Using focused ultrasound to treat dyskinetic cerebral palsy in young patients

Safety and Impact on Quality of Life of Focused Ultrasound Pallidotomy for Dyskinetic Cerebral Palsy in Pediatric and Young Adult Subjects

This study is testing a new ultrasound treatment to see if it can help young people with dyskinetic cerebral palsy who haven't found relief from other medications.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 10 (estimated)
• Ages: 8 Years to 22 Years
• Sex: All
• Sponsor: Children's National Research Institute (Academic / other)
• Locations: 1 site (Washington D.C., District of Columbia)
• Trial ID: NCT06036199 on ClinicalTrials.gov

What this trial studies

This study evaluates the safety of ExAblate Transcranial MRgFUS for creating lesions in the globus pallidus in pediatric and young adult patients with treatment-refractory secondary dystonia due to dyskinetic cerebral palsy. It aims to assess not only the safety of the procedure but also its impact on the quality of life, motor development, pain perception, speech, memory, attention, and cognition. Participants will be aged 8 to 22 years and must have previously insufficient responses to anti-dystonic medications. The study will involve careful monitoring and assessment of outcomes following the intervention.

Who should consider this trial

Good fit: Ideal candidates are children and young adults aged 8-22 with secondary dystonia due to cerebral palsy who have not responded to conventional treatments.

Not a fit: Patients with severe skeletal deformities requiring immediate orthopedic intervention may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could significantly improve motor function and quality of life for patients with dyskinetic cerebral palsy.

How similar studies have performed: Previous studies using focused ultrasound for similar conditions have shown promising results, indicating potential for success in this novel application.

Eligibility criteria

Inclusion Criteria:

* 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

Exclusion Criteria:

* 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 this trial is running

Washington D.C., District of Columbia

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

Study contacts

• Principal investigator: Chima Oluigbo, MD — Children's National Hospital, Washington, DC
• Study coordinator: Chima Oluigbo, MD

Conditions Cerebral Palsy, Dyskinetic; Secondary Dystonia; High intensity focused ultrasound; Pallidotomy