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 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
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
Note: other protocol defined inclusion / exclusion criteria apply
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 Details
Contition
Early-Onset Alzheimer Disease
Age
18+
Phase
PHASE1
Participants Needed
60
Est. Completion Date
Jul 1, 2027
Treatment Type
INTERVENTIONAL
Sponsor
Alnylam Pharmaceuticals
ClinicalTrials.gov NCT Identifier
NCT05231785
Study Number
ALN-APP-001
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