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

Recruiting now · Not applicable

Conditions studied: Cerebral Palsy, Dyskinetic, Secondary Dystonia

In brief

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

Key facts

Study ID

NCT06036199

Run by

Children's National Research Institute

People needed

10

Starts

2024-01-26

Expected to finish

2028-08-01

Last updated by the study team

2026-06-11

Who can join

Age: 8 and older, up to 22. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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.

Where it is running

• Children's National Hospital — Washington D.C., District of Columbia, United States (enrolling)

Full record on ClinicalTrials.gov

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