ExAblate 4000 System for Brain Tumor

Recruiting at 1 trial location

Age: < 65

Sex: Any

Trial Phase: Academic

Sponsor: InSightec

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

Compensation: Varies

Number of Visits: 1

Duration: 1 day

20 Participants Needed

Trial Summary

What is the purpose of this trial?

The goal of this prospective, non-randomized, single-arm, feasibility study is to develop data to evaluate the safety and feasibility of ExAblate 4000 treatment of benign intracranial tumors which require clinical intervention in pediatric and young adult subjects. Indication of Use: Ablation of benign intracranial tumors in children and young adults which are ExAblate accessible.

Will I have to stop taking my current medications?

The trial requires that you stay on a stable dose of all condition-related medications for 30 days before joining. However, you must stop taking anticoagulants (blood thinners) one week before the procedure and valproate derivatives (seizure medications) two weeks before.

How is the ExAblate 4000 System treatment different from other brain tumor treatments?

The ExAblate 4000 System is unique because it uses focused ultrasound to target brain tumors non-invasively, which is different from traditional methods like surgery or chemotherapy that involve more invasive procedures. ^{1 2 3 4 5}

Research Team

John Ragheb, MD

Principal Investigator

Miami Children's Research Institute - Nicklaus Children's Hospital

Eligibility Criteria

This study is for children and young adults with non-cancerous brain tumors that need treatment, can fit in an MRI machine, and have a head circumference of at least 52cm. They should be stable on current medications for their condition. Those with malignant tumors, unclear diagnosis without biopsy, or unable to undergo anesthesia are excluded.

Inclusion Criteria

My medication doses have been stable for the last 30 days.

I have a non-cancerous brain tumor that needs treatment and is unlikely to bleed.

Minimum head circumference will be 52cm

Exclusion Criteria

I cannot or do not want to have general anesthesia.

I am currently taking human growth hormone.

I have had thoughts of suicide or attempted suicide in the last year.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ExAblate 4000 treatment for benign intracranial tumors

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

Regular visits as per study protocol

Treatment Details

Interventions

• ExAblate 4000 System

Trial Overview The trial is testing the ExAblate 4000 System's safety and feasibility in treating benign brain tumors by using focused ultrasound waves to heat and destroy tumor tissue without invasive surgery.

Participant Groups

Group I: ExAblate 4000 System Experimental Treatment 1 Intervention

MR-Guided Focused Ultrasound

Who Is Running the Clinical Trial?

InSightec

Lead Sponsor

Trials

92

Recruited

3,800+

Dr. Maurice R. Ferré

InSightec

Chief Executive Officer

MD

Dr. Arjun Desai

InSightec

Chief Medical Officer

MD

Findings from Research

Malignant brain tumors remain difficult to treat, with long-term survival only improving from 6 to 15 months over the last 80 years, despite advancements in surgery, chemotherapy, and radiotherapy.

Immunotherapy, particularly using immune modulators like CTLA-4 and PD-1/PD-L1, is being actively researched in ongoing phase I and III trials, showing promise in targeting glioma antigens, but challenges remain in achieving complete responses and understanding the lack of adverse brain inflammation in these patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The value of EGFRvIII as the target for glioma vaccines. Lowenstein, PR., Castro, MG.[2021]

Cintredekin besudotox (CB) delivered via convection-enhanced delivery (CED) showed a favorable risk-benefit profile in treating recurrent glioblastoma multiforme (GBM), with a median survival of 42.7 weeks for GBM patients and 55.6 weeks for those with optimally positioned catheters.

The study involved 51 patients, with 46 having GBM, and found that the maximum tolerated concentration of CB was 0.5 microg/mL, leading to tumor necrosis, while infusion durations of up to 6 days were well tolerated, highlighting the importance of catheter placement for effective drug distribution.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Direct intracerebral delivery of cintredekin besudotox (IL13-PE38QQR) in recurrent malignant glioma: a report by the Cintredekin Besudotox Intraparenchymal Study Group. Kunwar, S., Prados, MD., Chang, SM., et al.[2022]

References

1. United States pubmed.ncbi.nlm.nih.gov

Drug encapsulated aerosolized microspheres as a biodegradable, intelligent glioma therapy. [2021]

2. England pubmed.ncbi.nlm.nih.gov

Biomarkers and smart intracranial devices for the diagnosis, treatment, and monitoring of high-grade gliomas: a review of the literature and future prospects. [2020]

3. Austria pubmed.ncbi.nlm.nih.gov

Implantable drug-releasing biodegradable microspheres for local treatment of brain glioma. [2019]

4. United States pubmed.ncbi.nlm.nih.gov

The value of EGFRvIII as the target for glioma vaccines. [2021]

5. United States pubmed.ncbi.nlm.nih.gov

Direct intracerebral delivery of cintredekin besudotox (IL13-PE38QQR) in recurrent malignant glioma: a report by the Cintredekin Besudotox Intraparenchymal Study Group. [2022]