RecruitingPhase 1NCT05762419

FUS Etoposide for DMG

A Feasibility Study Examining the Use of Non-Invasive Focused Ultrasound (FUS) With Oral Etoposide Administration in Children With Progressive Diffuse Midline Glioma (DMG)

Sponsor

Columbia University

Enrollment

10 participants

Start Date

Jun 5, 2023

Study Type

INTERVENTIONAL

Conditions

Diffuse Intrinsic Pontine Glioma Diffuse Midline Glioma, H3 K27M-Mutant

Summary

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.

Eligibility

Min Age: 4 YearsMax Age: 21 Years

Plain Language Summary

Simplified for easier understanding

This trial is studying whether focused ultrasound (FUS) can be used to temporarily open the protective barrier around the brain, allowing a chemotherapy drug called etoposide to better penetrate and treat diffuse midline glioma (DMG) — a rare, aggressive brain tumor that often affects children and young adults. **You may be eligible if...** - You are 4–21 years old - You have been diagnosed with diffuse midline glioma involving the brainstem (pons), thalamus, or confirmed by biopsy to have the H3K27M mutation - Your tumor has shown signs of growth or worsening (progression) - You are well enough to function (Lansky score at least 60 for those 16 and under; Karnofsky at least 60 for those over 16) - You have adequate blood counts and organ function **You may NOT be eligible if...** - You have active serious infection or uncontrolled bleeding disorder - You have a metal implant in your head or shrapnel that is incompatible with ultrasound - You are pregnant Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGEtoposide; Oral, 50 Mg

Subjects will receive focused ultrasound sonication followed by once daily oral etoposide (50mg/m\^2/dose). Oral etoposide will be taken every day for 21 days, followed by one week of rest. For the first cycle, etoposide will be administered immediately following confirming of the blood brain barrier opening through contrast magnetic resonance imaging (MRI) which will occur within 4 hours of the focused ultrasound procedure. For subsequent cycles, etoposide will be administered immediately following the focused ultrasound procedure. Subjects may continue for a maximum of 4 cycles.

DEVICEFocused ultrasound with neuro-navigator-controlled sonication

Focused ultrasound sonication will be performed a maximum of three times a week for two weeks with two weeks of rest.

Locations(1)

Columbia University Irving Medical Center

New York, New York, United States

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NCT05762419

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