Clinical trial Recruiting at 22 sites Phase 1 Interventional Ages 18 and up

Evaluating ALN-BCAT for liver cancer treatment

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

Run by Alnylam Pharmaceuticals · NCT06600321 · Data updated 2026-11-12

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
Ages18 Years and up
SexAll
Enrollment158 (estimated)
SponsorAlnylam Pharmaceuticals
Industry-sponsored
Drugs / interventionspembrolizumab
Locations23 sites (Phoenix, Arizona and 22 other locations)
StatusRecruiting
StartedDecember 30, 2024
Main results expectedSeptember 30, 2027
Study endsOctober 31, 2027
Registry record updatedOctober 27, 2026
Trial IDNCT06600321 on ClinicalTrials.gov

Is this trial a fit for you?

Likely a good fit

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

Probably 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.

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

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

Who can join: the full rules

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 it's running

  • Clinical Trial Site
    Phoenix, Arizona, United States
    Recruiting
  • Clinical Trial Site
    La Jolla, California, United States
    Recruiting
  • Clinical Trial Site
    Los Angeles, California, United States
    Recruiting
  • Clinical Trial Site
    Jacksonville, Florida, United States
    Recruiting
  • Clinical Trial Site
    Atlanta, Georgia, United States
    Recruiting
  • Clinical Trial Site
    Chicago, Illinois, United States
    Recruiting
Show 17 more locations
  • Clinical Trial Site
    Ann Arbor, Michigan, United States
    Recruiting
  • Clinical Trial Site
    Rochester, Minnesota, United States
    Recruiting
  • Clinical Trial Site
    New York, New York, United States
    Recruiting
  • Clinical Trial Site
    Cleveland, Ohio, United States
    Recruiting
  • Clinical Trial Site
    Pittsburgh, Pennsylvania, United States
    Recruiting
  • Clinical Trial Site
    Dallas, Texas, United States
    Recruiting
  • Clinical Trial Site
    Houston, Texas, United States
    Recruiting
  • Clinical Trial Site
    San Antonio, Texas, United States
    Recruiting
  • Clinical Trial Site
    Richmond, Virginia, United States
    Recruiting
  • Clinical Trial Site
    Milan, Italy
    Recruiting
  • Clinical Trial Site
    Rozzano, Italy
    Recruiting
  • Clinical Trial Site
    Verona, Italy
    Recruiting
  • Clinical Trial Site
    Busan, South Korea
    Recruiting
  • Clinical Trial Site
    Seongnam, South Korea
    Recruiting
  • Clinical Trial Site
    Seongnam, South Korea
    Recruiting
  • Clinical Trial Site
    Seoul, South Korea
    Recruiting
  • Clinical Trial Site
    New York, New York, United States
    Completed

Study contacts

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

How to take part

  1. Review the eligibility criteria above with your treating physician.
  2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
  3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.
Last reviewed 2026-11-14 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.