NCT04370665

Blood-Brain-Barrier Disruption With Cerezyme in Patient's With Parkinson's Disease

A Pilot Study for Brain Delivery of Cerezyme® in Patients With Parkinson's Disease Using MR-guided Focused Ultrasound Induced Opening of the Blood-brain Barrier

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: InSightec · Industry
• Sex: All
• Age: 35 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This clinical trial focus on the delivery of Cerezyme® in Parkinson's Disease (PD) patients using MR-guided focused ultrasound (MRgFUS) induced opening of the blood-brain barrier (BBB).

Detailed description

Leveraging the preclinical evidence and clinical experience with MRgFUS BBB opening in human subjects, the investigators propose a phase I open-label study to determine the safety and feasibility of three biweekly delivery of Cerezyme® (an analogue of the human enzyme beta-glucocerebrosidase) via MRgFUS induced BBB opening to unilateral putamen in PD. The putamen contains terminals of dopaminergic neurons from the substantia nigra, and is a critically affected brain region in PD. The primary objective is to determine the feasibility of repeated MRgFUS BBB opening for Cerezyme delivery in unilateral putamen. The secondary objective is to describe the safety and tolerability of the procedure. Another objective is to measure changes in PD relevant clinical scores and Cerezyme relevant biomarkers in the study.

Conditions

• Parkinson Disease

Interventions

Type	Name	Description
DEVICE	Exablate BBBD with Cerezyme	Blood Brain Barrier Disruption via Exablate to deliver Cerezyme. Other Names: Exablate Neuro

Timeline

• Start date: 2020-07-16
• Primary completion: 2021-12-31
• Completion: 2022-02-25
• First posted: 2020-05-01
• Last updated: 2026-03-20

Locations

1 site across 1 country: Canada