Parkinsons DiseaseNCT05565443
MR-guided Focused Ultrasound Plus GCase
A study testing Blood Brain Barrier Disruption - Functional in people with Parkinsons Disease, run by InSightec at 1 site in Canada.
Trial 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. Read more about this study
- Who is it for?
- Ages 35 to 80 years
- Conditions the study is for
- Parkinsons Disease
- What is being tested?
- Blood Brain Barrier Disruption - Functional
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteCanada
- Study dates
- 30 Nov 2022to 31 Dec 2028
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Men or women between age 35 and 80 years, inclusive.
- Able and willing to give informed consent.
- Diagnosis of PD satisfying MDS Clinical Diagnostic Criteria for PD
- At least 2 years from initial diagnosis
- Hoehn and Yahr Stage 1-3 on PD medication
- Dopaminergic deficit by a positive DAT SPECT scan
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- On stable regimen of PD medications for at least 90 days prior to the study
- American Society of Anesthesiologists (ASA) score 1-3
- Harbor at least one GBA mutation if enrolled in the GBA PD arm or two wild-type GBA alleles if enrolled in the idiopathic PD arm
Who cannot join
- Positive pregnancy test (for pre-menopausal women).
- Contraindication to DEFINITY ultrasound contrast agent or perflutren (e.g. hypersensitivity, known left or bidirectional cardiac shunt)
- Contraindication to MRI or gadolinium-DTPA (e.g. incompatible device, hypersensitivity)
- Insulin-dependent diabetes mellitus that is not well-controlled or that in the Investigator's opinion precludes participation in the study.
- Severely impaired renal function with estimated glomerular filtration rate <30 mL/min/1.73m2 and/or on dialysis.
- Currently participating in another clinical therapeutic trial
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- Patient receiving bevacizumab (Avastin) therapy
- Subjects with evidence of cranial or systemic infection.
- Cerebral or systemic vasculopathy.
- Documented cerebral infarction within the past 12 months or TIA in the past 1 month.
- Contraindication to GCase enzyme therapy, specifically previous hypersensitivity reaction to GCase enzyme therapy
- Parkinsonism plus symptoms, secondary parkinsonism
- Previous neurosurgical procedure for PD
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Canada1 site, 1 recruiting
- Recruiting
Sunybrook Research Institute
Toronto, Ontario
Inthuja Suthananthan, 647-687-8062, inthuja.suthananthan@sri.utoronto.ca
Nir Lipsman, M.D., Neurosurgery
About this study
Official title: 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
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.
Read the 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
- Being tested
- Blood Brain Barrier Disruption - Functional
- Keywords
- Parkinson's disease, Glucocerebrosidase, GCase
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Blood Brain Barrier Disruption - FunctionalDevice
Delivery of GCase across the BBB to form a disease modifying strategy for patients with Parkinson's Disease
| Study group | Role | What they receive |
|---|---|---|
| Idiopathic PD Patients | Experimental | Idiopathic PD patients receiving 3 cycles of BBBO. There is option to include simultaneous GCase delivery after safety profile review by Data and Safety Monitoring Board (DSMB). Receives: Device: Blood Brain Barrier Disruption - Functional |
| GBA PD Patients | Experimental | GBA PD patients receiving 3 cycles of BBBO. There is option to include simultaneous GCase delivery after safety profile review by Data and Safety Monitoring Board (DSMB). Receives: Device: Blood Brain Barrier Disruption - Functional |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Measure of the adverse events through the intervention phase and follow-up. All adverse events and/or Serious Adverse Events will be documented and reported according to CTCAE criteria.
Measured: 12 Months
Secondary outcomes (1)
Feasibility of MRgFUS BBB opening for GCase brain delivery
Using MRI images to measure:
- The qualitative measure of the MRI T1-weighted with gadolinium of contract extravasation in the sonicate putamen.
- The qualitative measure of the dynamic contrast enhanced (DCE) MRI
Measured: 12 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Oct 2022
- Study start30 Nov 2022
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinsons DiseaseStudies of the same condition, open and under way.
- 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
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- A two-arm, open-label, intervention only clinical study. One arm to enroll 7 GBA PD patients, the second arm to enroll 7 idiopathic PD patients. The study involves focused ultrasound procedures targeting bilateral putamen in patients with Parkinson's Disease.
- Masking
- None (open label)
- Enrolment
- 14 participants (estimated)
Sponsor and investigators
- Lead sponsor
- InSightec
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05565443
- Sponsor's protocol ID
- PD016
- Status last verified
- Jan 2026
- First submitted
- 7 Sep 2022
- Last updated
- 23 Jan 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 23 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05565443
- Acronym / sponsor ID
- PD016
- Conditions
- Parkinsons Disease
- Keywords
- Parkinson's disease, Glucocerebrosidase, GCase
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 14 participants (estimated)
- Lead sponsor
- InSightecIndustry (pharma, biotech, devices)
- Study start
- 30 Nov 2022 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 4 Oct 2022 (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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- A two-arm, open-label, intervention only clinical study. One arm to enroll 7 GBA PD patients, the second arm to enroll 7 idiopathic PD patients. The study involves focused ultrasound procedures targeting bilateral putamen in patients with Parkinson's Disease.
| Arm | Type | Receives |
|---|---|---|
| Idiopathic PD Patients | Experimental | Device: Blood Brain Barrier Disruption - FunctionalIdiopathic PD patients receiving 3 cycles of BBBO. There is option to include simultaneous GCase delivery after safety profile review by Data and Safety Monitoring Board (DSMB). |
| GBA PD Patients | Experimental | Device: Blood Brain Barrier Disruption - FunctionalGBA PD patients receiving 3 cycles of BBBO. There is option to include simultaneous GCase delivery after safety profile review by Data and Safety Monitoring Board (DSMB). |
Who may qualify
- Ages
- 35 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Men or women between age 35 and 80 years, inclusive.
- Able and willing to give informed consent.
- Diagnosis of PD satisfying MDS Clinical Diagnostic Criteria for PD
- At least 2 years from initial diagnosis
- Hoehn and Yahr Stage 1-3 on PD medication
Key exclusion criteria (13 listed)
- Positive pregnancy test (for pre-menopausal women).
- Contraindication to DEFINITY ultrasound contrast agent or perflutren (e.g. hypersensitivity, known left or bidirectional cardiac shunt)
- Contraindication to MRI or gadolinium-DTPA (e.g. incompatible device, hypersensitivity)
- Insulin-dependent diabetes mellitus that is not well-controlled or that in the Investigator's opinion precludes participation in the study.
- Severely impaired renal function with estimated glomerular filtration rate <30 mL/min/1.73m2 and/or on dialysis.
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 |
|---|---|---|
| Blood Brain Barrier Disruption - FunctionalDelivery of GCase across the BBB to form a disease modifying strategy for patients with Parkinson's Disease | Device | GBA PD Patients, Idiopathic PD Patients |
Primary outcome
Adverse Events
Time frame: 12 Months
Measure of the adverse events through the intervention phase and follow-up. All adverse events and/or Serious Adverse Events will be documented and reported according to CTCAE criteria.
1 secondary outcome
Feasibility of MRgFUS BBB opening for GCase brain delivery
Time frame: 12 Months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jan 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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Blood Brain Barrier Disruption - Functional in people with Parkinsons 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 9 inclusion and 13 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: looking for participants now, as of the sponsor's last update on 23 Jan 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Alyssa Voelker-Christy (+1-817-948-5219, alyssav@insightec.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Events (time frame: 12 Months). 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 23 Jan 2026.