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NCT Number: NCT06600321

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

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.

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Key information

Conditions

Advanced Hepatocellular Carcinoma Adenocarcinoma Carcinoma Carcinoma, Hepatocellular Digestive System Diseases Digestive System Neoplasms Liver Diseases Liver Neoplasms Metastatic Hepatocellular Carcinoma Neoplasms Neoplasms by Histologic Type Neoplasms by Site Neoplasms, Glandular and Epithelial

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Clinical Trial Site, Milan, Italy

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

ALN-BCAT

Drug

Administered by intravenous (IV) infusion

Pembrolizumab

Drug

Administered by intravenous (IV) infusion

Other names: Keytruda, MK-3475

Primary outcomes

  1. 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

  2. Severity of AEs

    Time frame: From the time of first dose of study drug administration to 30-37 days after the last dose

  3. Dose Escalation: Occurrence of Dose-limiting Toxicities (DLTs)

    Time frame: From the time of first dose of study drug administration up to 21 days

  4. 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

Secondary outcomes

  1. Concentrations of ALN-BCAT in Plasma

    Time frame: Up to the end of the last study drug administration

    Area Under the Plasma Concentration-time Curve (AUC), Maximum Observed Plasma Concentration (Cmax) Time to Maximum Plasma Concentration (Tmax)

  2. Percent Change in Gene that Encodes ß-catenin Protein (CTNNB1) Messenger Ribonucleic Acid (mRNA) Expression Comparing Pre- treatment with On-treatment Tumor Samples

    Time frame: Up to 30 days

  3. Dose Escalation: Antitumor Activity as assessed by RECIST v1.1

    Time frame: Up to 30-37 Days after the last dose

Study contacts

Contact information is provided by the study sponsor or research team.

Alnylam Clinical Trial Information Line

CONTACT

[email protected]

1-877-ALNYLAM

Alnylam Clinical Trial Information Line

CONTACT

[email protected]

1-877-256-9526

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
Sep 19, 2024
Registry last updated
Jun 17, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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