A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)
Starting soon · Phase 2 · Has a placebo group
Conditions studied: Down Syndrome-Associated Alzheimer's Disease (DS-AD)
In brief
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.
Key facts
- Study ID
- NCT07636811
- Run by
- Alnylam Pharmaceuticals
- People needed
- 58
- Starts
- 2026-07-31
- Expected to finish
- 2031-07-23
- Last updated by the study team
- 2026-07-15
Who can join
Age: 40 and older, up to 55. Sex: any. Healthy volunteers: not accepted.
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 is running
- Clinical Study Site — Maitland, Florida, United States
- Clinical Study Site — Naples, Florida, United States
Full record on ClinicalTrials.gov
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