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DNAJB1-PRKACA Fusion Kinase Peptide Vaccine Combined With Nivolumab and Ipilimumab for Patients With Fibrolamellar Hepatocellular Carcinoma
A Pilot Study of a DNAJB1-PRKACA Fusion Kinase Peptide Vaccine Combined With Nivolumab and Ipilimumab for Patients With Fibrolamellar Hepatocellular Carcinoma
Who can join
Ages 12 and older · All sexes
Full eligibility criteria
Inclusion Criteria for Cohort A, B and C: * Cohort A and B: Must have histologically confirmed FLC (fibrolamellar hepatocellular cancer) that is metastatic or unresectable. * Cohort C: Patients with histologically proven metastatic or unresectable DNAJB1-PRKACA fusion transcript positive solid tumor malignancies, non-FLC solid tumors. * Cohort A and B: Age \> 12 years. Note: Subjects age \> 12 years but \<18 are eligible to enroll only after 6 adult patients have enrolled on the study. * Cohort A and B: Patients \< 18 years old must have a body weight ≥40 kg. * Cohort C: Patients must be Age ≥ 18 years. All Cohorts: * Presence of DNAJB1-PRKACA fusion transcript, assessed by RNA-sequencing, DNA-sequencing, or in situ hybridization in the archival tissue. * ECOG performance status of ≤2 (Karnofsky ≥60%) * Patients must have adequate liver, kidney and marrow function defined by study-specified laboratory tests prior to initial study drug. * Patients must have measurable disease per RECIST 1.1. * Must be willing to provide tissue and blood samples for mandatory translational research. * Woman of childbearing potential must have a negative pregnancy test and follow contraceptive guidelines as defined per protocol. * Men must use acceptable form of birth control while on study. * Ability to understand and willingness to sign a written informed consent document. Exclusion Criteria for Cohorts A, B and C: * Cohort A and C: Patients with a history of prior treatment with checkpoint inhibitors, such as anti-PD-1, anti-PD-L1, anti-PD-L2, anti-CD137, anti-OX-40, anti-CD40, anti-CTLA-4, or anti-LAG-3 antibodies. NOTE: Prior therapy with interferon-alpha is allowed. * Cohort B: Participants a with history of unacceptable, life-threatening toxicity related to prior immune therapy (eg, anti-CTLA-4 or anti-PD-1/PD-L1 treatment, any other antibody or drug specifically targeting T-cell co-stimulation or immune checkpoint pathways) except those that are unlikely to re-occur with standard countermeasures (eg, hormone replacement after endocrinopathy). All Cohorts: * Have had chemotherapy or other systemic therapy or radiotherapy, as follows: * Have had chemotherapy, biological cancer therapy, or radiation 14 days prior to the first dose of study drug. * Have had surgery within 28 days of dosing of investigational agent, excluding minor procedures (dental work, skin biopsy, etc.), celiac plexus block, and biliary stent placement. * Have received other approved or investigational agents or device within 28 days of the first dose of study drug. * Have not recovered from acute adverse events to grade ≤1 or baseline due to agents administered. * Have received any non-oncology live vaccine therapy used for prevention of infectious diseases within 28 days of study treatment * Known sensitivity to or history of allergic reactions to investigational drug (s). * Hypersensitivity reaction to any monoclonal antibody. * Has active autoimmune disease that has required systemic treatment in the past 2 years, or a documented history of clinically severe autoimmune disease, or a syndrome that requires systemic steroids or immunosuppressive agents. * Presence of any tissue or organ allograft, regardless of need for immunosuppression, including corneal allograft. Patients with a history of allogeneic hematopoeitic stem cell transplant will be excluded. * Has a diagnosis of immunodeficiency. * Systemic treatment with either corticosteroids (\>10 mg daily prednisone equivalents) or other immunosuppressive medications within 7 days of study drug administration. * Symptomatic interstitial lung disease. * Has a pulse oximetry of \<92% on room air or is on supplemental home oxygen. * Active or untreated brain metastases or leptomeningeal metastases. * Uncontrolled intercurrent illness including, but not limited to, uncontrolled infection, symptomatic congestive heart failure, unstable angina, cardiac arrhythmia, metastatic cancer, or psychiatric illness/social situations that would limit compliance with study requirements. * Are pregnant or breastfeeding. * Infection with HIV or hepatitis B or C. * Have had evidence of active or acute diverticulitis, intra-abdominal abscess, or GI obstruction. * Unwilling or unable to follow the study schedule for any reason. * Any other sound medical, psychiatric, and/or social reason as determined by the Investigator. * Any illicit drugs or other substance abuse. * Clinically meaningful ascites. Inclusion Criteria for Re-Enrolling Patients: * Patients previously treated with the vaccine targeting the DNAJB1-PRKACA fusion kinase in combination with nivolumab and ipilimumab, who, in the opinion of the principal investigator, had clinical or radiological benefits. * Patients \< 18 years old must have a body weight ≥40 kg. * ECOG performance status of ≤2 (Karnofsky ≥60%, see Appendix A). * Patients must have adequate liver, kidney and marrow function defined by study-specified laboratory tests prior to initial study drug. * Patients must have measurable disease per RECIST 1.1. * Willingness to provide tissue and blood samples for mandatory translational research. * Woman of childbearing potential must have a negative pregnancy test and follow contraceptive guidelines as defined per protocol. * Men must use acceptable form of birth control while on study. * Ability to understand and willingness to sign a written informed consent document. Exclusion Criteria for Re-Enrolling Patients: * Participants with a history of prior unacceptable and/or life-threatening toxicities. * Patients who have had chemotherapy or other systemic therapy or radiotherapy, as follows: * Patients who have had chemotherapy, biological cancer therapy, or radiation 14 days prior to the first dose of study drug. * Patients who have had surgery within 28 days of dosing of investigational agent, excluding minor procedures (dental work, skin biopsy, etc.), celiac plexus block, and biliary stent placement. * Patients who have received other approved or investigational agents or device within 28 days of the first dose of study drug. * Patients who have not recovered from acute adverse events to grade ≤1 or baseline due to agents administered, with exception of alopecia or stable neuropathy, unless approved by the IND Sponsor. * Patients who have received any non-oncology live vaccine therapy used for prevention of infectious diseases within 28 days of study treatment. * Known sensitivity to or history of allergic reactions to investigational drug (s). * Hypersensitivity reaction to any monoclonal antibody. * Has active autoimmune disease that has required systemic treatment in the past 2 years, or a documented history of clinically severe autoimmune disease, or a syndrome that requires systemic steroids or immunosuppressive agents. * Has active autoimmune disease that has required systemic treatment in the past 2 years, or a documented history of clinically severe autoimmune disease, or a syndrome that requires systemic steroids or immunosuppressive agents. * Presence of any tissue or organ allograft, regardless of need for immunosuppression, including corneal allograft. Patients with a history of allogeneic hematopoeitic stem cell transplant will be excluded. * Has a diagnosis of immunodeficiency. * Systemic treatment with either corticosteroids (\>10 mg daily prednisone equivalents) or other immunosuppressive medications within 7 days of study drug administration. * Symptomatic interstitial lung disease. * Has a pulse oximetry of \<92% on room air or is on supplemental home oxygen. * Active or untreated brain metastases or leptomeningeal metastases. * Uncontrolled intercurrent illness including, but not limited to, uncontrolled infection, symptomatic congestive heart failure, unstable angina, cardiac arrhythmia, metastatic cancer, or psychiatric illness/social situations that would limit compliance with study requirements. * Are pregnant or breastfeeding. * Infection with HIV or hepatitis B or C. * Have had evidence of active or acute diverticulitis, intra-abdominal abscess, or GI obstruction. * Unwilling or unable to follow the study schedule for any reason. * Any other sound medical, psychiatric, and/or social reason as determined by the Investigator. * Any illicit drugs or other substance abuse. * Clinically meaningful ascites.
About the study
The primary objective of the trial is the safety and tolerability of administering a vaccine targeting the DNAJB1-PRKACA fusion kinase, in combination with nivolumab and ipilimumab in patients with unresectable or metastatic FLC and with non-FLC solid tumors and to assess the T-cell response.
What is being tested
- DNAJB1-PRKACA peptide vaccine (drug)
- Nivolumab (drug)
- Ipilimumab (drug)
Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · Participants: 56 · Started: Apr 20, 2020
Contact the study team
- Colleen Apostol, RN · Phone: 410-614-3644
- Marina Baretti, MD · Phone: 410-614-1058
Official record on ClinicalTrials.gov — NCT04248569
Locations in the U.S.
| Maryland | Sidney Kimmel Comprehensive Cancer Center, Baltimore |
From ClinicalTrials.gov, data retrieved Oct 2, 2026. Each study sets its own eligibility; the study team decides who can join.