What side effects are associated with this type of intervention?

Common treatments for Glioblastoma include surgery, radiation therapy, and chemotherapy. Known side effects of these treatments can include fatigue, headaches, hair loss, nausea, and cognitive changes. For treatments similar to Sonodynamic Therapy (SDT) activated by Focused Ultrasound, such as other forms of targeted therapy or non-invasive treatments, side effects may include localized pain, swelling, and temporary changes in neurological function. The trial of SONALA-001 aims to evaluate the safety and efficacy of this novel approach, with a focus on identifying any dose-limiting toxicities and other adverse effects.

What prior FDA approvals exist?

The FDA has approved several treatments for Glioblastoma (GBM), including temozolomide, an oral alkylating agent, and bevacizumab, an anti-VEGF monoclonal antibody. Temozolomide is often used in combination with radiation therapy as a standard treatment for newly diagnosed GBM. Bevacizumab is typically used for recurrent GBM. While there are no FDA-approved treatments exactly like the sonodynamic therapy (SDT) using SONALA-001 and focused ultrasound, these approvals highlight the ongoing efforts to find effective treatments for GBM. The SONALA-001 trial represents an innovative approach by combining a sonosensitizer with focused ultrasound to target GBM cells.

What other types of research exist?

Promising novel alternatives to SONALA-001 for Glioblastoma patients in 2024 include: 1) Tumor Treating Fields (TTFields) - a non-invasive treatment that uses electric fields to disrupt cancer cell division. 2) CAR T-cell therapy - a type of immunotherapy where a patient's T-cells are modified to target GBM cells. 3) Oncolytic virus therapy - using genetically modified viruses to selectively infect and kill cancer cells. 4) Targeted therapies such as Bevacizumab (anti-VEGF) and newer agents targeting specific genetic mutations in GBM. 5) Combination therapies involving checkpoint inhibitors (e.g., Pembrolizumab) with other treatments to enhance immune response against GBM.

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Procedure

Sonodynamic Therapy for Glioblastoma

Phase 1 & 2

Waitlist Available

Led By Stuart Marcus, MD, PhD

Research Sponsored by SonALAsense, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Must be 18 years or older at the screening visit

Histologically proven GBM that has recurred or progressed, and for which resection is not indicated

Must not have

Malignant disease, other than that being treated in this study

Diagnosis of porphyria

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 12 months

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new treatment for patients with a type of brain cancer called Glioblastoma Multiforme that has come back or continued to grow. The treatment uses a drug called SONALA-001, which is activated by sound waves from a special device. The goal is to see if this combination is safe and effective in targeting and killing cancer cells.

Who is the study for?

Adults with recurrent glioblastoma (GBM) who've had standard treatments and have a tumor volume of 5-30 mL. They must be stable on steroids if used, have good organ function, and a Karnofsky Performance Score over 70. Participants need to agree to effective birth control and not have certain medical conditions or recent treatments that could interfere with the study.

What is being tested?

The trial is testing SONALA-001 combined with MR-Guided Focused Ultrasound (MRgFUS) for safety, tolerable dosages, and early signs of effectiveness in treating GBM. It aims to find the best dose for future studies after one treatment session using this sonodynamic therapy approach.

What are the potential side effects?

Potential side effects are not detailed but may include reactions related to the drug SONALA-001 or issues from focused ultrasound like discomfort at the treatment site. Side effects will be monitored closely to determine safe dosage levels.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am 18 years old or older.

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My glioblastoma has returned or worsened and surgery isn't an option.

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I am not pregnant and will use effective birth control during the trial.

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I am mostly able to care for myself but may not be able to do active work.

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I have been treated with radiotherapy and temozolomide.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have no other cancer besides the one being treated in this study.

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I have been diagnosed with porphyria.

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I still have side effects from cancer treatment that are not mild.

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My cancer is located in the brainstem or is diagnosed as gliomatosis cerebri.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Progression-free survival rate at 6 Months (Phase 2)

Safety and Tolerability of SONALA-001 SDT as assessed by the frequency and severity of dose-limiting toxicities (DLTs)

Safety and Tolerability of SONALA-001 SDT as assessed by the number of subjects with Adverse Events (AEs), adverse device effects (ADEs), serious AEs (SAEs) and serious device effects (SADEs)

+3 more

Secondary study objectives

To evaluate preliminary efficacy and Duration of Clinical Benefit (DOCB) (Phase 2)

To evaluate preliminary efficacy and Duration of Response (DOR) (Phase 1)

To evaluate preliminary efficacy and Overall Survival (OS) (Phase 1)

+12 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

5Treatment groups

Experimental Treatment

Group I: Cohort 5Experimental Treatment1 Intervention

Recommended Phase 2 Dose (RP2D) IV SONALA-001 (ALA) and MR-guided Focused Ultrasound (MRgFUS) Energy Level 5

Group II: Cohort 4Experimental Treatment1 Intervention

10 mg/kg IV SONALA-001 (ALA) and MR-guided Focused Ultrasound (MRgFUS) Energy Level 4

Group III: Cohort 3Experimental Treatment1 Intervention

10 mg/kg IV SONALA-001 (ALA) and MR-guided Focused Ultrasound (MRgFUS) Energy Level 3

Group IV: Cohort 2Experimental Treatment1 Intervention

10 mg/kg IV SONALA-001 (ALA) and MR-guided Focused Ultrasound (MRgFUS) Energy Level 2

Group V: Cohort 1Experimental Treatment1 Intervention

10 mg/kg IV SONALA-001 (ALA) and MR-guided Focused Ultrasound (MRgFUS) Energy Level 1

0 / 9Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Glioblastoma include surgery, radiation therapy, and chemotherapy. Surgery aims to remove as much of the tumor as possible, reducing the tumor burden. Radiation therapy uses high-energy beams to kill cancer cells and shrink tumors by damaging their DNA. Chemotherapy, such as temozolomide, works by interfering with the DNA replication of cancer cells, leading to cell death. These treatments are crucial for GBM patients as they target the rapidly dividing cancer cells, aiming to control tumor growth and prolong survival. The SONALA-001 trial explores Sonodynamic Therapy (SDT) activated by Focused Ultrasound (FUS), which combines a sonosensitizer drug (SONALA-001) with FUS to produce reactive oxygen species that selectively kill cancer cells. This approach is significant as it offers a targeted treatment option, potentially reducing damage to surrounding healthy tissue and improving outcomes for GBM patients.