Gleolan for Brain Tumor

Not yet 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a drug called 5-ALA (Gleolan) to determine if it helps surgeons better visualize brain tumors in children during surgery. 5-ALA is already used for this purpose in adults, and the goal is to assess its effectiveness in children. The trial seeks children with a new or recurring brain tumor, scheduled for surgery, and who can swallow the medication. Participants will take 5-ALA before surgery and must avoid bright lights for a couple of days afterward to prevent skin reactions. This research could enhance the effectiveness of brain tumor removal in young patients. As a Phase 2 trial, it focuses on measuring the treatment's effectiveness in an initial, smaller group, allowing participants to contribute to significant advancements in pediatric brain tumor 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.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that 5-aminolevulinic acid (5-ALA) is approved by the FDA to help doctors see brain tumors more clearly during surgery in adults, indicating its general safety. Previous studies have investigated the use of 5-ALA for children and teenagers with brain tumors to ensure its safety for them as well.

5-ALA can increase skin sensitivity to light, potentially causing a reaction similar to sunburn. To prevent this, patients typically avoid bright lights for a period after taking the drug. Overall, studies have found 5-ALA to be well-tolerated, with careful monitoring to manage any possible side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for brain tumors?

Unlike the standard treatments for brain tumors, which often involve surgery, radiation, and chemotherapy, 5-Aminolevulinic Acid (5-ALA) offers a unique approach by enhancing tumor visualization during surgery. This treatment is administered as an oral solution and helps surgeons better distinguish tumor tissue from healthy brain tissue by causing the tumor cells to fluoresce under a special light. This precise targeting could potentially improve surgical outcomes by ensuring more complete removal of the tumor while sparing healthy tissue. Researchers are excited because it could lead to fewer postoperative complications and better overall survival rates for patients.

What evidence suggests that 5-ALA is effective for improving brain tumor visualization in pediatric patients?

Studies have shown that 5-aminolevulinic acid (5-ALA) helps doctors see brain tumors more clearly during surgery. Research indicates that it can lead to more complete removal of tumors, especially in aggressive brain tumors called high-grade gliomas. In one study, patients who underwent surgery with 5-ALA had a higher rate of complete tumor removal compared to those who did not use it. Another study found that patients treated with 5-ALA lived longer on average than those who had surgery with standard lighting. Overall, 5-ALA is considered safe and effective for improving tumor visibility, which can lead to better surgical outcomes.

In this trial, participants will receive a single oral solution of aminolevulinic acid hydrochloride at a dose of 20 mg/kg body weight, 6-12 hours before brain surgery for tumor resection. ^{6 7 8 9 10}

Who Is on the Research Team?

Constantinos G. Hadjipanayis, MD, PhD

Principal Investigator

University of Pittsburgh

John Ruge, MD, FAANS

Principal Investigator

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

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9629477/

Turning on the light for brain tumor surgery: A 5 ... 5-ALA has been shown to be safe and effective in recurrent HGG resection as well as newly diagnosed tumors. Similar to new HGG cases, 5-ALA ...

2. elsevier.es elsevier.es/en-revista-neurologia-english-edition--495-articulo-observational-retrospective-study-effectiveness-5-aminolevulinic-S217358081400025X

Observational, retrospective study of the effectiveness of 5- ... The study included 251 evaluable cases. CR and PFS6 rates were significantly higher in the group of patients treated surgically with 5-ALA: CR, 67% versus 45%, ...

3. clinmedjournals.org clinmedjournals.org/articles/ncr/neurosurgery-cases-and-reviews-ncr-7-159.php?jid=ncr

High Grade Glioma Surgery, Using 5-Aminolevulinic Acid ... The median survival was 502 days and 370 days for the 5-ALA and white light groups, respectively. 5-ALA was well-tolerated in all patients, with no reported ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT05850377

5-Aminolevulinic Acid (5-ALA) Gliolan®: Usage Increase ... The investigators are proposing a study that provides descriptive understanding of patients with High-Grade Gliomas in Ecuador. The primary objective of ...

5. journals.lww.com journals.lww.com/md-journal/fulltext/2024/01050/5_aminolevulinic_enhanced_brain_lesions_mimic.23.aspx

5-aminolevulinic enhanced brain lesions mimic glioblastoma 5-aminolevulinic acid-guided microscopic surgery has proven to be an effective way to boost the resection rate in high-grade glioma cells and ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7077938/

5-Aminolevulinic Acid Hydrochloride (5-ALA)–Guided Surgical ... 5-aminolevulinic acid hydrochloride (5-ALA) is an imaging agent that makes a high-grade glioma fluoresce under blue light, which can help guide the surgeon when ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT04738162

Clinical Safety Study on 5-Aminolevulinic Acid (5-ALA) in ... Primary objective of the study is to determine the safety of 5-ALA for fluorescence-guided resections in children and adolescents with intra-axial brain tumors.

8. trials.braintumor.org trials.braintumor.org/trials/NCT06665724

Clinical Trial Evaluating Safety of 5-Aminolevulinic Acid (5 ... This trial is designed to evaluate safety and explore possible efficacy of 5-aminolevulinic acid hydrochloride (5-ALA HCl, Gliolan®) with CV01 delivery of ...

9. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/nda/2017/208630Orig1s000MedR.pdf

208630Orig1s000 - accessdata.fda.gov 5-ALA HCl was granted orphan drug designation. Gliomas are a lethal brain tumor which are highly vascular, infiltrative (difficult to observe tumor-normal brain ...

10. thejns.org thejns.org/view/journals/j-neurosurg/136/6/article-p1525.xml

5-Aminolevulinic acid for enhanced surgical visualization ... 5-Aminolevulinic acid (5-ALA) can increase EOR by improving intraoperative visualization of contrast-enhancing tumor during fluorescence-guided surgery (FGS).