RECRUITING

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

Conditions

Cerebral Palsy, Dyskinetic Secondary Dystonia High intensity focused ultrasound (HIFU)Pallidotomy

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Official Title

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

Quick Facts

Study Start:2024-01-26

Study Completion:2025-08

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06036199

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:8 Years to 22 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT

Inclusion Criteria	Exclusion 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	* 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.

Inclusion Criteria

Exclusion 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

* 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.

Contacts and Locations

Study Contact

Chima Oluigbo, MD

CONTACT

2024763531

coluigbo@childrensnational.org

Saige Teti

CONTACT

2024763755

sateti@childrensnational.org

Principal Investigator

Chima Oluigbo, MD

PRINCIPAL_INVESTIGATOR

Children's National Hospital, Washington, DC

Study Locations (Sites)

Children's National Hospital

Washington, District of Columbia, 20010

United States

Collaborators and Investigators

Sponsor: Children's National Research Institute

Chima Oluigbo, MD, PRINCIPAL_INVESTIGATOR, Children's National Hospital, Washington, DC

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-01-26

Study Completion Date2025-08

Study Record Updates

Study Start Date2024-01-26

Study Completion Date2025-08

Terms related to this study

Keywords Provided by Researchers

High intensity focused ultrasound (HIFU)
Pallidotomy

Additional Relevant MeSH Terms

Cerebral Palsy, Dyskinetic
Secondary Dystonia