Evaluation of Different Dose Regimens of Aes-103 Given for 28 Days to Subjects With Stable Sickle Cell Disease
Status:
Terminated
Trial end date:
2015-03-16
Target enrollment:
Participant gender:
Summary
Sickle cell disease (SCD) is a genetic blood disorder characterized by the presence of
sickle-shaped red blood cells. In the U.S. and the U.K. this occurs primarily in persons of
African origin. There is only one drug (hydroxyurea) approved to manage SCD, but it is not
fully efficacious and can produce medically significant side effects. Aes-103 is being
evaluated as a novel agent for the long term management of SCD. By directly reducing the
sickling process, Aes-103 has a different mechanism of action than hydoxyurea. The active
ingredient in Aes-103 is 5-hydroxymethyl furfural, a naturally occurring small molecule that
is chemically related to glucose.
This study will evaluate the safety and pharmacokinetic profile of two dosing regimens of
Aes-103 for up to 28 days in up to 50 adult subjects with stable SCD compared with subjects
receiving placebo.