Recruiting
Early Phase 1

SONALA-001 & MRgFUS

Sponsor:

Mayo Clinic

Code:

NCT07076472

Conditions

Progressive Glioblastoma

Recurrent Glioblastoma

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Aminolevulinic Acid Intravenous Formulation SONALA-001

Biospecimen Collection

Computed Tomography

Electronic Health Record Review

Magnetic Resonance Imaging

Study Details

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

Progressive Glioblastoma

Recurrent Glioblastoma

Study ID

NCT07076472

Start date

Aug 14, 2025

Status verified date

Aug, 2026

Completion date

Dec 31, 2031

Anticipated

Primary completion date

Dec 31, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

8 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Treatment (SONALA-001, MRgFUS)

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.

Interventions

Aminolevulinic Acid Intravenous Formulation SONALA-001

Given IV

Biospecimen Collection

Undergo blood sample collection

Computed Tomography

Undergo CT

Electronic Health Record Review

Ancillary studies

Magnetic Resonance Imaging

Undergo MRI

MRI-Guided Focused Ultrasound Ablation

Undergo transcranial MRgFUS

Oral Aminolevulinic Acid Hydrochloride

Given PO

Primary outcome measure

  • Incidence of adverse events (AEs) [ Time Frame: Up to 30 days after last dose of study treatment ]
  • Incidence of grade 3 or higher non-hematologic AEs [ Time Frame: Up to 30 days after last dose of study treatment ]
  • Incidence of grade 4 or higher AE [ Time Frame: Up to 30 days after last dose of study treatment ]
  • Dose-limiting event (DLE) [ Time Frame: During first 6 weeks of therapy [first cycle of treatment (cycle length = 42 days)] ]
  • Maximum tolerated dose (MTD) [ Time Frame: Up to 6 weeks ]

Central Contacts and Locations

Central contacts

Clinical Trials Referral Office

855-776-0015mayocliniccancerstudies@mayo.edu

Locations

Mayo Clinic in Rochester

Recruiting

Rochester, Minnesota, United States, 55905

Contacts

Clinical Trials Referral Office

mayocliniccancerstudies@mayo.edu

Principal Investigator:

Terry Burns, MD, PhD

More Information

Sponsor

Mayo Clinic

Last update posted

Aug 25, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Mayo Clinic on 2026-08-25.