A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)

Last updated: December 18, 2025
Sponsor: Alnylam Pharmaceuticals
Overall Status: Active - Recruiting

Phase

1/2

Condition

Hemophilia

Healthy Volunteers

Treatment

Placebo

ALN-6400

Clinical Study ID

NCT06659640
ALN-6400-001
2025-522510-23-00
  • Ages 18-65
  • All Genders
  • Accepts Healthy Volunteers

Study Summary

The purpose of this study is to:

  • evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single ascending doses of ALN-6400 in healthy volunteers

  • evaluate the efficacy, safety, tolerability and PD of multiple doses of ALN-6400 in adult patients with HHT

Eligibility Criteria

Inclusion

Inclusion Criteria -

Part A:

  • Is a healthy adult volunteer

Part B:

  • Is an adult patient with a clinical diagnosis of HHT

Exclusion Criteria -

Part A:

  • Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > upper limit of normal (ULN)

  • Has known human immunodeficiency virus (HIV) infection; or known current or chronic hepatitis C virus or hepatitis B virus infection

  • Has an estimated glomerular filtration (eGFR) of <90 mL/min/1.73m^2 at screening

Part B:

  • Has ALT or AST >2×ULN

  • Has total bilirubin >1.5×ULN

  • Has eGFR of <30 mL/min/1.73m^2 at screening

Parts A and B:

  • Is not willing to comply with the contraceptive requirements during the study period

Note: other protocol defined inclusion / exclusion criteria apply

Study Design

Total Participants: 120
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 1/2
Study Start date:
November 11, 2024
Estimated Completion Date:
June 22, 2028

Connect with a study center

  • Clinical Trial Site

    Camperdown 2172563, New South Wales 2155400 2050
    Australia

    Site Not Available

  • Clinical Trial Site

    Parkville 2153770, Victoria 2145234 3050
    Australia

    Site Not Available

  • Clinical Trial Site

    Vancouver 6173331, British Columbia 5909050 V6Z 1Y6
    Canada

    Site Not Available

  • Clinical Trial Site

    Toronto 6167865, Ontario 6093943 M5B 1W8
    Canada

    Site Not Available

  • Clinical Trial Site

    Montreal 6077243, Quebec 6115047 H3P 3P1
    Canada

    Active - Recruiting

  • Clinical Trial Site

    Montreal,
    Canada

    Site Not Available

  • Clinical Trial Site

    Bron 3029931, Lyon 69500
    France

    Site Not Available

  • Clinical Trial Site

    Bourdeaux 3031069, 33000
    France

    Site Not Available

  • Clinical Trial Site

    Homburg 2899449, 66421
    Germany

    Site Not Available

  • Clinical Trial Site

    L'Hospitalet de Llobregat 3120619, B 8907
    Spain

    Site Not Available

  • Clinical Trial Site

    Birmingham 4049979, Alabama 4829764 35233
    United States

    Site Not Available

  • Clinical Trial Site

    Cypress 5341256, California 5332921 90630
    United States

    Active - Recruiting

  • Clinical Trial Site

    Gainesville 4156404, Florida 4155751 32608
    United States

    Site Not Available

  • Clinical Trial Site

    Boston 4930956, Massachusetts 6254926 02114
    United States

    Site Not Available

  • Clinical Trial Site

    Rochester 5043473, Minnesota 5037779 55905
    United States

    Site Not Available

  • Clinical Trial Site

    St Louis 4407066, Missouri 4398678 63110
    United States

    Site Not Available

  • Clinical Trial Site

    Chapel Hill 4460162, North Carolina 4482348 27514
    United States

    Site Not Available

  • Clinical Trial Site

    Cleveland 5150529, Ohio 5165418 44195
    United States

    Site Not Available

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