A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma

Last updated: February 13, 2026
Sponsor: Alnylam Pharmaceuticals
Overall Status: Active - Recruiting

Phase

1

Condition

Liver Cancer

Carcinoma

Primary Biliary Cholangitis

Treatment

ALN-BCAT

Pembrolizumab

Clinical Study ID

NCT06600321
ALN-BCAT-001
  • Ages > 18
  • All Genders

Study Summary

The purpose of the dose escalation part of the study is to characterize the safety and tolerability of ALN-BCAT as monotherapy and in combination with pembrolizumab; and to determine the recommended dose(s) for expansion (RDFE) of ALN-BCAT as monotherapy and in combination with pembrolizumab. The purpose of the dose expansion part of the of the study is to evaluate the antitumor activity of ALN-BCAT as monotherapy and in combination with pembrolizumab; to characterize the safety and tolerability of ALN-BCAT as monotherapy and in combination with pembrolizumab.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Has HCC confirmed histologically or cytologically, or, for patients with livercirrhosis, clinically by the American Association for the Study of Liver Diseases (AASLD) criteria

  • Has had at least one line of systemic therapy for unresectable advanced ormetastatic disease

  • Has at least one wingless-related integration site (WNT)-pathway activating mutation

  • Child-Pugh class A or B7

Exclusion

Exclusion Criteria:

  • Has fibrolamellar HCC, sarcomatoid HCC, or mixed cholangio-HCC tumors

  • Has symptomatic extrahepatic disease

  • Has received anti-cancer therapy or investigational drugs ≤3 weeks prior to thefirst dose of study drug

Note: other protocol defined inclusion / exclusion criteria apply

Study Design

Total Participants: 158
Treatment Group(s): 2
Primary Treatment: ALN-BCAT
Phase: 1
Study Start date:
December 30, 2024
Estimated Completion Date:
October 31, 2027

Connect with a study center

  • Clinical Trial Site

    Milan 6951411, 20132
    Italy

    Active - Recruiting

  • Clinical Trial Site

    Rozzano 3168837, 20089
    Italy

    Active - Recruiting

  • Clinical Trial Site

    Verona 3164527, 37134
    Italy

    Active - Recruiting

  • Clinical Trial Site

    Busan 1838524, 49241
    South Korea

    Active - Recruiting

  • Clinical Trial Site

    Seongnam 6876792, 13496
    South Korea

    Active - Recruiting

  • Clinical Trial Site

    Seongnam-si 1897000, 13496
    South Korea

    Site Not Available

  • Clinical Trial Site

    Seoul 1835848, 03722
    South Korea

    Active - Recruiting

  • Clinical Trial Site

    Phoenix 5308655, Arizona 5551752 85054
    United States

    Active - Recruiting

  • Clinical Trial Site

    Los Angeles, California 90033
    United States

    Site Not Available

  • Clinical Trial Site

    San Diego, California 92093
    United States

    Site Not Available

  • Clinical Trial Site

    La Jolla 5363943, California 5332921 92037
    United States

    Active - Recruiting

  • Clinical Trial Site

    Los Angeles 5368361, California 5332921 90033
    United States

    Active - Recruiting

  • Clinical Trial Site

    San Diego 5391811, California 5332921 92093
    United States

    Site Not Available

  • Clinical Trial Site

    Jacksonville 4160021, Florida 4155751 32224
    United States

    Active - Recruiting

  • Clinical Trial Site

    Atlanta 4180439, Georgia 4197000 30322
    United States

    Active - Recruiting

  • Clinical Trial Site

    Chicago, Illinois 60637
    United States

    Site Not Available

  • Clinical Trial Site

    Chicago 4887398, Illinois 4896861 60637
    United States

    Active - Recruiting

  • Clinical Trial Site

    Ann Arbor, Michigan 48109
    United States

    Site Not Available

  • Clinical Trial Site

    Ann Arbor 4984247, Michigan 5001836 48109
    United States

    Active - Recruiting

  • Clinical Trial Site

    Rochester 5043473, Minnesota 5037779 55905
    United States

    Active - Recruiting

  • Clinical Trial Site

    New York, New York 10032
    United States

    Site Not Available

  • Clinical Trial Site

    New York 5128581, New York 5128638 10029
    United States

    Active - Recruiting

  • Clinical Trial Site

    Cleveland, Ohio 44106
    United States

    Site Not Available

  • Clinical Trial Site

    Cleveland 5150529, Ohio 5165418 44106
    United States

    Active - Recruiting

  • Clinical Trial Site

    Pittsburgh, Pennsylvania 15232
    United States

    Site Not Available

  • Clinical Trial Site

    Pittsburgh 5206379, Pennsylvania 6254927 15232
    United States

    Active - Recruiting

  • Clinical Trial Site

    Dallas, Texas 75390
    United States

    Site Not Available

  • Clinical Trial Site

    Houston, Texas 77030
    United States

    Site Not Available

  • MD Anderson

    Houston, Texas 77030
    United States

    Active - Recruiting

  • Clinical Trial Site

    San Antonio, Texas 78229
    United States

    Active - Recruiting

  • South Texas Accelerated Research Therapeutics (START)

    San Antonio, Texas 78229
    United States

    Site Not Available

  • Clinical Trial Site

    Dallas 4684888, Texas 4736286 75390
    United States

    Active - Recruiting

  • Clinical Trial Site

    Houston 4699066, Texas 4736286 77030
    United States

    Active - Recruiting

  • Clinical Trial Site

    San Antonio 4726206, Texas 4736286 78229
    United States

    Active - Recruiting

  • Clinical Trial Site

    Richmond 4781708, Virginia 6254928 23298
    United States

    Active - Recruiting

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