NCT05565443

MR-guided Focused Ultrasound Plus GCase

A Phase I/II Study for Bilateral Putamenal Delivery of Recombinant Glucocerebrosidase in Patients With Parkinson's Disease Using MR-guided Focused Ultrasound Induced Opening of the Blood-brain Barrier

• Status: Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 14 (estimated)
• Sponsor: InSightec · Industry
• Sex: All
• Age: 35 Years – 80 Years
• Healthy volunteers: Not accepted

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

Interventions

Type	Name	Description
DEVICE	Blood Brain Barrier Disruption - Functional	Delivery of GCase across the BBB to form a disease modifying strategy for patients with Parkinson's Disease

Timeline

• Start date: 2022-11-30
• Primary completion: 2027-12-31
• Completion: 2028-12-31
• First posted: 2022-10-04
• Last updated: 2026-01-23

Locations

1 site across 1 country: Canada