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
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years and up |
| Sex | All |
| Enrollment | 158 (estimated) |
| Sponsor | Alnylam Pharmaceuticals Industry-sponsored |
| Drugs / interventions | pembrolizumab |
| Locations | 23 sites (Phoenix, Arizona and 22 other locations) |
| Status | Recruiting |
| Started | December 30, 2024 |
| Main results expected | September 30, 2027 |
| Study ends | October 31, 2027 |
| Registry record updated | October 27, 2026 |
| Trial ID | NCT06600321 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
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Clinical Trial Site
Phoenix, Arizona, United States Recruiting -
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La Jolla, California, United States Recruiting -
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Los Angeles, California, United States Recruiting -
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Jacksonville, Florida, United States Recruiting -
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Atlanta, Georgia, United States Recruiting -
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Chicago, Illinois, United States Recruiting
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Ann Arbor, Michigan, United States Recruiting -
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Rochester, Minnesota, United States Recruiting -
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New York, New York, United States Recruiting -
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Cleveland, Ohio, United States Recruiting -
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Pittsburgh, Pennsylvania, United States Recruiting -
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Dallas, Texas, United States Recruiting -
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Houston, Texas, United States Recruiting -
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San Antonio, Texas, United States Recruiting -
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Richmond, Virginia, United States Recruiting -
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Milan, Italy Recruiting -
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Rozzano, Italy Recruiting -
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Verona, Italy Recruiting -
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Busan, South Korea Recruiting -
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Seongnam, South Korea Recruiting -
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Seongnam, South Korea Recruiting -
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Seoul, South Korea Recruiting -
Clinical Trial Site
New York, New York, United States Completed
Study contacts
- Study coordinator: Alnylam Clinical Trial Information Line
- Email: clinicaltrials@alnylam.com
- Phone: 1-877-ALNYLAM
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.