Restore glucocerebrosidase activity in putamen
- Trial ID
- NCT05565443
- 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
- Goal
- Restore glucocerebrosidase activity in putamen
- Phase
- NA
- Status
- RECRUITING
- Sponsor
- InSightec
- Study Type
- INTERVENTIONAL
- Enrollment
- 14 participants
- Conditions
- Parkinsons Disease
- Interventions
- Blood Brain Barrier Disruption - Functional
Plain-Language Summary
The goal is to get the enzyme glucocerebrosidase into the putamen to fix lysosomal problems that can drive alpha-synuclein buildup, potentially slowing disease processes that are linked to GBA gene mutations. The team uses MRI-guided focused ultrasound to temporarily open the blood brain barrier over both putamen areas so intravenously delivered recombinant glucocerebrosidase can reach brain cells, where it should boost GCase activity and help clear misfolded proteins; it is given in addition to a person’s usual Parkinson’s medications, not as a replacement for levodopa. Adults 35 to 80 years old with Parkinson’s for at least 2 years, Hoehn and Yahr stage 1,3 on medication, a positive DAT SPECT, and stable PD meds are eligible, with one arm for people who carry at least one GBA mutation and another arm for those with two normal GBA alleles.
Locations
- Sunybrook Research Institute, Toronto, Ontario, Canada
Frequently Asked Questions
- What is this trial testing?
- This trial is studying Blood Brain Barrier Disruption - Functional. The goal is to get the enzyme glucocerebrosidase into the putamen to fix lysosomal problems that can drive alpha-synuclein buildup, potentially slowing disease processes that are linked to GBA gene mutations. The team uses MRI-guided focused ultrasound to temporarily open the blood brain barrier over both putamen areas so intravenously delivered recombinant glucocerebrosidase can reach brain cells, where it should boost GCase activity and help clear misfolded proteins; it is given in addition to a person’s usual Parkinson’s medications, not as a replacement for levodopa. Adults 35 to 80 years old with Parkinson’s for at least 2 years, Hoehn and Yahr stage 1,3 on medication, a positive DAT SPECT, and stable PD meds are eligible, with one arm for people who carry at least one GBA mutation and another arm for those with two normal GBA alleles.
- Who can participate?
- Participants must be between 35 Years and 80 Years.
- Where is this trial located?
- This trial is recruiting at 1 location.
- Does it cost anything to join?
- No. There is no cost to participate. Study-related care and treatment are provided at no charge.
- How long does the trial last?
- This trial is estimated to last approximately 5 years and 1 month.