Safety and Efficacy Study of ARQ 501 in Adult Patients With Leiomyosarcoma

A Phase 2 Multi Center Open Label Study of ARQ 501 in Adult Patients With Recurrent, Persistent or Metastatic Leiomyosarcoma

The purpose of this study is to assess the overall response rate (ORR) of persistent, recurrent or metastatic leiomyosarcoma in patients treated with ARQ 501.

Inclusion Criteria:

• Able to provide signed and dated informed consent prior to study-specific screening procedures.
• Histologically or cytologically confirmed leiomyosarcoma that is persistent, recurrent or metastatic.
• Measurable disease as defined by RECIST.
• Karnofsky performance status >= 70%
• Male or female patients of child-producing potential must agree to use contraception or avoidance of pregnancy measures during the study and for 30 days after the last infusion of ARQ 501.
• Hemoglobin (Hgb) >= 10 g/dL.
• Absolute neutrophil count (ANC) >= 1.5 x 10^9/L (>= 1,500/mm3).
• Platelet count >= 100 x 10^9/L (>= 100,000/mm3).
• Total bilirubin less than or equal to 1.5 x upper limit of normal (ULN) or less than or equal to 3.0 x ULN with metastatic liver disease.
• Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) less than or equal to 2.5 x ULN less than or equal to 5.0 ULN with metastatic liver disease.
• Creatinine less than or equal to 1.5 x ULN.

Exclusion Criteria:

• Received three or more prior anticancer chemotherapy regimens.
• Have active, uncontrolled systemic infection considered opportunistic, life threatening or clinically significant at the time of treatment.
• Have received anticancer chemotherapy, immunotherapy, radiotherapy, surgery or investigational agents within four weeks of first infusion.
• Have symptomatic or untreated central nervous system (CNS) involvement.
• Are pregnant or breastfeeding.
• Previous exposure to ARQ 501.