Actively Recruiting
A Phase 1 Study of ALN-BCAT Alone and With Pembrolizumab in Advanced or Metastatic Hepatocellular Carcinoma
Led by Alnylam Pharmaceuticals · Updated on 2026-06-01
158
Participants Needed
23
Research Sites
4 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating ALN-BCAT alone and in combination with pembrolizumab in patients with advanced or metastatic hepatocellular carcinoma (HCC). The study aims to understand the safety and tolerability of ALN-BCAT, identify recommended doses for further study, and assess the antitumor activity of these treatments. This is a phase 1 clinical trial sponsored by Alnylam Pharmaceuticals. Participants will receive multiple doses of ALN-BCAT either by itself or together with pembrolizumab, both given through intravenous infusion. The study includes a dose escalation phase to find safe dosage levels and a dose expansion phase to further evaluate treatment effects and safety. Both monotherapy and combination therapy arms are included. Throughout the study, participants will be monitored for adverse events and dose-limiting toxicities from the first treatment dose through 30 to 37 days after the final dose. Researchers will assess tumor response using established criteria and measure drug levels in plasma and gene expression changes in tumor samples. The overall study duration varies but includes close safety follow-up after treatment completion.
CONDITIONS
Brief Title
A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Has hepatocellular carcinoma confirmed by biopsy, cytology, or clinical criteria for liver cirrhosis
- Has received at least one systemic therapy for unresectable advanced or metastatic disease
- Has a wingless-related integration site (WNT) pathway activating mutation
- Has liver function classified as Child-Pugh class A or B7
- Is 18 years of age or older
You will not qualify if you...
- Has fibrolamellar hepatocellular carcinoma, sarcomatoid hepatocellular carcinoma, or mixed cholangio-hepatocellular carcinoma tumors
- Has symptomatic disease outside the liver
- Has received anti-cancer therapy or investigational drugs within 3 weeks before the first study drug dose
- Other protocol-defined exclusion criteria may apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - From first dose until 30-37 days after the last dose
Participants receive multiple doses of ALN-BCAT alone or in combination with pembrolizumab administered by intravenous infusion.
Multiple dosing visits for treatment administration
Duration - Up to 30-37 days after the last dose
Participants are monitored for adverse events and antitumor activity after treatment ends.
Follow-up visits during this period
Trial Site Locations
Total: 23 locations
1
Clinical Trial Site
Phoenix, Arizona, United States, 85054
Actively Recruiting
2
Clinical Trial Site
La Jolla, California, United States, 92037
Actively Recruiting
3
Clinical Trial Site
Los Angeles, California, United States, 90033
Actively Recruiting
4
Clinical Trial Site
Jacksonville, Florida, United States, 32224
Actively Recruiting
5
Clinical Trial Site
Atlanta, Georgia, United States, 30322
Actively Recruiting
6
Clinical Trial Site
Chicago, Illinois, United States, 60637
Actively Recruiting
7
Clinical Trial Site
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
8
Clinical Trial Site
Rochester, Minnesota, United States, 55905
Actively Recruiting
9
Clinical Trial Site
New York, New York, United States, 10029
Actively Recruiting
10
Clinical Trial Site
New York, New York, United States, 10032
Completed
11
Clinical Trial Site
Cleveland, Ohio, United States, 44106
Actively Recruiting
12
Clinical Trial Site
Pittsburgh, Pennsylvania, United States, 15232
Actively Recruiting
13
Clinical Trial Site
Dallas, Texas, United States, 75390
Actively Recruiting
14
Clinical Trial Site
Houston, Texas, United States, 77030
Actively Recruiting
15
Clinical Trial Site
San Antonio, Texas, United States, 78229
Actively Recruiting
16
Clinical Trial Site
Richmond, Virginia, United States, 23298
Actively Recruiting
17
Clinical Trial Site
Milan, Italy, 20132
Actively Recruiting
18
Clinical Trial Site
Rozzano, Italy, 20089
Actively Recruiting
19
Clinical Trial Site
Verona, Italy, 37134
Actively Recruiting
20
Clinical Trial Site
Busan, South Korea, 49241
Actively Recruiting
21
Clinical Trial Site
Seongnam, South Korea, 13496
Actively Recruiting
22
Clinical Trial Site
Seongnam, South Korea, 13620
Actively Recruiting
23
Clinical Trial Site
Seoul, South Korea, 03722
Actively Recruiting
Research Team
A
Alnylam Clinical Trial Information Line
A
Alnylam Clinical Trial Information Line
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
4
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