A Study of Varespladib Infusion in Subjects With Sickle Cell Disease.

A Randomized, Double-blind, Placebo-Controlled Study With Varespladib Infusion (A-001) in Subjects With Sickle Cell Disease and Vaso-Occlusive Crisis for the Prevention of Acute Chest Syndrome At-Risk Subjects.

The purpose of this study is to determine the effectiveness of A 001 infusion in preventing acute chest syndrome in sickle cell disease (SCD) subjects with vaso-occlusive crisis, fever, and elevated serum C-reactive protein (CRP).

Inclusion Criteria:

• Genotypes Hb SS, Hb SC, sickle β°-thalassemia, sickle β+ -thalassemia
• Pain consistent with vaso-occlusive crisis (clinical judgment) and not attributable to other causes
• Serum CRP ≥5.0 mg/L at time of screening
• Fever defined as oral temperature ≥38.0°C at time of screening
• Age ≥5 years

Exclusion Criteria:

• New or suspected new pulmonary infiltrate diagnosed by chest radiography
• Females who are nursing, pregnant or intend to become pregnant
• Renal dysfunction defined as a creatinine level >1.2 mg/dL for subjects aged 18 or less or a creatinine level >1.5 mg/dL for subjects over the age of 18
• Hepatic dysfunction (alanine aminotransferase [ALT] or aspartate aminotransferase [AST] >3 × upper limit of normal)
• Acute neurologic dysfunction
• Any medical condition for which transfusion may be needed imminently, and/or hemoglobin <5 g/dL
• Red blood cell transfusion within 30 days prior to screening
• Parenteral or oral corticosteroid therapy (inhaled steroids acceptable) within 7 days prior to screening