Phase 1b Study of the Novel GCN2 Kinase Activator NXP800 in Patients With Advanced Cholangiocarcionoma
This phase I trial tests the safety, best dose, and effectiveness of NXP800 in treating patients with cholangiocarcinoma that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced) or that has spread from where it first started (primary site) to other places in the body (metastatic). NXP800 inhibits a pathway called the heat shock factor 1 (HSF1) pathway. The inhibition of this pathway inhibits proliferation, migration, survival, and metastasis in susceptible tumor cells. Overexpressed, amplified and/or overactivated in many cancer cells, HSF1 activates a set of genes that play a key role in tumor initiation, progression and metastasis. Inhibiting this pathway may in turn inhibit tumor initiation, progression, and/or metastasis. Giving NXP800 may be safe, tolerable and/or effective in treating patients with advanced or metastatic cholangiocarcinoma.
• Age ≥ 18 years
• Histologically/cytologically confirmed biliary tract cancer
• Advanced or metastatic disease that is refractory to gemcitabine or fluoropyrimidine based therapy, or if there is intolerance to these regimens
• Measurable disease by RECIST 1.1
• Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0 or 1
• Anticipated life expectancy of \> 12 weeks
• Hemoglobin ≥ 9.0 g/dL (obtained ≤ 14 days prior to registration)
• Absolute neutrophil count (ANC) ≥ 1500/mm\^3 (obtained ≤ 14 days prior to registration)
• Platelet count ≥ 100,000/mm\^3 (obtained ≤ 14 days prior to registration)
• Aspartate aminotransferase (AST) ≤ 5 x upper limit of normal (ULN) (obtained ≤ 14 days prior to registration)
• Alanine aminotransferase (ALT) ≤ 5 x ULN (obtained ≤ 14 days prior to registration)
• Total bilirubin ≤ 1.5 x ULN (obtained ≤ 14 days prior to registration)
• Serum creatinine ≤ 1.5 x ULN (obtained ≤ 14 days prior to registration)
• Provide written informed consent
• Negative pregnancy test done ≤ 7 days prior to registration, for persons of childbearing potential only
⁃ NOTE: If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required
• Willing to use a highly effective method of contraception from the first dose of study medication through 180 days after the last dose of study medication, for persons of childbearing potential or persons able to father a child only
• Willing to return to enrolling institution for follow-up (during the active monitoring phase of the study)