Focused Ultrasound + Etoposide for Brain Tumor

Overseen By James H Garvin, MD, PhD

Age: < 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Columbia University

Must be taking: Oral etoposide

Must not be taking: Investigational drugs, Anticancer agents

Disqualifiers: Uncontrolled seizures, Pregnancy, Infection, others

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

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 3

Duration: Up to 16 weeks

10 Participants Needed

Trial Summary

What is the purpose of this trial?

The blood brain barrier (BBB) prevents some drugs from successfully reaching the target tumor. Focused Ultrasound (FUS) using microbubbles and neuro-navigator controlled sonication is a non-invasive method of temporarily opening up the blood brain barrier to allow a greater concentration of the drug to reach into the brain tumor. This may improve response and may also reduce system side effects in the patient. The primary purpose of this study is to evaluate the feasibility of safely opening the blood brain barrier in children with progressive diffuse midline gliomas (DMG) treated with oral etoposide using focused ultrasound with microbubbles and neuro-navigator-controlled sonication. For the purpose of the study, the investigators will be opening up the blood brain barrier temporarily in one or two locations around the tumor using the non-invasive focused ultrasound technology, and administrating oral etoposide in children with progressive diffuse midline glioma.

Research Team

James H Garvin, MD, PhD

Principal Investigator

Columbia University

Eligibility Criteria

Children and young adults aged 4-21 with a specific brain tumor called Diffuse Midline Glioma, involving the pons or thalami, can join this trial. They must have a certain level of physical ability (Lansky or Karnofsky score ≥60), proper organ function, and be off other cancer treatments for a set time. Those who've had etoposide before, are pregnant/breastfeeding, have uncontrolled seizures or infections, heart issues, bleeding disorders, metal implants/electrical devices in body cannot participate.

Inclusion Criteria

Subject able to give consent

I am between 4 and 21 years old.

My liver tests are within normal limits.

Exclusion Criteria

I have been treated with etoposide before.

Subjects unable to tolerate study procedures and/or anesthesia based on the opinion of the principal investigator

Pregnancy or Breast-Feeding

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive focused ultrasound (FUS) treatment with microbubbles and neuro-navigator-controlled sonication, concurrent with oral etoposide administration

Up to 16 weeks

Up to 3 visits per week during the first 2 weeks of each 28-day cycle

Follow-up

Participants are monitored for safety and effectiveness after treatment, including MRI and other radiological evidence to show successful BBB opening and closing

Up to 3 months

Monthly follow-up phone calls

Treatment Details

Interventions

• Etoposide
• Focused ultrasound with neuro-navigator-controlled sonication

Trial Overview The study is testing if focused ultrasound (FUS) can safely open the blood-brain barrier to let more of the drug etoposide reach brain tumors in children with progressive diffuse midline gliomas. FUS uses microbubbles and sound waves guided by neuro-navigation to target areas around the tumor while giving oral etoposide.

Participant Groups

Group I: Focused ultrasound using oral etoposide Experimental Treatment 2 Interventions

All patients enrolled in the study will be treated with oral etoposide after receiving focused ultrasound (FUS) treatment with microbubbles and neuro-navigator-controlled sonication.

Etoposide is already approved in United States, European Union, Canada for the following indications:

Approved in United States as Etoposide for:

• Testicular cancer
• Small cell lung cancer
• Non-Hodgkin lymphoma
• Acute myeloid leukemia

Approved in European Union as Etoposide for:

• Testicular cancer
• Small cell lung cancer
• Non-Hodgkin lymphoma
• Acute myeloid leukemia

Approved in Canada as Etoposide for:

• Testicular cancer
• Small cell lung cancer
• Non-Hodgkin lymphoma
• Acute myeloid leukemia

Who Is Running the Clinical Trial?

Columbia University

Lead Sponsor

Trials

1,529

Recruited

2,832,000+

Cheng-Chia (Fred) Wu

Lead Sponsor

Trials

2

Recruited

10+

Focused Ultrasound Foundation

Collaborator

Trials

18

Recruited

460+