<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT03028246</url>
  </required_header>
  <id_info>
    <org_study_id>BT005</org_study_id>
    <nct_id>NCT03028246</nct_id>
  </id_info>
  <brief_title>A Feasibility Safety Study of Benign Centrally-Located Intracranial Tumors in Pediatric and Young Adult Subjects</brief_title>
  <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</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>InSightec</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>InSightec</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>Yes</is_fda_regulated_device>
    <is_unapproved_device>Yes</is_unapproved_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The goal of this prospective, non-randomized, single-arm, feasibility study is to develop&#xD;
      data to evaluate the safety and feasibility of ExAblate 4000 treatment of benign intracranial&#xD;
      tumors which require clinical intervention in pediatric and young adult subjects.&#xD;
&#xD;
      Indication of Use: Ablation of benign intracranial tumors in children and young adults which&#xD;
      are ExAblate accessible.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The goal of this prospective, non-randomized, single-arm, feasibility study is to develop&#xD;
      data to evaluate the safety and feasibility of ExAblate 4000 treatment of benign intracranial&#xD;
      tumors which require clinical intervention in pediatric and young adult subjects.&#xD;
&#xD;
      Indication of Use: Ablation of benign intracranial tumors in children and young adults which&#xD;
      are ExAblate accessible.&#xD;
&#xD;
      This is a feasibility study intended to collect data for use in the development of future&#xD;
      studies. As such, no formal statistical hypothesis or hypothesis testing is proposed. The&#xD;
      purpose of this study is to evaluate the safety and feasibility of managing benign brain&#xD;
      tumor size using ExAblate 4000 treatment of progressing benign intracranial tumors which&#xD;
      require clinical intervention in pediatric and young adult subjects.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">February 28, 2017</start_date>
  <completion_date type="Anticipated">December 31, 2023</completion_date>
  <primary_completion_date type="Anticipated">December 31, 2022</primary_completion_date>
  <phase>N/A</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Incidence of Treatment-Emergent Adverse Events Safety and Tolerability</measure>
    <time_frame>Post ExAblate Procedure through 12 Month Follow-up</time_frame>
    <description>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.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Measurement of Tumor Volume</measure>
    <time_frame>Baseline through 12 Month Follow Up</time_frame>
    <description>ExAblate procedure will demonstrate feasibility along with measurement of tumor volumes at follow-up as compared to the baseline MRI.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Changes in the General Physical Exam</measure>
    <time_frame>Baseline through 12 Month Follow Up</time_frame>
    <description>Any changes in physical examination performed by a physician.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Changes in the Neurological Exam</measure>
    <time_frame>Baseline through 12 Month Follow Up</time_frame>
    <description>Any changes in neurological examination performed by the neurologist/neurosurgeon.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Confrontational Visual Field Testing</measure>
    <time_frame>Baseline through 3 Month Follow Up</time_frame>
    <description>Changes in confrontational visual field testing performed by a physician</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Global Impression of Change-Clinician</measure>
    <time_frame>Day 1 through 12 Month Follow Up</time_frame>
    <description>Impression of change as the result of the treatment by a physician</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Patient Global Impression of Change</measure>
    <time_frame>Day 1 through 12 Month Follow Up</time_frame>
    <description>Impression of change as the result of the treatment by the subject</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">10</enrollment>
  <condition>Benign Centrally-Located Intracranial Tumors</condition>
  <arm_group>
    <arm_group_label>ExAblate 4000 System</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>MR-Guided Focused Ultrasound</description>
  </arm_group>
  <intervention>
    <intervention_type>Device</intervention_type>
    <intervention_name>ExAblate 4000 System</intervention_name>
    <description>Focused ultrasound</description>
    <arm_group_label>ExAblate 4000 System</arm_group_label>
    <other_name>MRgFUS</other_name>
    <other_name>HIFU</other_name>
    <other_name>Focused Ultrasound</other_name>
    <other_name>MR-Guided Focused Ultrasound</other_name>
    <other_name>ExAblate Neuro System</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Subjects with benign (WHO grade I) centrally located intracranial tumors which require&#xD;
             clinical intervention and are known to carry minimal hemorrhage risk&#xD;
&#xD;
          -  Minimum head circumference will be 52cm&#xD;
&#xD;
          -  Subjects should be on a stable dose of all condition-related medications for 30 days&#xD;
             prior to study entry as determine by medical records&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Subjects who are taking human growth hormone (hGH), also known as somatotropin&#xD;
&#xD;
          -  Subjects with standard contraindications for MR imaging such as non-MRI compatible&#xD;
             implanted metallic devices including vagus nerve stimulator, responsive&#xD;
             neurostimulator, cardiac pacemakers, non-metallic shunts, size limitations, etc.&#xD;
&#xD;
          -  Subjects with suicidal ideation or previous suicide attempt within the past year&#xD;
&#xD;
          -  Subjects with malignant brain tumors, or the presence of any ambiguous clinical&#xD;
             features that could imply a malignant potential to the tumor, or for which a biopsy is&#xD;
             necessary&#xD;
&#xD;
          -  Subjects for whom histopathology is important for ongoing management&#xD;
&#xD;
          -  Subjects who are unwilling or unable to undergo general anesthesia&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>8 Years</minimum_age>
    <maximum_age>22 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>John Ragheb, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Miami Children's Research Institute - Nicklaus Children's Hospital</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Children's National Medical Center</name>
      <address>
        <city>Washington</city>
        <state>District of Columbia</state>
        <zip>20010</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Sophie Kopec</last_name>
      <phone>202-476-5016</phone>
      <email>skopec@childrensnational.org</email>
    </contact>
    <investigator>
      <last_name>Robert Keating</last_name>
      <role>Principal Investigator</role>
    </investigator>
    <investigator>
      <last_name>Roger Packer</last_name>
      <role>Sub-Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Nicklaus Children's Hospital Insititute</name>
      <address>
        <city>Miami</city>
        <state>Florida</state>
        <zip>33155</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>The Focused Ultrasound Team</last_name>
      <phone>305-662-8386</phone>
      <email>mrguidedfus@Nicklaushealth.org</email>
    </contact>
    <contact_backup>
      <last_name>John Ragheb, MD</last_name>
      <phone>305-662-8386</phone>
      <email>john.ragheb2@nicklaushealth.org</email>
    </contact_backup>
    <investigator>
      <last_name>John Ragheb, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
    <investigator>
      <last_name>Ian Miller, MD</last_name>
      <role>Sub-Investigator</role>
    </investigator>
    <investigator>
      <last_name>Luis Mendoza, MD</last_name>
      <role>Sub-Investigator</role>
    </investigator>
    <investigator>
      <last_name>Toba Niazi, MD</last_name>
      <role>Sub-Investigator</role>
    </investigator>
    <investigator>
      <last_name>Sanjiv Bhatia, MD</last_name>
      <role>Sub-Investigator</role>
    </investigator>
    <investigator>
      <last_name>Pat Dean, ARNP</last_name>
      <role>Sub-Investigator</role>
    </investigator>
    <investigator>
      <last_name>Ziad Khatib, MD</last_name>
      <role>Sub-Investigator</role>
    </investigator>
    <investigator>
      <last_name>Ann Hsylop, MD</last_name>
      <role>Sub-Investigator</role>
    </investigator>
    <investigator>
      <last_name>Nolan Altman, MD</last_name>
      <role>Sub-Investigator</role>
    </investigator>
    <investigator>
      <last_name>Christian Bauer, MD</last_name>
      <role>Sub-Investigator</role>
    </investigator>
    <investigator>
      <last_name>Frank Wang, MD</last_name>
      <role>Sub-Investigator</role>
    </investigator>
    <investigator>
      <last_name>Paquine Sadeghi, MD</last_name>
      <role>Sub-Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>August 2022</verification_date>
  <study_first_submitted>January 16, 2017</study_first_submitted>
  <study_first_submitted_qc>January 18, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">January 23, 2017</study_first_posted>
  <last_update_submitted>August 16, 2022</last_update_submitted>
  <last_update_submitted_qc>August 16, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">August 17, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Hamartoma</keyword>
  <keyword>Hypothalamic Hamartoma</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Brain Neoplasms</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Undecided</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT03028246
org study id: BT005
nct id: NCT03028246
lead sponsor: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: Yes
is unapproved device: Yes
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.
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.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Incidence of Treatment-Emergent Adverse Events Safety and Tolerability
time frame: Post ExAblate Procedure through 12 Month Follow-up
description: 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.
measure: Measurement of Tumor Volume
time frame: Baseline through 12 Month Follow Up
description: ExAblate procedure will demonstrate feasibility along with measurement of tumor volumes at follow-up as compared to the baseline MRI.
measure: Changes in the General Physical Exam
time frame: Baseline through 12 Month Follow Up
description: Any changes in physical examination performed by a physician.
measure: Changes in the Neurological Exam
time frame: Baseline through 12 Month Follow Up
description: Any changes in neurological examination performed by the neurologist/neurosurgeon.
measure: Confrontational Visual Field Testing
time frame: Baseline through 3 Month Follow Up
description: Changes in confrontational visual field testing performed by a physician
measure: Global Impression of Change-Clinician
time frame: Day 1 through 12 Month Follow Up
description: Impression of change as the result of the treatment by a physician
measure: Patient Global Impression of Change
time frame: Day 1 through 12 Month Follow Up
description: Impression of change as the result of the treatment by the subject
arm group label: ExAblate 4000 System
arm group type: Experimental
description: MR-Guided Focused Ultrasound
intervention type: Device
intervention name: ExAblate 4000 System
description: Focused ultrasound
arm group label: ExAblate 4000 System
other name: MRgFUS
other name: HIFU
other name: Focused Ultrasound
other name: MR-Guided Focused Ultrasound
other name: ExAblate Neuro System
criteria: 
gender: All
minimum age: 8 Years
maximum age: 22 Years
healthy volunteers: No
last name: John Ragheb, MD
role: Principal Investigator
affiliation: Miami Children's Research Institute - Nicklaus Children's Hospital
facility: 
status: Recruiting
contact: 
investigator: 
investigator: 
facility: 
status: Recruiting
contact: 
contact backup: 
investigator: 
investigator: 
investigator: 
investigator: 
investigator: 
investigator: 
investigator: 
investigator: 
investigator: 
investigator: 
investigator: 
investigator: 
country: United States
responsible party type: Sponsor
mesh term: Brain Neoplasms
sharing ipd: Undecided