NCT05231785 · Phase 1 · RECRUITING
A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD
This trial is testing ALN-APP, a drug injected directly into the spinal fluid that is designed to reduce production of amyloid precursor protein in the brain. It is focused on people with early-onset Alzheimer's disease who have mild symptoms. This is a Phase 1 trial, meaning the primary goal is to understand safety and dosing in humans — it is not yet known whether the drug works.
Eligibility 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
The sponsor's own eligibility wording, lightly reformatted. The study team makes the final eligibility decision — worth discussing with your doctor.
Eligibility criteria as of 2026-03-31