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

NCT07076472 | Recruiting Early Phase 1 DMC FDA Drug

• 4 other identifiers: MC240704NCI-2025-0450124-013192R44CA275508
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Recurrent Glioblastoma; Progressive Glioblastoma

Outcome Measures

Primary Outcomes (5)

• Incidence of adverse events (AEs)

  Defined per the Common Terminology Criteria for Adverse Events (CTCAE) version (v) 5 as AEs considered to be at least possibly related to study treatment. The maximum grade for each type of AE will be recorded for each patient, and frequency tables will be reviewed to determine patterns (by dose level and overall).

  Up to 30 days after last dose of study treatment

• Incidence of grade 3 or higher non-hematologic AEs

  Defined per the CTCAE v5 as AEs considered to be at least possibly related to study treatment. The maximum grade for each type of AE will be recorded for each patient, and frequency tables will be reviewed to determine patterns (by dose level and overall).

  Up to 30 days after last dose of study treatment

• Incidence of grade 4 or higher AE

  Defined per the CTCAE v5 as AEs considered to be at least possibly related to study treatment. The maximum grade for each type of AE will be recorded for each patient, and frequency tables will be reviewed to determine patterns (by dose level and overall).

  Up to 30 days after last dose of study treatment

• Dose-limiting event (DLE)

  AEs will be evaluated using CTCAE v5. The target DLE rate is \< 33%.

  During first 6 weeks of therapy [first cycle of treatment (cycle length = 42 days)]

• Maximum tolerated dose (MTD)

  Will be defined as the highest safety-tolerated dose level where at most one patient out of six experiences a DLE.

  Up to 6 weeks

Secondary Outcomes (8)

• Overall response rate (ORR)

  Up to 3 years

• Clinical benefit rate

  Up to 3 years

• Progression-free survival (PFS)

  Up to 3 years

• PFS at 6 months (PFS6)

  At 6 months

• Overall survival (OS)

  Up to 3 years

Study Arms (1)

Treatment (SONALA-001, MRgFUS)

EXPERIMENTAL

Patients receive SONALA-001 IV over 15 minutes or 5-ALA HCL 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 CT of the brain prior to treatment and blood sample collection and MRI throughout the study.

Drug: Aminolevulinic Acid Intravenous Formulation SONALA-001; Procedure: Biospecimen Collection; Procedure: Computed Tomography; Other: Electronic Health Record Review; Procedure: Magnetic Resonance Imaging; Procedure: MRI-Guided Focused Ultrasound Ablation; Drug: Oral Aminolevulinic Acid Hydrochloride

Interventions

Aminolevulinic Acid Intravenous Formulation SONALA-001 DRUG

Given IV

Also known as: ALA Intravenous Formulation SONALA-001, SONALA 001, SONALA-001, SONALA001

Treatment (SONALA-001, MRgFUS)

Biospecimen Collection PROCEDURE

Undergo blood sample collection

Also known as: Biological Sample Collection, Biospecimen Collected, Specimen Collection

Treatment (SONALA-001, MRgFUS)

Computed Tomography PROCEDURE

Undergo CT

Also known as: CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography, Computerized axial tomography (procedure), Computerized Tomography, Computerized Tomography (CT) scan, CT, CT Scan, tomography

Treatment (SONALA-001, MRgFUS)

Electronic Health Record Review OTHER

Ancillary studies

Treatment (SONALA-001, MRgFUS)

Magnetic Resonance Imaging PROCEDURE

Undergo MRI

Also known as: Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR, MR Imaging, MRI, MRI Scan, MRIs, NMR Imaging, NMRI, Nuclear Magnetic Resonance Imaging, sMRI, Structural MRI

Treatment (SONALA-001, MRgFUS)

MRI-Guided Focused Ultrasound Ablation PROCEDURE

Undergo transcranial MRgFUS

Also known as: Magnetic Resonance Imaging-guided High-Intensity Focused Ultrasound, Magnetic Resonance-guided Focused Ultrasound, MR-HIFU, MRgFUS, MRI Guided Focused Ultrasound Surgery, MRI-HIFU

Treatment (SONALA-001, MRgFUS)

Oral Aminolevulinic Acid Hydrochloride DRUG

Given PO

Also known as: 5-ALA fluorescence, 5-Aminolevulinic Acid Hydrochloride, 5-Aminolevulinic Acid Hydrochloride, Oral, Gleolan, Gliolan, Oral 5-ALA, Oral ALA HCl

Treatment (SONALA-001, MRgFUS)

Eligibility Criteria

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

You may qualify if:

• Age ≥ 18 years
• Diagnosis of recurrent or progressive glioblastoma (as defined in 2021 World Health Organization \[WHO\] Classification of Tumors of the Central Nervous System; Louis, Perry, et al. 2021) for which resection is not indicated as assessed by the study physician
• Radiographic evidence of disease which may be measurable or non-measurable
• Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0, 1 or 2
• Previous treatment with radiotherapy (RT)
• Have a life expectancy of ≥ 12 weeks
• Hemoglobin ≥ 9.0 g/dL (obtained ≤ 15 days prior to registration)
• Absolute neutrophil count (ANC) ≥ 1500/mm\^3 (obtained ≤ 15 days prior to registration)
• Platelet count ≥ 100,000/mm\^3 (obtained ≤ 15 days prior to registration)
• Total bilirubin ≤ 1.5 x upper limit of normal (ULN) (obtained ≤ 15 days prior to registration)
• Alanine aminotransferase (ALT) and aspartate transaminase (AST) ≤ 3 x ULN (≤ 5 x ULN for patients with liver involvement) (obtained ≤ 15 days prior to registration)
• Albumin ≥ 3 g/dL (obtained ≤ 15 days prior to registration)
• Potassium ≥ lower limit of normal (LLN) (obtained ≤ 15 days prior to registration)
• Serum total calcium ≥ LLN (obtained ≤ 15 days prior to registration)
• Creatinine ≤ 1.5 x ULN OR calculated creatinine clearance ≥ 60 mL/min using the Cockcroft-Gault formula (obtained ≤ 15 days prior to registration)

You may not qualify if:

• Any of the following because this study involves an investigational agent, the genotoxic, mutagenic, and teratogenic effects of which on the developing fetus and newborn are unknown:
• Pregnant persons
• Nursing persons
• Persons of childbearing potential (and persons able to father a child) who are unwilling to employ adequate contraception
• Recurrence ≤ 4 weeks after the completion of RT, defined from the imaging assessment immediately after completion of RT
• Three or more prior systemic treatments for recurrent or progressing disease
• Diagnosis of porphyria, or hypersensitivity to porphyrins
• Failure to recover to grade 1 or baseline from any adverse events (AEs) (Common Terminology Criteria for Adverse Events \[CTCAE\] version \[v\] 5.0) related to prior anticancer therapy
• EXCEPTIONS: Alopecia, lymphopenia, peripheral neuropathy, and ototoxicity ≤ grade 3)
• Known history of the following conditions:
• Allergy to gadolinium contrast agents
• Patients known to be HIV positive and currently receiving antiretroviral therapy
• NOTE: Patients known to be HIV positive, but without clinical evidence of an immunocompromised state, are eligible for this trial
• Inability to undergo MRI scans
• Uncontrolled intercurrent illness including, but not limited to:

Sponsors & Collaborators

• Mayo Clinic: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

RECRUITING

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Conditions

Glioblastoma

Interventions

Specimen Handling; Magnetic Resonance Spectroscopy; Aminolevulinic Acid

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques; Spectrum Analysis; Chemistry Techniques, Analytical; Levulinic Acids; Keto Acids; Carboxylic Acids; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins

Study Officials

• Terence C. Burns, MD, PhD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Clinical Trials Referral Office

CONTACT

855-776-0015; mayocliniccancerstudies@mayo.edu

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 15, 2025
• First Posted: July 22, 2025
• Study Start: August 14, 2025
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: February 23, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)