Tests ALN-APP, an injected drug, in early-onset Alzheimer's disease
Tests the safety of ALN-APP, given by spinal injection, in adults with early-onset Alzheimer's.
Plain-language title generated from the registry record
Registry title: A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD
Key facts
- Where
- Montreal and Toronto, CanadaSee the 2 sites
- Phase
- Phase 1
Where in Canada
- MontrealMontreal, CanadaTaking people
- TorontoToronto, CanadaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Alnylam Pharmaceuticals
- Trial code
- NCT05231785EU 2023-508363-79-00
- Record updated
- 20 October 2026
- Original description
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.Read full original descriptionShow less
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)
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.
Inclusion: 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: Has recently received an investigational agent; Has recent treatment with amyloid-targeting antibody; Note: other protocol defined inclusion / exclusion criteria apply; 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.