NCT07076472

Sonodynamic Therapy With SONALA-001 or 5-ALA HCL and Magnetic Resonance Guided Focused Ultrasound for the Treatment of Progressive or Recurrent Glioblastoma

MC240704 Pilot Dose Escalation and Expansion Study of Sonodynamic Therapy With Aminolevulinic Acid Hydrochloride (5-ALA HCl Or SONALA-001) in Combination With Exablate 4000 Type 2.0 MR-Guided Focused Ultrasound (MRgFUS) in Patients With Progressive or Recurrent Glioblastoma Multiforme (RGBM)

• Status: Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 8 (estimated)
• Sponsor: Mayo Clinic · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This early phase I trial tests the safety, best dose, and effectiveness of SONALA-001 or 5-ALA HCL in combination with magnetic resonance imaging-guided focused ultrasound (MRgFUS), also called sonodynamic therapy, in treating patients with glioblastoma that is growing, spreading, or getting worse (progressive) or that has come back after a period of improvement (recurrent). Sonodynamic therapy is a non-invasive combination therapy that uses low-intensity ultrasound, such as MRgFUS, to activate a drug, such as SONALA-001 or 5-ALA HCL, to kill tumor cells. SONALA-001 or 5-ALA HCL binds to the tumor and may help the sonodynamic therapy target the tumor. MRgFUS is an image-guided, non-invasive technique that uses high energy ultrasound from the Exablate 4000 Type 2.0 device to kill tumors without damaging surrounding healthy tissue. Giving sonodynamic therapy using SONALA-001 or 5-ALA HCL with MRgFUS may be safe, tolerable, and/or effective in treating patients with progressive or recurrent glioblastoma.

Detailed description

PRIMARY OBJECTIVE: I. To characterize the safety and toxicity after treatment with aminolevulinic acid intravenous formulation SONALA-001 (SONALA-001) or orally administered aminolevulinic acid hydrochloride (5-ALA HCL) in combination with MRgFUS in subjects with progressive or recurrent glioblastoma (rGBM). SECONDARY OBJECTIVES: I. To evaluate the preliminary antitumor activity as assessed by objective response rate (ORR; proportion of patients with complete or partial response) per Response Assessment in Neuro-Oncology (RANO) 2.0 guidelines. II. To evaluate preliminary signals of activity as measured by clinical benefit rate (CBR) (proportion of patients with complete response, partial response or stable disease), progression-free survival (PFS), PFS rate at 6 months, and overall survival (OS). OUTLINE: Patients receive SONALA-001 intravenously (IV) over 15 minutes or 5-ALA HCL orally (PO) and undergo transcranial MRgFUS 3-8 hours after infusion on day 1 of each cycle. Cycles repeat every 42 days in the absence of disease progression or unacceptable toxicity. Additionally, patients undergo a computed tomography (CT) of the brain prior to treatment and blood sample collection and magnetic resonance imaging (MRI) throughout the study. After completion of study treatment, patients are followed up at 30 days, and then every 3 to 6 months for up to 3 years after registration.

Conditions

• Progressive Glioblastoma
• Recurrent Glioblastoma

Interventions

Type	Name	Description
DRUG	Aminolevulinic Acid Intravenous Formulation SONALA-001	Given IV
PROCEDURE	Biospecimen Collection	Undergo blood sample collection
PROCEDURE	Computed Tomography	Undergo CT
OTHER	Electronic Health Record Review	Ancillary studies
PROCEDURE	Magnetic Resonance Imaging	Undergo MRI
PROCEDURE	MRI-Guided Focused Ultrasound Ablation	Undergo transcranial MRgFUS
DRUG	Oral Aminolevulinic Acid Hydrochloride	Given PO

Timeline

• Start date: 2025-08-14
• Primary completion: 2026-12-31
• Completion: 2026-12-31
• First posted: 2025-07-22
• Last updated: 2026-02-23

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study