Background
death, fatigue, telangiectasia, HCC, un-resectable hepatocellular carcinoma
ADVERSE EVENTS, THROMBOCYTOPENIA, SOLID TUMORS
Immune checkpoint inhibitors (ICIs) targeting the programmed cell death-1 (PD1)/programmed cell death ligand-1 (PDL1) pathway constituted the backbone of systemic therapy for patients with un-resectable hepatocellular carcinoma (HCC) [GT90001 (previously known as PF-03446962) is a monoclonal antibody targeting the activin receptor-like kinase 1 (ALK-1), a serine/threonine kinase receptor regulating angiogenesis through interaction with the transforming growth factor-β (TGFβ) signaling network [GT90001 has been evaluated in several early-phase clinical trials for patients with advanced solid tumors. The recommended dosage for phase 2 trials was 7 mg/kg intravenously every 2 weeks. The most common treatment-related adverse events demonstrated in these trials of GT90001 included thrombocytopenia, fatigue, and telangiectasia, which were also distinct from adverse associated with VEGFR inhibition [Based on the above evidence, we conducted a phase 1b/2 study of GT90001 combined with nivolumab to test the hypothesis that GT90001 may improve the therapeutic efficacy of anti-PD1 ICI therapy for patients with advanced HCC. This study consisted of a dose de-escalation stage to determine the recommended phase 2 dosage (RP2D) of GT-90001 in combination with the anti-PD1 nivolumab and a dose expansion stage to evaluate the safety and efficacy of GT-90001- nivolumab combination (Fig. Study profile. 