A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma
- Conditions
- Advanced Hepatocellular CarcinomaMetastatic Hepatocellular Carcinoma
- Interventions
- Registration Number
- NCT06600321
- Lead Sponsor
- Alnylam Pharmaceuticals
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 158
- 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
- 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
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Monotherapy: Dose Expansion ALN-BCAT Patients will be administered multiple doses of ALN-BCAT. Monotherapy: Dose Escalation ALN-BCAT Patients will be administered multiple doses of ALN-BCAT. Combination Therapy: Dose Escalation ALN-BCAT Patients will be administered multiple doses of ALN-BCAT in combination with pembrolizumab. Combination Therapy: Dose Expansion ALN-BCAT Patients will be administered multiple doses of ALN-BCAT in combination with pembrolizumab. Combination Therapy: Dose Expansion Pembrolizumab Patients will be administered multiple doses of ALN-BCAT in combination with pembrolizumab. Combination Therapy: Dose Escalation Pembrolizumab Patients will be administered multiple doses of ALN-BCAT in combination with pembrolizumab.
- Primary Outcome Measures
Name Time Method Frequency of Adverse Events (AEs) From the time of first dose of study drug administration to 30-37 days after the last dose Severity of AEs 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) 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) Up to 30-37 Days after the last dose
- Secondary Outcome Measures
Name Time Method Concentrations of ALN-BCAT in Plasma 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)
Percent Change in Gene that Encodes ß-catenin Protein (CTNNB1) Messenger Ribonucleic Acid (mRNA) Expression Comparing Pre- treatment with On-treatment Tumor Samples Up to 30 days Dose Escalation: Antitumor Activity as assessed by RECIST v1.1 Up to 30-37 Days after the last dose
Trial Locations
- Locations (1)
Clinical Trial Site
🇺🇸San Antonio, Texas, United States