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

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

Recruiting
18 years and older
All
Phase 1

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Overview

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

Inclusion Criteria:

  • Has HCC confirmed histologically or cytologically, or, for patients with liver cirrhosis, 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 or metastatic disease
  • Has at least one wingless-related integration site (WNT)-pathway activating mutation
  • Child-Pugh class A or B7

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 the first dose of study drug

Note: other protocol defined inclusion / exclusion criteria apply

Study details
    Advanced Hepatocellular Carcinoma
    Metastatic Hepatocellular Carcinoma

NCT06600321

Alnylam Pharmaceuticals

22 July 2025

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