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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
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
Children's National Hospital
Washington D.C., District of Columbia, United States
RECRUITINGIncidence and severity of device-related and procedure-related adverse events (AE)
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.
Time frame: 2 years
Burke-Fahn-Marsden-Dystonia Rating Scale movement and disability
Assessment of speech and swallowing. Score range: 0-30, 0 indicates complete independence, 30 indicates complete dependence.
Time frame: 2 years
Family Scale (FaBel)
Assessment of the burden for caregivers. Score range: 0-84, 0 indicates no burden, 84 indicates very severe burden.
Time frame: 2 years
Canadian Occupational Performance Measure (COPM)
Assessment of activities of daily living.
Time frame: 2 years
Gross Motor Function Measure (GMFM-66)
Assessment of physical disability. Score range: 0-3, 0 indicates no performance, 3 indicates complete performance.
Time frame: 2 years
Gross Motor Function Classification System (GMFCS)
Degree of physical impairment. Score range: 1-5, 1 indicates no limitations, 5 indicates severe limitations.
Time frame: 2 years
SF-36 for assessment of quality of life
Assessment of quality of life of subject. Score range: 0-100, 0 indicates bad health state, 100 indicates an excellent health state.
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Time frame: 2 years
Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD)
Assessment of Quality of life of Caregivers and Subject. Score range: 0-100, 0 indicates a bad score, 100 indicates an excellent score.
Time frame: 2 years
Frenchay Dysarthria Assessment
Assessment of speech and swallowing. Score range:
Time frame: 2 years
Assessment of Cognition
Snijders-Oomen-Non-Verbal-Intelligence Test (SON-R)
Time frame: 2 years
Strengths and Difficulties Questionnaire
Assessment of mood and attention. Score range: 0-40, 0 indicates normal, 40 indicates abnormal.
Time frame: 2 years
Assessment of Attention
Attentional Network Test (ANT)
Time frame: 2 years
Assessment of cognition
Non-Verbal-Learning Test (NVLT)
Time frame: 2 years
Wong Baker Faces
Assessment of pain. Score range; 0-10, 0 indicates no pain, 10 indicates worst pain.
Time frame: 2 years
Tardieu Scale
Assessment of the severity of spasticity. Score range: 0-5, 0 indicates no resistance, 5 indicates no movement.
Time frame: 2 years
Dyskinesia Impairment Scale (DIS)
Assessment of the severity of chorea and dystonia. Score range: 0-576, 0 indicates no dystonia, 576 indicates very severe dystonia.
Time frame: 2 years
Barry Albright Dystonia Scale
Score range: 0-32, 0 indicates no dystonia, 32 indicates severe dystonia.
Time frame: 2 years