This clinical trial focuses on pediatric patients aged 2 up to 18 years of age with a new or recurrent pediatric brain tumor, suspected to be either a high-grade or low-grade glioma, and scheduled for surgical removal. 5-aminolevulinic acid (5-ALA) is FDA-approved for improving brain tumor visualization in adults during surgery through fluorescence, enabling more complete removal of the tumor. This study aims to evaluate the feasibility of administering 5-ALA to pediatric brain tumor patients and to assess the quality of tumor fluorescence during surgery in this patient population.
For the clinical trial, the patient will orally ingest 5-ALA 6 to 12 hours before brain surgery. All study participants will be provided standard medical care for removal of the brain tumor. All children enrolled in the study will be closely monitored prior to, during, and after surgery to ensure there are no reactions to the study drug. 5-ALA can make the patient more sensitive to sunlight and direct indoor lighting, referred to as photosensitivity, and can cause a sunburn-type reaction. It is for this reason that patients will be kept in subdued light conditions for 48 hours following surgery. Study participation starts once the patient is enrolled in the study until 6-month post-surgery.
Subjects included in this trial must have had an MRI documentation of "a new or recurrent primary pediatric brain tumor" for which resection is indicated and has been planned.
The anticipated histology at resection will be "newly diagnosed" or "recurrent": pilocytic astrocytoma, pleomorphic xanthoastrocytoma (PXA), ganglioglioma, diffuse neuroepithelial tumor (DNET), astrocytoma, oligodendroglioma, ependymoma, anaplastic astrocytoma, anaplastic oligodendroglioma, anaplastic ependymoma, anaplastic ganglioglioma, anaplastic PXA, PNET, ATRT, or medulloblastoma.
Male or Female Age 2-17 years and 182 days
The effects of 5-ALA on the developing human fetus are unknown. A pregnancy test will be performed for all young women of childbearing ability prior to surgery (see
Ability for patient/patient's guardian to understand and the willingness to sign a written informed consent document. In appropriate cases, assent of pediatric patients will be obtained. Translation will be provided as appropriate.
Inclusion of Women and Minorities: Both males and females and members of all ethnic groups are eligible for this trial.
Patients who meet inclusion criteria but are unable to swallow 5-ALA solution.
Patients with radiographic tumors of the brain stem as assessed by MRI.
Single group
Aminolevulinic acid hydrochloride
DrugPediatric patients diagnosed with a new or recurrent brain tumor, suspected to be either a high-grade or low-grade glioma, and scheduled for surgical removal will receive aminolevulinic acid hydrochloride prior to surgery for resection of the brain tumor.
Overall frequency/rate of positive fluorescence in all patients that receive 5-ALA
Overall frequency/rate of positive fluorescence, which is defined as patients with at least one tissue sample with positive fluorescence (score of at least weak and patchy) after using 5-ALA, in all patients administrated 5-ALA with a suspected primary or recurrent brain tumor that is considered to be a low grade (WHO grade I/II) or high grade (WHO grade III/IV) glioma.
Frequency/rate of positive fluorescence within each tumor type
Presence or absence of fluorescence in each tumor type.
Fluorescence quality in all patients receiving 5-ALA and within each tumor type
Fluorescence will be categorized as 'none,' 'weak,' 'moderate,' or 'strong,' and described as 'patchy' or 'uniform.' If fluorescence is present, surgery will be guided by white light with appropriate filters to excite the tumor tissue. Intraoperative photos will capture tissue fluorescence. Biopsies will be taken, including 3-6 additional research specimens per patient for histopathologic analysis. If fluorescence is present, three fluorescent samples will be collected from the tumor core, margin, and areas near the margin, along with three non-fluorescent samples from the resection cavity. If no fluorescence is seen, only non-fluorescent samples from the tumor core, margin, and surrounding areas will be collected. All samples will undergo histopathological examination by a neuropathologist for WHO grade, tumor type, and cell count. Genetic and molecular analysis data will be collected if available.
Accrual rate for each tumor type
Histopathology assessment will characterize the standard of care biopsies by WHO grade and tumor type. If institutions perform routine genetic and molecular analysis of resected tissue, this data will also be collected.
Determination of the sensitivity, specificity, positive predictive value (PPV), and negative predictive value (NPV) of tumor fluorescence.
Sensitivity of 5-ALA induced fluorescence is calculated as proportion of all tumor (confirmed by histopathology analysis) specimens with positive fluorescence. Specificity is the proportion of all tumor (confirmed by histopathology analysis) specimens without the presence of fluorescence. Positive predictive value (PPV) is defined as proportion of tissues with 5-ALA induced fluorescence that were confirmed tumor on histopathology. Negative predictive value (NPV) is the proportion of tissues without fluorescence that were confirmed tumor by histopathology. In addition PPV will also be calculated with the proportion of tissues with no visible 5-ALA-induced fluorescence but with detectable levels of fluorescence through novel detection system that were confirmed malignant on pathology.
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Costas Hadjipanayis
Lead sponsor
University of Pittsburgh
Sponsor institution
NX Development Corp
Collaborator
Southeastern Brain Tumor Foundation
Collaborator
StacheStrong Foundation
Collaborator
Neuroscience Research Foundation
Collaborator
University of Pittsburgh Medical Center
Collaborator
Advocate Hospital System
Collaborator
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