RecruitingDown Syndrome-Associated Alzheimer's Disease (DS-AD)
A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)
Eligible age
40–55 yrs
Accepts
All genders
Locations
3 states
Healthy volunteers
No
See if you qualify for this study
Answer a few quick questions about your location and health. Takes about a minute.
About this study
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.
Sponsor: Alnylam Pharmaceuticals
You may qualify if…
- ✓ 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
You may not qualify if…
- ✕ Has severe intellectual disability (ID)
- ✕ Has a history of DS regression disorder
- ✕ Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>2×upper limit of normal (ULN) at Screening
- ✕ Has estimated glomerular filtration rate (eGFR) \<45 mL/min/1.73m\^2 at Screening
- ✕ Has recently received an investigational agent
- ✕ Has had treatment with amyloid-targeting antibody
- ✕ Comorbidities such as obstructive sleep apnea and hypothyroidism that are not well controlled
- ✕ Note: other protocol defined inclusion / exclusion criteria apply
Where it's recruiting
Source: ClinicalTrials.gov · NCT07636811 · last updated 2026-10-13