Phase 1 Study of ALN-BCAT Alone or with Pembrolizumab in Adults With Advanced or Metastatic Liver Cancer Hepatocellular Carcinoma

What this Trial Is About

Researchers are evaluating ALN-BCAT as a treatment for patients with advanced or metastatic hepatocellular carcinoma who have specific genetic mutations and have received prior systemic therapy. The study aims to understand the safety, tolerability, and antitumor activity of ALN-BCAT both alone and combined with pembrolizumab. This is a Phase 1 clinical trial sponsored by Alnylam Pharmaceuticals. Participants will receive multiple intravenous doses of ALN-BCAT either as monotherapy or in combination with pembrolizumab. The study includes two parts dose escalation to determine safe and tolerable dose levels, and dose expansion to further evaluate the antitumor effects at recommended doses. Treatments are administered through IV infusions and follow a parallel group design. During the trial, participants will be closely monitored for adverse events and dose-limiting toxicities from the first dose up to about one month after the last dose. Researchers will assess tumor response using standardized criteria and measure drug concentrations and gene expression changes related to the treatment. The total study duration extends until late 2027, with safety and efficacy evaluations throughout.

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