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)
Mayo Clinic
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.
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 activit…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
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 registrat…
Interventions
- DrugAminolevulinic Acid Intravenous Formulation SONALA-001
Given IV
- ProcedureBiospecimen Collection
Undergo blood sample collection
- ProcedureComputed Tomography
Undergo CT
- OtherElectronic Health Record Review
Ancillary studies
- ProcedureMagnetic Resonance Imaging
Undergo MRI
- ProcedureMRI-Guided Focused Ultrasound Ablation
Undergo transcranial MRgFUS
- Drug
Location
- Mayo Clinic in RochesterRochester, Minnesota