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RecruitingPhase 2

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

Alnylam Pharmaceuticals · NCT07636811

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 40–55 · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 2
Target enrollment
58
US sites recruiting
4
Study start
August 17, 2026
Record last updated
October 13, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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.

Being studied: Mivelsiran; Placebo

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

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full 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

Recruiting US locations

Study contacts (from the official record)

  • Alnylam Clinical Trial Information Line · 1-877-ALNYLAM · clinicaltrials@alnylam.com
  • Alnylam Clinical Trial Information Line · 1-877-256-9526 · clinicaltrials@alnylam.com
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A Study to Evaluate Mivelsiran in Adult Participants With… (NCT07636811) | Bored Studies