NCT05231785PHASE1Recruiting
A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD
Alnylam Pharmaceuticals
Start Date
2/4/2022
Completion Date
4/20/2029
Summary
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.
Eligibility Criteria
Age Range: 18 years to No maximum
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
Interventions
DRUG
ALN-APP
DRUG
Placebo
Conditions
Early-Onset Alzheimer Disease
Locations
Clinical Trial Site
La Jolla, California 92037
United States
Clinical Trial Site
Indianapolis, Indiana 46202
United States
Clinical Trial Site
Montreal, H3A 2B4
Canada
Clinical Trial Site
Toronto, M3B 2S7
Canada
Clinical Trial Site
Amsterdam, 1081 BT
Netherlands
Clinical Trial Site
Groningen, 9713 GG
Netherlands
Clinical Trial Site
Huntley Street, WC1E 6AG
United Kingdom
Clinical Trial Site
Sheffield, S10 2JF
United Kingdom