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

NCT04370665 | Completed DMC

• 1 other identifier: PD013
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

Brief 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).

Conditions

Parkinson Disease

Keywords

Exablate; Focused Ultrasound

Outcome Measures

Primary Outcomes (1)

• Contrast enhancement on MR imaging

  MRgFUS BBB Opening will be determined qualitatively by contrast enhancement in the sonicated putamen on T1-weighted with gadolinium MRI, and quantitatively by the change in intensity relative to contralateral unsonicated putamen immediately after MRgFUS BBBD procedure.

  Immediately after MRgFUS BBBD procedure

Secondary Outcomes (1)

• Safety --Adverse events

  Through study completion, an average of 3 months

Other Outcomes (2)

• Exploratory effectiveness of Cerezyme delivery via MRgFUS BBB Opening

  1 day after the final BBBD procedure

• Exploratory effectiveness of Cerezyme treatment via MRgFUS BBB Opening

  3 months after the final BBBD procedure.

Study Arms (1)

Open label single arm

EXPERIMENTAL

Using Exablate Model 4000 Type-2 to temporarily disrupt the blood brain barrier to deliver Cerezyme in patients with Parkinson's Disease.

Device: Exablate BBBD with Cerezyme

Interventions

Exablate BBBD with Cerezyme DEVICE

Blood Brain Barrier Disruption via Exablate to deliver Cerezyme. Other Names: Exablate Neuro

Open label single arm

Eligibility Criteria

• Age: 35 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Men or women between age 35 and 75 years, inclusive.
• Able and willing to give informed consent.
• Diagnosis of Parkinson's Disease.
• At least 2 years from initial diagnosis
• On stable regiment of PD medications for at least 90 days prior to the study
• Able to communicate during the Exablate MRgFUS procedure.
• Able to attend all study visits.

You may not qualify if:

• Previous neurosurgical procedure for PD
• Cerezyme hypersensitivity
• Patients who had recent intracranial hemorrhage or stroke
• Skull covered by scars, scalp disorders (e.g., eczema), or atrophy of the scalp. Patients who have clips or other metallic implanted objects in the skull or the brain.
• Patients who are on blood thinners within washout period prior to treatment (i.e., antiplatelet or vitamin K inhibitor anticoagulants within 7 days, non-vitamin K inhibitor anticoagulants within 72 hours, or heparin-derived compounds within 48 hours of treatment).
• Patients with a history of a bleeding disorder, blood clotting, coagulopathy or with a history of spontaneous tumor hemorrhage.
• Patients receiving bevacizumab (Avastin) therapy.
• Currently participating in another clinical therapeutic trial
• Documented myocardial infarction within six months of enrollment.
• Unstable angina on medication.
• Congestive heart failure.
• Unstable cardiac arrhythmia.
• Cardiac pacemaker.
• Severe hypertension (diastolic BP \> 100 on medication).
• Documented cerebral infarction within the past 12 months or TIA in the past 1 month.

Sponsors & Collaborators

• InSightec: lead

Study Sites (1)

Sunnybrook Health Sciences Centre

Toronto, Ontario, M4N 3M5, Canada

Location

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: DEVICE FEASIBILITY
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is a prospective, single-center, single-arm, open-labelled study to enroll six (6) patients with Parkinson's disease for three biweekly deliveries of Cerezyme® to the unilateral putamen corresponding to the most affected side using Exablate and Definity®-induced BBB opening. This is a dose escalation study. Each subject will be followed for three months after the final procedure.

Study Record Dates

• First Submitted: April 15, 2020
• First Posted: May 1, 2020
• Study Start: July 16, 2020
• Primary Completion: December 31, 2021
• Study Completion: February 25, 2022
• Last Updated: March 20, 2026

Record last verified: 2026-03

Locations

CA (1)