A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma
A Phase 1 Study of ALN-BCAT as Monotherapy and in Combination With Pembrolizumab in Patients With Advanced or Metastatic Hepatocellular Carcinoma
1 other identifier
interventional
158
3 countries
23
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_1
Started Dec 2024
Typical duration for phase_1
23 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 13, 2024
CompletedFirst Posted
Study publicly available on registry
September 19, 2024
CompletedStudy Start
First participant enrolled
December 30, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 30, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
October 31, 2027
May 1, 2026
April 1, 2026
2.8 years
September 13, 2024
April 29, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (4)
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 Outcomes (3)
Concentrations of ALN-BCAT in Plasma
Up to the end of the last study drug administration
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
Study Arms (4)
Monotherapy: Dose Escalation
EXPERIMENTALPatients will be administered multiple doses of ALN-BCAT.
Monotherapy: Dose Expansion
EXPERIMENTALPatients will be administered multiple doses of ALN-BCAT.
Combination Therapy: Dose Escalation
EXPERIMENTALPatients will be administered multiple doses of ALN-BCAT in combination with pembrolizumab.
Combination Therapy: Dose Expansion
EXPERIMENTALPatients will be administered multiple doses of ALN-BCAT in combination with pembrolizumab.
Interventions
Administered by intravenous (IV) infusion
Administered by intravenous (IV) infusion
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (23)
Clinical Trial Site
Phoenix, Arizona, 85054, United States
Clinical Trial Site
La Jolla, California, 92037, United States
Clinical Trial Site
Los Angeles, California, 90033, United States
Clinical Trial Site
Jacksonville, Florida, 32224, United States
Clinical Trial Site
Atlanta, Georgia, 30322, United States
Clinical Trial Site
Chicago, Illinois, 60637, United States
Clinical Trial Site
Ann Arbor, Michigan, 48109, United States
Clinical Trial Site
Rochester, Minnesota, 55905, United States
Clinical Trial Site
New York, New York, 10029, United States
Clinical Trial Site
New York, New York, 10032, United States
Clinical Trial Site
Cleveland, Ohio, 44106, United States
Clinical Trial Site
Pittsburgh, Pennsylvania, 15232, United States
Clinical Trial Site
Dallas, Texas, 75390, United States
Clinical Trial Site
Houston, Texas, 77030, United States
Clinical Trial Site
San Antonio, Texas, 78229, United States
Clinical Trial Site
Richmond, Virginia, 23298, United States
Clinical Trial Site
Milan, 20132, Italy
Clinical Trial Site
Rozzano, 20089, Italy
Clinical Trial Site
Verona, 37134, Italy
Clinical Trial Site
Busan, 49241, South Korea
Clinical Trial Site
Seongnam, 13496, South Korea
Clinical Trial Site
Seongnam, 13620, South Korea
Clinical Trial Site
Seoul, 03722, South Korea
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Director
Alnylam Pharmaceuticals
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 13, 2024
First Posted
September 19, 2024
Study Start
December 30, 2024
Primary Completion (Estimated)
September 30, 2027
Study Completion (Estimated)
October 31, 2027
Last Updated
May 1, 2026
Record last verified: 2026-04
Data Sharing
- IPD Sharing
- Will not share