Study of Non-Invasive Focused Ultrasound with Oral Etoposide in Children and Young Adults Ages 4 to 21 With Progressive Diffuse Midline Glioma

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 mgm2 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.

FUS Etoposide for DMG