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

NCT ID: NCT03028246

Last Updated: 2025-10-03

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: NA
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-02-28
• Study Completion Date: 2026-12-31

Brief 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.

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.

Conditions

Benign Centrally-Located Intracranial Tumors

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

ExAblate 4000 System

MR-Guided Focused Ultrasound

Group Type: EXPERIMENTAL

ExAblate 4000 System

Intervention Type: DEVICE

Focused ultrasound

Interventions

ExAblate 4000 System

Focused ultrasound

Intervention Type: DEVICE

Other Intervention Names

MRgFUS; HIFU; Focused Ultrasound; MR-Guided Focused Ultrasound; ExAblate Neuro System

Eligibility Criteria

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

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 22 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

InSightec

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

John Ragheb, MD

Role: PRINCIPAL_INVESTIGATOR

Miami Children's Research Institute - Nicklaus Children's Hospital

Locations

Children's National Medical Center

Washington D.C., District of Columbia, United States

Site Status: RECRUITING

Nicklaus Children's Hospital

Miami, Florida, United States

Site Status: RECRUITING

Countries

United States

Facility Contacts

Elizabeth Paronett

Role: primary

eparonett@childrensnational.org

202-476-5551

The Focused Ultrasound Team

Role: primary

mrguidedfus@Nicklaushealth.org

305-662-8386

John Ragheb, MD

Role: backup

john.ragheb2@nicklaushealth.org

305-662-8386

Other Identifiers

BT005

Identifier Type: -

Identifier Source: org_study_id