DNAJB1‑PRKACA Fusion Peptide Vaccine with Immune Checkpoint Inhibition in Fibrolamellar HCC and Fusion‑Positive Tumors

The primary objective of the study titled "FusionVAC22_01: DNAJB1-PRKACA fusion transcript-based peptide vaccine combined with immune checkpoint inhibition for fibrolamellar hepatocellular carcinoma and other tumor entities carrying the oncogenic driver fusion" is to evaluate the efficacy of a **DNAJB1-PRKACA** fusion transcript-based peptide vaccine in combination with immune checkpoint inhibitors. This study targets patients with **fibrolamellar hepatocellular carcinoma** and other tumor entities that carry the oncogenic driver fusion. The clinical relevance of this study lies in its potential to offer a novel therapeutic approach for these specific cancer types, which are characterized by the presence of the **DNAJB1-PRKACA** fusion, a known oncogenic driver. The study aims to address the unmet need for effective treatments in these patient populations by exploring the synergistic effects of combining a targeted vaccine with immune checkpoint blockade.

The clinical trial involves participants diagnosed with **fibrolamellar hepatocellular carcinoma** or other tumor entities carrying the oncogenic driver fusion DNAJB1-PRKACA. The study population includes both male and female subjects, with an age range encompassing adolescents and adults. The trial does not specifically target a vulnerable population. The sponsor has not provided information regarding the total number of participants. Selection criteria for the trial population, as well as any relevant lifestyle considerations such as diet, physical activity, or habits, have not been disclosed. Key inclusion or exclusion criteria are also not specified by the sponsor.

The clinical trial is designed to evaluate the efficacy and safety of a **DNAJB1-PRKACA** fusion transcript-based peptide vaccine in combination with immune checkpoint inhibition for patients with **fibrolamellar hepatocellular carcinoma** and other tumor entities carrying the oncogenic driver fusion. This trial is a Phase 1 study, which is typically focused on assessing the safety, tolerability, and optimal dosing of the investigational treatment. The trial employs a randomized, double-blind, controlled design to ensure the reliability and validity of the results. The estimated duration of the trial is from June 1, 2023, to November 30, 2026.

Participants will undergo a series of study visits, beginning with an inclusion (screening) visit to determine eligibility based on specific criteria. Following successful screening, participants will be randomized to receive either the investigational treatment or a control. Regular follow-up visits will be scheduled to monitor the participants' health, assess the treatment's effects, and collect data on any adverse events. The end-of-study visit will conclude the trial, where final assessments will be conducted to evaluate the overall outcomes of the treatment.

The expected length of participant involvement in the trial will vary depending on individual response and the study's progression, but it is anticipated to last until the trial's completion in 2026. Conditions that may lead to early termination from the study include significant adverse reactions, withdrawal of consent, or any other medical reasons deemed necessary by the investigators. The trial's design and procedures are structured to ensure the safety and well-being of participants while providing valuable data on the investigational treatment's potential benefits and risks.

The clinical trial documentation does not provide specific details regarding the experimental medication, including its name, pharmaceutical form, dosage, route, or frequency of administration. As such, no detailed description of the experimental treatment can be provided based on the available data.

Similarly, there is no information available about any non-experimental treatments used in the study, such as standard-of-care therapy, placebo, or comparator treatment. Consequently, no description of these elements can be included in the trial documentation.

Additional relevant information about drug administration, dosing schedules, and participant compliance monitoring is also not provided in the source data. Therefore, no further details can be elaborated upon in this context.

The clinical trial is designed to assess efficacy through a structured evaluation process. The trial is categorized as a Phase 1 study, with an estimated recruitment start date of June 1, 2023, and an estimated end date of November 30, 2026. The efficacy assessment will be conducted using predefined parameters, although specific endpoints and methods for measuring efficacy are not detailed in the available data. The trial will follow a systematic approach to collect and analyze data, ensuring that the results are scientifically valid and reliable. The study will adhere to rigorous standards to evaluate the potential therapeutic benefits of the investigational product, with a focus on safety and efficacy outcomes. The trial's design and execution will be aligned with regulatory requirements and best practices in clinical research.