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

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

Conditions

Brain Tumor

Study Overview

Study Details

Study overview

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

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)

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

Condition
Brain Tumor
Intervention / Treatment

-

Contacts and Locations

Washington

Children's National Medical Center, Washington, District of Columbia, United States, 20010

Miami

Nicklaus Children's Hospital, Miami, Florida, United States, 33155

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.

For general information about clinical research, read Learn About Studies.

Eligibility 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

Ages Eligible for Study

5 Years to 21 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

InSightec,

Study Record Dates

2026-01