Status: Not yet recruiting
A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)
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.
Participants needed: 58
Trial details
Phase: Phase 2Age: 40-55Biological sex: AllType: InterventionalSponsor: Alnylam PharmaceuticalsUpdated: Jun 9, 2026Locations: 2
Eligibility criteria
Clinical diagnosis of Down-Syndrome (DS) associated with trisomy 21 [+3]
Has severe intellectual disability (ID) [+6]