14 Participants Needed

MR-Guided Focused Ultrasound + GCase for Parkinson's Disease

NA
Overseen ByNadir Alikacem
Age: 18+
Sex: Any
Trial Phase: Academic
Sponsor: InSightec
Must be taking: PD medications
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Approved in 3 JurisdictionsThis treatment is already approved in other countries

Trial Summary

Do I need to stop my current medications for this trial?

The trial requires that you have been on a stable regimen of Parkinson's disease medications for at least 90 days before the study, so you should not stop taking your current medications.

What data supports the effectiveness of this treatment for Parkinson's Disease?

Research shows that magnetic resonance-guided focused ultrasound (MRgFUS) is approved and used for treating tremor-dominant Parkinson's Disease, indicating its potential effectiveness. It is a non-invasive method that has been shown to help reduce tremors in patients.12345

Is MR-guided Focused Ultrasound safe for humans?

MR-guided Focused Ultrasound (MRgFUS) has been used safely in humans for conditions like Parkinson's disease and essential tremor, with studies showing it as an incisionless (no cutting) treatment option. While some side effects have been noted, the procedure is generally considered safe based on experiences from multiple treatments.13467

How is the MR-guided Focused Ultrasound Plus GCase treatment different from other Parkinson's disease treatments?

The MR-guided Focused Ultrasound Plus GCase treatment is unique because it uses focused ultrasound waves guided by MRI to target specific brain areas without making any incisions, offering a non-invasive alternative to traditional surgical methods like deep brain stimulation.23489

What is the purpose of this trial?

This trial aims to test a new treatment for Parkinson's Disease by delivering an enzyme directly into the brain using MRI and focused ultrasound. It targets patients with genetic or unknown causes of Parkinson's. The enzyme may help reduce harmful substances in the brain and improve symptoms. Focused ultrasound combined with microbubbles is becoming a promising technology for the treatment of central nervous system diseases, including Parkinson's Disease.

Eligibility Criteria

This trial is for men and women aged 35-75 with Parkinson's Disease diagnosed at least 2 years ago, at stages 1-3 on medication. Participants must have a positive DAT SPECT scan, be on stable PD meds for over 90 days, and either carry a GBA mutation or not, depending on the study arm. Exclusions include pregnancy, severe kidney issues, other clinical trials participation, hypersensitivity to certain agents used in the study or MRI contrasts.

Inclusion Criteria

Dopaminergic deficit by a positive DAT SPECT scan
Able and willing to give informed consent
American Society of Anesthesiologists (ASA) score 1-3
See 6 more

Exclusion Criteria

My kidney function is severely impaired or I am on dialysis.
Insulin-dependent diabetes mellitus that is not well-controlled or that in the Investigator's opinion precludes participation in the study
I have had brain surgery for Parkinson's disease.
See 10 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Intervention

Participants receive three transcranial bilateral putamenal GCase treatments at 30 IU/kg IV every two weeks, followed by 60 IU/kg in the next three subjects

6 weeks
3 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

Treatment Details

Interventions

  • Blood Brain Barrier Disruption - Functional
  • MR-guided Focused Ultrasound Plus GCase
Trial Overview The trial tests if it's safe and doable to deliver GCase directly into the brain using MR-guided Focused Ultrasound (MRgFUS). This method has shown promise in animal studies for improving Parkinson’s symptoms by exposing part of the brain called putamen to GCase.
Participant Groups
2Treatment groups
Experimental Treatment
Group I: Idiopathic PD PatientsExperimental Treatment1 Intervention
Idiopathic PD patients receiving 3 cycles of BBBO. There is option to include simultaneous GCase delivery after safety profile review by Data and Safety Monitoring Board (DSMB).
Group II: GBA PD PatientsExperimental Treatment1 Intervention
GBA PD patients receiving 3 cycles of BBBO. There is option to include simultaneous GCase delivery after safety profile review by Data and Safety Monitoring Board (DSMB).

MR-guided Focused Ultrasound Plus GCase is already approved in United States, European Union, Canada for the following indications:

🇺🇸
Approved in United States as MRgFUS for:
  • Essential Tremor
  • Parkinson's disease-related tremor
🇪🇺
Approved in European Union as MRgFUS for:
  • Essential Tremor
  • Parkinson's disease-related tremor
🇨🇦
Approved in Canada as MRgFUS for:
  • Essential Tremor
  • Parkinson's disease-related tremor

Find a Clinic Near You

Who Is Running the Clinical Trial?

InSightec

Lead Sponsor

Trials
92
Recruited
3,800+

Dr. Maurice R. Ferré

InSightec

Chief Executive Officer

MD

Dr. Arjun Desai

InSightec

Chief Medical Officer

MD

Findings from Research

Magnetic resonance guided focused ultrasound (MRgFUS) has been FDA approved for treating essential tremor that doesn't respond to other treatments, highlighting its safety and efficacy as a new intervention.
The article discusses optimization strategies for imaging before, during, and after the procedure, emphasizing the importance of effective MRI techniques to ensure successful ablation during treatment.
Cranial MR-guided Focused Ultrasound for Essential Tremor : Technical Considerations and Image Guidance.Levi Chazen, J., Stradford, T., Kaplitt, MG.[2020]
In a study of 29 patients who underwent MRgFUS thalamotomy for tremor-predominant Parkinson's Disease, 96% experienced immediate tremor improvement, and 63% maintained this improvement at a median follow-up of 16 months, indicating the efficacy of this incisionless treatment.
Patient satisfaction was high, with 69% reporting improved quality of life, although 38% experienced mild long-term side effects, particularly related to speech when additional targeting of certain brain areas was performed, which did not enhance tremor control.
Patient-Reported Outcomes After Focused Ultrasound Thalamotomy for Tremor-Predominant Parkinson's Disease.Maragkos, GA., Kosyakovsky, J., Zhao, P., et al.[2023]
In a study of 48 patients with medically refractory tremor-dominant Parkinson's disease, MRgFUS thalamotomy provided significant and sustained tremor control over a follow-up period of up to 3 years.
The procedure was associated with mild side effects, such as gait imbalance and sensory deficits, which were mostly manageable and persisted in some cases, indicating a favorable safety profile.
Focused Ultrasound Thalamotomy for Tremor in Parkinson's Disease: Outcomes in a Large, Prospective Cohort.Chua, MMJ., Blitz, SE., Ng, PR., et al.[2023]

References

Cranial MR-guided Focused Ultrasound for Essential Tremor : Technical Considerations and Image Guidance. [2020]
Patient-Reported Outcomes After Focused Ultrasound Thalamotomy for Tremor-Predominant Parkinson's Disease. [2023]
Focused Ultrasound Thalamotomy for Tremor in Parkinson's Disease: Outcomes in a Large, Prospective Cohort. [2023]
Clinical application of magnetic resonance-guided focused ultrasound in Parkinson's disease: a meta-analysis of randomized clinical trials. [2022]
MRI follow-up after magnetic resonance-guided focused ultrasound for non-invasive thalamotomy: the neuroradiologist's perspective. [2021]
Initiating a Magnetic Resonance-Guided Focused Ultrasound Program: Comprehensive Workflow and Lessons Learned from the Initial 116 Cases. [2023]
Safety and accuracy of incisionless transcranial MR-guided focused ultrasound functional neurosurgery: single-center experience with 253 targets in 180 treatments. [2023]
MRI-Guided Focused Ultrasound in Parkinson's Disease: A Review. [2020]
Magnetic Resonance-Guided focused ultrasound surgery for Parkinson's disease: A mini-review and comparison between deep brain stimulation. [2023]
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