Brain TumorPhase 1/2NCT05630209
Blood Brain Barrier (BBB) Disruption Using Exablate Focused Ultrasound With Doxorubicin for Treatment of Pediatric Diffuse Intrinsic Pontine Gliomas (DIPG)
A Phase 1/2 study testing Exablate and Doxorubicin in people with Brain Tumor, run by InSightec at 3 sites in United States.
Trial 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 Read more about this study
- Who is it for?
- Ages 5 to 21 years
- Conditions the study is for
- Brain Tumor
- What is being tested?
- Exablate and Doxorubicin
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 3 sitesUnited States
- 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 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Show all 9Show fewer
- 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
Who cannot join
- 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
Show all 20Show fewer
- 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
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Cook Children's Health Care System
Fort Worth, Texas, 76104
Nicklaus Children's Hospital
Miami, Florida, 33155
Children's National Medical Center
Washington D.C., District of Columbia, 20010
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 Type2.0/2.1 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 up to three sites in the United States. 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 Ultrasound, 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 group | Role | What they receive |
|---|---|---|
| Blood Brain Barrier Disruption (BBBD) | Experimental | Exablate 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.
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)
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.
- First posted29 Nov 2022
- Study start4 Jan 2023
- Primary completionJun 2026 (estimated)
- Study completionOct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Brain TumorStudies of the same condition, open and under way.
- More trials run by InSightecThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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)
Sponsor and investigators
- Lead sponsor
- InSightec
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05630209
- Sponsor's protocol ID
- BT016
- Status last verified
- Apr 2026
- First submitted
- 18 Nov 2022
- Last updated
- 17 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 17 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT05630209
- Acronym / sponsor ID
- BT016
- Conditions
- Brain Tumor
- Keywords
- DIPG, Focused Ultrasound, Chemotherapy, Phase I trial, Pediatrics
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- InSightecIndustry (pharma, biotech, devices)
- Study start
- 4 Jan 2023 (actual)
- Primary completion
- Jun 2026 (estimated)
- Study completion
- Oct 2026 (estimated)
- First posted
- 29 Nov 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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 up to three sites in the United States. 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.
Study design
- Primary purpose
- Device feasibility
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Blood Brain Barrier Disruption (BBBD) | Experimental | Device: Exablate, Drug: DoxorubicinExablate MR Guided Focused Ultrasound for Blood Brain Barrier Disruption with Doxorubicin for treating pediatric patients with DIPG |
Who may qualify
- Ages
- 5 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (20 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ExablateAlso: Exablate BBBDBlood Brain Barrier Disruption (BBBD) via Exablate Type 2 system with microbubble resonators on the day of Doxorubicin infusion to treat DIPG brain tumors | Device | Blood Brain Barrier Disruption (BBBD) |
| DoxorubicinAlso: Adriamycin, RubexDoxorubicin infusion | Drug | Blood Brain Barrier Disruption (BBBD) |
Primary outcome
Adverse Events
Time frame: 2 years
All adverse events from first treatment to end of study will be documented and reported according to the CTCAE terminology and severity scale
1 secondary outcome
Blood Brain Barrier Disruption (BBBD)
Time frame: immediately post BBBD sonication
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Apr 2026.
Related trials for Brain Tumor
Not yet recruitingPhase 2
Molecularly Stratified Trial for Newly Diagnosed Infant-Type Hemispheric Glioma
Infant-Type Hemispheric Glioma
RecruitingObservational study
cfDNA Assay Prospective Observational Validation for Early Cancer Detection and Minimal Residual Disease
Brain Cancer, Breast Cancer
RecruitingPhase 1/2
PLX038 in Primary Central Nervous System Tumors Containing MYC or MYCN Amplifications
Glioma, Medulloblastoma
Active, not recruitingPhase 1/2
A Phase 1b / 2 Drug Resistant Immunotherapy With Activated, Gene Modified Allogeneic or Autologous γδ T Cells (DeltEx) in Combination With Maintenance Temozolomide in Subjects With Recurrent or Newly Diagnosed Glioblastoma
Glioblastoma
RecruitingPhase 1
PEACH TRIAL- Precision Medicine and Adoptive Cellular Therapy
Neuroblastoma, Diffuse Intrinsic Pontine Glioma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 1 country: see every site and its status.
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 3 sites in United States.
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 9 inclusion and 20 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 17 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 3 sites in United States. 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?
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 17 Apr 2026.