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

NCT05630209 | Active Not Recruiting Phase 1 DMC FDA Drug FDA Device

• 1 other identifier: BT016
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 3

Brief Summary

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

Keywords

DIPG; Focused Ultrasound; Chemotherapy; Phase I trial; Pediatrics

Outcome Measures

Primary Outcomes (1)

• Adverse Events

  All adverse events from first treatment to end of study will be documented and reported according to the CTCAE terminology and severity scale

  2 years

Secondary Outcomes (1)

• Blood Brain Barrier Disruption (BBBD)

  immediately post BBBD sonication

Other Outcomes (1)

• Progression Free Survival (PFS)

  2 years

Study Arms (1)

Blood Brain Barrier Disruption (BBBD)

EXPERIMENTAL

Exablate MR Guided Focused Ultrasound for Blood Brain Barrier Disruption with Doxorubicin for treating pediatric patients with DIPG

Device: Exablate; Drug: Doxorubicin

Interventions

Exablate DEVICE

Blood Brain Barrier Disruption (BBBD) via Exablate Type 2 system with microbubble resonators on the day of Doxorubicin infusion to treat DIPG brain tumors

Also known as: Exablate BBBD

Blood Brain Barrier Disruption (BBBD)

Doxorubicin DRUG

Doxorubicin infusion

Also known as: Adriamycin, Rubex

Blood Brain Barrier Disruption (BBBD)

Eligibility Criteria

• Age: 5 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age between 5 and 21 years, inclusive. Subjects younger than 5 years old may be eligible after discussion with the Sponsor Medical Monitor/designee (i.e. DSMB).
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• InSightec: lead

Study Sites (3)

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

Location

Cook Children's Health Care System

Fort Worth, Texas, 76104, United States

Location

MeSH Terms

Conditions

Brain Neoplasms

Interventions

Doxorubicin

Condition Hierarchy (Ancestors)

Central Nervous System Neoplasms; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

Daunorubicin; Anthracyclines; Naphthacenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polycyclic Compounds; Aminoglycosides; Glycosides; Carbohydrates

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: DEVICE FEASIBILITY
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 18, 2022
• First Posted: November 29, 2022
• Study Start: January 4, 2023
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): October 1, 2026
• Last Updated: April 17, 2026

Record last verified: 2026-04

Locations

US (3)