Benign Centrally-Located Intracranial TumorsNCT03028246
A Feasibility Safety Study of Benign Centrally-Located Intracranial Tumors in Pediatric and Young Adult Subjects
A study testing ExAblate 4000 System in people with Benign Centrally-Located Intracranial Tumors, run by InSightec at 3 sites in United States.
Trial summary
The goal of this prospective, non-randomized, single-arm, feasibility study is to develop data to evaluate the safety and feasibility of ExAblate 4000 treatment of benign intracranial tumors which require clinical intervention in pediatric and young adult subjects. Read more about this study
- Who is it for?
- Ages 5 to 22 years
- Conditions the study is for
- Benign Centrally-Located Intracranial Tumors
- What is being tested?
- ExAblate 4000 System
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 3 sitesUnited States
- Study dates
- 28 Feb 2017to 31 Dec 2026
- Phase
- Interventional
- 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 22 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subjects with benign (WHO grade I) centrally located intracranial tumors which require clinical intervention and are known to carry minimal hemorrhage risk
- Minimum head circumference will be 49cm
- Skull Density Ratio (SDR) should be ≥0.35
- Subjects should be on a stable dose of all condition-related medications for 30 days prior to study entry as determine by medical records
- Subjects and/or parent(s)/legal representative can provide accurate seizure diary log for the 30 days prior to FUS treatment and for the duration of the study
Who cannot join
- Subjects with unstable cardiac status that would increase anesthetic risk including
- Subjects exhibiting any behavior(s) consistent with ethanol or substance abuse as defined by the criteria outlined in the DSM-IV
- Subjects who are taking human growth hormone (hGH), also known as somatotropin
- Subjects with standard contraindications for MR imaging such as non-MRI compatible implanted metallic devices including vagus nerve stimulator, responsive neurostimulator, cardiac pacemakers, non-metallic shunts, size limitations, etc.
- Known intolerance or allergies to the MRI contrast agent (e.g. Gadolinium or Magnevist) or sedative medications
- Severely impaired renal function (estimated glomerular filtration rate <70% of normal GFR for age) or receiving dialysis
Show all 19Show fewer
- Any history of clinically significant abnormal bleeding and/or coagulopathy
- Receiving anticoagulant (e.g. warfarin) or antiplatelet (e.g. aspirin, NSAIDs) therapy within one week of focused ultrasound procedure or drugs known to increase risk or hemorrhage (e.g. Avastin) within one month of focused ultrasound procedure; or, unable or unwilling to stop anticoagulant for the purpose of focused ultrasound procedure
- Use of valproate derivatives for seizure control within the preceding 2 weeks
- Known or suspected acute, active, or uncontrolled infection
- History of postnatal stroke or intracranial hemorrhage within 6 months
- Clinical suspicion of increased intracranial pressure (as evidenced by symptoms of obstruction: headache, nausea, vomiting, lethargy, and papilledema)
- Have participated in another interventional trial in the last 30 days
- History of immunocompromise, including subject who is HIV positive with incomplete viral suppression
- Known life-threatening systemic disease
- Subjects with suicidal ideation or previous suicide attempt within the past year
- Subjects with malignant brain tumors, or the presence of any ambiguous clinical features that could imply a malignant potential to the tumor, or for which a biopsy is necessary
- Subjects for whom histopathology is important for ongoing management
- Female subjects who are pregnant, breast feeding or planning to become pregnant during the study or are unwilling to practice birth control during participation in the study, if of child-bearing age
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
Texas Children's Hospital
Houston, Texas, 77030
Sarah Martinez, 832-822-4075, sxwisor@texaschildrens.org
Daniel Curry, MD
- Recruiting
Nicklaus Children's Hospital
Miami, Florida, 33155
The Focused Ultrasound Team, 305-662-8386, mrguidedfus@Nicklaushealth.org
John Ragheb, MD, 305-662-8386, john.ragheb2@nicklaushealth.org
- Recruiting
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Sonora Robles, 202-476-3755, srobles@childrensnational.org
Robert Keating
About this study
Official title: A Feasibility Safety Study Using the ExAblate 4000 System in the Management of Benign Centrally-Located Intracranial Tumors Which Require Clinical Intervention in Pediatric and Young Adult Subjects
The goal of this prospective, non-randomized, single-arm, feasibility study is to develop data to evaluate the safety and feasibility of ExAblate 4000 treatment of benign intracranial tumors which require clinical intervention in pediatric and young adult subjects.
Indication of Use: Ablation of benign intracranial tumors in children and young adults which are ExAblate accessible.
Read the detailed description
The goal of this prospective, non-randomized, single-arm, feasibility study is to develop data to evaluate the safety and feasibility of ExAblate 4000 treatment of benign intracranial tumors which require clinical intervention in pediatric and young adult subjects.
Indication of Use: Ablation of benign intracranial tumors in children and young adults which are ExAblate accessible.
This is a feasibility study intended to collect data for use in the development of future studies. As such, no formal statistical hypothesis or hypothesis testing is proposed. The purpose of this study is to evaluate the safety and feasibility of managing benign brain tumor size using ExAblate 4000 treatment of progressing benign intracranial tumors which require clinical intervention in pediatric and young adult subjects.
- Being tested
- ExAblate 4000 System
- Keywords
- Hamartoma, Hypothalamic Hamartoma
- Medical subject headings
- Hamartoma, Hypothalamic hamartomas
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ExAblate 4000 SystemDevice
Also called MRgFUS, HIFU, Focused Ultrasound, MR-Guided Focused Ultrasound
Focused ultrasound
| Study group | Role | What they receive |
|---|---|---|
| ExAblate 4000 System | Experimental | MR-Guided Focused Ultrasound Receives: Device: ExAblate 4000 System |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events Safety and Tolerability
Safety will be assessed by tabulation of treatment related adverse events. All adverse events at least possibly procedure related will also be examined in detail for patterns of occurrence.
Measured: Post ExAblate Procedure through 12 Month Follow-up
Measurement of Tumor Volume
ExAblate procedure will demonstrate feasibility along with measurement of tumor volumes at follow-up as compared to the baseline MRI.
Measured: Baseline through 12 Month Follow Up
Secondary outcomes (5)
Changes in the General Physical Exam
Any changes in physical examination performed by a physician.
Measured: Baseline through 12 Month Follow Up
Changes in the Neurological Exam
Any changes in neurological examination performed by the neurologist/neurosurgeon.
Measured: Baseline through 12 Month Follow Up
Confrontational Visual Field Testing
Changes in confrontational visual field testing performed by a physician
Measured: Baseline through 3 Month Follow Up
Global Impression of Change-Clinician
Impression of change as the result of the treatment by a physician
Measured: Day 1 through 12 Month Follow Up
Patient Global Impression of Change
Impression of change as the result of the treatment by the subject
Measured: Day 1 through 12 Month Follow Up
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jan 2017 (estimated)
- Study start28 Feb 2017
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Benign Centrally-Located Intracranial TumorsStudies 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- InSightec
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- John Ragheb, MD, Principal investigator, Miami Children's Research Institute - Nicklaus Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03028246
- Sponsor's protocol ID
- BT005
- Status last verified
- May 2026
- First submitted
- 16 Jan 2017
- Last updated
- 29 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 29 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Feasibility Safety Study Using the ExAblate 4000 System in the Management of Benign Centrally-Located Intracranial Tumors Which Require Clinical Intervention in Pediatric and Young Adult Subjects
- ClinicalTrials.gov ID
- NCT03028246
- Acronym / sponsor ID
- BT005
- Keywords
- Hamartoma, Hypothalamic Hamartoma
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- InSightecIndustry (pharma, biotech, devices)
- Study start
- 28 Feb 2017 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 23 Jan 2017 (estimated)
- 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 goal of this prospective, non-randomized, single-arm, feasibility study is to develop data to evaluate the safety and feasibility of ExAblate 4000 treatment of benign intracranial tumors which require clinical intervention in pediatric and young adult subjects.
Indication of Use: Ablation of benign intracranial tumors in children and young adults which are ExAblate accessible.
Detailed description
The goal of this prospective, non-randomized, single-arm, feasibility study is to develop data to evaluate the safety and feasibility of ExAblate 4000 treatment of benign intracranial tumors which require clinical intervention in pediatric and young adult subjects.
Indication of Use: Ablation of benign intracranial tumors in children and young adults which are ExAblate accessible.
This is a feasibility study intended to collect data for use in the development of future studies. As such, no formal statistical hypothesis or hypothesis testing is proposed. The purpose of this study is to evaluate the safety and feasibility of managing benign brain tumor size using ExAblate 4000 treatment of progressing benign intracranial tumors which require clinical intervention in pediatric and young adult subjects.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ExAblate 4000 System | Experimental | Device: ExAblate 4000 SystemMR-Guided Focused Ultrasound |
Who may qualify
- Ages
- 5 to 22 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Subjects with benign (WHO grade I) centrally located intracranial tumors which require clinical intervention and are known to carry minimal hemorrhage risk
- Minimum head circumference will be 49cm
- Skull Density Ratio (SDR) should be ≥0.35
- Subjects should be on a stable dose of all condition-related medications for 30 days prior to study entry as determine by medical records
- Subjects and/or parent(s)/legal representative can provide accurate seizure diary log for the 30 days prior to FUS treatment and for the duration of the study
Key exclusion criteria (19 listed)
- Subjects with unstable cardiac status that would increase anesthetic risk including
- Subjects exhibiting any behavior(s) consistent with ethanol or substance abuse as defined by the criteria outlined in the DSM-IV
- Subjects who are taking human growth hormone (hGH), also known as somatotropin
- Subjects with standard contraindications for MR imaging such as non-MRI compatible implanted metallic devices including vagus nerve stimulator, responsive neurostimulator, cardiac pacemakers, non-metallic shunts, size limitations, etc.
- Known intolerance or allergies to the MRI contrast agent (e.g. Gadolinium or Magnevist) or sedative medications
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 |
|---|---|---|
| ExAblate 4000 SystemAlso: MRgFUS, HIFU, Focused Ultrasound, MR-Guided Focused UltrasoundFocused ultrasound | Device | ExAblate 4000 System |
Primary outcome
Incidence of Treatment-Emergent Adverse Events Safety and Tolerability
Time frame: Post ExAblate Procedure through 12 Month Follow-up
Safety will be assessed by tabulation of treatment related adverse events. All adverse events at least possibly procedure related will also be examined in detail for patterns of occurrence.
Measurement of Tumor Volume
Time frame: Baseline through 12 Month Follow Up
ExAblate procedure will demonstrate feasibility along with measurement of tumor volumes at follow-up as compared to the baseline MRI.
5 secondary outcomes
Changes in the General Physical Exam
Time frame: Baseline through 12 Month Follow Up
Changes in the Neurological Exam
Time frame: Baseline through 12 Month Follow Up
Confrontational Visual Field Testing
Time frame: Baseline through 3 Month Follow Up
Global Impression of Change-Clinician
Time frame: Day 1 through 12 Month Follow Up
Patient Global Impression of Change
Time frame: Day 1 through 12 Month Follow Up
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 29 Jun 2026.
- Individual participant dataSharing planned: Undecided
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.
Children's National Medical Center
Washington D.C., United States
John Ragheb, MD principal investigator
Miami Children's Research Institute - Nicklaus Children's Hospital
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing ExAblate 4000 System in people with Benign Centrally-Located Intracranial Tumors, 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 5 inclusion and 19 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: looking for participants now, as of the sponsor's last update on 29 Jun 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.
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?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events Safety and Tolerability (time frame: Post ExAblate Procedure through 12 Month Follow-up). The study lists 2 primary outcomes in all. 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 29 Jun 2026.