Recruiting

MR-guided Focused Ultrasound with GCaseMR-guided Focused Ultrasound Plus GCase

Sponsor:

InSightec

Code:

NCT05565443

Conditions

Parkinsons Disease

Eligibility Criteria

Sex: All

Age: 35 - 70+

Healthy Volunteers: Not accepted

Interventions

Blood Brain Barrier Disruption - Functional

Study Details

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

Study ID

NCT05565443

Start date

Nov 30, 2022

Status verified date

Jan, 2026

Completion date

Dec 31, 2028

Anticipated

Primary completion date

Dec 31, 2027

Anticipated

Eligibility Criteria

Sex: All

Age: 35 - 70+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

14 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: 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).

experimental: 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).

Interventions

Blood Brain Barrier Disruption - Functional

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

Primary outcome measure

Adverse Events [ Time Frame: 12 Months ]

Central Contacts and Locations

Central contacts

Alyssa Voelker-Christy

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

Locations

Sunybrook Research Institute

Recruiting

Toronto, Ontario, Canada

Contacts

Inthuja Suthananthan

647-687-8062 inthuja.suthananthan@sri.utoronto.ca

Principal Investigator:

Nir Lipsman, M.D., Neurosurgery

More Information

Sponsor

InSightec

Last update posted

Jan 23, 2026

Last verified

Jan, 2026

Keywords

Parkinson's disease
Glucocerebrosidase
GCase

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by InSightec on 2026-01-23.