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Fusion Kinase Peptide Vaccine Combined With Nivolumab and Ipilimumab

Trial snapshot

  • Formal name: DNAJB1-PRKACA Fusion Kinase Peptide Vaccine Combined With Nivolumab and Ipilimumab for Patients With Fibrolamellar Hepatocellular Carcinoma
  • Trial type: Specifically designed for fibrolamellar carcinoma
  • Phase: Phase I
  • Recruitment status: Active, not recruiting
  • Eligible ages: 12+
  • Location: Baltimore, MD (Johns Hopkins – Sidney Kimmel Comprehensive Cancer Center)
  • ClinicalTrials.gov ID: NCT04248569

Overview

This phase I clinical trial of a targeted immune therapy for fibrolamellar carcinoma (FLC) is being conducted at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University in Baltimore, MD. The study has been examining whether FLC patients can mount an effective immune response by targeting the unique chimeric protein (produced by the DNAJB1-PRKACA fusion), which drives the growth of most FLC tumors. In the trial, subjects have been given an experimental vaccine (the first patient in the trial received this vaccine on April 21, 2020) containing a peptide (a small segment of a protein) that corresponds to the junction region linking the two parts of the FLC fusion protein. Patients also simultaneously received two FDA-approved drugs, Opdivo (nivolumab) and Yervoy (ipilimumab), that may enhance the immune response against FLC by overcoming “checkpoint” systems that can limit the immune system’s ability to fight a cancer.

The results from the first 12 patients who participated in this trial were published in Nature Medicine in November 2025. As summarized in that publication:

  • The vaccine appeared safe.
  • About 75% of patients developed the desired immune response.
  • Every patient who mounted an immune response achieved at least some period of disease control.
  • Approximately one-third of responders experienced dramatic and durable tumor shrinkage.
  • Those patients have remained cancer-free several years after treatment.

This study was extended with additional funding from FCF. According to the research team, additional patients, treated in the extension of the original trial have shown similar successes.

However, while still an “active” study, this effort is no longer recruiting patients. Instead, patients may want to consider enrolling in a follow-on trial — Peptide Vaccine Combined with DRP-104, Nivolumab and Ipilimumab — which adds the glutamine antagonist DRP-104 to the combination treatment tested in this trial.

Trial eligibility

Enrollment in this trial was open to FLC patients 12 years old and older. The study’s principal investigator is Dr. Mark Yarchoan. More detail can be found at: https://www.clinicaltrials.gov/study/NCT04248569.

Dr. Mark Yarchoan, the principal investigator of this trial, discusses the rationale for the FLC fusion peptide vaccine, the accompanying drug regimen and the eligibility criteria for this first-in-human study in the video below:

Trial contact information:

Please visit clinicaltrials.gov for a comprehensive list of active trials. 

The Fibrolamellar Cancer Foundation does not provide medical advice.  We provide website users with information to help them better understand their health conditions and current approaches towards diagnosis, treatment and supportive care. Always seek the advice of your physician or other qualified healthcare provider.