A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorAlnylam Pharmaceuticals
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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
Trial design
Design model
Parallel
Treatments tested in this trial
ALN-BCAT
DrugAdministered by intravenous (IV) infusion
Pembrolizumab
DrugAdministered by intravenous (IV) infusion
Treatment groups
158 Participants
are divided into 4 treatment groupsGroup A: Monotherapy: Dose EscalationExperimental treatment 1 intervention
Group B: Monotherapy: Dose ExpansionExperimental treatment 1 intervention
Group C: Combination Therapy: Dose EscalationExperimental treatment 2 interventions
Group D: Combination Therapy: Dose ExpansionExperimental treatment 2 interventions
Trial outcomes
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 dose2
Severity of AEs
Time frame
From the time of first dose of study drug administration to 30-37 days after the last dose3
Dose Escalation: Occurrence of Dose-limiting Toxicities (DLTs)
Time frame
From the time of first dose of study drug administration up to 21 days4
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 doseSecondary outcomes
1
Concentrations of ALN-BCAT in Plasma
Area Under the Plasma Concentration-time Curve (AUC), Maximum Observed Plasma Concentration (Cmax) Time to Maximum Plasma Concentration (Tmax)
Time frame
Up to the end of the last study drug administration2
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 days3
Dose Escalation: Antitumor Activity as assessed by RECIST v1.1
Time frame
Up to 30-37 Days after the last doseSponsors and contacts
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Source ClinicalTrials.gov