Clinical trial protocol development

Timeframe: 2022 – 2023 Goals: Accelerate development of potential clinical trial Principal Investigator: Mark Yarchoan, MD 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 …

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

Timeframe: 2019 – 2023 Goal: Assess potential to induce immune response with a FLC therapeutic vaccine Principal Investigator: Mark Yarchoan, MD 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 …

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

Timeframe: 2020 – 2023 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 Study overview: This phase I/II trial studies the side effects and how well the combination of nivolumab, fluorouracil, and interferon alpha-2b work …

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

Timeframe: 2023 – 2028 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 Study overview: This phase Ib/2 clinical study, led by Dr. Marina Baretti and Dr. Mark …

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

Timeframe: 2014 – 2018 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 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 …

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Everolimus clinical trial

Timeframe: 2010 – 2014 Goal: Assess potential of everolimus (RAD001) as a treatment option for FLC Principal Investigator: Ghassan Abou-Alfa, MD 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 …

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