A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD
Recruiting · NCT05231785 · Interventional (participants receive a specific treatment) · Lead sponsor: Alnylam Pharmaceuticals
View the official record on ClinicalTrials.gov →Interventions studied
ALN-APPPlacebo
What this trial is about
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.
Who can take part
Age range
18 Years and older
Sex
All (male and female)
Healthy volunteers
No - a diagnosis or condition is required
Phase
Phase 1
Study type
Interventional (participants receive a specific treatment)
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
Where it is running
8 locations listed across 2 US states.
- Clinical Trial Site - La Jolla, California, United States
- Clinical Trial Site - Indianapolis, Indiana, United States
- Clinical Trial Site - Amsterdam, Netherlands
- Clinical Trial Site - Groningen, Netherlands
- Clinical Trial Site - Huntley Street, United Kingdom
- Clinical Trial Site - Montreal, Canada
- Clinical Trial Site - Sheffield, United Kingdom
- Clinical Trial Site - Toronto, Canada