--- title: A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma nct_id: NCT06600321 overall_status: RECRUITING phase: PHASE1 sponsor: Alnylam Pharmaceuticals study_type: INTERVENTIONAL primary_condition: Advanced Hepatocellular Carcinoma countries: United States, Italy, South Korea canonical_url: "https://parkinsonspathways.com/agent/trials/NCT06600321.md" clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT06600321" ct_last_update_post_date: 2026-05-01 last_seen_at: "2026-05-12T06:39:20.585Z" source: ClinicalTrials.gov (mirrored, no enrichment) --- # A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma **Official Title:** A Phase 1 Study of ALN-BCAT as Monotherapy and in Combination With Pembrolizumab in Patients With Advanced or Metastatic Hepatocellular Carcinoma **NCT ID:** [NCT06600321](https://clinicaltrials.gov/study/NCT06600321) ## Key Facts - **Status:** RECRUITING - **Phase:** PHASE1 - **Study Type:** INTERVENTIONAL - **Target Enrollment:** 158 - **Lead Sponsor:** Alnylam Pharmaceuticals - **Conditions:** Advanced Hepatocellular Carcinoma, Metastatic Hepatocellular Carcinoma - **Start Date:** 2024-12-30 - **Completion Date:** 2027-10-31 - **CT.gov Last Update:** 2026-05-01 ## 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. ## Eligibility - **Minimum age:** 18 Years - **Sex:** ALL - **Healthy Volunteers:** No ``` 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 ``` ## Arms - **Monotherapy: Dose Escalation** (EXPERIMENTAL) — Patients will be administered multiple doses of ALN-BCAT. - **Monotherapy: Dose Expansion** (EXPERIMENTAL) — Patients will be administered multiple doses of ALN-BCAT. - **Combination Therapy: Dose Escalation** (EXPERIMENTAL) — Patients will be administered multiple doses of ALN-BCAT in combination with pembrolizumab. - **Combination Therapy: Dose Expansion** (EXPERIMENTAL) — Patients will be administered multiple doses of ALN-BCAT in combination with pembrolizumab. ## Interventions - **ALN-BCAT** (DRUG) — Administered by intravenous (IV) infusion - **Pembrolizumab** (DRUG) — Administered by intravenous (IV) infusion ## Primary Outcomes - **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)_ ## 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)_ ## Locations (23) - Clinical Trial Site, Phoenix, Arizona, United States — _RECRUITING_ - Clinical Trial Site, La Jolla, California, United States — _RECRUITING_ - Clinical Trial Site, Los Angeles, California, United States — _RECRUITING_ - Clinical Trial Site, Jacksonville, Florida, United States — _RECRUITING_ - Clinical Trial Site, Atlanta, Georgia, United States — _RECRUITING_ - Clinical Trial Site, Chicago, Illinois, United States — _RECRUITING_ - Clinical Trial Site, Ann Arbor, Michigan, United States — _RECRUITING_ - Clinical Trial Site, Rochester, Minnesota, United States — _RECRUITING_ - Clinical Trial Site, New York, New York, United States — _RECRUITING_ - Clinical Trial Site, New York, New York, United States — _RECRUITING_ - Clinical Trial Site, Cleveland, Ohio, United States — _RECRUITING_ - Clinical Trial Site, Pittsburgh, Pennsylvania, United States — _RECRUITING_ - Clinical Trial Site, Dallas, Texas, United States — _RECRUITING_ - Clinical Trial Site, Houston, Texas, United States — _RECRUITING_ - Clinical Trial Site, San Antonio, Texas, United States — _RECRUITING_ - Clinical Trial Site, Richmond, Virginia, United States — _RECRUITING_ - Clinical Trial Site, Milan, Italy — _RECRUITING_ - Clinical Trial Site, Rozzano, Italy — _RECRUITING_ - Clinical Trial Site, Verona, Italy — _RECRUITING_ - Clinical Trial Site, Busan, South Korea — _RECRUITING_ - Clinical Trial Site, Seongnam, South Korea — _RECRUITING_ - Clinical Trial Site, Seongnam, South Korea — _RECRUITING_ - Clinical Trial Site, Seoul, South Korea — _RECRUITING_ ## Recent Field Changes (last 30 days) - `locations.clinical trial site|new york|new york|united states` — added _(2026-05-12)_ - `status.overallStatus` — added _(2026-05-12)_ - `status.primaryCompletionDate` — added _(2026-05-12)_ - `status.completionDate` — added _(2026-05-12)_ - `status.lastUpdatePostDate` — added _(2026-05-12)_ - `design.phases` — added _(2026-05-12)_ - `design.enrollmentCount` — added _(2026-05-12)_ - `eligibility.criteria` — added _(2026-05-12)_ - `eligibility.minAge` — added _(2026-05-12)_ - `eligibility.sex` — added _(2026-05-12)_ - `outcomes.primary` — added _(2026-05-12)_ - `outcomes.secondary` — added _(2026-05-12)_ - `armsInterventions.arms` — added _(2026-05-12)_ - `armsInterventions.interventions` — added _(2026-05-12)_ - `sponsor.lead` — added _(2026-05-12)_ - `results.hasResults` — added _(2026-05-12)_ - `locations.clinical trial site|phoenix|arizona|united states` — added _(2026-05-12)_ - `locations.clinical trial site|la jolla|california|united states` — added _(2026-05-12)_ - `locations.clinical trial site|los angeles|california|united states` — added _(2026-05-12)_ - `locations.clinical trial site|jacksonville|florida|united states` — added _(2026-05-12)_ - `locations.clinical trial site|atlanta|georgia|united states` — added _(2026-05-12)_ - `locations.clinical trial site|chicago|illinois|united states` — added _(2026-05-12)_ - `locations.clinical trial site|ann arbor|michigan|united states` — added _(2026-05-12)_ - `locations.clinical trial site|rochester|minnesota|united states` — added _(2026-05-12)_ - `locations.clinical trial site|cleveland|ohio|united states` — added _(2026-05-12)_ - `locations.clinical trial site|pittsburgh|pennsylvania|united states` — added _(2026-05-12)_ - `locations.clinical trial site|dallas|texas|united states` — added _(2026-05-12)_ - `locations.clinical trial site|houston|texas|united states` — added _(2026-05-12)_ - `locations.clinical trial site|san antonio|texas|united states` — added _(2026-05-12)_ - `locations.clinical trial site|richmond|virginia|united states` — added _(2026-05-12)_ - `locations.clinical trial site|milan||italy` — added _(2026-05-12)_ - `locations.clinical trial site|rozzano||italy` — added _(2026-05-12)_ - `locations.clinical trial site|verona||italy` — added _(2026-05-12)_ - `locations.clinical trial site|busan||south korea` — added _(2026-05-12)_ - `locations.clinical trial site|seongnam||south korea` — added _(2026-05-12)_ - `locations.clinical trial site|seoul||south korea` — added _(2026-05-12)_ --- *Canonical: https://parkinsonspathways.com/agent/trials/NCT06600321.md* *Source data (authoritative): https://clinicaltrials.gov/study/NCT06600321* *This page is a raw mirror with no AI summary, no editorial enrichment, and no Parkinson's-specific filtering.*