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.
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
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
Single group
Aminolevulinic Acid Intravenous Formulation SONALA-001
DrugGiven IV
Biospecimen Collection
Procedure/SurgeryUndergo blood sample collection
Computed Tomography
Procedure/SurgeryUndergo CT
Electronic Health Record Review
Other interventionAncillary studies
Magnetic Resonance Imaging
Procedure/SurgeryUndergo MRI
MRI-Guided Focused Ultrasound Ablation
Procedure/SurgeryUndergo transcranial MRgFUS
Oral Aminolevulinic Acid Hydrochloride
DrugGiven PO
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).
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).
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).
Dose-limiting event (DLE)
AEs will be evaluated using CTCAE v5. The target DLE rate is \< 33%.
Overall response rate (ORR)
Assessed by the proportion of patients with complete or partial response.
Clinical benefit rate
Assessed by the proportion of patients with complete response, partial response or stable disease.
Progression-free survival (PFS)
Defined as the time from registration to the earliest date of documentation of disease progression, relapse, or death due to any cause.
PFS at 6 months (PFS6)
The PFS6 rate will be estimated as the number of patients who are progression-free and alive at 6 months.
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Mayo Clinic
Lead sponsor
National Cancer Institute (NCI)
Collaborator
