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Cerebral Palsy, DyskineticNCT06036199

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

A study testing Focused Ultrasound Pallidotomy in people with Cerebral Palsy, Dyskinetic and Secondary Dystonia, run by Children's National Research Institute at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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 Read more about this study

Who is it for?
Ages 8 to 22 years
What is being tested?
Focused Ultrasound Pallidotomy
Type of treatment
Medical device
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteUnited States
Study dates
26 Jan 2024to Aug 2028
Phase
Interventional
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 8 to 22 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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
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  • 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

Who cannot join

  • 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.)
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  • 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.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Chima Oluigbo, MD contact

2024763531

coluigbo@childrensnational.org

Saige Teti contact

2024763755

sateti@childrensnational.org

United States1 site, 1 recruiting

  • Children's National Hospital

    Washington D.C., District of Columbia, 20010

    Chima Oluigbo, MD, 2024763531, coluigbo@childrensnational.org

    Saige Teti, BA, 2024763020, sateti@childrensnational.org

    Recruiting

Other trials in United States

About this study

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

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

Read the detailed 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.

The secondary is to assess the impact on Quality of Life of Focused Ultrasound Bilateral and unilateral Pallidotomy for Dyskinetic Cerebral Palsy in Pediatric and Young Adult Subjects. In addition, the impact of bilateral pallidotomy on motor development, pain perception, speech, memory, attention and cognition in these patients will also be assessed.

Being tested
Focused Ultrasound Pallidotomy
Keywords
High intensity focused ultrasound (HIFU), Pallidotomy
Medical subject headings
Cerebral Palsy, Dystonic Disorders

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Focused Ultrasound PallidotomyDevice

    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

Study groups in NCT06036199
Study groupRoleWhat they receive
Focused Ultrasound PallidotomyExperimentalPediatric 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
Receives: Device: Focused Ultrasound Pallidotomy

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: 2 years

Secondary outcomes (16)

  1. Burke-Fahn-Marsden-Dystonia Rating Scale movement and disability

    Assessment of speech and swallowing. Score range: 0-30, 0 indicates complete independence, 30 indicates complete dependence.

    Measured: 2 years

  2. Family Scale (FaBel)

    Assessment of the burden for caregivers. Score range: 0-84, 0 indicates no burden, 84 indicates very severe burden.

    Measured: 2 years

  3. Canadian Occupational Performance Measure (COPM)

    Assessment of activities of daily living.

    Measured: 2 years

  4. Gross Motor Function Measure (GMFM-66)

    Assessment of physical disability. Score range: 0-3, 0 indicates no performance, 3 indicates complete performance.

    Measured: 2 years

  5. Gross Motor Function Classification System (GMFCS)

    Degree of physical impairment. Score range: 1-5, 1 indicates no limitations, 5 indicates severe limitations.

    Measured: 2 years

  6. SF-36 for assessment of quality of life

    Assessment of quality of life of subject. Score range: 0-100, 0 indicates bad health state, 100 indicates an excellent health state.

    Measured: 2 years

  7. Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD)

    Assessment of Quality of life of Caregivers and Subject. Score range: 0-100, 0 indicates a bad score, 100 indicates an excellent score.

    Measured: 2 years

  8. Frenchay Dysarthria Assessment

    Assessment of speech and swallowing. Score range:

    Measured: 2 years

  9. Assessment of Cognition

    Snijders-Oomen-Non-Verbal-Intelligence Test (SON-R)

    Measured: 2 years

  10. Strengths and Difficulties Questionnaire

    Assessment of mood and attention. Score range: 0-40, 0 indicates normal, 40 indicates abnormal.

    Measured: 2 years

  11. Assessment of Attention

    Attentional Network Test (ANT)

    Measured: 2 years

  12. Assessment of cognition

    Non-Verbal-Learning Test (NVLT)

    Measured: 2 years

  13. Wong Baker Faces

    Assessment of pain. Score range; 0-10, 0 indicates no pain, 10 indicates worst pain.

    Measured: 2 years

  14. Tardieu Scale

    Assessment of the severity of spasticity. Score range: 0-5, 0 indicates no resistance, 5 indicates no movement.

    Measured: 2 years

  15. Dyskinesia Impairment Scale (DIS)

    Assessment of the severity of chorea and dystonia. Score range: 0-576, 0 indicates no dystonia, 576 indicates very severe dystonia.

    Measured: 2 years

  16. Barry Albright Dystonia Scale

    Score range: 0-32, 0 indicates no dystonia, 32 indicates severe dystonia.

    Measured: 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted13 Sep 2023
  2. Study start26 Jan 2024
  3. Primary completionAug 2028 (estimated)
  4. Study completionAug 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase N/A
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06036199
Sponsor's protocol ID
G230044
Status last verified
Apr 2026
First submitted
2 May 2023
Last updated
11 Jun 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
Yes
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A study testing Focused Ultrasound Pallidotomy in people with Cerebral Palsy, Dyskinetic and Secondary Dystonia, run by Children's National Research Institute at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 11 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Chima Oluigbo, MD (2024763531, coluigbo@childrensnational.org), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence and severity of device-related and procedure-related adverse events (AE) (time frame: 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06036199.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jun 2026.

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