A Multicenter Study to Assess the Feasibility of 5-Aminolevulinic Acid (5-ALA) in Pediatric Brain Tumor Patients

Launched by COSTAS HADJIPANAYIS · Mar 26, 2025

Keywords

ClinConnect Summary

This clinical trial is designed to explore the use of a medication called 5-aminolevulinic acid (5-ALA) in children and teens aged 2 to 18 who have a brain tumor that needs to be surgically removed. The goal is to see if 5-ALA can help doctors better see and remove the tumor during surgery by making it glow. While 5-ALA is already used in adults, this study will determine how safe and effective it is for younger patients.

To participate, children must have a brain tumor confirmed by an MRI and be scheduled for surgery to remove it. Participants will take 5-ALA in a drink 6 to 12 hours before their surgery, and their health will be closely monitored throughout the process to ensure their safety. After surgery, they will need to stay in low-light conditions for about 48 hours to avoid sensitivity to light, which is a side effect of the medication. This trial is not yet recruiting participants, but it aims to improve treatment options for pediatric brain tumors in the future.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. 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.
• • 2. 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.
• • 3. Male or Female Age 2-17 years and 182 days
• 4. Subjects must have normal organ and marrow function as defined below:
• • Leukocytes \>3,000/mL Platelets \>100,000/mL Total bilirubin below upper limit of normal AST (SGOT)/ALT (SGPT) GGT \<2.5 X institutional upper limit of normal Creatinine below upper limit of normal
• • OR
• • Creatinine clearance \>60 mL/min/1.73 m2 for patients with creatinine levels above institutional normal as defined per institution.
• • 5. 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 Exclusion Criteria below). Should a young woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
• • 6. 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.
• • 7. Inclusion of Women and Minorities: Both males and females and members of all ethnic groups are eligible for this trial.
• Exclusion Criteria:
• • 1. Patients who meet inclusion criteria but are unable to swallow 5-ALA solution.
• • 2. Patients with radiographic tumors of the brain stem as assessed by MRI.
• • 3. History of allergic reactions attributed to compounds of similar chemical or biologic composition to 5-ALA. Patients should refrain from use of other potential phototoxic substances (e.g., tetracyclines, sulfonamides, fluoroquinolones, hypericin extracts) for 24 h.
• • 4. Personal or family history of porphyria.
• • 5. Uncontrolled concurrent illness including, but not limited to: ongoing or active infection, heart disease, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with 5-ALA breastfeeding should be discontinued if the mother is treated 5-ALA.
• • 6. Young women who are pregnant or become pregnant will be excluded from the trial as it is unknown if 5-ALA is teratogenic or has abortifacient effects. A pregnancy test will be performed on all young women who are s/p menstruation prior to entering study.
• • 7. Prior history of GI perforation, diverticulitis, and/or peptic ulcer disease.
• • 8. Patients who meet inclusion criteria but are unable to swallow 5-ALA solution.