A Feasibility Safety Study of Benign Centrally-Located Intracranial Tumors in Pediatric and Young Adult Subjects

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.

Study Overview

• Status: Recruiting
• Conditions: Benign Centrally-Located Intracranial Tumors
• Intervention / Treatment: Device: ExAblate 4000 System

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 Type: Interventional
• Enrollment (Estimated): 20
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • District of Columbia
    • Washington D.C., District of Columbia, United States, 20010
      • Recruiting
      • Children's National Medical Center
      • Contact: Elizabeth Paronett; Phone Number: 202-476-5551; Email: eparonett@childrensnational.org
      • Principal Investigator: Robert Keating
      • Sub-Investigator: Roger Packer
  • Florida
    • Miami, Florida, United States, 33155
      • Recruiting
      • Nicklaus Children's Hospital
      • Contact: The Focused Ultrasound Team; Phone Number: 305-662-8386; Email: mrguidedfus@Nicklaushealth.org
      • Contact: John Ragheb, MD; Phone Number: 305-662-8386; Email: john.ragheb2@nicklaushealth.org
      • Principal Investigator: John Ragheb, MD
      • Sub-Investigator: Pat Dean, ARNP
      • Sub-Investigator: Marytery Fajardo, MD
      • Sub-Investigator: Matt Lallas, MD
  • Texas
    • Houston, Texas, United States, 77030
      • Recruiting
      • Texas Children's Hospital
      • Contact: Sarah Martinez; Phone Number: 832-822-4075; Email: sxwisor@texaschildrens.org
      • Principal Investigator: Daniel Curry, MD
      • Sub-Investigator: Irfan Ali, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 8 years to 22 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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
• 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ExAblate 4000 System; MR-Guided Focused Ultrasound	Device: ExAblate 4000 System; Focused ultrasound; Other Names: HIFU; Focused Ultrasound; MRgFUS; MR-Guided Focused Ultrasound; ExAblate Neuro System

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	Baseline through 12 Month Follow Up

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in the General Physical Exam	Any changes in physical examination performed by a physician.	Baseline through 12 Month Follow Up
Changes in the Neurological Exam	Any changes in neurological examination performed by the neurologist/neurosurgeon.	Baseline through 12 Month Follow Up
Confrontational Visual Field Testing	Changes in confrontational visual field testing performed by a physician	Baseline through 3 Month Follow Up
Global Impression of Change-Clinician	Impression of change as the result of the treatment by a physician	Day 1 through 12 Month Follow Up
Patient Global Impression of Change	Impression of change as the result of the treatment by the subject	Day 1 through 12 Month Follow Up

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Seizure Frequency	The change in seizure frequency will be evaluated from baseline through 12 months after treatment.	From baseline through 12 months after treatment
QOLCE-55	The change in scores on the QOLCE-55 questionnaire will be evaluated.	From baseline through 12 months after treatment

Collaborators and Investigators

• Sponsor: InSightec
• Investigators: Principal Investigator: John Ragheb, MD, Miami Children's Research Institute - Nicklaus Children's Hospital

Study record dates

Study Major Dates

• Study Start (Actual): February 28, 2017
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026

Study Registration Dates

• First Submitted: January 16, 2017
• First Submitted That Met QC Criteria: January 18, 2017
• First Posted (Estimated): January 23, 2017

Study Record Updates

• Last Update Posted (Actual): May 8, 2026
• Last Update Submitted That Met QC Criteria: May 6, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Hypothalamic Hamartoma; Hamartoma
• Additional Relevant MeSH Terms: Neoplasms; Hamartoma; Hypothalamic hamartomas
• Other Study ID Numbers: BT005

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: Yes
• product manufactured in and exported from the U.S.: No