A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Mivelsiran, Placebo.
- Who it may be relevant to
- Registry conditions: Down Syndrome-Associated Alzheimer's Disease (DS-AD). Basic parameters: 40 years — 55 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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)
Overview
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.
Interventions
- Drug Mivelsiran
Mivelsiran will be administered intrathecally - Drug Placebo
Placebo will be administered intrathecally
Primary outcome measures
- Double-Blind Period: Change from baseline in brain amyloid burden measured in centiloids (CLs) [Time frame: Up to 24 months]
Secondary outcome measures (5)
- Double-Blind Period: Change from baseline in APP protein concentration in cerebrospinal fluid (CSF) [Time frame: Up to 24 months]
- Double-Blind Period: Change from baseline in the concentration of amyloid beta proteins in CSF [Time frame: Up to 24 months]
- Double-Blind Period: Change from baseline in the concentration of tau proteins in plasma [Time frame: Up to 24 months]
- Double-Blind Period: Change from baseline on a cognitive scale [Time frame: Up to 24 months]
- Double-blind Period and Open-label Extension (OLE) Period: Frequency of adverse events (AEs) [Time frame: Up to 36 months]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 2 centers
- Clinical Study Site — Maitland
- Clinical Study Site — Naples
Identifiers
NCT: NCT07636811 · ALN-APP-003 · 2025-523390-42-00