Clinical trials Archives - Fibrolamellar Cancer Foundation

Exploiting a Critical Vulnerability to Glutamine Antimetabolite Therapy in Fibrolamellar Hepatocellular Carcinoma (FLC)

Goals: Assess the potential of an inhibitor of glutamine metabolism (DRP-104; sirpiglenastat) in combination with an immune checkpoint inhibitor (durvalumab) as a treatment option for unresectable disease Principal Investigators: Marina Baretti, MD and Mark Yarchoan, MD Grant length: Five years Study overview: This phase Ib/2 clinical study, led by Dr. Marina Baretti and Dr. Mark …

A therapeutic cancer vaccine plus ipilimumab and nivolumab for fibrolamellar hepatocellular carcinoma (FLC)

Goal: Assess potential to induce immune response with a FLC therapeutic vaccine Principal Investigator: Mark Yarchoan, MD and Marina Baretti, MD Grant length: Two years Study overview: This grant allows Dr. Mark Yarchoan and Dr. Marina Baretti of Johns Hopkins University to keep JHU’s successful “peptide vaccine” clinical trial open to enrollment by fibrolamellar patients. …

DT2216 in Combination with Irinotecan: A Phase 1 Study with a Phase 2 Feasibility Cohort for Fibrolamellar Carcinoma

Goal: Provide the drug necessary to enable a FLC clinical trial Principal Investigators of related clinical study: Dr. Allison O’Neill, Dana-Farber Cancer Institute and Dr. Michael Ortiz, Memorial Sloan Kettering Cancer Center Grant length: Three years Effort overview: This grant funds the manufacturing and distribution of an experimental drug, DT2216, for a clinical trial for …

Clinical trial protocol development

Goals: Accelerate development of potential clinical trial Principal Investigator: Mark Yarchoan, MD Grant length: Six months Study overview: This grant to Dr. Yarchoan is meant to support the development of a protocol for a potential clinical trial. In 2020, the Johns Hopkins team began a phase I clinical trial of an experimental vaccine containing a …

A Pilot Study of a DNAJB1-PRKACA fusion kinase vaccine combined with nivolumab and ipilimumab for patients with fibrolamellar carcinoma (FLC)

Goal: Assess potential to induce immune response with a FLC therapeutic vaccine Principal Investigator: Mark Yarchoan, MD Grant length: Two years Study overview: Many patients with FLC currently receive an immune checkpoint inhibitor off-label. These drugs “take the brakes off” the immune system, enhancing its ability to fight cancer. However, clinical experience suggests that they …

A phase I/II study of nivolumab plus 5-fluorouracil plus interferon-α2b for unresectable fibrolamellar hepatocellular carcinoma

Goals: Assess the potential of the combination of nivolumab, fluorouracil, and interferon alpha-2b as a treatment option for unresectable disease Principal Investigators: Sunyoung Lee, MD and Ahmed Kaseb, MD Grant length: Two years plus extension Study overview: This phase I/II trial studies the side effects and how well the combination of nivolumab, fluorouracil, and interferon …

FusionVAC22_02: DNAJB1-PRKACA fusion transcript-based peptide vaccine for fibrolamellar hepatocellular carcinoma patients

Goals: Assess the potential of a FLC therapeutic vaccine to prevent disease recurrence Principal Investigators: Juliane S. Walz, MD, CCU Translational Immunology, Department of Internal Medicine Grant length: Five years Study Background and Overview: Various research efforts supported by FCF over the last 6 years have shown that immunotherapy approaches can save FLC patients’ lives. …

Sequential related donor partial liver followed by bone marrow transplantation for treatment of extensive, liver-confined fibrolamellar hepatocellular carcinoma (FL-HCC)

Goals: Develop and implement a clinical trial for combined liver and bone marrow transplantation as a treatment for liver-confined FLC Principal Investigator: Ephraim Fuchs, MD Grant length: Three years Study overview: This grant supported the development of a phase II, single center study to assess the feasibility and effectiveness of combining bone marrow transplantation and …

Everolimus clinical trial

Goal: Assess potential of everolimus (RAD001) as a treatment option for FLC Principal Investigator: Ghassan Abou-Alfa, MD Grant length: Three years Study background and overview: At the time of this trial, the molecular drive of fibrolamellar, the DNAJB1-PRKACA fusion gene, had not been identified. However, several factors suggested that estrogen and estrogen-dependent pathways may play …