Brain injection clears sticky protein
- Trial ID
- NCT07157345
- Official Title
- A Study on the Safety, Tolerability, and Efficacy of PDR-001 Injection for Bilateral Stereotactic Subthalamic Nucleus (STN) Clearance of α-synuclein
- Goal
- Brain injection clears sticky protein
- Phase
- PHASE1
- Status
- RECRUITING
- Sponsor
- Ruijin Hospital
- Study Type
- INTERVENTIONAL
- Enrollment
- 12 participants
- Conditions
- Parkinson Disease (PD)
- Interventions
- PDR001
Summary For Families
The goal is to see if injecting PDR-001 directly into both subthalamic nuclei can safely reduce aggregated alpha-synuclein, the sticky protein linked to neuron damage in Parkinson's, and to look for early signs that clearing it might help symptoms or slow progression. The approach is a stereotactic neurosurgical injection of PDR-001 into the STN to target and promote removal of misfolded alpha-synuclein; it is not a dopamine replacement like levodopa, so it would be given alongside standard Parkinson's medications rather than replacing them. Eligible participants are adults 40 to 65 years old with early PD diagnosed within the past 5 years and Hoehn and Yahr stage 2 or less in the off state. People with prior brain surgery, significant cognitive impairment or depression, clotting problems, abnormal liver or kidney tests, or active viral infections are excluded.
Locations
- Ruijin hospital, Shanghai, Shanghai Municipality, China
Frequently Asked Questions
- What is this trial testing?
- This trial is studying PDR001. The goal is to see if injecting PDR-001 directly into both subthalamic nuclei can safely reduce aggregated alpha-synuclein, the sticky protein linked to neuron damage in Parkinson's, and to look for early signs that clearing it might help symptoms or slow progression. The approach is a stereotactic neurosurgical injection of PDR-001 into the STN to target and promote removal of misfolded alpha-synuclein; it is not a dopamine replacement like levodopa, so it would be given alongside standard Parkinson's medications rather than replacing them. Eligible participants are adults 40 to 65 years old with early PD diagnosed within the past 5 years and Hoehn and Yahr stage 2 or less in the off state. People with prior brain surgery, significant cognitive impairment or depression, clotting problems, abnormal liver or kidney tests, or active viral infections are excluded.
- Who can participate?
- Participants must be between 40 Years and 65 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 Phase 1 trial is estimated to last approximately 3 years and 3 months.
- Will I get the real drug or a placebo?
- It depends on the trial design. Some Parkinson's trials are placebo controlled, meaning a portion of participants get an inactive comparison while others get the experimental treatment. Some use crossover designs so everyone eventually receives the active treatment at some point. Observational trials do not use a placebo at all because they are not testing a new treatment. The consent form for any specific trial spells out the design and your odds of being assigned to placebo before you enroll.
- How often will I need to visit the study site?
- Visit frequency varies by trial. Many Parkinson's studies require an in person visit every 4 to 12 weeks during the active treatment phase, with shorter or longer gaps depending on the design. The site coordinator can give you the full visit schedule before you sign anything, so you know what the time commitment looks like.
- 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.
- Will travel or parking be reimbursed?
- Many trials reimburse for parking, mileage, and sometimes lodging if the site is far from your home. Reimbursement policies vary by sponsor and site. When you contact the trial team, ask specifically what is covered and how reimbursement is processed.
- 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.