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

NCT05615623 | Active Not Recruiting Phase 1 DMC

• 1 other identifier: BT016C
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to evaluate the safety and efficacy of targeted blood brain barrier disruption with Exablate Model 4000 Type 2.0/2.1 in combination with Doxorubicin therapy for the treatment of DIPG in pediatric patients

Conditions

Brain Tumor

Keywords

DIPG; Focused Utrasound; 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 (2)

• Tumor Volume

  Immediately post BBBD sonication

• Progression Free Survival (PFS) and Overall Survival (OS)

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

You may qualify if:

• Age between 5 and 18 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
• Able to attend all study visits and with life expectancy of at least 6 months
• Able and willing to give consent and/or assent or have a legal guardian who is able and willing to do so
• If on steroids, stable or decreasing dose for at least 7 days prior to study entry
• If brain surgery occurred, at least 14 days passed since last brain surgery and the patient is fully recovered and neurologically stable

You may not qualify if:

• Evidence of cranial or systemic infection
• Known life-threatening systemic disease
• Previous treatment with complete cumulative doses of Doxorubicin, daunorubicin, idarubicin, and/or other anthracyclines and anthracenediones
• Contraindication to Doxorubicin. - Known sensitivity to DEFINITY® ultrasound contrast agent or known hypersensitivity to perflutren microsphere or its components, e.g., polyethylene glycol. - Known sensitivity to gadolinium-based contrast agents
• 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
• Patients with positive HIV status. - Immunosuppression (corticosteroids to prevent/treat brain edema are permitted)
• Cerebral or systemic vasculopathy, including intracranial thrombosis, vascular malformation, cerebral aneurysm, or vasculitis
• Hypertension per age
• History of a bleeding disorder, coagulopathy or with a history of spontaneous tumour hemorrhage
• 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
• Patient receiving bevacizumab (Avastin) therapy or increasing doses of steroids
• Symptoms and signs of increased intracranial pressure
• Previous participation in other chemotherapy, molecularly targeted therapy or immunotherapy treatment-related phase 1 or 2 trials
• Tumor not visible on any pre-therapy or post-radiation imaging

Sponsors & Collaborators

• InSightec: lead

Study Sites (1)

Sunnybrook Research Institute

Toronto, Ontario, M4N 3M5, Canada

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 7, 2022
• First Posted: November 14, 2022
• Study Start: January 4, 2023
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): October 1, 2026
• Last Updated: April 21, 2026

Record last verified: 2026-04

Locations

CA (1)