A Phase 2, Multicenter, Randomized, Double-blind, Placebocontrolled 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)

Status

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2025-523390-42-00

Enrollment

Registered

2026-07-22

Start date

Unknown

Completion date

Unknown

Last updated

2026-07-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Early-Stage Down Syndrome-Associated Alzheimer’s Disease (DS-AD)

Brief summary

Change from baseline to Month 24 in amyloid PET as measured in CLs

Detailed description

Change from baseline to Month 24 in sAPPβ concentration in CSF, Change from baseline to Month 24 in sAPPα concentration in CSF, Change from baseline to Month 24 in concentration of Aβ42 in CSF, Change from baseline to Month 24 in concentration of p-tau217 in plasma, Change from baseline to Month 24 on mCRT

Interventions

DRUGMivelsiran

DRUGIsotonic aqueous buffer suitable for IT injection

Eligibility

Sex/Gender

All

Age

18 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Change from baseline to Month 24 in amyloid PET as measured in CLs	—

Secondary

Measure	Time frame
Change from baseline to Month 24 in sAPPβ concentration in CSF, Change from baseline to Month 24 in sAPPα concentration in CSF, Change from baseline to Month 24 in concentration of Aβ42 in CSF, Change from baseline to Month 24 in concentration of p-tau217 in plasma, Change from baseline to Month 24 on mCRT	—

Outcome results

None listed

Source: EU CTIS · Data processed: Jul 24, 2026