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A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD

A Randomized, Double-blind, Placebo-controlled Single Ascending Dose and Open-label Multi-dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ALN-APP in Adult Patients With Early-onset Alzheimer's Disease (EOAD)

Who can join

Ages 18 and older · All sexes

Inclusion Criteria:

* Has mild cognitive impairment or mild dementia due to EOAD
* Has Clinical Dementia Rating (CDR) global score 0.5 or 1.0 and Mini Mental State Examination (MMSE) \>20

Exclusion Criteria:

* Has Non-Alzheimer's disease dementia
* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>2×upper limit of normal (ULN)
* Has estimated glomerular filtration rate (eGFR) \<45 mL/min/1.73m\^2 at Screening
* Has recently received an investigational agent
* Has recent treatment with amyloid-targeting antibody

Note: other protocol defined inclusion / exclusion criteria apply

About the study

The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of a single dose and multiple doses of ALN-APP administered by intrathecal (IT) injection in adult patients with early-onset Alzheimer's Disease (EOAD). Maximum treatment duration for Part A: single dose. Maximum treatment duration for Part B: 12 months.

What is being tested

• ALN-APP (drug)
• Placebo (drug)

Sponsor: Alnylam Pharmaceuticals · Participants: 60 · Started: Feb 4, 2022

Contact the study team

• Alnylam Clinical Trial Information Line · Phone: 1-877-ALNYLAM
• Alnylam Clinical Trial Information Line · Phone: 1-877-256-9526

Official record on ClinicalTrials.gov — NCT05231785

Locations in the U.S.

Conditions

• Alzheimer's Disease

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.