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

Conditions

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

Eligibility Criteria

Sex

ALL

Age

40 Years - 55 Years

Healthy Volunteers

Not accepted

Interventions

• Mivelsiran — DRUG
  Mivelsiran will be administered intrathecally
• Placebo — DRUG
  Placebo will be administered intrathecally

Study Details

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.

Key Dates

First listed

Jun 9, 2026

Start date

Aug 17, 2026

Status verified

Sep 2026

Primary completion

May 18, 2030

Completion

Jul 23, 2031

Study Design

Enrollment

58 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Mivelsiran (Treatment Group A)
  Participants will be administered multiple low doses of mivelsiran.
• Experimental: Mivelsiran (Treatment Group B)
  Participants will be administered multiple high doses of mivelsiran.
• Placebo Comparator: Placebo (Treatment Group C)
  Participants will be administered multiple doses of placebo.

Primary Outcome Measure

Double-Blind Period: Change from baseline in brain amyloid burden measured in centiloids (CLs) [ Time Frame: Up to 24 months ]

Central Contacts

• Alnylam Clinical Trial Information Line
  1-877-ALNYLAM
  [email protected]
• Alnylam Clinical Trial Information Line
  1-877-256-9526
  [email protected]

Locations (2)

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