holidays). Each treatment the participant will lie on a table for 30-45 minutes. |
Who may qualify
- Ages
- 6 months to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Participants must have histologically confirmed neuroblastoma or ganglioneuroblastoma or elevated urinary catecholamine metabolites. If tumor tissue was obtained, pathological review of surgical specimen at the Massachusetts General Hospital or other DF/HCC institution is required, but preliminary report only required prior to enrollment. If no tumor tissue was obtained, urinary catecholamine metabolites are required.
- Participants do not need to have measurable disease at the time of radiation. Age ≥ 3 and ≤ 25 years at the time of diagnosis because this study evaluates this disease entity in the pediatric population which may differ from the adult population.
- Life expectancy of greater than 12 months.
- ECOG performance status ≤2 (Karnofsky ≥60%, Lansky ≥60%, see Appendix A).
- Diagnostic Imaging MRI and/or CT of the area to be treated within 8 weeks of any treatment. Baseline bone marrow biopsy and bone scan (with 99mTc-diphosphonate or MIBG scan (131I-MIBG or 123I-MIBG) from time of original diagnosis is required.
Key exclusion criteria (4 listed)
- Patients will be ineligible if any prior therapeutic radiation therapy > 500 cGy has been delivered.
- Patients will be ineligible if chemotherapy was completed ≥ 1 year from the planned start date of radiation therapy or if the patient is referred for radiation therapy after a relapse following a regimen with chemotherapy alone.
- Individuals with a history of a different malignancy are ineligible except for the following circumstances. Individuals with a history of other malignancies are eligible if they have been disease-free for at least 5 years and are deemed by the investigator to be at low risk for recurrence of that malignancy. Individuals with the following cancers are eligible if diagnosed and treated within the past 5 years: cervical cancer in situ, and basal cell or squamous cell carcinoma of the skin.
- Pregnant females are excluded. Women of childbearing age/menstruating must have a negative pregnancy test prior to initiation of radiation therapy.
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 Radiation TherapyProton radiation will be delivered daily for 3-4 weeks, depending on the dose prescribed by study doctor. Treatment is delivered (Monday - Friday) for 5 days (no weekends or holidays). Each treatment the participant will lie on a table for 30-45 minutes. | Radiation | Proton Beam Radiation Therapy (PBRT) |
Primary outcome
Describe late complications of radiation therapy delivered with proton radiotherapy in place of photon radiation for patients with neuroblastoma
Time frame: 5 years
To describe late complications of radiation therapy delivered with proton radiotherapy in place of photon radiation for patients with neuroblastoma. Specifically, cardiac, pulmonary, gastrointestinal, endocrine, growth and second malignancies will be described.
To evaluate acute and subacute toxicities of proton radiotherapy in place of photon radiation for patients with neuroblastoma.
Time frame: 5 years
To evaluate acute and subacute toxicities of proton radiotherapy in place of photon radiation for patients with neuroblastoma. Specifically, nausea, vomiting, diarrhea, radiation pneumonitis and skin toxicity will be described.
2 secondary outcomes
Progression free survival
Time frame: 10 years
Overall survival rates
Time frame: 10 Years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2026.
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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.
Shannon M. MacDonald, MD principal investigator
Massachusetts General Hospital
1 site 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 Therapy in people with Neuroblastoma and Ganglioneuroblastoma, run by Massachusetts General Hospital at 1 site 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 8 inclusion and 4 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: under way, not looking for participants, as of the sponsor's last update on 20 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
This study is still under way. Results are not available in the source record yet.
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?
Describe late complications of radiation therapy delivered with proton radiotherapy in place of photon radiation for patients with neuroblastoma (time frame: 5 years). 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?
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 20 Aug 2026.