Expanded Access for ABI-009 in Patients With Advanced PEComa and Patients With a Malignancy With Relevant Genetic Mutations or mTOR Pathway Activation

Inclusion criteria

• . a) Diagnosis of advanced (locally advanced and inoperable or metastatic) malignant PEComa confirmed by pathology and immunohistochemistry, whether or not previously exposed to an mTOR inhibitor, or b) diagnosis of any other malignancy with activation in any mTOR pathway component as identified by immunohistochemistry or an identified relevant rare genetic mutation in mTOR pathway genes, including but not limited to TSC1, TSC2, PIK3CA, PTEN, for which there are no FDA-approved treatments or no other comparable or satisfactory alternative therapies available whether or not they been previously exposed to a mTOR inhibitor.
• . 18 years or older, with Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2.
• . Acceptable blood chemistry levels at screening (obtained ≤14 days prior to enrollment (local laboratory) including:
• . total bilirubin ≤1.5 x upper limit of normal (ULN)
• . AST ≤2.5 x ULN (≤5 x ULN if attributable to liver metastases)
• . serum creatinine ≤1.5 x ULN
• . Adequate biological parameters as demonstrated by the following blood counts at screening (obtained ≤14 days prior to enrollment, local laboratory):
• . Absolute neutrophil count (ANC) ≥1.5 × 109/L;

Exclusion criteria