A Phase 2, Multicenter, Randomized, Double-blind, Placebocontrolled 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)

Condition studied

Early-Stage Down Syndrome-Associated Alzheimer’s Disease (DS-AD)

Investigational medicinal product(s)

Every trial of Mivelsiran across our registers →

Eligibility

Primary endpoint

Change from baseline to Month 24 in amyloid PET as measured in CLs

Endpoint detail

Change from baseline to Month 24 in sAPPβ concentration in CSF, Change from baseline to Month 24 in sAPPα concentration in CSF, Change from baseline to Month 24 in concentration of Aβ42 in CSF, Change from baseline to Month 24 in concentration of p-tau217 in plasma, Change from baseline to Month 24 on mCRT