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

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

Phase

1

Condition

Liver Cancer

Carcinoma

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

    Los Angeles, California 90033
    United States

    Active - Recruiting

  • Clinical Trial Site

    San Diego, California 92093
    United States

    Active - Recruiting

  • Clinical Trial Site

    Chicago, Illinois 60637
    United States

    Active - Recruiting

  • Clinical Trial Site

    Ann Arbor, Michigan 48109
    United States

    Active - Recruiting

  • Clinical Trial Site

    New York, New York 10029
    United States

    Active - Recruiting

  • Clinical Trial Site

    Cleveland, Ohio 44106
    United States

    Active - Recruiting

  • Clinical Trial Site

    Pittsburgh, Pennsylvania 15232
    United States

    Active - Recruiting

  • Clinical Trial Site

    Dallas, Texas 75390
    United States

    Active - Recruiting

  • Clinical Trial Site

    Houston, Texas 77030
    United States

    Active - Recruiting

  • 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

    Active - Recruiting

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