Parkinson DiseaseNCT04370665
Blood-Brain-Barrier Disruption With Cerezyme in Patient's With Parkinson's Disease
A study testing Exablate BBBD with Cerezyme in people with Parkinson Disease, run by InSightec at 1 site in Canada.
Trial 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). Read more about this study
- Who is it for?
- Ages 35 to 75 years
- Conditions the study is for
- Parkinson Disease
- What is being tested?
- Exablate BBBD with Cerezyme
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteCanada
- Study dates
- 16 Jul 2020to 25 Feb 2022
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 35 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
- Insulin-dependent diabetes mellitus that is not well-controlled or that in the Investigator's opinion precludes participation in the study.
- Known sensitivity to gadolinium
- Known sensitivity to DEFINITY ultrasound contrast agent or perflutren.
- Contraindications to MRI such as non-MRI-compatible implanted devices.
- Large subjects not fitting comfortably into the MRI scanner.
- Difficulty lying supine and still for up to 4 hours in the MRI unit or claustrophobia.
- Untreated, uncontrolled sleep apnea.
- Positive pregnancy test (for pre-menopausal women).
- Known life-threatening systemic disease.
- Severely impaired renal function and/or on dialysis.
- Right to left or bi-directional cardiac shunt.
- Subjects with evidence of cranial or systemic infection.
- Subjects with uncontrolled chronic pulmonary disorders.
- Subjects with a history of severe asthma, hay fever, or multiple allergies that is not well-controlled (e.g. with anaphylaxis).
- Subjects with a family or personal history of cardiac arrhythmia.
- Severe liver injury.
Trial locations and contacts
1 site in 1 country.
About this study
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
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).
Read the 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
- Being tested
- Exablate BBBD with Cerezyme
- Keywords
- Exablate, Focused Ultrasound
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Exablate BBBD with CerezymeDevice
Blood Brain Barrier Disruption via Exablate to deliver Cerezyme.
Other Names:
Exablate Neuro
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Device: Exablate BBBD with Cerezyme |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Immediately after MRgFUS BBBD procedure
Secondary outcomes (1)
Safety --Adverse events
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.
Measured: Through study completion, an average of 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 May 2020
- Study start16 Jul 2020
- Primary completion31 Dec 2021
- Study completion25 Feb 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinson DiseaseStudies of the same condition, open and under way.
- More trials run by InSightecThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Device feasibility
- Allocation
- Not applicable
- Intervention model
- Single group
- 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)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- InSightec
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04370665
- Sponsor's protocol ID
- PD013
- Status last verified
- Mar 2026
- First submitted
- 15 Apr 2020
- Last updated
- 20 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04370665
- Acronym / sponsor ID
- PD013
- Conditions
- Parkinson Disease
- Keywords
- Exablate, Focused Ultrasound
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- InSightecIndustry (pharma, biotech, devices)
- Study start
- 16 Jul 2020 (actual)
- Primary completion
- 31 Dec 2021 (actual)
- Study completion
- 25 Feb 2022 (actual)
- First posted
- 1 May 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Device feasibility
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- 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.
| Arm | Type | Receives |
|---|---|---|
| Open label single arm | Experimental | Device: Exablate BBBD with CerezymeUsing Exablate Model 4000 Type-2 to temporarily disrupt the blood brain barrier to deliver Cerezyme in patients with Parkinson's Disease. |
Who may qualify
- Ages
- 35 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (31 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Exablate BBBD with CerezymeBlood Brain Barrier Disruption via Exablate to deliver Cerezyme. Other Names: Exablate Neuro | Device | Open label single arm |
Primary outcome
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.
1 secondary outcome
Safety --Adverse events
Time frame: Through study completion, an average of 3 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Mar 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Exablate BBBD with Cerezyme in people with Parkinson Disease, run by InSightec at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 31 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Contrast enhancement on MR imaging (time frame: Immediately after MRgFUS BBBD procedure). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Mar 2026.