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Date:2/5/2008

nd markers of RBC destruction (hemolysis) in SCD patients. The investigators also sought to obtain additional safety data and identify the optimal dose for a phase III study. The primary efficacy endpoint was the change in blood hemoglobin level from baseline to end of study, and secondary endpoints included markers of hemolysis, changes in RBC count and indices, and frequency of painful events or "crises."

"An understanding of the pathophysiology of sickle cell disease is extremely important in order to identify new therapeutic targets," said Kenneth Ataga, MD, of the Division of Hematology/Oncology at the University of North Carolina at Chapel Hill and lead author of the study.

A total of 90 eligible patients were enrolled in the trial. Of these participants, 24 were also taking hydroxyurea, a drug already approved for the treatment of SCD. The patients were randomized into three treatment arms: placebo (n=30), low-dose senicapoc (6 mg/day, n=29), and high-dose senicapoc (10 mg/day, n=31). Safety and efficacy assessments were obtained at the end of week one and then every two weeks until completion of the study treatment.

Patients treated with senicapoc achieved measurable improvements in hemoglobin levels. Patients in the high-dose senicapoc arm achieved a hemoglobin increase of 0.68 g/dL (vs. 0.01 g/dL for patients taking placebo). The team also found corresponding increases in hematocrit and RBC count in the patients taking senicapoc when compared to placebo. Notably, the sub-groups of patients taking hydroxyurea achieved similar results. In the low-dose group, the increase in hemoglobin compared to placebo was not statistically significant.

Treatment with senicapoc also produced dose-dependent and statistically significant improvements in secondary endpoints, including the percentage of dense RBCs (-2.41 vs. -0.08, high-dose vs. placebo), reticulocytes (-4.12 vs. -0.46), and lactate dehydrogenase (-121 vs. -15), a marker of
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SOURCE American Society of Hematology Copyright©2008 PR Newswire. All rights reserved

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