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.
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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
14
Delivery of GCase across the BBB to form a disease modifying strategy for patients with Parkinson's Disease
Sunybrook Research Institute
Toronto, Ontario, Canada
RECRUITINGAdverse 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.
Time frame: 12 Months
Feasibility of MRgFUS BBB opening for GCase brain delivery
Using MRI images to measure: 1. The qualitative measure of the MRI T1-weighted with gadolinium of contract extravasation in the sonicate putamen. 2. The qualitative measure of the dynamic contrast enhanced (DCE) MRI
Time frame: 12 Months
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