Tests ALN-BCAT alone and with pembrolizumab in advanced liver cancer
Tests ALN-BCAT, alone or with pembrolizumab, in adults with advanced liver cancer.
Plain-language title generated from the registry record
Registry title: A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma
Key facts
- Where
- Milan, Rozzano, and Verona, ItalySee the 3 sites
- Phase
- Phase 1
Where in Italy
- Ospedale San Raffaele S.r.l.Milan, ItalyTaking people
- Humanitas Mirasole S.p.A.Rozzano, ItalyTaking people
- Centro Ricerche Cliniche di Verona s.r.l.Verona, ItalyTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Alnylam Pharmaceuticals
- Trial code
- NCT06600321EU 2024-519499-22-00
- Record updated
- 27 October 2026
- Original description
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.Read full original descriptionShow less
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.
Inclusion: 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: 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; Has symptomatic extrahepatic disease; Has fibrolamellar HCC, sarcomatoid HCC, or mixed cholangio-HCC tumors.