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)
Status: Recruiting
Location: See all (3) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 40
Maximum Age: 55
Healthy Volunteers: f
View:
• Clinical diagnosis of Down-Syndrome (DS) associated with trisomy 21
• Positive amyloid PET scan
• Cognitively stable in the opinion of the investigator
• Seizures must be well controlled with no occurrence of seizures in the 6 months prior to screening
Locations
United States
Florida
Clinical Study Site
RECRUITING
Maitland
Clinical Study Site
RECRUITING
Naples
Other Locations
Spain
Clinical Trial Site
RECRUITING
Seville
Contact Information
Primary
Alnylam Clinical Trial Information Line
clinicaltrials@alnylam.com
1-877-ALNYLAM
Backup
Alnylam Clinical Trial Information Line
clinicaltrials@alnylam.com
1-877-256-9526
Time Frame
Start Date: 2026-08-17
Estimated Completion Date: 2031-07-23
Participants
Target number of participants: 58
Treatments
Experimental: Mivelsiran (Treatment Group A)
Participants will be administered multiple low doses of mivelsiran.
Experimental: Mivelsiran (Treatment Group B)
Participants will be administered multiple high doses of mivelsiran.
Placebo_comparator: Placebo (Treatment Group C)
Participants will be administered multiple doses of placebo.
Related Therapeutic Areas
Sponsors
Leads: Alnylam Pharmaceuticals