Mivelsiran Tested in Early Down Syndrome-Associated Alzheimer's Disease
Tests mivelsiran against placebo in adults with early Down syndrome-associated Alzheimer's disease.
Plain-language title generated from the registry record
Registry title: A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)
Key facts
- Where
- Amsterdam, NetherlandsSee the site
- Phase
- Phase 2
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults aged 40 to 55 with Down syndrome and early-stage Alzheimer's disease. It tests a drug called mivelsiran against placebo.
- Age
- 40 Years to 55 Years
- Sex
- All sexes can join.
- Disease stage
- Early-stage Alzheimer's disease linked to Down syndrome (trisomy 21). Thinking skills must be stable in the investigator's opinion.
- Earlier treatment
- People who recently received an investigational agent or had treatment with an amyloid-targeting antibody cannot join.
- Biomarker or test result
- A positive amyloid PET scan.
Main reasons for not taking part
- Severe intellectual disability
- History of Down syndrome regression disorder
- ALT or AST above 2 times the upper limit of normal at screening
- eGFR below 45 mL/min/1.73m^2 at screening
- Seizures in the 6 months before screening, or poorly controlled sleep apnea or hypothyroidism
This trial only enrols participants with Down syndrome. The registry sentence this rests on: Clinical diagnosis of Down-Syndrome (DS) associated with trisomy 21
Read by machine on 13 November 2026.
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 40 and 55 years old?
Source: NCT07636811, who can take part
Where in Netherlands
- Amsterdam UMC StichtingAmsterdam, NetherlandsNot yet recruiting
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Alnylam Pharmaceuticals
- Trial code
- NCT07636811EU 2025-523390-42-00
- Record updated
- 13 October 2026
- Original description
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.Read full original descriptionShow less
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)
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.
Inclusion: 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: Has recently received an investigational agent; Has had treatment with amyloid-targeting antibody; 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 severe intellectual disability (ID); Comorbidities such as obstructive sleep apnea and hypothyroidism that are not well controlled; Note: other protocol defined inclusion / exclusion criteria apply.