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A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)
Sponsor: Alnylam Pharmaceuticals
Summary
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.
Official title: A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study, to Evaluate Efficacy, Safety, Tolerability, and Pharmacodynamics of Intrathecally Administered Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)
Key Details
Gender
All
Age Range
40 Years - 55 Years
Study Type
INTERVENTIONAL
Enrollment
58
Start Date
2026-07-01
Completion Date
2031-07-23
Last Updated
2026-06-09
Healthy Volunteers
No
Interventions
Mivelsiran
Mivelsiran will be administered intrathecally
Placebo
Placebo will be administered intrathecally
Locations (2)
Clinical Study Site
Maitland, Florida, United States
Clinical Study Site
Naples, Florida, United States