10 Participants Needed

Focused Ultrasound Pallidotomy for Cerebral Palsy

CO
ST
Overseen BySaige Teti
Age: < 65
Sex: Any
Trial Phase: Academic
Sponsor: Children's National Research Institute
Must be taking: Anti-dystonic medications
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Approved in 1 JurisdictionThis treatment is already approved in other countries

Trial Summary

Will I have to stop taking my current medications?

The trial requires that your anti-dystonic medication has been stable for the last 30 days, so you may not need to stop taking it. However, if you are on anticoagulant medications, you will need to discontinue them during the perioperative period (time around surgery).

What data supports the effectiveness of the treatment Focused Ultrasound Pallidotomy for Cerebral Palsy?

Research shows that MR-guided focused ultrasound pallidotomy has been effective in improving motor symptoms in Parkinson's disease, with significant improvements in movement scores and quality of life. This suggests potential benefits for similar neurological conditions, like cerebral palsy, although direct evidence for cerebral palsy is not yet available.12345

Is MR-guided focused ultrasound pallidotomy generally safe for humans?

MR-guided focused ultrasound pallidotomy has been studied for conditions like Parkinson's disease, showing mostly mild and temporary side effects such as headaches and nausea. However, there was a case of skull bone injury, indicating that while generally safe, there are potential risks that need further investigation.12567

How is the treatment Focused Ultrasound Pallidotomy unique for cerebral palsy?

Focused Ultrasound Pallidotomy is unique because it uses sound waves to create precise lesions in the brain without needing surgery, which is different from traditional surgical methods. This non-invasive approach is guided by MRI, allowing for targeted treatment with potentially fewer side effects.12358

What is the purpose of this trial?

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

Research Team

CO

Chima Oluigbo, MD

Principal Investigator

Children's National Hospital, Washington, DC

Eligibility Criteria

This trial is for young individuals aged 8-22 with secondary dystonia due to cerebral palsy caused by perinatal hypoxic injury. They should have tried other medications without enough benefit and be considered for pallidotomy or GPi-DBS by their doctor. Participants need stable medication for the last month, an intact globus pallidus on MRI, no severe skeletal deformations requiring surgery, and must consent to the study.

Inclusion Criteria

I, or my child, understand the study and agree to participate.
I do not have severe bone deformities that require urgent surgery.
I, or my legal guardian, understand the study and have given written consent.
See 9 more

Exclusion Criteria

I don't have brain conditions that prevent DBS surgery.
Minimum head circumference < 49cm
Skull Density Ratio (SDR) <0.40
See 20 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants undergo Focused Ultrasound Pallidotomy to create lesions in the globus pallidus (GPi)

1 day
1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness, including assessments of quality of life, motor development, and cognition

24 months
Regular visits over 2 years

Long-term follow-up

Continued monitoring of adverse events and quality of life impacts

Additional 6 months

Treatment Details

Interventions

  • Focused Ultrasound Pallidotomy
Trial Overview The trial tests ExAblate Transcranial MRgFUS's safety in creating lesions within a brain region called the globus pallidus. This non-invasive procedure aims to alleviate treatment-resistant dystonic movements in patients with dyskinetic cerebral palsy.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: Focused Ultrasound PallidotomyExperimental Treatment1 Intervention
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

Focused Ultrasound Pallidotomy is already approved in United States for the following indications:

🇺🇸
Approved in United States as ExAblate Neuro for:
  • Essential Tremor
  • Tremor-predominant Parkinson's disease

Find a Clinic Near You

Who Is Running the Clinical Trial?

Children's National Research Institute

Lead Sponsor

Trials
227
Recruited
258,000+

Findings from Research

Unilateral MR-guided focused ultrasound (MRgFUS) pallidotomy significantly improved dyskinesia scores in 20 patients with Parkinson's disease, showing a 59% reduction in the Unified Dyskinesia Rating Scale (UDysRS) scores at 3 months, which was sustained at 12 months.
The procedure was safe, with mostly mild and transient adverse events, and no serious adverse effects as defined by US FDA criteria, indicating that MRgFUS could be a viable treatment option for managing motor fluctuations in Parkinson's disease.
MR-guided focused ultrasound pallidotomy for Parkinson's disease: safety and feasibility.Eisenberg, HM., Krishna, V., Elias, WJ., et al.[2023]
In a study of 30 patients treated with MR-guided focused ultrasound (MRgFUS) for movement disorders, 7 patients developed new skull lesions after treatment, although all were asymptomatic and lesions were not visible immediately after the procedure.
The study found that higher energy levels applied during the MRgFUS procedure were associated with the development of skull lesions, suggesting that local skull properties may influence the risk of such lesions, warranting further investigation.
Skull bone marrow injury caused by MR-guided focused ultrasound for cerebral functional procedures.Schwartz, ML., Yeung, R., Huang, Y., et al.[2019]
The study presents an optimized strategy for performing pallidothalamic tractotomy (PTT) using MR-guided focused ultrasound (MRgFUS) in patients with chronic therapy-resistant Parkinson's disease, based on detailed histological analysis of 6 human brain hemispheres.
A standardized target coverage for PTT was established, involving 5 to 7 preplanned lesion sub-units, which enhances the precision and effectiveness of the procedure while maintaining thermal dose control.
Anatomical and Technical Reappraisal of the Pallidothalamic Tractotomy With the Incisionless Transcranial MR-Guided Focused Ultrasound. A Technical Note.Gallay, MN., Moser, D., Federau, C., et al.[2020]

References

MR-guided focused ultrasound pallidotomy for Parkinson's disease: safety and feasibility. [2023]
Skull bone marrow injury caused by MR-guided focused ultrasound for cerebral functional procedures. [2019]
Anatomical and Technical Reappraisal of the Pallidothalamic Tractotomy With the Incisionless Transcranial MR-Guided Focused Ultrasound. A Technical Note. [2020]
Magnetic Resonance-Guided Focused Ultrasound Thalamotomy May Spare Dopaminergic Therapy in Early-Stage Tremor-Dominant Parkinson's Disease: A Pilot Study. [2023]
The efficacy and limits of magnetic resonance-guided focused ultrasound pallidotomy for Parkinson's disease: a Phase I clinical trial. [2019]
Safety and efficacy of magnetic resonance imaging-guided focused ultrasound neurosurgery for Parkinson's disease: a systematic review. [2021]
Factors Associated with Headache and Nausea During Magnetic Resonance-Guided Focused Ultrasound for Tremor. [2022]
Technical Comparison of Treatment Efficiency of Magnetic Resonance-Guided Focused Ultrasound Thalamotomy and Pallidotomy in Skull Density Ratio-Matched Patient Cohorts. [2022]
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