Ignite Creation Date:
2024-05-06 @ 6:44 PM
Last Modification Date:
2024-10-26 @ 2:53 PM
Study NCT ID:
NCT05762419
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-05-17
First Post:
2023-02-06
Brief Title:
FUS Etoposide for DMG
Sponsor:
Cheng-Chia Fred Wu
Organization:
Columbia University
Study Overview
Official Title:
A Feasibility Study Examining the Use of Non-Invasive Focused Ultrasound FUS With Oral Etoposide Administration in Children With Progressive Diffuse Midline Glioma DMG
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
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
Detailed Description:
Diffuse midline gliomas constitute 10 of all pediatric central nervous system CNS tumors Subjects with Diffuse Intrinsic Pontine Gliomas DIPG have a poor prognosis with a median survival that is usually reported to be 9 months and nearly 90 of children die within 18 months from diagnosis The mainstay of treatment is radiation to the primary tumor site Surgical resection does not influence the outcome and is often not feasible in this part of the central nervous system
Many promising drugs for central nervous system disorders have failed to attain clinical success due to an intact blood brain barrier limiting their access from the systemic circulation into the brain Systemic administration of high doses may increase delivery to the brain but this approach risks significant side effects and systemic toxicities Direct delivery of the drugs to the brain by injection into the parenchyma bypasses the blood brain barrier however drug distribution from the site of injection tends to be limited
The technique of using focused ultrasound with microbubbles and neuro-navigator-controlled sonication can temporarily open up the blood brain barrier and allow for a greater concentration of drug to reach the tumor thus potentially improving response in patients
With the current study the investigators are planning to evaluate the safety and feasibility of using focused ultrasound and open-space neuronavigator-controlled sonication to open one to two tumor sites For the purpose of the study investigators will be administrating oral etoposide in children with progressive diffuse midline glioma This drug has a known toxicity profile dose and well-documented efficacy against many metastatic cancers Successful opening and closing of the blood brain barrier will be confirmed with periodic magnetic resonance imaging MRI
Many promising drugs for central nervous system disorders have failed to attain clinical success due to an intact blood brain barrier limiting their access from the systemic circulation into the brain Systemic administration of high doses may increase delivery to the brain but this approach risks significant side effects and systemic toxicities Direct delivery of the drugs to the brain by injection into the parenchyma bypasses the blood brain barrier however drug distribution from the site of injection tends to be limited
The technique of using focused ultrasound with microbubbles and neuro-navigator-controlled sonication can temporarily open up the blood brain barrier and allow for a greater concentration of drug to reach the tumor thus potentially improving response in patients
With the current study the investigators are planning to evaluate the safety and feasibility of using focused ultrasound and open-space neuronavigator-controlled sonication to open one to two tumor sites For the purpose of the study investigators will be administrating oral etoposide in children with progressive diffuse midline glioma This drug has a known toxicity profile dose and well-documented efficacy against many metastatic cancers Successful opening and closing of the blood brain barrier will be confirmed with periodic magnetic resonance imaging MRI
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
True
Is an Unapproved Device?:
True
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None