Gleolan for Brain Tumor

Recruiting at 1 trial location

Overseen By Kristy Boggs, PhD

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Costas Hadjipanayis

Must not be taking: Tetracyclines, Sulfonamides, Fluoroquinolones, others

Disqualifiers: Brain stem tumors, Porphyria, Pregnancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 3

20 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you should avoid using other substances that can make you sensitive to light, like certain antibiotics and herbal extracts, for 24 hours before taking the study drug.

What data supports the effectiveness of the drug 5-Aminolevulinic Acid (5-ALA) for brain tumors?

Research shows that 5-ALA helps surgeons see high-grade brain tumors more clearly during surgery, leading to better removal of the tumor and longer periods without the cancer getting worse. ^{1 2 3 4 5}

Is 5-ALA (Gleolan) safe for use in brain tumor surgeries?

5-ALA (Gleolan) has been used safely in both adults and children for brain tumor surgeries, with no reported side effects like blood or skin issues in the studies reviewed. It is approved for use in adults and has shown to be safe in children, although its use in children is still considered off-label and needs more research. ^{2 6 7 8 9}

How is the drug 5-ALA unique in treating brain tumors?

5-ALA is unique because it helps surgeons see brain tumors more clearly during surgery by making the tumor cells glow under a special light, which can lead to more complete removal of the tumor and potentially better outcomes for patients. ^{2 3 10 11 12}

Research Team

John Ruge, MD, FAANS

Principal Investigator

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

Constantinos G. Hadjipanayis, MD, PhD

Principal Investigator

University of Pittsburgh

Eligibility Criteria

This trial is for children aged 2-18 with a new or recurrent brain tumor, suspected to be high-grade or low-grade glioma, who are scheduled for surgical removal. Participants must not have any conditions that would interfere with the study drug or surgery.

Inclusion Criteria

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

My child's MRI shows a new or returning brain tumor that needs surgery.

My brain tumor is one of several specific types, either new or recurring.

Exclusion Criteria

History of allergic reactions attributed to compounds of similar chemical or biologic composition to 5-ALA

Personal or family history of porphyria

I cannot swallow the 5-ALA solution required for the trial.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Pre-Surgery Preparation

Participants receive an oral solution of 5-ALA 6-12 hours prior to brain surgery for tumor resection

1 day

1 visit (in-person)

Surgery and Immediate Post-Surgery

Surgical resection of the brain tumor with fluorescence guidance; patients are kept in subdued light conditions for 48 hours post-surgery due to photosensitivity

2 days

Inpatient stay

Follow-up

Participants are monitored for safety and effectiveness after treatment, including physical exams, neurological assessments, and MRIs

6 months

Visits at 2 weeks, 6 weeks, 3 months, and 6 months post-surgery

Treatment Details

Interventions

• 5-Aminolevulinic Acid (5-ALA)

Trial Overview The trial tests the use of Gleolan (5-ALA), which helps surgeons see brain tumors more clearly during surgery by making them glow. It's already used in adults and this study will check if it's also feasible and helpful in kids.

Participant Groups

Group I: Aminolevulinic acid hydrochloride Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Costas Hadjipanayis

Lead Sponsor

Southeastern Brain Tumor Foundation

Collaborator

NX Development Corp

Industry Sponsor

Trials

4

Recruited

330+

Neuroscience Research Foundation

Collaborator

University of Pittsburgh Medical Center

Collaborator

Trials

78

Recruited

77,600+

Advocate Hospital System

Collaborator

Trials

6

Recruited

490+

StacheStrong Foundation

Collaborator

References

1. United States pubmed.ncbi.nlm.nih.gov

A Phase 1 Dose-Escalation Study of Oral 5-Aminolevulinic Acid in Adult Patients Undergoing Resection of a Newly Diagnosed or Recurrent High-Grade Glioma. [2018]

2. United States pubmed.ncbi.nlm.nih.gov

Fluorescence-guided surgery for high-grade gliomas. [2018]

3. United States pubmed.ncbi.nlm.nih.gov

5-Aminolevulinic Acid-Guided Resection in Grade III Tumors-A Comparative Cohort Study. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

5-Aminolevulinic Acid-Guided Surgery for Recurrent Supratentorial Pediatric Neoplasms. [2023]

5. England pubmed.ncbi.nlm.nih.gov

Outcomes after combined use of intraoperative MRI and 5-aminolevulinic acid in high-grade glioma surgery. [2018]

6. England pubmed.ncbi.nlm.nih.gov

5-ALA fluorescence-assisted surgery in pediatric brain tumors: report of three cases and review of the literature. [2015]

7. United States pubmed.ncbi.nlm.nih.gov

Safety and Efficacy of 5-Aminolevulinic Acid for High Grade Glioma in Usual Clinical Practice: A Prospective Cohort Study. [2019]

8. Austria pubmed.ncbi.nlm.nih.gov

Predicting the "usefulness" of 5-ALA-derived tumor fluorescence for fluorescence-guided resections in pediatric brain tumors: a European survey. [2021]

9. United States pubmed.ncbi.nlm.nih.gov

5-ALA and FDA approval for glioma surgery. [2020]

10. Spain pubmed.ncbi.nlm.nih.gov

Observational, retrospective study of the effectiveness of 5-aminolevulinic acid in malignant glioma surgery in Spain (The VISIONA study). [2018]

11. Austria pubmed.ncbi.nlm.nih.gov

Comparison of commercial 5-aminolevulinic acid (Gliolan®) and the pharmacy-compounded solution fluorescence in glioblastoma. [2020]

12. England pubmed.ncbi.nlm.nih.gov

Turning on the light for brain tumor surgery: A 5-aminolevulinic acid story. [2022]