BTX-A51 for the Treatment of Patients with Metastatic and/or Recurrent Liposarcoma

Inclusion Criteria

• Study participants must have histologically-confirmed metastatic and/or recurrent liposarcoma (limited to the subtypes of well-differentiated and/or dedifferentiated liposarcoma, which are pathognominically associated with MDM2 amplifications), or myxoid liposarcoma), or CIC-rearranged sarcoma
• Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
• White blood cell (WBC) ≥ 3000/mm^3
• Platelets ≥ 75,000ul
• Absolute neutrophil count (ANC) ≥ 1500ul
• Hemoglobin (Hgb) ≥ 9g/dl
• Creatinine ≤ 1.5 x upper limit of normal (ULN) or measured creatinine clearance (CrCl) of ≥ 60ml/m^2/1.73m^2
• Total bilirubin ≤ 2 x ULN
• Aspartate aminotransferase (AST)/alanine aminotransferase (ALT) ≤ 3 x ULN
• Participants must have measurable disease, defined as at least one lesion that can be accurately measured in at least one dimension (longest diameter to be recorded for non-nodal lesions and short axis for nodal lesions) as ≥ 20 mm (≥ 2 cm) by chest x-ray or as ≥ 10 mm (≥ 1 cm) with CT scan, MRI, or calipers by clinical exam
• Patients must have recovered from toxicity related to prior therapy to grade =< 1 (defined by Common Terminology Criteria for Adverse Events [CTCAE] v5.0) (except alopecia and neuropathy, or immunotherapy related hypothyroidism)
• As the effect of this study drug on the developing human fetus is not known, women of child-bearing potential and men must agree to use at least 2 methods of contraception (abstinence; hormonal or barrier method of birth control) for the study and at least 2 months after completion
• Female patient of childbearing potential has a negative serum pregnancy test
• Ability to understand and the willingness to sign a written informed consent document
• Age ≥ 18 years
• Patients must have completed all prior anti-cancer treatment for liposarcoma, including radiation, ≥ 14 days prior to registration

Exclusion Criteria

• Patient with current evidence of active and uncontrolled infection, New York Heart Association (NYHA) class III-IV congestive heart failure (CHF), documented Child’s class B-C cirrhosis, or uncontrolled medical disease which in the opinion of the investigator or the sponsor could compromise safety and/or assessment of efficacy
• Active infection with hepatitis C virus (HCV) or hepatitis B virus (HBV); subjects who are positive for hepatitis B core antibody, hepatitis B surface antigen, or hepatitis C antibody must have a negative polymerase chain reaction (PCR) result before first dose of study drug; those who are PCR positive will be excluded
• Major surgical procedure or open surgical biopsy within 28 days of first dose of study drug
• Active central nervous system (CNS) disease involvement, or prior history of National Cancer Institute (NCI) CTCAE grade ≥ 3 drug-related CNS toxicity. Subject with known CNS metastases that are treated and stable (without evidence of CNS toxicity) and are not requiring systemic steroids are allowed to be enrolled
• Patient has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial
• Myocardial infarction within 12 months of screening
• Use of any other concurrent investigational agents or anticancer agents, excluding hormonal therapy for breast or prostate cancer
• Pregnant women are excluded from this study because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with BTX-A51, breastfeeding should be discontinued if the mother is treated with BTX-A51
• Inability to swallow pills or inadequate gastrointestinal (GI) absorption in the opinion of the treating investigator