Safety and Efficacy of Vertebral Body-Sparing Craniospinal Irradiation With Proton Therapy in Pediatric Tumors

his is a single-center, prospective observational study in children and adolescents with central nervous system tumors who need whole-brain and whole-spine radiation therapy (craniospinal irradiation, CSI). The study uses proton therapy with a special vertebral body-sparing (VBS) technique to protect the front and center of the vertebrae, which helps preserve bone marrow function and growth. The main goals are to find safe dose limits for the vertebrae and check how often severe side effects occur. The study will also look at bone marrow preservation, spinal deformity, tumor control, survival, chemotherapy completion, neurocognitive function, quality of life, and growth and development for up to 5 years after treatment.

Not Yet Recruiting

Trial opening soon.

Get Notified

Key information

Conditions

Pediatric Central Nervous System Tumors Atypical Teratoid/Rhabdoid Tumor (AT/RT) Ependymoma Germinoma Glioma Medulloblastoma Neoplasms Neoplasms by Histologic Type Neoplasms, Complex and Mixed Neoplasms, Germ Cell and Embryonal Neoplasms, Glandular and Epithelial Neoplasms, Nerve Tissue Neoplasms, Neuroepithelial Neuroectodermal Tumors Neuroectodermal Tumors, Primitive Rhabdoid Tumor

Age range

4 year–18 year

Sex eligibility

All sexes

Study type

Observational

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

The patient's legal guardian voluntarily signs the written informed consent form.
Age at diagnosis > 3 years and ≤ 18 years.
Histopathologically confirmed diagnosis of a central nervous system (CNS) tumor.
Indication for craniospinal irradiation (CSI) confirmed by multidisciplinary team (MDT) discussion.
Karnofsky Performance Score (KPS) ≥ 80.
For postoperative patients, complete healing of the surgical incision with no evidence of wound infection or other complications.
For women of childbearing potential, effective contraception must be used for at least one month prior to screening, and they must commit to continued contraception throughout the study period and for the required time after study completion.

Exclusion criteria

Previous receipt of any form of spinal radiotherapy.
Previous major spinal surgery for any reason.
Presence of clinically significant and confirmed spinal deformity at enrollment (e.g., Cobb angle > 10° measured by MRI or X-ray).
Presence of medical contraindications to proton radiotherapy.
Presence of any severe, uncontrolled systemic disease (e.g., active infection, severe cardiac, hepatic, renal, or respiratory insufficiency) that, in the investigator's judgment, makes the subject unsuitable for treatment under this protocol.
Female patients who are pregnant or breastfeeding.
Presence of any severe cognitive or psychiatric disorder that prevents understanding of or cooperation with study assessments.

Treatment and study plan

Vertebral Body-Sparing Craniospinal Proton Irradiation

Radiation

Vertebral body-sparing craniospinal irradiation (VBS-CSI) using intensity-modulated proton therapy (IMPT, pencil-beam scanning). The treatment actively restricts radiation dose to the anterior and central regions of thoracolumbar vertebral bodies to preserve active bone marrow and spinal growth potential, while ensuring adequate target coverage for the whole brain and spinal cord. Two dose levels are applied: 23.4 Gy(RBE)/13 fractions or 24 Gy(RBE)/15 fractions and 36 Gy(RBE)/20 fractions.

Primary outcomes

Incidence of ≥ Grade 3 acute adverse events

Time frame: Up to 3 months after completion of radiotherapy

To evaluate the incidence of treatment-related acute adverse events of grade 3 or higher, assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0 (CTCAE v5.0).

Secondary outcomes

Active Bone Marrow Preservation Rate

Time frame: Baseline, 3, 6, 12, 24, 36, 48, 60 months

The proportion of active bone marrow in the vertebral body is calculated by dividing the width of the low-signal area by the total diameter of the vertebral body, which allows a relatively quantitative assessment of the active bone marrow preservation rate.
Incidence of spinal deformity

Time frame: Baseline and at 6, 12, 18, 24, 30, 36, 42, 48, 54, and 60 months after treatment completion

Evaluation is performed using whole-neuraxis MRI or whole-spine anteroposterior and lateral X-ray films. Spinal scoliosis is quantitatively assessed using the Cobb angle measurement method by a radiologist who is blinded to the patient's specific treatment information. The degree of kyphosis is evaluated by assessing the presence of thoracic kyphosis. Additionally, vertebral body change characteristics of the thoracolumbar spine are measured, including the posterior-to-anterior ratio (PARs) of the vertebral body-defined as the ratio of the posterior vertebral body height to the anterior vertebral body height-as well as intervertebral disc height.

Study contacts

Contact information is provided by the study sponsor or research team.

Jiayi Chen

CONTACT

[email protected]

+86 021 64370045

Sponsors and collaborators

Lead sponsor

Ruijin Hospital

Other

Registry information

Official study title

Safety and Efficacy of Vertebral Body-Sparing Craniospinal Irradiation (VBS-CSI) With Proton Therapy in Pediatric Tumors: A Single-Center Prospective Observational Cohort Study

Acronym: VBS-CSI-PT-PED

Important dates

Study start: 2026
Primary completion: 2029
Study completion: 2033
First posted: May 15, 2026
Registry last updated: May 15, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed