Actively Recruiting
Safety and Impact on Quality of Life of Focused Ultrasound Pallidotomy for Dyskinetic Cerebral Palsy in Pediatric and Young Adult Subjects
Led by Children's National Research Institute · Updated on 2025-02-11
10
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and impact of ExAblate Transcranial MR-guided Focused Ultrasound (MRgFUS) pallidotomy in children and young adults aged 8 to 22 years who have treatment-resistant secondary dystonia caused by dyskinetic cerebral palsy. This study aims to create lesions in the globus pallidus internus (GPi) using bilateral or unilateral focused ultrasound and assess quality of life and other motor and cognitive functions. Participants will undergo Focused Ultrasound Pallidotomy targeting the globus pallidus to create lesions as a treatment for their condition. This device-based intervention is designed for patients who have not responded well to medication. The study includes evaluation of both bilateral and unilateral procedures and will monitor effects on motor skills, pain perception, speech, memory, attention, and cognition. During the study, participants will be followed for two years to monitor safety by recording any device-related or procedure-related adverse events. Numerous assessments will be conducted at regular intervals to measure dystonia symptoms, motor function, quality of life, speech, cognition, and caregiver priorities. The study includes various rating scales and questionnaires to capture comprehensive effects of the treatment over the long term.
CONDITIONS
Brief Title
Focused Ultrasound Pallidotomy for Dyskinetic Cerebral Palsy in Pediatric and Young Adult Subjects
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Treating physician has chosen GPi-DBS or pallidotomy for secondary dystonia due to cerebral palsy
- Patient or legal representative has chosen pallidotomy as treatment
- Consent to participate obtained from patient or legal representative
- Age between 8 and 22 years at enrollment
- Diagnosis of secondary dystonia due to cerebral palsy caused by perinatal hypoxic injury
- Insufficient response to anti-dystonic medication
- Stable anti-dystonic medication for at least 30 days
- Intact globus pallidus internus (pars posterior) and motor thalamus on MRI not older than 2 years if possible
- No fixed severe skeletal deformities needing immediate orthopedic surgery
- Sufficient compliance to participate in the study
- Written informed consent provided before study procedures
You will not qualify if you...
- Known primary or idiopathic dystonia (e.g., DYT1)
- Severe axial hypotonia with total loss of head control
- Fixed hemi-dystonia
- Severe spasticity in knee and elbow flexors or extensors (Modified Ashworth Scale >3)
- Fixed severe skeletal contractions needing immediate orthopedic surgery
- Other severe neurological diseases (e.g., brain tumor, neurodegenerative diseases, trauma)
- Condition likely requiring MRI in the future
- Any intracranial abnormality or medical condition contraindicating DBS surgery
- Neuropsychological findings contraindicating DBS surgery
- Current drug or alcohol abuse
- History of frequent grand-mal seizures unresponsive to treatment
- Presence of active implanted devices interfering with study device
- Presence of DBS leads
- History of neurostimulation intolerance
- Use of anticoagulants that cannot be stopped during surgery
- Significant medical conditions interfering with study or confounding results
- Participation in another trial within 30 days unless approved
- Female breastfeeding or of childbearing potential with positive pregnancy test or insufficient contraception
- Contraindications to anesthesia
- Unwillingness or inability to undergo general anesthesia
- Investigator judgment that participation compromises safety or data quality
- Head circumference less than 49 cm
- Skull density ratio less than 0.40
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 1 day
Participants undergo focused ultrasound pallidotomy, a procedure to create lesions in specific brain areas to treat dyskinetic cerebral palsy.
1 procedure visit (in-person)
Duration - Up to 2 years
Participants are monitored for safety and quality of life impact after the procedure with assessments on motor development, pain, speech, cognition, and other functions.
Multiple follow-up visits over 2 years
Trial Site Locations
Total: 1 location
1
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
Research Team
C
Chima Oluigbo, MD
S
Saige Teti
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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