24 Participants Needed

Verteporfin (Visudyne) for Glioblastoma

WL
WL
Overseen ByWilliam L. Read, MD
Age: 18+
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: Emory University
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Approved in 2 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a drug called Visudyne (Verteporfin) to evaluate its effectiveness against glioblastoma, a type of brain cancer, particularly when it recurs after treatment. Although Visudyne is typically used for eye diseases, the study aims to determine if it can kill cancer cells similarly to chemotherapy. Suitable participants have recurrent glioblastoma with a specific gene change (EGFR mutation) and have already undergone standard treatments like radiation and temozolomide. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering a chance to contribute to groundbreaking research.

Will I have to stop taking my current medications?

The trial protocol does not specify if you need to stop taking your current medications. However, you must stop any prior chemotherapy treatments for glioma at least 7 days before starting the study. If you are on bevacizumab, you may continue it while participating in the trial.

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

Research shows that verteporfin is usually well-tolerated for eye diseases. In animal studies, verteporfin helped animals live longer, which is encouraging for its potential use in humans. These results have led to more testing in clinical trials for other conditions, such as glioblastoma. In these trials, verteporfin slowed tumor cell movement and may help patients live longer.

Since the FDA has already approved verteporfin for eye diseases, there is some confidence in its safety. However, its use for glioblastoma remains under study. This trial aims to find the best dose with the fewest side effects. Participants might experience different reactions, but ensuring safety remains a primary focus of the trial.12345

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for glioblastoma, which typically involve surgery, radiation, and chemotherapy, Verteporfin offers a unique approach. Verteporfin is known for its use in photodynamic therapy for eye conditions, but in glioblastoma, it targets abnormal blood vessels within the tumor. This targeted action can potentially disrupt the tumor's blood supply, thus inhibiting its growth. Researchers are excited about Verteporfin because it introduces a novel mechanism of action, providing an alternative pathway to attack this aggressive brain cancer.

What evidence suggests that Visudyne might be an effective treatment for glioblastoma?

Research has shown that verteporfin, which participants in this trial will receive, may help treat glioblastoma, a type of brain cancer. Studies have found that verteporfin can slow the growth and spread of cancer cells in the lab. Unlike its use in treating eye diseases, verteporfin can kill these cancer cells without needing light. It also helps stop tumor cells from moving and improved survival in animal studies. These findings suggest that verteporfin might be useful in managing recurring high-grade glioblastoma, especially in people with certain genetic changes.12367

Who Is on the Research Team?

WL

William L Read, MD

Principal Investigator

Emory University

Are You a Good Fit for This Trial?

This trial is for adults with recurrent grade 4 glioblastoma that has EGFR mutations, who've had standard treatments including radiation and temozolomide. They may be on bevacizumab but not exclusively for anti-edema effects. Participants must have MRI evidence of tumor progression, agree to central venous access placement, and use effective contraception. ECOG performance status should be between 0-3.

Inclusion Criteria

I have stopped any cancer treatments, including chemotherapy, at least 7 days ago.
All participants must sign a written informed consent
I am currently using an Optune device and want to keep using it.
See 13 more

Exclusion Criteria

I have swelling that improved with bevacizumab, but my tumor still grew.
Persons who are deemed to have progression on clinical grounds only (new symptoms, declining PS) are ineligible. In the absence of MRI change one cannot be confident that clinical deterioration is a direct result of tumor progression, and could be due to intercurrent illness
I don't have severe health issues, except for brain tumor-related problems.
See 4 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive verteporfin intravenously over 83 minutes weekly for 6 weeks in cycle 1, then weekly for 5 weeks in subsequent cycles. Cycles repeat every 6 weeks in the absence of disease progression or unacceptable toxicity.

6 weeks per cycle

Follow-up

Participants are monitored for safety and effectiveness after treatment completion, with follow-ups every 12 weeks.

30 days initial follow-up, then every 12 weeks

Long-term follow-up

Participants are monitored for overall survival and progression-free survival up to 2 years.

Up to 2 years

What Are the Treatments Tested in This Trial?

Interventions

  • Verteporfin
Trial Overview The study tests the safety and optimal dosage of Visudyne (liposomal verteporfin), typically used for eye diseases, as a solo treatment like chemotherapy targeting sensitive tumor cells in high-grade EGFR-mutated glioblastoma patients.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: Treatment (verteporfin)Experimental Treatment1 Intervention

Verteporfin is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as Visudyne for:
🇪🇺
Approved in European Union as Visudyne for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

Emory University

Lead Sponsor

Trials
1,735
Recruited
2,605,000+

National Cancer Institute (NCI)

Collaborator

Trials
14,080
Recruited
41,180,000+

Published Research Related to This Trial

The VM spontaneous murine astrocytoma model effectively mimics human malignant glioma, allowing for reliable testing of cytotoxic drugs.
Drugs like BCNU, CCNU, and vincristine significantly reduced tumor volume and increased survival in treated mice, while procarbazine showed no effectiveness, highlighting the potential of these agents in glioma treatment.
The chemotherapeutic response of a murine (VM) model of human glioma.Bradford, R., Darling, JL., Thomas, DG.[2019]
Spirohydantoin mustard (spiromustine) is a promising therapy for glioblastoma multiforme that effectively penetrates the blood-brain barrier and targets brain tumors, potentially improving treatment outcomes.
Intracarotid administration of cisplatin enhances drug delivery directly to the tumor while minimizing systemic exposure, indicating a strategic approach to chemotherapy for better efficacy and safety in treating this aggressive brain cancer.
Spiromustine and intracarotid artery cisplatin in the treatment of glioblastoma multiforme.Resio, MJ., DeVroom, HL.[2019]
Vinblastine (VBL) was used to treat a 7-month-old girl with progressive low-grade glioma, resulting in a significant increase in liver enzymes (grade IV toxicity) that resolved after treatment ended.
The case suggests that younger patients may experience different toxicity profiles with VBL, indicating a need for age-specific toxicity screening in future treatment protocols.
Transient Hepatotoxicity Induced by Vinblastine in a Young Girl with Chiasmatic Low Grade Glioma.Franke, NE., Blok, GJ., Voll, ML., et al.[2021]

Citations

Repurposing the drug verteporfin as anti-neoplastic ...Verteporfin treatment decreased tumor cell migration in vitro and reduced infiltration and conferred survival benefit in vivo in a GSC xenograft model of ...
Verteporfin for the Treatment of Recurrent High Grade ...This phase I/II trial studies the side effects and best dose of Visudyne (liposomal verteporfin) and to see how well it works in treating patients with high ...
Verteporfin (Visudyne) for GlioblastomaThe combination of bevacizumab (BEV) and vorinostat (VOR) was well tolerated in patients with recurrent glioblastoma, with a 6-month progression-free survival ( ...
In-Vitro Use of Verteporfin for Photodynamic Therapy ...Photodynamic therapy with verteporfin causes cell death in glioma cells. · Verteporfin is a potential drug for targeting glioblastoma pharmacologically.
Verteporfin inhibits growth of human glioma in vitro without ...In summary, we demonstrate for the first time that verteporfin can inhibit growth and proliferation of human glioma cells without any light ...
Verteporfin for the Treatment of Recurrent High Grade ...A general description of the clinical trial arm. It identifies the role of the intervention that participants receive. Types of arms include experimental arm, ...
The impact of interaction between verteporfin and yes ...Animal studies have shown verteporfin to be successful in increasing survival rates, which have led to the conduction of phase 1 and 2 clinical trials to ...
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