Clinical Trials

Yvonne Janssen-Heininger, Ph.D.

New medical tests and treatments aren’t offered to the public as soon as they’re discovered. They need to be appropriately studied to determine their safety and effectiveness. Clinical trials are formal research studies to evaluate a medical treatment. Clinical trials can study many things, including:

  • New drugs not yet approved by the U.S. Food and Drug Administration (FDA)
  • New uses of drugs already approved by the FDA
  • New ways to administer drugs, such as in pill form
  • New tests to find and track disease.

Clinical trials typically advance through several phases when testing a new treatment:

  • Phase I trials test an experimental treatment on a very small group of people to assess its safety and side effects and to determine appropriate drug dosages.
  • Phase II trials enroll a larger group and focus on determining effectiveness of the treatment for individuals with a certain disease or condition. They also continue to study safety, including short-term side effects.
  • Phase III trials obtain additional information about safety and effectiveness, studying different populations and dosages, often using the drug in combination with other drugs. If the FDA decides that the Phase III results are positive, it will approve the treatment for clinical use.

In this video, Ghassan Abou-Alfa, M.D., a medical oncologist and FCF Advisor, discusses the importance of clinical trials for fibrolamellar patients.

Active clinical trials include:

Trials targeting fibrolamellar

  • DNAJB1-PRKACA Fusion Kinase Peptide Vaccine Combined With Nivolumab and Ipilimumab for Patients With Fibrolamellar Hepatocellular Carcinoma (Baltimore, MD)

    A new clinical trial of an immune therapy for fibrolamellar carcinoma (FLC) is now recruiting subjects at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University in Baltimore, MD. The study asks if individuals can mount an effective immune response against FLC by specifically targeting the unique chimeric protein (resulting from a DNAJB1-PRKACA gene fusion) believed to drive the growth of almost all such tumors. Trial subjects will be given an experimental vaccine (the first patient in the trial received this vaccine on April 21st) containing a peptide (small segment of a protein) that corresponds to the junction region linking the two parts of the chimeric protein. They will simultaneously receive 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 study’s principal investigator is Dr. Mark Yarchoan. Details and contact information can be found at:

Trials potentially open to fibrolamellar patients

  • Neratinib HER Mutation Basket Study (62 study locations)

    This is an open-label, multicenter, multinational, Phase 2 study exploring the efficacy and safety of neratinib therapy in patients with solid tumors with activating HER2, HER3 or EGFR mutations or with EGFR gene amplification. The trial will consist of a screening period, a treatment period, and an end of treatment visit occurring when neratinib is discontinued for any reason, a safety follow-up visit occurring 28 to 42 days after the last dose of neratinib and a survival follow-up period lasting for a maximum of 12 months for each patient after their last dose of neratinib or until initiation of additional anti-cancer therapy. For more information go to:

  • NCI Clinical Trial (multiple locations)

    The National Cancer Institute has launched a Molecular Analyses for Therapy Choice (MATCH) Clinical Trial, exploring the effectiveness of targeted cancer therapy based on genetic mutation rather than type of cancer. While the study is not testing any drugs known to target the principal genetic mutation involved in fibrolamellar, patients may be able to participate if their genetic profile includes a mutation targeted in the study  Please click the link below for details and talk to your doctor to see if this is right for you. Learn More

Please visit 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.