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Recruiting Phase 1 NCT05762419

FUS Etoposide for DMG

Trial Parameters

Condition Diffuse Intrinsic Pontine Glioma
Sponsor Columbia University
Study Type INTERVENTIONAL
Phase Phase 1
Enrollment 10
Sex ALL
Min Age 4 Years
Max Age 21 Years
Start Date 2023-06-05
Completion 2026-12
Interventions
Etoposide; Oral, 50 MgFocused ultrasound with neuro-navigator-controlled sonication

Brief 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 Criteria

Inclusion Criteria: * 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

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