A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD
About the study
The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single intrathecal (IT) doses of ALN-APP in adult patients with early-onset Alzheimer's Disease (EOAD). Maximum treatment duration for Part A: single dose. Maximum treatment for Part B: 12 months.
Who can take part
You may be eligible to participate in the study if you meet the following criteria:
INCLUSION 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
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
Study Locations
Enter your ZIP code/Postal code/PIN code to locate study sites near you:
How to Apply
Contact the study center to learn if this study is a good match for you.
Study’s details
Contition
Early-Onset Alzheimer Disease
Age (in years)
18+
Phase
Phase 1
Participants needed
60
Est. Completion Date
Jul 31, 2025
Treatment type
Interventional
Sponsor
Alnylam Pharmaceuticals
ClinicalTrials.gov identifier
NCT05231785
Study number
ALN-APP-001
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