Mivelsiran for early Down syndrome–associated Alzheimer's disease in adults

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This phase 2 trial tests mivelsiran versus placebo in adults with Down syndrome who have early-stage Alzheimer disease. It includes a 24-month double-blind period followed by an optional 12-month open-label extension to observe longer-term effects and safety. Participants will have confirmed DS, an amyloid PET positive scan, stable cognition, and well-controlled seizures, among other criteria. The study will measure safety, tolerability, and pharmacodynamics to understand how the drug works in this population.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* 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

Exclusion Criteria:

* 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 running

• Clinical Trial Site
  Maitland, Florida, United States Recruiting
• Clinical Trial Site
  Naples, Florida, United States Recruiting
• Clinical Trial Site
  Indianapolis, Indiana, United States Recruiting
• Clinical Trial Site
  St Louis, Missouri, United States Recruiting
• Clinical Trial Site
  Barcelona, Spain Recruiting
• Clinical Trial Site
  Dos Hermanas, Spain Recruiting

Study contacts

• Study coordinator: Alnylam Clinical Trial Information Line
• Email: clinicaltrials@alnylam.com
• Phone: 1-877-ALNYLAM

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.