Advanced Hepatocellular CarcinomaPhase 1NCT06600321
A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma
A Phase 1 study testing ALN-BCAT and Pembrolizumab in people with Advanced Hepatocellular Carcinoma and Metastatic Hepatocellular Carcinoma, run by Alnylam Pharmaceuticals at 23 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Advanced Hepatocellular Carcinoma, Metastatic Hepatocellular Carcinoma
- What is being tested?
- ALN-BCAT and Pembrolizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 158 participants
- Where
- 23 sitesin 3 countries
- Study dates
- 30 Dec 2024to 31 Oct 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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 locations and contacts
23 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
23 sites in 3 countries
United States16 sites, 15 recruiting
- Recruiting
Clinical Trial Site
Ann Arbor, Michigan, 48109
- Recruiting
Clinical Trial Site
Atlanta, Georgia, 30322
- Recruiting
Clinical Trial Site
Chicago, Illinois, 60637
- Recruiting
Clinical Trial Site
Cleveland, Ohio, 44106
- Recruiting
Clinical Trial Site
Dallas, Texas, 75390
- Recruiting
Clinical Trial Site
Houston, Texas, 77030
- Recruiting
Clinical Trial Site
Jacksonville, Florida, 32224
- Recruiting
Clinical Trial Site
La Jolla, California, 92037
- Recruiting
Clinical Trial Site
Los Angeles, California, 90033
- Recruiting
Clinical Trial Site
New York, New York, 10029
- Completed
Clinical Trial Site
New York, New York, 10032
- Recruiting
Clinical Trial Site
Phoenix, Arizona, 85054
- Recruiting
Clinical Trial Site
Pittsburgh, Pennsylvania, 15232
- Recruiting
Clinical Trial Site
Richmond, Virginia, 23298
- Recruiting
Clinical Trial Site
Rochester, Minnesota, 55905
- Recruiting
Clinical Trial Site
San Antonio, Texas, 78229
South Korea4 sites, 4 recruiting
- Recruiting
Clinical Trial Site
Busan, 49241
- Recruiting
Clinical Trial Site
Seongnam, 13496
- Recruiting
Clinical Trial Site
Seongnam, 13620
- Recruiting
Clinical Trial Site
Seoul, 03722
Italy3 sites, 3 recruiting
- Recruiting
Clinical Trial Site
Milan, 20132
- Recruiting
Clinical Trial Site
Rozzano, 20089
- Recruiting
Clinical Trial Site
Verona, 37134
About this study
Official title: A Phase 1 Study of ALN-BCAT as Monotherapy and in Combination With Pembrolizumab in Patients With Advanced or Metastatic 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.
- Being tested
- ALN-BCAT, Pembrolizumab
- Keywords
- Hepatocellular carcinoma (HCC), Pembrolizumab, CTNNB1, Liver disease, Liver cancer, Liver neoplasms, Carcinoma, hepatocellular, WNT pathway activating
- Medical subject headings
- Carcinoma, Hepatocellular, Liver Diseases, Liver Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALN-BCATDrug
Administered by intravenous (IV) infusion
PembrolizumabDrug
Also called Keytruda, MK-3475
Administered by intravenous (IV) infusion
| Study group | Role | What they receive |
|---|---|---|
| Monotherapy: Dose Escalation | Experimental | Patients will be administered multiple doses of ALN-BCAT. Receives: Drug: ALN-BCAT |
| Monotherapy: Dose Expansion | Experimental | Patients will be administered multiple doses of ALN-BCAT. Receives: Drug: ALN-BCAT |
| Combination Therapy: Dose Escalation | Experimental | Patients will be administered multiple doses of ALN-BCAT in combination with pembrolizumab. Receives: Drug: ALN-BCAT; Drug: Pembrolizumab |
| Combination Therapy: Dose Expansion | Experimental | Patients will be administered multiple doses of ALN-BCAT in combination with pembrolizumab. Receives: Drug: ALN-BCAT; Drug: Pembrolizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of Adverse Events (AEs)
Measured: From the time of first dose of study drug administration to 30-37 days after the last dose
Severity of AEs
Measured: 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)
Measured: 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)
Measured: Up to 30-37 Days after the last dose
Secondary outcomes (3)
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)
Measured: 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
Measured: Up to 30 days
Dose Escalation: Antitumor Activity as assessed by RECIST v1.1
Measured: Up to 30-37 Days after the last dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
- Study start30 Dec 2024
- Primary completion30 Sep 2027 (estimated)
- Study completion31 Oct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Advanced Hepatocellular CarcinomaStudies of the same condition, open and under way.
- More trials run by Alnylam PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 158 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06600321
- Sponsor's protocol ID
- ALN-BCAT-001
- Status last verified
- Jun 2026
- First submitted
- 13 Sep 2024
- Last updated
- 17 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study of ALN-BCAT as Monotherapy and in Combination With Pembrolizumab in Patients With Advanced or Metastatic Hepatocellular Carcinoma
- ClinicalTrials.gov ID
- NCT06600321
- Acronym / sponsor ID
- ALN-BCAT-001
- Keywords
- Hepatocellular carcinoma (HCC), Pembrolizumab, CTNNB1, Liver disease, Liver cancer, Liver neoplasms, Carcinoma, hepatocellular, WNT pathway activating
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 158 participants (estimated)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 30 Dec 2024 (actual)
- Primary completion
- 30 Sep 2027 (estimated)
- Study completion
- 31 Oct 2027 (estimated)
- First posted
- 19 Sep 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Monotherapy: Dose Escalation | Experimental | Drug: ALN-BCATPatients will be administered multiple doses of ALN-BCAT. |
| Monotherapy: Dose Expansion | Experimental | Drug: ALN-BCATPatients will be administered multiple doses of ALN-BCAT. |
| Combination Therapy: Dose Escalation | Experimental | Drug: ALN-BCAT, Drug: PembrolizumabPatients will be administered multiple doses of ALN-BCAT in combination with pembrolizumab. |
| Combination Therapy: Dose Expansion | Experimental | Drug: ALN-BCAT, Drug: PembrolizumabPatients will be administered multiple doses of ALN-BCAT in combination with pembrolizumab. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ALN-BCATAdministered by intravenous (IV) infusion | Drug | Combination Therapy: Dose Escalation, Combination Therapy: Dose Expansion, Monotherapy: Dose Escalation, Monotherapy: Dose Expansion |
| PembrolizumabAlso: Keytruda, MK-3475Administered by intravenous (IV) infusion | Drug | Combination Therapy: Dose Escalation, Combination Therapy: Dose Expansion |
Primary outcome
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
Severity of AEs
Time frame: 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)
Time frame: 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)
Time frame: Up to 30-37 Days after the last dose
3 secondary outcomes
Concentrations of ALN-BCAT in Plasma
Time frame: 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
Time frame: Up to 30 days
Dose Escalation: Antitumor Activity as assessed by RECIST v1.1
Time frame: Up to 30-37 Days after the last dose
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jun 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Medical Director study director
Alnylam Pharmaceuticals
23 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ALN-BCAT and Pembrolizumab in people with Advanced Hepatocellular Carcinoma and Metastatic Hepatocellular Carcinoma, run by Alnylam Pharmaceuticals at 23 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 17 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in United States, South Korea, Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Alnylam Clinical Trial Information Line (1-877-ALNYLAM, clinicaltrials@alnylam.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Jun 2026.