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

• ClinicalTrials.gov Identifier: NCT03028246
• Recruitment Status: Recruiting
• First Posted: January 23, 2017
• Last Update Posted: February 4, 2021
• Sponsor: InSightec
• Information provided by (Responsible Party): InSightec

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Benign Centrally-Located Intracranial Tumors	Device: ExAblate 4000 System	Not Applicable

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

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 10 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: February 28, 2017
• Estimated Primary Completion Date: December 31, 2022
• Estimated Study Completion Date: December 31, 2023

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

1. 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.
2. 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.

Secondary Outcome Measures :

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

Eligibility Criteria

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

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 52cm
• Subjects should be on a stable dose of all condition-related medications for 30 days prior to study entry as determine by medical records

Exclusion Criteria:

• 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.
• 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
• Subjects who are unwilling or unable to undergo general anesthesia

Contacts and Locations

Locations

United States, Florida

Nicklaus Children's Hospital Insititute; Recruiting

Miami, Florida, United States, 33155

Contact: John Ragheb, MD 305-662-8386 john.ragheb2@nicklaushealth.org

Contact: Leisa Tamayo 786-502-6122 leisa.tamayonenninger@nicklaushealth.org

Principal Investigator: John Ragheb, MD

Sub-Investigator: Shelly Wang, MD

Sub-Investigator: Marytery Fajardo, MD

Sponsors and Collaborators

InSightec

Investigators

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

More Information

• Responsible Party: InSightec
• ClinicalTrials.gov Identifier: NCT03028246
• Other Study ID Numbers: BT005
• First Posted: January 23, 2017
• Last Update Posted: February 4, 2021
• Last Verified: February 2021

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Undecided

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: Yes
• Device Product Not Approved or Cleared by U.S. FDA: Yes

Keywords provided by InSightec:

Hamartoma; Hypothalamic Hamartoma

Additional relevant MeSH terms:

Brain Neoplasms; Central Nervous System Neoplasms; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases