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Focused Ultrasound and Etoposide for Diffuse Midline Glioma

This study is looking at a new way to deliver the drug etoposide to brain tumors in children and young adults (ages 4-21) with Diffuse Midline Glioma (DMG) or Diffuse Intrinsic Pontine Glioma (DIPG). These are aggressive brain tumors. The study uses focused ultrasound (FUS) with microbubbles to temporarily open the blood-brain barrier (a natural protective shield around the brain) so more etoposide can reach the tumor. You would receive FUS treatments up to three times a week for two weeks, followed by a two-week rest. Oral etoposide (a chemotherapy drug) is taken daily for 21 days, followed by a one-week rest. The main goal is to see if this method can safely open the blood-brain barrier and how many side effects occur. The study plans to enroll 10 participants, but its current status is unclear.

Study design
This is an interventional study, meaning participants receive a specific treatment. It plans to enroll 10 participants.
What's involved
You would receive focused ultrasound treatments up to three times a week for two weeks, followed by a two-week rest. Oral etoposide is taken daily for 21 days, followed by a one-week rest, for up to four cycles.
Compensation
Not stated in the trial record.
Follow-up
Adverse events will be measured for up to 90 days after the last focused ultrasound treatment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05762419

FUS Etoposide for DMG

Recruiting
PHASE1Ages 4–21InterventionalTreatment
Columbia University
~10 participants
Updated 2026-01-28 on ClinicalTrials.gov
What's tested:Etoposide; Oral, 50 MgFocused ultrasound with neuro-navigator-controlled sonication

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of total adverse events
Measured over Up to 90 days after the end of the last focused ultrasound treatment
+1 more outcome measured

Conditions

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

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Where it's being run

1 sites across 1 states
New York1

Study leadership

  • Stergios Zacharoulis, MD · PRINCIPAL_INVESTIGATOR · Columbia University

Who to contact

Stergios Zacharoulis, MD
[email protected]
212-305-9770
Email the study team

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Eligibility criteria

Inclusion

Ages 4 - 21 years
Radiological diagnosis of Diffuse Midline Glioma with tumor involving the pons (intrinsic, pontine based infiltrative lesion; hypointense on T1 weighted images (T1WIs) and hyperintense in T2 sequences, with mass effect on the adjacent structures and occupying at least 50% of the pons), thalami, and/or histological confirmation of H3K27M mutation of pontine or thalamic glioma. Subjects must have evidence of clinical and/or radiographic progression of disease.
Lansky performance status score of at least 60 for subjects 16 years of age or younger.
Karnofsky performance status of at least 60 for subjects greater than 16 years of age
Organ Function:
Adequate hematologic function defined as:
Peripheral absolute neutrophil count ≥ 1,500/µL
Platelet count ≥ 100,000/µL
Partial thromboplastin time (PTT) and activated partial thromboplastin time (APTT): within normal institutional limits
Adequate renal function defined as:
Potassium and magnesium levels within institutional limits
Serum creatinine below the institutional upper limit of normal (ULN) for age and gender, or creatinine clearance: ≥ 60 mL/min/1.73m2
Adequate hepatic function defined as:
Total bilirubin below the institutional ULN for age
Aspartate aminotransferase (AST)/alanine aminotransferase (ALT) ≤ 2.5 × institutional ULN
Prior Therapy:
Subjects must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment.
Cytotoxic chemotherapy or anti-cancer agents known to be myelosuppressive: at least 21 days after the last dose of cytotoxic or myelosuppressive chemotherapy.
Anti-cancer agents not known to be myelosuppressive: at least 7 days must have elapsed from last dose of agent.
Antibodies: at least 21 days must have elapsed from infusion of last dose of antibody.
Interleukins, interferons, and cytokines: at least 21 days must have elapsed since the completion of interleukins, interferon, or cytokines.
Stem cell infusions: at least 42 days must have elapsed after completion of an autologous stem cell infusion, and at least 84 days must have elapsed after completion of an allogeneic stem cell infusion.
Cellular therapy: at least 42 days must have elapsed since the completion of any type of cellular therapy
Radiotherapy (XRT): at least 1 month must have elapsed after local XRT.
Subjects must be on a stable or decreasing dose of steroids, as well as stable dose of anti-seizure medication for at least 1 week.
Subject able to give consent

Exclusion

Subjects that have previously received etoposide therapy
Subjects unable to tolerate study procedures and/or anesthesia based on the opinion of the principal investigator
Uncontrolled seizure disorder
Pregnancy or Breast-Feeding: pregnant or breast-feeding women will not be entered on this study, since there is yet no available information regarding human fetal or teratogenic toxicities; a pregnancy test must be obtained in girls who are post-menarchal. Males with female partners of reproductive potential or females of reproductive potential may not participate unless they have agreed to use two effective methods of birth control- including a medically accepted barrier method of contraception (e.g., a male or female condom) for the entire period in which they are receiving protocol therapy and for at least 1 month following their last study treatment requirement. Abstinence is an acceptable method of birth control. Women of childbearing potential will be provided a routine quantitative beta-human chorionic gonadotropin (B-hCG) test during the pre-study phase, prior to enrollment and each cycle.
Concomitant medications: subjects who are currently receiving another investigational drug or other anti-cancer agents are not eligible.
Screening EKG with a QTc \> 450 msec.
Subjects with evidence of active systemic infection
Subjects with a documented allergy to compounds of similar chemical or biologic composition to etoposide or gadolinium compounds
Subjects with implanted metallic or electrical devices
Subjects with uncontrollable hypertension
Subjects with a documented bleeding disorder
Subjects with history of structural cardiac anomalies or arrhythmias
Subjects with history of unprovoked stroke or signs of stroke in the area of FUS target
Subjects with SARS-CoV-2 infection requiring hospitalization in the past month and requires anticoagulation as per the Columbia University Irving Medical Center (CUIMC) institutional "Anticoagulation for COVID-19 Positive Pediatric Inpatients" guidelines (See Appendix B)
Subjects with coagulopathy or under anticoagulant therapy.
Subjects with signs of impending herniation or an acute or previous intratumoral hemorrhage
Subjects with spinal cord diffuse midline glioma
Subjects receiving a drug where CNS toxicity is reasonably suspected

What this trial measures

  • Number of total adverse eventsUp to 90 days after the end of the last focused ultrasound treatment

    This is to evaluate the safety of using focused ultrasound to open the blood brain barrier at one or two sites for administration of etoposide in children with progressive diffuse midline glioma. Safety will be assessed by adverse events as per the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 (including abnormalities from physical and neurologic examinations, laboratory values, and radiographic results).

  • Number of patients with successful opening of the blood brain barrierTreatment will consist of up to 4 cycles, each lasting 28 days. During the first 2 weeks of each cycle, subjects will receive FUS therapy for a maximum of 3 times per week, concurrent with etoposide.

    This is to evaluate the feasibility of using FUS to open the blood brain barrier at one or two sites for administration of etoposide in children with progressive diffuse midline glioma. The study defines feasibility as successful opening of the blood brain barrier.