Proton Craniospinal Irradiation With Bone Sparing to Decrease Growth Decrement From Radiation

Craniospinal Irradiation Using Proton Beam Scanning With Selective Vertebral Body/Bone Sparing to Improve Marrow Reserve and Decrease Growth Decrement for Children

This research study is studying proton radiation as a possible treatment for brain tumor that requires radiation.

The radiation involved in this study is:

-Proton Radiation

Study Overview

• Status: Active, not recruiting
• Conditions: Brain Tumor
• Intervention / Treatment: Radiation: Proton Beam

Detailed Description

This research study is a Pilot Study to determine whether using proton therapy in participants that require craniospinal radiation (whole brain and spinal cord radiation therapy) with sparing of the bony spine will work. This is the first time investigators are examining bone sparing proton therapy in pediatric craniospinal radiation.

The FDA (the U.S. Food and Drug Administration) has not approved proton radiation for this specific disease but it has been approved for other uses.

In this research study, the investigators are studying proton radiation in participants that require craniospinal radiation. The standard of care for this procedure is photon radiation, which is very similar to proton radiation. The investigators believe that the precision of proton radiation may help to reduce the negative effects radiation has on the surrounding non-cancerous growing and developing tissue

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02214
      • Massachusetts General Hospital
  • Texas
    • Houston, Texas, United States, 77030
      • MD Anderson Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Age ≥ 3 years and ≤ 18 years at the time of registration
• Histologically proven malignancy necessitating cranio-spinal irradiation. This will include patients with a diagnosis of medulloblastoma, Supratentorial primitive neuroectodermal tumor (SPNET), germ cell tumor (GCT), disseminated ependymoma, embryonal tumor with abundant neuropil and true rosettes (ETANTR), Atypical Teratoid/Rhabdoid Tumor (ATRT), and disseminated low-grade glioma (LGG).
• Life expectancy ≥ 12 months.
• Signed informed consent document and assent when appropriate.
• HGB of > 10 g/L and PLT count > 80 K/uL

Exclusion Criteria:

• Any prior therapeutic radiation therapy > 500 cGy has been delivered.
• Individuals with a history of a different malignancy are ineligible except for the following circumstances: if they have been disease-free for at least 5 years and are deemed by the investigator to be a low-risk for recurrence of that malignancy; or, have had only cervical cancer in situ, or basal cell or squamous cell carcinoma of the skin.
• Any major uncontrolled or poorly controlled intercurrent illness that would limit compliance with study requirements.
• Pregnant females are excluded. Females of childbearing age/menstruating must confirm that either they are not sexually active or have a negative pregnancy test prior to initiation of radiation therapy.
• Patients that receive concurrent chemotherapy with the exception of concurrent Vincristine.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Proton Radiotherapy; Patients will be treated with Proton Beam once daily 5 days per week.; Doses will be prescribed such that maximum possible coverage is achieved	Radiation: Proton Beam; precision radiation that reduce the negative effects radiation has on the surrounding non-cancerous growing and developing tissue

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Feasibility of cranio-spinal irradiation (CSI) utilizing intensity-modulated proton therapy (IMPT) with pencil beam scanning (PBS) for vertebral body sparing (VBS) in pediatric patients.	IMPT using PBS delivery will be considered to be feasible as a VBS technique for CSI in pediatric patients if the rate of grade 3/4 hematologic toxicity were no more than 5% within 3 months after the completion of radiation treatment, within the range associated with conventional techniques of CSI. Toxicity will be assess using Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0.	3 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Early marrow changes in the vertebral bodies	Summary of early marrow changes in the vertebral bodies by Magnetic resonance imaging (MRI) of the spine during radiation therapy (RT) by comparing one study MRI to the baseline pre-RT MRI.	2 years
Change in sitting height and standing height	Summary of the changes in the participants sitting and standing heights.	Annually for 5 years
Time to abnormality in spinal curvature	Participants are monitored both clinically and by MRI for any abnormality in spinal curvature. The time to abnormality in spine curvature will be measured from the start of radiation to the date of documented abnormality or censored at the date of last follow-up for patients still alive with normal spine.	5 years
Disease Free Survival	Disease-free survival is measured from the start of radiation to the date of progressive disease based on imaging studies obtained as standard care or to the date of death due to any cause, whichever is earlier. Disease-free survival will be estimated using the Kaplan-Meier method. Patients without progressive disease and still alive will be censored at their date of last contact.	5 years
Pattern of Disease Relapse	Summary of the sites where the cancer relapses. Relapse is the regrowth of cancer cells.	2 years
Complete blood counts (CBC) over time	Summary of the change in complete blood counts (CBC) over time as determined by yearly blood samples.	Annually for 5 years
Change in levels of vitamin D, calcium, and growth hormones over time	Summary of the changes in blood levels of vitamin D, calcium, and growth hormones.	Annually for 5 years
Weight	Summary of the changes in weight of the participants as assessed by yearly evaluations.	Annually for 5 years
BMI	Summary of the body mass index (BMI) of the participants as assessed by yearly physical evaluations.	Annually for 5 years
Vertebral column growth (measured by MRI) over 1 year	Summary of vertebral column growth (measured by MRI) over 1 year following vertebral body sparing cranio-spinal irradiation in pediatric patients.	Last day of treatment to 1 year later

Collaborators and Investigators

• Sponsor: Massachusetts General Hospital
• Collaborators: National Cancer Institute (NCI)
• Investigators: Principal Investigator: Shannon MacDonald, MD, Massachusetts General Hospital

Study record dates

Study Major Dates

• Study Start (Actual): January 4, 2018
• Primary Completion (Estimated): June 30, 2027
• Study Completion (Estimated): December 31, 2027

Study Registration Dates

• First Submitted: September 8, 2017
• First Submitted That Met QC Criteria: September 11, 2017
• First Posted (Actual): September 13, 2017

Study Record Updates

• Last Update Posted (Actual): March 19, 2026
• Last Update Submitted That Met QC Criteria: March 16, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: Brain Tumor
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neoplasms by Site; Neoplasms; Nervous System Neoplasms; Central Nervous System Neoplasms; Brain Neoplasms; Therapeutics; Radiotherapy; Heavy Ion Radiotherapy; Proton Therapy
• Other Study ID Numbers: 17-283; U19CA021239-37 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: Yes
• product manufactured in and exported from the U.S.: No