Actively Recruiting

Phase 1
Age: 18Years +
All Genders
NCT05231785

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

Led by Alnylam Pharmaceuticals · Updated on 2026-05-07

60

Participants Needed

8

Research Sites

376 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • 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
Not Eligible

You will not qualify if you...

  • 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/1.73m² at Screening
  • Has recently received an investigational agent
  • Has recent treatment with amyloid-targeting antibody
  • Other protocol defined inclusion/exclusion criteria apply

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 8 locations

1

Clinical Trial Site

La Jolla, California, United States, 92037

Completed

2

Clinical Trial Site

Indianapolis, Indiana, United States, 46202

Actively Recruiting

3

Clinical Trial Site

Montreal, Canada, H3A 2B4

Actively Recruiting

4

Clinical Trial Site

Toronto, Canada, M3B 2S7

Actively Recruiting

5

Clinical Trial Site

Amsterdam, Netherlands, 1081 BT

Actively Recruiting

6

Clinical Trial Site

Groningen, Netherlands, 9713 GG

Completed

7

Clinical Trial Site

Huntley Street, United Kingdom, WC1E 6AG

Actively Recruiting

8

Clinical Trial Site

Sheffield, United Kingdom, S10 2JF

Actively Recruiting

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Research Team

A

Alnylam Clinical Trial Information Line

CONTACT

A

Alnylam Clinical Trial Information Line

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

TRIPLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

3

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