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Focused Ultrasound Pallidotomy for Dyskinetic Cerebral Palsy in Pediatric and Young Adult Subjects

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT06036199
Recruitment Status : Recruiting
First Posted : September 13, 2023
Last Update Posted : February 9, 2024
Sponsor:
Information provided by (Responsible Party):
Chima Oluigbo, Children's National Research Institute

Tracking Information
First Submitted Date  ICMJE May 2, 2023
First Posted Date  ICMJE September 13, 2023
Last Update Posted Date February 9, 2024
Actual Study Start Date  ICMJE January 26, 2024
Estimated Primary Completion Date August 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 12, 2023)
Incidence and severity of device-related and procedure-related adverse events (AE) [ Time Frame: 2 years ]
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: September 12, 2023)
  • Burke-Fahn-Marsden-Dystonia Rating Scale movement and disability [ Time Frame: 2 years ]
    Assessment of speech and swallowing. Score range: 0-30, 0 indicates complete independence, 30 indicates complete dependence.
  • Family Scale (FaBel) [ Time Frame: 2 years ]
    Assessment of the burden for caregivers. Score range: 0-84, 0 indicates no burden, 84 indicates very severe burden.
  • Canadian Occupational Performance Measure (COPM) [ Time Frame: 2 years ]
    Assessment of activities of daily living.
  • Gross Motor Function Measure (GMFM-66) [ Time Frame: 2 years ]
    Assessment of physical disability. Score range: 0-3, 0 indicates no performance, 3 indicates complete performance.
  • Gross Motor Function Classification System (GMFCS) [ Time Frame: 2 years ]
    Degree of physical impairment. Score range: 1-5, 1 indicates no limitations, 5 indicates severe limitations.
  • SF-36 for assessment of quality of life [ Time Frame: 2 years ]
    Assessment of quality of life of subject. Score range: 0-100, 0 indicates bad health state, 100 indicates an excellent health state.
  • Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) [ Time Frame: 2 years ]
    Assessment of Quality of life of Caregivers and Subject. Score range: 0-100, 0 indicates a bad score, 100 indicates an excellent score.
  • Frenchay Dysarthria Assessment [ Time Frame: 2 years ]
    Assessment of speech and swallowing. Score range:
  • Assessment of Cognition [ Time Frame: 2 years ]
    Snijders-Oomen-Non-Verbal-Intelligence Test (SON-R)
  • Strengths and Difficulties Questionnaire [ Time Frame: 2 years ]
    Assessment of mood and attention. Score range: 0-40, 0 indicates normal, 40 indicates abnormal.
  • Assessment of Attention [ Time Frame: 2 years ]
    Attentional Network Test (ANT)
  • Assessment of cognition [ Time Frame: 2 years ]
    Non-Verbal-Learning Test (NVLT)
  • Wong Baker Faces [ Time Frame: 2 years ]
    Assessment of pain. Score range; 0-10, 0 indicates no pain, 10 indicates worst pain.
  • Tardieu Scale [ Time Frame: 2 years ]
    Assessment of the severity of spasticity. Score range: 0-5, 0 indicates no resistance, 5 indicates no movement.
  • Dyskinesia Impairment Scale (DIS) [ Time Frame: 2 years ]
    Assessment of the severity of chorea and dystonia. Score range: 0-576, 0 indicates no dystonia, 576 indicates very severe dystonia.
  • Barry Albright Dystonia Scale [ Time Frame: 2 years ]
    Score range: 0-32, 0 indicates no dystonia, 32 indicates severe dystonia.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Focused Ultrasound Pallidotomy for Dyskinetic Cerebral Palsy in Pediatric and Young Adult Subjects
Official Title  ICMJE Safety and Impact on Quality of Life of Focused Ultrasound Pallidotomy for Dyskinetic Cerebral Palsy in Pediatric and Young Adult Subjects
Brief Summary 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
Detailed Description

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Cerebral Palsy, Dyskinetic
  • Secondary Dystonia
Intervention  ICMJE Device: Focused Ultrasound Pallidotomy
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
Study Arms  ICMJE Experimental: Focused Ultrasound Pallidotomy
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: Device: Focused Ultrasound Pallidotomy
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: September 12, 2023)
10
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE August 2025
Estimated Primary Completion Date August 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • The treating physician has chosen GPi-DBS or pallidotomy for the treatment of the secondary dystonia caused by cerebral palsy in this patient
  • Patient and/or legal representative, if the patient is underaged or not capable to give consent himself, have chosen pallidotomy as treatment
  • The consent to participate in the trial of the underaged patient, if he is capable to understand the study requirements, is required
  • Age at enrolment 8-22 years
  • Diagnosis of secondary dystonia due to cerebral palsy caused by perinatal hypoxic injury
  • Anti-dystonic pharmacotherapy insufficient
  • Stable anti-dystonic medication over the last 30 days
  • Globus pallidus internus (pars posterior) and thalamus (motor part) intact on MRI (not older than 2 years - if possible)
  • No fixed severe skeletal deformations with loss of function, which need immediate orthopaedic surgical intervention
  • Sufficient compliance of the patient or the legal representative if the patient is underaged or not capable to give consent himself to take part in the study
  • Informed consent to take part in the study from patient and/or legal representative if the patient is underaged or not capable to give consent himself
  • Patient and/or legal representative if the patient is underaged or not capable to give consent himself, understands the study requirements and the treatment procedures and provides written informed consent before any study-specific tests or procedures are performed

Exclusion Criteria:

  • Patients with known primary (e.g. DYT1) or idiopathic dystonia
  • Severe axial hypotonia with total loss of head control (e.g. absence of control at "upper thoracic level" in the SATCo score) (medication effect excluded)
  • Fixed hemi-dystonia
  • Severe spasticity in knee- and elbow-flexors and -extensors (Modified Ashworth Scale >3)
  • Fixed severe skeletal contractions with loss of function which require immediate orthopaedic surgical intervention
  • Patients with other severe concurrent neurological disease (e. g. brain tumor, neurodegenerative diseases, trauma etc.)
  • Condition likely to require use of MRI in the future
  • Any intracranial abnormality or medical condition that would contraindicate DBS surgery
  • Any findings in neuropsychological screening assessments that would contraindicate DBS surgery
  • Any current drug and / or alcohol abuse
  • Any history of frequent grand-mal seizures without response to anticonvulsive treatment
  • Any other active implanted device (e.g. Cochlear implant, pacemaker), whether turned on or off, would be allowed provided that they do not interfere with functioning of the device.
  • The presence of DBS leads due the risks of these ferromagnetic devices in the MRI environment.
  • A history of neurostimulation intolerance in any area of the body.
  • Currently on any anticoagulant medications that cannot be discontinued during perioperative period.
  • Any significant medical condition that is likely to interfere with study procedures or likely to confound evaluation of study endpoints, including any terminal illness with survival <24 months.
  • Participation in another drug, device, or biologics trial concurrently or within the preceding 30 days; any other trial participation should be approved by the Principal Investigator.
  • A female that is breastfeeding or of childbearing potential with a positive urine pregnancy test or - if a person is sexually active - not using sufficient contraception with a Pearl Index of less than 1% including all forms of hormonal contraception ("antibaby-pill", hormonal plaster, NuvaRing®, Implanon®, hormonal depot injections, contraceptive coil), the tubal ligature (female sterilization). Alternatively, the female of child bearing potential is sexually abstinent.
  • Subjects who have contraindications to anesthesia, in the judgment of the attending anesthesiologist
  • Subjects who are unwilling or unable to undergo general anesthesia
  • Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the subject or the quality of the data.
  • Minimum head circumference < 49cm
  • Skull Density Ratio (SDR) <0.40.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 8 Years to 22 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Chima Oluigbo, MD 2024763531 coluigbo@childrensnational.org
Contact: Saige Teti 2024763755 sateti@childrensnational.org
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT06036199
Other Study ID Numbers  ICMJE G230044
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: Yes
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party Chima Oluigbo, Children's National Research Institute
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Children's National Research Institute
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Chima Oluigbo, MD Children's National Hospital, Washington, DC
PRS Account Children's National Research Institute
Verification Date February 2024

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP