FUS Etoposide for DMG - A Feasibility Study
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ClinicalTrials.gov Identifier: NCT05762419 |
Recruitment Status :
Recruiting
First Posted : March 9, 2023
Last Update Posted : March 9, 2023
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Tracking Information | |||||||||
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First Submitted Date ICMJE | February 6, 2023 | ||||||||
First Posted Date ICMJE | March 9, 2023 | ||||||||
Last Update Posted Date | March 9, 2023 | ||||||||
Estimated Study Start Date ICMJE | March 2023 | ||||||||
Estimated Primary Completion Date | January 2025 (Final data collection date for primary outcome measure) | ||||||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE | Same as current | ||||||||
Change History | No Changes Posted | ||||||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE | Same as current | ||||||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||||||
Descriptive Information | |||||||||
Brief Title ICMJE | FUS Etoposide for DMG - A Feasibility Study | ||||||||
Official Title ICMJE | A Feasibility Study Examining the Use of Non-Invasive Focused Ultrasound (FUS) With Oral Etoposide Administration in Children With Progressive Diffuse Midline Glioma (DMG) | ||||||||
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. |
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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). |
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Study Type ICMJE | Interventional | ||||||||
Study Phase ICMJE | Phase 1 | ||||||||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE |
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Intervention ICMJE |
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Study Arms ICMJE | Experimental: Focused ultrasound using oral etoposide
All patients enrolled in the study will be treated with oral etoposide after receiving focused ultrasound (FUS) treatment with microbubbles and neuro-navigator-controlled sonication.
Interventions:
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Publications * | Not Provided | ||||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||||
Recruitment Status ICMJE | Recruiting | ||||||||
Estimated Enrollment ICMJE |
10 | ||||||||
Original Estimated Enrollment ICMJE | Same as current | ||||||||
Estimated Study Completion Date ICMJE | January 2027 | ||||||||
Estimated Primary Completion Date | January 2025 (Final data collection date for primary outcome measure) | ||||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 4 Years to 21 Years (Child, Adult) | ||||||||
Accepts Healthy Volunteers ICMJE | No | ||||||||
Contacts ICMJE |
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Listed Location Countries ICMJE | United States | ||||||||
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Administrative Information | |||||||||
NCT Number ICMJE | NCT05762419 | ||||||||
Other Study ID Numbers ICMJE | AAAU1228 | ||||||||
Has Data Monitoring Committee | Yes | ||||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | Cheng-Chia (Fred) Wu, Columbia University | ||||||||
Original Responsible Party | Same as current | ||||||||
Current Study Sponsor ICMJE | Cheng-Chia (Fred) Wu | ||||||||
Original Study Sponsor ICMJE | Same as current | ||||||||
Collaborators ICMJE | Focused Ultrasound Foundation | ||||||||
Investigators ICMJE |
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PRS Account | Columbia University | ||||||||
Verification Date | February 2023 | ||||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |