A Study of the Efficacy and Safety of ICA-17043 (With or Without Hydroxyurea) in Patients With Sickle Cell Anemia.

A Phase II, Multicenter, Twelve-Week, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Range-Finding Study of the Efficacy and Safety of ICA-17043 With or Without Hydroxyurea Therapy in Patients With Sickle Cell Anemia

ICA-17043 is being developed for the chronic treatment of patients with sickle cell disease (SCD) in both adults and children. ICA-17043 is a potent and specific inhibitor of a channel in human red blood cells (RBCs) that blocks RBC dehydration. ICA-17043 is expected to inhibit RBC dehydration and thus should prevent or delay the sickling process. By reducing sickled cells, an improvement in anemia, a reduction in painful crises, and ultimately, less end-organ disease is anticipated.

Inclusion Criteria:

• Homozygous (HbSS) Sickle Cell Anemia
• Otherwise healthy (based on medical history, physical examination, 12-lead ECG, and clinical laboratory tests)
• Patients may be receiving hydroxyurea, but must have been dose stabilized for at least 3 months
• Patient has a history of at least one acute vaso-occlusive event requiring hospitalization

Exclusion Criteria:

• Patient participating in a chronic transfusion program
• Patient having a total hemoglobin of < 4.0 g/dL or > 10.0 g/dL
• Patient having a HbA > 10%
• Patient considering undergoing an elective surgery
• Patient taking prohibited medications such as Epoetin, Warfarin, etc.
• Patient who has had previous gastrointestinal surgery, except cholecystectomy or appendectomy
• Patient with significant active cardiovascular, neurologic, endocrine, hepatic, or renal disorders unrelated to sickle cell anemia