A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD

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.

Interventions

• ALN-APP · DRUG

  ALN-APP will be administered intrathecally (IT)

• Placebo · DRUG

  Placebo will be administered IT

Published eligibility criteria

Verbatim from the registry. Only the site can decide how these apply to a specific patient.

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

Sites (8)

• Clinical Trial Site, La Jolla, California · COMPLETED
• Clinical Trial Site, Indianapolis, Indiana · RECRUITING
• Clinical Trial Site, Montreal, Canada · RECRUITING
• Clinical Trial Site, Toronto, Canada · RECRUITING
• Clinical Trial Site, Amsterdam, Netherlands · RECRUITING
• Clinical Trial Site, Groningen, Netherlands · COMPLETED
• Clinical Trial Site, Huntley Street, United Kingdom · RECRUITING
• Clinical Trial Site, Sheffield, United Kingdom · RECRUITING