A Dose-escalation Clinical Study of Intraoperative Photodynamic Therapy of Glioblastoma

NCT05736406 | Recruiting Phase 1 DMC FDA Drug FDA Device

• 3 other identifiers: HTX-GBM-012025-520563-41-002025-A00107-42
• Study Type: interventional
• Target: 12
• Locations: 2 countries
• Sites: 2

Brief 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. 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.

Conditions

Primary Glioblastoma

Keywords

Glioma; Glioblastoma; High Grade Glioma

Outcome Measures

Primary Outcomes (1)

• Maximal Tolerated Dose (MDT) of light for Photodynamic Therapy

  Evaluation of Dose Limiting Toxicities (DLT) to define MDT

  4 weeks after intervention

Secondary Outcomes (2)

• Treatment response / Progression-free survival (PFS)

  Up to 6 months after intervention

• Number of Adverse Events and Serious Adverse Events (Safety and Tolerability)

  6 months

Other Outcomes (1)

• Usability of the Heliance® Solution in the operating room

  During Procedure

Study Arms (2)

200 J/cm^2

EXPERIMENTAL

Patient will undergo intraoperative Photodynamic therapy at 200 J/cm\^2

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

Combination Product: 5-ALA HCl intraoperative Photodynamic Therapy (PDT) at 400 J/cm^2

Interventions

5-ALA HCl intraoperative Photodynamic Therapy (PDT) at 200 J/cm^2 COMBINATION_PRODUCT

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.

Also known as: Pentalafen®, 5-aminolevulinic acid hydrochloride, Heliance® Solution, 5-ALA PDT

200 J/cm^2

5-ALA HCl intraoperative Photodynamic Therapy (PDT) at 400 J/cm^2 COMBINATION_PRODUCT

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.

Also known as: Pentalafen®, 5-aminolevulinic acid hydrochloride, Heliance® Solution, 5-ALA PDT

400 J/cm^2

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• I02. Signed informed consent which includes compliance with requirements and restrictions listed in the informed consent.
• I03. Newly diagnosed GBM, presumed on the basis of clinical and MRI criteria (intra-axial brain tumor with peripheral rim contrast enhancement).
• I04. Karnofsky Performance Score ≥70 I05. Eligible for surgery. I06. Amenable to maximal tumor resection based on MRI. I07. Planned to receive SOC (i.e., Stupp Protocol) treatment after surgery. I08. Ability to take oral medications. I09. Tumor eligible to PDT procedure as validated by both investigator and sponsor based on pre-operative MRI data

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Hemerion Therapeutics: lead

Study Sites (2)

UPMC Hillman Cancer center

Pittsburgh, Pennsylvania, 15213, United States

RECRUITING

CHU De Lille, Hôpital Roger Salengro

Lille, 59037, France

RECRUITING

MeSH Terms

Conditions

Glioma; Glioblastoma

Interventions

1-phenyl-3,3-dimethyltriazene; Aminolevulinic Acid

Condition Hierarchy (Ancestors)

Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Astrocytoma

Intervention Hierarchy (Ancestors)

Levulinic Acids; Keto Acids; Carboxylic Acids; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins

Study Officials

• Jan Drappatz, MD

  UPMC Presbyterian Shadyside Hospital, Pittsburgh, PA

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Antoine Mequignon, MSc

CONTACT

+33 6 62 78 94 01; clinical@hemerion.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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).

Study Record Dates

• First Submitted: February 8, 2023
• First Posted: February 21, 2023
• Study Start: February 20, 2024
• Primary Completion: December 1, 2025
• Study Completion (Estimated): July 1, 2026
• Last Updated: September 11, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1); FR (1)