Actively Recruiting
First-in-Human Study of ALN-SNCA in Adult Participants With Early Parkinson's Disease (PD)
Led by Regeneron Pharmaceuticals · Updated on 2026-04-14
46
Participants Needed
2
Research Sites
182 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This study is researching an experimental drug called ALN-SNCA (called "study drug"). The study is focused on people with early Parkinson's disease, a disorder of the nervous system that affects movement. Parkinson's disease is caused by a gradual loss of nerve cells in the brain, especially those due to the harmful build-up of a protein called α-synuclein. The aim of the study is to see if the study drug is safe and tolerated well enough to continue testing it in future studies and what side effects may happen from taking the study drug. The study is looking at several other research questions, including: * Whether the study drug can lower the level of α-synuclein protein in the Cerebrospinal Fluid (also referred to as "CSF", the fluid that surrounds the brain and spinal cord) * How much study drug is in the blood, urine, and CSF at different times * Compatible research to better understand the study drug (ALN-SNCA) and Parkinson's disease, including (but not limited to), whether the study drug can slow down the progression of Parkinson's disease symptoms
CONDITIONS
Official Title
First-in-Human Study of ALN-SNCA in Adult Participants With Early Parkinson's Disease (PD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of Parkinson's disease according to Movement Disorder Society criteria with bradykinesia plus one other cardinal sign
- Diagnosis of Parkinson's disease for 4 years or less at screening
- Currently not on standard Parkinson's therapy and not expected to need it within 6 months, or on stable oral dopaminergic therapy for at least 3 months with no expected dose changes within 6 months
- Body mass index 35 kg/m2 or less at screening
You will not qualify if you...
- Medical history of Parkinsonian syndromes other than Parkinson's disease
- Significant cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, hematological, psychiatric, or neurological disease other than Parkinson's
- History of brain or spinal disease/injury interfering with lumbar puncture or cerebrospinal fluid circulation
- Contraindications to brain MRI
- Allergy or intolerance to lidocaine anesthetic
- History of intolerance to intrathecal injections
- Current bleeding disorders increasing risk of bleeding with lumbar puncture
- Previous gene therapy, cell therapy, or surgical treatments including deep brain stimulation for Parkinson's disease
- Other protocol-defined criteria apply
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 2 locations
1
Montreal Neurological Institute and Hospital
Montreal, Quebec, Canada, H3A 2B4
Actively Recruiting
2
Center for Human Drug Research
Leiden, South Holland, Netherlands, 2333 CL
Actively Recruiting
Research Team
C
Clinical Trials Administrator
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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