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Evaluating ALN-BCAT for liver cancer treatment

A Phase 1 Study of ALN-BCAT as Monotherapy and in Combination With Pembrolizumab in Patients With Advanced or Metastatic Hepatocellular Carcinoma

Phase 1 Interventional Alnylam Pharmaceuticals · NCT06600321

This study is testing a new treatment called ALN-BCAT, alone and with another drug, to see if it can safely help people with advanced liver cancer.

Quick facts

PhasePhase 1
Study typeInterventional
Enrollment158 (estimated)
Ages18 Years and up
SexAll
SponsorAlnylam Pharmaceuticals Industry-sponsored
Drugs / interventionspembrolizumab
Locations23 sites (Phoenix, Arizona and 22 other locations)
Trial IDNCT06600321 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the safety and tolerability of ALN-BCAT, both alone and in combination with pembrolizumab, in patients with advanced or metastatic hepatocellular carcinoma (HCC). The study consists of a dose escalation phase to determine the recommended doses for further evaluation and a dose expansion phase to investigate the antitumor activity of the treatment. Patients will be monitored for safety and efficacy throughout the trial.

Who should consider this trial

Good fit: Ideal candidates include patients with histologically confirmed hepatocellular carcinoma who have received prior systemic therapy and have specific genetic mutations.

Not a fit: Patients with fibrolamellar HCC, symptomatic extrahepatic disease, or those who have recently received anti-cancer therapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with advanced liver cancer.

How similar studies have performed: Other studies have shown promise with similar approaches in targeting liver cancer, but this specific combination is novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
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

Where this trial is running

Phoenix, Arizona and 22 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Advanced Hepatocellular CarcinomaMetastatic Hepatocellular CarcinomaHepatocellular carcinomaPembrolizumabCTNNB1Liver diseaseLiver cancerLiver neoplasms
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.