A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma

NCT ID: NCT06600321

Last Updated: 2025-11-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

158 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-12-30

Study Completion Date

2027-10-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Advanced Hepatocellular Carcinoma Metastatic Hepatocellular Carcinoma

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

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

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Monotherapy: Dose Escalation

Patients will be administered multiple doses of ALN-BCAT.

Group Type EXPERIMENTAL

ALN-BCAT

Intervention Type DRUG

Administered by intravenous (IV) infusion

Monotherapy: Dose Expansion

Patients will be administered multiple doses of ALN-BCAT.

Group Type EXPERIMENTAL

ALN-BCAT

Intervention Type DRUG

Administered by intravenous (IV) infusion

Combination Therapy: Dose Escalation

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

Group Type EXPERIMENTAL

ALN-BCAT

Intervention Type DRUG

Administered by intravenous (IV) infusion

Pembrolizumab

Intervention Type DRUG

Administered by intravenous (IV) infusion

Combination Therapy: Dose Expansion

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

Group Type EXPERIMENTAL

ALN-BCAT

Intervention Type DRUG

Administered by intravenous (IV) infusion

Pembrolizumab

Intervention Type DRUG

Administered by intravenous (IV) infusion

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

ALN-BCAT

Administered by intravenous (IV) infusion

Intervention Type DRUG

Pembrolizumab

Administered by intravenous (IV) infusion

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Keytruda MK-3475

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are 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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Alnylam Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Medical Director

Role: STUDY_DIRECTOR

Alnylam Pharmaceuticals

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Clinical Trial Site

Phoenix, Arizona, United States

Site Status RECRUITING

Clinical Trial Site

Los Angeles, California, United States

Site Status RECRUITING

Clinical Trial Site

San Diego, California, United States

Site Status RECRUITING

Clinical Trial Site

Jacksonville, Florida, United States

Site Status RECRUITING

Clinical Trial Site

Atlanta, Georgia, United States

Site Status RECRUITING

Clinical Trial Site

Chicago, Illinois, United States

Site Status RECRUITING

Clinical Trial Site

Ann Arbor, Michigan, United States

Site Status RECRUITING

Clinical Trial Site

Rochester, Minnesota, United States

Site Status RECRUITING

Clinical Trial Site

New York, New York, United States

Site Status RECRUITING

Clinical Trial Site

New York, New York, United States

Site Status RECRUITING

Clinical Trial Site

Cleveland, Ohio, United States

Site Status RECRUITING

Clinical Trial Site

Pittsburgh, Pennsylvania, United States

Site Status RECRUITING

Clinical Trial Site

Dallas, Texas, United States

Site Status RECRUITING

Clinical Trial Site

Houston, Texas, United States

Site Status RECRUITING

Clinical Trial Site

San Antonio, Texas, United States

Site Status RECRUITING

Clinical Trial Site

Richmond, Virginia, United States

Site Status RECRUITING

Clinical Trial Site

Verona, , Italy

Site Status RECRUITING

Clinical Trial Site

Busan, , South Korea

Site Status RECRUITING

Clinical Trial Site

Seongnam-si, , South Korea

Site Status RECRUITING

Clinical Trial Site

Seoul, , South Korea

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

United States Italy South Korea

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Alnylam Clinical Trial Information Line

Role: CONTACT

Phone: 1-877-ALNYLAM

Email: [email protected]

Alnylam Clinical Trial Information Line

Role: CONTACT

Phone: 1-877-256-9526

Email: [email protected]

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

ALN-BCAT-001

Identifier Type: -

Identifier Source: org_study_id