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

Alnylam Pharmaceuticals

Status and phase: Enrolling; Phase 1

Conditions: Metastatic Hepatocellular Carcinoma; Advanced Hepatocellular Carcinoma

Treatments: Drug: Pembrolizumab; Drug: ALN-BCAT

Study type: Interventional

Funder types: Industry

Identifiers

NCT06600321

ALN-BCAT-001

Details and patient eligibility

About

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.

Enrollment: 158 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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158 participants in 4 patient groups

Monotherapy: Dose Escalation

Experimental group

Description:

Patients will be administered multiple doses of ALN-BCAT.

Treatment:

Drug: ALN-BCAT

Monotherapy: Dose Expansion

Experimental group

Description:

Patients will be administered multiple doses of ALN-BCAT.

Treatment:

Drug: ALN-BCAT

Combination Therapy: Dose Escalation

Experimental group

Description:

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

Treatment:

Drug: ALN-BCAT

Drug: Pembrolizumab

Combination Therapy: Dose Expansion

Experimental group

Description:

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

Treatment:

Drug: ALN-BCAT

Drug: Pembrolizumab