NCT05565443Active, not recruiting
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
Source: ClinicalTrials.gov · Last updated 4 Nov 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Active, not recruiting
- Study type
- Interventional
- Enrollment
- 14 (estimated)
- First submitted
- 7 Sept 2022
- Start date
- 30 Nov 2022
- Primary completion date
- 1 Nov 2026
- Lead sponsor
- InSightec
- Diseases
- Parkinson disease
- NCT number
- NCT05565443
What you commit to
- Study type: Interventional
- Planned participants: 14
- From 30 Nov 2022 to the primary completion date, 1 Nov 2026
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Patient receiving bevacizumab (Avastin) therapy
- Contraindication to GCase enzyme therapy, specifically previous hypersensitivity reaction to GCase enzyme therapy
- Previous neurosurgical procedure for PD
Other illness
- 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.
- Subjects with evidence of cranial or systemic infection.
Other
- Positive pregnancy test (for pre-menopausal women).
- 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
- Cerebral or systemic vasculopathy.
- Documented cerebral infarction within the past 12 months or TIA in the past 1 month.
- Parkinsonism plus symptoms, secondary parkinsonism
Who the study is looking for
- 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
- 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
Age in the registry: 35 years to 80 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Sunybrook Research Institute Coordinator of this site? Confirm its status | Toronto | Canada | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.