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

Last updated: February 3, 2025
Sponsor: Alnylam Pharmaceuticals
Overall Status: Active - Recruiting

Phase

1

Condition

N/A

Treatment

ALN-APP

Placebo

Clinical Study ID

NCT05231785
ALN-APP-001
2023-508363-79-00
2021-003198-74
  • Ages > 18
  • All Genders

Study 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

Inclusion

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 StateExamination (MMSE) >20

Exclusion

Exclusion Criteria:

  • Has Non-Alzheimer's disease dementia

  • Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2×upperlimit 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 Design

Total Participants: 60
Treatment Group(s): 2
Primary Treatment: ALN-APP
Phase: 1
Study Start date:
February 04, 2022
Estimated Completion Date:
March 01, 2029

Connect with a study center

  • Clinical Trial Site

    Kelowna, British Columbia
    Canada

    Site Not Available

  • Clinical Trial Site

    Aurora, Ontario
    Canada

    Site Not Available

  • Clinical Trial Site

    Toronto, Ontario
    Canada

    Active - Recruiting

  • Clinical Trial Site

    Montréal, Quebec
    Canada

    Active - Recruiting

  • Clinical Trial Site

    Amsterdam,
    Netherlands

    Active - Recruiting

  • Clinical Trial Site

    Groningen,
    Netherlands

    Site Not Available

  • Clinical Trial Site

    London,
    United Kingdom

    Active - Recruiting

  • Clinical Trial Site

    Sheffield,
    United Kingdom

    Active - Recruiting

  • Clinical Trial Site

    San Diego, California 92037
    United States

    Site Not Available

  • Clinical Trial Site

    Indianapolis, Indiana 46202
    United States

    Active - Recruiting

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