Primary GlioblastomaPhase 1/2NCT05736406
A Dose-escalation Clinical Study of Intraoperative Photodynamic Therapy of Glioblastoma
A Phase 1/2 study testing 5-ALA HCl intraoperative Photodynamic Therapy (PDT) at 200 J/cm^2 and 5-ALA HCl intraoperative Photodynamic Therapy (PDT) at 400 J/cm^2 in people with Primary Glioblastoma, run by Hemerion Therapeutics at 2 sites in 2 countries.
Trial summary
The primary objective of this clinical trial is to determine the safety and tolerability of two doses of light in intraoperative PDT added to standard of care; temozolomide-based chemotherapy in male and female patients aged 18 to 75 with newly diagnosed glioblastoma. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Primary Glioblastoma
- What is being tested?
- 5-ALA HCl intraoperative Photodynamic Therapy (PDT) at 200 J/cm^2 and 5-ALA HCl intraoperative Photodynamic Therapy (PDT) at 400 J/cm^2
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 20 Feb 2024to Aug 2026
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
None listed.
Who cannot join
- 1.Medical conditions E01.
- Patient with bifocal or multifocal disease, assessed on MR1I T1Gd enhanced.
- Patient with tumor of deep location such as tumor involving the corpus callosum, the basal ganglia, the brain stem, or tumor involving the midline as assessed on MRI.
- Patient with prior brain surgery other than stereotactic biopsy E02. Patient with Lynch syndrome E03. Patient with Li-Fraumeni syndrome E04. Debilitating cardiopulmonary disease, unstable Type 1 or Type 2 diabetes (treated or not) E05. History or current condition of another malignancy (excluding basal cell carcinoma, or E05. carcinoma in-situ) unless treated and off all active therapy for more than 5 years E06. Clinically significant abnormal ECG results, including a corrected QT interval QTc > 480 ms E07. Creatinine clearance < 60 mL/min E08. Severe hepatic impairment (bilirubin > 1.5 x the upper limit of normal [ULN] or alkaline phosphatase or transaminases (AST, ALT) > 2.5 x ULN) E09. Known allergic reactions to silicone E10. Known allergic reactions or hypersensitivity to egg, soya, or peanut proteins.
- E11. Febrile illness
- Contraindication
Show all 10Show fewer
- E12. Contraindication to 5-ALA HCl administration, including:
- Porphyria
- Taking photosensitizing drugs 24 hours before and 14 days after the administration of Pentalafen® including but not limited to: St. John's wort, griseofulvin, thiazide diuretics, sulfonylureas, phenothiazines, sulphonamides, quinolones and tetracyclines, and topical preparations containing ALA (See Section 6.10)
- Inability to suspend a long-term hepatotoxic treatment (such as, but not limited to diclofenac, fenofibrate, carbamazepine) for 24 hours after 5-ALA HCl intake E13. Contraindication to MRI examination (e.g., MRI-incompatible pacemaker) E14. Treatment with another investigational drug or intervention within 30 days prior to or during the entire study
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United States1 site
UPMC Hillman Cancer center
Pittsburgh, Pennsylvania, 15213
About this study
Official title: An Interventional, Multicenter, and International Phase 1/2, Light-dose-escalation Study to Investigate the Safety and Feasibility of Intraoperative Photodynamic Therapy (PDT) With Pentalafen® Drug and Heliance® Solution Device in Male and Female Patients 18 to 75 Years of Age With Grade IV Glioblastoma.
The primary objective of this clinical trial is to determine the safety and tolerability of two doses of light in intraoperative PDT added to standard of care; temozolomide-based chemotherapy in male and female patients aged 18 to 75 with newly diagnosed glioblastoma.
This treatment will be carried out in addition to the maximal surgical resection. Data collected during this trial will be used to design the upcoming pivotal study .
The study will utilize an independent Data and Safety Monitoring Board (iDSMB) that will review safety data to allow dose escalation.
Read the detailed description
This study is a non randomized, open label, multicentercenter , phase 1/2 trial with a sequential enrollment in a 3+3 dose escalation design to establish the maximal tolerated dose of light (MTD).
The dose of light will be escalated in successive cohorts of patients until at least 1 patient experiences a dose-limiting toxicity (DLT).
A DLT is defined as any grade ≥ 3 Adverse Event (AE), or any relevant grade 2 AE of Central Nervous System or any Serious Adverse Events (SAEs) possibly, probably or definitively related to the intraoperative PDT (i.e., 5-aminolevulinic acid hydrochloride (5-ALA HCl) administration + brain cavity illumination), for which the onset date is within 28 days after the procedure, and where conservative therapy fails and surgical is required, according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
After dose escalation patient will be followed in the standard of care until visit at 6 months to evaluate the progression free survival.
- Conditions
- Primary Glioblastoma
- Being tested
- 5-ALA HCl intraoperative Photodynamic Therapy (PDT) at 200 J/cm^2, 5-ALA HCl intraoperative Photodynamic Therapy (PDT) at 400 J/cm^2
- Keywords
- Glioma, Glioblastoma, High Grade Glioma
- Medical subject headings
- Glioma, Glioblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
5-ALA HCl intraoperative Photodynamic Therapy (PDT) at 200 J/cm^2Combination product
Also called Pentalafen®, 5-aminolevulinic acid hydrochloride, Heliance® Solution, 5-ALA PDT
5-ALA HCl, 20 mg/kg, is orally administered 4-6 hours before the surgery. Then after resection of tumor tissue is judged maximal, the intraoperative PDT procedure is initiated at 200 J/cm^2.
5-ALA HCl intraoperative Photodynamic Therapy (PDT) at 400 J/cm^2Combination product
Also called Pentalafen®, 5-aminolevulinic acid hydrochloride, Heliance® Solution, 5-ALA PDT
5-ALA HCl, 20 mg/kg, is orally administered 4-6 hours before the surgery. Then after resection of tumor tissue is judged maximal, the intraoperative PDT procedure is initiated at 400 J/cm^2.
| Study group | Role | What they receive |
|---|---|---|
| 200 J/cm^2 | Experimental | Patient will undergo intraoperative Photodynamic therapy at 200 J/cm^2 Receives: Combination Product: 5-ALA HCl intraoperative Photodynamic Therapy (PDT) at 200 J/cm^2 |
| 400 J/cm^2 | Experimental | Patient will undergo intraoperative Photodynamic therapy at 400 J/cm^2 Receives: Combination Product: 5-ALA HCl intraoperative Photodynamic Therapy (PDT) at 400 J/cm^2 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximal Tolerated Dose (MDT) of light for Photodynamic Therapy
Evaluation of Dose Limiting Toxicities (DLT) to define MDT
Measured: 4 weeks after intervention
Secondary outcomes (2)
Treatment response / Progression-free survival (PFS)
MRI imaging and RANO criteria
Measured: Up to 6 months after intervention
Number of Adverse Events and Serious Adverse Events (Safety and Tolerability)
Global clinical safety data of 5-ALA HCl Intraoperative PDT
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2023
- Study start20 Feb 2024
- Primary completion6 Mar 2026
- Study completionAug 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary GlioblastomaStudies of the same condition, open and under way.
- 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
- Non-randomised
- Intervention model
- Sequential
- Model description
- 3+3 model.The dose of light will be escalated in successive cohorts of patients. The starting dose is at 200 J/cm2. Enroll 3 patients at the first dose level and proceed to the next higher dose level with a cohort of 3 patients until at least 1 patient experiences a dose-limiting toxicity (DLT).
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hemerion Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jan Drappatz, MD, Principal investigator, UPMC Presbyterian Shadyside Hospital, Pittsburgh, PA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05736406
- Sponsor's protocol ID
- HTX-GBM-01
- Other IDs
- 2025-520563-41-00, 2025-A00107-42, 2025
- Status last verified
- May 2026
- First submitted
- 8 Feb 2023
- Last updated
- 22 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Interventional, Multicenter, and International Phase 1/2, Light-dose-escalation Study to Investigate the Safety and Feasibility of Intraoperative Photodynamic Therapy (PDT) With Pentalafen® Drug and Heliance® Solution Device in Male and Female Patients 18 to 75 Years of Age With Grade IV Glioblastoma.
- ClinicalTrials.gov ID
- NCT05736406
- Acronym / sponsor ID
- HTX-GBM-01
- Conditions
- Primary Glioblastoma
- Keywords
- Glioma, Glioblastoma, High Grade Glioma
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Hemerion TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 20 Feb 2024 (actual)
- Primary completion
- 6 Mar 2026 (actual)
- Study completion
- Aug 2026 (estimated)
- First posted
- 21 Feb 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this clinical trial is to determine the safety and tolerability of two doses of light in intraoperative PDT added to standard of care; temozolomide-based chemotherapy in male and female patients aged 18 to 75 with newly diagnosed glioblastoma.
This treatment will be carried out in addition to the maximal surgical resection. Data collected during this trial will be used to design the upcoming pivotal study .
The study will utilize an independent Data and Safety Monitoring Board (iDSMB) that will review safety data to allow dose escalation.
Detailed description
This study is a non randomized, open label, multicentercenter , phase 1/2 trial with a sequential enrollment in a 3+3 dose escalation design to establish the maximal tolerated dose of light (MTD).
The dose of light will be escalated in successive cohorts of patients until at least 1 patient experiences a dose-limiting toxicity (DLT).
A DLT is defined as any grade ≥ 3 Adverse Event (AE), or any relevant grade 2 AE of Central Nervous System or any Serious Adverse Events (SAEs) possibly, probably or definitively related to the intraoperative PDT (i.e., 5-aminolevulinic acid hydrochloride (5-ALA HCl) administration + brain cavity illumination), for which the onset date is within 28 days after the procedure, and where conservative therapy fails and surgical is required, according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
After dose escalation patient will be followed in the standard of care until visit at 6 months to evaluate the progression free survival.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- 3+3 model.The dose of light will be escalated in successive cohorts of patients. The starting dose is at 200 J/cm2. Enroll 3 patients at the first dose level and proceed to the next higher dose level with a cohort of 3 patients until at least 1 patient experiences a dose-limiting toxicity (DLT).
| Arm | Type | Receives |
|---|---|---|
| 200 J/cm^2 | Experimental | Combination Product: 5-ALA HCl intraoperative Photodynamic Therapy (PDT) at 200 J/cm^2Patient will undergo intraoperative Photodynamic therapy at 200 J/cm^2 |
| 400 J/cm^2 | Experimental | Combination Product: 5-ALA HCl intraoperative Photodynamic Therapy (PDT) at 400 J/cm^2Patient will undergo intraoperative Photodynamic therapy at 400 J/cm^2 |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (0 listed)
Key exclusion criteria (10 listed)
- 1.Medical conditions E01.
- Patient with bifocal or multifocal disease, assessed on MR1I T1Gd enhanced.
- Patient with tumor of deep location such as tumor involving the corpus callosum, the basal ganglia, the brain stem, or tumor involving the midline as assessed on MRI.
- Patient with prior brain surgery other than stereotactic biopsy E02. Patient with Lynch syndrome E03. Patient with Li-Fraumeni syndrome E04. Debilitating cardiopulmonary disease, unstable Type 1 or Type 2 diabetes (treated or not) E05. History or current condition of another malignancy (excluding basal cell carcinoma, or E05. carcinoma in-situ) unless treated and off all active therapy for more than 5 years E06. Clinically significant abnormal ECG results, including a corrected QT interval QTc > 480 ms E07. Creatinine clearance < 60 mL/min E08. Severe hepatic impairment (bilirubin > 1.5 x the upper limit of normal [ULN] or alkaline phosphatase or transaminases (AST, ALT) > 2.5 x ULN) E09. Known allergic reactions to silicone E10. Known allergic reactions or hypersensitivity to egg, soya, or peanut proteins.
- E11. Febrile illness
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 |
|---|---|---|
| 5-ALA HCl intraoperative Photodynamic Therapy (PDT) at 200 J/cm^2Also: Pentalafen®, 5-aminolevulinic acid hydrochloride, Heliance® Solution, 5-ALA PDT5-ALA HCl, 20 mg/kg, is orally administered 4-6 hours before the surgery. Then after resection of tumor tissue is judged maximal, the intraoperative PDT procedure is initiated at 200 J/cm^2. | Combination product | 200 J/cm^2 |
| 5-ALA HCl intraoperative Photodynamic Therapy (PDT) at 400 J/cm^2Also: Pentalafen®, 5-aminolevulinic acid hydrochloride, Heliance® Solution, 5-ALA PDT5-ALA HCl, 20 mg/kg, is orally administered 4-6 hours before the surgery. Then after resection of tumor tissue is judged maximal, the intraoperative PDT procedure is initiated at 400 J/cm^2. | Combination product | 400 J/cm^2 |
Primary outcome
Maximal Tolerated Dose (MDT) of light for Photodynamic Therapy
Time frame: 4 weeks after intervention
Evaluation of Dose Limiting Toxicities (DLT) to define MDT
2 secondary outcomes
Treatment response / Progression-free survival (PFS)
Time frame: Up to 6 months after intervention
Number of Adverse Events and Serious Adverse Events (Safety and Tolerability)
Time frame: 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2026.
- 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.
Jan Drappatz, MD principal investigator
UPMC Presbyterian Shadyside Hospital, Pittsburgh, PA
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing 5-ALA HCl intraoperative Photodynamic Therapy (PDT) at 200 J/cm^2 and 5-ALA HCl intraoperative Photodynamic Therapy (PDT) at 400 J/cm^2 in people with Primary Glioblastoma, run by Hemerion Therapeutics at 2 sites in 2 countries.
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 0 inclusion and 10 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: under way, not looking for participants, as of the sponsor's last update on 22 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, France. 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?
This study is still under way. Results are not available in the source record yet.
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?
Maximal Tolerated Dose (MDT) of light for Photodynamic Therapy (time frame: 4 weeks after intervention). 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 22 May 2026.