GlioblastomaPhase 1/2NCT04590664
Verteporfin for the Treatment of Recurrent High Grade EGFR-Mutated Glioblastoma
A Phase 1/2 study testing Verteporfin in people with Glioblastoma and Recurrent Glioblastoma, run by Emory University at 1 site in United States.
Trial summary
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 grade EGFR-mutated glioblastoma that has come back (recurrent). Visudyne is FDA approved in combination with light to treat eye diseases. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Glioblastoma, Recurrent Glioblastoma
- What is being tested?
- Verteporfin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Jan 2021to 13 Feb 2025
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Show all 16Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Emory University Hospital/Winship Cancer Institute
Atlanta, Georgia, 30322
About this study
Official title: A Phase 1 / 2 Study of Visudyne (Liposomal Verteporfin) in Persons With Recurrent High Grade EGFR-Mutated Glioblastoma
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 grade EGFR-mutated glioblastoma that has come back (recurrent).
Visudyne is FDA approved in combination with light to treat eye diseases. In this study we use Visudyne by itself like chemotherapy to kill tumor cells which may be sensitive to verteporfin.
Read the detailed description
PRIMARY OBJECTIVES:
- To evaluate the safety and tolerability of successively higher doses of verteporfin (Visudyne [liposomal verteporfin]) and determine the maximum tolerated dose (MTD) in study participants with recurrent EGFR positive (+) glioblastoma (GBM). (Phase I) II. To evaluate the anti-tumor activity of Visudyne by assessing progression-free survival (PFS) and overall survival (OS). (Phase II) III. To describe the response rate of EGFR+ GBM in study participants treated with Visudyne. (Phase II)
SECONDARY OBJECTIVES:
- To evaluate the anti-tumor activity of Visudyne by assessing progression-free survival (PFS) and overall survival (OS). (Phase I) II. To describe the response rate of EGFR+ GBM in study participants treated with Visudyne. (Phase I) III. To describe pharmacokinetics of Visudyne administered on a weekly schedule. (Phase I) IV. To evaluate the safety and tolerability of successively higher doses of Visudyne in study participants with recurrent EGFR+ GBM. (Phase II)
OUTLINE: This is a dose-escalation study.
Patients receive verteporfin intravenously (IV) 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.
After completion of study treatment, patients are followed up for 30 days and then every 12 weeks.
- Conditions
- Glioblastoma, Recurrent Glioblastoma
- Being tested
- Verteporfin
- Medical subject headings
- Glioblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VerteporfinDrug
Also called Benzoporphyrin Derivative Monoacid Ring A, BPD-MA, Visudyne
Given IV
| Study group | Role | What they receive |
|---|---|---|
| Treatment (verteporfin) | Experimental | Patients receive verteporfin IV 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. Receives: Drug: Verteporfin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Adverse Events (Phase I)
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.[
Measured: Safety follow-up is 30 days after the last drug administered (at Day 36 +/- 1 day) *see protocol[
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 2020
- Study start15 Jan 2021
- Primary completion13 Feb 2025
- Study completion13 Feb 2025
Why it stopped: PI Decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for GlioblastomaStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Principal investigator, William Read, Emory University
- Investigators
- William L Read, MD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04590664
- Sponsor's protocol ID
- STUDY00000974
- Other IDs
- NCI-2020-05187, WINSHIP5070-20, P30CA138292
- Status last verified
- Oct 2026
- First submitted
- 11 Oct 2020
- Last updated
- 28 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 / 2 Study of Visudyne (Liposomal Verteporfin) in Persons With Recurrent High Grade EGFR-Mutated Glioblastoma
- ClinicalTrials.gov ID
- NCT04590664
- Acronym / sponsor ID
- STUDY00000974
- Conditions
- Glioblastoma, Recurrent Glioblastoma
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: PI Decision
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 15 Jan 2021 (actual)
- Primary completion
- 13 Feb 2025 (actual)
- Study completion
- 13 Feb 2025 (actual)
- First posted
- 19 Oct 2020 (actual)
- Results
- Posted 28 Oct 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 grade EGFR-mutated glioblastoma that has come back (recurrent).
Visudyne is FDA approved in combination with light to treat eye diseases. In this study we use Visudyne by itself like chemotherapy to kill tumor cells which may be sensitive to verteporfin.
Detailed description
PRIMARY OBJECTIVES:
- To evaluate the safety and tolerability of successively higher doses of verteporfin (Visudyne [liposomal verteporfin]) and determine the maximum tolerated dose (MTD) in study participants with recurrent EGFR positive (+) glioblastoma (GBM). (Phase I) II. To evaluate the anti-tumor activity of Visudyne by assessing progression-free survival (PFS) and overall survival (OS). (Phase II) III. To describe the response rate of EGFR+ GBM in study participants treated with Visudyne. (Phase II)
SECONDARY OBJECTIVES:
- To evaluate the anti-tumor activity of Visudyne by assessing progression-free survival (PFS) and overall survival (OS). (Phase I) II. To describe the response rate of EGFR+ GBM in study participants treated with Visudyne. (Phase I) III. To describe pharmacokinetics of Visudyne administered on a weekly schedule. (Phase I) IV. To evaluate the safety and tolerability of successively higher doses of Visudyne in study participants with recurrent EGFR+ GBM. (Phase II)
OUTLINE: This is a dose-escalation study.
Patients receive verteporfin intravenously (IV) 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.
After completion of study treatment, patients are followed up for 30 days and then every 12 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (verteporfin) | Experimental | Drug: VerteporfinPatients receive verteporfin IV 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VerteporfinAlso: Benzoporphyrin Derivative Monoacid Ring A, BPD-MA, VisudyneGiven IV | Drug | Treatment (verteporfin) |
Primary outcome
Incidence of Adverse Events (Phase I)
Time frame: Safety follow-up is 30 days after the last drug administered (at Day 36 +/- 1 day) *see protocol[
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.[
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
William L Read, MD principal investigator
Emory University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Verteporfin in people with Glioblastoma and Recurrent Glioblastoma, run by Emory University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 28 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Oct 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Adverse Events (Phase I) (time frame: Safety follow-up is 30 days after the last drug administered (at Day 36 +/- 1 day) *see protocol[). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Oct 2026.