Skip to content

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

Early-Onset Alzheimer Disease

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.

null

Participation Requirements

  • Sex:

    ALL

  • Eligible Ages:

    18 and up

Participation Criteria

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

Study Location

Clinical Trial Site
Clinical Trial Site
Toronto, Ontario
Canada

Contact Study Team

Clinical Trial Site
Clinical Trial Site
Montréal, Quebec
Canada

Contact Study Team

Study Sponsored By
Alnylam Pharmaceuticals
Participants Required
More Information
Study ID: NCT05231785