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Clinical Trials in Spain / NCT07636811
Recruiting Phase 2

A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)

NCT07636811 · tracked via the Priya Life Science Spain tracker
Sponsor
Alnylam Pharmaceuticals
Phase
Phase 2
Started
2026-08-17
Last updated
2026-09-04

Condition(s) studied

Down Syndrome-Associated Alzheimer's Disease (DS-AD)

Investigational drug(s) / intervention(s)

Mivelsiran →Placebo

Mivelsiran: Mivelsiran will be administered intrathecally

Placebo: Placebo will be administered intrathecally

Study summary

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.

Eligibility

Sex
ALL
Min age
40 Years
Max age
55 Years
Healthy volunteers
No
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

Primary outcome measure(s)

  • Double-Blind Period: Change from baseline in brain amyloid burden measured in centiloids (CLs) — Up to 24 months

Trial sites (3)

FacilityCityRegionStatus
Clinical Study Site Maitland Florida Recruiting
Clinical Study Site Naples Florida Recruiting
Clinical Trial Site Seville Spain Recruiting

More Alnylam Pharmaceuticals trials in Spain

Zilebesiran in Patients With Hypertension Not Adequately Controlled and With Either Established Cardiovascular Disease or High Risk for Cardiovascular Disease Recruiting
A Study to Evaluate ALN-5288 in Patients With Alzheimer's Disease Recruiting
A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy Enrolling by invitation
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy Recruiting
ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis) Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07636811 on ClinicalTrials.gov ↗ ← All trials in Spain