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

NCT03028246 | Recruiting DMC FDA Device

• 1 other identifier: BT005
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 3

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.

Conditions

Benign Centrally-Located Intracranial Tumors

Keywords

Hamartoma; Hypothalamic Hamartoma

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (5)

• Changes in the General Physical Exam

  Baseline through 12 Month Follow Up

• Changes in the Neurological Exam

  Baseline through 12 Month Follow Up

• Confrontational Visual Field Testing

  Baseline through 3 Month Follow Up

• Global Impression of Change-Clinician

  Day 1 through 12 Month Follow Up

• Patient Global Impression of Change

  Day 1 through 12 Month Follow Up

Other Outcomes (2)

• Change in Seizure Frequency

  From baseline through 12 months after treatment

• QOLCE-55

  From baseline through 12 months after treatment

Study Arms (1)

ExAblate 4000 System

EXPERIMENTAL

MR-Guided Focused Ultrasound

Device: ExAblate 4000 System

Interventions

ExAblate 4000 System DEVICE

Focused ultrasound

Also known as: MRgFUS, HIFU, Focused Ultrasound, MR-Guided Focused Ultrasound, ExAblate Neuro System

ExAblate 4000 System

Eligibility Criteria

• Age: 5 Years - 22 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• InSightec: lead

Study Sites (3)

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

RECRUITING

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

RECRUITING

Texas Children's Hospital

Houston, Texas, 77030, United States

RECRUITING

MeSH Terms

Conditions

Hamartoma; Hypothalamic hamartomas

Condition Hierarchy (Ancestors)

Neoplasms

Study Officials

• John Ragheb, MD

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

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 16, 2017
• First Posted: January 23, 2017
• Study Start: February 28, 2017
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: June 29, 2026

Record last verified: 2026-05

Locations

US (3)