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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Focused Ultrasound Pallidotomy | Experimental | 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 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Focused Ultrasound Pallidotomy | Device | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence 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. | 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 2 years |
| Family Scale (FaBel) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Chima Oluigbo, MD | Contact | 2024763531 | coluigbo@childrensnational.org | |
| Saige Teti | Contact | 2024763755 | sateti@childrensnational.org |
| Name | Affiliation | Role |
|---|---|---|
| Chima Oluigbo, MD | Children's National Hospital, Washington, DC | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's National Hospital | Recruiting | Washington D.C. | District of Columbia | 20010 | United States |
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| ID | Term |
|---|---|
| D002547 | Cerebral Palsy |
| D020821 | Dystonic Disorders |
| ID | Term |
|---|---|
| D001925 | Brain Damage, Chronic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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Assessment of the burden for caregivers. Score range: 0-84, 0 indicates no burden, 84 indicates very severe burden.
| 2 years |
| Canadian Occupational Performance Measure (COPM) | Assessment of activities of daily living. | 2 years |
| Gross Motor Function Measure (GMFM-66) | Assessment of physical disability. Score range: 0-3, 0 indicates no performance, 3 indicates complete performance. | 2 years |
| Gross Motor Function Classification System (GMFCS) | Degree of physical impairment. Score range: 1-5, 1 indicates no limitations, 5 indicates severe limitations. | 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. | 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. | 2 years |
| Frenchay Dysarthria Assessment | Assessment of speech and swallowing. Score range: | 2 years |
| Assessment of Cognition | Snijders-Oomen-Non-Verbal-Intelligence Test (SON-R) | 2 years |
| Strengths and Difficulties Questionnaire | Assessment of mood and attention. Score range: 0-40, 0 indicates normal, 40 indicates abnormal. | 2 years |
| Assessment of Attention | Attentional Network Test (ANT) | 2 years |
| Assessment of cognition | Non-Verbal-Learning Test (NVLT) | 2 years |
| Wong Baker Faces | Assessment of pain. Score range; 0-10, 0 indicates no pain, 10 indicates worst pain. | 2 years |
| Tardieu Scale | Assessment of the severity of spasticity. Score range: 0-5, 0 indicates no resistance, 5 indicates no movement. | 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. | 2 years |
| Barry Albright Dystonia Scale | Score range: 0-32, 0 indicates no dystonia, 32 indicates severe dystonia. | 2 years |
| D009069 | Movement Disorders |