A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy

Last updated: April 15, 2025
Sponsor: Alnylam Pharmaceuticals
Overall Status: Active - Recruiting

Phase

2

Condition

Alzheimer's Disease

Amyloidosis

Treatment

Placebo

ALN-APP

Clinical Study ID

NCT06393712
ALN-APP-002
2023-510137-29-00
  • Ages > 30
  • All Genders

Study Summary

The purpose of the study is to evaluate the effect of ALN-APP on measures of CAA disease progression and to characterize the safety, tolerability, and pharmacodynamics (PD) of ALN-APP in adult patients with sporadic CAA (sCAA) and Dutch-type CAA (D-CAA). The study will be conducted over 2 periods: a 24-month double-blind treatment period and an optional 18-month open-label extension (OLE) period. The estimated duration of study participation, inclusive of screening, treatment, and additional safety follow-up, is up to 50 months.

Eligibility Criteria

Inclusion

Inclusion Criteria (sporadic CAA patients):

  • Is 50 years or older

  • Has probable CAA per the Boston Criteria Version 2.0

Inclusion Criteria (Dutch-type CAA patients):

  • Is 30 years or older

  • Has known E693Q amyloid precursor protein (APP) gene mutation for Dutch-type CAA

Exclusion

Exclusion Criteria:

  • Moderate or severe stage Alzheimer's disease (AD) or significant cognitiveimpairment (CI)

  • Has a history of previous clinical intracerebral hemorrhage (ICH) with onset lessthan 90 days prior to anticipated randomization in the study

  • Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >3×upperlimit of normal (ULN) at Screening

  • Has estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m^2 at Screening

  • Has recently received an investigational agent

  • Has had treatment with amyloid-targeting antibody

Note: other protocol defined inclusion / exclusion criteria apply

Study Design

Total Participants: 200
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 2
Study Start date:
May 17, 2024
Estimated Completion Date:
November 01, 2029

Connect with a study center

  • Clinical Trial Site

    Heidelberg,
    Australia

    Active - Recruiting

  • Clinical Trial Site

    Herston,
    Australia

    Active - Recruiting

  • Clinical Trial Site

    Nedlands,
    Australia

    Active - Recruiting

  • Clinical Trial Site

    Parkville,
    Australia

    Active - Recruiting

  • Clinical Trial Site

    Calgary, Alberta
    Canada

    Active - Recruiting

  • Clinical Trial Site

    Richmond, British Columbia
    Canada

    Active - Recruiting

  • Clinical Trial Site

    Moncton, New Brunswick
    Canada

    Active - Recruiting

  • Clinical Trial Site

    Hamilton, Ontario
    Canada

    Active - Recruiting

  • Clinical Trial Site

    Ottawa, Ontario
    Canada

    Active - Recruiting

  • Clinical Trial Site

    Toronto, Ontario
    Canada

    Active - Recruiting

  • Clinical Trial Site

    Bern,
    Switzerland

    Active - Recruiting

  • Clinical Trial Site

    Geneve,
    Switzerland

    Active - Recruiting

  • Clinical Trial Site

    Denmark Hill,
    United Kingdom

    Active - Recruiting

  • Clinical Trial Site

    Glasgow,
    United Kingdom

    Active - Recruiting

  • Clinical Trial Site

    London,
    United Kingdom

    Active - Recruiting

  • Clinical Trial Site

    Sacramento, California 95816
    United States

    Active - Recruiting

  • Clinical Trial Site

    San Francisco, California 94117
    United States

    Active - Recruiting

  • Clinical Trial Site

    Jacksonville, Florida 32224
    United States

    Active - Recruiting

  • Clinical Trial Site

    Maitland, Florida 32751
    United States

    Active - Recruiting

  • Clinical Trial Site

    Naples, Florida 34105
    United States

    Active - Recruiting

  • Clinical Trial Site

    Lexington, Kentucky 40536
    United States

    Active - Recruiting

  • Clinical Trial Site

    New Orleans, Louisiana 70121
    United States

    Active - Recruiting

  • Clinical Trial Site

    Boston, Massachusetts 02114
    United States

    Active - Recruiting

  • Clinical Trial Site

    Plymouth, Massachusetts 02360
    United States

    Active - Recruiting

  • Clinical Trial Site

    Rochester, Minnesota 55905
    United States

    Active - Recruiting

  • Clinical Trial Site

    Saint Louis, Missouri 63110
    United States

    Active - Recruiting

  • Clinical Trial Site

    New York, New York 10029
    United States

    Active - Recruiting

  • Clinical Trial Site

    Canton, Ohio 44718
    United States

    Active - Recruiting

  • Clinical Trial Site

    Cleveland, Ohio 44195
    United States

    Active - Recruiting

  • Clinical Trial Site

    Abington, Pennsylvania 19001
    United States

    Active - Recruiting

  • Clinical Trial Site

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

  • Clinical Trial Site

    Dallas, Texas 75243
    United States

    Active - Recruiting

  • Clinical Trial Site

    Houston, Texas 77030
    United States

    Active - Recruiting

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