MR-guided Focused Ultrasound Plus GCase

NCT05565443 | Recruiting DMC

• 1 other identifier: PD016
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

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.

Conditions

Parkinsons Disease

Keywords

Parkinson's disease; Glucocerebrosidase; GCase

Outcome Measures

Primary Outcomes (1)

• Adverse Events

  Measure of the adverse events through the intervention phase and follow-up. All adverse events and/or Serious Adverse Events will be documented and reported according to CTCAE criteria.

  12 Months

Secondary Outcomes (1)

• Feasibility of MRgFUS BBB opening for GCase brain delivery

  12 Months

Study Arms (2)

Idiopathic PD Patients

EXPERIMENTAL

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).

Device: Blood Brain Barrier Disruption - Functional

GBA PD Patients

EXPERIMENTAL

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).

Device: Blood Brain Barrier Disruption - Functional

Interventions

Blood Brain Barrier Disruption - Functional DEVICE

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

GBA PD Patients; Idiopathic PD Patients

Eligibility Criteria

• Age: 35 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• InSightec: lead

Study Sites (1)

Sunybrook Research Institute

Toronto, Ontario, Canada

RECRUITING

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Central Study Contacts

Alyssa Voelker-Christy

CONTACT

+1-817-948-5219; alyssav@insightec.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: A two-arm, open-label, intervention only clinical study. One arm to enroll 7 GBA PD patients, the second arm to enroll 7 idiopathic PD patients. The study involves focused ultrasound procedures targeting bilateral putamen in patients with Parkinson's Disease.

Study Record Dates

• First Submitted: September 7, 2022
• First Posted: October 4, 2022
• Study Start: November 30, 2022
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2028
• Last Updated: January 23, 2026

Record last verified: 2026-01

Locations

CA (1)