Testing Blood Brain Barrier Disruption for parkinsons disease
Official title: MR-guided Focused Ultrasound Plus GCase
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
Interventions
Blood Brain Barrier Disruption - Functional
Delivery of GCase across the BBB to form a disease modifying strategy for patients with Parkinson's Disease
Canadian Sites (1)
Sunybrook Research Institute
Toronto, Ontario, Canada
Eligibility Criteria
See who this study is looking for22 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +Hoehn and Yahr Stage 1-3 on PD medication
- +American Society of Anesthesiologists (ASA) score 1-3
- +Men or women between age 35 and 80 years, inclusive.
- +Able and willing to give informed consentInformed consentThe process of being told what taking part involves, then choosing freely.Read more →.
- +Diagnosis of PD satisfying MDS Clinical Diagnostic Criteria for PD
- +At least 2 years from initial diagnosis
- +Dopaminergic deficit by a positive DAT SPECT scan
- +On stable regimen of PD medications for at least 90 days prior to the study
- +Harbor at least one GBA mutation if enrolledEnrolmentThe number of participants a study plans to include, or has included.Read more → in the GBA PD armArmOne of the groups in a study, each receiving something different.Read more → or two wild-type GBA alleles if enrolled in the idiopathic PD arm
Exclusion
- −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)
- −Contraindication to GCase enzyme therapy, specifically previous hypersensitivity reaction to GCase enzyme therapy
- −Insulin-dependent diabetes mellitus that is not well-controlled or that in the InvestigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more →'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
- −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.
- −Parkinsonism plus symptoms, secondary parkinsonism
- −Previous neurosurgical procedure for PD
- −Positive pregnancy test (for pre-menopausal women).
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
This study is not organised into the usual phase numbers — that is common for device, behavioural, and diagnostic research.
What is being given or done in this study: Blood Brain Barrier Disruption - Functional.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
- armsInterventionsModule.interventions[].name
How the study is run
Groups are assigned by the study team using set rules, rather than by chance.
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
There are 2 groups in this study.
From the trial registry
Built from this field:
- armsInterventionsModule.armGroups[].type
Who the study is looking for
The study lists an age range of 35 years to 80 years.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 14 people.
The study is currently expected to finish around December 2028.
The main measurement is taken over: 12 Months.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Adverse EventsAdverse eventAny medical problem that happens during a study, whether or not the treatment caused it.Read more → — measured over 12 Months.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT05565443 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About Blood Brain Barrier Disruption - FunctionalDevice
Delivery of GCase across the BBB to form a disease modifying strategy for patients with Parkinson's Disease
From the trial registry — its own words, unedited.
What a device is here: A physical instrument, implant, app or piece of equipment being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: an age range of 35 years to 80 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
Groups are assigned by the study team using set rules, rather than by chance.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists an age range of 35 years to 80 years.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: 12 Months.
The study as a whole is currently expected to finish around 2028-12-31.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 14 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists one location: Toronto, Ontario, Canada.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
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.
Worth passing on?
Save it to the Care Package and send it to them, or to their doctor, in one message. Whether they follow it up is theirs to decide.
Review the Care PackageThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.