Trial intelligence / Profile preview

Mivelsiran for early-stage Down syndrome-associated Alzheimer's disease

2025-523390-42-00Phase 2Authorised

Sponsor
Alnylam Pharmaceuticals
Start
Aug 2026
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Countries
7
01

Overview

Phase 2, randomised, double-blind trial in dementia due to Down syndrome (DS-AD) comparing Mivelsiran with Placebo (2 arms). Planned start Aug 2026; status: authorised.

02

Drugs

mivelsiran
03

Conditions

dementia due to Down syndrome (DS-AD)

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