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
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
46
Administered per the protocol
Administered per the protocol
Center for Human Drug Research
Leiden, South Holland, Netherlands
RECRUITINGOccurrence of Treatment-Emergent Adverse Events (TEAEs)
Time frame: Through Week 80
Severity of TEAEs
Time frame: Through Week 80
Concentration of alpha (a)-synuclein protein in CSF
Time frame: Through Week 48
Change from baseline in concentration of a-synuclein protein in CSF
Time frame: Through Week 48
Concentration of ALN-SNCA in plasma
Time frame: Through Week 48
Concentration of major metabolites in plasma
Time frame: Through Week 48
Concentration of ALN-SNCA in CSF
Time frame: Through Week 48
Concentration of major metabolites in CSF
Time frame: Through Week 48
Fraction [fc] of ALN-SNCA excreted in urine
Time frame: Over 24 hours
Fc of major metabolites excreted in urine
Time frame: Over 24 hours
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