A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma

NCT06600321
clinicaltrials.gov
Phase
Phase 1
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Recruitment status
Recruiting
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Participants
158
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Lead sponsor
Alnylam Pharmaceuticals
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Start date
2024-12-30
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Number of sites
23
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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.
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Short title
A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma
clinicaltrials.gov
Official title
A Phase 1 Study of ALN-BCAT as Monotherapy and in Combination With Pembrolizumab in Patients With Advanced or Metastatic Hepatocellular Carcinoma
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Control arm
Open label
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Registry reports results
No
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Listing at source
Listed
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Recruitment status
Recruiting
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Phase
Phase 1
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Study type
Interventional
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Completion date
2027-10-31
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First posted
2024-09-19
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Registry last updated
2026-06-17
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Primary completion date
2027-09-30
clinicaltrials.gov
Start date
2024-12-30
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Participants
158
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Participant count basis
Estimated
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Planned participants
158
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Protocol amendments
1
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Number of sites
23
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Data monitoring committee
No
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Registry sponsor category
Industry
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Lead sponsor
Alnylam Pharmaceuticals
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Source registry
ClinicalTrials.gov
clinicaltrials.gov

Registry ID
NCT06600321
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ClinicalTrials.gov ID
NCT06600321
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Registry record
Open registry record
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Allocation
Not randomised
Model
Parallel groups
Purpose
Treatment
Masking
No masking
Sources for study design
clinicaltrials.gov

  1. Frequency of Adverse Events (AEs)

    Timeframe: From the time of first dose of study drug administration to 30-37 days after the last dose

  2. Severity of AEs

    Timeframe: From the time of first dose of study drug administration to 30-37 days after the last dose

  3. Dose Escalation: Occurrence of Dose-limiting Toxicities (DLTs)

    Timeframe: From the time of first dose of study drug administration up to 21 days

  4. Dose Expansion: Antitumor Activity as assessed by Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1)

    Timeframe: Up to 30-37 Days after the last dose

  1. Concentrations of ALN-BCAT in Plasma

    Timeframe: Up to the end of the last study drug administration

    Area Under the Plasma Concentration-time Curve (AUC), Maximum Observed Plasma Concentration (Cmax) Time to Maximum Plasma Concentration (Tmax)

  2. Percent Change in Gene that Encodes ß-catenin Protein (CTNNB1) Messenger Ribonucleic Acid (mRNA) Expression Comparing Pre- treatment with On-treatment Tumor Samples

    Timeframe: Up to 30 days

  3. Dose Escalation: Antitumor Activity as assessed by RECIST v1.1

    Timeframe: Up to 30-37 Days after the last dose

Sources for outcomes
clinicaltrials.gov

Sex
All
Age groups
Adults, Older adults
Minimum age
18 Years
Healthy volunteers
No

  • 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

  • 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

Sources for eligibility
clinicaltrials.gov

  • Clinical Trial SitePhoenix, United StatesRecruiting
  • Clinical Trial SiteLa Jolla, United StatesRecruiting
  • Clinical Trial SiteLos Angeles, United StatesRecruiting
  • Clinical Trial SiteJacksonville, United StatesRecruiting
  • Clinical Trial SiteAtlanta, United StatesRecruiting
  • Clinical Trial SiteChicago, United StatesRecruiting
  • Clinical Trial SiteAnn Arbor, United StatesRecruiting
  • Clinical Trial SiteRochester, United StatesRecruiting
  • Clinical Trial SiteNew York, United StatesRecruiting
  • Clinical Trial SiteNew York, United StatesCompleted
  • Clinical Trial SiteCleveland, United StatesRecruiting
  • Clinical Trial SitePittsburgh, United StatesRecruiting
  • Clinical Trial SiteDallas, United StatesRecruiting
  • Clinical Trial SiteHouston, United StatesRecruiting
  • Clinical Trial SiteSan Antonio, United StatesRecruiting
  • Clinical Trial SiteRichmond, United StatesRecruiting
  • Clinical Trial SiteMilan, ItalyRecruiting
  • Clinical Trial SiteRozzano, ItalyRecruiting
  • Clinical Trial SiteVerona, ItalyRecruiting
  • Clinical Trial SiteBusan, South KoreaRecruiting
  • Clinical Trial SiteSeongnam, South KoreaRecruiting
  • Clinical Trial SiteSeongnam, South KoreaRecruiting
  • Clinical Trial SiteSeoul, South KoreaRecruiting
Sources for sites
clinicaltrials.gov

Related records

Drugs (1)

Lead sponsors (1)

Conditions (3)

Targets (1)