MR-guided Focused Ultrasound Plus GCase

NCT ID: NCT05565443

Last Updated: 2026-01-23

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: NA
• Total Enrollment: 14 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-11-30
• Study Completion Date: 2028-12-31

Brief Summary

The goal of this study is to establish safety and feasibility of intracerebral delivery of GCase via MRgFUS. This technique may offer potential benefits given the exposure of the putamen to GCase in animal models has been shown to be efficacious in improving Parkinson's disease pathology and phenotype.

Detailed Description

This is a two-arm, open-label, intervention only phase I/II clinical study. One arm of the study will enroll seven (7) GBA PD patients and the other arm seven (7) idiopathic PD patients. During the intervention phase, the first four subjects of each arm will receive three transcranial bilateral putamenal GCase at 30 IU/kg IV every two weeks, followed by 60 IU/kg in the next three subjects. Following three treatments in this study, the subjects will be followed for twelve months.

Conditions

Parkinsons Disease

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Idiopathic PD Patients

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 Type: EXPERIMENTAL

Blood Brain Barrier Disruption - Functional

Intervention Type: DEVICE

Delivery of GCase across the BBB to form a disease modifying strategy for patients with Parkinson's Disease

GBA PD Patients

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).

Group Type: EXPERIMENTAL

Blood Brain Barrier Disruption - Functional

Intervention Type: DEVICE

Delivery of GCase across the BBB to form a disease modifying strategy for patients with Parkinson's Disease

Interventions

Blood Brain Barrier Disruption - Functional

Delivery of GCase across the BBB to form a disease modifying strategy for patients with Parkinson's Disease

Intervention Type: DEVICE

Eligibility Criteria

Inclusion Criteria

• Men or women between age 35 and 80 years, inclusive.
• Able and willing to give informed consent.
• Diagnosis of PD satisfying MDS Clinical Diagnostic Criteria for PD
• At least 2 years from initial diagnosis
• Hoehn and Yahr Stage 1-3 on PD medication
• Dopaminergic deficit by a positive DAT SPECT scan
• On stable regimen of PD medications for at least 90 days prior to the study
• American Society of Anesthesiologists (ASA) score 1-3
• Harbor at least one GBA mutation if enrolled in the GBA PD arm or two wild-type GBA alleles if enrolled in the idiopathic PD arm

Exclusion Criteria

• Positive pregnancy test (for pre-menopausal women).
• Contraindication to DEFINITY ultrasound contrast agent or perflutren (e.g. hypersensitivity, known left or bidirectional cardiac shunt)
• Contraindication to MRI or gadolinium-DTPA (e.g. incompatible device, hypersensitivity)
• Insulin-dependent diabetes mellitus that is not well-controlled or that in the Investigator's opinion precludes participation in the study.
• Severely impaired renal function with estimated glomerular filtration rate <30 mL/min/1.73m2 and/or on dialysis.
• Currently participating in another clinical therapeutic trial
• Patient receiving bevacizumab (Avastin) therapy
• Subjects with evidence of cranial or systemic infection.
• Cerebral or systemic vasculopathy.
• Documented cerebral infarction within the past 12 months or TIA in the past 1 month.
• Contraindication to GCase enzyme therapy, specifically previous hypersensitivity reaction to GCase enzyme therapy
• Parkinsonism plus symptoms, secondary parkinsonism
• Previous neurosurgical procedure for PD

• Minimum Eligible Age: 35 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

InSightec

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Sunybrook Research Institute

Toronto, Ontario, Canada

Site Status: RECRUITING

Countries

Canada

Central Contacts

Alyssa Voelker-Christy

Role: CONTACT

alyssav@insightec.com

+1-817-948-5219

Facility Contacts

Inthuja Suthananthan

Role: primary

inthuja.suthananthan@sri.utoronto.ca

647-687-8062

Other Identifiers

PD016

Identifier Type: -

Identifier Source: org_study_id