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Brain TumorPhase 1/2NCT05615623

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

A Phase 1/2 study testing Exablate and Doxorubicin in people with Brain Tumor, run by InSightec at 1 site in Canada.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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 Read more about this study

Who is it for?
Ages 5 to 18 years
Conditions the study is for
Brain Tumor
What is being tested?
Exablate and Doxorubicin
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteCanada
Study dates
4 Jan 2023to Oct 2026
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 5 to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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

Who cannot join

  • 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)
Show all 14Show fewer
  • 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

Trial locations and contacts

1 site in 1 country.

Canada1 site

  • Sunnybrook Research Institute

    Toronto, Ontario, M4N 3M5

Other trials in Canada

About this study

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)

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

Read the detailed description

This is a prospective, single arm, non-randomized feasibility study to evaluate the safety, feasibility and preliminary efficacy of Blood Brain Barrier Disruption (BBBD) using the Exablate Type 2 system in pediatric patients with Diffuse Intrinsic Pontine Gliomas (DIPG) undergoing Doxorubicin chemotherapy. The study will be conducted at a single center in Canada. Patients will undergo 3 treatment cycles, approximately 4 -6 weeks apart. The study aims to establish feasibility and safety of Exablate BBBD in conjunction with Doxorubicin in the treatment of pediatric DIPG and assess preliminary efficacy in this patient population.

Conditions
Brain Tumor
Being tested
Exablate, Doxorubicin
Keywords
DIPG, Focused Utrasound, Chemotherapy, Phase I trial, Pediatrics
Medical subject headings
Brain Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ExablateDevice

    Also called Exablate BBBD

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

  • DoxorubicinDrug

    Also called Adriamycin, Rubex

    Doxorubicin infusion

Study groups in NCT05615623
Study groupRoleWhat they receive
Blood Brain Barrier Disruption (BBBD)ExperimentalExablate MR Guided Focused Ultrasound for Blood Brain Barrier Disruption with Doxorubicin for treating pediatric patients with DIPG
Receives: Device: Exablate; Drug: Doxorubicin

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

    Measured: 2 years

Secondary outcomes (1)

  1. Blood Brain Barrier Disruption (BBBD)

    BBBD will be assessed as a comparative ratio measured in pre and post-sonication contrast-enhanced MR images

    Measured: Immediately post BBBD sonication

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Nov 2022
  2. Study start4 Jan 2023
  3. Primary completionJun 2026 (estimated)
  4. Study completionOct 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Device feasibility
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05615623
Sponsor's protocol ID
BT016C
Status last verified
Apr 2026
First submitted
7 Nov 2022
Last updated
21 Apr 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Exablate and Doxorubicin in people with Brain Tumor, run by InSightec at 1 site in Canada.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 21 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Adverse Events (time frame: 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05615623.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Apr 2026.

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