The purpose of the study is to evaluate the effect of mivelsiran in adult participants with early-stage DS-AD and to characterize the safety, tolerability, and pharmacodynamics (PD) of mivelsiran. The study will be conducted over 2 periods: a 24-month double-blind period and an optional 12-month open-label treatment extension (OLE) period. The estimated duration of study participation, inclusive of screening, treatment, and additional safety follow-up, is up to 39 months.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
58
Mivelsiran will be administered intrathecally
Placebo will be administered intrathecally
Clinical Study Site
Maitland, Florida, United States
Clinical Study Site
Naples, Florida, United States
Double-Blind Period: Change from baseline in brain amyloid burden measured in centiloids (CLs)
Time frame: Up to 24 months
Double-Blind Period: Change from baseline in APP protein concentration in cerebrospinal fluid (CSF)
Time frame: Up to 24 months
Double-Blind Period: Change from baseline in the concentration of amyloid beta proteins in CSF
Time frame: Up to 24 months
Double-Blind Period: Change from baseline in the concentration of tau proteins in plasma
Time frame: Up to 24 months
Double-Blind Period: Change from baseline on a cognitive scale
Time frame: Up to 24 months
Double-blind Period and Open-label Extension (OLE) Period: Frequency of adverse events (AEs)
Time frame: Up to 36 months
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