Status:

NOT_YET_RECRUITING

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

Lead Sponsor:

Costas Hadjipanayis

Collaborating Sponsors:

NX Development Corp

Southeastern Brain Tumor Foundation

Conditions:

Pediatric Brain Tumor

Eligibility:

All Genders

2-17 years

Phase:

PHASE2

Brief Summary

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...

Detailed Description

The aim of this study is to assess the feasibility of administering 5-ALA pre-operatively and to access if the resulting 5-ALA-induced tumor fluorescence is adequate for fluorescence-guided brain tumo...

Eligibility Criteria

Inclusion

  • 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
  • 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 (ULN) AST (SGOT)/ALT (SGPT)/ GGT \<2.5 X institutional age-specific ULN Creatinine Below ULN
  • OR
  • Creatinine Clearance \>60 mL/min/1.73 m2 for patients with creatinine levels above institutional age-specific normal as defined per institution.
  • 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.
  • 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.

Exclusion

  • 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.
  • 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.
  • Personal or family history of porphyria.
  • 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.
  • 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.
  • Prior history of GI perforation, diverticulitis, and/or peptic ulcer disease.

Key Trial Info

Start Date :

December 1 2025

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

December 1 2027

Estimated Enrollment :

20 Patients enrolled

Trial Details

Trial ID

NCT06907485

Start Date

December 1 2025

End Date

December 1 2027

Last Update

July 17 2025

Active Locations (2)

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Page 1 of 1 (2 locations)

1

MidWest Children's Brain Tumor Center, Advocate Children's Hospital Park Ridge

Chicago, Illinois, United States, 60068

2

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States, 15224