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
- Rome, ItalySee the 2 sites
- Phase
- Phase 2
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 Italy
- Fondazione Policlinico Universitario Campus Bio-medico In Forma A Bbreviata FonRome, ItalyNot yet recruiting
- Ospedale Isola Tiberina - Gemelli IsolaRome, ItalyNot 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?
Trial 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.