FUS Etoposide for DMG
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age4-21
SponsorColumbia University
About this trial
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
Qualifiers
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
Disqualifiers
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.
Trial design
Treatments tested in this trial
- Etoposide; Oral, 50 Mg
- Focused ultrasound with neuro-navigator-controlled sonication
Treatment groups
10 Participants
are divided into 1 treatment groupSponsors and collaborators
Columbia University
Lead sponsor
Focused Ultrasound Foundation
Collaborator
Source ClinicalTrials.gov