A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD
Recruiting now · Phase 1 · Has a placebo group
Conditions studied: Early-Onset Alzheimer Disease
In brief
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.
Key facts
- Study ID
- NCT05231785
- Run by
- Alnylam Pharmaceuticals
- People needed
- 60
- Starts
- 2022-02-04
- Expected to finish
- 2029-04-20
- Last updated by the study team
- 2026-07-15
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
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 is running
- Clinical Trial Site — Sheffield, United Kingdom (enrolling)
- Clinical Trial Site — Indianapolis, Indiana, United States (enrolling)
- Clinical Trial Site — Montreal, Canada (enrolling)
- Clinical Trial Site — Toronto, Canada (enrolling)
- Clinical Trial Site — Amsterdam, Netherlands (enrolling)
- Clinical Trial Site — Huntley Street, United Kingdom (enrolling)
- Clinical Trial Site — La Jolla, California, United States
- Clinical Trial Site — Groningen, Netherlands
Full record on ClinicalTrials.gov
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