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

NCT ID: NCT07076472

Last Updated: 2025-11-20

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 8 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-08-14
• Study Completion Date: 2026-12-31

Brief Summary

This phase Ib trial tests the safety, best dose, and effectiveness of SONALA-001 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, to kill tumor cells. SONALA-001 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 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, dose limiting toxicities (DLTs), maximum tolerated dose (MTD), maximum administered dose (MAD) and recommended phase 2 dose (RP2D) for future study after treatment with aminolevulinic acid intravenous formulation SONALA-001 (SONALA-001) 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 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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment (SONALA-001, MRgFUS)

Patients receive SONALA-001 IV over 15 minutes 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.

Group Type: EXPERIMENTAL

Biospecimen Collection

Intervention Type: PROCEDURE

Undergo blood sample collection

Computed Tomography

Intervention Type: PROCEDURE

Undergo CT

Electronic Health Record Review

Intervention Type: OTHER

Ancillary studies

Magnetic Resonance Imaging

Intervention Type: PROCEDURE

Undergo MRI

MRI-Guided Focused Ultrasound Ablation

Intervention Type: PROCEDURE

Undergo transcranial MRgFUS

Aminolevulinic Acid Intravenous Formulation SONALA-001

Intervention Type: DRUG

Given IV

Interventions

Biospecimen Collection

Undergo blood sample collection

Intervention Type: PROCEDURE

Computed Tomography

Undergo CT

Intervention Type: PROCEDURE

Electronic Health Record Review

Ancillary studies

Intervention Type: OTHER

Magnetic Resonance Imaging

Undergo MRI

Intervention Type: PROCEDURE

MRI-Guided Focused Ultrasound Ablation

Undergo transcranial MRgFUS

Intervention Type: PROCEDURE

Aminolevulinic Acid Intravenous Formulation SONALA-001

Given IV

Intervention Type: DRUG

Other Intervention Names

ALA Intravenous Formulation SONALA-001; SONALA 001; SONALA-001; SONALA001; Biological Sample Collection; Biospecimen Collected; Specimen Collection; CAT; CAT Scan; Computed Axial Tomography; Computerized Axial Tomography; Computerized axial tomography (procedure); Computerized Tomography; Computerized Tomography (CT) scan; CT; CT Scan; tomography; 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; Magnetic Resonance Imaging-guided High-Intensity Focused Ultrasound; Magnetic Resonance-guided Focused Ultrasound; MR-HIFU; MRgFUS; MRI Guided Focused Ultrasound Surgery; MRI-HIFU

Eligibility Criteria

Inclusion Criteria

• 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)
• Negative pregnancy test done ≤ 8 days prior to registration, for persons of childbearing potential only
• Provide written informed consent
• Willing to participate in the neuro-oncology biorepository [Institutional Review Board (IRB) 12-003458, principal investigator (PI): Jann Sarkaria, MD, PhD] for collecting and archiving biospecimens samples on neuro-oncology patients
• Willing to return to enrolling institution for follow-up (during the active monitoring phase of the study)

Exclusion Criteria

• 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:

• Ongoing or active infection
• Patients with platelet count < 100
• Symptomatic congestive heart failure
• Unstable angina pectoris
• Cardiac arrhythmia
• Or psychiatric illness/social situations that would limit compliance with study requirements
• History of myocardial infarction ≤ 6 months, or congestive heart failure requiring use of ongoing maintenance therapy for life-threatening ventricular arrhythmias
• Co-morbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Cancer Institute (NCI)

NIH

Sponsor Role: collaborator

Mayo Clinic

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Terence C. Burns, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Mayo Clinic

Locations

Mayo Clinic in Rochester

Rochester, Minnesota, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Clinical Trials Referral Office

Role: CONTACT

mayocliniccancerstudies@mayo.edu

855-776-0015

Facility Contacts

Clinical Trials Referral Office

Role: primary

mayocliniccancerstudies@mayo.edu

Related Links

https://www.mayo.edu/research/clinical-trials

Mayo Clinic Clinical Trials

Other Identifiers

NCI-2025-04501

Identifier Type: REGISTRY

Identifier Source: secondary_id

24-013192

Identifier Type: OTHER

Identifier Source: secondary_id

MC240704

Identifier Type: OTHER

Identifier Source: secondary_id

R44CA275508

Identifier Type: NIH

Identifier Source: secondary_id

View Link

MC240704

Identifier Type: -

Identifier Source: org_study_id