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
Plain-Language Summary
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.