Blood Brain Barrier Disruption - F… for Parkinson's (NCT05565443)
Slow Parkinson progression with enzyme
- 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
- Slow Parkinson progression with enzyme
- Phase
- NA
- Status
- RECRUITING
- Sponsor
- InSightec
- Study Type
- INTERVENTIONAL
- Enrollment
- 14 participants
- Conditions
- Parkinsons Disease
- Interventions
- Blood Brain Barrier Disruption - Functional
Summary For Families
The goal is to find out if it is safe and practical to deliver a replacement enzyme called recombinant glucocerebrosidase directly into a deep brain area called the putamen by using magnetic resonance imaging-guided focused ultrasound to open the brain’s protective barrier, a technique that helped in animal studies. The approach gives three treatments where the enzyme is injected into the bloodstream while focused ultrasound temporarily opens both sides of the putamen; the first four people in each group get 30 units per kilogram every two weeks and the next three get 60 units per kilogram, then everyone is followed for 12 months. The study plans to enroll 14 people total, seven with a mutation in the GBA gene and seven with typical Parkinson’s, aged 35 to 80, who have had Parkinson’s at least two years, are in early to mid-stage while on medication (stage 1 to 3), have a dopamine transporter scan that shows a deficit, and are on stable Parkinson’s medicines.
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 find out if it is safe and practical to deliver a replacement enzyme called recombinant glucocerebrosidase directly into a deep brain area called the putamen by using magnetic resonance imaging-guided focused ultrasound to open the brain’s protective barrier, a technique that helped in animal studies. The approach gives three treatments where the enzyme is injected into the bloodstream while focused ultrasound temporarily opens both sides of the putamen; the first four people in each group get 30 units per kilogram every two weeks and the next three get 60 units per kilogram, then everyone is followed for 12 months. The study plans to enroll 14 people total, seven with a mutation in the GBA gene and seven with typical Parkinson’s, aged 35 to 80, who have had Parkinson’s at least two years, are in early to mid-stage while on medication (stage 1 to 3), have a dopamine transporter scan that shows a deficit, and are on stable Parkinson’s medicines.
- 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?
- You are never charged a fee to join a legitimate clinical trial, and the study treatment and study-specific tests are provided by the trial sponsor at no charge. In the US, routine care you would receive anyway can still be billed to you or your insurance; coverage rules differ by country, and trials in the UK and Canada usually run through public health systems. Travel, parking, and time are real costs, and some trials reimburse them while others do not. Before enrolling, ask the study coordinator to explain in writing what is and is not covered.
- How long does the trial last?
- This trial is estimated to last approximately 5 years and 1 month.
- Can I leave the trial if I change my mind?
- Yes. You can withdraw from any clinical trial at any time, for any reason, without affecting your standard medical care. Trials are voluntary by law. The team may ask if you are willing to do a brief exit visit so they can collect safety information, but you are not obligated.
- Can my spouse or care partner come with me to visits?
- In most cases yes, and it is often encouraged. Care partners can help with notes, questions, and getting home safely after a long visit. Some study assessments do need to happen one on one, but care partners are usually welcome for the rest of the appointment.
- Will I get a placebo in this trial?
- There is no placebo group. Everyone receives an active treatment; the study compares one treatment against another.
- How many visits does this trial involve?
- Participants will receive three treatments and then be followed for twelve months.
- What procedures are involved in this trial?
- Based on the protocol, this trial involves: Brain surgery / implanted device (invasive). Confirm the full schedule with the study coordinator.