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CompletedNCT01445288Updated Sep 22, 2026

Exploratory Study of Effects of Radiation Therapy in Pediatric Patients With Central Nervous System Tumors

An observational study in Diffuse Intrinsic Pontine Glioma, PNET and Ependymoma, sponsored by National Cancer Institute (NCI). Completed at 1 site in United States. Open to participants aged 1 Month to 21 Years. Per ClinicalTrials.gov, last updated 2026-09-22.

Sponsored by National Cancer Institute (NCI) · Observational

Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
76
Ages
1 Month to 21 Years
Sex
All
01

Study summary

This study will analyze the effects of radiation given to children who have tumors of the central nervous system (CNS). Researchers want to learn more about changes in the quality of life that patients may experience as a result of radiation.

Patients ages 21 and younger who have a primary CNS tumor and who have not received radiation previously may be eligible for this study. They will have a medical history and physical examination. Collection of blood (about 2-1/2 tablespoons) and urine will be done, as well as a pregnancy test. Patients will complete neuropsychological tests, which provide information about their changes in functioning over time. An expert in psychology will give a number of tests, and the patient's parents or guardian will be asked to complete a questionnaire about the patient's behavior. Also, patients will be given a quality of life questionnaire to complete and vision and hearing tests. The radiation itself is prescribed by patients' doctors and is not part of this study.

Magnetic resonance imaging (MRI) will give researchers information about the tumor and brain, through several scanning sequences . MRI uses a strong magnetic field and radio waves to obtain images of body organs and tissues. Patients will lie on a table that slides into the enclosed tunnel of the scanner. They will need to lie still, and medication may be given to help them to do that. They may be in the scanner for up to 2 hours. As the scanner takes pictures, patients will hear knocking or beeping sounds, and they will wear earplugs to reduce the noise. A contrast agent will be administered, to allow images be seen more clearly. Blood and urine tests will be conducted after the first dose of radiation. MRI scans will be done 2 weeks after patients finish radiation therapy and again at 6 to 8 weeks, 6 months, 12 months, and yearly. Also at those follow-up periods, patients will undergo similar procedures as previously, including blood and urine tests and neuropsychological testing. Patients can remain in this study for 5 years.

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Read the detailed description

Background:

This exploratory study will be performed in pediatric patients with CNS tumors who are undergoing radiation therapy to investigate pathophysiologic effects of radiation on the CNS. The study includes the analysis of blood, urine, and CSF (if available) to measure biological markers involved with angiogenesis, blood: brain barrier integrity, and neurotoxicity. It also entails comprehensive MR imaging techniques and neuropsychological testing in an effort to correlate changes with biomarker measurements.

Objectives:

  1. To detect changes in angiogenesis related to radiation of the CNS by:

    • Measurement of VEGF, bFGF, thrombospondin, TNF-alpha, IL-12, IL-8, and MMP in blood and urine specimens.
    • MR perfusion and DEMRI.
  2. To describe changes in blood:brain barrier permeability associated with radiation of the CNS.
  3. To characterize neurotoxicity by:

    • Measuring biomarkers associated with neurotoxicity
    • Documenting changes in neurobehavioral functioning through longitudinal comprehensive assessments
    • Describing changes in quality of life (QOL)
    • Assessing changes in memory
    • Defining changes in ophthalmologic studies associated with radiation.
    • Detecting changes in audiometry associated with radiation.

Eligibility Criteria:

  • Patients must have a primary CNS tumor for which radiation therapy is recommended.
  • Patients must be less than or equal to 21 years of age.
  • Prior/Concurrent: Patients will be eligible if they have not received prior radiation. Patients who have undergone surgery or received chemotherapy are eligible.
  • Performance Status: Patients will be eligible regardless of performance score.

Design:

This minimally invasive study is designed to explore various biologic effects of radiation on the pediatric CNS in an attempt to 1) obtain information on the pathophysiology of radiation-induced damage, 2) explore the association of neuropsychological deficits with biologic markers and neuroimaging abnormalities, 3) document changes in neurobehavioral functioning through longitudinal comprehensive neuropsychological assessments with comparison of various radiation therapy techniques, 4) describe changes in quality of life in pediatric patients who have received radiation therapy, and 5) attempt to identify children at increased risk of radiation-induced neurotoxicity.

02

Conditions studied

  • Diffuse Intrinsic Pontine Glioma
  • PNET
  • Ependymoma
  • Germinoma
  • Medulloblastoma

Keywords

  • Neurotoxicity
  • Brain Tumor
  • Children
  • Angiogenesis
  • Imaging
  • Natural History
  • Central Nervous System Tumor
  • CNS Tumor
03

Who can participate

Ages eligible
1 Month to 21 Years
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

Pediatric patients 21 years of age or younger with CNS tumors for which radiation therapy is recommended@@@

Inclusion criteria

Age: Patients must be less than or equal 21 years of age.

Tumor: Any primary CNS tumor.

Referred for radiation therapy at NCI.

Signed informed consent by patient, parent or legal guardian.

PERFORMANCE SCORE: any.

PRIOR/CONCURRENT THERAPY: Patients will be eligible if they have not received prior radiation.

Patients who have undergone prior surgery or who have received chemotherapeutic regimens are eligible.

Exclusion criteria

EXCLUSION CRITERIA:

Patients who have received prior radiation.

Patients who are unable to have MRI performed for any reason.

04

Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
76 participants (actual)

Groups and cohorts

  • 1

    Pediatric Patients with Central Nervous System Tumors

05

What researchers measure

Primary outcomes

  1. Measure changes in antiogenesis, blood brain barrier permability and neurotoxicity re to radiation of the CNS

    Measurement of VEGF, bFGF, thrombospondin, TNF-a, IL-12, IL-8, and MMP in blood and urine specimens

    Time frame: before and up to 8 years after

Secondary outcomes

  1. Describe changes in imaging, endocrine function after xrt to the brain

    Measurement of S100-beta and transthyretin in blood

    Time frame: before and up to 8 years after

  2. Monitor changes in serum proteome and germline polymorphisms

    Assesed changed in memory, QOL, neurobehavioral functioning, ophthalmologic studies, and audiometry associated with radiation

    Time frame: before and up to 8 years after

06

Study locations

1 site
  • National Institutes of Health Clinical Center
    Bethesda, Maryland 20892, United States
07

References and documents

Publications

  • Garcia-Barros M, Paris F, Cordon-Cardo C, Lyden D, Rafii S, Haimovitz-Friedman A, Fuks Z, Kolesnick R. Tumor response to radiotherapy regulated by endothelial cell apoptosis. Science. 2003 May 16;300(5622):1155-9. doi: 10.1126/science.1082504. PubMed 12750523 ↗
  • Wyllie AH, Kerr JF, Currie AR. Cell death: the significance of apoptosis. Int Rev Cytol. 1980;68:251-306. doi: 10.1016/s0074-7696(08)62312-8. No abstract available. PubMed 7014501 ↗
  • Mulhern RK, Hancock J, Fairclough D, Kun L. Neuropsychological status of children treated for brain tumors: a critical review and integrative analysis. Med Pediatr Oncol. 1992;20(3):181-91. doi: 10.1002/mpo.2950200302. PubMed 1574027 ↗

Individual participant data

Plan to share: Yes — All IPD recorded in the medical record will be shared with intramural investigators upon request.

Supporting information: Study protocol, Sap, Icf

08

Registry details

Key details

Study ID
NCT01445288
Lead sponsor
National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
Oct 3, 2011
Start date
Dec 5, 2006
Last update
Sep 22, 2026

Study contacts

John W Glod, M.D.
principal investigator · National Cancer Institute (NCI)

Oversight

FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.

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