SONALA-001 + Focused Ultrasound for Brain Cancer

Overseen By Clinical Trials Referral Office

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Mayo Clinic

Must not be taking: Antiretrovirals

Disqualifiers: Pregnancy, Porphyria, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 4

Duration: 6 weeks per cycle

8 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment approach for individuals with glioblastoma, a type of brain cancer that persists or recurs. The treatment combines a drug called SONALA-001 with focused ultrasound, a non-invasive technique using sound waves to target and kill tumor cells. The trial aims to determine if this combination is safe and effective in shrinking or controlling the tumor. Suitable candidates for this trial have glioblastoma that is worsening or has returned and have already undergone radiation treatment. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new therapy.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor to get personalized advice.

What prior data suggests that this combination therapy is safe for treating glioblastoma?

Research has shown that using SONALA-001 with MRgFUS (a type of ultrasound) appears safe for treating brain cancer. In earlier studies, participants did not experience serious side effects. No harmful reactions occurred at the tested doses, indicating the treatment is well-tolerated.

Another study found that patients lived longer than expected after their diagnosis, a positive sign. Although this trial remains in the early stages, the results so far are encouraging regarding its safety in humans. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about SONALA-001 for brain cancer because it combines a unique approach with focused ultrasound technology. Unlike standard treatments like surgery, radiation, or chemotherapy, SONALA-001 is delivered intravenously and then activated by transcranial MR-guided focused ultrasound (MRgFUS). This technique allows targeted activation of the drug directly at the tumor site, potentially increasing effectiveness while minimizing damage to surrounding healthy tissue. Additionally, the treatment cycles every 42 days, offering a new regimen that could reduce side effects and improve patient quality of life.

What evidence suggests that SONALA-001 with MRgFUS might be an effective treatment for glioblastoma?

Research has shown that SONALA-001, when combined with focused ultrasound (a type of sound wave therapy), might help treat glioblastoma, a kind of brain cancer. In early studies with the first five patients, two experienced tumor shrinkage of 25% or more. In this trial, participants will receive SONALA-001 with focused ultrasound, aiming to destroy tumor cells without harming nearby healthy tissue. SONALA-001 enhances the precision of focused ultrasound targeting, increasing treatment effectiveness. Initial results suggest it is well-tolerated and shows promise in reducing tumor size. ^{1 2 3 4 6}

Who Is on the Research Team?

Terence C. Burns, MD, PhD

Principal Investigator

Mayo Clinic

Are You a Good Fit for This Trial?

This trial is for patients with glioblastoma, a type of brain cancer, that's getting worse or has come back after treatment. Participants should be suitable for MRI scans and sonodynamic therapy using the Exablate 4000 Type 2.0 device.

Inclusion Criteria

Radiographic evidence of disease which may be measurable or non-measurable

Life expectancy of ≥ 12 weeks

Hemoglobin ≥ 9.0 g/dL

Exclusion Criteria

Allergy to gadolinium contrast agents

I have had a heart attack in the last 6 months or have heart failure needing treatment for serious irregular heartbeats.

Persons of childbearing potential unwilling to employ adequate contraception

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive SONALA-001 intravenously and undergo transcranial MRgFUS on day 1 of each cycle. Cycles repeat every 42 days.

6 weeks per cycle

1 visit per cycle (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment completion, with follow-ups at 30 days, and then every 3 to 6 months for up to 3 years.

Up to 3 years

Regular visits every 3 to 6 months

What Are the Treatments Tested in This Trial?

Interventions

• SONALA-001

Trial Overview The trial tests SONALA-001 combined with MRgFUS (sonodynamic therapy) to see if it's safe and effective against progressive or recurrent glioblastoma. It involves non-invasive ultrasound activation of SONALA-001 to target tumor cells.

How Is the Trial Designed?

Group I: Treatment (SONALA-001, MRgFUS) Experimental Treatment 6 Interventions

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.

Who Is Running the Clinical Trial?

Mayo Clinic

Lead Sponsor

Trials

3,427

Recruited

3,221,000+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

Citations

1. ascopubs.org ascopubs.org/doi/10.1200/JCO.2024.42.16_suppl.TPS2101

A phase 1/2 dose escalation and expansion study of ... A first-in-human Phase 0 trial (NCT04559685) indicated that SONALA-001 SDT was well-tolerated and not associated with off-target cellular or ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT07076472

NCT07076472 | Sonodynamic Therapy With SONALA-001 ... Giving sonodynamic therapy using SONALA-001 with MRgFUS may be safe, tolerable, and/or effective in treating patients with progressive or recurrent glioblastoma ...

3. sonalasense.com sonalasense.com/news/sonalasense-presents-preliminary-data-from-clinical-study-in-patients-with-deadly-pediatric-brain-tumor-

SonALAsense presents preliminary data from clinical study ... Initial clinical activity observed in the first 5 patients showed partial responses of at least 25% reduction in tumor size in 2 (40%) patients ...

4. abta.org abta.org/mindmatters/scientific_advances/sonala-001/

A Dose Escalation and Expansion Study of Sonodynamic ... When SONALA-001 is administered intravenously and combined with MRI ... Preliminary data from the first 10 patients treated in this Phase 0/1 ...

5. fusfoundation.org fusfoundation.org/posts/focused-ultrasound-for-pediatric-brain-cancer-initial-results-presented/

Focused Ultrasound for Pediatric Brain Cancer: Initial ... According to SonALAsense's press release, of the first five participants, two experienced at least a 25% reduction in tumor size through eight ...

6. endbraincancer.org endbraincancer.org/2024/06/pediatric-clinical-trial-for-dipg-brain-cancer/

Pediatric Clinical Trial for DIPG Brain Cancer Four patients treated have exceeded the median survival of 9-11 months post diagnosis. Patients treated have also maintained or improved their ...