Actively Recruiting
A Feasibility Study of Non-Invasive Focused Ultrasound with Oral Etoposide in Children with Progressive Diffuse Midline Glioma
Led by Columbia University · Updated on 2026-01-28
10
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
Sponsors
C
Columbia University
Lead Sponsor
F
Focused Ultrasound Foundation
Collaborating Sponsor
AI-Summary
What this Trial Is About
Diffuse midline gliomas, including diffuse intrinsic pontine gliomas, affect children and have a poor prognosis with limited treatment options. Many drugs fail to reach the brain tumor due to the blood brain barrier (BBB), which limits drug delivery. This study evaluates the safety and feasibility of using focused ultrasound (FUS) with microbubbles and neuro-navigator-controlled sonication to temporarily open the BBB in children with progressive diffuse midline glioma, allowing better drug delivery with oral etoposide treatment. Participants will receive focused ultrasound sonication targeting one to two tumor sites up to three times per week for two weeks, followed by two weeks of rest. Oral etoposide (50 mg/m2) will be taken daily for 21 days followed by a one-week break, for up to four cycles. The opening of the BBB will be confirmed by magnetic resonance imaging (MRI) after the FUS procedure. The study focuses on combining the non-invasive FUS technology with oral etoposide to assess if this method can safely increase drug delivery to the tumor. During the study, children will undergo regular MRI scans to confirm BBB opening and monitor tumor changes. Researchers will track adverse events up to 90 days after treatment to assess safety. The study will also evaluate progression-free survival and overall survival at three months after the last FUS treatment. Participants will be closely monitored throughout the treatment cycles and the follow-up period to assess feasibility and safety of this approach.
CONDITIONS
Brief Title
FUS Etoposide for DMG
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Ages 4 to 21 years
- Radiological diagnosis of diffuse midline glioma involving the pons or thalami, or histological confirmation of H3K27M mutation
- Evidence of clinical or radiographic progression of disease
- Lansky performance status of at least 60 for those 16 years or younger
- Karnofsky performance status of at least 60 for those older than 16 years
- Adequate hematologic function (ANC ≥ 1,500/µL, platelets ≥ 100,000/µL, normal PTT/APTT)
- Adequate renal function (potassium and magnesium within limits, creatinine below institutional upper limit or clearance ≥ 60 mL/min/1.73m2)
- Adequate hepatic function (bilirubin below institutional limit, AST/ALT ≤ 2.5 × upper limit)
- Recovered from prior anti-cancer therapies with specified minimum time intervals
- Stable or decreasing steroid dose and stable anti-seizure medication dose for at least 1 week
- Able to give consent
You will not qualify if you...
- Prior etoposide therapy
- Unable to tolerate study procedures or anesthesia
- Uncontrolled seizure disorder
- Pregnancy or breastfeeding
- Concurrent use of other investigational or anti-cancer drugs
- QTc greater than 450 msec on screening EKG
- Active systemic infection
- Allergy to etoposide or gadolinium compounds
- Implanted metallic or electrical devices
- Uncontrollable hypertension
- Documented bleeding disorders
- History of cardiac anomalies or arrhythmias
- History or signs of stroke in FUS target area
- Recent SARS-CoV-2 infection requiring hospitalization
- Coagulopathy or anticoagulant therapy
- Signs of impending herniation or intratumoral hemorrhage
- Spinal cord diffuse midline glioma
- Use of drugs with suspected CNS toxicity
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 4 cycles of 28 days each
Participants receive focused ultrasound sonication up to three times a week for two weeks, combined with once daily oral etoposide for 21 days followed by one week of rest. This cycle may be repeated up to 4 times.
Up to 3 focused ultrasound sessions per week during the first 2 weeks of each cycle; daily oral medication for 21 days per cycle
Duration - Up to 90 days after treatment ends
Participants are monitored for safety and treatment effects for up to 90 days after the last focused ultrasound treatment.
Regular follow-up visits (visit schedule may vary)
Trial Site Locations
Total: 1 location
1
Columbia University Irving Medical Center
New York, New York, United States, 10032
Actively Recruiting
Research Team
S
Stergios Zacharoulis, MD
J
James H Garvin, MD, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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