Gene therapy slows Parkinson progression
- Trial ID
- NCT07216066
- Official Title
- First-in-Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single Ascending Doses of Intrathecally Administered ALN-SNCA in Participants With Early Parkinson's Disease
- Goal
- Gene therapy slows Parkinson progression
- Phase
- PHASE1
- Status
- RECRUITING
- Sponsor
- Regeneron Pharmaceuticals
- Study Type
- INTERVENTIONAL
- Enrollment
- 46 participants
- Conditions
- Parkinsons Disease
- Interventions
- ALN-SNCA, Placebo
Summary For Families
The goal is to test whether lowering production of alpha-synuclein in the central nervous system is safe and biologically active in people with early Parkinson's, since alpha-synuclein buildup is linked to disease progression. The approach gives single ascending intrathecal doses of ALN-SNCA into the cerebrospinal fluid; ALN-SNCA is an RNA-based drug designed to reduce SNCA (alpha-synuclein) production in brain cells, and it is studied for safety, drug levels, and biological effects rather than immediate symptom relief, so it does not directly replace or change how levodopa works. The trial is enrolling people aged 50 to 80 with Parkinson's diagnosed within the last 4 years, who are either not yet on dopaminergic meds or have been on a stable oral dopaminergic regimen for at least 3 months, and who can safely undergo lumbar puncture and brain MRI; people with major medical issues, prior gene/cell or surgical PD treatments, or bleeding risks are excluded.
Locations
- Montreal Neurological Institute and Hospital, Montreal, Quebec, Canada
- Center for Human Drug Research, Leiden, South Holland, Netherlands
Frequently Asked Questions
- What is this trial testing?
- This trial is studying ALN-SNCA. The goal is to test whether lowering production of alpha-synuclein in the central nervous system is safe and biologically active in people with early Parkinson's, since alpha-synuclein buildup is linked to disease progression. The approach gives single ascending intrathecal doses of ALN-SNCA into the cerebrospinal fluid; ALN-SNCA is an RNA-based drug designed to reduce SNCA (alpha-synuclein) production in brain cells, and it is studied for safety, drug levels, and biological effects rather than immediate symptom relief, so it does not directly replace or change how levodopa works. The trial is enrolling people aged 50 to 80 with Parkinson's diagnosed within the last 4 years, who are either not yet on dopaminergic meds or have been on a stable oral dopaminergic regimen for at least 3 months, and who can safely undergo lumbar puncture and brain MRI; people with major medical issues, prior gene/cell or surgical PD treatments, or bleeding risks are excluded.
- Who can participate?
- Participants must be between 50 Years and 80 Years.
- Where is this trial located?
- This trial is recruiting at 2 locations.
- 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 6 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.