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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

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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