RECRUITING

Blood Brain Barrier (BBB) Disruption Using Exablate Focused Ultrasound With Doxorubicin for Treatment of Pediatric DIPG

Conditions

Brain Tumor DIPG Focused Ultrasound Chemotherapy Phase I trial Pediatrics

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The purpose of this study is to evaluate the safety and efficacy of targeted blood brain barrier disruption with Exablate Model 4000 Type2.0/2.1 in combination with Doxorubicin therapy for the treatment of DIPG in pediatric patients

Official Title

A Safety and Feasibility Study to Evaluate Blood Brain Barrier Disruption Using Exablate MR Guided Focused Ultrasound in Combination With Doxorubicin in Treating Pediatric Patients With Diffuse Intrinsic Pontine Gliomas (DIPG)

Quick Facts

Study Start:2023-01-04

Study Completion:2026-01

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05630209

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:5 Years to 21 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT

Inclusion Criteria	Exclusion Criteria
* Age between 5 and 21 years, inclusive * Patient diagnosed with DIPG * At least 4-week and not greater than 12 weeks from completion of radiation therapy * Post-radiation imaging does not show evidence of necrosis/ hemorrhage or other features that contraindicate MRgFUS * If brain surgery occurred, at least 14 days passed since last brain surgery and the patient is fully recovered and neurologically stable * If on steroids, stable or decreasing dose for at least 7 days prior to study entry * Stable or improving neurologic status for 7 days prior to study entry * Able and willing to give consent and/or assent or have a legal guardian who is able and willing to do so * Able to attend all study visits and with life expectancy of at least 6 months	* Tumor not visible on any pre-therapy or post-radiation imaging * Previous participation in other chemotherapy, molecularly targeted therapy, or immunotherapy treatment-related phase 1 or 2 trials * Symptoms and signs of increased intracranial pressure * Subject with metastatic disease * Subject with ventricular peritoneal shunt * Subject receiving bevacizumab (Avastin) therapy or increasing doses of steroids * Anti-coagulant therapy, or medications known to increase risk of hemorrhage, (e.g., ASA, non-steroidal anti-inflammatory drugs \[NSAIDs\], statins) within washout period prior to treatment * History of a bleeding disorder, coagulopathy or with a history of spontaneous tumor hemorrhage. * Hypertension per age * Cerebral or systemic vasculopathy, including intracranial thrombosis, vascular malformation, cerebral aneurysm, or vasculitis * Immunosuppression (corticosteroids to prevent/treat brain edema are permitted). * Patients with positive HIV status * Active seizure disorder or epilepsy (seizures despite medical treatment) for a minimum of 4 weeks prior to first cycle/Exablate BBBD procedure captured by history * Known sensitivity to gadolinium-based contrast agents * Known sensitivity to DEFINITY® ultrasound contrast agent or known hypersensitivity to perflutren microsphere or its components, e.g., polyethylene glycol * Contraindication to Doxorubicin * Previous treatment with complete cumulative doses of Doxorubicin, daunorubicin, idarubicin, and/or other anthracyclines and anthracenediones * Severely impaired renal function with estimated glomerular filtration rate \<2 standard deviations for age * Patients that may require trastuzumab during the study * Patients that may require inhibitors and inducers of CYP3A4, CYP2D6, and/or P-gp during the study

Inclusion Criteria

Exclusion Criteria

* Age between 5 and 21 years, inclusive
* Patient diagnosed with DIPG
* At least 4-week and not greater than 12 weeks from completion of radiation therapy
* Post-radiation imaging does not show evidence of necrosis/ hemorrhage or other features that contraindicate MRgFUS
* If brain surgery occurred, at least 14 days passed since last brain surgery and the patient is fully recovered and neurologically stable
* If on steroids, stable or decreasing dose for at least 7 days prior to study entry
* Stable or improving neurologic status for 7 days prior to study entry
* Able and willing to give consent and/or assent or have a legal guardian who is able and willing to do so
* Able to attend all study visits and with life expectancy of at least 6 months

* Tumor not visible on any pre-therapy or post-radiation imaging
* Previous participation in other chemotherapy, molecularly targeted therapy, or immunotherapy treatment-related phase 1 or 2 trials
* Symptoms and signs of increased intracranial pressure
* Subject with metastatic disease
* Subject with ventricular peritoneal shunt
* Subject receiving bevacizumab (Avastin) therapy or increasing doses of steroids
* Anti-coagulant therapy, or medications known to increase risk of hemorrhage, (e.g., ASA, non-steroidal anti-inflammatory drugs \[NSAIDs\], statins) within washout period prior to treatment
* History of a bleeding disorder, coagulopathy or with a history of spontaneous tumor hemorrhage.
* Hypertension per age
* Cerebral or systemic vasculopathy, including intracranial thrombosis, vascular malformation, cerebral aneurysm, or vasculitis
* Immunosuppression (corticosteroids to prevent/treat brain edema are permitted).
* Patients with positive HIV status
* Active seizure disorder or epilepsy (seizures despite medical treatment) for a minimum of 4 weeks prior to first cycle/Exablate BBBD procedure captured by history
* Known sensitivity to gadolinium-based contrast agents
* Known sensitivity to DEFINITY® ultrasound contrast agent or known hypersensitivity to perflutren microsphere or its components, e.g., polyethylene glycol
* Contraindication to Doxorubicin
* Previous treatment with complete cumulative doses of Doxorubicin, daunorubicin, idarubicin, and/or other anthracyclines and anthracenediones
* Severely impaired renal function with estimated glomerular filtration rate \<2 standard deviations for age
* Patients that may require trastuzumab during the study
* Patients that may require inhibitors and inducers of CYP3A4, CYP2D6, and/or P-gp during the study

Contacts and Locations

Study Contact

Nadir Alikacem

CONTACT

+12146302000

nadira@insightec.com

Study Locations (Sites)

Children's National Medical Center

Washington, District of Columbia, 20010

United States

Nicklaus Children's Hospital

Miami, Florida, 33155

United States

Collaborators and Investigators

Sponsor: InSightec

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-01-04

Study Completion Date2026-01

Study Record Updates

Study Start Date2023-01-04

Study Completion Date2026-01

Terms related to this study

Keywords Provided by Researchers

DIPG
Focused Ultrasound
Chemotherapy
Phase I trial
Pediatrics

Additional Relevant MeSH Terms

Brain Tumor