Clinical trial for Fibrolamellar Hepatocellular Carcinoma (FLC)
Official title DNAJB1-PRKACA Fusion Kinase Peptide Vaccine Combined With Nivolumab and Ipilimumab for Patients With Fibrolamellar Hepatocellular Carcinoma
ClinicalTrials.gov ID: NCT04248569
What this study is testing
What is DNAJB1-PRKACA peptide vaccine?
DNAJB1-PRKACA peptide vaccine is an investigational medicine, being studied as a potential treatment for fibrolamellar hepatocellular carcinoma (flc).
Also referred to as Hiltonol® (Poly-ICLC).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 and older
You may be able to join if
- 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...
- Cohort A and B: Age \> 12 years. Note: people 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.
You likely can't join if
- 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...
- 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...
- 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...
See the full eligibility criteria
Who can join
- 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: people 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.
What rules you out
- 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...
- 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...
- 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...
- 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...
- 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...
- 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...
- 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...
- 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...
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Baltimore, Maryland, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Baltimore, Maryland, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.