Phase 2 Study of MP4CO to Treat Vaso-occlusive Sickle Crisis
Status:
Withdrawn
Trial end date:
2015-10-01
Target enrollment:
Participant gender:
Summary
Sickle Cell disease is caused by an inherited hemoglobin disorder. Healthy red blood cells
are discoid and can deform and move through small blood vessels to carry oxygen to all parts
of the body. In Sickle Cell disease, as red blood cells circulate and oxygen is released, the
deoxygenated abnormal Hemoglobin S can begin to polymerize and cause red cells to become
sticky and elongated. These "sickled" red cells are less flexible and will obstruct small
blood vessels and prevent normal red cells from circulating freely, which limits oxygen
delivery to tissues and organs. This is known as a "sickling crisis" or "vaso-occlusive
crisis" and is the leading cause of hospitalization in patients with Sickle Cell disease.
Patients suffering from a sickle crisis experience severe pain and are at risk of stroke,
heart attack or even death. Current therapy is limited to hydration and symptomatic pain
relief. The administration of MP4CO as an adjunct treatment to standard therapy may alleviate
pain associated with a sickling crisis and potentially reduce the severity and duration of a
crisis. This may shorten the time in hospital and potentially improve the quality of life for
patients with sickle cell anemia.