Recruiting
Phase 1

ALN-BCAT

Sponsor:

Alnylam Pharmaceuticals

Code:

NCT06600321

Conditions

Advanced Hepatocellular Carcinoma

Metastatic Hepatocellular Carcinoma

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

ALN-BCAT

Pembrolizumab

Study Details

Brief summary:

The purpose of the dose escalation part of the study is to characterize the safety and tolerability of ALN-BCAT as monotherapy and in combination with pembrolizumab; and to determine the recommended dose(s) for expansion (RDFE) of ALN-BCAT as monotherapy and in combination with pembrolizumab. The purpose of the dose expansion part of the of the study is to evaluate the antitumor activity of ALN-BCAT as monotherapy and in combination with pembrolizumab; to characterize the safety and tolerability of ALN-BCAT as monotherapy and in combination with pembrolizumab.

Conditions

Advanced Hepatocellular Carcinoma

Metastatic Hepatocellular Carcinoma

Study ID

NCT06600321

Start date

Dec 30, 2024

Status verified date

Jun, 2026

Completion date

Oct 31, 2027

Anticipated

Primary completion date

Sep 30, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

158 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Monotherapy: Dose Escalation

Patients will be administered multiple doses of ALN-BCAT.

experimental: Monotherapy: Dose Expansion

Patients will be administered multiple doses of ALN-BCAT.

experimental: Combination Therapy: Dose Escalation

Patients will be administered multiple doses of ALN-BCAT in combination with pembrolizumab.

experimental: Combination Therapy: Dose Expansion

Patients will be administered multiple doses of ALN-BCAT in combination with pembrolizumab.

Interventions

ALN-BCAT

Administered by intravenous (IV) infusion

Pembrolizumab

Administered by intravenous (IV) infusion

Primary outcome measure

  • Frequency of Adverse Events (AEs) [ Time Frame: From the time of first dose of study drug administration to 30-37 days after the last dose ]
  • Severity of AEs [ Time Frame: From the time of first dose of study drug administration to 30-37 days after the last dose ]
  • Dose Escalation: Occurrence of Dose-limiting Toxicities (DLTs) [ Time Frame: From the time of first dose of study drug administration up to 21 days ]
  • Dose Expansion: Antitumor Activity as assessed by Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1) [ Time Frame: Up to 30-37 Days after the last dose ]

Central Contacts and Locations

Central contacts

Alnylam Clinical Trial Information Line

1-877-ALNYLAMclinicaltrials@alnylam.com

Alnylam Clinical Trial Information Line

1-877-256-9526clinicaltrials@alnylam.com

Locations

Clinical Trial Site

Recruiting

Phoenix, Arizona, United States, 85054

Clinical Trial Site

Recruiting

La Jolla, California, United States, 92037

Clinical Trial Site

Recruiting

Los Angeles, California, United States, 90033

Clinical Trial Site

Recruiting

Jacksonville, Florida, United States, 32224

Clinical Trial Site

Recruiting

Atlanta, Georgia, United States, 30322

Clinical Trial Site

Recruiting

Chicago, Illinois, United States, 60637

Clinical Trial Site

Recruiting

Ann Arbor, Michigan, United States, 48109

Clinical Trial Site

Recruiting

Rochester, Minnesota, United States, 55905

Clinical Trial Site

Recruiting

New York, New York, United States, 10029

Clinical Trial Site

Recruiting

Cleveland, Ohio, United States, 44106

Clinical Trial Site

Recruiting

Pittsburgh, Pennsylvania, United States, 15232

Clinical Trial Site

Recruiting

Dallas, Texas, United States, 75390

Clinical Trial Site

Recruiting

Houston, Texas, United States, 77030

Clinical Trial Site

Recruiting

San Antonio, Texas, United States, 78229

Clinical Trial Site

Recruiting

Richmond, Virginia, United States, 23298

More Information

Sponsor

Alnylam Pharmaceuticals

Last update posted

Jun 17, 2026

Last verified

Jun, 2026

Keywords

  • Hepatocellular carcinoma (HCC)
  • Pembrolizumab
  • CTNNB1
  • Liver disease
  • Liver cancer
  • Liver neoplasms
  • Carcinoma, hepatocellular
  • WNT pathway activating

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Alnylam Pharmaceuticals on 2026-06-17.