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

NCT ID
NCT07636811
Registry status
Recruiting
Study type
Interventional
Phase
Phase 2
Sponsor
Alnylam Pharmaceuticals
Conditions
Down Syndrome-Associated Alzheimer's Disease (DS-AD)

Overview

ClinicalTrials.gov lists this study as Recruiting.

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)

Enrollment: 58 participants (Estimated).

What is being studied

  • Drug: Mivelsiran
  • Drug: Placebo

Who may be eligible

Ages
40 Years to 55 Years
Sex
All
Healthy volunteers accepted
No

ClinicalTrials.gov lists the following 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

Study locations

10 study locations are listed.

Recruiting site status is explicitly provided for 10 locations; 10 are listed as Recruiting.

Countries: Spain, United States

  • Florida, United States: 2
  • Indiana, United States: 1
  • Missouri, United States: 1

Sample of listed locations

  • Clinical Trial Site — Barcelona, Spain — Recruiting
  • Clinical Trial Site — Dos Hermanas, Spain — Recruiting
  • Clinical Trial Site — Madrid, Spain — Recruiting
  • Clinical Trial Site — Pamplona, Spain — Recruiting
  • Clinical Trial Site — Seville, Spain — Recruiting
  • Clinical Trial Site — Valencia, Spain — Recruiting
  • Clinical Trial Site — Maitland, Florida, 32751, United States — Recruiting
  • Clinical Trial Site — Naples, Florida, 34105, United States — Recruiting
  • Clinical Trial Site — Indianapolis, Indiana, 46037, United States — Recruiting
  • Clinical Trial Site — St Louis, Missouri, 63110, United States — Recruiting

Study details

Start date
August 17, 2026
Primary completion date
May 18, 2030
Completion date
July 30, 2031
First posted
June 9, 2026

Source and update information

Source
ClinicalTrials.gov
NCT ID
NCT07636811
Registry last updated
October 13, 2026
QualifiedStudies registry data refreshed
October 19, 2026
QualifiedStudies record fetched
October 19, 2026

View NCT07636811 on ClinicalTrials.gov

Trial information can change. Confirm current study details with the study team or the registry.

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