RESEARCH / PARTICIPATIONBack to the library
← Find a study
Recruiting · registry status

Using ultrasound to help deliver an enzyme into the brain

Researchers check whether scan-guided focused ultrasound can safely help deliver an enzyme called GCase into a target brain area.

Testing a treatment or activity · Study reference: NCT05565443

Plain-language introduction written with AI from the registry; not independently checked by a clinician. Read the original details below ↓

This registry record was last updated over six months ago, or its update date is missing. Recruitment may have changed. Confirm with the team before making plans.

Open the official registry record ↗

Follow this study

Looking for volunteers

Start (reported actual date)
2022-11-30
Main measurements finished (planned)
2027-12-31
Study finished (planned)
2028-12-31

Planned dates can move. A study finishing does not tell us when a paper will be published.

No results summary has been confirmed in the registry records we imported. See connected papers below; we keep checking after recruitment ends.

Changes we have recorded
  • 2026-10-11 — recruiting

These are dates we observed a change, not necessarily the dates it happened.

Papers connected to this study

No connected paper has been found yet. The tracker checks the growing library for study identifiers and registry-linked publications.

Who can join?

Ages 35 years to 80 years · Does not accept healthy volunteers

These are starting points, not the full rules. The research team can tell you whether the study is right for your situation.

Read all the rules for taking part

Sex eligibility reported by registry: all

Inclusion Criteria: * 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 Exclusion Criteria: * 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 * 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
Full study name & original research details

Official study 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

Short title used by the registry

MR-guided Focused Ultrasound Plus GCase

Original description

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.

Further description from the registry

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 reported: Parkinsons Disease

Registry records for this study

Records are joined using registration identifiers. Titles alone do not establish that two studies are the same.

Study type
Interventional
Interventions
Blood Brain Barrier Disruption - Functional
Phases
NA
Sponsor
InSightec
Start date reported by registry
2022-11-30 (actual)

Registry updated: 2026-01-23 · Status last verified by the registry submitter: 2026-01

Registry records retrieved 2026-10-13 (UTC). Individual records may have older updates. Recruitment and eligibility must be confirmed with the study team.

Contact the research team

Public study contacts supplied to the registry. Ask whether recruitment is still open and what participation involves.

Alyssa Voelker-Christy · +1-817-948-5219 · alyssav@insightec.com

Study locations

Site status can differ from overall study status. “Status not reported” means local availability needs confirmation. Remote participation and travel arrangements must be checked with the team.

Sunybrook Research Institute

Toronto, Ontario, Canada

Recruiting

Inthuja Suthananthan · 647-687-8062 · inthuja.suthananthan@sri.utoronto.ca

Nir Lipsman, M.D., Neurosurgery

The original descriptions and participation rules come from the registry. Participation is voluntary and does not guarantee benefit.