xml: <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/NCT06036199</url>
    </required_header>
    <id_info>
      <org_study_id>G230044</org_study_id>
      <nct_id>NCT06036199</nct_id>
    </id_info>
    <brief_title>Focused Ultrasound Pallidotomy for Dyskinetic Cerebral Palsy in Pediatric and Young Adult Subjects</brief_title>
    <official_title>Safety and Impact on Quality of Life of Focused Ultrasound Pallidotomy for Dyskinetic Cerebral Palsy in Pediatric and Young Adult Subjects</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Children's National Research Institute</agency>
        <agency_class>Other</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Children's National Research Institute</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>
    </oversight_info>
    <brief_summary>
      <textblock>
        The primary objective of the proposed study is to evaluate the safety of ExAblate&#xD;
        Transcranial MRgFUS as a tool for creating bilateral or unilateral lesions in the globus&#xD;
        pallidus (GPi) in patients with treatment-refractory secondary dystonia due to dyskinetic&#xD;
        cerebral palsy&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        The primary objective of the proposed study is to evaluate the safety of ExAblate&#xD;
        Transcranial MRgFUS as a tool for creating bilateral or unilateral lesions in the globus&#xD;
        pallidus (GPi) in patients with treatment-refractory secondary dystonia due to dyskinetic&#xD;
        cerebral palsy.&#xD;
  &#xD;
        The secondary is to assess the impact on Quality of Life of Focused Ultrasound Bilateral and&#xD;
        unilateral Pallidotomy for Dyskinetic Cerebral Palsy in Pediatric and Young Adult Subjects.&#xD;
        In addition, the impact of bilateral pallidotomy on motor development, pain perception,&#xD;
        speech, memory, attention and cognition in these patients will also be assessed.&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">September 2023</start_date>
    <completion_date type="Anticipated">August 2025</completion_date>
    <primary_completion_date type="Anticipated">August 2025</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 and severity of device-related and procedure-related adverse events (AE)</measure>
      <time_frame>2 years</time_frame>
      <description>Safety will be determined by an evaluation of the incidence and severity of device-related and procedure-related adverse events (AE) from the first treatment day visit through the 24-month post-treatment time point. All AEs will be reported and categorized as related to the device versus the ablation procedure.</description>
    </primary_outcome>
    <secondary_outcome>
      <measure>Burke-Fahn-Marsden-Dystonia Rating Scale movement and disability</measure>
      <time_frame>2 years</time_frame>
      <description>Assessment of speech and swallowing. Score range: 0-30, 0 indicates complete independence, 30 indicates complete dependence.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Family Scale (FaBel)</measure>
      <time_frame>2 years</time_frame>
      <description>Assessment of the burden for caregivers. Score range: 0-84, 0 indicates no burden, 84 indicates very severe burden.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Canadian Occupational Performance Measure (COPM)</measure>
      <time_frame>2 years</time_frame>
      <description>Assessment of activities of daily living.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Gross Motor Function Measure (GMFM-66)</measure>
      <time_frame>2 years</time_frame>
      <description>Assessment of physical disability. Score range: 0-3, 0 indicates no performance, 3 indicates complete performance.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Gross Motor Function Classification System (GMFCS)</measure>
      <time_frame>2 years</time_frame>
      <description>Degree of physical impairment. Score range: 1-5, 1 indicates no limitations, 5 indicates severe limitations.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>SF-36 for assessment of quality of life</measure>
      <time_frame>2 years</time_frame>
      <description>Assessment of quality of life of subject. Score range: 0-100, 0 indicates bad health state, 100 indicates an excellent health state.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD)</measure>
      <time_frame>2 years</time_frame>
      <description>Assessment of Quality of life of Caregivers and Subject. Score range: 0-100, 0 indicates a bad score, 100 indicates an excellent score.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Frenchay Dysarthria Assessment</measure>
      <time_frame>2 years</time_frame>
      <description>Assessment of speech and swallowing. Score range:</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Assessment of Cognition</measure>
      <time_frame>2 years</time_frame>
      <description>Snijders-Oomen-Non-Verbal-Intelligence Test (SON-R)</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Strengths and Difficulties Questionnaire</measure>
      <time_frame>2 years</time_frame>
      <description>Assessment of mood and attention. Score range: 0-40, 0 indicates normal, 40 indicates abnormal.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Assessment of Attention</measure>
      <time_frame>2 years</time_frame>
      <description>Attentional Network Test (ANT)</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Assessment of cognition</measure>
      <time_frame>2 years</time_frame>
      <description>Non-Verbal-Learning Test (NVLT)</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Wong Baker Faces</measure>
      <time_frame>2 years</time_frame>
      <description>Assessment of pain. Score range; 0-10, 0 indicates no pain, 10 indicates worst pain.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Tardieu Scale</measure>
      <time_frame>2 years</time_frame>
      <description>Assessment of the severity of spasticity. Score range: 0-5, 0 indicates no resistance, 5 indicates no movement.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Dyskinesia Impairment Scale (DIS)</measure>
      <time_frame>2 years</time_frame>
      <description>Assessment of the severity of chorea and dystonia. Score range: 0-576, 0 indicates no dystonia, 576 indicates very severe dystonia.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Barry Albright Dystonia Scale</measure>
      <time_frame>2 years</time_frame>
      <description>Score range: 0-32, 0 indicates no dystonia, 32 indicates severe dystonia.</description>
    </secondary_outcome>
    <number_of_arms>1</number_of_arms>
    <enrollment type="Anticipated">10</enrollment>
    <condition>Cerebral Palsy, Dyskinetic</condition>
    <condition>Secondary Dystonia</condition>
    <arm_group>
      <arm_group_label>Focused Ultrasound Pallidotomy</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>Pediatric and young adult patients between ages of 8 and 22 years with pharmaco-resistant secondary dystonia due to dyskinetic cerebral palsy who have Focused Ultrasound Pallidotomy</description>
    </arm_group>
    <intervention>
      <intervention_type>Device</intervention_type>
      <intervention_name>Focused Ultrasound Pallidotomy</intervention_name>
      <description>Use of ExAblate Transcranial MR guided Focused Ultrasound as a tool for creating bilateral or unilateral lesions in the globus pallidus (GPi) in patients with treatment-refractory secondary dystonia due to dyskinetic cerebral palsy</description>
      <arm_group_label>Focused Ultrasound Pallidotomy</arm_group_label>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  The treating physician has chosen GPi-DBS or pallidotomy for the treatment of the&#xD;
               secondary dystonia caused by cerebral palsy in this patient&#xD;
  &#xD;
            -  Patient and/or legal representative, if the patient is underaged or not capable to&#xD;
               give consent himself, have chosen pallidotomy as treatment&#xD;
  &#xD;
            -  The consent to participate in the trial of the underaged patient, if he is capable to&#xD;
               understand the study requirements, is required&#xD;
  &#xD;
            -  Age at enrolment 8-22 years&#xD;
  &#xD;
            -  Diagnosis of secondary dystonia due to cerebral palsy caused by perinatal hypoxic&#xD;
               injury&#xD;
  &#xD;
            -  Anti-dystonic pharmacotherapy insufficient&#xD;
  &#xD;
            -  Stable anti-dystonic medication over the last 30 days&#xD;
  &#xD;
            -  Globus pallidus internus (pars posterior) and thalamus (motor part) intact on MRI (not&#xD;
               older than 2 years - if possible)&#xD;
  &#xD;
            -  No fixed severe skeletal deformations with loss of function, which need immediate&#xD;
               orthopaedic surgical intervention&#xD;
  &#xD;
            -  Sufficient compliance of the patient or the legal representative if the patient is&#xD;
               underaged or not capable to give consent himself to take part in the study&#xD;
  &#xD;
            -  Informed consent to take part in the study from patient and/or legal representative if&#xD;
               the patient is underaged or not capable to give consent himself&#xD;
  &#xD;
            -  Patient and/or legal representative if the patient is underaged or not capable to give&#xD;
               consent himself, understands the study requirements and the treatment procedures and&#xD;
               provides written informed consent before any study-specific tests or procedures are&#xD;
               performed&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Patients with known primary (e.g. DYT1) or idiopathic dystonia&#xD;
  &#xD;
            -  Severe axial hypotonia with total loss of head control (e.g. absence of control at&#xD;
               &quot;upper thoracic level&quot; in the SATCo score) (medication effect excluded)&#xD;
  &#xD;
            -  Fixed hemi-dystonia&#xD;
  &#xD;
            -  Severe spasticity in knee- and elbow-flexors and -extensors (Modified Ashworth Scale&#xD;
               &gt;3)&#xD;
  &#xD;
            -  Fixed severe skeletal contractions with loss of function which require immediate&#xD;
               orthopaedic surgical intervention&#xD;
  &#xD;
            -  Patients with other severe concurrent neurological disease (e. g. brain tumor,&#xD;
               neurodegenerative diseases, trauma etc.)&#xD;
  &#xD;
            -  Condition likely to require use of MRI in the future&#xD;
  &#xD;
            -  Any intracranial abnormality or medical condition that would contraindicate DBS&#xD;
               surgery&#xD;
  &#xD;
            -  Any findings in neuropsychological screening assessments that would contraindicate DBS&#xD;
               surgery&#xD;
  &#xD;
            -  Any current drug and / or alcohol abuse&#xD;
  &#xD;
            -  Any history of frequent grand-mal seizures without response to anticonvulsive&#xD;
               treatment&#xD;
  &#xD;
            -  Any other active implanted device (e.g. Cochlear implant, pacemaker), whether turned&#xD;
               on or off, would be allowed provided that they do not interfere with functioning of&#xD;
               the device.&#xD;
  &#xD;
            -  The presence of DBS leads due the risks of these ferromagnetic devices in the MRI&#xD;
               environment.&#xD;
  &#xD;
            -  A history of neurostimulation intolerance in any area of the body.&#xD;
  &#xD;
            -  Currently on any anticoagulant medications that cannot be discontinued during&#xD;
               perioperative period.&#xD;
  &#xD;
            -  Any significant medical condition that is likely to interfere with study procedures or&#xD;
               likely to confound evaluation of study endpoints, including any terminal illness with&#xD;
               survival &lt;24 months.&#xD;
  &#xD;
            -  Participation in another drug, device, or biologics trial concurrently or within the&#xD;
               preceding 30 days; any other trial participation should be approved by the Principal&#xD;
               Investigator.&#xD;
  &#xD;
            -  A female that is breastfeeding or of childbearing potential with a positive urine&#xD;
               pregnancy test or - if a person is sexually active - not using sufficient&#xD;
               contraception with a Pearl Index of less than 1% including all forms of hormonal&#xD;
               contraception (&quot;antibaby-pill&quot;, hormonal plaster, NuvaRing®, Implanon®, hormonal depot&#xD;
               injections, contraceptive coil), the tubal ligature (female sterilization).&#xD;
               Alternatively, the female of child bearing potential is sexually abstinent.&#xD;
  &#xD;
            -  Subjects who have contraindications to anesthesia, in the judgment of the attending&#xD;
               anesthesiologist&#xD;
  &#xD;
            -  Subjects who are unwilling or unable to undergo general anesthesia&#xD;
  &#xD;
            -  Presence of a condition or abnormality that in the opinion of the Investigator would&#xD;
               compromise the safety of the subject or the quality of the data.&#xD;
  &#xD;
            -  Minimum head circumference &lt; 49cm&#xD;
  &#xD;
            -  Skull Density Ratio (SDR) &lt;0.40.&#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>Chima Oluigbo, MD</last_name>
      <role>Principal Investigator</role>
      <affiliation>Children's National Hospital, Washington, DC</affiliation>
    </overall_official>
    <overall_contact>
      <last_name>Chima Oluigbo, MD</last_name>
      <phone>2024763531</phone>
      <email>coluigbo@childrensnational.org</email>
    </overall_contact>
    <overall_contact_backup>
      <last_name>Saige Teti</last_name>
      <phone>2024763755</phone>
      <email>sateti@childrensnational.org</email>
    </overall_contact_backup>
    <verification_date>September 2023</verification_date>
    <study_first_submitted>May 2, 2023</study_first_submitted>
    <study_first_submitted_qc>September 12, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">September 13, 2023</study_first_posted>
    <last_update_submitted>September 12, 2023</last_update_submitted>
    <last_update_submitted_qc>September 12, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">September 13, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Principal Investigator</responsible_party_type>
      <investigator_affiliation>Children's National Research Institute</investigator_affiliation>
      <investigator_full_name>Chima Oluigbo</investigator_full_name>
      <investigator_title>Principal Investigator and Sponsor</investigator_title>
    </responsible_party>
    <keyword>High intensity focused ultrasound (HIFU)</keyword>
    <keyword>Pallidotomy</keyword>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Cerebral Palsy</mesh_term>
      <mesh_term>Dystonia</mesh_term>
      <mesh_term>Dystonic Disorders</mesh_term>
    </condition_browse>
    <patient_data>
      <sharing_ipd>No</sharing_ipd>
    </patient_data>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: 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/NCT06036199
  org study id: G230044
  nct id: NCT06036199
  lead sponsor: 
  has dmc: Yes
  is fda regulated drug: No
  is fda regulated device: Yes
  The primary objective of the proposed study is to evaluate the safety of ExAblate
        Transcranial MRgFUS as a tool for creating bilateral or unilateral lesions in the globus
        pallidus (GPi) in patients with treatment-refractory secondary dystonia due to dyskinetic
        cerebral palsy
  The primary objective of the proposed study is to evaluate the safety of ExAblate
        Transcranial MRgFUS as a tool for creating bilateral or unilateral lesions in the globus
        pallidus (GPi) in patients with treatment-refractory secondary dystonia due to dyskinetic
        cerebral palsy.
  
        The secondary is to assess the impact on Quality of Life of Focused Ultrasound Bilateral and
        unilateral Pallidotomy for Dyskinetic Cerebral Palsy in Pediatric and Young Adult Subjects.
        In addition, the impact of bilateral pallidotomy on motor development, pain perception,
        speech, memory, attention and cognition in these patients will also be assessed.
  allocation: N/A
  intervention model: Single Group Assignment
  primary purpose: Treatment
  masking: None (Open Label)
  measure: Incidence and severity of device-related and procedure-related adverse events (AE)
  time frame: 2 years
  description: Safety will be determined by an evaluation of the incidence and severity of device-related and procedure-related adverse events (AE) from the first treatment day visit through the 24-month post-treatment time point. All AEs will be reported and categorized as related to the device versus the ablation procedure.
  measure: Burke-Fahn-Marsden-Dystonia Rating Scale movement and disability
  time frame: 2 years
  description: Assessment of speech and swallowing. Score range: 0-30, 0 indicates complete independence, 30 indicates complete dependence.
  measure: Family Scale (FaBel)
  time frame: 2 years
  description: Assessment of the burden for caregivers. Score range: 0-84, 0 indicates no burden, 84 indicates very severe burden.
  measure: Canadian Occupational Performance Measure (COPM)
  time frame: 2 years
  description: Assessment of activities of daily living.
  measure: Gross Motor Function Measure (GMFM-66)
  time frame: 2 years
  description: Assessment of physical disability. Score range: 0-3, 0 indicates no performance, 3 indicates complete performance.
  measure: Gross Motor Function Classification System (GMFCS)
  time frame: 2 years
  description: Degree of physical impairment. Score range: 1-5, 1 indicates no limitations, 5 indicates severe limitations.
  measure: SF-36 for assessment of quality of life
  time frame: 2 years
  description: Assessment of quality of life of subject. Score range: 0-100, 0 indicates bad health state, 100 indicates an excellent health state.
  measure: Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD)
  time frame: 2 years
  description: Assessment of Quality of life of Caregivers and Subject. Score range: 0-100, 0 indicates a bad score, 100 indicates an excellent score.
  measure: Frenchay Dysarthria Assessment
  time frame: 2 years
  description: Assessment of speech and swallowing. Score range:
  measure: Assessment of Cognition
  time frame: 2 years
  description: Snijders-Oomen-Non-Verbal-Intelligence Test (SON-R)
  measure: Strengths and Difficulties Questionnaire
  time frame: 2 years
  description: Assessment of mood and attention. Score range: 0-40, 0 indicates normal, 40 indicates abnormal.
  measure: Assessment of Attention
  time frame: 2 years
  description: Attentional Network Test (ANT)
  measure: Assessment of cognition
  time frame: 2 years
  description: Non-Verbal-Learning Test (NVLT)
  measure: Wong Baker Faces
  time frame: 2 years
  description: Assessment of pain. Score range; 0-10, 0 indicates no pain, 10 indicates worst pain.
  measure: Tardieu Scale
  time frame: 2 years
  description: Assessment of the severity of spasticity. Score range: 0-5, 0 indicates no resistance, 5 indicates no movement.
  measure: Dyskinesia Impairment Scale (DIS)
  time frame: 2 years
  description: Assessment of the severity of chorea and dystonia. Score range: 0-576, 0 indicates no dystonia, 576 indicates very severe dystonia.
  measure: Barry Albright Dystonia Scale
  time frame: 2 years
  description: Score range: 0-32, 0 indicates no dystonia, 32 indicates severe dystonia.
  arm group label: Focused Ultrasound Pallidotomy
  arm group type: Experimental
  description: Pediatric and young adult patients between ages of 8 and 22 years with pharmaco-resistant secondary dystonia due to dyskinetic cerebral palsy who have Focused Ultrasound Pallidotomy
  intervention type: Device
  intervention name: Focused Ultrasound Pallidotomy
  description: Use of ExAblate Transcranial MR guided Focused Ultrasound as a tool for creating bilateral or unilateral lesions in the globus pallidus (GPi) in patients with treatment-refractory secondary dystonia due to dyskinetic cerebral palsy
  arm group label: Focused Ultrasound Pallidotomy
  criteria: 
  gender: All
  minimum age: 8 Years
  maximum age: 22 Years
  healthy volunteers: No
  last name: Chima Oluigbo, MD
  role: Principal Investigator
  affiliation: Children's National Hospital, Washington, DC
  last name: Chima Oluigbo, MD
  phone: 2024763531
  email: coluigbo@childrensnational.org
  last name: Saige Teti
  phone: 2024763755
  email: sateti@childrensnational.org
  responsible party type: Principal Investigator
  investigator affiliation: Children's National Research Institute
  investigator full name: Chima Oluigbo
  investigator title: Principal Investigator and Sponsor
  mesh term: Cerebral Palsy
  mesh term: Dystonia
  mesh term: Dystonic Disorders
  sharing ipd: No