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

Last updated: November 10, 2025
Sponsor: Alnylam Pharmaceuticals
Overall Status: Active - Recruiting

Phase

1/2

Condition

Hemophilia

Healthy Volunteers

Treatment

ALN-6400

Placebo

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: ALN-6400
Phase: 1/2
Study Start date:
November 11, 2024
Estimated Completion Date:
June 21, 2028

Connect with a study center

  • Clinical Trial Site

    Montreal,
    Canada

    Site Not Available

  • Clinical Trial Site

    Montreal 6077243,
    Canada

    Active - Recruiting

  • Clinical Trial Site

    Cypress 5341256, California 5332921 90630
    United States

    Active - Recruiting

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