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Verteporfin for Recurrent EGFR-Mutated Glioblastoma

This study is testing a drug called Verteporfin (also known as Visudyne) for people with glioblastoma, a type of brain cancer, that has returned and has a specific genetic change called an EGFR mutation. Verteporfin is already approved for eye diseases, but in this study, it's being used differently, like a chemotherapy, to see if it can kill tumor cells. The study aims to find the safest and most effective dose of Verteporfin and to see how well it works in shrinking tumors and extending the time before the cancer grows again. You may be able to join if you have recurrent glioblastoma with an EGFR mutation and have already received standard treatments like radiation and temozolomide. The study plans to enroll 24 participants.

Study design
This is a dose-escalation study, meaning participants will receive increasing doses of Verteporfin to find the safest and most effective amount. It is a Phase I/II study, which means it looks at both safety and how well the treatment works.
What's involved
You would receive Verteporfin intravenously (through a vein) once a week for 6 weeks in the first cycle, and then weekly for 5 weeks in later cycles. These cycles repeat every 6 weeks as long as the treatment is working and you are tolerating it.
Compensation
Not stated in the trial record.
Follow-up
After your study treatment ends, you will be followed for 30 days, and then every 12 weeks after that.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04590664

Verteporfin for the Treatment of Recurrent High Grade EGFR-Mutated Glioblastoma

Recruiting
PHASE1Ages 18+InterventionalTreatment
Emory University
~24 participants
Updated 2025-02-25 on ClinicalTrials.gov
What's tested:Verteporfin

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of adverse events (Phase I)
Measured over From study enrollment until 100 days after the last day of study participation
+3 more outcomes measured

Conditions

Glioblastoma
Recurrent Glioblastoma

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Where it's being run

1 sites across 1 states
Georgia1

Study leadership

  • William L Read, MD · PRINCIPAL_INVESTIGATOR · Emory University

Who to contact

William L. Read, MD
[email protected]
404-778-1900
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Persons with recurrent or progressive grade 4 glioma (glioblastoma) are eligible for this study. Participants should have received standard first line therapy including radiation and temozolomide
Eligible participants have tumors that show mutant or amplified EGFR. This determination can be made using standard of care mutation analysis panels (e.g. Snapshot). It is often assessed at diagnosis as part of standard of care
Eligible participants must have evidence on magnetic resonance imaging (MRI) of progression. This may be as new or increased enhancement, or growth / increase in nonenhancing abnormality. Care should be taken to distinguish those with true progression from those with radiation related changes. Persons with changes in enhancement possibly due in part or in whole to late radiation effect should receive bevacizumab as standard of care, and defer study participation
Participants may be receiving bevacizumab, and show progression while on bevacizumab. These participants may continue bevacizumab while on study. Persons not on bevacizumab but who would benefit from the anti-edema effect of bevacizumab should not enroll on this study but should proceed with bevacizumab alone, and defer enrollment until such time as they progress
Visudyne is a vesicant. Participants will likely have poor veins, and will require repeated intravenous treatments. Participants must be willing to have placed a central venous access, such as a portacath
Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0-3. Participants who are ECOG 2 or 3 should ideally have been in that situation for some time, and not be in the midst of rapid clinical decline
Medical comorbidities (excepting neurological) must be grade 2 or less if graded as toxicity
Eligible participants may have grade 3 neurologic comorbidities (for example aphasia, ataxia) arising as a consequence of brain pathology
Participants should be reasonably expected to be able to complete 6 weeks (1 cycle) of treatment on study before death or worsening of PS to 4 or 5
Other anti-cancer medical treatments. Treatments in this category include chemotherapy and non-bevacizumab therapies. 7 days must have elapsed since discontinuation of prior chemotherapeutic treatments for glioma and study treatment. Participants may have had any number of prior treatments
All participants on this study must have had prior radiation to the brain. Radiation must have been completed 90 days prior to first study treatment
21 days must have elapsed since prior major surgery
Participants already using a Novo-tumor treating fields therapy (TTF) (Optune) device and who wish to continue may do so
All participants must sign a written informed consent
The effects of study drugs used in this study on the developing human fetus are unknown. For this reason, female of child-bearing potential (FCBP) must have a negative serum or urine pregnancy test prior to starting therapy
FCBP and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation and 8 weeks after. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation. A female of childbearing potential (FCBP) is a sexually mature woman who: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months

Exclusion

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
Persons with edema which might be due to late radiation effect, not true progression, should receive bevacizumab as standard of care, and defer study participation. If (short-term) followup imaging shows reduction of edema and also progression of tumor, these persons are eligible (and should continue bevacizumab)
Pregnant or breast-feeding women will not be entered on this study
Participants may not have any baseline comorbidities or laboratory abnormalities which would be of grade 3 or worse if graded as toxicities by Common Terminology Criteria for Adverse Events (CTCAE) (excepting alopecia). An exception is made for neurologic comorbidities (e.g. ataxia, aphasia) arising as a consequence of the brain tumor; symptoms severe enough to warrant medical treatment as is offered on this study are by definition grade III
Persons who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are ineligible
Illness or any other circumstances (as defined by the investigator), which would preclude safe performance of study procedures or compromise the ability of the patient to consent to study
Persons with hereditary porphyria are ineligible

What this trial measures

  • Incidence of adverse events (Phase I)From study enrollment until 100 days after the last day of study participation

    Will be graded by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. For each adverse event, information to be collected includes event description, time of onset, clinician assessment of severity, relationship to study product (assessed only by those with the training and authority to make a diagnosis), and time of resolution/stabilization of the event.

  • Progression free survival (PFS) (Phase II)At 6 weeks

    Will be assessed by Response Assessment in Neuro-Oncology Criteria (RANO) for magnetic resonance imaging (MRI) of glioblastoma. Progression-free survival is defined as no progression within 6 weeks. Progression-free survival will be estimated as a binary rate, and a 95% confidence interval will be estimated using the Clopper-Pearson method.

  • Response rate (RR) (Phase II)From study enrollment until 2 years

    Will be assessed by RANO for MRI of glioblastoma.

  • Overall survival (Phase II)Time from study enrollment to death or last follow-up, assessed up to 2 years

    Will be estimated using the Kaplan-Meier method.