Blood-Brain-Barrier Disruption With Cerezyme in Patient's With Parkinson's Disease
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|ClinicalTrials.gov Identifier: NCT04370665|
Recruitment Status : Active, not recruiting
First Posted : May 1, 2020
Last Update Posted : January 12, 2021
|Condition or disease||Intervention/treatment||Phase|
|Parkinson Disease||Device: Exablate BBBD with Cerezyme||Not Applicable|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||4 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||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.|
|Masking:||None (Open Label)|
|Primary Purpose:||Device Feasibility|
|Official Title:||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|
|Actual Study Start Date :||July 16, 2020|
|Estimated Primary Completion Date :||December 2021|
|Estimated Study Completion Date :||June 2022|
Experimental: Open label single arm
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
Blood Brain Barrier Disruption via Exablate to deliver Cerezyme.
- Contrast enhancement on MR imaging [ Time Frame: Immediately after MRgFUS BBBD procedure ]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.
- Safety --Adverse events [ Time Frame: Through study completion, an average of 3 months ]Safety will be assessed by recording all adverse events and/or Serious Adverse Events that are BBBD related. Each Adverse Event will be documented for patterns of occurrence.
- Exploratory effectiveness of Cerezyme delivery via MRgFUS BBB Opening [ Time Frame: 1 day after the final BBBD procedure ]Exploratory effectiveness of Cerezyme delivery via MRgFUS BBB Opening will be measured by changes in levels of Cerezyme (GCase) and GCase substrates levels (e.g. GL1 and glucosylsphingosine or lyso-GL1) in cerebralspinal fluid (CSF) samples.
- Exploratory effectiveness of Cerezyme treatment via MRgFUS BBB Opening [ Time Frame: 3 months after the final BBBD procedure. ]Exploratory effectiveness of Cerezyme treatment via MRgFUS BBB Opening will be measured by changes in cognitive impairment via Mini-Mental Status Exam (MMSE).
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04370665
|Sunnybrook Health Sciences Centre|
|Toronto, Ontario, Canada, M4N 3M5|