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

NCT06907485 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: STUDY22120058
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 2

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 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.

Conditions

Pediatric Brain Tumor

Keywords

Aminolevulinic Acid (ALA); Gleolan®; Fluorescence-Guided Resection; Brain Tumor; Pediatric

Outcome Measures

Primary Outcomes (1)

• 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.

  Day of Surgery

Secondary Outcomes (6)

• Frequency/rate of positive fluorescence within each tumor type

  Day of Surgery

• Fluorescence quality in all patients receiving 5-ALA and within each tumor type

  Day of Surgery

• Accrual rate for each tumor type

  Day of Surgery

• Determination of the sensitivity, specificity, positive predictive value (PPV), and negative predictive value (NPV) of tumor fluorescence.

  Day of Surgery

• Frequency/rate of AE in all patients receiving ALA and for each tumor type

  Day of Consent signing, day of surgery; within 48 hours of surgery; 2-weeks post-surgery, 6-weeks post-surgery, 3-months post-surgery, 6-months post-surgery

Study Arms (1)

Aminolevulinic acid hydrochloride

EXPERIMENTAL

Single arm study administering a single oral solution of aminolevulinic acid hydrochloride at a dose of 20 mg/kg body weight, 6-12 hours prior to brain surgery for resection of the tumor.

Drug: Aminolevulinic acid hydrochloride

Interventions

Aminolevulinic acid hydrochloride DRUG

Pediatric 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.

Also known as: Gleolan®, ALA HCl, 5-ALA, Aminolevulinic acid HCl, Aminolevulinic acid

Aminolevulinic acid hydrochloride

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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
• Creatinine Clearance \>60 mL/min/1.73 m2 for patients with creatinine levels above institutional age-specific normal as defined per institution.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Costas Hadjipanayis: lead
• 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

Study Sites (2)

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

Chicago, Illinois, 60068, United States

Location

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

MeSH Terms

Conditions

Brain Neoplasms

Interventions

Aminolevulinic Acid

Condition Hierarchy (Ancestors)

Central Nervous System Neoplasms; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

Levulinic Acids; Keto Acids; Carboxylic Acids; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins

Study Officials

• Constantinos G. Hadjipanayis, MD, PhD

  University of Pittsburgh

  STUDY DIRECTOR

• John Ruge, MD, FAANS

  Midwest Children's Brain Tumor Center, Advocate Children's Hospital-Park Ridge

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Kristy Boggs, PhD

CONTACT

412-647-8952; boggskd@upmc.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: L. Dade Lunsford Professor of Neurosurgery, Executive Vice Chairman

Study Record Dates

• First Submitted: March 26, 2025
• First Posted: April 2, 2025
• Study Start (Estimated): August 1, 2026
• Primary Completion (Estimated): February 1, 2028
• Study Completion (Estimated): August 1, 2028
• Last Updated: January 21, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)