Focused Ultrasound Pallidotomy for Dyskinetic Cerebral Palsy in Pediatric and Young Adult Subjects
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age8-22
SponsorChildren's National Research Institute
About this trial
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
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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)
Trial design
Design model
Single group
Treatments tested in this trial
Focused Ultrasound Pallidotomy
DeviceUse 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
Treatment groups
10 Participants
are divided into 1 treatment groupGroup A: Focused Ultrasound PallidotomyExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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.
Time frame
2 yearsSecondary outcomes
1
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 years2
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 years3
Canadian Occupational Performance Measure (COPM)
Assessment of activities of daily living.
Time frame
2 years4
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 yearsSponsors and contacts
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Source ClinicalTrials.gov