Focused Ultrasound Pallidotomy for Cerebral Palsy

Recruiting in Palo Alto (17 mi)

Overseen by Chima Oluigbo, MD

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Academic

Recruiting

Sponsor: Children's National Research Institute

Must be taking: Anti-dystonic medications

Must not be taking: Anticoagulants

Disqualifiers: Primary dystonia, Severe spasticity, others

No Placebo Group

Approved in 1 Jurisdiction

Trial Summary

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

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. ^{1 2 3 4 5}

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. ^{1 2 5 6 7}

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. ^{1 2 3 5 8}

Research Team

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.

Exclusion Criteria

I don't have brain conditions that prevent DBS surgery.

Minimum head circumference < 49cm

Skull Density Ratio (SDR) <0.40

Trial 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 (Procedure)

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

Group I: Focused Ultrasound Pallidotomy Experimental Treatment 1 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

Who Is Running the Clinical Trial?

Children's National Research Institute

Lead Sponsor

Trials

227

Recruited

258,000+

Michelle Riley-Brown

Children's National Research Institute

Chief Executive Officer since 2023

MHA from Washington University School of Medicine in St. Louis, Bachelor's degree from Tulane University

Catherine Bollard

Children's National Research Institute

Chief Medical Officer

MBChB, MD

Findings from Research

In a study involving 10 patients with early-stage tremor-dominant Parkinson's disease who underwent MRgFUS thalamotomy, the procedure was found to effectively reduce tremors without the need to increase dopaminergic medication over 6 months.

In contrast, a control group of 20 patients on standard dopaminergic therapy showed a significant increase in their medication dosage, highlighting the potential of MRgFUS as a beneficial alternative treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Magnetic Resonance-Guided Focused Ultrasound Thalamotomy May Spare Dopaminergic Therapy in Early-Stage Tremor-Dominant Parkinson's Disease: A Pilot Study. Golfrè Andreasi, N., Cilia, R., Romito, LM., et al.[2023]

References

1. United States pubmed.ncbi.nlm.nih.gov

MR-guided focused ultrasound pallidotomy for Parkinson's disease: safety and feasibility. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Skull bone marrow injury caused by MR-guided focused ultrasound for cerebral functional procedures. [2019]

3. Switzerland pubmed.ncbi.nlm.nih.gov

Anatomical and Technical Reappraisal of the Pallidothalamic Tractotomy With the Incisionless Transcranial MR-Guided Focused Ultrasound. A Technical Note. [2020]

4. United States pubmed.ncbi.nlm.nih.gov

Magnetic Resonance-Guided Focused Ultrasound Thalamotomy May Spare Dopaminergic Therapy in Early-Stage Tremor-Dominant Parkinson's Disease: A Pilot Study. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

The efficacy and limits of magnetic resonance-guided focused ultrasound pallidotomy for Parkinson's disease: a Phase I clinical trial. [2019]

6. Germany pubmed.ncbi.nlm.nih.gov

Safety and efficacy of magnetic resonance imaging-guided focused ultrasound neurosurgery for Parkinson's disease: a systematic review. [2021]

7. United States pubmed.ncbi.nlm.nih.gov

Factors Associated with Headache and Nausea During Magnetic Resonance-Guided Focused Ultrasound for Tremor. [2022]

8. Switzerland pubmed.ncbi.nlm.nih.gov

Technical Comparison of Treatment Efficiency of Magnetic Resonance-Guided Focused Ultrasound Thalamotomy and Pallidotomy in Skull Density Ratio-Matched Patient Cohorts. [2022]