MR-guided Focused Ultrasound Plus GCase
ConditionParkinsons Disease
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age35-80
SponsorInSightec
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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.
Trial design
Design model
Parallel
Treatments tested in this trial
Blood Brain Barrier Disruption - Functional
DeviceDelivery of GCase across the BBB to form a disease modifying strategy for patients with Parkinson's Disease
Treatment groups
14 Participants
are divided into 2 treatment groupsGroup A: Idiopathic PD PatientsExperimental treatment 1 intervention
Group B: GBA PD PatientsExperimental treatment 1 intervention
Trial outcomes
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.
Time frame
12 MonthsSecondary outcomes
1
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 Source ClinicalTrials.gov