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

NCT06036199 | Recruiting DMC FDA Device

• 1 other identifier: G230044
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Cerebral Palsy, Dyskinetic; Secondary Dystonia

Keywords

High intensity focused ultrasound (HIFU); Pallidotomy

Outcome Measures

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.

  2 years

Secondary Outcomes (16)

• Burke-Fahn-Marsden-Dystonia Rating Scale movement and disability

  2 years

• Family Scale (FaBel)

  2 years

• Canadian Occupational Performance Measure (COPM)

  2 years

• Gross Motor Function Measure (GMFM-66)

  2 years

• Gross Motor Function Classification System (GMFCS)

  2 years

Study Arms (1)

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

Device: Focused Ultrasound Pallidotomy

Interventions

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

Focused Ultrasound Pallidotomy

Eligibility Criteria

• Age: 8 Years - 22 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

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.

Sponsors & Collaborators

• Children's National Research Institute: lead

Study Sites (1)

Children's National Hospital

Washington D.C., District of Columbia, 20010, United States

RECRUITING

MeSH Terms

Conditions

Cerebral Palsy; Dystonic Disorders

Condition Hierarchy (Ancestors)

Brain Damage, Chronic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders

Study Officials

• Chima Oluigbo, MD

  Children's National Hospital, Washington, DC

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Chima Oluigbo, MD

CONTACT

2024763531; coluigbo@childrensnational.org

Saige Teti

CONTACT

2024763755; sateti@childrensnational.org

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator and Sponsor

Study Record Dates

• First Submitted: May 2, 2023
• First Posted: September 13, 2023
• Study Start: January 26, 2024
• Primary Completion: August 1, 2025
• Study Completion: August 1, 2025
• Last Updated: February 11, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)