Clinical Trial Evaluating Safety of 5-Aminolevulinic Acid (5-ALA) Combined With Ultrasound for Sonodynamic Therapy (SDT) in Patients With Newly Diagnosed Fast-growing Brain Tumor (High-Grade Glioma) Prior to Resection and Standard Adjuvant Therapy

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Universitaet Muenster

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

21 Nov 2025 → 9 Feb 2026

Decision date (initial)

2024-11-05

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Alpheus Medical, Inc.

External identifiers

EU CT number

2023-509238-20-00

WHO UTN

U1111-1296-1261

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Therapy

To evaluate safety and tolerability of SDT using 5-ALA + CV01-delivered SDT in newly diagnosed high grade glioma

Secondary objectives 2

1. To assess radiological changes in response to 5-ALA + CV01-delivered SDT
2. To assess the histopathological efficacy of SDT using 5-ALA + CV01- delivered SDT

Conditions and MedDRA coding

High grade glioma

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Age ≥ 18 years
2. WHO Performance Status of 0-2
3. Patients with cranial MRI displaying typical features of high-grade glioma on imaging or histologically proven high-grade glioma including GBM, anaplastic astrocytoma (AA), anaplastic oligodendroglioma (AO). No previous tumor specific treatment such as surgery (apart from biopsy), radio- or chemotherapy, antiangiogenic or immunotherapy).
4. Planned debulking or cytoreductive surgery
5. The following laboratory values at study entry: a. Absolute neutrophil count (ANC) ≥ 1500 cells/mm3, b. Platelet count ≥ 100,000 cells/mm3, c. Hemoglobin (Hgb) > 10g/dL, d. AST and ALT ≤ 2.5 x Upper Limit of Normal (ULN), e. Total bilirubin ≤ 1.5 x ULN, f. Creatine clearance (CrCL) as estimated by Cockcroft-Gault equation of ≥ 40 mL/min, g. Blood clotting within acceptable limits according to investigator
6. For female and male patients and their female partners of childbearing/reproductive potential: Willingness to apply highly effective contraception (Pearl index < 1) during the entire study and in female patients for 6 months after the last application of Gliolan® and in male patients and their female partners for 3 months after the last application of Gliolan®.
7. Ability to understand and provide informed consent

Exclusion criteria 18

1. Infra-tentorial tumors
2. Gastrointestinal disorder that negatively affects absorption
3. Known active hepatitis B or C (Note: testing is not required)
4. Known Human Immunodeficiency Virus (HIV) infection (Note: testing is not required)
5. Unable to avoid photosensitising drugs (eg, St. John’s wort, griseofulvin, thiazide diuretics, sulfonylureas, phenothiazines, sulfonamides, quinolones, and tetracyclines) up to 2 weeks following 5-ALA administration
6. Any other concurrent severe and/or uncontrolled concomitant medical condition that could compromise participation in the study (e.g, clinically significant pulmonary disease, clinically significant psychiatric or neurological disorder, active or uncontrolled infection)
7. Diagnosis of other invasive cancer (except basal cell carcinoma/squamous cell carcinoma of the skin) within the last 5 years; adequately treated carcinoma in situ is allowed
8. Patient has a condition the investigator believes would interfere with the ability to provide informed consent or comply with study instructions, or that might confound the interpretation of the study results or put the patient at undue risk
9. Patients who have clinically significant edema or tumor mass effect requiring urgent intervention (e.g., surgery, initiation of steroids, escalating doses of steroids)
10. Women who are pregnant or breastfeeding
11. Inability to undergo MRI or receive gadolinium (Gd)
12. Hypersensitivity to 5-ALA or porphyrins
13. Average skull thickness at the treatment field > 10 mm from standard navigation CTs. The treatment field is defined as the various locations on the head where the transducer will be coupled to the patient. The average skull thickness at each treatment field will be determined through post-processing the thin cut head computed tomography (CT) scan (without contrast).
14. Hemorrhagic or ischemic stroke (including transient ischemic attacks) and central nervous system bleeding in the preceding 6 months that are not related to glioma biopsy. History of prior intratumoral bleeding is not an exclusion criterion; however, all patient’s navigation CTs will be reviewed for the presence of fresh blood.
15. Participation in another interventional clinical trial during this trial or within 4 weeks before entry into this trial
16. Known acute or chronic types of porphyria
17. Patients who are dependent on the sponsor, investigator or trial site
18. Patients who are committed to an institution by virtue of an order issued either by the judicial or the administrative authorities

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Clinical safety and tolerability will be assessed based on the observation of adverse events (AEs). AEs will be graded as per the NCI CTCAE v5. Patients will be assessed for AEs for 28 days following administration of SDT (marking study completion). Pathological findings of laboratory evaluations (specifically, complete blood counts and serum chemistries [including liver function tests] and physical examinations [including a detailed neurological examination]) will be documented as AEs.

Secondary endpoints 2

1. Radiological changes: Changes on MRI between visit 1 (pre SDT) and visit 3 (post SDT) of tumor volume (enhancing tumor including necrosis, cm³), volume of FLAIR hyperintensity (cm³), volume of cytotoxic edema based on diffusion restriction on corresponding ADC and DWI sequences (cm³), relative cerebral blood volume (rCBVmax) and leakage (K2, 0.001/min).
2. Histopathological efficacy: Anti-tumor activity assessed by number of cells / mm³ with antibodies against IBA1 and number of cells / mm³ with antibodies against Caspase III

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 3

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Universitaet Muenster

Sponsor organisation

Universitaet Muenster

Address

Schlossplatz 2, Schlossbezirk Schlossbezirk

City

Muenster

Postcode

48149

Country

Germany

Scientific contact point

Organisation

Universitaet Muenster

Contact name

Central study office

Public contact point

Organisation

Universitaet Muenster

Contact name

Central study office

Third parties 1

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Temporary halts 1 · Art. 38 CTR

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 3 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications