RecruitingEarly-Onset Alzheimer Disease
A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD
Eligible age
18+ yrs
Accepts
All genders
Locations
2 states
Healthy volunteers
No
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About this 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.
Sponsor: Alnylam Pharmaceuticals
You may qualify if…
- ✓ 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
You may not qualify if…
- ✕ 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
Where it's recruiting
California
La Jolla
Indiana
Indianapolis
Source: ClinicalTrials.gov · NCT05231785 · last updated 2026-09-14